On January 20, 2021 ONCOtherapeutics and Dr. James Berenson reported that promising new data demonstrating that ruxolitinib (RUX), an oral JAK1/2 inhibitor, with only steroids is well tolerated and shows promising efficacy for treating heavily previously treated MM patients (Press release, Oncotherapeutics, JAN 20, 2021, View Source [SID1234574155]).

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Recently published results from a Phase 1 trial for 28 heavily previously treated MM patients administered RUX, LEN and methylprednisolone (MP) demonstrated that the therapy was well tolerated and RUX overcame refractoriness to LEN and steroids (Berenson et al., Clin Cancer Res. 2020). The clinical and overall response rates were 46% and 28%, respectively, and all 12 responding patients were refractory to LEN.

To further evaluate whether RUX and steroids without LEN demonstrate clinical activity and its tolerability in the same patient population, an ongoing Phase I trial was expanded to also include a cohort of patients treated with only this two-drug combination (NCT03110822). RUX (15mg BID) was given orally twice a day continuously and oral methylprednisolone (MP; 40 mg) every other day.

Of the 17 evaluable patients treated with RUX and steroids so far, the CBR and ORR were 53% (n=8) and 33% (n=5), respectively. The median duration of response was 4 months (range, 1-12) and median progression free survival was 5 months (range, 1-14). The combination was well tolerated with no unanticipated adverse effects.

As noted by lead investigator Dr. James R. Berenson, "Multiple myeloma patients continue to need new treatment options, especially for those who have become refractory to previously effective treatment options. Besides having a direct MM killing effect, ruxolitinib’s ability to resensitize lenalidomide is a major advantage that allows clinicians to recycle drugs and recapture disease control, while maintaining the convenience of an all-oral regimen. This is an entirely new treatment approach for MM patients."

With these promising results, ONCOtherapeutics has expanded the current study to further investigate the potential of this novel two-drug combination for treating MM including those with poor kidney function.