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more context and examples, particularly on early interactions with the agency, as part of the companies’ feedback on draft guidance from January related to developing gene therapies to treat neurodegenerative diseases.
The 15-page draft guidance outlines various considerations on manufacturing, preclinical and clinical trials for gene therapies targeting amyotrophic lateral sclerosis, Alzheimer’s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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January 17, 2022 06:00 AM EST
ZS Perspective: 3 Predictions on the Future of Cell & Gene Therapies
Ben Hohn
The field of cell and gene therapies (C>s) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C>s that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.
Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.
Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.
What lured Hal Barron away?; Top FDA minds on accelerated approval reforms; ‘Dead wrong’ Aduhelm ad blitz; 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.
Nothing can really compete with Hal Barron’s departure from GlaxoSmithKline as the news of the week, but we do have plenty of original reporting and analysis from the Endpoints team in this edition. Enjoy and have a nice weekend.
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'Altos was really a once-in-a-lifetime opportunity': Hal Barron reflects on his big move
By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.
Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.
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FDA+ roundup: FDA's neuroscience deputy departs amid ongoing Aduhelm investigations; Califf on the ropes?
Zachary Brennan
Senior Editor
Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.
Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.
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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)
Failing to confirm clinical benefit, Gilead pulls 2 accelerated approval indications for cancer drug
Zachary Brennan
Senior Editor
Gilead recently decided to pull two indications for its cancer drug Zydelig — in relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic leukemia (SLL) — after failing to complete the confirmatory trials required as part of the accelerated approvals from 2014.
“As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” Gilead said in a statement, noting it formally notified the FDA of its decision to voluntarily withdraw these indications.
Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)
European Parliament signs off on strengthening drug regulator's ability to tackle shortages
Zachary Brennan
Senior Editor
The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.
By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.
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What kind of biotech startup wins a $3B syndicate, woos a gallery of marquee scientists and recruits GSK's Hal Barron as CEO in a stunner? Let Rick Klausner explain
John Carroll
Editor & Founder
It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.
At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.
Milner’s question on one of those mornings on foot: “What do you want to do?”
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Time limits on accelerated approvals? FDA's oncology chief Rick Pazdur eyes potential reforms via international approaches
Zachary Brennan
Senior Editor
The spotlight on the accelerated approval pathway continues to shine bright, with the FDA’s top oncology official writing in an opinion that the pathway may be strengthened with bits and pieces of what other regulators in Europe and elsewhere have done with their expedited approval pathways, such as adding expiration dates for these faster approvals to ensure they confirm clinical benefit in a timely manner.
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Merck wins legal battle over insurance coverage after ransomware attack
Nicole DeFeudis
Editor
Merck has emerged victorious from a years-long legal battle with insurers over the coverage of more than a billion dollars in losses from the malware NotPetya, with a New Jersey Superior Court judge concluding that the responsibility is on insurers to clarify their policies around cyber attacks.
The pharma giant was one of several victims of a global cyber attack back in 2017 that also hit Danish shipping company Maersk, American food company Mondelēz, French construction giant Saint-Gobain and even the systems monitoring the Chernobyl nuclear power stations, Bloomberg reported back in 2019.
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