10-Q – Quarterly report [Sections 13 or 15(d)]: v

Lixte Biotechnology has filed a 10-Q – Quarterly report [Sections 13 or 15(d)]: v with the U.S. Securities and Exchange Commission (Filing, 10-Q, Lixte Biotechnology, AUG 4, 2017, View Source [SID1234520042]).

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ArQule Reports Second Quarter 2017 Financial Results

On August 4, 2017 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the second quarter of 2017 (Filing, Q2, ArQule, 2017, AUG 4, 2017, View Source [SID1234520137]).

For the quarter ended June 30, 2017, the Company reported a net loss of $7,201,000 or $0.10 per share, compared with a net loss of $5,100,000 or $0.07 per share, for the second quarter of 2016. For the six-month period ended June 30, 2017, the Company reported a net loss of $14,777,000 or $0.21 per share, compared with a net loss of $10,081,000 or $0.15 per share, for the six-month period ended June 30, 2016.

At June 30, 2017, the Company had a total of approximately $31,007,000 in cash, equivalents and marketable securities.

Key Highlights

· Derazantinib (ARQ 087), a pan-FGFR inhibitor, has begun recruiting in a registrational phase 3 trial for FGFR2 fusion positive second-line intrahepatic cholangiocarcinoma (iCCA). Enrollment is planned to commence in the current quarter. In May, compelling data from the phase 1/2 trial in second-line iCCA was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting highlighting a disease control rate of 83% and an objective response rate of 21%.

· ARQ 531, an orally bioavailable, potent and reversible BTK inhibitor, has been dosed in a phase 1a/b trial. The trial is enrolling patients with B-cell malignancies, including B-cell lymphomas, chronic lymphocytic leukemia, and Waldenstrom’s macroglobulinemia, who are refractory to other therapeutic options, including ibrutinib. Up to 120 patients can be enrolled in the trial. The company also presented preclinical data for ARQ 531 in diffuse large B-cell lymphoma at the Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) which further strengthens the preclinical package for this molecule.

· ARQ 092, lead AKT inhibitor, has been dosed in a phase 1/2 company-sponsored trial in Overgrowth Diseases with genetic alterations of the PI3K/AKT1 pathway, including PROS (PIK3CA-related Overgrowth Spectrum) and Proteus syndrome. The trial is designed to enroll six patients in a dose escalation cohort as part of the phase 1 portion of the trial. An additional 10 patients will be enrolled in an expansion cohort as part of the phase 2 portion of the trial. The objective of this study is to determine a clinically meaningful endpoint to pursue in a registrational trial.

"We have made significant progress over the past few months by initiating phase 1 trials for ARQ 092 and ARQ 531 with the aim of achieving clinical proof of principle, and we are now poised to initiate a registrational trial with derazantinib," said Paolo Pucci, Chief Executive Officer of ArQule. "We achieved all of our targeted pipeline milestones for the first half of 2017, most notably moving ARQ 531, our BTK inhibitor, into the clinic. We believe ARQ 531 was the first reversible BTK inhibitor to be dosed in patients with B-cell malignancies. With four programs in the clinic, including derazantinib, ARQ 092, ARQ 751, and ARQ 531, we are poised to continue to achieve our goals for 2017."

"Our pipeline achieved two important milestones with the dosing of the first patient in two biomarker driven clinical trials targeting patients in areas of high unmet need," said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "In the first of these clinical trials, ARQ 531 aims to demonstrate its potential to address a large patient population with B-cell malignancies who become refractory to current therapies. This is a significant emerging clinical need, particularly in C481S-mutant patients. In the second trial, ARQ 092 is now being dosed in Overgrowth Diseases driven by the PI3K/AKT1 mutation targeting a completely unmet clinical need in a patient population comprised of multiple orphan diseases. Both programs have the potential to be transformational and represent well ArQule’s mission to bring life-changing therapies to address unmet medical needs."

Revenues and Expenses

Revenues for the quarter ended June 30, 2017, were zero compared with revenues of $1,072,000 for the quarter ended June 30, 2016. Revenues in the six-months ended June 30, 2017 were zero compared with revenues of $2,299,000 in the six-months ended June 30, 2016. Revenue in the three and six-month periods of 2016 is comprised of revenue from the Daiichi Sankyo tivantinib development agreement and the Kyowa Hakko Kirin exclusive license agreement. No further revenue is anticipated from these agreements.

Research and development expense in the second quarter of 2017 was $4,983,000, compared with $4,337,000 for the second quarter of 2016. Research and development expense increased $0.6 million in the second quarter of 2017 primarily due to higher outsourced preclinical, clinical and product development costs.

Research and development expense in the six-months ended June 30, 2017 was $10,177,000 compared with $8,535,000 in the six-months ended June 30, 2016. The $1.6 million increase in research and development expense in the six-months ended June 30, 2017 was primarily due to higher outsourced preclinical, clinical and product development costs.

General and administrative expense was $1,866,000 in the second quarter of 2017 compared with $1,887,000 in the second quarter 2016.

General and administrative expense was $3,940,000 in the six-months ended June 30, 2017 compared with $3,931,000 in the six-months ended June 30, 2016.

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Epizyme Reports Second Quarter 2017 Financial Results and Clinical and Business Progress

On August 4, 2017 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported operating results for the second quarter 2017 and highlighted recent business and clinical progress (Press release, Epizyme, AUG 4, 2017, View Source [SID1234520030]).

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"Our focus throughout 2017 has been on executing important clinical and regulatory milestones across our tazemetostat program to enable us to bring this first-in-class agent to patients as quickly as we can," said Robert Bazemore, president and chief executive officer of Epizyme. "In addition, we have continued to pursue avenues to accelerate patient enrollment in our studies, including the recently established collaboration with US Oncology Research to support recruitment for our NHL study. We are also pleased to have reached key milestones in our solid tumor program, including completing enrollment in both our Phase 2 mesothelioma study and the epithelioid sarcoma cohort in our Phase 2 INI1-negative solid tumor study, and establishing the recommended dose in our Phase 1 study in children. We believe we have a significant opportunity with tazemetostat in hematological malignancies and solid tumors, and look forward to further evaluating its monotherapy and combination potential and engaging with FDA for our NHL program in the second half of the year."

Tazemetostat Clinical Program Updates

In July 2017, Epizyme completed enrollment in the epithelioid sarcoma (ES) cohort of its ongoing, multi-arm Phase 2 study in adult patients with INI1-negative solid tumors. Following the announcement of positive interim data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and discussion with the U.S. Food and Drug Administration, the company has identified a path to submission for accelerated approval for the treatment of patients with ES, and is targeting the first New Drug Application for tazemetostat in this indication in 2018.
In July 2017, Epizyme announced that the National Cancer Institute (NCI) initiated its Pediatric MATCH study, which will evaluate tazemetostat in one of the study arms as a monotherapy for pediatric patients with certain genetically defined solid tumors or non-Hodgkin lymphoma. This study is part of the company’s Cooperative Research and Development Agreement (CRADA) executed between Epizyme and the NCI.
In July 2017, the company established the recommended dose for tazemetostat in its study of pediatric patients with solid tumors, and initiated the dose-expansion portion of the study.
In June 2017, the company completed enrollment in its ongoing Phase 2 study designed to evaluate tazemetostat as a treatment for adults with mesothelioma characterized by BAP1 loss-of-function. The company expects to report data from this study in 2018.
In June 2017, Epizyme announced that tazemetostat will be evaluated in combination with atezolizumab (TECENTRIQ) in a Phase 1b/2 clinical study of metastatic non-small cell lung cancer as part of MORPHEUS, Genentech’s open-label, multi-center, randomized umbrella study evaluating the efficacy and safety of multiple immunotherapy-based treatment combinations in solid tumors. This study is an expansion of the company’s clinical collaboration with Genentech, a member of the Roche Group, and is expected to begin enrolling patients by the end of the year.
In June 2017, positive interim efficacy data, grouped by EZH2 mutational status, were presented during a plenary session at the International Conference on Malignant Lymphoma (ICML) from the ongoing Phase 2 clinical trial of tazemetostat as a single-agent treatment for relapsed or refractory patients with follicular lymphoma (FL) or diffuse large B-cell lymphoma (DLBCL). Promising clinical activity and durability were observed across all study cohorts, with enhanced efficacy in the EZH2 mutation cohorts. Importantly, tazemetostat continues to demonstrate a favorable safety profile across the clinical development program.

Recent Business Highlights

In August 2017, Epizyme announced a collaboration with US Oncology Research to implement a separate screening protocol in 68 locations in the U.S. (including satellite locations affiliated with primary sites) to identify relapsed or refractory FL and DLBCL patients with tumors bearing EZH2 mutations who may be candidates for enrollment in Epizyme’s ongoing Phase 2 clinical trial. US Oncology Research will direct identified patients to the tazemetostat Phase 2 clinical trial for protocol screening and potential enrollment into the trial. Sites began screening patients in July 2017.
In June 2017, the U.S. FDA granted Orphan Drug designation to tazemetostat for the treatment of patients with soft tissue sarcoma (STS). Orphan Drug designation conveys eligibility for certain development incentives and market exclusivity for STS independent from Epizyme’s intellectual property protection.

Upcoming 2017 Milestones

Epizyme anticipates engaging with the U.S. FDA in the second half of 2017 to review data from its ongoing Phase 2 study in relapsed or refractory patients with NHL and discussing potential registration paths for tazemetostat.
The company plans to begin a clinical study evaluating tazemetostat as a combination agent for the treatment of patients with FL by the end of 2017.
Epizyme plans to announce the next development candidate from its novel, internally discovered pipeline of epigenetic programs by the end of 2017.
Second Quarter 2017 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $193.0 million as of June 30, 2017, as compared to $211.2 million as of March 31, 2017.
Revenue: $10.0 million was recognized in the second quarter of 2017, compared to $0.5 million for the second quarter of 2016. Revenue in the second quarter of 2017 represents a $10.0 million milestone payment from GlaxoSmithKline (GSK) following their initiation of GLP toxicology studies for a first-in-class methyltransferase inhibitor discovered by Epizyme and licensed to GSK.
R&D Expenses: Research and development (R&D) expenses were $27.3 million for the second quarter of 2017, compared to $21.5 million for the second quarter of 2016. The increase is primarily due to the expansion of the tazemetostat clinical development program, as well as increased research activities related to Epizyme’s next potential development candidate and new target families.
G&A Expenses: General and administrative (G&A) expenses were $11.2 million for the second quarter of 2017, compared to $7.4 million for the second quarter of 2016. The increase is primarily due to an increase in commercial-related activities and legal spending to support the company’s growing intellectual property portfolio.
Net Loss: Net loss was $28.0 million for each of the quarters ended June 30, 2017 and June 30, 2016.
2017 Financial Guidance
Epizyme believes, based on its current operating plan, that its cash, cash equivalents and marketable securities of $193.0 million as of June 30, 2017 will be sufficient to fund the Company’s planned operations into at least the third quarter of 2018.

About the Tazemetostat Clinical Trial Program
Tazemetostat, a first-in-class EZH2 inhibitor, is currently being studied as a monotherapy in ongoing Phase 2 programs in both follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) forms of non-Hodgkin lymphoma (NHL); certain molecularly defined solid tumors, including epithelioid sarcoma and other INI1-negative tumors; and mesothelioma and combination studies in DLBCL. Tazemetostat has been granted Fast Track designation by the U.S. Food and Drug Administration for FL regardless of EZH2 mutation status and for DLBCL with EZH2-activating mutations, as well as Orphan Drug designation for soft tissue sarcoma and malignant rhabdoid tumors.

10-Q – Quarterly report [Sections 13 or 15(d)]

Acceleron Pharma has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Acceleron Pharma, AUG 3, 2017, View Source [SID1234520054]).

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Aclaris Therapeutics Submits Marketing Authorization Application in Europe for A-101 40% Topical Solution as a Novel Treatment for Seborrheic Keratosis

On August 3, 2017 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company focused on identifying, developing and commercializing innovative and differentiated therapies to address significant unmet needs in medical and aesthetic dermatology, reported it has submitted a Marketing Authorization Application (MAA) with the Medicines Product Agency (MPA) in Sweden for its product candidate A-101 40% Topical Solution for the treatment of seborrheic keratosis (Press release, Aclaris Therapeutics, AUG 3, 2017, View Source [SID1234525294]). The MPA will act as the reference member state in this decentralized procedure for review of the MAA for potential marketing approval throughout Europe. If approved, A-101 40% Topical Solution would be available to be commercialized in 16 countries in the European Union.

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Seborrheic keratosis (SK) lesions are common, non-cancerous skin lesions which can have a negative physical and emotional impact on patients because they may be perceived as cosmetically unattractive and associated with aging. Existing treatments are often painful, invasive and can have undesirable outcomes such as pigmentary changes or scarring. Fewer than 10% of people [in the United States] with SK lesions currently receive treatment.

Positive results from two pivotal Phase 3 trials – SEBK-301 and SEBK-302 – were reported in late 2016 and provide the clinical basis for this MAA submission. In these trials, A-101 40% Topical Solution (A-101 40%) met all primary and secondary endpoints, achieving clinically and statistically significant clearance of SK lesions. The two trials, which were identical in design and together enrolled 937 patients, evaluated the safety and efficacy of A-101 compared with vehicle (placebo) in patients with four target SK lesions on the face, trunk, and extremities.

"This MAA submission represents a major step toward our goal of delivering a novel, topical treatment to address a significant unmet need in the dermatology market," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris. "If approved, we believe A-101 will have broad appeal across aesthetic and medical dermatology patients – both men and women."