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FDA Advisory Committee Votes 8 to 5 Recommending FDA Wait for the Results from the Ongoing
Phase 3 BOSTON Study to Make an Approval Decision for Selinexor --
-- Company Working with FDA to Evaluate Best Path Forward as FDA Completes Its Review of Selinexor
NDA; Assigned PDUFA Action Date of April 6, 2019 --
-- Conference Call Scheduled for Today at 8:30 a.m. ET --
NEWTON, Mass., Feb. 28, 2019 (GLOBE NEWSWIRE) -- Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today reported financial results for the fourth quarter and full year 2018 and provided a business update and an overview of recent accomplishments for selinexor, its first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound.
“Earlier this week, the U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) voted 8 to 5, recommending that the FDA wait for the results from the ongoing Phase 3 BOSTON study before making a final approval decision regarding selinexor for the treatment of patients with triple class refractory multiple myeloma who have received at least three prior therapies,” said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. “While we are disappointed with the ODAC’s recommendation, we are encouraged by the support communicated by a number of ODAC members. We intend to work closely with the FDA to evaluate the best path forward as they complete their review of our New Drug Application (NDA). We remain committed to delivering on our vision of bringing selinexor into the hands of the physicians and patients who are battling highly refractory multiple myeloma.”
Fourth Quarter 2018 and Recent Events
Selinexor in Multiple Myeloma
The ODAC is an independent panel of experts that evaluates data concerning the efficacy and safety of marketed and investigational products for use in the treatment of cancer and makes appropriate recommendations to the FDA. Although the FDA will consider the recommendation of the panel, the final decision regarding the approval of the product is made by the FDA solely, and the recommendations by the panel are non-binding.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)
Selinexor in Solid Tumors
Full Year and Fourth Quarter 2018 Financial Results
Cash, cash equivalents and investments as of December 31, 2018, including restricted cash, totaled $330.9 million, compared to $176.4 million as of December 31, 2017.
On October 26, 2018, Karyopharm completed a private offering of $172.5 million aggregate principal amount of 3.00% convertible senior notes due in 2025, including the full exercise of the initial purchasers’ option to purchase additional notes. After deducting the initial purchasers’ discounts and commissions and other offering expenses the net proceeds were $166.9 million.
License and other revenue for the year ended December 31, 2018 was $30.3 million, compared to $1.6 million for the year ended December 31, 2017, primarily related to the Company’s license agreements with Biogen and ONO.
For the year ended December 31, 2018, research and development expense was $161.4 million compared to $107.3 million for the year ended December 31, 2017. For the year ended December 31, 2018, general and administrative expense was $48.8 million compared to $24.9 million for the year ended December 31, 2017.
Karyopharm reported a net loss of $178.4 million, or $3.14 per share, for the year ended December 31, 2018, compared to a net loss of $129.0 million, or $2.81 per share, for the year ended December 31, 2017. Net loss includes stock-based compensation expense of $17.3 million and $20.4 million for the years ended December 31, 2018 and December 31, 2017, respectively.
For the quarter ended December 31, 2018, research and development expense was $38.9 million, compared to $34.8 million for the quarter ended December 31, 2017. For the quarter ended December 31, 2018, general and administrative expense $18.8 million, compared to $6.2 million for the quarter ended December 31, 2017.
Karyopharm reported a net loss of $58.2 million, or $0.96 per share for the quarter ended December 31, 2018, compared to a net loss of $39.0 million, or $0.80 per share for the quarter ended December 31, 2017. Net loss includes stock-based compensation expense of $3.9 million and $4.5 million for the quarters ended December 31, 2018 and December 31, 2017, respectively.
Based on its current operating plans, Karyopharm expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations into the second half of 2020, which currently assumes the commercial launch of selinexor in the U.S. in the second quarter of 2019. If the FDA decides to delay its approval decision for selinexor until the BOSTON data is available, Karyopharm will re-evaluate its spending expectations for 2019. Additional key activities expected in 2019 include supporting the ongoing multiple myeloma regulatory filings for selinexor in the U.S. and Europe, progressing the pivotal Phase 3 BOSTON study in multiple myeloma and potentially submitting an NDA and MAA, in the U.S. and Europe, respectively, in DLBCL.
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 overall response rate. 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 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 ongoing pivotal, Phase 3 BOSTON study, and has reminded the Company that accelerated approval requires patients to have exhausted all available approved therapies.
Conference Call Information
Karyopharm will host a conference call today, Thursday, February 28, 2019, at 8:30 a.m. Eastern Time, to discuss the fourth quarter and full year 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 4246798. 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). In 2018, Karyopharm reported positive data from the Phase 2b STORM study evaluating selinexor in combination with low-dose dexamethasone in patients with triple class refractory multiple myeloma who have been previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available. Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track designation for the patient population evaluated in the STORM study. Karyopharm’s New Drug Application has been accepted for filing and granted Priority Review by the FDA, and oral selinexor is currently under review by the FDA as a possible new treatment for patients with triple class refractory multiple myeloma who have received at least three prior therapies. The Company has also submitted a Marketing Authorization Application to the European Medicines Agency with a request for conditional approval and was granted accelerated assessment. Selinexor is also being studied in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). In 2018, Karyopharm reported positive top-line results from the Phase 2b SADAL study evaluating selinexor in patients with relapsed or refractory DLBCL after at least two prior multi-agent therapies and who are ineligible for transplantation, including high dose chemotherapy with stem cell rescue. Selinexor has received Fast Track designation from the FDA for the patient population evaluated in the SADAL study. 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), as a potential backbone therapy in combination with approved therapies (STOMP), in 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 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. 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 our expectations relating to submissions, to and the review and potential approval of selinexor by, regulatory authorities, including the anticipated timing of such submissions and actions, and the potential availability of accelerated approval pathways, the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, especially selinexor, and the plans for commercialization. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond Karyopharm’s control, 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 triple class refractory myeloma or the SADAL study in patients with relapsed or refractory DLBCL or that any of Karyopharm's drug candidates, including selinexor, will successfully complete necessary 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, 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; 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 September 30, 2018, which was filed with the Securities and Exchange Commission (SEC) on November 8, 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
Darzalex® is a registered trademark of Janssen Biotech, Inc.
Karyopharm Therapeutics Inc.
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Karyopharm Therapeutics Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
|Cash and cash equivalents||$||118,021||$||68,997|
|Prepaid expenses and other current assets||6,413||1,754|
|Total current assets||334,612||148,423|
|Property and equipment, net||3,863||2,185|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Other current liabilities||327||133|
|Total current liabilities||46,904||49,467|
|Convertible senior notes||102,664||—|
|Deferred revenue, net of current portion||4,532||—|
|Deferred rent, net of current portion||3,922||1,363|
|Commitments and contingencies (Note 8)|
|Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding||—||—|
|Common stock, $0.0001 par value; 100,000,000 shares authorized; 60,829,308 and 49,533,150
shares issued and outstanding at December 31, 2018 and 2017, respectively
|Additional paid-in capital||857,156||625,017|
|Accumulated other comprehensive loss||(244||)||(217||)|
|Total stockholders’ equity||183,170||129,464|
|Total liabilities and stockholders’ equity||$||341,192||$||180,294|
Karyopharm Therapeutics Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
For the Quarter Ended,
For the Year Ended
|License and other revenue||$||206||$||1,534||$||30,336||$||1,605|
|Research and development||38,890||34,833||161,372||107,273|
|General and administrative||18,771||6,153||48,847||24,870|
|Total operating expenses||57,661||40,986||210,219||132,143|
|Loss from operations||(57,455||)||(39,452||)||(179,883||)||(130,538||)|
|Other income (expense):|
|Total other (expense) income, net||(738||)||421||1,502||1,617|
|Loss before income taxes||(58,193||)||(39,031||)||(178,381||)||(128,921||)|
|Provision for income taxes||(17||)||(9||)||(26||)||(63||)|
|Net loss per share—basic and diluted||$||(0.96||)||$||(0.80||)||$||(3.14||)||$||(2.81||)|
|Weighted-average number of common shares outstanding used
in net loss per share—basic and diluted