Zevra Therapeutics, Inc. Common Stock Canaccord Genuity's 46th Annual Growth Conference
Review the key takeaways and the transcript of this earnings call.
- Zevra Therapeutics reported a very good quarter following their recent earnings announcement.
- The company has 184 prescription enrollment forms for Miplyffa in the US to date, representing the majority of the 300 to 350 diagnosed and treated Niemann-Pick type C (NPC) patients, with newly diagnosed patients increasing each quarter.
- The US market for Miplyffa is expanding beyond the previously estimated 300 to 350 patients towards a potential total addressable market of 900 patients.
- Miplyffa is approved in the US for NPC in combination with Miglustat, and the average monthly price per patient is approximately $80,000 to $85,000.
- The company has seen a surprising number of adult NPC patients being diagnosed and treated, roughly equal to pediatric patients, which has expanded the market opportunity.
- In Europe, the Medicines Agency gave a negative opinion on Miplyffa, but Zevra has submitted a request for reexamination and is confident based on unmet need and clinical evidence.
- Zevra is advancing a Celiprolol program for vascular Ehlers-Danlos syndrome, engaging with the FDA to accelerate development and enrollment in the phase 3 trial.
- The company has $260 million in cash as of June 30th, no debt, and is focused on disciplined investment in commercial efforts and potential inorganic growth through business development of complementary ultra-rare disease assets.
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Transcript
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Good morning, everyone. I'm Sumant Kulkarni, a senior biotechnology analyst here at Canaccord Genuity, and it's my pleasure to have Zevra Therapeutics with us here today. Zevra, as you might know, has a product that's approved and on the market already for an ultra-rare disease called Niemann-Pick disease type C. The product is called MIPLYFFA, and it's been launched for some time now. We've had some interesting things back and forth with Europe as well, the European Medicines Agency there. We have a bunch of questions for you guys, and thanks for making it. For Zevra, we have CEO Neil McFarlane and CFO Justin Renz. Out there in the audience, we have Nichol Ochsner, who knows everything about the company as well. We'll keep this interactive. We do have a mic going around, so please feel free to raise your hands, and we'll get the mic across to you in case you have any questions.
With that, I'll kick it off. Neil, can you set the stage a little bit on exactly what's happening with MIPLYFFA, the unmet need that that product serves, the Niemann-Pick disease type C community, and how it's received that, and what you've done so far with the product?
Yeah. Thank you, Sumant, and thanks for having us. We'll be making some forward-looking statements, so take a look at our most recent SEC filings for the most up-to-date information. Yes, we had our earnings last week, and we ended up announcing a very good quarter. Again, we are three prongs on a stool here. We have MIPLYFFA in the U.S. that it remains our core focus on driving value for patients in combination with miglustat. We are also working through our geographic expansion strategy. We'll talk a little bit more about that in a moment, not only with our MAA process in Europe, which is going through a re-examination right now, but also in our market expansions that we're working on through named reimbursement and expanding access there.
Lastly, our celiprolol program, where we are driving with engaging with the FDA in the second half of this year, after earlier this year engagement on how we can accelerate the development of this program for patients with vascular Ehlers-Danlos syndrome. Very proud of the execution in the first half of the year, and looking forward to taking some questions and having a conversation today.
Thanks for that. You mentioned prongs on the stool. I am going to ask you to pick the highest priority prong right now. Is it MIPLYFFA in the U.S.? Is it the MIPLYFFA pending application or the resubmission that you have in to the EMA or something else entirely?
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