On November 2, 2017 Arbutus Biopharma Corporation (Nasdaq:ABUS), an industry-leading Hepatitis B Virus (HBV) therapeutic solutions company, reported its third quarter 2017 unaudited financial results and provided a corporate update (Press release, Arbutus Biopharma, NOV 2, 2017, View Source [SID1234521510]).

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“We’ve achieved major milestones this quarter to advance our HBV pipeline,” said Dr. Mark J Murray, Arbutus’ President and CEO. “Our novel clinical candidate ARB-1467 is advancing into a combination study with HBV standard of care tenofovir and pegylated interferon to potentially drive durable loss of HBV DNA and HBV s-antigen (HBsAg) in patients. A strategic financing from our largest shareholder, Roivant Sciences Ltd. (Roivant), strengthens our financial position and extends our cash runway to further advance and validate our strategy of curing chronic HBV through a combination regimen.”

Recent Highlights and Developments

LNP licensee Alnylam announced positive Phase III results for LNP-enabled patisiran program, which met its primary efficacy endpoint and all secondary endpoints. Arbutus to receive single digit royalties on sales of patisiran. A New Drug Application (NDA) filing is anticipated in 2017.
Licensing transaction completed with Gritstone Oncology (Gritstone) to access Arbutus’ LNP delivery technology to deliver RNA-based neoantigen immunotherapy products. Gritstone will pay Arbutus an upfront payment, payments for achievement of development, regulatory and commercial milestones, royalties, and reimburse Arbutus for technology development, manufacturing, and regulatory support.
On October 16, 2017, Arbutus closed the issue and sale of 500,000 Series A participating convertible preferred shares to Roivant, plus 8.75% per annum compounded annually (subject to mandatory conversion in 4 years at $7.13 per share – 15% premium at closing), for gross proceeds of $50 million (Tranche 1), representing the first of two tranches of preferred shares comprising the previously announced $116.4 million strategic investment by Roivant in Arbutus. Arbutus is expected to close the remaining amount of $66.4 million (Tranche 2) by the end of 4Q17, subject to customary closing conditions including regulatory and shareholder approvals, as applicable, under Canadian securities law.
Top-line results reported for ARB-1467 Phase II Cohort 4 (bi-weekly dosing): all twelve patients experienced reductions in serum HBsAg (average reduction of 1.4 log10, 64% of evaluable patients met the predefined response criteria of HBsAg ≤1000 IU/mL with ≥1 log10 decline during the first 10 weeks of treatment, and 71% of patients who met the response criteria had their serum HBsAg reduced to low absolute levels (below 50 IU/mL) during the bi-weekly dosing period. Results have informed the design of the combination study with ARB-1467, tenofovir, and pegylated interferon that will start later this year. Initial results for the monthly dosing extension suggest that monthly dosing is not sufficient to maintain or improve upon these reductions in HBsAg levels so Arbutus has discontinued the monthly extension and the next study will utilize bi-weekly dosing.
Top-line results from the healthy volunteer study of AB-423 show that it has been generally well-tolerated with no serious adverse events following single doses up to 800mg. AB-423’s favorable safety and pharmacokinetics (PK) profile following single doses supported further evaluation of multiple-dose administration of AB-423, which is now complete and will be followed by a multi-dosing study in HBV patients, which is expected to start in 1Q18.
2 oral presentations and 5 posters presented at the 2017 AASLD Liver Meeting confirm Arbutus’ LNP siRNA (ARB-1467) drove significant reductions in serum HBsAg levels, and capsid inhibitors (AB-423 and AB-506) and HBV RNA destabilizer (AB-425) have significant potential to contribute to future curative combination treatment regimens.
Dr. Michael Sofia, Arbutus’ Chief Scientific Officer, was awarded the Research & Development Council of New Jersey’s highest award, the 2017 Edison Patent Award Science & Technology Medal.
Phase II Triple Combination Study of ARB-1467

Arbutus will initiate a Phase II triple combination, multi-dose study with ARB-1467, tenofovir, and pegylated interferon to maximize reduction of HBsAg and evaluate the importance of immune stimulation in patients who have achieved low HBV DNA and HBsAg levels. This study will enroll 20 HBeAg- patients who will receive 30-weeks of bi-weekly dosing of ARB-1467 at 0.4 mg/kg and daily tenofovir. Predefined treatment responders at 6-weeks will qualify for the addition of weekly pegylated interferon treatment while continuing to receive bi-weekly doses of ARB-1467 and daily tenofovir for the remaining 24-weeks. The study will conclude with a 24-week post-treatment follow-up period. Interim on-treatment results from this study are expected in the second half of 2018, followed by final results in 2019.

Upcoming Milestones

4Q17: Alnylam expected to file NDA application for patisiran (Arbutus to receive royalties on sales).
4Q17: Initiate 30-week Phase II triple combination study of ARB-1467 tenofovir, and pegylated interferon.
4Q17: Close of Tranche 2, subject to shareholder approval, for remaining amount ($66.4 million) of strategic investment from Roivant.
1Q18: Initiate AB-423 Phase II multi ascending dose (MAD) study in HBV patients.
Mid-2018: AB-506 IND (or equivalent) filing.
Mid-2018: AB-452 IND (or equivalent) filing.
Mid-2018: Results from AB-423 multi-dosing study in HBV patients.
2H18: Interim on-treatment results from triple combination study of ARB-1467, tenofovir, and pegylated interferon.
2018: Expected FDA approval decision for Alnylam’s patisiran.
Financial Results

Cash, Cash Equivalents and Investments

As at September 30, 2017, Arbutus had cash, cash equivalents, short-term investments and restricted investments totaling $100.8 million, as compared to $143.2 million at December 31, 2016.

On October 16, 2017, the Company closed Tranche 1 for the issue and sale of 500,000 Preferred Shares to Roivant for gross proceeds of $50 million. Arbutus is expected to close Tranche 2, subject to shareholder approval, for the remaining amount of $66.4 million by 4Q17 for total gross proceeds of $116.4 million, subject to customary closing conditions including regulatory and shareholder approvals, as applicable, under Canadian securities law. For further details with respect to the Preferred Shares, please refer to Arbutus’ Form 8-K filed with the U.S. Securities and Exchange Commission on October 3, 2017 or Arbutus’ material change report filed with the Canadian securities regulatory authorities on SEDAR on October 5, 2017.

Net Loss

For Q3 2017, the net loss was $11.6 million ($0.21 basic and diluted loss per common share) as compared to a net loss of $19.6 million ($0.37 basic and diluted loss per common share) for Q3 2016. The net loss for the nine-months ended September 30, 2017 was $48.5 million ($0.89 basic and diluted loss per common share) as compared to a net loss of $165.5 million ($3.15 basic and diluted loss per common share) for the nine-months ended September 30, 2016.

Non-GAAP Net Loss

The non-GAAP net loss for Q3 2017 was $9.6 million ($0.17 loss per common share) as compared to a non-GAAP net loss of $16.6 million ($0.31 per common share) for Q3 2016. The non-GAAP net loss for the nine-months ended September 30, 2017 was $40.5 million ($0.74 loss per common share) as compared to a non-GAAP net loss of $45.0 million ($0.86 loss per common share) for the nine-months ended September 30, 2016. The non-GAAP net loss for Q3 2017 has been adjusted to exclude non-cash compensation expense in connection with certain share repurchase provisions arising from the merger with Arbutus Inc. in March 2015.

Revenue

Revenue was $6.9 million in Q3 2017 as compared to $0.8 million in Q3 2016.

In March 2017, Arbutus signed a License Agreement with Alexion that granted them exclusive use of the Company’s proprietary lipid nanoparticle (LNP) technology in one of Alexion’s rare disease programs. Licensing fee revenue recognized in Q3 2017 relates to the non-refundable upfront payment of $7.5 million for the use of Arbutus’ technology. In addition, Arbutus recognized revenue for services provided to Alexion related to technology development, manufacturing and regulatory support for the advancement of Alexion’s mRNA product candidate. In July 2017, the Company received notice of termination from Alexion for the LNP license agreement. Therefore, Arbutus recorded the remaining deferred revenue for the non-refundable upfront payment as well as any revenue for any work done related to closeout procedures in Q3 2017.

Revenue in Q3 2016 related primarily to the Dicerna license and collaboration that was terminated in November 2016.

In addition, Arbutus has ongoing license agreements with Alnylam and Spectrum, under which Arbutus is eligible to receive commercial royalties.

Research, Development, Collaborations and Contracts Expenses

Research, development, collaborations and contracts expenses were $15.5 million in Q3 2017 as compared to $15.7 million in Q3 2016.

R&D expenses remained consistent in Q3 2017 and Q3 2016. The Company’s R&D expenses predominantly relate to its HBV programs during both periods. Arbutus initiated a Phase I clinical trial for AB-423 in Q1 2017 and continues to incur costs related to the Company’s clinical trials for ARB-1467 as well as costs for IND enabling studies for the Company’s recent candidate nominations – a second capsid inhibitor (AB-506) and an HBV RNA destabilizer (AB-452), as well as costs related to research and preclinical studies for the Company’s other HBV programs.

General and Administrative

General and administrative expenses were $3.7 million in Q3 2017, consistent with $3.7 million in Q3 2016.

Outstanding Shares

The Company had 55.0 million common shares issued and outstanding and 60.4 million shares on a fully diluted basis as at September 30, 2017. Subsequent to September 30, 2017, Roivant Sciences Ltd. completed its initial purchase of 500,000 convertible preferred shares, which will be mandatorily convertible into 7,037,839 common shares on October 16, 2021. Assuming that the convertible preferred shares were converted on October 16, 2017 (the closing date of the issuance of the convertible preferred shares), the Company would have had 62,089,834 common shares outstanding at October 16, 2017.

Other Income (Losses)

The Company continues to incur substantial expenses and hold a portion of its cash and investment balances in Canadian dollars, and as such, will remain subject to risks associated with foreign currency fluctuations. During Q3 2017, Arbutus recorded a foreign exchange gain of $1.2 million, which is primarily an unrealized gain related to an appreciation in the value of the Company’s Canadian dollar funds from the previous period, when converted to the Company’s functional currency of U.S. dollars.

Contingent consideration is a liability assumed by the Company from acquiring Arbutus Inc. in March 2015. In Q3 2017 Arbutus recorded and increase in the fair value of contingent consideration of $0.2 million. In general, increases in the fair value of the contingent consideration are related to the progress of the Company’s programs as they get closer to triggering contingent payments.

On November 2, 2017 MorphoSys reported Presentation of Clinical Data on Proprietary Blood Cancer Compound MOR208 at Upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2017 (Press release, MorphoSys, NOV 2, 2017, View Source [SID1234521453]).

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– Abstract on preliminary data from ongoing phase 2 trial with antibody MOR208 in combination with lenalidomide in relapsed/refractory DLBCL accepted for poster presentation at ASH (Free ASH Whitepaper) 2017

– Poster presentation will take place on December 11, 2017, 6:00-8:00pm EST (December 12, 0:00-2:00am CET)

– Conference call by MorphoSys scheduled for December 12, 2017, 11:00am EST (5:00pm CET)

MorphoSys AG (FSE: MOR; Prime Standard Segment; TecDAX, OTC: MPSYY) reported the upcoming presentation of data on the Company’s proprietary investigational hemato-oncological program MOR208 at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 9-12, 2017 in Atlanta, Georgia/USA.

“We are pleased that updated clinical trial results of the phase 2 L-MIND study with our antibody MOR208 in combination with lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma (R/R DLBCL) who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation will be shown at the upcoming ASH (Free ASH Whitepaper) conference in a poster presentation,” commented Dr. Malte Peters, Chief Development Officer of MorphoSys AG. “We see a particularly high unmet medical need for these blood cancer patients and look forward to presenting an update from our ongoing study in this patient group.”

Based on preliminary data from the L-MIND study presented in June 2017, the FDA had recently granted Breakthrough Therapy designation for MOR208, in combination with lenalidomide, for the treatment of patients with R/R DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation.

Details about the abstract from MorphoSys’s proprietary program MOR208 accepted for presentation at ASH (Free ASH Whitepaper) 2017:

Single-Arm Phase II Study of MOR208 Combined with Lenalidomide in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma: L-Mind

The poster presentation will include updated clinical results, in particular with respect to safety and efficacy, from our phase 2 study L-MIND with MOR208 in combination with lenalidomide in adult patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation.

Abstract #4123; Poster III

The poster will be presented during the Session #626 “Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)-Results from Prospective Clinical Trials” on Monday, December 11, 2017, 6:00pm-8:00pm EST (Dec. 12, 2017, 0:00am-2:00am CET), in the Georgia World Congress Center, Bldg A, Lvl 1, Hall A2.

In addition to the presentation, the abstract will be published online in the December 8, 2017 supplemental volume of Blood. Additional information can be found at www.hematology.org, including the abstract.

MorphoSys will hold an investor & analyst conference call after the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2017 on December 12, 2017, 11:00am EST(5:00pm CET).

Dial-in details will be made available in time.

The presentation, a live webcast and a replay of the webcast will be made available at View Source

Arbutus Biopharma has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Arbutus Biopharma, 2017, NOV 2, 2017, View Source [SID1234521500]).

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On November 2, 2017 Progenics Pharmaceuticals, Inc. (Nasdaq: PGNX) reported financial results and provided a business update for the third quarter of 2017 (Press release, Progenics Pharmaceuticals, NOV 2, 2017, View Source [SID1234521488]).

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“The recent completion of our NDA submission for AZEDRA represents a major achievement for our growing company,” said Mark Baker, Chief Executive Officer of Progenics. “AZEDRA offers a potentially transformative therapy for metastatic pheochromocytoma and paraganglioma patients who have no treatment options in the U.S. for this devastating condition. We are continuing to build the necessary team and infrastructure to support a rapid commercial launch of this important therapy following a potential approval.”

Third Quarter and Recent Key Business Highlights

AZEDRA, Ultra-Orphan Radiotherapeutic Candidate

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New Drug Application (NDA) for AZEDRA (iobenguane I 131) Submitted to the U.S. Food and Drug Administration (FDA) on October 31st
As announced earlier today, Progenics has submitted the NDA for AZEDRA in patients with malignant, recurrent, and/or unresectable pheochromocytoma and paraganglioma, rare neuroendocrine tumors for which there are currently no approved treatment options in the United States. The NDA remains subject to FDA regulatory review and approval. AZEDRA holds Breakthrough Therapy and Orphan Drug statuses, as well as Fast Track designation.

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Multiple Presentations of Additional Data from Phase 2b Trial of AZEDRA
Progenics has recently presented the positive results from its pivotal Phase 2b study evaluating AZEDRA at the 5th International Symposium on Pheochromocytoma and Paraganglioma (ISP), the North American Neuroendocrine Tumor Society (NANETS) 2017 Annual Symposium, and the 30th Annual Congress of the European Association of Nuclear Medicine (EANM). The Phase 2b trial met the primary endpoint evaluating the proportion of metastatic pheochromocytoma and paraganglioma patients who achieved a 50% or greater reduction of all antihypertensive medications for at least six months. The results further demonstrated that sustained reduction of antihypertensive medications was positively correlated with favorable tumor responses, including radiographic tumor responses, tumor biomarker response and overall survival. AZEDRA was shown to be safe and generally well tolerated.



Progenics Announces Third Quarter 2017 Financial Results
Page 2

PSMA-Targeted Prostate Cancer Pipeline

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Progress Continues for Clinical Studies of 1404, PyL and 1095
Progenics continues to enroll patients in: the Phase 3 trial of 1404, a SPECT/CT imaging agent; the Phase 2/3 study of PyL, a PET/CT imaging agent; and the Phase 1 open-label dose escalation study of 1095, a radiotherapeutic that selectively binds to PSMA.

RELISTOR, Treatment for Opioid-Induced Constipation (partnered with Valeant Pharmaceuticals International, Inc.)

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Third Quarter 2017 RELISTOR Net Sales of $17.1 Million
The third quarter 2017 sales, as reported to Progenics by its partner Valeant, translated to $2.6 million in royalty revenue for Progenics for the quarter. Oral RELISTOR prescriptions increased 40% over the preceding quarter.

Third Quarter 2017 Financial Results

Third quarter revenue totaled $2.7 million, down from $53.9 million in the third quarter of 2016, reflecting RELISTOR royalty income of $2.6 million compared to $3.3 million in the corresponding period of 2016. The prior year period included milestone revenue of $50.0 million for the approval of RELISTOR oral tablets as well as a non-recurring favorable sales return adjustment included in Valeant’s reported net sales of RELISTOR.

Third quarter research and development expenses increased by $0.5 million compared to the corresponding prior year period, resulting primarily from higher clinical trial costs for PyL, partially offset by lower manufacturing scale-up costs for 1095. Third quarter general and administrative expenses decreased by $1.3 million compared to the corresponding prior year period, which included an accrual for litigation with a former employee. Partially offsetting this decrease were higher costs associated with building commercial capabilities in preparation for a potential AZEDRA approval and launch. For the three months ended September 30, 2017, Progenics recognized interest expense of $1.0 million related to the RELISTOR royalty-backed loan.

Net loss attributable to Progenics for the third quarter was $15.4 million, or $0.22 per diluted share, compared to net income of $36.3 million, or $0.52 per diluted share, in the corresponding 2016 period. Progenics ended the third quarter with cash and cash equivalents of $98.3 million, a decrease of $40.6 million compared to cash and cash equivalents as of December 31, 2016.

Conference Call and Webcast

Progenics will review third quarter financial results in a conference call today at 8:30 a.m. ET. To participate, please dial (877) 250-8889 (domestic) or (720) 545-0001 (international) and reference conference ID 5589567. A live webcast will be available in the Media Center of the Progenics website, www.progenics.com, and a replay will be available for two weeks.

On November 2, 2017 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the third quarter 2017 and provided an overview of recent accomplishments and clinical development plans for its lead, novel, oral SINE compound selinexor (KPT-330), and other pipeline assets including KPT-8602, its second-generation oral SINE compound, KPT-9274, its oral, dual inhibitor of p21-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase (NAMPT) (Press release, Karyopharm, NOV 2, 2017, View Source [SID1234521482]).

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“We are very proud of our accomplishments to date in 2017, especially the recent execution of an exclusive licensing transaction, valued at up to $193 million, plus royalties, with Ono Pharmaceutical Co. Ltd. for the development and commercialization of selinexor and KPT-8602 for all human oncology indications in Japan and certain other countries in Asia,” said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. “As we head into year end, we look forward to reporting clinical data from multiple treatment arms of the Phase 1b/2 STOMP study evaluating oral selinexor in combination with several current ‘backbone’ therapies for multiple myeloma (MM) at this year’s American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Fourteen abstracts highlighting data for selinexor and our other pipeline assets have been selected for presentation at ASH (Free ASH Whitepaper), and we are pleased to be able to share these data with the medical community at the meeting this year. We continue to execute on the Phase 2b STORM study on schedule, and we expect to report top-line results by April 2018.”

Third Quarter 2017 and Recent Events, Highlights and Milestones:

Partnerships and Other Key Corporate Developments

Signed Exclusive License Agreement with Ono Pharmaceutical Co., Ltd (Ono) to Develop and Commercialize Selinexor and KPT-8602 in Japan and Other Countries in Asia. In October 2017, Karyopharm announced its entry into an exclusive license agreement with Ono for the development and commercialization of selinexor and KPT-8602, the Company’s second-generation oral SINE compound. The agreement includes the development of selinexor and KPT-8602 for the diagnosis, treatment and/or prevention of all human oncology indications in Japan, South Korea, Taiwan, Hong Kong, and the ASEAN countries (the Territory). The transaction, which carries a total deal value of up to $193.0 million based on the exchange rate on the effective date of the license agreement, includes a one-time upfront payment of ¥2.5 billion ($22.3 million) from Ono to Karyopharm and up to an additional ¥19.15 billion ($170.7 million) if certain specified future development and commercial milestones are achieved by Ono. In addition, Karyopharm is also eligible to receive low double-digit royalties based on future net sales of selinexor and KPT-8602 in the Territory. In exchange, Ono received exclusive rights to develop and commercialize both compounds in the Territory, at its own cost and expense. Ono will also have the ability to participate in any global clinical study of selinexor and KPT-8602, and will bear the cost and expense for patients enrolled in clinical studies in the Territory. Karyopharm retains all rights to selinexor and KPT-8602 outside the Territory.

Michael Falvey Appointed Chief Financial Officer. In September 2017, Karyopharm announced the appointment of Michael Falvey as Executive Vice President, Chief Financial Officer and Treasurer. Mr. Falvey brings 35 years of experience in executing business growth and financial strategies for publicly-traded and privately-held companies, including senior financial leadership roles at healthcare-focused, scientific organizations. Mr. Falvey leads the Company’s financial and capital markets strategy, as well as advises on business development and transactional activities.

Other Key Personnel Appointments. In September 2017, Karyopharm also announced the appointment of Jatin Shah, MD, as Vice President, Clinical Strategy and Joan Wood as Chief Human Resources Officer. Dr. Shah brings significant medical oncology experience, including treating patients with MM and clinical research. Dr. Shah formerly served as Associate Professor and Director of the Myeloma Clinical/Translational Research Department at MD Anderson Cancer Center. Ms. Wood is an experienced human resources executive with significant experience in global talent management in the biopharma industry. Prior to joining Karyopharm, she served in senior leadership roles at Sarepta Therapeutics and Genzyme Corporation.
Selinexor in Multiple Myeloma

Phase 1b/2 STOMP Data Selected for Presentation at ASH (Free ASH Whitepaper) 2017. Four abstracts featuring clinical data from the four treatment arms of the ongoing Phase 1b/2 STOMP study have been selected for poster presentations at the upcoming ASH (Free ASH Whitepaper) 2017 annual meeting in early December (ASH 2017). The poster presentations will include updated data from the arm evaluating selinexor in combination with Velcade (bortezomib) and low-dose dexamethasone (SVd); updated data from the arm evaluating selinexor in combination with Pomalyst (pomalidomide) and low-dose dexamethasone (SPd); updated data from the arm evaluating selinexor in combination with Revlimid (lenalidomide) and low-dose dexamethasone (SRd); and preliminary data from the arm evaluating selinexor in combination with Darzalex (daratumumab) and low-dose dexamethasone (SDd).

Several Investigator-sponsored Trial and Preclinical Abstracts Selected for Presentation at ASH (Free ASH Whitepaper) 2017. Four abstracts featuring clinical data from investigator-sponsored clinical studies evaluating selinexor either as a single-agent or in combination with other anti-cancer agents for the treatment of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and MM have been selected for oral or poster presentations at ASH (Free ASH Whitepaper) 2017. In addition, three abstracts describing preclinical research exploring the use of selinexor in models of AML, lymphoma, mantle cell lymphoma (MCL) and MM have also been selected for oral or poster presentations at the meeting.

Pivotal Phase 3 BOSTON Study Underway. Karyopharm’s pivotal, randomized Phase 3 BOSTON (Bortezomib, Selinexor and dexamethasone) study is now underway. BOSTON is designed to evaluate 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 progression-free survival (PFS) and overall response rate (ORR). Both the trial design and endpoints have been agreed to by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency as acceptable for approval. The Company expects to enroll approximately 360 patients at over 100 clinical sites internationally and is projecting completion of enrollment in 2018, with top-line data anticipated in 2019.

Ongoing Phase 2b STORM Study Expansion in Patients with Penta-refractory MM. The Phase 2b STORM study, which was previously 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 oral selinexor in this heavily pretreated MM patient population.
Selinexor in Diffuse Large B-Cell Lymphoma

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 diffuse large B-cell lymphoma (DLBCL). The Company recently reported positive updated Phase 2b SADAL study results (n=63) where selinexor demonstrated an ORR of 33% for the overall study population and 35% in patients with “double-” or “triple-hit” DLBCL, indicating anti-cancer activity in this patient population who usually have a particularly poor prognosis. Median duration of response (DOR) for the overall study population was greater than 7 months. Side effects were consistent with those previously reported with selinexor, with no new safety signals identified. 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 with two or more lines of prior therapy. Karyopharm plans to report top-line results in the second half of 2018 and, assuming a positive outcome, the Company intends to use the data from the SADAL study to support a request for accelerated approval for oral selinexor in this relapsed/refractory DLBCL patient population.
Selinexor in Solid Tumors

Phase 3 Portion of the Phase 2/3 SEAL Study in Liposarcoma Underway Following Successful Outcome from Phase 2 Portion. Karyopharm recently reported a successful outcome from the 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 SEAL study’s primary endpoint of PFS, oral selinexor showed superiority over placebo, achieving a hazard ratio (HR) of 0.60, representing a 40% reduction in the risk of progression or death, as assessed by Independent Central Radiological Review (ICRR) (RECIST v1.1). Oral selinexor demonstrated an expected and manageable safety profile with no new or unexpected safety signals identified. The majority of treatment-related adverse events were low grade and reversible with dose modifications and/or standard supportive care. Importantly, the incidence of infections in the selinexor arm was less than that reported in the placebo arm.

The Phase 3 portion of the SEAL study, which was originally initiated in North America, is ongoing and has been expanded to include Europe. In this blinded, placebo-controlled Phase 3 study, up to 222 patients are expected to be enrolled and randomized 2:1 to receive either oral selinexor, (60mg twice weekly) until disease progression or intolerability, or placebo. Patients whose disease progresses on placebo will be permitted to cross over to the selinexor arm. The primary endpoint of the Phase 3 portion of the study is PFS (RECIST v1.1) as assessed by the ICRR. The Phase 3 study design and primary endpoint of PFS were agreed to by the FDA. Top-line data from the Phase 3 portion of the SEAL study are anticipated by the end of 2019. Assuming a positive outcome, these data are expected to support a New Drug Application for oral selinexor as a potential new treatment for patients with advanced unresectable dedifferentiated liposarcoma.
Poster Presentation Featuring Selinexor Phase 1 Safety and Tolerability Data in Ovarian and Endometrial Cancers at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Annual Meeting (ESMO 2017). Top-line Phase 1 data from this ongoing investigator-sponsored study evaluating selinexor in combination with paclitaxel and carboplatin, was presented by Dr. Vikky Makker, Memorial Sloan Kettering Cancer Center, and showed encouraging early efficacy and a manageable safety profile in patients with advanced ovarian and endometrial cancers. A recommended Phase 2 dose (RP2D) regimen for selinexor was established (60mg once weekly in combination with carboplatin AUC5 on Day 1 and paclitaxel 80 mg/m2 on Days 1, 8 and 15 of each 21-day cycle) and expansion cohorts for the RP2D regimen are planned.
KPT-8602

Two Abstracts Highlighting KPT-8602 Preclinical Research Selected for Presentation at ASH (Free ASH Whitepaper) 2017. Two abstracts describing preclinical research exploring the use of KPT-8602 in models of MM and myelofibrosis have been selected for poster presentations at the meeting.
KPT-9274

Preclinical Research for KPT-9274 Selected for Oral Presentation at ASH (Free ASH Whitepaper) 2017. An abstract describing preclinical research exploring the use of KPT-9274 in a model of Waldenstrom macroglobulinemia has been selected for an oral presentation at the meeting.

Poster Presentation Featuring KPT-9274 Phase 1 Safety and Tolerability Data in Advanced Solid Malignancies or Relapsed NHL at ESMO (Free ESMO Whitepaper) 2017. Top-line Phase 1 data from this ongoing study evaluating KPT-9274 in patients with advanced solid malignancies (including sarcoma, colon and lung cancer) or relapsed non-Hodgkin’s lymphoma (NHL) following standard therapy(s), was presented by Dr. Aung Naing, MD Anderson Cancer Center, and showed a manageable safety profile and early signals of anti-tumor activity. The poster also reported findings that niacin can be safely administered with KPT-9274 and may improve tolerability, particularly with respect to anemia. These study findings indicate that in patients whose disease has progressed despite most available therapies, KPT-9274 can induce tumor shrinkage and disease stabilization. Dose escalation remains ongoing.
Third Quarter 2017 Financial Results

Cash, cash equivalents and investments as of September 30, 2017, including restricted cash, totaled $159.4 million, compared to $175.5 million as of December 31, 2016.

For the quarter ended September 30, 2017, research and development expense was $25.2 million compared to $19.9 million for the quarter ended September 30, 2016. For the quarter ended September 30, 2017, general and administrative expense was $5.8 million compared to $5.9 million for the quarter ended September 30, 2016.

Karyopharm reported a net loss of $30.6 million, or $0.65 per share, for the quarter ended September 30, 2017, compared to a net loss of $25.4 million, or $0.69 per share, for the quarter ended September 30, 2016. Net loss includes stock-based compensation expense of $4.9 million and $5.6 million for the quarters ended September 30, 2017 and September 30, 2016, respectively.

Financial Outlook

Karyopharm expects its operating cash burn, including research and development and general and administrative expenses, for the year ending December 31, 2017 to be approximately $95 million. Based on current operating plans, Karyopharm expects that its existing cash and cash equivalents will be sufficient to fund its research and development programs and operations into 2019, including the continued clinical development of selinexor in the Company’s lead indications with a focus on filing a new drug application with the FDA requesting accelerated approval in MM during 2018, assuming positive data from the STORM study, and preparing the commercial infrastructure for the potential launch of selinexor in North America and Western Europe. Additional key activities for 2018 include topline data from the SADAL study targeted for the second half of 2018 and continued enrollment in the Phase 3 BOSTON and SEAL studies.

Conference Call Information

Karyopharm will host a conference call today, Thursday, November 2, 2017, at 8:30 a.m. Eastern Time, to discuss the third quarter 2017 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (855) 437-4406 or (484) 756-4292 (international) at least five minutes prior to the start time and refer to conference ID: 98527624. An audio recording of the call will be available under “Events & Presentations” in the “Investor” section of Karyopharm’s website, View Source, approximately two hours after the event.