FDA lifts clinical hold on Audentes’ gene therapy program after three patient deaths triggered alarms

FDA HQ in Silver Spring, Maryland (Credit: Ting Shen via Getty)

December 28, 2020 10:23 AM ESTUpdated 10:49 AM

FDA lifts clinical hold on Audentes' gene therapy program after three patient deaths triggered alarms

Max Gelman

Associate Editor

The big prize of Astellas’ $3 billion gene therapy buyout last year is one step closer to getting back on track.

The FDA lifted its clinical hold of Audentes Therapeutics’ trial for AT132 on Monday, clearing the way back to the clinic for a gene therapy aimed at treating X-linked myotubular myopathy. Regulators had pressed pause in June after two patients died on the high dose of the therapy, a third individual in the study also died in August from gastrointestinal bleeding.

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Data Literacy: The Foundation for Modern Trial Execution

Kristin Mauri

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

January 25, 2021 06:45 AM ESTUpdated 07:07 AM

Coronavirus

Merck scraps Covid-19 vaccine programs after they fail to measure up on efficacy in another major setback in the global fight

John Carroll

Editor & Founder

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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January 25, 2021 11:05 AM EST

Regulatory

Can struggling Iterum turn the corner to an antibiotic success story? They will know in six months

Amber Tong

Senior Editor

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Ron Cooper, Albireo CEO

January 25, 2021 08:00 AM EST

Regulatory

Albireo just advanced down to the 10-yard line at the FDA. And Ron Cooper’s team is getting prepped for the next big play

John Carroll

Editor & Founder

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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Matt Gline (L) and Vivek Ramaswamy

January 25, 2021 09:30 AM ESTUpdated 09:46 AM

Bioregnum

Scoop: Vivek Ramaswamy is handing the CEO job to a top lieutenant at Roivant — but he’s not exactly leaving the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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Bahija Jallal, Immunocore

January 25, 2021 10:59 AM EST

Startups

Buried in Immunocore's IPO filings? A kickback scheme from a now former employee

Jason Mast

Editor

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

January 25, 2021 10:57 AM EST

Coronavirus

Covid-19 roundup: Moderna doubles down on Covid-19 with new booster trials; Australia plans domestic production of AstraZeneca vaccine amid distribution lag

Nicole DeFeudis

Associate Editor

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

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Jean-Christophe-Hyvert, Lonza

January 25, 2021 10:52 AM EST

Pharma

Manufacturing

Lonza looking to build on 'differentiated advantage' in Covid-19, CDMO marketplace in 2021

Conner Mitchell

Associate Editor

It’s not new for Lonza, the Swiss CDMO nearing its quasquicentennial anniversary, to be in the upper echelon of the biotech manufacturing industry.

But 2020 — as it was for many CDMOs — was a special year even by Lonza’s standards. The company inked a deal to produce 1 billion worldwide doses of Moderna’s Covid-19 vaccine and tapped pharma vet Pierre-Alain Ruffieux to lead its operations, moves which have allowed Lonza to make a myriad of other deals that will continue to ramp up its global production capacity.

January 25, 2021 10:29 AM EST

News Briefing

News briefing: Jeffrey Leiden to chair Tmunity board of directors; Opdivo wins new approval in advanced RCC

Max Gelman

Associate Editor

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.