Leveraging Non-Dilutive Funding for Ultra-Rare Disease Development with Dr. George Magrath Opus Genetics

Elizabeth Jeffords, CEO and President of Iolyx Therapeutics, discusses dry eye disease and its connection to autoimmune conditions. The company's novel therapeutic topical immune modulator is designed to treat the root inflammation in the eye, which systemic drugs often fail to reach due to the blood-retina barrier. Patients with dry eye disease often have multiple comorbidities and are excluded from clinical trials, making this research even more significant in finding treatments for a growing population. Elizabeth explains, "Some people have physical dry eye, i.e., they have a dysfunction in their meibomian glands, and they can't make enough tears or those tears aren't the right composition. But more than half of the patients with dry eye have an underlying autoimmune disease. And they might know that, and they might not. So, patients with either Sjogren's disease or any of the thyroid conditions, patients with rheumatoid arthritis, MS, connective tissue dysfunction, most of those patients have some ocular comorbidities, and specifically, dry eye is probably one of the biggest ones." "Sometimes we treat the body, and we can treat autoimmune diseases successfully, but you don't really get most drugs into the eye. And so those alarm bells are still going off in the eye. And unfortunately, these patients with autoimmune disease tend to have more severe disease. They respond differently to the drugs that are out there today and probably most troublesome to us, and why Iolyx is really targeted these patients is that they get excluded from most of trials because they're just more difficult to treat, but they're also more difficult to treat because they have systemic medications that they're on, and most of those drugs get excluded." #IolyxTherapeutics #DryEyeDisease #Ophthalmology #AutoimmuneDisease #EyeCare #ImmunoOphthalmology #ClinicalTrials Iolyx.com Download the transcript here

Gene Mack, CEO and President of Gain Therapeutics, is combining AI-powered drug discovery with the development of allosteric modulators, drugs that bind to unique sites on proteins. The company's AI platform, Magellan, is crucial for accelerating drug discovery by reducing the time for computational screening of potential drug compounds.  Their lead compound is showing promising results as a potential disease-modifying therapy for Parkinson's disease, aiming to halt the progression of the disease rather than just treating symptoms. Gene explains, "So allosteric modulators of protein, it's a bit of a word salad, but what we're trying to achieve here is finding unique binding sites on proteins that are sort of away from the active site of that protein." "So, a lot of physics calculations go into these binding site calculations. The idea is to complete these quickly during the screening of hundreds or thousands of compounds. This process takes 10 to 15 minutes to run a set of computations and determine if a particular molecule is a fit for a specific protein. If that takes 10 or 15 minutes per compound, it's not a very big deal to go to that library if you need to get through billions, trillions of those compounds, and you need that computational speed to really fire up." "We are able to speed up those calculations from, let's say, 10 minutes to milliseconds. You can screen through much larger numbers of compounds and potentially even construct new molecules that are not known to the public domain, which would be a real key innovation." "What we think we have in our lead program, which is GT-02287, another molecule that was discovered through our application of Magellan. What we hope we have in GT-02287 is a disease-modifying approach to Parkinson's. Up until now, the only available treatments for Parkinson's are really just focused on the symptoms and allaying the severity of the symptoms." #Parkinsons #ParkinsonsDisease #AI #DrugDiscovery #GAINtherapeutics #DiseaseModification gaintherapeutics.com Download the transcript here

Paul Howe, COO of Protega Pharmaceuticals, is focused on how abuse-deterrent technology can address the opioid crisis and the need for mandates for insurance coverage of safer abuse-deterrent formulations. The SentryBond technology was specifically designed for immediate-release opioids to make it difficult to manipulate pills for abuse via crushing, inhaling, or injecting. Protega partnered with software company Opus to offer a program that helps educate chronic pain patients, manage their treatment, and provides physicians with risk stratification data to improve care and reduce the likelihood of abuse. Paul explains, "Most importantly from our standpoint is to protect from the risk of misuse, abuse, and diversion, which is escalation from orally taking medications to crushing, inhaling, injecting, or smoking. Unfortunately, when that escalation happens, many times patients end up on illicit fentanyl and heroin through the black market. So what we're trying to do is stop that escalation through our abuse deterrent technology and our medication. It's called SentryBond abuse deterrent technology, the company's Protega Pharmaceuticals."   "We also have a software program that we're now offering to physicians that deal and work with a lot of patients with chronic pain that really helps patients with chronic pain understand how to treat their pain and also titrate down off medications when possible, or at least get on the lowest possible dose and try multimodal care, try other types of avenues of procedures, different things they can do to help with their chronic pain." #ProtegaPharmaceuticals #PainManagement #ChronicPain #OpioidAbuse #OpioidPolicy #AbuseDeterrent. protegapharma.com Download the transcript here

Dr. George Magrath, CEO of Opus Genetics, discusses the company's experience in obtaining non-dilutive funding for its ultra-rare inherited retinal disease program. He highlights the challenges and advantages of this funding source, as well as the importance of establishing relationships with patient advocacy groups and foundations to mitigate risk and increase their attractiveness to traditional investors. The drive for non-dilutive funding for ultra-rare diseases is expected to become increasingly important in helping to get drugs to clinical trials. George explains, "Opus Genetics is a gene therapy company for eye disorders that occur in children. And these disorders are ultra-rare. It's 200 patients, a thousand patients per indication, and it's really good science. It's from Gene Bennett, who was the inventor of Luxturna, which was the first approved genetic medicine. And it does require some non-conventional thought on the financing, though, because these are so rare. And what we've done at Opus Genetics and have been really fortunate to be a part of is non-diluted funding from external sources. And that comes in the form of partnerships with patient families and organizations, like we just announced last week with RDH 12 Alliance." " It also comes with the Foundation Fighting Blindness, which has given us a number of grants and non-dilutive funding deals. And so those have been really important to Opus and have really helped us progress the programs. The way we think about that is the first principle is the clinical data we typically generate using our equity dollars. The preclinical work we try to do with non-dilutive funding, and that way we're able to focus on spending in rare scenarios." #OpusGenetics #RareDiseases #UltraRareDiseases #EyeDiseases #GeneTherapy #NonDilutiveFunding #Blindness #Ophthalmology opusgtx.com Download the transcript here

Lindsay Mateo is the Chief Commercial Officer at Weave. This company has developed a platform to automate and streamline the regulatory documentation process for FDA submission for pharmaceutical and biotech companies.  The regulatory lifecycle for drug development currently involves data from various sources in digital and paper formats. Weave Bio's tools are designed to automate and streamline administrative aspects of the regulatory process and create a living digital record of the development of the drug, which supports collaboration and saves time. Lindsay explains, "I look at the regulatory life cycle for any given drug program, and experts are at the core of that. Those experts, who are scientists, strategists, and project managers, essentially put all the work into the documentation that goes to regulators like the FDA here in the US. And that information goes on to allow this drug to continue through various stages of development to ultimately get to market and obviously help patients." "That is everything from early studies looking at how drugs are being handled in animals, in mice and rats, all the way through to clinical development. We start to get into humans and then again out through what gets drugs to market and even post-market expansion of various labels. So this is critical to getting any therapy to any patient with any condition. The process itself, the challenge with the process is that it's manual."  #Weave #WeaveBio #Pharma #Biopharma #WeavePlatform #AINative #AutoCT #AutoND #FDASubmission #RegulatoryDocumentation Weave.bio Download the transcript here  

Aspen Noonan, CEO of Elevate Holistics, a company that helps patients obtain medical marijuana cards through a telehealth consultation with licensed doctors. The qualifications for obtaining a medical marijuana card vary by state, and Elevate Holistics helps patients navigate the regulatory landscape. Many doctors are hesitant to recommend cannabis due to the federal illegality and restrictions from employers, and Elevate Holistics is filling the gaps by connecting patients looking for alternative treatments for conditions such as anxiety, depression, PTSD, and chronic pain. Aspen explains, "We are a one-stop shop for getting your online medical marijuana card. We connect doctors with patients 100% online in over 18 states to go through the process, which basically means book your appointment, fill out forms, join a video chat, call, and check for your emails because it's a hands-off experience. We really help people. I say A to Z, we don't just see you and get out. We ensure that your state application is filed correctly, so you ca your medical card at the end of the day."   "You have every right to ask your primary care provider for a medical marijuana recommendation. The problem is, and the reason we started, is that a lot of doctors do not want to sign their name next to something that says cannabis. They aren't allowed to go through their clinics. Let's say they work at a hospital, they work at a big organization, and they're not allowed to attach their name to the cannabis industry. And so that's where we come in and just take patients, take a provider, put them together."  #ElevateHolistics #Marijuana #Cannabis #MedialMarijuana #MedicalMarijuanaCard elevate-holistics.com Download the transcript here 

Mark Goddard, Vice President of Clinical Services at Infobionic.AI,  describes the remote cardiac monitoring system that provides near real-time monitoring of patients with potential cardiac irregularities.  The benefit of continuous monitoring allows for early detection of arrhythmias like atrial fibrillation, a growing concern driven by an aging population and factors like obesity and high blood pressure. AI-driven algorithms are used to analyze ECG data and identify potential issues, enabling proactive treatment and prevention of complications like stroke.    Mark explains, "The focus of our monitoring system is to provide near real-time monitoring of patients who are reporting maybe cardiac irregularities. The whole idea is to provide that near-real-time access to cardiac information so that arrhythmias can be identified relatively quickly. Additionally, the treatments for those arrhythmias are relatively quick as well, providing better patient outcomes. Just related to the fact that the data is always there, and it comes in maybe a minute or two behind live."   "We are partnered with a major health system that has an AI engine that is basically developing AI tools that can be utilized in cardiac monitors. And just looking at the patient's ECG, they're able to determine the potential for arrhythmias that may not have occurred yet. And that's kind of what we're focusing on. The ability to review ECG and understand those little nuances that may indicate, hey, this patient's going to have an event like atrial fibrillation, which is the most common irregular rhythm there is, especially for an aging population. Identifying those folks early can really help with not only the outcome for the patient, in that they're not going to run into the problems you may have if you don't recognize you have atrial fibrillation, but it'll also decrease healthcare costs, which in the end helps everybody." #InfobionicAI #MedAI #Cardiology #AFib #HeartMonitor #CardiacTracking infobionic.ai Download the transcript here

Dr. Steven Quay, Chairman and CEO of Atossa Therapeutics, is dedicated to addressing unmet needs in breast cancer prevention and treatment. Dense breasts are a significant risk factor for breast cancer, and Atossa's lead drug candidate has shown effectiveness in reducing breast density and lowering the risk of cancer. This dual-action drug blocks estrogen receptor signaling and induces programmed cell death in cancer cells, potentially offering a more effective and more tolerable treatment option. Steven explains, "So we are very focused on breast cancer to the point that Princess of Atossa, the company is named after, was the first woman in recorded history with breast cancer about four 50 BC. She was the wife of Darius the Great, who had the Persian Empire, the largest piece of real estate before the Roman Empire. And she had a breast lesion. It was documented that her slave cauterized it with a hot poker from the fire. We didn't get a follow-up from that, but anyway, we are dedicated to all the women, including her, and since then, who have been dealing with breast cancer."  "So it's a very interesting molecule. We call it a Janus molecule. Janus is the two-faced goddess. In this case, it is two-faced in a positive fashion because she addresses two different ways that cancer cells control themselves to drive the growth. Its major activity is to block estrogen binding to the estrogen receptor. So, as I said at the beginning, if 75% of all breast cancers are driven by estrogen, our drug is going to really nail 75% of all breast cancers. But then it gets even more exciting because it also has a second activity in another pathway in cancer driving called PKC beta."  #AtossaTherapeutics #BreastCancer #Cancer #DenseBreasts #Estrogen atossatherapeutics.com Download the transcript here

Dr. Alex Meves, distinguished dermatologist at the Mayo Clinic and a spokesperson for SkylineDx, discusses the challenges in diagnosing and treating melanoma and how a new molecular test called Merlin can help improve risk stratification and treatment decisions for patients with early-stage melanoma. Diagnosis and treatment have traditionally used tumor thickness as a primary risk factor. This molecular test measures the expression of genes in the tumor biopsy and can help determine the extent of surgery needed and whether additional therapies may be beneficial. Alex explains, "My department, when I started to work here at Mayo, wanted me to do some translational research, and I had just come back from a postdoc in Germany, at the Max Planck Institute, and I was tasked to get some research going. And so I focused on melanoma because I thought at the time there wasn't a lot of molecular research going on in melanoma that could be translated to patients. And so we started to develop biomarkers, sort of molecular tests that we could apply to tissue, and then help patients with." "Yes, so the problem that our research is focused on is what to do once you're diagnosed with a melanoma. What you want to do is to match the right therapy to the right patient. That's the goal. There are lots of melanomas that might not be very aggressive, and you don't have to do a lot of treatment. And then there's some melanoma that's very aggressive, and you want to do lots of treatment, but it's not always obvious which melanoma is low risk and which melanoma is high risk. And so this idea of risk stratification at diagnosis becomes very important to match therapy to patients." #skincancer #melanoma #SkylineDx #PersonalizedMedicine #PrecisionDiagnostics skylinedx.com Download the transcript here

Elaina McMillan, a director of product marketing at Edifecs,  describes the challenges and opportunities around healthcare data interoperability with a focus on prior authorization. Standardizing prior authorization workflows and data submissions across payers is a major focus to reduce delays and errors. Ensuring data security and patient consent are critical concerns, especially for smaller payers with limited resources to comply with mandates.  Elaina explains, "Primarily, what I work on and what Edifecs is most known for is our healthcare data interoperability platform, which includes EDI standards and the new-ish since 2020 FHIR standards. So we're really focused on that. How I would explain it is the data engine that helps payers get things done with quality data through the standardization of that. In addition, we have a whole set of what we call workflow solutions or applications that you can actually build on top of the data platform. So it helps to do things like claims correction, enrollment management, value-based payments, risk adjustment, prior authorization, and consent management."   "So the one that actually comes to mind for me right now, and largely that's because of the recent interoperability and prior authorization mandate, is the prior authorization workflow. A lot of providers don't like the workflow itself and the prior authorizations. What happens today generally is that providers are contracted with a lot of different payers. All of these payers have their own systems and processes through which the provider needs to submit. So one problem is that the providers are managing multiple different workflows, processes, platforms, and technologies." #HealthTech #Interoperability #HealthcareInnovation #PatientCare #PatientAccess #WomenInTech  #WhatIRun edifecs.com Download the transcript here