On November 1, 2018 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq: RDUS), reported its financial and operating results for the third quarter ended September 30, 2018 and provided a business update (Press release, Radius, NOV 1, 2018, View Source [SID1234530571]).

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"I am very pleased with the continued strong trajectory of our launch and success in growing the U.S. anabolic osteoporosis market. Our financial guidance reflects our confidence in continuing to capture further market share gains for TYMLOS and expanding the osteoporosis anabolic market," said Jesper Hoeiland, President and Chief Executive Officer of Radius.

"With our updated Phase 3 protocol for elacestrant, we are excited to have the opportunity to address a larger patient population with unmet needs. We are on track to deliver on this key milestone and initiate the study in the fourth quarter of this year," said Dr. Charles Morris, Chief Medical Officer of Radius.

TYMLOS (abaloparatide) injection

Third quarter 2018 U.S. net sales of TYMLOS were $27.6 million, a 22% increase over the prior quarter and approximately sevenfold increase from the third quarter of 2017. TYMLOS prescriptions in the third quarter of 2018 accounted for 22% of the total U.S. anabolic osteoporosis market on average (based on Patient Months on Therapy, TRx PMOT).

In October 2018, the U.S. Food and Drug Administration (FDA) approved a labeling supplement for TYMLOS to include additional information from the ACTIVExtend study. The labeling now reflects that after 24 months of open-label alendronate therapy, the vertebral fracture risk reduction achieved with TYMLOS therapy was maintained.

The growth trajectory of the U.S. anabolic market since TYMLOS launched in May 2017 continued in the third quarter of 2018, showing 9% volume growth as compared to the third quarter of 2017, all driven by TYMLOS.

Effective January 1, 2019, TYMLOS is expected to be covered for approximately 274 million U.S. insured lives, representing approximately 95% of U.S. commercial and 64% of Medicare insured lives. 2019 Medicare Part D coverage for TYMLOS increased to approximately 26.2 million lives or 64% of those enrolled in Medicare Part D plans in the U.S., after the decisions from SilverScript Insurance Company (CVS), WellCare Health Plans, Inc., Prime Therapeutics and others to cover TYMLOS for their Medicare Part D beneficiaries. The increased access for TYMLOS in Medicare Part D Formularies for 2019 represents an incremental 28% of anabolic volume opportunity in Medicare Part D. Express Scripts, Inc., UnitedHealthcare (AARP), Kaiser Permanente, and EnvisionRx, among others, decided to continue Medicare Part D coverage of TYMLOS through 2019.

Radius maintains its guidance for TYMLOS to capture on average 19-21% of the U.S. anabolic osteoporosis market in 2018 and its expectations for the U.S. anabolic market to grow at a rate of 7-9% in volume versus 2017.

Financial Guidance

In 2018, Radius expects full-year TYMLOS U.S. net revenues to be between $95 to $98 million and its year-end cash, cash equivalents and investments balance to exceed $220 million.

In 2019, Radius expects full-year TYMLOS U.S. net revenues to be between $155 to $175 million and its year-end cash, cash equivalents and investments balance to exceed $100 million.

Pipeline Highlights

Abaloparatide-Transdermal Patch (abaloparatide-patch)

Radius remains on track with its preparations for the Phase 3 trial and expects to start the study in mid-2019. The Company is in the process of conducting development activities in preparation for the Phase 3 trial and potential NDA filing, including qualification and validation of analytical methods, process development and design control activities. Planning for the facility build-out is also ongoing at the commercial manufacturing site, including scaling up equipment and ensuring readiness for the manufacture of commercial abaloparatide-patches.

Elacestrant (RAD1901)

In the third quarter 2018, the Company finalized the Phase 3 study protocol for elacestrant based on regulatory feedback and additional considerations. The Phase 3 trial will be a single, randomized, open label, active-controlled study of elacestrant as a second- or third-line monotherapy in approximately 460 patients with ER+/HER2- advanced/metastatic breast cancer who have received prior treatment with one or two lines of endocrine therapy, including a cyclin-dependent kinase (CDK) 4/6 inhibitor. Patients in the study will be randomized to receive either elacestrant or the investigator’s choice of an approved hormonal agent. The primary endpoint of the study will be progression-free survival ("PFS"), which the Company will analyze in the overall patient population and in patients with estrogen receptor 1 gene (ESR1) mutations. Secondary endpoints will include evaluation of overall survival (OS), objective response rate (ORR), and duration of response (DOR). Depending on the results, this single trial is intended to support applications for marketing approvals for elacestrant as a second- and third-line monotherapy in the U.S., European Union (EU), and other markets.

Radius expects to initiate the Phase 3 study in the fourth quarter of 2018 with a planned recruitment period of 18-21 months and potential data read-out in 2021.

RAD140

Patient enrollment is ongoing in the Phase 1 study evaluating the safety and maximum tolerated dose of RAD140, a nonsteroidal selective androgen receptor modulator (SARM), in patients with hormone receptor-positive, locally advanced or metastatic breast cancer. The Company expects to present two posters on RAD140 at the San Antonio Breast Cancer Symposium (SABCS) and to provide an update on the RAD140 Phase 1 development program by the end of 2018.

Anticipated Upcoming Milestones

Elacestrant

Initiate a Phase 3 clinical trial as second or third-line monotherapy in advanced/metastatic ER-positive/HER2-negative breast cancer patients in the fourth quarter of 2018

Clinical collaboration agreement for elacestrant combination therapy

RAD140

Continue enrollment in the Phase 1 study and provide a program update by the end of 2018

Abaloparatide

Enter into a partnership for the potential commercialization of abaloparatide-SC outside the U.S. and Japan

Expected Radius Presentations at Upcoming Conferences

On January 7-10, 2019, the Company will present and host one-on-one meetings at the JP Morgan Annual Healthcare Conference in San Francisco.

Third Quarter 2018 Financial Results

Three Months Ended September 30, 2018

For the three months ended September 30, 2018, Radius reported a net loss of $49.8 million, or $1.09 per share, compared to a net loss of $57.8 million, or $1.31 per share, for the three months ended September 30, 2017.

For the three months ended September 30, 2018, non-GAAP adjusted net loss, which excludes expenses related to stock-based compensation, restructuring plans, non-cash interest obligations under debt obligations, and amortization of intangible assets, was $38.8 million, or $0.85 per share, compared to non-GAAP adjusted net loss of $49.3 million, or $1.12 per share, for the three months ended September 30, 2017.

For the three months ended September 30, 2018, TYMLOS net product revenues were $27.6 million compared to approximately $3.5 million for the three months ended September 30, 2017.

Research and development expense for the three months ended September 30, 2018 was $26.8 million compared to $21.0 million for the three months ended September 30, 2017, an increase of $5.8 million, or 28%. This increase was primarily driven by a $2.8 million increase in elacestrant project costs, a $2.3 million increase in abaloparatide-patch project costs, a $1.0 million increase in abaloparatide-SC project costs, and a $0.8 million increase in RAD140 project costs. These increases were partially offset by a $0.2 million decrease in other project related spending, and $0.5 million decrease in personnel related spending attributed to a decrease in headcount from 105 research and development employees as of September 30, 2017 to 95 research and development employees as of September 30, 2018.

For the three months ended September 30, 2018, selling, general and administrative expense was $43.7 million compared to $47.7 million for the three months ended September 30, 2017, a decrease of $4.1 million, or 9%. This decrease was primarily the result of $2.8 million and $0.9 million decreases in compensation and travel related expenses, respectively.

Nine Months Ended September 30, 2018

For the nine months ended September 30, 2018, Radius reported a net loss of $180.2 million, or $3.98 per share, compared to a net loss of $183.2 million, or $4.21 per share, for the nine months ended September 30, 2017.

For the nine months ended September 30, 2018, non-GAAP adjusted net loss, which excludes expenses related to stock-based compensation, restructuring plans, non-cash interest obligations under debt obligations, litigation related payments, and amortization of intangible assets, was $134.4 million, or $2.97 per share, compared to non-GAAP adjusted net loss of $154.2 million, or $3.54 per share, for the nine months ended September 30, 2017.

For the nine months ended September 30, 2018, TYMLOS net product revenues were $64.8 million compared to approximately $4.4 million for the nine months ended September 30, 2017.

Research and development expense for the nine months ended September 30, 2018 was $76.0 million compared to $60.2 million for the nine months ended September 30, 2017, an increase of $15.8 million, or 26%. This increase was primarily driven by a $7.6 million increase in elacestrant project costs, a $4.0 million increase in abaloparatide-SC project costs, a $3.8 million increase in abaloparatide-patch project costs, and a $2.1 million increase in RAD140 project costs. These increases were partially offset by a $0.7 million decrease in other project related spending and a $0.9 million decrease in compensation and travel related expenses attributed to a decrease in headcount from 105 research and development employees as of September 30, 2017 to 95 research and development employees as of September 30, 2018.

Selling, general, and administrative expense for the nine months ended September 30, 2018, was $140.3 million compared to $135.9 million for the nine months ended September 30, 2017, an increase of $4.3 million, or 3%. This increase was primarily the result of $2.5 million and $0.7 million increases in compensation and travel related expenses, respectively attributed to an increase in headcount from 361 selling, general and administrative employees as of September 30, 2017 to 384 selling, general and administrative employees as of September 30, 2018. Additionally, there was a $0.8 million increase in professional fees and other operating expenses.

As of September 30, 2018, Radius had $276.9 million in cash, cash equivalents, restricted cash, marketable securities and investments. Based upon our cash, cash equivalents, marketable securities and investments balance as of September 30, 2018, we believe that, prior to the consideration of potential proceeds from partnering and/or collaboration activities, we have sufficient capital to fund our development plans, U.S. commercial and other operational activities for not less than twelve months from the date of this press release.

Webcast and Conference Call

In connection with today’s reporting of Third Quarter Financial Results, Radius will host a conference call and live audio webcast at 8:00 a.m. ET today, November 1, 2018, to discuss the commercial outlook for TYMLOS, review the financial results and provide a Company update.

Conference Call Information:

Date: November 1, 2018

Time: 8:00 a.m. ET

Domestic Dial-in Number: (866) 323-7965

International Dial-in Number: (346) 406-0961

Conference ID: 3875777

Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available from November 1, 2018 at 11:00 a.m. ET and will be archived on the Company’s website for 90 days. To access the replay, dial (855) 859-2056 for U.S. or (404) 537-3406 for International, using conference ID number 3875777.

A live audio webcast of the call can be accessed from the Investors section of the Company’s website, www.radiuspharm.com. The full text of the announcement and financial results will also be available on the Company’s website.

Use of Non-GAAP Financial Measures
To supplement our condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), we use the following non-GAAP financial measures: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with Radius’ GAAP financial statements, because it provides greater transparency regarding Radius’ operating performance. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of Radius’ operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three and nine months ended September 30, 2017 and 2018 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

On November 1, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported it will report its third quarter 2018 financial results and business progress on Thursday, November 8, 2018, after the close of the U.S. financial markets (Press release, Tocagen, NOV 1, 2018, View Source;p=RssLanding&cat=news&id=2374897 [SID1234530570]).

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On November 1, 2018 GlycoMimetics (NASDAQ: GLYC)reported that six abstracts covering data from the company’s research and clinical portfolio have been accepted for presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held December 1-4, 2018 in San Diego (Press release, GlycoMimetics, NOV 1, 2018, View Source [SID1234530568]).

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Of particular note, the final analysis from the recently completed Phase 1/2 AML trial of uproleselan, an E-selectin antagonist and the company’s lead wholly owned clinical candidate, has been selected for an oral presentation on Sunday, December 2 during a platform session on novel therapies in AML. Clinical data to be presented include analyses of measurable residual disease (MRD), analyses of response in high-risk subgroups, and updated event-free and overall survival data, which continue to compare favorably with matched historical controls. New data will also be presented showing a correlation between E-selectin ligand expression on leukemic blasts and leukemic stem cells (LSCs), supporting the premise that binding to E-selectin is a mechanism of resistance in AML.

In addition, a poster presentation of complementary work by investigators at the Fred Hutchinson Cancer Research Center will highlight new clinical data showing E-selectin ligand expression on leukemic blasts is associated with poor outcomes in patients at their center with AML. The combined clinical dataset from these two studies provides further scientific rationale for the potential value of uproleselan as a selective, bone-marrow microenvironment-disrupting agent in patients with AML.

"Our oral presentation at ASH (Free ASH Whitepaper) is noteworthy in that we show for the first time that the majority of evaluable patients (11 out of 16) in the R/R cohort and more than half of evaluable patients (5 out of 9) in the newly diagnosed cohort achieved measurable residual disease negativity as assessed by either flow and/or RT-PCR, when uproleselan is added to a standard chemotherapy regimen," noted Helen Thackray, M.D., FAAP, GlycoMimetics Senior Vice President, Clinical Development and Chief Medical Officer. "Furthermore, we now have an updated final analysis of survival that reflects longer term follow up and fewer patients’ data censored, and these data also continue to compare very favorably to historical studies of matched patient groups. As we initiate sites for our Phase 3 study, the ASH (Free ASH Whitepaper) data on the addition of uproleselan to chemotherapy, namely improved remission rates, low mucositis rates, high MRD and transplant rates – as well as promising survival – give us greater confidence in the opportunity to provide a potentially transformative regimen for not only patients with R/R AML but also for the newly diagnosed patients."

According to the Phase 1/2 trial’s lead investigator Daniel J. DeAngelo, M.D., Ph.D., Director of Clinical and Translational Research, Adult Leukemia Program, at the Dana-Farber Cancer Institute and Brigham and Women’s Hospital, "Two of the most striking findings from this study are the number of patients negative for measurable residual disease and the low rate of mucositis. These two potential benefits of the candidate drug, combined with the strong survival signals, distinguish uproleselan from other agents in development. These potential benefits clearly underscore the mechanism of action of uproleselan and, importantly, point to the opportunity we have to help change the face of this disease."

Featured GlycoMimetics’ ASH (Free ASH Whitepaper) data include the following:

R/R AML Cohort: At the recommended Phase 2 dose (RP2D), CR (complete response)/CRi (complete remission with incomplete blood count recovery) rate was 41%, median overall survival was 8.8 months (95% CI 5.7-11.4) and 69% of evaluable patients (11/16) achieved measurable residual disease negativity as assessed by either flow and/or RT-PCR. Overall survival (OS) will be the primary outcome measure in the company’s Phase 3 trial in R/R AML patients, and the data reported today compares to 5.2-5.4 months OS in comparable historical controls. 1,2 (Abstract #331)
Newly Diagnosed (ND) AML Cohort: At the RP2D, CR/CRi rate was 72%, median overall survival was 12.6 months (95% CI 9.9-not reached), Event Free Survival (EFS) was 9.2 months (3.0-12.6) and 56% of evaluable patients (5 out of 9) achieved measurable residual disease negativity as assessed by either flow and/or RT-PCR. EFS will be the primary outcome measure for the interim analysis in the National Cancer Institute clinical trial in newly diagnosed patients, and the data presented today compare to 2-6.5 months for EFS in historical controls which represent lower risk patient populations than those treated in our study. 3,4 (Abstract #331)
Based on data from 89 serially acquired AML patient samples at the Fred Hutchinson Cancer Research Center, mean fluorescence intensity of E-selectin-Fc binding is four-fold higher for R/R patients than for newly diagnosed patients (p=0.0026), suggesting that sequestration in the vascular niche of the bone marrow is associated with chemotherapy resistance. (Abstract #1513)
Other GlycoMimetics ASH (Free ASH Whitepaper) presentations will showcase the potential use of uproleselan to mobilize hematopoietic stem cells with increased reconstitution potential, as well as highlight two new novel compounds discovered by GlycoMimetics that are for the first time being presented to the hematology community. Specifically, Abstract 4678 demonstrates that a highly-potent antagonist of E-selectin, GMI-1687, shows equivalent activity to uproleselan, but at 250-fold lower dose and via subcutaneous administration. In addition, GlycoMimetics researchers will also present preclinical data for a novel dual-function galectin-3 / E-selectin inhibitor, GMI-1757, in a well-established model of venous thrombosis.

Presentation details:

Publication Number: 331
TITLE: Uproleselan (GMI-1271), an E-Selectin Antagonist, Improves the Efficacy and Safety of Chemotherapy in Relapsed/Refractory (R/R) and Newly Diagnosed Older Patients with Acute Myeloid Leukemia: Final, Correlative and Subgroup Analyses
Session Name: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Combination Therapy
Session Date: Sunday, December 2, 2018
Session Time: 9:30 AM – 11:00 AM PST
Presentation Time: 9:30 AM
Room: Manchester Grand Hyatt San Diego, Grand Hall B

Publication Number: 1513
TITLE: E-Selectin Ligand Expression by Leukemic Blasts Is Associated with Prognosis in Patients with AML
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: Poster I
Session Date: Saturday, December 1, 2018
Presentation Time: 6:15 PM – 8:15 PM PST
Location: San Diego Convention Center, Hall GH

Publication Number: 2211
TITLE: A Novel Glycomimetic Compound (GMI-1757) with Dual Functional Antagonism to E-Selectin and Galectin-3 Demonstrates Inhibition of Thrombus Formation in an Inferior Vena Cava Model
Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster I
Session Date: Saturday, December 1, 2018
Presentation Time: 6:15 PM – 8:15 PM PST
Location: San Diego Convention Center, Hall GH

Publication Number: 3846
TITLE: The Vascular Bone Marrow Niche Influences Outcome in Chronic Myeloid Leukemia
Session Name: 506. Hematopoiesis and Stem Cells: Microenvironment, Cell Adhesion, and Stromal Stem Cells: Poster III
Session Date: Monday, December 3, 2018
Presentation Time: 6:00 PM – 8:00 PM PST
Location: San Diego Convention Center, Hall GH

Publication Number: 4552
TITLE: Vascular E-Selectin Acts as a Gatekeeper Inducing Commitment and Loss of Self-Renewal in HSC Transmigrating through the Marrow Vasculature
Session Name: 711. Cell Collection and Processing: Poster III
Session Date: Monday, December 3, 2018
Presentation Time: 6:00 PM – 8:00 PM PST
Location: San Diego Convention Center, Hall GH

Publication Number: 4678
TITLE: A Novel and Potent Inhibitor of E-Selectin, GMI-1687, Attenuates Thrombus Formation and Augments Chemotherapeutic Intervention of AML in Preclinical Models Following Subcutaneous Administration
Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster III
Session Date: Monday, December 3, 2018
Presentation Time: 6:00 PM – 8:00 PM PST
Location: San Diego Convention Center, Hall G

Meeting abstracts are available on ASH (Free ASH Whitepaper)’s website.

1 Feldman et al, J Clin Oncol. 2005 Jun 20;23(18):4110-6.
2 Greenberg et al, J Clin Oncol. 2004 Mar 15;22(6):1078-86.
3 Lowenberg et al, N Engl J Med. 2009 Sep 24;361(13).
4 Lancet et al, Blood. 2014 May 22;123(21):3239-46.

About Uproleselan (GMI-1271)

Uproleselan (yoo’ pro le’sel an) is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed/refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better than expected remission rates and overall survival compared to historical controls, which have been derived from results from third party clinical trials evaluating standard chemotherapy, as well as lower than expected induction-related mortality rates. Treatment in these patient populations was generally well tolerated, with fewer than expected adverse effects. The U.S. Food and Drug Administration (FDA) has granted uproleselan Breakthrough Therapy Designation for the treatment of adult AML patients with relapsed/refractory (R/R) disease. GlycoMimetics is currently implementing a comprehensive development program across the clinical spectrum of AML. This includes a company sponsored Phase 3 trial in R/R AML and two consortia-sponsored trials in newly diagnosed patients. One consortium trial is being sponsored by the NCI and will enroll newly diagnosed patients fit for intensive chemotherapy. The other trial is sponsored by the HOVON group in Europe and will enroll newly diagnosed patients unfit for intensive chemotherapy.

On November 1, 2018 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, 2018 (Press release, Fate Therapeutics, NOV 1, 2018, View Source [SID1234530566]).

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"We continue to see strong momentum in the enrollment of our Phase 2 PROTECT study of ProTmune, and an encouraging set of initial clinical data for FATE-NK100 is emerging across the dose-escalation phases of three Phase 1 clinical trials. In addition, we are poised to achieve a significant milestone for Fate Therapeutics as well as the entire cell therapy field, as we continue working with the FDA on the allowance of our landmark IND application for FT500, a first-of-kind, off-the-shelf NK cell product derived from a clonal master iPSC line," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "At ASH (Free ASH Whitepaper), we plan to share preclinical data across our entire pipeline of off-the-shelf NK cell and CAR T-cell product candidates that demonstrate the unique value in using clonal engineered master iPSC lines as a renewable source for manufacture and delivery of cell-based cancer immunotherapies."

Clinical Programs

Exceeded 50% Enrollment in Phase 2 PROTECT Study of ProTmune. The randomized, controlled and double-blinded Phase 2 PROTECT study of ProTmune is over 50% enrolled. The clinical trial is intended to enroll a total of 60 adult subjects with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation (HCT). Subjects are being randomized, in a 1:1 ratio, to receive either ProTmune or a conventional matched unrelated donor hematopoietic cell graft. New clinical data from the seven subjects receiving ProTmune in the Phase 1 PROTECT study, including data on key secondary endpoints assessing disease-free survival and freedom from chronic graft-versus-host disease (GvHD), cancer relapse, and death at one-year following HCT, will be featured at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in a poster presentation.
20th Subject Treated across Three Phase 1 Studies of FATE-NK100. The twentieth subject has been treated with FATE-NK100, the Company’s first-in-class, donor-derived adaptive memory NK cell cancer immunotherapy, across the dose-escalation phases of three Phase 1 clinical trials. At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, the Company plans to hold an investor event and share new clinical data of FATE-NK100, including safety, persistence and anti-tumor activity, in advanced hematologic malignancies and solid tumors. An oral presentation at the 2018 ASH (Free ASH Whitepaper) Annual Meeting will describe a next-generation, GMP-compliant protocol, established by Dr. Karl-Johan Malmberg under the Company’s research collaboration with Oslo University Hospital, that enables robust ex vivo expansion of adaptive memory NK cells having homogeneous expression of a single inhibitory killer cell immunoglobulin-like receptor (KIR).
Universal Off-the-Shelf Cancer Immunotherapy Preclinical Pipeline

Submitted First-of-Kind IND Application to FDA for FT500. In July, the Company submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for FT500, a universal, off-the-shelf NK cell product derived from a clonal master induced pluripotent stem cell (iPSC) line. In response to a request by the FDA, the Company is conducting additional adventitious agents testing of the master iPSC bank used for the production of FT500, and intends to submit these test results to the FDA during the fourth quarter of 2018. Upon FDA allowance of the IND, the Company expects to begin clinical investigation of FT500 in combination with FDA-approved checkpoint inhibitors in subjects with advanced solid tumors.
Achieved IND-Enabling Milestone under FT516 CIRM Grant. In September, the Company received a $1.1 million milestone payment under its California Institute for Regenerative Medicine (CIRM) award for the preclinical development of FT516, a universal, off-the-shelf NK cell product candidate derived from a clonal master iPSC line engineered to uniformly express a high-affinity, non-cleavable CD16 Fc receptor. Since CD16 binds to the Fc region of tumor-targeted antibodies, FT516 can be combined with FDA-approved monoclonal antibody therapy to target a broad spectrum of tumor-associated antigens. The Company expects to submit an IND application to the FDA by the end of 2018 for first-in-human clinical investigation of FT516 in combination with CD20 antibody rituximab and with SLAMF7 antibody elotuzumab.
Five Presentations covering iPSC Product Platform Scheduled for ASH (Free ASH Whitepaper). Two oral presentations, including new preclinical data of FT500 in combination with checkpoint inhibitors and initial preclinical data of engineered iPSC-derived NK cells in combination with target-cell specific engagers, were accepted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting. Additionally, three poster presentations on other product candidates emerging from the Company’s iPSC product platform, including the Company’s first iPSC-derived chimeric antigen receptor (CAR) T-cell (FT819) and CAR NK cell (FT519) product candidates, are scheduled for presentation.
Corporate Highlights

Entered into iPSC-derived CAR T-cell Collaboration with ONO Pharmaceutical. In September 2018, the Company entered into a Collaboration and Option Agreement with Ono Pharmaceutical Co. Ltd. for the joint development and commercialization of two off-the-shelf, iPSC-derived CAR T-cell product candidates. Fate Therapeutics is entitled to receive up to $70 million during the preclinical option stage of the collaboration. In addition, in connection with the development and commercialization of the product candidates, the Company is eligible to receive up to $1.2 billion in aggregate milestone payments, plus tiered royalties on net sales by Ono.
Extended iPSC Technology Leadership Position to include CRISPR-based Cell Reprogramming. In September, the Company exclusively licensed intellectual property from the J. David Gladstone Institutes that covers the generation of iPSCs using CRISPR-mediated gene activation. This new approach uses CRISPR to induce pluripotency by directly targeting a specific location of the genome and activating endogenous gene expression, and does not rely on established methods of cellular reprogramming that require the transduction of multiple transcription factors.
Completed $144 Million Common Stock Offering. In September, the Company closed an underwritten public offering of 10,648,149 shares of its common stock at a public offering price of $13.50 per share.
Third Quarter 2018 Financial Results

Cash & Short-term Investment Position: Cash, cash equivalents and short-term investments as of September 30, 2018 were $211.2 million compared to $100.9 million as of December 31, 2017. The increase was primarily driven by $134.9 million in net cash proceeds received by the Company from its September 2018 public offering of common stock. These proceeds were offset by the Company’s use of cash to fund operating activities.
Total Revenue: Revenue was $1.0 million for the third quarter of 2018 as well as for the same period in 2017. All revenue was derived from the Company’s research collaboration and license agreement with Juno Therapeutics.
R&D Expenses: Research and development expenses were $13.6 million for the third quarter of 2018, compared to $8.6 million for the same period in 2017. In the third quarter of 2018, the Company incurred a one-time $1.4 million expense associated with the in-license of intellectual property from the J. David Gladstone Institutes covering the use of CRISPR for cellular reprogramming and iPSC generation. The remaining increase in R&D expenses was primarily attributable to an increase in expenses associated with the clinical development of FATE-NK100 and with the preclinical development of the Company’s iPSC-derived product candidates, including regulatory and manufacturing activities to support the submission of its FT500 IND application, and in employee compensation associated with growth in headcount.
G&A Expenses: General and administrative expenses were $4.1 million for the third quarter of 2018, compared to $2.8 million for the same period in 2017. The increase in G&A expenses was primarily attributable to an increase in advisory fees, including audit and legal fees, and in employee compensation.
Shares Outstanding: Common shares outstanding were 64.5 million as of September 30, 2018 and 52.6 million as of December 31, 2017. Preferred shares outstanding as of September 30, 2018 and December 31, 2017 were 2.8 million, each of which is convertible into five shares of common stock. All preferred shares outstanding are from the Company’s sale and issuance of non-voting Class A convertible preferred stock to Redmile Group, LLC in November 2016.
Today’s Conference Call and Webcast
The Company will conduct a conference call today, Thursday, November 1, 2018 at 5:00 p.m. ET to review financial and operating results for the quarter ended September 30, 2018. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 6998539. 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 ProTmune
ProTmune is an investigational next-generation hematopoietic cell graft for the prevention of acute graft-versus-host disease (GvHD) in patients undergoing allogeneic hematopoietic cell transplantation (HCT). ProTmune is manufactured by pharmacologically modulating a donor-sourced, mobilized peripheral blood graft ex vivo with two small molecules (FT1050 and FT4145) to decrease the incidence and severity of acute GvHD while maintaining the anti-leukemia activity of the graft. ProTmune has been granted Orphan Drug and Fast Track Designations by the U.S. Food and Drug Administration, and Orphan Medicinal Product Designation by the European Commission. ProTmune is currently being investigated in a randomized, controlled and double-blinded Phase 2 clinical trial in adult subjects with hematologic malignancies undergoing matched unrelated donor HCT.

About FATE-NK100
FATE-NK100 is an investigational, first-in-class, allogeneic donor-derived natural killer (NK) cell cancer immunotherapy comprised of adaptive memory NK cells, a highly specialized and functionally distinct subset of activated NK cells expressing the maturation marker CD57. Higher frequencies of CD57+ NK cells in the peripheral blood or tumor microenvironment in cancer patients have been linked to better clinical outcomes. In August 2017, non-clinical data describing the unique properties and anti-tumor activity of FATE-NK100 were published by Cancer Research (doi:10.1158/0008-5472.CAN-17-0799), a peer-reviewed journal of the American Association of Cancer Research. Three clinical trials of FATE-NK100 are currently being conducted: VOYAGE for the treatment of refractory or relapsed acute myelogenous leukemia; APOLLO for the treatment of recurrent ovarian cancer; and DIMENSION for the treatment of advanced solid tumors, including in combination with monoclonal antibody therapy.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary iPSC product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of 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 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 to treat many patients. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 100 issued patents and 100 pending patent applications.

On November 1, 2018 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported operating results for third-quarter 2018 and provided an update on the Company’s commercial products and development programs (Press release, TESARO, NOV 1, 2018, View Source [SID1234530565]).

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"In the third quarter, we launched several initiatives to grow the use of ZEJULA for recurrent ovarian cancer and we continued to execute on our development strategies focused on gynecologic and lung cancers as we approach a period of significant data readouts," said Lonnie Moulder, CEO of TESARO. "Following results of the Phase 3 PRIMA trial next year, we intend for ZEJULA to benefit patients throughout all stages of their ovarian cancer journey, including first-line, recurrent, and late-line treatment settings. Our immuno-oncology pipeline continues to advance, led by our anti-PD-1 antibody, TSR-042, for which we are on track to submit a BLA next year. We look forward to initial data from the Phase 1 AMBER trial of our anti-TIM-3 antibody, TSR-022, in combination with TSR-042, which will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting next week."

Recent Business Highlights

ZEJULA net revenue increased 61% year-over-year to $63.2 million for the third quarter of 2018. In the U.S. and Europe, approximately 10,000 patients have been treated with ZEJULA since its launch in April 2017. ZEJULA is now approved in 33 countries and is reimbursed and launched in Germany, the U.K., Italy and several other European countries.
Safety data were presented from the Phase 3 PRIMA trial of ZEJULA monotherapy in first-line ovarian cancer during the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress in October. Data demonstrated a favorable tolerability profile for niraparib when dosed according to a patient’s weight and platelet count compared to a fixed starting dose. The PRIMA Phase 3 trial is fully enrolled and top-line results are expected in late 2019.
Based upon the responses observed in the first stage of the study, the second stage of the Phase 2 JASPER study was initiated, which evaluates ZEJULA in combination with TSR-042 as a first-line treatment for patients with non-small cell lung cancer and high levels of PD-L1 expression.
Data were presented from the GARNET trial of TSR-042 monotherapy at ESMO (Free ESMO Whitepaper) and demonstrated TSR-042 is well tolerated and has robust clinical activity in patients with MSI-H endometrial cancer.
Zai Lab Limited announced ZEJULA approval in Hong Kong on October 22, 2018. ZEJULA is the first and only PARP inhibitor approved in Hong Kong for the maintenance treatment of platinum-sensitive relapsed ovarian cancer regardless of BRCA mutation status.
In October, TESARO and actress Cobie Smulders launched Not on My Watch, a national movement to empower the ovarian cancer community, especially women with recurrent ovarian cancer, to take informed and proactive steps against the threat of disease recurrence.
Development milestones were achieved in October related to Janssen’s ongoing GALAHAD trial of niraparib monotherapy for the treatment of men with metastatic castration-resistant prostate cancer (mCRPC) and DNA-repair anomalies. The achievement of these milestones triggered an $18 million payment from Janssen to TESARO.
Third Quarter 2018 Financial Results

TESARO reported net product revenue of $63.6 million for the third quarter of 2018, compared to a total of $41.8 million for the third quarter of 2017. ZEJULA net revenue increased 61% to $63.2 million for the third quarter of 2018, compared to $39.4 million for the third quarter of 2017. Cost of goods sold increased to $14.2 million for the third quarter of 2018, compared to $6.2 million for the same period in 2017, primarily related to increased volume and new supplier set-up expenses.

Research and development expenses increased to $94.2 million for the third quarter of 2018, compared to $73.4 million for the third quarter of 2017, driven primarily by higher manufacturing and clinical development costs associated with TSR-042, TSR-022, and ZEJULA, and research collaborations.

Selling, general and administrative expenses increased to $93.5 million for the third quarter of 2018, compared to $84.0 million for the third quarter of 2017, primarily due to increased headcount and activities in support of the launches of ZEJULA in the U.S. and Europe.

Operating expenses as described above include total non-cash, stock-based compensation expense of $24.8 million for the third quarter of 2018, compared to $25.0 million for the third quarter of 2017.

Net loss totaled $137.1 million, or ($2.49) per share, for the third quarter of 2018, compared to a net loss of $25.3 million, or ($0.47) per share, for the third quarter of 2017. The increase in net loss was primarily due to the $100.0 million up-front payment received and recorded as revenue in the third quarter of 2017 as part of the license agreement with Takeda, partially offset by a $17.6 million gain in the third quarter of 2018 associated with the divestiture of VARUBI in the U.S., for which TESARO received an up-front payment of $35 million.

(in thousands, except per share amounts) Three Months Ended
September 30,
2018
2017

Product revenue, net
ZEJULA $ 63,226 $ 39,375
VARUBI/VARUBY $ 386 $ 2,380
Total product revenue, net $ 63,612 $ 41,755
License, collaboration, and other revenue $ 787 $ 101,011
Total revenues $ 64,399 $ 142,766

Net loss $ (137,088 ) $ (25,277 )

Net loss per share, basic and diluted $ (2.49 ) $ (0.47 )

(in thousands) Three Months Ended
September 30,
2018 2017
Cost of sales – product $ 14,225 $ 6,216
Cost of sales – intangible asset amortization $ 728 $ 1,254
Research and development (R&D) $ 94,188 $ 73,388
Selling, general and administrative (SG&A) $ 93,497 $ 83,998
Acquired in-process R&D $ - $ -
Cash and Cash Equivalents

As of September 30, 2018, TESARO had approximately $476.8 million in cash and cash equivalents and approximately 55.0 million outstanding shares of common stock.

2018 Financial Guidance

TESARO is updating its 2018 financial guidance for ZEJULA:

Total Revenue, net, worldwide (FY) $258 to $265 million
(previously $250 to $265 million)
ZEJULA (FY) $233 to $238 million
(previously $225 to $235 million)
ZEJULA (Q4) $67 to $72 million
Other revenue, including licensing and
VARUBY oral (FY) $25 to $27 million
(previously $25 to $30 million)
Interest expense (FY) Approximately $60 million,
including non-cash interest expense of $14 million
In the third quarter, TESARO’s cash and cash equivalents balance declined by approximately $98 million. TESARO anticipates year-end 2018 cash and cash equivalents to be approximately $400 million.

Key Development Milestones

Gynecologic Cancers:

Submit QUADRA sNDA for treatment of late-line ovarian cancer beyond BRCAmut near year-end
Results of the AVANOVA Phase 2 study of ZEJULA in combination with bevacizumab for treatment of recurrent ovarian cancer to be submitted for presentation at a medical meeting in 1H 2019
Report Phase 2 OVARIO results of ZEJULA in combination with bevacizumab in first-line ovarian cancer maintenance in late 2019
Report Phase 3 PRIMA results of ZEJULA in first-line ovarian cancer maintenance in late 2019
Lung Cancer:

Report initial data for the AMBER trial of TSR-022 in combination with TSR-042 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) Annual Meeting on November 9
Report additional data from the NSCLC cohort of the GARNET trial of TSR-042 at SITC (Free SITC Whitepaper)
Initiate Phase 2 registration enabling trial of TSR-042 versus standard of care in first-line NSCLC in early 2019
Report additional data from the Phase 2 JASPER study of ZEJULA in combination with anti-PD-1 at a medical meeting in 1H 2019
Breast Cancer:

Submit BRAVO data for ZEJULA in germline BRCAmut breast cancer patients for publication in Q4 2018
Complete protocol development for registration study of ZEJULA in combination with TSR-042 and other agents in breast cancer
Prostate Cancer:

Janssen to advance GALAHAD trial of ZEJULA in mCRPC and DNA-repair anomalies to support global regulatory filings in 2019
Planning underway by Janssen for a future Phase 3 trial that will assess the clinical benefit of niraparib in combination with abiraterone acetate + prednisone in mCRPC patients
Immuno-oncology Pipeline:

Continue dose-escalation in the CITRINO trial (combination of TSR-033 plus TSR-042) and report Phase 1 monotherapy dose-escalation data for TSR-033 at SITC (Free SITC Whitepaper)
Advance IND-enabling studies of PD-1/LAG-3 bi-specific antibody (TSR-075)
Today’s Conference Call and Webcast

TESARO will host a conference call to discuss the Company’s third quarter operating results and provide an update on the Company’s commercial products and development programs today at 4:15 P.M. Eastern time. The accompanying slide presentation and live webcast of the conference call can be accessed by visiting the TESARO website at www.tesarobio.com. The call can be accessed by dialing (877) 853-5334 (U.S. and Canada) or (970) 315-0307 (international). A replay of the webcast will be archived on the Company’s website for 30 days following the call.

About ZEJULA (niraparib)

ZEJULA (niraparib) is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. In preclinical studies, ZEJULA concentrates in the tumor relative to plasma, delivering greater than 90% durable inhibition of PARP 1/2 and a persistent antitumor effect. Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML), including some fatal cases, was reported in patients treated with ZEJULA. Discontinue ZEJULA if MDS/AML is confirmed. Hematologic adverse reactions (thrombocytopenia, anemia and neutropenia), as well as cardiovascular effects (hypertension and hypertensive crisis) have been reported in patients treated with ZEJULA. Monitor complete blood counts to detect hematologic adverse reactions, as well as to detect cardiovascular disorders, during treatment. ZEJULA can cause fetal harm and females of reproductive potential should use effective contraception. Please see full prescribing information, including additional important safety information, available at www.zejula.com.