Lorecivivint

LOR is now under regulatory review in both the United Kingdom and the European UnionSAN DIEGO, Oct. 02, 2026 (GLOBE NEWSWIRE) -- Biosplice Therapeutics, Inc. (“Biosplice”), a late-stage biopharmaceutical company developing lorecivivint (“LOR”), a first-in-class small-molecule inhibitor of CLK/DYRK kinases, for the treatment of knee osteoarthritis (“OA”), announced today that the European Medicines Agency (“EMA”) validated Biosplice’s marketing authorization application for LOR on September 30, 2026. Validation confirms that the application is complete and starts the EMA’s centralized review. LOR is already under assessment in the United Kingdom, where the Medicines and Healthcare products Regulatory Agency (“MHRA”) validated Biosplice’s application on August 28, 2026. LOR is a small-molecule suspension intended to be injected 1–2 times per year into the knee joint. It has been evaluated in eleven clinical trials, with improvements in joint structure, pain and function, together with a well-established safety profile. Evidence supportive of disease modification was generated primarily in Biosplice’s Phase 3 OA-07 trial, which showed that LOR improved medial joint space width (JSW) on imaging over multiple years—change in JSW measured by X-ray is the structural endpoint European regulators accept in OA. These results support LOR’s potential to be the first disease-modifying therapy for OA, a milestone long sought by patients, physicians and researchers. The EMA’s validation follows the Committee for Medicinal Products for Human Use (“CHMP”) confirming in February 2026 that LOR is eligible for the centralized procedure as a new active substance. Validation is an administrative step that confirms the dossier is sufficiently complete for the full assessment to begin. Dr. Yusuf Yazici, Chief Medical Officer of Biosplice, commented, “European regulators specified what counts as evidence of structural damage in osteoarthritis back in 2010: joint space narrowing measured by standing X-ray, with standardized methodology, is an acceptable primary endpoint. We included this standard in our clinical development program, which also demonstrated significant benefit in pain and function.” Erich Horsley, Chief Executive Officer of Biosplice, added, “The UK validated our application on August 28, and the EU validated it on September 30, so lorecivivint is now under review in both major markets. Fifty million American adults have osteoarthritis and not one of them has yet been offered a disease-modifying option. LOR was discovered and developed in America by an American company. We would like nothing more than to bring this potentially life-altering OA therapy to American patients, in addition to those in the UK and Europe, as soon as possible.” The EMA’s guideline on medicinal products for the treatment of osteoarthritis (CPMP/EWP/784/97 Rev.1), in force since August 2010, states that joint space narrowing measured by X-ray, “with appropriately standardised methodology, is an acceptable primary endpoint for assessment of structural damage.” The same guideline records that X-ray measurement of joint space narrowing “has been shown to correlate with the subsequent need for total joint replacement,” while cautioning that the precision of the measurement varies with radiographic technique, and it calls for structural studies to run no shorter than two years. OA-07 measured medial joint space width by X-ray for two years, consistent with that guidance. OA-07 demonstrated joint structure benefits rarely observed in OA research: patients treated with LOR maintained and ultimately improved their medial JSW, as measured by X-ray at two years following annual injections. Placebo patients whose medial JSW decreased during the first year of OA-07, once crossed over to active treatment with LOR, also showed increases in medial JSW. Beyond structural improvement, the Phase 3 OA-07 study showed patients dosed with LOR significantly improved their pain scores at 6 months and both pain and function at 12 months, compared with placebo. Improvements in pain and function, and structural benefit in a prespecified subgroup, were also seen in Phase 2 trials, consistent with the Phase 3 results. The figure below shows pain scores in the OA-07 study. 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 that 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 that break down hyaline cartilage. Inhibition of CLK2 alters local Wnt pathway signaling, which is believed to promote chondrocyte formation and protect hyaline cartilage. Any application for the approval of a new medicine in OA must be supported by data demonstrating that it is safe. Throughout the decade-long clinical development program, LOR’s safety profile has been consistent with that of placebo. LOR’s safety is enhanced by the fact that, when injected into the knee joint, the drug has no detectable systemic exposure. 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 about 25 million U.S. adults, 1 in 10, have knee OA alone.1 Although OA is often considered a disease of old age, approximately half of symptomatic knee OA diagnoses occur by age 55.2 Those who live with OA experience pain, stiffness and swelling, which limit 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 U.S. 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 space of the knee 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 of DYRK1A and CLK2 kinases. DYRK1A inhibition has been shown to reduce inflammation and catabolic enzyme activity, and inhibition of CLK2 is believed to provide cartilage-protective and regenerative properties. The extensive clinical development program for LOR has provided evidence of statistically and clinically significant improvement in pain, function and medial JSW, with a well-established safety profile. LOR is an investigational medicine and has not been approved by the MHRA, the EMA, the FDA or any other regulatory authority. About Biosplice Biosplice is a late-stage biopharmaceutical company focused on lorecivivint, a first-in-class small-molecule inhibitor of CLK/DYRK kinases being developed as a potentially disease-modifying treatment for knee osteoarthritis. Biosplice’s work is grounded in novel biology linking CLK/DYRK kinases to the therapeutic regulation of alternative splicing. Lorecivivint is under regulatory review in the United Kingdom and the European Union. Biosplice also holds a majority interest in TenaRx, which is developing programs in oncology, diabetes and neurology. Learn more at https://www.biosplice.com. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding the MHRA’s and the EMA’s assessment of Biosplice’s marketing authorization applications for lorecivivint, the expected timing and outcome of those assessments, the review and potential approval of lorecivivint by the MHRA, the EMA or any other regulatory authority, 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 from those expressed or implied by those statements. These include, among others, the uncertainties inherent in the regulatory review process, including the possibility that a regulatory authority may request additional information, may extend or suspend its review timetable, or may decline to grant approval, and other risks associated with drug development and commercialization. Statements regarding statutory review timetables reflect published regulatory guidance and are not assurances as to the timing of any regulatory decision. 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. 1 Institute for Health Metrics and Evaluation (IHME). GBD 2021 Results. Seattle, WA: IHME, University of Washington; accessed October 1, 2026. https://vizhub.healthdata.org/gbd-results/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. https://oarsi.org/sites/oarsi/files/docs/2016/oarsi_white_paper_oa_serious_disease_121416_1.pdf Photos accompanying this announcement are available at https://www.globenewswire.com/NewsRoom/AttachmentNg/85203e0e-0da9-4862-bdf4-b1e88ad295f3 https://www.globenewswire.com/NewsRoom/AttachmentNg/cf255baa-c3ea-412f-8a35-44ca94937cc2 Corporate Contact: Phil Wilson, CFO Biosplice Therapeutics, Inc. phil.wilson@biosplice.com