-Positive Interim Clinical Trial Data for the First Oculopharyngeal Muscular Dystrophy (OPMD) Subject Dosed with BB-301 in the Phase 1b/2a Clinical Treatment Study Reported in April-
-The Second OPMD Subject was Safely Dosed with BB-301 in February-
-Additional Interim Clinical Safety Data and Clinical Efficacy Data are Expected for Several Study Subjects in the Second Half of 2024-
-23 OPMD Subjects have Enrolled into the OPMD Natural History Study with Multiple Subjects Eligible for Entry into the BB-301 Phase 1b/2a Clinical Treatment Study-
–Closed an Oversubscribed Private Placement Financing of $40.0 Million on April 22nd, Cash Runway Extended Through 2025–
HAYWARD, Calif., May 13, 2024 (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 third fiscal quarter ended March 31, 2024. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission.
“The interim clinical trial results for the first subject enrolled onto the BB-301 Phase 1b/2a Clinical Treatment Study demonstrated consistent, clinically meaningful improvements in each of the central study endpoints, with significant improvements noted across the radiographic assessments of swallowing and corresponding improvements observed for the key subject-reported outcome measure,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We look forward to presenting the results of longer-term follow-up for the first study subject, along with the accruing data for additional study subjects, later this year”. “Following the recent financing, Benitec is well-positioned to advance the BB-301 clinical development program through the end of 2025.”
Operational Updates
On April 18th the Company announced an oversubscribed $40.0 million private investment in public equity (PIPE) financing from the sale of 5,749,152 shares of its common stock at a price per share of $4.80, and, in lieu of shares of common stock, pre-funded warrants to purchase up to an aggregate of 2,584,239 shares of its common stock at a price per pre-funded warrant of $4.7999, to certain institutional accredited investors.
The financing was led by Suvretta Capital Management, LLC with participation from new and existing investors including Adage Capital Partners L.P., Nantahala Capital, multiple healthcare-focused funds, and a leading mutual fund. Gross proceeds from the PIPE financing totaled approximately $40.0 million, before deducting offering expenses. The closing of the PIPE occurred on April 22, 2024.
In connection with the PIPE financing, the Company has agreed with long-term investor Suvretta Capital Management to consider Kishen Mehta, a portfolio manager at Suvretta Capital Management, for appointment to the Company’s board of directors.
The key milestones related to the development of BB-301 for the treatment of OPMD-related Dysphagia, along with other corporate updates, are outlined below:
Interim BB-301 Phase 1b/2a Clinical Treatment Study Results for Subject 1:
During the OPMD Natural History (NH) Study, which represents the pre-dose observational period for each subject, Subject 1 experienced progressive worsening of dysphagia as demonstrated by the results of the videofluoroscopic swallowing studies (VFSS), the cold water timed drinking test, and the key subject-reported outcome measure (the Sydney Swallow Questionnaire). VFSS represents the gold standard analytical method for the quantitative assessment of dysphagia (swallowing difficulty) in the clinical setting.
At the 90-day post-dose assessment following the administration of BB-301, Subject 1 demonstrated improvements in key videofluoroscopic assessments which correlated with the observation of similar improvement in the key subject-reported outcome measure as compared to the average values for the respective assessments completed during the pre-dose observational period (as summarized in Figure 1). Notably, the results of many assessments completed at the 90-day timepoint demonstrated improvements over the initial measurements assessed at the subject’s first visit for the natural history observational study which occurred more than 12 months prior to the 90-day post-dose assessment.
The most significant VFSS improvements at Day 90 were observed for swallowing tasks centered on the evaluation of pharyngeal constrictor muscle function and swallowing efficiency in the context of the consumption of thin liquids (e.g., water), solid foods, and thick non-solid foods (e.g., yogurt or pudding) (Figure 1). The VFSS improvements correlated with an improvement in the key subject-reported outcome measure (the Sydney Swallow Questionnaire), indicating an improvement in swallowing function as reported by Subject 1 (Figure 1).
Figure 1: Improvement in All Outcomes at 90-Days Post-BB-301 Injection*
*Company data on file
Regarding the BB-301 safety profile observed to date, no Serious Adverse Events (SAEs) have been observed for the two subjects that have received BB-301. Transient Grade 2 Gastroesophageal Reflux Disease or “GERD” (i.e., “acid reflux” or “heartburn”) was observed for the two subjects that received BB-301. For both study subjects, the GERD resolved following the use of common prescription medications approved for the treatment of GERD.
Corporate Updates:
Twenty-three (23) subjects have been enrolled into the OPMD NH Study, and the BB-301 Phase 1b/2a Clinical Treatment Study is designed to treat 21-to-30 subjects. Two (2) subjects have rolled over from the OPMD NH Study into the BB-301 Phase 1b/2a Clinical Treatment Study, and a third OPMD subject is expected to enter the BB-301 Phase 1b/2a Clinical Treatment Study in June 2024.
Additional interim safety data and efficacy data are expected to be reported from the BB-301 Phase 1b/2a Clinical Treatment Study in 2H 2024.
Financial Highlights
Third Fiscal Quarter 2024 Financial Results
Total Revenues for the quarter ended March 31, 2024, were $0 compared to $54,000 in licensing revenues collected for the quarter ended March 31, 2023.
Total Expenses for the quarter ended March 31, 2024, were $4.1 million compared to $4.4 million for the quarter ended March 31, 2023. For the quarter ended March 31, 2024, the Company incurred ($3,000) in royalties and license fees due to the reversal of an accrual compared to $0 for the three months ended March 31, 2023. The Company incurred $2.6 million of research and development expenses compared to $3.2 million for the comparable quarter ended March 31, 2023. 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 $1.2 million for the quarter ended March 31, 2023.
The loss from operations for the quarter ended March 31, 2024, was $4.1 million compared to a loss of $4.3 million for the quarter ended March 31, 2023. Net loss attributable to shareholders for the quarter ended March 31, 2024, was $4.3 million, or $1.64 per basic and diluted share, compared to a net loss of $4.4 million, or $2.67 per basic and diluted share for the quarter ended March 31, 2023. As of March 31, 2024, the Company had $14.1 million in cash and cash equivalents. Following the closing of the recent PIPE transaction on April 22, 2024, the Company received approximately $37.2 million.
BENITEC BIOPHARMA INC.
Consolidated Balance Sheets
(in thousands, except par value and share amounts)
March 31,
June 30,
2024
2023
(Unaudited)
Assets
Current assets:
Cash at bank
$
14,143
$
2,477
Restricted cash
13
13
Trade and other receivables
53
55
Prepaid and other assets
157
1,184
Total current assets
14,366
3,729
Property and equipment, net
204
87
Deposits
25
25
Prepaid and other assets
69
97
Right-of-use assets
335
526
Total assets
$
14,999
$
4,464
Liabilities and stockholders’ equity
Current liabilities:
Trade and other payables
$
2,628
$
3,231
Accrued employee benefits
517
472
Lease liabilities, current portion
292
275
Total current liabilities
3,437
3,978
Lease liabilities, less current portion
62
284
Total liabilities
3,499
4,262
Commitments and contingencies (Note 11)
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