On May 14, 2018 Precision BioSciences, the ARCUS genome editing company with novel product development programs in gene therapy, cancer immunotherapy and non-GMO food, reported that it will give three presentations at the upcoming ASGCT (Free ASGCT Whitepaper) 21st Annual Meeting held in Chicago, Illinois, May 16-19, 2018 (Press release, Precision Biosciences, MAY 14, 2018, View Source [SID1234526597]).

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Precision’s co-founder and CSO, Derek Jantz, will attend the conference along with a group of Precision scientists. He notes,

"The Precision team is looking forward to participating in the research dialogue at ASGCT (Free ASGCT Whitepaper) this year. We are particularly eager to share our experiences towards developing an in vivo gene editing solution, including results of extensive NHP testing. We will also present new data from our gene-edited allogeneic T cell platform, including manufacturing results for our lead CAR T program."

Precision BioSciences presentations at ASGCT (Free ASGCT Whitepaper) include:

1. Development and Optimization of a PCSK9-Specific Meganuclease That Mediates Long-Term LDL Reduction in Non-Human Primates (#658; follows content of #657).
Oral Session Title: Cardiovascular and Pulmonary Diseases
Date and Time: Friday, May 18, 2018, 4:00 p.m. – 5:45 p.m. CDT
Location: International Ballroom South

2. Development of a Clinical-Grade Meganuclease for Allogeneic CAR T Cell Production (#784).
Poster Session Title: Cell Therapies III
Session Date and Time: Friday, May 18, 2018, 5:45 p.m. – 7:45 p.m. CDT
Location: Stevens Salon C & D

3. A P23H RHO-Specific Meganuclease Rescues Photoreceptor Morphology and Function in Mouse Models of Retinitis Pigmentosa (#565).
Poster Session Title: Neurologic Diseases II
Session Date and Time: Thursday, May 17, 2018, 5:15 p.m. – 7:15 p.m. CDT
Location: Stevens Salon C & D

On May 14, 2018 Coherus BioSciences, Inc. (NASDAQ:CHRS), reported that the U.S. Food and Drug Administration (FDA) has accepted and acknowledged for review the re-submission of the biologics license application (BLA) for CHS-1701, a pegfilgrastim (Neulasta) biosimilar candidate (Press release, Coherus Biosciences, MAY 14, 2018, View Source;p=RssLanding&cat=news&id=2348926 [SID1234526578]). In the communication, FDA indicated that they consider the resubmission a complete response to their June 9, 2017 action letter. FDA provided a biosimilar user fee act (BSUFA) action date of November 3, 2018. The letter did not indicate the need to prepare for an advisory committee meeting.

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"We appreciate FDA’s prompt action on our file and look forward to working with them on the review," said Denny Lanfear, President and CEO of Coherus BioSciences. "We believe that CHS-1701 is well-positioned to deliver greater access to oncology patients and savings to the healthcare system. We are continuing to make good progress in building inventory of CHS-1701 and in preparing for commercial launch in the U.S."

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 Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, reported financial results for the first quarter of 2018 (Press release, Abeona Therapeutics, MAY 11, 2018, View Source;p=RssLanding&cat=news&id=2348683 [SID1234526535]). The Company will host a call to update investors on recent clinical developments and quarter financial results on Monday, May 14th at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-407-9210 (toll free domestic) or 201-689-8049 (International) or via webcast View Source

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"Abeona has made significant progress across multiple fronts, including various regulatory achievements, additional enrollments in our MPS IIIA trial, and the reporting of encouraging initial results for our MPS IIIB trial," stated Carsten Thiel, Ph.D., Abeona’s CEO. "Notably, both our lead clinical programs, EB-101 and ABO-102, were recently granted RMAT designation by the FDA, supporting the strong safety and biopotency evidence from these programs, and their potential to address the unmet medical need for RDEB and MPS IIIA patients."

1st Quarter Summary Financial Results:

– Cash position: Cash and cash equivalents as of March 31, 2018 were $132 million, compared to $137.8 million as of December 31, 2017.
– Revenues: Revenues were $2.6 million for the first quarter of 2018, compared to $186 thousand in the first quarter of 2017. A large portion of the increased quarterly revenues consisted of the recognition of Foundation grants that were announced during the 4th quarter of 2017. A portion of the grants were received in the 4th quarter of 2017 and in the 1st quarter of 2018, and the amount recognized is matched against corresponding expenditures for drug manufacture and clinical readiness. Additional revenues consisted of royalties from marketed products, specifically MuGard. In the quarter, Abeona adopted ASC 606 pertaining to revenue recognition, and therefore there will no longer be any recognition of deferred revenues related to upfront payments from earlier license agreements.
– Loss per share: Loss per share was $0.18 for the first quarter of 2018, compared to a loss per share of $0.13 in the comparable period in 2017.

Abeona Recent Highlights:

April 23, 2018: Announced FDA Grants RMAT Designation to ABO-102 Gene Therapy in MPS IIIA
April 20, 2018: Announced EMA Grants Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease
April 2, 2018: Announced Appointment of Carsten Thiel, Ph.D., as Chief Executive Officer
March 15, 2018: Announced FDA Grants Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Disease
February 12, 2018: Announced FDA Grants Orphan Drug Designation for ABO-202 Gene Therapy Program in Infantile Batten Disease
February 8, 2018: Reported Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA
— ABO-102 results presented at WORLDSymposium for Lysosomal Diseases show significant time- and dose-dependent reduction of underlying disease pathology, including decreased CSF and urine GAGs (HS fragments) and diminished liver volumes
— Evidence of cognitive benefit at six months post treatment in Cohort 2 and at one year in Cohort 1
— Company receives FDA allowance to lower enrollment age to six months
February 7, 2018: Reported on Initial Safety and Biopotency Signals in MPS IIIB Gene Therapy Clinical Trial
— ABO-101 is well tolerated and demonstrates early biopotency signals with significant disease-specific heparan sulfate (HS) reductions in cerebral spinal fluid, urine, and plasma and greater than 300-fold increase in NAGLU enzyme activity observed in first subject at 30 days post injection
January 29, 2018: Announced FDA Grants Regenerative Medicine Advanced Therapy Designation for EB-101 Gene Therapy in Epidermolysis Bullosa

"2018 continues to be a year of execution for Abeona. With the expanded leadership, we look to a number of milestones in the year including the commencement of our pivotal Phase 3 trial in EB, the completion of enrollment in our Phase 1/2 trial in MPS IIIA, and significant progress on our in-house GMP manufacturing facility," stated Steven H. Rouhandeh, Abeona’s Executive Chairman. "With the team focusing their efforts and leading the charge on clinical development, we look forward to our continued dialogue with the regulatory bodies and advancing our gene therapies to rare disease patients."

On May 11, 2018 Paul Changelian, Ph.D., Vice President, Biology, Aclaris Therapeutics, Inc. (NASDAQ:ACRS), reported that it will speak at the 2018 American Hair Research Summit on "Preclinical to Clinical Translation of JAK Kinase Inhibitors" on May 15 (Press release, Aclaris Therapeutics, MAY 11, 2018, View Source [SID1234526536]). The meeting is organized by the American Hair Research Society (AHRS) and will take place May 14-16 in Orlando, Florida.

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Dr. Changelian will review the history of drug discovery targeting Janus kinase (JAK) enzymes. JAK inhibitors initially were conceived as drugs to reduce organ transplant rejection but gradually were found to have utility in autoimmune diseases and oncology. Harnessing the benefits of JAK inhibition for dermatological conditions will require minimizing the potential for over suppression of the immune system and subsequent infections, through strategies such as identifying molecules highly selective for JAK1 and JAK3 inhibition or developing topical formulations with high skin permeability while minimizing systemic absorption. Current efforts to expand the range of diseases that could be targeted with JAK inhibitors will be discussed with emphasis on hair disorders, including alopecia areata and androgenetic alopecia.

Dr. Changelian is known for his key role in the discovery and development of tofacitinib, a pan-JAK inhibitor, while working as a scientist at Pfizer. In 1993, he first heard about the National Institutes of Health’s discovery of an enzyme in the Janus family of kinases involving the immune function. Seven years after initiating the discovery program, Dr. Changelian and the Pfizer immunology team selected tofacitinib for clinical development. Tofacitinib became the first JAK inhibitor approved by the FDA for an autoimmune disease. Dr. Changelian joined Aclaris in 2017 when Aclaris acquired Confluence Life Sciences, Inc., a company founded by former Pfizer scientists.