Neubase Therapeutics Inc Stock Price, News & Analysis

NeuBase Therapeutics (Nasdaq: NBSE) announced significant progress in its myotonic dystrophy type 1 (DM1) development program, presenting new preclinical pharmacokinetics and biodistribution data for its candidate NT-0231.F at the ASGCT 25th Annual Meeting. Following a single IV injection in mice, NT-0231.F exhibited rapid clearance and extensive tissue distribution, suggesting potential for whole-body therapeutic effects. This promising data supports NeuBase's aim to address the complex symptoms of DM1, including cognitive and muscular impairments.

NeuBase Therapeutics (Nasdaq: NBSE) reported progress in its pipeline, including IND-enabling studies for its myotonic dystrophy type 1 (DM1) candidate, expected to submit an IND application to the FDA in Q4 CY2022. The company aims to advance its Huntington's disease (HD) program by nominating a development candidate this year. Recent updates include promising preclinical data on the DM1 candidate, NT-0231.F, and ongoing research for KRAS-driven cancers. However, financial results showed a net loss of approximately $9.9 million for Q2 FY2022, a significant increase from the prior year.

NeuBase Therapeutics (Nasdaq: NBSE) announced acceptance of two abstracts for the ASGCT 25th Annual Meeting, scheduled for May 16-19, 2022. The abstracts focus on NT-0231.F, a precision genetic medicine targeted for myotonic dystrophy type 1 (DM1). NeuBase aims to present preclinical data demonstrating NT-0231.F's efficacy in biodistribution and pharmacokinetics. The company’s innovative PATrOL™ platform offers a method for targeted therapeutic delivery, which could significantly impact the treatment landscape for DM1.

NeuBase Therapeutics (NASDAQ: NBSE) announced promising preclinical results for its candidate NT-0231.F aimed at treating myotonic dystrophy type 1 (DM1). A single intravenous dose demonstrated effective splicing rescue and myotonia reversal, with enduring effects lasting up to six weeks. Multiple subcutaneous doses showed similar efficacy, suggesting a patient-friendly administration method. The company plans to submit an Investigational New Drug (IND) application to the FDA by Q4 CY2022. These findings support the therapeutic potential of NT-0231.F in addressing DM1’s unmet medical needs.

NeuBase Therapeutics (Nasdaq: NBSE) has announced its participation in Oppenheimer’s 32nd Annual Healthcare Conference from March 15-17, 2022. The company will hold a virtual fireside chat on March 16 from 2:40 PM to 3:10 PM ET. Interested parties can register for the event through the registration link. A replay of the webcast will be available for 90 days. NeuBase is focused on developing precision genetic medicines using its proprietary PATrOL™ platform.

NeuBase Therapeutics (Nasdaq: NBSE) announced the presentation of new preclinical data on its myotonic dystrophy type 1 (DM1) program at the 2022 MDA Clinical & Scientific Conference, occurring from March 13-16. The presentations include details on pharmacology, biodistribution, and tolerability of its PATrOL™-enabled investigational genetic therapy. Results from the HSA^LR mouse model highlight the therapy's effectiveness in targeting toxic RNA structures and restoring RNA splicing, crucial for DM1 treatment, which may modify disease impact on patients.

NeuBase Therapeutics (Nasdaq: NBSE) reported advancements in its myotonic dystrophy type 1 (DM1) program, with IND-enabling studies proceeding as planned. The company anticipates submitting an IND application to the FDA in Q4 2022. Additionally, they implemented GMP manufacturing for Phase 1/2 trials and are set to nominate a development candidate for Huntington’s Disease in 2022. Financially, NeuBase posted a net loss of $7.7 million for Q1 2022, an increase from $4.1 million in the previous year, while cash reserves decreased to approximately $47.3 million.

NeuBase Therapeutics (NBSE) announced the appointment of Todd P. Branning as Chief Financial Officer, bringing over 25 years of experience in corporate finance and investor relations. Branning, previously CFO at Phathom Pharmaceuticals and Amneal Pharmaceuticals, aims to enhance NeuBase's growth and develop its pioneering genetic medicines platform. He expressed excitement about the company's potential to transform the pharmaceutical industry by addressing diseases with limited treatment options. NeuBase is focused on filing its first INDs and expanding its therapeutic pipeline.

NeuBase Therapeutics (NBSE) has appointed Dr. Eric J. Ende to its Board of Directors, bringing nearly 25 years of biopharmaceutical experience. His strategic insight is expected to enhance the company's development of its precision genetic medicines, which aim to treat diseases with limited options. Dr. Ende believes NeuBase's technology can transform the genetic medicine landscape by enabling targeted and repeatable treatments. The company focuses on addressing genetic mutations linked to neurological, neuromuscular, and oncologic disorders, leveraging its proprietary PATrOL™ platform.