En­cour­ag­ing gene ther­a­py da­ta give uniQure an in­jec­tion of con­fi­dence as it preps PhI­II show­down with Spark

Gene ther­a­py com­pa­ny uniQure start­ed the week in style with the news that it’s dosed the first pa­tient in its Phase III tri­al — a cru­cial step to­ward its quest for the best-in-class, first-in-mar­ket ti­tle in he­mo­phil­ia B gene ther­a­py. And to­day it’s of­fer­ing an up­date on the kind of mid-stage da­ta it’s armed with.

Matt Ka­pus­ta

Mean Fac­tor IX lev­els hit 38% of nor­mal lev­els 12 weeks af­ter the three pa­tients tracked in the Phase IIb study re­ceived the one-time in­jec­tion, reach­ing what the com­pa­ny calls “the nor­mal range” and well above the 12% com­mon­ly re­gard­ed as suf­fi­cient to sub­stan­tial­ly re­duce spon­ta­neous bleeds.

It’s the first look at da­ta three months post-ad­min­is­tra­tion — sig­nif­i­cant be­cause most pa­tients don’t be­gin to start plateau­ing un­til that point, CEO Matt Ka­pus­ta tells me in a pre­view of the re­sults, which fol­lows an up­beat snap­shot in No­vem­ber on six-week da­ta.

So have they reached the peak in FIX lev­els? Prob­a­bly not, Ka­pus­ta said.

The best way to glean that is we ac­tu­al­ly did a Phase I/II study with the first gen­er­a­tion prod­uct that is near­ly iden­ti­cal to the AMT-061 and in­clud­ed pa­tients that were at the same dose. […] Of the 9 pa­tients that were off of pro­phy­lax­is, 8 of them had Fac­tor IX ac­tiv­i­ty that was high­er at 52 weeks or — their lat­est mea­sure­ment was high­er than what it was at 12 weeks. […] So we don’t know if that will con­tin­ue to be the case, but that’s prob­a­bly — look­ing at those ki­net­ics from the Phase I/II study sug­gests that there is po­ten­tial for mean­ing­ful in­creas­es go­ing for­ward.

Since the pa­tients be­gan dos­ing at dif­fer­ent times, uniQure al­so gleaned in­sights in­to the (slight­ly) longer term FIX ac­tiv­i­ties: 48% for the first pa­tient at week 16; 25% for the sec­ond pa­tient at week 14; and 51% for the third pa­tient at 12 weeks.

“It’s not un­usu­al to have a three, maybe four-fold mul­ti­ple be­tween your min­i­mum and your max­i­mum,” Ka­pus­ta says. “Here of course we’ve got about a two-fold. So in our view the vari­abil­i­ty is ac­tu­al­ly tighter.”

The new round of da­ta drew uni­ver­sal ap­plause from an­a­lysts, with Chardan spec­u­lat­ing that the com­pa­ny is now a prime takeover tar­get. Shares are up 4%.

A chief ri­val of Spark Ther­a­peu­tics — which is tap­ping in­to he­mo­phil­ia as a fol­low-up to its ground­break­ing gene ther­a­py for the eye — Lex­ing­ton, MA- and Am­s­ter­dam-based uniQure $QURE pleased in­vestors and an­a­lysts back in No­vem­ber when it re­port­ed mean FIX lev­els of 31% at six weeks.

While care­ful to note that “we don’t love com­ment­ing on oth­er peo­ple’s da­ta,” Ka­pus­ta cit­ed the high lev­els of FIX ac­tiv­i­ty, low im­muno­genic­i­ty — none of uniQure’s pa­tients have re­quired im­muno­sup­pres­sion so far — and the fact that it doesn’t ex­clude pa­tients with pre-ex­ist­ing an­ti­bod­ies to AAV vec­tors as rea­sons to be­lieve that they have a “high­ly com­pa­ra­ble if not po­ten­tial­ly an op­ti­mized or su­pe­ri­or tar­get prod­uct pro­file.”

But the re­al show­down will take place in the piv­otal stage, as uniQure catch­es up with a Phase III pro­gram that Spark part­ner Pfiz­er be­gan last sum­mer.

With 20 ac­tive sites across the US and Eu­rope, uniQure’s Phase III is ex­pect­ed to en­roll 50 pa­tients by the end of the year. Top-line da­ta on the pri­ma­ry end­point — FIX ac­tiv­i­ty af­ter six months — should be ready in late 2020, paving the way for a BLA sub­mis­sion in 2021, Ka­pus­ta said.

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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