Weekly Quick Hits (Research Triangle Park) – Week of September 11, 2023

It has been a busy week for RTP life sciences companies heading into the last part of the year. TARGAN opened a new manufacturing facility in a converted 100,000 square-foot former grocery store. The National Sciences Foundation backed N.C. A&T’s quantum research programs with $5 million and the N.C. Board of Science, Technology & Innovation provided funding for dozens of small life sciences companies in RTP and across the state.

By Alex Keown | September 15, 2023

Quick Hits is BioBuzz’s weekly round up of all the life science news you don’t want to miss, in your region of interest. Don’t miss a beat; SUBSCRIBE to our weekly Quick Hits newsletter via LinkedIn (BioHealth Capital Region, Greater Philadelphia, Research Triangle Park) or get it delivered to your inbox every week.

Funding, Awards and Collaborations 

65 North Carolina Companies Awarded Grants for Innovative Research and Development

The North Carolina Board of Science, Technology & Innovation announced that 65 innovative small businesses in North Carolina have received 73 grants to develop and commercialize groundbreaking new technologies with the potential for commercialization. During the fiscal year, the One North Carolina Small Business Program awarded nearly $2.05 million to pioneering small businesses in 19 counties across the state. The full list of One North Carolina Small Business Program awardees can be reviewed at the North Carolina Department of Commerce website.

Polarean Partners with VIDA to Streamline Adoption of Advanced MRI of the Lungs

Durham-based Polarean has partnered with VIDA Diagnostics, a clinical imaging intelligence company, to develop solutions that further enable the Polarean xenon 129 MRI platform to accelerate clinical and research use. The collaboration between Polarean and VIDA intends to support the integration of xenon 129 MRI workflow into the clinical continuum of care, establish a clinical trial network for pharma-sponsored drug and device development using standardized xenon 129 MR image acquisition and data-sharing methods, and create automated image processing workflows and algorithms. These new products and services will be utilized to expand access to xenon 129 MRI as a lung imaging service and to investigate new indications and biomarkers in broader populations with unmet medical needs.

Sparta Biomedical Closes Oversubscribed Convertible Round to Advance Development

Privately-held Sparta Biomedical closed a heavily oversubscribed convertible funding round. The capital will accelerate Sparta’s preparations for the Investigational Device Exemption study preparations of its breakthrough device, Ormi. Ormi, which is in development to treat knee osteoarthritis, was granted Breakthrough Device Designation by the FDA. New medtech and angel investors supported Sparta’s funding. 

Syneos Health and Oracle Expand Relationship to Accelerate Patient Recruitment

Syneos Health announced a strategic collaboration expanding its relationship with Oracle. Using the Oracle Cerner Learning Health Network (LHN) and elements of Oracle’s suite of study startup solutions, the companies seek to help reduce the time it takes to recruit patients for clinical studies and increase the diversity of patient populations participating in medical research. Through its collaboration with the Oracle Cerner LHN, Syneos Health’s objective is to improve performance throughout the entire clinical trial lifecycle, with a strong emphasis on clinical trial enrollment to help close diversity gaps in patient recruitment.

Science 37 Named Market Leader for Global Decentralized Clinical Trials

Science 37 Holdings was tapped for the prestigious Frost & Sullivan Market Leadership Award in the global decentralized clinical trials industry. This acknowledgment solidifies the company’s role as an industry leader as it continues to transform clinical trial conduct through the Metasite. Frost & Sullivan’s Best Practices Market Leadership Award is bestowed upon companies that exhibit outstanding performance and visionary approaches in their respective industries. The receipt of this prestigious award highlights Science 37’s dedication to accelerating clinical research by enabling universal trial access for patients, the company said. 

Dignify Therapeutics and Aayam Therapeutics Enter into Licensing Agreement

Dignify Therapeutics, which focuses on restoring bowel and bladder control for elderly and neurologically impaired individuals, entered into a licensing agreement with California-based Aayam Therapeutics to develop compound ATX-003 and related proprietary molecules for on-demand defecation. The agreement expands Dignify’s intellectual property portfolio. Financial details were not disclosed. 

National Science Foundation Supports N.C. A&T’S Quantum Research with $5 Million

North Carolina Agricultural and Technical State University was awarded a five-year, $5 million grant from the National Science Foundation to support the ExpandQISE: Track 2: N.C. A&T QISE Research Workforce Development Programs. NSF developed the ExpandQISE program through the National Quantum Initiative Act, passed in 2018, to lower barriers to access and broaden the diversity of participating institutions.

Grant Avenue Capital Invests in Life Sciences and Healthcare Consultancy CREO

Grant Avenue Capital and Durham-based CREO, Inc., announced a recapitalization of CREO. Terms of the transaction were not disclosed. Founded in 2015, CREO aims to advance innovation in human health by providing holistic solutions to its life science and healthcare company customers that help them reach their full potential and overcome multi-faceted industry challenges.

BioSkryb Genomics Joins NIST Genome Editing Consortium

Durham-based BioSkryb Genomics joined the NIST Genome Editing Consortium to help address the measurements and standards needed to increase confidence in utilizing genome editing technologies in research and commercial products. The goal of the consortium is to establish greater confidence in the characterization of genome editing outputs through the evaluation of genome editing assay pipelines, generation of benchmark data and more.

TARGAN Opens New Facility in Raleigh

Biosystems company TARGAN, based in Morrisville, opened its new facility called the Midtown BioCenter. It’s the first life science-focused facility in the Midtown Raleigh area, WRALTechwire reported. The new 100,000 square-foot space is in a building that previously housed a grocery store.

In the Clinic

Parion Sciences Published CLEAN-PCD Study in The Lancet Respiratory Medicine

Parion Sciences, which is based in Durham, announced the publication of positive Phase II data from its study of idrevlodried in hypertonic saline in people with primary ciliary dyskinesia in The Lancet Respiratory Medicine. In the CLEAN-PCD study, idrevloride in hypertonic saline was safe and associated with significant improvement in lung function over 28 days in people with primary ciliary dyskinesia compared with hypertonic saline alone. Results of the study underscore the potential of idrevloride Inhalation Solution to be a safe and effective nebulized treatment for people living with primary ciliary dyskinesia.  

Sparta Biomedical’s Breakthrough Device to be Featured at the ICRS World Congress

Privately-held Sparta Biomedical, a Morrisville company specializing in partial knee resurfacing, will share preclinical data for Ormi at the 2023 ICRS World Congress in Barcelona, Spain. This will be a unique opportunity for conference attendees to hear firsthand about Ormi’s potential to redefine partial knee resurfacing. Ormi, which is in development to treat knee osteoarthritis, was granted Breakthrough Device Designation by the FDA.

Humacyte Sees Positive Data in Mayo Clinic Study of HAV for Chronic Limb Ischemia

A Mayo Clinic study assessing Humacyte’s Human Acellular Vessel treatment in patients with chronic limb-threatening ischemia revealed the treatment was a safe, resilient, and effective conduit for arterial bypass and limb salvage. This is an important result since approximately 40% of patients requiring lower extremity bypass do not have saphenous veins available, which is the standard of care for treating this challenging disease state. The data was presented at the Midwestern Vascular Conference in Minneapolis, MN, entitled Outcomes of Arterial Bypass Using the Human Acellular Vessel (HAV) In Patients With Chronic Limb Threatening Ischemia.

Humacyte Posts Positive Results from Phase II/III Trial for Patients with Vascular Trauma

In its second clinical announcement this week, Humacyte announced positive top-line results from its V005 Phase II/III trial of the Human Acellular Vessel (HAV) in vascular trauma repair. The single-arm clinical trial was a success and showed that the HAV in this study had higher rates of patency, and lower rates of amputation and infection, compared to historic synthetic graft benchmarks. Humacyte plans to file a Biologics License Application (BLA) for the treatment of vascular trauma with the FDA during the 4th quarter of 2023.

Aerami Therapeutics Presents Phase I Data for Lead Program AER-901 at Conference

Aerami Therapeutics presented results from its Phase I clinical trial assessing AER-901 at the 2023 European Respiratory Society (ERS) International Congress. AER-901, a proprietary liquid formulation of imatinib for inhalation delivered by a breath-activated, high-performance, handheld, smart nebulizer, is in development to address the unmet medical need for people with serious and rare forms of pulmonary hypertension, including the lead indication, pulmonary hypertension associated with interstitial lung disease, as well as pulmonary arterial hypertension. In animal models of pulmonary hypertension, imatinib reversed the proliferative, fibrotic, and inflammatory pulmonary vascular remodeling process believed to drive symptom severity, disease progression, and outcomes in many forms of the disease. 

New Products

KBI Biopharma Launches SUREmAb for Faster Monoclonal Antibody Development

Durham-based KBI Biopharma launched SUREmAb, an offering built on the robustness of KBI’s SUREtechnology Platform, that allows for optimized, safe, and cost-efficient monoclonal antibody (mAb) development and manufacturing. SUREmAb is a vertically integrated global offering designed to minimize development timelines for global biopharmaceutical manufacturers, delivering research cell bank development in as little as 9 weeks and the ability for customers to move from DNA to GMP drug substance in as few as 11 months. 

Research Roundup

Duke: Concussions Cause Greater Cognitive Declines in Later Life

A study in twins found that even a single traumatic brain injury at any age was associated with worse cognitive function in later life. This was independent of genes and environmental factors, according to the report. The study, led by Duke Health researchers, offers further evidence that traumatic brain injuries such as concussions can lead to a faster rate of cognitive decline later in life, particularly if the injury occurs after the age of 24, or results in a loss of consciousness. The findings appear online in the journal Neurology. Read more about the study here.

N.C. State: Study Sheds Light on Increased Reports of Suicidal Behavior in Teens

Reports of increasing suicidal behaviors in children in the decade leading up to the COVID-19 pandemic suggest there was already a mental health crisis among teens and children. However, an N.C. State-led analysis of data from New Jersey, suggests some of this increase is due to changes in how health professionals screen for and report on suicidal thoughts or ideas in young people. The researchers looked at data on all hospital visits of children ages 10-18 in New Jersey from 2008 through 2019. Read more here.

UNC: Sickle Cell Disease Continues to Face Underfunding, Lack of Research

UNC School of Medicine researchers detail the current struggles and challenges facing patients with sickle cell disease, including inadequate funding for sickle cell programs, lack of research, and limited access to healthcare. Of the “scant research being conducted on sickle cell disease,” much is concentrated on curative treatments like gene therapy and stem cell therapy. These therapies, although promising and ingenious, often involve long hospital stays and incur harsh side effects that may hinder young patient’s social lives and quality of life, the researchers said. Read more here.

Duke Center for Precision Health, Aims to Transform Population Health, Patient Care

The Duke Clinical and Translational Science Institute (CTSI) announced the launch of the Center for Precision Health (CPH), a collaboration that will harness the power of genomic, biomarker, and health data to transform patient care and population health. With continuing advances in genomics, biomarker technology, and computational biology alongside improvements in electronic health records and machine learning, the CPH will work to bridge discovery science with personalized patient care.  

On the Hill – Regulatory and Advocacy

FDA Accepts Verona Pharma’s New Drug Application Filing for Ensifentrine

Verona Pharma announced the U.S. Food and Drug Administration accepted for review the company’s New Drug Application seeking approval of ensifentrine for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD). The FDA assigned a Prescription Drug User Fee Act target action date of June 26, 2024. The FDA is not currently planning to hold an advisory committee meeting to discuss the application. The NDA included efficacy and safety data from Verona Pharma’s Phase III ENHANCE trials, in which ensifentrine demonstrated improvements in lung function and symptom endpoints and substantially reduced the rate and risk of COPD exacerbations. Ensifentrine was well-tolerated in a broad population of subjects with moderate to severe COPD.

People on the Move

Precise Bio Appoints Ori Hadomi as Chairman of the Board of Directors

Precise Bio, a pioneering regenerative medicine company with a large presence in N.C., announced the appointment of Ori Hadomi as its new Chairman of the Board of Directors. With over three decades of leadership experience in the medical device sector, Hadomi has excelled in various senior executive roles across multinational corporations and innovative MedTech startups. He currently holds the position of Vice President for Strategic Initiatives & Partnerships at Medtronic, after serving for a notable period of 17-years as CEO of Mazor Robotics. Mr. Hadomi is also actively engaged, in various capacities, with other prominent medical device companies.

Evecxia Therapeutics Announces CEO Transition

Evecxia Therapeutics announced that Dr. Jacob Jacobsen has been appointed President and CEO. Jacobsen succeeds Joseph Patti, who has stepped down from his role as President and CEO of Evecxia to pursue other interests. Jacobsen is the inventor of the serotonin synthesis amplification therapeutic concept the company uses to treat brain disorders. Prior to joining Evecxia, he was a scientist at Duke University and Duke-National University Singapore. While at Duke, he co-founded Evecxia with the late James B Duke Professor, Dr. Marc Caron.