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.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Jan van de Winkel, Genmab CEO

Seat­tle Ge­net­ics, Gen­mab turn on TV for a high­light reel in cer­vi­cal can­cer — but a ri­val biotech promis­es a bet­ter show

Seattle Genetics $SGEN and their partners at Genmab $GMAB polished up some positive Phase II numbers for their antibody drug conjugate tisotumab vedotin — you can call it TV — for recurrent cervical cancer. And while they mapped out a shortcut to a potential quick approval, the big challenge for this team is being presented by a rival biotech which muscled its way into the spotlight for the same indication a year ago.

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Paul Tesar (Convelo Therapeutics)

Io­n­is, lead­ing MS re­searcher throw an­ti­sense at a new type of brain cells

No matter how many molecules he threw at them, Paul Tesar couldn’t get the brain cells to survive. Or he got them to survive, but then — to everyone’s bafflement — they still couldn’t do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.