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Ultragenyx Shares Tumble On Initial Efficacy Missing Expectations, This Analyst Sees 'Viable Path Forward"

Published 19/07/2022, 19:51
© Reuters. Ultragenyx Shares Tumble On Initial Efficacy Missing Expectations, This Analyst Sees 'Viable Path Forward"
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  • 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 (SIX:CSGN) 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.
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