Full Press Release Details
Karyopharm Reports First Quarter 2018 Financial Results and Highlights Recent Progress
Oral selinexor Achieves 25.4% Overall Response Rate and Median Duration of Response of 4.4 Months in Patients with Penta-Refractory
Completed $155.3 Million Public Equity Offering, Including Full Exercise of Underwriters Option to Purchase
Company Plans to Submit NDA Requesting Accelerated Approval for Selinexor in Penta-Refractory
Myeloma During Second Half of 2018
Conference Call Scheduled for Today at 8:30 a.m. ET
NEWTON, Mass. May 10, 2018 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today reported
financial results for the first quarter 2018 and provided an overview of recent accomplishments and clinical development plans for selinexor, its lead, novel, oral SINE compound, eltanexor, its second-generation oral SINE compound, and KPT-9274 its novel, oral, dual inhibitor of PAK4 and NAMPT.
top-line data recently reported from the Phase 2b STORM study evaluating selinexor in patients with penta-refractory myeloma are an important step forward toward the approval of selinexor, said Michael
G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. Our progress is further enabled by the successful completion of a $155.3 million equity financing and the receipt of Fast Track designation from the U.S. Food and Drug
Administration (FDA) for selinexor for the treatment of patients with penta-refractory multiple myeloma. We look forward to submitting a New Drug Application (NDA) to the FDA during the second half of 2018, with a request for accelerated approval
for selinexor in penta-refractory myeloma, followed thereafter by a Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in early 2019, with a request for conditional approval in the same indication.
First Quarter 2018 and Recent Events
Selinexor in Diffuse Large
B-Cell Lymphoma (DLBCL)
Selinexor in Solid Tumors
Corporate and Financial
March 31, 2018 Financial Results
Cash, cash equivalents and investments as of March 31, 2018, including restricted cash, totaled
$141.5 million, compared to $176.4 million as of December 31, 2017.
On May 7, 2018, Karyopharm completed an underwritten public
offering of 10,525,424 shares of its common stock at a price to the public of $14.75 per share. The gross proceeds to Karyopharm from the offering were $155.3 million. After deducting the underwriting discounts and commissions and other
estimated offering expenses the net proceeds were $145.6 million.
For the quarter ended March 31, 2018, Karyopharm recognized
$10.0 million in revenue, compared to $0.1 million for the three months ended March 31, 2017. The increase in revenue was the result of the upfront payment received from the asset sale of
KPT-350 to Biogen in January 2018.
For the quarter ended March 31, 2018, research and development expense
was $41.3 million compared to $24.1 million for the quarter ended March 31, 2017. For the quarter ended March 31, 2018, general and administrative expense was $7.6 million compared to $6.3 million for the quarter ended
Karyopharm reported a net loss of $38.5 million, or $0.78 per share, for the quarter ended March 31, 2018, compared to a
net loss of $29.9 million, or $0.71 per share, for the quarter ended March 31, 2017. Net loss includes stock-based compensation expense of $4.2 million and $5.9 million for the quarters ended March 31, 2018 and
March 31, 2017, respectively.
Karyopharm expects its operating cash burn, including research and development and general and administrative expenses, for the year ending December 31,
2018 to be in the range of $175 to 185 million. Based on current operating plans, Karyopharm expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations into the third quarter of 2019. These plans
include the continued clinical development of selinexor in the Company s lead indications with a focus on filing an NDA with the FDA requesting accelerated approval in multiple myeloma during 2018 and preparing the commercial infrastructure and
hiring a sales force for the potential launch of selinexor in the U.S. Additional key milestones expected in 2018 include preparing for a potential MAA submission to the EMA requesting conditional approval for selinexor in multiple myeloma, topline
data from the SADAL study and completion of enrollment in the Phase 3 BOSTON study.
Further Information About Potential Accelerated Approval for
Selinexor in Multiple Myeloma
The FDA instituted its Accelerated Approval Program to allow for expedited approval of drugs that treat serious
conditions and that fill an unmet medical need based on a surrogate endpoint or an intermediate clinical endpoint thought to predict clinical benefit, like ORR. Accelerated approval is available only for drugs that provide a meaningful therapeutic
benefit over existing treatments at the time of consideration of the application for accelerated approval, which the FDA has recently reiterated in its feedback to the Company. Particularly in disease areas with multiple available and potential new
therapies, such as multiple myeloma, accelerated approval carries a high regulatory threshold. Consistent with its general guidance, the FDA has noted to the Company its preference for randomized studies geared toward full approval, which the
Company has undertaken with the pivotal, Phase 3 BOSTON study, and has reminded the Company that accelerated approval requires patients to have exhausted approved therapies. The Company recently received Fast Track designation for selinexor for the
treatment of patients with penta-refractory myeloma from the FDA, which is available to therapeutics treating an unmet medical need in a serious condition. In light of this recognition that the STORM patient population represents an unmet medical
need and the positive top-line data reported previously, the Company believes that the STORM study should support its request to the FDA for accelerated approval.
Conference Call Information
Karyopharm will host a
conference call today, Thursday, May 10, 2018, at 8:30 a.m. Eastern Time, to discuss the first quarter 2018 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference ID 6798355. A live audio webcast of the call will be
available under Events & Presentations in the Investor section of the Company s website, http://investors.karyopharm.com/events-presentations. An archived webcast will be available on the Company s website
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 2,400 patients have been treated with selinexor. In April 2018, Karyopharm reported positive top-line data from the Phase 2b STORM study
evaluating selinexor in combination with low-dose dexamethasone in patients with penta-refractory multiple myeloma. Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track
designation for the patient population evaluated in the STORM study. Karyopharm plans to submit a
New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) during the second half of 2018, with a request for accelerated approval for oral selinexor as a new treatment for
patients with penta-refractory multiple myeloma. The Company also plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in early 2019 with a request for conditional approval. Selinexor is also being
evaluated in several other 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) and as a potential backbone therapy in combination with approved therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), liposarcoma (SEAL), and an investigator-sponsored study in endometrial cancer (SIENDO), 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 Karyopharm 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.
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 timing of submissions to regulatory authorities and the potential availability of accelerated approval pathways, therapeutic potential of and potential clinical development plans for
Karyopharm s drug candidates, including the timing of enrollment of certain trials, the reporting of data from such trials and the impact on potential regulatory filings, the potential to receive milestone and royalty payments under third party
arrangements 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 regulators will agree that selinexor qualifies for accelerated approval in the U.S. or conditional approval in the E.U. as a result of the data from the STORM study
in patients with penta-refractory myeloma or that any of Karyopharm s drug candidates, including selinexor (KPT-330), eltanexor (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 its third party collaborators or successors in interest to fully perform their respective
obligations under collaboration or license agreements and the potential future implications of such agreements; 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, 2017, which was filed with the Securities and Exchange Commission (SEC) on March 15, 2018, 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
Kyprolis is a registered trademark of Onyx Pharmaceuticals, Inc.
Darzalex is a registered trademark of Janssen Biotech, Inc.
Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED BALANCE SHEETS
thousands, except share and per share amounts)
| March 31, 2018 | December 31, 2017 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 37,499 | $ | 68,997 | ||||
| Short-term investments | 93,418 | 77,472 | ||||||
| Prepaid expenses and other current assets | 2,396 | 1,754 | ||||||
| Restricted cash | 200 | |||||||
| Total current assets | 133,313 | 148,423 | ||||||
| Property and equipment, net | 2,454 | 2,185 | ||||||
| Long-term investments | 10,314 | 29,396 | ||||||
| Restricted cash | 292 | 290 | ||||||
| Total assets | $ | 146,373 | $ | 180,294 | ||||
| Liabilities and stockholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 4,949 | $ | 5,665 | ||||
| Accrued expenses | 21,545 | 21,445 | ||||||
| Deferred revenue | 19,729 | 21,921 | ||||||
| Deferred rent | 178 | 303 | ||||||
| Other current liabilities | 333 | 133 | ||||||
| Total current liabilities | 46,734 | 49,467 | ||||||
| Deferred revenue, net of current portion | 2,192 | |||||||
| Deferred rent, net of current portion | 1,918 | 1,363 | ||||||
| Total liabilities | 50,844 | 50,830 | ||||||
| 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; 49,670,328 and 49,533,150 shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively | 5 | 5 | ||||||
| Additional paid-in capital | 629,610 | 625,017 | ||||||
| Accumulated other comprehensive loss | (286 | ) | (217 | ) | ||||
| Accumulated deficit | (533,800 | ) | (495,341 | ) | ||||
| Total stockholders equity | 95,529 | 129,464 | ||||||
| Total liabilities and stockholders equity | $ | 146,373 | $ | 180,294 |
Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
thousands, except share and per share amounts)
| Three Months Ended, March 31, | ||||||||
| 2018 | 2017 | |||||||
| License and other revenue | $ | 10,000 | $ | 68 | ||||
| Operating expenses: | ||||||||
| Research and development | 41,321 | 24,083 | ||||||
| General and administrative | 7,621 | 6,264 | ||||||
| Total operating expenses | 48,942 | 30,347 | ||||||
| Loss from operations | (38,942 | ) | (30,279 | ) | ||||
| Other income (expense): | ||||||||
| Interest income | 509 | 400 | ||||||
| Other expense | (14 | ) | (15 | ) | ||||
| Total other income, net | 495 | 385 | ||||||
| Loss before income taxes | (38,447 | ) | (29,894 | ) | ||||
| Provision for income taxes | (12 | ) | (23 | ) | ||||
| Net loss | $ | (38,459 | ) | $ | (29,917 | ) | ||
| Net loss per share basic and diluted | $ | (0.78 | ) | $ | (0.71 | ) | ||
| Weighted-average number of common shares outstanding used in net loss per share basic and diluted | 49,602,809 | 41,894,796 |