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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.

How the Drug Delivery System (DDS) works

  • ORAL ADMINISTRATION: Convenient oral capsule the size of a fish-oil pill
  • AUTONOMOUS LOCATION: Proprietary autolocation in the GI tract for accurate drug delivery, regardless of fasted or fed state2
  • TARGETED DRUG DELIVERY: Method designed to coat the length of the colon with liquid formulation, minimizing systemic uptake
 
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Therapeutic challenges for UC

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

Biora’s potential solution

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

Targeted Therapeutics Pipeline

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
Device

 —
Clinical

PGN-600
Tofacitinib + Device

 Ulcerative Colitis
Preclinical

PGN-001
Adalimumab variant + Device

 Ulcerative Colitis
Preclinical
  • 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.  

Targeted Therapeutics Publications

A scintigraphic study to evaluate the localization and delivery function of a Drug Delivery System (DDS) device in patients with active ulcerative colitis in fasted state
#Targeted Therapeutics
A scintigraphic study to evaluate the safety, tolerability, and functionality of a Drug Delivery System (DDS) device in healthy volunteers in fasted state
#Targeted Therapeutics
Pilot study to assess pharmacokinetic and pharmacodynamic markers following enema-dosing with adalimumab in patients with active ulcerative colitis
#Targeted Therapeutics
See More

References

  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. Biora Therapeutics internal data.
  3. 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. 
  4. 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.