Bound for the clin­ic with a new ap­proach to syn­thet­ic lethal­i­ty, Cyteir bags $29M

As an as­so­ciate pro­fes­sor at The Jack­son Lab­o­ra­to­ry, Kevin Mills ze­roed in on the sup­port­ing role that the RAD51 pro­tein played in re­pair­ing the DNA dam­age caused by el­e­vat­ed lev­els of ac­ti­va­tion-in­duced cy­ti­dine deam­i­nase, or AID.

Kevin Mills

Trip­ping up RAD51 with a small mol­e­cule, he thought, would throw a mon­key wrench in the whole DNA re­pair path­way, al­low­ing mu­tat­ed can­cer cells and oth­er stressed cells in­volved in au­toim­mune dis­eases to die, play­ing an as­sist­ing role in syn­thet­ic lethal­i­ty.

Mills would go on to found a biotech in Cam­bridge, MA called Cyteir. And to­day a group of high pro­file ven­ture back­ers are com­ing up with the $29 mil­lion that Mills and his team need to get in­to the clin­ic with a lead can­cer drug. The CSO is al­so be­ing joined by Cel­gene vet Markus Ren­schler, who’s now the new CEO.

“My whole ca­reer has been spent on DNA dam­age and DNA dam­age re­spons­es,” Mills tells me. The work on RAD51 goes back a decade, and Cyteir was spun out of The Jack­son Lab in 2012, pa­tient­ly do­ing the pre­clin­i­cal work need­ed to ad­vance a lead pro­gram.

Markus Ren­schler

If their first Phase I/II study goes well, Ren­schler says there are a num­ber of op­tions for de­vel­op­ing the pro­gram. “We can de­vel­op it as a monother­a­py for over-ex­pressed AID,” he notes, as well as in com­bi­na­tion with plat­inum-based chemo, PARP in­hibitors (which al­so trig­ger syn­thet­ic lethal­i­ty), ra­di­a­tion as well as the pop­u­lar new gen­er­a­tion of check­point in­hibitors.

Ren­schler, though, wants to re­tain rights to the drug for now, look­ing to part­ner lat­er as they get deep­er in­to the clin­ic. With enough mon­ey to nab proof-of-con­cept hu­man da­ta, he has op­tions on rais­ing more cash lat­er. Right now he wants to ex­plore just how much val­ue is here be­fore dick­er­ing any of it away.

Start­ing in hema­tol­ogy just makes sense, he adds. Tu­mors that arise from B cells have this ac­ti­vat­ed path­way, he says, high­light­ing DL­B­CL, non-Hodgkin lym­phoma and mul­ti­ple myelo­ma as prime tar­gets. That’s where it’s eas­i­est to find a high num­ber of pa­tients with the AID bio­mark­er.

But Mills and Ren­schler are al­so pumped about their prospects in au­toim­mune dis­eases, not­ing that the same tar­get­ed ap­proach could pro­vide plen­ty of added up­side.

It’s ear­ly days for clin­i­cal work at Cyteir, which will now grow from a vir­tu­al staff of 6 to 20. But they have big plans.

Ven­rock led the round, joined by re­turn­ing in­vestors in­clud­ing Cel­gene. New in­vestors in­clud­ed Light­stone Ven­tures and DROIA On­col­o­gy Ven­tures.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.