FDA lifts hold on Abeona's butterfly disease therapy, paving way for pivotal study
It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

The therapy, EB-101, is being developed to treat recessive dystrophic epidermolysis bullosa, which is caused by the deficiency of the protein COL7 and for which there is no approved therapy. Abeona’s autologous therapy involves transferring COL7A1 genes into a patient’s own skin cells, following which the cells are transplanted back into the patient to enable normal Type VII collagen expression and wound healing.
Early Phase I/II data show that EB-101 was safe and induced durable wound healing with up to five years of followup, and Type VII collagen expression was observed more than two years after treatment. However, in September, the FDA imposed a hold on the program, asking for additional data points on the “transport stability of EB-101 to clinical sites.”
The company, which also said it was reviewing ‘strategic options’ in response to interest earlier this year, now expects to kick off the late-stage VIITAL study in the first quarter of 2020. The trial is expected to enroll 10 to 15 patients, with roughly 30 chronic wound sites. The main endpoint of the study is the proportion of wounds with greater than 50% healing at three months, comparing treated with untreated wound sites on the same patient.
Abeona’s shares $ABEO jumped more than 9% to $3.28 in early Monday trading.
Epidermolysis bullosa (EB) is a group of genetic skin conditions that cause the skin to blister and tear due to minimal contact — infants born with the disease are called ‘butterfly children’ as their skin is considered as fragile as a wing of a butterfly.
In April, Castle Creek swooped to partner with the embattled gene and cell therapy Fibrocell to shepherd its lead gene therapy for recessive dystrophic epidermolysis bullosa into late-stage development. Months later, the New Jersey-based dermatology company acquired its partner in a deal worth $63.3 million.