Benzinga | Jun 8, 2021 07:34AM EDT

Kiniksa Outlines Next Steps For Development Of Mavrilimumab

- Defined regulatory pathway for Phase 3 clinical development of mavrilimumab in multiple indications -



- Continued development in COVID-19-related ARDS represents fastest path to potential registration for mavrilimumab; Phase 3 data expected in Q1 2022 -

HAMILTON, Bermuda, June 08, 2021 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (NASDAQ:KNSA) ("Kiniksa"), a biopharmaceutical company with a portfolio of assets designed to modulate immunological pathways across a spectrum of diseases, today outlined next steps for the development of mavrilimumab, an investigational fully-human monoclonal antibody that blocks activity of granulocyte macrophage colony stimulating factor (GM-CSF) by binding specifically to the alpha subunit of the GM-CSF receptor.

"Recent favorable interactions with the FDA, based upon the clinical data generated with mavrilimumab in COVID-19-related ARDS, giant cell arteritis, and rheumatoid arthritis, underscore the broad utility of mavrilimumab and define a regulatory pathway for Phase 3 clinical development for each indication," said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. "We believe the ongoing Phase 3 study of mavrilimumab in COVID-19-related ARDS represents the fastest path to potential registration for the asset, and there remains a significant unmet need in these patients. Enrollment in our Phase 3 clinical trial is ongoing, and we expect data in the first quarter of 2022."

Mavrilimumab Development and Capital Allocation

* Interactions between Kiniksa and the U.S. Food and Drug Administration (FDA) on mavrilimumab in COVID-19--related acute respiratory distress syndrome (ARDS), giant cell arteritis (GCA), and rheumatoid arthritis (RA) resulted in a defined path for Phase 3 clinical development for each indication.

* Kiniksa believes development of mavrilimumab in COVID-19-related ARDS represents the fastest path to potential registration. Based upon data from the Phase 2 portion of the Phase 2/3 clinical trial of mavrilimumab in non-mechanically ventilated patients with COVID-19-related ARDS and discussions with the FDA, Kiniksa has adjusted the final sample size to appropriately power the Phase 3 portion of the clinical trial. The Phase 3 clinical trial will enroll a total of approximately 600 non-mechanically ventilated patients. The primary efficacy endpoint remains as the proportion of patients alive and free of mechanical ventilation at Day 29. The Phase 3 portion of the clinical trial has already enrolled over 400 patients. Kiniksa expects data from the Phase 3 portion of the clinical trial in the first quarter of 2022. Kiniksa is engaged with the FDA and other government agencies on pathways to accelerate access to mavrilimumab as a potential therapeutic option for COVID-19-related ARDS.

* Kiniksa completed an end-of-Phase 2 meeting for GCA with the FDA. Kiniksa received guidance that a single, well-controlled pivotal Phase 3 clinical trial in approximately 450 patients could be sufficient for registration. The FDA agreed with the proposed 150 mg and 100 mg subcutaneous (SC) bi-weekly dose levels versus placebo on top of a steroid taper and agreed with a 52-week exposure duration to support chronic use.

* Kiniksa completed a pre-IND meeting for RA with the FDA. Kiniksa received guidance that two well-controlled Phase 3 trials in 1,000-1,500 patients with at least one-year of exposure to mavrilimumab could be sufficient for registration in a broad RA population, such as patients with inadequate response to methotrexate and/or methotrexate na?ve patients. The FDA agreed with the proposed 150 mg and 100 mg SC bi-weekly dose levels versus placebo. Mavrilimumab was dosed at 150 mg and 100 mg SC bi-weekly in over 550 RA patients in Phase 2b clinical studies in Europe which achieved prospectively-defined primary endpoints of efficacy and safety.

* Kiniksa intends to provide additional updates on the development of mavrilimumab following data from the Phase 3 clinical trial in COVID-19-related ARDS. Kiniksa's resources are currently focused on the commercial launch of ARCALYST(r) (rilonacept) in recurrent pericarditis, the Phase 3 clinical trial of mavrilimumab in COVID-19-related ARDS, the Phase 2b clinical trial of vixarelimab in prurigo nodularis, and the planned Phase 2 clinical trial of KPL-404 in RA. The company continues to expect that its approximately $264.0 million of cash, cash equivalents and short-term investments will fund its current operating plan into 2023