Vivek Ramaswamy, JPM20 (Credit: Jeff Rumans)

Vivek Ra­maswamy's LNP de­liv­ery tech play Genevant scores $600M li­cens­ing deal from Take­da for nu­cle­ic acid ther­a­pies

With mR­NA vac­cines in high de­mand and new RNA ther­a­peu­tics crowd­ing on­to the mar­ket, the de­mand for lipid nanopar­ti­cles has nev­er been high­er. Now, one of se­r­i­al en­tre­pre­neur Vivek Ra­maswamy’s Vant out­fits has scored a big deal with Take­da to de­vel­op LNP-de­liv­ered liv­er drugs.

The Japan­ese drug­mak­er will ear­mark $600 mil­lion for a li­cens­ing deal with Genevant, an LNP de­liv­ery tech out­fit, to de­vel­op nu­cle­ic acid ther­a­pies for liv­er fi­bro­sis, the com­pa­nies said Mon­day.

The part­ner­ship will laser in on “pre­vi­ous­ly in­ac­ces­si­ble” tar­gets in he­pat­ic stel­late cells us­ing Genevant’s LNP de­liv­ery sys­tem. The pact al­so stip­u­lates roy­al­ties on fu­ture sales.

Genevant, one of the com­pa­nies in Ra­maswamy’s ex­ten­sive Vant uni­verse, co­a­lesced in April 2018 around the idea of de­liv­er­ing ad­vanced RNA ther­a­pies with LNP tech­nol­o­gy sourced from Ar­bu­tus Bio­phar­ma. That tech, which Ar­bu­tus out-li­censed to Al­ny­lam for its FDA-ap­proved On­pat­tro (patisir­an), al­lows drug­mak­ers to pack­age RNA ther­a­pies with­in small lipid cap­sules that can be more eas­i­ly cir­cu­lat­ed in the blood.

In Jan­u­ary, Genevant reached a deal with Grit­stone On­col­o­gy to li­cense out its LNP tech for a suite of “self am­pli­fy­ing RNA” vac­cines for Covid-19. Grit­stone agreed to dole out $192 mil­lion in fu­ture pay­outs as part of the agree­ment.

Lipid nanopar­ti­cles have been in in­creas­ing­ly high de­mand as more and more RNA ther­a­peu­tics hit the mar­ket and as a suite of Covid-19 vac­cines con­tin­ue to notch ap­provals. Of those, both Pfiz­er/BioN­Tech and Mod­er­na use LNP to de­liv­er their vac­cines, which has put an enor­mous strain on the raw ma­te­ri­als and in­ter­me­di­ates mar­kets to keep up with de­mand.

LNPs are cru­cial for de­liv­er­ing those vac­cines be­cause they help pro­tect of­ten volatile mR­NA as it makes its way in­to hu­man cells. In re­sponse to chron­ic short­ages, drug­mak­ers are tap­ping con­tract man­u­fac­tur­ers to rapid­ly scale sup­ply.

In Feb­ru­ary, Ger­many’s BioN­Tech tapped Mer­ck KGaA to “sig­nif­i­cant­ly ac­cel­er­ate” pro­duc­tion for their LNP sup­ply agree­ment. Mer­ck KGaA said it’s han­dling con­tract work for around 50 sep­a­rate vac­cine pro­grams al­though not all of those use LNP.

George Scangos (L) and Marianne De Backer

Pi­o­neer­ing biotech icon George Scan­gos hands in his re­tire­ment pa­pers — and this time it’s for re­al

George Scangos, one of the all-time great biotech CEOs, says the time has come to turn over the reins one last time.

The 74-year-old biotech legend spent close to three decades in a CEO post. The first was at Exelixis — which is still heavily focused on a drug Scangos advanced in the clinic. The second “retirement” was at Biogen, where he and his team were credited with a big turnaround with the now fading MS blockbuster Tecfidera. And the third comes at Vir, where he traded in his Big Biotech credentials for a marquee founder’s role back on the West Coast, hammering out a Covid-19 alliance with Hal Barron — then R&D chief at GSK — and breaking new ground on infectious diseases with some high-powered venture players.

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Jeanne Loring, director of the Center for Regenerative Medicine (Credit: Jamie Scott Lytle)

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last July, Jeanne Loring stood on a dirt road surrounded by Florida swampland and watched as a nearby SpaceX rocket blasted into the sky. The payload included a very personal belonging: cell clusters mimicking parts of her brain.

For more than two decades, Loring has been at the forefront of a stem cell field that always seems on the brink of becoming the next thing in medicine, but has been slow to lift off.

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In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

Elon Musk (GDA via AP Images)

Neu­ralink em­ploy­ees cite lay­offs at Elon Musk’s brain-com­put­er in­ter­face start­up

At least two Neuralink employees have posted to LinkedIn in recent days saying they’ve been laid off from Elon Musk’s brain-computer interface startup, which has received backlash for animal testing.

A former staffer working on preclinical study design and an ex-lab director working on assessing the safety of Neuralink’s implanted devices (prior to human testing) announced recently they’d been laid off, specifically using that terminology. Both had worked at the startup for at least two years.

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Matthew Stober, newly-appointed Abzena CEO (Abzena)

Abzena lays off more than 60 em­ploy­ees in Cal­i­for­nia

Contract manufacturer Abzena has permanently laid off 66 employees at one of its San Diego sites, marking the latest in a string of layoffs spanning the biotech industry.

The layoffs took effect on Jan. 11 at the company’s 8810 Rehco Road site, according to a WARN notice filed with the Employment Development Department of California.

Abzena does have another location in the San Diego area, where the manufacturer invested $60 million and added 50,000 square feet in 2020. Endpoints News reached out to Abzena but has not received a response as of press time.

#JPM23: What's re­al­ly dri­ving the cost of health­care and drugs in 2023?

Executive Editor Drew Armstrong spoke with PhRMA CEO Steve Ubl, EmsanaRx CEO Greg Baker and ICER President Steve Pearson about how the debate over drug costs has changed (or not) in the last decade, the shifting payer landscape and why there seems to be so little movement on drug rebates. This transcript has been edited for brevity and clarity.

Drew Armstrong:

So first of all, thank you to everybody for being here and for our panel for being here. Incredibly excited to have this discussion on the cost of healthcare and drugs and what’s driving that. We’re here with Steve Ubl, the head of PhRMA. Thank you so much. Steve Pearson from ICER, and Greg Baker from EmsanaRx. I want to start this conversation with a little bit of a personal reminiscence. So about almost 10 years exactly. I was a reporter back in my previous job and I was covering drug pricing and Gilead had just launched their hepatitis C drug and I was having a conversation with another Steve over at Express Scripts and he made some comments essentially about how they intended to launch a price war over hepatitis C therapies.

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Pa­tient death spurs tri­al halt for Ma­gen­ta Ther­a­peu­tics

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.