FRAMINGHAM, Mass.--(BUSINESS WIRE)--Alzheon, Inc., a clinical stage biopharmaceutical company focused on developing new medicines for patients suffering from Alzheimer’s disease (AD) and other neurological and psychiatric disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead clinical investigational drug, ALZ-801, for the treatment of Alzheimer’s disease.
Alzheon is implementing a Precision Medicine approach for treating Alzheimer’s disease (AD) by evaluating ALZ-801 based on genetic markers and stage of the disease. The initial pivotal Phase 3 program for ALZ-801 will focus on approval in the genetically-defined subpopulation of high risk patients who are homozygous for the ε4 allele of apolipoprotein E (APOE4/4 homozygotes) at the Mild stage of AD. Future clinical plans include expanding the evaluation of ALZ-801 in additional populations of AD patients. To date, there are no approved drugs that target the underlying pathology, and slow the progressive cognitive and functional decline of Alzheimer’s disease.
Fast Track is the FDA process designed to facilitate the development, and expedite the review of investigational drugs to treat serious conditions and fill an unmet medical need. When granting Fast Track designation, the FDA evaluates whether a drug will affect factors such as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress to a more serious condition. With Fast Track designation, early and frequent communications between the FDA and the Sponsor is encouraged throughout the drug development and review process to help to ensure that questions are resolved quickly, often leading to earlier drug approval.
"We are pleased that the FDA has granted Fast Track designation to ALZ-801 development in Alzheimer’s disease," said Martin Tolar, MD, PhD, Founder, President and Chief Executive Officer of Alzheon. "We have built a strong body of original research and clinical analyses that support the initial evaluation of ALZ-801 in the genetically-defined high risk population of APOE4/4 homozygotes, enabling us to pioneer a Precision Medicine approach in Alzheimer’s, and to move toward an upcoming pivotal study and potential approval as quickly as possible. We look forward to working closely with the FDA, as we seek to make progress in developing a disease modifying treatment for Alzheimer’s disease to address the needs of Alzheimer’s patients and their families who struggle with this devastating disease."
ALZ-801 is a novel, oral anti-amyloid drug candidate that is an optimized prodrug of tramiprosate, which has shown promising results in analyses of clinical data and therapeutic mechanism of action. This includes the discovery of its novel molecular mechanism of action blocking the formation of toxic amyloid oligomers1 associated with the development and progression of AD.2 The clinical data for ALZ-801 and its active agent, tramiprosate, suggest long-term clinical efficacy in AD patients with the APOE4 genotype, along with a favorable safety profile.3 The initial Phase 3 program for ALZ-801 will focus on patients with the homozygous APOE4/4 genotype at the Mild stage of AD, with the potential for future expansion to additional Alzheimer’s populations.
About Apolipoprotein E
Apolipoprotein E, or APOE, is a gene that provides a predictive window into an individual’s Alzheimer’s disease prognosis. In the brain, apolipoprotein E helps shuttle cholesterol to neurons to support their normal function. There are three forms, or alleles, of the APOE gene, called ε2, ε3 and ε4. The ε4 allele has been found to correlate with high risk and earlier onset of Alzheimer’s disease. It is estimated that up to 65% of all AD patients in the U.S. are carriers of at least one APOE4 allele, and that 10-15% of the AD population, or approximately 560,000 individuals in the U.S., are APOE4/4 homozygotes.4 APOE4 carriers – and more considerably APOE4/4 homozygotes – show faster rates of cognitive decline, at pre-symptomatic, early and dementia stages of the disease.5 In addition, APOE4 carriers, in comparison to non-carriers, show a higher and faster accumulation of amyloid pathology, including soluble amyloid oligomers.6
Alzheon, Inc. is committed to developing innovative medicines by directly addressing the underlying pathology of devastating neurodegenerative disorders. Our lead Alzheimer’s clinical candidate, ALZ-801, is a Phase 3-ready, first-in-class, small molecule oral inhibitor of amyloid aggregation and neurotoxicity – hallmarks of Alzheimer’s disease. ALZ-801 is a novel prodrug that builds on the safety and efficacy profile of the active compound tramiprosate, which has been evaluated in clinical trials involving over 2,000 Alzheimer’s patients. Our clinical expertise and technology platform is focused on developing drug candidates using a Precision Medicine approach based on individual genetic and biological information to advance therapies with the greatest impact for patients.
et al. 2017
2 Esparza et al. 2013, Lesne et al. 2012
3 Abushakra et al. 2016, Abushakra et al. 2017
4 Ward et al. 2012
5 Caselli et al. 2009; Martins et al. 2005
6 Grimmer et al. 2010, Hashimoto et al. 2012