Crystalys Therapeutics does not yet have data from pivotal testing of its gout drug, but the company is already considering potentially bringing the drug to market, plans supported by a new $130 million in capital.
The new financing announced last Wednesday comes less than a year later Crystalys launched, revealing in-licensed gout drug candidate and $205 million investment Series A round. The San Diego-based company has since advanced that drug, dotinurad, to two Phase 3 studies that could support a request for FDA approval. The company also has an ongoing Phase 2 study.
Together, the three trials are designed to evaluate the study drug in a broad range of gout patients. Beyond clinical trial funding, the company said proceeds from its new capital infusion will allow the drug to move through regulatory milestones and market readiness activities.
In gout, high levels of uric acid in the blood cause crystals to form in joints and surrounding tissues. Patients suffer from inflammation and joint pain. Dotinurad is a small molecule inhibitor of URAT1, a transporter protein that leads to the reabsorption of uric acid by the kidneys.
“If you block the carrier [protein]you’re essentially urinating more uric acid,” CEO James Mackay explained last year in an interview about the company’s launch.
Crystalys’ rights to dotinurad for the United States and certain markets worldwide have been licensed by Urica Therapeutics. Urica had obtained these rights from Fuji Yakuhin, the Japanese company which discovered and developed dotinurad. Urica’s agreement with Crystalys gave it an equity stake in the biotechnology. The agreement also makes Urica eligible for a 3% royalty on sales of an approved product in authorized markets, according to regulatory filings. In addition to Japan, dotinurad is already commercially available in some Asian markets.
Gout affects millions of people, but it is an indication for which there have not been many drug innovations. The standard of care is the decades-old drug allopurinol, a small oral molecule designed to inhibit an enzyme essential for uric acid production. The problem is that many patients find this approach insufficient. Amgen’s Krystexxa, an engineered version of an enzyme that breaks down uric acid, is commercially available as a third-line treatment for gout. Second-line treatment was provided by a Takeda Pharmaceutical drug whose label included a black box warning regarding cardiovascular risks. Takeda discontinued the product earlier this year.
Inhibition of URAT1 is attracting further interest due to its potential to integrate into second-line gout treatment. Last December, the Swedish Orphan Biovitrum (Sobi) reached a $950 million deal to acquire Arthrosi Therapeutics. The biotech’s lead program, pozdeutinurad, formerly AR882, is a URAT1 inhibitor in late-stage development. In May, Sobi reported preliminary results of phase 3 demonstrating that both doses tested in the trials achieved the primary goal of reducing blood uric acid levels measured at six months compared to a placebo. Both studies continue for up to 12 months. The daily pill was well tolerated and the safety profile was consistent with previous studies. Sobi said detailed trial results would be presented at a scientific meeting in the fourth quarter of this year.
Frazier Life Sciences led the Series B financing of Crystalys. This company and many other participants in the round are crossover investors, companies that back both private and public companies. Funding from cross-investors is seen as a sign that a company is preparing for an IPO. When asked via email if Crystalys’ latest funding was a cross-round, Mackay responded: “We are keeping all of our funding options open at this time.”
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. Previous participants included Novo Holdings, SR One, Catalys Pacific, Perceptive Xontogeny Venture Funds, Lightstone Ventures, AN Venture Partners, funds managed by abrdn Inc., KB Investments, Pontifax, Longwood Fund, Alexandria Venture Investments, Wedbush Healthcare Partners and Prebys Ventures Fund.
Illustration: Kateryna Kon/Scientific Photo Library, via Getty Images
