Experimental drug KZR-616 tested for rare muscle disorders

NCT ID NCT04033926

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 2 trial tested a drug called KZR-616 (zetomipzomib) in 25 adults with active polymyositis or dermatomyositis, two rare diseases that cause muscle inflammation and weakness. Participants received either the drug or a placebo for 16 weeks, then switched to the other treatment for another 16 weeks. The study measured changes in a muscle improvement score to see if the drug helps control symptoms.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

KZR-616 (also called zetomipzomib)

What this could lead to

If it works, this could point toward a new treatment option for people with polymyositis or dermatomyositis, helping control muscle inflammation and weakness.

What could go wrong

This is a small Phase 2 trial with only 25 participants, so results may not apply to everyone. The drug may not show clear benefit over placebo, and side effects are still being studied.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

dermatomyositis muscle tissue disorder musculoskeletal system disorder myositis disease polymyositis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • KZR Research Site

    Beverly Hills, California, 90211, United States

  • KZR Research Site

    Orange, California, 92868, United States

  • KZR Research Site

    Miami, Florida, 33136, United States

  • KZR Research Site

    Atlanta, Georgia, 30322, United States

  • KZR Research Site

    Kansas City, Kansas, 66160, United States

  • KZR Research Site

    Baltimore, Maryland, 21224, United States

  • KZR Research Site

    Ann Arbor, Michigan, 48109, United States

  • KZR Research Site

    Great Neck, New York, 11021, United States

  • KZR Research Site

    Duncansville, Pennsylvania, 16635, United States

  • KZR Research Site

    Pittsburgh, Pennsylvania, 15213, United States

  • KZR Research Site

    Austin, Texas, 78756, United States

  • KZR Research Site

    Henrico, Virginia, 23233, United States

  • KZR Research Site

    Prague, Czechia

  • KZR Research Site

    Göttingen, Germany