Gene therapies for neurodegenerative diseases: Pfizer, Novartis, Biogen and others seek more from FDA

April 12, 2021 09:05 AM EDT

Gene therapies for neurodegenerative diseases: Pfizer, Novartis, Biogen and others seek more from FDA

Zachary Brennan

Senior Editor

Top biopharma companies are calling on the FDA to provide more context and examples, particularly on early interactions with the agency, as part of the companies’ feedback on draft guidance from January related to developing gene therapies to treat neurodegenerative diseases.

The 15-page draft guidance outlines various considerations on manufacturing, preclinical and clinical trials for gene therapies targeting amyotrophic lateral sclerosis, Alzheimer’s disease and other neurodegenerative disorders that are notoriously difficult to treat.

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MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

Innovative MedTech Demands Specialist Clinical Trial Regulatory Affairs and Design

Tom Avery

Scientific and Medical Affairs Specialist, Medical Devices

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Tony Coles, Cerevel CEO

August 11, 2022 07:26 AM EDT

Financing

Cerevel takes the public offering route, with a twist — raising big money thanks to rival data

Max Gelman

Senior Editor

As public biotechs seek to climb out of the bear market, a popular strategy to raise cash has been through public offerings on the heels of positive data. But one proposed raise Wednesday appeared to take advantage not of a company’s own data, but those from a competitor.

Cerevel Therapeutics plans to raise $250 million in a public offering and another $250 million in debt, the biotech announced Wednesday afternoon, even though it did not report any news on its pipeline. However, the move comes days after rival Karuna Therapeutics touted positive Phase III data in schizophrenia, a field where Cerevel is pursuing a similar program.

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Hervé Affagard, MaaT Pharma CEO

August 11, 2022 07:16 AM EDT

FDA+

One year into clinical hold, FDA has more questions about 'pooled' microbiome therapy

Amber Tong

Senior Editor

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

ENDPOINTS CAREERS

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Experimental Drug Development Centre

Singapore

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August 10, 2022 08:25 AM EDT

Editor's note

Who are the women blazing trails in biopharma R&D? Nominate them for Endpoints' 2022 special report

Nicole DeFeudis

Editor

Amber Tong

Senior Editor

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Bernat Olle, Vedanta Biosciences CEO

August 11, 2022 07:30 AM EDT

People

Citing 'challenging economic environment,' PhIII-ready microbiome biotech lays off 20% of staffers

Amber Tong

Senior Editor

The market downturn isn’t just sweeping up public biotechs.

Vedanta Biosciences, a developer of oral drugs derived from the human microbiome, is laying off about 20% of its staff — an unfortunately common occurrence these days. But CEO Bernat Olle took the unusual step of sharing the decision on LinkedIn and offering to connect the employees being let go with any company that’s hiring in their areas.

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August 10, 2022 07:33 AM EDTUpdated 2 hours ago

Pharma

Updated: Amid massive restructuring, Biogen looks to reduce physical presence in Boston

Amber Tong

Senior Editor

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Illustration: Kim Ryu for Endpoints News

August 9, 2022 07:00 AM EDTUpdated 08:48 AM

R&D

In Focus

Why non-opioid pain drugs keep failing — and what's next for the field

Lei Lei Wu

News Reporter

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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ENDPOINTS CAREERS

Senior Project Manager

CAI

Boston, MA, USA

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Robert Califf, FDA commissioner (Tom Williams/CQ Roll Call via AP Images)

August 10, 2022 07:19 AM EDT

FDA+

Hoping to expand monkeypox vaccine supply, US paves the way for new route of administration

Nicole DeFeudis

Editor

After making it clear that the US’ current monkeypox vaccine supply is insufficient, the FDA on Tuesday authorized a new route of administration that should increase the number of available doses by five-fold.

Regulators cleared Bavarian Nordic’s Jynneos vaccine for intradermal injection in adults older than 18. Unlike subcutaneous injection — the current method by which vaccine is delivered under the skin — an intradermal jab goes directly into the skin. It’s believed that this method requires less vaccine, since the dermis is rich in dendritic cells which specialize in taking up foreign antigens and presenting them to the immune system, according to Daniel Kuritzkes, chief of infectious diseases at Brigham and Women’s Hospital in Boston.

August 9, 2022 01:07 PM EDTUpdated 04:25 PM

Pharma

Marketing

'Messy at best': Is the US repeating the same Covid missteps with monkeypox messaging?

Beth Snyder Bulik

Senior Editor

When Kyle Planck first suspected he might have monkeypox in late June, he went to the CDC website and found six photos of different types of lesions. And that was about it for general public information.

Planck, who is a sixth-year PhD pharmacology researcher at Weill Cornell, kept looking though and found a separate part of the CDC website meant for healthcare professionals. There he found a medical slide deck with more pictures, professional journal articles and more details about symptoms and diagnosis.

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