Benitec Biopharma Ltd (BNTC)

[georgejjl]

Update on Oculopharyngeal Muscular Dystrophy Program
Sydney, Australia, 6 June 2019: Benitec Biopharma Limited (“Benitec” or the “Company”) (ASX:BLT;
NASDAQ: BNTC; NASDAQ: BNTCW) today announced the termination of the License and Collaboration
Agreement with Axovant Sciences, as the Benitec team endeavors to conduct several additional
exploratory analyses prior to the initiation of the clinical study in order to potentially improve the
biological efficacy of the compound via further optimization of the proprietary delivery method employed
to dose the target tissues.
Preclinical data derived from recently concluded in vivo evaluations of AXO-AAV-OPMD (formerly
designated as BB-301) in two distinct large animal species suggest that the opportunity exists to further
improve the biological efficacy of the compound via additional optimization of the proprietary delivery
method employed to dose key target tissues that underlie the morbidity and mortality associated with
the progression of Oculopharyngeal Muscular Dystrophy. The initial biological efficacy profile observed
for BB-301 following in vivo testing in the murine model of Oculopharyngeal Muscular Dystrophy,
including full correction of the disease phenotype, remains unchanged. However, the Benitec team
endeavors to conduct several additional exploratory analyses prior to the initiation of clinical testing.
Completion of the experimental work noted above would delay the initiation of the BB-301 clinical study
beyond the timelines that were initially outlined following the execution of the License and
Collaboration Agreement between Benitec Biopharma and Axovant Sciences. As such, the License and
Collaboration Agreement between Benitec Biopharma and Axovant Sciences will be terminated, and all
rights and licenses granted to Axovant Sciences will cease, including the rights to BB-301, which was in
preclinical development for the treatment of Oculopharyngeal Muscular Dystrophy, and all other early
stage research collaboration programs.
The termination of the License and Collaboration Agreement will be effective on September 3, 2019.
Updates will be provided over the coming months regarding the new research programs to be advanced
by the Benitec team.

Good luck and GOD bless,