Lorecivivint

LOR is the first drug candidate with disease-modifying potential for osteoarthritis submitted for approval, demonstrating statistically and clinically significant improvements in pain, function and medial joint space width when compared with placebo, along with very good safety SAN DIEGO, Jan. 06, 2026 (GLOBE NEWSWIRE) -- Biosplice Therapeutics, Inc. (“Biosplice”), a clinical-stage biotechnology company pioneering first-in-class therapeutics based on small-molecule inhibition of CLK/DYRK kinases, announced today that it has submitted its NDA to the FDA for approval of its novel therapy for the treatment of knee osteoarthritis (“OA”). Biosplice’s drug candidate, lorecivivint (“LOR”), is a small-molecule suspension intended to be injected 1-2 times per year into the knee joint. LOR, evaluated in 11 clinical trials, significantly improved pain and function in patients with a very good safety profile. Results from Biosplice’s Phase 3 OA-07 trial also showed that LOR led to improvements in medial joint space width (JSW) on imaging over multiple years. These results support LOR’s potential as the first disease-modifying therapy for OA, a milestone long sought by patients, physicians and researchers. In the 2-year Phase 3 OA-07 study, patients dosed with LOR significantly improved their pain at 6 months and both pain and function at 12 months, compared to placebo. Similar improvements were also seen in Phase 2 trials, confirming the symptomatic benefit of LOR in OA patients. The figure below shows pain scores, which historically represented the primary approval endpoint for OA with the FDA, from Biosplice’s OA-07 study. Beyond symptom relief, OA-07 demonstrated joint structure benefits rarely observed in OA research: patients treated with LOR in the OA-07 study, maintained and ultimately improved their medial JSW, as measured by X-ray at two years following annual injections. Placebo patients who decreased their medial JSW in the first year of OA-07, once crossed over to active treatment with LOR, also showed increases in medial JSW following cross-over. Erich Horsley, CEO of Biosplice, commented, “After over a decade of clinical trials, we are pleased to be the first company to submit a data package in the US for a potentially disease-modifying drug in knee OA for approval. As many knee OA patients are aware, the patient journey for someone diagnosed with OA is currently painful and disheartening. We are excited about the prospects of materially improving the OA patient’s journey with a new treatment that is both safe and effective and has the potential to delay further structural progression.” Dr. Yusuf Yazici, Chief Medical Officer of Biosplice, continued, “Once approved, LOR promises to bring near-term pain relief and long-term structural benefit to 25 million American knee OA patients. We are particularly excited by LOR’s safety profile, which is similar to placebo across over 1,800 patients dosed, while demonstrating significant improvement in pain, function and structure.” Biosplice has been developing LOR for over a decade and initially synthesized and identified the novel compound in 2011. LOR is a small-molecule drug which selectively inhibits two kinases, DYRK1A and CLK2. DYRK1A is an inflammatory kinase, the inhibition of which results in significant reductions in multiple inflammatory cytokines and catabolic enzymes which break down hyaline cartilage. CLK2 alters local Wnt pathway signaling, which is believed to promote chondrocyte formation and the protection of hyaline cartilage. Any new drug application in OA must be supported by a good safety profile. Throughout the decade-long clinical development program, LOR’s safety pro been consistent with and similar to that of placebo. LOR’s safety is enhanced by the fact that, when injected into the knee joint, the drug has no detectible systemic exposure throughout the body. Professor Tim McAlindon, from UMass Chan School of Medicine, who has advised Biosplice and has been involved in the development program for LOR, commented, “It is rare to see a drug with LOR’s safety profile, to say nothing of its disease-modifying potential. The unmet need in OA is well-known, and patients have not had access to novel treatments in decades. The approval of LOR would potentially be a remarkable development for OA patients.” Seth Ginsberg, Co-Founder and President of Global Healthy Living Foundation and CreakyJoints, amplifies the voices of millions of arthritis patients through the efforts of his organization. He commented, “Osteoarthritis affects more than 50 million Americans and remains one of the leading causes of pain, disability, and loss of independence worldwide. For patients, the disease shapes daily decisions, careers and quality of life, often over many years, while meaningful progress that addresses the disease itself rather than symptoms alone has remained out of reach. For these reasons, an approval of LOR could be immensely impactful for OA patients.” About Osteoarthritis Osteoarthritis (OA), the most common form of arthritis, is a serious chronic disease that affects an estimated 50 million U.S. adults and over 500 million adults globally 1 . OA is the most prevalent joint disease and a leading source of chronic pain and disability in the United States. OA may affect the knees, hips, shoulders, hands, spine, or great toes; current estimates indicate that there are about 25 million, 1 in 10 U.S. adults, with knee OA alone in the US i . While often considered a disease of old age, approximately half of symptomatic knee OA diagnoses occur by the age of 55. 2 Those who live with OA experience pain, stiffness, and swelling, which limits their function and mobility, often impacting their daily lives. Functional limitations are present in about 80% of people with OA, and 25% are unable to perform major activities of daily living 3 . Mortality is higher in patients with knee and hip OA largely due to cardiovascular disease, with a higher risk of death associated with severity of walking disability 4 . Given both the increased morbidity and mortality, the US FDA has accepted knee OA as a ‘serious condition’. About Lorecivivint (LOR) Lorecivivint is a small-molecule suspension drug intended to be injected into the intra-articular knee joint space for the treatment of OA. LOR has been tested as a once-per-year or every-six-month injection in Biosplice’s development program. LOR’s mechanism of action is based on selective and potent inhibition DYRK1A and CLK2 kinases. DYRK1A inhibition has been shown to reduce inflammation and catabolic enzymes, and inhibition of CLK2 is believed to provide cartilage protective and regenerative properties. The extensive clinical development program for LOR has provided evidence for statistically and clinically significant improvement in pain, function and medial JSW with a very good safety profile. LOR has not yet been approved by the FDA. About Biosplice Biosplice is developing first-in-class, small-molecule therapeutics based on novel biology linking CLK/DYRK kinases to the therapeutic regulation of alternative splicing. Biosplice strives for disease-modifying solutions in all of its drug candidates. Biosplice’s drug candidates in development include lorecivivint for osteoarthritis (in NDA stage), and, through its majority-owned subsidiary, TenaRx, programs in oncology, diabetes and neurology. Learn more at . Forward-Looking Statements This press release contains forward-looking statements, including statements regarding the review and potential approval of Biosplice’s New Drug Application for lorecivivint and the therapeutic potential of lorecivivint. Forward-looking statements are based on current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to differ materially. These risks and uncertainties include, among others, uncertainties inherent in the regulatory review process and other risks associated with drug development and commercialization. Biosplice undertakes no obligation to update or revise any forward-looking statements contained herein, whether as a result of new information, future events, or otherwise. Corporate Contact: Phil Wilson, CFO Biosplice Therapeutics, Inc. phil.wilson@biosplice.com 1 IFHMaE. 2021. ‘GBDx Results Tool,’ IHME, University of Washington, Accessed January 7, 2021. 2 Losina E, et al. Arthritis Care Res (Hoboken). 2013. 3 Neogi T. Osteoarthritis Cartilage. 2013. 4 Osteoarthritis Research Society (OARSI). Osteoarthritis: A Serious Disease. Published December 1, 2016. Accessed June 25, 2024. Photos accompanying this announcement are available at: