When Respiratorius’ CEO Johan Drott sums up 2022, he looks back on a transformative year. Arcede Pharma was spun off, a new formulation of VAL001 was developed and now the company is looking for a partner to advance the project further. BioStock contacted Drott in connection with the release of the Q3 report to learn more.

Lund-based Respiratorius has developed the drug candidate VAL001 for the treatment of diffuse large B-cell lymphoma (DLBCL). VAL001 is intended to be used as a pretreatment to R-CHOP, which is today’s standard treatment of the disease. R-CHOP is a combination of chemotherapy and immunotherapy and the idea is that VAL001, which contains the active ingredient valproate, with its mechanism of action should increase the effect of chemotherapy.

The efficacy data that has been generated so far has been so good that the European Medicines Agency (EMA) has recommended taking VAL001 directly from phase I/IIa to phase III. It has also recommended Respiratorius to only conduct one pivotal study before marketing approval.

Spinn off of Arcede Pharma

With the project having reached the licensing phase, the respiratory part of the business was spun off in the spring of 2022 into its own company, Arcede Pharma. In its new form Respiratorius has been able to fully focus on the last part of the development of VAL001, as well as on finding a partner to advance the project further. The company has developed a new formulation of the drug candidate, where the patient takes two daily doses instead of three. This is a great advantage for the patients considering that the dosage should be done in connection with food intake.

Positive results with new formulation

But that is not all. Respiratorius recently announced results from a pharmacokinetic study in which the new formulation appears to have a higher effect compared to the reference preparation used in previous studies. Valproate inhibits histone deacetylase, which alters and loosens the chromatin structure of DNA. This, in turn, potentiates the effect of the chemotherapy.

An analysis of a biomarker for histone acetylation showed that the expression of the biomarker was higher in the individuals dosed with VAL001 compared to the reference formulation at all measurement points in the study. At most, the expression was 40 percentage points higher when dosing with the new VAL001 formulation, compared to the reference formulation.

BioStock recently invited Respiratorius’ CEO Johan Drott to the studio to tell us more about the results, in an interview that you can see here.

The search for partners continues

The new formulation is a very important component of the VAL001 project. To attract a partner, a clear differentiation is needed compared to generic valproate, a substance that has been used since the 1960s and that is primarily used in the treatment of epilepsy and bipolar disorder. A clear differentiation makes it possible to set an advantageous price for the final product, something that BioStock wrote about in a previous article. It will also enable good protection for the product and last year, Respiratorius was able to deliver some positive news regarding this.

So far, a so-called Notice of Allowance has been received from the Japanese Patent Office regarding VAL001 in the new version. This means that Respiratorius enjoys market exclusivity in the country until 2036. The company states that it has come a long way in the patent review process in several countries and that more approvals can be expected in the future.

The marketing of VAL001 is ongoing, with Respiratorius hiring a specialist in this type of partnering processes. Among other things, the project was discussed with stakeholders at JP Morgan’s annual partnering conference in January.

Comments from the CEO

BioStock contacted Johan Drott to get his thoughts on the past year and on the partnering process that is now underway.

First and foremost Johan, 2022 was a very eventful year. What are your thoughts on the past year?

– Of course, the year has been characterised by conducting and completing the PK study. We did not get all the results before the end of the year that we were aiming for, but at least we could end 2022 in style. The results from the analysis of the biomarker, which shows the histone deacetylating effect of VAL001, came this year, but this meant that 2023 got off to a very good start.

– Administratively, last year was intense with a successful capital raise and spin-off of Arcede Pharma AB.

All eyes are now set on the partnering process. What expectations should a shareholder have, especially regarding the time until an agreement can be reached?

– The most important thing as a shareholder is to know that we are doing our best to get an agreement in place as soon as possible. We focus on the candidates who have already shown interest, but as long as we do not have an agreement in place, we will search further and supplement with additional stakeholders.

You recently presented positive results from your PK study, which indicated, among other things, that the  new formulation can have a greater effect compared to the reference formulation. Could there be a reason for you to study that effect further?

– At present, we have no such plans. We have not changed anything other than improved the release profile of valproate and thus feel comfortable that this is what has given VAL001 its superior histone deacetylating effect, compared to the reference.

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