Sanofi sends a pair of gene ther­a­pies back to Ox­ford Bio­med­ica. Are they sal­vage­able?

The same day Ox­ford Bio­med­ica signed a 5-year deal to make As­traZeneca’s Covid-19 vac­cine, the UK gene ther­a­py com­pa­ny an­nounced they re­ceived word Sanofi was send­ing a cou­ple of decade-old ex­per­i­men­tal drugs back to them.

In 2009, Sanofi and Bio­med­ica slashed a co-de­vel­op­ment deal on TroVax, a can­cer vac­cine that had been one of the cen­ter­pieces of the then 14-year-old com­pa­ny. But the pair im­me­di­ate­ly re­placed it with a deal, 8 years be­fore the ap­proval of Spark Ther­a­peu­tics’ Lux­tur­na, to de­vel­op gene ther­a­pies for two eye-re­lat­ed dis­or­ders.

Now, with lit­tle to show for it but a pair of very ear­ly stud­ies, Sanofi is kick­ing the ther­a­pies back to Bio­med­ica. The French drug­mak­er had al­ready an­nounced last year, amid an in­ter­nal shake­up, that they were not in­ter­est­ed in de­vel­op­ing the drugs fur­ther, but what would hap­pen to the ther­a­pies hung in the air.

In some ways, it still does. Bio­med­ica wouldn’t com­mit to pro­gress­ing or re­tir­ing them. It wasn’t even clear when Bio­med­ica would re­gain the rights.

“The tim­ing of re­turn of these pro­grams and op­er­a­tional de­tails are yet to be de­ter­mined,” the biotech said in a state­ment. “How­ev­er, when the rights to the two pro­grams are re­turned, the group will un­der­take its own in­ter­nal eval­u­a­tion to de­ter­mine the po­ten­tial fu­ture for these pro­grams and de­cide whether to com­mit fur­ther re­sources to them.”

Bio­med­ica’s $100 mil­lion part­ner­ship with Sanofi’s rare blood dis­or­der sub­sidiary Biover­a­tiv re­mains in­tact.

The two gene ther­a­pies are SAR422459, for treat­ing a pro­gres­sive mac­u­lar dis­ease called Star­gardt dis­ease, and SAR421869, for treat­ing Ush­er’s Syn­drome, a dis­or­der that can cause both vi­sion and hear­ing loss. The Star­gardt ther­a­py be­gan a Phase I/II study in France in 2011, but the study was end­ed pre­ma­ture­ly last Au­gust af­ter Sanofi repri­or­i­tized its pipeline. Al­though the study was de­signed to mea­sure bi­o­log­i­cal mark­ers of ef­fi­ca­cy, Sanofi in­clud­ed no such da­ta when it post­ed re­sults on clin­i­cal­tri­als.gov ear­li­er this year.

The ther­a­py for Ush­er’s Syn­drome en­tered its Phase I/II study in 2012. That study was sus­pend­ed in 2017 while Sanofi re­viewed its pipeline and then al­so ter­mi­nat­ed last year.

If Bio­med­ica con­sid­ers the ther­a­pies to still have po­ten­tial, it’s like­ly they will try to out-li­cense them again. The com­pa­ny is built around part­ner­ships, li­cens­ing out a few in-house can­di­dates and help­ing man­u­fac­ture No­var­tis’s CAR-T ther­a­py Kym­ri­ah and, as of yes­ter­day, As­traZeneca’s Covid-19 vac­cine.  They are ex­plic­it­ly not a com­mer­cial-stage biotech. All of the pre­clin­i­cal can­di­dates on its web­site are list­ed as “to be spun out, or out-li­censed,” and they run no clin­i­cal tri­als.

So the best ques­tion may be not whether Bio­med­ica sees po­ten­tial, but if any­one else does.

So­cial: Shut­ter­stock

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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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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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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Paul Tesar (Convelo Therapeutics)

Io­n­is, lead­ing MS re­searcher throw an­ti­sense at a new type of brain cells

No matter how many molecules he threw at them, Paul Tesar couldn’t get the brain cells to survive. Or he got them to survive, but then — to everyone’s bafflement — they still couldn’t do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.

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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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.”

Covid-19 roundup: Vac­cines will need to beat place­bo by 50% to qual­i­fy for FDA OK; UK tri­al drops Kale­tra

The FDA will set the bar for approving a Covid-19 vaccine at 50% efficacy, the Wall Street Journal reported, meaning any successful candidate will have to reduce the risk of coronavirus disease by at least half compared to placebo.

That requirement is part of guidance that the agency is set to release later today, laying out detailed criteria for vaccine developers — some of whom are eyeing an OK by the end of the year, in line with expectations at Operation Warp Speed.

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