Benitec Biopharma Releases First Quarter 2023 Financial Results and Provides Operational Update
November 10 2022 - 8:00AM
Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “the Company”),
a development-stage, gene therapy-focused, biotechnology company
developing novel genetic medicines based on its proprietary
DNA-directed RNA interference ("ddRNAi") platform, today announced
financial results for its Fiscal Year First Quarter ended September
30, 2022. The Company has filed its quarterly report on Form 10-Q
for the quarter ended September 30, 2022, with the U.S. Securities
and Exchange Commission.
“Following the receipt of a favorable decision
from the Institutional Review Board of the lead clinical site for
the OPMD Natural History Study in the United States and the formal
completion of the Site Initiation Visit at this key clinical
enrollment site, the screening of OPMD subjects for enrollment into
the OPMD Natural History Study will begin this month. The
initiation of subject screening for the OPMD Natural History Study
represents a major milestone for Benitec, and we look forward to
enrolling the first subjects into the OPMD Natural History Study
this year. The enrollment of subjects into the OPMD Natural History
Study in 2022 continues to support our central clinical development
goal of administering the first dose of BB-301 in 2023,” said Jerel
A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive
Officer of Benitec Biopharma. “The Principal Investigator of the
OPMD Natural History Study in the United States expects high
enrollment interest for this incurable genetic disease with no
approved treatments, and we continue to work with regulators
globally to open additional sites in geographies outside of the
United States.”
Operational Updates
The key milestones related to the development of
BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy
(OPMD), along with other corporate updates, are outlined below:
BB-301 Clinical Development Program
Overview:
- The BB-301 clinical development
program will be conducted in the United States, Canada, and France,
and the primary elements of the clinical development plan are
summarized below.
- The clinical development plan will
begin in 2022 and comprise approximately 76 weeks of follow-up:
- 6-month pre-treatment observation
periods for the evaluation of baseline disposition and natural
history of OPMD-derived dysphagia (swallowing impairment) in each
study participant.
- 1-day of BB-301 dosing to initiate
participation in the Phase 1b/2a single-arm, open-label,
sequential, dose-escalation cohort study.
- 52-weeks of post-dosing follow-up
for conclusive evaluation of the primary and secondary endpoints of
the Phase 1b/2a BB-301 treatment study.
- The OPMD Natural History (NH) Study
(i.e., the 6-month pre-treatment observation period) will
facilitate the characterization of OPMD patient disposition at
baseline and assess subsequent rates of progression of dysphagia
via the use of the following quantitative radiographic measures
(i.e., videofluoroscopic swallowing studies or “VFSS”), with the
VFSS outlined below collectively providing objective assessments of
global swallowing function and the function of the pharyngeal
constrictor muscles (i.e., the muscles whose functional
deterioration drives disease progression in OPMD):
- Total Pharyngeal Residue
%(C2-4)2
- Pharyngeal Area at Maximum
Constriction (PhAMPC)
- Dynamic Imaging Grade of Swallowing
Toxicity Scale (DIGEST)
- Vallecular Residue %(C2-4)2,
Pyriform Sinus Residue %(C2-4)2, and Other Pharyngeal Residue
%(C2-4)2
- Normalized Residue Ratio Scale
(NRRSv, NRRSp)
- Pharyngeal Construction Ratio
(PCR)
- The NH study will also employ
clinical measures of global swallowing capacity and oropharyngeal
dysphagia, along with two distinct patient-reported outcome
instruments targeting the assessment of oropharyngeal
dysphagia.
- Upon the achievement of 6-months of
follow-up in the NH Study, participants will be eligible for
enrollment onto the BB-301 Phase 1b/2a treatment study.
- BB-301 Phase 1b/2a Treatment Study:
- This first-in-human (FIH) study
will be a Phase 1b/2a, open-label, dose-escalation study to
evaluate the safety and clinical activity of intramuscular doses of
BB-301 administered to subjects with OPMD.
- Upon rollover onto the Phase 1b/2a
BB-301 treatment study, the follow-up of OPMD study participants
will continue for 52 weeks.
- The primary endpoints of the FIH
study will be safety and tolerability, with secondary endpoints
comprising quantitative radiographic measures of global swallowing
function and pharyngeal constrictor muscle function (i.e., VFSS) as
well as clinical assessments (all of which will be equivalent to
those employed for the NH study). These endpoints will be evaluated
during each 90-day period following BB-301 intramuscular injection
(Day 1).
- The natural history of dysphagia
observed for each OPMD study participant, as characterized by the
VFSS and clinical swallowing assessments carried out during the NH
Study, will serve as the baseline for comparative assessment of
safety and efficacy of BB-301 upon rollover from the NH Study onto
the Treatment Study.
Operational and Regulatory Updates for
the Clinical Development Program:
North America:
- Formal submission of the
comprehensive NH Study trial package to the Institutional Review
Board (IRB) at the lead clinical site in the United States was
completed in August 2022.
- The submission package included:
the NH Study Protocol, the subject Informed Consent Form, the
patient-facing clinical documents and recruitment documents, the
physician-facing clinical and laboratory manuals and recruitment
documents.
- Additionally, the primary clinical
study site was formally qualified, the Principal Investigator and
Sub-Investigators were identified at the clinical study site, the
core VFSS site was selected and qualified, and the Central Reader
for the VFSS data was selected.
- The IRB for the lead clinical site
in the United States approved the NH Study trial package in
September 2022.
- Following the favorable IRB decision, the Site Initiation Visit
was completed in October 2022, and the screening of OPMD subjects
for enrollment into the OPMD Natural History Study will begin at
the lead clinical site in the United States in November 2022.
- Planning for the formal submission
of the comprehensive NH Study trial package to the Research Ethics
Board (REB) for the lead clinical site in Canada is ongoing.
- Approval of the overall NH Study trial package by the REB
allows for clinical study site activation and OPMD patient
screening and enrollment to begin in Canada.
France:
- In the Third Calendar Quarter 2022,
Benitec submitted responses to the Central Ethics Committee (CEC)
related to minor non-clinical queries.
- In November 2022 two additional
non-clinical queries have been received from the CEC regarding the
General Data Protection Regulation (GDPR) in the European Union.
- GDPR is a regulation that requires
businesses to protect the personal data and privacy of European
Union (EU) citizens.
- GDPR became effective on May 25,
2018, and is applicable to organizations within the EU that use
personal data, as well as international organizations that provide
goods and services to individuals in the EU or monitor their
behavior.
- Approval of the overall NH Study
trial package by the CEC allows for clinical study site activation
and OPMD patient screening and enrollment to begin in France
BB-301 Phase 1b/2a Regulatory Updates:
- Investigational New Drug (IND) and
Clinical Trial Application (CTA) filings are anticipated in the
First Calendar Quarter 2023.
- IND and CTA filings are required to
initiate the BB-301 Phase 1b/2a Treatment Study in the United
States, Canada, and France.
- The first NH Study subject is
anticipated to be eligible for BB-301 administration in the Second
Calendar Quarter 2023 (following 6-months of NH Study enrollment
and follow-up and final confirmation of eligibility for the BB-301
Phase 1b/2a Treatment Study).
- Interim safety and efficacy data
for subjects enrolled onto the BB-301 Phase 1b/2a Treatment Study
are anticipated to become available for disclosure approximately
every 90-days following BB-301 administration.
- In preclinical proof-of-concept
studies for BB-301, the OPMD disease phenotype was reversed at 14
weeks (i.e., approximately 90-days) after the administration of
BB-301 (with the strength of BB-301 injected muscles being restored
in the diseased animals and robust evidence of anatomical and
microscopic improvement of the BB-301 injected muscle tissues being
observed).
Financial Highlights
First Quarter 2023 Financial Results
Total Revenues for the quarter ended September
30, 2022, were $0 compared to $0 for the quarter ended September
30, 2021.
Total Expenses for the quarter ended September
30, 2022, were $4.6 million compared to $4.8 million for the
quarter ended September 30, 2021. The Company incurred $2.7 million
of research and development expenses compared to $2.8 million for
the comparable quarter ended September 30, 2021. Research and
development expenses relate primarily to the OPMD project. The
Company continued with the GMPmanufacturing project after
concluding the BB-301 Regulatory Toxicology Study and the Parallel
Assay Method Development, Qualification, and Validation project.
For the quarter ended September 30, 2022, general and
administrative expenses were $1.9 million compared to $2.0 million
for the quarter ended September 30, 2021. The decrease was due to
the decrease in salaries and wages, insurance, state and local
taxes.
The loss from operations for the quarter ended
September 30, 2022, was $4.6 million compared to a loss of $4.8
million for the quarter ended September 30, 2021. Net loss
attributable to shareholders for the quarter ended September 30,
2022, was $5.1 million, or $0.47 per basic and diluted share,
compared to a net loss of $5.0 million, or $0.62 per basic and
diluted share for the quarter ended September 30, 2021. As of
September 30, 2022, the Company had $16.5 million in cash and cash
equivalents.
|
BENITEC BIOPHARMA
INC. |
Consolidated Balance
Sheets |
(in thousands,
except par value and share amounts) |
|
|
|
|
|
|
|
September 30, |
|
|
|
|
June 30, |
|
|
|
2022 |
|
|
|
|
2022 |
|
|
(Unaudited) |
|
|
|
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
$ |
16,534 |
|
|
|
$ |
4,062 |
|
Restricted cash |
|
13 |
|
|
|
|
14 |
|
Trade and other receivables |
|
3 |
|
|
|
|
3 |
|
Prepaid and other assets |
|
531 |
|
|
|
|
741 |
|
Total
current assets |
|
17,081 |
|
|
|
|
4,820 |
|
Property and
equipment, net |
|
179 |
|
|
|
|
222 |
|
Deposits |
|
25 |
|
|
|
|
25 |
|
Other
assets |
|
119 |
|
|
|
|
135 |
|
Right-of-use
assets |
|
711 |
|
|
|
|
771 |
|
Total
assets |
$ |
18,115 |
|
|
|
$ |
5,973 |
|
Liabilities
and stockholders' equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Trade and other payables |
$ |
2,368 |
|
|
|
$ |
1,880 |
|
Accrued employee benefits |
|
385 |
|
|
|
|
400 |
|
Lease liabilities, current portion |
|
258 |
|
|
|
|
252 |
|
Total
current liabilities |
|
3,011 |
|
|
|
|
2,532 |
|
Lease
liabilities, less current portion |
|
491 |
|
|
|
|
559 |
|
Total
liabilities |
|
3,502 |
|
|
|
|
3,091 |
|
Commitments
and contingencies (Note 10) |
|
|
|
|
Stockholders' equity: |
|
|
|
|
Common stock, $0.0001 par value-40,000,000 shares authorized;
25,809,533 shares and 8,171,690 sharesissued and outstanding at
September 30, 2022 and June 30, 2022, respectively |
|
3 |
|
|
|
|
1 |
|
Additional paid-in capital |
|
168,768 |
|
|
|
|
152,453 |
|
Accumulated deficit |
|
(153,420) |
|
|
|
|
(148,327) |
|
Accumulated other comprehensive loss |
|
(738) |
|
|
|
|
(1,245) |
|
Total
stockholders' equity |
|
14,613 |
|
|
|
|
2,882 |
|
Total
liabilities and stockholders' equity |
$ |
18,115 |
|
|
|
$ |
5,973 |
|
|
|
|
|
|
|
BENITEC BIOPHARMA
INC. |
Consolidated
Statements of Operations and Comprehensive Loss |
(Unaudited) |
(in thousands,
except share and per share amounts) |
|
|
|
|
|
|
Three Months Ended September 30 |
|
|
2022 |
|
|
|
|
2021 |
|
Revenue: |
|
|
|
|
Licensing revenues from customers |
|
|
|
|
Total revenues |
$ |
- |
|
|
|
$ |
- |
|
|
|
- |
|
|
|
|
- |
|
Operating Expenses |
|
|
|
|
Research and development |
|
2,660 |
|
|
|
|
2,780 |
|
General and administrative |
|
1,920 |
|
|
|
|
2,042 |
|
Total operating expenses |
|
4,580 |
|
|
|
|
4,822 |
|
Loss from operations |
|
(4,580) |
|
|
|
|
(4,822) |
|
Other income (loss): |
|
|
|
|
Foreign currency transaction loss |
|
(507) |
|
|
|
|
(240) |
|
Interest expense, net |
|
(9) |
|
|
|
|
(1) |
|
Unrealized gain on investment |
|
3 |
|
|
|
|
18 |
|
Total other income (loss), net |
|
(513) |
|
|
|
|
(223) |
|
Net loss |
$ |
(5,093) |
|
|
|
$ |
(5,045) |
|
Other comprehensive income: |
|
|
|
|
Unrealized foreign currency translation gain |
|
507 |
|
|
|
|
239 |
|
Total other comprehensive income |
|
507 |
|
|
|
|
239 |
|
Total comprehensive loss |
$ |
(4,586) |
|
|
|
$ |
(4,806) |
|
Net loss |
$ |
(5,093) |
|
|
|
$ |
(5,045) |
|
Net loss per share: basic and diluted |
$ |
(0.47) |
|
|
|
$ |
(0.62) |
|
Weighted average number of shares outstanding: basic and
diluted |
|
10,855,710 |
|
|
|
|
8,171,690 |
|
|
|
|
|
|
About Benitec Biopharma Inc.
Benitec Biopharma Inc. (“Benitec” or the
“Company”) is a development-stage biotechnology company focused on
the advancement of novel genetic medicines with headquarters in
Hayward, California. The proprietary platform, called DNA-directed
RNA interference, or ddRNAi, combines RNA interference, or RNAi,
with gene therapy to create medicines that facilitate sustained
silencing of disease-causing genes following a single
administration. The Company is developing ddRNAi-based therapeutics
for chronic and life-threatening human conditions including
Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview
of the Company can be found on Benitec’s website at
www.benitec.com.
Forward Looking Statements
Except for the historical information set forth
herein, the matters set forth in this press release include
forward-looking statements, including statements regarding
Benitec's plans to develop and commercialize its product
candidates, the timing of the initiation and completion of
pre-clinical 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, and
other forward-looking statements.
These forward-looking statements are based on
the Company's current expectations and subject to risks and
uncertainties that may cause actual results to differ materially,
including unanticipated developments in and risks related to:
unanticipated delays; further research and development and the
results of clinical trials possibly being unsuccessful or
insufficient to meet applicable regulatory standards or warrant
continued development; the ability to enroll sufficient numbers of
subjects in clinical trials; determinations made by the FDA and
other governmental authorities; the Company's ability to protect
and enforce its patents and other intellectual property rights; the
Company's dependence on its relationships with its collaboration
partners and other third parties; the efficacy or safety of the
Company's products and the products of the Company's collaboration
partners; the acceptance of the Company's products and the products
of the Company's collaboration partners in the marketplace; market
competition; sales, marketing, manufacturing and distribution
requirements; greater than expected expenses; expenses relating to
litigation or strategic activities; the Company's ability to
satisfy its capital needs through increasing its revenue and
obtaining additional financing; given market conditions and other
factors, including our capital structure; our ability to continue
as a going concern; the length of time over which the Company
expects its cash and cash equivalents to be sufficient to execute
on its business plan; the impact of the current COVID-19 pandemic,
the disease caused by the SARS-CoV-2 virus, which may adversely
impact the Company's business and pre-clinical and future clinical
trials; the impact of local, regional, and national and
international economic conditions and events; and other risks
detailed from time to time in the Company's reports filed with the
Securities and Exchange Commission. The Company disclaims any
intent or obligation to update these forward-looking
statements.
Investor Relations Contact:
William WindhamVP, Solebury Strategic
CommunicationsPhone: 646-378-2946Email:
wwindham@soleburystrat.com
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