Centessa Pharmaceuticals Initiates Global Phase 3 ACTION Study of Lixivaptan in Autosomal Dominant Polycystic Kidney Disease – BioSpace

Centessa Pharmaceuticals Initiates Global Phase 3 ACTION Study of Lixivaptan in Autosomal Dominant Polycystic Kidney Disease – BioSpace

~ Initiation of registrational Phase 3 ACTION clinical study with lixivaptan is an important milestone to bring this potential new treatment option to ADPKD patients ~

~ All four subjects in the ALERT Study who previously discontinued JYNARQUE® due to liver toxicity successfully titrated to maintenance dose of lixivaptan; no subjects met pre-specified stopping criteria; no cases of suspected drug-induced liver injury (DILI) ~

~Issuance of new patent would cover use of lixivaptan in ADPKD through at least 2038 ~

BOSTON and LONDON, Dec. 14, 2021 (GLOBE NEWSWIRE) — Centessa Pharmaceuticals plc (“Company”) (Nasdaq: CNTA), together with subsidiary Palladio Biosciences, Inc. (“Palladio”), today announced the initiation of active recruitment of the global ACTION Study, a pivotal Phase 3 clinical trial evaluating lixivaptan as a potential treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD). Additionally, the Company reported initial safety data from four subjects who participated in the ongoing open-label ALERT Study of ADPKD subjects who previously discontinued JYNARQUE® (tolvaptan) due to liver toxicity and announced the Notice of Allowance for a U.S. Patent application covering use of lixivaptan in ADPKD.

“Patients with ADPKD need alternative treatment options to the currently approved therapy, which is associated with a Risk Evaluation and Mitigation Strategies (REMS) program due to its side effect profile. I have been encouraged by the pharmacodynamic and tolerability data generated to date with lixivaptan and look forward to seeing the benefit and safety data from the upcoming pivotal ACTION Study,” said Vicente Torres, MD, PhD, Professor of Medicine, Mayo Clinic and Chairman of the Steering Committee and Principal Investigator of the ACTION Study.

“We are thrilled to begin the registrational ACTION Study so we can further evaluate lixivaptan’s potential as a new treatment option in the broader ADPKD patient population,” said Saurabh Saha, MD, PhD, Chief Executive Officer of Centessa. “Furthermore, the initial safety data from the ALERT Study in subjects who have stopped JYNARQUE due to liver toxicity continues to support the differentiated safety and tolerability profile of lixivaptan. In addition, the allowed claims addressed in the recently issued Notice of Allowance from the U.S. Patent & Trademark Office should provide patent protection for the covered use of lixivaptan in ADPKD treatment in the U.S. to at least 2038.”

“The initial safety data we shared today from the ALERT Study is similar to the case study we previously reported from the Mayo Clinic and provides additional evidence of lixivaptan’s tolerability profile, especially in a group of ADPKD subjects who had previous liver chemistry abnormalities while taking tolvaptan,” said Neil Shusterman, MD, Chief Medical Officer of Palladio. “We look forward to bringing this potential new treatment option to ADPKD patients.”

ACTION Study Initiation

Recruitment has commenced in the global Phase 3 ACTION Study which consists of a two-arm, double-blind, placebo-controlled, randomized phase (Part 1) followed by a single-arm, open-label phase (Part 2). The study will evaluate the benefit and safety of lixivaptan that has been titrated to a maximum tolerated dose between 100-200 mg BID in subjects with ADPKD and a Mayo Clinic MRI imaging classification of 1C, 1D or 1E and an estimated glomerular filtration rate (eGFR) ≥25 and ≤90 mL/min/1.73 m<…….

Source: https://www.biospace.com/article/centessa-pharmaceuticals-initiates-global-phase-3-action-study-of-lixivaptan-in-autosomal-dominant-polycystic-kidney-disease-/

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