Homology unveils first data for PKU gene therapy, marking early milestone

Data presented Tuesday are from just three patients, but set the pace in a field that includes BioMarin and, most recently, Sangamo Therapeutics.

By Andrew Dunn • Dec. 17, 2019

GSK advances blood cancer drug to FDA, but rivals threaten

Belantamb mafodotin, a crucial drug for GSK's ambitions in oncology, could be the first anti-BCMA therapy approved. But new data show it's at risk of being outclassed.

By Ned Pagliarulo • Dec. 17, 2019

Explore the Trendline➔

Radiopharmaceuticals

A series of technological advances have led to an explosion of scientific progress — and dealmaking — involving medicines that can precisely deliver radioactive materials into tumors.   

By BioPharma Dive staff

With trial win, Alnylam to seek approval for first treatment of rare kidney disease

The biotech's RNAi therapy lumasiran appears positioned to become the first drug for primary hyperoxaluria. Potential rivals, though, are close behind.

By Andrew Dunn • Dec. 17, 2019

BeiGene's Brukinsa bet falls short against Imbruvica

The Chinese biotech's recently approved cancer drug wasn't able to beat out Imbruvica in a rare lymphoma, raising questions about its next regulatory filing.

By Jonathan Gardner • Dec. 16, 2019

Novartis shutters late-stage asthma program, spelling trouble for Gossamer

After four Phase 3 trials fell short, the Swiss pharma is ceasing development of fevipiprant in asthma.

By Andrew Dunn • Dec. 16, 2019

Gilead's NASH ambitions buckle under the weight of ATLAS

The Phase 2 ATLAS study was Gilead's last chance to stay in the pack of companies leading the charge toward a first-ever drug for NASH, a fatty liver disease.

By Jacob Bell • Dec. 16, 2019

Wave cancels its Exondys competitor and heads back to the lab in muscular dystrophy

Suvodirsen failed to stimulate production of a muscle-building protein, a finding that prompted suspension of development of a second Duchenne drug.

By Jonathan Gardner • Dec. 16, 2019

BioMarin drug for dwarfism meets goal in late-stage study

Positive data in hand, BioMarin plans to soon discuss with regulators filing vosoritide for approval, setting up a consequential year ahead for the biotech.

By Ned Pagliarulo • Dec. 16, 2019

How to implement risk-based quality management into your clinical trial

Change isn't easy in the pharmaceutical industry, but it's necessary to accommodate the diversity of data sources and study designs.

Dec. 16, 2019

Biogen's Alzheimer's drug gets caught in Sarepta's spotlight

The unexpected approval of Sarepta's Vyondys 53 has some wondering how lenient the FDA might be in reviewing aducanumab. Others, however, see little impact.

By Jacob Bell • Dec. 13, 2019

UniQure, Pfizer updates hint at gene therapy potential in hemophilia B

The two companies are advancing toward market one-time treatments for the blood disorder, with Takeda about to begin clinical testing of its own therapy.

By Jonathan Gardner • Dec. 10, 2019

Leukemia rivals try to stay ahead as Merck, Lilly chase

Merck's surprise buyout of ArQule will heat up competition in a class of cancer drugs that includes AbbVie and J&J's Imbruvica and AstraZeneca's Calquence.

By Jonathan Gardner • Dec. 10, 2019

Get ready for 2020: What to know about the 15 most important trends impacting healthcare

New report reveals the critical trends and market changes impacting biopharmaceutical companies in the year ahead.

Dec. 10, 2019

New drugs for sickle cell excite but, at ASH, a recognition that more is needed

Approvals for Adakveo and Oxbryta double the number of treatment options for sickle cell disease in the U.S., bringing hope that more may soon follow.

By Ned Pagliarulo • Dec. 9, 2019

As CAR-T presses ahead in multiple myeloma, alternative approaches gain supporters

Three different ways of attacking the blood disease are emerging, raising hopes for life-extending drugs for the sickest patients.

By Jonathan Gardner • Dec. 9, 2019

Readout of the Year: Aducanumab's surprise revival

If approved, the Alzheimer's drug would likely earn billions. But researchers are divided over whether Biogen's data show an unexpected benefit, or are just a statistical mirage.

By Jacob Bell • Dec. 9, 2019

Sangamo keeps pressure on rivals with updated hemophilia data

Longer follow-up on Sangamo and Pfizer's gene therapy show treated patients continue to be free of bleeds, but one patient's results could spark new questions.

By Ned Pagliarulo • Dec. 7, 2019

Biogen's Alzheimer's pitch yields few converts

The drugmaker's about-face on aducanumab has divided the Alzheimer's field. A closely followed CTAD presentation didn't appear to bridge the gap.

By Jacob Bell • Dec. 7, 2019

Myeloma cell therapies, now in pharma hands, move into spotlight

Long-awaited results from Bristol-Myers and Bluebird position the drugmakers to win a first-in-class CAR-T approval. Rival J&J, however, appears close behind. 

By Ned Pagliarulo • Dec. 7, 2019

Cancer gene therapy backed by Blackstone gets trial win

Results released Thursday are the first to be presented from a Phase 3 study of the therapy, which was recently made the cornerstone of a new company launched by Blackstone and Ferring.

By Jonathan Gardner • Dec. 5, 2019

The clearest picture of Biogen's Alzheimer's drug is still fuzzy

Biogen laid out its best case for aducanumab on Thursday, but even more detailed data couldn't clear up the uncertainties that have made its surprising decision to revive the drug so controversial.

By Jacob Bell • Dec. 5, 2019

Trial setback strikes blow to Sage antidepressant ambitions

SAGE-217 failed to meet the main goal of a Phase 3 depression study, a result that cut the drugmaker's market value by roughly $4 billion.

By Ned Pagliarulo • Dec. 5, 2019

New approval for Roche's Tecentriq heats up lung cancer battle with Keytruda

One Wall Street analyst expects the FDA's OK will help Tecentriq outpace Bristol-Myers Squibb's Opdivo in 2020 in the non-small cell lung cancer market.

By Jonathan Gardner • Dec. 4, 2019

What to watch as Biogen presents its all-important Alzheimer's data

During what some have called the biotech event of the year, doctors will be paying close attention to how Biogen accounted for missing data in its pivotal aducanumab studies.

By Jacob Bell • Dec. 4, 2019

Biogen scores a rare lupus win, lending support to its new immunology focus

In a two-part study, Biogen's BIIB059 hit both primary endpoints. But statistical significance, as well as a lack of safety data, create some uncertainty about the drug's future prospects.

By Jacob Bell • Dec. 3, 2019