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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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 Radius Health, Inc. (Nasdaq:RDUS) reported that Jesper Høiland, President and Chief Executive officer and Pepe Carmona, Senior Vice President and Chief Financial Officer of the Company, will present a corporate update at the Bank of America Merrill Lynch 2018 Annual Healthcare Conference on Tuesday, May 15, 2018 (Press release, Radius, MAY 11, 2018, View Source [SID1234526549]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Information on the presentation is as follows:

Event: Bank of America Merrill Lynch 2018 Annual Healthcare Conference
Date: Tuesday, May 15, 2018
Time: 1:40 p.m. PDT
Location: Encore Hotel, Las Vegas, NV
A live webcast of the presentation will be available by visiting the Investors section of Radius’ website at View Source A replay of the webcast will be archived on Radius’ website for 30 days following the presentation

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

On May 11, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported financial results for the quarter ended March 31, 2018, and provided an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 11, 2018, View Source;p=irol-newsArticle&ID=2348660 [SID1234526542]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2018 is off to a great start for Rocket. During the first quarter, we continued to make excellent progress advancing our pipeline of lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy programs towards key value inflection points. We look forward to presenting updated patient data from the Fanconi Anemia program later this month, and to disclosing our AAV-based gene therapy program by the end of the year," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We remain on track for multiple regulatory filings across our pipeline over the next 12 months. As a result, there is the potential for four programs to be in the clinic in 2019, with clinical data for up to two programs."

Dr. Shah continued, "While near-term milestones are certainly important, Rocket’s long-term strategy is based on a sustainable and integrated multi-platform approach. We remain focused on our mission of continued innovation to deliver best-in-class curative cell and gene therapies to patients living with devastating diseases."

Anticipated Milestones Over the Next 12 -18 Months

Fanconi Anemia (FA): Updated patient data from the ongoing Phase 1/2 study of RP-L102 conducted with CIEMAT is expected over the next 12 months. The next update will be provided at the American Society of Gene and Cell Therapy’s (ASGCT) (Free ASGCT Whitepaper) 21st Annual Meeting in Chicago. The oral presentation is scheduled during the Presidential Symposium on Friday, May 18, 2018, at 2:45 p.m. Central Time.
Leukocyte Adhesion Deficiency-I (LAD-I): An Investigational Medicinal Product Dossier (IMPD) is expected to be filed in the fourth quarter of 2018. Clinical data is expected over the next 12-18 months.
Pyruvate Kinase Deficiency (PKD): An IMPD application is expected to be filed in early 2019.
Infantile Malignant Osteopetrosis (IMO): Preclinical studies continue to advance in support of filing a first-in-human clinical study.
AAV: Disclosure of the disease indication and preclinical data is planned for the second half of 2018.
Upcoming Conferences

Bank of America Merrill Lynch Health Care Conference 2018. Rocket is scheduled to present on Tuesday, May 15, at 9:20 a.m. Pacific Time.
UBS Global Healthcare Conference. Rocket is scheduled to present on Wednesday, May 23, at 9:30 a.m. Eastern Time.
Jefferies Global Healthcare Conference. Rocket is scheduled to present on Thursday, June 7, at 2:00 p.m. Eastern Time.
First Quarter 2018 Financial Results

Cash position. Cash, cash equivalents and short-term investments as of March 31, 2018, were $182.7 million, which includes a $52.0 million fully convertible debenture which expires in 2021.
R&D expenses. Research and development expenses were $5.7 million for the quarter ended March 31, 2018, compared to $2.3 million for the quarter ended March 31, 2017.
G&A expenses. General and administrative expenses were $8.7 million for the quarter ended March 31, 2018, compared to $0.6 million for the quarter ended March 31, 2017. The increase in G&A expenses was primarily due to one-time merger related expenses of $5.3 million.
Net loss. Net loss was $15.3 million or $(0.42) per share (basic and diluted) for the quarter ended March 31, 2018, compared to $2.7 million or $(0.39) per share (basic and diluted) for the quarter ended March 31, 2017.
Shares outstanding. Approximately 39.4 million shares of common stock were outstanding as of March 31, 2018.
Financial Guidance

Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and short-term investments as of March 31, 2018, will be sufficient to run its operations into 2020.

On May 11, 2018 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported that company executives are scheduled to participate in the following upcoming investor conferences (Press release, Ligand, MAY 11, 2018, View Source [SID1234526541]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

• Berenberg Conference USA in Tarrytown, New York. Presentation takes place on Thursday, May 24, 2018 at 11:45 a.m. Eastern time (8:45 a.m. Pacific time). Matt Korenberg, CFO will attend for Ligand.

• 15th Annual Craig-Hallum Institutional Investor Conference in Minneapolis. Conference takes place on Wednesday, May 30, 2018 with one-on-one meetings only. John Higgins, CEO, Matt Foehr, COO and Matt Korenberg, CFO will attend for Ligand.

• Jefferies Healthcare Conference in New York City. Presentation takes place on Wednesday, June 6, 2018 at 8:00 a.m. Eastern time (5:00 a.m. Pacific time). Matt Korenberg, CFO will attend for Ligand.

A live webcast of the Jefferies conference presentation will be available on Ligand’s website at www.ligand.com. A replay of the presentations will be archived on the website for 30 days.