On November 15, 2018 Exelixis, Inc. (Nasdaq: EXEL) reported that its partner Ipsen received approval from the European Commission (EC) for CABOMETYX (cabozantinib) tablets as a monotherapy for hepatocellular carcinoma (HCC) in adults who have previously been treated with sorafenib (Press release, Exelixis, NOV 15, 2018, View Source;p=irol-newsArticle&ID=2377258 [SID1234531351]). This approval allows for the marketing of CABOMETYX in this indication in all 28 member states of the European Union, Norway and Iceland.

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"The approval of CABOMETYX in the Europe Union is a very important milestone for our partner Ipsen and marks significant progress for people living with liver cancer, which is the second-leading cause of cancer death worldwide," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. "This patient community is in dire need of new options to treat this aggressive disease, and we are excited to work with Ipsen to make this treatment available to patients in the European Union and other countries worldwide."

Under the terms of the Collaboration Agreement with Ipsen, Exelixis will receive a milestone payment of $40 million for the approval of the second-line treatment of HCC. This milestone will be paid by Ipsen within the next 70 days.

"Today’s European Commission approval of CABOMETYX provides a much-needed new option for HCC patients. Until now, physicians in Europe had only one approved therapy for the second-line treatment of this aggressive and difficult-to-treat cancer. We are proud to offer CABOMETYX as an innovative treatment that has been shown to extend survival in previously treated patients with HCC," said Harout Semerjian, Chief Commercial Officer of Ipsen. "This new indication reinforces Ipsen’s commitment to improving patients’ lives through the expansion of the clinical benefit of CABOMETYX in the treatment of solid tumors."

The EC approval is based on results from the CELESTIAL trial of CABOMETYX in patients with advanced HCC who received prior sorafenib. In this phase 3 pivotal trial, CABOMETYX demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo. CABOMETYX is also approved in the European Union for the treatment of advanced renal cell carcinoma (RCC) in adults who have received prior VEGF-targeted therapy and for previously untreated intermediate- or poor-risk advanced RCC.

On May 29, 2018, Exelixis announced that the U.S. Food and Drug Administration (FDA) accepted for filing the supplemental New Drug Application (sNDA) for CABOMETYX for previously treated advanced HCC and assigned a Prescription Drug User Fee Act action date of January 14, 2019. An sNDA is an application to the FDA that, if approved, will allow a drug sponsor to make changes to a previously approved product label, including modifications to the indication.

About the CELESTIAL Study

CELESTIAL is a randomized, double-blind, placebo-controlled study of cabozantinib in patients with advanced HCC conducted at more than 100 sites globally in 19 countries. The trial was designed to enroll 760 patients with advanced HCC who received prior sorafenib and may have received up to two prior systemic cancer therapies for HCC and had adequate liver function. Enrollment of the trial was completed in September 2017. Patients were randomized 2:1 to receive 60 mg of cabozantinib once daily or placebo and were stratified based on etiology of the disease (hepatitis C, hepatitis B or other), geographic region (Asia versus other regions) and presence of extrahepatic spread and/or macrovascular invasion (yes or no). No cross-over was allowed between the study arms during the blinded treatment phase of the trial. The primary endpoint for the trial is OS, and secondary endpoints include objective response rate and PFS. Exploratory endpoints include patient-reported outcomes, biomarkers and safety.

In October 2017, Exelixis announced that the independent data monitoring committee for the CELESTIAL study recommended that the trial be stopped for efficacy following review at the second planned interim analysis, with cabozantinib providing a statistically significant and clinically meaningful improvement in OS compared with placebo in patients with previously treated advanced HCC. The data, originally presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Gastrointestinal Cancers Symposium (ASCO-GI) in January 2018, were published in The New England Journal of Medicine in July 2018.1

About HCC

Liver cancer is the second-leading cause of cancer death worldwide, accounting for more than 700,000 deaths and 800,000 new cases each year.2 In the U.S., the incidence of liver cancer has more than tripled since 1980.3 HCC is the most common form of liver cancer, making up about three-fourths of the estimated nearly 42,000 new cases in the U.S. in 2018.3 HCC is the fastest-rising cause of cancer-related death in U.S.4 Without treatment, patients with advanced HCC usually survive less than 6 months.5

About the Exelixis and Ipsen Collaboration

In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. Under the terms of the Collaboration Agreement with Ipsen, Exelixis is entitled to receive a tiered royalty of 22 percent to 26 percent of annual net sales.

About CABOMETYX (cabozantinib)

CABOMETYX tablets are approved in the United States for the treatment of patients with advanced RCC. CABOMETYX tablets are also approved in: the European Union, Norway, Iceland, Australia, Switzerland, South Korea, Canada, Brazil and Taiwan for the treatment of advanced RCC in adults who have received prior VEGF-targeted therapy; in the European Union for previously untreated intermediate- or poor-risk advanced RCC; in Canada for adult patients with advanced RCC who have received prior VEGF targeted therapy; and in the European Union, Norway and Iceland for HCC in adults who have previously been treated with sorafenib. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan.

Please see Important Safety Information below and full U.S. prescribing information at View Source

U.S. Important Safety Information

Hemorrhage: Severe and fatal hemorrhages have occurred with CABOMETYX. In two RCC studies, the incidence of Grade ≥ 3 hemorrhagic events was 3% in CABOMETYX-treated patients. Do not administer CABOMETYX to patients that have or are at risk for severe hemorrhage.
Gastrointestinal (GI) Perforations and Fistulas: In RCC studies, fistulas were reported in 1% of CABOMETYX-treated patients. Fatal perforations occurred in patients treated with CABOMETYX. In RCC studies, gastrointestinal (GI) perforations were reported in 1% of CABOMETYX-treated patients. Monitor patients for symptoms of fistulas and perforations, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a fistula which cannot be appropriately managed or a GI perforation.
Thrombotic Events: CABOMETYX treatment results in an increased incidence of thrombotic events. In RCC studies, venous thromboembolism occurred in 9% (including 5% pulmonary embolism) and arterial thromboembolism occurred in 1% of CABOMETYX-treated patients. Fatal thrombotic events occurred in the cabozantinib clinical program. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or any other arterial thromboembolic complication.
Hypertension and Hypertensive Crisis: CABOMETYX treatment results in an increased incidence of treatment-emergent hypertension, including hypertensive crisis. In RCC studies, hypertension was reported in 44% (18% Grade ≥ 3) of CABOMETYX-treated patients. Monitor blood pressure prior to initiation and regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume CABOMETYX at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy. Discontinue CABOMETYX if there is evidence of hypertensive crisis or severe hypertension despite optimal medical management.
Diarrhea: In RCC studies, diarrhea occurred in 74% of patients treated with CABOMETYX. Grade 3 diarrhea occurred in 11% of patients treated with CABOMETYX. Withhold CABOMETYX in patients who develop intolerable Grade 2 diarrhea or Grade 3-4 diarrhea that cannot be managed with standard antidiarrheal treatments until improvement to Grade 1; resume CABOMETYX at a reduced dose.
Palmar-Plantar Erythrodysesthesia (PPE): In RCC studies, palmar-plantar erythrodysesthesia (PPE) occurred in 42% of patients treated with CABOMETYX. Grade 3 PPE occurred in 8% of patients treated with CABOMETYX. Withhold CABOMETYX in patients who develop intolerable Grade 2 PPE or Grade 3 PPE until improvement to Grade 1; resume CABOMETYX at a reduced dose.
Reversible Posterior Leukoencephalopathy Syndrome (RPLS), a syndrome of subcortical vasogenic edema diagnosed by characteristic finding on MRI, occurred in the cabozantinib clinical program. Perform an evaluation for RPLS in any patient presenting with seizures, headache, visual disturbances, confusion or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.
Embryo-fetal Toxicity may be associated with CABOMETYX. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during CABOMETYX treatment and for 4 months after the last dose.
Adverse Reactions: The most commonly reported (≥25%) adverse reactions are: diarrhea, fatigue, nausea, decreased appetite, hypertension, PPE, weight decreased, vomiting, dysgeusia, and stomatitis.
Strong CYP3A4 Inhibitors: If concomitant use with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage.
Strong CYP3A4 Inducers: If concomitant use with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage.
Lactation: Advise women not to breastfeed while taking CABOMETYX and for 4 months after the final dose.
Hepatic Impairment: In patients with mild to moderate hepatic impairment, reduce the CABOMETYX dosage. CABOMETYX is not recommended for use in patients with severe hepatic impairment

On November 15, 2018 Syros Pharmaceuticals (NASDAQ: SYRS), a leader in the development of medicines that control the expression of genes, reported that data from the dose escalation portion of its Phase 1 trial of SY-1365, its first-in-class selective cyclin-dependent kinase 7 (CDK7) inhibitor, demonstrated proof-of-mechanism at tolerable doses in patients with advanced solid tumors (Press release, Syros Pharmaceuticals, NOV 15, 2018, View Source [SID1234531367]). These data, the first clinical data reported on a selective CDK7 inhibitor, were highlighted in an oral plenary session at the 30th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Molecular Targets and Cancer Therapeutics Symposium in Dublin.

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"These initial data on SY-1365 are highly encouraging," said Dejan Juric, M.D., Director of the Termeer Center for Targeted Therapies at Massachusetts General Hospital and a clinical investigator in the Phase 1 study of SY-1365. "Patient data from the SY-1365 dose escalation study confirm the unique mechanism-of-action of this agent and demonstrate an acceptable tolerability profile along with early signs of single-agent activity. These data, coupled with preclinical evidence showing robust anti-tumor activity in a range of relapsed and treatment-refractory cancer models, support the ongoing development of SY-1365 for patients who currently have few, if any, effective treatment options."

"As the first clinical data ever reported on a selective CDK7 inhibitor, these results mark an important milestone for SY-1365 and for the field of CDK7 inhibition," said David A. Roth, M.D., Syros’ Chief Medical Officer. "We believe CDK7 inhibition is a potentially transformative new approach for treating many cancers that have eluded effective treatment with existing approaches. Now that we have demonstrated proof-of-mechanism at tolerable doses, we are committed to thoroughly exploring the potential of CDK7 inhibition for currently underserved patients. We are working to rapidly enroll the expansion cohorts in our ongoing Phase 1 study, focused initially on ovarian and breast cancers, while building on our leadership by advancing our highly selective and potent oral CDK7 inhibitor, SY-5609, as our next development candidate."

Dose Escalation Data

The dose escalation portion of the Phase 1 trial characterized the safety, pharmacokinetics (PK) and pharmacodynamics (PD) of SY-1365 in patients with advanced solid tumors to establish a dose and regimen for the expansion portion of the trial. PD assays used to establish proof-of-mechanism included a CDK7 occupancy assay to evaluate SY-1365 binding and a custom gene expression assay to evaluate downstream transcriptional changes in patients. Preliminary anti-tumor activity was also assessed.

Enrollment in the dose escalation portion of the trial was completed in September. In total, 32 patients were treated with SY-1365 as a single agent at doses ranging from 2 mg/m2 to 112 mg/m2 using either a weekly or twice weekly dosing regimen. Patients were treated for three weeks out of each four-week cycle. Patients had a range of solid tumors, the most prevalent being ovarian cancer (eight patients), breast cancer (eight patients) and endometrial cancer (five patients). Patients’ median age was 63 (ranging from 25 to 87), with a median of five prior therapies (ranging from one to 13). As of an October 15th data snapshot, the median treatment duration was 46.5 days (ranging from two to 147 days) and four patients remained on treatment.

Safety

Adverse events (AEs) were predominantly low-grade, reversible and generally manageable.
The most commonly reported AEs were headache, nausea, vomiting and fatigue.
No neutropenia was reported.
Dose-limiting toxicities were headache, coronary vasospasm and fatigue.
A maximum tolerated dose was not defined.
Pharmacokinetics

Plasma PK exposures were linear over the doses tested.
No drug accumulation was observed with repeat dosing.
Proof-of-Mechanism

SY-1365 demonstrated dose-dependent effects on CDK7 occupancy and downstream gene expression changes in blood cells.
At doses of 32 mg/m2 and higher, CDK7 occupancy was greater than 50 percent when measured three days following dose administration, exceeding target occupancy levels in preclinical models that correlated with anti-tumor activity.
CDK7 occupancy in blood cells was similar to target occupancy in tumor tissue biopsies available from two patients in the clinical trial.
Early Signs of Clinical Activity

As of the October 15th data snapshot, clinical activity per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria was observed in seven of the 19 patients (37%) who were evaluable for clinical responses, including:

One patient with ovarian cancer in her fourth relapse who had a confirmed partial response (PR) after two cycles of treatment at the 80 mg/m2 twice-weekly dose. The patient remained in PR at her CT assessment after six cycles and recently entered her seventh month on study treatment.
Six additional patients who had stable disease, lasting between 50 and 127 days. Most of these patients received doses equal to or greater than 32 mg/m2.
Based on these data, Syros selected a twice-weekly dose of 80 mg/m2 of SY-1365 when administered as a single agent, and a once-weekly target dose of 80 mg/m2 of SY-1365 when administered in combination with other agents, for further evaluation in the ongoing Phase 1 expansion cohorts in multiple ovarian and breast cancer patient populations.

Ongoing Expansion of Phase 1 Trial

Upon completing enrollment in the dose-escalation portion of the trial, Syros opened expansion cohorts to further assess the safety and anti-tumor activity of SY-1365 in multiple ovarian and breast cancer patient populations. The initial expansion strategy is based on preclinical data showing anti-tumor activity in these tumor types, a strong mechanistic rationale and high unmet need. The expansion cohorts are evaluating SY-1365: as a single agent in primary platinum-refractory ovarian cancer patients; as a single agent in ovarian cancer patients who have relapsed after three or more therapies; in combination with carboplatin in ovarian cancer patients who have relapsed after one or more prior therapies; and in combination with fulvestrant in patients with hormone-receptor positive (HR+) metastatic breast cancer who have progressed after treatment with a CDK4/6 inhibitor. An additional cohort is enrolling patients with any solid tumor accessible for biopsy to further evaluate the mechanism of action of SY-1365. Additional details about the trial can be found using the identifier NCT03134638 at www.clinicaltrials.gov.

The dose escalation data presented at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) meeting is now available on the Publications and Abstracts section of the Syros website at www.syros.com.

Conference Call and Webcast

Syros will host a conference call today at 4:00 p.m. ET to discuss the data from the dose escalation portion of its Phase 1 trial.

The live call may be accessed by dialing (866) 595-4538 for domestic callers or (636) 812-6496 for international callers and referencing conference ID number: 4567679. A live webcast of the conference call will be available online on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 90 days.

On November 15, 2018 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported that Center for Drug Evaluation of China’s National Medical Product Administration (NMPA, formerly known as CFDA) has granted priority review status to the New Drug Applications (NDAs) for the company’s investigational BTK inhibitor zanubrutinib in patients with relapsed/refractory (R/R) mantle cell lymphoma (MCL) and for its investigational anti-PD-1 antibody tislelizumab in patients with R/R classical Hodgkin’s lymphoma (cHL) (Press release, BeiGene, NOV 15, 2018, View Source;p=irol-newsArticle&ID=2377287 [SID1234531352]).

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"We’re excited that the NDAs for both zanubrutinib and tislelizumab are receiving priority review by the NMPA," commented Wendy Yan, Senior Vice President, Global Head of Regulatory Affairs at BeiGene. "We look forward to working closely with the NMPA and CDE in the coming months and hope to bring more treatment options to cancer patients."

Zanubrutinib and tislelizumab were both discovered in BeiGene’s research facilities in Beijing, China. Zanubrutinib is being developed globally by BeiGene as a monotherapy and in combination with other therapies to treat various hematologic malignancies. The NDAs for zanubrutinib as a potential treatment for patients with R/R MCL and for patients with R/R chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) were accepted by the NMPA in August and October 2018, respectively. Tislelizumab is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid and hematologic cancers. The NDA for tislelizumab as a potential treatment for patients with R/R cHL was accepted by the NMPA in August 2018.

Priority review and approval was established in China to facilitate drug registration management and accelerate the development of new drugs with clinical value under the guidance of Opinions on the Reform of the Review & Approval System for Drugs and Medical Devices issued by the State Council in August 2015, and Opinions on Encouraging Pharmaceutical Innovation via Priority Review & Approval issued by CFDA in December 2017. According to these guidelines, the regulatory authority will prioritize the review process and evaluation resources for applications under priority review should expect prioritized review and inspection resource and reduced review and approval timelines.

About Zanubrutinib
Zanubrutinib (BGB-3111) is an investigational small molecule inhibitor of Bruton’s tyrosine kinase (BTK). Zanubrutinib was discovered in BeiGene’s research facilities in Beijing, China, and is being developed globally by BeiGene as a monotherapy and in combination with other therapies to treat various hematologic malignancies.

About Tislelizumab
Tislelizumab (BGB-A317) is an investigational humanized monoclonal antibody that belongs to a class of immuno-oncology agents known as immune checkpoint inhibitors. Discovered by BeiGene scientists in Beijing, tislelizumab is designed to bind to PD-1, a cell surface receptor that plays an important role in downregulating the immune system by preventing the activation of T-cells. Tislelizumab has demonstrated high affinity and specificity for PD-1 in pre-clinical studies. It is potentially differentiated from the currently approved PD-1 antibodies in an engineered Fc region, which is believed to minimize potentially negative interactions with other immune cells, based on preclinical data. Tislelizumab is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers. BeiGene and Celgene Corporation have a global strategic collaboration for the development of tislelizumab in solid tumor cancers outside of Asia (except Japan).

On November 15, 2018 NantHealth, Inc. (NASDAQ-GS: NH), a next-generation, evidence-based, personalized healthcare company, reported financial results for its third quarter ended September 30, 2018 (Press release, NantHealth, NOV 15, 2018, View Source [SID1234531369]).

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Sequencing and Molecular Analysis – Highlights

In September 2018, at the IASLC 19th World Conference on Lung Cancer, the company and NantOmics presented two posters and a mini-oral presentation titled "Blood Based Biomarkers: RNA, KRAS and PD-L1 Strongly Matching with Tissue and Showing Correlation with Clinical Responses In NSCLC Patients," with investigational data showing (i) association between clinical response and changes in plasma cfRNA levels and (ii) that plasma levels of PD-L1 expression, while on immunotherapy, could be used to monitor clinical responses.
In October 2018, at ESMO (Free ESMO Whitepaper), the world’s second largest cancer symposium, the company, along with NantOmics, conducted one oral presentation and presented four papers, including papers demonstrating the promise of Liquid GPS, the company’s blood-based molecular test that provides oncologists with a powerful tool for non-invasive tumor profiling and quantitative monitoring of treatment response.
Expanded Sequencing and Molecular Analysis Reimbursement Arrangements: In Q3, the company expanded its GPS reimbursement contract with a large, national employer in the healthcare industry to include reimbursement for its Liquid GPS test and, in Q4, the company expanded GPS Cancer and Liquid GPS coverage for firefighters through the execution of an additional reimbursement contract benefiting members of a city-wide firefighter union.
"We recently made two oral presentations at major medical congresses, highlighting the unique aspects of our liquid biopsy platform that are different from ctDNA platforms offered by Guardant Health, Foundation Medicine and other laboratories," said Sandeep (Bobby) Reddy, M.D., Chief Medical Officer of NantHealth. "Only Liquid GPS can produce this type of data on chemotherapy selection and immunotherapy selection. Moreover, the review committees of two different organizations felt that this data was sufficiently important to warrant oral presentation. This further validates our belief that circulating RNA expression, not DNA alone, is the liquid biopsy technology that will transform medical practice."

Software and Services Highlights:

Payer Engagement (NaviNet):
In Q3, executed a large-scale implementation of NaviNet Open Authorizations with a key customer, adding more than 40,000 end users, bringing total active number of users to 862,000
Clinical Decision Support (Eviti):
Released new functionality with drug specific justifications, enabling the capture of key data to ensure quicker pre-authorization and clinical integrity.
Completed a new statewide implementation with a large existing payer in Kentucky, adding 250,000 covered lives. This implementation followed a similar success in Florida in Q2 for the same national payer.
Connected Care:
In Q4, collaborated with B. Braun Australia, GE Healthcare and iProcedures to demonstrate the exchange of data between patient devices and medical records at the HIMSS Interoperability Showcase as part of the Healthcare Information and Management Systems Society (HIMSS) Asia Pacific Conference.
"We are pleased to report that total net revenue for the 2018 third quarter increased to $22.3 million, up from the comparable prior year period, as well as on a sequential quarterly basis," said Bob Petrou, Interim Chief Financial Officer of NantHealth. "Looking ahead, the recent addition of two new executives to lead our strategy and business development, and global sales efforts will help us expand our business in both the U.S. and international markets."

Business and Financial Highlights

The company adopted a new revenue recognition standard on January 1, 2018. Please note that the financial results presented below include both amounts "as presented," which reflect implementation of the new revenue recognition standard, as well as amounts prior to the impact of the new revenue recognition standard to allow for comparability against historical results. Starting in fiscal year 2019, the company will no longer present its GAAP and Non-GAAP financial results under the previous revenue recognition standard. For additional information and reconciliations of our financial results between the new and previous revenue recognition standard, see the additional tables included in this press release and in the company’s Form 10-Q to be filed with the Securities and Exchange Commission.

For the 2018 third quarter, total net revenue as presented was $22.3 million. Total 2018 third quarter net revenue prior to the impact of the new revenue recognition standard increased to $22.0 million from $21.8 million in 2017 third quarter. Gross profit as presented was $11.1 million, or 50% of total net revenue. Gross profit prior to the impact of the new revenue recognition standard was $10.8 million, or 49% of total net revenue, compared with $10.3 million, or 47% of total net revenue, for the prior-year third quarter. Selling, general and administrative (SG&A) expenses as presented were $17.0 million. SG&A prior to the impact of the new revenue recognition standard was $16.9 million compared with $17.5 million in 2017 third quarter. Research and development (R&D) expenses as presented decreased to $4.8 million from $7.7 million; the new revenue recognition standard did not impact R&D expenses.

Financial results for the third quarter of 2018 included a non-cash charge for loss from related party equity method investment, including impairment, of $83.3 million. Net loss from continuing operations, net of tax, as presented was $97.4 million, or $0.89 per share. Net loss from continuing operations, net of tax, prior to the impact of the new revenue recognition standard was $97.5 million, or $0.89 per share, from $23.0 million, or $0.20 per share for the 2017 third quarter. Loss from discontinued operations, net of tax, as presented was $32,000, or $0.00 per share, compared with $19.4 million, or $0.17 per share; the new revenue recognition standard did not impact loss from discontinued operations. Net loss as presented was $97.5 million, or $0.89 per share. Net loss prior to the impact of the new revenue recognition standard was $97.5 million, or $0.89 per share, compared with $42.4 million, or $0.37 per share, for 2017 third quarter.

For the 2018 third quarter, on a non-GAAP basis, adjusted net loss from continuing operations as presented was $10.8 million, or $0.10 per share. On a non-GAAP basis, adjusted net loss from continuing operations prior to the impact of the new revenue recognition standard was $10.9 million, or $0.10 per share, compared with $14.9 million, or $0.13 per share, for the 2017 third quarter.

In August 2017, NantHealth sold its provider/patient engagement assets to Allscripts to focus on core competencies and accelerate the plan to achieve profitability. As a result, the company has classified the current and prior period operating results of its provider/patient engagement business as discontinued operations. All results presented above represent the company’s continuing operations.

Conference Call Information and Forward-Looking Statements

Later today, the company will host a conference call at 1:30 p.m. PT (4:30 p.m. ET) to review its results of operations for the third quarter ended September 30, 2018. The conference call will be available to interested parties by dialing 844-309-3709 from the U.S. or Canada, or 281-962-4864 from international locations, passcode 9992769. The call will be broadcast via the Internet at www.nanthealth.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary audio software. A playback of the call will be archived and accessible on the same website for at least three months.

Discussion during the conference call may include forward-looking statements regarding topics such as the company’s financial status and performance, regulatory and operational developments, and other comments the company may make about its future plans or prospects in response to questions from participants on the conference call.

Use of Non-GAAP Financial Measures

This news release contains references to Non-GAAP financial measures, including adjusted net loss and adjusted net loss per share, which are financial measures that are not prepared in conformity with United States generally accepted accounting principles (U.S. GAAP). The Company’s management believes that the presentation of Non-GAAP financial measures provides useful supplementary information regarding operational performance, because it enhances an investor’s overall understanding of the financial results for the Company’s core business. Additionally, it provides a basis for the comparison of the financial results for the Company’s core business between current, past and future periods. Other companies may define these measures in different ways. Non-GAAP financial measures should be considered only as a supplement to, and not as a substitute for or as a superior measure to, financial measures prepared in accordance with U.S. GAAP. Non-GAAP per share numbers are calculated based on one class of common stock and do not incorporate the effects, if any, of using the two-class method.

On N ovember 14, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported its financial results for the third quarter ended September 30, 2018 and announces several business updates, including new management appointments and a new Company name and ticker symbol (Press release, RXi Pharmaceuticals, NOV 14, 2018, View Source [SID1234531293]).

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"In the past few months, we have focused on continuing to generate meaningful data through our R&D activities, while at the same time being extremely diligent in spending our cash in a way that can maximize the outcome," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. "The significant reduction in net loss for the third quarter, and for the first nine months of 2018, has been the result of a careful selection of academic and industrial partners who have shown a high interest in the potential of our self-delivering platform for RNAi in immuno-oncology (IO) and adoptive cell transfer therapy (ACT). In addition, we have been able to complete a financing of $15 million last month, providing us with a financial runway we expect to last until at least through the first half of 2020."

Dr. Cauwenbergh continued, "The pending name change from RXi Pharmaceuticals to Phio Pharmaceuticals signifies our commitment to develop our sd-rxRNA platform as a key therapeutic modality in the rapidly expanding field of IO and ACT by weaponizing the body’s immune system in its fight against cancer. Also, in that context, I am very pleased to announce the appointment of Dr. Gerrit Dispersyn as our President and Chief Operating Officer. In the past 18 months, Gerrit has shown a remarkable aptitude in handling the complexities of preclinical and clinical R&D activities in our Company and I believe that under his leadership our Company will succeed in executing its goals, which should result in our shareholders doing well, and most importantly, patients doing well."

Dr. Gerrit Dispersyn said, "I am grateful for the opportunity to provide additional leadership for our Company’s new chapter and looking forward to delivering on our upcoming milestones. Under Geert’s direction, the Company built an impressive dataset that allows us to confidently develop the next generation of immuno-oncology therapeutics based on our sd-rxRNA platform. I look forward to building on that foundation alongside Geert."

Conference Call Information

The Company will host a conference call today at 4:30 p.m. EST to discuss financial results and provide an update on the Company. The webcast link will be available under the "Investors – Event Calendar" section of the Company’s website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States and Canada: +1 844-376-4678. International participants may access the event by dialing: +1 209-905-5958. An archive of the webcast will be available on the Company’s website approximately two hours after the presentation.

Select Financial Highlights for Q3 2018

Cash Position

At September 30, 2018, the Company had cash and cash equivalents of $3.2 million as compared with $3.6 million at December 31, 2017.

On October 3, 2018, the Company closed an underwritten public offering of (i) 3,725,714 units, at a public offering price of $0.70 per unit, with each unit consisting of one share of common stock and one warrant to purchase one share of common stock and (ii) 17,702,858 pre-funded units, at a public offering price of $0.69 per pre-funded unit, with each pre-funded unit consisting of one pre-funded warrant to purchase one share of common stock and one warrant. Assuming the warrants are not exercised, net proceeds from the offering were approximately $13.3 million after deducting underwriting discounts and commissions and offering expenses paid by the Company.

The Company believes that with its current cash on hand and the proceeds from the underwritten offering, it has sufficient cash to fund operations through the first half of 2020, which includes initiating a clinical trial with RXI-762 and advancing our immuno-oncology programs toward clinical development. This assumes no new funding through licensing or collaboration agreements.

Revenues

Revenues for the three months ended September 30, 2018 were $57,000 and related to the work performed by the Company as a sub-awardee under the government grant awarded for the development of a novel sd-rxRNA compound, BA-434, that targets PTEN for the treatment of spinal cord injury. The Company had no revenues during the three months ended September 30, 2017.

Research and Development Expenses

Research and development expenses for the quarter ended September 30, 2018 were $0.8 million as compared with $1.5 million for the quarter ended September 30, 2017. The decrease was primarily due to a decrease in clinical trial-related expenses as subject participation is complete for all of the Company’s clinical trials in dermatology and ophthalmology and a decrease in payroll-related expenses due to a reduction in headcount as compared with the prior year period.

General and Administrative Expenses

General and administrative expenses for the quarter ended September 30, 2018 were $0.7 million as compared with $1.0 million for the quarter ended September 30, 2017. The decrease was primarily due to a decrease in payroll-related expenses, including those as a result of a reduction in headcount, as compared to the prior year period.

Net Loss

Net loss for the quarter ended September 30, 2018 was $1.5 million compared with $2.5 million for the quarter ended September 30, 2017. The decrease was primarily due to a decrease in operating expenses, as discussed above.

Recent Business Highlights

Research Collaboration with Karolinska Institutet

In August 2018, the Company announced it entered into a research collaboration with the Karolinska Institutet in Stockholm, Sweden. This collaboration will explore RXi’s sd-rxRNA compounds against targets involved in T-cell and NK cell differentiation and/or in the immune cell tumor-induced stress response with the aim of producing anti-tumor adoptive cell therapy grafts with improved functionality and persistence.

New Data Using sd-rxRNA in NK Cells

In September 2018, the Company presented data from its immuno-oncology program using sd-rxRNA therapeutic compounds targeting intracellular checkpoint Cbl-b in NK cells at the 16th Annual Discovery on Target Conference. Data showed that sd-rxRNAs are rapidly and efficiently taken up by immune effector cells without the use of transfection reagents. Using sd-rxRNA compounds against checkpoint inhibitors, we can suppress their expression levels up to 95% in immune cells, including T-cells and NK cells. By treating NK cells ex-vivo, prior to adoptive cell transfer with sd-rxRNA reducing the expression of proteins such as Cbl-b, the anti-tumor response of these cells may be improved.

In November 2018, the Company presented a poster highlighting data demonstrating the potential of sd-rxRNA to improve NK cell potency in ACT at the 33rd Annual Meeting & Pre-Conference Programs of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). Data showed that by treating NK cells with sd-rxRNA targeting inhibitory receptors such as TIGIT ex vivo, prior to ACT, the anti-tumor response of these cells may be enhanced resulting in a more effective therapy for hematological malignancies.

Additional Corporate Events Announced

Corporate Name Change

As the Company transformed into an immuno-oncology company with the acquisition of MirImmune in 2017 and the strategic decision to focus solely on immuno-oncology in 2018, it became clear that the name RXi Pharmaceuticals no longer accurately reflected the Company’s current business and future direction. The new name reflects the Company’s commitment to providing new treatment options for patients battling cancer.

The Company expects the change in the Company’s name from RXi Pharmaceuticals Corporation to Phio Pharmaceuticals Corp. and the change in the Company’s ticker symbol from RXII to the new trading symbol PHIO to become effective in the coming week.

Appointment of Gerrit Dispersyn as President and Chief Operating Officer

Dr. Dispersyn has been the Company’s Chief Development Officer since April 2017. In connection with the Company’s recently announced corporate name change to better reflect its core focus on immuno-oncology, Dr. Dispersyn will become the Company’s President and Chief Operating Officer as of today. As COO, he will oversee all day-to-day activities of the Company and is in line to become the next CEO of the Company. Dr. Cauwenbergh remains as CEO and a member of the Board of Directors of the Company and will focus on financial, intellectual property and strategic matters.