Vitalis is planning to open a pivotal clinical trial into its new formulation of fumarate, called VTS-72, that has shown promise in easing flushing — a common and troublesome side-effect of Tecfidera (dimethyl fumarate), an oral treatment for relapsing-remitting multiple sclerosis (RRMS).
The company announced plans to launch a pivotal study of VTS-72 in 2020, and use its data to help support a New Drug Application (NDA), requesting approval, which it plans to file with the U.S. Food and Drug Administration (FDA) later that year.
If approved, it hopes to market its lead treatment candidate sometime in 2021.
VTS-72 consists of a new formulation of fumarate combined with VTS-Aspirin, a Vitalis-owned platform based on aspirin. The therapy is being investigated as an alternative for RRMS patients who experience flushing as a side effect of Tecfidera treatment.
Flush is characterized by feelings of warmth and a rapid reddening of the skin, sometimes accompanied by spots or solid patches of redness, and is often painful. The intensity, frequency, and unpredictability of flush can be a burden on patients’ quality of life, and affect how well they comply with medications.
Tecfidera pills, whose active substance is fumaric acid or fumarate, is one of the most prescribed treatments for RRMS. Marketed by Biogen, it is known to effectively reduce MS flares and slow disease progression, but a downside is side effects associated with its use.
Along with gastrointestinal problems, flush is also commonly reported and estimated to affect 40% of the people taking Tecfidera.
Aspirin has long been used to reduce fumarate flush, but it only works if taken at least 30 minutes in advance — something patients can forget to do.
By combining VTS-Aspirin with fumarates, VTS-72 is believed to enhance the pharmacokinetics of fumarate (its absorption, bioavailability, distribution, metabolism, and elimination in the body) while reducing flushing.
A pilot study in 18 healthy subjects found that VTS-72 led to a mean reduction of 63.3% in flush compared with standard fumarate therapy, Vitalis reports on its website. In addition, 39% of the people who had flush with fumarate were said to be completely free of flush after taking VTS-72.
Based on these results, Vitalis asked for a Type-C meeting with the FDA and, if the outcome is positive, expects to open the pivotal study in MS patients.
Vitalis reports that VTS-72 has been designated an orphan drug by the FDA as a potential treatment for people with MS who experience fumarate flush.
“The fumarate flush is not only prevalent but its impact on patient quality of life and compliance with treatment is often significantly underestimated, exacting both a physical and psychological toll on the patient,” Joseph Habboushe, MD, founder of Vitalis and inventor of the VTS platform, said in the press release.
“Flush is also a common side effect of other therapies … and is known to lead to treatment noncompliance, a high discontinuation rate, and could represent a barrier to initiating patients to fumarate therapy,” Habboushe added. “Through this propriety combination, we believe that VTS-72 can meaningfully reduce flush while improving the pharmacokinetics of the fumarate, resulting in a potentially significant advancement in the treatment of MS.”