3 Rare Autoimmune Disorders That Biotechs Are Striving to Treat

When one thinks of diseases, it’s natural to attribute them to bacteria, a virus, or perhaps poor lifestyle choices. In such cases, it is advised to strengthen the immune system, responsible for warding off germs and foreign substances.

But what if the immune system turns against the body? There is a group of disorders primarily caused by a hyper-responsive immune system. When the body’s defenses are acting against it, the results can be toxic.

As per MedlinePlus, an autoimmune disorder may lead to –

• Changes in the functioning of the affected organ
• Destruction of bodily tissue
• Abnormal organ growth

These conditions generally affect the joints, muscles, skin, endocrine glands, red blood cells, and connective tissues. The University of Oxford published a study from The Lancet, which found that one in ten people suffer from an autoimmune condition.

It is not uncommon to find an individual diagnosed with more than one autoimmune condition at a time. Some rare autoimmune diseases can take a toll on patients’ health, and severely so. In this article, we will discuss three such conditions that biotech companies are fighting to treat.

Graves’ Disease

Graves’ disease is one of the various autoimmune conditions associated with the thyroid gland. It attacks 2% to 3% of the population, which means 1 in 200 people. Studies have shown that women are more vulnerable to this condition than men.

Graves’ disease is marked by an unrestrained production of the thyroid hormone. According to the Mayo Clinic, the following are the common symptoms –

• Tremor in fingers or even hands
• Irritability and anxiety
• Increased heat sensitivity
• Changes in menstruation
• Reduced libido or erectile dysfunction
• Fatigue
• Irregular heartbeat or arrhythmia
• Sleep disturbances
• Unexplained weight loss (despite no changes in diet)

An even rarer form of this condition, Graves’ Ophthalmology or Thyroid Eye Disease (TED), affects the muscles around the eyes. Both conditions are debilitating, and the British-Belgian Biotech Apitope received approval for Epitopes in 2018 to treat Graves’ disease.

This treatment uses a protein-based approach to inactivate the white blood cells attacking the thyroid gland. Within 18 weeks of treatment, most of the 12 patients used in Phase I clinical trials showed complete recovery. 

As for the more complex TED, Horizon Therapeutics’ Tepezza or Teprotumumab was the first drug to receive Food and Drug Administration (FDA) approval in 2020. Later, complaints started emerging that Tepezza led to auditory-related issues. 

Victims filed a Tepezza lawsuit against the manufacturer, alleging that it engaged in misleading marketing. Tepezza’s clinical trials for FDA approval revealed that only 10% of patients showed any otologic symptoms.

On the contrary, a study published in the Endocrine Society journal found that partial or permanent hearing loss, tinnitus, etc. were present in 65% of patients. It was only in 2023 that the FDA added the warning of hearing loss on all Tepezza labels. 

According to TorHoerman Law, the lawsuit is consolidated into a multi-district litigation and is still awaiting settlements. As of now, Viridian Therapeutics is giving Horizon a run for its money with a novel treatment for chronic TED. 

Despite Phase I trials, it is expected that Viridian’s solution could significantly reduce proptosis when compared to its counterpart.

Sarcoidosis

Primarily affecting the lungs, sarcoidosis is a rare autoimmune condition with an estimated 27,000 new cases annually. This condition often begins in the form of tiny, grain-like lumps known as granulomas.

It is important to note that sarcoidosis may affect any organ (albeit the lungs are the primary target site). Though medical science believes this condition is autoimmune, the actual cause remains unknown. 

Some scientists believe that sarcoidosis occurs when the immune system reacts to dust, chemicals or self-proteins in a hyperactive manner. Even then, the individual must have some genetic predisposition for granulomas to form. The condition manifests itself through the following symptoms –

• Swollen lymph nodes
• Sudden weight loss
• Chest pain or wheezing
• Swelling or soreness in joints
• Increased fatigue
• Shortness of breath

Some patients may exhibit eye, heart, and skin symptoms, including –

• Eye pain
• Sensitivity to light
• Blurred vision
• Lesions on ears, cheeks, and nose
• Uneven skin tone
• Reddish-purple rash on the ankles or shin
• Irregular heartbeat
• Edema or excessive fluid
• Heart palpitations

The latest drug that has received the FDA’s fast-track designation in the treatment of pulmonary sarcoidosis is aTyr Pharma’s Efzofitimod. In its clinical trials, including 37 patients (receiving at least one dose), the drug was well-tolerated by all with positive outcomes in disease management. 

The drug was also found to be safe, with no reported adverse side effects. Phase 3 of the clinical trials is underway to further test the efficacy of this treatment. Besides this, AbbVie’s Humira or Adalimumab may reduce sarcoidosis symptoms in patients who did not respond to standard treatments. 

Psoriasis

Currently, over 8 million people across the United States have been diagnosed with psoriasis. Even this condition affects 2% to 3% of the total world population. It is an autoimmune condition attacking the skin, leading to the formation of thick scale-like patches.

Being an autoimmune skin inflammation that is often chronic, psoriasis plaques may flare up suddenly. Currently, medical science has not found a permanent cure, but rigorous efforts are ongoing. Most people have little knowledge about psoriasis, associating its greatest symptom with cosmetic discomfort.

However, patients may experience burn-like symptoms, some becoming life-threatening during flare-ups. This is because psoriasis interferes with the body’s ability to regulate internal temperature. As a result, patients become vulnerable to deadly infections.

In 2022, the FDA gave its approval to Dermavant’s VTAMA (Tapinarof) cream 1% for psoriasis treatment. First in its class, this topical applicator uses small molecule therapy to reduce skin inflammation and regulate the immune cells’ gene expression.  

As per the University of Manchester, a new drug has shown wondrous signs in psoriasis treatment, clearing symptoms in over 60% of patients. Known as Bimekizumab, the drug was administered to 373 patients during trials for 16 weeks. 

61% (230 patients) showed complete skin clearance at week 16. Even side effects associated with this treatment were rare. Besides these drugs, dermatologists are vouching for the traditional Mediterranean diet that may support faster recovery.

Closing Thoughts  

Studies have found that the risk of autoimmune inflammatory conditions has increased post-COVID-19. This spectrum of disorders is being given special attention as biotech companies tirelessly work to discover remedies.

As more cases emerge, the new treatments show promise in enhancing the patient’s quality of life and prognosis. From small molecule to antibody and cell therapies, diligent efforts in all areas are paving the way for revolutionary autoimmune disease treatments.