Stu­art Schreiber’s bid to tack­le huge class of cru­cial pro­teins nabs an­oth­er $50M

Four years af­ter launch­ing with pres­ti­gious co-founders and am­bi­tions to go af­ter a fam­i­ly of an­cient and cru­cial pro­teins, it’s still not clear how far Jnana Ther­a­peu­tics has ad­vanced. But it’s pushed far enough that in­vestors are will­ing to in­fuse more cash.

Jnana an­nounced a $50 mil­lion Se­ries B on Wednes­day led by RA Cap­i­tal. Al­though light com­pared to the megarounds that have be­come in­creas­ing­ly rou­tine for biotechs, it con­tains the same melange of A-list in­vestors that backed the com­pa­ny’s sci­en­tists from the be­gin­ning, in­clud­ing Po­laris, Ver­sant, Ab­b­Vie and Pfiz­er.

Stu­art Schreiber

Found­ed by Ver­tex co-founder Stu­art Schreiber, a cou­ple of his col­leagues at the Broad In­sti­tute, and Bris­tol My­ers vet Joel Bar­rish, Jnana’s big idea was to go af­ter SLCs, a fam­i­ly of about 450 pro­teins that traf­fic metabo­lites in­to and out of cells. Al­though drug de­vel­op­ers had tar­get­ed these re­cep­tors in­di­vid­u­al­ly — SGLT2 in­hibitors, a high­ly ef­fec­tive class of di­a­betes drugs, for ex­am­ple —no one had ever tried to go af­ter them com­pre­hen­sive­ly.

“What’s made them re­al­ly hard to go af­ter from a drug­ging per­spec­tive is they’re in­cred­i­bly di­verse, one from the oth­er,” CEO and co-founder Joanne Kotz told End­points News. “They’re di­verse in terms of their struc­ture, a wide va­ri­ety of struc­tures with­in the fam­i­ly. They’re di­verse in terms of their sub­strate; huge va­ri­ety of metabo­lites, all the way from cho­les­terol to iron.”

There was al­so the prob­lem that the pro­teins are bound to a mem­brane, so they couldn’t be eas­i­ly iso­lat­ed to study in a dish. Jnana came up with a cou­ple tech­no­log­i­cal so­lu­tions: It tried to study di­rect­ly in live cells, in­stead of iso­lat­ing them. And, rather than im­me­di­ate­ly look for small mol­e­cules that in­hib­it a giv­en pro­tein, its sci­en­tists first come up with “binders” that latch on any­where.

“The ad­van­tage this gives us is that we can de­vel­op a com­mon ap­proach to look­ing at bind­ing that you can move from one to the next,” Kotz said.

So far the fruits of those ef­forts re­main un­clear. The team has won high-pro­file part­ners, snar­ing a col­lab­o­ra­tion with Neu­ro­crine to go af­ter SLCs in the cen­tral ner­vous sys­tem, and a $40 mil­lion up­front, $1 bil­lion mile­stone deal with Roche to go af­ter re­cep­tors rel­e­vant for neu­rol­o­gy and im­munol­o­gy.

All of the com­pa­ny’s pro­grams, though, re­main pre­clin­i­cal and ex­ec­u­tives have yet to say pre­cise­ly how pre­clin­i­cal they are. With the fi­nanc­ing on Thurs­day, Jnana dis­closed that its lead pro­gram would be in PKU, a rare ge­net­ic dis­ease caused by an over­abun­dance of a mol­e­cule called pheny­lala­nine (Phe) in the blood; Jnana will look to block a re­cep­tor in kid­ney cells that re­ab­sorbs Phe, there­by al­low­ing more of the metabo­lite to fil­ter out in­to the kid­ney.

Kotz, though, de­clined to say how far along the pro­gram was or if they yet had a lead mol­e­cule.

“What I can say is that our Se­ries B fund­ing is suf­fi­cient to get the PKU pro­gram in­to the clin­ic,” she said.

Mean­while, the com­pa­ny is now look­ing to go be­yond SLCs and in­to oth­er class­es. Kotz claimed they had de­vel­oped a plat­form suf­fi­cient­ly that they can quick­ly build small mol­e­cule binders to vir­tu­al­ly any pro­teins, and then ei­ther try to in­hib­it or de­grade those pro­teins. She sees it as part of a wave of re­newed in­ter­est from phar­ma in new small mol­e­cule tech­nolo­gies, point­ing to Bay­er’s re­cent $2 bil­lion buy­out of Vi­vid­ion.

“We joke in­ter­nal­ly that SLCs as a tar­get class are so di­verse, so chal­leng­ing, that in or­der to build a plat­form to drug any SLC, we had to build what we laugh­ing­ly re­fer to as a Fer­rari en­gine,” she said. “A plat­form that re­al­ly has the ca­pa­bil­i­ty to drug any pro­tein.”

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.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Alaa Halawa, 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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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Quince Ther­a­peu­tics faces takeover bid from share­hold­er Echo Lake Cap­i­tal

A bid to take over the biotech Quince Therapeutics has been put forward by one of its shareholders.

On Tuesday, Echo Lake Capital sent a letter to Quince’s board of directors putting forth a proposal to acquire all the biotech’s stock for $1.60 per share, which would value a takeover at around $58 million.

In the letter, Echo Lake said that it believes Quince’s stock is severely undervalued and that no drugs are being actively marketed or developed that require cash expenditures. It’s trading below the value of its assets, Echo Lake said.

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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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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Genen­tech to stop com­mer­cial man­u­fac­tur­ing at Cal­i­for­nia head­quar­ters

Genentech is halting commercial manufacturing at its California headquarters — and laying off several hundred employees.

The move is the result of a decision Genentech made in 2007 to relocate manufacturing operations from its South San Francisco headquarters location to other facilities or move the work to CDMOs, said Andi Goddard, Genentech’s SVP of quality and compliance for pharmaceutical technical operations, in an interview with Endpoints News. Genentech has made changes in capabilities and invested more in technology, so it doesn’t need as many large-scale manufacturing facilities as it did in the past, she said.

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In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.