Acadia Pharmaceuticals ($17.26)

We started back into Acadia Pharmaceuticals today, after being extremely patient, since selling shares the morning that the CEO announced resignation in March 2015.  We wrote about that here, Acadia CEO Resigns - Effective Immediately.  Fundamentally not much has changed with their lead drug Nuplazid.  They have an FDA potential approval date of May 1st, 2016.  A list of important reiterating facts are below. 

-Nuplazid will be the only approved drug for Parkinson's Disease Psychosis (PDP).
-Their patent runs through 2028 (prior to any Hatch-Waxman addition) in the U.S. and 2025 in the EU.  So approximately 12 years of exclusivity U.S., unless a new drug comes to market during that time frame.  
-The drug could receive premium pricing up to around $15,000 per year for PDP patients, and be seen as a cost savings for family and the medical system.
-Nuplazid may have the potential to treat Alzheimer's Agitation, which at present is another unmet need, as the company plans to run a phase 2 clinical trial mid 2016.  
-The drug could potentially be used as maintenance therapy for schizophrenia patients, as a safer alternative to current anti-psychotics.  
-The company currently has around 124 mn shares outstanding.
-Their cash position is around $500 mn.  
-ADCOM (advisory committee) will meet on March 29th.


Bottom Line:  A weak overall market and biotechnology in particular, has allowed us to buy back shares of ACAD at reasonable prices.  I am expecting a black box warning label upon approval around May 1st, but do not believe that will have a major impact on the success of Nuplazid for PDP.  Thank you for reading. 
 

GLPG1837 for Cystic Fibrosis

GLPG1837 has advanced into a phase 2 clinical trial for Cystic Fibrosis people with the G551D, or S1251N class III mutation.  This will be the first of two, phase 2 clinical trials testing the drug in CF. The Galapagos press release is here Galagapos starts SAPHIRA Phase 2 study.  In a previous post we compared the phase 1 clinical trial results of GLPG1837 and Concert's CTP-656 side by side, CTP-656 & GLPG1837 Drug Comparison.  The drug will be dosed twice daily, and the trial should be complete by the end of 2016.  Thank you for reading.
 

CTP-656 Clinical Trial Chronology

The company has advanced CTP-656 nicely since receiving U.S. patent in the fall of 2014. Below is a chronology of the drug's clinical progress.
    
    First Half 2017 - European switching study CTP-656, and Kalydeco.
✔ January 2017 - Start phase 2 for mono-therapy, various gating mutations.
✔ February 2016 - Food effect study in healthy male volunteers, solid oral dose.
✔ November 2015 - Multiple ascending dose (PK) crossover study, with CTP-656 and         Kalydeco in healthy volunteers, solid oral dose.
✔ March 2015 - Single ascending dose (PK) crossover study, with CTP-656, and Kalydeco in healthy volunteers, oral suspension.  Second part of phase 1.
✔ March 2015 - Phase 1, D9 and D18 analog (PK) comparison in healthy volunteers.  The first part of phase 1.
✔ 2012 - Pre-clinical D9, D18 and Kalydeco plasma study comparison in dogs.

Thank you for reading.

Vertex: Complete Response Letter (CRL) From U.S. FDA

On February 5th it was press released that Vertex Pharmaceuticals received a CRL from the FDA.  A Food and Drug Administration CRL is issued when the FDA communicates to a company that an application to market a drug (NDA, new drug application) will not be approved in it's present form, and that the review by the FDA has been complete.
  
Vertex submitted a supplemental new drug application (sNDA) for the already approved Kalydeco.  The sNDA was filed for Cystic Fibrosis people 2 years and older, who have one of 23 residual function mutations in the cystic fibrosis gene.   Eight of the 23 were represented in a phase 2A study. It is estimated that over 1,500 people in the U.S. have one of the 23 gene mutations. The numbers do not seem all that large.  But if you factor in that Kalydeco is priced around $300k for annual therapy, then the numbers become quite large, up to around $450mn in potential annual revenue for the company if the sNDA was approved.
The 23 residual function mutations included in the sNDA were: 2789+5G- > A, 3849+10kbC- > T, 3272-26A- > G, 711+3A- > G, E56K, P67L, R74W, D110E, D110H, R117C, L206W, R347H, R352Q, A455E, D579G, E831X, S945L, S977F, F1052V, R1070W, F1074L, D1152H, and D1270N.  The press release is here Vertex Complete Response Letter.  More than likely the CRL was issued to Vertex because only eight of the 23 residual function mutations were represented in the phase 2A study.  Thank you for reading.