Clinical trials have historically hewn to a rigid format. A trial is designed, conducted according to that design, and then results are analyzed. Making changes in the midst of the study was a no-no.
In recent years, regulators have showed openness to clinical trial designs intended to make drug testing more efficient. We’ll get to see an example play out in tests for a coronavirus infection treatment. This week, the first US clinical trial to evaluate a potential treatment for the disease enrolled its first patient. It’s an adaptive clinical trial, meaning changes can be made along the way.
The study will test different investigational therapies against a placebo. If a therapy shows safety problems or doesn’t look like it works, investigators can stop work on that arm of the study without halting the entire trial and starting over again. If one of the drugs proves effective, it will become the control against which other investigational products will then be tested. Adaptive clinical trials are intended to more quickly find answers about how a drug works. And given the rapid global spread of coronavirus infection, finding those answers is a top public health priority.
In non-coronavirus news this week, an opioid maker unveiled a legal settlement, a drug in the NASH race gained ground, and one lucky biotech celebrated two FDA approvals. Let’s get to those stories and more in this week’s biotech roundup.
DRUG DECISIONS & DEVELOPMENTS
—Gilead Sciences (NASDAQ: GILD) announced its investigational antiviral drug remdesivir would be tested in US trials starting next month as a treatment for cases of COVID-19. And Moderna (NASDAQ: MRNA) sent a vaccine it developed, designed to prevent the disease, to the National Institute of Allergy and Infectious Diseases for use in a Phase 1 study.
—Lundbeck won FDA approval for eptinezumb, a migraine-prevention drug it added to its pipeline via the acquisition of Alder BioPharmaceuticals last year.
—The agency also approved rimegepant (Nurtec), a treatment for acute migraine developed bv Biohaven Pharmaceutical (NYSE: BHVN). It’s the New Haven, CT-based company’s first FDA-approved product.
—The FDA granted marketing authorization to a diagnostic developed by Asuragen to detect the genetic mutation indicative of Fragile X syndrome. The agency says the Austin, TX, company’s genetic test is the first such test for the disorder.
—By a 6 to 5 vote, an FDA advisory committee recommended approval of Eli Lilly (NYSE: LLY) drug ramucirumab (Cyramza) in combination with cancer drug erlotinib as a treatment for non-small cell lung cancer patients whose tumors have EGFR mutations.
—The March 25 target date for an FDA decision for fenfluramine (Fintepla), an experimental Zogenix (NASDAQ: ZGNX) treatment for the seizures caused by Dravet syndrome, has been pushed to June 25. The extension gives the agency more time to review additional data it requested from the Emeryville, CA, biotech.
—Johnson & Johnson (NYSE: JNJ) and Apple (NASDAQ: AAPL) will collaborate on a study to determine how earlier detection of atrial fibrillation impacts stroke in people age 65 or older using data from an iPhone app and the Apple Watch.
—A Menlo Therapeutics (NASDAQ: MNLO) drug failed a Phase 2 study in pruritus, or itching, of unknown origin. The company says the results won’t affect a forthcoming Phase 3 test in prurigo nodularis, nor will it affect its pending merger with Foamix Pharmaceuticals (NASDAQ: FOMX).
—In other itching news, Vanda Pharmaceuticals (NASDAQ: VNDA) drug tradipitant failed a Phase 3 test for pruritus associated with atopic dermatitis. The Washington, DC, company is waiting for data from another late-stage test before assessing its next steps.
—NGM Biopharmaceuticals (NASDAQ: NGM) shared preliminary data from a Phase 2 study of its experimental nonalcoholic steatohepatitis (NASH) drug, aldafermin, showing the treatment bested a placebo in reversing liver scarring and resolving the condition.
—Akili Interactive published results from a study of a digital therapeutic it designed to improve attention in children with attention-deficit/hyperactivity disorder showing improvements across a range of attention measures.
—Nektar Therapeutics (NASDAQ: NKTR) is winding down its etirinotecan pegol (Onzeald) program. The therapy had failed a late-stage breast cancer study in 2015, but appeared to help patients whose cancer had spread to the brain. On Thursday, Nektar reported that a Phase 3 test of the drug in that subgroup of patients showed that the treatment did not help patients live longer.
DOLLARS & DEALS
—Passage Bio (NASDAQ: PASG) sold 12 million shares priced at the high end of its expected range to raise $216 million in its initial public offering, funds it plans to use to advance its three lead gene therapy programs into human testing.
—Genentech agreed to pay Bicycle Therapeutics (NASDAQ: BCYC) $30 million up front in a drug discovery and development pact focused on cancer immunotherapy.
—Boston biotech startup Invetx raised $15 million in Series A financing to support development of a pipeline of novel biologic drugs for animal health.
—AstraZeneca (NYSE: AZN) agreed to sell global rights to opioid-induced constipation drug naloxegol (Movantik) to RedHill Biopharma (NASDAQ: RDHL) for $67.5 million. The deal excludes Europe, Canada, and Israel.
—MPM Capital has teamed up with the Dana-Farber Cancer Institute in a venture philanthropy initiative that has created two funds: a $100 million fund for early-stage biotechs developing new cancer therapies, and a $26 million fund to support early-stage cancer research at Dana-Farber.
—Redwood City, CA-based Karius raised $165 million in Series B financing to support development of its liquid biopsy technology, which is intended to diagnose infectious disease by detecting traces of microbial DNA in the blood.
—Mallinkrodt Pharmaceuticals (NYSE: MNK) announced a proposed $1.6 billion settlement that would resolve all opioid-related claims against the company and its subsidiaries. Under the deal, the company’s generic drugs unit—a maker of some generic opioids— would file for Chapter 11 bankruptcy.
—Biogen NASDAQ: BIIB) agreed to pay Sangamo Therapeutics (NASDAQ: SGMO) $350 million up front for rights to gene-regulation therapies associated with the buildup of the harmful tau protein. The deal covers experimental treatments for Alzheimer’s, Parkinson’s, and an undisclosed neuromuscular disease target.
PEOPLE ON THE MOVE
Skyhawk Therapeutics appointed Terry Connolly its chief operating officer… Max Colao joined Aurinia Pharmaceuticals (NASDAQ: AUPH) as chief commercial officer… Adverum Biotechnologies (NASDAQ: ADVM) promoted Angela Thedinga to chief technology officer… Nouscom named Marina Udier its permanent CEO and appointed Patricia Delaite its chief medical officer… XenoTherapeutics added Michael Yaremchuk as its first chief medical officer… and Halozyme (NASDAQ: HALO) said Elaine Sun would succeed Laurie Stelzer as chief financial officer.
Sarah de Crescenzo contributed to this report.
Image: iStock/Panorama Images