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.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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CEO Lex Rovner (64x Bio)

A George Church spin­out fight­ing the vi­ral vec­tor bot­tle­neck in cell and gene ther­a­py lands $55M

A synthetic biology company spun out of George Church’s lab is set to tackle the gene therapy manufacturing bottleneck, and it just landed $55 million in a Series A financing round to do so.

64x Bio comes out of the Harvard Department of Genetics. CEO Lex Rovner and her team — which right now, sits around 10 people — are looking to tackle a key hurdle for major companies: manufacturing cell and gene therapies.