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

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So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

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The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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The IPO engine is thriving, never mind the rampage of the coronavirus crisis on R&D timelines.

On Friday, along with synthetic lethality-focused biotech Repare Therapeutics, another Bristol Myers partner Forma Therapeutics also unveiled its plans to vault on to the Nasdaq — penciling in a target of $150 million.

The Watertown, Massachusetts-based company — which poached senior Genentech executive Frank Lee to take over the reins last year after more than a decade under founder Steve Tregay — raised a plump $100 million late last year, while shepherding its sickle cell disease (SCD) drug through an early-stage trial.

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With a Bristol Myers Squibb endorsement in tow, Versant-backed cancer drug developer Repare Therapeutics has set its sights on a Nasdaq debut.

On Friday, the Montreal-based company with operations in Cambridge, Massachusetts that is yet to enter the clinic, unveiled plans for a $100 million IPO, banking on its “synthetic lethality” platform.

The basic idea is to target the genetic basis of tumors, a common idea across precision oncology medicines. But instead of targeting the perpetrator mutation directly, the compound is designed to go after the other gene in the gene pair. The rationale is based on the decades-old genetic principle that indicates two mutations are lethal only when combined together.