Fresh off pos­i­tive Nu­plazid da­ta, Aca­dia adds new clin­i­cal drug from Van­der­bilt in mile­stone-heavy deal

Steve Davis

Aca­dia Phar­ma’s pri­ma­ry strat­e­gy over the last few years has been to get its con­tro­ver­sial Parkin­son’s drug ap­proved for oth­er dis­eases, in­clud­ing de­pres­sion and schiz­o­phre­nia. Now, for the first time in 2 years, the com­pa­ny is adding a new chem­i­cal to its pipeline.

Aca­dia has agreed to a mile­stone-heavy deal with Van­der­bilt Uni­ver­si­ty on a pro­gram to al­loster­i­cal­ly tar­get a re­cep­tor called mus­carinic M1, a po­ten­tial path to­ward treat­ing cer­tain cen­tral ner­vous sys­tem dis­or­ders, in­clud­ing Alzheimer’s and schiz­o­phre­nia. The deal will pay Van­der­bilt $10 mil­lion, with $515 mil­lion in po­ten­tial mile­stones.

“While the study of mus­carinic mod­u­la­tors has been an area of high in­ter­est in the treat­ment of CNS dis­or­ders, it has proved dif­fi­cult to sep­a­rate ef­fi­ca­cy from un­want­ed ef­fects,” Aca­dia CEO Steve Davis said in a state­ment. Van­der­bilt’s “ap­proach rep­re­sents a com­pelling op­por­tu­ni­ty for ACA­DIA to ad­vance new po­ten­tial ther­a­pies.”

Jef­frey Conn

The li­cens­ing deal cen­ters around the work of pro­fes­sor Craig Lind­s­ley and Van­der­bilt’s War­ren Cen­ter for Neu­ro­science Drug Dis­cov­ery di­rec­tor Jef­frey Conn, who from 2006 to 2019 re­ceived an­nu­al NIH grants to study chem­i­cals that ac­ti­vate the mus­carinic re­cep­tor as po­ten­tial an­ti-psy­chotics.

In 2011, the pair co-au­thored a pa­per in Bioor­gan­ic & Med­i­c­i­nal Chem­istry Let­ters de­scrib­ing the dis­cov­ery and lead op­ti­miza­tion of ML169. The chem­i­cal mod­u­lates the M1 re­cep­tor, bet­ter pen­e­trates the brain than oth­er sim­i­lar chem­i­cals and could be used in Alzheimer’s or schiz­o­phre­nia. Since then, they’ve pub­lished sim­i­lar stud­ies on sev­er­al dif­fer­ent mol­e­cules, not­ing their po­ten­tial ef­fi­ca­cy in the two dis­or­ders.

Craig Lind­s­ley

Aca­dia said they have al­ready start­ed a Phase I tri­al — which, based on con­flict-of-in­ter­est dis­clo­sures ap­pears to date back to at least 2018 — but they have not dis­closed which in­di­ca­tion the tri­al is in.

For Aca­dia, the deal comes sev­er­al months af­ter it fi­nal­ly got a break­through in its ef­fort to ex­pand the in­di­ca­tion for the Parkin­son’s drug Nu­plazid, with plans to for an sN­DA in de­men­tia-re­lat­ed psy­chosis af­ter a Phase III tri­al showed enough ef­fi­ca­cy to be halt­ed ear­ly.

The com­pa­ny is al­so test­ing Trofine­tide, which they li­censed from Neuren Phar­ma­ceu­ti­cals in 2018, in a Phase III tri­al for girls with Rett Syn­drome.

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Tim Walbert, Horizon Therapeutics CEO (via YouTube)

Hori­zon Ther­a­peu­tics in takeover talks with Am­gen, J&J, Sanofi as po­ten­tial buy­ers

Amgen, J&J’s Janssen and Sanofi are all in talks to acquire Horizon Therapeutics, the rare disease biotech disclosed late Tuesday.

Horizon confirmed “highly preliminary discussions” with those companies regarding a potential buyout offer after the Wall Street Journal reported takeover interest.

Although the company — which commands a market cap of close to $18 billion — emphasized that “there can be no certainty that any offer will be made for the Company,” shares $HZNP still surged 31% in after-hours trading to near $103, bringing it to the point where it started the year.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Bris­tol My­ers scraps gene ther­a­py deal with uniQure for car­dio­vas­cu­lar dis­eases

Bristol Myers Squibb is hitting the exit on a collaboration with a gene therapy biotech.

The Big Pharma company will no longer partner with uniQure on finding new treatments for cardiovascular diseases, the biotech reported to the SEC last week, following a rocky relationship that saw the pair break off an earlier agreement — before coming back to the table. The deal will officially terminate on Feb. 21, 2023.

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Sana, Codex­is lay off staff, reshuf­fle pipeline in bid to fo­cus cell ther­a­py, en­zyme en­gi­neer­ing work

As its market cap shrinks to a fraction of its heyday, flashy cell therapy startup Sana Biotechnology is laying off 15% of its staffers in a move to rejig the pipeline and restructure the company.

Sana is among a growing group of biotechs that, feeling the weight of a broader market downturn and seeing their shares tumble steadily, are tightening the purse strings and adjusting their focus. Also on Tuesday, Codexis, an enzyme engineering company based in California and now helmed by former Sierra Oncology CEO Stephen Dilly, announced it will reduce the workforce by 18%.

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Jeb Keiper, Nimbus Therapeutics CEO

PhI­Ib win puts Nim­bus one step clos­er to chal­leng­ing Bris­tol My­ers in TYK2

Bristol Myers Squibb might be the first to clinch an FDA approval for a TYK2 inhibitor, but Nimbus Therapeutics is out to prove that it has the best drug in the class. The biotech says it now has positive mid-stage data to back up those claims — although it’s saving the hard numbers for now.

Topline results from a Phase IIb study involving 259 patients with moderate-to-severe plaque psoriasis showed that Nimbus’ drug, NDI-034858, hit the primary endpoint of helping more patients achieve PASI-75 than placebo at 12 weeks.

John Carroll with David Chang, Allogene CEO (Credit: Jeff Rumans Photography)

Al­lo­gene takes the stage in New York to go deep on its off-the-shelf cell ther­a­pies — de­clar­ing a first for sol­id tu­mors

NEW YORK — In most cases, a biotech like Allogene would wait until the next big science conference to offer its latest series of snapshots of its data. But most biotechs aren’t like Allogene, where the veteran leaders from Kite garnered a substantial number of kudos over the years for their in-depth reviews of the company’s progress.

So on Tuesday, the leaders at Allogene converged on Manhattan once again to give a detailed breakdown of their latest steps forward, looking to stay out front in the busy off-the-shelf cell therapy arena, keep a clean bill of health on the safety front and prove that they can not only match the autologous pioneers they helped create but make the all-important leap into solid tumors. It’s another step forward in a journey that has a long way to go before even the first big regulatory finish lines appear on the track. But for CEO David Chang, who spent some time with me running through the data ahead of the Tuesday session, it all amounts to forward momentum toward the desired goal.

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