-Topic 1 and Topic 2 skilled sturdy, clinically significant enhancements in swallowing at 9-months and 6-months post-BB-301 therapy, respectively, with Topic 2 reaching a Sydney Swallow Questionnaire Rating Consultant of Clinically Regular Swallowing-
-Administration plans to host a convention name on October 14 at 8:30 am EDT to debate the interim outcomes, particulars below-
HAYWARD, Calif., Oct. 12, 2024 (GLOBE NEWSWIRE) — Benitec Biopharma Inc. (NASDAQ: BNTC) (Benitec or Firm), a clinical-stage, gene therapy-focused, biotechnology firm growing novel genetic medicines primarily based on its proprietary Silence and Substitute DNA-directed RNA interference (ddRNAi) platform, at this time declares continued sturdy enhancements in swallowing following administration of the low-dose of BB-301 within the examine’s first two topics handled within the BB-301 Section 1b/2a single-arm, open-label, sequential, dose-escalation cohort examine (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). Interim scientific examine information had been introduced at this time in an oral late-breaking podium presentation on the twenty ninth Annual Congress of the World Muscle Society, happening in Prague, Czech Republic by the examine’s principal investigator, Professor Milan R. Amin, M.D., Division of Otolaryngology-Head and Neck Surgical procedure, New York College Grossman College of Medication, Director, New York College Langone Voice Heart.
The interim scientific examine replace introduced on the Annual Congress of the World Muscle Society detailed the 9-month (270-day) post-dose outcomes for the primary topic and the 6-month (180-day) post-dose outcomes for the second topic, each of whom have been safely handled with BB-301. Key efficacy endpoints introduced included videofluoroscopic swallowing examine (VFSS) assessments of Complete Pharyngeal Residue (TPR, i.e., the quantity of strong meals or liquid materials remaining within the pharynx after the primary swallow) and the Topic-Reported Final result Instrument (i.e., the Sydney Swallow Questionnaire or SSQ). The post-dose outcomes had been in comparison with the common pre-dose outcomes for every topic as evaluated throughout the 5 scientific evaluation visits carried out throughout their enrollment within the Benitec-sponsored OPMD Pure Historical past (NH) Research. The post-dose outcomes for every topic had been additionally in comparison with the pre-dose outcome derived from the ultimate pre-dose scientific go to previous to the administration of BB-301. An in depth description of the interim scientific trial outcomes might be discovered within the presentation accessible on the Firm web site right here.
We’re grateful to the themes and their households for his or her robust assist and continued participation within the BB-301 scientific improvement program. We’re extremely inspired by the numerous, clinically significant enhancements noticed for each topics handled on the low-dose of BB-301, with Topic 2 reaching a clinically regular swallowing profile primarily based on the outcomes of the Sydney Swallow Questionnaire, stated Jerel A. Banks, M.D., Ph.D., Government Chairman and Chief Government Officer of Benitec. The third topic can be handled with the low dose of BB-301 this month, and we’re optimistic in regards to the potential for continued profit in topics enrolled within the ongoing examine. We stay up for enrolling further topics on the subsequent, greater doses of BB-301 in 2025.
Abstract of Outcomes:
- Two topics have acquired the lowest-dose of gene remedy BB-301 (1.2e13 vg/topic), and there have been no Vital Antagonistic Occasions.
- Dysphagic signs at baseline for Topic 1 (7-years submit analysis) had been extra extreme than these of Topic 2 (6-years submit analysis) as assessed by pre-dose SSQ and TPR outcomes, however each Topics skilled vital ranges of scientific profit per the post-dose SSQ scores and TPR outcomes.
- The SSQ Complete Scores and SSQ Sub-Scores correlate strongly with the VFSS TPR outcomes.
- Topic 1 skilled clinically significant enhancements in post-dose SSQ Complete Rating and SSQ Sub-Scores at Day 270 pushed by corresponding reductions in VFSS TPR values.
- Topic 2 skilled clinically significant enhancements in post-dose SSQ Complete Rating and SSQ Sub-Scores at Day 180, with an SSQ Complete Rating consultant of a standard swallowing profile, pushed by a corresponding discount within the frequency of pathologic low-volume sequential swallows.
- These information characterize the primary reported profitable enhancements in swallow operate utilizing a novel gene remedy for OPMD.
Topics enrolled within the NH Research and the BB-301 Section 1b/2a Scientific Research have been proven to be impacted by:
- Extreme accumulation of pharyngeal residue post-swallow
- Pathologic low-volume sequential swallows (i.e., speedy contractions of the pharyngeal muscle groups throughout the consumption of low volumes of skinny liquids)
Topic 1 (270 Days post-BB-301 dose):
World inefficiency of swallowing for strong meals, skinny liquid, and thick liquids drives dysphagia for Topic 1.
Topic 1 displayed continued clinically significant reductions (i.e., enhancements) in SSQ Complete Rating (35% discount) and SSQ Sub-Scores (42% discount for Skinny Liquid, 16% discount for Stable Meals, and 22% discount for Thick Liquids). Topic 1 displayed correspondingly vital reductions (i.e., enhancements) in VFSS TPR (33% discount for Skinny Liquid, 18% discount for Stable Meals, and 30% discount for Thick Liquids) following the administration of the low-dose of BB-301 as in comparison with the common values recorded for Topic 1 throughout the pre-dose interval.
Topic 2 (180 Days post-BB-301-dose):
Pathologic low-volume sequential swallowing for skinny liquid drives dysphagia for Topic 2. Pathologic low-volume sequential swallows are skilled by the topic as a number of swallows and are detected throughout VFSS as a collection of speedy contractions of the pharyngeal muscle groups interrupting the discrete peristaltic contraction sample sometimes noticed throughout swallows of low volumes of skinny liquids.
Topic 2 displayed clinically significant reductions (i.e., enhancements) in SSQ Complete Rating (89% discount) and the SSQ Sub-Rating for the need of repeat swallows throughout consumption (84% discount) as in comparison with the common values recorded for Topic 2 throughout the pre-dose interval. The common post-dose SSQ Complete Rating of 82 is consultant of a clinically regular swallowing profile for Topic 2. Topic 2 displayed correspondingly vital reductions (i.e., enhancements) within the post-dose frequency of low-volume sequential swallows as evaluated by VFSS (92% discount) following the administration of the low-dose of BB-301 as in comparison with the pre-dose values recorded for Topic 2 throughout the pre-dose interval.
All examine Topics are blinded to their SSQ Complete Scores and VFSS TPR evaluation outcomes, and the Central Reader for the VFSS assessments is blinded to the SSQ Complete Scores and SSQ Sub-Scores for all Research Topics.
Firm Webcast Data:
A stay webcast of the interim scientific information presentation, together with administration and Emily Plowman, PhD, CCC-SLP, FASHA “ Professor, Division of Otolaryngology “ Head and Neck Surgical procedure, The Ohio State College Faculty of Medication, can be held at 8:30AM EDT on Monday October 14th, 2024, and might be accessed right here. Traders might also dial in toll-free at 1-877-269-7751 or 1-201-389-0908 (worldwide). The convention ID is 13749637. The occasion replay can be positioned on the Information & Occasions tab on the Investor web page of the Benitec web site.
About OPMD
OPMD is a uncommon progressive muscle-wasting illness attributable to a mutation within the poly(A)-binding protein nuclear 1 (PABPN1) gene, for which there’s at the moment no efficient drug remedy. The illness is characterised by swallowing difficulties (dysphagia), limb weak spot and eyelid drooping (ptosis). Dysphagia worsens over time and may result in continual choking, regurgitation, aspiration pneumonia, and in extreme circumstances, loss of life. Obtainable scientific and surgical interventions are restricted in scope and effectiveness and don’t handle the underlying progressive muscle weak spot.
About BB-301
BB-301 is a novel, modified AAV9 capsid expressing a singular, single bifunctional assemble selling co-expression of each codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) towards mutant PABPN1. The 2 siRNAs are modeled into microRNA backbones to silence expression of defective mutant PABPN1, whereas permitting expression of the codon-optimized PABPN1 to interchange the mutant with a useful model of the protein. We consider the silence and change mechanism of BB-301 is uniquely positioned for the therapy of OPMD by halting mutant expression whereas offering a useful substitute protein.
About Benitec Biopharma, Inc.
Benitec Biopharma Inc. (Benitec or the Firm) is a clinical-stage biotechnology firm targeted on the development of novel genetic medicines with headquarters in Hayward, California. The proprietary Silence and Substitute DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene remedy to create medicines that concurrently facilitate sustained silencing of disease-causing genes and concomitant supply of wildtype substitute genes following a single administration of the therapeutic assemble. The Firm is growing Silence and Substitute-based therapeutics for continual and life-threatening human circumstances together with Oculopharyngeal Muscular Dystrophy (OPMD). A complete overview of the Firm might be discovered on Benitec’s web site at www.benitec.com.
Ahead Trying Statements
Apart from the historic data set forth herein, the issues set forth on this press launch embody forward-looking statements, together with statements relating to Benitec’s plans to develop and probably commercialize its product candidates, the timing of completion of pre-clinical and scientific trials, the timing of the supply of information from our scientific trials, the timing and sufficiency of affected person enrollment and dosing in scientific trials, the timing of anticipated regulatory filings, the scientific utility and potential attributes and advantages of ddRNAi and Benitec’s product candidates, the mental property place, and different forward-looking statements.
These forward-looking statements are primarily based on the Firm’s present expectations and topic to dangers and uncertainties which will trigger precise outcomes to vary materially, together with unanticipated developments in and dangers associated to: unanticipated delays; additional analysis and improvement and the outcomes of scientific trials presumably being unsuccessful or inadequate to fulfill relevant regulatory requirements or warrant continued improvement; the power to enroll enough numbers of topics in scientific trials; determinations made by the FDA and different governmental authorities; the Firm’s capacity to guard and implement its patents and different mental property rights; the Firm’s dependence on its relationships with its collaboration companions and different third events; the efficacy or security of the Firm’s merchandise and the merchandise of the Firm’s collaboration companions; the acceptance of the Firm’s merchandise and the merchandise of the Firm’s collaboration companions within the market; market competitors; gross sales, advertising, manufacturing and distribution necessities; larger than anticipated bills; bills regarding litigation or strategic actions; the Firm’s capacity to fulfill its capital wants via growing its income and acquiring further financing, given market circumstances and different elements, together with our capital construction; our capacity to proceed as a going concern; the size of time over which the Firm expects its money and money equivalents to be enough to execute on its marketing strategy; the affect of native, regional, and nationwide and worldwide financial circumstances and occasions; and different dangers detailed every now and then within the Firm’s experiences filed with the Securities and Change Fee. The Firm disclaims any intent or obligation to replace these forward-looking statements.
Investor Relations Contact:
Irina Koffler
LifeSci Advisors, LLC
(917) 734-7387
ikoffler@lifesciadvisors.com
Supply: Benitec Biopharma Inc.
