FDA knocks back Sarepta in Duchenne gene therapy race with Pfizer, but analysts urge caution

Doug Ingram, Sarepta CEO (Sarepta)

September 10, 2020 07:07 AM EDT

FDA knocks back Sarepta in Duchenne gene therapy race with Pfizer, but analysts urge caution

Jason Mast

Editor

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positive results and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a “program update” for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but “needed additional dialogue” with the agency to confirm.

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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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Usama Malik

December 2, 2021 04:37 PM EST

Pharma

Ex-Immunomedics CFO charged with insider trading, faces up to 20 years in prison after allegedly tipping off girlfriend and relatives of a PhIII success

Max Gelman

Editor

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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

December 2, 2021 09:02 AM ESTUpdated 7 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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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.

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Vas Narasimhan, Novartis CEO (Thibault Camus/Pool via AP Images)

December 2, 2021 10:24 AM EST

R&D

With generic competition heating up, Vas Narasimhan outlines Novartis' growth plans at R&D day

Max Gelman

Editor

Thursday marks Novartis’ annual R&D day, and with it comes CEO Vas Narasimhan’s attempt to spotlight the company’s pipeline strategy and emerging stars.

The biggest question entering Thursday’s presentation dealt with how the big biopharma will make up revenues from upcoming generic competition — Novartis says within the next five years, generics will eat away roughly $9 billion in sales. To offset this, Narasimhan outlined a strategy for 4% growth or higher until 2026, focusing on six key medicines he believes will see multibillion dollar profits during this time.

Incorporating External Data into Clinical Trials: Comparing Digital Twins to External Control Arms

Unlearn.AI

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.