Onyx Pharmaceuticals, Inc. recently announced that it has finished enrolling patients for the phase III FOCUS (CarFilzOmib for AdvanCed Refractory MUltiple Myeloma European Study) trial which is being conducted with Kyprolis (carfilzomib). The study is being conducted in treatment-experienced patients (who have received three or more prior therapies) with relapsed and refractory myeloma.
The study has been designed to compare single-agent Kyprolis with best supportive care of low-dose steroids plus optional cyclophosphamide. While the primary endpoint is overall survival, secondary endpoints include progression-free survival, overall response rate, clinical benefit response, duration of response and safety.
We remind investors that Kyprolis gained US Food and Drug Administration (FDA) approval in July for use in treatment-experienced multiple myeloma patients (who have received at least two prior therapies, including Takeda/Johnson & Johnson’s (JNJ - Free Report) Velcade (bortezomib) and an immunomodulatory agent) and whose disease has progressed on or within 60 days of completion of the last therapy.
Approval was based on results from a phase IIb study (003-A1). The indication for Kyprolis is based on response rate. We note that currently no data demonstrating an improvement in progression free survival or overall survival is available on Kyprolis.
Immense Commercial Potential
Onyx Pharma has priced Kyprolis at $1,658 per vial, at a premium to Velcade. The price per 28-day cycle is $9,950 based on the dosing schedule in the label and assuming a 78 kilogram individual. The mean duration of treatment was 4.4 months in study 003-A1, which means treatment cost could reach $44,000.
According to the American Cancer Society, about 20,000 people in the US could be diagnosed with multiple myeloma every year. Onyx Pharma estimates that Kyprolis-eligible patients could be about 10,000 - 15,000 annually in the US. This represents significant commercial potential for Onyx Pharma.
Onyx Pharma has a dedicated 100 person oncology sales force in place for the launch. The Kyprolis sales force will call on about 2,000 physician offices and hospitals where a major part of Kyprolis-eligible patients are treated. As far as reimbursement and formulary coverage is concerned, Onyx Pharma expects coverage for about 80% of the indicated population.
While a miscellaneous J-code will be used for the time being, a Kyprolis specific Medicare J-code is expected in January 2014.
We are encouraged by Onyx Pharma’s efforts to expand Kyprolis’ label. Besides the FOCUS trial, Kyprolis is being evaluated in several other studies including a randomized phase III trial (ASPIRE), which is evaluating the combination of Celgene’s (CELG - Free Report) Revlimid and low dose dexamethasone with or without Kyprolis in relapsed multiple myeloma patients.
Kyprolis is also in a head-to-head phase III study (ENDEAVOR) which is being conducted in patients whose disease relapsed after one to three treatments. The study is comparing Kyprolis plus low dose dexamethasone to Velcade plus low dose dexamethasone with progression free survival being the primary endpoint.
We currently have a Neutral recommendation on Onyx Pharma.