Details:
AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C).
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 08, 2023
Details:
Patients treated with KH176 (sonlicromanol) for 28 days did not achieve statistically significant improvement in attention domain score of cognitive functioning, as assessed by the computerised Cogstate visual identification test, compared to those who received placebo.
Lead Product(s): Sonlicromanol
Therapeutic Area: Genetic Disease Product Name: KH176
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 22, 2022
Details:
Topline data from KHENERGYZE Phase IIb trial for KH176 (sonlicromanol) is expected in the third quarter of 2022, Phase IIa study showed a significant improvement in attention and mood-related outcomes in patients with MELAS spectrum disorder.
Lead Product(s): Sonlicromanol
Therapeutic Area: Genetic Disease Product Name: KH176
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 10, 2022
Details:
With the acceptance of the PIP, Khondrion plans to initiate a paediatric trial to evaluate the safety and efficacy of sonlicromanol in children from birth to less than 18 years with mitochondrial disease affecting oxidative phosphorylation and symptoms of motor impairment.
Lead Product(s): Sonlicromanol
Therapeutic Area: Genetic Disease Product Name: KH176
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 18, 2021
Details:
An ongoing Phase 2B clinical trial in patients with MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) spectrum disorders is investigating the effect of sonlicromanol on cognitive functioning.
Lead Product(s): Sonlicromanol
Therapeutic Area: Genetic Disease Product Name: KH176
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 27, 2020
Details:
The study’s primary objective is to evaluate the dose-effect of Sonlicromanol on the attention domain score of cognitive functioning, Results are expected in the second half of 2020.
Lead Product(s): Sonlicromanol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 27, 2020