On November 8, 2018 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the third quarter 2018 and provided an overview of recent accomplishments for selinexor, its lead, novel, oral SINE compound, and its other pipeline programs (Press release, Karyopharm, NOV 8, 2018, View Source [SID1234531001]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continued to make tremendous progress towards bringing selinexor, our lead drug candidate, to patients with highly refractory multiple myeloma. Most notably, the U.S. FDA has now accepted our New Drug Application (NDA), granting it a Priority Review with an action date of April 6, 2019, under the Prescription Drug User-Fee Act (PDUFA)," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "In this NDA, we are requesting accelerated approval for selinexor as a new treatment for patients with penta-refractory multiple myeloma. If selinexor is approved, we believe that its novel mechanism of action and oral administration, along with its compelling clinical profile, will make it a meaningful treatment option for patients with highly refractory myeloma. Additionally, we continue to believe selinexor holds broad utility beyond highly refractory myeloma. We recently received Fast Track Designation for selinexor in relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and were also very pleased to learn that the top-line results from the Phase 2b SADAL study in patients with relapsed or refractory DLBCL have been selected for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 1, 2018. Finally, by the end of 2018, we remain on track to complete enrollment in the pivotal Phase 3 BOSTON study evaluating selinexor in combination with once weekly Velcade and low-dose dexamethasone versus standard twice weekly Velcade – dexamethasone in patients with multiple myeloma who have had one to three prior lines of therapy."

"As we await the upcoming FDA review decision for selinexor, we are building our U.S. commercial capabilities, which will include hiring our U.S. sales force in early 2019. In order to strengthen our financial position, we recently increased our cash position through a private offering of convertible senior notes resulting in net proceeds of approximately $166.9 million which helps extend our estimated cash runway into the second quarter of 2020. Additionally, our future capital needs could potentially be partially offset with cash generated from sales of selinexor following commercialization, which could come as early as the second quarter of 2019, pending FDA approval," concluded Dr. Kauffman.

Third Quarter 2018 and Recent Events

Selinexor in Multiple Myeloma

U.S. FDA Accepts Selinexor NDA and Grants Priority Review. The U.S. FDA accepted for filing with Priority Review Karyopharm’s NDA seeking accelerated approval for selinexor, its first in class, oral SINE compound, as a new treatment for patients with penta-refractory multiple myeloma. The FDA also assigned an action date of April 6, 2019 under the PDUFA. Provided marketing approval is granted by the FDA, Karyopharm plans to commercialize selinexor in the U.S. in the first half of 2019. The Company also plans to submit a Marketing Authorization Application to the European Medicines Agency (EMA) in early 2019 with a request for conditional approval.

Phase 2b STORM Data Selected for Oral Presentation at ASH (Free ASH Whitepaper) 2018. Additional results from Part 2 of the Phase 2b STORM study have been selected for oral presentation at the upcoming ASH (Free ASH Whitepaper) 2018 Annual Meeting in early December. Karyopharm previously reported results from the Phase 2b STORM study evaluating selinexor plus low dose dexamethasone (Sd) in patients with penta-refractory multiple myeloma in September at the Society of Hematologic Oncology (SOHO) 2018 Annual Meeting. For the STORM study’s primary objective, the overall response rate (ORR) was 26.2%, which included two stringent complete responses (sCRs), six very good partial responses (VGPRs) and 24 partial responses (PRs) in these patients with penta-refractory myeloma. The two sCRs were negative for minimal residual disease, one at the level of 1×10-6 and one at 1×10-4; this is particularly significant in this penta-refractory population. The ORR for Sd in patients who had previously received Darzalex combination therapy (n=86) was 29.1%. The Disease Control Rate for patients who had achieved stable disease or better was 78.6%. All responses were confirmed by an Independent Review Committee. Median progression-free survival (PFS) was 3.7 months and the median duration of response (DOR) was 4.4 months (range <1 to 9.9 months). Median overall survival (OS) across the study was 8.6 months. Median OS in the approximately 40% of patients with at least a minimal response (MR) on Sd was 15.6 months compared to a median OS of 1.7 months in patients whose disease progressed or was not evaluable (p<0.0001). The short median OS of patients with no response to selinexor is consistent with the lack of available effective therapies for the very heavily pretreated population who entered the study. Across the relevant patient population, side effects of oral selinexor were generally predictable and manageable with dose adjustments and/or supportive care, with safety results that were consistent with those previously reported from Part 1 of this STORM study (Vogl et al., J Clin Oncol, 2018) and from other selinexor studies.

Two Phase 1b/2 STOMP Abstracts Selected for Presentation at ASH (Free ASH Whitepaper) 2018. Two abstracts featuring clinical data from two treatment arms of the ongoing Phase 1b/2 STOMP study in patients with relapsed or refractory multiple myeloma have been selected for oral and poster presentations at ASH (Free ASH Whitepaper) 2018. The oral presentation will highlight data from the arm evaluating selinexor in combination with Darzalex (daratumumab) and low-dose dexamethasone (SDd). The poster presentation will provide updated data from the arm evaluating selinexor in combination with Pomalyst (pomalidomide) and low-dose dexamethasone (SPd). In data reported previously from these arms, selinexor has demonstrated evidence of additive or synergistic anti-myeloma activity when combined with these standard approved therapies.

Pivotal Phase 3 BOSTON Study in Progress. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is underway and enrolling patients globally. BOSTON is evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (once weekly) and low dose dexamethasone (SVd), compared to standard twice weekly Velcade and low dose dexamethasone (Vd) in patients with multiple myeloma who have had one to three prior lines of therapy. The primary endpoints of the study are PFS and ORR. Data from the BOSTON study, if positive, would be used to support regulatory submissions to the FDA and EMA requesting full approvals for use of selinexor in second line multiple myeloma, following the Company’s requests for accelerated and conditional approvals, respectively, using data from the Phase 2b STORM study. The Company expects to enroll approximately 360 patients at over 100 clinical sites internationally and expects to complete enrollment by the end of 2018, with top-line data anticipated at the end of 2019.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)

Received Fast Track Designation from FDA for the Treatment of Patients with Relapsed or Refractory DLBCL. In addition to Orphan Drug Designation, selinexor was recently granted Fast Track designation by the FDA for the treatment of patients with relapsed or refractory DLBCL.

Phase 2b SADAL Data in DLBCL Selected for Presentation at ASH (Free ASH Whitepaper) 2018. An abstract featuring data from the fully enrolled Phase 2b SADAL study has been selected for poster presentation at ASH (Free ASH Whitepaper) 2018. The SADAL study is designed to evaluate single agent oral selinexor 60mg for patients with relapsed or refractory DLBCL who are not eligible for stem cell transplantation. The SADAL study has enrolled approximately 125 patients with DLBCL who received two to five lines of prior therapy at single-agent selinexor dosed 60mg twice weekly in patients. Assuming the results from the SADAL study are positive, Karyopharm plans to submit an NDA to the FDA with a request for accelerated approval, and a Marketing Authorization Application (MAA) to the EMA with a request for conditional approval, for oral selinexor in this relapsed or refractory DLBCL patient population.
Selinexor in Solid Tumors

Ongoing Phase 3 Portion of the Phase 2/3 SEAL Study in Liposarcoma. Karyopharm previously reported results from the successful Phase 2 portion of the blinded, randomized Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with previously treated, advanced unresectable dedifferentiated liposarcoma. Enrollment and dosing is currently ongoing in the Phase 3 portion of the SEAL study and, assuming a positive outcome on the primary end point of PFS, the Company intends to use the data from the SEAL study to support an NDA and an MAA submission requesting full approval for oral selinexor for patients with advanced unresectable dedifferentiated liposarcoma. Top-line data from the Phase 3 portion of the SEAL study are anticipated by the end of 2019.

Ongoing Investigator Sponsored Phase 2/3 Trial as Maintenance Therapy in Endometrial Cancer. A randomized Phase 2/3 study of selinexor versus placebo as maintenance therapy in patients with one or two prior platinum-based treatments for advanced endometrial cancer, led by Dr. Ignace Vergote, Head of the Department of Obstetrics and Gynaecology and Gynaecologic Oncology at the Catholic University of Leuven, Belgium, is currently ongoing. In the U.S., endometrial cancer is the most common gynecological cancer with approximately 58,000 cases expected to be diagnosed and an estimated 10,000 women expected to die from this cancer in 20181, revealing a meaningful patient population in need of novel therapies.
Eltanexor

Phase 1/2 Eltanexor Data in Metastatic Colorectal Cancer (mCRC) Presented at ESMO (Free ESMO Whitepaper) 2018. At the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress in October, John Hays. MD, PhD, Ohio State University, Comprehensive Cancer Center, presented preliminary results from the ongoing Phase 1/2 investigator-sponsored study investigating eltanexor in patients with heavily pre-treated (median of 4 prior treatment regimens) metastatic colorectal cancer (mCRC). The presented results showed that 37% of patients experienced disease control at ≥8 weeks on eltanexor and the median preliminary progression free survival (PFS) for all patients in the 30 mg cohort was 3.5 months. Eltanexor was generally well tolerated with manageable adverse events. The highest observed treatment-related grade ≥3 adverse events were hyponatremia (23%), fatigue (20%) and anemia (20%). Karyopharm is encouraged by these preliminary results which demonstrated promising efficacy with a median PFS longer than currently available third line therapies and an acceptable safety and tolerability profile.
Other ASH (Free ASH Whitepaper) 2018 Highlights

Several Investigator-sponsored Trials and Preclinical Abstracts Selected for Presentation at ASH (Free ASH Whitepaper) 2018. Three abstracts featuring clinical data from investigator-sponsored clinical studies evaluating selinexor either as a single-agent or in combination with other anti-cancer agents for the treatment of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and pediatric leukemia have been selected for oral or poster presentations at ASH (Free ASH Whitepaper) 2018. Three abstracts describing preclinical research exploring the use of selinexor in models of multiple myeloma and AML have also been selected for poster presentations at the meeting. One abstract featuring preclinical research investigating the use of KPT-9274, Karyopharm’s oral dual inhibitor of PAK4 and NAMPT, for the treatment of AML, has also been selected for a poster presentation. A complete list of the ASH (Free ASH Whitepaper) 2018 abstracts can be accessed here.
Corporate Updates

Carsten Thiel, Ph.D. Appointed to the Board. Karyopharm announced the appointment of Carsten Thiel, Ph.D., to its Board of Directors. Dr. Thiel is currently Chief Executive Officer of Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies. Prior to joining Abeona, Dr. Thiel served as the Executive Vice President and Chief Commercial Officer of Alexion Pharmaceuticals, Inc., a leading global biopharmaceutical company focused on serving patients affected by rare diseases.
Third Quarter Ended September 30, 2018 Financial Results

Cash, cash equivalents and investments as of September 30, 2018, including restricted cash, totaled $212.3 million, compared to $176.4 million as of December 31, 2017.

On October 26, 2018, Karyopharm completed a private offering securing a $172.5 million aggregate principal amount of 3.00% convertible senior notes due in 2025, including the full exercise of the initial purchasers’ option to purchase additional notes. After deducting the initial purchasers’ discounts and commissions and other offering expenses the net proceeds are estimated to be $166.9 million.

For the quarter ended September 30, 2018, research and development expense was $36.4 million compared to $25.2 million for the quarter ended September 30, 2017. For the quarter ended September 30, 2018, general and administrative expense was $13.0 million compared to $5.8 million for the quarter ended September 30, 2017.

Karyopharm reported a net loss of $48.1 million, or $0.79 per share, for the quarter ended September 30, 2018, compared to a net loss of $30.6 million, or $0.65 per share, for the quarter ended September 30, 2017. Net loss includes stock-based compensation expense of $4.8 million and $4.9 million for the quarters ended September 30, 2018 and September 30, 2017, respectively.

Financial Outlook

Karyopharm expects its operating cash burn, including research and development and general and administrative expenses, for the year ending December 31, 2018 to be in the range of $175 to $185 million. Following Karyopharm’s private placement of convertible senior notes during October 2018, and based on its current operating plans, Karyopharm expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations into the second quarter of 2020. This estimate does not account for any cash generated from product sales that the Company expects to generate from the commercial launch of selinexor, which could come as early as the second quarter 2019, if selinexor is approved by the FDA’s assigned PDUFA date. Karyopharm’s future capital needs could potentially be partially offset by cash generated from these sales of selinexor.

Karyopharm’s current operating plans include the continued clinical development of selinexor in the Company’s lead indications and on preparing the commercial infrastructure and hiring a sales force for the potential launch of selinexor in the U.S. Additional key activities expected in 2018 include preparing for a potential MAA submission to the EMA requesting conditional approval for selinexor in multiple myeloma, topline data from the SADAL study and completion of enrollment in the Phase 3 BOSTON study.

Further Information About Potential Accelerated Approval for Selinexor in Multiple Myeloma

The FDA instituted its Accelerated Approval Program to allow for expedited approval of drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint or an intermediate clinical endpoint thought to predict clinical benefit, like overall response rate (ORR). Accelerated approval is available only for drugs that provide a meaningful therapeutic benefit over existing treatments at the time of consideration of the application for accelerated approval, which the FDA has reiterated in its feedback to the Company. Particularly in disease areas with multiple available and potential new therapies, such as multiple myeloma, accelerated approval carries a high regulatory threshold. Consistent with its general guidance, the FDA has noted to the Company its preference for randomized studies geared toward full approval, which the Company has undertaken with the ongoing pivotal, Phase 3 BOSTON study, and has reminded the Company that accelerated approval requires patients to have exhausted all available approved therapies. FDA’s Fast Track designation is available to therapeutics treating an unmet medical need in a serious condition; the Company has received Fast Track designation from the FDA specifically for the population treated in the STORM trial. In light of this recognition that the STORM patient population represents an unmet medical need and the positive data reported in April and September 2018, the Company believes that the STORM study should support its request to the FDA for accelerated approval.

Conference Call Information

Karyopharm will host a conference call today, Thursday, November 8, 2018, at 8:30 a.m. Eastern Time, to discuss the third quarter 2018 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 7946498. 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.

On November 8, 2018 OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing intratumoral cancer immunotherapies, reported the appointment of Robert Ward to its Board of Directors. Mr. Ward is currently Chairman and Chief Executive Officer of Eloxx Pharmaceuticals (Press release, OncoSec Medical, NOV 8, 2018, View Source [SID1234530999]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"It is with great pleasure that we welcome Bob to our Board. His considerable industry experience leading pharmaceutical companies in bringing new therapies to market and deep insight into successful commercialization strategies will be extremely valuable to OncoSec as we continue to advance our TAVO platform," said Avtar Dhillon, Chairman of OncoSec.

Mr. Ward brings to OncoSec over 30 years of experience as a global biopharmaceutical industry leader with experience across the full spectrum of drug development and commercialization. Mr. Ward currently serves as Chairman and Chief Executive Officer of Eloxx Pharmaceuticals. Prior to joining Eloxx, Mr. Ward was President and Chief Executive Officer of Radius Health, Inc., where he helped the company successfully complete its initial public offering and lead the development, approval and launch of the company’s TYMLOS injection. Prior to joining Radius, Mr. Ward was Vice President for Strategy and External Alliances for the New Opportunities iMed of Astra Zeneca. He has held a series of progressive management and executive roles with established companies such as NPS Pharmaceuticals, Schering-Plough (Merck), Pharmacia (Pfizer), Bristol-Myers Squibb and Genentech. Mr. Ward currently serves as a Director of the Massachusetts High Technology Council and had served as a Director of Akari Therapeutics from October 2016 to August 2018.

Mr. Ward received a B.A. in Biology and a B.S. in Physiological Psychology, both from the University of California, Santa Barbara, an M.S. in Management from the New Jersey Institute of Technology and an M.A. in Immunology from the John Hopkins University School of Medicine.

On November 8, 2018 Cardinal Health (NYSE: CAH) reported first quarter fiscal year 2019 revenues of $35.2 billion, an increase of 8 percent (Press release, Cardinal Health, NOV 8, 2018, View Source [SID1234530990]). The company also reported growth in GAAP operating earnings of 211 percent to $816 million and a decrease in non-GAAP operating earnings of 11 percent to $542 million. GAAP operating earnings included a gain of $508 million ($378 million after-tax) related to the naviHealth divestiture. GAAP diluted earnings per share (EPS) from continuing operations increased 439 percent to $1.94, while non-GAAP diluted EPS increased 18 percent to $1.29.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased that the first quarter provided a solid start to fiscal year 2019," said Mike Kaufmann, CEO of Cardinal Health. "Operating performance came in as expected, and we are on track in executing on our strategic initiatives to deliver increased value to our shareholders, our customers and their patients. We look forward to making further strides over the balance of the year and building on Cardinal Health’s essential role in healthcare."

Tax rate

During the three months ended September 30, 2018 and 2017, GAAP effective tax rates were 19.4 percent and 34.2 percent, respectively, and non-GAAP effective tax rates were 14.0 percent and 34.1 percent, respectively.

This quarter’s lower effective tax rates are attributable to the lower U.S. federal income tax rate due to the U.S. Tax Cuts and Jobs Act, and the approximately $0.18 per share positive impact of discrete tax benefits primarily related to international legal entity changes.

Segment results
Pharmaceutical segment
First quarter revenue for the Pharmaceutical segment increased 9 percent to $31.4 billion due to sales growth from Pharmaceutical Distribution and Specialty Solutions customers, partially offset by the divestiture of the company’s China distribution business.

Fiscal year 2019 outlook
The company does not provide GAAP EPS outlook because it is unable to reliably forecast most of the items that are excluded from GAAP EPS to calculate non-GAAP EPS. These items could cause EPS to differ materially from non-GAAP EPS. See "Use of Non-GAAP Measures" following the attached schedules for additional explanation.

The company reaffirmed its fiscal year 2019 guidance range for non-GAAP diluted EPS of $4.90 to $5.15.

Additional first-quarter and recent highlights

Announced Victor Crawford will join the company as chief executive officer of the Pharmaceutical segment on November 12

Exited transition services agreement (TSA) with Medtronic for the Europe, Middle East and Africa region during the last week of October and on track for TSA exits in the remaining regions in early 2019

The Cardinal Health board of directors approved a quarterly dividend of $0.4763 per share. The dividend will be payable on January 15, 2019 to shareholders of record at the close of business on January 2, 2019

Recently completed a $600 million share repurchase program, and this week, the board of directors approved a three-year authorization to repurchase up to an additional $1 billion of Cardinal Health common shares, which will expire on December 31, 2021. The company is now authorized to repurchase up to $1.3 billion of its common shares

Sponsored inaugural Women Pharmacist Day on October 12, in conjunction with National Pharmacist Month, to recognize the important contributions women pharmacists make to delivering quality care to patients

Webcast

Cardinal Health will host a webcast today at 8:30 a.m. Eastern to discuss first quarter results. To access the webcast and corresponding slide presentation, go to the Investor Relations page at ir.cardinalhealth.com. No access code is required.

Presentation slides and a webcast replay will be available on the Cardinal Health website at ir.cardinalhealth.com until November 7, 2019.

Upcoming webcasted investor events

Credit Suisse 27th Annual Healthcare Conference on November 14 at 8:35 a.m. Mountain in Scottsdale, Ariz.

37th Annual J.P. Morgan Healthcare Conference on January 7-10, 2019 in San Francisco, Calif.

On November 8, 2018 Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the third quarter ended September 30, 2018 and highlighted recent progress and upcoming milestones for its pipeline programs (Filing, 8-K, Scholar Rock, NOV 8, 2018, View Source [SID1234530987]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to make important progress with our pipeline having now completed enrollment in the multiple ascending dose portion of the ongoing Phase 1 clinical trial for SRK-015 with interim results and the initiation of a Phase 2 proof-of-concept study in patients with Spinal Muscular Atrophy (SMA) expected in the first quarter of 2019," said Nagesh Mahanthappa, Ph.D, President and CEO of Scholar Rock. "We also look forward to our upcoming presentation of preclinical data at the SITC (Free SITC Whitepaper) annual meeting that will demonstrate the ability of a highly specific inhibitor of TGFβ1 to render resistant solid tumors vulnerable to anti-PD1 while minimizing the toxicities traditionally associated with pan-TGFβ inhibitors."

Key R&D Highlights and Upcoming Milestones

SRK-015 Program:

Completed Enrollment in Multiple Ascending Dose Portion of Phase 1 Clinical Trial for SRK-015 with Interim Results Expected the First Quarter of 2019. A placebo-controlled, double-blind Phase 1 clinical trial was initiated in May 2018 to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses of intravenous SRK-015, a selective inhibitor of the activation of myostatin, in healthy adult volunteers. Enrollment in the single ascending dose and multiple ascending dose portions of the trial have been completed. Interim results from this ongoing Phase 1 trial are expected in the first quarter of 2019.
Plan to Initiate Phase 2 Proof-of-Concept Study for SRK-015 in SMA in the First Quarter of 2019. Pending supportive safety data from the Phase 1 clinical trial, Scholar Rock plans to initiate a Phase 2 proof-of-concept study in the first quarter of 2019 to evaluate the safety and efficacy of SRK-015 in patients with SMA as a monotherapy or in conjunction with an approved survival motor neuron (SMN) upregulator therapy as background standard of care.
Plan to Identify Second Indication for SRK-015 in the First Half of 2019. Scholar Rock is actively assessing numerous potential clinical settings in which the selective inhibition of the activation of myostatin may offer therapeutic benefit and intends to identify a second indication for SRK-015 in the first half of 2019.
TGFβ1 Program:

Presenting Preclinical Data at Upcoming SITC (Free SITC Whitepaper) Annual Meeting Highlighting the Role of TGFβ1 Inhibition in Overcoming Primary Resistance to Anti-PD1. At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting being held November 9-11, 2018, Scholar Rock will be presenting preclinical results demonstrating the ability of a highly specific inhibitor of TGFβ1, SRTβ1-Ab3, to render tumors vulnerable to checkpoint blockade therapy in syngeneic tumor models of primary resistance (Poster #550). Combination treatment with SRTβ1-Ab3 and anti-PD1 resulted in tumor regression or tumor control, and a significant survival benefit, while minimizing toxicities traditionally associated with pan-TGFβ inhibitors.
Plan to Nominate Product Candidate for TGFβ1 Program by the End of the First Half of 2019. Scholar Rock’s second antibody program is focused on the development of highly specific inhibitors of the activation of TGFβ1, and the company intends to nominate a product candidate and first indication in oncology, immuno-oncology or fibrosis by the end of the first half of 2019.
Third Quarter 2018 Financial Results

Net loss for the quarter ended September 30, 2018 was $10.8 million or $0.44 per share compared to a net loss of $5.9 million or $3.70 per unit for the same quarter last year.

Research and development expense was $8.1 million for the quarter ended September 30, 2018, compared to $4.8 million in the same quarter in 2017. The $3.3 million increase year-over-year reflects development and manufacturing costs associated with lead product candidate, SRK-015, research costs associated with preclinical studies, as well as increased personnel-related costs to support continued progress with the pipeline.
General and administrative expense was $3.2 million for the quarter ended September 30, 2018, compared to $1.2 million in the same quarter in 2017. The $2.0 million increase year-over-year was primarily attributable to increased headcount and higher professional and consulting fees associated with ongoing business activities and operating as a public company.
As of September 30, 2018, Scholar Rock had cash, cash equivalents, and marketable securities of $104.0 million, compared to $58.0 million at the end of 2017. Scholar Rock believes its cash and cash equivalents balance will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2020.

On November 8, 2018 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported its two poster presentations by its licensee NantCell, Inc. at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting, taking place November 7-11, 2018 in Washington, DC (Press release, CytRx, NOV 8, 2018, View Source [SID1234530976]). The abstracts describe early safety and efficacy data from the Phase 1b portion of Phase 1b/2 clinical trials evaluating NantCell’s high-affinity natural killer (haNK) cell therapy in combination with anti-cancer agents, including aldoxorubicin, in patients with third-line or greater triple negative breast cancer (TNBC), fourth-line or greater head and neck squamous cell carcinoma (HNSCC) or recurrent metastatic pancreatic cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Preliminary Phase 1b Results in TNBC and HNSCC

In this Phase 1b, single-arm, open-label trial, treatment was administered in 3-week cycles of low-dose chemotherapy (aldoxorubicin, cyclophosphamide, cisplatin, nab-paclitaxel, 5-FU/L), antiangiogenic therapy (bevacizumab), engineered allogeneic high affinity CD-16 NK-92 cells (haNK), IL-15RαFc (N803), adenoviral vector-based CEA, MUC1, Brachyury, HER2 vaccine, yeast vector-based RAS, Brachyury and CEA vaccine, and an IgG1 PD-L1 inhibitor, avelumab plus cetuximab. All patients in both trials received aldoxorubicin. The primary endpoint is incidence of treatment-related adverse events (AEs). Secondary endpoints include overall response rate (ORR), disease control rate (DCR), progression-free survival (PFS), and overall survival (OS).

The abstract includes data from three patients with third-line or greater TNBC and two patients with fourth-line or greater HNSCC. All treatment was completed in the outpatient setting, with no immune-related AEs. Four hematologic dose limiting toxicities (DLTs) were observed and managed with a planned dose reduction of cisplatin. Of the three patients with TNBC, two (67%) experienced a partial response (PR). Of the two patients with HNSCC, both (100%) experienced objective tumor response (100% and 47% decrease, respectively). Overall, four out of the five TNBC and HNSCC patients (80%) had confirmed overall responses, including one patient (20%) with fifth-line metastatic disease who demonstrated a complete response (CR). All responding patients are still undergoing therapy. These preliminary data suggest that the NANT Cancer Vaccine (NCV), comprised of low-dose chemo-radiation combined with innate and adaptive immunotherapy, can be administered safely in an outpatient setting without any observed increase in immune-related AEs.

Preliminary Phase 1b Results in Metastatic Pancreatic Cancer

In this Phase 1b, single-arm, open-label trial, treatment was administered in 3-week cycles of low-dose chemotherapy (aldoxorubicin, cyclophosphamide, oxaliplatin, nab-paclitaxel, 5-FU/L), antiangiogenic therapy (bevacizumab), engineered allogeneic high affinity CD-16 NK-92 cells (haNK), IL-15RαFc (N-803), adenoviral vector-based CEA vaccine, yeast vector-based RAS vaccine, and an IgG1 PDL1 inhibitor, avelumab. All metastatic pancreatic cancer patients received aldoxorubicin. The primary endpoint is incidence of treatment-related AEs. Secondary endpoints include ORR, DCR, PFS, and OS.

The abstract includes data from ten patients with third-line or greater metastatic pancreatic cancer. All treatment was safely administered in the outpatient setting. AEs were primarily hematologic which were managed by appropriate planned chemo dose reductions. No DLTs have occurred and no haNK-related AEs have occurred to date. Of the ten evaluable patients, nine have achieved stable disease (SD) for ≥ 8 weeks for a DCR of 90%. Median PFS was 5.8 months (95% confidence interval: 3.3 – 8.8) and OS was 9.5 months (95% CI: 5.0 – upper limit not yet reached) with patients continuing treatment. One patient demonstrated resolution of a metastatic lung tumor within 8 weeks of initiating NCV therapy. These preliminary results suggest that the NCV treatment regimen was well tolerated and support the safety and tolerability of the regimen. These preliminary efficacy results are encouraging and the overall survival of 9.5 months currently exceeds all standards of care for patients at this advanced stage of disease.

Eric Curtis, CytRx’s President and Chief Operating Officer, stated, "The data presented by NantCell at SITC (Free SITC Whitepaper)’s 33rd Annual Meeting are optimistic. We are pleased to see that these NCV regimens including aldoxorubicin are well tolerated and support the overall thesis being investigated. These data may ultimately lead to more combinations of aldoxorubicin with cutting edge therapies being studied in tumor types with high unmet needs. We look forward to seeing more data as these clinical trials progress."

"In the oncology marketplace, we have seen clear treatment trends by Big Pharma and recent regulatory approvals moving toward combining the category of potential Centurion drug candidates such as LADR 7, 8, 9 and 10 with immunotherapy."

Aldoxorubicin is a rationally-engineered cytotoxic which employs a linker bound to albumin to deliver doxorubicin directly into the tumor. CytRx out-licensed global development, manufacturing, and commercialization rights for aldoxorubicin to NantCell, Inc., a private subsidiary of NantWorks, LLC, in July 2017.

These data were highlighted at SITC (Free SITC Whitepaper)’s 33rd Annual Meeting in a presentation titled, "First in Human Data in Advanced Solid Tumors of NANT Cancer Vaccine: A Novel Temporospatial Orchestration of the Innate (NK) & Adaptive Immune System to Induce Antigen Cascade & Immunogenic Cell Death" which took place on Wednesday, November 7, 2018 from 5:45-6:00pm ET.

About the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)

The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) is the world’s leading member-driven organization specifically dedicated to improving cancer patient outcomes by advancing the science and application of cancer immunotherapy. Established in 1984, SITC (Free SITC Whitepaper), a 501(c)(3) not-for-profit organization, serves scientists, clinicians, academicians, patients, patient advocates, government representatives and industry leaders from around the world. Currently, SITC (Free SITC Whitepaper) has more than 2,000 members who represent 22 medical specialties in 42 countries around the world. Through emphasis on high-caliber scientific meetings; dedication to education and outreach activities; focus on initiatives of major importance in the field; and commitment to collaborations with like-minded domestic and international organizations, government and regulatory agencies, associations and patient advocacy groups, SITC (Free SITC Whitepaper) brings together all aspects of the cancer immunology and immunotherapy community. Through educational programs that foster scientific exchange and collaboration, SITC (Free SITC Whitepaper) aims to one day make the word "cure" a reality for cancer patients everywhere.