Benitec Biopharma Releases First Quarter 2024 Financial Results and Provides Operational Update
November 13 2023 - 8:00AM
Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a
clinical-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 First Fiscal Quarter ended September 30,
2023. The Company has filed its quarterly report on Form 10-Q for
the quarter ended September 30, 2023, with the U.S. Securities and
Exchange Commission.
“With the receipt of FDA clearance for the
BB-301 IND application this year and the pace of enrollment into
the OPMD Natural History Study continuing to exceed our
expectations, we are well positioned to begin the evaluation of
BB-301 in subjects diagnosed with Oculopharyngeal Muscular
Dystrophy,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman
and Chief Executive Officer of Benitec. “The Principal Investigator
and the clinical team at the U.S. clinical trial site are
finalizing the plan to dose the first study subject with BB-301 in
December 2023 or January 2024. The strong interest from members of
the Oculopharyngeal Muscular Dystrophy community, and the
exceptional clinical trial execution by the U.S. clinical trial
site, will support the generation of robust safety and efficacy
data for BB-301 over the coming months.”
Operational Updates
The key milestones related to the development of
BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy
(OPMD)-related Dysphagia, along with other corporate updates, are
outlined below:
BB-301 Clinical Development Program
Overview:
- The BB-301 Phase 1b/2a clinical
development program will be conducted in the United States and
Canada, 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-related 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 constrictor 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 Clinical 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 OPMD 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 OPMD NH Study, participants will, potentially, be
eligible for enrollment into the BB-301 Phase 1b/2a Clinical
Treatment Study.
- BB-301 Phase 1b/2a Clinical
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-related
Dysphagia.
- 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 OPMD NH study to facilitate
comparative clinical and statistical analyses for each study
subject.
- 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 OPMD NH
Study, will serve as the baseline for comparative assessments of
safety and efficacy of BB-301 upon rollover from the OPMD NH Study
onto the BB-301 Phase 1b/2a Clinical Treatment Study.
Corporate Updates:
- 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 OPMD NH Study in
the United States.
- As of October 2023, 19 subjects
have been enrolled into the OPMD NH study in the United
States.
- The pace of enrollment of OPMD
subjects into the OPMD NH Study at the U.S. clinical trial site
supports our central clinical development goals of: (1) initiating
the clinical dosing of BB-301 to OPMD subjects in December 2023 or
January 2024, and (2) disclosing the initial interim safety and
efficacy data over the next 12 months for subjects that have
received BB-301.
Regulatory Updates for the Clinical
Development Program:
North America:
- Formal submission of the comprehensive OPMD 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 OPMD NH Study trial package by the REB is
required for clinical study site activation and OPMD patient
screening and enrollment to begin in Canada.
Financial Highlights
First Quarter 2024 Financial Results
Total Revenues for the quarter ended September
30, 2023, were $0 compared to $0 for the quarter ended September
30, 2022.
Total Expenses for the quarter ended September
30, 2023 were $5.9 million compared to $4.6 million for the quarter
ended September 30, 2022. For the quarter ended September 30, 2023,
the Company received royalties and license fee credits of $106
thousand compared to $0 for the three months ended September 30,
2022. The credit to expense during the quarter ended September 30,
2023, reflects the reversal of an accrual for license fees no
longer due. The Company incurred $4.4 million of research and
development expenses compared to $2.7 million for the comparable
quarter ended September 30, 2022. Research and development expenses
relate primarily to ongoing clinical development of BB-301 for the
treatment of OPMD. General and administrative expenses were $1.6
million compared to $1.9 million for the quarter ended September
30, 2022.
The loss from operations for the quarter ended
September 30, 2023, was $5.9 million compared to a loss of $4.6
million for the quarter ended September 30, 2022. Net loss
attributable to shareholders for the quarter ended September 30,
2023, was $6 million, or $2.76 per basic and diluted share,
compared to a net loss of $5.1 million, or $7.98 per basic and
diluted share for the quarter ended September 30, 2022. As of
September 30, 2023, the Company had $25.9 million in cash and cash
equivalents.
BENITEC BIOPHARMA INC. |
Consolidated Balance Sheets |
(in thousands, except par value and share amounts) |
|
|
|
|
|
|
|
September 30, |
|
June 30, |
|
|
|
2023 |
|
|
|
2023 |
|
|
|
(Unaudited) |
|
|
Assets |
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
$ |
25,864 |
|
|
$ |
2,477 |
|
|
Restricted cash |
|
13 |
|
|
|
13 |
|
|
Trade and other receivables |
|
53 |
|
|
|
55 |
|
|
Prepaid and other assets |
|
482 |
|
|
|
1,184 |
|
Total current assets |
|
26,412 |
|
|
|
3,729 |
|
Property and equipment, net |
|
68 |
|
|
|
87 |
|
Deposits |
|
25 |
|
|
|
25 |
|
Prepaid and other assets |
|
85 |
|
|
|
97 |
|
Right-of-use assets |
|
463 |
|
|
|
526 |
|
Total assets |
$ |
27,053 |
|
|
$ |
4,464 |
|
Liabilities and stockholders' equity |
|
|
|
Current liabilities: |
|
|
|
|
Trade and other payables |
$ |
3,794 |
|
|
$ |
3,231 |
|
|
Accrued employee benefits |
|
460 |
|
|
|
472 |
|
|
Lease liabilities, current portion |
|
280 |
|
|
|
275 |
|
Total current liabilities |
|
4,534 |
|
|
|
3,978 |
|
Lease liabilities, less current portion |
|
211 |
|
|
|
284 |
|
Total liabilities |
|
4,745 |
|
|
|
4,262 |
|
Commitments and contingencies (Note 10) |
|
|
|
Stockholders' equity: |
|
|
|
|
Common stock, $0.0001 par value-160,000,000 shares authorized;
2,547,434 shares and 1,671,485 shares issued |
|
- |
|
|
|
- |
|
|
and outstanding at September 30, 2023 and June 30, 2023,
respectively |
|
|
|
|
|
|
|
|
Additional paid-in capital |
|
196,931 |
|
|
|
168,921 |
|
|
Accumulated deficit |
|
(173,843 |
) |
|
|
(167,889 |
) |
|
Accumulated other comprehensive loss |
|
(780 |
) |
|
|
(830 |
) |
Total stockholders' equity |
|
22,308 |
|
|
|
202 |
|
Total liabilities and stockholders' equity |
$ |
27,053 |
|
|
$ |
4,464 |
|
|
|
|
|
|
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 September 30, |
|
|
|
2023 |
|
|
|
2022 |
|
Revenue: |
|
|
|
|
Licensing revenues from customers |
|
|
|
Total revenues |
$ |
- |
|
|
$ |
- |
|
Operating Expenses |
|
|
|
|
Royalties and license fees |
|
(106 |
) |
|
|
- |
|
|
Research and development |
|
4,429 |
|
|
|
2,660 |
|
|
General and administrative |
|
1,551 |
|
|
|
1,920 |
|
Total operating expenses |
|
5,874 |
|
|
|
4,580 |
|
Loss from operations |
|
(5,874 |
) |
|
|
(4,580 |
) |
Other income (loss): |
|
|
|
|
Foreign currency transaction loss |
|
(56 |
) |
|
|
(507 |
) |
|
Interest expense, net |
|
(6 |
) |
|
|
(9 |
) |
|
Other expense, net |
|
(18 |
) |
|
|
- |
|
|
Unrealized gain on investment |
|
- |
|
|
|
3 |
|
Total other loss, net |
|
(80 |
) |
|
|
(513 |
) |
Net loss |
$ |
(5,954 |
) |
|
$ |
(5,093 |
) |
Other comprehensive income: |
|
|
|
|
Unrealized foreign currency translation gain |
|
50 |
|
|
|
507 |
|
Total other comprehensive income |
|
50 |
|
|
|
507 |
|
Total comprehensive loss |
$ |
(5,904 |
) |
|
$ |
(4,586 |
) |
Net loss |
$ |
(5,954 |
) |
|
$ |
(5,093 |
) |
Net loss per share: basic and diluted |
$ |
(2.76 |
) |
|
$ |
(7.98 |
) |
Weighted average number of shares outstanding: basic and
diluted |
|
2,157,065 |
|
|
|
638,572 |
|
|
|
|
|
|
The accompanying notes are an integral part of these consolidated
financial statements. |
|
|
|
|
|
About BB-301
BB-301 is a novel, modified AAV9 capsid
expressing a unique, single bifunctional construct promoting
co-expression of both codon-optimized Poly-A Binding Protein
Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against
mutant PABPN1. The two siRNAs are modeled into microRNA backbones
to silence expression of faulty mutant PABPN1, while allowing
expression of the codon-optimized PABPN1 to replace the mutant with
a functional version of the protein. We believe BB-301’s silence
and replace mechanism is uniquely positioned for the treatment of
OPMD by halting mutant expression while providing a functional
replacement protein.
About Benitec Biopharma
Inc.
Benitec Biopharma Inc. (“Benitec” or the
“Company”) is a clinical-stage biotechnology company focused on the
advancement of novel genetic medicines with headquarters in
Hayward, California. The proprietary DNA-directed RNA interference
“Silence and Replace” platform combines RNA interference, or RNAi,
with gene therapy to create medicines that simultaneously
facilitate sustained silencing of disease-causing genes and
concomitant delivery of wildtype replacement genes following a
single administration of the therapeutic construct. The Company is
developing Silence and Replace-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 enrollment
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 COVID-19 pandemic, the
disease caused by the SARS-CoV-2 virus and similar events, 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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