Vamil Divan

Vamil Divan from Guggenheim Securities, LLC asked for more details on the Phase 3 trial design for VESPER-3, specifically regarding flexibility for patient side effects and potential efficacy improvements, and inquired about other data expected at ADA, including Phase 2 data for the internal GIP antagonist.

Answer

Dr. Chris Boshoff, Chief Scientific Officer, Pfizer, explained that the Phase 3 design for VESPER-6 would test different titrations and an additional 9.6 mg dose, unlike the VESPER-3 study's limited step-up doses. He also mentioned plans to share updated data on the ultra-long-acting amylin (3945) and potential early data for its combination with 3944 at ADA, along with progress on the internal GLP antagonist oral candidate and other early-stage peptide programs.

Vamil Divan inquired about the design of Pfizer's VESPER-3 phase III trials, specifically regarding down titration flexibility to manage side effects and potentially improve efficacy, and asked for an update on other data from Pfizer's internal programs or the Metsera portfolio expected at ADA, including phase II data for the internal GIP antagonist.

Answer

Chief Scientific Officer Chris Boshoff explained that the VESPER-3 data presented today involved only two step-up doses, but the phase III design for VESPER-6 will test different titrations and an additional 9.6 mg dose. He also mentioned plans to share updated data on the ultra long-acting amylin (3945) and potential early data for its combination with 3944 at ADA, along with progress on the oral GLP antagonist in randomized phase II and other phase I programs.

Vamil Divan asked about the competitive landscape for VYNDAMAX, including pricing and market share dynamics with new entrants, and the commercial uptake of PADCEV, specifically how the potential muscle-invasive indication could impact its growth.

Answer

Aamir Malik, Chief U.S. Commercial Officer and EVP at Pfizer, explained that VYNDAMAX continues to show strong demand growth and market leadership, despite new competition, with volume growth offset by IRA manufacturer rebates and payer contracting. For PADCEV, he clarified that Q2 sales were boosted by a one-time transition to a wholesaler model, and future growth is expected from the expanding muscle-invasive bladder cancer patient population. Albert Bourla, Chairman and CEO, Pfizer, also contributed.

Vamil Divan asked about the commercial performance of Vyndamax, inquiring about pricing, market share dynamics, and the impact of new competitors. He also questioned the commercial uptake of Padcev in the quarter, seeking insight into how the potential muscle-invasive indication could drive future growth.

Answer

Albert Bourla, Chairman and CEO, introduced the response. Aamir Malik, Chief U.S. Commercial Officer and EVP, explained that Vyndamax showed strong demand and market share leadership, with volume growth offset by IRA rebates and payer contracting. He noted competition taking some first-line share but minimal switching. For Padcev, he highlighted strong share in LAMUC and clarified that Q2 sales included a one-time boost from a distribution model change, expecting a return to growth in Q4. He also pointed to the MIDC indication as a future growth driver.

Vamil Divan asked for reinforcement of Pfizer's commitment to its dividend amid tariff uncertainty and questioned the stability of the COVID franchise business for 2025.

Answer

CFO Dave Denton affirmed the dividend remains a critical component of the capital allocation strategy, supported by improving operating margins. Aamir Malik, Chief U.S. Commercial Officer, noted that Paxlovid utilization tracks infection rates and that the company anticipates typical seasonal waves. Alexandre de Germay, Chief International Commercial Officer, highlighted strong international Comirnaty performance and expanding commercial Paxlovid sales.

Vamil Divan asked for details on the PREVNAR 25-valent vaccine's progress to Phase III and the status of the pediatric program. He also questioned the rationale for changes to the ponsegromab heart failure study.

Answer

Executive Chris Boshoff highlighted promising immunogenicity data for PCV-25 against serotype 3 and confirmed the pediatric study is ongoing. He clarified that the ponsegromab cardiovascular study was stopped, while the cancer cachexia program is advancing to Phase III. CEO Albert Bourla added that PCV-25 development will help accelerate the next-generation PCV-30+ vaccine.

Vamil Divan asked if the implied $500 million reduction in full-year non-COVID sales guidance was entirely due to Oxbryta. He also inquired about any changes to the GBT601 program following the Oxbryta setback.

Answer

CFO David Denton confirmed the reduction was largely due to the Oxbryta withdrawal and that the underlying non-COVID business remains strong, maintaining its 9-11% growth guidance. Chief Scientific Officer Mikael Dolsten stated he is encouraged by GBT601, noting its higher potency, and said learnings from Oxbryta are being applied, such as focusing the trial on high-performance clinical sites.

Vamil Divan from Guggenheim Securities asked for initial feedback on the Inlexo launch, the expected timing for a permanent J code, how SunRISe-5 data might impact the product's addressable population, and an update on the timing for SunRISe-3 data.

Answer

EVP and Worldwide Chairman of Innovative Medicine Jennifer Taubert reported strong interest and receptivity for Inlexo from urologists and patients in the BCG unresponsive population. She anticipates a permanent J code at the beginning of Q2, which is expected to be a significant catalyst for utilization, reinforcing Inlexo's potential as a $5 billion-plus asset. EVP of Innovative Medicine R&D John Reed elaborated on the broader platform, mentioning the next product, TAR-210 (containing Erleda), targeting the intermediate-risk population with over 90% complete response rates, expanding coverage for localized bladder cancer patients. He highlighted the collaboration between innovative medicines and MedTech for these bladder-sparing technologies.

Vamil Divan asked for initial feedback on the Inlexo launch and an update on when a permanent J code is expected. He also inquired about how SunRISe-5 data might impact the product's addressable population and sought an update on the SunRISe-3 data timeline.

Answer

Jennifer Taubert (EVP and Worldwide Chairman of Innovative Medicine, Johnson & Johnson) reported positive launch feedback and strong interest for Inlexo in the BCG unresponsive population. She anticipated a permanent J code in Q2 (April) as a catalyst, reiterating Inlexo as a $5 billion-plus asset. John Reed (EVP of Innovative Medicine R&D, Johnson & Johnson) expanded on the Inlexo platform, mentioning TAR-210 (ERLEADA-carrying device) for intermediate-risk bladder cancer, with over 90% complete response rates, broadening coverage for localized bladder cancer patients. He praised the MedTech/Innovative Medicine collaboration.

Vamil Divan posed a two-part question on Inlexa: first, about near-term uptake, initial doctor feedback, and the impact of the J code; second, on longer-term clinical and regulatory updates expected in the next 12-18 months to expand the addressable population and build towards a $2 billion+ product by 2028.

Answer

EVP, Worldwide Chairman, Innovative Medicine Jennifer Taubert reported strong receptivity for Inlexa, highlighting its design by urologists for high unmet need in BCG-unresponsive high-risk non-muscle invasive bladder cancer, with transformational results. She noted the J code in April next year as an important catalyst. EVP, Innovative Medicine Research and Development John Reed detailed a broad development program with several Phase 3 studies covering BCG-experienced, BCG-relapsed, and head-to-head frontline against BCG, as well as data for Inlexa + citralumab in neoadjuvant muscle-invasive disease. He also mentioned the next-generation device, TAR210, for bladder cancer.

Vamil Divan asked a two-part question regarding INLEXZO: first, about its near-term uptake, initial doctor feedback, and the impact of the upcoming J code; second, about longer-term expansion plans, including clinical/regulatory updates to broaden the addressable bladder cancer population and the path to achieving the projected $2 billion+ sales by 2028.

Answer

EVP, Worldwide Chairman, Innovative Medicine, Jennifer Taubert reported very strong receptivity for INLEXZO, designed by urologists to address high unmet needs in BCG-unresponsive high-risk non-muscle invasive bladder cancer, with transformational response rates. She noted initial insertions have occurred, and the J code in April next year is expected to be a significant catalyst. EVP, Innovative Medicine Research and Development, John Reed detailed a broad development program with several Phase 3 studies covering BCG-experienced, relapsed, and frontline settings, as well as studies in muscle-invasive disease. He also highlighted TAR210, a next-generation device for bladder cancer, as a future growth driver.

Vamil Divan of Guggenheim Securities, LLC requested an update on the data readout timing for the co-antibody therapy 4804 in IBD and psoriatic arthritis, and asked for management's perspective on its potential.

Answer

EVP John Reed stated that Phase 2b data for the co-antibody in Crohn's and colitis will read out mid-year. He then pivoted to express significant excitement for icotrokinra, an oral IL-23 inhibitor, which achieved a compelling proof of concept in ulcerative colitis. He noted a broad Phase 3 program is being planned for icotrokinra, which aims to offer biologic-level efficacy in a once-daily pill.

Vamil Divan from Guggenheim Partners questioned why CARVYKTI, TECVAYLI, and TALVEY were no longer featured on the slide highlighting upside versus consensus estimates.

Answer

EVP Jessica Moore explained that while a gap may still exist, analyst estimates for these multiple myeloma assets have risen significantly since late 2023, reducing the disconnect. CFO Joseph Wolk and EVP Jennifer Taubert reaffirmed their bullish outlook, with Taubert reiterating conviction in CARVYKTI as a $5 billion-plus asset with expanding capacity.

Vamil Divan of Guggenheim Securities asked for more detail on the lighter-than-expected U.S. sales for TREMFYA and STELARA, specifically questioning the 'unfavorable patient mix' impact and whether it was a J&J-specific issue or a broader market trend.

Answer

Jessica Moore, VP of Investor Relations, clarified that 'patient mix' refers to J&J-specific gross-to-net accounting true-ups as final bills are received from different patient channels. Jennifer Taubert, Executive VP of Innovative Medicine, added that despite this Q3 adjustment, the underlying growth prospects for TREMFYA remain very strong, especially with its recent and upcoming IBD launches.

Vamil Divan asked about the FDA's timeline for responding to the IXCHIQ suspension and requested quantification of the DUKORAL sales impact from currency fluctuations and the German distributor shift, along with future growth expectations for the asset.

Answer

CEO Thomas Lingelbach explained that there is no predefined process or precedent for the IXCHIQ suspension, making a timeline from the FDA uncertain, but expressed hope for a collaborative dialogue this year. CFO Peter Bühler noted the currency impact was mentioned in the call and clarified that the German distributor shift was a technical delay due to existing stock, without quantifying specific country sales, but reiterated expectations for continued DUKORAL brand growth.

Vamil Divan asked about the timeline for hearing from the FDA regarding the IxchIQ BLA suspension and requested quantification of the impact of currency fluctuations and the distributor shift in Germany on Ducoral sales, as well as the outlook for Ducoral's growth.

Answer

CEO Thomas Lingelbach explained that there's no predefined FDA timeline due to lack of precedent for this type of suspension, but they hope for dialogue this year. CFO Peter Bühler clarified the currency impact on Ducoral was EUR 400,000 and noted the German distributor shift caused a technical delay, but they expect continued growth for Ducoral in 2026.

Vamil Divan from Guggenheim Securities questioned why full-year sales guidance was maintained despite a strong Q1 beat. He asked about the sequencing of DoD sales for IXIARO and whether IXCHIQ conservatism was a factor. He also inquired if the company could still reach pre-pandemic gross margin levels if IXCHIQ uptake is affected by new safety information.

Answer

An executive explained that Q1 was strong but in line with internal expectations, partly due to a different sales phasing for IXIARO compared to the prior year, which was impacted by supply constraints. Therefore, the full-year guidance remains unchanged. Regarding gross margin, the executive acknowledged IXCHIQ is expected to have a better margin over time and expressed satisfaction with the strong Q1 margin performance, but did not commit to a specific 2025 target, reiterating the goal to reach pre-pandemic levels over time.

Vamil Divan of Guggenheim Securities questioned why full-year sales guidance was maintained despite a strong Q1 beat, asking about the sequencing of DoD sales for IXIARO and potential conservatism on IXCHIQ. He also asked about confidence in reaching pre-pandemic gross margin levels if IXCHIQ uptake is slower than expected.

Answer

CEO Thomas Lingelbach explained that Q1 was strong but in line with internal expectations, noting that the prior year's Q1 was impacted by supply constraints, resulting in different sales phasing this year. He confirmed DoD supply is ongoing as planned. On gross margins, he reiterated that IXCHIQ is expected to yield better margins over time and expressed satisfaction with the strong Q1 performance without committing to a specific 2025 gross margin target.

Vamil Divan asked about updating the 2027 guidance for SKYRIZI and RINVOQ given their strong performance, and sought comments on the latest market stress affecting the aesthetics portfolio, including BOTOX and JUVEDERM in the U.S.

Answer

Chairman and CEO Rob Michael confirmed that AbbVie expects to exceed the long-term SKYRIZI and RINVOQ guidance, emphasizing the diversified growth platform and underappreciated pipeline depth in immunology, neuroscience, and oncology. EVP and CCO Jeff Stewart detailed the flattish U.S. toxin market and double-digit decline in fillers, outlining AbbVie's investment to stimulate market recovery.

Vamil Divan asked about updating the longer-term outlook for SKYRIZI and RINVOQ, given the expectation to exceed 2027 guidance, and commented on the latest market stress in U.S. aesthetics for BOTOX and JUVÉDERM.

Answer

Rob Michael, Chairman and CEO, confirmed that AbbVie expects to exceed the 2027 guidance for SKYRIZI and RINVOQ, emphasizing the rapid return to growth and the underappreciated depth of the immunology and neuroscience pipelines. Jeff Stewart, Executive Vice President, Chief Commercial Officer, described the U.S. aesthetics market as flat for toxins and down double digits for fillers, but noted AbbVie's stable market share and investments in consumer campaigns and training centers.

Vamil Divan of Guggenheim Securities inquired about the commercial opportunity for RINVOQ in alopecia areata following impressive trial data and asked for more detail on the macroeconomic impacts affecting the aesthetics market.

Answer

EVP & CCO Jeffrey Stewart noted the alopecia data was transformative and that this next wave of indications could add approximately $2 billion in peak sales for RINVOQ, with potential upside. On aesthetics, he explained the market is stable but the dermal filler segment is challenged by price sensitivity and a consumer shift towards a 'natural look,' which AbbVie is actively addressing.

Vamil Divan from Guggenheim Securities inquired if aesthetics products like Botox and Juvederm are subject to current tariffs and asked about market share dynamics for toxins and fillers beyond macroeconomic pressures.

Answer

CFO Scott Reents confirmed that some aesthetics products are subject to current tariffs but the impact is modest, approximately $30 million, and has been absorbed in the company's guidance. CCO Jeffrey Stewart detailed market share dynamics, noting a Q1 share point gain in U.S. toxins after a Q4 hit, with a goal to recover more share. U.S. filler share is stable, while in China, both Botox and Juvederm have seen positive share momentum following new approvals.

Vamil Divan inquired about the pricing dynamics for Skyrizi and Rinvoq, including the impact of the Medicare Part D redesign, and asked for an update on the current market share for BOTOX and Juvederm following recent challenges.

Answer

EVP & CCO Jeffrey Stewart stated that Skyrizi and Rinvoq pricing sees modest, low single-digit declines from rebates, with a one-time impact from the 2025 Part D redesign. CEO Robert Michael reiterated that volume is the primary growth driver. SVP Carrie Strom reported BOTOX's Q4 share was in the low to mid-60s and Juvederm's was in the low to mid-40s, noting encouraging signs of recovery after reinstating the original Alle loyalty program.

Vamil Divan of Guggenheim Securities questioned expectations for the upcoming emraclidine data, specifically regarding its efficacy and safety profile relative to competitors and the potential for liver testing requirements.

Answer

CSO Roopal Thakkar highlighted emraclidine's potential for a differentiated profile, noting its once-daily dosing, lack of food effect, and absence of bladder or hepatic issues seen in Phase Ib data. He stated they do not anticipate a requirement for laboratory monitoring based on current data.

Vamil Divan asked about the Phase 3 design for TPIP in IPF and PPF trials, specifically how the company plans to overcome orphan drug exclusivity, given progress by United Therapeutics.

Answer

Will Lewis, Chairman and CEO, expressed confidence in overcoming orphan designation, citing multiple strategies. Martina Flammer, Chief Medical Officer, explained that the TETON study informs the design, emphasizing TPIP's ability to dose and titrate optimally. She noted opportunities to demonstrate superiority in efficacy, safety, or patient care impact (e.g., once-daily dosing) to overcome existing orphan exclusivity.

Vamil Divan asked about the Phase III design for TPIP in IPF and PPF, specifically how Insmed plans to overcome orphan drug exclusivity given United Therapeutics' progress.

Answer

Will Lewis, Chairman and CEO of Insmed, and Martina Flammer, Chief Medical Officer, explained that learnings from the TETON study will inform the design. Martina highlighted that orphan designation can be overcome by demonstrating superiority in efficacy, safety, or patient care impact, citing TPIP's strong PAH results and once-daily dosing as potential advantages.

Speaking on behalf of Vamil Divan, an analyst asked about the real-world definition of exacerbations in bronchiectasis and whether the number of identified exacerbations might increase once a treatment becomes available.

Answer

CEO William Lewis agreed that the availability of a new treatment for a previously untreated disease almost always triggers a 'cascade of greater awareness.' He expects this will lead to physicians and patients tracking symptoms more closely, raising the index of suspicion and resulting in more patients being properly identified and documented as having exacerbations, which is key for treatment eligibility.

An associate for Vamil Divan asked about the next-generation DPP1 inhibitors, questioning the rationale for prioritizing COPD and rheumatoid arthritis and the desired molecular properties compared to Brensocatib.

Answer

CEO William Lewis explained that the indication choices are driven by high unmet medical need and promising early animal data. He noted that the next-generation molecules are being designed with different properties than Brensocatib to optimize for clinical benefit in these new, large indications, with the first candidate potentially entering the clinic next year.

An analyst on behalf of Vamil Divan asked for the rationale behind selecting COPD and rheumatoid arthritis for next-generation DPP1 inhibitors and the desired properties of these new molecules compared to Brensocatib.

Answer

CEO William Lewis stated the choices were driven by significant unmet medical need and promising early animal data. The goal is to develop next-generation molecules that differ from Brensocatib in ways that could provide clinical benefits in these new, larger indications. He emphasized the aim is to have the biggest influence on the largest number of patients, with the first new molecule potentially entering the clinic next year.

Vamil Divan inquired about the Phase II data expectations for brensocatib in Chronic Rhinosinusitis (CRS) without nasal polyps, asking what would constitute a positive outcome.

Answer

CEO Will Lewis and CMO Martina Flammer responded. Lewis highlighted that the CRS opportunity could be larger than bronchiectasis, which has a peak revenue potential over $5 billion. Flammer detailed the BIRCH study's design, noting the primary endpoint is the sinus total symptom score. She stated the trial is powered to detect a 0.9-point change, referencing Lyra Therapeutics' REOPEN-2 study as a comparator which showed effects between 0.9 and 1.7 points.

An analyst on behalf of Vamil Divan at Guggenheim Securities asked for an overview of Insmed's current financial position and future plans following the amended Pharmakon term loan and recent ATM usage, particularly in preparation for the brensocatib launch.

Answer

CEO Will Lewis stated that Insmed's balance sheet is the strongest in its history. CFO Sara Bonstein elaborated that the recent financial maneuvers were deliberate and strategic. She noted the ATM usage was opportunistic at a high premium to the last raise, reflecting sensitivity to dilution. The restructured Pharmakon loan provides additional capital, lowers the interest rate to a fixed single-digit, and extends maturity to 2029, ensuring the brensocatib launch is fully resourced.

An analyst on for Vamil Divan asked for an overview of Insmed's financial position and future plans following the recent Pharmakon loan amendment and ATM usage, particularly in preparation for the brensocatib launch.

Answer

CEO Will Lewis expressed high satisfaction with the company's strengthened balance sheet. CFO Sara Bonstein detailed the strategic actions, noting the ATM was used opportunistically at a premium price to minimize dilution. She explained the restructured Pharmakon loan adds capital, lowers the interest rate to a fixed 9.6%, and extends maturity to 2029, ensuring the brensocatib launch is fully resourced.

Vamil Divan from Guggenheim Securities, LLC asked for quantification of the patient population for the CADENCE trial and questioned the ex-US uptake strategy for Winrevir, including pricing and market opportunity.

Answer

Dr. Dean Li, President of Merck Research Labs, noted the CADENCE patient population is underdiagnosed but could be in the range of the PAH population or larger. Chairman & CEO Robert Davis added that the ex-US launch is on track, with most reimbursements expected in the second half of the year, and that a pricing adjustment affected Q2 revenue but the outlook remains strong.

Vamil Divan of Guggenheim Securities, LLC inquired about the potential patient population size for Winrevir in the CADENCE trial's indication and asked about the ex-U.S. uptake strategy, pricing considerations, and overall market opportunity for Winrevir outside the United States.

Answer

Dr. Dean Li, President of Merck Research Laboratories, noted that the patient population for the CADENCE indication is underdiagnosed but could be in the range of PAH or larger. Chairman & CEO Robert Davis added that the ex-U.S. launch is in its early stages, with most reimbursements expected in the second half of the year. He confirmed the ex-U.S. market (Europe and Japan) is roughly half of the 90,000 global PAH patients and the launch is on track.

Vamil Divan of Guggenheim Securities asked about the impact of macro volatility on business development and the company's perspective on potential international reference pricing for U.S. drugs.

Answer

CEO Robert Davis stated that Merck's BD strategy is unchanged despite a complex environment and a disconnect in seller valuation expectations. On reference pricing, he acknowledged the price differential needs addressing and highlighted PBM reform and fair value from foreign governments as key areas for discussion with the administration.

Vamil Divan asked if the positive news from the ZENITH and HYPERION trials for WINREVAIR changes expectations for the upcoming Cadence trial in a different patient population. He also asked about any inventory fluctuations for WINREVAIR.

Answer

Dr. Dean Li, President of Merck Research Labs, stated that confidence in WINREVAIR for PAH is very high and that the company is eager to see the Cadence trial results to explore its potential in other forms of pulmonary hypertension. CFO Caroline Litchfield confirmed there were no unusual inventory levels for WINREVAIR, though an adjustment was made for the Medicare Part D redesign.

On behalf of Vamil Divan, an analyst from Guggenheim Securities asked about the commercial experience with WINREVAIR, specifically regarding its safety profile, the split between low and high doses, and patient discontinuation rates.

Answer

Dr. Dean Li, President of Merck Research Labs, reported that no significant safety signals have emerged since WINREVAIR's launch. CEO Robert Davis added that adherence has been strong with fewer discontinuations than expected. He noted that the sickest patients are being treated first, and most are starting on single vials, with an expectation that dosing will align with the STELLAR trial's mix (2/3 single vial, 1/3 double vial) over time.

Daniel on for Vamil Divan asked for a comparison of the commercial opportunity for seralutinib in PAH versus PH-ILD and inquired about the expected geographic distribution of enrollment for the PH-ILD trial.

Answer

An executive, likely CMO Dr. Richard Aranda or CCO Bob Smith, stated the PH-ILD market is 3-4 times larger than PAH, citing Tyvaso's ~$2 billion run rate in PH-ILD as evidence of the opportunity. CFO & COO Bryan Giraudo expects the PH-ILD trial's geographic mix to be similar to PROSERA but with an even larger contribution from the European Union, where there is a significant unmet need with no approved therapies for the condition.

Vamil Divan asked about management's expectations for the NEW DAY clinical trial results and their potential commercial impact on the ophthalmology business. He also inquired about the specific contribution from the generic drug prucalopride in Q1 to better understand the outlook for Q2.

Answer

President and CEO Nikhil Lalwani stated that a successful NEW DAY study could allow ILUVIEN to be used earlier in the treatment of diabetic macular edema (DME), significantly expanding the addressable market. On prucalopride, he declined to provide product-level revenue but confirmed its strong contribution. He guided that Q2 revenue for the product would be lower than Q1 as the company manages inventory ahead of its 180-day exclusivity period ending in late June.

Vamil Divan of Guggenheim Securities asked for more details on the market access issues affecting the Alimera assets (ILUVIEN and YUTIQ), the expected timeline for resolution, and the long-term growth impact. He also questioned the expected Q1 seasonality for Cortrophin given its strong momentum.

Answer

Executive Nikhil Lalwani clarified the access issue relates to underfunded patient assistance programs for Medicare patients, which ANI is addressing with its own programs and by working with HCPs. He expressed confidence in long-term growth due to a large addressable market. For Cortrophin, he confirmed a typical Q1 sales dip is expected due to insurance resets, followed by strong sequential growth, noting the raised guidance reflects strong underlying momentum.

Vamil Divan of Guggenheim Securities sought to understand the specific drivers behind Cortrophin's consistent outperformance in 2024, asking if it was due to volume, price, or certain segments. He also requested clarification on a statement about the rare disease business becoming the company's largest unit in 2025.

Answer

Executive Nikhil Lalwani responded that Cortrophin's growth is almost entirely driven by increased volume, not price. He specified that this growth is broad-based, occurring across all targeted therapeutic areas and from both new and existing prescribers. Lalwani clarified his earlier remark, stating that the rare disease business is expected to be the 'largest driver of growth' for ANI moving forward, rather than necessarily the largest segment by revenue.

Vamil Divan from Guggenheim Securities asked about the potential scope of the FILSPARI label for FSGS, questioning whether it would be for all patients or include specific restrictions.

Answer

Chief Research Officer Dr. William Rote stated his expectation that the indication would be broad, covering the treatment of FSGS in patients aged 8 and up, consistent with the DUPLEX study's population. He emphasized that the study included various subtypes, like primary and genetic FSGS, and showed consistent effects, supporting a broad label.

Vamil Divan asked about the potential competitive impact on FILSPARI from new entrants in the IgAN space, such as Novartis's atrasentan, particularly concerning uptake and differences in liver safety monitoring requirements.

Answer

CEO Eric Dube and CCO Peter Heerma expressed confidence in FILSPARI's competitive profile. Heerma highlighted that despite new competition like Fabhalta, Q4 was their strongest quarter. He differentiated FILSPARI by its dual-action mechanism, superior long-term kidney preservation data from the PROTECT study, and flexible dosing. He anticipates new competitors will initially have narrower labels for high-risk patients, positioning FILSPARI as a foundational therapy. Dube added that the company is prepared for any REMS scenario.

An associate for Vamil Divan asked about the planned healthy volunteer study for ABS-101, specifically what the company aims to achieve regarding antibody half-life and what they consider the target benchmark.

Answer

SVP of Drug Creation Christian Stegmann explained the study will measure pharmacokinetics. He stated the goal is to achieve a half-life profile similar to other half-life engineered antibodies, which would support dosing levels ranging from once monthly to potentially once quarterly.

Vamil Divan from Mizuho Securities asked if the Pfizer agreement contained a standstill clause and commented on recent new-to-brand (NBRx) share trends. He also inquired about the sales split between treatment and prevention and if the Zavegepant program was included in the Pfizer deal.

Answer

CEO Dr. Vlad Coric confirmed there is no standstill clause and that Zavegepant rights are included in the Pfizer agreement. Dr. Coric and CCO Bj Jones acknowledged NBRx volatility but emphasized their commanding 57% market share. Dr. Coric added that the company does not break down sales by indication, as its strategy is to position NURTEC ODT as a single, unified migraine therapy.

Ari for Vamil Divan of Mizuho Securities asked for expectations on the upcoming Zavegepant Phase 3 readout, its potential market positioning, and for an update on the European Union regulatory filing for NURTEC ODT.

Answer

CEO Vlad Coric described Zavegepant as complementary to NURTEC. CMO Dr. Elyse Stock added that the intranasal formulation is being developed for ultra-rapid relief, which is ideal for patients with severe nausea. She confirmed a filing is expected by year-end. Regarding the EU filing, she stated the process is on track for a potential approval in early 2022 and is proceeding as expected.

Vamil Divan of Mizuho Securities requested an update on European partnership discussions for NURTEC ODT and asked about the migraine frequency of current patients, questioning if a prevention label would attract a more severe population.

Answer

CEO Vladimir Coric confirmed that partnership discussions with both regional and global players are ongoing but declined to give a timeline. He noted that over 65% of current patients have less frequent migraines, representing a large acute-need market, and expects the prevention indication will attract new patient segments and redefine treatment paradigms.