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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty


I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

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Raymond Stevens, ShouTi Pharma CEO

A new Schrödinger-backed start­up emerges from the sci­en­tist who mapped the first hu­man GPCR

One of the most popular targets in drug development, representing about a third of existing drugs, are G-protein coupled receptors — the tiny but integral membrane proteins responsible for recognizing things like light, taste, smell, hormones and pain.

But due to challenges in mapping their structure, the protein family remains largely unexplored.

A slate of companies has emerged over the last few years to change that. If one can figure out the structure of these elusive membrane receptors, it might be possible to create small molecule drugs that overcome the limitations of, say, biologic and peptide therapies. That promise is what gets serial entrepreneur Raymond Stevens out of bed in the morning.

Man­u­fac­tur­ing woes for No­vavax’s Covid jab bad­ly dis­rupt plans for roll­out to the poor — re­port

Production problems at a Novavax facility in Maryland have led to delays in the Covax vaccine sharing program. Now, a shortage of 1 billion doses is expected, as the supplier tries to navigate producing a shot up to regulators’ standards, Politico reported Tuesday.

The company has run into trouble with the purity of the vaccine. Novavax has had trouble proving it can produce a shot consistently up to standards, and it has caused significant delays in the rollout to low- and middle-income countries. This follows several delays at Novavax that has put the executive crew on the defensive.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)

Up­dat­ed: Bio­gen sells just $300K worth of Aduhelm in Q3, as ques­tions on long-term vi­a­bil­i­ty re­main

Barely anyone is accessing Biogen’s controversial Alzheimer’s treatment, with the company reporting just $0.3 million in Aduhelm sales in the third quarter. Although investors will be looking to the longer term, when CMS may decide to cover the drug and open the floodgates for more reimbursement, use of the drug is currently stalled.

Since June, when the FDA first signed off on the drug under its accelerated pathway, Biogen said Wednesday that it’s sold a total of $2 million worth of Aduhelm. That’s a far cry from the peak Wall Street sales estimate of about $9 billion in annual sales.

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With hun­dreds of mil­lions spent on failed ac­cel­er­at­ed ap­provals, re­searchers call for faster FDA with­drawals

Between 2017 and 2019, Medicare spent more than $220 million on cancer drugs for which the indications were either voluntarily pulled by their applicants or FDA’s oncology adcomm had recommended their withdrawal.

That kind of massive spending on cancer drugs lacking overall survival benefit is wasteful and risks harming people’s health, a research letter published in JAMA Internal Medicine on Monday said. The researchers from Harvard and the London School of Economics called on the FDA to move faster in both requiring timely postmarketing trials and accelerating the speed in pulling these dangling approvals when the confirmatory studies fail.

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Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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