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.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,600+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

José Baselga, AstraZeneca cancer chief (Brent N. Clarke/FilmMagic via Getty Images)

As­traZeneca's Calquence nabs an­oth­er win against Im­bru­vi­ca, but Eli Lil­ly is on its heels

Three years after first launching Calquence as a second generation BTK inhibitor, AstraZeneca continues to tout new data to compete with J&J and AbbVie’s first generation blockbuster Imbruvica.

The British pharma announced on Monday that Calquence passed a head-to-head Phase III study against Imbruvica in chronic lymphocytic leukemia, proving non-inferior — i.e. just as good — as the older drug. Although AstraZeneca did not break down any of the numbers, they said the drug proved superior on safety, triggering fewer cases of atrial fibrillation, an irregular heartbeat that can lead to stroke or heart failure.

Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Roche amps up its bis­pe­cif­ic at­tack on Eylea with more PhI­II da­ta — but just how threat­en­ing is it?

Roche has another stack of data to back up its longer-acting challenger to Eylea — although it’s still far from certain just how much they can threaten Regeneron’s dominance.

The latest Phase III results come from two trials that enrolled 1,329 patients with neovascular age-related macular degeneration. With 45% of people in both studies getting faricimab 16 weeks apart during the first year, the bispecific still induced the same level of gains in visual acuity as Eylea every 8 weeks did, Roche’s Genentech reported.

Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.