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The Muscular Dystrophy Drugs That You Never Heard Of.

|Includes: BMRN, CATB, LEAF, LIFE, PTCT, SMMT, Sarepta Therapeutics, Inc. (SRPT)

After a dismal failure from Biomarin's (NASDAQ:BMRN) exon 51 skipping mutation, the FDA will still be looking to approve Muscular Dystrophy drugs in 2016. The rare disease of DMD has no FDA approved treatments, and as recent as last week, market consensus also turned negative on Sarepta's (NASDAQ:SRPT) exon 51 skipping drug. A few months ago analysts were pricing these drugs and now the FDA is requiring both companies to conduct more research and show more evidence of good patient outcomes. Ultimately, both drugs, drisapersen and eteplirsen, have not completed phase III studies and are suffering delays to market due to their "effectiveness". Even though both drugs have the opportunity to capture FDA approval later this year, analysts across Wall Street have cut their losses for now and are patiently waiting for new data to capitalize on this untapped market.


A catalyst in 1H 2016 in a Phase 1b/2 Resolaris trial could send this beaten down orphan drug company near all time highs. The company trades close to cash value ($125M) and just selected another preclinical physiocrine to put forth as an Investigational New Drug (IND). Recent studies and data suggest that aTyr has one of the most unique pipelines in the world. The largest shareholders of aTyr as of 09/30 were Fidelity and the Baker Brothers, who owned almost 10% of the shares outstanding. Analysts at JP Morgan and Citi rated the company a buy and overweight as of June 2015.

Catabasis (NASDAQ:CATB)

Another company in the DMD space that analysts will be looking for phase 1/2 data from is Catabasis Pharmaceuticals . The Cambridge based pharma company has fast-track and orphan drug designation for their blockbuster. In December, the company initiated a 12 week trial and should post results in 1H 2016. In July 2015, Wedbush, Oppenheimer, and Citi initiated buys on CATB. Also CATB makes up a large portion of fidelity ETFs and mutual funds in biotech and growth.


Lastly, New Jersey based PTC Therapeutics has the largest phase III DMD study in the U.S. and a rolling NDA with the FDA for their Translarna therapy. The company reported last week that they expect an FDA review in mid-2016. The company has the largest market cap of all three alt DMD companies I have mentioned. Vanguard and Wells Fargo own over 10% of the companies outstanding shares and JP Morgan and Citi also gave this one a buy in 2015.

UPDATE: Price cut for Translarna by 60% in Germany and rolling NDA dismissed by FDA.

Supporting Documents

  1. Sarepta__SRPT__Plunges_on_Harsh_FDA_Eteplirsen_Rev...

Disclosure: I am/we are long LIFE, CATB, PTCT.