Clinical Trials

CRISPR touts new results as fresh questions surround 'off-the-shelf' CAR-T

The biotech's lymphoma treatment initially seems as potent as earlier cell therapies, but its effects waned, adding to doubts about off-the-shelf CAR-T.

By Jonathan Gardner • UPDATED: Oct. 13, 2021 at 9:55 a.m.

Sarepta outlines final push for Duchenne gene therapy

The biotech still believes a speedy approval filing for the closely watched treatment is possible, but expects to have to wait for the results of the recently launched Phase 3 trial, CEO Doug Ingram said on a conference call. 

By Ben Fidler • Oct. 11, 2021

Explore the Trendline➔

Trendline

Inside the race to bring coronavirus vaccines to the world

More than a dozen shots are cleared for use in some fashion around the world. Producing enough supply and addressing the spread of coronavirus variants are the next challenges. 

By BioPharma Dive staff

Sponsored by Clinical Ink

eCOA clinical trials: A simple, cost-effective approach to study build

This new data capture model gives clinical trial sponsors control over study build. 

Relay's targeted cancer drug could be safer than its competitors. Is it more effective?

Initial study results for Relay's experimental treatment offer some support for the biotech's protein motion technology, but raise some questions, too. 

By Ned Pagliarulo • Oct. 08, 2021

Allogene cell therapy trials halted by FDA after unexpected safety finding

Researchers found evidence of a "chromosomal abnormality" in one patient treated with Allogene's CAR-T cell therapy, spurring the clinical hold and an investigation.

By Ben Fidler • UPDATED: Oct. 8, 2021 at 9:09 a.m.

Takeda halts studies of touted sleep disorder drug over safety concern

The drugmaker suspended dosing in two mid-stage studies of its experimental narcolepsy treatment after a "safety signal" emerged.

By Ned Pagliarulo • Oct. 06, 2021

Collins to step down as NIH head in transition for research agency

After 12 years in the top job, the veteran director says a new scientist should assume leadership of the world's largest biomedical research institution.

By Jonathan Gardner • Oct. 05, 2021

J&J heats up RSV race with new vaccine data

The company's candidate is one of two to enter late-stage testing for a respiratory virus that causes hundreds of thousands of hospitalizations a year.

By Jonathan Gardner • Oct. 04, 2021

Sarepta embarks on late-stage clinical trial of Duchenne gene therapy

Initiation of the Phase 3 trial is an important milestone for the biotech after earlier setbacks, as well as for patients with the inherited muscle disease.

By Shoshana Dubnow • Oct. 04, 2021

Merck says antiviral pill effective against COVID-19, lifting hopes for first oral drug

The drugmaker, along with partner Ridgeback Biotherapeutics, plan to ask the FDA for emergency authorization "as soon as possible" following results so striking that testing was stopped early. 

By Ben Fidler • Oct. 01, 2021

After long wait, Editas reveals first data for CRISPR gene editing treatment

Early study results offer some hopeful signs but were difficult to interpret. Shares in the biotech slid by nearly 20% in response on Wednesday. 

By Ned Pagliarulo • Sept. 29, 2021

Sanofi claims positive early data for mRNA COVID shot, but pivots to flu instead

With Pfizer's and Moderna's vaccines broadly used, Sanofi says it will focus on its protein-based coronavirus candidate. The French pharma sees more promise for its mRNA work in influenza.

By Jonathan Gardner • Sept. 28, 2021

As FDA scrutinizes immunotherapy approvals, Merck says Keytruda extends survival in liver cancer trial

Phase 3 results showed Merck's drug improved overall survival versus placebo, which should lessen pressure for a market withdrawal in the indication.

By Jonathan Gardner • Sept. 27, 2021

Chinese drugmaker Clover says vaccine prevents COVID-19 in large trial

Positive results from Clover's Phase 3 study could mean another coronavirus vaccine option for the many countries still grappling with short supplies.

By Jonathan Gardner • Sept. 22, 2021

Pfizer says coronavirus vaccine is safe and spurs immune response in children

The results position Pfizer and BioNTech to seek clearance in children as young as 5 years old. But the companies haven't yet shared data on a rare heart inflammation associated with their shot that's a concern for regulators.

By Jonathan Gardner • Sept. 20, 2021

ESMO21: Mirati sets new KRAS bar, AstraZeneca's breast cancer data and Keytruda challengers line up

Mirati's KRAS-blocking drug looks to have an edge over Amgen's, while AstraZeneca and Daiichi reported impressive data for their drug Enhertu.

By Ned Pagliarulo • UPDATED: Sept. 21, 2021 at 10:59 p.m.

Biogen says experimental pain drug helps neuropathy patients in mid-stage trial

With Aduhelm approved but struggling to gain traction, Biogen is talking up its pipeline. Mixed trial results, however, may make it challenging to design a larger study that can persuade regulators to approve the pain drug.

By Jonathan Gardner • Sept. 17, 2021

Amgen to advance KRAS drug combination after improved results in colon cancer

Combining Amen's Lumakras with its drug Vectibix appeared to work better in treating colorectal cancer than Lumakras alone, spurring the biotech to unveil plans for a new Phase 3 trial. 

By Ned Pagliarulo • Sept. 16, 2021

New data from Israel, Pfizer show potent effect from third coronavirus shot

The much-anticipated data arrive ahead of an important FDA advisory meeting to discuss authorization of a booster dose for Pfizer's coronavirus vaccine.

By Jonathan Gardner • Sept. 15, 2021

Apellis eye drug succeeds in one key study, but falls short in its twin

Highly anticipated study results showed divergent outcomes for the biotech's therapy in treating a form of vision loss known as geographic atrophy. Even so, Apellis plans to soon ask for FDA approval.

By Ned Pagliarulo • UPDATED: Sept. 10, 2021 at 9:36 a.m.

Sanofi drug for rare skin disease, acquired in buyout, fails in late-stage trial

The French drugmaker bought Principia Biopharma last year for two therapies that block a protein called BTK. The second targets a much bigger disorder, multiple sclerosis.

By Jonathan Gardner • Sept. 09, 2021

Autism drug fails in late-stage trials, Servier says

The French drugmaker and its biotech partner Neurochlore agreed to end two Phase 3 studies early after finding no signs that treatment was effective. 

By Kristin Jensen • Sept. 08, 2021

Pfizer, Merck launch large new trials of oral COVID-19 drugs

Both companies are racing to prove their oral antivirals as treatments for COVID-19. Key clinical results could come later this year.

By Kristin Jensen • Sept. 01, 2021

Hint of benefit in Alzheimer's drug study fuel stock surge for Swiss biotech

A once-failed drug being developed by AC Immune and Roche appeared to slow cognitive decline in a small trial. But other study goals were missed, muddying the seemingly positive result. 

By Ned Pagliarulo • Aug. 31, 2021

Pfizer drug bests Sanofi, Regeneron's Dupixent in head-to-head study

Abrocitinib, a JAK inhibitor Pfizer's developing, outperformed Dupixent in treating moderate or severe eczema. Hanging over the study's success, however, is an ongoing review by the FDA of the drug's class. 

By Ned Pagliarulo • Aug. 30, 2021