A New Era of Hope for Idiopathic Pulmonary Fibrosis

By: Anjali Pandey, Ph.D.

SVP of Nonclincal R&D & Chemistry at Aria Pharmaceuticals

Idiopathic pulmonary fibrosis (IPF) is devastating. It causes lung tissue scarring that becomes thicker over time, causing chronic pain and difficulty breathing. However, it’s not just the physical toll of the disease that impacts patients, it’s also a significant decline in quality of life. As I read with one patient’s account, “You’re not free, can’t laugh or cry fully because if you do, you won’t be able to breathe.”

Today marks the last day of Pulmonary Fibrosis Awareness Month, a month designed by the Pulmonary Fibrosis Foundation (PFF) to raise awareness of this complex disease and help to mobilize the community to educate the public about its symptoms and treatments, in effort to help increase diagnosis and gather support for those living with IPF.

What’s challenging about IPF is that its cause is idiopathic, or unknown, and can arise spontaneously. While it’s still considered a rare disease, according to the American Lung Association, IPF is more common than we once thought, with up to 132,000 people affected in the United States and around 50,000 new cases diagnosed each year. It is progressive and irreversible, with no cure, and ultimately a terminal condition. The mean survival for an individual diagnosed with IPF is only 3–5 years. However, given its unpredictable nature, it is impossible to know how slow or fast IPF will progress with each person.

While there is no cure for IPF there are treatments to help slow the progression of the disease. Receiving a proper diagnosis and starting treatment is critical to helping increase the quality and longevity of life for people with IPF, but treatment is limited.

Right now, there are only two medications, pirfenidone and nintedanib, approved by the Food and Drug Administration (FDA) for IPF. Both agents have been shown to slow the progression of IPF through antifibrotic effects, which can help preserve lung function. Before pirfenidone and nintedanib, failures and negative clinical trial results were common in IPF research.

While their approvals are welcome and needed, I believe these treatments are just the beginning in a new era of hope for patients suffering from IPF. It remains, without question, that the development of novel therapeutic approaches in treating IPF is still the largest unmet need for those living with the disease, in part because both pirfenidone and nintedanib carry significant side effects, limiting their broad use, and their effectiveness is only modest in treating mild to moderate disease. But it’s also urgent because the response to pirfenidone and nintedanib is heterogeneous, varying from patient to patient. That is partially because of the unknown cause of the disease and its varying progression across different patients.

Several companies are taking aim at IPF with novel targeted approaches, including Aria. And we must take all shots on goal for this complex disease. In fact, we are taking two. Earlier this year, we announced that two of our IPF candidates, TXR-1002 and TXR-1007, decrease lung fibrosis as measured by a significant reduction in lung collagen staining, with mean levels slightly lower than that observed with nintedanib along with a decrease in inflammation. Both candidates use novel mechanisms of action. These early results show us that we are on the right track to create a new drug with a differentiated mechanism of action that we expect will be more effective across heterogenous patient population and better tolerated than the current standard of care.

IPF Awareness Month is only a point in time. To patients it’s a daily fight. The same rings true for us, and many other researchers who are working daily to find and develop new, promising treatments. I remain excited and committed to this therapeutic area and Aria remains committed to advancing these potential treatments into clinical research, with the hope that one day patients will benefit from our work. I remain equally excited by the wave of new research into IPF, as several companies test dozens of novel therapies. I truly believe we are entering a new era of hope for patients living with IPF. While there is much still to learn, and today’s prognosis can be devastating, within a few years that may change completely.

Aria Pharmaceuticals is a preclinical-stage pharmaceutical company discovering and developing novel small molecule therapies for complex diseases.