I just concluded attending an online press conference with officials from Onyx Pharmaceuticals, manufacturers of the experimental anti-myeloma drug, carfilzomib.
This was primarily a business related press conference, especially the Q & A portion at the end. Since journalists weren’t presented a written transcript, I will keep the details basic. You can expect a follow-up soon after official press packets are released.
Top line results of Onyx’s latest carfilzomib Phase 2 Study featured a 24% overall response rate among heavily pretreated patients. 256 patients participated in the study. All patients had received three or more prior therapies. Well over 30% of patients received a statistical benefit from using carfilzomib, with an average of 7.4 months disease free progression. Good long term tolerability, with patients staying on treatment for as long as 18 consecutive months. Doses tended to be low—between 20 and 27 mg per dose. Researchers are hopeful higher doses may achieve even better results.
Study details were understandably scarce, but Dr. Michael Kaufman did mention 99% of patients in the study had used Velcade in the past.
A stock market analyst asked how the 24% number compared to the use of existing therapies. Onyx officials used a soon to be published study, headed by Mayo Clinic’s Dr. Kumar, which resulted in a 6-10 months survival, and 11% response rate using previously approved multiple myeloma drugs. Overall survival data was not available for this study at this press conference, but the implication was it far exceeded 10 months.
Responses happen quickly if the drug is going to work—often within the first treatment cycle. Ongoing combination drug studies are expected to produce far more impressive results.
Onyx officials plan to meet with FDA as soon as possible to apply for fast track approval, which could come as early as spring of 2011.
Securities company questioners all seemed impressed with the results from a stock market perspective.
Official, detailed results will be presented at ASH in Orlando next December.
My impression: Positive, but no surprises here. Considering it is becoming clear carfilzomib is far more effective when combined with other novel therapies—and that results should improve once dosing is tweaked and company researchers learn more about optimum dosing—my fellow patients should be encouraged.
Feel good and keep smiling! Pat