PTC Therapeutics: A Speculative Buy On Rare Disease Portfolio And Potential Takeover Target
Small Pharma Analyst • Thu, Aug. 7
- PTCT has a drug candidate, Translarna (ataluren), in Phase 3 for treatment of both Cystic Fibrosis and Duchenne Muscular Dystrophy (DMD).
- The European Commission gave conditional approval for Translarna for DMD on August 4. FDA on July 31 promised enhanced support and accelerated approval of DMD drugs.
- Translarna has a risk-adjusted NPV of in the $2-3B range, depending on assumptions.
- Vertex expressed its intent to buy or partner to build out its cystic fibrosis franchise and develop additional specialty drug capabilities – PTCT is a potential takeover target.
- Given the NPV of Translarna and the potential for a takeover, PTCT is an attractive buy for a high risk-high reward biopharma investor.