Investing in Inflammation: Thriving R&D in Immune-Mediated Neurological Disease Sector

With the help of advanced technologies such as artificial intelligence, gene editing, and precision medicine, biotech companies are now better equipped to develop new treatment strategies. One promising approach gaining significant attention in recent years involves targeting the overactive immune system and reducing systemic inflammation to mitigate the detrimental effects of neuroinflammation.

Evidence for the Role of Inflammation 

Per our current understanding, Parkinson’s begins as a result of abnormal accumulation of α-synuclein, a protein that blocks the transmission of dopamine in the brain. For over 30 years, researchers have understood that inflammation occurs in the brains of Parkinson’s patients, but only recently has it come to be seen as part of its etiology.

Studies have revealed that non-aspirin, non-steroidal anti-inflammatory drugs (NSAIDs) are associated with a significant reduction in Parkinson’s disease risk. Furthermore, researchers now believe that the abnormal accumulation of the α-synuclein protein into Lewy bodies could trigger the body’s inflammatory response, leading to damage and disease progression.

Researchers now believe they might not have to keep the α-synuclein protein from accumulating if they can halt the immune response. With a growing understanding of the role of this protein and its influence on the inflammatory response, the focus has shifted toward developing new drug candidates targeting this mechanism.

Neurodegenerative Disease Market 

Parkinson’s disease alone impacts approximately 1 million Americans, expected to rise 20% in the next decade. According to the 2023 Alzheimer’s Disease Facts and Figures report, an estimated 6.7 million Americans aged 65 or older live with Alzheimer’s dementia. As this population segment grows in the coming years, experts project it will reach 88 million by 2050.

In 2021, the global Parkinson’s disease treatment market was estimated to be worth $5.7 billion and projected to reach $10.4 billion by 2031, rising at a compound annual growth rate of 6.25. This growth is due to an increasing addressable market, a lack of practical innovation in the field, and the absence of a breakthrough treatment since levodopa was introduced over 50 years ago. Several companies are now working to change that, focusing on developing therapies that target the inflammatory component of Parkinson’s.

The Biotech Sector Promise

With rapid development in the neurodegenerative disease space, including continuously evolving treatment targets for Alzheimer’s and Parkinson’s disease, neurodegenerative diseases are emerging as a category that’s prime for investment. While the Nasdaq Composite has continued its precipitous decline, down 16% over the last 12 months, the Nasdaq Biotechnology Index rose by 2% – one of the only sectors to report gains.

From an investor standpoint, biotechnology company stocks can be a lucrative opportunity if their drug candidates can meet clinical trial targets and receive FDA approval. And promising developments in the field are reflected by growing investments in the research and development efforts of biotechnology companies. While there are thousands of biotech companies in the US alone, only a few of them have captured and held the market’s attention.

Companies and Drug Candidates To Watch:

BioVie, NE3107

BioVie’s NE3107 is a promising drug candidate for treating Parkinson’s disease. In December 2022, the company announced Phase 2 clinical trial results for the drug, which was tested in combination with levodopa and met both objectives. The primary objective was to determine safety and drug interactions with levodopa, while the secondary objective was to determine whether patients experienced improvements in motor-related symptoms.

The combination treatment of levodopa and NE3107 resulted in more significant improvements in patients’ symptoms as compared to those treated with levodopa alone. After 28 days of treatment, less than 64% of patients treated with levodopa alone saw a more than 30% improvement in symptoms, compared to 80% for those treated with levodopa and BioVie’s NE3107. Additionally, 89% of patients over 70 who received the combination treatment experienced an improvement of more than 30%.

Notably, researchers observed no drug-related adverse events in the Phase 2 clinical trial.

NE3107’s mechanism of action is to block extracellular signal-regulated kinase (ERK) activation in the brain. Blocking them prevents the production of tumor necrosis factor-alpha (TNF-α), widely considered the master regulator of inflammation. By blocking TNF-α, NE3107 is believed to halt the continual inflammatory cycle that leads to neurodegeneration.

Current Status: BioVie achieved the revised enrollment target for their Phase III trial in March 2023, and they expect top-line data in October 2023.

Annovis Bio, Buntanetap

Annovis Bio is a biotech company that has been making waves in the field of neurodegenerative diseases. The company’s novel inflammation-focused drug candidate, buntanetap, has shown promising results in Phase 2a trials for both Parkinson’s and Alzheimer’s patients. Buntanetap is designed to target multiple proteins implicated in neurodegenerative diseases and is found to be well-tolerated.

The Phase 2a trial of buntanetap showed a statistically significant improvement in motor function in Parkinson’s patients, which is a promising result. The drug’s ability to target multiple proteins implicated in neurodegenerative diseases makes it an attractive candidate for further development and testing.

In July 2022, Annovis received permission to proceed with a Phase III trial of their novel inflammation-focused drug candidate.

Current Status: The Company was Granted Approval for an additional 48 trial sites across five European Union countries as part of its ongoing Phase 3 study of buntanetap. 

Biohaven, BHV-8000

Biohaven, a recently revived biotech company, is exploring the potential of TYK2 inhibitors as a treatment for neurodegenerative diseases. TYK2 inhibitors have surfaced as effective therapeutics for common autoimmune disorders such as psoriasis. TYK2 proteins are crucial to the inflammatory signaling pathway between cells, making them a promising target for immune-mediated diseases.

The company recently closed a licensing deal with Chinese drugmaker Hangzhou Highlightll and plans to initiate a Phase 1 clinical trial to test whether blocking TYK2 in the brain can help treat neurological diseases associated with inflammation. Biohaven’s drug candidate, BHV-8000, is a first-in-class brain-penetrant, dual inhibitor of TYK2/JAK1. It could offer a broad therapeutic index with TYK2 inhibition and high selectivity for JAK1 inhibition without the severely limiting adverse class effects of JAK2/JAK3 inhibitors.

Biohaven CEO Vlad Coric is optimistic about the potential of TYK2 inhibitors in treating neurodegenerative diseases. “We have gained tremendous insight into the role of the immune system and critical inflammatory signaling pathways with events that drive the onset, propagation, and relentless progression of neurodegenerative diseases,” Coric said in a statement.

Current Status: Biohaven purchased the exclusive global rights for BHV-8000 to treat immune-mediated neurological disorders in March 2023. They plan to initiate a Phase 1 trial by the end of the year.

Numerous other treatments for Parkinson’s are in the pipeline of clinical trials exhibiting varying degrees of success. Many companies are altering existing drugs used in treating neurological diseases, developing new formulations or delivery methods for those commonly prescribed – highlighting the ingenuity of those developing brand-new compounds. These recent advancements hold great promise for developing more effective treatments that can slow down or halt the progression of neurodegenerative disorders, improving the lives of patients worldwide.

Explore Systemic Inflammation with Best-In-Class Live Education

Curious about the latest advances in neuroinflammation and emerging therapeutic options for increasingly prevalent immune-mediated neurodegenerative diseases? Join leading brain health and functional medicine experts at the 31st Annual Spring Congress – The Fire Inside: Inflammation: The Common Pathway to Disease between May 18-20, 2023, in Orlando, FL. Click here to learn more about the upcoming conference and save your spot today!