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.

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; 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.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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UP­DAT­ED: Three biotechs price hefty IPOs just be­fore the week­end, while a fourth and a SPAC seek spots on Wall Street

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

A handful of biotechs are hitting Wall Street just before the start of the weekend, with three companies — Caribou Biosciences, Sophia Genetics and Absci — all pricing big raises Wednesday and Thursday. Gamma delta T cell-focused IN8bio relaunched its IPO campaign months after postponing it last November, seeking a slightly lower raise. And another SPAC has filed for a public debut.

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Victor Perlroth, Kodiak Sciences CEO

Ko­di­ak turns down $125M pay­ment from Bak­er Bros. deal, slash­es roy­al­ty cap by 55%

Following a massive public raise last November, Kodiak Sciences has re-worked a royalty sale agreement with an old partner — and declined new funds in the process.

Kodiak is turning down a planned $125 million payment from Baker Bros. Advisors, according to an SEC filing, cutting short an agreement that saw the biotech hand over a 4.5% stream of royalty sales on its experimental anti-VEGF therapy KSI-301 for retinal vascular diseases. In conjunction with the move, Kodiak is shrinking the royalty cap from just over $1 billion to $450 million.

EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Mol­e­c­u­lar Di­ag­nos­tics Can Trans­form Can­cer Care. Let’s Make It Hap­pen.

Like so many people around the world, my life has been profoundly shaped by cancer. Those personal experiences, along with a deep love of clinical laboratory science and a passion to apply the power of genomics in medicine, motivated me to launch a company that would improve cancer care through better diagnostics. Thirteen years later, I am proud that we are delivering more accurate information at multiple points along the patient journey, with a focus on eight of the 10 cancers that are most commonly diagnosed in the United States.