Amylyx Pharmaceuticals (NasdaqGS:AMLX) FY 2025 Conference December 03, 2025 04:00 PM ET Company ParticipantsJustin Klee - Co-CEo and Co-FounderConference Call ParticipantsGeoff Meacham - Senior AnalystGeoff MeachamAll right. Okay, welcome to the Citi Global Healthcare Conference. I'm Jeff Meacham.Global Head of Healthcare.Global Head of Healthcare, thank you. And Senior Analyst, big cap and mid-cap biotech and pharma. Obviously, Jarvis Fang is up here with me from my team. We're thrilled to have Amylyx. So ...

Summary of BioCryst Pharmaceuticals Conference Call Company Overview - **Company**: BioCryst Pharmaceuticals (NasdaqGS: BCRX) - **Event**: Conference call discussing the proposed acquisition of Astria Therapeutics - **Date**: October 14, 2025 Key Points Acquisition Announcement - BioCryst announced the acquisition of Astria Therapeutics, which is expected to enhance its product pipeline and support its strategic growth pillars [2][10][20] Strategic Growth Pillars 1. **ORLADEYO**: A highly profitable product projected to reach $1 billion in peak sales by the end of the decade, with intellectual property protection until 2040 [10] 2. **Future Products**: Focus on rare disease assets, including the development of BCX17725 for Netherton Syndrome and the newly acquired Nevenibart for hereditary angioedema (HAE) [10][11] Nevenibart's Market Potential - Nevenibart is seen as a transformative therapy for HAE patients, particularly those currently on injectable therapies [12][13] - Approximately 5,000 HAE patients in the U.S. are on injectable prophylaxis, with a significant unmet need for less frequent dosing [14] - The potential for Nevenibart includes a dosing schedule of every three to six months, which could significantly improve patient compliance and satisfaction [14][59] Financial Projections - BioCryst anticipates that the combination of ORLADEYO and Nevenibart could drive double-digit annual revenue growth, reaching at least $1.8 billion by 2033 [15][19] - The acquisition is expected to be accretive to operating profit in the first full year post-launch of Nevenibart [19] - BioCryst has secured a strategic financing partnership with Blackstone, providing access to up to $400 million for the acquisition [19] Market Dynamics - The company believes that the introduction of Nevenibart will not negatively impact ORLADEYO, as both products can cater to different patient needs [86] - The competitive landscape includes established products like Takhzyro, but Nevenibart's unique dosing profile is expected to provide a first-mover advantage [29][58] Regulatory Confidence - BioCryst expressed confidence that the acquisition will pass regulatory scrutiny, citing a competitive market landscape with numerous existing and emerging players [89] Future Outlook - The company plans to continue pursuing additional acquisition opportunities after successfully integrating Nevenibart [47][48] - BioCryst's management emphasized their strong commercial execution capabilities in the rare disease space, which will be crucial for the successful launch of Nevenibart [15][78] Additional Insights - The call highlighted the importance of patient feedback in shaping treatment options and the company's commitment to addressing unmet needs in the HAE community [12][13] - The management team underscored their experience and readiness to execute the acquisition and subsequent product launch effectively [78][91]

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]