KPAX002-1: Parkinson's Disease 

Fatigue and excessive daytime sleepiness (EDS) are key non-motor symptoms of Parkinson’s disease (PD). These symptoms severely impact quality of life and are the most common reasons PD patients apply for disability. They represent a significant unmet medical need in this population.

KPAX002, our phase 2 patented compound, is a proprietary version of methylphenidate. In essence, it is an enhanced, modified CNS stimulant, bolstered by our proprietary mitochondrial support technology, which possesses the ability to safely treat fatigue, cognitive slowness, and decreased alertness in patients with PD and other neurodegenerative diseases.

Methylphenidate has already shown positive preliminary evidence as a treatment for PD non-motor symptoms but, due to lack of IP protection, no further development has occurred. 

Identifying an effective and well tolerated treatment for the non-motor symptoms of Parkinson's disease addresses a key unmet medical need and presents a clinical stage market opportunity with significant potential. 

KPAX002 is patented through 2032. 

KPAX002-2: Alzheimer's Disease

There is a growing body of evidence linking mitochondrial dysfunction to the development and progression of cognitive decline in AD patients.

KPAX002, our phase 2 patented compound, is a proprietary version of methylphenidate. In essence, it is an enhanced, modified CNS stimulant, bolstered by our proprietary mitochondrial technology, which possesses the ability to safely treat fatigue, cognitive slowness, and decreased alertness in patients with neurodegenerative diseases. This proprietary therapeutic has significant potential to address the cognitive decline seen in AD patients. The mitochondrial modulator acts as a free radical scavenger to support and improve mitochondrial metabolism, while low dose methylphenidate enhances the residual cognitive function of remaining cortical neurons.

We are prepared to implement a phase 2 trial to demonstrate this patented compound can become the first disease-modifying treatment for Alzheimer’s disease.  In addition, KPAX002 could be used as part of combination therapy, to enhance the effectiveness of other treatments with different mechanisms of action (i.e. beta and gamma secretase inhibitors). By making use of the 505(b)2 regulatory pathway, the approval of KPAX002 would be expedited. 

KPAX002-3:  Myalgic Encephalomyelitis/CFS 

Chronic Fatigue Syndrome (CFS), also known as Myalgic Encephalomyelitis (ME), is a serious unmet medical need afflicting 2-3 million patients in the US. The FDA has issued a draft guidance to industry and is on record as being highly motivated to approve a first treatment for this disease. KPAX002 is the leading candidate ready to take advantage of this unique opportunity.

KPAX002 is an enhanced, modified version of methylphenidate being developed as a safe and effective treatment for fatigue. K-PAX Pharmaceuticals’ patented mitochondrial modulator technology improves the metabolism and long term tolerability of the methylphenidate API.

Having completed successful phase 1 (n=15) and phase 2 (n=128) placebo-controlled trials in patients with ME/CFS, we are now ready to implement a pivotal phase 3 trial. KPAX002 possesses strong IP protection through 2032.