MT-7117-E01

6 evidence topics · 2 sources

Objective

Purpose of the first-in-human study

“In this double-blind, placebo-controlled phase 1 study, the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending oral doses of dersimelagon in healthy participants were evaluated.” (opens the source at this quote in a new tab)

Location and study date

No evidence found.

Study design

Single and multiple ascending dose design

“In this double-blind, placebo-controlled phase 1 study, the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending oral doses of dersimelagon in healthy participants were evaluated.” (opens the source at this quote in a new tab)

Eligibility criteria

No evidence found.

Treatment

Doses administered

“No treatment-related effects on melanin density (MD) were observed following single doses of dersimelagon; however, after multiple doses, increases in MD were observed in participants receiving 150 and 300 mg dersimelagon.” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using melanin density as a pharmacodynamic endpoint

Melanin density as the pharmacodynamic measure of target engagement

“Treatment-related increases in MD warrant further investigation in a larger study population and in patients with EPP and XLP.” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

No evidence found.

Description of analysis sets

No evidence found.

Results

Participant disposition

No evidence found.

Baseline characteristics

No evidence found.

Efficacy results

Not applicable.

Health-related quality of life and patient-reported outcomes

Not applicable.

Safety results

Treatment-emergent adverse events in healthy participants

“Dersimelagon was generally well tolerated in healthy participants, with the most common TEAEs being lentigo (52.8%) and skin hyperpigmentation (50.0%) after multiple doses.” (opens the source at this quote in a new tab)
“Overall, the incidence of TEAEs was relatively low, with most events occurring during Part A (SAD) and Part E (MAD) and the majority being mild to moderate in severity. No deaths or serious AEs were reported during any part of the study.” (opens the source at this quote in a new tab)

Study limitations

Summary: limitations of the first-in-human study

The study enrolled healthy participants rather than people with either disorder, so it does not describe clinical efficacy. Melanin density is a pharmacodynamic measure of target engagement and not a clinical outcome. The study was not designed to detect uncommon adverse events, and the transaminase elevations observed over 14 days of dosing were the basis for the intensified liver monitoring built into the later trials.