Product Development

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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Kite’s success in DLBCL CAR-T rollout hinges on logistics, physician education, but uptake enthusiasm prevails - experts

Kite Pharma (NASDAQ:KITE) has delicate logistics and physician education hurdles to overcome if it is to be successful in launching axicabtagene ciloleucel (axi-cel) for aggressive non-Hodgkin’s lymphoma, experts said.

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AstraZeneca’s Lynparza AML/MDS side-effect rate not alarming, but concerns over future maintenance use remain - Special Report

AstraZeneca’s (LON:AZN) Lynparza (olaparib) is not exhibiting an alarming level of secondary malignancies, said experts based on an FDA adverse events (AEs) document this news service obtained through a Freedom of Information Act (FOIA) request.

The risk for secondary malignancies developing due to Lynparza treatment was a concern for the Advisory Committee that reviewed the drug’s maintenance treatment approval application.

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Longevity drugs face labelling and reimbursement quandaries with regulatory/payer reluctance, out-of-pocket payments possible – experts

Human longevity drugs may still face labelling and reimbursement challenges should they demonstrate evidence of extending a healthy life span, according to experts. As ageing is not considered a disease, regulators and payers may both take issue with labelling and paying for drugs for longevity. However, should generic drugs like metformin and rapamycin be repurposed for use as longevity agents, patients may just pay out-of-pocket given the drugs’ minimal cost, they added.

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Human longevity trials to focus on accumulation of age-related diseases rather than survival for extended life, QoL improvements – experts

Drug trials targeting human longevity will likely have endpoints focused on the accumulation of age-related diseases rather than survival to demonstrate an extended life span and improvements in health and quality of life (QoL), experts said. Trials designed around survival, however, would be challenging due to their expected high cost and likely long durations, they added.

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