Respiratorius AB has secured a pre-IND meeting with the U.S. Food and Drug Administration (FDA) for its cancer drug candidate VAL001, marking a significant regulatory milestone as the company prepares to enter the strategically important U.S. market. The meeting will provide regulatory guidance for the next steps in clinical development of the innovative oral formulation of sodium valproate.
Strong Physician Support Drives Commercial Confidence
The FDA meeting request follows extensive market research conducted in collaboration with an international partner, involving 135 interviews with treating physicians and key opinion leaders across the U.S., Europe, and Japan. The results demonstrate substantial clinical interest, with more than 70% of physicians and KOLs indicating they would be willing to prescribe VAL001.
According to the survey findings, physicians perceive VAL001 as an attractive treatment option, citing favorable clinical efficacy combined with a well-known and established safety profile. These positive reception metrics have informed a comprehensive commercial analysis projecting strong market potential for the drug candidate.
Significant Revenue Projections Support Development Strategy
The commercial analysis indicates VAL001 is expected to achieve strong profitability shortly after potential marketing authorization. A base-case scenario projects annual sales of USD 190 million at estimated market penetration, with cumulative revenue reaching USD 2.3 billion over the period of market exclusivity.
These projections reflect the unmet medical need in cancer treatment, particularly for Diffuse Large B-cell Lymphoma (DLBCL), where VAL001 is being developed as a pre-treatment to enhance the efficacy of standard cancer therapy.
Comprehensive Patent Protection Across Key Markets
Respiratorius has established robust intellectual property protection for VAL001, with patents approved across all major markets. The company recently received a European patent for the formulation, joining previously approved patents in the U.S., Japan, and Canada. These patents ensure protection of the formulation and its intended use, with market exclusivity extending at least 20 years from the priority date of April 21, 2016.
Novel Formulation Addresses Clinical Challenges
VAL001 represents an innovative approach to cancer pre-treatment, utilizing a tailored release profile that combines immediate-release and extended-release properties of sodium valproate. Preclinical data suggest the formulation can sensitize tumor cells to chemotherapy, potentially improving treatment outcomes across several cancer indications.
The formulation has been evaluated in a pharmacokinetic study demonstrating that VAL001, administered twice daily, achieves systemic exposure levels of valproate equivalent to those seen with the reference product used in the company's completed Phase I/IIa study, which requires dosing three times daily. This improved dosing convenience could enhance patient compliance and treatment outcomes.
"We are very pleased that the FDA has granted us a meeting, allowing for direct dialogue with the agency rather than written correspondence only," said Johan Drott, CEO of Respiratorius. "This marks an important milestone for VAL001 and our efforts to offer a potentially improved first-line treatment for patients with DLBCL."
The pre-IND meeting represents a natural progression following the positive results previously communicated and aligns with the company's established strategy to enable broader clinical access to the drug candidate. The direct dialogue with the FDA will be crucial for determining the regulatory pathway forward as Respiratorius prepares its Investigational New Drug application.
