MedPath

Clinical Trial News

Molgramostim Shows Sustained Benefits in Phase 3 Trial for Autoimmune Pulmonary Alveolar Proteinosis

  • Molgramostim demonstrated a statistically significant improvement in lung diffusing capacity (DLCO%) at Week 24, which was sustained through Week 48 in aPAP patients.
  • The IMPALA-2 trial showed molgramostim significantly improved disease severity scores and ground glass opacification, indicative of reduced surfactant burden.
  • Responder analysis revealed higher proportions of patients achieving clinically meaningful improvements in DLCO% and respiratory questionnaire scores with molgramostim.
  • Savara plans to submit a Biologics License Application (BLA) for molgramostim in the first half of 2025, seeking approval for aPAP treatment.

Celcuity's Gedatolisib Projected to Reach $155M Annual Revenue by 2034 for Breast Cancer Treatment

  • Gedatolisib, a dual PI3K/mTOR inhibitor developed by Celcuity, shows promising market potential with projected annual revenue of $155 million by 2034 in the US market.
  • The drug is being evaluated across multiple solid tumors, including ER+/HER2-negative metastatic breast cancer, prostate cancer, and various gynecological cancers.
  • Celcuity reported increased investment in R&D, with operating losses expanding from $39.4M in FY2022 to $66.2M in FY2023, reflecting intensive development efforts.

Tenaya Therapeutics' Gene Therapy TN-201 Shows Promise with Projected $165M Revenue for Cardiomyopathy Treatment

• Tenaya Therapeutics' lead candidate TN-201, an AAV9-based gene therapy targeting MYBPC3, is being developed for treating hypertrophic cardiomyopathy with projected annual revenues of $165 million by 2038.
• The novel gene therapy utilizes adeno-associated virus serotype 9 vectors for intravenous delivery of the cardiac myosin-binding protein-C gene, representing a potential breakthrough in cardiac disease treatment.
• Despite operating losses of $131.2 million in FY2023, Tenaya continues advancing its diverse cardiovascular pipeline, including treatments for various forms of cardiomyopathy and heart failure.

Bristol-Myers Squibb's MYK-224 for Hypertrophic Cardiomyopathy Projects $41M Revenue by 2035

  • Bristol-Myers Squibb is developing MYK-224, an oral myosin-targeting drug for obstructive hypertrophic cardiomyopathy and diastolic heart failure, with projected US revenue of $41 million by 2035.
  • The drug's development strategy focuses on addressing significant unmet needs in cardiac conditions, particularly heart failure with preserved ejection fraction (HFpEF).
  • BMS demonstrates continued investment in cardiovascular therapeutics despite recent overall revenue decrease of 2.5% in FY2023, maintaining an operating margin of 20.9%.

Regulus Therapeutics' RGLS-8429 Projected to Reach $213M Annual Revenue by 2037 for ADPKD Treatment

  • Regulus Therapeutics' microRNA therapeutic RGLS-8429 for autosomal dominant polycystic kidney disease is forecasted to generate $213 million in annual US revenue by 2037.
  • The company, focused on developing microRNA-based therapeutics for orphan kidney diseases, reported an operating loss of $31.1 million in FY2023, up from $28.2 million in FY2022.
  • Regulus leverages its proprietary microRNA development platform to advance therapies targeting various conditions, including cancer, fibrosis, and inflammatory disorders.

JCR Pharmaceuticals' Laronidase for Hurler Syndrome Expected to Generate $38M by 2040

  • JCR Pharmaceuticals is developing JR-171, a novel blood-brain barrier penetrating enzyme replacement therapy for Hurler syndrome, utilizing their proprietary J-Brain Cargo technology.
  • Financial projections indicate Laronidase could reach annual global revenues of $38 million by 2040, demonstrating significant market potential for this rare disease treatment.
  • The company has shown strong financial performance with a 24.8% revenue increase in FY2024 and improved operating margins from 14.4% to 17.6%.

iTeos Therapeutics' TIGIT Inhibitor Belrestotug Projects $366M Peak Revenue for Solid Tumors by 2038

• iTeos Therapeutics' Belrestotug (EOS-448), a TIGIT-targeting checkpoint inhibitor, is forecasted to reach $366 million in annual US revenue by 2038 for multiple solid tumor indications.
• The intravenous immunotherapy is being developed for various cancers including non-small cell lung cancer, PD-1 resistant melanoma, and head and neck squamous cell carcinoma.
• iTeos faces financial headwinds with a 95.3% revenue decrease in FY2023 and a net loss of $112.6 million, highlighting the critical importance of Belrestotug's clinical development success.

Insmed's Treprostinil Palmitil Shows Promise for Rare Pulmonary Disorders with Projected $66M Revenue by 2037

  • Insmed's innovative dry powder inhalation therapy, Treprostinil Palmitil, targets multiple rare pulmonary disorders including PAH and PH-ILD through a novel sustained-release nanoparticle formulation.
  • The drug candidate, targeting the prostacyclin receptor, is projected to generate annual revenues of $66 million by 2037 in the US market according to GlobalData's analysis.
  • Insmed demonstrates strong market presence with 24.4% revenue growth in FY2023, reaching $305.2 million, while continuing development of their rare disease therapeutic pipeline.

Altimmune's Pemvidutide Projected to Reach $759M in Global Sales by 2039

  • Pemvidutide, Altimmune's novel peptide-based therapeutic, is being developed for multiple indications including obesity, MASH, and type 2 diabetes, targeting both GLP-1 and glucagon receptors.
  • Financial projections indicate Pemvidutide could achieve annual global sales of $759 million by 2039, highlighting significant market potential for this subcutaneously administered drug.
  • Altimmune reported increased operating losses of $95.9 million in FY2023, up from $87.7 million in FY2022, as the company continues investment in Pemvidutide's development program.

Dupilumab Shows Sustained Efficacy in Chronic Rhinosinusitis with Nasal Polyps Over Three Years

• A real-world study demonstrates dupilumab's long-term effectiveness in treating chronic rhinosinusitis with nasal polyps (CRSwNP) over three years. • Patients experienced significant and sustained improvements in symptoms, disease-related impairment, nasal polyp burden, and olfactory function. • The majority of patients achieved an 'excellent' treatment response based on EUFOREA23 criteria, highlighting the clinical benefit of dupilumab. • The study confirms the enduring therapeutic effects of dupilumab, with most adverse events being self-limiting, supporting its use in CRSwNP management.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.