Oppenheimer Sees 'Significant Upside Potential' In PTC Therapeutics

Oppenheimer’s Christopher Marai maintained an Outperform rating for PTC Therapeutics, Inc. PTCT, while reducing the price target from $135 to $111.

PTC Therapeutics is a biotechnology company focused on the development of Translarna, an orally delivered treatment for rare diseases caused by nonsense mutations including Duchenne muscular dystrophy [DMD] and cystic fibrosis [CF].

Analyst Christopher Marai believes that the FDA’s recent RTF policy is unlikely to have an impact on the EU-approval timeline for Translama. He wrote, “We do not anticipate the EMA will pull Translarna from the market, and anticipate that the EMA will fully approve, or extend Translarna’s conditional approval by July 31, 2016.”

The analyst pointed out that the potential new NDA info requested in RTF is not reflective of the FDA’s opinion or review of the drug. Marai mentioned that PTC Therapeutics’ trial data is fileable. The FDA’s RTF policy suggests that failed trials do not preclude a filing, but explanations for failure may.

Checks with KOL’s close to the FDA indicate that the regulators may accept PTC Therapeutics’ pooled/subgroup analysis which was based on the clinical understanding of DMD and pooling is based upon statistical dogma.

The downward revision in the price target reflects the removal of “RG7800 in SMA and pushing out our timeline for Translarna sales in nmDMD to mid-2017,” Marai stated.