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

Paul Hudson, Getty Images

Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

Kite Phar­ma's ex-CEO con­tra­dicts founder as CAR-T patent tri­al heats up, with con­flict­ing val­u­a­tions

Two days after Kite Pharma founder Arie Belldegrun told a federal courtroom that a meeting he had with a Memorial Sloan Kettering executive wasn’t about licensing their immunotherapy patent, Kite’s ex-CEO Aya Jakobovits said it was.

The admission came Tuesday during cross-examination in a patent infringement case that features two of the biggest cancer biotechs and some of the most well-known names in American medicine.

Jakobovits initially said she was not in attendance, didn’t know it was going to happen and didn’t know what took place, according to Law360. But then the plaintiff’s lawyer handed her a document – whose contents were not publicly revealed – and asked again if she learned after-the-fact that the meeting involved a potential patent license.

“Yes,” Jakobovits eventually said.

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On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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