Bluebird, BMS win FDA nod for first BCMA-targeting CAR T therapy | 2021-03-29

The first BCMA-targeted CAR T therapy, idecabtagene vicleucel, cleared FDA approval for use in adults with multiple myeloma (MM) who have received four or more prior lines of therapy. Developed by partners Bluebird Bio Inc. and Bristol Myers Squibb Co., the drug, branded Abecma, is also the first CAR T drug indicated for MM. It is designed for use as a one-time infusion, with a recommended dose range of 300 to 460 x 106 CAR-positive T cells. The personalized therapy will be manufactured at BMS’ cellular manufacturing facility in Summit, N.J. Bluebird developed the lentiviral vector used in Abecma.

Antibody therapies show new benefits for both high and low-risk COVID-19 patients

Shares of Humanigen Inc. leapt 86% by midday on news that its monoclonal antibody, lenzilumab, improved the relative likelihood of survival without mechanical ventilation in hospitalized patients with COVID-19, setting the company up to submit an application for emergency use authorization in the U.S. “as soon as possible,” it said. Separately, a combination of two monoclonal antibodies, Eli Lily and Co.’s bamlanivimab and Vir Biotechnology Inc.’s VIR-7831, demonstrated a 70% relative reduction in persistently high SARS-CoV-2 viral load at day seven compared to placebo for low-risk adult patients with mild to moderate COVID-19, the companies reported.

Orphazyme’s arimoclomol misses endpoints in a phase II/III

Top-line data from Orphazyme A/S’ phase II/III trial of arimoclomol for treating inclusion body myositis, a muscle-wasting disease, failed to hit its primary and secondary endpoints. The data caused investors to pull back sharply as shares of Copenhagen-based Orphazyme (NASDAQ:ORPH) had dropped 30% by midday. The study’s primary endpoint was disease progression as measured by the inclusion body myositis functional rating scale. The randomized, placebo-controlled trial was conducted among 150 patients in North America and Europe who were randomized 1-to-1 to receive either 400 mg of arimoclomol citrate three times daily or placebo for up to 20 months. Orphazyme also has arimoclomol in a phase III study in treating amyotrophic lateral sclerosis and expects data before summertime.

Vasculitis race heats up as Chemocentryx adcom, Inflarx readout draw nearer

Inflarx NV in January submitted a special protocol assessment to the FDA for the phase III trial with vilobelimab against the skin condition hidradenitis suppurativa. Meanwhile, work goes on with the I.V.-infused targeter of complement activation product C5a in antineutrophil cytoplasmic antibody-associated vasculitis (AAV), where a U.S. phase II readout is expected in the middle of the year and a European study by the end of this year. In AAV, Inflarx, of Jena, Germany, is going up against Mountain View, Calif.-based Chemocentryx Inc., with avacopan, an oral inhibitor of the complement c5 inhibitor, which bears a PDUFA date of July 7. A meeting of the FDA’s Arthritis Advisor Committee is slated to evaluate avacopan on May 6.

Apnimed awaits new data as it closes a series B

Apnimed Inc., which just closed on a $25 million series B to help drive its lead program in treating obstructive sleep apnea (OSA) into a phase III registrational trial, is also waiting on data that will soon come from two ongoing studies in the indication. The Cambridge, Mass.-based company has oral, once-daily AD-109 in a phase II study for treating mild to severe OSA in late 2020 and initiated another phase II in January to gauge AD-109’s efficacy in treating mild to moderate OSA. The company said it expects the studies to yield top-line data sometime during the second quarter of 2021.

Also in the news

Adagene, Adamas, Aikido, Amarin, Amryt, Antibe, Aveo, Bayer, Biohaven, Biomea, Bluebird, Bristol Myers Squibb, Cardior, Celldex, Celltrion, Cerecor, Chemomab, Cohbar, Cynata, Daiichi, Destiny, Diffusion, Dyadic, Exagen, Foresee, Fortis, Glaxosmithkline, Gradalis, Gritstone, GT, Henlius, Humanigen, Hutchmed, Immune, Immunitybio, Ionis, Johnson & Johnson, Kazia, Lilly, Lineage Cell, Medsenic, Menarini, Mezzion, Millendo, Mirum, Mitolytix, Moderna, Motif, Myovant, Neurorx, Nippon Shinyaku, Nymox, Orphazyme, Oryzon, Paion, Pierre Fabre, Qihan, Rocket, Samsung Bioepis, Tempest, Tetra, TG, Therapeutic Solutions, Union, Uniqure, Valeo, Vir, Xenetic, Y-Biologics

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