Ahead of ad­u­canum­ab fil­ing, Bio­gen nabs Pfiz­er castoff for $75M to ad­dress Alzheimer's, Parkin­son's symp­toms

Bio­gen be­lieves (not every­one agrees) it has a dis­ease-mod­i­fy­ing ther­a­py for Alzheimer’s on the cusp of an FDA ap­proval. Now it’s bring­ing in an ear­ly drug to al­so treat one of its symp­toms.

Al San­drock

PF-05251749 is an in­hibitor of ca­sein ki­nase 1, a fam­i­ly of en­zymes key to the reg­u­la­tion of cir­ca­di­an rhythm, that Pfiz­er had tak­en through Phase Ia be­fore culling its en­tire neu­ro­sciences unit. While some as­sets were sal­vaged in the form of a spin­out, this ap­par­ent­ly went up for sale and it’s go­ing for $75 mil­lion up­front. Bio­gen has com­mit­ted to $635 mil­lion in mile­stones.

In ad­di­tion to sun­down­ing in Alzheimer’s — a state of con­fu­sion, anx­i­ety, ag­gres­sion, ag­i­ta­tion or rest­less­ness be­set­ting about 20% of all pa­tients in the evenings — Bio­gen al­so sees po­ten­tial in a non-mo­tor symp­tom of Parkin­son’s dubbed ir­reg­u­lar sleep wake rhythm dis­or­der. Pa­tients with ISWRD have trou­ble sleep­ing at night and ex­pe­ri­ence sub­se­quent day­time sleepi­ness and se­vere fa­tigue, dis­rupt­ing their dai­ly lives.

At Pfiz­er, PF-05251749 had been test­ed in around 100 healthy vol­un­teers (in one case in con­junc­tion with mela­tonin). Bio­gen will now ini­ti­ate its own Phase Ib stud­ies for each of the two in­di­ca­tions it high­light­ed

For Baird an­a­lysts, it’s all a bit rem­i­nis­cent of the BMS-986168 (go­suranemab) deal from 2017, wrote Bri­an Sko­r­ney.

Back then Bio­gen paid $300 mil­lion up­front — siz­able, as with this new deal — to ac­quire go­suranemab from Bris­tol-My­ers Squibb — an­oth­er big phar­ma part­ner.

“Re­call, we re­cent­ly not­ed that go­suranemab flopped in the piv­otal Phase 2 PASS­PORT tri­al in pa­tients with pro­gres­sive supranu­clear pal­sy (PSP),” he added. “With re­spect to go­suranemab, it seems that Bio­gen got the short end of the stick, how­ev­er, on­ly time will tell as to whether or not the case will be the same with re­spect to PF-05251749.”

Al San­drock, Bio­gen head of R&D and big ad­u­canum­ab cheer­leader, re­mains op­ti­mistic about the po­ten­tial to ad­dress be­hav­ioral and neu­ro­log­i­cal symp­toms us­ing a CNS-pen­e­trant drug.

“This as­set is high­ly com­ple­men­tary to our ex­ist­ing pipeline of po­ten­tial dis­ease-mod­i­fy­ing ther­a­pies in Alzheimer’s and Parkin­son’s dis­eases,” San­drock, who’s al­so held on­to the CMO ti­tle, for now, said in a state­ment.

The deal kicks off a year when Alzheimer’s will loom ever larg­er at Bio­gen, as the com­pa­ny be­gins to find out whether its de­ci­sion to res­ur­rect the ad­u­canum­ab pro­gram fol­low­ing post hoc analy­sis of two Phase III stud­ies (one of which still failed re­gard­less) will fly with FDA reg­u­la­tors. The biotech has said it’d file for ap­proval in ear­ly 2020.

Their the­o­ry is that pa­tients need­ed to be con­sis­tent­ly ex­posed to a high-dose — 14 dos­es at 10mg/kg — as well as a bet­ter titra­tion reg­i­men to reap the full cog­ni­tive ben­e­fits of ad­u­canum­ab. But crit­ics slammed the ar­gu­ment, con­tend­ing that even if that were true, it would have to be borne out by a third, con­fir­ma­to­ry study be­fore the be­ta-amy­loid bind­ing an­ti­body can be ap­proved.

“We don’t file willy nil­ly,” San­drock pre­vi­ous­ly said in de­fense of the de­ci­sion to shoot for an OK with what they have. “We on­ly go to fil­ing when we be­lieve that there is a ben­e­fit-risk ar­gu­ment based on sci­ence, based on da­ta. And if you look at our his­to­ry, we haven’t done fil­ings right and left with­out good rea­son.”

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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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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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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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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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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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

Covid-19 roundup: Vac­cines will need to beat place­bo by 50% to qual­i­fy for FDA OK; UK tri­al drops Kale­tra

The FDA will set the bar for approving a Covid-19 vaccine at 50% efficacy, the Wall Street Journal reported, meaning any successful candidate will have to reduce the risk of coronavirus disease by at least half compared to placebo.

That requirement is part of guidance that the agency is set to release later today, laying out detailed criteria for vaccine developers — some of whom are eyeing an OK by the end of the year, in line with expectations at Operation Warp Speed.

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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.