On November 7, 2017 Novavax, Inc., (Nasdaq: NVAX) reported its financial results for the third quarter and nine months ended September 30, 2017 (Press release, Novavax, NOV 7, 2017, View Source [SID1234521686]).

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Third Quarter Achievements:

· Novavax initiated a Phase 1/2 clinical trial of its nanoparticle influenza vaccine candidate with its proprietary Matrix-M adjuvant (NanoFlu) in older adults. The trial is a randomized, observer-blinded, active comparator-controlled trial in approximately 330 healthy older adults. The primary objective of the trial is to assess the safety and immunogenicity of two antigen concentrations (15 µg or 60 µg) of NanoFlu compared to a licensed influenza vaccine, Fluzone High-Dose (Fluzone HD).

· Novavax reported that NanoFlu demonstrated superior immunogenicity and protection compared to the market leader in a preclinical challenge study. In a head-to-head comparison study against Fluzone HD, NanoFlu demonstrated significantly stronger and broader immune responses against matched and unmatched influenza strains, including a series of "drifted" strains evolved over more than a decade of influenza seasons. NanoFlu was also protective in an established challenge model against both a matched and a ten-year old unmatched strain. Data from this study was published in the journal Vaccine.

· The Prepare clinical trial for infants via maternal immunization, supported by an $89 million grant from the Bill and Melinda Gates Foundation (BMGF), accelerated into the third global season of enrollment. Prepare’s global footprint is expected to grow from 16 sites in five countries in its first season of enrollment to over 90 sites in 12 countries in 2018.

Anticipated 2017 Event:

· Announce topline data from a Phase 1/2 clinical trial of the NanoFlu vaccine candidate in a head-to-head comparison with Fluzone HD before the end of 2017.

Summary

"We were very pleased to initiate and fully enroll the Phase 1/2 clinical trial of our NanoFlu vaccine candidate in older adults, following impressive results in preclinical challenge models. Given this timing, we are well positioned to announce data from the ongoing trial before the end of the year," said Stanley C. Erck, President and CEO. "We also continue to make significant progress in our RSV F Vaccine program, highlighted by the continued execution of the Phase 3 Prepare trial for infants via maternal immunization. We are also evaluating plans to conduct a clinical trial for older adults in 2018."

Financial Results for the Three and Nine Months Ended September 30, 2017

Novavax reported a net loss of $44.6 million, or $0.15 per share, for the third quarter of 2017, compared to a net loss of $66.3 million, or $0.24 per share, for the third quarter of 2016. For the nine months ended September 30, 2017, the net loss was $132.9 million, or $0.47 per share, compared to a net loss of $222.9 million, or $0.82 per share, for the same period in 2016.

Novavax revenue in the third quarter of 2017 was $8.4 million, compared to $3.2 million in the same period in 2016. This 158% increase was driven by higher revenue recorded under the BMGF grant of $89 million.

Research and development expenses decreased 21% to $41.9 million in the third quarter of 2017, compared to $53.0 million for the same period in 2016. The decrease was primarily due to reduced activities related to the development of the RSV F Vaccine for older adults, other general R&D project-related expenses and lower employee-related costs.

General and administrative expenses decreased 40% to $8.1 million in the third quarter of 2017, compared to $13.6 million for the same period in 2016. The decrease was primarily due to lower professional fees for pre-commercialization activities and lower employee-related costs.

Interest income (expense), net for the third quarter of 2017 and 2016 was ($3.0) million.

As of September 30, 2017, the company had $172.6 million in cash, cash equivalents and marketable securities, compared to $235.5 million as of December 31, 2016. Net cash used in operating activities for the first nine months of 2017 was $106.6 million, compared to $194.2 million for the same period in 2016. The decrease in cash usage was primarily due to decreased costs relating to our RSV F Vaccine and lower overall employee-related costs.

Conference Call

Novavax management will host its quarterly conference call today at 4:30 p.m. ET. The dial-in number for the conference call is (877) 212-6076 (Domestic) or (707) 287-9331 (International), passcode 2479419. A replay of the conference call will be available starting at 7:30 p.m. ET on November 7, 2017 until 7:30 pm ET on November 14, 2017. To access the replay by telephone, dial (855) 859-2056 (Domestic) or (404) 537-3406 (International) and use passcode 2479419.

A webcast of the conference call can also be accessed via a link on the home page of the Novavax website (novavax.com) or through the "Investor Info"/"Events" tab on the Novavax website. A replay of the webcast will be available through the "Investor Info"/"Events" tab on the Novavax website until February 7, 2018.

On November 7, 2017 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, reported recent events and financial results for the three and nine months ended September 30, 2017 (Press release, Regulus, NOV 7, 2017, View Source [SID1234521688]).

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Regulus Therapeutics Inc. Logo

"The third quarter was marked by the successful completion of our financing, as well as significant operational progress on our two clinical programs," said Jay Hagan, President and Chief Executive Officer of Regulus. "We are very pleased that both of our Alport studies are now active, the investigational new drug (IND) for the treatment of autosomal dominant polycystic kidney disease (ADPKD) has been filed, and a Phase I study remains on track to initiate by year-end."

Third Quarter Corporate Highlights and Recent Events

In July 2017, Regulus completed a public offering of its common stock, and received net proceeds of approximately $43.0 million after deducting underwriting discounts, commissions and other offering expenses.
In September, Regulus initiated HERA, the Phase II randomized, double-blinded, placebo-controlled study evaluating the safety and efficacy of RG-012 in Alport syndrome patients. In parallel, the Phase I renal biopsy study was also initiated.
Recently, Regulus filed an IND application with the FDA for RGLS4326, targeting microRNA-17 for the treatment of ADPKD.
Third Quarter Financial Results

Cash Position: As of September 30, 2017, Regulus had cash, cash equivalents and short-term investments of $71.4 million.

Research and Development (R&D) Expenses: R&D expenses were $12.7 million and $42.7 million for the three and nine months ended September 30, 2017, respectively, compared to $14.6 million and $49.3 million for the same periods in 2016. The decrease in R&D expenses for the three months ended September 30, 2017 compared to 2016 was driven by the planned reduction in personnel-related costs as a result of our May 2017 corporate restructuring. The decrease in R&D expenses for the nine months ended September 30, 2017 compared to 2016 was primarily driven by the wind-down of clinical activities related to the RG-101 program.

General and Administrative (G&A) Expenses: G&A expenses were $2.7 million and $13.8 million for the three and nine months ended September 30, 2017, respectively, compared to $4.8 million and $13.6 million for the same periods in 2016. The decrease in G&A expenses for the three months ended September 30, 2017 compared to 2016 was attributable to the planned reduction in personnel-related costs and non-cash stock-based compensation.

Revenue: Revenue was less than $0.1 million for each of the three and nine months ended September 30, 2017, compared to $0.2 million and $1.2 million for the same periods in 2016.

Net Loss: Net loss was $15.8 million and $57.5 million for the three and nine months ended September 30, 2017, respectively, compared to a net loss of $19.5 million and $61.8 million for the same periods in 2016. Basic and diluted net loss per share was $0.18 and $0.88 for the three and nine months ended September 30, 2017, respectively, compared to $0.37 and $1.17 for the same periods in 2016.

Conference Call Details

Regulus will host a conference call and webcast today at 5:00 p.m. Eastern Time to discuss third quarter 2017 financial results and corporate highlights. A live webcast of the call will be available online at www.regulusrx.com. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 9297128. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), conference ID 9297128. The webcast and telephone replay will be archived on the Company’s website following the call.

On November 7, 2017 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported financial results for the third quarter and nine months ended September 30, 2017 (Press release, Rigel, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2315079 [SID1234521689]).

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Recent Achievements

On October 2, 2017, Rigel announced that the U.S. Food and Drug Administration (FDA) is not currently planning on holding an Oncology Drugs Advisory Committee (ODAC) meeting to discuss the New Drug Application (NDA) for fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, in adult patients with chronic or persistent immune thrombocytopenia (ITP).
Rigel recently completed enrollment of Stage 1 of its Phase 2 study of fostamatinib for the treatment of warm antibody autoimmune hemolytic anemia (AIHA). On a top-line, preliminary basis, the Phase 2 study achieved the pre-specified primary efficacy endpoint for Stage 1. Given this achievement, Rigel is preparing to begin enrollment of Stage 2 of this study.
Rigel announced the closing of a public offering of common stock in October, with proceeds of $69,730,250, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Rigel.
"Our NDA review is progressing smoothly," said Raul Rodriguez, president and chief executive officer of Rigel. "As we prepare for an FDA decision, we are simultaneously implementing our commercial plan for a potential launch of fostamatinib in ITP, as well as continuing our clinical studies of fostamatinib in other disorders."

For the third quarter of 2017, Rigel reported a net loss of $17.7 million, or $0.14 per basic and diluted share, compared to a net loss of $22.6 million, or $0.24 per basic and diluted share, in the same period of 2016.

Contract revenues from collaborations of $900,000 in the third quarter of 2017 were related to a payment received from a license agreement with a third party. Contract revenues from collaborations of $3.8 million in the third quarter of 2016 represent the remaining amortization of the $30.0 million upfront payment which was fully amortized in September 2016, pursuant to Rigel’s collaboration and license agreement with Bristol-Myers Squibb.

Rigel reported total costs and expenses of $18.8 million in the third quarter of 2017, compared to $26.5 million for the same period in 2016. The decrease in costs and expenses was primarily due to the decreases in personnel costs as a result of the reduction in workforce in September 2016 and the completion of the pivotal Phase 3 clinical trials in ITP in 2016, partially offset by the increase in costs related to the preparation for the potential commercial launch of fostamatinib in ITP.

For the nine months ended September 30, 2017, Rigel reported a net loss of $52.1 million, or $0.43 per basic and diluted share, compared to a net loss of $53.6 million, or $0.58 per basic and diluted share, for the same period of 2016.

As of September 30, 2017, Rigel had cash, cash equivalents and short-term investments of $68.1 million, compared to $74.8 million as of December 31, 2016. In October 2017, Rigel completed an underwritten public offering in which it received proceeds of approximately $65.3 million, net of underwriting discounts and commissions and estimated offering expenses. Rigel expects that its cash, cash equivalents and short-term investments will be sufficient to support its current and projected funding requirements, including the potential U.S. commercial launch, through at least the next 12 months. Rigel continues to evaluate ex-U.S. partnerships for fostamatinib and other partnering opportunities across its pipeline.

Corporate Update
Rigel continues to execute on its commercial readiness plan to support the potential commercial launch of fostamatinib in 2018. This includes the hiring of key personnel across several functions such as marketing, sales, market access, business operations and medical affairs.

Portfolio Update
TAVALISSE (fostamatinib disodium) in ITP
During the Mid-Cycle Communication teleconference, the FDA confirmed that it was not currently planning to hold an ODAC meeting to discuss the NDA for fostamatinib in patients with chronic or persistent ITP. The FDA indicated that the review of fostamatinib is proceeding according to the standard internal review timeline as described in the Guidance on Good Review Management Principles and Practices for Prescription Drug User Fee Act (PDUFA) Products. Under the PDUFA, the action date for the FDA to complete its review will be April 17, 2018.

Update on Fostamatinib in Autoimmune Hemolytic Anemia (AIHA)
The Phase 2, open-label, multi-center, Simon two-stage study of fostamatinib for the treatment of warm AIHA recently completed enrollment of Stage 1. The study is evaluating the safety and efficacy of fostamatinib, at 150mg BID (twice daily), in patients with warm AIHA who have previously received at least one treatment for this disease, but did not have a meaningful benefit and are still anemic.

Stage 1 of the 12-week study enrolled 17 patients. As of September 30, 2017, four patients responded during the 12-week evaluation period and an additional two patients met the response criteria in the extension study after 12 weeks of dosing, for a response rate of 35% (6/17) on fostamatinib (these data are preliminary and require further verification). A response was defined as achieving a hemoglobin level of greater than 10 g/dl and at least a 2 g/dl increase from baseline. The safety profile was consistent with the existing fostamatinib safety database.

Fostamatinib in IgA Nephropathy (IgAN)
Rigel has completed enrollment of the second cohort in its Phase 2 study of fostamatinib in IgAN. Similar to the first cohort, which reported results in January 2017, the study will evaluate the efficacy, safety, and tolerability of fostamatinib as measured by change in proteinuria, renal function, and histology (comparing the pre- and post-study renal biopsies). However, the second cohort evaluates a higher dose of fostamatinib, 150mg BID, while the first cohort evaluated 100mg BID. The primary efficacy endpoint is the mean change of proteinuria from baseline at 24 weeks.

Additional Product Development
During the second quarter of 2017, Rigel selected a molecule from its IRAK program for preclinical development. The molecule is differentiated in that it inhibits both the IRAK 1 and IRAK 4 signaling pathways, with potential to treat autoimmune and inflammatory diseases such as lupus, gout, psoriatic arthritis and multiple sclerosis. The company expects to initiate clinical trials in 2018.

About ITP
ITP affects approximately 65,000 adult primary patients in the US. In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising, bleeding and fatigue. People suffering with chronic ITP may live with increased risk of severe bleeding events that can result in serious medical complication, or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPOs) and splenectomy. However, not all patients are adequately treated with existing therapies and as a result, there remains a significant medical need for additional treatment options for patients with chronic ITP.

About AIHA
AIHA is a rare, serious blood disorder where the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 40,000 adult patients in the US and can be a severe, debilitating anemia. To date, there are no FDA approved disease-targeted therapies for AIHA, despite the tremendous medical need that exists for these patients as disease relapse is common. Instead, physicians generally treat acute and chronic cases of the disorder with corticosteroids, IV immunoglobulin infusion, other immuno-suppressants, or splenectomy (surgical removal of the spleen).

About IgAN
IgAN (also known as Berger’s disease) is a chronic autoimmune disease associated with inflammation in the kidneys that diminishes their ability to filter blood. It is the most common primary glomerular disease affecting an estimated 82,500 to 165,000 cases in the US, with a higher prevalence in Asia. For as many as 25 percent of those living with IgAN, the disease results in end-stage renal failure requiring dialysis or kidney transplantation. Other than angiotensin blockade (primarily for blood-pressure control), there are no disease-targeted therapies for IgAN.

Conference Call and Webcast Today at 5:00PM Eastern Time
Rigel will hold a live conference call and webcast today at 5:00pm Eastern Time (2:00pm Pacific Time).

Participants can access the live conference call by dialing 855-892-1489 (domestic) or 720-634-2939 (international) and using the Conference ID number 4894258. The conference call will also be webcast live and can be accessed from Rigel’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

On November 7, 2017 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing entinostat and SNDX-6352 in multiple cancer indications, reported its financial results for the third quarter ended September 30, 2017 (Press release, Syndax, NOV 7, 2017, View Source [SID1234521690]). In addition, the Company provided a clinical and business update. As of September 30, 2017, Syndax had $120.6 million in cash, cash equivalents and short-term investments.

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The Company continues to expand its pipeline, and recently announced that it has entered into an exclusive worldwide license agreement with Vitae Pharmaceuticals, Inc., a subsidiary of Allergan plc, for a portfolio of preclinical, orally-available small molecule inhibitors of the interaction of Menin with the Mixed Lineage Leukemia ("MLL") protein. Syndax plans to initially study these compounds for the treatment of a genetically-defined subset of acute leukemias with chromosomal rearrangements in the MLL gene ("MLL-r").

All four cohorts of ENCORE 601, an open-label, Simon two-stage design, Phase 1b/2 clinical trial evaluating the combination of entinostat, the Company’s class I selective HDAC inhibitor, plus Merck’s anti-PD-1 (programmed death receptor-1) blocking therapy, KEYTRUDA, continue to proceed on schedule. Enrollment in the first stage of the cohort of patients with microsatellite stable colorectal cancer (MSS-CRC) is complete, and a decision on whether to continue to the second stage is expected in the first quarter of 2018. Enrollment in the second stage of the PD-(L)1 refractory non-small cell lung cancer (NSCLC) cohort is complete, and Syndax expects to share updated data from this cohort in the first half of 2018.

"As we near the end of 2017, the momentum we’ve built throughout the year continues to yield meaningful progress and growth for our pipeline of potential best-in-class candidates," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "The recent expansion of our pipeline represents what we believe will be a long-term value enhancing transaction for the Company, while also potentially changing the treatment paradigm for acute leukemic patients harboring MLL translocations, where there exists a high unmet need. For entinostat, the ENCORE 601 program remains on track and we look forward to presenting data from both NSCLC cohorts, as well as biomarker data from the melanoma cohort, at the upcoming SITC (Free SITC Whitepaper) Annual Meeting. We are also developing a global registration plan for entinostat in combination with a PD-1 inhibitor for patients with PD-1 refractory melanoma. We anticipate sharing details of our plan in the first half of 2018, in parallel with the full Phase 2 data from the melanoma cohort of ENCORE 601.

Pipeline Updates

The Phase 3 registration trial of entinostat plus exemestane in advanced HR+, HER2- breast cancer, E2112, is 83% enrolled as of the end of October. ECOG-ACRIN Cancer Research Group, the trial sponsor, has notified the Company that the Data Safety Monitoring Committee (DSMC) completed the final progression free survival analysis and the first interim analysis for overall survival. The results of this analysis are held confidentially by the ECOG-ACRIN study statistician and the DSMC. No communication regarding this analysis will be released until completion of enrollment, which ECOG-ACRIN expects will occur in the first half of 2018.
The ENCORE 601 cohort enrolling NSCLC patients naïve to PD-(L)1 therapy has satisfied the pre-specified efficacy criteria for advancement to the second stage, with ≥ 4 responses out of 17.
Enrollment in the second stage of the ENCORE 601 cohort enrolling patients with PD-(L)1 refractory NSCLC is complete. Data from this cohort is expected to be available in the first half of 2018.
Following a meeting with the U.S. Food and Drug Administration (FDA) in June, the Company is continuing to meet with individual regulatory agencies in Europe to align on a global registration plan for entinostat in combination with a PD-1 inhibitor for patients with PD-(L)1 refractory melanoma. The Company anticipates being in a position to outline a regulatory plan for this indication around the time that full Phase 2 data from the melanoma cohort of ENCORE 601 are available in the first half of 2018.
An oral presentation highlighting the data from stage one of both the ENCORE 601 NSCLC cohorts, as well as a poster presentation covering the biomarker data from the ENCORE 601 melanoma cohort, will be presented at the upcoming Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. The Company anticipates sharing full Phase 2 trial data from both the PD-(L)1 refractory NSCLC and melanoma cohorts at a medical congress in the first half of 2018. Details on both SITC (Free SITC Whitepaper) presentations are available here.
Enrollment in the first stage of the cohort enrolling patients with microsatellite stable (MSS)–CRC is complete, and a decision on whether to advance to the second stage is expected in the first half of 2018. At least 2 confirmed objective responses are required to proceed to the second stage.
Enrollment continues in the ENCORE 602 and ENCORE 603 trials, both of which are aimed at exploring the ability of entinostat to enhance the efficacy of checkpoint (PD-L1) inhibitor therapies. ENCORE 602, the Phase 1b/2 clinical trial evaluating the combination of entinostat plus Genentech’s PD-L1 inhibitor, TECENTRIQ, in patients with triple negative breast cancer, is now expected to complete enrollment in the Phase 2 portion in the first half of 2018, with results anticipated in the second half of the year. ENCORE 603, the Phase 1b/2 clinical trial evaluating entinostat in combination with Pfizer/Merck KGaA’s BAVENCIO in patients with ovarian cancer, continues to enroll patients into the Phase 2 portion and is on track to complete enrollment in the first half of 2018, with results anticipated in the first half of 2019.
Dosing of patients with solid tumors in the Phase 1 multiple ascending dose (MAD) clinical trial of SNDX-6352, the Company’s anti-CSF-1R monoclonal antibody, has commenced. A poster highlighting the safety, pharmacokinetic and pharmacodynamic data from the single ascending dose (SAD) trial of SNDX-6352 in healthy volunteers will be presented at the upcoming SITC (Free SITC Whitepaper) Annual Meeting. Details on the presentation are available here.
The Company entered into an exclusive worldwide license agreement with Vitae Pharmaceuticals, Inc., a subsidiary of Allergan plc, for a portfolio of preclinical, orally-available small molecule inhibitors of the interaction of Menin with the MLL protein. These compounds have potential application in the treatment of a genetically-defined subset of acute leukemias with chromosomal rearrangements in the MLL gene ("MLL-r"). The Company expects to initiate clinical trials in 2019.

Third Quarter 2017 Financial Results

As of September 30, 2017, Syndax had cash, cash equivalents and short-term investments of $120.6 million and 22.3 million shares issued and outstanding. In October 2017, the Company reported the sale of 2.0 million common shares in a registered direct offering with net proceeds of $24.8 million.

Third quarter 2017 research and development expenses decreased to $12.2 million from $12.3 million for the comparable period in the prior year. The decrease was primarily due to increased clinical trial activities of $3.9 million and increased employee compensation expense of $0.9 million, offset by a $5.0 million upfront payment in 2016 related to the in-license of SNDX-6352 from UCB. The increase in clinical trial activities was primarily due to additional cohorts added to ENCORE 601, increased activities in ENCORE 602 and ENCORE 603, costs related to SNDX-6352, Phase 1 clinical pharmacology trials and CMC activities. The increase in employee compensation costs was primarily due to increased headcount.

General and administrative expenses totaled $3.6 million during the third quarter of 2017 compared with $3.3 million in the comparable period in the prior year. The increase in general and administrative expenses was primarily due to an increase in non-cash stock-based compensation of $0.4 million and an increase in salary expense of $0.2 million due to increased headcount, offset by decreased legal fees of $0.3 million.

For the three months ended September 30, 2017, Syndax reported a net loss attributable to common stockholders of $15.1 million, or $0.68 per share, compared to $15.0 million, or $0.84 per share, for the comparable prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the fourth quarter and full year 2017. For the fourth quarter and full year 2017, research and development expenses are expected to be $15 to $18 million and $47 to $50 million, respectively, and total operating expenses are expected to be $19 to $22 million and $63 to $66 million, respectively. Research and development expenses for the fourth quarter includes $5.0 million paid to Allergan in connection with the Menin-MLL license. This expense will be offset on a cash basis with a $5.0 million development milestone earned in the fourth quarter under our agreement with KHK.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Tuesday, November 7, 2017.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 4569859
Domestic Dial-in Number: 1-855-251-6663
International Dial-in Number: 281-542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On November 7, 2017 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported operating results for third-quarter 2017 and provided an update on the Company’s commercial products and development programs (Press release, TESARO, NOV 7, 2017, View Source [SID1234521691]).

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"Exiting the third quarter, ZEJULA achieved 60% market share of the ovarian cancer patient population treated with a PARP inhibitor. This is a result of our team’s solid execution, and is supported by the feedback from physicians and patients, which continues to be excellent with regards to the benefit ZEJULA provides for women living with ovarian cancer," said Lonnie Moulder, CEO of TESARO. "Looking ahead, we are actively preparing for two additional product launches in 2017 — ZEJULA in Europe and VARUBI IV in the U.S. — and expanding our niraparib development programs to broaden its use with the PRIMA Phase 3 first line ovarian cancer study and the initiation of multiple combination studies in ovarian, lung, and breast cancer. We are rapidly advancing our pipeline of immuno-oncology candidates with three antibodies now in the clinic, and we are excited about the potential for the combination of TSR-022 and TSR-042 to meaningfully benefit patients with advanced solid tumors."

Recent Business Highlights

On October 25, 2017, the U.S. Food and Drug Administration (FDA) approved the intravenous (IV) formulation of VARUBI (rolapitant), and the U.S. commercial launch is planned for November. The unit demand for VARUBI oral capsules increased 74% for Q3 2017 vs. Q3 2016, as the brand continues to penetrate the U.S. oral NK-1 market.
ZEJULA (niraparib) is the most prescribed PARP inhibitor in the U.S., with approximately 2,500 patients treated during the month of September.
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for ZEJULA as a monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete response (CR) or partial response (PR) to platinum-based chemotherapy.
Pre-launch preparations continue in support of a European launch of ZEJULA by year-end 2017 beginning with Germany, pending European Commission approval.
The niraparib expanded access program (EAP) in Europe has enrolled more than 350 patients to date.
Clinical trials were initiated to support planned Phase 3 studies of niraparib combined with our anti-PD-1 antibody, TSR-042, in patients with lung and ovarian cancers and to evaluate niraparib plus TSR-042 in patients with advanced or metastatic cancers suitable for treatment with an anti-PD-1 antibody, including ovarian and lung.
Enrollment continues in the PRIMA trial of niraparib for patients with first-line ovarian cancer and the QUADRA trial of niraparib for the treatment of patients with ovarian cancer who have received three or more prior lines of chemotherapy.
The Phase 2 TOPACIO trial of niraparib plus pembrolizumab is ongoing with additional data expected in 1H 2018. Preliminary Phase 2 data presented at ESMO (Free ESMO Whitepaper) showed activity in patients with platinum-resistant ovarian cancer and patients with triple-negative breast cancer.
The Janssen GALAHAD Phase 2 efficacy and safety study of niraparib in men with metastatic castration-resistant prostate cancer and DNA-repair anomalies is ongoing to support a planned regulatory submission in 2019.
Phase 1 data for TSR-042 (anti-PD-1 antibody) presented at ESMO (Free ESMO Whitepaper) demonstrated a predictable safety profile and clinical activity in heavily pre-treated patients. The GARNET registration trial of TSR-042 continues to enroll patients with metastatic microsatellite instability-high (MSI-H) cancers.
To support initiation of planned Phase 3 studies, a clinical study was initiated to evaluate TSR-042 in combination with carboplatin-paclitaxel in patients with advanced or metastatic cancer.
Enrollment continues in the Phase 1 AMBER combination study of TSR-022 (anti-TIM-3 antibody) plus TSR-042, and the Phase 1 CITRINO dose-escalation trial of TSR-033 (anti-LAG-3 antibody). Data from the monotherapy, dose-escalation portion of the AMBER trial of TSR-022 will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting on November 10.
Third Quarter 2017 Financial Results

TESARO reported total revenue of $142.8 million for the third quarter of 2017, compared to $17.0 million for the third quarter of 2016.

Net product revenue totaled $41.8 million for the third quarter of 2017, which included ZEJULA revenues of $39.4 million and VARUBI/VARUBY revenues of $2.4 million. This compares to net product revenue of $1.3 million for the third quarter of 2016.

License, collaboration, and other revenue totaled $101.0 million for the third quarter of 2017 and included the $100.0 million up-front payment received as part of the license agreement with Takeda. This compares to license, collaboration, and other revenue of $15.7 million for the third quarter of 2016, which included the majority of the $15.0 million up-front payment received as part of the Zai Lab license agreement.

Cost of sales totaled $7.5 million for the third quarter of 2017 and included $6.2 million associated with product revenue and $1.3 million related to amortization of milestones recorded upon FDA approval of ZEJULA and first commercial sales of VARUBI/VARUBY in the U.S. and Europe. Cost of sales totaled $0.8 million for the third quarter of 2016.

Research and development expenses increased to $73.4 million for the third quarter of 2017, compared to $60.8 million for the third quarter of 2016, driven primarily by increased headcount, the advancement of our earlier-stage immuno-oncology portfolio, and expansion of the TSR-042 and TSR-022 programs.

Selling, general and administrative expenses increased to $84.0 million for the third quarter of 2017, compared to $37.7 million for the third quarter of 2016, primarily due to increased headcount, activities in support of the launches of ZEJULA and VARUBY in the U.S. and Europe, and higher professional service fees.

Operating expenses as described above include total non-cash, stock-based compensation expense of $25.0 million for the third quarter of 2017, compared to $12.9 million for the third quarter of 2016.

Net loss totaled $25.3 million, or ($0.47) per share, for the third quarter of 2017, compared to a net loss of $87.9 million, or ($1.72) per share, for the third quarter of 2016.

As of September 30, 2017, TESARO had approximately $521.3 million in cash and cash equivalents and approximately 54.4 million outstanding shares of common stock.

Corporate Objectives

TESARO intends to achieve the following key objectives:

Commercial Products:

Continue to execute on the ongoing U.S. launch of ZEJULA and solidify its position as the market-leading PARP inhibitor for patients with recurrent ovarian cancer;
Launch ZEJULA in Europe by year-end 2017, pending European Commission approval; and
Launch VARUBI IV in the U.S.
Pipeline Candidates:

Rapidly advance the GARNET registration trial of TSR-042 in MSI-H cancers, with the intent of supporting accelerated FDA approval;
Define the registration path in platinum-resistant ovarian cancer and triple negative breast cancer in Q1 2018, pending data from TOPACIO trial;
Report initial data for the AMBER trial of TSR-022 in combination with TSR-042 in 2018;
Complete the dose escalation phase of the TSR-033 CITRINO trial and in early 2018 initiate a combination trial of TSR-033 plus TSR-042;
Initiate OVARIO, a Phase 2 clinical trial of niraparib in combination with bevacizumab in patients with first-line ovarian cancer by year end;
Initiate a Phase 3 clinical trial of niraparib in combination with TSR-042 in first-line ovarian cancer in 1H 2018; and
Initiate a Phase 3 clinical trial of niraparib in combination with TSR-042 in NSCLC in 2H 2018.