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PF-06823859 low

Phase 2

Dermatomyositis | Small molecule | Other |Pfizer, Inc.|Last Updated: Sep 14, 2023

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment75
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03181893A Study In Adults With Moderate To Severe DermatomyositisPHASE2 COMPLETED 75Jan 23, 2018Nov 28, 2022Sep 14, 202341 United States, Germany +3
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Study Endpoints
Primary Endpoints
Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 12 (Stage 1, Stage 2 and Amended Stage 2)
Baseline and Week 12

The treatment effect was defined as the difference (mean chg from baseline at Week12 in the active treatment group minus that in the placebo group) in the mean change of CDASI activity score from baseline at Week 12. The score (range: 0-100) consists of the extent score (ES), Gottorn hands score (GHS), peringual score (PS) and allopecia score (AS). ES (range: 0-90) was obtained by summing up scores for the total erythema (ER \[0-45\], redness of the skin or mucous membranes), scaling (SC \[0-30\], peeling of the skin) and erosion/ulceration (EU \[0-15\], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity.

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3)
Up to Week 40

Adverse event (AE) was any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was any untoward medical occurrence that at any dose resulted in any of following outcomes/deemed significant for any other reason: death; initial /prolonged inpatient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly/birth defect and suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. AEs included both serious (if occurred) and all non-serious adverse events. TEAEs are events between first dose of study drug and up to Week 40 that were absent before treatment or that worsened relative to pretreatment state.

Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3)
Up to Week 40

Hemoglobin(HGB),hematocrit,erythrocytes(ery.),HDL cholesterol(chl.)\<0.8\*lower limit of normal(LLN);reticulocytes (ret.), ret./ery.(%)\<0.5\*LLN,\>1.5\*upper limit of normal (ULN);ery. mean corpuscular(EMC) volume,EMC HGB concentration,potassium,chloride,calcium,bicarbonate\<0.9\*LLN,\>1.1\*ULN;platelets\<0.5\*LLN,\>1.75\*ULN; leukocytes(leu.),glucose\<0.6\*LLN,\>1.5\*ULN;lymphocytes(lym.), lym./leu.(%),neutrophils (neu.), neu./leu.(%), protein,albumin\<0.8\*LLN,\>1.2\*ULN;basophils(bas.), bas./leu.(%), eosinophils(eos.), eos./leu., monocytes(mon.), mon./leu.(%), urate\>1.2\*ULN;bilirubin (total, direct,indirect)\>1.5\*ULN;aspartate/alanine aminotransferase,gamma glutamyl transferase,lactate dehydrogenase,alkaline phosphatase\>3.0\*ULN;urea nitrogen,creatinine,triglycerides, chl.\>1.3\*ULN; sodium \<0.95\*LLN,\>1.05\*ULN; creatine kinase \>2.0\*ULN;Urine: pH\<4.5,\>8;glucose, ketones,protein, HGB,urobilinogen,bilirubin,nitrite,leukocyte esterase\>=1;ery., leu.\>= 20;hyaline casts\>1;bacteria\>20.

Number of Participants With Vital Sign Abnormalities (Stage 3)
Baseline up to Week 40

Abnormality in vital signs: Sitting pulse rate \<40 beats per minute (bpm) to \>120 bpm, sitting diastolic blood pressure (DBP) \< 50 millimeter of mercury (mmHg), sitting systolic blood pressure (SBP) \<90 mmHg.

Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3)
Baseline up to Week 40

ECG abnormalities criteria included: 1) QTc interval adjusted according to Fridericia formula (QTcF) (msec): \>450, \>480, \>500, increase from baseline \>=30, increase from baseline \>=60; 2) Pulse rate (PR) (msec): \>=300, change from baseline (Chg) \>=25% or 50%; 3) QT (msec): \>=500; 4) QRS (msec): \>=200, Chg \>=25% or 50%. Categories, with at least 1 participant having ECG abnormality in any of the reporting arms, were reported in this outcome measure.

Secondary Endpoints
Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2)
Up to Week 28
Number of Participants With TEAEs and SAEs (Amended Stage 2)
Up to Week 40
Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2)
Up to Week 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Placebo ARMPLACEBO_COMPARATOR -
PF-06823859 ARM highEXPERIMENTAL -
PF-06823859 ARM lowEXPERIMENTAL -
Interventions
NameTypeDescription
PF-06823859 lowDRUGA humanized immunoglobulin neutralizing antibody
Placebo ArmDRUGPlacebo contains histidine, sucrose, PS80, ethylene diamine, and triacetic acid
PF-06823859 highDRUGA humanized immunoglobulin neutralizing antibody
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites41

Inclusion Criteria for Patients with Skin Predominant Activity: * Must have CDASI Activity score of greater than or equal to 14, and have failed at least 1 standard of care systemic treatment, (eg, corticosteroids). * Confirmation of DM by the investigator and two of the following: 1. Gottron's ...

Countries:United StatesGermanyHungaryPolandSpain
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