“We remain pleased with the progress of our pivotal Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1, which the Data Monitoring Committee (DMC) recommended continuing after their review of prespecified safety and efficacy data at 48 weeks. We believe that Trappsol® Cyclo™ could provide an important new treatment option for patients suffering from this rare and fatal genetic disease,” said
“We are on track to complete this 96-week Phase 3 clinical trial, the largest ever conducted in this indication, and report preliminary top line results in the third quarter of this calendar year,” said
As of
For the three months ended
Research and development expenses were
General and administrative expenses were
For the six months ended
Research and development expenses were
General and administrative expenses were
About
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
Contact:
Barbara.ryan@rafaelholdings.com
(203) 274-2825
| CONSOLIDATED BALANCE SHEETS | |||||||
| (in thousands, except share and per share data) | |||||||
| (Unaudited) | |||||||
| ASSETS | |||||||
| CURRENT ASSETS | |||||||
| Cash and cash equivalents | $ | 37,779 | $ | 52,769 | |||
| Prepaid clinical costs | 3,229 | 1,045 | |||||
| Other receivables | — | 1,206 | |||||
| Accounts receivable, net of allowance for credit losses of | 295 | 627 | |||||
| Inventory | 271 | 281 | |||||
| Prepaid expenses and other current assets | 873 | 786 | |||||
| Total current assets | 42,447 | 56,714 | |||||
| Property and equipment, net | 1,545 | 1,596 | |||||
| Investments | 750 | — | |||||
| Convertible notes receivable classified as available-for-sale | 1,858 | 1,858 | |||||
| 19,939 | 19,939 | ||||||
| Intangible assets, net | 929 | 994 | |||||
| In-process research and development | 31,575 | 31,575 | |||||
| Non-current prepaid clinical costs | 244 | 1,399 | |||||
| Other assets | 28 | 34 | |||||
| TOTAL ASSETS | $ | 99,315 | $ | 114,109 | |||
| LIABILITIES AND EQUITY | |||||||
| CURRENT LIABILITIES | |||||||
| Accounts payable | $ | 8,172 | $ | 6,893 | |||
| Accrued expenses | 2,778 | 3,304 | |||||
| Convertible notes payable | 608 | 614 | |||||
| Other current liabilities | 66 | 66 | |||||
| Due to related parties | 733 | 723 | |||||
| Total current liabilities | 12,357 | 11,600 | |||||
| Accrued expenses, noncurrent | 3,483 | 3,895 | |||||
| Convertible notes payable, noncurrent | 52 | 78 | |||||
| Other liabilities | 27 | 27 | |||||
| Deferred income tax liability | 138 | 138 | |||||
| TOTAL LIABILITIES | 16,057 | 15,738 | |||||
| COMMITMENTS AND CONTINGENCIES | |||||||
| EQUITY | |||||||
| Class A common stock, | 8 | 8 | |||||
| Class B common stock, | 513 | 508 | |||||
| Additional paid-in capital | 323,081 | 322,161 | |||||
| Accumulated deficit | (248,496 | ) | (232,263 | ) | |||
| (168 | ) | (168 | ) | ||||
| Accumulated other comprehensive income related to unrealized income on available-for-sale securities | 358 | 358 | |||||
| Accumulated other comprehensive income related to foreign currency translation adjustment | 3,873 | 3,787 | |||||
| Total equity attributable to | 79,169 | 94,391 | |||||
| Noncontrolling interests | 4,089 | 3,980 | |||||
| TOTAL EQUITY | 83,258 | 98,371 | |||||
| TOTAL LIABILITIES AND EQUITY | $ | 99,315 | $ | 114,109 | |||
| CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | |||||||||||||||
| (unaudited, in thousands, except share and per share data) | |||||||||||||||
| Three Months Ended | Six Months Ended | ||||||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||||||
| REVENUE | |||||||||||||||
| Infusion Technology | $ | — | $ | — | $ | — | $ | 51 | |||||||
| Rental – Third Party | 57 | 48 | 113 | 98 | |||||||||||
| Rental – | 29 | 29 | 59 | 56 | |||||||||||
| Product revenue | 125 | — | 279 | — | |||||||||||
| Total revenue | 211 | 77 | 451 | 205 | |||||||||||
| COSTS AND EXPENSES | |||||||||||||||
| Cost of Infusion Technology revenue | — | 38 | — | 75 | |||||||||||
| Cost of Product revenue | 12 | — | 21 | — | |||||||||||
| General and administrative | 2,270 | 2,591 | 5,108 | 5,114 | |||||||||||
| Research and development | 4,529 | 947 | 12,013 | 2,273 | |||||||||||
| Depreciation and amortization | 55 | 90 | 105 | 176 | |||||||||||
| Loss on impairment of goodwill | — | 3,050 | — | 3,050 | |||||||||||
| Loss from operations | (6,655 | ) | (6,639 | ) | (16,796 | ) | (10,483 | ) | |||||||
| Interest income | 337 | 489 | 736 | 1,057 | |||||||||||
| Realized (loss) gain on available-for-sale securities | — | (16 | ) | — | 178 | ||||||||||
| Unrealized gain (loss) on investment - Cyclo | — | 614 | — | (3,751 | ) | ||||||||||
| Unrealized gain (loss) on convertible notes receivable, due from Cyclo | — | 486 | — | (1,102 | ) | ||||||||||
| Interest expense | (161 | ) | (163 | ) | (321 | ) | (325 | ) | |||||||
| Other (loss) income, net | (1 | ) | (78 | ) | 80 | (80 | ) | ||||||||
| Loss before income taxes | (6,279 | ) | (5,307 | ) | (16,066 | ) | (14,506 | ) | |||||||
| Provision for income taxes | (48 | ) | (20 | ) | (58 | ) | (32 | ) | |||||||
| Consolidated net loss | (6,327 | ) | (5,327 | ) | (16,124 | ) | (14,538 | ) | |||||||
| Net income (loss) attributable to noncontrolling interests | 90 | (686 | ) | 109 | (891 | ) | |||||||||
| Net loss attributable to | $ | (6,417 | ) | $ | (4,641 | ) | $ | (16,233 | ) | $ | (13,647 | ) | |||
| Loss per share attributable to common stockholders | |||||||||||||||
| Basic and diluted | $ | (0.13 | ) | $ | (0.19 | ) | $ | (0.32 | ) | $ | (0.57 | ) | |||
| Weighted average number of shares used in calculation of loss per share | |||||||||||||||
| Basic and diluted | 51,226,095 | 24,150,218 | 51,217,699 | 24,121,186 | |||||||||||
Source: 