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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

Blue­bird sends blood dis­or­der drug to FDA for ap­proval; CG On­col­o­gy en­ters col­lab­o­ra­tion with Roche for Tecen­triq

Bluebird bio announced it completed the rolling submission of its BLA to the FDA for betibeglogene autotemcel gene therapy.

The therapy, designed for patients with beta-thalassemia who require regular red blood cell transfusions, was previously granted breakthrough therapy designation for treating transfusion-dependent beta-thalassemia (TDT). If approved, beti-cel will be the first hematopoietic stem cell ex-vivo gene therapy for patients in the US.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.