As­traZeneca spin­off scores maid­en FDA ap­proval, where it will field Alex­ion com­pe­ti­tion and an im­mi­nent Roche ri­val

About a year af­ter the first-ever FDA ap­proval for pa­tients with neu­romyelitis op­ti­ca spec­trum dis­or­der (NMOSD), the agency has ap­proved a sec­ond ther­a­py for the rare au­toim­mune con­di­tion.

On Thurs­day, As­traZeneca spin­off Viela Bio scored its maid­en ap­proval for its lead drug, Up­liz­na, to treat NMOSD pa­tients. It will com­pete with Alex­ion’s en­trenched key­stone ther­a­py Soliris — a mon­o­clon­al an­ti­body that tar­gets com­ple­ment pro­tein C5 — which se­cured its NMOSD ap­proval last June.

The dis­or­der is of­ten con­fused with mul­ti­ple scle­ro­sis as the im­mune sys­tem at­tacks its host, dam­ag­ing healthy tis­sue in the spine and eyes. Al­though the ma­jor­i­ty of NMOSD pa­tients are well-man­aged on Rit­ux­an, there are still about a third of pa­tients who re­lapse and are at risk for se­vere dis­abil­i­ties and even death, if left un­treat­ed. NMOSD can be as­so­ci­at­ed with an­ti­bod­ies that bind to a pro­tein called aqua­por­in-4 (AQP4) — bind­ing of these an­ti­bod­ies ap­pears to ac­ti­vate oth­er com­po­nents of the im­mune sys­tem, caus­ing in­flam­ma­tion and dam­age to the cen­tral ner­vous sys­tem.

Up­liz­na, known chem­i­cal­ly as inebi­lizum­ab, is an in­tra­venous an­ti-CD19 drug. Its ap­proval was based on a 230-pa­tient study, in which 213 pa­tients had an­ti­bod­ies against AQP4. The risk of re­lapse in the 161 an­ti-AQP4 an­ti­body-pos­i­tive pa­tients who were treat­ed with Up­liz­na was di­min­ished by 77% ver­sus place­bo. In ad­di­tion, 89% of pa­tients in the AQP4 an­ti­body-pos­i­tive group re­mained re­lapse-free dur­ing the six-month pe­ri­od post-treat­ment com­pared to 58% of place­bo sub­jects.

There was no ev­i­dence of a ben­e­fit in pa­tients who were an­ti-AQP4 an­ti­body neg­a­tive.

The ap­proval is large­ly in line with base case ex­pec­ta­tions — the drug has got no black box warn­ing and the ap­proval is for AQP4+ pa­tients on­ly, not­ed Stifel’s Derek Archi­la, an­tic­i­pat­ing the drug will gen­er­ate about half a bil­lion dol­lars in US sales at peak.

Viela’s shares $VIE jumped more than 6.5% to $54.82 in Fri­day pre­mar­ket trad­ing.

Soliris’ ap­proval, which came with a black box warn­ing, was based on a study that test­ed the drug in an­ti-AQP4 an­ti­body-pos­i­tive pa­tients. Da­ta showed 98% of Soliris-treat­ed pa­tients were re­lapse-free ver­sus 63% on place­bo. SVB Leerink’s Ge­of­frey Porges has fore­cast Soliris sales in NMOSD reach­ing about $700 mil­lion by 2024.

With­in the NMOSD realm is an­oth­er ri­val wait­ing in the wings: Roche and Chugai’s satral­izum­ab.  The drug in­hibits IL-6 sig­nal­ing, which is be­lieved to play a key role in the in­flam­ma­tion that oc­curs in peo­ple with NMOSD.

Da­ta from a late-stage study in the sub­group of pa­tients that are AQP4-IgG seropos­i­tive — who tend to ex­pe­ri­ence a more se­vere dis­ease course — saw a 74% re­duc­tion in the risk of re­laps­es in the satral­izum­ab group. About 82.9% were re­lapse-free at 48 weeks and 76.5% re­lapse-free at 96 weeks when treat­ed with satral­izum­ab, com­pared to 55.4% and 41.1% with place­bo, re­spec­tive­ly. A mar­ket­ing ap­pli­ca­tion for the ther­a­py is cur­rent­ly un­der FDA re­view.

Viela ex­ec­u­tives have un­der­scored that the fact Up­liz­na does not car­ry a black box warn­ing should help it achieve first line sta­tus, and that the la­bel re­flects the drug’s abil­i­ty to re­duce hos­pi­tal­iza­tion rates could prove to be a dif­fer­en­tia­tor against oth­er agents, no­tably satral­izum­ab, Stifel’s Archi­la not­ed.

“While we agree with the lat­ter, we do be­lieve the ex­ten­sive ex­pe­ri­ence physi­cians have with rit­ux­imab in NMOSD and rit­ux­imab’s large safe­ty data­base from its many years on the mar­ket will like­ly be a chal­lenge for first-line us­age of Up­liz­na,” Archi­la said.

While Viela has not dis­closed Up­liz­na’s list price so far, they have gen­er­al­ly laid out a range of $200,000-$280,000/year as fea­si­ble. That range is still far be­low Soliris, which at a stag­ger­ing cost of more than $500,000 per year is one of the most ex­pen­sive drugs in the world.

“Our es­ti­mate for the an­nu­al cost of treat­ment for NMOSD with biosim­i­lar or brand­ed rit­ux­imab is around $33,000-$37,000 and is con­sid­er­ably less than Viela’s pro­posed pric­ing for Up­liz­na, which could cre­ate an­oth­er chal­lenge for adop­tion,” Archi­la said.

Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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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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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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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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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.”

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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On a roll, Mer­ck blazes through a new seg­ment of the bio­mark­er trail

Merck has notched an approval for using Keytruda to treat a biomarker-based subset of first-line colorectal cancer patients with unresectable or metastatic tumors, as the pharma giant continues to find new niches for its blockbuster PD-1 star.

The OK is significant in a number of ways. Not only does it build on an accelerated approval for all tumors characterized as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR); it also marks the first single treatment for colorectal cancer that doesn’t contain chemotherapy.