{"id":813385,"date":"2025-02-14T08:13:05","date_gmt":"2025-02-14T13:13:05","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/benitec-biopharma-releases-second-quarter-2025-financial-results-and-provides-operational-update\/"},"modified":"2025-02-14T08:13:05","modified_gmt":"2025-02-14T13:13:05","slug":"benitec-biopharma-releases-second-quarter-2025-financial-results-and-provides-operational-update","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/benitec-biopharma-releases-second-quarter-2025-financial-results-and-provides-operational-update\/","title":{"rendered":"Benitec Biopharma Releases Second Quarter 2025 Financial Results and Provides Operational Update"},"content":{"rendered":"
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\n -Fifth Subject in BB-301 Phase 1b\/2a Clinical Treatment Study safely treated in February 2025-<\/em>\n <\/p>\n

\n -Interim clinical study update to be presented in late-breaking oral presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference on March 19, 2025-<\/em>\n <\/p>\n

HAYWARD, Calif., Feb. 14, 2025 (GLOBE NEWSWIRE) — Benitec Biopharma Inc. (NASDAQ: BNTC) (\u201cBenitec\u201d or \u201cCompany\u201d), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary \u201cSilence and Replace\u201d DNA-directed RNA interference (“ddRNAi”) platform, today announced financial results for its second fiscal quarter ended December 31, 2024. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission. The Company also announced the acceptance of a late breaking oral abstract for the BB-301 Phase 1b\/2a Clinical Treatment Study ongoing in Subjects diagnosed with Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia.<\/p>\n

\u201cIn 2024 the interim clinical study data demonstrated durable, clinically meaningful improvements in swallowing function for the first Subjects safely treated with BB-301, and we remain highly optimistic about the potential for continued benefit in Subjects enrolled in the ongoing clinical study,\u201d said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. \u201cWe continue to be extremely grateful for the strong support of the Subjects and their families and for their continued participation in the BB-301 clinical development program, and we look forward to enrolling additional Subjects at the low dose and, this year, at the next, higher dose of BB-301.\u201d<\/p>\n

The key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia, are outlined below:<\/p>\n

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\n Summary of Interim Clinical Study Results for Subject 1 and Subject 2 as Disclosed in October 2024:<\/u>
\n <\/strong>\n <\/p>\n

Oculopharyngeal Muscular Dystrophy (OPMD) is a rare, autosomal dominant, late-onset degenerative muscle disorder presenting in patients at 40-60 years of age. OPMD is principally characterized by severe progressive dysphagia, impacting 97% of patients, which can lead to chronic choking, malnutrition, aspiration pneumonia and, in severe cases, death. OPMD is caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene.<\/p>\n

There is no effective drug therapy available for OPMD. Current interventions are limited to palliative surgical procedures and dietary modifications, which do not address the underlying cause of disease.<\/p>\n

BB-301, a novel investigational gene therapy designed to improve the dysphagic symptoms of OPMD, is being evaluated in a Phase 1b\/2a, open-label dose escalation study (NCT06185673) to assess safety and clinical activity.<\/p>\n

Two causes of dysphagia have been observed in study Subjects: excessive accumulation of solid and liquid residue (Total Pharyngeal Residue or \u201cTPR\u201d) remaining post-swallow or \u201cinefficient swallowing\u201d, and recurrent pathologic sequential swallowing (i.e., rapid involuntary contractions of the pharyngeal muscles, between which the resting diameter of the pharynx is not restored) or \u201cineffective swallowing\u201d. Pathologic sequential swallowing is experienced by Subjects as involuntary swallows.<\/p>\n

Outcome measures for NCT06185673 include videofluoroscopic swallowing studies for serial assessment of TPR and frequency of pathologic sequential swallowing, and the use of a patient-reported outcome instrument (Sydney Swallow Questionnaire).<\/p>\n

Five Subjects have been safely treated with the lowest-dose of BB-301, and interim results for the first two Subjects were presented in October 2024 and are summarized here:<\/p>\n

Interim study results for the first two Subjects treated with BB-301 following 9-months and 6-months on treatment, respectively, demonstrated durable, clinically significant reductions in both causes of dysphagic deficits. There have been no Severe Adverse Events in study Subjects.<\/p>\n

These data represent successful improvements in swallowing function driven by a novel gene therapy for OPMD.<\/p>\n

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\n Enrollment into the BB-301 Phase 1b\/2a Clinical Treatment Study is Ongoing:<\/u>
\n <\/strong>\n <\/p>\n