Ex-Ab­b­Vie part­ner Prin­cip­ia posts en­cour­ag­ing PhII re­sults for its BTK-in­hibitor

Months af­ter their breakup with high-pro­file part­ner Ab­b­Vie, Prin­cip­ia an­nounced pos­i­tive pre­lim­i­nary re­sults from the sec­ond half of a Phase II tri­al on their lead drug.

The San Fran­cis­co biotech an­nounced da­ta from part B of its Phase II open-la­bel tri­al test­ing the BTK in­hibitor PRN1008 on pa­tients with pem­phi­gus vul­garis, a rare au­toim­mune dis­ease af­fect­ing the skin and mu­cous mem­branes. Of 15 en­rolled pa­tients, 6 achieved com­plete re­spons­es and 4 re­main on the ther­a­py.

Nine of the pa­tients achieved a PDAI score – a 0-to-250 met­ric for the sever­i­ty of blis­ter­ing dis­eases – of one or ze­ro.

Mar­tin Babler

Part B mea­sured the drug over a 24-week pe­ri­od rather than Part A’s 12-week pe­ri­od, and found im­prove­ment: The per­cent­age of pa­tients with a com­plete re­cov­ery went from 25% to 40%.

Baird’s Bri­an Sko­r­ney wrote that the re­sults bod­ed well for Prin­cip­ia as it con­tin­ues with a Phase III study on the same com­pound that be­gan last year and is still un­der­way. That tri­al will dose pa­tients for 37 weeks. It aims at 120 en­rollees and will be com­plet­ed at the end of 2021.

“We be­lieve that these re­sults fur­ther sup­port the util­i­ty of BTK in­hi­bi­tion in pem­phi­gus and should fur­ther de­risk this pro­gram as Prin­cip­ia looks ahead to top-line da­ta from the Phase 3 PE­GA­SUS which are ex­pect­ed in 1H22,” Sko­r­ney wrote, adding that the longer dos­ing pe­ri­od in Phase III may yield more com­plete re­mis­sions.

PRN1008 re­ceived FDA or­phan drug sta­tus for pem­phi­gus vul­garis in 2017. The dis­ease, which caus­es blis­ter­ing and ul­cers and is spurred by au­toan­ti­bod­ies break­ing the bond be­tween skin cells, af­fects 40,000 Amer­i­cans and 170,000 peo­ple world­wide.

As re­cent­ly as March, Ab­b­Vie was work­ing on ear­ly-stage re­search with Prin­cip­ia un­der a 2017 deal that gave Prin­cip­ia $15 mil­lion up­front and $667.5 mil­lion in po­ten­tial mile­stones. Prin­cip­ia abrupt­ly an­nounced in March that Ab­b­Vie had de­cid­ed to end the col­lab­o­ra­tion.

“Ab­b­Vie has com­plet­ed an ini­tial eval­u­a­tion of Prin­cip­ia’s high­ly se­lec­tive, oral­ly bioavail­able co­va­lent in­hibitors of the im­muno­pro­tea­some and, af­ter an as­sess­ment of their bi­o­log­ic pro­files rel­a­tive to Ab­b­Vie’s de­sired dis­ease ar­eas of fo­cus, has de­ter­mined that there is no longer a strate­gic fit,” Prin­cip­ia CEO Mar­tin Babler said at the time.

That news came nine days af­ter Prin­cip­ia pub­licly an­nounced pos­i­tive re­sults from their Phase II, part A study.

The au­toim­mune spe­cial­ist went pub­lic last year for an up­sized $106 mil­lion on the strength of its line of BTK in­hibitors. The Ab­b­Vie de­par­ture leaves it with a main ma­jor col­lab­o­ra­tor in Sanofi. In 2017, the French gi­ant li­censed PRN2246, their BTK in­hibitor for mul­ti­ple scle­ro­sis, for $40 mil­lion up­front and $765 mil­lion in po­ten­tial mile­stones.

BTK (Bru­ton’s ty­ro­sine ki­nase) in­hibitors have long been a ma­jor play­er in hema­tol­ogy but in re­cent years they have al­so be­come a tar­get for au­toim­mune re­searchers – a nat­ur­al leap giv­en they act on an en­zyme in B cells, a crit­i­cal part of the body’s im­mune sys­tem.

Re­sults, though, have been mixed thus far. Mer­ck KGaA an­nounced last month they were mov­ing their BTK, evo­bru­ti­nib, in­to Phase III for mul­ti­ple scle­ro­sis de­spite mixed Phase IIb re­sults. Last year, Eli Lil­ly wrote off a BTK for rheuma­toid arthri­tis it had li­censed from Ko­rea’s Han­mi, lead­ing to the col­lapse of a $690 mil­lion pact. Cel­gene sim­i­lar­ly saw its au­to-im­mune BTK fiz­zle in Phase II, while Bris­tol-My­ers Squibb has giv­en few up­dates on its ear­ly-stage BTK pro­gram.

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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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

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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