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SNDX Rises On Entinostat Data, Kalydeco Gets FDA Nod, Concert Fails To Enthrall

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Today's Daily Dose brings you news about Avita Medical's ReCell device trial results; Blueprint's upcoming event to watch out for; Concert Pharma's phase II alopecia areata trial; GlycoMimetics' acute myeloid leukemia drug candidate; Repros' public offering; Syndax' ENCORE 601 trial results; FDA approval for expanded use of Vertex Pharma's Kalydeco and Ziopharm's phase I glioblastoma trial.

Read on.

Avita Medical Ltd.'s (AVMXY.OB) pivotal trial of ReCell device to treat severe burns has achieved both co-primary endpoints.

The Company noted that it is on track to file a Premarket Approval application for the ReCell Autologous Cell Harvesting device with the FDA in mid-2017.

AVMXY.OB closed Wednesday's trading at $1.34, up 2.29%.

Blueprint Medicines Corp. (BPMC) is scheduled to present new data from its ongoing phase 1 clinical trial of BLU-285 for the treatment of advanced gastrointestinal stromal tumors on June 5, 2017 at the 2017 American Society of Clinical Oncology Annual Meeting.

BPMC closed Wednesday's trading at $38.70, down 6.34%.

The FDA has placed a clinical hold on Concert Pharmaceuticals Inc.'s (CNCE) Phase 2a clinical trial of CTP-543 for alopecia areata, also known as spot baldness.

The regulatory agency has informed the Company that a review of certain recently completed non-clinical toxicology studies is required before CTP-543 can advance to phase IIa trial. Safety concerns are not the reason for the FDA's decision to impose the clinical hold, the Company noted.

CNCE closed Wednesday's trading at $14.85, down 7.48%.

Shares of GlycoMimetics Inc. (GLYC) surged more than 37% in extended trading on Wednesday after being granted Breakthrough Therapy Designation by the FDA for its drug candidate GMI-1271 for the treatment of adult relapsed/refractory acute myeloid leukemia.

GMI-1271 is currently under phase 2 portion of a Phase 1/2 clinical trial in patients with acute myeloid leukemia.

GLYC closed Wednesday's trading at $5.37, unchanged from the previous day's close. In after-hours, the stock was up 37.06% to $7.36.

Shares of Repros Therapeutics Inc. (RPRX) plunged more than 13% in extended trading on Wednesday after the Company's proposed public offering of common stock and warrants to purchase common stock.

RPRX closed Wednesday's trading at $0.80, down 5.76%. In after-hours, the stock fell 13.73% to $0.69.

Syndax Pharmaceuticals Inc. (SNDX) announced results from the melanoma cohort of the ongoing phase II trial of Entinostat in combination with Merck's KEYTRUDA, dubbed ENCORE 601.

The trial data has shown that with the addition of Entinostat, meaningful responses can occur in patients who have progressed on an anti-PD-1 or anti-PD-1/anti-CTLA-4 regimen like Keytruda, noted the Company.

SNDX closed Wednesday's trading at $13.58, up 44.62%. In after-hours, the stock gained another 6.77% to $14.50.

Vertex Pharmaceuticals Inc.'s (VRTX) KALYDECO has won FDA approval for expanded use in people with cystic fibrosis ages 2 and older who have one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator gene.

More than 900 people ages 2 and older in the U.S. have one of the 23 mutations.

KALYDECO was approved by the FDA initially in January 2012 for use in patients ages 6 years and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator gene. In December 2014, the drug was approved for additional use in people with cystic fibrosis ages 6 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Barely after 3 months, i.e., in March 2015, KALYDECO was approved for use in children ages 2 to 5 with cystic fibrosis who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H).

KALYDECO had sales of $703 million in 2016. For 2017, the Company now expects product revenues of KALYDECO to range between $740 million and $770 million.

VRTX closed Wednesday's trading at $114.38, down 3.14%. In after-hours, the stock was up 1.42% to $116.00.

ZIOPHARM Oncology Inc. (ZIOP) will present updated results from its Phase 1, multicenter, dose-escalation study and 20-mg expansion cohort of Ad-RTS-hIL-12 + orally administered Veledimex in patients with recurrent or progressive glioblastoma on June 5, 2017.

The primary endpoint of the study was safety and tolerability of Ad-RTS-hIL-12 + Veledimex, with secondary endpoints including overall survival.

ZIOP closed Wednesday's trading at $7.14, down 1.52%.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

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