NEW YORK, Sept. 29, 2025 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT) (the Company), a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for rare diseases, today announced that the Company completed a meeting with the U.S. Food and Drug Administration (FDA) to discuss the potential new drug application (NDA) submission for govorestat for the treatment of Charcot-Marie-Tooth Sorbitol Dehydrogenase (SORD) Deficiency (CMT-SORD) in the third quarter of 202 ...

Core Insights - Rigel Pharmaceuticals (RIGL) and Amicus Therapeutics (FOLD) are focused on developing treatments for rare medical conditions, aiming to establish leadership in their respective fields [1][2] Rigel Pharmaceuticals (RIGL) - Rigel's lead drug, Tavalisse, is an oral spleen tyrosine kinase inhibitor approved for chronic immune thrombocytopenia (ITP), generating $68.5 million in sales in the first half of 2025, a 44% increase year over year [3][6] - The company is also progressing with Rezlidhia, approved for relapsed/refractory acute myeloid leukemia (AML), with sales increasing 31% year over year in the first half of 2025 [4] - Rigel added Gavreto to its portfolio in 2024, contributing to revenue growth in the first half of 2025 [5] - The company raised its total revenue guidance for 2025 to $270-$280 million, up from a previous estimate of $200-$210 million, due to strong sales performance [6] - Rigel has a pipeline product, R289, in early-stage studies for myelodysplastic syndrome (MDS) and is exploring Rezlidhia's use in other cancers [7] Amicus Therapeutics (FOLD) - Amicus' lead product, Galafold, generated $233.1 million in sales in the first half of 2025, an 11% year-over-year increase, with patent protection extending to 2038 [8][9] - The company’s two-component therapy, Pombiliti + Opfolda, approved for late-onset Pompe disease, generated $46.8 million in sales in the first half of 2025, a 74% increase year over year [10] - Amicus is experiencing a shift of patients from Sanofi's Pompe disease drugs to Pombiliti and Opfolda, with expectations for increased patient starts in new markets in the second half of 2025 [11] - Despite the growth of Galafold and Pombiliti + Opfolda, FOLD remains heavily reliant on Galafold for revenue, posing a risk to the company's overall growth [12] Financial Estimates and Performance - The Zacks Consensus Estimate for Rigel's 2025 sales and EPS indicates a year-over-year increase of approximately 57% and 415%, respectively [13] - In contrast, Amicus' 2025 sales and EPS estimates imply a year-over-year increase of around 18% and 29%, respectively, with EPS estimates for 2025 trending downward [17] - Year-to-date, RIGL shares have increased by 80.1%, while FOLD shares have decreased by 11.3%, compared to an industry return of 11.7% [19] - Amicus is valued higher than Rigel based on the price-to-sales (P/S) ratio, with FOLD trading at 4.51 times trailing sales compared to RIGL's 2.05 [20] Comparative Analysis - Rigel holds a Zacks Rank 1 (Strong Buy), indicating a more favorable investment outlook compared to Amicus, which has a Zacks Rank 3 (Hold) [24] - Rigel's strong performance and optimistic guidance for 2025, driven by Tavalisse and other products, suggest significant upside potential [25] - Amicus' reliance on Galafold and competition from established players like Sanofi present challenges for sustained growth [26] - Rigel's expanding portfolio, improving earnings estimates, and lower valuation position it as a more attractive investment compared to Amicus [27]

Core Insights - The company is advancing its CMT-SORD program and has scheduled a meeting with the FDA in Q3 2025 to discuss the New Drug Application (NDA) submission for govorestat [1][4] - Full 12-month clinical results from the INSPIRE Phase 2/3 trial of govorestat for CMT-SORD were presented, showing slowed disease progression observed via MRI at 24 months [1][4] - A new sponsored Sorbitol Assay has been launched to assist in identifying patients suspected of having CMT-SORD [1][4] - The company has entered into an out-licensing agreement with Biossil, Inc. for AT-001, an investigational therapy for Diabetic Cardiomyopathy [1][9] CMT-SORD Program - A meeting with the FDA is planned to align on the NDA submission strategy for govorestat, focusing on comprehensive data for Sorbitol Dehydrogenase Deficiency [4] - The newly launched Urine Sorbitol Assay aims to support healthcare providers in identifying suspected CMT-SORD cases at no cost to patients [4] - Clinical results from the INSPIRE trial indicated that govorestat was generally safe and well-tolerated, with significant improvements in key secondary endpoints at 12 months [4] Other Development Programs - New data on govorestat for PMM2-CDG will be presented at the 2025 ASHG Annual Meeting [2][6] - The review of the govorestat development program for Classic Galactosemia is progressing as planned, including responses to the Complete Response Letter from the FDA [5] Financial Performance - As of June 30, 2025, cash and cash equivalents totaled $30.4 million, down from $79.4 million at the end of 2024 [8] - Research and development expenses for Q2 2025 were $9.9 million, slightly down from $10.0 million in Q2 2024 [8] - General and administrative expenses increased to $13.2 million in Q2 2025 from $10.6 million in Q2 2024 [8] - The net loss for Q2 2025 was $21.3 million, compared to a net income of $2.9 million in Q2 2024 [8][15]

Core Viewpoint - Mereo BioPharma is advancing its clinical programs for setrusumab and alvelestat, with significant financial results and operational updates provided for the second quarter of 2025, indicating a focus on rare diseases and a strong cash position to support operations into 2027 [1][2][9]. Group 1: Clinical Development - Data from the Phase 3 Orbit and Cosmic studies for setrusumab in osteogenesis imperfecta (OI) is expected by the end of 2025, with ongoing patient dosing and a focus on reducing fractures and improving functional parameters [2][5]. - The Phase 3 studies are being conducted in partnership with Ultragenyx, which has confirmed an acceptable safety profile for setrusumab during an interim analysis [5]. - Pre-commercial efforts for setrusumab are ongoing in Europe, including the SATURN program to understand the natural history of OI and generate data for health economic models [5]. Group 2: Financial Performance - For the second quarter of 2025, total research and development expenses increased to $5.4 million, up from $4.9 million in the same period of 2024, primarily due to increased costs associated with setrusumab [4]. - General and administrative expenses decreased to $5.5 million from $7.9 million in the second quarter of 2024, attributed to reimbursements and lower professional fees [7]. - The net loss for the second quarter of 2025 was $14.6 million, compared to $12.3 million in the same period of 2024, influenced by a foreign currency transaction loss of $5.4 million [8]. Group 3: Cash Position and Guidance - As of June 30, 2025, the company had cash and cash equivalents of $56.1 million, down from $69.8 million at the end of 2024, but sufficient to fund operations into 2027 [9]. - The company maintains its guidance that existing cash will support committed clinical trials and operational expenses, excluding potential partnership payments for alvelestat [9]. Group 4: Shareholder Information - Total ordinary shares issued as of June 30, 2025, were 795,001,444, with total ADS equivalents of 159,000,288 [10].

Soleno used $12.6 million of cash in its operating activities during the three months ended June 30, 2025, and had $293.8 million of cash, cash equivalents and marketable securities as of the end of the quarter. Cash as of the end of the second quarter excludes $230 million of gross proceeds that the company raised in July through an underwritten offering of common stock. Product revenue, net was $32.7 million for the three months ended June 30, 2025. VYKAT XR had not been approved or commercially launched ...

Company Overview - Soligenix Inc. is a late-stage biopharmaceutical company focused on developing and commercializing products for rare diseases with unmet medical needs [4] - The company is advancing its HyBryte(TM) platform, a novel therapy for treating cutaneous T-cell lymphoma (CTCL), primarily affecting older adults [3][4] - Successful U.S.-based manufacturing of HyBryte's active ingredient has been established, positioning the company for significant impact in the healthcare sector [3] Product Development - The Specialized BioTherapeutics segment is moving towards potential commercialization of HyBryte(TM) after completing the second Phase 3 study, with regulatory approvals anticipated [4] - Other development programs include synthetic hypericin (SGX302) for psoriasis, and dusquetide (SGX942) for inflammatory diseases, including oral mucositis in head and neck cancer [4] - The Public Health Solutions segment includes vaccine candidates such as RiVax(R) for ricin toxin, and CiVax(TM) for COVID-19, supported by government grants and contracts [5] Market Context - The aging U.S. population is facing increasing challenges from chronic and rare diseases, with over 30 million Americans affected by rare diseases [2] - The Trump administration's initiatives aim to improve access to treatments and accelerate medical innovation in response to this healthcare crisis [2]

Core Insights - Amicus Therapeutics reported adjusted earnings of 1 cent per share for Q2 2025, missing the Zacks Consensus Estimate of 2 cents, compared to a loss of 6 cents per share in the same quarter last year [1][6] Financial Performance - Revenues for Q2 2025 totaled $154.7 million, representing a 22% year-over-year increase on a reported basis and an 18% increase at constant exchange rates, surpassing the Zacks Consensus Estimate of $148 million [2][6] - Galafold sales reached $128.9 million, up 12% year-over-year at constant exchange rates, although it fell short of the Zacks Consensus Estimate of $133 million [4][6] - Sales of the newly approved combination drug Pombiliti + Opfolda amounted to $25.8 million, exceeding both the Zacks Consensus Estimate of $24 million and the internal model estimate of $25 million [4][6] Growth Drivers - The revenue increase was attributed to higher sales from Galafold and additional revenues from Pombiliti + Opfolda, with ongoing studies aimed at expanding treatment labels for both Fabry and Pompe diseases [3][6] - The company maintained its 2025 revenue growth guidance of 15-22% at constant exchange rates, with Galafold expected to grow by 10-15% and Pombiliti + Opfolda by 50-65% [8] Operating Expenses and Cash Position - Adjusted operating expenses rose 56% to $127.8 million in Q2 2025, primarily due to a $30 million upfront payment for licensing rights to DMX-200 [7] - As of June 30, 2025, Amicus had cash, cash equivalents, and marketable securities totaling $231 million, down from $250.6 million as of March 31, 2025 [7] Future Outlook - The company expects to achieve positive GAAP net income in the second half of 2025 and aims to exceed $1 billion in total sales by 2028 [9] - Amicus has licensed exclusive rights for the commercialization of DMX-200, a potential first-in-class treatment for FSGS, with the pivotal phase III study on track for full enrollment by the end of 2025 [10]

Financial Performance - Total revenue for Q1 2025 was $125 million, a 15% increase at CER (constant exchange rates)[4,56] - Galafold revenue for Q1 2025 reached $104.2 million, representing a 14% increase[10,12] - Pombiliti + Opfolda revenue for Q1 2025 was $21 million, a 92% increase at CER[18] - The company expects total revenue to surpass $1 billion in FY 2028[5] - The company is guiding for total revenue growth of 15% to 22% for FY 2025[60] Product Performance and Growth - Galafold is the only approved oral treatment for Fabry disease, holding a 69% share of treated amenable patients[9,10] - The company anticipates Galafold revenue growth of 10% to 15% for FY 2025[4,15,60] - Pombiliti + Opfolda revenue growth guidance is updated to 50% to 65% for FY 2025[5,20,60] - Approximately 2,730 individuals have been treated with Galafold as of the end of 2024[10] DMX-200 and Strategic Initiatives - The company has an exclusive U S license agreement with Dimerix for DMX-200, a Phase 3 program for FSGS (Focal Segmental Glomerulosclerosis)[4,34,76] - The upfront investment for DMX-200 is $30 million, with potential success-based milestone payments up to $560 million and tiered royalties[38] - DMX-200 targets a market of over 40,000 people in the U S with FSGS, a rare and fatal kidney disease[37]