logo
  

Pharming : Positive Data From Phase 3 Study Of Leniolisib In Rare Primary Immunodeficiency Published

Pharming Group N.V. (PHAR) said that the positive results of a Phase 3 clinical trial of the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor, in adult and adolescent patients with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, have been published in Blood, the peer-reviewed international medical journal of the American Society of Hematology.

The company noted that Leniolisib was well tolerated and significant improvement over placebo was notable in the co-primary endpoints, reflecting a favorable impact on patients' immune dysregulation and deficiency.

Activated Phosphoinositide 3-Kinase d Syndrome is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. It is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3Kd (phosphoinositide 3-kinase delta) pathway.
Balanced signaling in the PI3Kd pathway is essential for physiological immune function.

For More Such Health News, visit rttnews.com

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
Integrated energy company Chevron reported Friday that profit for the fourth quarter increased 25 percent from last year, reflecting 17 percent revenue growth amid higher realizations and higher margins on refined product sales. Adjusted earnings per share missed analysts' estimates, while quarterly revenues topped it. Reno, Nevada-based ZLINE Kitchen and Bath has expanded its recall of gas ranges citing serious risk of injury or death from carbon monoxide poisoning, the U.S. Consumer Product Safety Commission announced. The recall now includes about 30,000 units of ZLINE gas ranges, including about 28,000 units... The Food and Drug Administration's independent advisory committee unanimously recommended replacing the current original COVID vaccines used in the U.S. for primary shots with new bivalent omicron shots authorized as boosters, reports said. The committee also considered the proposal for annual COVID vaccination, however, didn't vote on it.
Follow RTT