On November 9, 2015 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a clinical-stage pharmaceutical company, reported financial results for the third quarter 2015 and commented on recent accomplishments and clinical development plans for its pipeline of SINE-based therapeutics including selinexor, its lead product candidate (Filing, 8-K, Karyopharm, NOV 9, 2015, View Source [SID:1234508114]).

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"This has been a very busy period for Karyopharm with active enrollment across our ongoing selinexor clinical studies and new studies, primarily in combination with other anticancer agents, continuing to come on-line," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "We look forward to sharing additional data on our pipeline of first-in-class oncology therapeutics at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2015 annual meeting, including selinexor activity in combination regimens across hematologic malignancies."

Conference Call Information:

Karyopharm will host a conference call today, Monday, November 9, 2015, at 8:30 a.m. Eastern Time, to discuss the third quarter 2015 financial results, recent accomplishments and clinical developments plans. To access the conference call, please dial (855) 437-4406 (US) or (484) 756-4292 (international) at least five minutes prior to the start time and refer to conference ID: 69164993. An audio recording of the call will be available under "Events & Presentations" in the Investor section of Karyopharm’s website, View Source, approximately two hours after the event.
Clinical Development Plans:

• Based on striking preclinical synergy in animal models of myeloma, Karyopharm initiated a multi-center, Phase 1b/2 clinical study of selinexor called STOMP ("Selinexor and Backbone Treatments of Multiple Myeloma Patients") with support from Myeloma Canada. In this multi-arm study, Karyopharm is evaluating the combination of selinexor and low dose dexamethasone with backbone therapies bortezomib, pomalidomide or lenalidomide in patients with previously treated multiple myeloma. Approximately 220 patients with multiple myeloma will be enrolled in this study with preliminary top-line data anticipated in 2017. Selinexor and low dose dexamethasone are already being combined with carfilzomib in an Investigator-sponsored trial (IST), where promising preliminary data were presented at the ASH (Free ASH Whitepaper) 2014 annual meeting and will be updated with additional patient data at the ASH (Free ASH Whitepaper) 2015 annual meeting.

• Karyopharm is actively enrolling patients in four later phase clinical studies evaluating single-agent selinexor: one in older patients with relapsed/refractory acute myeloid leukemia (SOPRA study), the second in patients with relapsed/refractory diffuse large B-cell lymphoma (SADAL study), the third in patients with multiple myeloma (STORM study) and the fourth in patients with Richter’s transformation (SIRRT study). Interim data are expected from the SOPRA and STORM studies in the middle of 2016. Preliminary top-line data from the SOPRA and SADAL studies are anticipated in the fourth quarter of 2016.

Targeting Disease at the Nuclear Pore

• Karyopharm is currently conducting company-sponsored trials of single-agent selinexor in three solid tumor indications including heavily pretreated patients with gynecologic malignancies (SIGN study), recurrent glioblastoma multiforme (KING study) and hormone-refractory prostate cancer (SHIP study). Based on promising data observed in a Phase 1 study, a randomized, blinded Phase 2/3 trial of selinexor versus placebo in liposarcoma (SEAL study) is planned to commence in the fourth quarter of 2015.

• In addition, a number of ISTs or company-sponsored trials evaluating the potential of selinexor in combination with either chemotherapy or targeted agents are currently ongoing or planned.

• Based on promising preclinical data, Karyopharm filed an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) for KPT-8602 and, pending review, plans to initiate a clinical study in multiple myeloma in early 2016.

• Karyopharm also plans to file an IND for its first-in-class, oral PAK4 Allosteric Modulator, KPT-9274, and initiate clinical development in patients with heavily pretreated solid tumors or lymphoma in the first half of 2016.
Scientific Presentations and Publications:

• Five oral presentations and twelve poster presentations describing Karyopharm’s pipeline of first-in-class oncology therapeutics were accepted for presentation at the upcoming 57th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) 2015 meeting being held December 5 – 8, 2015 in Orlando, Florida. The accepted abstracts include:

• Clinical and preclinical data demonstrating the promising activity of selinexor (KPT-330), Karyopharm’s most advanced, novel, oral Selective Inhibitor of Nuclear Export/SINE compound, in combination with other anti-cancer agents;

• Preclinical data on KPT-8602, a second generation SINE compound in early-stage development with the potential for distinct pharmaceutical characteristics; and

• Preclinical data on KPT-9274, a first-in-class oral PAK4 Allosteric Modulator.

• Preclinical data demonstrating the anti-tumor benefits of combining selinexor with immunotherapy in aggressive melanoma models were presented at the Society for Immunotherapy Cancer (SITC) (Free SITC Whitepaper) 2015 Annual Meeting held November 4 – 8, 2015 in National Harbor, Maryland. In an oral presentation entitled "Selinexor, a Selective Inhibitor of Nuclear Export (SINE), enhanced activity in combination with PD-1/PD-L1 blockade in syngeneic murine models of colon cancer and melanoma" Karyopharm researchers and collaborators at The Ohio State University demonstrated that the combination of selinexor with PD-1 or PDL-1 immune checkpoint inhibitors exerts considerable anti-tumor and immune-stimulating activity in an aggressive murine melanoma model.

• Data demonstrating the beneficial pharmacological effects of SINE-based compounds in neurodegenerative models, including Amyotrophic Lateral Sclerosis (ALS), were recently published in the journal Nature and presented at the American Neurological Association (ANA) and the Society for Neuroscience (SfN) meetings. These data confirm that nuclear transport is disrupted by a common gene mutation found in ALS and describe the neuroprotective effects of SINE compounds, similar to the neuroprotective effects previously observed in Multiple Sclerosis (MS). Karyopharm’s SINE compound research efforts in ALS are being supported entirely by collaborator grant funding, with MS research supported by the National Multiple Sclerosis Society. Given the tremendous unmet need for new treatments for ALS and MS, Karyopharm is advancing its oral SINE compound KPT-350 for these indications.

Targeting Disease at the Nuclear Pore

Third Quarter September 30, 2015 Financial Results
Cash, cash equivalents and investments as of September 30, 2015, including restricted cash, totaled $230.2 million, compared to $256.0 million as of June 30, 2015.

For the quarter ended September 30, 2015, research and development expense was $25.9 million compared to $16.0 million for the quarter ended September 30, 2014. For the quarter ended September 30, 2015, general and administrative expense was $4.8 million compared to $3.8 million for the quarter ended September 30, 2014. The increase in research and development expense resulted primarily from the increase in expenses related to the continued clinical development of selinexor. The increase in general and administrative expense resulted primarily from the costs of being a public company and an increase in stock-based compensation.

Karyopharm reported a net loss of $30.4 million, or $0.85 per share, for the quarter ended September 30, 2015, compared to a net loss of $19.7 million, or $0.61 per share, for the quarter ended September 30, 2014. Net loss includes stock-based compensation expense of $3.5 million and $2.9 million for the quarters ended September 30, 2015 and September 30, 2014, respectively.

Financial Outlook
Based on current operating plans, Karyopharm expects that its existing cash and cash equivalents will fund its research and development programs and operations into 2018, including moving the four later-stage clinical studies to their next data inflection points. Karyopharm expects to end 2015 with greater than $200 million in cash, cash equivalents and investments.