Matthew Kaplan

Matthew Kaplan of Ladenburg Thalmann inquired about the company's launch preparations for YUTREPIA, focusing on the size and structure of the commercial sales force.

Answer

Chief Commercial Officer Scott Moomaw detailed that an experienced sales team, primarily from the PH space, has been in place for approximately a year. The force is sized to cover 6,000-7,000 targets, including large PH centers and community pulmonologists treating ILD. A specialized team of key account directors will also provide deep coverage for the most significant academic medical centers.

Matthew Kaplan of Ladenburg Thalmann & Co. Inc. asked if the FDA has identified any material issues that could prevent YUTREPIA's approval and lead to a Complete Response Letter (CRL), given that legal impediments have passed.

Answer

CEO Dr. Roger Jeffs declined to comment on specific FDA discussions but framed the four months since the legal stay was lifted as a positive period of constructive dialogue. He emphasized that the company remains optimistic and is still actively seeking approval for both PAH and PH-ILD indications as submitted in the amended NDA, without backing away from that position.

Inquired about the statistical power of the CHAPERONE interim analysis, the process following the independent review, the potential timeline for an NDA filing if results are positive, and the ability to leverage existing atropine safety data.

Answer

The study is believed to be adequately powered at around 85%. The independent committee will provide a go/no-go recommendation, after which Eyenovia would unblind and analyze the full data. A positive result could accelerate an NDA filing to the first half of 2026. The FDA already allows leveraging data from the LAMP and ATOM studies, but Eyenovia also believes its own safety data will be superior and important for the label.

Matthew Kaplan of Ladenburg Thalmann asked about the company's current thinking on the launch plan for AXS-05 in ADA, specifically whether it would be filed as a line extension for Auvelity or as a new brand.

Answer

CEO Herriot Tabuteau responded that the company has the flexibility to file AXS-05 as either a supplemental NDA (sNDA) for Auvelity or a freestanding New Drug Application (NDA). He noted that the final approach is still being evaluated and will be a 'very well thought-out decision.'

Asked about the review timeline for the next-gen device, the nature (smooth vs. chunky) of future revenue growth, and the gross margin trajectory for calendar year 2025.

Answer

The next-gen device review should be smoother but the timeline is uncertain. Revenue growth is expected to be chunky due to varying contract sizes. Gross margins are targeted to approach 60% by the end of calendar 2025, with some potential variability from international sales mix.

Matthew Kaplan from Ladenburg Thalmann asked about the next-generation transport-ready device, including whether its PMA supplement review could be faster than the first-generation's. He also sought more color on the expected 'accelerated top line revenue growth,' questioning if it would be smooth or 'chunky,' and asked for the expected gross margin evolution through calendar year 2025.

Answer

Executive Steven Lisi expressed hope that the PMA supplement review for the next-gen device would be smoother and faster, confirming the system is ready and will be showcased soon. He stated that revenue growth will likely be 'chunky' due to the varying sizes of hospital contracts, which can range from under $100k to over $1M annually. Lisi also reiterated the long-term gross margin target of 60-65%, suggesting the company could be close to 60% by the end of calendar 2025, with international sales mix being a potential variable.

Asked for an update on the device software upgrade timeline, the primary drivers of growth (new vs. existing customers), and the reasons for the updated timeline for the cardiac surgery PMA supplement and its potential revenue impact.

Answer

Device upgrades will continue for the next few quarters and finish near the end of the fiscal year. Growth is primarily driven by acquiring new customers, aided by referrals. The PMA supplement timeline is still for the second half of the calendar year, just later in the half, and any potential revenue from it is not included in the current fiscal '25 guidance.

Matthew Kaplan asked for details on the unmet medical need within the progressive MS (PMS) market, including its specific subgroups.

Answer

CEO Dr. Daniel Vitt and President & COO Jason Tardio addressed the question. Dr. Vitt highlighted the significant unmet need, noting there are no approved treatments for non-active secondary progressive MS and only one infused option for primary progressive MS. Jason Tardio quantified the market, citing ~175,000 non-relapsing SPMS patients and ~120,000 PPMS patients in major markets. He emphasized that vidofludimus calcium's non-immunosuppressive, neuroprotective profile could make it a gold standard, especially for older patients where immunosuppressants are a concern.

Matthew Kaplan asked for more detail on the unmet medical need within progressive MS (PMS) and its specific subgroups.

Answer

CEO Dr. Daniel Vitt highlighted the significant unmet need, particularly for non-active secondary progressive MS, which has no approved treatments. President and COO Jason Tardio added that this subtype affects ~175,000 patients in major markets. He also noted the ~120,000 patients with primary progressive MS, where the only approved therapy is an infusion, and positioned vidofludimus calcium's non-immunosuppressant, neuroprotective profile as a potential gold standard for these older, progressing patients.

Matthew Kaplan from Ladenburg Thalmann asked for more detail on the unmet medical need within progressive MS (PMS) and its specific subgroups.

Answer

CEO Dr. Daniel Vitt and President & COO Jason Tardio addressed the question. Dr. Vitt highlighted the critical unmet need, especially for non-active secondary progressive MS, which has no approved treatments. Mr. Tardio provided market data, noting approximately 175,000 non-relapsing SPMS patients and 120,000 PPMS patients in major markets. He emphasized the reluctance to use immunosuppressants in older, progressing patients and positioned vidofludimus calcium's non-immunosuppressant, neuroprotective profile as a potential future standard of care.

Matthew Kaplan of Ladenburg Thalmann inquired about the reception of the interim neurofilament light chain (NfL) data from the CALLIPER study at the ACTRIMS meeting and asked about the potential outcomes of the upcoming interim futility analysis for the Phase III ENSURE program.

Answer

CEO Dr. Daniel Vitt stated that the strong NfL reduction data was very well-received by experts, noting it was a uniquely strong result. He clarified that the ENSURE futility analysis is a technical check on statistical assumptions and event rates, not a review of medical data. The potential outcomes are a recommendation to continue as planned or a suggestion for a sample size adjustment.

Matthew Kaplan questioned if the FDA's call for an ODAC was due to controversy over the single-arm ENVISION trial design and also inquired about the company's cash runway through the UGN-102 launch.

Answer

CMO Dr. Mark Schoenberg clarified that the FDA had agreed in writing that the single-arm trial could support approval and believes the ODAC is for a public vetting of a paradigm-changing therapy, not due to controversy. CFO Chris Degnan affirmed that the company's $254 million cash position is sufficient to reach profitability based on current plans.

Matthew Kaplan of Ladenburg Thalmann asked for details on patient persistence rates for BRIUMVI compared to competitors and inquired about the timeline for completing studies to get the faster infusion data from the ENHANCE trial into the product's label.

Answer

Chief Commercialization Officer Adam Waldman stated that based on competitor published data, BRIUMVI's week 24 compliance appears to be at least as good, if not better. CEO Michael Weiss estimated that getting the faster infusion into the label is likely a 2-to-3-year process, involving a randomized trial that would start next year and take about a year to enroll.

Matthew Kaplan asked for an update on progress with the Kidney Care Act, specific court timelines for the APA lawsuit injunction, and feedback from the recent ASN meeting regarding how physicians are utilizing XPHOZAH.

Answer

President and CEO, Mike Raab, highlighted the growing support for the Kidney Care Act, with 38 House and 2 Senate co-sponsors, and noted the court must decide on the injunction by year-end. Chief Commercial Officer, Eric Foster, added that feedback from ASN was strong, with physicians using XPHOZAH on-label with binders and seeing significant results in patients.

Matthew Kaplan of Ladenburg Thalmann asked if the initial CATALYST study results, which showed high hypercortisolism prevalence, were driving increased screening and Korlym prescriptions.

Answer

Sean Maduck, President of the Endocrinology division, confirmed that the CATALYST results are beginning to have an impact, though the full effect on clinical practice will take time and is expected in 2025 and beyond. He noted that increased physician awareness of hypercortisolism's prevalence is already driving volume growth for Korlym, and the superior profile of relacorilant is expected to accelerate this, positioning the company for a potential $3 billion business in five years.

Matt Kaplan of Ladenburg Thalmann inquired about plans for releasing additional open-label data from the Phase Ib study, the expected enrollment timeline for the Phase II BESTOW study, and the company's perspective on using neurofilament light chain (NFL) as a biomarker in future ALS studies.

Answer

CEO David-Alexandre Gros confirmed that Eledon will continue to share data from the Phase Ib study at major conferences as more patients are enrolled and longer-term data becomes available. He projected that enrollment for the BESTOW study would take approximately a year and a half. President and Chief Scientific Officer Steven Perrin added that the company would incorporate NFL as a biomarker in future ALS studies, acknowledging the FDA's recent receptiveness to it.

Matt Kaplan from Ladenburg Thalmann asked about the potential read-through from the initial Phase 1b renal transplant data to the planned Phase 2 superiority study and what key metrics to look for in the initial three-month data from the islet cell transplant program.

Answer

Chief Medical Officer Jeff Bornstein stated that the Phase 1b kidney transplant data will provide insights into safety, rejection prevention, and graft function (GFRs), which will directly inform the endpoint for the Phase 2 study. CEO David-Alexandre Gros added that the trial designs are similar. For the islet cell transplant program, Bornstein and Gros explained that the initial data will focus on safety, graft function, rejection rates, glucose control, and the need for exogenous insulin, with the hope that a single transplant could be sufficient.

Matt Kaplan from Ladenburg Thalmann asked about the company's cash runway following cost cuts and recent financing, the Q1 revenue breakdown for Talicia and Movantik, the regulatory path for opaganib in COVID-19, and the development timelines for RHB-204 with or without external funding.

Answer

CEO Dror Ben-Asher and CFO Micha Ben-Chorin affirmed the company has sufficient cash runway to reach operational profitability. Micha Ben-Chorin provided the Q1 revenue breakdown: Movantik (over $14M), a Talicia out-licensing deal ($2M), and Talicia commercial sales. COO Gilead Raday stated that a positive confirmatory study is the likely path for opaganib's approval and that external funding could accelerate the RHB-204 NTM program.

Matthew Kaplan asked for the timeline of the opaganib Phase 2/3 data release, the trial's powering assumptions, next steps for the prostate cancer program, the timeline for the RHB-204 NTM program, and a revenue breakdown for Movantik and Talicia.

Answer

CEO Dror Ben-Asher confirmed opaganib data is 'upcoming' and that the prostate cancer program's next steps will be decided after data presentation. COO Gilead Raday detailed the trial's powering assumptions based on Phase 2 results. CEO Dror Ben-Asher noted the RHB-204 timeline is uncertain due to COVID-related enrollment delays. CFO Micha Ben-Chorin and CEO Dror Ben-Asher clarified that Movantik constitutes about 90% of revenues.

Matthew Kaplan from Ladenburg Thalmann & Co. Inc. asked about the market opportunity for XOMA 358, the estimated cost of Phase 3 development, the company's current debt levels, and its repayment schedule.

Answer

SVP Paul Rubin detailed the market size for congenital hyperinsulinism and post-bariatric surgery hypoglycemia, noting pivotal trials could be small. CFO Tom Burns outlined the debt structure, including €12M to Servier, $13.5M to Novartis, and a $20M facility from Hercules Capital, along with their respective repayment timelines. CEO John Varian added that cash projections exclude potential new deals.

Matt Kaplan of Ladenburg Thalmann requested more detail on the XOMA 358 program costs, the company's expected future burn rate, clarification on the gevokizumab program's path forward, and a breakdown of the company's debt obligations.

Answer

Tom Burns, VP of Finance and CFO, explained that the XOMA 358 studies are significantly less expensive than the EYEGUARD trials and that the company's cash burn will be substantially lower than the previous $60-65 million guidance. Paul Rubin, SVP of R&D and CMO, confirmed that only the pyoderma gangrenosum study for gevokizumab will continue, with strict go/no-go checkpoints. John Varian, CEO, and Tom Burns then detailed the company's debt, including obligations to Servier, Novartis, and Hercules, and expressed confidence in resolving the Novartis debt.