NBSE

NeuBase Therapeutics, Inc. (Nasdaq: NBSE) is a biotechnology platform company Drugging the Genome™ to address disease at the base level using a new class of precision genetic medicines. As Founder, Chief Executive Officer, and Chairman of NeuBase, Dietrich A. Stephan, Ph.D., stated recently,  “NeuBase is focused on significantly reducing the burden of untreatable morbidity and mortality caused by rare and common diseases across the globe. To achieve this goal, we designed, built, and validated a new precision genetic medicines platform technology that can uniquely drug the double-stranded human genome and address disease at the root of causality without many of the limitations of early precision genetic medicine technologies. We are poised to file our first Investigational New Drug (‘IND’) applications with the U.S. Food and Drug Administration (‘FDA’) beginning in calendar year 2022 and intend to scale into additional indications with increasing speed and efficiency thereafter.”

In the past year, NeuBase has confirmed that they have validated the ability of their technology in proof-of-concept studies to directly drug the double-helix of the human genome, including difficult double-stranded structures of RNA targets, and engage with mutant genes to resolve most causal mechanisms of disease. The validation of their platform’s capabilities included data describing that they have overcome many limitations of early precision genetic medicine technologies, such as biodistribution, tolerability, selectivity, manufacturability, durability, and scalability. They also presented data that their delivery shuttle enables compounds to elicit pharmacologic effects in multiple tissues, including in the brain and muscle, after subcutaneous administration in preclinical animal models.

They recently nominated the development candidate for their myotonic dystrophy type 1 (DM1) program, which they believe has the potential to be a best-in-class therapy that offers a patient-friendly route of administration, a whole-body solution for the muscle, heart, and brain manifestations of the disease. Furthermore, the mechanism of action of their development candidate is designed to engage with the toxic RNA hairpin structure to release the splicing proteins, restoring normal RNA splicing and downstream protein production, including DMPK. They have initiated IND-enabling studies for this candidate, with data read-outs expected across CY2022. They expect these data will support the submission of an IND filing to the FDA in the fourth quarter of CY2022. 

As a result of the nomination of their DM1 program candidate, NeuBase has established CMC expertise at their new facility in Cambridge, Massachusetts that is co-located with their clinical development team, finalized the formulation of their development candidate to enable systemic routes, and completed process development. They also scaled-up manufacturing in-house and with contract manufacturing partners to support non-clinical toxicology, product stability, and Phase 1/2 clinical trials. 

In parallel, wNeuBase has stated that they are making significant progress in their therapeutic program for Huntington’s disease. For example, they have illustrated with preclinical in vivo data that our proprietary delivery technology allows  their genome-targeting compounds to advance beyond intrathecal delivery and enabling a systemically administered allele-selective therapy, overcoming challenges seen with other programs. Furthermore, preclinical data show that their PATrOL™-enabled compounds can silence activating KRAS point mutations in vivo to inhibit protein production, which has the potential to target G12D and G12V, the two most common and historically ‘undruggable’ cancer-driving point mutations that represent the majority of KRAS tumors. NeuBase believes these data set the stage for a potentially first-in-class precision genetic medicine approach for oncology capable of selectively targeting mutations at the single-base level.