Rezolute Provides Update on its Phase 3 sunRIZE Study of Ersodetug for the Treatment of Hypoglycemia Due to Congenital Hyperinsulinism
Rezolute (NASDAQ: RZLT) announced key updates for its Phase 3 sunRIZE study of ersodetug, treating hypoglycemia due to congenital hyperinsulinism. The Data Monitoring Committee (DMC) reviewed the open-label arm involving 8 infant participants aged 3 months to 1 year, confirming that target drug concentrations were safely achieved at tested doses of 5 or 10 mg/kg.
Key findings show ersodetug was generally safe and well-tolerated in infants, with drug levels comparable to effective exposures in older pediatric participants from the Phase 2b RIZE study. The DMC approved infant enrollment in the double-blind portion of the study.
Study enrollment completion is expected in Q2 2025, with topline results in Q4 2025, subject to an upcoming interim analysis. The interim analysis, scheduled for early Q2 2025, could recommend continuing the study as planned, increasing the sample size by 33%, or stopping for futility. If expanded, enrollment would extend to Q4 2025 with results by mid-2026.
Rezolute (NASDAQ: RZLT) ha annunciato aggiornamenti chiave per il suo studio di Fase 3 sunRIZE relativo a ersodetug, un trattamento per l'ipoglicemia dovuta a iperinsulinemia congenita. Il Comitato per il Monitoraggio dei Dati (DMC) ha esaminato il braccio open-label che coinvolge 8 partecipanti infantili di età compresa tra 3 mesi e 1 anno, confermando che le concentrazioni target del farmaco sono state raggiunte in sicurezza alle dosi testate di 5 o 10 mg/kg.
I risultati principali mostrano che ersodetug è stato generalmente sicuro e ben tollerato nei neonati, con livelli di farmaco comparabili alle esposizioni efficaci nei partecipanti pediatrici più grandi dello studio RIZE di Fase 2b. Il DMC ha approvato l'arruolamento di neonati nella parte doppio cieco dello studio.
Il completamento dell'arruolamento dello studio è previsto per il secondo trimestre del 2025, con risultati preliminari attesi nel quarto trimestre del 2025, soggetti a un'analisi intermedia imminente. L'analisi intermedia, programmata per l'inizio del secondo trimestre del 2025, potrebbe raccomandare di continuare lo studio come pianificato, aumentando la dimensione del campione del 33%, o di fermarsi per insufficienza. Se espanso, l'arruolamento sarebbe esteso al quarto trimestre del 2025 con risultati entro la metà del 2026.
Rezolute (NASDAQ: RZLT) anunció actualizaciones clave para su estudio de Fase 3 sunRIZE sobre ersodetug, un tratamiento para la hipoglucemia debida a hiperinsulinemia congénita. El Comité de Monitoreo de Datos (DMC) revisó el grupo de etiqueta abierta que involucra a 8 participantes infantiles de entre 3 meses y 1 año, confirmando que se lograron de manera segura las concentraciones de fármaco objetivo a las dosis probadas de 5 o 10 mg/kg.
Los hallazgos clave muestran que ersodetug fue generalmente seguro y bien tolerado en los lactantes, con niveles de fármaco comparables a exposiciones efectivas en participantes pediátricos mayores del estudio RIZE de Fase 2b. El DMC aprobó el reclutamiento de lactantes en la parte de doble ciego del estudio.
Se espera completar el reclutamiento del estudio en el segundo trimestre de 2025, con resultados preliminares para el cuarto trimestre de 2025, sujetos a un próximo análisis intermedio. El análisis intermedio, programado para principios del segundo trimestre de 2025, podría recomendar continuar el estudio según lo planeado, aumentando el tamaño de la muestra en un 33%, o detenerse por futilidad. Si se expande, el reclutamiento se extendería hasta el cuarto trimestre de 2025 con resultados a mediados de 2026.
Rezolute (NASDAQ: RZLT)는 선천성 고인슐린혈증으로 인한 저혈당 치료를 위한 ersodetug의 3상 sunRIZE 연구의 주요 업데이트를 발표했습니다. 데이터 모니터링 위원회(DMC)는 3개월에서 1세 사이의 영아 8명을 포함하는 오픈 라벨 군을 검토하여 5 또는 10 mg/kg의 테스트된 용량에서 목표 약물 농도가 안전하게 달성되었음을 확인했습니다.
주요 결과는 ersodetug이 영아에게 일반적으로 안전하고 잘 견뎌졌으며, 약물 수준이 2b RIZE 연구의 더 나이 많은 소아 참가자에서 효과적인 노출과 유사하다는 것을 보여줍니다. DMC는 연구의 이중 맹검 부분에 영아 등록을 승인했습니다.
연구 등록 완료는 2025년 2분기에 예상되며, 2025년 4분기에 주요 결과가 나올 것으로 보입니다. 이는 예정된 중간 분석에 따라 다릅니다. 2025년 2분기 초로 예정된 중간 분석은 연구를 계획대로 진행하거나 샘플 크기를 33% 증가시키거나 무용성으로 중단하는 것을 권장할 수 있습니다. 확장될 경우, 등록은 2025년 4분기까지 연장되며, 결과는 2026년 중반까지 나올 것입니다.
Rezolute (NASDAQ: RZLT) a annoncé des mises à jour clés pour son étude de Phase 3 sunRIZE concernant l'ersodetug, un traitement pour l'hypoglycémie due à l'hyperinsulinisme congénital. Le Comité de Surveillance des Données (DMC) a examiné le groupe à étiquette ouverte impliquant 8 participants âgés de 3 mois à 1 an, confirmant que les concentrations cibles du médicament ont été atteintes en toute sécurité aux doses testées de 5 ou 10 mg/kg.
Les résultats clés montrent que l'ersodetug était généralement sûr et bien toléré chez les nourrissons, avec des niveaux de médicament comparables aux expositions efficaces chez des participants pédiatriques plus âgés de l'étude RIZE de Phase 2b. Le DMC a approuvé l'inclusion des nourrissons dans la partie en double aveugle de l'étude.
La finalisation de l'inclusion des participants à l'étude est attendue au 2ème trimestre 2025, avec des résultats préliminaires au 4ème trimestre 2025, sous réserve d'une prochaine analyse intermédiaire. L'analyse intermédiaire, prévue pour début 2025 Q2, pourrait recommander de poursuivre l'étude comme prévu, d'augmenter la taille de l'échantillon de 33 % ou d'arrêter l'étude pour futilité. Si élargi, le recrutement s'étendra jusqu'au 4ème trimestre 2025 avec des résultats d'ici la mi-2026.
Rezolute (NASDAQ: RZLT) hat wichtige Aktualisierungen zu seiner Phase-3-Studie sunRIZE zu ersodetug bekannt gegeben, das zur Behandlung von Hypoglykämie aufgrund von kongenitaler Hyperinsulinismus eingesetzt wird. Das Data Monitoring Committee (DMC) prüfte die offene Behandlungsgruppe mit 8 Teilnehmern im Alter von 3 Monaten bis 1 Jahr und bestätigte, dass die Zielmedikamentenkonzentrationen bei den getesteten Dosen von 5 oder 10 mg/kg sicher erreicht wurden.
Wichtige Ergebnisse zeigen, dass ersodetug im Allgemeinen sicher und gut verträglich bei Säuglingen war, mit Medikamentenwerten, die mit wirksamen Expositionen bei älteren pädiatrischen Teilnehmern aus der Phase-2b-RIZE-Studie vergleichbar waren. Das DMC genehmigte die Rekrutierung von Säuglingen für den doppelblinden Teil der Studie.
Der Abschluss der Studienrekrutierung wird für das 2. Quartal 2025 erwartet, mit den vorläufigen Ergebnissen im 4. Quartal 2025, abhängig von einer bevorstehenden Zwischenanalyse. Die für Anfang des 2. Quartals 2025 geplante Zwischenanalyse könnte empfehlen, die Studie wie geplant fortzusetzen, die Stichprobengröße um 33 % zu erhöhen oder bei Nutzlosigkeit zu stoppen. Falls erweitert, würde die Rekrutierung bis zum 4. Quartal 2025 verlängert und die Ergebnisse Mitte 2026 vorliegen.
- DMC confirmed safety and efficacy of drug concentrations in infant participants
- Received FDA Breakthrough Therapy Designation
- Study sites expected to activate and begin enrollment this quarter
- Drug shows comparable effectiveness to Phase 2b RIZE study results
- Potential study extension could delay results to mid-2026 if sample size needs 33% increase
- Possibility of study termination for futility after interim analysis
Insights
The latest update on Rezolute's Phase 3 sunRIZE study presents several significant developments that strengthen the clinical profile of ersodetug. The successful completion of the open-label arm (OLA) with 8 infant participants has yielded important safety and pharmacokinetic data, demonstrating that the drug achieves target concentrations at both 5 and 10 mg/kg doses.
Three critical aspects deserve attention:
- The DMC's validation of safety in infants as young as 3 months is particularly noteworthy, as this age group represents a highly vulnerable patient population where safety concerns are paramount.
- The comparable drug exposure levels between infants and older pediatric participants from the Phase 2b RIZE study suggests consistent pharmacokinetics across age groups - a important factor for dosing confidence.
- The upcoming interim analysis presents a structured risk-mitigation approach, with the possibility of sample size adjustment (up to 33% increase) to enhance statistical power.
The recent Breakthrough Therapy Designation from the FDA adds significant regulatory momentum and could accelerate the pathway to potential approval. However, investors should note the timeline implications of the upcoming interim analysis: while the base case projects topline results in Q4 2025, a sample size increase would push results to mid-2026. This flexibility in trial design demonstrates statistical rigor but introduces timeline uncertainty that warrants careful consideration.
The congenital hyperinsulinism market currently lacks effective treatment options, making ersodetug's potential entry particularly significant. The drug's safety profile in infants, if maintained through study completion, could position it as a first-line treatment option in this rare but serious condition.
Open-label arm (infant participants < 1 year old) has been recently reviewed by a Data Monitoring Committee (DMC) and target drug concentrations were safely reached at tested doses
DMC approved enrollment of infants into the double-blind portion of the study
sunRIZE enrollment completion expected in Q2 2025, subject to outcomes from upcoming interim analysis
REDWOOD CITY, Calif., Feb. 04, 2025 (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 outcomes from an independent Data Monitoring Committee (DMC) review of the open label arm (OLA) portion of the sunRIZE Phase 3 study of ersodetug for the treatment of hypoglycemia due to congenital hyperinsulinism (HI) and provided additional study updates including guidance on its upcoming interim analysis (IA) by the DMC. This IA will assess the adequacy of the sample size for the primary endpoint and may recommend adjustments if necessary.
The open label arm of 8 infant participants ages 3 months to 1 year has been fully enrolled and accrued sufficient data for the DMC to review the safety and pharmacokinetics (PK) of ersodetug, which was administered at doses of 5 or 10 mg/kg over a bi-weekly loading phase followed by a monthly maintenance phase. The task of the DMC was to independently review the safety and PK data in infants for purposes of dose confirmation, continued benefit-risk determinations, and the appropriateness of opening enrollment of the double-blind portion of the study to infant participants at the dose regimen under study. The DMC did not review glycemic efficacy and the Company remains blinded to glucose results and efficacy outcomes.
Findings from the DMC include the following:
- Ersodetug was generally safe and well-tolerated in participants 3 months to 1 year of age.
- Observed ersodetug drug levels at peak and trough were comparable to exposures in older pediatric participants in the Phase 2b RIZE study, which were demonstrated to be effective.
- Safety and exposures validate the chosen dose regimen of 5 and 10 mg/kg administered bi-weekly and monthly.
- Subsequent infant participants may now be enrolled into the double-blind, placebo-controlled study.
“We are encouraged by these preliminary outcomes from the open label arm of the sunRIZE study, particularly as it is a strong indication of the safety profile of ersodetug and provides additional validation of the selected doses in very young participants,” said Brian Roberts, M.D., Chief Medical Officer at Rezolute. “Having recently received Breakthrough Therapy Designation from the FDA, 2025 has already been an exciting year for Rezolute and we look forward to additional milestones over the course of this year as we progress in our mission to provide a safe and effective therapy for people living with hyperinsulinism.”
Study start up activities in the U.S. have progressed and sites are expected to be activated and enrolling this quarter. Study enrollment completion is anticipated in the second quarter of 2025 and topline results are expected in the fourth quarter of 2025, pending recommendations from the DMC based on an upcoming IA.
An IA of the study primary hypoglycemia endpoint is being conducted following the accumulation of sufficient patient data and is designed to optimize the study sample size and statistical confidence in the final analysis outcomes. The IA will be conducted at the end of this quarter and the Company plans to announce the conclusions of the DMC early in the second quarter of 2025 while also providing additional overall study updates. There are three possible outcomes from the analysis: (i) futility and the study should be stopped, (ii) continue the study as is or (iii) continue the study as is but increase the sample size by
If the study sample size is increased, the Company estimates completion of enrollment would shift to the fourth quarter of 2025 and that top line results would be available in mid-2026. Other than for futility, the outcomes of the IA should not be viewed as a read-through to end of study efficacy, either favorably or unfavorably.
For more information on our pipeline and the sunRIZE study, please visit www.rezolutebio.com/for-patients/.
About Congenital Hyperinsulinism
Congenital hyperinsulinism (HI) is the most common cause of recurrent and persistent hypoglycemia in children. Patients with congenital HI typically present with signs or symptoms of hypoglycemia within the first month of life. These episodes can result in significant brain injury and death if not recognized and managed appropriately. Additionally, recurrent, or cumulative, hypoglycemia can lead to progressive and irreversible damage over time, including serious and devastating brain injury, seizures, neuro-developmental problems, feeding difficulties, and significant impact on patient and family quality of life. In cases of congenital HI that are unresponsive to medical management, surgical removal of the pancreas may be required. More than half of children with congenital HI require long-term medical treatment for hypoglycemia that is not addressed by available therapies.
About Ersodetug
Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor to counteract the effects of insulin receptor over-activation by insulin and related substances (such as IGF-2), thereby shifting over-signaling back into a more normalized range and 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 congenital or acquired form of HI.
About sunRIZE
The Phase 3 sunRIZE study is a multi-center, randomized, double-blind, placebo-controlled, parallel arm study designed to evaluate the efficacy and safety of ersodetug in patients with congenital HI who are experiencing poorly controlled hypoglycemia. Participants between the ages of 3 months to 45 years old are eligible to participate. The study is enrolling up to 56 participants in more than a dozen countries around the world.
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 efficacy of ersodetug in patient populations of 3 months to 1 year of age, the DMC approval of enrollment of infants into a double-blind control study of ersodetug, the FDA’s grant of the Breakthrough Therapy Designation for ersodetug, 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 sunRIZE study. 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 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
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