Topotecan Hydrochloride

COPENHAGEN, Denmark I March 17, 2025 I Genmab A/S (Nasdaq: GMAB) announced today updated data from cohort B1 of the Phase 1/2 RAINFOL-01 study of rinatabart sesutecan (Rina-S ® ), an investigational folate receptor-alpha (FRα)-targeted, TOPO1 antibody-drug conjugate (ADC) that showed Rina-S 120 mg/m 2 every 3 weeks (Q3W) resulted in a confirmed objective response rate (ORR) of 55.6% (95% CI: 30.8-78.5) in heavily pre-treated ovarian cancer (OC) patients regardless of FRα expression levels. With a median on-study follow-up of 48 weeks, 1 out of 10 patients experienced disease progression and the median duration of response (mDOR) was not reached (95% CI: 40.14-NR). The data are from the dose expansion cohort of the multi-part study evaluating the safety and efficacy of Rina-S as a single agent in solid tumors that are known to express FRα and were presented at the 2025 Society of Gynecologic Oncology Annual Meeting on Women’s Cancer ® (SGO) in Seattle, Washington. “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-Swill 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/m 2 Q3W at a median on-study follow-up of 48 weeks showed encouraging antitumor activity; the confirmed ORR was 55.6% (95% CI: 30.8-78.5), the disease control rate (DCR) was 88.9% (95% CI: 65.3-98.6), and the median duration of response (mDOR) was not reached (95% CI: 40.14-NR). In the 18 patients evaluable for response treated with 120 mg/m 2 Q3W, complete responses were observed in 4 patients (2 confirmed; 2 unconfirmed) and 8 patients experienced confirmed partial responses (44.4%). Most responses with Rina-S 120 mg/m 2 were observed early (at week 6). Only one patient in the 120 mg/m 2 treatment arm was not evaluable. In the Rina-S 100 mg/m 2 Q3W treatment arm (N=22), at a median on study follow-up of 46 weeks, the confirmed ORR was 22.7% (95% CI: 7.8-45.4), the DCR was 86.4% (95% CI: 65.1-97.1), and the mDOR was not reached (95% CI, 16.3-NR). Partial responses were observed in 4 patients (18.2%) and 1 patient (4.5%) experienced a complete response. Rina-S 120 mg/m 2 has been selected for further evaluation in the RAINFOL-01 and Phase 3 RAINFOL-02 trials for patients with platinum resistant ovarian cancer (PROC). 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 RAINFOL TM -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/m 2 and 120 mg/m 2 dose cohorts. Median age was 62.5 and 64.5 years in the 100 mg/m 2 and 120 mg/m 2 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 (90.9% in the 100 mg/m 2 group and 90.0% in the 120 mg/m 2 group respectively), PARP inhibitors (68.2%; 65%), and mirvetuximab soravtansin (18.2%; 19%). Initial results from Part B of this trial were presented during a mini-oral session at the European Society of Medical Oncology Congress 2024 (ESMO). 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 70-90% of women with advanced-stage ovarian cancer worldwide experience a recurrence after initial treatment. v Ovarian cancer has a low five-year survival rate, which varies significantly by region, but generally hovers around 30-50%. vi,vii 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 U.S. Food and Drug Administration granted Fast Track designation to Rina-S for the treatment of patients with FRα-expressing high-grade serous or endometrioid platinum-resistant ovarian cancer. 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 Copenhagen, Denmark, with international presence across North America, Europe and Asia Pacific. For more information, please visit Genmab.com and follow us on LinkedIn and X . 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 UK Collaborative Trial of Ovarian Cancer Screening (UKCTOCS). Gynecologic oncology vol. 158,2 (2020): 316-322. doi:10.1016/j.ygyno.2020.05.002. 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 . SOURCE: Genmab

Final data analysis from Phase 3 MIRASOL trial after 30.5-month median follow-up demonstrated 32% reduction in risk of death with ELAHERE compared to chemotherapy in folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer (PROC) Data presented in a late-breaking oral presentation at the Society of Gynecologic Oncology (SGO) Annual Meeting on Women's Cancer in Seattle NORTH CHICAGO, Ill., March 15, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the final analysis of the confirmatory Phase 3 MIRASOL trial evaluating the efficacy and safety of ELAHERE® (mirvetuximab soravtansine-gynx) in women with folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer (PROC) compared to chemotherapy. At 30.5 months median follow-up, treatment with ELAHERE continued to show significant improvements in progression-free survival (PFS) and overall survival (OS) compared to investigator's choice (IC) chemotherapy.1 Ovarian cancer patients often present with late-stage disease and are historically first treated with platinum-based chemotherapy, which they may become resistant to and require another therapy, such as ELAHERE.2 "Ovarian cancer can be devastating, and when cancer cells stop responding to chemotherapy patients may feel hopeless about their journey. The data presented today reinforce the importance of ELAHERE as a transformative therapy for patients with limited options," said Svetlana Kobina, MD, PhD, vice president, oncology medical affairs, AbbVie. "We remain steadfast in our commitment to bring forward innovative therapies that improve the lives of patients with difficult-to-treat cancers." In the United States, ovarian cancer is the leading cause of death from gynecological cancers.3 Each year, approximately 20,000 women are diagnosed.4 Unfortunately, most patients develop platinum-resistant disease, which is difficult to treat.5 In this setting, single-agent chemotherapies are associated with minimal survival benefit while adding significant toxicity burden.6 The Phase 3 MIRASOL study included 453 patients with high-grade serous epithelial PROC whose tumors express high levels of FRα and had been treated with up to three prior therapies.1 Key findings from the 30.5-month median follow-up include: ELAHERE treatment achieved superior efficacy versus IC chemotherapy, with a median PFS of 5.59 months versus 3.98 months, representing a 37% reduction in the risk of tumor progression or death (HR 0.63; [95% CI: 0.51, 0.79]) and a higher objective response rate of 41.9% versus 15.9%. Superior and clinically meaningful overall survival for patients receiving ELAHERE (median 16.85 months) compared to IC chemotherapy (median 13.34 months), representing a 32% reduction in the risk of death (HR 0.68 [95% CI: 0.54, 0.84]). Other endpoints included safety and duration of response (DOR), which were consistent with the primary data analysis at 13.1-months median follow-up. The most common treatment-emergent adverse events (TEAEs) occurring in at least 20% of patients in the ELAHERE arm were blurred vision, keratopathy, abdominal pain, fatigue, diarrhea, dry eye, constipation, nausea and peripheral neuropathy. Compared with IC chemotherapy, treatment with ELAHERE was overall associated with lower rates of grade ≥3 TEAEs, serious AEs and discontinuations due to AEs. "The final data showcase the significant improvement in overall survival benefit of treatment with ELAHERE compared to standard of care chemotherapy," said investigator and presenter, Toon Van Gorp, MD, PhD, Professor of Gynecologic Oncology, University of Leuven. "The significant improvements in survival, along with the well-characterized safety profile, reinforce ELAHERE as an emerging standard of care for difficult-to-treat ovarian cancer and warrants further study of this medicine in earlier treatment settings." A separate analysis from the Phase 3 MIRASOL study evaluating the impact of [ELAHERE] treatment-emergent ocular events on patient-reported health-related quality of life (HRQoL), will be shared during an oral presentation March 17 at the SGO Annual Meeting scientific plenary session. ELAHERE was granted full approval by the U.S. Food and Drug Administration in March 2024 and was approved by the European Commission in November 2024. Marketing Authorization Applications for ELAHERE are also under review in multiple other countries. About the Phase 3 MIRASOL Trial MIRASOL is a randomized Phase 3 trial of ELAHERE versus investigator's choice (IC) of single-agent chemotherapy (weekly paclitaxel, pegylated liposomal doxorubicin, or topotecan). Eligibility criteria include patients with PROC whose tumors express high levels of FRα, using the Ventana FOLR1 RxDx Assay, and who have been treated with up to three prior regimens. The primary endpoint of this trial is progression-free survival (PFS) by investigator assessment. Key secondary endpoints include objective response rate (ORR) and overall survival (OS). The trial enrolled 453 patients. Patients were stratified by number of prior lines of therapy (14% had one prior line of therapy, 39% had two prior lines of therapy, and 47% had three prior lines of therapy) and by IC chemotherapy, with paclitaxel as the most commonly chosen (41%), followed by PLD (36%) and topotecan (23%). Sixty-two percent of patients received prior bevacizumab; 55% received a prior PARP inhibitor. More information can be found on (NCT04209855). About ELAHERE ELAHERE (mirvetuximab soravtansine-gynx) is a first-in-class antibody-drug conjugate (ADC) comprising a folate receptor alpha-binding antibody, cleavable linker, and the maytansinoid payload DM4, a potent tubulin inhibitor designed to kill the targeted cancer cells. Patients requiring access support may call 1-833-ELAHERE or visit . ELAHERE U.S. USE and IMPORTANT SAFETY INFORMATION7 What is ELAHERE? ELAHERE is a prescription medicine used to treat adults with folate receptor-alpha positive ovarian cancer, fallopian tube cancer, or primary peritoneal cancer who: have not responded to or are no longer responding to treatment with platinum-based chemotherapy and have received 1 to 3 prior types of chemotherapy. Your healthcare provider will perform a test to make sure that ELAHERE is right for you. It is not known if ELAHERE is safe and effective in children. IMPORTANT SAFETY INFORMATION What is the most important information I should know about ELAHERE? ELAHERE can cause serious side effects, including: Eye problems. Eye problems are common with ELAHERE and can also be severe. Tell your healthcare provider right away if you develop any eye problems during treatment with ELAHERE, including blurred vision, dry eyes, sensitivity to light, eye pain, eye redness, or new or worsening vision changes. Your healthcare provider will send you to see an eye care professional to check your eyes before you start treatment with ELAHERE, during treatment with ELAHERE, and as needed for any worsening signs and symptoms of eye problems. Your healthcare provider will prescribe steroid eye drops and lubricating eye drops before you start and during your treatment with ELAHERE. You should use eye drops as directed by your healthcare provider. Do not wear contact lenses throughout your treatment with ELAHERE unless you are told to use them by your healthcare provider. What should I tell my healthcare provider before receiving ELAHERE? Tell your healthcare provider about all of your medical conditions, including if you: have vision or eye problems. have numbness or tingling in your hands or feet. have liver problems. are pregnant or plan to become pregnant. ELAHERE can harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with ELAHERE. Patients who are able to become pregnant: Your healthcare provider should do a pregnancy test before you start treatment with ELAHERE. You should use an effective birth control (contraception) during treatment and for 7 months after your last dose of ELAHERE. are breastfeeding or plan to breastfeed. It is not known if ELAHERE passes into your breast milk. Do not breastfeed during treatment and for 1 month after your last dose of ELAHERE. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking certain other medicines during treatment with ELAHERE may cause side effects. What are the possible side effects of ELAHERE? ELAHERE can cause serious side effects, including: Eye problems. Eye problems are common with ELAHERE and can also be severe. Tell your healthcare provider right away if you develop any eye problems during treatment with ELAHERE, including blurred vision, dry eyes, sensitivity to light, eye pain, eye redness, or new or worsening vision changes. Lung problems (pneumonitis). ELAHERE can cause severe or life-threatening inflammation of the lungs that may lead to death. Tell your healthcare provider right away if you get new or worsening symptoms, including trouble breathing, shortness of breath, cough, or chest pain. Peripheral neuropathy. Nerve problems called peripheral neuropathy are common during treatment with ELAHERE and can also be severe. Your healthcare provider will monitor you for signs and symptoms of nerve problems. Tell your healthcare provider if you get new or worsening numbness, tingling, burning sensation or pain in your hands or feet or muscle weakness. The most common side effects and abnormal labs of ELAHERE include: Your healthcare provider may change your dose of ELAHERE, delay treatment, or completely stop treatment if you have certain side effects. These are not all of the possible side effects of ELAHERE. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit or call 1‑800‑FDA‑1088. Please see Full Prescribing Information, including Boxed WARNING and Medication Guide. About AbbVie in Oncology At AbbVie, we are committed to transforming standards of care for patients living with difficult-to-treat cancers. We are advancing a dynamic pipeline of investigational therapies across a range of cancer types in both blood cancers and solid tumors. We are focusing on creating targeted medicines that either impede the reproduction of cancer cells or enable their elimination. We achieve this through various, targeted treatment modalities and biology interventions, including small molecule therapeutics, antibody-drug conjugates (ADCs), Immuno-Oncology-based therapeutics, multi-specific antibody and in situ CAR-T platforms. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potential breakthrough medicines. Today, our expansive oncology portfolio comprises approved and investigational treatments for a wide range of blood and solid tumors. We are evaluating more than 20 investigational medicines in multiple clinical trials across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit . About AbbVie AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas including immunology, oncology, neuroscience and eye care – and products and services in our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at . Follow @abbvie on LinkedIn, Facebook, Instagram, X (formerly Twitter), and YouTube. AbbVie Forward-Looking Statements Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2024 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. Disclosure: Dr. Van Gorp has financial interests related to AbbVie. References: Van Gorp T, et al. Final overall survival analysis among patients with FRα-positive platinum-resistant ovarian cancer (PROC) treated with mirvetuximab soravtansine (MIRV) vs. investigator's choice chemotherapy (ICC) in the Phase 3 MIRASOL (GOG 3045/ENGOT-ov55) study. Data presented at the 2025 Society of Gynecologic Oncology Annual Meeting on Women's Cancer. Hanker, LC, et al. The impact of second to sixth line therapy on survival of relapsed ovarian cancer after primary taxane/platinum-based therapy, Annals of Oncology, Volume 23, Issue 10, 2012. WHO International Agency for Research on Cancer. GLOBOCAN 2022. Cancer Today. Absolute numbers, Mortality, Females, age [0-74], in 2022. Continents. Available here. Whittemore AS. Characteristics relating to ovarian cancer risk: implications for prevention and detection. Gynecol Oncol. 1994 Dec;55(3 Pt 2):S15-9. doi: 10.1006/gyno.1994.1334. PMID: 7835800. Hanker, LC, et al. The impact of second to sixth line therapy on survival of relapsed ovarian cancer after primary taxane/platinum-based therapy, Annals of Oncology, Volume 23, Issue 10, 2012. Matulonis, UA, er al. Efficacy and safety of mirvetuximab soravtansine in patients with platinum-resistant ovarian cancer with high folate receptor alpha expression: Results from the SORAYA Study. J Clin Oncol. 2023 May 1;41(13):2436-2445. doi: 10.1200/JCO.22.01900. Epub 2023 Jan 30. PMID: 36716407; PMCID: PMC10150846. ELAHERE [package insert]. North Chicago, IL: AbbVie Inc: 2025. SOURCE AbbVie WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED