Dive Brief:
- Gut microbiome specialist Ritter Pharmaceuticals has signed a deal with contract research organization Medpace to carry out the first of two pivotal Phase 3 trials for RP-G28, a candidate in development for the treatment of lactose intolerance.
- The planned trial will involve about 525 patients, and measure the compound's efficacy compared to placebo at reducing lactose intolerance symptoms in patients consuming lactose.
- RP-G28 acts by changing the balance of the bacteria in the gut away from gas-producing bacteria and towards lactose metabolizers. Ritter hopes one 30-day course will produce durable results and, if all goes to plan, the drug could become the first approved treatment for lactose intolerance.
Dive Insight:
Interest in microbiome research has surged, with biopharmas exploring a variety of therapeutic areas ranging from gastrointestinal disorders through cancer and metabolic disease.
Yet microbiome therapeutics are still mostly in early stages, mearning companies will have to construct clinical trials carefully in order to meet the Food and Drug Administration's requirements. This could open the door for CROs like Medpace, which says it has expertise in both the gastrointestinal and microbiome fields.
"With our CRO partner secured, we remain on track to initiate our RP-G28 Phase 3 trial this quarter," said Andrew Ritter, president of Ritter Pharmaceuticals.
True lactose intolerance is more than a simple food fad. Even small amounts of milk in lactose-intolerant people can trigger diarrhea, nausea, stomach cramps, bloating and gas.
In its Phase 2 trial, 40% of patients treated with RP-G28 responded compared with 31% on placebo — missing statistical signifiance among all patients who received at least one dose of drug.
But Ritter said results from one study center showed "significant irregularities." Excluding the patients treated at that center, the study met statistical significance with 40% of treated patients responding versus 26% given placebo.
These issues were discussed with the FDA during an End of Phase 2 meeting, with the regulator's guidance incorporated into the Phase 3 design. Further results from the Phase 2 trial showed a beneficial shift in the microbiome, and highlighted the potential of the agent in other indications, Ritter said.