HAYWARD, Calif., Feb. 14, 2022 /PRNewswire/ -- Benitec
Biopharma Inc. (NASDAQ: BNTC) ("Benitec" or "the Company"), a
development-stage, gene therapy-focused, biotechnology company
developing novel genetic medicines based on the proprietary
DNA-directed RNA interference ("ddRNAi") platform, today announced
the financial results for its Fiscal Year Q2 ended December 31, 2021. The Company has filed its
quarterly report on Form 10-Q for the quarter ended December 31, 2021, with the U.S. Securities and
Exchange Commission.
Operational Updates
The key milestones related to the investigational agents under
development by the Company and other corporate updates are outlined
below:
Regulatory Interactions:
- Benitec successfully completed the regulatory interactions
required to support initiation of the BB-301 clinical development
program in 2022
- Successful regulatory engagement comprised the completion of
the following meetings:
-
- Pre-Clinical Trial Application (Pre-CTA) Consultation Meeting
with Health Canada
- Scientific Advice Meeting with The National Agency for the
Safety of Medicines and Health Products in France (L'Agence nationale de sécurité du
médicament et des produits de santé or "ANSM")
- Type C Meeting with the U.S. Food and Drug Administration
("FDA")
BB-301 Clinical Development Program:
- The BB-301 clinical development program will begin in mid-2022,
and the conduct of the development program will comprise
approximately 76-weeks of follow-up for each Oculopharyngeal
Muscular Dystrophy (OPMD) study participant, inclusive of :
-
- 6-month pre-treatment observation periods for evaluation of the
baseline disposition and natural history of OPMD-derived dysphagia
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 Study will begin in mid-2022 and this
observational study will facilitate the characterization of OPMD
patient disposition at baseline and assess subsequent rates of
progression of dysphagia (swallowing impairment) in subjects with
OPMD via the use of:
-
- Quantitative radiographic measures of global swallowing
function and pharyngeal constrictor muscle force generation
-
- Inclusive of Videofluoroscopic Swallowing Studies (VFSS)
conducted to complete the following methodological
assessments:
-
- Dynamic Imaging Grade of Swallowing Toxicity Scale
(DIGEST)
- Pharyngeal Area at Maximum Constriction (PAMC)
- Pharyngeal Constriction Ratio (PCR)
- Clinical measures of global swallowing capacity and
oropharyngeal dysphagia
- Patient-reported measures of oropharyngeal dysphagia
- The natural history of dysphagia observed for each OPMD study
participant, as characterized by the quantitative measures and
clinical assessments outlined above, will serve as the baseline for
comparative assessment of safety and efficacy of BB-301 upon
rollover of OPMD patients from the Natural History Study onto the
Phase 1b/2a treatment study
- Upon the achievement of 6-months of follow-up in the Natural
History Study, OPMD study participants will become eligible for
enrollment onto the treatment study with the investigational
genetic medicine, BB-301, which uses an AAV9-based gene therapy
approach for the treatment of OPMD-derived dysphagia
-
- This first-in-human study (FIH) 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, and the primary
endpoints (safety and tolerability) and secondary endpoints
(comprising the quantitative radiographic measures of global
swallowing function and pharyngeal constrictor muscle force
generation and the clinical assessments noted above) will be
evaluated during each 90-day period following Day 0 (the day of
BB-301 intramuscular injection)
- On September 8, 2021 Benitec
provided key updates related to the primary and secondary endpoints
for the Phase 1b/2a BB-301 treatment
study
BB-301 Pre-Clinical Development Program:
- On September 8, 2021 Benitec
provided key updates related to the progress of the BB-301
pre-clinical development program, including updated results for the
BB-301 Pilot Dosing Study in large animals
Financial Highlights
Total Revenues for the quarter ended December 31, 2021 were twenty-five thousand
compared to one thousand dollars in
total revenue for three months ended December 31, 2020. The increase in revenues from
customers is due to the increase in licensing and royalty revenues
in the current period.
Total Operating Expenses were $4.9
million for the quarter ended December 31, 2021 compared to $3.2 million for the comparable period in 2020.
For the quarters ended December 31,
2021, and 2020, respectively, Benitec incurred $0 and $19 thousand
in royalties and license fees. During the three months ended
December 31, 2021 and 2020,
respectively, the Company incurred $3.2
million and $1.2 million in
research and development expenses. The increase in research and
development expenses is primarily related to the commencement of
the BB-301 Regulatory Toxicology Study in Beagle dogs at Charles
River Laboratories in Evreux, France. As planned, the Company began
incurring more costs related to the execution of two large
non-clinical studies in Beagle dogs, along with the
commercial-scale GMP-grade manufacturing of BB-301, all of which is
required to facilitate the Clinical Trial Application (CTA) filing
and the Investigational New Drug (IND) filing for BB-301 during
2022. General and administrative expenses were $1.7 million and $2.1
million for the three months ended December 31, 2021 and 2020, respectively. The
increase during this three-month period was due to increases in
costs related to insurance, consultant fees, legal fees, and
accounting fees.
Jerel A. Banks, M.D., Ph.D.,
Executive Chairman and Chief Executive Officer of Benitec Biopharma
commented, "The robust biological activity observed for BB-301 in
our Pilot Dosing Study underpins our enthusiasm for the initiation
of the BB-301 clinical development program in 2022. We remain
dedicated to our central goal of improving the lives of patients
with OPMD."
BENITEC
BIOPHARMA INC.
Consolidated Balance Sheets
(in thousands, except par value and share amounts)
|
|
|
|
|
|
December
31,
2021
|
|
June 30,
2021
|
|
(Unaudited)
|
|
|
Assets
|
|
|
|
Current
assets:
|
|
|
|
Cash and cash equivalents
|
$
12,329
|
|
$
19,769
|
Trade and other receivables
|
5
|
|
25
|
Prepaid and other assets
|
338
|
|
814
|
Total current assets
|
12,672
|
|
20,608
|
Property and
equipment, net
|
268
|
|
375
|
Deposits
|
25
|
|
9
|
Other assets
|
161
|
|
185
|
Right-of-use assets
|
888
|
|
202
|
Total assets
|
$ 14,
014
|
|
$
21,379
|
Liabilities and
Stockholders' Equity
|
|
|
|
Current
liabilities:
|
|
|
|
Trade and other payables
|
$
1,946
|
|
$
880
|
Accrued employee benefits
|
321
|
|
276
|
Lease liabilities, current
portion
|
213
|
|
213
|
Total current
liabilities
|
2,480
|
|
1,369
|
Lease liabilities,
less current portion
|
698
|
|
—
|
Total liabilities
|
3,178
|
|
1,369
|
Commitments and
contingencies (Note 10)
|
|
|
|
Stockholders' equity:
|
|
|
|
Common stock, $0.0001 par
value-40,000,000 shares authorized; 8,171,690 shares issued and
outstanding at December 31, 2021 and June 30, 2021
|
1
|
|
1
|
Additional paid-in capital
|
152,093
|
|
151,583
|
Accumulated deficit
|
(139,985)
|
|
(130,119)
|
Accumulated other
comprehensive loss
|
(1,273)
|
|
(1,455)
|
Total stockholders'
equity
|
10,836
|
|
20,010
|
Total liabilities and
stockholders' equity
|
$
14,014
|
|
$
21,379
|
BENITEC
BIOPHARMA INC.
|
Consolidated
Statements of Operations and Comprehensive Loss
|
(Unaudited)
|
(in thousands,
except share and per share amounts)
|
|
|
Three Months
Ended
December 31,
|
|
Six Months
Ended
December 31,
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
Revenue:
|
|
|
|
|
|
|
|
Licensing
revenues from customers
|
$
25
|
|
$
1
|
|
$
25
|
|
$
56
|
Total
revenues
|
25
|
|
1
|
|
25
|
|
56
|
Operating
expenses
|
|
|
|
|
|
|
|
Royalties and license
fees
|
—
|
|
(19)
|
|
—
|
|
114
|
Research and
development
|
3,146
|
|
1,168
|
|
5,926
|
|
1,942
|
General and
administrative
|
1,714
|
|
2,111
|
|
3,756
|
|
3,948
|
Total operating
expenses
|
4,860
|
|
3,260
|
|
9,682
|
|
6,004
|
Loss from
operations
|
(4,835)
|
|
(3,259)
|
|
(9,657)
|
|
(5,948)
|
Other income
(loss):
|
|
|
|
|
|
|
|
Foreign currency
transaction gain (loss)
|
48
|
|
—
|
|
(193)
|
|
(54)
|
Interest expense,
net
|
(11)
|
|
(2)
|
|
(12)
|
|
(3)
|
Other income,
net
|
—
|
|
10
|
|
—
|
|
36
|
Unrealized loss on
investment
|
(23)
|
|
(1)
|
|
(5)
|
|
(1)
|
Total other income
(loss), net
|
14
|
|
7
|
|
(210)
|
|
(22)
|
Net loss
|
$
(4,821)
|
|
$
(3,252)
|
|
$
(9,867)
|
|
$
(5,970)
|
Other comprehensive
income:
|
|
|
|
|
|
|
|
Unrealized foreign
currency translation (loss) gain
|
(57)
|
|
208
|
|
182
|
|
386
|
Total other
comprehensive (loss) income
|
(57)
|
|
208
|
|
182
|
|
386
|
Total comprehensive
loss
|
$
(4,878)
|
|
$
(3,044)
|
|
$
(9,685)
|
|
$
(5,584)
|
Net loss
|
$
(4,821)
|
|
$
(3,252)
|
|
$
(9,867)
|
|
$
(5,970)
|
Net loss per
share:
|
|
|
|
|
|
|
|
Basic and
diluted
|
$
(0.59)
|
|
$
(0.76)
|
|
$
(1.21)
|
|
$
(2.21)
|
Weighted average number
of shares outstanding: basic and diluted
|
8,171,690
|
|
4,300,073
|
|
8,171,690
|
|
2,704,223
|
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; 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.
Media & Investor Relations Contact:
Jay A. Morakis
CEO of M Group Strategic Communications (for Benitec Biopharma,
Inc.)
Phone: 646-859-5951
Email: jmorakis@mgroupsc.com
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SOURCE Benitec Biopharma Inc.