IntraBio Inc., a late-stage biopharmaceutical company developing novel therapies for common and rare (“orphan”) neurodegenerative diseases, announced results for its lead compound series (IB1000s) for the treatment of dementia, including positive clinical results from compassionate-use studies in patients with Lewy body dementia and frontotemporal dementia, and from pre-clinical studies in Alzheimer’s disease.

Recent compassionate-use studies with IB1000s in patients with Lewy body dementia and frontotemporal dementia showed meaningful improvement in quality of life, cognition, mobility, speech, and a disease-modifying effect. Clinical examinations and gait analysis revealed marked improvement in gait and balance, and neuropsychological testing revealed measurable improvement of psychomotor function, alertness, and attention.

Additional pre-clinical in vivo studies (in Npc1−/− mouse model) investigating the effect of IB1000s on Alzheimer’s disease pathology were highly supportive of the treatment’s effect, demonstrating a statistically significant improvement in two critical biomarkers: microtubule-associated protein 1A/1B-light chain 3-phosphatidylethanolamine conjugate (LC3-II) was reduced by 68%, and amyloid precursor protein C-terminal fragments (APP-CTFs) was reduced by 28%.

The discovery of IB1000s’ symptomatic and neuroprotective properties and disease-modifying potential could be a breakthrough for patients suffering from a broad range of neurodegenerative diseases, including dementia.

“While the initial sample size is small, to have such a drug with a long-term excellent safety profile, showing promising effects in these intractable diseases is a very exciting and an important development,” commented Professor Antony Galione FRS FMedSci, statutory professor of pharmacology, University of Oxford and founding scientist of IntraBio.

Alzheimer’s disease, Lewy body dementia, and frontotemporal dementia together account for an estimated 85%-90% of all dementia cases, affecting more than 50 million people worldwide, and are expected to increase in prevalence over the next decades to affect more than 100 million people in 2050. They are progressive neurological disorders in which abnormal deposits of proteins accumulate in areas of the brain that regulate cognition, behavior and movement. As a consequence, they present with a wide range of cognitive, behavioral, and physical symptoms, in particular problems with memory, thought, sleep, motor function, and changes in behavior.

IntraBio, with its collaborators, has evaluated the effect of IB1000s in compassionate-use studies in over 175 patients, forming the scientific basis for IB1000s to be further investigated for the treatment of 18 indications, including neurodegenerative diseases and lysosomal storage disorders. Future opportunities to develop the IB1000s series further in additional indications include parkinsonisms, restless leg syndrome (RLS), amyotrophic lateral sclerosis (ALS), and pultiple sclerosis (MS), all of which of have high-unmet medical needs.

IntraBio is currently in the process of applying for multi-national clinical trials with its lead asset (IB1001) for the treatment of Niemann-Pick disease type C (NPC), GM2 gangliosidosis (Tay-Sachs and Sandhoff disease) and certain inherited cerebellar ataxias (CA).