On May 16, 2016 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company with three clinical-stage development programs focused on the emerging field of regenerative medicine, reported financial results for the first quarter ended March 31, 2016 (Press release, BioTime, MAY 16, 2016, View Source;p=RssLanding&cat=news&id=2168708 [SID:1234512445]). The company also announced plans to expand the AST-OPC1 Phase 1/2a trial in spinal cord injury patients following recent clearance for the expansion by the U.S. Food and Drug Administration (FDA) based on the favorable safety profile observed so far with AST-OPC1 in the current study.
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"Recent FDA clearance for expanding the AST-OPC1 clinical study in spinal cord injury patients and the recent capital raise needed to fund this expansion are key developments for Asterias," said Steve Cartt, President and Chief Executive Officer. "This important trial expansion should increase the statistical confidence of the safety and efficacy readouts, expand the range of spinal cord injury patients being evaluated, and better position the product for a potential accelerated regulatory pathway should we observe positive efficacy signals and continued safety in the study."
Mr. Cartt continued, "In addition, the capital we recently raised, despite the very challenging capital markets, significantly strengthened our cash position. Combined with continued non-dilutive funding from leading scientific institutions, this will allow Asterias to immediately progress our three clinical-stage programs toward important milestones targeted for late 2016 and into 2017."
As of May 15, 2016, the company’s cash, cash equivalents and available-for-sale securities totaled over $33 million. This includes a recent $2.5 million grant payment from the California Institute of Regenerative Medicine (CIRM) related to progress in the ongoing AST-OPC1 study.
Corporate Highlights
On May 13, 2016, Asterias raised approximately $16.2 million in net proceeds from a public offering of shares of its common stock and warrants. Asterias has granted the underwriters a 30-day option to purchase up to an additional 772,059 shares of common stock and/or additional warrants to purchase up to 386,029 shares of common stock to cover over-allotments, if any. The underwriters exercised their over-allotment option to purchase 386,029 additional warrants. If the over-allotment option to purchase the additional shares is exercised in full, additional net proceeds from the offering to Asterias will be approximately $2.4 million.
In April, Howard I. Scher M.D., one of the world’s leading oncology experts, was appointed to the Board of Directors of Asterias. Dr. Scher is internationally recognized for his expertise in clinical oncology. He has extensive experience in the design of clinical trials for novel anti-cancer agents, including monoclonal antibodies and other biologic therapies, cytotoxics, and drugs that target specific signaling pathways.
Research and Development Highlights
AST-OPC1 (oligodendrocyte progenitor cells)
Patient recruitment is ongoing for the second cohort in a Phase 1/2a clinical trial of AST-OPC1 in complete cervical spinal cord injury, in which five patients will be administered a dose of 10 million AST-OPC1 cells. This cohort is the first of two dose cohorts receiving doses in the predicted efficacious range based on preclinical studies. Asterias has been granted FDA clearance to expand patient enrollment in the Phase 1/2a clinical trial from 13 patients to up to 35 patients, based on the continued favorable safety profile observed in the ongoing clinical study. The company believes that the trial expansion should increase the statistical confidence of the safety and efficacy readouts, expand the range of spinal cord injury patients being evaluated, and better position the product for a potential accelerated regulatory pathway should the company observe positive efficacy signals and continued safety in the study. This trial is being funded in part by a $14.3 million grant from CIRM.
In February, the FDA granted Orphan Drug Designation for AST-OPC1 for the treatment of acute spinal cord injury. Orphan Drug Designation qualifies the sponsor of the drug certain benefits and incentives, including seven years of marketing exclusivity following regulatory approval, and financial incentives such as potential tax credits for certain activities and waiver of certain administrative fees.
In April, Asterias presented an overview of the AST-OPC1 therapeutic development program in spinal cord injury at the American Spinal Injury Association Annual Meeting and at the Stem Cell Summit 2016 meeting.
AST-VAC1 (antigen-presenting autologous dendritic cells)
In February, Asterias successfully completed the End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for AST-VAC1, the company’s investigational cancer immunotherapy and lead clinical program targeting maintenance of relapse-free-survival in acute myeloid leukemia (AML) patients. The company currently is evaluating plans for progressing the AST-VAC1 program towards a pivotal Phase 3 trial which would begin in late 2017.
Clinical data from the Phase 2 trial of AST-VAC1 in AML was presented during an oral session at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 19th Annual Meeting on May 5, 2016. The data was first presented at the 2015 annual meeting of the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper).
AST-VAC2 (antigen-presenting allogeneic dendritic cells)
AST-VAC2 is Asterias’ innovative allogeneic (non-patient-specific) immunotherapy product that contains mature dendritic cells derived from pluripotent stem cells. The company’s research partner, Cancer Research UK (CRUK), will execute the first clinical trial of AST-VAC2. As part of this partnership, CRUK will perform cGMP manufacture of AST-VAC2 at its Biotherapeutics Development Unit, and will submit a Clinical Trial Authorisation application to the UK regulatory authorities for a Phase 1/2a clinical trial in non-small cell lung cancer. The trial will be sponsored, managed and funded by the CRUK Centre for Drug Development. Asterias anticipates receiving approval from UK regulatory authorities for clinical development of AST-VAC2 by the end of 2016 and beginning enrollment for the Phase 1/2a clinical trial in the first quarter of 2017. The trial will examine the safety, immunogenicity and activity of AST-VAC2 in non-small cell lung cancer patients and could potentially position the product for development in numerous cancer indications.
First Quarter Financial Update
Total revenues were $1.6 million for the first quarter. Revenues are comprised of grant income as well as royalty revenues on product sales by licensees. Research and development expenses were $6.3 million for the first quarter. General and administrative expenses were $6.3 million for the first quarter. Net loss was $10.3 million for the three months ended March 31, 2016, or $0.27 per share, including a deferred income tax benefit of $902,000. For the first quarter, net cash used in operating activities was $4.2 million.