US FDA grants orphan drug designation to Avstera

Avstera Therapeutics Corp, an oncology-focused biotech dedicated to address the large disease burden and significant unmet need of cancer patients, has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Avstera's AVS100 compound, which is a highly specific HDAC6 inhibitor, for the treatment of stage IIB through IV melanoma. AVS100 is a novel highly specific HDAC6 inhibitor with unique mechanisms in its ability to polarize macrophages to the anti-tumoural M1 phenotype. Macrophage differentiation and stabilization of M1 is a key element in maintaining overall anticancer responses. AVS100 has been shown to significantly reduce tumour growth and improve overall survival in preclinical animal models. AVS100 remains on track for planned IND filing next year targeting locally advanced or metastatic solid tumours, including melanoma. "The FDA's grant of this ODD for AVS100 highlights the significant unmet need there remains for this patient population and the impact our therapy can have in potentially improving clinical outcomes. Advanced stage melanoma remains a complex and difficult cancer to treat with current approaches, and AVS100 has the potential to provide an opportunity to potentially reduce the disease burden shared by these patients." added Karthik Musunuri, CEO & co-founder of Avstera Therapeutics Corp. The research behind the HDAC6 inhibitor technology stems from Alejandro Villagra's lab at Georgetown; through licenses made with the George Washington University. "The use of selective HDAC6i treatment is an exciting approach to manipulate the tumour microenvironment and prime the host to increase the therapeutic response to checkpoint inhibitor therapy. This provides hope to patients who may have progressed on single agent treatment and are looking to boost response, or those unable to tolerate dual immunotherapy for refractory or more advanced cases." added Vishal Patel, MD, FAAD, FACMS, Director of the Cutaneous Oncology Programme at the George Washington Cancer Center and Member of Avstera's SAB. The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the US Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including a seven-year period of US marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act filing fees.

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