On February 26, 2015 Amgen (NASDAQ: AMGN) and its subsidiary Onyx Pharmaceuticals reported that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) of Kyprolis (carfilzomib) for Injection for the treatment of patients with relapsed multiple myeloma who have received at least one prior therapy (Press release, Amgen, FEB 26, 2015, View Source [SID:1234501993]). The MAA has been granted accelerated assessment by the EMA.

Kyprolis is a proteasome inhibitor, one of the classes of drugs used to treat multiple myeloma, an incurable blood cancer affecting approximately 89,000 people in Europe.1 Nearly all patients with the disease experience periods of remission, followed by relapses and eventually their disease becomes resistant to treatment.

“Achieving deep and durable responses for patients with relapsed multiple myeloma is critical towards extending the time they live without their disease progressing,” said Pablo J. Cagnoni, M.D., president, Onyx Pharmaceuticals, Inc. “We look forward to working with European regulators to potentially make this important medication available.”

The MAA includes data from the Phase 3 ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) trial as well as other relevant data.

Kyprolis previously received orphan drug designation by the EMA in the European Union (EU). Orphan designation is granted for medicines intended for the treatment, prevention or diagnosis of a disease that is life threatening and has a prevalence in the EU of no more than five in 10,000. The intended medicine must aim to provide significant benefit to those affected by the condition.2

Kyprolis was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in July 2012. Kyprolis is also approved for use in Argentina, Israel and Mexico.3-5

MM-111 Update

MM-111 is being investigated in a Phase 2 clinical trial in combination with trastuzumab and paclitaxel versus a control arm of trastuzumab and paclitaxel in patients with second-line HER2+ gastric/gastroesophageal cancer (Press release, Merrimack, FEB 26, 2015, View Source [SID:1234501985]). The trial was designed with two co-primary endpoints: progression free survival in the overall population and progression free survival in the heregulin high population.

The Data Safety Monitoring Board (DSMB) for the trial met recently and recommended closure of the trial to further patient enrollment due to shorter progression free survival on the experimental arm relative to the control arm in the overall patient population. Based on the DSMB’s recommendation, Merrimack decided to stop enrollment in this trial. A preliminary analysis shows that a vast majority of patients in the trial were below the threshold of heregulin levels necessary to benefit from MM-111. As a result, Merrimack does not believe that further development of MM-111 in second line HER2+ gastric cancer is warranted. In addition, Merrimack does not plan to invest in additional development of MM-111 at this time.