Jasper Therapeutics’ briquilimab has been granted orphan drug designation by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) as a conditioning treatment for patients before being given a haematopoietic stem cell transplant (HSCT), the company announced.
The anti-c-KIT monoclonal antibody is currently being evaluated as a conditioning agent prior to HSCT for patients with severe combined immunodeficiency (SCID) and separately for patients with acute myeloid leukaemia (AML) or myelodysplastic syndromes (MDS).
In these diseases, prognosis is poor for patients and transplant rates are low as a result of the highly toxic conditioning required, the biotech outlined.
Jasper’s ongoing clinical trial in SCID – a rare and life-threatening paediatric disorder that causes major abnormalities of the immune system – has already shown briquilimab to be well tolerated, with no treatment-related adverse events across multiple patients. Successful stem cell engraftment and immune reconstitution have also been observed in the trial.
Briquilimab has also demonstrated a tolerable safety profile, with full donor chimerism in all 24 patients with MDS – a group of blood cancers characterised by ineffective production of healthy red blood cells, white blood cells and platelets – or AML.
The company’s president and chief executive officer, Ronald Martell, said: “Transplants have the potential to cure several haematologic cancers and genetically inherited diseases. However, the toxicities associated with genotoxic conditioning needed to prepare patients for these procedures often limit their use.
“We believe that briquilimab has the potential to fill this gap, effectively expanding access to curative stem cell transplant across a range of indications. With the orphan designation, the EMA has demonstrated support of briquilimab in Europe and Jasper is committed to advancing this therapy globally.”
Previously, the US Food and Drug Administration granted orphan drug designation to briquilimab in HSCT, as well as rare paediatric disease designation for the treatment of SCID.
The company also announced positive results earlier this month from a phase 1/2 trial of briquilimab as a conditioning treatment in patients with sickle cell disease (SCD) and beta thalassaemia considered at high risk for complications from or ineligible for standard myeloablative HSCT.
In the study, all three SCD patients treated with briquilimab were successfully engrafted with no treatment-related severe adverse events observed.