Kiora Pharma CEO Brian Strem, which is developing a small-molecule retinal drug, shares early thoughts about KIO-301’s commercial positioning with Jack O’Brien. Lecia Bushak discusses former President Trump’s comments over the weekend seeking to revive Republican debate around repealing Obamacare. And Dr. Ruth Westheimer’s appointment as New York state’s honorary ambassador to loneliness tops our Trends segment, along with concerns over lead-tainted applesauce and one woman’s dying pledge to buy up others’ medical debt. Music by Sixieme Son.
Note: The MM+M Podcast uses speech-recognition software to generate transcripts, which may contain errors. Please use the transcript as a tool but check the corresponding audio before quoting the podcast.
Hey, it’s Marc.
We hope everyone out there had a peaceful holiday weekend. Maybe you enjoyed a meal with family, friends or a significant other. Maybe you even did some of the cooking. As for my Thanksgiving – it involved getting lost while trying to follow a somewhat ambiguous turkey recipe. And while all turned out OK in the end, I hope your kitchen adventures were less eventful than mine.
With calendars turning to December in a day or two, it means the JPMorgan Healthcare Conference is right around the corner.
In anticipation, we’re kicking off the end-of-year sprint with an interview with the CEO of pharma company Kiora Pharma.
The co is focusing on a group of genetic or age-related retinal diseases that result in pats losing their low-level light vision. Kiora’s lead asset is designed to restore vision for these patients, and phase 1b clinical trial resuts were positive .
Pres and CEO Brian Strem tells Jack OBrien about the initial finduings , and some of ways Kiora is thinking about commercially positioning the drug, esp in light of gene therapies like Novartis’ Luxturna , which is the only other treatment for these patients but has a significant price tag.
And on a programming note, MM+M is planning to cover the JPM conference once again. So if you’d like to set up a meeting, pls don’t hesitate to send me email to [email protected]
And Lecia’s here with a health policy update.
Hey Marc, today I’ll discuss President Trump’s comments over the weekend seeking to revive the old Republican debate around repealing Obamacare, or the Affordable Care Act, leading up to the election year.
And Jack, what’s trending in healthcare this week?
This week, we’re talking about an FDA recall on applesauce pouches containing lead, Dr. Ruth being named New York state’s loneliness ambassador and how the passing of a cancer patient eliminated millions in medical debt.
it’s great to have you on the show here for those in our audience who may be unfamiliar with Kiora. Do you want to give a quick rundown of your organization? Yeah.
Happy tuna. It’s a real pleasure to be here as well Jack. Thanks for having me.
So cure is a publicly listed microcap pharmaceutical company that is focused on developing new therapies for patients with orphan retinal diseases. So basically the types of diseases that someone is born with and start to have some onset of symptoms anywhere from the first few years through about teenage years of life that then leads to them going blind.
And I we’re gonna get into the space in Greater detail, but I want to focus in on and I’m gonna try and say this correctly retinitis Pigmentosa.
I nail that perfect. Awesome. Can you give our audience a sense of what that is in the larger orphan retinal disease State? Absolutely. So
retin, I just Pigmentosa or just even simpler way to say it RP is actually a group of different diseases. If you will it’s been known to be caused by over a hundred and fifty different unique gene mutations. Any one of these mutations can actually cause this disease and what happens in this disease is typically individuals and about their second decade of life.
Start to exhibit some or feel some signs of inability to see in dark situations. So losing their low-level light vision.
And then that tends to progress to losing peripheral vision and then eventually central vision.
It really is going to be dependent on the specific mutation that they have as to what that rate of progression could be. Some patients are fully blind in their 30s. Whereas others can make it into their 60s or 70s before they’re full vision loss kicks in and
obviously it’s such a key area that I know you and other companies are focusing on in the vision loss space and being able to give these patients some element of control or treatment to that and can you talk to me about Kio 301 and the abstract that you just presented in early November at the aao conference.
Absolutely. So po301 is a small molecule.
Has the ability to turn cells in the retina that are normally not light sensitive into light sensing cells.
And so what’s happening in retinitis Pigmentosa is the cells that normally take light and convert it to the Electoral energy required by the brain is something or there’s something called rods and cones or photoreceptors and I think most people have heard of their rods and their cones in their eyes. These are the cells that actually are dying in our p
But some of the downstream neurons actually remain alive for quite some time and our molecule is able to take one of these specific ones called retinal ganglion cells and make them light sensitive.
So we ran a phase one B clinical trial. It was a first in human study.
That had occurred over the course of basically 2023.
And what we did is we injected into vitrially which is a very common and frequently used way of delivering drugs into the eye.
And tested patients who had ultra low vision or were totally blind. Does this drug first and foremost. Is it safe and tolerable any first in human study you need to look at that and that is critical, but we also assessed a host of different efficacy assessments.
Trying to understand is this drug making any meaningful difference for these patients?
And so that’s what we recently presented at the American Academy of ophthalmologies some special today for retina.
And it was incredibly well received and what we were able to show was.
patients who had very little or no vision
tended to have improvements in their ability to perceive light.
We saw improvements in their visual Fields. So kind of what is that peripheral vision look like and how much can they see coming in from the side for instance?
And also they were able to go through some what we call functional Vision tests more efficiently. So can they walk through a little course where there’s little objects and not bump into them.
So we had tested all sorts of these types of you know, ethic issue measures to see is our drug actually having a positive benefit for these patients. And obviously we are quite excited with the data that we saw and what has been presented showing that yes, it it certainly is appearing to give a benefit.
Based upon again the underlying mechanism that we understand and so then just lastly as part of our study. We also included something called functional MRI. So it’s a way to look at the region and the brain responsible for vision processing.
And see is it changing is it having more electrical activity by having the drug on board and it’s pretty clear from that assessment as well that yes, there is lighting up of that
visual cortex certainly interesting to see the results in the abstract that you presented there. Obviously gives you a sense that there is potential with this drug candidate when it comes to the further clinical development down the line where the plans there. Yeah. So we’re gonna kick off in q1
of 2024 a proper phase two clinical trial. This will be a controlled study that and has essentially a control arm that does not receive drug or a micro dose depending on discussions with the FDA that we’re about to have.
And versus those on you know proper doses if you will of the drug.
And so this will take about a year to complete we expect with data coming out in late 24 early 25 more likely but we’re incredibly excited to move. This one’s this forward because of the fact that we are seeing some benefits and now it’s a matter of can we confirm that the benefits that we’ve already seen?
Actually will translate that above any you know, what would be typically known as a
placebo response. I’m glad they’re having this conversation obviously about the clinical results and the abstract that you presented but also the fact that you’re organization just released its latest earnings report and you had said in there that in the financial situation that your Runway has been extended into May of 2024. Curious what the go forward on an operational basis looks like for your company is it you know, is it positioned around this drug candidate? I’m kind of curious what the next few months look like.
Yeah. So as as most Pharma companies are sizeable say, you know, we have a bunch of great assets and we’re really excited about all of them. I will be the first person to say we have a favorite child parents aren’t supposed to say that we but we do and so that one certainly is around this drug candidate and we are dedicating the majority of our resources to this drug candidate and to this upcoming clinical trial. We do have Capital until May of 24.
Um, we have some options and opportunities to extend that and so we certainly will ensure that this drug gets its best shot on goal and that our job of ensuring the capital is there behind it we’ll
be taking care of I am curious your thoughts being a leader of a company of this size. I mean, we have a lot of companies that come on here that are I guess you would say maybe more of the Blue Chip pharmas and that’s obviously not a consideration, but they are mindful of the macroeconomic environment that we’re in you know issues around inflation and having access to Capital and things of that nature. What is the most critical thing for you is you’re heading into 24 with the company. What are you seeing in the environment? That’s you know things to navigate or maybe an opportunities.
Yeah, look the reality is I would love to see a little bit more risk turn on it’s it’s in pretty heavily in the offsetting because of those interest rates and where you know, if you look at the PE Shops and and some of the hedge funds where they’re currently putting their money today and some of the safety around those interest rates that being said there’s a lot of different ways of ensuring that there’s Capital available for for companies like us to continue to develop our programs. A lot of that is non-diluted frankly and especially with the recent data. You can imagine that there are strategic Blue Chip companies that have interest in what we’re doing. So, I think there’s a lot on the table today for us to consider. We also do have what’s called an e-lock or an equity line of credit available to us that can continue to draw upon if we choose to and that can provide a relatively lower cost of capital for US versus some of the bigger more dilutive efforts which while they’re on the table are just frankly
kind of going back to the clinical side for a second. I’m curious, you know, because I think a lot of people in our audience they obviously, you know, they may represent brands that are doing something in the Vision Care space, but may not know as much about orphan retinal disease when it comes to having a better sense of that space What are some of the most critical things to remember or maybe account for when it’s talking about the issues that may be patients face or providers face in terms of trying to treat these patients.
Yeah, so again.
We have to keep in mind these patients really have nothing available to them today. They get diagnosed with these inherited retinal diseases and with one very small exception being a gene therapy that was approved called Lux Turner the vast majority of these patients are just preparing to live life in the dark and that to me is unacceptable.
So when you think about you know Market access and payers and how they’re going to think about this if our drug or any drug for that matter is able to provide patience with clinically meaningful improvements in their day-to-day living.
That is something that we think will get across the finish line and we’ll certainly get covered. Now. Here’s where we differentiate versus a lot of our competitors is that we are not a gene therapy. We are a small molecule which means we don’t need to charge millions of dollars for a treatment that everyone thinks is a one and done or at least say that it is but the reality is it’s not really necessarily proving out that way per se and so with our small molecule approach.
We could even price it at levels as low as some of the mass Market drugs today and still see a very attractive opportunity from a company perspective while obviously helping patients who are in utmost need so we do see that there’s a lot more flexibility with our approach and the interesting part is a lot of Gene therapies, you know, being that they are highly inflammatory and and you’re modifying genomes. They’re considered to be that they’re not upgradable as new technologies come available. If someone got a million plus dollar gene therapy the probability that they’re going to be eligible for a new or another therapy. It’s gonna be pretty low.
whereas for our small molecule approach
the body naturally clears out small molecules our current formulation. We think we have about a monthly durability and we think we can extend that with some other technologies that are out there already.
But the reality then comes into play that a patient who’s been on our drug maybe for a year maybe for five years.
They just wait a few months. Let it wash out. They’re back to Baseline and can then be eligible potentially for another therapy? I think it’s interesting that you bring up the gene therapy aspect because
I talk to a lot of leaders in the industry and obviously they see the potential with Gene therapies. But again, it always comes down to the cost and you highlight the access challenges. They are not only for the patients but then certainly payers have had a lot to say when it comes to Gene therapies across the board too and that does limit, you know, what options patients ultimately have available for treatments for you know, retinol diseases like this that maybe they don’t have any other
options to go to. Yeah and look. I’m right there as excited as anybody else about Gene therapies. I hope that they do come across the finish line and help the patients in need another way to think about some of the positioning of our molecules are although you know, maybe we could for a sub class of Gene therapies called optogenetics, you know, maybe we’re the first past through it that a patient has to go through to show that yes, they can respond to essentially turning Vision back on.
And before they’re eligible for a gene therapy. So I think there’s a lot of ways to commercially position technology like ours, but at the end of the day, we need to find a way to help these patients and that’s really what it all comes down to.
I want to wrap up our conversation here Brian with a question about just the feedback that maybe you’ve had from the patient and the caregiver Community around these retinal diseases because I know that I’ve seen certainly this year and maybe I’ve just been more mindful of it a lot of ads for different therapies or treatments for vision loss and being able to restore Vision. It’s one of those things where you think about it’s like we only have five sentences in that one seems to really stick out more than others. Can you just talk about maybe the feedback that you as an organization have had from the patient community that you’re trying to work with here?
Yeah, I mean there’s a lot of Hope and I think that’s first and foremost. This is a motivated patient population and they are willing to participate in things like clinical research because they are hoping for a cure and a lot of these diseases. They know that they may be handed down to children or grandchildren. And so it’s one thing if you’re affected you can kind of tend to cope with it. But knowing if you passed it on to a loved one, you’re willing to participate in the more clinical research to see if you can make sure that whatever you’re testing can help your loved ones who maybe have these diseases as well. So we are doing everything we can to make sure that we understand our patients. We talk with all of the patients in our clinical studies and we get them to actually record testimonials which are some are on our website.
That I think are incredibly encouraging at this point. But again, we still have a bit of a journey ahead of us. It’ll probably be a few years before we think that we’re even close to to getting this product to Market. But if things continue on the on the path that we’re on we think based on some of the early signs already that we will really be able to help these patients. I I lied when I said I had one last question. This is my
last question you talk about kind of the long process there and I know that it’s not for the faint of heart. I know that a lot of people want to see instant results. They want to see things turned over when it comes to the patient’s game. Maybe you can talk about this from a leadership perspective. I mean what goes into that some people? I I it’s not like the covid vaccines. It’s not like it’s something that we can just do operation warp speed some of these things take a lot of time and you know, I can imagine that you as a leader have to say like Hey, we’re going through our due process and we we have to see it through.
Yeah, look, we’re not Cowboys. We we got to make sure that we do things in an ethical fashion as we advance our programs and clearly in compliance with all of the regulatory bodies globally. So that that’s first and foremost. Now that being said because there is so much unmet need here. We do think that there could be accelerated Pathways and we’re literally on a journey right now that if things go well with our current and continued clinical testing, you know, we could see bringing this drug to Market in the 2027 2028 timeframe. So it’s really not that far away versus again. Nothing against my gene therapy colleagues, but we know that they have a lot longer requirements on their safety and efficacy follow-ups that are being put in front of them from those regulatory bodies.
Absolutely. Well certainly wish you and your organization the best of luck. I really appreciate you coming on here to talk about the drug candidate talk about the disease state that you’re working in and we’ll be curious to see any sort of clinical.
Results in the months and years ahead. I really appreciate you having me Jack great to chat with you today.
Health Policy Update with Lecia Bushak.
Republican talk of repealing Obamacare, or the Affordable Care Act, has largely quieted down in recent years – and policy wonks previously agreed that it probably wouldn’t be a big priority for GOP candidates heading into the 2024 elections.
But over the weekend, former President Donald Trump sought to revive the debate – and claimed that he would “seriously look at alternatives” to replace the landmark healthcare bill if he’s the Republican nominee for president.
In a Truth Social post, Trump said that QUOTE “The cost of Obamacare is out of control, plus, it’s not good healthcare. I’m seriously looking at alternatives. We had a couple of Republican Senators who campaigned for 6 years against it, and then raised their hands not to terminate it. It was a low point for the Republican party, but we should never give up.”
Trump’s Truth Social post included a recent Wall Street Journal editorial that claims the ACA has increased healthcare prices.
But whether the old push to repeal the ACA actually takes off is up in the air. After all, repeated attempts in 2017 to repeal the law failed, with the late Sen. John McCain siding with Democrats in a vote to keep the law in place.
Plus, it appears the majority of Americans are fine with the law. According to a Kaiser Family Foundation poll, nearly 60% of Americans feel favorably toward the ACA, compared to 40% who don’t.
Biden campaign spokesperson Ammar Moussa has posted on X over the last few days in response to Trump’s comments, retweeting a Protect Our Care tweet that says QUOTE “repealing the ACA means ripping away protections for the over 135 million Americans with pre-existing conditions, hiking premium costs for millions… and putting insurance companies back in charge.”
Moussa confirmed on X that the Biden campaign is happy to discuss the ACA if Trump succeeds in reviving the full-blown debate. I’m Lecia Bushak, Senior Reporter at MM+M.
And this is the part of the broadcast when we welcome Jack O’Brien to tell us what’s trending on healthcare social media.
Hey Marc, a few weeks ago, we talked about the FDA issuing warnings about contaminated eyedrops and this week, we’re talking about the FDA’s decision to recall cinnamon-flavored applesauce pouches linked to lead poisoning symptoms affecting more than 50 children.
The agency recalled products sold under the WanaBana, Schnucks and Weis brands.
Concerns over the presence of lead in applesauce products had been rising throughout the month and this latest action marks an expansion to an earlier recall that focused on WanaBana products.
Children in more than 20 states and ranging from the ages of 1 to 4 have been sickened by lead poisoning that the FDA believes comes from the cinnamon in the pouches.
Of note, the agency has warned that some contaminated products are still available on shelves in multiple states and has urged the manufacturers to conduct an effective recall to protect consumers.
Now I’m going to throw it over to you mark given that you are the one that has had children of the three of us. And obviously we have talked about concerns related to Children a lot of them being in sort of a you know, talking about social media and the impact on their mental health, but this is one that actually has a physical implication there and I imagine it’s a concern for a lot of Americans Nationwide if it’s oh I’m just buying this product for my children and all sudden you have this warning that there’s a significant amount of lead in a product that you know would seem otherwise harmless.
Yes. This is a rather disturbing news, especially in light of my Thanksgiving kitchen Follies and but also, you know, seriously because of you know, I have kids and you know, you never want to expose kids infants which was the case with one of the moms here. She was apparently transitioning her baby from you know, breast milk over to solid foods and you know using the wanna banana wanna banana, excuse me, apple cinnamon fruit puree pouches to do that and
Have been marketed, you know, obviously as a healthy option preservative free kosher gluten-free, you know, she was obviously, you know, disappointed after you know, discovering the the link to lead and is now bringing suit against the company for alleged injuries caused by this apple puree. So it’s all the more so disturbing given the fact that this food is, you know, marketed toward the youngest people who are you know, you know kids and pediatric age who are the most vulnerable. So it’s really, you know, really makes it more of a dangerous situation.
Yeah. I know that we’ve covered, you know, some of these recalls in the past and I feel like you know, it always reignites my sort of like hypochondriac slash contamination fear part of my brain. Oh new fear on locked apple sauce pouches, apparently our dangerous, but you know, it’s usually in these situations fortunately. They typically are limited to you know, a small amount of people who’ve been affected and they usually able to catch it pretty quickly and
Release the recall so glad they were able to do that with this one.
And I think another important thing to bring up here is you know, we’ve talked a lot on the show the lack of effective messaging and communication from some of the federal agencies that oversee Healthcare in this country the FDA and I was looking through as we were about to include the story in the podcast for weeks has been, you know, advising the public and updating them on where they stand with this because again kind of going back to the eyedrop thing. We talked about a few weeks ago. If you don’t know as a consumer, if you’re not informed you can make a decision that could inadvertently impact your health in a very negative way. So Props the FDA. I hope certainly other agencies take note of that going forward in terms of their messaging.
Earlier this month, New York Governor Kathy Hochul tapped media personality and famed sex expert Dr. Ruth Westheimer to serve as the state’s honorary ambassador to loneliness, a role that’s the first of its kind in the nation.
Hochul said the decision was made as the Empire State, like the nation at large, battles the growing public health challenges stemming from social isolation and loneliness. New York is aiming to build “age-friendly communities” and a “robust system of mental healthcare.”
In the press release accompanying the announcement, Hochul attributed the idea to create the loneliness ambassadorship to Dr. Ruth, noting that she approached her office with the concept last year as the state reeled from the effects of the COVID-19 pandemic.
Dr. Ruth stated: “Hallelujah! I am deeply honored and promised the Governor that I will work day and night to help New Yorkers feel less lonely.”
less. I know you’ve covered in depth in your columns certainly as it relates to the federal level with the work of Surgeon General Vivek Murthy, but I’m curious your thoughts on this move by the state of New York.
Yeah. I think it’s it’s definitely a cool concept and something that’s clearly still in the early works, you know, this idea that loneliness is a public health issue and that we need new solutions for addressing. It is pretty recent one. It was just this year that Murphy brought the loneliness epidemic to the Public’s attention and there are other lawmakers as well who are making it a priority in health policy. Obviously. This was all driven by covid and we’re still seeing the effects of isolation from covid extending into 2023 and likely moving forward in the next years, but I’m really curious to see what Westheimer actually does with the new role. There was a, New York.
Article that came out about it where she said that, you know, she was going to be drawing on some of her own experiences with loneliness throughout her life specifically her diary writing she did when she was a teenager when she was separated from her family due to the Holocaust during World War two and you know, one of the things that she notes in that article is that the key to battling loneliness and isolation is to continually try new projects help others and stay busy, but not just any type of business. She says it should be meaningful business. So I’m definitely excited to see what she actually does with the role and you know how she develops some solutions that we can take away.
Yeah, it’s great Point slash and I really enjoyed your peace.
about you know, the Vic murthy’s
Statement about the loneliness as an epidemic and you know, we’ve seen some efforts like, you know scan Health, which is one of the Medicare Advantage plans based on the west coast, you know launching a togetherness program which you know use senior to senior interactions both in-person virtually to combat this this problem, but you know in the coverage here involving Dr. Ruth, you know, she underscores that it’s a national epidemic among all ages. And so, you know, I think it’s it’s great. This is bringing more attention to the problem. It’s obviously as you mentioned propelled by the pandemic and everybody, you know, I think seniors included but other age groups are also affected by it. So it’s something that will resonate I think, you know across the populations band.
Our last story is a sad one with a bright silver lining.
Casey McIntyre, a New York City woman who died from cancer a few weeks ago, has raised enough money to erase millions of dollars in medical debt with a posthumous plea for help.
In a selfless act to help others, McIntyre and her husband arranged to buy the medical debt of complete strangers in order to celebrate her life.
By working with the nonprofit RIP Medical Debt, McIntyre posted a farewell message on X, the social media platform formerly known as Twitter, with a link to the fundraising campaign.
“a note to my friends: if you’re reading this I have passed away. I’m so sorry, it’s horseshit and we both know it. The cause was stage four ovarian cancer,” she wrote. “I loved each and every one of you with my whole heart and I promise you, I knew how deeply I was loved.”
Though the initial goal was to raise $20,000, the campaign ultimately raised $140,000, which is enough to eradicate around $14 million in medical debt.
Yeah. This is another one of those examples where we’re seeing patients or regular ordinary people or YouTube influencers raising money to help people afford basic health care and help people with medical debt. I know we did a couple segments about Mr. Beast and him raising money to allow people to get basic care like dental care and things like that I care and this is another example of in the absence of you know, a system that provides Affordable Health Care patience and ordinary people have to sort of fill in that Gap and help others get
Care they need so it’s a positive story in that sense. But definitely highlights some of the the Brokenness of the healthcare system as we’ve highlighted before.
Yeah, I thought it was a very cool story as well. You know when I read it and you know that this woman is posthumously decided to wipe out other people’s medical debt as a sort of a partying gift and she you know, certainly, you know beyond the grave is helping out a lot of people here, but it does underscore certainly the problem of unresolved medical debt and how it’s such a big issue, you know for so many people in this country who are struggling under the weight of you know, medical debt and many times in many cases, which is surprise medical debt and a lot of those stories are just coming to the surface as we see great story.
Thanks for joining us on this week’s episode of the MM+M Podcast. Be sure to listen to next week’s episode when Jack and I will talk with MM+M Editor-in-Chief Larry Dobrow about the release of our upcoming Best Places to Work rankings.