Monday, January 11, 2016

CTP-656 Cystic Fibrosis Market Opportunity

CTP-656 is the deutered version of Vertex drug Kalydeco.  Today, Vertex gave an updated sales forecast for Kalydeco at the JP Morgan Healthcare Conference.  The company expects 2016 revenues in the range of $675 million.  Kalydeco is provided to Cystic Fibrosis patients as mono therapy for the G551D and R117H mutation.  The annual cost of the therapy is approximately $300,000 annually.
Concert Pharmaceuticals plans on seeking regulatory approval for CTP-656 as mono therapy for people with the G551D and other gating mutations.  What could peak revenue look like for this set of patients excluding the more common F508del.  Below is the worldwide patient population for Kalydeco as mono-therapy, excluding any combination therapies for the most common form of CF, the F508del mutation, for which Orkambi is prescribed for.
  • G551D ages 6+ (US) 
  • G551D ages 6+ (EU, AUS & CAN)  
  • Gating, R117H & Ages 2-5
  • 4,000 eligible patients (Vertex Pharmaceuticals 2015 Year End Presentation)
The unique situation Concert finds itself in, if CTP-656 potentially does get FDA approval as mono-therapy, is that the footprint for those 4,000 patients available, have already been established from being prescribed Kalydeco.  The switch to CTP-656 from Kalydeco, could happen seamlessly if the drug is marketed right.
 
Penetration rate of 70% = 2,800 patients
Annual therapy price of $240,000 (20% discounted)
Potential revenue 2,800 patients * $240,000 annual therapy = $672 million

The advantages that CTP-656 offers is a once daily, with less dietary restrictions, that should lead to higher adherence rates. Thank you for reading.
 

2 comments:

  1. So what about the title? What is your view on revenue potential? ~10% penetration into the US population alone at a 25% discount to Kalydeco = 3,000 patients x $225,000/year = $675mn as monotherapy?

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  2. We have updated the revenue potential, which hits your revenue numbers but in a different manner. We are using a high penetration rate of 70%, due to the high visibility that the CF community shares. The once daily, and less restrictive meal preparation should be accepted well, if CTP-656 makes it through approval.

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