On November 1, 2017 Geron Corporation (Nasdaq:GERN) reported financial results for the three and nine months ended September 30, 2017 (Press release, Geron, NOV 1, 2017, View Source [SID1234521431]).

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For the third quarter of 2017, the company reported a net loss of $6.9 million, or $0.04 per share, compared to $3.6 million, or $0.02 per share, for the comparable 2016 period. Net loss for the first nine months of 2017 was $20.5 million, or $0.13 per share, compared to $21.1 million, or $0.13 per share, for the comparable 2016 period. The company ended the third quarter of 2017 with $112.7 million in cash and investments.

Revenues for the three and nine months ended September 30, 2017 were $163,000 and $874,000, respectively, compared to $5.1 million and $6.1 million for the comparable 2016 periods. Revenues for the three and nine month periods ending September 30, 2016 included license fee revenue of $5.0 million in connection with an upfront payment under a license agreement signed in September 2016 with Janssen Pharmaceuticals, Inc. for certain rights to specialized oligonucleotide backbone chemistry and novel amidates.

Total operating expenses for the three and nine months ended September 30, 2017 were $7.4 million and $22.3 million, respectively, compared to $9.0 million and $27.9 million for the comparable 2016 periods. Research and development expenses for the three and nine months ended September 30, 2017 were $2.6 million and $8.5 million, respectively, compared to $4.3 million and $13.9 million for the comparable 2016 periods. The decrease in research and development expenses for the three and nine month periods ending September 30, 2017, compared to the same periods in 2016, primarily reflects lower costs for the company’s proportionate share of clinical development expenses under the imetelstat collaboration with Janssen Biotech, Inc. and reduced personnel related expenses. General and administrative expenses for the three and nine months ended September 30, 2017 were $4.8 million and $13.8 million, respectively, compared to $4.7 million and $14.0 million for the comparable 2016 periods. The increase in general and administrative expenses for the three-month period ending September 30, 2017, compared to the same period in 2016, primarily reflects higher non-cash stock-based compensation expense. The decrease in general and administrative expenses for the nine-month period ending September 30, 2017, compared to the same period in 2016, primarily reflects lower consulting and legal costs, partially offset by higher non-cash stock-based compensation expense.

Interest and other income for the three and nine months ended September 30, 2017 was $363,000 and $1.0 million, respectively, compared to $322,000 and $871,000 for the comparable 2016 periods. The increase in interest and other income for the three and nine month periods ending September 30, 2017, compared to the same periods in 2016, primarily reflects higher yields on the company’s marketable securities portfolio.

Conference Call and Webcast

At 4:30 p.m. ET on November 1, 2017, Geron’s management will host a conference call to discuss the company’s third quarter results as well as recent events.

Participants can access the conference call live via telephone dialing 877-303-9139 (U.S.); 760-536-5195 (international). The conference ID is 1056465. A live audio-only webcast is also available through the Investors section of the company’s website at www.geron.com or at View Source The audio webcast of the conference call will be available for replay approximately one hour following the live broadcast through December 1, 2017.

On November 1, 2017 Foundation Medicine, Inc. (NASDAQ: FMI) reported financial and operating results for its third quarter ended September 30, 2017. Results and business highlights for the quarter included (Press release, Foundation Medicine, NOV 1, 2017, View Source [SID1234521430]):

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Achieved third quarter revenue of $42.7 million, 45% year-over-year growth;
Reported 17,474 clinical tests in the third quarter, 50% year-over-year growth;
Presented validation data for FoundationOne CDx at the World Conference on Lung Cancer which demonstrated high concordance with multiple FDA-approved companion diagnostics across multiple solid tumor cancer types. FoundationOne CDx is currently under the Parallel Review process with FDA and CMS;
Presented validation data for a novel assay measuring tumor mutational burden in blood (bTMB) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) providing evidence that response to immunotherapy can be predicted using a blood sample. Based on these findings, the bTMB assay is being integrated as part of Roche/Genentech’s prospective, randomized Phase III Blood First Assay Screening Trial (BFAST) as a companion diagnostic assay to investigate bTMB as a non-invasive biomarker of response to first-line atezolizumab in advanced NSCLC patients; and
Published 19 manuscripts in high-quality, peer-reviewed journals and delivered 21 podium and poster talks at various medical and scientific meetings.
“Foundation Medicine made great progress in the third quarter as evidenced by record revenue and clinical volume,” said Troy Cox, chief executive officer of Foundation Medicine. “Our recent accomplishments, including progress with the parallel review process with FDA and CMS for FoundationOne CDx, expanding relationships with our biopharma partners, and driving continued innovation in our molecular information solutions pipeline with bTMB, continue to position our company for further growth, and competitive differentiation to transform cancer care.”

Foundation Medicine reported total revenue of $42.7 million in the third quarter of 2017, compared to $29.4 million in the third quarter of 2016. Revenue from biopharmaceutical customers was $29.6 million in the third quarter of 2017, compared to $20.7 million in the third quarter of 2016. The results of 2,817 tests were reported to biopharmaceutical customers in this year’s third quarter.

Revenue from clinical testing in the third quarter of 2017 was $13.1 million, compared to $8.7 million in the third quarter of 2016. The company reported 17,474 tests to clinicians in the third quarter of 2017, a 50% increase from the same quarter last year. This number includes 14,398 FoundationOne tests, 1,478 FoundationOne Heme tests, 1,488 FoundationACT tests, and 110 FoundationFocus CDxBRCA tests.

Total operating expenses for the third quarter of 2017 were approximately $56.0 million, compared with $44.9 million for the third quarter of 2016. The increase in operating expenses was partially driven by investments in product development such as FoundationOne CDx and bTMB, investments in the company’s technology infrastructure, and certain non-recurring cash and non-cash expenses.

Net loss was approximately $32.6 million in the third quarter of 2017, or a $0.90 loss per share. At September 30, 2017, the company held approximately $76.8 million in cash, cash equivalents and marketable securities.

Based on the new revenue reporting the company initiated last quarter, Molecular Information Services revenue was $28.4 million, including $13.1 million in revenue generated from our clinical customers, and $15.3 million in revenue generated from our biopharma customers during the third quarter. Pharma Research and Development Services revenue was $14.3 million.

2017 Outlook

Foundation Medicine’s business and financial outlook for 2017 is the following:

The company expects 2017 revenue will be in the range of $135 million to $145 million.
The company is increasing clinical volume guidance and now expects to deliver between 64,000 and 66,000 clinical tests in 2017.
The company expects 2017 operating expenses will be in the range of $215 million and $225 million.
The company expects to advance its comprehensive genomic profiling assay, FoundationOne CDx, through the FDA and CMS Parallel Review process with a decision anticipated in the fourth quarter of 2017.
The company expects to continue reimbursement progress and pursue additional coverage decisions for its CGP assays.
Conference Call and Webcast Details

The company will conduct a conference call today, Wednesday, November 1st at 4:30 p.m. Eastern Time to discuss its financial performance for the 2017 third quarter and other business activities, including matters related to future performance. To access the conference call via phone, dial 1-877-270-2148 from the United States or dial 1-412-902-6510 internationally. Dial in approximately ten minutes prior to the start of the call. The live, listen-only webcast of the conference call may be accessed by visiting the investors section of the company’s website at investors.foundationmedicine.com. A replay of the webcast will be available shortly after the conclusion of the call and will be archived on the company’s website for two weeks following the call.

On November 1, 2017 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, 2017 (Press release, Fate Therapeutics, NOV 1, 2017, View Source [SID1234521429]).

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"We are poised to release initial Phase 1 clinical data for FATE-NK100 and ProTmune at prominent scientific conferences during the coming weeks," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "Additionally, we are pleased that two first-of-kind product candidates from our proprietary iPSC-derived cancer immunotherapy pipeline have been selected for oral presentations at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Thousands of doses of homogeneous drug product can be produced from a clonal iPSC master cell line in a single manufacturing run. This represents a transformative approach to enable off-the-shelf delivery of cancer immunotherapies that are uniformly engineered and identical in composition from dose-to-dose across patients. At ASH (Free ASH Whitepaper) we will be unveiling exciting new preclinical and manufacturing data to support our 2018 path to clinic for iPSC-derived NK- and T-cell product candidates."

Recent Highlights & Program Updates

Initial Clinical Data from VOYAGE Study of FATE-NK100 in AML to be Presented at SITC (Free SITC Whitepaper) 2017. VOYAGE is an open-label, accelerated dose-escalation clinical trial of FATE-NK100, the Company’s first-in-class adaptive memory natural killer (NK) cell product candidate, for the treatment of refractory or relapsed acute myelogenous leukemia (AML).

FATE-NK100 has advanced through the first two of three dose cohorts in VOYAGE. The Company will present the post-manufacturing potency, in vivo persistence and anti-tumor activity of FATE-NK100 from the first two subjects at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting during a poster session on November 10. The peer-reviewed non-clinical data describing the unique properties and anti-tumor activity of FATE-NK100 were published in Cancer Research in August.

APOLLO Study of FATE-NK100 in Recurrent Ovarian Cancer Open for Enrollment. In October, enrollment was opened in the APOLLO study of FATE-NK100 for the treatment of women with ovarian cancer resistant to, or recurrent on, platinum-based treatment. APOLLO is designed to evaluate the safety and determine the maximum dose of a single infusion of FATE-NK100 when administered directly into the peritoneum in an outpatient setting.

Intraperitoneal delivery of NK cells is a novel strategy intended to promote co-localization with tumor cells and maximize NK cell persistence and anti-tumor activity. Other study endpoints include objective response rate at 28 days post-infusion and progression-free and overall survival.

First Subject Treated in PROTECT Phase 2 Efficacy Stage. PROTECT is a combined open-label Phase 1 / blinded Phase 2 clinical trial of ProTmune, a next-generation hematopoietic cell graft for patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation (HCT). In October, the first subject was treated in the randomized, controlled and blinded Phase 2 stage. The Phase 2 stage is assessing the safety and efficacy of ProTmune in 60 subjects, where subjects are being randomized, in a 1:1 ratio, to receive either ProTmune or a conventional matched unrelated donor mobilized peripheral blood cell graft. The primary efficacy endpoint is incidence of acute graft-versus-host disease (GvHD) by Day 100 post-HCT, where prospective clinical studies have shown that 40% to 80% of patients undergoing matched unrelated donor HCT experience Grades 2-4 acute GvHD.

Day 100 Data from PROTECT Phase 1 Stage to be Presented at 2017 ASH (Free ASH Whitepaper). The Company will present data on all seven subjects administered ProTmune in the Phase 1 stage of PROTECT at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting during a poster session on December 11. Key clinical outcomes, including incidence of acute GvHD, cancer relapse and survival, at 100 days following HCT will be released. An ASH (Free ASH Whitepaper) abstract released today highlighted early data on the first five Phase 1 subjects, three of whom had not yet reached Day 100, as of a July 31, 2017 data cut-off.

First iPSC-derived T-Cell Product Candidate to be Showcased during Oral Presentation at 2017 ASH (Free ASH Whitepaper). An oral presentation will describe the generation of CD8αβ+ T cells from an induced pluripotent stem cell (iPSC) line engineered to express a chimeric antigen receptor (CAR). This breakthrough was led by Dr. Michel Sadelain, MD, PhD, Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center (MSK), under the Company’s multi-year sponsored research collaboration with MSK. The Company’s first iPSC-derived CAR T-cell product candidate FT819, which is derived from a clonal iPSC master cell line engineered to express a CAR targeting CD19 and edited to remove T-cell receptor (TCR) expression, is undergoing preclinical development.

First iPSC-derived NK Cell Product Candidate FT500 to be Showcased during Oral Presentation at 2017 ASH (Free ASH Whitepaper). An oral presentation by Jeffrey S. Miller, MD, Deputy Director of the Masonic Cancer Center, University of Minnesota, will describe the production under current good manufacturing practice (cGMP) conditions of FT500, the Company’s first-of-kind NK cell product candidate derived from a clonal iPSC master cell line. Fate Therapeutics plans to file a landmark Investigational New Drug (IND) application with the U.S. Food & Drug Administration (FDA) in the first quarter of 2018 to initiate first-in-human clinical investigation of FT500 in combination with FDA-approved checkpoint inhibitors for the treatment of advanced solid tumors.

Key Patent Issued for Enhanced Genetic Engineering of CD34+ Cells. In August, the Company announced that the U.S. Patent and Trademark Office issued U.S. Patent No. 9,675,641 covering the use of prostaglandins as viral transduction enhancers for the genetic modification of CD34+ hematopoietic cells. The patent, which broadly covers methods of using prostaglandins to enhance ex vivo genetic engineering of hematopoietic cells using viral vectors, is owned by the Indiana University Research and Technology Corporation and is licensed exclusively to Fate Therapeutics in all fields. Investigators recently highlighted in Molecular Therapy that this practice consistently increased transduction efficiency in primary CD34+ cells sourced from multiple normal human donors and from patients with β-thalassemia or sickle cell disease, concluding that prostaglandins may be critical to ensuring successful clinical gene therapy using lentivirus-modified CD34+ cells.

Third Quarter 2017 Financial Results

Cash & Short-term Investment Position: Cash, cash equivalents and short-term investments as of September 30, 2017 were $69.2 million compared to $92.1 million as of December 31, 2016. The decrease was primarily driven by the Company’s use of cash to fund operating activities and to service principal and interest obligations under its loan agreement with Silicon Valley Bank. This use was offset by $7.5 million in net cash proceeds received by the Company in July 2017 in connection with the amendment of its loan agreement with Silicon Valley Bank.
Total Revenue: Revenue was $1.0 million for the third quarter of 2017 and as well as for the comparable period in 2016. All revenue was derived from the Company’s research collaboration and license agreement with Juno Therapeutics.

Total Operating Expenses: Total operating expenses were $11.4 million for the third quarter of 2017 compared to $9.4 million for the comparable period in 2016. Operating expenses for the third quarter of 2017 included $0.9 million of stock compensation expense, compared to $0.8 million for the comparable period in 2016.

R&D Expenses: Research and development expenses were $8.6 million for the third quarter of 2017 compared to $6.8 million for the comparable period in 2016. The increase in R&D expenses was primarily related to an increase in third-party service provider fees to support the clinical development of ProTmune and FATE-NK100 and the preclinical advancement of the Company’s off-the-shelf iPSC-derived cellular immunotherapy programs, and an increase in facilities costs associated with the expansion of the Company’s laboratory space.

G&A Expenses: General and administrative expenses were $2.8 million for the third quarter of 2017 compared to $2.6 million for the comparable period in 2016. The increase in G&A expenses was primarily related to an increase in employee compensation and benefits expense, including employee stock-based compensation expense, and an increase in facilities costs associated with the expansion of the Company’s office space.

Shares Outstanding: Common shares outstanding were 41.5 million as of September 30, 2017 and 41.4 million as of December 31, 2016. Preferred shares outstanding as of September 30, 2017 and December 31, 2016 were 2.82 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, Wednesday, November 1, 2017 at 5:00 p.m. ET to review financial and operating results for the quarter ended September 30, 2017. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 9892619. 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-NK100
FATE-NK100 is a first-in-class 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. FATE-NK100 is produced through a feeder-free, seven-day manufacturing process during which NK cells sourced from a healthy donor are activated ex vivo with pharmacologic modulators.

About ProTmune
ProTmune is an investigational next-generation hematopoietic cell graft for the prevention of acute graft-versus-host disease (GvHD) in subjects 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 enhance the biological properties and therapeutic function of the graft. Acute GvHD is a severe immunological disease that commonly arises in patients during the first weeks following allogeneic HCT when the newly-transplanted donor immune cells attack the patient’s tissues and organs, resulting in a potentially fatal immune system reaction. The disease is the leading cause of early morbidity and mortality in matched unrelated donor transplant, and there are currently no FDA-approved preventive therapies and very few treatment options for acute GvHD. ProTmune has been granted Orphan Drug and Fast Track Designations by the FDA, and Orphan Medicinal Product Designation by the European Medicines Agency.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables genetic engineering, high-throughput single-cell isolation and clonal selection of human iPSCs and supports long-term maintenance of human iPSCs as master pluripotent cell lines. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. Similar to master cell lines used for the manufacture of monoclonal antibodies, clonal iPSC master cell lines can serve as a renewable cell source for the consistent and repeated manufacture of homogeneous cell products with the potential to treat many different diseases and many thousands of patients in an off-the-shelf manner. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 90 issued patents and 100 pending patent applications.

On November 1, 2017 Exelixis, Inc. (NASDAQ: EXEL) reported financial results for the third quarter of 2017 and provided an update on progress toward fulfilling its key corporate objectives, as well as commercial and clinical development milestones (Press release, Exelixis, NOV 1, 2017, View Source [SID1234521427]).

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Exelixis is focused on maximizing the opportunity for its two internally discovered compounds, cabozantinib and cobimetinib, to improve care and outcomes for people with cancer around the world. The company’s top priority remains the ongoing commercialization of CABOMETYX (cabozantinib) tablets as a treatment for patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy. During the third quarter of 2017, CABOMETYX generated $90.4 million in net product revenue, while COMETRIQ (cabozantinib) capsules for the treatment of patients with progressive, metastatic medullary thyroid cancer generated an additional $6.1 million in net product revenue, for a combined $96.4 million in net product revenue for the cabozantinib franchise.

“In addition to strong financial performance, the third quarter of 2017 was marked by significant clinical and regulatory milestones that continue to drive us forward in our mission to help cancer patients recover stronger and live longer,” said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. “In August, we completed the filing for CABOMETYX in previously untreated advanced RCC, which has been accepted by the FDA and granted Priority Review. With an upcoming FDA action date of February 15, 2018, our commercial team is fully prepared for a potential launch of CABOMETYX in this expanded indication to bring this much needed option to even more patients with advanced RCC as quickly as possible. In addition, based on the positive results from the CELESTIAL pivotal trial, demonstrating that cabozantinib provided a statistically significant and clinically meaningful improvement in overall survival for patients with advanced hepatocellular carcinoma, we are moving rapidly to complete our U.S. regulatory filing in the first quarter of next year.”

Cabozantinib Highlights

Strong Growth in Cabozantinib Franchise Net Revenue. Cabozantinib generated $96.4 million in net product revenue during the third quarter of 2017, an increase of 10 percent from the second quarter of 2017 and an increase of 126 percent year-over-year.

Phase 3 CELESTIAL Trial Meets Primary Endpoint of Overall Survival (OS), with supplemental New Drug Application (sNDA) Filing Planned for Q1 2018. In October, Exelixis announced that the CELESTIAL trial met its primary endpoint of OS, with cabozantinib providing a statistically significant and clinically meaningful improvement in OS compared to placebo in patients with advanced hepatocellular carcinoma (HCC). The independent data monitoring committee for the study recommended that the trial should be stopped for efficacy following review of the second planned interim analysis. CELESTIAL is a randomized, global phase 3 trial of cabozantinib compared to placebo in patients with advanced HCC who have been previously treated with sorafenib. The safety data in the study were consistent with the established profile of cabozantinib. Based on these results, Exelixis plans to submit an sNDA to the U.S. Food and Drug Administration (FDA) in the first quarter of 2018. Detailed results from CELESTIAL will be submitted for presentation at a future medical conference.

Submission and Acceptance of sNDA for CABOMETYX for the Treatment of Previously Untreated Advanced RCC with FDA Priority Review. In August, Exelixis announced it had completed the submission of its sNDA to the FDA for CABOMETYX for the treatment of previously untreated advanced RCC. The sNDA submission is based on results from the CABOSUN randomized phase 2 trial of CABOMETYX compared to sunitinib in patients with previously untreated advanced RCC with intermediate- or poor-risk disease. After the quarter ended, the company announced the FDA had accepted the sNDA and granted Priority Review, assigning a Prescription Drug User Fee Act (PDUFA) action date of February 15, 2018.

Start of Phase 3 Trial of Cabozantinib in Combination with Nivolumab or with Nivolumab and Ipilimumab in Previously Untreated Advanced or Metastatic RCC. In July, Exelixis and Bristol-Myers Squibb (BMS) announced the initiation of CheckMate 9ER, the pivotal phase 3 trial evaluating cabozantinib in combination with two of BMS’ leading immunotherapies, nivolumab and ipilimumab, compared to sunitinib. The trial is planned to enroll 1,014 treatment-naïve patients, with a primary endpoint of progression-free survival.

Cabozantinib and Cobimetinib Data Presentations at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress. In September, Exelixis-discovered compounds were the subject of 10 presentations at the ESMO (Free ESMO Whitepaper) 2017 Congress held in Madrid, Spain. Data from CABOSUN, the randomized phase 2 trial of cabozantinib compared to sunitinib in patients with previously untreated advanced RCC with intermediate- or poor-risk disease, were the subject of a poster discussion which showed cabozantinib demonstrated a clinically meaningful and statistically significant reduction in the rate of disease progression or death. Other cabozantinib presentations included an oral presentation of data from the phase 1b trial of cabozantinib, nivolumab, and ipilimumab in advanced genitourinary malignancies, as well as additional analyses of the phase 3 METEOR trial in advanced RCC. Cobimetinib presentations at the Congress included two data sets concerning forms of metastatic melanoma. The company, along with its collaboration partner Ipsen, also hosted an investor and media event in Madrid to discuss the data for cabozantinib presented at the Congress and to take part in a question and answer session with Drs. Toni Choueiri, Sumanta Pal and Thomas Powles.

Cobimetinib Highlights

Settlement of Arbitration between Exelixis and Genentech Regarding Companies’ Collaboration Agreement for Cobimetinib. In July, Exelixis announced a settlement of its arbitration with Genentech concerning claims asserted by Exelixis against Genentech related to the development and commercialization of cobimetinib, the Exelixis-discovered medicine that is marketed as COTELLIC. The revised revenue and cost-sharing arrangement resolves the companies’ dispute pursuant to the arbitration demand filed on June 3, 2016, and aligns both companies’ interests in advancing cobimetinib as a promising therapy for patients with multiple forms of cancer. Moving forward, the revenue applied to the profit and loss statement for the COTELLIC collaboration (Collaboration P&L) will now be calculated using the average of the quarterly net selling prices of COTELLIC and any additional branded Genentech product(s) prescribed with COTELLIC. Exelixis will continue to share U.S. commercialization costs, while Genentech’s portion of these costs will now be allocated to the Collaboration P&L in proportion to the number of Genentech products in any given combination including COTELLIC.

Corporate Highlights

Updates from Partnered Programs with Daiichi Sankyo and BMS. In the third quarter and shortly after the quarter ended, Exelixis announced milestones for compounds from two of its partnered programs.

In September, collaborator Daiichi Sankyo announced positive top-line results from ESAX-HTN, a phase 3 pivotal trial of esaxerenone (formerly CS-3150) in patients with essential hypertension in Japan. As a result, Daiichi Sankyo plans to submit a Japanese regulatory application for esaxerenone for an essential hypertension indication in the first quarter of 2018. Daiichi Sankyo also announced the initiation of a pivotal trial of esaxerenone in patients with diabetic nephropathy. ESAX-DN is a phase 3 study in patients with type-2 diabetes with microalbuminuria who are taking an angiotensin II receptor blocker (ARB) or an angiotensin converting enzyme (ACE) inhibitor in Japan.

In October, Exelixis earned a $10 million milestone from BMS as part of the two companies’ worldwide collaboration for compounds targeting retinoic acid-related orphan receptor (ROR), a family of nuclear hormone receptors implicated in inflammatory conditions. The milestone was triggered by BMS’ filing of a Clinical Trial Authorization in Europe for a first-in-human study of a RORγt inverse agonist.

Debut of New Mission-Driven Corporate Branding and Website. In September, Exelixis introduced new corporate branding aligned with its mission, growth strategy and commitment to bring best-in-class oncology medicines to market. The new branding included a redesigned logo crafted as a wordmark with an extractable symbol that will become emblematic of Exelixis, as well as the revised corporate tagline, Resilience.Results.Remission. The new corporate branding celebrates the company’s unwavering perseverance to deliver results, and its aspirational commitments to the diverse audiences it serves.

Third Quarter 2017 Financial Results

Total revenue for the quarter ended September 30, 2017 was $152.5 million, compared to $62.2 million for the comparable period in 2016. Total revenue includes $96.4 million and $56.1 million of net product revenue and collaboration revenue, respectively, compared to $42.7 million and $19.5 million for the comparable period in 2016. The increase in net product revenues primarily reflects the growth in product sales of CABOMETYX since the product’s launch in late April 2016. Collaboration revenues for the quarter ended September 30, 2017 include two milestones totaling $45.0 million resulting from Ipsen’s receipt of the validation from the European Medicines Agency for the application for variation to the CABOMETYX marketing authorization for the addition of a new indication in first-line treatment of advanced RCC in adults; we also recognized $11.1 million in additional revenue from the company’s collaboration agreements with Ipsen, Takeda and Genentech during the quarter. Collaboration revenues for the comparable period in 2016 include the recognition of a $15.0 million milestone from Daiichi Sankyo and $4.5 million in revenue from the company’s collaboration agreements with Ipsen, Takeda and Genentech.

Research and development expenses for the quarter ended September 30, 2017 were $28.5 million, compared to $20.3 million for the comparable period in 2016. The increase in research and development expenses was primarily a result of increases in personnel expenses, clinical trial costs and consulting and outside services. The increase in personnel-related expenses was primarily a result of an increase in headcount associated with the re-launch of the company’s internal discovery program and the build-out of the company’s medical affairs organization. The increase in clinical trial costs was predominantly due to start-up costs associated with CheckMate 9ER and start-up costs associated with the phase 1b trial of cabozantinib and atezolizumab in locally advanced or metastatic solid tumors; those increases were partially offset by decreases in costs related to METEOR, the company’s completed phase 3 pivotal trial comparing CABOMETYX to everolimus in patients with advanced RCC. The increase in consulting and outside services was primarily in support of the company’s medical affairs organization.

Selling, general and administrative expenses for the quarter ended September 30, 2017 were $38.1 million, compared to $32.5 million for the comparable period in 2016. The increase in selling, general and administrative expenses was primarily a result of increases in consulting and outside services to support the company’s marketing activities and in personnel expenses resulting primarily from an increase in general and administrative headcount to support the company’s commercial and research and development organizations. Those increases were partially offset by a decrease in losses under the collaboration agreement with Genentech driven by Genentech’s change in cost allocation approach in December 2016.

Other income (expense), net for the quarter ended September 30, 2017 was $3.4 million compared to $(18.5) million for the comparable period in 2016. The increase in other income (expense), net, was primarily due to a $13.8 million loss on extinguishment of debt associated with the conversions of the 4.25% Convertible Subordinated Notes due 2019 (2019 Notes) during the third quarter of 2016, and a $7.8 million decrease in interest expense due to the conversions and the redemption of the 2019 Notes during the third and fourth quarters of 2016, the repayment of the Silicon Valley Bank term loan in March 2017 and the repayment of the Deerfield Notes in June 2017.

Net income for the quarter ended September 30, 2017 was $81.4 million, or $0.28 per share, basic and $0.26 per share, diluted, compared to a net loss of $(11.3) million, or $(0.04) per share, basic and diluted, for the comparable period in 2016. The transition to profitability was primarily due to the increase in net product revenues, reflecting the growth in product sales of CABOMETYX since the launch in late April 2016, which was supplemented by the growth in our collaboration revenues and partially offset by the increase in operating expenses.

Cash and cash equivalents, short- and long-term investments and long-term restricted cash and investments totaled $422.3 million at September 30, 2017, as compared to $479.6 million at December 31, 2016.

2017 Financial Guidance

The company is updating its guidance that total costs and operating expenses for the full year will be between $285 million and $295 million. This guidance includes approximately $25 million of non-cash costs and expenses related primarily to stock-based compensation expense.

Basis of Presentation

Exelixis adopted a 52- or 53-week fiscal year that generally ends on the Friday closest to December 31st. For convenience, references in this press release as of and for the fiscal periods ended September 29, 2017, December 30, 2016 and September 30, 2016 are indicated as being as of and for the periods ended September 30, 2017, December 31, 2016 and September 30, 2016, respectively.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the third quarter of 2017 and provide a general business update during a conference call beginning at 5:00 p.m. EDT / 2:00 p.m. PDT today, Wednesday, November 1, 2017.

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 855-793-2457 (domestic) or 631-485-4921 (international) and provide the conference call passcode 96645455 to join by phone.

A telephone replay will be available until 8:00 p.m. EDT on November 3, 2017. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 96645455. A webcast replay will also be archived on www.exelixis.com for one year.

On November 1, 2017 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months ended September 30, 2017 and provided a corporate update (Press release, DURECT, NOV 1, 2017, View Source [SID1234521425]).

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Executed a patent purchase agreement with Indivior relevant to Indivior’s RBP-7000, yielding DURECT a $12.5 million upfront payment, as well as a potential $5 million milestone payment and potential earn-out payments. Indivior submitted a NDA for RBP-7000 to the U.S. FDA on September 28, 2017.

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Including the $12.5 million described above, total revenues were $20.7 million and net income was $6.1 million for the three months ended September 30, 2017 as compared to total revenues of $3.7 million and net loss of $8.8 million for the three months ended September 30, 2016.

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At September 30, 2017, cash and investments were $41.8 million, compared to cash and investments of $25.2 million at December 31, 2016.


“The top-line results from the Phase 3 clinical trial of POSIMIR did not meet expectations, trending in favor of POSIMIR but not reaching statistically significance. We will be working to understand the trial results more fully in the coming weeks,” stated James E. Brown, D.V.M., President and CEO of DURECT. “On other fronts, positive highlights of the third quarter included reaching an agreement with Indivior which resulted in a meaningful upfront payment to DURECT as well the potential for further payments based on the progress of RBP-7000, a drug candidate for schizophrenia for which Indivior has submitted an NDA to the FDA. We are also making significant progress towards initiating multiple Phase 2 studies with DUR-928.”

Update on Selected Programs and Transactions:

POSIMIR (SABER-Bupivacaine) Post-Operative Pain Relief Depot. POSIMIR is our investigational post-operative pain relief depot that utilizes our patented SABER technology and is designed to deliver bupivacaine to provide up to 3 days of pain relief after surgery.

In October 2017, we reported that PERSIST, a Phase 3 clinical trial for POSIMIR did not meet its primary efficacy endpoint of reduction in pain on movement over the first 48 hours after surgery as compared to standard bupivacaine HCl. While results trended in favor of POSIMIR versus the comparator, they did not achieve statistical significance. We and Sandoz, our licensee for commercialization rights for POSIMIR in the United States, will be working to understand the trial results more fully in the coming weeks.

Epigenetic Regulator Program. DUR-928, the lead product candidate in our Epigenetic Regulator Program, is an endogenous, small molecule, new chemical entity (NCE), which may have broad applicability in several metabolic diseases such as nonalcoholic steatohepatitis (NASH) and other disorders of the liver, in acute organ injuries such as acute liver and/or kidney injury, and in autoimmune/inflammatory skin disorders such as psoriasis and atopic dermatitis.

Oral Administration
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We are actively working towards initiating a Phase 2 trial in primary sclerosing cholangitis (PSC) with orally administered DUR-928. Our protocol has been reviewed by the FDA and our IND is now open. Clinical trial site preparation is underway, and we are targeting dosing our first patient by the end of 2017. PSC is a chronic liver disease characterized by a progression of cholestasis (decrease in bile flow) with inflammation and fibrosis of bile ducts. We have received orphan drug designation for DUR-928 to treat patients with PSC. We believe that data generated from this trial will be relevant to other chronic liver conditions, such as NASH.

Injectable Administration

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We now have an open IND for an initial Phase 2 trial with an injectable formulation of DUR-928. We are currently finalizing the protocol based on detailed input received in October 2017 from our expert advisors during The Liver Meeting (the annual meeting of the American Association for the Study of Liver Diseases or AASLD). This first study will be conducted in patients with moderate and severe acute liver function impairment to assess the safety and pharmacokinetics of several doses of DUR-928.
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At AASLD, a poster was presented by Dr. Shunlin Ren of Virginia Commonwealth University / McGuire VA Medical Center which included newly disclosed data from animal studies with DUR-928 in various acute organ injuries. One of the new disclosures was the dose-dependent effect of DUR-928 in stabilizing mitochondrial membranes, which is an important factor in
cell viability and prevention of cell death. Previous results had been reported with DUR-928 in an acute animal model where the toxicity was caused by lipopolysaccharide (LPS); in this poster, similar results (i.e., 90% survival with DUR-928 vs. 10% survival on placebo) were shown when the toxicity was caused by injected acetaminophen. This poster also demonstrated the pharmacological effect of DUR-928 in animal models of both endotoxin and drug induced multiple organ injuries, including the liver, the kidney and the lungs. This poster is available at www.durect.com under Science and Technologies, Papers.

Topical Administration

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Based on promising results from a previous exploratory Phase 1b trial in psoriasis utilizing intralesional micro injections of DUR-928, we have developed topical formulations of DUR-928 and have recently completed GLP skin irritation / sensitization studies with the lead formulations in two species. We have had pre-IND interactions with the FDA and are incorporating FDA’s comments in our upcoming IND while we are actively working with expert advisors to finalize our study protocol for a Phase 2 proof-of-concept study with topically applied DUR-928. We expect to initiate this study in the first half of 2018. We believe that there is a large market opportunity for new topical drugs for inflammatory skin diseases such as psoriasis and atopic dermatitis.

Indivior Agreement. In September 2017, we entered into a patent purchase agreement with an affiliate of Indivior PLC, whereby DURECT assigned certain of its U.S. patent rights to Indivior. This assignment may provide further intellectual property protection for RBP-7000, Indivior’s investigational once-monthly injectable risperidone product for the treatment of schizophrenia. Indivior submitted a New Drug Application (NDA) for RBP-7000 to the U.S. Food and Drug Administration (FDA) on September 28, 2017.

Under the terms of the agreement, Indivior has made an upfront non-refundable payment to DURECT of $12.5 million, with the potential for an additional $5 million based on NDA approval of RBP-7000, as well as quarterly earn-out payments that are based on a single digit percentage of U.S. net sales for certain products covered by the patent rights, including RBP-7000. The patent rights include granted patents extending through at least 2026.

REMOXY ER (oxycodone) Extended-Release Capsules CII. Based on our ORADUR technology, the investigational drug REMOXY ER is a unique long-acting formulation of oxycodone designed to discourage common methods of tampering associated with opioid misuse and abuse. In March 2017, Pain Therapeutics announced that it plans to resubmit the REMOXY ER NDA after completing two additional studies regarding REMOXY ER based on guidance obtained in a meeting with the FDA. The two studies are a clinical abuse potential study via the intranasal route of abuse and a non-clinical abuse potential study using household solvents. Pain Therapeutics stated that it expects to complete these studies by year end 2017. In October 2017, Pain Therapeutics announced that there is a pre-NDA guidance meeting with the FDA planned for November 14, 2017 and Pain Therapeutics is planning an NDA resubmission in the first quarter of 2018.


ORADUR-ADHD Program. ORADUR-Methylphenidate ER is an investigational drug that has the potential for rapid onset of action and long duration with once-a-day dosing, utilizes a small capsule size relative to the leading existing long-acting products on the market and incorporates our ORADUR anti-tampering technology. Orient Pharma, our licensee in defined Asian and South Pacific countries, has reported that a Phase 3 study conducted in Taiwan has achieved positive results. We retain rights to all other markets in the world, notably including the U.S., Europe and Japan. We have started a process of contacting potential development and commercialization partners for major markets not licensed to Orient Pharma.

Upcoming investor conference. DURECT will be presenting at the Stifel 2017 Healthcare Conference at 11:45 am Eastern time on November 15. The conference is being held at the Lotte New York Palace Hotel. A live audio webcast of the presentation will be available by accessing View Source . A live audio webcast of these presentations will also be available by accessing DURECT’s homepage at www.durect.com and clicking “Investor Relations.” If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under Audio Archive in the “Investor Relations” section.

Earnings Conference Call
A live audio webcast of a conference call to discuss third quarter 2017 results and provide a corporate update will be broadcast live over the internet at 4:30 p.m. Eastern Time on November 1 and is available by accessing DURECT’s homepage at www.durect.com and clicking “Investor Relations.” If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under Audio Archive in the “Investor Relations” section.