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.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Alan Hirzel, Abcam

Drug sup­pli­er Ab­cam brings a long­time col­lab­o­ra­tor in house as part of $340M buy­out pact

BioVision has supplied Abcam with research tools since 1999, and now the two are making it official as part of a merger unveiled Monday.

Abcam will buyout BioVision as part of a $340 million acquisition deal to bring aboard the supplier’s biochemical and cell-based assays for biological research, as well as recombinant proteins, antibodies and enzymes.

The deal will give Abcam control of BioVision’s portfolio and allow for both the expansion of research existing areas of focus such as oncology, neuroscience and epigenetics and preparation to expand into new products. As a part of the deal, Abcam will develop and supply products and services to NKY, the previous owner of BioVision and receive support for ongoing development and commercialization of in vitro diagnostic products.

Anthony Sun, Zentalis and Zentera CEO (Zentalis)

With clin­i­cal tri­als lined up for Zen­tal­is drugs, Chi­na's Zen­tera sets its sights on more deal­mak­ing and an IPO

As Zentalis geared up for an AACR presentation of early data on its WEE1 inhibitor earlier this year, its Chinese joint venture Zentera wasn’t idle, either.

Zentera, which has headquarters in Shanghai, had already nabbed clearance to start clinical trials in China for three of the parent company’s drugs. In May — just a month after Zentalis touted three “exceptional responses” out of 55 patients for their shared lead drug, ZN-c3 — it got a fourth CTA approval.

Thomas Soloway, T-knife CEO

What hap­pens when you give a mouse a hu­man self-anti­gen? In­vestors bet $110M to find out

T-knife Therapeutics launched last August on a mission to isolate T cell receptors not from human donors, but from mice. Now, with a new CEO and a candidate bound for the clinic, the Versant-backed company is reloading with a fresh $110 million.

“What we are trying to do for the field of TCR therapy and solid tumor therapy is very analogous to what the murine platforms have done in antibody development,” CEO Thomas Soloway told Endpoints News. 

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Tib­so­vo clears an­oth­er hur­dle for Servi­er, but can it make Agios' old drug prof­itable?

When European regulators saw the data Agios used to win US approval for their AML drug Tibsovo, they sent the more than decade-old biotech back to the drawing board. A single, single-armed trial was not going to cut it.

On Monday, though, the drug’s new owners announced it had cleared a more rigorous study. In a randomized, Phase III trial of certain newly diagnosed patients, those who received a combination of Tibsovo and chemotherapy lived longer than those who received a combination of placebo and chemotherapy. Those patients also had higher response rates and complete remission rates.

UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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