Abeona Therapeutics (NASDAQ:ABEO) is a good speculative biotech play to look into. The reason why I state that is because it is gearing up to file a Biologics Licensing Application [BLA] to the FDA of EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa [RDEB]. It expects to file a BLA of this drug for this indication in late Q2 or early Q3 of 2023. The last time I wrote an article on this biotech, it had not yet released results from its phase 3 VITAL study. Well, it was not only able to show positive data from this late-stage study, but will now be able to move towards the BLA filing it is gearing up for. In addition, it has several other preclinical stage gene therapies in its pipeline which it can advance. It is working on developing novel adeno-associated virus [AAV] capsids in AAV based therapies for the treatment of patients with serious eye diseases such as the three of the following: Stargardt disease, X-linked retinoschisis [XLRS] and autosomal dominant optic atrophy [ADOA]. Investors will get an early look into preclinical data for several of these indications, because Abeona recently announced that three abstracts were accepted for the 26th Annual Meeting Of the American Society of Gene & Cell therapy [ASGCT], which is taking place May 16th – 20th of 2023.
Positive Results From The Phase 3 VITAL study Using EB-101
As I stated above, Abeona Therapeutics will be able to file its Biologics Licensing Application [BLA] of EB-101 to the FDA for the treatment of patients with recessive dystrophic epidermolysis bullosa [RDEB] in the coming months. The specific expected time frame for the filing of this BLA is late Q2 or early Q3 of 2023. The reason why it will be able to file a BLA for this gene therapy for the treatment of this patient population is because of positive results it had obtained from the phase 3 VITAL study. Again, the last time I went over this biotech it had not yet released its results for this study. Not only did it achieve success with respect to the late-stage VITAL study, but will also be able to move towards filing a BLA of EB-101 for RDEB.
Abeona and the FDA came to an agreement on what co-primary endpoints would be needed in order for EB-101 to be eventually be approved for RDEB. The two co-primary endpoints necessary for regulatory approval stated were:
- 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)
The Wong-Baker Faces scale was developed by Donna Wong and Connie Baker. The scale shows a series of faces where a happy face is marked at “0” and then a crying face is marked as a “10” (worst pain possible). The patient chooses their level of pain on this scale.
Back in November of 2022, it was reported that the phase 3 VITAL study was able to meet on both co-primary endpoints. That is, treatment of RDEB patients with EB-101 resulted in statistically significant improvements in wound healing and pain reduction. With respect to the wound healing endpoint, it was measured from baseline all the way to 6 months of treatment. As such, wound healing percentage improvement from baseline of 81.4% was observed in EB-101 treated randomize wounds, compared to only a 16.3% improvement from baseline seen with those who were given control. As you can see, treatment with EB-101 resulted in a much larger percentage of improvement in wound healing, compared to control. The co-primary endpoint though was only calling for the drug to achieve a 50% improvement for this co-primary endpoint to be met. Not only did Abeona achieve this, it went way beyond the requirement. With respect to the co-primary endpoint dealing with pain, EB-101 showed a statistically significant pain reduction associated with wound dressing changes compared to control over a 6-month period. This endpoint was met with statistical significance with a p-value of p =0.0002. That is, treatment with EB-101 established a mean pain reduction associated with wound dressing of 3.08 points from baseline, compared to control which only established a 0.90 mean pain reduction instead.
Financials
According to the 10-K SEC Filing, Abeona Therapeutics had cash, cash equivalents, restricted cash and short-term investments of $52.5 million as of December 31, 2022. A big reason for the cash on hand is because in November of 2022, it successfully completed a $35 million private placement financing agreement with new and existing institutional investors. That is when it entered into a securities purchase agreement to sell 7,065,946 shares of its common stock and in lieu of shares of common stock, pre-funded warrants exercisable for 543,933 shares of common stock and accompanying warrants to purchase 7,609,879 shares of its common stock to a group of new and existing investors in a private placement. Abeona estimates that it has enough cash to fund its operations into Q3 of 2024. Considering that it is gearing up to file its BLA of EB-101, plus potential FDA approval thereafter, it will need to raise additional cash at some point in 2023. My guess would be if the stock trades much higher after it is announced that the BLA has been filed.
Risks To Business
There are several risks that traders/investors should be aware of before investing in this biotech. The first risk to consider would be with respect to the BLA filing of EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa for possible FDA approval. The risk is that the FDA has to first accept the BLA filing upon submission before any review can take place. There is no assurance that the BLA filing will be accepted for review by the FDA. In addition, even if the BLA of EB-101 is accepted for review by the FDA, there is no guarantee that this treatment will be approved to treat patients with RDEB. A second risk to consider would be with respect to the ongoing preclinical gene therapy programs in the pipeline. Three Abstracts are to be presented at the upcoming 26th Annual Meeting Of the American Society of Gene & Cell Therapy [ASGCT], which is expected to take place between May 16th – 20th of 2023. There is no guarantee that these preclinical results to be released from a few of these Abstracts will end up being successful.
Conclusion
The final conclusion is that Abeona Therapeutics is a good speculative biotech play to look into. That’s because it is gearing up to file its Biologics Licensing Application [BLA] of EB-101 for the treatment of patients with RDEB in late Q2 or early Q3 of 2023. In addition to having this BLA filing of EB-101 act as a catalyst opportunity for traders/investors. It is also advancing several AAV capsids in AAV based therapies for the treatment of patients with serious eye diseases such as: Stargardt disease, X-linked retinoschisis [XLRS] and autosomal dominant optic atrophy [ADOA]. The only downside with respect to several of these programs is that they are only in preclinical testing. However, that doesn’t mean that traders/investors won’t have another catalyst to look forward to in the short-term. It is expected that Abeona Therapeutics will release preclinical data from several of the gene therapy programs noted directly above at a medical conference. That is, three abstracts were accepted and announced for the 26th Annual Meeting Of the American Society of Gene & Cell therapy [ASGCT], which is taking place May 16th – 20th of 2023. With the BLA filing of EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa [RDEB] expected in the coming months, plus the release of three abstracts expected at a medical meeting next month in May of 2023, these are the reasons why I believe that Abeona is a good speculative biotech play to look into.
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