Bol­stered by promis­ing PhI­Ib snap­shot, uniQure plays catch-up with Spark in the race to a he­mo­phil­ia B gene ther­a­py

The piv­otal lead switch that uniQure ex­e­cut­ed late last year ap­pears to have paid off, as the gene ther­a­py pi­o­neer of­fers some pos­i­tive, if ear­ly, num­bers for its tweaked he­mo­phil­ia B treat­ment.

Robert Gut

Among three pa­tients treat­ed with AMT-061 in the dose-con­fir­ma­tion study, re­searchers re­port­ed a mean fac­tor IX lev­el that rep­re­sent­ed 31% of nor­mal lev­els at six weeks, ex­ceed­ing the 12% com­mon­ly re­gard­ed as suf­fi­cient to sub­stan­tial­ly re­duce spon­ta­neous bleeds. A quick break­down by in­di­vid­ual showed FIX ac­tiv­i­ty in the first pa­tient was 37% of nor­mal 10 weeks af­ter ad­min­is­tra­tion; in the sec­ond pa­tient, 23% at week 8; and the third, 30% at week 6.

Hap­py to hear that none of the pa­tients re­ceived fac­tor in­fu­sions, ex­pe­ri­enced re­port­ed bleed­ing events or re­quired im­muno­sup­pres­sion, in­vestors sent the stock up $QURE 39%.

Ed­ward Nash

It’s al­so mu­sic to the ears of some sup­port­ive an­a­lysts, who are call­ing the re­sults “very good” and “a clear pos­i­tive” and large­ly agree­ing with ex­ecs that a “mild, asymp­to­matic and tran­sient in­crease in liv­er en­zyme lev­els” ob­served in the tri­al was not a cause for con­cern.

The re­sults af­firm uniQure’s com­pet­i­tive po­si­tion with Spark Ther­a­peu­tics, which is al­so de­vel­op­ing a he­mo­phil­ia B ther­a­py to fol­low up its his­toric reti­na eye dis­ease treat­ment.

Ed­ward Nash of Sun­Trust notes:

As a ref­er­ence, SPK-9001 from Spark showed in a Phase I/II study that the range of FIX ac­tiv­i­ty lev­els was be­tween 14% and 68% at Week 6 and be­tween 16% and 78% at Week 8.

Joseph Schwartz

An­oth­er de­tail that may sep­a­rate uniQure from Spark, Leerink’s Joseph Schwartz points out, is that AMT-061 may be able to tar­get a broad­er spec­trum of pa­tients. Two of the three pa­tients in uniQure’s Phase IIb tri­al were en­rolled af­ter pre­vi­ous­ly screen-fail­ing an­oth­er gene ther­a­py study due to pre-ex­ist­ing neu­tral­iz­ing an­ti­bod­ies to a dif­fer­ent AAV vec­tor. In fact, ex­ecs were so con­fi­dent about the AAV5 vec­tor they have vowed not to ex­clude any pa­tients on the pa­tients of pre-ex­ist­ing neu­tral­iz­ing an­ti­bod­ies.

Am­s­ter­dam- and Lex­ing­ton, MA-based uniQure re­placed its lead pro­gram, AMT-060, with AMT-061 last Oc­to­ber, claim­ing that a new gene vari­ant called FIX-Pad­ua in­tro­duced in the tweaked as­set will boost FIX ac­tiv­i­ty while keep­ing the same de­liv­ery tech and man­u­fac­tur­ing process.

“Based on the long-term FIX da­ta from our Phase I/II tri­al of AMT-060, where we saw FIX ac­tiv­i­ty lev­els con­tin­ue to in­crease be­yond the lev­els achieved at six to ten weeks, we are hope­ful that we will con­tin­ue to see sim­i­lar trends in these pa­tients,” said CMO Robert Gut in a state­ment.

Catch­ing up with Spark and its Big Phar­ma al­lies at Pfiz­er, uniQure plans to be­gin dos­ing for a Phase III study in Q1 2019.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Mar­ket­ingRx roundup: Ab­b­Vie’s Hu­mi­ra TV turns fo­cus to HS skin con­di­tion; Sanofi amps par­ent­ing pol­i­cy

After years as the top spending pharma TV advertiser, AbbVie’s Humira brand finally downshifted earlier this year, ceding much of its marketing budget to up-and-coming sibling meds Skyrizi and Rinvoq. However, now Humira is back on TV with ads for another condition — Hidradenitis suppurativa (HS).

The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.