Vimseltinib

FDA Approves Vimseltinib for Tenosynovial Giant Cell Tumor Treatment, Showing 40% Response Rate

3 months ago

• The FDA has granted approval to vimseltinib (DCC-3014) for treating symptomatic tenosynovial giant cell tumor (TGCT), based on positive Phase 3 MOTION trial results. • The pivotal MOTION study demonstrated a significant 40% objective response rate for vimseltinib compared to 0% for placebo, with notable improvements in patient mobility and pain. • The twice-weekly oral medication showed a manageable safety profile, with mostly grade 1-2 adverse events, providing a new therapeutic option for TGCT patients.

Deciphera's Qinlock Fails Phase 3 Trial in Second-Line GIST, Shares Plummet 75%

3 months ago

• Deciphera Pharmaceuticals' Qinlock failed to outperform Pfizer's Sutent in second-line gastrointestinal stromal tumor treatment, with progression-free survival of 8.0 versus 8.3 months respectively. • The disappointing INTRIGUE trial results severely impact Qinlock's projected blockbuster potential, previously estimated at $1.5 billion in annual sales. • Currently approved as a fourth-line therapy, Qinlock generates modest quarterly sales of around $20 million, with a pending European approval offering limited upside potential.

FDA Approves Brentuximab Vedotin Combination for Relapsed/Refractory Large B-Cell Lymphoma

4 months ago

• The FDA has approved brentuximab vedotin in combination with lenalidomide and rituximab for adult patients with relapsed or refractory LBCL. • The approval is specifically for those ineligible for auto-HSCT or CAR T-cell therapy after two or more lines of systemic therapy. • Data from the ECHELON-3 trial demonstrated a statistically significant improvement in overall survival with the brentuximab vedotin combination. • Common adverse events included neutropenia, thrombocytopenia, anemia, and peripheral neuropathy, manageable with dose modifications.

Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments

5 months ago

• Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions. • Several companies, including Intellia Therapeutics and IGM Biosciences, are cutting a significant portion of their workforce to focus on key programs and extend cash runways. • These workforce reductions are often accompanied by discontinuation of certain drug development programs and a shift in strategic focus towards core assets. • The layoffs reflect a broader trend in the industry as companies adapt to evolving market dynamics and prioritize efficiency and long-term sustainability.

FDA Gears Up for Critical Decisions on Alzheimer's, Breast Cancer, and Neurological Therapies in Early 2025

5 months ago

• The FDA is set to decide on Biogen and Eisai's Leqembi for monthly intravenous maintenance in early Alzheimer's disease, potentially improving patient convenience. • AstraZeneca and Daiichi Sankyo await a decision on Dato-DXd for metastatic HR-positive, HER2-negative breast cancer, offering a new antibody-drug conjugate approach. • Vertex's suzetrigine, a non-opioid analgesic for moderate-to-severe acute pain, anticipates FDA verdict, representing a novel drug class for pain management. • SpringWorks' mirdametinib is under priority review for neurofibromatosis type 1-associated plexiform neurofibromas, addressing a significant unmet need.

FDA Approves SpringWorks' Gomekli (Mirdametinib) for Neurofibromatosis Type 1

6 months ago

• The FDA has approved Gomekli (mirdametinib) for both adult and pediatric patients with Neurofibromatosis Type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. • Approval was based on Phase 2b ReNeu trial data, demonstrating a 41% ORR in adults and 52% in children, alongside deep and durable tumor volume reductions and manageable safety profiles. • Gomekli is the first and only medicine approved for both adults and children with NF1-PN, addressing a significant unmet need, particularly for adult patients who previously lacked approved treatments. • SpringWorks received a rare pediatric disease priority review voucher from the FDA, potentially expediting the review of future drug candidates.

Advancements in Tenosynovial Giant Cell Tumors Clinical Trials Highlighted for 2024

6 months ago

The Tenosynovial Giant Cell Tumors (TGCT) clinical trials landscape for 2024 showcases significant progress, with over 5 key companies developing more than 5 treatment therapies. Notable developments include the FDA's priority review for vimseltinib, a CSF1R inhibitor, and the EMA's acceptance of its Marketing Authorization Application, signaling a promising future for TGCT treatment.

Abbisko's Pimicotinib Shows Promise in Phase 3 Trial for Tenosynovial Giant Cell Tumor

6 months ago

• Pimicotinib, an oral drug developed by Abbisko Therapeutics, demonstrated a 54% tumor shrinkage rate in patients with tenosynovial giant cell tumor (TGCT). • The Phase 3 MANEUVER study evaluated pimicotinib against placebo in patients with TGCT who had not received prior CSF-1R inhibitor treatment. • Pimicotinib showed significant improvements in secondary endpoints like stiffness and pain and was well-tolerated among the patients. • Merck KGaA holds commercial rights to pimicotinib in mainland China, Hong Kong, Macau, and Taiwan, with plans to explore its use in other solid tumors.

Pimicotinib Achieves Primary Endpoint in Phase 3 Trial for Tenosynovial Giant Cell Tumor

6 months ago

• Merck KGaA's pimicotinib demonstrated a significant objective response rate (ORR) of 54% at week 25 compared to 3.2% for placebo in the MANEUVER trial. • The CSF-1R inhibitor showed efficacy in reducing joint stiffness and pain, as measured by NRS and BPI pain scores, respectively. • Pimicotinib is poised to compete with existing TGCT therapies, including Daiichi Sankyo's Turalio and Ono Pharma's vimseltinib. • Abbisko Therapeutics will collaborate with Merck on the registration of pimicotinib as a systemic therapy for TGCT in China.

Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025

7 months ago

• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD. • EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration. • Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option. • Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.

FDA Approvals and Designations in Oncology: September 2024

8 months ago

• The FDA approved a subcutaneous formulation of atezolizumab (Tecentriq Hybreza) for various cancers, offering a more convenient administration option for patients. • Ribociclib (Kisqali) gained approval for early-stage breast cancer, expanding treatment options for this disease and showing improved invasive disease-free survival. • Pembrolizumab (Keytruda) in combination with chemotherapy was approved for unresectable advanced or metastatic malignant pleural mesothelioma, improving overall survival. • Isatuximab (Sarclisa) plus bortezomib, lenalidomide, and dexamethasone was approved for transplant-ineligible, newly diagnosed multiple myeloma patients.

AbbVie's Pivekimab Sunirine and Deciphera's Vimseltinib: Valuation and Clinical Overview

8 months ago

• Pivekimab sunirine, developed by AbbVie, is projected to generate $21 million in US revenue by 2038, targeting CD123-positive hematologic malignancies. • Vimseltinib, by Deciphera Pharmaceuticals, is expected to reach $173 million in US revenue by 2034, focusing on advanced malignancies and tenosynovial giant cell tumor. • Risk-adjusted net present value (rNPV) models, incorporating phase transition success rates and approval likelihood, offer conservative valuations for both drugs. • Both drugs leverage kinase inhibitor technology, with Pivekimab sunirine using an antibody-drug conjugate and Vimseltinib targeting the FMS kinase (CSF1R).

FDA Grants Priority Review to Vimseltinib for Tenosynovial Giant Cell Tumor

9 months ago

• The FDA has granted priority review to vimseltinib for treating tenosynovial giant cell tumor (TGCT), setting a PDUFA goal date of February 17, 2025. • Vimseltinib, a CSF1R inhibitor, demonstrated a 40% overall response rate in the MOTION phase 3 trial, significantly higher than placebo (0%). • The MOTION trial also showed statistically significant improvements in active range of motion, physical function, and pain reduction with vimseltinib. • Vimseltinib's manageable safety profile, without cholestatic hepatotoxicity or drug-induced liver injury, positions it as a potential new treatment option.

Tenosynovial Giant Cell Tumors Treatment Market to See Significant Growth by 2034

10 months ago

The Tenosynovial Giant Cell Tumors (TSGCTs) treatment market, valued at approximately USD 300 million in 2023, is expected to grow significantly by 2034. This growth is driven by the increasing prevalence of TSGCTs, advancements in treatment options, and the introduction of novel therapies. Key developments include SynOx Therapeutics securing USD 75 million in Series B funding for the development of emactuzumab, a promising treatment for TSGCTs.

Drug Development Updates