ONO PHARMACEUTICAL : Enters into Exclusive License Agreement with Karyopharm to Develop and Commercialize Selinexor and KPT-8602, XPO1 Inhibitors (77.9KB)

October 12, 2017

Ono Pharmaceutical Co., Ltd. (Osaka, Japan; President, Representative Director, Gyo Sagara; "ONO") announced that it entered into an exclusive license agreement with Karyopharm Therapeutics Inc. (Newton, MA, U.S.; CEO, Michael G. Kauffman; "Karyopharm") for the development and commercialization of Selinexor, their first-in-class oral Selective Inhibitor of Nuclear Export (SINETM) compound, and KPT-8602, a second-generation oral SINETM compound, both of which are XPO1 (Exportin 1) inhibitors under the clinical development. This agreement grants to ONO all rights to develop and commercialize Selinexor and KPT-8602 for all oncology indications exclusively in Japan, South Korea, Taiwan, Hong Kong and ASEAN countries ("ONO's territory").

Under the terms of the agreement, ONO will pay to Karyopharm a one-time upfront payment of

¥2.5 billion and up to an additional ¥19.15 billion if certain development and commercial milestones are achieved. ONO will also pay to Karyopharm low double-digit royalties based on net sales of the products in the ONO's territory. Furthermore, ONO will have the right to participate in any global clinical studies of the products by bearing the development cost in the ONO's territory. Karyopharm continuously retains all rights to develop and commercialize the products outside the ONO's territory.

"We are very delighted to collaborate on the development of Selinexor and KPT-8602, an early development stage XPO1 inhibitor with Karyopharm, a leading pharmaceutical company focused on the research and development of novel first-in-class drugs in the oncology field," said Gyo Sagara, President, Representative Director of ONO. "We believe both products will present a new treatment option to patients suffering from devastating cancers in Asian countries."

"Given ONO's established leadership in oncology, including Opdivo® (nivolumab) and Kyprolis® (carfilzomib) in Japan, we believe there is no company better suited to advance both selinexor and KPT-8602 in Japan and the other licensed territories," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "ONO is well-known and widely respected for its clinical development and commercial expertise, and this partnership provides important validation for both compounds, while allowing us to remain focused on executing our late-phase Selinexor trials and pursue regulatory approval in the United States and European Union. We look forward to working with the ONO team to advance both compounds with the goal of rapidly bringing them to patients who are in need of new treatment options."

Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE™ compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 2,200 patients have been treated with selinexor, and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade® (bortezomib) and low-dose dexamethasone (BOSTON), in combination with low-dose dexamethasone (STORM) and backbone therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), and liposarcoma (SEAL), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with one or more approved therapies in a variety of tumor types to further inform Karyopharm's clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

KPT-8602 is a second generation oral SINE™ compound. KPT-8602 functions by binding to and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. KPT-8602 has demonstrated minimal brain penetration in animals, which has been associated with reduced toxicities in preclinical studies while maintaining potent anti-tumor effects.

Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a clinical-stage pharmaceutical company focused on the discovery and development of novel first-in-class drugs directed against nuclear transport and related targets for the treatment of cancer and other major diseases. Karyopharm's SINE™ compounds function by binding with and inhibiting the nuclear export protein XPO1 (or CRM1). In addition to single-agent and combination activity against a variety of human cancers, SINE™ compounds have also shown biological activity in models of neurodegeneration, inflammation, autoimmune disease, certain viruses and wound-healing. Karyopharm, which was founded by Dr. Sharon Shacham, currently has several investigational programs in clinical or preclinical development. For more information, please visit www.karyopharm.com.

Contacts:

ONO PHARMACEUTICAL CO., LTD.

Corporate Communications public_relations@ono.co.jp