Gene therapy consortium targets eight rare diseases for clinical trials

Backed by nearly $100 million, the public-private consortium aims to create a standard development roadmap for gene therapies using AAV viral vectors.

By Christopher Newman • Updated May 16, 2023

Sanofi, AstraZeneca study results add support for their RSV drug in infants

The antibody reduced RSV infections requiring hospitalization by 83% in a large study, bolstering evidence for the drug ahead of a U.S. approval decision later this year.

By Christopher Newman • May 12, 2023

Explore the Trendline➔

Neuroscience drug development

Enthusiasm is running higher among drugmakers and investors for neuroscience drug development, buoyed by recent approvals of new Alzheimer’s, ALS and depression medicines.

By BioPharma Dive staff

Alzheimer’s doctors see promise, limits in Lilly’s latest drug data

While some see the new donanemab results as adding to a “watershed moment” in Alzheimer’s research, others say they reinforce the limitations of so-called anti-amyloid therapies.

By Jacob Bell • May 10, 2023

Roche pays China-based biotech $70M for a new HER2 drug

The drug, currently in Phase 1, is an oral medicine designed to cross the blood-brain barrier and better treat HER2 tumors that have spread to the brain.

By Jonathan Gardner • May 9, 2023

FibroGen’s anemia pill falls short in blood cancer study

The medicine, available in Europe but rejected two years ago by U.S. regulators, didn’t eliminate the need for blood transfusions among patients with myelodysplastic syndrome.

By Jonathan Gardner • May 5, 2023

Valneva, Pfizer push back timeline for Lyme disease vaccine

After issues with a contract research group, the partners now expect to file an approval application for their shot in 2026, one year later than initially anticipated.

By Delilah Alvarado • May 4, 2023

Lilly’s new Alzheimer’s data may both help and hinder rival Biogen

The success of Lilly’s donanemab in slowing Alzheimer’s progression should reinforce the potential of Leqembi — a similar, recently approved medicine from Eisai and Biogen — but may also make for stiff competition.

By Jacob Bell • May 3, 2023

Lilly drug slows Alzheimer’s decline in large study

The company plans to quickly submit an application for U.S. approval based on the trial results, which showed a consistent benefit to treatment with the drug, called donanemab.

By Jonathan Gardner , Ned Pagliarulo • Updated May 3, 2023

Travere set back by study failure of kidney disease drug

Shares in the biotech, formerly Retrophin, sank after the company disclosed its newly approved drug sparsentan failed a study meant to expand its use.

By Ned Pagliarulo • May 2, 2023

Alnylam’s early Alzheimer’s results encourage, but company discloses new hurdle

Partner Regeneron said the data, which showed substantial reduction of a key protein, suggests hope for the “bold dream” of silencing genes in the brain.

By Kristin Jensen • April 27, 2023

Lilly drug Mounjaro succeeds in second weight loss study

Trial participants nearly 16% of their body weight in a finding that should help the closely watched GLP-1 drug gain an FDA approval for obesity.

By Jonathan Gardner • April 27, 2023

Novartis trims 10% of drug pipeline in research cutback

The company cut about 20 early-stage projects, including nine solid tumor programs, after reviewing their strategic fit and commercial potential.

By Christopher Newman • April 25, 2023

Morphic shares swing on study data for drug viewed as ‘oral Entyvio’

Results from a small trial suggested Morphic's pill could be effective in treating inflammatory bowel disease, briefly lifting the company’s valuation by hundreds of millions of dollars. 

By Ned Pagliarulo • Updated April 25, 2023

Ionis, AstraZeneca detail data backing rare disease rival to Alnylam drugs

A second-generation treatment called eplontersen halted nerve damage and improved quality of life in a late-stage study the companies are using to seek U.S. approval.

By Ned Pagliarulo • April 24, 2023

Apellis’ immune system drug hits a setback in ALS

The company has chosen to stop an extension portion of a key study after an external group of advisers reviewed the data available and concluded further treatment with the drug was unwarranted.

By Jacob Bell • April 21, 2023

J&J study data show dramatic benefit to multiple myeloma cell therapy

A study abstract inadvertently posted online showed J&J and Legend’s therapy, Carvykti, reduced the risk of disease progression or death by more than 70% over standard drugs.

By Ned Pagliarulo • April 19, 2023

Vaxcyte strengthens case for experimental pneumococcal vaccine

The company reported positive results from a second Phase 2 study of a shot that’s meant to challenge vaccines from Pfizer, Merck and GSK.

By Delilah Alvarado • Updated May 3, 2023

Patient-centered clinical trials improve recruitment and retention

Let’s face facts. Patient enrollment in clinical trials is time-intensive & costly. We must do better.  

April 17, 2023

Navigate the complexities of cell and gene therapies

See how the effective use of CMC expertise helped guide a biopharma company’s development of a CGT product.

April 17, 2023

Moderna, Merck data support claim of cancer vaccine’s promise

Data presented at AACR suggest a personalized shot made with messenger RNA may amplify the effects of a widely prescribed cancer immunotherapy.

By Jonathan Gardner • April 16, 2023

New data raise more doubts about Moderna’s flu vaccine

The shot appears to generate an immune response that matches or exceeds existing vaccines, but investigators aren’t yet sure how well it can prevent illness.

By Jonathan Gardner • April 11, 2023

Merck, Eisai report pair of study setbacks for cancer drug combo

Phase 3 trials testing Merck’s Keytruda with Eisai’s Lenvima in colon and skin cancers didn’t show a survival benefit, denting the partners’ plans to expand the drugs’ use.

By Ben Fidler • April 7, 2023

In NEJM, full Pfizer data show RSV vaccine’s protection

Shots from Pfizer and GSK could soon become the first approved for the common respiratory infection. Results published in the high-profile medical journal give a closer look at the former.

By Delilah Alvarado • Updated April 6, 2023

Cytokinetics to end late-stage study of ALS drug

The decision comes after an external group of advisers who got an interim look at the data found Cytokinetics’ drug had “no effect” on the study’s goals.

By Jacob Bell • March 31, 2023

J&J abandons RSV vaccine in major strategy shift

The pharma, once a frontrunner in the race to develop the first vaccine for the respiratory virus, said it will end a 23,000-person study of its experimental shot amid a restructuring of its infectious disease division.

By Christopher Newman • Updated March 29, 2023