Establishing an integrated evidence plan for medical affairs and beyond

Over the life of a biopharmaceutical product, there are many situations when data from randomized controlled trials (RCTs) may not be sufficient to address stakeholder questions of value, safety and effectiveness.

Nov. 15, 2021

Cortexyme plans path forward for Alzheimer's drug that failed study

Detailed trial results presented last week are "interesting," one Alzheimer's expert said, but there are still "many challenges and uncertainties" with Cortexyme's unorthodox approach to treating the disease.

By Ned Pagliarulo • Nov. 12, 2021

Explore the Trendline➔

Emerging biotech

New biotechs continue to emerge despite a challenging market environment that has forced venture firms to build their drug startups more cautiously.

By BioPharma Dive staff

Moderna, escalating dispute with NIH, claims government had no role in key vaccine patent

The biotech claimed its NIH partners were involved only after a key discovery was made "by Moderna scientists using Moderna technology." The dispute could end up in court.

By Jonathan Gardner , Ben Fidler • Nov. 11, 2021

Detailed Roche study results muddy another Alzheimer's hypothesis

Data presented at an Alzheimer's meeting raised more questions about the seeming benefit of Roche and AC Immune's drug semorinemab, which they claim is the first of its kind to slow deterioration of memory.

By Jonathan Gardner • Nov. 11, 2021

FibroGen cuts jobs as it weighs anemia drug's future in US

The company, which had its treatment rejected by the FDA in August, eliminated 30 jobs and 70 open positions. An upcoming meeting with the agency looms as a key event.

By Kristin Jensen • Nov. 10, 2021

Pfizer buys into Biohaven's fast-selling migraine medicine

The pharmaceutical giant will pay Biohaven $500 million for ex-U.S. rights to rimegepant — a sizable licensing deal, though not the buyout that some of the biotech's investors appeared to be hoping for.

By Ned Pagliarulo • Nov. 9, 2021

Moderna founder unveils new drug company focused on a different kind of RNA

Encouraged by Moderna's success, Flagship Pioneering has been busy creating startups like Alltrna, which launched Tuesday. The biotech is researching how to make medicines from transfer RNA molecules.

By Jacob Bell • Nov. 9, 2021

Mirati gives first look at KRAS drug combination in lung cancer

Study results for Mirati's drug combined with Keytruda have been much anticipated, as the San Diego biotech aims to challenge Amgen and its rival KRAS-blocking drug Lumakras.

By Ned Pagliarulo • Updated Nov. 8, 2021

Eye-focused gene therapy startup gets $60M cash infusion from Sanofi

Shortly after postponing a $135 million IPO, Gyroscope has found another way to back a treatment for a disease that's become a top target of multiple drugmakers.  

By Jonathan Gardner • Nov. 8, 2021

Beam gets green light to begin first clinical test of base editing

Alongside the milestone, the gene editing biotech also announced John Maraganore, who will soon step down as Alnylam's CEO, will join its board.

By Ned Pagliarulo • Nov. 8, 2021

Cystic fibrosis group partners with Moderna founder to find better, potentially curative treatments

With an initial investment of $20 million, the collaboration will use technologies from various Flagship-backed companies to discover and develop new therapies for the disease.

By Jacob Bell • Nov. 3, 2021

Moderna, teaming with a startup, wades further into gene editing

The biotech has identified gene editing as a natural fit for the technology it uses to deliver its vaccines and drugs. A deal with Metagenomi, a startup backed by Bayer, will further those ambitions.

By Ben Fidler • Nov. 2, 2021

Amylyx asks FDA to approve ALS drug, while also preparing late-stage study

The biotech has officially filed for review of its closely watched ALS treatment, an outcome that seemed unlikely just a few months ago.  

By Jacob Bell • Updated Nov. 2, 2021

Moderna faces delay in plans to bring coronavirus vaccine to US teenagers, young kids

The FDA wants to better understand the risk of vaccine-associated myocarditis in 12- to 17-year-olds based on real-world use overseas, which will set back Moderna’s authorization plans in the U.S. 

By Ben Fidler • Nov. 1, 2021

Despite strong quarter, some wonder how long Gilead can rely on coronavirus drug

While other drugs in Gilead’s arsenal fell short, third quarter revenue from Veklury tripled what analysts had expected, reaching nearly $2 billion.

By Jacob Bell • Oct. 29, 2021

John Maraganore, pioneering RNAi executive, to step down as Alnylam CEO

Alnylam shares fell 15% on the news, which surprised Wall Street analysts and ends Maraganore's 19-year run overseeing the company's journey from startup to a biotech worth nearly $20 billion.  

By Ben Fidler • Oct. 28, 2021

Cortexyme drug fails in Alzheimer's trial, but company sees validation of unorthodox approach

Cortexyme shares lost more than three-fourths of their value after the the biotech's therapy, which is designed to work differently than drugs like Aduhelm, missed both primary goals in a late-stage study.

By Ned Pagliarulo • Oct. 26, 2021

Moderna vaccine safe and spurs immune response in kids, company says

The trial results should allow the company to ask the FDA for approval of its vaccine in younger children, just as Pfizer nears a crucial decision for its shot in 5- to 11-year olds.

By Jonathan Gardner • Oct. 25, 2021

Why natural language processing (NLP) is a critical part of your real world data strategy

The barrier to knowledge can be eliminated when pharmaceutical companies use natural language processing to analyze unstructured content.

By Hywel Evans, Director, IQVIA NLP Insights Hub • Oct. 25, 2021

With Merck blocking its path, a small biotech reroutes, to the dismay of investors

Shares of Agenus fell around 25% Friday, after the company said the FDA has recommended withdrawing an approval application for its cancer immunotherapy balstilimab.

By Jacob Bell • Oct. 22, 2021

Biogen's Alzheimer's drug is struggling, and a turnaround might not be coming soon

Sales of Aduhelm during the third quarter totaled only $300,000, falling well below expectations amid controversy over its approval and physician reluctance to prescribe it.

By Jacob Bell • Oct. 20, 2021

Atea, Roche change plans for oral COVID-19 drug after trial setback

A closely watched competitor to Merck's molnupiravir failed a Phase 2 study, prompting its makers to change their strategy in a move that could delay an answer on the drug's efficacy until late next year.

By Ben Fidler , Jonathan Gardner • Oct. 19, 2021

Biogen's closely watched ALS drug comes up short in late-stage study

Though the study's primary goal was missed, Biogen claimed there were some signs its drug, tofersen, could slow the disease's progression. The company is now engaging with regulators to discuss tofersen's future.

By Jacob Bell • Oct. 18, 2021

CRISPR touts new results as fresh questions surround 'off-the-shelf' CAR-T

The biotech's lymphoma treatment initially seems as potent as earlier cell therapies, but its effects waned, adding to doubts about off-the-shelf CAR-T.

By Jonathan Gardner • Updated Oct. 13, 2021

CureVac scraps its first COVID-19 vaccine and pivots to a second

The German biotech has abandoned plans to seek approval of the shot in Europe, citing tough competition, waning demand and the progress of a second candidate it's developing with GlaxoSmithKline. 

By Ben Fidler • Oct. 12, 2021