Biora Therapeutics (BIOR) Study Update summary

Unmet needs in ulcerative colitis

• Ulcerative colitis affects millions globally, including about 1.5 million in the US, with significant psychological and quality-of-life impacts.

• Current therapies have limited efficacy, with clinical remission rates often below 30% and notable safety concerns.

• Achieving sufficient drug activity at the disease site is challenging, and systemic toxicity limits dosing and combination therapy options.

• There is a need for targeted, topical therapies to improve efficacy and reduce systemic toxicity.

• Topical delivery to the colon could improve efficacy and reduce adverse events by increasing local drug exposure and minimizing systemic uptake.

NaviCap platform and technology overview

• NaviCap is an oral, electromechanical capsule designed for targeted, needle-free drug delivery to the colon, using GITrac autolocation technology for precise release.

• The platform enables delivery of up to 500μl of drug, functioning effectively in both fasted and fed states.

• NaviCap aims to coat the colon with a liquid drug formulation, minimizing systemic absorption and enabling consistent delivery regardless of GI motility or food intake.

• Targeted delivery aims to improve efficacy and safety for inflammatory bowel disease by increasing local drug activity and reducing systemic exposure.

• Biora is also developing the BioJet platform for systemic, needle-free oral delivery of large molecules.

BT-600 phase I clinical trial design

• Phase I was a randomized, double-blind, placebo-controlled trial in 48 healthy participants, evaluating safety, tolerability, pharmacokinetics (PK), and device function for single and multiple ascending doses of BT-600 (tofacitinib in NaviCap).

• Endpoints included safety, PK/PD, and confirmation of targeted colonic delivery.

• Flexible sigmoidoscopy and tissue biopsies were performed to assess colonic drug concentrations.

• The trial included 5 mg or 10 mg tofacitinib or placebo cohorts.

• Biopsy and device analysis confirmed pan-colonic drug exposure and accurate device function, with 100% (SAD) and 98% (MAD) of devices detecting colon entry.