Raghuram Selvaraju

Raghuram Selvaraju from H.C. Wainwright asked about the expected size and scope of the commercial infrastructure for imsidolimab, assuming timely approval, and sought additional details on promotional activities for Fanapt and Bysanti, including any planned direct-to-consumer campaigns for 2026. He also inquired about Nereus's commercial availability, the regulatory outlook for tradipitant in gastroparesis, and the timeline for completing enrollment in the GLP-1 phase III trial. Finally, he asked about the anticipated completion of enrollment for the iloperidone LAI phase III program by year-end.

Answer

Dr. Mihael Polymeropoulos, CEO, stated that imsidolimab would utilize a small, dedicated specialty sales force targeting dermatologists for the rare GPP condition. He clarified that no Bysanti DTC campaign is currently planned, but existing Vanda brand awareness, Fanapt, and Ponvory DTC campaigns will continue. For Nereus, commercial availability is expected late Q2 or early Q3 2026. Regarding tradipitant in gastroparesis, Vanda is preparing for an FDA hearing. The GLP-1 phase III study is initiating in H1 2026, with results anticipated by late Q3/Q4 2026. For iloperidone LAI, enrollment is ongoing but slower than desired due to European study delays, making year-end completion uncertain despite picking up in the U.S.

Raghuram Selvaraju of H.C. Wainwright & Co., LLC inquired about the commercial launch strategy for Bisanti following its potential approval, the nature of a revenue dispute concerning Ponvory, expectations for Ponvory's revenue growth in multiple sclerosis, and the regulatory outlook for Tradipitant.

Answer

President, CEO & Chairman Mihael Polymeropoulos explained that the Bisanti launch timing is a strategic decision and that current investments in the Fanapt sales force are directly transferable. CFO Kevin Moran clarified the Ponvory issue involves a disputed $3 million gross-to-net item from Q4 2024. Moran anticipates steady, not rapid, growth for Ponvory, while Polymeropoulos added that the sales force is being reset to increase prescriber frequency. Regarding Tradipitant, Polymeropoulos detailed the ongoing review for motion sickness and the pending hearing request for gastroparesis, noting the FDA has granted expanded access to over 100 patients.

An analyst from H.C. Wainwright & Co. asked about the total market opportunity for Bysanti in Major Depressive Disorder (MDD), its competitive positioning against CAPLYTA, and the clinical development timeline for its long-acting injectable (LAI) formulation.

Answer

President and CEO Dr. Mihael Polymeropoulos stated that Bysanti will compete directly with CAPLYTA in treatment-resistant depression, highlighting a similar study design and a convenient once-daily dosing. He suggested Bysanti may offer tolerability advantages regarding akathisia and peripheral neuropathy. He clarified that the Fanapt LAI is entering Phase III first, while the Bysanti LAI remains in the formulation phase, though its chemical structure is amenable to creating long-duration lipid esters.

Raghuram Selvaraju from H.C. Wainwright & Co. asked for the development timeline for the iloperidone long-acting injectable (LAI), whether 2025 revenue guidance excludes new products, the outlook for HETLIOZ generic competition, and any significant upcoming reimbursement changes.

Answer

CEO Mihael Polymeropoulos stated the Fanapt LAI program is ready to start but is pending FDA policy guidance after an IRB objection to a placebo-controlled design, with results not expected until 2027. He confirmed the 2025 revenue guidance only includes currently marketed products and that no new HETLIOZ generic competitors are expected. Executive Kevin Moran added that the primary reimbursement change, the Medicare benefit redesign, has already been factored into the 2025 financial projections.

Raghuram Selvaraju requested a payer mix breakdown (Medicare vs. commercial) for Vanda's three products to assess the impact of the Medicare benefit redesign. He also asked for the timeline of the Fanapt LAI Phase III study, future applications for milsaperidone, and the development timeline for PONVORY in new indications like ulcerative colitis.

Answer

Executive Kevin Moran explained that PONVORY's payer mix leans heavily commercial, while Fanapt and HETLIOZ have a significant portion, likely over half, from governmental payers. CEO Mihael Polymeropoulos stated the Fanapt LAI pivotal study will begin in Q4 2024 and could take over two years. He noted they are initiating a study for milsaperidone in major depression. For PONVORY, an IND for ulcerative colitis was recently submitted, with work potentially starting next year, and an IND for psoriasis, backed by strong existing data, will be submitted shortly.

Raghuram Selvaraju inquired about the German observational study findings on setmelanotide's impact on steatotic liver disease and kidney function in BBS, and how these findings might influence other indications and prescribing decisions.

Answer

David Meeker, Chairman, CEO and President, highlighted the remarkable improvement in liver function in BBS patients, noting it didn't tightly correlate with BMI change. He suggested this raises the possibility of broader MC4R mechanism effects beyond hyperphagia and weight loss, indicating there's more to learn about the drug's full impact.

Raghuram Selvaraju inquired about the German observational study findings on setmelanotide's impact on steatotic liver disease and kidney function in BBS patients, and how these results might influence other indications and prescribing decisions.

Answer

David Meeker, Chairman, CEO and President, Rhythm Pharmaceuticals, highlighted the remarkable improvement in liver function in BBS patients, noting that it didn't tightly correlate with BMI change. He suggested this raises the possibility of other melanocortin biology aspects mediating these changes, indicating there's more to learn about the mechanism beyond hyperphagia reduction and weight decrease. He emphasized that these were significant results worth highlighting.

Raghuram Selvaraju of H.C. Wainwright & Co., LLC asked about CMC progress, specifically the status of developing a smaller pill for Bivamelagon and the objectives for the RM-718 auto-injector, including if a less frequent dosing schedule was being explored.

Answer

David Meeker, Chairman, President & CEO, clarified that the main CMC challenge for Bivamelagon was solved by achieving a 90% drug load, allowing 600mg in a single pill, making smaller pills for lower doses technically straightforward. He stated the auto-injector development for RM-718 is entirely focused on a weekly formulation, with no current plans to explore less frequent dosing.

Raghuram Selvaraju of H.C. Wainwright inquired about the qualitative differences planned for the hypothalamic obesity (HO) commercial rollout compared to other indications and asked about the company's forward strategy for China after the RareStone termination.

Answer

Jennifer Chien, Head of North America, highlighted that the HO launch will be more targeted as a vast majority of patients are already under the care of endocrinologists. David Meeker, CEO, added that Rhythm is pleased to have the rights to China back. He noted that while the genetic opportunity is smaller there, the HO opportunity is significant. The company is still developing its China strategy, which will likely involve a partner.

Raghuram Selvaraju asked about the market impact of the IMCIVREE pediatric label expansion in the U.S. versus ex-U.S. and whether the Trispera partnership in Turkey is a model for future international expansion.

Answer

EVP Yann Mazabraud explained that while pediatric use in Europe has a significant clinical impact, its revenue impact is not major. He and CEO David Meeker clarified that the partnership strategy is evaluated country-by-country, using partners like Trispera where deep local expertise is advantageous over a direct model.

Raghuram Selvaraju asked for an update on planned activities for the congenital hyperinsulinism (CHI) program in 2025 and inquired about which additional countries might institute reimbursed early access programs for setmelanotide.

Answer

Chairman, CEO and President David Meeker committed to providing an update on the CHI program in 2025, stating that good progress has been made in identifying a lead molecule. EVP, Head of International Yann Mazabraud noted that the company is selective and plans to launch in only 2 or 3 additional countries in the next 12 months by design, to ensure focus and sustainability.

Raghuram Selvaraju inquired about the impact of international anti-dumping practices on Balchem's sales, particularly in the Human Nutrition and Health segment outside the U.S. He specifically asked for an update on the status of European anti-dumping campaigns related to choline and their expected impact. He also asked for an update on the Orange County microencapsulation manufacturing facility's construction timeline and its anticipated impact on revenues and earnings quality. Finally, he asked CFO Martin Bengtsson about the expected pace of debt repayment and the appropriate effective tax rate assumption for the remainder of 2025.

Answer

Chairman, President, and CEO Ted Harris confirmed that the EU's preliminary anti-dumping duties on China-origin choline chloride (for human and animal use) are a recognition of unfair trade practices. He noted that initial duties were slightly reduced, with a final vote expected by year-end 2025 to enact them for five years. He also mentioned efforts to prevent product rerouting and an improved environment for bringing such cases to governments. Regarding the Orange County facility, Mr. Harris stated construction is expected to be completed in early 2027, with new product production by mid-2027. The new plant will double capacity and incorporate more efficient technology, supporting continued double-digit growth for the high-margin encapsulates business, which grew 26% in Q3. CFO Martin Bengtsson indicated the effective tax rate for 2025 is estimated around 22.5%. He explained that the pace of debt repayment, while strong due to free cash flow, would depend on the timing of strategic M&A opportunities and share repurchases for anti-dilutive purposes, with M&A being the primary determinant.

Raghuram Selvaraju from H.C. Wainwright & Co., LLC asked for clarification on the new facility's timeline and funding, the market progress of VitaCholine ProFlo, and Balchem's broader strategic outlook on human health, including its clinical studies. He also inquired about the debt reduction strategy and the effective tax rate outlook.

Answer

Chairman, President & CEO Ted Harris stated the new facility will be funded within the existing CapEx budget over three years and come online in late 2027/early 2028. He noted that while VitaCholine ProFlo is being introduced, a more significant opportunity is the inclusion of choline in nutritional beverages. He clarified that the focus on clinical studies is a continuation of their science-first strategy, not a pivot towards pharmaceuticals. EVP & CFO Martin Bengtsson confirmed the capital allocation strategy is unchanged, with a focus on paying down debt, and projected the full-year effective tax rate would be near the low end of the 22-23% range.

Raghuram Selvaraju of H.C. Wainwright asked about the most significant business tailwinds for 2025, focusing on new products in the Human Nutrition & Health segment like VitaCholine and K2 DELTA. He also inquired about the potential impact of currency fluctuations, the company's debt repayment strategy, and the expected effective tax rate for the year.

Answer

Chairman, President and CEO Ted Harris confirmed that choline and vitamin K2 are significant tailwinds, with both categories showing strong double-digit growth, and highlighted strength in the food business. CFO Martin Bengtsson addressed the financial questions, stating that a weaker dollar could provide a $10-15 million annualized revenue tailwind, while transactional FX impact is immaterial. He noted that with leverage low, the company is evaluating M&A and may consider share buybacks. Bengtsson projected a full-year effective tax rate between 22% and 22.5%.

Raghuram Selvaraju of H.C. Wainwright & Co. asked about marketing plans for VitaCholine Pro-Flo, the potential impact of tariffs from Mexico or China, the nature of the quarter's foreign tax impact, and the company's exposure to the avian flu crisis.

Answer

Theodore Harris, Chairman, President & CEO, stated that marketing for VitaCholine Pro-Flo will leverage existing partnerships and social media to target the adult multivitamin market. He noted Balchem is well-positioned regarding tariffs, with minimal sourcing from China and Mexico. Carl Bengtsson, CFO, clarified the Q4 foreign tax impact was a one-off event and explained that the impact of the bird flu on demand is currently not significant as it is offset by other market dynamics.

Raghuram Selvaraju inquired about the market performance of Optifolin+, the expected gross margin impact of new HNH products, and how VitaCholine Pro-Flo might expand the VitaCholine franchise. He also asked about capital allocation for HNH marketing and the company's strategic interest in the anti-aging and weight loss markets.

Answer

Chairman, President and CEO Ted Harris reported that Optifolin+ is performing ahead of expectations with an annualized sales rate of about $2 million. He explained that new products like VitaCholine Pro-Flo are accretive to gross margin and that Pro-Flo specifically opens up the significant multivitamin market. Harris confirmed plans to increase capital allocation to HNH marketing and R&D, focusing on social media. He noted anti-aging is a core focus, while Balchem's role in weight loss is more ancillary. CFO Carl Bengtsson added that the long-term tax rate is expected to remain in the 22-23% range and that the company's debt strategy remains flexible.

Raghuram Selvaraju inquired about the typical profile of a repeat ZUNVAIL prescriber, the outlook for payer contracts by year-end, the evolving prior authorization landscape, and the company's capital allocation priorities for future R&D activities.

Answer

COO Lauren D'Angelo explained that repeat prescribers are typically from targeted high-volume nursing homes and that she expects at least one more large national plan to be contracted by year-end. She noted that while prior authorizations have increased with demand, 90% of orders are filled, albeit with delays. CEO Michael McFadden added that funding for R&D is already built into the current expense guidance and provided a Q3 net revenue run rate estimate of $5.75 million to $6.25 million.

Asked about the profile of repeat prescribers, the outlook for payer contracts, the evolving prior authorization situation, capital allocation for future R&D, and the current Q3 revenue run rate.

Answer

The company targets high-volume facilities for repeat prescribers and expects another large national payer contract by year-end. Prior authorizations have increased but are manageable, causing delays but not denials. Funding for R&D is included in current expense guidance, and the Q3 net revenue run rate is projected to be between $5.75M and $6.25M.

Raghuram Selvaraju inquired about early commercial metrics for ZUNVEYL, including the number of repeat prescribers, average patient time on therapy, and current prior authorization requirements. He also asked about the expected evolution of payer contracting, the market potential beyond Medicare, the likelihood of galantamine-specific prior authorizations, and future promotional strategies such as sampling and advertising.

Answer

CEO Michael McFadden and COO Lauren D'Angelo addressed the questions. McFadden stated the longest time on therapy is since the April 1st launch, with expectations for long-term use given the drug's profile, and noted a lack of reported GI or insomnia adverse events. D'Angelo reported that over half of the initial 100 ordering accounts have already reordered. She described prior authorizations as simple, mostly checkbox-based, and noted that while contracting discussions are progressing, nearly all prescriptions are being filled without contracts. D'Angelo also clarified that the immediate focus is on the Medicare Part D market, which constitutes about 80% of long-term care, and that sampling and broader advertising are planned for a future expansion into neurology.

Raghuram Selvaraju of H.C. Wainwright & Co., LLC inquired about Rockwell Medical's expansion plans in the Western U.S., the status of negotiations for a new long-term agreement with its former largest customer, and the company's capital allocation priorities.

Answer

President and CEO Dr. Mark Strobeck confirmed the Western U.S. remains a key ~$100 million opportunity, with more clarity expected later in the year pending discussions with major providers. He stated that negotiations with their former largest customer, DaVita, are for a long-term supply agreement, not short-term gap-filling, which could positively impact future financials. Regarding capital, Dr. Strobeck prioritized investment in manufacturing automation to reduce costs, stating the company has sufficient resources to manage its debt obligations while pursuing new customer acquisition.

Raghuram Selvaraju from H.C. Wainwright & Co. inquired about the potential outcomes of negotiations with Rockwell's largest historical customer and the expected revenue cadence for the remainder of 2025, including the anticipated low point for the year.

Answer

President and CEO Dr. Mark Strobeck explained that discussions with their largest customer are aimed at a smaller-scale, long-term supply arrangement. He expressed confidence in the full-year guidance but noted new customer acquisitions make specific quarterly predictions difficult. CFO Jesse Neri added that Q2 is expected to be the revenue low point for the year, with growth anticipated thereafter.

Inquired about the reasons for the largest customer's departure, the risk of other customers following suit, and the growth strategy for the new bicarbonate cartridge business.

Answer

The executives explained the largest customer's decision was for supplier diversification and was made years ago, not reflecting current risks to other customers who are on long-term contracts. The bicarbonate cartridge will initially be marketed to the existing customer base to fill a portfolio gap, with long-term growth tied to market adoption of compatible machines.

Raghuram Selvaraju of H.C. Wainwright & Co., LLC asked about the mechanics of payment for ZivaSkin, specifically if it's staged or upfront, and whether payers are requiring prior failure of other RDEB treatments before approving ZivaSkin.

Answer

CCO Madhav Vasanthavada explained that revenue is recognized in full only after the patient is treated. He noted that hospitals secure financial agreements with payers before ordering the product, minimizing risk. He also confirmed that payers have not required patients to fail other therapies, attributing this to the strong clinical value proposition of ZivaSkin, which is resonating well with them.

Raghuram Selvaraju of H.C. Wainwright & Co. asked about the plan for scaling commercial manufacturing, the net proceeds from the PRV sale, the expected pro forma cash balance, and the prior treatment history of potential ZEVASKYN patients.

Answer

Chief Technical Officer Dr. Brian Kevany detailed the manufacturing ramp-up from 4 patients/month at launch to 10 patients/month by early next year using the existing facility. CEO Dr. Vish Seshadri added that a further expansion for post-2027 capacity is being designed. CFO Joe Vazzano stated the net proceeds from the PRV sale will be approximately $152 million. Chief Commercial Officer Dr. Madhav Vasanthavada noted that the majority of potential patients will have been exposed to prior treatments.

Raghuram Selvaraju of H.C. Wainwright asked for clarification on the FDA's statutory timeline for responding to the BLA resubmission and setting a PDUFA date. He also posed a strategic question about how the company might deploy proceeds from a potential Priority Review Voucher (PRV) sale to broaden its pipeline.

Answer

CEO Vishwas Seshadri explained that the FDA typically indicates acceptance of a resubmission within two weeks and would likely set a six-month PDUFA date for what they anticipate will be a Type 2 resubmission. Regarding strategy, Seshadri outlined several potential growth avenues, including pz-cel life cycle management, leveraging their cell therapy platform in other diseases, and advancing their AAV ophthalmology assets, while stressing the immediate priority is the pz-cel launch.

Raghuram Selvaraju inquired about the market similarities between Hypoplastic Left Heart Syndrome (HLHS) and pediatric dilated cardiomyopathy (PDC), the regulatory implications of a potential HLHS approval on the PDC program, and the comparative cost of the proposed PDC Phase 2 trial.

Answer

CEO Wa'el Hashad explained that while the markets are similar in being rare orphan diseases, the prevalence and administration methods differ, with PDC potentially involving chronic use. He and Chief Medical Officer Nataliya Agafonova noted that a positive HLHS outcome would support the PDC review, but the FDA would still evaluate the PDC trial on its own merits, which is already agreed upon as a single pivotal study. Mr. Hashad provided a preliminary total trial cost estimate for the PDC study in the range of $15 to $20 million.

Asked about the pricing strategy for laromestrocel in HLHS, specifically the applicability of value-based pricing and relevant benchmarks, and also inquired about the timing requirements for securing a Priority Review Voucher (PRV).

Answer

The company believes a strong value-based pricing case can be made, citing its one-time use, life-saving potential, and ability to reduce healthcare costs. They referenced Mesoblast's GvHD therapy ($1.5M) as a benchmark. Regarding the PRV, while the current program expires before their expected approval, they are confident it will be reauthorized by Congress, making them eligible.

Raghuram Selvaraju of H.C. Wainwright & Co. inquired about the ELPIS II trial's enrollment timeline and data release, long-term data from ELPIS I, the regulatory path and funding strategy for the Alzheimer's program, and the growth timeline and strategic nature of the contract manufacturing business, including the relationship with Secretome Therapeutics.

Answer

CEO Wael Hashad and Dr. Nataliya Agafonova explained that the ELPIS II trial is 70% enrolled, targeting completion by year-end 2024, with a potential BLA filing in Q1 2026. They noted that 5-year survival data from ELPIS I is expected by the end of August to support the BLA. For the Alzheimer's program, a Type B meeting with the FDA is planned before year-end to discuss a streamlined path, and the company is pursuing non-dilutive funding. The contract manufacturing business is projected to reach its $4-5 million revenue goal within 1-2 years.

An analyst on behalf of Raghuram Selvaraju of H.C. Wainwright & Co., LLC asked about the company's plans for future strategic financing arrangements, including the number of deals and capital outlay, and inquired about the performance of the recent Stella MSO investment.

Answer

CEO Hadar Levy outlined a strategic goal to sign at least five new investment contracts by the end of 2025 and ten more in 2026, with individual investments ranging from $2 million to $5 million. He highlighted the Stella MSO partnership as a great success, noting that BrainsWay's support has led to a more than 50% increase in TMS patient utilization at Stella's clinics.

On behalf of Raghuram Selvaraju, Dan asked about the company's plans for future strategic financing arrangements, including the target number of deals and the expected capital outlay per investment. He also inquired about the early results of the Stella MSO partnership.

Answer

CEO Hadar Levy detailed a strategic goal to sign at least five new financing agreements by the end of 2025 and ten more in 2026, with investment sizes ranging from $2 million to $5 million each. He described the Stella MSO collaboration as a 'great example,' noting it has led to a more than 50% increase in TMS utilization at Stella's clinics.

Representing Raghuram Selvaraju of H.C. Wainwright & Co., an analyst asked about the company's plans for future strategic financing arrangements, including the number of deals and capital outlay, and inquired about the early results of the Stella MSO partnership.

Answer

CEO Hadar Levy outlined a strategic goal to sign at least five new financing agreements by the end of 2025 and ten more in 2026, with investment sizes ranging from $2 million to $5 million per deal. He highlighted the success of the Stella MSO collaboration, noting it has led to a more than 50% increase in TMS patient utilization at Stella's clinics, a model Brainsway intends to replicate with future partners.

Speaking on behalf of Raghuram Selvaraju from H.C. Wainwright & Co., LLC, an analyst asked about the company's plans for future strategic financing arrangements, including the target number of deals, expected capital outlay per investment, and the performance of the recent Stella MSO financing.

Answer

CEO Hadar Levy outlined a strategic goal to sign at least five similar contracts by the end of 2025 and ten more in 2026, with investment sizes ranging from $2 million to $5 million each. He described the Stella MSO collaboration as a great success, noting that BrainsWay's support has led to a more than 50% increase in TMS patient utilization at Stella's clinics, a model they intend to replicate with future partners.

Raghuram Selvaraju from H.C. Wainwright & Co., LLC asked for clarification on remaining litigation, the primary drivers for ROLVOIDON's demand, the intellectual property status of OTREXUP, and the expected SG&A savings from ceasing its commercialization.

Answer

CEO Brendan O’Grady explained that remaining legal matters are primarily shareholder lawsuits that are being worked through. He identified an expanding customer base as the key driver for ROLVOIDON demand. For OTREXUP, he clarified that while the methotrexate molecule has no IP, the delivery pen device does, and the decision to halt commercialization was strategic to focus on more profitable growth assets. CFO Ajay Patel added that this move is expected to save $2-3 million in SG&A annually starting next year and was a factor in raising the lower end of the 2025 adjusted EBITDA guidance.

Raghuram Selvaraju of H.C. Wainwright & Co. asked about additional marketing strategies for Rolvedon, the expected impact of further genericization on Indocin, and for an update on the company's strategic M&A plans.

Answer

Executive Brendan O’Grady outlined a multi-pronged strategy for Rolvedon growth, including gaining more share in Medicare Part B clinics and expanding commercial payer coverage to penetrate new channels. For Indocin, he noted the plan assumes two more generic entrants this year, which would impact volume and price. On the M&A front, O'Grady expressed optimism about closing a strategic transaction in 2025 to position the company for its growth phase, confirming that current 2025 guidance does not depend on any such deal.

An analyst on behalf of Raghuram Selvaraju asked about the competitive landscape for Indocin, including the current and expected number of generic competitors. He also inquired about recent uptake trends for Sympazan following its initial commercial pilot study.

Answer

CEO Brendan O’Grady detailed that Indocin currently faces two generic competitors and a compounder, with an expectation of one or two more generics emerging during 2025. Regarding Sympazan, he confirmed the pilot showed it was promotionally sensitive and that adding a small field sales team resulted in one of the highest prescription months for the product in January, validating the impact of in-person promotion.

Raghuram Selvaraju from H.C. Wainwright & Co. asked for more detail on the market dynamics driving new customer adoption of ROLVEDON and inquired about future trends for gross margin, specifically its sustainability in the low-to-mid 70% range as the product mix evolves.

Answer

Executive Brendan O’Grady explained that ROLVEDON adoption is driven by its safety and efficacy profile, which opens doors, but is ultimately a contract-driven play where practice managers make decisions based on access and ASP management. CFO Ajay Patel added that contracting helps with customer stickiness. Regarding margins, Patel reiterated that gross margin erosion is expected to continue with the shift from INDOCIN to ROLVEDON, but the company is focused on maintaining its 2024 blended gross margin guidance of 70% to 75%.

Raghuram Selvaraju from H.C. Wainwright & Co., LLC asked for quantification of the expanded prescriber base post-J code, any beneficial spillover effect on Jelmyto, and potential advantages of the UGN-103 formulation.

Answer

CCO David Lin explained that post-J code in 2026, the company will expand its focus from 2,000 early adopters to the full universe of 8,500 healthcare providers. He noted it's too early to see a spillover effect on Jelmyto but expects the increased market presence to support its growth. CMO Mark Schoenberg clarified that UGN-103's new formulation addresses solubility and is not expected to change the clinical, safety, or tolerability profile.

Raghuram Selvaraju questioned if the lack of precedent for approvals in this indication would be a key topic at the ODAC meeting, whether the JELMYTO approval would serve as a helpful precedent, and asked for an update on the UGN-103 program.

Answer

Chief Medical Officer Dr. Mark Schoenberg acknowledged the FDA likely wants a public discussion on the novel approach and confirmed they will use JELMYTO's history to familiarize the committee with the treatment concept. CEO Liz Barrett added this is crucial as the panel is mostly medical oncologists. Regarding UGN-103, she stated enrollment is nearly complete and will finish by the end of summer, with data expected in 2026.

Raghuram Selvaraju from H.C. Wainwright asked for more detail on the UGN-102 reimbursement environment, specifically the difference between the initial miscellaneous J-code and the permanent J-code, and also requested an update on enrollment for the UGN-103 UTOPIA trial.

Answer

Chief Commercial Officer David Lin explained that the miscellaneous J-code period involves a longer reimbursement timeline (50-60 days), which is expected to be cut in half upon receiving a permanent J-code in January 2026. President and CEO Liz Barrett added that initial uptake will likely be higher in hospitals. Chief Medical Officer Mark Schoenberg confirmed that enrollment for the UGN-103 UTOPIA trial is expected to complete in 2025, with data anticipated in 2026.

Raghuram Selvaraju asked about JELMYTO's gross-to-net headwinds and why they aren't expected for UGN-102, the impact of standard review on UGN-102's prelaunch timeline, patent timing, and the likely composition of the FDA advisory committee.

Answer

CFO Chris Degnan addressed the gross-to-net question, stating UGN-102 is not expected to be subject to the Medicare wastes provision and will have less 340B exposure. CCO David Lin noted the prelaunch timeline is not significantly altered. CMO Dr. Mark Schoenberg anticipated a typical AdCom panel of urologists, oncologists, and biostatisticians. CEO Liz Barrett did not have an update on the patent timing.

Raghuram Selvaraju of H.C. Wainwright & Co., LLC inquired about the planned promotional strategy for LUMRYZ in idiopathic hypersomnia (IH) versus narcolepsy. He also asked if upcoming orexin data at the WorldSleep conference could change the competitive outlook.

Answer

CEO Gregory Divis noted the different patient profiles but highlighted the significant overlap in prescribers. COO Susan Rodriguez added that the value proposition of LUMRYZ's extended-release profile, which drives narcolepsy adoption, is also highly valuable for IH patients. Regarding orexins, Divis stated they are awaiting more data but reiterated that physician feedback consistently points to a continued, important role for oxybates.

Speaking on behalf of Raghuram Selvaraju from H.C. Wainwright & Co., LLC, Eduardo asked about the impact of new synthetic opioids on NARCAN demand, the competitive landscape for smallpox antivirals like Tembexa, and whether to expect additional MCM contract modifications for specific programs in the second half of 2025.

Answer

President and CEO Joseph Papa explained that the rise of stronger opioids reinforces the need for wider NARCAN access. Regarding smallpox antivirals, he highlighted the ongoing clinical trials for Tembexa in Africa to gather data on its potency and confirmed continued discussions with the U.S. government. Papa also affirmed that the company expects more revenue-generating contract modifications in H2 2025. CFO Richard Lindahl added that these anticipated contracts are already incorporated into the company's financial guidance.

Raghuram Selvaraju asked about the expected trends for Cimbravo's gross-to-net and coverage for the remainder of the year, and what physicians and patients see as its main differentiators.

Answer

Chief Financial Officer Nick Pizzie projected that Cimbravo's gross-to-net will remain high, in the low 80% range, for the rest of the year. Chief Commercial Officer Ari Maizel identified the multi-mechanistic approach as a key differentiator, with early feedback highlighting its rapid onset and durability of response.

Raghuram Selvaraju from H.C. Wainwright & Co. inquired about the expected timeline for achieving broad payer coverage for SYMBRAVO after its launch. He also asked which of the additional indications for solriamfetol, such as binge-eating disorder, appears most attractive from a competitive positioning standpoint.

Answer

Ari Maizel, Chief Commercial Officer, stated it was premature to provide a specific coverage cadence for SYMBRAVO but affirmed the team is focused on securing access quickly. Herriot Tabuteau, CEO, addressed solriamfetol, noting the encouraging ADHD data and highlighting that for binge-eating disorder, being potentially the second product approved would create a very favorable competitive dynamic, pending Phase III results.

Raghuram Selvaraju inquired about the planned cadence and strategy for direct-to-consumer (DTC) promotional activities for both Auvelity and the newly approved Symbravo.

Answer

Chief Commercial Officer Ari Maizel confirmed that a broad-based media outreach for Auvelity is planned for the coming months, with spending timed to align with periods of high patient flow to HCP offices. He noted that specific plans for Symbravo's go-to-market strategy, including any DTC components, would be shared in the future.

Raghuram Selvaraju of H.C. Wainwright asked about Aquestive's direct-to-consumer (DTC) advertising plans for Anaphylm, its distribution infrastructure, and its strategy for optimizing the product's value outside the United States.

Answer

CEO Daniel Barber and executive Sherry Korczynski described a phased launch strategy, prioritizing HCP awareness first, then layering in DTC (social media, digital) as payer access grows. For distribution, Korczynski stated it will be predominantly retail (CVS, Walgreens) but they are exploring alternate channels. Barber identified Canada, the U.K., and Germany as key ex-U.S. markets and confirmed engagement with their respective regulatory bodies is planned to support partnerships.

Raghuram Selvaraju asked about the commercial experience of the competitor nasal spray, the specifics of the Libervant court ruling and appeal process, and the drivers behind the wide 2025 revenue guidance.

Answer

CEO Daniel Barber commented that competitor awareness spending is a 'rising tide' that benefits the entire space. Regarding Libervant, he explained that court outcomes are uncertain but they are committed to fighting for patient access until VALTOCO's exclusivity expires in early 2027. CFO Ernie Toth attributed the wide revenue guidance to uncertainties surrounding Libervant revenue due to the court case and potential Suboxone erosion.

Raghuram Selvaraju of H.C. Wainwright & Co. asked about the pricing paradigm for Libervant relative to VALTOCO, the specific timing for the Libervant filing ahead of orphan drug exclusivity expiration, the company's market research on neffy prescribers for Anaphylm, and whether the AQST-108 timeline remains on track for a launch by 2028.

Answer

CEO Daniel Barber confirmed that the AQST-108 program is on schedule, with the briefing book submitted to the FDA. Regarding Libervant, he stated that price has not been an obstacle and the filing will be timed to launch immediately upon ODE expiration. SVP, Chief Commercial Officer Sherry Korczynski noted that market research indicates Anaphylm's ease of use and carrying will appeal to new patients, those with existing prescriptions, and those who currently avoid treatment.

Asked about potential new ex-U.S. territories, the cadence of European country launches for the rest of 2025, and the company's updated thinking on pursuing strategic M&A opportunities in oncology supportive care.

Answer

The European partner, Norgine, is focused on the 'big 5' countries, with Spain, Italy, and France expected to be next after the U.K. and Germany. Regarding strategic opportunities, management's focus was first on internal execution, but they are now open to exploring synergistic assets in supportive care and have received inbound interest. The potential partnership in Japan is also a future opportunity.

Raghuram Selvaraju inquired about Norgine's progress in Europe, including the development of relationships with key centers, the expected rollout cadence, and which countries are likely to be the primary revenue contributors. He also asked if the AYA population would be a comparable growth driver in Europe and about Fennec's U.S. business development initiatives.

Answer

CEO Jeffery Hackman reported that the commercial launch with Norgine is underway in Germany and the U.K., which are expected to be two of the largest European markets, and that progress is good. He noted the European approval is for patients up to 17 years old, and Norgine will evaluate the AYA opportunity separately. Regarding U.S. business development, Hackman confirmed ongoing discussions with key academic institutions about expanding PEDMARK's use and stated the company will provide updates as they materialize.

The analyst asked about the shift from compounded sodium thiosulfate to PEDMARK, the company's future business development priorities, and the status of ongoing IP litigation.

Answer

Executives confirmed that some institutions are shifting from compounded products to PEDMARK, sometimes due to side effects. Key business development priorities include the European launch with Norgine and evaluating options for Japan following trial results. The IP litigation is ongoing, and the company noted its patent portfolio has been significantly strengthened.

Raghuram Selvaraju asked about shifts in purchasing behavior away from compounded sodium thiosulfate, the company's future business development priorities, and the current status of ongoing IP litigation.

Answer

CEO Jeff Hackman stated that several institutions began moving from compounded products to PEDMARK in the quarter, sometimes prompted by side effects from compounding. Regarding business development, he highlighted Japan as a key area of evaluation pending trial results in 2025. Executive Robert Andrade added that the European launch with Norgine is expected to contribute revenue in 2025. On IP litigation, he confirmed the case is ongoing and noted Fennec's portfolio has strengthened to six Orange Book listed patents.

Raghuram Selvaraju asked for physician feedback on the competitive landscape versus biologics, particularly regarding administration frequency, and inquired about the company's strategy for in-licensing or acquiring new products.

Answer

CEO David Zaccardelli reiterated that biologics are seen as complementary, not competitive, due to different mechanisms and use cases, with Ohtuvayre's bronchodilation being a key differentiator. Chief Commercial Officer Christopher Martin added that physicians view Ohtuvayre as a broad-use foundational therapy. Regarding M&A, Zaccardelli confirmed the company aims to leverage its capabilities by acquiring mid- to late-stage assets, primarily in the respiratory space, to continue its growth.

Raghuram Selvaraju of H.C. Wainwright & Co. asked about the competitive threat from upcoming biologics, physician views on administration frequency, and the company's broader strategy for in-licensing or acquiring new assets.

Answer

CEO David Zaccardelli and CCO Christopher Martin asserted that physicians view Ohtuvayre as a broad, foundational COPD therapy due to its unique bronchodilator and anti-inflammatory profile, making biologics complementary rather than competitive. On strategy, Zaccardelli stated that the company will actively look for mid-to-late-stage respiratory assets to leverage its commercial and development capabilities as the Ohtuvayre launch matures.

Raghuram Selvaraju inquired about emerging prescriber preferences, particularly regarding Ohtuvayre's use with triple therapy, and asked about lucrative ex-U.S. territories beyond Europe and China, as well as pricing dynamics in Macau.

Answer

Executive David Zaccardelli stated that while regions like Japan and South America are important incremental opportunities, the primary focus remains the U.S. launch and securing partners for ex-U.S. territories. Chief Commercial Officer Chris Martin addressed prescribing trends, noting the key driver is persistent symptoms. He believes that over time, Ohtuvayre's use will grow in patients on therapies other than triple therapy, as physicians seek effective non-steroidal options, which could reduce reliance on ICS.

Raghuram Selvaraju asked about emerging prescriber trends for Ohtuvayre combinations, how the commercial launch experience might influence the strategy for new indications like bronchiectasis, and for an accounting clarification on pre-launch versus post-launch expenses.

Answer

Executive David Zaccardelli stated that prescribing is broad across all patient types with no specific combination preference, though use on top of triple therapy is high due to unmet need. Chief Financial Officer Mark Hahn clarified that pre-approval manufacturing costs were expensed as R&D, while all ongoing commercial activities, like speaker programs, are booked as SG&A.

Raghuram Selvaraju asked for details on the company's commercial marketing strategy, including social media, direct-to-consumer (DTC) advertising plans, physician speaker programs, and patient advocacy involvement. He also requested a timeline for the non-CF bronchiectasis trial.

Answer

Chief Commercial Officer Chris Martin detailed a multi-faceted marketing plan, highlighting digital outreach to over 50,000 physicians, leading to high engagement from nearly 7,000. He confirmed that speaker programs are being scheduled and that patient-focused marketing is active through a dedicated website and future point-of-care initiatives. An unnamed executive projected the non-CF bronchiectasis trial could take around two years to fully enroll.

Asked about the ideal US commercial partner for D-PLEX100, the division of responsibilities in a partnership, control over manufacturing, other potential applications for the PLEX platform, and specifics of the ImmunoGenesis collaboration.

Answer

The ideal partner is a large pharma or med device company with a strong presence in the surgical suite. PolyPid intends to control manufacturing as a core strength. In a partnership, PolyPid would handle global strategy and medical affairs, while the partner would manage daily sales activities. The PLEX platform has been tested with various compounds like chemotherapy and peptides. The ImmunoGenesis collaboration aims to use PLEX to provide prolonged, local delivery of a STING agonist to enhance its efficacy, with potential for future combination therapies.

An analyst from H.C. Wainwright & Co., speaking on behalf of Raghuram Selvaraju, inquired about market factors affecting Elfabrio uptake in the U.S., the competitive landscape for refractory gout, the design of future pivotal trials for PRX-115, and whether Protalix intends to operate as a contract manufacturer.

Answer

Executive Dror Bashan positioned Elfabrio as a strong alternative for adult Fabry patients, highlighting its safety profile and Chiesi's ongoing investment. For PRX-115, he noted active discussions with U.S. and European regulators about the Phase II plan. Bashan firmly stated that Protalix will not pursue a contract manufacturing (CMO) business model, instead focusing on its core strategy as a boutique development company for rare diseases to maximize shareholder value.

Speaking on behalf of Raghuram Selvaraju, an analyst from H.C. Wainwright asked about the competitive positioning of PRX-115 against existing treatments, the status of early-stage R&D programs including the oral anti-TNF candidate, and the company's strategy for its convertible notes and future capital structure.

Answer

Executive Dror Bashan stated that PRX-115 could be a very interesting alternative to existing gout enzymes, noting the company is aiming for a superior dosing profile. He confirmed the oral anti-TNF program has been discontinued and that Protalix is evaluating over half a dozen new early-stage programs, with updates expected in the next one to two quarters. Regarding capital structure, Mr. Bashan affirmed the plan to repay the convertible notes in September with cash on hand, without refinancing, citing sufficient resources and growing revenues that have moved the company to a more stable financial position.

Raghuram Selvaraju inquired about the specific patient subpopulation for PRX-115, its competitive positioning against KRYSTEXXA, and its potential advantages regarding efficacy, safety, and convenience. He also asked about the expected efficacy endpoints for a Phase II trial and whether PRX-115 would be a direct competitor or used in refractory patients.

Answer

Dror Bashan, an executive, explained that PRX-115 is being developed for uncontrolled gout patients and that the company is optimistic following initial results, leading to preparations for a Phase II study. He stated the goal is to offer improved safety and better dosing frequency compared to existing treatments like KRYSTEXXA. Bashan confirmed the intention is for PRX-115 to compete directly in the uncontrolled gout market, and that the specific design of the Phase II trial and its endpoints would be finalized after discussions with regulators.

Raghuram Selvaraju of H.C. Wainwright & Co. inquired about any remaining crop trait assets that could be monetized, the potential timing for new Zola flavor introductions, and the approximate market share Zola would need to achieve sustainable profitability.

Answer

President and CEO T.J. Schaefer confirmed that Arcadia still possesses a few monetizable traits and is actively exploring options like licensing agreements or asset sales. He noted that the new Zola flavors just began shipping in Q2 2024 and that the company is exploring an innovation pipeline for 2025 and beyond. To reach breakeven, Schaefer estimated Zola would need to capture a low single-digit market share, an increase from its current position of approximately 1%.

Inquired about the potential for synergistic activity between CT1812 and GLP-1 receptor agonists in Alzheimer's treatment, and asked if the company was considering or studying doses between 100mg and 300mg.

Answer

The company is open to exploring synergy with GLP-1s if they are approved for Alzheimer's. They confirmed they are already studying an intermediate 200mg dose in two ongoing trials, believing it could be an optimal 'sweet spot' for efficacy and safety.

Raghuram Selvaraju asked about any favorable commercial tailwinds for IGALMI and their potential impact on revenue growth in the second half of 2024. He also requested an update on the potential monetization of OnkosXcel (BXCL701) and inquired about plans for the neurology pipeline beyond BXCL501.

Answer

Executive Matthew Wiley identified a key tailwind as growing interest from community behavioral health centers, which have lower administrative hurdles. Executive Vincent O'Neill confirmed that partner outreach has been initiated to monetize BXCL701. Executive Vimal Mehta added that while the focus is on late-stage trials, the company is exploring partnerships for its earlier neuroscience pipeline assets (502, 503).

Raghuram Selvaraju asked for an update on IGALMI's group contracting discussions and inquired about the strategic options for the company's earlier-stage Neuroscience assets.

Answer

Executive Matthew Wiley confirmed that the volume contracting strategy for IGALMI is working, with Q2 unit volume already surpassing the entire first quarter. Executive Vimal Mehta added that while the company's primary focus is on the two late-stage programs, they will explore potential partnerships for early-stage assets.

Raghuram Selvaraju of H.C. Wainwright asked for details on expected efficacy takeaways from the Trokie/Radicle collaboration, the INM-755 study, and the development plan for the glaucoma candidate, INM-088.

Answer

President and CEO Eric Adams described the Trokie collaboration as a consumer experience study on THCV's effects on energy and appetite, not a formal clinical trial. For INM-755, he highlighted key readouts including itch, inflammation, and pain. SVP, Preclinical Research and Development Dr. Eric Hsu added that the glaucoma program's initial Phase 1/2 trial will focus on safety as the primary readout and IOP reduction as the secondary readout.

On behalf of Raghuram Selvaraju from H.C. Wainwright, an associate asked about the potential for using higher doses of SYNB1618 in the pivotal study, the use of Ginkgo's codebase for future candidates, and whether any new partnerships were imminent.

Answer

President and CEO Aoife Brennan explained that instead of increasing the cell dose, the strategy is to use the more potent SYNB1934 strain, which has double the activity. She also noted the Phase 3 study will likely feature individualized dosing. David Hava added that they will continue to leverage Ginkgo's capabilities rather than building them internally. Aoife Brennan concluded by stating she could not comment on unannounced deals but reiterated the strategy of internal development for metabolic diseases and partnerships for other areas.

Raghuram Selvaraju inquired about the applicability of the ACDx companion diagnostic across all LADR candidates, the intellectual property status of the diagnostic, and the nature of ongoing strategic partnership discussions, specifically whether they were product-specific or platform-based.

Answer

Chief Operating Officer Eric Curtis clarified that the ACDx companion diagnostic is designed to work with all LADR candidates (LADR-7, 8, 9, 10) and future albumin-binding drugs. He confirmed that the intellectual property process for ACDx is progressing as expected without any issues. Regarding partnerships, Curtis stated that discussions with several pharmaceutical companies are underway under CDA, and the addition of the ACDx personalized medicine approach has made the pipeline more attractive, though he could not share specific details on the nature of the potential deals.