Our Targeted Therapeutics program uses an ingestible smart capsule designed for targeted delivery of therapeutics in the gastrointestinal tract to improve the treatment of IBD, with an initial focus on ulcerative colitis.
Delivering therapeutics directly to the site of disease could enable safer and more effective drug therapies for patients.
Inflammatory bowel disease (IBD) includes Crohn’s disease and ulcerative colitis (UC).
About 1 million people in the U.S. are affected with UC, and ~40,000 new cases are diagnosed each year1.
UC causes inflammation and damage to the large intestine.
Difficulty of achieving sufficient drug levels at site of disease
Systemic toxicity issues may limit daily dosage of UC drugs
Combination therapy is limited by toxicity
Targeted delivery is designed to increase drug levels at the site of disease, which is correlated with improved outcomes3
Reduced systemic uptake is designed to reduce toxicity and adverse events
Reduced toxicity could enable combination therapy4
Reduced developmental risk by using approved drugs
More drug to the site of disease shows promising efficacy profile
Less drug delivered systemically may lead to improved safety profile
Unique combinations designed to yield promising efficacy profile
We are developing a pipeline of investigational drug/device combinations designed to overcome the limitations of current treatments for ulcerative colitis.
| Program | Indication | Design / Feasibility | Preclinical | Clinical |
|---|---|---|---|---|
DDS |
— | |||
PGN-600 |
Ulcerative Colitis | |||
PGN-001 |
Ulcerative Colitis | |||
We are developing PGN-600 as an orally delivered liquid formulation of tofacitinib for the treatment of ulcerative colitis. Tofacitinib is approved for ulcerative colitis and is dose limited based on safety concerns, making it an ideal therapy for targeted delivery.
Our preclinical data demonstrates that targeted delivery using PGN-600 can lead to reduced drug levels in blood and to increased levels in tissue at least 25 times higher along the length of the colon versus the equivalent standard oral dose.
If we demonstrate similar results clinically to what has been observed preclinically, and given the known efficacy of the currently approved doses of tofacitinib, we believe PGN-600 has the potential to greatly improve patient outcomes in ulcerative colitis.
We are developing PGN-001 as an orally-delivered variant of adalimumab for the treatment of ulcerative colitis (UC). Multiple anti-TNF-alpha targeting therapies have been approved for UC, but data suggests patients may not have enough drug in the tissue to engage the target, TNF-alpha, and reduce inflammation. We have developed our own anti-TNF-alpha antibody formulation for use in further development.
PGN-001 is currently in preclinical stage development. We have conducted a series of preclinical studies demonstrating the potential of locally delivered anti-TNF-alpha antibodies to reduce disease burden in ulcerative colitis models.
Connect with us