Seeking Alpha • Oct 19
Abeona: Pivotal Data Readout EB-101 Before Year End Could Boost Shareholder Value
Summary
Results from the pivotal VITAL study, using EB-101 for the treatment of patients with RDEB are expected to be released before the end of 2022.
Should EB-101 be approved by FDA for RDEB, it would give company a Priority Review Voucher, which could be sold for around $100 million or more to another pharmaceutical company.
ABO-102, which is being developed to treat patients with Sanfilippo syndrome type A has been offloaded to Ultragenyx Pharmaceutical for advancement.
Abeona has $26 million in cash as of June 30, 2022. It believes enough cash to fund operations until Q2 of 2023, but will likely have to raise cash before or after data from this late-stage study.
Abeona Therapeutics (ABEO) is a great speculative biotech play to look into. That's because it is gearing up to report results from its phase 3 pivotal study known as VITAL. This late-stage study is using its cell therapy known as EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). Results from this trial are expected any day now before the end of 2022, therefore, this provides traders/investors with a huge catalyst to look forward to. Abeona wants to focus on this program for its pipeline, therefore, it chose to offload ABO-102 (now UX111) for Sanfilippo syndrome type A ((MPS IIIA)) to Ultragenyx Pharmaceutical (RARE). That is, Ultragenyx will be responsible for taking over this program and moving it forward in the clinic. In return, Abeona will be receiving tiered royalties and commercial milestone payments should this drug ultimately be approved by regulators. With proof of concept established with EB-101 in a prior study, plus year-end 2022 data readout of EB-101 for RDEB, these are the reasons why I believe it is a great speculative biotech play to look into.
EB-101 Could Possibly Provide A Great Long-Term Durable Treatment Option For RDEB Patients
The main program in the pipeline is EB-101, which is being developed for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a terrible skin condition by which there is lack of collagen protein in the skin. It is a condition by which wounds are large and blisters tend to form. Patients with RDEB suffer greatly because of pain and complications which occur because of it. That's not the only problem to consider though for this patient population. The average life expectancy of someone who has it is 50 years of age. There are not currently any cures available for these patients, but there are treatments that attempt to control symptoms and pain. The main problem is that a mutation in the COL7A1 gene doesn't allow the formation of Type VII collagen, which is necessary for the skin to be healthy. EB-101 is a cell therapy which is being developed to provide functional collagen VII to the patient's own cells. The therapy is autologous, that is, cells are extracted from the patient and then transduced with the correct collagen gene necessary for proper formation. The final outcome is a fully formed sheet which is 5 to 7 cell layers thick. EB-101 is eventually applied to the wound in surgery which helps the patient to restore their functionality of COL7A1 gene encoding provided by the cells. In turn, EB-101 holds the potential to heal and provide pain relief for the patient. Even better, it provides a "one and done" scenario whereby it may be possible to avoid daily wound care. Abeona expects that EB-101 could produce estimated cumulative revenues of $1.5 billion over the products life cycle.
As I highlighted in the beginning Abeona Therapeutics is currently running its phase 3 VITAL study, which is using EB-101 for the treatment of patients with RDEB. It has been reported that the final patient completed their 6-month follow-up visit for this late-stage trial on October 4, 2022. This means within the next 3 to 4 weeks, investors should get an idea on whether or not this cell therapy succeeds in meeting the co-primary endpoints for this study. This means before the end of 2022, there will be a data readout from this VITAL study which could possibly boost shareholder value. Of course, both co-primary endpoints must be met for this to happen. Developing a treatment for large chronic wounds is not easy, as such, Abeona and the FDA came to an agreement on what the co-primary endpoints need to be in order for EB-101 to be approved for RDEB. The two co-primary endpoints necessary for regulatory approval are:
Proportion of chronic wounds with ≥50% healing from baseline at week 24 (Those treated with EB-101 versus those not treated at all with EB-101)
Pain reduction at dressing changes assesses by a mean difference in scores using the Wong-Baker FACES scale at week 24 (Treated EB-101 versus untreated EB-101)
