Weekly Quick Hits (Research Triangle Park) – Week of March 27, 2023

By Alex Keown, Mark Terry, and Sarah Ellinwood

Funding, Awards and Collaborations

9 Meters Initiates Cost Reduction Plan

Despite a $5 million infusion of cash, the 9 Meters Biopharma Board of Directors approved a cost reduction plan that will extend the company’s cash runway. The plan will reduce operating expenses and further align 9 Meter’s workforce with immediate business needs, the company announced in its most recent financial report. The reduction plan comes as Durham-based 9 Meters prepres for a pre-IND meeting with the FDA that is expected to provide the Company with important regulatory guidance on the preclinical development plan to support an IND for NM-136 in obesity disorders.

Milestone Pharmaceuticals Raises $125 Million to Advancement Etripamil for PSVT

Milestone Pharmaceuticals Inc. struck a $125 million strategic funding agreement with existing shareholder, RTW Investments, LP and some affiliates. Proceeds from this investment will be combined with $64.6 million of existing cash to support  the potential approval and launch of etripamil, the company’s investigational calcium channel blocker envisioned to empower patients with paroxysmal supraventricular tachycardia (PSVT) to treat their attacks outside of the emergency department.

Bayer Strikes New Collaboration With Oerth Bio To Advance Crop Protection Innovations

Bayer and Durham-based agricultural biotech company Oerth Bio announced a new collaboration to develop the next generation of more sustainable crop protection products. The unique protein degradation technology used by Oerth Bio has the potential to generate products that support Bayer’s agricultural sustainability objective. Initially developed to fight human diseases like cancer and other difficult to treat diseases, Oerth’s patented PROTAC (PROteolysis TArgeting Chimera) protein degradation technology provides an innovative pathway to entirely novel crop protection and climate resilient farm solutions.

Jigsawdio Receives NIH Grant for Audiovisual Jigsaw Puzzle Device

Jigsawdio, a woman-owned small business headquartered in Raleigh, received a $500,000 Science and Technology Transfer (STTR) grant from the National Institutes of Health to fund the prototype development and use study of its audiovisual jigsaw puzzle device created specifically for the Alzheimer’s and dementia populations. TheJigsawdio prototypes currently include 12-, 24-, and 36-piece puzzle options, which will be completed by April. 

In the Clinic

AskBio to Present Phase Ib Results for Parkinson’s Disease Gene Therapy 

Asklepios BioPharmaceutical, Inc. (AskBio) will present preliminary results of a clinical Phase Ib study investigating the safety and efficacy a gene therapy for the treatment of mild to moderate Parkinson’s disease at the AD/PD 2023 Advances in Science & Therapy Conference. AskBio’s AB-1005 is a an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy. Initial results from the open-label study will be presented during an onsite oral session. Preliminary data suggest that both the gene therapy and the neurosurgical administration are generally safe, well tolerated and associated with enhanced mean putaminal coverage and clinical improvements. The study included 11 participants who had completed 9–18 months of clinical follow-up post treatment, of which 10 had completed more than 12 months.

Validation of a Pragmatic Clinical Risk-Based Classification of NASH

New research published today by Durham-based Target RWE validates a practical risk-based classification of nonalcoholic fatty liver disease (NAFLD). The article “Validation of a Clinical Risk-Based Classification System in a Large Nonalcoholic Fatty Liver Disease Real-world Cohort,” was published in Clinical Gastroenterology and Hepatology. The research addresses an unmet need for simple and easily available noninvasive methods that can be used in routine practice to identify those at risk of adverse outcomes from NAFLD.

New Products

Yunu Opens Yunuverse, Increasing Trial Access for CRO Customers

Cary-based Yunu, a clinical trials imaging assessment workflow and data management platform, opened access to their new Yunuverse for collaboration among pharma sponsors, trial sites, clinical research organizations and on-demand clinician groups to transform the way imaging-based trials are conducted. These new Yunu platform capabilities, called the Yunuverse, create an expansive network allowing stakeholders to invite new participants into their trials. Clinical research coordinators and administrative staff can choose local or offsite resources and make these selections on a trial-by-trial or individual study basis, with the freedom to adjust these preferences at any time.

Pro-ficiency Releases Simulation-Based Good Clinical Practice Training Program

Durham-based Pro-ficiency launched a new Good Clinical Practice (GCP) Simulation training program. The GCP Simulation program immerses learners in an adaptive learning environment, building critical thinking and decision-making skills needed to apply GCP principles in the real world. 

Research Roundup

Duke Study Reveals How UV Radiation May Drive Melanoma

Researchers from Duke University used an in-house-developed method called UV Bind to examine how transcription factors bind to DNA following exposure to UV radiation. The team found that binding doesn’t follow the exact same patterns that it does for non-irradiated DNA. Testing revealed patterns that indicate potential for transcription factors to rearrange where they go in the genome after UV radiation exposure.  

Gut Microbiome May Hold the Key to Healthy Aging: Duke Research

A Duke University researcher hopes to identify novel mechanisms of gut-host interactions and help develop therapeutics to treat age-related declines in health. The research hopes to build on previous studies that showed gut microbiome pattern differences in older adults who are lean and physically active compared to their less-fit peers. Other research connected early frailty to reduced gut microbial diversity. Some animal studies showed microbiota transplantation from a younger to an older host “acts as a reset button for aging mice, increasing lifespan, and changing metabolism.”

Metabolon’s Metabolomic Platform, Services Cited in 3,000+ Peer-Reviewed Articles

Morrisville-based Metabolon, Inc. announced its metabolomics platform outputs and related services have been cited in more than 3,000 peer-reviewed journals. Publications referencing Metabolon, its platform and results have appeared in world-renowned, high-impact journals such as Nature, Nature Medicine, Nature Genetics, Cell, Proceedings of the National Academy of Sciences (PNAS), and many others, demonstrating the dramatic increase in awareness and recognition of the importance of metabolomics in research.

Regulatory and Advocacy

Renovion Receives FDA Fast Track Designation for ARINA-1 

The FDA granted Fast Track designation to Renovion Inc.’s experimental asset ARINA-1, which is being developed for the prevention of bronchiolitis obliterans syndrome (BOS) progression in adults with a bilateral lung transplant. Renovion will use the rolling submission of the Fast Track designation to expedite NDA submission based on the results from the ALOFT study to help people with early BOS who are in dire need of safe and effective therapies to prevent BOS progression and further lung function decline.

Ad Astra Diagnostics files 510(k) for QScout hematology analyzer

Morrisville-based Ad Astra Diagnostics announced that it has submitted a 510(k) application to the FDA for its QScout RLD rapid-result hematology system intended to report white blood cell count, neutrophil-to-lymphocyte ratio, and counts of six white blood cell types in fingerstick or venous blood. QScout is the first hematology platform designed for simple, fast operation at point of care to provide measures of disease and infection including a ratio of two white blood cell types and count of immature granulocytes. 

People on the Move

James Borneman Joins Bryn Pharma as Chief Operating Officer

Sanofi veteran James “Jim” Borneman has been named chief operating officer of Raleigh-based Bryn Pharma. Boreman will be responsible for day-to-day operational oversight as Bryn prepares for the U.S. Food and Drug Administration approval and market launch of its innovative intranasal spray Utuly, an investigational self-administered form of epinephrine being studied for the treatment of type 1 allergic reactions, including anaphylaxis. For the past 20 years, Boreman held roles of increasing responsibility with Sanofi.