SYDNEY, July 8, 2018
/PRNewswire/ -- Benitec Biopharma Limited ("Benitec" or the
"Company") (ASX:BLT; NASDAQ: BNTC; NASDAQ: BNTCW) today announced
that it has licensed to Axovant Sciences ("Axovant") the exclusive
global rights for BB-301 (now named AXO-AAV-OPMD) intended for the
treatment of oculopharyngeal muscular dystrophy (OPMD), and has
also entered into a fully funded research collaboration for the
development of five additional gene therapy products in
neurological disorders.
Under the terms of the agreement, Benitec will receive an
upfront cash payment of US$10 million
and additional cash payments totaling US$17.5 million upon completion of four specific
near-term manufacturing, regulatory and clinical milestones.
Axovant has been granted worldwide rights to AXO-AAV-OPMD and will
assume all future development costs. The total potential
value of all of the development, regulatory and commercial
milestones achievable by Benitec, of which there are eight
milestones including the four near-term milestones, is US$187.5 million. Benitec, working in
partnership with Axovant over the next few years, hopes to achieve
all eight milestones and thus realize the maximum amount of
US$187.5 million. There can be no
assurance as to the total amount of payments that the Company will
actually receive or when they will be received. Importantly,
upon commercialization, Benitec will retain 30% of the net profits
on worldwide sales of AXO-AAV-OPMD.
Jerel Banks, MD PhD, Executive
Chairman, Benitec Biopharma commented on today's news, "Today marks
a milestone for Benitec as we believe this transaction to be
transformative for our company. In addition to bolstering our
opportunity to drive broad-based, clinically meaningful patient
benefit across several areas of clinical medicine with true unmet
need, this partnership significantly enhances the financial,
intellectual, and clinical development resources available to
facilitate our efforts to build Benitec into a diversified
biopharmaceutical company. The non-dilutive capital expected
over the near term will allow Benitec to continue to invest in
proprietary R&D programs across a range of indications."
Dr. Banks continued, "Our management team is focused exclusively
on expanding the research, development, and commercial
opportunities for the core 'silence-and-replace' platform with the
dual goals of enhancing patient benefit and generating shareholder
value. We believe Axovant is the ideal partner to advance our
OPMD program, and we look forward to working closely with them to
develop AXO-AAV-OPMD as we quickly progress towards clinical trials
in 2019."
OPMD is a rare progressive, and often fatal, muscle-wasting
disease caused by mutation in the poly(A)-binding protein nuclear 1
(PABPN1) gene, that is characterized by eyelid drooping,
swallowing difficulties, and proximal limb weakness.
AXO-AAV-OPMD is a single vector, gene therapy construct system that
uses a unique "Silence-and-Replace" methodology that employs DNA
directed RNA interference (ddRNAi) to silence expression of the
mutant gene associated with OPMD, while simultaneously expressing a
copy of the normal, healthy version of the same gene to restore the
function of that gene. Axovant plans to initiate a
placebo-controlled clinical study in 2019 in which a one-time
intramuscular administration AXO-AAV-OPMD will be given to patients
to treat the dysphagia associated with OPMD.
Commenting on the agreement, Pavan Cheruvu, MD, Chief Executive
Officer of Axovant said, "The 'silence-and-replace' platform is a
targeted approach which directly addresses the underlying genetic
cause of diseases arising from expression of dysfunctional
proteins, including those caused by nucleotide repeat expansion. I
am excited about the potential of this platform for patients
suffering from OPMD, many of whom have limited treatment options
today."
In addition to AXO-AAV-OPMD, Axovant and Benitec will
collaborate on a total of five additional investigational gene
therapy products for neurological disorders, with Axovant fully
funding each of the research programs. Axovant will have exclusive
global rights to products developed under these programs. The first
additional investigational gene therapy product will focus on
developing a single vector "Silence-and-Replace" gene therapy
product targeting the c9orf72 gene, which is associated with
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia
(FTD). In addition to receiving funding for development of
the new research programs, each new research program target is
eligible for development, regulatory and commercial milestones
totaling US$93.5 million and tiered
royalties on global sales. There can be no assurance as to the
total amount of payments that the Company will actually receive or
when they will be received.
Dr. Banks concluded, "We are extremely excited about Axovant's
collaborative and financial commitments to these five additional
research programs as it plants the seeds for a long and robust
partnership between our organizations. This partnership provides
Benitec with an extraordinarily rare opportunity to unambiguously
demonstrate the exceptional breadth of the scientific, clinical,
and commercial applications of the 'silence-and-replace'
platform. Additionally, the non-dilutive capital expected by
Benitec over the near term will be used to fund operations as we
will continue to innovate and strengthen our platform. I look
forward to making future announcements on our joint progress with
Axovant as well as on other material developments."
For further information regarding Benitec and its activities,
please contact the persons below, or visit the Benitec website at
www.benitec.com.
United States
Investor Relations
Jay
Morakis
President &
CEO
M Group Strategic
Communications
Tel: +1 646 417
8514
Email:
jmorakis@mgroupsc.com
|
Australia Investor
Relations
Megan
Boston
Executive
Director
Benitec Biopharma
Limited
Tel:
+61 400 195 388
Email:
mboston@benitec.com
|
About Benitec Biopharma Limited:
Benitec
Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) is a
biotechnology company developing innovative therapeutics based on
its patented gene-silencing technology called ddRNAi or 'expressed
RNAi'. Based in Sydney, Australia
with laboratories in Hayward,
California (USA), and collaborators and licensees around the
world, the company is developing ddRNAi-based therapeutics for
chronic and life-threatening human conditions including OPMD, head
& neck squamous cell carcinoma, retinal based diseases such as
wet age-related macular degeneration, and hepatitis B.
About OPMD:
OPMD is a rare inherited myopathy
characterized by dysphagia (difficulty in swallowing), the loss of
muscle strength, and weakness in multiple parts of the body.
Patients typically suffer from severe dysphagia, ptosis (eye lid
drooping), tongue atrophy, proximal lower limb weakness, dysphonia
(altered and weak voice), limitation in looking upward, as well as
facial muscle and proximal upper limb weakness. Progressing
throughout that patient's life, OPMD is not typically diagnosed
until the individuals reach their late 40s. As the dysphagia
becomes more severe, patients become malnourished, lose significant
weight, become dehydrated and suffer from repeated incidents of
aspiration pneumonia. The last two symptoms are often the cause of
death. No cure is currently available for OPMD. The
cricopharyngeal myotomy is the only treatment available to improve
swallowing in these patients, but because the root cause of the
genetic disease has not been addressed, the pharyngeal musculature
still undergoes progressive degradation leading to the previously
mentioned complications.
Safe Harbor Statement:
This press release
contains "forward-looking statements" within the meaning of section
27A of the US Securities Act of 1933 and section 21E of the US
Securities Exchange Act of 1934. Any forward-looking statements
that may be in this ASX/Nasdaq announcement are subject to risks
and uncertainties relating to the difficulties in Benitec's plans
to develop and commercialise its product candidates, the timing of
the initiation and completion of preclinical and clinical trials,
the timing of patient enrolment and dosing in clinical trials, the
timing of expected regulatory filings, the clinical utility and
potential attributes and benefits of ddRNAi and Benitec's product
candidates, potential future out-licenses and collaborations, the
intellectual property position and the ability to procure
additional sources of financing. Accordingly, you should not rely
on those forward-looking statements as a prediction of actual
future results.
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SOURCE Benitec Biopharma