How The Same Treatments May Work For Multiple Neurodegenerative Diseases
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Anyone who suffers from a neurodegenerative disease (NDD) or cares for someone who does knows how devastating these conditions are — and how challenging they are to diagnose and treat. A critical challenge with many of the already approved medications is that they don't get into the patient's brain because they don't cross the blood-brain barrier.
As a result, several companies have found that their proposed treatments for a particular NDD simply don't work very well or, in some cases, at all. However, extensive research on NDDs has uncovered a different treatment pathway that's enabling some biotechnology firms to target more than one NDD with the same drug candidate.
Why using the same medications for multiple NDDs makes sense
As many major pharmaceutical companies have discovered while developing treatments for autoimmune diseases, some medications do treat several of those diseases. A critical component of those treatments is inflammation, and several biotech companies are targeting the same issue in NDDs — even though inflammation was not traditionally thought to be important in the pathogenesis of NDDs.
A key advantage of developing the same medication for multiple diseases is that it increases the drug's total addressable market. In other words, the company developing the medication has more opportunities for revenue than it would if the treatment were only for one condition.
According to Research and Markets, the NDD treatment market will be worth about $42.4 billion this year and see a compound annual growth rate (CAGR) of about 3%. That would make the market worth approximately $52.8 billion by 2030.
Of the total market value, Alzheimer's treatments accounted for approximately $5.1 billion globally in 2022, while Parkinson's treatments were valued at around $5 billion. These two illnesses are projected to see CAGRs of 8% and 6.4%, respectively, making the Alzheimer's treatment market worth $6.9 billion in 2026 and the Parkinson's market worth $8 billion in 2030.
Some biotech companies are developing drug candidates that show promise in both Alzheimer's and Parkinson's, but those aren't the only NDDs with potential crossovers in treatments. Some of these other NDDs include other types of dementia and ALS.
What sets NDDs apart from other chronic illnesses is that most of them impact motor or cognitive functions or both. Alzheimer's, Parkinson's, and other NDDs mostly impact seniors because aging raises the risk of developing them. Even though these illnesses cause similar symptoms, researchers have not fully understood their causes or how they work and affect the body, which has restrained treatment development.
However, researchers have begun looking at inflammation as a possible driver of NDDs instead of just a symptom or result of them. This new approach to targeting these illnesses has led to some promising clinical trial results for the biotech companies that have adopted it.
Inflammation isn't the only new approach to treating NDDs that's being studied. Additionally, inflammation can be attacked in a variety of different ways. Here are four biotech companies currently developing novel treatments that are showing promise in more than one NDD.
Coya Therapeutics (COYA)
This company is targeting inflammation in Alzheimer's and amyotrophic lateral sclerosis, better known as Lou Gehrig's disease, or simply, ALS, using regulatory T cells (Tregs). Through its research, Coya has discovered a connection between neuroinflammation and dysfunctional Tregs, an issue that's been found to occur in multiple NDDs, including Parkinson's, Alzheimer's and ALS.
The company's COYA 301 drug candidate is a biologic that enhances Treg function to reduce inflammation. COYA 302 is a biologic that combines COYA 301 with abatacept, sold under the brand name Orencia, to block other inflammatory cell types.
Coya Therapeutics recently announced the results of an open-label study in Alzheimer's patients. It reported that COYA 301 was found to boost Treg function while halting the cognitive decline that is the hallmark of Alzheimer's.
In a separate proof-of-concept, open-label study in ALS, COYA 302 was also found to improve Treg function. In fact, patients who received COYA 302 experienced either zero decline or no decline in their symptoms. Coya Therapeutics used the Revised ALS Functional Rating Scale (ALSFRS-R) to measure the patients' symptoms over 48 weeks.
Athira Pharma (ATHA)
Athira is developing a treatment for Parkinson's, Lewy body dementia (LBD) and Alzheimer's. In fact, two of the clinical trials on its candidate fosgonimeton in Alzheimer's are now in Phase 2 and Phase 3, and enrollment in the open-label extension of Athira's Phase 2/3 clinical trial is underway.
Additionally, fosgonimeton is in an exploratory Phase 2 trial for LBD and Parkinson's disease dementia. Like Coya's drug candidate, Athira is targeting neuroinflammation. In fact, it has developed its proprietary drug discovery platform to target a wide range of neurological conditions with a small number of potential treatments.
Athira presented its recent findings at the International Conference on Alzheimer's and Parkinson's Diseases and Related Neurological Disorders (AD/PD) earlier this year. The company's presentations covered a number of preclinical evaluations showing that fosgonimeton or its active metabolite fosgo-AM can address multiple factors in neurodegeneration.
Alector (ALEC)
This company has been studying the immune system's role in NDDs and is targeting microglial cells, which are the brain's immune cells. With its AL001 and AL101, Alector aims to improve the function of these cells to redirect their activity inside the brain, treating frontotemporal dementia (FTD), ALS and Alzheimer's in the process.
Alector's drug candidates target the regulatory immune protein called progranulin to treat Alzheimer's and FTD. Importantly, deficiencies in this protein have been discovered in multiple NDDs, including Parkinson's and others. Progranulin actually regulates inflammation.
Among the results for its Phase 2 clinical trial on AL001, also referred to as latozinemab, Alector reported normalized cerebrospinal fluid and plasma levels of glial fibrillary acidic protein (GFAP). Separate studies have shown that FTD patients whose brains atrophy more quickly than in others usually have higher levels of GFAP. A Phase 3 trial on AL001 is now underway.
BioVie (BIVI)
BioVie is reporting promising results from its drug candidate NE3107, which targets Parkinson's and Alzheimer's. Thus far, BioVie's clinical trials have demonstrated improvements in both the motor symptoms that are the hallmark of Parkinson's and the cognitive symptoms that are widely known to impact Alzheimer's patients.
In the past, Alzheimer's researchers have targeted the ß-amyloid protein, which has been shown to accumulate in the brains of patients with the disease. On the other hand, the typical target of Parkinson's treatments has traditionally been the α-synuclein protein, which researchers believe reduces dopamine levels in the brains of patients with Parkinson's.
However, BioVie's NE3107 approaches both diseases by targeting inflammation. The company presented a summary of its latest findings in a poster presentation at the International Association of Parkinsonism and Related Disorders World Congress earlier this year.
Currently, the standard of care for Parkinson's is a combination of the drugs levodopa and carbidopa. BioVie reported that adding NE3107 to that standard of care saw their symptoms improve more than those receiving placebo with the standard of care. The trial used the Motor Disease Society – Unified Parkinson's Disease Rating Scale (MDS UPDRS) Part III to measure the symptom improvements.
Additionally, BioVie said five of the 19 patients who received NE3107 with the standard of care reported being in an "on state" in the morning, which means their tremors and other motor symptoms were well-controlled. On the other hand, none of the patients who received the placebo with the standard of care were in an "on state."
The company also reported improved biomarker, neuropsychological, and neurophysiological status in patients with Alzheimer's. In more than 60% of the 18 patients who received NE3107, BioVie observed lower levels of the TNF-α cytokine, pointing to a reduction in inflammation. The company also reported statistically significant memory and mood improvements in the patients who received its treatment.
Investing in treatments for multiple NDDs
Many biotech stocks have exploded in recent years as investors reacted to promising drug candidates and clinical trial results. Unfortunately, it can be a risky sector to invest in, but as with any other asset, the possibility of substantial rewards often accompanies significant risk.
It's always advisable for investors to do their own due diligence before making any investments, no matter what type of asset or sector they might be in. Within biotech, the high risk may mean an even deeper analysis is in order.
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Disclaimer: Coya Therapeutics and BioVie Inc. are clients of Quantum Media Group LLC, and Quantum Research Group LLC.
Disclosure: None.
Fascinating.