• Tuesday, February 28, 2023 @ 9:00 am

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

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 profile demonstrated 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.


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