Patrick Gunning, Janpix founder and CSO (Gunning Group)

Medicxi splash­es more fund­ing in­to Jan­pix's STAT pro­tein de­grad­er pro­gram

Three years af­ter giv­ing Uni­ver­si­ty of Toron­to pro­fes­sor Patrick Gun­ning a $19 mil­lion launch round to start Jan­pix, Medicxi is reach­ing deep­er in­to its pock­ets to nudge the biotech’s first STAT pro­tein de­grad­er in­to the clin­ic.

On Wednes­day, Cam­bridge, MA-based Jan­pix an­nounced the clos­ing of a mod­est $10 mil­lion Se­ries B led by Medicxi. Ro­man Fleck — CEO and Medicxi ven­ture ad­vi­sor — says the fund­ing will see the com­pa­ny’s first can­di­date to hu­man test­ing, and ex­pand its pipeline of STAT3 and STAT5 pro­tein de­graders.

A steady stream of fund­ing has flowed in­to pro­tein degra­da­tion this year. Back in March, Nurix and Kymera scooped up a to­tal of $222 mil­lion in con­sec­u­tive days. And a month lat­er, Am­phista launched with a $7.5 mil­lion Se­ries A and help from field ex­pert Alessio Ciul­li. C4 Ther­a­peu­tics, mean­while, land­ed its IPO just days ago. The gen­er­al idea is to make en­tire pro­teins dis­ap­pear — rather than just block­ing their ki­nase bind­ing sites — by us­ing the body’s garbage dis­pos­al sys­tem.

“Se­lec­tive pro­tein degra­da­tion is emerg­ing as a new ther­a­peu­tic par­a­digm and it will have a ma­jor im­pact on how we treat se­ri­ous dis­eases,” Sandy Zweifach, chair­man of the Jan­pix board and ven­ture part­ner at Medicxi, said in a state­ment. “Jan­pix’s small mol­e­cules present a new modal­i­ty to achieve pro­tein degra­da­tion and will like­ly ex­pand the range of pro­teins that can be ef­fi­cient­ly tar­get­ed.”

Jan­pix’s lead pro­gram, for “var­i­ous hema­to­log­i­cal can­cers,” tar­gets both STAT3 and STAT5, short for the sig­nal trans­duc­er and ac­ti­va­tor of tran­scrip­tion pro­teins that have a role in can­cer de­vel­op­ment. Gun­ning has linked STAT3, for in­stance, to the pro­mo­tion of tu­mor growth and drug re­sis­tance in glioblas­toma. Back in 2017, he said his work was shap­ing up to fight ag­gres­sive blood, brain and breast can­cers, ac­cord­ing to a Uni­ver­si­ty of Toron­to re­port.

In June, the biotech brought da­ta to the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion and Amer­i­can As­so­ci­a­tion for Can­cer Re­search vir­tu­al meet­ings show­ing that its can­di­date JPX-1188 de­grad­ed STAT3 & STAT5 in leukemic cells. The drug achieved a sus­tained degra­da­tion of greater than 90% af­ter 2 hours, ac­cord­ing to Jan­pix. Plus, re­sults showed sig­nif­i­cant tu­mor re­gres­sion in an AML mouse mod­el, it an­nounced.

Gun­ning said in a state­ment:

As Jan­pix ad­vances our STAT3/5 de­graders to­wards the clin­ic, we con­tin­ue to ex­pand our un­der­stand­ing of the bi­o­log­i­cal im­pact of dual STAT3/5 degra­da­tion … Our da­ta al­so high­light the po­ten­tial for STAT3/5 de­graders to treat a broad­er set of leukemia pa­tients, as most leukemic pri­ma­ry pa­tient sam­ples test­ed re­spond­ed to our com­pounds.

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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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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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.

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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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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CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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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.