Tri­ci­da 'fore­shad­owed' its CRL — but shares still fall with fear of years-long de­lay

No­body likes re­jec­tions. But it does help to have a heads-up.

Such is the case at Tri­ci­da, which was slapped with a com­plete re­sponse let­ter as reg­u­la­tors turned down its lead and on­ly drug for meta­bol­ic aci­do­sis in pa­tients with chron­ic kid­ney dis­ease.

“The CRL was not sur­pris­ing as it was fore­shad­owed by an an­nounce­ment in mid-Ju­ly that the FDA has iden­ti­fied de­fi­cien­cies in vev­er­imer’s ap­pli­ca­tion,” Cowen’s Phil Nadeau wrote.

Ger­rit Klaern­er

That, though, doesn’t guar­an­tee Tri­ci­da won’t run in­to the worst-case sce­nario in which the de­lay can stretch in­to years. It al­so didn’t stop the stock from drop­ping an­oth­er 23.64% to $10.11, fol­low­ing a pre­cip­i­tous fall from $26.20 back in Ju­ly.

CEO Ger­rit Klaern­er of­fered more can­did com­ments in an in­vestor call ear­li­er this month, not­ing that “it is un­like­ly we will re­ceive ap­proval to mar­ket vev­er­imer in the Unit­ed States on our PDU­FA goal date” since the FDA would pre­sum­ably clar­i­fy its con­cerns in a CRL.

Hav­ing that ex­pec­ta­tion set lend­ed Klaern­er some cred­i­bil­i­ty when he said he’s pleased that the FDA has pro­vid­ed help­ful, spe­cif­ic com­ments and ap­pears will­ing to work with the Tri­ci­da team.

Nadeau, who sees block­buster po­ten­tial for the drug, is still a fan. The Phase III re­sults were “con­vinc­ing­ly pos­i­tive,” with vev­er­imer hit­ting all the end­points on durable blood bi­car­bon­ate lev­els and safe­ty. A few oth­er things are to blame, ac­cord­ing to the an­a­lyst:

  1. the nov­el­ty of vev­er­imer’s sur­ro­gate end­point
  2. the lack of prece­dent for car­dio-re­nal drugs be­ing grant­ed ac­cel­er­at­ed ap­proval, and
  3. the FDA’s di­min­ished com­mu­ni­ca­tion with spon­sors in re­cent months in light of the lack of in-per­son meet­ings due to Covid-19.

Reg­u­la­tors have laid out mul­ti­ple op­tions to ad­dress the prob­lems they see, Tri­ci­da wrote in a re­lease. The dif­fer­ences can be huge — and they won’t find out un­til a Type A meet­ing lat­er in the year.

For in­stance, the FDA wants ad­di­tion­al da­ta be­yond TR­CA-301 and its ex­ten­sion tri­al to as­sess “the mag­ni­tude and dura­bil­i­ty of the treat­ment ef­fect of vev­er­imer on the sur­ro­gate mark­er of serum bi­car­bon­ate” and how ap­plic­a­ble it is to the US pop­u­la­tion, giv­en on­ly 9% in the drug arm was en­rolled in the coun­try.

If reg­u­la­tors end up de­cid­ing they need re­sults from the on­go­ing VAL­OR-CKD study, Tri­ci­da will have to wait un­til 2022-2023 for in­ter­im da­ta and 2024-2025 for fi­nal da­ta.

With cash, cash equiv­a­lents and in­vest­ments worth $437 mil­lion at the end of the sec­ond quar­ter, Tri­ci­da re­port­ed that it has a run­way up to ear­ly 2022.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”