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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Pascal Soriot, AstraZeneca CEO (via Getty images)

UP­DAT­ED: FDA slaps As­traZeneca's MCL-1 can­cer drug with a hold af­ter safe­ty is­sue — 2 years af­ter Am­gen axed a trou­bled ri­val

There are new questions being posed about a class of cancer drugs in the wake of the second FDA-enforced clinical hold in the field.

Two years after the FDA hit Amgen with a clinical hold on its MCL-1 inhibitor AMG 397 following signs of cardiac toxicity, AstraZeneca says that regulators hit them with a hold on their rival therapy of the same class.

The pharma giant noted on clinicaltrials.gov that its Phase I/II study for the MCL-1 drug AZD5991 “has been put on hold to allow further evaluation of safety related information.”

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Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

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