In collaboration with an international partner, Respiratorius has conducted a large number of interviews (N=135) with treating physicians and key opinion leaders (KOLs) in the U.S., Europe, and Japan. The results indicate that VAL001 is perceived as an attractive treatment option, demonstrating favorable clinical efficacy, with a well-known and established safety profile. A significant proportion, more than 70%, of the physicians and KOLs stated they would be willing to prescribe the drug.

The findings have formed the basis for a comprehensive commercial analysis indicating that VAL001 is expected to achieve strong profitability shortly after a potential marketing authorization. A base-case scenario projects annual sales of USD 190 million at estimated market penetration, with cumulative revenue of USD 2.3 billion over the period of market exclusivity.

A European patent for the formulation of VAL001 was recently granted. Similar patents have already been approved in the U.S., Japan, and Canada, ensuring protection of the formulation and its intended use across all key markets-particularly in the U.S.

VAL001 is an innovative oral formulation of sodium valproate, developed to enhance efficacy with a low risk of side effects when used as pre-treatment prior to standard cancer therapy, primarily Diffuse Large B-cell Lymphoma (DLBC). Preclinical data suggest that VAL001 sensitize tumor cells to chemotherapy, potentially improving treatment outcomes across several cancer indications.

The formulation has been evaluated in a pharmacokinetic study showing 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.
 

"We are very pleased that the FDA has granted us a meeting, allowing for direct dialogue with the agency rather than written correspondence only," says 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 with the FDA represents a natural next step following the positive results previously communicated. The company looks forward to continuing development in line with its established strategy to enable broader clinical access to the drug candidate.