Market Newsdesk

New Drug Application and Marketing Authorization Application for

neffy



^®


Currently Under Review with the FDA and EMA; FDA PDUFA Target Action Date Anticipated in Mid-2023

Strong Financial Position with $274.4 Million in Cash, Cash Equivalents and Short-term Investments to Support Operating Runway for the Next Three Years

SAN DIEGO, March 23, 2023 (GLOBE NEWSWIRE) — ARS Pharmaceuticals, Inc. (Nasdaq: SPRY), a biopharmaceutical company dedicated to empowering at-risk patients and caregivers to better protect themselves from severe allergic reactions that could lead to anaphylaxis, today highlighted recent progress and reported fourth quarter and full year 2022 financial results.

“This is an incredibly exciting time for ARS, with potential regulatory approvals for neffy® around the corner and a resulting transition to a commercial-stage company. Our goal with neffy is to provide patients with the ability to deliver epinephrine with comparable pharmacokinetics to an intramuscular injection, but in an easy to use and rapidly administered needle-free nasal spray. We believe we are well on our way to achieving this goal,” said Richard Lowenthal, president and chief executive officer of ARS Pharmaceuticals. “Both our NDA and MAA for neffy are currently under review by the regulators in the U.S. and E.U. and we are on-track with our commercial preparedness activities for a potential U.S. launch later this year, if approved. We also recently reacquired European rights to neffy which enhance our optionality to evaluate potential partnerships or strategic transactions. The team has done a remarkable job executing the development of neffy, and we look forward to engaging with the regulatory agencies in an effort to impact the lives of millions of people with serious allergic reactions.”

neffy

Progress

• 
  neffy
  
  NDA and MAA for the Treatment of Allergic Reactions (Type 1), Including Anaphylaxis, Under Review with FDA and EMA: The Company’s new drug application (NDA) and marketing authorization application (MAA) for neffy for the emergency treatment of allergic reactions (Type I), including anaphylaxis in adults and children ≥30 kg (66 lbs), were accepted for review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), respectively. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date that is anticipated in mid-2023. If approved, neffy would be the first non-injectable treatment available to patients with allergic reactions (Type I), including anaphylaxis.

  The regulatory submissions to the FDA and EMA were based on data from four primary registrational studies showing that 2.0 mg intranasal dose of neffy met all clinical endpoints recommended by regulators and that its pharmacokinetics were within the range of approved epinephrine injection products. These data, which were presented at the 2022 American College of Allergy Asthma and Immunology Annual Scientific Meeting (ACAAI) and the 2023 American Academy of Allergy, Asthma and Immunology (AAAAI) meeting, included studies in adults, with self-administration and caregiver administration, as well as in children with Type I allergies ≥30 kg (66 lbs). In addition, neffy has been well-tolerated to date with more than 600 individuals receiving at least one dose, and many with repeat administration. Adverse events in the clinical trials were generally mild in nature without any meaningful nasal irritation or pain.

• Presented Data Supporting Use of
  
  neffy
  
  During the 2023 AAAAI Meeting: ARS presented positive data supporting neffy during the 2023 American Academy of Allergy, Asthma and Immunology (AAAAI) meeting. Presentations highlighted clinical trial data demonstrating that neffy delivered consistent epinephrine levels to attain a pharmacokinetic (PK) and pharmacodynamic (PD) profile within the range of approved intramuscular (IM) injection products with a dose proportional exposure between once and twice dosing.

  ARS also presented findings from surveys of 300 patients identifying that the needle is the principal reason why patients do not fill their epinephrine prescription today, and why they delay epinephrine use and use OTC products first despite treatment guidelines recommending immediate use of epinephrine.

Fourth Quarter and Full Year 2022 Financial Results

• Cash Position: Cash, cash equivalents and short-term investments were $274.4 million as of December 31, 2022. ARS believes its existing cash, cash equivalents and short-term investments will be sufficient to fund its current operating plan for at least three years.
• R&D Expenses: Research and development (R&D) expenses were $4.7 million for the quarter ended December 31, 2022, and $18.4 million for the year ended December 31, 2022.
• G&A Expenses: General and administrative (G&A) expenses were $10.7 million for the quarter ended December 31, 2022, and $18.5 million for the year ended December 31, 2022. G&A expenses for the fourth quarter increased over the prior three quarters mainly due to costs related to the merger with Silverback Therapeutics, Inc. and beginning preparations for the potential commercialization of neffy.
• Net Loss: Net loss was $14.4 million for the quarter ended December 31, 2022, and $34.7 million for the year ended December 31, 2022.

About Type I Allergic Reactions including Anaphylaxis

Type I severe allergic reactions are serious and potentially life-threatening events that can occur within minutes of exposure to an allergen and require immediate treatment with epinephrine, the only FDA-approved medication for these reactions. While epinephrine autoinjectors have been shown to be highly effective, there are well published limitations that result in many patients and caregivers delaying or not administering treatment in an emergency situation. These limitations include fear of the needle, lack of portability, needle-related safety concerns, lack of reliability, and complexity of the devices. There are approximately 25 to 40 million people in the United States who experience Type I severe allergic reactions. Of those, only 3.3 million currently have an active epinephrine autoinjector prescription, and of those, only half consistently carry their prescribed autoinjector. Even if patients or caregivers carry an autoinjector, more than half either delay or do not administer the device when needed in an emergency.

About ARS Pharmaceuticals, Inc.

ARS is a biopharmaceutical company dedicated to empowering at-risk patients and caregivers to better protect themselves from severe allergic reactions that could lead to anaphylaxis. The Company is developing neffy^® (also referred to as ARS-1), an intranasal epinephrine product in clinical development for patients and their caregivers with Type I allergic reactions including food, medications and insect bites that could lead to life-threatening anaphylaxis. For more information, visit www.ars-pharma.com.

Forward-Looking Statements

Statements in this press release that are not purely historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, ARS’s projected cash runway; the anticipated timing for regulatory review decisions on neffy and the potential approval of neffy; ARS’s commercial readiness for the potential US launch of neffy, if approved, and the timing thereof; ARS’s strategy of pursuing potential partnerships or strategic transactions for neffy in Europe; the estimated addressable patient population for neffy; and other statements that are not historical fact. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “anticipate,” “believe,” “plan,” “expect,” “goal,” “look forward to,” “will,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon ARS’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the ability to obtain and maintain regulatory approval for neffy; results from clinical trials may not be indicative of results that may be observed in the future; potential safety and other complications from neffy; the labelling for neffy, if approved; the scope, progress and expansion of developing and commercializing neffy; the size and growth of the market therefor and the rate and degree of market acceptance thereof vis-à-vis intramuscular injectable products; ARS’s ability to protect its intellectual property position; the impact of government laws and regulations; ARS’s ability to execute its plans and strategies; and uncertainties related to ARS’s capital requirements. Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption “Risk Factors—Risks Related to ARS Pharma” in the company’s definitive merger proxy statement filed with the Securities and Exchange Commission (“SEC”) on October 6, 2022, and under the caption “Risk Factors” in the company’s Annual Report on Form 10-K for the year ended December 31, 2022, being filed with the SEC later today. These documents can also be accessed on ARS’s web page at ir.ars-pharma.com by clicking on the link “Financials & Filings.”

The forward-looking statements included in this press release are made only as of the date hereof. ARS assumes no obligation and does not intend to update these forward-looking statements, except as required by law.

ARS Investor Contact:

Justin Chakma
ARS Pharmaceuticals
[email protected]

ARS Pharmaceuticals, Inc.

Consolidated Balance Sheets

(In thousands, except par value and share amounts)

ARS Pharmaceuticals, Inc.

Consolidated Statements of Operations and Comprehensive Loss

(In thousands, except share and per share information)

MARIETTA, Ga., March 23, 2023 (GLOBE NEWSWIRE) — MiMedx Group, Inc. (Nasdaq: MDXG) (“MIMEDX” or the “Company”), a pioneer and leader in placental biologics, today announced that Peter M. Carlson, Chief Financial Officer, has decided to leave the Company to pursue other opportunities. The Company has initiated a search for its next Chief Financial Officer and Mr. Carlson will be assisting MIMEDX throughout this process to ensure a smooth transition.

“In the three years Pete has been with MIMEDX, he has played a critical role bringing excellence to our finance and accounting functions,” stated Joseph H. Capper, MIMEDX Chief Executive Officer. “As a result of his leadership, we are on sound footing. More specifically, Pete helped the Company raise debt and equity to strengthen the Company’s financial position, successfully led MIMEDX out of a de-listing, enhanced our internal controls and was the architect of our new segment reporting. We are extremely appreciative of Pete’s contributions and accomplishments and wish him our sincere best in his future endeavors.”

“MIMEDX has been truly transformed over the last few years. I am proud to have been a part of the process to stabilize and strengthen the Company and feel privileged to have represented such wonderful technology,” stated Mr. Carlson. “I look forward to watching MIMEDX continue to grow and prosper, as my colleagues build on the Company’s momentum.”

About MIMEDX

MIMEDX is a pioneer and leader in placental biologics, developing and distributing placental tissue allografts to help address unmet clinical needs in multiple sectors of healthcare, including the Advanced Wound Care market as well as in surgical recovery settings. MIMEDX is also focused on advancing a promising late-stage pipeline opportunity targeted at decreasing pain and improving function for patients with knee osteoarthritis. Our products are derived from human placental tissues and processed using our proprietary methods, including the Company’s own PURION® process. We employ Current Good Tissue Practices, Current Good Manufacturing Practices, and terminal sterilization to produce our allografts. MIMEDX has supplied over two million allografts, through both direct and consignment shipments. For additional information, please visit www.mimedx.com.

Contact:

Matt Notarianni
Investor Relations
470-304-7291
[email protected]

Advancing VYD222 into clinical development
based on in vitro data
demonstrating neutralizing activity against multiple important variants of concern, including XBB.1.5

Anticipate near-term designation of an additional monoclonal antibody against SARS-CoV-2 with complementary binding properties to VYD222 for development

Well capitalized with $372 million in cash, cash equivalents and marketable securities expected to support operating runway into second half of 2024

Conference call scheduled for Thursday, March 23

^rd

at 4:30 p.m. ET

WALTHAM, Mass., March 23, 2023 (GLOBE NEWSWIRE) — Invivyd, Inc. (Nasdaq: IVVD), a clinical-stage biopharmaceutical company on a mission to protect the vulnerable from serious viral infectious diseases, today announced financial results for the year ended December 31, 2022, and recent business highlights.

“2022 was a transformational year for Invivyd as we reformed our strategy and are now squarely focused on leveraging our integrated discovery platform to advance a stream of SARS-CoV-2 monoclonal antibody (mAb) candidates. There continues to be an urgent, unmet medical need for vulnerable populations given no antibodies are currently authorized,” said Dave Hering, CEO of Invivyd. “We are pleased to have recently announced plans to advance VYD222 into clinical development as a novel mAb therapeutic option for COVID-19. We believe VYD222 is a highly attractive candidate that could potentially leverage adintrevimab’s strong clinical data package to support potential accelerated development. Importantly, in vitro data on VYD222 has shown neutralizing activity against multiple currently circulating variants of concern, including those that led to the obsolescence of products previously authorized in the U.S. We continue to plan for a Phase 1 clinical trial start in Q1 2023. Assuming positive Phase 1 data, we anticipate rapidly initiating Phase 3 pivotal trials that could support regulatory filings globally.”

Recent Business Highlights

• Earlier in March 2023, the company announced plans to advance VYD222 into the clinic as a mAb therapeutic option for COVID-19 with a focus on serving vulnerable populations. VYD222 is one of the two mAb components of NVD200, a combination mAb product candidate that Invivyd had previously selected for advancement prior to evolution in the current global COVID-19 regulatory paradigm. The company is prioritizing the clinical development of VYD222 instead of NVD200 with the aim of providing patients with a therapeutic option for COVID-19 as quickly and efficiently as possible. VYD222 was engineered from adintrevimab, Invivyd’s investigational mAb that has a robust safety data package and demonstrated clinically meaningful results in global Phase 3 clinical trials for both the prevention and treatment of COVID-19. The adintrevimab clinical data package has the potential to support accelerated development of VYD222. 
• In March 2023, the company and collaborators published an article in the journal Science Translational Medicine titled, “Antibody-mediated protection against symptomatic COVID-19 can be achieved at low serum neutralizing titers.” The work builds on vaccine studies demonstrating neutralizing antibody titers as correlates of protection against disease and could inform the evolution of regulatory frameworks for therapeutic antibodies.
• The company continues to leverage its integrated discovery platform, mapping common mutational escape routes to predict potential products to treat future variants of the SARS-CoV-2 virus. Beyond VYD222, the company is continuously monitoring evolving variants and engineering to optimize pipeline candidates and has initiated a new mAb campaign that targets re-engineering and affinity maturations of current molecules against the most recent variants of concern, such as XBB.1.5. The company is currently evaluating several of these candidates in preclinical studies to support nomination of additional candidates for IND-enabling and clinical development.
• In December 2022, the company was invited to participate in a U.S. Food and Drug Administration-European Medicines Agency workshop to explore expedited development pathways for mAbs. The meeting featured presentations by scientists, clinicians, regulators, and industry representatives to discuss alternative strategies to support the expedited availability of novel monoclonal antibody therapies. The company aims to leverage evolving COVID-19 regulatory paradigms to deliver this much-needed product for immunocompromised individuals and other vulnerable populations.
• The company has announced key leadership positions that broaden the company’s industry expertise in support of its development and commercial planning.
  • Appointed Jeremy Gowler as chief operating officer and commercial officer. Mr. Gowler brings 20 years of experience across multiple key commercialization functions throughout the product lifecycle.
  • Promoted Pete Schmidt, M.D. to chief medical officer. Dr. Schmidt had served as Invivyd’s vice president of clinical research for the past two years, and is now responsible for overseeing all medical, clinical development and regulatory activities at Invivyd.

Year End 2022 Financial Results

• Cash Position: Cash, cash equivalents and marketable securities were $372 million as of December 31, 2022.
• Cash Runway: Based on current operating plans, Invivyd expects its existing total cash, cash equivalents and marketable securities will enable the company to fund its operating expenses into the second half of 2024.
• Research & Development (R&D) Expenses (including In-process Research & Development): R&D expenses were $183.6 million for the year ended December 31, 2022, compared to $190.4 million for the comparable period of 2021. This decrease is attributable to wind-down of adintrevimab clinical trials, partially offset by an increase in contract manufacturing and personnel-related expenses, including an increase of $6.2 million of stock-based compensation expense.
• Selling, General & Administrative (SG&A) Expenses: SG&A expenses were $47.0 million for the year ended December 31, 2022, compared to $36.5 million for the comparable period of 2021. This increase is attributable to higher public company costs and personnel-related expenses.
• Warrant Expense: Warrant expense was $17.4 million for the year ended December 31, 2022, compared to $0 for the comparable period of 2021. This increase is attributable to a one-time charge associated with warrants issued to Population Health Partners, L.P. (PHP) as compensation for consulting services to be provided by PHP to the company under the agreement entered into in the fourth quarter of 2022.
• Net Loss and Net Loss per Share: Net loss was $241.3 million for the year ended December 31, 2022, compared to $226.8 million for the comparable period in 2021. Basic and diluted net loss per share was $2.23 for the year ended December 31, 2022, compared to $5.32 for the comparable period in 2021.
  • The net loss of $241.3 million for the year ended December 31, 2022 included a one-time charge of $17.4 million related to the fair value of the warrants issued to PHP.

Conference Call

In connection with this announcement, Invivyd will host a conference call and webcast today at 4:30 p.m. ET. A live audio webcast will be available at invivyd.com/investors. Interested parties may also register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company’s investor website approximately two hours after the call’s conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.

About Invivyd

(Nasdaq: IVVD)
Invivyd, Inc., is a biopharmaceutical company on a mission to rapidly and perpetually deliver antibody-based therapies that protect vulnerable people from the devastating consequences of circulating viral threats, beginning with SARS-CoV-2. ​​Invivyd’s technology works at the intersection of evolutionary virology, predictive modeling, and antibody engineering, and is designed to identify high-quality, long-lasting antibodies with a high barrier to viral escape. The company is generating a robust pipeline of product candidates which could be used in prevention or treatment of serious viral diseases, starting with COVID-19 and expanding into influenza and other high-need indications. Visit https://invivyd.com/ to learn more.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “could,” “expects,” “intends,” “potential,” “projects,” and “future” or similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements include statements concerning, among other things, the ability for Invivyd, other companies or combination of companies and industry representatives to influence regulators to change or adopt new development pathways or timelines; the ability of Invivyd to accelerate development timelines for the unmet need for treatment of COVID-19; the interest or acceptance by regulatory authorities of regulatory and clinical strategies to support potentially expedited development of novel monoclonal antibody therapies; the potential for success and or expedited discovery, development, or commercialization of antibody therapies for COVID-19; the continued unmet need for prevention and treatment of COVID-19, particularly for immunocompromised and other vulnerable populations; the viability and acceptability of new regulatory strategy, policy or approach to drug development and the potential of the same to maintain pace with changing COVID-19 variants; the future of the COVID-19 landscape including the expectation of continued evolution and emergence of new variants and subvariants; our ongoing research and clinical development plans and the timing thereof; our plans to advance VYD222 or other early stage candidates as a potential prophylaxis and treatment option for COVID-19, including disease caused by most variants, as either a single or combination agent; the potential for VYD222 or other product candidates to demonstrate activity against predominant SARS-CoV-2 variant(s) in the U.S. and globally; the potential for the clinical data package resulting from clinical trials of adintrevimab to support accelerated VYD222 monotherapy development; our plans to advance VYD222 into the clinic; our expectations that we will be able to achieve regulatory alignment and advance pivotal studies with VYD222; our expectations regarding the anticipated timeline of our cash runway; anticipated benefits to the company of recent executive officer appointments and promotions; our plans, technology and resources to develop therapeutic or preventative options for other infectious diseases, such as additional coronaviruses and seasonal influenza, in the U.S. and globally; and other statements that are not historical fact. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements and you should not place undue reliance on our forward-looking statements. These forward-looking statements involve risks and uncertainties that could cause our actual results to differ materially from the results described in or implied by the forward-looking statements, including, without limitation: the ability to gain alignment with the applicable regulatory authorities on the clinical development pathway for VYD222 and the timing thereof; the ability for Invivyd and/or other companies, scientists, clinicians or industry representatives to impact the strategy, policy or approach to drug development drafted or applied by regulatory authorities, including the FDA and EMA; the impact of any such change on the speed or success of development and commercialization of antibodies for the prevention and/or treatment of COVID-19; the ability of the company to generate and utilize tools to discover and develop antibodies to treat current and potential future variants; the impacts of the COVID-19 pandemic on our business and those of our collaborators, our clinical trials and our financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials; the predictability of clinical success of VYD222 or other pipeline candidates or combination of candidates based on neutralizing activity in pre-clinical studies; variability of results in models used to predict activity against SARS-CoV-2 variants of concern; clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process, including the outcome of our discussions with regulatory authorities concerning our clinical trials; whether VYD222 or any other pipeline candidate or combination of candidates is able to demonstrate activity against predominant SARS-CoV-2 variant(s) in the U.S. and globally; whether we are able to successfully submit an emergency use authorization in the future, and the outcome of any such emergency use authorization submission; whether research and development efforts will improve efficacy of adintrevimab against predominant variants or identify additional monoclonal antibodies or combination of antibodies for the prevention and treatment of COVID-19 and other infectious diseases; whether research and development efforts will identify and result in safe and effective therapeutic or preventative options for other infectious diseases in the U.S. or globally and whether we have adequate funding to meet future operating expenses and capital expenditure requirements. Other factors that may cause our actual results to differ materially from those expressed or implied in the forward-looking statements in this press release are described under the heading “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2022 filed with the Securities and Exchange Commission (the “SEC”), and in our other filings with the SEC, and in Invivyd’s future reports to be filed with the SEC and available at www.sec.gov. Such risks may be amplified by the impacts of the COVID-19 pandemic. Forward-looking statements contained in this press release are made as of this date, and Invivyd undertakes no duty to update such information whether as a result of new information, future events or otherwise, except as required under applicable law.

This press release contains hyperlinks to information that is not deemed to be incorporated by reference in this press release.

Contacts

Media Contact:

Kate Burdick, Evoke Canale
860-462-1569
[email protected]

Investor Contact:

Chris Brinzey, ICR Westwicke
339-970-2843
[email protected]

INVIVYD, INC.

CONSOLIDATED BALANCE SHEETS

(UNAUDITED)

(In thousands, except share and per share amounts)

INVIVYD, INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(UNAUDITED)

(In thousands, except share and per share amounts)

<sup>(1) Includes related-party amounts of $8,154 and $4,150 for the years ended December 31, 2022 and 2021, respectively.

(2) Includes related-party amounts of $4,400 and $7,500 for the years ended December 31, 2022 and 2021, respectively.

(3) Includes related-party amounts of $17,373 and $0 for the years ended December 31, 2022 and 2021, respectively.</sup>

Phase 2 trials of VTX958 (TYK2 inhibitor) are ongoing in plaque psoriasis, Crohn’s disease and psoriatic arthritis, with topline Phase 2 data in plaque psoriasis expected in Q4 2023

The Phase 2 trial of VTX002 (S1P1R modulator) in ulcerative colitis (UC) is on track to complete enrollment by mid-2023, with topline data expected in H2 2023

Initiated a Phase 2 proof-of-mechanism trial of VTX2735 (peripheral NLRP3 inhibitor) in patients with cryopyrin-associated periodic syndrome (CAPS)

Cash, cash equivalents and marketable securities of $356.6 million at the end of 2022 plus an additional $48.4 million in net proceeds raised in Q1 2023 under our “At-the-market” (ATM) program are expected to fund operations into 2025

Ventyx to host conference call and webcast today at 4:30PM ET

ENCINITAS, Calif., March 23, 2023 (GLOBE NEWSWIRE) — Ventyx Biosciences, Inc. (Nasdaq: VTYX) (“Ventyx”), a clinical-stage biopharmaceutical company focused on advancing novel oral therapies that address a broad range of inflammatory diseases with significant unmet medical need, today announced financial results for the fourth quarter and full year ended December 31, 2022, and highlighted recent pipeline and business progress.

“I am proud of our team’s exceptional performance in 2022 as we continued to build an amazing team, strengthen our financial position and successfully advance our portfolio of wholly-owned, internally-discovered small molecules targeting large immunology markets with high unmet medical need,” said Raju Mohan, Chief Executive Officer. “We look forward to a potentially breakthrough year in 2023, with multiple key clinical milestones, including topline Phase 2 data for VTX002 in ulcerative colitis expected in H2 2023 and topline Phase 2 data for VTX958 in plaque psoriasis expected in Q4 2023. Additionally, we recently initiated a Phase 2 proof-of-mechanism trial of VTX2735 in CAPS patients, and we look forward to initiating a Phase 1 trial of our novel CNS-penetrant NRLP3 inhibitor VTX3232 in the first half of 2023.”

Pipeline Updates

• VTX958 (TYK2 Inhibitor): Enrollment is ongoing in the Phase 2 SERENITY trial of VTX958 in moderate to severe plaque psoriasis, the Phase 2 HARMONY trial in moderately to severely active Crohn’s disease and the Phase 2 TRANQUILITY trial in active psoriatic arthritis. Topline data from the Phase 2 SERENITY psoriasis trial are anticipated in Q4 2023. Topline readouts from the Phase 2 HARMONY and Phase 2 TRANQUILITY trials are expected in 2024.

  Additionally, we are developing an extended release (ER) tablet formulation for VTX958 in collaboration with leading formulation development partners. We expect to provide an update on VTX958 ER tablet development in mid-2023 following completion of initial in-human testing. We have also begun a wide range of other Phase 3 enabling activities.

• VTX002 (S1P1R Modulator): We continue to make significant progress enrolling the Phase 2 trial of VTX002 in moderately to severely active ulcerative colitis. We expect to complete enrollment by mid-2023 and topline results are anticipated in the second half of 2023. At our investor R&D Day in January, we announced preliminary pharmacodynamic (PD) data from the open-label extension of the ongoing Phase 2 trial. Among patients completing Week 26 (13 weeks of blinded therapy followed by 13 weeks of open-label treatment with VTX002 60mg) as of January 15, 2023, a mean reduction from baseline in absolute lymphocyte count of 74% was observed. We believe these preliminary data suggest VTX002 may achieve a greater pharmacodynamic response compared to other S1P receptor modulators approved or in development for the treatment of ulcerative colitis, which may translate to an improved efficacy profile in UC.
• VTX2735 (Peripheral NLRP3 Inhibitor): We have initiated a Phase 2 proof-of-mechanism trial of VTX2735 in patients with familial cold autoinflammatory syndrome (FCAS). FCAS is the most common subset of cryopyrin-associated periodic syndrome (CAPS), a group of rare autoinflammatory conditions caused by gain-of-function mutations in the NLRP3 gene. In addition to CAPS, we believe systemic NLRP3 inhibition with VTX2735 may have therapeutic potential across a broad range of chronic inflammatory conditions that are characterized by NLRP3-induced excess IL-1β, including dermatologic, rheumatologic and cardiovascular diseases.
• VTX3232 (CNS-penetrant NLRP3 Inhibitor): We expect to initiate a Phase 1 trial of VTX3232 during the first half of 2023. The Phase 1 trial is expected to characterize the safety, target engagement and bioavailability of VTX3232 in the central nervous system of healthy volunteers. We believe that the profile of VTX3232 may define it as a class-leading therapeutic for a range of neuroinflammatory conditions with high unmet medical need, including Parkinson’s disease, Alzheimer’s disease and amyotrophic lateral sclerosis, among others.
• Discovery Programs: We continue our lead optimization efforts on the recently disclosed program focused on small molecule antagonists of IL-4Rα, a target validated by biologics in multiple large autoimmune indications, including atopic dermatitis, asthma and eosinophilic esophagitis. We are advancing multiple internally discovered novel chemical series through lead optimization with the goal of establishing in vivo proof-of-concept and nominating a lead candidate in 2023.

Fourth Quarter and Full Year 2022 Financial Results:

The amounts presented below for the fourth quarter and full year ended December 31, 2022 reflect the financial results of Ventyx Biosciences, Inc. and its two wholly-owned subsidiaries, Oppilan Pharma Limited (Oppilan) and Zomagen Biosciences Ltd. (Zomagen), on a consolidated basis. The amounts presented below in the Consolidated Statements of Operations and Comprehensive Loss for the year ended December 31, 2021 reflect the financial results of Ventyx, as well as the financial results of Oppilan and Zomagen from the date of acquisition (February 26, 2021), on a consolidated basis.

• Cash Position: Cash, cash equivalents and marketable securities were $356.6 million as of December 31, 2022. Subsequent to December 31, 2022, we raised $48.4 million in net proceeds through our “At-the-market” (ATM) program. We believe our current cash, cash equivalents and marketable securities are sufficient to fund our planned operations into 2025.
• Research
  and Development (R&D) expenses: R&D expenses were $30.2 million for the fourth quarter of 2022, compared to $13.8 million for the fourth quarter of 2021. R&D expenses were $87.7 million for the year ended December 31, 2022, compared to $58.5 million for the year ended December 31, 2021.
• General and Administrative (G&A) expenses: G&A expenses were $8.4 million for the fourth quarter of 2022, compared to $4.0 million for the fourth quarter of 2021. G&A expenses were $25.4 million for the year ended December 31, 2022, compared to $8.7 million for the year ended December 31, 2021.
• Net loss: Net loss was $35.2 million for the fourth quarter of 2022, compared to $17.8 million for the fourth quarter of 2021. Net loss was $108.4 million for the year ended December 31, 2022, compared to $83.7 million for the year ended December 31, 2021.

Conference Call Information

Ventyx will host a conference call today at 4:30 p.m. ET to discuss its fourth quarter and full year 2022 financial results and provide a corporate update. To participate in the conference call, please dial (800) 343-4136 (U.S.) or (203) 518-9783 (international) and reference passcode VTYXQ422. A live audio webcast will be available in the Investors section of the company’s website at www.ventyxbio.com. A recording of the webcast will be available for thirty days following the call.

About Ventyx Biosciences

Ventyx is a clinical-stage biopharmaceutical company focused on developing innovative oral medicines for patients living with autoimmune and inflammatory disorders. We believe our ability to efficiently discover and develop differentiated drug candidates will allow us to address important unmet medical need with novel oral therapies that can shift immunology markets from injectable to oral drugs. Our current pipeline includes three internally discovered clinical programs targeting TYK2, S1P1R and NLRP3, positioning us to become a leader in the development of oral immunology therapies. Ventyx is headquartered in Encinitas, California. For more information about Ventyx, please visit www.ventyxbio.com.

Forward-Looking Statements

Ventyx cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on Ventyx’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: management’s beliefs regarding the potential of Ventyx’s product candidates; the anticipated timing of commencement, enrollment and completion of clinical trials for Ventyx’s product candidates; the anticipated timing for releasing topline data for clinical trials of Ventyx’s product candidates; the anticipated timing for providing an update on VTX958 ER tablet development; and the expected timeframe for funding Ventyx’s operating plan with current cash, cash equivalents and marketable securities. The inclusion of forward-looking statements should not be regarded as a representation by Ventyx that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Ventyx’s business, including, without limitation, potential delays in the commencement, enrollment and completion of clinical trials; Ventyx’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; disruptions in the supply chain, including raw materials needed for manufacturing and animals used in research, delays in site activations and enrollment of clinical trials; the results of preclinical studies and early clinical trials not necessarily being predictive of future results; interim results not necessarily being predictive of final results; the potential of one or more outcomes to materially change as a trial continues and more patient data become available and following more comprehensive audit and verification procedures; regulatory developments in the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of Ventyx’s product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Ventyx’s ability to obtain and maintain intellectual property protection for its product candidates; the use of capital resources by Ventyx sooner than expected; disruption to Ventyx’s operations from the ongoing global outbreak of the COVID-19 pandemic, or from the ongoing military conflict in Ukraine, including clinical trial delays; and other risks described in Ventyx’s prior press releases and Ventyx’s filings with the Securities and Exchange Commission (SEC), including in Part I, Item 1A (Risk Factors) of Ventyx’s Annual Report on Form 10-K for the year ended December 31, 2022 filed on or about the date hereof, and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Ventyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

Investor Relations Contact

Patti Bank
Managing Director
ICR Westwicke
(415) 513-1284
[email protected]

• Initial clinical data supports founding vision that engineered hematopoietic stem cells can enable treatment options after AML transplant
  
• Additional trem-cel data expected by year-end 2023; VCAR33
  
  ^ALLO
  
  on track for IND submission in first half of 2023
• $116M financing extends expected cash runway into Q1 2025
  

CAMBRIDGE, Mass., March 23, 2023 (GLOBE NEWSWIRE) — Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period and full year ended December 31, 2022, and provided a business update. 

“We are encouraged with the initial proof of concept demonstrated in patients treated in our VBP101 study,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. “We remain focused on rapid enrollment and plan to share additional clinical data later this year. Our IND for VCAR33^ALLO is on-track for submission in the first half of 2023 which, together with trem-cel, has the potential to transform outcomes for patients with blood cancers.”

Corporate Highlights

Initial VBP101 clinical data represents an important milestone for Vor Bio’s founding vision and further validates the Company’s novel platform. Clinical data presented at the 2023 TANDEM meetings (Transplantation & Cellular Therapy Meetings of ASTCT^™ and CIBMTR^®) in February 2023 demonstrated sustained hematopoiesis in the first patient treated with trem-cel five months (147 days) post-transplant and Mylotarg (gemtuzumab ozogamicin) was well-tolerated through three cycles of treatment at the initial dose level of 0.5 mg/m². Mylotarg first-dose pharmacokinetics revealed 0.5 mg/m² achieved Cmax and AUC parameters equivalent to Mylotarg doses of 1-2 and 4-5 mg/m², respectively, potentially due to the decreased CD33 antigen sink. CD33 deletion was observed in donor cells of myeloid and lymphoid origin which were both enriched following Mylotarg, suggesting that CD33 is expressed early in hematopoietic differentiation and that Mylotarg treatment effectively removes CD33-positive cells. Due to detectable measurable residual disease (MRD), the patient was moved to other therapies following administration of the third dose of Mylotarg and subsequently relapsed with CD33+ blasts.

A second patient successfully received a trem-cel transplant and showed robust cell recovery with neutrophil engraftment occurring at Day 11 and platelet recovery on Day 17. Trem-cel was well tolerated in both patients.

$116 million financing extends Company’s expected cash runway into Q1 2025. In December 2022, Vor Bio announced the pricing of an underwritten offering and a private placement, with combined gross proceeds of approximately $115.8 million. Vor Bio intends to use the net proceeds from the financing primarily to fund the continued clinical development of pipeline programs and for working capital and general corporate purposes. 

GMP qualification of in-house clinical manufacturing facility underway. Current Good Manufacturing Practices (cGMP) qualification activities at the new facility are well underway, and the Company is on track to begin clinical manufacturing of VCAR33^ALLO post IND submission. The Company plans to commence trem-cel production at the in-house facility in 2023.

Strategic additions to Clinical and Scientific Advisory Board. As the Company continues to evolve toward providing next-generation transplants for patients, it is building out a world-class Clinical and Scientific Advisory Board comprised of luminaries in the field who can provide the Company with deep advisory expertise in genome engineering, hematopoietic stem cell (HSC) biology, cancer immunotherapy and clinical development of therapies to treat blood cancers. Scientific and Clinical Advisors currently include Siddhartha Mukherjee, MD, DPhil; Hans-Peter Kiem, MD, PhD; Malcolm K Brenner, MD, PhD; Steven Devine, MD; Rob Soiffer, MD; Eric Sievers, MD; and Yi-Bin Chen, MD.

For more information, visit our website at: https://www.vorbio.com/about/scientific-clinical-advisors/.

Program Updates

Trem-cel (formerly VOR33): Trem-cel is a genome-edited allogeneic hematopoietic stem cell transplant (HSCT), that is lacking the CD33 protein. It is designed to replace standard of care transplants for patients suffering from acute myeloid leukemia (AML) and potentially other blood cancers. Trem-cel has the potential to enable powerful targeted therapies in the post-transplant setting including CD33-targeted CAR-Ts.

• The Company continues to actively enroll patients into the VBP101 clinical study and is progressing as planned with dose escalation of Mylotarg per the 3+3 schema in the protocol.
• An encore poster presentation of VBP101 data has been accepted at the 49th Annual Meeting of the EBMT to be held in Paris, France, April 23-26, 2023.
• The Company expects to share new data from additional patients transplanted with trem-cel and treated with Mylotarg at scientific/medical forums by year-end 2023.

VCAR33

^ALLO

: VCAR33^ALLO is a T-cell therapy derived from allogeneic healthy donors using a chimeric antigen receptor (CAR) specifically binding to CD33.

• The Company is on-track to submit an IND in the first half of 2023.
• The initial clinical trial will focus on patients who have relapsed following allogeneic stem cell transplant, where T cells harvested from the original donor are used as starting material for the drug product.
• The Company intends to evaluate VCAR33^ALLO in combination with trem-cel as a Treatment System, aiming at prolonged remissions or cures following transplant.

Upcoming Milestones

• VCAR33^ALLO IND submission expected in the first half of 2023
• Additional trem-cel engraftment and hematologic protection data updates expected by year-end 2023

Fourth Quarter and Full Year 2022 Financial Results

• Cash Position: Cash, cash equivalents and investments were $230.2 million as of December 31, 2022, which is projected to fund operations into the first quarter of 2025.
• Research & Development (R&D) Expenses: R&D expenses for the fourth quarter of 2022 were $17.1 million, compared to $12.7 million for the fourth quarter of 2021, and for the year ended December 31, 2022, were $64.6 million, compared to $47.5 million for the year ended December 31, 2021. The increase in R&D expenses was primarily due to an increase in personnel expenses, including an increase in stock compensation expense, an increase in facility costs from our laboratory and cGMP manufacturing facility expansion, and an increase in clinical, manufacturing and consulting expenses as a result of the ongoing trem-cel clinical trial and the development of the VCAR33^ALLO program.
• General & Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2022 were $7.7 million, compared to $5.6 million for the fourth quarter of 2021, and for the year ended December 31, 2022, were $28.9 million, compared to $21.5 million for the year ended December 31, 2021. The increase in G&A expense was primarily due to increased personnel expenses, including an increase in in stock compensation expense, an increase in facilities and other expenses as a result of our corporate headquarters office expansion, and an increase in professional fees.
• Net Loss: Net loss for the fourth quarter of 2022 was $23.9 million, compared to $18.3 million for the fourth quarter of 2021, and for the year ended December 31, 2022, was $92.1 million, compared to $68.9 million for the year ended December 31, 2021.

About Vor Bio

Vor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells to enable targeted therapies post-transplant. For more information, visit: www.vorbio.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “aim,” “anticipate,” “can,” “continue,” “could,” “design,” “enable,” “expect,” “initiate,” “intend,” “may,” “on-track,” “ongoing,” “plan,” “potential,” “should,” “target,” “update,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include Vor Bio’s statements regarding the potential of its product candidates to positively impact quality of life and alter the course of disease in the patients it seeks to treat, the timing of patient enrollment in clinical trials and the availability of data therefrom, the timing of regulatory filings, the expected safety profile of its product candidates, the potential cGMP qualification of its manufacturing facility and the success and timing of manufacturing clinical supply for its product candidates, its intentions to use VCAR33^ALLO in combination with trem-cel as a Treatment System, its potential upcoming milestones, its intended use of proceeds from capital raising activities, cash runway and expected capital requirements. Vor Bio may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Vor Bio’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; the success of Vor Bio’s in-house manufacturing capabilities and efforts; and availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in Vor Bio’s most recent annual or quarterly report and in other reports it has filed or may file with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law.

Contact:
Investors & Media
Sarah Spencer
+1 857-242-6076
[email protected]