LK-01 Granted Orphan Drug Designation by the U.S. FDA for Acute Myeloid Leukemia

BARCELONA, Spain - Leukos Biotech, a spin-off company founded by the Barcelona based Jose Carreras Leukaemia Research Institute and supported by VCF Inveready, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for LK-01, a product based on Apomorphine, for the treatment of Acute Myeloid Leukemia.

Acute Myeloid Leukaemia (AML) is a blood cancer arising from the myeloid lineage of haematopoietic stem cells. Most patients with acute myeloid leukaemia (AML) die from progressive disease after relapse, which is associated with a small sub-fraction of leukemic cells termed leukemic stem cells (LSC). Current therapeutics target only the rapidly proliferating leukemic progenitors (blasts) , and not the more chemoresistant LSC. Therefore the pharmaceutical industry is making a serious effort to develop drugs which selectively target the LSC compartment with minimum toxicity to the healthy stem cells.

Leukos Biotech has shown that LSC overexpress Serotonin receptors and that apomorphine acts as an inhibitor of these, provoking the death of LSC. LK-01 is a new solid form of apomorphine that can be administered as a subcutaneous injection, and maintain clinically relevant concentrations of apomorphine in blood for several days.

The potential benefit of LK-01 is being explored in a Leukos Biotech-sponsored phase 1/2 trial in elderly patients and relapsed patients with AML. Phase II trial results are expected later in 2019.

The US FDA Orphan Drug Designation provides incentives for companies to develop drugs for rare diseases affecting fewer than 200,000 patients. These incentives may include FDA assistance in clinical trial design, tax credits towards the cost of clinical trials, prescription drug user fee waivers, and potential market exclusivity for seven years following approval.

Leukos US regulatory strategy for LK-01 is supported by Hemex AG, a Swiss consulting company specialised in collaborations with start-ups and spin-offs.

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Our CSO Dr. M. Risueño, publishes work on new therapeutic target for acute myeloid leukaemia.

Dr. Ruth M. Risueño's group has recently identified a new mechanism to combat acute myeloid leukaemia. The results of the study have been published in the journal Leukemia, a world standard bearer in the field of hematology. This study, the main author of which is the researcher Amaia Etxabe, describes a specific potential therapy for leukaemia stem cells.

Acute myeloid leukaemia was the first tumour in which a population of leukaemia stem cells was described. These are cells with the same properties as normal stem cells, so they have the capacity to reproduce malignant cells.

With standard chemotherapy, the rates of remission for this kind of leukaemia are currently between 50% and 85%, although most patients suffer a relapse of the disease. The leukaemia stem cells are the population of cells within the tumour responsible for initiating and maintaining the disease and for causing a relapse. For this reason treatment must eradicate them so that the disease can be eliminated completely.

During their research, the IJC group found that leukaemia cells express the serotonin receptor type 1 (HTR1) on their surface. The HTR receptors are typically associated with the nervous system, and this is the first time that their implication in the cancer process has been identified.

The inhibition of this receptor would lead to the destruction of this kind of leukaemia cell, but would have a minimal effect on healthy blood cells.

The study therefore shows the importance of HTR1 in the production of this kind of leukaemia and identifies this group of receptors as a new therapeutic target in AML with prognosis value.

More information:

http://www.nature.com/leu/journal/vaop/ncurrent/abs/leu201752a.html