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.

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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Ab­bott pumps $450M+ in­to new Ire­land-based man­u­fac­tur­ing site project and hir­ing spree

As Ireland continues to see more investments and building projects from pharma companies, another contender is looking to place more investment in the Emerald Isle.

According to a report from The Irish Times on Friday, Abbott Laboratories is investing €440 million, or about $451 million, to build a new manufacturing plant in Kilkenny, located in the country’s southeast, to make more of its glucose monitors.

No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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Tony Coles, Cerevel CEO

Cerev­el takes the pub­lic of­fer­ing route, with a twist — rais­ing big mon­ey thanks to ri­val da­ta

As public biotechs seek to climb out of the bear market, a popular strategy to raise cash has been through public offerings on the heels of positive data. But one proposed raise Wednesday appeared to take advantage not of a company’s own data, but those from a competitor.

Cerevel Therapeutics plans to raise $250 million in a public offering and another $250 million in debt, the biotech announced Wednesday afternoon, even though it did not report any news on its pipeline. However, the move comes days after rival Karuna Therapeutics touted positive Phase III data in schizophrenia, a field where Cerevel is pursuing a similar program.

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Sanofi, GSK, Ha­le­on see stock prices dip and dive amid lit­i­ga­tion for re­called heart­burn drug

Zantac became one of the most well-known drugs on the market after being FDA-approved in 1983 — and now close to four decades later, lawsuits over safety concerns are rattling analysts and investors.

Sanofi, GSK and Haleon, GSK’s former consumer healthcare unit, have lost billions of dollars in market cap since Tuesday’s market close, according to Bloomberg. While Zantac is no longer on the market, the drop came after a suite of analysts, from Morgan Stanley and other firms, sounded the alarm on the potential impact of ongoing personal injury litigation.

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