CTP-656 Receives Orphan Drug Designation

Concert Pharmaceuticals received Orphan Drug Designation from the FDA in press release here, Orphan Drug Designation for CTP-656 for the Treatment of Cystic Fibrosis.  The significance of this, is that CTP-656 could receive up to seven years of marketing exclusivity, and even more importantly, gets FDA assistance in clinical trial design.  Orphan Drug Designation is given to a disease or condition that affects less than 200,000 people in the U.S.  Thank you for reading.
 

What is CTP-543

CTP-543 is the deuterated version of FDA approved drug Ruxolitinib, approved for myelofibrosis, and polycythemia vera.  Concert Pharmaceuticals plans to address unmet need Alopecia Areata with CTP-543.  Proof of concept was confirmed with this academic clinical trial, by Columbia University here JCI.  The company will be in a phase 2 clinical trial with around 90 participants beginning in the first quarter of 2017, with readout by the end of 2018. Thank you for reading.

Super Refractory Status Epilepticus (SRSE)

Sage Therapeutics - SAGE, is currently in phase 3 for unmet need (SRSE) with drug SAGE-547.   SRSE is a rare neurological condition, and a sub-type of Status Epilepticus (SE), which is a prolonged seizure.  A diagram from this link explains the stages. The Treatment of Super-refractory Status Epilepticus.

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SAGE Therapeutics has a Special Protocol Assessment (SPA) with the FDA for this phase 3 clinical trial.  The company mentioned, that Refractory Status Epilepticus (RSE) has up to 50% response rate, and that (SRSE) has approximately 35% response rate.  The trial should complete first half of 2017.  Thank you for reading.        

CTP-656 For Cystic Fibrosis

CTP-656 is the name of Concert Pharmaceuticals deuterated version of Vertex's Kalydeco, which is approved for cystic fibrosis patients with the G551D mutation, and as combo with corrector Lumacaftor (known as Orkambi), for patients with the F508del mutation.  The hope is that CTP-656 enhances therapy for cystic fibrosis patients by improving the metabolic profile, reduce dosage to once daily, or ameliorate drug interaction, over currently FDA approved Kalydeco.  Thank you for reading.