Lineage Cell Therapeutics (LCTX) FY 2025 Conference May 20, 2025 02:30 PM ET Speaker0 Hey. Good afternoon, everybody. I'm gonna turn this on. Good afternoon, everybody. My name is Joe Pangenis, managing director here at HC Wainwright. Welcome back to our next fireside chat. Very happy to have with us Brian Culley, chief executive officer of Lineage Cell Therapeutics. And, so thank you very much for being here. And little before this presentation, I thought of a little analogy. Think, feel like I'm in front ...

Entrada Therapeutics (TRDA) FY 2025 Conference May 20, 2025 02:00 PM ET Speaker0 Hello, and welcome to the latest in this series of fireside chats here at the H. C. Wainwright BioConnect Conference. Thank you very much all for joining us. My name is Ram Selvaraju, and I'm a Managing Director and Senior Healthcare Equity Research Analyst within Wainwright's Equity Research Department. I'm joined here today by the next in our series of presenting companies, Entrada Therapeutics, represented by Nathan Dowden, ...

Aura Biosciences (AURA) FY 2025 Conference May 20, 2025 02:00 PM ET Speaker0 Great. I'd like to welcome everybody back to our afternoon session of the H. C. Wainwright BioConnect Investor Conference at NASDAQ. My name is Andres Maldonado. I'm a covering analyst here at the firm. And I'd like to welcome our next presenters, Aura Biosciences. And today we're joined by Jill Hopkins, Chief Medical Officer, President of R and D as well and Tony Gibney, Chief Financial and Business Officer. Welcome on behalf of H ...

Financial Data and Key Metrics Changes - The company is currently conducting a phase three pivotal trial focused on hormone receptor positive, HER2 negative breast cancer, with the lead asset being palisestrant [2][3] - The ongoing phase three trial is a monotherapy trial in the second and third line setting, with top-line results expected next year [3] - The company plans to initiate a first-line trial in the second half of the year, which will include ribociclib [3][4] Business Line Data and Key Metrics Changes - The company has two clinical stage assets: palisestrant in phase three and OP3136, a CAT6 inhibitor, in phase one dose escalation [4] - The phase two data for palisestrant showed a progression-free survival (PFS) of 7.3 months in the ESR1 mutant population, which is significantly better than the control arm [13] - The combination of ribociclib and palisestrant is expected to show favorable results compared to other combinations, with a median PFS of 15 months reported [20][21] Market Data and Key Metrics Changes - The company is competing in a rapidly evolving market for oral SERDs, with recent trials showing benefits primarily in ESR1 mutant patients [6][7] - The company aims to differentiate palisestrant by demonstrating its efficacy in both mutant and wild-type populations [13][18] Company Strategy and Development Direction - The company is focused on advancing its clinical trials and establishing palisestrant as a competitive treatment option in the breast cancer market [2][3] - The strategy includes a combination approach with ribociclib and palisestrant, which is expected to enhance treatment outcomes [20][21] - The company is also exploring the potential of OP3136 in various cancer types, including castration-resistant prostate cancer and non-small cell lung cancer [42][43] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the efficacy of palisestrant in the first-line setting, particularly for treatment-naive patients [25] - The company anticipates that the upcoming data updates will provide further insights into the performance of its assets and their potential market impact [33][34] Other Important Information - The company has a clinical collaboration with Novartis for the supply of ribociclib, which is a key component of its trials [30] - The company is preparing for potential data updates and has several catalysts expected later this year [34][46] Q&A Session Summary Question: How does palisestrant differentiate from other oral SERDs? - Management highlighted that palisestrant is a complete estrogen receptor antagonist, unlike others that are partial agonists, which may limit their efficacy in certain populations [6][7] Question: What are the expectations for the OPERA one trial design? - The trial is designed to test both mutant and wild-type populations separately, allowing for a more targeted statistical analysis [18] Question: How does the combination of ribociclib and palisestrant compare to other treatments? - The combination is expected to show significantly better results than existing treatments, with a median PFS of 15 months compared to 5 months in previous studies [20][21]

Financial Data and Key Metrics Changes - Fiscal year 2024 marked the second consecutive year and eighth consecutive quarter of market share gains for the company, with a decrease in operating expenses and the highest diluted earnings per share since 2018 [20] - The company ended the year with an increase in cash, cash equivalents, and short-term investments compared to fiscal year 2023 [20] Business Line Data and Key Metrics Changes - Old Navy, the largest brand, achieved a 3% sales comp for the year, becoming the number one specialty apparel brand and retailer in the US [21] - Gap brand regained market share in all four quarters of fiscal year 2024, reestablishing its presence in cultural conversations [22] - Banana Republic made progress in the premium lifestyle space, while Athleta stabilized revenue and maintained its position in women's active [23] Market Data and Key Metrics Changes - The company reported significant market share gains across its brands, particularly in the competitive landscape of specialty apparel [20][21][22] Company Strategy and Development Direction - The company is focused on four strategic pillars: operational and financial rigor, brand reinvigoration, strengthening its platform, and energizing its culture [5][20] - A new chief technology officer has been hired to enhance capabilities in e-commerce, data, and AI, integrating AI-driven analytics into various business functions [24] Management's Comments on Operating Environment and Future Outlook - The management acknowledged the dynamic environment and emphasized the need for continued focus on executing strategic priorities to create shareholder value [26] - The company aims to navigate uncertain times while building on its strengths and maintaining operational excellence [26] Other Important Information - The board has undergone refreshment with the addition of new directors to align with the evolving needs of the business [6][7] - The company has established a marketing shared services function to improve advertising spend returns and brand relevance [24] Q&A Session Summary Question: Are there any questions? - No questions were submitted during the Q&A session [27]

Financial Data and Key Metrics Changes - The company provided guidance for 2025 revenues between $768 million and $793 million, representing a year-over-year growth of approximately 25% to 29% [4][5] - Adjusted non-GAAP EBITDA guidance for 2025 is between $195 million and $205 million [4] - Q1 2025 revenues reached a record $197 million with adjusted non-GAAP EBITDA of $51 million [6] Business Line Data and Key Metrics Changes - The rare disease segment is the primary growth driver, with Cortrophin Gel expected to generate $265 million to $274 million in 2025, reflecting a year-over-year growth of 384% to 38% [8][9] - Generic revenues reached a record high of $98.7 million in Q1 2025, driven by successful new product launches [8] - The generics business is expected to sustain high single-digit to low double-digit growth, with a mid double-digit growth forecast for 2025 [37] Market Data and Key Metrics Changes - The ACTH market, which includes Cortrophin Gel, is projected to grow to $792 million in 2025, with a 16% growth rate [16] - The addressable market for Cortrophin Gel is significantly larger than the current patient population, indicating substantial growth potential [19][20] Company Strategy and Development Direction - The company aims to reinvest cash flows from generics and established brands into the rare disease business, which is expected to account for approximately half of 2025 revenues [4][36] - The acquisition of Alimera is intended to expand the rare disease business and align with the company's M&A strategy [24][32] - The company is focusing on expanding the adoption of Cortrophin Gel in targeted specialties and addressing sales factors impacting ILUVIEN and YUTIQ [38] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's positioning, with over 90% of revenues coming from U.S.-manufactured products and minimal reliance on China [5] - The management highlighted the strong growth momentum in the rare disease segment, particularly for Cortrophin Gel, and the ongoing investment in R&D to support this growth [20][23] Other Important Information - The company has a healthy balance sheet with $150 million in cash and a net leverage of 2.7 turns [4][38] - The generics business launched 17 products in 2024, including two competitive generic therapy products with exclusivity [33] Q&A Session Summary Question: What is the growth outlook for Cortrophin Gel? - Management indicated that Cortrophin Gel is on a strong multiyear growth trajectory, with significant headroom for expansion in its addressable market [19][20] Question: How is the company addressing the sales impact on ILUVIEN and YUTIQ? - The company is focusing on capturing larger opportunities in diabetic macular edema and chronic noninfectious uveitis, while also expanding the sales team to drive growth [38] Question: What are the priorities for 2025? - The company outlined four key priorities: expanding Cortrophin Gel adoption, addressing sales factors for ILUVIEN and YUTIQ, sustaining generics momentum, and expanding the rare disease business through M&A [38]

Financial Data and Key Metrics Changes - The company ended the fourth quarter with approximately $221 million in cash, providing a runway until the fourth quarter of 2026 with existing operations [40] Business Line Data and Key Metrics Changes - Respag, a drug developed by the company, demonstrated dose-dependent efficacy in a Phase 1b study for atopic dermatitis, with a significant improvement in the eczema area and severity index (EASI) score after correcting previous data errors [4][6] - The Phase 2b study for atopic dermatitis is set to enroll 400 patients, extending the dosing period to 16 weeks and including a maintenance arm for ongoing treatment [14][15] Market Data and Key Metrics Changes - The company is entering the alopecia market, where currently only JAK inhibitors are approved, positioning Respag as a potential first biologic treatment in this space [35] Company Strategy and Development Direction - The company aims to leverage its unique mechanism of action with Respag, which stimulates regulatory T cells, to differentiate itself from existing treatments in the atopic dermatitis and alopecia markets [10][32] - The partnership with TrialNet focuses on the role of Tregs in type 1 diabetes, indicating a strategic move into autoimmune disease prevention [38] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the Phase 2b study for atopic dermatitis, citing the robust design and previous positive results as indicators of potential success [16][19] - The company anticipates presenting top-line results for both the atopic dermatitis and alopecia studies in June and December respectively, highlighting a proactive approach to clinical development [34] Other Important Information - The company has transitioned Respag to a wholly owned asset after terminating its collaboration with Eli Lilly, allowing for independent development in multiple clinical trials [2] Q&A Session Summary Question: Can you provide an overview of the Respag history? - The company developed Respag and initially partnered with Eli Lilly in 2017, completing nine clinical studies before terminating the collaboration in April 2023 [2] Question: What are the expectations for the Phase 2b study? - The company aims to replicate the significant efficacy observed in the Phase 1b study, targeting a similar effect size with a larger patient population [19] Question: How is the company addressing the placebo effect in clinical trials? - The company has minimized the US footprint in trial enrollment and ensured that the majority of sites are led by board-certified dermatologists to maintain consistency in disease rating [24][26]

Financial Data and Key Metrics Changes - COYA Therapeutics is publicly traded on NASDAQ under the ticker symbol COYA, with a buy rating and a twelve-month price target of $18 per share [2] - The company anticipates receiving $8.4 million upon IND approval and trial initiation, with a total deal value of $700 million from the partnership with Dr. Reddy's [18][19] Business Line Data and Key Metrics Changes - The primary focus is on neuroinflammation, which is believed to drive the progression of neurodegenerative diseases [4] - COYA's lead asset, COIA-302, is prioritized for ALS, with an IND filing expected by the end of the current quarter and a Phase IIb trial planned [11][12] Market Data and Key Metrics Changes - The company is targeting niche orphan indications like ALS and frontotemporal dementia, while also pursuing mass market indications like Alzheimer's [9] - The current landscape for ALS trials has become more favorable for recruitment due to the withdrawal of other products, which has created enthusiasm among trial sites [20][22] Company Strategy and Development Direction - COYA aims to maximize value by pursuing both niche and broader market opportunities, with a focus on ALS and frontotemporal dementia for regulatory flexibility [10] - The company plans to continue generating data in frontotemporal dementia and Alzheimer's, while also exploring strategic partnerships to enhance its market position [43][45] Management's Comments on Operating Environment and Future Outlook - Management believes that the FDA remains receptive to ALS drug approvals if data supports it, despite recent challenges in the market [21][23] - The company is confident in its approach to neuroinflammation and its potential to address unmet needs in Alzheimer's disease, which is characterized by a lack of effective treatments [30][31] Other Important Information - COYA is developing a regulatory T cell-derived exosome platform aimed at treating systemic and neurodegenerative diseases driven by chronic neuroinflammation, which complements its existing programs [40][42] - The partnership with Dr. Reddy's provides COYA with a strategic advantage and a steady line of sight to non-dilutive funding [45] Q&A Session Summary Question: What is the current focus for COYA Therapeutics? - The immediate focus is on filing for ALS and starting the ALS study, which is expected to be a significant value driver [43] Question: How has the regulatory environment changed for ALS? - There has been no communicated change from the FDA, and recruitment for ALS trials is expected to be easier due to fewer ongoing trials [21][22] Question: What are the expected outcomes for future clinical development? - Relevant outcome measures will include Treg function and cognitive scales, with a focus on stabilizing or improving cognition in Alzheimer's patients [36][38]

Financial Data and Key Metrics Changes - The company reported half a million in revenue from the first month of the product launch, which is considered encouraging for a new product [13] - The cash position as of March 31 was reported to be between 113 million and 119 million, with a reduced cash burn rate extending the cash runway by up to 50 million, allowing the company to expect to get through the end of 2026 [38][39] Business Line Data and Key Metrics Changes - The company is tracking sales and intermediary metrics, with active files submitted to value analysis committees in over 40 trauma centers, which corresponds to nearly a quarter of the 200 level one trauma centers in the US [15] - The company anticipates that the substantial bulk of sales will occur in the second half of the year due to the lengthy value analysis committee process [19] Market Data and Key Metrics Changes - There are approximately 26,000 cases of vascular injury requiring surgery annually in the US, concentrated in about 200 level one trauma centers, indicating a finite number of call points for the company's sales efforts [7][8] - The company has developed a budget impact model showing that the cost of caring for patients treated with SIMVEST is lower than that for patients treated with synthetic grafts, which could drive adoption [20][21] Company Strategy and Development Direction - The company is focusing on expanding its product offerings, including evaluating engineered blood vessels for fistula repair in end-stage renal patients and has embarked on a phase three trial for this indication [23][25] - The company plans to file a supplemental BLA for the dialysis indication in the second half of 2026, contingent on positive interim analysis results from the ongoing trial [28] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the commercial launch, noting that while the initial uptake may be slow, they expect significant results as the process matures [18] - The company has not provided specific revenue guidance for the first year due to the lengthy value analysis committee process but indicated a consensus estimate of around 7 to 8 million for the year [37] Other Important Information - The company has completed three phase two trials for peripheral arterial disease, showing outstanding limb salvage rates, and is in the process of designing a phase three program [31][35] - Long-term military results and civilian clinical data are expected to be published soon, indicating a strong pipeline of news flow [30] Q&A Session Summary Question: What is the company's current cash position and expected cash runway? - The company reported a cash position of between 113 million and 119 million as of March 31 and expects to extend its cash runway through the end of 2026 due to a reduced cash burn rate [38][39] Question: What metrics are being tracked to ensure the success of SymVest in the market? - The company is tracking sales and intermediary metrics, including submissions to value analysis committees in trauma centers, which are crucial for hospital adoption [15] Question: What are the next steps in the company's clinical programs? - The company plans to publish results from the phase three trial for dialysis access and expects to file a supplemental BLA in the second half of 2026 if interim results are positive [28]

Zevra Therapeutics (ZVRA) FY 2025 Conference May 20, 2025 12:30 PM ET Speaker0 Alright. Good afternoon, everyone. My name is Brandon Folks. I'm one of the biopharma analysts here at H. C. Wainwright. And joining me for a fireside chat from Zebra Therapeutics is CEO, Neil McFarlane. Neil, thank you very much. Speaker1 Yeah. Thank you for having us. Speaker0 So maybe just to start, do you wanna just spend a few minutes giving people a quick overview of Zebra and the products that you currently have? Speaker1 ...