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Anti-Beta Interferon

Phase 2

Dermatomyositis | Small molecule | Other |Pfizer, Inc.|Last Updated: Feb 6, 2025

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05192200An Extension Study for Participants Who Have Completed the Treatment Period of a Qualifying Parent StudyPHASE2 COMPLETED 24Dec 20, 2021Nov 20, 2023Feb 6, 202525 United States, Hungary +2
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
From Day 1 of dosing maximum up to Week 68

An Adverse Event (AE) was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE was considered treatment emergent relative to a given treatment if the event occurred for the first time during the effective duration of treatment and was not seen prior to the start of treatment, or the event was seen prior to the start of treatment but increased in severity during treatment. AEs included both serious adverse events (SAEs) and all non-SAEs.

Number of Participants With Laboratory Abnormalities
From Day 1 of dosing maximum up to Week 68

Hematology laboratory parameters: hemoglobin (grams per deciliter \[g/dL\]); hematocrit (percentage \[%\]); lymphocytes (10\^3 per (/) millimeter\[mm\]\^3); lymphocytes/leukocytes (%); neutrophils (10\^3/mm\^3) less than (\<)0.8\*lower limit of normal (LLN), leukocytes (10\^3/mm\^3) \<0.6\*LLN, neutrophils (10\^3/mm\^3); basophils (10\^3/mm\^3); basophils/leukocytes (%); monocytes/leukocytes (%); activated partial thromboplastin time (seconds \[sec\]); prothrombin time (sec) more than (\>)1.2\*upper limit of normal (ULN). Clinical chemistry: potassium (milliequivalents per liter \[mEq/L\]); bicarbonate (mEq/L) \<0.9\*LLN, creatine kinase (units per liter \[U/L\]) \>2.0\*ULN, glucose (milligram per deciliter \[mg/dl\]); glucose-fasting (mg/dl) \>1.5\*ULN. Urinalysis: Urine glucose; ketones; urine protein; urine hemoglobin; nitrite; leukocyte esterase; hyaline casts (1/per leukocytosis promoting factor (more than or equal to \[\>=\] 1, urine erythrocytes (scalar); urine leukocytes (scalar) \>=20.

Number of Participants According to Categorization of Changes in Vital Signs
From Day 1 of dosing maximum up to Week 68

Vital signs included the following parameters: sitting diastolic blood pressure (millimetres of mercury \[mmHg\]) change \>=20 mmHg increase; sitting systolic blood pressure (mmHg) change \>=30 mmHg increase, sitting diastolic blood pressure (mmHg) change \>=20 mmHg decrease and sitting systolic blood pressure (mmHg) change \>=30 mmHg decrease.

Number of Participants According to Categorization of Electrocardiogram (ECG) Findings
From Day 1 of dosing maximum up to Week 68

ECG parameters evaluated were: PR interval value \>=300 milliseconds (msec); QRS duration value \>=200 msec; QT interval value \>=500 msec; corrected QT Interval using Fridericia's formula (QTCF) 450 less than or equal to (\<=) value \<480 msec, 480 \<=value\<500 msec and value\>=500 msec.

Secondary Endpoints
Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 52
Baseline (before dose 1), Week 52
Change From Baseline in CDASI Activity Score at Weeks 12, 24, 36, and 48
Baseline (before dose 1), Weeks 12, 24, 36 and 48
Absolute Values of CDASI Activity Score at Weeks 12, 24, 36, 48, and 52
Weeks 12, 24, 36, 48 and 52
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Anti-Beta Interferon drug (PF-06823859)EXPERIMENTALIV infusion
Interventions
NameTypeDescription
Anti-Beta Interferon (PF-06823859)DRUGIV infusion
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites25

Inclusion Criteria: * Participants aged ≥18 and ≤80 with moderate to severe dermatomyositis (DM), that have completed the treatment period of a qualifying study. * Capable of giving signed informed consent. * Participants who are willing and able to comply with all scheduled visits, treatment plan,...

Countries:United StatesHungaryPolandSpain
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