Merger boosts development of Sanfilippo treatment

Following acquisition of Abeona, PlasmaTech plans to push company’s pipeline forward with two Phase 1/2 clinical trials in 2015

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NEW YORK—PlasmaTech Biopharmaceuticals has bolstered its focus on developing treatments for rare diseases with the acquisition of Abeona Therapeutics, a company focused on developing treatments for lysomal storage diseases. Abeona’s previous preclinical studies of its drugs ABX-101 and ABX-102 have demonstrated promising efficacy in cognitive function, motor and survival in Sanfilippo syndromes A and B (MPS-IIIA and MPS-IIIB). PlasmaTech will now push forward Abeona’s pipeline by launching two Phase 1/2 clinical trials for the drugs in 2015. The U.S. Food and Drug Administration (FDA) last month granted both product candidates Orphan Drug Designation and Rare Pediatric Disease Designation.
Jeffrey Davis, chief operating officer of PlasmaTech, tells DDNews that the merger was the result of an interest in gene and cell therapy that arose from the company’s work in developing a new-generation plasma processing technology that could be used in the treatment of rare genetic conditions such as alpha-1 antitrypsin deficiency. “As we were raising money to fund its development, we were often faced with the question of what happens when next-generation therapy is able to fix the broken gene that results in these conditions,” he says. “So we do see this as synergistic as well as a natural progression from our desire to build a franchise in the cell and gene therapy space and really focus on rare disease opportunities.”
PlasmaTech specializes in developing protein biologic therapies and oncology supportive care products. The company has two commercial products for patients undergoing cancer treatments, MuGard and ProctiGard, and is actively developing and commercializing its human plasma-derived therapeutics, including the alpha-1 protease inhibitor, known as SDF Alpha. Abeona specializes in developing gene therapies for patients with rare genetic diseases, including MPS-III, which is a rare autosomal recessive lysosomal storage disease. The company was formed in early 2013 to focus the search for a cure for MPS-III and provide a unifying voice for patient advocate groups, researchers, clinicians and investors.
“We believe this acquisition represents an exciting cornerstone of our strategy to build a world-class cell and gene therapy company focused on rare diseases,” said Steven H. Rouhandeh, PlasmaTech’s executive chairman.
There is currently no FDA-approved treatment for MPS-III. PlasmaTech executives say the acquisition of Abeona will provide its programs to develop a cure increased access to capital and organizational capabilities that will accelerate development and internationalization. Abeona’s operating and scientific teams are expected to remain intact. The increased funding provided by PlasmaTech will supplement $4.8 million in financing Abeona was recently awarded from leading Sanfilippo foundations worldwide to support its clinical programs. The company has an additional $1 million commitment for manufacturing.
Shortly after the merger of PlasmaTech and Abeona was announced, the FDA approved Orphan Drug Designation and Rare Pediatric Disease designations for Abeona.
“We are very pleased to announce that the FDA has provided these two important designations to our Sanfilippo drug candidates, reflecting their potential to address the significant unmet medical needs of children with these rare and devastating lysosomal storage diseases,” said Abeona’s co-founder Tim Miller. “The benefits and incentives associated with these designations, including marketing exclusivity periods and the potential to obtain two valuable Pediatric Disease Priority Review vouchers, are strategically important from a regulatory and commercial perspective and potentially very value-creating for shareholders.”
Miller, who was previously CEO and president of Abeona, will now become president and CEO of PlasmaTech, replacing Scott Schorer, who will serve as a consultant to the company going forward. Miller has more than 16 years of scientific research, product development, clinical operations and business development expertise, with a focus on transitioning novel biotherapeutics through preclinical development and into Phase 1 and 2 human clinical trials. He has managed all aspects of biotechnology business development, manufacturing of biologics and multiple clinical programs from start-up and has direct experience engaging FDA and National Institutes of Health advisory agencies on multiple Investigational New Drug submissions.
“Tim Miller brings significant experience and leadership in developing and commercializing novel cell and gene therapies,” said Rouhandeh.
PlasmaTech’s board of directors and the managers of Abeona have unanimously approved the transaction, which at press time was expected to close in the second quarter of 2015. PlasmaTech announced its acquisition of Abeona on May 6. The terms of the agreement call for PlasmaTech to issue Abeona members a total of 3,979,761 common shares upon closing of the transaction and up to an additional $9 million in performance milestones, in common stock or cash, at the company’s option.

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