Epizyme drug granted approval by FDA for rare cancer

Five years after buying back rights from Eisai, Epizyme won approval for its first-ever drug. 

By Ned Pagliarulo • Updated Jan. 24, 2020

Roche strengthens case for SMA drug with new study data

Results showed the oral drug can help improve motor function in babies with the severest form of a rare degenerative muscular disease.

By Jonathan Gardner • Jan. 23, 2020

Explore the Trendline➔

Cell therapy

The continued emergence of CAR-T therapy has fueled research into next-generation approaches and new applications, such as its use in autoimmune diseases.

By BioPharma Dive staff

Safety risks highlighted in FDA letter on Sarepta's Vyondys

In rejecting Vyondys last August, one FDA official wrote that, without confirmatory trials for either of Sarepta's muscular dystrophy drugs, it's difficult to judge whether they're worth the risk.

By Jonathan Gardner • Jan. 22, 2020

Drug trial success a consolation prize for Akcea, Ionis

Although Novartis chose not to license Akcea and Ionis' high triglyceride drug, positive data could open up new development opportunities.

By Ned Pagliarulo • Jan. 22, 2020

5 biopharmas to watch as the decade's last earnings kick off

Biogen faces down analysts one more time on its aducanumab plans, while two biotechs could give early glimpses into their first-ever drug launches. 

By Ned Pagliarulo , Jacob Bell • Jan. 21, 2020

After big year, RNAi drugmakers compete to prove what comes next

"We are in the first out of the first inning of the lifespan of RNAi," said Arrowhead CEO Christopher Anzalone. Two RNAi drugs are now approved, but the field still has much to prove.

By Andrew Dunn • Jan. 17, 2020

BioNTech rescues cancer biotech Neon in bargain buyout

The all-stock deal, worth $67 million, valued Neon at a 86% discount to the cell therapy developer's IPO price of $16 per share.

By Ned Pagliarulo • Jan. 16, 2020

Orchard isn't for the picking, Biohaven's Allergan headache, and who's on Vascepa?

Amarin CEO John Thero had one question for investors eager to hear about his company's plans for its heart pill, while Orchard made a case for independence.

By Jacob Bell • Jan. 16, 2020

Nektar quickly drops opioid drug after FDA panel rejection

After a decade of research, the biotech said it will withdraw its application for oxycodegol and make no further investment in the drug.

By Kristin Jensen • Jan. 15, 2020

Galapagos finds a new normal at biotech's biggest dealmaking event

Though the Belgian drug company is tied to a 10-year, $5 billion research pact with Gilead, its CEO says there's still some "wheeling and dealing" left to do.

By Jacob Bell • Jan. 15, 2020

Neuroscience's decade, a biotech's 'crown jewel,' and Ionis gets selective

Roche, maker of the last decade's top cancer drugs, is betting that neuroscience R&D can make strides in the 2020s — an outlook that contrasts with research retreats by the pharma's peers. 

By Andrew Dunn , Jacob Bell • Jan. 15, 2020

Astellas swoops in for Adaptimmune deal a day after positive data

For $50 million, the Japanese pharma signed up for three programs in a deal that could net Adaptimmune hundreds of millions more if all goes to plan. 

By Jonathan Gardner • Jan. 14, 2020

Alexion's neuro bet, Rubius' trial troubles and biotech's gene therapy milestones

On the first day of the JPM conference, Alexion mapped out ambitious plans in neuroscience, while Rubius did its best to explain delays in testing its first drug.

By Jacob Bell , Andrew Dunn • Jan. 14, 2020

BioMarin sets gene therapy sights beyond hemophilia

At the JPM conference, the biotech announced a manufacturing lift and a new trial start that affirm its ambitions in gene therapy go beyond its closely watched hemophilia A treatment.

By Jacob Bell • Jan. 13, 2020

Biogen finds $75M home for Pfizer Alzheimer's drug

Having stepped back from neuroscience research, Pfizer will sell off an early-stage therapy to Biogen, which has made the space its central focus.

By Jonathan Gardner • Jan. 13, 2020

Incyte turns to German Morphosys to refresh cancer pipeline

A licensing deal for Morphosys' near-to-market lymphoma drug will pad a pipeline that was recently weakened by a surprising study setback for Incyte. 

By Ned Pagliarulo • Jan. 13, 2020

Blueprint wins 1st FDA approval with targeted cancer drug

The Boston-area biotech advanced Ayvakit from initial human testing to regulatory approval in just four years, a rapid timeline reflective of the drug's gene-targeted profile.

By Ned Pagliarulo • Jan. 10, 2020

5 biotech trends to follow in 2020

Enthusiasm for new drugs and burgeoning companies will collide with an election year spotlight.

By Ned Pagliarulo • Jan. 9, 2020

Moderna, though early in development, prepares its vaccine for market

The vaccine just began mid-stage testing, but Moderna is already preparing for the next steps, which include a large Phase 3 study and hiring of sales associates.

By Jacob Bell • Jan. 9, 2020

Solid cuts one-third of staff, goes all in on delayed gene therapy

Re-starting a halted muscular dystrophy trial is Solid's priority as it puts a second drug and an assistive device on the backburner.

By Jonathan Gardner • Jan. 9, 2020

Lilly looms over Blueprint as the biotech files targeted cancer drug

Positive results in certain lung cancer patients support Blueprint's decision to submit pralsetinib, a RET-inhibitng drug, for approval in the first quarter.

By Jacob Bell • Jan. 8, 2020

Alexion's blockbuster Soliris bested by Apellis in Phase 3 trial

The study result sent shares in Apellis up by more than 20%, as investors saw potential for the Massachusetts biotech to take on Alexion's top-seller. 

By Andrew Dunn • Jan. 7, 2020

Merck adds to lung cancer wall with Otsuka deal

The $50 million deal with two Japanese biotechs is a small bet to match challengers in Amgen and Mirati, who are advancing agents targeting KRAS mutations.

By Jonathan Gardner • Jan. 6, 2020

Trial failure keeps Incyte searching for its next act

A setback for Incyte's experimental drug itacitinib adds to a list of pipeline disappointments for the Delaware biotech. 

By Ned Pagliarulo • Jan. 3, 2020

Wave's Huntington's drug shows effect but falls short of mark set by Roche and Ionis

The gene-targeting drug lowered levels of a damaging mutant protein, but the therapy's modest effect disappointed investors.

By Jonathan Gardner • Jan. 2, 2020