Joe Biden, AP Images

Biden 2022 bud­get wish list calls for Medicare ne­go­ti­a­tions on drug prices, new $6.5B NIH agency

Pres­i­dent Joe Biden late last week re­leased his FY 2022 bud­get plan, call­ing for Medicare to ne­go­ti­ate pay­ment for cer­tain high-cost drugs, in ad­di­tion to the cre­ation of a new NIH agency to de­vel­op next-gen bio­med­ical break­throughs, and a slight in­crease in FDA fund­ing.

“The Pres­i­dent sup­ports re­forms that would bring down drug prices by let­ting Medicare ne­go­ti­ate pay­ment for cer­tain high-cost drugs and re­quir­ing man­u­fac­tur­ers to pay re­bates when drug prices rise faster than in­fla­tion,” the bud­get says, of­fer­ing few de­tails on how to ac­com­plish these re­forms but not­ing that they could “yield over half a tril­lion in Fed­er­al sav­ings over 10 years.”

House En­er­gy & Com­merce Com­mit­tee chair Frank Pal­lone (D-NJ) praised the an­nounce­ment on rein­ing in drug prices, adding, “The days of Amer­i­cans pay­ing three to four times more than peo­ple in oth­er coun­tries for the same pre­scrip­tion drugs are num­bered.”

While the over­all plan of­fers most­ly a high-lev­el wish list for Con­gress to mull over as it de­cides how to fund the gov­ern­ment next year, both FDA and NIH pro­vid­ed de­tails on what they’re look­ing in­to.

Chief among the new ad­di­tions is a re­quest for $6.5 bil­lion to cre­ate a new, NIH-based Ad­vanced Re­search Pro­jects Agency for Health (ARPA-H), tasked with de­vel­op­ing new ways to help pre­vent, de­tect, and treat dis­eases like can­cer, di­a­betes, and Alzheimer’s. While there may be some crossover be­tween this agency and oth­er NIH re­search at the NCI and else­where, the new agency will use term-lim­it­ed pro­gram man­agers and a fed­er­al ad­vi­so­ry pan­el for in­ter­a­gency co­or­di­na­tion and idea gen­er­a­tion.

“Po­ten­tial ar­eas of trans­for­ma­tive re­search dri­ven by ARPA-H in­clude an ‘in­no­va­tion fun­nel’ for ac­cu­rate, wear­able, am­bu­la­to­ry blood pres­sure tech­nol­o­gy, prepa­ra­tion of mR­NA vac­cines against com­mon forms of can­cer, or ac­cel­er­at­ing de­vel­op­ment of ef­fi­cient gene/drug de­liv­ery sys­tems to tar­get any or­gan, tis­sue, or cell type – a zip code for the hu­man body,” NIH said.

Mod­eled af­ter the De­fense Ad­vanced Re­search Pro­jects Agency (DARPA), the new agency could be a boon for the bio­phar­ma in­dus­try, as NIH ex­plains most of the fund­ing from ARPA-H will go to in­dus­try, uni­ver­si­ties, and non­prof­it re­searchers.

As the NIH seeks to add this new $6.5 bil­lion agency, the FDA’s FY 2022 to­tal bud­get re­quest is for $6.5 bil­lion, which would be an in­crease of $343 mil­lion in bud­get au­thor­i­ty (out­side of user fees).

An ad­di­tion­al $75.9 mil­lion will sup­port the FDA’s da­ta mod­ern­iza­tion work, about $39 mil­lion will go to opi­oids-re­lat­ed ef­forts, an in­crease of $18.8 mil­lion will help ad­dress the FDA in­spec­tions de­layed by the pan­dem­ic, $5.6 mil­lion will go for more drug safe­ty sur­veil­lance and over­sight, and $4.7 mil­lion more will go to en­hanc­ing FDA’s abil­i­ty to sup­port and ex­pand health eq­ui­ty and health dis­par­i­ty ef­forts.

In terms of safe­ty sur­veil­lance, the FDA notes that it “cur­rent­ly faces sig­nif­i­cant chal­lenges to its abil­i­ty to main­tain an ef­fi­cient and ef­fec­tive post­mar­ket safe­ty sur­veil­lance pro­gram. Staffing lev­els have not kept pace with the in­creas­ing amount of post­mar­ket work from the grow­ing num­ber and com­plex­i­ties of re­cent ap­provals, and the in­creas­ing amount of da­ta need­ing re­view.”

FDA al­so says it will as­sem­ble a mul­ti­dis­ci­pli­nary team of reg­u­la­to­ry coun­sels, project man­agers, and sci­en­tists to craft a risk-based ap­proach to de­ter­min­ing what types of sci­en­tif­i­cal­ly valid in­for­ma­tion the agency re­quires in­dus­try to sub­mit, and how that in­for­ma­tion will be used.

“This ap­proach—which will be dy­nam­ic—may fac­tor in dif­fer­en­tial risks in­clud­ing those as­so­ci­at­ed with ac­tive phar­ma­ceu­ti­cal in­gre­di­ents, ex­cip­i­ents, or de­liv­ery mech­a­nisms. FDA’s goal with these ef­forts is to de­ter­mine which set of in­for­ma­tion we can both re­quire of in­dus­try and uti­lize in­ter­nal­ly to max­i­mize the val­ue of da­ta that we re­ceive, while con­sid­er­ing the le­gal, fi­nan­cial, and eco­nom­ic bur­dens im­posed on all stake­hold­ers. This team will evolve in­to a per­ma­nent, cross-dis­ci­pline func­tion ded­i­cat­ed to post-mar­ket safe­ty pol­i­cy,” the FDA said.

In terms of leg­isla­tive pro­pos­als, the agency seeks to fur­ther clar­i­fy the FDA’s au­thor­i­ty to re­quire in­for­ma­tion that would im­prove its abil­i­ty to as­sess phar­ma­ceu­ti­cal crit­i­cal in­fra­struc­ture as well as man­u­fac­tur­ing qual­i­ty and ca­pac­i­ty.

“For ex­am­ple, FDA is seek­ing to re­quire de­tailed drug list­ings on a quar­ter­ly ba­sis for fin­ished drug prod­uct or in-process ma­te­r­i­al, re­gard­less of whether they were di­rect­ly or in­di­rect­ly im­port­ed in­to the U.S.,” the agency says.

The FDA al­so notes that it cur­rent­ly has lim­it­ed au­thor­i­ty to re­quest records and oth­er in­for­ma­tion in ad­vance of or in lieu of drug in­spec­tions, and it seeks to ex­pand FDA’s au­thor­i­ty in this ca­pac­i­ty. The agency al­so seeks to amend the FD&C Act, to al­low for the dis­clo­sure of cer­tain non-pub­lic in­for­ma­tion to state, lo­cal, and US ter­ri­to­r­i­al gov­ern­ment agen­cies.

Mean­while, as gene and cell-based ther­a­pies con­tin­ue to be sub­mit­ted to the agency, the FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search is re­quest­ing $900,000 to ac­cel­er­ate ef­forts to mod­ern­ize and stream­line the re­view of these com­plex bi­o­log­ics. CBER al­so says it’s de­vel­op­ing a reg­u­la­to­ry pro­gram for in­di­vid­u­al­ized ther­a­pies and fos­ter­ing glob­al reg­u­la­to­ry con­ver­gence for cell and gene ther­a­pies.

To help that cause, NIH notes that it’s al­so work­ing to re­solve a pro­duc­tion bot­tle­neck for gene-based ther­a­pies as wait times to pro­duce vec­tor ther­a­pies that meet the man­u­fac­tur­ing stan­dards nec­es­sary for clin­i­cal tri­als are long, of­ten one to two years.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Rick Pazdur (via AACR)

FDA's on­col­o­gy head Rick Paz­dur de­fends the ac­cel­er­at­ed ap­proval path­way, claim­ing it is 'un­der at­tack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; 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.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

FTC pulls re­main­ing case against Ab­b­Vie; New EU clin­i­cal tri­als sys­tem com­ing in 2022; Abing­worth bets big on CymaBay

The Federal Trade Commission on Friday withdrew its remaining case against AbbVie after the Supreme Court declined to review a lower court’s ruling.

The punt by SCOTUS means that while the Illinois pharma company illegally blocked patients’ access to lower-cost alternatives to its testosterone drug AndroGel, the FTC will no longer be able to return about $500 million directly to AndroGel consumers.

Alan Hirzel, Abcam

Drug sup­pli­er Ab­cam brings a long­time col­lab­o­ra­tor in house as part of $340M buy­out pact

BioVision has supplied Abcam with research tools since 1999, and now the two are making it official as part of a merger unveiled Monday.

Abcam will buyout BioVision as part of a $340 million acquisition deal to bring aboard the supplier’s biochemical and cell-based assays for biological research, as well as recombinant proteins, antibodies and enzymes.

The deal will give Abcam control of BioVision’s portfolio and allow for both the expansion of research existing areas of focus such as oncology, neuroscience and epigenetics and preparation to expand into new products. As a part of the deal, Abcam will develop and supply products and services to NKY, the previous owner of BioVision and receive support for ongoing development and commercialization of in vitro diagnostic products.