Release Date: August 13, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- ZTALMY achieved profitability on a commercial investment in the first quarter of 2024, ahead of expectations.
- ZTALMY was approved in China as the first treatment for seizures associated with CDKL5 deficiency disorder (CDD) in patients aged two years and older.
- Marinus Pharmaceuticals Inc (MRNS, Financial) has established commercial collaboration agreements in Europe and China, and distribution agreements in MENA and Russia.
- The company is preparing for the upcoming Phase 3 readout in tuberous sclerosis complex (TSC) and expanding access to ZTALMY globally.
- Marinus Pharmaceuticals Inc (MRNS) generated net product revenue of $8 million in the second quarter, representing growth of over 85% compared to the same period in 2023.
Negative Points
- The company reported a net loss before income taxes of $35.8 million for the second quarter of 2024.
- Research and development expenses were $20.9 million for the second quarter, reflecting high ongoing costs.
- Selling, general, and administrative expenses increased to $16.7 million for the second quarter.
- The Phase 3 RAISE trial failed to achieve statistical significance for the second co-primary endpoint.
- Marinus Pharmaceuticals Inc (MRNS) had to initiate cost reduction plans and restructure credit agreements to extend its cash runway into the second quarter of 2025.
Q & A Highlights
Q: Can you provide more color on the new patient additions for ZTALMY in the quarter and clarify the statement about 70% of patients remaining active on the drug since launch?
A: Scott Braunstein (CEO): We've seen relatively steady growth in new patient adds every quarter. The persistence of effect has been strong, with more than 70% of patients remaining on long-term therapy. Lisa Lejuwaan (SVP): We are proud of the durability of ZTALMY, with a less than 30% discontinuation rate, indicating effective seizure reduction and minimal side effects.
Q: Can you discuss the subgroup analyses from the Phase 2 open-label study that give you confidence in the Phase 3 study for TSC?
A: Scott Braunstein (CEO): We have over 100 patients in the open-label extension, with a high percentage completing the study. Joseph Hulihan (CMO): Patients with focal seizure types had a better response, and those on cannabidiol and everolimus also showed good responses. The revised titration schedule has reduced discontinuation rates significantly.
Q: How does the higher baseline seizure frequency and number of prior therapies failed in the Phase 3 study impact your expectations?
A: Scott Braunstein (CEO): Higher baseline seizure frequency helps minimize placebo rates. We ensured disease stability at baseline to avoid regression to the mean. Joseph Hulihan (CMO): Eliminating patients with very low seizure numbers reduces variability. The lower titration rate and reduced somnolence also give us optimism about efficacy.
Q: What is the timing for the type C meeting with the FDA regarding the IV program, and what scenarios could come out of it?
A: Scott Braunstein (CEO): We plan to submit the filing by the end of the month, with a typical 90-day review period. The goal is to realign on endpoints that are more objective and discuss study design and patient stratification with the FDA.
Q: Can you elaborate on the milestones you are eligible for this year?
A: Steven Pfanstiel (CFO): The nearest term milestone is a EUR10 million payment from Orion upon commercialization of CDD. There are similar payments associated with TSC commercialization and additional payments from our partnership with Tenacia in China.
Q: What proportion of patients today have a CDD diagnosis, and how are you identifying additional patients?
A: Scott Braunstein (CEO): We have good line of sight on roughly 2,000 patients with a CDD diagnosis. Lisa Lejuwaan (SVP): We have shifted to a more data-driven approach, leveraging increased genetic testing and ICD-10 code usage to identify more patients.
Q: How are you preparing for manufacturing capacity to meet the expected demand for TSC by the end of 2025?
A: Scott Braunstein (CEO): We feel confident in our current manufacturing capabilities and have plans to expand capacity. We are making investments to ensure we can meet demand by 2027 and beyond, including establishing a second manufacturing site.
Q: Are you seeing any discontinuations in the long-term extension for TSC?
A: Scott Braunstein (CEO): We are encouraged by the durability of the drug across disease states. We will present long-term extension data at AES and share top-line data at our Analyst Day in September.
Q: Can you discuss any off-label use of ganaxolone?
A: Lisa Lejuwaan (SVP): About 15% of patients on therapy do not have a CDD diagnosis but are affected by other DEEs. Most payers are covering the drug for these patients, acknowledging the significant unmet need.
Q: What are your expectations for the TSC market, and how are you preparing for the launch?
A: Scott Braunstein (CEO): We believe the TSC market is at least 5 to 10 times larger than the CDD market. We are preparing for a robust launch with strong volumes expected in the US, Europe, and China. We are also making investments to expand our manufacturing capacity to meet this demand.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
