Rein Therapeutics 3rd Annual Guggenheim Healthcare Innovation Conference

Sure. At Rein, we're focused on pulmonary orphan conditions. We've got two programs that are phase II ready, both in respiratory diseases. Idiopathic pulmonary fibrosis is really our focus. The Rein story, I've been with this company now just over 13 years. We started as a private company called Lung Therapeutics out of Austin, Texas. We did a reverse merger in October 2023 with Aileron Therapeutics. They had been cancer-focused, obviously didn't make it. Really liked our program in IPF, and then we rebranded to Rein early 2025 to kind of highlight our focus on reining in fibrosis, which we love to say.

Yeah. It is a really amazing mechanism, and one that I think has been underappreciated, maybe because of the fact that it is missing in fibrosis. We are targeting, in a sense, caveolin-1. Caveolin-1, in addition to the structural role that it plays in cells, has a regulatory function, and it affects this regulation through a small 20 amino acid region called the caveolin scaffolding domain, and that domain interacts with a large number of proteins across multiple fibrosis pathways, and it really kind of promotes homeostasis in the cell. CAV1 is missing in a fibrotic state, not just in the lung. In any fibrosis, liver, skin, ocular, kidney, it is just missing. What we are doing is we are adding back a portion of that caveolin scaffolding domain region. We are basically supplying the regulation back to these cells that they normally would have.

It is impacting virtually every cell type in the fibrotic lung and adding back this regulatory capacity.

Absolutely. So we use an off-the-shelf dry powder inhaler, and we get great local lung coverage without kind of commensurate systemic uptake. The reason that we wanted to do an inhaled approach was when we had an ad board very, very early on related to this program, every KOL in the space said, "We have got inhaled programs for COPD, for asthma, for other lung diseases. Why can't we get one for IPF?" These patients have enough inspiratory capacity, and that has been our experience, is that they really do. We saw that in our phase I-B study. So again, we get really good kind of local lung coverage without the systemic uptake.

Sure. Our RENEW study is a global trial, 24 weeks of dosing in IPF patients. We will do a CT scan to confirm diagnosis before the study, and then they also get one after the trial. We are looking at HRCT, we are looking at forced vital capacity. We announced earlier this year that enrollment was going great. We hit 25% faster than we thought. So enrollment is good across all the countries in the study. Now we are enrolling in the U.S., the U.K., Australia, Germany, Poland, and we just opened Bulgaria and Romania as well. So other countries, and so far, I think the study is going great.

Sure. The criteria, really, they've just got to have a diagnosis of IPF, and we reconfirm that diagnosis when they get into the study, and enough lung capacity to be progressive in their disease. So just enough lung capacity so that they progress. We allow background standard of care, any standard of care. So nintedanib, pirfenidone, nerandomilast, anything that's approved, they can take, because we've seen benefit of LTI-03 basically on top of other compounds. So that's really kind of the key inclusion and probably the differentiator of this trial, is that we're allowing any kind of background standard of care.