| Date: | Wednesday, August 6, 2025 | | --- | --- | | Time: | 4:30 PM Eastern Time | | United States: | 1-800-717-1738 | | International: | 1-646-307-1865 | | Conference ID: | 96180 | | Webcast: | Link | The conference call will be broadcast live and will be available for replay for 30 days on the Company's website, www.soleno.life. INDICATION REDWOOD CITY, Calif., July 28, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. ("Soleno") (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics ...

CELEBRATION, Fla., July 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid ...

Core Insights - Zevra Therapeutics is actively participating in the 42nd Annual Meeting of the Southeastern Regional Genetics Group (SERGG), showcasing its therapies MIPLYFFA and OLPRUVA, which are approved for treating rare diseases [1][2] Group 1: MIPLYFFA (arimoclomol) - MIPLYFFA is approved in the U.S. for treating Niemann-Pick disease type C (NPC) and has shown disease-modifying activity leading to sustained disease stabilization in patients over multiple years [2][7] - Data from an Expanded Access Program (EAP) indicates that patients treated with MIPLYFFA experienced stable disease over up to 3 years, contrasting with the average progression of 1.0–2.0 points per year on the Niemann-Pick type C Clinical Severity Scale [3] - A 48-month open-label trial demonstrated that patients treated with MIPLYFFA experienced sustained reduction in disease progression for at least 5 years [3] - MIPLYFFA targets the pathophysiology of NPC through two pathways, enhancing the expression of genes in the Coordinated Lysosomal Expression and Regulation (CLEAR) network [3] Group 2: OLPRUVA (sodium phenylbutyrate) - OLPRUVA is approved for treating certain patients with urea cycle disorders (UCDs) and can be administered via a gastrostomy tube, which is significant for managing UCD in some patients [2][4][18] - Studies confirm that OLPRUVA meets administration criteria through gastrostomy tubes at evaluated doses and volumes [4] - OLPRUVA is indicated for long-term management of UCDs in adults and children weighing 44 pounds (20 kg) or greater, but is not used for acute hyperammonemia [18] Group 3: Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases, aiming to provide life-changing treatments for patients with limited or no options [23] - The company employs unique, data-driven strategies to overcome challenges in drug development and commercialization [23]

Core Insights - Zevra Therapeutics announced the publication of long-term efficacy and safety results for MIPLYFFA (arimoclomol) in treating Niemann-Pick Disease Type C (NPC), demonstrating sustained disease progression halting over multiple years [1][2] Company Overview - Zevra Therapeutics is a commercial-stage company focused on therapies for rare diseases, aiming to provide transformational treatments for conditions with limited options [14] Product Information - MIPLYFFA is an approved therapy for NPC, indicated for use in combination with miglustat for patients aged 2 years and older [4] - The drug received FDA approval on September 20, 2024, and has shown the ability to activate transcription factors that regulate lysosomal function [3] Clinical Trial Results - The Phase 2/3 trial demonstrated that MIPLYFFA halted disease progression compared to placebo over a one-year duration, with long-term data showing sustained efficacy for at least five years in over 270 patients [2][3] - The open-label extension study confirmed no new safety concerns, aligning with previous trial results [2] Safety Information - Common adverse reactions reported include upper respiratory tract infection, diarrhea, and decreased weight, with hypersensitivity reactions noted in some patients [5][9][10] - MIPLYFFA may cause increases in serum creatinine without affecting glomerular function, primarily occurring in the first month of treatment [8]

Core Insights - Soligenix, Inc. is focused on developing and commercializing products for rare diseases with unmet medical needs, with a promising late-stage pipeline and potential strategic options including partnerships and M&A opportunities [1][3] Financial Overview - The company has sufficient capital and cash runway to meet its goals into Q1 2026, with expected peak annual net sales of HyBryte™ in the U.S. exceeding $90 million and a total addressable worldwide CTCL market estimated at over $250 million annually [2][13] - The total addressable worldwide psoriasis market opportunity with SGX302 is estimated to exceed $1 billion annually, while SGX945 in Behçet's Disease (BD) has a market opportunity of approximately $200 million annually, leading to potential global annual sales of around $2 billion [2][11] Clinical Development - The confirmatory Phase 3 FLASH2 study for HyBryte™ is ongoing, with top-line results expected in the second half of 2026, and enrollment is on track [4][5] - The ongoing Phase 2a study of SGX302 in mild-to-moderate psoriasis has shown promising results, with previous cohorts demonstrating biologic activity and early clinical success [7][10] - A pilot Phase 2a proof of concept clinical trial for SGX945 in Behçet's Disease is expected to yield top-line results in Q3 2025 [11] Market Potential - Psoriasis affects approximately 60-125 million people globally, with the global treatment market valued at around $30 billion in 2023 and projected to reach $58-67 billion by 2030 [8][9] - Behçet's Disease is an orphan disease with around 18,000 known cases in the U.S. and 50,000 in Europe, impacting the quality of life for patients [12] Manufacturing and Partnerships - The company has successfully transferred the manufacturing of synthetic hypericin to a U.S. facility, optimizing the process for larger batch production [10] - Soligenix is actively pursuing partnerships in ex-U.S. markets to enhance its marketing authorizations for HyBryte™ and explore other potential cutaneous indications [6][14]

Core Insights - Agios Pharmaceuticals has appointed Dr. Jay Backstrom to its Board of Directors, which is expected to enhance the company's strategic direction and clinical program advancements [1][2][3] Company Overview - Agios Pharmaceuticals is a leader in cellular metabolism and pyruvate kinase (PK) activation, focusing on therapies for rare diseases [1][4] - The company markets a first-in-class PK activator for adults with PK deficiency, representing the first disease-modifying therapy for this condition [4] Leadership and Expertise - Dr. Backstrom brings extensive experience from his previous roles, including President and CEO of Scholar Rock and Executive Vice President of Research and Development at Acceleron Pharma [2][3] - His leadership has been instrumental in advancing clinical programs to regulatory approval, particularly in rare diseases [2][3] Strategic Focus - Agios is focused on maximizing the potential of its PK activator, PYRUKYND (mitapivat), and advancing its pipeline of rare disease medicines [2][3] - The company is developing investigational medicines for conditions such as alpha- and beta-thalassemia, sickle cell disease, and myelodysplastic syndromes [4]

RESEARCH TRIANGLE PARK, N.C., July 07, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the appointment of Babar Ghias as chief financial officer (CFO) and head of corporate development. Mr. Ghias joins BioCryst from AvenCell Therapeutics, a clinical-stage CAR-T company focused on hematologic malignancies, where he served as CFO since 2022. "Babar brings extensive deal making and operational experience in addition to his previous roles as a CFO at rare disease companies ...

RLYB116 - C5 Inhibitor - RLYB116 is being developed as a potential first- and best-in-class treatment for severe, refractory hematologic diseases[4, 12] - The estimated peak commercial opportunity for RLYB116 in immune PTR (Platelet Transfusion Refractoriness) is over $1.1 billion annually, targeting approximately 20,000 patients[13, 50, 51] - The estimated peak commercial opportunity for RLYB116 in refractory APS (Antiphospholipid Syndrome) is over $4.0 billion, also targeting approximately 20,000 patients[13, 50, 51] - A Phase 2 trial for immune PTR is expected to begin in the middle of 2026, and a Phase 2 trial for refractory APS is expected to begin in the second half of 2026[13] - Clinical PK/PD study data for RLYB116 is expected in the second half of 2025, with Cohort 1 data in Q3 2025 and Cohort 2 data in Q4 2025[4, 18, 66] REV102 - ENPP1 Inhibitor - REV102 is a potential first- and best-in-class ENPP1 inhibitor for patients with hypophosphatasia (HPP)[4, 54] - Phase 1 study on track to initiate 2H 2026[55] - Preclinical data for REV102 in a later-onset HPP model is expected in the second half of 2025[4, 66] Financial Position - Rallybio's cash position as of March 31, 2025, was $54.5 million, which is expected to support operations into the first half of 2027[5]

Financial Performance & Growth - Catalyst Pharmaceuticals experienced a 43.6% increase in total net product revenue in 1Q25 compared to 1Q24[55] - The company's 1Q25 net product revenues reached $141.4 million, up from $98.4 million in 1Q24[61] - Net income (GAAP) for 1Q25 was $56.7 million, significantly higher than the $23.3 million reported in 1Q24[61] - Catalyst forecasts total revenues between $545 million and $565 million for the full year 2025[60] Product Portfolio - FIRDAPSE revenue for 1Q25 was $83.7 million, compared to $66.8 million in 1Q24[56] - AGAMREE revenue for 1Q25 was $30.4 million[56] - FYCOMPA revenue for 1Q25 was $35.6 million, compared to $22.0 million in 1Q24[56] - FIRDAPSE is projected to generate between $355 million and $360 million in revenue for 2025[60] - AGAMREE is projected to generate between $100 million and $110 million in revenue for 2025[60] - FYCOMPA is projected to generate between $90 million and $95 million in revenue for 2025[60] Market & Strategic Focus - The total addressable market for the LEMS population is estimated to be over $1 billion[29] - The total addressable market for the DMD population is estimated to be over $1 billion[39]

Financial Performance & Growth - Total revenue for Q1 2025 reached $125 million, representing a 15% growth [6] - Galafold revenue in Q1 2025 was $1042 million, with a 14% patient demand growth [23] - Pombiliti + Opfolda revenue in Q1 2025 was $21 million, showing a 92% growth at CER [50] - The company expects to surpass $1 billion in total revenue in FY 2028 [6] Strategic Priorities & Guidance - The company aims for a total revenue growth of 15-22% at CER in FY 2025 [7, 77] - Galafold revenue is projected to grow by 10-15% at CER in FY 2025 [6, 7] - Pombiliti + Opfolda revenue is expected to increase by 50-65% at CER in FY 2025 [6, 7, 53, 77] - The company anticipates positive GAAP net income during H2 2025 [7, 77] Product & Market Overview - The global Fabry market is projected to reach approximately $3 billion by 2029 [14, 15] - The global Pompe market was approximately $15 billion in 2024 and is expected to exceed $2 billion by 2029 [42, 43] - DMX-200, a Phase 3 program for FSGS, addresses a market affecting over 40000 people in the US [67]