Rafael to continue phase 3 study of NPC treatment after DMC review

NEW YORK - Rafael Holdings, Inc. (NYSE: RFL), a clinical-stage pharmaceutical company with a market capitalization of $68 million, announced Wednesday that its 96-week pivotal phase 3 study of intravenous TrappsolCyclo for treating Niemann-Pick Disease Type C1 (NPC1) will continue following an independent Data Monitoring Committee review of safety and efficacy data at the prespecified 48-week interim analysis.

The FDA has also accepted the statistical analysis plan for the TransportNPC study, which is evaluating hydroxypropyl-beta-cyclodextrin as a potential treatment for the rare genetic disease.

The study has enrolled 94 patients across more than 25 sites in 13 countries. According to the company’s press release, the data indicates that the investigational drug is well-tolerated with a safety profile consistent with previously completed phase 1 and 2 studies.

"NPC is a rare, fatal, and progressive genetic disease, and there is a need for safe and effective treatment that addresses its root cause," said Howard S. Jonas, CEO of Rafael Holdings.

The primary endpoints of the study are the mean change in the 4-domain NPC Clinical Severity Scale score in the United States and the 5-domain NPC Clinical Severity Scale score in Europe. These scales measure disease progression across functions including ambulation, fine motor skills, speech, and swallowing.

NPC1 affects approximately 1 in 100,000 live births globally and is characterized by cells’ inability to transport and process cholesterol, resulting in accumulation that damages organs including the liver, spleen, and brain.

Rafael Holdings recently strengthened its financial position with the closing of a $25 million rights offering earlier this month, which the company states will support its strategic objectives. According to InvestingPro data, the company maintains a healthy liquidity position with a current ratio of 3.85 and holds more cash than debt on its balance sheet. While the company posted negative EBITDA of $16.17 million in the last twelve months, its revenue grew by 35.56% during the same period.

The company’s announcement was based on a press release statement issued Wednesday. The stock has shown strong momentum with a year-to-date return of 19.26%. InvestingPro offers additional insights with 6 more key tips about Rafael Holdings’ financial health and market position. Subscribers can access detailed analysis including Fair Value estimates and comprehensive financial metrics.

In other recent news, Rafael Holdings has made significant strides with its strategic initiatives. The company recently secured shareholder approval for its merger with Cyclo Therapeutics, with an overwhelming 99.92% voting in favor of the share issuance necessary for the acquisition. This merger is expected to bolster Rafael Holdings’ portfolio in the pharmaceutical sector. Additionally, the approval process demonstrated strong shareholder support, with 99.77% also agreeing to adjourn the meeting if more proxies were needed.

In another development, Rafael Holdings’ CEO, Howard S. Jonas, acquired 13,080,457 shares of the company, amounting to approximately $16.7 million. This insider purchase marks the first in over two years and suggests confidence in the company’s future prospects. The acquisition has increased Jonas’s ownership stake and is viewed by many as a positive indicator of the company’s potential. These recent developments highlight Rafael Holdings’ active engagement in expanding its business and reinforcing investor confidence.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.