A Phase 1 clinical trial found T20K, Cyxone’s investigational plant protein-derived treatment for multiple sclerosis (MS), to be safe and well-tolerated in healthy men, and raising the possibility that it might work at low doses, the company announced.
A next step is to develop an oral formulation for the compound, which Cyxone believes has prophylactic — or disease preventing — properties, for further human testing.
Preclinical studies showed that T20K works to lower the production of pro-inflammatory cytokines — signaling molecules that mediate inflammation — such as IL-2, and can effectively ease disease symptoms in animal models of MS.
Results also noted that T20K, administered orally to the animals, had low toxicity and favorable pharmacokinetics (absorption, distribution, metabolism, and excretion in the body), suggesting that it may be a safe and effective therapy at low doses and with a low frequency of administration that might be every two weeks or monthly.
Cyxone believes that T20K could be prescribed to patients immediately or shortly after diagnosis to reduce or prevent MS episodes, and possibly delay disease progression. According to the company, such an early disease intervention would be unique, since MS treatments are largely designed to treat active disease flares.
The double-blind, single center Phase 1 study evaluated the safety, tolerability, and pharmacokinetics of T20K in eight healthy men. It was conducted in the Netherlands, in collaboration with the clinical research organization (CRO) QPS Netherlands.
Volunteers received a first low dose of T20K – 0.005 mg per kilo – intravenously (administered directly into the bloodstream), and researchers assessed the compound’s levels in their blood.
Results showed that T20K was safe and well-tolerated, with no serious adverse events and only one report of a mild and transient headache. Since this initial and very low dose of T20K showed a favorable pharmacokinetics profile, researchers did not need to study a planned second and higher dose, the company said in its release.
“There is always an element of uncertainty when taking a new substance to man despite successful testing in several animal species,” said Kjell G. Stenberg, Cyxone’s CEO.
“To our great delight, the results were far above our expectations already after the first, lowest, dose level reinforcing our conviction of T20K’s exceptional potential,” Stenberg added.