Weekly Quick Hits (RTP) – Week of June 19, 2023
Funding, Awards and Collaborations
Marius Pharmaceuticals Supports AXYS 2023 Virtual Family Conference
Marius Pharmaceuticals, which specializes in therapies for hypogonadism or testosterone deficiency, announced its sponsorship of the AXYS 2023 Virtual Family Conference. This conference serves as a significant platform for individuals and families affected by XY Chromosome Variations, including conditions such as Klinefelter’s Syndrome. Kyzatrex (testosterone undecanoate), an oral medication developed by Marius Pharmaceuticals, has emerged as a viable treatment option for those with Klinefelter’s Syndrome. As the XY Chromosome Variations community seeks oral alternatives for testosterone replacement therapy (TRT), Marius Pharmaceuticals recognizes the significance of KYZATREX™ in meeting this demand.
Solve Therapeutics Acquires Duke University Spin-Out Cereius, Inc.
San Diego-based Solve Therapeutics will acquire Cereius, Inc., a private company developing innovative next-generation, targeted radiodiagnostics and radiotherapeutics for cancer patients. Cereius was co-founded by Professor Michael Zalutsky and Dr. Kimberly Lynn Blackwell around technology initially developed at Duke University and further advanced by the Cereius research team. SolveTx will continue to refine the Cereius chemistry platform while developing targeted radionuclide therapeutics and evaluating companion radiodiagnostics for use with other types of targeted cancer therapeutics. The company is building out a research and development team and is also investing in research facilities located at the recently completed Biolabs NC Gradlab building in downtown Durham, to support this development initiative.
restor3d to Acquire Conformis
Durham-based restor3d and Conformis, Inc. entered into a definitive merger agreement. restor3d, a leading personalized 3D-printed orthopedic company, will acquire all outstanding shares of common stock of Conformis at $2.27 per share in cash. The merger will create a leading personalized 3D-printed medical device company, company officials said. The transaction, which represents an approximate 96 percent premium to the closing price of Conformis stock on June 22, is expected to be finalized by the end of the third quarter of this year.
In the Clinic
Milestone Pharmaceuticals Publishes Phase III Data for Etripamil Nasal Spray
Milestone Pharmaceuticals published results from the Phase III RAPID clinical trial evaluating etripamil nasal spray, an investigational calcium channel blocker, in patients with paroxysmal supraventricular tachycardia (PSVT) in The Lancet. Key findings in the article support the potential self-administration of etripamil outside of the healthcare setting. The findings include:
- The RAPID trial achieved its primary endpoint, with patients taking etripamil demonstrating a highly statistically significant and clinically meaningful difference in time to supraventricular tachycardia conversion compared to placebo.
- Significant reductions in time to conversion in patients who took etripamil were evident early and were durable, persisting throughout the observation window of the study compared to placebo.
- Data demonstrated statistically significant improvement in multiple defined symptoms of PSVT in patients receiving etripamil compared to placebo.
- Reduction in emergency-department visits and, for example, need for intravenous treatments, was observed, though not with statistical significance in this dataset alone.
New Products
CMP Pharma Announces that Liqrev is Now Available
CMP Pharma announced that Liqrev (Sildenafil) Oral Suspension, 10 mg/mL, the only FDA-approved ready-made oral liquid of sildenafil, is now available. Liqrev is a liquid oral suspension of sildenafil formulated for patients with dysphagia or difficulty swallowing tablets. Liqrev is indicated for the treatment of pulmonary arterial hypertension to improve exercise ability and delay clinical worsening. Liqrev addresses the inconsistencies that come from unapproved crushed or compounded tablets and removes the need for complex preparation of powder formulations. Liqrev has a strawberry flavor and is available in 122 mL bottles with a 24-month shelf life.
Research Roundup
New Research Shows HIV Can Lie Dormant in the Brain
HIV-infected immune cells can transition into a dormant state that ultimately protects them from current antiretroviral therapies. If treatment is discontinued, the virus can rebound from latency and reignite the progression of HIV infection to AIDS. Scientists from the HIV Cure Center at the UNC School of Medicine, University of California San Diego, Emory University, and University of Pennsylvania have been searching for where exactly these latent cells are hiding in the body. New research published in the Journal of Clinical Investigations confirms that microglial cells – which are specialized immune cells with a decade-long lifespan in the brain – can serve as a stable viral reservoir for latent HIV.
Precision BioSciences to Present Positive ARCUS Gene Editing Data
Precision BioSciences, which is developing ARCUS-based in vivo gene editing and ex vivo allogeneic CAR T therapies, presented positive preclinical data from its PBGENE-HBV development program for the treatment of patients with chronic hepatitis B during an oral presentation at the European Association for Study of the Liver (EASL) Congress 2023 in Vienna, Austria. There is no cure for chronic hepatitis B and current treatments rarely result in functional cure, primarily due to persistence of viral DNA in the liver. HBV inserts its DNA into the human genome of infected liver cells. This integrated HBV DNA produces the viral protein, hepatitis B surface antigen (HBsAg), which is secreted in the blood. Presence of HBsAg is associated with poorer outcomes and elimination of HBsAg is necessary for functional cure of chronic hepatitis B. Using ARCUS, company scientists generated a highly specific and effective nuclease to eradicate chronic HBV infection. PBGENE-HBV is designed to inactivate cccDNA with direct cuts and edits as well as to inactivate integrated HBV DNA leading to long lasting reductions in HBsAg. In an episomal adeno-associated virus mouse model, company researchers demonstrated that administration of lipid nanoparticles containing mRNA encoding an HBV-targeted ARCUS nuclease resulted in a 96% reduction in serum HBsAg. In a follow-on experiment, treatment of HBV-infected primary human hepatocytes with the HBV-targeted ARCUS nuclease resulted in a 90% reduction of covalently closed circular DNA (cccDNA) and high specificity.
Is the U.S. Ready For An African Swine Fever Outbreak?
In a new study, researchers from North Carolina State University used a computer model to understand how African swine fever (ASF) might spread among swine farms in the southeastern U.S. – and examined the effectiveness of existing response plans. They found that although control actions would help, an outbreak could still prove persistent and costly. ASF is a highly contagious viral disease in pigs, with a mortality rate that can reach 100%. The cost of a potential ASF outbreak in the U.S. has been estimated at $80 billion. The researchers used an epidemiological model, called PigSpread, to look at the different ways a potential outbreak in the southeastern U.S. could unfold. The PigSpread model evaluates six transmission routes including between-farm swine movements, vehicle movements, and local spread, to model the dissemination of ASF. Between-farm movements accounted for 71% of disease transmissions in the model, with local spread and vehicular transmission both contributing about 14% each.
Real Estate
Azzur Group To Celebrate Opening of Azzur Cleanrooms on Demand in Raleigh
Azzur Cleanrooms on Demand, a provider for early-phase life sciences GxP manufacturing, will host a Grand Opening and Ribbon Cutting Ceremony for its latest facility in Morrisville on June 27. Azzur’s Cleanrooms on Demand model provides turnkey GMP support to the pharmaceutical,biotechnology and medical device industries with on-demand cleanroom facilities, materials management, storage, asset management, and supply chain solutions. This latest addition to the Azzur Cleanrooms on Demand portfolio has 24 cleanrooms along with labs, storage space, both ambient and controlled temperatures and 24 office spaces all for client use. By 2024, the facility will provide expanded services for cell and gene therapy manufacturing, with state-of-the-art equipment. The facility will create approximately 60 jobs, including staff positions and support services.
People on the Move
Clinical ink Appoints Andrew Kraus as Chief Technology Officer
Clinical ink, a global life science technology company, announced the appointment of Andrew Kraus as Chief Technology Officer. Kraus will lead the development of the company’s expanding technology portfolio to support clinical trials. Prior to Clinical ink, Kraus served as Chief Operating Officer for World Care Clinical, a CRO providing centralized imaging and other services supporting clinical trials. Previously, Kraus held executive roles at several companies leading clinical technology and operations. These included experience as Chief Operating Officer at Bioclinica; SVP Service, Strategy and Operations at Signant; and Chief Operating Officer and Treasurer for the Cardiovascular Research Foundation. He currently sits on the Board of Directors for Algorics, a specialized clinical data services company.
Polarean Imaging Names New CEO
Polarean Imaging plc appointed Christopher von Jako as Chief Executive Officer and director of the company. von Jako succeeds Richard Hullihen, CEO since 2017, will be retiring and will be stepping down as a director of the company, effective immediately. In his most recent executive position, von Jako served as president and CEO of BrainsWay from 2020 to 2023. Prior to this, he was CEO of Dynatronics. Before Dynatronics, von Jako served as president and CEO of NinePoint Medical, Inc. He also served as president and CEO of NeuroTherm, Inc., a pain management business until its acquisition by St. Jude Medical Corporation.
