NEW YORK, NY and CLEVELAND, OH -- (Marketwired) -- 03/08/16 -- Abeona Therapeutics, Inc. (NASDAQ: ABEO), a biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, today announced summary financial results for the fourth quarter and full fiscal year ended December 31, 2015. The Company will provide a business update for investors and other stakeholders on a conference call, Wednesday, March 9th, at 4:45 pm (Eastern). Tim Miller, Ph.D., President and CEO and Jeffrey Davis, Chief Operating Officer, together with other executives, will conduct the call. Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international). The call will consist of an overview of the Company's 4Q15 financials, and a discussion of business highlights.
"The past year has led to significant advancements in our goal of building a leadership position in the field of gene therapy and plasma protein therapies towards transforming the lives of patients with rare diseases," stated Steven H. Rouhandeh, Executive Chairman. "In 2015, we expanded our pipeline with two clinical stage AAV gene therapies for Sanfilippo syndrome types A and B, added a third AAV gene therapy product in Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) (also known as juvenile Batten disease), signed a license to an innovative CRISPR-Cas9 gene editing platform in rare blood disorders, with an initial focus in Fanconi anemia, strengthened our team, and added substantial financial resources to our balance sheet. In 2016, our priorities include driving our AAV gene therapy and alpha-1 protease inhibitor programs into the clinic, and advancing our gene editing programs including defining of regulatory pathways to bring our CRISPR product candidates to patients."
Tim Miller, Ph.D., President and CEO, stated, "2016 will be an exciting, transformative year for Abeona Therapeutics as we position ourselves to enter multiple human clinical trials with our pipeline of innovative product candidates. As recently announced, the FDA allowance of the IND for the Phase 1/2 clinical study of ABO-102 for patients with Sanfilippo syndrome type A (MPS IIIA) moves our programs into the clinic here in the US, and we look forward to working with our collaborators to expand this program into Europe and Australia later this year. We believe that our gene therapy programs in Sanfilippo syndrome type B (ABO-101) and Juvenile Neuronal Ceroid Lipofuscinosis (ABO-201) will follow shortly. Lastly, we would like to thank our dedicated researchers and clinical collaborators, as well as the many dedicated patient foundations, for their tireless efforts and commitment to advancing new treatment options for these devastating unmet medical needs."
Recent Abeona Highlights
- Sanfilippo syndrome gene therapy programs: On February 29, 2016, Abeona announced the FDA allowance of an Investigational New Drug (IND) for systemic AAV Phase 1/2 clinical study with ABO-102 gene therapy for patients with Sanfilippo syndrome type A (MPS IIIA). On January 11, 2016, we announced that initial regulatory approvals from European bodies -- the Genetically Modified Organism (GMO) Voluntary Release regulatory filings, and the ethical committee regulatory filings -- for both the ABO-101 and ABO-102 programs in Spain. Abeona plans to commence both programs for the upcoming human clinical trials to be conducted at Cruces University Hospital in Bilbao, Spain. Both the ABO-101 and ABO-102 programs have received Orphan Drug and Pediatric Rare Disease designations from the FDA.
SDF-Alpha plasma protein program: Abeona has completed optimization of the downstream chromatography steps for our SDF-Alpha™ (alpha-1 protease inhibitor) for inherited COPD. Additional provisional patent applications have been filed to provide the Company with expanded intellectual property protection. The Company has expanded its CMO relationships to ensure it has the ability to manufacture clinical material for future trials. The Company confirms that its proprietary SDF platform provides significantly enhanced yields of alpha-1 protease inhibitor, at levels up to 10 times of that achievable with the industry standard Cohn processes, and with purity levels consistent with that achieved by other commercial processes.
Advanced pipeline programs: Together with its academic collaborators, the Company continued to progress its pre-clinical programs in juvenile Batten disease and its CRISPR-Cas9 program in Fanconi anemia (FA) and other rare blood disorders. Juvenile Batten disease is the most common form of a group of disorders known as neuronal ceriod lipofuscinosis (NCL), a lysosomal storage disease that affects the nervous system in children and for which there are no approved treatment options. Fanconi anemia is a rare pediatric blood disease characterized by multiple physical abnormalities, bone marrow failure and a higher than normal risk of cancer.