Vimseltinib

According to the topline results, a total of 54% of patients given the treatment saw their tumors shrink compared to just 3.2% in the placebo group, hitting the primary endpoint of the trial. TGCT is a rare condition ​ mainly caused by an overexpression of the protein colony stimulating factor-1 receptor (CSF-1R), which drives the proliferation of a range of cells such as microglial cells and macrophages. This leads to joint pain, stiffness and potentially irreversible damage to joints and bones. Surgery is the main approach for treating TGCT but can be difficult to carry out in some patients, while the only systemic therapy approved by the U.S. Food and Drug Administration – Daiichi Sankyo’s CSF-1R blocker pexidartinib (Turalio) – which needs to be taken twice daily and can show side effects such as hepatotoxicity. “Based on these new data from the MANEUVER study, together with once-daily oral administration that may promote long-term adherence and pimicotinib’s selective inhibition of CSF-1R, this investigational medicine has the potential to establish a new treatment paradigm for patients with TGCT,” said Niu Xiaohui, study investigator and director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital, in a public statement ​. The randomized, double-blind, placebo-controlled study tested the benefits of 50 mg pimicotinib daily against a placebo in 94 patients with TGCT in Europe, China and North America who haven’t received previous treatments blocking CSF-1R. The pimicotinib group also saw significant improvements in secondary endpoints such as stiffness and pain, and the drug was well-tolerated. googletag.cmd.push(function () { googletag.display('text-ad1'); }); Abbisko licensed the commercial rights for pimicotinib in all indications in Chinese mainland, Hong Kong, Macau, and Taiwan to Merck KGaA in a deal worth $70 million upfront in 2023. The Chinese player is also aiming to develop the compound for the treatment of solid tumors, chronic graft-versus-host disease and advanced pancreatic cancer. Pexidartinib (Turalio) was first approved in the U.S. in 2019, based on achieving an overall response rate (ORR) of 38% in phase 3 data. Another advanced contender is Deciphera’s vimseltinib, which hit an ORR of 40% in a phase 3 trial for the treatment of TGCT last year. On the other hand, CSF-1R blockers have struggled to perform ​ in clinical trials for the treatment of other tumor types, which may be due to the heterogeneity of cancer in general.

The asset is intended at treating tenosynovial giant cell tumours. Credit: Gorodenkoff via Shutterstock. The US Food and Drug Administration (FDA) has accepted for priority review the new drug application (NDA) for Ono Pharmaceutical subsidiary Deciphera Pharmaceuticals ’ vimseltinib, a treatment for tenosynovial giant cell tumours (TGCTs). Vimseltinib, designed to hinder the colony-stimulating factor 1 receptor (CSF1R), is a product of Deciphera’s switch-control kinase inhibitor platform. The US regulator set a Prescription Drug User Fee Act goal date of 17 February 2025. The FDA’s decision is underpinned by results from the Phase III MOTION study, a two-part, randomised, double-blind, placebo-controlled trial. It assessed vimseltinib’s efficacy and safety in patients with TGCT not amenable to surgery and without prior anti-CSF1/CSF1R therapy, except for imatinib or nilotinib. See Also: Pfizer and BioNTech’s Covid-19/flu combination vaccine falters in Phase III readout AstraZeneca wins approval for Imfinzi in NSCLC perioperative setting At week 25, the asset offered a statistically significant and clinically meaningful objective response rate in the intent-to-treat population versus placebo. It also showed significant improvements in all key secondary endpoints over placebo. Vimseltinib’s safety profile in the trial has been deemed manageable, with safety data from the MOTION study aligning with earlier Phase I/II clinical trial findings. The European Medicines Agency (EMA) initiated the centralised review process for vimseltinib, having accepted a Marketing Authorization Application in July this year. Deciphera Pharmaceuticals CEO and president Steve Hoerter said: “Building upon positive results from the MOTION pivotal Phase III study and following our recent announcement that EMA review of the vimseltinib MAA has begun, we are excited to initiate the regulatory review process in the US and we look forward to working with the FDA to deliver a new treatment option to patients with TGCT.” In April this year, Ono and Deciphera signed a definitive merger agreement for a deal valued at $2.4bn.