Benitec Biopharma Releases Third Quarter 2023 Financial Results and Provides Operational Update
15 Maggio 2023 - 2:00PM
Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “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 Third Fiscal Quarter ended March 31,
2023. The Company has filed its quarterly report on Form 10-Q for
the quarter ended March 31, 2023 with the U.S. Securities and
Exchange Commission.
“We continue to screen and enroll OPMD subjects
into the Natural History Study at the U.S. clinical study site, and
enrollment is proceeding at a rapid pace,” said Jerel A. Banks,
M.D., Ph.D., Executive Chairman and Chief Executive Officer of
Benitec. “With 9 subjects enrolled to date, the current pace of
enrollment supports our central clinical development goals of
administering BB-301 to OPMD subjects in 2H2023 and disclosing
interim safety and efficacy data in 2H2023 for one or more subjects
that have received BB-301.”
Dr. Banks continued, “We remain focused on
opening additional clinical study sites in Canada and France,
pending ongoing discussions with Institutional Review Boards and
regional regulators. While those discussions progress, we continue
to advance the development of BB-301 in the United States and
expect to receive a response from the U.S. FDA regarding the
Investigational New Drug (IND) application for BB-301 in the Second
Calendar Quarter of 2023.”
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 program are summarized below:
- The program will comprise
approximately 76 weeks of follow-up which we anticipate will
consist of:
- The OPMD Natural History
(NH) Study: 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.
- Dosing with
BB-301: 1-day of BB-301 dosing to initiate participation
in the Phase 1b/2a single-arm, open-label, sequential,
dose-escalation cohort study. BB-301 will be delivered directly to
the pharyngeal muscles of each study subject.
- Phase 1b/2a Treatment
Evaluation: 52-weeks of post-dosing follow-up for
conclusive evaluation of the primary and secondary endpoints of the
BB-301 Phase 1b/2a treatment study, with interim safety and
efficacy results expected to be available at the end of each 90-day
period following the administration of BB-301.
- The OPMD NH Study will characterize
the level of dysphagia borne by each OPMD subject at baseline and
assess subsequent progression of dysphagia via the use of the
following quantitative radiographic measures (i.e.,
videofluoroscopic swallowing studies or “VFSS”). The VFSS outlined
below collectively provide 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 into the BB-301 Phase 1b/2a treatment study.
- BB-301 Phase 1b/2a Treatment Study:
- This first-in-human (FIH) study
will evaluate the safety and clinical activity of intramuscular
doses of BB-301 administered to subjects with OPMD.
- The primary endpoint of the FIH
study will be safety.
- Secondary endpoints are designed to
determine the impact of BB-301 on swallowing efficiency, swallowing
safety, and pharyngeal constrictor muscle function in subjects
diagnosed with OPMD with dysphagia via the use of serial clinical
and videofluoroscopic assessments. Critically, each of the clinical
and videofluoroscopic assessments employed in the FIH study will be
equivalent to those employed for the NH study to facilitate
comparative clinical and statistical analyses.
- The primary and secondary endpoints
will be evaluated during each 90-day period following BB-301
intramuscular injection (Day 1).
- The NH 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 assessments of safety
and efficacy of BB-301 upon rollover from the NH Study onto the
BB-301 Phase 1b/2a Treatment Study.
- In December 2022, Benitec began
screening OPMD subjects at the lead clinical study site in the
United States.
- In January 2023, Benitec announced
the enrollment of the first OPMD subject into the NH Study. As of
today, 9 subjects have been enrolled into the NH study in the
United States.
- The pace of enrollment of OPMD
subjects into the NH Study at the U.S. clinical study site supports
our central clinical development goals of: (1) administering BB-301
to OPMD subjects in 2H2023, and (2) disclosing interim safety and
efficacy data in 2H2023 for one or more subjects that have received
BB-301.
- Jerel A. Banks, M.D., Ph.D.,
Executive Chairman and Chief Executive Officer of Benitec, will
present at the OPMD International Conference in Tel Aviv, Israel,
taking place on Tuesday, May 16th, 2023.
Regulatory Updates for the Clinical
Development Program:
North America:
- Formal submission of the
comprehensive NH Study trial package to the Research Ethics Board
(REB) for the lead clinical study site in Canada was completed, and
Benitec awaits the formal response from the REB.
- Approval of the NH Study trial package by the REB is required
for clinical study site activation and OPMD patient screening and
enrollment to begin in Canada.
- BB-301 Investigational New Drug (IND) application acceptance by
the U.S. FDA is expected in the Second Calendar Quarter of 2023.
- BB-301 IND acceptance is required to initiate the dosing of
OPMD subjects with BB-301 in the Phase 1b/2a Treatment Study in the
United States.
- BB-301 Clinical Trial Application (CTA) clearance is expected
in 2H2023.
- BB-301 CTA clearance is required to initiate the dosing of OPMD
subjects with BB-301 in the Phase 1b/2a Treatment Study in
Canada.
- The first NH Study subject is anticipated to be eligible for
BB-301 administration in the Third Calendar Quarter of 2023,
following the completion of 6 months of NH Study clinical follow-up
and confirmation of eligibility for the BB-301 Phase 1b/2a
Treatment Study. Interim safety and efficacy results are expected
to become available approximately 90 days following the
administration of BB-301.
France:
- BB-301 CTA filing to support the conduct of a comprehensive
BB-301 clinical study in France, inclusive of a 6-month
pre-treatment observation period, one day of BB-301 dosing, and a
subsequent 52-week follow-up period, is planned for completion in
the Third Calendar Quarter of 2023.
Financial Highlights
Third Quarter 2023 Financial Results
Revenue for the quarter ended March 31, 2023,
was $54 thousand compared to $48 thousand for the quarter ended
March 31, 2022.
Operating expenses for the quarter ended March 31, 2023, were
$4.4 million compared to $3.5 million for the quarter ended March
31, 2022. The Company incurred $3.17 million of research and
development expenses compared to $2.17 million for the comparable
quarter ended March 31, 2022. Research and development expenses
relate primarily to the OPMD project. The year over year increase
in research and development costs for the period relates primarily
to the continuation of the GMP manufacturing project and the
Natural History Study. For the quarter ended March 31, 2023,
general and administrative expenses were $1.2 million compared to
$1.3 million for the quarter ended March 31, 2022. The year over
year decrease for the three-month periods ended March 31 relates to
lower listing and filing fees and stock-based compensation.
The loss from operations for the quarter ended
March 31, 2023, was $4.4 million compared to a loss of $3.5 million
for the quarter ended March 31, 2022. Net loss attributable to
stockholders for the quarter ended March 31, 2023, was $4.4
million, or $0.16 per basic and diluted share, compared to a net
loss of $3.3 million, or $0.40 per basic and diluted share for the
quarter ended March 31, 2022. As of March 31, 2023, the Company had
$6.6 million in cash and cash equivalents.
BENITEC BIOPHARMA INC. |
Consolidated Balance Sheets |
(in thousands, except par value and share amounts) |
|
|
|
|
|
|
|
|
|
|
|
March 31, |
|
|
|
June 30, |
|
|
|
|
2023 |
|
|
|
|
|
2022 |
|
|
|
|
(Unaudited) |
|
|
|
|
Assets |
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
6,551 |
|
|
|
|
$ |
4,062 |
|
|
Restricted cash |
|
|
14 |
|
|
|
|
|
14 |
|
|
Trade and other receivables |
|
|
56 |
|
|
|
|
|
3 |
|
|
Prepaid and other assets |
|
|
774 |
|
|
|
|
|
741 |
|
Total current assets |
|
|
7,395 |
|
|
|
|
|
4,820 |
|
Property and equipment, net |
|
|
106 |
|
|
|
|
|
222 |
|
Deposits |
|
|
|
25 |
|
|
|
|
|
25 |
|
Other assets |
|
|
105 |
|
|
|
|
|
135 |
|
Right-of-use assets |
|
|
589 |
|
|
|
|
|
771 |
|
Total assets |
|
$ |
8,220 |
|
|
|
|
$ |
5,973 |
|
Liabilities and stockholders' equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
Trade and other payables |
|
$ |
2,455 |
|
|
|
|
$ |
1,880 |
|
|
Accrued employee benefits |
|
|
429 |
|
|
|
|
|
400 |
|
|
Lease liabilities, current portion |
|
|
269 |
|
|
|
|
|
252 |
|
Total current liabilities |
|
|
3,153 |
|
|
|
|
|
2,532 |
|
Lease liabilities, less current portion |
|
|
354 |
|
|
|
|
|
559 |
|
Total liabilities |
|
|
3,507 |
|
|
|
|
|
3,091 |
|
Commitments and contingencies (Note 11) |
|
|
|
|
|
|
Stockholders' equity: |
|
|
|
|
|
|
|
Common stock, $0.0001 par value-160,000,000 shares authorized;
27,981,161 shares and 8,171,690 shares issued and outstanding at
March 30, 2022 and June 30, 2022, respectively |
|
3 |
|
|
|
|
|
1 |
|
|
Additional paid-in capital |
|
|
168,791 |
|
|
|
|
|
152,453 |
|
|
Accumulated deficit |
|
|
(163,228 |
) |
|
|
|
|
(148,327 |
) |
|
Accumulated other comprehensive loss |
|
|
(853 |
) |
|
|
|
|
(1,245 |
) |
Total stockholders' equity |
|
|
4,713 |
|
|
|
|
|
2,882 |
|
Total liabilities and stockholders' equity |
|
$ |
8,220 |
|
|
|
|
$ |
5,973 |
|
|
|
|
|
|
|
|
|
The accompanying
notes are an integral part of these consolidated financial
statements |
|
|
|
|
|
|
|
|
BENITEC BIOPHARMA INC. |
Consolidated Statements of Operations and Comprehensive Loss |
(Unaudited) |
(in thousands, except share and per share amounts) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
Nine Months Ended |
|
|
March 31, |
|
March 31, |
|
|
|
2023 |
|
|
|
|
2022 |
|
|
|
2023 |
|
|
|
|
2022 |
|
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
Licensing revenues from customers |
$ |
54 |
|
|
|
$ |
48 |
|
|
$ |
68 |
|
|
|
$ |
73 |
|
Total revenues |
|
54 |
|
|
|
|
48 |
|
|
|
68 |
|
|
|
|
73 |
|
Operating Expenses |
|
|
|
|
|
|
|
|
|
|
Research and development |
|
3,167 |
|
|
|
|
2,171 |
|
|
|
9,588 |
|
|
|
|
8,096 |
|
|
General and administrative |
|
1,228 |
|
|
|
|
1,337 |
|
|
|
5,011 |
|
|
|
|
5,093 |
|
Total operating expenses |
|
4,395 |
|
|
|
|
3,508 |
|
|
|
14,599 |
|
|
|
|
13,189 |
|
Loss from operations |
|
(4,341 |
) |
|
|
|
(3,460 |
) |
|
|
(14,531 |
) |
|
|
|
(13,116 |
) |
Other income (loss): |
|
|
|
|
|
|
|
|
|
|
Foreign currency transaction gain (loss) |
|
(45 |
) |
|
|
|
229 |
|
|
|
(391 |
) |
|
|
|
36 |
|
|
Interest expense, net |
|
(7 |
) |
|
|
|
(10 |
) |
|
|
(25 |
) |
|
|
|
(22 |
) |
|
Other income, net |
|
- |
|
|
|
|
(29 |
) |
|
|
50 |
|
|
|
|
(29 |
) |
|
Unrealized loss on investment |
|
(4 |
) |
|
|
|
(5 |
) |
|
|
(4 |
) |
|
|
|
(10 |
) |
Total other income (loss), net |
|
(56 |
) |
|
|
|
185 |
|
|
|
(370 |
) |
|
|
|
(25 |
) |
Net loss |
|
$ |
(4,397 |
) |
|
|
$ |
(3,275 |
) |
|
$ |
(14,901 |
) |
|
$ |
(13,141 |
) |
Other comprehensive income: |
|
|
|
|
|
|
|
|
|
|
Unrealized foreign currency translation gain (loss) |
|
45 |
|
|
|
|
(233 |
) |
|
|
392 |
|
|
|
|
(51 |
) |
Total other comprehensive income (loss) |
|
45 |
|
|
|
|
(233 |
) |
|
|
392 |
|
|
|
|
(51 |
) |
Total comprehensive loss |
$ |
(4,352 |
) |
|
|
$ |
(3,508 |
) |
|
$ |
(14,509 |
) |
|
$ |
(13,192 |
) |
Net loss |
|
$ |
(4,397 |
) |
|
|
$ |
(3,275 |
) |
|
$ |
(14,901 |
) |
|
$ |
(13,141 |
) |
Net loss per share: |
|
|
|
|
|
|
|
|
|
Basic and diluted |
$ |
(0.16 |
) |
|
|
$ |
(0.40 |
) |
|
$ |
(0.67 |
) |
|
|
$ |
(1.61 |
) |
Weighted average number of shares outstanding: basic and
diluted |
|
27,981,161 |
|
|
|
|
8,171,690 |
|
|
|
22,090,191 |
|
|
|
|
8,171,690 |
|
|
|
|
|
|
|
|
|
|
|
|
The accompanying notes are an integral part of these consolidated
financial statements. |
|
|
|
|
|
|
|
|
|
|
|
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
Grafico Azioni Benitec Biopharma (NASDAQ:BNTC)
Storico
Da Dic 2024 a Gen 2025
Grafico Azioni Benitec Biopharma (NASDAQ:BNTC)
Storico
Da Gen 2024 a Gen 2025