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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

In­fa­mous biotech ex­ec Mar­tin Shkre­li gets out of prison, hits the street

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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De­spite fed­er­al ef­forts to di­ver­si­fy clin­i­cal tri­als, progress re­mains 'stag­nan­t' — re­port

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

Paul Chaplin, Bavarian Nordic president and CEO

Bavar­i­an Nordic se­cures BAR­DA con­tract for small­pox vac­cine

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.

Lina Khan, FTC chair (Saul Loeb/Pool via AP)

New FTC com­mis­sion­er could turn the tide for an in­ves­ti­ga­tion in­to PBMs

The Senate last week voted along party lines, 51-50, with Vice President Kamala Harris casting the tie-breaker, to make President Biden appointee Alvaro Bedoya the deciding vote on a split 2-2 Federal Trade Commission.

The addition of Bedoya to the FTC could not only spell more trouble for biopharma M&A activity, as he may align with his Democrat partners to break the FTC ties, but it may also mean that FTC Chair Lina Khan has what she needs to move forward on a study around the pharma middlemen known as pharmacy benefit managers.

Henrietta Lacks

UP­DAT­ED: Fed­er­al judge weighs mo­tion to dis­miss HeLa law­suit against Ther­mo Fish­er

The story of Henrietta Lacks’ immortal cell line and her family’s fight for justice caught the attention of national media outlets and Hollywood years ago. Now, the case faces an uncertain fate as a Baltimore federal judge considers tossing the case.

After a hearing on Tuesday, Judge Deborah Boardman is weighing Thermo Fisher’s motion to dismiss the claims against it on the grounds that the statute of limitations has passed, and the continuing harm doctrine does not apply. Boardman is grappling with the “extraordinarily unique facts” of the case, according to Maryland Matters, which first reported the news.