Jason Gerberry

Jason Gerberry of Bank of America questioned why the Indore facility issues would impact revenues outside the United States and asked about the 2025 generic pipeline assumptions, including the impact of competition on generic Symbicort (Breyna) and the overall risk profile of the pipeline's contribution.

Answer

CEO Scott Smith explained that active remediation at the Indore facility can cause manufacturing pauses and supply shortages for certain products shipped to markets like Europe. CFO Theodora Mistras stated the $450M-$550M new product revenue range is 'derisked,' with 20% from already approved products. Chief Commercial Officer Corinne Le Goff added that Breyna is expected to remain a key contributor in 2025.

Jason Gerberry of Bank of America inquired about a recent investigation into a competitor in China, asking if it was an isolated incident or a broader crackdown. He also requested updates on the approval timelines for Sandostatin LAR and the resubmission status of GA Depot.

Answer

CEO Scott Smith stated that Viatris could not comment on another company's investigation but emphasized Viatris's high internal standards and strong compliance structure in China. Chief R&D Officer Philippe Martin added that a meeting with the FDA regarding GA Depot is set for mid-December, with an update expected next year, and that the Sandostatin LAR generic is still under review with a launch anticipated next year.

Jason Gerberry asked about the dynamic between oral therapies and Lutathera in second-line neuroendocrine tumors (NET), and if orals are gaining a greater share. He also inquired about the timeline for the MSI-H patent appeal.

Answer

PJ Haley, EVP of Commercial, noted that orals constitute a greater portion of the second-line plus NET market, and Cabozantinib is preferred post-Lutathera due to its unique inclusion of Lutathera-pretreated patients in its study. Michael Morrissey, President and CEO, stated he had nothing to offer on the MSI-H patent appeal timeline.

Jason Gerberry asked whether oral therapies are gaining a larger share relative to Lutathera in the neuroendocrine tumor (NET) market following the Cabozantinib launch, or if the dynamic between them remains stable. He also inquired about a timeline for the MSI-H patent appeal.

Answer

PJ Haley, Executive Vice President of Commercial, noted that Cabometyx has exceeded 40% new patient share in the second-line plus oral segment of NETs and expects continued growth. He explained that while orals constitute a larger portion of the overall second-line plus market, Cabozantinib is preferred post-Lutathera due to its unique inclusion of Lutathera-pretreated patients in its study. Michael Morrissey, President and CEO, declined to offer a timeline on the MSI-H patent appeal.

Jason Gerberry from Bank of America Securities asked about the enrollment demographics of the STELLAR-303 trial, particularly the proportion of patients with non-liver metastases, and requested an estimate of the U.S. patient market size for adjuvant CRC.

Answer

EVP & CMO Amy Peterson declined to share specific enrollment details. CEO Michael Morrissey did not provide a patient number for adjuvant CRC but stated the commercial opportunity for the new adjuvant CRC and meningioma studies is approximately three times greater than the discontinued head and neck trial, justifying the strategic pivot.

Jason Gerberry followed up on the zanzalintinib $5 billion peak sales forecast, noting investor difficulty in reconciling the 'non-overlap' comment with RCC being a large market, and asked for clarity on the development strategy.

Answer

President and CEO Michael Morrissey reiterated that the focus should be on the pivotal trials and their specific comparators, which are contemporary standards of care. He stressed that the RCC treatment landscape will evolve by 2030 and that Exelixis is planning its trials to define future standards of care, rather than focusing on simple comparisons between zanzalintinib and cabozantinib.

Jason Gerberry asked about maximizing the benefit of Epilum Tersen's CardioTransform data, particularly in combination with Tafamidis, to ensure disproportionate benefit and avoid 'free-riding' by competitors. He also asked for context on the Number Needed to Treat (NNT) data for Olzarsen in SHTG, considering both all-comer and high-risk groups, and its importance for payers.

Answer

Brett Monia, CEO, highlighted CardioTransform as the largest TTR cardiomyopathy study, with combination usage as a key secondary endpoint, expecting added benefit over monotherapy. He stated that NNT data for Olzarsen would be shared at AHA, showing strikingly positive results across subgroups, but did not want to preempt the data. Kyle Jenne, Chief Global Product Strategy Officer, emphasized that the totality of Olzarsen's data, including triglyceride reduction, AP reduction, hospitalizations, and visits, would drive payer engagement and effective reimbursement, focusing on preventing first AP attacks.

Jason Gerberry inquired about the pelacarsen program, asking how the addressable market might change if the primary endpoint is met in the subgroup with Lp(a) levels above 90 mg/dL versus 70 mg/dL. He also asked about the confidence in the mid-2025 data readout timeline.

Answer

Executive Kyle Jenne, while noting the questions were best for partner Novartis, stated that an indication for the >90 mg/dL subgroup would still represent a 'massive' patient population. He confirmed that Novartis's confidence in the 2025 readout timeline is based on regular monitoring of blinded event rates in the ongoing study.

Jason Gerberry of Bank of America Merrill Lynch inquired about the specific data points planned for the September REMAIN-one midpoint update, beyond weight maintenance, and asked for an update on the Rejuva IND clearance status.

Answer

Harith Rajagopalan, Co-Founder, CEO & Director, stated the update will cover safety, tolerability, and weight maintenance effectiveness, with enough detail to assess the pivotal study's outlook. He clarified that DEXA scans occur at six and twelve months, not three. Regarding Rejuva, he noted that GMP product lot release is weeks away and the company remains on track for preliminary data in 2026.

Jason Gerberry from Bank of America asked about gross-to-net expectations for the second half of the year, the reasons for the 25% of prescriptions that have not yet converted to starts, and the company's view on using Symphony data going forward.

Answer

CFO & COO Michael Kaseta declined to project gross-to-net but noted that as new-to-market blocks are removed with major payers, more prescriptions will be subject to rebates. He attributed the non-converted scripts primarily to temporary headwinds like these blocks, which are expected to ease. He also stated the company will not rely on Symphony data for reporting and will provide its own metrics on quarterly calls.

Jason Gerberry from Bank of America asked about potential friction in payer coverage for the YUTREPIA launch and whether the INCREASE study serves as a suitable framework for the L606 pivotal trial design.

Answer

CFO Michael Kaseta expressed confidence in achieving broad market access for YUTREPIA close to launch, citing ongoing payer engagement. Chief Medical Officer Dr. Rajeev Saggar confirmed the INCREASE study is a 'replicable' model for the L606 trial, which is planned as a global study with 300-400 patients. CEO Dr. Roger Jeffs added that L606's unique formulation should enable higher dosing and potentially superior outcomes compared to those observed in the INCREASE study.

Jason Gerberry of Bank of America asked about the company's flexibility to preserve its cash runway if the YUTREPIA launch is delayed, and inquired about any plans to submit a public response to United Therapeutics' citizens' petition.

Answer

CFO Michael Kaseta affirmed confidence in the company's $133 million cash position to fund all current objectives, including the YUTREPIA launch and clinical trials, but noted they can make decisions to adjust spending if necessary. General Counsel Russell Schundler stated that the company does not currently anticipate filing a public response to the citizens' petition, handling any necessary communications directly with the FDA.

Jason Gerberry of Bank of America Merrill Lynch inquired about the blinded demographics of the EXTOL-two trial, specifically patient severity, the potential for site overlap between the MDD and BPD studies to speed up enrollment, and whether the Phase I pain studies would use an induced challenge model for efficacy assessment.

Answer

President and CEO Ian Mortimer confirmed that blinded patient characteristics in EXTOL-two look very similar to the original EXTOL study and stated they will not use induced pain challenges in Phase I due to variability. Chief Medical Officer Christopher Kenney noted there would be some site overlap between the MDD and BPD trials to leverage resources, but it is not a primary driver of the strategy.

Jason Gerberry of Bank of America inquired if the bipolar depression (BPD) program would evaluate Azetukalner as an adjunctive therapy and whether the second-generation Kv7 program aims to find novel indications or a follow-on to Azetukalner.

Answer

President and CEO Ian Mortimer deferred specifics on the BPD trial design (mono vs. adjunctive) until closer to the study's initiation. Regarding the next-gen Kv7 program, he explained that since Azetukalner has a strong profile, the goal is not to fix a specific problem but to build on its success. He sees the potential for these new molecules to be used in both existing indications like epilepsy and neuropsychiatry, as well as new areas like pain.

Jason Gerberry asked about the strength of Xenon's food effect patent for azetukalner and the company's confidence in securing label language recommending dosing near mealtime.

Answer

CFO Sherry Aulin expressed high confidence in their patent portfolio, which extends to 2039/2040. She noted the food effect is unique to azetukalner and not a class effect. Because the drug has been consistently dosed with food in all Phase II and Phase III trials, the company reasonably expects this instruction to be included in the final product label, which is key for both efficacy and tolerability.

Jason Gerberry asked for confirmation that Arrowhead's Phase 3 SHTG trial baseline demographics are similar to a competitor's and questioned the potential to show a trend in pancreatitis events.

Answer

Interim Chief Medical Scientist Bruce Given clarified that comparing pancreatitis event data is difficult because Arrowhead counts true adjudicated cases, whereas the competitor includes less specific abdominal pain events. He emphasized that pancreatitis is the more severe and important measure and confirmed Arrowhead will publish its baseline demographics at a future medical conference.

Jason Gerberry of Bank of America asked about the potential for plozasiran's pancreatitis reduction data to be prominently featured on its FDA label and how this might differentiate it from competitors.

Answer

Interim Chief Medical Scientist, Dr. Bruce Given, explained that labeling negotiations with the FDA have not yet occurred. He highlighted that Arrowhead's PALISADE study used stricter, confirmed pancreatitis criteria, which could be a key differentiator. President and CEO, Dr. Christopher Anzalone, added that the primary goal is lowering triglycerides, noting the impressive reductions achieved in the Phase III study.

Jason Gerberry asked about the commercial prospects for plozasiran in FCS during the initial 2-3 year launch window, questioning if revenues could be meaningful or if the launch is primarily an educational effort ahead of the larger SHTG indication.

Answer

Andy Davis, SVP and Head of Global Cardiometabolic Franchise, expressed strong confidence in the FCS launch, calling plozasiran a 'game changer' and expecting a return on investment within that timeframe. He also highlighted the significant physician overlap between FCS and SHTG, meaning the initial launch activities will directly support the future, broader indication.

Jason Gerberry asked if the new debt facility provides flexibility to use milestone payments to retire debt and avoid interest costs. He also questioned if the ARO-TSLP program is redundant with ARO-RAGE and what that implies about confidence in ARO-RAGE.

Answer

President and CEO Dr. Christopher Anzalone positioned the facility as one of several tools to bridge to SHTG commercial revenue, noting some but not all future deal proceeds might be used for repayment. Regarding ARO-TSLP, Dr. Anzalone and Dr. James Hamilton clarified it was primarily investigated as a component for a future dimer therapeutic, not as a standalone program, and that RAGE is mechanistically upstream of TSLP.

Jason Gerberry asked how recent developments with Elevidys might impact the quarterly uptake of Agamry and questioned the expected time lag for the new NCCN recommendation for Firdapse to have a meaningful commercial impact.

Answer

EVP & CCO Jeff Del Carmen and CMO Dr. Will Andrews clarified that Elevidys developments do not impact Agamry, as corticosteroids are a foundational and different modality of treatment. President & CEO Richard Daly and Jeff Del Carmen explained the NCCN guideline impact would follow a cadence, starting with frictionless testing, then embedding the guidelines into oncology care pathways, with a commercial revenue increase expected in the second half of 2026 and beyond.

Jason Gerberry from Bank of America, after joining the call late, asked for clarification on whether FIRDAPSE's strong quarterly performance involved any sales pull-forward and inquired about the adoption momentum in the small cell lung cancer LEMS population.

Answer

CFO Michael Kalb and CEO Richard Daly reiterated that while the year-over-year comparison was flattered by the Q1 2024 Change Healthcare incident, the underlying organic growth for FIRDAPSE remains in the 15-20% range. CCO Jeffrey Del Carmen repeated that the focus for the SCLC-LEMS segment in 2025 is on establishing screening programs, with patient growth expected to materialize in 2026.

Pavan, on behalf of Jason Gerberry, asked about the source of FIRDAPSE's patient uptake (idiopathic vs. small cell lung cancer), the stability of its patient pipeline, and whether AGAMREE's peak sales potential could exceed its competitor, EMFLAZA.

Answer

CCO Jeffrey Del Carmen stated that the small cell lung cancer LEMS patient mix is stable at 20-25% of total patients and that the pipeline of approximately 500 diagnosed patients has been consistently replenished for the last 2-3 years. CEO Rich Daly affirmed that EMFLAZA remains a good surrogate for AGAMREE's potential, though Catalyst believes it has a superior product, which the SUMMIT study aims to prove over time.

Jason Gerberry of Bank of America Merrill Lynch questioned the Q4 volume growth outlook, the specialty mix of new prescribers, and whether the revised peak sales potential implies a major step-up from prior guidance.

Answer

CFO Jeff Farrow advised that using last year's Q3-to-Q4 growth rate would be too aggressive given the higher current baseline, expecting more measured growth. CCO Aziz Mottiwala stated the new prescriber mix is consistent with the overall base (approx. 60% optometry, 40% ophthalmology). CEO Bobak Azamian noted it's too early to provide a new peak sales figure but expects Xtendi to be one of the largest eye drops ever.

Jason Gerberry asked about the potential size and cost of a Phase III prevention study for the Lyme disease program (TP-05) to gauge the required investment. He also requested a breakdown of the 15,000 prescribing ECPs into low versus high prescribers.

Answer

Chief Operating Officer Seshadri Neervannan stated that while the FDA guided to a Phase III study with 'thousands of patients,' the final design and cost will be determined after the Phase II study is complete. Chief Commercial Officer Aziz Mottiwala noted it is too early to segment the prescriber base, as the focus in 2024 was on broad adoption, and the 2025 goal is to drive prescribing depth with the newly expanded sales force and improved market access.

Jason Gerberry of Bank of America asked for clarification on the expected impact of Medicare Part D coverage expansion in 2025 and whether payers are imposing limits on the frequency of patient retreatments.

Answer

CEO Bobak Azamian characterized the Part D expansion as a steady build throughout 2025 rather than an immediate "light switch" effect, citing Q1 plan resets as a moderating factor. He stated that they do not expect payer pushback on retreatments, as the potential for multiple treatments per year was factored into contracting discussions, and retreatment itself validates the drug's effectiveness for that patient.

Jason Gerberry of Bank of America Merrill Lynch questioned the importance of the frontline pancreatic cancer (PDAC) opportunity in the upfront consideration for the Revolution Medicines deal and asked to identify the single biggest variable in the 2025 portfolio receipt guidance range.

Answer

Head of Research & Investments Marshall Urist confirmed the frontline PDAC opportunity is exciting and was contemplated in the deal structure, with a funding tranche tied to positive Phase III data in that setting. CFO Terrance Coyne explained that the guidance range incorporates a variety of scenarios, citing Promacta generics, Part D redesign, and FX as key factors, rather than a single variable.

Jason Gerberry of Bank of America Securities asked for context on the clinical benefit of ecopipam, whether its market opportunity is comparable to tardive dyskinesia, and if there is a time limit for Royalty Pharma to bring a claim against Vertex in their dispute.

Answer

EVP Marshall Urist highlighted that the Tourette's market has lacked innovation and that the tardive dyskinesia market is an interesting parallel for how a new therapy can drive market growth. EVP, CFO Terrance Coyne stated that the company could not comment on the timing of any potential dispute resolution with Vertex.

Speaking on behalf of Jason Gerberry, Dina inquired about the materiality of the IRA Part D redesign's impact on the portfolio and whether any products are mis-modeled by the Street. She also asked for color on the probability of the Vertex matter being resolved amicably versus proceeding to a formal arbitration.

Answer

EVP, Head of Research and Investments Marshall Urist stated that the portfolio's overall IRA exposure is modest and that the impact on key products like Imbruvica, Xtandi, and Trelegy was expected. EVP and CFO Terrance Coyne addressed the Vertex question by stating he could not provide specifics on the arbitration process or the likelihood of an alternative resolution at this time.

Jason Gerberry of Bank of America Merrill Lynch asked if Organon still plans to invest in the endometriosis space following the setback with molecule 6219. He also inquired about the FDA's product-specific guidance for a generic Nexplanon, specifically whether a five-year release study would become mandatory after the new indication is approved.

Answer

Juan Camilo Ferreira, Head of R&D, confirmed that due to the lack of an efficacy signal with 6219, the backup molecule program has also been discontinued. Regarding Nexplanon, he stated Organon doesn't comment on future FDA decisions but is working to get the right labeling. CEO Kevin Ali added that the market would likely move securely to the five-year product, creating another hurdle for potential generics.

Jason Gerberry's team inquired about Organon's view on the NEXPLANON Paragraph IV filing, asking about the filer's credibility and the FDA's position on applicator similarity. They also requested a breakdown of 2025's onetime costs and the free cash flow outlook for 2026.

Answer

CFO Matt Walsh detailed 2025 onetime costs, including ~$150M for manufacturing separation and ~$200M for restructuring. For NEXPLANON, CEO Kevin Ali and Head of R&D Juan Camilo Arjona Ferreira expressed confidence, citing a strong applicator patent through 2030 and significant regulatory hurdles for a generic, including demonstrating bioequivalence and applicator safety, which they believe sets a very high bar for FDA approval.

Jason Gerberry sought details on the source of the $200 million in OpEx savings and future restructuring opportunities. He also asked for clarification on the 2025 NEXPLANON guidance, which seems conservative given its long-term growth potential.

Answer

CFO Matt Walsh explained the savings come from holistic organizational streamlining, with 75% impacting OpEx and 25% COGS in 2025. CEO Kevin Ali reiterated that NEXPLANON's growth to $1.5 billion by the decade's end will be driven by high single-digit growth, with some years potentially hitting low double-digits, making the 'greater than $1 billion' 2025 forecast a prudent target.

Jason Gerberry sought clarification on the Dermavant deal's financial profile, questioning how Organon's projected $180 million in OpEx compares to Dermavant's prior disclosures, and asked for the backstory on the recently filed NEXPLANON Citizen's Petition.

Answer

Chief Financial Officer Matthew Walsh explained that Organon is onboarding approximately $240 million in costs, and the $180 million OpEx figure for 2025 reflects synergies and the roll-off of prior R&D costs. Chief Executive Officer Kevin Ali addressed the petition by emphasizing NEXPLANON's applicator patent protection until 2030 and the significant infrastructure investment required for any generic competitor, suggesting a long runway for the brand.

Bhavan Patel on behalf of Jason Gerberry at Bank of America asked about modeling patient adherence and discontinuation rates for ZYN-two based on prior data. He also asked which secondary endpoints would provide the most conviction for a regulatory filing.

Answer

Chief Medical & Scientific Officer Dr. Kumar Budur emphasized the strong data for the irritability subscale as a key secondary endpoint, citing a recent presentation showing sustained benefit. He highlighted very high adherence, with over 90% of eligible patients from prior studies opting into the long-term extension, and noted the product's favorable tolerability profile. He did not have the specific discontinuation rate from the CONNECT study immediately available.

Jason Gerberry asked for a preview of the preclinical orexin data to be presented at the upcoming sleep meeting and questioned how amenable payers are to covering multiple branded narcolepsy drugs.

Answer

Dr. Kumar Budur, CMSO, confirmed the presentation will include comprehensive preclinical data with standard metrics like sleep latency, supporting the drug's high potency. CEO Dr. Jeffrey Dayno explained that because narcolepsy is a rare disease, covering multiple branded therapies for a small number of patients per plan has not been a significant budget issue or challenge for payers.

Jason Gerberry asked if EPX-100 was compared to a competitor in the zebrafish model and inquired about the key outcomes of the upcoming WAKIX IP litigation Markman hearing.

Answer

Chief Medical and Scientific Officer Dr. Kumar Budur stated that while they have not tested the competitor's compound directly, their own assets (EPX-100 and EPX-200) showed great efficacy in the model. CEO Dr. Jeffrey Dayno declined to comment on active litigation but clarified the Markman hearing's purpose is to establish claim construction for the case, reiterating confidence in the company's IP portfolio.

Jason Gerberry of Bank of America Merrill Lynch asked about the commercial opportunity in hypochondroplasia (HCH), questioning if the milder nature of the disease might lead to later treatment initiation compared to achondroplasia.

Answer

EVP and CCO Cristin Hubbard acknowledged that HCH patients are often diagnosed later but stressed a significant unmet need exists due to comorbidities. EVP and R&D Officer Gregory Friberg added that the pivotal study includes patients from age 3 to 18, an infant study is ongoing, and the company is working to publish data on the condition's healthcare burden to create an urgency to treat.

Jason Gerberry from Bank of America asked about the commercial opportunity in hypochondroplasia (HCH), noting the challenge of treating patients earlier given its milder nature and later average age of diagnosis compared to achondroplasia.

Answer

EVP & CCO Cristin Hubbard acknowledged that HCH patients are diagnosed later but stressed there is still a large unmet need due to comorbidities. She said BioMarin is focused on educating about this need to encourage earlier diagnosis. EVP & Chief R&D Officer Gregory Friberg added that the company is actively working to publish data on the healthcare burdens of HCH to create an urgency to treat.

Jason Gerberry questioned the revenue recognition for Cimbravo, asking how to model the next few quarters given the co-pay card impact and the new contracting status.

Answer

Chief Financial Officer Nick Pizzie explained that Q2 revenue included initial wholesaler stocking orders with a gross-to-net in the low 80% range. Chief Commercial Officer Ari Maizel added that they are already seeing covered claims, so not all patients are relying on co-pay support, though refill velocity will be lower than Auvelity's.

Jason Gerberry from Bank of America questioned the assertion that MDD with EDS comprises half the MDD population, given it was a small subgroup in a recent study. He also asked when Axsome might narrow its $1-3 billion peak sales guidance for Auvelity and what key variables would determine the final outcome within that range.

Answer

Herriot Tabuteau, CEO, clarified that the study subgroup was defined as patients with *severe* EDS to ensure specificity, whereas the broader MDD with EDS population is estimated at 50%. Regarding the peak sales guidance, he stated that the current sales trajectory is a key variable to watch and that the company feels the stated range is very achievable.

Dina, on behalf of Jason Gerberry, asked if the $1-$1.5 billion peak sales estimate for solriamfetol in ADHD would change if the drug showed stimulant-like efficacy, and also inquired about plans for the adolescent ADHD population.

Answer

CEO Dr. Herriot Tabuteau stated that the ADHD market is large and underserved, representing a sizable opportunity regardless, and that the company looks forward to seeing the data. He confirmed that generating pediatric data is required for an NDA submission and that the company is in the process of getting FDA feedback on its pediatric study plan.

Jason Gerberry of Bank of America asked about the potential competitive threat from new atypical antipsychotics to Auvelity and how Axsome plans to overcome the high out-of-pocket cost hurdle in the Medicare-heavy Alzheimer's Disease Agitation (ADA) market.

Answer

An executive, likely CCO Ari Maizel, responded that Auvelity is positioned as a first or second-line monotherapy, distinct from later-line adjunctive atypicals. Regarding ADA, the strategy is to leverage strong payer relationships developed for Auvelity and Sunosi to help mitigate out-of-pocket costs for a future ADA launch, though it is premature to specify co-pay amounts.

Jason Gerberry from Bank of America Merrill Lynch inquired about the status of share buybacks as a capital deployment option and asked for clarification on the focus of the XIFAXAN DTC campaign, specifically whether it targeted the IBS-D or OHE indication.

Answer

CFO Jean-Jacques Charhon explained that deleveraging and reinvesting in the business, such as the recent Direct Corporation acquisition agreement, are the primary capital allocation priorities, moving share buybacks to the "back burner." CEO Thomas Appio clarified that the current DTC investment is focused on the OHE indication for XIFAXAN, which is driving significant volume growth and new patient starts.

Jason Gerberry asked about the feasibility of moving Solta's consumable manufacturing to mitigate potential China tariffs, sought quantification of onetime items affecting Q1 EBITDA, and inquired about where share buybacks rank in capital allocation priorities.

Answer

CEO Thomas Appio explained that moving Solta's highly precise manufacturing from the U.S. is difficult in the short term but is a long-term consideration. CFO JJ Charhon stated the onetime EBITDA benefits were related to expense timing and gross-to-net adjustments. He reiterated that the primary capital allocation priority is optimizing the capital structure, but given the current stock price, share buybacks are being evaluated as an option to unlock shareholder value.

Jason Gerberry revisited the topic of capitalization targets, asking if RemainCo's leverage is a moving target and if extending the 2027-2028 debt maturities is critical. He also questioned the flat XIFAXAN script growth, asking if it's time to optimize for cash flow over growth, and how to reconcile strong new prescriptions (NBRx) with flat total prescriptions (TRx).

Answer

Interim CFO John Barresi confirmed that managing the 2027 and 2028 maturities is a key focus. CEO Thomas Appio defended XIFAXAN investment, citing strong NBRx growth, a large untreated patient population, and the impact of new commercial initiatives. He attributed the TRx/NBRx divergence to a market shift toward non-retail channels and growth in extended units.

Jason Gerberry of Bank of America Merrill Lynch asked about the importance of the TETON trial showing a meaningful improvement on exacerbations and a statistically significant benefit in the monotherapy subgroup, in light of recent competitor data.

Answer

Dr. Leigh Peterson, VP of Product Development, responded that while competitor data is noted, the TETON trial is 80% powered to detect an 80-milliliter change in FVC. She stated the company fully anticipates achieving a clinically meaningful effect greater than that threshold and that the blinded results will be available in September.

Jason Gerberry from Bank of America Merrill Lynch questioned the AUSTEDO revenue trend relative to its volume and mix shift, its impact on gross-to-net, and asked about Teva's reliance on China for key starting materials in light of potential tariffs.

Answer

President and CEO Richard Francis explained that AUSTEDO's revenue dynamics are playing out as expected, with some Q2 2024 stocking affecting comparisons, but declined to detail IRA pricing. EVP & CFO Eli Kalif asserted that Teva has almost no reliance on China for materials, having divested its API business there years ago, which strengthens its supply chain.

Jason Gerberry from Bank of America Merrill Lynch asked about the PK/cataplexy relationship, whether a BID dosing approach could offer an advantage, and if the 8mg dose's safety profile allows for potentially higher doses in future NT1 studies.

Answer

Marcus Yountz, VP of Clinical Development, stated that analyses are ongoing but the company does not believe there is a meaningful difference between QD and BID dosing. He also confirmed full confidence in the 8mg toxicology profile, which is being factored into Phase 3 dose selection.

Jason Gerberry sought clarification on the company's exposure to potential tariffs and transfer pricing reform, and asked what might be learned about visual disturbance adverse events from the Vibrance-1 study's ophthalmic testing.

Answer

COO Blair Jackson confirmed that with U.S. manufacturing and minimal API imports, tariffs are not a material risk, and transfer pricing reform would likely require complex legislation. CEO Richard Pops added that all Phase II patients undergo visual exams and the DSMB, which includes an ophthalmologist, has cleared the study to proceed without changes.

Jason Gerberry asked for a general assessment of the overall health and growth outlook for the long-acting injectable (LAI) antipsychotic market, citing cautionary language and competitive actions in the space.

Answer

CCO Todd Nichols responded that while the treatment algorithm for LAIs hasn't changed (typically later-line use), the market is seeing lower overall growth, which he attributed partly to seasonality and lower schizophrenia patient visits. He noted that new brand launches are primarily causing shifts within the market rather than expanding it. He also acknowledged payer pressure from the Part D redesign is a topic of ongoing discussion.

Jason Gerberry sought clarification on the flat sales outlook for 2025, questioning the BAQSIMI growth forecast given flat Q1 results, and asked if Q1's pressured margins are a good run rate for the year.

Answer

CFO William Peters reiterated that the forecast relies on sales from two of three potential new product launches. Executive Tony Marrs clarified that FDA response times could be 90 days for a minor issue or 8-10 months for a major one. Peters explained last year's Q1 BAQSIMI sales were inflated by channel loading during the Lilly transition and that recent weekly data shows a return to strong growth. He noted that while glucagon will see more price pressure, this should be offset by growth in higher-margin products like Primatene MIST and BAQSIMI, along with internal cost-saving measures.

Jason Gerberry from Bank of America Corporation inquired about Amphastar's 2025 outlook for its key products, Primatene Mist and glucagon, given the company was not providing formal guidance.

Answer

William Peters, CFO and Executive Vice President of Finance, responded by forecasting high single-digit sales growth for Primatene Mist. For BAQSIMI, he noted a recent 3% U.S. price increase and anticipated high single-digit unit growth. Conversely, he projected that glucagon would experience declines in both pricing and unit volume due to mounting competitive pressures.

Jason Gerberry of Bank of America inquired about Jazz's Xywav supply chain, specifically asking if its U.S. contract manufacturer has the capacity to fully supply the U.S. market to mitigate potential tariffs and if the API can also be sourced domestically.

Answer

Bruce Cozadd, Chairman and CEO, confirmed that Jazz Pharmaceuticals has a U.S. supplier for oxybate products, including Xywav, with sufficient capacity to meet all U.S. demand. He stated this would be a very effective option to mitigate tariff exposure and noted no particular issues regarding API sourcing being subject to tariffs.

Jason Gerberry of Bank of America questioned the company's M&A strategy, asking why Jazz has not executed a large-scale transaction since the GW Pharmaceuticals acquisition and how the new Epidiolex patent settlement, which secures longevity into the next decade, alters its approach to M&A.

Answer

Bruce Cozadd, Chairman and CEO, reminded that the post-GW period was focused on deleveraging and highlighted the zanidatamab deal as a key strategic transaction. Philip Johnson, CFO, added that net leverage is now down to 1.8x, providing significant financial capacity for M&A. He stated that the extended Epidiolex runway makes the epilepsy franchise an even more attractive area for further investment.

Jason Gerberry from Bank of America questioned the company's M&A strategy, asking why no major deals have occurred since the GW acquisition and how the extended Epidiolex patent life alters their approach.

Answer

CEO Bruce Cozadd noted the company focused on deleveraging post-GW and highlighted the zanidatamab deal as a significant transaction. CFO Phil Johnson added that leverage is now down to 1.8x, providing significant capacity for M&A. He stated the Epidiolex longevity makes the epilepsy franchise an even more attractive area for investment.

Jason Gerberry from Bank of America asked for clarification on the regulatory pathway for Zepzelca in first-line small cell lung cancer and the potential commercial opportunity.

Answer

EVP, Global Head of R&D, Rob Iannone, explained that the positive Phase III results are expected to be sufficient for a standalone label expansion into first-line maintenance. He noted this could also serve as a confirmatory trial for the second-line accelerated approval. The commercial opportunity is significant, targeting a larger patient pool of approximately 27,000 extensive-stage patients annually with a potentially longer duration of therapy.

Jason Gerberry asked for clarification on Zepzelca's regulatory path for first-line small cell lung cancer, including whether the submission would be based solely on the new Phase III results and if this data could serve as confirmatory for the second-line accelerated approval. He also sought to frame the commercial opportunity.

Answer

EVP, Global Head of R&D Robert Iannone stated that the positive Phase III data alone are expected to support a first-line label expansion and could potentially serve as confirmatory for the second-line approval, depending on timing. He confirmed the first-line setting represents a larger patient population and likely a longer duration of therapy, expanding the overall opportunity.

Dina Ramadane, on for Jason Gerberry at Bank of America, asked about the read-through from VAX-24 infant data to the VAX-31 program, the flexibility to adjust doses for Phase III, and the specific political and regulatory hurdles required to change the PCV vaccination schedule.

Answer

CEO Grant Pickering stated that the VAX-24 data, especially from the mixed-dose cohort, will be a key indicator for the VAX-31 infant program, and confirmed they can tweak individual serotype doses for Phase III. On policy, Pickering and Executive Andrew Guggenhime asserted that changing the PCV schedule is highly unlikely given the clear public health need and the complex, multi-stakeholder system (ACIP, AAP) that relies on scientific evidence to set recommendations.

Asked about the reasons for the quarterly step-down in Q4 revenue, the dynamics of converting patient starts to revenue, and the expected flow of new patient starts in the first half versus the second half of 2024.

Answer

The company explained that the Q4 revenue dip was due to the timing of patient infusions, with several patients collected in late 2023 scheduled for infusion in Q1 2024. They expect a linear build in patient starts for LYFGENIA throughout 2024, with the majority occurring in the second half of the year. They also noted that Q1 2024 has seen the strongest quarter for patient starts to date.

Asked for clarification on enrollment numbers for proact 1, the likelihood and nature of an interim analysis, and potential financing catalysts in the absence of Phase III interim data.

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The company clarified that over 80 patients are enrolled, with about 50 meeting the new criteria, and they are targeting 600 new patients. A decision on a Phase III interim analysis is pending, but it wouldn't yield public data. Key financing catalysts are the upcoming data from the 007 and 002 studies in 2024 and 2025.

Jason Gerberry of Bank of America inquired about the history of the DUBLIN-3 trial, asking about any protocol changes, interim analyses, and whether the primary endpoint was ever modified from Kaplan-Meier OS. He also asked about the regulatory view on a meaningful clinical benefit if supportive median OS results were weak, even with a statistically significant Kaplan-Meier curve.

Answer

CEO Lan Huang explained that the DUBLIN-3 protocol was updated during the trial to include and stratify for PD-1/PD-L1 failed patients as the treatment landscape evolved. She asserted the primary endpoint was always overall survival analyzed by the log-rank P-value, which was never changed, and that the final p-value met the required statistical significance after accounting for two interim analyses. For full transparency, both median OS and restricted mean survival time will be presented at ESMO.

Jason Gerberry of Bank of America inquired about the company's confidence level in avoiding an FDA advisory committee (AdCom) for plinabulin, given its new mechanism of action. He also asked about the potential for using the plinabulin plus I/O triple combination in non-small-cell lung cancer (NSCLC).

Answer

Dr. Lan Huang, Co-Founder, Chairwoman and CEO, expressed confidence in avoiding an AdCom, citing a successful FDA inspection with no 483s and a positive orientation meeting with the agency. She also highlighted data correlating plinabulin's endpoints with clinical outcomes. Dr. Ramon Mohanlal, EVP of R&D and CMO, confirmed that moving the combination into first-line NSCLC is part of the plan, noting plinabulin's unique potential to increase survival while preventing both neutropenia from chemo and immune-related events from I/O agents.

Jason Gerberry from Bank of America asked about the company's launch readiness for plinabulin, particularly in a potential accelerated review scenario, considering cash burn and hiring plans. He also questioned if the DUBLIN-3 topline results would include ORR and PFS data for comparison purposes and asked for the rationale behind the small cell lung cancer trial.

Answer

Richard Daly, COO, explained that the company is preparing for launch but will be judicious, likely hiring sales reps upon approval to manage costs, and suggested a 2022 launch might be more effective than a late Q4 launch. Dr. Lan Huang, CEO, stated that while ORR and PFS data from DUBLIN-3 are important for comparison, the decision to release them depends on the data quality. Dr. Ramon Mohanlal, CMO, and Dr. Huang elaborated that the small cell lung cancer trial aims to show that plinabulin can enhance survival and reduce immune-related adverse effects in combination with checkpoint inhibitors, including in patients who have already failed such therapies.

Jason Gerberry from Bank of America asked for details on the key agenda items for the upcoming end-of-Phase 2 meeting with the FDA, including potential variables and the company's strategy for study size and inclusion criteria to ensure a broad label covering both polyp and non-polyp patients.

Answer

President and CEO Maria Palasis explained that the primary agenda item for the FDA meeting is the primary endpoint, for which they will propose a composite of cardinal symptoms at a later time point up to 24 weeks. She added that discussions will also cover key secondary endpoints, such as rescue treatments, and a planned study size of approximately 350 patients using the 7,500 microgram dose to ensure statistical power.