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.”

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; 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.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

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