A stem cell ther­a­py preps de­but in Japan­ese mar­ket to the dis­may of US re­searchers

As Japan ap­proves a stem cell treat­ment for spinal cord in­juries in a land­mark — if lit­tle no­ticed — de­ci­sion, ex­perts around the world have voiced con­cerns that un­der­score the trou­ble that still plague first-gen stem cell ther­a­pies.

Com­ment­ing at the re­quest of Na­ture News, 10 spe­cial­ists in stem cell sci­ence or spinal cord in­juries poke holes in the small clin­i­cal tri­al that formed the ba­sis of the con­di­tion­al ap­proval, ques­tion­ing both the ef­fi­ca­cy and safe­ty of the treat­ment.

The ther­a­py, Stemirac, in­volves ex­tract­ing mes­enchy­mal stem cells from a per­son who suf­fered from a spinal cord in­jury, grow­ing about 50 mil­lion to 200 mil­lion of them in a lab, then in­tra­venous­ly in­fus­ing the MSCs back in­to the pa­tient with­in 40 days of the in­jury. Ex­act­ly how it works to re­pair dam­age to the spinal cord is a bit of a black box now: Re­searchers led by pro­fes­sor Os­amu Hon­mo at Sap­poro Med­ical Uni­ver­si­ty be­lieve it might have to do with re­duc­ing in­flam­ma­tion, pro­tect­ing ex­ist­ing neu­rons or turn­ing in­to nerve cells them­selves.

Their be­lief is based on an un­pub­lished 13-pa­tient tri­al, which saw 12 of them “im­prove by at least one lev­el on the Amer­i­can Spinal In­jury As­so­ci­a­tion im­pair­ment scale, an in­ter­na­tion­al­ly rec­og­nized sys­tem that ranks peo­ple’s abil­i­ty to con­tract mus­cles and sense touch on parts of the body,” Na­ture News re­port­ed.

Skep­tics, how­ev­er, re­main leery.

Bruce Dobkin

With­out a dou­ble-blind tri­al de­sign, there’s no way to prove that the im­prove­ments weren’t due to nat­ur­al heal­ing and phys­i­cal re­ha­bil­i­ta­tion in the months af­ter an in­jury, Bruce Dobkin, a neu­rol­o­gist at the Uni­ver­si­ty of Cal­i­for­nia, Los An­ge­les, told Na­ture News.

“This tri­al, as de­signed, can­not re­veal ef­fi­ca­cy,” he said, adding the claim that the MSCs con­vert­ed to neu­rons in the tri­al is al­so a red flag.

Pamela Robey

An­oth­er rea­son to be cau­tious, ac­cord­ing to NIH stem cell re­searcher Pamela Robey: The fact, shown in pre­vi­ous re­search, that MSCs in­fused in­tra­venous­ly tend to get trapped in the lungs “makes it dif­fi­cult to see how they can be ef­fec­tive in the spinal cord.”

Masanori Fukushi­ma, a gov­ern­ment-fund­ed ad­vis­er to the project, has stood by Hon­mo’s work, em­pha­siz­ing that some pa­tients in the tri­al were too se­vere­ly in­jured to have had achieved nat­ur­al heal­ing.

When the med­ical equip­ment man­u­fac­tur­er Nipro Corp be­gins sell­ing Stemirac, it can mark some long await­ed ac­tiv­i­ty for re­gen­er­a­tive med­i­cine in the mar­ket­place af­ter years of de­lays and dis­ap­point­ments. But it’s not nec­es­sar­i­ly the en­try that the in­dus­try — which is now ex­plor­ing next-gen, of­ten off-the-shelf, stem cell-based ther­a­pies for di­a­betes, Crohn’s dis­ease and graft ver­sus host dis­ease among oth­ers — has been look­ing for.

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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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.