Neuraltus recognizes the urgent need for new ALS treatments and is dedicated to working in collaboration with the ALS community as we continue the development of our lead product candidate, NP001. We are committed to conducting well-designed and robust clinical research that will yield meaningful insights about the safety and efficacy of NP001 and recognize that clinical development is an involved – and often lengthy – process.
We have explored several options to potentially provide NP001 to ALS patients in a fair and unbiased fashion in advance of its review by the U. S. Food and Drug Administration, but have yet to identify a program that would allow patients access while protecting their well-being and insuring the integrity of the NP001 clinical research. As a result, Neuraltus has made the decision to focus its limited resources and drug supply to expedite enrollment and completion of the ongoing North American Phase 2 double-blind, randomized, placebo-controlled trial. At this time there are no plans to offer NP001 on an open-label basis or through an early access program.
The ongoing Phase 2 study will enroll up to 120 ALS patients with evidence of systemic inflammation. Patients will receive either NP001 2mg/kg or placebo over a period of 6 months. The study is designed to evaluate the change from baseline in ALSFRS-R during the study period. Secondary objectives include a change in pulmonary function as measured by vital capacity readings. Additional information about the confirmatory Phase 2 study can be found on ClinicalTrials.gov.
We are grateful to the patients, caregivers, advocacy groups, investigators and researchers who have contributed to and supported the clinical advancement of NP001. We will continue our discussions with all of these groups as we assess how we can best address the needs of this community and advance the development of NP001.