Last winter, Respiratorius presented positive results from the pharmacokinetic study with the new formulation of VAL001. The company has now announced that it has filed a new patent application regarding the formulation, which further strengthens the protection for the candidate. At the same time, work continues to build a strong case in the partnering process that is currently underway.
Lund-based Respiratorius 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 as a pretreatment for R-CHOP, today’s standard treatment of DLBCL. R-CHOP is a combination of chemotherapy and immunotherapy. The goal of VAL001, which contains the active ingredient valproate, is to increase the effect of this chemotherapy through VAL001’s unique mechanism of action.
Looking for partners to take the project further
The project is backed by strong efficacy data, where VAL001 has demonstrated the ability to reduce the risk of mortality – that is, the risk of dying from DLBCL. This has led the European Medicines Agency (EMA) to recommend that VAL001 go directly from phase I/IIa to phase III, where only one pivotal study needs to be conducted before it can be approved for marketing.
This is where we find Respiratorius. The company is in the middle of the process of finding the right partner to take the project further in development. While that is underway, work continues to build as attractive a development package as possible. One important milestone 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-release and delayed-release 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 resulted in lower peak values of the maximum concentration of valproate and a more even 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 project is the protection of VAL001. In connection with the results of the pharmacokinetic study, Respiratorius announced that it had received the green light from the Japanese Patent Office regarding a patent covering the new formulation of VAL001. Japan is the first country to approve the patent application for VAL001 in the new version and the company expects further approvals in the future.
Respiratorius is now taking the next step in its patent strategy, filing a new and more detailed patent application. Going forward, the company will thus have two patent processes running in parallel. The first covers the intended use of VAL001 in its new version, while the new one is more detailed and based on the results generated in the work with the new formulation. The new application gives exclusive rights on the market for at least 20 years from the priority date, which would mean that VAL001 in the event of a patent approval is protected until 2043.
Model further supports VAL001
In addition, Respiratorius has also carried out a project together with researchers at the University of North Carolina. The university has developed models for prediction of results in phase III studies, where researcher Amy Pomeroy in her work focused on DLBCL. According to the model, there is a high probability that VAL001 would succeed well in a future phase III study. The probability of a positive result is assessed as higher than in all previous studies that was part of the comparison. In an interview with BioStock, which you can see in full here, CEO Johan Drott tells us more about the results and gives an update on the ongoing partnering process.The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.