Third Rock­'s Am­bys hauls $140M for liv­er dis­ease, ink­ing Take­da deal ahead of launch

It won’t shock any­one that a new com­pa­ny has rolled off Third Rock’s start­up con­vey­or belt (this VC firm is a ma­chine at cre­at­ing new com­pa­nies), but their lat­est ven­ture is leap­ing off the start line with a dis­tinct ad­van­tage: $140 mil­lion in launch mon­ey and a rare deal with Big Phar­ma part­ner Take­da.

The new com­pa­ny is called Am­bys Med­i­cines, chris­tened af­ter the Mex­i­can sala­man­der famed for re­gen­er­at­ing limbs: Am­bystoma mex­i­canum. You guessed it, the com­pa­ny will be work­ing in re­gen­er­a­tive med­i­cine.

Jeff Tong

I spoke with Third Rock’s ven­ture part­ner Jeff Tong, who’s serv­ing as in­ter­im CEO for the time be­ing, about the com­pa­ny’s tech. He said they’ve li­censed a smat­ter­ing of re­search from the labs of Am­bys’ sci­en­tif­ic founders, and are build­ing an in-house R&D unit led by a cou­ple of in­ter­im Third Rock vets sit­ting in as ex­ecs.

Mar­tin Burke

The com­pa­ny is tack­ling three dif­fer­ent av­enues in liv­er dis­ease: a cell ther­a­py plat­form, a gene ther­a­py, and gain-of-func­tion small mol­e­cules. Tong said it was im­per­a­tive that Am­bys pur­sue all three ar­eas at once. This is part of the rea­son they de­cid­ed to part­ner with Take­da — to get a big chunk of cash that would sup­port its am­bi­tious R&D plans.

“It will al­low us to pur­sue the three ar­eas si­mul­ta­ne­ous­ly,” Tong said. “They’re all am­bi­tious, and run­ning them in par­al­lel is very im­por­tant.”

Markus Grompe

He says many young biotechs make the fa­tal mis­take of fun­nel­ing all their cash in­to the first pro­gram that shows big promise. “It sucks all the re­sources out of the com­pa­ny, and then the oth­er pro­grams — even though they’re al­so promis­ing — die on the line,” he said.

So Am­bys and Third Rock de­signed a deal with Take­da that brought an in­fu­sion of cap­i­tal to the com­pa­ny’s launch. Here’s the de­tails: Take­da chipped in $100 mil­lion up­front (in­clud­ing par­tic­i­pat­ing in the com­pa­ny’s $60 mil­lion Se­ries A round), bring­ing Am­bys’ launch cash to $140 mil­lion to­tal. In re­turn, Take­da will get an op­tion for ex-US rights for the first four prod­ucts — what­ev­er they may be — that reach IND at Am­bys. If Take­da choos­es to ex­er­cise those op­tions, then the phar­ma gi­ant al­so coughs up 50% of the de­vel­op­ment costs and some mile­stone pay­ments come in­to play.

Juan Car­los Izpisua Bel­monte

A key win in the deal, though, is that Am­bys has a tight grip on US rights.

“We see many ex­cit­ing deals be­ing struck with sig­nif­i­cant up­fronts, but the chal­lenge for many is they give up world­wide rights on at least the first pro­gram if not more. To con­trol the com­pa­ny’s des­tiny, you must have US rights,” Tong said.

Hol­ger Wil­len­bring

Sci­en­tif­ic founders at Am­bys in­clude Mar­tin Burke of Uni­ver­si­ty of Illi­nois, Markus Grompe at Ore­gon Health & Sci­ence Uni­ver­si­ty, Juan Car­los Izpisua Bel­monte at the Salk, and Hol­ger Wil­len­bring from UCSF. The com­pa­ny’s ex­ec­u­tive team in­cludes Third Rock’s Jef­frey Fin­er (CTO) and Glenn Pierce (CMO), along­side Michael Holmes as CSO and Stan­ley Hol­len­back as SVP of phar­ma­col­o­gy.


Im­age: SHUT­TER­STOCK

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Tony Johnson, Goldfinch Bio CEO (Goldfinch via YouTube)

Kid­ney dis­ease drug­mak­er Goldfinch Bio shuts down

Goldfinch Bio, attempting to make treatments for kidney diseases and diabetic nephropathy, is shutting down.

President and CEO Tony Johnson confirmed to Endpoints News Friday afternoon that the biotech shut down after “fundraising challenges in the current macro-environment.” Fierce Biotech first reported the news.

Johnson, who joined in 2017 after a stint as SVP of early clinical development at AstraZeneca, said in a text that the company “entered the ABC process recently,” referring to an assignment for the benefit of the creditors, which provides a different wind-down avenue than a bankruptcy.

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Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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Elon Musk (GDA via AP Images)

Neu­ralink em­ploy­ees cite lay­offs at Elon Musk’s brain-com­put­er in­ter­face start­up

At least two Neuralink employees have posted to LinkedIn in recent days saying they’ve been laid off from Elon Musk’s brain-computer interface startup, which has received backlash for animal testing.

A former staffer working on preclinical study design and an ex-lab director working on assessing the safety of Neuralink’s implanted devices (prior to human testing) announced recently they’d been laid off, specifically using that terminology. Both had worked at the startup for at least two years.

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Ali Madani, Profluent founder and CEO

Proflu­ent de­buts to de­sign pro­teins with ma­chine learn­ing in bid to move past 'AI sprin­kled on top'

While OpenAI’s Microsoft-allied ChatGPT takes the world by storm, a fledgling startup in Berkeley, CA is debuting to take a similar language-learning model approach, but with the goal of designing new proteins.

Profluent, founded by a former Salesforce AI research leader, has secured $9 million to kick-start its work, with proceeds going toward building out an integrated wet lab and recruiting machine learning scientists and biologists. Insight Partners led the seed round. The investor base also includes Air Street Capital, AIX Ventures and Phoenix Venture Partners.

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