Alkahest‘s Phase 2 clinical trial testing GRF6019 — an experimental blood-derived protein — in patients with mild to moderate Alzheimer’s disease has dosed its first participant, the company recently announced.
The trial (NCT03520998), which will assess the safety, tolerability, and feasibility of GRF6019, is still recruiting participants. Researchers expect to enroll 40 patients.
“The initiation of this study represents a significant milestone for Alkahest, and reflects our commitment to developing innovative therapies to treat this devastating disease,” Karoly Nikolich, president and CEO of Alkahest, said in a press release.
Proteins in the circulatory system can directly affect motor and cognitive function. GRF6019 is a blood plasma-derived protein being developed by Alkahest in collaboration with Grifols to treat neurodegenerative disorders. The compound was found to improve age-related cognitive and motor decline in preclinical studies. Most importantly, the therapy is not restricted by blood type matching and is optimized for therapeutic uses.
In a previous open-label clinical trial — the PLasma for Alzheimer SymptoM Amelioration (PLASMA) study (NCT02256306) — researchers found that repeated intravenous infusions of blood from young donors, 18 to 30 years old, into patients with mild to moderate Alzheimer’s were well-tolerated and significantly improved patients’ functional activity.
“Based on the promising data from our PLasma for Alzheimer SymptoM Amelioration (PLASMA) study last year, we have progressed the development of GRF6019, a proprietary plasma fraction developed and provided by Grifols, our development and clinical partner,” Nikolich said.
“We are excited to advance this lead plasma-derived product into the clinic, and we look forward to working with clinical experts, patients and their families as we continue to pursue this novel approach to the treatment of Alzheimer’s disease,” he said.
In the trial, patients are being randomized to one of two doses of GRF6019 — a high or a low dose. The therapy is administered once a day, directly into the vein, for five consecutive days during the first week. The same scheme is repeated during week 13 for a total of 10 doses per participant.
At the end of the six-month study, researchers will assess the therapy’s safety by measuring the incidence of treatment-related adverse events. Additional objectives will include efficacy parameters, such as changes in the scores of the Alzheimer’s Disease Assessment Scale-Cognitive Subscale (ADASCog/11) and clinical dementia rating scale. Researchers will evaluate patients’ response at the beginning and end of the study.
“As the population ages, the number of people with Alzheimer’s disease and other neurodegenerative diseases will increase sharply, precipitating an urgent need for innovative therapies,” said Sam Jackson, MD, Alkahest’s chief medical officer.