Phase 1 and 2 clinical trials of efzofitimod from ATYR Pharmaceuticals demonstrated favorable safety profiles and meaningful efficacy signals, particularly in pulmonary sarcoidosis and systemic sclerosis-related interstitial lung disease (SSc-ILD).
Phase 1 Results
- Efzofitimod was found to be **safe and well tolerated** in healthy volunteers and patients during Phase 1 and early Phase 1b/2a studies.
- No serious treatment-related adverse events were reported, and dose-dependent improvements in key clinical endpoints were observed.
- The drug’s pharmacokinetic exposure correlated positively with beneficial effects on pulmonary function and quality of life measurements.
Phase 2 Results
- In the EFZO-CONNECT™ Phase 2 study, efzofitimod showed **early signals of efficacy** for SSc-ILD, with 3 out of 4 treated diffuse patients exhibiting clinically meaningful improvement in skin fibrosis as measured by the modified Rodnan Skin Score (mRSS) at 12 weeks.
- All treated patients showed either stable or improved mRSS, and inflammatory biomarker reductions were noted.
- Safety was confirmed, with no serious adverse events reported through 12 weeks.
- The sample size was small (interim data on eight patients), but these findings supported initiation of larger confirmatory studies.
- In pulmonary sarcoidosis, the drug demonstrated dose-dependent improvements in forced vital capacity (FVC) and patient-reported lung health scores, further confirming clinical proof of concept.
The consistently positive safety profile and efficacy signals in Phase 1 and 2 lay the groundwork for the current pivotal Phase 3 study, which is expected to provide definitive answers on efzofitimod’s therapeutic potential.
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