Tovorafenib

Grit Biotechnology to Present Breakthrough Immunotherapy Advances at ASGCT 2025

10 days ago

• Grit Biotechnology will showcase three innovative research programs at the ASGCT 2025 Annual Meeting, including an APC-targeted neoantigen cancer vaccine, non-viral in vivo CAR-T therapy, and LNP-mediated gene editing in TILs. • The presentations, developed in collaboration with Vitalgen BioPharma, demonstrate significant advancements in precision delivery technologies for next-generation immunotherapies targeting solid tumors. • Grit Bio's clinical pipeline includes GT101, the first TIL therapy in pivotal Phase II trials, and GT201, the first TIL therapy with membrane-bound IL-15 complex that has received IND clearance in both US and China.

Pipeline Advances in Head and Neck Cancer Treatment: Over 100 Drugs in Development for 2025

2 months ago

• DelveInsight's latest report reveals a robust pipeline with over 80 companies developing 100+ drug candidates for head and neck cancer, highlighting significant research momentum in this therapeutic area. • Key late-stage candidates include Debiopharm/Merck's xevinapant, GlaxoSmithKline's dostarlimab, and QBiotics' tigilanol tiglate, each employing novel mechanisms to potentially improve outcomes for patients with limited treatment options. • Multiple clinical trials are underway testing innovative approaches including IAP inhibitors, immune checkpoint blockers, and oncolytic therapies, with several Phase III studies expected to deliver results that could reshape treatment paradigms.

Brain Hemorrhage Treatment Market Expected to Grow Significantly by 2032 as AI Solutions Gain FDA Approval

2 months ago

• The brain hemorrhage treatment market is projected to experience substantial growth through 2032, driven by an aging population and increasing prevalence of risk factors such as hypertension and traumatic brain injuries. • Recent FDA approvals for AI-based diagnostic tools, including JLK-LVO and Heuron ICH, mark significant advancements in early detection and treatment of brain hemorrhages. • Several pharmaceutical companies including Baxter, Pfizer, and Novartis are developing promising pipeline therapies such as tissue plasminogen activator, alteplase, and BAF312 to address the significant unmet needs in brain hemorrhage treatment.

Ipsen Secures €500 Million Inaugural Rated Public Bond Following Investment Grade Ratings

2 months ago

• Ipsen has successfully completed its first Rated Public Bond of €500 million with a 3.875% coupon maturing in March 2032, following Investment Grade ratings from S&P (BBB-) and Moody's (Baa3). • The transaction was heavily oversubscribed by institutional investors, demonstrating market confidence in Ipsen's financial strategy and long-term growth prospects. • This bond issuance is part of Ipsen's broader refinancing plan, which recently included the renewal of a €1.5 billion Revolving Credit Facility, significantly extending the company's debt maturity profile.

XOMA Royalty Expands Portfolio to Over 120 Assets, Reports Financial Results for 2024

2 months ago

• XOMA Royalty doubled its portfolio to over 120 royalty assets through five strategic transactions in 2024, strengthening its position as a biotech royalty aggregator. • The company completed two whole company acquisitions and celebrated FDA approvals for Day One's OJEMDA™ (tovorafenib) and Zevra's MIPLYFFA™ (arimoclomol), generating significant milestone payments. • Despite reporting a net loss of $13.8 million for 2024, XOMA Royalty received $46.3 million in cash receipts and maintains over $100 million in cash, positioning it for sustainable cashflow from royalties.

Ipsen Advances Pipeline with Multiple Regulatory Wins and Strategic Partnerships in 2024

3 months ago

• Ipsen secured FDA approval for Onivyde in first-line pancreatic cancer treatment and gained approvals for Iqirvo and Kayfanda in the U.S. and EU markets, expanding their therapeutic portfolio. • The company strengthened its pipeline through strategic partnerships, including agreements with Sutro Biopharma, Foreseen Biotechnology, and DayOne Biopharmaceuticals for innovative oncology assets. • Looking ahead to 2025, Ipsen projects sales growth exceeding 5.0% and anticipates key regulatory decisions for Cabometyx in neuroendocrine tumors and tovorafenib in pediatric low-grade glioma.

FDA Approves FoundationOne CDx as Companion Diagnostic for Tovorafenib in Pediatric Low-Grade Glioma

4 months ago

• The FDA has approved FoundationOne CDx as a companion diagnostic for tovorafenib (Ojemda) in pediatric low-grade glioma (pLGG) patients with specific BRAF alterations. • FoundationOne CDx detects BRAF fusions, rearrangements, and V600 mutations, enabling precise identification of pLGG patients who may benefit from tovorafenib treatment. • Tovorafenib received accelerated approval in April 2024 for relapsed/refractory BRAF-altered pLGG, supported by a 67% overall response rate in the FIREFLY-1 trial. • This approval marks Foundation Medicine’s first companion diagnostic indication exclusively supporting pediatric patients, enhancing precision medicine in pediatric oncology.

FDA Approvals in 2024: Advancing Treatment Paradigms in Solid Tumors and Hematologic Malignancies

5 months ago

• The FDA granted over 65 approvals in 2024, significantly impacting treatment paradigms across various cancers, including breast, gynecologic, skin, and genitourinary malignancies. • Several tumor-agnostic approvals, such as fam-trastuzumab deruxtecan-nxki (Enhertu) for HER2-positive solid tumors and repotrectinib (Augtyro) for NTRK fusion-positive tumors, marked advancements in precision medicine. • Immunotherapies like nivolumab (Opdivo) and pembrolizumab (Keytruda) received multiple approvals, including combinations with chemotherapy for urothelial and endometrial carcinomas, improving patient outcomes. • Targeted therapies like vorasidenib (Voranigo) for low-grade glioma and selpercatinib (Retevmo) for RET-mutated thyroid cancers addressed unmet needs and demonstrated high efficacy and tolerability.

FDA Approvals in 2024: Novel Therapies for MASH, WHIM Syndrome, Pediatric Glioma, MDS, and Bladder Cancer

5 months ago

• The FDA approved Madrigal's Rezdiffra, the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), addressing a significant unmet need in liver disease. • X4 Pharmaceuticals' Xolremdi gained approval as the first targeted therapy for WHIM syndrome, a rare immunodeficiency, marking a milestone for the company. • Day One Biopharmaceuticals' Ojemda secured approval for BRAF-altered pediatric low-grade glioma, offering a new treatment option for this common childhood brain tumor. • Geron Corporation's Rytelo, a telomerase inhibitor, received FDA approval for lower- to intermediate-risk myelodysplastic syndromes (MDS), after 34 years in business. • ImmunityBio's Anktiva, a novel IL-15 superagonist, was approved for non-muscle invasive bladder cancer, providing a new therapeutic approach for BCG-unresponsive patients.

Tovorafenib Demonstrates Improved Quality of Life in Pediatric Low-Grade Glioma

6 months ago

• Tovorafenib (Ojemda) shows promise in improving health-related quality of life (HRQOL) in pediatric patients with BRAF-altered relapsed/refractory low-grade glioma. • The phase 2 FIREFLY-1 trial exploratory analysis indicates stable or improved HRQOL, particularly in anxiety and cognition domains, during the first year of treatment. • The findings complement previously reported efficacy data, suggesting that tovorafenib not only provides clinical benefit but also enhances patients’ quality of life. • Long-term HRQOL analysis was limited, underscoring the need for future studies to assess the sustained impact of tovorafenib on overall well-being.

Tovorafenib Shows Durable Off-Treatment Responses in Pediatric BRAF-Altered Low-Grade Glioma

6 months ago

• Tovorafenib (Ojemda) demonstrates durable responses in pediatric patients with BRAF-altered relapsed/refractory low-grade glioma during drug holidays in the FIREFLY-1 trial. • 97% of patients on a drug holiday showed no clinical progression, with 15% experiencing additional tumor shrinkage of at least 25%. • The safety profile of tovorafenib remained consistent in patients rechallenged after a drug holiday, with retreatment ongoing in some cases. • These findings support tovorafenib's role as a systemic therapy for pediatric low-grade glioma with BRAF alterations, including fusions and V600 mutations.

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.

Drug Development Updates