Q32 Bio grabs $60M to kick off hu­man stud­ies for next-gen com­ple­ment drugs — with some Covid-19 tweaks along the way

For a com­pa­ny that launched in the ear­ly months of the pan­dem­ic, Q32 Bio had its fair share of run-ins with the new nor­mals un­der Covid-19.

Mike Brox­son

The orig­i­nal plan, for in­stance, was to con­duct first-in-hu­man stud­ies of the IL-7 re­cep­tor an­ti­body it li­censed from Bris­tol My­ers Squibb in the Nether­lands. But they re­al­ized short­ly af­ter that while the coun­try was be­gin­ning to open up clin­i­cal tri­als, there were ad­di­tion­al re­stric­tions on drugs that tam­pered with im­muno­log­i­cal mech­a­nisms.

“We didn’t have any sense for the tim­ing of be­ing able to get in­to the clin­ic,” CEO Mike Brox­son told End­points News. “The team de­cid­ed in June with board sup­port to piv­ot and do our Phase I work in Aus­tralia.”

So the group led by CSO She­lia Vi­o­lette re­vised its fil­ing, fig­ured out man­u­fac­tur­ing and quick­ly got a green light to move their study to the South­ern Hemi­sphere. And on Tues­day, the first health vol­un­teer was dosed with ADX-914 — set­ting them up nice­ly to take the wraps off $60 mil­lion in fresh Se­ries B cash.

Next year will be piv­otal for the biotech, said Brox­son, a Take­da vet for whom quar­ters come as a nat­ur­al way of mea­sur­ing time. Not on­ly are in­ter­im read­outs for -914 slat­ed for Q4, but the first fu­sion pro­tein gen­er­at­ed on Q32’s plat­form is ex­pect­ed to en­ter Phase I around the same time.

She­lia Vi­o­lette

By grab­bing tis­sue-spe­cif­ic tar­gets on one end and fer­ry­ing a com­ple­ment reg­u­la­tor on the oth­er, the tech­nol­o­gy promis­es to gen­er­ate next-gen com­ple­ment drugs that can be de­liv­ered to or­gans where com­ple­ment frag­ments have been de­posit­ed — and get out of cir­cu­la­tion once that’s done.

“Our goal is to pre­serve sys­temic com­ple­ment sur­veil­lance, not in­hibit­ing in a longterm way,” Brox­son said.

While the mech­a­nism is dis­tinct from IL-7R, Brox­son added that the com­ple­ment pro­gram, ADX-097, ac­tu­al­ly shares cer­tain ap­pli­ca­tions with the lead drug such as lu­pus and arthri­tis.

With -914, though, Q32 is ini­tial­ly aim­ing to start with Sjö­gren’s dis­ease, pro­vid­ed the da­ta pan out. France’s Servi­er is part­ner­ing with OSE on a clin­i­cal drug that sim­i­lar­ly tar­gets IL-7R, so they will al­so be watch­ing the field close­ly.

As for the com­ple­ment drug pipeline — there is a slew of pre­clin­i­cal pro­grams lined up af­ter -097 — the fo­cus will be on the kid­ney and the skin.

The new fi­nanc­ing should last them two and a half years while they sort it all out, Brox­son said, and grow out the team to 30 staffers. Most of Q32 Bio’s team is still work­ing re­mote­ly while lab-based staffers set­tle in­to ro­ta­tion sched­ules.

Com­pared to op­er­a­tions, though, the fundrais­ing process struck him as fun­da­men­tal­ly dif­fer­ent.

The two years he spent as the chief of Goldfinch Bio was filled with trips to New York, San Fran­cis­co and Boston, he re­called. With­out trav­el and the need to get dressed up, his talks with in­vestors this time around were less for­mal and more ef­fi­cient.

“I think it will be a hy­brid — some as­pects of it will be re­mote, per­haps first calls, sec­ond calls,” Brox­son said when asked for pre­dic­tions about the fu­ture.

Or­bimed Ad­vi­sors and Acorn Bioven­tures led the Se­ries B. At­las Ven­ture, an ear­ly backer of Q32 Bio, re­turned along­side Os­age Uni­ver­si­ty Part­ners, At­las Ven­ture, Abing­worth, Sanofi Ven­tures, Uni­ver­si­ty of Col­orado and Chil­dren’s Hos­pi­tal Col­orado Cen­ter for In­no­va­tion.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,400+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,400+ biopharma pros reading Endpoints daily — and it's free.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,400+ biopharma pros reading Endpoints daily — and it's free.

Christophe Bourdon, Leo Pharma CEO

Leo Phar­ma looks 'be­yond the skin' in atopic der­mati­tis aware­ness cam­paign

As Leo Pharma aims to take on heavyweight champ Dupixent in atopic dermatitis, the company is launching “AD Days Around the World,” an awareness campaign documenting real patient stories across Europe.

The project, unveiled on Monday, spotlights four patients: Marjolaine, Laura, Julia and África from France, Italy, Germany and Spain, respectively, in short video clips on the challenges of living with AD, the most common form of eczema.

Ying Huang, Legend CEO

Lentivi­ral vec­tor ramp-up: J&J and Leg­end to in­vest $500M in New Jer­sey man­u­fac­tur­ing to sup­port Carvyk­ti

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.

Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.