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.”

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Andrew Phillips, Nexo Therapeutics CEO

Scoop: Ver­sant, NEA launch new biotech helmed by ex-CEO of pro­tein de­grad­er C4 Ther­a­peu­tics

Long-time biotech venture firms Versant and New Enterprise Associates are backing a new startup run by former C4 Therapeutics chief executive Andrew Phillips.

The fledgling biotech has raised at least $30 million so far, according to paperwork filed with the SEC this week. The round could balloon to $60 million.

Phillips, who left protein degradation startup C4 in 2020 to be a managing director at Cormorant Asset Management, is running the show of the new venture as president, the SEC filing outlines. He also served as interim CEO of Cormorant-backed and Hansoh Pharmaceutical-partnered Blossom Bioscience last year.

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J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.