"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
Conference Call Information:
Karyopharm will host a conference call today,
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 2014 annual meeting and will be updated with additional patient data at the ASH 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.
- 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) 2015 meeting being heldDecember 5 - 8, 2015 inOrlando, 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) 2015 Annual Meeting heldNovember 4 - 8, 2015 inNational 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 atThe 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 theSociety 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 theNational 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.
Third Quarter
Cash, cash equivalents and investments as of
For the quarter ended
Karyopharm reported a net loss of
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
About
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, including the timing of initiation of certain trials and of the reporting of data from such trials. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the company's current expectations. For example, there can be no guarantee that any of Karyopharm's SINE compounds, including selinexor (KPT-330), KPT-8602, Karyopharm's next generation SINE compound, or KPT-9274, Karyopharm's first-in-class PAK4 Allosteric Modulator, or any other drug candidate that Karyopharm is developing will successfully complete necessary preclinical and clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: Karyopharm's results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the
| Karyopharm Therapeutics Inc. | ||
| CONDENSED CONSOLIDATED BALANCE SHEETS | ||
| (unaudited) | ||
| (in thousands, except share and per share amounts) | ||
| 2015 | 2014 | |
| Assets | ||
| Current assets: | ||
| Cash and cash equivalents | ||
| Short-term investments | 151,738 | 55,115 |
| Prepaid expenses and other current assets | 3,107 | 2,027 |
| Total current assets | 198,344 | 207,751 |
| Property and equipment, net | 3,660 | 2,754 |
| Long-term investments | 34,430 | 8,658 |
| Other assets | 52 | 774 |
| Restricted cash | 485 | 400 |
| Total assets | ||
| Liabilities and stockholders' equity | ||
| Current liabilities: | ||
| Accounts payable | ||
| Accrued expenses | 9,469 | 5,825 |
| Deferred rent | 200 | 126 |
| Other current liabilities | 203 | 62 |
| Total current liabilities | 14,440 | 12,301 |
| Deferred rent, net of current portion | 1,939 | 1,242 |
| Total liabilities | 16,379 | 13,543 |
| Stockholders' equity: | ||
| Preferred stock, |
— | — |
| Common stock, |
4 | 3 |
| Additional paid-in capital | 448,095 | 345,166 |
| Accumulated other comprehensive loss | (26) | (29) |
| Accumulated deficit | (227,481) | (138,346) |
| Total stockholders' equity | 220,592 | 206,794 |
| Total liabilities and stockholders' equity | ||
| Karyopharm Therapeutics Inc. | ||||
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||
| (unaudited) | ||||
| (in thousands, except share and per share amounts) | ||||
| Three Months Ended, | Nine Months Ended, | |||
| 2015 | 2014 | 2015 | 2014 | |
| Contract and grant revenue | ||||
| Operating expenses: | ||||
| Research and development | 25,923 | 15,951 | 73,680 | 40,089 |
| General and administrative | 4,762 | 3,814 | 16,318 | 10,028 |
| Total operating expenses | 30,685 | 19,765 | 89,998 | 50,117 |
| Loss from operations | (30,610) | (19,744) | (89,773) | (49,903) |
| Other income (expense): | ||||
| Interest income | 239 | 20 | 647 | 54 |
| Other expense | (2) | — | (9) | — |
| Total other income (expense), net | 237 | 20 | 638 | 54 |
| Net loss | $ (30,373) | $ (19,724) | $ (89,135) | $ (49,849) |
| Net loss per share—basic and diluted | $ (0.85) | $ (0.61) | $ (2.51) | $ (1.63) |
| Weighted-average number of common shares outstanding used in net loss per share—basic and diluted | 35,708,739 | 32,558,646 | 35,575,745 | 30,619,074 |
CONTACT:Justin Renz (617) 658-0574 jrenz@karyopharm.comGina Nugent (617) 460-3579 nugentcomm@aol.com
Source: Karyopharm Therapeutics
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