Strug­gling gene ther­a­py pi­o­neer uniQure brings out the ax, chop­ping pro­grams and staffers in over­haul

UniQure was able to snare the world’s first ap­proval for a gene ther­a­py. But it wasn’t able to cash in. And with its top pipeline pro­grams strug­gling against heavy­weight com­pe­ti­tion, the Dutch/US biotech $QURE to­day took out the ax and start­ed to chop away at its R&D struc­ture in search of some big cost sav­ings.

The ax is be­ing aimed at R&D in The Nether­lands as uniQure plans to con­sol­i­date man­u­fac­tur­ing in the US, where it has fa­cil­i­ties in Lex­ing­ton, MA. Up to 60 jobs, a quar­ter of its work­force, will be cut in the re­or­ga­ni­za­tion.

UniQure is look­ing to drop AMT-110 for San­fil­ip­po B, ax­ing its fo­cus on that dis­ease along with Parkin­son’s as it re­views its op­tions. The top pri­or­i­ties now: he­mo­phil­ia B, Hunt­ing­ton’s dis­ease and its col­lab­o­ra­tion with Bris­tol-My­ers Squib in car­dio­vas­cu­lar dis­ease.

As part of the com­pa­ny’s plan, Deya Cor­zo, M.D., se­nior vice pres­i­dent of the liv­er and meta­bol­ic ther­a­peu­tic area, and Charles Richard, M.D., Ph.D., se­nior vice pres­i­dent of the cen­tral ner­vous sys­tem ther­a­peu­tic area, are on the way out of uniQure and a shrunk­en R&D group in Am­s­ter­dam will move in­to new digs next year.

The re­trench­ing comes af­ter uniQure has faced off against some tough com­pe­ti­tion in he­mo­phil­ia B, with Spark Ther­a­peu­tics and oth­ers ad­vanc­ing ri­val pro­grams.

Trou­ble has been brew­ing at uniQure for some time. It has one gene ther­a­py ap­proved in Eu­rope, but no mar­ket for it. In Sep­tem­ber, the biotech’s new CEO, Dan Soland, left abrupt­ly af­ter on­ly 9 months on the job. And that change­up came a few months af­ter uniQure fared poor­ly in the lat­est beau­ty con­test among the gene ther­a­pies be­ing de­vel­oped for he­mo­phil­ia B. A crowd­ed field, Spark Ther­a­peu­tics drew praise for achiev­ing 27% to 35% Fac­tor IX (FIX) ac­tiv­i­ty among 4 pa­tients, while uniQure had to set­tle for a 2% to 6.3% range for a sin­gle in­fu­sion of AMT-060 — though Spark re­cent­ly ex­pe­ri­enced a set­back of its own. Two months lat­er Shire opt­ed to drop its ri­val gene ther­a­py in the field, the one-time leader BAX 335, in fa­vor of a pre­clin­i­cal ef­fort af­ter an­a­lysts panned the vari­able re­sults the pro­gram had post­ed.

Matthew Ka­pus­ta, in­ter­im chief ex­ec­u­tive of­fi­cer of uniQure, had this to say:

“Along with our in­ves­ti­ga­tors and col­lab­o­ra­tion part­ner, we are en­thu­si­as­tic about the in­ter­im da­ta from our on­go­ing Phase I/II study of AMT-060 in he­mo­phil­ia B and will al­lo­cate nec­es­sary re­sources to ex­pe­dite bring­ing AMT-060 to mar­ket, with com­mer­cial man­u­fac­tur­ing ca­pa­bil­i­ties in our state-of-the-art U.S. fa­cil­i­ty which is al­ready in place.”

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.