54% of Patients with AL Amyloidosis Reported Difficulty Tolerating Current Off-Label Treatments
Diagnosis of AL Amyloidosis is Often Missed by Cardiologists and Non-Specialist Hematologists
49% of Patients Required Visits to At Least Four Physicians and 34% Required Visits to At Least Five Physicians Before Diagnosis
Demonstrates Need for Safe, Effective Disease-Modifying Treatment Options for Systemic Amyloidosis
DUBLIN, Ireland, June 12, 2015 (GLOBE NEWSWIRE) -- Prothena Corporation plc (Nasdaq:PRTA), a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel protein immunotherapy programs, in collaboration with the Amyloidosis Research Consortium, presented results from a systemic amyloidosis patient experience survey identifying new opportunities to improve diagnosis and treatment of amyloidosis. The survey results (Abstract #E1262), released during the 20th Congress of the European Hematology Association, highlight that AL amyloidosis represents the vast majority of systemic amyloidoses and the need for safe, effective disease-modifying treatment options for systemic amyloidosis.
"Consistent with the survey results indicating that AL amyloidosis accounts for the vast majority of diagnosed systemic amyloidoses, a growing body of evidence now indicates that it is severely underdiagnosed and often misdiagnosed," said Isabella Lousada, President and CEO of the Amyloidosis Research Consortium. "As we educate specialists and other medical professionals to improve diagnosis, we see a clear need for safe and effective therapeutic options specifically designed to treat the underlying cause of AL amyloidosis as more than half of all patients with systemic amyloidoses reported difficulty tolerating current off-label treatment today."
Patient Experience Survey Results
Prothena collaborated with the Amyloidosis Research Consortium to gain insight into the experience of patients with systemic amyloidosis. The Amyloid Research Consortium conducted a 16-question survey of more than 500 participants, both patients and families. Despite AL amyloidosis being seen as a disease of the elderly, 70% were under the age of 65 and 34% were under the age of 55. Patients or families of patients with AL amyloidosis represented more than 72% of responses. These survey data demonstrated that diagnosis of AL amyloidosis is often missed by cardiologists and hematologists/oncologists who do not specialize in AL amyloidosis, where 49% of patients required visits to at least four physicians and 34% required visits to at least five physicians. More than 75% were ultimately diagnosed by a specialist, most frequently a hematologist/oncologist who specializes in AL
Consistent with the literature, most respondents experienced heart or kidney involvement. For treatment, 54% of respondents had difficulty tolerating current off-label treatments. Only 30% reported a definitive improvement in quality of life following treatment. Almost half of responding patients stated they would consider enrolling in a clinical trial, if informed.
"For patients with AL amyloidosis, establishing an early and accurate diagnosis presents a challenge today," said Dale Schenk, PhD, President and CEO of Prothena. "This survey highlights the opportunity to continue to partner with key opinion leaders, healthcare professionals, advocacy organizations and patients and their families to increase awareness of AL amyloidosis and to educate all constituencies about new potential treatment options, which may ultimately improve patient care."
NEOD001 is a humanized monoclonal antibody that specifically targets the circulating soluble amyloid and deposited insoluble amyloid that accumulates in both the AL and AA forms of amyloidosis. The ongoing multi-center Phase 1/2 clinical trial is evaluating the safety, tolerability, pharmacokinetics and immunogenicity of NEOD001 in patients with AL amyloidosis and persistent organ dysfunction. The study is also evaluating exploratory biomarkers for cardiac and renal function. Separately, The VITAL Amyloidosis Study, a double-blind, placebo-controlled, global Phase 3 registrational trial, will evaluate NEOD001 in newly-diagnosed, treatment-naïve patients with AL amyloidosis, and will assess all-cause mortality and cardiac hospitalizations in addition to biomarker, functional and quality of life endpoints. For more information on both the Phase 1/2 and VITAL Phase 3 trials, please
visit www.clinicaltrials.gov, and search identifiers NCT01707264 (Phase 1/2) and NCT02312206 (VITAL Phase 3).
About AL Amyloidosis
Systemic amyloidoses are a complex group of progressive diseases caused by tissue deposition of misfolded proteins that result in progressive organ damage. The most common type, AL amyloidosis or primary amyloidosis, involves a hematological disorder caused by plasma cells that produce misfolded AL protein resulting in deposits of abnormal AL protein (amyloid) in the tissues and organs of individuals with this disease. There are no approved treatments for AL amyloidosis that directly target potentially toxic forms of the AL protein. AL amyloidosis is a rare disorder and it is estimated that about 30,000 to 45,000 patients in the U.S. and Europe suffer from this disease. Both the causes and origins of AL amyloidosis remain poorly understood. For more information on AL amyloidosis, please visit the websites of the Amyloidosis Support Group and the Amyloidosis Foundation.
About the Amyloidosis Research Consortium (ARC)
The ARC was formed to help improve the understanding of the systemic amyloid diseases. The consortium brings together experts in the field to work as a team on cutting edge research. The ARC is [monitoring] a pipeline of key clinical trials and promising therapies. This research organization is committed to building relationships among patients, academia, industry, foundations, government, and regulators in order to swiftly deliver new treatments to patients. In partnership with the Amyloidosis Foundation, the ARC is driven by patient needs and dedicated to advocating on their behalf.
Prothena Corporation plc is a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel protein immunotherapy programs for the potential treatment of diseases that involve amyloid or cell adhesion. The company is developing antibody-based product candidates that target a number of potential indications including AL amyloidosis (NEOD001), Parkinson's disease and other related synucleinopathies (PRX002), and psoriasis and other inflammatory diseases (PRX003).