Block­buster per­for­mance: Re­gen­eron, Sanofi add stel­lar PhI­II dupilum­ab da­ta on dis­play at EADV

Af­ter a steady drum­beat of praise from Re­gen­eron and its part­ners at Sanofi, in­ves­ti­ga­tors to­day backed up their un­blush­ing op­ti­mism over their late-stage drug prospect dupilum­ab with a batch of stel­lar late-stage tri­al da­ta for atopic der­mati­tis, adding to the pos­i­tive pro­file al­ready sketched out.

The sci­en­tists turned up at the Eu­ro­pean Acad­e­my of Der­ma­tol­ogy and Venere­ol­o­gy in Vi­en­na to re­view the da­ta from SO­LO1 and SO­LO2. And it was worth the wait for a drug they plan to mar­ket as Dupix­ent as they look to start rack­ing up rev­enue against the multi­bil­lion-dol­lar peak sales es­ti­mates that have hov­ered around this drug.

On every ma­jor score, dupilum­ab hand­i­ly out­per­formed the place­bo, un­der­scor­ing the part­ners’ high ex­pec­ta­tions for a ther­a­py that’s al­so in late-stage test­ing for asth­ma. And new da­ta on itch­ing and de­pres­sion should help close the sale.

Gi­an­lu­ca Pirozzi

“I tru­ly be­lieve this is go­ing to be rev­o­lu­tion­ary for many pa­tients,” Gi­an­lu­ca Pirozzi, the glob­al project head for Sanofi, tells me. This is the first mon­o­clon­al an­ti­body that blocks both Il-4 and IL-13 cy­tokines, and there’s noth­ing that looks like a ri­val threat on the near hori­zon, leav­ing these two big play­ers a lot of room to move.

The pa­tients in these sto­ries rep­re­sent a se­vere­ly af­flict­ed group, he adds, with no chance of re­lief. And while a long way from a uni­ver­sal ef­fect, the da­ta in­di­cate that this drug has a tremen­dous amount of com­mer­cial po­ten­tial, with lit­tle in the way of safe­ty is­sues to fret about.

The full re­port card now in­cludes:

On the Pru­ri­tus NRS ranges from 0 (no itch) to 10 (worst itch imag­in­able). At 16 weeks, for SO­LO 1 and SO­LO 2, re­spec­tive­ly, 41 and 36 per­cent of pa­tients who re­ceived Dupix­ent 300 mg every two weeks, and 40 and 39 per­cent of pa­tients who re­ceived Dupix­ent 300 mg week­ly, achieved a four-point or greater re­duc­tion in their NRS score com­pared to 12 and 10 per­cent with place­bo.

Lest any­one for­get, the two big part­ners have al­so post­ed an im­pres­sive amount of pos­i­tive da­ta.

On the pri­ma­ry end­point: 37 and 36 per­cent of adult pa­tients who re­ceived Dupix­ent 300 mg week­ly, and 38 and 36 per­cent of pa­tients who re­ceived Dupix­ent 300 mg every two weeks, achieved clear­ing or near-clear­ing of skin le­sions as mea­sured by the 5-point In­ves­ti­ga­tor’s Glob­al As­sess­ment (IGA) scale. In the place­bo arm, those rates were tracked at 10 and 8 per­cent with place­bo.

On an­oth­er EU pri­ma­ry and US sec­ondary end­point: 52 and 48 per­cent of adult pa­tients who re­ceived Dupix­ent 300 mg week­ly, and 51 and 44 per­cent of pa­tients who re­ceived Dupix­ent 300 mg every two weeks, achieved a 75 per­cent or greater re­duc­tion in their Eczema Area and Sever­i­ty In­dex score (EASI-75). In the place­bo arm that hit 15 and 12 per­cent.

On the per­cent im­prove­ment in EASI from base­line:  The score was 72 and 69 per­cent in pa­tients who re­ceived the 300 mg week­ly dose, and 72 and 67 per­cent for pa­tients who re­ceived Dupix­ent 300 mg every two weeks, com­pared to 38 and 31 per­cent for place­bo.

Based on the da­ta they’ve seen so far, it looks like they will go with a drug dosed once every two weeks. Dupilum­ab was re­cent­ly filed with the FDA and is get­ting a pri­or­i­ty re­view, with a PDU­FA date on March 29. So far, this drug has been sail­ing through a big Phase III with­out a hitch.

An­a­lysts haven’t over­looked the po­ten­tial here, but there has been some fret­ting that pay­ers are al­ready lay­ing in wait for a big new ther­a­py like this. And that could make it hard for these part­ners to re­al­ize the kind of big gains that have been fore­cast. Ge­of­frey Porges notes:

We now look to the part­nered com­mer­cial or­ga­ni­za­tions to ex­plain how they plan to price and pro­mote dupi, at a man­age­able ex­pense in­vest­ment, to avoid the for­mi­da­ble road­blocks that have stalled the re­cent launch­es of oth­er chron­ic use spe­cial­ty ther­a­peu­tics with large com­mer­cial op­por­tu­ni­ties. Oth­er­wise we have con­cerns that the stock is fac­ing launch es­ti­mates that are too high, and launch rev­enue that falls short of po­ten­tial.

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.