GLPG1837 for Cystic Fibrosis

Galapagos released phase 2, SAPHIRA(1) results this week with drug GLPG1837, for Cystic Fibrosis patients with the G551D mutation.  The link to the conference call is here http://edge.media-server.com/m/p/aen3zb2d.

Some details from the conference call:

• The company reported similar ppFEV1, in comparison to FDA approved Kalydeco
• Kalydeco patients at baseline had a sweat chloride reading of around 45 mmol/L
• After the seven day washout period, the sweat chloride level increased to a mean value of 98 mmol/L*
• After 28 days, and dosing up to 500 mg GLPG1837 twice daily, sweat chloride fell to 66 mmol/L
• For patients who exceeded the predicted target concentration, sweat chloride changed from a mean 94 mmol/L to 52 mmol/L
• Sweat chloride did not fall to the original baseline Kalydeco level, of around 45 mmol/L
• GLPG1837 was mentioned as being dose limited up to 600 mg twice daily

* The concentration of chloride in sweat is elevated in individuals with CF.

Thank you for reading.               

SAGE-217 Patent Issued

SAGE-217 was issued patent number US 9,512,165 B2, which runs to April 2034.  The link to other patent applications can be found here SAGE Patent Applications.  SAGE-217 is the oral version of SAGE-547, and will be running four proof-of-concept, phase 2 clinical trials in 2017.  Thank you for reading.                

CTP-543 Phase 1 Top-Line Results

Concert Pharmaceuticals announced top-line results this week for CTP-543.  The single and multiple ascending dose study was well-tolerated across all dose groups.  The company is planning on testing four doses (4, 8, 12, and 16 mg BID) in the phase 2 clinical trial scheduled to begin early 2017.  The phase 2 clinical trial will enroll around 100 patients with moderate to severe alopecia areata, with six month readout by the end of 2017.  Thank you for reading.

SAGE-547 Expedited Pathway Revealed

We initially wrote about Sage here, SAGE-547.  This week SAGE revealed through this press release Sage Therapeutics, the expedited pathway for drug SAGE-547 for Postpartum Depression.  The important news was that the FDA in agreement with Sage, anticipates that no additional controlled studies will be necessary to file an NDA.  The company believes that they may be able to file for a new drug application for Postpartum Depression in 2018.  If approved by the end of 2019, the company will have patent until around 2034, or approximately 15 years of exclusivity.  Below are the important aspects of the expedited pathway for SAGE-547, agreed upon by the FDA.

• Current SAGE-547 clinical studies confirmed as appropriate to support registration, if successful
• No additional efficacy studies expected to be required beyond those currently underway
• Trial design of studies 202B and 202C are considered appropriate for registration, with increase in size and other minor modifications
• The primary clinical endpoint for these pivotal trials was unchanged and agree upon with FDA
• Additional patient safety data may be acquired through an open-label program

Below is a year-to-date chart of SAGE.  Despite the company having an extraordinary year with SAGE-547 the stock is down -4.49% this year.  The company currently has $431 million in cash, or about $13.00 per share in cash.  

Source: Shaw Investments, StockCharts.com

Thank you for reading.                

CTP-656 Clinical Trial Initiated

CTP-656 for cystic fibrosis patients with CFTR gating mutations is now loaded in the clinicaltrials.gov website here NCT02971839.  The phase 2 trial is expected to start enrollment in January of 2017 to avoid the busy holiday season approaching.  The readout completion date is by the end of 2017.  Sweat chloride will be the primary endpoint of the study with FEV1 secondary.  With only 6-8 patients in each arm, the trial is not specifically powered for efficacy, but more likely to compare the three different dosages, and decide which one will be taken further into a phase 3 clinical trial, where FEV1 will be the primary endpoint.

Estimated Enrollment:	40
Study Start Date:	January 2017
Estimated Primary Completion Date:	December 2017 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: CTP-656, Dose 1, QD Oral tablet dosed once-daily for 28 days	Drug: CTP-656, Dose 1 (QD)
Experimental: CTP-656, Dose 2, QD Oral tablet dosed once-daily for 28 days	Drug: CTP-656, Dose 2 (QD)
Experimental: CTP-656, Dose 3, QD Oral tablet dosed once-daily for 28 days	Drug: CTP-656, Dose 3 (QD)
Active Comparator: Kalydeco, 150 mg Tablet (open label) 150 mg, oral tablet dosed twice-daily for 28 days	Drug: Kalydeco, 150 mg Tablet (BID; open-label)
Placebo Comparator: Placebo, Oral Tablet, QD Oral tablet dosed once-daily for 28 days	Drug: Placebo (QD)

Detailed Description:

This is a randomized, parallel-group, double-blind, placebo controlled multicenter study to evaluate the safety and efficacy of CTP-656 in CF patients with CFTR gating mutations, compared to Kalydeco, for a total of 28 days. Subjects will be randomized to receive either double-blind CTP-656 or placebo, or open-label Kalydeco.