IM Therapeutics said its lead, and only, clinical-stage candidate exhibited positive safety, tolerability, pharmacokinetics and mechanism of action in a phase 1b clinical trial.
The oral drug, IMT-002, was tested in patients with Type 1 diabetes and is meant to block the HLA-DQ8 genetic trait, an isoform of genes that increase the risk for the disease and is seen in a majority of patients, IM said Wednesday.
Human leukocyte antigens (HLAs) are the "earliest triggers of autoimmune disease," said Nandan Padukone, Ph.D., CEO of IM, in an interview with Fierce Biotech. HLAs recognize natural proteins like insulin, myelin, collagen and others in autoimmune diseases as foreign and then attacks them.
IM is "knocking out that very first step" in autoimmune diseases by attempting to block off the HLAs from behaving errantly, Padukone said. "We are taking small-molecule drugs and we are attacking specifically those variants that are involved in unfortunately recognizing those natural proteins," the CEO said.
This would be a novel step in treating Type 1 diabetes, said Padukone and Peter Gottlieb, M.D., co-founder and chief medical officer of IM, in a joint interview.
"Up until now, we’ve really been trying to affect the autoimmunity of Type 1 [diabetes] primarily with immunosuppressants and a lot of those are antibody therapies, biologics, so they require shots, they require infusions over many days," Gottlieb said. "What we think is an advantage, particularly for Type 1, where 50% of the people affected are kids, is this is an oral medicine that they would take either once or twice a day that looks to be really safe, and so I think we have some relative advantages."
The drug was "really pretty well tolerated" in the phase 1b study, which launched in October, Gottlieb said. The multiple ascending dose trial included 30 T1D patients preselected for HLA-DQ8 between ages 18 and 45 and the drug was compared to placebo over two weeks of dosing and a one-week follow-up post-dose completion.
The two executives said IM would like to start a phase 2 study next year. "Design-wise, we’re hoping that with meeting with the regulatory agencies, getting their blessing, sometime late Q1 next year, we should be ready for a phase 2 launch," Padukone said.
That study will look at the efficacy of the drug, which would be the "maintenance of an endogenous insulin production," Gottlieb said. That is an endpoint accepted by the FDA and European Medicines Agency as an endpoint, the CMO said, noting the study will take places across sites in the U.S. and potentially in Europe and other regions.
"We’re going to try to work to hopefully get the trial to run between six and 18 years of age. We may have to do some adults initially to address safety issues for the FDA," Gottlieb said.
RELATED: Provention Bio slapped with FDA rejection for diabetes hopeful teplizumab
The diabetes space has been a mixed bag this month.
Two weeks ago, the FDA rang the alarm bells and flashed red lights for Provention Bio's T1D drug hopeful, teplizumab. The regulatory body gave a complete response letter on the drug after Provention had said the treatment delayed the onset of T1D by a median of two years.
Meanwhile, last week, Eli Lilly paid $1 billion to acquire the remainder of smart insulin tech player Protomer, and Kriya Therapeutics hauled in a $100 million series B to help fund an adeno-associated virus gene therapy for T1D. The injectable drug, KT-A112, aims to deliver genes to produce insulin and glucokinase.
RELATED: Kriya grabs $100M to launch gene therapies into the mainstream, starting with diabetes
For its part, IM is looking to raise additional capital, as well. The biotech has raised $24 million to date, Padukone said, including a series A in October 2019 co-led by the JDRF T1D Fund and Morningside Ventures. The Colorado University Healthcare Innovation Fund is also a backer of IM, which was founded out of the university's Barbara Davis Center for Diabetes.
Existing funds will get IM through the end of this year or early Q1 of 2022, Padukone said, noting the company is working on pulling together another round of financing to extend the runway about 24 to 30 months. Fresh proceeds would go toward the phase 2 study, funding a phase 1 clinical trial of its second program, focused on celiac disease, as well as boost its pipeline of other autoimmune disease programs, the CEO said.
IM expects to have candidate selection for its celiac indication in the second half of this year, Padukone said.
Errant HLA proteins is an issue rampant across autoimmune diseases, the CEO said, which means IM's science can be applied across a plethora of indications.
"We want to expand this to celiac; we want to expand this to other autoimmune diseases where these same HLA proteins have a big role to play," Padukone said.