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

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

Seagen warns in­vestors against TRC Cap­i­tal’s lat­est 'mi­ni-ten­der of­fer'; BeiGene goes af­ter a new in­di­ca­tion for top PD-1 play­er

TRC Capital, which has selected various biotechs like Vertex and Biogen for the “mini-tender” treatment, jumped back into the game last month with an offer to buy shares in Seagen for $151. The problem, says Seagen, is that price was 4.28% lower than what the stock was selling for at the time they made the offer on Feb. 20, giving TRC a shot at an instant windfall.

So why sell for less than what it’s worth? Seagen notes warnings from regulatory authorities that these offers essentially try to trick investors into believing that they’re being offered a premium for the stock.

UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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Paul Hudson, Getty Images

How does Paul Hud­son's $13.5M comp pack­age stack up against oth­er CEOs? He's in the 'first quar­tile'

Paul Hudson arrived at Sanofi like a hurricane, chopping off duds in the pipeline, shaking up the C-suite, striking big M&A deals and jumping into the Covid-19 vaccine race — all in an attempt to reboot a pharma giant notorious for its setbacks.

Now, we’re getting a look at what the CEO brought home in his first year on the job.

When all is said and done, Hudson will have made about $6.7 million in 2020, about $2.5 million of which has already been paid. The bigger figure includes a $2.3 million bonus that’s subject to approval at an April meeting, and another $1.8 million in variable compensation that has yet to be paid.

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Af­ter three years of courtship (and turn­downs), Mer­ck pounced on the first glance of clin­i­cal da­ta in $1.85B Pan­dion takeover

It’s almost become cliché for biotech executives to talk about the importance of keeping your options open and being prepared to go all the way. But when it comes to negotiating with a giant like Merck, a little patience can indeed go a long way.

Just ask Pandion Therapeutics.

Days ago we already learned that Merck is shelling out $1.85 billion to pick up the biotech and its slate of autoimmune hopefuls. What we didn’t know until the SEC disclosure dropped Thursday is that the deal comes after Pandion turned down two other proposals from Merck over the past three years and held out until the last minute for a sweetened deal.

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