Valeant wins first round in $445M gam­ble on bro­dalum­ab ap­proval

Valeant made an un­usu­al ap­pear­ance in front of an FDA ex­pert pan­el on Tues­day, and came out with a clear en­dorse­ment for an FDA ap­proval of their pso­ri­a­sis drug bro­dalum­ab.

The pan­el vot­ed 18 to 0 in fa­vor of ap­prov­ing bro­dalum­ab, de­spite the agency’s in­ter­nal re­view, which linked the drug to 6 sui­cides and sui­ci­dal ideation as well as car­dio risks. Four of the pan­elists rec­om­mend­ed an ap­proval with noth­ing more than a clear la­bel warn­ing on the risks in­volved, 14 vot­ed yes on an ap­proval with a risk man­age­ment plan in place.

Some of the pan­el mem­bers rec­om­mend­ed a black box warn­ing, which could lim­it the drug’s com­mer­cial po­ten­tial.

The pan­el, though, was won over by the ef­fi­ca­cy da­ta, the op­por­tu­ni­ty of adding a new weapon to be used against pso­ri­a­sis and a will­ing­ness to find a way to man­age what most con­sid­ered a mar­gin­al risk of sui­cide. Most of the pan­elists dis­missed any con­nec­tion with MACE or car­dio con­cerns.

“It seems to me the ben­e­fit for this drug has tremen­dous po­ten­tial for these pa­tients who are ab­solute­ly mis­er­able,” says Har­vard’s Lynn Drake, who sits on the FDA’s Der­ma­to­log­ic and Oph­thalmic Drugs Ad­vi­so­ry Com­mit­tee. “I think we have a drug here that is at some lev­els a bit of a game chang­er.” As for the six sui­cides: “It’s a sug­ges­tion, not a sig­nal.”

Valeant, known for its aver­sion to R&D risk, bought the pso­ri­a­sis drug bro­dalum­ab from As­traZeneca for the bar­gain base­ment rate of $100 mil­lion up front, $170 mil­lion in prelaunch mile­stones and an­oth­er $175 mil­lion in sales bonus­es. The big phar­ma un­loaded the drug af­ter its part­ner Am­gen bailed out ear­li­er af­ter a close look at a link be­tween the drug and sui­cides and sui­ci­dal think­ing con­vinced them that the com­mer­cial op­por­tu­ni­ty had been up­end­ed.

Where Am­gen saw fail­ure Valeant smelled op­por­tu­ni­ty, bag­ging the drug ahead of the reg­u­la­to­ry re­view. The com­pa­ny has made a prac­tice of buy­ing con­tro­ver­sial or mar­gin­al drugs, like Provenge and bro­dalum­ab. But in re­cent months its busi­ness mod­el, which de­pends heav­i­ly on ever-high­er prices for port­fo­lio drugs, has wilt­ed un­der a bright spot­light of un­fa­vor­able at­ten­tion, which has wilt­ed its share price.

The FDA usu­al­ly fol­low the guid­ance of its ex­perts, but there’s no guar­an­tee the agency will ap­prove the drug as-is be­fore first re­quir­ing a safe­ty study. That’s the big risk that still re­mains, though the odds in Valeant’s fa­vor clear­ly just im­proved by a wide mar­gin.

Even if Valeant does over­come the safe­ty is­sues and wins a for­mal ap­proval, it will face off against a crowd­ed field of ri­vals: Eli Lil­ly’s ix­ek­izum­ab, ap­proved in March as Taltz, Mer­ck’s MK-3222 and J&J’s IL-23 in­hibitor guselkum­ab. No­var­tis’ was first out in­to the mar­ket, with Cosen­tyx.

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 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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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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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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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.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.