On May 14, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ:MBIO), a Fortress Biotech (NASDAQ:FBIO) Company focused on the development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell (CAR-T) technology, reported financial results for the first quarter ended March 31, 2018 (Press release, Mustang Bio, MAY 14, 2018, View Source [SID1234526588]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "In the first quarter of 2018, Mustang continued to execute on our strategy of developing a portfolio of differentiated CAR-T therapies for patients with aggressive forms of cancer. We are pleased to report significant progress on the build-out of our proprietary CAR-T cell processing facility at UMass Medicine Science Park in Worcester, Mass., which is on track to be fully operational and ready to process cells by the end of the year. We are also transitioning two preclinical CAR-T programs at City of Hope into the clinic in 2018, and plan to file our first Investigational New Drug Application during the fourth quarter. In March, we were delighted to announce the promotion of Sadik Kassim, Ph.D., to Chief Scientific Officer, and Knut Niss, Ph.D., to Chief Technology Officer, and look forward to continuing to work together to innovate in cell processing and to explore opportunities to leverage best-in-class science to strengthen our CAR-T pipeline. To this end, we will expand our internal research capabilities and plan to hire a team of scientists that will be fully dedicated to preclinical and translational research efforts."

Financial Results:

As of March 31, 2018, Mustang’s consolidated cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $55.3 million, compared to $61.5 million as of December 31, 2017, a decrease of $6.2 million for the quarter.
Research and development expenses were $4.3 million for the first quarter of 2018, compared to $0.7 million for the first quarter of 2017. Non-cash, stock-based compensation expenses included in research and development were $1.5 million for first quarter of 2018, compared to $0 million for the first quarter of 2017.
Research and development expenses from license acquisitions totaled $0.1 million for the first quarter of 2018, compared to $0.6 million for the first quarter of 2017.
General and administrative expenses were $2.1 million for the first quarter of 2018, compared to $2.0 million for the first quarter of 2017. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.5 million for the first quarter of 2018, compared to $1.2 million for the first quarter of 2017.
Net loss attributable to common stockholders was $6.3 million, or $0.24 per share, for the first quarter of 2018, compared to $3.2 million, or $0.14 per share, for the first quarter of 2017.

On May 14, 2018 Celgene Corporation (NASDAQ: CELG) reported to present at four upcoming investor conferences and events where Celgene management will provide an overview of the Company (Press release, Celgene, MAY 14, 2018, View Source [SID1234526564]). The events will be webcast live and will be available in the Investor Relations section of the Company’s website at www.celgene.com.

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Thursday, May 24, 2018, Celgene will host an R&D Deep Dive on Multiple Myeloma at 1:30pm ET

Wednesday, May 30, 2018, Celgene will present at the Sanford C. Bernstein Strategic Decisions Conference at 3:00 pm ET

Sunday, June 3, 2018, Celgene will host an Analyst & Investor Event at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting at 7:30 pm ET

Wednesday, June 6, 2018, Celgene will present at the Jefferies 2018 Global Healthcare Conference at 1:30 pm ET

On May 14, 2018 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a biopharmaceutical company studying topsalysin (PRX302), a first-in-class, pore-forming protein, in late stage clinical trials for the treatment of patients with urological diseases, reported first quarter 2018 financial results (Press release, Sophiris Bio, MAY 14, 2018, View Source [SID1234526589]).

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"It has been an exciting quarter at Sophiris as we continue to make progress in advancing topsalysin," said Randall E. Woods, president and CEO of Sophiris. "We are looking ahead to two key events in 2018. By the end of the second quarter, we expect to announce the biopsy results from all patients receiving the first administration of topsalysin in our Phase 2b study, and by the end of the year, we expect complete data from all patients including those patients who received a second administration of topsalysin. In advance of these milestones, we have been actively preparing for a Phase 3 registration study, including engaging in initial discussions with European regulatory agencies. In addition, we are actively moving forward with our manufacturing plans to provide sufficient drug substance for a potential Phase 3 registration study in localized prostate cancer and also a potential second Phase 3 in BPH."

Upcoming Milestones:

Advancement of Phase 2b Localized Prostate Cancer Study. The Company announced in December 2017 that it had completed enrollment in its Phase 2b localized prostate cancer study to evaluate the safety and tolerability of topsalysin in treating men with clinically significant localized prostate cancer. A total of 38 patients have been treated with topsalysin in the study. The Company expects biopsy data from all patients receiving the first dose of topsalysin to be available by the end of the second quarter for 2018.

During the first quarter of 2018, the independent data monitoring committee (IDMC) for the Phase 2b trial met to review the reported adverse events from all patients after the first administration of topsalysin. The IDMC unanimously recommended the clinical trial continue without changes to the protocol. The Company believes that topsalysin continues to demonstrate a favorable safety profile.

The Phase 2b study was designed to include an option to re-treat patients who did not have any clinically significant adverse events and who responded to the first administration of topsalysin but still had a clinically significant lesion. These patients will have the option to receive a second administration of topsalysin followed by an additional, targeted biopsy six months following the second administration. The Company expects to have final biopsy data in the fourth quarter of 2018 from all patients who receive a second administration. This will be the first data potentially supporting repeat administration of topsalysin.

Financial Results:

At March 31, 2018, the Company had cash, cash equivalents and securities available-for-sale of $22.1 million and working capital of $19.2 million. The Company expects that its cash and cash equivalents will be sufficient to fund its operations to the middle of 2019, assuming no new clinical trials are initiated. The Company reported a net loss of $3.3 million or $(0.11) per share for the three months ended March 31, 2018, compared to a net loss of $2.6 million or $(0.09) per share for the three months ended March 31, 2017.

Research and development expenses

Research and development expenses were $3.3 million for the three months ended March 31, 2018, compared to $1.2 million for the three months ended March 31, 2017. The increase in research and development costs is primarily attributable to increases in the costs associated with manufacturing activities for topsalysin, and to a lesser extent, an increase in clinical costs associated with our Phase 2b clinical trial of topsalysin for the focal treatment of localized prostate cancer.

General and administrative expenses

General and administrative expenses were $1.2 million for the three months ended March 31, 2018, compared to $1.4 million for the three months ended March 31, 2017. The decrease in general and administrative expense is primarily due to a decrease in non-cash stock-based compensation expense which was partially offset by an increase in professional services.

Gain (loss) on revaluation of the warrant liability

Gain on revaluation of the warrant liability was $1.4 million for the three months ended March 31, 2018, compared to a loss of $86,000 for the three months ended March 31, 2017. As these warrants may require the Company to pay the warrant holder cash under certain provisions of the warrant, the Company accounts for these warrants as a liability, and the Company is required to calculate the fair value of these warrants each reporting date. The non-cash gain reported for the three months ended March 31, 2018, is associated with a decrease in the fair value of the Company’s warrant liability from December 31, 2017, to March 31, 2018, which is calculated using a Black-Scholes pricing model. Certain inputs utilized in the Company’s Black-Scholes fair value calculation may fluctuate in future periods based upon factors which are outside of the Company’s control. A significant change in one or more of these inputs used in the calculation of the fair value may cause a significant change to the fair value of the Company’s warrant liability, which could also result in a material non-cash gain or loss being reported in the Company’s consolidated statement of operations and comprehensive loss.

On May 14, 2018 ABIVAX (Euronext Paris: FR0012333284 – ABVX), a biotechnology company harnessing the immune system to develop a functional cure for HIV as well as treatments for inflammatory/autoimmune diseases and cancer, reported the completion of enrollment of its Phase IIA clinical trial ABX464-101 in 30 patients with moderate-to-severe ulcerative colitis (Press release, ABIVAX, MAY 13, 2018, View Source [SID1234526552]).

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"ABIVAX is very pleased to announce the completion of enrolment in this first clinical trial exploring ABX464 for treatment of inflammatory indications," said Prof. Dr. Hartmut Ehrlich, M.D., chief executive officer at ABIVAX. "I would like to highlight the outstanding enthusiasm and dedication of Prof. Dr. Severine Vermeire, M.D., the Principal Investigator of this multinational trial and past president of ECCO (European Crohn’d and Colitis Organization), and thank the clinical trial sites, which kept recruitment on target, allowing us to report the top-line results from this important clinical trial in the autumn of this year," added Dr. Ehrlich.

ABX464-101 is a randomized, double-blind, placebo-controlled clinical trial (Phase 2a proof-ofconcept study) aimed at evaluating the safety and efficacy of ABX464 50 mg given once daily versus placebo for two months in subjects with moderate-to-severe active ulcerative colitis who have failed or are intolerant to immunomodulators, anti-TNFα, vedolizumab and/or corticosteroids. This clinical study is being conducted in 17 centers in seven European countries: Belgium, France, Germany, Austria, Hungary, Poland and Czech Republic. As of today, 30 out of the planned 30 patients have been randomized 2:1 to receive ABX464 or placebo, respectively. The study employs state-of-the art
technologies for monitoring potential treatment effects, including numerical recording of the colonoscopies with centralized reading.

ABX464-102 is a 12 months open label follow-up study for patients who completed ABX464-101, and 10 patients are already recruited into this clinical trial as of today. The rationale for the ABX464-101 study was derived from encouraging preclinical data, which demonstrated ABX464 had a strong anti-inflammatory effect. In macrophages, this effect was shown to be mediated by a 50-fold increase of the expression of IL-22, a cytokine known as a potent
suppressor of inflammatory processes, and a ten-fold increase of miR124 in peripheral blood mononuclear cells(PBMCs). mIR124 is a micro-RNA with potent anti-inflammatory properties and has recently been described as a tumor suppressor gene. Inflammation is a cornerstone of IBD, including ulcerative colitis and Crohn’s disease. When evaluated in a mouse model of IBD, ABX464 demonstrated a long-lasting effect in preventing the typical
symptoms of inflammatory colitis, including histological changes1
.
Prof. Dr. Severine Vermeire, M.D., Head of the IBD center at the University Hospitals Leuven, Belgium and Principal Investigator of the study, said: "reaching our recruitment goal of 30 patients marks an important step as there is a strong need to develop additional drugs in this indication; too many patients still do not respond or stop responding to current treatments. We are looking forward to the top-line data from this study and also to transferring as many patients as possible onto the one year open-label extension study with ABX464, which will provide us with very important long-term safety and efficacy data."

Ulcerative colitis is a debilitating inflammatory bowel disease in adults and children, with limited therapeutic management options for many patients. There is an estimated number of close to 1 million patients with ulcerative colitis in the United States, and global pharmaceutical sales for this disease are estimated to be around 5.7 billion US$ in 2017.

On May 11, 2018 Advaxis, Inc. (NASDAQ: ADXS), a late-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported on recently presented preclinical data demonstrating the anti-cancer potential of their Lm vector that presents tumor neoantigens, and is being evaluated in the ADXS-NEO program (Press release, Advaxis, MAY 11, 2018, View Source [SID1234526525]). ADXS-NEO is derived from the Company’s proprietary Lm Technology and is being developed in partnership with Amgen.

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These preclinical findings were discussed in poster presentations at the recent American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Additionally, portions of these data were presented by Amgen at a podium presentation during the European Neoantigen Summit 2018.

The first study, as discussed in a poster presentation at AACR (Free AACR Whitepaper) entitled "Neoantigens that fail to elicit measurable T cell responses following peptide immunization can control tumor growth when delivered using a Listeria-based immunotherapy platform," showed that ADXS-NEO generates T cell responses against neoantigen peptides that control tumor growth, even when they were identified as "non-immunogenic" using a conventional peptide-adjuvant immunization. The poster is available here: View Source

In the second study, discussed in a poster presentation at AACR (Free AACR Whitepaper) entitled "Targeting frameshift mutations with a Listeria monocytogenes immunotherapy drives neoantigen-specific antitumor immunity in MC38 and CT26 mouse tumor models," Advaxis’ Lm platform was shown to target frameshift mutations and generate T cells to multiple neoantigens per frameshift in MC38 and CT26 mouse tumor models. The poster is available here: View Source

"The results of these studies highlight the therapeutic potential of targeting neoantigens and suggest that ADXS-NEO could impact the way we approach treatment across a range of cancers," said Ken Berlin, President and CEO of Advaxis. "We look forward to our continued collaboration with Amgen, and to moving into the clinic with this product candidate."

"These preclinical findings provide foundational rationale suggesting that ADXS-NEO has the potential to generate immune responses against multiple neoantigens with the ability to control tumor growth. This is a personalized approach that uses a patient’s own immune system to recognize and eliminate cancer cells harboring multiple mutations that caused their malignancy," said Robert G. Petit, Ph.D., Chief Scientific Officer and Executive Vice President of Advaxis. "We also saw potent immune responses targeting frameshift mutations and control of tumor growth via multiple complementary mechanisms. This is important because frameshift mutations are observed to generate up to nine times more neoantigens per mutation than in-frame mutations" according to Turajlic, et al.

About ADXS-NEO

ADXS-NEO is a personalized Listeria monocytogenes (Lm)-based immunotherapy designed to generate immune response against mutation-derived tumor-specific neoantigens. The program focuses on creating a customized treatment with neoantigens specifically selected based on a biopsy of the patient’s tumor. The approach enables the development of tailored immune-therapies. ADXS-NEO is being evaluated in collaboration with Amgen.