Dose-ranging, PK, Safety, Efficacy Study of Osanetant in Patients With Moderate/Severe VMS Associated With Menopause

A Phase 2A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy, Safety and Pharmacokinetics (PK) of ACER-801 for Treatment of Moderate to Severe Vasomotor Symptoms (VMS) Associated With Menopause

In this clinical research study, subjects will be given the study drug, ACER-801 (osanetant) or placebo (looks like the study drug but contains no active ingredients). The study drug works on a receptor in the brain and the intended purpose is for the study treatment of moderate to severe Vasomotor Symptoms (VMS) also referred to as hot flashes or flushes associated with menopause. Hot flashes are a change in your temperature that occurs due to changes in your hormones.

This is a Phase 2a, randomized, double-blind, placebo-controlled, dose-ranging study in post menopausal women in which the pharmacokinetics, safety and efficacy of ACER-801 (osanetant 50 mg twice daily [BID], 100 mg BID, and 200 mg BID) will be compared to placebo.

Subjects will enter a Screening Period to determine eligibility. Subjects will be required to complete hot flash diaries for 2 weeks prior to randomization. Eligible subjects will be admitted to a Clinical Research Unit and remain in the clinic for 14 days after completion of treatment and all study assessments. The study includes a 14 day safety follow-up assessment. Subjects will be randomized in a 1:1:1:1 ratio.

Key Inclusion Criteria:

1. Post-menopausal female subjects 40-65 years of age, inclusive.

   Menopause will be defined as:

   1. At least 12 months of spontaneous, continuous amenorrhea, or
   2. At least 6 months of spontaneous, continuous amenorrhea with serum follicle stimulating hormone (FSH) levels > 40 mIU/mL at screening, or
   3. At least 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy.

2. At baseline women:

   1. With an average number of moderate to severe hot flashes/day for 2 weeks prior to randomization (per continuous hot flash diary).

   2. That have a change of < 50% in average 24-hour hot flash frequency 2 weeks prior to randomization.

      • Moderate: defined as sensation of heat with sweating, able to continue activity.
      • Severe: defined as sensation of heat with sweating, causing cessation of activity.

Key Exclusion Criteria:

1. Any active comorbid disease deemed by the investigator to be clinically significant, which could impact safety during study conduct including renal or hepatic impairment.
2. Use of any prohibited medications.
3. Body mass index (BMI) >35 kg/m2.
4. Any active ongoing condition that could cause difficulty in interpreting vasomotor symptoms.
5. Inability to complete questionnaires and continuous hot flash diary for any reason.
6. Subjects who, in the opinion of the investigator, should not participate in the study for any other reason.