PaTHway Phase 3 Trial Analysis
TransCon PTH PATHway Trial Top-Line Data at Week 26
Pathway
TRIAL
PaTHway Trial met primary and all key secondary endpoints
78.7% of patients (48 of 61) treated with TransCon PTH achieved the primary endpoint, compared to 4.8%
(1 of 21) of patients in the control group (p-value <0.0001)
Statistically significant improvements observed on all key prespecified secondary endpoints compared to control:
HPES Symptom measures: Physical domain score (p-value = 0.0038) and Cognitive domain score
(p-value = 0.0055)
HPES Impact measures: Physical Functioning domain score (p-value = 0.0046) and Daily Life domain score
(p-value = 0.0061)
SF-36v2Ⓡ - Physical Functioning subscale score (p-value = 0.0347)
TransCon PTH was generally well tolerated, with no discontinuations related to study drug
82% of TransCon PTH patients and 100% of patients in control group reported treatment-emergent adverse events
(TEAEs), the majority of which were Grade 1, 2 in severity.
One serious related TEAE in the TransCon PTH arm was reported due to a dosing error
One death in the TransCon PTH arm was assessed as unrelated to study drug
TransCon PTH-treated patients showed a mean decrease in 24-hour urine calcium excretion into the normal range,
from 390 mg/24 hours down to 220 mg/24 hours
3 HPES, Hypoparathyroidism Patient Experience Scales.
TransCon PTH is an investigational product candidate.
For investor communication only.
Not for use in product promotion. Not for further distribution.
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