Life Science Today 013 – Alzheimer’s, COVID19, AKG, Vitamin D, and Gene Therapy

Originally Published as The Niche Podcast

Introduction

Welcome to The Niche Podcast – Your weekly 5-minute rundown of the biotech, clinical research, and applied science industries. I’m your host, Dr. Noah Goodson. This week, another Alzheimer’s failure, COVID19 updates, two stories in the supplements space, and Taysha goes public.


AC Immune's Alzheimer's Failure

You can forget about an Alzheimer’s cure any time soon. Last week another approach to Alzheimer’s therapeutics bit this dust. For decades, a rabid industry-wide focus on Aß plaques has led to exactly zero breakthrough medications. The latest hope has been a pivot into Tau proteins. AC Immune reported last week that their Tau-targeting therapeutic semorinemab failed to slow the progression of Alzheimer’s. This is yet another disappointment in a long list of therapies with promising early results that fail to bear fruit. Biogen’s Aducanumab is still lurking in the potentially approvable but not that useful space. While it would be a blockbuster drug because of the paucity of options for those afflicted, significantly more work needs to occur before a truly life-changing therapy for Alzheimer’s emerges. In addition to the direct loss of 45% of their value, AC Immune’s results cast further doubt on the numerous Tau-targeting therapies in global pipelines.


J&J COVID19 Vaccine

Johnson & Johnson reported good safety results for a s ingle-dose COVID19 vaccine. This early-phase clinical trial was intended to assess the health and safety of the vaccine candidate Ad26.COV2.S. The published results suggest that patients acquired an immune response to COVID19, however the protective effect was not established within the parameters of this study. J&J has moved the vaccine forward to larger clinical trials to assess effectiveness. 

While far behind its competitors like Moderna and Pfizer in timeline, the J&J vaccine is designed to work at a single dose, rather than two, making it a superior option for many individuals. With about 7 billion unvaccinated people globally there is certainly enough market space for more than one company.


Sponsors

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AKG for Aging

The supplements market is worth more than $120 billion dollars globally. This is less than 10% of the value of the pharmaceutical market but more than the value of the entire sports industry in the United States – it is not a small market. Supplements may vary from multivitamins, to work-out boosters, to companies that make borderline therapeutic claims. Most supplements rely on small studies that back claims. We highlight two of those today. 

First is a study published in Cell Metabolism finding that Alpha-ketoglutarate (AKG) may be helpful in increasing longevity and vitality in aging. AKG is naturally occurring in humans and other mammals where it is involved in nitrogen transport, the Krebs cycle, and the formation of amino acids. The supplement is most often advertised to those looking to increase fitness results. Some previous work has suggested that it could increase longevity in certain contexts. 

This study from the Buck Institute for Research on Aging found that female mice on a specific caAKG supplement lived longer and had increased vitality across 31 metrics. There was also a decrease in certain kinds of inflammation and no major side effects. The company with patents on caAKG is currently backing research in humans and has used this supplement in some humans to suggest that it turns back the biological clock. How do you interpret complex data from mice and odd measurements of human biological age? I’d suggest healthy skepticism. This was work in mice and the life extension only happened for females, suggesting that the effects are sex-specific. Predicting biological age from DNA methylation is questionable at best. caAKG may be valuable in assisting in holistic health of some individuals. However, it seems unlike that a $150/month supplement will automatically extend your life by 10 years.


Vitamin D for COVID19

A deficiency in vitamin D may decrease your risk of severe symptoms of COVID19. The most recent study published in PlosOne last week compared the blood serum levels of vitamin D in 235 hospitalized COVID19 patents. Those with sufficient levels of vitamin D (>30ng/mL) survived at twice the rate of those with insufficient levels (<25ng/mL). Additionally, they experienced less severe symptoms across several metrics including direct measures of inflammation. This data, along with other studies and reports indicates that those with low vitamin D levels could benefit from supplementation. There is no evidence that this will prevent infection, nor will it cure the disease, but vitamin sufficiency will benefit your health whether you contract COVID19 or not.


Taysha Gene Therapy IPO

Taysha Gene Therapies has gone public with a $20/share IPO. Biotech IPOs have been quite successful across 2020. Taysha took a running jump onto this bandwagon and has moved rapidly from initial funding to IPO. The truncated time-frame from a $30 million seed round at the end of April to an IPO in September is very quick for the industry. But Taysha is doing things differently across the board. First, rather than a single gene therapy target, they have a wide suite of targets in the pipeline. How is this possible? The had significant financial backing from day one but far more important is a partnership with UT Southwestern that allows them to vet therapies and targets through the academic system, hand pick the most viable, and slide them into the company portfolio. Based on my read, the company is far from being profitable or even having a product. But they have focused on creating a business model designed to be successful in a risky space. I am clearly not alone in this positive outlook as shares rose by more than 20% after the initial offering to close at $24.06 on Friday.


Closing Credits

Thanks for joining me on The Niche Podcast; your weekly 5-minute summary of top news in the biotech, clinical trials, and applied science industries. You can find us on your favorite podcast app. Like, comment, subscribe, and most of all share with your friends. If you like what you hear, please rate and review, it really helps us. Once again, I’m Dr. Noah Goodson, I’ll see you next week. 

Sponsors

https://www.thescopemethod.com

Story References

https://ir.acimmune.com/news-releases/news-release-details/ac-immune-reports-top-line-results-tauriel-phase-2-trial

https://www.medrxiv.org/content/10.1101/2020.09.23.20199604v1.full.pdf

https://www.cell.com/cell-metabolism/fulltext/S1550-4131(20)30417-4

https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0239799

https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-announces-pricing-initial-public-offering

Music by Luke Goodson

https://www.soundcloud.com/lukegoodson

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Life Science Today 014 – Bristol Myers, Trump, NIH, and Sophia Genetics

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Life Science Today 012 – Probiotics, Lipid Nanoparticles, Transparency, and Immuno-therapy