Core Viewpoint - BioMarin Pharmaceutical has agreed to acquire Amicus Therapeutics for $4.8 billion, enhancing its portfolio with therapies for lysosomal storage disorders [1] Group 1: Acquisition Details - BioMarin will pay $14.50 per share for Amicus, representing a 33% premium over Amicus' stock value of $10.89 on December 18 [1] - This acquisition is the largest in BioMarin's history and has resulted in a 17.71% increase in BioMarin's stock price, from $51.95 to $61.15 [2] Group 2: Acquired Therapies - The acquisition includes rights to Amicus' Fabry disease therapy, Galafold, which generated $458.1 million in 2024, an 18% increase from 2023 [2] - Galafold is the only FDA-approved chaperone therapy for Fabry disease and has market exclusivity until January 30, 2027, following a patent litigation with Teva Pharmaceuticals [3] - BioMarin will also acquire the FDA-approved Pompe disease combination therapy, Pombiliti plus Opfolda, which generated $22 million in 2024 [4][5] Group 3: Future Growth Strategy - BioMarin has been focusing on growth in the pharmaceutical sector, undergoing significant restructuring in 2024, which included laying off 225 employees [7] - The company has adopted a strong dealmaking strategy, signing 10 deals in 2025, the highest number since 2010 according to GlobalData's Pharmaceutical Intelligence Center [7] - BioMarin is also acquiring rights to the investigational oral small molecule DMX-200, currently in Phase III trials for focal segmental glomerulosclerosis [6]

Summary of Amicus Therapeutics Conference Call Company Overview - **Company**: Amicus Therapeutics (NasdaqGM:FOLD) - **Focus**: Developing transformative therapies for rare diseases, particularly Fabry disease and Pompe disease [4][5] Key Products and Financial Performance - **Galafold**: - Approved for Fabry disease, on the market for nearly 10 years in Europe and 8 years in the U.S. - Achieved over $500 million in revenue for the first time this year, with a growth rate of 10%-15% [5][6] - The global market for Fabry disease is estimated at $2.4 billion, up from $1 billion previously [10] - **Pombiliti and Opfolda (POMOP)**: - Approved for Pompe disease, launched in the last couple of years. - Expected revenue growth of 50%-65% this year [5][6] - Projected to generate over $100 million in sales this year [23] Market Dynamics and Growth Drivers - **Fabry Disease Market**: - Initial estimates of incidence were one in 50,000 live births; recent studies suggest it may be closer to one in 3,000 [10] - The diagnosed untreated population has grown from 5,000 to 6,000 patients [10] - Increased awareness and availability of genetic testing are driving patient identification [11][12] - **Patient Identification Initiatives**: - Collaborations with healthcare systems (e.g., NHS in the U.K.) to identify undiagnosed patients [13][14] - Use of AI and partnerships (e.g., with OM1) to enhance patient finding [15][19] Competitive Landscape - **Galafold vs. Competitors**: - Concerns about Sanofi's upcoming phase three data for their substrate reduction therapy (SRT) were addressed, noting that Galafold's mechanism is different and may not be directly impacted [21][22] - **Pombiliti Market Position**: - The company is seeing increased patient switches from competitor products, with a significant uptick in the last two quarters [26][29] - The U.S. market is showing strong momentum, with the company gaining market share in the U.K. and other European regions [27][32] Pipeline and Future Opportunities - **FSGS Program**: - Amicus has licensed DMX-200 for FSGS, a rare kidney disease, with potential for significant market opportunity [36][38] - The FDA has indicated that proteinuria could serve as a primary endpoint for the ongoing phase three study, which is promising for regulatory approval [39][40] - **Regulatory Strategy**: - Plans to engage with the FDA regarding the study endpoints and potential accelerated approval paths [40][41] Financial Position and Strategic Focus - **Cash Position**: - The company has over $260 million in cash and is GAAP profitable, indicating a strong financial foundation [52] - **Business Development Strategy**: - Focus on late-stage, de-risked assets with potential for revenue growth, particularly in the Fabry and Pompe disease spaces [52] Conclusion - Amicus Therapeutics is positioned for continued growth with strong product performance, strategic initiatives to identify and treat more patients, and a promising pipeline in rare diseases. The company is committed to maintaining financial discipline while pursuing opportunities for expansion and profitability [52][53]

Summary of Amicus Therapeutics (FOLD) Conference Call Company Overview - Amicus Therapeutics focuses on developing therapies for rare diseases, currently offering two commercial products: Galafold and Pombility AppFolda for Pompe disease [3][4] Financial Performance - Galafold generated over $128 million in global sales in Q2, maintaining a growth rate of 10% to 15% [4] - Pombility AppFolda achieved $26 million in global sales in Q2, aligning with a growth guidance of 50% to 65% [5] - The company is on track to achieve GAAP profitability in the second half of the year [6] Market Dynamics - The Fabry disease market has seen a significant increase in diagnosed patients, with the treated market doubling from 10,000 to 12,000 patients [10][11] - There are now 6,000 diagnosed but untreated patients, leading to a total diagnosed market of 18,000 patients [11] - The potential for Galafold is growing as more patients are identified, with estimates suggesting that 25,000 diagnosed patients could exist in the future [15] Product Insights - Galafold is established as the standard of care for patients with amenable mutations, capturing approximately 70% of the global market share for treated amenable patients [21] - The company has a large registry to support real-world evidence of Galafold's impact on patient outcomes [21] - The demand is shifting towards naive patients, with 80% of demand now coming from newly diagnosed patients [22] Pipeline Development - Amicus has licensed DMX-200 from Dimerix, targeting focal segmental glomerulosclerosis (FSGS), a rare kidney disease with no approved treatments [5][31] - There are an estimated 40,000 to 80,000 patients with FSGS in the U.S., presenting a significant market opportunity [53] - The company aims to combine DMX-200 with standard treatments to improve patient outcomes [41] Future Projections - The company anticipates that Galafold could contribute approximately 60% of projected billion-dollar sales in the coming years [24] - The Pompe disease market is expected to grow from $1.5 billion to $2 billion, with Amicus aiming for a 50% market share [57] - The company is optimistic about the potential for DMX-200 to become a billion-dollar product, especially as it explores additional indications [55] Challenges and Opportunities - The complexity of diseases like FSGS presents challenges in targeting effective treatments [35] - There is a need for improved diagnostic capabilities, particularly for underrepresented patient populations [19] - The company is focused on building its pipeline and leveraging its financial strength to support growth [62] Conclusion - Amicus Therapeutics is positioned for growth with its existing products and new pipeline developments, particularly in the rare disease space, while maintaining a strong financial outlook and commitment to patient care [61][62]

Summary of Alterity Therapeutics Conference Call Company Overview - **Company**: Alterity Therapeutics (ATHE) - **Focus**: Development of therapies for neurodegenerative disorders, specifically targeting multiple system atrophy (MSA) and other Parkinsonian disorders [10][11] Industry Context - **Biotech Sector**: The conference is part of the Pink Asset Management Monsoon Communications Biotech Showcase 2025, highlighting the growing interest in biotech investments [1][2] - **Market Potential**: There are approximately 175 listed life sciences companies on the ASX, making it the third largest biotech exchange globally [2] Core Points and Arguments - **Therapeutic Development**: Alterity's lead asset, ATH434, is a first-in-class oral therapy for MSA, which has received FDA fast-track designation and EU orphan designation [10][17] - **Clinical Trials**: Positive phase two data was disclosed, showing significant efficacy in slowing disease progression in MSA patients [11][27] - **Mechanism of Action**: ATH434 targets the misfolding of alpha-synuclein and imbalanced iron in the central nervous system, which are critical in the pathology of neurodegenerative diseases [15][16] - **Patient Population**: MSA affects up to 50,000 patients in the U.S., with a median survival of seven to eight years post-symptom onset [17][18] Clinical Trial Results - **201 Study**: A randomized double-blind placebo-controlled study with 75 patients showed that the 50 mg dose declined by 48% less than placebo, while the 75 mg dose declined by 30% less than placebo [23][24] - **Orthostatic Hypotension**: A significant symptom in MSA, the placebo group deteriorated by six points, while both active treatment groups stabilized or improved slightly [24] - **Safety Profile**: ATH434 demonstrated a clean safety profile with no serious adverse events related to the study drug [25][27] Future Plans - **Upcoming Presentations**: Alterity plans to present at the International Congress on Parkinson's Disease and Movement Disorders and target an end-of-phase two meeting with the FDA by the end of the year [28] - **Funding**: The company raised $40 million following the positive phase two data in February [31] Additional Insights - **Investor Engagement**: The event emphasized the importance of networking among biotech investors and companies, highlighting the collaborative nature of the sector [5][6] - **Historical Context**: The narrative included a historical perspective on Australian biotech, referencing early challenges faced by companies like Circadian [2][3] This summary encapsulates the key points discussed during the Alterity Therapeutics conference call, focusing on the company's advancements, clinical trial results, and future directions within the biotech industry.

Summary of Dimerix (DXB) Update / Briefing August 18, 2025 Company Overview - Dimerix is an ASX listed company (Code: DXB) focused on inflammatory diseases, particularly kidney and respiratory diseases [1] Key Asset and Clinical Study - The primary asset is DMX200 (also known as KYTOVRA), which is undergoing a phase three clinical study for focal segmental glomerulosclerosis (FSGS), a rare kidney disease with no current treatments available globally [2][3] - Dimerix has received orphan drug designation for FSGS, providing advantages such as expedited market access, pricing incentives, and market exclusivity [2] Clinical Trial Details - The phase three clinical trial aims to assess the efficacy of DMX200 in reducing inflammation and preventing kidney scarring [8] - Key surrogate endpoints include estimated glomerular filtration rate (eGFR) and proteinuria, which are critical for evaluating kidney function and disease progression [9][13] - The trial is designed to include approximately 286 patients, with 225 already recruited [15][16] - Interim analyses have shown positive outcomes, indicating that patients on DMX200 are performing better than those on placebo [17] Regulatory Engagement - Dimerix is collaborating with the FDA and a working group called Parasol to define appropriate clinical endpoints for FSGS [4][19] - The potential for accelerated approval is being explored based on the correlation between early and later endpoints [21][22] Market Opportunity - FSGS is classified as a rare disease, but increasing biopsy rates are expected to raise its prevalence and incidence, making it a commercially attractive opportunity [25][26] - There are currently no approved treatments for FSGS, but similar rare kidney diseases have products priced between $100,000 and $500,000 per patient per year [27][28] Commercial Partnerships - Dimerix has established partnerships with four commercial marketing partners across various regions, including the US, Europe, Canada, Australia, New Zealand, the Middle East, and Japan [29] - The total deal value with these partners is up to $1.4 billion, with over $65 million already received [30] Financial Position - As of June, Dimerix had nearly $70 million in cash, sufficient to support the phase three clinical trial and explore additional pipeline opportunities [31] Future Catalysts - Key upcoming milestones include FDA feedback from the Parasol group, completion of the blinded interim analysis, and full recruitment by the end of the year [32] - There are also opportunities for further licensing deals in regions such as China and Latin America [32][33]

Dimerix Overview - Dimerix is developing DMX-200, a lead drug candidate in Phase 3 clinical trial for focal segmental glomerulosclerosis (FSGS)[14] - FSGS is a rare kidney disease with no approved treatments, leading to irreversible kidney damage, dialysis, transplant, or death[14] - Dimerix has secured orphan drug designation for DMX-200, providing regulatory, marketing exclusivity, and pricing benefits in key territories[14] - Dimerix has licensing partners across key territories for DMX-200[14] Financial Achievements and Partnerships - Dimerix has received over AU$65 million in total payments to date[14,54] - Licensing deals are collectively valued up to approximately AU$1.4 billion in total upfront and potential milestone fees plus royalties[14,53] - A 3rd development and license agreement for DMX-200 in Japan is valued up to ¥10.5 billion (~AU$107 million) in upfront/milestones, plus royalties[17] - A 4th license agreement for DMX-200 in the United States is valued up to US$590 million (~AU$940 million) in upfront/milestones, plus royalties[17] Clinical Trial and Regulatory Progress - The FDA confirmed proteinuria as an acceptable endpoint for full marketing approval in the US[17] - The ACTION3 study has enrolled 225 patients, with 52 patients enrolled in the Open Label Extension Study as of August 5, 2025[33] - The Phase 3 trial aims to recruit a total of approximately 286 patients[33] - Dimerix held a positive Type C meeting with the FDA in March 2025 regarding proteinuria trial endpoints for full approval and potential accelerated approval for DMX-200[41] Market Opportunity - The estimated global incidence of FSGS is over 200,000 per year[61] - The estimated incidence of FSGS per year across all Dimerix licensed territories is approximately 50,071[61]

Financial Data and Key Metrics Changes - Total revenue for Q2 2025 reached $154.7 million, a 22% increase compared to Q2 2024, with an 18% growth at constant exchange rates [24] - Galafold revenue was $128.9 million, up 12% at constant exchange rates and 16% in reported terms [10] - Non-GAAP net income for Q2 2025 was $1.9 million, compared to $18.5 million in Q2 2024 [26] Business Line Data and Key Metrics Changes - Galafold saw a 13% year-over-year patient growth, with over 69% global market share for treated Fabry patients with amenable mutations [6][10] - Pombility and Upholda revenue reached $25.8 million, up 58% at constant exchange rates, with the U.S. contributing approximately 42% of revenue [14][15] - The company expects Pombility and Upholda to achieve a combined revenue growth of 50% to 65% for 2025 at constant exchange rates [16] Market Data and Key Metrics Changes - The U.S. market remains a significant driver, with over 1,000 patient starts for Galafold since launch [11] - The company launched Pombility and Upholda in six new countries in Q2 2025, expanding its market presence [18] - The untreated population for Fabry disease is estimated to be significantly larger than previously believed, indicating substantial market opportunity [13] Company Strategy and Development Direction - The company aims to achieve GAAP profitability in the second half of 2025, maintaining financial discipline while expanding its product portfolio [9] - Strategic partnerships, such as with Dimerix for DMX200, are key to addressing unmet needs in rare diseases [7][21] - The company is focused on expanding into new markets and increasing patient access, with plans for further geographic expansion in 2026 [85] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory for the remainder of 2025, citing strong demand for Galafold and the potential of Pombility and Upholda [5][6] - The company highlighted the importance of real-world evidence in driving physician and patient adoption of its therapies [33] - Management remains optimistic about the long-term potential of its products, particularly in underdiagnosed populations [13] Other Important Information - The company reiterated its full-year financial guidance for 2025, expecting total revenue growth of 15% to 22% [27] - Cash, cash equivalents, and marketable securities were $231 million as of June 30, 2025, down from $250 million at the end of 2024 [26] Q&A Session Summary Question: What clinical assessments resonate with physicians and KOLs for switching from Nexviazyme to Pombility? - Management indicated that physicians look for stability or improvement in patient outcomes, including biomarkers and quality of life measures [32][34] Question: Update on the additional manufacturing facility in Ireland? - The company expects the Irish facility to come online in the latter half of 2025, which will help meet global demand [38][40] Question: Confidence in IP position and potential for settlement? - Management remains confident in the strength of its IP and the potential for favorable settlements, reinforced by previous agreements [46][48] Question: Timelines for the new U.S. manufacturing process and impacts on COGS? - The U.S. drug product manufacturing facility is progressing, with timelines not yet specified, but the company is optimistic about its overall manufacturing strategy [52][54] Question: Insights on patient starts in ex-U.S. markets? - The company reported strong market share growth in the UK and Germany, with ongoing launches in new countries [62][64] Question: Drivers for patient switches at the two-year mark? - Management noted that switches are influenced by patient outcomes and physician assessments, with a focus on proactive treatment strategies [68][70] Question: Regulatory alignment with the FDA on proteinuria for DMX200? - The FDA has indicated that proteinuria can serve as a primary endpoint, which is a positive development for the program [74][80]

Summary of Amicus Therapeutics (FOLD) FY Conference Call - June 10, 2025 Company Overview - **Company**: Amicus Therapeutics - **Core Business**: Development of therapies for rare diseases, primarily focusing on Galafold for Fabry disease and Pombility for Pompe disease - **Sales Projections**: Expected to surpass $500 million in sales for Galafold and $100 million for Pombility in 2025 [3][4] Key Points and Arguments Financial Performance and Growth - **Galafold**: - Projected to achieve $1 billion in peak sales with a growth rate of 10% to 15% in 2025 [4] - Currently in a $2 billion global market, expected to grow to $3 billion over the next decade [8][9] - **Pompe Disease Therapies**: - Pombility and Upholda projected to achieve $100 million in sales with a growth rate of 50% to 65% [4] - Anticipated to reach $1 billion in sales in the future [4] - **New Asset - DMX200**: - Licensed from Dimerix for FSGS, a rare kidney disease, with potential blockbuster status [5] - Expected to contribute to significant revenue growth by 2028 [5] Market Position and Competitive Landscape - **Macro Environment**: - Company believes it is well-positioned to navigate macroeconomic challenges affecting the sector [7][8] - Emphasizes the undervaluation of Galafold and the potential for increased diagnoses in the Fabry disease space [8][9] - **Launch Dynamics**: - Initial launch delays due to COVID-19 impacted market entry for Pompe therapies [37] - Positive trends observed in the U.S. market with increasing patient switches from competitors [39][41] Regulatory and Operational Considerations - **Impact of Tariffs and Regulations**: - Minimal impact from tariffs due to low cost of goods for Galafold, which is manufactured in Switzerland [16][18] - Company is adapting to regulatory changes and believes it is insulated from potential negative impacts [20][22] - **FDA Dynamics**: - New leadership at the FDA is expected to support rare disease regulatory reforms, potentially benefiting Amicus [23][24] Guidance and Future Outlook - **Profitability Expectations**: - Guidance for GAAP profitability in the second half of 2025, with revenue growth projected at 15% to 22% [25][26] - Operating expenses expected to remain flat or increase slightly, allowing for positive free cash flow [27][29] - **Long-term Strategy**: - Focus on maintaining financial discipline while expanding the product portfolio [6][12] - Plans to leverage cash flow for new development opportunities in rare diseases [80] Additional Important Insights - **Market Dynamics**: - The company is seeing strong uptake in initial launch countries, with market shares reaching 20-30% in key markets [47] - New countries like the Netherlands are expected to significantly contribute to revenue through patient switches [50][52] - **Patient Diagnosis Trends**: - Increased diagnosis rates for Fabry disease due to lower costs of genetic testing and newborn screenings [64][66] - Potential for significant patient population growth as awareness and testing improve [67][70] Conclusion Amicus Therapeutics is positioned for strong growth in the rare disease market, with a focus on financial discipline and strategic expansion. The company anticipates achieving profitability in the near term while navigating macroeconomic challenges and regulatory changes. The potential for increased patient diagnoses and successful product launches underpins a positive outlook for future revenue growth.

Financial Data and Key Metrics Changes - Total revenue for Q1 2025 reached $125.2 million, a 13% increase year-over-year, with a 15% growth at constant exchange rates [33][34] - GAAP net loss for Q1 2025 was $21.7 million, or $0.07 per share, compared to a net loss of $48.4 million, or $0.16 per share in Q1 2024 [34] - Non-GAAP net income for Q1 2025 was $9 million, or $0.03 per share, compared to a non-GAAP net loss of $4.6 million, or $0.02 per share in the same period last year [34] Business Line Data and Key Metrics Changes - Galafold revenue reached $104.2 million, up 6% at constant exchange rates, with new patient starts globally increasing by 14% [9][11] - Pombility revenue for Q1 2025 was $21 million, up 92% at constant exchange rates, primarily driven by the US, Germany, and Spain [13][14] - Adjusted revenue growth guidance for Pombility and Upholda was revised from 65%-85% to 50%-65% at constant exchange rates [36] Market Data and Key Metrics Changes - The US contributed significantly to Galafold's growth, with an 18% increase in sales, while UK sales were impacted by higher VPAG rebates [11][45] - The company achieved a record number of patients on Galafold in Australia following its first-line listing [10] - Revenue was recorded in seven countries, with new patient starts anticipated in four additional countries in Q2 2025 [18] Company Strategy and Development Direction - The company entered a strategic collaboration with Dimerix to commercialize DMX200, a treatment for FSGS, enhancing its late-stage pipeline [24][26] - The company is focused on diversifying its supply chain and has onshored a portion of drug product manufacturing to the US [6][19] - The long-term outlook for Galafold and Pombility remains positive, with expectations of reaching combined sales of $1 billion by 2028 [7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving GAAP profitability in the second half of 2025, despite challenges faced in Q1 [7][36] - The management acknowledged the impact of unexpected factors on quarterly revenues but remains optimistic about sales acceleration for the remainder of the year [7][15] - The company is well-positioned to create substantial value for shareholders and deliver on its mission for patients [7][41] Other Important Information - The company reiterated its financial discipline and updated its full-year revenue growth guidance to 15%-22% from the previous 17%-24% [36] - The company is committed to maintaining high compliance and adherence rates for its therapies, which are crucial for long-term growth [16] Q&A Session Summary Question: What led to the higher-than-anticipated rebate for Galafold and Pombility? - Management explained that the rebate was a negotiated rate by the industry association in the UK, which was higher than expected, impacting revenue throughout the year [45][46] Question: What has changed regarding the cadence of ex-US launches and patient starts? - Management noted that reimbursement settlements led to a delay in patient starts, which are now expected to accelerate in the second half of the year [49][51] Question: Can you elaborate on the diligence process for the DMX200 licensing agreement? - Management highlighted that the diligence process involved careful review of data, market research, and discussions with physicians, leading to confidence in the asset's differentiation [58][60] Question: What is the expected timeline for the next interim analysis for DMX200? - Management indicated that the timeline for the next interim analysis could be around nine months, depending on the collaboration with Parasol and the FDA [72][74] Question: Will the VPAG rebate impact be recurring? - Management clarified that while the rebate will impact revenue this year, it is not expected to be a recurring event [86][87]

Financial Data and Key Metrics Changes - The company achieved total revenue of $125.2 million for Q1 2025, representing a 13% increase year-over-year, and a 15% increase at constant exchange rates [33] - GAAP net loss for Q1 2025 was $21.7 million, or $0.07 per share, compared to a net loss of $48.4 million, or $0.16 per share in Q1 2024 [34] - Non-GAAP net income was $9 million, or $0.03 per share, compared to a non-GAAP net loss of $4.6 million, or $0.02 per share in the same period last year [35] - Cash, cash equivalents, and marketable securities were $251 million as of March 31, 2025, compared to $250 million at December 31, 2024 [35] Business Line Data and Key Metrics Changes - Revenue for Galafold reached $104.2 million, up 6% at constant exchange rates, with a 14% increase in new patient starts globally [10] - Revenue for Pombility reached $21 million, up 92% at constant exchange rates, driven primarily by the US, Germany, and Spain [14] - The company adjusted its revenue growth guidance for Pombility and Upholda from 65%-85% to 50%-65% at constant exchange rates [36] Market Data and Key Metrics Changes - The US contributed significantly to growth, with 60% of total revenue generated outside the US and 40% from within [33] - The UK sales were impacted by higher VPAG rebates than previously anticipated, with the negotiated rate increasing from 15% to 22% [46] - The company recorded revenue in Q1 across seven countries, including the US, Germany, Austria, Spain, the UK, Switzerland, and Sweden [18] Company Strategy and Development Direction - The company entered a strategic collaboration with Dimerix to commercialize DMX200, a treatment for FSGS, enhancing its late-stage pipeline [25] - The company aims to achieve GAAP profitability in the second half of 2025 while maintaining financial discipline [9] - The company is focused on expanding its portfolio in rare diseases, particularly in neuromuscular and rare kidney diseases [92] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth trajectory, expecting continued double-digit revenue growth in core business areas [6] - The management acknowledged challenges in the UK market due to unexpected rebate increases but remains optimistic about patient growth compensating for this [46] - The company anticipates a significant acceleration in patient starts in the second half of 2025, particularly in new launch markets [52] Other Important Information - The company is onshoring a portion of its drug product manufacturing to enhance supply chain diversification [8] - The company reiterated its commitment to maintaining high compliance and adherence rates for its therapies [13] - The company is actively enrolling in pediatric clinical trials and generating real-world data to support its therapies [24] Q&A Session Summary Question: What led to the higher-than-anticipated rebate for Galafold and Pombility? - Management explained that the rebate was a negotiated rate by the industry association in the UK, which unexpectedly increased from 15% to 22% [46] Question: What has changed regarding the cadence of ex-US launches and patient starts? - Management noted that reimbursement settlements led to delays in patient starts, which are now expected to accelerate in the second half of the year [50][52] Question: Can you elaborate on the diligence process for the DMX200 licensing agreement? - Management highlighted that the diligence included careful review of data, market research, and discussions with physicians, leading to confidence in the asset's differentiation [60] Question: What is the expected timeline for the next interim analysis for DMX200? - Management indicated that the timeline for the next interim analysis could be around nine months, depending on the collaboration with Parasol and the FDA [71] Question: How does the company plan to position Pombility against traditional ERT in new markets? - Management emphasized the importance of demonstrating the product's value proposition and differentiated data to drive adoption in new markets [90]