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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Bris­tol-My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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