Cyclo Therapeutics

Phase 3 Trial Shows Delgocitinib Cream Effective for Adolescent Chronic Hand Eczema

3 months ago

• LEO Pharma's delgocitinib 20 mg/g cream demonstrated significant efficacy in Phase 3 DELTA TEEN study for adolescents aged 12-17 with chronic hand eczema. • The topical pan-JAK inhibitor met both primary and secondary endpoints in treating moderate to severe chronic hand eczema, addressing an important unmet medical need. • The positive results support potential regulatory submissions for adolescent use of delgocitinib, which is already approved for adults in several regions.

Trappsol Cyclo Shows Promise in Stabilizing or Improving Niemann-Pick Disease Type C1 in Young Patients

4 months ago

• Preliminary data from Cyclo Therapeutics' TransportNPC trial shows that 87% of patients under three years old with Niemann-Pick Disease Type C1 (NPC1) demonstrated stabilization or improvement after 24 weeks of Trappsol Cyclo treatment. • Six out of seven patients who continued Trappsol Cyclo treatment for 48 weeks showed continued improvement, suggesting sustained efficacy over a longer duration. • Trappsol Cyclo targets cholesterol mobilization, addressing neurodegeneration associated with NPC1, and Cyclo Therapeutics plans to seek regulatory approval from the FDA and EMA based on these results. • The ongoing TransportNPC study is a randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of Trappsol Cyclo in a broader NPC1 patient population.

Blarcamesine Shows Sustained Cognitive Benefits in Early Alzheimer's Patients

4 months ago

• Anavex Life Sciences' blarcamesine demonstrates significant reduction in clinical decline over three years for early Alzheimer's patients. • Delayed-start analysis reveals early treatment with blarcamesine leads to greater cognitive stability, highlighting the importance of early intervention. • The ATTENTION-AD trial confirms blarcamesine's favorable safety profile, with mostly mild to moderate adverse events and no treatment-related deaths. • Peer-reviewed data from a Phase IIb/III study published in JPAD supports blarcamesine's potential as a novel oral treatment for early Alzheimer's.

FDA Approves First Treatments for Niemann-Pick Type C, Offering New Hope to Patients

8 months ago

• The FDA has approved two new treatments for Niemann-Pick Type C, a rare and fatal genetic disorder, marking a significant milestone for patients and families. • Miplyffa (arimoclomol) by Zevra Therapeutics, in combination with miglustat, is approved for neurological symptoms in patients aged 2 and older. • Aqneursa (levacetylleucine) by IntraBio Inc. has also been approved as a stand-alone therapy for neurological manifestations of Niemann-Pick disease type C. • These approvals underscore the FDA's commitment to supporting the development of new treatments for rare diseases, providing hope for improved management of symptoms.