Empowered Patient Podcast

Rich Pascoe, CEO of Zevra Therapeutics, is working with a team with deep experience developing drugs and getting them approved by the FDA. Having developed multiple prodrugs for pain and ADHD, Zevra is now developing rare disease therapies with an initial focus on Niemann Pick Disease Type C and idiopathic hypersomnia. Rich explains, "Our lead product is a drug called arimoclomol. It's intended for the treatment of Niemann Pick Type C disease, or NPC for short. Niemann Pick primarily affects children. It's a lysosomal storage disorder condition, meaning patients lack the ability to break down certain things, including cholesterol. As a result, they develop a myriad of symptoms that ultimately lead to, in most cases, the patient's death. The issues they deal with revolve around loss of ambulation and the ability to swallow, and it affects their cognitive function. It's a progressive disease, and oftentimes, it can advance very rapidly." "A little history on arimoclomol. The drug was previously in the hands of a company out of Denmark. We acquired the asset last year from that company along with some of the individuals at the company that are critical for the success of the product going forward." "In that setting, Orphazyme, the company that had the drug before us, had submitted it for approval here in the US with the FDA. The FDA rejected that approval request based on a number of factors that we have subsequently been working to address. We believe that given our track record as a company of being able to work with the FDA to get drugs approved, in some cases under some very challenging circumstances, that we are well-equipped to handle and manage this submission." @Zevratx #ZevraTherapeutics #RareDisease #RareSleepDisorders #NiemannPickDiseaseC #IdiopathicHypersomnia zevra.com Download the transcript here

Steve Reed, Co-Founder, President, and CEO of HDT Bio, has spent his life focusing on tropical diseases and points out how the cost of developing and manufacturing vaccines determines access for a global population. HDT Bio was founded to develop more cost-effective technology to induce broader immune responses to various diseases. Their advanced RNA platform AMPLIFY is a technology applied to developing mRNA vaccines for infectious diseases and a therapeutic vaccine to help prevent recurrence and prolong survival from cancer. Steve elaborates, "COVID was something that came up right after we founded the company, and so the National Institute of Health asked us and funded us to develop a novel platform that could complement the platforms already in place for mRNA vaccines. Other indications that are incredibly important include other viruses. They're always coming up, and they're always mutating. But you probably read in the news about fungal infections on the increase in the United States and other countries." "The AMPLIFY technology is an RNA platform that expands in a limited way once it's introduced into the cell and then enables you to use a much lower amount of RNA. So that's significant in terms of cost, as well as safety. And then to be able to deliver the RNA, you have to protect it into a formulation, and the formulation we've developed is one that can be mass-produced at low cost and is very safe." "Back in the '90s and 2000s, when molecular biology, and monoclonal antibodies, those technologies became widely used and understood, we all naively thought that the road to a parasitic disease vaccine would be relatively straightforward. And in some ways, while the technology has enabled such vaccines, the practical limitations have stopped progress or severely limited it." #Vaccination #Vaccines #HealthForAll #COVID #mRNA #RNA #InfectiousDiseases #Cancer HDT.bio Download the transcript here

Jeff Liter is the CEO and Founder of Luminary Therapeutics, which is using gamma delta cells in their approach to CART-T cell therapy because of the ability of these cells to infiltrate solid tumors. With a multi-targeting system, Luminary has demonstrated promise treating multiple types of solid tumors, blood tumors, and autoimmune diseases. Their goal is to create a scalable model to manufacture allogeneic cells to drive down the cost and availability of effective cell therapies. Jeff explains, "We're focused primarily on three big challenges in the CAR-T industry. The first is the overall manufacturing approach and cost. The second is why do CAR-Ts, to date, not work in solid tumors. And then finally, the third is antigen escape and relapse of patients currently being treated by CAR-T therapies." "We have moved into coming back to the manufacturing area. We have moved into an allogeneic platform, which for your listeners, if they're not familiar with that term, means that we can make multiple products from one healthy donor. And we do that in a very special way, using gamma delta cells and preserving both subsets of the gamma delta cells. Then we also add some immune cloaking with our non-viral gene engineering, and that makes us very unique on the allogeneic front." "If you look at those 800 companies, probably 80, 85% of them use what's called alpha beta normal white blood T cells. A fair number of companies, maybe 7% to 8%, use NK cells. And then there's a new group using a different kind of subset of cells called IPSC. But in the gamma delta, there are only 12 to maybe 15 companies. So we're very differentiated on that front from the 800. And then, as I noted, we are the only company to have both Vdelta1 and Vdelta2 subsets in our final product." #LuminaryTherapeutics #CARTCellTherapy #CARTTherapies #Allogeneic #Autologous #CellTherapy #GammaDeltaCells #SolidTumors #Cancer #BloodCancers #AutoimmuneDiseases luminarytx.com Download the transcript here

Joe Tucker, the CEO of Enveric Biosciences, is discovering, developing and testing psychedelic-inspired therapies for the treatment of depression, anxiety, and PTSD. Current treatments target receptors in the brain to increase the concentration of serotonin in the brain by going through classic neurotransmitter pathways. The Enveric Psybrary portfolio and PsyAI drive drug discovery to create new molecules that synthesize chemistry and how nature makes molecules to target new receptors. Joe elaborates, "This one's completely different. This one is actually going through a receptor that's never been targeted before, 5-HT2A. And it's an adjunct to psychotherapy at this point. The leading molecules are an adjunct to psychotherapy. Although there's some interesting new work, including stuff that we're working on, no one has yet been able to separate those two in humans. But that's where we'd like to go next, is to separate them. But right now, it's really this 5-HT2A receptor, this induction of hallucination. No one else is doing that. No other molecules out there do that as part of a treatment program." "The issues with those classic psychedelics are, as you can imagine, they weren't designed as mental health treatment molecules. They came about for other reasons. And so they have a lot of side effects or safety issues that really make them suboptimal for patient use. So psychedelic-inspired says, let's take what we can learn from here. If you target the 5-HT2A receptor, you can induce some interesting effects in the patient, but can we then modify the molecules so they have better attributes?" "And in doing so, we also realized we're breaking new ground. I mean, we're making hundreds and hundreds of new molecules in an area where a handful of molecules had existed before. Five molecules, ten molecules, is what everyone else has looked at. We realized we were in this completely new field, and we had to come up with a way to identify prospectively which of these new molecules were going to be likely good drug candidates for various mental health indications." @Enveric_Bio #EnvericBiosciences #MentalHealth #Psilocybin #Anxiety #Depression #PsychedelicInspiredTherapeutic #PsychedelicBasedTherapeutic #Psychedelics enveric.com Download the transcript here

Dr. Joshua Schiffman is the CEO and Co-Founder of Peel Therapeutics and has taken the understanding of the natural resistance to cancer by elephants to develop PEEL-224 to attack tumors. Using a plant toxin and modifying existing approaches, Peel has created a drug that increases the amount of the drug that gets to the cancer cells with reduced side effects. Joshua explains, "Peel is actually the Hebrew word for elephant. So that's why we call ourselves Peel Therapeutics. My Co-Founder, Dr. Avi Schroeder, is from the Technion-Israel Institute of Technology in Israel. While we were working on the elephant drug, we realized, you know what, it's more than just elephants. Looking around nature, we understand that evolution has had hundreds of millions of years to perfect, in some cases, perfect cancer-fighting protein or peptide or anti-inflammatory molecule. Our lead drug, which we call PEEL-224, actually works really well targeting the DNA repair mechanism in cancer cells." "This DNA repair mechanism is called topoisomerase 1. And by blocking it, we're able to actually ensure that cancer cells essentially self-destruct. At Peel, we say that we like to engineer nature better than evolution intended. So, in the case of PEEL-224, we've taken this plant toxin, it's called camptothecin. It comes from an ancient Chinese tree called the Happy Tree, and we've modified it so that it can avoid chemoresistance. So, in other words, you give this drug to the patient, which goes to the tumor, and the tumor cell can't spit it out anymore. And we're seeing remarkable early results in mice and now have begun a clinical trial." @peel_tx #PeelTherapeutics #PeopleofPeel #Cancer #Inflammation #PEEL224 #Nature #Biotech #NaturesMoleculesIntoMedicine peeltx.com Download the transcript here

Cristina Varner is the life science Practice Leader at Newfront, a technology-focused insurance broker. While digital health and telemedicine companies have robust technology infrastructure, they tend to be less medically focused and unaware of insurance requirements to cover bodily injury, financial injury, data breaches, cyber liability, and state regulatory requirements. Wearables and data collected by them are adding issues of privacy, security, and data accuracy. Cristina elaborates, "I'm not going to say I'm surprised because a lot of the founders of these companies come from technology backgrounds, from tech companies. And so insurance just is not, and risk management is just not top of mind for many of them. And so we're not surprised. They're moving very quickly. I think when COVID came to be, digital health definitely exploded. And during the COVID period is when we started to recognize where some companies were lacking in risk management infrastructure. And I think that many of the companies we see now are much better positioned from a risk management perspective and also are more aware." "There are more customers and patients with wearables that are recognizing that their privacy and their security are important. I don't think historically, a lot of owners of wearables ever really considered those types of concerns. But now there's a lot more scrutiny in this area. And the other issue that comes up in tandem with privacy and security is data accuracy. If your data is not accurate on your wearable, for example, and it allows a patient to make a decision based on inaccurate data, which can result again in potential bodily injury to the patient." @NewfrontHQ #DigitalHealth #Telemedicine #Insurance #Wearables #RiskInsurance Newfront.com Download the transcript here

Seli Fakorzi, Director of Mental Health Operations at TimelyCare, and Gaurang Choksi, CEO and Founder of Violet, join me to discuss their partnership to bring comprehensive healthcare to college students from all socioeconomic backgrounds, genders, ethnicities, and physical abilities. Training healthcare providers with the tools to understand different communities allows clinicians to learn new ways to be more inclusive and build trusted relationships with patients. Seli explains, "Providing quality, equitable access to care is a foundational mission for Timely. We are aware that students from underserved and underrepresented populations many times possess unique barriers to care, and that has made seeking help increasingly difficult. Breaking down those barriers in as many areas as possible has been our goal. Forming this relationship and partnership with Violet will help in those areas." Gaurang elaborates, "I'll briefly share exactly how we get to understand how a clinician is doing, and then I'll share more about how we educate. At Violet, when a clinician joins us, we go through a process called benchmarking their cultural competence. We take a look at five pillars of information, including who is that specific person, what communities their family or they belong to, we look at what are the communities they feel really confident serving. That's a clinical measure called self-efficacy that we've pulled in." "Then we also look at what are the communities they want to work with, the communities they've studied about and the communities they have worked with, using claims data and their voices. Then using all this information, we get to have clarity on is that specific provider doing well working with the BIPOC or LGBQ or TGNC communities." #TimelyCare #JoinViolet #Healthcare #VirtualCare #LGBTQHealth #TGNCHealth #BIPOCHealth #MentalHealth #HealthDisparities #HealthEquity #MinorityMentalHealth timelycare.com joinviolet.com Download the transcript here

Michael Axt, the Chief Member Empowerment Officer at Zelis, is focused on helping healthcare payers and clinicians provide more information to patients about expected costs for procedures and visits to providers and hospitals. A recent survey conducted with Hanover Research revealed insights about patients' challenges in understanding healthcare billing and correcting errors. Driven by the move to transparency, Zelis acquired Sapphire to offer the Sapphire Digital solution to enable patients to better understand insurance benefits and the correct amount the patient owes. Michael explains, "We found some interesting insights. Over 40 percent of the consumers in our study indicated that they were significantly frustrated in trying to find the right person to resolve an error. So they might have found an error, but they didn't know how to do anything about it. For those individuals, it took a month or more for nearly half of our respondents. For 70 percent of them they spent over two hours of their own time trying to work to correct this." "To the extent that there's greater clarity for the member or the patients in understanding the bill and what they owe, they're more likely to pay it more promptly. So we all know that cash flow is a challenge, and collections on patient liabilities are frankly atrociously low in the healthcare space." "For the health insurance companies and payers, there are options as well. Obliviously improving the member experience helps to improve retention and reduce churn in the plans. Also, if they're bringing tools that help to accelerate payment to the provider, it has opportunities for them to improve their relationships with their provider networks, which is a huge strategic asset for the health insurance companies." @ZelisHealthcare #Healthcare #PaymentIntegrity #HealthcareCosts #ConsumerBilling #PriceTransparency #HealthcareBilling #MedicalBillingErrors zelis.com Download the transcript here

Patricia Panzarino, otherwise known as Pidgie, was born with spinal muscular atrophy, SMA, a severe progressive neuromuscular disease. As a singer and songwriter and one of the oldest women living with SMA, Pidgie is celebrating the release of her new album, Just Breathe. Her experience of successfully taking Evrysdi from Genentech has given her more energy and enthusiasm. Pidgie elaborates, "They didn't know as much when I was born. I was six months old and did not achieve the milestone of crawling properly. My mom took me to the doctor. I had an older sister who had it as well. She was 12 years older than me, so they said, "Here we go again." They called it Amyotonia back then. But as far as a solid diagnosis of blood tests and DNA and stuff, truthfully, that was three years ago. But SMA used to be diagnosed clinically. So I knew I had it, but it was a different way to diagnose it." "Before the medication, you're talking 61 years of lifestyle—great parents kept me active. My father adapted things for me to participate more fully. For example, we had a boat that you could sleep on with a flying bridge, and he took a Hoyer lift that you use to transfer, put it on the flying bridge, and hoisted me up, so I could pilot this 31-foot boat. I was kept very active, taking vitamins and eating healthily. I never really did drugs or got really into alcohol. I just tried to keep a healthy balance and positivity until the medication. Now I'm still doing all of that because that really helps. You can't take it and just party and not eat well and stay up late and not sleep." #PidgieMusic #Pidgie #SpinalMuscularAtrophy #SMA #NeuromuscularDisease #Evrysdi PidgieMusic.com Download the transcript here

Dr. Myron Czuczman is the Chief Medical Officer and Executive Vice President of Citius Pharmaceuticals, which is developing an advanced treatment for cutaneous T cell lymphoma, CTCL, a rare form of non-Hodgkin lymphoma. The original version of their drug ONTAK has been reformulated, and I/ONTAK is now in clinical trials to confirm efficacy in controlling severe itching and improving the quality of life for patients. Myron explains, "This is a protein, a fusion protein, of interleukin-2, the whole molecule that has been fused with diphtheria toxin. With respect to any of the cells like the cells we're talking about, CTCL, it actually binds to the IL-2 receptor, internalizes, and in that manner, kills the tumor cells." "The agent ONTAK was taken off the market, and this new version, I/ONTAK or E7777, is a reformulation. It's more purified and it's about one and a half to two times more bioactive than the original ONTAK. From the clinical trials which led to our submission of the BLA recently, we've looked at the safety and efficacy, and it's consistent with the original formulation. There are no new safety issues, and it has good activity in patients, even heavily pretreated patients." "The issue here is, unfortunately, this disease, the relapsed/refractory CTCL, is incurable. The only time one has a chance to cure it is if you get it early, if you have young patients that have what we call an HLA compatible donor. In a very small number of patients, an allogeneic stem cell transplant could be curative. But the vast majority of these patients, who are elderly, are not candidates for allogeneic transplant. So, unfortunately, these are incurable individuals with bad diseases." @CitiusPharma #Cancer #Lymphoma #RareDisease #Immunotherapy $CTXR citiuspharma.com Download the transcript here