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Intrexon Announces Third Quarter 2018 Financial Results

On November 8, 2018 Intrexon Corporation (NASDAQ: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported its third quarter financial results for 2018.

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Recent Commercial Achievements:

Intrexon continues discussions with several major energy companies concerning partnering of its Methane Bioconversion Platform;
Site selection on Intrexon’s first 2,3 BDO plant is on track for year end;
Okanagan Specialty Fruits, a wholly owned subsidiary of Intrexon, completed the 2018 Arctic Apple harvest, yielding ten times last year’s production, and expects to make pre-sliced Arctic apples available through over 500 retail outlets this month;
EnviroFlight, Intrexon’s joint venture with Darling Ingredients Inc., is scheduled to open phase one of the largest black soldier fly larvae (BSFL) facility in the U.S. this month; and
Intrexon commenced deployment of its Botticelli platform, beginning with a collaboration in tomato with a large international producer.
Recent Technical/Business Achievements:

Precigen, Inc., a wholly owned subsidiary of Intrexon, recently reported data on a multigenic therapeutic candidate that in appropriate pre-clinical models suggests potential superiority to approved anti-PD-1 checkpoint inhibitors;
From Intrexon’s methane bioconversion platform, the Company now is producing 2,3, BDO from natural gas at roughly 50% of the theoretical target yield, has demonstrated performance at 500X scale-up and has conducted sustained production runs exceeding 1,000 hours;
Oxitec, Ltd., a wholly owned subsidiary of Intrexon, entered into a second cooperative agreement with the Bill & Melinda Gates Foundation to develop a new strain of Oxitec’s Friendly biological engineering platform to develop a self-limiting Anopheles stephensi mosquito to help combat this mosquito that spreads malaria in India, Middle East and the Horn of Africa;
ActoBio Therapeutics, Inc., a wholly owned subsidiary of Intrexon, and T1D Partners, LLC, announced that the first patient has been dosed in the Company’s Phase Ib/IIa clinical trial for AG019 for the treatment of early onset type 1 diabetes (T1D);
Continuing its expansion into regenerative medicine, Exemplar Genetics, a wholly owned subsidiary of Intrexon, reported the first pig has been born with the potential to produce organs for human transplant;
Trans Ova Genetics, a wholly owned subsidiary of Intrexon, created six bull calves that rank at the top of the global Holstein bull population;
Intrexon announced advances in the development of its engineered yeast platform to produce cannabinoids for medical use via fermentation. This microbe-based process has potential to provide greater supply-chain security and avoids the resource-intensive isolation that often leads to quality and quantity variability in end products;
The U.S. Food and Drug Administration (FDA) recommended amendment of the Association of American Feed Control Officials (AAFCO) ingredient definition of dried BSFL to include feeding to poultry. The approval of BSFL for use in poultry feed expands the potential for this ingredient as a more sustainable source of protein and enables EnviroFlight to support this new market opportunity with its new facility;
Collaborator Fibrocell Science, Inc. (NASDAQ: FCSC) reported the FDA granted Fast Track Designation to FCX-013, the company’s clinical stage candidate for the treatment of moderate to severe localized scleroderma, and the FDA’s Office of Orphan Products Development awarded Fibrocell a $1.4 million clinical trial research grant for continued clinical development of FCX-007, the company’s gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB); and
Collaborator Oragenics, Inc. (NYSE: OGEN) announced the resumption of its Phase 2 clinical trial for AG013 for the potential treatment of oral mucositis (OM).
Recent Corporate Highlights:

Intrexon completed its registered underwritten public offering of $200 million aggregate principal amount of 3.50% convertible senior notes due in 2023 (Convertible Notes);
Precigen Inc., a wholly owned subsidiary of Intrexon, and Ziopharm Oncology, Inc. (NASDAQ: ZIOP) announced a new definitive license agreement to replace all existing agreements between the companies that will provide Ziopharm exclusive and non-exclusive rights to technology controlled by Precigen, Inc., as well as securing for Precigen developmental control over the majority of its portfolio.
The Company, through its subsidiary ActoBio Therapeutics, acquired the remaining interests in certain entities previously owned by a related party. As a result, ActoBio owns the exclusive rights to use its technologies to develop therapeutics in the fields of chronic rhinosinusitis and celiac disease;
Intrexon transferred its stock exchange listing from the New York Stock Exchange (NYSE) to the Nasdaq Global Select Market (Nasdaq) and began trading under "XON" ticker on the Nasdaq exchange on September 25, 2018; and
Intrexon announced the formation of a Bioinformatics Hub in Munich, establishing Intrexon Bioinformatics Germany GmbH (IBG).
Third Quarter 2018 Financial Highlights:

Total revenues of $32.4 million, a decrease of 30% from the third quarter of 2017;
Net loss of $57.3 million attributable to Intrexon, or $(0.44) per basic share, including non-cash charges of $38.7 million;
Adjusted EBITDA of $(28.9) million, or $(0.22) per basic share;
The net change in deferred revenue related to upfront and milestone payments, which represents the cash and stock received from collaborators less the amount of revenue recognized during the period, was a decrease of $17.3 million compared to a decrease of $8.6 million in the third quarter of 2017; and
Cash, cash equivalents, and short-term investments totaled $246.6 million, the value of preferred shares totaled $158.4 million, and the value of common equity securities totaled $4.7 million at September 30, 2018.
Year-to-Date 2018 Financial Highlights:

Total revenues of $117.4 million, a decrease of 24% from the nine months ended September 30, 2017;
Net loss of $168.9 million attributable to Intrexon, or $(1.31) per basic share, including non-cash charges of $109.0 million;
Adjusted EBITDA of $(75.3) million, or $(0.58) per basic share; and
The net change in deferred revenue related to upfront and milestone payments, which represents the cash and stock received from collaborators less the amount of revenue recognized during the period, was a decrease of $34.5 million compared to a decrease of $28.2 million in the nine months ended September 30, 2017.
"Our company is transitioning from one with great science and technology to one with great products and product candidates that embody our engineered biology," commented Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon. "Recognizing our requirements, we are fully occupied on the development of systems, teams and plans to successfully commercialize products that we believe will be transformative in their fields."

Mr. Kirk concluded, "While we do not underestimate the challenges that face us – or anyone – in making such a transition, we have persevered in the execution of our plans laid long ago and believe that we are seeing the fruits of our prior labors. It long has been our goal to create one of the truly great companies in the world and all of us at intrexon are excited to be where we are today and looking with great anticipation toward our future."

Third Quarter 2018 Financial Results Compared to Prior Year Period

Total revenues decreased $13.6 million, or 30%, from the quarter ended September 30, 2017. Collaboration and licensing revenues decreased $13.8 million from the quarter ended September 30, 2017 due to (i) a decrease in research and development services for certain of the Company’s exclusive channel collaborations, or ECCs, as the Company redeployed certain resources towards supporting prospective new platforms and partnering opportunities and began to focus more on the further development of relationships and structures that provide the Company with more control and ownership over the development process and commercialization path, (ii) a decrease in research and development services for certain of the Company’s ECCs as a result of program progression where the Company’s collaborators have taken responsibility of the execution of the programs, (iii) changes in revenue recognition for upfront and milestone payments under the new Accounting Standards Codification 606, or ASC 606, revenue standard whereby revenues are recognized based on the amount of services the Company performs for its collaborators, and (iv) the mutual termination of the Company’s second ECC with ZIOPHARM for the treatment of graft-versus-host disease in December 2017. Gross margin on products declined in the current period as a result of increased operating costs associated with new product offerings and cloned products. Gross margin on services improved in the current period as a result of pricing changes and an increase in the number of embryos produced per bovine in vitro fertilization cycle due to improved production results.

Research and development expenses increased $8.4 million, or 23%, and include $8.7 million expense related to in-process research and development reacquired as part of an asset acquisition in September 2018. Although selling, general and administrative (SG&A) expenses were consistent period over period, legal and professional fees decreased $2.9 million primarily due to a decline in the use of regulatory and other consultants. This decrease was offset primarily by higher compensation expenses related to performance and retention incentives for SG&A employees.

Year-to-Date 2018 Financial Results Compared to Prior Year Period

Total revenues decreased $36.6 million, or 24%, from the nine months ended September 30, 2017. Collaboration and licensing revenues decreased $37.8 million from the nine months ended September 30, 2017 primarily due to (i) a decrease in research and development services for certain of the Company’s ECCs as the Company redeployed certain resources towards supporting prospective new platforms and partnering opportunities and began to focus more on the further development of relationships and structures that provide the Company with more control and ownership over the development process and commercialization path, (ii) a decrease in research and development services for certain of the Company’s ECCs as a result of program progression where the Company’s collaborators have taken responsibility of the execution of the programs, (iii) changes in revenue recognition for upfront and milestone payments under the new ASC 606 revenue standard whereby revenues are recognized based on the amount of services the Company performs for its collaborators, and (iv) the mutual termination of the Company’s second ECC with ZIOPHARM for the treatment of graft-versus-host disease in December 2017. Product revenues decreased $2.2 million or 9% primarily due to lower customer demand for live calves, cows previously used in production, and cloned products. These decreases were partially offset by increased customer demand for pregnant recipients. Gross margin on products declined in the current period as a result of lower product sales and increased operating costs associated with new product offerings and cloned products. The increase in service revenues of $2.5 million, or 7%, as well as the gross margin thereon relates to pricing changes and an increase in the number of embryos produced per bovine in vitro fertilization cycle due to improved production results.

Research and development expenses increased $19.4 million, or 19%, and include (i) $8.7 million expense related to in-process research and development reacquired as part of an asset acquisition in September 2018 and (ii) $5.3 million of one-time costs associated with closing one of Oxitec’s Brazilian subsidiary’s leased research and development facilities as the Company decentralized operations previously conducted in this facility. Research and development consultants and lab supplies increased $3.1 million primarily due to increased expenses from contract research organizations and consultants providing services for both programs being developed internally and pursuant to some of the Company’s collaborations. Depreciation and amortization increased $2.1 million primarily as a result of the depreciation expense on research and development assets and amortization of developed technology acquired from GenVec, Inc. in June 2017. Although SG&A expenses were consistent period over period, legal and professional fees decreased $7.5 million primarily due to decreased legal fees associated with ongoing litigation and decreased fees incurred for regulatory and other consultants. This decrease was offset by an increase of $6.9 million in compensation expenses related to performance and retention incentives for SG&A employees.

Conference Call and Webcast

The Company will host a conference call today Thursday, November 8th, at 5:30 PM ET to discuss the third quarter 2018 financial results and provide a general business update. The conference call may be accessed by dialing 1‑888-317-6003 (Domestic US), 1-866-284-3684 (Canada), and 1-412-317-6061 (International) and providing the number 8225818 to join the Intrexon Corporation Call. Participants may also access the live webcast through Intrexon’s website in the Investors section at View Source

Magenta Therapeutics Reports Recent Operational Progress and Third Quarter 2018 Financial Results

On November 8, 2018 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of bone marrow transplant to more patients, reported financial results and business highlights for the third quarter ended September 30, 2018 (Press release, Magenta Therapeutics, NOV 8, 2018, View Source [SID1234531017]).

"Over the third quarter of 2018, Magenta has continued its progress across the portfolio and remains well funded and on track for our 2020 vision of three programs moving forward in the clinic in multiple indications," said Jason Gardner, D.Phil., chief executive officer and president, Magenta Therapeutics. "We look forward to sharing important data updates on our stem cell expansion, mobilization and targeted conditioning programs at ASH (Free ASH Whitepaper) this year as we work to build a fully integrated company with the singular goal of allowing more patients to receive curative cell therapies."

Recent Business Highlights:

Nine Clinical and Preclinical Abstracts Accepted for Presentation at the ASH (Free ASH Whitepaper) Annual Meeting: Magenta announced on November 1st, 2018 that nine abstracts from the Company and its collaborators were accepted for presentation at the ASH (Free ASH Whitepaper) annual meeting in December 2018. The abstracts cover the breadth of Magenta’s integrated portfolio of programs and include preliminary clinical data from the Company’s Phase 2 study of cell therapy MGTA-456 in patients with inherited metabolic disorders.

Continued to Strengthen Robust Intellectual Property Position: Magenta’s first U.S. patent, directed to dosage regimens of mobilization therapy MGTA-145 and plerixafor, was issued in August 2018. The Company’s second U.S. patent was issued for the C200 conditioning program in October 2018, with claims directed to methods of treatment using an anti-CD117-amatoxin antibody-drug conjugate (ADC). For the C100 targeted patient preparation program, an Australian patent application was allowed with claims directed to methods of treatment using an anti-CD45-amatoxin ADC.

Financial Results:

Cash Position: Cash and cash equivalents as of September 30, 2018, were $159.7 million compared to $51.4 million on December 31, 2017. The increase is primarily driven by net proceeds from the $52.2 million Series C preferred stock financing completed in April 2018, and net proceeds of $89.9 million from Magenta’s IPO completed in June 2018. Magenta anticipates that its cash and cash equivalents will be sufficient to fund operations and capital expenditures through at least the first quarter of 2020 on the Company’s current business plan.

Research and Development Expenses: Research and development (R&D) expenses were $11.4 million in the third quarter of 2018, compared to $5.2 million for the same period in 2017. The increase was largely due to increased preclinical costs, toxicology studies and manufacturing to support our mobilization program, the advancement of the MGTA-456 Phase 2 clinical trial, continued progression of the Company’s pipeline and increased costs associated with the growth of the Company.

General and Administrative Expenses: General and administrative (G&A) expenses were $5.3 million for the third quarter of 2018, compared to $1.8 million for the same period in 2017. The increase was largely due to increased G&A personnel and facility costs associated with the growth of the Company.

Net Loss: Net loss was $16 million for the third quarter of 2018, compared to net loss of $6.9 million for the same period in 2017

Lilly to Participate in Credit Suisse 27th Annual Healthcare Conference

On November 8, 2018 Eli Lilly and Company (NYSE: LLY) reported that it will participate in the Credit Suisse 27th Annual Healthcare Conference on Tuesday, November 13, 2018 (Press release, Eli Lilly, NOV 8, 2018, View Source [SID1234531002]). Enrique Conterno, senior vice president of Lilly and president of Lilly Diabetes and Lilly USA, will participate in a fireside chat at 3:40 p.m., Eastern Time.

A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

Karyopharm Reports Third Quarter 2018 Financial Results and Highlights Recent Company Progress

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]).

"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.

OncoSec Appoints Robert Ward to its Board of Directors

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]).

"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.