Pfizer and IBM Team Up On Remote Monitoring Project for Parkinson’s Disease

Pfizer Inc. and IBM are joining forces to develop remote monitoring solutions aimed at transforming how clinicians deliver care to patients suffering from Parkinson’s disease. The experimental approach will rely on a system of sensors, mobile devices, and machine learning to provide real-time, around-the-clock disease symptom information to clinicians and researchers. The ultimate goal is to obtain a better understanding of a patient’s disease progression and medication response to help inform treatment decisions and clinical trial design, while also speeding the development of new therapeutic options. “We have an opportunity to potentially redefine how we think about patient outcomes and 24/7 monitoring, by combining Pfizer’s scientific, medical and regulatory expertise with IBM’s ability to integrate and interpret complex data in innovative ways,” said Mikael Dolsten, MD, PhD, President of Pfizer Worldwide Research and Development, in a release.

The collaboration seeks to create a holistic view of a patient’s well-being by seeking to accurately measure health indicators, including motor function, dyskinesia, cognition, sleep, and daily activities such as grooming and eating. Insights from these data could help clinicians understand the effect of a patient’s medication as the disease progresses, enabling them to help optimize the patient’s treatment regimen. Data generated through the system could also arm researchers with the insights and real-world evidence needed to help accelerate potential new and better therapies. “The key to our success will be to deliver a reliable, scalable system of measurement and analysis that would help inform our clinical programs across important areas of unmet medical need, potentially accelerating the drug development and regulatory approval processes and helping us to get better therapies to patients, faster,” said Dr. Dolsten.

The two companies project that the system will move into initial clinical testing quickly. Pfizer and IBM will convene an external advisory board of patient groups, advocacy organizations, clinicians, and neuroscientists for guidance on the use of technology, medical devices, data management, and research protocols, and to ensure the needs of patients guide the program.

THE FDA FILE

New Drug Class Established as Nuplazid Receives FDA Approval for Parkinson’s Disease Psychosis

The FDA has approved Nuplazid (pimavanserin) from Acadia Pharmaceuticals for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis, which affects roughly 40 percent of patients with Parkinson’s disease. The first FDA-approved medicine for this indication, Nuplazid preferentially targets 5-HT2A receptors (which are thought to play an important role in Parkinson’s disease psychosis) while not interfering with patients’ dopaminergic therapy and thus not impairing motor function. According to Acadia, Nuplazid’s novel pharmacology establishes a new class of drug: a selective serotonin inverse agonist (SSIA).

In a pivotal Phase III study published in The Lancet, Nuplazid significantly reduced the frequency and severity of psychotic symptoms compared to placebo on the Scale for Assessment of Positive Symptoms – Parkinson’s Disease (SAPS-PD). The most common adverse reactions in this study were peripheral edema (seven percent for Nuplazid-treated patients versus three percent for those on placebo) and confusional state (six percent in Nuplazid-treated patients versus three percent in the placebo group).

Acadia plans to make Nuplazid commercially available to patients suffering from hallucinations and delusions associated with Parkinson’s disease psychosis in the US in June.

Marinus Pharmaceuticals Receives FDA Orphan Drug Designation for Ganaxolone IV to Treat Status Epilepticus

The FDA has granted Orphan Drug Designation to the intravenous (IV) formulation of a CNS-selective GABAA modulator, ganaxolone, for the treatment of status epilepticus from Marinus Pharmaceuticals. A Phase 1 clinical trial evaluating the safety, tolerability and pharmacokinetics of ganaxolone IV is expected to initiate in the first half of 2016. “Status epilepticus is a life-threatening condition, associated with high mortality and if not treated immediately, can result in brain damage, cognitive impairment and death,” remarked Christopher M. Cashman, Chief Executive Officer of Marinus Pharmaceuticals, in a release. “We are pleased to receive the Orphan Drug Designation for ganaxolone IV in this difficult to treat seizure disorder with limited treatment options.”

Investigational Progressive MS Agent Decreases Disease Progression in Phase 3 Trial

An investigational agent for the treatment of primary progressive multiple sclerosis has shown considerable promise in new phase 3 data presented at the recent AAN Annual Meeting. The study demonstrated evidence of the efficacy and safety of MD1003, a highly concentrated pharmaceutical-grade biotin administered at a dose of 300mg per day in the treatment of primary and secondary progressive MS. The primary endpoint was met in the intent to treat population, with 12.6 percent of patients in the MD1003 arm showing an improvement of EDSS or TW25 at Month 9, confirmed at Month 12, compared to none of the patients in the placebo arm. According to manufacturer medDay Pharmaceuticals, the data represent the first time a drug has decreased the rate of disease progression in addition to improving a significant proportion of patients with the progressive MS primary endpoint met.


Biomarker Analysis of Phase 2 Data Shows Potential for ALS Agent for Patients with Systemic Inflammation

Secondary analysis of Neuraltus Pharmaceuticals’ Phase 2 clinical trial of its investigational treatment, NP001, for amyotrophic lateral sclerosis (ALS), suggest 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. NP001 is designed to regulate inflammatory cell activity, as measured by CRP. In the Phase 2 study, Neuraltus prospectively measured CRP levels in participating patients. CRP is easily measured and high levels are recognized as indicating systemic inflammation. 

According to the company, the additional pre-defined analysis of the Phase 2 data involving CRP, in conjunction with other independent research in ALS patients, make it a credible biomarker for enriching patient selection in future studies of NP001.


Skin Disorder Tied to Increased Risk of Alzheimer’s Disease

A new study has uncovered an increased risk of dementia—in particular Alzheimer’s disease—in patients with rosacea, a chronic inflammatory skin disease. The risk may be highest in older patients and in patients where rosacea was diagnosed by a hospital dermatologist, according to the findings published in the April 2016 edition of the Annals of Neurology.

Rosacea is characterized by elevated expression of certain proteins—including matrix metalloproteinases and antimicrobial peptides—that are also involved in various neurodegenerative disorders such as Alzheimer’s disease and other forms of dementia. A Danish research team explored this link further, reviewing cases of more than 82,000 patients with rosacea from 1997 to 2012. After adjustments for potential confounding factors, patients with rosacea were estimated to have had a seven percent increased risk of dementia and a 25 percent increased risk of Alzheimer’s disease compared to individuals without rosacea. Women had a 28 percent increased risk of Alzheimer’s disease and men had a 16 percent increased risk if they had rosacea, while the risk of Alzheimer’s disease was only significantly increased in individuals older than 60 years (who had a 20 percent increased risk). When analyses were limited to patients with a hospital dermatologist diagnosis of rosacea only, the increased risks of dementia and Alzheimer’s disease were 42 percent and 92 percent, respectively. n

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Migraine Prevention Drug Shows Potential in Phase 2b Trial

An investigational agent from Alder BioPharmaceuticals has shown promise for migraine prevention. In a Phase 2b study, ALD403, a monoclonal antibody that targets calcitonin gene-related peptide (CGRP), acted rapidly and prevented migraine over a 12-week period, meeting both primary and secondary efficacy endpoints. Researchers randomized patients to receive a single intravenous infusion of 10mg, 30mg, 100mg, or 300mg of ALD403 or placebo, and found that the 300 mg and 100mg dose levels of ALD403 met the primary efficacy endpoint of 75 percent response rate in 12 weeks in 33 percent and 31 percent of patients, respectively.