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Ultragenyx Shares Tumble On Initial Efficacy Missing Expectations, This Analyst Sees ‘Viable Path Forward”

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…
byVandana Singh
July 19, 2022
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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 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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Burna Boy’s Vapor Products Go Global: When Are They Hitting Nigeria, Ghana, South Africa, EU & UK?

By Jelena Martinovic
December 14, 12:43 PM
Ispire Technology Inc., a vaping technology company, has entered into a five-year exclusive global manufacturing and distribution agreement with lifestyle brand BrkFst. Under the agreement, Ispire will distribute and commercialize BrkFst branded vapor products, including a range of unique varieties and flavors. The products will be initially launched in Nigeria, Ghana, and South Africa, with plans to expand to the European Union and United Kingdom in 2024. Ispire is known for its innovation in vaping technology and holds over 500 patents in the industry. This collaboration aims to offer consumers exclusive vaping technologies and products.

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