On August 6, 2019 Myovant Sciences (NYSE: MYOV), a clinical-stage healthcare company focused on developing and commercializing innovative therapies for women’s health and prostate cancer, reported recent corporate updates and reported financial results for the first fiscal quarter ended June 30, 2019 (Press release, Myovant Sciences, AUG 6, 2019, View Source [SID1234538252]).

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"Myovant Sciences recently announced positive top-line data for the LIBERTY 1 and LIBERTY 2 studies evaluating relugolix combination therapy in women with uterine fibroids, as well as the positive results from a separate bioequivalence study supporting a potential one pill, once-a-day dosing regimen of relugolix combination therapy," said Lynn Seely, M.D., President and Chief Executive Officer of Myovant Sciences. "These results confirm the potential of relugolix to offer a constellation of attributes in a single pill, taken once-a-day and we are now focused on preparing for the New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA), which we plan to file by the end of this calendar year. We also look forward to reporting data from our Phase 3 prostate cancer study later this calendar year and results from our two Phase 3 endometriosis studies in the first and second quarters of calendar year 2020."
First Fiscal Quarter 2019 and Recent Business Highlights
Relugolix Phase 3 Clinical Programs

On May 14, 2019, Myovant Sciences announced positive top-line results from the LIBERTY 1 Phase 3 study evaluating relugolix combination therapy (relugolix 40 mg plus estradiol 1.0 mg and norethindrone acetate 0.5 mg) once-a-day in women with uterine fibroids and heavy menstrual bleeding. The study met its primary endpoint with a p-value of <0.0001 and achieved six key secondary endpoints with a well-tolerated safety profile.

On July 23, 2019, Myovant Sciences announced positive top-line results from the LIBERTY 2 Phase 3 study evaluating relugolix combination therapy in women with uterine fibroids and heavy menstrual bleeding. This study also met its primary endpoint with a p-value of <0.0001 and achieved six key secondary endpoints with a well-tolerated safety profile.

On July 23, 2019, Myovant Sciences also announced that the single-tablet relugolix combination therapy met all required FDA criteria in a separate bioequivalence study supporting a potential one-pill, once-a-day dosing regimen of relugolix.

Based on the positive top-line results for LIBERTY 1 and LIBERTY 2, Myovant Sciences currently plans to submit an NDA for one-pill, once-a-day relugolix combination therapy for the treatment of heavy menstrual bleeding and uterine fibroids to the FDA in the fourth quarter of calendar year 2019 and the Marketing Authorisation Application to the European Medicines Agency in the first quarter of calendar year 2020.

Enrollment of approximately 130 additional men with metastatic prostate cancer in the Phase 3 HERO study was completed in July 2019. The objective of enrolling these men was to assess the secondary objective of demonstrating that relugolix can delay the time to progression of the lethal state of the disease, castration-resistant prostate cancer, as compared to leuprolide.
MVT-602 Clinical Program

Myovant Sciences completed a successful dose-finding pharmacokinetic/pharmacodynamic Phase 2a study of MVT-602, a kisspeptin-1 receptor agonist, in healthy women undergoing a minimal controlled ovarian stimulation protocol. Top-line results were presented at the European Society of Human Reproduction in Vienna, Austria in June 2019. The study demonstrated that MVT-602 was generally well-tolerated and produced the desired luteinizing hormone surge associated with high and dose-dependent rates of ovulation in healthy women following a minimal controlled ovarian stimulation protocol.
Corporate

On June 4, 2019, Myovant Sciences completed an underwritten public equity offering, receiving net proceeds of approximately $134.5 million.

In the first quarter of fiscal year 2019, Myovant Sciences received aggregate net proceeds of $2.5 million pursuant to the issuance of common shares under its "at-the-market" equity offering program.
First Fiscal Quarter 2019 Financial Summary
Research and development (R&D) expenses for the quarter ended June 30, 2019, were $51.1 million compared to $51.3 million for the comparable prior year period. The composition of R&D expenses in both periods is similar, and primarily includes expenses related to Myovant Sciences’ Phase 3 clinical studies as well as personnel-related expenses for employees engaged in R&D activities. R&D expenses for the quarter ended June 30, 2018 reflected a ramp up in relugolix Phase 3 study costs primarily related to study enrollment, whereas R&D expenses for the quarter ended June 30, 2019 reflect lower relugolix Phase 3 study costs as certain studies are in the process of winding down. The decrease in relugolix Phase 3 study costs were partially offset by increases in other R&D spending related to Myovant Sciences’ preparations to seek regulatory approval for its product candidates.
General and administrative (G&A) expenses for the quarter ended June 30, 2019, were $14.2 million compared to $8.7 million for the comparable prior year period. The increase primarily reflects increases in personnel-related expenses, professional service fees, share-based compensation, and other general overhead and administrative expenses to support Myovant Sciences’ headcount growth and expanding operations.
Interest expense for the quarter ended June 30, 2019, was $3.8 million compared to $1.6 million in the comparable prior year period. The increase for the quarter was primarily the result of higher outstanding debt balances under the financing agreements as compared to the prior year period.
Interest income for the quarter ended June 30, 2019, was $0.8 million. There was no interest income for the quarter ended June 30, 2018. During the quarter ended June 30, 2019, a portion of Myovant Sciences’ cash was invested in a combination of money market funds and commercial paper. There were no such investments during the prior year period.

Net loss for the quarter ended June 30, 2019, was $67.9 million, compared to $62.1 million for the comparable prior year period. On a per common share basis, net loss was $0.89 and $0.98 for the quarters ended June 30, 2019, and 2018, respectively. The increase in the net loss for the quarter was driven primarily by the increase in costs outlined above.
Capital resources: Cash and cash equivalents totaled $226.7 million as of June 30, 2019. During the quarter ended June 30, 2019, Myovant Sciences raised net proceeds of approximately $134.5 million from an underwritten public equity offering and approximately $2.5 million from its "at-the-market" equity offering program. Myovant Sciences currently has approximately $10.4 million of capacity available under the "at-the-market" equity offering program that it initiated in April 2018.

About Relugolix
Relugolix is a once-a-day, oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces ovarian estradiol and progesterone production, hormones known to stimulate uterine fibroids and endometriosis. Myovant Sciences has successfully completed two Phase 3 clinical studies (LIBERTY 1 and LIBERTY 2) evaluating relugolix combination therapy (relugolix 40 mg plus 1.0 mg estradiol with 0.5 mg norethindrone acetate) in women with heavy menstrual bleeding and uterine fibroids and is studying relugolix combination therapy in two Phase 3 clinical studies (SPIRIT 1 and SPIRIT 2) in women with endometriosis-associated pain. Data from SPIRIT 2 and SPIRIT 1 are expected in the first and second quarters, respectively, of calendar year 2020. Relugolix also lowers testosterone in men, an androgen known to drive the growth of prostate cancer. Myovant Sciences is evaluating relugolix, 120 mg once-a-day, in the Phase 3 HERO study in men with advanced prostate cancer. Top-line results from the HERO study are expected in the fourth quarter of calendar year 2019.

About MVT-602

MVT-602 is an oligopeptide kisspeptin-1 receptor agonist. Kisspeptin, the ligand, is a naturally-occurring peptide that stimulates GnRH release and is required for puberty and maintenance of normal reproductive function, including production of sperm, follicular maturation and ovulation, and production of estrogen and progesterone in women and testosterone in men. A Phase 2a clinical study in healthy female volunteers to characterize the dose-response curve in a minimal controlled ovarian stimulation setting has been completed.

On August 6, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported financial results for the second quarter 2019 (Press release, Karyopharm, AUG 6, 2019, View Source [SID1234538195]). In addition, Karyopharm highlighted select corporate milestones, including an update regarding the initial commercial launch of XPOVIO, and provided an overview of its key clinical development programs.

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"Our second quarter progress was followed by a transformational milestone: the U.S. Food and Drug Administration (FDA) granting accelerated approval of oral XPOVIO (www.XPOVIO.com), indicated for patients with heavily pre-treated multiple myeloma," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "The commercial launch of XPOVIO, our first approved product, is off to a strong start with encouraging early prescribing trends from both academic and community-based physicians throughout the U.S. While we are laser-focused on the success of the initial launch, we remain deeply committed to serving the future needs of patients well beyond those directly indicated in the U.S. accelerated approval. To that end, we eagerly await the clinical trial results from the ongoing Phase 3 BOSTON study and continue to support the evaluation of a Marketing Authorization Application (MAA) of selinexor currently under review by the European Medicines Agency (EMA). And finally, we expect to rapidly advance the regulatory filings for selinexor in both the U.S. and Europe requesting accelerated and conditional approval, respectively, for patients with relapsed or refractory diffuse large B-cell lymphoma."

Second Quarter 2019 Highlights and Recent Progress

Selinexor in Multiple Myeloma

XPOVIO (selinexor) Receives Accelerated Approval from the FDA. On July 3, 2019, the FDA approved oral XPOVIO, Karyopharm’s first-in-class, nuclear export inhibitor. XPOVIO was approved 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. The first indication is approved under accelerated approval based on response rate. The ongoing Phase 3 BOSTON study will serve as the confirmatory trial for the accelerated approval of XPOVIO. As with all accelerated approvals, continued approval the treatment of myeloma may be contingent upon verification and description of clinical benefit in a confirmatory trial.

XPOVIO Commercial Launch Underway in the U.S. On July 9, 2019, XPOVIO became commercially available to patients in the U.S. The commercial launch of XPOVIO is being supported by approximately 70 Karyopharm sales representatives and nurse liaisons as well as an extensive patient and healthcare provider support program called KaryForwardTM. Karyopharm’s commercial efforts are also being supplemented by patient support initiatives coordinated by our dedicated network of participating specialty pharmacy providers. Early prescribing trends are encouraging with robust demand from both academic and community-based physicians throughout the U.S. with early prescriptions being filled for patients with Medicare and commercial insurance coverage.

European Medicines Agency (EMA) Validates Marketing Authorization Application (MAA). In January 2019, Karyopharm submitted a 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. As a customary part of the MAA review process, Karyopharm received the consolidated list of questions from EMA in early May 2019 and received additional feedback, including the integrated inspection report, based on routine site audits and other activities. The Company promptly addressed the questions and feedback with EMA and the evaluation process of the MAA is ongoing. The Company expects to receive a decision on the application by the end of 2019 or early 2020.

Pivotal Phase 3 BOSTON Study in Progress. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is progressing and patient enrollment is complete. Top-line data are expected by the end of 2019 or 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, is expected to be used to support regulatory submissions to the FDA and EMA requesting the use of selinexor in patients with multiple myeloma who have received at least one prior therapy.

New and Updated Phase 1b/2 STOMP Data Presented at European Hematology Association (EHA) (Free EHA Whitepaper) 2019 Annual Meeting. Three selinexor abstracts highlighting new and updated clinical data from patients receiving a combination of selinexor and standard of care myeloma drugs were presented in June at the EHA (Free EHA Whitepaper) 2019 Annual Meeting. Specifically, clinical data from the Kyprolis (carfilzomib), Darzalex (daratumumab) and Pomalyst (pomalidomide) arms of the Phase 1b/2 STOMP study were presented. For the Kyprolis arm, once weekly oral selinexor in combination with low dose dexamethasone demonstrated a 78% overall response rate (ORR) in patients with heavily pre-treated, Kyprolis-naïve multiple myeloma. For the Darzalex arm, once weekly oral selinexor in combination with low dose dexamethasone demonstrated a 73% ORR in patients with heavily pre-treated, Darzalex-naïve multiple myeloma. And finally, for the Pomalyst arm, once weekly oral selinexor in combination with low dose dexamethasone demonstrated a 57% ORR in patients with Revlimid (lenalidomide)-relapsed or -refractory, Pomalyst-naïve multiple myeloma with PFS of 12.2 months. Across all 3 arms of the STOMP study presented, the most common treatment-related adverse events (AEs) were cytopenias, along with gastrointestinal and constitutional symptoms; most were manageable with dose modifications and/or standard supportive care.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)

NDA and MAA Expected to be Submitted between Q4 2019 and Q1 2020. 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 and an MAA to the EMA requesting accelerated and conditional approval of selinexor, respectively, 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. In addition to Orphan Drug Designation, selinexor was granted Fast Track designation 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. 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. Top-line data from the Phase 3 portion of the SEAL study are anticipated in 2020.

Company-Sponsored Phase 3 SIENDO Study Evaluating Selinexor as Maintenance Therapy in Endometrial Cancer Now Underway. During the first quarter of 2019, an Investigational New Drug Application (IND) was submitted and accepted by the FDA for a randomized, blinded Phase 2/3 study evaluating selinexor versus placebo as a maintenance therapy in patients with advanced or recurrent endometrial cancer following one prior platinum-based treatment. There are currently no approved therapies to treat patients with advanced or recurrent endometrial cancer in the maintenance setting. The primary endpoint of the SIENDO study is PFS. This study is being led by Professor Ignace Vergote, MD, PhD, Head of the Department of Obstetrics and Gynaecology and Gynaecologic Oncology at the Catholic University of Leuven, Belgium. Karyopharm is targeting enrollment completion for SIENDO in 2020.

Updated Phase 2 KING Data in Glioblastoma Presented at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting. An abstract highlighting updated efficacy and safety results from the Phase 2 KING study evaluating single-agent selinexor in patients with recurrent glioblastoma was presented in June at the ASCO (Free ASCO Whitepaper) 2019 Annual Meeting. The KING study evaluated three different dosing schedules with selinexor (50mg/m2 twice per week, 60mg twice per week and 80mg once per week) and the reported results followed completion of accrual in the non-surgical cohorts in the study (n=68). Based on the efficacy and tolerability results from the study, the 80mg once per week dosing regimen is recommended for further evaluation. Of the 30 patients treated in the 80mg dosing cohort, the overall response rate reported was 10%, with 19% of patients achieving a 6-month PFS rate and 30% of patients achieving a 6-cycle PFS rate. The most common non-hematologic AEs in this cohort were nausea, fatigue, anorexia, and vomiting, all of which were Grade 1 or 2 events. The most common treatment-related hematological AEs were primarily Grade 1 and 2 and included leukopenia, neutropenia, anemia, and thrombocytopenia. There was one case (3%) of Grade 4 treatment-related lymphopenia and no Grade 5 treatment-related AEs were reported.
Corporate Updates

Founder Sharon Shacham Receives New York Intellectual Property Law Association (NYIPLA) Inventor of the Year Award. In May, Karyopharm’s founder, President and Chief Scientific Officer, Sharon Shacham, PhD, MBA, received the esteemed NYIPLA 2019 "Inventor of the Year" award. Dr. Shacham was recognized for her scientific research that led to the development of oral selinexor and related compounds. Past winners of this award have included the inventors of CAR-T therapy, Gleevec, Valium, LASIK laser vision correction and Priceline.com, among many others.
Second Quarter 2019 Financial Results

Cash, cash equivalents and investments as of June 30, 2019, including restricted cash, totaled $217.9 million, compared to $330.9 million as of December 31, 2018.

License and other revenue for the quarter ended June 30, 2019 was $9.5 million, compared to $19.9 million for the quarter ended June 30, 2018, both of which were primarily related to the Company’s license agreements with Antengene and Ono, respectively.

For the quarter ended June 30, 2019, research and development expense was $26.5 million, compared to $44.7 million for the quarter ended June 30, 2018. Karyopharm expects research and development expense to be relatively consistent for the remainder of 2019 compared to the second quarter of 2019. For the quarter ended June 30, 2019, general and administrative expense was $24.7 million compared to $9.5 million for the quarter ended June 30, 2018. The increase in general and administrative expenses compared to the prior year period was due primarily to the hiring of the Karyopharm commercial team and related commercial launch preparation activities to support the U.S. commercial launch of XPOVIO.

Karyopharm reported a net loss of $43.4 million, or $0.71 per share, for the quarter ended June 30, 2019, compared to a net loss of $33.7 million, or $0.60 per share, for the quarter ended June 30, 2018. Net loss includes non-cash stock-based compensation expense of $4.1 million and $4.4 million for the quarters ended June 30, 2019 and June 30, 2018, respectively.

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 $215 million. The Company expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations into the second half of 2020. Additional key activities expected in 2019 include supporting the ongoing multiple myeloma regulatory filing for selinexor in Europe, progressing the pivotal Phase 3 BOSTON study in multiple myeloma and potentially submitting an NDA and MAA, in the U.S. and Europe, respectively, in DLBCL.

Non-GAAP Financial Information and Other Disclosures

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. Karyopharm has not provided GAAP reconciliation for its forward-looking non-GAAP annual operating expense 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 forward-looking non-GAAP financial measure.

Conference Call Information

Karyopharm will host a conference call today, Tuesday, August 6, 2019, at 8:30 a.m. Eastern Time, to discuss the second 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 5550468. 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%.

The full Prescribing Information for XPOVIO is available at www.XPOVIO.com.

On August 6, 2019 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported that a supply of Namodenoson has been manufactured and is ready for use in the treatment of advanced liver cancer patients under compassionate use at the Rabin Medical Center in Israel (Press release, Can-Fite BioPharma, AUG 6, 2019, View Source [SID1234538211]).

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Compassionate use allows doctors and their patients the option of early access to investigational new drugs, under closely controlled and monitored circumstances, when a patient who is facing serious illness has exhausted all available treatment options.

Salomon M. Stemmer, MD, the Principal Investigator of the Company’s prior Phase II liver cancer study said, "Given the evidence of clinical benefit of Namodenoson in patients with hepatocellular carcinoma and Child Pugh B7 to whom there is no accepted well established treatment, I plan to offer Namodenoson to certain HCC CPB7 patients in the compassionate use setting."

Can-Fite recently announced results from its Phase II study of Namodenoson in the treatment of advanced liver cancer. Namodenoson was found to increase overall survival in HCC patients with Child Pugh B7, the largest subpopulation of the study, as compared to placebo, even though the trial did not meet its primary endpoint.

An end of Phase II meeting with the U.S. Food and Drug Administration to review study data and to present the design of a Phase III clinical trial is expected soon. The FDA has granted Namodenoson both Orphan Drug and Fast Track status providing a pathway for accelerated approval based on unmet need in the treatment of advanced liver cancer. Fast Track designation offers advantages including more frequent meetings with the FDA and rolling review, which provides the opportunity to submit parts of its New Drug Application (NDA) for review prior to completing the entire application for commercialization. Orphan Drug designation includes 7-year market exclusivity following marketing approval, FDA assistance during the drug development process, and exemption of application fees.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

On August 6, 2019 CytoSorbents Corporation (NASDAQ: CTSO), a critical care immunotherapy leader using its CytoSorb blood purification technology to treat deadly inflammation in critically-ill and cardiac surgery patients around the world, reported financial and operational results for the quarter ending June 30, 2019 (Press release, Cytosorbents, AUG 6, 2019, View Source [SID1234538227]).

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Second Quarter 2019 Financial Results:

Total Q2 2019 revenues were $6.2 million, including both product sales and grant income, an increase of approximately 8% from $5.8 million in Q2 2018
Product sales for Q2 2019 were approximately $5.9 million, compared to $5.2 million for Q2 2018, an increase of approximately 12%. Q2 2019 product sales would have been approximately $6.2 million, or $357,000(18%) higher than a year ago, had the Euro remained unchanged
Product gross margins for Q2 2019 increased to 76%, compared to 74% in Q2 2018
Executed a loan amendment with Bridge Bank which provided an additional $5 million in debt financing which was received on July 31, 2019, extending the interest-only period of the entire term loan to October 2020, provided certain financial targets are met
Strong cash position with $16.3M at the end of the quarter, and approximately $20 million at the end of July, reflecting the additional proceeds from debt
Second Quarter 2019 Operational Highlights:

67,000+ cumulative CytoSorb treatments have been delivered, up from 46,000 a year ago
CytoSorb product registration has been submitted in Mexico and South Korea with partner Fresenius Medical Care, with concurrent initiation of commercialization planning and pre-launch activities including conferences, marketing, and key opinion leader events. Feedback on the status of registration is expected before the end of this year
Publication of the U.S. REFRESH I study in Seminars in Thoracic and Cardiovascular Surgery, a journal of the American Association for Thoracic Surgery (AATS) and an associated editorial commentary on the clinical trial and the potential clinical importance and value that CytoSorb could have in complex cardiac surgery with future studies
Enrollment in the 400 patient U.S. REFRESH 2-AKI pivotal randomized, controlled trial is currently at 109 patients with 24 active sites
The German government-funded REMOVE endocarditis trial is approximately 90% enrolled, with 222 patients out of a target 250 patients at 15 clinical sites. Completed enrollment is expected by year-end
The HemoDefend-RBC program remains on track for an investigational device exemption (IDE) submission to the FDA this year, enabling the start of the U.S. pivotal trial
Dr. Phillip Chan, Chief Executive Officer of CytoSorbents Corporation, stated, "Q2 2019 product sales rebounded significantly over the past quarter, achieving our guidance as the best quarter of product sales since we began commercialization, and in-line with our historical product sales trajectory of growth. Results were aided by the resumption of ordering from one of our major distributors. We also attained blended product gross margins of 76%, an increase of 200 basis points from the prior quarter, primarily resulting from continued efficiencies in manufacturing, and closing in on our goal of 80% on a quarterly basis this year."

"The new loan amendment with Bridge Bank has expanded our effective working capital position by approximately $9 million, including deferment of principal repayments, and will allow us to continue making the investments in our business that will drive future growth. We expect that product sales in the second half of this year will solidly exceed product sales in the first half, catalyzed by the impact of our expanding sales team, organic growth in existing markets, increased international expansion, publication of new clinical data in a wide variety of clinical applications, and increased partner support."

Dr. Chan concluded, "Lastly, in the independent editorial commentary to the REFRESH I paper that was recently published, we were pleased to note that the authors described the complications of extended cardiopulmonary bypass – including the activation of inflammation, release of free hemoglobin due to hemolysis, and organ injury and failure – as the ‘Achilles heel of complex cardiac surgery’, and that the ‘holy grail of research in this subject would be to find something able to mitigate or eliminate the mediators responsible for these potentially catastrophic downstream effects of prolonged cardiopulmonary bypass.’ In reality, CytoSorb has demonstrated utility in so many different life-or-death applications in critical care and cardiac surgery, that we believe we are one of the leading innovators in bringing game-changing advances to medicine."

"Please join us on our earnings conference call today, details for which are below."

Conference Call Details:
Date: Tuesday, August 6, 2019
Time: 4:45 PM Eastern Time
Participant Dial-In: 877-451-6152
Conference ID: 13692360
Live Presentation Webcast: View Source

It is recommended that participants dial in approximately 10 minutes prior to the start of the call. There will also be a simultaneous live webcast of the conference call that can be accessed through the following audio feed link: View Source

An archived recording of the conference call will be available under the Investor Relations section of the Company’s website at View Source

Results of Operations

Comparison for the three months ended June 30, 2019 and 2018:

Revenues:

Revenue from product sales was approximately $5,850,000 in the three months ended June 30, 2019, as compared to approximately $5,246,000 in the three months ended June 30, 2018, an increase of approximately $604,000, or 12%. This increase was driven by an increase in direct sales of approximately $520,000 resulting from sales to both new customers and repeat orders from existing customers and an increase in distributor sales of approximately $84,000. In addition, sales were negatively impacted by approximately $357,000 as a result of the decrease in the average exchange rate of the Euro to the U.S. dollar. For the three months ended June 30, 2019, the average exchange rate of the Euro to the U.S. dollar was $1.12 as compared to an average exchange rate of $1.19 for the three months ended June 30, 2018.

Grant income was approximately $382,000 for the three months ended June 30, 2019 as compared to approximately $510,000 for the three months ended June 30, 2018, a decrease of approximately $128,000 or 25%. This decrease was a result of timing of certain grant revenue.

Total revenues were approximately $6,233,000 for the three months ended June 30, 2019, as compared to total revenues of approximately $5,755,000 for the three months ended June 30, 2018, an increase of approximately $478,000, or 8%.

Cost of Revenues:

For the three months ended June 30, 2019 and 2018, cost of revenue was approximately $1,834,000 and $1,786,000, respectively, an increase of approximately $48,000. Product cost of revenues increased approximately $31,000 during the three months ended June 30, 2019 as compared to the three months ended June 30, 2018 due to increased sales. Product gross margins were approximately 76% for the three months ended June 30, 2019 and approximately 74% for the three months ended June 30, 2018.

Research and Development Expenses:

For the three months ended June 30, 2019, research and development expenses were approximately $2,930,000 as compared to research and development expenses of approximately $1,576,000 for the three months ended June 30, 2018. The increase of approximately $1,354,000 was due to an increase in clinical trial costs of approximately $1,215,000, which is primarily related to our REFRESH 2-AKI trial, an increase in non-clinical research and development salary related costs of approximately $11,000 and an increase in other non-clinical research and development costs of approximately $150,000. These increases were offset by an increase in direct labor and other costs being deployed toward grant-funded activities of approximately $18,000, which had the effect of decreasing the amount of our non-reimbursable research and development costs, and a decrease in new product development costs of approximately $4,000.

Legal, Financial and Other Consulting Expense:

Legal, financial and other consulting expenses were approximately $592,000 for the three months ended June 30, 2019, as compared to approximately $458,000 for the three months ended June 30, 2018. The increase of approximately $134,000 was due to an increase in legal fees of approximately $28,000 related to patent matters and certain corporate initiatives, an increase in consulting fees of approximately $55,000, an increase in employment agency fees of approximately $31,000 and an increase in accounting fees of approximately $20,000.

Selling, General and Administrative Expense:

Selling, general and administrative expenses were approximately $4,507,000 for the three months ended June 30, 2019, as compared to approximately $6,124,000 for the three months ending June 30, 2018, a decrease of $1,617,000. The decrease of $1,617,000 was due to a decrease in non-cash stock compensation of approximately $2,228,000, a decrease in travel and entertainment costs of approximately $64,000 and a decrease in other general and administrative expenses of approximately $70,000. These decreases were offset by an increase in salaries, commissions and related costs of approximately $588,000, additional sales and marketing costs, which include advertising and conferences of approximately $92,000, an increase in royalty expenses of approximately $53,000 due to the increase in product sales and an increase in rent expense of approximately $12,000 related to the expansion of manufacturing and office facilities.

Interest Expense, net:

For the three months ended June 30, 2019, interest expense was approximately $215,000, as compared to interest expense of approximately $840,000 for the three months ended June 30, 2018. This decrease in interest expense of approximately $625,000 was primarily a result of the settlement of the Success Fee with Bridge Bank in the amount of $637,000 that became due in May 2018 in accordance with the terms of the 2016 Success Fee Letter with Bridge Bank.

Gain (Loss) on Foreign Currency Transactions:

For the three months ended June 30, 2019, the gain on foreign currency transactions was approximately $297,000 as compared to a loss of approximately $(794,000) for the three months ended June 30, 2018. The 2019 gain was directly related to the increase in the spot exchange rate of the Euro to the U.S. dollar at June 30, 2019 as compared to March 31, 2018. The spot exchange rate of the Euro to the U.S. dollar was $1.14 per Euro at June 30, 2019, as compared to $1.12 per Euro at March 31, 2019. The 2018 loss was directly related to the decrease in the spot exchange rate of the Euro at June 30, 2018 as compared to March 31, 2018. The spot exchange rate of the Euro to the U.S. dollar was $1.17 per Euro at June 30, 2018, as compared to $1.23 per Euro at March 31, 2018.

Comparison for the six months ended June 30, 2019 and 2018:

Revenues:

Revenue from product sales was approximately $10,427,000 in the six months ended June 30, 2019, as compared to approximately $9,679,000 in the six months ended June 30, 2018, an increase of approximately $748,000, or 8%. This increase was driven by an increase in direct sales of approximately $1,160,000 resulting from sales to both new customers and repeat orders from existing customers, offset by a decrease in distributor sales of approximately $412,000. In addition, sales were negatively impacted by approximately $737,000 as a result of the decrease in the average exchange rate of the Euro to the U.S. dollar. For the six months ended June 30, 2019, the average exchange rate of the Euro to the U.S. dollar was $1.13 as compared to an average exchange rate of $1.21 for the six months ended June 30, 2018.

Grant income was approximately $997,000 for the six months ended June 30, 2019 as compared to approximately $1,001,000 for the six months ended June 30, 2018, a decrease of approximately $4,000 or less than 1%.

Total revenues were approximately $11,424,000 for the six months ended June 30, 2019, as compared to total revenues of approximately $10,680,000 for the six months ended June 30, 2018, an increase of approximately $744,000, or 7%.

Cost of Revenues:

For the six months ended June 30, 2019 and 2018, cost of revenue was approximately $3,573,000 and $3,353,000, respectively, an increase of approximately $220,000. Product cost of revenues increased approximately $83,000 during the six months ended June 30, 2019 as compared to the six months ended June 30, 2018 due to increased sales. Product gross margins were approximately 75% for the six months ended June 30, 2019 and approximately 74% for the six months ended June 30, 2018.

Research and Development Expenses:

For the six months ended June 30, 2019, research and development expenses were approximately $5,348,000 as compared to research and development expenses of approximately $3,356,000 for the six months ended June 30, 2018. The increase of approximately $1,992,000 was due to increase in clinical trial costs of approximately $1,993,000, which is primarily related to our REFRESH 2-AKI trial, an increase in non-clinical research and development salary related costs of approximately $20,000 and an increase in other non-clinical research and development costs of approximately $186,000. These increases were offset by an increase in direct labor and other costs being deployed toward grant-funded activities of approximately $136,000, which had the effect of decreasing the amount of our non-reimbursable research and development costs, and a decrease in new product development costs of approximately $71,000.

Legal, Financial and Other Consulting Expense:

Legal, financial and other consulting expenses were approximately $1,154,000 for the six months ended June 30, 2019, as compared to approximately $874,000 for the six months ended June 30, 2018. The increase of approximately $280,000 was due to an increase in legal fees of approximately $194,000 related to patent matters and certain corporate initiatives, an increase in consulting fees of approximately $73,000, and an increase in accounting fees of approximately $17,000. These increases were offset by a decrease in employment agency fees of approximately $4,000.

Selling, General and Administrative Expense:

Selling, general and administrative expenses were approximately $9,265,000 for the six months ended June 30, 2019, as compared to approximately $10,385,000 for the six months ending June 30, 2018. The decrease of $1,120,000 was due to a decrease in non-cash stock compensation of approximately $2,426,000 and other general and administrative expenses of approximately $140,000. These decreases were offset by an increase in salaries, commissions and related costs of approximately $1,142,000, an increase in travel and entertainment and other costs of approximately $33,000, additional sales and marketing costs, which include advertising and conferences of approximately $189,000, an increase in royalty expenses of approximately $60,000 due to the increase in product sales and an increase in rent expense of approximately $22,000 related to the expansion of manufacturing and office facilities.

Interest Expense, net:

For the six months ended June 30, 2019, interest expense was approximately $420,000, as compared to interest expense of approximately $1,079,000 for the six months ended June 30, 2018. This decrease in interest expense of approximately $659,000 was primarily a result of the settlement of the Success Fee with Bridge Bank in the amount of $637,000 that became due in May 2018 in accordance with the terms of the 2016 Success Fee Letter with Bridge Bank and interest earned on our cash balances during the six months ended June 30, 2019.

Gain (Loss) on Foreign Currency Transactions:

For the six months ended June 30, 2019, the loss on foreign currency transactions was approximately $96,000 as compared to a loss of approximately $435,000 for the six months ended June 30, 2018. The 2019 loss was directly related to the decrease in the spot exchange rate of the Euro to the U.S. dollar at June 30, 2019 as compared to December 31, 2018. The spot exchange rate of the Euro to the U.S. dollar was $1.14 per Euro at June 30, 2019, as compared to $1.15 per Euro at December 31, 2018. The 2018 loss was directly related to the decrease in the spot exchange rate of the Euro at June 30, 2018 as compared to the December 31, 2017. The spot exchange rate of the Euro to the U.S. dollar was $1.17 per Euro at June 30, 2018, as compared to $1.20 per Euro at December 31, 2017.

History of Operating Losses:

We have experienced substantial operating losses since inception. As of June 30, 2019, we had an accumulated deficit of approximately $177,955,000, which included losses of approximately $8,431,000 and $8,803,000 for the six-month periods ended June 30, 2019 and 2018, respectively. Historically, losses have resulted principally from costs incurred in the research and development of our polymer technology, clinical studies, and general and administrative expenses.

Liquidity and Capital Resources:

Since inception, our operations have been primarily financed through the issuance of debt and equity securities. At June 30, 2019, we had current assets of approximately $22,214,000 including cash on hand of approximately $16,342,000 and current liabilities of approximately $6,919,000. On July 31, 2019, the Company executed an Amendment to its Loan Agreement with Bridge Bank and simultaneous with this Amendment received $5 million in proceeds from an additional term loan. In addition, the Amendment extends the interest only period of the loan for an additional six months through April 2020, with the ability to further extend the interest-only period for another six months through October 2020 should the Company meet certain conditions.

We believe that we have sufficient cash to fund our operations into 2020.

2019 Third Quarter Revenue Guidance:

CytoSorbents has not historically given specific financial guidance on quarterly results until the quarter has been completed. However, we expect our third quarter 2019 product sales will exceed sales reported in the third quarter of 2018. In addition, we expect that second half 2019 product sales will exceed first half 2019 product sales.

For additional information, please see the Company’s Annual Report on Form 10-K for the year ended December 31, 2018 filed on March 7, 2019 on View Source

On August 6, 2019 Alder BioPharmaceuticals, Inc. (NASDAQ: ALDR), a biopharmaceutical company focused on developing novel therapeutic antibodies for the treatment of migraine, reported its financial results for the second quarter ended June 30, 2019 (Press release, Alder Biopharmaceuticals, AUG 6, 2019, View Source [SID1234538253]).

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"We continue to make significant progress toward the potential launch of eptinezumab in the U.S. in the first quarter of 2020. We have our commercial leadership team in place, with experienced personnel leading sales, marketing and market access. The team is sharpening our go-to-market strategy and expects to complete our commercial footprint later this year. We recently presented additional data further confirming eptinezumab’s differentiated clinical profile and benefits in patient-reported outcomes at the 2019 American Headache Society and American Academy of Neurology Annual Meetings," said Bob Azelby, Alder’s president and chief executive officer. "Looking forward, we believe there is a large opportunity to build value at Alder in the latter half of the year, with the initiation of eptinezumab’s acute study and the advancement of our product candidate, ALD1910, into the clinic. These collective activities align with our mission to forever change the migraine treatment landscape and give people with migraines their lives back."

Second Quarter 2019 Highlights and Recent Developments

Commercial readiness activities ongoing under the leadership of recently appointed chief commercial officer, Nadia Dac: As Alder prepares for the potential commercial launch of eptinezumab in the U.S. in the first quarter of 2020, the company has conducted new market research that further confirms eptinezumab’s differentiated profile and is prepared to begin account-level engagement with payers. The company plans to focus its marketing efforts on the high-prescribing headache specialist population and accounts, where eptinezumab fits well into their treatment protocols of quarterly patient visits with its short, 30-minute IV administration and its well-tolerated safety profile, as demonstrated in the company’s clinical trials. The company plans to utilize a specialty sales organization sized between 75 and 100 sales representatives.

New migraine-free months, migraine severity and quality of life data presented at AHS Annual Meeting: In July 2019, Alder presented new data from post-hoc analyses from its PROMISE 1 and PROMISE 2 Phase 3 clinical trials for eptinezumab at the American Headache Society’s (AHS) 61st Annual Scientific Meeting in Philadelphia, PA from July 11-14, 2019. Key highlights from the new data presented show 18.1% of episodic migraine patients treated with 100 mg of eptinezumab experienced no migraine days for at least half of the study period (≥six months), compared with 12.6% of placebo-treated patients, and 14.0% of chronic migraine patients treated with 100 mg of eptinezumab experienced no migraine days for at least half of the study period (≥three months), compared with 4.9% of placebo-treated patients. Data presented from PROMISE 2 also showed consistent clinically significant improvements in migraine severity in chronic migraine patients, a large contributor to impact on quality of life, starting at Month 1. Eptinezumab treatment resulted in clinically meaningful improvements in Headache Impact Test (HIT-6) scores in chronic migraine patients as early as Month 1 after treatment, which were maintained or further improved throughout the six-month study period, compared to placebo, which did not achieve a clinically meaningful improvement until Month 6. In addition, eptinezumab treatment resulted in clinically meaningful improvements greater than placebo in the 36-item Short-Form Health Survey (SF-36) scores in chronic migraine patients as early as Month 1 after treatment and through the six-month study period. From a safety perspective, longer-term exposure has demonstrated no change in the overall safety profile for eptinezumab.

Data showing consistency of rapid onset of migraine prevention and improvements in most bothersome migraine symptoms and patient reported outcomes data presented at AAN Annual Meeting: In May 2019, Alder presented efficacy data highlighting the consistency of the rapid onset of migraine prevention across four clinical trials with eptinezumab at the 71st American Academy of Neurology (AAN) Annual Meeting in Philadelphia, PA from May 4-10, 2019. Across the Phase 2 and Phase 3 clinical trials, it was observed that eptinezumab, with its 100% bioavailability at the end of infusion, showed a rapid onset of migraine prevention. The rapid response observed on both Day 1 and through Month 1 in PROMISE 1 and PROMISE 2 was also sustained through the first quarter following a single eptinezumab infusion, and was maintained or further increased through subsequent infusions. Alder also presented a new analysis of patient-reported outcomes data from the PROMISE 2 Phase 3 clinical trial of eptinezumab for the prevention of chronic migraine, showing improvements in the most bothersome migraine symptoms and patients’ global impression of change in their migraine status by Month 1 after treatment, with improvements sustained in overall response through the first and second quarterly infusions.

Upcoming Anticipated Milestones

Eptinezumab PDUFA target action date set for early 2020: The U.S. Food and Drug Administration (FDA) accepted the Biologics License Application (BLA) filing for eptinezumab in April 2019, and set a Prescription Drug User Fee Act (PDUFA) target action date of February 21, 2020. If approved, eptinezumab will be the first-to-market IV therapy for migraine prevention, providing rapid and sustained prevention that begins on Day 1.

Acute study for eptinezumab to begin in 2H 2019: Alder plans to initiate a Phase 3 clinical trial evaluating eptinezumab as a treatment for acute migraine in patients who are candidates for prevention therapy in the second half of 2019. The trial will seek to leverage eptinezumab’s 100% bioavailability and rapid onset of prevention demonstrated in clinical testing, with the objective of securing an indication for the acute treatment of migraine for patients and positioning eptinezumab as the first anti-CGRP monoclonal antibody for both the treatment and prevention of migraine, if approved for both these indications.

ALD1910 to enter clinical development in 2019: Alder continues to advance its preclinical candidate, ALD1910, a monoclonal antibody targeting PACAP (pituitary adenylate cyclase-activating peptide) for migraine prevention. ALD1910 is currently undergoing Investigational New Drug (IND)-enabling preclinical studies. Alder expects to initiate a first in-human clinical study by the end of 2019.

Second Quarter 2019 Financial Results

As of June 30, 2019, Alder had $440.7 million in cash, cash equivalents, investments and restricted cash, compared to $412.4 million as of December 31, 2018.

Research and development expenses for the second quarter ended June 30, 2019 totaled $34.1 million, compared to $52.8 million for the same period in 2018. The year-over-year decrease was primarily due to a decrease in process development costs and a decrease in clinical trial costs due to the completion of patient treatments for several of the company’s clinical trials.

General and administrative expenses for the second quarter ended June 30, 2019 totaled $21.6 million, compared to $11.9 million for the same period in 2018. The year-over-year increase reflects efforts to support commercial readiness activities for eptinezumab.

Net loss applicable to common stockholders for the second quarter ended June 30, 2019 totaled $59.9 million, or $0.72 per share, compared to net loss of $70.7 million, or $1.04 per share on a fully-diluted basis, for the same period in 2018.

Financial Outlook

Alder continues to expect that full-year 2019 net cash used in operating activities and purchases of property and equipment will be in the range of $285 to $315 million. The majority of the spend is focused on ensuring that Alder is prepared for the potential launch of eptinezumab in the first quarter of 2020, including advancing eptinezumab’s supply chain, building commercial inventory, continuing to build out Alder’s commercial footprint and other pre-launch market readiness activities.

Alder believes its available cash, cash equivalents, investments and restricted cash will be sufficient to meet its projected operating requirements through the anticipated launch of eptinezumab and into the latter part of 2020.

Conference Call and Webcast

Alder will host a conference call today at 5:00 p.m. ET to discuss these financial results and recent corporate highlights. The live call may be accessed by dialing (877) 430-4657 for domestic callers or (484) 756-4339 for international callers, and providing conference ID number 6561825. The webcast will be broadcast live and can be accessed from the Events & Presentations page in the Investors section of Alder’s website at www.alderbio.com. The webcast will be available for replay following the call for at least 30 days.