Eric Ostertag, Poseida CEO

Take­da en­lists Po­sei­da in $3.6B+ non­vi­ral gene ther­a­py deal, though da­ta re­main years away from hu­man stud­ies

Take­da has been be­hind oth­er ma­jor play­ers in terms of cell and gene ther­a­py de­vel­op­ment, but af­ter an­nounc­ing a col­lab­o­ra­tion with Se­lec­ta Bio­sciences last week, it’s now on a bit of a spend­ing spree.

The Big Phar­ma has re­cruit­ed Po­sei­da Ther­a­peu­tics to de­vel­op in vi­vo gene ther­a­pies in six dif­fer­ent in­di­ca­tions, the com­pa­nies an­nounced Tues­day, with Take­da re­tain­ing an op­tion to ask for two more. Po­sei­da will get a $45 mil­lion up­front pay­ment in the deal, $125 mil­lion in short-term pre­clin­i­cal mile­stones and up to $435 mil­lion in clin­i­cal mile­stones per pro­gram.

If all eight can­di­dates hit all their check­points, Take­da will be on the hook for more than $3.6 bil­lion. Each of the pro­grams will be di­rect­ed at the liv­er and hematopoi­et­ic stem cells, with he­mo­phil­ia A be­ing the first tar­get.

Though last week’s Se­lec­ta deal was much nar­row­er in scope, fo­cus­ing on tamp­ing down the im­mune re­sponse seen in AAV-based gene ther­a­pies, Take­da’s Tues­day agree­ment is a bet on the fu­ture of gene ther­a­py, Po­sei­da CEO Er­ic Os­tertag told End­points News. The biotech’s gene ther­a­py plat­form is cen­tered around a non­vi­ral ap­proach, which Os­tertag says can pro­vide a true cure for ge­net­ic dis­eases.

Po­sei­da ar­gues it is dif­fer­ent from ear­li­er gene ther­a­py at­tempts be­cause that non­vi­ral ap­proach is bet­ter suit­ed at in­te­grat­ing in­to a pa­tient’s genome. Os­tertag said the pro­grams in Tues­day’s deal will use its pro­pri­etary nanopar­ti­cle tech­nol­o­gy to ed­it tar­get­ed genes in vi­vo, re­sult­ing in high­er ex­pres­sion lev­els, and give Po­sei­da the op­por­tu­ni­ty to go af­ter pe­di­atric pa­tients.

It’s an ap­proach un­like those be­ing in­ves­ti­gat­ed by oth­er com­pa­nies that he says are pri­mar­i­ly treat­ing adults, though Os­tertag did not name names.

“I think there are some ap­proach­es that as­pire to be sin­gle treat­ment cures. We’ve ac­tu­al­ly proven it with nu­mer­ous an­i­mal mod­els,” Os­tertag told End­points. “That is unique.”

As Os­tertag points out, how­ev­er, Po­sei­da has yet to put any of its gene ther­a­pies in hu­mans and like­ly won’t do so for sev­er­al years. The ear­li­est gene ther­a­py slat­ed for IND-en­abling stud­ies is an OTC de­fi­cien­cy pro­gram not in­volved in Tues­day’s deal shoot­ing for 2023, and even that isn’t en­tire­ly cer­tain — Po­sei­da is still choos­ing be­tween two dif­fer­ent tech­nolo­gies.

The lack of in­te­gra­tion seen in AAV-based gene ther­a­py and some nanopar­ti­cles-based ap­proach­es are “par­tic­u­lar­ly a prob­lem when you have a rapid­ly di­vid­ing tis­sue,” Os­tertag said. “Tran­sient ap­proach­es are not go­ing to work at all in that [pe­di­atric] pa­tient pop­u­la­tion.”

He lat­er added, “To date, the oth­er gene ther­a­py com­pa­nies are re­al­ly on­ly ad­dress­ing those few pa­tients that had mild dis­ease that made it in­to adult­hood. We can ad­dress the pe­di­atric pop­u­la­tion.”

Po­sei­da has large­ly made noise for its CAR-T pro­grams, pro­vid­ing proof of ef­fi­ca­cy for a next-gen sol­id tu­mor CAR-T in a da­ta read­out at the end of Au­gust. The re­sults came about 10 months af­ter the FDA re­moved a clin­i­cal hold, spurred by a pa­tient death on its lead can­cer drug.

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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