ALEXION PHARMACEUTICALS, INC.

Lupus Nephritis Market Expected to Grow as Novel Therapies Enter Clinical Trials

14 days ago

• The global lupus nephritis market is projected to grow significantly by 2034, with the United States accounting for over 80% of the current $1.12 billion market share across major regions. • Several innovative therapies are advancing through clinical trials, including CAR-T cell treatments from companies like Allogene Therapeutics, Adicet Bio, and Caribou Biosciences, which received FDA Fast Track designations. • Key pharmaceutical players including Aurinia Pharmaceuticals, AstraZeneca, and Roche are developing treatments such as voclosporin, anifrolumab, and obinutuzumab to address the significant unmet needs in lupus nephritis management.

CDMO Market Report: Key Regulatory Approvals and Clinical Advances in March-April 2025

a month ago

• Multiple CDMOs secured significant contract manufacturing opportunities as regulatory bodies approved new indications for established drugs, particularly in oncology and rare diseases. • AstraZeneca's portfolio saw substantial growth with expanded approvals for Imfinzi, Tagrisso, and Lynparza, strengthening partnerships with contract manufacturers including Lonza, Dottikon, and Samsung Biologics. • Contract manufacturers supporting treatments for autoimmune conditions showed strong performance, with Argenx's Vyvgart Hytrulo receiving expanded indications for myasthenia gravis and CIDP.

New Report Reveals Critical Insights into Clinical-Stage Pharma Partnerships from 2020-2025

2 months ago

• A comprehensive new industry report analyzes over 1,670 clinical-stage partnering agreements in pharma and biotech from 2020-2025, providing unprecedented access to deal structures and financial terms. • The report reveals detailed intelligence on how licensing agreements typically grant exclusive rights across Phase I-III trials, with multi-component structures involving collaborative R&D and commercialization strategies. • Business development professionals can now access actual contract documents and payment triggers often missing from press releases, enabling more effective negotiation strategies and competitive deal structuring.

Rallybio Advances FNAIT Candidate RLYB212 to Phase 2 Trial, Eyes Expanded Market

4 months ago

• Rallybio is set to begin a Phase 2 trial for RLYB212 in Q2 2025, targeting Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) prevention, with interim data expected in Q3. • Recent epidemiological analysis has expanded the estimated market for FNAIT treatment to over 30,000 at-risk pregnancies annually in the U.S. and EU. • The company is also progressing its C5 inhibitor RLYB116 and ENPP1 inhibitor REV102, with studies planned to broaden its therapeutic pipeline. • Rallybio's current cash reserves of $89 million are projected to sustain operations until mid-2026, supporting the advancement of its clinical programs.

Teva and Samsung Bioepis Launch Epysqli, a 30% Discounted Biosimilar to Soliris for Rare Diseases

4 months ago

• Teva and Samsung Bioepis have launched Epysqli (eculizumab-aagh), a biosimilar to Alexion's Soliris, offered at a 30% discount off the reference product's wholesale acquisition cost. • The biosimilar is approved for three rare diseases: paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor antibody positive. • This strategic partnership aligns with Teva's Pivot to Growth strategy, expanding its biosimilar portfolio to 18 assets while addressing the critical need for more affordable treatment options for underserved patient communities.

Tenpoint Therapeutics' BRIMOCHOL™ PF Shows Positive Phase 3 Results for Presbyopia

4 months ago

• Tenpoint Therapeutics' BRIMOCHOL™ PF met primary endpoints in the BRIO-II Phase 3 trial, demonstrating statistically significant near vision improvement over 8 hours. • The study showed BRIMOCHOL™ PF was well-tolerated with no serious adverse events during the 12-month at-home dosing period. • BRIMOCHOL™ PF also demonstrated statistically significant improvement in ocular hyperemia compared to carbachol alone and reduced pupil size. • Tenpoint plans to file a new drug application with the FDA in the first half of 2025, potentially launching in 2026.

Nipocalimab Receives FDA Priority Review for Generalized Myasthenia Gravis Treatment

4 months ago

• The FDA granted Priority Review to nipocalimab for treating gMG in antibody-positive patients, expediting its potential availability to patients. • Phase 3 Vivacity-MG3 study results supported the application, demonstrating sustained disease control and significant MG-ADL score reduction. • Nipocalimab, a monoclonal antibody, aims to reduce IgG autoantibodies, addressing the underlying cause of gMG without broad immunosuppression. • Johnson & Johnson also submitted a Marketing Authorisation Application to the EMA, seeking approval of nipocalimab in gMG in Europe.

Regeneron's Veopoz and Ordspono Show Promise in PNH and Lymphoma

5 months ago

• Regeneron's Veopoz, combined with an siRNA drug from Alnylam, demonstrated superior disease control compared to Ultomiris in paroxysmal nocturnal hemoglobinuria (PNH). • In follicular lymphoma, Regeneron's bispecific antibody Ordspono achieved a 100% complete response rate in previously untreated patients in a Phase 3 study. • Regeneron is also developing other drugs for PNH, with the potential to disrupt the market with improved efficacy and convenient administration. • Ordspono's efficacy in late-line follicular lymphoma showed an 80% objective response rate, with 73% achieving complete remission, suggesting best-in-class potential.

Regeneron's Pozelimab and Cemdisiran Combo Shows Superior Hemolysis Control in PNH Patients

5 months ago

• Regeneron's pozelimab and cemdisiran combination (poze-cemdi) demonstrated superior disease control in paroxysmal nocturnal hemoglobinuria (PNH) patients compared to ravulizumab in a Phase 3 trial. • The trial showed 96% of patients achieved adequate LDH control with poze-cemdi, compared to 80% with ravulizumab, indicating better management of intravascular hemolysis. • Patients on poze-cemdi also experienced greater LDH normalization (93% vs. 65%) and a more significant decrease in LDH levels from baseline compared to those on ravulizumab. • The combination offers a convenient four-week subcutaneous delivery, potentially improving patient adherence and quality of life compared to intravenous treatments.

Emerging Therapies Offer Hope for Geographic Atrophy Treatment

6 months ago

• Several therapies are under evaluation in clinical trials for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), addressing the unmet need for effective treatments. • Complement pathway inhibitors like ANX007, danicopan, and AVD-104 are being investigated to modulate the inflammatory response and cell lysis associated with GA progression. • Gene therapies such as JNJ-1887 and cell-based therapies like ASP7317 and RG6501 are showing promise in protecting retinal cells and improving visual function in GA patients. • Antioxidants like elamipretide, glideuretinal, and AREDS2 supplements are also being explored for their potential to reduce GA lesion progression and improve visual outcomes.

Iptacopan Shows Promise in Reducing Proteinuria in IgA Nephropathy

7 months ago

• Phase III APPLAUSE-IgAN study demonstrates iptacopan significantly reduces proteinuria in patients with IgA nephropathy compared to placebo. • Iptacopan, an alternative pathway inhibitor, led to a 38.3% reduction in 24-hour urinary protein-to-creatinine ratio (UPCR) at 9 months. • The study also revealed significant changes in complement pathway biomarkers, indicating selective alternative pathway inhibition. • While showing promise, further data on eGFR is needed to confirm iptacopan's impact on slowing kidney function decline.

FDA Grants Full Approval to Travere's Filspari for IgA Nephropathy

9 months ago

• The FDA granted full approval to Travere Therapeutics' Filspari (sparsentan) for IgA nephropathy, allowing broader use for patients at risk of disease progression. • The approval was based on the PROTECT study, which demonstrated Filspari significantly slowed kidney function decline over two years compared to irbesartan. • Filspari is now positioned as a foundational, non-immunosuppressive treatment option, potentially replacing the current standard of care for IgAN patients. • The label update removes a specific urine protein level requirement, expanding the eligible patient population and increasing Filspari's market potential.