Toleranzia advances its projects despite challenges linked to Covid-19
Toleranzia, that develops potentially curative treatments for severe autoimmune diseases, is, like many other companies, affected by the Covid-19 pandemic. The company recently announced that the planned clinical trial of its drug candidate TOL2 will be postponed until the second half of 2023, due to the fact that the unabated global need for Covid-19 vaccine in the world is causing an extensive shortage of materials needed to purify Toleranzia’s drug candidate. BioStock contacted Toleranzia’s CEO Charlotte Fribert to find out how the company is affected by the delay.
Biotech company Toleranzia is developing TOL2 for the rare autoimmune disease myasthenia gravis (MG), a disease for which there are no effective treatments, and the medical need is substantial. In addition, the company portfolio includes TOL3 directed at another rare autoimmune disease, ANCA vasculitis, which also lacks effective treatment options.
During 2021, Toleranzia has brought TOL2 closer to clinical development, not least through important advances regarding the candidate’s manufacturing process. Moreover, the company´s financial position was recently strengthened through new major shareholders, e.g. the renowned life science investors Flerie Invest and Nordic Tender.
CEO comments on the delay
Despite the fact that the internal processes have proceeded according to, or sometimes exceeded, expectations, the worldwide pandemic has brought its challenges. Yesterday, the company communicated new timelines for the upcoming clinical study with TOL2, the first study in humans with the candidate. BioStock contacted CEO Charlotte Fribert for a comment.
Can you, in more detail, describe what is causing the delay?
– The production of TOL2 and many other biologic drug compounds relies on materials and equipment that are also used in the production of Covid-19 vaccines. In order to address the global health threat from the pandemic, priority is obviously given to the supply of these to vaccine manufacturing companies. This has led to material shortages and production delays for pharmaceutical manufacturers around the world, and our production partner, 3P Biopharmaceuticals, is no exception. Without the necessary materials, we cannot produce purified TOL2 for our clinical trial and preliminary toxicology programme as originally planned.
What does the delayed start of studies mean for Toleranzia?
– It is a natural consequence of the current pandemic that we will have some delay in the start of the first patient study with TOL2, but our drug concept to cure severe autoimmune diseases is as strong as ever. We are now working to be well prepared for study start, including optimising the toxicology programme to be performed before the clinical trial can start. We have a very good dialogue with the regulatory authorities on the safety parameters to be evaluated and the duration of the studies. In addition, we are working, in collaboration with regulatory authorities and our clinical advisors, to further optimise the design of the first clinical trial in patients with myasthenia gravis.
How far along are you in planning the first patient study of TOL2?
– As I mentioned, we have a strong support from the Swedish Medical Products Agency and our external clinical advisors. A positive effect of moving the start of the study forward is that we will have additional valuable time to analyse which biomarkers to study to best evaluate the clinical efficacy of the treatment and which study centres to include to maximise efficiency in patient recruitment, as well as to apply an adaptive study design to deliver high quality data.
Is production development completely halted until the supply of necessary materials resumes?
– No, not at all. We now have more time to evaluate new opportunities to improve the cost efficiency of the whole production process, which is of course very valuable, especially in the longer term. It is also important to underscore that our production partner has made significant progress and established the cultivation of TOL2 on an industrial 1,000 litre scale. The cultivation process has proven to be stably scalable, highly robust and fully usable as a starting point for future industrial production.
How does the lack of materials for the pure production of TOL2 affect your other pharmaceutical project, TOL3?
– The TOL3 project, which aims to develop a new treatment for the rare autoimmune disease ANCA vasculitis, is not affected at all. We are now establishing our own lab-scale manufacturing for performing studies in preclinical disease models, aimed at establishing Proof-of-Principle for the drug candidate. For the lab-scale manufacturing process, we have secured the necessary materials since a long time and the work is proceeding according to plan. We look forward to the first results in our preclinical disease model, which will guide the way forward to clinical trials.
