FDA advisors vote 6-4 against Amylyx’s ALS drug, further dimming its hope for regulatory success

Photo: Manuel Balce Ceneta/AP Images

March 30, 2022 04:52 PM EDTUpdated 07:21 PM

FDA advisors vote 6-4 against Amylyx's ALS drug, further dimming its hope for regulatory success

Max Gelman

Senior Editor

In a marathon hearing Wednesday, outside FDA advisors recommended to the agency that a single randomized study did not provide conclusive proof over whether an experimental ALS drug is effective.

Panelists voted 6-4 in the negative, substantiating the agency’s concerns about the robustness of data in a Phase II study run by Amylyx Pharmaceuticals — by the slimmest possible margin. The drug, known as AMX0035, produced a statistically significant reduction on a functional rating scale, but the effect proved unconvincing to the committee.

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The Factors Driving a Rapid Evolution of Gene & Cell Therapy and CAR-T Clinical Research in APAC

Kasey Kime

Director of Regulatory Affairs at Novotech

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region.

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

September 24, 2022 06:00 AM EDT

Weekly

The Endpoints 11; bluebird's $3M gene therapy; Biogen tout new neuro data; Harsh reviews for cancer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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September 23, 2022 12:30 PM EDT

Pharma

Manufacturing

EMA warns of shortages of two Boehringer heart drugs due to a spike in demand

Tyler Patchen

News Reporter

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

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Head of Site Finance mRNA - Cell & Gene

Lonza

Geleen, Netherland

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September 22, 2022 07:01 AM EDTUpdated September 23, 04:21 AM

Special

The Endpoints 11: The top private biotechs in pursuit of new drugs. Pushing the envelope with powerful new technologies

John Carroll

Editor & Founder

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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September 23, 2022 02:38 PM EDTUpdated 03:37 PM

R&D

AstraZeneca, Merck cull one Lynparza indication in heavily pretreated ovarian cancer patients

Paul Schloesser

Associate Editor

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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September 23, 2022 09:58 AM EDTUpdated 01:23 PM

Pharma

FDA+

FDA's outside experts vote in favor of Ferring's fecal transplant for C. difficile, setting the stage for Seres

Kyle LaHucik

Associate Editor

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Mene Pangalos (AstraZeneca via YouTube)

September 23, 2022 07:29 AM EDT

R&D

AstraZeneca shuts the PhIII door for Ionis' PCSK9 drug despite positive PhIIb

Amber Tong

Senior Editor

When Ionis and AstraZeneca unveiled the first round of mid-stage data for their antisense PCSK9 drug, Mene Pangalos, AstraZeneca’s EVP of biopharmaceuticals R&D, underscored the drug’s “potential best-in-class efficacy profile.”

But now that the second batch is in, it appears AZD8233 isn’t hitting the mark after all.

Ionis announced Friday morning that although the candidate, also dubbed ION449, met the primary endpoint in the Phase IIb SOLANO trial, its partners at AstraZeneca have decided not to move it into Phase III studies because the “results did not achieve pre-specified efficacy criteria.”

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Head of Quality Systems

Lonza

Lexington, MA, USA

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September 22, 2022 11:40 AM EDTUpdated September 22, 08:34 PM

R&D

Updated: Biogen throws itself back into muddled data arguments with more details on its antisense ALS drug

Lei Lei Wu

News Reporter

With a highly watched FDA decision deadline coming in late January, Biogen and Ionis dropped the full data on the Phase III study of their ALS drug tofersen in the New England Journal of Medicine on Wednesday.

Biogen is looking for approval for tofersen in a very small subset of ALS patients — some 2%, according to the paper — who have a SOD1 gene mutation, which has previously been linked to ALS. Tofersen is meant to reduce levels of mutant SOD1 proteins.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

September 22, 2022 11:11 AM EDT

FDA+

Law

FDA's OCE makes the case for accelerated approval rider in user fee reauthorization

Zachary Brennan

Senior Editor

Four experts from the FDA’s Oncology Center of Excellence took to the New England Journal of Medicine yesterday to make the case for not only improving the agency’s ability to expeditiously pull dangling accelerated approvals when, on the rare occasion, confirmatory trials fail, but also better building “quality and efficiency into the AA on-ramp.”

The timely perspective arrives as Congress has exactly one week left to draft, release and sign off on the reauthorized user fee deals before layoff notices will be sent to drug reviewers. That package, which is likely to hitch a ride with the continuing resolution, may or may not include several policy riders (opposed by Republicans), including one that would allow the FDA to require confirmatory trials to be underway before an AA is granted, and would improve the process by which FDA can withdraw AAs.