Targeted Therapeutics

Targeted drug delivery to the GI tract designed to improve patient outcomes

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.

About ulcerative colitis

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.

The Drug Delivery System capsule

  • The DDS capsule is approximately the size of a fish oil capsule, and delivers a payload of up to 500µl liquid or solid formulation.
  • Once swallowed, the capsule is designed to autonomously identify specific locations in the GI tract and release a therapeutic dose.
  • Targeted delivery method is designed to minimize systemic uptake, potentially reducing adverse effects.

The clinical dilemma

  • UC drugs have systemic toxicity issues that may limit daily dosage
  • Achieving sufficient drug levels at the site of disease is difficult with systemic delivery
  • Only 1 in 4 UC patients achieves short-term response to drug therapy2
  • UC has multiple pathways,3 but current protocols target single pathways due to toxicity concerns

Our targeted approach

  • Reduced systemic uptake should reduce toxicity and adverse events 
  • Increased drug levels in tissue are correlated with improved endoscopic outcomes4
  • Targeted delivery could enable rapid induction, which should improve patient response
  • Targeted delivery could enable combination therapy to target multiple inflammatory pathways simultaneously3 

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

Targeted drug delivery to the GI tract

We are developing a pipeline of investigational drug/device combinations that are designed to overcome the limitation of current treatments for ulcerative colitis. 

Our products are designed to treat disease at its site in the GI tract and achieve high concentration in the affected tissues with the potential to increase efficacy and minimize systemic exposure and toxicity.

Program Indication Design / Feasibility Preclinical Clinical



Tofacitinib + Device

Ulcerative Colitis

Adalimumab + Device

Ulcerative Colitis
  • PGN-600
  • PGN-001

PGN-600: Liquid formulation of tofacitinib delivered with the DDS for the treatment of 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.

PGN-001: Liquid formulation of Anti-TNF-alpha monoclonal antibody delivered with the DDS for the treatment of 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.  


  1. Shivashankar R, Tremaine WJ, Harmsen WS, Loftus EV Jr. Incidence and Prevalence of Crohn’s Disease and Ulcerative Colitis in Olmsted County, Minnesota From 1970 Through 2010. Clin Gastroenterol Hepatol. 2017;15(6):857-863.
  2. Alsoud D, Verstockt B, Fiocchi C, Vermeire S. Breaking the therapeutic ceiling in drug development in ulcerative colitis. Lancet Gastroenterol Hepatol. 2021;6(7):589-595. doi:10.1016/S2468-1253(21)00065-0
  3. Van Oostrom J, Hanzel J, Verstockt B, et al. Pharmacokinetic stratification of cytokine profiles during anti-TNF induction treatment in moderate-to-severe ulcerative colitis. Poster presented at the 17th Congress of the European Crohn’s and Colitis Organisation (ECCO); February 18, 2022. 
  4. Verstockt B, Alsoud D, van Oostrom J, et al. Tofacitinib tissue exposure correlates with endoscopic outcome. Oral presentation at the 34th edition of the Belgian Week of Gastroenterology, February 9, 2022.