Acquired Hemophilia A Pipeline Expands with 10+ Novel Therapies in Development
• Global Acquired Hemophilia A pipeline comprises over 10 companies developing innovative therapies across various clinical stages, according to DelveInsight's 2025 report.
• Recent clinical advances include promising results for rituximab as a potential first-line therapy and Pfizer's positive Phase 3 AFFINE trial results for giroctocogene fitelparvovec gene therapy.
• Key pharmaceutical players including Novo Nordisk, Sanofi, Pfizer, and Belief Biomed are advancing treatments ranging from gene therapies to monoclonal antibodies targeting this rare autoimmune bleeding disorder.
Rivus Pharmaceuticals Strengthens Leadership Team Ahead of Critical Phase 2b MASH Trial Results
• Rivus Pharmaceuticals has appointed David Grainger, Ph.D., as Chairman of Development and Meg Fitzgerald, J.D., as Chief Legal Officer while establishing a new office in South San Francisco.
• The company is advancing its novel Controlled Metabolic Accelerators (CMAs), led by HU6, which has shown promising Phase 2a results in treating obesity-related conditions without muscle mass loss.
• Rivus is approaching a critical milestone with the imminent readout of its Phase 2b M-ACCEL study in metabolic dysfunction-associated steatohepatitis (MASH), potentially advancing HU6 to pivotal trials.
EPCORE NHL-2 Trial Shows Promise Combining Chemotherapy and Immunotherapy for DLBCL
• A phase Ib/II clinical trial led by Mount Sinai researchers demonstrates successful outcomes in combining chemotherapy with immunotherapy for diffuse large B-cell lymphoma treatment.
• The groundbreaking research, published in Blood journal, was conducted under the leadership of Dr. Joshua Brody, director of the Lymphoma Immunotherapy Program at the Tisch Cancer Institute.
• The study represents a significant advancement in lymphoma treatment, supported by the Leukemia & Lymphoma Society's research initiatives, which have invested over $600 million in hematologic oncology research.
Centessa Pharmaceuticals Appoints Stephen Kanes as Chief Medical Officer to Advance Orexin Agonist Program
• Centessa Pharmaceuticals appointed Stephen Kanes as Chief Medical Officer, leveraging his 30+ years of experience in neuroscience and drug development.
• Dr. Kanes will focus on advancing Centessa's orexin receptor 2 (OX2R) agonist program, including ORX750, currently in Phase 2 trials for sleep-wake disorders.
• Centessa's ORX750 has shown promise in Phase 1 trials, restoring wakefulness in sleep-deprived individuals, with Phase 2a studies underway for narcolepsy and idiopathic hypersomnia.
• Kanes previously led the development of ZULRESSO® for postpartum depression at Sage Therapeutics, bringing valuable expertise to Centessa's neurology and psychiatry pipeline.
Hemophilia B Therapeutics: Advances in Clinical Trials and Emerging Therapies
• Several companies are actively involved in developing therapies for Hemophilia B, with Belief Biomed's drug candidates reaching Phase III clinical trials.
• Recent trials include Novo Nordisk's assessment of Nonacog Beta Pegol in Chinese patients and ApcinteX Ltd's study of SerpinPC in severe Hemophilia A or B.
• Emerging therapies like BBM-H901 (Belief Biomed) and Fidanacogene elaparvovec (Pfizer/Spark Therapeutics) are under investigation across various clinical stages.
• Hemophilia B therapies are being developed using various routes of administration, including oral, intravenous, and subcutaneous, and molecule types, such as gene therapy and recombinant fusion proteins.
Amgen's Obesity Drug MariTide Faces Bone Density Concerns; AstraZeneca Invests $3.5B in US Expansion
• Amgen's MariTide, an obesity drug, faced scrutiny after an analyst suggested a potential link to bone density loss based on Phase 1 trial data.
• Lexeo Therapeutics reached an agreement with the FDA for its gene therapy LX2006 targeting Friedreich ataxia with cardiomyopathy, potentially leading to accelerated approval.
• AstraZeneca is set to invest $3.5 billion in expanding its US research and manufacturing facilities, creating over 1,000 jobs.
• Roche is collaborating with Flare Therapeutics in a deal worth up to $1.8 billion to develop small molecule drugs targeting transcription factors in oncology.
Centessa Pharmaceuticals Initiates Phase 2 Trial of ORX750 for Sleep-Wake Disorders
• Centessa Pharmaceuticals has commenced a Phase 2 clinical trial of ORX750 for narcolepsy type 1 (NT1), narcolepsy type 2 (NT2), and idiopathic hypersomnia.
• ORX750, an orally administered orexin receptor 2 (OX2R) agonist, was designed using Nxera Pharma's technology to target orexin neuron loss.
• The trial initiation triggers a US$3.5 million milestone payment to Nxera Pharma, to be recognized as revenue in Q4 FY2024.
• Nxera Pharma holds a significant equity stake in Centessa and is eligible for further milestone payments and royalties upon successful development of ORX750.
AnaptysBio Reports Financial Growth and Clinical Trial Progress in Q3 2024
• AnaptysBio's collaboration revenue significantly increased to $30.0 million in Q3 2024, compared to $3.3 million in the same period of 2023, driven by strategic partnerships.
• The company's clinical pipeline advances with rosnilimab in Phase 2b trials for rheumatoid arthritis and ulcerative colitis, and ANB032 in Phase 2b for atopic dermatitis.
• AnaptysBio anticipates key data readouts, including top-line results from the rosnilimab rheumatoid arthritis trial in February 2025 and ANB032 atopic dermatitis trial in December 2024.
• A new BDCA2 modulator antibody, ANB101, is set to begin Phase 1 clinical trials in Q1 2025, expanding AnaptysBio's focus on autoimmune and inflammatory diseases.
Hemophilia A and B Pipeline Advances with Novel Therapies in Clinical Trials
• Several companies are actively developing novel therapies for Hemophilia A and B, with many in various phases of clinical trials, showing promise for improving treatment options.
• TiumBio Co., Ltd. has submitted a Clinical Trial Application to initiate a Phase 1b investigation of TU7710, a new recombinant activated factor VII for hemophilia patients with inhibitors.
• The FDA approved ALTUVIIIO (efanesoctocog alfa) in February 2023, a factor VIII replacement therapy for adults and children with Hemophilia A, for routine prophylaxis and on-demand treatment.
• BioMarin received FDA approval for Roctavian in June 2023, a single-dose gene therapy for severe Hemophilia A, marking a significant advancement in treatment.
Centessa's ORX750 Advances to Phase 2 for Narcolepsy and Idiopathic Hypersomnia After Promising Phase 1 Results
• Centessa Pharmaceuticals' ORX750, an orexin receptor 2 (OX2R) agonist, demonstrated significant improvements in wakefulness in sleep-deprived healthy volunteers.
• The 2.5 mg dose of ORX750 restored normal wakefulness, achieving a mean sleep latency of 32 minutes compared to 17 minutes with placebo.
• ORX750 exhibited a favorable safety profile, with mild and transient adverse events and no significant on-target effects observed.
• Based on these positive Phase 1 results, Centessa plans to initiate Phase 2 trials in patients with narcolepsy types 1 and 2, and idiopathic hypersomnia in late 2024.
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