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.”

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Mathai Mammen (Rob Tannenbaum, Endpoints News at BIO 2018)

Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck five years ago, where the soft-spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The company calls it “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” the Boston Globe reported, quoting a spokesperson.

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Robert Califf, FDA commissioner (Tom Williams/CQ Roll Call via AP Images)

Hop­ing to ex­pand mon­key­pox vac­cine sup­ply, US paves the way for new route of ad­min­is­tra­tion

After making it clear that the US’ current monkeypox vaccine supply is insufficient, the FDA on Tuesday authorized a new route of administration that should increase the number of available doses by five-fold.

Regulators cleared Bavarian Nordic’s Jynneos vaccine for intradermal injection in adults older than 18. Unlike subcutaneous injection — the current method by which vaccine is delivered under the skin — an intradermal jab goes directly into the skin. It’s believed that this method requires less vaccine, since the dermis is rich in dendritic cells which specialize in taking up foreign antigens and presenting them to the immune system, according to Daniel Kuritzkes, chief of infectious diseases at Brigham and Women’s Hospital in Boston.

John Quisel, Disc Medicine CEO

Disc Med­i­cine goes pub­lic in re­verse merg­er with strug­gling Gem­i­ni Ther­a­peu­tics

After licensing a failed Roche schizophrenia drug last year, Disc Medicine is going public via a reverse merger with Gemini Therapeutics.

The combined company, while still named Disc Medicine, will trade under the stock symbol $IRON, in reference to Disc’s lineup of therapies for blood iron disorders. Alongside the merger, Disc has secured $53.5 million in another financing round, building on the $90 million Series B it raised in September.

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Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Samantha Du, Zai Lab CEO

Any­one still look­ing for a CD47? Zai Lab shelves PhI pro­gram af­ter re­view­ing 'com­pet­i­tive land­scape'

Over the past few years, the promise of blocking CD47 — a “don’t eat me” signal co-opted by cancer cells — has sent drugmakers big and small into a frenzy. But one biotech is now bowing out.

Zai Lab is deprioritizing ZL-1201, its CD47 inhibitor, scrapping plans for a Phase II trial. It will now “pursue out-licensing opportunities,” the company said in its Q2 update. The decision was based on a review of the competitive landscape, it added, without going into further details.

HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

US weighs new route of ad­min­is­tra­tion for mon­key­pox vac­cine as cas­es climb — re­port

Less than a week after HHS Secretary Xavier Becerra declared monkeypox a national health emergency, reports have emerged that the US plans to extend its vaccine supply by opting for a different route of administration.

Officials are expected to call for intradermal injection of Bavarian Nordic’s Jynneos vaccine — the only shot approved specifically for monkeypox in the US — as opposed to subcutaneous injection, unnamed sources told both the New York Times and Washington Post on Tuesday.

'Messy at best': Is the US re­peat­ing the same Covid mis­steps with mon­key­pox mes­sag­ing?

When Kyle Planck first suspected he might have monkeypox in late June, he went to the CDC website and found six photos of different types of lesions. And that was about it for general public information.

Planck, who is a sixth-year PhD pharmacology researcher at Weill Cornell, kept looking though and found a separate part of the CDC website meant for healthcare professionals. There he found a medical slide deck with more pictures, professional journal articles and more details about symptoms and diagnosis.

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