Full Press Release Details
Karyopharm Reports Fourth Quarter and Full Year 2014 Financial Results and Highlights Recent Progress
Initiated Multiple Registration-directed Trials and Presented Positive Selinexor Clinical Data in 2014
Aggressive Selinexor Development Campaign Continues in 2015
Conference Call Scheduled for today at 8:30 a.m. ET
Newton, Mass. March 16, 2015 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today reported
financial results for the fourth quarter and year ended December 31, 2014 and commented on recent accomplishments and clinical development plans for selinexor, its lead product candidate.
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 Michael Kauffman, MD, PhD, CEO of Karyopharm. We initiated two additional registration-directed clinical studies
in hematologic malignancies, bringing our current total to three. We also presented positive clinical data demonstrating selinexor s 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 dexamethasone and/or carfilzomib in patients with heavily pretreated multiple myeloma. In addition, we have now been awarded orphan drug designation for selinexor by both the U.S. and
European regulatory authorities in multiple myeloma, acute myeloid leukemia (AML), diffuse large B-cell lymphoma (DLBCL), chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), including Richter s transformation.
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 Sharon Shacham, PhD, Karyopharm s President and
Chief Scientific Officer. In addition to our own internal efforts, selinexor is being studied broadly alone and in combination with other therapies in a variety of investigator-sponsored trials across both hematologic and solid tumor
indications and we look forward to reporting those results when available.
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
Scientific Presentations and Publications:
Regulatory and Intellectual Property Updates:
Clinical Development Plans:
Fourth Quarter and Year End December 31, 2014 Financial Results
Cash, cash equivalents and investments as of December 31, 2014, including restricted cash, totaled $214.8 million, compared to $156.0 million as of
December 31, 2013. In addition, Karyopharm raised $90.9 million in a common stock follow-on offering which closed in January 2015.
ended December 31, 2014, research and development expense was $60.1 million compared to $28.5 million for the year ended December 31, 2013. For the year ended December 31, 2014, general and administrative expense was $15.9 million,
compared to $5.9 million for the year ended December 31, 2013. The increase in research and development expenses resulted primarily from the increase in expenses related to the significantly expanded clinical development activities for our lead
drug candidate, selinexor. The increase in general and administrative expense resulted primarily from an increase in personnel costs including headcount and stock-based compensation expense and the costs of operating as a public company.
Karyopharm reported a net loss of $75.8 million, or $2.43 per share, for the year ended December 31, 2014, compared to a net loss of $33.9 million, or
$5.59 per share, for the year ended December 31, 2013. Net loss includes stock-based compensation expense of $14.2 million and $3.8 million for the years ended December 31, 2014 and 2013, respectively. The weighted average number of shares
of common stock outstanding increased to 31.1 million for the year ended December 31, 2014 from 6.1 million for the year ended December 31, 2013 as a result of our initial public offering in November 2013 and follow-on offering in July
For the quarter ended December 31, 2014, research and development expense was $20.0 million compared to $9.7 million for the quarter ended
December 31, 2013. For the quarter ended December 31, 2014, general and administrative expense was $5.9 million compared to $2.5 million for the quarter ended December 31, 2013. The increase in research and development expenses
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 $25.9 million, or $0.79 per share, for the quarter ended December 31, 2014, compared to
a net loss of $12.1 million, or $0.72 per share, for the quarter ended December 31, 2013. Net loss includes stock-based compensation expense of $4.6 million and $2.0 million for the quarters ended December 31, 2014 and December 31,
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 $200 million in cash, cash equivalents and investments.
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 targets for the treatment of cancer and other major diseases. Karyopharm s Selective Inhibitor of Nuclear Export / SINE compounds function by binding with and inhibiting the nuclear export protein XPO1
(or CRM1). The inhibition of XPO1 by Karyopharm s lead drug candidate, Selinexor (KPT-330), a first-in-class, oral SINE compound, leads to the accumulation of tumor suppressor proteins in the cell nucleus, which subsequently reinitiates
and amplifies their tumor suppressor function. SINE compounds have shown biological activity in models of cancer, inflammation, autoimmune disease, certain viruses, and wound-healing. Karyopharm was founded by Dr. Sharon Shacham and
is located in Newton, Massachusetts. For more information about Karyopharm, 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 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 U.S. Food and Drug Administration and other regulatory authorities,
investigational review boards at clinical trial sites and publication review bodies; Karyopharm s ability to obtain and maintain requisite regulatory approvals and to enroll patients in its clinical trials; 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 Annual Report on Form 10-K for the year ended December 31, 2014, which is on file
with the Securities and Exchange Commission (SEC) as of March 13, 2015. Any forward-looking statements contained in this press release speak only as of the date hereof, and Karyopharm expressly disclaims any obligation to update any
forward-looking statements, whether as a result of new information, future events or otherwise.
Karyopharm Therapeutics Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
| December 31, 2014 | December 31, 2013 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 150,609 | $ | 155,974 | ||||
| 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 | $ | 220,337 | $ | 158,226 | ||||
| LIABILITIES AND STOCKHOLDERS EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 6,288 | $ | 1,740 | ||||
| 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, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding | ||||||||
| Common stock, $0.0001 par value; 100,000,000 shares authorized at December 31, 2014 and 2013, respectively; 32,699,380 and 29,587,258 shares issued and outstanding at December 31, 2014 and 2013, respectively | 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 | $ | 220,337 | $ | 158,226 |
Karyopharm Therapeutics Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
| For the Quarter Ended December 31, | For the Year Ended December 31, | |||||||||||||||
| 2014 | 2013 | 2014 | 2013 | |||||||||||||
| Contract and grant revenue | $ | 16 | $ | 21 | $ | 229 | $ | 387 | ||||||||
| 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 | $ | (25,928 | ) | $ | (12,146 | ) | $ | (75,777 | ) | $ | (33,947 | ) | ||||
| Net loss per share applicable to common stockholders-basic and diluted | $ | (0.79 | ) | $ | (0.72 | ) | $ | (2.43 | ) | $ | (5.59 | ) | ||||
| 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 |