11/06/2025 | Press release | Distributed by Public on 11/06/2025 06:46
Management's Discussion and Analysis of Financial Condition and Results of Operations
The following discussion and analysis of our financial conditions and results of operations should be read together with our consolidated financial statements and related notes appearing elsewhere in this Quarterly Report and our audited financial statements and notes thereto as of and for the years ended December 31, 2024 and 2023 and the related Management's Discussion and Analysis of Financial Condition and Results of Operations, included in our Annual Report filed with the Securities and Exchange Commission ("SEC"), on March 20, 2025. In addition to historical information, this discussion and analysis contains forward-looking statements that involve risks, uncertainties and assumptions and other factors that could cause actual results to differ materially from those made, projected or implied in the forward-looking statements. Our actual results may differ materially from those discussed below. Please see "Special Note Regarding Forward-Looking Statements" and "Risk Factors" included in Part I, Item 1A of our Annual Report for factors that could cause or contribute to such differences.
Overview
We are a clinical-stage biopharmaceutical company engaged in the discovery and development of innovative, small molecule therapeutics targeting age-related degenerative diseases and disorders of the central nervous system ("CNS") and retina. Currently available therapies for these diseases are limited, with few Alzheimer's disease treatments, two approved treatments for geographic atrophy ("GA") secondary to dry age-related macular degeneration ("dAMD") and no approved treatments for dementia with Lewy bodies ("DLB"). Our goal is to develop disease-modifying treatments for people with these degenerative disorders by initially leveraging our expertise in the σ-2 (sigma-2) receptor ("S2R"), which is expressed by multiple cell types, including at neuronal synapses, and acts as a key regulator of cellular damage commonly associated with certain age-related degenerative diseases of the CNS and retina.
Our lead candidate for the treatment of age-related degenerative diseases is zervimesine (CT1812). Data indicate that zervimesine antagonizes the binding and toxicity of amyloid beta oligomers via targeting the S2R complex and represents a mechanism that is functionally distinct from other current approaches in clinical development for the treatment of degenerative diseases. Clinical results support this hypothesis, and our clinical trial findings provide evidence that the displacement of oligomers from synapses via zervimesine engagement with the S2R results in improved synapse function.
Top-line results from the Phase 2 COG0201 SHINE study were reported in July 2024 with additional data reported in October 2024. An end-of-Phase 2 meeting was conducted on July 9, 2025 to review the results of the SHINE study and Cognition's proposed plan for a Phase 3 clinical program with the U.S. Food and Drug Administration ("FDA"). Formal minutes were provided by the FDA in August 2025 confirming our Phase 3 clinical program plan.
SHINE was a randomized, double-blind, placebo-controlled trial that enrolled 153 adults with mild-to-moderate Alzheimer's disease. Participants were evenly randomized into two zervimesine dose groups (100 mg or 300 mg) and one placebo group, who were dosed daily for six months. Endpoints included safety and biomarker evidence of disease modification as well as cognitive function, as measured by ADAS-Cog 11. SHINE met its primary endpoints of safety and tolerability.
A prespecified analysis conducted on SHINE results identified plasma p-tau217 as a biomarker that may predict participants with mild-to-moderate Alzheimer's disease likely to respond to zervimesine therapy. Participants treated with zervimesine (pooled 100 mg and 300 mg) who had baseline levels of plasma p-tau217 below the median of 1.0 pg/mL experienced a 95% reduction of cognitive decline at week 26 as measured by ADAS-Cog 11 relative to placebo-treated participants. P-tau217 is an important biomarker that reflects total brain amyloid and tau pathology.
In the overall modified intent-to-treat population in SHINE, participants treated with once-daily oral zervimesine experienced less cognitive decline than those treated with placebo. As measured with ADAS-Cog 11, zervimesine-treated participants (pooled 100 mg and 300 mg) experienced a mean 38% slowing of decline at six months from baseline compared to placebo-treated participants. This difference did not achieve statistical significance. These results are comparable in magnitude to what was achieved with currently approved monoclonal antibody treatments, with the convenience of once-daily oral administration. There were consistent trends favoring zervimesine in other cognitive
measures: ADAS-Cog 13, cognitive composite, mini-mental state exam, or MMSE; as well as in functional measures of activities of daily living (ADCS-ADL) and of clinical global impression of change (ADCS-CGIC).
In the overall study population, participants treated with zervimesine for six months had reductions in plasma biomarkers associated with Alzheimer's disease processes compared to placebo participants. Further analysis showed that the individuals with below-median plasma p-Tau217 experienced a pronounced reduction in these key plasma biomarkers compared to placebo. Significant reductions were observed in the level of glial fibrillary acidic protein (GFAP), a protein associated with neuroinflammation. Neurofilament light, a protein associated with neurodegeneration was also reduced in participants treated with zervimesine compared to placebo. Similarly, amyloid beta monomers (Aβ) and p-Tau217, which are proteins that build up in patients with Alzheimer's disease, were lower in participants treated with zervimesine for six months compared to placebo-treated individuals. These findings were presented at the AD/PD™ 2025 Alzheimer's & Parkinson's Diseases Conference in April 2025.
Top-line results from the Phase 2 COG1201 SHIMMER study were presented at the International Lewy Body Dementia Conference (ILBDC) in January 2025 and at the Alzheimer's Association International Conference (AAIC) in July 2025. The study enrolled 130 participants with mild-to-moderate DLB who were randomized evenly to one of three dose groups: two treated with once-daily oral zervimesine (100 mg or 300 mg) and one treated with placebo. To be eligible, participants were between 50 and 80 years of age, received a diagnosis of probable DLB, and had a MMSE score of between 18 and 27. The study met its primary endpoints of safety and tolerability.
Zervimesine-treated DLB patients scored an average of 86% better than placebo-treated patients on the neuropsychiatric inventory (NPIA-L or NPI-12) at the end of the study. This tool describes the frequency and severity of 12 behavioral symptoms including hallucinations, delusions and anxiety. Compared to placebo-treated participants, those treated with zervimesine performed an average of 52% better on the ADCS-ADL scale, a measure of activities of daily living; an average of 91% better on the CAF, a measure of cognitive fluctuations; an average of 62% better on the Unified Parkinson's Disease Rating Scale (UPDRS) Part III, a measure of motor function such as gait, balance, and tremor.
In June 2025, Cognition received an anonymous philanthropic donation to substantially fund an expanded access program ("EAP") for people with DLB. The EAP will be open to eligible SHIMMER participants who completed the Phase 2 study as well as additional patients with a diagnosis of mild-to-moderate DLB who meet the criteria for this program. Participants will be provided with 100 mg of oral zervimesine to take daily for approximately one year. Eight U.S. sites, all of which were active in the SHIMMER study, were selected to participate in the EAP.
Top-line results from the Phase 2 COG2201 MAGNIFY study of zervimesine in adults with GA secondary to dry AMD were reported in May 2025. The MAGNIFY study was voluntarily concluded in January 2025 after approximately 100 of the planned 246 participants were enrolled to allow the Company to focus its resources on clinical programs in Alzheimer's disease and DLB. Top-line results show zervimesine-treated participants had 29% slower GA lesion growth (by slope analysis) on average and at 18 months their lesions were 28% smaller compared to placebo. The GA lesion reduction is comparable to what was reported with currently approved complement inhibitors. Notable differences between the intravitreal complement inhibitors and zervimesine treatment included the convenience of once-daily oral administration and no conversion from dry AMD to choroidal neovascularization (or "CNV"), which is a risk factor of treatment with intravitreal complement inhibitors.
A "segment" analysis was also conducted to determine the change in GA lesion growth between six-month treatment periods. This analysis shows that the reduction in GA lesion growth increased after 12 months of treatment. The change in lesion growth between 12 and 18 months was 52.7% slower in zervimesine-treated versus placebo-treated participants. The change in ellipsoid zone area, a biomarker of GA progression, also trended in favor of zervimesine treatment compared to placebo. The thickness and integrity of the ellipsoid zone has been correlated with photoreceptor health and visual acuity. Importantly, this effect was also observed to widen over time.
In the above three Phase 2 studies, zervimesine was observed to be generally well tolerated. The average age of participants was approximately 75 years. Among the 238 participants treated with zervimesine, there were 23 incidents of transient treatment-emergent elevations in the liver function test ("LFT") that were greater than 3xULN (9.6%) during the treatment period. Of these incidents, ten occurred in the SHINE study, nine in the SHIMMER study and four in the
MAGNIFY study. In these participants, the elevated liver enzymes subsided after cessation of drug with no evidence of permanent liver injury. Overall, adverse events (AEs) were well balanced between treatment and placebo arms; serious AEs occurred at a comparable or higher rate in placebo-treated participants than in those treated with zervimesine in the above studies. Twenty-eight zervimesine-treated (11.8%) and 11 placebo-treated (4.6%) participants discontinued from these studies due to treatment-emergent adverse events.
The above Overview covers only the most recently concluded studies in each indication. The following table highlights findings from these and subsequent clinical programs:
|
Indication |
Study Identifier |
NCT Number |
Clinical Phase |
Status |
Key Findings |
||||||
|
Alzheimer's Disease (AD) |
|
||||||||||
|
MCI-early |
|
COG0203 (START) |
|
NCT05531656 |
|
Phase 2 |
|
ongoing |
|
The study is enrolling up to 540 participants with MCI or early AD |
|
|
mild-moderate |
|
COG0201 (SHINE) |
|
NCT03507790 |
|
Phase 2 (n=153) |
|
complete |
|
Participants treated with zervimesine experienced a cognitive benefit compared to placebo |
|
|
mild-moderate |
|
COG0202 (SEQUEL) |
|
NCT04735536 |
|
Phase 2 (n=16) |
|
complete |
|
Participants treated with zervimesine exhibited improvement across prespecified EEG parameters |
|
|
mild-moderate |
|
COG0105 (SPARC) |
|
NCT03493282 |
|
Phase 1 (n=23) |
|
complete |
|
Treatment with zervimesine was assessed using various imaging modalities, including PET imaging and volumetric MRI (vMRI) |
|
|
mild-moderate |
|
COG0104 (SNAP) |
|
NCT03522129 |
|
Phase 1 (n=3) |
|
complete |
|
Confirmed preclinical findings showing an increase in Aβ oligomers in CSF, suggesting increased off-rate from receptors |
|
|
Dementia with Lewy Bodies (DLB) |
|
||||||||||
|
mild-moderate |
|
COG1201 (SHIMMER) |
|
NCT05225415 |
|
Phase 2 (n=130) |
|
complete |
|
Participants treated with zervimesine experienced benefits across behavioral, functional, cognitive and motor scales |
|
|
Mild-moderate |
|
COG1202 (EAP) |
|
NCT06961760 |
|
n/a |
|
ongoing |
|
Initially, the EAP will accommodate approximately 30 indviduals with DLB |
|
|
Geographic Atrophy Secondary to Dry AMD |
|
||||||||||
|
GA |
|
COG2201 (MAGNIFY) |
|
NCT05893537 |
|
Phase 2 (n=100) |
|
concluded |
|
Participants treated with zervimesine experienced slower growth of their GA lesions over the course of the study |
|
To date, we have funded our operations primarily with proceeds from grants awarded by the National Institute of Aging (the "NIA"), a division of the National Institutes of Health (the "NIH"), and proceeds from our initial public offering (the "IPO"), completed in October 2021, proceeds from our follow-on public offerings, sales of our common stock through our ATM (as defined below), sales of our convertible promissory notes, convertible preferred stock, simple agreements for future equity ("SAFE") and stock option exercises. Since our inception, we have received approximately $176.7 million in net proceeds from sales of our equity securities, convertible notes, SAFE, stock option exercises, IPO follow-on public offerings, registered direct offering in August 2025, at-the-market offerings, and equity line financing with Lincoln Park. As of September 30, 2025, we had cash, cash equivalents, and restricted cash equivalents of $39.8 million. As of September 30, 2025, we had approximately $36.3 million available from obligated NIA funds for applicable expenses to be incurred in the future.
On December 23, 2022, we entered into a sales agreement with Cantor Fitzgerald & Co. and B. Riley Securities, Inc. (the "Sales Agents"), providing for the offering, issuance and sale by us of up to $40.0 million of our common stock from time to time in "at-the-market" offerings (the "ATM"). For the nine months ended September 30, 2025, we sold 13,159,619 shares of common stock pursuant to the ATM for gross proceeds of approximately $8.6 million. As of September 30, 2025, we have approximately $13.3 million remaining in gross proceeds available for future issuances of common stock under the ATM.
On March 10, 2023, we entered into a purchase agreement with Lincoln Park Capital Fund, LLC ("Lincoln Park") for an equity line financing (the "Lincoln Park Purchase Agreement"). The Lincoln Park Purchase Agreement provides that, subject to the terms and conditions set forth therein, we have the right, but not the obligation, to direct Lincoln Park to purchase up to $35.0 million of shares of common stock at our sole discretion, over a 36-month period commencing on March 10, 2023. We filed a prospectus supplement to our registration statement on Form S-3 (File No. 333-268992)
covering the resale of shares of common stock that are issued under the Lincoln Park Purchase Agreement. During the nine months ended September 30, 2025, we did not sell any shares of common stock to Lincoln Park. As of September 30, 2025, $34.8 million was available to draw pursuant to the Lincoln Park Purchase Agreement.
On March 14, 2024, we completed our follow-on public offering, pursuant to which we issued and sold 6,571,428 shares of our common stock at a public offering price of $1.75 per share. On March 28, 2024, the underwriters exercised their option to purchase 985,714 shares of our common stock at a public offering price of $1.75 per share. In connection with the follow-on public offering, we received net proceeds of approximately $11.9 million, after deducting underwriting discounts and commissions and other offering related expenses.
On August 29, 2025, we completed our registered direct offering, pursuant to which we issued and sold 14,700,000 shares of our common stock at an offering price of $2.05 per share. We received net proceeds of approximately $27.9 million, after deducting underwriting discounts, commissions, placement agent fees, and other offering related expenses payable by us. In connection with the registered direct offering, we agreed to pay the placement agent an aggregate cash fee of 7.0% of the gross proceeds raised from the sale and issuance of the shares of common stock minus certain expenses. We agreed to issue warrants to the placement agent to purchase up to 514,500 shares of common stock which have an exercisable price equal to $2.78 and will be exercisable commencing six months from the close of the registered direct offering with a term of five (5) years from the date of the Placement Agency Agreement.
We expect to continue to incur significant and increasing expenses and net losses for the foreseeable future, as we advance our current and future product candidates through preclinical and clinical development, manufacture drug product and drug supply, seek regulatory approval for our current and future product candidates, maintain and expand our intellectual property portfolio, hire additional research and development and business personnel and operate as a public company. We will not generate revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for our product candidates. In addition, if we obtain regulatory approval for our product candidates and do not enter into a third-party commercialization partnership, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing, manufacturing and distribution activities.
As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until we can generate significant revenue from product sales, if ever, we expect to finance our operations through a combination of public or private equity offerings, debt financings or other sources, such as potential collaboration agreements and strategic alliances, licensing or similar arrangements with third parties. To the extent available, we expect to continue our pursuit of non-dilutive research contributions, or grants, including additional NIA grant funding. However, we may fail to receive additional NIA grants, or we may be unable to raise additional funds or enter into such other agreements or arrangements when needed on acceptable terms, or at all. Our failure to obtain additional NIA grants or raise capital or enter into such agreements as and when needed could have a material adverse effect on our business, results of operations and financial condition.
Because of the numerous risks and uncertainties associated with product development, we are unable to accurately predict the timing or amount of increased expenses or when, or if, we will be able to achieve profitability. Even if we do achieve profitability, we may not be able to sustain or increase profitability on a quarterly or annual basis. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to raise capital, maintain our research and development efforts, expand our business or continue our operations at planned levels, and as a result we may be forced to substantially reduce or terminate our operations.
We do not own or operate manufacturing facilities. We rely, and expect to continue to rely, on third parties for the manufacture of zervimesine for preclinical studies and clinical trials, as well as for commercial manufacture if zervimesine obtains marketing approval. We also rely, and expect to continue to rely, on third parties to manufacture, package, label, store, and distribute zervimesine, if marketing approval is obtained. We believe that this strategy allows us to maintain a more efficient infrastructure by eliminating the need for us to invest in our own manufacturing facilities, equipment, and personnel while also enabling us to focus our expertise and resources on the development of zervimesine.
Components of Our Results of Operations
Operating Expenses
Research and Development Expenses
Research and development expenses consist primarily of direct and indirect costs incurred for our research activities, including development of our drug discovery efforts and the development of our product candidates. Direct costs include laboratory materials and supplies, contracted research and manufacturing, clinical trial costs, consulting fees, and other expenses incurred to sustain our research and development program. Indirect costs include personnel-related expenses, consisting of employee salaries, related benefits, and stock-based compensation expense for employees engaged in research and development activities, facilities, and other expenses consisting of direct and allocated expenses for rent and depreciation, and lab consumables.
We expense research and development costs as incurred. Non-refundable advance payments for goods and services that will be used over time for research and development are capitalized and recognized as goods are delivered or as the related services are performed. In-licensing fees and other costs to acquire technologies used in research and development that have not yet received regulatory approval and that are not expected to have an alternative future use are expensed when incurred. We track direct costs by stage of program, clinical or preclinical. However, we do not track indirect costs on a program specific basis because these costs are deployed across multiple programs and, as such, are not separately classified.
We cannot reasonably determine the nature, timing, and estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, any of our product candidates. Product candidates in later stages of development generally have higher development costs than those in earlier stages. We expect that our research and development expenses will increase substantially for the foreseeable future as we continue to invest in research and development activities related to developing our product candidates, as our product candidates advance into later stages of development, as we begin to conduct larger clinical trials, as we seek regulatory approvals for any product candidates that successfully complete clinical trials, as we expand our product pipeline, as we maintain, expand, protect and enforce our intellectual property portfolio, and as we incur expenses associated with hiring additional personnel to support our research and development efforts.
General and Administrative Expenses
General and administrative expenses consist primarily of personnel-related costs, including employee salaries, related benefits, and stock-based compensation expense for our employees in the executive, finance and accounting, and other administrative functions. General and administrative expenses also include third-party costs such as legal costs, insurance costs, accounting, auditing and tax related fees, consulting fees and facilities and other expenses not otherwise included as research and development expenses. We expense general and administrative costs as incurred.
Other Income (Expense)
Grant Income
Grant income relates to the grants and donations received from government and other (non-government) parties. Grants awarded are conditional cost reimbursement grants and are recognized as grant income as allowable costs are incurred and the right to payment is realized. The grants awarded relate to agreed upon direct and indirect costs for specific studies or clinical trials, which may include personnel and consulting costs, costs paid to CROs, research institutions and /or consortiums involved in the grant, as well as facilities and administrative costs. These grants are cost plus fixed fee arrangements in which we are reimbursed for eligible direct and indirect costs over time, up to the maximum amount of each specific grant award. Only costs that are allowable under the grant award, certain government regulations and the NIH's supplemental policy and procedure manual may be claimed for reimbursement, and the reimbursements are subject to routine audits from governmental agencies from time to time. As of September 30, 2025, the Company has been awarded grants with project periods that extend through May 31, 2027, subject to extension. Our clinical trials have been
funded by approximately $171.0 million in cumulative grants awarded primarily by the NIA, which includes an approximately $81.0 million grant from the NIA to fund our Phase 2 (COG0203-START) study of zervimesine in patients with early-stage Alzheimer's disease, an approximately $30.5 million grant from the NIA to fund our Phase 2 (COG0201-SHINE) study of zervimesine in patients with mild-to-moderate Alzheimer's disease, and an approximately $29.5 million grant from the NIA to fund our Phase 2 (COG1201-SHIMMER) study of zervimesine in patients with DLB.
Other Income, Net
Other income, net consists primarily of interest income from money market funds, offset partially by other fees such as costs incurred to establish financing opportunities.
Interest Expense
Interest expense primarily consists of interest expense related to the insurance premium financing arrangement with a lender.
Results of Operations
Comparison of the Three Months Ended September 30, 2025 and 2024
The following table summarizes our results of operations (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
|
|
||||
|
|
2025 |
2024 |
Change |
||||||
|
Operating Expenses: |
|
|
|
||||||
|
Research and development |
|
$ |
3,773 |
|
$ |
11,392 |
|
$ |
(7,619) |
|
General and administrative |
|
2,602 |
|
3,071 |
|
(469) |
|||
|
Total operating expenses |
|
6,375 |
|
14,463 |
|
(8,088) |
|||
|
Loss from operations |
|
(6,375) |
|
(14,463) |
|
8,088 |
|||
|
Other income (expense): |
|
|
|
|
|||||
|
Grant income |
|
1,215 |
|
4,293 |
|
(3,078) |
|||
|
Other income, net |
|
230 |
|
236 |
|
(6) |
|||
|
Interest expense |
|
|
- |
|
|
(3) |
|
|
3 |
|
Total other income, net |
|
1,445 |
|
4,526 |
|
(3,081) |
|||
|
Net loss |
|
$ |
(4,930) |
|
$ |
(9,937) |
|
$ |
5,007 |
Research and Development Expenses
The following table summarizes our research and development expenses (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
|
|
||||
|
|
2025 |
2024 |
Change |
||||||
|
Clinical programs |
|
$ |
1,435 |
|
$ |
7,977 |
|
$ |
(6,542) |
|
Personnel |
|
1,555 |
|
2,591 |
|
(1,036) |
|||
|
Manufacturing |
|
751 |
|
515 |
|
236 |
|||
|
Preclinical programs |
|
37 |
|
259 |
|
(222) |
|||
|
Other expense |
|
(5) |
|
50 |
|
(55) |
|||
|
|
|
$ |
3,773 |
|
$ |
11,392 |
|
$ |
(7,619) |
Research and development expenses were $3.8 million for the three months ended September 30, 2025, compared to $11.4 million for the three months ended September 30, 2024. The decrease of approximately $7.6 million was primarily due to the following:
| ● | a decrease of $6.5 million in clinical programs primarily related to decreased Phase 2 trial activities with contract research organizations; |
| ● | a decrease of $1.0 million in personnel costs related to reduced professional fees and headcount; |
| ● | a decrease of $0.3 million in preclinical programs and other expense, primarily due to decreased research activities; and |
| ● | an increase of $0.2 million in manufacturing related to higher costs with contract manufacturing organizations for the replenishment of clinical trial supply. |
General and Administrative Expenses
General and administrative expenses were $2.6 million for the three months ended September 30, 2025, compared to $3.1 million for the three months ended September 30, 2024. The change in general and administrative expenses was driven primarily by a decrease in equity-based compensation, which was partially offset by an increase in professional fees.
Other Income (Expense)
Grant Income
Grant income was $1.2 million for the three months ended September 30, 2025, compared to $4.3 million for the three months ended September 30, 2024. The change in grant income is correlated with the decrease in eligible reimbursable costs related to clinical trials incurred during 2025 as compared to 2024, offset partially by grant income recognized from the donation received.
Other Income, Net
Other income, net was $0.2 million for the three months ended September 30, 2025, compared to other income, net of $0.2 million for the three months ended September 30, 2024. The change in other income, net was insignificant period over period.
Interest Expense
Interest expense was less than $0.1 million for the three months ended September 30, 2025, compared to interest expense of less than $0.1 million for the three months ended September 30, 2024. Interest expense was not significant in either period.
Comparison of the Nine Months Ended September 30, 2025 and 2024
The following table summarizes our results of operations (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Nine Months Ended September 30, |
|
|
|
||||
|
|
2025 |
2024 |
Change |
||||||
|
Operating Expenses: |
|
|
|
||||||
|
Research and development |
|
$ |
26,040 |
|
$ |
33,522 |
|
$ |
(7,482) |
|
General and administrative |
|
8,088 |
|
9,721 |
|
(1,633) |
|||
|
Total operating expenses |
|
34,128 |
|
43,243 |
|
(9,115) |
|||
|
Loss from operations |
|
(34,128) |
|
(43,243) |
|
9,115 |
|||
|
Other income (expense): |
|
|
|
|
|||||
|
Grant income |
|
13,407 |
|
16,516 |
|
(3,109) |
|||
|
Other income, net |
|
585 |
|
813 |
|
(228) |
|||
|
Interest expense |
|
(8) |
|
(20) |
|
12 |
|||
|
Loss on currency translation from liquidation of subsidiary |
|
|
- |
|
|
(195) |
|
|
195 |
|
Total other income, net |
|
13,984 |
|
17,114 |
|
(3,130) |
|||
|
Net loss |
|
$ |
(20,144) |
|
$ |
(26,129) |
|
$ |
5,985 |
Research and Development Expenses
The following table summarizes our research and development expenses (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Nine Months Ended September 30, |
|
|
|
||||
|
|
2025 |
2024 |
Change |
||||||
|
Clinical programs |
|
$ |
16,486 |
$ |
22,312 |
|
$ |
(5,826) |
|
|
Personnel |
|
7,169 |
|
|
8,324 |
|
(1,155) |
||
|
Manufacturing |
|
1,901 |
|
|
1,926 |
|
(25) |
||
|
Preclinical programs |
|
262 |
|
|
784 |
|
(522) |
||
|
Other expense |
|
222 |
|
|
176 |
|
46 |
||
|
Total research & development expenses |
|
$ |
26,040 |
|
$ |
33,522 |
|
$ |
(7,482) |
Research and development expenses were $26.0 million for the nine months ended September 30, 2025, compared to $33.5 million for the nine months ended September 30, 2024. The decrease of $7.5 million was primarily due to the following:
| ● | a decrease of $5.8 million in clinical programs primarily related to decreased Phase 2 trial activities with contract research organizations; |
| ● | a decrease of $1.2 million in personnel costs related to reduced professional fees and headcount; and |
| ● | a decrease of $0.5 million in manufacturing, preclinical programs and other expense, primarily due to decreased research activities. |
General and Administrative Expenses
General and administrative expenses were $8.1 million for the nine months ended September 30, 2025, compared to $9.7 million for the nine months ended September 30, 2024. The change in general and administrative expenses was driven primarily by a decrease in equity-based compensation, which was partially offset by an increase in professional fees.
Other Income (Expense)
Grant Income
Grant income was $13.4 million for the nine months ended September 30, 2025, compared to $16.5 million for the nine months ended September 30, 2024. The change in grant income is correlated with the decrease in eligible reimbursable costs related to clinical trials incurred during 2025 as compared to 2024, offset partially by grant income recognized from the donation received.
Other Income, Net
Other income, net was $0.6 million for the nine months ended September 30, 2025, compared to other income, net of $0.8 million for the nine months ended September 30, 2024. The change in other income, net was insignificant period over period.
Interest Expense
Interest expense was less than $0.1 million for the nine months ended September 30, 2025, compared to interest expense of less than $0.1 million for the nine months ended September 30, 2024. Interest expense was not significant in either period.
Liquidity and Capital Resources
Sources of Liquidity
To date, we have funded our operations primarily with proceeds from grants awarded by the NIA, and proceeds from the sales of our convertible promissory notes, convertible preferred stock, SAFE, stock option exercises, follow-on equity offerings, sales under our ATM and equity line financing, and our IPO. Since our inception, we have been awarded grant awards primarily from the NIA in the aggregate amount of approximately $171.0 million and have raised approximately $176.7 million in net proceeds from sales of our equity securities, convertible notes and SAFE, stock option exercises, our ATM, our equity line financing with Lincoln Park, our IPO and our follow-on public offering. The net proceeds from our IPO, which closed on October 13, 2021, were approximately $44.2 million, after deducting underwriting discounts and commissions and other offering related expenses payable by us. On November 15, 2022, we closed our follow-on public offering, selling 5,000,000 shares of our common stock at a public offering price of $1.20 per share. The net proceeds were approximately $5.2 million, after deducting underwriting discounts and commissions and other offering related expenses payable by us. On December 23, 2022, we entered into a sales agreement with the Sales Agents, providing for the offering, issuance and sale by us of up to $40.0 million of our common stock from time to time in ATM offerings. As of September 30, 2025, we sold 35,931,882 shares of common stock under the ATM for gross proceeds of approximately $26.7 million. As of September 30, 2025, there was $13.3 million of common stock remaining available for sale under the ATM. In addition, in March 2023, we entered into the Lincoln Park Purchase Agreement with Lincoln Park Capital Fund, LLC ("Lincoln Park"), giving the Company the right, but not the obligation to sell to Lincoln Park up to $35.0 million worth of shares of our common stock. As of September 30, 2025, we have sold 125,000 shares of common stock to Lincoln Park for proceeds of $0.2 million, as part of the equity line financing arrangement. As of September 30, 2025, $34.8 million was available to draw pursuant to the Lincoln Park Purchase Agreement. On March 14, 2024, we closed a follow-on public offering of 6,571,428 shares of our common stock at a public offering price of $1.75 per share. As part of the follow-on offering, the underwriters exercised their option to purchase 985,714 shares of our common stock on March 28, 2024, at a public offering price of $1.75 per share. The net proceeds were approximately $11.9 million, after deducting underwriting discounts and commissions and other offering related expenses payable by us. On August 29, 2025, we completed the registered direct offering of 14,700,000 shares of our common stock at an offering price of $2.05 per share. As part of the registered direct offering, we agreed to issue warrants to the placement agent to purchase up to 514,500 shares of common stock which have an exercise price equal to $2.78. The net proceeds were approximately $27.9 million, after deducting underwriting discounts, commissions, placement agent fees, and other offering related expenses payable by us.
As of September 30, 2025, we had $39.8 million in cash, cash equivalents, and restricted cash equivalents and have not generated positive cash flows from operations. Based on our current business plans, we believe that our existing cash and cash equivalents, income from non-dilutive grants and donations, and net proceeds from our March 2024 follow-on public offering and August 2025 registered direct offering will be sufficient for us to fund our operating expenses and capital expenditures requirements into the second quarter of 2027, which assumes no usage from the remaining ATM nor the Lincoln Park Purchase Agreement. We have based these estimates on assumptions that may prove to be incorrect or require adjustment as a result of business decisions, and we could utilize our available capital resources sooner than we currently expect.
Future Funding Requirements
We expect to continue to incur significant and increasing expenses and net losses for the foreseeable future, as we advance our current and future product candidates through preclinical and clinical development, manufacture drug product and drug supply, seek regulatory approval for our current and future product candidates, maintain and expand our intellectual property portfolio, hire additional research and development and business personnel, and operate as a public company. We anticipate that we will need to raise additional funding in the future to fund our operations, including the commercialization of any approved product candidates. We are subject to the risks typically related to the development of new products, and we may encounter unforeseen expenses, difficulties, complications, delays, and other unknown factors that may adversely affect our business.
Our future funding requirements will depend on many factors, including, but not limited to:
| ● | the scope, progress, costs and results of our ongoing and planned clinical trials of zervimesine, as well as the associated costs, including any unforeseen costs we may incur as a result of preclinical study or clinical trial delays due to a pandemic, such as the COVID-19 pandemic or other diseases, macroeconomic conditions, global or political instability, such as the ongoing global and regional conflicts, inflation, or other delays; |
| ● | the scope, progress, costs and results of preclinical development, laboratory testing and clinical trials for any future product candidates we may decide to pursue; |
| ● | the extent to which we develop, in-license or acquire other product candidates and technologies; |
| ● | the costs and timing of process development and manufacturing scale-up activities associated with our product candidates and other programs as we advance them through preclinical and clinical development; |
| ● | the availability, timing, and receipt of any future NIA grants, or any changes to our grants based on political or regulatory pressures; |
| ● | the number and development requirements of other product candidates that we may pursue; |
| ● | the costs, timing and outcome of regulatory review of our product candidates; |
| ● | the costs and timing of future commercialization activities, including product manufacturing, marketing, sales and distribution, for any of our product candidates for which we receive marketing approval; |
| ● | the revenue, if any, received from commercial sales of our product candidates for which we receive marketing approval; |
| ● | our ability to establish collaborations to commercialize zervimesine or any of our other product candidates outside the United States; |
| ● | the costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending any intellectual property-related claims; and |
| ● | the additional costs we may incur as a result of operating as a public company, including our efforts to enhance operational systems and hire additional personnel, including enhanced internal controls over financial reporting. |
Until such time as we can generate significant revenue from product sales, we expect to finance our operations through a combination of public or private equity offerings, debt financings or other sources, such as potential collaboration agreements and strategic alliances, licensing or similar arrangements with third parties. To the extent available, we expect to continue our pursuit of non-dilutive research contributions, or grants, including additional NIA grant funding. However, we may fail to receive additional NIA grants, or we may be unable to raise additional funds or enter into such other agreements or arrangements when needed on acceptable terms, or at all. Our failure to obtain additional NIA grants or raise capital or enter into such agreements as and when needed could have a material adverse effect on our business, results of operations and financial condition.
To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our stockholders will be or could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise funds through collaborations, licenses and other similar arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us and/or may reduce the value of our common stock. Adequate funding may not be available when needed or on terms acceptable to us, or at all. Our ability to raise additional funds may be adversely impacted by potential worsening global economic conditions and the recent disruptions to, and volatility in, the credit and financial markets in the United States and worldwide resulting from the effects of the COVID-19 pandemic or other diseases, the ongoing global and regional conflicts, inflation, liquidity constraints, failures and instability in U.S. and international financial banking systems, and otherwise. If we fail to obtain necessary capital when needed on acceptable terms, or at all, it could force us to delay, limit, reduce or terminate our product development programs, commercialization efforts or other operations. Insufficient liquidity may also require us to relinquish rights to product candidates at an earlier stage of development or on less favorable terms than we would otherwise choose. We cannot assure you that we will ever be profitable or generate positive cash flows from operating activities.
Cash Flows
The following table summarizes our cash flows for the periods indicated (in thousands):
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|
|
|
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Nine Months Ended September 30, |
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|
2025 |
2024 |
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Cash flows used in operating activities |
|
$ |
(21,151) |
$ |
(20,092) |
|
|
Cash flows used in investing activities |
|
- |
|
(3) |
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|
Cash flows provided by financing activities |
|
35,965 |
|
12,184 |
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|
Net increase (decrease) in cash, cash equivalents, and restricted cash equivalents |
|
$ |
14,814 |
|
$ |
(7,911) |
Cash used in operating activities
Net cash used in operating activities for the nine months ended September 30, 2025 and 2024 was $21.2 million and $20.1 million, respectively. The change in cash used in operating activities of $1.1 million was driven by a decrease in net loss of $6.0 million, combined with a decrease of $5.4 million in operating assets and liabilities primarily related to an increase in grant receivables of $2.9 million, and a decrease in non-cash adjustments of $1.8 million primarily related to a decrease in equity-based compensation of $1.5 million.
Cash used in investing activities
Net cash used in investing activities for the nine months ended September 30, 2025 and 2024 was zero and less than $0.1 million, respectively.
Cash provided by financing activities
Net cash provided by financing activities was $36.0 million and $12.2 million for the nine months ended September 30, 2025 and 2024, respectively. The change in net cash provided by financing activities is primarily related to net proceeds from the issuance of common stock in the registered direct offering in August 2025 of $27.9 million and net proceeds from the issuance of common stock under the ATM program of $8.3 million, as compared to the net proceeds of $11.9 million in our follow-on offering in March 2024 and net proceeds of $0.9 million under the ATM program.
Contractual Obligations
The following table summarizes our contractual obligations as of September 30, 2025 (in thousands):
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Less than |
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1 to 3 |
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3 to 5 |
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More than 5 |
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|
|||||||||||||
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|
1 Year |
Years |
Years |
years |
Total |
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Operating lease obligations |
|
$ |
189 |
|
$ |
174 |
|
$ |
61 |
|
$ |
- |
|
$ |
424 |
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Other obligations |
|
|
- |
|
|
- |
|
|
- |
|
|
- |
|
|
- |
|||||||||
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Total: |
|
$ |
189 |
|
$ |
174 |
|
$ |
61 |
|
$ |
- |
|
$ |
424 |
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In October 2023, we entered into an insurance premium financing arrangement with a lender whereby we financed $0.7 million of certain premiums at a 8.65% annual interest rate. Payments of less than $0.1 million are due monthly from November 2023 through October 2024. As of December 31, 2024, there was no outstanding balance on the loan.
In October 2024, we entered into an insurance premium financing arrangement whereby we financed $0.4 million of certain premiums at a 8.65% annual interest rate. Payments of less than $0.1 million are due monthly from November 2024 through July 2025. As of September 30, 2025, there was no outstanding balance on the loan.
In October 2025, we entered into an insurance premium financing arrangement whereby we financed $0.4 million of certain premiums at a 7.95% annual interest rate. Payments of less than $0.1 million are due monthly from November 2025 through August 2026.
We have entered into an operating leases for office and laboratory facilities under agreements that run through May 31, 2029. The amounts reflected in the table above consist of the future minimum lease payments under the non-cancelable lease arrangements.
On August 31, 2022, we entered into an agreement to lease 2,980 square feet of office space in Pittsburgh, Pennsylvania. The lease has a term of 45 months and commenced on October 1, 2022. The annual base rent under the lease is less than $0.1 million throughout the term of the lease. Total payments due over the term of the lease are $0.2 million. Additionally, on August 31, 2022, we modified one of our existing lease agreements with the landlord for approximately 3,706 square feet of lab space at the same location to extend the lease term termination date from June 30, 2023 until June 30, 2026.
On July 1, 2021, we entered into an agreement to lease 2,864 square feet of office space in Purchase, New York. The lease has a term of 89 months and commenced on December 9, 2021. The annual base rent under the lease is less than $0.1 million for the first lease year and is subject to annual increases of between 1.82% and 2.04%. We provided a security deposit in the form of a Letter of Credit in the amount of less than $0.1 million pursuant to the terms of the lease.
We enter into contracts in the normal course of business with CROs and other vendors to assist in the performance of our research and development and other services and products for operating purposes. These contracts typically do not contain minimum purchase commitments and generally provide for termination on notice, and therefore are cancelable contracts and not included in the table of contractual obligations.
Critical Accounting Policies and Use of Estimates
The Critical Accounting Policies and Significant Judgements and Estimates included in our Annual Report on Form 10-K have not materially changed. See "Critical Accounting Policies and Use of Estimates" included in Part II, Item 7 of our Annual Report on Form 10-K filed with the SEC on March 20, 2025.
Recent Accounting Pronouncements
For a description of recent accounting pronouncements, see Note 2 of the notes to our consolidated financial statements included in this Quarterly Report.
Emerging Growth Company Status
We are an emerging growth company, as defined in the Jumpstart Our Business Startups Act of 2012 (the "JOBS Act"). Under the JOBS Act, emerging growth companies can delay adopting new or revised accounting standards issued subsequent to the enactment of the JOBS Act until such time as those standards apply to private companies. We elected to use this extended transition period for complying with new or revised accounting standards that have different effective dates for public and private companies until the earlier of the date that we (1) are no longer an emerging growth company or (2) affirmatively and irrevocably opt out of the extended transition period provided in the JOBS Act. As a result, our financial statements may not be comparable to companies that comply with the new or revised accounting pronouncements as of public company effective dates.
We will remain an emerging growth company until the earliest to occur of: (1) the last day of the fiscal year in which we have at least $1.235 billion in annual revenue; (2) the last day of the fiscal year in which we are deemed to be a "large accelerated filer," as defined in Rule 12b-2 under the Exchange Act, which would occur if the market value of our common stock held by non-affiliates exceeded $700.0 million as of the last business day of the second fiscal quarter of such year; (3) the date on which we have issued more than $1.0 billion in non-convertible debt securities during the prior three-year period; and (4) the last day of the fiscal year ending after the fifth anniversary of our IPO.