At last, it has finally become time to resume communication with your shareholders via newsletters. Cyxone has kept a low profile while we prepared for our clinical studies and now, when we have reached clinical development with both of our candidates, we can finally start sharing more about what is happening and what is planned in the respective studies.
I hope, too, that you, our shareholders, are enjoying the nice summer we have, and I hope to be able to share more news with you soon!
Malmö June 26, 2019
Kjell G. Stenberg
T20K and MS
Completion of the final studies prior to clinical trial
Since the completion of our preclinical program in November 2018, we have carried out a number of essential but perhaps not very “innovative” studies with T20K. This primarily regards regulatory studies that have to be conducted to prove that T20K is chemically correct, stable when stored, as non-toxic as previously noted, that the effect on MS in animal models is real and not a “coincidence”, as well as that T20K can be filled into ampoules and is a stable solution that does not react with other substances in dissolved form. We have also gathered and evaluated all chemical and biological information for the reports that have been the basis of the relevant authorities’ assessment that T20K is “mature” and safe enough to be administrated to humans. In summary, these studies and this information gathering thus created the basis for the application to initiate First-in-Human studies with T20K, which last week also was approved by the relevant authorities.
First-in-Human study design
Last week we finally received the news that our application to initiate a First-in-Human (FiH) study, also called Phase 1, with T20K has been approved. This was, of course, an extremely gratifying decision and thanks to solid preparations we were able to start the study as soon as we had the approval in hand by initiating the recruitment and screening process.
This study is conducted to show that healthy people are not adversely affected by T20K at levels of the drug candidate that are above the doses that we expect will be given to MS patients. The confirmation of the drug candidate’s safety is normal procedure in all FiH studies, but what, on the other hand, distinguishes the studies with T20K from studies from a “regular” drug candidate is that we conduct a so-called infusion study (a slow injection of the drug into a vein) even though we intend to make an oral product. This is because the T20K is so rapidly distributed to the immune regulating organs such as the spleen and the small intestine (as well as the liver and kidney) that it is not possible to measure the free levels of T20K circulating in the blood at all. While this distribution is very positive for the MS patients who in the future will be treated with T20K, it unfortunately also means that we must take a “detour” in the development. This is because a company, in order to claim that it has a safe substance, must be able to show that a certain level of the substance can circulate in the blood without causing any side effects.
In our study, we will test one or two doses of T20K per cohort until we have been able to determine a safe level of T20K in the blood. Since T20K will be administrated orally as an MS drug, future patients will never be exposed to such high levels of free substance in the blood as can be “forced” with an injection directly into the blood. When we switch to developing T20K as an oral treatment, a “phase 1b” study in healthy volunteers will take place with doses that creates significantly less free T20K circulating in the blood.
As previously announced, we have chosen to conduct the FiH study in the Netherlands where we both have a very competent partner in QPS and a regulatory process that is considerably faster than the one in e.g. Sweden, without, for that reason, any increased risk for the male healthy volunteers.
Rabeximod and RA
The preparation of the last few months
In the case of Rabeximod and the Phase 2b study in patients with rheumatoid arthritis (RA), we, together with experts from two clinical research organizations, CROs, have signed an agreement with a very knowledgeable and well-known Clinical Advisor who has supported us in the preparation of an IMPD (Investigational Medical Product Dossier), an IB (” Investigator’s Brochure) and the upcoming Phase 2b study protocol.
In addition to the work with regulatory documents, we have also planned for how and where the study will be carried out “in practice”, how we can guarantee the substance’s durability, a 24-week toxicology study in two animal species and which company should fill capsules with Rabeximod under GMP (Good Manufacturing Practice). We will report back as soon as possible with a more detailed activity and time plan regarding Rabeximod’s development, and with more details about the candidate’s development in the next newsletter.
The company’s development
As Cyxone’s projects now have entered into the clinical development phase, we have faced an increased interest in our projects from pharmaceutical companies and investors. We are currently meeting old and new stakeholders who have indicated an interest in Cyxone’s technology and are updating them with our latest results and plans. Many biotech companies at an early stage have very optimistic plans for how to drive the development. At Cyxone, we have instead chosen to keep a low profile while preparing for our clinical studies and now, when we enter into the clinical phase, we can finally begin to talk about what is happening and what is planned in the respective studies.
With both T20K and Rabeximod now in clinical development, we are also strengthening the expertise within Cyxone, increasing our presence at scientific conferences and around potential buyers, as well as increased our ambitions, with the goal of becoming a successful development company for novel therapeutics in autoimmune diseases.