In a PhI­II show­down with Spark, uniQure switch­es out its lead gene ther­a­py for he­mo­phil­ia B

Late last year, the strug­gling gene ther­a­py biotech uniQure $QURE did some painful cost cut­ting, drop­ping pro­grams, ax­ing staff and tai­lor­ing the pipeline to con­cen­trate on a he­mo­phil­ia B pro­gram for AMT-060, which they said could be hus­tled through a piv­otal pro­gram and in­to the mar­ket, rac­ing with a ri­val ther­a­py from in­dus­try leader Spark Ther­a­peu­tics.

Steven Pipe

This morn­ing, though, uniQure ex­e­cut­ed a quick switch on its lead ef­fort. AMT-060, which had been des­ig­nat­ed as a break­through drug at the FDA, has been re­placed by AMT-061. And the ex­ecs at uniQure say that both the FDA and the EMA have es­sen­tial­ly de­cid­ed to keep this new drug in their VIP drug pro­gram and on the fast track to a reg­is­tra­tion tri­al.

In­vestors loved it. UniQure’s stock shot up more than 60% af­ter the news hit.

The way uniQure makes this sound, re­searchers tweaked their orig­i­nal ther­a­py to come up with some­thing bet­ter. In their state­ment, the biotech says that AMT-060 and AMT-061 are iden­ti­cal ex­cept for “two nu­cleotide sub­sti­tu­tions in the cod­ing se­quence for FIX.”

The gene vari­ant, re­ferred to as FIX-Pad­ua, ex­press­es a pro­tein with a sin­gle amino acid sub­sti­tu­tion that has been re­port­ed in mul­ti­ple pre­clin­i­cal and non­clin­i­cal stud­ies to pro­vide an ap­prox­i­mate 8- to 9-fold in­crease in FIX ac­tiv­i­ty com­pared to the wild-type FIX pro­tein. All oth­er crit­i­cal qual­i­ty at­trib­ut­es of AMT-061 are ex­pect­ed to be com­pa­ra­ble to those of AMT-060, as AMT-061 uti­lizes the same AAV5 cap­sid and pro­pri­etary in­sect cell-based man­u­fac­tur­ing plat­form.

They al­so want to style this new AMT-061 as a much, much bet­ter ther­a­py.

Steven Pipe, pro­fes­sor of pe­di­atrics and pathol­o­gy and pe­di­atric med­ical di­rec­tor of the he­mo­phil­ia and co­ag­u­la­tion dis­or­ders pro­gram at the Uni­ver­si­ty of Michi­gan, had this to say:

“Based on the da­ta gen­er­at­ed to date, AMT-061 may be the first gene ther­a­py to pro­vide durable, cu­ra­tive ben­e­fits to near­ly all pa­tients with he­mo­phil­ia B, with­out the com­pli­ca­tions as­so­ci­at­ed with cap­sid-re­lat­ed im­mune re­spons­es.  I very much look for­ward to serv­ing as an in­ves­ti­ga­tor in this ex­cit­ing Phase III pro­gram.”

Once a leader in gene ther­a­py, uniQure had to drop Gly­bera — ap­proved in Eu­rope but nev­er pitched in the US — af­ter it was re­port­ed­ly used just one time. Now Spark ap­pears to be in the lead af­ter an FDA pan­el en­dorsed its gene ther­a­py for a rare eye dis­ease. And one-time play­er Di­men­sion Ther­a­peu­tics was forced out of he­mo­phil­ia B by weak re­sults, with Ul­tragenyx pick­ing up the rest of the pipeline in a buy­out.

The race goes on.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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