aTyr Pharma shares plunged more than 80% after its Phase III trial in pulmonary sarcoidosis failed to meet its primary endpoint.
In the EFZO-FIT study (NCT05415137), efzofitimod failed to show any change in mean daily oral corticosteroid (OCS) dose at week 48, with the OCS dose reducing by an average of 2.79mg for 5.0 mg/kg efzofitimod compared to 3.52 mg for placebo.
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Complete steroid withdrawal was achieved for 52.6% of patients treated with 5.0 mg/kg efzofitimod versus 40.2% on placebo.
On the King’s Sarcoidosis Questionnaire (KSQ)-Lung score, there was clinical improvement in the treatment group of 10.36 for efzofitimod compared to 6.19 for placebo. The proportion of patients who achieved complete steroid withdrawal with KSQ-Lung improvement was 29.5% in the efzofitimod cohort and 14.4% in the placebo cohort.
Despite the primary endpoint and KSQ not being statistically significant, aTyr said that due to ‘improvements across multiple clinically relevant efficacy endpoints’, it will still engage with the US Food and Drug Administration (FDA) about the data to determine the path forward for efzofitimod in pulmonary sarcoidosis.
Dr Sanjay S. Shukla, president and CEO of aTyr Pharma, said: “This study demonstrates that patients with chronic, symptomatic sarcoidosis can be managed with substantially lower steroid doses than previously thought. In spite of a higher than anticipated placebo response, we found that treatment with efzofitimod was associated with a greater amount of steroid reduction and an improvement in the KSQ-Lung score.”
After aTyr Pharma released the results, its stock dropped by 83.25%, from a 12 September market close of $6.03 to a 15 September market close of $1.01. The company has a market cap of $99.46m.
EFZO-FIT enrolled 268 patients with pulmonary sarcoidosis, evaluating the efficacy and safety of efzofitimod as a means to remove patients from steroid treatments over 48 weeks of treatment.
The Phase I/II trial (NCT03824392) of the therapy also only showed trends towards efficacy, with a change in corticosteroid burden of -48.9% (+/-19.7) and -58.1% (+/-23.4) in 3mg/kg and 5mg/kg doses, respectively.
There are very few drugs approved by the FDA for pulmonary sarcoidosis; prednisone and other glucocorticoids are on the US market for the disease. No therapies have been approved since 1952, showing a substantial unmet medical need for new treatments for the patient population. There are other drugs in development, including Molecure’s OATD-01, a chitotriosidase 1 (CHIT1) inhibitor, which is in a Phase II trial.
