BEIJING – China kickstarted a phase III trial Feb. 3 to determine whether patients with 2019-nCoV can be treated with Gilead Sciences Inc.’s NUC inhibitor, remdesivir, which was originally developed for Ebola, four days after a U.S. patient was said to have recovered by using the drug candidate. The study, expected to be completed on April 27, is a phase III randomized, double-blind, placebo-controlled multicenter study to evaluate the efficacy and safety of remdesivir in hospitalized adult patients with mild and moderate 2019-nCoV infections. It will enroll 270 patients and be carried out in the China-Japan Friendship Hospital in Beijing.
The U.S. Department of Health and Human Services has declared a public health emergency in the U.S. over the coronavirus for several reasons, including that asymptomatic patients can transmit the virus. However, Robert Redfield, director of the CDC, said the CDC’s test for the coronavirus returns inconsistent results on a given patient, thus creating a tremendous degree of uncertainty around a pathogen that has killed hundreds and roiled markets across the globe.
The FDA has issued two new approvals, one a cell-based pandemic influenza A (H5N1) vaccine and the other the first approval for treating peanut allergy. Seqirus Inc. received approval for Audenz to protect people ages 6 months and older against H5N1. It’s the first adjuvanted, cell-based influenza vaccine to be approved. Also, the FDA approved Aimmune Therapeutics Inc.’s Palforzia, the first treatment for patients with peanut allergy. Palforzia, an oral biologic immunotherapy, is designed to mitigate allergic reactions, including anaphylaxis, that come with an accidental exposure to peanut. Peanut allergy is one of the world’s most common food allergies. A recent survey from the nonprofit Allergy and Asthma Network showed 89% of providers would switch their patients from a peanut allergy immunotherapy to one with fewer side effects, while 46% prefer an oral product and 18% favor a powder mixed into food and eaten.
Biotech investors had every reason to feel bullish heading into the new decade. The sector had turned around in 2019 and was riding a wave of a very strong fourth quarter performance, with the BioWorld Biopharmaceutical Index closing up 14% for the year after being underwater from April through to September. Unfortunately, those great expectations were quickly erased during J.P. Morgan Healthcare conference week (Jan. 10 – Jan. 17), which turned out to be a very low-key affair absent of any blockbuster M&A revelations. As a result, confidence has now given way to concerns about the prospects for biopharmaceutical companies going forward, particularly as unfavorable political rhetoric on drug pricing will certainly be dialed up during this election year.
The National Institute of Allergy and Infectious Diseases has stopped a late-stage HIV vaccine study it sponsored after an interim review by the trial's independent data and safety monitoring board found the regimen did not prevent HIV. The phase IIb/III trial, one of several trials testing different approaches to preventing HIV, was testing two experimental vaccines, one supplied by Sanofi Pasteur, the vaccines division of Sanofi SA, and the other by Glaxosmithkline plc. Out of nearly 2,700 participants evaluated for the interim analysis, 129 vaccine recipients contracted HIV vs. 123 placebo recipients.
Shares of Insmed Inc. (NASDAQ:INSM) were trading midday at $27.42, up $6.88, or 33%, as Wall Street learned of positive top-line results from the global, randomized, double-blind placebo-controlled phase II study called Willow, testing INS-1007 once daily in adults with non-cystic fibrosis bronchiectasis. The compound, an oral, selective, reversible inhibitor of dipeptidyl peptidase 1, met its primary endpoint of time to first pulmonary exacerbation over the 24-week treatment period for both the 10-mg and 25-mg dosage groups compared to placebo (p=0.027, p=0.044, respectively). INS-1007 also resulted in a reduction in the frequency of pulmonary exacerbations, a key secondary endpoint, vs. placebo. Specifically, patients given the drug experienced a 36% reduction in the 10-mg arm (p=0.041) and a 25% reduction in the 25-mg arm (p=0.167). Change in concentration of active neutrophil elastase in sputum vs. placebo from baseline to the end of the treatment period was also statistically significant (p=0.034 for 10 mg, p=0.021 for 25 mg). Insmed plans to advance the candidate to phase III development.
PERTH, Australia – Australian stem cell therapy company Mesoblast Ltd. has filed the final module of its rolling BLA submission for its allogeneic mesenchymal precursor cell therapy, remestemcel-L, after it showed strong survival rates in children with acute steroid-refractory graft-vs.-host disease. Submitted Jan. 31, the final module covered quality control and manufacturing for the stem cell candidate, which is branded as Ryoncil. Mesoblast's allogeneic candidates are based on mesenchymal lineage cells collected from the bone marrow of healthy adult donors
HONG KONG – Aiming to increase the financial stability of the national health care insurance system, Japan has announced a list of 17 drugs for which prices will be reduced. The country’s Central Social Insurance Medical Council, an advisory group from the Ministry of Health, Labour and Welfare, approved the drug repricing system on Jan. 22. The repricing system will take effect from April 2020, with an average price reduction of 4.38%.
Affyxell, Agex, Aimmune, Anavex, Anticancer, Applied Biomath, Artelo, Astrazeneca, Avacta, Be Cool, Biocryst, Biomx, Bio-Thera, Can-Fite, Cellink, Citius, Cocrystal, Cormedix, Corvus, CTI, Daewoong, Denali, Enochian, Glaxosmithkline, Greenwich, Gritstone, GW, Inositec, Inovio, Insmed, Merck, Mesoblast, Moleculin, Nektar, Oncosec, Passage, Precigen, PTC, Regenerx, Resverlogix, Revolution, Sandoz, Scancell, Seqirus, Teva, Therapeuticsmd, United, Xortx, Zynerba