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.


Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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Vir's CMO says he's sur­prised that a low dose of their he­pati­tis B drug ap­pears promis­ing in ear­ly slice of da­ta — shares soar

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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Eli Lil­ly demon­strates that 2 an­ti­bod­ies beat 1 for guard­ing against se­vere Covid-19. But can that solve the first an­ti­body’s prob­lem amid slow up­take?

It seems safe to say that two antibodies are better than one.

Eli Lilly released the largest results yet on Tuesday for their Covid-19 neutralizing antibody cocktail, announcing that the combo reduced deaths and hospitalizations in coronavirus patients by 70%. Across 1,000 patients, there were 11 such events in the treatment group and 36 in the placebo group.

The breakdown for deaths alone was even starker: 10 in the placebo group and 0 in the treatment group. Lilly added that the drug hit secondary endpoints for reducing viral load and alleviating symptoms, although they did not disclose numbers.

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George Yancopoulos (L) and Len Schleifer (Regeneron)

Re­gen­eron touts pos­i­tive pre­lim­i­nary im­pact of its Covid an­ti­body cock­tail, pre­vent­ing symp­to­matic in­fec­tions in high-risk group

Regeneron flipped its cards on an interim analysis of the data being collected for its Covid-19 antibody cocktail used as a safeguard against exposure to the virus. And the results are distinctly positive.

The big biotech reported Tuesday morning that their casirivimab and imdevimab combo prevented any symptomatic infections from occurring in a group of 186 people exposed to the virus through a family connection, while the placebo arm saw 8 of 223 people experience symptomatic infection. Symptomatic combined with asymptomatic infections occurred in 23 people among the 223 placebo patients compared to 10 of the 186 subjects in the cocktail arm.

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Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

IPO track­er: 2021 gets start­ed with a flur­ry of new of­fer­ings

A global pandemic couldn’t slow down what turned out to be a record year for biotech IPOs. With the calendar turning toward 2021, the Endpoints News team is prepped to track each new filing this year, and the outcome. We’re off to another hot start at least.

Below, you’ll find the companies that have filed to go public, in addition to those that have already priced. Through the first two business weeks of January, there have already been 9 biotechs that have filed or priced, and the number is only expected to grow. We’ll keep the tracker updated as it does.