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.”

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Roger Perlmutter, Eikon Therapeutics CEO

Roger Perl­mut­ter builds Eikon's pipeline with deal-mak­ing flur­ry, rais­ing $106M more

Eikon Therapeutics announced three business development deals on Thursday, effectively dropping in a pipeline of cancer drugs alongside more than $100 million in fresh funding.

The Hayward, CA-based company has become one of biotech’s richest startups since its 2019 founding, having raised nearly $775 million. It’s developing a massive, automated research approach built around Nobel Prize-winning microscope science to peer inside cells and watch proteins in action. After its Series B last year, PitchBook reported a $3.02 billion valuation. And while CEO Roger Perlmutter declined to comment on that figure, he said its first tranche of nearly $106 million in Series C funding is a “meaningful step-up to our Series B valuation.”

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The Modulo Bio team with CEO Michael Horowitz (fourth from right in semicircle)

Ex­clu­sive: With $8M, neu­ro start­up Mod­u­lo Bio emerges to test small mol­e­cules for ALS, de­men­tia in CEO’s per­son­al mis­sion

Embarking on a personal mission after his best friend’s mother was diagnosed with a mutation-driven case of frontotemporal dementia, Michael Horowitz has pulled together $8 million in venture funding at Modulo Bio to create small molecules for neurodegenerative diseases.

The San Diego and Bay Area biotech will select its lead development candidate and some backup options within six months and then raise a Series A to investigate therapeutics for C9orf72 mutation-driven cases of ALS and frontotemporal dementia, Horowitz told Endpoints News.

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Eu­ro­pean Par­lia­ment calls mem­ber states to ac­tion on an­timi­cro­bial re­sis­tance

Members of the European Parliament have called on EU countries to develop national action plans against antimicrobial resistance (AMR), calling it a top-three priority health threat.

Parliament on Thursday announced recommendations for the fight against AMR, including national action plans that must be updated at least every two years, an EU-level database tracking AMR and antimicrobial use and increased partnership between the pharma industry, patient groups and academia.

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Roche plans to di­vest from lega­cy Genen­tech man­u­fac­tur­ing fa­cil­i­ty in Cal­i­for­nia

Roche is planning to make some changes to its subsidiary’s manufacturing network in California.

The Swiss pharma announced Wednesday that it plans to divest from Genentech’s manufacturing facility in Vacaville, CA, around 58 miles northeast of San Francisco. According to a statement from Roche, the move is part of a “broader strategy” to bring its manufacturing capabilities in line with its future pipeline. Roche is starting the process of finding a buyer for the site but has not named any candidates yet.

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Peter van de Sande, Synaffix CEO

Lon­za shells out $107M cash to snap up Synaf­fix and its ADC plat­form

After lining up a string of partnerships over the years, Dutch antibody-drug conjugate specialist Synaffix has found a new home: Lonza, the contract development and manufacturing giant.

Lonza is paying about $107 million (€100 million) in cash to acquire Synaffix, with up to $64 million (€60 million) in “additional performance-based consideration” on the table. Synaffix’s ADC tech platform will now become part of Lonza’s offering for biopharma clients, lending its bioconjugate technologies to not just ADCs but also targeted gene therapy, immune cell engagers and other applications.

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Who's con­fi­dent­ly in­vest­ing in biotech star­tups dur­ing these tense days? We've got some an­swers

We’ve got a changeup to our event schedule in Boston next week, where we’ll be doing a mix of live/streaming events at our base at The Seaport Hotel as part of a two-day lineup of webinars, virtual firesides and a cocktail hour Q&A with a veteran of the biotech financing scene.

The 9:30-10:30 am ET live slot on Tuesday, June 6, will now feature a panel conversation on the current state of affairs for VC investing in biotech, focusing on what startups are getting cash — and how. Alaa Halawa, head of US ventures at Mubadala, is confirmed, along with Brian Goodman at MPM and Geoff von Maltzahn, a general partner at Moderna-buoyed Flagship. I have a couple of other invites out and will let you know how that plays out.

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Albert Bourla, Pfizer CEO (Michel Euler/AP Images, Pool)

FDA ap­proves Pfiz­er’s RSV shot for old­er adults, tee­ing up a com­pet­i­tive $17B vac­cine mar­ket

The FDA approved Pfizer’s RSV vaccine called Abrysvo for older adults on Wednesday, placing another Big Pharma onto the commercial stage ahead of the next RSV season.

Pfizer’s approval comes weeks after GSK won approval for its rival shot, Arexvy. Those two vaccines are both approved for use in adults 60 years and older and will be reviewed by a CDC panel in June before they’re expected to commercially launch this fall. Wall Street analysts see RSV as the next multibillion-dollar vaccine market, with Jefferies analysts recently forecasting the RSV market will grow to $17 billion over the next decade.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

FDA's can­cer chief weighs in on com­mon chemo short­ages — re­port

Richard Pazdur, director of the FDA’s Oncology Center of Excellence, attributes the current shortage of two cancer drugs to drug companies that haven’t invested in building out their production capacity.

In an interview with The Cancer Letter, a weekly cancer publication, Pazdur said that the current shortages of cisplatin and carboplatin, a pair of drugs used to treat a wide range of cancer patients, are the result of two problems: manufacturers not investing in enhancing production capacity, and drug companies being dependent on one supplier of raw ingredients. The cisplatin shortage followed an inspection that revealed quality issues at a manufacturing facility, which then led to the shutdown of production. This led to a surge in carboplatin demand, creating a secondary shortage.

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