FDA nomination in hand, Gottlieb prepares to disentangle himself from a long list of biopharma jobs and investments
Steve Burd (R), president and CEO, Safeway, Inc. and Scott Gottlieb (L), resident fellow at American Enterprise Institute participate in a Senate Health, Education, Labor and Pensions Committee hearing on Capitol Hill, June 11, 2009 in Washington, DC. (Credit: Mark Wilson/Getty Images)【阅读全文】
Vertex investors toast birth of the son of Orkambi
A double pivotal trial success with tezacaftor, a novel CFTR corrector, has given Vertex a neat follow-on to its blockbuster cystic fibrosis combination Orkambi, and should help it capture a greater market share of this respiratory disease.
Of course neither study compared the tezacaftor combo head to head against Orkambi, but on an across-trial basis efficacy looks marginally better, with safety – Orkambi’s main stumbling block – much improved. Moreover, the new combination bodes well for Vertex’s plans to cement its hold on cystic fibrosis with a triple combo.【阅读全文】
Roche prices Ocrevus for maximum disruption
Roche now has the first approved drug for both the relapsing and primary progressive forms of multiple sclerosis in the shape of Ocrevus. But the company is not taking any chances on price – at $65,000 it will cost less than existing MS therapies, giving the Swiss firm a good shot at penetrating the $20bn market.【阅读全文】
Novartis gets a speedy review for its CAR-T pioneer as FDA lines up a marketing decision
Novartis $NVS has pulled into the inside track at the FDA with its CAR-T CTL019, picking up a priority review from the agency that will land a marketing decision for relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia.
The pioneering application comes as we wait for Kite to make its move at the agency after being forced to wait for 6-month data on its rival CAR-T. Kite $KITE has promised to finish its rolling application by the end of Q1, now just days away. And a spokesperson for Kite tells me now that the biotech is on track to wrap the submission by Friday.【阅读全文】
CML treatment-free remission possible with nilotinib
The ENESTfreedom study of patients who stopped first-line nilotinib after achieving a sustained deep molecular response has shown that around half of chronic myeloid leukemia patients continue in remission.
This proportion is “clinically significant”, the investigators say, adding that, when considered alongside the results from the ENESTnd trial, which showed higher rates of deep responses and sustained deep responses with nilotinib versus imatinib, “more patients may become eligible to stop treatment and sustain remission following frontline nilotinib therapy than following imatinib therapy.”【阅读全文】
Immunovaccine Announces Positive Interim Clinical Data from Ovarian Cancer Study of DPX-Survivac in Combination with Epacadostat
Immunovaccine Inc. ("Immunovaccine" or the "Company") (TSX:IMV) (OTCQX:IMMVF), a clinical stage vaccine and immunotherapy company, today announced the first interim data analysis from its ongoing Phase 1b clinical study of its novel T-cell activating immuno-oncology candidate, DPX-Survivac, in combination with epacadostat and low-dose cyclophosphamide. The analysis included the results of blood tests, tumor biopsies and CT scans to assess safety, disease progression and T-cell response for the first four evaluable patients in the trial.【阅读全文】
New marker could break ALS drug development deadlock
Amyotrophic lateral sclerosis (ALS) doesn't have a cure, and the only drug approved to treat it extends life by just a few months. Now, a Mayo Clinic-led team has landed on a protein that could serve as a marker in the development of new therapies.
ALS affects motor neurons, which transmit messages from the brain or spinal cord to muscles or glands. As the disease progresses, patients lose the ability to move their muscles, which weaken and waste away, eventually leading to paralysis and death. While researchers have conducted more than 50 clinical trials in as many years, only the drug Riluzole, an incomplete solution, has earned FDA approval.【阅读全文】
Ritter lactose intolerance phase 2b/3 hits endpoint—but only after it excludes 20% of patients from the analysis
Ritter Pharmaceuticals has posted phase 2b/3 lactose intolerance data it claims set it up to run a confirmatory late-stage study and file for approval. But the trial missed its primary endpoint, with Ritter only able to make the figures hit statistical significance by removing 20% of patients from its analysis.【阅读全文】
Is Vertex's New CF Drug Unprecedented? Incremental? Expensive? Maybe All Three
Shares in Boston’s Vertex Pharmaceuticals are up 22% to $109 after the biotechnology company released data on an experimental combination pill to treat cystic fibrosis, a genetic disease that shortens patients' lives by affecting their lungs and digestive systems. That gives the biotech a market capitalization of $27 billion.【阅读全文】美中药源原创文章，转载注明出处并添加超链接，商业用途需经书面授权。
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