PNP

The Orphan Drug Designation underscores the significant unmet medical need and will grant LR 09 seven-year marketing exclusivity following drug approval by the FDA ZUG, Switzerland, Feb. 28, 2023 (GLOBE NEWSWIRE) -- Laevoroc Immunology (‘Laevoroc’ or ‘the company’), a privately-owned, Swiss oncology development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for LR 09, a novel metabolic immune checkpoint inhibitor, for the treatment of patients with haematological malignancies who are diagnosed with a relapse after allogeneic stem cell transplant (SCT). LR 09 is a novel, rationally-designed form of the small molecule drug, Ulodesine, a purine nucleoside phosphorylase (PNP) inhibitor that was originally developed for the treatment of autoimmune and inflammatory disorders. The decision to develop LR 09 for the treatment of relapse after stem cell transplantation follows the achievement of complete remission in a three-year-old patient using a pharmacologically analogous PNP inhibitor (1) whose US development was later discontinued, and the discovery that inhibition of PNP activates (instead of suppressing) the immune system (2). Recent research at the University of California, Los Angeles (UCLA), has revealed a novel, ground-breaking mode of action for LR 09 as a metabolic immune checkpoint inhibitor and supports its development for the treatment of patients experiencing relapse after allogeneic SCT. The data, published in 2022 in the Journal of Clinical Investigation, has documented the immune-activating effects of LR 09, through the elevation of intracellular guanosine, TLR7 activation, and the activation and proliferation of T-cells and germinal center B-cells, resulting in the initiation of a graft-versus-leukemia effects (3). With its favourable safety pro in earlier clinical development and its improved stability and manufacturing process versus earlier PNP inhibitors, the Orphan Drug Designation for the US market now adds to the promise of LR 09 as an oral immuno-oncology agent. “Receiving the FDA’s Orphan Drug Designation for LR 09 is a significant milestone for Laevoroc, bringing us one step closer to delivering a potential cure to patients who suffer relapse after allogeneic transplantation,” said Dr. Thomas Mehrling, haematologist-oncologist, co-founder, and CEO of Laevoroc. “Today, the prognosis for these patients remains extremely poor and they have no standard of care available to them. The Orphan Drug Designation underlines the significant unmet need for novel treatment options and Laevoroc is more determined than ever to permanently change this situation and restore hope for relapsing patients and their families.” The FDA grants Orphan Drug Designation to investigational therapies for rare diseases or conditions affecting fewer than 200,000 people in the United States. Receiving orphan status qualifies the sponsor for certain development incentives, including tax credits for clinical development costs and seven-year marketing exclusivity following drug approval by the FDA. Laevoroc Immunology acquired the commercial rights to LR 09 for new indications in 2021. The Company has active research collaborations with UCLA and the Fred Hutchinson Cancer Center in Seattle to support the drug’s development, with preclinical models of leukemia relapse after allogeneic SCT well underway. The Company has successfully raised USD 1.1 million in seed capital and is currently raising Series A financing to bring LR 09 to the clinic, and to patients, as swiftly as possible. References (1) (2) (3) About Laevoroc Immunology Laevoroc Immunology is a Swiss immuno-oncology development company based in Zug and founded in 2021 by Thomas Mehrling MD PhD, and Davide Guggi PhD, who have over 40 years’ combined experience in oncology and the pharmaceutical industry. The company is focused on developing immuno-oncology compounds with the potential to transform the treatment outcomes of medically underserved haematological malignancies and become game-changers in a wider range of applications. Its lead compound, LR 09 (a novel salt form of the small molecule drug, ulodesine), is a PNP inhibitor in preclinical development for the treatment of leukemia relapse following allogeneic stem cell transplantation. The development is supported by research collaborations with the University of California, Los Angeles (UCLA) and the Fred Hutchinson Cancer Center. Visit our website – Laevoroc.com and follow us on LinkedIn Contacts Laevoroc info@laevoroc.com Scius Communications (for Laevoroc) Katja Stout, Daniel Gooch Phone: +44(0)7789435990 Email: katja@sciuscommunications.com

UCLA data in the Journal of Clinical Investigation supports LR09 drug development as a metabolic immune checkpoint inhibitor for the treatment of leukemia Zug, Switzerland, 8 December 2022: Laevoroc Oncology (‘Laevoroc’ or ‘the company’), a privately-owned, Swiss oncology development company, announced today the publication of key preclinical data carried out at the University of California, Los Angeles (UCLA) which provides mechanistic insights into the immunoregulatory functions of purine nucleoside phosphorylase (PNP) with Laevoroc’s PNP inhibitor, LR 09. (1). The research shows that PNP is a novel metabolic immune checkpoint and LR 09 (Ulodesine), originally developed, and approved, for the treatment of gout, is a metabolic immune checkpoint inhibitor that activates the immune system through release of cytokines, expansion of germinal center B-cells and follicular helper T-cells. Relapse post-transplantation is the most frequent cause of treatment failure, in children and adults with leukemia. LR 09 has demonstrated an excellent safety pro the clinic and is formulated as an oral once-a-day treatment. Relapsed leukemia places a heavy economic burden on health systems. In the US healthcare setting, the mean total episode cost (from relapse date to death or end of study period), in a sample of around 700 patients, has been estimated, in 2019, at around $439,000 per patient (2). Laevoroc’s co-founder and CEO, Thomas Mehrling, MD, PhD, said, “We are delighted to have achieved this discovery with UCLA, which supports our vision for LR 09 to enable the treatment of relapsed leukemia — a devastating diagnosis for the patient and their families and an important cost burden for healthcare systems. Using our approach, we can help the grafted immune system recognize and combat the host leukemia, resulting in long-term response or cure.” Laevoroc Immunology’s CSO, Shanta Bantia, PhD, said, “We believe our innovation is a game-changing new therapy with the potential to become standard of care. Our vision with LR 09 is to enable the cure of relapsed leukemia.” Laevoroc Immunology continues to focus on fundraising to bring LR 09 to the clinic, and to patients, as swiftly as possible. Laevoroc acquired the commercial rights to LR 09 for new indications in 2021. References (1) (2) -Ends- About Laevoroc Immunology Laevoroc Immunology is a Swiss immuno-oncology development company based in Zug and founded in 2021, by Thomas Mehrling MD PhD, and Davide Guggi PhD, who have over 40 years’ combined experience in oncology and the pharmaceutical industry. The company is focused on developing immuno-oncology compounds with the potential to transform the treatment outcomes of medically underserved haematological malignancies and become game-changers in a wider range of applications. The company has raised over USD 1.1M in seed finance and is actively raising Series A financing for its subsidiaries. Visit our website – Laevoroc.com Contacts Laevoroc Oncology info@laevoroc.com Scius Communications (for Laevoroc Oncology) Katja Stout/Daniel Gooch Phone: +44(0)7789435990 Email: katja@sciuscommunications.com