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
Deciphera Pharmaceuticals announced the Phase 3 results of their MOTION study on vimseltinib for treating tenosynovial giant cell tumor (TGCT) at the 2024 ASCO Annual Meeting and in The Lancet. The study showed significant efficacy, quality-of-life improvements, and a well-tolerated safety profile. The company plans to submit a New Drug Application (NDA) in Q2 2024 and a Marketing Authorization Application (MAA) in Q3 2024. Key findings included a 40% overall response rate (ORR) for vimseltinib compared to 0% for placebo, significant improvements in range of motion, physical function, and pain reduction. Vimseltinib was well tolerated with mainly mild side effects. Additionally, Deciphera is conducting a Phase 1/2 study of DCC-3116 in combination with ripretinib for advanced gastrointestinal stromal tumors, currently in the dose escalation phase.
- Vimseltinib showed a 40% ORR at Week 25 vs. 0% for placebo.
- Significant improvements in all six secondary endpoints.
- DCPH plans to submit NDA in Q2 2024 and MAA in Q3 2024.
- Vimseltinib was well-tolerated with mainly grade 1 or 2 adverse events.
- No evidence of cholestatic hepatotoxicity or drug-induced liver injury.
- Phase 1/2 study of DCC-3116 in combination with ripretinib is ongoing.
- TEAEs led to treatment discontinuation in 6% of vimseltinib patients.
- The Phase 3 study's data cutoff is based on a single time point (August 22, 2023).
Insights
The results from the MOTION Phase 3 study of vimseltinib offer notable advancements in the treatment of Tenosynovial Giant Cell Tumor (TGCT). The study demonstrated a 40% Overall Response Rate (ORR) at Week 25 for patients treated with vimseltinib, a meaningful improvement over the 0% response rate seen with placebo. These figures are promising within the context of TGCT, a rare and often debilitating condition.
Vimseltinib's efficacy across multiple secondary endpoints, such as improvements in quality-of-life and reductions in pain and stiffness, underscore its potential as a comprehensive treatment solution. These secondary endpoints are important as they directly affect the daily lives of patients, offering not just tumor reduction but an enhanced quality of life.
The favorable safety profile, with no significant hepatotoxicity, further strengthens the case for vimseltinib. Safety in long-term cancer treatments is pivotal and the lack of severe adverse events signifies that patients can potentially maintain this therapy over extended periods without detrimental side effects.
From a financial perspective, the announcement holds substantial implications for Deciphera Pharmaceuticals. The statistically significant and clinically meaningful results from the Phase 3 study pave the way for an NDA submission in the second quarter of 2024. This timeline suggests a potential market entry by late 2024 or early 2025, assuming regulatory approval is granted without substantial delays.
With TGCT being an area of high unmet need, vimseltinib's approval could provide Deciphera with a valuable revenue stream. The company's focus on submitting both an NDA in the U.S. and an MAA in Europe demonstrates a strategic approach to capturing a broad market share.
However, investors should remain vigilant about competitive dynamics and pricing strategies. While the data is promising, the financial success will also depend on market penetration and the ability to establish vimseltinib as the preferred treatment option in a competitive oncology market.
– 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 Lancet.
“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 Hans Gelderblom, M.D., Ph.D., Chair of the Department of Medical Oncology at Leiden University Medical Center and senior author of the manuscript. “In addition to its robust antitumor activity and tolerability, vimseltinib also demonstrated clinically significant functional and symptomatic improvements in key quality-of-life measures, which can provide long-term, meaningful benefits to TGCT patients.”
“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 Matthew L. Sherman, M.D., Executive Vice President and Chief Medical Officer of Deciphera. “We remain on track to submit an NDA for vimseltinib in the second quarter of 2024, and an MAA in the third quarter of 2024, and look forward to bringing this important new therapy to TGCT patients globally.”
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: William D. Tap, M.D., FASCO, Memorial Sloan Kettering Cancer Center
Presentation Date: Monday, June 3, 2024
Presentation Time: 3:00 – 3:12 PM CT
Key Highlights:
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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.
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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.
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In the ITT population, the ORR at Week 25 was
40% (95% CI:29% ,51% ) for the vimseltinib arm and0% (95% CI:0% ,9% ) for the placebo arm resulting in a response difference (vimseltinib versus placebo) of40% (95% CI:29% ,51% ) (p<0.0001).
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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.
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ORR by TVS: The ORR at Week 25 based on IRR per TVS was
67% (95% CI:56% ,77% ) for the vimseltinib arm and0% (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 a3.8% 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).
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Brief Pain Inventory (BPI) Worst Pain Response Rate: The BPI worst pain response rate was
48% for patients receiving vimseltinib versus23% for placebo (p=0.0056).
-
ORR by TVS: The ORR at Week 25 based on IRR per TVS was
-
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.
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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: Sreenivasa Chandana, M.D., Ph.D., START Midwest, The Cancer & Hematology Centers
Session Date: Saturday, June 1, 2024
Session Time: 1:30 – 4:30 PM CT
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 Pharmaceuticals
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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Source: Deciphera Pharmaceuticals, Inc.
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