In a lupus treatment landscape that’s “poised for evolution” in the coming years, Ventus Therapeutics is looking to make a mark with an early-stage prospect that features desirable characteristics.
Thursday, the company reported that its first-in-class cyclic GMP-AMP synthase (cGAS) inhibitor succeeded in a phase 1 study, teeing up plans for the company to commence a phase 2 study in patients with systemic lupus erythematosus (SLE) next year.
The phase 1 study assessed pharmacokinetics (PK), target engagement, safety, and tolerability of VENT-03 in various doses among 72 healthy adults, the biotech said in an Oct. 31 release.
A Fierce 15 winner last year, Ventus said its drug, VENT-03, “was safe and well-tolerated at doses far exceeding those planned for use in Phase 2 trials.” The drug’s "favorable PK profile” supports daily oral dosing, Ventus said.
“Available treatments for lupus today include injectable medicines for the type I interferon or BAFF pathways that address only limited aspects of patients’ symptoms and disease course,” Ventus’ clinical development head Xavier Valencia, M.D., said in a statement. “A once-daily oral cGAS inhibitor has the potential to modulate both pathways validated by biologics, impact multiple facets of SLE, and provide superior efficacy compared to existing treatments and therapies in development.”
Ventus was founded in 2019 and has secured fundraising rounds of $60 million, $100 million and $140 million over the years. Besides those raises, the company picked up $70 million in upfront cash through its licensing of a NLRP3 inhibitor to Novo Nordisk.
Using its ReSOLVE platform, Ventus models proteins “at an unparalleled resolution” to develop small-molecule drugs in the fields of immunology, inflammation and neurology, according to its press release.
Besides VENT-03, the company’s NLRP3 inhibitors VENT-02 and VENT-01 have completed phase 1 studies. The VENT-01 program has been licensed out to Novo Nordisk for further development in diseases such as metabolic dysfunction-associated steatohepatitis (MASH) and chronic kidney disease.
In lupus, meanwhile, many drugmakers large and small are looking to make a mark. Just this week, GSK paid $300 million in upfront cash to Chimagen Biosciences for rights to the China-based drugmaker’s clinical-stage T-cell engager CMG1A46. GSK already markets the SLE market leader Benlysta, but other companies are vying to challenge its lead—both with approved candidates and pipeline meds.
AstraZeneca’s Saphnelo won its FDA nod in 2021 in SLE, while Biogen has a pair of late-stage SLE hopefuls in dapirolizumab pegol and litifilimab.
Based on a survey of 101 rheumatologists in the U.S., analysts with Spherix Global Insights recently projected that the lupus landscape was “poised for evolution" in the years to come. Besides the pipeline meds, the rheumatologists expressed interest in potential SLE label expansions for AbbVie’s marketed Rinvoq and Bristol Myers Squibb’s Sotyktu.