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Breakthrough Developments in Diabetes and Pain Management Therapies

In recent advancements, a study involving 50 patients with difficult-to-control diabetes is focusing on severe hypoglycemic events that occur despite optimal medical management.

Phase 1/2/3 Study Aims for Insulin Independence

This ongoing Phase 1/2/3 study primarily aims to measure the percentage of patients who achieve insulin independence without severe hypoglycemic incidents. As we embark on critical development for this potentially curative therapy for severe type 1 diabetes, it’s significant that this pivotal trial of allogeneic regenerative cell therapy follows the first-ever approval of CRISPR/Cas9 gene-edited cell therapy, CASGEVY, less than a year ago. These milestones reflect substantial progress in cell and gene therapy.

To summarize the VX-880 program, I will present data shared at the EASD conference this September. This new data builds on earlier findings and included a larger patient group with extended follow-up compared to our American Diabetes Association presentation.

Specifically, the data showed that four patients who participated saw a complete follow-up over 12 months, achieving both primary and secondary endpoints. All four had no severe hypoglycemic events, reached insulin independence, and maintained hemoglobin A1c levels below 7%, as recommended by the ADA. Additionally, we reported that five more patients were able to stop using exogenous insulin while working towards the 12-month primary endpoint evaluation. Safety and tolerability data from this recent update were consistent with those reported in June.

Progress of VX-264 in Medical Trials

Beyond VX-880, our VX-264 program incorporates the same VX-880 cells within a proprietary device that aims to eliminate the need for immunosuppressants. Currently, patients are being enrolled and dosed in Part B of the trial at the full target dose with staggered administration. Looking ahead, Part C will also feature the full target dose without staggering. We anticipate sharing findings from Parts A and B in 2025.

Povetacicept’s Milestones and Potential

Turning our attention to the povetacicept program, Pove functions as a dual antagonist of the BAF and APRIL cytokines, which are critical in autoimmune diseases that affect B cells. Its dual action and once-monthly dosing route provide us confidence in Pove as a transformative option for patients with IgA nephropathy and possibly other conditions. Recently, Pove reached several significant milestones.

We are pleased to announce the initiation of the global Phase 2 RAINIER study for patients with IgA nephropathy. This randomized double-blind trial compares 80 milligrams of Pove with a placebo, integrated with standard care. The study is currently screening and enrolling patients across various clinical sites worldwide and includes a preplanned interim analysis to assess proteinuria at the 36-week mark, which could support accelerated approval in the U.S.

Further, the trial will continue through week 104 for full approval while evaluating GFR. At the recent American Society of Nephrology Meeting, we shared positive updates from the RAINIER Phase 3 study. The updated data demonstrated a mean reduction of 66% in UPCR among patients receiving Pove, maintaining stable renal function. Impressively, five out of the eight participants achieved clinical remission.

We are energized by these results and their implications for the about 130,000 individuals diagnosed with IgA nephropathy in the U.S. Moreover, emerging data from Pove related to primary membranous nephropathy—another renal B-cell-mediated disease—indicate promising developments. This condition currently affects approximately 60,000 Americans, lacking approved targeted therapies.

Advancements in Pain Management Therapies

Shifting focus to our pain management portfolio, the FDA’s review of suzetrigine for moderate to severe acute pain is progressing, with a PDUFA date set for January 30, 2025. Our Phase 3 data arising from two randomized control trials and a single-arm safety study were showcased at the recent American Society of Anesthesiology Annual Meeting, garnering strong interest from physicians.

As we await regulatory review outcomes, our pipeline also includes other pain management assets. The next-generation NaV1.8 inhibitor, VX-993, is currently undergoing studies for two acute pain trials—a Phase 2 trial post-bunionectomy using an oral formulation and a Phase 1 study utilizing an IV formulation. We are also making headway in our NaV1.7 pain signal inhibitor program, which may provide beneficial options alone or alongside NaV1.8 inhibitors.

Expanding Innovations in Peripheral Neuropathic Pain

Addressing diabetic peripheral neuropathic pain, which affects about 2 million Americans annually, I am excited to report two key updates. Firstly, we have launched the Phase 3 pivotal program for suzetrigine. Secondly, we commenced a Phase 2 study for VX-993’s oral formulation specifically targeting lumbosacral radiculopathy, a type of neuropathic pain impacting over 4 million Americans each year with no approved treatments in the U.S.

This ongoing 12-week trial includes around 200 patients randomized to receive either suzetrigine at 69 milligrams or a placebo. We aim to measure changes in the NPRS scores from baseline to the end of the study, allowing for an effective evaluation of treatment impact, which could inform a possible Phase 3 trial.

Progress on Myotonic Dystrophy Type 1 Treatment

Lastly, I’ll provide an update on the myotonic dystrophy type 1 (DM1) program, the most common type of muscular dystrophy, impacting 110,000 patients in the U.S. and Europe, with no approved therapies currently available. Our Phase 1/2 clinical study of VX-670 began last year and has recently accelerated. While the single ascending dose (SAD) component is complete, we have now initiated the multiple ascending dose (MAD) phase, which will evaluate both safety and efficacy.

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Vertex Pharmaceuticals Reports Strong Growth and Future Initiatives in CF and Pain Management

Vertex Pharmaceuticals is making significant strides with its research and development projects, focusing on CF therapies and CASGEVY, a treatment for sickle cell disease.

Stuart A. Arbuckle — Executive Vice President, Chief Operating Officer

Thanks, Reshma. I’ll start with cystic fibrosis (CF), followed by updates on the CASGEVY launch and the outlook for suzetrigine in treating acute pain. We’ve seen impressive results in CF as we increase the number of eligible patients using our CFTR modulators, both in the U.S. and internationally.

Our CF treatments have achieved quick regulatory and reimbursement progress. KAFTRIO is now approved and accessible in every EU country. We expect our CF revenue to grow significantly in the short, medium, and long term. Initially, our priority is to connect with more eligible patients worldwide using our approved medications.

Looking ahead, we believe that the introduction of vanzacaftor, a triple-combination therapy, will further enhance our growth in CF. If approved, this new option could attract patients currently on a CFTR modulator or the 6,000 who stopped using our existing CFTR therapies. Additionally, targeting over 5,000 CF patients who do not respond to CFTR modulators with VX-522 could yield more growth opportunities.

Now, let’s discuss CASGEVY, our innovative therapy designed for patients with sickle cell disease and beta-thalassemia. We are pleased to see a positive reception from patients, doctors, and policymakers, which is fueling momentum in our launch across various regions. Key indicators of our launch success include the number of authorized treatment centers and patient cell collections. Currently, we have 45 authorized treatment centers, an increase from just over 35 last quarter, and we aim to reach approximately 75 centers globally. So far, around 40 patients have undergone at least one cell collection, up from about 20 in Q2.

We are also proud to report that the first commercial patients have received infusions of CASGEVY. In the U.S., coverage has not been a major barrier to accessing this therapy. In the U.K., NHS England reached a positive coverage agreement for TDT in September, just six months after regulatory approval.

Moreover, we have begun discussions with NHS to expand access for patients with sickle cell disease. We are also making regulatory advancements, having received approvals for both sickle cell disease and TDT in Switzerland and Canada this quarter, with plans for submissions in Kuwait and the UAE by the end of the year. Given increasing demand for CASGEVY, we are investing in additional manufacturing capacity. In September, we approved a third manufacturing facility for this therapy with our partner, Lonza. We are determined to finish this year strong for CASGEVY, as evidence of its value continues to grow among patients, healthcare professionals, and governments. This therapy represents a multibillion-dollar opportunity, and we are enthusiastic about its potential as we move towards 2025.

Now, let’s shift gears to suzetrigine in acute pain management. With the U.S. PDUFA date fast approaching, our launch preparations are well underway. Suzetrigine could potentially revolutionize treatment for the 80 million Americans seeking prescription therapies for moderate to severe acute pain annually.

Despite existing options, a significant unmet need for effective pain management persists, as highlighted by a recent nationwide survey we conducted. This survey involved over 500 healthcare providers and 1,000 patients, showcasing the difficulties faced with current treatments for acute pain. Notably, 78% of providers expressed concerns about the side effects and addiction risks associated with opioids prescribed for acute pain. Almost 90% of providers felt that side effects of current therapies hinder their ability to treat acute pain effectively. Additionally, two-thirds of patients stated they would prefer a non-opioid option for future pain experiences.

These survey findings echoed discussions held with anesthesiologists and pain specialists during the ASA Annual Meeting. Our Phase 3 results were well received, affirming our belief that physicians are eager for a new treatment option to bridge the gap between NSAIDs and opioids. Given the grim facts surrounding opioid use—such as 10% of acute pain patients becoming long-term users, and 85,000 developing opioid use disorder in the first year—it’s clear there’s a pressing need for alternatives. The opioid crisis continues to impose a staggering $180 billion yearly burden, with $60 billion of that going towards healthcare costs related to opioid use disorder.

We are receiving positive feedback from pre-approval conversations with payers and decision-makers at hospitals and insurance plans as well. These discussions are aimed at speeding up formulary and coverage decisions by highlighting unmet needs and highlighting suzetrigine’s benefits. Ultimately, we aspire to fundamentally change pain treatment protocols. In 2025, our focus for suzetrigine will be on ensuring broad access and establishing a smooth experience for patients and healthcare providers.

To achieve this, we are investing in strategies to facilitate quick patient access to suzetrigine, a critical aspect given the urgency in treating acute pain. Among our initiatives are working to secure national retail distribution and setting up financial assistance programs to support patient access ahead of traditional payer coverage timelines. These efforts ensure that patients can receive the treatment their doctors believe is most effective. We are committed to building a strong base of prescribers for this acute pain management program to create long-term value.

Vertex Pharmaceuticals Reports Strong Q3 Results and Future Pipeline Aspirations

Key Developments in Pain Management Innovations

Vertex Pharmaceuticals is advancing its pain relief pipeline with several exciting developments. Among these is suzetrigine, which targets peripheral neuropathic pain. The company has plans for IV and oral formulations, along with VX-993, NaV1.7s, and potential combination therapies. Vertex aims to start benefiting patients suffering from moderate to severe acute pain following the expected approval of suzetrigine in early 2025. In terms of policy, the NOPAIN Act will launch on January 1, 2025, providing additional coverage for new oral non-opioid treatments in hospitals and outpatient centers.

Momentum in Legislative Support for Non-Opioid Pain Management

The proposed alternatives to the PAIN Act have garnered significant bipartisan support, with 64 members of Congress signing on. This initiative seeks to balance co-pays for branded and generic medications and eliminate the requirement to use opioids before trying non-opioid treatments for Medicare Part D patients. Furthermore, all 50 states are introducing guidelines to limit opioid prescriptions, and about one-third are pushing for legislation to improve access to non-opioid options in Medicaid and state-managed healthcare plans.

Vertex’s Growth and Exciting New Launches on the Horizon

This is an exciting time for Vertex as they continue to see growth in cystic fibrosis (CF), with the launch of vanzacaftor triple therapy expected in early 2025. The ongoing launch of CASGEVY in the U.S., Europe, and the Middle East signals a new era of commercial diversification for the company. Vertex is preparing to launch suzetrigine for acute pain while also advancing various programs in Phase 3 development and even more in earlier stages.

Financial Overview of Q3 2024

Charles F. Wagner — Executive Vice President, Chief Financial Officer

Thank you, Stuart. Vertex’s impressive Q3 results reaffirm our strong performance and growth trajectory. In Q3 2024, the company reported a revenue increase of 12% year-over-year, amounting to $2.77 billion, with a notable 10% growth in the U.S. and 14% internationally.

Overall, year-to-date revenue has reached $8.1 billion, marking a 10% rise over the same period in 2023. This includes 9% growth in the U.S. and 13% outside the U.S. During this quarter, we recorded $2 million in revenue from the first patient dosed with CASGEVY. On the expenditure side, combined non-GAAP R&D, acquired IPR&D, and SG&A expenses totaled $1.08 billion, up from $993 million in Q3 2023.

In terms of further details, Q3 2024 showed $15 million in AIPR&D charges, down from $52 million in Q3 2023. Non-GAAP R&D expenses for this quarter were $764 million, reflecting a 5% year-on-year increase as we continue to invest in our broad R&D initiatives, including several clinical trials. We are balancing this growth with the transition of certain costs as we achieve clinical milestones. The increase in Q3 non-GAAP SG&A expenses to $300 million, a 39% rise from last year, is largely due to our investments in commercial activities, including the launch of CASGEVY and preparing for suzetrigine’s market entry.

Reporting a non-GAAP operating income of $1.31 billion for Q3 2024, we saw an increase compared to $1.17 billion in Q3 2023. The non-GAAP tax rate for the quarter was 19.8%, consistent with 19.4% reported last year. Vertex ended Q3 with $11.2 billion in cash and investments, utilizing over $300 million to repurchase 640,000 shares. To date, we have repurchased over 1.7 million shares at a cost exceeding $750 million.

Outlook and Guidance for 2024

Looking ahead, we are raising our product revenue forecast for 2024. With one quarter left, we anticipate revenues in the range of $10.8 billion to $10.9 billion, reflecting 10% growth at the midpoint. The guidance for non-GAAP operating expenses remains consistent at $4.2 billion to $4.3 billion for combined R&D and SG&A expenses. As for acquired IPR&D, we continue to expect about $4.6 billion for the year, which includes a charge from the acquisition of Alpine in Q2. Consequently, we project a non-GAAP tax rate of around 90%, which is influenced mainly by the non-deductible charge incurred with the Alpine acquisition. Absent that, the effective tax rate would have been between 20% and 21% this year.

In conclusion, Vertex has once again delivered strong results with double-digit revenue growth. We are advancing our initiatives, including the CASGEVY launch and preparing for regulatory approvals expected in early 2025. With multiple programs in Phase 3 and further developments in our pipeline, we look forward to sharing updates in future communications. Now, I’ll turn the call over to Susie to start the Q&A session.

Questions & Answers:

Operator

We will now begin the question-and-answer session. [Operator instructions] Please pause momentarily as we prepare our roster. The first question will come from Jessica Fye with JPMorgan. Please go ahead.

Jessica Fye — Analyst

Hi, everyone. Good evening, and congratulations on the progress with povetacicept at ASN. I understand you are targeting an at-home monthly dosing regimen for that product, but I’ve noticed that the Phase 3 RAINIER trial involves post-dose monitoring. Could you elaborate on what steps are necessary to secure approval for at-home delivery? Will you be required to run a human factors trial or a bridging study? Could this potentially be managed during an open-label extension phase to facilitate an at-home launch? Thank you.

Reshma Kewalramani — President and Chief Executive Officer

Hi, Jess, thanks for your kind words. We’re also excited about our results at ASN, particularly regarding proteinuria and the emerging GFR outcomes, along with improvements in conditions such as membranous nephropathy. You’re correct that the current protocol includes an in-office injection administered by a healthcare professional. We are preparing for regulatory review to enable at-home delivery on a monthly basis, and we have had regulatory interactions to support this path. Our goal is for commercialization to align with at-home administration, which involves small volume subcutaneous injections on a monthly schedule.

Jessica Fye — Analyst

Thank you.

Reshma Kewalramani — President and Chief Executive Officer

You got it.

Operator

The next question will follow shortly.

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Vertex Pharmaceuticals Shares Insights on Suzetrigine and Future Trials

Salveen Richter — Analyst

Thank you. It’s nice to see the progress here. For the upcoming Phase 2 suzetrigine data and LSR, could you help frame what Vertex is looking for in the delta and the NPRS score, for the drug versus baseline, but also versus placebo recognizing that you’re not powered on this front just in order to kind of decide to move forward into this Phase 3. And separately, if you could just help us understand the confidence in defining this patient population for your trial.

Reshma Kewalramani — President and Chief Executive Officer

Yeah. Sure thing, Salveen. We are very close to having the results. I’ll let the results speak for themselves.

As I mentioned in the prepared remarks, I fully expect to share them before the end of the year. In terms of study goals, I would focus on three key areas. First, we prioritize establishing a strong safety profile for the drug. Second, and more to your point, we are focused on efficacy measurements, specifically the VX-548 NPRS score from baseline to 12 weeks. We are aiming for statistically significant results and are particularly interested in the magnitude of the treatment effect.

We also want to analyze the results from the placebo group in the same manner. It’s important to note, as you mentioned, that we did not power the study to compare VX-548 against placebo, which we intend to address in the Phase 3 study. This phase will help us determine the appropriate size of the trial, contingent upon positive results in Phase 2.

Regarding our confidence level in the trial, I remain optimistic based on several factors. First is the mechanism of action itself. Second, we have a growing body of clinical evidence from both acute pain studies and diabetic peripheral neuropathy (DPN) involving VX-548 and its predecessor molecule, VX-150. Last but not least, patient selection for the LSR study has been conducted meticulously, ensuring we target individuals suffering from lumbosacral radiculopathy. We have put an emphasis on thorough physical examinations to accurately identify these patients, which strengthens our confidence in the findings.

Salveen Richter — Analyst

Thank you.

Operator

Next question will come from Terence Flynn with Morgan Stanley. Please go ahead.

Unknown speaker — — Analyst

Hi. Good evening. This is Chris on for Terence. Thanks for taking our questions.

Just one question, Susie. We know that you won’t comment directly on its pricing, but can you provide some of the inputs that you are considering as you make decisions on how to price the drug? Thank you.

Reshma Kewalramani — President and Chief Executive Officer

Absolutely. Stuart?

Stuart A. Arbuckle — Executive Vice President, Chief Operating Officer

Yeah, Chris. We will consider the same factors we consider for all our medicines, particularly how much clinical benefit we believe suzetrigine provides and the unmet medical need it addresses for individuals and society as a whole. We will integrate all these inputs to reach a pricing decision closer to the approval time.

Unknown speaker — — Analyst

Thank you.

Operator

Your next question will come from Mohit Bansal with Wells Fargo. Please go ahead.

Mohit Bansal — Analyst

OK. Thank you for taking my question. I have a two-part question on LSR. Firstly, can you compare the mechanisms of action between Lyrica and VX-548? Also, considering the subgroup analysis results for neuropathic pain, can you confirm Lyrica’s impact, particularly in relation to LSR-related neuropathic pain? Thank you.

Reshma Kewalramani — President and Chief Executive Officer

Sure. Mohit, you’ve raised some important questions regarding gabapentinoids like Lyrica. Let’s first discuss the mechanisms of action.

These mechanisms are fundamentally different. Gabapentinoids, including Lyrica, were initially designed as antiepileptic drugs and work by depressing the central nervous system, which does not directly relate to pain management. Conversely, VX-548 specifically targets NaV1.8 channels located in C fibers within the peripheral nervous system. This targeted action is important because it plays a vital role in how pain signals are transmitted. This specificity is why I’m confident as we await results for both acute pain and DPN studies.

As for Lyrica, it has shown efficacy in diabetic peripheral neuropathy but has not produced positive results specifically for lumbosacral radiculopathy in the U.S. While it is possible that some subgroup studies indicate a signal of efficacy, I cannot confirm that for LSR.

Mohit Bansal — Analyst

Thank you.

Reshma Kewalramani — President and Chief Executive Officer

Sure thing.

Operator

Next question will come from Tazeen Ahmad with Bank of America. Please go ahead.

Tazeen Ahmad — Analyst

Hi, everyone. Good evening. I appreciate you taking my questions. Considering LSR and its implications for other indications you are exploring, regardless of LSR results, should we draw conclusions about what these results could signal, especially with other studies reading out next year? Additionally, I am curious about the recent competitor news from Orion Corporation regarding the discontinuation of its NaV1.8 ODM-111 in both acute and chronic pain studies due to a narrow therapeutic window. What insights can be gained from this development?

Reshma Kewalramani — President and Chief Executive Officer

Sure, Tazeen. Addressing your question regarding the discontinued molecule, I’m not surprised. While I won’t comment in detail on that specific molecule, it is vital to recognize that challenges in development can occur, particularly when the mechanisms are similar. Our focus remains on our clinical developments and the unique characteristics of our therapeutic candidates.

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Major Developments in NaV1.7 and NaV1.8: A New Dawn in Pain Management

Overview of NaV1.7 and NaV1.8 Research

Many companies have dedicated years, even decades, to explore NaV1.7 and NaV1.8—targets often dubbed the ‘holy grail’ in pain management. This is primarily due to their pivotal role in pain transmission. However, researchers face a significant challenge: the similarity among NaV1.1 through NaV1.9 channels. Efforts to create drugs that can specifically target NaV1.7 or 1.8 have struggled with achieving the needed specificity. Interestingly, our molecule boasts a remarkable 40,000-fold increased specificity for the NaV1.8 channel compared to others.

NaV1.7 vs. DPN: Understanding the Distinction

Both LSR and DPN share characteristics as forms of peripheral neuropathic pain, yet there’s no need to directly link the outcomes from LSR to those of DPN. Importantly, the same compound, which is already showing promise in DPN, is also in Phase 3 development. We are currently in the process of screening, enrolling participants, and dosing for these trials, and I anticipate no changes in our plans moving forward.

Pipeline Insights from Jefferies’ Michael Yee

Michael Yee raised a couple of points regarding our pipeline. He expressed enthusiasm for our compound 993, related to NaV1.8, and inquired about its benefits beyond potency compared to 548. While 548 has shown satisfactory results regarding drug delivery, our strategy with 993 revolves around innovation and broadening the scope of pain management. Additionally, 993 can be formulated for intravenous use, which 548 cannot. With ongoing progress on our NaV1.7 program, we aim to identify optimal combinations and pairings effectively.

APOL1 Research and Regulatory Developments

Turning to our work in APOL1-mediated kidney disease, we are steadily moving through the Phase 3 development stages, with patient enrollment ongoing. Recent discussions have highlighted a positive shift in the regulatory landscape, especially concerning homogeneous proteinuria conditions. We foresee potential for accelerated approval based on one-year data from GFR slope assessments, emphasizing the need for precise endpoints in these trials.

Insights from CASGEVY’s Commercial Process

Evan Seigerman from BMO Capital Markets was curious about our learnings from the CASGEVY administration process. The journey of commercializing a medicine in real-world scenarios always provides valuable insights. We’ve recognized the gravity of decisions made by both patients and physicians regarding starting a lengthy treatment process. The importance of thorough discussions before the cell collection and actual CASGEVY infusion cannot be understated. Furthermore, treatment centers are eager to offer CASGEVY, reflecting the excitement from policymakers regarding its scientific advancements.

Looking Ahead: Strategy for Suzetrigine’s Commercialization

Phil Nadeau from TD Cowen mentioned ongoing contractual discussions regarding the commercialization of suzetrigine. As we progress, we are exploring strategic initiatives like co-pay assistance to enhance patient access and ensure a smoother adoption process. The lessons we’ve learned will certainly shape our approach as we move forward in delivering these innovative therapies.

Updates on Non-Opioid Treatments and Clinical Trials from Vertex Pharmaceuticals

Contracting Discussions: Progress and Expectations

In response to inquiries about contracting discussions, Stuart A. Arbuckle, Executive Vice President and COO at Vertex Pharmaceuticals, indicated that conversations with insurance payers are progressing positively. There is a clear recognition of the unmet needs in the market, particularly concerning non-opioid alternatives like suzetrigine, which aims to treat acute pain. This drug is particularly noteworthy as it represents the first new non-opioid treatment option in decades.

Payers are fully aware of the scale of acute pain sufferers in the U.S. and are considering potential budget impacts. As discussions are still early in the process, Arbuckle emphasized the importance of securing reimbursed access as close as possible to the drug’s approval date.

Focus on Access: Ensuring Availability for Patients

While anticipating some delays in reimbursement decisions after approval, the company is working proactively to reduce barriers for patients. Arbuckle stated that it is critical to address gaps that may prevent some patients from accessing suzetrigine immediately following its approval. He highlighted the enthusiasm among healthcare providers and patients for the new medication, aiming to facilitate a smooth transition to its usage, even if plans haven’t finalized their decisions on reimbursement.

An essential aspect of these efforts includes ensuring that retail pharmacies have sufficient supplies of suzetrigine available for patients who require it urgently.

Prospects for Inaxaplin: Commercial Opportunities and Patient Enrollment

Next, Debjit Chattopadhyay from Guggenheim Partners raised questions regarding inaxaplin, particularly about enrollment timelines. Reshma Kewalramani, President and CEO, acknowledged the commercial potential for inaxaplin, particularly within the growing Vertex renal franchise. Arbuckle later elaborated that approximately 100,000 patients in the U.S. and Europe are affected by a specific kidney disease, presenting a significant market opportunity for inaxaplin. Many patients remain undiagnosed due to the relatively recent discovery of the associated gene.

The company is engaged in initiatives aimed at boosting disease awareness, diagnosis, and genotyping efforts to tap into this market more fully.

Evaluating VX-548: Key Decision Factors for Advancement

In a follow-up question concerning the treatment VX-548, analyst Olivia Brayer sought to understand the criteria used to advance it into a registrational program. Kewalramani responded that the company is looking closely at the treatment effect’s magnitude and safety profile. They will assess how early the clinical data signal separates from placebo results, which could influence the design of subsequent trials.

As they await relevant data on these investigations, the executives foresee more comprehensive discussions about their findings and strategic next steps shortly.

Final Thoughts

The discussions and strategies shared by Vertex illustrate their commitment to addressing critical unmet healthcare needs while navigating the complexities of market access and patient engagement. As the company continues its innovative work, the coming months will be pivotal for their product launches and the potential impact on patient care.

Vertex Pharmaceuticals Discusses Suzetrigine Authorization Challenges

Analysts Question Prior Authorization for Suzetrigine

Ms. Liisa Bayko from Evercore ISI opened the discussion, expressing her interest in the potential absence of prior authorization for sapprezzig, a new drug being examined by Vertex Pharmaceuticals. She highlighted the importance of reducing barriers for patients to access this medication.

Response on Policy and Market Access Efforts

Reshma Kewalramani — President and Chief Executive Officer

Reshma directed the question to Stuart A. Arbuckle, the company’s Chief Operating Officer, to offer insight into both policy discussions and commercial initiatives regarding the drug.

Stuart A. Arbuckle — Executive Vice President, Chief Operating Officer

Stuart acknowledged the concerns about how certain management controls, primarily prior authorizations, could hinder suzetrigine’s market entry. He mentioned the Alternatives to PAIN Act, which is intended to eliminate such restrictions for Medicare Part D beneficiaries. During their preapproval exchange discussions with various health plans, they have addressed the sensitivity of this issue.

Stuart emphasized the significance of the unmet medical needs that suzetrigine seeks to address. While Vertex is able to discuss the benefits and risks of the drug, accessing reimbursement remains complex, as different healthcare providers and plans have varied requirements. For instance, there may be cases where prior authorization is necessary even before a definitive coverage decision is made.

He added that while some prior authorization processes might be in place, it is unlikely patients would be forced to try a generic opioid before being approved for suzetrigine. This variability means that there is no single answer regarding the authorization issue, but Vertex aims to facilitate easier access for both patients and healthcare providers. He anticipates strong interest in suzetrigine when it is approved early in 2025.

Conclusion of the Conference Call

Operator

The operator wrapped up the session, thanking all participants. The call can be replayed by dialing specific numbers provided, allowing for further review of today’s discussion.

Conference Call Participants

Susie Lisa — Senior Vice President, Investor Relations

Reshma Kewalramani — President and Chief Executive Officer

Stuart A. Arbuckle — Executive Vice President, Chief Operating Officer

Charles F. Wagner — Executive Vice President, Chief Financial Officer

Jessica Fye — Analyst

Salveen Richter — Analyst

Unknown speaker — — Analyst

Stuart Arbuckle — Executive Vice President, Chief Operating Officer

Mohit Bansal — Analyst

Tazeen Ahmad — Analyst

Michael Yee — Analyst

Evan Seigerman — Analyst

Phil Nadeau — Analyst

Debjit Chattopadhyay — Guggenheim Partners — Analyst

Olivia Brayer — Cantor Fitzgerald — Analyst

Liisa Bayko — Analyst

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