Release Date: August 15, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Completed enrollment for Phase 2 trial for risvodetinib in Parkinson's disease, with top-line data expected in November.
- Productive engagement with the FDA regarding IkT-001Pro for pulmonary arterial hypertension (PAH), with plans to open clinical development soon.
- Raised $4 million in May, extending cash runway into December 2024.
- Net loss decreased to $5.0 million from $5.8 million year-over-year, indicating improved financial management.
- Ongoing development of new antibody diagnostic and clinical biomarker tools for Parkinson's disease, with grant applications under review.
Negative Points
- Six participants withdrew from the Phase 2 trial for risvodetinib without completing the 12-week dosing.
- Research and development expenses decreased by $1.5 million year-over-year, potentially indicating reduced investment in new projects.
- Selling, general, and administrative expenses increased by $0.2 million, driven by higher legal and consulting fees.
- Existing cash and cash equivalents are only sufficient to fund operations into December 2024, necessitating further funding.
- Potential delays in launching the 12-month open-label extension study for risvodetinib due to financial constraints.
Q & A Highlights
Q: As far as the risvo Phase 2 study, is there going to be some lag time between when patients complete this trial and when they have the option to move to the open-label extension (OLE)? Is there the potential that they might opt for symptomatic treatment during that time?
A: Dr. Milton Werner, CEO: Yes, there may be some lag time, and some patients might opt for symptomatic treatment. However, the patient experience has been positive, and we are working to launch the OLE study soon. We are tracking the number of patients who opt for symptomatic treatments.
Q: When you think about the Phase 3 program, would that entail two studies? Would you be looking to go out beyond 3 months to 12 to 18 months targeting avoidance of use of symptomatic treatments?
A: Dr. Milton Werner, CEO: Yes, the Phase 3 program will likely involve two studies, each running for up to 12 months. We aim to evaluate risvodetinib in untreated Parkinson's disease to avoid confounding factors from symptomatic treatments.
Q: Can you provide details on the size and scope of the Phase IIb study for PAH?
A: Dr. Milton Werner, CEO: The Phase IIb study will involve roughly 100 patients across two doses and a placebo. It will include a 12-week safety review and a futility analysis at 24 weeks. The goal is to confirm the safety and efficacy of the pro-drug.
Q: What do you want to see from the top-line data in November to proceed to Phase 3 for risvo?
A: Dr. Milton Werner, CEO: We aim to see trends across assessments and potential effects on underlying protein pathology. Even small effects in 12 weeks would be encouraging and could support moving directly to Phase 3.
Q: What are your expectations for IND clearance for 001Pro? Are there any gating factors that could delay the start of the trial?
A: Dr. Milton Werner, CEO: We expect the IND to clear without issues. The main gating factors are capital and infrastructure setup. We estimate it will take 9 to 12 months to gear up the trial fully.
Q: Can you clarify the number of patients for the Phase 3 trials for risvo?
A: Dr. Milton Werner, CEO: We estimate between 300 and 400 patients to cover both trials, depending on the effect size. The final number will be determined after reviewing the unblinded data in November.
Q: Are there any specific challenges you foresee in recruiting patients for the Phase 3 trials?
A: Dr. Milton Werner, CEO: We have developed effective patient recruitment tools and do not anticipate significant challenges in recruiting the required patient population for the Phase 3 trials.
Q: How do you plan to manage the financial constraints mentioned for the OLE study?
A: Dr. Milton Werner, CEO: We have mostly worked out the financial constraints and are optimistic about launching the OLE study soon. We are also exploring alternative financing opportunities, including potential grant funding.
Q: What are the next steps for IkT-001Pro after the Phase IIb study?
A: Dr. Milton Werner, CEO: If the Phase IIb study shows positive results, we may seek conditional approval or proceed to Phase 3, depending on the overall benefit and safety profile observed.
Q: How do you plan to differentiate IkT-001Pro from generic imatinib?
A: Dr. Milton Werner, CEO: We are developing new dosage forms and scaling our manufacturing efforts to support late-stage clinical development and NDA batch requirements, in alignment with FDA feedback.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
