Amyndas Pharmaceuticals S.A. is pleased to announce today that the Aplastic Anemia & MDS International Foundation (AA&MDSIF) is endorsing the clinical development of Amyndas' lead candidate AMY-101, as a novel treatment for paroxysmal nocturnal hemoglobinuria (PNH).
The AA&MDSIF awarded Prof Risitano and Prof Calado, a grant to fund the first clinical trial of the C3 complement inhibitor AMY-101, in untreated PNH patients. Profs Risitano and Calado are both distinguished experts in PNH research, based at Federico II University in Naples and the University of Sao Paolo, respectively.
"AMY-101 is currently considered one of the best candidates for addressing the unmet clinical need in PNH" commented Dr. Risitano. "This is the first time that AA&MDSIF funds a clinical trial, and we consider this support an important endorsement for the clinical potential of this novel peptide; we are grateful for this award and will use the funds to enroll the first 10-12 PNH patients in an upcoming Phase I/II clinical trial."
AMY-101 has already undergone extensive pre-clinical development and evaluation, and was found to provide complete and sustained complement inhibition. AMY-101 is therefore expected to be effective in patients suffering from PNH, even those who do not fully benefit from the current complement-directed standard treatment.
"This grant will accelerate the development of this new promising therapeutic for untreated PNH patients; if this drug candidate is successful, it could relieve the socioeconomic burden associated with the clinical management of PNH" said Dr. John D. Lambris, founder of Amyndas and Professor of Research Medicine at the University of Pennsylvania, adding "We are pleased to have Dr. Risitano at Federico II University in Naples as a partner to evaluate the potential therapeutic benefits of our compound in PNH clinical trials. Our aim is to bring an effective treatment to patients who remain untreated".
AMY-101 is a novel therapeutic based on the next-generation compstatin analogue Cp40, discovered by Dr. John Lambris of the University of Pennsylvania. AMY-101 inhibits complement component C3 and interrupts the complement activation cascade, which plays a pivotal role in PNH. Compared to first-generation compstatin analogues, AMY-101 shows an up to 6,000-fold improvement in binding affinity to human C3, enhanced inhibitory potency and an extended circulating half-life in vivo.
Amyndas has an exclusive, worldwide licensing agreement with the University of Pennsylvania that provides broad rights to develop and commercialize the University of Pennsylvania's next-generation compstatin analogues, which are potent complement-inhibiting peptides for the treatment of a range of complement-mediated disorders.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare but life-threatening hematological disorder. In PNH, important regulatory proteins are missing from the surface of blood cells, leaving them vulnerable to complement attack. Activation of complement causes red blood cells to break apart, a process called hemolysis, which clinically results in severe anemia and contributes to a high risk of clotting.
By targeting complement component C5, the monoclonal antibody eculizumab (Soliris®, an Alexion Pharmaceuticals drug) - to date the only approved therapeutic for PNH – reduces hemolysis and the need for blood transfusions for many PNH patients. However, one third of PNH patients continue to require blood transfusions to manage their anemia. The presence of fragments of complement component C3 on the surface of their red blood cells, which are eventually attacked by immune cells, is regarded as a major contributor to this insufficient response.
Inhibition of the complement cascade at the level of C3 is therefore considered a superior treatment strategy for PNH, as it can prevent both hemolysis and immune cell recognition. AMY-101 inhibits complement component C3 and interrupts the complement activation cascade.
About Amyndas Pharmaceuticals
Amyndas Pharmaceuticals S.A. and Amyndas Pharmaceuticals LLC, two transatlantic sister companies (based in Glyfada, Greece and Philadelphia, Pa., USA, respectively), are developing novel therapeutics to treat complement-mediated disorders. The distinct and enhanced therapeutic profile of these drugs when compared to other complement inhibitors is expected to open up prospects for treating new indications. The companies work closely together to move their novel drug candidates to the clinic, starting with applications in PNH and ABO-incompatible kidney transplantation, and potentially including other complement-mediated disorders, such as C3 glomerulopathy, periodontal diseases, age-related macular degeneration (AMD), hemodialysis-related inflammation, and ischemia-reperfusion injury.
The Aplastic Anemia & MDS International Foundation (AA&MDSIF, http://www.aamds.org) is an independent nonprofit organization whose mission is to support patients, families, and caregivers coping with aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and related bone marrow failure diseases. AA&MDSIF awards competitive grants to expert investigators to advance the understanding and treatment of aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH), leading to the development of new therapeutic approaches to treat these diseases. The grants are awarded according to merit, following evaluation by a distinguished panel of research scientists.
For more information please contact
Dr. Despina Yancopoulou
Amyndas Pharmaceuticals SA
N. Zerva 28, Glyfada 16675, Greece
T: +30 (210) 894-1213
e-mail: [email protected]