Sarepta gene therapy for Duchenne misses main goal of key study

Results from the EMBARK study, which were meant to confirm the approval of Sarepta's Elevidys, sent the company's shares down more than 40% Tuesday.

By Ned Pagliarulo • Updated Oct. 31, 2023

Novartis bet on kidney disease drug yields positive study data

The pharma paid more than $3 billion this summer to acquire Chinook Therapeutics and a therapy that just met its goal in a Phase 3 trial.

By Ned Pagliarulo • Oct. 30, 2023

Explore the Trendline➔

COVID-19 vaccines

The urgency of the pandemic’s first two years is giving way to questions of how to sustain public health responses and prepare for what could come next. In the U.S., health officials are urging booster shots and preparing for private sector distribution of vaccines.

By BioPharma Dive staff

Pfizer, BioNTech say combo flu, COVID shot met goal in study

The partners announced the trial had succeeded weeks after Moderna disclosed positive results from a study of its similar combination vaccine.

By Kristin Jensen • Oct. 26, 2023

Eisai says new data could support more convenient form of Alzheimer’s drug Leqembi

An under-the-skin injection of Leqembi performed about the same as the already marketed intravenous form, according to trial results presented Wednesday. A new approval application is expected by the end of March.

By Jacob Bell • Updated Oct. 26, 2023

Excision gives first look at gene editing therapy for HIV

The study results, while only from three participants, offer a glimpse at how one of the few in vivo CRISPR therapies in U.S. human testing is working. 

By Ned Pagliarulo • Oct. 25, 2023

Patient groups have become influential in drug R&D. Here’s a look at their impact.

“They have the money” and they’re using it to influence drug development, according to the director of the IQVIA Institute for Human Data Science.

By Kelly Bilodeau • Oct. 25, 2023

Seagen trial data impresses at ESMO, lifting Merck, Pfizer in the process

A combination of Seagen and Astellas’ antibody-drug conjugate Padcev with Merck’s Keytruda dramatically improved survival in first-line bladder cancer.

By Ned Pagliarulo • Oct. 23, 2023

Verve gets FDA green light to run base editing study in US

The trial, which is ongoing in the U.K. and New Zealand, has been on hold in the U.S. since late last year as the FDA sought more details on Verve’s in vivo treatment for heart disease.

By Ned Pagliarulo • Oct. 23, 2023

Why the evolution of patient advocacy organizations means better data for your rare disease trial

Rare disease drug development is a unique and challenging field that demands strategic development expertise and uniting of the various stakeholders working toward the same goal.

Oct. 23, 2023

5 steps to help increase the odds of clinical trial success

There are many common reasons clinical trials fail, including failure to demonstrate efficacy, safety concerns, issues with inclusion and exclusion criteria and patient recruitment. Follow these five steps to increase the odds of clinical trial success.

Oct. 16, 2023

Landing a CRO that’s an extension of your team

In an industry where the convergence of cutting-edge science and a human-centered approach is equally imperative, Worldwide Clinical Trials has found the sweet spot, delivering excellence in the clinical trial experience.

Oct. 16, 2023

PepGen cleared by FDA to begin study of muscular dystrophy drug

The company expects to deliver first results next year from the early-stage study of people with myotonic dystrophy type 1.

By Kristin Jensen • Oct. 12, 2023

Novo Nordisk’s Ozempic shows kidney benefit in large study

The finding from the study of its diabetes drug, which Novo didn’t detail, could help the company in its competition with rival drugmaker Eli Lilly.

By Jonathan Gardner • Oct. 11, 2023

Merck data show survival benefit to early Keytruda treatment

In the perioperative setting, Merck says its immunotherapy helps patients live longer. An approval decision is expected later this month.

By Jonathan Gardner • Oct. 10, 2023

Akero drug for NASH misses goal in mid-stage study

The biotech claimed the results still showed promise for its experimental fatty liver disease treatment, but shares sank sharply on the news.

By Ned Pagliarulo • Oct. 10, 2023

NIH funds research for three experimental ALS drugs

The funding, which is part of ACT for ALS, will support expanded access studies of drugs developed by Prilenia Therapeutics, Clene Nanomedicine and Rapa Therapeutics.

By Jacob Bell • Oct. 6, 2023

FDA panel finds KRAS drug data unreliable, in blow to Amgen

The negative panel vote likely means Amgen will need more data to support a full approval for its conditionally cleared lung cancer drug Lumakras.

By Ned Pagliarulo • Updated Oct. 6, 2023

Moderna claims positive results in early study for combo COVID, flu shot

The company is planning to start a Phase 3 trial of the vaccine this year, and is targeting a regulatory approval in 2025.

By Delilah Alvarado • Oct. 4, 2023

Novartis’ closely watched rare disease drug scores in kidney disorder

Called iptacopan, the experimental medicine has now scored positive results in three late-stage clinical trials, the latest in an uncommon kidney disorder known as IgA nephropathy.

By Jacob Bell • Oct. 2, 2023

Structure boosted by early data for experimental obesity drug

Shares in the biotech surged on Phase 1 trial results for an oral GLP-1 drug, reflecting investor enthusiasm for the weight loss medicines. 

By Ned Pagliarulo • Sept. 29, 2023

J&J cancer drug combo beats Tagrisso in closely watched trial

Interim results from a study called “Mariposa” found that a regimen of two J&J medicines improved progression-free survival versus AstraZeneca’s widely used therapy.

By Ned Pagliarulo • Sept. 28, 2023

BrainStorm’s ALS therapy not effective, FDA panel finds

Expert advisers to the agency voted 17-1 that Brainstorm's clinical trial data did not show the company's stem cell treatment to be effective for treating ALS. 

By Ned Pagliarulo • Updated Sept. 28, 2023

Immunovant data show potential for autoimmune disease drug

Much anticipated clinical trial data for Immunovant’s experimental FcRn inhibitor could catalyze business development decisions for its parent, Roivant Sciences.

By Ned Pagliarulo • Sept. 26, 2023

Novartis radiopharmaceutical drug succeeds in first-line gut cancer study

Novartis has made a large investment in targeted radiotherapies, sparking competition from fellow pharma companies and young biotechs.

By Jonathan Gardner • Sept. 25, 2023

AstraZeneca and Daiichi’s next cancer medicine scores in breast tumors

Results show that the medicine helped patients who progressed on earlier-line treatments live longer than those receiving chemotherapy without their disease getting worse, according to a Friday announcement.

By Jonathan Gardner • Sept. 22, 2023