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.”

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.

Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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