Polaryx Therapeutics, Inc.

Previous wording

We are advancing PLX-200, our most advanced product candidate, through a Phase 2 proof-of-concept basket trial which we refer to as SOTERIA (PLX-200-600). We expect to initiate this trial in the second half of 2026. SOTERIA is an open-label, multi-indication, master study for the treatment of certain LSDs which we believe represent approximately one quarter of our addressable LSD population, including CLN2, CLN3, Krabbe disease, and Sandhoff disease. We held a pre-IND submission meeting in April 2025. We submitted an IND application to the FDA for the SOTERIA trial in August 2025 and received a safe to proceed letter in October 2025. Data readouts from SOTERIA are expected to provide guidance and a clear pathway for each of the four indications towards potentially registrable trials. Further, with the precedent approval of Brineura, a drug approved to treat CLN2 on the basis of a single-arm, natural history comparator, open-label trial, we believe there may be an opportunity in CLN2 and CLN3 for us to seek expedited approval from the FDA for PLX-200 based on precedent approval for a third-party drug with a similar trial design. Should PLX-200 evidence overwhelming efficacy from the CLN2 and CLN3 cohorts in the SOTERIA trial, we believe there may be a case to seek expedited approval. Products studied for their safety and effectiveness in treating serious or life-threatening diseases or conditions may receive expedited approval upon a determination that the product has demonstrated a clinically meaningful treatment effect. The precedent case of cerliponase alfa, a drug approved by FDA in treatment of CLN2, provides a benchmark for expedited approval based on results generated from an open-label, single arm trial comparing to natural history data studying Batten disease. SOTERIA’s current trial design for the CLN2 and CLN3 cohorts share the same open-label, single-arm design using natural history.

Current wording

We are advancing PLX-200, our most advanced product candidate, through a Phase 2 proof-of-concept basket trial which we refer to as SOTERIA (PLX-200-600). We expect to initiate this trial in the second half of 2026. SOTERIA is an open-label, multi-indication, master study for the treatment of certain LSDs which we believe represent approximately one quarter of our addressable LSD population, including CLN2, CLN3, Krabbe disease, and Sandhoff disease. We held a pre-IND submission meeting in April 2025. We submitted an IND application to the FDA for the SOTERIA trial in August 2025 and received a safe to proceed letter in October 2025. In July 2026, we amended SOTERIA’s trial design; among other amendments, we expanded the number of participants in each of the Sandhoff disease and Krabbe disease cohorts from three to six, thereby aligning enrollment targets across all four cohorts and increasing the total number of trial participants from 18 to 24. We believe that the increase in number of trial participants will generate additional valuable data. We have additionally opened recruitment for the six-patient sentinel safety group, from three patients each drawn from the CLN2 and CLN3 cohorts to six patients drawn from any of the four cohorts. Data readouts from SOTERIA are expected to provide guidance and a clear pathway for each of the four indications towards potentially registrable trials. Further, with the precedent approval of Brineura, a drug approved to treat CLN2 on the basis of a single-arm, natural history comparator, open-label trial, we believe there may be an opportunity in CLN2 and CLN3 for us to seek expedited approval from the FDA for PLX-200 based on precedent approval for a third-party drug with a similar trial design. Should PLX-200 evidence overwhelming efficacy from the CLN2 and CLN3 cohorts in the SOTERIA trial, we believe there may be a case to seek expedited approval. Products studied for their safety and effectiveness in treating serious or life-threatening diseases or conditions may receive expedited approval upon a determination that the product has demonstrated a clinically meaningful treatment effect. The precedent case of cerliponase alfa, a drug approved by FDA in treatment of CLN2, provides a benchmark for expedited approval based on results generated from an open-label, single arm trial comparing to natural history data studying Batten disease. SOTERIA’s current trial design for the CLN2 and CLN3 cohorts share the same open-label, single-arm design using natural history.

Previous wording

Since our inception in August 2014, we have devoted substantially all of our resources to raising capital, organizing and staffing our company, business and scientific planning, conducting discovery and research activities, acquiring product programs, establishing and protecting our intellectual property portfolio, developing and progressing our pipeline, establishing arrangements with third parties for the manufacture of our programs and component materials, and providing general and administrative support for these operations. We do not have any product candidates approved for sale and have not generated any revenue from product sales. Since our inception through the filing date of this Quarterly Report, we have funded our operations primarily through the issuance of approximately $21.7 million of common stock and preferred stock.

Current wording

Since our inception in August 2014, we have devoted substantially all of our resources to raising capital, organizing and staffing our company, business and scientific planning, conducting discovery and research activities, acquiring product programs, establishing and protecting our intellectual property portfolio, developing and progressing our pipeline, establishing arrangements with third parties for the manufacture of our programs and component materials, and providing general and administrative support for these operations. We do not have any product candidates approved for sale and have not generated any revenue from product sales. Since our inception through the filing date of this Quarterly Report, we have funded our operations primarily through the issuance of approximately $31.5 million of common stock and preferred stock.

Previous wording

We have incurred significant operating losses since inception and expect to incur losses in the future as we continue our research and development activities. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of any product candidates we may develop. We incurred net losses of approximately $2.5 million and $5.1 million for the three months ended March 31, 2026 and 2025, respectively. As of March 31, 2026, we had an accumulated deficit of approximately $102.2 million.

Current wording

We have incurred significant operating losses since inception and expect to incur losses in the future as we continue our research and development activities. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of any product candidates we may develop. We incurred net losses of approximately $1.4 million and $1.0 million for the three months ended June 30, 2026 and 2025, respectively. We incurred net losses of approximately $3.9 million and $6.1 million for the six months ended June 30, 2026 and 2025, respectively. As of June 30, 2026, we had an accumulated deficit of approximately $103.6 million.

Previous wording

As of March 31, 2026, we had cash and cash equivalents of approximately $3.1 million. Based on our current operating plan, we estimate that our existing cash and cash equivalents as of the filing date of this Quarterly Report, will be sufficient to enable us to fund our operating expenses and capital expenditure requirements through the third quarter of 2026. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

Current wording

As of June 30, 2026, we had cash and cash equivalents of approximately $11.5 million. Based on our current operating plan, we estimate that our existing cash and cash equivalents as of the filing date of this Quarterly Report, will be sufficient to enable us to fund our operating expenses and capital expenditure requirements through the first quarter of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

Previous wording

Other expense — direct listing offering costs was $586 thousand for the three months ended March 31, 2026, as compared to zero for the three months ended March 31, 2025 due to legal, accounting, and advisory expenses related to the preparation of the direct listing offering.

Current wording

Other expense — direct listing offering costs was zero for the three months ended June 30, 2026, as compared to $124 thousand for the three months ended June 30, 2025 due to legal, accounting, and advisory expenses related to the preparation of the direct listing offering in January 2026.

Previous wording

Research and development expenses decreased by $4.0 million to $679 thousand for the three months ended March 31, 2026, as compared to $4.7 million for the three months ended March 31, 2025. The reduction in research and development expenses was primarily due to stock-based compensation of approximately $4.3 million related to the issuance of 3,704,307 shares of common stock in March of 2025 to two existing stockholders in return for an exclusive gene therapy patent license totaling $4.3 million, which was not repeated in 2026. Of the total 3,704,307 shares issued, 277,823 shares were issued to Rush and 3,426,484 shares were issued to Mstone. The decrease in stock-based compensation expense was partially offset by expenses incurred for the three months ended March 31, 2026 under three statements of work pursuant to the Rush MSA that increased by $92 thousand. CRO formulation services increased by $139 thousand. Research and development expenses were substantially related to PLX-200 except for the stock-based compensation expense in 2025.

Current wording

Research and development expenses decreased by $3.8 million to $1.4 million for the six months ended June 30, 2026, as compared to $5.2 million for the six months ended June 30, 2025. The reduction in research and development expenses was primarily due to stock-based compensation of approximately $4.3 million related to the issuance of 3,704,307 shares of common stock in March of 2025 to two existing stockholders in return for an exclusive gene therapy patent license totaling $4.3 million, which was not repeated in 2026. Of the total 3,704,307 shares issued, 277,823 shares were issued to Rush and 3,426,484 shares were issued to Mstone. The decrease in stock-based compensation expense was partially offset by CRO expenses incurred for the six months ended June 30, 2026 related to SOTERIA that increased by $322 thousand, CRO formulation services that increased by $177 thousand and salary and wages that increased by $162 thousand as a result of the timing of new hires. Research and development expenses were substantially related to PLX-200 except for the stock-based compensation expense in 2025.

Previous wording

General and administrative expenses increased by $943 thousand to $1,279 thousand for the three months ended March 31, 2026, as compared to $336 thousand for the three months ended March 31, 2025, primarily due to increased public company related expenses incurred in connection with the direct listing in January 2026 and due to an increase in stock-based compensation. Public company related expenses including legal, audit, investor relations, board compensation, and directors and officers insurance expenses increased by $435 thousand. Stock-based compensation increased by $359 thousand due to shares that were issued to a financial advisor in October of 2024. Of the total shares issued, 50% was fully vested upon issuance as compensation for advisory services and concluded in October of 2025 which decreased stock-based compensation by $108 thousand in the first quarter of 2026. The remaining 50% vested upon a public listing of the Company’s common stock. As of March 31, 2026, the remaining 50% was fully vested as our common stock began trading on Nasdaq on February 2, 2026. As such, the Company recorded $467 thousand to stock-based compensation.

Current wording

General and administrative expenses increased by $1.2 million to $1.9 million for the six months ended June 30, 2026, as compared to $744 thousand for the six months ended June 30, 2025, primarily due to increased public company related expenses incurred in connection with the direct listing in January 2026, due to an increase in stock-based compensation, and due to an increase in salary and wages. Public company related expenses including legal, audit, accounting advisory, investor relations, board compensation, and directors and officers insurance expenses increased by $712 thousand. Stock-based compensation increased by $252 thousand due to shares that were issued to a financial advisor in October of 2024. Of the total shares issued, 50% was fully vested upon issuance as compensation for advisory services and concluded in October of 2025 which decreased stock-based compensation by $216 thousand in the second quarter of 2026. The remaining 50% vested upon a public listing of the Company’s common stock. As of March 31, 2026, the remaining 50% was fully vested as our common stock began trading on Nasdaq on February 2, 2026. As such, the Company recorded $467 thousand to stock-based compensation. Salary and wages increased by $149 thousand as a result of the timing of new hires.

Previous wording

Since our inception, we have incurred significant operating losses. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the clinical development of our programs. From our inception through the filing date of this Quarterly Report, we have funded our operations primarily with proceeds from the sales of our equity securities totaling approximately $21.7 million. As of March 31, 2026, we have no outstanding debt.

Current wording

Since our inception, we have incurred significant operating losses. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the clinical development of our programs. From our inception through the filing date of this Quarterly Report, we have funded our operations primarily with proceeds from the sales of our equity securities totaling approximately $31.5 million. As of June 30, 2026, we have no outstanding debt.

Previous wording

The following table presents cash provided by (used in) operating and financing activities during the three months ended March 31, 2026 and 2025:

Current wording

The following table presents cash provided by (used in) operating and financing activities during the six months ended June 30, 2026 and 2025:

Previous wording

Net cash used in operating activities was $2.1 million for the three months ended March 31, 2026 and was primarily due to a net loss of $2.5 million, offset by an increase in stock-based compensation of $467 thousand and an increase in working capital changes of $14 thousand.

Current wording

Net cash used in operating activities was $3.4 million for the six months ended June 30, 2026 and was primarily due to a net loss of $3.9 million, offset by stock-based compensation of $467 thousand and an increase in working capital changes of $25 thousand.

Previous wording

Net cash used in operating activities was $709 thousand for the three months ended March 31, 2025 and was primarily due to a net loss of $5.1 million and working capital decreases of $108 thousand related to accounts payable and accrued expenses, offset by stock-based compensation of $4.5 million.

Current wording

Net cash used in operating activities was $1.6 million for the six months ended June 30, 2025 and was primarily due to a net loss of $6.1 million and a decrease in working capital changes of $67 thousand, offset by stock-based compensation of $4.6 million.

Previous wording

Net cash provided by financing activities was $250 thousand for the three months ended March 31, 2025 and was due to proceeds from the issuance of common stock. We had no cash provided by or used in financing activities for the three months ended March 31, 2026.

Current wording

Net cash provided by financing activities was $9.8 million and $250 thousand for the six months ended June 30, 2026 and 2025, respectively, and was due to proceeds from the issuance of common stock.

Previous wording

The financial statements have been prepared as though we will continue as a going concern, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business. We have incurred operating losses and negative cash flows from operations since inception. As of March 31, 2026, we had an accumulated deficit of approximately $102.2 million. Management expects to continue to incur operating losses and negative cash flows.

Current wording

The financial statements have been prepared as though we will continue as a going concern, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business. We have incurred operating losses and negative cash flows from operations since inception. As of June 30, 2026, we had an accumulated deficit of approximately $103.6 million. Management expects to continue to incur operating losses and negative cash flows.

Previous wording

We believe that our existing capital will enable us to fund our operations through the third quarter of 2026. We will need to raise additional capital in connection with our cash needs for capital expenditures and working capital beyond the third quarter of 2026. We have based the foregoing estimate on assumptions that may prove to be incorrect, and we could use our capital resources sooner than we expect.

Current wording

We believe that our existing capital will enable us to fund our operations through the first quarter of 2027. We will need to raise additional capital in connection with our cash needs for capital expenditures and working capital beyond the first quarter of 2027. We have based the foregoing estimate on assumptions that may prove to be incorrect, and we could use our capital resources sooner than we expect.