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.”

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus -- chop­ping di­a­betes, car­dio and slash­ing costs in com­pa­ny-wide re­org

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy reveal tomorrow with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

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Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

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KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.