Sandra Glucksmann (Cedilla)

Third Rock-backed Cedil­la reels in $57.6M for small mol­e­cule pro­grams to fight can­cer

Cedil­la Ther­a­peu­tics launched just over two years ago with a $56 mil­lion Se­ries A and a plan to chart new ter­ri­to­ry in the pro­tein degra­da­tion space. Now, CEO San­dra Glucks­mann is fol­low­ing up with a $57.6 mil­lion Se­ries B round to steer the Cam­bridge, MA-based biotech in a slight­ly dif­fer­ent di­rec­tion.

The com­pa­ny is cur­rent­ly fo­cused on find­ing new small mol­e­cule bind­ing sites — which may af­fect pro­tein degra­da­tion, in­hi­bi­tion or both — to drug the un­drug­gable.

“The sci­ence takes you to places, and we broad­ened that um­brel­la,” Glucks­mann told End­points News, adding lat­er: “You’ve got to go where the sci­ence goes, right?”

The Se­ries B — led by Cas­din Cap­i­tal and Box­er Cap­i­tal of Tavi­s­tock Group — will fund ear­ly de­vel­op­ment of the biotech’s small mol­e­cule pro­grams, in­clud­ing pre­clin­i­cal work on its first two prod­uct can­di­dates, which Cedil­la is keep­ing un­der wraps for now. Glucks­mann said the com­pa­ny has about 5 or 6 on­col­o­gy pro­grams run­ning in par­al­lel, which are still years away from the clin­ic.

“One of our tar­gets is some­thing that peo­ple have been try­ing to drug for over 20 years,” she hint­ed.

Back in 2018, Cedil­la was aim­ing to up­stream a com­mon ap­proach to pro­tein degra­da­tion by us­ing small mol­e­cule drugs to desta­bi­lize dis­ease-caus­ing pro­teins. The pro­tein degra­da­tion field has seen plen­ty of cash flow this year, with new­com­er Jan­pix rak­ing in a $19 mil­lion launch round ear­li­er this month, days af­ter C4, one of the pi­o­neers, land­ed its IPO.

Cedil­la’s fo­cus to­day is still on small mol­e­cules, but for in­hi­bi­tion. “By un­der­stand­ing the nov­el tar­get bi­ol­o­gy, how the pro­teins are mod­i­fied, how they take on dif­fer­ent states, we are able to re­veal new small mol­e­cule bind­ing pock­ets, or bind­ing sites,” Glucks­mann said. “The bind­ing of that small mol­e­cule may af­fect degra­da­tion and/or in­hi­bi­tion,” she added lat­er.

In ad­di­tion to the Se­ries B, Cedil­la is bring­ing Cas­din CIO and founder Eli Cas­din and Box­er se­nior VP Do­minik Naczyn­s­ki on­to its board of di­rec­tors. Since launch­ing in 2018, the biotech has grown from a hand­ful of em­ploy­ees to 35 staffers.

“It takes a vil­lage to de­vel­op drugs,” Glucks­mann said. “And it’s great to have so­phis­ti­cat­ed and ex­pe­ri­enced in­vestors to be part of the team.”

Glucks­mann was re­cruit­ed by Third Rock as an en­tre­pre­neur-in-res­i­dence in 2017, with the in­ten­tion that she would launch Cedil­la. The team took six months to nail down a busi­ness and re­search plan, then un­veiled the com­pa­ny in 2018.

“For me what was ex­cit­ing was to take well-val­i­dat­ed on­col­o­gy tar­gets — where we un­der­stand the bi­ol­o­gy [and] we un­der­stand the role that they play in hu­man dis­ease — and then a tool, which is small mol­e­cules that we know how to de­vel­op, and com­bin­ing those two … to do some­thing nov­el with a team that is ex­pe­ri­enced,” she said.

Third Rock chipped in again to the Se­ries B, as well as new in­vestors Eli Lil­ly and Schroder Ad­veq.

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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