Neu­rona Ther­a­peu­tics is dash­ing to the clin­ic with its cell ther­a­py for epilep­sy — but first, an­oth­er ven­ture round

Six years ago, a band of neu­ro­sci­en­tists from the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co com­bined decades of re­search and jumped in­to the hunt for an off-the-shelf cell ther­a­py. Now, that team is sprint­ing to­ward the clin­ic with a treat­ment for epilep­sy — but first, it’s mak­ing a pit stop at the ven­ture well.

Neu­rona Ther­a­peu­tics un­veiled a $41.5 mil­lion round on Tues­day morn­ing, bring­ing the San Fran­cis­co-based biotech’s to­tal raise to $135 mil­lion. The cash will be used to ad­vance the com­pa­ny’s pipeline, in­clud­ing an up­com­ing Phase I/IIa for its lead can­di­date, NRTX-1001, in chron­ic fo­cal epilep­sy.

“We see cell ther­a­py as a promis­ing ap­proach for chron­ic dis­or­ders of the ner­vous sys­tem for which cur­rent treat­ment op­tions are not op­ti­mal,” said UCB Ven­tures head Er­i­ca Whit­tak­er, who’s join­ing Neu­rona’s board of di­rec­tors, in a state­ment.

Cory Nicholas

The cen­tral ner­vous sys­tem con­tains ex­ci­ta­to­ry cells and in­hibito­ry cells, and nor­mal brain and spinal cord func­tion de­pends on a del­i­cate bal­ance be­tween the two, ac­cord­ing to Neu­rona. The bal­ance is nor­mal­ly es­tab­lished just af­ter birth — but the de­gen­er­a­tion of par­tic­u­lar CNS cell types can lead to the dys­reg­u­la­tion of neur­al cir­cuits, which can cause epilep­tic seizures, neu­ro­path­ic pain, spas­tic­i­ty and cer­tain types of cog­ni­tive im­pair­ments.

Neu­rona’s founders be­lieve that cer­tain types of neu­ron trans­plants can re­bal­ance ner­vous sys­tem ac­tiv­i­ty and re­pair neur­al cir­cuits. NRTX-1001 is de­rived from hu­man pluripo­tent stem cells, and com­pris­es in­terneu­rons that se­crete gam­ma-aminobu­tyric acid (GA­BA), an in­hibito­ry neu­ro­trans­mit­ter. It tar­gets seizure-on­set re­gions in the brain, ac­cord­ing to CEO Cory Nicholas, to re­store bal­ance to hy­per­ac­tive neur­al net­works.

The can­di­date is de­liv­ered to the hip­pocam­pus — the tis­sue most fre­quent­ly af­fect­ed in fo­cal epilep­sy — us­ing an MRI-guid­ed pro­ce­dure, ac­cord­ing to the com­pa­ny.

“We be­lieve there is an un­prece­dent­ed op­por­tu­ni­ty to cre­ate a nov­el class of ther­a­peu­tics with the po­ten­tial to per­ma­nent­ly re­pair dys­func­tion­al neur­al cir­cuits,” Tim Kutzkey, a part­ner at The Col­umn Group, said in a state­ment when the com­pa­ny launched with a $23.5 mil­lion Se­ries A round back in 2015.

The Col­umn Group re­turned to co-lead the newest round, along with new in­vestor UCB Ven­tures. Sphera Fund Man­age­ment, Alexan­dria Ven­ture In­vest­ments and Iron­fire Ven­tures chipped in.

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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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.

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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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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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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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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