Benitec Biopharma Releases Full Year 2025 Financial Results and Provides Operational Update

Rhea-AI Summary

Benitec Biopharma (NASDAQ: BNTC) released its full year 2025 financial results and provided updates on its BB-301 clinical program for OPMD treatment. The company reported a net loss of $37.9 million ($1.05 per share) compared to $22.4 million in 2024. Total expenses increased to $41.8 million, with R&D expenses at $18.3 million and G&A expenses at $23.4 million.

Key operational highlights include a favorable DSMB recommendation to continue enrollment into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study, expected to begin in Q4 2025. The company maintains a strong financial position with $97.7 million in cash and cash equivalents as of June 30, 2025. Interim clinical results for Cohort 1 are anticipated in Q4 2025.

Positive

• Strong cash position of $97.7 million, up from $50.9 million in 2024
• Favorable DSMB recommendation to continue enrollment into Cohort 2
• Successful completion of Cohort 1 enrollment with six subjects

Negative

• Net loss increased to $37.9 million from $22.4 million year-over-year
• Total expenses nearly doubled to $41.8 million from $22.5 million
• G&A expenses significantly increased to $23.4 million from $7.0 million

Insights

Biotechnology Investment Analyst

Benitec shows widening losses despite strong cash position; interim BB-301 trial results for OPMD expected in Q4.

Benitec Biopharma's latest financial results reveal some critical developments for this clinical-stage gene therapy company. The company reported significantly increased operating expenses of $41.8 million for FY2025, up from $22.5 million in the previous year. This 85.8% increase in expenses was driven primarily by a substantial rise in general and administrative costs, which jumped from $7.0 million to $23.4 million – with $14.5 million of this increase attributed to share-based compensation.

Research and development expenses also increased to $18.3 million from $15.6 million, reflecting ongoing investment in the BB-301 clinical program for Oculopharyngeal Muscular Dystrophy (OPMD). The net loss attributable to shareholders widened to $37.9 million ($1.05 per share) from $22.4 million ($1.22 per share) in the prior year, though the per-share loss decreased due to a significantly larger share count.

The company's cash position appears strong at $97.7 million, nearly doubled from $50.9 million at the end of FY2024, providing an important runway for their clinical development programs. This enhanced liquidity should support operations through critical milestones for their lead candidate.

On the operational front, Benitec has completed enrollment for Cohort 1 of the BB-301 Phase 1b/2a treatment study, with the Independent Data Safety Monitoring Board recommending continuation to Cohort 2. The company expects to begin enrolling subjects in Cohort 2 in Q4 2025, while also releasing interim clinical results from Cohort 1 in the same timeframe. These upcoming data points represent critical catalysts that could validate Benitec's proprietary "Silence and Replace" ddRNAi platform technology.

BB-301's unique mechanism, which aims to silence mutant PABPN1 gene expression while replacing it with functional protein, represents a potentially transformative approach for OPMD patients suffering from dysphagia (swallowing difficulties). The continued advancement of this clinical program signals technical feasibility of their platform, though efficacy data remains pending with the anticipated Q4 readout.

Clinical Trial Research Specialist

Benitec's BB-301 gene therapy advancing through clinical stages with safety confirmed in first cohort; efficacy data pending in Q4.

The progression of Benitec's BB-301 Phase 1b/2a treatment study represents a significant milestone in addressing Oculopharyngeal Muscular Dystrophy (OPMD), a rare genetic condition characterized by progressive dysphagia (difficulty swallowing). The favorable safety assessment from the Independent Data Safety Monitoring Board (DSMB) following complete enrollment of Cohort 1 is particularly noteworthy, as it addresses the primary concern for novel gene therapies – safety profile.

The DSMB's recommendation to continue enrollment into Cohort 2 indicates no unexpected safety signals were observed in the first six subjects receiving BB-301. This is a critical hurdle cleared in early-phase gene therapy development, as safety concerns can often derail promising candidates. The phase 1b/2a trial design with sequential cohorts suggests a dose-escalation or parameter-optimization approach typical for gene therapy studies.

BB-301's dual-action mechanism employing DNA-directed RNA interference (ddRNAi) is scientifically elegant – simultaneously silencing the mutant PABPN1 gene while providing a functional replacement protein. This "Silence and Replace" approach differentiates it from traditional gene therapies that typically only add functional genes without addressing the mutant protein's potential toxic effects.

The modified AAV9 vector being used is a rational choice for this indication, as this serotype has demonstrated efficiency in transducing muscle tissue in previous clinical applications. The bifunctional construct design with codon-optimized PABPN1 expression and two small inhibitory RNAs targeting mutant PABPN1 reflects sophisticated vector engineering.

The anticipated interim clinical results in Q4 2025 will be the first opportunity to assess potential efficacy signals, likely focusing on biomarkers and functional measures of swallowing improvement. These data will be crucial for determining whether this mechanistically sound approach translates to clinical benefit. The continuation to Cohort 2 enrollment also suggests potential dose-finding or expanded patient populations being evaluated, a logical progression in the clinical development pathway.

Interim Clinical Study Results for Cohort 1 of the BB-301 Phase 1b/2a Treatment Study Anticipated in Q4 2025

Enrollment of the First Subject into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study Expected in Q4 2025

HAYWARD, Calif., Sept. 22, 2025 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “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, 2025. The Company has filed its annual report on Form 10-K with the U.S. Securities and Exchange Commission.

“We remain incredibly thankful for our continued close collaboration with families, clinical researchers, and healthcare providers as we advance the BB-301 clinical development program. With increasing durations of clinical follow-up for Subjects enrolled into Cohort 1 of the BB-301 Phase 1b/2a Treatment Study, our enthusiasm continues to be strong for the potential to develop BB-301 as a safe and efficacious therapy for the improvement of swallowing in patients diagnosed with OPMD with dysphagia,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We are also grateful to have received a favorable recommendation to continue enrollment into Cohort 2 from our Independent Data Safety Monitoring Board following the safe treatment of the sixth and final Subject of Cohort 1. We look forward to beginning enrollment of Subjects into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study, as well as providing additional interim clinical study results for Cohort 1 Subjects, in the fourth calendar quarter of this year.”

Corporate Highlights

In accordance with the protocol for the BB-301 Phase 1b/2a Treatment Study, a meeting of the Independent Data Safety Monitoring Board (DSMB) was convened following the completion of the 28-day post BB-301 dosing visit for the sixth Subject enrolled into Cohort 1. At the conclusion of the meeting, the DSMB formally recommended the continuation of subject enrollment into the Phase 1b/2a Treatment Study, which is expected to begin in calendar Q4 2025.

We look forward to providing additional interim clinical study results for Cohort 1 Subjects in the fourth calendar quarter of this year.

Financial Highlights

Full Year 2025 Financial Results

For the year ended June 30, 2025, the Company reported total expenses of $41.8 million compared to $22.5 million for the year ended June 30, 2024. Research and development expenses were $18.3 million in 2025, up from $15.6 million in 2024, and were primarily related to the ongoing clinical development of BB-301 for the treatment of OPMD. The increase in research and development expenses reflected the timing of contract manufacturing activities and payments associated with the OPMD Natural History and Dosing study.

General and administrative expenses totaled $23.4 million in 2025 compared to $7.0 million in 2024. The increase was primarily driven by higher share-based compensation of $14.5 million, as well as increases in legal fees of $492,000, consulting fees of $605,000, travel expenses of $219,000, and salaries and wages of $685,000.

The loss from operations for the year ended June 30, 2025, was $37.9 million compared to $21.8 million for the prior year. Net loss attributable to shareholders was $37.9 million, or $1.05 per basic and diluted share, compared to a net loss of $22.4 million, or $1.22 per basic and diluted share, for the year ended June 30, 2024. As of June 30, 2025, the Company had $97.7 million in cash and cash equivalents.

BENITEC BIOPHARMA INC.
Consolidated Balance Sheets
(in thousands, except par value and share amounts)
		June 30,					June 30,
		2025					2024
Assets
Current assets:
Cash and cash equivalents		$	97,744				$	50,866
Restricted Cash			113					63
Trade and other receivables			33					229
Prepaid and other assets			628					516
Total current assets			98,518					51,674
Property and equipment, net			131					179
Deposits			55					25
Other assets			28					62
Right-of-use assets			860					270
Total assets		$	99,592				$	52,210
Liabilities and Stockholders’ Equity
Current liabilities:
Trade and other payables		$	1,022				$	4,165
Accrued employee benefits			426					475
Lease liabilities, current portion			354					284
Total current liabilities			1,802					4,924
Non-current accrued employee benefits			-					38
Lease liabilities, less current portion			495					-
Total liabilities			2,297					4,962
Commitments and contingencies (Note 11)
Stockholders’ equity:
Preferred stock, $0.0001 par value - 5,000,000 shares authorized; no shares issued and outstanding at June 30, 2025 and 2024, respectively			-					-
Common stock, $0.0001 par value - 160,000,000 shares authorized; 26,250,469 and 10,086,119 shares issued and outstanding at June 30, 2025 and 2024, respectively			2					1
Additional paid-in capital			326,308					238,398
Accumulated deficit			(228,176	)				(190,259	)
Accumulated other comprehensive loss			(839	)				(892	)
Total stockholders’ equity			97,295					47,248
Total liabilities and stockholders’ equity		$	99,592				$	52,210

BENITEC BIOPHARMA INC.
Consolidated Statements of Operations and Comprehensive Loss
(in thousands, except share and per share amounts)
	Year Ended June 30,
	2025					2024
Revenues	$	-				$	-
Operating expenses
Royalties and license fees		-					(108	)
Research and development		18,332					15,609
General and administrative		23,433					6,989
Total operating expenses		41,765					22,490
Loss from operations		(41,765	)				(22,490	)
Other income (loss):
Foreign currency transaction gain (loss)		(71	)				40
Interest income (expense), net		3,286					904
Other income (expense), net		(131	)				(204	)
Gain on extinguishment of liabilities		764					-
Unrealized gain (loss) on investment		-					(1	)
Total other income (loss), net		3,848					739
Net loss	$	(37,917	)			$	(21,751	)
Other comprehensive income:
Unrealized foreign currency translation gain (loss)		53					(62	)
Total other comprehensive income		53					(62	)
Total comprehensive loss	$	(37,864	)			$	(21,813	)
Net loss	$	(37,917	)			$	(21,751	)
Deemed dividends		-					(619	)
Net loss attributable to common shareholders	$	(37,917	)			$	(22,370	)
Net loss per share:
Basic and diluted	$	(1.05	)			$	(1.22	)
Weighted average number of shares outstanding: basic and diluted		36,209,271					18,364,386

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 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; 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:

Irina Koffler
LifeSci Advisors, LLC
(917) 734-7387
ikoffler@lifesciadvisors.com

FAQ

What were Benitec Biopharma's (BNTC) key financial results for full year 2025?

Benitec reported a net loss of $37.9 million ($1.05 per share), total expenses of $41.8 million, and held $97.7 million in cash as of June 30, 2025.

What is the current status of Benitec's BB-301 clinical trial?

The company completed enrollment of Cohort 1 with six subjects and received DSMB approval to proceed with Cohort 2 enrollment, which is expected to begin in Q4 2025.

How much did Benitec's (BNTC) research and development expenses increase in 2025?

R&D expenses increased to $18.3 million in 2025 from $15.6 million in 2024, primarily related to BB-301 clinical development and OPMD studies.

What is BB-301 and what is it designed to treat?

BB-301 is a modified AAV9 capsid therapy designed to treat OPMD using a 'silence and replace' mechanism, combining siRNAs to silence mutant PABPN1 while expressing a functional version of the protein.

When will Benitec release interim clinical results for BB-301 Cohort 1?

Benitec expects to release interim clinical results for Cohort 1 in Q4 2025.