Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, today announced positive top-line results from the MOTION pivotal Phase 3 study of vimseltinib in patients with TGCT not amenable to surgery. Vimseltinib is the Company’s investigational, orally administered, potent, and highly selective switch-control kinase inhibitor of CSF1R.
“Patients suffering from TGCT are in need of a new treatment option that offers both strong clinical benefit and a well-tolerated safety profile,” said Hans Gelderblom, M.D., Ph.D., Chair of the Department of Medical Oncology at Leiden University Medical Center. “TGCT has a significant negative impact on the daily life of patients who face substantial pain, stiffness, and impaired mobility. Success across both the primary and all key secondary endpoints in MOTION underscores vimseltinib’s ability to help TGCT patients feel and function better. The top-line results from MOTION, together with the impressive data announced today from the Phase 1/2 study showing that the response rates with vimseltinib continue to increase over time, and that patients continue to receive long-term clinical benefit as evidenced by the median duration of treatment, demonstrates vimseltinib’s potential to become a best-in-class agent.”
“We are excited about the potential for vimseltinib to become our next approved medicine, supporting our continued evolution to a company with multiple marketed products,” said Steve Hoerter, President and Chief Executive Officer of Deciphera Pharmaceuticals. “The totality of data shown today demonstrate the potential for vimseltinib to offer a new and differentiated treatment option for patients with TGCT. On behalf of the entire Deciphera team, I would like to thank the patients, their caregivers, and the healthcare professionals who participated in the MOTION and Phase 1/2 studies. We look forward to working with regulatory agencies worldwide as we focus on delivering this important new treatment option to patients with TGCT.”
Top-line Results from the MOTION Pivotal Phase 3 Study of Vimseltinib in TGCT
The MOTION pivotal Phase 3 study is a two-part, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed). In Part 1, patients (n=123) were randomized two-to-one to receive either 30 mg twice weekly of vimseltinib (n=83) or placebo (n=40) for 24 weeks. The primary endpoint of the study is ORR at Week 25 as measured by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 by blinded independent radiologic review (IRR). The open-label Part 2 portion of MOTION, in which patients from both the vimseltinib and placebo arms receive treatment with vimseltinib, remains ongoing. The results for Part 1 of the study are based on a data cutoff date of August 22, 2023.
The study met its primary endpoint in the intent-to-treat (ITT) population demonstrating statistically significant and clinically meaningful improvement versus placebo in ORR at Week 25 based on IRR per RECIST v1.1. In the ITT population, the ORR at Week 25 was 40% (95% CI: 29%, 51%) for the vimseltinib arm and 0% (95% CI: 0%, 9%) for the placebo arm resulting in a response difference (vimseltinib versus placebo) of 40% (95% CI: 29%, 51%) (p<0.0001).
In addition to meeting the primary endpoint, the study also achieved statistically significant and clinically meaningful improvements versus placebo in all key secondary endpoints assessed at Week 25 including ORR per tumor volume score (TVS), active range of motion (ROM), physical function, stiffness, quality of life, and pain.
In the ITT population, the ORR at Week 25 based on IRR per TVS was 67% (95% CI: 56%, 77%) for the vimseltinib arm and 0% (95% CI: 0%, 9%) for the placebo arm (p<0.0001). Treatment with vimseltinib also demonstrated an improvement in mean change from baseline in active ROM at Week 25 of 18.4% versus a 3.8% improvement for placebo (p=0.0077).
Vimseltinib was well-tolerated and the safety profile in the MOTION study was consistent with previously disclosed data. There was no evidence of cholestatic hepatotoxicity in patients treated with vimseltinib. Patients with treatment-emergent adverse events (TEAEs) leading to treatment discontinuation was 6% in the vimseltinib arm. The table below lists TEAEs >15% in either arm during Part 1 of the study.
| Preferred Term n (%) | Vimseltinib (n=83) | Placebo (n=39)1 | ||
| All Grades | Grade 3/4 | All Grades | Grade 3/4 | |
| Periorbital edema^ | 37 (45%) | 3 (4%) | 5 (13%) | 0 |
| Fatigue ^ | 27 (33%) | 0 | 6 (15%) | 0 |
| Face edema^ | 26 (31%) | 1 (1%) | 3 (8%) | 0 |
| Pruritus^ | 24 (29%) | 2 (2%) | 3 (8%) | 0 |
| Headache^ | 23 (28%) | 1 (1%) | 10 (26%) | 0 |
| Asthenia^ | 22 (27%) | 1 (1%) | 9 (23%) | 1 (3%) |
| Nausea ^ | 21 (25%) | 0 | 8 (21%) | 1 (3%) |
| CPK increased | 20 (24%) | 8 (10%) | 0 | 0 |
| AST increased | 19 (23%) | 0 | 1 (3%) | 0 |
| Arthralgia ^ | 16 (19%) | 0 | 6 (15%) | 1 (3%) |
| Rash ^ | 16 (19%) | 0 | 2 (5%) | 0 |
| Rash maculo-papular^ | 16 (19%) | 1 (1%) | 0 | 0 |
| Edema peripheral ^ | 15 (18%) | 0 | 3 (8%) | 0 |
| Hypertension | 14 (17%) | 4 (5%) | 4 (10%) | 1 (3%) |
| Diarrhea | 10 (12%) | 0 | 8 (21%) | 1 (3%) |
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