Roche says its new in­ter­im up­date on he­mo­phil­ia A drug is pos­i­tive. But it’s hard to gauge.

Roche to­day sent out a small, blur­ry snap­shot of the in­ter­im da­ta from a Phase III study of emi­cizum­ab in chil­dren suf­fer­ing from he­mo­phil­ia A.

What did we find out?

San­dra Horn­ing

The da­ta are pos­i­tive and “clin­i­cal­ly mean­ing­ful” — though pre­sum­ably not sta­tis­ti­cal­ly sig­nif­i­cant, which is the phrase you look for in de­ter­min­ing suc­cess or fail­ure — in cut­ting the bleed rate among chil­dren 12 or younger with in­hibitors to Fac­tor VI­II.

The most com­mon ad­verse events: in­jec­tion site re­ac­tions and na­sopharyn­gi­tis.

Any se­ri­ous ad­verse events, like the ones seen in HAVEN 1?

In a fol­lowup, a spokesper­son tells me: “No throm­boem­bol­ic (TE) events or cas­es of throm­bot­ic mi­croan­giopa­thy (TMA) oc­curred in this study pop­u­la­tion (HAVEN 2).”

And as for the “clin­i­cal­ly mean­ing­ful” bot­tom line, she adds: “(G)iv­en the study de­sign is a sin­gle arm (no com­para­tor), we can’t say “sta­tis­ti­cal­ly sig­nif­i­cant” be­cause there is no hy­poth­e­sis test­ing, but the re­duc­tion is clin­i­cal­ly mean­ing­ful to treaters and pa­tients. This type of study de­sign is stan­dard for pe­di­atric stud­ies in he­mo­phil­ia. We look for­ward to shar­ing de­tails of the bleed rates when the da­ta are pre­sent­ed at con­gress. We’ve sub­mit­ted to ISTH in Ju­ly.”

Fair enough.

Roche’s re­cent suc­cess in gain­ing an ap­proval for Ocre­vus in mul­ti­ple scle­ro­sis set them on a di­rect course to dis­rupt­ing the mul­ti­ple scle­ro­sis field, with an­oth­er like­ly block­buster to add to a list that al­so in­cludes the PD-L1 check­point Tecen­triq. Emi­cizum­ab, bet­ter known as ACE910, is in­tend­ed to be the next on the list with peak sales es­ti­mates from an­a­lysts hov­er­ing around $1.4 bil­lion.

Two months ago Roche trig­gered fresh doubts about its drug af­ter a pa­tient in HAVEN 1 died fol­low­ing two se­ri­ous ad­verse events. The death spurred fresh ques­tions about the drug’s safe­ty when in­ves­ti­ga­tors had to fend off per­sis­tent ques­tions about 4 spon­ta­neous­ly re­port­ed SAEs af­ter two pa­tients had throm­boem­bol­ic events and two pa­tients de­vel­oped throm­bot­ic mi­croan­giopa­thy, or TMA. That news helped briefly buoy Shire and No­vo Nordisk, which both see a big ri­val to their block­buster he­mo­phil­ia fran­chis­es in emi­cizum­ab.

Two more Phase III stud­ies in the pro­gram are on­go­ing.

“Man­ag­ing haemophil­ia A with in­hibitors to fac­tor VI­II is es­pe­cial­ly chal­leng­ing for chil­dren and their care­givers, be­cause bleed­ing is dif­fi­cult to con­trol and cur­rent treat­ments re­quire fre­quent in­tra­venous in­fu­sions”, said San­dra Horn­ing, Roche’s CMO and prod­uct de­vel­op­ment chief. “We are en­cour­aged that once-week­ly sub­cu­ta­neous emi­cizum­ab pro­phy­lax­is showed a clin­i­cal­ly mean­ing­ful re­duc­tion in the num­ber of bleeds over time in chil­dren and are pleased to share these re­sults with the com­mu­ni­ty as we join in cel­e­brat­ing World He­mo­phil­ia Day.”

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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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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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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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.