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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).