WEBVTT
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Thank you so much for your time today. So my
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name is Lukasz Jaszyna. I work for Leo Pharma here
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in the US. I oversee our rare disease team that
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we've been building now at Leo for over a year.
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It's a really exciting project that we have started here
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at Leo over a year ago. Leo is a really
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is a company with history, over 60 years of history
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and expertise in dermatology. And over the past year, Leo
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has been also growing in rare skin disease using this
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dermatology expertise to help rare disease patients. And over the
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past 12 months, we have completed three acquisitions in rare
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skin diseases to help those patients with huge unmet need.
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And the first one was Spivigo, the partnership with Behringer Ingelheim,
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that we are also about to actually celebrate one year
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anniversary because that closed on, we officially acquired Spivigo end
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of September of last year.
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Earlier this year, we acquired.
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A gene therapy, a topical gene therapy platform from a
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company called Replay. And just now, today, we announced that
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we closed the acquisition of another potential product that is
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right now being reviewed by FDA for the treatment of
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EPP and XLP. So if you look at this, you know,
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the commitment that Leo has to those patients and the
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growth that we're seeing, especially here in the US with
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those products that we're bringing, it's something extraordinary. And, you know,
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I'm just really happy to be part of this team
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and have an opportunity to lead this great team as
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we go on this journey.
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So to start off, could you provide an overview of
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EPP and XLP? What are the common symptoms, current treatment options,
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and what that disease burden looks like for patients?
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Yeah, thanks for this question. And here at Leo, we
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always start with patients and their caregivers. As you will learn,
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EPP and XLP, the first symptoms often start in childhood.
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So people living with EPP and XLP, they face an
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extreme burden.
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There is limited treatment options. It's devastating.
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It's a lifelong disease and it can severely impact patient's
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ability to live every day. Their everyday life is free
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from sunlight-induced pain. And that's the biggest thing, that those patients,
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when they go in the sun, it really starts impacting
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their quality of life.
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Talking to patients... they.
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Often refer to it as burning sensations because of this
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risk of severe recurrent phototoxic pain triggered by everyday light exposure.
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It drives them to, over time, to have this lifelong
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daylight avoidance. So there is a profound quality of life loss,
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including high rates of depression, anxiety, and social isolation. On
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top of the actual skin rashes and blistering, But again,
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I think the one thing that talking to patients that
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really touched me was that burning sensations and often those,
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especially kids, not being able to articulate what's happening. And
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one of the biggest unmet needs, I would say, in
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this disease state is the diagnosis part. So if you
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look at average time to diagnosis for EPP patients, and
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actually before I even say that, one of the statistics
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that I'm sure you know it's always rare disease companies,
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rare disease communities. We always talk about the diagnostic odyssey.
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And it's around five years on average that it takes
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a rare disease patient to get diagnosed with a rare disease.
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And it was shocking to me to learn that in
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EPP and XLP, this diagnostic odyssey can be anywhere between
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10 to 15 years. And, you know, like if you
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think about this, you're, Your everyday activities, especially, you know,
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as a caregiver, as a parent of a child, go
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out there, the child is in pain, they cannot articulate
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what's happening. You go from one physician to another physician,
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you get referred. And this is something that's really, really,
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really difficult and something that I hope we'll be able
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to address.
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So can you describe the mechanism of action of Dersimiligin,
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how it relates to the pathophysiologies of both of those diseases?
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Yes, happy to.
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And, you know, first, and I will always start with that,
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this is not an approved treatment.
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It is being reviewed by FDA. Incredibly exciting.
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We announced this morning that FDA did grant the priority review,
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accepted the filing and granted the priority review. And what's
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even more, it's a coincidence, but hey, why not? The
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PDUFA date coincides actually with the Rare Skin Disease Day
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next year.
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So Rare Disease Day next year. So it's kind of exciting,
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you know.
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To be able to potentially, if approved, celebrate both Rare
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Disease Day and the approval of this potential new treatment.
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So if approved, nursing malagon is a first in-class oral,
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once daily MC1 agonist for those patients with EPP and XLP.
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And the way it works is it increases humanly-mediated photoprotection.
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And through this, it extends sunlight exposure and decreases pain events.
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So it raises the light dose that's required to photoactivate
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protoporphyrin 9, which is then ultimately, through accumulation, causes those
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phototoxic events.
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So I said a lot of things here right now.
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I know it's a bit scientific. I'm happy to.
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I'm sure someone can share some specific wording around this,
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but it's basically addressing that production of... It increases this
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protection from the daylight, from the visible sunlight that allows
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those patients to be able to spend more time outside
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and really helps them from potential pain events that we
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hear are so, so crucial and devastating on everyday basis.
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But again, if approved, no promotional claims, right?
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Can you summarize some of the phase three data that
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the FDA was just reviewing that led to the NDA
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being approved today and the fast track designation?
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Again, as you said, the FDA accepted the fire today.
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Actually, right now, together with a partner, Tanabe, we're working
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to prepare the publication.
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Of the phase three data later this year.
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Basically the key thing that the data showed, it was
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a phase three study that was designed, it was a
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really robust phase three study, placebo arm as well. And
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what the data showed, the primary endpoint showed that patients
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using this potential treatment were able to stay longer outside
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before having.
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Those pain events.
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So what the company measured was the onset to the
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first symptoms and the prodromal symptoms. And so what we
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were able to show, the company that was running the study,
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was that patients using this potential treatment were able to
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stay significantly longer outside in the sunlight before experiencing heartburn.
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Those first symptoms.
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And as I said, we're working right now on the
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publication and looking forward to being able to publish it
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in one of the journals in the near future.
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Can you talk about Leo Pharma's commitment to rare dermatology
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and rare skin diseases?
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Where do I start?
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I mean, look, I think the key thing that, and
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I mentioned this at the beginning, first of all, Leo
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Pharma is not a new game in We've been out
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there in dermatology. At Leo Pharma, actually, we've been out
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there for over 100 years, but in dermatology for over
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six decades. And I think that's something that makes Leo
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Pharma very special in this space because the expertise that
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we have built globally in dermatology really makes the teams
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that we have, the scientific teams, the R & D
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teams that we have can really help us find the
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best solutions for patients with dermatological diseases. And we are
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a leader in medical dermatology. And here in the US,
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we've been really growing over the past years by bringing
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new products to the market and launching new products also
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in the broader medical dermatology, like Anzupco for chronic anexoma
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last year.
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And then again, by.
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Taking this expertise, taking our footprint in the dermatological space,
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in medical dermatology, we were able to take it and
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translate it into what can we do to help rare
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skin disease patients. Because if you look at it, it's
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something that actually excited me to join this company.
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Let me tell you a story.
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I joined this company now a year ago, almost exactly
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a year ago. I just celebrated my one year anniversary
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at the leading rare disease company, Alexion. And I spent
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over 10 years at Alexion. I think what we have
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built and what Alexion continues to build now with AstraZeneca
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is a really great platform for rare diseases. But what
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really attracted me to join Leo and what I saw
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in the story here is there's no company that said, hey,
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let's solve the problems of rare skin disease patients.
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And it's not easy. It's incredibly complex.
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It's complex journeys, like in any rare disease, but specifically
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in rare skin disease, there wasn't any company and a
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player that said, okay, hey, let's try to work on this.
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There is a few companies here and they're trying to,
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we're having some products. But what we're trying to do
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is build this really footprint framework and a broader strategy
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to be able to serve those patients that don't have
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any solutions today. there is approximately 30% of skin diseases
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are rare. So if you think about it, 30% of
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skin diseases are rare. Now, if you look at the
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pipeline in dermatology, only around 1.5% of the dermatology pipeline
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actually addresses diseases that are rare. So I think it's
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a huge transmit need, but I also think it's a
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great potential to be able to step into this space
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and help. And because we have the six decades of
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experience in dermatology, we know, Leo knows what we need
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to do. We know, we know, we understand skin and
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it's really about bringing that heritage and experience, bringing the
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rare disease experience from other places as well, connecting those
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two and continuing to build. And I think in terms
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of our commitment, if you look at what we have
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done over the past 12 months and now having really
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three assets, One of them already approved, Spevigo for GPP
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and being studied for other indications. Replay, the topical gene
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therapy I mentioned. And the third one now, there's Himalagon
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for potential treatment for EPP and XLP. That's our commitment
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and that's our commitment to those patients.
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Going forward, what are some of those unmet needs and
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areas of opportunity in rare skin?
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So there's over 800 rare skin diseases, most of them
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with no approved treatment. We often quote these statistics, I'm
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sure you know, but 95% of rare diseases don't have
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an FDA-approved treatment. And unfortunately, the story is no different
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for rare skin diseases. So as we continue growing and
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as we continue expanding our portfolio, We're looking where we
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can make the biggest difference in patients' lives, where is
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the biggest unmet need, and also where the science is.
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We have, again, as I mentioned, we have six decades
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of dermatology experience. We have some really amazing world-class dermatology
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expert scientists who are helping us bring the science and
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understand where we have the highest probability of success to
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actually be able to help those patients. So we're continuing
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on this journey 800 to go. It's going to take
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a while, but we're continuing to identify where can we
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make the biggest difference. And as I said, we are,
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of course, excited about the potential to commercialize this treatment.
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But we're also building the pipeline. Spavigo is in phase
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three for treatment of PG, pyoderma gangrenosum, another devastating disease.
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We will see what the data shows. And we are
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really accelerating the development of the topical gene therapy that
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I mentioned.
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And we're not stopping here. We'll go beyond because those
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patients need us.
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Is there anything you wanted to add or anything you
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felt I missed in my questions?
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Incredibly excited by the news this morning. The team is
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working incredibly hard.
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To prepare for this potential launch coinciding with the World
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Rare Disease Day. And it's just exciting to see the
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growth that we can bring, not just in terms of
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what we're doing here in the US, but also the
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growth of the team. We are adding new resources, we
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are adding new people to continue building this story. of
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how we can serve patients with rare skin diseases.
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So thank you. It was some really great questions. I
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appreciate your time this morning.
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Thank you so much for your time today. So my
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name is Lukasz Jaszyna. I work for Leo Pharma here
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in the US. I oversee our rare disease team that
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we've been building now at Leo for over a year.
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It's a really exciting project that we have started here
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at Leo over a year ago. Leo is a really
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is a company with history, over 60 years of history
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and expertise in dermatology. And over the past year, Leo
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has been also growing in rare skin disease using this
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dermatology expertise to help rare disease patients. And over the
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past 12 months, we have completed three acquisitions in rare
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skin diseases to help those patients with huge unmet need.
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And the first one was Spivigo, the partnership with Behringer Ingelheim,
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that we are also about to actually celebrate one year
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anniversary because that closed on, we officially acquired Spivigo end
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of September of last year.
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Earlier this year, we acquired.
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A gene therapy, a topical gene therapy platform from a
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company called Replay. And just now, today, we announced that
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we closed the acquisition of another potential product that is
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right now being reviewed by FDA for the treatment of
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EPP and XLP. So if you look at this, you know,
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the commitment that Leo has to those patients and the
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growth that we're seeing, especially here in the US with
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those products that we're bringing, it's something extraordinary. And, you know,
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I'm just really happy to be part of this team
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and have an opportunity to lead this great team as
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we go on this journey.
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So to start off, could you provide an overview of
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EPP and XLP? What are the common symptoms, current treatment options,
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and what that disease burden looks like for patients?
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Yeah, thanks for this question. And here at Leo, we
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always start with patients and their caregivers. As you will learn,
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EPP and XLP, the first symptoms often start in childhood.
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So people living with EPP and XLP, they face an
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extreme burden.
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There is limited treatment options. It's devastating.
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It's a lifelong disease and it can severely impact patient's
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ability to live every day. Their everyday life is free
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from sunlight-induced pain. And that's the biggest thing, that those patients,
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when they go in the sun, it really starts impacting
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their quality of life.
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Talking to patients... they.
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Often refer to it as burning sensations because of this
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risk of severe recurrent phototoxic pain triggered by everyday light exposure.
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It drives them to, over time, to have this lifelong
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daylight avoidance. So there is a profound quality of life loss,
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including high rates of depression, anxiety, and social isolation. On
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top of the actual skin rashes and blistering, But again,
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I think the one thing that talking to patients that
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really touched me was that burning sensations and often those,
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especially kids, not being able to articulate what's happening. And
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one of the biggest unmet needs, I would say, in
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this disease state is the diagnosis part. So if you
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look at average time to diagnosis for EPP patients, and
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actually before I even say that, one of the statistics
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that I'm sure you know it's always rare disease companies,
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rare disease communities. We always talk about the diagnostic odyssey.
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And it's around five years on average that it takes
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a rare disease patient to get diagnosed with a rare disease.
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And it was shocking to me to learn that in
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EPP and XLP, this diagnostic odyssey can be anywhere between
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10 to 15 years. And, you know, like if you
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think about this, you're, Your everyday activities, especially, you know,
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as a caregiver, as a parent of a child, go
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out there, the child is in pain, they cannot articulate
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what's happening. You go from one physician to another physician,
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you get referred. And this is something that's really, really,
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really difficult and something that I hope we'll be able
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to address.
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So can you describe the mechanism of action of Dersimiligin,
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how it relates to the pathophysiologies of both of those diseases?
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Yes, happy to.
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And, you know, first, and I will always start with that,
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this is not an approved treatment.
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It is being reviewed by FDA. Incredibly exciting.
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We announced this morning that FDA did grant the priority review,
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accepted the filing and granted the priority review. And what's
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even more, it's a coincidence, but hey, why not? The
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PDUFA date coincides actually with the Rare Skin Disease Day
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next year.
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So Rare Disease Day next year. So it's kind of exciting,
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you know.
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To be able to potentially, if approved, celebrate both Rare
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Disease Day and the approval of this potential new treatment.
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So if approved, nursing malagon is a first in-class oral,
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once daily MC1 agonist for those patients with EPP and XLP.
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And the way it works is it increases humanly-mediated photoprotection.
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And through this, it extends sunlight exposure and decreases pain events.
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So it raises the light dose that's required to photoactivate
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protoporphyrin 9, which is then ultimately, through accumulation, causes those
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phototoxic events.
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So I said a lot of things here right now.
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I know it's a bit scientific. I'm happy to.
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I'm sure someone can share some specific wording around this,
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but it's basically addressing that production of... It increases this
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protection from the daylight, from the visible sunlight that allows
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those patients to be able to spend more time outside
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and really helps them from potential pain events that we
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hear are so, so crucial and devastating on everyday basis.
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But again, if approved, no promotional claims, right?
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Can you summarize some of the phase three data that
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the FDA was just reviewing that led to the NDA
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being approved today and the fast track designation?
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Again, as you said, the FDA accepted the fire today.
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Actually, right now, together with a partner, Tanabe, we're working
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to prepare the publication.
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Of the phase three data later this year.
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Basically the key thing that the data showed, it was
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a phase three study that was designed, it was a
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really robust phase three study, placebo arm as well. And
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what the data showed, the primary endpoint showed that patients
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using this potential treatment were able to stay longer outside
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before having.
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Those pain events.
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So what the company measured was the onset to the
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first symptoms and the prodromal symptoms. And so what we
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were able to show, the company that was running the study,
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was that patients using this potential treatment were able to
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stay significantly longer outside in the sunlight before experiencing heartburn.
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Those first symptoms.
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And as I said, we're working right now on the
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publication and looking forward to being able to publish it
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in one of the journals in the near future.
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Can you talk about Leo Pharma's commitment to rare dermatology
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and rare skin diseases?
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Where do I start?
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I mean, look, I think the key thing that, and
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I mentioned this at the beginning, first of all, Leo
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Pharma is not a new game in We've been out
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there in dermatology. At Leo Pharma, actually, we've been out
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there for over 100 years, but in dermatology for over
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six decades. And I think that's something that makes Leo
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Pharma very special in this space because the expertise that
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we have built globally in dermatology really makes the teams
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that we have, the scientific teams, the R & D
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teams that we have can really help us find the
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best solutions for patients with dermatological diseases. And we are
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a leader in medical dermatology. And here in the US,
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we've been really growing over the past years by bringing
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new products to the market and launching new products also
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in the broader medical dermatology, like Anzupco for chronic anexoma
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last year.
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And then again, by.
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Taking this expertise, taking our footprint in the dermatological space,
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in medical dermatology, we were able to take it and
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translate it into what can we do to help rare
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skin disease patients. Because if you look at it, it's
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something that actually excited me to join this company.
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Let me tell you a story.
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I joined this company now a year ago, almost exactly
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a year ago. I just celebrated my one year anniversary
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at the leading rare disease company, Alexion. And I spent
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over 10 years at Alexion. I think what we have
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built and what Alexion continues to build now with AstraZeneca
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is a really great platform for rare diseases. But what
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really attracted me to join Leo and what I saw
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in the story here is there's no company that said, hey,
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let's solve the problems of rare skin disease patients.
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And it's not easy. It's incredibly complex.
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It's complex journeys, like in any rare disease, but specifically
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in rare skin disease, there wasn't any company and a
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player that said, okay, hey, let's try to work on this.
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There is a few companies here and they're trying to,
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we're having some products. But what we're trying to do
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is build this really footprint framework and a broader strategy
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to be able to serve those patients that don't have
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any solutions today. there is approximately 30% of skin diseases
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are rare. So if you think about it, 30% of
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skin diseases are rare. Now, if you look at the
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pipeline in dermatology, only around 1.5% of the dermatology pipeline
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actually addresses diseases that are rare. So I think it's
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a huge transmit need, but I also think it's a
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great potential to be able to step into this space
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and help. And because we have the six decades of
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experience in dermatology, we know, Leo knows what we need
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to do. We know, we know, we understand skin and
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it's really about bringing that heritage and experience, bringing the
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rare disease experience from other places as well, connecting those
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two and continuing to build. And I think in terms
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of our commitment, if you look at what we have
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done over the past 12 months and now having really
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three assets, One of them already approved, Spevigo for GPP
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and being studied for other indications. Replay, the topical gene
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therapy I mentioned. And the third one now, there's Himalagon
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for potential treatment for EPP and XLP. That's our commitment
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and that's our commitment to those patients.
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Going forward, what are some of those unmet needs and
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areas of opportunity in rare skin?
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So there's over 800 rare skin diseases, most of them
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with no approved treatment. We often quote these statistics, I'm
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sure you know, but 95% of rare diseases don't have
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an FDA-approved treatment. And unfortunately, the story is no different
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for rare skin diseases. So as we continue growing and
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as we continue expanding our portfolio, We're looking where we
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can make the biggest difference in patients' lives, where is
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the biggest unmet need, and also where the science is.
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We have, again, as I mentioned, we have six decades
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of dermatology experience. We have some really amazing world-class dermatology
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expert scientists who are helping us bring the science and
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understand where we have the highest probability of success to
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actually be able to help those patients. So we're continuing
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on this journey 800 to go. It's going to take
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a while, but we're continuing to identify where can we
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make the biggest difference. And as I said, we are,
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of course, excited about the potential to commercialize this treatment.
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But we're also building the pipeline. Spavigo is in phase
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three for treatment of PG, pyoderma gangrenosum, another devastating disease.
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We will see what the data shows. And we are
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really accelerating the development of the topical gene therapy that
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I mentioned.
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And we're not stopping here. We'll go beyond because those
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patients need us.
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Is there anything you wanted to add or anything you
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felt I missed in my questions?
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Incredibly excited by the news this morning. The team is
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working incredibly hard.
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To prepare for this potential launch coinciding with the World
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Rare Disease Day. And it's just exciting to see the
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growth that we can bring, not just in terms of
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what we're doing here in the US, but also the
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growth of the team. We are adding new resources, we
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are adding new people to continue building this story. of
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how we can serve patients with rare skin diseases.
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So thank you. It was some really great questions. I
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appreciate your time this morning.
