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FDA Grants Orphan Drug Designation to DiagnaMed's Molecular Hydrogen Therapy for ALS

• DiagnaMed Holdings Corp. has received FDA Orphan Drug Designation for molecular hydrogen as a potential treatment for ALS, offering key development incentives including seven years of market exclusivity.

• The company has entered into a letter of intent with Revive Therapeutics Ltd. to transfer full rights of its molecular hydrogen intellectual property for ALS treatment, with the acquisition expected to close by March 31, 2025.

• Molecular hydrogen's antioxidant and anti-inflammatory properties show promise in addressing oxidative stress and inflammation in ALS, a devastating neurodegenerative disease affecting 50,000 patients in the U.S. and Europe.

DiagnaMed Holdings Corp. has achieved a significant milestone in its pursuit of novel treatments for rare diseases, securing Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration for molecular hydrogen in treating amyotrophic lateral sclerosis (ALS). This designation marks a crucial step forward in addressing the urgent needs of patients suffering from this devastating neurodegenerative condition.

Disease Impact and Current Therapeutic Landscape

ALS presents as a progressive neuromuscular disease that targets nerve cells controlling voluntary muscle movement. The condition invariably leads to paralysis and respiratory failure, with patients facing a stark life expectancy of just two to six years post-diagnosis. Currently affecting approximately 50,000 individuals across the United States and Europe, with over 5,000 new diagnoses annually, ALS represents a significant unmet medical need with no known cure.

Molecular Hydrogen's Therapeutic Potential

The therapeutic approach centers on molecular hydrogen's unique properties as a small molecule possessing both antioxidant and anti-inflammatory capabilities. Preclinical studies have demonstrated promising results in addressing two critical factors in ALS progression: oxidative stress and inflammation. This novel mechanism of action positions molecular hydrogen as a potentially valuable addition to the limited treatment options currently available for ALS patients.

Strategic Partnership and Commercialization Plans

In a parallel development, DiagnaMed has entered into a non-binding letter of intent with Revive Therapeutics Ltd. dated February 28, 2025. The agreement outlines plans for Revive to acquire full rights to DiagnaMed's intellectual property related to molecular hydrogen for ALS treatment. The acquisition is expected to conclude by March 31, 2025, subject to due diligence and final agreement terms.

Regulatory Benefits and Market Implications

The FDA's Orphan Drug Designation brings substantial benefits to DiagnaMed's development program, including:
  • Tax credits for clinical trial expenses
  • Exemption from certain FDA fees
  • Potential seven-year market exclusivity upon approval
Fabio Chianelli, Chairman and CEO of DiagnaMed, emphasized the significance of this development: "Receiving orphan drug designation from the FDA is a significant milestone for DiagnaMed and validates the promise of molecular hydrogen as a potential treatment for ALS. We are excited to partner with Revive Therapeutics to advance this program and meet the urgent needs of the ALS community."
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