Ravidasvir

Taiwan Approves Chugai's PiaSky as First Subcutaneous Treatment for Paroxysmal Nocturnal Hemoglobinuria

3 days ago

• Taiwan FDA has granted orphan drug approval for PiaSky, making it the first subcutaneous treatment for paroxysmal nocturnal hemoglobinuria (PNH) available in Taiwan for patients 13 years and older. • PiaSky, developed with Chugai's proprietary Recycling Antibody technology, allows for convenient subcutaneous administration every 4 weeks, significantly reducing treatment burden compared to existing biweekly intravenous options. • The approval was based on positive results from three Phase III clinical trials, including COMMODORE 2, which demonstrated efficacy in transfusion avoidance and control of hemolysis compared to eculizumab.

Novartis' FABHALTA Poised to Transform Treatment Landscape for Multiple Complement-Mediated Diseases

23 days ago

• FABHALTA (iptacopan), Novartis' first-in-class oral factor B inhibitor, has received FDA approvals for paroxysmal nocturnal hemoglobinuria (PNH), IgA nephropathy (IgAN), and C3 glomerulopathy (C3G), marking a significant shift from traditional intravenous therapies. • The drug's oral administration route and ability to control both intravascular and extravascular hemolysis addresses key limitations of current therapies, potentially expanding the market for complement-targeted treatments estimated at over $1.2 billion in 2024. • With patent protection extending to 2041 and ongoing investigations for additional indications including myasthenia gravis and atypical hemolytic uremic syndrome, FABHALTA is expected to significantly reshape the complement inhibitor market landscape.

FDA Approves Pre-Filled Syringe for Self-Injection of Efgartigimod in gMG and CIDP Patients

a month ago

• The FDA has approved a new pre-filled syringe formulation of efgartigimod (VYVGART Hytrulo) for self-injection in adults with generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy. • The self-injection option provides patients greater independence and flexibility, allowing treatment at home or while traveling, reducing the need for frequent clinic visits while maintaining the medication's established safety and efficacy profile. • Developed through argenx's partnership with Halozyme's ENHANZE drug delivery technology, the pre-filled syringe enables rapid 20-30 second subcutaneous administration, with regulatory decisions expected in the EU, Japan, and Canada by 2025.

Omeros Advances Zaltenibart Phase 3 Trials for PNH with Potential Superiority Over Current Treatments

2 months ago

• Omeros Corporation has initiated site activation for its Phase 3 clinical trials of zaltenibart (OMS906) in paroxysmal nocturnal hemoglobinuria (PNH), with data for regulatory submission expected in Q4 2026. • Zaltenibart offers a significant advantage over current PNH therapies with convenient once-every-eight-weeks dosing and ability to inhibit both intravascular and extravascular hemolysis, potentially achieving normal hemoglobin levels. • The Phase 3 program includes head-to-head comparisons against C5 inhibitors eculizumab and ravulizumab across 120 clinical sites in 30 countries, targeting a global PNH market projected to reach $11.7 billion by 2034.

Relay Therapeutics to Launch Phase 3 Trial for RLY-2608 in Advanced Breast Cancer Mid-2025

3 months ago

• Relay Therapeutics plans to initiate ReDiscover-2, a Phase 3 trial evaluating RLY-2608 plus fulvestrant in PI3Kα-mutated, HR+/HER2- advanced breast cancer patients, with enrollment target of 540 patients. • Interim Phase 1b data showed promising results with RLY-2608 plus fulvestrant achieving 11.4-month median progression-free survival in second-line treatment and 39% confirmed overall response rate. • The company maintains strong financial position with approximately $780 million in cash reserves as of Q4 2024, sufficient to fund operations into second half of 2027.

Gazyva Shows Significant Improvement in Lupus Nephritis Treatment: Phase III REGENCY Trial Results

4 months ago

• Genentech's Phase III REGENCY trial demonstrates that Gazyva plus standard therapy achieved 46.4% complete renal response in lupus nephritis patients compared to 33.1% with standard therapy alone. • The study, published in NEJM, showed clinically meaningful improvements in complement levels and reductions in anti-dsDNA markers, indicating reduced disease activity and inflammation. • Results were consistent across all patient subgroups, including those with Class IV lupus nephritis and higher baseline proteinuria levels, while maintaining Gazyva's established safety profile.

AstraZeneca's Oral Drug Danicopan Receives CHMP Backing for PNH-Related Anaemia Treatment

4 months ago

• The EMA's CHMP has recommended approval of AstraZeneca's danicopan as an add-on therapy for patients with paroxysmal nocturnal haemoglobinuria (PNH) who experience residual anaemia despite C5 inhibitor treatment. • Clinical trial data from the ALPHA study demonstrated significant improvements in haemoglobin and reticulocyte count levels when danicopan was combined with existing C5 inhibitors Soliris or Ultomiris. • If approved, danicopan will offer the first oral alternative to current injectable treatments for PNH patients with residual anaemia, affecting approximately 10-20% of those receiving C5 inhibitor therapy.

Eculizumab Shows Promise in Treating Thymoma-Associated Myasthenia Gravis

4 months ago

• A real-world study demonstrates that eculizumab significantly reduces Myasthenia Gravis Activities of Daily Living (MG-ADL) scores in patients with thymoma-associated myasthenia gravis. • The research indicates a substantial decrease in the need for corticosteroids among patients treated with eculizumab by week 12. • Clinically meaningful improvements were observed in 81.8% of patients, with initial benefits appearing within an average of 1.7 weeks after starting eculizumab. • The study suggests eculizumab is a safe and effective treatment option for this severe myasthenia gravis subtype, warranting further investigation through larger randomized controlled trials.

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.

Aviceda Therapeutics Secures $207.5 Million to Advance Geographic Atrophy Treatment

4 months ago

• Aviceda Therapeutics has raised $207.5 million in a Series C financing round to advance its AVD-104 program for geographic atrophy (GA). • The funding will support the ongoing Phase 2b/3 trial and planned pivotal Phase 3 clinical trial of AVD-104, a novel immunomodulator. • AVD-104 targets both immune modulation and complement inhibition, showing promise in slowing GA lesion growth and preserving visual function. • The Phase 2b/3 trial data, comparing AVD-104 to avacincaptad pegol (Izervay), is expected in the second half of 2025.

Gene Therapy Trial for Geographic Atrophy Shows Limited Efficacy Despite Sustained Factor I Expression

5 months ago

A Phase I/II clinical trial of PPY988, an AAV2-CFI gene therapy for geographic atrophy, demonstrated sustained elevation of complement Factor I levels but insufficient potency compared to existing treatments. The study provides crucial insights for future complement-targeting gene therapies while confirming complement modulation as a viable therapeutic approach for age-related macular degeneration.

AbbVie Acquires Nimble Therapeutics for $200M, Expanding Oral Immunology Pipeline

5 months ago

• AbbVie announces $200 million acquisition of Nimble Therapeutics, gaining access to novel oral peptide drug platform and pipeline targeting immune diseases. • Nimble's lead candidate is an oral IL-23 inhibitor in preclinical development, potentially offering a non-injectable alternative to existing treatments like Skyrizi for psoriasis and IBD. • The acquisition marks AbbVie's third immunology-focused buyout this year, strengthening their position in the competitive autoimmune disease market.

Emerging Therapies Show Promise for Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

5 months ago

• IC-MPGN affects approximately 7,100 individuals across the 7MM, with the US accounting for 55% of cases, highlighting a significant unmet need for targeted treatments. • The IC-MPGN market in the US, valued at approximately $12 million in 2023, is projected to grow by 2034 due to increased diagnosis and the introduction of premium-priced emerging therapies. • Late-stage drugs like Iptacopan (Novartis) and Pegcetacoplan (Apellis) are under development, offering potential as targeted complement inhibitors for this rare kidney disease. • Current IC-MPGN treatment relies on off-label drugs such as immunosuppressants and steroids, underscoring the need for approved therapies addressing the root cause of the disease.

Kira Pharmaceuticals' KP104 Shows Sustained Efficacy and Safety in PNH Phase 2 Trial

5 months ago

• KP104, a dual-targeting complement inhibitor, demonstrates long-term safety and efficacy in complement inhibitor-naïve PNH patients. • The Phase 2 study shows sustained improvements in hemoglobin levels, with 100% of patients achieving a ≥2 g/dL increase from baseline. • 94% of patients achieved near-normal LDH levels, indicating strong intravascular hemolysis suppression, and remained transfusion-free. • Global Phase 3 studies are planned to further establish KP104 as a potential new standard of care for PNH.

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.

Rallybio Initiates Phase II Trial of RLYB212 for FNAIT Prevention in Europe

6 months ago

• Rallybio has dosed the first participant in a Phase II trial of RLYB212, a potential treatment for fetal and neonatal alloimmune thrombocytopenia (FNAIT). • The open-label, single-arm trial will assess the pharmacokinetics and safety of RLYB212 in pregnant women at high risk for HPA-1a alloimmunization and FNAIT. • Initial data from the second trimester are expected in Q2 2024, with further PK and safety outcomes anticipated in Q3 2024. • RLYB212 is a monoclonal antibody designed to prevent maternal alloimmunization by eliminating HPA-1a positive fetal platelets from the mother's circulation.

Rallybio Advances RLYB212 Phase 2 Trial for FNAIT Prevention in Pregnant Women

6 months ago

• Rallybio has initiated a Phase 2 clinical trial for RLYB212, a potential treatment for Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT). • The trial focuses on pregnant women at higher risk of HPA-1a alloimmunization, aiming to confirm the dosing regimen of RLYB212. • Preliminary pharmacokinetic and safety data from the sentinel participant are expected in Q2 2025, with delivery data in Q3 2025. • RLYB212 is a subcutaneously administered monoclonal antibody designed to prevent maternal alloimmunization and subsequent FNAIT.

Rallybio's RLYB212 Phase 2 Trial for FNAIT Prevention Set to Initiate Screening

6 months ago

• Rallybio has received CTA approvals for its RLYB212 Phase 2 clinical trial, with screening of pregnant women at higher risk for FNAIT expected to begin in Q4 2024. • Preclinical data presented at ASH demonstrated that prophylactic administration of RLYB212 safely and effectively prevents FNAIT in pregnant mice. • Rallybio anticipates providing updates on manufacturing process enhancements and biomarker characterization, along with future plans for RLYB116, in December 2024. • The company's cash, cash equivalents, and marketable securities totaled $75.1 million as of September 30, 2024, providing runway into mid-2026.

AstraZeneca Highlights Hematology Portfolio at ASH 2024 with Calquence and Novel Therapies

6 months ago

• AstraZeneca will present AMPLIFY Phase III trial results, reinforcing CALQUENCE® as a leading BTK inhibitor in frontline chronic lymphocytic leukemia with a fixed-duration regimen. • New data on next-generation cell therapy AZD0120 and T-cell engager AZD0486 will demonstrate promising early results in multiple types of blood cancer. • Long-term data for VOYDEYA™ as add-on to ULTOMIRIS® or SOLIRIS® will show low rates of breakthrough hemolysis and sustained quality-of-life improvements in PNH patients with extravascular hemolysis.

Complement Inhibitors Show Promise in Diverse Clinical Trials

7 months ago

• The complement inhibitors market is experiencing growth due to increased understanding of the complement system's role in rare and chronic diseases. • Over 40 companies are actively developing more than 50 complement inhibitor therapies, targeting conditions like PNH, aHUS and Guillain-Barré syndrome. • Clinical trials are underway for novel inhibitors like Gefurulimab, ANX005 and DNTH103, showing potential in myasthenia gravis and multifocal motor neuropathy. • Recent FDA clearances and orphan drug designations highlight the increasing regulatory support for complement-targeted therapies, fostering innovation.

Drug Development Updates