Kyverna Therapeutics (KYTX) FY 2025 Conference September 03, 2025 11:00 AM ET Speaker0right, everyone. I think we'll get started with our fireside discussion. My name is Derek Archila. I'm one of the Wells biotech analysts. Very excited to have Qiverna Therapeutics and their CEO, Warner Biddle.Warner, thank you so much for joining us.Speaker1Thanks for inviting us, Derek, and thanks for hosting us. It's been great so far. Awesome. Well I didn't know there was a Las Vegas casino in the middle of Boston. Here ...

Disc Medicine (IRON) 2025 Conference September 03, 2025 10:55 AM ET Company ParticipantsJohn Quisel - CEO & DirectorConference Call ParticipantsKristen Kluska - Equity Research AnalystJohn QuiselReady.Kristen KluskaOkay. Hi. Good morning. I think it's still morning, everybody. I'm Kristen, one of the biotech analysts at Cantor.Very happy to be hosting Disc Medicine. We have doctor John Quisel from the team here. Thank you so much for taking the time to come to our conference today.John QuiselThank you. Grea ...

ProKidney (PROK) Conference September 03, 2025 10:30 AM ET Company ParticipantsYigal Nochomovitz - DirectorBruce Culleton - CEO & DirectorEthan Holdaway - VP - IR & Strategic FinanceYigal NochomovitzI think we can, we can get going since it's exactly 10:30. So, welcome everyone to the next session of the, Citi's BioPharma Back to School Summit. So we are literally back to school checking up on all the companies. So the next one is is my pleasure to introduce the the management from Pro Kidney. We have Bruce ...

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]

Here are three stocks with buy rank and strong momentum characteristics for investors to consider today, September 3rd:Halozyme Therapeutics (HALO) : This biopharmaceutical company which is focused on the development and commercialization of novel treatments for oncology indications by targeting tumor microenvironment, has a Zacks Rank #1 (Strong Buy), and witnessed the Zacks Consensus Estimate for its current year earnings increasing 10.5% over the last 60 days.Halozyme Therapeutics' shares gained 36.2% ov ...

Protagonist Therapeutics (PTGX) Conference September 03, 2025 09:45 AM ET Company ParticipantsGeoff Meacham - Managing DirectorDinesh Patel - Director, President & CEOGeoff MeachamAlright. Well, great. Well, welcome to the second day of the Citi Biopharma Back to School Summit. So I'm Jeff Beecham. I'm the senior biopharma analyst, and my my team's with me here as well.So we're thrilled to have Protagonist with us, and we have Dinesh Patel, CEO and President. So great to to see you. Thanks for joining. So m ...

Artiva Biotherapeutics (ARTV) 2025 Conference September 03, 2025 09:45 AM ET Company ParticipantsJosh Schimmer - Managing DirectorFred Aslan - President, CEO & DirectorJosh SchimmerAll right, I think we're ready to get started. Welcome everyone. I'm Josh Shimmer from the Kanter Biotech Equity Research Team. Very pleased to introduce from Artiva Biotherapeutics, Fred Aslan, Chief Executive Officer, to give us a snapshot of the program. I have a feeling we're going to be talking a lot about CYFLU during the c ...

BridgeBio Pharma (BBIO) FY 2025 Conference September 03, 2025 09:30 AM ET Company ParticipantsTiago Fauth - Director - Equity Research Small/Mid Cap BiotechnologyNone - ExecutiveAnanth Sridhar - COOTiago FauthAll right, perfect. Thanks, everyone, for joining us. I'm Chad Lefauche, I'm a biotech analyst here at Wells Fargo. We're joined today by the BridgeBio team. We're going to have a fireside chat, focus a little bit on the pipeline and some upcoming catalysts that we have. Towards the end, we'll talk a l ...

Financial Data and Key Metrics Changes - The company reported a significant increase in the number of treatment-naive patients on acoramidis, indicating a positive trend in commercial execution [47][53] - The gross-to-net ratio is expected to stabilize in the range of 30% to 40%, reflecting a more representative payer mix as of Q2 [55][56] Business Line Data and Key Metrics Changes - The ADH-1 program is transitioning from a single-center phase two study to a global phase three study with over 25 sites, which may lead to a more heterogeneous patient population [3][4] - In the phase two study of ADH-1, approximately 70% of patients responded to encaleret, and the company aims to replicate this success in phase three [16][38] Market Data and Key Metrics Changes - The prevalence of ADH-1 has been estimated at about 1 in 25,000 based on data from multiple biobanks, indicating a consistent finding across different cohorts [18][19] - The company is comparing the patient identification and market development strategies for ADH-1 to those used in the ATTR field, which has seen significant improvements in diagnosis rates [22] Company Strategy and Development Direction - The company is focused on driving disease education and the utilization of genetic testing to improve diagnosis rates for ADH-1 [19] - The strategy for encaleret includes differentiating the product through urine calcium reduction and long-term safety, aiming to establish it as a standard of care [27][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the launch of acoramidis, noting that the share of treatment-naive patients continues to increase, which is a positive indicator for long-term success [47][48] - The company is optimistic about the upcoming data presentations that may address skepticism regarding the efficacy of encaleret in chronic hypoparathyroidism [25][26] Other Important Information - The company is preparing for a webinar to provide more details on the ADH-1 program, indicating a commitment to transparency and stakeholder engagement [3] - Management highlighted the importance of compelling clinical data and new treatment guidelines in driving diagnosis rates for ADH-1 [13][14] Q&A Session Summary Question: What are the risks in translating phase two findings to phase three for ADH-1? - The main difference is the number of sites and patients, with phase three being a global study involving over 25 sites [3] Question: Why is PTH replacement therapy not optimal for ADH-1 patients? - PTH replacement does not address the root cause of the condition, and urine calcium levels remain high even with treatment [10][11] Question: How is the company addressing patient identification for ADH-1? - The company is focusing on compelling clinical data, new treatment guidelines recommending genetic testing, and increased awareness through ICD-10 coding [13][14][18] Question: What is the expected impact of competition on acoramidis? - The company has not seen significant pressure from competitors and maintains a strategy of parity access without needing to offer larger rebates [57][59] Question: What is the confidence level regarding the safety profile of infigratinib? - The phase two data provided confidence in a clean safety profile, and the company expects to continue monitoring safety closely in phase three [65][66]

Financial Data and Key Metrics Changes - The company reported a significant increase in the number of treatment-naive patients on acoramidis, indicating a positive trend in commercial execution [47][53] - The gross-to-net ratio is expected to stabilize in the range of 30% to 40%, reflecting a more representative payer mix as of Q2 [55][56] Business Line Data and Key Metrics Changes - The ADH-1 program is transitioning from a single-center phase two study to a global phase three study with over 25 sites, which may lead to a more heterogeneous patient population [3][4] - In the phase two study of ADH-1, approximately 70% of patients responded to encaleret, and the company aims to replicate this success in phase three [16][38] Market Data and Key Metrics Changes - The prevalence of ADH-1 has been estimated at about 1 in 25,000, consistent across multiple biobanks, indicating a stable understanding of the patient population [18][19] - The company is comparing its market potential to that of ATTR and X-linked hypophosphatemia, suggesting a similar trajectory in patient identification and market development [22][23] Company Strategy and Development Direction - The company is focused on driving disease education and the utilization of genetic testing to improve diagnosis rates for ADH-1 [19] - The strategy includes leveraging compelling clinical data and new treatment guidelines to enhance awareness and diagnosis of the condition [13][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the launch of acoramidis, noting that the share of treatment-naive patients continues to increase, which is a positive indicator for long-term success [47][52] - The company is optimistic about the upcoming data presentations that may address skepticism regarding the efficacy of encaleret in chronic hypoparathyroidism [25][66] Other Important Information - The company is preparing for a webinar to provide more details on the ADH-1 program, indicating a commitment to transparency and stakeholder engagement [3] - Management highlighted the importance of long-term safety and efficacy data for encaleret, particularly in relation to urine calcium reduction, which could differentiate it in the market [27][66] Q&A Session Summary Question: What are the risks in translating phase two findings to phase three for ADH-1? - The main difference is the number of sites and patients, with phase three being a global study involving over 25 sites [3] Question: Why is PTH replacement therapy not optimal for ADH-1 patients? - PTH replacement does not address the root cause of the condition, and urine calcium levels remain high even with treatment [10][11] Question: How is the company addressing patient identification for ADH-1? - The company is focusing on compelling clinical data, new treatment guidelines recommending genetic testing, and increased awareness through ICD-10 coding [13][14][18] Question: What is the expected impact of competition on acoramidis? - The company has not seen significant pressure from competitors and maintains a strategy of parity access without needing to offer larger rebates [57][58] Question: How does the company view the potential for generic competition for tafamidis? - The company believes that the existing patents provide strong protection against generics, and the discontinuation of Vyndaqel complicates entry for potential competitors [60][61]