Cyclo Therapeutics' Phase 3 trial for Trappsol® Cyclo™ in Niemann-Pick Disease Type C1 progresses, with key leadership changes announced.
Quiver AI Summary
Cyclo Therapeutics is progressing with its Phase 3 clinical trial of TransportNPC™ for treating Niemann-Pick Disease Type C1, as recommended by the Data Monitoring Committee after a 48-week safety and efficacy review. Rafael Holdings, Inc. reported its fourth-quarter financial results for fiscal year 2025, highlighting a net loss of $12.1 million, affected by the integration of Cyclo Therapeutics' expenses after its acquisition. The company bolstered its financial position with a $25 million rights offering, which will help fund the Trappsol® Cyclo™ program. CEO Howard Jonas welcomed Joshua Fine as the new Chief Operating Officer and expressed optimism about the company's future, supported by a total cash balance of $52.8 million as of July 31, 2025, and an overall net loss for the fiscal year of $30.5 million, reduced from the previous year's losses.
Potential Positives
- The ongoing Phase 3 clinical trial of Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 continues to progress positively, as recommended by the Data Monitoring Committee, indicating potential for a new treatment option for patients.
- The company has strengthened its financial position with a $25 million rights offering, providing resources to advance its clinical programs and explore further opportunities.
- Improvements in year-over-year financial performance are highlighted, showing a reduced net loss attributable to shareholders from $34.4 million to $30.5 million, suggesting better overall financial management and operational efficiency.
- Key appointments in the leadership team, including Joshua Fine as Chief Operating Officer, signal focused leadership and strategic direction for the company's future initiatives.
Potential Negatives
- The reported net loss attributable to Rafael Holdings increased to $12.1 million for the three months ended July 31, 2025, compared to $4.5 million in the same period the previous year, indicating a significant deterioration in financial performance.
- Research and development expenses surged to $7.5 million for the latest quarter, up from $1.5 million year-over-year, which raises concerns about the sustainability of the company's financial resources.
- The acquisition of Cyclo Therapeutics resulted in an increase in general and administrative expenses to $5.5 million from $2.3 million in the prior year, suggesting operational challenges and rising costs associated with the integration of new assets.
FAQ
What is the purpose of the TransportNPC™ Phase 3 trial?
The trial evaluates Trappsol® Cyclo™ as a treatment for Niemann-Pick Disease Type C1.
Who is the newly elected Chief Operating Officer?
Joshua Fine was elected as the Chief Operating Officer of Rafael Holdings on August 4, 2025.
What financial position did Rafael Holdings report at the end of fiscal year 2025?
Rafael Holdings reported cash and cash equivalents of $52.8 million as of July 31, 2025.
What was the net loss for Rafael Holdings in the fourth quarter of 2025?
The net loss attributable to Rafael Holdings was $12.1 million for the quarter ending July 31, 2025.
What are the implications of the recent data from the TransportNPC™ trial?
The Data Monitoring Committee recommends continuation, indicating potential efficacy and safety of Trappsol® Cyclo™.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
Cyclo Therapeutics’ TransportNPC™ Phase 3 clinical trial for Trappsol ® Cyclo™ for the treatment of Niemann-Pick Disease Type C1, a rare and fatal genetic disease, is continuing at the recommendation of the Data Monitoring Committee (DMC) following their review of prespecified safety and efficacy date at 48 weeks
On August 4, 2025, Joshua Fine was elected as the Company’s Chief Operating Officer
NEWARK, N.J., Oct. 29, 2025 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL; NYSE American: RFL-W), today reported its financial results for the fourth quarter and full fiscal year 2025 ended July 31, 2025.
“We are pleased with the continued progress of our pivotal Phase 3 TransportNPC ™ study evaluating Trappsol ® Cyclo™ for the treatment of Niemann-Pick Disease Type C1, which we believe could provide an important new treatment for option for patients suffering from this rare and fatal genetic disease,” said Howard Jonas, Chief Executive Officer, Executive Chairman and Chairman of the Board of Rafael Holdings. Mr. Jonas added, “We enhanced our financial position with the closing of a $25 million rights offering in June, which positions our Company well to advance the Trappsol ® Cyclo™ program and invest in additional opportunities we may identify. I would also like to take this opportunity to congratulate Joshua Fine on his appointment as the Company’s Chief Operating Officer, Alan Grayson on his addition to our Board of Directors and Markus Sieger on being named as Chair of our Audit Committee. I look forward to their important contributions towards advancing the Company and driving value for all stakeholders.”
Rafael Holdings, Inc. Fourth Quarter Fiscal Year 2025 Financial Results
As of July 31, 2025, we had cash and cash equivalents of $52.8 million. On June 4, 2025, the Company closed a $25 million rights offering, which included the funding of the backstop commitment in the amount of $21.0 million by the Jonas family.
For the three months ended July 31, 2025, we recorded a net loss attributable to Rafael Holdings of $12.1 million, or $0.28 per share, versus a net loss of $4.5 million, or $0.19 per share in the year ago period. The year over year increase in net loss is attributable to the consolidation of Cyclo Therapeutic’s expenses following the acquisition of Cyclo in March 2025 and the activity of Cornerstone and Day Three which were consolidated with Rafael Holdings during fiscal 2024.
Research and development expenses were $7.5 million for the three months ended July 31, 2025, compared to $1.5 million in the year ago period. The year over year increase relates to the inclusion in the current year period of spending at Cyclo following the March 2025 acquisition and the activity of Cornerstone and Day Three which were consolidated with Rafael Holdings during fiscal 2024.
General and administrative expenses were $5.5 million for the three months ended July 31, 2025, compared to $2.3 million in the year ago period. The year over year increase relates to the inclusion of expenses at Cyclo following closing of the acquisition, and the activity of Cornerstone and Day Three, following their consolidation.
Rafael Holdings, Inc. Full Year Fiscal Year 2025 Financial Results
For the twelve months ended July 31, 2025, we recorded a net loss attributable to Rafael Holdings of $30.5 million, or $1.04 per share, versus a net loss of $34.4 million, or $1.45 per share in the year ago period. The year over year decrease in net loss is attributable to in-process R&D expense of $89.9 million related to the acquisition (when we increased our ownership position) of Cornerstone netted with a $31.3 million recovery of receivables from Cornerstone in the year ago period, $5.9 million in unrealized gains on the Company’s investment in Cyclo equity and the inclusion of Cyclo following closing of the acquisition in March 2025 and the activity of Cornerstone and Day Three, following their consolidation.
Research and development expenses were $12.8 million for the twelve months ended July 31, 2025, compared to $4.2 million in the year ago period. The year over year increase relates to the inclusion of spending at Cyclo following the March 2025 acquisition of with Cyclo, and the activity of Cornerstone and Day Three, which were consolidated with Rafael Holdings during fiscal 2024.
For the twelve months ended July 31, 2025, general and administrative expenses were $13.8 million compared to $8.9 million in the same period in the prior year. The year over year increase relates to spending at Cyclo following the acquisition, and the activity of Cornerstone and Day Three, following their consolidation.
About Rafael Holdings, Inc.
Rafael Holdings, Inc. is a biotechnology company that develops pharmaceuticals and holds interests in clinical and early stage companies that develop pharmaceuticals and medical devices. Our lead candidate is Trappsol® Cyclo™, which is being evaluated in clinical trials for the potential treatment of Niemann-Pick Disease Type C1 (“NPC1”), a rare, fatal and progressive genetic disorder. We also hold interests in other clinical-stage and early-stage pharmaceutical development companies and an orthopedic-focused medical device company developing instruments to advance minimally invasive surgeries. Our lead candidate, Trappsol® Cyclo™, is the subject of an ongoing pivotal Phase 3 clinical trial.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans regarding the further evaluation of clinical data; and the potential of our pipeline, including our internal cancer metabolism research programs. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, those disclosed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended July 31, 2024, and our other filings with the SEC. These factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.
Contact:
Barbara Ryan
[email protected]
(203) 274-2825
| RAFAEL HOLDINGS, INC. | ||||||||
| CONSOLIDATED BALANCE SHEETS | ||||||||
| (in thousands, except share and per share data) | ||||||||
| July 31, 2025 | July 31, 2024 | |||||||
| ASSETS | ||||||||
| CURRENT ASSETS | ||||||||
| Cash and cash equivalents | $ | 52,769 | $ | 2,675 | ||||
| Available-for-sale securities | — | 63,265 | ||||||
| Prepaid clinical trial costs | 1,045 | — | ||||||
| Interest receivable | — | 515 | ||||||
| Convertible notes receivable, due from Cyclo | — | 5,191 | ||||||
| Other receivables | 1,206 | — | ||||||
| Accounts receivable, net of allowance for credit losses of $245 at July 31, 2025 and July 31, 2024 | 627 | 426 | ||||||
| Inventory | 281 | — | ||||||
| Prepaid expenses and other current assets | 786 | 430 | ||||||
| Total current assets | 56,714 | 72,502 | ||||||
| Property and equipment, net | 1,596 | 2,120 | ||||||
| Investments – Hedge Funds | — | 2,547 | ||||||
| Investments – Cyclo | — | 12,010 | ||||||
| Convertible notes receivable classified as available-for-sale | 1,858 | 1,146 | ||||||
| Goodwill | 19,939 | 3,050 | ||||||
| Intangible assets, net | 994 | 1,847 | ||||||
| In-process research and development | 31,575 | 1,575 | ||||||
| Non-current prepaid clinical trial costs | 1,399 | — | ||||||
| Other assets | 34 | 35 | ||||||
| TOTAL ASSETS | $ | 114,109 | $ | 96,832 | ||||
| LIABILITIES AND EQUITY | ||||||||
| CURRENT LIABILITIES | ||||||||
| Accounts payable | $ | 6,893 | $ | 2,556 | ||||
| Accrued expenses | 3,304 | 1,798 | ||||||
| Convertible notes payable | 614 | 614 | ||||||
| Other current liabilities | 66 | 113 | ||||||
| Due to related parties | 723 | 733 | ||||||
| Installment note payable | — | 1,700 | ||||||
| Total current liabilities | 11,600 | 7,514 | ||||||
| Accrued expenses, noncurrent | 3,895 | 2,982 | ||||||
| Convertible notes payable, noncurrent | 78 | 73 | ||||||
| Other liabilities | 27 | 5 | ||||||
| Deferred income tax liability | 138 | — | ||||||
| TOTAL LIABILITIES | $ | 15,738 | $ | 10,574 | ||||
| COMMITMENTS AND CONTINGENCIES | ||||||||
| EQUITY | ||||||||
| Class A common stock, $0.01 par value; 35,000,000 shares authorized, 787,163 shares issued and outstanding as of July 31, 2025 and July 31, 2024 | 8 | 8 | ||||||
| Class B common stock, $0.01 par value; 200,000,000 shares authorized, 50,789,697 issued and outstanding (excluding treasury shares of 101,487) as of July 31, 2025, and 24,142,535 issued and 23,819,948 outstanding (excluding treasury shares of 101,487) as of July 31, 2024 | 508 | 238 | ||||||
| Additional paid-in capital | 322,161 | 280,048 | ||||||
| Accumulated deficit | (232,263 | ) | (201,743 | ) | ||||
| Treasury stock, at cost; 101,487 Class B shares as of July 31, 2025 and July 31, 2024 | (168 | ) | (168 | ) | ||||
| Accumulated other comprehensive income related to unrealized income on available-for-sale securities | 358 | 111 | ||||||
| Accumulated other comprehensive income related to foreign currency translation adjustment | 3,787 | 3,691 | ||||||
| Total equity attributable to Rafael Holdings, Inc. | 94,391 | 82,185 | ||||||
| Noncontrolling interests | 3,980 | 4,073 | ||||||
| TOTAL EQUITY | 98,371 | 86,258 | ||||||
| TOTAL LIABILITIES AND EQUITY | $ | 114,109 | $ | 96,832 | ||||
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RAFAEL HOLDINGS, INC.
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CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)
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(in thousands, except share and per share data)
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| Three Months Ended July 31, | Year Ended July 31, | ||||||||||||||
| 2025 | 2024 | 2025 | 2024 | ||||||||||||
| Revenues | $ | 350 | $ | 165 | $ | 917 | $ | 637 | |||||||
| Cost of infusion Technology revenue | - | 69 | 106 | 154 | |||||||||||
| Cost of product revenue | 19 | - | 28 | - | |||||||||||
| G&A Expenses | 5,497 | 2,330 | 13,781 | 8,854 | |||||||||||
| R&D Expenses | 7,547 | 1,543 | 12,823 | 4,170 | |||||||||||
| In-process research and development expense | - | - | - | 89,861 | |||||||||||
| Depreciation and amortization | 50 | 68 | 288 | 225 | |||||||||||
| Loss on impairment of goodwill | - | - | 3,050 | - | |||||||||||
| Operating Loss | (12,763 | ) | (3,845 | ) | (29,159 | ) | (102,627 | ) | |||||||
| Interest income | 467 | 606 | 1,996 | 2,383 | |||||||||||
| Loss on initial investment in Day Three upon acquisition | - | - | - | (1,633 | ) | ||||||||||
| Realized gain on available-for-sale securities | - | 251 | 178 | 1,772 | |||||||||||
| Realized loss on investment in equity securities | - | - | - | (46 | ) | ||||||||||
| Realized gain on investment - Cyclo | - | - | - | 424 | |||||||||||
| Unrealized (loss) gain on investment - Cyclo | - | (3,162 | ) | (5,144 | ) | 37 | |||||||||
| Unrealized (loss) gain on convertible notes receivable, due from Cyclo | - | 1,191 | (719 | ) | 1,191 | ||||||||||
| Unrealized gain on investment - Hedge Funds | - | 181 | - | 63 | |||||||||||
| Recovery of receivables from Cornerstone | - | - | - | 31,305 | |||||||||||
| Interest expense | (168 | ) | (163 | ) | (658 | ) | (248 | ) | |||||||
| Other income, net | 236 | - | 310 | 118 | |||||||||||
| Loss before incomes taxes | (12,228 | ) | (4,941 | ) | (33,196 | ) | (67,261 | ) | |||||||
| Benefit from taxes | 174 | 87 | 2,553 | 2,680 | |||||||||||
| Equity in loss of Day Three | - | - | - | (422 | ) | ||||||||||
| Consolidated net loss | (12,054 | ) | (4,854 | ) | (30,643 | ) | (65,003 | ) | |||||||
| Consolidated net loss | (12,054 | ) | (4,854 | ) | (30,643 | ) | (65,003 | ) | |||||||
| Net loss attributable to noncontrolling interests | 40 | (386 | ) | (123 | ) | (30,593 | ) | ||||||||
| Net loss attributable to Rafael Holdings, Inc. | $ | (12,094 | ) | $ | (4,468 | ) | $ | (30,520 | ) | $ | (34,410 | ) | |||
| Loss per share attributable to common stockholders | |||||||||||||||
| Basic and diluted | $ | (0.28 | ) | $ | (0.19 | ) | $ | (1.04 | ) | $ | (1.45 | ) | |||
| Weighted average number of shares used in calculation of loss per share - basic and diluted | 43,011,360 | 23,916,839 | 29,422,221 | 23,745,516 | |||||||||||