Rezolute Receives Breakthrough Therapy Designation from FDA for Ersodetug in the Treatment of Hypoglycemia Due to Tumor Hyperinsulinism
The company plans to initiate a registrational study for tumor HI patients in mid-2025, with topline results expected in H2 2026. Rezolute will engage with the FDA to discuss the registrational trial and requirements for a BLA filing, aiming to expand ersodetug's indication beyond congenital HI.
The BTD status acknowledges ersodetug's potential to offer substantial improvement over existing treatments for this serious condition, where current standard care often fails to manage persistent hypoglycemia effectively.
L'azienda prevede di avviare uno studio registrativo per pazienti con HI tumorale a metà 2025, con risultati preliminari attesi nella seconda metà del 2026. Rezolute collaborerà con la FDA per discutere lo studio registrativo e i requisiti per la presentazione di una BLA, con l'obiettivo di estendere l'indicazione di ersodetug oltre l'HI congenito.
Lo status BTD riconosce il potenziale di ersodetug di offrire un miglioramento significativo rispetto ai trattamenti attuali per questa grave condizione, dove le terapie standard spesso non riescono a gestire efficacemente l'ipoglicemia persistente.
La empresa planea iniciar un estudio registracional para pacientes con HI tumoral a mediados de 2025, con resultados preliminares esperados en la segunda mitad de 2026. Rezolute colaborará con la FDA para discutir el ensayo registracional y los requisitos para la presentación de una BLA, con el objetivo de ampliar la indicación de ersodetug más allá del HI congénito.
El estatus BTD reconoce el potencial de ersodetug para ofrecer una mejora sustancial frente a los tratamientos existentes para esta grave condición, donde la atención estándar actual a menudo no logra controlar eficazmente la hipoglucemia persistente.
회사는 2025년 중반에 종양성 HI 환자를 대상으로 하는 등록 연구를 시작할 계획이며, 주요 결과는 2026년 하반기에 발표될 예정입니다. Rezolute는 등록 시험 및 BLA 제출 요건에 대해 FDA와 협의하여 에르소데터그의 적응증을 선천성 HI를 넘어 확장하는 것을 목표로 하고 있습니다.
BTD 상태는 현재 표준 치료가 지속적인 저혈당을 효과적으로 관리하지 못하는 심각한 상태에서 에르소데터그가 기존 치료법보다 상당한 개선을 제공할 잠재력이 있음을 인정하는 것입니다.
L'entreprise prévoit de lancer une étude d'enregistrement pour les patients atteints d'HI tumoral à la mi-2025, avec des résultats préliminaires attendus au second semestre 2026. Rezolute travaillera avec la FDA pour discuter de l'essai d'enregistrement et des exigences pour le dépôt d'une demande d'autorisation de mise sur le marché (BLA), visant à étendre l'indication d'ersodetug au-delà de l'HI congénital.
Le statut BTD reconnaît le potentiel d'ersodetug à offrir une amélioration substantielle par rapport aux traitements existants pour cette maladie grave, où les soins standards actuels échouent souvent à gérer efficacement l'hypoglycémie persistante.
Das Unternehmen plant, Mitte 2025 eine registrierende Studie für Patienten mit tumorbedingtem HI zu starten, mit erwarteten Topline-Ergebnissen in der zweiten Hälfte 2026. Rezolute wird sich mit der FDA abstimmen, um die registrierende Studie und die Anforderungen für eine BLA-Einreichung zu besprechen, mit dem Ziel, die Indikation von Ersodetug über das angeborene HI hinaus auszuweiten.
Der BTD-Status erkennt das Potenzial von Ersodetug an, eine erhebliche Verbesserung gegenüber bestehenden Behandlungen für diese schwere Erkrankung zu bieten, bei der die derzeitige Standardtherapie oft nicht in der Lage ist, die anhaltende Hypoglykämie effektiv zu kontrollieren.
- Received FDA Breakthrough Therapy Designation for ersodetug in tumor hyperinsulinism
- Second BTD received for ersodetug (also granted for congenital HI)
- Successful real-world treatment experience through Expanded Access Program
- Clear development timeline with registrational study starting mid-2025 and topline results expected H2 2026
- Results from registrational study won't be available until late 2026
- Still requires successful completion of trials and FDA approval before commercialization
Insights
FDA's second BTD for ersodetug accelerates development for tumor hyperinsulinism, strengthening Rezolute's position in rare disease treatment.
The Breakthrough Therapy Designation for ersodetug represents a significant regulatory milestone that substantially accelerates Rezolute's development timeline. This designation is granted only when preliminary evidence suggests substantial improvement over existing therapies for serious conditions, offering several advantages: intensive FDA guidance, senior management involvement, and eligibility for expedited review pathways.
Particularly noteworthy is that this marks Rezolute's second BTD for ersodetug, following their earlier designation for congenital hyperinsulinism. This dual-indication approach creates regulatory efficiencies as the company plans to leverage their existing data package.
The company's strategy to engage with FDA about using their congenital HI data to support the tumor HI indication demonstrates a sophisticated regulatory approach. Their planned registrational study timeline (mid-2025 start with results by H2 2026) appears relatively compressed, benefiting from the expedited review considerations of BTD status.
The successful treatment outcomes in their Expanded Access Program provided compelling real-world evidence that complemented their clinical trial data in securing this designation, offering additional validation of the drug's clinical utility beyond controlled trial environments.
Ersodetug addresses critical treatment gap for tumor hyperinsulinism patients facing life-threatening hypoglycemia with few effective options.
Tumor hyperinsulinism presents a complex clinical challenge where pancreatic tumors secrete excess insulin, causing dangerous hypoglycemic episodes that can lead to seizures, brain damage, and potentially death. Current standard of care is notably insufficient, especially for patients with inoperable tumors or those awaiting surgery.
Traditional management relies on dietary modifications, diazoxide (with significant side effects), and surgical resection when possible. For patients with malignant insulinomas or metastatic disease, surgical intervention may be insufficient or contraindicated, leaving them with limited effective options.
The FDA's BTD recognition acknowledges ersodetug's potential to address this significant unmet need. Particularly important is the drug's potential to manage hypoglycemia that impedes surgery or other tumor-directed therapies, suggesting it could function both as a standalone treatment and as a complementary therapy enabling interventions otherwise complicated by severe hypoglycemia.
The success observed treating tumor HI patients through the Expanded Access Program over two years provides important validation of ersodetug's clinical utility. For this patient population with limited treatment options, a therapy specifically addressing the metabolic complications represents a significant advancement in care.
Registrational study in patients with tumor hyperinsulinism (HI) expected to commence mid-year
Designation underscores need for therapies to treat severe hypoglycemia in the oncology setting
REDWOOD CITY, Calif., May 05, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), 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.
About Tumor Hyperinsulinism
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.
About Ersodetug
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.
About Rezolute, Inc.
Rezolute is a late-stage rare disease company focused on treating 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 its authorized officers, may contain certain forward-looking statements regarding our prospective performance and within the meaning of Section 27A of the Securities Act of 1933, as amended, 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," 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 ability of ersodetug to be an effective treatment for tumor HI as well as statements regarding the timing of the registrational study of ersodetug and the timing of topline results. 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 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 by 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.
Contacts
Rezolute, Inc.
Christen Baglaneas
cbaglaneas@rezolutebio.com
(508) 272-6717
Media
Sarah Lima
Sarah@GalvinPR.com
(774) 766-0200
FAQ
What is the significance of RZLT receiving Breakthrough Therapy Designation for ersodetug?
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What evidence supported the FDA's Breakthrough Therapy Designation for RZLT's ersodetug?
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