Scoop: Ferring, Blackstone’s $570M gene therapy venture has imploded after CMC issues stymied a once high-profile quest

David Meek (Photographer: Marlene Awaad/Bloomberg via Getty Images)

April 8, 2021 07:04 PM EDTUpdated April 9, 07:19 AM

Scoop: Ferring, Blackstone’s $570M gene therapy venture has imploded after CMC issues stymied a once high-profile quest

John Carroll

Editor & Founder

Back in late 2019, FerGene looked golden. It had a gene therapy for bladder cancer — spun out of Ferring — at the FDA, with a priority review and breakthrough status indicating that great things awaited on the near horizon.

Ferring backed up the venture with $170 million, with Nick Galakatos’ Blackstone Life Sciences chipping in another $400 million with great fanfare to get the company running. David Meek jumped from the CEO job at Ipsen to run it all, and a little over a year ago much of the executive team was assembled to bring it all home.

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At the Inflection Point for the Next Generation of Cancer Immunotherapy

Willem Overwijk, Ph.D.

Vice President, Oncology Research, Nektar Therapeutics

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

December 4, 2021 06:00 AM EST

Weekly

All about Omicron; We need more Covid antivirals; GSK snags Pfizer’s vaccine exec; Janet Woodcock’s future at FDA; and more

Amber Tong

Senior Editor

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The slate of products we’re offering here at Endpoints is continuing to grow, and it’s not just limited to editorial. If you haven’t, do visit your reader profile to see if there are any other weekly newsletters you’re interested in — as each comes with its own exclusive content. And don’t miss the publisher’s note from Arsalan Arif on Endpoints Studio, our latest avenue for advertising on Endpoints.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

December 2, 2021 09:02 AM ESTUpdated 14 hours ago

Pharma

Coronavirus

As Omicron spread looms, oral antivirals appear to be one of the best defenses — now we just need more

Paul Schloesser

Associate Editor

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Deep Genomics

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December 3, 2021 10:54 AM EST

R&D

FDA+

Pfizer, Amgen and Janssen seek further clarity on FDA's new benefit-risk guidance

Zachary Brennan

Senior Editor

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Janet Woodcock (AP Images)

December 3, 2021 09:51 AM EST

FDA+

Janet Woodcock plots her future at FDA, with senior advisor role to fall back on if Califf wins confirmation

Zachary Brennan

Senior Editor

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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Richard Pazdur (via AACR)

December 3, 2021 07:16 AM ESTUpdated 07:29 AM

FDA+

Accelerated approval reforms need meaningful confirmatory trial improvements, professors write in Science

Zachary Brennan

Senior Editor

Outside of Covid-19, 2021 has been the year of the accelerated approval.

Beginning last spring, FDA openly challenged six “dangling” accelerated approvals (hadn’t confirmed their clinical benefit yet), three of which were later pulled by the companies.

Then in June, FDA pulled out the accelerated approval pathway, seemingly out of nowhere, to sign off on Biogen’s controversial Alzheimer’s drug Aduhelm. It hadn’t even been mentioned at the drug’s adcomm.

December 2, 2021 01:29 PM EST

Manufacturing

One worker at a Latvian manufacturer is dead after a fire breaks out at facility

Josh Sullivan

Associate Editor

A press release referred to it as simply, “the incident.” But a fire at an Olainfarm site in Latvia last week has left one person dead, and injured another, the company announced Monday.

Just before midnight Nov. 26, a fire broke out in the production building of JSC Olainfarm, as the result of an “accident,” a company spokesperson said in an email to Endpoints News. The two victims were both company employees, and the causes of the accident are still being determined.

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December 2, 2021 11:40 AM EST

Manufacturing

Fujifilm Diosynth dumps another $454M into its supply chain, this time at a facility in the UK

Josh Sullivan

Associate Editor

Fujifilm Diosynth Biotechnologies is in the middle of a monumental point in the company’s 10-year history, and the CDMO is about to grow even more, as it sets out to be the “beating heart” of the UK’s North East Life Sciences ecosystem.

A site in Billingham, Teeside, UK will receive a $453.72 million investment package from the manufacturer to double the existing footprint and create the largest multi-modal biopharmaceutical manufacturing site in the UK, bringing another 350 jobs to the region by late 2023.

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Lisa Deschamps, AviadoBio CEO

December 2, 2021 11:07 AM ESTUpdated December 3, 02:00 AM

Financing

Startups

Cell/Gene Tx

Ex-Novartis business head hops over to a gene therapy startup — and she's reeled in $80M for a dash to the clinic

Nicole DeFeudis

Editor

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News.