About Aurinia Pharmaceuticals

Aurinia Pharmaceuticals Inc. operates as a fully integrated biopharmaceutical company. The company focuses on delivering therapies to people living with autoimmune diseases with high unmet medical needs. In January 2021, the company introduced LUPKYNIS (voclosporin), the first FDA-approved oral therapy for the treatment of adult patients with active LN. The company continues to conduct clinical and regulatory activities to support the LUPKYNIS development program. The company contracted with Otsuka Pharmaceutical Co., Ltd. (Otsuka) as a collaboration partner for the development and commercialization of LUPKYNIS in the European Union (EU), Japan, as well as the United Kingdom, Russia, Switzerland, Norway, Belarus, Iceland, Liechtenstein and Ukraine (collectively, the Otsuka Territories). LUPKYNIS is an orally administered CNI immunosuppressant that has been demonstrated to improve near and long-term outcomes in LN when used in combination with mycophenolate mofetil (MMF) and steroids. By inhibiting calcineurin, LUPKYNIS reduces cytokine activation and blocks interleukin IL-2 expression and T-cell mediated immune responses. LUPKYNIS also potentially stabilizes podocytes, which can protect against proteinuria. Voclosporin, the active ingredient in LUPKYNIS, is made by a modification of a single amino acid of the cyclosporine molecule. The mechanism of action of LUPKYNIS has been validated with certain earlier generation CNIs for the prevention of rejection in patients undergoing solid organ transplants and in several autoimmune indications, including uveitis, keratoconjunctivitis sicca, psoriasis, rheumatoid arthritis, and for LN in Japan. LUPKYNIS possesses pharmacologic properties with the potential to demonstrate best-in-class differentiation. On September 15, 2022, the EC granted marketing authorization of LUPKYNIS. The centralized marketing authorization is valid in all EU member states, as well as in Iceland, Liechtenstein, Norway and Northern Ireland. On November 29, 2022 the Medicines and Healthcare products Regulatory Agency (MHRA) had granted marketing authorization of LUPKYNIS in Great Britain. On April 24, 2023, LUPKYNIS received regulatory approval in Switzerland. On November 10, 2023, Otsuka filed a new drug application (NDA) for voclosporin for the treatment of LN with the Japanese Ministry of Health, Labour, and Welfare for the manufacture and sale in Japan of voclosporin. Strategy The company’s business strategy is to optimize the clinical and commercial value of LUPKYNIS and evolve as a commercial biopharmaceutical company with a global product portfolio focused on autoimmune, kidney and rare diseases with high unmet medical needs. The company has developed a strategic plan to execute on its commercialization of LUPKYNIS as a treatment of adult patients with active LN. The key tactics to achieve the company’s corporate strategy are to focuses on educating physicians, patients and payers to increase their awareness of the risks and impacts of LN as a disease (including that elevated proteineuria levels can have significant impact on kidneys and the disease needs to be diagnosed and addressed quickly), and the benefits of LUPKYNIS (as demonstrated in its clinical trials) as a treatment option; engage Otsuka as a collaboration partner for the development and commercialization of LUPKYNIS in the Otsuka Territories; conduct post-marketing studies to satisfy regulatory requirements and better understand LUPKYNIS' characteristics in real-world usage; ensuring adequate supply of LUPKYNIS by entering into strategic long term supply agreements with its key suppliers; and evaluate external assets with the potential to be synergistic and complementary to its clinical, regulatory and therapeutic areas of expertise. Developments Regulatory/Commercial On April 11, 2023, the company announced that the United States Patent and Trademark Office (USPTO) had issued a new and refined method of use patent titled IMPROVED PROTOCOL FOR TREATMENT OF LUPUS NEPHRITIS. The company’s newly issued U.S. Patent (No. 11,622,991) reflects the unique and proprietary dosing regimen of its marketed product, LUPKYNIS. Specifically, this patent further refines the method of using LUPKYNIS in combination with MMF and corticosteroids using eGFR as a method of pharmacodynamically dosing the product in patients with LN. The newly issued patent provides coverage that supplements Aurinia’s existing U.S. Patent No. 10,286,036, which is listed in the Orange Book and claims an FDA-approved method of using LUPKYNIS. The claims in this additional patent add further specificity on dosing consistent with the FDA approved product label. On December 20, 2023, the company announced the submission of its Investigational New Drug application (IND) to the FDA for AUR200, a potential next generation therapy for B-cell mediated autoimmune diseases. The IND was made effective on January 18, 2024. On February 15, 2024, the company announced that it is ceasing future development of AUR200 and AUR300 research and development programs and prioritizing resource allocation. Study Updates On April 5, 2023, the company announced promising results from the AURORA Renal Biopsy Sub-Study. The addition of LUPKYNIS on top of the then current standard of care MMF and low-dose steroids in its Phase 3 AURORA 1 and AURORA 2 studies led to significantly earlier and greater reductions in proteinuria while maintaining stable renal function, as evidenced by a stable eGFR slope over time. To further characterize the long-term impact of LUPKYNIS on the kidney at the histologic level, repeat biopsies were collected from selected patients in both treatment arms (the active control arm with patients treated with only MMF and steroids, and the study arm of voclosporin in combination with MMF and steroids). FDA Approval and Commercial Launch of LUPKYNIS On January 22, 2021, the FDA approved LUPKYNIS in combination with a background immunosuppressive therapy regimen to treat adult patients with active LN. As a condition of approval, the company was required to conduct four Post Marketing Studies (PMRs) and one Post Marketing Commitment (PMC). The company submitted the final study report for AURORA-2 continuation study (PMR) in March 2022, a drug-drug interaction study (PMC) was submitted in October 2022 and a milk only lactation study was submitted in June 2023. The remaining PMRs are all progressing within the expected timelines. The company expects to complete two pediatric studies due in 2025 and 2031. Completion of AURORA 2 Continuation Study On December 9, 2021, the company announced positive topline results from the AURORA 2 two-year continuation study evaluating the long-term safety and tolerability of LUPKYNIS. AURORA 2 (NCT03597464) was a Phase 3 randomized, double-blind, placebo-controlled clinical trial to assess the long-term safety and tolerability of voclosporin, in addition to MMF and steroids. Patients who completed 12 months of treatment in the Phase 3 AURORA 1 study were eligible to enroll in the AURORA 2 continuation study with the same randomized treatment of voclosporin at 23.7 mg twice daily or placebo, in combination with MMF at 1 g twice daily with low-dose oral steroids, for up to an additional 24 months. A total of 216 LN patients out of 357 who were enrolled in the AURORA 1 study continued into AURORA 2, with 116 patients in the voclosporin group and 100 patients in the control group. 90 and 78 patients, respectively, received 36 months of total treatment at the completion of the study. Compared to the active control group, the voclosporin-treated group showed an increase from baseline eGFR at the end of the studies of +2.7 mL/min. The study demonstrated a favorable risk/benefit profile over a three-year period, with safety comparable to AURORA 1, and sustained efficacy. Research and Development Expenses The company’s research and development expenses included $49.6 million for the year ended December 31, 2023. Intellectual Property The company has been granted patent portfolio covering voclosporin, including granted United States patents, for composition of matter, methods of use, formulations and synthesis, and the rights to certain corresponding Canadian, South African and Israeli patents are owned by Paladin Labs Inc. Patent protection for patents related to the composition of matter of voclosporin are expected to be extended in the United States and certain other major markets, including Europe and Japan, until at least October 2027 under the Hatch-Waxman Act in the United States and comparable patent extension laws in other countries (including the Supplementary Protection Certificate program in Europe). The company has applied for a patent term extension, and are awaiting confirmation from the USPTO. As the patent term extension was not granted prior to the expiry of the patent term for its composition of matter patent for voclosporin, it applied for, and have received, an interim patent term extension until October 17, 2024. If the patent term extension is not granted prior to the expiration of the interim patent term extension that was granted, it intends to file future interim patent term extensions until the USPTO completes its review of the patent term extension application. In addition to patent rights, the company has received new chemical entity exclusivity for LUPKYNIS in the United States, which provides for exclusivity until January 22, 2026, and new chemical entity equivalent exclusivity for voclosporin in certain European countries, which provides exclusivity for up to ten years in Europe. In May 2019, the company was granted U.S. Patent No. 10,286,036 (the '036 patent) with a term extending to December 2037, with claims directed at its LUPKYNIS dosing protocol for LN used in its clinical trials. This dosing protocol is reflected on the prescribing information approved by the FDA for LUPKYNIS. Notably, the allowed claims cover a method of modifying the dose of LUPKYNIS in patients with LN based on patient specific pharmacodynamic parameters. The company has also filed for protection of this subject matter under the Patent Cooperation Treaty (PCT) and are applying for similar protection in the member countries thereof. In April 2023, the USPTO issued a new and refined method of use patent. The company’s newly issued U.S. Patent (No. 11,622,991) reflects the unique and proprietary osing regimen of LUPKYNIS. Specifically, this patent further refines the method of using LUPKYNIS in combination with MMF and corticosteroids using eGFR as a method of pharmacodynamically dosing the product in patients with LN. The newly issued patent provides coverage that supplements the '036 patent. The claims in this additional patent add further specificity on dosing consistent with the FDA approved product label. This patent has the potential to provide an additional layer of patent protection for LUPKYNIS up to 2037. Manufacturing of Drug Substance Voclosporin requires a specialized drug substance manufacturing process and is manufactured by Lonza Ltd. the company’s sole supplier for drug substance. Government Regulation The company worked with Otsuka to prepare a marketing authorization application (MAA) filing with the European Medicines Agency (EMA) that was filed during the first half of 2021 and an MAA filing with Swissmedic that was filed during the second half of 2021 and an MAA filing submitted to the UK's Medicines and Healthcare products (MHRA) for approval in Great Britain. Otsuka is required under the Otsuka License Agreement to use commercially reasonable efforts to prepare and submit filings for regulatory approvals in the other territories in which the company has granted them rights, including Japan, (in which the Japanese New Drug Application (J-NDA) was submitted on November 10, 2023) and selected other European countries. History Aurinia Pharmaceuticals Inc. was incorporated in 1993.