Galera claims pos­i­tive up­date in pan­cre­at­ic can­cer but does­n't re­port p-val­ues; Am­gen launch­es Cana­di­an fund in tan­dem with CCRM

Malvern, PA-based Galera Ther­a­peu­tics re­leased up­dat­ed fol­low-up da­ta Wednes­day af­ter­noon for a Phase I/II tri­al in pan­cre­at­ic can­cer, though it did not re­veal any p-val­ues as­so­ci­at­ed with the re­sults.

Ac­cord­ing to the com­pa­ny, af­ter six months of fol­low-up from 42 pa­tients with lo­cal­ly ad­vanced pan­cre­at­ic can­cer, the me­di­an over­all sur­vival was 20.1 months in the drug arm com­pared to 10.9 in the con­trol group for the GC4419 can­di­date. Ad­di­tion­al­ly, 29% of GC4419 pa­tients saw a par­tial re­sponse ver­sus just 11% on con­trol, Galera said.

Galera did not say whether these re­sults were sta­tis­ti­cal­ly sig­nif­i­cant. The com­pa­ny al­so high­light­ed that pos­i­tive re­sults were al­so ob­served in lo­cal tu­mor con­trol, time to metas­tases and pro­gres­sion-free sur­vival, but did not show any da­ta points or p-val­ues re­gard­ing these mea­sures.

Nev­er­the­less, in­vestors jumped for joy at the news, with Galera $GRTX shares trad­ing up near­ly 50% in pre-mar­ket move­ment Thurs­day.

“Fi­nal” re­sults from this Phase I/II pi­lot study af­ter a min­i­mum of one-year fol­low-up, Galera added, which should come in the sec­ond half of 2021.

Wednes­day’s news fol­lows in­ter­im da­ta from Oc­to­ber re­gard­ing the same pro­gram. That re­lease showed me­di­an OS hadn’t been reached in the drug arm com­pared to 38.7 weeks on con­trol, good for a p-val­ue of p=0.06. Galera al­so did not see any sta­tis­ti­cal­ly sig­nif­i­cant changes in PFS at the time, with that fig­ure’s p-val­ue clock­ing in at a measly p=0.29.

Am­gen launch­es Cana­di­an fund in tan­dem with CCRM

Am­gen is team­ing up with a Cana­di­an cell and gene ther­a­py out­fit to fund ear­ly-stage tech in the coun­try, the pair said Thurs­day.

Go­ing 50/50 with CCRM, Am­gen is look­ing to iden­ti­fy, de­vel­op and com­mer­cial­ize what they con­sid­er promis­ing tech­nolo­gies and ther­a­pies from re­search con­duct­ed in in­sti­tu­tions that form CCRM’s glob­al net­work. Con­tri­bu­tions will range from fi­nan­cial sup­port to in-kind tech­ni­cal ser­vices and ex­per­tise.

The pair will form a com­mit­tee with rep­re­sen­ta­tives from both part­ners to de­ter­mine which pro­pos­als will qual­i­fy for the pro­gram.

“There are few places in the world that have clus­tered all the nec­es­sary re­sources and tal­ent to dri­ve re­gen­er­a­tive med­i­cine from the bench to the bed­side. Cana­da has con­sis­tent­ly led the way for decades,” said Am­gen VP of re­search Alan Rus­sell in a state­ment.

CCRM is a glob­al, pub­lic-pri­vate part­ner­ship head­quar­tered in Cana­da that re­ceives fund­ing from the Cana­di­an gov­ern­ment, On­tario and oth­er aca­d­e­m­ic and in­dus­try part­ners. CCRM sup­ports the de­vel­op­ment of re­gen­er­a­tive med­i­cines and as­so­ci­at­ed en­abling tech­nolo­gies, with a spe­cif­ic fo­cus on cell and gene ther­a­py.

Mirum li­cens­es Chi­nese de­vel­op­ment of pru­ri­tus pro­gram to CAN­bridge

Mirum Phar­ma­ceu­ti­cals has en­tered in­to a new li­cens­ing agree­ment where CAN­bridge Phar­ma­ceu­ti­cals will com­mer­cial­ize the ex­per­i­men­tal cholesta­t­ic pru­ri­tus drug mar­al­ix­i­bat in Chi­na, Hong Kong, Macau and Tai­wan, the com­pa­nies said Thurs­day.

Mar­al­ix­i­bat is cur­rent­ly be­fore the FDA un­der pri­or­i­ty re­view, with Mirum shoot­ing for an in­di­ca­tion in cholesta­t­ic pru­ri­tus in pa­tients with Alag­ille syn­drome. The drug can­di­date tar­gets the api­cal sodi­um de­pen­dent bile acid trans­porter (AS­BT), ul­ti­mate­ly re­sult­ing in low­er lev­els of bile acid sys­tem­i­cal­ly, which could me­di­ate liv­er dam­age.

Un­der the deal, Mirum will re­ceive $11 mil­lion up­front, ad­di­tion­al R&D fund­ing and up to $109 mil­lion in fu­ture mile­stones. CAN­bridge has al­so agreed to over­see Mirum’s clin­i­cal study sites in Chi­na.

The drug is fur­ther be­ing stud­ied to treat cholesta­t­ic pru­ri­tus in pro­gres­sive fa­mil­ial in­tra­hep­at­ic cholesta­sis and bil­iary atre­sia, with a glob­al Phase IIb study in the lat­ter hav­ing re­cent­ly been launched.

Reper­toire and Yale to study caus­es of mul­ti­ple scle­ro­sis in new re­search agree­ment

Reper­toire Im­mune Med­i­cines has signed a new re­search agree­ment with Yale Uni­ver­si­ty to go in­to the depths of cel­lu­lar im­mu­ni­ty in mul­ti­ple scle­ro­sis, the duo said Thurs­day.

The Cam­bridge, MA-based biotech and Yale will try de­ter­min­ing what types of anti­gens are ac­ti­vat­ing T cells in pa­tients. They will work to­geth­er to iden­ti­fy the speci­fici­ty of var­i­ous sub­sets of T cells with a goal of un­der­stand­ing the im­muno­log­ic caus­es of MS.

To do so, the pair will ex­am­ine the cere­brospinal flu­id of pa­tients liv­ing with MS, look­ing for po­ten­tial caus­es of the dis­ease and how the anti­gens ac­ti­vate path­o­gen­ic T cells. Re­searchers from Yale will pro­vide hu­man T cell re­cep­tor se­quences to Reper­toire, which will use its pro­pri­etary plat­form to de­ter­mine the anti­gens that these TCRs iden­ti­fy.

“By un­der­stand­ing the im­mune codes in T cells from pa­tients with MS, we will un­der­stand the ba­sis of cel­lu­lar im­mu­ni­ty and hope to de­vel­op trans­for­ma­tion­al med­i­cines that no longer in­volve im­muno­sup­pres­sion,” Reper­toire R&D chief An­tho­ny Coyle said in a state­ment.

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.

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

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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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