Aca­dia adopts a new PhI­II game plan for 'break­through' med, dumps Alzheimer's PhII

Aca­dia $ACAD has de­cid­ed to change the game for its drug Nu­plazid (pi­ma­vanserin) go­ing fur­ther in­to Phase III, af­ter the FDA hand­ed out its break­through drug des­ig­na­tion for the ther­a­py.

Steve Davis, Aca­dia

Ap­proved al­ready for Parkin­son’s dis­ease psy­chosis, the biotech had been set­ting the stage for a piv­otal tri­al in Alzheimer’s dis­ease psy­chosis for their 5-HT2A ther­a­py. But in an­nounc­ing the launch of their HAR­MO­NY study, ex­ecs say now that they will re­cruit a mix of pa­tients with Alzheimer’s dis­ease, de­men­tia with Lewy bod­ies, Parkin­son’s dis­ease de­men­tia, vas­cu­lar de­men­tia and fron­totem­po­ral de­men­tia.

The move comes as reg­u­la­tors pro­vid­ed the BTD ti­tle to Nu­plazid as a treat­ment for dis­ease psy­chosis, an en­dorse­ment for eas­i­er ac­cess at the FDA that should help the biotech push through the next round of late-stage work.

At the same time, they’re scrap­ping a Phase II Alzheimer’s study af­ter post­ing some­what con­tro­ver­sial mid-stage da­ta with mixed re­sults. The drug hit at week 6 but failed to on the psy­chosis score at week 12.

The biotech says it de­cid­ed to stop the study be­cause it over­lapped with their Phase III sites. Per­haps not co­in­ci­den­tal­ly, it re­moves any chance of neg­a­tive da­ta squir­rel­ing their work in Phase III.

Shares at Aca­dia surged about 6% this morn­ing.

Leerink’s Paul Mat­teis added up the news and con­clud­ed that it pro­vid­ed a mixed pic­ture for the com­pa­ny at this stage.

On the plus side, FDA feed­back award­ing break­through des­ig­na­tion and al­low­ing for a sin­gle piv­otal study is en­cour­ag­ing, es­pe­cial­ly with re­spect to how the agency views pi­ma­vanserin safe­ty. More­over, if the study suc­ceeds, the ad­dress­able pop­u­la­tion could be very large, ren­der­ing how we cur­rent­ly mod­el the ADP rev­enue opp­ty con­ser­v­a­tive. How­ev­er, ACAD’s “bas­ket” ap­proach to this new ph3 may in­tro­duce ad­di­tion­al chal­lenges that are sim­ply dif­fi­cult to hand­i­cap: while the tri­al works to en­rich the place­bo-con­trolled por­tion for pi­ma­vanserin re­spon­ders, we see the in­clu­sion of such a het­ero­ge­neous pop­u­la­tion in a sin­gle piv­otal as a risky strat­e­gy.

“We are pleased the FDA has agreed to an ef­fi­cient de­vel­op­ment path for pi­ma­vanserin in this broad in­di­ca­tion and grant­ed Break­through Ther­a­py Des­ig­na­tion in recog­ni­tion of this se­ri­ous un­met need,” said Aca­dia R&D chief Serge Stankovic.

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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