Clene expects to submit a New Drug Application for CNM-Au8® in ALS under the accelerated approval pathway in early fourth quarter of 2026Clene announces survival and functional benefit in CNM-Au8-treated patients whose NfL declined or stabilized
“We plan to submit our new drug application (NDA) for CNM-Au8 under the accelerated approval pathway for patients with ALS and believe the evidence connecting the magnitude of NfL reduction to clinical benefit data we have generated since our last FDA meeting will be critical for a successful review,” said
Second Quarter 2026 and Recent Operating Highlights
CNM-Au8 for the treatment of ALS
New biomarker analyses of the Company’s two completed Phase 2 ALS trials revealed additional evidence that CNM-Au8-treated patients whose NfL declined or stabilized lived significantly longer than concurrently randomized controls and performed significantly better on combined measures of survival and function, including the ALS Functional Rating Scale-Revised (ALSFRS-R) and breathing capacity (Slow Vital Capacity; SVC). These new data intend to show the FDA that the “magnitude of change in NfL is reasonably likely to predict clinical benefits in patients with ALS,” as noted from the FDA minutes.
These findings were derived from multiple lines of analysis: clinical benefit versus concurrently randomized controls; the relationship between the size of the NfL reduction and clinical outcome; a causal analysis that identified likely NfL responders from pre-treatment characteristics alone, preserving the randomized comparison; and replication of the association between NfL change and survival across independent datasets.
The NDA submission is expected to be made under the accelerated approval pathway and will be supported by NfL biomarker and clinical data from the Phase 2 HEALEY ALS Platform Trial and its open-label extension, the Phase 2 RESCUE-ALS Trial, and the
Corporate Update
In May, the Company closed an underwritten registered direct common stock offering to a single investor totaling
Also in May, the Company amended its existing
Second Quarter 2026 Financial Results
Research and development expenses were
General and administrative expenses were
Total other expense, net, was
About
About CNM-Au8®
CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8® is a federally registered trademark of Clene Nanomedicine, Inc.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended, which are intended to be covered by the “safe harbor” provisions created by those laws. Clene’s forward-looking statements include, but are not limited to, statements regarding the timing of the Company’s meeting with the FDA, the timing of the Company’s NDA submission, and that the biomarker findings support an NDA submission. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” “contemplate,” “continue,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “will,” “would,” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements represent our views as of the date of this press release and involve a number of judgments, risks and uncertainties. We anticipate that subsequent events and developments will cause our views to change. We undertake no obligation to update forward-looking statements to reflect events or circumstances after the date they were made, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Accordingly, forward-looking statements should not be relied upon as representing our views as of any subsequent date. As a result of a number of known and unknown risks and uncertainties, our actual results or performance may be materially different from those expressed or implied by these forward-looking statements. Some factors that could cause actual results to differ include general market conditions, whether clinical trials demonstrate the efficacy and safety of our drug candidates to the satisfaction of regulatory authorities, or do not otherwise produce positive results which may cause us to incur additional costs or experience delays in completing, or ultimately be unable to complete the development and commercialization of our drug candidates; the clinical results for our drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; our ability to achieve commercial success for our drug candidates, if approved; our limited operating history and our ability to obtain additional funding for operations and to complete the development and commercialization of our drug candidates; and other risks and uncertainties set forth in “Risk Factors” in our most recent Annual Report on Form 10-K and any subsequent Quarterly Reports on Form 10-Q. In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this press release, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and you are cautioned not to rely unduly upon these statements. All information in this press release is as of the date of this press release. The information contained in any website referenced herein is not, and shall not be deemed to be, part of or incorporated into this press release.
Investor Contact: Kevin Gardner, LifeSci Advisors; kgardner@lifesciadvisors.com; 617-283-2856
Media Contact: Caroline Wagner, FTP; CWagner@ftpadvocacy.com; (267) 294-6563
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands, except share and per share amounts) (Unaudited) | ||||||||||||||||
| Three Months Ended | Six Months Ended | |||||||||||||||
| 2026 | 2025 | 2026 | 2025 | |||||||||||||
| Revenue: | ||||||||||||||||
| Product revenue | $ | 74 | $ | 1 | $ | 75 | $ | 65 | ||||||||
| Royalty revenue | 16 | 26 | 30 | 43 | ||||||||||||
| Total revenue | 90 | 27 | 105 | 108 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Cost of revenue | 36 | — | 36 | 20 | ||||||||||||
| Research and development | 3,471 | 3,514 | 3,800 | 4,995 | ||||||||||||
| General and administrative | 1,930 | 2,377 | 3,677 | 5,033 | ||||||||||||
| Total operating expenses | 5,437 | 5,891 | 7,513 | 10,048 | ||||||||||||
| Loss from operations | (5,347 | ) | (5,864 | ) | (7,408 | ) | (9,940 | ) | ||||||||
| Other income (expense), net: | ||||||||||||||||
| Interest income | 55 | 62 | 102 | 143 | ||||||||||||
| Interest expense | (695 | ) | (679 | ) | (1,486 | ) | (1,287 | ) | ||||||||
| Issuance costs for common stock warrant liabilities | — | — | (393 | ) | — | |||||||||||
| Loss on initial issuance of equity | — | — | (4,582 | ) | — | |||||||||||
| Change in fair value of common stock warrant liabilities | (4,132 | ) | (515 | ) | (5,192 | ) | 1,995 | |||||||||
| Change in fair value of derivative liabilities | (3,333 | ) | (439 | ) | (2,620 | ) | 708 | |||||||||
| Research and development tax credits and unrestricted grants | 28 | 16 | 64 | 211 | ||||||||||||
| Total other income (expense), net | (8,077 | ) | (1,555 | ) | (14,107 | ) | 1,770 | |||||||||
| Net loss before income taxes | (13,424 | ) | (7,419 | ) | (21,515 | ) | (8,170 | ) | ||||||||
| Income tax expense | — | — | — | — | ||||||||||||
| Net loss | $ | (13,424 | ) | $ | (7,419 | ) | $ | (21,515 | ) | $ | (8,170 | ) | ||||
| Other comprehensive income (loss): | ||||||||||||||||
| Foreign currency translation adjustments | $ | (8 | ) | $ | 63 | $ | 36 | $ | 78 | |||||||
| Total other comprehensive income (loss) | (8 | ) | 63 | 36 | 78 | |||||||||||
| Comprehensive loss | $ | (13,432 | ) | $ | (7,356 | ) | $ | (21,479 | ) | $ | (8,092 | ) | ||||
| Net loss per share – basic and diluted | $ | (1.08 | ) | $ | (0.78 | ) | $ | (1.79 | ) | $ | (0.89 | ) | ||||
| Weighted average common shares used to compute basic and diluted net loss per share | 12,382,702 | 9,523,592 | 12,015,498 | 9,176,063 | ||||||||||||
CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands, except share and per share amounts) (Unaudited) | ||||||||
| 2026 | 2025 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 9,671 | $ | 5,189 | ||||
| Accounts receivable | 43 | — | ||||||
| Inventory | 37 | 37 | ||||||
| Prepaid expenses and other current assets | 5,163 | 3,751 | ||||||
| Total current assets | 14,914 | 8,977 | ||||||
| Restricted cash | 58 | 58 | ||||||
| Operating lease right-of-use assets | 2,753 | 3,073 | ||||||
| Property and equipment, net | 5,316 | 6,023 | ||||||
| TOTAL ASSETS | $ | 23,041 | $ | 18,131 | ||||
| LIABILITIES AND STOCKHOLDERS’ DEFICIT | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,142 | $ | 892 | ||||
| Accrued liabilities | 2,482 | 5,002 | ||||||
| Operating lease obligations, current portion | 815 | 808 | ||||||
| Notes payable, current portion | 413 | 1,696 | ||||||
| Convertible notes payable, current portion | — | 2,378 | ||||||
| Total current liabilities | 4,852 | 10,776 | ||||||
| Operating lease obligations, net of current portion | 2,805 | 3,250 | ||||||
| Notes payable, net of current portion | 5,374 | 3,741 | ||||||
| Convertible notes payable, net of current portion | 12,937 | 9,800 | ||||||
| Common stock warrant liabilities | 16,137 | 5,063 | ||||||
| Derivative liabilities | 5,713 | 3,093 | ||||||
| TOTAL LIABILITIES | 47,818 | 35,723 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ deficit: | ||||||||
| Common stock, | 1 | 1 | ||||||
| Additional paid-in capital | 304,825 | 290,531 | ||||||
| Accumulated deficit | (329,811 | ) | (308,296 | ) | ||||
| Accumulated other comprehensive income | 208 | 172 | ||||||
| TOTAL STOCKHOLDERS’ DEFICIT | (24,777 | ) | (17,592 | ) | ||||
| TOTAL LIABILITIES AND STOCKHOLDERS’ DEFICIT | $ | 23,041 | $ | 18,131 | ||||
