Corinne Johnson

Corinne Johnson inquired about the expected steady-state percentage of new patient starts from NPM1 for Revuforj and the go-forward decision criteria for the axatilimab IPF study, including partnership dynamics with Incyte.

Answer

Michael Metzger, CEO, and Steve Closter, Chief Commercial Officer, discussed the anticipated growth of NPM1 patient starts, expecting it to quickly reach 50/50 with KMT2A and eventually exceed it. Nick Botwood, Head of R&D and Chief Medical Officer, outlined IPF study criteria including statistical significance, clinical relevance, and FVC difference, with Michael Metzger adding that Syndax and Incyte are eager to collaborate on Phase III upon positive results.

Corinne Johnson inquired about the expected steady-state percentage for new patient starts from NPM1, given the current 30% early data, and the go-forward decision criteria for the IPF study, including how the decision is made with partner Incyte.

Answer

Michael Metzger, Chief Executive Officer, and Steve Closter, Chief Commercial Officer, stated that the NPM1 percentage is expected to grow meaningfully, potentially reaching 50/50 with KMT2A and eventually surpassing it due to the larger NPM1 population. Nick Botwood, Head of R&D and Chief Medical Officer, outlined IPF decision criteria, including statistical significance, clinical relevance, and an FVC difference of 40 mils at 26 weeks (80-100 mils annualized), aiming for over 40% relative difference. Michael Metzger added that both Syndax and Incyte are eager for positive results and expect to collaborate on the Phase 3 program.

Corinne Johnson asked for TG Therapeutics' perspective on the future role of a sub-Q offering in the MS market, particularly concerning IV versus sub-Q share dynamics, given the variety of dosing options expected in the coming years, including BRIUMVI's sub-Q formulation.

Answer

Michael Weiss, Chairman and CEO, noted that the sub-Q portion of the market has been relatively stable (35-40%) but believes that more available options will create energy and likely expand this segment over time. He suggested that forces will push patients towards at-home sub-Q administration, leading to market growth.

Corinne Johnson asked for TG Therapeutics' perspective on the future role of a sub-Q offering in the market, specifically regarding the potential shift in IV versus sub-Q share, considering competitor commentary and the variety of upcoming dosing options.

Answer

Michael Weiss, Chairman and CEO, noted that the sub-Q market has been relatively stable at 35-40% but believes that new options will create more energy and marketing in the space, leading to an expansion of the sub-Q market over time. He suggested that forces will likely push people towards at-home sub-Q administration. Adam Waldman, Chief Commercialization Officer, concurred with this assessment.

Corinne Johnson asked about the implications of the FDA's requirement for a year-long Phase 3 for new molecules like bivamelagon on the development of RM-718 in the same indication, and how Rhythm Pharmaceuticals plans to manage quality control for ex-U.S. patient enrollment in the Phase 3 program.

Answer

David Meeker, Chairman, CEO, and President, stated that the requirement for a year-long Phase 3 would likely apply to RM-718, though he noted RM-718 is a peptide analogous to setmelanotide, which might warrant further discussion with the FDA. He expressed confidence in managing quality control for ex-U.S. trials, citing the sophistication of global CROs and international centers, emphasizing that finding sites with good patient access is the primary challenge.

Corinne Johnson asked how the FDA's requirement for a full Phase 3 for new molecules like bivamelagon would impact the development of RM-718 in the same indication. She also inquired about Rhythm Pharmaceuticals' strategy for maintaining quality control in Phase 3 programs when enrolling patients predominantly outside the U.S.

Answer

David Meeker, Chairman, CEO, and President, indicated that the conservative base case is that RM-718 would likely face similar Phase 3 requirements as bivamelagon, though he noted RM-718 is a peptide, potentially warranting further discussion with the FDA. Regarding quality control for ex-U.S. trials, Dr. Meeker expressed confidence in the sophistication of international centers and CROs, stating that finding sites with good patient access is the primary challenge, not quality.

Eric, on behalf of Corinne Johnson, asked for more detail on the HO launch, specifically the process and cadence of reimbursement and the anticipated gross-to-net ratio compared to BBS.

Answer

David Meeker, Chairman, CEO and President, explained that the gross-to-net ratio is primarily driven by the Medicare/commercial mix (around 50/50 for BBS) and Medicaid share, with potential for a favorable shift if Medicare access is gained. Jennifer Lee, EVP, Head of North America, reiterated ongoing payer engagement and the ability to secure reimbursement even before formal policies are in place, leveraging the experience gained with BBS.

Eric, on behalf of Corinne Johnson, requested more color on the process and cadence of reimbursement for the HO launch and the anticipated gross-to-net, specifically in comparison to BBS.

Answer

David Meeker, Chairman, CEO and President, Rhythm Pharmaceuticals, stated that the gross-to-net for HO is difficult to predict but will primarily depend on the Medicare/commercial mix, as rebates are not a significant factor. Jennifer Lee, EVP, Head of North America, Rhythm Pharmaceuticals, explained that Rhythm is already engaging with payers for pre-approval information exchange. She anticipates a similar reimbursement process to BBS, where access was secured even before formal payer policies were in place, leveraging the existing knowledge of the drug.

Corinne Johnson of Goldman Sachs asked for clarity on the nature of the clinical data that might be shared for the RM-718 weekly formulation later this year. She also inquired about any visibility into off-label use of setmelanotide for younger patients in the U.S.

Answer

David Meeker, Chairman, President & CEO, clarified that due to enrollment timelines for the RM-718 study now extending into Q1 2026, it is 'extremely unlikely' any data will be shared by the end of this year. Jennifer Lee, EVP & Head of North America, responded that off-label use for HO in the U.S. is currently minimal, with only a handful of prescriptions received to date.

Corinne Johnson asked what percentage of patients in the first and second-line settings (currently 30%) needs to be achieved to meet 2026 guidance, and about patterns in converting new prescribers to repeat prescribers and related depth metrics.

Answer

Harout Semerjian, President and CEO, explained that achieving guidance requires both increasing the number of new centers using RYTELO (breadth), evidenced by 150 new accounts quarterly, and increasing repeat prescribers in high-volume accounts (depth). He noted that efforts are customized to address both breadth and depth among prescribers.

Corinne Johnson requested context for the 30% first and second-line usage figure by asking for the prior period's metric, and inquired about the leading indicators being monitored to track the impact of the expanded sales force.

Answer

Jim Ziegler, EVP & CCO, clarified the 30% figure is from IQVIA's rolling three-month claims data, using a consistent methodology, but did not provide a comparative number from the prior period. He stated that leading indicators for the sales team's impact include regional sales performance and execution metrics like call reach and frequency on target physicians.

Corinne Johnson asked about the expected timeline for a judge's decision in the WAKIX litigation, the anticipated appeal process, and a reminder of the regulatory exclusivity timelines. She also inquired about the potential impact of this litigation on acceleration clauses in the existing settlements with generic manufacturers.

Answer

Dr. Jeffrey Dayno, President and CEO, stated that the timing of a judge's ruling is uncertain and cannot be speculated upon, but noted a stay is in place until February 2027. He confirmed that an appeals process is available to both sides and takes time. Dr. Dayno reiterated confidence in the IP strength and commitment to vigorously defend it, highlighting the progress made with six of seven generic filers settled. He reminded that regulatory exclusivity for EDS extends to March 2026 and for cataplexy to October 2027. Adam Zaeske interjected to clarify a date as 'August twenty-fifth'.

Corinne Johnson asked for insights into the timeline for expected decisions in the ongoing litigation, the anticipated appeal process, and a reminder of the regulatory exclusivity timelines. She also sought clarification on the potential impact of this litigation on acceleration clauses within the existing settlements with other generic manufacturers.

Answer

Jeffrey M. Dayno, President and CEO, stated that the timing for a judge's ruling is hard to predict, noting that the legal process is ongoing and an appeals process is available to both sides. He reminded that a stay is in place until February 2027 and reiterated confidence in the strength of the IP. Kumar Budur, Chief Medical and Scientific Officer, clarified that regulatory exclusivity for WAKIX for excessive daytime sleepiness extends to March 2026, and for cataplexy to October 2027.

Eric on behalf of Corinne Johnson at Goldman Sachs asked about the net price evolution for Wakix during the quarter and for the remainder of the year, and whether price is being used as a lever to drive volume.

Answer

CFO & CAO Sandip Kapadia explained that net price follows a predictable annual pattern, being lowest in Q1 due to insurance resets and then improving throughout the year. He confirmed that Q2 saw this expected improvement and that the trend should continue, allowing the company to realize a good portion of the list price increase taken earlier in the year.

Corinne Johnson asked if Fulcrum feels confident that the dose range explored (12mg and 20mg) is sufficient for a registrational study, considering FDA requirements and maximizing the agent's benefit. She also requested clarification on patient 10, who showed a higher HbF percentage at day 56 but a slight drop by week 12.

Answer

Iain Fraser, SVP of Early Development, confirmed confidence in the 12mg and 20mg doses, noting that a 30mg dose did not show incremental HBG mRNA induction in healthy volunteers. He explained that patient 10's slight HbF drop was likely an anomaly due to small numbers and assay variability, not clinically meaningful, as the patient still achieved a robust HbF induction from baseline. Dr. Martin Steinberg agreed, attributing it to assay variability. Alex C. Sapir, President and CEO, reiterated that 20mg is the dose they will recommend to the FDA for the next study, aiming to maintain their lead over competitors.

Corinne Johnson from Goldman Sachs Group questioned Fulcrum's confidence in the explored dose range (12mg and 20mg) for a registrational study, considering FDA requirements and maximizing the agent's benefit, and asked for details regarding patient 10, who showed a higher HbF at day 56 but a slight dip at week 12.

Answer

Iain Fraser (SVP of Early Development) stated that the robustness of HbF increases and downstream biomarker improvements at 12mg and 20mg doses provide confidence. He noted that a 30mg dose did not show further HBG mRNA induction in healthy volunteers, leading to the decision not to pursue it. Regarding patient 10, Fraser and Dr. Martin Steinberg (Professor of Medicine, Boston University) attributed the slight dip to assay variability and small patient numbers, emphasizing that the patient still achieved a robust HbF induction from baseline, and the difference was not clinically meaningful. Alex C. Sapir (President and CEO) confirmed that 20mg is the dose they will recommend to the FDA for the next study.

Corinne Johnson asked for an update on the progress towards clinical trials for Hypercon products, detailing the remaining steps before patient testing, and what updates to anticipate regarding product identification and development strategy once these drugs enter Phase 1 studies.

Answer

Helen Torley, President and CEO of Halozyme Therapeutics, stated that the Hypercon team is supporting two partners for Phase 1 clinical testing in 2026, involving approved blockbuster MOAs. Remaining steps include completing clinical scale-up batches and filing IND packages. She noted that product and development strategy updates would be partner-driven, but studies are likely to be posted on clinicaltrials.gov, offering initial public visibility into the partners and Phase 1 study designs. The development pathway is expected to be similar to ENHANZE, involving Phase 1 for dose identification and Phase 3 for non-inferiority.

Corinne Johnson inquired about the progress towards clinical testing for Hypercon products, specifically what steps remain before patient testing can begin. She also asked what updates to anticipate throughout the year as these drugs enter the clinic, including whether product details and development strategies would become clearer once Phase I studies commence.

Answer

Helen Torley, President and CEO, stated that the Hypercon team is supporting two partners for Phase I clinical testing in 2026, involving approved blockbuster MOAs. Remaining steps include completing clinical scale-up batches and filing IND packages. She noted that updates would be partner-confidential but anticipated that studies would likely be posted on clinicaltrials.gov, providing initial public indication of partners and Phase I design. The development pathway is expected to be familiar, involving Phase I for dose identification and then Phase III non-inferiority studies.

Corinne Johnson of Goldman Sachs inquired about the expected pace and peak potential for subcutaneous conversion in neurology launches compared to oncology. She also asked for details on the high-volume auto-injector human factor study and if such studies will become a standard prerequisite for future partnerships.

Answer

President and CEO Dr. Helen Torley expressed expectations for a robust conversion for drugs like OCREVUS in neurology, driven by the strong value proposition of a 10-minute injection versus a multi-hour IV infusion. Regarding the auto-injector, Dr. Torley explained that human factor studies are straightforward usability tests that may be used by some partners to optimize device design before clinical trials, but it will be a mix depending on the partner's needs.

Corinne Johnson inquired about the anticipated volume impact on INGREZZA in 2026, considering Austedo becoming a negotiated product, and how Neurocrine expects formularies to manage peer products with potentially more competitive pricing.

Answer

Chief Commercial Officer Eric Benevich stated the company is well-prepared for the 2027 formulary year and Austedo's negotiated price, focusing on maintaining INGREZZA's strong momentum and favorable Medicare coverage in 2026. CEO Kyle Gano added that 2026 contracting is stable, expecting net revenue per prescription to be consistent, leading to strong double-digit volume growth and increased market share.

Corinne Johnson asked about INGREZZA's share of new-to-category or new-to-class patients versus Osado, and where that share could go with the expanded sales force over the next year, contextualizing it against a year ago.

Answer

Eric Benevich, Chief Commercial Officer, explained that new patient starts (NRX) are a single-digit percentage of total TRX but are critically important due to high persistency. He stated that since the beginning of the year, with prior sales force expansion and increased formulary coverage, INGREZZA is capturing the majority of new patient starts, driving increased total market share. He intends to continue this momentum into 2026 and the critical 2027 period.

Corinne Johnson asked about INGREZZA's share of new-to-category or new-to-class patients versus Osado, where it could go, and how it compares to a year ago, especially with the expanded sales force.

Answer

Eric Benevich, Chief Commercial Officer, explained that new patient starts are a single-digit percentage of total prescriptions but are critically important due to high persistency. He stated that INGREZZA is currently gaining the majority of new patient starts, driving increased total market share, a trend they intend to continue with the sales force expansion.

Corinne Johnson of Goldman Sachs inquired about the reimbursement process for Crinesity, asking when it might become more formulary-driven and how that could affect net pricing.

Answer

CCO Eric Benevich explained that since Crinesity is a 'class of one,' many health plans may continue to cover it as a non-formulary product rather than conduct a formal review. He highlighted that reimbursement has been surprisingly fast, with over 75% of dispensed prescriptions being paid for, minimizing the need for the free goods program. Consequently, he does not expect to provide formulary coverage statistics as a key metric.

Corinne Johnson asked about Roivant's strategy for further development expansion opportunities for brepocitinib, how the recent data informs this direction, and the potential market size for cutaneous sarcoidosis relative to NIU and dermatomyositis.

Answer

Matt Gline, CEO of Roivant, expressed enthusiasm for brepocitinib's continued development in high unmet need, large orphan markets. Benjamin Zimmer, CEO of Priovant Therapeutics, added that the data reinforces the mechanistic alignment of TYK2 JAK1 inhibition to TH1/TH17-driven T-cell polarization and type 1 interferon suppression.

Corinne Johnson asked about Roivant's strategy for further development expansion opportunities for brepocitinib, how the recent data informs this direction, and the potential market size for cutaneous sarcoidosis relative to NIU and dermatomyositis.

Answer

Matt Gline, CEO of Roivant, expressed enthusiasm for brepocitinib's further development, noting its strong agent profile in high-unmet-need patient populations and the large orphan market opportunity. Benjamin Zimmer, CEO of Priovant Therapeutics, added that the data reinforces the alignment of TYK2 JAK1 inhibition with T-cell polarization, supporting its distinctive benefits.

Corinne Johnson asked about the evolving competitive clinical landscape in Graves' disease and what factors will inform sequencing decisions, as well as an update on business development.

Answer

CEO Matthew Gline highlighted FcRn's strong position in myositis and its suitability for Graves' biology, emphasizing its safety profile. He believes increased market activity will benefit all. On business development, he noted Roivant is well-capitalized and actively seeking pipeline expansion opportunities of significant scale.

Corinne Johnson asked about the evolving competitive clinical landscape in Graves' disease and its impact on sequencing decisions, and also requested an update on business development.

Answer

CEO Matt Gline highlighted FcRn as a well-understood, safe, and effective mechanism exquisitely suited for Graves' disease biology, positioning it as an important early-line therapy. He noted that increased activity in the space benefits all. For business development, he mentioned strong capitalization, excitement for pipeline expansion, and a focus on programs that can significantly impact the existing pipeline.

Corinne Johnson requested context on the upcoming Graves' disease remission data for batoclimab, asking what would constitute a clinically meaningful outcome. She also inquired about Roivant's business development outlook, including the types and sizes of deals being considered given its significant cash balance.

Answer

CEO Matt Gline explained that physicians consider any level of remission in this refractory Graves' patient population to be practice-changing, suggesting even a 10% rate would be an 'extraordinary outcome.' On business development, Gline stated that the current market is attractive for an 'asset hunter' and that Roivant will remain opportunistic, looking at potentially transformational late-stage assets.

Corinne Johnson from Goldman Sachs inquired about the expected cadence of research and development (R&D) spending as the Phase 2 trials in Alcohol Use Disorder (AUD) and Alcohol-Associated Liver Disease (ALD) increase enrollment.

Answer

CFO Greg Weaver clarified that the costs for the AUD and ALD trials are modest, already incorporated into the current budget and cash runway, and are not expected to cause any unusual changes to the company's burn rate in the near term.

Corinne Johnson inquired about the implications of Roux-en-Y surgery's prevalence on Avexatide's potential label and market, and how recent trends in bariatric surgery types might affect the PBH incidence rate.

Answer

Co-CEO & Co-Founder Josh Cohen explained that while the Phase III trial focuses on Roux-en-Y for consistency, the drug's mechanism should work across surgical types, supported by Phase IIb data. He noted that after a period of decline, Roux-en-Y is regaining popularity versus sleeve gastrectomy due to better long-term weight loss outcomes.

Asked about the long-term sales force strategy, specifically if the planned 2025 expansion will be sufficient for the MDD opportunity or if further expansions are likely.

Answer

The company expects that after the 2025 expansion, the sales force will be appropriately sized to optimize the MDD launch and be highly competitive. While they will always reevaluate based on market conditions, the current plan is for next year's expansion to get them to the right size.