Shoot­ing for a new stan­dard on os­teoarthri­tis, Mer­ck KGaA out­lines promis­ing da­ta for a po­ten­tial game-chang­er

Mer­ck KGaA’s phar­ma group is tak­ing one more step in a long jour­ney to­ward re­hab­bing its rep in the R&D field this week­end. Com­pa­ny ex­ecs turned up at the ACR sci­en­tif­ic con­fer­ence in San Diego to roll out a promis­ing look at some key Phase II os­teoarthri­tis da­ta — which comes with a crit­i­cal caveat.

The drug is sprifer­min, which Mer­ck Serono has had in its pipeline for the past 13 years af­ter bag­ging it from Zy­mo­Ge­net­ics, now a part of Bris­tol-My­ers Squibb. In the first two-year ef­fi­ca­cy as­sess­ment of the five-year study, re­searchers are now proud­ly point­ing to a slight in­crease of car­ti­lage in the knee, as op­posed to the re­morse­less de­cline that leads to ex­pen­sive knee re­place­ment surgery.

Lu­ciano Ros­set­ti

By Mer­ck re­search chief Lu­ciano Ros­set­ti’s reck­on­ing, this is the first time a drug has ac­tu­al­ly trig­gered a re­ver­sal of this dis­ease — rather than just man­ag­ing the symp­toms of the dis­ease — leav­ing the Ger­man Mer­ck with a po­ten­tial­ly ground­break­ing drug in a field that ac­counts for 5% of the world’s pop­u­la­tion, with 237 mil­lion peo­ple whose lives have been crimped by this dis­ease.

“The most strik­ing re­sponse is the dis­ease re­sponse,” Ros­set­ti tells me. And what’s par­tic­u­lar­ly ex­cit­ing, he added, is that the drug — re­com­bi­nant hu­man FGF-18 pro­tein — is not just block­ing the de­cline of car­ti­lage, but sig­nif­i­cant­ly build­ing nor­mal car­ti­lage in a 2-year time frame.

At the high­est dosage, re­searchers tracked a 0.05 mm in­crease in car­ti­lage thick­ness, with a step down in dose to a 0.04 mm in­crease. Con­sid­er­ing that a 0.04 mm loss puts pa­tients at a great­ly high­er risk of knee re­place­ment, he says, they’re look­ing at a ma­jor treat­ment shift.

If the da­ta are borne out in Phase III, Mer­ck KGaA be­lieves it can line up an­oth­er drug be­hind its big PD-1 drug avelum­ab, part­nered with Pfiz­er in the biggest up­front ever record­ed in biotech. Mer­ck KGaA al­so re­cent­ly won a Eu­ro­pean ap­proval for the MS drug cladrib­ine, ini­tial­ly aban­doned in 2010 af­ter it was spurned by reg­u­la­tors on both sides of the At­lantic. That marked a low point for Mer­ck KGaA, which went for more than a decade with­out a sin­gle ma­jor win on the R&D side of the busi­ness.

Ros­set­ti and the rest of the team want to put the bad old days be­hind the com­pa­ny for good, and sprifer­min could help.

But there’s a big catch. The drug failed to reg­is­ter any kind of no­tice­able im­prove­ment over place­bo on pa­tients’ symp­toms in­volv­ing pain or func­tion, an es­sen­tial part of the sec­ondary end­point pro­file.

Just get­ting an in­jec­tion of ei­ther place­bo or drug in the knee had a big im­pact, says Ros­set­ti, mak­ing it im­pos­si­ble to tease out the drug’s ef­fect. They now have to see if reg­u­la­tors are open to us­ing a mark­er on car­ti­lage thick­ness rather than an ef­fect on symp­toms as a Phase III pri­ma­ry end­point.

There is no set plan for Phase III, he adds, with up­com­ing reg­u­la­to­ry ex­changes need­ed to sort out tri­al de­sign and length. You can be sure it won’t be easy or quick, though. Glob­al­Da­ta re­cent­ly es­ti­mat­ed that the drug could hit the mar­ket in 2021, with fair­ly lim­it­ed near-term com­mer­cial prospects in a mar­ket dom­i­nat­ed by cheap gener­ics.

Still, it’s for­ward progress for Mer­ck KGaA in the clin­ic. And that is big.

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.