Affymax, Inc., a clinical-stage pharmaceutical company, has initiated a clinical trial to evaluate the use of Hematide to treat anaemia in chronic kidney disease (CKD) patients with pure red cell aplasia (PRCA).

PRCA, a rare autoimmune disorder, occurs when the body produces neutralizing antibodies to recombinant human erythropoietin (EPO), thus suppressing the production of red blood cells by the bone marrow. Hematide is a synthetic, peptide-based, next-generation erythropoiesis-stimulating agent (ESA) with no sequence homology to human EPO. Hematide is designed to stimulate the production of red blood cells and is being evaluated in Phase 2 clinical trials to treat anaemia in patients with CKD and cancer.

Hematide has a novel amino acid sequence that is unrelated to EPO, a hormone that stimulates red blood cell formation, or to any other known naturally-occurring human sequences. Compared to therapeutic proteins, Hematide has the potential properties of a simple dosing schedule characterized by once-monthly administration, uncomplicated chemical synthesis, and room temperature storage.

"Although PRCA is a rare complication of treatment with recombinant EPO, it represents a significant adverse effect when it does occur. Patients with PRCA have no options to treat their anaemia except regular blood transfusions, a kidney transplant, or immunosuppressive therapy in an attempt to suppress antibody production, all of which are associated with adverse consequences," said Anne-Marie Duliege, vice president of clinical, regulatory, and medical affairs at Affymax. "Hematide may provide a treatment for these patients because antibodies generated to recombinant EPO have not been shown to cross-react with Hematide, and preclinical studies have shown that Hematide can effectively correct anaemia caused by EPO-specific antibodies," Anne-Marie added.


The open-label, multi-centre trial in PRCA patients will evaluate the efficacy and safety of Hematide administered subcutaneously every four weeks. The primary endpoint is the change in haemoglobin from baseline over time. Secondary endpoints include safety and the effectiveness of Hematide in reducing the frequency of red blood cell transfusions over time.

"We expect that this trial will provide important information about the safety and potential efficacy of Hematide in this select, transfusion-dependent group of patients with EPO antibody-induced anaemia," Robert B. Naso, executive vice president of research and development at Affymax said adding, "While we believe the PRCA population is too small to be considered as a target indication, we anticipate that the additional mechanistic information from this study may, if Hematide is approved by regulatory authorities, help us better differentiate Hematide from currently marketed recombinant EPO-based ESAs."