Neuraltus Pharmaceuticals Initiates Confirmatory Phase 2 Study of NP001 in Patients with Amyotrophic Lateral Sclerosis

PALO ALTO, CA, USA I September 22, 2016 I Neuraltus Pharmaceuticals, Inc., a privately-held biopharmaceutical company dedicated to developing innovative therapeutics for neurodegenerative diseases, announced today the initiation of a second Phase 2 study of its investigational treatment, NP001, in patients with amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease) in North America. This Phase 2 study (NCT02794857) is being conducted to confirm previous Phase 2 clinical study findings in ALS patients with elevated levels of baseline inflammation.

ALS is a rare and fatal neurodegenerative disease affecting approximately 400,000 individuals worldwide. ALS progression is believed to be driven, in part, by systemic inflammation and NP001 is designed to regulate cells associated with the body's inflammatory response. In the first Phase 2 study of NP001, Neuraltus assessed the safety, tolerability, and preliminary efficacy of two dose levels of NP001 versus placebo using the ALS Functional Rating Score Revised (ALSFRS-R), which measures key activities of daily living and function for patients with ALS. A secondary analysis of the study results suggested that increased levels of a biomarker for systemic inflammation, C-reactive protein (CRP), may indicate which patients are more likely to have a positive response.

The primary objective of the second Phase 2 study is to further explore the effects of NP001, determine if patients with elevated baseline inflammation might benefit from NP001, and generate additional data about NP001's safety and tolerability to guide a Phase 3 trial.

"NP001 is a very promising investigational therapy for ALS and other related neurodegenerative diseases," said Dr. Lawrence Steinman, chairman of Neuraltus' scientific advisory board and a professor of pediatrics, neurology and neurological sciences at Stanford University. "The drug's anti-inflammatory mechanism of action is very attractive and capitalizes on numerous recent research insights about the importance of the immune system in driving the progression rates of many neurodegenerative disorders. The preliminary evidence from the Company's first Phase 2 clinical trial of NP001 is quite compelling and should be confirmed by this next Phase 2 trial in patients with elevated levels of CRP."

"The initiation of the second Phase 2 study of NP001 marks an important step in ALS research," said Rich Casey, chief executive officer, Neuraltus Pharmaceuticals. "We are encouraged by the results of the first Phase 2 study of NP001 and pleased to continue our evaluation of this innovative, targeted investigational therapy for people suffering from this progressive and life-threatening condition."

Study Design

The second Phase 2 study is a randomized, double-blind, placebo-controlled, multicenter study enrolling up to 120 subjects in North America with ALS and evidence of systemic inflammation. Patients will receive either NP001 2 mg/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. Further information about the study is available at https://clinicaltrials.gov.

About Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) is a rare and fatal neurodegenerative disease characterized by degeneration of motor neurons in the spinal cord and brain. The cause of the disease is currently unknown, but there is increasing evidence that implicates neuroinflammation with the progression of the disease. It is believed that in people with ALS, there are increased levels of inflammatory (activated) macrophages, a type of white blood cell, resulting in the release of factors in the central nervous system that damage motor neurons. NP001, a regulator of macrophage activation, exerts its effect by converting these activated inflammatory macrophages back to their normal state.

There are approximately 400,000 ALS patients worldwide. There are no existing treatments that meaningfully slow the progression of or stabilize the disease, underscoring the need for new and effective drug therapy.

About Neuraltus Pharmaceuticals, Inc.

Neuraltus Pharmaceuticals, Inc. is a privately-held biopharmaceutical company dedicated to developing and commercializing innovative therapeutics that address critical unmet needs for patients and physicians in the treatment of neurodegenerative diseases. The Company is collaborating with global ALS specialists and seeking input from patient advocacy organizations to help inform the clinical development path and better understand the needs of people affected by the disease.

SOURCE: Neuraltus Pharmaceuticals

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