PresentationGood afternoon, everyone. My name is Will Pickering. I cover U.S. biotech at Bernstein. Very pleased to be joined this afternoon by the team from United Therapeutics. I will pass it over to them for some opening remarks, and then we'll have a fireside chat after that.Yes. Thanks, Will. So to start today, we may make some forward-looking statements, and I encourage you to read our latest public filings for any risks and uncertainties associated with those statements. But now I'll pass it off to o ...

Core Points - ProPhase Labs Inc. has filed for Chapter 11 bankruptcy for its three COVID-19 testing lab units, which are owed tens of millions of dollars by insurance companies [1][2] - The bankruptcy filing aims to expedite the recovery of funds that ProPhase Labs believes are lawfully owed for its testing services [2] - The company anticipates cash collections of $50 million or more, net of legal and contingency fees, through litigation with insurance companies [3] Bankruptcy Details - The bankruptcy action is strictly limited to the COVID-19 testing lab units and does not affect ProPhase Labs or its other subsidiaries [6] - The bankruptcy process is unrelated to the company's exploration of a crypto treasury strategy [6] Crypto Treasury Strategy - ProPhase Labs' stakeholders approved proposals to pursue a crypto treasury strategy aimed at enhancing stockholder value and diversifying the corporate balance sheet [4][5] - The CEO stated that the crypto treasury strategy is designed to be accretive to current shareholders, leveraging the significant underlying value of the company's assets [5]

Group 1 - The presentation is part of the Bank of America Healthcare Conference, indicating a focus on the healthcare sector [1] - Tazeen Ahmad, a senior SMID biotech analyst, is leading the presentation for Ultragenyx [1] - Eric Crombez, the Chief Medical Officer of Ultragenyx, is present for the discussion [1]

Core Insights - Defence Therapeutics Inc. is advancing its Accum® platform, which enhances the delivery of antibody-drug conjugates (ADCs) for cancer treatment, potentially transforming precision oncology [1][4][5] Company Overview - Defence Therapeutics is a clinical-stage biotechnology company focused on developing next-generation ADC products using its proprietary Accum® technology, which allows for precision delivery of ADCs to target cells [9] Technology and Innovation - The Accum® platform provides "laser-guided precision" in delivering drugs inside cancer cells, improving payload efficiency and reducing toxic side effects [2][3] - The analogy used illustrates that while traditional methods send many soldiers (drugs) to target a cancer cell, Accum® allows for a more effective approach with fewer drugs achieving the desired effect [3] Market Potential - ADCs are among the most effective anti-cancer drugs, but their use has been limited due to dose-limiting toxicity; Accum® may enable their application as first-line therapies [3][4] - Defence plans to partner with existing ADC developers and expand the applications of Accum® to novel therapies, indicating a strong near-term revenue outlook [4][5] Intellectual Property Strategy - The company employs a "picket fence" approach to protect its intellectual property portfolio globally, covering multiple aspects of the delivery platform [4]

Alnylam Pharmaceuticals FY Conference Summary Company Overview - Alnylam Pharmaceuticals is a pioneer in RNAi therapeutics, with over 20 years of development focused on drug delivery and innovation. The company currently has six commercial products, with four being actively commercialized. The primary focus is on the launch of products targeting cardiomyopathy in the ATTR market, which is significantly larger than previous markets the company has operated in [3][4]. Financial Performance - Alnylam is on the verge of achieving profitability, a goal set five years ago. The company reported a significant increase in top-line growth, with TTR revenue globally reaching $544 million, representing a 77% year-over-year growth. In the U.S., revenue was $383 million, with a $170 million increase from Q1, driven primarily by the cardiomyopathy launch [4][6]. Market Dynamics - The ATTR market has been segmented into hereditary polyneuropathy (PN) and cardiomyopathy (CM). The hereditary market is estimated to have about 50,000 patients, while the CM market is approximately ten times larger. The company has successfully launched its products in the CM segment, with an estimated 1,400 patients on therapy by the end of Q2 [5][6]. Launch Strategy and Execution - The rapid establishment of provider setups and payer coverage was a key driver for the successful launch. The company initially expected a six to nine-month setup period but achieved this in a much shorter timeframe. The focus was on getting the drug on formulary at health systems, which are critical for patient treatment [7][8][10]. Demand and Patient Segmentation - The demand for the product has been balanced between first-line and second-line treatments. Initially, demand was primarily from second-line patients, but by the end of the quarter, first-line demand was building. The company estimates that only about 20% of patients are currently treated, indicating significant growth potential [15][16][17]. Competitive Landscape - The competitive environment includes products from Pfizer and BridgeBio. The majority of patients are still being treated with tafamidis, but there is a growing interest in new products. The company aims to leverage its data from the HELIOS-B study to drive market share [21][22]. Pricing Strategy - Alnylam maintained the same pricing for its new product as for its previous offerings, with expectations of gradual price reductions over time. The company reported that approximately 70% of patients had zero out-of-pocket costs, making the product accessible despite its high cost [24][25][26]. Guidance and Future Outlook - The company raised its net product revenue guidance by $600 million, reflecting strong performance and expectations for continued growth. The TTR franchise is expected to grow by about $1 billion in revenue compared to 2024, with plans to expand into additional markets outside the U.S. starting in 2026 [27][30][31]. Long-term Market Evolution - Alnylam anticipates the entry of generics for tafamidis around the end of 2028, which may shift the market dynamics. The company is preparing for this by focusing on combination therapies and positioning its products effectively [39][40][41]. Pipeline and Future Innovations - The company is developing ALN-TTRsc04, a third-generation TTR product expected to provide better efficacy and convenience with a dosing schedule of once every six months. This product is anticipated to launch around 2030, coinciding with the expected generic entry of tafamidis [43][49]. Conclusion - Alnylam Pharmaceuticals is positioned for significant growth with a strong pipeline and a focus on innovative therapies. The company is committed to achieving long-term profitability and expanding its market presence while navigating competitive dynamics and evolving market conditions [64][65].

Core Insights - FibroBiologics, Inc. is a clinical-stage biotechnology company focused on developing therapeutics for chronic diseases using fibroblasts and fibroblast-derived materials, holding over 270 patents [1][4] - The company will present updates on its fibroblast-based technologies at the 2025 Cell & Gene Meeting on the Mesa Conference scheduled for October 6-8, 2025 [2] - A clinical trial for diabetic foot ulcers is planned to be initiated in the first quarter of 2026 [2] Company Overview - FibroBiologics is based in Houston and is developing a pipeline of treatments targeting chronic diseases, including wound healing, multiple sclerosis, disc degeneration, psoriasis, orthopedics, human longevity, and cancer [4] - The company represents advancements in cell therapy and tissue regeneration [4] Event Details - The presentation at the 2025 Cell & Gene Meeting on the Mesa will take place on October 7, 2025, at 2:15 p.m. MST in FLW Ballroom F [2]

Core Insights - Stifel Financial Corp. is expanding its Venture Banking team by hiring senior bankers from Silicon Valley Bank, enhancing its expertise in the life sciences and healthcare sectors [1][2] - The new team members have extensive backgrounds in venture banking, corporate finance, and strategic advisory, collectively supporting hundreds of early- and growth-stage companies [2] - Stifel Bank has seen significant growth in its Venture & Fund Banking division, with over 100 bankers, more than $10 billion in loan commitments, and a doubling of venture client deposits in the past year [3] Company Strategy - The addition of the new team is part of Stifel's strategy to strengthen its capabilities in healthcare and life sciences, providing more resources to founders and investors [4] - The life sciences and healthcare sectors are experiencing transformative changes driven by AI, positioning Stifel to offer unique solutions for scaling companies [4] Recent Achievements - Stifel has acted as joint bookrunner on four biopharma financings since early September, raising a total of $1.1 billion, including the first biopharma IPO in seven months [4]

Core Insights - Vor Bio has appointed Dr. Navid Z. Khan as Chief Medical Affairs Officer, bringing over two decades of experience in medical affairs, commercial, and R&D functions [1][2] - Dr. Khan has overseen more than 40 development programs and successfully guided seven product launches in neurology and immunology [2] - The company is focused on advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development to address serious autoantibody-driven conditions [5] Company Overview - Vor Bio is a clinical-stage biotechnology company dedicated to transforming the treatment of autoimmune diseases [5] - The company aims to ensure strong scientific exchange, evidence generation, and meaningful engagement with patient and physician communities worldwide [3] Leadership Background - Dr. Khan previously held senior leadership positions at argenx, Akouos Inc., and Sarepta Therapeutics, where he played a pivotal role in launching therapies for Duchenne muscular dystrophy [2][3] - He earned his Ph.D. in Biomedical Engineering and Biotechnology and holds a B.S. in Biochemistry and Molecular Biology [4]

Core Viewpoint - ProPhase Labs, Inc. has announced that its three COVID-19 testing laboratory subsidiaries have filed for reorganization under Chapter 11 bankruptcy, aiming to recover tens of millions of dollars owed by insurance companies and to ensure long-term viability and growth of the laboratories [1][2][3]. Group 1: Bankruptcy Filing Details - The bankruptcy action is limited to the COVID-19 lab testing units, which are owed tens of millions of dollars by insurance companies [2]. - The company aims to streamline the recovery of funds that it believes are lawfully owed for approved testing services, with many claims previously approved but underpaid by insurance companies [2][3]. - The CEO stated that the Crown Medical Collections group will pursue litigation against insurance companies, estimating potential cash collections of $50 million or more, which could significantly exceed the company's current market capitalization [3]. Group 2: Impact on Company Operations - The bankruptcy process does not affect ProPhase Labs, Inc. as a parent company or its other initiatives, which include the development of a groundbreaking esophageal cancer test [3][4]. - The company continues to advance projects in health technology, including its Nebula Genomics and DNA Complete subsidiaries, which are making progress in genomic and DNA-based testing [3][4]. Group 3: Future Outlook - The company is focused on innovation and growth in health technology, with the bankruptcy filing seen as a necessary step to collect owed funds for reinvestment in ongoing projects [4]. - The exploration of a crypto treasury strategy by the parent company is unrelated to the bankruptcy proceedings involving the lab subsidiaries [4].

Group 1 - Seres Therapeutics plans to reduce operating costs and cut its workforce by approximately 25% [1] - These measures, along with current operating plans, are expected to extend the company's cash runway into the second quarter of 2026 [1]