Weekly Quick Hits (Research Triangle Park) – Week of April 10, 2023

By Alex Keown, Mark Terry, and Sarah Ellinwood
April 14, 2023

Funding, Awards and Collaborations

RedHill Biopharma Regains Compliance with Nasdaq 

RedHill Biopharma received confirmation from The Nasdaq Stock Market LLC that it had regained compliance with the minimum bid price requirement under Nasdaq Listing Rule 5450(a)(1) for continued Nasdaq listing. To regain compliance, the company was required to maintain a minimum closing bid price of $1 or more for at least 10 consecutive trading days, which was achieved on April 5, 2023.

In the Clinic

Nuance Pharma Doses First Patient in Phase III ENHANCE Trial for COPD

Raleigh-based Nuance Pharma dosed the first subject in its ENHANCE – China Phase III trial for the maintenance treatment of chronic obstructive pulmonary disease (COPD). In August 2022, Nuance Pharma received clearance from China’s Center of Drug Evaluation for its Investigational New Drug application to conduct both Phase I and Phase III studies with ensifentrine for the maintenance treatment of COPD in mainland China. Nuance Pharma initiated a Phase I trial with ensifentrine in healthy volunteers in March 2023.

Atsena Therapeutics Announces Six-Month Data from Phase I/II Clinical Trial of ATSN-101

Atsena Therapeutics, a clinical-stage gene therapy company based in Durham, announced it will present six-month safety and efficacy data from the ongoing Phase I/II clinical trial of ATSN-101 at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting later this month. ATSN-101 is being evaluated in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). LCA1 is a monogenic eye disease that disrupts the function of the retina and results in early and severe vision impairment or blindness. According to previously reported interim data from the Phase I/II clinical trial, ATSN-101 has demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events.

Humacyte Completes Enrollment in Phase III Study

Humacyte, based in Durham, completed enrollment of a Phase III trial in hemodialysis access. The “V007 Trial” is designed to assess the efficacy and safety of the Human Acellular Vessel (HAV) in establishing vascular access for hemodialysis patients with end-stage renal disease (ESRD) as compared to autogenous arteriovenous (AV) fistulas. The V007 trial is a prospective, multi-center, randomized, comparative study in 240 hemodialysis patients suffering from ESRD in the United States. Enrolled individuals were randomly assigned to either the ​​HAV, or an AV fistula for hemodialysis.

New Patents

Precision BioSciences Receives Patent for Proprietary PCSK9 ARCUS Nuclease

Precision BioSciences received a Notice of Allowance from the U.S. Patent and Trademark Office for U.S. Patent Application No. 16/606,856, titled “Engineered meganucleases specific for recognition sequences in the PCSK9 gene.” The allowed composition of matter claims in these U.S. and international applications encompass a PCSK9-specific ARCUS nuclease. This ARCUS nuclease has been used preclinically for both gene deletion approaches for cardiovascular diseases such as familial hypercholesterolemia. It is also used as a viable and safe site to insert a functional copy of a specific gene to restore function addressing other diseases requiring gene addition. Corresponding international applications in this family have also been allowed in Europe, Australia, Mexico, Japan, and Israel since October 2022. 

Research Roundup

Researchers Harness Cell Self-Destruction for Brain Tumors

Researchers from the University of North Carolina at Chapel Hill in partnership with a team from Columbia University found that glioblastoma tumor cells are sensitive to ferroptosis, which is a type of cell death caused by the removal of certain amino acids from the diet. The researchers found that the deprivation of cysteine and methionine through a customized diet in animal models resulted in higher death rate of glioblastoma cells through gerroptosis. The diet also made chemotherapy drugs more effective at initiating programmed cell death. The animal models demonstrated improved survival after going on the diet. Their findings were published in Nature Communications.

BioSkryb Genomics Unveils New Findings Related to Breast Cancer Tumor Evolution

BioSkryb Genomics will share what it calls “groundbreaking research” related to single-cell analysis of breast cancer biopsies and the expansion of its ResolveDNA product line at the AACR 2023 Annual Meeting in Orlando. At AACR, BioSkryb will present new data on the benefits of ResolveOME in understanding the evolution of breast cancer in a way that has not been seen before. The two poster presentations will review over a dozen ductal cell carcinoma biopsies, surveying the impact of multiomic analysis at the single-cell level in nearly 1000 cells, and demonstrating how it enables copy number, structural variation, fusion detection, allele-specific expression, and standard variant identification.

People on the Move

Tune Therapeutics Strengthens Leadership Team with Two Additions

Durham-based Tune Therapeutics added two new members to its senior leadership team. Derek Jantz joins as chief scientific officer and Zachary Hale takes on the role of general counsel. Jantz comes to Tune with deep expertise in developing cell and gene therapeutic candidates through the use of innovative genomic technologies. Jantz co-founded Precision BioSciences in 2006 as co-inventor of a novel method for modifying the DNA-recognition properties of the I-Crel homing endonuclease. Hale joins the company after over a decade of specialized legal work with biotechnology startups. He previously served as Senior Vice President and Deputy General Counsel at Juno Therapeutics.

Alcami Appoints Vice President of Marketing

CDMO Alcami Corporation appointed Jessica Cao to Vice President of Marketing. Cao brings over 20 years of industry experience, with an extensive track record in developing successful marketing strategies and building brands in a B2B setting. Before joining Alcami, she was the Senior Vice President of Marketing at AeroSafe Global, a cold chain solution provider for biopharma companies. Prior to that, she spent almost a decade at Catalent Pharma Solutions, a global CDMO, where she held senior leadership roles in marketing, strategy, and business development. Early in her career, Caoheld marketing leadership roles at large life science companies, including Becton Dickinson, Baxter, and Bayer. 

EmVenio Research names new chief medical officer

Durham’s EmVenio Research appointed Mark McKenzie as its chief medical officer. In this role, McKenzie will lead and strategize new initiatives in the clinical and therapeutic areas. He will also provide medical and clinical oversight to EmVenio’s team of principal investigators involved in trials at community research sites globally. McKenzie most recently served as the vice president of clinical affairs at M-3 Wake Research. Prior to that, he was the regional medical director and principal investigator at ClinSearch.

Bioventus Announces CEO Leadership Change

Bioventus Inc. announced that Ken M. Reali stepped down as chief executive officer  and as a member of the board of directors. The board has initiated a comprehensive search process to identify a permanent CEO. Until a successor is named, the board has appointed Anthony Bihl, as interim CEO, effective immediately. Bihl is a seasoned executive with more than 35 years of proven industry experience. He formerly served as CEO and member of the board of managers of Bioventus LLC from 2013 until his retirement in 2020. Bihl currently serves as a board member for Sonendo Inc. and Spectral Medical Inc.

GeneCentric Therapeutics Appoints Brian Kelly as SVP Business Development

GeneCentric Therapeutics appointed Brian Kelly as Senior Vice President of Business Development. Kelly will be responsible for spearheading the company’s global business growth strategy, with a focus on diagnostic partnering and biopharmaceutical alliance programs and other corporate development initiatives to advance the Company’s strategic priorities. He most recently served as Vice President, Global Biopharma and Companion Diagnostics Strategy at Caris Life Sciences and Global Director, Diagnostic Partnering at Thermo Fisher Scientific. Prior to that, he held various clinical market development and commercial roles at Life Technologies, Pacific Biosciences and Affymetrix. Before his time in industry, he held research positions at Johns Hopkins University School of Medicine, where he focused on biomarker discovery in oncology.