EC expands approval of Amgen's Blincyto; FDA gives uniQure green signal to begin Huntington's study in humans, Cytokinetics feedback on SMA trial
→ The first therapy to win approval for cancer patients in remission with minimal residual disease in Europe is Amgen‘s $AMGN BiTE immunotherapy, Blincyto. The drug’s EC approval has been expanded to include certain patients with acute lymphoblastic leukemia in first or second complete remission with minimal residual disease (MRD) – the presence of cancer cells that remain detectable despite remission – greater than or equal to 0.1%. The EC approval is based on data from the Phase II BLAST study, which demonstrated the drug led to no detectable MRD, in 78 percent of patients within one treatment cycle. The EC nod follows a similar FDA approval last March.
→ Despite stretched resources at the FDA, the agency has been busy. On Tuesday it cleared an application by gene therapy developer uniQure $QURE to test its experimental drug, AMT-130, in Huntington’s disease patients. This puts the Dutch company on track to dose patients in a Phase I/II study in the second half of 2019. “We believe future results from AMT-130 will serve as important proof of concept for uniQure’s CNS programs, and more specifically, the miQURE technology. For now, the majority of our focus remains on the ongoing Phase 3 trial for AMT-061, for hemophilia B,” Cantor Fitzgerald’s Elemer Piros wrote in a note.
Separately, the agency also provided Cytokinetics $CYTK feedback that the six minute walk test is an acceptable primary efficacy endpoint for a potential registration program for the company’s experimental SMA drug, reldesemtiv, in patients who have maintained ambulatory function. “Next up is the determination of next development steps in conjunction with global partner Astellas, which is now also to include a Phase 1 in healthy volunteers (slated to begin in 1Q19) to test the effect of higher doses on plasma concentrations,” HC Wainwright’s Joseph Pantginis wrote in a note.
→ Peter Thiel has joined a slate of American and Chinese investors for Immunochina’s $20.4 million Series C as the company extends its CAR-T ambitions beyond China. The financing is expected to fund an expansion of manufacturing capabilities as well as trials for its lead product for B-cell acute lymphoblastic leukemia and non-Hodgkin’s lymphoma. Shougang Fund, Qingzhe Capital, China Resources Innovative Equity Investment Fund and Sherpa Venture Capital are also in for the ride.
→ An interim snapshot of early-stage data evaluating its non-opioid painkiller, resiniferatoxin, is compelling enough to start planning pivotal trials this year, according to Sorrento Therapeutics $SRNE. Data on 30 patients with pain due to osteoarthritis in the knee were examined from the ongoing study, and the results suggested pain reduction was observed “as early as the day following drug administration and sustained over time”, and patients will be monitored for 12 months following drug administration, after which a dose will be selected for further development.
→ As it continues a solo — and still low-profile — effort to prove its cell therapy can serve as a universal bone marrow donor source, Gamida Cell $GMDA will get a hand from the service provider Be The Match BioTherapies in both executing late-stage development programs and reaching patients if the treatment becomes available. The collaboration gives Israel-based Gamida Cell access to Be The Match BioTherapies’ donor registry as well as case management, logistics tools and relationships with cord blood banks around the world.