Prax­is Pre­ci­sion Med­i­cines launch­es with $100M and bold sights on long-eva­sive neu­ro­log­i­cal dis­or­ders

It’s go­ing to be the era of neu­ro­science, Roche CEO Bill An­der­son de­clared on stage at the JP Mor­gan Health­care Con­fer­ence in Jan­u­ary. The field, he said, had “the po­ten­tial to be in the ‘20s what on­col­o­gy was for the last decade.”

Five months in­to that decade, a new biotech is emerg­ing from stealth mode with large in­vest­ments from Black­stone and two drugs al­ready in Phase II, one of them near­ing a piv­otal tri­al. Called Prax­is Pre­ci­sion Med­i­cines, since 2016 it’s raised $100 mil­lion — with No­vo Hold­ings, Vi­da Ven­tures and Even­tide al­so chip­ping in — to back a bet that, by find­ing the un­der­ly­ing cause of rare neu­ro­log­i­cal dis­eases, they could find and treat mech­a­nisms be­hind more com­mon ones.

For all An­der­son’s op­ti­mism about the fu­ture, though, Prax­is emerges at a tough time for neu­ro­science-fo­cused biotechs. Much of the rest of Big Phar­ma has all but aban­doned the field. The last ma­jor piece of neu­ro-news of the pre­vi­ous decade was the an­nounce­ment, in De­cem­ber, that Sage Ther­a­peu­tics’ vaunt­ed drug for ma­jor de­pres­sive dis­or­der had failed a large tri­al, a read­out that has since cost the com­pa­ny $6 bil­lion in mar­ket cap and in­duced them to cut more than half their staff. Prax­is’s lead drug goes af­ter the same in­di­ca­tion.

Mar­cio Souza

“It’s a chal­leng­ing field,” Prax­is CEO Mar­cio Souza ac­knowl­edged in an in­ter­view.

The com­pa­ny be­gan as an ef­fort to find de no­vo mu­ta­tions that caused epilep­sy — the spon­ta­neous ge­net­ic mal­func­tions that cause the dis­ease in pa­tients who did not in­her­it it from their par­ents. The search turned up, among oth­er things, a gene that af­fects cal­ci­um chan­nels in the brain. But in­stead of try­ing to fix that mu­ta­tion, they used that in­for­ma­tion to fig­ure out how that chan­nel — and how that chan­nel falling out of bal­ance, with neu­rons fir­ing too much or too lit­tle —played a role in oth­er dis­eases.

“What be­came quite clear is that when you were look­ing be­yond just the pure mu­ta­tions … we were all talk­ing about im­bal­ances in a giv­en part of the brain, and nor­mal­ly as it re­lates to a spe­cif­ic chan­nel,” Souza said. “A lot of peo­ple jumped in the past from mu­ta­tion — cor­rect­ing that mu­ta­tion, or cor­rect­ing the ge­net­ic de­fect. What we’re do­ing dif­fer­ent­ly is look­ing in­to how that man­i­fests and at­tempt­ing to cor­rect the ac­tu­al man­i­fes­ta­tion.”

The re­sult was PRAX-944, a T-type cal­ci­um chan­nel block­er that is in the ear­ly stages of de­vel­op­ment for rare forms of ge­net­ic epilep­sy, but which the com­pa­ny is more ag­gres­sive­ly tak­ing for­ward in es­sen­tial tremor, one of the more com­mon neu­ro­log­i­cal con­di­tions. They ex­pect to have Phase II proof-of-con­cept re­sults be­fore the end of the year.

The lead pro­gram, though, is PRAX-114, a de­pres­sion drug. The over­lap­ping in­di­ca­tions here are per­i­menopausal de­pres­sion, a rel­a­tive­ly rare con­di­tion com­pared with ma­jor de­pres­sive dis­or­der. De­spite the vast med­ical need and mar­ket, sci­en­tists have strug­gled for years lead­ing up to Sage’s flop to build bet­ter an­ti-de­pres­sants.

Prax­is hopes to have an an­swer on their drug soon, with plans to en­ter a piv­otal tri­al be­fore the end of the year. The drug works by al­loster­i­cal­ly tar­get­ing GABAa, the neu­ro­trans­mit­ter im­pli­cat­ed in a long list of dis­or­ders and tar­get­ed head-on by ben­zo­di­azepines. Souza said the new tri­al will try to track pa­tients as they would use it in their dai­ly lives, hop­ing to show what he says they saw in Phase II: a safe and quick drug.

“They have the right safe­ty pro­file,” Souza said. “Most of the is­sue is not on­ly with the ef­fi­ca­cy, but with the safe­ty of these com­pounds. And we think 114 has that bal­ance.”

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Alaa Halawaa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Flare Therapeutics biochemists Yong Li (L) and Valerie Vivat

A $123M Flare will get Third Rock on­col­o­gy biotech in­to the clin­ic this year

Flare Therapeutics will start its first human trial this year with an investigational urothelial cancer drug after pulling together a $123 million Series B from Big Pharmas, VCs and its incubator, Third Rock Ventures.

Launched in 2021 on the idea that a biotech could finally succeed at drugging the much-sought-after but stubborn transcription factor, Flare Therapeutics said Wednesday it is now primed for the clinic after closing its large financing haul earlier this year. The raise is a relatively stark figure in a tough startup financing environment but further buoys the upbeat signals coming out of other Third Rock biotechs in recent weeks, including the $200 million CARGO Therapeutics and $100 million Rapport Therapeutics rounds.

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No­var­tis touts sev­en years of dura­bil­i­ty da­ta for Zol­gens­ma

The same day that Roche touted positive durability and safety data for its spinal muscular atrophy drug Evrysdi, Novartis also made a splash with its multi-million dollar gene therapy for the disease.

Novartis rolled out interim data from two long-term follow-up studies Monday at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. In the first study, LT-001, all children in the trial that were treated after showing symptoms of SMA “maintained all previously achieved motor milestones” up to 7.5 years after being dosed. The average time since Zolgensma was given was 6.86 years.

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'Band­ing to­geth­er': 50 fe­male biotech ex­ec­u­tives lay out plans for board di­ver­si­ty, new com­pa­nies and men­tor­ing founders

Earlier this month, during the Silicon Valley Bank meltdown, Angie You recalled the speed with which female biotech CEOs were helping each other connect with bankers, get their wires through and assuage concerns during a financial implosion.

This past weekend, 50 of about 125 women who are part of that Slack group and a broader coalition self-dubbed the Biotech Sisterhood met in person in Cancun for the second rendition of an annual summit connecting female biotech CEOs. The attendance list doubled that of the inaugural gathering in Arizona 12 months ago.

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Rohan Palekar, 89bio CEO

89bio’s PhII da­ta add to quick suc­ces­sion of NASH read­outs as field seeks turn­around

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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Roche and Lil­ly team up to de­vel­op blood test to de­tect ear­ly signs of Alzheimer's

Eli Lilly is teaming up with Roche to help develop a blood test to detect early signs of Alzheimer’s disease and determine whether a patient should go for further confirmatory testing.

Roche’s Elecsys Amyloid Plasma Panel (EAPP) measures pTau 181 protein assay and APOE E4 assay in human blood plasma – elevations in pTau 181 are present in the early stages of Alzheimer’s, while the presence of APO E4 is the most common genetic risk factor for the disease.

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