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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M&A: a critical driver for sustainable top-line growth in healthcare

Andrew Callaway

Global Head, Healthcare Investment Banking, RBC Capital Markets

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

June 25, 2022 06:00 AM EDT

Weekly

15 LGBTQ leaders in biopharma; Paul Stoffels’ Galapagos revamp; AstraZeneca catches up in ATTR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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David Loew (Ipsen)

June 27, 2022 03:19 AM EDT

Deals

Ipsen snags an approved cancer drug in $247M M&A deal as another battered biotech sells cheap

John Carroll

Editor & Founder

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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ImmunityBio

Dunkirk, NY, USA

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

June 23, 2022 11:53 AM EDT

Pharma

Marketing

AstraZeneca debuts first consumer campaign for its Covid-19 prophylactic Evusheld — and a first for EUA drugs

Beth Snyder Bulik

Senior Editor

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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June 25, 2022 09:00 AM EDT

R&D

GSK says its drug for chronic hep B could ‘lead to a functional cure’ — but will it be alone or in combination?

Lei Lei Wu

News Reporter

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Joe Wiley, Amryt Pharma CEO

June 24, 2022 10:14 AM EDT

Pharma

FDA+

Amryt Pharma submits a formal dispute resolution to the FDA over rejected skin disease drug

Tyler Patchen

News Reporter

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

June 24, 2022 09:48 AM EDT

Pharma

FDA+

Pharma-friendly senator calls on FDA for a third time to show patent protections shouldn't be blamed for high drug prices

Zachary Brennan

Senior Editor

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

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June 24, 2022 08:51 AM EDTUpdated 09:45 AM

Pharma

FDA+

EMA signs off on 3 drugs recently rejected by FDA, including BioMarin's new hemophilia gene therapy

Zachary Brennan

Senior Editor

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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June 24, 2022 07:37 AM EDTUpdated 02:31 PM

R&D

Coronavirus

Sanofi, GSK tout 72% Omicron efficacy in PhIII trial of next-gen, bivalent shot — with an eye to year-end rollout

Amber Tong

Senior Editor

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.