FDA Approves Drug to Treat Rare Autoimmune Disease

The U.S. Federal Drug Administration (FDA) has approved a drug manufactured by ChemoCentryx to treat a rare group of autoimmune diseases, called anti-neutrophil cytoplasmic autoantibody-associated vasculitis, or ANCA-AAV for short. According to ANCA Vasculitis News, ANCA-AAV causes inflammation and damage to the body’s small blood vessels. This inflammation is the result of antibodies that bind to certain cells of the immune system, called neutrophils, and overly activate them.

Since small blood vessels are found throughout the body, ANCA-AAV causes a variety of symptoms, including:

  • Poor kidney function, leading to kidney failure
  • Severe respiratory problems, including shortness of breath, hoarse voice, cough with blood or mucus, and chest pain
  • Neurological symptoms, including tingling, burning, numbness, and weakness
  • Central nervous system (CNS) involvement, such as headaches, cognitive impairment, and memory deficits. In severe cases, seizures, paralysis or loss of consciousness may also result.
  • Ear, nose, and throat (ENT) symptoms, like sinusitis, nasal discharge, rhinitis, and tinnitus (ringing in the ears)
  • Eye symptoms, including eye pain, vision impairment, and vision loss
  • Joint pain, muscle pain, and muscle loss
  • Skin lesions, including rashes, sores, ulcers, bumps, and bleeding underneath the skin
  • Digestive problems, like vomiting, diarrhea, nausea, abdominal pain, and blood in one’s stool
  • And more…

Due to the numerous symptoms ANCA-AAV causes, it can often be fatal for patients; in fact, ChemoCentryx states that the first year mortality rate for patients living with the disease is between 11 and 18 percent. Current ANCA-AAV treatments on the market include steroids and immunosuppressant medications which compromise the body’s ability to fight off infections. That’s why having a new treatment on the market that works via a different mechanism may be a saving grace for many ANCA-AAV patients.

The new drug, called avacopan, will be sold by ChemoCentryx under the brand name Tavneos. It works by blocking a protein called C5a receptor that is responsible for causing numerous inflammatory diseases. The drug’s wholesale price will be an astronomical $150,000-$200,000 per patient per year. However, it could be the life-saving treatment that the 40,000 ANCA-AAV patients in the US need.

The drug is currently being tested for use on other conditions as well, including the autoimmune skin disease Hidradenitis Suppurativa (HS). The drug has already received approval for commercialization in Japan, and is expected to be approved in Europe by the end of 2021.

Martina, a 29-year-old mother-of-two from Stuttgart, Germany, describes what it was like being diagnosed with this challenging autoimmune condition:

“It began rather unspectacularly, with flu-like symptoms, fever, headaches, and joint pain. So anyone would think, ‘It’s just a normal flu.’ Then it got worse over the course of a week and I went to the hospital.”

She was eventually diagnosed with ANCA-AAV after undergoing a series of blood tests. Unfortunately, she had to give up her career as an educator in the process, since she didn’t have a strong enough immune system to be interacting with children while taking steroids and immunosuppressants to control the disease. Patients like Martina are the ones that could potentially stand to benefit from new drug therapies like avacopan.

Martina, 29, appears in an ad to raise awareness for vasculitis conditions.

To find resources for those living with ANCA-AAV and their caretakers, visit: https://www.myancavasculitis.com/living-with-aav/

Berkeley Alumni Create Startup Focused on Autoimmune Disease Therapies

Geo Guillen, Marco Lobba, and Matthew Francis, the co-founders of autoimmune disease biotechnology company Catena Biosciences. Image courtesy of Berkeley News.

Marco Lobba was pursuing his PhD in Chemistry at UC Berkeley when he and his lab partners made a discovery. He had been studying the modification of proteins when he happened upon a technique called “oxidative coupling,” which modifies proteins so that they can be fused together. He and his partners also found that the enzyme tyrosinase could be used to make oxidative coupling much faster and more efficient. Tyrosinase is a naturally-occurring enzyme, found in fruits and vegetables, and is responsible for turning apples and avocadoes brown as they ripen.

The accelerated oxidative coupling method could be used to fuse proteins together, faster and more selectively, than any other method currently in use. This opens the door to treating autoimmune diseases, which attack the body by convincing a person’s antibodies to attack their own healthy cells. Using this discovery, scientists can attach ‘safe’ signals to healthy cells, helping the body’s immune system identify its own cells and refrain from attacking them.

“Think of it almost like Pavlov’s dogs,” explains Lobba. “Or tricking children into eating their vegetables by covering them in cheese,” he elaborated. “If you present the immune system with something it likes — at the same time as something it is attacking — it starts to associate that target as a good thing.”

Lobba presented his discovery during a course on entrepreneurship at UC Berkeley’s Haas School of Business. During the presentation, fellow classmate Geo Guillen saw how passionate he was about his research, and the value of his discovery in the treatment of autoimmune disease. It was this purpose that drove the pair to work together alongside Berkeley Chemistry professor, Matthew Francis, to co-found a startup called Catena Biosciences, focused on making autoimmune disease therapies.

Their startup launched remotely during the COVID-19 pandemic, at a time when biotechnology and pharmaceutical companies have come into focus for the role their organizations play in helping to keep our communities healthy and thriving. The startup has been valued at $10 million for its innovative technology and ground-breaking research.

Guillen commented on his company’s founding, saying: “We identified that the autoimmune market is one that is particularly ripe for disruption because a lot of the approaches to treating autoimmune disease focus on the symptoms, instead of the root cause. It’s a pretty large, untapped market.”

Catena Biosciences is aiming to conduct pre-clinical trials by the end of August 2021, which will test the impact of their therapeutics on autoimmune disease reactions in patients. Next month, the company will be looking to raise more funds for their startup to help them commercialize the treatment. The founders’ hope is that they can have a positive impact on those living with autoimmune diseases, like multiple sclerosis, lupus, and Type 1 diabetes.

The company has been awarded the 2021 Berkeley Big Ideas Award for their entrepreneurial endeavors. To learn more about Catena Biosciences, read about the company on the Berkeley News blog.

3 BioTech Companies You Can Invest In to Fund Autoimmune Disease Research

At Autoimmune Warrior, we believe that scientific research and development holds the key to unlocking new, innovative treatments and ultimately, a cure for autoimmune disease. In today’s blog post, we’ll explore three different biotechnology companies that are pioneering research about autoimmune diseases.

Why should companies research autoimmune diseases?

According to the American Autoimmune Diseases & Related Disorders Association (AARDA), there are over 100 different types of autoimmune diseases affecting 50 million people in the US alone. This demonstrates that autoimmune diseases are one of the most prevalent conditions nationwide. Furthermore, the AARDA reports that autoimmunity is one of the top 10 leading causes for death among American women. These figures show the high impact that medical research could have on autoimmune patients.

There is, of course, a financial incentive for biotech companies as well. A Research & Markets report indicated that as of 2017, the global autoimmune disease therapeutics market was estimated to be worth over US$109 billion. This figure was projected to grow to US$153 billion by 2025. Part of this growth has been attributed to the rise in autoimmune diseases among the general population and specific groups; although it’s been argued that medical professionals are becoming more aware of autoimmune conditions, and therefore, are simply getting better at diagnosing patients.

How can I help fund research & development?

If you or someone you love suffers from an autoimmune condition, you’ll know how important it is to find effective treatment options. As a result, you may consider investing your hard-earned dollars in companies that are pioneering autoimmune disease research. Below are three companies that I have personally researched that are contributing to this cause.

1. Landos Biopharma

Landos Biopharma is a Virginia-based company started by former Virginia Tech inflammation & immunology professor Josep Bassaganya-Riera, PhD. Landos is considered to be clinical-stage biopharmaceutical company focused on the discovery and development of oral therapeutics for patients with autoimmune diseases. In particular, the company is developing therapeutics for those with autoimmune diseases of the gastrointestinal tract, such as ulcerative colitis (UC) and Crohn’s disease.

Landos, which was founded in 2017, is a publicly-listed company on the NASDAQ stock exchange under the ticker symbol LABP. As of writing, the stock sits at just above US$12 per share.

2. UCB

UCB is a Brussels, Belgium-based multinational company, with a long history of research and development in the area of immunology. Some of the company’s autoimmune disease research areas include: rheumatoid arthritis, psoriasis, Crohn’s disease, lupus, and myasthenia gravis (MG). The company actively works with clinics worldwide to recruit for clinical studies with autoimmune patients; some of the studies they are actively recruiting for at the time of writing (June 2021) include patients with hidradenitis suppurativa, lupus, psoriasis and myasthenia gravis.

UCB is a 90-year-old company, and is publicly listed on the EBR stock exchange under the ticker symbol UCB. As of writing, the stock sits at just above 85 euros per share.

3. Abbvie

Abbvie is a Chicago-based multinational company that was spun off from Abbott Laboratories. Abbvie has been striving to advance the standard of care in rheumatology for more than 20 years. The company says that they are focused on developing therapeutics for patients with chronic diseases, which is said to account for 75 percent of all healthcare costs. Some of the company’s autoimmune research areas include: rheumatoid arthritis, systemic lupus erythematosus, psoriasis and multiple sclerosis. The company has already developed a number of well-known anti-inflammatory treatments, including HUMIRA (adalimumab).

Abbvie was spun-off in 2013, and is a publicly-listed company on the NYSE under the ticker symbol ABBV. As of writing, the stock sits at just above US$114 per share.

Would you consider investing in these biotech companies? Why or why not? Let us know in the comments below!

Disclaimer: This blog post is not intended to provide financial advice, but to raise awareness about companies conducting research & development towards advancing autoimmune disease therapeutics. Always consult with your physician before beginning a new treatment plan.