Roche shelved these psy­chi­a­try drugs. So its for­mer head of neu­ro cooked up some new plans — and raised $59M for his start­up

As the for­mer head of neu­ro­science prod­uct de­vel­op­ment at Roche for a decade, George Garibal­di can per­son­al­ly vouch for the qual­i­ty of what the Swiss phar­ma gi­ant does. But hav­ing worked on psy­chi­atric dis­or­ders, he al­so knew way too well the down­side of a field where an­i­mal mod­els were a poor pre­dic­tion of how drugs would per­form in hu­mans.

George Garibal­di

So when he caught word that Roche was putting part of its psy­chi­a­try pipeline on the shelf, Garibal­di, by then run­ning his own con­sul­tan­cy, im­me­di­ate­ly took in­ter­est in li­cens­ing those com­pounds. He’d go down a slight­ly dif­fer­ent di­rec­tion, at a start­up even­tu­al­ly named Noe­ma Phar­ma.

“The sto­ry of Noe­ma is very sim­i­lar to Acte­lion in terms of how it start­ed,” CEO Lui­gi Cos­ta told End­points News, re­fer­ring to the biotech spun out from Roche with its top car­dio­vas­cu­lar re­searchers and pro­grams.

In par­tic­u­lar Sofinno­va Part­ners, which had backed Acte­lion’s launch round and since start­ed two oth­er com­pa­nies lever­ag­ing a sim­i­lar mod­el with Roche, helped build Noe­ma from scratch. Man­ag­ing part­ner An­toine Pa­piernik in­vit­ed Cos­ta to take it on as his first en­tre­pre­neur-in-res­i­dence project, and they put a plan to­geth­er with Garibal­di.

Sofinno­va is al­so co-lead­ing a $59 mil­lion Se­ries (CHF$54 mil­lion) Se­ries A with Po­laris Part­ners. Roche al­so chimed in, along­side Gilde Health­care, In­vus and Bio­Med Part­ners.

The fund­ing should take the biotech in­to read­outs of three Phase IIb stud­ies in or­phan neu­ro­log­i­cal dis­eases: seizures in tuber­ous scle­ro­sis com­plex, trigem­i­nal neu­ral­gia and Tourette syn­drome.

Lui­gi Cos­ta

NOE-101, a neg­a­tive mod­u­la­tor of mGluR5, cov­ers the first two in­di­ca­tions while a sec­ond, NOE-105, is de­signed to re­duce the in­vol­un­tary tics in Tourette pa­tients by in­hibit­ing PDE10A.

Both of those can­di­dates were al­ready in Phase IIb at Roche for psy­chi­atric dis­or­ders (a pre­vi­ous ef­fort to de­vel­op an mGluR5 drug for Frag­ile X had floun­dered). The oth­er two com­pounds are slat­ed for Phase II stud­ies lat­er in 2021.

In­stead of mod­u­lat­ing be­hav­ior, Noe­ma is try­ing to dri­ve changes in tan­gi­ble symp­toms that have a big im­pact on dai­ly func­tions.

“We’re di­rect­ing them to more neu­rol­o­gy, where it’s more dis­eases with clear symp­toms that you and I can see,” he said. “So seizures, some­thing that we can see. But in psy­chi­a­try, you don’t see what peo­ple think.”

That doesn’t nec­es­sar­i­ly make it eas­i­er. Yet some­what serendip­i­tous­ly, Garibal­di said, be­tween 2017 and 2019 a host of sci­en­tif­ic pa­pers pub­lished in­de­pen­dent­ly by labs had pro­vid­ed ev­i­dence that in­spired them to se­lect the lead in­di­ca­tions with con­fi­dence. Some, for in­stance, sug­gest­ed that block­ing the mGluR5 re­cep­tor could re­duce tu­mor growth as well as seizures, the two defin­ing fea­tures of TSC. An­oth­er showed that NOE-101 goes di­rect­ly in­to the nu­cle­us of spinal cord cells, which is key for chron­ic pain.

John Kemp, who had re­cent­ly left his role lead­ing Janssen’s neu­ro­science dis­cov­ery, joined ear­ly as CSO. The in­ter­nal team at Noe­ma to­tals on­ly six staffers — a size that al­lows them to be nim­ble and flex­i­ble.

The same phi­los­o­phy is in play as they tai­lor clin­i­cal tri­als against the back­drop of Covid-19. While hav­ing oral, small mol­e­cule drugs to ad­min­is­ter al­ready avoids some headaches, Noe­ma is still play­ing safe. The glob­al pro­gram will start, for now, in Aus­tralia, with an eye to min­i­miz­ing the num­ber of vis­its to clin­i­cal sites and max­i­miz­ing re­mote mon­i­tor­ing.

If the da­ta bear out — and we should have a first look by the end of next year — Garibal­di said Noe­ma won’t “ex­clude in the long term go­ing back to Roche” for more in-li­cens­ing deals.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

Mod­er­na los­es lat­est bat­tle in key vac­cine de­liv­ery patent fight as fed­er­al ap­peal falls flat

The US Court of Appeals for the Federal Circuit on Wednesday rejected Moderna’s attempt to overturn key patents related to the delivery vehicle for its Covid-19 vaccine after the biotech sought to preempt a potentially risky infringement lawsuit.

For years, Moderna has been battling a tiny Pennsylvania biotech known as Arbutus over patents for a technology required to deliver its mRNA drugs and vaccines, known as lipid nanoparticles or LNP. Moderna is concerned there’s a substantial risk that Arbutus will assert the ’069 patent in an infringement suit targeting Moderna’s Covid-19 vaccine, particularly as Arbutus has boasted of its patent protection and refused to grant a covenant not to sue Moderna.

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Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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What's fair? New ICER re­port shows pay­ers gen­er­al­ly en­sur­ing fair ac­cess to drugs

The nonprofit Institute for Clinical and Economic Review on Wednesday released a new report highlighting the ways in which payers are generally ensuring fair access to prescription drugs, even when based on a set of criteria set by the nonprofit.

While noting the lack of transparency hindered the report’s results, ICER said that the “great majority” of payer policies in the formularies evaluated are structured in a way to support many key elements of how ICER defines “fair access.”