On August17, 2016 Cerulean Pharma Inc. (NASDAQ:CERU), a clinical-stage company developing nanoparticle-drug conjugates (NDCs), reported top-line results from the Company’s Phase 2, randomized, multi-center clinical trial of its lead candidate, CRLX101, in combination with Avastin (bevacizumab) in the treatment of patients with advanced renal cell carcinoma (RCC) (Press release, Cerulean Pharma, AUG 17, 2016, View Source [SID1234514632]).

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The trial was conducted at 43 sites in the US and South Korea, and enrolled 115 patients with RCC who progressed through two or three prior lines of therapy. Patients were randomized to receive CRLX101 in combination with Avastin or investigator’s choice standard of care (SOC) therapy. SOC agents included axitinib, bevacizumab, everolimus, pazopanib, sorafenib, sunitinib, and temsirolimus. The primary endpoint was progression free survival (PFS) in the clear cell population (n=102) assessed by independent radiological review. Secondary endpoints included overall response rate, duration of response and overall survival.

The study demonstrated no statistically significant difference in median PFS and objective response rate for the CRLX101 combination compared to SOC. Median PFS was 3.7 months for the CRLX101 combination compared with a median PFS of 3.9 months for SOC (hazard ratio: 1.25, p=0.822). The 95% confidence interval for PFS for the CRLX101 combination was 2.0 months to 4.3 months and for SOC was 2.2 months to 5.4 months. Objective response rate by independent radiological review for patients who received the CRLX101 combination was 5% (2/42) compared to 14% (6/43) for SOC (p=0.836). The CRLX101 and Avastin combination appeared to be safe and well-tolerated and the safety and tolerability profile of the combination was consistent with that observed in previous studies. The full data set from the trial are expected to be submitted for presentation at an upcoming medical conference.

"We are disappointed with this outcome and will undertake a thorough analysis of the data to understand why CRLX101 plus Avastin underperformed compared to the results we saw in an earlier investigator-sponsored trial," said Christopher D. T. Guiffre, President and Chief Executive Officer of Cerulean. "This outcome did not support our hypothesis that targeting hypoxia inducible factor (HIF) in combination with VEGF inhibitor in RCC, a HIF-overexpressing tumor type, would be beneficial, so we will not pursue HIF as a target going forward. We will continue to focus on the potent topoisomerase 1 inhibition of CRLX101’s payload, camptothecin, in topoisomerase 1-sensitive tumors. Our combinations with weekly paclitaxel and LYNPARZA (olaparib) are examples of ongoing trials that leverage CRLX101’s topoisomerase 1 inhibition in combination with chemotherapies and DNA damage repair agents."

Conference Call Details

Cerulean will host a conference call today at 4:30 pm Eastern Daylight Time to discuss the results and provide an update on the CRLX101 development program. The call can be accessed by dialing (844) 831-3031 or (443) 637-1284 prior to the start of the call and referencing conference ID: 67807137. The conference call will also be webcast live over the Internet and can be accessed on the "Investors" section of the Cerulean website, www.ceruleanrx.com. The webcast will be available on Cerulean’s website for two weeks.

About CRLX101

CRLX101 is a nanoparticle-drug conjugate (NDC) designed to concentrate in tumors and slowly release its anti-cancer payload, camptothecin, inside tumor cells. CRLX101 inhibits topoisomerase 1 (topo 1), which is involved in cellular replication. CRLX101 has shown activity in multiple tumor types, both as monotherapy and in combination with other cancer treatments. CRLX101 is in Phase 2 clinical development and has been dosed in more than 400 patients. The U.S. FDA has granted CRLX101 Orphan Drug designation for the treatment of ovarian cancer, Fast Track designation in combination with paclitaxel for platinum-resistant ovarian carcinoma, fallopian tube or primary peritoneal cancer, and Fast Track designation in combination with Avastin in metastatic renal cell carcinoma.

On August 16, 2016 Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, reported financial results for the three and six months ended June 30, 2016 (Press release, Delcath Systems, AUG 16, 2016, View Source;p=RssLanding&cat=news&id=2195645 [SID:1234514611]).

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Highlights for the second quarter of 2016 and recent weeks include:

Issuance of $35 million in senior convertible notes to support Melphalan/HDS Phase 3 Focus Trial enrollment and CHEMOSAT European commercialization through the end of 2017;
Presentation of data from a large single hospital experience conducted at Southampton University Hospital in the United Kingdom at the 6th European Post-Chicago Melanoma/Skin Cancer Meeting, which demonstrated overall survival benefit using Delcath’s CHEMOSAT to treat metastatic uveal melanoma;
Acceptance of abstracts from two studies conducted in Germany using Delcath’s CHEMOSAT to treat patients with liver metastases for presentation as posters at the Cardiovascular and Interventional Radiology Society of Europe (CIRSE) Annual Meeting in September 2016;
Acceptance of a review of clinical research treatment outcomes using Melphalan Hydrochloride for Injection with the Delcath Hepatic Delivery System (Melphalan/HDS) in patients with hepatic metastases for publication in the prestigious journal, Cancer Control;
Promotion of John Purpura to Executive Vice President, Global Head of Operations from Executive Vice President-Regulatory Affairs and Quality Assurance; and
Launch of CHEMOSAT at the HM Sanchinarro University Hospital in Madrid.
"Throughout the second quarter we made considerable clinical and commercial progress advancing CHEMOSAT as an innovative new treatment option for primary and metastatic liver cancers," noted Jennifer K. Simpson, Ph.D., MSN, CRNP, President and Chief Executive Officer of Delcath. "Importantly, we secured $35 million in committed financing that provides us with the resources to advance our clinical development plan through to key inflection points while also supporting our commercialization programs in Europe.

"We were particularly pleased to have real-world data from Southampton University Hospital’s experience presented at the Melanoma/Skin Cancer meeting as the progression free and overall survival benefits observed in this study are dramatic, especially given the limited treatment options for patients suffering with these life-threatening cancers. These supportive data provide us with considerable confidence that similar results may be formally validated by our FOCUS Phase 3 Trial in hepatic dominant ocular melanoma that is currently underway in the U.S. and Europe. We look forward to additional presentations and publication of data in support of CHEMOSAT in the treatment of cancers of the liver during the second of half of the year.

"The addition of CHEMOSAT to centers in Spain and Turkey highlight our continued progress commercializing the system in Europe. We continue with negotiations to determine reimbursement levels for CHEMOSAT under the ZE national system in Germany and expect coverage levels to be defined later this year. We believe that favorable reimbursement levels will enhance growth in procedure volumes in Germany and provide important validation for reimbursement appeals in other markets in Europe.

"The advances we made during the first half of 2016 have positioned us to achieve important clinical inflection points in our FOCUS trial and our global Phase 2 program in HCC and ICC, as we work to expand global access to our CHEMOSAT for the benefit of patients suffering with primary and metastatic liver cancers," concluded Dr. Simpson.

Second Quarter Financial Results

Total revenues for the second quarter of 2016 and 2015 were $0.5 million. Selling, general and administrative expenses for the second quarter of 2016 were $2.3 million, compared with $2.5 million for the same period in 2016, primarily attributable to a reduction in depreciation and corporate expenses. Research and development expenses increased to $1.9 million for the 2016 second quarter from $1.5 million for the same period in 2015, primarily due to increased investment in clinical development initiatives, specifically the global Phase 3 FOCUS clinical trial.

Total operating expenses for the second quarter of 2016 increased to $4.2 million from $4.0 million for the same period in 2015. This reflects an increase in clinical development initiatives, partially offset by reductions in depreciation and corporate expenses.

The Company recorded a net loss for the three months ended June 30, 2016 of $6.7 million, an increase of $3.0 million from a net loss of $3.7 million for the same period in 2015. This was primarily driven by amortization of debt discounts related to the convertible note issued in June 2016 and a change in the fair value of the warrant liability, a non-cash item.

First Half Financial Results

Total revenues for the first half of 2016 and 2015 were $0.9 million. Selling, general and administrative expenses for the first six months of 2016 were $4.7 million, an improvement of $0.8 million or 15% from $5.5 million reported for the same period in 2015, primarily attributable to a reduction in facility expenses related to the lease restructurings. Research and development expenses during the first half of 2016 increased to $3.3 million compared with $2.4 million for the same period in 2015, primarily due to increased investment in clinical development initiatives.

Total operating expenses for the first half of 2016 were $8.0 million compared with $8.0 million for the same period in 2015.

The Company recorded a net loss for the six months ended June 30, 2016 of $8.5 million, an increase of $1.3 million from a net loss of $7.2 million for the six months ended June 30, 2015. This was primarily driven by amortization of debt discounts related to the convertible note issued in June 2016 and a change in the fair value of the warrant liability, a non-cash item.

Balance Sheet Highlights

As of June 30, 2016, Delcath had cash and cash equivalents of $7.5 million, compared with $12.6 million as of December 31, 2015. During the first half of 2016, the Company used $7.0 million in cash to fund its operating activities. In June 2016, Delcath issued $35.0 million of senior convertible notes and related common stock purchase warrants. As a result, Delcath believes it has sufficient capital to fund its operating activities through the end of 2017.

On August 16, 2016 Medtronic plc (NYSE: MDT) reported that it will report financial results for the first quarter of fiscal year 2017, on Thursday, August 25, 2016 (Press release, Medtronic, AUG 16, 2016, View Source;p=RssLanding&cat=news&id=2195681 [SID:1234514612]). A news release will be issued at approximately 5:45 a.m. Central Daylight Time (CDT) and will be available at View Source The news release will include summary financial information for the company’s first quarter of fiscal year 2017, which ended on Friday, July 29, 2016.
Medtronic will host a webcast at 7:00 a.m. CDT to discuss financial results for its first quarter of fiscal year 2017. The webcast can be accessed at View Source on August 25, 2016.

Within 24 hours of the webcast, a replay and transcript of the prepared remarks will be available by clicking on the Investor Events link at View Source.
Looking ahead, Medtronic plans to report its 2017 fiscal year second, third, and fourth quarter financial results on Tuesday, November 22, 2016, Tuesday, February 21, 2017, and Thursday, May 25, 2017, respectively. Additional details will be provided closer to the specific quarterly earnings release date.

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On August 16, 2016, OncoGenex Pharmaceuticals, Inc. (Company) announced results from the final analysis of AFFINITY, the Phase 3 trial of custirsen in men with metastatic castrate-resistant prostate cancer (CRPC) whose disease has progressed after treatment with docetaxel (Filing, 8-K, OncoGenex Pharmaceuticals, AUG 16, 2016, View Source [SID1234515887]). The trial did not meet the primary endpoint of demonstrating a statistically significant improvement in overall survival for patients treated with custirsen in combination with cabazitaxel/prednisone compared to cabazitaxel/prednisone alone. The median overall survival for the custirsen arm was 14.2 months versus 13.4 months for the control arm with a hazard ratio of 0.946. Safety data were consistent with those observed in previous trials of custirsen in CRPC.

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As a result of these data and previous custirsen findings, the Company plans to initiate discussions with the U.S. Food and Drug Administration (FDA) to evaluate options related to an early analysis of the Phase 3 ENSPIRIT trial investigating custirsen in combination with docetaxel as second-line chemotherapy in patients with non-small cell lung cancer. In the Company’s proposal to the FDA, the trial’s hypothesized hazard ratio of 0.75 and the p-value for the final survival analysis will remain the same, with a minimal reduction in power and a small change in the critical hazard ratio from 0.84 to 0.83. The ENSPIRIT trial is over 90% enrolled and more than 80% of the events have occurred. The Company believes this is sufficient to assess the potential effect of custirsen in non-small cell lung cancer.

The Company has also engaged MTS Health Partners, LP as its advisor to assist with the exploration of strategic alternatives.

A copy of the Company’s press release is filed as Exhibit 99.1 to this Current Report on Form 8-K.

On August 16, 2016 Accurexa Inc. (the "Company" or "Accurexa") (ACXA), a biotechnology company focused on the development of novel neurological therapies to be directly delivered into the brain reported that it received a written response from the FDA (U.S. Food and Drug Administration) regarding a pre-IND (Investigational New Drug) meeting request for its ACX-31 program for the local delivery of temozolomide as adjunctive therapy to BCNU, both chemotherapeutics, in the treatment of brain tumors in conjunction with surgery and radiation (Press release, Accurexa, AUG 16, 2016, View Source [SID1234516522]). The FDA confirmed the acceptability of a 505(b)(2) application pathway with one first-in-human Phase 2 clinical trial followed by one Phase 3 clinical trial.

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Key Points of Written Response:

Acceptability of a 505(b)(2) application pathway confirmed
Pharmacology studies appear adequate
Non-clinical studies appear adequate with the exception of one GLP-compliant toxicology study in a single animal species using the route and schedule of administration proposed for the planned Phase 2 trial to be required
Phase 2 clinical trial strongly recommended to be conducted in patients with relapsed/recurrent glioblastoma
Proposed Phase 2 trial primary endpoints appear adequate
If an adequately designed Phase 2 trial demonstrates tolerability and suggests activity, FDA would encourage Accurexa to request an End-of-Phase 2 meeting to discuss the design of an adequate and well-controlled, randomized Phase 3 trial
"We are very pleased with the outcome of the FDA meeting. The FDA’s acceptance of a 505(b)(2) application pathway reduces the cost and time for the development of our ACX-31 Brain Cancer program than it would otherwise require. The FDA provided valuable guidance and we continue moving our ACX-31 program forward towards clinical development," said George Yu, MD, Accurexa’s President & CEO.

Under a 505(b)(2) application pathway the Company can rely on studies of previously approved drugs that were not conducted by the Company. The benefits of a 505(b)(2) application include lower risk due previous drug approvals, lower cost and accelerated development due to fewer studies, than a typical new drug application.