MMJ International Holdings has developed MMJ-001, a pharmaceutical-grade cannabis drug that represents a historic first in plant-based cannabis medicine for treating chorea associated with Huntington's Disease. The investigational new drug (IND) contains a precisely balanced combination of natural THC and CBD extracted from proprietary cannabis strains and formulated in soft gelatin capsules under FDA standards.
The biopharmaceutical company, led by CEO Duane Boise, has taken a strictly pharmaceutical approach to cannabis drug development, distinguishing itself from the wellness and recreational markets. "We're not interested in chasing the wellness trends or the recreational market," says Boise. "This is about providing real medicine to real patients-starting with one of the most vulnerable populations: those suffering from Huntington's Disease."
FDA Orphan Drug Designation Provides Regulatory Advantages
MMJ-001 has received Orphan Drug Designation from the U.S. Food and Drug Administration, a rare status granted to treatments targeting conditions that affect fewer than 200,000 Americans. This designation provides significant benefits including seven years of market exclusivity, tax credits, waived FDA fees, and access to accelerated review pathways.
"Orphan status is not just a regulatory milestone-it's a lifeline," explains Boise. "It shows the FDA recognizes the urgent unmet medical need and our serious commitment to bringing a therapeutic option to these patients."
Natural Cannabis Extracts Differentiate from Synthetic Alternatives
Unlike synthetic single-molecule THC products like Marinol (dronabinol) or nabiximols, which failed its Phase III trial in the U.S., MMJ-001 preserves the full therapeutic potential of the cannabis plant. The drug is manufactured using natural full-spectrum extracts from MMJ's proprietary cultivars, grown specifically for pharmaceutical use, with each softgel standardized to ensure consistent dosing and bioavailability.
Dr. Elio Mariani, the lead pharmaceutical chemist overseeing the formulation process, emphasizes the complexity of working with natural cannabis compounds. "You cannot recreate the complexity of the cannabis plant in a lab," says Dr. Mariani. "We're not chasing isolated molecules-we're working with the entourage effect of nature's own design, under GMP conditions."
Clinical Development Progress and FDA Collaboration
MMJ-001 is currently progressing toward Phase II FDA-sanctioned human trials after successful preclinical investigations and collaborative input from top neurologists at a leading U.S. research university specializing in Huntington's Disease. The company has worked closely with the FDA throughout its development, submitting its Investigational New Drug application and receiving validation for its pharmaceutical-grade approach.
Dr. Mariani, who oversees a team of chemists at a secure facility licensed by the DEA and registered with the FDA, describes the rigorous manufacturing standards required. "This isn't just about blending oils," he says. "We're developing a pharmaceutical product-meeting rigorous standards for stability, purity, and reproducibility. Our work is governed by science, not hype."
Market Potential in Pharmaceutical Cannabis
MMJ International Holdings joins a small but elite group of companies pursuing true pharmaceutical cannabis, a field with few successes but significant upside potential. GW Pharmaceuticals established precedent with Epidiolex, a plant-derived CBD formulation approved by the FDA for seizure disorders, which reached nearly $1 billion in annual sales before GW Pharmaceuticals was acquired by Jazz Pharmaceuticals for $7.2 billion.
Boise sees similar potential for MMJ-001: "With an addressable patient population in the tens of thousands, no existing cannabis-based therapy approved for chorea in Huntington's, and a patent-pending formulation, MMJ-001 is poised to fill a critical gap in neurodegenerative care."
Strategic Focus on FDA Compliance
The company's strategy has centered on FDA compliance from inception, distinguishing it from the supplement market where companies like Charlotte's Web faced market saturation and regulatory challenges. "We're not trying to be the next dispensary brand," says Boise. "We are here to meet the FDA's gold standard for drug development-and bring real relief to patients who have been ignored by Big Pharma for far too long."
With Phase II trials on the horizon and international partners already secured for future production and distribution, MMJ-001 represents what the company positions as a signal that botanical cannabis-based pharmaceuticals are both viable and essential for addressing unmet medical needs in neurodegenerative diseases.
