GW Phar­ma’s ral­ly rolls on as cannabi­noid drug scores again in PhI­II Epid­i­olex an­ti-con­vul­sive study

GW Phar­ma­ceu­ti­cals’ late-stage cannabi­noid high is on a roll.

The UK biotech post­ed strong ef­fi­ca­cy da­ta for its ex­per­i­men­tal an­ti-con­vul­sive ther­a­py Epid­i­olex in treat­ing Lennox-Gas­taut syn­drome, three months af­ter get­ting the par­ty start­ed with a big ral­ly on its piv­otal da­ta for Dravet syn­drome, a rare form of se­vere epilep­sy.

In­ves­ti­ga­tors said their drug ex­act­ly dou­bled the 22% me­di­an re­duc­tion in drop seizures among a group of LGS pa­tients with an av­er­age age of 15 — 44% vs 22%.

The Lon­don-based com­pa­ny looks to be set­ting the stage to wrap up a slate of new drug ap­pli­ca­tions for Epid­i­olex, and in­vestors re­spond­ed with a 20% spike in the biotech’s share price this morn­ing. The surge came af­ter the Brex­it vote rat­tled mar­kets at the end of last week, send­ing biotech shares down as in­vestors fled risk in search of a port in the storm.

Back in March the com­pa­ny ex­cit­ed in­vestors with re­sults for the first of four late-stage stud­ies on Epid­i­olex, with the drug re­duc­ing con­vul­sions in chil­dren with treat­ment-re­sis­tant Dravet syn­drome by a me­di­an av­er­age of 39% com­pared to on­ly 13% in the place­bo arm.

There are cur­rent­ly no drugs ap­proved for the dis­ease. And if GW can con­tin­ue to rack up pos­i­tive da­ta, it may well prove to be the pi­o­neer for cannabi­noid treat­ments. Epid­i­olex us­es a pure form of CBD, a com­pound found in mar­i­jua­na. The plan now is to fol­low up with pre-NDA meet­ings with the FDA with a fil­ing planned next year.

The com­pa­ny de­clined to spec­u­late on a po­ten­tial price for the drug in a call this morn­ing with an­a­lysts, but some of the most bull­ish an­a­lysts cov­er­ing the com­pa­ny have is­sued some dizzy­ing peak sales es­ti­mates rang­ing from $2 bil­lion to $3 bil­lion a year.

GW will have to ex­plain an un­fa­vor­able safe­ty pro­file, though. Re­searchers re­port­ed that 20 pa­tients on Epid­i­olex “ex­pe­ri­enced a se­ri­ous ad­verse event (nine of which were deemed treat­ment re­lat­ed) com­pared with four pa­tients on place­bo (one of which was deemed treat­ment re­lat­ed). Twelve pa­tients on Epid­i­olex dis­con­tin­ued treat­ment due to ad­verse events com­pared with one pa­tient on place­bo. There was one death in the Epid­i­olex group, which was deemed un­re­lat­ed to treat­ment. Of the pa­tients who com­plet­ed this tri­al, 100 per­cent have opt­ed to con­tin­ue in­to an open-la­bel ex­ten­sion tri­al.”

The type of se­ri­ous events tracked in this sec­ond piv­otal study is “very sim­i­lar” to what was seen in the first tri­al, not­ed CMO Stephen Wright in the call. There were no un­ex­pect­ed se­ri­ous ad­verse events and no changes in the safe­ty pro­file.

GW al­so has a dose-rang­ing study for Lennox-Gas­taut syn­drome that should read out lat­er in the year with a Phase III tri­al of Epid­i­olex in Tuber­ous Scle­ro­sis Com­plex al­ready un­der­way and a late-stage study in in­fan­tile spasms due to launch in the fourth quar­ter.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

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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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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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