10 clinical trials to watch in the first half of 2024
A non-addictive pain pill faces its definitive test, while study results in ALS, a rare heart disease and lung cancer could have far-reaching implications.
Medicine
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As ALS research booms, one treatment center finds itself in the spotlight
Merit Cudkowicz has spent her career treating some of the most unforgiving illnesses. She’s watched Alzheimer’s disease steal memories and Huntington’s disease take control of muscles. But the cruelest may be ALS, or amyotrophic lateral sclerosis, a rare, fatal condition that leaves patients unable to walk, talk and breathe as their nerve cells rapidly break…
Sanofi scraps a top ADC prospect after study setback
Dive Brief: Sanofi will stop developing one of its top cancer drug prospects after the experimental treatment fell short in a late-stage study in advanced lung cancer, the company said Thursday. Trial monitors found the drug, dubbed tusamitamab ravtansine, missed both of its main study goals, failing to significantly delay tumor progression or extend patients’…
BioMarin strikes deal with Elliott, agrees to review business
Dive Brief: Weeks after installing a new CEO, BioMarin Pharmaceutical is overhauling oversight of the company at the board level. Facing pressure from activist investor Elliott Investment Management, BioMarin’s board named three new independent directors and set up a Strategic and Operating Review Committee to evaluate the company’s operations and priorities. Two of the new…
Ionis, Astrazeneca win FDA approval of competitor to Alnylam drug
Dive Brief: The Food and Drug Administration on Thursday approved a new medicine for a form of the rare disease transthyretin amyloidosis, clearing AstraZeneca and Ionis Pharmaceuticals’ Wainua for adults with the condition. The drug, formerly known as eplontersen, has been approved for use in transthyretin amyloidosis polyneuropathy, the variety of the disease that affects…
Bristol Myers to acquire brain drug developer Karuna for $14B
Bristol Myers Squibb has agreed to acquire Karuna Therapeutics for $14 billion, betting that the biotechnology company’s experimental schizophrenia drug will become a top-selling medicine. Per deal terms announced Friday, Bristol Myers will pay $330 a share for Karuna, a roughly 53% premium to its closing price on Dec. 21. Net of the cash that…
Sarepta tests FDA flexibility with bid to expand Duchenne gene therapy’s approval
Sarepta Therapeutics has asked the Food and Drug Administration to expand approval of its gene therapy for Duchenne muscular dystrophy, banking on the regulator’s flexibility in reviewing conflicting clinical trial data. The biotechnology company is requesting the FDA clear its treatment, called Elevidys, for people with Duchenne and a confirmed mutation in the relevant gene….
J&J bets bigger on MeiraGTx’s eye gene therapy
Dive Brief: Gene therapy developer MeiraGTx is selling its remaining financial interest in an eye-disease treatment partnered with Johnson & Johnson, announcing Thursday it is receiving $65 million up front and potentially another $65 million next year in exchange for foregoing future sales royalties. Per deal terms, MeiraGTx could receive up to $285 million more…
Clene says its ALS drug data is not yet enough for FDA
Dive Brief: Clene Nanomedicine on Thursday said the Food and Drug Administration doesn’t believe the company has gathered enough evidence to support an accelerated approval of its experimental drug for ALS, or amytrophic lateral sclerosis. A solution of gold nanoparticles, Clene’s drug is meant to protect neurons by promoting cell energy production. In ALS, it’s…
Argenx autoimmune drug study fails in blow to expansion hopes
Dive Brief: Argenx lost a quarter of its value Wednesday after reporting the second research setback in two months for its bid to expand use of its only approved drug. The latest study, called Address, showed no significant benefit for Argenx’s Vyvgart Hytrulo in people with two forms of an autoimmune skin disease called pemphigus….