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

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

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

Intra-Cellular approval keeps focus on FDA's neuroscience review process

Lumateperone, to be sold as Caplyta, had conflicting results in late-stage trials​. Its approval suggests the FDA is using a "glass half full lens" to review neuroscience drugs, said one analyst.

By Jacob Bell • Dec. 23, 2019

Roche hands $1B to Sarepta in major return to gene therapy deals

The Swiss pharma acquired a muscular dystrophy treatment for markets outside the U.S. in a pact that could reach a value of $10 billion.

By Jonathan Gardner • Dec. 23, 2019

Stealth's rare disease drug falls short in study, sinking shares

The setback could also put in jeopardy an option Stealth recently granted to Alexion to partner on elamipretide following the Phase 3 readout.

By Ned Pagliarulo • Dec. 20, 2019

Paratek wins US contract to develop anthrax treatment

A BARDA contract will provide Paratek with up to $285 million, some of which would go toward to acquiring the biotech's antibiotic for a national stockpile.

By Kristin Jensen • Dec. 19, 2019

Bladder cancer approval gives Seattle Genetics its second drug

Padcev, an antibody-drug conjugate developed jointly with Astellas, will expand Seattle Genetics' business beyond Adcetris.

By Ned Pagliarulo • Dec. 19, 2019

Global Blood taps Syros for its next act in blood disorders

Fresh off a sickle cell approval, Global Blood is looking to Syros' technology to keep up with the rapid innovation happening there and in beta thalassemia.

By Jacob Bell • Dec. 18, 2019

Muscle data doesn't help Solid build faith in its gene therapy

A regulatory delay due to an adverse event, meanwhile, has put Solid further behind Sarepta in the pursuit of a Duchenne muscular dystrophy gene therapy.

By Jonathan Gardner • Dec. 18, 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

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

In stunning twist, FDA approves Sarepta's Duchenne drug it rejected

Following an August rejection of Vyondys 53, Sarepta appealed the FDA's decision through a formal dispute resolution process that led to the agency's about-face.

By Ned Pagliarulo , Jonathan Gardner • Dec. 12, 2019