Lund-based Respirator has developed the drug candidate VAL001 for the treatment of diffuse large B-cell lymphoma (DLBCL) – one of the most common forms of non-Hodgkin lymphoma. VAL001 has been designed to be used as a pre-treatment for R-CHOP, the current standard treatment for DLBCL. R-CHOP is a combination of chemotherapy and immunotherapy. The aim of VAL001, which contains the active ingredient valproate, is to increase the effectiveness of this chemotherapy through VAL001’s unique mechanism of action.
Looking for partners to take the project forward
The company has strong efficacy data behind it, where VAL001 has been able to demonstrate a significant ability to reduce mortality risk – that is, the risk of dying in DLBCL. This has led to the European Medicines Agency EMA recommended that VAL001 go directly from phase I/IIa to phase III, where only a pivotal study needs to be conducted before it can be approved for marketing.
This is where we are now. Respiratorius is in the middle of a process to find the right partner who can take the project further in development. While that process is underway, work continues to build as attractive a development package as possible. An important milestone in that work was reached last winter when promising results were announced in the pharmacokinetic study with the new formulation of VAL001.
Good results with the new formulation
The new formulation is distinguished by its tailored release profile, which combines immediate and sustained release of sodium valproate. With twice-daily dosing, VAL001 showed the same total plasma concentration of valproate as achieved with the reference formulation dosed three times daily. VAL001 provided lower peak valproate concentrations and a more consistent plasma concentration over time compared to the reference formulation.
In addition to the positive PK profile, analysis of a biomarker for histone acetylation also showed promising results. Biomarker expression was higher at all measured time points and at most 40 percentage points higher at 48 hours after dosing with VAL001 compared to the reference formulation.
Filed a new patent application
Another important part of the work is the protection that VAL001 enjoys. In connection with the results of the pharmacokinetic study, Respiratorius announced that it has received the green light from the Japanese Patent Office for a patent covering the new formulation of VAL001. Japan is the first country to approve the patent application for VAL001 in the new embodiment and the company expects further approvals in the future.
Respiratorius is now taking the next step in its patent strategy and submitting a new, more detailed patent application. The company will therefore have two patent processes running in parallel in the future. The first covers the intended use of VAL001 in its new embodiment, while the new one is more detailed and is based on the results generated in the work with the new formulation. The new application provides exclusive rights on the market for at least 20 years from the priority date, which would mean that VAL001, if a patent is approved, is protected until 2043.
Model provides further support for VAL001
In addition to this, Respiratorius has also carried out a project together with researchers at University of North CarolinaThe university has developed models for predicting results in phase III studies, where the researcher Amy Pomeroy in his work focused on DLBCL. According to the model, there is a high probability that VAL001 will be successful in an upcoming phase III study. The probability of a positive result is considered higher than in all previous studies that were compared. In an interview with BioStock, which you can see in its entirety here, CEO Johan Drott tells more about the results and at the same time provides an update on the ongoing partnering process.