Establishing a New Standard of Care in Tenosynovial giant cell tumors (TSGCT)

Currently, surgical resection is the standard of care for TGCT. Localized-type TGCT is in most cases managed by direct removal of the tumor nodule. DTGCT (Diffused-type tenosynovial giant cell tumor) is normally seen as larger tumors affecting larger joints, and therefore, the management of these tumors is more complicated and often involves total synovectomy, joint replacement or, in very rare cases, amputation. Other potential treatment involves Radiation Therapy, Radiosynovectomy, and Systemic Therapies (CSF1 inhibitors and Tyrosine Kinase Inhibitors).

In August 2019, the US Food and Drug Administration (FDA) approved Daiichi Sankyo’s Turalio (pexidartinib) as the first and only treatment for adult patients with symptomatic TGCT associated with severe morbidity or functional limitations and is not amenable to improvement with surgery.

Moving onto the emerging therapies, TSGCT pipeline possesses potential drugs in late and mid-stage development, which includes SynOx Therapeutics’ Emactuzumab and Deciphera Pharmaceuticals’ Vimseltinib in Phase III trials, whereas AmMax Bio’s AMB-05X is all set for a revelation during the conference. The overall clinical profile of intra-articular AMB-05X aims to address a huge unmet need by offering an effective treatment with a noticeably more acceptable side effect profile. AMB-05X showed evidence of clinical benefit, including tumor reduction based on RECIST criteria. In terms of current treatments, there aren't many other options besides surgery, which has its risks and post-recovery health issues. By providing an efficient treatment with a significantly more tolerable side effect profile, the overall clinical profile of intra-articular AMB-05X looks to answer this substantial unmet need. AmMax also intends to start a Phase II clinical trial for idiopathic pulmonary fibrosis in response to promising preclinical proof-of-concept findings. It will be interesting to watch the progressive outcome of AMB-05X at ESMO 2022.