"We made significant progress advancing the development of our lead product candidate, selinexor, a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE™ compound across a number of hematologic and solid tumor indications in the fourth quarter of 2014," said
"Our aggressive selinexor development program continues in 2015 with plans to initiate potential registration-directed studies for selinexor in multiple myeloma and liposarcoma. We look forward to the presentation of selinexor clinical data updates in both solid and hematologic tumors at upcoming medical conferences later this year," said
Conference Call Information:
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 97861837. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of Karyopharm's website, http://www.karyopharm.com, approximately two hours after the event.
Recent Corporate Accomplishments
- Selinexor (KPT-330) registration-directed studies:
- Initiated a Phase 2b registration-directed study called SADAL, or Selinexor Against Diffuse Aggressive Lymphoma. This randomized, multi-center study is evaluating single-agent Selinexor with a target enrollment of approximately 200 patients with relapsed/refractory DLBCL in approximately 90 sites worldwide. Enrolled patients will be randomized to receive either 60 mg or 100 mg of selinexor twice weekly. Per the protocol, at least 50% of each arm of the study will have Germinal Center B-Cell (GCB) type. Overall response rate is the primary endpoint of this study.
- Initiated a Phase 2 registration-directed study called SIRRT, or Selinexor in Relapsed/Refractory Richter's Transformation. This single-arm, open-label study is also evaluating selinexor as a single agent and will enroll approximately 50 patients with refractory and/or relapsed Richter's transformation in approximately 32 sites worldwide. Enrolled patients will receive 60 mg of Selinexor twice weekly. Overall response rate is the primary endpoint of this study.
- A third, previously initiated registration-directed study called SOPRA, or Selinexor in Older Patients with Relapsed/Refractory AML, is also actively enrolling (with an interim data analysis targeted for the first quarter of 2016). Overall survival is the primary endpoint of this study, which has a target enrollment of 150 patients in approximately 80 sites worldwide. Patients are randomized in a two-to-one ratio to either selinexor or physician's choice in patients who are older than 60 years with relapsed or refractory AML after one line of therapy and are ineligible for intensive chemotherapy or transplantation.
Scientific Presentations and Publications:
- Presented positive data for selinexor including anti-tumor activity, durable cancer control and tolerability across all non-Hodgkin's lymphoma types studied and high rates of durable responses in combination with approved multiple myeloma therapies at the
American Society of Hematology (ASH) 2014 annual meeting.- In an ongoing Phase 1 clinical trial, selinexor in patients with heavily pre-treated, progressive non-Hodgkin's lymphoma (NHL), including aggressive B-cell
NHL such as DLBCL, Richter's transformation, Burkitt lymphoma, mantle cell lymphoma, T-cell lymphoma and follicular/indolent lymphoma, demonstrated a 37% overall response rate (partial response or better) and a 73% disease control rate (stable disease or better) in 52 evaluable patients. Responses included five complete responses, four in patients with DLBCL and one in a patient with T-cell lymphoma. In the patients with heavily pretreated DLBCL, selinexor demonstrated a median duration of response (DOR), which measures time from response to progression, of approximately seven months. Clear activity was observed across all DLBCL subtypes evaluated, including a 36% overall response rate in patients with GCB and a 50% response rate and 75% disease control rate among four patients with double-hit DLBCL. One of these patients with double hit DLBCL has a durable complete response in excess of 14 months and a second patient with a partial response was on study for approximately eight months. - In an ongoing Phase 1 clinical trial, selinexor dosed twice weekly at 45mg/m2 in combination with low-dose dexamethasone demonstrated a 67% overall response rate (partial response or better) and an 89% clinical benefit rate (minimal response or better) in nine evaluable patients with heavily pre-treated and refractory multiple myeloma. Six of these patients remained on study for at least 16 weeks and the overall median DOR among patients with a partial response or better was approximately seven months.
- In a separate ongoing Phase 1/2 study, selinexor in combination with the proteasome inhibitor carfilzomib (Kyprolis®) and low-dose dexamethasone induced responses in all three of the patients enrolled to date: one very good partial response (VGPR) and two partial responses (PR) with good tolerability. All of these patients had received carfilzomib in their most recent regimen and their disease was refractory to it.
- In an ongoing Phase 1 clinical trial, selinexor in patients with heavily pre-treated, progressive non-Hodgkin's lymphoma (NHL), including aggressive B-cell
- Published data in the journal Nature Neuroscience reporting that Karyopharm's first-in-class oral Selective Inhibitor of Nuclear Export, or SINE™, compounds reduce the progression of multiple sclerosis in preclinical models. In the paper, scientists from Icahn School of Medicine at Mount Sinai and Karyopharm describe the ability of SINE exportin 1 (XPO1) inhibitors to decrease neurodegenerative symptoms and disease progression in multiple sclerosis by reducing neuronal inflammation and preventing toxic factors in the brain from attacking and destroying the neuronal myelin coating.
Regulatory and Intellectual Property Updates:
- Received orphan drug designation for selinexor from the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma. Orphan designation was created to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases. Selinexor has previously received orphan designation in multiple myeloma from the European Medicines Agency (EMA). Orphan designation has also been granted in AML and DLBCL from both the FDA and the EMA. In addition, the EMA has previously granted orphan designation of selinexor for the treatment of CLL and SLL, including Richter's transformation.
- Received U.S. patent allowance covering composition of matter for selinexor. Once issued, this patent will provide patent protection for selinexor and pharmaceutical compositions comprising Selinexor through the middle of 2032.
Business Updates:
- Completed an underwritten public offering in
January 2015 of 2,950,000 shares of common stock at a price of$33.00 per share, before underwriting discounts and commission and estimated offering expenses, with net proceeds to Karyopharm of approximately$90.9 million . - Selected for addition to the NASDAQ Biotechnology Index which is designed to track the performance of the securities of NASDAQ-listed companies classified as either biotechnology or pharmaceuticals according to industry classification benchmarks.
Clinical Development Plans:
- Karyopharm is actively enrolling patients in three registration-directed clinical studies evaluating selinexor in older patients with relapsed/refractory AML (SOPRA study), in patients with relapsed/refractory DLBCL (SADAL study) and in patients with relapsed/refractory Richter's transformation (SIRRT study). Preliminary top-line data from all three studies are anticipated in the second half of 2016.
- Karyopharm met with the
FDA during the fourth quarter of 2014 to discuss the criteria and study design for potential accelerated approval of selinexor in patients with relapsed/refractory multiple myeloma. Based on that meeting, Karyopharm plans to initiate a single-arm trial in multiple myeloma called STORM, Selinexor Treatment of Refractory Myeloma, in the first half of 2015, which will initially include 80 patients. If the data from the initial 80 patients is promising, the study may be expanded to potentially support accelerated approval. - Karyopharm is also actively enrolling patients in four Phase 2 solid tumor studies evaluating selinexor in gynecologic malignancies (SIGN Study), glioblastoma multiforme (KING Study), metastatic prostate cancer (SHIP Study) and squamous head and neck, lung and esophageal cancers (STARRS Study). Karyopharm hopes to present interim data from SIGN and KING at an oncology meeting in mid-2015.
- Karyopharm plans to initiate a pivotal Phase 3 study of selinexor to treat liposarcoma in the second half of 2015.
- In addition, a number of investigator-sponsored (ISTs) or company-sponsored clinical studies evaluating the potential of selinexor in combination with either chemotherapy or targeted agents are currently ongoing or planned.
Fourth Quarter and Year End
Cash, cash equivalents and investments as of
For the year ended
Karyopharm reported a net loss 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 registration-directed 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) or any PAK4 inhibitor, 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. | ||
| Consolidated Balance Sheets | ||
| (in thousands, except share and per share amounts) | ||
| (unaudited) | ||
2014 |
2013 |
|
| ASSETS | ||
| Current assets: | ||
| Cash and cash equivalents | ||
| Short-term investments | 55,115 | — |
| Prepaid expenses and other current assets | 2,027 | 1,982 |
| Total current assets | 207,751 | 157,956 |
| Property and equipment, net | 2,754 | 240 |
| Long-term investments | 8,658 | — |
| Other assets | 774 | 30 |
| Restricted cash | 400 | — |
| Total assets | ||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||
| Current liabilities: | ||
| Accounts payable | ||
| Accrued expenses | 5,825 | 1,168 |
| Deferred revenue | — | 79 |
| Deferred rent | 126 | — |
| Other current liabilities | 62 | 305 |
| Total current liabilities | 12,301 | 3,292 |
| Deferred rent, net of current portion | 1,242 | — |
| Total liabilities | 13,543 | 3,292 |
| Commitments and contingencies | ||
| Stockholders' equity: | ||
| Preferred stock, |
— | — |
| Common stock, |
3 | 3 |
| Additional paid‑in capital | 345,166 | 217,500 |
| Accumulated other comprehensive loss | (29) | — |
| Accumulated deficit | (138,346) | (62,569) |
| Total stockholders' equity | 206,794 | 154,934 |
| Total liabilities and stockholders' equity | ||
| Karyopharm Therapeutics Inc. | ||||
| Consolidated Statements of Operations | ||||
| (in thousands, except share and per share amounts) | ||||
| (unaudited) | ||||
| For the Quarter Ended |
For the Year Ended |
|||
| 2014 | 2013 | 2014 | 2013 | |
| Contract and grant revenue | ||||
| Operating expenses: | ||||
| Research and development | 20,038 | 9,689 | 60,127 | 28,452 |
| General and administrative | 5,920 | 2,480 | 15,948 | 5,885 |
| Total operating expenses | 25,958 | 12,169 | 76,075 | 34,337 |
| Loss from operations | (25,942) | (12,148) | (75,846) | (33,950) |
| Other income (expense): | ||||
| Interest income | 42 | 2 | 97 | 3 |
| Interest expense | (1) | — | (1) | — |
| Other expense | (27) | — | (27) | — |
| Total other income (expense) | 14 | 2 | 69 | 3 |
| Net loss | ||||
| Net loss per share applicable to common stockholders‑basic and diluted | ||||
| Weighted‑average number of common shares outstanding used in net loss per share applicable to common stockholders‑ basic and diluted | 32,668,705 | 16,973,108 | 31,135,694 | 6,067,679 |
CONTACT:Justin Renz (617) 658-0574 jrenz@karyopharm.comGina Nugent (617) 460-3579 nugentcomm@aol.com
Source: Karyopharm Therapeutics
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