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

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.