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Benitec Biopharma Releases Full Year 2024 Financial Results and Provides Operational Update -Positive 90-day and 180-day Interim Clinical Trial Data for the First Oculopharyngeal Muscular Dystrophy (OPMD) Subject Dosed w

Key Takeaway: Benitec Biopharma has released its financial results for the full year 2024, highlighting positive interim clinical trial data for the BB-301 treatment in treating Oculopharyngeal Muscular Dystrophy (OPMD). The company successfully dosed a second subject and anticipates a third by the end of 2024. Despite delivering promising clinical updates, Benitec reported zero revenues for the year, reflecting a significant drop from the previous year's figures. The company has extended its cash runway through 2025 following a successful oversubscribed financing round of $40 million.

Market Sentiment Analysis

POSITIVE FACTORS

  • Positive interim clinical trial data for the first subject dosed with BB-301.
  • Significant improvements in dysphagia-focused outcome measures reported.
  • The recent funding strengthens the company's financial position for future trials.

CONCERNS & RISKS

  • Total revenues declined to $0 million compared to $75,000 in the previous year.
  • The company reported an increased net loss of $22.4 million for the last year.

Full Press Release Details

Benitec Biopharma Releases Full Year 2024 Financial Results and Provides Operational Update
-Positive 90-day and 180-day Interim Clinical Trial Data for
the First Oculopharyngeal Muscular Dystrophy (OPMD) Subject Dosed with the Low-Dose of BB-301 in the Phase 1b/2a Clinical Treatment Study Reported in April and July-
-Second Subject Dosed with the Low-Dose of BB-301
in February 2024, and Third Subject Expected to Receive the Low-Dose of BB-301 in Calendar Quarter Four of 2024
-Closed an Oversubscribed Private Placement Financing of $40.0 Million on April
22nd, Cash Runway Extended Through 2025-
HAYWARD, Calif., September 26, 2024
(GLOBE NEWSWIRE) Benitec Biopharma Inc. (NASDAQ: BNTC) ( Benitec or the Company ), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary Silence
and Replace DNA-directed RNA interference ( ddRNAi ) platform, today announced financial results for its full year ended June 30, 2024. The Company has filed its annual report on Form 10-K with the U.S. Securities and Exchange Commission.
The 90-day and 180-day interim clinical study results for the first subject enrolled into the low-dose cohort of the BB-301 Phase 1b/2a Clinical
Treatment Study demonstrated clinically meaningful improvements in the central study endpoints, with significant improvements noted across the radiographic assessments of swallowing efficiency and corresponding improvements observed for the key
dysphagia-focused subject-reported outcome measure, said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. Additional clinical data for the BB-301 Phase 1b/2a
Clinical Treatment Study were accepted for oral presentation during the Late Breaking session of the 29th Annual Congress of the World Muscle Society on October 12, 2024. The recent financing
comfortably positions Benitec to advance the BB-301 clinical development program through the end of 2025.
related to the development of BB-301 for the treatment of OPMD-related Dysphagia, are outlined below:
Interim BB-301 Phase 1b/2a Clinical Treatment Study Results
Background Information Regarding the Key Clinical Assessments and Radiographic Outcome Measures Employed in the BB-301 Phase 1b/2a:
90-Day Post-Dose Interim Clinical Study Results:
180-Day Post-Dose Interim Clinical Study Results:
Continued Enrollment for the BB-301 Phase 1b/2a Clinical
Safety and Tolerability:
Financial Highlights
Full Year 2024 Financial Results
Total Revenues for the
year ended June 30, 2024, were $0 million compared to $75,000 in licensing revenues collected for the year ended June 30, 2023.
Expenses for the year ended June 30, 2024, were $22.5 million compared to $19.2 million for the year ended June 30, 2023. For the year ended June 30, 2024, the Company incurred ($108,000) in royalties and license fees due to
the reversal of an accrual for the year ended June 30, 2024 compared to $0 for the year ended June 30, 2023. The Company incurred $15.6 million of research and development expenses for the year ended June 30, 2024 compared to
$12.8 million for the year ended June 30, 2023. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD. General and administrative
expenses were $7.0 million for the year ended June 30, 2024 compared to $6.4 million for the year ended June 30, 2023.
operations for the year ended June 30, 2024, was $21.8 million compared to a loss of $19.6 million for the year ended June 30, 2023. Net loss attributable to shareholders for the year ended June 30, 2024, was
$22.4 million, or $5.51 per basic and diluted share, compared to a net loss of $19.6 million, or $14.12 per basic and diluted share for the year ended June 30, 2023. As of June 30, 2024, the Company had $50.9 million in cash
and cash equivalents.
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 causative gene for OPMD). 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 the silence and replace mechanism of BB-301 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 Silence and Replace DNA-directed RNA interference 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 completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical
trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec s product candidates, 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: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of
preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and
approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of
ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a one shot cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future
revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors, including our capital structure; the length of time over which we expect our cash and cash
equivalents to be sufficient to execute on our business plan; 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 and other regulatory developments; 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 impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; 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:
LifeSci Advisors, LLC
BENITEC BIOPHARMA INC.
Consolidated Balance Sheets
(in thousands, except par value and share amounts)
June 30, 2024 June 30, 2023
Assets
Current assets:
Cash and cash equivalents $ 50,866 $ 2,477
Restricted Cash 63 13
Trade and other receivables 229 55
Prepaid and other assets 516 1,184
Total current assets 51,674 3,729
Property and equipment, net 179 87
Deposits 25 25
Other assets 62 97
Right-of-use assets 270 526
Total assets $ 52,210 $ 4,464
Liabilities and Stockholders Equity
Current liabilities:
Trade and other payables $ 4,165 $ 3,231
Accrued employee benefits 475 472
Lease liabilities, current portion 284 275
Total current liabilities 4,924 3,978
Non-current accrued employee benefits 38
Lease liabilities, less current portion 284
Total liabilities 4,962 4,262
Commitments and contingencies (Note 10)
Stockholders equity:
Common stock, $0.0001 par value - 160,000,000 shares authorized; 10,086,119 and 1,671,485 shares issued and outstanding at June 30, 2024 and 2023, respectively 1
Additional paid-in capital 238,398 168,921
Accumulated deficit (190,259 ) (167,889 )
Accumulated other comprehensive loss (892 ) (830 )
Total stockholders equity 47,248 202
Total liabilities and stockholders equity $ 52,210 $ 4,464
The accompanying notes are an integral part of these consolidated financial statements.
BENITEC BIOPHARMA INC.
Consolidated Statements of Operations and Comprehensive Loss
(in thousands, except share and per share amounts)
Year Ended
June 30,
2024 2023
Revenue:
Licensing revenues from customers $ $ 75
Total revenues 75
Operating expenses
Royalties and license fees (108 )
Research and development 15,609 12,774
General and administrative 6,989 6,382
Total operating expenses 22,490 19,156
Loss from operations (22,490 ) (19,081 )
Other income (loss):
Foreign currency transaction gain (loss) 40 (415 )
Interest income (expense), net 904 (33 )
Other income (expense), net (204 ) (30 )
Unrealized gain (loss) on investment (1 ) (3 )
Total other income (loss), net 739 (481 )
Provsion for income tax
Net loss $ (21,751 ) $ (19,562 )
Other comprehensive income:
Unrealized foreign currency translation gain (loss) (62 ) 415
Total other comprehensive income (62 ) 415
Total comprehensive loss $ (21,813 ) $ (19,147 )
Net loss $ (21,751 ) $ (19,562 )
Deemed dividends (619 ) $
Net loss attributable to common shareholders $ (22,370 ) $ (19,562 )
Net loss per share:
Basic and diluted $ (6 ) $ (14 )
Weighted average number of shares outstanding: basic and diluted 4,060,182 1,385,818
The accompanying notes are an integral part of these consolidated financial statements.

Frequently Asked Questions

What were the key findings from the BB-301 clinical trial?

Interim results showed clinically significant improvements in swallowing efficiency for OPMD subjects.

How much financing did Benitec secure in 2024?

Benitec closed an oversubscribed private placement totaling $40 million in April 2024.

What was Benitec's revenue for the fiscal year 2024?

Benitec reported total revenues of $0 million for the year ending June 30, 2024.

What are research and development expenses for 2024?

Research and development expenses amounted to $15.6 million for the year ended June 30, 2024.

What is BB-301's mechanism of action?

BB-301 uses a unique construct to silence mutant PABPN1 while providing functional protein replacement.

Last updated: Sep 26, 2024