Josh Cohen (L) and Justin Klee (Amylyx)

Af­ter an about-face on a PhI­II tri­al, FDA sets up an ad­comm to re­view con­tro­ver­sial ALS drug next month

Fol­low­ing a U-turn from the FDA, the agency’s Pe­riph­er­al and Cen­tral Ner­vous Sys­tem Drugs Ad­vi­so­ry Com­mit­tee will meet on March 30 for an all-day tele­con­fer­ence to dis­cuss what could be on­ly the third ALS drug ap­proved since 1995.

Al­though the po­ten­tial drug from Amy­lyx, known as AMX0035, may work, the FDA pre­vi­ous­ly sought an­oth­er tri­al to con­firm ear­ly pos­i­tive re­sults. The agency had asked Amy­lyx to con­duct a Phase III study last April to see whether Phase II da­ta from 2020 held up.

But then, rough­ly five months lat­er, the agency re­versed course, say­ing the new study wouldn’t be need­ed for re­view. Amy­lyx sub­mit­ted its ALS drug for US ap­proval in No­vem­ber af­ter meet­ing with reg­u­la­tors, and it was grant­ed pri­or­i­ty re­view the next month. The ex­pect­ed PDU­FA date is June 29.

The biotech pre­vi­ous­ly pub­lished its Phase II/III tri­al re­sults in the New Eng­land Jour­nal of Med­i­cine, show­ing AMX0035 could slow the on­set of ALS against a stan­dard-of-care-based place­bo. Based on a func­tion­al rat­ing scale, which Amy­lyx used to mea­sure its pri­ma­ry end­point, AMX0035 showed a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in dis­ease pro­gres­sion over about six months.

The 137-pa­tient study showed how those in the ac­tive arm scored on av­er­age 2.32 points high­er on the sur­vey than place­bo af­ter 24 weeks, good for a p-val­ue of p=0.03.

While the re­sults weren’t de­fin­i­tive, and ALS is his­tor­i­cal­ly a tough field for R&D and a fa­tal dis­ease where most pa­tients live an av­er­age of two to five years af­ter re­ceiv­ing their di­ag­noses, the re­sults were good enough for of­fi­cials in the EU and Cana­da to con­sid­er ap­proval.

While it’s un­known how the FDA or its out­side ad­vi­sors will view the fi­nal da­ta pack­age, the an­nounce­ment of the meet­ing is an­oth­er sign that FDA is adopt­ing more re­laxed ap­proval stan­dards in neu­ro.

Sim­i­lar­ly to Bio­gen’s con­tro­ver­sial Alzheimer’s drug ap­proval, the FDA’s neu­ro head Bil­ly Dunn, who’s be­ing in­ves­ti­gat­ed for an im­prop­er­ly close re­la­tion­ship with Bio­gen, helped dri­ve the shift by the agency with Amy­lyx.

In an in­ter­view with End­points News in Sep­tem­ber, Amy­lyx co-CEO Josh Co­hen said mul­ti­ple con­ver­sa­tions with US reg­u­la­tors, specif­i­cal­ly Dunn’s group in the neu­ro di­vi­sion, led to the agency to change its mind. By Co­hen’s retelling, the FDA had been ex­cit­ed about the Phase II CEN­TAUR da­ta but was in­ter­est­ed in see­ing more da­ta be­fore mov­ing for­ward.

Help­ing with a po­ten­tial drug launch, should the FDA OK the drug, the Mass­a­chu­setts-based Amy­lyx IPO-ed last month, rais­ing $190 mil­lion af­ter pric­ing its stock at the midrange of the of­fer­ing.

The com­pa­ny is led by a pair of for­mer col­lege room­mates — Co­hen and Justin Klee — who got some key ear­ly sup­port from the late Hen­ri Ter­meer.

Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Post-hoc analy­sis: EMA's CHMP re­jects Ipsen's po­ten­tial drug for rare ge­net­ic dis­ease

The European Medicines Agency’s Committee for Medicinal Products for Human Use on Friday rejected Ipsen Pharma’s potential treatment for a rare genetic disease known as fibrodysplasia ossificans progressiva (FOP), which causes extra bone to form outside the skeleton.

The EMA said on its website that it could not draw any firm conclusions on the benefits of the French biopharma’s Sohonos (palovarotene), which selectively targets the retinoic-acid receptor gamma (RARγ), “as the applicant’s conclusion was based on a post-hoc analysis which was neither scientifically nor clinically justified and pre-specified study objectives were not met.”

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FDA ap­proves an­oth­er in­di­ca­tion for Keytru­da, this time in the ad­ju­vant NSCLC set­ting

Merck’s blockbuster cancer treatment Keytruda has been handed another indication by the FDA.

The US regulator announced on Thursday that it has approved Keytruda to serve as an adjuvant treatment for non-small cell lung cancer (NSCLC), which is its fifth indication in NSCLC and 34th indication overall.

According to a Merck release, the approval is based on data from a Phase III trial, dubbed Keynote-091, which measured disease-free survival in patients who received chemotherapy following surgery. The data from Merck displayed that Keytruda cut down on the risk of disease recurrence or death by 27% versus placebo.

FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.