Rigel’s 2nd PhI­II for fos­ta­ma­tinib scut­tled by a sin­gle place­bo re­sponse, shares crater

Rigel Phar­ma­ceu­ti­cals con­tin­ues to be plagued by a low re­sponse rate in its big Phase III pro­gram for fos­ta­ma­tinib. But un­like its first late-stage da­ta drop, which scored a hit on the pri­ma­ry end­point, the biotech now has to ex­plain why its sec­ond con­fir­ma­to­ry study failed.

Raul Ro­driguez, Rigel CEO

It’s not an en­vi­able task. At the end of Au­gust the South San Fran­cis­co-based biotech’s share price $RIGL went on a roller coast­er ride with a pos­i­tive but unim­pres­sive 18% re­sponse rate for im­mune throm­bo­cy­tope­nia pa­tients who achieved a sta­ble platelet re­sponse. That was ex­act­ly the same rate seen in the sec­ond Phase III, on­ly this time one pa­tient on a place­bo al­so achieved a sta­ble platelet re­sponse, and that scut­tled any shot at hit­ting the pri­ma­ry end­point. For an­a­lysts who had been look­ing for a re­sponse rate at least in the low 20s with clear signs of ef­fi­ca­cy, this stum­ble will not play well.

In­vestors didn’t care for news, ei­ther. Rigel’s shares plunged 33% in ear­ly trad­ing Thurs­day.

Rigel CEO Raul Ro­driguez, though, says if you add all the da­ta from both Phase III stud­ies to­geth­er, you still come up with a pos­i­tive out­come. And that’s what they want to take to the FDA.

“We be­lieve that the to­tal­i­ty and con­sis­ten­cy of da­ta from the FIT Phase III pro­gram, which in­clud­ed two Phase III stud­ies and one long-term ex­ten­sion study, strong­ly sup­ports a clear treat­ment ef­fect, with a sus­tained clin­i­cal ben­e­fit of fos­ta­ma­tinib,” said Ro­driguez in a state­ment.  “We are en­cour­aged by these re­sults and be­lieve that the risk/ben­e­fit ra­tio for fos­ta­ma­tinib is pos­i­tive for pa­tients with chron­ic/per­sis­tent ITP, a pop­u­la­tion with a se­ri­ous un­met med­ical need.  As a re­sult, we will con­tin­ue to pur­sue this op­por­tu­ni­ty. Our next step is to seek feed­back from the FDA.”

The ther­a­py is a spleen ty­ro­sine ki­nase (Syk) in­hibitor, which should help stop the bleed­ing that oc­curs when some­one has a low platelet count. But in a small proof-of-con­cept study for throm­bo­cy­tope­nia, the ther­a­py scored a 50% re­sponse rate, which had whet­ted ap­petites for some­thing sol­id in Phase III.

There’s no turn­ing back now, though. Rigel laid off close to half of its staff last month as it cleared the decks for an FDA ap­pli­ca­tion and launch plans.

Back in 2013 fos­ta­ma­tinib flopped in a Phase III study of rheuma­toid arthri­tis, forc­ing As­traZeneca to wash its hands of their part­ner­ship and leav­ing Rigel to un­der­go an over­haul, shed­ding staff and re­or­ga­niz­ing around dry eye dis­eases (R348) and lu­pus (R333). They both quick­ly failed Phase II. An­oth­er drug, R118, had to be aban­doned af­ter in­ves­ti­ga­tors tracked some se­ri­ous side ef­fects.

George Yancopoulos (Regeneron)

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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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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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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No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.

Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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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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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.