Ronald Park, Ambys Medicines CEO

Third Rock­'s Am­bys clos­es a megaround with the clin­ic still a ways off for its re­gen­er­a­tive cells for liv­er dis­ease

When Am­bys Med­i­cines, a Third Rock Ven­tures-backed re­gen­er­a­tive play, launched in 2018, it came with some se­ri­ous fan­fare for its liv­er dis­ease-fo­cused ap­proach. Now, af­ter years of qui­et de­vel­op­ment, the com­pa­ny has a new trough of mon­ey to play with as it slow­ly ap­proach­es the clin­ic.

Am­bys closed $107 mil­lion in to­tal Se­ries A fund­ing — with a $47 mil­lion ex­ten­sion add — Tues­day with a lead pro­gram cre­at­ing healthy ver­sions of liv­er cells still more than a year from the clin­ic, the biotech said.

Am­bys, which launched back in mid-2018 with a goal to rewrite the rules against se­vere liv­er dis­ease us­ing a broad range of modal­i­ties, says it’s now ready to bring its first shot at that mis­sion to the clin­ic by Q2 2023. The pro­gram, dubbed AMI-918, is an off-the-shelf cell ther­a­py look­ing to re­place dam­aged liv­er tis­sue with healthy cells, Am­bys said.

Mean­while, the com­pa­ny is work­ing on a fol­low-up pro­gram for which the de­tails are still very slim. In a re­lease, the com­pa­ny de­scribed the pro­gram this way:

Am­bys’s sec­ond pro­gram is de­signed to bring the promise of its liv­er-cell plat­form to many more pa­tients by pro­vid­ing ex­tend­ed dura­bil­i­ty of re­place­ment cells and eas­i­er dos­ing and ad­min­is­tra­tion with­out the need for im­muno­sup­pres­sive ther­a­py, which is in­tend­ed to en­able ac­cess to the broad­er pop­u­la­tion of pa­tients with chron­ic liv­er dis­ease. This fol­low-up pro­gram aims to slow dis­ease pro­gres­sion and ul­ti­mate­ly re­verse dis­ease in pa­tients with de­com­pen­sat­ed liv­er dis­ease. This ad­vanced tech­nol­o­gy has the po­ten­tial to ben­e­fit more than a half-mil­lion liv­er dis­ease pa­tients in the U.S. alone each year.

Am­bys launched with much fan­fare and $140 mil­lion in back­ing from Third Rock Ven­tures as part of a re­gen­er­a­tive plan of at­tack against liv­er dis­ease. Third Rock has once again led this round of fund­ing with par­tic­i­pa­tion from Take­da, Schroders Cap­i­tal, Lau­ri­on Cap­i­tal, Smi­le­gate In­vest­ment, and Alexan­dria Ven­ture In­vest­ments.

Named af­ter the sala­man­der Am­bystoma mex­i­canum famed for its re­gen­er­a­tive abil­i­ties, the biotech promised to lever­age both cell and gene ther­a­py ap­proach­es in its pipeline.

Markus Grompe

In its years since launch, Am­bys has erect­ed a clin­i­cal-scale man­u­fac­tur­ing fa­cil­i­ty for its cells the biotech says has al­lowed it to pro­duce its off-the-shelf cells at scale — a sig­nif­i­cant lo­gis­ti­cal hur­dle for the com­pa­ny’s fu­ture chances.

“When we start­ed this ef­fort, it was a moon­shot, giv­en the tremen­dous chal­lenge of pro­duc­ing the types of cells need­ed at scale,” CSO Markus Grompe said in a re­lease. “With the open­ing of our pro­duc­tion fa­cil­i­ty ear­li­er this year, we are well on our way to over­com­ing that hur­dle and will be able to pro­duce high-qual­i­ty, ma­ture liv­er cells to sup­port clin­i­cal de­vel­op­ment and ear­ly com­mer­cial­iza­tion ac­tiv­i­ties.”

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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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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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.

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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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

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

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.