PTC Therapeutics, Inc. (NASDAQ:PTCT) recently announced that it started dosing patients after the first patient received a dose in its phase one clinical trial evaluating PTC857.
The announcement represents a landmark moment for PTC Therapeutics in the development of PTC857. The latter is a developmental small molecule therapy that PCT discovered through its Bio-e platform. The therapy’s mechanism is designed to block an enzyme called 15-Lipoxygenase, which plays a key role in regulating inflammation response pathways, protein aggregation, and oxidative stress regulation. Lack of proper regulation of the three aspects is linked to neurodegenerative diseases.
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“We are excited about the initiation of this study for PTC857, which was developed from our novel Bio-e platform,” stated Dr. Stuart W. Peltz, the CEO of PTC Therapeutics.
The CEO further pointed out that PTC is designed to inhibit the 15-Lipoxygenase enzyme production that modulates the pathways that cause conditions that are precursors to multiple disorders. One of the most notable indications that PTC Therapeutics targets with PTC857 is Parkinson’s disease. The pipeline treatment represents significant hope in combatting such illnesses and providing much-needed relief to patients that suffer from neurodegenerative diseases.
The company is optimistic about the current phase 1 clinical trial for PTC857 and it hopes to advance the therapy to patients. The ongoing study aims to determine how healthy volunteers will respond to PTC857 dosage. The information collected from this phase 1 trial will help determine whether the treatment is ready to advance to patient trials.
The phase 1 study also aims to determine the developmental therapy’s pharmacology and safety profile. Thus, the company will administer multiple and single ascending doses that will allow them to collect the necessary data. The findings will also be vital for the development of phase 2 clinical trials. PTC857 already demonstrated significant safety and efficacy in various in vitro and in vivo evaluation programs relevant to numerous GBA Parkinson’s disease.
PTC Therapeutics is excited about PTC857 because it aligns with its main objective of developing therapies that target rare disorders. If the drug proves to be an effective solution against degenerative disorders, it will add to its robust product line, thus boosting its portfolio and revenue.