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While tonlamarsen missed one of two co-primary endpoints, Kardigan says the drug has shown a clinically meaningful effect on blood pressure, supporting advancement into Phase 2b.
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Accumulating scientific evidence and industry interest from Eli Lilly, Altimmune and startup Baseline Therapeutics is driving further research on the therapeutic potential of GLP-1 receptor agonists in treating substance use disorders.
At the AD/PD annual meeting, Eisai presented real-world data suggesting Leqembi’s long-term safety and efficacy in people homozygous for APOE4, who were identified in trials as being at higher risk of brain bleeds while on the treatment. Alzheon, meanwhile, added further detail to trial results of its candidate in patients with the same genetic profile.
Overall, the top 16 largest pharmaceutical companies spent $159 billion on research and development in 2025, compared to $165 billion the year prior. Here’s where all that cash went at companies like Johnson & Johnson, Amgen and Pfizer.
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While requests by government officials for anonymity when speaking to the media are nothing new, the practice attracts more scrutiny when the Department for Health and Human Services has pledged a commitment to “radical transparency.”
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Former ACIP vice chair Robert Malone claimed that Andrew Nixon, spokesperson for the Department of Health and Human Services, “trashed” him with the media, adding that he resigned because “I do not like drama.”
While the accelerated approval unlocks only a small market opportunity for Rocket Pharmaceuticals, it will give the biotech a chance to prepare for future product launches, according to Jefferies.
The Excellergy acquisition will complement Novartis’ existing allergy profile, anchored by the IgE blocker Xolair that in February 2024 was approved to treat food allergies in children and adults.
While participants on a lower dose of Wave Life Sciences’ RNA therapy lost 5.3% total fat at the six-month mark, those receiving the higher dose saw a less than 1% drop at three months.
Beam is now in the strongest position of all companies advancing a genetic therapy for alpha-1 antitrypsin deficiency, according to analysts at William Blair.
Avlayah is the first Hunter syndrome therapy approved to address the condition’s neurologic complications, according to Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research.
Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong,” according to analysts at Jefferies, which projected over $1 billion in peak sales.
In this episode of Denatured, you’ll listen to Sergey Jakimov, managing partner at LongeVC and Artem Trotsyuk, operating partner, US, LongeVC. We speak about how developers in the longevity space should stop chasing aging as an abstract target and concentrate on specific mechanisms that can clearly tackle age-related conditions.
Besting Vertex Pharmaceuticals’ kidney disease asset wasn’t enough to impress Wall Street, which appears to be “getting hung up” with the broad population data in Maze Therapeutics’ trial, according to Mizuho.
This year’s catalysts in the space include a near-term FDA decision on Eli Lilly’s oral challenger to the new Wegovy pill. Looking further ahead, Novo Nordisk is expecting more clinical data for next-gen weight loss asset CagriSema, which recently lost a head-to-head battle with Lilly’s Zepbound.