Market Newsdesk

SOUTH SAN FRANCISCO, Calif., May 08, 2023 (GLOBE NEWSWIRE) — 23andMe Holding Co. (Nasdaq: ME) (“23andMe”), a leading human genetics and biopharmaceutical company, today announced the appointment of Reza Afkhami as its Chief Corporate Development Officer overseeing corporate development efforts across its therapeutics, research services and consumer groups. Afkhami will be responsible for spearheading and driving a wide range of industry collaborations for the Company.

23andMe Therapeutics has a pipeline of genetically validated discovery and clinical programs, which it intends to advance through collaborations with pharmaceutical companies. Furthermore, 23andMe hopes to help the pharmaceutical industry improve its R&D productivity through genetically-driven target identification and portfolio prioritization. Beyond that work, the Company plans to support patient recruitment and disease awareness campaigns. 23andMe is pursuing strategic collaborations in digital health and wellness, clinical testing and data collection that will continue to enhance its consumer offerings and best in class direct-to-consumer preventive health services. The ultimate goal of these future collaborations will be to form an ecosystem of consumers, healthcare professionals, and drug developers who can together leverage the genetic revolution to dramatically improve human health.

Before joining 23andMe, Afkhami was Senior Vice President of Corporate Development and Strategy at Global Blood Therapeutics, a clinical-stage biopharmaceutical company
focused on innovative therapies for the treatment of sickle cell disease. He led business development, licensing, and strategy for the company. He also played a key role in Pfizer’s acquisition of Global Blood Therapeutics in late 2022.

“Reza comes to 23andMe at a perfect time as we sunset the exclusive discovery period of our collaboration with GSK in July and begin exploring new partnerships that leverage our unique genetic insights,” said Anne Wojcicki, Chief Executive Officer and Co-Founder of 23andMe. “Reza’s vast experience in the biotech world complements our world class research and therapeutics development expertise.”

Afkhami brings over 20 years of corporate strategy and business development experience across the therapeutics industry. Prior to Global Blood Therapeutics, Afkhami served as Vice President of Corporate Development and Strategy at Surrozen, a biotechnology company focused on developing tissue-specific antibodies to create therapies for a range of diseases. He has held numerous positions overseeing global commercial strategy and planning, led teams handling business development and market intelligence, and managed and supported partnerships to help to grow a company from early-stage drug discovery to a fully integrated biopharmaceutical company.

“I’m excited by the innovative approach that 23andMe brings to drug discovery and development, and the power that genetics can play in leading us to new medical treatments,” said Afkhami. “I look forward to helping make 23andMe’s mission a reality by allowing many more consumers and patients to truly benefit from the human genome.”

Afkhami holds a Bachelor of Science (BS) degree from Georgetown University, and a Master of Business Administration (MBA) degree from the University of California, Berkeley.

About 23andMe

23andMe is a genetics-led consumer healthcare and therapeutics company empowering a healthier future. For more information, please visit www.23andMe.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, including, without limitation, statements regarding the future performance of 23andMe’s businesses in consumer genetics and therapeutics and the growth and potential of its proprietary research platform. All statements, other than statements of historical fact, included or incorporated in this press release, including statements regarding 23andMe’s strategy, financial position, funding for continued operations, cash reserves, projected costs, plans, and objectives of management, are forward-looking statements. The words “believes,” “anticipates,” “estimates,” “plans,” “expects,” “intends,” “may,” “could,” “should,” “potential,” “likely,” “projects,” “predicts,” “continue,” “will,” “schedule,” and “would” or, in each case, their negative or other variations or comparable terminology, are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements are predictions based on 23andMe’s current expectations and projections about future events and various assumptions. 23andMe cannot guarantee that it will actually achieve the plans, intentions, or expectations disclosed in its forward-looking statements and you should not place undue reliance on 23andMe’s forward-looking statements. These forward-looking statements involve a number of risks, uncertainties (many of which are beyond the control of 23andMe), or other assumptions that may cause actual results or performance to differ materially from those expressed or implied by these forward-looking statements. The forward-looking statements contained herein are also subject generally to other risks and uncertainties that are described from time to time in the company’s filings with the Securities and Exchange Commission, including under Item 1A, “Risk Factors” in the company’s most recent Annual Report on Form 10-K and in its subsequent reports on Forms 10-Q and 8-K. The statements made herein are made as of the date of this press release and, except as may be required by law, 23andMe undertakes no obligation to update them, whether as a result of new information, developments, or otherwise.

Contacts:

Investor Relations Contact: [email protected]
Media Contact: [email protected]

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MELVILLE, N.Y., May 08, 2023 (GLOBE NEWSWIRE) — BioRestorative Therapies, Inc . (“BioRestorative”, “BRTX” or the “Company”) (NASDAQ: BRTX ) , a clinical stage company focused on stem cell-based therapies, today announced that it has signed a clinical trial agreement with Northwell Health, New York State’s largest health care provider and private employer, and Northwell’s Feinstein Institutes for Medical Research. The signing of the agreement will allow Northwell Health to participate in BioRestorative Therapies ongoing Phase 2 clinical trial targeting chronic lumber disc disease (cLDD).

BRTX-100, the Company’s lead clinical candidate, is a novel cell-based therapeutic engineered to target areas of the body that have little blood flow. BRTX-100 is currently being evaluated in connection with a Phase 2 clinical trial to treat cLDD. The BRTX-100 approach involves an office based injection procedure which introduces the patient’s own cells which have been uniquely expanded and conditioned to result in therapeutic impact within the harsh environment of the intervertebral disc and for an all too common pain which is otherwise not reliably treated. The trial is prospective, randomized, double-blinded and controlled. The trial will evaluate the safety and preliminary efficacy of a single dose of BRTX-100. A total of up to 99 eligible patients will be randomized at up to 15 clinical sites in the United States to receive either the investigational drug (BRTX-100) or control in a 2:1 fashion. BioRestorative recently completed enrollment for the safety run-in phase of its Phase 2 trial.

Lance Alstodt, Chief Executive Officer of BioRestorative, stated, “Partnering with Northwell Health and Northwell’s Feinstein Institutes for Medical Research, the largest hospital system and health provider in New York State, is a testament to the strength of our corporate profile, our technology and the prospects for our product candidate, BRTX 100. We are delighted to enter into such an agreement with Northwell and their world class team of non-surgical and surgical spine clinicians and researchers across the disciplines. We hope to identify other areas where our two organizations can work closely with one another. Research is a very obvious area of focus for both of us and we look forward to exploring strategic possibilities to enhance and strengthen our relationship with Northwell.”

Dr. Jeffry Beer, of Northwell, stated “Chronic low back pain remains one of the most common and difficult clinical conditions we encounter as spine specialists. We don’t currently have highly effective treatment options. The current phase 2 FDA trial is meticulously designed to provide us greater insight into the role of this unique and autologous stem cell therapy.” Dr. Jeffry R. Beer is a Voluntary Staff Physician at Long Island Jewish Medical Center, Northwell Health; Clinical Assistant Professor, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell; and Partner, Long Island Spine Rehabilitation Medicine, PC, East Meadow, NY.

Northwell Health is New York State’s largest health care provider and private employer, with 21 hospitals, 850 outpatient facilities and more than 12,000 affiliated physicians. The partnership will enable Northwell to participate in BioRestorative’s clinical trial targeting patients suffering from chronic lumbar disc disease.

About
BioRestorative
Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

• Disc/Spine Program (brtxDISC^™): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a person’s own) cultured mesenchymal stem cells collected from the patient’s bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patient’s bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patient’s damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have commenced a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

• Metabolic Program (ThermoStem^®): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (“BAT”). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company’s latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:

Email: [email protected]

NEW YORK and MCLEAN, Va., May 08, 2023 (GLOBE NEWSWIRE) — ID.me, the market leading federally conformant identity verification platform, and Sterling Check Corp. (NASDAQ: STER) (“Sterling”), a leading provider of identity and background services, today announced that their in-person identity verification solution has been recognized by the Kantara Initiative for meeting federal digital identity guidelines set by the National Institute of Standards and Technology (NIST). This achievement adds to ID.me’s online verification methods, including 43 million Americans who have already verified with ID.me to NIST standards and can prove their identity simply by logging in. This approach improves customer experience, equity, and security. ID.me’s innovative digital identity platform and Sterling’s network of retail-based kiosks enable federal, state, and local US governments to now deliver omnichannel, NIST-compliant access to identity verification.

Kantara Initiative, Inc is an international ethics based, mission-led nonprofit industry ‘commons’ focused on improving the trustworthy use of identity and personal data. ID.me was the first solution to earn Kantara’s Trustmark for Identity Assurance Level 2 (IAL2) in 2018, and now it achieves another milestone by being the first verification solution to have three verification methods independently assessed to be IAL2: online self-serve, video chat, and in-person. All of these options are now available through Sterling’s suite of background screening and identity verification services.

Currently used by five government agencies, in-person verification allows individuals — even those without a mobile device or dedicated internet access — to verify their identity at a physical location with the help of a trained agent. These locations are part of the Sterling Identity Network of safe, convenient, professional biometric collection and identity verification facilities in all 50 states and Washington DC. Additionally, agencies can tailor the customer experience by deploying kiosks within their offices or service centers. In fact, kiosks have already been installed in several state workforce centers, allowing for quick access to in-person verification during emergency recovery efforts.

ID.me and Sterling first deployed in-person verification with the New Jersey Department of Labor in November of 2021, and at the time, Robert Asaro-Angelo, Commissioner of the New Jersey Department of Labor and Workforce Development, noted his agency’s focus on making sure all residents can access benefits with the added protection against attempted fraud.

“We have been focused on delivering equity and accessibility for all New Jersey residents since day one, and this is another tool that allows us to continue with that mission,” Asaro-Angelo said in 2021. “Enhancing our ability to verify individuals through ID.me with the addition of in-person channels enables us to more effectively reach those who, for whatever reason, had trouble doing so online without compromising trust and security.”

Sterling and ID.me have been fine-tuning their in-person verification service to ensure it meets NIST guidelines and also integrates seamlessly with ID.me’s online and video chat verification capabilities.

“Earning Kantara’s TrustMark for in-person verification shows ID.me’s commitment to consumer choice and equitable access,” said Blake Hall, founder and CEO of ID.me. “Users that don’t have access to technology or prefer to engage outside of purely digital channels can do so in a way that meets the government’s high bar for security and assurance. Every day, we move closer to realizing our goal of ‘No Identity Left Behind’ in partnership with Sterling. Being the first independently accredited, omni-channel solution helps us raise the bar on equitable access.”

ID.me partners can purchase the IAL2 IPV offering to help expand access to secure, easy-to-use identity verification. Once an individual verifies their identity in person, they can use their ID.me credential to gain secure access elsewhere within ID.me’s network of 12 federal agencies, 36 state agencies, and over 500 companies. Sterling’s 50K+ clients have access to a suite of identity verification services to enhance their background screening programs.

“In-person verification is critical for achieving equitable access for all who need their identity verified” said Josh Peirez, CEO of Sterling.  “Whether individuals are seeking access to government services or applying for a new job, Sterling and ID.me are together providing their best path forward. Partnering with ID.me to achieve IAL2 certification is an accomplishment we are very proud of and affirms Sterling’s deep commitment and strong belief in our identity solutions.”

As part of the exclusive partnership between Sterling and ID.me, their teams are continuing to collaborate on innovative ways to strengthen and increase the efficiency of identity verification.

To learn more about ID.me’s in-person identity verification vision, visit https://bit.ly/inpersonverification.

To learn more about Sterling’s identity verification services, visit https://hubs.ly/Q01NVrWb0.

About ID.me

ID.me simplifies how individuals prove and share their identity online. More than 100 million members can use their ID.me Wallet to easily verify their identity across 30 states, 14 federal agencies, and over 500 name-brand retailers across ID.me’s secure digital identity network. The company provides secure login, identity proofing, and community affiliation verification for organizations across sectors. ID.me’s technology meets the federal standards for consumer authentication set by the Commerce Department and is approved as a NIST 800-63-3 IAL2 / AAL2 credential service provider by the Kantara Initiative. ID.me is committed to “No Identity Left Behind” to enable all people to have a secure digital identity. To learn more, visit www.ID.me.

About Sterling

Sterling (NASDAQ: STER) — a leading provider of background and identity services — offers background and identity verification to help over 50,000 clients create people-first cultures built on a foundation of trust and safety. Sterling’s tech-enabled services help organizations across all industries establish great environments for their workers, partners, and customers. With operations around the world, Sterling conducted more than 110 million searches in the twelve months ending December 31, 2022. Visit us at www.sterlingcheck.com.

Contacts

Sterling: Jodi Perkins | [email protected]
ID.me: Kevin Madden | [email protected]

• Data from Phase 1 dose escalation study of FHD-286, a BRG1/BRM inhibitor, in metastatic uveal melanoma expected in the second quarter of 2023

• Selective BRM, ARID1B, EP300 and CBP, targeting key regulators of gene expression, continue to advance towards IND

• Cash, cash equivalents and marketable securities of $316.0 million, as of March 31, 2023, provides cash runway into the second half of 2025

CAMBRIDGE, Mass., May 08, 2023 (GLOBE NEWSWIRE) — Foghorn^® Therapeutics Inc. (Nasdaq: FHTX), a clinical-stage biotechnology company pioneering a new class of medicines that treat serious diseases by correcting abnormal gene expression, today provided a financial and corporate update in conjunction with the Company’s 10-Q filing for the quarter ended March 31, 2023. With an initial focus in oncology, Foghorn’s Gene Traffic Control^® Platform and resulting broad pipeline have the potential to transform the lives of people suffering from a wide spectrum of diseases.

“In the coming months, we anticipate the initial Phase 1 results for FHD-286 in metastatic uveal melanoma and we continue to advance our exciting early-stage oncology programs—including our BRM selective inhibitor, CBP, EP300 and ARID1B—toward the clinic while showcasing our ability to repeatedly generate selective chemical matter against important targets in oncology,” said Adrian Gottschalk, President and Chief Executive Officer of Foghorn. “These programs have the potential to deliver novel therapies that hold tremendous value for large patient populations in a broad range of different cancers.”

Key Recent Updates and Upcoming Milestones

• FHD-286. FHD-286 is a potent, selective inhibitor of the BRG1 and BRM subunits of the BAF chromatin remodeling complex where dependency on BRG1/BRM is well-established pre-clinically with multiple tumor types, including uveal melanoma, acute myelogenous leukemia (AML)/myelodysplastic syndrome (MDS), non-small cell lung cancer (NSCLC) and prostate cancer.
  • mUM Update. Phase 1 dose escalation of FHD-286 in metastatic uveal melanoma (mUM) continues to enroll patients per protocol. Top-line Phase 1 safety and efficacy data is expected in the second quarter of 2023.
  • AML/MDS Update. In August 2022, the U.S. Food and Drug Administration (FDA) placed a full clinical hold on the Phase 1 dose escalation study of FHD-286 in relapsed and/or refractory AML and MDS. The Company anticipates providing a regulatory update for FHD-286 in AML/MDS in the second quarter of 2023.
• FHD-609 Update. On April 24, 2023, Foghorn provided an update on the FHD-609 Phase 1 program in synovial sarcoma and SMARCB1-deleted tumors. (Link to press release here).

• Differentiated Pipeline Advancement. Foghorn continues to expand its platform and pipeline. The Company anticipates the potential for six new molecular investigational new drug (IND) applications in the next four years. The Company continues to progress programs for multiple targets which include chromatin remodeling complexes, transcription factors, helicases and other chromatin related factors. These targets include Selective BRM* and wholly owned programs including CBP, EP300 and ARID1B, as well as other undisclosed targets, which combined could address more than 20 tumor types impacting more than 500,000 new patients annually.

• Medical Conference Participation. In April 2023, Foghorn participated at the 2023 American Association for Cancer Research Annual Meeting and the 18^th Annual Drug Discovery Chemistry Meeting, highlighting preclinical data from its selective CBP and EP300 protein degrader programs, preclinical data for FHD-286 and its transcription factor and protein degradation capabilities. To access the presentations, please visit the “Our Data” section of the Foghorn website.

• Strategic Collaborations. During the first quarter of 2023, Foghorn continued to progress the Company’s strategic collaborations with two world-leading pharmaceutical companies, which validate the rigor of our science, highlight the importance of the targets we are tackling and confirm the relevance of the biology on which we are focused.
  • In December 2021, Foghorn entered into a strategic collaboration with Loxo@Lilly. In 2023, Foghorn anticipates continued progress across the collaboration including a co-development and co-commercialization agreement on the Selective BRM program*, an additional undisclosed oncology target and three additional discovery programs. The Selective BRM program is on track to transition to Loxo@Lilly in the second half of 2023.
  • In July 2020, Foghorn entered into a strategic collaboration with Merck Sharp & Dohme. In 2023, Foghorn will continue to utilize its Gene Traffic Control platform to discover and develop novel therapeutics under the collaboration based on disruptors of a specified transcription factor target.

*In December 2021, Foghorn announced a strategic collaboration with Loxo@Lilly to create novel oncology medicines. The collaboration includes a co-development and co-commercialization agreement for Foghorn’s Selective BRM oncology program and an additional undisclosed oncology target. In addition, the collaboration includes three discovery programs using Foghorn’s proprietary Gene Traffic Control platform.

First Quarter 2023 Financial Highlights

• Strong Balance Sheet and Cash Runway. As of March 31, 2023, the Company had $316.0 million in cash, cash equivalents and marketable securities, which provides a cash runway into the second half of 2025.

• Collaboration Revenues. Collaboration revenue was $5.3 million for the three months ended March 31, 2023, compared to $3.9 million for the three months ended March 31, 2022. The increase year-over-year was primarily driven by revenue recognized under the Lilly collaboration agreement.

• Research and Development Expenses. Research and development expenses were $30.0 million for the three months ended March 31, 2023, compared to $24.5 million for the three months ended March 31, 2022. This increase was primarily due to costs associated with continued investment in R&D personnel and platform and early-stage research investments.

• General and Administrative Expenses. General and administrative expenses were $8.6 million for the three months ended March 31, 2023, compared to $7.2 million for the three months ended March 31, 2022. This increase was primarily due to an increase in investments to support the growing business which included increases in personnel-related costs and stock-based compensation expense.

• Net Loss. Net loss was $30.5 million for the three months ended March 31, 2023, compared to a net loss of $26.9 million for the three months ended March 31, 2022.

About FHD-286

FHD-286 is a highly potent, selective, allosteric and orally available, small-molecule, enzymatic inhibitor of BRG1 and BRM, two highly similar proteins that are the ATPases, or the catalytic engines across all forms of the BAF complex, one of the key regulators of the chromatin regulatory system. In preclinical studies, FHD-286 has shown anti-tumor activity across a broad range of malignancies including both hematologic and solid tumors. To learn more about these studies, please visit ClinicalTrials.gov. (Link here for metastatic uveal melanoma and here for AML and MDS).

About Uveal Melanoma

Uveal (intraocular) melanoma (UM) is a rare eye cancer that forms from cells that make melanin in the iris, ciliary body and choroid. It is the most common eye cancer in adults. It is diagnosed in about 2,000 adults every year in the United States and occurs most often in lightly pigmented individuals with a median age of 55 years. However, it can occur in all races and at any age. UM metastasizes in approximately 50% of cases, leading to very poor prognosis.

About AML

Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow and the most common type of acute leukemia in adults. AML is a diverse disease associated with multiple genetic mutations. It is diagnosed in about 20,000 people every year in the United States.

About Foghorn Therapeutics

Foghorn^® Therapeutics Inc. is discovering and developing a novel class of medicines targeting genetically determined dependencies within the chromatin regulatory system. Through its proprietary scalable Gene Traffic Control^® platform, Foghorn is systematically studying, identifying and validating potential drug targets within the chromatin regulatory system. The Company is developing multiple product candidates in oncology. Visit our website at www.foghorntx.com for more information on the company, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains “forward-looking statements” regarding the Company’s clinical programs for FHD-286 and FHD-609, including its efforts to resolve the full clinical hold relating to FHD-286 in AML and MDS, the anticipated timing of release of clinical data, its collaborations with Lilly and Merck and its research pipeline, including the status of its Selective BRM program, the filing of INDs and its protein degrader efforts. Forward-looking statements include statements regarding the Company’s clinical trials, product candidates and research efforts and other statements identified by words such as “could,” “may,” “might,” “will,” “likely,” “anticipates,” “intends,” “plans,” “seeks,” “believes,” “estimates,” “expects,” “continues,” “projects” and similar references to future periods. Forward-looking statements are based on our current expectations and assumptions regarding capital market conditions, our business, the economy and other future conditions. Because forward-looking statements relate to the future, by their nature, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. As a result, actual results may differ materially from those contemplated by the forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include regional, national or global political, economic, business, competitive, market and regulatory conditions, including risks relating to our clinical trials and other factors set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the Securities and Exchange Commission. Any forward-looking statement made in this press release speaks only as of the date on which it is made.

Condensed Consolidated Balance Sheets (In thousands)
	March 31, 2023				Dec. 31, 2022
Cash, cash equivalents and marketable securities	$	315,970			$	345,798
All other assets		56,913				59,085
Total assets	$	372,883			$	404,883
Deferred revenue, total	$	331,511			$	336,820
All other liabilities		65,958				67,951
Total liabilities		397,469				404,771
Total stockholders’ equity (deficit)		(24,586	)			112
Total liabilities and stockholders’ equity	$	372,883			$	404,883

 

Condensed Consolidated Statements of Operations (In thousands, except share and per share amounts)
	Three Months Ended March 31,
		2023				2022
Collaboration revenue	$	5,309			$	3,920
Operating expenses:
Research and development		29,985				24,508
General and administrative		8,641				7,216
Total operating expenses		38,626				31,724
Loss from operations		(33,317	)			(27,804	)
Total other income, net		3,389				890
Provision for income taxes		(560	)			—
Net loss	$	(30,488	)		$	(26,914	)
Net loss per share attributable to common stockholders—basic and diluted	$	(0.73	)		$	(0.65	)
Weighted average common shares outstanding—basic and diluted		41,811,087				41,370,186

Contact:

Ben Strain, Foghorn Therapeutics Inc. (Media and Investors)
[email protected]

Karin Hellsvik, Foghorn Therapeutics Inc. (Media)
[email protected]

Michael Lampe, ScientPR (Media)
[email protected]

Hans Vitzthum, LifeSci Advisors (Investors)
[email protected]

BOTHELL, Wash., May 08, 2023 (GLOBE NEWSWIRE) — Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced that it will participate in the following upcoming investor conferences:

JMP Securities Life Sciences Conference

Fireside chat on Monday, May 15, 2023, at 10:30 am ET in New York.

Mizuho Neurosciences Summit

One-on-one meetings on Tuesday, May 16, 2023, in Boston.

A live webcast of the JMP Securities presentation can be accessed from the Investors section of the Athira website at https://investors.athira.com/news-and-events/events-and-presentations-investor. An archived replay of the webcast will be available for at least 30 days following the event.

About Athira Pharma, Inc.

Athira Pharma, Inc., headquartered in the Seattle, Washington area, is a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration. Athira aims to provide rapid cognitive improvement and alter the course of neurological diseases with its novel mechanism of action. Athira is currently advancing its pipeline of therapeutic candidates targeting the HGF/MET neurotrophic system for Alzheimer’s and Parkinson’s disease, Dementia with Lewy bodies and ALS. For more information, visit www.athira.com. You can also follow Athira on Facebook, LinkedIn and @athirapharma on Twitter and Instagram.

Investor & Media Contact:

Julie Rathbun
Athira Pharma
[email protected]
206-769-9219

Prioritizing Late-Stage Clinical CNS Programs in Fibromyalgia, Depression, Migraine, and Cocaine Intoxication

Topline Results Expected in Fourth Quarter 2023 for Potentially Confirmatory Phase 3 Trial of TNX-102 SL for Fibromyalgia

Potentially Pivotal Phase 2 Trial of TNX-601 ER for Major Depressive Disorder (MDD) Enrolling; TNX-601 ER Represents an Innovative Approach for MDD through Restoration of Neuroplasticity and Neurogenesis

Cash and Cash Equivalents of Approximately $72.0 Million at March 31, 2023

CHATHAM, N.J., May 08, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced financial results for the first quarter ended March 31, 2023, and provided an overview of recent operational highlights.

“With much achieved already, we expect 2023 will continue to serve as an important milestone year for Tonix,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “The prioritization of key programs in our pipeline highlights our commitment to helping bring relief and value to patients suffering from diseases with limited or insufficient therapeutic options. We are pleased with the enrollment in our current RESILIENT Phase 3 study for TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in fibromyalgia. We are looking forward to topline results from the trial in the fourth quarter of this year. If successful, we believe it will be the second and final adequate and well-controlled efficacy trial required for filing a New Drug Application (NDA) for approval by the U.S. Food and Drug Administration (FDA). Moreover, we believe we have satisfied all the other clinical and non-clinical requirements for an NDA submission. In addition, we are excited to have initiated enrollment in the potentially pivotal UPLIFT Phase 2 study of TNX-601 ER (tianeptine hemioxalate extended release tablets) for major depressive disorder (MDD). TNX-601 ER represents a novel approach to treating depression in the U.S., since the active ingredient tianeptine restores neuroplasticity and neurogenesis rather than modulating neurotransmitter levels and activity.”

Recent Highlights—Key Product Candidates*

Central Nervous System (CNS) Pipeline

TNX-102 SL: small molecule for the management of fibromyalgia (FM)

• In March 2023 at the 5^th International Congress on Controversies in Fibromyalgia, the Company presented positive efficacy and safety data from Phase 3 RELIEF Study of TNX-102 SL for the management of fibromyalgia. Titled “Efficacy and Safety of TNX-102 SL (Sublingual Cyclobenzaprine) for the Treatment of Fibromyalgia: Results from the Randomized, Placebo Controlled RELIEF Trial”, the presentation displayed the data that TNX-102 SL met its pre-specified primary endpoint in the Phase 3 RELIEF trial, significantly reducing daily pain compared to placebo (p=0.01) in participants with fibromyalgia. In addition, TNX-102 SL was well tolerated with the most common adverse event associated with active treatment being oral numbness or hypoaesthesia, an administration site reaction that is typically transient, was never rated as severe, and led to only one discontinuation.
• The Company recently announced new plans to eliminate the interim analysis to streamline the ongoing Phase 3 RESILIENT trial and to provide topline data in the fourth quarter of 2023. Target enrollment for the TNX-102 SL fibromyalgia study remains approximately 470 participants.

TNX-102 SL for the treatment of Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)

• In April 2023, enrollment of 63 participants was completed in the PREVAIL study, a Phase 2 study of TNX-102 SL for fibromyalgia-type Long COVID.
• Topline results from the PREVAIL Phase 2 trial are expected in the third quarter of 2023.
• In February 2023 at a virtual event co-hosted by BIO and Solve M.E. titled, “Long COVID: What Will it Take to Accelerate Therapeutic Progress?”, the Company presented its analysis that the majority of Long COVID patients present with a constellation of symptoms including multisite pain, fatigue, sleep disorders and cognitive dysfunction or brain fog. These symptoms overlap with fibromyalgia and chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME). Fibromyalgia-type Long COVID, like fibromyalgia and CFS/ME, appears to be one of several chronic overlapping pain conditions that have in common the neurological process called central sensitization.

TNX-601 ER: a once-daily orally-administered small molecule for the treatment of MDD, Posttraumatic Stress Disorder (PTSD), neurocognitive dysfunction associated with corticosteroid use and potentially Alzheimer’s disease

• In March 2023, enrollment was initiated in the potentially pivotal Phase 2 ‘UPLIFT’ Study for the treatment of MDD. Results from a planned interim analysis are expected to be released in the fourth quarter of 2023.
• TNX-601 ER represents a novel approach to treating depression in the U.S., since the active ingredient tianeptine induces a neuroprotective and resilient phenotype in both neurons and microglia under conditions of stress. The Phase 2 UPLIFT study is a double-blind, randomized, multicenter, placebo-controlled study to evaluate the efficacy and safety of TNX-601 ER taken orally once-daily for 6 weeks to treat MDD. It is a parallel design study with two arms, a TNX-601 ER 39.4 mg arm and a placebo arm. A total of 300 participants will be randomized in a 1:1 ratio into the two arms across approximately 30 U.S. sites, enrolling adult patients 18-65 years old. The primary efficacy endpoint is mean change from baseline in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score at Week 6.

TNX-1900 (intranasal potentiated oxytocin): small peptide for migraine, craniofacial pain, insulin resistance and related disorders, and obesity-associated binge eating disorder

• In February 2023, enrollment began in the Phase 2 PREVENTION study of TNX-1900 for the prevention of migraine headache in chronic migraineurs. The double-blind, placebo-controlled study has a target enrollment of 150 participants at approximately 25 sites across the U.S, with topline results expected in the fourth quarter of 2023.
• In January 2023, data from clinical and nonclinical studies were presented at the 16^th Annual Headache Cooperative of the Pacific (HCOP) Winter Conference by collaborator Professor David Yeomans. The oral presentation titled, “Primary vs Secondary Sex Hormones and Migraine,” includes research sponsored and licensed by Tonix. Preliminary results from a positron emission tomography study in humans showed that intranasal delivery of a radioisotope of magnesium-potentiated oxytocin is delivered to the trigeminal ganglia, which have known roles in migraine headaches. In addition, preliminary results of data collected from isolated human trigeminal ganglia neurons in vitro show co-expression of oxytocin receptors and calcitonin gene-related peptide, which are believed to represent the first observation of oxytocin receptors in human trigeminal ganglia. Furthermore, the presentation highlights data which suggest a sex difference in oxytocin potency.

TNX-1300 (recombinant double mutant cocaine esterase): biologic for life-threatening cocaine intoxication

• Tonix expects to initiate a potentially pivotal, Phase 2 clinical study of TNX-1300 for the treatment of cocaine intoxication in the third quarter of 2023.
• As previously disclosed, in 2022, Tonix received a Cooperative Agreement grant from the National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH), to support development of TNX-1300.
• TNX-1300 has been granted Breakthrough Therapy designation by the FDA.

Rare Disease Pipeline

TNX-2900 (intranasal potentiated oxytocin): small peptide for the treatment of Prader-Willi syndrome (PWS)

• TNX-2900 has been granted Orphan Drug designation from the FDA for the treatment of PWS.
• In March 2023, Tonix delivered a presentation titled, “TNX-2900 (Intranasal Oxytocin + Magnesium) in Development for the Treatment of Hyperphagia in Adolescents and Young Adults with Prader-Willi Syndrome” at the Rare Disease Innovation and Partnership Summit. The presentation displayed data showing the enhancing effects of magnesium (Mg²⁺) on the activation of oxytocin receptors. The Mg²⁺ enhanced formulation of intranasal oxytocin is the basis for TNX-2900, in development to treat hyperphagia, or pathological over-eating, in children and young adult patients with PWS.

Immunology Pipeline

TNX-1500 (anti-CD40L monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis of organ transplant rejection and treatment of autoimmune disorders.

• In May 2023, the IND for prevention of organ rejection in patients receiving a kidney transplant was cleared by FDA. A First-in-Human Phase 1 study is expected to initiate in the third quarter of 2023.The first indication for TNX-1500 will be prophylaxis of organ rejection in adult patients receiving a kidney transplant, but multiple additional indications are possible, including autoimmune diseases.
• Tonix announced a research agreement with Boston Children’s Hospital to study TNX-1500 for the prevention of graft-versus-host diseases (GvHD) after hematopoietic stem cell transplantation (HCT) in animals. HCT from unrelated donors is a component of the treatment protocol for several hematologic malignancies, but GvHD complicates treatment and limits the success of engraftment after HCT. In April 2023, the Company announced the online publication of two papers in the American Journal of Transplantation by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital (MGH) in collaboration with Tonix Pharmaceuticals^1,2. The publications include data demonstrating that TNX-1500 showed activity in preventing organ rejection and was well tolerated in non-human primates. To date, there has not been a humanized anti-CD40L antibody that can effectively prevent transplant rejections with an acceptable level of safety.

Infectious Disease Pipeline

TNX-801 (live horsepox virus vaccine for percutaneous administration): vaccine to protect against smallpox and monkeypox (mpox) designed as a single-administration vaccine to elicit T cell immunity.

• As previously announced, a Phase 1 study is expected to start in the second half of 2023.
• In January 2023, the Company appointed Zeil Rosenberg, M.D., M.P.H., as Executive Vice President, Medical for Infectious Disease programs. Dr. Rosenberg is responsible for leading the Company’s clinical development efforts for vaccines, including TNX-801.
• A publication describing the activity of TNX-801 to protect non-human primates against a lethal challenge with intra-tracheal monkeypox was published in the peer-reviewed journal, Viruses³.

      *All of Tonix’s product candidates are investigational new drugs or biologics and none has been approved for any indication.

      ¹Lassiter, G., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate renal allograft survival. American Journal of Transplantation. https://doi.org/10.1016/j.ajt.2023.03.022

      ²Miura, S., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate cardiac allograft survival. American Journal of Transplantation. https://doi.org/10.1016/j.ajt.2023.03.025

      ³Noyce RS, et al. (2023). Single Dose of Recombinant Chimeric Horsepox Virus (TNX-801) Vaccination Protects Macaques from Lethal Monkeypox Challenge. Viruses. 15(2):356. doi: 10.3390/v15020356

Recent Highlights—Corporate and Other

• In April 2023, Tonix announced it is reallocating resources and cash to streamline its pipeline and focus on its mid- and late-stage clinical programs within its core CNS portfolio. The pipeline realignment prioritizes key near-term value drivers, reduces investment in several longer-term programs, particularly COVID-19-related studies, and delays the start of a PTSD study in Kenya.

Recent Highlights—Financial

As of March 31, 2023, Tonix had $72.0 million of cash and cash equivalents, compared to $120.2 million as of December 31, 2022. Net cash used by financing activities was approximately $11.5 million for first quarter 2023, compared to net cash provided by financing activities of $13.1 million for the same period in 2022.

On April 8, 2020, the Company entered into a sales agreement (the “Sales Agreement”) with AGP of up to $320.0 million in at-the-market offerings (“ATM”) sales. During the quarter ended March 31, 2023, the Company sold approximately 3.2 million shares of common stock under the Sales Agreement, for net proceeds of approximately $2.0 million. Subsequent to March 31, 2023, the Company has sold 0.9 million shares of common stock under the Sales Agreement, for net proceeds of approximately $0.5 million.

In January 1, 2023, the Company repurchased 16,700,269 shares of common stock under its share repurchase programs at an average price per share of $0.82 for a gross aggregate cost of approximately $13.6 million. 

Cash used in operations was approximately $32.9 million for the first quarter ended March 31, 2023, compared to $31.0 million for the first quarter ended March 31, 2022.

Cash used by investing activities for the first quarter ended March 31, 2023, and 2022 was approximately $3.8 million and $20.2 million, respectively, related to the purchase of property and equipment.

First Quarter 2023 Financial Results

R&D expenses for the first quarter 2023 were $26.5 million, compared to $18.4 million for the same period in 2022. As planned, R&D expenses will be increasing during 2023 as we move our clinical development programs forward and invest in our development pipeline.

G&A expenses for the first quarter 2023 were $7.4 million, compared to $8.0 million for the same period in 2022. The decrease is primarily due to decreased employee-related and financial reporting expenses.

Net loss was $33.0 million, or $0.52 per share, basic and diluted, for the first quarter 2023, compared to net loss of $26.4 million, or $1.61 per share, basic and diluted, for the same period in 2022. The basic and diluted weighted average common shares outstanding for the first quarter 2023 was 63,352,898 compared to 16,445,010 shares for the same period in 2022.

Tonix Pharmaceuticals Holding Corp.

^*

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia with topline data expected in the fourth quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 study has been completed, and topline results are expected in the third quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), in development for chronic migraine, is currently enrolling with topline data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim data expected in the fourth quarter of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the third quarter of 2023. Tonix’s rare disease portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum small molecule oral antivirals.

^*
All of Tonix’s product candidates are investigational new drugs or biologics and
none has
been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

TONIX PHARMACEUTICALS HOLDING CORP. 
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(In Thousands, Except Share and Per Share Amounts)
(unaudited)

													Three Months Ended March 31,
													2023								2022
COSTS AND EXPENSES:
Research and development													$	26,511							$	18,422
General and administrative														7,391								8,014
														33,902								26,436
Operating loss														(33,902					)			(26,436		)
Interest income														897								19
Net loss													$	(33,005					)		$	(26,417		)
Net loss per common share, basic and diluted													$	(0.52					)		$	(1.61		)
Weighted average common shares outstanding, basic and diluted														63,352,898								16,445,010

See the accompanying notes to the condensed consolidated financial statements

TONIX PHARMACEUTICALS HOLDING CORP.

CONDENSED CONSOLIDATED BALANCE SHEETS

(In Thousands)

(Unaudited)

	March 31, 2023			December 31, 20221
Assets
Cash and cash equivalents	$	71,975		$	120,229
Prepaid expenses and other		11,751			10,548
Total current assets		83,726			130,777
Other non-current assets		95,362			94,913
Total assets	$	179,088		$	225,690
Liabilities and stockholders’ equity
Total liabilities	$	13,661		$	18,508
Stockholders’ equity		165,427			207,182
Total liabilities and stockholders’ equity	$	179,088		$	225,690

¹The condensed consolidated balance sheet for the year ended December 31, 2022 has been derived from the audited financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
[email protected]
(862) 904-8182

Maddie Stabinski (media)
Russo Partners
[email protected]
(212) 845-4273

Peter Vozzo (investors)
ICR Westwicke
[email protected]
(443) 213-0505

LATHAM, N.Y., May 08, 2023 (GLOBE NEWSWIRE) — Plug Power Inc. (NASDAQ: PLUG), a leading provider of turnkey hydrogen solutions for the global green hydrogen economy, will announce its 2023 first quarter results on Tuesday, May 9, 2023.

Join the call:

• Date: May 9, 2023
• Time: 8:00 am ET
• Toll-free: 1-800-750-5857 or 1-212-231-2900
• Direct webcast: https://viavid.webcasts.com/starthere.jsp?ei=1614940&tp_key=df4cc70b65

The webcast can also be accessed directly from the Plug homepage (www.plugpower.com). A playback of the call will be available online for a period of time following the call.

About Plug

Plug Power is building the hydrogen economy as the leading provider of comprehensive hydrogen fuel cell (HFC) turnkey solutions. The Company’s innovative technology powers electric motors with hydrogen fuel cells amid an ongoing paradigm shift in the power, energy, and transportation industries to address climate change and energy security, while providing efficiency gains and meeting sustainability goals. Plug Power created the first commercially viable market for hydrogen fuel cell (HFC) technology. As a result, the Company has deployed over 60,000 fuel cell systems for e-mobility, more than anyone else in the world, and has become the largest buyer of liquid hydrogen, having built and operated a hydrogen highway across North America. Plug Power delivers a significant value proposition to end-customers, including meaningful environmental benefits, efficiency gains, fast fueling, and lower operational costs. Plug Power’s vertically integrated GenKey solution ties together all critical elements to power, fuel, and provide service to customers such as Amazon, BMW, The Southern Company, Carrefour, and Walmart. The Company is now leveraging its know-how, modular product architecture and foundational customers to rapidly expand into other key markets including zero-emission on-road vehicles, robotics, and data centers.

SOURCE: PLUG POWER

Plug Media Contact
Caitlin Coffee
Allison+Partners
(312) 635-8204
[email protected]

– Management to Host Conference Call at 8:00 AM ET Today –

WAYNE, Pa., May 08, 2023 (GLOBE NEWSWIRE) — Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, today announced its financial results for the first quarter of 2023 and provided a corporate update.

“The first quarter of 2023 represented another period of continued progress advancing our clinical stage development programs toward important data milestones,” stated Doug Manion, M.D., Chief Executive Officer of Aclaris. “As we continue to move toward data catalysts for zunsemetinib in rheumatoid arthritis and psoriatic arthritis as well as ATI-1777 in atopic dermatitis, we also are making positive progress towards bringing our next potentially broadly applicable candidate, ATI-2138, into its first proof of concept trial in ulcerative colitis.”

Continued Dr. Manion, “Regarding our proof-of-concept trial of zunsemetinib in hidradenitis suppurativa, which we reported in March, while we did not see positive efficacy results in this particularly challenging disease, we were able to strengthen our safety database and demonstrate mechanistically that our potentially first-in-class MK2 inhibitor performed as expected.”

Research and Development Highlights:

Clinical Development Programs:

• Zunsemetinib, an investigational oral small molecule MK2 inhibitor:
  Currently being developed as a potential treatment for immuno-inflammatory diseases
  • Rheumatoid Arthritis (ATI-450-RA-202): This Phase 2b dose ranging trial to investigate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of multiple doses (20 mg and 50 mg twice daily) of zunsemetinib in combination with methotrexate in subjects with moderate to severe rheumatoid arthritis (RA) is ongoing. Based on the continued positive enrollment momentum, Aclaris is narrowing its timing guidance for topline data to the fourth quarter of 2023.
  • Psoriatic Arthritis (ATI-450-PsA-201): This Phase 2a trial to investigate the efficacy, safety, tolerability, PK and PD of zunsemetinib (50 mg twice daily) in subjects with moderate to severe psoriatic arthritis (PsA) is ongoing. Based on a slower than anticipated study start up in Europe, the trial enrollment has taken longer than expected. Based on current enrollment trends and momentum, particularly in Poland, Aclaris now expects topline data in the first half of 2024, rather than year end 2023.

• ATI-1777, an investigational topical “soft” Janus kinase (JAK) 1/3 inhibitor:
  Currently being developing as a potential treatment for mild to severe atopic dermatitis (AD)
  • Atopic Dermatitis (ATI-1777-AD-202): This Phase 2b trial to determine the efficacy, safety, tolerability, and PK of multiple doses and application regimens of ATI-1777 in subjects with mild to severe AD is ongoing. In April 2023, Aclaris modified the protocol to expand the inclusion criteria for the trial to enroll patients with milder disease to broaden the drug’s target indication potential and to further aide enrollment which was challenged by an unexpected milder winter season. As a result, Aclaris currently projects topline data in the second half of 2023, rather than mid-year 2023.

• ATI-2138, an investigational oral covalent ITK/JAK3 inhibitor:
  Currently being developed as a potential treatment for T cell-mediated autoimmune diseases
  • Aclaris has selected ulcerative colitis as the intended first clinical development target for ATI-2138. Aclaris is also exploring additional indications that are relevant to the mechanism of action.
  • Healthy Volunteers (ATI-2138-PKPD-102): This Phase 1 MAD (multiple ascending dose) trial to investigate the safety, tolerability, PK and PD of ATI-2138 in healthy volunteers is ongoing. Aclaris continues to expect topline data in the second half of 2023.

Preclinical Development Program

• ATI-2231, an investigational oral MK2 inhibitor compound:
  Currently being explored as a potential treatment for pancreatic cancer and metastatic breast cancer as well as in preventing bone loss in patients with metastatic breast cancer
   
  • Second MK2 inhibitor generated from Aclaris’ proprietary KINect® drug discovery platform and designed to have a long plasma half-life.
  • Aclaris expects clinical development activities to be initiated in 2023, which is expected to advance as a collaboration with an academic third party.

Financial Highlights:

Liquidity and Capital Resources

As of March 31, 2023, Aclaris had aggregate cash, cash equivalents and marketable securities of $204.4 million compared to $229.8 million as of December 31, 2022.

Additionally, in March 2023, Aclaris issued a placement notice to sell approximately 3.4 million shares under its ATM facility for aggregate net proceeds of $26.7 million. This transaction closed in April 2023.   

Aclaris continues to anticipate that its cash, cash equivalents and marketable securities as of March 31, 2023 in combination with the $26.7 million in net proceeds from sales under the ATM facility subsequent to quarter end, will be sufficient to fund its operations through the end of 2025, without giving effect to any potential business development transactions or additional financing activities.

Financial Results

First Quarter 2023

• Net loss was $28.2 million for the first quarter of 2023 compared to $18.8 million for the first quarter of 2022.

• Total revenue was $2.5 million for the first quarter of 2023 compared to $1.5 million for the first quarter of 2022. The increase was driven by higher licensing revenue primarily from royalties earned on out-licensed intellectual property in the first quarter of 2023.

• Research and development (R&D) expenses were $22.6 million for the quarter ended March 31, 2023 compared to $14.3 million for the prior year period.
  • The $8.3 million increase was primarily the result of higher:
    • Zunsemetinib development expenses related to drug candidate manufacturing and costs associated with clinical activities for a Phase 2b trial for RA.
    • ATI-1777 development expenses related to costs associated with a Phase 2b clinical trial for AD.
    • ATI-2138 development expenses, including costs associated with a Phase 1 MAD trial and other preclinical activities.
    • Compensation-related expenses due to an increase in headcount.

• General and administrative (G&A) expenses were $8.8 million for the quarter ended March 31, 2023 compared to $6.1 million for the prior year period. The increase was primarily due to an increase in compensation-related expenses due to an increase in headcount.

• Licensing expenses were $1.1 million for the quarter ended March 31, 2023 resulting from separate third-party contractual obligations related to the non-exclusive patent license agreement with Lilly. There were no licensing expenses for the quarter ended March 31, 2022.

• Revaluation of contingent consideration resulted in a $0.8 million credit for the quarter ended March 31, 2023 compared to a credit of $1.2 million for the prior year period.

Conference Call and Webcast

As previously disclosed on May 2, 2023, management will host a conference call and webcast, with an accompanying slide presentation, at 8:00 AM ET today to provide a corporate update. To access the live webcast of the call and the accompanying slide presentation, please visit the “Events” page of the “Investors” section of Aclaris’ website, www.aclaristx.com. The webcast will be archived for at least 30 days on the Aclaris website.

About Aclaris Therapeutics, Inc.

Aclaris Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates to address the needs of patients with immuno-inflammatory diseases who lack satisfactory treatment options. The company has a multi-stage portfolio of drug candidates powered by a robust R&D engine exploring protein kinase regulation. For additional information, please visit www.aclaristx.com.

Cautionary Note Regarding Forward-Looking Statements

Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “believe,” “expect,” “intend,” “may,” “plan,” “potential,” “will,” and similar expressions, and are based on Aclaris’ current beliefs and expectations. These forward-looking statements include expectations regarding the development of Aclaris’ drug candidates, including the timing of its clinical trials, availability of data from those trials, and regulatory filings, and its belief that its existing cash, cash equivalents and marketable securities will be sufficient to fund its operations through the end of 2025. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Aclaris’ reliance on third parties over which it may not always have full control, Aclaris’ ability to enter into strategic partnerships on commercially reasonable terms, the uncertainty regarding the macroeconomic environment and other risks and uncertainties that are described in the Risk Factors section of Aclaris’ Annual Report on Form 10-K for the year ended December 31, 2022, and other filings Aclaris makes with the U.S. Securities and Exchange Commission from time to time. These documents are available under the “SEC Filings” page of the “Investors” section of Aclaris’ website at www.aclaristx.com. Any forward-looking statements speak only as of the date of this press release and are based on information available to Aclaris as of the date of this release, and Aclaris assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise.

Aclaris Therapeutics, Inc. Condensed Consolidated Statements of Operations (unaudited, in thousands, except share and per share data)
	Three Months Ended
	March 31,
	2023				2022
Revenues:
Contract research	$	889			$	1,221
Licensing		1,639				202
Other		—				30
Total revenue		2,528				1,453
Costs and expenses:
Cost of revenue (1)		808				1,155
Research and development (1)		22,587				14,306
General and administrative (1)		8,790				6,099
Licensing		1,061				—
Revaluation of contingent consideration		(800	)			(1,200	)
Total costs and expenses		32,446				20,360
Loss from operations		(29,918	)			(18,907	)
Other income, net		1,758				118
Net loss	$	(28,160	)		$	(18,789	)
Net loss per share, basic and diluted	$	(0.42	)		$	(0.31	)
Weighted average common shares outstanding, basic and diluted		66,872,778				61,431,026
(1) Amounts include stock-based compensation expense as follows:
Cost of revenue	$	299			$	228
Research and development		2,602				(113	)
General and administrative		3,905				2,231
Total stock-based compensation expense	$	6,806			$	2,346

Aclaris Therapeutics, Inc. Selected Consolidated Balance Sheet Data (unaudited, in thousands, except share data)
	March 31, 2023				December 31, 2022
Cash, cash equivalents and marketable securities	$	204,405			$	229,813
Total assets	$	229,705			$	254,596
Total current liabilities	$	18,258			$	21,938
Total liabilities	$	52,895			$	56,975
Total stockholders’ equity	$	176,810			$	197,621
Common stock outstanding		67,206,025				66,688,647

Aclaris Therapeutics, Inc. Selected Consolidated Cash Flow Data (unaudited, in thousands)
	Three Months Ended March 31, 2023				Three Months Ended March 31, 2022
Net loss	$	(28,160	)		$	(18,789	)
Depreciation and amortization		198				208
Stock-based compensation expense		6,806				2,346
Revaluation of contingent consideration		(800	)			(1,200	)
Changes in operating assets and liabilities		(4,397	)			(3,534	)
Net cash used in operating activities	$	(26,353	)		$	(20,969	)

Aclaris Therapeutics Contact:

Robert A. Doody Jr.
Vice President, Investor Relations
484-639-7235
[email protected]

IGALMI™ (dexmedetomidine) commercial momentum accelerating with doubling of formulary wins, unlocking more than $55 million in targeted market opportunity, and an additional $255 million scheduled to vote

Top-line data from pivotal SERENITY III Phase 3 trial (Part 1) for BXCL501 in bipolar or schizophrenia-associated agitation for at-home use expected in May 2023

Top-line data from repeat dosing of BXCL501 in Phase 1b trial for Major Depressive Disorder program in healthy volunteers expected in May 2023

Top-line data from pivotal TRANQUILITY II Phase 3 trial for BXCL501 for acute treatment of agitation associated with Alzheimer’s disease expected in June 2023

Company to host conference call at 8:00 a.m. ET today

NEW HAVEN, Conn., May 08, 2023 (GLOBE NEWSWIRE) — BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced its financial results for the first quarter ended March 31, 2023, and provided an update on key strategic initiatives.

“The first quarter marked a strong start to the year with numerous advancements in our clinical programs and continued commercial focus building the agitation market for our new therapeutic option in a historically underdiagnosed and underserved medical condition,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “We are gearing up to announce top-line data readouts in agitation from two Phase 3 pivotal trials as well as BXCL501 potential as an adjunctive treatment for chronic use in our MDD program. In addition, IGALMI’s launch momentum is expanding our reach into addressable market opportunities. We believe the second quarter of 2023 represents a defining moment for the Company as we expand the full potential of BXCL501 in agitation for at-home use and long-term care settings, and in depression. These upcoming catalysts may have a transformational impact for patients in need and all our stakeholders.”

Company Highlights

Neuroscience Franchise

IGALMI™ (dexmedetomidine) sublingual film

IGALMI is approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.
¹
Up to an estimated 16 million institutional episodes occur annually within these two patient populations in the U.S.
^2
–
4
*

Key Commercial Parameters

• IGALMI launch is accelerating with continued deployment of integrated commercial team in 2023:
  • Secured more than 130 hospital formulary wins to date, which have doubled in the last two months; equates to $55 million of addressable market with Integrated Delivery Network (IDN) formulary approvals.
  • Six hundred additional Pharmacy & Therapeutics (P&T) Committee votes scheduled and approximately 25% of target IDN beds representing an additional $255 million of addressable market scheduled to vote.
  • More than half of all ordering hospitals have reordered; repeat orders demonstrate real-world utility and growing health care provider (HCP) interest.
  • Sales team has reached over 75% of 1,700 targeted hospitals with focus on deepening advocacy and driving demand.  
  • Group Purchasing Organization (GPO) process largely complete with nearly 80% of targeted beds under contract.
  • Amplifying IGALMI awareness through multi-channel approach including digital and print media, peer influence initiatives, and Free Trial Program.

Medical Affairs

• Medical Science Liaison and Medical Managed Care teams engaging in scientific dialogue with HCPs ahead of IDN and hospital formulary reviews.
  • Observed a 73% increase in medical community requests for IGALMI clinical information over prior quarter.
  • Conducted a real-world survey of early-adopter HCPs; results expected to be presented at the American Society of Health-System Pharmacists Annual Meeting in June 2023.
  • Continuing extensive medical education initiatives through multiple clinical and scientific publications and presentations.

Development Pipeline  
BXCL501, an investigational proprietary, sublingual film formulation of dexmedetomidine, has received Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia.

• Alzheimer’s Disease-related Agitation: TRANQUILITY program is designed to evaluate BXCL501 for the acute treatment of Alzheimer’s-related agitation; up to 100 million Alzheimer’s-related agitation episodes are estimated to occur in the U.S. annually.^2*
  • TRANQUILITY II: Trial is fully enrolled, and all patients have completed the study in assisted living facilities (ALFs) and residential care settings.
    • Data cleaning and verification in progress.
    • Top-line data from pivotal trial expected in June 2023.
  • TRANQUILITY III: Continuing enrollment of patients with moderate to severe dementia in long-term-care facilities.
• Bipolar or Schizophrenia-related Agitation (At-Home Use): SERENITY III program is designed to evaluate BXCL501 for at-home use, where up to 23 million bipolar or schizophrenia-related agitation episodes are estimated to occur in the U.S. annually.^2–4*

SERENITY III consists of two parts:

• Part 1: Assessing the efficacy and safety of 60 mcg dose in acute treatment of agitated patients with bipolar I or II disorder or schizophrenia.
  • Data cleaning and verification in progress.
  • Top-line efficacy data from pivotal trial expected in May 2023.
• Part 2: Evaluating the safety of 60 mcg dose, and a second 60 mcg dose is allowed if required, at-home.
  • Expect to initiate Part 2 in Q2 2023.

• Adjunctive Treatment for Major Depressive Disorder (MDD) for At-Home Use: Phase 1b Multiple Ascending Dose (MAD) trial was designed to test safety and tolerability of daily dosing of BXCL501 for seven days in healthy volunteers to inform proof-of-concept (POC) trial dose selection. In Phase 2 program, treatment in MDD patients will be evaluated with BXCL501 in combination with selective serotonin- or serotonin-norepinephrine reuptake inhibitors (SSRIs or SNRIs, respectively) to assess rapid antidepressant response. Over 300 million antidepressant prescriptions are filled annually in the U.S.,^5* and current treatments are limited by slow onset of action and incomplete responses.
  • Data cleaning and verification in progress.
  • Top-line results are expected in May 2023.

OnkosXcel Therapeutics

OnkosXcel Therapeutics is a subsidiary of BioXcel Therapeutics focused on the sustained growth of the Company’s immuno-oncology (I-O) franchise, including BXCL701, its most advanced I-O program. BXCL701 is an investigational, oral innate immune activator in development for the treatment of aggressive forms of prostate cancer and other solid and liquid tumors.

• Continuing to actively evaluate strategic options for OnkosXcel Therapeutics:
  • Small Cell Neuroendocrine Prostate Cancer (SCNC) Program:  In 2023, it is estimated there will be 288,300⁶ new prostate cancer patients in the U.S., with approximately 11,500 patients progressing to SCNC.⁷
    • Planned Phase 2b potential pivotal study for BXCL701 monotherapy and in combination with KEYTRUDA® (pembrolizumab) in SCNC expected to initiate in 2H 2023, subject to further discussions with FDA.
  • Predictive Biomarker for BXCL701: Additional findings on DPP9 overexpression, a potential actionable biomarker for BXCL701 response, expected to be presented at an upcoming medical meeting.
  • Investigator-Sponsored Trials at Top Academic Centers: BXCL701 to be studied in combination with KEYTRUDA® (pembrolizumab) at Georgetown Lombardi Cancer Center and Dana-Farber Cancer Institute in pancreatic cancer and AML, respectively.

First Quarter 2023 Financial Results

Net Revenue: Net revenue was approximately $206,000 for the quarter, in line with the fourth quarter. The Company expects to see a notable uptick in revenues in the second half of the year in connection with additional formulary approvals.

Research and Development (R&D) Expenses: R&D expenses were $27.8 million for the first quarter of 2023, compared to $18.6 million for the same period in 2022. The increased expenses were primarily attributable to multiple clinical trials and CMC costs related to the upcoming three data readouts.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $23.6 million for the first quarter of 2023, compared to $12.9 million for the same period in 2022. The increased expenses were primarily attributable to personnel and sales, market access, and marketing costs associated with the commercialization of IGALMI in the U.S.

Net Loss: BioXcel Therapeutics had a net loss of $52.8 million for the first quarter of 2023, compared to a net loss of $31.5 million for the same period in 2022.

Cash and cash equivalents totaled $165.5 million as of March 31, 2023. BioXcel Therapeutics believes that full execution of its strategic financing with Oaktree and Qatar Investment Authority, and IGALMI revenues, would result in a cash runway into 2025.

Anticipated Milestones

• Top-line Clinical Trial Data Readouts
  • Pivotal SERENITY III Phase 3 trial (Part 1): May 2023
  • Phase 1b MAD trial for MDD program: May 2023
  • Pivotal TRANQUILITY II Phase 3 trial: June 2023
• Expected Clinical Trial Initiations
  • Pivotal SERENITY III Phase 3 trial (Part 2): Q2 2023
  • Phase 2b potential pivotal study of BXCL701 in SCNC: 2H 2023

Conference Call

BioXcel Therapeutics will host a conference call and webcast on May 8 at 8:00 a.m. ET to discuss its first quarter 2023 financial results and provide an update on recent operational highlights. To access the call, please dial 877-407-5795 (domestic) and 201-689-8722 (international). A live webcast will be available on the Investors section of the corporate website, bioxceltherapeutics.com, and a replay will be available through August 8, 2023. 

BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the “Email Alerts” option under the News/Events menu of the Investors & Media section of its website. 

About IGALMI™ (dexmedetomidine) sublingual film

INDICATION

IGALMI™ (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. The safety and effectiveness of IGALMI has not been studied beyond 24 hours from the first dose. It is not known if IGALMI is safe and effective in children.

IMPORTANT SAFETY INFORMATION

IGALMI can cause serious side effects, including:

• Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint.
• Heart rhythm changes (QT interval prolongation). IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations.
• Sleepiness/drowsiness. Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI.
• Withdrawal reactions, tolerance, and decreased response/efficacy. IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.

The most common side effects of IGALMI in clinical studies were sleepiness or drowsiness, a prickling or tingling sensation or numbness of the mouth, dizziness, dry mouth, low blood pressure, and low blood pressure upon standing.

These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.

Patients should tell their healthcare provider about their medical history, including if they suffer from any known heart problems, low potassium, low magnesium, low blood pressure, low heart rate, diabetes, high blood pressure, history of fainting, or liver impairment. They should also tell their healthcare provider if they are pregnant or breastfeeding or take any medicines, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Patients should especially tell their healthcare provider if they take any drugs that lower blood pressure, change heart rate, or take anesthetics, sedatives, hypnotics, and opioids.

Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201-1088 or [email protected].

Please see full
Prescribing Information.

About BioXcel Therapeutics, Inc.

BioXcel Therapeutics, Inc. is a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology. The Company’s drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications. The Company’s commercial product, IGALMI™ (developed as BXCL501), is a proprietary, sublingual film formulation of dexmedetomidine approved for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. The safety and effectiveness of IGALMI has not been established beyond 24 hours from the first dose. For more information, please visit igalmi.com and also see the IGALMI full Prescribing Information. BXCL501 is under evaluation for at-home use for the acute treatment of agitation in bipolar and schizophrenia patients, for acute treatment of agitation associated with probable Alzheimer’s disease, and as an adjunctive treatment for major depressive disorder. The safety and efficacy of BXCL501 for these uses have not been established. The Company is also developing BXCL502 as a potential therapy for chronic agitation in dementia. Under its subsidiary, OnkosXcel Therapeutics, the Company is developing BXCL701, an investigational, oral systemic innate immune activator for the treatment of aggressive forms of prostate cancer and other solid and liquid tumors. The safety and efficacy of BXCL502 and BXCL701 have not been established. For more information, please visit bioxceltherapeutics.com.

Forward-Looking Statements

This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended (the “Securities Act”) and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). All statements contained in this press release other than statements of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the Company’s expected timing of, and data results from, trials and clinical studies involving its product candidates; its ongoing marketing, commercialization and expansion efforts, plan and strategy for IGALMI; strategic options for OnkosXcel; the Company’s participation in upcoming events and presentations; and the Company’s future financial and operational results, including future revenue growth. The words “anticipate,” “believe,” “can,” “continue,” “could,” “designed,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon the Company’s current expectations and various assumptions. The Company believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. The Company may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, its limited operating history; its incurrence of significant losses; its need for substantial additional funding and ability to raise capital when needed; its significant indebtedness and other contractual obligations; its limited experience in drug discovery and drug development; its dependence on the success and commercialization of IGALMI™, BXCL501, BXCL502 BXCL701 and BXCL702 and other product candidates; its lack of experience in marketing and selling drug products; the risk that IGALMI or the Company’s product candidates may not be accepted by physicians or the medical community in general; the failure of preliminary data from its clinical studies to predict final study results; failure of its early clinical studies or preclinical studies to predict future clinical studies; its ability to receive regulatory approval for its product candidates; its ability to enroll patients in its clinical trials; undesirable side effects caused by the Company’s product candidates; its novel approach to the discovery and development of product candidates based on EvolverAI; the significant influence of and dependence on BioXcel LLC; its exposure to patent infringement lawsuits; its reliance on third parties; its ability to comply with the extensive regulations applicable to it; impacts from data breaches or cyber-attacks, if any; impacts from the COVID-19 pandemic; risks associated with the increased scrutiny relating to environmental, social and governance (ESG) matters; its ability to commercialize its product candidates; and the other important factors discussed under the caption “Risk Factors” in its Annual Report on Form 10-K for the fiscal year ended December 31, 2022, as such factors may be updated from time to time in its other filings with the SEC, including without limitation, its Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2023, which are accessible on the SEC’s website at www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While the Company may elect to update such forward-looking statements at some point in the future, except as required by law, it disclaims any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release.

Website Disclosure

We announce material financial and operational information to our investors using press releases, SEC filings and public conference calls webcasts, as well as the Investors & Media section of our website at www.bioxceltherapeutics.com. We may use our website as a distribution channel of material information about the Company. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the “Email Alerts” option under the News / Events menu of the Investors & Media section of our website at www.bioxceltherapeutics.com.

Contact Information

Corporate

BioXcel Therapeutics
Erik Kopp
1.203.494.7062
[email protected]

Investor Relations

BioXcel Therapeutics
Brennan Doyle
1.475.355.8462
[email protected]

Media

Russo Partners
David Schull or Scott Stachowiak
1.858.717.2310
[email protected]
[email protected]

Source: BioXcel Therapeutics, Inc.

IGALMI is a trademark of BioXcel Therapeutics, Inc. All other trademarks are the properties of their respective owners. Copyright © 2023, BioXcel Therapeutics, Inc. All rights reserved.

References and Notes

*Prevalence estimates show prominent variability and gradients

1. IGALMI™ (dexmedetomidine) [package insert]. New Haven, CT. BioXcel Therapeutics, Inc.; 2022.
2. Wu EQ, Shi L, Birnbaum H, et al. Annual prevalence of diagnosed schizophrenia in the USA: a claims data analysis approach. Psychol Med. 2006;36(11):1535-1540.
3. National Institute of Mental Health. Bipolar Disorder. Accessed April 5, 2022. https://rb.gy/lqz4rn.
4. UN Population Prospectus. Retrieved May 6, 2021. https://population.un.org/wpp.
5. IQVIA, 2021.
6. American Cancer Society. About Prostate Cancer. Accessed March 7, 2023.   https://www.cancer.org/cancer/prostate-cancer/about/key-statistics.html.
7. B.R. Alabi, S. Liu and T. Stoyanova, Current and emerging therapies for neuroendocrine prostate cancer, Pharmacology and Therapeutics (2022), https://doi.org/10.1016/j.pharmthera.2022.108255.

 

BioXcel Therapeutics, Inc.
Statements of operations
(in thousands, except per share amounts)
	Three months ended March 31,
		2023				2022
Revenues	$	206			$	–
Operating expenses
Cost of goods sold	$	9			$	–
Research and development		27,800				18,559
Selling, general and administrative		23,595				12,921
Total operating expenses	$	51,404			$	31,480
Loss from operations	$	(51,198	)		$	(31,480	)
Other expense (income)
Interest expense		3,367				7
Interest income		(2,015	)			(15	)
Other expense, net		246				–
Net loss and comprehensive loss	$	(52,796	)		$	(31,472	)
Net loss per share – basic and diluted	$	(1.84	)		$	(1.12	)
Weighted average shares outstanding – basic and diluted		28,616				27,980
Condensed Balance Sheets
(in thousands)
		March 31, 2023				December 31, 2022
Cash and cash equivalents	$	165,521			$	193,725
Working capital	$	148,509			$	169,970
Total assets	$	180,103			$	205,853
Long-term liabilities	$	98,339			$	96,180
Total liabilities	$	127,098			$	129,078
Total stockholders’ equity	$	53,005			$	76,775

 

 

This news release constitutes a “designated news release” for purposes of the Company’s prospectus supplement dated August 16, 2021, to its short form base shelf prospectus dated August 12, 2021.

TORONTO and BROSSARD, Québec, May 08, 2023 (GLOBE NEWSWIRE) — Bitfarms Ltd. (NASDAQ: BITF//TSX: BITF), a global vertically integrated Bitcoin mining company, today announces that in six years of operations it has mined 21,000 BTC with renewable hydropower.

“On May 7^th, 2023, Bitfarms achieved an important milestone by producing its 21,000^th BTC mined with renewable power, representing 1/1,000 of all BTC to ever be mined,” said Geoff Morphy, CEO of Bitfarms. “Leading the industry, this accomplishment is a testament to the proven capabilities of our mining operations that have powered on average between 1% and 2% of the BTC network, and we are proud to have done so with sustainable hydropower.

Morphy continued, “Bitfarms recognized early on the advantages of hydropower as a renewable energy resource and its sustainable and consistent economics. Our hydropower contracts provide a low direct cost of production and deliver higher mining margins than if we prioritized exahash growth alone. Bitfarms strategic expansions into Washington state and Paraguay are similar to our initial operations in Quebec, as we take advantage of excess hydropower infrastructure that became available with the migration of heavy industry. We have harnessed sustainable power with favorable economics for our shareholders, while simultaneously revitalizing communities with new jobs, capital investment, incremental tax revenues, and upgrades to the local electrical infrastructure. We thank our shareholders, directors, and team members, past and present, who made this great achievement possible.”

BTC Network Protocol and 21 Million BTC Limitation

Bitcoin is a cryptocurrency that operates on a decentralized network of computers. One of its most distinctive features is that there is a maximum limit of 21 million BTC that can ever be created. This limit was built into the Bitcoin protocol from the beginning and is expected to be reached in the year 2140.

As of May 7^th, 2023, there were approximately 19.4 million BTC in circulation, with a total value of approximately $550 billion. The remaining 1.6 million BTC are expected to be mined over the next century or so, with the rate of new BTC creation slowing down over time due to a process known as the “halving”. The next halving event is expected to occur in the year 2024.

Having mined 21,000 BTC, Bitfarms has now produced one of every 1,000 BTC that will ever be created. With its extensive portfolio of hydro powered operations, it has achieved this using 100% renewable energy. This accomplishment has been permanently memorialized on the Bitcoin blockchain at block 788,607.

About Bitfarms Ltd.

Founded in 2017, Bitfarms is a global, publicly traded (NASDAQ/TSX: BITF) Bitcoin mining company. Bitfarms develops, owns, and operates vertically integrated mining farms with in-house management and company-owned electrical engineering, installation service, and multiple onsite technical repair centers. The Company’s proprietary data analytics system delivers best-in-class operational performance and uptime.

Bitfarms currently has 10 farms, which are located in four countries: Canada, the United States, Paraguay, and Argentina. Powered by predominantly environmentally friendly hydro-electric and long-term power contracts, Bitfarms is committed to using sustainable, locally based, and often underutilized energy infrastructure.

To learn more about Bitfarms’ events, developments, and online communities:

Website: www.bitfarms.com

https://www.facebook.com/bitfarms/

https://twitter.com/Bitfarms_io

https://www.instagram.com/bitfarms/

https://www.linkedin.com/company/bitfarms/

Glossary of Terms

• BTC BTC/day = Bitcoin or Bitcoin per day
• EH or EH/s = Exahash or exahash per second
• MW or MWh = Megawatts or megawatt hour
• PH or PH/s = Petahash or petahash per second
• TH or TH/s = Terahash or terahash per second
• w/TH = Watts per Terahash
• KWh = Kilowatt per hour
  

Cautionary Statement

Trading in the securities of the Company should be considered highly speculative. No stock exchange, securities commission or other regulatory authority has approved or disapproved the information contained herein. Neither the Toronto Stock Exchange, Nasdaq, or any other securities exchange or regulatory authority accepts responsibility for the adequacy or accuracy of this release.

Forward-Looking Statements

This news release contains certain “forward-looking information” and “forward-looking statements” (collectively, “forward-looking information”) that are based on expectations, estimates and projections as at the date of this news release and are covered by safe harbors under Canadian and United States securities laws. The forward-looking information includes, but is not limited to, information concerning: the intentions, plans and future actions of the Company, as well as Bitfarms’ ability to successfully mine digital currency, revenue increasing as currently anticipated, the ability to profitably liquidate current and future digital currency inventory, volatility of network difficulty and digital currency prices and the potential resulting significant negative impact on the Company’s operations, the construction and operation of expanded blockchain infrastructure as currently planned, and the regulatory environment for cryptocurrency in the applicable jurisdictions.

Any statements that involve discussions with respect to predictions, expectations, beliefs, plans, projections, objectives, assumptions, future events or performance (often but not always using phrases such as “expects”, or “does not expect”, “is expected”, “anticipates” or “does not anticipate”, “plans”, “budget”, “scheduled”, “forecasts”, “estimates”, “believes” or “intends” or variations of such words and phrases or stating that certain actions, events or results “may” or “could”, “would”, “might” or “will” be taken to occur or be achieved) are not statements of historical fact and may be forward-looking information and are intended to identify forward-looking information.

This forward-looking information is based on assumptions and estimates of management of the Company at the time they were made, and involves known and unknown risks, uncertainties and other factors which may cause the actual results, performance, or achievements of the Company to be materially different from any future results, performance or achievements expressed or implied by such forward-looking information. Such factors include, among others, risks relating to: the availability of financing opportunities, risks associated with economic conditions, dependence on management and conflicts of interest, the ability to service debt obligations and maintain flexibility in respect of debt covenants; economic dependence on regulated terms of service and electricity rates; the speculative and competitive nature of the technology sector; dependency on continued growth in blockchain and cryptocurrency usage; lawsuits and other legal proceedings and challenges; conflict of interests with directors and management; government regulations;
the global economic climate; dilution; the Company’s limited operating history; future capital needs and uncertainty of additional financing, including the Company’s ability to utilize the Company’s at-the-market equity offering program (the “ATM Program”) and the prices at which the Company may sell Common Shares in the ATM Program, as well as capital market conditions in general; risks relating to the strategy of maintaining and increasing Bitcoin holdings and the impact of depreciating Bitcoin prices on working capital; the competitive nature of the industry; currency exchange risks; the need for the Company to manage its planned growth and expansion; the effects of product development and need for continued technology change; the ability to maintain reliable and economical sources of power to run its cryptocurrency mining assets; the impact of energy curtailment or regulatory changes in the energy regimes in the jurisdictions in which the Company operates; protection of proprietary rights; the effect of government regulation and compliance on the Company and the industry; network security risks; the ability of the Company to maintain properly working systems; reliance on key personnel; global economic and financial market deterioration impeding access to capital or increasing the cost of capital; share dilution resulting from the ATM Program and from other equity issuances; and volatile securities markets impacting security pricing unrelated to operating performance. In addition, particular factors that could impact future results of the business of Bitfarms include, but are not limited to: the construction and operation of facilities may not occur as currently planned, or at all; expansion may not materialize as currently anticipated, or at all; the digital currency market; the ability to successfully mine digital currency; revenue may not increase as currently anticipated, or at all; it may not be possible to profitably liquidate the current digital currency inventory, or at all; a decline in digital currency prices may have a significant negative impact on operations; an increase in network difficulty may have a significant negative impact on operations; the volatility of digital currency prices; the anticipated growth and sustainability of hydroelectricity for the purposes of cryptocurrency mining in the applicable jurisdictions; the inability to maintain reliable and economical sources of power for the Company to operate cryptocurrency mining assets; the risks of an increase in the Company’s electricity costs, cost of natural gas, changes in currency exchange rates, energy curtailment or regulatory changes in the energy regimes in the jurisdictions in which the Company operates and the adverse impact on the Company’s profitability; the ability to complete current and future financings, any regulations or laws that will prevent Bitfarms from operating its business; historical prices of digital currencies and the ability to mine digital currencies that will be consistent with historical prices; an inability to predict and counteract the effects of COVID-19 on the business of the Company, including but not limited to the effects of COVID-19 on the price of digital currencies, capital market conditions, restriction on labour and international travel and supply chains; and, the adoption or expansion of any regulation or law that will prevent Bitfarms from operating its business, or make it more costly to do so. For further information concerning these and other risks and uncertainties, refer to the Company’s filings on

www.SEDAR.com

(which are also available on the website of the U.S. Securities and Exchange Commission at

www.sec.gov

), including the annual information form for the year-ended December 31, 2022, filed on March 21, 2023. The Company has also assumed that no significant events occur outside of Bitfarms’ normal course of business. Although the Company has attempted to identify important factors that could cause actual results to differ materially from those expressed in forward-looking statements, there may be other factors that cause results not to be as anticipated, estimated or intended. There can be no assurance that such statements will prove to be accurate as actual results and future events could differ materially from those anticipated in such statements. Accordingly, readers should not place undue reliance on any forward-looking information. The Company undertakes no obligation to revise or update any forward-looking information other than as required by law.

Contacts:

LHA Investor Relations

David Barnard
+1 415-433-3777
[email protected]

Actual Agency

Lisa Helfer
+1 646-373-9946
[email protected]

Québec Media: Tact

Louis-Martin Leclerc
+1 418-693-2425
[email protected]