On August 16, 2016 Tioma Therapeutics, Inc., a venture-stage biopharmaceutical company developing anti-CD47 antibodies for the treatment of solid and hematologic cancers, reported it has raised $86 million in Series A venture financing (Press release, Tioma Therapeutics, AUG 16, 2016, View Source [SID:1234514627]). Proceeds from this financing will be used to further develop Tioma’s antibody portfolio, including its lead drug candidate – an anti-CD47 immune checkpoint inhibitor – through advanced proof-of-concept in human clinical trials.

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The Series A financing was co-led by RiverVest Venture Partners (including 3×5 RiverVest Fund), Novo Ventures, Roche Venture Fund and S.R. One, Limited (the corporate venture capital arm of GlaxoSmithKline). Pursuant to this financing, John McKearn, PhD, of RiverVest Venture Partners, Peter Moldt, PhD, of Novo Ventures, Carole Nuechterlein of Roche Venture Fund and Jill Carroll of S.R. One will serve on the company’s board of directors, with Dr. McKearn serving as Chairman of the Board.

"We find CD47 to be an extremely interesting target in the evolving cancer immunotherapy landscape," said Peter Moldt, PhD, Partner at Novo Ventures. "We believe Tioma Therapeutics, with its portfolio of diverse, functionally heterogeneous antibodies, is well positioned to test the CD47 hypothesis in the clinic."

The company also announced today that John Donovan will lead the Tioma executive management team as President, Chief Executive Officer and member of the board of directors. John has more than two decades of industry experience, first as an investment banker and then as a member of senior leadership within several biotechnology companies. Most recently, he was a co-founder of Alios BioPharma and served as its Chief Business Officer and Chief Financial Officer through its acquisition by Johnson & Johnson.

"We are delighted to have John’s strong leadership skills, extensive corporate strategy experience and financial expertise to guide Tioma’s management team during this new phase of the company’s development," commented John McKearn, PhD, Managing Partner, RiverVest Venture Partners.

"We’re pleased by the degree of investor interest in this financing and thrilled to have enabled the robust advancement of our therapeutic antibodies well into clinical development," stated John Donovan, President and Chief Executive Officer of Tioma Therapeutics. "We’re particularly excited by the prospect of using these compounds in combination with other therapeutic agents, such as PD-L1/PD-1 inhibitors. We believe we have the ability to meaningfully improve outcomes for patients suffering from a variety of debilitating cancers."

About Immunotherapy and CD47

There are two distinct but interdependent arms of the immune system that work in concert to recognize cancer cells and destroy them. Both the innate and adaptive immune responses are capable of recognizing and destroying cancer cells but tumors evolve mechanisms to evade them.

Immunotherapy is intended to "wake" the immune system so that it can recognize and destroy cancer cells. Currently approved immuno-oncology therapies have been effective in a subset of patients despite harnessing only the adaptive immune response. Anti-CD47 antibodies have the potential to promote coordination by the innate and adaptive immune systems with both acting in concert to attack tumors.

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 Abeona Therapeutics Inc. (NASDAQ: ABEO) a clinical-stage biopharmaceutical company focused on delivering gene and plasma-based therapies for life-threatening rare diseases, reported financial results for the second quarter (Press release, Abeona Therapeutics, AUG 16, 2016, View Source;p=RssLanding&cat=news&id=2195628 [SID:1234514594]). The Company will provide investors an update on recent and ongoing business activities and an overview of its 2Q16 financials on, Wednesday, August 17th, at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international).

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"The second fiscal quarter of 2016 brought significant advancements in our goal of building a strong leadership position in the development of innovative therapies for rare diseases," stated Steven H. Rouhandeh, Executive Chairman. "We are excited about the initial biopotency signals seen in our Sanfilippo Type A clinical trial, and are looking forward to advancing multiple important new therapeutic candidates for the treatment of epidermolysis bullosa (EB), as announced last week. We thank our collaborators, shareholders and staff for their ongoing commitment and support as we move these therapies further into clinical development."

Timothy J. Miller, Ph.D, stated, "Abeona achieved many important regulatory and clinical milestones in the second quarter of 2016. The milestones include the dosing of the first patient in our Phase 1/2 clinical trial with ABO-102 for patients with Sanfilippo syndrome type A, the FDA allowance of our Phase 1/2 clinical study in Sanfilippo syndrome type B (MPS IIIB), and, most recently, the European regulatory approval for our MPS IIIA Phase 1/2 clinical study to be conducted at Cruces University Hospital in Bilbao, Spain. Additionally, we are excited about our new collaboration with the EB Research Partnership, EB Medical Research Foundation and Stanford University to accelerate up to three new promising EB product candidates toward commercialization."

Recent Abeona Operating Highlights

On May 17, 2016, Abeona announced that the first patient in its Phase 1/2 trial for ABO-102 (AAV-SGSH), a single treatment gene therapy strategy for patients with Sanfilippo syndrome type A (MPS IIIA), has been enrolled at Nationwide Children’s Hospital in Columbus, Ohio.
On May 24, 2016, Abeona announced the FDA Allowance of its Investigational New Drug (IND) for a Phase 1/2 clinical study with ABO-101 (AAV-NAGLU) for patients with Sanfilippo syndrome type B (MPS IIIB).
On August 2, 2016, Abeona provided an update on the initial subjects enrolled in this trial, stating that ABO-102 had been well tolerated with no safety or tolerability concerns identified through 30-days post-injection, and that encouraging signs of early biopotency had been observed in urinary and CSF GAG (heparan sulfate) measurements as well as potential disease-modifying effects in the liver and spleen.
On August 4, 2016, the Company announced it had received European regulatory approval by the Agencia Espanola de Medicamentos y Productos Sanitarios for its Phase 1/2 trial for ABO-102 (AAV-SGSH) to be conducted at Cruces University Hospital (Bilbao, Spain).
On August 9, 2016, Abeona announced a collaboration with the EB Research Partnership, EB Medical Research Foundation and Stanford University for the development of treatments for recessive dystrophic epidermolysis bullosa (RDEB). Clinical results for the lead EB program (EB-101) were recently presented at the opening Plenary Session of the Society for Investigative Dermatology in May 2016, and Investigators at Stanford are recruiting patients for a Phase 2 clinical trial of EB-101 in adolescents age 13 and older to determine the effect of type VII collagen gene corrective grafts on wound healing efficacy.
Second Quarter Summary Financial Results

Cash Position : Cash, cash equivalents and marketable securities as of June 30, 2016 were $34.3 million, compared to $37.4 million as of March 31, 2016. Net cash used in operating activities in the Six Months Ended June 30, 2016 was $5.6 million as compared to $5.0 million in the same period in 2015, an increase of $0.6 million.
Revenues : Revenues were $214 thousand for the second quarter of 2016, compared to $282 thousand in in the second quarter of 2015. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and recognition of deferred revenues related to upfront payments from early license agreements.
Loss per share : Loss per share was $0.20 for the second quarter of 2016, compared to a loss per share of $0.16 in comparable period in 2015.

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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