Product Development

Roche’s emicizumab has largely uncertain haemophilia A approval forecasts after undetailed Phase III safety read outs – experts

Despite positive efficacy results, Roche’s (VTX:ROG) ACE910’s (emicizumab) Phase III outcomes lack sufficient detail to gauge the drug’s safety profile and subsequent approval prospects in haemophilia A, three experts agreed.

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Mylan’s Advair generic approval hinges on solidity in meeting FDA guidelines; true equivalence a hurdle – experts

Mylan's (NASDAQ: MYL) bid for approval of MGR001—generic of GlaxoSmithKline’s (LON:GSK) Advair (fluticasone/salmeterol) inhaler—is within reach but dependent on the data-package’s precision to specific FDA guidelines introduced in 2013, said experts.

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Stealth BioTherapeutics’ planned Phase III trial divides physicians over potential elamipretide protocol length, functional endpoints desired

Stealth BioTherapeutics’ planned Phase III trial of elamipretide in genetically-confirmed mitochondrial myopathy has drawn physician debate over whether Stealth’s proposed 4-week protocol is long enough to capture relevant efficacy and safety data.

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Gilead’s bictegravir faces tough HIV market but TAF combo attractive enough to drive use over Viiv’s Tivicay – experts

Gilead Sciences’ (NASDAQ:GILD) bictegravir faces tough HIV space dominated by Viiv’s Tivicay (dolutegravir), but a fixed dose combination comprising tenofovir alafenamide (TAF) could give it an edge, said experts.

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Two patients drop out of Catalyst-financed Phase II/III trial of Firdapse in MuSK-myasthenia gravis, results likely at May conference – source

Two patients out of 10 have dropped out of Catalyst Pharmaceuticals’ (NASDAQ:CPRX)-financed, investigator-sponsored Phase II/III trial of Firdapse (amifampridine phosphate; 3,4-DAP) in the myasthenia gravis MuSK protein-seropositive subgroup (MuSK-MG), a source familiar said.

The source said the 10th patient was enrolled and dosed in early December, and final read-outs from this cohort are expected this month. After unblinding, data analysis will take one month, the source said. Catalyst stated in a company filing top-line results will be published in 1H17.

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Seattle Genetics’ vadastuximab may be steered toward non-transplant AML if hepatotoxicity is drug-related – experts

Seattle Genetics’ (NASDAQ:SGEN) FDA hold on its vadastuximab talirine studies left experts hesitant about using the drug in the allogeneic stem cell transplant (alloSCT) setting of acute myeloid leukemia (AML) if the toxicity proves drug-related.

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Lexicon’s telotristat for carcinoid syndrome incites FDA approval optimism, Phase III endpoint and depression fears draw some pause – experts

Lexicon Pharmaceuticals’ (NASDAQ:LXRX) LX1032 (telotristat ethyl) for carcinoid syndrome has a positive FDA approval shot due to its encouraging Phase III data, which shows statistical significant improvement in reducing bowel movements (BMs), experts said. The oral therapy’s novel mechanism, which directly targets the cause of diarrhea, is also a cause for optimism, they added.

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Ipsen has enrolled less than 30% of patients in Phase III Somatuline trial for lung NETs, CRO Icon utilised – investigator

Ipsen’s (EPA:IPN) Phase III SPINET trial investigating Somatuline Autogel/Depot (lanreotide) for lung neuroendocrine tumours (NETs) has enrolled under 30% of its 216 subjects, a trial investigator said. The study, which started enrolling subjects a year ago, is enrolling slower than projected in all open sites due to the low incident rate of lung NET patients, he explained.

Ipsen’s financial reports nor press releases do not have any enrolment information on the SPINET trial.

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Advanced Accelerator switches to CRO Brightech from Accelovance to tackle FDA CRL data query; confident on 2017 approval – CEO

Advanced Accelerator Application (NASDAQ:AAAP) has switched to CRO Brightech to handle data management for Lutathera’s (lutetium Lu177 dotatate) approval package for gastroenteropancreatic neuroendocrine tumors (GEP-NETs), CEO Stefano Buono told this news service on the sidelines of the JP Morgan conference in San Francisco today (January 11).

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Capricor’s Phase I/II CAP-1002’s uncertain efficacy forecasts in DMD cardiomyopathy prompts future trial discussion – experts

Capricor Therapeutics’ (NASDAQ:CAPR) CAP-1002’s cardiac stem cell mechanism of action has unclear efficacy projections in its Phase I/II cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) trial, experts said. Given the lack of certainty regarding the therapy’s efficacy, experts discussed potential future trial designs that might elucidate an effect.

Some experts noted that cardiovascular (CV) cell therapies have previously struggled to demonstrate efficacy, with limited benefits largely derived from paracrine effects rather than myocardial regeneration.

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