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.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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FDA ap­proves the third NSOMD drug in 18 months as Roche/Genen­tech beefs up its port­fo­lio of drugs for neu­ro­log­i­cal dis­or­ders

There were no FDA approved treatments for neuromyelitis optica spectrum disorder at the start of 2019. Now, as of Friday, there are three.

The latest entrant to the market is the Roche/Genentech drug satralizumab after US regulators gave it the thumbs up late Friday. An IL-6 inhibitor, the drug joins Alexion’s Soliris and AstraZeneca spinout Viela Bio’s Uplizna. The annual cost of satralizumab — which will hit the market as Enspryng — will be $190,000 for 13 doses, a Genentech spokesperson said, though the first year of treatment requires 15 doses and cost about $220,000.

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Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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