Scott Pescatore, Executive VP of the Rare Diseases Business at Recordati, is focused on rare and ultra-orphan diseases with high unmet needs by acquiring promising compounds from other companies and advancing them through development and approval. An example is the company's drug Isturisa, acquired from Novartis, which is an FDA-approved effective treatment for patients with Cushing syndrome, a rare endocrine condition. Raising awareness of rare diseases among physicians, patients, and the general public is a priority for Recordati to improve diagnosis rates and clinical trial participation, and to encourage more research and funding in the rare disease space.

Scott explains, "We have two primary divisions at Recordati. One is our specialty primary care business, and the other is the rare disease business, which I have the honor and privilege to look after. And we have a very simple sort of work ethic or business mantra, if you will, and that's focused on the few. And we really dedicate ourselves to focusing on disease areas and patient groups and therapeutic areas that have a high unmet need and really low or limited options for patients. And really focusing on diseases and areas that are rare and considered ultra-orphan by the definitions in the US, where really there's a very small patient base. And that's where we began back in 2007, when the rare disease business was formed. And that's really what our focus has been since then. And we continue to focus on this segment of the market."

"So Isturisa is really a fantastic product. We acquired this product through a deal we did with Novartis Pharmaceuticals back in 2019, and this product has FDA approval for patients who have endogenous hypercortisolemia with Cushing syndrome. So it's quite a nasty disease, but it's a very efficacious product. The product is what's considered a cortisol inhibitor in the blocks in a particular enzyme to help normalize hypercortisolemia in patients with Cushing syndrome. And Cushing syndrome, for those who aren't familiar, is a rare endocrine condition that really has a significant impact on patients' quality of life, on the caregivers, on the families. And the indication I mentioned was supported by quite robust phase 3 trials."

#Recordati #RareDisease #FocusedontheFew #CushingSyndrome #IMCD #CastlemansDisease

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Martin Burns, CEO of Bruin Biometrics, is focused on preventing pressure injuries, which are often not detected through visual and tactile assessments because damage starts invisibly under the skin. The Bruin scanner technology identifies pressure injuries by measuring sub-epidermal moisture, which can predict later tissue death, highlighting the condition that can be treated before it develops further. This handheld device is skin tone agnostic, addressing a significant health disparity and providing an objective number indicating the presence of excess fluid, allowing clinicians to act quickly.

Martin explains, "They are surprisingly common, and I emphasize surprisingly because pressure injuries are typically not talked about in the mainstream, but actually they affect about three to three and a half million patients a year in the US. And really shockingly, of those three to three and a half million, about 60,000 people die from them every year. So think about that as in the list of the top 10 leading causes of mortality in the United States, which is the surprising part. When you speak to friends and relatives, they've often heard of them potentially as bedsores, but nobody really has an appreciation of just how significant they are. How widespread or how deadly."

"The initial stages of it are imperceptible to any practitioner, but are actually measurable by objective technologies, and they don't become visibly manifest or physically manifest until much later. And what happens is they end up breaking the skin surface. As you can imagine, every time the skin is broken is an opening for infections and significant complications, which actually is the thing that ends up causing huge amounts of additional lengths of stay and costly treatments. And to the extent that those don't work, mortality."

"Thankfully, our scanner is one in which the mechanism it uses disregards skin tone entirely. In other words, skin color doesn't matter for the scanner. What we are measuring is an increase or a decrease in fluid at the specific site that we're measuring. And so it's skin tone agnostic, which is rather brilliant because dark skin tone patients die at a rate four times more than any other cohort, which is an absolute travesty and one in which it simply doesn't need to happen. And our scanner is a leading reason why it doesn't need to happen."

#BruinBiometrics #Prevention #PressureInjuries #HospitalSafety #PressureInjuryPrevention #PatientSafety #Hospitals

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Robert Jacks, President and CEO of Sparrow Pharmaceuticals, identifies that an elevated cortisol level is a newly recognized cause of treatment-resistant type 2 diabetes. A significant portion of patients with diabetes who do not respond to standard treatments, including GLP-1 agonists, have underlying high cortisol. Sparrow has developed a drug designed to lower cortisol levels inside cells, directly addressing the underlying driver of the disease, and to be used as a complement to existing treatments. This concept of targeting cortisol-driven resistance could be extended to other conditions, such as treatment-resistant hypertension.

Robert explains, "I feel as though Sparrow has come full circle, actually, with the mechanism of our drug. Originally, we have a drug that targets HSD-1. We can talk about what that is, but it's involved in intracellular cortisol regulation. This was a class of drugs that was originally developed targeting cardiometabolic diseases like type 2 diabetes. And the drugs had some moderate efficacy, but they weren't well differentiated in a broad population and largely were just continued for commercial reasons."

"Our company was founded a number of years ago based on the idea that these drugs had real potential but hadn't been used in the right patient population. And that being the patient population with the disease that we know is driven by excess cortisol toxicity, because that's aligned with the mechanism, as I was mentioning. So we generated some really interesting data in a rare disease called Endogenous Cushing syndrome. This is a very severe orphan disease with patients who have very severely elevated cortisol, showing in fact that yes, this mechanism does seem like it could have a very major impact in the right patient population."

"Simultaneously, another company published some data showing that actually there's a very large population of people with treatment-resistant type 2 diabetes, a very high level of medical need, and that their underlying disease actually is being driven by elevated levels of cortisol. And so when you bring together the data that we generated and what appears to be a large amount needed in a large population, it seems like we may have the perfect solution for that. So we've refocused our efforts on a broad population of treatment-resistant type 2 diabetes in patients whose disease is being impacted or driven by elevated cortisol levels."

#SparrowPharmaceuticals #Type2Diabetes #CardiometabolicDisease #CortisolRegulation #Cortisol #GLP1 #RareDisease

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Jesse Levey, CEO and Founder of Longevity Health, has a focus on extending the health span not just the lifespan of more people by providing access to tools and preventative health information. Lessons learned from expensive concierge medical services are being applied to a broader population, driving down health costs and democratizing longevity medicine. Using AI to build a scalable and personalized approach to wellness not just sick care, Longevity Health aims to make an AI doctor available around the globe.

Jesse explains, "Our mission is to help a billion people live to a hundred in good health. That's the vision. And the way that we get there is by building an AI doctor trains of ed on longevity medicine and distributed around the world. We have a three-phased business model. Phase one is to take the sort of hundred-thousand-dollar-a-year longevity concierge experience and deliver it for $10,000. So we've taken this really high-end experience that combines an executive physical with a longevity concierge physician and a team to help you implement the recommendations. And we've delivered that for $10,000. That's been around for about two years."

"Phase two takes that down to $1,000 with the help of AI. So it reduces the time spent by humans and the need to spend a lot of time with clinicians. And so it's a mix between clinician time and AI interactions. And then eventually, as the regulation allows and as the technology improves, we believe that we'll be able to deliver this experience for as low as $10 a month or $100 a year via an AI doctor. And so that's the future. That's what we're building towards."

#LongevityHealth #Longevity #HealthyAging #HealthSpan #FunctionalMedicine #HealthAI #Aging

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Sean Cassidy, CEO and Co-Founder of Lucem Health, is applying AI to identify patients at high risk of undiagnosed conditions like cancer and diabetes. The goal is to facilitate earlier diagnosis and treatment by flagging patients that need screening based on the AI plaform's ability to analyze EHR data and demographics of diverse patient populations to ensure broad scalability. This technology was designed to integrate into existing clinical workflows for established screening procedures rather than making direct treatment recommendations.

Sean explains, "The origin of the company, the idea for the company, originated within Mayo Clinic in about 2020. Mayo Clinic has faced a challenge, and I think sometimes continues to face a challenge that a lot of researchers in AI have faced, which is how do you get promising AI in a clinical context from the so-called bench to the bedside? How do you get it from the lab into clinical practice? And what they realized was that while the data science and the AI part of it is really interesting, what was needed was scaffolding around the AI to facilitate integration with data and integration with workflows, a measurement and monitoring system, and so on and so forth."

"We are trying to facilitate, and you're going to see us begin to expand the aperture, if you like, or open the aperture of how we position the company. Because as we've gone on, we have realized that the opportunity here is to actually help healthcare provider organizations, health systems, and so on, create really high-impact care delivery programs that have at their core or feature at their core earlier diagnosis, accelerated treatment, earlier treatment and therefore better outcomes for patients and hopefully even saved lives. So that's the generic approach that we take."

#LucemHealth #AIinHealthcare #HealthcareAI #HealthTech #EarlyDiseaseDetection

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Dr. Waqaas Al-Siddiq, CEO and Founder of Biotricity, has designed a direct-to-consumer service to shift healthcare from a reactive to a preventive model by simplifying and accelerating access to remote cardiac screening. Applying AI to sift through patient data allows the cardiologist to focus on clinically relevant information and to identify sporadic, intermittent heart issues that are often difficult to detect using traditional heart screening techniques. Waqaas predicts an expansion of specialized solutions using AI and large datasets to support clinicians and patients in the drive to identify early signs of disease.

Waqaas explains, "The thing that we've been focused on is, as you know, heart issues. We've talked extensively about heart issues, which are the number one killer. And so what we've done now is we've collected this massive dataset, but we're trying to look at the nuances of those needles in a haystack. So it's not about the individuals who have arrhythmias to catch. It's about the ones who are very, very sporadic and intermittent, something that happens once every four months. Is there a way to predict that? Can we grab additional data from the patient about their environment, about their history, to get a more holistic view of the patient? "

"Often, with a massive amount of data in a set, you can get into prediction, but you need a more holistic view. Our focus has now been on now that we've captured 90% of the scenarios, the last 10% are incredibly complicated, and how do we leverage that? How do we use our data to basically get into those specialized nuances?"

#HeartSecure #hearthealth #preventative #heartdiease #healthyheart #selfcare #healthylifestyle #healthtestathome #HeartYourHeart #Biotricity #CareInnovation #HealthcareAI #Bioheart #Cardiology

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Neil Klompas, President and CEO of Augurex, has a mission to improve the diagnosis of autoimmune diseases, with a primary focus on axial spondyloarthritis, a form of inflammatory back pain that is often misdiagnosed. The Augurex blood test SpineStat is designed to differentiate axSpA from mechanical back pain by identifying a specific biomarker present in axSpA. Currently, there is an average nine-year delay from the onset of symptoms to an accurate diagnosis of axSpA, causing irreversible spinal damage and years of using drugs and therapy with no benefit.

Neil explains, "I think we're all familiar with back pain. Some of the NIH data and WHO data out there show that almost 28% of adult Americans live with chronic back pain, back pain lasting more than three months. It can be a real journey to figure out what the drivers are, with some patients really never getting resolution, resulting in pain management and, in some cases, even opioid pain management. But if you dive deeper into that category of back pain, there are really two broad classifications we can think about. We can think about mechanical back pain, which is due to lifting, twisting, sports injury, or maybe a car accident. But then there's this whole other category, as you indicated, called inflammatory back pain, which really has its roots in autoimmune, where the body's immune system is actually attacking itself. "

"Well, this is when the body's immune system attacks its peripheral joints. In inflammatory back pain, or as you said, axial spondyloarthritis, it's an autoimmune condition where the body's immune system actually attacks the spine. This can lead to pain increasing over time, new bone growth, fusion of the spinal cord, and permanent stiffness and loss of mobility. It's progressive. It keeps getting worse, and because it's an autoimmune condition. Simply stretching or swearing to take up yoga or getting a new pair of runners or doing PT or massage, while it might help with some of the symptoms, it's not going to slow down or stop the disease. The challenge with axSpA, with axial spondyloarthritis, is that while it's not really that well known or talked about in the media, it's actually more than twice as prevalent as rheumatoid arthritis, a condition which many people have heard about."

#Augurex #AdvancedDiagnostics #AutoimmuneDiseases #axSpA #AxialSpondyloarthritis #BackPain

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Dr. Petri Bono, Chief Medical Officer at Faron Pharmaceuticals, describes the development of bexmarilimad, a novel first-in-class immunotherapy that, unlike existing checkpoint inhibitors targeting T cells, targets the Clever 1 receptor on macrophages. This treatment is designed to reprogram the tumor microenvironment by switching marcophages from suppressive to active, enabling the patient's immune system to recognize and attack cancer cells. The primary disease target is higher-risk Myelodysplastic syndromes because the cancer cells in virtually all MDS patients express the Clever 1 target.

Petri explains, "We are developing a completely new type of treatment. Currently, cancer patients are treated with immunotherapies called checkpoint inhibitors that target immune checkpoints. But our approach is targeting completely different cells, not T cells, but rather macrophages. And that's why we are first in class with a novel mode of action. And that's why it's important that these macrophages are shown to, for example, contribute to treatment resistance in many tumors."

"Clever 1 actually is a receptor that was identified about 20 years ago. It found a certain macrophage as well as myeloid cells. And Clever 1 keeps the immune system in a tolerant and suppressive state. In cancer, for example, these Clever 1-positive macrophages essentially help the malignancy grow instead of helping to fight against it. And then our approach is that we want to block Clever 1 with our monoclonal antibody, bexmarilimab. So those macrophages switch the phenotype into an active antigen, preventing a pro-inflammatory state, and this reawakens immune surveillance. It allows T cells in the system to actually recognize the malignant cells themselves as dangerous and mount a proper antitumor response. So, a completely new mode of action by targeting Clever 1, we are not just adding another cytotoxic mechanism. We are removing the immune break and enabling the patient's own immune system to do the job that it was originally designed to do."

#FaronPharmaceuticals #BloodCancer #MDS #MyelodysplasticSyndrome #HR-MDS #CancerResearch #novelimmunotherapy #Bexmarilimab #Clever1

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