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.

In a sec­ond big set­back for Covid-19 an­ti­body treat­ment hopes, Re­gen­eron halts en­roll­ment for more se­vere pa­tients

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The New York biotech said today that an independent monitoring committee recommended halting enrollment of patients who need high-flow oxygen or mechanical ventilation in one of the trials on their antibody cocktail, after finding “a potential safety signal” and “an unfavorable risk/benefit profile.” The news comes a week after the NIH scrapped a trial of Eli Lilly’s Covid-19 antibody after finding it was having little effect on an initial cohort of hospitalized patients.

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Daphne Koller, Getty

Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bristol Myers Squibb is turning to one of the star upstarts in the machine learning world to go back to the drawing board and come up with the disease models needed to find drugs that can work against two of the toughest targets in the neuro world.

Daphne Koller’s well-funded insitro is getting $70 million in cash and near-term milestones to use their machine learning platform to create induced pluripotent stem cell-derived disease models for ALS and frontotemporal dementia.

As­traZeneca sells off heart fail­ure and hy­per­ten­sion drugs to Chep­lapharm for $400M

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Cheplapharm paid $200 million for the European rights to Atacand (candesartan cilexetil) and Atacand Plus (candesartan cilexetil and hydrochlorothiazide) back in 2018. They’re now doubling that amount for commercial control in more than 70 countries.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Ar­cus and As­traZeneca part­ner on a high stakes an­ti-TIG­IT/PD-L1 PhI­II can­cer study, look­ing to im­prove on a stan­dard of care

For AstraZeneca, the PACIFIC trial in Stage III non-small cell lung cancer remains one of the big triumphs for AstraZeneca’s oncology R&D group. It not only made their PD-L1 Imfinzi a franchise player with a solid advance in a large niche of the lung cancer market, the study — which continues to offer data on the long-range efficacy of their drug — also helped salve the vicious sting of the failure of the CTLA-4 combo in the MYSTIC study.

No­var­tis buys a new gene ther­a­py for vi­sion loss, and this is one pre­clin­i­cal ven­ture that did­n't come cheap

Cyrus Mozayeni got excited when he began to explore the academic work of Ehud Isacoff and John G. Flannery at UC Berkeley.

Together, they were engaged in finding a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness, potentially restoring the vision of a broad group of patients. And they did it by using a vector to deliver the genetic sequence for light sensing proteins.

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Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized Covid-19 patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $812 million.

No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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News brief­ing: Ax­o­vant faces months of de­lay on lead Parkin­son's gene ther­a­py; Chi­nese CAR-T biotech nabs $100M

One of Axovant’s top gene therapy prospects for its second act is hitting a roadblock that could push its clinical timelines back by almost a year.

In an update, the biotech said it was informed about delays in CMC data and third-part fill-finish issues around mid-October by its manufacturing partner, Oxford Biomedica. Axovant has been developing a suspension-based process for the Parkinson’s drug; with that taking longer than expected, it now believes “it is unlikely that its planned randomized, sham-controlled trial of AXO-Lenti-PD will enroll patients by the end of calendar year 2021.”