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The race to develop the first FDA-approved treatment for Niemann-Pick disease type C1 (NPC) is intensifying. Rafael Holdings and Cyclo Therapeutics have announced a merger aimed at advancing Cyclo’s lead drug candidate, Trappsol Cyclo, currently in phase 3 trials. NPC is a rare genetic disorder that disrupts the body’s ability to process cholesterol and lipids, leading to harmful accumulations in organs such as the liver and spleen. Cyclo’s CEO, N. Scott Fine, emphasized the partnership's role in progressing toward treatment options for this devastating condition.
Trappsol Cyclo, a formulation of hydroxypropyl beta cyclodextrin, is being studied in a phase 3 trial, with results from its 48-week interim analysis expected in 2025. Rafael, which began investing in Cyclo in 2023, has committed to funding the trial. The merger involves Rafael acquiring Cyclo’s shares at a valuation of $0.95 each, a move both companies believe will bolster the drug’s development and bring hope to patients and shareholders.
The urgency in NPC research is underscored by Zevra Therapeutics’ rival drug, arimoclomol, which has received support from an FDA advisory committee. Although initially rejected in 2021, Zevra resubmitted their application with additional data and could potentially receive FDA approval before year-end. If successful, it would compete with Cyclo’s Trappsol Cyclo in providing a much-needed therapy for NPC, which currently lacks FDA-approved treatments in the U.S.
