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BLPH's INOPulse Misses Rhythm, ONCE Bleeds, FDA Panel Nod For INSM

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Today's Daily Dose brings you news about FDA panel recommendation of Insmed's inhaled antibiotic; Bellerophon's disappointing INOpulse study results; mixed study results of Spark's investigational gene therapy SPK-8011 for hemophilia A, and FDA approval of Vertex Pharma's cystic fibrosis drug.

Read on...

An FDA panel has voted 12 to 2 recommending approval of Insmed Inc.'s (INSM) ALIS for adults with nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC) who have limited or no treatment options.

The committee also voted in favor of the surrogate endpoint of sputum culture conversion used in the Phase 3 CONVERT study being reasonably likely to predict clinical benefit.

If approved, ALIS will be the first and only therapy in the U.S. specifically indicated for the treatment of patients with NTM lung disease caused by MAC.

The FDA decision on ALIS is expected to be announced on September 28, 2018.

Nontuberculous Mycobacterial (NTM) lung disease is a rare and serious disorder, with patients experiencing a multitude of symptoms such as fever, weight loss, cough, lack of appetite, night sweats, blood in the sputum, and fatigue.

INSM closed Tuesday's trading at $24.37, down 6.81%.

Shares of Bellerophon Therapeutics Inc. (BLPH) plunged more than 68% on Tuesday, following the Data Monitoring Committee's recommendation to stop the Company's Phase 3 INOvation-1 study evaluating INOpulse for the treatment of pulmonary arterial hypertension.

Based on the pre-specified interim analysis from the first 75 enrolled subjects in the INOvation-1 study, the Data Monitoring Committee has concluded that the overall change in 6 minute walk distance, the primary endpoint of the trial, is insufficient to support the continuation of the study.

The Company will determine the next steps in its PAH program over the next few weeks after further analyzing the full data set of the trial.

BLPH closed Tuesday's trading at $0.71, down 68.44%.

Shares of Spark Therapeutics Inc. (ONCE) took a hit as investors drove down the price on safety concerns over the Company's investigational gene therapy SPK-8011 for hemophilia A, currently being evaluated in a phase 1/2 clinical trial, which has 12 participants.

Across all participants, a single administration of investigational SPK-8011 at one of the three doses, say 5x1011 vector genomes (vg)/kg body weight, or 1x1012 vg/kg or 2x1012 vg/kg, resulted in a 97-percent reduction in annualized bleeding rate (ABR) and a 97-percent reduction in annualized infusion rate (AIR). There was also evidence of a dose-dependent increase in mean FVIII activity levels across the three dose cohorts.

However, two participants in the 2x1012 vg/kg cohort had an immune response that caused their FVIII levels to decline to less than 5 percent.

"Both participants have moved from prophylactic to on-demand treatment and have seen meaningful reductions in their bleeding and infusion rates. One of these participants did not rapidly respond to oral steroids and he elected to be admitted to the hospital to receive two intravenous (IV) methylprednisolone infusions rather than have the infusions on an outpatient basis. The event was subsequently resolved. The admission to hospital for these infusions met the criteria for a serious adverse event (SAE)", the Company noted.

Seven of the 12 participants received a tapering course of oral steroids in response to an alanine aminotransferase (ALT) elevation above patient baseline, declining FVIII levels and/or positive IFN-g enzyme-linked immunospots (ELISPOTs). For these seven participants, steroids led to normalization of ALT and ELISPOTs. For all but the two above mentioned 2x1012 vg/kg cohort participants, oral steroids led to stabilization of target FVIII level, the Company added.

Spark Therapeutics intends to initiate a Phase 3 run-in study in the fourth quarter of 2018.

ONCE closed Tuesday's trading at $56.01, down 27.83%.

The FDA has approved Vertex Pharmaceuticals Inc.'s (VRTX) ORKAMBI to include use in children ages 2 through 5 years with cystic fibrosis who have two copies of the F508del-CFTR mutation.

ORKAMBI oral granules are available in two dosage strengths (lumacaftor 100mg/ivacaftor 125mg and lumacaftor 150mg/ivacaftor 188mg) for weight-based dosing. ORKAMBI oral granules should be available for fulfillment within 2 to 4 weeks, according to the Company.

ORKAMBI was approved in the U.S. and EU in 2015 for the treatment of cystic fibrosis in patients ages 6 and older who have two copies of the F508del-CFTR mutation.

Orkambi is a blockbuster drug, and it generated annual revenue of $1.32 billion last year, up from $979.6 million in 2016. The drug had sales of $665 million in the first half of 2018.

The decision of the European Medicines Agency for the additional approval of ORKAMBI in children ages 2 through 5 years is anticipated in the first half of 2019.

VRTX closed Tuesday's trading at $177.51, up 1.49%.

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