Investigational Rinatabart Sesutecan (Rina-S®) Continues to Show Encouraging Antitumor Activity in Patients with Advanced Ovarian Cancer
Genmab A/S (GMAB) reported updated data from Phase 1/2 RAINFOL-01 study of rinatabart sesutecan (Rina-S®), showing promising results in treating advanced ovarian cancer. With a median follow-up of 48 weeks, Rina-S at 120 mg/m2 demonstrated:
- Confirmed objective response rate (ORR) of 55.6%
- Disease control rate (DCR) of 88.9%
- Median duration of response (mDOR) not reached
- Complete responses in 4 patients (2 confirmed, 2 unconfirmed)
- Confirmed partial responses in 8 patients (44.4%)
At 100 mg/m2, the study showed a 22.7% ORR and 86.4% DCR. Common side effects included anemia, nausea, and neutropenia. The 120 mg/m2 dose has been selected for further evaluation in ongoing Phase 2 RAINFOL-01 and Phase 3 RAINFOL-02 trials for platinum-resistant ovarian cancer (PROC).
Genmab A/S (GMAB) ha riportato dati aggiornati dallo studio di Fase 1/2 RAINFOL-01 su rinatabart sesutecan (Rina-S®), mostrando risultati promettenti nel trattamento del cancro ovarico avanzato. Con un follow-up mediano di 48 settimane, Rina-S a 120 mg/m2 ha dimostrato:
- Un tasso di risposta obiettiva confermata (ORR) del 55,6%
- Un tasso di controllo della malattia (DCR) dell'88,9%
- Una durata mediana della risposta (mDOR) non raggiunta
- Risposte complete in 4 pazienti (2 confermate, 2 non confermate)
- Risposte parziali confermate in 8 pazienti (44,4%)
A 100 mg/m2, lo studio ha mostrato un ORR del 22,7% e un DCR dell'86,4%. Gli effetti collaterali comuni includevano anemia, nausea e neutropenia. La dose di 120 mg/m2 è stata selezionata per ulteriori valutazioni negli studi in corso di Fase 2 RAINFOL-01 e Fase 3 RAINFOL-02 per il cancro ovarico resistente al platino (PROC).
Genmab A/S (GMAB) informó datos actualizados del estudio de Fase 1/2 RAINFOL-01 sobre rinatabart sesutecan (Rina-S®), mostrando resultados prometedores en el tratamiento del cáncer de ovario avanzado. Con un seguimiento medio de 48 semanas, Rina-S a 120 mg/m2 demostró:
- Tasa de respuesta objetiva confirmada (ORR) del 55,6%
- Tasa de control de la enfermedad (DCR) del 88,9%
- Duración media de la respuesta (mDOR) no alcanzada
- Respuestas completas en 4 pacientes (2 confirmadas, 2 no confirmadas)
- Respuestas parciales confirmadas en 8 pacientes (44,4%)
A 100 mg/m2, el estudio mostró un ORR del 22,7% y un DCR del 86,4%. Los efectos secundarios comunes incluyeron anemia, náuseas y neutropenia. La dosis de 120 mg/m2 ha sido seleccionada para una evaluación adicional en los ensayos en curso de Fase 2 RAINFOL-01 y Fase 3 RAINFOL-02 para el cáncer de ovario resistente al platino (PROC).
Genmab A/S (GMAB)는 rinatabart sesutecan (Rina-S®)에 대한 1/2상 RAINFOL-01 연구의 업데이트된 데이터를 발표하였으며, 진행성 난소암 치료에서 유망한 결과를 보여주었습니다. 48주간의 중간 추적 관찰 결과, Rina-S 120 mg/m2에서 다음과 같은 결과가 나타났습니다:
- 확인된 객관적 반응률(ORR) 55.6%
- 질병 조절률(DCR) 88.9%
- 반응의 중간 지속기간(mDOR) 도달하지 못함
- 4명의 환자에서 완전 반응(2명 확인, 2명 미확인)
- 8명의 환자에서 확인된 부분 반응(44.4%)
100 mg/m2에서 연구는 22.7%의 ORR과 86.4%의 DCR을 보여주었습니다. 일반적인 부작용으로는 빈혈, 메스꺼움 및 호중구감소증이 포함되었습니다. 120 mg/m2 용량은 백금 내성 난소암(PROC)에 대한 진행 중인 2상 RAINFOL-01 및 3상 RAINFOL-02 시험에서 추가 평가를 위해 선택되었습니다.
Genmab A/S (GMAB) a rapporté des données mises à jour de l'étude de Phase 1/2 RAINFOL-01 sur rinatabart sesutecan (Rina-S®), montrant des résultats prometteurs dans le traitement du cancer de l'ovaire avancé. Avec un suivi médian de 48 semaines, Rina-S à 120 mg/m2 a démontré :
- Taux de réponse objective confirmée (ORR) de 55,6%
- Taux de contrôle de la maladie (DCR) de 88,9%
- Durée médiane de la réponse (mDOR) non atteinte
- Réponses complètes chez 4 patients (2 confirmées, 2 non confirmées)
- Réponses partielles confirmées chez 8 patients (44,4%)
À 100 mg/m2, l'étude a montré un ORR de 22,7% et un DCR de 86,4%. Les effets secondaires courants comprenaient l'anémie, les nausées et la neutropénie. La dose de 120 mg/m2 a été sélectionnée pour une évaluation supplémentaire dans les essais en cours de Phase 2 RAINFOL-01 et de Phase 3 RAINFOL-02 pour le cancer de l'ovaire résistant au platine (PROC).
Genmab A/S (GMAB) hat aktualisierte Daten aus der Phase 1/2-Studie RAINFOL-01 zu rinatabart sesutecan (Rina-S®) veröffentlicht, die vielversprechende Ergebnisse bei der Behandlung von fortgeschrittenem Eierstockkrebs zeigen. Bei einer medianen Nachbeobachtungszeit von 48 Wochen zeigte Rina-S bei 120 mg/m2:
- Eine bestätigte objektive Ansprechrate (ORR) von 55,6%
- Eine Krankheitskontrollrate (DCR) von 88,9%
- Die mediane Ansprechdauer (mDOR) wurde nicht erreicht
- Vollständige Antworten bei 4 Patienten (2 bestätigt, 2 unbestätigt)
- Bestätigte partielle Antworten bei 8 Patienten (44,4%)
Bei 100 mg/m2 zeigte die Studie eine ORR von 22,7% und eine DCR von 86,4%. Häufige Nebenwirkungen umfassten Anämie, Übelkeit und Neutropenie. Die Dosis von 120 mg/m2 wurde für eine weitere Bewertung in den laufenden Phase-2-RAINFOL-01- und Phase-3-RAINFOL-02-Studien für platinresistenten Eierstockkrebs (PROC) ausgewählt.
- Strong efficacy with 55.6% objective response rate at 120 mg/m2 dose
- High disease control rate of 88.9% in advanced ovarian cancer patients
- Early response observed at week 6 in most patients
- Low frequency of dose reductions and treatment discontinuations
- Treatment-emergent adverse events affecting multiple body systems
- Lower efficacy (22.7% ORR) observed with 100 mg/m2 dose
Insights
The data from Genmab's rinatabart sesutecan (Rina-S) Phase 1/2 trial demonstrates remarkably strong efficacy signals in a notoriously difficult-to-treat population. The
What's most compelling is the durability of responses - with 48 weeks of follow-up, the median duration of response hasn't been reached, suggesting sustained benefit. The
The response pattern is particularly encouraging - most occurred early (week 6) and only 1 of 10 patients experienced disease progression during the follow-up period. Additionally, efficacy independent of FRα expression levels potentially broadens the eligible patient population, unlike some competitor therapies requiring biomarker selection.
While the cohort size (18 evaluable patients) is , these results provide strong justification for the ongoing Phase 3 RAINFOL-02 trial. The safety profile appears consistent with antibody-drug conjugate class effects, with manageable hematologic and gastrointestinal toxicities.
Genmab's rinatabart sesutecan data represents a significant value-creation opportunity in the company's clinical pipeline. The stark difference in efficacy between the 120 mg/m² dose (
Platinum-resistant ovarian cancer represents a substantial market opportunity with approximately 15,000 new cases annually in the US alone. Current treatment options show efficacy, creating a significant unmet need that Rina-S could potentially address.
The rapid advancement into Phase 3 trials indicates Genmab's confidence in this asset and suggests potential accelerated development timelines. The favorable safety profile, with infrequent dose reductions and discontinuations, further strengthens the drug's commercial prospects.
This ADC targets folate receptor-alpha (FRα), a clinically validated target in ovarian cancer. However, unlike some competitor therapies, Rina-S shows activity regardless of FRα expression levels, potentially expanding its addressable patient population.
For Genmab, successfully developing Rina-S would significantly diversify its commercial portfolio beyond its current CD38-focused pipeline and partnerships. With Phase 3 trials actively recruiting, we could see pivotal data within the next 18-24 months, representing a meaningful near-term catalyst.
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Results from Phase 2 RAINFOLTM-01 trial (B1 cohort) showed that with a median on-study follow-up of 48 weeks, Rina-S 120 mg/m2 led to a confirmed objective response rate (ORR) of
55.6% and median duration of response (mDOR) was not reached - Phase 2 RAINFOLTM-01 and Phase 3 RAINFOLTM-02 trials evaluating the safety and efficacy of Rina-S at 120 mg/m2 in patients with platinum resistant ovarian cancer (PROC) are actively recruiting
“The antitumor activity observed in the dose expansion cohort continues to demonstrate the potential for a much-needed treatment option for patients with PROC, who have historically had poor prognosis. I am hopeful that further exploration of Rina-S will lead to advancements in the treatment landscape.” said Elizabeth Lee, M.D., a medical oncologist in the gynecologic oncology program at Dana-Farber.
The B1 cohort is a dose expansion study in patients with histologically or cytologically confirmed advanced OC (epithelial ovarian cancer, primary peritoneal cancer or fallopian tube cancer). Rina-S 120 mg/m2 Q3W at a median on-study follow-up of 48 weeks showed encouraging antitumor activity; the confirmed ORR was
In this Phase 1/2 study, common treatment-emergent adverse events (TEAEs) included anemia, nausea, neutropenia, leukopenia, fatigue, thrombocytopenia, vomiting, diarrhea, alopecia, and hypokalemia. Dose reductions and treatment discontinuations were infrequent and no new safety signals were observed.
“The updated results reinforce the potential of Rina-S and further validate our development approach in advanced ovarian cancer,” said Judith Klimovsky, M.D., Executive Vice President and Chief Development Officer of Genmab. “We are excited to keep moving forward with the ongoing Phase 3 trial, to evaluate the potential of Rina-S as a treatment option for patients facing this challenging disease.”
The safety and efficacy of rinatabart sesutecan has not been established for these investigational uses.
About the RAINFOLTM -01 Trial
RAINFOL-01 (NCT05579366) is an open-label, multicenter Phase 1/2 study, designed to evaluate the safety and efficacy of rinatabart sesutecan (Rina-S) as a single agent Q3W at various doses in solid tumors that are known to express FRα. The study consists of multiple parts including Part A dose-escalation cohorts; Part B tumor-specific monotherapy dose-expansion cohorts; Part C platinum-resistant ovarian cancer (PROC) cohort; and Part D combination therapy cohorts.
Part B of the trial includes the B1 cohort, a dose expansion study in patients with histologically or cytologically confirmed advanced OC (epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer). Patients were randomized 1:1 to 100 mg/m2 and 120 mg/m2 dose cohorts. Median age was 62.5 and 64.5 years in the 100 mg/m2 and 120 mg/m2 cohorts, respectively. Study participants were previously treated with a median of 3 prior lines of therapy (range 1-4). Patients received prior treatment with bevacizumab (
About Ovarian Cancer
Ovarian cancer is a major global health issue, with over 320,000 new cases diagnosed annually worldwide.i It ranks as the eighth most common cancer and the eighth leading cause of cancer-related deaths among women globally.ii The disease is often diagnosed at an advanced stage due to its subtle and non-specific symptoms, such as abdominal bloating, pelvic pain and difficulty eating.iii Standard of care for platinum resistant ovarian cancer typically involves single agent chemotherapy (pegylated liposomal doxorubicin (PLD), topotecan, gemcitabine or paclitaxel). iv Approximately 70
About Rinatabart Sesutecan (Rina-S®; GEN1184)
Rinatabart sesutecan (Rina-S; GEN1184) is a FRα-targeted, TOPO1 ADC, currently being evaluated for the potential treatment of ovarian cancer and other FRα-expressing cancers. A Phase 3 trial (RAINFOL-02, NCT06619236) evaluating Rina-S in patients with platinum resistant ovarian cancer compared to treatment of investigator's choice is ongoing. In January 2024, the
Please visit www.clinicaltrials.gov for more information.
About Genmab
Genmab is an international biotechnology company with a core purpose of guiding its unstoppable team to strive toward improving the lives of patients with innovative and differentiated antibody therapeutics. For more than 25 years, its passionate, innovative and collaborative team has invented next-generation antibody technology platforms and leveraged translational, quantitative and data sciences, resulting in a proprietary pipeline including bispecific T-cell engagers, antibody-drug conjugates, next-generation immune checkpoint modulators and effector function-enhanced antibodies. By 2030, Genmab’s vision is to transform the lives of people with cancer and other serious diseases with knock-your-socks-off (KYSO) antibody medicines®.
Established in 1999, Genmab is headquartered in
This Media Release contains forward looking statements. The words “believe,” “expect,” “anticipate,” “intend” and “plan” and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with preclinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmab’s most recent financial reports, which are available on www.genmab.com and the risk factors included in Genmab’s most recent Annual Report on Form 20-F and other filings with the
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i World Cancer Research Fund International. https://www.wcrf.org/cancer-trends/ovarian-cancer-statistics/. Accessed August 2024.
ii World Ovarian Cancer Coalition. https://worldovariancancercoalition.org/about-ovarian-cancer/key-stats/. Accessed August 2024.
iii Dilley, James et al. Ovarian cancer symptoms, routes to diagnosis and survival - Population cohort study in the 'no screen' arm of the
iv Eskander RN, Moore KN, Monk BJ, Herzog TJ, Annunziata CM, O’Malley DM and Coleman RL (2023) Overcoming the challenges of drug development in platinum-resistant ovarian cancer. Front. Oncol. 13:1258228
Ovarian Cancer Research Alliance. https://ocrahope.org/patients/diagnosis-and-treatment/recurrence/.
v Ovarian Cancer Research Alliance. https://ocrahope.org/patients/diagnosis-and-treatment/recurrence/.
vi European Institute of Women's Health. https://eurohealth.ie/policy‐brief‐women‐and‐ovarian‐cancer‐in‐the‐eu‐2018/. Accessed August 2024.
vii American Cancer Society. Stages of Ovarian Cancer. https://www.cancer.org/cancer/types/ovarian-cancer/detection-diagnosis-staging/survival-rates.html. Accessed August 2024.
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FAQ
What are the latest efficacy results for Rina-S 120 mg/m2 in the RAINFOL-01 trial for ovarian cancer?
How does the 120 mg/m2 dose of GMAB's Rina-S compare to the 100 mg/m2 dose in ovarian cancer treatment?
What are the main side effects reported in the Rina-S RAINFOL-01 trial?
Which dose of Rina-S has been selected for Phase 3 RAINFOL-02 trial in platinum-resistant ovarian cancer?
