On November 4, 2019 Acorda Therapeutics, Inc. (NASDAQ: ACOR) provided a financial update for the quarter ended September 30, 2019 (Press release, Acorda Therapeutics, NOV 4, 2019, View Source [SID1234550224]).

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"Our focus has been on ensuring physician awareness and clearing a pathway for access to Inbrija. We have been receiving encouraging feedback on Inbrija from physicians suggesting it should become standard of care for the treatment of Parkinson’s. This is consistent with market research from healthcare professionals and people living with Parkinson’s indicating that Inbrija will become a significant product," said Ron Cohen, M.D., Acorda’s President and CEO. "Acorda’s recent restructuring, albeit difficult, substantially reduced our expense structure, so that we may more effectively focus our resources on the launch of Inbrija and the restructuring of our convertible debt."

Third Quarter 2019 Financial Results

For the quarter ended September 30, 2019, the Company reported INBRIJA net revenue of $4.9 million. INBRIJA became commercially available on February 28, 2019.

For the quarter ended September 30, 2019, the Company reported AMPYRA net revenue of $37.6 million compared to $137.8 million for the same quarter in 2018. In September 2018, AMPYRA lost its exclusivity and generics entered the market. Consequently, the Company expects AMPYRA revenue to continue to decline.

Research and development (R&D) expenses for the quarter ended September 30, 2019 were $16.1 million, including $0.7 million of share-based compensation compared to $22.9 million, including $1.1 million of share-based compensation for the same quarter in 2018.

Sales, general and administrative (SG&A) expenses for the quarter ended September 30, 2019 were $48.7 million, including $2.4 million of share-based compensation compared to $43.6 million, including $4.0 million of share-based compensation for the same quarter in 2018.

The Company reviewed its goodwill for the quarter ended September 30, 2019 as part of its normal reporting process. The Company determined that a triggering event occurred due to a decline in the trading price of the Company’s common stock at and around the end of the quarter ended September 30, 2019. Based on the analysis performed, the Company determined that the goodwill was fully impaired and recorded a non-cash impairment charge of $277.6 million.

Provision for income taxes for the quarter ended September 30, 2019 was $0.02 million compared to a provision for income taxes of $38.0 million for the same quarter in 2018.

The Company reported a GAAP net loss of $263.5 million for the quarter ended September 30, 2019, or $5.55 per diluted share. GAAP net loss in the same quarter of 2018 was $13.9 million, or $0.29 per diluted share.

Non-GAAP net loss for the quarter ended September 30, 2019 was $21.9 million, or $0.46 per diluted share. Non-GAAP net income in the same quarter of 2018 was $8.1 million, or $0.17 per diluted share. This quarterly non-GAAP net (loss) income measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, changes in the fair value of acquired contingent consideration, and goodwill impairment charges. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At September 30, 2019, the Company had cash, cash equivalents and short-term investments of $253 million. The Company has $345 million of convertible senior notes due in 2021 with a conversion price of $42.56.

Revised 2019 Financial Guidance; 2020 Financial Guidance

2019: R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million.
2020: R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A expenses for the full year 2020 are expected to be $160 – $165 million.
These are non-GAAP projections that exclude restructuring costs and share-based compensation, as more fully described below under "Non-GAAP Financial Measures."
Highlights

Corporate Restructuring
– In October, the Company announced a corporate restructuring to reduce costs and focus its resources on the launch of INBRIJA. As part of this restructuring, Acorda is reducing headcount by approximately 25% through a reduction in force which is expected to be completed in Q1 2020. In connection with the restructuring, the Company also announced the reduced estimated 2019 operating expenses and 2020 operating expense guidance described above.

– The Company expects to realize estimated annualized cost savings related to headcount reduction of approximately $21 million beginning in 2020. Acorda estimates that it will incur approximately $8 million of pre-tax charges for severance and other costs related to the restructuring, through the first quarter of 2020.

– Total operating expenses in 2020 are estimated to be ~$60 million less than in 2019.

INBRIJA launch metrics through October 2019
– ~ 6,400 prescription request forms (PRFs)

– > 3,100 patients received a first dispense

– > 12,750 total cartons dispensed

– > 1,600 unique prescribers; ~55% repeat prescribers

As of October 30, 2019, INBRIJA is available in the U.S. without the need for a medical exception for ~66% of commercial and ~25% of Medicare plan lives.
On September 24, the European Commission (EC) granted Marketing Authorization for Inbrija 33 mg inhalation powder, hard capsules. The Marketing Authorization approves Inbrija for use in the 28 countries of the European Union, as well as Iceland, Norway and Liechtenstein.
On October 7, the U. S. Supreme Court denied the Company’s petition for certiorari requesting review of the Federal Circuit Court of Appeals’ decision in Acorda Therapeutics, Inc. v. Roxane Laboratories, et al. That decision upheld the invalidation of certain Ampyra patents in litigation against various generics manufacturers.
Webcast and Conference Call

The Company will host a conference call today at 4:30 p.m. ET. To participate in the conference call, please dial (833) 236-2756 (domestic) or (647) 689-4181 (international) and reference the access code 5464078. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 7:30 p.m. ET on November 4, 2019 until 11:59 p.m. ET on December 4, 2019. To access the replay, please dial (800) 585-8367 (domestic) or (416) 621-4642 (international); reference code 5464078. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain historical and forward-looking non-GAAP financial measures. In particular, Acorda has provided non-GAAP net (loss) income, adjusted to exclude the items below, and has provided 2019 guidance for R&D and SG&A expenses on a non-GAAP basis. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes the presentation of non-GAAP net (loss) income, when viewed in conjunction with our GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because this measure excludes (i) non-cash compensation charges and benefits that are substantially dependent on changes in the market price of our common stock, (ii) non-cash interest charges related to the accounting for our outstanding convertible debt which are in excess of the actual interest expense owing on such convertible debt, as well as non-cash interest related to the Fampyra monetization, and acquired Biotie debt, (iii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods, (iv) goodwill impairment which is a non-cash charge that relates to a reduction in the market capitalization of the Company and is not routine to the operation of the business, and (v) expenses that pertain to non-routine restructuring events. The Company believes its non-GAAP net (loss) income measure helps indicate underlying trends in the Company’s business and is important in comparing current results with prior period results and understanding projected operating performance. Also, management uses this non-GAAP financial measure to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

In addition to non-GAAP net (loss) income, we have provided 2019 and 2020 guidance for R&D and SG&A expenses on a non-GAAP basis, as both exclude restructuring costs and share-based compensation charges. Due to the forward looking nature of this information, the amount of compensation charges needed to reconcile these measures to the most directly comparable GAAP financial measures is dependent on future changes in the market price of our common stock and is not available at this time. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, The Company believes that the presentation of these non-GAAP measures, when viewed in conjunction with our GAAP results, provides investors with a more meaningful understanding of our projected operating performance because they exclude (i) expenses that pertain to non-routine restructuring events, and (ii) non-cash charges that are substantially dependent on changes in the market price of our common stock. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

On November 4, 2019 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company" or "we") reported its third-quarter 2019 financial results (Press release, Bausch Health, NOV 4, 2019, View Source [SID1234550217]).

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"In the third quarter, Bausch Health delivered another strong quarter with both reported and organic revenue growth1,2, demonstrating that our efforts to grow our core businesses are continuing to gain traction. In addition to organic revenue growth1,2 in both the Bausch + Lomb/International and Salix segments due to higher revenues in several of our durable, established brands, such as XIFAXAN, BioTrue ONEday and Bausch + Lomb ULTRA, our performance was also strengthened by the success of newer products, such as LUMIFY and Thermage FLX, " said Joseph C. Papa, chairman and CEO, Bausch Health. "Additionally, we are excited by the potential we see from the early days of the launch of DUOBRII, the newest product in our dermatology portfolio."

Company Highlights

Executing on Core Businesses and Advancing Pipeline

The Bausch + Lomb/International segment comprised approximately 53% of the Company’s reported revenue in the third quarter of 2019

Increased reported revenue in the Bausch + Lomb/International segment by 2% compared to the third quarter of 2018; revenue in this segment grew organically1,2 by 5% compared to the third quarter of 2018; growth was primarily driven by the Global Consumer, International Rx and Global Vision Care business units

Delivered 12th consecutive quarter of organic revenue growth1,2
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1 Please see the tables at the end of this news release for a reconciliation of this and other non-GAAP measures to the nearest comparable GAAP measure.
2 Organic growth/change, a non-GAAP metric, is defined as a change on a period-over-period basis in revenues on a constant currency basis (if applicable) excluding the impact of recent acquisitions, divestitures and discontinuations.

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Launched Ocuvite Eye Performance vitamins in the United States, which are formulated to help protect the eye from digital device blue light and UV exposure from the sun

The Salix segment comprised approximately 25% of the Company’s reported revenue in the third quarter of 2019

Increased reported revenue in the Salix segment by 20% compared to the third quarter of 2018

Increased reported revenue of XIFAXAN by 24% compared to the third quarter of 2018

The Ortho Dermatologics segment comprised approximately 7% of the Company’s reported revenue in the third quarter of 2019

The U.S. Food and Drug Administration accepted the New Drug Application for ARAZLO3 (IDP-123) Lotion with a PDUFA action date of Dec. 22, 2019; if approved, ARAZLO3 will be the first tazarotene acne treatment available in a lotion form

Expanded the cash-pay prescription program, Dermatology.com, to all Walgreens U.S. retail pharmacy locations

Reported revenues in the Global Solta business unit increased by 62% compared to the third quarter of 2018, driven by continued strong demand of Thermage FLX following the launch in the Asia Pacific region

Released annual Corporate Social Responsibility report

Strategic Capital Allocation and Debt Management

Increased Research and Development (R&D) by approximately 15%, or $16 million, compared to the third quarter of 2018

Repaid debt by approximately $450 million in the third quarter of 2019, including the net impact of activity under our revolving credit facility

Third-Quarter 2019 Revenue Performance
Total reported revenues were $2.209 billion for the third quarter of 2019, as compared to $2.136 billion in the third quarter of 2018, an increase of $73 million, or 3%. Excluding the unfavorable impact of foreign exchange of $15 million, the impact of a 2019 acquisition of $14 million and the impact of divestitures and discontinuations of $13 million, revenue grew organically1,2 by 4% compared to the third quarter of 2018, driven by organic growth2 in the Salix and Bausch + Lomb/International segments.

Bausch + Lomb/International Segment
Bausch + Lomb/International segment revenues were $1.175 billion for the third quarter of 2019, as compared to $1.147 billion for the third quarter of 2018, an increase of $28 million, or 2%. Excluding the unfavorable impact of foreign exchange of $15 million and the impact of divestitures and discontinuations of $9 million, the Bausch + Lomb/International segment grew organically1,2 by approximately 5% compared to the third quarter of 2018. The increase was primarily driven by growth in the Global Consumer, International Rx and Global Vision Care business units.

Salix Segment
Salix segment revenues were $551 million for the third quarter of 2019, as compared to $460 million for the third quarter of 2018, an increase of $91 million, or 20%. The increase was primarily driven by XIFAXAN, which grew 24% as compared to the third quarter of 2018.

Ortho Dermatologics Segment
Ortho Dermatologics segment revenues were $147 million for the third quarter of 2019, as compared to $176 million for the third quarter of 2018, a decrease of $29 million, or 16%, due to lower volumes primarily driven by the loss of exclusivity of ZOVIRAX, SOLODYN and ELIDEL, partially offset by organic revenue growth2 in the Global Solta business unit and from new product launches in the Ortho Dermatologics business unit.

Diversified Products Segment
Diversified Products segment revenues were $336 million for the third quarter of 2019, as compared to $353 million for the third quarter of 2018, a decrease of $17 million, or 5%. The decrease was primarily attributable to the previously reported loss of exclusivity for a basket of products.

Operating Income
Operating income was $329 million for the third quarter of 2019, as compared to an operating income of $117 million for the third quarter of 2018, an increase of $212 million. The increase in operating results for the third quarter of 2019 was primarily driven by a decrease in amortization and impairments and the increase in reported revenues and higher gross margins in 2019 as compared to 2018, offset by increased Selling, general and administrative (SG&A) expenses and R&D expenses.

Net Loss
Net loss for the three months ended Sept. 30, 2019 was $49 million, as compared to net loss of $350 million for the same period in 2018, a favorable impact of $301 million. The change is primarily due to the increased operating results noted above and the favorable change in income tax provision.

Adjusted net income (non-GAAP)1 for the third quarter of 2019 was $425 million, as compared to $403 million for the third quarter of 2018, an increase of $22 million, or 5%.

Cash Generated from Operations
The Company generated $515 million of cash from operations in the third quarter of 2019, as compared to $522 million in the third quarter of 2018.

EPS
GAAP Earnings Per Share (EPS) Diluted for the third quarter of 2019 was ($0.14), as compared to ($1.00) for the third quarter of 2018.

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Adjusted EBITDA (non-GAAP)1
Adjusted EBITDA (non-GAAP)1 was $942 million for the third quarter of 2019, as compared to $916 million for the third quarter of 2018, an increase of $26 million, or 3%.

2019 Financial Outlook
Bausch Health raised its revenue and Adjusted EBITDA (non-GAAP) guidance ranges for the full year of 2019:

Raised full-year revenue range from $8.40 – $8.60 billion to $8.475 – $8.625 billion

Raised full-year Adjusted EBITDA (non-GAAP) range from $3.425 – $3.575 billion to $3.50 – $3.60 billion

Mid-point of the November guidance ranges is up by $50 million versus the mid-point of the August 2019 guidance ranges

Other than with respect to GAAP Revenues, the Company only provides guidance on a non-GAAP basis. The Company does not provide a reconciliation of forward-looking Adjusted EBITDA (non-GAAP) to GAAP net income (loss), due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation. In periods where significant acquisitions or divestitures are not expected, the Company believes it might have a basis for forecasting the GAAP equivalent for certain costs, such as amortization, which would otherwise be treated as non-GAAP to calculate projected GAAP net income (loss). However, because other deductions (such as restructuring, gain or loss on extinguishment of debt and litigation and other matters) used to calculate projected net income (loss) vary dramatically based on actual events, the Company is not able to forecast on a GAAP basis with reasonable certainty all deductions needed in order to provide a GAAP calculation of projected net income (loss) at this time. The amount of these deductions may be material and, therefore, could result in projected GAAP net income (loss) being materially less than projected Adjusted EBITDA (non-GAAP). The guidance provided in this section represents forward-looking information, and actual results may vary. Please see the risks and assumptions referred to in the Forward-looking Statements section of this news release. The primary reasons for our change in our 2019 full-year guidance ranges are higher expected revenues from certain of our products with loss of exclusivity, better than expected base performance and improvements in our gross margins, partially offset by higher than expected R&D expenses.

Additional Highlights

Bausch Health’s cash, cash equivalents and restricted cash were $827 million at Sept. 30, 2019

The Company’s availability under the Revolving Credit Facility was approximately $1.055 billion at Sept. 30, 2019

Basic weighted average shares outstanding for the quarter were 352.4 million shares. Diluted weighted average shares outstanding for the quarter were 356.8 million shares5

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5 Diluted weighted average shares includes the dilutive impact of options and restricted stock units, which are 4,453,000 common shares for the 3 months ended Sept. 30, 2019, and which are excluded when calculating GAAP diluted loss per share because the effect of including the impact would be anti-dilutive.

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Conference Call Details
Date:
Monday, Nov. 4, 2019
Time:
8:00 a.m. ET
Webcast:
View Source
Participant Event Dial-in:
+1 (888) 317-6003 (United States)

+1 (412) 317-6061 (International)

+1 (866) 284-3684 (Canada)
Participant Passcode:
0458346
Replay Dial-in:
+1 (877) 344-7529 (United States)

+1 (412) 317-0088 (International)

+1 (855) 669-9658 (Canada)
Replay Passcode:
10135669 (replay available until Nov. 11, 2019)

On November 4, 2019 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported that the U.S. Food and Drug Administration has cleared its investigational new drug (IND) application for COM902, its immuno-oncology therapeutic antibody targeting TIGIT in patients with advanced malignancies (Press release, Compugen, NOV 4, 2019, View Source [SID1234550216]).

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Under this IND, the Company intends to initiate a Phase 1 clinical trial in patients with advanced malignancies for whom standard of care therapies are currently ineffective. Expected to begin in early 2020, the clinical trial is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of COM902. The study is planned to be conducted at multiple centers in the United States and site selection activities are currently underway.

"IND clearance for COM902 is an important milestone that marks the third program based on new drug targets we discovered to be evaluated in the clinic," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "We believe that TIGIT inhibitors may have an important role in the immunotherapy landscape and that our biology driven approach of combining anti-TIGIT and anti-PVRIG inhibitors, with or without PD-1 blockers, has the potential to improve clinical responses in patients who are unresponsive and refractory to currently approved immunotherapies. As the only company with clinical candidates targeting both PVRIG and TIGIT, we hold a differentiated position in the crowded immuno-oncology space."

Dr. Cohen-Dayag continued, "We are proud of our tremendous progress in recent years, transforming into a clinical-stage company with three anticipated Phase 1 programs in the clinic expected in 2020, two of which we are being developed internally. Importantly, these three programs address targets which originated from our unique computational discovery platform, highlighting the power and value of our computational capabilities to discover new, potentially significant biological pathways and targets for innovative therapeutics."

About COM902
COM902, a high affinity, fully human antibody targeting TIGIT, was developed for combination treatment with COM701. Preclinical data demonstrate that TIGIT inhibition, either alone or in combination with other checkpoint inhibitors, can enhance T cell activation and increase anti-tumor immune responses. Parallel inhibition of TIGIT and PVRIG, the two coinhibitory arms of the DNAM-1 axis, results in synergistic effects on effector T cell function and tumor growth inhibition in various model systems that can be further increased with the addition of PD-1 blockade. Based on preclinical data these combinations may be clinically important for enhancing anti-tumor immune response and expanding the patient population responsive to checkpoint inhibition.

Compugen discovered TIGIT in 2009 leveraging its immune checkpoint computational discovery platform through which PVRIG was also discovered. The TIGT discovery was published by Compugen in October 2009 in the Proceedings of the National Academy of Sciences (PNAS).

On November 4, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported financial results for the third quarter 2019 (Press release, Karyopharm, NOV 4, 2019, View Source [SID1234550215]). In addition, Karyopharm highlighted select corporate milestones, including an update regarding the initial U.S. commercial launch of XPOVIO (selinexor), and provided an overview of its key clinical development programs.

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"This has been a landmark quarter for Karyopharm as we saw the accelerated approval and commercial launch of XPOVIO, the first and only oral nuclear export inhibitor approved in the U.S., indicated for patients with heavily pretreated multiple myeloma," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "The XPOVIO launch is off to a very strong start and we are extremely pleased with the early commercial uptake and feedback from prescribing physicians. As we look ahead to the next few months, we eagerly await the top-line results from the ongoing Phase 3 BOSTON study, which, if positive, would represent another significant advancement in the treatment of patients with relapsed or refractory multiple myeloma. Finally, we remain on track to file, by the end of 2019, a New Drug Application in the U.S. for selinexor requesting accelerated approval for patients with relapsed or refractory diffuse large B-cell lymphoma."

Third Quarter 2019 Highlights and Recent Progress

XPOVIO (selinexor) in Multiple Myeloma

XPOVIO U.S. Commercial Rollout Off to a Strong Start. Oral XPOVIO, Karyopharm’s first-in-class, nuclear export inhibitor, became commercially available to patients in the U.S on July 9, 2019 and generated net product sales of $12.8 million in the third quarter. As of September 30, 2019, over 500 XPOVIO prescriptions have been fulfilled, driven by strong demand from both academic and community-based oncologists. In less than 3 months on the market, XPOVIO has been prescribed by more than 300 unique physicians and healthcare accounts.

Third quarter sales were driven by a combination of new patient starts, prescription refills, and initial channel inventory to Karyopharm’s distribution partners. Patient demand for XPOVIO continued to increase in each subsequent month of the quarter following its accelerated approval by the U.S. Food and Drug Administration (FDA) in July. Rapid insurance coverage for XPOVIO has been a key contributor to its early commercial success with XPOVIO already being added to numerous national commercial and Medicare formularies and coverage policies. Based on prescription fulfillment data through the specialty pharmacy channel, Karyopharm estimates that approximately 60% of XPOVIO prescriptions have been dispensed to patients with Medicare coverage and 35% to patients with commercial insurance, with the remaining 5% of patients having either Medicaid or another form of prescription coverage.

XPOVIO Receives Accelerated Approval from the FDA. On July 3, 2019, the FDA approved XPOVIO for use in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. XPOVIO is the first of a novel drug class designated selective inhibitors of nuclear export (SINE) and is the first ever nuclear export inhibitor approved for human use. This first indication received accelerated approval based on response rate. As with all accelerated approvals, continued approval for the treatment of myeloma may be contingent upon verification and description of clinical benefit in a confirmatory trial. The ongoing Phase 3 BOSTON study is intended to serve as the confirmatory trial for the accelerated approval of XPOVIO.

XPOVIO Phase 2b STORM Study Results Published in the New England Journal of Medicine. Results from Karyopharm’s Phase 2b STORM study evaluating XPOVIO in patients with heavily pretreated, triple class refractory multiple myeloma were published in the New England Journal of Medicineon August 22, 2019. In STORM, XPOVIO achieved a 26% overall response rate, median overall survival (OS) of 8.6 months, and a median survival of 15.6 months in the 39% of patients with a minimal response or better.

XPOVIO Granted Seven Years Orphan Drug Market Exclusivity by FDA. In September 2019, Karyopharm received confirmation from the FDA that the Company is entitled to seven years of orphan-drug exclusivity for XPOVIO’s approved indication for the treatment of patients with relapsed or refractory multiple myeloma, pursuant to section 527 of the Federal Food, Drug, and Cosmetic Act.

Five Abstracts Presented at the 17thInternational Myeloma Workshop (IMW). Multiple data presentations at this year’s IMW, held September 12-15, 2019, continue to reinforce the potential clinical utility of XPOVIO as a new therapeutic option for patients with relapsed or refractory multiple myeloma. One key abstract, in particular, titled, "Outcomes of Triple Class Refractory Penta-Exposed Multiple Myeloma (MM)," (Cornell, et al) compared the OS rate from the retrospective MAMMOTH study (Gandhi, 2019), which evaluated outcomes from patients with relapsed or refractory multiple myeloma after their disease became refractory to CD38 monoclonal antibodies, with a similarly-matched cohort of patients from Karyopharm’s Phase 2b STORM study. Patients in STORM, who received selinexor and dexamethasone as first line of therapy after their disease became triple class refractory (n=64), compared to matched patients receiving currently available therapies from the MAMMOTH cohort (n=128), showed an unadjusted hazard ratio (HR) of 0.64 (p=0.043). An adjusted analysis, which takes into consideration differences in baseline characteristics between the two groups, showed a HR of 0.55 (p=0.009).

Decision from European Medicines Agency (EMA) for Marketing Authorization Application (MAA) Expected in Early 2020. In January 2019, Karyopharm submitted an MAA to the EMA requesting conditional approval for selinexor, in combination with dexamethasone, as a new treatment for patients with heavily pretreated multiple myeloma based on the results of the Phase 2b STORM study. The Company expects to receive a decision on the MAA in early 2020.

Pivotal Phase 3 BOSTON Study On Track. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is progressing and patient enrollment was completed in January 2019. Top-line data are expected in early 2020 contingent upon the occurrence of progression-free survival (PFS) events, the primary endpoint of the study. The BOSTON study is evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (bortezomib) (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. Data from the BOSTON study, if positive, are expected to be used to support regulatory submissions to the FDA and EMA requesting the use of selinexor in combination with Velcade and dexamethasone in patients with multiple myeloma who have received at least one prior therapy.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)

NDA Expected to be Submitted by End of 2019. Following the positive results from the Phase 2b SADAL study that were first presented at the America Society of Hematology 2018 Annual Meeting and then updated in June at the 2019 International Conference on Malignant Lymphoma, Karyopharm expects to submit a New Drug Application (NDA) to the FDA by the end of 2019 requesting accelerated approval for selinexor as a treatment for patients with relapsed or refractory DLBCL after at least two prior multi-agent therapies and who are ineligible for stem cell transplantation including CAR-T (chimeric antigen receptor modified T cell) therapy. The Company also expects to submit an MAA to the EMA in 2020 requesting conditional approval for selinexor in the same indication. In addition to orphan drug designation, selinexor was granted fast track designation for this indication by the FDA in 2018.
Selinexor in Solid Tumors

Ongoing Phase 3 Portion of the Phase 2/3 SEAL Study in Liposarcoma. Karyopharm previously reported positive results from the Phase 2 portion of the randomized, blinded Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with previously treated, advanced unresectable dedifferentiated liposarcoma. Enrollment is currently ongoing in the Phase 3 portion of the SEAL study. Top-line data from the Phase 3 portion of the SEAL study are anticipated in 2020. Assuming a positive outcome on the primary endpoint of PFS, the Company intends to use the data from the SEAL study to support NDA and MAA submissions requesting approval for selinexor for patients with advanced unresectable dedifferentiated liposarcoma.
Corporate and Financial Updates

Executed Royalty Agreement with HealthCare Royalty Partners for up to $150 Million. In September 2019, Karyopharm executed a royalty agreement with HealthCare Royalty Partners (HCR) for up to $150 million to support the ongoing development and commercialization of XPOVIO. Under the terms of the agreement, Karyopharm received $75 million in September 2019 and is eligible to receive an additional $75 million upon the achievement of future regulatory and commercial milestones, as well as closing conditions. In exchange for the first $75 million, HCR will receive a tiered royalty in the mid-single digits based on worldwide net revenues of XPOVIO and any other future products beginning in October 2019.
Third Quarter 2019 Financial Results

Net product revenue for the quarter ended September 30, 2019 was $12.8 million, which reflects the first quarter of recorded sales for XPOVIO. Karyopharm did not have any product revenue for the quarter ended September 30, 2018.

License and other revenue for the third quarter 2019 was $0.3 million, compared to $0.2 million for the third quarter of 2018.

Cost of sales for the third quarter 2019, was $1.0 million, which reflects the costs of XPOVIO units sold and third-party royalties on our net product revenue. Karyopharm did not have any cost of sales for the third quarter 2018.

Research and development expense for the third quarter 2019 was $26.3 million, compared to $36.4 million for the third quarter of 2018. Karyopharm expects research and development expense to be relatively consistent for the final quarter of 2019 compared to the third quarter of 2019. For the third quarter 2019, selling, general and administrative expense was $25.3 million, compared to $13.0 million for the third quarter 2018. The increase in selling, general and administrative expenses compared to the prior year period was due primarily to the hiring of the Karyopharm commercial team and related activities to support the U.S. commercial launch of XPOVIO.

Karyopharm reported a net loss of $41.4 million, or $0.67 per share, for the third quarter 2019, compared to a net loss of $48.1 million, or $0.79 per share, for the third quarter 2018. Net loss includes non-cash stock-based compensation expense of $3.7 million and $4.8 million for the 2019 and 2018 quarters, respectively.

Cash, cash equivalents, restricted cash and investments as of September 30, 2019 totaled $270.3 million, compared to $330.9 million as of December 31, 2018.

2019 Financial Outlook

Based on its current operating plans, Karyopharm expects its non-GAAP operating expenses, which excludes stock-based compensation expense, for the full year 2019 to be in the range of $200 million to $210 million. Karyopharm has not reconciled the full year 2019 outlook for non-GAAP operating expenses to full year 2019 outlook for GAAP operating expenses because Karyopharm cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the full year 2019 outlook for non-GAAP operating expenses.

The Company expects that its existing cash, cash equivalents and investments, and the revenue it expects to generate from XPOVIO product sales, will be sufficient to fund its planned operations into the middle of 2021.

Additional key activities expected in the remainder of 2019 include supporting the ongoing multiple myeloma regulatory filing for selinexor in Europe, progressing the pivotal Phase 3 BOSTON study in multiple myeloma and submitting an NDA in the U.S. in DLBCL.

Non-GAAP Financial Information

Karyopharm uses a non-GAAP financial measure, non-GAAP operating expense, to provide operating expense guidance. Karyopharm believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Karyopharm’s operating performance as it excludes non-cash stock compensation expense. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP total operating expense and should not be considered a measure of Karyopharm’s liquidity. Instead, non-GAAP operating expense should only be used to supplement an understanding of Karyopharm’s operating results as reported under GAAP.

Conference Call Information

Karyopharm will host a conference call today, Monday, November 4, 2019, at 8:30 a.m. Eastern Time, to discuss the third quarter 2019 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 3353586 . A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

Important Safety Information

The most common adverse reactions observed in patients treated with XPOVIO (incidence ≥20%) are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea, and upper respiratory tract infection.

The treatment discontinuation rate due to adverse reactions was 27%; 53% of patients had a reduction in the XPOVIO dose, and 65.3% had the dose of XPOVIO interrupted. The most frequent adverse reactions requiring permanent discontinuation in 4% or greater of patients who received XPOVIO included fatigue, nausea, and thrombocytopenia. The rate of fatal adverse reactions was 8.9%.

On November 4, 2019 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that the HALO-301 Phase 3 clinical study evaluating investigational new drug PEGPH20 as a first-line therapy for treatment of patients with metastatic pancreas cancer failed to reach the primary endpoint of overall survival (Press release, Halozyme, NOV 4, 2019, View Source [SID1234550214]).

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The treatment arm of PEGPH20 in combination with gemcitabine and nab-paclitaxel (ABRAXANE) failed to demonstrate an improvement in median overall survival compared to gemcitabine and nab-paclitaxel alone (11.2 months compared to 11.5 months, HR=1.00, p=0.9692). While there was a higher response rate in the PEGPH20 treatment arm, this did not translate into an improvement in duration of response, Progression Free Survival or Overall Survival.

"Patients in both treatment arms of the HALO-301 trial surpassed the published median overall survival rates from the pivotal registration study of ABRAXANE plus gemcitabine as first-line therapy for metastatic pancreas cancer, published in 2013. Based on the lack of benefit over standard-of-care in this study, which performed well versus published data, we will be discontinuing PEGPH20 clinical development," said Dr. Helen Torley, president and CEO of Halozyme. "This well-designed and well-executed study did not have the outcome that we or the study participants wanted or expected. I would like to extend a heartfelt thank you to all those who supported and who made this study possible, including the patients who were enrolled, their families, our investigators, their staff, our investors and all of the dedicated Halozyme employees."

Halozyme intends to halt development activities for PEGPH20 and implement an organizational restructuring to focus its operations solely on its ENHANZE drug delivery technology.

Further details regarding the organizational restructuring and other strategic actions to position Halozyme for the future can be found in a separate press release issued today.

Conference Call and Webcast Information
Halozyme will webcast a conference call today at 8:30 a.m. ET / 5:30 a.m. PT to discuss its plans to focus strategy on its ENHANZE drug delivery technology. Dr. Helen Torley, president and chief executive officer, will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a replay will be available following the close of the call. To access the webcast, please visit www.halozyme.com approximately fifteen minutes prior to the call to register, download and install any necessary audio software. The call may also be accessed by dialing (877) 824-0907 (domestic callers) or (647) 689-5655 (international callers) using passcode 3069304. A telephone replay will be available after the call by dialing (800) 585-8367 (domestic callers) or (416) 621-4642 (international callers) using replay ID number 3069304.