Bill Gates and rich al­lies help fund an alt-biotech start­up path for the gene ex­pres­sion ex­perts at Ex­i­cure

Ex­i­cure CEO David Giljo­hann has been busy.

In the past cou­ple of months, he’s round­ed up two tranch­es of cash to­tal­ing $31.5 mil­lion from a grow­ing band of back­ers, with Bill Gates com­ing back to join new, wealthy in­vestor Fred Knoll in a syn­di­cate that has strayed far off the typ­i­cal start­up fund­ing path in biotech.

But that’s not his on­ly di­ver­gence from the stan­dard course. He’s ex­e­cut­ed a “clean” re­verse merg­er with a com­plete­ly emp­ty shell to go out on­to the OTC mar­ket — not Nas­daq. And, back in the norm, he’s fol­lowed up on his first round of Phase I da­ta with plans to line up a se­ries of Phase II stud­ies to put his plat­form tech to a cru­cial test.

David Giljo­hann

“We start­ed on a very dif­fer­ent path,” ac­knowl­edges the CEO, “with high­ly ed­u­cat­ed tech in­vestors who be­lieve in” spher­i­cal nu­cle­ic acid nan­otech­nol­o­gy.

Aside from Gates, the group in­cludes Er­ic Lefkof­sky, co-founder of Groupon, for­mer Mi­crosoft strat­e­gy chief Craig Mundie, the Rath­mann Fam­i­ly Foun­da­tion, re­tired Aon CEO Patrick Ryan, Il­lu­mi­na co-founder David Walt as well as Mark Tomp­kins and Kat­a­lyst Se­cu­ri­ties. They’re all back­ing a pub­lic com­pa­ny now, though shares aren’t trad­ing yet.

“We thought, giv­en the in­vestor base, this was a sim­pler way” to go pub­lic, Giljo­hann told me over the week­end about his OTC plans, af­ter de­tail­ing the lat­est batch of $11.2 mil­lion to come through from his syn­di­cate. “For us it was the best avail­able op­tion to take the com­pa­ny pub­lic.” The Nas­daq route was not avail­able to them.

It hasn’t hurt hav­ing Bill Gates in­vest in each round. This new batch of in­vestors in­cludes Luye Phar­ma, a big Chi­nese group that has been spread­ing its wings in the US and Knoll Cap­i­tal In­vest­ment, which man­ages a fam­i­ly for­tune that wasn’t hurt at all af­ter Pfiz­er bought out Medi­va­tion, where Knoll was one of the top 5 in­vestors to cash out. And let’s not for­get Ea­ger In­fo In­vest­ments and Pur­ple Arch Ven­tures, a group of North­west­ern alum, hap­py to help sup­port a biotech which emerged from the lab of North­west­ern chemist Chad Mirkin.

Ex­i­cure has now raised a to­tal of $75 mil­lion-plus, says the CEO. That mon­ey is be­ing used to pur­sue the de­vel­op­ment of new ther­a­pies that start with an ar­ti­fi­cial nanos­phere as a scaf­fold, as­sem­bling sin­gle- and dou­ble-strand­ed nu­cle­ic acids on the sur­face — cre­at­ing a “third form” of nu­cle­ic acids de­signed to eas­i­ly slip in­to cells with­out trig­ger­ing an im­mune re­sponse.

The SNAs can use an­ti­sense or RNAi path­ways to do their du­ty in reg­u­lat­ing gene ex­pres­sion. And the com­pa­ny is putting it on the line first with skin cells.

There are three clin­i­cal stage pro­grams.

  • AST-005 tar­gets TNF for mild to mod­er­ate pso­ri­a­sis. Pur­due came in and part­nered on it, with Phase I done and a Phase Ib wrap­ping soon.
  • XCUR 17 al­so tar­gets pso­ri­a­sis through an IL-17 re­cep­tor in the skin. Clin­i­cal tri­als be­gin in Q1 ’18.
  • Then there’s a TLR9 ag­o­nist — AST 008 — for sol­id tu­mors in com­bi­na­tion with a check­point. A tri­al is slat­ed to be­gin soon.

Go­ing pub­lic puts them on the SEC’s map, where they’ll have to file all their fi­nan­cials. An ini­tial 8K pro­vides some in­sight in­to their part­ner­ship with Pur­due, which signed a $780 mil­lion part­ner­ship on 005, where they are tak­ing the lead on the clin­i­cal work. Ac­cord­ing to the fil­ing, Pur­due got that deal rolling with a mod­est $10 mil­lion up front, with the rest due in mile­stones.

The next big stage for Ex­i­cure in­volves some de­tailed Phase II da­ta in some big dis­eases. Mak­ing a splash here won’t be easy. But it’s all been work­ing so far.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er will in­vest $1.2B+ in Irish man­u­fac­tur­ing site, adding 500 em­ploy­ees

Covid-19 trailblazer Pfizer has confirmed its commitment to a large expansion project on the Emerald Isle.

The New York-based company announced on Thursday that it will make a €1.2 billion ($1.26 billion) capital investment into its manufacturing site at Grange Castle in Dublin.

The expansion of the site marks Pfizer’s largest expansion investment in Ireland to date. The expansion includes the construction of a new facility on the premises as well as adding in more laboratory space and will ultimately double the capacity for “biological drug substance manufacturing” in the oncology and rare disease space as well as inflammation, immunology and internal medicines.

In­tel­lia and Iver­ic sell stocks to raise mon­ey, each net­ting $300M

Wednesday afternoon, Gene editing company Intellia and eye disease company Iveric Bio announced that they had each raised $300 million by selling off some of their stocks. The two biotechs are the latest to raise money via public stock offerings, an increasingly popular tactic used by public companies as the industry falls back from its pandemic boom.

Intellia’s raise comes a few weeks after it posted an update on its hereditary angioedema program that uses CRISPR/Cas9 to directly edit the gene that makes the protein responsible for the attacks that occur with the disease. In that interim cut, Intellia showed that patients dosed with its one-time therapy became attack free (at least thus far) after an observation period of 16 weeks, with the longest patient remaining attack free for 10 months.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.