Saturday, January 23, 2016

Galapagos Updates Cystic Fibrosis Program

Galapagos (GLPG) and AbbVie developed a partnership aimed at treating 90% of the Cystic Fibrosis population with a triple combination drug therapy.  The triple combo will involve a potentiator and two correctors for patients with the F508del mutation.  Under the agreement dated back to September of 2013, Galapagos leads discovery and development through phase 2, shares Phase 3 responsibility with AbbVie.  Below is a list of potential candidates that GLPG will be taking to the clinic for further exploration. 

Potentiators:
GLPG1837  Completed phase 1 multiple dosing trial, and heading into phase 2 for G551D and S1251N CF 2016.  
GLPG2451  Additional potentiator expected to move into a phase 1 clinical trial 2Q 2016.

Correctors:
GLPG2222  First generation corrector began dosing in healthy volunteers January.  Expects top line results in Q2 2016.  GLPG receives $10 million milestone from ABBV.
GLPG2851  Another first generation corrector, aiming to initiate phase 1, end of 2016.  
GLPG2665  Next-generation corrector, expected to enter phase 1, middle of 2016.  
GLPG2737  Next-generation corrector, expected to enter phase 1, Q4, 2016.  

Bottom Line:  It's good to see a company attempt to improve upon the currently approved combo Orkambi for Cystic Fibrosis people.  By the end of 2016, we should at least have some indication how corrector GLPG2222 and possibly GLPG2665 have done in phase 1 clinical trials.  Thank you for reading.  

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.