Daniel Nomura, Vicinitas Therapeutics founder

'Tip of the ice­berg': No­var­tis-part­nered UC Berke­ley lab churns out a start­up to tack­le pro­tein sta­bi­liza­tion

For the past decade, Daniel No­mu­ra’s lab at UC Berke­ley has been work­ing to find chem­i­cal ap­proach­es to drug the so-called “un­drug­gable,” and with the help of No­var­tis’ re­search arm for the past five years, he has built out the foun­da­tion for a new biotech in the pro­tein sta­bi­liza­tion field.

Vicini­tas Ther­a­peu­tics joins a small field of up­starts look­ing to flip the switch and fo­cus on sta­bi­liz­ing cer­tain pro­teins, rather than de­grad­ing them. Cys­tic fi­bro­sis is a good ex­am­ple of a dis­ease that could ben­e­fit from small mol­e­cules aimed at sta­bi­liz­ing the CFTR pro­tein, which is what No­mu­ra’s lab did in a re­cent Na­ture Chem­i­cal Bi­ol­o­gy pa­per, serv­ing as the ba­sis for Vicini­tas.

The biotech emerged Thurs­day morn­ing with $65 mil­lion in Se­ries A fi­nanc­ing, near­ly the same sized round that its main com­peti­tor in the sta­bi­liza­tion field, the apt­ly named Sta­blix Ther­a­peu­tics, launched with last June. A coastal chal­lenge could be brew­ing as the South San Fran­cis­co biotech thinks it can move in­to the dri­ver’s seat in a race against its Co­lum­bia Uni­ver­si­ty peer. Vicini­tas has named its plat­form the Deu­biq­ui­ti­nase Tar­get­ing Chimera, or DUB­TAC, where­as Sta­blix is call­ing its drugs RESTORACs.

“We are cer­tain­ly the first ones to pub­lish on a ful­ly syn­thet­ic small mol­e­cule-based DUB­TAC plat­form for tar­get­ed pro­tein sta­bi­liza­tion, and I be­lieve that re­al­ly puts us in a strong po­si­tion to be­come the lead­ing com­pa­ny in the space,” No­mu­ra told End­points News.

The pro­fes­sor serves as founder, sci­en­tif­ic ad­vi­sor to and board mem­ber of Vicini­tas, which is cur­rent­ly search­ing for a CEO af­ter hav­ing land­ed CSO Daniel Mar­quess, who pre­vi­ous­ly served in the same po­si­tion at Uni­ty Biotech­nol­o­gy.

In the pa­per, No­mu­ra’s lab was able to link its “deu­biq­ui­ti­nase re­cruiter on to the Ver­tex drug lumacaftor that helps to act as a chem­i­cal chap­er­one for CFTR to sub­se­quent­ly deu­biq­ui­ti­nate and sta­bi­lize the lev­els of the CFTR pro­tein, and al­so im­prove chlo­ride chan­nel func­tion in cys­tic fi­bro­sis cells,” the pro­fes­sor said. The two lead co-au­thors on the pa­per are sci­en­tists at the biotech, he added.

Com­pared to the degra­da­tion field’s PRO­TACs, No­mu­ra char­ac­ter­ized Vicini­tas’ DUB­TACs as “es­sen­tial­ly the op­po­site.” In a nod to the biotech’s name, he said the com­pa­ny’s small mol­e­cules “in­duce the prox­im­i­ty, or vicin­i­ty, of two pro­teins that usu­al­ly don’t in­ter­act in this case to sta­bi­lize the lev­els of aber­rant­ly de­grad­ed pro­teins.”

While cys­tic fi­bro­sis was one of the fo­cus­es of the lab’s pa­per, the lung and di­ges­tive dis­ease is not the on­ly area Vicini­tas will pur­sue. CF will serve as a “test­bed” for broad­en­ing the plat­form’s reach, which could go in­to can­cer, mono­genic dis­eases and hap­loin­suf­fi­cien­cy dis­eases, No­mu­ra said. The lat­ter in­volves hav­ing “on­ly one good copy of a gene, re­sult­ing in half the lev­el of the pro­tein and,” the pro­fes­sor ex­plained, “by slow­ing down the nat­ur­al turnover rate of that pro­tein, you can po­ten­tial­ly in­crease the lev­el of that pro­tein and sub­se­quent­ly con­fer ther­a­peu­tic ben­e­fit.”

Deer­field Man­age­ment and a16z led the Se­ries A, with ad­di­tion­al back­ing from Droia Ven­tures, GV, The Mark Foun­da­tion for Can­cer Re­search and the Berke­ley Cat­a­lyst Fund. The biotech holds the ex­clu­sive li­cense to the work from No­mu­ra’s lab in con­junc­tion with No­var­tis. Deer­field is al­so a backer of an­oth­er No­mu­ra-found­ed biotech, Fron­tier Med­i­cines, a pro­tein degra­da­tion start­up al­lied with Ab­b­Vie.

Aside from Vicini­tas, No­mu­ra will con­tin­ue work­ing with the No­var­tis In­sti­tutes for Bio­Med­ical Re­search in a sec­ond re­search col­lab­o­ra­tion, al­so un­veiled Thurs­day. He will be part of a team that in­cludes fel­low UC Berke­ley pro­fes­sors Dean Toste, Thomas Mai­mone, Ziyang Zhang, and James Olz­mann. The aim of the new No­var­tis-Berke­ley Trans­la­tion­al Chem­i­cal Bi­ol­o­gy In­sti­tute is to find small mol­e­cule com­pounds that could lay the ground­work for new prox­im­i­ty-based ther­a­peu­tics.

“This whole space of in­duced prox­im­i­ty, we’re re­al­ly just at the tip of the ice­berg of the full po­ten­tial of that gen­er­al ap­proach, be­yond degra­da­tion and sta­bi­liza­tion,” No­mu­ra said.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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An­oth­er warn­ing let­ter for Lupin as FDA iden­ti­fies de­fi­cien­cies at In­dia-based site

With few new details of what needs fixing, Lupin disclosed last week that the FDA recently sent a warning letter to its Tarapur, India-based site.

After an inspection from March 22 to April 4, Lupin disclosed in an April stock filing that it received a Form 483 with four observations, but it didn’t offer any details on the observations.

Similar to comments made in April, the company said last week it does not believe the FDA slap will disrupt its drug supplies or the existing revenues from operations of this facility.

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