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FDA Nod For VSTM, ANAB Breathes Easy, GEMP Trims Workforce By 33%

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Today's Daily Dose brings you news about AnaptysBio's (ANAB) trial results of Etokimab in adult patients with severe eosinophilic asthma; near-term catalyst of Audentes Therapeutics; lifting of partial clinical hold that was slapped on Epizyme's Tazemetostat clinical trials; workforce reduction in Gemphire and FDA approval of Verastem's COPIKTRA.

Read on...

Amgen's (AMGN) BLINCYTO has been granted marketing approval in Japan for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia.

BLINCYTO was developed in Japan by Amgen Astellas BioPharma K.K. (AABP), a joint venture between Amgen and Tokyo-based Astellas Pharma Inc.

BLINCYTO is now approved in 57 countries, including the U.S., all member countries in the European Union and the European Economic Area, Canada and Australia.

The drug netted sales of $175 million in the full year of 2017, and $109 million in the first half of 2018.

AMGN closed Monday's trading at $207.21, up 1.03%.

AnaptysBio Inc.'s (ANAB) ongoing single dose phase IIa clinical trial of Etokimab in adult patients with severe eosinophilic asthma has yielded positive results.

Patients administered with Etokimab rapidly improved their Forced Exhaled Volume In One Second (FEV1), which is a measure of lung function, with an 8 percent FEV1 improvement over placebo at Day 2. FEV1 improvement was sustained through Day 64, with an 11 percent increase over placebo, the Company added.

The study also showed that blood eosinophil reduction was sustained through the interim analysis period, with a 31 percent reduction at Day 2 and a 46 percent reduction at Day 64 over placebo, which was consistent with FEV1 improvement observed in this trial. Etokimab was generally well tolerated in all patients and no serious adverse events were reported as of this interim analysis.

A multi-dose phase IIb trial of Etokimab in eosinophilic asthma is expected to be initiated in 2019.

ANAB closed Monday's trading at $101.95, up 9.86%.

Audentes Therapeutics Inc. (BOLD) is scheduled to announce new interim data from ASPIRO, the Phase 1/2 clinical trial of AT132 in patients with X-linked Myotubular Myopathy at the 23rd International Congress of the World Muscle Society (WMS) on October 25, 2018.

X-linked Myotubular Myopathy is a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age.

BOLD closed Monday's trading at $37.65, unchanged from the previous day's close.

The FDA has lifted the partial clinical hold that had paused U.S.-based enrollment of new patients in Epizyme Inc.'s (EPZM) Tazemetostat clinical trials.

The partial clinical hold was slapped in April of this year, following a safety report of a pediatric patient who developed a secondary lymphoma in the company's phase I pediatric study of Tazemetostat.

Commenting on the FDA lifting the partial clinical hold, Robert Bazemore, president and chief executive officer of Epizyme, said, "This allows us to turn our full attention to our key priorities: preparing for our first NDA submission for Tazemetostat in epithelioid sarcoma and defining our registration path in follicular lymphoma."

The Tazemetostat clinical program continues to be on partial clinical hold in France and Germany, and Epizyme will now engage with regulators in France and Germany to resolve the issue.

EPZM closed Monday's trading at $10.25, unchanged from the previous day's close. In after-hours, the stock was up 1.46% to $10.40.

Gemphire Therapeutics Inc. (GEMP) is reducing its workforce by about 33% in order to reduce costs and conserve cash resources.

The move comes in the light of the previously announced request by the FDA for additional pre-clinical data required in order to schedule an End of phase II meeting for Gemcabene.

The Company is working with the FDA to release the partial hold on its investigational drug Gemcabene with respect to clinical trials of longer than six months in duration. A phase IIa trial of Gemcabene in familial partial lipodystrophy is underway.

As a result of the workforce reduction, the Company expects to record severance related charges totaling approximately $1.4 million, which includes one-time cash severance payments of $0.5 million, a non-cash charge of approximately $0.9 million related to the accelerated vesting of outstanding stock options for certain affected employees and $26,300 for continued health insurance coverage.

GEMP closed Monday's trading at $2.29, down 4.18%. In after-hours, the stock was down 14.85% to $1.95.

The FDA has lifted the clinical hold for Sarepta Therapeutics Inc.'s (SRPT) Duchenne muscular dystrophy micro-dystrophin gene therapy program.

On July 25, 2018, the clinical hold was placed on a phase 1/2a trial evaluating the Company's microdystrophin gene therapy candidate for Duchenne muscular dystrophy due to the presence of trace amounts of DNA fragment in the raw material used to make the drug.

SRPT closed Monday's trading at $153.10, up 3.73%.

Syndax Pharmaceuticals Inc. (SNDX) now expects progression free survival results from E2112, the ongoing phase III trial of Entinostat plus endocrine therapy in HR+, HER2- breast cancer, in the fourth quarter.

Earlier, it was expected that the Company might report the progression free survival results this month.

SNDX closed Monday's trading at $7.23, up 6.79%. In after-hours, the stock was down 17.01% to $6.00.

Tilray Inc. (TLRY) has successfully exported Tilray CBD 100 from Canada to Australia to distribute to critically ill children suffering from intractable epilepsy.

Tilray CBD 100 is an oral solution of concentrated cannabinoid extract. It has a target concentration of 100mg/ml CBD in a 25ml vial.

The product will be distributed to three hospitals in the state of Victoria via its wholly-owned subsidiary Tilray Australia New Zealand Pty Ltd.

TLRY closed Monday's trading at $99.50, down 19.11%. In after-hours, the stock was up 8.85% to $108.31.

The third patient in uniQure N.V.'s (QURE) phase IIb dose-confirmation study of AMT-061 for the treatment of patients with severe and moderately severe hemophilia B has been treated.

The topline data from the study are expected to be available by the end of the year.

QURE closed Monday's trading at $40.29, up 4.76%.

The FDA has approved Verastem Inc.'s (VSTM) COPIKTRA for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies.

COPIKTRA (duvelisib) also received accelerated approval for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies.

The drug will be available in the U.S. market immediately, the Company noted.

VSTM closed Monday's trading at $8.89, up 2.77%. In after-hours, the stock was down 1.96% to $8.76.

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