- 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.
EXCLUSIVE: Fifteen New F1 Hybrid Cannabis Strains To Hit Shelves In US For 4/20 Under New Partnership
California-based I Love Growing Marijuana (ILGM), announced on Tuesday an exclusive new partnership with cannabis genetics and breeding company, Phylos Bioscience, that brings 15 new F1 hybrid strains to home growers across legal markets nationwide. ILGM is launching half the collection of strains that are "crafted for potency, flavor, and diversity" on its website just in time for marijuana culture's high holiday on 4/20. In doing so, the company is leveraging Phylos Bioscience's cutting-edge technology and extensive genetic library, which Benzinga has learned exclusively. Ernst Rustenhoven, CEO of ILGM, commented on the new partnership. "ILGM caters to a customer base of novice and veteran home growers on the hunt for increasingly unique strains of superior genetics, and we have been seeking an innovative partner to help elevate and diversify our product offerings," Rustenhoven said. "In forging a relationship with Phylos, we are taking our seed catalog to the next level with a highly curated selection of sought-after F1 hybrid strains."
