Swedish Orphan Biovitrum Ltd.

A gout drug that Swedish Orphan Biovitrim picked up in a $100 million deal has achieved the main goal in two Phase 3 studies, showing statistically significant reductions blood levels of the protein associated with the inflammatory condition. Based on those data, the company, which is more commonly referred to as Sobi, said it now plans to seek FDA approval. Gout is a form of arthritis that stems from excess uric acid that forms deposits in the joints. Patients who have gout experience chronic inflammation and joint pain. Sobi licensed rights to its gout drug candidate, SEL-212, from Selecta Biosciences. The drug employs pegadricase, a yeast-derived enzyme, to break down excess urate. However, this enzyme can spark an immune response that limits the administration of the therapy to a single dose that is effective for less than a month. Watertown, Masssachusetts-based Selecta overcomes the dosing limitation by inducing immune tolerance. The company uses a technology called ImmTOR, which employs biodegradable nanoparticles that encapsulate an immunosuppressant. SEL-212, dosed as an infusion every 28 days, is comprised of ImmTOR and pegadricase. The placebo-controlled Phase 3 studies enrolled patients whose chronic gout had not responded to standard urate-lowering drugs. The trials evaluated two different doses of SEL-212. According to preliminary results reported Tuesday by Sobi and Selecta, 56% of patients in the U.S. Phase 3 test met the goal of achieving at least an 80% reduction in serum urate in month six. By comparison, 4% of patients who received the placebo achieved that goal. In the global Phase 3 study, 47% of patients achieved the serum urate reduction goal compared to 12% of patients in the placebo arm. No data were disclosed for the secondary trial goals, which included assessing tenderness and swelling of joints as well as assessing changes in urate deposits in the joints. But the companies said both doses of the study drug were well tolerated in both Phase 3 studies. Adverse events included inflammation and redness in the mouth, which was reported in 3.4% of participants in the low dose group and 9.2% of those in the high dose group. Treatment-related adverse events were reported in six patients, including two that had cases of anaphylaxis and one gout flare in both the high and low dose groups. All infusion reactions occurred within the first three infusions and resolved with a stop to the infusion and symptomatic treatment. The results of the study appear to be durable. Of those patients who responded to the study drug and continued to receive treatment in a six-month extension study, the companies said 75% of them continued to be successfully treated through one year with no infusion reactions or safety issues. The companies said more detailed results will be presented at an upcoming medical meeting. An FDA submission is expected in the first half of next year. Patients whose gout does not respond to standard treatments can be treated with Krystexxa, a Horizon Therapeutics drug that is comprised of an engineered version of the enzyme uricase. The drug, which the company has projected will become a blockbuster seller, was one of the products that enticed Amgen to agree to pay $28 billion to buy Horizon. Krystexxa must be administered every two weeks. If SEL-212 wins regulatory approval, it would give patients a more convenient dosing option. “SEL-212’s favorable safety profile, coupled with the convenient once monthly treatment regimen, will be welcomed by patients with this challenging form of gout and the physicians who treat them,” Herbert Baraf, clinical professor of medicine at the George Washington University School of Medicine and Health Sciences and the principal investigator of the Phase 3 program said in a prepared statement. In 2020, Sobi paid Selecta $75 million up front and made a $25 million equity investment in the company to license SEL-212. Though Sobi took on responsibility for further development of the drug candidate for all markets except China, Selecta conducted the Phase 3 study, which was funded by Sobi. Selecta could receive up to $630 million in milestone payments, plus royalties from sales of the drug if it reaches the market.