Market Newsdesk

HOUSTON, Nov. 13, 2020 (GLOBE NEWSWIRE) — Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize™ liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, today announced its financial results for the third quarter ended September 30, 2020 and provided an update on recent corporate developments.

“We made meaningful progress across our programs throughout the third quarter despite continued headwinds related to the COVID-19 pandemic. Importantly, enrollment continues in Stage 2 of our Phase 2 trial of prexigebersen (BP1001), a liposomal Grb2 antisense, as a combination treatment for patients suffering with acute myeloid leukemia (AML),” said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. “We further strengthened our intellectual property portfolio with a strategic patent providing broad protection for application of prexigebersen in the treatment of a variety of cancers in combination with front-line therapies.”

“We remain on track to initiate a Phase 1 study of prexigebersen for the treatment of solid tumors by year end. This is a particularly important advancement for Bio-Path as it marks our first-in-human study in solid tumors, an area of significant need where current treatment options are often ineffective,” continued Mr. Nielsen.

Recent Corporate


Highlights

• Receive
  d
  Notice of Allowance for Strategic Patent for Prexigebersen in
  Combination with Front
  –
  Line
  Therapies. In October, Bio-Path announced that the United States Patent and Trademark Office had issued a notice of allowance for claims related to the Company’s lead product candidate, prexigebersen, in combination with either a cytidine analogue, such as decitabine, or the Bcr-Abl tyrosine kinase inhibitors dasatinib and nilotinib. The patent provides broad protection for application of prexigebersen in the treatment of a variety of cancers in combination with front-line therapies.

• Announced First Patient Dosed in Amended Stage 2 of the Phase 2 Clinical Trial Evaluating Prexigebersen in Acute Myeloid Leukemia. In August, Bio-Path announced the enrollment and dosing of the first patient in the amended Stage 2 of the Phase 2 clinical study of prexigebersen for the treatment of AML in combination with front-line therapy decitabine and venetoclax.

Financial Results for


the


Third


Quarter


Ended


September


30


, 20


20

• The Company reported a net loss of $3.0 million, or $0.80 per share, for the three months ended September 30, 2020, compared to a net loss of $2.2 million, or $0.78 per share, for the three months ended September 30, 2019.
• Research and development expenses for the three months ended September 30, 2020 increased to $2.0 million, compared to $1.4 million for the three months ended September 30, 2019 primarily due to increased enrollment for our Phase 2 clinical trial of prexigebersen in AML, as well as increased preclinical study expenses.
• General and administrative expenses for the three months ended September 30, 2020 increased to $1.0 million, compared to $0.9 million for the three months ended September 30, 2019 primarily due to increased franchise tax expense.
• As of September 30, 2020, the Company had cash of $12.1 million, compared to $20.4 million at December 31, 2019. Net cash used in operating activities for the nine months ended September 30, 2020 was $8.4 million compared to $6.1 million for the comparable period in 2019. Subsequent to September 30, 2020, Bio-Path issued 850,000 shares of its common stock for gross proceeds of approximately $4.6 million through its at-the-market offering agreement with H.C. Wainwright.

Conference Call and Webcast Information

Bio-Path Holdings will host a conference call and webcast today at 8:30 a.m. ET to review these third quarter 2020 financial results and to provide a general update on the Company. To access the conference call please dial (844) 815-4963 (domestic) or (210) 229-8838 (international) and refer to the conference ID 5878804. A live audio webcast of the call and the archived webcast will be available in the Media section of the Company’s website at www.biopathholdings.com.

About Bio-Path Holdings, Inc.

Bio-Path is a biotechnology company developing DNAbilize®, a novel technology that has yielded a pipeline of RNAi nanoparticle drugs that can be administered with a simple intravenous infusion. Bio-Path’s lead product candidate, prexigebersen (BP1001, targeting the Grb2 protein), is in a Phase 2 study for blood cancers and prexigebersen-A, a drug product modification of prexigebersen, is under consideration by the FDA to commence Phase 1 studies in solid tumors. This is followed by BP1002, targeting the Bcl-2 protein, where it is being evaluated in lymphoma clinical studies.

For more information, please visit the Company’s website at http://www.biopathholdings.com.

Forward-Looking Statements

This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws. These statements are based on management’s current expectations and accordingly are subject to uncertainty and changes in circumstances. Any express or implied statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Any statements that are not historical facts contained in this release are forward-looking statements that involve risks and uncertainties, including the impact, risks and uncertainties related to COVID-19 and actions taken by governmental authorities or others in connection therewith, Bio-Path’s ability to raise needed additional capital on a timely basis in order for it to continue its operations, have success in the clinical development of its technologies, the timing of enrollment and release of data in such clinical studies and the accuracy of such data, limited patient populations of early stage clinical studies and the possibility that results from later stage clinical trials with much larger patient populations may not be consistent with earlier stage clinical trials, and such other risks which are identified in Bio-Path’s most recent Annual Report on Form 10- K, in any subsequent quarterly reports on Form 10-Q and in other reports that Bio-Path files with the Securities and Exchange Commission from time to time. These documents are available on request from Bio-Path Holdings or at www.sec.gov. Bio-Path disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact Information:
                        

I
nvestors

Will O’Connor
Stern Investor Relations, Inc.
212-362-1200
[email protected]   

Doug Morris
Investor Relations
Bio-Path Holdings, Inc.
832-742-1369

—


Data p


resented at the 2020


Annual Scientific Meeting of the


American College of Allergy, Asthma & Immunology (ACAAI)


—

RESEARCH TRIANGLE PARK, N.C., Nov. 13, 2020 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced data from a cross-sectional study among patients, caregivers and physicians capturing the burden of injectable prophylactic therapy experienced by hereditary angioedema (HAE) patients and caregivers, and differences in perceptions between physicians and HAE patients.

The three abstracts were presented at the 2020 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is being conducted virtually from November 13-15.

Poster presentations:

• Patient Perspectives on the Treatment Burden of Injectable Medication Administration for Hereditary Angioedema (#160)
• Prophylactic Treatment Burden: Assessment by Caregivers of Patients with Hereditary Angioedema (#161)
• Understanding Differences in Perceptions of Hereditary Angioedema Treatment Burden May Improve Patient-Physician Treatment Care Dialogue (#162)

“These data are consistent across HAE patients, caregivers and treating physicians showing many patients experience a significant treatment burden associated with current prophylactic HAE therapies. New therapies with easier routes of administration may meet a significant unmet need for HAE patients seeking improved quality of life,” said study lead Cristine Radojicic, M.D., assistant professor of medicine at Duke University School of Medicine.

Overall, the burden of treatment reported across all groups surveyed suggests an unmet need still remains in HAE clinical management. These study findings collectively highlight the opportunity to strengthen the shared decision making between patients and physicians with more effective dialogue about the burden of treatment and patients’ individual needs and preferences.

Following is a brief summary of the data from the cross-sectional study conducted via three double-blinded surveys with HAE patients (n=75), caregivers (n=30) and physicians (n=109), respectively:

• Almost nine in 10 patients with HAE report they have learned to tolerate difficult aspects of their treatment and 58 percent report they are tired of their injections. Even though patients are satisfied with their current prophylactic medications, 86 percent are still interested in a less burdensome route of administration.
• Over 50 percent of caregivers agree it was challenging to learn how to administer HAE treatment, specifically gaining comfort with using needles and learning how to self-administer. Seventy-one percent of caregivers agree that patients experience needle fatigue with their HAE prophylactic medications and an even greater proportion of caregivers believe a once-daily pill would provide the patient more freedom (86 percent), independence (85 percent), and reduce caregivers’ burden.
• Most physicians (94 percent) and patients (84 percent) agree there is a need for newer and more novel HAE treatments. In addition, 86 percent of caregivers believe that, while their patient is satisfied with current treatment, the patient would still be interested in one that is easier to administer.
• Over 70 percent of physicians surveyed believe that starting prophylaxis treatment was overwhelming, becoming comfortable with needles was intimidating, and learning how to self-administer was challenging for their patients. The study also shows that physicians tend to underestimate time required for preparation and administration of prophylaxis medications. Importantly, despite recognition of the burden with current treatments, there is discordance between patients and physicians regarding the person initiating conversations about medication challenges, suggesting an opportunity to improve the dialogue to help with an individualized approach to the management of HAE.

About BioCryst Pharmaceuticals

BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. BioCryst has several ongoing development programs including ORLADEYO^™ (berotralstat), an oral treatment for hereditary angioedema, BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB^® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst’s first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the Company’s website at www.BioCryst.com.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst’s and its partners’ development, regulatory processes and supply chains, negatively impact BioCryst’s ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst files periodically with the Securities and Exchange Commission; developing and commercializing ORLADEYO or any HAE product candidate may take longer or may be more expensive than planned; BioCryst may not be able to enroll the required number of subjects in planned clinical trials of product candidates; BioCryst may not advance human clinical trials with product candidates as expected; the FDA, EMA, PMDA or other applicable regulatory agency may require additional studies beyond the studies planned for product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on product candidates, may impose a clinical hold with respect to such product candidates, or may withhold market approval for product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its product candidates, manage its growth, and compete effectively; risks related to the international expansion of BioCryst’s business; and actual financial results may not be consistent with expectations, including that 2020 operating expenses and cash usage may not be within management’s expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, all of which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s forward-looking statements.

BCRXW

Contact:


John Bluth
+1 919 859 7910
[email protected]

Catherine Collier Kyroulis
+1 917 886 5586
[email protected]

– Seed Therapeutics to Use Proprietary “Molecular Glue” Protein Degradation Technology to Develop Potential New Medicines –

NEW YORK, Nov. 13, 2020 (GLOBE NEWSWIRE) — Seed Therapeutics (“the Company”), a global research company and subsidiary of BeyondSpring focused on discovering and developing “molecular glues” to degrade disease-causing protein previously believed to be undruggable, announced today that it has entered into a research collaboration and license agreement with Eli Lilly and Company (“Lilly”) to discover and develop new chemical entities (NCEs) that could produce therapeutic benefit through targeted protein degradation (TPD).

The TPD field allows for the targeting of hundreds of proteins that are known to be associated with human diseases but were previously thought to be undruggable. Seed Therapeutics has pioneered a strategy called “molecular glue” to induce the protein degrading machinery which is present in all cells to recognize and degrade the disease-causing protein that is not normally targeted for elimination. More importantly, Seed Therapeutics’ molecular glue program focuses on NCEs with more drug-like chemical properties, differentiated from the strategy of developing proteolysis-targeting chimeras (PROTACs).

Under the terms of the agreement, Seed Therapeutics will receive a $10 million upfront cash payment to fund research, as well as a $10 million equity investment from Lilly. Seed Therapeutics will also be eligible to receive up to approximately $780 million in potential pre-clinical and clinical development, regulatory and commercial milestones, as well as tiered royalties on net sales of products that result from the collaboration. The transaction is subject to customary closing conditions.

“This agreement further allows us to advance our pioneering platform to deliver new molecules targeting proteins that cause human diseases,” added Dr. Lan Huang, CEO of both Seed Therapeutics and BeyondSpring. “Our alliance with Lilly is the catalyst for the world-class team at Seed Therapeutics to begin developing a pipeline of TPD therapies for diseases in which common strategies have failed.”

“Our pre-clinical research and licensing collaboration with Seed Therapeutics will enable both companies to better study the potential of targeted protein degradation to support the development of future medicines,” said Dr. Utpal Singh, Ph.D., Vice President of Discovery Chemistry at Lilly.

About Seed Therapeutics

Seed Therapeutics, a subsidiary of BeyondSpring (NASDAQ: BYSI), is a global research company focused on harnessing and engineering molecules that use “molecular glue” protein degradation to attack previously believed undruggable targets. Backed by a comprehensive intellectual property portfolio, Seed Therapeutics’ mission is to positively impact human health by creating novel protein degradation therapeutics for the treatment of various severe diseases that currently have limited options for patients and their families.

The great majority of approved treatments for human diseases act by binding molecular targets in or outside of cells to impact target-related signaling or actions. The cellular targets of drugs and drug candidates discovered with this typical strategy are predominately proteins, the work-horse of cells that, when gone astray, contribute to disease onset and / or progression. Importantly, less than 30 percent of proteins thought to be involved in diseases are likely to be “druggable” utilizing this drug development strategy. Therapeutic development in many serious indications has, therefore, suffered due to a lack of proteins that are druggable, rather than being due to a lack of understanding the biology of the disease.

Seed Therapeutics is overcoming this challenge by developing novel therapies that aim to inhibit the function of disease-causing proteins, or proteins responsible for resistance to other therapies, by inducing specific degradation of the protein using novel E3s. This groundbreaking strategy has the potential to offer meaningful benefits to hundreds of thousands of patients suffering from serious conditions, as diverse as cancer and Alzheimer’s disease. Through ongoing collaborations with world-leading academic experts in the field, and in partnership with seasoned drug development and commercialization experts at the parent company, BeyondSpring, Seed Therapeutics is establishing a growing pipeline of novel drug candidates on a path to clinical and commercial success. To learn more about Seed Therapeutics, please visit us at seedtherapeutics.com.

About BeyondSpring

Headquartered in New York, BeyondSpring is a global, clinical-stage biopharmaceutical company focused on developing innovative immuno-oncology cancer therapies to improve clinical outcomes for patients with high unmet medical needs. BeyondSpring’s first-in-class lead immune asset, Plinabulin, is a potent antigen-presenting cell (APC) inducer. It is currently in two Phase 3 clinical trials for two severely unmet medical needs indications: one is for the prevention of chemotherapy-induced neutropenia (CIN), the most frequent cause for a chemotherapy regimen dose’s decrease, delay, downgrade or discontinuation, which can lead to suboptimal clinical outcomes. The second is for non-small cell lung cancer (NSCLC) treatment in EGFR wild-type patients. As a “pipeline drug,” Plinabulin is in various I/O combination studies to boost PD-1 / PD-L1 antibody anti-cancer effects. In addition to Plinabulin, BeyondSpring’s extensive pipeline includes three pre-clinical immuno-oncology assets.

Cautionary Note Regarding Forward-Looking Statements

This press release includes forward-looking statements that are not historical facts. Words such as “will,” “expect,” “anticipate,” “plan,” “believe,” “design,” “may,” “future,” “estimate,” “predict,” “objective,” “goal,” or variations thereof and variations of such words and similar expressions are intended to identify such forward-looking statements. Forward-looking statements are based on BeyondSpring’s current knowledge and its present beliefs and expectations regarding possible future events and are subject to risks, uncertainties and assumptions. Actual results and the timing of events could differ materially from those anticipated in these forward-looking statements as a result of several factors including, but not limited to, difficulties raising the anticipated amount needed to finance the Company’s future operations on terms acceptable to the Company, if at all, unexpected results of clinical trials, delays or denial in regulatory approval process, results that do not meet our expectations regarding the potential safety, the ultimate efficacy or clinical utility of our product candidates, increased competition in the market, and other risks described in BeyondSpring’s most recent Form 20-F on file with the U.S. Securities and Exchange Commission. All forward-looking statements made herein speak only as of the date of this release and BeyondSpring undertakes no obligation to update publicly such forward-looking statements to reflect subsequent events or circumstances, except as otherwise required by law.

Media Contacts

Caitlin Kasunich / Raquel Cona
KCSA Strategic Communications
212.896.1241 / 212.896.1276
[email protected] / [email protected]

Waterloo, Canada, Nov. 13, 2020 (GLOBE NEWSWIRE) — The Centre for International Governance Innovation (CIGI) and Gateway House: Indian Council on Global Relations will welcome Canada and India’s top diplomats on Tuesday to discuss the growing strategic partnership between Canada and India in the era of COVID-19.

This is the third Canada-India Track 1.5 Dialogue on Innovation, Growth and Prosperity meeting. The partnership began in 2018 with the support of Prime Minister Justin Trudeau and Prime Minister Narendra Modi as an initiative to explore areas for closer cooperation between the two countries. Track 1.5 dialogues are unofficial conversations between countries that include a mix of government officials and non-governmental experts. 

Media are invited to observe the opening discussion of the meeting, featuring Canada’s Minister of Foreign Affairs, The Honourable François-Philippe Champagne, and India’s Minister of External Affairs, The Honourable Subrahmanyam Jaishankar. Please note that no questions will be taken from the audience.

WHO:

• The Honourable François-Philippe Champagne, Canada’s Minister of Foreign Affairs
• The Honourable Subrahmanyam Jaishankar, India’s Minister of External Affairs
• Rohinton P. Medhora, President, CIGI
• Manjeet Kripalani, Executive Director, Gateway House

WHEN:

Tuesday, November 17^th from 8:30 AM – 9:30 AM EST

WHERE:

A WebEx link to the virtual meeting will be provided upon registration. 

To register, please contact Rebecca MacIntyre or for more information, visit www.cigionline.org/Canada-India.

– 30 –

Attachments

• GH_Logo-Regular-Stacked
• 2018-10-30 – Can-India Ottawa Meetings – AM Sessions-19 copy

Rebecca MacIntyre
Centre for International Governance Innovation
6478616800
[email protected]

NEW HAVEN, Conn., Nov. 13, 2020 (GLOBE NEWSWIRE) — BioXcel Therapeutics, Inc. (“BTI” or the “Company”) (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, today announced that Dr. Vimal Mehta, Founder and Chief Executive Officer of BTI, will participate in a fireside chat at the Jefferies Virtual London Healthcare Conference.

Presentation Details:

Event: Jefferies Virtual London Healthcare Conference
Date: Thursday, November 19, 2020
Time: 7:20 PM GMT/2:20 PM ET

A live webcast of the fireside chat will be accessible through the Investors section of the Company’s website at www.bioxceltherapeutics.com. Following the conference, the webcast will be archived on the Company’s website for at least 30 days.

About BioXcel Therapeutics, Inc.

BioXcel Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on drug development that utilizes artificial intelligence to identify improved therapies in neuroscience and immuno-oncology. BTI’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 indices. BTI’s two most advanced clinical development programs are BXCL501, an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine for the treatment of agitation and opioid withdrawal symptoms, and BXCL701, an investigational, orally administered, systemic innate immunity activator in development for the treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors. For more information, please visit www.bioxceltherapeutics.com.

Contact Information:
BioXcel Therapeutics, Inc.
www.bioxceltherapeutics.com

Investor Relations:
John Graziano
[email protected]
1.646.378.2942

Media:
Julia Deutsch
[email protected]
1.646.378.2967

GREENWICH, Conn., Nov. 13, 2020 (GLOBE NEWSWIRE) —  

XPO Logistics, Inc
. (NYSE: XPO), a leading global provider of supply chain solutions, has been named to the 2021 FreightTech 25 as one of the transportation industry’s most technologically disruptive companies. This is the third consecutive year XPO has been recognized by FreightWaves, a leading source of information about global freight markets.

To arrive at the 25 foremost innovators, an independent panel of judges selected 100 companies from over 500 nominees to comprise the FreightTech 100. A separate panel of industry experts voted to determine the final FreightTech 25.

Mario Harik, chief information officer of XPO Logistics, said, “This unpredictable year has highlighted the critical role of technology in ensuring supply chain continuity. We thank FreightWaves for recognizing our commitment to advance the industry through innovation, with greater visibility and control of operations.”

The company recently announced two new global milestones for its XPO Connect™ digital freight marketplace: more than 65,000 carriers registered on the platform, and over 200,000 downloads of the Drive XPO™ mobile app for truck drivers.

About XPO Logistics

XPO Logistics, Inc. (NYSE: XPO) is a top ten global logistics provider of cutting-edge supply chain solutions to the most successful companies in the world. The company operates as a highly integrated network of people, technology and physical assets in 30 countries, with 1,499 locations and approximately 97,000 employees. XPO uses its network to help more than 50,000 customers manage their goods most efficiently throughout their supply chains. XPO’s corporate headquarters are in Greenwich, Conn., USA, and its European headquarters are in Lyon, France. xpo.com

Media Contact

XPO Logistics, Inc.
Joe Checkler
+1-203-423-2098
[email protected]

• Continued progress across pipeline of gene therapy programs, including recent IND clearance for AXO-AAV-GM2 in Tay-Sachs/Sandhoff diseases
• Completed rebranding to Sio Gene Therapies as part of corporate transformation aligning corporate structure and governance with current and future business activity
• Company had $63.2 million of cash and cash equivalents as of September 30, 2020, with sufficient cash runway into Q4 2021

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Nov. 13, 2020 (GLOBE NEWSWIRE) — Sio Gene Therapies, Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today provided financial results for its second fiscal quarter ended September 30, 2020.

“In recent months, we have taken significant strides forward with our AAV-based gene therapy programs in GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases. We obtained rare pediatric disease designation for both programs, and following IND clearance of AXO-AAV-GM2 from the FDA, we’re delivering on our goal of advancing the first potentially curative gene therapy clinical development programs for both GM1 and GM2 gangliosidosis,” said Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies. “We also advanced AXO-Lenti-PD in the SUNRISE-PD dose-escalation study and presented detailed patient-level data from the mid-dose cohort at our Parkinson’s disease focused R&D Day last month. Our rebranding as Sio Gene Therapies signifies a new beginning for the company – with a scientific focus, management team, Board of Directors, and portfolio strategy that is wholly committed to developing disease-modifying and curative genetic medicines on behalf of patients in need.”

Key Highlights and Development Updates

AXO-AAV-GM1 gene therapy for GM1 gangliosidosis

• On-track to report 6-month topline data, with a focus on safety and tolerability, from 5 children in the low-dose juvenile cohort (Type II) by year-end 2020.
• The U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for AXO-AAV-GM1 in GM1 gangliosidosis.
• Expect to complete dosing of juvenile (Type II) patients in the high-dose cohort of the ongoing AXO-AAV-GM1 clinical study before year-end 2020.

AXO-AAV-GM2 gene therapy for Tay-Sachs/Sandhoff disease

• FDA cleared Company-sponsored Investigational New Drug (IND) application for AXO-AAV-GM2 in Tay-Sachs and Sandhoff diseases.
• FDA granted Rare Pediatric Disease Designation for AXO-AAV-GM2.

AXO-Lenti-PD gene therapy for Parkinson’s disease (PD)

• Hosted an R&D Day on October 30, 2020, during which individual patient-level 6-month follow up data were presented from the second cohort of the SUNRISE-PD dose escalation trial. In addition, key opinion leaders in Parkinson’s disease clinical research and the Michael J. Fox Foundation discussed the treatment landscape and the potential role of AXO-Lenti-PD gene therapy in the treatment paradigm.
• Reported positive 6-month follow-up data from the second cohort of the SUNRISE-PD Phase 2 trial
  • AXO-Lenti-PD was observed to be well-tolerated with no treatment related serious adverse events at 6 months
  • Greater than 2-hour improvement from baseline in both diary “good ON time” and diary OFF time assessments observed across all four patients in Cohort 2
  • Reported a 21-point mean improvement in UPDRS Part III “OFF” score in the two patients with evaluable data, a 40% improvement from baseline 
  • Totality of individual patient outcomes across cohort demonstrate consistency of treatment benefit
• Based on new information received from our manufacturing partner, Oxford Biomedica, in mid-October regarding delays in CMC data and third-party fill/finish issues, the development of a suspension-based manufacturing process for AXO-Lenti-PD will take longer than expected. As a result, the Company believes that it is unlikely that its planned randomized, sham-controlled trial of AXO-Lenti-PD will enroll patients by the end of calendar year 2021. Manufacturing of several GMP batches is now underway and planned at Oxford Biomedica with a goal of generating material for use in future clinical trials as soon as possible. The Company expects to provide an update in the first quarter of 2021 or as program timelines are clarified.

Corporate Updates

• Continued corporate transformation activities, including:
  • Company name change to Sio Gene Therapies. In connection with the name change, the Company’s ticker on the NASDAQ exchange will change to “SIOX” and will be effective at market open on November 13, 2020
  • Appointment of Kristiina Vuori, M.D., Ph.D, as a new director, establishing a majority independent Board of Directors
  • No longer being a majority-owned and controlled public company
  • Completed redomiciliation to Delaware
• Signed strategic gene therapy development and manufacturing partnership with Viralgen, an AskBio subsidiary, securing access to cGMP capacity and resources to support the development and commercialization of AAV gene therapy programs in GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases.
• Opening of new laboratory space in Research Triangle Park, North Carolina, focused on in-house preclinical and analytical development activities.
• Promoted Parag V. Meswani, Pharm.D. to Chief Commercial Officer to support Axovant’s commercialization efforts across the clinical-stage pipeline.

Fiscal Second Quarter Financial Summary

For the second fiscal quarter ended September 30, 2020, research and development expenses were $5.1 million, a decrease of $1.8 million compared to the prior year quarter, primarily due to (i) lower AXO-Lenti-PD clinical expenses of approximately $0.9 million as the enrollment of Cohort 2 was completed in February 2020, (ii) reduced costs of $0.7 million while awaiting FDA clearance of the IND for the AXO-AAV-GM2 program, and (iii) a $0.5 million reversal of an accrual for manufacturing development services for our AXO-AAV-GM1 and AXO-AAV-GM2 programs under an agreement that was terminated.

General and administrative expenses for the second fiscal quarter ended September 30, 2020 were $4.5 million, a decrease of $0.6 million compared to the prior year quarter, primarily due to reductions in personnel costs (including severance) attributable to reduced headcount.

The net loss for the second fiscal quarter ended September 30, 2020 was $10.0 million, or $0.21 per share, compared to a net loss of $13.9 million, or $0.61 per share, in the prior year quarter.

Fiscal First-Half Financial Summary

For the six months ended September 30, 2020, research and development expenses were $10.3 million, a decrease of $17.7 million compared to the six months ended September 30, 2019. Excluding the net amount of $13.0 million due to Oxford for a development milestone achieved in the prior year period as well as a decrease of $2.1 million of expenses associated with our discontinued legacy AXO-AAV-OPMD program that was terminated in September 2019, research and development expenses decreased by $2.6 million in the current year period. The current period decrease was primarily due to (i) reduced costs of $1.0 million while awaiting FDA clearance of the IND for the AXO-AAV-GM2 program, (ii) a $0.8 million payment in the prior year period to our licensor, University of Massachusetts Medical School, for reaching a manufacturing milestone for the AXO-AAV-GM1 program, and (iii) a $0.5 million reversal of an accrual for manufacturing development services for our AXO-AAV-GM1 and AXO-AAV-GM2 programs under an agreement that was terminated.

General and administrative expenses for the six months ended September 30, 2020 were $9.1 million, a decrease of $2.4 million compared to the six months ended September 30, 2019, primarily related to reductions in (i) personnel costs (including severance) of $1.3 million and stock-based compensation expense of $0.2 million attributable to reduced headcount, and (ii) pharmaceutical market research expenses of $0.6 million.

The net loss for the six months ended September 30, 2020 was $18.6 million, or $0.41 per share, compared to a net loss of $41.9 million, or $1.84 per share, in the six months ended September 30, 2019. Net cash used in operating activities was $25.3 million for the six months ended September 30, 2020.

As of September 30, 2020, we had $63.2 million of cash and cash equivalents. The Company holds no short-term or long-term debt on the balance sheet. We expect the cash and cash equivalents to sustain our operations into the fourth calendar quarter of 2021.

On October 2, we filed a prospectus supplement with the SEC pertaining to a $50 million at-the-market equity financing facility. No sales under the facility occurred prior to our press release on October 6 and no sales have occurred since October 9. Approximately 1.2 million shares for total proceeds of $5.1 million, net of brokerage fees, were sold under the facility during this period.

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson’s disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit www.siogtx.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “will,” “expect,” “believe,” “estimate,” and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Sio’s management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sio’s scientific approach and general development progress; and the availability or commercial potential of Sio’s product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sio’s most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.
LifeSci Communications
(646) 751-4361
[email protected]
[email protected]

Investors and Analysts

David Nassif
Sio Gene Therapies, Inc.
Chief Financial Officer and General Counsel
(646) 677-6770
[email protected]

SIO GENE THERAPIES INC.

Condensed Consolidated Statements of Operations

(Unaudited, in thousands, except share and per share amounts)

	Three Months Ended September 30,								Six Months Ended September 30,
	2020				2019				2020				2019
Operating expenses:
Research and development expenses
(includes stock-based compensation expense of $458 and $409 for the three months ended September 30, 2020 and 2019 and $1,021 and $1,130 for the six months ended September 30, 2020 and 2019, respectively)	$	5,058			$	6,833			$	10,252			$	27,923
General and administrative expenses
(includes stock-based compensation expense of $650 and $482 for the three months ended September 30, 2020 and 2019 and $1,677 and $1,896 for the six months ended September 30, 2020 and 2019, respectively)	4,491				5,051				9,131				11,519
Total operating expenses	9,549				11,884				19,383				39,442
Other (income) expenses:
Interest expense	1				1,313				797				2,871
Other expense (income)	580				560				(1,486		)		(537		)
Loss before income tax (benefit) expense	(10,130		)		(13,757		)		(18,694		)		(41,776		)
Income tax (benefit) expense	(146		)		127				(116		)		165
Net loss	$	(9,984	)		$	(13,884	)		$	(18,578	)		$	(41,941	)
Net loss per common share — basic and diluted	$	(0.21	)		$	(0.61	)		$	(0.41	)		$	(1.84	)
Weighted-average common shares outstanding — basic and diluted	46,731,666				22,783,182				45,018,855				22,781,657

SIO GENE THERAPIES INC.

Condensed Consolidated Balance Sheets

(Unaudited, in thousands, except share and per share amounts)

	September 30, 2020				March 31, 2020
Assets
Current assets:
Cash and cash equivalents	$	63,171			$	80,752
Prepaid expenses and other current assets	5,406				2,971
Income tax receivable	1,747				1,707
Total current assets	70,324				85,430
Long-term investment	8,055				5,871
Other non-current assets	169				46
Operating lease right-of-use assets	663				1,532
Property and equipment, net	560				801
Total assets	$	79,771			$	93,680
Liabilities and Shareholders’ Equity
Current liabilities:
Accounts payable	$	2,172			$	4,412
Accrued expenses	7,837				11,319
Current portion of operating lease liabilities	34				889
Current portion of long-term debt	—				15,423
Total current liabilities	10,043				32,043
Operating lease liabilities, net of current portion	55				79
Total liabilities	10,098				32,122
Stockholders’ equity:
Common stock, par value $0.00001 per share, 1,000,000,000 shares authorized, 47,249,729 and 39,526,299 issued and outstanding at September 30, 2020 and March 31, 2020, respectively	—				—
Additional paid-in capital	846,558				820,257
Accumulated deficit	(777,222		)		(758,644		)
Accumulated other comprehensive loss	337				(55		)
Total stockholders’ equity	69,673				61,558
Total liabilities and stockholders’ equity	$	79,771			$	93,680

BOSTON, Nov. 13, 2020 (GLOBE NEWSWIRE) — DraftKings Inc. (Nasdaq: DKNG) (“DraftKings” or the “Company”) today reported its financial results for the third quarter of 2020. For the three months ended September 30, 2020, DraftKings reported revenue of $133 million, an increase of 98% compared to $67 million during the same period in 2019. After giving pro forma effect to the business combination with SBTech (Global) Limited and Diamond Eagle Acquisition Corp., as if it had occurred on January 1, 2019, revenue grew 42% compared to the three months ended September 30, 2019.

“The resumption of major sports such as the NBA, MLB and the NHL in the third quarter, as well as the start of the NFL season, generated tremendous customer engagement,” said Jason Robins, DraftKings’ co-founder, CEO and Chairman of the Board. “In addition to our year-over-year pro forma revenue growth of 42%, DraftKings recorded an increase in monthly unique payers of 64% to over 1 million, demonstrating the effectiveness of our data-driven sales and marketing approach. Our product offerings and scalable platform provide a distinctive and personalized experience for customers across the ten states where we operate mobile sports betting today, and we look forward to entering additional jurisdictions at the earliest opportunity.”

Return of Major Sports and Unique Sports Calendar Drive Growth and Attractive Customer Acquisition Opportunities

• Monthly Unique Payers (“MUPs”) for our B2C segment increased by 64% compared to the third quarter of 2019. On average, more than a million monthly unique paying customers engaged with DraftKings each month during the third quarter. This improvement reflected continued growth of our core DFS product spurred by on-going product innovation, strong engagement from existing Sportsbook and iGaming players, and expansion of our player base in several new states.
• Average Revenue Per Monthly Unique Payer (“ARPMUP”) for our B2C segment was $34 due to limited sports activity in July and atypical hold rates from NFL wagering through the third week of the season, which was partially offset by increased engagement with our iGaming product offering.
• GAAP sales and marketing expense increased to $203 million in the three months ended September 30, 2020. The increase over the third quarter of 2019 was primarily due to being live in seven more states, including Illinois, for Week 1 of the NFL season this year versus Week 1 of 2019. DraftKings experienced strong returns on its marketing spend due in part to pent-up demand, the unique sports calendar, and the stay-at-home nature of the COVID-19 pandemic.

Increasing 2020 Revenue Guidance and Introducing 2021 Revenue Guidance

• DraftKings is raising its fiscal year 2020 pro forma revenue guidance from a range of $500 to $540 million to a range of $540 to $560 million, which equates to year-over-year pro forma revenue growth of 25% to 30% in 2020, despite COVID-19’s impact on the major sports calendar. This guidance assumes that all announced sports calendars are maintained through the end of the year and that we continue to operate in states in which we are live today.
• DraftKings is also introducing 2021 revenue guidance of $750 million to $850 million, which equates to 45% year-over-year growth based on the mid-points of the Company’s 2020 pro forma revenue guidance range and the Company’s 2021 revenue guidance range. This range is based on the same assumptions used for the Company’s 2020 guidance, in particular that all professional and college sports calendars that have been announced come to fruition, including the commencement of their 2020 to 2021 seasons, and that we continue to operate in states in which we are live today.
• Detailed financial data and other information is available in DraftKings’ Quarterly Report on Form 10-Q, being filed today with the Securities and Exchange Commission, as well as in a slide presentation that can be accessed through the “Investors” section of the Company’s website at investors.draftkings.com.

DraftKings Grows Its Nation-Leading Mobile Sports Betting Footprint

During the third quarter, DraftKings launched mobile sports betting in Illinois and iGaming in West Virginia.

• As a result of Illinois Governor J.B. Pritzker’s suspension of the in-person registration requirement, DraftKings was able to effectively acquire new Illinois customers to the platform as well as cross-sell from its existing product offerings. Illinois has quickly become the Company’s fastest-growing state as well as one of its largest states in terms of handle.
• Following its successful launch in Tennessee, DraftKings is now live with mobile sports betting in 10 states, which is more than any other company in the industry. These 10 states together represent 20% of the U.S. population, a position that DraftKings has achieved just two and a half years after the Supreme Court struck down the Professional and Amateur Sports Protection Act of 1992.
• The Company continues to work with state officials on regulations and licensing in Michigan for sports betting and iGaming and in Virginia for sports betting and expects to launch in these states at the earliest practicable opportunity. Michigan and Virginia together account for 6% of the U.S. population and have already legalized these offerings.
• Maryland (with 67% voter approval), South Dakota (with 58% voter approval) and 55 of 64 parishes in Louisiana (representing approximately 97% of the state’s population) recently passed referendums in favor of sports betting. These states together account for 3.5% of the U.S. population.
• In addition, Ontario’s government recently presented its annual budget, which included language that would modify the long-standing statutory internet gaming framework in order to allow private operators to join the province in offering sports betting and iGaming products. Ontario’s population would make it the fifth largest U.S. state by population. DraftKings has offered its DFS product in Canada since 2012.

Commercial and Strategic Agreements

DraftKings announced several advantageous commercial and strategic agreements in the quarter that are expected to provide the Company with access to unique and valuable content, intellectual property and marketing assets, including:

• an expansion of a multi-year content and marketing relationship with the PGA TOUR to become the first Official Betting Operator of the PGA TOUR.
• an exclusive multi-year extension to remain the Official Daily Fantasy Sports Partner of Major League Baseball.
• an exclusive multi-year deal with the Chicago Cubs, making DraftKings the Official Sports Betting Operator and Official Daily Fantasy Partner of the Cubs, including a plan to pursue a first-of-its-kind sportsbook at Wrigley Field.
• a multi-year agreement with ESPN to collaborate in a variety of areas including becoming a co-exclusive sportsbook link-out provider and exclusive daily fantasy sports link-out provider.
• an exclusive, multi-year agreement with the New York Giants, making DraftKings the official sports betting, gaming & casino and daily fantasy partner of the team.
• an agreement with the Colorado Rockies to be named both the franchise’s Official Daily Sports Partner and the franchise’s first Official Sports Betting Partner.

Product and Technology

DraftKings unveiled several technology enhancements and new product features during the third quarter:

• launched standalone casino app in Pennsylvania and West Virginia, offering users a more holistic product suite and dynamic gaming experience in a DraftKings-created casino app.
• launched Best Ball, a new season-long DFS game variant, featuring snake drafts. The streamlined draft format enables the Company to engage with customers long before the traditional pre-NFL period. DraftKings will launch Best Ball versions for fantasy basketball and hockey in the near future.
• introduced several new DraftKings-created games for online casino, including new versions of blackjack, roulette and baccarat.
• made various improvements to the mobile and online Sportsbook to further differentiate the customer experience including the addition of dark mode and shifting of main navigation (online) to the left rail.

Environmental-Social-Governance Initiatives

DraftKings had several notable ESG-related highlights during the quarter, including:

• strengthened our corporate governance foundation by appointing two new Board members, Jocelyn Moore and Valerie Mosley. The two directors each bring unique skills, experiences and ideas, and will play an important role in shaping the future of DraftKings and helping us achieve our long-term goals.
• welcomed Michael Jordan as a special advisor to the Board. Jordan will provide strategic and creative input to the Board on brand strategy, product development, inclusion, equity and belonging, marketing activities and other key initiatives.
• donated all of the Company’s daily fantasy sports revenue from the NBA and WNBA games on August 28 and 29, totaling approximately $340,000, to organizations promoting racial justice.
• announced Election Day as an official DraftKings company holiday; employees at all DraftKings offices worldwide will now receive their respective election day as a holiday.
• raised $20,000 through charity DFS contests in support of the Company’s Tech for Heroes initiative, which provides recent and returning veterans and their spouses with free, high-tech skills training in areas like front end web development and cybersecurity.

Webcast and Conference Call Details

DraftKings will host a conference call and audio webcast today at 8:30 a.m. EDT, during which management will discuss the Company’s third quarter results and provide commentary on business performance. A question and answer session will follow the prepared remarks.

The conference call may be accessed by dialing (833) 644-0686 for domestic callers or (918) 922-6762 for international callers. Once connected with the operator, please provide the conference ID of 2644858.

A live audio webcast of the earnings conference call may be accessed on the Company’s website at investors.draftkings.com, along with a copy of this press release, the Company’s Form 10-Q filing, and a slide presentation. The audio webcast and accompanying presentation will be available on the Company’s investor relations website until 11:59 p.m. EDT on December 14, 2020.

About DraftKings

DraftKings Inc. (Nasdaq: DKNG) is a digital sports entertainment and gaming company created to fuel the competitive spirits of sports fans with products that range across daily fantasy, regulated gaming and digital media. Headquartered in Boston, and launched in 2012 by Jason Robins, Matt Kalish and Paul Liberman, DraftKings is the only U.S.-based vertically integrated sports betting operator. DraftKings is a multi-channel provider of sports betting and gaming technologies, powering sports and gaming entertainment for 50+ operators across more than 15 regulated U.S. and global markets, including Arkansas and Oregon in the U.S. DraftKings’ Sportsbook offers mobile and retail betting for major U.S. and international sports and operates in the United States pursuant to regulations in Colorado, Illinois, Indiana, Iowa, Mississippi, New Hampshire, New Jersey, New York, Pennsylvania, Tennessee and West Virginia. DraftKings’ daily fantasy sports product is available in 8 countries internationally with 15 distinct sports categories. DraftKings is the official daily fantasy partner of the NFL, MLB and the PGA TOUR as well as an authorized gaming operator of the NBA and MLB and an official betting operator of the PGA TOUR.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act, about us and our industry that involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this press release, including statements regarding guidance, our future results of operations or financial condition, business strategy and plans, user growth and engagement, product initiatives, and objectives of management for future operations, and the impact of COVID-19 on our business and the economy as a whole, are forward-looking statements. In some cases, you can identify forward-looking statements because they contain words such as “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “going to,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “propose,” “should,” “target,” “will,” or “would” or the negative of these words or other similar terms or expressions. We caution you that the foregoing may not include all of the forward-looking statements made in this press release.

You should not rely on forward-looking statements as predictions of future events. We have based the forward-looking statements contained in this press release primarily on our current expectations and projections about future events and trends, including the ongoing COVID-19 pandemic that we believe may affect our business, financial condition, results of operations, and prospects. These forward-looking statements are not guarantees of future performance, conditions or results, and involve a number of known and unknown risks, uncertainties, assumptions and other important factors, many of which are outside DraftKings’ control, that could cause actual results or outcomes to differ materially from those discussed in the forward-looking statements. Important factors, among others, that may affect actual results or outcomes include DraftKings’ ability to manage growth; DraftKings’ ability to execute its business plan and meet its projections; potential litigation involving DraftKings; changes in applicable laws or regulations, particularly with respect to gaming; general economic and market conditions impacting demand for DraftKings’ products and services, and in particular economic and market conditions in the media / entertainment / gaming / software industry in the markets in which DraftKings’ operates; the potential adverse effects of the ongoing global coronavirus (COVID-19) pandemic on capital markets, general economic conditions, unemployment and DraftKings’ liquidity, operations and personnel, as well as risks, uncertainties, and other factors described in “Risk Factors” in our filings with the SEC, which are available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. In addition, any forward-looking statements contained in this press release are based on assumptions that we believe to be reasonable as of this date. We undertake no obligation to update any forward-looking statements to reflect events or circumstances after the date of this press release or to reflect new information or the occurrence of unanticipated events, including future developments related to the COVID-19 pandemic, except as required by law.

Contacts

Media:
[email protected]
@DraftKingsNews 

Investors:
[email protected]

WATERTOWN, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) — C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a biopharmaceutical company pioneering a new class of small-molecule drugs that selectively destroys disease-causing proteins through degradation, today announced that the Company will present at the Jefferies Virtual London Healthcare Conference on Thursday, November 19, 2020 at 8:30 a.m. ET.

A live webcast of the presentation can be accessed under “Events & Presentations” in the Investors section of the company’s website at www.c4therapeutics.com. A replay of the webcast will be archived on the C4T website for at least two weeks following the presentation.

About C4 Therapeutics

C4 Therapeutics (C4T) is a biopharmaceutical company focused on harnessing the body’s natural regulation of protein levels to develop novel therapeutic candidates to target and destroy disease-causing proteins for the treatment of cancer, neurodegenerative conditions and other diseases. This targeted protein degradation approach offers advantages over traditional therapies, including the potential to treat a wider range of diseases, reduce drug resistance, achieve higher potency, and decrease side effects through greater selectivity. To learn more about C4 Therapeutics, visit www.C4Therapeutics.com.

Investor & Media Contact:
Kendra Adams
SVP, Communications & Investor Relations
[email protected]

TORONTO and HOUSTON, Nov. 13, 2020 (GLOBE NEWSWIRE) — Medicenna Therapeutics Corp. (“Medicenna” or “the Company”) (NASDAQ: MDNA; TSX: MDNA), a clinical stage immuno-oncology company, today announced its financial results and operational highlights for the quarter ended September 30, 2020. All dollar amounts are expressed in Canadian currency unless otherwise noted.

“Over the last several months, we have achieved key clinical, regulatory, and corporate milestones that have left us well positioned for continued growth,” said Dr. Fahar Merchant, Chairman, President and Chief Executive Officer of Medicenna. “We believe that the clinical data presented at the ENA Meeting strongly supports MDNA55’s ability to improve long-term survival and tumor control in recurrent glioblastoma (“rGBM”), a common and uniformly fatal from of brain cancer. This data is complemented by preclinical results from our IL-2 and IL-13 Superkine platform programs that further demonstrates the potential of MDNA11 and highlights the platform’s ability to generate cytokine-based treatments that may have the potential to overcome the shortcomings of currently available immunotherapies.”

Dr. Merchant continued, “Moving forward, we aim to build on these achievements as we work to advance and expand our clinical pipeline. We continue to assess potential partnership strategies to facilitate the progression of our MDNA55 program, and have been bolstered by our positive data and the FDA’s pioneering recommendation to conduct a hybrid registration trial with a comparator arm that utilizes both traditional and matched external controls. Meaningful progress is also being made toward the advancement of MDNA11 to the clinic, as we recently completed a Scientific Advice Meeting with UK’s MHRA and are on track to submit the IMPD, for a Phase 1/2 clinical study, in the middle of the next calendar year. We believe that the continued progression of these programs, together with our recent Nasdaq listing, will enable Medicenna to deliver short- and long-term value to its stakeholders.”

Program highlights for the quarter ended September 30, 2020, along with recent developments, include:

MDNA55: Recurrent Glioblastoma Program
:

• On October 15, 2020 Medicenna provided an update on the clinical development of MDNA55, an interleukin-4 (IL-4)-guided toxin targeting recurrent glioblastoma (rGBM), the most common and uniformly fatal form of brain cancer. The FDA agreed that we could conduct an innovative open-label hybrid Phase 3 trial that allows use of a substantial number of subjects (two-thirds) from a matched external control arm to support regulatory approval of MDNA55 for rGBM. The FDA also expressed their willingness to consider interim analysis of the trial if certain criteria are met. Unlike conventional randomized control trials, the hybrid trial design will reduce the overall number of subjects needed in the study to achieve the primary endpoint as well as reduce the cost and timelines associated with completing the trial.
• On October 26, 2020, Medicenna announced a Late Breaking Abstract poster presentation at the 32nd ENA Symposium on Molecular Targets and Cancer Therapeutics. Amongst an all-comer population, a single treatment with MDNA55 resulted in at least 100% increase in both 12-month progression free survival (PFS-12 of 27% versus 2 to 10%) and 2-year survival (OS-24 of 20% vs 5 to10%) when compared to what is achieved with approved therapies. In a subset of all-comer patients treated with transient low dose bevacizumab, to reduce steroid use, median survival (mOS) was 21.8 months and OS-24 was 44%.

MDNA11: IL-2
Superkine
Program

• On November 4, 2020 Medicenna held a Scientific Advice Meeting for MDNA11 (similar to a pre-IND meeting) with the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA). It confirmed that our plans for CMC, pre-clinical and Phase 1/2 clinical trial were appropriate for submission of an Investigational Medical Product Dossier (IMPD) in mid-calendar 2021 in order to commence first in human studies with MDNA11 in the UK.
• On October 26, 2020, Medicenna announced a poster presentation at the 32nd ENA Symposium on Molecular Targets and Cancer Therapeutics. The preclinical data, featured results with MDNA11 as well as data related to a long acting bispecific IL-2/IL-13 Superkine designed to simultaneously activate cancer killing immune cells while reversing anti-inflammatory tumor micro-environment (TME). The results substantiated the potent therapeutic efficacy of MDNA11 as a monotherapy agent in multiple tumor models. Medicenna’s novel bispecific IL-2/IL-13 Superkines demonstrated the potential of the platform to address a critical unmet need by effectively targeting immunologically “cold” tumors that are often resistant to immunotherapeutic agents.

Operational Highlights

• On August 24, 2020 Medicenna’s common shares began trading on The Nasdaq Capital Market (“Nasdaq”). Medicenna now trades on both the Nasdaq and the Toronto Stock Exchange under the symbol “MDNA”.
• On September 30, 2020, Dr. Jack Geltosky, an experienced pharmaceutical licensing executive with a strong research and development background, was elected to Medicenna’s Board of Directors.

Upcoming Milestones

Medicenna will focus on achieving the following milestones in the upcoming quarters:

• Submit an IMPD to MHRA in support of initiating a Phase 1/2 study for MDNA11 in mid-calendar 2021.
• Report results from the safety portion of a Phase 1/2 MDNA11 monotherapy study late in the second half of calendar 2021.
• Execute a partnership for a registration trial and commercialization of MDNA55 for recurrent GBM.
• Declare a lead candidate for its bispecific Superkine program in calendar 2021.

Financial Results

Net loss for the quarter ended September 30, 2020 was $3.8 million, or $0.08 per share, compared to a loss of $1.9 million, or $0.07 per share, for the quarter ended September 30, 2019. The increase in net loss for the quarter ended September 30, 2020 compared with the quarter ended September 30, 2019 was primarily a result of no reimbursement under the CPRIT grant in the current year period, increased research and development expenditures related to the MDNA11 program as well as costs associated with the Nasdaq listing.

Research and development expenses of $2.2 million were incurred during the quarter ended September 30, 2020, compared with $1.2 million incurred in the quarter ended September 30, 2019. The increase in expenses in the current quarter is primarily attributable to no reimbursement of expenditures under the CPRIT grant in the current year period and increased manufacturing and development expenditures related to the MDNA11 program.

General and administration expenses of $1.7 million were incurred during the quarter ended September 30, 2020, compared with $0.6 million during the quarter ended September 30, 2019.  This increase in expenditures is primarily attributed to public company expenses in the current periods due to activities associated with our Nasdaq listing and related directors and officers liability insurance premiums.

Medicenna had cash, cash equivalents and marketable securities of $34.2 million as at September 30, 2020. These funds provide the Company with sufficient capital to mid-2022 based on its current plans and projections.

Medicenna’s condensed consolidated interim financial statements for the quarter ended September 30, 2020 and the related management’s discussion and analysis (MD&A) will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov

About Medicenna

Medicenna is a clinical stage immunotherapy company focused on the development of novel, highly selective versions of IL-2, IL-4 and IL-13 Superkines and first in class Empowered Superkines for the treatment of a broad range of cancers. Medicenna’s long-acting IL2 Superkine asset, MDNA11, is a next-generation IL-2 with superior CD122 binding without CD25 affinity and therefore preferentially stimulating cancer killing effector T cells and NK cells when compared to competing IL-2 programs. It is anticipated that MDNA11 will be ready for the clinic in 2021. Medicenna’s lead IL4 Empowered Superkine, MDNA55, has completed a Phase 2b clinical trial for rGBM, the most common and uniformly fatal form of brain cancer. MDNA55 has been studied in five clinical trials involving 132 subjects, including 112 adults with rGBM. MDNA55 has obtained Fast-Track and Orphan Drug status from the FDA and FDA/EMA, respectively. For more information, please visit www.medicenna.com.

Forward-Looking Statement

This news release contains forward-looking statements under applicable securities laws and relate to the future operations of the Company and other statements that are not historical facts. Forward-looking statements are often identified by terms such as “will”, “may”, “should”, “anticipate”, “expects”, “believes” and similar expressions. All statements other than statements of historical fact, included in this release, including MDNA55’s ability to ability to improve long-term survival and tumor control in rGBM, the expansion of our clinical pipeline, the anticipated timing as to when MDNA11 will be ready for the clinic and when clinical trial results will be available, the Phase 3 trial for MDNA55 and a potential interim analysis by the FDA, the submission of an IMPD in order to commence human studies with MDNA11, the timing on declaring a lead candidate from the bi-specifics platform, that we are well positioned for continued growth, that our MDNA11 Superkine platform has the ability to generate cytokine-based treatments that overcome the shortcomings of currently available immunotherapies, partnership plans for MDNA55, that Medicenna is enabled to deliver short- and long-term value to its stakeholders, timing to report results from the safety portion of the MDNA11 Phase 1/2, the period of time that the Company’s cash on hand will fund its current plans and operations and the future plans and objectives of the Company, are forward-looking statements that involve risks and uncertainties. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those anticipated in such statements. Important factors that could cause actual results to differ materially from the Company’s expectations include the risks detailed in the annual information form of the Company dated May 14, 2020 and in other filings made by the Company with the applicable securities regulators from time to time in Canada and the United States.

The reader is cautioned that assumptions used in the preparation of any forward-looking information may prove to be incorrect and that study results could change over time as the study is continuing to follow up all subjects and new data are continually being received which could materially change study results. Events or circumstances may cause actual results to differ materially from those predicted, as a result of numerous known and unknown risks, uncertainties, and other factors, many of which are beyond the control of the Company. The reader is cautioned not to place undue reliance on any forward-looking information. Such information, although considered reasonable by management at the time of preparation, may prove to be incorrect and actual results may differ materially from those anticipated. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company will update or revise publicly any of the included forward-looking statements only as expressly required by Canadian and United States securities law.

 

Further Information

For further information about the Company please contact:

Elizabeth Williams, Chief Financial Officer, 416-648-5555, [email protected]

Investor Contact

For more investor information, please contact:

Dan Ferry, Managing Director, LifeSci Advisors, 617-430-7576, [email protected]