Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) was a top gainer in Friday’s trading session, and closed the day with gains of 23.39%. This came after it announced positive topline cohort 3 results. It also announced a much better long-term cohort 2 results from phase 1/2 study of its DTX301 therapy for patients with Ornithine Transcarbamylase Deficiency. Cohort 3 used 3 female responders in need of long-term follow-up to record their response rate.
On the other hand, the company announced that cohort 2 has a female patient that showed a positive response at week 52, and with a confirmation on week 78. The company further announced that the two responders in cohort 1 and 2 showed clinical and metabolic stability at week 78 and 104. Overall, out of the 9 patients that got a dosage, 6 responded to it.
In its summary of Cohort 3, the company reported that one female patient had a clinically significant response, standing at 79% change in ureagenesis. This was an improvement from 24% of normal and remaining within 44% of normal by week 52. The company reported that the patient reported feeling much better and even dropped other treatments. She has maintained stability and without an increase in ammonia levels. Patient 8 also showed a significant decrease in ammonia levels by up to 90%. Patient 9 had a 123% increase in ureagenesis levels from 25% of normal to 56% of normal by the 12th week.
In its summary of cohort 2, the company announced that patient 6 had 218% improvement in the rate of ureagenesis. She also had a 74% decrease in the levels of ammonia by week 78. That is the point where she started to drop her other medical alternatives and take a more liberal diet.
From its price action, RARE is trading is in an early bull trend. The stock gapped up in Friday’s session to open the day at $48.91. It then traded between a low of $48.84 and a high of $58.54 before closing the day at $53.96. Volumes in the day stood at 3.26 million.
About Ultragenyx Pharmaceutical Inc
Ultragenyx Pharmaceutical Inc is a biopharma company focused on the development and sale of treatments for ultra-rare genetic diseases. It is based in Novato, California.