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.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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