Pharming To Develop Leniolisib For Additional PIDs Beyond APDS; Phase 2 Trial To Initiate In Q2

(RTTNews) - Dutch biopharmaceutical company Pharming Group N.V. (PHAR) announced Wednesday its plans to develop leniolisib for additional primary immunodeficiencies or PIDs beyond activated phosphoinositide 3-kinase delta syndrome or APDS.

As part of its expansion plans for its rare disease pipeline, the company said it has engaged with the US Food and Drug Administration and has received feedback on its plans to develop leniolisib for PID disorders with immune dysregulation.

The FDA recently reviewed a Phase 2, proof of concept, clinical trial protocol in PIDs with immune dysregulation linked to PI3K? signaling submitted under the existing leniolisib IND.

Pharming noted that Joenja (leniolisib) is an oral small molecule phosphoinositide 3-kinase delta (PI3K?) inhibitor approved in the US as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kd) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

Pharming said the Phase 2 clinical trial will evaluate leniolisib in PIDs with immune dysregulation linked to PI3K? signaling in lymphocytes, with similar clinical phenotypes to APDS.

The Phase 2 clinical trial, a single arm, open-label, dose range-finding study, will be conducted in around 12 patients, starting in the second quarter of 2024.

The objectives for the trial will be to assess safety and tolerability, pharmacokinetics, pharmacodynamics, and explore clinical efficacy of leniolisib in this new PID population. The trial has been designed to inform a subsequent Phase 3 program, the company noted.

Jocelyn Farmer, Director, Clinical Immunodeficiency Program, Beth Israel Lahey Health, said, "PI3K? is an important regulator of lymphocytes, and unbalanced PI3K? signaling in lymphocytes is a key signature of immune dysregulation among PID patients who develop lymphoproliferative and autoimmune disease. Therefore, I am very excited to see Pharming progressing the evaluation of the PI3K? inhibitor leniolisib into PIDs beyond the FDA-approved APDS indication, where it promises an opportunity to provide critical benefit to patients with a large, currently unmet, clinical need."

In Tuesday's after hours trading on Nasdaq, Pharming Group shares were at $10.64, down 4.3 percent.

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