Hi, good morning, everyone. Thanks for joining us for our 45th Annual Healthcare Conference. I am Vishwesh Shah, an associate on the biotech team here at TD Cowen. It is our pleasure to introduce Todd Bazemore, Interim CEO of KALA BIO, as well as Mary Reumuth, the Chief Financial Officer in the audience.
Thank you, Vish. Thank you very much, and thank you for joining us this morning. I'm going to start off with our disclaimer and notices before getting into the presentation. KALA BIO is leading the emerging field of mesenchymal stem cell secretome therapies. We have a platform that allows us to pursue multiple potential orphan indications, both in the anterior and posterior segment of the eye. Our first program is KPI-012, and we are targeting a rare ophthalmic indication called persistent corneal epithelial defects. We estimate this market alone in the US to be worth more than $1 billion and growing at double-digit rates annually. We also have a preclinical program for a secretome called KPI-014, which is a mesenchymal stem cell secretome being developed specifically to target inherited retinal diseases such as Stargardt's and retinitis pigmentosa.
We have had a very meaningful milestone accomplishment over the past couple of years. We filed and received acceptance for an IND in late 2022. That IND was accepted in early 2023. We began a Phase II b pivotal trial for KPI-012 and PCED in about May of 2023. We have received both Fast Track and orphan designation for this product candidate, and we are currently on Track to complete the ongoing pivotal trial towards the end of the second quarter of 2025. In April of 2023, we announced that we had received a $15 million grant from the California Institute for Regenerative Medicine. We did complete a couple of different PIPEs in 2024, $12.5 million back in the mid-year timeframe and nearly $11 million at the end of the year. Our cash position as of the end of Q3 of 2024 was just shy of $50 million.
Of course, we have since added the proceeds from the PIPE that was executed in the fourth quarter of last year. We estimate that our current cash runway brings us well into the first quarter of 2026. We have a very experienced management team in place with deep and significant experience across both ophthalmic diseases as well as orphan indication products. What exactly is a secretome? We go through a process in which we take human bone marrow-derived mesenchymal stem cells. We then, in our manufacturing process, are able to culture up these stem cells to get them to produce biomolecules that are associated with wound healing. These biomolecules are what are known as the secretomes. We currently have CMC plans in place that are allowing us to execute on good GMP manufacturing with consistency across batches.
We believe that by delivering just these proteins or these secretomes associated with the wound healing and not also administering the cells, we are able to avoid some of the logistical and safety concerns typically associated with cell therapies. We also believe that this approach allows us to target multiple orphan ophthalmic indications in both the anterior and posterior segment. We are initially focused on persistent corneal epithelial defects. I'll spend some time in a moment talking about the Phase I results of the initial trial that was conducted, as well as the design of the ongoing Phase IIb trial. If successful in PCED, we would then plan to quickly turn our attention to limbal stem cell deficiency, which we believe is an orphan indication that has a similar market size opportunity to PCED.
As I stated earlier, we are in preclinical development work of a back-of-the-eye secretome that would allow us to target a number of inherited retinal diseases in a gene-agnostic approach and potentially allow us to treat all comers with those diseases. KPI-012 is made up of a series of these Secretomes. Specifically in our product, some of the most important secretomes contributing to the clinical effects of the drug are growth factors, protease inhibitors, matrix proteins, and neurotrophic agents. This really allows us to have a multifactorial approach towards wound healing. This is going to be really important, as you'll see in a moment as I get into the vast array of different etiologies that can possibly result in a persistent defect on the cornea. We believe, because of our multifactorial approach, in the ability to address all PCEDs regardless of etiology.
As I said earlier, the FDA has already granted both orphan drug and fast track designations for KPI-012. A PCED is a non-healing corneal defect that's refractory to conventional treatments or conventional therapies. It's a defect that has been on the eye for typically 10 to 14 days and has been non-responsive to therapy. At that point, these defects can start to cause a number of different sequelae, including pain, photophobia, and visual impairment. If these wounds are not healed very quickly, the risk of infection becomes quite high. With the potential of infection or infection occurring in the eye, these lesions can result in stromal thinning and scarring and have the risk of potential permanent vision loss. There are a number of etiologies that can cause these defects on the cornea.
It includes things like neurotrophic keratitis, diabetic keratopathy, severe ocular surface disease, trauma to the eye, infectious keratitis, ocular surgery, and there's probably about a half dozen or so other potential causes of these persistent defects. Patients often can have more than one underlying etiology. It is not uncommon for the patient to have several of these etiologies resulting in the defect. As a result, we believe the importance in having a multifactorial approach to treating and healing these wounds. The estimated incidence is 100,000 patients or cases per year in the US and 238,000 patients in the US, EU, and Japan combined. We have successfully conducted a Phase Ib clinical trial showing efficacy where the wound was completely healed in six of eight patients treated. We are currently conducting a large Phase IIb trial.
PCED is highly clinically burdensome to patients, and it's an area that has a significant amount of unmet need. When you talk to corneal specialists, of which there's about 1,800 in the US, and these are ultimately the physicians that are typically seeing and treating these patients, they say that there are several benefits that they're looking for in a therapy. They want one therapy that can address multiple etiologies. That is important because there's one product approved in the market today. That product is called Oxervate from an Italian private pharmaceutical company by the name of Dompé. It's been a very successful launch over the last five years in the PCED market. That drug is only indicated for neurotrophic keratitis, which we estimate to be about one-third of all of the etiologies resulting in PCED.
We have heard consistently from corneal specialists that they would like a therapy that's not limited to just neurotrophic keratitis, but has the ability to treat all etiologies. Also, rapid and sustained wound healing, right? The longer that wound is on the surface of the eye, the higher the risk of infection and resulting in potential permanent vision loss. The ability to very rapidly heal these wounds with good durability and for that effect to maintain over time. Additionally, therapies that are well-administered, well-tolerated, and easy to administer. We hear consistently that they're looking for a product that will not have significant AEs that inhibit or prevent the patient from staying on therapy.
They are looking for therapies that are very easy to administer, which we feel like we are solving with our product we are developing, which you will see in a moment comes in a premixed unit dose vial. This is a very large orphan disease market in the US. Oxervate achieved over $800 million in reported revenues in 2023 and is expected to have succeeded $1 billion and achieved blockbuster status this past year in 2024, which was just their fifth year post-launch. If successful, KPI-012 will be the first product with a broad indication to treat all comers, all PCEDs, regardless of etiology. It will be a simple-to-use product administered four times a day. We believe from our Phase Ib results, we will not have adverse events such as eye pain when administering the drug.
This slide denotes what impaired healing looks like on an eye with persistent corneal epithelial defects and the multiple pathways that are affected when a patient has PCED. The approach that we're using with KPI-012 in order to heal these wounds, and our multifactorial approach specifically focuses in on four key areas. That's growth factors that we're providing, such as HGF and PEDF. It's protease inhibitors like TIMP-1, matrix proteins like fibronectin and collagen, and neurotrophic agents such as PEDF, so that we have the ability to impact the healing process along multiple pathways, which again, we believe will contribute to our ability to heal these PCEDs regardless of etiology. We did conduct a Phase Ib trial several years ago. There were eight patients enrolled in this trial. Six of the eight achieved complete healing within four weeks, or 75% of the patients that were treated.
You can see here on this graphic on the right, this is patient—let me back that up. We went too far. Just one moment, please. This is patient 016 from the Phase Ib trial. You can see the staining, which is what's used. This is corneal fluorescein staining, which is used to measure the efficacy of the drug. You can see significant staining on the eye at day one. After just one week or seven days of treatment, complete healing of that wound, which is demonstrated by there being no corneal staining on that patient after a week of therapy. If we look at each individual patient in the Phase Ib trial, you can see that there were a number of etiologies represented in this trial. Multiple diabetic patients with neurotrophic disease. There was a patient with Stevens-Johnson and dry eye disease, a patient with post-infectious keratitis.
We had a patient with severe dry eye. As you can see, of the six patients that achieved complete healing, four of the six were completely healed within the first week of treatment. A fifth patient achieved complete healing by week two. The sixth patient achieved complete healing by week four, with those six patients all having been healed within four weeks of therapy. We also measured pain as an important secondary outcome of efficacy. In the trial at baseline, six of the eight patients reported pain as a symptom of their persistent defect. What we saw is a rapid response by week one. All patients experienced a reduction in their pain. By week three, the six patients had all achieved complete resolution of their pain.
This is really important because the one product that's currently in the market has demonstrated pain as an actual adverse event that occurs in a significant number of patients once they are on therapy. We, of course, wanted to take a look at and make sure that we did not have pain as an AE, but have also measured it as an efficacy outcome and have been able to demonstrate that any patient that experienced pain at baseline achieved complete resolution of that pain within three weeks. We are progressing towards a phase IIb readout at the end of Q2 of this year.
We have recently had a successful, or I guess it's within the past year, a successful Type C meeting with the FDA to discuss our CMC and manufacturing plan, in which we had complete agreement and alignment on a matrixed approach that will use both biopotency assays and quantitative measurement of CQAs to ensure consistent batch-to-batch reliability. We have already demonstrated that now across multiple engineering batches, as well as clinical supply batches. The ongoing Phase IIb trial is a study in 90 patients. There are two active treatment groups, a one-unit and three-unit dose, and the vehicle or placebo group. We are targeting 30 patients per treatment arm. We are treating these patients. There is a one-to-one-to-one randomization into the trial. We are then treating the patients for eight weeks.
At the conclusion of the eight weeks, we will be assessing the degree to which the wound has healed. There is then a follow-up with that patient at week 10, two weeks after they have been off of therapy to ensure that the wound has remained healed. There is an additional follow-up six months post-treatment to determine whether there has been a reoccurrence in any patients with this wound. The primary endpoint for this study is maintaining, or rather achieving, complete healing of the wound at week 10. Treated for eight weeks, two weeks off of therapy, we bring them back at week 10. The rate of healing at week 10 is the primary endpoint. We are targeting a top-line data readout at this point towards the end of second quarter of 2025.
In closing, KALA is a leader in this emerging field of mesenchymal stem cell secretome therapies. This is a very large market within PCED alone, but really provides us a pipeline and a product opportunity that, if successful in PCED, we can quickly pivot and begin doing clinical trials with the exact same formulation supported by the exact same CMC and IND in order to pursue other front-of-the-eye orphan indications. As we said, we've had a number of successful, meaningful milestones achieved over the past two years. We have our biggest one in front of us coming up here at the end of second quarter of this year when the Phase II study will readout. The company is in a very strong cash position, having successfully raised a couple of pipes in 2024, expecting data mid-year this year with cash well into the first quarter of 2026.
That essentially provides us nearly three full quarters of cash beyond an expected data readout. As we stated earlier, our current projected cash runway is until nearly the end of the first quarter of 2026. I'm going to stop there. That's all I have for today. I thank you for your time and your attention.