"Our second quarter achievements marked significant progress across several of our development programs, and especially for selinexor," said
Second Quarter 2017 and Recent Events, Highlights and Milestones:
Selinexor in Multiple Myeloma
- Pivotal Phase 3 BOSTON Study Initiated. Karyopharm initiated the pivotal, randomized Phase 3 BOSTON (Bortezomib, Selinexor and dexamethasone) study, evaluating once weekly selinexor 100mg in combination with the proteasome inhibitor Velcade (bortezomib, once weekly) and dexamethasone (SVd), compared to standard dose Velcade (twice weekly) and low-dose dexamethasone (Vd) in patients with MM who have had one to three prior lines of therapy. The primary endpoints of the study are PFS and ORR. The
BOSTONstudy is expected to enroll approximately 360 patients at over 100 clinical sites internationally. Karyopharm is projecting to complete enrollment in 2018, with top-line data anticipated in 2019.
- Selinexor Named Among the "Top 5 Oncology R&D Products Worldwide in 2022" by EvaluatePharma®. In EvaluatePharma's recent report, World Preview 2017, Outlook to 2022, selinexor was projected to be one of the top five selling oncology research and development products worldwide in 2022, with the potential to generate estimated revenues of
$920 millionin worldwide annual sales and capture 0.5% of the worldwide oncology market share in the same timeframe. This analysis is based on EvaluatePharma's coverage of the world's 6,500 leading pharmaceutical and biotech companies and highlights certain important industry trends by therapy area.
- Ongoing Phase 2b STORM Study Expansion in Patients with Penta-refractory MM. The Phase 2b STORM study, which was recently expanded to include 122 additional patients with penta-refractory MM, continues to enroll on track. Karyopharm expects to report top-line data from the expanded cohort by
April 2018, and, assuming a positive outcome, intends to use the data from the expanded STORM study to support a request for accelerated approval for selinexor in heavily pretreated MM.
- Ongoing Phase 1b/2 STOMP Study Evaluating Selinexor in Combination with Several Key MM Drugs. Enrollment is complete in the Phase 1b/2 STOMP arm evaluating selinexor in combination with Velcade and low-dose dexamethasone (SVd) in heavily pretreated patients with MM. The SVd arm of the STOMP study enrolled 42 patients. Dose escalation is complete and expansion is ongoing in the arms evaluating oral selinexor plus immunomodulatory drug (IMID) combinations, including selinexor + Revlimid® (lenalidomide) + dexamethasone (SRd), and selinexor + Pomalyst® (pomalidomide) and dexamethasone (SPd). The Company expects to report updated data on these convenient, all oral regimens by year end 2017.
- New Study Arm Initiated in Phase 1b/2 STOMP Study Evaluating Selinexor in Combination with Darzalex® (daratumumab). Karyopharm has dosed patients in a new Phase 1b/2 STOMP study arm designed to evaluate selinexor in combination with the anti-CD38 monoclonal antibody Darzalex and low-dose dexamethasone (SDd) in heavily pretreated patients with MM. The SDd arm of the STOMP study is expected to enroll up to 16 patients and the Company expects to report top-line data in the first half of 2018.
Selinexor in Diffuse Large B-Cell Lymphoma
- Updated Data from Phase 2b SADAL Study in DLBCL Presented at EHA 2017. At the 2017 EHA Annual Meeting in June, an oral presentation was given that highlighted updated data from the ongoing Phase 2b SADAL study evaluating single-agent selinexor in patients with relapsed or refractory DLBCL. This latest data demonstrated that selinexor achieved an ORR of 33.3% and a duration of response (DOR) of > 7 months in the first 63 patients, as adjudicated by an independent central radiological committee. Patients were randomized to one of two single-agent selinexor arms, a higher dose arm of 100 mg twice weekly and a lower dose arm of 60 mg twice weekly. The median overall survival was 8 months for all patients, consistent with published data in this population which has a very poor prognosis. As of the data cutoff date, the median survival for the responders had not been reached and was over 9 months. Most responses occurred at the first response evaluation (~2 months). As of the data cutoff date, 9 of the 21 responding patients remained on treatment, including 6 patients who had a complete response (CR). Selinexor also showed robust, single-agent activity against GCB and non-GCB subtypes of DLBCL. Of the 32 patients with DLBCL of the GCB-subtype, 9 responded (4 patients with a CR, 5 patients with a partial response (PR)) for an ORR of 28.1%. Of the 31 patients with DLBCL of the non-GCB (or
ABC)-subtype, 12 responded (5 patients with a CR, 7 patients with a PR) for an ORR of 38.7%. Amongst the 14 patients with "double-" or "triple-hit" DLBCL, the ORR was consistent with the ORR across the SADAL patient population, indicating anti-cancer activity in this population, which usually has a particularly poor prognosis. Side effects were consistent with those previously reported with selinexor, and no new safety signals were identified. Importantly, side effects were reduced in the 60mg cohort in comparison with the 100mg cohort.
In consultation with the
U.S. Food and Drug Administration(FDA), Karyopharm amended the SADAL study, removing the 100mg arm and continuing enrollment only in the 60mg twice weekly arm. The FDAhas agreed that the single-arm trial design appears appropriate for accelerated approval in DLBCL, though eligibility for accelerated approval will depend on the complete trial results and available therapies at the time of regulatory action. The SADAL study is expected to enroll up to a total of 130 patients in the 60mg single-arm cohort and Karyopharm plans to report top-line results in the second half of 2018.
Selinexor in Other Hematologic Malignancies
Published Phase 1 Data Demonstrating Selinexor's Activity in Patients with Relapsed/Refractory Non-Hodgkin's Lymphoma (NHL) in the Journal Blood. A paper describing results from the first in human Phase 1 clinical study assessing safety and preliminary activity of selinexor in patients with relapsed or refractory NHL was recently published in the journal Blood. In the paper, authored by
Selinexor in Solid Tumors
- Ongoing Phase 2/3 SEAL Study in Liposarcoma. Enrollment is now complete in the Phase 2 portion of the blinded, randomized Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with advanced liposarcoma. Karyopharm expects to report the hazard ratio for PFS from the Phase 2 portion of the SEAL study and providing an update regarding the planned development path in this indication during September or
October 2017. The primary endpoint of the SEAL study is PFS and both the trial design and endpoints have been accepted by the FDAand the European Medicines Agency.
- Oral Presentation Highlighting Efficacy, Safety and Intratumoral Pharmacokinetic Data for Selinexor in Glioblastoma at the 2017
World Federation of Neuro-Oncology Societies (WFNOS) Meeting. Clinical data from a Phase 2 study evaluating selinexor in patients with recurrent glioblastoma was highlighted in an oral presentation at the 2017 WFNOS meeting by Andrew Lassman, MD, Columbia University Medical Center. The data demonstrated that oral selinexor achieved responses and sufficient intratumoral penetration, with a manageable tolerability profile when accompanied by standard supportive care. Importantly, disease control rates using selinexor dosed at 80 mg once weekly were as high or higher than those observed with more intensive dosing, and tolerability was improved.
- Signed Global License Agreement with Anivive Lifesciences for Verdinexor for Animal Health Applications. Karyopharm and Anivive, a privately-held biotech company, executed a licensing agreement under which Anivive licensed from Karyopharm exclusive worldwide rights to research, develop and commercialize verdinexor for the treatment of cancer in companion animals. Under the terms of the agreement, Anivive made a one-time upfront payment of
$1 millionto Karyopharm. Anivive also agreed to pay up to an additional $43.5 millionbased on technology transfer and achievement of specified regulatory, clinical and commercial milestones, assuming approval in both the U.S.and the European Union. In addition, Anivive agreed to pay Karyopharm a low double-digit royalty based on future net sales of verdinexor.
- Preclinical Efficacy Highlighting KPT-9274's Anti-Cancer Activity in Dogs with Spontaneous Lymphomas Presented as a Late-Breaking Poster at the
American Association of Cancer Research(AACR) 2017 Annual Meeting. At the AACR 2017 Annual Meeting in April, Karyopharm collaborator Cheryl Londonof Tufts Universitypresented a late-breaking poster highlighting preclinical data demonstrating the activity and synergy of KPT-9274, the Company's oral dual inhibitor of PAK4/NAMPT, with doxorubicin to treat dogs with lymphoma. KPT-9274 is currently being evaluated in a Phase 1 safety and tolerability study in patients with advanced solid malignancies (including sarcoma, colon and lung cancer) or non-Hodgkin's lymphoma (NHL) whose disease has relapsed after standard therapy(s). Top-line data from this clinical study are expected later this year.
Other Corporate and Clinical Developments
$52.3 Millionin Equity Financings. In April 2017, the Company sold approximately 3.9 million shares of common stock in an underwritten public offering at a price to the public of $10.25per share, resulting in net proceeds to the Company of approximately $37.9 millionafter deducting underwriting discounts and commissions and other offering expenses, and sold approximately 1.3 million shares under its ATM offering facility for net proceeds of approximately $14.4 million.
Second Quarter 2017 Financial Results
Cash, cash equivalents and investments as of
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
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 therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, including the timing of initiation and enrollment of certain trials and of the reporting of data from such trials, 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 any of Karyopharm's SINE™ compounds, including selinexor (KPT-330) 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 Takeda Pharmaceutical Company Limited
Revlimid® and Pomalyst® are registered trademarks of Celgene Corporation
Darzalex® is a registered trademark of
EvaluatePharma® World Preview 2017, Outlook to 2022 is copyrighted by Evaluate Ltd.
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|(in thousands, except share and per share amounts)|
|Cash and cash equivalents||$||55,381||$||49,663|
|Prepaid expenses and other current assets||2,070||2,084|
|Total current assets||145,724||131,636|
|Property and equipment, net||2,473||2,836|
|Liabilities and stockholders' equity|
|Other current liabilities||80||83|
|Total current liabilities||17,520||16,476|
|Deferred rent, net of current portion||1,516||1,666|
|Additional paid-in capital||592,534||528,617|
|Accumulated other comprehensive loss||(165||)||(274||)|
|Total stockholders' equity||166,714||162,243|
|Total liabilities and stockholders' equity||$||185,750||$||180,385|
|Karyopharm Therapeutics Inc.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|Three Months Ended,
||Six Months Ended
|Contract and grant revenue||$||3||$||59||$||71||$||59|
|Research and development||23,120||24,579||47,203||46,374|
|General and administrative||6,635||5,956||12,899||11,510|
|Total operating expenses||29,755||30,535||60,102||57,884|
|Loss from operations||(29,752||)||(30,476||)||(60,031||)||(57,825||)|
|Other income (expense):|
|Total other income, net||383||318||768||608|
|Loss before income taxes||(29,369||)||(30,158||)||(59,263||)||(57,217||)|
|Provision for income taxes||(18||)||—||(41||)||—|
|Net loss per share—basic and diluted||$||(0.64||)||$||(0.84||)||$||(1.35||)||$||(1.59||)|
|Weighted-average number of common shares outstanding used in net loss per share—basic and diluted||45,831,239||35,956,470||43,873,892||35,917,486|
Michelle Carroll(212) 600-1902 firstname.lastname@example.org Media: Eliza Schleifstein(917) 763-8106 email@example.com
Source: Karyopharm Therapeutics
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