Remdesivir, the investigational Gilead Sciences antiviral drug, looks more likely than ever to become the first treatment authorized by the FDA to treat patients with COVID-19.
National Institute of Allergy and Infectious Diseases (NIAID) head Anthony Fauci praised the drug after preliminary data released this week from a federally funded trial involving more than 1,000 patients. Those results showed that those treated with remdesivir recovered more rapidly than similar patients who received a placebo, meeting the trial’s main goal. According to NIAID, patients who received the Gilead (NASDAQ: GILD) drug recovered in a median of 11 days compared to those taking the placebo, whose median time to recovery was 15 days.
But questions remain about remdesivir’s efficacy and safety in light of conflicting results from the trials in which it is being evaluated, including a trial in China that didn’t show similar improvement after ending early due to low enrollment. More analyses are anticipated, though, once the full data set is released and researchers get to review it.
In non-COVID-19 biotech developments, startups continued a steady pace of venture capital-backed debuts, the FDA approved a new Parkinson’s disease drug, and a preclinical cancer cell therapies developer found an alternative way to go public. That, and more, in this week’s roundup.
—In addition to the topline NIAID results, Gilead reported this week that its Phase 3 trial of remdesivir, which tested a 10-day dose and five-day dose of the drug in patients with severe COVID-19 infections, showed little difference between the dosing regimens. That means more patients could be treated with the existing supply of the drug were it OK’d for use by regulators.
—A Phase 2/3 study testing Sanofi (NYSE: SNY) and Regeneron Pharmaceuticals (NASDAQ: REGN) drug Kevzara in COVID-19 patients classified as either “severe” or the more advanced “critical” will proceed only evaluating the drug in critical patients. The companies reported “negative trends” in the Phase 2 portion of the study testing the drug in the severe group.
—Moderna (NASDAQ: MRNA) this week formally asked the FDA for permission to start a Phase 2 test of its messenger RNA (mRNA) vaccine candidate for COVID-19. That study is expected to enroll 600 patients.
—BioNTech (NASDAQ: BNTX) dosed the first group of patients in a Phase 1 study testing its COVID-19 mRNA vaccine candidate. The Germany-based company is developing its vaccine under a partnership with Pfizer (NYSE: PFE).
NEW CASH, FRESH STARTS
—Cambridge, MA-based Rome Therapeutics spun out of the investment arm of Google parent Alphabet (NASDAQ: GOOGL) with $50 million and plans to use new discoveries around the repeatome—the repetitive sequences of nucleic acids that make up 60 percent of the non-coding portion of the human genome—to develop novel drugs.
—San Diego’s Erasca, a cancer drug development company that launched in 2018, hauled in a $200 million Series B round to advance its cancer programs.
—Six months after withdrawing its plans to go public, ADC Therapeutics resubmitted its IPO paperwork to support a regulatory filing and potential commercialization of an antibody-drug conjugate for diffuse large B-cell lymphoma.
—Lyra Therapeutics went public, raising $56 million. The Watertown, MA-based company is developing an implantable device that delivers a single dose that lasts months. Lyra priced its shares at $16 apiece, which was the high end of its projected price range.
—Avalyn Pharma of Seattle closed a $35.5 million Series B round to back Phase 2/3 tests of its aerosolized formulation of pirfenidone as a treatment for idiopathic pulmonary fibrosis.
—Milan, Italy-based Genespire, a spin-out of the San Raffaele-Telethon Institute for Gene Therapy founded in March, raised €16 million (about $17.5 million) in Series A financing from Sofinnova Partners to develop gene therapies for primary immunodeficiencies and metabolic genetic diseases.
—Dascena, an Oakland, CA-based startup that has been developing machine learning tools to predict sepsis in hospital patients, emerged from stealth with a $50 million Series B round.
—A Neurocrine Biosciences (NASDAQ: NBIX) drug opicapone (Ongentys) got the FDA’s approval as a treatment for Parkinson’s disease patients looking to increase “on” time between doses of levodopa, a standard treatment for the neurodegenerative condition.
—Axsome Therapeutics (NASDAQ: AXSM) reported that AXS-05 met the main goal of a Phase 2/3 study testing it as a treatment for agitation in Alzheimer’s disease patients. The drug is already being prepared for an FDA submission after succeeding last December in a separate study testing it in major depressive disorder.
—Partners Sanofi and Regeneron Pharmaceuticals (NASDAQ: REGN) announced that a Phase 3 test of cemiplimab (Libtayo) in advanced or metastatic non-small cell lung cancer was stopped early after an interim analysis. An independent committee concluded that patients treated with the drug showed a “highly significant increase” in overall survival.
—Avadel Pharmaceuticals (NASDAQ: AVDL) reported preliminary data showing that FT218, an experimental narcolepsy drug, met the main goals of a Phase 3 study. The Dublin, Ireland-based company said more details will be published in scientific journals and presented in upcoming medical conferences.
—One-year data from the second part of a pivotal test of risdiplam, an experimental Roche treatment for the muscle-wasting disease spinal muscular atrophy, showed patients achieving a “significant increase” in measures of motor skills. The drug has already been submitted to the FDA and a decision is expected by August 24.
—Pear Therapeutics released its investigational digital schizophrenia product, Pear-004, under emergency FDA guidance that expands access to prescription digital therapeutics during the COVID-19 pandemic. Last week, Akili Interactive made available its investigational therapeutic game for attention deficit hyperactivity disorder game under the same FDA guidance.
LET’S MAKE A DEAL
—ResTORbio (NASDAQ: TORC) and privately held preclinical biotech Adicet Bio agreed to merge. The combined company will use its resources to move ahead Adicet’s pipeline of cell therapies for cancer.
—Supernus Pharmaceuticals (NASDAQ: SUPN) is adding Parkinson’s disease to its portfolio via a $300 million deal to acquire four products from US WorldMeds.
—Vertex Pharmaceuticals (NASDAQ: VRTX) is committing $80 million in upfront payments and research milestones to kickstart a gene therapy partnership with Affinia Therapeutics. The agreement calls for Affinia to develop novel capsids—protein shells enclosing the virus used to deliver a gene therapy—that will be used in treatments Vertex is developing.
—Pfizer is paying Valneva $130 million up front to begin a partnership developing and potentially commercializing a vaccine for Lyme disease. France-based Valneva could earn $308 million in milestones tied to the progress of VLA15, which is currently in mid-stage testing.
PEOPLE ON THE MOVE
Kelly Martin was appointed Radius Health’s new president and CEO…Forma Therapeutics named David Cook as its chief scientific officer…Mirum Pharmaceuticals (NASDAQ: MIRM) added Peter Radovich as chief operating officer…Xilio Therapeutics tapped Martin Huber to serve as its chief medical officer…Mark Exley joined Imvax as its chief scientific officer…AMAG Pharmaceuticals (NASDAQ: AMAG) appointed Scott Myers as its new CEO…and Aurinia Pharmaceuticals (NASDAQ: AUPH) announced Joe Miller would become its new chief financial officer.
Frank Vinluan contributed to this report.
Image: iStock/Bertrand Blay