T20K in MS
T20K – Lead candidate for multiple sclerosis
T20K is Cyxone’s main candidate as first-line treatment for the autoimmune disease multiple sclerosis (MS). T20K is a unique substance derived from natural plant protein that scientists at the Medical University in Vienna, Austria, and the university clinic in Freiburg, Germany, first showed inhibiting pro-inflammatory cytokines such as IL-2 and effectively reduce clinical symptoms in an animal model for MS after oral administration. No signs of toxicity has been observed at therapeutic dosages.
The development program of T20K is proceeding according to plan and a first study in humans is planned to start in 2018.
About multiple sclerosis
Multiple sclerosis (MS) is a chronic autoimmune disease that attacks the central nerve system (CNS), with a name that literally translates to “scar tissue in multiple areas”. The disease affects the whole brain, spinal cord and the optic nerve, but symptoms varies greatly between individuals, where mild cases may include numbness while severe cases may implicate paralysis, vision loss and balance disorders. Both genes and environmental factors have proven to affect the risk of disease and it is believed to be link between D-vitamin deficiency through sunlight and MS, which could be a possible explanation to why the disease is more prevalent in Western countries, where the disease has shown to increase while the levels of D-vitamin has decreased. Additionally, life style factors, such as smoking, has shown to increase the risk of the disease. Today, approximately 2.5 million people worldwide are affected by MS, and diagnosis occur most often among 20-40-year-olds, where double the number of women are affected compared to men.
There are four different types of MS, with a disease progression that is difficult to predict. Approximately 85% of the affected gets RRMS (relapsing-remitting multiple sclerosis),1 which means that the disease suddenly appears in relapses with symptoms that revert within a few weeks or months, but in most cases permanent disabilities are also developed. Remaining cases of MS are affected by the types SPMS (secondary progressive MS), PPMS (primary progressive MS) and benign MS. SPMS is a phase of MS that more than half of everyone affected by relapse reaches after approximately 15-20 years, which then passes into a slow deterioration but with significantly longer periods of relapses. In the change between RRMS, the relapses are sometimes happening in combination with a creeping deterioration. PPMS means that one does not get any relapses at all, but the affected are constantly progressing worse without recovery. With benign MS, the relapses are continuous at a start, but do not give any visual disabilities even after many years as being affected. More than half of those with benign MS have proven to have cognitive disabilities, depression or a what is called MS-related fatigue.
Cyxone assessment is that the MS-market’s growth is restricted by the drugs’ oftentimes many and severe side effects, that some of the drugs can only treat a portion of MS sufferers (RRMS patients) and that the drugs often must be administrated by injections. In the past 10-year period, several big pharmaceutical companies have developed substances that can be taken as tablets, but their side effects are still a limitation for long-term usage by MS-patients. Several biological drugs with strong pharmacological effects have also been developed, although also these with greater risk of severe side effects.
The trend points toward attempting to identify drugs with few and less severe side effects that can, therefore, be administered over longer periods of time, even to non-RRMS patients, in tablet form. It should be noted that pharmaceutical companies that are established on the MS market are, due to expiring patents for many existing MS drugs, highly focused on finding new products that can replace old products to protect and increase their market share.
Cyxone’s potential market for T20K is considerable and is expected to grow in the coming years. In Sweden, approximately 18 000 people with MS and an estimated 2.5 million people are affected by the disease worldwide. Based on a patient population of approximately 37,000 patients in the U.S. and 52,000 patients in the five largest EU markets (EU5), potential sales of approximately 1 billion US-dollar, annually, are estimated for T20K. The calculation is based on the assumption that the drug is marketed through a larger partner that can take 10-30% of the market, which is according to plan.
A new drug in tablet form that can slow the development of MS, and is at the same time “kind” enough to the body making it possible to be taken throughout a patients’ life, could replace many of the existing drugs that causes a variety of side effects. Cyxone strives to develop such a drug that could come to improve the quality of life for those affected by MS, and in best case, prevent disease progression while at the same time contribute with a drug that causes considerably fewer side effects or risks to develop other life-threatening diseases, which is one of the problems with the existing drugs. Thus, a part of Cyxone’s vision is to develop a candidate that is possible to take not only during acute disease episodes but helps those affected to manage their disease in the long term, providing them with an opportunity to take control of their disease and in extension, their lives.
- Yonar, D. et al. Relapsing-Remitting Multiple Sclerosis diagnosis from cerebrospinal fluids via Fourier transform infrared spectroscopy coupled with multivariate analysis. Nature Scientific Reports 2018;8:1025