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.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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Joel Dudley, new partner at Innovation Endeavors (Tempus Labs)

For­mer Google CEO’s VC is mak­ing a big­ger push in­to the biotech world, hir­ing promi­nent Ther­a­nos skep­tic

Venture capital firm Innovation Endeavors has mainly had its focus on investments across the tech space, but it has been slowly turning its attention to the biotech world. Now, a new partner is coming into the fold showing that its interest in biotech is likely to grow further.

The Silicon Valley-based company, which is headed up by former Google CEO Eric Schmidt, has brought on Joel Dudley as a partner. According to Dudley’s LinkedIn page, he is joining Innovation Endeavors after serving as the chief science officer of biotech startup Tempus Labs from 2020.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.