DelveInsight's comprehensive 2025 pipeline analysis reveals a thriving thyroid cancer therapeutic landscape, with over 50 companies actively developing 51+ pipeline therapies across various stages of clinical development. The report highlights significant momentum in both differentiated and anaplastic thyroid cancer treatment approaches.
Major Clinical Trial Developments
Novartis Pharmaceuticals announced on August 13, 2025, a Phase III study evaluating the combination of dabrafenib plus trametinib in adult patients with locally advanced or metastatic BRAFV600E mutation-positive, differentiated thyroid cancer. The study targets patients who are refractory to radioactive iodine and have progressed following prior VEGFR targeted therapy. Approximately 150 patients will be randomized in a 2:1 ratio to receive either the combination therapy or placebo.
Children's Hospital of Philadelphia simultaneously launched a study on the same date to evaluate the efficacy and safety of larotrectinib followed by 131I therapy for patients with NTRK fusion differentiated thyroid cancer. The primary Phase II objective focuses on evaluating the pulmonary structural response rate at 18 months to the combination of larotrectinib administered for 6 months followed by 131I therapy.
Innovative Therapeutic Approaches
CAR-T Cell Therapy Advancement
AffyImmune Therapeutics is developing AIC100, an affinity-tuned, ICAM-1 targeted, third-generation CAR-T cell therapy currently in Phase I clinical trials. The therapy features a CAR with affinity to ICAM-1 similar to naturally-occurring T cells, significantly lower than most existing CARs, which reduces on-target off-tumor toxicities. AIC100 also expresses SSTR2 for real-time monitoring of CAR-T cell distribution and activity. Preclinical studies demonstrate robust and enduring tumor eradication without toxicity-related relapse or fatality across various solid tumor models.
Novel BRAF Inhibition
Suzhou NeuPharma's RX208 represents a potential "best-in-class" BRAF V600E inhibitor with a proprietary novel chemical structure distinct from other marketed BRAF inhibitors. The compound exhibited high bioavailability and excellent anti-tumor efficacy in preclinical studies, with early clinical data demonstrating preliminary efficacy and good safety and tolerability profiles in cancer patients.
Established Therapies in Development
Bristol-Myers Squibb's nivolumab, a human immunoglobulin G4 monoclonal antibody that blocks PD-1 receptor interaction with PD-L1 and PD-L2, is currently being evaluated in Phase II clinical trials for thyroid cancer treatment. The therapy releases PD-1 pathway-mediated inhibition of immune response, including anti-tumor immune response.
Suzhou Zelgen Biopharmaceuticals is advancing recombinant human TSH (rhTSH) through Phase III clinical trials. The therapy was developed to provide TSH stimulation without requiring thyroid hormone suppression therapy withdrawal and associated metabolic disturbances, addressing limitations of bovine TSH.
Anaplastic Thyroid Cancer Focus
The anaplastic thyroid cancer pipeline features 8+ companies developing 8+ therapies. Takeda's sapanisertib, a dual TORC 1/2 inhibitor, targets key survival mechanisms in KEAP1/NRF2-mutated tumor cells. The compound has demonstrated promising single-agent activity in patients with relapsed/refractory NRF2-mutated squamous non-small cell lung cancer and exhibits differential anti-tumor activity compared to rapalog inhibitors.
Shanghai Henlius Biotech's HLX208, a BRAF V600E small-molecule inhibitor, shows potential for treating various solid tumors and may be combined with the company's proprietary EGFR or PD-1 targeted antibodies to enhance treatment efficacy.
Pipeline Diversity and Administration Routes
The thyroid cancer pipeline encompasses diverse therapeutic modalities, including monoclonal antibodies, small molecules, and peptides. Administration routes span intravenous, subcutaneous, oral, and intramuscular delivery methods, providing flexibility in treatment approaches.
Leading companies in the space include AstraZeneca, Verastem Oncology, Thryv Therapeutics, Molecular Targeting Technologies, Chimerix, Codiak BioSciences, LeadArtis, Tyra Biosciences, Roche, Sichuan Kelun-Biotech Biopharmaceutical, and Advenchen Laboratories, among others.
The robust pipeline activity reflects growing investment in thyroid cancer therapeutics, with companies pursuing various mechanisms of action and combination strategies to address unmet medical needs in both differentiated and anaplastic thyroid cancer patient populations.
