Myeloma Research Foundation and Karyopharm Hoping Myeloma Therapy Progress Continues

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by Alice Melão |

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Myeloma therapy quest

The Multiple Myeloma Research Foundation and Karyopharm Therapeutics are hoping that 2018 will be as momentous a year as 2017 in their collaborative effort to develop a new treatment for multiple myeloma and other cancers.

Last year’s milestones included the therapy, selinexor, advancing to Phase 2 clinical trials; Ono Pharmaceutical obtaining the rights to market it in Japan and other Asian countries for $193 million; and Karyopharm passing on some of the proceeds from the Ono deal to the foundation, which has helped fund selinexor’s development.

Selinexor is designed to prevent tumor-suppressing proteins from leaving the nucleus of cells. Keeping the proteins in the nucleus gives them more punch when they fight cancer cells. This approach also spares healthy cells.

Selinexor targets the XPO1 protein, which helps other proteins leave the nucleus.

The foundation, also known by its acronym MMRF, gave Karyopharm $1 million in 2011 to kick-start the development of selinexor as a myeloma therapy. The award was part of the organization’s Biotech Investment Award program.

The foundation is “committed to ensuring that organizations have the funding and resources necessary to accelerate the availability of promising new therapies,” Paul A. Giusti, its president and CEO, said in a press release.  The foundation is “proud to have partnered with Karyopharm over the last several years to support the development of selinexor,” he added.

Selinexor has shown promise as a myeloma treatment in clinical trials.

As an example, myeloma patients who had failed to respond to other therapies did respond to a combination of selinexor and dexamethasone, a Phase 1 trial (NCT01607892) showed.

Preliminary data from the ongoing Phase 2 STORM trial (NCT02336815) and Phase 1/2 STOMP trial (NCT02343042) also showed that selinexor was effective, both as a stand-alone treatment and in combination with other therapies, researchers said. It was also safe, and patients tolerated it well, they said.

In addition, a pivotal Phase 3 trial (NCT03110562) is under way. The BOSTON study will evaluate a combination of selinexor, Velcade (bortezomib) and low-dose dexamethasone as a treatment for relapsed or refractory myeloma patients.

The trial is recruiting participants. Key results are expected in 2019. A pivotal trial is one designed to provide evidence needed for a therapy’s regulatory approval.

Under their partnership agreement, Karyopharm will pass on to the foundation some of the revenue that selinexor generates from up-front payments or sales. The Ono agreement triggered the first installment.

The foundation “has been an invaluable strategic partner for several years, and it is highly gratifying that their early support for oral selinexor would lead to a robust clinical development program in myeloma,” said Sharon Shacham, the president and chief scientific officer of Karyopharm.

“We are pleased that the recent licensing transaction” with Ono, “a widely respected oncology leader in Japan, an important global territory, will provide a return on MMRF’s investment in Karyopharm and allow further investments to treat myeloma,” she said. “We look forward to continuing our long and constructive relationship with the MMRF for many years to come.”