Alzheon
Overview
Alzheon, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for Alzheimer's disease (AD) and related neurodegenerative disorders. The company was founded in 2013 by Martin Tolar, MD, PhD, and is headquartered in Framingham, Massachusetts. Alzheon has successfully raised $150 million in its last two funding rounds, including a recent Series E funding of $100 million in June 2024. Alzheon concentrates on small molecule-based drugs, significantly its lead candidate, ALZ-801, aiming to become the first oral disease-modifying treatment for Alzheimer's disease.
Recent Developments
- June 2024: Alzheon secured $100 million in Series E funding, led by Alerce Medical Technology Partners. The proceeds will complete the pivotal APOLLOE4 Phase 3 trial for ALZ-801 in APOE4/4 homozygotes, a genetic subgroup at high risk for Alzheimer's.
- April 2024: The company initiated a long-term extension of the APOLLOE4 Phase 3 trial, offering participants who completed 78 weeks additional treatment to explore long-term safety and efficacy.
- February 2024: Interim results from the Phase 2 study of ALZ-801 showed a sustained reduction in plasma p-tau181, a biomarker for Alzheimer's, suggesting positive effects on disease modification.
- December 2023: The company presented long-term Phase 2 study results at the AAIC conference, demonstrating significant reductions in neurodegenerative biomarkers and supporting the oral ALZ-801 therapy's clinical efficacy.
- July 2023: At the Alzheimer's Association International Conference, Alzheon presented Phase 2 biomarker study findings that linked clinical benefits to decreased brain volume atrophy in APOE4 carriers.
- April 2023: Alzheon completed the enrollment of 325 patients for its pivotal APOLLOE4 Phase 3 trial, which aims to prove the efficacy of ALZ-801 in genetically targeted APOE4/4 individuals.
- January 2023: Alzheon announced its Phase 3 APOLLOE4 trial in APOE4/4 homozygotes had allowed a more inclusive patient population, enhancing the study’s applicability.
Company Information
| Attribute | Information |
|---|---|
| Founding Date | 2013 |
| Headquarters | Framingham, Massachusetts, USA |
| Founders | Martin Tolar, MD, PhD |
| Revenue | Not publicly disclosed |
| Profits | Not publicly disclosed |
| Key Investors | Alerce Medical Technology Partners, NIH |
| Industry | Biopharmaceutical |
| Number of Employees | Approximately 30 |
| Major Product | ALZ-801 (valiltramiprosate) |
Early History
Alzheon was founded in 2013 by Dr. Martin Tolar, focusing initially on exploring disruptive therapeutic approaches for Alzheimer's disease. Its lead drug candidate, ALZ-801 (valiltramiprosate), is derived from tramiprosate. It was developed to offer an oral alternative to monoclonal antibodies, pursuing a path less trodden by others in the Alzheimer’s pharmaceutical landscape. Early studies emphasized valiltramiprosate's ability to inhibit amyloid beta oligomer formation, a crucial factor contributing to Alzheimer's disease progression.
Company Profile and Achievements
Alzheon adopts a precision medicine approach, concentrating on genetically predisposed populations such as APOE4/4 homozygotes, who face the highest risk of developing Alzheimer's. Their investigational drug, ALZ-801, stands out due to its oral administration advantages over conventional plaque-clearing treatments.
Achievements:
- ALZ-801 received Fast Track designation from the FDA in 2017.
- Completed Series D and Series E funding rounds, raising over $150 million.
- Advanced ALZ-801 into pivotal Phase 3 trials (APOLLOE4) targeting a high-risk genetic subgroup.
- Presented promising Phase 2 biomarker data demonstrating sustained reductions in amyloid and tau biomarkers, supporting the drug's disease-modifying potential.
- Implementation of a long-term extension study to expand on safety and efficacy data.
Current Operations and Market Position
Alzheon operates at the forefront of Alzheimer’s research with its focus on the challenging APOE4 genetic segment. By progressing through rigorous clinical trials and incorporating extensive biomarker integration, Alzheon aims to establish ALZ-801 as the first oral therapy capable of modifying Alzheimer's pathology. Its ongoing Phase 3 trial is crucial for broadening therapy access, leveraging an oral form's simplicity and efficacy. If successful, ALZ-801 could secure a solid foothold as a treatment for Alzheimer's, enhancing patient compliance through oral administration.
Alzheon IPO
Alzheon has considered public offerings to support its ambitious clinical endeavors, though prior IPO attempts were postponed. Successful completion and positive results from its Phase 3 trials may reignite these plans, reflecting increased market confidence and financial viability.
Alzheon Funding
The company's latest funding initiatives include a significant $100 million in Series E financing acquired in mid-2024, led by existing investors Alerce Medical Technology and supported by new participants to facilitate the completion of crucial Phase 3 trials and regulatory filings by year-end.
Alzheon Phase 3
The APOLLOE4 Phase 3 trial, crucial for the future of ALZ-801, comprises a robust dataset to evaluate the effectiveness and safety of ALZ-801 in patients with early-stage Alzheimer's. This trial is pivotal for preparing a new drug application in 2024, aiming for a commercial launch by 2025 if regulatory milestones are achieved.
Conclusion
Alzheon, through its pioneering work on ALZ-801, is positioned to revolutionize Alzheimer's disease treatment. With a strong emphasis on safety and efficacy, especially in genetically susceptible populations, Alzheon could provide a crucial tool in combating Alzheimer's. Their patient-focused approach, leveraging oral drug design, creates a compelling pathway to market success, potentially leading to innovative treatments and changing the standard care framework. Continued pursuit of regulatory approval and successful trial outcomes could soon position Alzheon among leading contenders in transformative dementia therapeutics.