FDA panel backs Alnylam drug despite doubts over benefit

“There is a light wind for benefit, and no wind for risk,” said one adviser who voted with eight others to recommend the biotech’s drug Onpattro for cardiomyopathy of ATTR amyloidosis.

By Jonathan Gardner • Updated Sept. 14, 2023

After IPO success, Acelyrin hits trial setback with inflammation drug

Acelyrin’s share value collapsed Tuesday after its lead drug fell short in the company’s first big trial test since it raised $540 million in a May IPO.

By Kristin Jensen • Sept. 12, 2023

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

Daiichi Sankyo, with new data, to seek FDA approval of lung cancer drug

The HER3-targeting treatment could become the next antibody-drug conjugate to emerge from Daiichi Sankyo’s laboratories, after the AstraZeneca-partnered Enhertu.

By Jonathan Gardner • Sept. 11, 2023

In advocates’ push for superbug funding, ‘$6 billion is nothing’

The PASTEUR Act could be heading for another setback, but advocates say they see a future for funding research into antimicrobial resistance.

By Karissa Waddick • Sept. 8, 2023

Alnylam says hypertension drug succeeded in mid-stage study

The trial found two higher doses of Alynlam’s RNAi therapy lowered blood pressure better than placebo. But analysts say results from a different study will be more important for the drug’s future.

By Jacob Bell • Sept. 7, 2023

Wave readies clinical testing for first RNA editing therapy

The biotech, one of several advancing RNA editing medicines, expects to obtain “proof-of-mechanism” data in patients with the inherited disorder alpha-1 antitrypsin deficiency by next year.

By Kristin Jensen • Updated Sept. 5, 2023

Roche claims study success for targeted drug in early lung cancer

The Swiss pharma said, without details, that it observed “unprecedented” results that could make Alecensa the first treatment specifically available after surgery for ALK-positive lung tumors.

By Ben Fidler • Sept. 1, 2023

FibroGen’s Duchenne drug fails second trial in latest study setback

The failure marks another Phase 3 miss for the drug, pamrevlumab, and follows a restructuring and CEO switch for the struggling biotech.

By Delilah Alvarado • Aug. 30, 2023

Bayer reports positive early data for Parkinson’s cell therapy

The treatment, developed by a biotech Bayer acquired in 2019, appeared safe in a small trial and showed signs of an effect on disease progression. 

By Jacob Bell • Aug. 28, 2023

Merck kicks off Phase 3 tests for PCSK9 cholesterol pill

The company plans to enroll roughly 17,000 people in three Phase 3 trials of the drug, including a large cardiovascular study set to begin later this year.

By Delilah Alvarado • Aug. 25, 2023

Novo, with new data, builds case for using Wegovy to protect heart health

After trial results showed the obesity drug can prevent heart attacks, the company now has data indicating it can help people with heart failure, too.

By Jonathan Gardner • Aug. 25, 2023

Roche’s surprise study results spur new optimism for TIGIT drugs

Data inadvertently published from a closely watched lung cancer study suggest a drug blocking the protein TIGIT may help extend survival, a finding that boosted shares of other developers. 

By Jonathan Gardner • Aug. 23, 2023

Fulcrum rejoins sickle cell drug race as FDA lifts study hold

The biotech, which aims to develop an alternative to existing medicines and emerging gene-based treatments, will focus further testing on sicker patients.

By Ben Fidler • Aug. 22, 2023

FDA partially halts leukemia studies of Gilead cancer drug

The hold is the latest setback for a drug that was the center of Gilead’s $5 billion acquisition of biotech Forty Seven in 2020.

By Jonathan Gardner • Aug. 21, 2023

Moderna builds case for updated COVID shot ahead of latest booster push

But the demand for booster shots ahead of an expected surge of infections this fall remains an unanswered question weighing heavily on Moderna’s future outlook. 

By Jonathan Gardner • Aug. 18, 2023

Merck says drug acquired in $1B buyout scores in large kidney cancer study

The treatment, which Merck picked up through a 2019 acquisition of Peloton Therapeutics, helped delay disease progression in patients with advanced renal cell carcinoma. 

By Jacob Bell • Aug. 18, 2023

Boehringer moves Wegovy competitor into late-stage testing

The German pharmaceutical company is launching three Phase 3 trials of an obesity drug called survodutide, which like Wegovy targets the GLP-1 receptor

By Ned Pagliarulo • Aug. 17, 2023

Gilead antibody drug shows signs of potential in early lung cancer

A combination of Trodelvy and Merck’s Keytruda appears active against lung tumors, but has a high bar to clear and faces competition from AstraZeneca and Daiichi Sankyo.

By Jonathan Gardner • Aug. 17, 2023

FDA lifts hold on Arcellx’s Gilead-partnered cancer cell therapy

The agency had paused testing after a patient death, but is now permitting more types of bridging treatment to help keep participants’ disease at bay.

By Jonathan Gardner • Aug. 15, 2023

A biotech scraps two cancer trials in latest setback for emerging drug class

ALX Oncology’s decision to end studies in leukemia and myelodysplastic syndrome follows a similar setback from Gilead and casts further doubt on drugs known as CD47 inhibitors.

By Jonathan Gardner • Aug. 11, 2023

Novartis says drug helps control chronic hives in studies

The studies are another example of drugmakers exploring how so-called BTK inhibitors can be used to treat diseases outside of blood cancer. 

By Kristin Jensen • Aug. 9, 2023

Novo obesity drug protects heart health in large trial

Treatment with Wegovy led to a 20% reduction in the relative risk of cardiovascular complications or death, a finding that could further shift how doctors treat obesity.

By Jonathan Gardner • Updated Aug. 8, 2023

How the right technology and tools can accelerate progress in rare disease clinical trials

The need for more research into rare diseases is clear, but researchers face significant challenges. Fortunately, technology has created opportunities to innovate in clinical trials for rare diseases.

Aug. 7, 2023

Amgen says KRAS drug met goal in late-stage colon cancer trial

The company disclosed the study result alongside second quarter earnings showing Lumakras sales were flat versus the same period one year prior.

By Ned Pagliarulo • Aug. 3, 2023

Gilead-backed cell therapy startup Kyverna adds fresh funds for new strategy

A treatment licensed from the NIH and in clinical testing for lupus is the startup’s lead program, while its founding “Treg” cell therapy research matures, according to CEO Peter Maag.

By Ben Fidler • Aug. 3, 2023