Stéphane Bancel, AP Images

Mod­er­na takes on a low-risk pact with CAR-T play­er Au­to­lus for mR­NA-based can­cer drugs

Mod­er­na’s Covid-19 vac­cine has trans­formed the once-back­wa­ter biotech in­to one of the most high­ly val­ued drug­mak­ers in the world in the span of a year. But what does the fu­ture hold for Mod­er­na’s star turn? A small-scale dis­cov­ery pact could of­fer a clue.

Mod­er­na will hold ex­clu­sive rights to four mR­NA-based im­muno-on­col­o­gy can­di­dates us­ing pro­pri­etary bind­ing tech from Au­to­lus, a biotech best known for its work on “off-the-shelf” CAR-T ther­a­pies, the part­ners said Mon­day.

Un­like some oth­er dis­cov­ery pacts in­volv­ing big-name drug­mak­ers, the deal doesn’t come with up­front cash straight away. Au­to­lus will be due an up­front pay­ment for each can­di­date if Mod­er­na choos­es to opt-in with down­stream reg­u­la­to­ry and com­mer­cial mile­stones and roy­al­ty rights.

De­spite the scanty de­tails, shares of $AUTL were trad­ing up about 17% be­fore the bell.

It’s a low risk en­deav­or for Mod­er­na, which un­der the watch­ful eye of CEO Stéphane Ban­cel has seen its mar­ket cap ex­plode to near­ly $142 bil­lion amid the rise of its Covid-19 vac­cine and its plat­form po­ten­tial in oth­er in­di­ca­tions.

The pact, while mea­ger, could al­so of­fer some in­sight in­to how Mod­er­na is think­ing about its next steps, look­ing for out­side part­ners to help de­vel­op bet­ter ways to traf­fic mR­NA-based ther­a­peu­tics in­to tu­mor cells.

The linch­pin of the deal is the binders that Au­to­lus has tout­ed as a key tech­nol­o­gy. Al­though it didn’t dis­close the binders it’ll ex­plore with Mod­er­na, the com­pa­ny us­es a CAT binder in its CAR-T can­di­dates, a syn­thet­ic an­ti­body the biotech be­lieves has a short­er half-life than the stan­dard FMC63 binder used in oth­er ther­a­pies, po­ten­tial­ly lim­it­ing the se­ri­ous side ef­fects as­so­ci­at­ed with the class. In a note to clients, Mizuho an­a­lysts called the deal a “broad val­i­da­tion” of Au­to­lus’ plat­form if, ul­ti­mate­ly, a heav­i­ly qual­i­fied one.

But the tie-up will like­ly come as cold so­lace for mar­ket watch­ers wait­ing for if, or when, Mod­er­na will use its grow­ing cash po­si­tion to start ink­ing re­search deals or po­ten­tial­ly add a small­er biotech to the fold out­right. Dri­ven by grow­ing sales for its Covid-19 vac­cine and the val­i­da­tion of its mR­NA vac­cine plat­form writ large, Mod­er­na has seen its mar­ket cap sky­rock­et in re­cent days — now sur­pass­ing no less a light than old stal­wart Am­gen — with no end in sight to the growth.

How Mod­er­na will cap­i­tal­ize on that suc­cess to set it­self up in the fu­ture is still yet to be seen.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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UCB buys its way to epilep­sy show­down with Jazz with $1.9B Zo­genix ac­qui­si­tion

Zogenix’s epilepsy drug Fintepla may only have brought in around $100 million of sales in its first year, but UCB clearly believes it can go much, much higher.

The Belgian pharma has inked a $1.9 billion deal to buy out Zogenix, paying $26 per share in cash and offering a contingent value right worth $2 more per share if Fintepla lands an extra EU approval by the end of 2023.

But even the upfront marks a 72% premium to California-based Zogenix’s shares, which were trading just north of $15 on Tuesday.

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