Cannabis-Derived MS, Huntington Treatments Submitted to FDA for Fast Track, Orphan Drug Status

February 14, 2019

MMJ International Holdings’ 2 cannabis-derived treatments, MMJ-001 and MMJ-002, were submitted for Fast Track and Orphan Drug designations, respectively, the latter of which was approved by the FDA.

Duane Boise

MMJ International Holdings, a company committed to developing FDA-approved cannabis medicines that best serve patients with unmet medical needs, has announced that it is seeking a Fast Track designation from the regulatory agency for MMJ-001, its cannabis-based pharmaceutical candidate for the treatment of multiple sclerosis (MS).1

A dose-ranging study is being planned by the company for MMJ-001, in which it plans to compare its formulation of both tetrahydrocannabinol (THC) and cannabidiol (CBD) with placebo. According to the MMJ Group, the company is “focused on collaborative scientific research projects that follow the FDA guidelines.”

MMJ is expecting the trial to demonstrate acceptable and tolerable doses of MMJ-001. It noted that it will conduct clinical trials to assess the potential of its cannabis-derived medicines for both primary progressive MS (PPMS) and Huntington disease.

"The positive data from this dosing study is important in that it will provide us with the necessary evidence on patient tolerance to different doses of THC and CBD for the treatment for PPMS,” Duane Boise, MMJ’s chief executive officer, said in a statement. “This information will prove value to assist clinicians with prescribing decisions to address individual patient needs should this medicine be FDA approved for use."

The company’s principal investigator is Bianca Weinstock-Guttman, MD, a professor of neurology at the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo, as well as the executive director of the New York State MS Consortium. She will oversee the study exploring the potential therapeutic applications of cannabinoids for patients with PPMS, according to the company.

In addition to its application for Fast Track designation, another of the company’s agents, MMJ-002, was granted an Orphan Drug designation by the FDA for the treatment of Huntington disease.2 The therapy is one of the company’s lead agents, and MMJ “is confident that it will bring much-needed relief to patients suffering from the debilitating effects of Huntington disease.”

Boise said in a statement that “MMJ’s Orphan Drug award is another milestone that the company has met as we continue to set industry standards. MMJ has established a leading position in the development of plant-derived cannabinoid therapeutics through its proven drug discovery and development processes, intellectual property portfolio, and regulatory and manufacturing expertise.”

“However, with this Orphan Drug award, the most significant benefit is to the patients with Huntington disease, since our development process can be advanced quickly,” Boise added.

The company has a scheduled phase 2 clinical trial for the MMJ-002 development program, in which Boise expressed confidence that the candidate would demonstrate an ability to slow or stop the deleterious effects of Huntington chorea.

“We are working diligently to address the significant unmet medical need in people suffering from this deadly disease and this designation furthers our mission to develop impactful cannabinoid-derived medicines to improve clinical outcomes for patients with Huntington disease,” Elio Mariani, PhD, the company’s director of research and development said.

REFERENCES

1. MMJ Files FDA Fast Track Approval Application For Cannabis Multiple Sclerosis Drug [press release]. Reston, VA: MMJ International Holdings; Published February 13, 2019. markets.businessinsider.com/news/stocks/mmj-files-fda-fast-track-approval-application-for-cannabis-multiple-sclerosis-drug-1027948379. Accessed February 13, 2019.

2. FDA Awards MMJ International Holdings Orphan Drug Designation for Huntington’s Cannabis Trial [press release]. Reston, VA: MMJ International Holdings; Published February 13, 2019. mmjih.com/fda-awards-mmj-international-holdings-orphan-drug-designation-for-huntingtons-cannabis-trial. Accessed February 13, 2019.