On March 30, 2015 Amgen reported that the U.S. Food and Drug Administration (FDA) has accepted the supplemental New Drug Application (sNDA) 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, MAR 30, 2015, View Source [SID:1234502871]). The sNDA is designed to support the conversion of accelerated approval to full approval and expand the current Kyprolis indication. As part of the acceptance, the FDA granted Kyprolis priority review with a Prescription Drug User Fee Act (PDUFA) target action date of July 26, 2015.

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"Achieving deep and durable responses for patients with relapsed multiple myeloma is critical towards extending the time they live without their disease progressing," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "The FDA’s priority review designation for Kyprolis underscores the need for new treatment options for patients with relapsed multiple myeloma, and we look forward to working with regulatory authorities throughout the review process."

The sNDA is based on data from the Phase 3 ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) trial and other relevant data.

Priority review is assigned to applications for drugs that treat serious conditions and would, if approved, provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.1

Kyprolis is currently approved by the FDA for the treatment of patients with multiple myeloma who have received at least two prior therapies, including bortezomib and an immunomodulatory agent (IMiD), and have demonstrated disease progression on or within 60 days of completion of the last therapy.

About ASPIRE
The international, randomized Phase 3 ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) trial evaluated Kyprolis in combination with lenalidomide and low-dose dexamethasone, versus lenalidomide and low-dose dexamethasone alone, in patients with relapsed multiple myeloma following treatment with one to three prior regimens. The primary endpoint of the trial was progression-free survival, defined as the time from treatment initiation to disease progression or death. Secondary endpoints included overall survival, overall response rate, duration of response, disease control rate, health-related quality of life and safety. Patients were randomized to receive Kyprolis (20 mg/m2 on days 1 and 2 of cycle 1 only, escalating to 27 mg/m2 on days 8, 9, 15 and 16 of cycle 1 and continuing on days 1, 2, 8, 9, 15 and 16 of subsequent cycles), in addition to a standard dosing schedule of lenalidomide (25 mg per day for 21 days on, 7 days off) and low-dose dexamethasone (40 mg per week in 4 week cycles), versus lenalidomide and low-dose dexamethasone alone. The study randomized 792 patients at sites in North America, Europe and Israel.

The ASPIRE data were presented at the 56th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2014 and published in the New England Journal of Medicine.

The European Medicines Agency (EMA) provided Scientific Advice on the design and planned analysis of the ASPIRE trial and it was conducted under a Special Protocol Assessment (SPA) from the FDA.

About Multiple Myeloma
Multiple myeloma is the second most common hematologic cancer and results from an abnormality of plasma cells, usually in the bone marrow.1,2 Worldwide, nearly 230,000 people are living with multiple myeloma and approximately 114,000 new cases are diagnosed annually.3 In the U.S., there are nearly 96,000 people living with, or in remission from, multiple myeloma. The estimated number of new cases of multiple myeloma in 2014 was more than 24,000 and the estimated number of deaths was 11,090.4 In Europe, approximately 89,000 people are living with the disease and in 2012 there was an estimated 39,000 newly diagnosed cases and 24,000 deaths.3

On March 30, 2015 Merck Serono and Intrexon reported an exclusive strategic collaboration and license agreement to develop and commercialize Chimeric Antigen Receptor T-cell (CAR-T) cancer therapies (Press release, Merck KGaA, MAR 30, 2015, View Source Therapy_EN.pdf [SID:1234502861]). This collaboration advances Merck Serono’s comprehensive, science-driven strategy to develop innovative therapies that modulate the immune system’s natural ability to fight tumors.

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"The collaboration with Intrexon underlies Merck Serono’s focus on innovation, and enhances its R&D technology portfolio in immuno-oncology," says Belen Garijo, President and CEO of Merck Serono. "Moreover, it showcases Merck Serono’s commitment to developing therapies that have the potential to significantly evolve the way cancer is treated."

CAR-T cells are genetically engineered T-cells with synthetic receptors that recognize a specific antigen expressed on tumor cells. When CAR-T cells bind to a target, an immunological attack against the cancer cells is triggered.

Utilizing Intrexon’s cell engineering techniques and RheoSwitch platform, the collaboration aims to develop leading-edge products that empower the immune system in a regulated manner to overcome the current challenges of CAR-T therapy.

The agreement provides Merck Serono exclusive access to Intrexon’s proprietary and complementary suite of technologies to engineer T cells with optimized and inducible gene expression, as recently strengthened by a license agreement with the University of Texas MD Anderson Cancer Center.

Intrexon will be responsible for all platform and product developments until IND filing. Merck will nominate targets of interest for which CAR-T products will be developed. Merck will also lead the IND filing and pre-IND interactions, clinical development and commercialization. In addition, Intrexon has the opportunity to explore targets independently, granting Merck opt-in rights during clinical development.

"Merck is an ideal partner in CAR-T for us because of their long-term perspective, extraordinary character, worldwide reach and commitment to leadership in immuno-oncology," says Randal J. Kirk, Chairman and CEO of Intrexon. "We look forward to working together to benefit patients through the creation of a leading franchise in this very promising field."

Under the terms of the agreement, Intrexon will receive an upfront payment of $115 million. For the first two targets of interest selected by Merck Serono, Intrexon will receive research funding and is eligible to receive up to $826 million development, regulatory and commercial milestones, as well as tiered royalties on product sales. In addition, Intrexon is also eligible to receive further payments upon achievement of certain technology development milestones.