Rina-S cuts risk of ovarian cancer progression by 71% and meets overall survival goal
Key Facts
On October 23, 2026, Genmab said the global Phase 3 RAINFOL-02 trial of its investigational drug Rina-S met its primary endpoint of extending progression-free survival in patients with platinum-resistant ovarian cancer. The company said the trial also met its key secondary overall survival endpoint, with a statistically significant improvement in both analysis populations. In the ITT population, the risk of disease progression or death fell 71% compared with investigator’s choice of chemotherapy, while the reduction was 67% in the PAS population. Chief Executive Jan van de Winkel said the results "represent an important milestone for Genmab and strengthen our confidence in Rina-S’s potential." The results provide a Phase 3 readout for the drug program in a disease that affects more than 320,000 people annually worldwide, according to background information provided by the company.
The hazard ratio for progression-free survival or death was 0.29 in the ITT population, with a 95% confidence interval of 0.23 to 0.37 and p<0.0001, according to Genmab’s statement. In the PAS population, the hazard ratio was 0.33, with a confidence interval of 0.26 to 0.42 and p<0.0001. For overall survival, the hazard ratio for death was 0.43 in ITT, with a confidence interval of 0.31 to 0.59 and p<0.0001. The ratio was 0.44 in PAS, with a confidence interval of 0.31 to 0.63 and p<0.0001. The company described the overall survival improvement in both populations as statistically significant and clinically meaningful, and said the trial met this key secondary endpoint.
The ITT population included all 584 randomized patients with platinum-resistant ovarian cancer, regardless of their level of folate receptor alpha expression. The PAS population included 492 patients, and the inclusion of patients with high receptor expression, at a threshold of ≥75% PS2+, was contingent on their having received prior mirvetuximab treatment, according to the company’s definition. The different criteria mean PAS results apply to a defined subset of patients, while ITT covers all randomized participants. RAINFOL-02 was designed as a randomized, open-label, multicenter trial, with participants assigned 1:1 between the two treatment arms. Patients received Rina-S at a dose of 120 mg/m² on day one of a three-week treatment cycle, or one of four standard chemotherapy drugs: paclitaxel, topotecan, liposomal doxorubicin or gemcitabine.
Genmab said the observed safety profile was consistent with the drug’s known profile and that it had identified no new safety signals or unexpected concerns. The company is placing these results alongside an earlier Phase 2 readout from the RAINFOL-01 trial, which showed antitumor activity regardless of folate receptor alpha expression, including among patients with low expression or no expression. In one part of RAINFOL-01, 53% of patients had received three or four prior lines of treatment, and all had received bevacizumab and a taxane, according to Genmab. The announced measures differ between the two phases: RAINFOL-02 tested survival, while the earlier results focused on tumor response. Rina-S’s development program includes five Phase 3 trials covering platinum-resistant ovarian cancer and endometrial cancer, as well as trials in platinum-sensitive ovarian cancer and maintenance treatment options.
In trading, Genmab shares closed at $43.79 on October 23, compared with $40 in the previous session, according to EL7 data. Investing.com reported that the stock rose about 9% on Friday following the announcement of positive topline results. Genmab said it would discuss the RAINFOL-02 results and potential next steps with regulators worldwide, without announcing a regulatory filing. It also plans to present the data at a future medical meeting. The company scheduled an investor update for Wednesday, October 28, at 5:00 p.m. Central European Time, corresponding to 12:00 p.m. U.S. Eastern Time.