Cumberland Pharmaceuticals Inc Status update

Good afternoon, everyone. My name is A.J. Kazimi, and I am Chief Executive Officer at Cumberland Pharmaceuticals. I am also joined by Dr. Ines Macias-Perez, Vice President, Product Development, who manages our clinical and regulatory activities. Today, we are issuing a press release providing an update on our product development activities, which can be found on our corporate website. We would now like to share that update with you through this presentation. We appreciate this opportunity to introduce you to the new strategy for our company. I would like to start with a question investors may ask, why Cumberland, why now? First, we have significant value creation opportunities. Our lead program in Duchenne muscular dystrophy is advancing towards a New Drug Application, and behind it are multiple phase II programs built on the same core molecule.

Second, we are targeting attractive markets in which the addressable patient populations have limited or no effective treatment options. In Duchenne cardiomyopathy, for example, there is no approved therapy today. Third, we have compelling phase II data in Duchenne, a dose-dependent improvement in heart function with a 5.4 percentage point difference in ejection fraction versus control corroborated by biomarkers. Fourth, we now have a defined path to an NDA. Today, I am pleased to announce that we held a pre-NDA meeting with the FDA this fall. Given the urgent medical need, we have decided to proceed with the submission of an NDA for this program. After the pre-NDA meeting with FDA, they granted our request for a rolling submission of the NDA. Next, we have an established safety profile with roughly 1,400 subjects dosed across 30 clinical studies.

Sixth, Cumberland possesses the worldwide commercial rights for all its candidates in clinical development. Seventh, we feature a strong balance sheet that is able to fund our clinical studies. Finally, we expect meaningful near-term news flow led by our rolling NDA submission and long-term extension data for Duchenne. Over the rest of today's presentation, we will walk you through each of these points in more detail. Before we go further, I would like to briefly note that today's presentation does include forward-looking statements, including statements about our regulatory plans and our financial projections. For further information, please see our SEC filings, including our annual report and quarterly updates, which outline our risk factors, as well as our corporate website. Let me give you some context on how we have gotten here. You may know Cumberland as a specialty pharmaceutical firm with a portfolio of FDA-approved brands.

Today, we are a very different company. This summer, we closed on a strategic transaction, which integrated our commercial products and organization with the U.S. branded business of Apotex Health, the largest pharmaceutical company based in Canada. Apotex went on to conclude a successful initial public offering and listing on the Toronto Stock Exchange and featured our transaction as a milestone in building their U.S. branded business. For Cumberland, we unlocked significant value receiving over $100 million associated with the transaction, eliminating 75% of our expenses, and dramatically improving our share price. Meanwhile, we retained our pipeline of new medicines for the future, which we believe is the most valuable part of our business. Looking ahead, we will sharpen our focus on advancing new therapies for patients with significant unmet medical needs. This is a defining moment for Cumberland and an an important step in our evolution.

With a strategy focused on late-stage clinical development and a promising pipeline, we're well-positioned to build on our experience in developing, registering, and delivering differentiated medicines. Today, our development efforts are led by ifetroban, a novel oral therapy we're evaluating across several indications. Our clinical programs are designed to address serious conditions in which patients continue to face significant unmet needs. Now, let's look a little more closely at the asset behind this opportunity. This slide summarizes what makes ifetroban a de-risked molecule, and we'll expand on each point as we proceed through today's presentation. First, and most importantly, we have positive phase II data in Duchenne muscular dystrophy, where ifetroban improved heart function over 12 months against a disease course in which heart function is expected to decline.

Heart failure is the leading cause of death in these patients, so this is a meaningful place to show such a benefit. Second, we have an unusually well-established safety profile for a compound at this stage. Third, we hold three key FDA designations for the Duchenne program: orphan drug, Rare Pediatric Disease, and Fast Track, each providing important benefits for our efforts. Fourth, it's a platform, not a single shot on goal. One molecule studied in multiple indications with additional expansion work underway. Fifth, our team has a proven record of taking products through the FDA into the market. Sixth, we're well-capitalized following our recent transaction, and we carry no debt. Here's the company in numbers.

Over $100 million in net proceeds from the divestiture of our commercial assets, three core clinical programs, all in phase II, and roughly 30 clinical studies of ifetroban completed to date with 1,400 subjects dosed. On the left, our record. Cumberland has a long history of successfully developing branded products and gaining FDA approval for them. That experience is directly relevant here because the hardest part of a program like ours involves not just the science, but also the regulatory pathway and expertise needed to navigate it. We believe the sale of our commercial portfolio was in many ways a validation of that track record and allows us to now be singularly focused on advancing ifetroban. On the right, the asset itself, our first new chemical entity originally discovered and developed by Bristol Myers Squibb.

We manufactured both intravenous and oral formulations, and we've advanced development programs in collaboration with the guidance of research teams and clinical teams at Vanderbilt University and their medical center. With that overview, I'd now like to pass the presentation on to Ines to walk you through ifetroban and its clinical profile.

Ines? Thank you, A.J. Let me spend a moment on the molecule itself because everything else follows from it.

Ifetroban is a once-daily oral therapy taken as capsules in a fasted state. It works by blocking a single receptor called the thromboxane receptor. What makes that receptor interesting is where it lives on. It is on heart muscles, it is on immune cells, and it is on smooth muscle and lines blood vessels. Blocking this one receptor interrupts three injury pathways at once. It can dampen the inflammatory signaling implicated in tissue injury. It targets fibrosis, which is simply scarring, and scarring contributes to progression in all three of our diseases. It relaxes blood vessels, which is intended to improve blood supply to the affected tissue. The molecule is also well-characterized, with approximately 1,400 subjects dosed across roughly 30 clinical studies and about 90 patient years of exposure in Duchenne patients.

The key point is one target, many tissues. A shared biological pathway is what makes it rational to develop one molecule across three very different diseases. Here is the pipeline on one page. One molecule, three core programs, and an expansion opportunity. Today, we will focus on Dyscorban for the cardiomyopathy associated with Duchenne muscular dystrophy, which is our lead program. We have completed the double-blind portion of phase II, held our pre-NDA meeting with the FDA, and now plan to submit a New Drug Application. Our phase IIa study in systemic sclerosis is complete, with top-line results now reported, and our phase II study in pulmonary fibrosis is enrolling. In oncology, a phase IIa study in metastasis prevention sponsored by Vanderbilt University Medical Center has also been completed. Here is the shape of the news flow ahead of us.

Again, focusing on Duchenne muscular dystrophy, we plan to initiate our rolling NDA submission, followed by open label extension data at 24 and 36 months, which is going to be included in the NDA, presentation of those data at a scientific conference, and completion of the NDA submission. In systemic sclerosis, following our top-line results, we plan to submit the clinical study report to the FDA and complete correlative biomarker and cardiac imaging work that will inform our decision on further development. Let us turn now to our lead program, ifetroban in Duchenne muscular dystrophy, where we are addressing cardiomyopathy. First, some background on the disease. Because the natural history is central to understanding our data, Duchenne muscular dystrophy is a rare, fatal genetic disorder that causes progressive deterioration of skeletal, heart, and respiratory muscle. It affects boys almost exclusively.

Over the last two decades, respiratory care has improved and patients are living longer. The consequence is that the heart has become the limiting factor. Heart failure is now a leading cause of death in Duchenne. Current treatments like ACE inhibitors, ARBs, ARNIs, beta blockers, aldosterone inhibitors, they all slow the decline in heart function, but they do not stop it and they do not reverse it. So early cardioprotection is a genuine unmet need. Two other points worth noting. Cardiac function in these patients is closely monitored using cardiac MRI and echocardiography. So the endpoint is measurable and routinely used in clinical care. Because ifetroban acts on the downstream consequences of dystrophin loss rather than on the genetic mutation itself, it has the potential to benefit patients regardless of their underlying mutation type.

Its mechanism is also distinct from current cardiac therapies, and it was studied as an add-on to standard of care, not as a replacement for it. Let me turn to the market opportunity for Dyscorban in DMD cardiomyopathy. There are roughly 15,300 Duchenne patients in the U.S., with more than 300 new cases each year. Virtually all of them develop cardiomyopathy as they reach adulthood. Yet today, there are no approved therapies for DMD cardiomyopathy. This is now the leading cause of death in these patients. The development pipeline is thin, just 3 candidates, and Dyscorban is the only oral candidate of the 3, taken once daily. How does ifetroban protect the heart? The Duchenne gene mutation produces weakened muscle cells. That leads to calcium overload inside heart cells, oxidative stress, the death of those cells, and then progressive scarring of the heart muscle.