- Abeona Therapeutics has inked a deal with Regenxbio to secure supply of AAV9 vector for gene therapies targeting four rare lysosomal disorders, including Sanfilippo syndrome types A and B as well as two types of Batten disease.
- Abeona will pay $20 million upfront, as well as a potential $160 million in fees and milestone payments, to secure rights to Regenxbio's NAV AAV9 vector in these disease areas.
- For Regenxbio, the deal is another validation of its gene therapy delivery platform and helps to bolster the biotech's cash position.
Securing vector delivery systems is a key hurdle for gene therapy development. Obtaining a well-validated vector could help take out some of that uncertainty for Abeona
Regenxbio's NAV AAV9 vector is used in Novartis' AVXS-101, for which the Swiss pharma expects to soon secure U.S. approval in spinal muscular atrophy. And just last month, Regenxbio licensed vectors, including AAV9, to Ultragenyx to develop a gene therapy for CDKL5 deficiency disorder.
"This license agreement further validates the potential of NAV AAV9 for the treatment of systemic and CNS manifestations of lysosomal storage diseases," said Kenneth Mills, President and Chief Executive Officer of Regenxbio in a statement.
Abeona's share price has slipped by nearly half to date this year. Maury Raycroft, an analyst at Jefferies, sees this deal as a potential turnaround for Abeona, following the appointment of Carsten Thiel as CEO in April 2018.
"The new CEO made reaching a license agreement [with Regenxbio] a priority and executed in a short amount of time," the analyst wrote in a Nov. 5 note to investors. "We expect more events to follow suit, including key ones such as starting the EB-101 [Phase 3] and then providing regulatory feedback on [the next step] for MPS IIIA."
Abeona's lead AAV programs, ABO-101 and ABO-102, are in Phase 1/2 testing in, respectively, MPSIIIB and MPSIIIA, also known as Sanfilippo syndrome types B and A. Raycroft sees potential for two other programs, currently in preclinical development, to progress more quickly given the experience that the company has gained in gene therapy development.