While data from pivotal trials for its gout treatment is still pending, Crystalys Therapeutics is proactively preparing for potential commercialization, bolstered by a fresh $130 million capital infusion.
The new funding, announced last Wednesday, follows the company’s launch less than a year ago, which included a $205 million Series A round and the introduction of its in-licensed gout drug candidate. The San Diego-based firm has since progressed the candidate, dotinurad, into two Phase 3 studies aimed at supporting an FDA approval application. Additionally, the company is currently conducting a Phase 2 study.
These three concurrent trials are designed to evaluate the drug’s efficacy and safety across a diverse range of gout patients. Beyond funding clinical development, Crystalys stated that the new capital will support regulatory milestones and activities to prepare for commercial launch.
Gout is characterized by high levels of uric acid in the blood, which leads to the formation of crystals in joints and surrounding tissues, causing inflammation and intense pain. Dotinurad functions as a small molecule inhibitor of URAT1, a transporter protein responsible for the reabsorption of uric acid by the kidneys.
Crystalys holds the rights to dotinurad in the U.S. and several international markets through a license from Urica Therapeutics. Urica originally secured these rights from Fuji Yakuhin, the Japanese company that discovered and developed the compound. As part of the agreement, Urica holds an equity stake in Crystalys and is eligible for a 3% royalty on sales in licensed markets. Dotinurad is already available in certain Asian markets outside of Japan.
Despite affecting millions, the gout market has seen limited drug innovation. The current standard of care is allopurinol, a long-standing oral medication that inhibits a key enzyme in uric acid production, though many patients find it insufficient. Amgen’s Krystexxa serves as a third-line treatment, while the second-line market was previously served by a Takeda Pharmaceutical drug that was discontinued earlier this year following a black box warning for cardiovascular risks.
URAT1 inhibition is gaining momentum as a viable second-line treatment option. Last December, Swedish Orphan Biovitnum (Sobi) entered a $950 million deal to acquire Arthrosi Therapeutics, whose lead late-stage program, pozdeutinurad, is also a URAT1 inhibitor. In May, Sobi announced that preliminary Phase 3 results showed both tested doses successfully met the primary goal of reducing blood uric acid levels at six months compared to a placebo. These studies are ongoing through the 12-month mark. The daily pill showed a safety profile consistent with previous studies, and Sobi plans to present detailed results at a scientific meeting in the fourth quarter of this year.
The Series B financing led by Frazier Life Sciences included several crossover investors—firms that invest in both private and public companies—which often signals a company’s preparation for an IPO. When asked if this round was intended as a crossover, Mackay noted, “We are keeping all our financing options open for now.”
Other participants in the round include Wellington Management, HBM Healthcare Investments, Soleus Capital, Cormorant Asset Management, Trails Edge Capital Partners, Pivotal bioVenture Partners, and KCap Biotechnology Fund. Also participating were Novo Holdings, SR One, Catalys Pacific, Perceptive Xontogeny Venture Funds, Lightstone Ventures, AN Venture Partners, abrdn Inc., KB Investments, Pontifax, Longwood Fund, Alexandria Venture Investments, Wedbush Healthcare Partners, and Prebys Ventures Fund.
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