Single Dose Of NeuBase's Lead Program Shows Encouraging Preclinical Studies

NeuBase Therapeutics Inc NBSE announced new preclinical data for its lead development candidate, NT-0231.F, to treat myotonic dystrophy type 1 (DM1), a multisystem disorder that affects skeletal and smooth muscle.
A single intramuscular dose confirmed that NT-0231.F is pharmacologically active in the muscle and drives molecular and functional rescue in an animal model and myotonia (delayed muscle relaxation after contraction) reversal.
A single intravenous (IV) dose of NT-0231.F provides initial splice rescue at around two weeks, with significant splice rescue around three weeks.
Myotonia reversal was achieved at around four weeks, with effects enduring at least six weeks.
A time course of multiple subcutaneous (SC) doses across increasing concentrations of NT-0231.F was also investigated and showed splice rescue and myotonia reversal in a dose-responsive manner, illustrating the feasibility of the differentiated and patient-friendly SC route.
In pharmacokinetic studies of NT-0231.F in wild-type BALB/C mice, a single IV or SC dose showed a high volume of distribution, suggesting wide tissue distribution.
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