Steven Kelly, Carisma Therapeutics CEO

Con­tin­u­ing push be­yond Covid-19 vac­cines, Mod­er­na part­ners with 'CAR-M' biotech on in vi­vo can­cer cell ther­a­pies

Since com­ing in­to a mas­sive wind­fall with its Covid-19 vac­cines, Mod­er­na has been look­ing for ways to ex­pand its mR­NA tech­nolo­gies in­to oth­er promis­ing ar­eas. On Mon­day, the biotech took its next step to­ward that goal.

Mod­er­na is part­ner­ing with Caris­ma Ther­a­peu­tics on a huge slate of “CAR-M” pro­grams to treat can­cer, the com­pa­nies an­nounced Mon­day morn­ing, promis­ing $45 mil­lion up­front and $35 mil­lion in eq­ui­ty. Though the amount of mile­stones was not spec­i­fied, the biotechs not­ed Mod­er­na has the op­tion to nom­i­nate up to 12 tar­gets for de­vel­op­ment.

Caris­ma will lead pre­clin­i­cal and dis­cov­ery ef­forts while Mod­er­na will take charge for clin­i­cal stud­ies and po­ten­tial com­mer­cial­iza­tion.

“It’s a clear tech­nol­o­gy fit for our mR­NA LNP plat­form,” Rose Lough­lin, Mod­er­na SVP of re­search and ear­ly de­vel­op­ment, told End­points News of the deal. “By part­ner­ship, it re­al­ly lets us quick­ly ex­pand the reach of that plat­form … It’s ide­al for this type of ap­pli­ca­tion, giv­en what they can do. They re­al­ly com­ple­ment our cur­rent on­col­o­gy port­fo­lio.”

Stéphane Ban­cel

The Covid-19 vac­cine has turned Mod­er­na from a biotech with no ap­proved prod­ucts in­to a com­pa­ny worth more than $87 bil­lion as of Mon­day morn­ing — and its CEO, Stéphane Ban­cel, in­to a multi­bil­lion­aire. Pri­or to Mon­day, Mod­er­na al­ready had a few on­col­o­gy pro­grams un­der its belt, in­clud­ing can­cer vac­cines and ex­per­i­men­tal ther­a­peu­tics in­ject­ed di­rect­ly in­to tu­mors.

But Mon­day’s deal marks a new step in that port­fo­lio ex­pan­sion, push­ing Mod­er­na in­to in vi­vo cell ther­a­py tech­nol­o­gy. When the plat­forms are used to­geth­er, Lough­lin said, the mR­NA and LNPs will be used to de­liv­er the CAR-M ther­a­pies, where ‘M’ stands for mono­cytes, a class of in­nate im­mune cells.

“As we think about this ap­pli­ca­tion, in im­muno-on­col­o­gy, it is very much di­rect­ing the im­mune im­pact of these cells to the tu­mor, chang­ing that tu­mor mi­croen­vi­ron­ment and then uti­liz­ing the rest of the im­mune sys­tem, re­al­ly train­ing it to fight the tu­mor,” Lough­lin said. “They’re al­so pre­sent­ing anti­gens from that can­cer cell.”

For Caris­ma, the part­ner­ship marks the next step in the com­pa­ny’s growth, CEO Steve Kel­ly told End­points. The biotech, launched back in 2017 to de­vel­op sol­id tu­mor cell ther­a­pies us­ing CARs and macrophages, put its first pro­gram in­to the clin­ic last year for an au­tol­o­gous CAR-M tar­get­ing HER2-ex­press­ing tu­mors.

Mono­cytes, though, are the pre­cur­sor to macrophages, Kel­ly not­ed, es­sen­tial­ly a pro­drug for the white blood cells. The com­pa­nies will fo­cus on de­liv­er­ing mR­NA ex­pres­sion for CARs to mono­cytes in cir­cu­la­tion, which will then “traf­fic to the tu­mor and dif­fer­en­ti­ate in­to macrophages at that point,” Kel­ly said.

The way the two plat­forms fit to­geth­er “comes down to the speci­fici­ties of the lipid nanopar­ti­cle,” Kel­ly added.

It re­mains to be seen whether Mod­er­na’s suc­cess­es in Covid-19 can be repli­cat­ed in oth­er fields, but ear­ly re­turns have not al­ways im­pressed in­vestors. Last month, Mod­er­na re­leased da­ta for its mR­NA flu shots tout­ing sig­nif­i­cant boost­ing of an­ti­bod­ies, but they didn’t ap­pear to of­fer any clear ad­van­tages over al­ready ap­proved jabs.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Peter Hecht, Cyclerion CEO

Cy­cle­ri­on board quick­ly nix­es CEO Pe­ter Hecht's un­ortho­dox pitch for low cash re­serves

It’s been less than two months since Cyclerion laid out a new R&D strategy around its lead drug in mitochondrial diseases, one that triggered the company to lay off close to half of its employees and explore licensing deals for the rest of the pipeline. But CEO Peter Hecht apparently has other plans in mind.

Hecht, who led Ironwood for close to 20 years before spinning out Cyclerion, disclosed in an SEC filing Monday that a “newly-formed private company” that he “may have or may acquire an interest” submitted a proposal to Cyclerion the day prior to purchase Cyclerion’s CNS assets, including CY6463 and CY3018 — the top two programs listed in the pipeline.

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Karen Aiach, Lysogene CEO (RE(ACT) Discovery Institute)

Gene ther­a­py flunks PhII/III study, but for­mer Sarep­ta part­ner sees a path for­ward — if it can find the cash

The development path for Lysogene’s gene therapy for MPS IIIA has been a rocky one. After the FDA slapped a partial clinical hold on a Phase II/III study, a patient already dosed in the trial died, although it was deemed unrelated to treatment. Then earlier this year, Sarepta pulled out of their three-year partnership due to disagreements on who will handle commercial supply.

And now, Lysogene reported the trial has failed its primary endpoint.

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J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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