vTv Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, vTv Therapeutics, 2017, AUG 2, 2016, View Source [SID1234521331]).

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On August 02, 2016 Supernus Pharmaceuticals, Inc. (NASDAQ:SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for second quarter 2016 and associated company developments (Press release, Supernus, AUG 2, 2016, View Source [SID:1234514195]).

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Commercial Update

Second quarter 2016 product prescriptions for Trokendi XR and Oxtellar XR, as reported by IMS, totaled 123,758, a 38.9% increase over the second quarter of 2015.

Prescriptions
Q2 2016 Q1 2015 Change %

Trokendi XR 93,094 65,552 42.0 %
Oxtellar XR 30,664 23,534 30.3 %

Total 123,758 89,086 38.9 %

Source: IMS

Total revenue for the second quarter of 2016 was $50.4 million, a 43.8% increase over $35.1 million in the same period last year. Total revenue for both periods consisted almost exclusively of net product sales.

Net Product Sales ($mil.)
Q2 2016 Q2 2015 Change %

Trokendi XR $ 37.6 $ 26.3 43.3 %
Oxtellar XR $ 12.7 $ 8.0 58.7 %

Total $ 50.3 $ 34.3 46.9 %

"We are pleased with the growth in prescriptions and net product sales in the second quarter and through the first half of 2016. For the first six months of 2016, net product sales increased approximately 50%, as compared to the same period last year," said Jack Khattar, President and CEO of Supernus Pharmaceuticals. "The solid growth in prescriptions behind our products three years post launch reinforces our belief that the combined annual peak sales for Oxtellar XR and Trokendi XR can exceed $500 million."

In June 2016 the Company submitted the revised label for Trokendi XR requesting approval to expand the label to include treatment of migraine in adults. As previously announced, this resubmission was requested by the Food and Drug Administration (FDA) to review the proposed label in a different format. The FDA has set a target date in the third quarter of 2016 to complete its review. We continue to prepare and will be ready to launch the migraine indication soon after receiving full FDA approval.

Progress of Product Pipeline

Enrollment continues for both Phase III trials for SPN-810, which is currently in development for Impulsive Aggression in patients aged 6 to 12 years who have ADHD. The pace of enrollment is slower than anticipated due to challenges such as those experienced by caregivers in recording patient information on the new electronic diary, and lack of compliance during the screening period regarding ‘washing out’ of current medications. As a consequence, we have instituted a number of measures to improve patient enrollment and retention. These include lengthening the screening period to provide increased education for site coordinators and caregivers on the electronic diary and to make it easier for caregivers and patients to comply with the trial protocol. Although the pace of recruitment has picked up recently for both Phase III trials, it is likely that enrollment will continue into 2017. The Company continues to expect to launch SPN-810 in 2019. During the third quarter of 2016, patients began enrolling into the open-label extension portion of the Phase III study.

Regarding SPN-812, currently in development for patients aged 6 to 12 years with ADHD, the Phase IIb trial is now fully recruited. The final patient visit was completed during the third quarter of 2016. Eligible patients are now entering the open label extension portion of the study. The Company continues to expect data from the SPN-812 Phase IIb trial to be available by early 2017.

"With the SPN-812 Phase IIb trial fully enrolled, we have reached another important clinical milestone as we continue to advance SPN-812 into late-stage development," said Jack Khattar. "Regarding SPN-810, we are encouraged by the recent progress in improving recruitment and retention, and we remain focused on the successful execution of the trials."

Mr. Khattar added, "We believe the 84% rate of enrollment into the open-label extension of the Phase IIb study for SPN-812 reflects a high level of satisfaction from physicians and patients."

Operating Expenses

Research and development expenses in the second quarter of 2016 were $11.1 million, as compared to $6.9 million in the same quarter last year. This increase is primarily due to the ongoing Phase III testing of SPN-810 and Phase IIb testing of SPN-812, as well as the open-label extension studies associated with both SPN-810 and SPN-812.

Selling, general and administrative expenses in the second quarter of 2016 were $26.1 million, as compared to $23.3 million in the same quarter last year. The increase is primarily due to the efforts in preparing for the launch of the migraine indication for Trokendi XR.

Operating Income and Earnings Per Share

Operating income in the second quarter of 2016 was $10.4 million, as compared to $3.1 million in the same period last year. This improvement in operating income is primarily due to increased net product sales.

Diluted earnings per share were $0.18 in the second quarter of 2016, as compared to $0.03 in the same period last year.

Weighted-average diluted common shares outstanding were approximately 51.7 million in the second quarter of 2016, as compared to approximately 52.3 million in the same period last year.

Capital Resources

As of June 30, 2016, the Company had $128.0 million in cash, cash equivalents, marketable securities, and long term marketable securities, as compared to $117.2 million at December 31, 2015. As of June 30, 2016, approximately $6.6 million of the Company’s six year, $90 million notes, bearing interest at 7.5% per annum, remain outstanding.

Financial Guidance

For full year 2016, the Company is reiterating guidance for net product sales and adjusting guidance for R&D expenses and operating income as set forth below:

Net product sales will remain in the range of $200 million to $210 million.
R&D expenses in the range of $50 million to $55 million, compared to the previously expected range of $55 million to $65 million.
Operating income in the range of $32 million to $37 million, compared to the previously expected range of $28 million to $35 million.

On August 1, 2016 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, www.provectusbio.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus" or "The Company"), reported that a poster presentation titled "Intralesional rose bengal for stage III and IV melanoma" is to be presented at the European Society for Medical Oncology 2016 Congress on October 9, 2016, in Hall E of the Bella Center in Copenhagen, Denmark (Press release, Provectus Pharmaceuticals, AUG 1, 2016, View Source [SID:1234514149]).

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ESMO’s Scientific Committee accepted the abstract for this poster presentation and further details can be found at View Source – 2z95v (Abstract 1158TiP).

In addition, the abstract will be published in the ESMO (Free ESMO Whitepaper) 2016 Congress Abstract Book, a supplement to the official ESMO (Free ESMO Whitepaper) journal, Annals of Oncology. ESMO (Free ESMO Whitepaper) has not yet announced the final publication number. The ESMO (Free ESMO Whitepaper) 2016 Congress will be held in Copenhagen, Denmark, from October 7-11, 2016.

Provectus believes the poster itself will be available online following the final session of the Congress.

About ESMO (Free ESMO Whitepaper)

ESMO is the leading European professional organization for medical oncology. With more than 13,000 members representing oncology professionals from over 130 countries, ESMO (Free ESMO Whitepaper) is the society of reference for oncology education and information. For more information, visit: View Source

On August 1, 2016 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that the results of an additional analysis of a Phase III clinical study (Study 309) of its in-house discovered and developed anticancer agent Halaven (eribulin mesylate, "eribulin") in patients with locally advanced, recurrent or metastatic soft tissue sarcoma (liposarcoma or leiomyosarcoma) have been presented at a plenary session of the 14th Japanese Society of Medical Oncology (JSMO) Annual Meeting held in Kobe from July 28 to 30, 2016 (Press release, Eisai, AUG 1, 2016, View Source [SID:1234514150]). (Presenter: Dr. Patrick Schöffski, University Hospitals Leuven, Belgium)

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Study 309 was a Phase III clinical study which examined the efficacy and safety of eribulin versus dacarbazine in 452 patients aged 18 years and older with locally advanced, recurrent or metastatic soft tissue sarcoma (liposarcoma or leiomyosarcoma) who had disease progression following standard therapies (including an anthracycline and at least one other additional regimen).1

According to the results of preplanned subgroup analyses of the duration of response in patients with liposarcoma and leiomyosarcoma, median duration of response for the eribulin treatment was 12.5 months [95% CI: 1.7-Not Estimable] and median duration of response for dacarbazine treatment was 4.2 months [95% CI: 2.2-14.7].
Furthermore, as part of an additional analysis, patients with liposarcoma in Study 309 were categorized by three subtypes, dedifferentiated liposarcoma, myxoid/round liposarcoma and pleomorphic liposarcoma, and additional analyses were carried out on each subtype. According to the results of these analyses, eribulin demonstrated a trend for extension in overall survival over dacarbazine in each subtype.
In this study, the most common treatment-emergent adverse events (incidence greater than or equal to 25%) in patients treated with eribulin were fatigue, neutropenia, nausea, alopecia, constipation, peripheral neuropathy, abdominal pain, and pyrexia, which is consistent with the known side-effect profile of eribulin.

Eribulin is a halichondrin class microtubule dynamics inhibitor with a novel mechanism of action. Recent non-clinical studies showed that eribulin is associated with increased vascular perfusion and permeability in tumor cores.2 Eribulin promotes the epithelial state and decreases the capacity of breast cancer cells to migrate.3 It was first approved for use in the treatment of metastatic breast cancer in the United States in November 2010, and is currently approved for use in the treatment of breast cancer in approximately 60 countries including Japan and countries in Europe, the Americas and Asia. In addition, eribulin was first approved for use in the treatment of soft tissue sarcoma in the United States in January 2016, and is approved in countries including Japan and in Europe.

Eisai positions oncology as a key therapeutic area, and is aiming to discovery revolutionary new medicines with the potential to cure cancer. Eisai remains committed to providing further clinical evidence for eribulin aimed at maximizing value of the drug as it seeks to contribute further to addressing the diverse needs of, and increasing the benefits provided to, patients with cancer, their families, and healthcare providers.

1. About Halaven (eribulin mesylate)
Halaven is a halichondrin class microtubule dynamics inhibitor with a novel mechanism of action. Structurally Halaven is a simplified and synthetically produced version of halichondrin B, a natural product isolated from the marine sponge Halichondria okadai. Halaven is believed to work by inhibiting the growth phase of microtubule dynamics which prevents cell division. In addition, recent non-clinical studies showed that Halaven is associated with increased vascular perfusion and permeability in tumor cores.2 Halaven promotes the epithelial state and decreases the capacity of breast cancer cells to migrate.3
Halaven was first approved as a treatment in the United States in November 2010 for patients with metastatic breast cancer. Halaven is currently approved for use in the treatment of breast cancer in over 60 countries worldwide, including Japan and countries in Europe, the Americas and Asia. Furthermore, Halaven was first approved as a treatment for soft tissue sarcoma in the United States in January 2016, and is approved in countries including Japan and in Europe. Applications seeking approval for use in the treatment of soft tissue sarcoma are currently under review throughout the world including Switzerland, Australia, Brazil, and countries in Asia. Furthermore, Halaven has been designated as an orphan drug for soft tissue sarcoma in the United States and Japan.

Specifically, Halaven is approved for the following indications.
In the United States for the treatment of patients with:
Metastatic breast cancer who have previously received at least two chemotherapeutic regimens for the treatment of metastatic disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting.
Unresectable or metastatic liposarcoma who have received a prior anthracycline-containing regimen.
In Japan for the treatment of patients with:
Inoperable or recurrent breast cancer
Soft tissue sarcoma
In Europe for the treatment of adult patients with:
Locally advanced or metastatic breast cancer who have progressed after at least one chemotherapeutic regimen for advanced disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting, unless patients were not suitable for these treatments.
Unresectable liposarcomas who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic disease.

2. About Soft Tissue Sarcoma
Soft tissue sarcoma is a collective term for a diverse group of malignant tumors that occur throughout the soft tissue (fat, muscle, nerves, fibrous tissues and blood vessels) in the body. As the structures where the tumors originate are diverse, there are various types of soft tissue sarcoma, and the most common types include leiomyosarcoma, liposarcoma and malignant fibrous histiocytoma. Approximately 12,000 patients in the United States and 29,000 patients in Europe are diagnosed with soft tissue sarcoma each year. According to a patient survey conducted by Japan’s Ministry of Health, Labour and Welfare, there are approximately 4,000 patients with soft tissue sarcoma in Japan. While treatment of soft tissue sarcoma is focused on curative surgery, if the degree of malignancy is high, treatment then becomes a combination of chemotherapy and radiation therapy. As outcomes are poor for patients with advanced disease, it remains a disease with significant unmet medical need.

3. About Study 3091
Conducted primarily in Europe and the United States, Study 309 was a multicenter, open-label, randomized Phase III study comparing the efficacy and safety of Halaven versus dacarbazine in 452 patients (aged 18 or over) with locally advanced, recurrent or metastatic soft tissue sarcoma (liposarcoma or leiomyosarcoma) who had disease progression following standard therapies which must have included an anthracycline and at least one other additional regimen. Patients received either Halaven (1.4 mg/m2 administered intravenously on Day 1 and Day 8) or dacarbazine (850–1200 mg/m2 administered intravenously on Day 1) every 21 days until disease progression.
From the results for the study, Halaven demonstrated a statistically significant extension in the study’s primary endpoint of overall survival (OS) over the comparator treatment dacarbazine (Halaven median OS: 13.5 months vs dacarbazine median OS: 11.5 months; Hazard Ratio (HR) 0.77 [95% CI=0.62-0.95], p=0.0169). Furthermore, in the study’s secondary endpoint of progression-free rate at 12 weeks (PFR12wks), while there was a numerical difference in PFR12wks between the Halaven and dacarbazine arms (33% vs 29%), this was not statistically significant. Median progression-free survival was 2.6 months in both arms.
The most common adverse reactions (incidence greater than or equal to 25%) in patients treated with Halaven were fatigue, neutropenia, nausea, alopecia, constipation, peripheral neuropathy, abdominal pain, and pyrexia, which was consistent with the known side-effect profile of Halaven.

In Study 309 patients with liposarcoma were categorized into three subtypes, dedifferentiated liposarcoma, myxoid/round liposarcoma and pleomorphic liposarcoma, and additional analyses were carried out on each subtype. The respective results of these additional analyses are as follows:
Dedifferentiated liposarcoma: eribulin median OS: 18.0 months vs dacarbazine median OS: 8.1 months (Hazard Ratio [HR]: 0.43 [95% CI=0.23-0.79])
Myxoid/round liposarcoma: eribulin median OS: 13.5 months vs dacarbazine median OS: 9.6 months (HR: 0.79 [95% CI=0.42-1.49])
Pleomorphic liposarcoma: eribulin median OS: 22.2 months vs dacarbazine median OS: 6.7 months (HR: 0.18 [95% CI=0.04-0.85])

On August 01, 2016 Cempra, Inc. (Nasdaq:CEMP), a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases, reported financial results for the quarter ended June 30, 2016 and provided an update on recent corporate developments (Press release, Cempra, AUG 1, 2016, View Source [SID:1234514158]). The company will host a webcast and conference call today at 4:30 p.m. EDT.

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Second Quarter 2016 and Recent Corporate Highlights

On July 5, Cempra announced that the U.S. Food and Drug Administration (FDA) had accepted for filing Cempra’s two New Drug Applications (NDAs) for intravenous and oral capsule formulations of Solithera (solithromycin) in community-acquired bacterial pneumonia (CABP). Having been granted qualified infectious diseases product (QIDP) designation in 2013, solithromycin’s NDAs have qualified for an eight month priority review. The PDUFA dates are December 27, 2016 for oral and December 28, 2016 for intravenous. Subject to approval by the FDA, Cempra plans to launch Solithera in the U.S. in the first quarter of 2017.

In late June, Cempra completed the submission of its Marketing Authorization Application (MAA) for solithromycin to the European Medicines Agency (EMA) for the treatment of community-acquired bacterial pneumonia. The MAA is for both intravenous and oral capsule formulations.

In May, Toyama Chemical, a subsidiary of FUJIFILM Holdings Corporation, announced successful results for its Phase 2 community-acquired bacterial pneumonia clinical trial of solithromycin. In this study, 154 patients with CABP were randomized in a 2:1 ratio to receive five days of oral treatment with solithromycin or levofloxacin. All efficacy outcome measures favored solithromycin. Overall safety and tolerability was similar in both treatment groups, including hepatic events such as increases in alanine aminotransferase (ALT). Cempra has licensed solithromycin to Toyama Chemical for certain exclusive rights in Japan.
"Cempra continues to advance its programs successfully and I am excited by the progress we are making with both our clinical development programs and our commercial initiatives as we prepare for the launch of Solithera, subject to approval, early next year," said Prabhavathi Fernandes, Ph.D., president and chief executive officer of Cempra. "We remain confident in the data underpinning our NDA and MAA submissions and look forward to working with the regulators to bring the product to the patients who need treatment. In addition, we believe we have the people, strategy and financing in place to see us through our key milestones near-term including the initial commercialization of Solithera in the U.S."

Upcoming Clinical Development Milestones

Solithromycin

Solithromycin pediatric
Patient enrollment for Phase 1b trial continues.
Phase 2/3 pivotal trial with solithromycin for bacterial infections in pediatric patients has been initiated.
Phase 3 trial for solithromycin in urogenital gonorrhea is ongoing.
Phase 2 trial in chronic obstructive pulmonary disease (COPD) is ongoing.
Phase 2 trial in nonalcoholic steatohepatitis (NASH) is ongoing, interim results expected in fourth quarter 2016.
Review of EMA submission for solithromycin in the treatment of CABP is continuing.
Review of NDAs by the FDA is ongoing with 2016 PDUFA dates of December 27 for the oral formulation and December 28 for intravenous.
Taksta

Phase 3 trial in ABSSSI is ongoing and completion of patient enrollment is expected by the end of 2016.
An exploratory trial for Taksta in patients with refractory bone or joint infections is ongoing.
Financial Results for the Three Months Ended June 30, 2016

For the quarter ended June 30, 2016, Cempra reported a net loss of $24.8 million, or $0.51 per share, compared to a net loss of $25.0 million, or $0.57 per share for the second quarter in 2015. Research and development expense in the second quarter of 2016 was $16.0 million, a decrease of 32% compared to $23.7 million in the second quarter of 2015. The lower R&D expense was primarily due to the timing of payments for the order of active pharmaceutical ingredient (API) necessary to support the launch of solithromycin as the company begins its commercial readiness activities and a decrease in clinical expenses as the Phase 3 Oral and Phase 3 IV-to-Oral studies are complete. General and administrative expense was $12.0 million compared to $5.7 million in the second quarter of 2015, driven primarily by commercial readiness activities and increased headcount as the company continues to plan for commercialization of Solithera.

As of June 30, 2016, Cempra had cash and equivalents of $250.7 million and 50.7 million shares outstanding. During the second quarter Cempra implemented an at the market (ATM) financing program through which it sold 2.5 million shares resulting in net proceeds of $45.8 million which is included in cash and cash equivalents at the end of the second quarter. So far in the third quarter, the company has continued to utilize its ATM facility with the sale of approximately 1.6 million shares resulting in proceeds of approximately $29.2 million. In total, the company’s utilization of its ATM has resulted in the sale of approximately 4.1 million shares and net proceeds of approximately $75.0 million.

Updated Financial Guidance

The company’s current cash and equivalents, including the proceeds of the sale of common stock through the company’s ATM through the end of July, are expected to be sufficient to fund ongoing operations through 2017, assuming continued timely receipts under the BARDA contract and receipt of expected milestone payments from Toyama. This projection does not include any funds from additional financings or partnerships.