Introduction
Tricaprilin, also known as caprylic triglyceride or AC-1204, is a promising drug candidate developed by Cerecin Pte. Ltd. (formerly Accera) for the treatment of Alzheimer's disease and other neurological conditions. Here, we will delve into the current development status, clinical trial updates, and market projections for this innovative therapeutic agent.
Background and Mechanism
Tricaprilin is designed to induce mild chronic ketosis, providing an alternative energy source to glucose for brain cells. This approach is based on the observation that Alzheimer's disease is characterized by regional reductions in cerebral glucose utilization. By metabolizing caprylic acid into ketone bodies, tricaprilin aims to boost cellular metabolism and energy production via the citric acid cycle[1].
Clinical Trial Updates
Phase 1 and Phase 2 Trials
Cerecin has conducted several Phase 1 trials to evaluate the pharmacokinetics, safety, and tolerability of tricaprilin. In 2016, three Phase 1 studies were initiated in healthy volunteers to compare serum ketone body levels and interactions with different formulations and diets. These studies led to the selection of the AC-SD-03 formulation for further development due to its favorable pharmacokinetics and tolerability[1].
Phase 2/3 Trials
In December 2019, Cerecin registered a Phase 3 trial for AC-SD-03 (CER-0001), which was intended to enroll 300 patients with mild to moderate Alzheimer's disease. However, this trial was withdrawn in April 2023 before enrollment began. Instead, Cerecin initiated Phase 1 testing of a new formulation, AC-OLE-01-VA, in July 2022. A second trial comparing different dosing schedules started in November 2022 and was set to conclude in February 2023[1].
Current Phase 3 Trial
In April 2023, Cerecin registered a new Phase 3 trial for AC-OLE-01-VA, scheduled to start in January 2024. This trial aims to enroll 535 patients with mild to moderate Alzheimer's disease who are taking cholinesterase inhibitors or memantine. Participants will titrate to a dose of 20g twice daily for six months, with primary outcomes focusing on the ADAS-Cog and ADCS-Clinical Global Impression of Change[1].
Safety and Tolerability
Clinical trials have identified gastrointestinal side effects as the most common adverse reactions to tricaprilin, including abdominal distention, discomfort, nausea, indigestion, abdominal pain, distension, and diarrhea. However, these side effects have been manageable, and the maximum ketone concentrations and total plasma exposure have not differed significantly between Caucasian and Chinese participants[1].
Other Indications
Apart from Alzheimer's disease, tricaprilin is being explored for other neurological conditions. For instance, it has shown promise in migraine prevention, with positive results presented at the 65th Annual Society Meeting of the American Headache Society in 2023. Additionally, tricaprilin has been granted Orphan Drug designation for the treatment of infantile spasms, a rare form of epilepsy, and has demonstrated positive results in an open-label trial in Australia[1][5].
Market Projections
Alzheimer's Disease Market
The Alzheimer's disease market is expected to surge significantly, with a projected Compound Annual Growth Rate (CAGR) of 20% from 2019 to 2032. The market size was approximately $3.4 billion in 2022, with an estimated 15 million diagnosed prevalent cases in the 7MM (the United States, EU-4, the United Kingdom, and Japan)[2].
Competitive Landscape
Cerecin is part of a robust competitive landscape that includes other prominent companies such as Eli Lilly, Eisai, BioVie, and AB Science, among others. These companies are developing novel therapies, including monoclonal antibodies, small molecules, and gene therapies, which are expected to enter the market in the coming years. The convergence of novel therapies, advanced biomarker technologies, and personalized medicine is transforming the Alzheimer's treatment landscape[2][3].
Potential Impact
Tricaprilin's unique mechanism of action, which targets mitochondrial metabolism and provides an alternative energy source, positions it as a potential game-changer in the treatment of Alzheimer's disease. If successful, it could offer a new therapeutic option for patients, especially those who do not respond well to current treatments. The drug's potential in other neurological conditions, such as migraine prevention and infantile spasms, further broadens its market potential[1][5].
Key Takeaways
- Clinical Trials: Tricaprilin is currently in Phase 3 trials for Alzheimer's disease, with a new formulation (AC-OLE-01-VA) being tested.
- Mechanism: It induces mild chronic ketosis to provide an alternative energy source for brain cells.
- Safety: Common side effects include gastrointestinal issues, but these are generally manageable.
- Market Potential: The Alzheimer's disease market is projected to grow significantly, with tricaprilin offering a unique therapeutic approach.
- Other Indications: Tricaprilin is being explored for migraine prevention and infantile spasms.
FAQs
What is tricaprilin, and how does it work?
Tricaprilin is a proprietary oral formulation of caprylic triglyceride designed to induce mild chronic ketosis. It provides an alternative energy source to glucose for brain cells, which is particularly beneficial in Alzheimer's disease where cerebral glucose utilization is reduced.
What is the current clinical trial status of tricaprilin?
Tricaprilin is currently in Phase 3 trials for Alzheimer's disease, with a new formulation (AC-OLE-01-VA) being tested. The trial is scheduled to start in January 2024 and aims to enroll 535 patients.
What are the common side effects of tricaprilin?
The most common side effects of tricaprilin include gastrointestinal issues such as abdominal distention, discomfort, nausea, indigestion, abdominal pain, distension, and diarrhea.
Is tricaprilin being explored for other conditions besides Alzheimer's disease?
Yes, tricaprilin is being explored for other neurological conditions, including migraine prevention and infantile spasms. It has shown promise in these areas and has been granted Orphan Drug designation for infantile spasms.
What is the projected growth of the Alzheimer's disease market?
The Alzheimer's disease market is projected to grow at a CAGR of 20% from 2019 to 2032, with the market size estimated to be approximately $3.4 billion in 2022.
Which companies are key players in the Alzheimer's disease market?
Key players in the Alzheimer's disease market include Cerecin, Eli Lilly, Eisai, BioVie, AB Science, and several others. These companies are developing a range of novel therapies, including monoclonal antibodies, small molecules, and gene therapies.
Sources
- ALZFORUM: Tricaprilin | ALZFORUM
- GlobeNewswire: Alzheimer's Disease Market Predicted to Surge at a CAGR of 20% During the Study Period (2019–2032), Assesses DelveInsight
- DelveInsight: Clinical Trials on Alzheimer's Disease Conference 2024 - DelveInsight
- Patsnap Synapse: Tricaprylin - Drug Targets, Indications, Patents - Patsnap Synapse
- Clinical Trials Arena: Cerecin presents findings from Phase II trial for tricaprilin
Last updated: 2025-01-02
