Renevia® is in a pivotal clinical trial in Europe to assess its efficacy in delivering and engrafting transplanted cells to treat HIV-related facial lipoatrophy. Renevia® consists of our cell delivery matrix, HyStem®, combined with the patient’s own adipose, or fat, cells. We expect Renevia® to lead to better, more natural outcomes by enabling higher cell survival and engraftment. If successful, this outcome could result in a more natural appearance and texture to the face leading to a clinically differentiated increased quality of life. We expect to complete patient enrollment by the second half of 2016 with top-line efficacy data expected in early 2017. If the data are positive, we expect to file for CE Mark approval in the European Union in the first half of 2017. We consider the Renevia® trial to be an important “gateway” trial that could serve as the basis for targeting Renevia® for broader use in medical aesthetics, such as age-related and trauma-related facial fat loss, which represent a multi-billion dollar market opportunity. We continue to believe there are many other potential applications for Renevia® and the HyStem® platform in combination with a diverse range of cell therapies and surgical applications.
The loss of retinal pigment epithelial, or RPE, cells in the eye can cause either wet AMD (age-related macular degeneration) or dry AMD, the latter being a condition for which there are no currently approved therapies. Therapeutics for the “wet” form of AMD currently generate around $8 billion in annual sales, while the unserved dry AMD market has about nine times as many potential patients. OpRegen® is designed to treat dry AMD by integrating new RPE cells into the subretinal space to replace missing RPE cells. OpRegen® has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the treatment of dry AMD.
Patients are currently being treated with OpRegen® in a Phase I/IIa dose-escalation clinical trial. We expect to report completion of the first cohort of this trial and clearance from the independent Data and Safety Monitoring Board (DSMB) to begin the second cohort in the second quarter of 2016. The second cohort is expected to be enrolled, and we anticipate approval from the DSMB to proceed to the third cohort, by the end of this year.
Last year, promising long-term data was reported from a Phase II study of AST-VAC1 as an immunotherapy for Acute Myeloid Leukemia, and a successful End-of-Phase II meeting was held with the FDA. The FDA indicated general agreement with a proposed development plan for registration of AST-VAC1 via an accelerated development pathway with a Phase III clinical trial and a potential Biologic License Application or BLA filing. This year, a Special Protocol Assessment, or SPA, with the FDA will be sought to confirm the primary endpoint and other design elements of the pivotal Phase III trial.
Another therapeutic product candidate, AST-OPC1, is currently in a Phase I/IIa clinical trial to treat spinal cord injuries. The clinical trial has completed enrolling the first two cohorts and continues to enroll patients. More than 12,000 people sustain spinal cord injuries in the U.S. each year, but there are no FDA-approved therapeutics or devices that can restore motor function in these individuals.
Simplification: We took several steps to simplify our structure last year and are committed to continuing this process.
Last fall, we combined our ESI BIO Division with the drug toxicity screening business of Hepregen Corporation to form a new company called Ascendance Biotechnology, Inc. This transaction provided an experienced leader for the business and enabled BioTime to eliminate its ESI BIO operating expenses, while providing key stem cell technologies and cell lines to Ascendance for use in strengthening the drug toxicity screening product lines that Ascendance acquired from Hepregen. Since December, Ascendance has generated meaningful revenue growth from customers that include Boehringer Ingelheim, Pfizer, and Merck.
