Dietrich Stephan, one of the pioneers in the field of precision medicine, was in process of raising venture funds for his gene-silencing company NeuBase Therapeutics when he was introduced to the team at the struggling eye drug developer Ohr Pharmaceutical $OHRP. Initial conversations commenced less than a month ago, and on Thursday, the two sides came to an agreement — NeuBase would catapult onto the Nasdaq via a reverse merger with Ohr, and propel the development of its rare genetic disease drugs.
NeuBase’s modular antisense peptide nucleic acid (PNA) platform — dubbed PATrOL — is designed to improve gene silencing therapies by fusing the advantages of synthetic approaches with the precision of antisense technologies, which inhibit the production of disease-causing proteins.
NeuBase’s technology has several advantages over traditional antisense oligonucleotides, according to Stephan, who currently serves as a professor of human genetics at the University of Pittsburgh and has founded and co-founded at least 13 companies, including NeuBase.
NeuBase’s technology allows for rapid drug design due to the modular nature of the PATrOL platform, selectivity for specific RNA/DNA sequences, the ability to penetrate the blood brain barrier, broad systemic distribution for multi-tissue disease and high cell permeability. Early in vivo data have also indicated no immune response and a low cost of goods, which could be essential for scalability in potentially addressing a wide range of genetic diseases, including cancer.
But at this point, NeuBase is still in the process of validating its approach and has much work ahead — it is still conducting preclinical studies, and expects to enter the clinic within the next few years.
“Ionis has generated awareness around the potential for antisense technologies, but we are confident that our PATrOL platform has significant advantages that will allow us to expand beyond the space that Ionis $IONS has created,” Stephan told Endpoints News.
The company plans to file an IND for a drug for Huntington’s disease in 2020 and one for myotonic dystrophy in 2021.
If the deal with Ohr is consummated, NeuBase has proposed trading under the symbol $NBSE and will retain the majority 80% stake of the combined company, while Ohr will get a 20% stake. Stephan will lead the combined company.
“Following a comprehensive review of strategic alternatives, Ohr’s Board of Directors concluded that the proposed transaction with NeuBase is in the best interest of our stockholders…we intend to hold a special meeting of Ohr shareholders in the first half of 2019 to vote on this merger,” Ohr CEO Jason Slakter said in a statement on Thursday.
Ohr’s shares $OHRP skyrocketed nearly 82% before the bell on the news.
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