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

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

Anthony Fauci, AP Images

First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.