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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Spe­cial re­port 2022: Meet 20 women blaz­ing trails in bio­phar­ma R&D

When you run a special report for a fourth year, it can start feeling a little bit like a ritual. You go through the motions — in our case opening up nominations for top women in biopharma R&D and reviewing more than 500 entries — you make your choices of inclusion and exclusion. You host a ceremony.

But then things happen that remind you why you do it in the first place. Perhaps a Supreme Court rules to overturn the constitutional right to abortion and a group of women biotech leaders makes it clear they strongly dissent; perhaps new data on gender diversity in the industry come out that look all too similar to the old ones, suggesting women are still dramatically underrepresented at the top; perhaps protests and conflicts around the world put in stark terms the struggles that many women still face in earning the most basic recognition.

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Prometheus moves to raise cash hours af­ter PhII da­ta leads to stock surge

After releasing better-than-anticipated data on two mid-stage studies Wednesday morning, Prometheus Biosciences’ CEO said the company would “take some time to assess” its next financing options.

It only needed about seven hours. Wednesday afternoon after the market closed, the biotech announced it would seek $250 million through an equity offering as the company looks to edge out anti-TL1A competitor Pfizer and its new partner Roivant.

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Mark McKenna, Prometheus Biosciences chairman & CEO

With clear PhII win in IBD, Prometheus thwarts Pfiz­er com­par­isons as it fol­lows Hu­mi­ra 'play­book'

Prometheus Biosciences reported a clear Phase II win in two inflammatory bowel disease conditions in a clinical development race with Pfizer, planting the biotech’s flag in a field of antibodies attempting to go against black box-cornered JAK inhibitors and AbbVie’s Humira.

Shares $RXDX have soared since the summer — a small dip last week notwithstanding when rival Pfizer teamed up with Roivant on a new company for their competing anti-TL1A monoclonal antibody. And they skyrocketed once again Wednesday morning, climbing from $36 apiece to more than $100 on the back of two Phase II studies: one placebo-controlled in ulcerative colitis and the other an open-label trial in patients with Crohn’s disease.

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Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Alex Martinez, Intrinsic Medicine CEO

They with­drew their IPO. Then, they broke off their SPAC merg­er. Now what?

If at first an IPO doesn’t succeed, try, try a SPAC. But what happens when that fails too?

Intrinsic Medicine and its blank-check partner Phoenix Biotech Acquisition Corp. called off their reverse merger Tuesday night, citing “current market conditions” as the reason it went kaput. The pair decoupled just weeks after agreeing to combine in late October as investors’ appetite for new IPOs and SPACs has been limited, at best.

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Just days be­fore pre­sent­ing at ASH con­fer­ence, Syn­dax out­lines plan to raise $150M

Syndax is banking that upcoming clinical data will be enough to woo investors — to the tune of $150 million.

The biotech kept it short in its announcement Tuesday afternoon, saying it is planning to sell $150 million worth of shares of its stock via a public offering. While no date or exact number of shares was given for the offering, the biotech noted it will be giving underwriters 30 days to purchase up to an additional 15% of shares sold in the public offering.

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Pen­ny stock pulls back on of­fer­ing up more of its shares amid ‘cur­rent mar­ket con­di­tions’

As some biotechs continue to rethink IPOs or SPAC deals in the current market, other companies are pausing on selling off more shares.

The Boston-based RNA biotech TransCode Therapeutics on Tuesday announced that it has withdrawn its S-1 filing with the SEC that it made in late November. TransCode said in a brief press release that the withdrawal is reflective of TransCode’s belief that “current market conditions are not conducive for an offering on terms that would be in the best interests of the company’s stockholders.”