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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Andy Plump, Takeda R&D chief (Jeff Rumans for Endpoints News)

What kind of PhI­Ib da­ta is worth $4B cash? Take­da’s Andy Plump has some thoughts on that

A few months back, when Takeda caused jaws to drop with its eye-watering $4 billion cash upfront for a mid-stage TYK2 drug from Nimbus, it had already taken a deep dive on the solid Phase IIb data Nimbus had assembled from its dose-ranging study in psoriasis.

Now, it’s rolling that data out, eager to demonstrate what inspired the global biopharma to go long in a neighboring, but new, disease arena for the pipeline. And the most avid students of the numbers will likely be at Bristol Myers Squibb, who will have a multi-year head start on pioneering the TYK2 space with Sotyktu (deucravacitinib) as Takeda makes its lunge for best-in-class status.

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No­vo Nordisk re­mains un­der UK scruti­ny as MHRA con­ducts its own re­view in 'in­cred­i­bly rare' case

The UK’s Medicines and Healthcare products Regulatory Agency is now reviewing Novo Nordisk’s marketing violation that resulted in its loss of UK trade group membership last week. Novo Nordisk was suspended on Thursday from the Association of the British Pharmaceutical Industry (ABPI) for two years after an investigation by its regulatory arm found the pharma broke its conduct rules.

MHRA said on Tuesday that its review of the Prescription Medicines Code of Practice Authority (PMCPA) investigation is standard practice. An MHRA spokesperson emphasized in an email to Endpoints News that the situation with Novo Nordisk is “incredibly rare” while also noting ABPI took “swift and proportionate action.”

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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FDA warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

FTC says patent bat­tle over Parkin­son's drug could have 'sig­nif­i­cant im­pli­ca­tion­s' for pa­tients

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

Mar­ket­ingRx roundup: What could a US Tik­Tok ban mean for phar­ma? Pfiz­er, Lil­ly lead phar­ma March Mad­ness ad­ver­tis­ers

Just as pharma marketers finally make moves into TikTok, the threat of a US ban on the social media channel is now looming. Already banned on federal employee phones by an initial Congressional act, more bills and maybe bans are on the way. With rare bipartisan agreement, lawmakers have introduced legislation that would give the US president the power to ban TikTok (although not mentioned by name) and other foreign-owned technology platforms that represent a security threat to the US.

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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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