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3 Things In Biotech, April 22: All These 'A' Companies

by: Zach Hartman

Abeona gets an orphan into Europe.

AB Science gets bad news from the EMA.

Adamas provides a deeper update in its Parkinson's trial.

Note: Subscribers to Avisol Capital Partners Total Pharma Tracker got an early look at this publication. Try for free today and see what we're talking about! Here is some more information if you're curious.

Welcome to another edition of "3 Things In Biotech You Should Learn Today," a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.

Abeona gets an orphan into Europe

Company: Abeona Therapeutics (NASDAQ:ABEO)

Therapy: ABO-202

Disease: Neuronal ceroid lipofuscinosis

News: ABEO announced that its experimental gene therapy ABO-202 has been granted orphan designation in Europe for the treatment of neuronal ceroid lipofuscinosis, a fatal disease affecting young children's nervous systems. This builds upon similar designations granted by the FDA, based on encouraging animal work demonstrating increased survival and improved motor function with the therapy.

Looking forward: This is still very early news, but it's encouraging for yet another adeno-associated virus program, a vector that has inched its way towards clinical relevance in an increasingly impressive number of indications. Certainly, this is not ABEO's most important program, but it gives the company another foothold in a rare disease space, which will lead to rapid progress through the FDA, in relative terms.

I wouldn't buy on this news alone, but ABEO is a company well worth paying attention to at this point.

AB Science gets bad news from the EMA

Company: AB Science (OTCPK:ABSCF)

Therapy: Masitinib

Disease: Amyotrophic lateral sclerosis (ALS)

News: ABSCF announced that the EMA's CHMP has adopted a negative opinion on the application to approve masitinib for the treatment of ALS. This opinion was rendered on the grounds that data were not reliable enough to warrant approval, and it had issues with the interpretation of endpoints in the pivotal study, namely the distinction between "normal" progressors and "rapid" progressors. ABSCF will provide further analysis as part of the re-examination, at which time the CHMP will render a second opinion in July.

Looking forward: This is certainly not looking all that good for ABSCF at this point. I don't personally have a strong enough foundation in ALS biology or treatment to know whether these concerns are addressable within the next few months, but it is not often the case that these committees hear the company out, throw their hands in the air, and admit that they misunderstood the data. Therefore, consider me a pessimist on the outlook of this particular application.

Adamas provides a deeper update in its Parkinson's trial

Company: Adamas Pharmaceuticals (NASDAQ:ADMS)

Therapy: Amantadine

Disease: Parkinson's-associated dyskinesia

News: ADMS announced an analysis of final data from its EASE LID 2 study, a phase 3, open-label trial investigating amantadine to treat dyskinesia associated with Parkinson's disease. The study met its objective of favorable tolerability, as well as maintaining a durable treatment of motor complications.

Looking forward: In large part, the final analysis is a confirmation of findings published previously, but this is important to keep following up on, as patients receiving amantadine are going to receive it indefinitely, and the dyskinesia is not generally a life-threatening condition. Therefore, long-term safety is of paramount importance for the ongoing marketing success of this agent. Mission accomplished, it would seem!

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