Rezolute, Inc, a late-stage rare disease company focused on treating hypoglycemia caused by HI, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to its investigational therapy, ersodetug, for the treatment of hypoglycemia caused by tumor HI. 

BTD for ersodetug was granted based on clinical trial data across the overall program and a recognition of the mechanistic applicability to tumor HI, further validated by real-world experience in tumor HI patients who have been successfully treated with ersodetug throughout the U.S. in the Company's Expanded Access Program. BTD is intended to expedite the development and regulatory review of therapies for serious or life-threatening conditions where preliminary clinical evidence indicates the potential for substantial improvement over existing treatment options. 

“This designation highlights FDA’s recognition of ersodetug’s potential therapeutic benefit in this life-threatening condition where the current standard of care is often insufficient to manage persistent hypoglycemia, particularly when it is refractory or an impediment to surgery or other tumor-directed therapies,” said Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute. “We believe that this is a validation of ersodetug’s unique mechanism that enables the treatment of various forms of hyperinsulinism and the success that we have observed in treating patients with tumor HI over the last two years.” 

Rezolute plans to initiate a registrational study of ersodetug in patients with tumor HI in the middle of 2025, with topline results anticipated in the second half of 2026. In parallel and utilizing its BTD, the Company plans to engage further with FDA to discuss the registrational trial, including the necessary data package to support a BLA filing and potential approval for the tumor HI indication, as an expansion of the congenital HI indication.   

Earlier this year, the Company announced that BTD was granted to ersodetug for the treatment of hypoglycemia caused by congenital HI, a separate late-stage program currently progressing in an ongoing Pase 3 clinical study.  

Tumor hyperinsulinism (HI) is a rare disease that may be caused by two distinct types of tumors: islet cell tumors (ICTs) and non-islet cell tumors (NICTs), both of which lead to hypoglycemia as a result of excessive activation of the insulin receptor. Insulinomas are the most common type of ICT and may cause hypoglycemia by stimulating the over production of insulin. A variety of different NICTs, particularly hepatocellular carcinoma, can cause hypoglycemia by producing and secreting insulin-like paraneoplastic substances such as IGF-2 that bind to and activate the insulin receptor. With high morbidity and mortality rates within tumor HI, there remains a significant unmet need for new therapies directed at hypoglycemia treatment. Ersodetug has shown real-world benefit in patients with insulinoma and preclinical studies have shown that ersodetug can similarly blunt IGF-2 and insulin-mediated insulin-receptor signaling. 

Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor to decrease receptor over-activation by insulin and related substances (such as IGF-2) in the setting of hyperinsulinism (HI), thereby improving hypoglycemia. Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any congenital or acquired form of HI. 

Source: globenewswire.com