Alzheimer’s re­search in line for $350M boost as Sen­ate pro­pos­es $3B hike to NIH bud­get; Al­lo­gene teams up with Stan­ford re­searchers

→ Law­mak­ers are once again lin­ing up to boost NIH spend­ing, re­ject­ing Pres­i­dent Don­ald Trump’s re­peat­ed pro­pos­als to slice bil­lions out of their bud­get.

In an un­usu­al move, the Sen­ate ap­pro­pri­a­tions com­mit­tee re­leased their bill ahead of a vote in what was seen as an ef­fort to push things along be­fore the start of the new fis­cal year for the fed­er­al gov­ern­ment on Oc­to­ber 1. But there’s clear­ly sub­stan­tial sup­port in both par­ties for in­creas­ing the NIH bud­get by $3 bil­lion — $1 bil­lion more than the House has sup­port­ed so far.

The Sen­ate bill would raise the NIH bud­get to $42.1 bil­lion.

Alzheimer’s re­search, which has been a dis­as­ter zone in the clin­ic, would gain $350 mil­lion — bring­ing the to­tal close to $3 bil­lion. And there’s an­oth­er $50 mil­lion be­ing added to an­tibi­otics re­search. But the Sen­ate ver­sion al­so clips out mon­ey to sup­port a study on firearm in­juries sup­port­ed by House Dems.

If com­plet­ed, law­mak­ers will have raised the NIH bud­get 40% over the past 5 years. Last year Con­gress hiked the NIH bud­get by $2 bil­lion, af­ter sidelin­ing the ad­min­is­tra­tion’s at­tempt to find sav­ings there in­stead. Re­pub­li­cans and De­moc­rats — in­clud­ing ap­pro­pri­a­tions com­mit­tee chair Roy Blunt (R-MO) — have been push­ing for more mon­ey to ad­dress key re­search ar­eas which have strug­gled in re­cent years.

It’s un­like­ly a bud­get will be agreed to by the Oc­to­ber 1 dead­line, set­ting up an­oth­er stretch where the NIH would op­er­ate at cur­rent lev­els un­til the fi­nal num­bers are blessed — like­ly by the end of this year.

→ Al­lo­gene Ther­a­peu­tics — fo­cused on the de­vel­op­ment of al­lo­gene­ic CAR-T ther­a­pies for can­cer — and Stan­ford Uni­ver­si­ty are team­ing up to in­ves­ti­gate a nu­cle­ic acid de­liv­ery sys­tem that more ef­fec­tive­ly, safe­ly and flex­i­bly de­liv­ers in­tra­cel­lu­lar RNA or DNA in­to lym­pho­cytes, in­clud­ing T cells. The sys­tem was de­vel­oped by Stan­ford re­searchers.

→ Ad­di­tion­al da­ta were re­leased from blue­bird bio for its Phase II/III Star­beam clin­i­cal study (ALD-102) of Lenti-D gene ther­a­py for boys 17 years and un­der with cere­bral adrenoleukody­s­tro­phy (CALD) — a ge­net­ic and rapid­ly pro­gres­sive dis­ease that can lead to se­vere loss of neu­ro­log­ic func­tion and death.

“With the longest fol­low-up from the Phase 2/3 Star­beam study now up to five years, the da­ta show that all boys with CALD who were treat­ed with Lenti-D and were free of ma­jor func­tion­al dis­abil­i­ties (MFDs) at 24 months con­tin­ued to be MFD-free. Im­por­tant­ly, there were no re­ports of graft fail­ure or treat­ment-re­lat­ed mor­tal­i­ty, and ad­verse events were gen­er­al­ly con­sis­tent with mye­loab­la­tive con­di­tion­ing,” said David David­son, chief med­ical of­fi­cer of blue­bird bio.

In ad­di­tion, the com­pa­ny pre­sent­ed “up­dat­ed da­ta from the on­go­ing ob­ser­va­tion­al study (ALD-103) of al­lo­gene­ic hematopoi­et­ic stem cell trans­plant (al­lo-HSCT) in boys 17 years of age and un­der with CALD.”

Reuters has re­port­ed that Chi­na Bi­o­log­ic $CBPO, a de­vel­op­er of plas­ma-based ther­a­pies, has re­ceived a $4.59 bil­lion in cash take-pri­vate buy­out of­fer from a buy­er group which in­cludes Beach­head Hold­ings, CITIC Cap­i­tal Chi­na Part­ners IV, PW Medtech Group, Parfield In­ter­na­tion­al, HH Sum-XXII Hold­ings and V-Sci­ences In­vest­ments. The com­pa­ny, which was list­ed on the Nas­daq in 2009, had its shares jump 7% af­ter mar­ket. Reuters said, and “the of­fer of $120 per share rep­re­sents a pre­mi­um of 16.3% to Chi­na Bi­o­log­ic’s Wednes­day close of $103.10.” This isn’t the first deal re­ceived by the com­pa­ny. In Au­gust 2018, the com­pa­ny re­ject­ed a $3.9 bil­lion of­fer made by its for­mer CEO, David Gao.

GV-backed Broad spin­out Cel­sius Ther­a­peu­tics inked a num­ber of agree­ments — with the Park­er In­sti­tute for Can­cer Im­munother­a­py (San Fran­cis­co, Cal­i­for­nia), In­sti­tut Gus­tave Roussy (Paris, France) and the Uni­ver­si­ty Health Net­work (Toron­to, Cana­da) — to ac­cess tis­sue sam­ples from pa­tients re­ceiv­ing im­mune check­point in­hibitor ther­a­pies for triple-neg­a­tive breast can­cer, blad­der can­cer and kid­ney can­cer, re­spec­tive­ly.

→ En­docrine dis­ease-fo­cused Spruce Bio­sciences has re­leased pos­i­tive re­sults from its 12-week, Phase IIa study of tildac­er­font in adults with con­gen­i­tal adren­al hy­per­pla­sia.

The com­pa­ny said the study en­rolled pa­tients with clas­sic CAH ac­com­pa­nied by el­e­vat­ed an­dro­gens at base­line who were treat­ed with a dai­ly tildac­er­front pill over 12 weeks. Da­ta showed “mean re­duc­tions from base­line of 74% for ACTH, 82% for 17-OHP and 55% for A4. ACTH is the di­rect tar­get of tildac­er­font, while A4 is the crit­i­cal down­stream bio­mark­er used in clin­i­cal man­age­ment of pa­tients with CAH. Max­i­mum mean re­duc­tions ob­served at any time­point in the study were 84% for ACTH, 82% for 17-OHP and 79% for A4. In ad­di­tion, 60% of pa­tients with el­e­vat­ed, ab­nor­mal ACTH and 40% of pa­tients with el­e­vat­ed A4 saw re­duc­tions to nor­mal­iza­tion by week 12.”


With con­tri­bu­tion from John Car­roll

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

Endpoints Premium

Premium subscription required

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

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Mar­ket­ingRx roundup: Ab­b­Vie’s Hu­mi­ra TV turns fo­cus to HS skin con­di­tion; Sanofi amps par­ent­ing pol­i­cy

After years as the top spending pharma TV advertiser, AbbVie’s Humira brand finally downshifted earlier this year, ceding much of its marketing budget to up-and-coming sibling meds Skyrizi and Rinvoq. However, now Humira is back on TV with ads for another condition — Hidradenitis suppurativa (HS).

The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.

Sci­wind gets li­cens­ing deal with Sanofi for meta­bol­ic dis­ease can­di­dates; Im­mu­ni­ty­Bio and En­Gene­IC reach deal for nano cell tech

Sciwind Biosciences announced last night that it signed an expansive license agreement with Sanofi to develop and commercialize Sanofi’s long-lasting glucose-dependent insulinotropic polypeptide (GIP) receptor agonists. According to Sciwind, the lead candidate is currently under preclinical development.

The deal covers all applicable indications for the molecule. Sciwind plans to develop the lead GIP receptor agonist as part of combination with XWoo3, its GLP-1 peptide candidate which is currently being evaluated in Phase II studies to potentially treat diabetes, obesity and NASH.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.