Weekly Quick Hits (Research Triangle Park) – Week of March 6, 2023

By Alex Keown, Mark Terry, and Sarah Ellinwood
March 10, 2023

Funding and Collaborations

New Initiative Aims to Put NC State at Forefront of Viral Vector Manufacturing

A team of professors and researchers from N.C. State University launched the North Carolina Viral Vector Initiative in Research and Learning (NC-VVIRAL), which aims to address the challenges of viral vectors for drug development. The initiative will build partnerships between academia, industry and government to support biomanufacturing of viral vectors in North Carolina and beyond. NC-VVIRAL has already brought together expertise from N.C. State, Duke University, the University of North Carolina at Chapel Hill, and other schools around the state, as well as Merck and several more partners in the biopharmaceutical and life sciences industries, according to the announcement.  

Congruence Therapeutics Raises $65 Million in Series A Extension

Congruence Therapeutics, a Raleigh-based biotechnology company designing novel small molecules for diseases of protein misfolding, closed an extension to its Series A financing, bringing the total amount raised to over $65 million. This extension was led by new investor BDC Capital’s Thrive Venture Fund, with participation from current investors Amplitude Ventures, Fonds de solidarité FTQ, OrbiMed, Investissement Quebec, SilverArc and others, the company disclosed.

RedHill Biopharma Announces $6 Million Private Placement

RedHill Biopharma Ltd. announced an agreement with a single investor for the issuance of convertible promissory note of up to $6 million. The funding will be made in three tranches of $2 million, $1 million, and $3 million, respectively. RedHill intends to use the net proceeds from this placement for general working capital, acquisitions, research and development and general corporate purposes.

Women-owned biotech startup, Tiamat Sciences raises $2 Million

Biotechnology startup Tiamat Sciences announced a $2 million seed-extension financing round that was led by 8090 Industries. Harnessing the power of plants, Tiamat provides animal-free recombinant growth factors with a highly flexible technology, at a fraction of their cost and with a reduced carbon footprint. The new funds will accelerate product validation in the cellular agriculture and regenerative medicine markets and establish strategic partnerships with industry leaders.

In the Clinic

Milestone Pharmaceuticals Eyes NDA for Paroxysmal Supraventricular Tachycardia Drug

Milestone Pharmaceuticals Inc. announced significant progress in clinical and regulatory activities for etripamil, the company’s investigational calcium channel blocker administered by patients outside of the healthcare setting to treat paroxysmal supraventricular tachycardia (PSVT). Data from the completed NODE-303 open-label safety and RAPID extension studies will be included in the etripamil PSVT New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) which is expected in the third quarter of 2023. The company believes feedback received from the FDA supports and aligns with its proposed approach for the NDA.

vTv Takes Type 1 Diabetes Treatment Into Phase III Trials

North Carolina’s vTv Therapeutics will initiate the first of two planned Phase III trials of its adjunctive insulin therapy TTP399 for Type 1 diabetes, setting up potential approval of the medication if data is positive. The studies are expected to recruit approximately 1,000 patients and at least one of the studies will assess one year of treatment. The trials are expected to begin later this year. 

Precision Biosciences Advances ARCUS Gene Editing Program

Michael Amoroso, chief executive officer of Durham-based Precision BioSciences, anticipates 2023 will be an important year for the company. In Precision’s year-end financial report, the CEO said the company plans to file an Invesigational New Drug application for its first ARCUS nuclease for in vivo gene insertion. An In Vivo Gene Editing R&D Day is planned for later this year. Over the last year, Precision invested in the manufacturing optimization of its allogeneic CAR-T therapies. The company said that resulted in improved product candidate attributes in azer-cel, a potential first-in-class allogeneic CAR T treatment for patients who have relapsed following autologous CAR T treatment.

Areteia Therapeutics Initiates Phase III Trials of Dexpramipexole in Eosinophilic Asthma

Chapel Hill-based Areteia Therapeutics dosed the first patients in both the EXHALE-2 and EXHALE-4 Phase III trials investigating the efficacy and safety of oral dexpramipexole in severe eosinophilic asthma. A total of three clinical studies are planned as part of the overall Phase III development program. EXHALE-2 is a 52 week, randomized, double-blind, placebo-controlled global study, which will evaluate the efficacy of dexpramipexole in reducing severe asthma exacerbations as compared to placebo as its primary endpoint. EXHALE-4 is a 24 week, randomized, double-blind, placebo-controlled global study, which will evaluate change in lung function compared to placebo as its primary endpoint. An additional global Phase III study, EXHALE-3, is planned to similarly evaluate reduction in exacerbations as compared to placebo as its primary endpoint.

New Products

Genixus Expands Manufacturing Capacity to Meet Demand for KinetiX RTA Syringes

Genixus, a pharmaceutical company focused on transforming acute and critical care medicines, is finalizing the buildout of a new manufacturing site in Concord, N.C., complete with automated manufacturing and cutting-edge technology to support the KinetiX syringe platform and expanding portfolio. Production of the company’s first product, KinetiX™ RTA Propofol, began at the company’s headquarters in Kannapolis and will expand into the new Concord facility. Genixus also plans to triple its staff by the end of 2024 in line with the expansion, while holding an option for incremental space as demand unfolds.

Calyx Launches Simulation Service to Forecast and Optimize Clinical Trial Supplies

Calyx, the e-clinical and regulatory solutions and services provider, launched the Calyx Supply Simulation, a clinical trial supply forecasting service available through Calyx’s in-house expert statistical design and trial supplies consultants. The new service is designed to help sponsors improve clinical trial efficiencies by evaluating the amount of medication required to start and maintain clinical trial enrolment, predicting how long an existing amount of study medication will last, and determining the optimal site and depot buffer stock quantities required to ensure dispensation while reducing the cost and burden of excessive drug wastage, according to the Morrisville-based company. 

Verona Pharma Plans NDA for COPD Drug

Following the announcement of Phase III trial results Raleigh’s Verona Pharma called “transformational,” the company is planning to file a New Drug Application for ensifentrine as a potential treatment for COPD. “Supported by the groundbreaking results from our Phase 3 ENHANCE (“Ensifentrine as a Novel inHAled Nebulized COPD thErapy”) trials, we believe ensifentrine, if approved, has the potential to change the treatment paradigm for COPD,” Verona Chief Executive Officer David Zaccardelli said in a statement. The company expects to file the NDA in the third quarter of 2023. 

Research Roundup

Diabetes Diagnoses On the Rise, WFU Researchers Find

Researchers from the Wake Forest University School of Medicine found that rates of Type 1 and Type 2 diabetes continue to increase in children and young adults. Non-Hispanic Black and Hispanic children and young adults also had higher incidence rates of diabetes. The research team identified more than 18,000 children and young people from infants to 19 years of age with a physician diagnosis of Type 1 diabetes and more than 5,200 young people between the ages of 10 and 19 with Type 2 diabetes at five centers in the U.S. between 2002 and 2018. The annual incidence of Type 1 diabetes was 22.2 per 100,000 in 2017–18 and 17.9 per 100,000 for Type 2 diabetes. The study appears online in the current issue of The Lancet Diabetes & Endocrinology.

Regulatory and Advocacy

Novan Moves Forward with Unified Corporate Branding

Durham’s Novan, Inc. announced that its commercial subsidiary, EPI Health, will now unify under one brand name, Novan. In early 2022, Novan completed the acquisition of EPI Health, which gave the company the commercial infrastructure necessary to bring its lead asset berdazimer gel, 10.3% to market, if approved. The combined organization is a fully integrated pharmaceutical company with capabilities across drug development and commercialization with a proven product portfolio across multiple disease states within dermatology.

People on the Move

Elixia Taps Sam Searcy as Chief Commercial Officer

Sam Searcy joins Elixia, a site management organization, to oversee and increase business growth. Searcy will oversee activities related to marketing, enhance service offerings, expand Elixia’s national site network, maintain client customer service, strategize Elixia’s business growth and increase Elixia’s overall market share. Searcy will also manage Elixia’s RTP office and the N.C.-based operations team.

Former BDSI CEO Lands New Role at Corium Pharma

Mark Sirgo, the founder and former chief executive officer of Raleigh’s BioDelivery Sciences

International, Inc., will take on a new executive role at Corium Pharma Solutions in Michigan. Sirgo will be the new CEO of the contract development and manufacturing organization specializing in novel drug delivery technologies. BDSI was acquired in 2022 for about $604 million by Massachusetts-based Collegium Pharmaceutical, Inc. 

M3 Wake Research Adds Head of Phase I Studies

M3 Wake Research, a fully integrated network of clinical research sites, named Lucian Cappoli as Head of Early Development and Phase I Operations. As Head of Early Development and Phase I Operations, Cappoli will lead M3 Wake Research’s future growth initiatives for early phase research, including enhancing capabilities of the network’s Phase 1 research sites, and strategically expanding these early phase capabilities to additional sites in the network.