Regulatory roundup: Novartis rare disease drug sees approval chances rise after trial completes
By Reynald Castaneda27 Sep 2021 (Last Updated September 27th, 2021 16:10)
Theravance's ampreloxetine and Novartis in rare disease are among recent events reviewed by GlobalData’s Investigative News team.
Novartis’ PI3K-delta inhibitor CDZ173 (leniolisib) had its LoA increase by five points to 20% on the heels of its Phase II/III trial, which has completed.
Novartis’ PI3K-delta inhibitor CDZ173 (leniolisib) had its LoA increase by five points to 20% on the heels of its Phase II/III trial, which has completed. The trial is recruiting patients with PASLI disease (p110d-activating mutation causing senescent T cells, lymphadenopathy, and immunodeficiency), also known as activated phosphoinositide 3-kinase delta syndrome (APDS).
The LoA appraisal occurred on 21 September, after the Phase II/III trial’s ClinicalTrials.gov listing was updated on 17 September to show its actual completion was on 16 August. The Phase II/III trial had two parts: the first is an open-label, within-patient up-titration study, and the second is a blinded, placebo-controlled fixed-dose study. Part I has coprimary endpoints collecting safety and pharmacokinetic data, while Part II had efficacy-relevant endpoints.
High expression of PI3K-delta in leukocytes, as well as its pivotal role in B and T cells, is assumed that PI3K-delta selective inhibition would be valuable in allergic and inflammatory disease. PASLI disease is recognised by recurrent sinopulmonary infections that can pave the way to airway damage.
Kalyra’s pain approach sinks by 36 points
Kalyra Pharmaceuticals’ KP-1199 for chronic pain saw its PTSR nosedive 36 points to 31% after its Phase I trial was terminated. The PTSR change occurred on 21 September after ClinicalTrials.gov listed the trial as “terminated early by sponsor” on 9 September.
The San Diego, California-based Kalyra had planned to enroll 97 healthy subjects in a Phase I single and multiple ascending dose study. The trial was designed to enrol participants into three groups where they would receive KP-1199, placebo or the active comparator oxycodone. The study enrolled only 26 subjects before it was terminated. KP-1199 is a small molecule acetaminophen analog that can help alleviate chronic pain.
The trial termination also resulted in a four-point drop to the drug’s Likelihood of Approval (LoA), which fell to 3%.