GeneDx (WGS) 2025 Conference June 04, 2025 09:55 AM ET Speaker0 Good. Good morning. I'm Tycho Peterson from the Life Science Tools Diagnostics team. It's my pleasure to introduce our next company this morning, GeneDx. Before we jump into Q and A, going turn it over to Katherine to introduce some media. Speaker1 Wonderful. Well, thank you so much, Tycho. Thank you to the entire Jefferies team. Coming out of another busy ASCO news cycle, I'm happy to turn everyone's attention to another really important area ...

Gary Ingenito, MD, PhD is retiring from Catalyst after a successful careerCORAL GABLES, Fla., June 02, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX) today announced the appointment of William (Will) T. Andrews, MD, FACP as Chief Medical Officer. Dr. Andrews will report directly to the Company’s President and Chief Executive Officer, Richard J. Daly, and will serve as a member of the Company’s executive leadership team. He succeeds Dr. Gary Ingenito, who is ...

PRINCETON, N.J., May 30, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that management will participate in upcoming presentations at the following investor conferences in June. Jefferies Global Healthcare Conference 2025 in New York, NY, on Wednesday, June 4, 2025, at 11:40 a.m. ETGoldman Sachs 46th Annual Global Healthcare Conference 2025 in Miami, FL, on Tuesday, June 10, 2025, at 2:00 p.m. ET A live audio webcast of each presentation can also be accessed via the investors se ...

Core Insights - Eton Pharmaceuticals has received FDA approval for KHINDIVI, the first and only hydrocortisone oral solution, aimed at pediatric patients with adrenocortical insufficiency [1][3][9] - The commercial launch of KHINDIVI is expected in the week of June 2, with projected combined peak sales of KHINDIVI and ALKINDI SPRINKLE exceeding $50 million annually [1][6] Product Details - KHINDIVI is formulated as a 1mg/ml oral solution, designed for easy dosing without the need for refrigeration or mixing, making it suitable for patients with swallowing difficulties [2][4] - The product is indicated for replacement therapy in pediatric patients aged five years and older with adrenocortical insufficiency [9] Market Context - There are over 5,000 pediatric patients in the U.S. diagnosed with adrenal insufficiency, highlighting a significant market opportunity for KHINDIVI [6] - Eton's existing commercial team will promote KHINDIVI alongside ALKINDI SPRINKLE, providing additional treatment options for patients and caregivers [5][7] Strategic Partnerships - KHINDIVI will be available exclusively through Anovo, a specialty pharmacy, which will also manage the Eton Cares Program to assist patients with prescription fulfillment and financial support [7][8] Company Overview - Eton Pharmaceuticals focuses on developing treatments for rare diseases and currently has eight commercial products, including KHINDIVI and ALKINDI SPRINKLE [41]

Ms. Kline to lead Palvella’s commercial buildout for planned standalone U.S. launch of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for microcystic lymphatic malformations, a serious, rare, and lifelong genetic disease affecting an estimated more than 30,0000 patients in the U.S., if approved Under Kline’s leadership at Dompé, Oxervate®, a first-in-disease topical therapy for rare disease neurotrophic keratitis, scaled to over $500 million in annual U.S. sales while achieving early profitability ...

- Reports continued progress in the evaluation of assets for potential in-licensing and development in the areas of oncology and rare disease - - New data from pilot phase of ongoing CheMo4METPANC Phase 2b combination trial of motixafortide in PDAC, sponsored by Columbia University, to be presented at upcoming 2025 ASCO Annual Meeting – - APHEXDA performing well under Ayrmid stewardship - - Management to host conference call today, May 27th, at 8:30 am EDT - TEL AVIV, Israel, May 27, 2025 /PRNewswire/ -- Bi ...

Core Viewpoint - SpringWorks Therapeutics has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use for its MEK inhibitor, mirdametinib, aimed at treating a rare genetic disorder [1][2]. Company Developments - The CHMP has recommended conditional marketing authorization for mirdametinib to treat symptomatic, inoperable plexiform neurofibromas in patients aged two years and older with neurofibromatosis type 1 [2]. - If approved, mirdametinib will be the first therapy authorized for treating NF1-PN in the European Union [3]. - The FDA approved mirdametinib under the brand name Gomekli in February 2025, generating sales of $4.9 million in the U.S. during Q1 2025 [3]. Financial Performance - Year-to-date, SpringWorks shares have increased by 28.6%, contrasting with a 5.5% decline in the industry [5]. - Merck KGaA announced plans to acquire SpringWorks for $47 per share, totaling approximately $3.9 billion, with the transaction expected to close in the second half of 2025 [6]. Product Pipeline - Ogsiveo, an oral gamma-secretase inhibitor, was approved in the U.S. for treating desmoid tumors in November 2023 and generated $172 million in sales in 2024, with $44.1 million in Q1 2025 [7][8]. - Ongoing label expansion studies for Ogsiveo are targeting various cancer indications, with the EMA's opinion on its MAA expected in Q2 2025 [8]. Market Position - The acquisition by Merck KGaA is anticipated to enhance SpringWorks' ability to reach global markets and support patients with rare tumors [9].

Goldman Sachs 46 Healthcare Conference Presentation Date: Tuesday, June 10, 2025 at 8:40 AM ET Presentation Format: Corporate Presentation Webcast: Here Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The Company's first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, formerly known as DCCR, is a once-daily oral treatment for hyperphagia in adults and children four years of age and older with Prader-Willi syndro ...

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]

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 ...