Skip to main content

Monopar Therapeutics

For the period ended Mar 31, 2026. Filed May 14, 2026.

View on SEC.gov

About this filing

A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Mar 31, 2026 · Filed May 14, 2026

MNPR$71.42+6.2%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2025-09-30; filed 2025-11-13.

0001437749-25-034617

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Current report: 10-Q

Period ended 2026-03-31; filed 2026-05-14.

0001437749-26-016982

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.

Compared 93 previous paragraphs and 94 current paragraphs.

54 unchanged; 31 reworded; 9 added; 6 removed; 2 previous and 0 current paragraphs rearranged.

Tables are not compared: 10 previous and 13 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.

A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.

Added paragraphs (9)

Current report

On April 19, 2026, at the American Academy of Neurology Annual Meeting (AAN), new analyses were presented from the Phase 3 FoCus trial of ALXN1840 showing greater neurologic benefit versus standard of care in Wilson disease patients with neurologic symptoms at baseline, with durable neurologic benefit observed over multiple years of treatment. The data presented highlight the following:

Current report

Based on recent regulatory interactions with the FDA regarding ALXN1840, during which the agency has encouraged continued dialogue with us on certain questions before we submit our NDA, we currently expect to submit our NDA in mid-2026. This anticipated timing reflects our efforts to incorporate additional information and further refine the submission package. However, there can be no assurance that the FDA will accept the NDA for filing or that the application will ultimately be approved.

Current report

We account for warrants as either equity-classified or liability-classified instruments based on an assessment of the warrants’ specific terms and applicable authoritative guidance set forth in ASC 480,Distinguishing Liabilities from Equity (“ASC 480”) and ASC 815,Derivatives and Hedging (“ASC 815”). The assessment considers whether the warrants are freestanding financial instruments pursuant to ASC 480, whether the warrants meet the definition of a liability pursuant to ASC 480, or whether the warrants meet all of the requirements for equity classification under ASC 815.

Current report

Warrants that meet all of the criteria for equity classification are required to be recorded as a component of additional paid-in capital at the time of issuance, or when the conditions for equity classification are met, and are not remeasured. We will assess whether the warrants are indexed to the Company’s own common shares and whether the warrant holders could potentially require “net cash settlement” in a circumstance outside of the Company’s control, among other conditions for equity classification. Liability classified warrants are required to be accounted for at fair value both on the date of issuance and on subsequent accounting period ending dates, with all changes in fair value after the issuance date recorded in the condensed consolidated statements of operations and comprehensive income (loss). In accordance with GAAP, and through the application of professional judgment, we conclude on the appropriate classification of warrants as either liability or equity. The pre-funded warrants issued in 2024 and in 2025 met the equity classification criteria and are recorded in additional-paid-in-capital as permanent equity.

Current report

The increase of $84,521 in cash flow used in financing activities during thethree months ended March 31, 2026, compared to thethree months ended March 31, 2025, was primarily a result of higher net share settlement taxes on vested RSUs in 2026.
Removed paragraphs (6)

Previous report

We are preparing to submit an NDA to the FDA in early 2026.

Previous report

R&D expenses for thenine months ended September 30, 2025, were $5,963,124, compared to $3,081,366 for thenine months ended September 30, 2024. This represents an increase of$2,881,758 attributed to (1) a $1,199,119 increase in manufacturing activities related to ALXN1840 and (2) a $1,864,491 increase in R&D personnel expenses including stock-based compensation partially offset by (3) a net decrease of $181,852 in other R&D expenses.

Previous report

G&A expenses for thenine months ended September 30, 2025, were $4,586,063, compared to $2,005,711 for thenine months ended September 30, 2024. This represents an increase of $2,580,352 primarily attributed to (1) a $1,156,506 increase in Board compensation resulting from the grant of stock options in March 2025 (no stock options were granted to the Board in 2024), (2) a $834,495 increase in G&A personnel expenses including stock-based compensation, (3) a $229,817 increase in legal fees, (4) a $124,248 increase in insurance expenses, and (5) a net increase of $235,286 in other G&A expenses.

Previous report

Interest income for thenine months ended September 30, 2025, increased by $1,778,103 compared to the same period in 2024. The increase is attributed to interest earned on U.S. Treasury securities and higher bank balances in 2025, resulting from net proceeds of approximately $91.9 million, after deducting underwriting discounts and commissions but before offering expenses, from the September 2025 capital raise and Share Repurchase.

Previous report

The increase in cash flow provided by financing activities during thenine months ended September 30, 2025, compared to thenine months ended September 30, 2024, of $88,803,654 was primarily due to higher net proceeds of approximately $91.9 million, after deducting underwriting discounts and commissions but before offering expenses, from the September 2025 capital raise and Share Repurchase.
Reworded paragraphs (31)

Previous wording

We are a clinical-stage biopharmaceutical company with late-stage ALXN1840 for Wilson disease, and radiopharmaceutical programs, including Phase 1-stage MNPR-101-Zr for imaging advanced cancers along with Phase 1a-stage MNPR-101-Lu and late preclinical-stage MNPR-101-Ac225 for the treatment of advanced cancers. We leverage our scientific and clinical experience to help reduce the risk and accelerate the clinical development of our drug product candidates.

Current wording

We are a clinical-stage biopharmaceutical company with late-stage ALXN1840 for Wilson disease, and radiopharmaceutical programs, including MNPR-101-Zr (Phase 1) for imaging advanced cancers along with MNPR-101-Lu (Phase 1a) and MNPR-101-Ac225 (late preclinical) for the treatment of advanced cancers. We leverage our scientific and clinical experience to help reduce the risk and accelerate the clinical development of our drug product candidates.

Previous wording

Our cash, cash equivalents and investments as of September 30, 2025, were $143.7 million. As discussed further below and elsewhere in this Quarterly Report, we expect that our current funds will be sufficient at least through December 31, 2027, in order for us to: (1) assemble a regulatory package and file an NDA for the in-licensed ALXN1840 investigational drug candidate for Wilson disease; (2) continue to conduct and conclude our first-in-human imaging and dosimetry clinical trial with MNPR-101-Zr, continue to conduct our first-in-human therapeutic clinical trial of MNPR-101-Lu, and advance our preclinical MNPR-101-Ac program into the clinic; and (3) invest in internal R&D projects to expand our radiopharmaceutical pipeline.

Current wording

Our cash, cash equivalents and investments as of March 31, 2026, were $137.5 million. As discussed further below and elsewhere in this Quarterly Report, we expect that our current funds will be sufficient at least through December 31, 2027, in order for us to: (1) assemble a regulatory package and file an NDA for the in-licensed ALXN1840 investigational drug candidate for Wilson disease; (2) continue to conduct and conclude our first-in-human imaging and dosimetry clinical trial with MNPR-101-Zr, continue to conduct our first-in-human therapeutic clinical trial of MNPR-101-Lu, and advance our preclinical MNPR-101-Ac program into the clinic; and (3) invest in internal R&D projects to expand our radiopharmaceutical pipeline.

Previous wording

Since our initial public offering in December 2019 and prior to the fourth quarter of 2024, our primary funding source was sales of shares of our common stock under at-the-market sales programs. At-the-market sales were through the Capital on Demand™ Sales Agreements with Jones Trading.

Current wording

Prior to the fourth quarter of 2024, our primary funding source since our initial public offering was sales of shares of our common stock under at-the-market sales programs.

Previous wording

On October 30, 2024, pursuant to a placement agent agreement with Rodman & Renshaw LLC, we sold 1,181,540 shares of our common stock at $16.25 per share in a public offering, yielding net proceeds of approximately $17.8 million, after deducting placement agent fees and other offering costs.

Current wording

On October 30, 2024, we sold 1,181,540 shares of our common stock at $16.25 per share in a public offering, yielding net proceeds of approximately $17.8 million, after deducting placement agent fees and other offering costs.

Previous wording

On December 23, 2024, pursuant to an underwriting agreement with Piper Sandler & Co., we sold 798,655 shares of our common stock at $23.79 per share in a public offering. Concurrent with that offering, we completed a private placement of pre-funded warrants to purchase 882,761 shares of common stock at a purchase price of $23.789 per pre-funded warrant to an institutional investor. The net proceeds of the shares and the pre-funded warrants sold were approximately $37.4 million after fees, commissions and other offering costs.

Current wording

On December 23, 2024, we sold 798,655 shares of our common stock at $23.79 per share in a public offering. Concurrent with that offering, we completed a private placement of pre-funded warrants to purchase 882,761 shares of common stock at a purchase price of $23.789 per pre-funded warrant to an institutional investor. The net proceeds of the shares and the pre-funded warrants sold were approximately $37.4 million after fees, commissions and other offering costs.

Previous wording

On September 23, 2025, pursuant to an underwriting agreement with Morgan Stanley & Co. LLC, Leerink Partners LLC, and Barclays Capital Inc., we sold 1,034,433 shares of our common stock at $67.67 per share in a public offering and pre-funded warrants to purchase 960,542 shares of our common stock at $67.669 per pre-funded warrant, reflecting the $0.001 exercise price. The net proceeds of the shares and pre-funded warrants sold were approximately $126.9 million after underwriting discounts and commissions but before offering expenses. On September 24, 2025, pursuant to a share purchase agreement with Tactic Pharma, an existing significant stockholder that held approximately 13.4% of our common stock prior to this offering, we used $35 million of the Offering proceeds to repurchase 500,229 shares of our common stock from Tactic Pharma at $63.6098 per share, which equals the public offering price per share less underwriting discounts and commissions. After giving effect to the repurchase, our net proceeds from the offering were approximately $91.9 million before estimated offering expenses.

Current wording

On September 23, 2025, we sold 1,034,433 shares of our common stock at $67.67 per share and pre-funded warrants to purchase 960,542 shares of our common stock at $67.669 per pre-funded warrant, reflecting the $0.001 exercise price, in a public offering. The net proceeds of the shares and pre-funded warrants sold were approximately $126.9 million after underwriting discounts and commissions but before offering expenses. On September 24, 2025, pursuant to a share purchase agreement with Tactic Pharma, an existing significant stockholder that held approximately 13.4% of our common stock prior to this purchase, we used $35 million of the offering proceeds to repurchase 550,229 shares of our common stock from Tactic Pharma at $63.6098 per share, which equals the public offering price per share less underwriting discounts and commissions. After giving effect to the repurchase, our net proceeds from the offering were approximately $91.9 million before estimated offering costs.

Previous wording

ALXN1840 (tiomolybdate choline) is an investigational once-daily, orally-administered drug candidate in development for the treatment of Wilson disease, a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised. Over time this excess copper results in the build-up of toxic copper levels in the liver and brain, leading to damage that greatly impacts a patient’s life. Patients can develop a wide range of symptoms, including liver disease and psychiatric or neurological manifestations, such as personality changes, tremors and difficulty walking, swallowing or talking. In some cases, the damage and loss of function may be irreversible. ALXN1840 is a novel small molecule designed to selectively and tightly bind and remove copper from the body’s tissues and blood. ALXN1840 for the treatment of Wilson disease has been granted Orphan Drug Designation and Fast Track designation in the U.S. and orphan designation in the EU.

Current wording

ALXN1840 (tiomolybdate choline) is an investigational once-daily, orally-administered drug candidate in development for the treatment of Wilson disease, a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised. Over time this excess copper results in the build-up of toxic copper levels in the liver and brain, leading to damage that greatly impacts a patient’s life. Patients can develop a wide range of symptoms, including liver disease and psychiatric or neurological manifestations, such as personality changes, tremors and difficulty walking, swallowing or talking. In some cases, the damage and loss of function may be irreversible. ALXN1840 is a novel small molecule designed to selectively and tightly bind and remove copper from the body’s tissues and blood. ALXN1840 has been granted Orphan Drug Designation for the treatment of Wilson disease in the U.S. and the EU, as well as Fast Track designation from the FDA.

Previous wording

Alexion terminated the ALXN1840 program in Wilson disease based on its review of results from Phase 2 mechanistic trials and discussions with regulatory authorities. Their analysis of the Phase 2 mechanistic trials was that they failed to demonstrate a net-negative copper balance in Wilson disease patients during short-term treatment with ALXN1840 and to reduce hepatic copper concentration after treatment with ALXN1840. The decision not to progress the ALXN1840 program in Wilson disease was not related to any safety signals.

Current wording

Alexion terminated the ALXN1840 program in Wilson disease based on its review of results from the Phase 2 mechanistic trials and discussions with the regulatory authorities. Their analysis of the Phase 2 mechanistic trials was that they failed to demonstrate a net-negative copper balance in Wilson disease patients during short-term treatment with ALXN1840 and to reduce hepatic copper concentration after treatment with ALXN1840. The decision not to progress the ALXN1840 program in Wilson disease was not related to any safety signals.

Previous wording

Following Alexion’s decision, in October 2024, we entered into an exclusive worldwide license for the program and assumed responsibility for all future global development and commercialization activities. On May 7, 2025, the Company presented data on the long-term efficacy and safety of ALXN1840 at the European Association for the Study of the Liver (EASL) International Liver Congress 2025, one of the most prominent global conferences in liver disease. Monopar’s late-breaker poster presentation supports the potential use of ALXN1840 as a therapeutic option for Wilson disease. Efficacy data were pooled and analyzed from three clinical trials: Phase 2 WTX101-201, Phase 2 ALXN1840-WD-205, and Phase 3 WTX101-301 (n=255). For safety analysis, data from the Phase 2 ALXN1840-WD-204 trial were also included (n=266). The median treatment duration with ALXN1840 treatment was 961 days (2.63 years) and 943.5 days (2.58 years) for the efficacy and safety datasets, respectively. The data presented highlight the following:

Current wording

Following Alexion’s decision, in October 2024, we entered into an exclusive worldwide license for the program and assumed responsibility for all future global development and commercialization activities. On May 7, 2025, we presented data on the long-term efficacy and safety of ALXN1840 at the European Association for the Study of the Liver (EASL) International Liver Congress 2025, a prominent global conference in liver disease. Efficacy data were pooled and analyzed from three clinical trials: Phase 2 WTX101-201, Phase 2 ALXN1840-WD-205, and Phase 3 WTX101-301 (n=255). For safety analysis, data from the Phase 2 ALXN1840-WD-204 trial were also included (n=266). The median treatment duration with ALXN1840 treatment was 961 days (2.63 years) and 943.5 days (2.58 years) for the efficacy and safety datasets, respectively. The data presented highlight the following:

Previous wording

On June 6, 2025, Alexion officially transferred sponsorship of the investigational new drug (“IND”) application for ALXN1840 to Monopar. The FDA acknowledged this change on July 29, 2025, confirming that the transfer was effective as of June 6, 2025. We are now fully responsible for the program, including its commercial advancement and compliance with all applicable federal regulations.

Current wording

On June 6, 2025, Alexion officially transferred sponsorship of the investigational new drug (“IND”) application for ALXN1840 to us. The FDA acknowledged this change on July 29, 2025, confirming that the transfer was effective as of June 6, 2025. We are now fully responsible for the program, including its commercial advancement and compliance with all applicable federal regulations.

Previous wording

On September 14-15, 2025, we presented new data on the long-term neurological efficacy and safety of ALXN1840 at the 150th American Neurological Association (ANA) Annual Meeting. Matthew Lorincz, M.D., Ph.D., Professor of Neurology and Co-Director of the Wilson Disease Center of Excellence at the University of Michigan delivered the poster and oral presentations. The analysis pooled efficacy and safety data from the same clinical trials pooled and analyzed for the EASL presentation above. The new findings presented at ANA highlight the long-term neurological benefit of ALXN1840, and follow the EASL presentation of long-term hepatic and systemic safety and efficacy data. Together, these findings underscore the potential of ALXN1840 to favorably impact both neurological and hepatic manifestations of Wilson disease. The ANA data presented highlight the following:

Current wording

On September 14-15, 2025, we presented new data on the long-term neurological efficacy and safety of ALXN1840 at the 150th American Neurological Association (ANA) Annual Meeting. The analysis pooled efficacy and safety data from the same clinical trials pooled and analyzed for the EASL presentation above. The new findings presented at ANA highlight the long-term neurological benefit of ALXN1840, and follow the EASL presentation of long-term hepatic and systemic safety and efficacy data. Together, these findings underscore the potential of ALXN1840 to favorably impact both neurological and hepatic manifestations of Wilson disease. The ANA data presented highlight the following:

Previous wording

On November 9, 2025, we presented new data and analyses from the Phase 2 ALXN1840-WD-204 copper balance study at the American Association for the Study of Liver Diseases (AASLD) - The Liver Meeting® 2025. In an oral presentation titled*“Rapidly Improved Cu Balance in Wilson Disease Patients on Tiomolybdate Choline,”* Professor Aftab Ala, MBBS, M.D., FRCP, Ph.D., Professor of Hepatology and Consultant Hepatologist at the Institute of Liver Studies at King’s College Hospital in London, shared results showing that treatment with ALXN1840 (tiomolybdate choline) led to a rapid and sustained improvement in daily copper balance in patients with Wilson disease, primarily through increased fecal copper excretion.

Current wording

On November 9, 2025, we presented new data and analyses from the Phase 2 ALXN1840-WD-204 copper balance study at the American Association for the Study of Liver Diseases (AASLD)– The Liver Meeting® 2025. In an oral presentation titled*“Rapidly Improved Cu Balance in Wilson Disease Patients on Tiomolybdate Choline,”* we shared results showing that treatment with ALXN1840 (tiomolybdate choline) led to a rapid and sustained improvement in daily copper balance in patients with Wilson disease, primarily through increased fecal copper excretion.

Previous wording

We are also actively exploring opportunities to expand our radiopharmaceutical pipeline primarily through internal development efforts. In October 2024, we announced the filing of a provisional patent application for new radiopharmaceutical compounds and a family of linkers used to connect radioisotopes with targeting agents, including our uPAR-targeting antibody MNPR-101.

Current wording

We are also actively exploring opportunities to expand our radiopharmaceutical pipeline primarily through internal development efforts. In October 2024, we announced the filing of a provisional patent application for new radiopharmaceutical compounds and a family of linkers used to connect radioisotopes with targeting agents, including our uPAR-targeting antibody MNPR-101. In March 2025, we filed a corresponding international patent application claiming priority to the October 2024 provisional filing (International Patent Application No. PCT/US2025/021595).

Previous wording

While our significant accounting policies are described in Note 2 of our condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q, we believe the following accounting policies to be critical to the judgments and estimates used in the preparation of our condensed consolidated financial statements.

Current wording

While our significant accounting policies are described in more detail in Note 2 of our condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q, we believe the following accounting policies to be critical to the judgments and estimates used in the preparation of our condensed consolidated financial statements.

Previous wording

Stock-based compensation costs for stock awards granted to our employees, non-employee directors and consultants are based on the fair value of the underlying instruments calculated using the Black-Scholes option-pricing model on the date of grant for stock options and using the closing stock price on the date of grant for RSUs and recognized as an expense on a straight-line basis. Determining the appropriate fair value model and related assumptions requires judgment, including selecting methods for estimating our future stock price volatility and expected holding term. During the nine months ended September 30, 2025, we granted 2,000 options to purchase shares of our common stock to a consultant, 52,766 options to purchase shares of our common stock to non-employee directors, 154,204 options to purchase shares of our common stock to officers, and 39,411 options to purchase shares of our common stock to non-officer employees. The expected stock price volatility is based on an analysis of the Company’s stock price history over a period commensurate with the expected term of the options, trading volume of the Company’s stock, look-back volatilities and Company specific events that affected volatility in a prior period. Forfeitures only include actual forfeitures to-date as the Company accounts for forfeitures as they occur due to a limited history of forfeitures. The expected term for options granted to date is estimated using the simplified method. We have not paid dividends and do not anticipate paying a cash dividend in future vesting periods and, accordingly, use an expected dividend yield of zero. The risk-free interest rate is based on the rate of U.S. Treasury securities with maturities consistent with the estimated expected term of the awards.

Current wording

Stock-based compensation costs for stock awards granted to our employees, non-employee directors and consultants are based on the fair value of the underlying instruments calculated using the Black-Scholes option-pricing model on the date of grant for stock options and using the closing stock price on the date of grant for RSUs and recognized as an expense on a straight-line basis. Determining the appropriate fair value model and related assumptions requires judgment, including selecting methods for estimating our future stock price volatility and expected holding term. During the three months ended March 31, 2026, we granted 2,000 options to purchase shares of our common stock to a consultant, 48,728 options to purchase shares of our common stock to officers, and 11,500 options to purchase shares of our common stock to non-officer employees. The expected stock price volatility is based on an analysis of the Company’s stock price history over a period commensurate with the expected term of the options, trading volume of the Company’s stock, look-back volatilities and Company specific events that affected volatility in a prior period. Forfeitures only include actual forfeitures to date as the Company accounts for forfeitures as they occur due to a limited history of forfeitures. The expected term for options granted to date is estimated using the simplified method. We have not paid dividends and do not anticipate paying a cash dividend in future vesting periods and, accordingly, use an expected dividend yield of zero. The risk-free interest rate is based on the rate of U.S. Treasury securities with maturities consistent with the estimated expected term of the awards.

Previous wording

Comparison of the Three and Nine Months Ended September 30, 2025 and 2024

Current wording

Comparison of the Three Months Ended March 31, 2026 and 2025

Previous wording

The following table summarizes the results of our operations for the three and nine months ended September 30, 2025 and 2024:

Current wording

The following table summarizes the results of our operations for the three months ended March 31, 2026 and 2025:

Previous wording

R&D expenses for the three months ended September 30, 2025, were $2,589,749, compared to $984,278 for the three months ended September 30, 2024. This represents an increase of$1,605,471 attributed to (1) a $937,582 increase in manufacturing activities related to ALXN1840, (2) a $617,667 increase in R&D personnel expenses including stock-based compensation and (3) a net increase of $50,223 in other R&D expenses.

Current wording

R&D expenses for the three months ended March 31, 2026, were $3,487,247 compared to $1,643,375 for the three months ended March 31, 2025. This represents an increase of $1,843,872 primarily attributed to (1) an $825,972 increase in R&D contractor and consulting expenses, (2) a $799,593 increase in R&D personnel expenses including stock-based compensation and (3) a net increase of $218,307 in other R&D expenses.

Previous wording

G&A expenses for the three months ended September 30, 2025, were $1,503,326, compared to $590,624 for the three months ended September 30, 2024. This represents an increase of $912,702 primarily attributed to (1) a $369,959 increase in Board compensation resulting from the grant of stock options in March 2025 (no stock options were granted to the Board in 2024), (2) a $287,749 increase in G&A personnel expenses including stock-based compensation, and (3) a net increase of $254,993 in other G&A expenses.

Current wording

G&A expenses for the three months ended March 31, 2026, were $1,738,006, compared to $1,578,442 for the three months ended March 31, 2025. This represents an increase of $159,564 primarily attributed to (1) a $134,599 increase in G&A personnel expenses including stock-based compensation, and (2) a net increase of $24,965 in other G&A expenses.

Previous wording

Interest income for the three months ended September 30, 2025, increased by $556,129 compared to the same period in 2024. The increase is attributed to interest earned on U.S. Treasury securities and higher bank balances in 2025, resulting from net proceeds of approximately $91.9 million, after deducting underwriting discounts and commissions but before offering expenses, from the September 2025 capital raise and Share Repurchase.

Current wording

Interest income for the three months ended March 31, 2026, was $1,332,203 compared to $596,845 forthe three months ended March 31, 2025. The increase is attributed to interest earned on U.S. Treasury securities and commercial paper, and higher bank balances in 2026, due to the net proceeds of approximately $91.9 million from the September 2025 capital raise.

Previous wording

We have incurred losses and cumulative negative cash flows from operations since we commenced operations resulting in an accumulated deficit of approx imately $84.3 millionas of September 30, 2025. We anticipate that we will continue to incur losses for the foreseeable future. We expect that our R&D and G&A expenses will increase to enable the execution of our strategic plan. We anticipate that the currently available funds as of October 31, 2025, will fund our planned operations at least through December 31, 2027. We will seek to obtain needed capital through a variety of methods, including but not limited to the sale of our common stock, debt financing, strategic partnerships or other sources of capital at our disposal.

Current wording

We have incurred losses and cumulative negative cash flows from operations since we commenced operations, resulting in an accumulated deficit of approx imately $93.4 millionas of March 31, 2026. We anticipate that we will continue to incur losses for the foreseeable future. We expect that our R&D and Selling, General and Administrative (“SG&A”) expenses will increase to enable the execution of our strategic plan, including our ALXN1840 pre-commercial activities and, if approved, commercialization activities. We anticipate that the currently available funds as of March 31, 2026, will fund our planned operations at least through December 31, 2027. We will seek to obtain needed capital through a variety of methods, including but not limited to the sale of our common stock, debt financing, strategic partnerships or other sources of capital at our disposal.

Previous wording

We invest our cash equivalents in money market accounts and U.S. Treasury securities.

Current wording

We invest our cash equivalents in money market accounts, U.S. Treasury securities and commercial paper.

Previous wording

The following table provides information regarding our cash flows for the nine months ended September 30, 2025 and 2024.

Current wording

The following table provides information regarding our cash flows for the three months ended March 31, 2026 and 2025.

Previous wording

During thenine months ended September 30, 2025 and 2024, we had a net cash inflow of $81,861,071 and a net cash outflow of $1,245,996, respectively. During thenine months ended September 30, 2025, versus thenine months ended September 30, 2024, the increase in net cash inflow of $83,107,067 primarily consisted of (1) an increase in cash flow provided by financing activities of $88,803,654 due to the net proceeds of approximately $91.9 million, after deducting underwriting discounts and commissions but before offering expenses, from the September 2025 capital raise and Share Repurchase, partially offset by (2) an increase in net cash used in investing activities due to purchases of investments of $1,264,561 and (3) an increase in net cash used in operating activities of $4,425,498.

Current wording

During thethree months ended March 31, 2026 and 2025, we had a net cash outflow of $9,289,289 and a net cash outflow of $6,104,880, respectively. The net change in cash and cash equivalents as of thethree months ended March 31, 2026, compared to thethree months ended March 31, 2025, primarily consisted of (1) an increase in cash outflow used in investing activities of $5,257,724, which is primarily due to increased investment activity following the $91.9 million in net proceeds from the September 2025 capital raise, partially offset by (2) a decrease in net cash used in operating activities of $2,189,410 due to the payment of $3 million related to ALXN1840 in 2025 (no similar payment was made in 2026).

Previous wording

The increase of $4,425,498 in cash flow used in operating activities during thenine months ended September 30, 2025, compared to thenine months ended September 30, 2024, was primarily a result of a higher net loss due to an increase in research and development expenses and a payment of$3 million related to ALXN1840 in 2025.

Current wording

The decrease of $2,189,410 in cash flow used in operating activities during thethree months ended March 31, 2026, compared to thethree months ended March 31, 2025, was primarily a result of a payment of $3 million related to ALXN1840 in 2025 (no similar payment was made in 2026).

Previous wording

The increase of $1,264,561 in cash used in investing activities during thenine months ended September 30, 2025, compared to thenine months ended September 30, 2024, was primarily a result of net investment in U.S. Treasury securities maturing and/or invested in during the periods reported.

Current wording

The increase of $5,257,724 in cash flow used in investing activities during thethree months ended March 31, 2026, compared to thethree months ended March 31, 2025, was primarily a result of an increase in net investment in U.S. Treasury securities and commercial paper maturing and/or invested in during the periods reported.

Previous wording

To date, we have not generated any revenue from product sales. We do not know when, or if, we will generate any revenue from product sales. We do not expect to generate any revenue from product sales or royalties unless and until we obtain regulatory approval of and commercialize any of our current or future drug product candidates or we out-license or sell a drug product candidate to another party. At the same time, we expect our expenses to increase in connection with our ongoing development activities, particularly as we continue the research, development, future preclinical studies and clinical trials of, and seek regulatory approval for, our current and future drug product candidates. If we obtain regulatory approval of any of our current or future drug product candidates, we will need substantial additional funding for precommercial and commercialization requirements and our continuing drug product development operations.

Current wording

To date, we have not generated any revenue from product sales. We do not know when, or if, we will generate any revenue from product sales. We do not expect to generate any revenue from product sales or royalties unless and until we obtain regulatory approval of and commercialize any of our current or future drug product candidates or we out-license or sell a drug product candidate to another party. At the same time, we expect our expenses to increase in connection with our ALXN1840 pre-commercialization activities, and if approved, commercialization activities, as well as ongoing development activities, particularly as we continue the research, development, future preclinical studies and clinical trials of, and seek regulatory approval for, our current and future drug product candidates. If we obtain regulatory approval of any of our current or future drug product candidates, we will need substantial additional funding for pre-commercial and commercialization requirements and our continuing drug product development operations.

Previous wording

On October 23, 2024, we executed a License Agreement with Alexion, pursuant to which Alexion granted us an exclusive worldwide license for the development and commercialization of ALXN1840, a drug candidate for Wilson disease. As initial upfront consideration for the License Agreement, we issued Alexion 387,329 shares (representing 9.9% of our outstanding shares at the time) of our common stock and agreed to make an upfront cash payment of $4.0 million. The foregoing cash payment consisted of $1.0 million paid at the time of signing and the remaining $3.0 million paid in January 2025, pursuant to the terms of the agreement. As of September 30, 2025, we have paid an aggregate of $4.0 million in cash under the License Agreement. We agreed to an anti-dilution provision that entitled Alexion to receive additional shares at no cost to maintain their 9.9% ownership until we raised the next $25.0 million of our common stock, subject to a maximum of 705,015 shares unless we obtained stockholder approval. Pursuant to the anti-dilution right, we issued an additional 157,188 shares of common stock to Alexion. No further obligations exist pursuant to the anti-dilution right.

Current wording

On October 23, 2024, we executed a License Agreement with Alexion, pursuant to which Alexion granted us an exclusive worldwide license for the development and commercialization of ALXN1840, a drug candidate for Wilson disease. As initial upfront consideration for the License Agreement, we issued Alexion 387,329 shares (representing 9.9% of our outstanding shares at the time) of our common stock and agreed to make an upfront cash payment of $4.0 million. The foregoing cash payment consisted of $1.0 million paid at the time of signing and the remaining $3.0 million paid in January 2025, pursuant to the terms of the agreement. We agreed to an anti-dilution provision that entitled Alexion to receive additional shares at no cost to maintain their 9.9% ownership until we raised the next $25.0 million of our common stock, subject to a maximum of 705,015 shares unless we obtained stockholder approval. Pursuant to the anti-dilution right, we issued an additional 157,188 shares of common stock to Alexion. No further obligations exist pursuant to the anti-dilution right.

Previous wording

Additionally, we are obligated to pay Alexion milestone payments of up to $94.0 million for the achievement of regulatory approval and sales-related milestones. In addition, the Company is obligated to pay tiered royalties based on net salesat rates falling within a range of 10% to 20%. As of September 30, 2025, no milestone or royalty payments have been made under the License Agreement. We have also given Alexion the right of first negotiation regarding any rights should we intend to sublicense ALXN1840. Furthermore, we will have to pay Alexion a percentage in the mid-double digits of any sublicensing income received by us. As part of this License Agreement, we have assumed an agreement from Alexion, under which we will also owe a third-party single digit millions in cash milestone payment upon regulatory approval in Europe and a single digit percentage royalty on net sales in Europe.

Current wording

Additionally, we are obligated to pay Alexion milestone payments of up to $94.0 million for the achievement of regulatory approval and sales-related milestones. In addition, we are obligated to pay tiered royalties based on net salesat rates falling within a range of 10% to 20%. As of March 31, 2026, no milestone or royalty payments have been made under the License Agreement. We have also given Alexion the right of first negotiation regarding any rights should we intend to sublicense ALXN1840. Furthermore, we will have to pay Alexion a percentage in the range of 35% to 45% of any sublicensing income received by us. As part of this License Agreement, we have assumed an agreement from Alexion, under which we will also owe a third-party single digit millions in cash milestone payment upon regulatory approval in Europe and a single digit percentage royalty on net sales in Europe.

Previous wording

Either party may terminate the agreement in the event of an uncured material breach of the agreement following written notice, and the Company may terminate the agreement for convenience upon 90 days prior written notice to Alexion.

Current wording

Either party may terminate the agreement in the event of an uncured material breach of the agreement following written notice, and we may terminate the agreement for convenience upon 90 days prior written notice to Alexion.

Previous wording

We entered into a 36-month lease that commenced on April 1, 2025, for our executive headquarters at 1000 Skokie Blvd in the Village of Wilmette, Illinois, at a monthly rate of $3,580 per month; we also have a month-to-month lease for additional space at the same location for $2,379 per month. We also entered into a lease that commenced on January 16, 2025, for a small wet laboratory space and certain equipment at the Helix 51 Bioscience Incubator at The Rosalind Franklin University of Medicine and Science in North Chicago, Illinois, at a rate of $1,000 per month, which is cancellable after 6 months with 30 days advance written notice.

Current wording

We entered into a 36-month lease that commenced on April 1, 2025, for our executive headquarters at 1000 Skokie Blvd in the Village of Wilmette, Illinois, at a monthly rate of $3,580 per month. On November 1, 2025, we entered into an additional 36-month lease at the same location at a monthly rate of $5,002, which replaced a previous month-to-month arrangement for that space. Additionally, on March 22, 2026, we entered into a one-year lease commencing January 16, 2026, for a small wet laboratory space and certain equipment at the Helix 51 Bioscience Incubator at The Rosalind Franklin University of Medicine and Science in North Chicago, Illinois, at a rate of $875 per month.
Rearranged previous paragraphs (2)

Previous report — wording carried elsewhere

Cash Flow Used in Operating Activities

Previous report — wording carried elsewhere

Cash Flow Used in Investing Activities