TORONTO - Cardiol Therapeutics Inc. (NASDAQ: NASDAQ:CRDL) (TSX: CRDL), a clinical-stage life sciences company, announced that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to its lead drug candidate, CardiolRx™, for the treatment of pericarditis, including recurrent cases. The ODD was granted based on pre-clinical and initial clinical data from the company's ongoing MAvERIC-Pilot Phase II study.
The FDA's ODD is aimed at encouraging the development of treatments for rare diseases—those affecting fewer than 200,000 people in the U.S. The designation comes with benefits such as potential seven-year marketing exclusivity, fee waivers, and tax credits for clinical trials. Moreover, drugs with ODD can potentially be fast-tracked for regulatory review.
CardiolRx™ is currently being evaluated in the MAvERIC-Pilot study (NCT05494788), which is an open-label pilot Phase II trial. It focuses on the tolerance, safety, and efficacy of the drug in patients with recurrent pericarditis, a condition characterized by inflammation of the pericardium, the sac surrounding the heart. The primary efficacy endpoint of the study is the change in patient-reported pericarditis pain over eight weeks, measured by an 11-point numeric rating scale. Secondary endpoints include the pain score after 26 weeks and changes in levels of C-reactive protein, an inflammation marker.
Recurrent pericarditis can lead to severe chest pain, shortness of breath, and fatigue, significantly affecting the quality of life and leading to frequent emergency department visits and hospitalizations. The current FDA-approved therapy for this condition, introduced in 2021, is often used as a third-line treatment due to its high cost.
Cardiol Therapeutics is also engaged in the development of a subcutaneous formulation of cannabidiol for heart failure treatment, a leading cause of death and hospitalization with substantial healthcare costs.
The company's focus remains on researching and developing anti-inflammatory and anti-fibrotic therapies for heart disease treatment.
The information reported is based on a press release statement.
InvestingPro Insights
As Cardiol Therapeutics Inc. (NASDAQ: CRDL) (TSX: CRDL) progresses with its clinical trials and receives Orphan Drug Designation from the FDA, investors may be closely watching the company's financial health and market performance. According to InvestingPro data, Cardiol Therapeutics has a market capitalization of $68.4 million USD and an adjusted P/E ratio for the last twelve months as of Q3 2023 at -3.31, reflecting the company's current pre-profit stage. Despite the challenges, analysts are optimistic, forecasting net income growth and predicting profitability this year.
InvestingPro Tips reveal that Cardiol Therapeutics holds more cash than debt on its balance sheet, which could provide financial flexibility as they continue their research and development efforts. Additionally, the company's liquid assets exceed its short-term obligations, suggesting a degree of financial stability in the near term. However, it's important to note that the company has been quickly burning through cash, which could impact its operations if not managed effectively.
From a market performance perspective, Cardiol Therapeutics has experienced a strong return over the last three months, with a 23.81% price total return, and an even more impressive 82.46% return over the last year. This performance may indicate investor confidence in the company's potential for its lead drug candidate, CardiolRx™, and its other projects.
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