Alex­ion gam­bles on an ear­ly-stage drug for rare dis­eases, bag­ging Ap­ple Tree-backed Syn­tim­mune in $1.2B buy­out

Alex­ion Phar­ma­ceu­ti­cals is adding a clin­i­cal-stage as­set to the pipeline, buy­ing Syn­tim­mune for $400 mil­lion up­front with an­oth­er $800 mil­lion on the ta­ble for mile­stones.

The big prize here is the an­ti­body SYNT001, which is be­ing stud­ied for rare IgG-me­di­at­ed dis­eases. The lead pro­gram is a Phase Ib/IIa study that is ex­am­in­ing its ef­fi­ca­cy in warm au­toim­mune he­molyt­ic ane­mia (WAI­HA) and in pa­tients with pem­phi­gus vul­garis (PV) or pem­phi­gus fo­li­aceus. 

Jean-Paul Kress

Alex­ion’s new ex­ec­u­tive crew, brought in to do a com­plete makeover of the com­pa­ny, has some proof-of-con­cept da­ta it can re­ly on for now show­ing the drug’s abil­i­ty to re­duce IgG, a key bio­mark­er for these dis­eases. The drug works by in­ter­fer­ing with the in­ter­ac­tion of FcRn with Im­munoglob­u­lin G.

New York City-based Syn­tim­mune re­cruit­ed bio­phar­ma vet Jean-Paul Kress — out of Bio­gen and Sanofi — to run the com­pa­ny at the be­gin­ning of the year. That move fol­lowed the biotech’s $50 mil­lion raise in 2017; most of that mon­ey came from Ap­ple Tree Part­ners.

David de Graaf

“I think we have the abil­i­ty to go all the way,” for­mer CEO David de Graaf told me af­ter the raise. The new cash in­fu­sion was de­signed to get the com­pa­ny to a reg­is­tra­tion study, when they could dis­cuss the fu­ture. 

But they won’t need to dis­cuss any­thing now.

“Tar­get­ing FcRn holds great promise in trans­form­ing the treat­ment of IgG-me­di­at­ed dis­eases. SYNT001 has suc­cess­ful­ly demon­strat­ed proof of mech­a­nism – the abil­i­ty to rapid­ly low­er IgG lev­els – in ear­ly clin­i­cal stud­ies and has the po­ten­tial to treat a num­ber of rare IgG-me­di­at­ed dis­eases,” said Alex­ion CEO Lud­wig Hantson. “The ac­qui­si­tion of Syn­tim­mune rep­re­sents a crit­i­cal step in re­build­ing Alex­ion’s pipeline and fur­ther di­ver­si­fy­ing the com­pa­ny’s clin­i­cal-stage rare dis­ease port­fo­lio. It of­fers a strong strate­gic fit with Alex­ion’s ex­ist­ing rare dis­ease fran­chis­es and pro­vides the op­por­tu­ni­ty to trans­form pa­tient care in dis­eases like warm au­toim­mune he­molyt­ic ane­mia, where SYNT001 is the first, and cur­rent­ly the on­ly, an­ti-FcRn ther­a­py in clin­i­cal de­vel­op­ment.”


Im­age: Lud­wig Hantson. ALEX­ION

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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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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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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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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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.”

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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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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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.

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.