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

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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News brief­ing: No­var­tis' Zol­gens­ma hits block­buster sta­tus, trig­ger­ing $80M mile­stone; Caris rais­es $310M for ge­nom­ic pro­fil­ing ex­pan­sion

Novartis reported that Zolgensma earned $291 million in Q3, which puts the SMA gene therapy squarely in the blockbuster category. That, in turn, is paying benefits to Regenxbio, which provided the NAV AAV9 vector used by Zolgensma.

The biotech $RGNX reported Tuesday morning that it is getting an $80 million milestone payment from Novartis, which now has earned more than $1 billion cumulatively from the new gene therapy franchise. And with large percentages of newborns now being screened for the rare, lethal ailment, the franchise can continue to grow.

Cedric Francois, Apellis CEO (Optum via YouTube)

UP­DAT­ED: So­bi bets $250M cash, about $1B in mile­stones for rights to a C3 ther­a­py be­ing pushed through 5 piv­otal tri­als

A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.

The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Image: Shutterstock

Covid-19 roundup: Sanofi and GSK pledge 200M vac­cine dos­es for a glob­al dis­tri­b­u­tion cam­paign

Sanofi and GSK have agreed to give 200 million doses of their vaccine candidate to the COVAX Facility, which is part of a program set up by CEPI, the WHO and Gavi to equitably distribute vaccines around the world.

The idea behind COVAX is to give all participating countries equal access to vaccines, regardless of income level. As of Oct. 14, more than 180 countries had signed agreements to the COVAX Facility, including France and the UK. China joined earlier this month, pledging to make its vaccines a “global public good.” One country notably off the list is the United States.