Release Date: August 06, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Karyopharm Therapeutics Inc (KPTI, Financial) reported a second consecutive quarter of growth in XPOVIO revenue, leading to an increase in the lower end of their revenue guidance for 2024.
- The company presented promising long-term data for selinexor in endometrial cancer, showing a median progression-free survival (PFS) of nearly 40 months in patients with TP53 wild-type pMMR tumors.
- Strong physician interest and favorable competitive environment for the Phase 3 myelofibrosis study, with positive preclinical and clinical data supporting selinexor's potential.
- Karyopharm Therapeutics Inc (KPTI) has expanded its global footprint with regulatory and reimbursement approvals for selinexor in multiple international markets, including the UK, South Korea, and China.
- The company has implemented disciplined cost management, resulting in a reduction of R&D and SG&A expense guidance for 2024, and expects to fund operations into Q1 2026.
Negative Points
- The timeline for the endometrial cancer trial has been pushed to early 2026 due to higher-than-expected screen failure rates and complexities in the molecularly targeted maintenance trial.
- Enrollment rates for the Phase 3 EMN29 trial in multiple myeloma have slowed due to increased global competitive trial activity, leading to a plan to enroll fewer participants.
- The gross-to-net discount for XPOVIO increased to 29% in Q2 2024 from 22% in Q2 2023, driven by higher utilization of Medicare rebates and expired product returns.
- Total expenses for Q2 2024 increased by 6% year-over-year, primarily due to higher clinical trial costs related to the pivotal Phase 3 program.
- The company's cash, cash equivalents, restricted cash, and investments decreased to $152.5 million as of June 30, 2024, from $192.4 million as of December 31, 2023.
Q & A Highlights
Q: What is driving the change in timelines for the endometrial trial and the higher screen failure rates? Are there any other changes being implemented in the trial?
A: Reshma Rangwala, Executive Vice President, Chief Medical Officer, explained that the main driver for the change in timelines is the increased screen failure rate, which is unique to a biomarker-driven trial. Physicians are submitting biopsies early during chemotherapy, but patients need to complete months of chemotherapy, leading to potential screen failures for various reasons. To address this, Karyopharm is activating new sites and enhancing medical affairs support.
Q: How many new sites do you anticipate opening for the endometrial trial, and in what regions?
A: Reshma Rangwala stated that they are not providing specific details on the number of new sites or regions but are focusing on opening new sites in existing countries and potentially additional countries to increase the screening pool.
Q: Can Karyopharm do anything to mitigate the biopsy process issue in the endometrial trial?
A: Reshma Rangwala noted that the biopsy process is already smooth, with quick turnaround times for results. The main issue is the interval between biopsy submission and chemotherapy completion, which cannot be offset. They expect to see a rise in patients with p53 wild-type status over time.
Q: Can you comment on any PFS metrics so far in the endometrial study and whether enrolling pMMR TP53 wild-type patients might be pushing out the data?
A: Reshma Rangwala mentioned that they do not have insight into PFS results as the trial is double-blinded. However, the data from the SIENDO trial continues to show strong PFS benefits, which have been incorporated into their projections for the early 2026 top-line result readout.
Q: Is the TP53 wild-type biomarker as prevalent as expected in the screening process?
A: Reshma Rangwala confirmed that the TP53 wild-type biomarker is prevalent as expected, with more than half of all patients showing this status.
Q: Can you give a sense of the duration of treatment for pre and post T-cell therapies in academic centers?
A: Sohanya Cheng, Executive Vice President, Chief Commercial Officer, explained that while they do not disclose exact months of duration, they have seen an increasing duration trend since the drug's launch. Recent dynamics in the competitive landscape have impacted duration, but the majority of patients remain in earlier lines of treatment, helping to propel duration up.
Q: Are there opportunities to adjust the powering or design of the Phase 3 myelofibrosis study based on ongoing data or regulatory outcomes from other drugs?
A: Reshma Rangwala stated that they remain confident in the myelofibrosis data and that any adjustments would be based on assessments by an independent DMC. They are currently blinded to the results.
Q: How confident are you that you have sufficient cash runway to get to the updated endometrial cancer top-line readout?
A: Michael Mason, Chief Financial Officer, Executive Vice President, Treasurer, explained that they have tightened their 2024 OpEx guidance and have sufficient cash runway into early 2026, excluding the convertible notes due in October 2025.
Q: Can you comment on the potential for out-licensing other programs in the pipeline, similar to KPT-9274?
A: Richard Paulson, President, Chief Executive Officer, Director, mentioned that they are looking to partner or out-license KPT-9274 and other assets like eltanexor. They have optionality in their pipeline and are opportunistic in recognizing value from these assets.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
