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.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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