“We have made tremendous progress toward advancing our lead drug candidate, selinexor, as we strive to improve the lives of patients with myeloma and other forms of cancer. Most notably, we have now completed the submission of our first New Drug Application (NDA) to the
Second Quarter 2018 and Recent Events
Selinexor in Multiple Myeloma
- Submitted NDA in Penta-Refractory Myeloma. Karyopharm completed its submission of an NDA to the
FDAseeking accelerated approval for selinexor, its lead, novel, oral SINE compound, as a new treatment for patients with penta-refractory multiple myeloma. Patients with penta-refractory myeloma have previously received the two proteasome inhibitors (PIs), Velcade® (bortezomib) and Kyprolis® (carfilzomib), the two immunomodulatory drugs (IMiDs), Revlimid® (lenalidomide) and Pomalyst® (pomalidomide), and the anti-CD38 monoclonal antibody Darzalex® (daratumumab) as well as alkylating agents, and their disease is refractory to at least one PI, at least one IMiD, Darzalex and their most recent therapy. The Company also plans to submit an MAA to the EMA in early 2019 with a request for conditional approval in the same indication.
- Reported Positive Top-line Data from the Phase 2b STORM Study in Patients with Penta-Refractory Myeloma. Karyopharm reported positive top-line results from Part 2 of the Phase 2b STORM study evaluating selinexor plus low dose dexamethasone (Sd) in patients with penta-refractory multiple myeloma. The STORM study’s primary endpoint of overall response rate (ORR) was 25.4%, which included two stringent complete responses (sCRs) and 29 partial or very good partial responses. The two sCRs were negative for minimal residual disease, one at 1:10-6 and one at 1:10-4, which is particularly significant in this penta-refractory population. The median duration of response, a key secondary objective, was 4.4 months. Across the relevant patient population, side effects of oral selinexor were generally predictable and often managed with dose adjustments or supportive care. Safety results were consistent with those previously reported from Part 1 of this study and from other selinexor studies and no new safety signals were identified. Karyopharm plans to submit detailed STORM study results for presentation at an upcoming medical oncology meeting.
- Presented Updated Data from the Phase 1b/2 STOMP Study at the
European Hematology Association(EHA) 2018 Annual Meeting. At EHA 2018, Karyopharm presented three posters with updated data from STOMP evaluating selinexor and dexamethasone in combination with standard approved therapies, Velcade (SVd), Pomalyst (SPd) or Darzalex (SDd), in patients with previously treated multiple myeloma. The SVd arm demonstrated progression-free survival (PFS) of 17.8 months, an ORR of 83% in the same patient population eligible for the pivotal Phase 3 BOSTON study. The SDd arm demonstrated an ORR of 82% in patients with heavily pretreated Darzalex-naïve disease. The all oral SPd arm demonstrated an ORR of 55% in patients with Pomalyst-naïve and Revlimid-relapsed or -refractory disease, with a PFS of 11.6 months. Adverse events across all three arms were consistent with those reported previously with selinexor and the combination therapies, with no new safety signals specific to the combinations identified. These results suggest that selinexor can be combined with other anti-myeloma agents and induce durable responses in patients with previously treated MM.
- Initiated New STOMP Arm Evaluating All Oral Regimen of Selinexor, Revlimid and Dexamethasone (SRd) in Patients with Newly Diagnosed Myeloma. Based on the positive STOMP results reported to date evaluating SRd in patients with relapsed myeloma, the Company initiated a new, all oral STOMP arm to investigate the combination of SRd in the front-line setting. Given the observed synergistic activity of selinexor with standard approved multiple myeloma therapies, Karyopharm believes oral selinexor has the potential to be a future backbone therapy in multiple myeloma.
- Pivotal Phase 3 BOSTON Study in
Progress. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is underway and enrolling patients in 14 countries. BOSTONis evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (once weekly) and low-dose dexamethasone (SVd), compared to standard twice weekly Velcade and low-dose dexamethasone (Vd) in patients with multiple myeloma who have had one to three prior lines of therapy. The primary endpoints of the study are PFS and ORR. Data from the BOSTONstudy, if positive, would be used to support regulatory submissions to the FDAand EMA requesting full approvals for use of selinexor in second line multiple myeloma, following the Company’s requests for accelerated and conditional approvals, respectively, using data from the Phase 2b STORM study. The Company expects to enroll approximately 360 patients at over 100 clinical sites internationally and expects to complete enrollment by the end of 2018, with top-line data anticipated in 2019.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)
- Ongoing Phase 2b SADAL Study in DLBCL. Karyopharm is also investigating oral selinexor as a single-agent for the treatment of patients with relapsed or refractory DLBCL who are not eligible for stem cell transplantation. The SADAL study is expected to enroll up to a total of 130 patients in the single-arm cohort evaluating single-agent selinexor dosed 60mg twice weekly in patients who received two to five lines of prior therapy. Karyopharm plans to report top-line results by the end of 2018. Assuming the results from the SADAL study are positive, Karyopharm plans to submit an NDA to the
FDAwith a request for accelerated approval, and an MAA to the EMA with a request for conditional approval, for oral selinexor in this relapsed/refractory DLBCL patient population.
Selinexor in Solid Tumors
- Presented Data from the Phase 2 Portion of the Phase 2/3 SEAL Study in Liposarcoma at the
American Society of Clinical Oncology( ASCO) 2018 Annual Meeting. At ASCO2018, Karyopharm presented positive results from the successful Phase 2 portion of the blinded, randomized Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with previously treated, advanced unresectable dedifferentiated liposarcoma. For the primary endpoint of PFS, oral selinexor showed superiority over placebo, achieving a median PFS of 5.5 months, compared to 2.7 months for placebo with a hazard ratio (HR) of 0.67, representing a 33% reduction in the risk of progression or death. Across the relevant patient population, side effects of oral selinexor were generally predictable and often managed with dose adjustments or supportive care, with the most frequent events being nausea, fatigue, anorexia and weight loss, with low levels of Grade 3/4 cytopenias, and no new or unexpected safety signals identified. The Phase 3 portion of the SEAL study is underway and, assuming a positive outcome on the primary end point of PFS, the Company intends to use the data from the SEAL study to support an NDA and an MAA submission requesting full approval for oral selinexor for patients with advanced unresectable dedifferentiated liposarcoma. Top-line data from the Phase 3 portion of the SEAL study are anticipated by the end of 2019.
- Ongoing Investigator Sponsored Phase 2/3 Trial as Maintenance Therapy in Endometrial Cancer Underway. A randomized Phase 2/3 study of selinexor versus placebo as maintenance therapy in patients with one or two prior platinum-based treatments for advanced endometrial cancer lead by Dr.
Ignace Vergote, Head of the Department of Obstetrics and Gynaecologyand Gynaecologic Oncology at the Catholic University of Leuven, Belgium, is currently ongoing. In the U.S., endometrial cancer is the most common gynecological cancer with approximately 58,000 cases expected to be diagnosed and an estimated 10,000 women expected to die from this cancer in 20181, revealing a meaningful patient population in need of novel therapies.
- Anand Varadan Appointed Chief Commercial Officer. Karyopharm announced the appointment of
Anand Varadanas Executive Vice President, Chief Commercial Officer. Mr. Varadan brings over 25 years of commercial operations and strategy experience with a proven track record of building and leading commercial and cross-functional teams and successfully launching and marketing new therapeutics at publicly-traded, healthcare-focused companies. Mr. Varadan leads the Company's commercial strategy and operations, including for the launch of selinexor.
- Ian Karp Appointed Vice President, Investor and Public Relations. The Company also appointed
Ian Karpas Vice President, Investor and Public Relations. Mr. Karp brings over 20 years of investor relations, corporate development, and commercial experience and has successfully led global teams in achieving key corporate communications objectives, including in the areas of oncology and orphan diseases. Mr. Karp leads all of the Company’s corporate communications activities, including corporate visibility, financial communications, and media and investor relations.
- Exclusive License Agreement Executed with Antengene to Develop and Commercialize Selinexor, Eltanexor, Verdinexor and KPT-9274 in
Chinaand Other Regions in Asia. The agreement includes the development of selinexor and eltanexor for the diagnosis, treatment and/or prevention of all human oncology indications in Chinaand Macau. The agreement also includes the development and commercialization of KPT-9274 in all human oncology indications and verdinexor in human non-oncology indications in mainland China, Macau, Taiwan, Hong Kong, South Korea, and the ASEAN countries. The transaction carries a total deal value of up to $162 million, plus royalties.
Second Quarter Ended
Cash, cash equivalents and investments as of
For the quarter ended
For the quarter ended
Karyopharm reported a net loss of
Karyopharm expects its operating cash burn, including research and development and general and administrative expenses, for the year ending
Further Information About Potential Accelerated Approval for Selinexor in Multiple Myeloma
Conference Call Information
Karyopharm will host a conference call today,
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, 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 plans for commercialization, 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
Velcade® is a registered trademark of
Revlimid® and Pomalyst® are registered trademarks of
Kyprolis® is a registered trademark of
Darzalex® is a registered trademark of
Vice President, Investor and Public Relations
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Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share amounts)
|Cash and cash equivalents||$||118,966||$||68,997|
|Prepaid expenses and other current assets||3,438||1,754|
|Total current assets||244,559||148,423|
|Property and equipment, net||2,611||2,185|
|Liabilities and stockholders’ equity|
|Other current liabilities||229||133|
|Total current liabilities||36,544||49,467|
|Deferred revenue, net of current portion||4,532||—|
|Deferred rent, net of current portion||2,041||1,363|
|Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding||—||—|
|Common stock, $0.0001 par value; 100,000,000 shares authorized; 60,501,260 and 49,533,150 shares issued and outstanding at June 30, 2018 and December 31, 2017, respectively||6||5|
|Additional paid-in capital||781,180||625,017|
|Accumulated other comprehensive loss||(260||)||(217||)|
|Total stockholders’ equity||213,472||129,464|
|Total liabilities and stockholders’ equity||$||256,589||$||180,294|
Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
|Three Months Ended,
|Six Months Ended
|License and other revenue||$||19,891||$||3||$||29,891||$||71|
|Research and development||44,734||23,120||86,055||47,203|
|General and administrative||9,489||6,635||17,110||12,899|
|Total operating expenses||54,223||29,755||103,165||60,102|
|Loss from operations||(34,332||)||(29,752||)||(73,274||)||(60,031||)|
|Other income (expense):|
|Other income (expense)||7||(29||)||(7||)||(44||)|
|Total other income, net||660||383||1,155||768|
|Loss before income taxes||(33,672||)||(29,369||)||(72,119||)||(59,263||)|
|Income tax benefit (provision)||17||(18||)||5||(41||)|
|Net loss per share—basic and diluted||$||(0.60||)||$||(0.64||)||$||(1.36||)||$||(1.35||)|
|Weighted-average number of common shares outstanding used in net loss per share—basic and diluted||56,089,159||45,831,239||52,862,194||43,873,892|
Source: Karyopharm Therapeutics Inc.