Orphan drug designation for Laevoroc metabolic immune checkpoint inhibitor

stem cell transplant

Laevoroc Immunology, a privately-owned, Swiss oncology development company, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for immune checkpoint inhibitor LR 09.

LR 09 is a novel metabolic immune checkpoint inhibitor, for the treatment of patients with hematological malignancies who are diagnosed with a relapse after allogeneic stem cell transplant (SCT). 

LR 09 is a novel 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, whose U.S. development was later discontinued, and the discovery that inhibition of PNP activates the immune system, rather than suppressing it. 

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 effect. With its favorable safety profile demonstrated in earlier clinical development and its improved stability and manufacturing process versus earlier PNP inhibitors, orphan drug designation for the U.S. market now adds to the promise of LR 09 as an oral immuno-oncology agent.

Thomas Mehrling, hematologist-oncologist, co-founder, and CEO of Laevoroc, said the FDA designation brings Laevoroc one step closer to delivering a potential cure for patients who suffer relapse after allogeneic transplantation. 

“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.”

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 under way.

Laevoroc Immunology has raised $1.1 million in seed capital and is currently raising Series A financing to bring LR 09 to the clinic, and to patients, as quickly as possible. 

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