In a recent press release, clinical-stage biopharmaceutical company Abeona Therapeutics Inc. (“Abeona”) shared that the company had a successful Type B meeting with the FDA. During the meeting, Abeona explained endpoints from the Phase 3 VIITAL trial, which would evaluate EB-101 for patients with recessive dystrophic epidermolysis bulls (RDEB).
According to the FDA, a Type B meeting can take a few forms, including, but not limited to:
- Discussing drug development programs for drugs which received Breakthrough Therapy designation
- Pre-investigational New Drug Application (pre-IND), pre-new drug application (pre-NDA), and pre-biologics license application (pre-BLA) meetings
- Evaluating risk evaluation and mitigation strategies
- End-of-phase 2 and pre-phase 3 meetings
In this case, Abeona wanted to ensure that they were aligning with their proposal for the VIITAL trial. Approximately 10-15 patients with RDEB will enroll. Between these patients will exist around 30 large, chronic wounds.
During the trial, patients will receive EB-101, an autologous, gene-corrected cell therapy. EB-101 works by delivering a functional version of COL7A1 to patients, allowing for increased collagen expression and additional wound healing assistance. In a prior study, EB-101 showed long-lasting responses, assisting with wound healing for 2-5+ years. Now, for the VIITAL study, primary endpoints include 50+% healing over a 6~ month period, and a reduction of pain.
Epidermolysis Bullosa (EB)
Typically caused by KRT5 or KRT14 gene mutations, epidermolysis bullosa (EB) consists of connective tissue diseases that cause moderate-to-severe skin blistering. There are three main forms of EB. First, epidermolysis bullosa simplex (EBS) causes thickened palm and sole skin, alongside widespread blistering on the hands. Next, junctional epidermolysis bullosa (JEB) results in thin skin and large, ulcerative blisters. Without treatment, these blisters can be lethal. Finally, dystrophic epidermolysis bullosa (DEB) usually occurs on the hands and feet. This form causes an increased risk of skin cancer.
The VIITAL trial focuses on recessive dystrophic epidermolysis bullosa (RDEB). According to Cure EB, RDEB is an incredibly painful condition which can cause:
- Eye, mouth, and esophageal blistering
- Painful eating
- Difficulty swallowing
- Anal fissures
- Gastrointestinal pain
- Light sensitivity
- Vision loss
- Loss of mobility
Unfortunately, many people with RDEB will develop skin cancer, such as squamous cell carcinoma, before their 30s. Currently, there is no cure for EB.