Ben Auspitz, F-Prime

From $30M launch round to $400M buy­out deal — with $250M up­front — in 10 short months

Stephen Farr Zo­genix

F-Prime’s Ben Aus­pitz has turned a mod­est in­vest­ment in his new­ly launched biotech Modis Ther­a­peu­tics in­to a quick score on the M&A side. And the deal comes with some lessons on the speed rare dis­ease biotechs can move these days, as well as the val­ue they can cre­ate in short or­der.

Ra­zor fo­cused on rare mi­to­chon­dr­i­al DNA de­ple­tion dis­or­ders, Oak­land, CA-based Zo­genix $ZGNX has come up with a $400 mil­lion deal to bag the com­pa­ny and its fledg­ling pipeline — just 10 months af­ter Aus­pitz put to­geth­er a $30 mil­lion A round. The buy­out terms in­clude $250 mil­lion in cash and stock along with $150 mil­lion for reg­u­la­to­ry ap­provals in the US and Eu­rope.

The deal gives Zo­genix chief Stephen Farr the chance to get this one right from the start. The com­pa­ny was em­bar­rassed by a refuse-to-file no­tice from the FDA last April for their drug to treat Dravet syn­drome. Reg­u­la­tors called out the com­pa­ny for slop­py and in­ad­e­quate work.

Mi­chio Hi­ra­no Co­lum­bia

Aus­pitz gam­bled on re­search by Mi­chio Hi­ra­no of Co­lum­bia Uni­ver­si­ty Irv­ing Med­ical Cen­ter and Ra­mon Martí at Barcelona’s Vall d’He­bron Re­search In­sti­tute. They came up with MT1621, the lead drug for thymi­dine ki­nase 2 de­fi­cien­cy (TK2d). 

The VC is shar­ing the wealth with the rest of the syn­di­cate, which in­cludes Or­biMed, Ac­eras Life Sci­ences and Os­age Uni­ver­si­ty Part­ners.

While they were dis­cussing an­i­mal da­ta for the launch round, the drug has since com­plet­ed a Phase II piv­otal pro­gram in 38 pa­tients. Modis re­ports a strong p val­ue (0.004) in sur­vival, com­par­ing their da­ta to a his­tor­i­cal con­trol group. And Modis is al­so boast­ing about hit­ting key goals for “func­tion­al abil­i­ties, in some cas­es re-ac­quir­ing pre­vi­ous­ly lost mo­tor mile­stones.”

Ra­mon Martí VHIR

In re­cent months the biotech al­so picked up break­through sta­tus at the FDA and PRIME sta­tus at the EMA, en­cour­ag­ing plans for a quick di­a­logue with the FDA.

The ther­a­py is not free of safe­ty pit­falls, though.

Modis ex­ecs say that 2 adult-on­set pa­tients “stopped treat­ment due to asymp­to­matic in­creas­es in amino­trans­feras­es, with­out in­creas­es in biliru­bin, which re­solved up­on dis­con­tin­u­a­tion of treat­ment.”

It’s all in Zo­genix’s hands now.

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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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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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.