Anyone who has struggled with a neurodegenerative disease (NDD) or has cared for someone who has one of these illnesses knows how challenging they are to treat and even diagnose. NDDs damage cognitive functions, motor functions, or sometimes both.
Many of these illnesses, including Parkinson's and Alzheimer's, primarily affect seniors because the risk of developing them increases with age. Unfortunately, a lack of understanding of how these diseases work and what causes them has restricted available treatments for them — despite the similarities often seen in the kinds of symptoms they cause.
What's even worse is that the field of NDD treatments has many past failures, which have led many large pharmaceutical companies to halt their attempts to treat such conditions.
However, a growing number of up-and-coming biotech firms aim to flip the script on this issue by searching for new types of treatments that target the root causes of these NDDs.
The value of expanding a drug's total addressable market
Some biotech firms have found that what works for one NDD often works for another. No matter the size of the company, there are clear benefits to developing one drug to treat multiple conditions. Such benefits are outsized for smaller biotechs as they race toward profitability by expanding the total addressable market for their candidate pipeline.
According to one estimate, the global market for NDD treatments should be worth about $42.4 billion in 2023, with a compound annual growth rate (CAGR) of about 3%, valuing the market at around $52.8 billion by 2030.
Of that total, Parkinson's disease treatment costs $14 billion annually in the U.S. Meanwhile, total payments in 2022 for health care, long-term care and hospice services for people with dementia age 65 and older were an estimated $321 billion in 2022.
The biotech companies that develop these drug candidates to treat multiple NDDs could see a fast path to profitability.
Four biotech firms are currently developing treatments targeting multiple neurodegenerative diseases (NDDs), listed from largest to smallest by market capitalization.
Amylyx Pharmaceuticals Inc.
Amylyx Pharmaceuticals Inc. NASDAQ: AMLX works to find a treatment for amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, progressive supranuclear palsy, Wolfram syndrome and Alzheimer's disease.
The U.S. Food and Drug Administration has already approved AMX0035 to treat ALS. Health Canada has also approved it but with conditions.
The drug is now reaching the end of Phase 2 trials to treat progressive supranuclear palsy, and Amylyx expects to begin a global Phase 3 trial in this illness sometime this year.
Trials for AMX0035 in Alzheimer's and Wolfram syndrome are both in Phase 2. The company expects topline results from its Wolfram trial in 2024. Amylyx is also evaluating the results of one of its other trials as it considers potential next steps to develop the drug to treat Alzheimer's.
Amylyx's pipeline also includes AMX0114 to treat ALS, additional unnamed NDDs, and other novel drug candidates.
Alector Inc.
Alector Inc. NASDAQ: ALEC is developing treatments that focus on the immune system's role in NDDs, focusing on microglia, a type of cell found in the brain. The company hopes to redirect the brain's problematic immune cell activity to improve function. Alector is currently working on a treatment for ALS, Alzheimer's and frontotemporal dementia (FTD).
AL001 and AL101 treat Alzheimer's and FTD by targeting the protein progranulin. Progranulin deficiency has been implicated in multiple NDDs, including not only Alzheimer's and FTD but also ALS, Parkinson's and others. Progranulin is a protein of the immune system that's essential for regulating inflammation.
Thus far, Alector has revealed some promising results from its clinical trials and is now moving forward with the next phases. With its third-quarter earnings report, the company said it had completed enrollment in its Phase 2 trial of AL002 in Alzheimer's in collaboration with AbbVie Inc. NYSE: ABBV.
Alector also completed enrollment of FTD patients for its Phase 3 clinical trial of latozinemab in collaboration with GlaxoSmithKline plc NYSE: GSK.
A Phase 2 trial of the drug revealed normalized levels of glial fibrillary acidic protein in FTD patients; typically, higher levels of this protein is associated with faster rates of brain atrophy in FTD.
Athira Pharma Inc.
Athira Pharma Inc. NASDAQ: ATHA is developing a platform to support the discovery of treatments for various neurological diseases and conditions, including multiple NDDs. The lead candidate in the company's pipeline is fosgonimeton, which is currently in clinical trials to treat Lewy body dementia (LBD), Parkinson's and Alzheimer's.
The drug candidate is currently in Phase 2 and 3 trials to treat Alzheimer's and in Phase 2 trials for Parkinson's disease, dementia and LBD. Athira is also developing ATH-1020 for neuropathic pain and NDDs and ATH-1105 to treat ALS.
Coya Therapeutics Inc.
Coya Therapeutics Inc. NASDAQ: COYA develops treatments that target regulatory T cells, or Tregs, to treat Alzheimer's and ALS. The company has linked dysfunctional Tregs with inflammation in the brain, implicated in multiple NDDs.
In an open-label study of COYA 301, an investigational low-dose interleukin-2 (IL-2) designed to reduce inflammation, COYA 301 increased Treg function while halting cognitive decline in Alzheimer's patients. The topline results from a randomized, double-blind, Phase 2 study with low-dose IL-2 should be released in summer 2024. A proof-of-concept, open-label study in ALS patients revealed that COYA 302 significantly improved Treg function.
COYA 302 combines COYA 301 with a proprietary formulation of abatacept, a drug sold under the brand name Orencia to treat rheumatoid arthritis and other autoimmune diseases. Over 48 weeks, the COYA 302 study found no decline or minimal decline in ALS symptoms, as measured on one scale commonly used to measure the severity of ALS symptoms. The company plans a randomized, double-blind, Phase 2 trial in the first half of 2024.
Investing in biotechs targeting NDDs
Recently, many biotech stocks have soared following promising results from clinical trials. Unfortunately, the sector can sometimes be extremely volatile, requiring investors to invest carefully.
However, what's interesting about investing in biotech companies is that they allow investors to support the battle against the specific neurodegenerative disease(s) they want to help fight. After all, most investors know at least one person who suffers or has suffered from one of these devastating illnesses.
With some due diligence and close attention to individual stocks in the biotech sector, investors may be able to uncover companies that are not only long-term winners but can also help treat the illnesses they are most concerned about.
Ari Zoldan is CEO of Quantum Media Group LLC, and Coya Therapeutics is a client of Quantum Media Group.
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