CRISPER-Cas9 - Intellia Therapeutics

We previously wrote about the science of CRISPER-Cas9. Over the weekend Intellia Therapeutics (NTLA) released data at the American Society of Gene & Cell Therapy Annual Meeting with this press release Intelliatx.com. With this news the company may be getting closer to in human clinical trials for the first time. Intellia Therapeutics is our favorite Crisper-Cas9 company for many reasons, but primarily because of their collaborations with Novartis, and Regeneron. Still many years away from any FDA approval for any indication, the company is worth monitoring, along with it's competitors Crispr, Editas, and Cellectis. Thank you for reading!

CTP-543 Clinical Trial Initiated

This phase 2 for Alopecia Areata will have a primary endpoint with the number of responders achieving at least 50% improvement in SALT score at 24 weeks.

Estimated Enrollment: 100
Anticipated Study Start Date:	May 2017
Estimated Study Completion Date:	July 2018
Estimated Primary Completion Date:	March 2018 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: CTP-543, 4 mg Oral tablet, dosed twice-daily	Drug: CTP-543 tablets
Experimental: CTP-543, 8 mg Oral tablet, dosed twice-daily	Drug: CTP-543 tablets
Experimental: CTP-543, 12 mg Oral tablet, dosed twice-daily	Drug: CTP-543 tablets
Experimental: CTP-543, 16 mg Oral tablet, dosed twice-daily	Drug: CTP-543 tablets
Experimental: Placebo Oral tablet, dosed twice-daily	Drug: Placebo tablets

Detailed Description:

This is a double-blind, randomized, placebo-controlled, parallel dose-comparison, multi-center study of CTP-543. Subjects will be randomized to either an active dose of CTP-543 or placebo for an initial 24-week efficacy period. A 28-week dose-adjustment period will follow where patients tolerating treatment may adjust dose at Week 24 as needed, and placebo patients will be switched to active treatment.

 

Below is a daily chart of Concert Pharmaceuticals. The stock has been basing since the big move higher on March 6, when the company announced the sale of CTP-656 to Vertex. Thank you for reading.