With new name, new CEO, Cas­ca­di­an now rolls out a new piv­otal plan for lead can­cer drug

Cas­ca­di­an CEO Scott My­ers

Cas­ca­di­an $CASC changed its name (from On­cothyre­on) and in­stalled a new CEO in the hope of shed­ding the seem­ing curse that dogged al­most every step at the biotech. And now it has a new, ac­cel­er­at­ed de­vel­op­ment pro­gram in place for its lead drug that puts it square­ly in the piv­otal phase.

The drug tu­ca­tinib — for­mer­ly known as ONT-380 in the pre-makeover days — is a HER2 in­hibitor that has been matched up with Her­ceptin and Xelo­da in com­bat­ting treat­ment-re­sis­tant breast can­cer. It’s a pill, in­tend­ed to get through the blood-brain bar­ri­er to help some des­per­ate­ly ill pa­tients.

In new Phase Ib da­ta, the triplet scored a me­di­an pro­gres­sion-free sur­vival rate of 7.8 months, an over­all re­sponse rate of 61% and a me­di­an du­ra­tion of re­sponse of 10 months.

With that in mind, CEO Scott My­ers, who stepped in last sum­mer, went back to the FDA and asked for a makeover on the Phase II study. And My­ers says that the FDA signed off, agree­ing to let Cas­ca­di­an in­crease en­roll­ment in the mid-stage study to 480 and make it a reg­is­tra­tional tri­al in the process. It’s al­ready en­rolling in the US and Cana­da, and Cas­ca­di­an will now add sites in Eu­rope, Aus­tralia and Is­rael.

Long­time biotech ob­servers will re­call that the com­pa­ny suf­fered through a se­ries of set­backs. Its can­cer vac­cine Stimu­vax helped es­tab­lish the ac­cept­ed view that the first wave of can­cer vac­cines has been a bust. Mer­ck KGaA tried, twice, with big late-stage ef­forts on that and failed.

The FDA’s ap­proval of the ac­cel­er­at­ed time­line for tu­ca­tinib fits in with its en­thu­si­as­tic en­dorse­ment of find­ing ways to ac­cel­er­ate R&D in on­col­o­gy. Of all the var­i­ous dis­ci­plines at the FDA, the on­col­o­gy group has been will­ing to sign off on some of the most dra­mat­ic ways to speed up de­vel­op­ment, carv­ing years out of the process in some cas­es.

Cas­ca­di­an still has a long way to go be­fore it can claim a come­back. Its mar­ket cap is a small $138 mil­lion. But it does have a plan for chang­ing the nar­ra­tive.

My­ers had this to say:

“We look for­ward to con­tin­u­ing a col­lab­o­ra­tive re­la­tion­ship with the agency and our clin­i­cal in­ves­ti­ga­tors as we ad­vance the de­vel­op­ment of tu­ca­tinib in com­bi­na­tion in the third-line metasta­t­ic breast can­cer set­ting where there is no sin­gle stan­dard-of-care and a need for more tol­er­a­ble ther­a­peu­tic op­tions. The im­prove­ment in the up­dat­ed da­ta from our Phase 1b “triplet com­bi­na­tion” study re­in­forces our strat­e­gy with tu­ca­tinib in this pa­tient pop­u­la­tion.”

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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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.