PTC Therapeutics Could Quintuple In Price, Oppenheimer Predicts

• Shares of PTC Therapeutics, Inc. PTCT lost nearly 8 percent by Monday afternoon.
• The company released Phase 2 data at the International Congress of the World Muscle Society.
• Christopher Marai of Oppenheimer maintained an Outperform rating and unchanged $155 price target following the conference.

Christopher Marai of Oppenheimer spent the weekend at the International Congress of the World Muscle Society (WMS) and observed PTC Therapeutics' presentations.

Marai noted that the three key takeaways from the company include: 1) Translarna P3-study is "overpowered," which "bodes well for success," 2) the company has an agreement with the FDA to pool data from P2 and P3, which could support approval, and 3) some patients in trial showing benefit (while blinded), and doctors are assuming this reflects the benefit from Translarna.

Marai expanded on the P3 data, stating, "Our analysis suggests even a less 6MWT benefit in the P3-ACT-DMD trial would result in a stat sig, clinically meaningful benefit (28m) suitable for approval. Recall, this pooling approach was pursued in the accepted drisapersen NDA."

Marai added, "PTCT specified P3-enrollment to <80% %-predicted; note in the P2 patients <70% %-predicted receiving low-dose Translarna showed a 41-47m benefit vs placebo. Investor focus has been on age and baseline 6MWT enrollment criteria, missing the more sensitive %-predicted value that incorporates height, and can provide a robust assessment of drug benefit on a patient-by-patient basis."

Bottom line, Marai is expecting a positive P3 trial readout as soon as the fourth quarter 2015 with approval in early 2016. In addition, past data sets represent "added value" to the company and investors, and not "greater risk" as some would have assumed.

Shares remain Outperform rated with an unchanged $155 price target.