Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) shares made a handy gain to close out the week after the company announced positive results from its midstage trial for the treatment of glycogen storage disease type Ia (GSDIa).
Specifically, the firm announced positive topline results from its ongoing Phase 1/2 study of DTX401, an adeno-associated-virus-based gene therapy for the treatment of GSDIa.
All three patients saw a biologic response reflected by improved glucose control and increased time to hypoglycemia during fasting, with two patients demonstrating a clinically meaningful improvement in time to hypoglycemia during a controlled fasting challenge.
GSDIa is caused by a defective gene for the enzyme G6Pase-α, resulting in the inability to regulate blood sugar (glucose). Hypoglycemia in patients with GSDIa can be life-threatening, while the accumulation of the complex sugar glycogen in certain organs and tissues can impair the ability of these tissues to function normally.
Eric Crombez, M.D., chief medical officer of the Ultragenyx Gene Therapy development unit, commented:
Two of three patients in this first dose cohort experienced a sufficient increase in time to hypoglycemia such that they may be able to sleep through the night without taking supplemental cornstarch. In addition to the promising results observed with the controlled fasting challenge, all three patients have demonstrated improvement in glucose control throughout the day, and all three patients have been able to decrease their daily cornstarch intake by approximately half. We are extremely encouraged by these results and look forward to beginning enrollment in the second cohort this month, with results expected in mid-2019.
Shares of Ultragenyx were last seen up about 8% at $43.20, in a 52-week range of $37.44 to $90.98. The consensus analyst price target is $70.53.
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