Q1 2024 Earnings Summary
- Vertex's acute pain medication, VX-548, is expected to receive priority FDA review due to fast track and breakthrough therapy designations and positive interactions with the FDA, indicating high enthusiasm for its efficacy and safety profile.
- Strong early momentum for CASGEVY, with over 25 activated treatment centers globally since approval in December and January, including significant growth potential in the Middle East where reimbursement agreements have been secured, potentially tapping into a market of over 23,000 patients, larger than the U.S.
- Vertex is positioning VX-548 for a broad moderate to severe acute pain label, emphasizing its strong efficacy and safety, including lack of addictive potential, which could significantly disrupt the acute pain market dominated by opioids.
- 1. Potential reimbursement hurdles for suzetrigine (VX-548) due to step therapy requirements:*
- 75% of physicians surveyed thought that patients would have to step through a generic opioid before accessing suzetrigine.
- This could limit the uptake of suzetrigine, as patients may be required to try cheaper opioids first.
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Suzetrigine Reimbursement
Q: Will suzetrigine be reimbursed after opioids in acute pain without legislative support?
A: Management hopes suzetrigine won't require patients to step through opioids before access. They believe it's unreasonable to expect patients to use treatments with significant side effects and addictive potential when suzetrigine offers strong efficacy without addiction risks. Legislative initiatives aim to reduce financial disincentives for prescribing non-opioids, and the company isn't advocating for step-through policies.
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Priority Review for VX-548
Q: How confident are you in getting priority review for VX-548 in acute pain?
A: Management is optimistic about receiving priority review for VX-548. With the rolling submission nearing completion, they expect to know within about 60 days post-submission. They highlight favorable indicators like fast track status and breakthrough designation, and note FDA enthusiasm for effective non-addictive pain treatments.
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CASGEVY Patient Enrollment
Q: Can you provide details on CASGEVY's patient cell collections in the U.S.?
A: While not disclosing specific patient numbers, management is pleased with the activation of 25 ATCs globally since approval in December and January. They see momentum building in activations and cell collections across all operating regions, including the U.S.
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Advancing VX-993 to Phase 2
Q: How will VX-993 differ, and does this affect plans for VX-973?
A: VX-993 is advancing to Phase 2 due to its readiness in preclinical and manufacturing aspects. The company seeks molecules suitable for both oral and IV use and that can combine with NAV1.7 inhibitors. VX-973 is slightly behind but remains part of their serial innovation strategy.
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CF Market in Brazil
Q: How significant is Brazil for the cystic fibrosis franchise?
A: Brazil represents about 1,500 eligible patients for TRIKAFTA. With a reimbursement agreement now in place, the company can provide access to all these patients, expanding from previous limited access via named patient sales.
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Inventory Movements Impact
Q: Were there any inventory moves this quarter to note?
A: The company acknowledged a benefit from international channel inventory phasing, amounting to $75 million to $100 million in the first quarter, expected to reverse in the second quarter.
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Vanzacaftor Triple Label Expectations
Q: Will sweat chloride data be included in the vanzacaftor triple's label?
A: Management expects sweat chloride data to be reflected in the label, as it has been with previous CFTR modulators. While label negotiations haven't begun, historical precedent suggests inclusion of this pharmacodynamic marker.
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NAV1.8 and NAV1.7 Programs
Q: Would you consider partnering for musculoskeletal pain indications?
A: The company intends to address musculoskeletal pain with their NAV1.8 and NAV1.7 assets but will not commercialize these indications themselves due to the primary care nature of the market. They plan to sequence development, focusing first on acute and neuropathic pain, which they'll handle internally.
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Hospital Administrators vs. Physicians
Q: How important are hospital administrators vs. physicians in suzetrigine adoption?
A: Both stakeholders are important in institutional decisions. Administrators and physician advocates collectively decide on formulary inclusion. The company anticipates early uptake in discharge settings and emphasizes that each patient treated with suzetrigine avoids opioid use, which is impactful for these stakeholders.
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VX-548 Phase III Data Presentation
Q: When and where will VX-548 Phase III data be presented?
A: The company plans to share more data on VX-548 at fall meetings through congress presentations and publications, coinciding with Phase III results.