Product Development

Genkyotex’s Phase II GKT831 trial has sound mechanism rationale though not specific to PBC; GGT primary endpoint lacks clinical relevance, experts say

Genkyotex’s (EPA:GKTX) Phase II trial investigating GKT831 for primary biliary cholangitis (PBC) has a positive mechanism rationale for efficacy as its antioxidant and anti-inflammatory effect may have an impact on fibrosis, experts said. However, this comes with the caveat that its targets NOX1/NOX4 are not directly linked to the disease, drawing some expert caution.

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J&J, Pfizer biosimilar payer policy case could survive motion to dismiss but proving anticompetitiveness remains challenging, experts say

Pfizer’s (NYSE:PFE) biosimilar antitrust claim against Johnson & Johnson (NYSE:JNJ) could be strong enough to reach the discovery stage of litigation but will survive only if the latter company's exclusionary tactics for Remicade (infliximab), beyond just lowered prices and bundling deals, are proven, said experts.

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Audentes Therapeutics’ AT132 Phase I/II ASPIRO trial in XLMTM will continue with low dose for next drug treatment cohort based on safety, efficacy signals, source says

Audentes Therapeutics (NASDAQ:BOLD) does not plan to increase the dose for its next drug treatment cohort in its Phase I/II ASPIRO trial of gene therapy AT132 against X-linked myotubular myopathy (XLMTM) despite the company saying otherwise, said a source familiar. Rather, due in part to a potentially worrisome safety signal, the company intends to keep dosing patients at the same level as announced in recent data, the source said.

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Blueprint’s BLU-285 mastocytosis ASH data promising, but KIT mutation-targeting knife may cut both ways – experts

Blueprint Medicines' (NASDAQ:BPMC) Phase I data for avapritinib in advanced systemic mastocytosis (SM) is praiseworthy but its advantageous target specificity could also engender resistance mutations down the line, experts said on the sidelines of the 2017 ASH meeting.

While the drug is seen as competitive to Novartis’ (VTX:NOVN) approved Rydapt (midostaurin), avapritinib’s greater specificity to KIT mutations – the driver mutation in SM – could also be a disadvantage with the resistance mutations having the potential to render the drug ineffective, experts said.

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Unum plans additional cohort after cytokine release syndrome, neurotoxicity reports in Phase I lymphoma cell therapy study – source

Cytokine release syndrome (CRS) and neurotoxicity incidents have prompted Unum Therapeutics to evaluate a new dosing cohort in its Phase I study of cell therapy ACTR087 in relapsed/refractory non-Hodgkin’s lymphoma, said a source briefed on the situation.

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Seres’ SER-287 elicits overall uncertainty for microbiome change for ulcerative colitis, maintenance use a possible prospect but with caveats – experts

Seres Therapeutics’ (NASDAQ:MCRB) Phase Ib SER-287 has drawn overall skeptical reviews from experts about its potential to change the microbiome of mild-to-moderate ulcerative colitis (UC) patients for clinical benefit. But any signal of microbial change in the trial’s patients would be welcome to help confirm SER-287 mechanism, they added.

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Roche’s (VTX:ROG) Ocrevus (ocrelizumab) having additional reported cases of progressive multifocal leukoencephalopathy (PML) has not raised neurologist alarms despite analyst concern. Most physicians, familiar with treating PML, said the events should not negatively affect the medication’s uptake in multiple sclerosis (MS), despite contrary market opinions.

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Biogen’s (NASDAQ:BIIB) recently announced Phase II trial design for opicinumab in relapsing remitting multiple sclerosis (RRMS) has experts optimistic that patient inclusion criteria could allow a positive trial outcome. That said, the company’s decision to allow any disease-modifying therapy (DMT) in the trial had some experts noting overall data analysis would be challenging.

Some experts also noted lingering doubts on positive results considering previous mixed trial results and the drug’s uncertain mechanism of action.

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Advanced Accelerator Applications’ (NASDAQ:AAAP) Lutathera (177Lu-DOTATATE) for gastroenteropancreatic neuroendocrine tumours (GEP-NET) could face further FDA approval delays if concerns about its safety profile persist due to its radiolabeled molecule, experts said. However, they were overall optimistic Lutathera would be eventually approved due to its encouraging efficacy data.

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Kala Pharmaceuticals’ (NASDAQ:KALA) KPI-121 (loteprednol etabonate) for acute symptoms of dry eye will have difficulty achieving its Phase III co-primary endpoint of significant ocular discomfort reduction as the drug demonstrated a failure in Phase II, said experts.

The trial design between Phase II and Phase III are very similar, with the only discernable difference being duration, and could see a replication in the failure. However, they added, the other co-primary endpoint of bulbar conjunctival hyperemia reduction has a good chance of repeating Phase II success.

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