Life Science Today 003 – BeiGene, ALS, Moderna, and Rheumatoid Arthritis.
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. Today on The Niche, big deals in Chinese big-pharma as BeiGene raises another $2 billion, Biogene and Moderna both release papers on crucial clinical trials, and a new cell-type could play a pivotal role in rheumatoid arthritis.
Big Money for BeiGene
BeiGene raised $2.08 billion last week on a registered direct offering of shares. This brings the Chinese big-pharma giant’s stock-pile to more than $5.5 billion in cash. Amgen alone put in $420 million, maintaining their priority status, holding 20.5% of the company. What is the 10-year-old company doing with all this money? Attempting to capture swathes of the rapidly expanding oncology market in China.
In the last decade, per capita spending on pharmaceuticals has nearly quadrupled in China. Years of a one-child policy means the aging middle class of over 400 million are spending more on new treatments. BeiGene has capitalized on this market in several critical ways. They have a pipeline of drugs, including 6 approved therapies and 37 targets in various stages of clinical research. The results are real with their leading drug BRUKINSA approved globally for the treatment of mantel cell lymphoma. Their PD-1 inhibitor, Tislelizumab, has garnered early approvals in China and is currently involved in more than 25 clinical trials for dozens of indications across the cancer space. In fact, there could be a launch of up to 8 products in the next 18 months.
The oncology space is notoriously fickle. So why all the confidence from foreign investment? The declaration on the homepage of beigene.com says it all: “Cancer has no borders. Neither do we.” Read further into the deal with Amgen and you find that BeiGene is providing an inroad to China for US developed drugs. As part of the deal, Amgen, who has more than 20 cancer therapies in the pipeline, can use BeiGene to launch in China. BeiGene is currently running over 60 clinical trials across 35 countries, but it is the control of the Chinese market that is a massive value-added proposition. Yes, their drug pipeline and past success makes them a strong company. But the ability to run trials in China, navigate their regulatory systems, and then successfully recruit physician backing from nearly 1000 hospitals means BeiGene is delivering a lot more than just an intellectually successful candidate pipeline. They have made themselves an indispensable liaison into the worlds largest growing drug market. BeiGene’s shares rose more than 25% this week closing at an all time high of $248.67 on Friday.
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Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS), often called Lou Gehrig’s disease, is a progressive degenerative disorder causing loss of muscle control eventually resulting in death. While most cases have no known cause, around 10% of individuals have a hereditary component. Copper zinc superoxide dismutase 1 (SOD1) is a protein implicated in driving inherited ALS. Biogene published results of a phase 1/2 clinical trial of Tofersen. The treatment uses an antisense RNA to SOD1. This works by binding the mRNA of the gene and preventing the protein from being formed. While the results showed promising biological outcomes and hinted at delayed loss-of-function in ALS patients, the treatment is given through an intrathecal injection directly into the cerebral spinal fluid, a less than ideal delivery mechanism. There was enough biological promise to merit proceeding to a phase 3 trial.
Moderna and COVID-19
Moderna has been dripping data about their mRNA COVID-19 vaccine into the market. Last week, they published their results in the New England Journal of Medicine. Overall, the vaccine appears to be functional with few serious adverse events and robust generation of immunity to COVID19 in serum samples. Will the positive results continue in a larger sample? The market certainly thinks so, with their shares increasing almost 50% last week.
Rheumatoid Arthritis Discovery
Rheumatoid arthritis is an autoimmune mediated inflammatory disease caused by the immune system attacking the synovium membrane surrounding joints. Painful and debilitating, a hallmark of many cases are seemingly random “flares” that heighten the disease symptoms. These can occur even when the disease is being treated with systemic immune suppressors. A recent study from Rockefeller University identified new preinflammatory mesenchymal (PRIME) cells which appear in the blood 1 to 2 weeks before a flareup occurs. PRIME cells may be an excellent target for future rheumatoid arthritis treatments.
To perform the study, researchers wanted access to RNA from the patients’ blood. This would historically require office visits to collect samples at regular intervals. To get around this, they designed a new buffering solution which allowed patients to provide samples at home using a simple pin prick and drop them in the mail. The buffering solution stabilized the blood till it could be analyzed in the lab. This technology could prove essential to tracking numerous diseases from home in the future and may have implications far beyond the specific problem it solved.
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 in the industry. 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.
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Story References
https://doi.org/10.1016/j.cct.2018.01.003
https://www.ppd.com/
https://www.businesswire.com/news/home/20200514005477/en/PPD-Honored-Quality-Capabilities-CRO-Leadership-Awards
https://www.clinicaltrialsarena.com/comment/biogen-aducanumab-fda-approval-alzheimers/
https://www.fiercebiotech.com/biotech/a-test-for-hahn-s-fda-biogen-submits-controversial-alzheimer-s-drug-aducanumab
https://www.gilead.com/news-and-press/press-room/press-releases/2020/7/gilead-presents-additional-data-on-investigational-antiviral-remdesivir-for-the-treatment-of-covid-19
Music by Luke Goodson