In short
EXPD-101 is an investigational medicine being studied for chronic obstructive pulmonary disease (COPD) exacerbations. A Phase 2 trial with 600 participants is currently authorised to assess whether EXPD-101 can reduce the annualized rate of moderate and severe COPD exacerbations over 52 weeks of treatment. The trial is randomised, double-blind, and placebo-controlled, comparing two doses of EXPD-101 against placebo.
Key points
- EXPD-101 is an investigational medicine studied in a Phase 2 trial for COPD exacerbations.
- The trial is randomised, double-blind, and placebo-controlled, comparing two doses of EXPD-101 against placebo.
- 600 participants are planned for enrolment in this 52-week study.
- The primary outcome measures the annualized rate of moderate and severe COPD exacerbations.
- Safety is assessed through adverse events, vital signs, ECGs, and laboratory evaluations.
- The trial is currently authorised in the EU Clinical Trials Information System.
What is EXPD-101?
EXPD-101 is an investigational medicine that has not yet received marketing authorisation. The full name of the substance is EXPD-101, and it is also known by the development code DPX-101. According to the AdisInsight drug profile, EXPD-101 is a small molecule, meaning it is a chemically synthesised substance. It is classified as a new molecular entity, which means it is a substance that has not previously been authorised as a medicine. The substance was originated by Damona Pharmaceuticals.
How does EXPD-101 work?
EXPD-101 is described as a GABA A alpha 5 receptor modulator. GABA A receptors are proteins found on the surface of nerve cells in the brain. When the neurotransmitter GABA binds to these receptors, it reduces the activity of the nerve cell. The alpha 5 subtype of the GABA A receptor is found mainly in certain regions of the brain involved in learning and memory. A substance that modulates this receptor may influence the activity of these brain regions. In preclinical research, EXPD-101 has been studied in conditions affecting the brain, including Alzheimer's disease, cognition disorders, and schizophrenia. The drug profile lists EXPD-101 under the classes of antidementias, antipsychotics, and nootropics, which are substances that may affect cognitive function.
Which conditions is it used or studied for?
EXPD-101 is not yet authorised for any condition. It has been studied in different disease areas at different stages of development:
- Chronic obstructive pulmonary disease (COPD) exacerbations — In the current Phase 2 trial, EXPD-101 is being studied to see whether it can reduce the number of moderate and severe flare-ups of COPD over one year.
- Alzheimer's disease — Preclinical research in Canada has investigated EXPD-101 in Alzheimer's disease, prior to March 2026.
- Cognition disorders — The drug profile lists cognition disorders among the conditions studied in preclinical development.
- Schizophrenia — Schizophrenia is also listed among the conditions investigated in preclinical research.
How is EXPD-101 given?
In the Phase 2 COPD trial, EXPD-101 is given by mouth (oral use). The trial tests two doses of EXPD-101, one of which is reported as 40 mg. The pharmaceutical form used in the trial is not specified in the registry record. The AdisInsight profile indicates that in earlier preclinical work in Alzheimer's disease, EXPD-101 was also given by mouth.
What do the clinical trials show?
The clinical development of EXPD-101 in COPD is at an early stage. One Phase 2 trial is currently authorised in the EU Clinical Trials Information System. The trial is a randomised, double-blind, placebo-controlled study with a parallel arm design, meaning participants are assigned by chance to receive either EXPD-101 or a placebo, and neither the participants nor the researchers know which treatment is being given.
The trial plans to enrol 600 participants with COPD. Two doses of EXPD-101 are being compared with placebo over a treatment period of 52 weeks. The main measure of effectiveness is the annualized rate of moderate and severe COPD exacerbations, which means the number of flare-ups of COPD that each participant experiences per year. The trial also measures safety and tolerability by recording adverse events, vital signs, ECGs, physical examinations, and clinical laboratory evaluations. The trial is currently in the authorised status, meaning it has received approval to begin but results are not yet available.
What side effects are known?
No side effect data from clinical trials of EXPD-101 are available yet. The Phase 2 COPD trial will collect information on adverse events, vital signs, ECGs, physical examinations, and clinical laboratory evaluations to assess the safety and tolerability of EXPD-101 compared with placebo. The AdisInsight drug profile does not report side effects from the preclinical research.