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.”

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

In search of elu­sive NASH break­through, Pfiz­er spot­lights com­bo ap­proach

Pfizer’s second crack at steering a NASH candidate through a battered field seems to be going better than the first.

The pharma giant has scored the FDA’s fast track designation for an experimental combination therapy as a treatment for NASH with liver fibrosis. The combo consists of ervogastat, a diacylglycerol O-acyltransferase 2 inhibitor (DGAT2i), and clesacostat, an acetyl-CoA carboxylase inhibitor (ACCi).

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

AS­CO ab­stracts mint some ear­ly win­ners, but Io­vance, Spring­Works get hit by mas­sive loss­es

Before cancer-focused biotechs start their trek to the first in-person American Society of Clinical Oncology (ASCO) annual conference since the pandemic began, investors have taken a good look at the teasers for the data scheduled to be presented — and started placing bets.

With its power to confer overnight fame, ASCO is a stage where impressive or surprise debut performances can go a long way. On the other hand, disappointing details could be punishing.

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Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Levi Garraway, Roche CMO (Genentech)

Roche's CD20xCD3 does­n't beat Gen­mab at ORR, but sets bar for CR da­ta on lym­phoma drug

On its way to potentially becoming the first to market with a CD20xCD3 bispecific for aggressive lymphoma, Big Pharma’s largest R&D spender has some more data to pad its case with an FDA filing slated for later this year.

Roche dropped some more details from a Phase II expansion study of its fixed-duration glofitamab, to be presented at next week’s ASCO annual meeting, in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). The patients had received a median of three prior therapies.

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