FDA Grants Orphan Drug Designation to Rezolute’s Ersodetug (RZ358) for the Treatment of Hypoglycemia Due to Tumor Hyperinsulinism
Rhea-AI Summary
Rezolute (Nasdaq: RZLT) announced that the FDA has granted Orphan Drug Designation to ersodetug for treating hypoglycemia due to tumor hyperinsulinism (HI). The company plans to initiate a Phase 3 registrational trial for ersodetug in tumor HI patients in 2025. The FDA's designation offers seven years of market exclusivity and potential expedited approval pathways. Ersodetug aims to provide a universal treatment for hypoglycemia caused by all forms of HI, addressing a serious unmet medical need and enabling patients to continue cancer treatments while managing hypoglycemia.
Positive
- FDA granted Orphan Drug Designation, providing 7 years market exclusivity
- Potential expedited approval pathway through FDA designation
- Phase 3 trial planned for 2025, advancing clinical development
Negative
- Phase 3 trial not starting until 2025, indicating extended timeline to potential market
News Market Reaction – RZLT
On the day this news was published, RZLT gained 4.90%, reflecting a moderate positive market reaction.
Data tracked by StockTitan Argus on the day of publication.
Phase 3 registrational trial for ersodetug in patients with tumor hyperinsulinism (HI) expected to commence in 2025; real-world patient benefit demonstrated in Expanded Access Program
REDWOOD CITY, Calif., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a late-stage biopharmaceutical company dedicated to developing transformative therapies for rare diseases with serious unmet needs, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ersodetug for the treatment of hypoglycemia due to tumor HI.
"FDA's granting of Orphan Drug Designation is a recognition of the serious unmet need patients with tumor hyperinsulinism face and validates the potential significant benefit that ersodetug can provide," said Susan Stewart, J.D, Chief Regulatory Officer of Rezolute. "Tumor HI requires directed treatments for hypoglycemia to prevent serious adverse outcomes and improve patients’ quality of life, as well as to enable patients to continue cancer treatments. We are thrilled with FDA’s designation, which allows us to continue developing a potential universal treatment for hypoglycemia caused by all forms of HI.”
The FDA's Orphan Drug Designation is intended to encourage the development of treatments for rare diseases. Orphan Drug Designation offers benefits including seven years of market exclusivity protection and may shorten the clinical development path through potential qualification for expedited pathways to approval.
About Tumor Hyperinsulinism (HI)
Tumor 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.
About Ersodetug
Ersodetug is a fully human monoclonal antibody that binds to a unique allosteric site on insulin receptors to counteract the effects of insulin receptor over-activation by insulin and related substances (such as IGF-2), thereby improving hypoglycemia in the setting of hyperinsulinism (HI). Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any form of HI.
About Rezolute, Inc.
Rezolute is a late-stage rare disease company focused on significantly improving outcomes for individuals with hypoglycemia caused by hyperinsulinism (HI). The Company’s antibody therapy, ersodetug, is designed to treat all forms of HI and has shown substantial benefit in clinical trials and real-world use for the treatment of congenital HI and tumor HI. For more information, visit www.rezolutebio.com.
Forward-Looking Statements
This release, like many written and oral communications presented by Rezolute and our authorized officers, may contain certain forward-looking statements regarding our prospective performance and strategies within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of 1934, as amended. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and are including this statement for purposes of said safe harbor provisions. Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, and expectations of Rezolute, are generally identified by use of words such as "anticipate," "believe," "estimate," "expect," "intend," "plan," "project," "seek," "strive," "try," “potential,” or future or conditional verbs such as "could," "may," "should," "will," "would," or similar expressions. These forward-looking statements include but are not limited to statements regarding the ersodetug Expanded Access Program, the ability of ersodetug to become an effective treatment for congenital hyperinsulinism, the effectiveness or future effectiveness of ersodetug for the treatment of congenital hyperinsulinism, statements regarding clinical trial timelines for ersodetug, the timing of the Phase 3 registration trial, and the FDA’s Orphan Drug Designation as it relates to ersodetug. Our ability to predict results or the actual effects of our plans or strategies is inherently uncertain. Accordingly, actual results may differ materially from anticipated results. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release. Except as required by applicable law or regulation, Rezolute undertakes no obligation to update these forward-looking statements to reflect events or circumstances that occur after the date on which such statements were made. Important factors that may cause such a difference include any other factors discussed in our filings with the SEC, including the Risk Factors contained in the Rezolute’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, which are available on the SEC’s website at www.sec.gov. You are urged to consider these factors carefully in evaluating the forward-looking statements in this release and are cautioned not to place undue reliance on such forward-looking statements, which are qualified in their entirety by this cautionary statement.
Contact:
Rezolute, Inc.
Christen Baglaneas
cbaglaneas@rezolutebio.com
508-272-6717
FAQ
What is the FDA Orphan Drug Designation granted to Rezolute (RZLT)?
When will Rezolute (RZLT) begin Phase 3 trials for ersodetug?
What benefits does the FDA Orphan Drug Designation provide to Rezolute (RZLT)?
What condition does Rezolute's (RZLT) ersodetug treat?
