Josh Cohen (L) and Justin Klee, Amylyx co-CEOs

UP­DAT­ED: Amy­lyx's ALS drug gets a three-month PDU­FA de­lay, set­ting up the piv­otal de­ci­sion for Sep­tem­ber

ALS pa­tients in the US are go­ing to have to wait a lit­tle bit longer to find out whether they’ll have a new treat­ment op­tion.

The FDA has ex­tend­ed the re­view pe­ri­od for Amy­lyx Phar­ma­ceu­ti­cals’ ex­per­i­men­tal ALS drug AMX0035 by three months, the biotech an­nounced Fri­day morn­ing, set­ting up a de­ci­sion date of Sept. 29. Reg­u­la­tors had pre­vi­ous­ly said the ap­proval or re­jec­tion would come by June 29.

Ac­cord­ing to Amy­lyx, the FDA had re­quest­ed more time to re­view the com­pa­ny’s da­ta, and the biotech said the ex­ten­sion is clas­si­fied as a “ma­jor amend­ment.” Typ­i­cal­ly, such ex­ten­sions in­volve the agency re­quest­ing new da­ta from the com­pa­ny, though it’s not im­me­di­ate­ly clear if FDA asked for new ef­fi­ca­cy or safe­ty analy­ses.

In an email to End­points News, Amy­lyx co-CEO Justin Klee de­clined to com­ment be­yond what the com­pa­ny shared in its press re­lease. The com­pa­ny’s stock $AM­LX rose on the news, jump­ing about 40% pre-mar­ket af­ter ris­ing more than 14% Thurs­day.

The biotech has been work­ing to get AMX0035 across the fin­ish line and win just the third ALS drug ap­proval in the US. ALS pa­tients and ad­vo­cates have in­ten­si­fied pres­sure on the FDA to green­light AMX0035, ar­gu­ing that be­cause their dis­ease is uni­ver­sal­ly fa­tal, they should have the op­tion to try the drug.

But the FDA ex­pressed se­ri­ous reser­va­tions about its ef­fec­tive­ness at an ad­vi­so­ry com­mit­tee hear­ing in March, call­ing Amy­lyx’s Phase II da­ta in­to ques­tion. Though the drug slowed dis­ease pro­gres­sion by a sta­tis­ti­cal­ly sig­nif­i­cant mar­gin com­pared to place­bo ac­cord­ing to a func­tion­al rat­ing scale, agency of­fi­cials took is­sue with the way Amy­lyx con­duct­ed its study and the ro­bust­ness of the da­ta.

Ul­ti­mate­ly, the FDA’s pan­el of ad­vi­sors said in a 6-4 vote that the Phase II da­ta did not “es­tab­lish” a con­clu­sion that the drug is ef­fec­tive.

Nev­er­the­less, ALS pa­tients have re­mained un­de­terred in their quest for more treat­ment op­tions, with the pres­sure on FDA man­i­fest­ing in a near­ly two-hour pub­lic com­ment ses­sion dur­ing March’s ad­comm. Many with ALS end up with ex­per­i­men­tal drugs as their on­ly op­tions, sign­ing up for clin­i­cal tri­als in the hopes that some­thing will work.

In a quick-take re­ac­tion to the news, SVB Se­cu­ri­ties an­a­lyst Marc Good­man not­ed it was “not sur­pris­ing” that Amy­lyx isn’t spec­i­fy­ing the na­ture of the FDA’s re­quest for new da­ta, “giv­en the is­sues raised” at the ad­comm. Good­man said that most of the in­vest­ment com­mu­ni­ty is ex­pect­ing the drug to be re­ject­ed, but the PDU­FA de­lay sug­gests the FDA is strug­gling to make a de­ci­sion.

“Sev­er­al KOLs have writ­ten in­di­vid­ual ed­i­to­ri­als re­but­ting many of the con­tro­ver­sies from the meet­ing in ad­di­tion to the con­tin­ued ag­gres­sive lob­by­ing by the ALS groups, but it’s still un­clear how much these ef­forts are hav­ing an im­pact,” Good­man wrote to in­vestors.

The FDA has pre­vi­ous­ly ap­proved two ALS drugs — rilu­zole in the mid-1990s, which has a mod­est ef­fect on ex­tend­ing sur­vival times; and edar­avone in 2017, af­ter it showed lim­it­ed im­prove­ment on clin­i­cal func­tion.

What­ev­er the agency’s de­ci­sion for Amy­lyx, the process has al­ready echoed ap­provals for Sarep­ta’s Duchenne mus­cu­lar dy­s­tro­phy drug Ex­ondys 51 and Bio­gen’s Alzheimer’s ther­a­py Aduhelm. Both of those drugs faced sim­i­lar, sig­nif­i­cant ef­fi­ca­cy ques­tions as Amy­lyx, as well as neg­a­tive ad­comm votes, but were green­light­ed re­gard­less.

This sto­ry has been up­dat­ed to in­clude Amy­lyx’s re­sponse to End­points’ re­quest for com­ment, the stock mar­ket’s re­ac­tion and analy­sis from SVB Se­cu­ri­ties an­a­lyst Marc Good­man.  

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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An­oth­er warn­ing let­ter for Lupin as FDA iden­ti­fies de­fi­cien­cies at In­dia-based site

With few new details of what needs fixing, Lupin disclosed last week that the FDA recently sent a warning letter to its Tarapur, India-based site.

After an inspection from March 22 to April 4, Lupin disclosed in an April stock filing that it received a Form 483 with four observations, but it didn’t offer any details on the observations.

Similar to comments made in April, the company said last week it does not believe the FDA slap will disrupt its drug supplies or the existing revenues from operations of this facility.

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Silviu Itescu, Mesoblast CEO

Mesoblast sends in im­proved po­ten­cy as­say, look­ing to re­sub­mit to FDA on acute graft-ver­sus-host dis­ease drug

In 2020, the FDA rejected Mesoblast’s remestemcel-L, or Ryoncil, its lead candidate for pediatric acute graft-versus-host disease (aGVHD) that didn’t respond to steroids. The FDA raised a number of concerns, first objecting to Mesoblast’s single arm, open-label trial, though regulators struggled to describe how a randomized trial would work, since pediatricians and parents were reluctant to put children with aGVHD in a placebo arm.

Tim Whitten, Taiho Oncology CEO

FDA green­lights Ot­su­ka sub­sidiary's bile duct can­cer drug

Otsuka subsidiary Taiho Oncology nabbed an accelerated approval on Friday for Lytgobi (futibatinib), its new kinase inhibitor to treat adults with a type of previously treated bile duct cancer, known as metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

The accelerated approval (meaning a confirmatory trial is necessary to show clinical benefit) follows a shortened six-month review, thanks to the FDA’s Real-Time Oncology Review program.