On May 14, 2018 Immunicom, Inc., a medical technology company developing revolutionary non-pharmaceutical approaches for treating cancer and autoimmune diseases, reported the company has received Breakthrough Device designation from the U.S. Food and Drug Administration (FDA) for its Immunopheresis therapy, which is based on a proprietary technology that selectively removes immune inhibitors from a patient’s bloodstream potentially enabling their natural immune system to more-effectively attack cancer tumors (Press release, Immunicom, MAY 14, 2018, View Source [SID1234637423]).

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To achieve Breakthrough Device designation, a technology must demonstrate compelling potential to provide more effective treatment or diagnosis for life-threatening or irreversibly debilitating diseases. In addition, there must be no FDA approved treatments presently available, or the technology must offer significant advantages over existing approved alternatives.
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The FDA’s intent in granting this specific designation to qualified devices is to collaboratively facilitate expediting the device’s assessment and review processes through more interactive communication and planning with the FDA; ensuring proper data collection, efficient clinical study design, senior management engagement of Agency personnel, and priority review of applicable filings.

In contrast to immunotherapy pharmaceuticals and biologicals that introduce foreign compounds and material into a patient’s body, and which are often accompanied by negative side effects, Immunicom’s patented technology is based on a "subtractive" approach that is intended to potentially limit treatment-associated adverse effects. By removing immune system inhibitors from the blood without introducing new substances, Immunicom’s therapeutic approach has been shown in preliminary preclinical studies to enhance anti-cancer immune system response without causing unwanted side effects.
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Immunicom’s blood-filtering device technology is the first step in execution of a broader corporate strategic vision. "We are very pleased with FDA’s granting of Breakthrough Device designation for Immunopheresis, our initial immunotherapy product for treating late/end stage IV cancer patients with metastatic solid tumors," said Amir Jafri, CEO of Immunicom, "This significant milestone will enable us to more efficiently pursue device regulatory approval and address critical unmet patient needs sooner, while also continuing our commitment to invest in other exciting treatment options in our product pipeline."

On May 14, 2018 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies based on its novel, universal Antibody-Coupled T-cell Receptor (ACTR) technology platform, reported financial results and provided a corporate update for the first quarter ended March 31, 2018 and recent activities (Press release, Unum Therapeutics, MAY 14, 2018, View Source [SID1234526563]).

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"Following our successful initial public offering in April 2018 and concurrent private placement, we are in a strong financial position to continue developing our proprietary, universal ACTR technology platform and rapidly advancing our pipeline of cellular immunotherapies through clinical development," said Chuck Wilson, CEO of Unum. "We are currently evaluating the potential of ACTR in combination with different tumor-targeting antibodies, in three ongoing multi-center Phase I trials, ATTCK-20-2 and ATTCK-20-03 evaluating ACTR087 and ACTR707, respectively, in combination with rituximab in patients with CD20+ r/r Non-Hodgkin Lymphoma (NHL), and ATTCK-17-01 evaluating ACTR087 in combination with SEA-BCMA in patients with r/r multiple myeloma. We expect to report preliminary data from these three trials late this year. In the second half of 2018 we also look forward to filing an IND and initiating clinical development of ACTR707 in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers, our first solid tumor product candidate."

Recent Business Highlights and Outlook

Successfully Completed IPO and Concurrent Private Placement: In April, 2018, Unum successfully completed an initial public offering (IPO) of 5,985,000 shares of common stock at a public offering price of $12.00 per share, including the exercise by the underwriters of 215,000 shares of their overallotment option, raising $66.8 million in net proceeds. In addition, with a private placement concurrent with the IPO, Seattle Genetics, Inc. purchased $5.0 million shares of common stock at the initial public offering price. The proceeds from the IPO and the concurrent private placement will be used primarily to advance Unum’s four lead ACTR development candidates.

Initiated Cohort Expansion Phase of ATTCK-20-2 Phase I trial; Plans to Expand Clinical Development: In May 2018, Unum initiated the cohort expansion phase of the ATTCK-20-2 trial evaluating safety and anti-lymphoma activity of ACTR087 at the preliminary recommended phase 2 dose (RP2D) level used in combination with rituximab in patients with CD20+ r/r NHL. Unum expects to report updated data, including preliminary data from this phase of the ATTCK-20-2 trial, in the fourth quarter of 2018.

These data will also inform the strategy for a planned multi-center Phase II clinical trial exploring ACTR T cells used in combination with rituximab in patients with CD20+ r/r NHL who received prior CD19 CAR T cell therapy.

In addition, Unum intends to file a protocol amendment to the ATTCK-20-2 trial in the second half of 2018 to explore ACTR087 in combination with an alternative rituximab dosing regimen from that currently being studied. Preclinical experiments have shown that the level of ACTR T cell activity depends upon the amount of the co-administered antibody. As such, ACTR087 safety and anti-tumor activity in combination with rituximab in CD20+ r/r NHL may be even further optimized by an alternative rituximab regimen. Testing the alternative regimen will complement the clinical data being generated to support additional clinical trials with the combination.

Initiated Patient Enrollment and Dosing in ATTCK-17-01 Phase I trial: In the first quarter, Unum initiated patient enrollment ATTCK-17-01, a Phase I, multi-center, open-label clinical trial designed to test the safety, tolerability, and anti-myeloma activity of ACTR087 used in combination with SEA-BCMA in patients with r/r multiple myeloma. Unum is currently enrolling and dosing patients in this trial and expects to report preliminary data in the fourth quarter of 2018.

Continued Enrollment in ATTCK-20-03 Phase I trial: In the fourth quarter of 2017, Unum initiated patient enrollment in a Phase I, multi-center, open-label clinical trial called ATTCK-20-03, evaluating the safety, tolerability, and anti-lymphoma activity of ACTR707 used in combination with rituximab in patients with CD20+ r/r NHL. Unum has completed enrollment in the first dose level of this ongoing dose escalation study and expects to report preliminary data from the trial in the fourth quarter of 2018.

On Track to File IND for First Solid Tumor ACTR Product Candidate in the Second Half of 2018: Unum is on track to file an IND in the second half of 2018 for ACTR707 in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers.
First Quarter 2018 Financial Results

Collaboration Revenue: Collaboration revenue recognized during the three months ended March 31, 2018 and 2017 of $2.2 million and $1.8 million, respectively, reflects the recognition of a portion of the $25.0 million upfront payment received from Seattle Genetics under Unum’s collaboration agreement as well as reimbursements of research and development costs by Seattle Genetics. Effective January 1, 2018, Unum adopted the new revenue recognition standard, ASC 606, which changed the manner in which the Company recognizes revenue from this collaboration agreement.

R&D Expenses: Research and development expenses were $8.1 million for the three months ended March 31, 2018, compared to $7.0 million for the same period last year. The increase reflects higher clinical trial costs for the three active Phase I clinical trials, as well as increased personnel-related costs, materials and facility-related costs related to scaling manufacturing processes, and increased consultant costs. This was partially offset primarily by a decrease in consulting and manufacturing costs incurred for the Phase I clinical trial of ACTR087 in combination with rituximab as there was no production activity in the first quarter of 2018.

G&A Expenses: General and administrative expenses for the three months ended March 31, 2018 were $1.1 million, compared to $0.9 million for the prior year period.

Net Loss: Net loss attributable to common stockholders was $6.8 million, or $0.66 per share, for the three months ended March 31, 2018, and $6.0 million, or $0.58 per share, for the three months ended March 31, 2017.

Cash, Cash Equivalents and Marketable Securities: As of March 31, 2018, Unum had cash, cash equivalents, and marketable securities of $32.4 million. This amount does not include the approximately $66.8 million in net proceeds from its IPO in April 2018, $5.0 million from the concurrent private placement, and available borrowings under its loan and security agreement of $15.0 million. The Company believes that the net proceeds from the IPO and concurrent private placement, together with its existing cash, cash equivalents, and marketable securities, will fund operating expenses and capital expenditure requirements through at least December 2019, without considering available borrowings under its loan and security agreement.

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 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
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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 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results for the three months ended March 31, 2018 (Press release, Trillium Therapeutics, MAY 11, 2018, View Source [SID1234526543]).

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"Following the initial signals of monotherapy responses reported at ASH (Free ASH Whitepaper) last year, we have increasingly focused both our TTI-621 clinical trials on patients with T-cell malignancies throughout the first quarter of 2018" said Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics. "We have also been preparing to launch our second clinical CD47 program, TTI-622. Having both an IgG1 and an IgG4 SIRPaFc fusion protein in clinical testing should allow us to address important scientific questions related to the impact of the Fc region in various clinical scenarios, including combination therapy."

2018 First Quarter Highlights:

Reported refinements to both our phase 1 trials of TTI-621 to focus near-term efforts on patients with cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL). This action builds on the monotherapy results of TTI-621 presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 where weekly infusions of TTI-621 were shown to be well tolerated and intratumoral injection was observed to reduce local lesions in 9 out of 10 patients with mycosis fungoides, a common type of CTCL.
The U.S. Food and Drug Administration granted an Orphan Drug Designation to TTI-621 for the treatment of cutaneous T-cell lymphoma. Orphan Drug Designation qualifies the sponsor of the drug candidate for various development incentives, which include an exemption from fees under the Prescription Drug User Fee Act and a seven-year marketing exclusivity period following approval.
Presented preclinical TTI-622 data at the 2018 AACR (Free AACR Whitepaper) Annual Meeting demonstrating that TTI-622 induces the phagocytosis of a broad panel of tumor cells derived from patients with both hematological and solid tumors. As a monotherapy, TTI-622 treatment resulted in decreased tumor growth and improved survival in a B cell lymphoma xenograft model, as well as enhanced the efficacy of cetuximab (anti-EGFR) and daratumumab (anti-CD38) antibodies in solid and hematological xenograft models, respectively. We expect to enroll the first patient in a Phase 1 clinical trial of TTI-622 in Q2 2018.
First Quarter 2018 Financial Results

As of March 31, 2018, Trillium had cash and cash equivalents and marketable securities, and working capital of $73.9 million and $61.7 million, respectively, compared to $81.8 million and $68.9 million, respectively at December 31, 2017. The decrease in cash and cash equivalents and marketable securities, and working capital was due mainly to cash used in operations of approximately $9.5 million.

Net loss for the three months ended March 31, 2018 of $8.6 million was lower than the loss of $11.5 million for the three months ended March 31, 2017. The net loss was lower due mainly to a net foreign currency gain of $1.6 million for the three months ended March 31, 2018, compared to a net foreign currency loss of $0.4 million in the prior year period. Research and development expenses decreased by $0.9 million in 2018 as a result of lower manufacturing activity for TTI-621 and TTI-622. These decreases were partially offset by higher clinical trial expenses.