PharmaTher Holdings Nabs New Orphan Drug Status, Discusses Further Fast-Track, Partners For MDMA Patch

Ketamine products manufacturer PharmaTher Holdings Ltd. PHRRF had lots happening this past week. Here are the company's top three news items.

FDA’s Orphan Drug Status To Ketamine For Rett Syndrome

The FDA granted an orphan drug designation to the company’s proprietary racemic ketamine KETARX for the treatment of the rare genetic neurological disorder Rett Syndrome. 

Ketamine has been tested in Phase 2 clinical trial for Rett syndrome, of which unpublished results will be used to support a potential Phase 3 clinical study and obtain the FDA’s green light on a regulatory plan for approval via the 505(b)(2) regulatory pathway.

The new designation adds to four others held by PharmaTher on KETARX, for the prevention of Ischemia-reperfusion injury from organ transplantation, the treatment of Status Epilepticus and Amyotrophic Lateral Sclerosis (ALS), as well as complex regional pain syndrome.

Potential Fast-Track Designation To Ketamine For Parkinson’s

PharmaTher submitted an FDA meeting package to discuss its Phase 3 program as well as fast-track designation for KETARX in Parkinson’s disease.

Phase 3 development would target the company’s proprietary ketamine as a treatment for levodopa-induced dyskinesia in Parkinson’s disease (LID-PD). 

Should results be positive, they would support a new drug application under the 505(b)(2) regulatory pathway for KETARX in treating this specific condition.

Furthermore, the company has requested FDA guidance towards obtaining Fast Track Designation for KETARX, a response the company expects to receive by March 20, 2023.

PharmaTher expects the case may be supported by results from its completed clinical study, which include safe, well-tolerated and reduction in dyskinesias from baseline in participants -all with moderate to advanced Parkinson’s-, specifically 51% reduction during infusion 2, 49% at 3 weeks, and 41% at 3 months post-ketamine.

On the same topic, PharmaTher is currently retaining the rights to a US Patent covering compositions and methods for treating motor disorders, including ketamine in the potential treatment of Parkinson’s and motor disorders that cause involuntary or uncontrollable movement or actions of the body.

A Partnership On MDMA Delivery With Novel Transdermal Patch

PharmaTher entered into a research collaboration agreement with life sciences psychedelics company Revive Therapeutics Ltd. RVVTF towards evaluating MDMA delivery through the former’s novel microneedle patch delivery technology.

Results from PharmaTher’s completed non-clinical study assessing its MDMA microneedle patch will be available in the first half of 2023 and will be used to support a potential human clinical study. 

If the results are positive, partners will finalize a product and clinical development plan to initiate regulatory discussions for future clinical studies in various indications for MDMA including depression, anxiety, abuse disorders and PTSD.

Revive’s CEO Michael Frank explained that this test will complement the company’s therapeutic psilocybin programs, which include a Phase 1/2 clinical study for meth use disorder, developing a novel psilocybin oral thin film strip, and developing a biosynthetic version of psilocybin based on a natural biosynthesis enzymatic platform.

Based on novel biocompatible and biodegradable gelatin methacryloyl material delivery to deliver water-soluble and insoluble drugs, the new patch is expected to enable flexible drug load capacity and combinations, controlled release delivery, and be able to present desired pharmacokinetic (PK) and safety profiles potentially overcoming obstacles of oral dosing.

Photo: Benzinga edit with photo by DyrElena by Shutterstock and Wikimedia Commons.