Release Date: August 01, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Ionis Pharmaceuticals Inc (IONS, Financial) achieved significant milestones in the first half of 2024, including the launch of new medicines and progress in their pipeline.
- The US launch of WAINUA, a co-branded medicine with AstraZeneca, is progressing well, with approvals in Canada and anticipated approvals in Europe.
- Olezarsen, a wholly-owned medicine for FCS, received priority review from the FDA with a PDUFA date set for December 19, 2024.
- The company is preparing for the launch of Donidalorsen for HAE prophylaxis, with positive Phase 3 data and plans to submit an NDA soon.
- Ionis Pharmaceuticals Inc (IONS) reported strong financial results, with revenues of $225 million in Q2 2024 and $345 million for the first half of the year, keeping them on track to achieve their financial guidance.
Negative Points
- Despite progress, Ionis Pharmaceuticals Inc (IONS) faces uncertainties and risks that could impact their actual results, as highlighted in their forward-looking statements.
- The company’s R&D expenses decreased slightly in Q2 2024 and were flat for the first half of the year, which may indicate challenges in advancing their pipeline.
- Ionis Pharmaceuticals Inc (IONS) is heavily investing in the commercialization of new medicines, which has led to a significant increase in SG&A expenses.
- The company’s revenue is expected to be slightly lower in the second half of 2024 compared to the first half, with a heavier weighting towards Q4.
- Ionis Pharmaceuticals Inc (IONS) discontinued development for ION541 targeting Ataxin 2 for ALS and IONIS-FB-LRX for geographic atrophy, indicating setbacks in their pipeline.
Q & A Highlights
Q: On ATTR-CM, there's been some debate on whether you need a certain level of TTR to have a cardioprotective effect. Have you seen anything in your own data around this theory? And do you expect any differences with the silencer approach using an all-cause mortality versus the CBD mortality composite primary endpoint? Finally, do you have the flexibility to change your primary endpoint if needed?
A: We don't believe there's any evidence of a threshold effect for TTR lowering to achieve benefit in either polyneuropathy or cardiomyopathy endpoints. We're very pleased with the magnitude of TTR reductions in our studies. Our primary endpoint is cardiovascular mortality and hospitalizations, and we also have secondary endpoints like CV mortality and all-cause mortality. We think both are very important.
Q: On the timing of the cardio TTR transform readout, is it fair to say that you're at least waiting for data at ASA in London that might have read-through to your trial?
A: We're very pleased with the way the cardio transform study is advancing. We are particularly pleased with the blinded events that we're continuing to evaluate. As the first silencer readout in this indication, we're looking forward to any additional data, which we think will be very informative for our cardio transform study.
Q: Can you talk a little bit about any early insights from the WAINUA launch and how you see that informing a potential launch strategy in cardiomyopathy?
A: We're very pleased with the early insights regarding the WAINUA launch. We're seeing patients new to treatment, switching from existing treatments, and using WAINUA as an add-on. This is a foundation year for the launch, and we think there's significant opportunity with both PN and CM patient populations. We're seeing prescribing from both neurology and cardiology, which bodes well for future indications.
Q: What's your takeaway from Roche's recent presentation of their Angelman syndrome Phase 2 data at the ASF meeting? And do you think FDA will want to apply the same pivotal endpoint to both programs?
A: We were pleased with the data Roche presented, which supported our confidence in our Phase 1, 2 data. The rank order of improvements they reported was exactly what we reported. Regarding the primary endpoint, we believe our data gives us a good idea of what we would like to see in a pivotal study, but it's still to be seen what the specific primary endpoint will be.
Q: On the back of the top-line (inaudible) data, what's your latest expectation for whether silencers in TTR cardiomyopathy will mainly be used in combination with stabilizers versus as monotherapy?
A: We believe that the data will ultimately drive this decision. We are seeing WAINUA used in combination with stabilizers for HATTR polyneuropathy, and we think that will continue. Physicians and patients will make decisions based on the profile of treatment they want, and we believe we will have the data to support combination usage.
Q: Could you talk about your launch preparation for Olezarsen in FCS? How should we expect the launch uptake and eventual opportunity in FCS?
A: We're pleased with the progress we're making. Preparation started a couple of years ago, and we've built out all the commercial functions. The expanded access program is in place, and patients from our pivotal registration studies will have the opportunity to move on to commercial drug at launch.
Q: Can you highlight what you view as the next key opportunity in your neurology pipeline after Angelman syndrome?
A: Following the Angelman Phase 3 program, we are excited about our programs for Alexander disease, chronic hepatitis B, and our pre-owned program making great strides in first-in-human experiments. We are looking forward to sharing more data in the coming months and years.
Q: For Donidalorsen, when you submit the NDA, what indication will you be looking for in the label? Are you optimistic the switch data will be included on the label?
A: We are seeking a broad indication for prophylaxis of attacks in hereditary angioedema. We believe our comprehensive pivotal program supports this. The switch data is important for informing physicians on how to safely switch patients, and we think it bodes well for inclusion in the label.
Q: Could you speak to the work you're doing on oligos with next-generation backbones and other modalities? When might these programs enter the clinic?
A: The first compound incorporating our MSPA backbone will enter the clinic for a neurology indication later this year. We also have a compound with AstraZeneca using our buy cycle program to deliver to cardiac muscle, and an internal SI RNA program for liver targets. We are making great progress in our blood-brain barrier work and expect to have molecules crossing the BBB in development early next year.
Q: Can you talk about the cardio transform enrolled population and any differences between the geographies and the follow-up from the originally enrolled population?
A: We haven't shared the demographics yet, but we are pleased with the enrollment and balance between monotherapy and combination treatment patients. We have a good mix of New York health class categories and very small dropout rates. Everything supports our decision to expand the study to include patients diagnosed earlier in their disease.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
