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

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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[via AP Images]

Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.