Jim Sullivan, Vanqua Bio CEO

Spurred on by Or­biMed part­ner's di­ag­no­sis, Van­qua Bio takes aim at neu­rode­gen­er­a­tive dis­ease with blue-chip back­ers

The FDA’s re­cent ap­proval of Bio­gen’s Aduhelm has pro­vid­ed a spark plug for neu­ro­science re­search across the bio­phar­ma spec­trum with in­vestors more like­ly than ever to jump on board. A Chica­go biotech now has its own clutch of blue-chip back­ers to pur­sue drugs across a range of in­di­ca­tions — and it all start­ed from a close col­league’s own di­ag­no­sis and search for a cure.

Or­biMed start­up Van­qua Bio closed an $85 mil­lion Se­ries B led by Omega Funds to put its lead can­di­date tar­get­ing the GCase en­zyme in the clin­ic, first in Gauch­er dis­ease and then for a rare form of Parkin­son’s, the com­pa­ny said Tues­day.

The com­pa­ny’s lead drug, a small mol­e­cule ac­ti­va­tor of GCase, tar­gets the mu­tant GBA1 gene path­way, which can cause Gauch­er and is cor­re­lat­ed with a form of Parkin­son’s known as GBA-PD that af­fects around 10% of all pa­tients and a form of Lewy body de­men­tia known as GBA-LBD.

Dim­itri Krainc

The biotech launched back in 2019 em­bold­ened by the work of Jonathan Sil­ver­stein, a for­mer man­ag­ing part­ner at Or­biMed who was di­ag­nosed with GBA-PD some five years ago and kick­start­ed the Sil­ver­stein Foun­da­tion to pur­sue cures for that dis­ease. Funds from the foun­da­tion helped pay for piv­otal ge­net­ics re­search in the lab of Dim­itri Krainc, the chair of neu­rol­o­gy at North­west­ern Uni­ver­si­ty’s Fein­berg School of Med­i­cine, which now un­der­girds Van­qua’s plat­form.

Van­qua re­lies both on ge­net­ics re­search out of Krainc’s lab and in vit­ro mod­el­ing tech­nol­o­gy that tests com­pounds in pa­tient-de­rived neu­rons, po­ten­tial­ly of­fer­ing an ear­ly sneak peek at the drug’s ef­fi­ca­cy for hu­man tests. The neu­rons are de­rived from in­duced pluripo­tent stem cells, which are in turn de­rived and con­vert­ed from pa­tients’ skin cells.

Kevin Hunt

It’s an ap­proach that looks to fill in the gap left by in­ad­e­quate an­i­mal mod­el­ing for neu­ro­science, where the chances of suc­cess in large in­di­ca­tions like Alzheimer’s dis­ease and Parkin­son’s have left a slew of fail­ures in its wake. Ac­cord­ing to Van­qua CSO Kevin Hunt, ro­dent mod­els sim­ply aren’t good pre­dic­tors of in vi­vo hu­man test­ing in neu­ro­science, and adding a sta­ble pre­clin­i­cal re­search tool de­rived from po­ten­tial pa­tients could even­tu­al­ly help crack the code for a range of dis­eases with huge un­met clin­i­cal needs.

CEO Jim Sul­li­van, a for­mer VP of re­search at Ab­b­Vie who helped shep­herd JAK in­hibitor Rin­voq and blood can­cer med­i­cine Ven­clex­ta in­to the clin­ic, and has worked as a ven­ture part­ner at Or­biMed since 2019, sees Van­qua’s plat­form as a best of all worlds strat­e­gy: com­bin­ing cut­ting-edge ge­net­ics re­search and pre­clin­i­cal tools to de­vel­op ef­fec­tive mol­e­cules with a next-gen­er­a­tion mod­el­ing ap­proach to up the chances of suc­cess.

“Our ap­proach builds on break­throughs in a cou­ple dif­fer­ent ar­eas: one, ge­net­ics and pin­point­ing caus­es of neu­rode­gen­er­a­tive dis­ease and two, much more en­hanced pre­clin­i­cal mod­els,” he said. “We’re re­al­ly try­ing to over­come the lim­i­ta­tions of neu­ro­science re­search for the last decade.”

The team ex­pects “mean­ing­ful” clin­i­cal da­ta from its lead drug to be pub­lished with­in the next two years, Sul­li­van said.

In GBA-PD, which may af­fect around 800,000 pa­tients world­wide and 100,000 in the US, Van­qua is look­ing to pair ge­net­ic mark­er re­searchers pi­o­neered in Krainc’s lab to part­ner with pa­tient cells and es­tab­lished bi­ol­o­gy around the GBA1 path­way to tack­le Gauch­er. But in Alzheimer’s or ALS — or even Parkin­son’s, in the case of GBA1 — the ge­net­ic cor­re­la­tion is less un­der­stood, leav­ing a big gap where a team like Van­qua could add to the sci­en­tif­ic heavy lift­ing and po­ten­tial­ly find a break­through along the way.

The Van­qua team ac­knowl­edges that crack­ing the code on Alzheimer’s, for in­stance, de­spite the re­cent ap­proval of Bio­gen’s Aduhelm, will re­quire a great deal more work in un­der­stand­ing the ge­net­ic path­ways and de­vel­op­ing ther­a­peu­tic modal­i­ties to tar­get them.

But that doesn’t mean they’re not ready to try. Af­ter the GCase lead drug, Van­qua has an undis­closed ther­a­py in the pre­clin­i­cal stage that lever­ages the com­ple­ment fac­tor path­way to po­ten­tial­ly mod­u­late a range of neu­rode­gen­er­a­tive dis­or­ders. The re­search is still rel­a­tive­ly new, but Sul­li­van said the pro­gram could even­tu­al­ly prove to be a game-chang­er in oth­er large in­di­ca­tions.

It’s still ear­ly days, but Van­qua has all the trap­pings of an am­bi­tious plat­form com­pa­ny and enough sci­en­tif­ic ca­chet to pull in a no­table in­vestor syn­di­cate. In ad­di­tion to round leader Omega, the syn­di­cate is joined by Se­ries A in­vestor Or­biMed and new in­vestors Sur­vey­or Cap­i­tal, Avoro Ven­tures, Pon­tif­ax, Cas­din Cap­i­tal Man­age­ment, Eli Lil­ly, Lo­gos Cap­i­tal and Os­age Uni­ver­si­ty Part­ners.

The biotech ex­pects to use the pro­ceeds to ad­vance its two lead pro­grams and add to its team of 15, po­ten­tial­ly dou­bling in size in the com­ing years. Van­qua has head­quar­ters in Chica­go, near Krainc’s lab from which the team is shut­tling re­searchers over to join the clin­i­cal ef­fort. Sul­li­van high­light­ed the grow­ing Chica­go biotech hub as a ma­jor draw for Van­qua and a source of un­tapped po­ten­tial for the neu­ro­science field writ large.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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