Merck and Orna Therapeutics, a biotechnology company pioneering novel circular RNA (oRNA) technology, today announced a collaboration agreement to discover, develop, and commercialize multiple programs, including vaccines and infectious disease and oncology therapeutics. The company reports that, compared to linear mRNA, oRNA has key advantages, such as simplified production, increased protein expression, and a superior immunogenicity profile.
Orna also announced the initial closing of its $221 million Series B financing. Merck participated as a new investor in the financing, with $100 million, alongside founding investors MPM Capital and BioImpact Capital, an affiliate of MPM, among others. Orna was created in 2019 by MPM Capital and BioImpact Capital, with funding from the UBS Oncology Impact Fund.
“This broad strategic collaboration brings together Merck’s significant expertise in nucleic acid biology, clinical development, and manufacturing with Orna’s compelling circular RNA technology to explore the opportunity to develop a new generation of potential vaccines and therapeutics,” said Fiona Marshall, senior vice president and head of discovery, preclinical and translational medicine at Merck Research Laboratories.
Merck has been late to the mRNA game, although in 2018 it expanded an earlier collaboration with Moderna to develop and commercialize personalized mRNA-based cancer vaccines. That started with four collaborations but only one remains: mRNA-4157 as a monotherapy and in combination with anti-PD-1 therapy KEYTRUDA (pembrolizumab) in Phase II for unresectable solid tumors.
Under the terms of this collaboration with Orna, Merck will make an upfront payment of $150 million, then Orna will be eligible to receive up to $3.5 billion in development, regulatory, and sales milestones associated with the progress of the multiple vaccine and therapeutic programs, as well as royalties on any approved products. Orna will retain rights to its oRNA-LNP (lipid nanoparticle) technology platform and will advance its own programs in areas such as oncology and genetic disease.
The financing will enable Orna to continue development of its circular RNA + LNP delivery platform through its joint venture with ReNAgade Therapeutics, an RNA delivery company. Orna recently reported their lead in situCAR (isCAR) program demonstrated tumor suppression and eradication in animal models. Orna anticipates advancing this program into clinical trials in 2024.
“In under three years, Orna has taken proprietary circular RNA from academic literature to the first proof of concept data in preclinical models,” said Tom Barnes, PhD, Chief Executive Officer of Orna and Entrepreneur at BioImpact Capital.
The proprietary oRNA technology creates circular RNAs (oRNAs) from linear RNAs by self-circularization. These oRNA molecules have been shown to have greater stability in vivo than linear mRNA and have the potential to produce larger quantities of therapeutic proteins inside the body. Newly synthesized oRNA molecules are also more compactly packaged into custom lipid nanoparticles (LNPs), which Orna has engineered to target key tissues in the body. Promising preclinical data was presented at the 2022 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting.
“We are thrilled to collaborate with Merck, a company committed to breakthrough science, which has recognized the potential our platform can bring to patients. Our oRNA technology plus novel delivery solutions are designed to unlock the full potential of RNA in therapeutics and vaccines,” said Tom Barnes, Chief Executive Officer of Orna.
Orna Therapeutics was founded in 2019 based on research by Alex Wesselhoeft, Ph.D., and Daniel G. Anderson, Ph.D., from MIT.