Pharming thinks it has the data to support approval of its ex-Novartis rare disease drug. With a phase 2/3 trial of the PI3Kδ inhibitor hitting its co-primary endpoints, Pharming is preparing to file for approvals in the second quarter.
The Dutch biotech revealed the success of leniolisib in a clinical trial of patients with the rare primary immunodeficiency disease activated phosphoinositide 3-kinase δ syndrome (APDS) two months ago, but that statement lacked details of the data. Pharming filled in those key details Friday at the Clinical Immunology Society 2022 Annual Meeting.
One of the co-primary endpoints looked at lymphadenopathy lesions. The trial linked leniolisib to a -0.30 mean change, compared to a -0.06 change in the placebo cohort, resulting in a hit against the first of the co-primary endpoints.
The second co-primary endpoints measured the change in the proportion of naive B cells from a baseline level of less than 48%. Pharming saw a 34.8% increase in the leniolisib arm, compared to a 5.4% decline in the placebo cohort. The difference was big enough for the trial to hit its other co-primary endpoint.
“It is extremely encouraging to see that this medication is capable of targeting the cause of this difficult disease, both improving care and reducing patients’ symptoms. Progress toward a treatment that is tailor-made for our patients with APDS is a milestone we have long awaited,” Charlotte Cunningham-Rundles, M.D., Ph.D., a professor of immunology at the Mount Sinai School of Medicine in New York, said in a statement.
Novartis was behind the long-awaited success, advancing the candidate, then also known as CDZ173, into the phase 2/3 clinical trial before deciding to cash out midstudy in 2019. Pharming took the other side of the deal, paying $20 million upfront for the rights to the drug. At the time, Pharming penciled a late 2021 or early 2022 launch into its calendar, but the target has slipped.
Pharming now expects to file for approval in the second quarter, putting it in a position to launch the drug in the U.S. in the first quarter of 2023 and to roll the drug out in European markets in the second half of the year.