Full Press Release Details
Benitec Biopharma Releases First Quarter 2025 Financial Results and Provides Operational Update
-Positive 270-day Interim Clinical Study Data for the First Subject and Positive 180-day Interim Clinical Study Data for the Second Subject Treated with the Low-Dose of BB-301 in the Phase 1b/2a Clinical Treatment
Study Reported in October as a Late-Breaking Oral Presentation at the 29th Annual Congress of the World Muscle Society-
-Third Subject Safely Treated with the Low-Dose of BB-301 in
October 2024, and Fourth Subject Expected to Receive Treatment with the Low-Dose of BB-301 in December 2024
HAYWARD, Calif., November 14, 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 first fiscal quarter ended September 30, 2024. The Company has filed its quarterly report on Form 10-Q for the quarter ended September 30, 2024
with the U.S. Securities and Exchange Commission (SEC).
We recently announced the 270-day interim clinical
study results for the first Subject and the 180-day interim clinical study results for the second Subject enrolled into the low-dose cohort of the BB-301 Phase 1b/2a Clinical Treatment Study. We continue to be extremely grateful for the strong support of the Subjects and their families and for their continued participation in the
BB-301 clinical development program. We were highly encouraged by the significant, clinically meaningful improvements observed for both Subjects treated at the low-dose
of BB-301, with Subject 1 experiencing durable improvements in core dysphagic symptoms of 35% to 40%, and Subject 2 achieving a clinically normal swallowing profile based on the results of the Sydney Swallow
Questionnaire, said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. The third Subject was treated with the low-dose of
BB-301 in October 2024, and we anticipate the treatment of the fourth Subject in December 2024. We remain optimistic about the potential for continued benefit in Subjects enrolled in the ongoing study and,
including funds from recent exercises of shareholder-held warrants, Benitec is well-funded to advance the BB-301 clinical development program. We look forward to enrolling additional Subjects at the next,
higher dose of BB-301, in 2025.
The key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy
(OPMD)-related Dysphagia, are outlined below:
Summary of Interim Clinical Study Results for Subject 1 and Subject 2:
Subjects Enrolled in the BB-301 Clinical Development Program
are Impacted by Two Discrete Drivers of Dysphagic Symptoms:
Interim Clinical Study
Results for Subject 1 (270-Days Post Treatment with BB-301):
Clinical Study Results for Subject 2 (180-Days Post Treatment with BB-301):
Both Subjects were blinded to their SSQ Total
Scores and VFSS TPR assessment results, and the Central Reader for the VFSS assessments was blinded to the SSQ Total Scores and SSQ Sub-Scores for each Subject.
Enrollment into the BB-301 Phase 1b/2a Clinical Treatment Study is Ongoing:
Financial Highlights
First Quarter 2025 Financial Results
Revenues for the quarter ended September 30, 2024, were $0 million compared to $0 revenues collected for the quarter ended September 30, 2023.
Total Expenses for the quarter ended September 30, 2024, were $5.8 million compared to $5.9 million for the quarter ended September 30,
2023. For the quarter ended September 30, 2024, the Company received no royalties and license fees compared to a royalties and license fee credit of $106,000 for the quarter ended September 30, 2023. The Company incurred $3.6 million
of research and development expenses for the quarter ended September 30, 2024 compared to $4.4 million for the quarter ended September 30, 2023. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD-related Dysphagia. General and administrative expenses were $2.2 million for the quarter ended September 30, 2024 compared to $1.6 million for the quarter ended
The loss from operations for the quarter ended September 30, 2024, was $5.2 million compared to a loss of
$5.9 million for the quarter ended September 30, 2023. Net loss attributable to shareholders for the quarter ended September 30, 2024, was $5.1 million, or $(0.48) per basic and diluted share, compared to a net loss of
$6.6 million, or $(3.05) per basic and diluted share for the quarter ended September 30, 2023. As of September 30, 2024, the Company had $67.8 million in cash and cash equivalents.
BENITEC BIOPHARMA INC.
Consolidated Balance Sheets
(in thousands, except par value and share amounts)
| September 30, | June 30, | |||||||
| 2024 | 2024 | |||||||
| (Unaudited) | ||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 67,841 | $ | 50,866 | ||||
| Restricted Cash | 64 | 63 | ||||||
| Trade and other receivables | 4 | 229 | ||||||
| Prepaid and other assets | 426 | 516 | ||||||
| Total current assets | 68,335 | 51,674 | ||||||
| Property and equipment, net | 154 | 179 | ||||||
| Deposits | 25 | 25 | ||||||
| Other assets | 56 | 62 | ||||||
| Right-of-use assets | 204 | 270 | ||||||
| Total assets | $ | 68,774 | $ | 52,210 | ||||
| Liabilities and Stockholders Equity | ||||||||
| Current liabilities: | ||||||||
| Trade and other payables | $ | 3,847 | $ | 4,165 | ||||
| Accrued employee benefits | 495 | 475 | ||||||
| Lease liabilities, current portion | 211 | 284 | ||||||
| Total current liabilities | 4,553 | 4,924 | ||||||
| Non-current accrued employee benefits | 41 | 38 | ||||||
| Lease liabilities, less current portion | ||||||||
| Total liabilities | 4,594 | 4,962 | ||||||
| Commitments and contingencies (Note 10) | ||||||||
| Stockholders equity: | ||||||||
| Common stock, $0.0001 par value - 160,000,000 shares authorized; 17,893,765 and 10,086,119 shares issued and outstanding at September 30, 2024 and June 30, 2024, respectively | 1 | 1 | ||||||
| Additional paid-in capital | 260,490 | 238,398 | ||||||
| Accumulated deficit | (195,318 | ) | (190,259 | ) | ||||
| Accumulated other comprehensive loss | (993 | ) | (892 | ) | ||||
| Total stockholders equity | 64,180 | 47,248 | ||||||
| Total liabilities and stockholders equity | $ | 68,774 | $ | 52,210 |
BENITEC BIOPHARMA INC.
Consolidated Statements of Operations and Comprehensive Loss
(in thousands, except share and per share amounts)
| Three Months Ended September 30, | ||||||||
| 2024 | 2023 | |||||||
| Revenue: | ||||||||
| Licensing revenues from customers | $ | $ | ||||||
| Total revenues | ||||||||
| Operating expenses | ||||||||
| Royalties and license fees | (106 | ) | ||||||
| Research and development | 3,585 | 4,429 | ||||||
| General and administrative | 2,206 | 1,551 | ||||||
| Total operating expenses | 5,791 | 5,874 | ||||||
| Loss from operations | (5,791 | ) | (5,874 | ) | ||||
| Other income (loss): | ||||||||
| Foreign currency transaction gain (loss) | 93 | (56 | ) | |||||
| Interest income (expense), net | 604 | (6 | ) | |||||
| Other income (expense), net | 35 | (18 | ) | |||||
| Unrealized gain (loss) on investment | ||||||||
| Total other income (loss), net | 732 | (80 | ) | |||||
| Net loss | $ | (5,059 | ) | $ | (5,954 | ) | ||
| Other comprehensive income: | ||||||||
| Unrealized foreign currency translation gain (loss) | (101 | ) | 50 | |||||
| Total other comprehensive income | (101 | ) | 50 | |||||
| Total comprehensive loss | $ | (5,160 | ) | $ | (5,904 | ) | ||
| Net loss | $ | (5,059 | ) | $ | (5,954 | ) | ||
| Deemed dividends | (619 | ) | ||||||
| Net loss attributable to common shareholders | $ | (5,059 | ) | $ | (6,573 | ) | ||
| Net loss per share: | ||||||||
| Basic and diluted | $ | (0.48 | ) | $ | (3.05 | ) | ||
| Weighted average number of shares outstanding: basic and diluted | 10,644,533 | 2,157,065 |
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