{
  "id": 10923891,
  "title": "Rafael drug misses primary endpoint in rare disease trial",
  "url": "https://urgent.news/2026/09/30/rafael-drug-misses-primary-endpoint-in-rare-disease-trial",
  "topic": "finance",
  "section": "Finance & Markets",
  "published": "2026-09-30T11:04:39.000Z",
  "source": {
    "name": "Investing.com",
    "slug": "investing-com",
    "url": "https://www.investing.com/news/company-news/rafael-drug-misses-primary-endpoint-in-rare-disease-trial-93CH-4924746"
  },
  "original_language": "en",
  "account": "NEWARK, N.J. - Rafael Holdings Inc. reported that its experimental drug Trappsol Cyclo did not meet the primary goal in a Phase 3 trial for a rare genetic disease, Niemann-Pick Disease Type C. The TransportNPC study included 94 patients aged 3 to 70, who received either Trappsol Cyclo or a placebo for a year. The trial assessed the 4-domain NPC Clinical Severity Scale at the trial's end. While Trappsol Cyclo showed a favorable direction, it failed to show statistical significance (p=0.19). However, when comparing patients on background medications, Trappsol Cyclo demonstrated a statistically significant effect (p=0.046). Serious side effects were reported more frequently in the treatment group (35.9%) compared to the placebo group (16.7%). Hearing issues were also more common in the treatment group (15.6%) than the placebo group (13.3%). Rafael Holdings plans to submit a New Drug Application to the FDA in late 2026.",
  "summary": null,
  "key_points": [
    "Rafael's Trappsol Cyclo failed primary endpoint in Niemann-Pick Disease trial",
    "94 patients in TransportNPC study, aged 3 to 70",
    "Serious side effects more common in treatment group"
  ],
  "editors_take": null,
  "illustration": null,
  "coverage": {
    "outlets": 1,
    "also_reported_by": []
  },
  "ai_generated": true,
  "disclaimer": "Summaries, key points and the editor’s take are written by software from other outlets’ reporting and may contain errors — always check the linked original."
}