BTIG initiated coverage of NeuBase Therapeutics (NASDAQ:NBSE) with a “buy” rating and $15 price target. The stock closed at $6.55 on Sept. 23.
NeuBase has developed a modular antisense peptide nucleic acid (PNA) platform, with the capability to address rare genetic diseases caused by mutant proteins.
“While very early, we see parts of the NeuBase platform as risk-mitigated and collectively contributing to a next-generation iteration of antisense oligonucleotides (ASOs), known as PNAs, targeting meaningful improvements on the ASO approach already with proven life-changing capabilities,” writes analyst Thomas Shrader.
He said NeuBase’s PNAs take two basic forms: linear molecules that modulate gene function, much like ASOs, and more complex in vivo-formed repetitive PNAs that use bifacial bases to simultaneously engage two strands of a structured RNA.
The PNA platform has been around for decades, but NeuBase has both optimized the target recognition and cell permeability, he added.
Mr. Shrader said the first clinical studies in Huntington’s disease and myotonic dystrophy 1 are slated to begin in 2021 and, “perhaps more importantly for such a novel platform, the first animal data are expected within the next six months.”