Recently, biopharmaceutical company Acceleron Pharma Inc. (“Acceleron”) announced that its investigational therapy ACE-1334 was granted Orphan Drug designation by the FDA. The treatment is designed for patients with systemic sclerosis. However, researchers will soon evaluate the drug for systemic sclerosis-associated interstitial lung disease (SSc-ILD). If their plans go smoothly, Acceleron’s Phase 1b/Phase 2 clinical trial will take place in 2021.
Discovered by Acceleron, ACE-1334 is described as a:
TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands.
However, the therapy does not bind or inhibit TGF-beta 2. This is because researchers believe both 1 and 3 play a role in the development of fibrotic conditions. In preclinical studies, ACE-1334 reduced fibrotic activity. Additionally, Acceleron tested the safety, tolerability, pharmacokinetics, and pharmacodynamics of ACE-1334 in a Phase 1 ascending dose study. Beyond Orphan Drug status, ACE-1334 also received Fast Track designation.
Orphan Drug designation is provided to companies looking to create safe, effective treatments for patients with rare diseases. A rare disease is defined as any condition affecting under 200,000 Americans. For companies whose therapies receive Orphan Drug status, they also receive benefits such as 7 years of market exclusivity, clinical trial funding, and tax benefits.
SSc-ILD is interstitial lung disease (pulmonary fibrosis) caused by or related to systemic sclerosis, or systemic scleroderma. Overall, systemic scleroderma is an autoimmune disorder. The immune system mistakenly attacks healthy tissue, causing scar tissue on the skin and organs. An estimated 40,000-165,000 American citizens have systemic scleroderma. Females are more likely to have this condition than males. Symptoms include:
- Raynaud’s phenomenon
- Hand and finger inflammation
- Hardening of the skin
- Open sores on fingers
- High blood pressure
- Joint pain
- Shortness of breath / difficulty breathing
If fibrosis affects the internal organs, it can lead to damage, poor function, and even organ failure. Learn more about systemic scleroderma here.
The Cleveland Clinic describes interstitial lung disease (ILD) as:
scarring and inflammation of the interstitium (the tissue that surrounds the lung’s air sacs, blood vessels and airways). ILD may be limited to the lung, or it can be related to a condition that may affect other parts of the body.
So SSc-ILD refers to lung fibrosis caused by systemic scleroderma. Symptoms, in addition to those listed above, include:
- A nonproductive cough
- Exercise intolerance
- Unintended weight loss
SSc-ILD is one of the most common causes of death among those with systemic scleroderma. An estimated 50,000 Americans and Europeans have SSc-ILD. The current survival rate within 10 years of diagnosis is 56%.