On August 7, 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 that it will report its financial results for the second quarter ended June 30, 2018, and provide a business update on Tuesday, August 14, 2018 after the close of the U.S. financial markets (Press release, RXi Pharmaceuticals, AUG 7, 2018, View Source [SID1234528489]).

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A live audio webcast will begin at 4:30 p.m. EDT. The webcast link is available under the "Investors – Events and Presentations" section of the Company’s website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States: +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.

On August 7, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the second quarter of 2018 and updated financial guidance for 2018 (Press release, Jazz Pharmaceuticals, AUG 7, 2018, View Source [SID1234528488]).

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"We had another highly productive quarter, including strong commercial performance, achievement of significant regulatory and R&D milestones, and further strengthening of our balance sheet," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "We head into the second half of 2018 focused on supporting our sales momentum, progressing our pre-clinical and clinical pipeline, deploying our resources to expand the business through R&D and corporate development activities, and preparing for multiple near-term regulatory milestones, including three potential marketing approvals."

GAAP net income for the second quarter of 2018 was $92.3 million, or $1.50 per diluted share, compared to $105.6 million, or $1.72 per diluted share, for the second quarter of 2017. GAAP net income for the second quarter of 2018 included an impairment charge of $42.9 million resulting from the company’s decision to sell its rights related to Prialt.

Adjusted net income for the second quarter of 2018 was $214.6 million, or $3.49 per diluted share, compared to $157.4 million, or $2.56 per diluted share, for the second quarter of 2017. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

Total revenues increased 27% in the second quarter of 2018 compared to the same period in 2017 due to the contribution of strong sales from Xyrem, Erwinaze/Erwinase, Defitelio and the addition of Vyxeos following the launch in August 2017.

Xyrem net product sales increased 19% in the second quarter of 2018 compared to the same period in 2017.

Erwinaze/Erwinase net product sales increased 20% in the second quarter of 2018 compared to the same period in 2017. The company experienced supply disruptions during both periods and fluctuations in quarterly results reflect, in part, the timing of supply availability. The company expects continued supply challenges from time to time for the remainder of 2018.

Defitelio/defibrotide net product sales increased 34% in the second quarter of 2018 compared to the same period in 2017. The company continues to expect inter-quarter variability in Defitelio net sales given that veno-occlusive disease is an ultra-rare disease.

Operating expenses changed over the prior year period primarily due to the following:

Selling, general and administrative (SG&A) expenses increased in the second quarter of 2018 compared to the same period in 2017 on a GAAP and on a non-GAAP adjusted basis due to higher expenses resulting from the expansion of the company’s business, including pre-launch activities for the potential approvals of Vyxeos in the EU and solriamfetol in the U.S.
Research and development (R&D) expenses increased in the second quarter of 2018 compared to the same period in 2017 on a GAAP and on a non-GAAP adjusted basis due to an increase in expenses related to the company’s pre-clinical and clinical development programs, regulatory activities and support of partner programs.
Cash Flow and Balance Sheet

As of June 30, 2018, cash, cash equivalents and investments were $815.1 million and the outstanding principal balance of the company’s long-term debt was $1.8 billion. During the six months ended June 30, 2018, the company generated $354.0 million of cash from operations, purchased a priority review voucher for $110.0 million and used $55.6 million to repurchase approximately 373,000 ordinary shares under the company’s share repurchase program at an average cost of $149.16 per ordinary share.

In June 2018, the company refinanced its senior credit facilities to increase the borrowing capacity available under the revolving credit facility to $1.60 billion from $1.25 billion and to extend the maturity profile of the facilities to June 2023 from July 2021.

Recent Developments

At the Associated Professional Sleep Societies meeting in June 2018, the company presented long-term safety and efficacy results from its global multi-center studies evaluating Xyrem for the treatment of cataplexy in pediatric patients with narcolepsy and solriamfetol in adult patients with excessive sleepiness associated with obstructive sleep apnea and with narcolepsy.

In June 2018, the U.S. Food and Drug Administration (FDA) accepted for priority review the company’s supplemental new drug application (sNDA) seeking revised labeling for Xyrem to include an indication to treat cataplexy and excessive daytime sleepiness in pediatric narcolepsy patients. The Prescription Drug User Fee Act goal date for an FDA decision is October 27, 2018.

In June 2018, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending marketing authorization of Vyxeos for the treatment of adults with newly diagnosed t-AML or AML-MRC.

In July 2018, the company announced that data from the pivotal Phase 3 study of Vyxeos compared to standard of care cytarabine and daunorubicin (7+3) was published online in the Journal of Clinical Oncology.

In August 2018, the company announced that the United States Centers for Medicare and Medicaid Services granted approval for a New Technology Add-on Payment for Vyxeos for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

In August 2018, the company and The University of Texas MD Anderson Cancer Center announced a five-year collaboration to evaluate potential treatment options for hematologic malignancies, with a near-term focus on Vyxeos.

Conference Call Details

Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. EDT (9:30 p.m. IST) to provide a business and financial update and discuss its 2018 second quarter results. The live webcast may be accessed from the Investors section of the company’s website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 4989706.

A replay of the conference call will be available through August 14, 2018 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 4989706. An archived version of the webcast will be available for at least one week in the Investors section of the company’s website at www.jazzpharmaceuticals.com.

On August 7, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported financial results and business highlights for the second quarter ended June 30, 2018 (Press release, Tocagen, AUG 7, 2018, View Source [SID1234528487]).

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"We conclude the mid-point of 2018 on a high note, with robust site activation and enrollment in our Phase 3 Toca 5 clinical trial, a license agreement with ApolloBio for development and commercialization of Toca 511 & Toca FC in the greater China region, and continued progress in our pipeline-expanding R&D efforts," said Marty Duvall, chief executive officer of Tocagen. "We expect to continue to achieve a number of milestones during the second half of the year, including the completion of enrollment for the Toca 5 clinical trial in patients with recurrent brain cancer and conducting the first of two pre-planned interim analyses of the trial. During the second half of 2018, we also plan to advance our R&D programs to build our gene therapy pipeline."

Tocagen finished 2017 with $88.7 million in cash and cash equivalents. The cash guidance for 2018, provided early this year, implied a year-end projected cash balance of approximately $40 million. Recent progress has substantially strengthened Tocagen’s cash position to execute, expand and explore the company’s highly differentiated cancer-selective gene therapy platform. Tocagen now expects to conclude 2018 with approximately $70 million in cash and cash equivalents.

Second Quarter 2018 and Recent Highlights

Closed license agreement with ApolloBio: In July 2018, Tocagen and ApolloBio closed a license agreement to develop and commercialize Toca 511 & Toca FC within the greater China region. Under the terms of the agreement, Tocagen received an initial upfront payment of $16 million, before taxes. Tocagen is eligible to receive additional potential payments of $4 million in near-term development milestones, including completion of enrollment in the ongoing Phase 3 Toca 5 study, and an additional $107 million in development and commercial milestones, plus additional double-digit tiered sales royalties.
Restructured 2015 venture debt to strengthen cash position: In May 2018, Tocagen entered into an amended and restated loan and security agreement with Oxford Finance and Silicon Valley Bank. Under the agreement, approximately $18 million was added to Tocagen’s cash reserve and may be used to satisfy the company’s future working capital needs and to fund its general business requirements. Additionally, further principal amortization on this debt was deferred until January 2020.
Presentation and publication of Toca 511 & Toca FC data: At the 2018 American Academy of Neurology (AAN) Annual Meeting and the 2018 American Association of Neurological Surgeons (AANS) Annual Scientific Meeting, research collaborators presented updated durable response data from the Phase 1 study involving patients with recurrent high-grade glioma (rHGG) who received Toca 511 & Toca FC at the time of surgical resection. In May 2018, data were published online in Neuro-Oncology demonstrating the multiyear durable responses observed in rHGG patients treated in the Toca 511 & Toca FC Phase 1 trial. These data will be included in the Neuro-Oncology October print edition to be distributed at the 23rd Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology.
Advanced Toca 521 as part of pipeline-building strategy: At the 2018 Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), research collaborators introduced Toca 521, a retroviral replicating vector expressing a single-chain variable fragment targeting PD-L1. The preclinical data demonstrate Toca 521 has robust, durable and highly selective anti-tumor activity superior to systemically administered anti-PD-1 or anti-PD-L1 monoclonal antibodies. These data inform Tocagen’s plans to advance Toca 521 into investigational new drug application (IND) enabling studies this year.
Second Quarter 2018 Financial Results

Research and Development (R&D) Expenses: R&D expenses were $12.8 million for the quarter ended June 30, 2018, compared to $6.6 million for the quarter ended June 30, 2017. The increase in R&D expenses in 2018 was primarily driven by higher costs to support the expanded Toca 5 trial, manufacturing activities related to Toca 511 & Toca FC, and personnel and related costs due to increased headcount.

General and Administrative (G&A) Expenses: G&A expenses were $2.6 million for the quarter ended June 30, 2018, compared to $2.0 million for the quarter ended June 30, 2017. The increase in G&A expenses was primarily due to increased stock-based compensation expense.

Net Loss: Net loss was $16.1 million, or $0.81 per common share (basic and diluted), for the quarter ended June 30, 2018, compared to a net loss of $9.1 million, or $0.56 per common share (basic and diluted), for the quarter ended June 30, 2017. The 2018 calculation is based on 19.9 million average common shares outstanding for the second quarter of 2018, compared to 16.3 million average common shares outstanding for the second quarter of 2017. The average common shares outstanding for the second quarter of 2017 includes the issuance of 9.8 million common shares, as well as the conversion of convertible preferred stock and convertible promissory notes into 7.8 million common shares, upon Tocagen’s initial public offering in April 2017.

2018 Six-Month Results

R&D Expenses: R&D expenses were $23.2 million for the six months ended June 30, 2018 compared to $13.3 million for the six months ended June 30, 2017. Similar to the second quarter results, the R&D expenses primarily reflect increased costs to support the expanded Toca 5 Phase 3 clinical trial, manufacturing activities related to Toca 511 & Toca FC, and personnel and related costs due to increased headcount.

G&A Expenses: G&A expenses were $5.0 million for the six months ended June 30, 2018 compared to $4.0 million for the first six months ended June 30, 2017, with the increase primarily driven by higher stock-based compensation expense.

Net Loss: Net loss for the first six months ended June 30, 2018 was $29.0 million, or $1.45 per common share (basic and diluted), compared to a net loss of $18.1 million, or $1.95 per common share (basic and diluted), for the six months ended June 30, 2017. This calculation is based on 19.9 million average common shares outstanding for the six months ended June 30, 2018, compared to 9.3 million average shares outstanding for the same period in 2017.

Cash Position and Guidance

Cash, cash equivalents and marketable securities were $79.5 million at June 30, 2018 compared to $88.7 million at December 31, 2017. The decrease of approximately $10 million reflects the use of $29 million of cash during the six months ended June 30, 2018, partially offset by the net proceeds of approximately $18 million from the restructured debt and a $1 million installment payment from ApolloBio. Subsequent to the end of the second quarter, Tocagen closed its license agreement with ApolloBio and received a total upfront payment of $16 million before applicable China taxes. Tocagen reiterates its annual cash burn guidance and continues to estimate the total cash used in 2018 to fund operations, capital expenditures and debt amortization will not exceed $50 million, resulting in a year-end cash balance of approximately $70 million.

About Toca 511 & Toca FC

Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprised of an investigational biologic, Toca 511 and an investigational small molecule, Toca FC. Toca 511 (vocimagene amiretrorepvec) is a retroviral replicating vector (RRV) that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase (CD). Through this targeted delivery, infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered, extended-release formulation of the prodrug, 5-fluorocytosine (5-FC), which is converted into an anti-cancer drug, 5-fluorouracil (5-FU), when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells in the tumor microenvironment resulting in anti-cancer immune activation and subsequent tumor killing.

On August 7, 2018 Myovant Sciences (NYSE: MYOV), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of women’s health and endocrine diseases, reported corporate updates and reported financial results for the first fiscal quarter ended June 30, 2018 (Press release, Myovant Sciences, AUG 7, 2018, View Source [SID1234528486]).

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"We are off to an excellent start to the year, highlighted by the completion of patient screening for our Phase 3 LIBERTY 1 trial evaluating relugolix in women with heavy menstrual bleeding associated with uterine fibroids, and screening in the replicate LIBERTY 2 trial expected to be completed this quarter," said Lynn Seely, M.D., President and Chief Executive Officer of Myovant Sciences. "We are at an exciting stage in the evolution of the Company, with top-line safety and efficacy data expected for relugolix in 2019 in three distinct indications, each with large markets and wholly owned North American and European rights."

Recent Business Highlights and Upcoming Milestones

On July 10, 2018, Myovant announced completion of patient screening for its LIBERTY 1 trial, the first of two Phase 3 replicate trials evaluating relugolix in women with heavy menstrual bleeding associated with uterine fibroids. Top-line safety and efficacy data from this trial are expected in the second quarter of 2019.
Screening for the LIBERTY 2 trial is anticipated to be completed this quarter.
Completion of enrollment and top-line safety and efficacy data from the two Phase 3 SPIRIT trials of relugolix in women with endometriosis-associated pain are expected in 2019.
Enrollment is expected to be completed in 2018 for the Phase 3 HERO trial of relugolix in men with advanced prostate cancer, and top-line safety and efficacy data are expected in 2019.
Myovant raised aggregate net proceeds of approximately $150.0 million during fiscal 2018 from the issuance and sale of 3,333,334 common shares in an underwritten public offering in July 2018, 1,110,015 common shares in a private placement to the Company’s controlling shareholder, Roivant Sciences, Ltd. in April 2018, and 2,767,129 common shares in an "at-the-market" equity offering program in April 2018.
On May 30, 2018, Myovant entered into a Commercial Manufacturing and Supply Agreement with Takeda Pharmaceutical Company Ltd. pursuant to which Takeda will manufacture and supply Myovant with relugolix drug substance to support the commercial launch of relugolix, if marketing authorization is granted. Takeda has also agreed to assist with the transfer of technology and manufacturing know-how to a second contract manufacturing organization of Myovant.
In May 2018, following the completion of a Phase 1 study, Myovant initiated a Phase 2a clinical trial in healthy female volunteers to characterize the dose-response curve in the controlled ovarian stimulation setting prior to studying MVT-602 in infertile woman seeking pregnancy.
On April 20, 2018, Myovant announced a partnership with PERIOD, Inc., a youth-led non-profit organization focused on menstrual equity, to elevate the conversation around period health, including an Ask Me About Periods campaign on college campuses around the country.
First Fiscal Quarter 2018 Financial Summary

Research and development (R&D) expenses for the quarter ended June 30, 2018, were $51.3 million compared to $17.7 million for the comparable period in 2017. The increase for the quarter primarily reflects the progress of Myovant’s ongoing Phase 3 clinical trials of relugolix, which were initiated in 2017, and additional personnel-related expenses.

General and administrative (G&A) expenses for the quarter ended June 30, 2018, were $8.7 million compared to $4.2 million for the comparable period in 2017. The increase for the quarter primarily reflects increases in personnel-related expenses, professional service fees, and other administrative expenses to support Myovant’s headcount growth and expanding operations.

Interest expense for the quarter ended June 30, 2018, was $1.6 million compared to no interest expense in the comparable prior year period. Interest expense for the quarter consisted of interest expense related to financing agreements with NovaQuest Pharma Opportunities Fund IV L.P. and Hercules Capital, Inc., as well as the associated non-cash amortization of debt discount and issuance costs.

Net loss for the quarter ended June 30, 2018, was $62.1 million, compared to $23.3 million for the comparable period in 2017. On a per common share basis, net loss was $0.98 and $0.39 for the quarters ended June 30, 2018, and 2017, respectively. The increases in the net loss and net loss per common share for the quarter were driven primarily by the increase in costs associated with Myovant’s ongoing Phase 3 clinical trials, which were initiated in 2017, as well as increased personnel-related expenses.

Capital resources: Cash and committed funding totaled $235.6 million at June 30, 2018, consisting of $143.6 million in cash and $92.0 million in remaining financing commitments available from NovaQuest under the NovaQuest Securities Purchase Agreement and the NovaQuest Equity Purchase Agreement. In the second quarter of fiscal year 2018, Myovant further strengthened its balance sheet, raising net proceeds of approximately $70.2 million from the issuance and sale of 3,333,334 common shares in an underwritten public offering. An additional $40.6 million of capacity remains available under the "at-the-market" equity offering program that Myovant initiated in April 2018.

About Relugolix

Relugolix is an oral, once-daily, small molecule that acts as a gonadotropin-releasing hormone, or GnRH, receptor antagonist that has been evaluated in approximately 1,600 study participants in Phase 1, Phase 2 and Phase 3 clinical trials. In these trials, relugolix has been shown to be generally well tolerated and to suppress estrogen and progesterone levels in women and testosterone levels in men. Common side effects observed were consistent with suppression of these hormones. In the ongoing Phase 3 LIBERTY clinical trials in women with heavy menstrual bleeding associated with uterine fibroids and the ongoing Phase 3 SPIRIT clinical trials in women with endometriosis-associated pain, relugolix is undergoing evaluation alone as monotherapy and in combination with estradiol and norethindrone acetate, a progestin. Myovant is studying whether the combination decreases estradiol levels to the range required to treat signs and symptoms of endometriosis and uterine fibroids while minimizing the side effects such as bone mineral density loss and hot flashes, associated with low estrogen levels. The ongoing Phase 3 HERO study is evaluating relugolix alone in men with advanced prostate cancer.

On August 7, 2018 Vical Incorporated (Nasdaq:VICL) reported financial results for the three months ended June 30, 2018 (Press release, Vical, AUG 7, 2018, View Source [SID1234528485]). Net loss for the second quarter of 2018 was $4.9 million, or $0.22 per share, compared with a net loss of $3.3 million, or $0.30 per share, for the second quarter of 2017. Revenues for the second quarter of 2018 were $0.7 million, compared with revenues of $3.4 million for the second quarter of 2017, reflecting a decline in revenues from Astellas Pharma Inc. for services performed under ASP0113 collaborative agreements.

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Vical recently announced plans to explore a range of strategic options to enhance shareholder value. The company retained MTS Health Partners, L.P. to assist in the strategic review process. There is no set timetable for the review process and there can be no assurance that the process will result in a transaction.

Vical had cash and investments of $54.3 million at June 30, 2018. The Company’s cash burn for the second quarter of 2018 was $4.0 million, which was consistent with the Company’s full year 2018 guidance of between $20 million and $24 million. The Company anticipates ending 2018 with a minimum of $40 million, which, in the absence of a strategic transaction, Vical believes to be sufficient to fund operations through the announcement of top-line data from its Phase 2 clinical trial of VL-2397, expected in 2020.

Program updates include:

VL-2397 Antifungal Candidate

The multinational Phase 2 registration trial comparing VL‑2397 to standard first-line treatment for invasive aspergillosis in immunocompromised adults with acute leukemia or recipients of an allogeneic hematopoietic cell transplant is ongoing (ClinicalTrials.gov Identifier: NCT03327727). Vical expects to conduct the trial in approximately 40 major cancer and transplantation centers in North America, Europe and Asia. The FDA has advised that VL‑2397 would be eligible for a Limited Use Indication (LUI) approval for the treatment of invasive aspergillosis for patients with limited treatment options. The FDA has also granted Vical Qualified Infectious Disease Product (QIDP), Orphan Drug and Fast Track designations for VL‑2397 for the treatment of invasive aspergillosis. VL-2397 has a novel mechanism of antifungal action and could be the first therapeutic in a new class of antifungals.
VR-CHB01 Hepatitis B Virus (HBV) Therapeutic Candidate

The Company is pursuing preclinical development of a novel treatment for chronic HBV infection based on its DNA and lipid-delivery technologies. The initial aim of this program will be to demonstrate proof of concept for inhibiting HBV infection in an in vivo model. The Company expects to complete the initial stage of preclinical development in the fourth quarter of 2018.