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%.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Uğur Şahin, BioNTech CEO (ddp images/Sipa USA/Sipa via AP Images)

BioN­Tech bets on dif­fi­cult STING field via small mol­e­cule pact with a Pol­ish biotech

BioNTech is beefing up its relatively thin small molecule pipeline by adding weight to a clinically difficult corner of oncology R&D: STING agonists. To do so, BioNTech is teaming up with a 15-year-old Polish biotech and doling out €40 million, about $41.5 million, to start.

The deal is broken into two parts: First, BioNTech obtains an exclusive global license to develop and market Ryvu Therapeutics’ STING agonist portfolio as small molecules, whether alone or in combination with other agents.

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