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Ricolinostat in Multiple Myeloma

Acetylon is currently evaluating its lead experimental drug candidate, ricolinostat (ACY-1215), in several clinical studies for the treatment of multiple myeloma and lymphoma. Ricolinostat is a selective inhibitor of an enzyme called histone deacetylase (HDAC) 6. HDACs are a family of 18 different enzymes which are present in most cells of the human body. The way in which HDAC inhibitors work is very complex, and the current generation of HDAC inhibitors (sometimes called “pan-HDAC” inhibitors) affect indiscriminately many gene functions as well as protein transport within cells, resulting in significant side effects.

Acetylon scientists believe that selectively inhibiting HDAC6 (versus other enzymes of the HDAC family) may lead to effective therapy with fewer serious side effects. Ricolinostat may also make myeloma or lymphoma cancer cells more susceptible to the effects of other beneficial anti-cancer medications.

Early data have been reported from the Phase 1b sections of two ongoing clinical trials, and thus far, ricolinostat has been well-tolerated and has demonstrated preliminary signs of clinical activity.

Highlights of the data reported at the 19th Congress of the European Hematology Association from an ongoing Phase 1b clinical trial of ricolinostat in combination with Revlimid® (lenalidomide) and dexamethasone include:

  • All 22 evaluable patients demonstrated stable disease or better
  • 64% of patients demonstrated partial response or better, including 1 complete response, 5 very good partial responses, and 8 partial responses
  • 3 patients achieved minor response
  • 10 patients were previously refractory to either full dose or maintenance Revlimid treatment, meaning they had stopped responding to Revlimid

Highlights of the data reported at the 19th Congress of the European Hematology Association of ricolinostat in combination with Velcade® (bortezomib) and dexamethasone include:

  • Overall response rate (defined as partial response or better) of 53% in 19 evaluable patients, or 36% of the intention-to-treat population (i.e. the total number of patients who began the study, regardless of if they continued on the study for the full protocol)
  • 10 patients were previously refractory to Velcade

NOTE: Ricolinostat is currently being evaluated in clinical trials and is not approved for other human use.

Click here to find out more about the ricolinostat clinical trials in multiple myeloma and in lymphoma.