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.

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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FDA calls for non­in­fe­ri­or­i­ty tri­als for an­tibac­te­ri­als, thanks to new drugs for re­sis­tant in­fec­tions

The FDA on Monday said it updated its recommendations for the clinical development of new antibacterial drugs, and thanks to a new clutch of drugs, sponsors will have to conduct noninferiority trials that include subjects with infections caused by certain drug-resistant organisms because an effective active control can be provided.

The guidance, which is likely to be released within the next day or so, builds on a 2017 document and specifies what noninferiority trial designs may be used “with a wider NI margin,” including cases in which the trial population is enriched for subjects with infections caused by certain drug-resistant organisms, FDA said.

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Bris­tol My­ers dusts off an old Cel­gene chemother­a­py for use in chil­dren with a rare blood can­cer

Bristol Myers Squibb is bringing new life to a chemotherapy drug from the old Celgene pipeline as it touts another approval in a rare form of blood cancer affecting young children.

The FDA on Friday approved Vidaza (chemically known as azacitidine) for pediatric patients 1 month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML). The new indication marks the first approval for Vidaza in more than a decade, though it’s commonly used to treat acute myeloid leukemia and myelodysplastic syndromes (MDS).