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ACER-801

Phase 1

Post-menopausal Vasomotor Symptoms | Small molecule | Other |Zevra Therapeutics, Inc.|Last Updated: Aug 7, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment49

FDA Designations

No designations recorded

Clinical trial landscape

ACER-801 · 1 trial · 1 indication

Phase 1 1
NCT05325775Dose-ranging, PK, Safety, Efficacy Study of Osanetant in Patients With Moderate/Severe VMS Associated With MenopausePost-menopausal Vasomotor Symptoms
COMPLETED49 Analytics
PHASE1COMPLETED
Dose-ranging, PK, Safety, Efficacy Study of Osanetant in Patients With Moderate/Severe VMS Associated With Menopause
Post-menopausal Vasomotor SymptomsUnlock trial analytics

Study Endpoints

Primary Endpoints

Peak Plasma Concentration (Cmax) of ACER-801
Day 1

maximum concentration of ACER-801 measured at Day 1 Blood sampling included predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, 10, 12 hrs (prior to evening dose) and 15 hrs post morning dose. Only Cmax (mean, SD) data are available due to abbreviated data analyses.

Peak Plasma Concentration (Cmax) of ACER-801 Metabolite
Day 1

peak concentration of ACER-801 metabolite measured at Day 1 Blood sampling included predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, 10, 12 hrs (prior to evening dose) and 15 hrs post morning dose. Only Cmax (mean, SD) data are available due to abbreviated data analyses.

Time to Reach Maximum Concentration (Tmax) of ACER-801
Day 1

time to reach maximum concentration of ACER-801 measured at Day 1 Blood sampling included predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, 10, 12 hrs (prior to evening dose) and 15 hrs post morning dose. Only Tmax (mean, SD) data are available due to abbreviated data analyses.

Time to Reach Maximum Concentration (Tmax) of ACER-801 Metabolite
Day 1

time to reach maximum concentration of ACER-801 metabolite at Day 1 Blood sampling included predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, 10, 12 hrs (prior to evening dose) and 15 hrs post morning dose. Only Tmax (mean, SD) data are available due to abbreviated data analyses.

Area Under the Concentration Curve From Dosing to the Time of the Last Measured Concentration (AUClast) of ACER-801
Day 1

Area under the concentration time curve (AUC) from the time of dosing (0 hour) to the time of the last quantifiable (positive) concentration (Tlast) of ACER-801, calculated by a combination of linear and logarithmic trapezoidal methods (linear up/log down method) Blood sampling included predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, 10, 12 hrs (prior to evening dose) and 15 hrs post morning dose. Only AUClast (mean, SD) data are available due to abbreviated data analyses.

Area Under the Concentration Curve From Dosing to the Time of the Last Measured Concentration (AUClast) of ACER-801 Metabolite
Day 1

Area under the concentration time curve (AUC) from the time of dosing (0 hour) to the time of the last quantifiable (positive) concentration (Tlast) of ACER-801 metabolite, calculated by a combination of linear and logarithmic trapezoidal methods (linear up/log down method) Blood sampling included predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, 10, 12 hrs (prior to evening dose) and 15 hrs post morning dose. Only AUClast (mean, SD) data are available due to abbreviated data analyses.

Half-life (T1/2) of ACER-801
Day 1

Terminal elimination half-life of ACER-801

Half-life (T1/2) of ACER-801 Metabolite
Day 1

Terminal elimination half-life of ACER-801 metabolite

Number and Percentage of Adverse Events ≥ 5%
2 weeks

An Adverse Event (AE) is defined as any untoward medical occurrence associated with the use of the investigational product in humans, whether or not considered related to investigational product. An AE can be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with any use of the investigational product, without any judgment about causality and irrespective of route of administration, formulation, or dose, including an overdose.

Number and Percentage of Serious Adverse Events (SAE)
2 weeks

An AE is considered "serious" if, in the view of either the investigator or Acer, it results in any of the following outcomes: Death, Is immediately life threatening; Requires in-patient hospitalization or prolongation of existing hospitalization; Results in persistent or significant disability or incapacity; Results in a congenital abnormality or birth defect; Is an important medical event that may jeopardize the subject or may require medical intervention to prevent one of the outcomes listed above.

Number and Percentage of Subjects Who Discontinued From the Study
Over 2 weeks

Discontinuation or withdrawal from the study.

Number of Patients With a Clinically Significant Change From Baseline in Abnormalities Detected During Physical Examination
At Day 14 relative to Baseline

A physician or appropriately qualified delegate conducted a full physical examination at baseline and at Day 14. The investigator decides if findings are considered abnormal at baseline and at Day 14 and whether the change is clinically significant. Only clinically significant changes will be reported.

Accumulation Ratio for Cmax (ARcmax) of ACER-801
Day 14

Cmax (maximum concentration) Accumulation Ratio calculated as Cmax,Day14/ Cmax,Day1 and AUCt,Day14/ AUCt,Day1, where the dosing interval t is 12h.

Accumulation Ratio for AUC (ARauc) of ACER-801
Day 14

AUC (area under the curve) Accumulation Ratio calculated as Cmax,Day14/ Cmax,Day1 and AUCt,Day14/ AUCt,Day1, where the dosing interval t is 12h.

Accumulation Ratio for Cmax (ARcmax) of ACER-801 Metabolite
Day 14

Cmax (maximum concentration) Accumulation Ratio calculated as Cmax,Day14/ Cmax,Day1 and AUCt,Day14/ AUCt,Day1, where the dosing interval t is 12h.

Accumulation Ratio for AUC (ARauc) of ACER-801 Metabolite
Day 14

AUC (area under the curve) Accumulation Ratio calculated as Cmax,Day14/ Cmax,Day1 and AUCt,Day14/ AUCt,Day1, where the dosing interval t is 12h.

Metabolite: Parent Ratio of AUC (MRauc)
Day 1

MR = Metabolite:Parent Ratio calculated as Cm,day14/ Cp,day1 and AUCt,m,day14/ AUCt,p,day1, where m and p are the metabolite and parent drug, respectively, and t is the dosing interval (12 h). AUC = area under the curve

Metabolite:Parent Ratio of Cmax (MRcmax)
Day 1

MR = Metabolite:Parent Ratio calculated as Cm,day14/ Cp,day1 and AUCt,m,day14/ AUCt,p,day1, where m and p are the metabolite and parent drug, respectively, and t is the dosing interval (12 h). ACER-801 (parent); Cmax (maximum concentration)

Area Under the Concentration Curve From t0 to Infinite Time (AUCinf) of ACER-801
Day 1

AUC from the time of dosing (0 hour) extrapolated to infinity, calculated as AUClast + Clast/λz, where Clast was the last quantifiable concentration, and λz was the terminal elimination rate constant Reliability of AUCinf values was contingent upon AUCext and λz Profiles with AUCext ≤20% and a λz which met reporting criteria were considered reliable and AUCinf was reported. For profiles that did not meet these criteria, AUCinf and any parameter requiring AUCinf for computation were not reported.

Area Under the Concentration Curve From t0 to Infinite Time (AUCinf) of ACER-801 Metabolite
Day 1

AUC from the time of dosing (0 hour) extrapolated to infinity, calculated as AUClast + Clast/λz, where Clast was the last quantifiable concentration, and λz was the terminal elimination rate constant Reliability of AUCinf values was contingent upon AUCext and λz Profiles with AUCext ≤20% and a λz which met reporting criteria were considered reliable and AUCinf was reported. For profiles that did not meet these criteria, AUCinf and any parameter requiring AUCinf for computation were not reported.

Number of Subjects With a Clinically Significant Change From Baseline for Clinical Laboratory Evaluations: HEMATOLOGY
Over 2 weeks

Blood samples will be measured for hemoglobin, hematocrit, white blood count with differential (neutrophils, lymphocytes, monocytes, eosinophils, basophils), platelet count, red blood cell count (including mean corpuscular volume, mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration). Only clinically significant changes will be reported.

Number of Subjects With a Clinically Significant Change From Baseline for Clinical Laboratory Evaluations: SERUM CHEMISTRY
2 weeks

Blood samples will be measured for albumin, alkaline phosphatase, total bilirubin, alanine aminotransferase (ALT), aspartate aminotransferase (AST), blood urea nitrogen, calcium, carbon dioxide, chloride, potassium, sodium, total cholesterol, creatinine, gamma glutamyl transferase, glucose, lactate dehydrogenase, phosphorus, total protein, uric acid. Only clinically significant changes will be reported.

Number of Subjects With a Clinically Significant Change From Baseline for Clinical Laboratory Evaluations: COAGULATION
2 weeks

Blood samples will be measured for prothrombin time, partial thromboplastin time, international normalized ratio. Only clinically significant changes will be reported.

Number of Subjects With a Clinically Significant Change From Baseline for Clinical Laboratory Evaluations: URINALYSIS
2 weeks

Urine samples will be measured for pH, specific gravity, protein, glucose, ketones, bilirubin. Only clinically significant changes will be reported.

Number of Subjects With a Clinically Significant Change From Baseline for Clinical Laboratory Evaluations: BONE DENSITY MARKERS
2 weeks

Blood samples not available/collected for testing. Blood samples will be measured for Bone Specific Alkaline Phosphatase (BSAP), osteocalcin, amino terminal propeptide of type 1 collagen (P1NP) and Collagen Type- C-Telopeptide (CTX). Only clinically significant changes will be reported.

Number of Subjects With a Clinically Significant Change From Baseline for Clinical Laboratory Evaluations: HORMONES
2 weeks

Blood samples not available/collected for testing. Blood samples will be measured for catecholamines, vasopressin, gonadotropins, estradiol, testosterone, follicle stimulating hormone (FSH), luteinizing hormone (LH), adrenocorticotropic hormone (ACTH), cortisol, thyroid-stimulating hormone (TSH), T3 (Total and Free), T4 (Total and Free), prolactin, sex hormone binding globulin (SHBG), and insulin. Only clinically significant changes will be reported.

Secondary Endpoints

Change in Frequency of Vasomotor Symptoms (Hot Flashes) From Baseline
At Week 1 relative to Baseline
Change in Frequency Vasomotor Symptoms (Hot Flashes) From Baseline
At Week 2 relative to Baseline
Change in Severity of Vasomotor Symptoms (Hot Flashes) From Baseline
At Week 1 relative to Baseline
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ACER-801 50 mg BIDEXPERIMENTALACER-801 (osanetant) 50 mg BID (1 x 50 mg and 3 x placebo, twice daily)
ACER-801 100 mg BIDEXPERIMENTALACER-801 (osanetant) 100 mg BID (2 x 50 mg and 2 x placebo, twice daily)
ACER-801 200 mg BIDEXPERIMENTALACER-801 (osanetant) 200 mg BID (4 x 50 mg and 0 x placebo, twice daily)
PlaceboPLACEBO_COMPARATORPlacebo (4 x Placebo of ACER-801 twice daily)

Interventions

NameTypeDescription
ACER-801 50 mg BIDDRUG50 mg BID (twice daily)
ACER-801 100 mg BIDDRUG100 mg BID (twice daily)
ACER-801 200 mg BIDDRUG200 mg BID (twice daily)
PlaceboDRUGPlacebo
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Eligibility Criteria

Age Range40 Years to 65 Years
SexFEMALE
Healthy VolunteersNo
Study Sites1

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) ...

Countries:United States
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Frequently asked questions about ACER-801

What is ACER-801 used for?

ACER-801 is an investigational small molecule being developed for post-menopausal vasomotor symptoms, commonly known as hot flashes. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes ACER-801?

ACER-801 is being developed by Zevra Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol ZVRA.

What phase is ACER-801 in?

ACER-801 is in Phase 1 clinical development. It is an investigational drug and has not yet been approved for any use. The Phase 1 trial has been completed.

What clinical trials is ACER-801 in?

ACER-801 has one completed Phase 1 clinical trial, identified as NCT05325775. This was a dose-ranging, pharmacokinetic, safety, and efficacy study in patients with moderate to severe vasomotor symptoms associated with menopause.

Is ACER-801 the same as osanetant?

Yes, ACER-801 is also known as osanetant. The Phase 1 clinical trial for ACER-801 is titled as a study of osanetant in patients with moderate to severe vasomotor symptoms.