The race to best treat a dis­ease long con­fused with MS heats up with Roche's PhI­II re­sults

With in­flamed myelin and fre­quent op­tic neu­ri­tis episodes, NMOSD pa­tients were for years con­fused with MS pa­tients. Now mul­ti­ple treat­ments for the rare au­toim­mune dis­ease, of­fi­cial­ly called neu­romyelitis op­ti­ca spec­trum dis­or­der, are out or near­ing ap­proval.

Roche just pub­lished Phase III da­ta on its NMOSD drug satral­izum­ab in the New Eng­land Jour­nal of Med­i­cine, the sec­ond pos­i­tive round of re­sults. Of the 41 NMOSD pa­tients who re­ceived the IL-6 an­ti­body, 89% didn’t ex­pe­ri­ence a re­lapse af­ter 48 weeks. Mean­while, 66% of the 42 pa­tients in the place­bo were re­lapse-free at that point.

Un­like mul­ti­ple scle­ro­sis, where dis­abil­i­ty is caused by over­all dis­ease pro­gres­sion, NMOSD strikes through at­tacks or re­laps­es that cu­mu­la­tive­ly dis­able pa­tients. Enough re­laps­es can even­tu­al­ly cause blind­ness or para­ple­gia. If you pre­vent the re­laps­es, you can halt the dis­ease.

Af­ter 96 and 144 weeks, 78% and 74% of the treat­ment arm were re­lapse-free. That com­pared with 59% and 49% in the con­trol arm. These were sim­i­lar to the num­bers shown in a sep­a­rate study un­veiled in Sep­tem­ber.

The drug al­so showed a height­ened re­sponse in pa­tients with a spe­cif­ic bio­mark­er, AQP4-IgG or aqua­por­in-4, which of­ten ap­pears in pa­tients with the harsh­est symp­toms. Near­ly 4x as many AQP4 pa­tients re­lapsed in the place­bo arm as in the treat­ment arm.

Com­bined with the Sep­tem­ber re­sults and an FDA break­through tag from 2018, the lat­est da­ta should help ush­er Roche’s drug past US reg­u­la­tors.

It would join Alex­ion’s Soliris, which be­came the first drug specif­i­cal­ly ap­proved for NMOSD, al­so known as De­vic’s Dis­ease, in June. And it may fol­low an­oth­er can­di­date in Viela Bio’s inebi­lizum­ab. The As­traZeneca spin­out sub­mit­ted a BLA in late Au­gust for inebi­lizum­ab based off of a piv­otal show­ing 88% in the drug arm went with­out re­lapse.

Most NMOSD pa­tients have used off-la­bel Rit­ux­an — a drug not specif­i­cal­ly ap­proved for the in­di­ca­tion but one that’s staved off symp­toms to a de­gree that some an­a­lysts doubt whether new ther­a­pies will be able to pen­e­trate the mar­ket.

The ques­tion is whether the ben­e­fit of new treat­ments jus­ti­fies the cost, SVB Leerink’s Ge­of­frey Porges wrote af­ter the Soliris ap­proval. For Soliris, the price in the first year is $700,000, com­pared to $60,000 for off-la­bel Rit­ux­an.

For Roche and Viela Bio, the pres­sure to price well will be even steep­er. Over 96% of pa­tients in the Soliris tri­al hadn’t re­lapsed af­ter 144 weeks.

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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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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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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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.

Look­ing for 'ex­ter­nal in­no­va­tion,' Boehringer In­gel­heim re­serves $500M+ for new Shang­hai hub

Now that Boehringer Ingelheim’s bet on contract manufacturing in China has paid off, the German drugmaker is anteing up more to get into the research game.

Boehringer has set aside $507.9 million (€451 million) for a new External Innovation Hub to be built in Shanghai over five years. The site will become one of its “strategic pillars” as the team strives to get 71 approvals — either for new products or indications — by 2030, said Felix Gutsche, president and CEO of Boehringer Ingelheim China.