Carsten Brunn, Selecta Biosciences CEO

Has Se­lec­ta found a way around gene ther­a­py's de­liv­ery prob­lem? Take­da puts down $1B-plus to find out

While gene ther­a­pies were tra­di­tion­al­ly thought of as one-and-done treat­ments, sci­en­tists now see a ben­e­fit in re­dos­ing pa­tients down the road. The on­ly prob­lem? Some pa­tients mount an im­mune re­sponse to the ade­no-as­so­ci­at­ed virus­es (AAV) used to de­liv­er the ther­a­pies.

It’s one of gene ther­a­py’s most press­ing is­sues, and a raft of com­pa­nies are work­ing on non-vi­ral de­liv­ery meth­ods to get around it. But Se­lec­ta Bio­sciences thinks it’s found an­oth­er way to tamp down the body’s im­mune re­sponse — and Take­da is ready to dole out more than $1 bil­lion to get in on the ac­tion.

Take­da is putting down an undis­closed amount up­front and more than $1.1 bil­lion in biobucks to li­cense Se­lec­ta’s Imm­TOR plat­form for its two undis­closed gene ther­a­pies for lyso­so­mal stor­age dis­or­ders, the com­pa­nies said on Mon­day.

Se­lec­ta’s plat­form makes use of a biodegrad­able nanopar­ti­cle that en­cap­su­lates the im­muno­sup­pres­sive drug ra­pamycin, and is de­signed to be giv­en in con­junc­tion with gene ther­a­pies to in­duce anti­gen-spe­cif­ic im­mune tol­er­ance.

“Most of the safe­ty events [in the field] have been seen at the high­er dos­es,” CEO Carsten Brunn told End­points News. “So if you can give a low­er dose or mul­ti­ple low­er dos­es, that could re­al­ly trans­form the treat­ment reg­i­men.”

Brunn de­clined to com­ment on Take­da’s spe­cif­ic tar­gets or their stage of de­vel­op­ment. But he did say that Se­lec­ta filed an IND last week for its lead in-house gene ther­a­py pro­gram, a treat­ment for methyl­malonic acidemia (MMA) called MMA-101. Right be­hind that pro­gram is a treat­ment for or­nithine tran­scar­bamy­lase de­fi­cien­cy (OTCD), which Brunn said should be ready for an IND by the end of next year.

Back in April, AskBio hit the ex­it on a li­cens­ing deal for MMA-101 af­ter find­ing fault with a con­tract man­u­fac­tur­er’s work. Brunn said the prob­lem was a fill-fin­ish is­sue, which has since been re­solved.

Se­lec­ta is far from alone in the hunt for gene ther­a­py 2.0. An­jar­i­um Bio­sciences and GenEd­it both un­veiled hefty Se­ries A rounds last month to sup­port their non-vi­ral ap­proach­es. Glax­o­SmithK­line vet Bri­an McVeigh launched Code Bio­ther­a­peu­tics just a few months ago to find a non-vi­ral de­liv­ery method. And Gen­er­a­tion Bio closed a $230 mil­lion IPO last June to fund its pre­clin­i­cal push to treat dis­eases of the liv­er and reti­na.

“Se­lec­ta’s Imm­TOR plat­form is de­signed to mit­i­gate un­want­ed im­mune re­spons­es al­low­ing for re­dos­ing, which could have broad ap­plic­a­bil­i­ty across our gene ther­a­py pro­grams for a range of dis­eases,” Mad­hu Natara­jan, head of Take­da’s rare dis­eases drug dis­cov­ery unit, said in a state­ment.

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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