On November 5, 2020 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported business highlights and financial results for the third quarter ended September 30, 2020 (Press release, Fate Therapeutics, NOV 5, 2020, View Source [SID1234570043]).

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"The clinical data across our iPSC product platform continue to solidify our conviction that multiple doses of iPSC-derived NK cells can be administered off-the-shelf in the outpatient setting, are well-tolerated, and can drive anti-tumor activity, including in combination with monoclonal antibody therapy," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We have now expanded the scope of clinical investigation for FT516 to solid tumors as well as for FT596 to chronic lymphocytic leukemia after observing clinical activity in diffuse large B-cell lymphoma at the first dose level. In addition, we have initiated first-in-human investigation of the first-ever CRISPR-edited, iPSC-derived cell therapy FT538, which incorporates three engineered elements to enhance multiple mechanisms of innate immunity, in acute myeloid leukemia and multiple myeloma."

Clinical Programs

FT596 (CAR19 + hnCD16 + IL-15RF) NK Cell Product Candidate

First Patients Treated with Dual-Antigen Targeting Regimen of FT596 in Combination with Rituximab. The Company is conducting a multi-center Phase 1 clinical trial of FT596, its universal, off-the-shelf, chimeric antigen receptor (CAR) natural killer (NK) cell product candidate, as a monotherapy and in combination with CD20-targeted monoclonal antibody therapy for the treatment of relapsed / refractory B-cell lymphoma (NCT04245722). The first patients have been treated at the first dose level (30 million cells) in combination with rituximab, which enables dual-antigen targeting of both CD19 and CD20 antigens expressed on malignant B cells. In addition, the Company has initiated enrollment at the second dose level (90 million cells) as monotherapy. FT596 is derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary CAR optimized for NK cell biology that targets CD19 (CAR19); a novel high-affinity, non-cleavable CD16 (hnCD16) Fc receptor that enhances antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells; and an IL-15 receptor fusion (IL-15RF) that augments NK cell activity.
Clinical Scope of FT596 Phase 1 Study Expanded to CLL. In August, the Company amended the clinical protocol of its FT596 Phase 1 clinical trial to include treatment of relapsed / refractory chronic lymphocytic leukemia (CLL). Under the amended protocol, the Company has initiated enrollment at the first dose level (30 million cells) as monotherapy, and plans to begin enrollment of FT596 in combination with obinutuzumab upon dose-limiting toxicity clearance of monotherapy at the first dose level.
Investigator-initiated Clinical Trial of FT596 for Relapse Prevention Opened to Enrollment. The Phase 1 study, which is sponsored by investigators from the Masonic Cancer Center, University of Minnesota, is designed to assess the potential of FT596 to prevent relapse for patients with B-cell lymphoma who have undergone autologous hematopoietic stem cell transplant and are considered high risk for early relapse (NCT04555811). Up to three dose levels of FT596, beginning at 90 million cells per dose, in combination with rituximab will be evaluated.
FT516 (hnCD16) NK Cell Product Candidate

First Patient Treated with FT516 in Combination with Avelumab for Advanced Solid Tumors. FT516 is the Company’s universal, off-the-shelf NK cell product candidate derived from a clonal master iPSC line engineered to express its novel hnCD16 Fc receptor. The Company has treated the first patient in a multi-center Phase 1 clinical trial of FT516 in combination with avelumab, an FDA-approved monoclonal antibody targeting PD-L1 (NCT04551885). Each patient is to receive three once-weekly doses of FT516, beginning at 90 million cells per dose, for up to two 30-day cycles in combination with avelumab. The Company is also continuing to enroll its multi-center Phase 1 clinical trial of FT516 as a monotherapy for the treatment of acute myeloid leukemia (AML) and in combination with CD20-targeted monoclonal antibody therapy for the treatment of relapsed / refractory B-cell lymphoma (NCT04023071).
First Patients Treated with FT516 for COVID-19. Investigators from the Division of Infectious Diseases and International Medicine, University of Minnesota have treated the first patients with FT516 for Coronavirus Disease 2019 (COVID-19). The investigator-initiated Phase 1 clinical trial is evaluating up to three escalating doses of FT516 administered over one week for patients with COVID-19 at high risk of developing critical life-threatening illness (NCT04363346).
FT538 (hnCD16 + IL-15RF + CD38KO) NK Cell Product Candidate

Enrollment Initiated with FT538 in Phase 1 Clinical Trial. The multi-center Phase 1 study of FT538, the first-ever CRISPR-edited, iPSC-derived cell therapy, is designed to assess the safety and efficacy of three once-weekly doses of FT538 as a monotherapy for patients with relapsed / refractory AML and in combination with the CD38-targeted monoclonal antibody daratumumab for patients with relapsed / refractory multiple myeloma. Up to four dose levels of FT538, beginning at 100 million cells per dose, will be evaluated. FT538 is derived from a clonal master iPSC line engineered with three functional components to enhance innate immunity: a novel hnCD16 Fc receptor that enhances ADCC; an IL-15RF that augments NK cell activity; and the deletion of the CD38 gene (CD38KO), which promotes persistence under oxidative stress and prevents NK cell fratricide in combination with CD38-targeted monoclonal antibody therapy.
FT500 NK Cell Product Candidate

FT500 Dose Expansion Ongoing in Solid Tumors Resistant to Checkpoint Inhibitor Therapy. At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting being held virtually from November 9-14, 2020, the Company plans to present previously disclosed clinical data from the dose-escalation stage of the Company’s Phase 1 clinical trial of FT500 (NCT03841110). Fifteen heavily pre-treated patients, ten of whom were refractory to their last prior therapy, were administered up to six doses of FT500 as a salvage treatment for patients with advanced solid tumors. No dose-limiting toxicities, and no FT500-related severe adverse events (AEs) or Grade ≥ 3 AEs, were observed. In addition, there were no reported cases of cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, or graft-versus-host disease. Eleven patients had a best overall response of stable disease. The Company is currently enrolling the dose-expansion stage of the FT500 Phase 1 clinical trial for patients with non-small cell lung cancer or classical Hodgkin lymphoma who are refractory to, or have relapsed on, checkpoint inhibitor therapy.
FT819 (TRAC-integrated 1XX-CAR19) T-Cell Product Candidate

FT819 GMP Manufacturing Campaign Completed. In the fourth quarter of 2020, the Company plans to initiate a multi-center Phase 1 clinical trial of FT819, the first-ever off-the-shelf, allogeneic CAR T-cell therapy derived from a clonal master iPSC line, for patients with B-cell lymphoma, chronic lymphocytic leukemia, or acute lymphoblastic leukemia. The Company has now completed its first GMP manufacturing campaign for FT819 and is conducting final release testing to enable clinical disposition and off-the-shelf availability. FT819 is engineered with several first-of-kind features designed to improve the safety and efficacy of CAR T-cell therapy including a novel 1XX CAR signaling domain targeting CD19+ malignancies (1XX-CAR19) that extends T-cell effector function without eliciting exhaustion; integration of the CAR transgene directly into the T-cell receptor alpha constant (TRAC) locus, which promotes uniform CAR expression and enhances T-cell potency; and complete bi-allelic disruption of T-cell receptor expression to prevent graft-versus-host disease, a potentially life-threatening complication associated with allogeneic T-cell therapy.
Corporate Highlights

Twelve Abstracts Accepted for Presentation at ASH (Free ASH Whitepaper). At the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held virtually from December 5-8, 2020, the Company plans to present 12 abstracts, including four oral presentations, for the Company’s iPSC product platform. In addition, the Company plans to host a virtual investor event entitled "The Power of hnCD16" to highlight the unique features and functionality of its novel hnCD16 Fc receptor, which is incorporated into the Company’s FT516, FT596, FT538 and FT576 product candidates.
Five Abstracts Accepted for Presentation at SITC (Free SITC Whitepaper). At SITC (Free SITC Whitepaper), the Company plans to present five abstracts including an oral presentation highlighting a new iPSC-derived CAR immune cell program targeting the pan-tumor associated antigen B7-H3, which is commonly expressed on hematological and solid tumors and is associated with metastasis and poor patient prognosis.
Preclinical Data Published in Science Translational Medicine Demonstrate iPSC-derived NK Cells Augment Checkpoint Inhibitor Therapy. Under a research collaboration between the University of Minnesota and the Company, scientists demonstrated that iPSC-derived NK (iNK) cells have the potential to overcome mechanisms of resistance to checkpoint inhibitor therapy. In preclinical studies, iNK cells were shown to rapidly infiltrate and kill solid tumors including those having alterations in antigen presentation or HLA downregulation, which are primary mechanisms of resistance to checkpoint inhibitor therapy. In addition, iNK cells in concert with T cells and anti-PD-1 antibody were shown to significantly improve anti-tumor activity in vivo compared to T cells and anti-PD-1 antibody either alone or in combination. The peer-reviewed findings were published online in Science Translational Medicine on November 4 (Cichocki et al).
Appointed Edward Dulac as Chief Financial Officer. In August, Mr. Dulac joined the Company from Celgene Corporation, where he most recently served as Vice President, Business Development & Strategy and was responsible for business development opportunities in the therapeutic areas of hematology and oncology, inflammation and immunology, and neuroscience. Mr. Dulac has extensive biopharmaceutical experience, having served for over 20 years in positions in finance, business development, and product portfolio strategy.
Third Quarter 2020 Financial Results

Cash & Investment Position: Cash, cash equivalents and investments as of September 30, 2020 were $502.0 million.
Total Revenue: Revenue was $7.6 million for the third quarter of 2020, which was derived from the Company’s collaborations with Janssen and Ono Pharmaceutical.
R&D Expenses: Research and development expenses were $30.7 million for the third quarter of 2020, which includes $4.7 million of non-cash stock-based compensation expense.
G&A Expenses: General and administrative expenses were $8.4 million for the third quarter of 2020, which includes $3.1 million of non-cash stock-based compensation expense.
Other Expenses: Other expenses, net were $27.2M, which includes a $27.6M non-cash charge equal to the fair value of certain contingent milestone payments that will be owed to Memorial Sloan Kettering Cancer Center upon the Company’s achievement of a specified clinical milestone with an iPSC-derived CAR T-cell product candidate and the subsequent appreciation of the Company’s common stock price per share.
Shares Outstanding: Common shares outstanding were 87.0 million, and preferred shares outstanding were 2.8 million, as of September 30, 2020. Each preferred share is convertible into five common shares.
Today’s Conference Call and Webcast
The Company will conduct a conference call today, Thursday, November 5, 2020 at 5:00 p.m. ET to review financial and operating results for the quarter ended September 30, 2020. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 1060606. The live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

About FT500

FT500 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line. The product candidate is being investigated in an open-label, multi-dose Phase 1 clinical trial for the treatment of advanced solid tumors (NCT03841110). The study is designed to assess the safety and tolerability of FT500 as a monotherapy and in combination with one of three FDA-approved immune checkpoint inhibitor (ICI) therapies – nivolumab, pembrolizumab or atezolizumab – in patients that have failed prior ICI therapy. Despite the clinical benefit conferred by approved ICI therapy against a variety of tumor types, these therapies are not curative and, in most cases, patients either fail to respond or their disease progresses on these agents. One common mechanism of resistance to ICI therapy is associated with mutations in genes that disrupt antigen presentation and/or down-regulate HLA Class I proteins on cancer cells, which enables T-cell evasion. A potential strategy to overcome resistance is through the administration of allogeneic NK cells, which have the inherent capability to recognize and directly kill cancer cells with these mutations.

About FT516
FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. ADCC is dependent on NK cells maintaining stable and effective expression of CD16, which has been shown to undergo considerable down-regulation in cancer patients. In addition, CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG1 antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. FT516 is being investigated in an open-label, multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-targeted monoclonal antibodies for the treatment of advanced B-cell lymphoma (NCT04023071). Additionally, FT516 is being investigated in an open-label, multi-dose Phase 1 clinical trial in combination with avelumab for the treatment of advanced solid tumor resistant to anti-PDL1 checkpoint inhibitor therapy (NCT04551885).

About FT596
FT596 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology that targets B-cell antigen CD19; a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that augments NK cell activity. In preclinical studies of FT596, the Company has demonstrated that dual activation of the CAR19 and hnCD16 targeting receptors enhances cytotoxic activity, indicating that multi-antigen engagement may elicit a deeper and more durable response. Additionally, in a humanized mouse model of lymphoma, FT596 in combination with the anti-CD20 monoclonal antibody rituximab showed enhanced killing of tumor cells in vivo as compared to rituximab alone. FT596 is being investigated in an open-label, multi-center Phase 1 clinical trial for the treatment of relapsed / refractory B-cell lymphoma as a monotherapy and in combination with rituximab and for the treatment of relapsed / refractory chronic lymphocytic leukemia (CLL) as a monotherapy and in combination with obinutuzumab (NCT04245722).

About FT538
FT538 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three functional components: a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; an IL-15 receptor fusion (IL-15RF) that augments NK cell activity; and the deletion of the CD38 gene, which promotes persistence and function in high oxidative stress environments. FT538 is designed to enhance innate immunity in cancer patients, where endogenous NK cells are typically diminished in both number and function due to prior treatment regimens and tumor suppressive mechanisms. In preclinical studies, FT538 has shown superior NK cell effector function, as compared to peripheral blood NK cells, with the potential to confer significant anti-tumor activity to patients through multiple mechanisms of action. FT538 is being investigated in an open-label, multi-dose Phase 1 clinical trial for the treatment of acute myeloid leukemia (AML) and in combination with daratumumab, a CD38-targeted monoclonal antibody therapy, for the treatment of multiple myeloma (NCT04614636).

On November 5, 2020 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results and provided a business update for the third quarter ended September 30, 2020 (Press release, Deciphera Pharmaceuticals, NOV 5, 2020, View Source [SID1234570042]).

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"The strong U.S. commercial launch of QINLOCK, which is reflected in our first full quarter of results since the approval in May, is a testament to the potential for this new medicine to be a best-in-class treatment for people with GIST," said Steve Hoerter, President and Chief Executive Officer of Deciphera Pharmaceuticals. "During the third quarter, we also made substantial progress in preparing to bring QINLOCK to eligible patients around the world, including the submission and validation of the MAA by the EMA and the establishment of distribution agreements in Canada and Australia."

Mr. Hoerter continued, "In addition to executing on the successful launch of QINLOCK, we continue to advance our pipeline of promising product candidates. Notably, we look forward to presenting new data from our Phase 1/2 study of DCC-3014 in TGCT patients at the CTOS 2020 Virtual Meeting later this month."

Third Quarter 2020 Highlights and Recent Business Updates

QINLOCK (ripretinib) Commercialization
Recorded $15.2 million in net product revenue in the third quarter of 2020, including $14.7 million in U.S. net product revenue in the first full quarter of commercial launch following FDA approval in May 2020.
Submitted and received validation of a Marketing Authorisation Application (MAA) for QINLOCK in fourth-line gastrointestinal stromal tumor (GIST) by the European Medicines Agency (EMA). Validation of the MAA confirms that the application is sufficiently complete for the EMA to begin its formal review process.
Announced plans to establish a targeted commercial infrastructure in key European markets to support the potential launch of QINLOCK, as well as to support additional future product launches.
Entered into exclusive distribution agreements with the following partners to distribute QINLOCK in other territories:
Medison to distribute QINLOCK in Canada and Israel. Health Canada approved QINLOCK for fourth-line GIST in June 2020 under the U.S. FDA’s Project Orbis, an initiative that enables concurrent review of oncology products by international regulatory agencies.
Specialised Therapeutics Asia (STA) to distribute QINLOCK in Australia, New Zealand, Singapore, Malaysia, and Brunei. The Australian Therapeutic Goods Administration (TGA) approved QINLOCK in July 2020 under the U.S. FDA’s Project Orbis.
Presented two mini oral presentations on QINLOCK at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 in September. The first mini oral presentation was on the nine-month follow-up data from the Phase 3 INVICTUS study in patients with fourth-line and fourth-line plus GIST. The second mini oral presentation was on the ongoing Phase 1 study of QINLOCK in patients with second-line through fourth-line plus GIST. The presentation highlighted that the patients receiving QINLOCK who, upon disease progression, dose escalated to QINLOCK 150 mg twice daily (BID) experienced additional, clinically meaningful, progression-free survival benefit across all lines of therapy.

Rebastinib
Presented results from Part 2 (Stage 1) of the platinum-resistant ovarian cancer cohort in the ongoing Phase 1b/2 study of rebastinib in combination with paclitaxel in an E-poster presentation at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020. Data presented demonstrated encouraging efficacy with an objective response rate of 38%, confirmed and unconfirmed, and a clinical benefit rate of 88% at eight weeks. Treatment with rebastinib 50 mg BID in combination with paclitaxel was generally well-tolerated. Enrollment in Stage 2 of the platinum-resistant ovarian cancer cohort at the rebastinib 50 mg BID dose is completed and further efficacy and safety evaluation is ongoing.
Presented results from Part 1 of the Phase 1b/2 study of rebastinib in combination with carboplatin in an E-poster presentation at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020. The clinical benefit rate was 50% at six weeks and 36% at twelve weeks, and the median duration of treatment was 7.8 weeks. Rebastinib in combination with carboplatin was generally well-tolerated. The Part 2 portion of the ongoing Phase 1/2 study is currently enrolling patients and will evaluate the safety and efficacy of rebastinib at the recommended Phase 2 dose of 50 mg BID in combination with carboplatin.
Upcoming Scientific Congress Presentations

Connective Tissue Oncology Society (CTOS) 2020 Virtual Annual Meeting, November 18-21
DCC-3014
Oral Presentation: "Phase 1 dose-escalation study of the safety, tolerability, pharmacokinetics, and pharmacodynamics of DCC-3014 in advanced solid tumors and tenosynovial giant cell tumor"
QINLOCK (ripretinib)
Oral Presentation: "Characterization of the extensive heterogeneity of KIT/PDGFRA mutations in patients with fourth-line advanced gastrointestinal stromal tumor: genomic analysis of the phase 3 INVICTUS study"
Poster Presentation: "Ripretinib demonstrated activity across all KIT/PDGFRA mutations in patients with fourth-line advanced gastrointestinal stromal tumor: analysis from the phase 3 INVICTUS study"
Oral Presentation: "Ripretinib intra-patient dose escalation following disease progression provides clinically meaningful progression-free survival in gastrointestinal stromal tumor in phase 1 study"
Poster Presentation: "Clinical benefit with ripretinib as ≥4th line treatment in patients with advanced gastrointestinal stromal tumor: update from the phase 3 INVICTUS study"
Third Quarter 2020 Financial Results

Revenue: Total revenue for the third quarter of 2020 was $15.5 million, which includes $15.2 million of net product revenue from sales of QINLOCK and $0.3 million of collaboration revenue. Net product revenues for the third quarter of 2020 includes U.S. sales of QINLOCK of $14.7 million and ex-U.S. sales of QINLOCK of $0.5 million. In the third quarter of 2019, the Company did not generate revenue.
Cost of Sales: Cost of sales for the third quarter of 2020 was $0.1 million as the majority of the manufacturing costs related to third quarter QINLOCK sales were incurred prior to FDA approval, and thus, were recorded as R&D expense. Cost of sales will not be significant until the initial pre-launch inventory is depleted, and additional inventory is manufactured and sold. In the third quarter of 2019, there were no cost of sales as no product sales were generated during that period.
R&D Expenses: Research and development expenses for the third quarter of 2020 were $49.2 million, compared to $40.4 million for the same period in 2019. The increase was primarily due to personnel costs, preclinical costs, and clinical trial costs related to DCC-3014, rebastinib, and the Phase 3 INTRIGUE trial in second-line GIST. The increase was partially offset by a decrease in clinical trial expenses related to the Phase 3 INVICTUS trial in fourth-line and fourth-line plus GIST. Non-cash, stock-based compensation was $4.5 million and $2.0 million for the third quarters of 2020 and 2019, respectively.
SG&A Expenses: Selling, general and administrative expenses for the third quarter of 2020 were $30.1 million, compared to $18.0 million for the same period in 2019. The increase was primarily a result of personnel costs as well as external spend associated with commercial preparedness and launch of QINLOCK, increased expenses incurred in connection with Deciphera’s new headquarters that commenced in October 2019, and technology-related costs to support the growth of the business. Non-cash, stock-based compensation was $5.3 million and $2.7 million for the third quarters of 2020 and 2019, respectively.
Net Loss: For the third quarter of 2020, Deciphera reported a net loss of $63.7 million, or $1.13 per share, compared with a net loss of $56.2 million, or $1.28 per share, for the same period in 2019. The increase in net loss was primarily related to increases in R&D and SG&A expenses, partially offset by the recognition of revenues in the third quarter of 2020, as discussed above.
Cash Position: As of September 30, 2020, cash, cash equivalents and marketable securities were $584.3 million, compared to $579.6 million as of December 31, 2019. The increase was primarily due to the Company’s follow-on public offering in February 2020 that provided net proceeds of $188.4 million, partially offset by cash used in operations. Based on its current operating plans, Deciphera expects its current cash, cash equivalents, and marketable securities together with anticipated product revenues, but excluding any potential future milestone payments or other payments under its collaboration or license agreements, will enable the Company to fund its operating and capital expenditures into the second half of 2022.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, November 5, 2020 at 4:30 PM ET. To access the live call by phone please dial (866) 930-5479 (domestic) or (409) 216-0603 (international); the conference ID is 6048987. A live audio webcast of the event may also be accessed through the "Investors" section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On November 5, 2020 DCprime, the front-runner in the field of relapse vaccines, reported the upcoming oral presentation of interim results from its ongoing Phase II clinical trial (ADVANCE II, Clintrials.gov: NCT03697707) at the virtual 62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition on December 5, 2020 (Press release, DCPrime, NOV 5, 2020, View Source [SID1234570041]). The Interim data on both primary and secondary endpoints of the trial highlight the potential of the company’s lead vaccine candidate, DCP-001, to improve the treatment options for patients with Acute Myeloid Leukemia (AML) in the post-remission setting. The study continues to enroll patients at the higher of two vaccine doses tested and updated data will be shown at ASH (Free ASH Whitepaper).

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The oral presentation entitled "Conversion from MRD positive to negative status in AML patients in CR1 after treatment with an Allogeneic Leukemia-derived Dendritic Cell Vaccine" will be presented by Prof. Dr. Arjan van de Loosdrecht from the Department of Hematology at Amsterdam UMC, Amsterdam, The Netherlands, and Principal Investigator for the study.

The accepted abstract is listed below and is now available online on the ASH (Free ASH Whitepaper) website: View Source

ORAL PRESENTATION DETAILS:
Presentation Title: #168: Conversion from MRD positive to negative status in AML patients in CR1 after treatment with an Allogeneic Leukemia-derived Dendritic Cell Vaccine

Session Title: Acute Myeloid Leukemia: Novel therapy, excluding transplantation: Advances in immunotherapeutics for management of AML

Session Date: Saturday, December 5, 2020

Presentation Time: 12:45 PM EST

Presenter: Prof. Dr. Arjan van de Loosdrecht – Department of Hematology, VUmc

On November 5, 2020 Veracyte, Inc. (Nasdaq: VCYT), a pioneering genomic diagnostics company, reported that the Federal Joint Committee (G-BA) has approved its Prosigna Breast Cancer Gene Signature Assay (Press release, Veracyte, NOV 5, 2020, View Source [SID1234570040]). The G-BA decision to reimburse the Prosigna test will provide access to the test for all breast cancer patients in Germany with HR+/HER2- early-stage breast cancer.

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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

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The Prosigna Assay is a second-generation breast cancer test, meaning that it uses advanced genomic technology combined with clinical and pathologic features to inform next steps for patients with early-stage breast cancer. The test analyzes the activity of 50 genes known as the PAM50 gene signature, along with tumor size, lymph node involvement, and a tumor proliferation score to provide early-stage breast cancer patients and their physicians with a prognostic score indicating the probability of cancer recurrence during the next 10 years.

"We are pleased with the G-BA decision, which will enable more breast cancer patients and their physicians in Germany to benefit from the genomic insights offered by our Prosigna test," said Bonnie Anderson, chairman and chief executive officer of Veracyte. "Further, because Prosigna is performed by laboratories locally, this decision will enable German laboratories to deliver precision medicine solutions directly to their physician customers."

The Prosigna test is recommended in guidelines from the German Association of Gynecologic Oncology (AGO), as well as the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the National Institute for Health and Care Excellence (NICE) in the United Kingdom.

Every year around 70,000 women in Germany develop early breast cancer. In many cases, a clear therapy recommendation for or against adjuvant chemotherapy is challenging based on the clinicopathological criteria alone. The Federal Joint Committee supports the use of biomarkers, now including Prosigna, to inform treatment decisions based upon the patient’s individual cancer recurrence risk.

About Prosigna

Prosigna is a prognostic Breast Cancer Gene Signature assay indicated in female breast cancer patients who have undergone either mastectomy or breast-conserving therapy in conjunction with locoregional treatment consistent with standard of care, either as a prognostic indicator for distant recurrence-free survival at 10 years in post-menopausal women with Hormone Receptor- Positive (HR+), lymph node-negative, Stage I or II breast cancer or lymph node-positive (1–3 positive nodes, or 4 or more positive nodes), Stage II or IIIA breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors.

In addition to the risk of recurrence (ROR) information, in Europe the assay provides the intrinsic subtypes of the tumor tissue within three groups – low, intermediate and high. The test’s performance is validated for use on the nCounter Analysis System in laboratories across Europe.

On November 5, 2020 AstraZeneca and MSD’s Lynparza (olaparib) has been approved in the European Union (EU) for the 1st-line maintenance treatment with bevacizumab of patients with homologous recombination deficient (HRD)-positive advanced ovarian cancer (Press release, AstraZeneca, NOV 5, 2020, View Source [SID1234570039]).

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Ovarian cancer is the fifth most common cause of cancer death in the EU and the five-year survival rate is approximately 45%, due partly because women are often diagnosed with advanced disease (Stage III or IV).1-3

The approval by the European Commission was based on a biomarker subgroup analysis of the PAOLA-1 Phase III trial which showed Lynparza, in combination with bevacizumab maintenance treatment, demonstrated a substantial progression-free survival (PFS) improvement versus bevacizumab alone for patients with HRD-positive advanced ovarian cancer. It follows the recommendation for approval by the Committee for Medicinal Products for Human Use of the European Medicines Agency in September 2020.

Isabelle Ray-Coquard, principal investigator of the PAOLA-1 Phase III trial and medical oncologist, Centre Léon Bérard and President of the GINECO group, Paris, France, said: "For women with advanced ovarian cancer, the goal of 1st-line treatment is to delay disease progression for as long as possible with the intent of achieving long-term remission. Unfortunately, once a patient’s cancer recurs, it historically has been incurable. Lynparza together with bevacizumab has demonstrated an impressive median progression-free survival benefit of more than three years and is poised to become the standard of care for eligible patients with HRD-positive tumours in the EU."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "Half of all newly diagnosed patients with advanced ovarian cancer have HRD-positive tumours. Women treated with Lynparza in combination with bevacizumab in the PAOLA-1 Phase III trial lived progression free for a median of more than three years, showing that HRD testing should be an essential component of clinical diagnosis. HRD status can help physicians select a personalised 1st-line treatment regimen for patients to substantially delay relapse in this devastating disease."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "Biomarker testing has rapidly enhanced our understanding of how PARP inhibition can help target this disease. The EU approval reinforces that HRD-positive tumours represent a distinct subset of advanced ovarian cancer and HRD testing is critical for women in this setting."

The PAOLA-1 Phase III trial showed that Lynparza, in combination with bevacizumab maintenance treatment, reduced the risk of disease progression or death by 67% (based on a hazard ratio of 0.33; 95% confidence interval 0.25-0.45). The addition of Lynparza improved PFS to a median of 37.2 months versus 17.7 with bevacizumab alone in patients with HRD-positive advanced ovarian cancer. The data from the PAOLA-1 trial was published in The New England Journal of Medicine in 2019.

Further results recently presented at the European Society for Medical Oncology Virtual Congress 2020 showed a statistically significant improvement in the key secondary endpoint of the time to second disease progression (PFS2). Lynparza with bevacizumab provided benefit beyond first disease progression, improving PFS2 to a median of 50.3 months versus 35.3 with bevacizumab alone.

The full EU indication is for Lynparza in combination with bevacizumab for the maintenance treatment of adult patients with advanced (FIGO Stages III and IV) high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of 1st-line platinum-based chemotherapy in combination with bevacizumab and whose cancer is associated with HRD positive status defined by either a breast cancer susceptibility gene 1/2 (BRCA1/2) mutation and/or genomic instability.

Lynparza in combination with bevacizumab is approved in the US and in several other countries as a 1st-line maintenance treatment for patients with HRD-positive advanced ovarian cancer and is currently under regulatory review in other countries around the world.

Financial considerations

Following this approval for Lynparza in the EU, AstraZeneca will receive a regulatory milestone payment from MSD of $25m, anticipated to be booked as collaboration revenue during the fourth quarter of 2020.

Ovarian cancer

In 2018, there were nearly 68,000 new cases of ovarian cancer diagnosed in the EU and around 45,000 deaths.3 Approximately 50% of ovarian cancers are HRD-positive including BRCA1/2 mutation.4,5 Approximately 15% of ovarian cancers have a BRCA1/2 mutation.6 The primary aim of 1st-line treatment is to delay disease progression for as long as possible with the intent to achieve long-term remission.7-9

Homologous recombination deficiency

HRD, which defines a subgroup of ovarian cancer, encompasses a wide range of genetic abnormalities, including BRCA mutations and beyond. As with BRCA gene mutations, HRD interferes with normal cell DNA repair mechanisms and confers sensitivity to PARP inhibitors including Lynparza.10

PAOLA-1

PAOLA-1 is a double-blinded Phase III trial testing the efficacy and safety of Lynparza added to standard-of-care bevacizumab versus bevacizumab alone, as a 1st-line maintenance treatment for newly diagnosed advanced FIGO Stage III-IV high-grade serous or endometroid ovarian, fallopian tube, or peritoneal cancer patients who had a complete or partial response to 1st-line treatment with platinum-based chemotherapy and bevacizumab. AstraZeneca and MSD announced in August 2019 that the trial met its primary endpoint of PFS in the overall trial population.

Lynparza

Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair (HRR), such as mutations in BRCA1 and/or BRCA2. Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. Lynparza is being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.

Lynparza is currently approved in a number of countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer. It is approved in the US, the EU, Japan, China, and several other countries as 1st-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in the US as a 1st-line maintenance treatment with bevacizumab for patients with HRD-positive advanced ovarian cancer (BRCAm and/or genomic instability). Lynparza is approved in the US, Japan, and a number of other countries for germline BRCA-mutated, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer. It is also approved in the US, the EU and several other countries for the treatment of germline BRCAm metastatic pancreatic cancer. Lynparza is approved in the US for homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (BRCAm and other HRR gene mutations). Regulatory reviews are underway in several countries for ovarian, breast, pancreatic and prostate cancers.

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, has been used to treat over 30,000 patients worldwide. Lynparza has the broadest and most advanced clinical trial development programme of any PARP inhibitor, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

The AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With seven new medicines launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.