Patient Recruitment Completed for Phase 2 Study of Mucopolysaccharidosis VI Drug Candidate

According to a press release from the French biotechnology company Inventiva, the company has completed recruitment for a phase 2 clinical trial evaluating its experimental mucopolysaccharidosis VI drug, odiparcil.

About Mucopolysaccharidosis

Mucopolysaccharidosis (MPS) is a group of genetic conditions characterized by the body’s inability to break down glycosaminoglycans (GAGs) — long chains of complex sugar molecules that are mostly found on the surface of cells. These sugars are used by the body to create skin, cartilage, and other tissues — in fact, all human tissues have some amount of GAGs as a normal part of their structure.

In individuals with MPS, however, GAGs build up in cells throughout the body, contributing to a number of serious health problems. Depending on the particular genetic origins, symptoms, and their extent, cases of MPS are categorized into seven subtypes (numbered 1 through 9, excluding 5 and 8 — which are no longer recognized as valid conditions).

MPS VI (or Maroteaux-Lamy syndrome) is characterized by the progressive enlargement and scarring of tissues and major organs. Skeletal abnormalities are also common in this subtype of MPS.

Because MPS is caused by the buildup of GAGs over time, individuals with the condition don’t display any characteristic features at birth. Symptoms typically appear in early childhood. Life expectancy for the condition can vary considerably between cases — if untreated, however, severe MPS VI is often fatal by adolescence.

Recruitment Complete for Phase 2 Trial

Twenty individuals have been recruited for the phase 2 odiparcil trial. Though a small number of participants, Inventiva believes the number is sufficient to evaluate odiparcil’s safety profile — one of the key aspirations of a phase 2 study.

The individuals will be split into two research arms. Fifteen of the individuals will receive standard enzyme replacement therapy (ERT) along with odiparcil or a placebo. The other five participants will form an “open label” cohort — receiving only a large dose of odiparcil and no enzyme replacement therapy. The results of the double-blind arm will likely be published by the end of the year; the results of the open label arm by Q1 2020.

MPS VI is still considered an orphan disease. Apart from the standard enzyme replacement therapy, there is little currently available for its treatment.

Even if odiparcil is found to be safe and effective, FDA approval could be years away at earliest. Why do you think it’s important to announce when trial recruitment periods have closed? Share your thoughts with Patient Worthy!

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