Product Development

Merck KGaA (ETR:MRK) has offered to pay for any additional disease-modifying therapies that multiple sclerosis (MS) patients may require during Mavenclad (cladribine) therapy, as part of ongoing reimbursement discussions with the UK’s National Institute for Health and Care Excellence (NICE), according to two sources familiar with the situation.

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Gilead Sciences’ (NASDAQ:GILD) early ASH 2017 abstract data from the Phase I/II ZUMA-6 study of Yescarta (axicabtagene ciloleucel)/Roche’s (VTX:ROG) Tecentriq (atezolizumab) in diffuse large B-cell lymphoma (DLBCL) drew concerns from experts about high toxicity rates, albeit with the caveat of the data’s earliness and small patient numbers.

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AstraZeneca’s (LON:AZN) benralizumab will likely have a dosing advantage over competitors in severe eosinophilic asthma, but may struggle to reach optimal market expectations, experts said. Convincing some physicians that it offers a superior efficacy profile for the majority of patients will be the major challenge, experts said.

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Multiple reported patient deaths through the FDA reporting system for patients taking Roche’s (VTX:ROG) Ocrevus (ocrelizumab) for multiple sclerosis has led to the company launching an investigation into the cases, said a source familiar with the situation. Two additional sources familiar with the drug said they also heard about the deaths, adding a number of questions regarding the nature of the cases remain unanswered.

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GlaxoSmithKline’s (LON:GSK)/ViiV Healthcare’s Phase III Tivicay (dolutegravir)/ Epivir (lamivudine) combination for HIV treatment-naïve patients versus triple therapy has experts unsure about its success, due to the comparator’s high bar and uncertainty associated with doublets when a triple has been approved.

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Asterias Biotherapeutics’ (NYSEAMERICAN:AST) AST-OPC1 initial cohort data from its Phase I/IIa study has experts cautiously excited for its potential to treat spinal cord injury in this and additional cohorts. Given the uncontrolled trial design, efficacy possibility can be hedged, they said; however, the data was strong enough to convince them of a real effect size.

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BeiGene’s (NASDAQ:BGNE) Phase III study comparing BGB-3111 against AbbVie (NYSE:ABBV)/Johnson & Johnson's (NYSE:JNJ) Imbruvica (ibrutinib) in Waldenström’s macroglobulinemia (WM) has enrolled 53 patients out of 167 as of 3 October with CRO Parexel (NASDAQ:PRXL), said a source briefed on the trial.

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Fortress Biotech’s (NASDAQ:FBIO) Phase II trials of CEVA101 are likely to see similar meaningful decreases in brain matter volume loss following traumatic brain injury (TBI) as observed in Phase I, experts said. Phase II's imaging-related endpoint is not necessarily a conventional TBI endpoint but is indeed clinically meaningful and is expected to correlate with global functional outcomes, they added.

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Reata Pharmaceuticals’ (NASDAQ:RETA) Phase II trial of RTA408 (omaveloxolone) in primary mitochondrial myopathy (MM) has triggered expert efficacy optimism based on the drug’s Phase II success in the related indication Friedreich’s ataxia (FA). Experts differed in their certainty of RTA408 achieving clinically significant efficacy and disagreed on the threshold of clinical benefit in MM.

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By: Alaric DeArment in New York and Fiona Barry in London

Ra Pharmaceuticals’ (NASDAQ:RARX) potential Phase III trial of C5 inhibitor RA101495 in paroxysmal nocturnal hemoglobinuria (PNH) patients should have a switching design in patients demonstrating suboptimal response to the standard order of care (SOC) to overcome patient recruitment difficulties, experts said.

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