Deciphera Pharmaceuticals Announces Oral Presentation of Results from MOTION Pivotal Phase 3 Study of Vimseltinib in Patients with Tenosynovial Giant Cell Tumor (TGCT) at the 2024 ASCO Annual Meeting and Online Publication in The Lancet
– MOTION Phase 3 Data Demonstrate Robust Efficacy, Clinically Meaningful Improvements in Quality-of-Life Measures, and Well-Tolerated Safety Profile, Positioning Vimseltinib as Potential New TGCT Treatment –
– Company Expects to Submit a New Drug Application (NDA) in the Second Quarter of 2024 and Marketing Authorisation Application (MAA) in the Third Quarter of 2024 –
The article titled “Vimseltinib versus placebo for tenosynovial giant cell tumour (MOTION): a randomised phase 3 trial” is now available online and will be published in a future print issue of
“The results from the MOTION pivotal Phase 3 study provide compelling evidence that vimseltinib can address the unmet medical need in TGCT for an effective and well-tolerated therapy without cholestatic hepatotoxicity,” said
“Building upon the strong efficacy results disclosed previously at topline, we are excited to share details on the statistically significant and clinically meaningful improvements that vimseltinib offered TGCT patients across all six key secondary endpoints, along with its favorable safety profile,” said
In addition to the results from the MOTION pivotal Phase 3 study, the Company will also be presenting a trial-in-progress poster for the ongoing Phase 1/2 study of DCC-3116 in combination with ripretinib at the 2024 ASCO Annual Meeting.
Both presentations are available on the Company’s website at www.deciphera.com/presentations-publications. Presentation details are as follows:
Abstract Number: 11500
Title: Efficacy, safety, and patient-reported outcomes of vimseltinib in patients with tenosynovial giant cell tumor: Results from the phase 3 MOTION trial.
Presenter:
Presentation Date:
Presentation Time: 3:00 –
Key Highlights:
- Study Design: 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 results for Part 1 of the study are based on a data cutoff date of
August 22, 2023 . - The open-label Part 2 portion of MOTION, in which patients from both the vimseltinib and placebo arms receive treatment with vimseltinib, remains ongoing.
- 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 results for Part 1 of the study are based on a data cutoff date of
- ORR: 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 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).
- Secondary Endpoints: In addition to meeting the primary endpoint, the study also achieved statistically significant and clinically meaningful improvements versus placebo in all six key secondary endpoints assessed at Week 25 including ORR by tumor volume score (TVS), active range of motion (ROM), physical function, stiffness, quality of life, and pain.
- ORR by TVS: 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).
- Active ROM: Treatment with vimseltinib 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).
- Physical Function by PROMIS-PF: Treatment with vimseltinib demonstrated an improvement in mean change from baseline in PROMIS-PF at Week 25 of 4.6 versus 1.3 for placebo (p=0.0007).
- Worst Stiffness Numeric Rating Scale (NRS): Patients treated with vimseltinib reported a decrease of 2.1 versus 0.3 for placebo in worst stiffness (p<0.0001).
- Quality of Life by EuroQol Visual Analogue Scale (EQ-VAS): The mean change from baseline for EQ-VAS was significantly higher with 13.5 in the vimseltinib arm versus 6.1 in the placebo arm (p=0.016).
- Brief Pain Inventory (BPI) Worst Pain Response Rate: The BPI worst pain response rate was 48% for patients receiving vimseltinib versus 23% for placebo (p=0.0056).
- Safety and Tolerability:
- Vimseltinib was well tolerated with most treatment-emergent adverse events (TEAEs) were grade 1 or 2.
- There was no evidence of cholestatic hepatotoxicity, drug-induced liver injury, or hair hypopigmentation.
- Serum enzyme elevations were consistent with the known mechanism of action of CSF1R inhibitors.
- TEAEs leading to treatment discontinuation was 6% in the vimseltinib arm.
- If approved, vimseltinib offers an effective systemic treatment to patients with TGCT and provides proven functional health and symptomatic benefit to a population living with substantial morbidity and limited treatment options.
Abstract Number: TPS11587
Title: DCC-3116 in combination with ripretinib for patients with advanced gastrointestinal stromal tumor: A phase 1/2 study.
Presenter:
Session Date:
Session Time: 1:30 –
Key Highlights:
- This is a phase 1/2, multicenter study designed to evaluate the safety, tolerability, and efficacy of DCC-3116 in combination with ripretinib.
- In Part 1, eligible patients will receive escalating oral doses of DCC-3116 combined with ripretinib 150 mg once daily (QD); the safety will be evaluated and the recommended phase 2 dose (RP2D) will be determined.
- In Part 2, eligible patients will receive the RP2D of DCC-3116 in combination with ripretinib 150 mg QD; antitumor activity will be evaluated.
- The trial is currently enrolling in the phase 1 dose escalation portion.
About
Deciphera is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer. We are leveraging our proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines. In addition to advancing multiple product candidates from our platform in clinical studies, QINLOCK® is Deciphera’s switch-control inhibitor for the treatment of fourth-line GIST. QINLOCK is approved in
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