aTyr Pharma’s Efzofitimod Trial Failure: Pulmonary Sarcoidosis Drug Faces Setback and Market Crash

aTyr Pharma’s Efzofitimod trial failed, dealing a major blow to the development of a drug for lung disease, and the company’s future progress depended on it.
The world of pharmaceuticals and biotech has always been an area of ​​great hope and great risk. If a successful drug is developed, it can give a new life to millions of patients, and on the contrary, if the same trial fails, the company and investors can lose billions of dollars. An example is the case of aTyr Pharma, a biotech company. The company’s ambitious drug for a serious lung disease, Efzofitimod, has failed in the third phase of the third trial. The news caused a stir in the market and the company’s shares have fallen by as much as 80%.

What is pulmonary sarcoidosis?

Sarcoidosis is an inflammatory disease. In this, small lumps of inflammatory cells form in various organs, especially the lungs and lymph nodes, due to an overreaction of the body’s immune system. These lumps are called granulomas. When these lumps form in the lungs, it is called pulmonary sarcoidosis.

What are the symptoms of this disease?

Persistent dry cough

Difficulty breathing and shortness of breath

Chest pain and discomfort

Extreme fatigue and weakness

If this disease is not controlled in time, this disease can become serious. Permanent scarring (fibrosis) can form on the lungs and permanently reduce the function of the lungs, which greatly reduces the patient’s quality of life. 
What are the current treatments for steroids for control?   
Currently, there is no definitive cure for pulmonary sarcoidosis. The main goal of treatment is to reduce inflammation and control symptoms. Corticosteroids, such as prednisone, are mainly used for this. Although steroids are very effective in reducing inflammation, they can be dangerous when taken long-term. They can have serious side effects, such as weight gain, high blood pressure, diabetes, osteoporosis, and an increased risk of infection.

What was the ‘steroid-sparing’ role of efzofitimod
Efzofitimod is an immunomodulator drug. It works on the body’s immune system in a way that reduces inflammation. Etir Pharma was developing the drug as a ‘steroid-sparing agent’. This simply meant that the drug would help patients significantly reduce their steroid doses or stop them altogether, thereby relieving them of the serious side effects of steroids.

And for this purpose, the company conducted a large phase III clinical trial (ORCA study). The primary endpoint of this trial was to prove whether the daily steroid dose of patients taking efzofitimod was significantly reduced after 48 weeks of treatment compared to patients taking placebo. A placebo is an inactive substance that looks like the real drug but has no medicinal properties. It can be used to measure the effect of the real drug more accurately.

aTyr Pharma Efzofitimod trial failure and what was the explanation given about it  

The results of this trial by Atir Pharma were shocking to everyone. The drug efzofitimod did not perform better than placebo and the trial completely failed to meet its primary endpoint.
The company’s CEO, Dr. Sanjay Shukla, explained the reason for this failure. According to him, the main reason for the failure was that the patients in the placebo group showed much greater improvement than expected. “Our drug performed as expected. However, patients in the placebo group also reduced their steroid dose so much that the difference between the two groups was no longer statistically significant,” he said.