PharmaTher Announces Positive Topline Results from Clinical Study of Ketamine for Parkinson’s Disease

Rhea-AI Summary

PharmaTher Holdings Ltd. (OTCQB: PHRRF) announced positive topline results from its ketamine study for treating levodopa-induced dyskinesia in Parkinson's disease. The study found that 100% of patients treated showed a reduction in dyskinesias, and ketamine was well tolerated with no serious adverse events reported. These results support the preparation for a Phase 3 clinical study aimed at FDA approval of KETARX™ under the 505(b)(2) pathway. The global market for Parkinson's disease is projected to grow significantly, indicating potential commercial opportunities.

Positive

• 100% of patients treated with ketamine showed reduction in dyskinesias.
• Ketamine was well tolerated with no serious adverse events.
• Study results support moving to a Phase 3 clinical study.
• Potential market opportunity for ketamine in treating LID is significant, estimated over USD $3 billion annually in the U.S.

Negative

• No assurance that FDA will support expedited approval for ketamine.

100% of patients treated with ketamine demonstrated reduction in dyskinesias as measured by the Unified Dyskinesia Rating Scale

Ketamine was well tolerated with no serious adverse events reported

Study results are adequate to give an effect size in powering a Phase 3 clinical study

PharmaTher planning a Phase 3 clinical study to allow for FDA approval of KETARX™ (ketamine) for Parkinson’s disease under the 505(b)(2) regulatory pathway

TORONTO, March 23, 2022 (GLOBE NEWSWIRE) -- PharmaTher Holdings Ltd. (the “Company” or “PharmaTher”) (OTCQB: PHRRF) (CSE: PHRM), a leader in specialty ketamine pharmaceuticals, is pleased to announce positive topline results from the dose-finding and tolerability clinical study (the “Study”) of ketamine for the treatment of levodopa-induced dyskinesia (“LID”) in patients with Parkinson’s disease. The Study results are adequate to give an effect size in powering a Phase 3 clinical study, which is expected to bridge the design with increased treatment duration relative to the Company’s Phase 2 clinical study (NCT04912115).

The Study evaluated the safety, tolerability and efficacy of low-dose ketamine infusion for the treatment of LID in patients with Parkinson’s disease. Efficacy data from the Study demonstrated that 100% of patients treated with ketamine demonstrated a reduction in dyskinesias as measured by the Unified Dyskinesia Rating Scale during the Study period compared to the pre-treatment baseline. Safety data from the Study demonstrated that ketamine was well tolerated with no serious adverse events reported. All adverse events were mild or moderate and reflected the expected side-effects of ketamine administration. This Study further supports the previous findings in the observational study publication, titled “Case Reports Showing a Long-Term Effect of Subanesthetic Ketamine Infusion in Reducing l-DOPA-Induced Dyskinesias,” regarding the positive impact of ketamine in reducing LID. Full results of the Study are expected to be submitted for presentation at a medical congress by June 2022.

Dr. Scott Sherman, MD, PhD, Principal Investigator of the Study, commented: “The results of this clinical study further support that ketamine is well-tolerated even in an older population of patients with advanced Parkinson’s disease. This opens the door to a fast-track development of a novel non-surgical treatment for levodopa-induced dyskinesia that will fill a major gap in the existing therapeutic arsenal.” Dr. Scott Sherman is the Associate Professor of Neurology and Director of the Movement Disorders Center at the University of Arizona College of Medicine, Medical Director of the Arizona Chapter of American Parkinson’s Disease Association, and Director of the Parkinson’s Disease Program of the HealthSouth Rehabilitation Institute of Tucson.

Fabio Chianelli, CEO of PharmaTher, commented: “We are very pleased with the results from this clinical study as it gives us further confidence that ketamine can safely and effectively reduce levodopa-induced dyskinesia in patients with Parkinson’s disease and it paves the way for a potential Phase 3 clinical study to support FDA approval via the 505(b)(2) regulatory pathway.”

Based on the Study results, the Company is preparing to engage the FDA to establish the next steps for a planned Phase 3 clinical study to allow for ketamine’s approval for Parkinson’s disease under the 505(b)(2) regulatory pathway. The Company plans to use its proprietary ketamine intravenous product, KETARX™, for the planned Phase 3 clinical study.

Ketamine’s Potential In Parkinson’s Disease

Parkinson’s disease is a debilitating disorder that affects over 1 million people in the U.S. and more than 7 million people worldwide. The global Parkinson’s disease market is expected to grow from USD $5 billion in 2019 to USD $7.5 billion by the end of 2025 [360iResearch 2020] and it is estimated that the potential market opportunity for LID Parkinson’s disease to be over USD $3 billion in the U.S. alone.

Ketamine is an FDA-approved N-methyl-D-aspartate receptor-modulating (NDMA) drug that is widely used as an anesthetic agent either alone or in combination with other anesthetic agents [Smith et al, 1987; Pacheco et al, 2014]. The possible therapeutic effect of low-dose ketamine on levodopa-induced dyskinesia was noted in a retrospective analysis of Parkinson’s disease patients who received ketamine for pain relief. During this analysis, it was observed that the patients experienced an improvement in LID lasting several weeks beyond treatment [Sherman et al, 2016]. These results were corroborated in a test of low-dose ketamine in a rodent LID model, and this possible effect has also been examined in a controlled study [Bartlett et al, 2016]. Ketamine may also have additional benefits in the treatment of pain [Niesters et al, 2014] and depression [Diamond et al, 2014; Murrough et al, 2013], which are frequent comorbidities of Parkinson’s disease.

About Parkinson’s Disease        

There is currently no cure for Parkinson’s disease, although some drug combinations are used to treat the disease symptoms. Although the etiology of Parkinson’s disease is not fully understood, it is thought to result from loss of pigmented dopaminergic neurons in the Substantia nigra and their striatal projections, leading to dopamine deficiency in the striatum [Schapira and Jenner, 2011]. This ultimately affects the cortico-striatal system that controls movement. As a progressive neurogenerative disorder of the central nervous system that primarily affects the motor nerve system, symptoms of Parkinson’s disease may emerge slowly and include tremors, rigidity, bradykinesia, and postural instability [Paulson and Stern, 2004]. Also, patients may experience non-motor symptoms such as autonomic dysfunction (orthostatic hypotension, constipation, bladder dysfunction), psychiatric (depression), cognitive and sensory symptoms (pain) [Olanow, et al, 2009]. These non-motor symptoms become more common as the disease progresses. Treatments, including levodopa and dopamine agonists, which restore the dopamine deficits in the brain, have been employed for almost 50 years. However, with continued treatment using levodopa, dose-limiting motor side-effects often emerge. This includes the emergence of abnormal involuntary movements termed Levodopa Induced Dyskinesias, which can be identified in about 50% of patients within five years after initiation of levodopa treatment and in almost all patients within ten years post-treatment initiation. These side effects often limit further dose increases in dopaminergic therapy.

There can be no assurance that the FDA will support any potential request for an expedited path to approval or further development for ketamine in the treatment of Parkinson’s disease.

About PharmaTher Holdings Ltd.

​PharmaTher Holdings Ltd. (OTCQB: PHRRF) (CSE: PHRM) is focused on the development and commercialization of specialty ketamine pharmaceuticals for mental health, neurological, and pain disorders. Learn more at PharmaTher.com.

For more information about PharmaTher, please contact:

Fabio Chianelli
Chief Executive Officer
PharmaTher Holdings Ltd.
Tel: 1-888-846-3171
Email: info@pharmather.com
Website: www.pharmather.com

Neither the Canadian Securities Exchange nor its Regulation Services Provider have reviewed or accept responsibility for the adequacy or accuracy of this release.

Cautionary Statement

This press release contains 'forward-looking information' within the meaning of applicable Canadian securities legislation. These statements relate to future events or future performance. The use of any of the words "could", "intend", "expect", "believe", "will", "projected", "estimated", "potential", "aim", “may” and similar expressions and statements relating to matters that are not historical facts are intended to identify forward-looking information and are based on PharmaTher Holdings Ltd. (the "Company") current belief or assumptions as to the outcome and timing of such future events. Forward-looking information is based on reasonable assumptions that have been made by the Company at the date of the information and is subject to known and unknown risks, uncertainties, and other factors that may cause actual results or events to differ materially from those anticipated in the forward-looking information. Given these risks, uncertainties and assumptions, you should not unduly rely on these forward-looking statements. The forward-looking information contained in this press release is made as of the date hereof, and Company is not obligated to update or revise any forward-looking information, whether as a result of new information, future events or otherwise, except as required by applicable securities laws. The foregoing statements expressly qualify any forward-looking information contained herein. Factors that could cause actual results to differ materially from those anticipated in these forward-looking statements are described under the caption "Risk Factors" in Company's management's discussion and analysis for the period of November 30, 2021 ("MD&A"), dated January 27, 2022, which is available on the Company's profile at www.sedar.com.

This news release does not constitute an offer to sell or the solicitation of an offer to buy, and shall not constitute an offer, solicitation or sale in any state, province, territory or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state, province, territory or jurisdiction.

FAQ

What were the results of PharmaTher's ketamine study for Parkinson's disease?

The study showed that 100% of patients treated with ketamine experienced a reduction in dyskinesias.

When is PharmaTher planning to initiate the Phase 3 clinical study for KETARX™?

PharmaTher is preparing to engage the FDA to establish next steps for the Phase 3 clinical study.

What is the market potential for ketamine in treating levodopa-induced dyskinesia?

The potential market opportunity for LID treatment in Parkinson's disease is estimated to be over USD $3 billion in the U.S. alone.

What are the safety results of the ketamine study by PharmaTher?

Ketamine was reported to be well tolerated with no serious adverse events occurring during the study.

What regulatory pathway is PharmaTher pursuing for KETARX™ approval?

PharmaTher is pursuing FDA approval for KETARX™ under the 505(b)(2) regulatory pathway.