No­var­tis-backed In­fla­zome rais­es $46M for their clin­i­cal chal­lenge to prove NL­RP3 is a great an­ti-in­flam­ma­to­ry tar­get

The big CAN­TOS study may not have pro­duced the block­buster ap­proval that No­var­tis had been promis­ing in car­dio, but its suc­cess in the IL-1 be­ta path­way has helped in­spire a slate of star­tups that are mov­ing up­stream to what’s be­come a hot field in NL­RP3 in­hi­bi­tion.

To­day’s sto­ry cen­ters on In­fla­zome, which de­scribes it­self as one of the pi­o­neers in NL­RP3. The biotech has raised $46 mil­lion for its Se­ries B round to shift out of pre­clin­i­cal work and head straight in­to the clin­ic next year, with plans to tee up a safe­ty study fol­lowed by a for­ay in­to an or­phan CNS con­di­tion.

Based in Dublin and Cam­bridge, UK, where its re­search is cen­tered, the biotech al­so has an of­fice in Aus­tralia to al­low for a quick shift to hu­man tri­als, where the gov­ern­ment of­fers a hefty 43% tax cred­it for the drug tri­als un­der­tak­en Down Un­der.

Luke O’Neill

Pfiz­er, As­traZeneca, No­var­tis and oth­ers may have failed at their ear­ly at­tempts in this are­na, says CEO Matthew Coop­er, but they have all helped pave the way for In­fla­zome. Coop­er co-found­ed the com­pa­ny with Trin­i­ty Col­lege’s Luke O’Neill. They now have a dozen staffers, with plans to beef up the team now that the B round has ar­rived.

The biotech’s ear­ly work has in­volved hun­dreds of com­pounds and 29 patents cen­tered around NL­RP3, an in­flam­ma­zome that ap­pears to play a key role in push­ing in­flam­ma­tion in a long ros­ter of ail­ments big and small.

Their goal has been to iden­ti­fy the best drug can­di­date de­pend­ing on the tar­get, which starts with cry­opy­rin-as­so­ci­at­ed pe­ri­od­ic syn­drome, an or­phan in­di­ca­tion in­volv­ing hy­per-in­flam­ma­tion that can lead to men­tal re­tar­da­tion. That gives them a chance to pro­vide a proof-of-con­cept dis­play of the tech with a small study be­fore they branch out in­to big­ger dis­eases.

The biotech doesn’t go out of its way to at­tract at­ten­tion. “We’re very Eu­ro­pean in that way,” says Coop­er, a pro­fes­sor at the Uni­ver­si­ty of Queens­land who I queried af­ter the re­lease hit the wire.

At the same time, he’s clear­ly pumped and be­lieves that In­fla­zome is on a great jour­ney, start­ing with CNS and the pos­si­bil­i­ty of go­ing in­to some tough con­di­tions like Parkin­son’s and Alzheimer’s, where tamp­ing down on longterm in­flam­ma­tion could pay sol­id ben­e­fits over time.

That will re­quire com­pounds that can get in­to the brain to do their job. But out­side the brain there are plen­ty of ad­di­tion­al tar­gets, run­ning a gamut rang­ing from gout to arthri­tis to car­dio.

Like his biotech ri­vals — a group that in­cludes NodThera and IFM — Coop­er is pur­su­ing the be­lief that rather that tack­ling the en­tire IL-1 be­ta path­way, it’s pos­si­ble to much more safe­ly sin­gle out the “lone wolf” that they be­lieve is the prime sus­pect for the laun­dry list of con­di­tions it may trig­ger.

In­ter­est­ing­ly, there’s al­so been ear­ly work done to link the ke­to­genic di­et to hit­ting NL­RP3, which may ex­plain why the ul­tra low carb di­et could work in a range of cas­es for child­hood epilep­sy, as prac­ticed at Johns Hop­kins for decades. Coop­er, though, says it’s go­ing to take some­thing with re­al ther­a­peu­tic strength to have an im­pact on the dis­eases they’re af­ter, and ke­to may not qual­i­fy on that lev­el.

For­bion led the round, with Lon­gi­tude Cap­i­tal and found­ing in­vestors No­var­tis Ven­ture Fund and Foun­tain Health­care Part­ners al­so par­tic­i­pat­ing.

Im­age: Matthew Coop­er. UNI­VER­SI­TY OF QUEENS­LAND

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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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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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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Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

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.

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.