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.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Scynex­is takes out $60M loan ahead of ex­pect­ed ap­proval; Com­pass Ther­a­peu­tics ac­quires new bis­pe­cif­ic an­ti­body in buy­out

As the PDUFA date for their vaginal yeast infections quickly approaches, Scynexis is taking out a loan to ensure it can hit the ground running for a potential commercial launch.

Scynexis closed terms on a $60 million loan with Hercules Capital and Silicon Valley Bank, the New Jersey biotech announced Friday, with its ibrexafungerp candidate set for a June 1 PDUFA. Approval of the program is anticipated, the company said, after which the oral antifungal will be branded as Brexafemme.

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).