- Earlier today, Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) released updated interim GTX-102 data, including Day 128 assessments from 9 patients.
- The Overall CGI-C-AS score improved by 1 point in 5/6 patients (average 0.8) in Cohort 4 and 1 point in 2/3 patients (average 0.7) in Cohort 5.
- Credit Suisse says that the result is slightly lower than Street expectations.
- But the physicians’ CS spoke to highlighted a very low bar for a clinically meaningful product, and higher loading doses/longer dosing period could potentially improve these initial results.
- Additional efficacy metrics presented were supportive of clinical improvements.
- The most common AE was vomiting, which may be related to the procedure and anesthesia.
- Data from the U.S. cohort that was dosed at 2 mg showed a 2-point overall improvement in two evaluable patients. The ex-US study protocol is now amended to enroll two additional cohorts sequentially, Cohort 6 (younger, starting at 7.5 mg loading doses) and Cohort 7 (older, starting at 10 mg).
- Management noted that higher loading doses could significantly improve the efficacy profile.
- Price Action: RARE shares are down 13.70% at $52.66 during the market session on the last check Tuesday.
Double Flops For Merck’s Lung Cancer Studies
Merck & Co Inc (NYSE: MRK) announced full results from the Phase 2 KeyVibe-002 trial of vibostolimab/pembrolizumab combo, with or without docetaxel for metastatic non-small cell lung cancer (NSCLC) with progressive disease after treatment with immunotherapy and
