Keith Dionne, Casma CEO (Casma)

Kei­th Dion­ne's Cas­ma lines up a $50M ven­ture round to con­tin­ue the pre­clin­i­cal work on a cel­lu­lar re­cy­cling pro­gram

Two years af­ter launch­ing out of Third Rock’s in­cu­ba­tor, Cas­ma Ther­a­peu­tics has land­ed a $50 mil­lion Se­ries B for pre­clin­i­cal de­vel­op­ment of its mus­cu­lar dy­s­tro­phy and Au­tophagy De­grad­er Plat­form (ADP) pro­grams.

Cas­ma kicked off in 2018 with a $58.5 mil­lion Se­ries A and a staff of ex­perts in au­tophagy, the cell’s “garbage dis­pos­al.” The biotech’s TRPML1 ag­o­nist pro­gram for mus­cu­lar dy­s­tro­phy is ex­pect­ed to hit the clin­ic in 2022, ac­cord­ing to CEO Kei­th Dionne, and the ADP pro­gram is about a year be­hind that.

“If you imag­ined your house, where you plug up the garbage dis­pos­al, plug up the toi­lets and stop tak­ing out the trash, you would find, prob­a­bly fair­ly quick­ly, the house would be­come un­liv­able. And that’s es­sen­tial­ly the same thing that hap­pens in­side of a cell,” Dionne said. “If we don’t have these mech­a­nisms of main­tain­ing home­osta­sis to clear out ma­te­ri­als that were maybe used once, or maybe mu­tat­ed to be­gin with but oth­er­wise built up in­side of the cell —  the cell be­comes dys­func­tion­al.”

The biotech seeks to boost the cell’s re­cy­cling sys­tem, which could treat a range of ill­ness­es, in­clud­ing lyso­so­mal stor­age and in­flam­ma­to­ry dis­or­ders, liv­er and mus­cle dis­eases, and neu­rode­gen­er­a­tion.

The dis­cov­ery of au­tophagy mech­a­nisms won the No­bel Prize in 2016. While there are cur­rent­ly no ap­proved drugs in the field, some have shown to en­hance au­tophagy as a side ef­fect.

“When we launched the com­pa­ny, it was re­al­ly with the re­al­iza­tion that we thought we could, and that we need­ed to, de­vel­op drugs that were spe­cif­ic to the in­duc­tion of au­tophagy, not hit­ting au­tophagy as one of the 5 or 10 oth­er path­ways that they (oth­er drugs) were touch­ing on,” Dionne said. “Look­ing at the pre­clin­i­cal lit­er­a­ture it be­came very ap­par­ent that the num­ber of dis­eases that are im­pact­ed by this is quite large. And again, that is part of the ex­cite­ment of get­ting go­ing.”

The com­pa­ny will use the Se­ries B fund­ing to ad­vance both its ADP and TRPML1 pro­grams. TRPML1 reg­u­lates the re­pair of the plas­ma mem­brane of mus­cle cells fol­low­ing dam­age, ad­dress­ing the “core pathol­o­gy in mul­ti­ple forms of mus­cu­lar dy­s­tro­phy,” ac­cord­ing to Cas­ma.

Skip Vir­gin

The biotech’s sci­en­tif­ic founders in­clude au­tophagy ex­pert Beth Levine, who died ear­li­er this year af­ter a fight with breast can­cer. The for­mer Cen­ter for Au­tophagy Re­search di­rec­tor was known for dis­cov­er­ing the mam­malian au­tophagy gene, BECN1. The team al­so in­cludes An­drea Bal­labio, a leader in tran­scrip­tion­al reg­u­la­tion of lyso­so­mal bio­gen­e­sis and au­tophagy; James Hur­ley, who’s stud­ied the struc­ture of au­tophagy com­plex­es; and Vir Biotech­nol­o­gy CSO and EVP of re­search Her­bert “Skip” Vir­gin, who’s re­searched the role of au­tophagy genes in in­flam­ma­tion and im­mu­ni­ty.

The Se­ries B was led by The Col­umn Group, and joined by Third Rock, Even­tide As­set Man­age­ment, Schroder Ad­veq, and oth­er undis­closed in­vestors.

“It’s ex­cit­ing to work in a new space of sci­ence, open­ing up a whole new field, and then to see that start to trans­late in­to ther­a­peu­tics. So that’s pulled us through this whole Covid pe­ri­od and … kept us go­ing through the en­tire process to make progress on them. So (there’s) just a pal­pa­ble sense of ex­cite­ment around the com­pa­ny,” Dionne said.

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.