Eight years af­ter Eli Lil­ly dumped it, an up­start biotech has nabbed teplizum­ab and filed a $56M IPO to back a PhI­II come­back

Eight years ago, a drug called teplizum­ab went down in flames af­ter it failed in a piv­otal di­a­betes study un­der­tak­en by Eli Lil­ly. But yes­ter­day a lit­tle start­up called Proven­tion Bio in-li­censed it from Macro­Gen­ics, the orig­i­nal de­vel­op­er, whipped around and to­day used it as their lead drug for a $56 mil­lion IPO fil­ing.

Teplizum­ab wasn’t com­plete­ly dead back in 2010 when Lil­ly dumped the whole thing. But it came aw­ful­ly close to that.

Jef­frey Blue­stone

Jef­frey Blue­stone, a high-pro­file sci­en­tist at UCSF who had worked long and hard on the drug, nev­er lost faith over 30 years of la­bor­ing on it. He con­tin­ued to pur­sue hu­man stud­ies for the drug, watch­ing large groups of di­a­bet­ics re­spond fa­vor­ably to the drug, but weary­ing at times at the task of try­ing to get some re­al mon­ey to back a come­back ef­fort. We ex­changed sev­er­al mes­sages about this drug a cou­ple of years ago as Blue­stone — now run­ning the Park­er In­sti­tute — pushed ahead.

The drug has shown val­ue in pre­serv­ing be­ta cells in the pan­creas, re­duc­ing the need for in­sulin among Type 1 pa­tients. And that’s where it will be test­ed in Phase III, with the study planned to launch next year.

“I am very ex­cit­ed about the “re­vival” of Teplizum­ab,” Blue­stone tells me in an email. “My col­leagues and I have al­ways felt that the drug could be im­pact­ful for re­cent on­set pa­tients with Type 1 Di­a­betes and more­over oth­er au­toim­mune dis­eases. The suc­cess in mul­ti­ple Phase II tri­als con­duct­ed by the aca­d­e­m­ic com­mu­ni­ty gives me great hope for the po­ten­tial for this drug to have an im­pact in a sig­nif­i­cant per­cent­age of these pa­tients. On­ward and up­ward.”

Ash­leigh Palmer

Proven­tion has been work­ing off of a $28 mil­lion A round it nabbed a year ago. That’s not much. And the plans call for on­ly a slice of it to be used to set up the Phase III. Proven­tion al­so has PRV-6527 (the CSF-1R in­hibitor JNJ-40346527, ob­tained from J&J) for Crohn’s dis­ease, and the ul­cer­a­tive col­i­tis can­di­date JNJ-42915925 (PRV-300), an an­ti-TLR3 an­ti­body.

The S-1 spells out all its li­cens­ing deals, in­clud­ing the $42.5 mil­lion it will owe Macro­Gen­ics based up­on “the achieve­ment of cer­tain de­vel­op­men­tal and ap­proval mile­stones for the first in­di­ca­tion, and an ad­di­tion­al $22.5 mil­lion up­on the achieve­ment of cer­tain reg­u­la­to­ry ap­provals for a sec­ond in­di­ca­tion. In ad­di­tion, we are ob­lig­at­ed to make con­tin­gent mile­stone pay­ments to Macro­Gen­ics to­tal­ing $225 mil­lion…” for sales mile­stones.

J&J al­so has a buy-back pro­vi­sion on PRV-6527, giv­ing them the right to claw it back for $50 mil­lion af­ter the next study.

The de­vel­op­ment of teplizum­ab will now lie pri­mar­i­ly in the hands of Ash­leigh Palmer, the CEO at Proven­tion who owns 12.2% of the com­pa­ny, and Fran­cis­co Leon, a J&J vet who once ran ear­ly-stage de­vel­op­ment work for J&J in im­munol­o­gy who al­so owns 12.2% of the com­pa­ny. Macro­Gen­ics now owns 10% of the com­pa­ny and J&J In­no­va­tion is a backer with 11.2%. MDB owns 10.8%.

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.