Alnylam says heart drug succeeds in closely watched study

Positive results from a Phase 3 trial of vutrisiran in transthyretin amyloidosis cardiomyopathy come eight months after the FDA rejected another medicine Alnylam developed for the condition.

Updated June 24, 2024

Obesity drugs from Altimmune, Hengrui show potential; Lilly details Zepbound sleep apnea data

The American Diabetes Association’s annual meeting provided a forum for early trial results on several would-be weight loss drug competitors.

Novo fills in positive picture for preventive hemophilia drug

Phase 3 trial results should support an approval application for the drug, which analysts view as a competitor to Roche’s blockbuster medicine Hemlibra.

Duchenne approval exposes FDA rift over Sarepta gene therapy

Peter Marks’ decision to override the objections of agency staff and broaden use of Elevidys could have a “lasting impact” on gene therapy as well as the FDA, one analyst wrote.

Sarepta Duchenne gene therapy wins broader use from FDA

The approval makes Elevidys available to most Duchenne patients at least 4 years of age, despite mixed trial results that have led to skepticism about its effectiveness. 

Updated June 21, 2024

Twice-yearly shots of Gilead HIV drug effective in large prevention study

Researchers recommended Gilead end testing early as lenacapavir proved 100% effective in protecting cisgender women in the Phase 3 trial.

Jazz drug for tremor fails study; Radiopharma drug developer ITM swaps CEOs

A medicine Jazz acquired in 2019 missed the goal of a Phase 2 study. Elsewhere, Vanda rejected two takeover bids and Ashibio emerged from stealth with an antibody licensed from Gilead.

Obesity drug from Zealand shows potential in early trial

Interim study data suggest the Danish drugmaker can remain in the race to develop new kinds of weight loss drugs as doctors seek alternatives to the now dominant GLP-1s.

FDA lifts hold on PTC Huntington’s disease trial

The agency has lifted a partial trial suspension based on one-year data showing PTC’s pill suppressed a key protein associated with the disorder.

Amazon expands drug subscription program to Medicare members

RxPass, which fills prescriptions for generic drugs, is now available to more than 50 million Medicare members after Amazon brought it into compliance with the insurance program’s regulatory standards.

Merck wins FDA OK for vaccine rival to Pfizer’s pneumococcal shot

The new vaccine, which Merck will sell as Capvaxive, will compete with Pfizer’s blockbuster Prevnar franchise.

Lingering cholesterol can keep heart risk high. Marea Therapeutics has $190M to target it.

With a drug licensed from Novartis, the new biotech startup aims to reduce the ‘remnant’ cholesterol it says existing medicines can’t address. 

Intra-Cellular depression drug succeeds in second late-stage study

The results position the company to unlock what’s believed to be a multibillion-dollar sales opportunity for Caplyta, which is already used to treat schizophrenia and bipolar disorder. 

Roche partners with RNA editing biotech Ascidian

While the Swiss pharma recently pruned its neurology drug pipeline, it has shown interest in the possibilities of RNA-focused approaches like what Ascidian is exploring.

Sanofi taps Belharra for immune drug research; AstraZeneca’s new cancer drug falls short

Belharra will use its chemoproteomics technology to identify immune drugs for Sanofi. Elsewhere, Regenxbio mapped a regulatory path and Taysha shared updated results for its Rett treatment.

New data showcase promise, growing pains of CAR-T in autoimmune disease

One expert described trial results presented at EULAR in June as “unprecedented.” But reports of relapses in some patients drew questions about the therapies’ ultimate potential.

Takeda drug for rare types of epilepsy misses goal in late-stage trial

Called soticlestat, the drug had seemed promising in earlier studies for people with Dravet and Lennox-Gastaut syndrome, enough to persuade Takeda to take on full rights to the drug.

Abortion pill ruling offers measure of relief for FDA, biotech

A biotech executive called the Supreme Court’s decision a “very important win” for the industry, although new challenges to the FDA’s regulation of the drug could still be forthcoming.

Syncona melds two gene therapy biotechs for better shot at new nervous system treatments

The new company, Spur Therapeutics, is getting another $50 million from Syncona to support its broadened pipeline, which targets Parkinson’s disease as well as heart conditions.

AbbVie joins in latest gastrointestinal drug chase

Following Roche, Merck and others into a hot field, the Humira maker will pay $150 million to license a preclinical TL1A antibody for IBD.

Radiopharma drugmaker Telix pulls US IPO plans

The Australia-based company had proposed raising $202 million via an initial stock sale, but withdrew its filing, citing its shares’ recent price appreciation on Australia’s stock exchange. 

Takeda circles a leukemia drug; J.P. Morgan moves into biotech investing

The Japanese pharma paid $100 million to secure option rights to an Ascentage drug. Elsewhere, J.P. Morgan Private Capital closed its first fund and the FDA gave new COVID shot advice.

Pfizer setback brings questions for Duchenne gene therapy ahead of Sarepta decision

The failure of another trial adds to uncertainty around the benefits of the gene-based treatments, though industry analysts still expect Sarepta’s Elevidys’ to be unaffected.

Versant debuts biotech working on ‘anger management for the immune system’

Investor enthusiasm for precision immunology medicines is providing a tailwind for Santa Ana Therapeutics, a new startup backed by Versant, GV and a16z, among others.

Supreme Court preserves access to abortion pill in unanimous ruling

The high court said the plaintiffs’ “desire to make a drug less available to others” did not give them standing to challenge the FDA's expansion of mifepristone’s approval.