Tricida CEO Gerrit Klaerner

Go­ing for broke, Tri­ci­da is slam­ming the brakes on a piv­otal tri­al ear­ly while plan­ning a run at the FDA

Two years af­ter a bit­ter set­back at the FDA, with its cash re­serves ebbing away, Tri­ci­da is ex­e­cut­ing a high-stakes gam­ble on win­ning a swift ap­proval at the FDA. A win would put them back in play with a new drug to com­mer­cial­ize. A loss, as one Wall Street an­a­lyst re­cent­ly as­sessed, would take the stock to ze­ro.

To­day Tri­ci­da $TC­DA an­nounced an “ad­min­is­tra­tive stop” for its VAL­OR-CKD tri­al af­ter watch­ing 237 pa­tients reach a pri­ma­ry end­point event — re­nal death, end-stage re­nal dis­ease, or greater than or equal to 40% re­duc­tion in es­ti­mat­ed glomeru­lar fil­tra­tion rate — in the re­nal out­comes study for their meta­bol­ic aci­do­sis and CKD drug vev­er­imer. They’ll con­tin­ue to track events in­to Q3 as they look for a top-line read­out in the near term.

When they start­ed out in the fourth quar­ter of 2018, Tri­ci­da re­searchers had set a tar­get for 511 events, which the biotech says would stretch the study in­to 2024. But op­er­at­ing cap­i­tal will be ef­fec­tive­ly tapped out by the ear­ly part of Q2 2023.

Ex­ecs say they got the FDA to sign off on the ear­ly tri­al stop af­ter re­view­ing their fi­nan­cial sit­u­a­tion with reg­u­la­tors along with study close-out pro­ce­dures.

Phil Nadeau at Cowen re­cent­ly as­sessed their chances, not­ing:

As­sum­ing a true haz­ard ra­tio (HR) of 0.70, the tri­al would be 78% pow­ered at 250 events. Switch­ing from pow­er to ob­served HR, VAL­OR-CKD could be suc­cess­ful at 250 events if the ob­served HR is 0.78. There is on­ly a mod­est in­crease to 0.79 ob­served HR should 300 events be in the fi­nal analy­sis. This cal­cu­la­tion on the ob­served HR re­quired for sig­nif­i­cance is based on 300 sim­u­la­tions on tri­al out­comes.

A win, he added, wold spur a big ral­ly — pre­sum­ably set­ting up a raise — while a fail would amount to an ex­tinc­tion lev­el event.

None of this will come as a big sur­prise to the biotech’s in­vestors, as Tri­ci­da ex­ecs have been painful­ly clear about what they’re do­ing and why. CEO Ger­rit Klaern­er was equal­ly trans­par­ent back in the sum­mer of 2020, as reg­u­la­tors wound up to de­liv­er a CRL for the drug.

This year, though, the in­dus­try has been roiled by a sav­age bear mar­ket for biotech stocks, and Tri­ci­da wants a few months of op­er­at­ing rev­enue in the bank when the biotech will make a fresh pitch.

“With re­gards to tim­ing over­all to­wards po­ten­tial com­mer­cial launch, pre­sum­ing we have the an­nounce­ment of the VAL­OR study ear­ly in Q4, we would an­tic­i­pate fil­ing or re­sub­mit­ting the NDA in Q2 of ‘23 and have a po­ten­tial PDU­FA date in Q4 of 2023,” Tri­ci­da CFO Ge­off Park­er said dur­ing their Q1 call a few days ago. “That’s a six-month re­view for a re­sub­mis­sion. And then rough­ly speak­ing, we would have a launch in Q1 of 2024. So pre­sum­ing pos­i­tive da­ta on VAL­OR-CKD, we will put those plans in­to mo­tion lat­er this year.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

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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