TLY012 Receives FDA Orphan Drug Status for Systemic Scleroderma

TLY012 Receives FDA Orphan Drug Status for Systemic Scleroderma
4.4
(14)

The U.S. Food and Drug Administration has granted orphan drug designation to Theraly Fibrosis’ TLY012 for the treatment of systemic scleroderma.

This designation is given to medications with the potential to treat rare conditions — those that affect fewer than 200,000 people in the U.S. Benefits include exemption from fees, financial incentives for clinical development, and seven years of market exclusivity if the treatment is approved.

“We look forward to initiating our clinical trial in systemic scleroderma potentially within the next year and advancing this important drug candidate for patients with significant unmet need,” Viktor Roschke, PhD, chief scientific officer and head of research and development at Theraly, said in a press release.

TLY012 is a version of the human protein TRAIL that has been modified to be more stable. TRAIL (tumor necrosis factor-related apoptosis-inducing ligand) is an inflammatory protein that induces cell death in certain kinds of cells, especially cancer cells.

In systemic scleroderma, also referred to as systemic sclerosis, cells called myofibroblasts drive excess scarring in the skin. Myofibroblasts also contribute to organ scarring in other fibrotic diseases, such as liver fibrosis. TLY012 is designed to bind to proteins called death receptor 5 (or TRAIL receptor 2) on myofibroblasts and shut down fibrotic pathways.

An early study showed that TLY012 killed skin myofibroblasts from people with scleroderma, while it did not have the same effect in cells derived from people without the disease. Together with the higher levels of TRAIL and associated proteins seen in diseased cells, the findings suggested that TLY012 can be effective while sparing healthy cells.

In the same study, TLY012 reduced skin thickness and scarring in two mouse models of scleroderma, without any apparent detrimental effects on non-scarred parts of the skin.

“Targeting the TRAIL pathway with TLY012 may represent a promising anti-fibrotic strategy to arrest scleroderma in humans,” Roschke said. “Because TLY012 targets and blocks the formation of [myofibroblasts] regardless of tissue location, it has the potential to not only be a first-in-class treatment for systemic scleroderma, but also for other fibrotic diseases.”

In fact, according to Theraly, TLY012 has shown promising effects in preclinical models of liver and pancreas scarring. It has also been granted orphan drug designation for the treatment of chronic pancreatitis — a condition characterized by scarring and inflammation in the pancreas — in 2019.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
Total Posts: 27
José holds a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
×
Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
Latest Posts
  • sleep apnea
  • TLY012 orphan drug status
  • COVID-19 and scleroderma
  • heart

How useful was this post?

Click on a star to rate it!

Average rating 4.4 / 5. Vote count: 14

No votes so far! Be the first to rate this post.

As you found this post useful...

Follow us on social media!

We are sorry that this post was not useful for you!

Let us improve this post!

Tell us how we can improve this post?