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Concert's (CNCE) CTP-656 Gets Orphan Drug Status in U.S.

Concert Pharmaceuticals, Inc.CNCE announced that the FDA has granted orphan drug designation to its candidate CTP-656 for the treatment of cystic fibrosis.

We note that orphan drug designation is granted to drugs capable of treating diseases affecting less than 200,000 people in the U.S.

Moreover, Concert's share price has declined 41.7% in the past one year compared with Zacks classified Medical-Drugs industry's fall of 13.1%.

As per estimates from the Cystic Fibrosis Foundation, about 70,000 people worldwide suffer from cystic fibrosisWith the orphan drug status, the company is eligible for certain other benefits, including tax credits related to clinical trial expenses, exemption from the FDA-user fee and assistance from the FDA in clinical trial design.

Concert commenced a placebo-controlled phase II study in Dec 2016 in the U.S. to assess CTP-656 in cystic fibrosis patients with gating mutations. Following the initiation of the study, the FDA asked the company for an adequate washout period, in which Vertex Pharmaceuticals' VRTX Kalydeco treatment is to be withheld in order to support dose selection for phase III, besides the requirement of placebo-control study. Top-line data from the study are expected to be out by the end of 2017.

We note that for the treatment of patients with cystic fibrosis, Concert had reported new data from part 1 of a multiple ascending dose phase I study on CTP-656 by comparing the candidate to Kalydeco in late February, last year, Results from which confirmed that CTP-656 has a superior pharmacokinetic profile to Kalydeco. This included a longer half-life of approximately 15 hours, a reduction in the rate of clearance and a substantial increase in exposure along with greater plasma levels at 24 hours.

Zacks Rank & Key Picks

Concert currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Enzo Biochem, Inc. ENZ and Anika Therapeutics Inc. ANIK . While Enzo Biochem sports a Zacks Rank #1 (Strong Buy), Anika carries a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank stocks here .

Enzo Biochem's loss estimates for 2017 narrowed 5.88% over the past 60 days. The company recorded a positive earnings surprise in three of the last four quarters, the average being 22.50%. Its share price was up 44.9% in the past one year.

Anika's earnings estimates for 2016 and 2017 were up 3.9% and 0.5%, respectively, over the last 60 days. The company recorded a positive earnings surprise in each of the last four quarters, the average being 37.8%. Its share price was up 36% in the past one year.

Concert Pharmaceuticals Inc. Price

Concert Pharmaceuticals Inc. Price | Concert Pharmaceuticals Inc. Quote

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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