Sanofi turns to an up­start in the mR­NA field in lat­est at­tempt to quick­ly squelch a glob­al pan­dem­ic

You can add one more mes­sen­ger RNA pro­gram in the works for Covid-19 — and phar­ma gi­ant Sanofi is part­ner­ing on it, adding to its more tra­di­tion­al vac­cine ap­proach head­ed slow­ly but steadi­ly to the clin­ic.

David Loew Sanofi

Sanofi $SNY chose Trans­late Bio’s $TBIO mR­NA plat­form for the al­liance, hitch­ing a ride with an­oth­er biotech look­ing to dis­patch nu­cleotide se­quences en­cod­ed for anti­gens, or vi­ral pro­teins, that can kick up an im­mune re­sponse. New se­quenc­ing tech has giv­en these plat­forms a big leg up over old­er — and much slow­er — plat­forms that re­ly on eggs and cell cul­tures to re­li­ably dis­cov­er and de­liv­er the vi­ral pro­teins need­ed to safe­ly spur im­mune re­spons­es to prep them to re­pel a spe­cif­ic in­vad­er.

Sanofi Pas­teur, their big sub­sidiary en­gaged in this new ef­fort, knows all about how the old plat­form works and now hopes to use those in­sights to speed the Trans­late Bio pro­gram along. In a re­cent in­ter­view with End­points News’ Ja­son Mast, they de­scribed the race to a new vac­cine as some­thing of a tor­toise and hare sit­u­a­tion.

Now they want one of each.

“We be­lieve the more ap­proach­es we ex­plore, the bet­ter our like­li­hood of suc­cess in achiev­ing this goal,” said David Loew, glob­al head of vac­cines at Sanofi.

Ac­cord­ing to an SEC fil­ing, Trans­late isn’t get­ting an up­front for the deal and they plan to work out a roy­al­ty agree­ment lat­er.

Ron Re­naud

Trans­late and Sanofi — which ini­tial­ly al­lied on 5 in­fec­tious dis­ease pro­grams 2 years ago — are al­so al­ready plan­ning to build up new man­u­fac­tur­ing ca­pac­i­ty through a con­tract man­u­fac­tur­ing or­ga­ni­za­tion. Trans­late is helmed by CEO Ron Re­naud, who was re­cent­ly hit by a par­tial hold for one of their R&D pro­grams.

The mR­NA field’s Covid-19 ef­forts are led by Mod­er­na, which the NIH turned to in an ef­fort to see if they could de­vel­op and field a new vac­cine in record time. BioN­Tech and Cure­Vac are al­so at work on their own pro­grams.

In­ter­est­ing­ly, Mod­er­na had built up a full pipeline of mR­NA ther­a­pies — in­struct­ing cells to make a drug — be­fore shift­ing the spot­light square­ly on vac­cines. Their lead ef­fort was cen­tered on CMV, which has since been tem­porar­i­ly eclipsed by a 24/7 plan to com­bat coro­n­avirus as new cas­es and deaths swift­ly mount around the globe.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

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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Boost­er bo­nan­za: FDA en­dors­es 'mix-and-match' scheme, and Mod­er­na and J&J too

The FDA late Wednesday signed off on authorizing the use of heterologous — or what FDA calls a “mix and match” of a primary vaccine series and different booster doses — for all currently available Covid-19 vaccines, in addition to separately authorizing Moderna and J&J boosters.

On the mix-and-match approach, which FDA officials insisted isn’t too confusing in a press conference, the agency offered the example of an 18-year-old who received the J&J shot at least two months ago and may now receive a single booster of the J&J, a half dose of the Moderna, or the Pfizer-BioNTech booster.

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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Man­u­fac­tur­ing woes for No­vavax’s Covid jab bad­ly dis­rupt plans for roll­out to the poor — re­port

Production problems at a Novavax facility in Maryland have led to delays in the Covax vaccine sharing program. Now, a shortage of 1 billion doses is expected, as the supplier tries to navigate producing a shot up to regulators’ standards, Politico reported Tuesday.

The company has run into trouble with the purity of the vaccine. Novavax has had trouble proving it can produce a shot consistently up to standards, and it has caused significant delays in the rollout to low- and middle-income countries. This follows several delays at Novavax that has put the executive crew on the defensive.

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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Bill Gates at the Global Investment Summit in London, Oct. 19, 2021 (Leon Neal/Pool via AP Images)

Gates Foun­da­tion pledges $120M to ramp up gener­ic sup­ply of Mer­ck­'s Covid-19 pill while ac­tivists blast Pfiz­er's dis­pro­por­tion­ate pow­er

Merck’s molnupiravir may not be officially authorized anywhere in the world yet, but who will get access to it has shaped up to be a huge issue. The Bill & Melinda Gates Foundation is now stepping up to ensure lower-income countries won’t be left behind — and calling on others to follow its lead.

The oral antiviral pill, which was shown to dramatically cut the risk of severe Covid-19 disease and death in a Phase III study, is the latest rallying symbol in the battle against not just the coronavirus but the inequality it’s exposed.