Full Press Release Details
Karyopharm Reports Third Quarter 2017 Financial Results and Highlights Recent Progress
Company Completes Exclusive Licensing Transaction Valued at Up To $193 Million Plus Royalties with Ono
Pharmaceutical Co. Ltd. for Selinexor and KPT-8602 In Japan and Certain Other Countries in Asia
Michael Falvey Named Chief Financial Officer
Fourteen Abstracts Selected for ASH 2017, Including Three Oral Presentations
Conference Call Scheduled for Today at 8:30 a.m. ET
NEWTON, Mass. November 2, 2017 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today reported
financial results for the third quarter 2017 and provided an overview of recent accomplishments and clinical development plans for its lead, novel, oral SINE compound selinexor (KPT-330), and other
pipeline assets including KPT-8602, its second-generation oral SINE compound, KPT-9274, its oral, dual inhibitor of
p21-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase (NAMPT).
We are very proud of our
accomplishments to date in 2017, especially the recent execution of an exclusive licensing transaction, valued at up to $193 million, plus royalties, with Ono Pharmaceutical Co. Ltd. for the development and commercialization of selinexor and KPT-8602 for all human oncology indications in Japan and certain other countries in Asia, said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. As we head into year end, we look
forward to reporting clinical data from multiple treatment arms of the Phase 1b/2 STOMP study evaluating oral selinexor in combination with several current backbone therapies for multiple myeloma (MM) at this year s American
Society of Hematology (ASH) Annual Meeting. Fourteen abstracts highlighting data for selinexor and our other pipeline assets have been selected for presentation at ASH, and we are pleased to be able to share these data with the medical community at
the meeting this year. We continue to execute on the Phase 2b STORM study on schedule, and we expect to report top-line results by April 2018.
Third Quarter 2017 and Recent Events, Highlights and Milestones:
Partnerships and Other Key Corporate Developments
Selinexor in Multiple Myeloma
Selinexor in Diffuse Large B-Cell Lymphoma
Selinexor in Solid Tumors
The Phase 3 portion of the SEAL study, which
was originally initiated in North America, is ongoing and has been expanded to include Europe. In this blinded, placebo-controlled Phase 3 study, up to 222 patients are expected to be enrolled and randomized 2:1 to receive either oral selinexor,
(60mg twice weekly) until disease progression or intolerability, or placebo. Patients whose disease progresses on placebo will be permitted to cross over to the selinexor arm. The primary endpoint of the Phase 3 portion of the study is PFS (RECIST
v1.1) as assessed by the ICRR. The Phase 3 study design and primary endpoint of PFS were agreed to by the FDA. Top-line data from the Phase 3 portion of the SEAL study are anticipated by the end
of 2019. Assuming a positive outcome, these data are expected to support a New Drug Application for oral selinexor as a potential new treatment for patients with advanced unresectable
dedifferentiated liposarcoma.
Third Quarter 2017 Financial Results
equivalents and investments as of September 30, 2017, including restricted cash, totaled $159.4 million, compared to $175.5 million as of December 31, 2016.
For the quarter ended September 30, 2017, research and development expense was $25.2 million compared to $19.9 million for the quarter ended
September 30, 2016. For the quarter ended September 30, 2017, general and administrative expense was $5.8 million compared to $5.9 million for the quarter ended September 30, 2016.
Karyopharm reported a net loss of $30.6 million, or $0.65 per share, for the quarter ended September 30, 2017, compared to a net loss of
$25.4 million, or $0.69 per share, for the quarter ended September 30, 2016. Net loss includes stock-based compensation expense of $4.9 million and $5.6 million for the quarters ended September 30, 2017 and
September 30, 2016, respectively.
Karyopharm expects its operating cash burn, including research and development and general and administrative expenses, for the year ending December 31,
2017 to be approximately $95 million. Based on current operating plans, Karyopharm expects that its existing cash and cash equivalents will be sufficient to fund its research and development programs and operations into 2019, including the
continued clinical development of selinexor in the Company s lead indications with a focus on filing a new drug application with the FDA requesting accelerated approval in MM during 2018, assuming positive data from the STORM study, and
preparing the commercial infrastructure for the potential launch of selinexor in North America and Western Europe. Additional key activities for 2018 include topline data from the SADAL study targeted for the second half of 2018 and continued
enrollment in the Phase 3 BOSTON and SEAL studies.
Conference Call Information
Karyopharm will host a conference call today, Thursday, November 2, 2017, at 8:30 a.m. Eastern Time, to discuss the third quarter 2017 financial results,
recent accomplishments, clinical developments and business plans. To access the conference call, please dial (855) 437-4406 or (484) 756-4292
(international) at least five minutes prior to the start time and refer to conference ID: 98527624. An audio recording 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.
About Karyopharm Therapeutics
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). The Company s initial focus is on seeking regulatory approval and commercialization of its lead drug candidate, oral selinexor
(KPT-330). 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 the Company s
clinical development priorities for selinexor. 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 five investigational programs in clinical or preclinical development. For more information, please visit www.karyopharm.com.
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 potential to receive milestone and royalty payments under the license agreement with
Ono; the success of Karyopharm s arrangement with Ono and the parties ability to work effectively together; therapeutic potential of and potential clinical
development plans for Karyopharm s drug candidates, including the timing of enrollment of certain trials and of the reporting of data from such trials, and Karyopharm s financial
outlook and financial projections for Karyopharm. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Karyopharm 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 second-generation oral
SINE compound, or KPT-9274, Karyopharm s first-in-class oral dual inhibitor of PAK4 and NAMPT, 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 FDA and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, including with respect to the need for additional clinical studies;
Karyopharm s ability to obtain and maintain requisite regulatory approvals and to enroll patients in its clinical trials; the ability of Karyopharm or Ono to fully perform their respective obligations under the license agreement; the impact of
volatility in currency exchange rates; unplanned cash requirements and expenditures; development of drug candidates by Karyopharm s competitors for diseases in which Karyopharm is currently developing its drug candidates; and Karyopharm s
ability to obtain, maintain and enforce patent and other intellectual property protection for any drug candidates it is developing. These and other risks are described under the caption Risk Factors in Karyopharm s Quarterly Report
on Form 10-Q for the quarter ended June 30, 2017, which was filed with the Securities and Exchange Commission (SEC) on August 8, 2017, and in other filings that Karyopharm may make with the SEC in
the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by law, Karyopharm expressly disclaims any obligation to update any forward-looking statements, whether as a result
of new information, future events or otherwise.
Velcade is a registered trademark of Takeda
Pharmaceutical Company Limited
Pomalyst are registered trademarks of Celgene Corporation
Darzalex is a registered trademark of Janssen Biotech, Inc.
Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED BALANCE SHEETS
thousands, except share and per share amounts)
| September 30, 2017 | December 31, 2016 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 54,450 | $ | 49,663 | ||||
| Short-term investments | 64,956 | 79,889 | ||||||
| Restricted cash | 200 | |||||||
| Prepaid expenses and other current assets | 2,076 | 2,084 | ||||||
| Total current assets | 121,682 | 131,636 | ||||||
| Property and equipment, net | 2,304 | 2,836 | ||||||
| Long-term investments | 39,498 | 45,434 | ||||||
| Restricted cash | 289 | 479 | ||||||
| Total assets | $ | 163,773 | $ | 180,385 | ||||
| Liabilities and stockholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,890 | $ | 4,751 | ||||
| Accrued expenses | 17,715 | 11,362 | ||||||
| Deferred revenue | 1,050 | |||||||
| Deferred rent | 298 | 280 | ||||||
| Other current liabilities | 202 | 83 | ||||||
| Total current liabilities | 21,155 | 16,476 | ||||||
| Deferred rent, net of current portion | 1,441 | 1,666 | ||||||
| Total liabilities | 22,596 | 18,142 | ||||||
| Stockholders equity: | ||||||||
| Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding | ||||||||
| Common stock, $0.0001 par value; 100,000,000 shares authorized; 47,154,204 and 41,887,829 shares issued and outstanding at September 30, 2017 and December 31, 2016, respectively | 5 | 4 | ||||||
| Additional paid-in capital | 597,562 | 528,617 | ||||||
| Accumulated other comprehensive loss | (90 | ) | (274 | ) | ||||
| Accumulated deficit | (456,300 | ) | (366,104 | ) | ||||
| Total stockholders equity | 141,177 | 162,243 | ||||||
| Total liabilities and stockholders equity | $ | 163,773 | $ | 180,385 |
Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
thousands, except share and per share amounts)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2017 | 2016 | 2017 | 2016 | |||||||||||||
| Contract and grant revenue | $ | $ | 48 | $ | 71 | $ | 107 | |||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 25,237 | 19,893 | 72,440 | 66,267 | ||||||||||||
| General and administrative | 5,818 | 5,897 | 18,717 | 17,407 | ||||||||||||
| Total operating expenses | 31,055 | 25,790 | 91,157 | 83,674 | ||||||||||||
| Loss from operations | (31,055 | ) | (25,742 | ) | (91,086 | ) | (83,567 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income | 454 | 311 | 1,266 | 926 | ||||||||||||
| Other income (expense) | (26 | ) | 6 | (70 | ) | (1 | ) | |||||||||
| Total other income (expense), net | 428 | 317 | 1,196 | 925 | ||||||||||||
| Loss before income taxes | (30,627 | ) | (25,425 | ) | (89,890 | ) | (82,642 | ) | ||||||||
| Provision for income taxes | (13 | ) | (54 | ) | ||||||||||||
| Net loss | $ | (30,640 | ) | $ | (25,425 | ) | $ | (89,944 | ) | $ | (82,642 | ) | ||||
| Net loss per share basic and diluted | $ | (0.65 | ) | $ | (0.69 | ) | $ | (2.00 | ) | $ | (2.28 | ) | ||||
| Weighted-average number of common shares outstanding used in net loss per share basic and diluted | 47,141,146 | 36,819,329 | 44,974,945 | 36,223,324 |