Full Press Release Details
Karyopharm Reports First Quarter 2016 Financial Results and Highlights Recent Progress
Phase 2/3 SEAL Clinical Trial Initiated with Selinexor for Treatment of Liposarcoma
First-in-Human Study Initiated with Second-Generation SINE Compound KPT-8602 in Multiple Myeloma
Phase 2 Clinical Data in Multiple Myeloma Expected Mid-Year
Conference Call Scheduled for Today at 8:30 a.m. ET
Newton, Mass. May 9, 2016 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today reported
financial results for the first quarter 2016 and commented on recent accomplishments and clinical development plans for its pipeline of several Selective Inhibitor of Nuclear Export (SINETM)-based
therapeutics, including selinexor, its lead product candidate, as well as KPT-9274, its oral dual inhibitor of PAK4 and NAMPT.
part of 2016, Karyopharm continued to execute on its clinical strategy with the initiation of two clinical trials a Phase 2/3 trial evaluating selinexor (KPT-330) in liposarcoma, and a first-in-human trial with its novel second-generation SINE
compound, KPT-8602, in multiple myeloma, said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. We also highlighted the depth and breadth of our development pipeline with the presentation of preclinical data
demonstrating encouraging activity with selinexor in combination with immune checkpoint inhibitors, a Bcl2 antagonist, and alkylating agents in oncology, KPT-8602 in multiple myeloma, KPT-350 in traumatic brain injury, and verdinexor (KPT-335)
across a number of viral disease indications. Looking ahead, we expect to report top-line data from two of our ongoing studies, STORM and STOMP in relapsed/refractory multiple myeloma, during the second half of 2016.
Conference Call Information:
Karyopharm will host a
conference call today, Monday, May 9, 2016, at 8:30 a.m. Eastern Time, to discuss the first quarter 2016 financial results, recent accomplishments, clinical developments and business 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: 95849798. 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.
Development Plans and Timelines:
Scientific Presentations and Publications:
2016 American Association for Cancer Research (AACR) Annual Meeting, April 18-20, 2016 in New Orleans
29th International Conference on Antiviral Research (ICAR),
April 17-21, 2016 in La Jolla, California.
A number of viruses require XPO1 during their life cycle; XPO1 inhibition may inhibit viral
replication in these cases. Preclinical data demonstrating the activity of verdinexor across a number of infectious disease indications including influenza, HIV, respiratory syncytial virus (RSV) and Venezuelan equine encephalitis virus (VEEV) were
2016 Annual Meeting of the American Academy of Neurology (AAN), April 15-21, 2016 in Vancouver, Canada.
Preclinical data demonstrating the activity of KPT-350, for the treatment of neuro-inflammatory disorders including traumatic brain injury (TBI), were
First Quarter 2016 Financial Results
Cash, cash equivalents and investments as of March 31, 2016, including restricted cash, totaled $187.1 million, compared to $210.0 million as of
For the quarter ended March 31, 2016, research and development expense was $21.8 million compared to $20.8 million for the
quarter ended March 31, 2015. For the quarter ended March 31, 2016, general and administrative expense was $5.6 million compared to $5.4 million for the quarter ended March 31, 2015. The increase in research and development expenses
resulted primarily from the increase in expenses related to the continued clinical development of selinexor.
Karyopharm reported a net loss of $27.1
million, or $0.75 per share, for the quarter ended March 31, 2016, compared to a net loss of $26.1 million, or $0.74 per share, for the quarter ended March 31, 2015. Net loss includes stock-based compensation expense of $5.2 million and
$3.7 million for the quarters ended March 31, 2016 and March 31, 2015, respectively.
Based on current operating plans, Karyopharm expects that its existing cash and cash equivalents will fund its research and development programs and operations
into the middle of 2018, including advancing the STORM, SOPRA, SADAL and SEAL clinical studies to their next data inflection points. Karyopharm expects to end 2016 with at least $120 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 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). 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 was founded by Dr. Sharon Shacham and is located in Newton, Massachusetts. For more information, please visit
Forward-Looking Statements
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 oral dual inhibitor of PAK4 and NAMPT, verdinexor (KPT-335), KPT-350, 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,
2015, which was filed with the Securities and Exchange Commission (SEC) on March 15, 2016, 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 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.
CONDENSED CONSOLIDATED BALANCE SHEETS
thousands, except share and per share amounts)
| March 31, 2016 | December 31, 2015 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 42,188 | $ | 58,358 | ||||
| Short-term investments | 94,228 | 117,275 | ||||||
| Prepaid expenses and other current assets | 1,726 | 1,967 | ||||||
| Total current assets | 138,142 | 177,600 | ||||||
| Property and equipment, net | 3,343 | 3,483 | ||||||
| Long-term investments | 50,165 | 33,878 | ||||||
| Restricted cash | 485 | 482 | ||||||
| Total assets | $ | 192,135 | $ | 215,443 | ||||
| Liabilities and stockholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 4,109 | $ | 3,808 | ||||
| Accrued expenses | 8,652 | 11,023 | ||||||
| Deferred rent | 263 | 206 | ||||||
| Other current liabilities | 198 | 95 | ||||||
| Total current liabilities | 13,222 | 15,132 | ||||||
| Deferred rent, net of current portion | 1,877 | 1,946 | ||||||
| Total liabilities | 15,099 | 17,078 | ||||||
| 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; 35,924,738 and 35,864,765 shares issued and outstanding at March 31, 2016 and December 31, 2015, respectively | 4 | 4 | ||||||
| Additional paid-in capital | 460,524 | 455,170 | ||||||
| Accumulated other comprehensive income (loss) | 94 | (282 | ) | |||||
| Accumulated deficit | (283,586 | ) | (256,527 | ) | ||||
| Total stockholders equity | 177,036 | 198,365 | ||||||
| Total liabilities and stockholders equity | $ | 192,135 | $ | 215,443 |
Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
thousands, except share and per share amounts)
| Three Months Ended, March 31, | ||||||||
| 2016 | 2015 | |||||||
| Operating expenses: | ||||||||
| Research and development | $ | 21,795 | $ | 20,751 | ||||
| General and administrative | 5,554 | 5,399 | ||||||
| Total operating expenses | 27,349 | 26,150 | ||||||
| Loss from operations | (27,349 | ) | (26,150 | ) | ||||
| Other income (expense): | ||||||||
| Interest income | 286 | 141 | ||||||
| Other income (expense) | 4 | (58 | ) | |||||
| Total other income (expense), net | 290 | 83 | ||||||
| Net loss | $ | (27,059 | ) | $ | (26,067 | ) | ||
| Net loss per share basic and diluted | $ | (0.75 | ) | $ | (0.74 | ) | ||
| Weighted-average number of common shares outstanding used in net loss per share basic and diluted | 35,878,502 | 35,317,181 |