After decades of nothing but frustration, 2023 has been a watershed year in the battle against Alzheimer’s Disease. The FDA has approved two new agents proven to slow the progress of the dread disease by 30-60%, and Medicare has agreed to cover the new agents. While these results still leave much to be desired, they mark what could be a real turning point: whereas before biotechs only saw failure and financial ruin, now many are willing to take a risk on Alzheimer’s research. Below we discuss three biotech innovators aiming to make their mark on this burgeoning new treatment market.
By Sudheer Narasimha, Biotech Analyst
1) Axsome Therapeutics ($AXSM)
Axsome Therapeutics is a biopharmaceutical company that develops innovative therapies for various central nervous system (CNS) disorders, including Alzheimer’s disease. AXS-05 is one of their experimental drugs, and it represents a novel approach to tackling the complications of Alzheimer’s disease. Many caregivers of Alzheimer’s patients can tell you horror stories about patients acting out with intense verbal or physical violence, a challenge which worsens an already tough situation for caregivers. AXS-05 is specially formulated to help with this kind of Alzheimer’s associated agitation.
As of now, AXS-05 has been FDA approved making it a breakthrough for the company and Alzheimer’s treatment as a whole. AXS-05 is a combination drug that consists of bupropion (a norepinephrine-dopamine reuptake inhibitor) and dextromethorphan (an NMDA receptor antagonist). This unique combination is believed to work synergistically to address multiple pathways implicated in Alzheimer’s disease, potentially offering a more comprehensive treatment approach. Axsome could establish itself as a key player in the Alzheimer’s therapeutics market, potentially commanding premium pricing for AXS-05 due to its distinct mechanism of action and potential benefits over existing treatments. Moreover, being one of the few approved therapies for Alzheimer’s, Axsome could benefit from market exclusivity, further enhancing its profitability. The revenue generated from sales of AXS-05 in the Alzheimer’s market could be reinvested into the company’s research and development efforts, supporting the exploration of other potential CNS therapies and driving further growth. Additionally, successful commercialization of AXS-05 in Alzheimer’s could also open doors for exploring its potential in other CNS disorders, expanding Axsome’s market reach and potential for long-term profitability.
2) Alnylam Pharmaceutical ($ALNY)
Another potential big player in the world of Alzheimer’s is Alnylam Pharmaceutical. Alnylam Pharmaceuticals is a leading biopharmaceutical company focused on the development of novel RNA interference (RNAi) therapeutics. Founded in 2002, Alnylam is a pioneer in the field of RNAi, which is a natural cellular process that regulates the activity of specific genes. The company’s proprietary RNAi platform allows for the targeted silencing of disease-causing genes, offering a promising avenue for treating a wide range of genetic and rare diseases. Alnylam’s commitment to scientific excellence and cutting-edge research has led to the successful development of several groundbreaking RNAi-based treatments that have been approved by regulatory agencies. Alnylam continues to advance a robust pipeline of investigational therapies across various therapeutic areas, striving to bring hope and improved quality of life to patients with unmet medical needs. Alynlam can use its RNAi technology to revolutionize Alzheimer’s treatment, which they are already in the process of doing. RNA interference (RNAi) holds great promise as a potential therapeutic approach for Alzheimer’s disease. Alzheimer’s is characterized by the accumulation of abnormal proteins, such as beta-amyloid and tau, in the brain, leading to neurodegeneration and cognitive decline. RNAi technology allows for the targeted silencing of specific genes responsible for producing these toxic proteins. By using small RNA molecules to interfere with the expression of these genes, RNAi can potentially reduce the production of beta-amyloid and tau, thereby slowing or halting disease progression. Just this past July, the company announced positive phase 1 results, demonstrating a proof of concept, that RNAi can be used to reduce harmful proteins in the brain.
Additionally, RNAi can be designed to target other genes involved in inflammation and neuronal damage, offering a multi-faceted approach to tackling Alzheimer’s. Although challenges remain, such as the effective delivery of RNAi molecules to the brain, ongoing research and advancements in the field continue to show promise in harnessing the power of RNAi for Alzheimer’s therapy, offering hope for a future where this devastating disease can be more effectively managed or even treated. Overall, Alnylam seems to be another key player in the Alzheimer’s market who could prove to be very profitable in the future.
3) AC Immune ($ACIU)
The final potentially big player in the Alzheimer’s field is AC Immune. Founded in 2003, AC Immune specializes in developing therapies and diagnostics targeting misfolded proteins associated with Alzheimer’s disease, Parkinson’s disease, and other neurodegenerative disorders. The company leverages its expertise in understanding the complex interactions between proteins in the brain to create innovative drug candidates that aim to prevent, slow, or halt disease progression. AC Immune’s approach includes the development of vaccines and small molecule drugs to target the abnormal protein aggregates, such as beta-amyloid and tau, which play a critical role in the pathogenesis of neurodegeneration. Furthermore, AC Immune collaborates with leading pharmaceutical companies to advance their research and increase the potential impact of their treatments. One of these leading pharmaceutical companies is Johnson and Johnson. In 2021, AC Immune published a press release that showcased positive phase 2 results for their new antibody that targeted the Tau protein. This kind of treatment has no approved rivals, currently. Targeting the Tau protein is considered a promising approach for several reasons. Firstly, tau pathology is closely associated with the cognitive decline observed in Alzheimer’s patients, making it a key target for intervention. By reducing the levels of pathological tau, the treatment aims to slow down or halt the neurodegenerative process and potentially preserve cognitive function. In fact, AC Immune has a whole cohort of different therapies that target the dysfunctional Tau protein. Many thought leaders believe this kind of innovation is likely to be profitable in the future.
According to the American Alzheimer’s Association, more than six million Americans are currently living with Alzheimer’s. As the Baby Boom generation continues to age, this staggering number could double. These stark facts represent a powerfully enticing risk/reward calculation to all three companies listed above. Now that we have two FDA approved Alzheimer’s treatments, we have proven that humans are capable of generating new science that can modify the disease state. However, the two recently approved treatments still leave much to be desired, and the field is wide open for any biotech brave enough to push forward with new approaches. Axsome Therapeutics, Alnylam Pharmaceuticals and AC Immune have accepted the challenge.