Clinical Trials

Blueprint drug succeeds in rare disease study important to its market prospects

Trial results position the company to seek approval for indolent systemic mastocytosis, which executives have painted as a “multibillion dollar” market opportunity. Even so, shares fell by double digits in trading Wednesday.

By Ben Fidler • Updated 3 hours ago

Ventyx touts early data for potential rival to Bristol Myers psoriasis drug

The biotech plans to start Phase 2 studies for a drug it believes may more effectively hit its target than Bristol Myers’ deucravacitinib, a skin disease treatment regulators could approve next month. 

By Jonathan Gardner • Aug. 16, 2022

Explore the Trendline➔

Cell therapy

Six CAR-T cell therapies are now approved in the U.S., and their continued emergence has helped fueled further research into next-generation approaches.

By BioPharma Dive staff

Novartis bid to repurpose rare disease drug for cancer falls short in third trial

A study testing Novartis’ canakinumab as an adjuvant lung cancer treatment missed its main goal, closing off an opportunity for an expanded approval.

By Ned Pagliarulo • Aug. 15, 2022

Bristol Myers claims success in study testing earlier CAR-T use in multiple myeloma

Abecma, which 2Seventy bio helps develop and sell, is currently approved for patients who have tried at least four other medicines for the blood cancer.

By Ned Pagliarulo • Aug. 10, 2022

Pfizer, Valneva take next step with Lyme disease shot, starting large trial

The experimental vaccine is the most advanced candidate for the tick-borne disease and, if successful, would be the first to reach the market since withdrawal of GSK’s Lymerix.

By Delilah Alvarado • Aug. 9, 2022

Fresh off Enhertu success, AstraZeneca and Daiichi claim progress for next cancer drug

A treatment that AstraZeneca acquired for up to $6 billion in 2020 is showing early promise as a combination agent in lung cancer, where it’s being tested alongside Merck & Co.’s Keytruda.

By Ben Fidler • Aug. 9, 2022

Amgen data on KRAS cancer drug disappoints, opening door for rival Mirati

Highly anticipated trial results for Lumakras combinations with immunotherapy fell short of expectations, showing significant liver toxicity that limited dosing.

By Ned Pagliarulo • Aug. 8, 2022

Lilly takes long view on Alzheimer’s drug as FDA starts expedited review

The pharma said Thursday the FDA had accepted its application for donanemab, starting a six-month evaluation for accelerated approval.

By Ned Pagliarulo • Aug. 5, 2022

Alnylam drug succeeds in key heart disease study, boosting company

The success of Alnylam's treatment in a trial known as APOLLO-B could pave the way for the biotech to reach consistent profitability, should the FDA sign off on a planned approval application.

By Ben Fidler • Aug. 3, 2022

The promise of oral therapies for cytokine inhibition

The science behind new oral therapies for cytokine inhibition and why they could be transformational for patients.

By David Lee, Global Therapeutic Area Head, Immunology • Aug. 1, 2022

Alnylam reveals longer wait for anticipated drug trial results

Important data from the APOLLO study of the biotech's drug Onpattro are due before the end of August, Alnylam said Thursday.

By Ned Pagliarulo • July 28, 2022

Pfizer, BioNTech to study 'bivalent' booster as FDA weighs approach

The small-scale study is part of the companies’ efforts to update their vaccine in time for a potential fall vaccination campaign to help boost waning immunity.

By Christopher Newman • July 27, 2022

BridgeBio advances drug for dwarfism after study data

The highest tested dose of BridgeBio’s drug helped the bones of children with achondroplasia grow faster, leading the company to expand study enrollment.

By Ned Pagliarulo • July 26, 2022

Seagen, Astellas claim positive results in study key to cancer drug's success

The mid-stage trial tested the companies’ drug Padcev with Merck’s Keytruda in first-line bladder cancer. The data are reportedly a factor in Merck’s talks with Seagen over a potential buyout.

By Ned Pagliarulo • July 26, 2022

Vertex to move non-opioid painkiller into late-stage tests after FDA agreement

Trial results released earlier this year showed potential for the drug, which has become one of Vertex’s top pipeline prospects.

By Delilah Alvarado • July 22, 2022

Atara shares sink after update on multiple sclerosis trial

Much-anticipated interim results from Atara’s Phase 2 study revealed little about the likelihood of the trial’s success, spurring a market sell-off.

By Ned Pagliarulo • July 13, 2022

With new data, Pliant drug shows promise in tough-to-treat lung disease

The drug’s potential impact on the lung health of idiopathic pulmonary fibrosis patients, many of whom take medicines from Roche and Boehringer Ingelheim, came as a “surprise” given the study’s small size, according to one analyst.

By Ben Fidler • July 11, 2022

New analysis encourages Intercept to refile NASH drug for approval

The company said the new examination is more “in line” with FDA guidance and still found that its drug, known as OCA, helped reduce liver scarring.

By Jacob Bell • July 7, 2022

FDA, under fire for Aduhelm approval, starts review of another Alzheimer's drug

The regulator could clear Biogen and Eisai’s lecanemab by early January. A decision before Phase 3 results could amplify the criticism the agency already faces, however.

By Jonathan Gardner • July 6, 2022

10 clinical trials to watch in the second half of 2022

The biotech industry's downturn accelerated in the first half of the year. Important study readouts for Eisai, Gilead, Merck and Seagen, among others, could determine whether the slump endures.

By Ben Fidler , Ned Pagliarulo , Jacob Bell • July 5, 2022

Celldex, continuing comeback, adds to early promise for skin disease drug

New study results show the medicine appears as effective, or stronger, than standard care for chronic urticarias, a common condition that drugs from Regeneron and Novartis have struggled to treat.  

By Ben Fidler • July 1, 2022

FDA halts tests of Sanofi drug, acquired in $3.7B buyout, due to side effects

Liver problems in trial volunteers led the agency to curtail studies of the drug, marking the second major setback related to Sanofi’s acquisition of Principia Biopharma.

By Jonathan Gardner • June 30, 2022

Jazz drug fails in late-stage study for multiple sclerosis

The drug, part of Jazz’s $7 billion acquisition of GW Pharmaceuticals, didn’t perform significantly better than placebo in treating the muscle spasticity that’s tied to the disease.

By Jacob Bell • Updated June 29, 2022

Sanofi, GSK say dual-acting vaccine prevents COVID-19 from omicron in large trial

A shot the partners have been developing reduced symptomatic infections associated with the variant by 72% compared to a placebo, positioning it as a potential option, if approved, to combat omicron.  

By Jonathan Gardner • June 24, 2022

FDA suspends US testing of Sarepta Duchenne drug over safety concerns

The regulator stopped dosing in the U.S. for a drug that’s meant to be a more potent version of Sarepta's marketed medicine Exondys 51, after a patient experienced dangerously low magnesium levels.

By Ben Fidler • June 23, 2022