CHIESI FARMACEUTICI S.P.A.

Advancements in Bronchopulmonary Dysplasia Treatment: A Look into the 2024 Pipeline

4 months ago

The 2024 Bronchopulmonary Dysplasia (BPD) pipeline showcases significant progress with over 12 companies developing therapies. Key developments include Airway Therapeutics, Inc.'s Phase 3 trial for zelpultide alfa and Chiesi Farmaceutici SpA's collaboration with Oak Hill Bio on OHB-607. These efforts aim to address the unmet needs in BPD treatment, particularly for preterm infants.

Protalix BioTherapeutics Advances Pipeline and Receives EMA Validation for Fabry Disease Treatment

5 months ago

• Protalix BioTherapeutics announced the validation by the EMA of Chiesi's variation submission for pegunigalsidase alfa, aiming for less frequent dosing for Fabry disease. • A Phase I clinical trial of PRX-115, a recombinant PEGylated uricase for uncontrolled gout, showed promising safety and efficacy results with potential for wide dosing intervals. • Protalix is refining its R&D strategy, focusing on plant-based drug delivery systems for renal rare diseases, leveraging its ProCellEx platform. • The company reported satisfaction with sales growth of Elfabrio to Chiesi, Elelyso to Pfizer, and Uplyso to Brazil, and has fully repaid its outstanding convertible notes.

EMA Validates Less Frequent Dosing Regimen for Elfabrio in Fabry Disease

5 months ago

• The EMA has validated a variation submission for Elfabrio, proposing a less frequent dosing regimen for adult Fabry disease patients. • The new regimen suggests 2 mg/kg every four weeks, an alternative to the current 1 mg/kg bi-weekly dose. • Validation is supported by Phase 3 BRIGHT trial data, showing potential benefit for patients previously treated with other therapies. • This milestone aims to reduce treatment burden and improve the quality of life for Fabry disease patients.

XOMA Royalty Acquires Pulmokine for $20 Million, Expanding PAH Portfolio with Seralutinib

6 months ago

• XOMA Royalty Corporation acquired Pulmokine for $20 million, gaining rights to seralutinib, a Phase 3 asset for pulmonary arterial hypertension (PAH). • The acquisition adds seralutinib to XOMA's portfolio, which already includes six commercial royalty assets, enhancing its late-stage pipeline. • XOMA Royalty stands to gain up to $25 million in milestone payments and a low to mid-single digit royalty on seralutinib's commercial sales. • Phase 3 study results for seralutinib, co-developed by Gossamer Bio and Chiesi Farmaceutici, are expected in the fourth quarter of 2025.

Protalix BioTherapeutics Announces Positive Phase 1 Results for PRX-115 in Uncontrolled Gout

6 months ago

• Protalix BioTherapeutics reports completion of Phase 1 trial for PRX-115, a recombinant uricase candidate for uncontrolled gout, with encouraging preliminary results. • The Phase 1 study demonstrated dose-dependent increases in PRX-115 exposure and rapid reduction of plasma uric acid levels, with levels remaining below 6.0 mg/dL for up to 12 weeks at the highest doses. • PRX-115 was well-tolerated, with mostly mild to moderate adverse events; Protalix is actively planning a Phase 2 clinical trial in gout patients for the second half of 2025. • Protalix reported a net income of $3.2 million for the third quarter of 2024, driven by increased revenues from sales of goods, particularly to Chiesi Farmaceutici and Pfizer.

NCFB Pipeline Shows Promise with Over 15 Companies Developing New Therapies

6 months ago

• The non-cystic fibrosis bronchiectasis (NCFB) market is experiencing growth due to rising prevalence and increased awareness, leading to early diagnosis and treatment. • Over 15 companies are actively developing more than 15 NCFB drugs, aiming to improve the treatment landscape for this chronic lung condition. • Key players like Insmed, AstraZeneca, and Verona Pharma are advancing promising therapies such as brensocatib, benralizumab and ensifentrine through clinical trials. • Recent clinical trial milestones, including Phase III results for brensocatib and Phase II enrollment for AP-PA02, signal progress in addressing unmet needs in NCFB treatment.

Protalix Bio Presents Positive Phase 1 Data for PRX-115 in Uncontrolled Gout

7 months ago

• Protalix BioTherapeutics announced Phase 1 clinical trial data for PRX-115, a novel PEGylated uricase, showing promise in lowering urate levels. • The study evaluated PRX-115 in participants with elevated urate, demonstrating prolonged plasma urate-lowering effects after a single intravenous dose. • These findings were presented at the American College of Rheumatology (ACR) Convergence 2024, highlighting the potential of PRX-115 for uncontrolled gout. • Protalix plans to make the poster presentation available on their website, offering further insights into the study's results and methodology.

FDA Approves Lamzede for Alpha-Mannosidosis Treatment

2 years ago

The U.S. Food and Drug Administration (FDA) has approved Lamzede (velmanase alfa-tycv) for the treatment of non-central nervous system manifestations of alpha-mannosidosis in both adult and pediatric patients. This marks a significant advancement as Lamzede becomes the first and only enzyme replacement therapy available in the United States for this ultra-rare, progressive lysosomal storage disorder.