La Jol­la whips up some love for iron over­load drug with promis­ing da­ta snap­shot

With rev­enue trick­ling in from its first prod­uct and an NDA in place for a sec­ond, La Jol­la Phar­ma­ceu­ti­cal is ready to shine a bright light on the third can­di­date com­ing through its pipeline.

Jeff Vacir­ca NY Can­cer & Blood

An in­ter­im analy­sis of a Phase II study sug­gests that LJPC-401 could sig­nif­i­cant­ly low­er the ex­cess iron lev­els in pa­tients with hered­i­tary he­mochro­mato­sis. The drug — a sub­cu­ta­neous in­jec­tion of syn­thet­ic hu­man hep­cidin — in­duced a mean re­duc­tion in TSAT from base­line of 42%, ver­sus 6% on place­bo (p<0.0001), mark­ing a clear score on the pri­ma­ry end­point.

It’s a small study with 12 and 14 pa­tients on the drug and place­bo co­horts re­spec­tive­ly but ap­par­ent­ly looked promis­ing enough to send the stock $LJPC surg­ing 84%.

Jeff Vacir­ca, an in­ves­ti­ga­tor and chief of clin­i­cal re­search at New York Can­cer & Blood Spe­cial­ists, ex­plained his en­thu­si­asm this way:

There have been no new treat­ment modal­i­ties in­tro­duced for pa­tients with hered­i­tary he­mochro­mato­sis in more than a decade. In light of the neg­a­tive im­pact that re­peat­ed phle­boto­my pro­ce­dures have on pa­tient qual­i­ty-of-life, pa­tients would wel­come a phar­ma­co­log­ic treat­ment that they can self-ad­min­is­ter and that ad­dress­es the un­der­ly­ing patho­phys­i­ol­o­gy of the dis­ease.

LJPC-401 al­so met the key sec­ondary end­point of low­er­ing the fre­quen­cy of phle­boto­my pro­ce­dures with pa­tients av­er­ag­ing 0.06 phle­botomies per month (3 pro­ce­dures be­tween 2 pa­tients) com­pared to place­bo-treat­ed pa­tients, who had 0.41 (9 pa­tients col­lec­tive­ly re­ceived 24). The p-val­ue came in at 0.003.

In­jec­tion site re­ac­tions, which on­ly hap­pened to 6% of the place­bo group, oc­curred in 79% of pa­tients treat­ed with the drug — though La Jol­la would em­pha­size they were mild.

LJPC-401 was a key part of the San Diego biotech’s pitch for a $100 mil­lion pub­lic of­fer­ing in 2018 along­side LJPC-0118, the se­vere malar­ia treat­ment that re­cent­ly re­ceived a break­through ther­a­py des­ig­na­tion. Topline re­sults are ex­pect­ed lat­er in the year.


Im­age: La Jol­la via Linkedin

Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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John Hood [file photo]

UP­DAT­ED: 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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Jim Mellon [via YouTube]

Health­i­er, longer lifes­pans will be a re­al­i­ty soon­er than you think, Ju­ve­nes­cence promis­es as it clos­es $100M round

Earlier this year, an executive from Juvenescence-backed AgeX predicted the field of longevity will eventually “dwarf the dotcom boom.” Greg Bailey, the UK-based anti-aging biotech’s CEO, certainly hopes so.

On Monday, Juvenescence completed its $100 million Series B round of financing. The company is backed by British billionaire Jim Mellon — who wrote his 400-page guide to investing in the field of longevity shortly after launching the company in 2017.  Bailey, who served as a board director for seven years at Medivation before Pfizer swallowed the biotech for $14 billion, is joined by Declan Doogan, an industry veteran with stints at Pfizer and Amarin.

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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