aTyr Pharma, Inc. (NASDAQ:LIFE)
aTyr Pharma, Inc. (NASDAQ:LIFE) announced today that the Food & Drug Administration (FDA) has granted Orphan Drug Designation to Atyr’s product candidate Resolaris – a drug developed to treat limb girdle muscular dystrophy. Shares of LIFE are up 10% in pre-market and trading at $3.80.
In October 2016, aTyr announced that Resolaris was granted Fast Track designation by the FDA for the treatment of facioscapulohumeral muscular dystrophy (FSHD), making it the first known therapeutic candidate for the treatment of FSHD to receive the designation In mid-January of 2016, aTyr announced that Resolaris was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of limb girdle muscular dystrophy 2B (LGMD2B), making it the first known therapeutic candidate for the treatment of LGMD2B to receive the designation. In addition, the FDA removed its partial clinical hold on a dosing ceiling for Resolaris in clinical trials. Clearly, aTyr Pharma, Inc. (NASDAQ:LIFE) fortunes are heavily tied to the performance of Resolaris.
The FDA’s Orphan Drug Designation program is intended to advance the development of products which demonstrate promise in diagnosing or treating rare conditions that affect fewer than 200,000 people in the U.S. Sponsors developing orphan-designated products are eligible for incentives under the program, including seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and certain tax credits.
Limb girdle muscular dystrophy (LGMD) refers to a group of rare genetic myopathies, of which there are more than 20 different subtypes, none with approved therapies. LGMD affects an estimated 16,000 patients in the U.S., approximately 3,000 of whom have LGMD2B. LGMD2B is a recessive genetic disease caused by a toxic loss of function in the dysferlin gene. Patients experience progressive debilitating muscle weakness and atrophy as well as immune cell invasion in the skeletal muscle. Resolaris is being developed by aTyr Pharma, Inc. (NASDAQ:LIFE) as a potential first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component.
aTyr Pharma, Inc. (NASDAQ:LIFE) likely welcomes the good news. In December 2016 JP Morgan downgraded the shares from “Overweight” to “Neutral. aTyre has no reported sales and has lost EPS every year – LIFE shares had an EPS loss of $2.03 in 2015. All three firms that follow ATyr Pharma rate the shares as a “Hold”.
|Last Price a/o 8:30 AM EST||$ 3.45|
|Market Cap (mlns)||$ 83.00|
|Shares Outstanding (mlns)||24.06|
|Share Float (mlns)||23.15|
|Short Interest Ratio||3.51|
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About the author: Steve Clark is a 23-year Wall St professional with stints in M&A, risk management, and algorithm trading.