Y-mAbs Therapeutics

US Orphan Drug Market Set to Exceed $190 Billion by 2030 as FDA Designations Accelerate

9 days ago

• The US orphan drug market is projected to surpass $190 billion by 2030, with over 7,300 molecules receiving FDA Orphan Drug Designation to date, of which approximately 17.9% have gained approval. • Since 2020, more than half of all new drug approvals by the FDA's Center for Drug Evaluation and Research have been granted orphan status, highlighting the growing importance of rare disease treatments in pharmaceutical development. • Despite criticism over high pricing, exemplified by Abeona Therapeutics' Zevaskyn at $3.1 million per treatment, the orphan drug model has evolved into a sound business strategy offering fewer competitors, faster approvals, and seven-year market exclusivity.

Y-mAbs Initiates Phase 1 Trial of CD38-SADA Radioimmunotherapy for Relapsed Non-Hodgkin Lymphoma

a month ago

• Y-mAbs Therapeutics has dosed the first patient in its Phase 1 trial of CD38-SADA PRIT platform for relapsed/refractory non-Hodgkin Lymphoma, marking its first clinical program in hematological malignancies. • The innovative two-step approach delivers CD38-SADA protein that binds to lymphoma cells, followed by a radioactive 177Lu-DOTA payload that targets tumor-bound molecules while minimizing radiation exposure to healthy tissues. • This trial (1201) is designed as a dose-escalation, open-label study to determine optimal protein dosing and administration intervals, potentially offering new treatment options for NHL patients with limited alternatives.

B-Cell Lymphoma Pipeline Expands with 300+ Therapies in Development for 2025

2 months ago

• DelveInsight's latest report reveals a robust B-cell lymphoma pipeline with over 295 companies developing 300+ therapies, highlighting significant industry investment in this area. • Several major pharmaceutical companies including BeiGene, Celgene, Hoffmann-La Roche, and Allogene Therapeutics have initiated pivotal late-stage clinical trials for novel B-cell lymphoma treatments in March 2025. • Emerging therapies include CAR-T cell approaches, bispecific antibodies, and novel targeted agents, with many incorporating dual-targeting mechanisms to overcome resistance seen with single-target therapies.

Robust Pipeline for Ewing Sarcoma Treatment: 25+ Companies Advancing Novel Therapies

3 months ago

• A comprehensive pipeline analysis reveals over 25 pharmaceutical companies actively developing treatments for Ewing Sarcoma, with multiple promising candidates in various clinical stages. • Gradalis's Vigil, a personalized cancer immunotherapy, has advanced to Phase III trials in combination with irinotecan and temozolomide for Ewing Sarcoma treatment. • Key emerging therapies include Oncternal's ONCT216, a first-in-class ETS-family transcription factor inhibitor, and Salarius's SP-2577, currently in Phase I/II trials for multiple sarcoma types.

Naxitamab Shows Promising Results in Phase 2 Trial for High-Risk Neuroblastoma

3 months ago

• Phase 2 trial of naxitamab with GM-CSF achieved 50% overall response rate in patients with relapsed/refractory high-risk neuroblastoma, meeting its primary endpoint. • The treatment demonstrated strong efficacy with 58% bone compartment response and 74% bone marrow compartment response, showing particular promise for patients with bone involvement. • Notable one-year survival outcomes include 93% overall survival rate, while some patients previously treated with anti-GD2 antibodies showed response to naxitamab therapy.

Atara Biotherapeutics Navigates Regulatory Hurdles and Financial Uncertainty

4 months ago

• Atara Biotherapeutics faces an FDA clinical hold on ATA3219 and EBVALLO due to manufacturing issues, delaying product sales and increasing perceived risk. • The company's need for additional financing to support its key programs is crucial, but uncertainties around resolving the clinical hold complicate matters. • Recent workforce reductions, affecting about half of its employees, reflect ongoing challenges following the FDA's Complete Response Letter for EBVALLO. • Atara is exploring strategic options, including potential mergers or acquisitions, to secure funding amidst regulatory and manufacturing challenges.

Y-mAbs Announces Strategic Realignment and Positive Preliminary Data for SADA PRIT Platform

4 months ago

• Y-mAbs is reorganizing into two business units focusing on radiopharmaceuticals and DANYELZA to enhance clinical development and commercial growth. • Preliminary Phase 1 data for GD2-SADA demonstrates tolerability and validity of the SADA PRIT pre-targeting approach in solid tumors. • The company anticipates presenting GD2-SADA Phase 1 data in Q2 2025 and dosing the first patient in the CD38-SADA Phase 1 trial in Q1 2025. • Y-mAbs reports estimated 2024 total net revenue of $88 million and anticipates cash to support operations into 2027.

Oppenheimer Predicts Significant Growth for Sagimet and Y-mAbs Therapeutics

5 months ago

Oppenheimer analysts forecast substantial growth for Sagimet Biosciences and Y-mAbs Therapeutics, citing their innovative treatments and market potential. Sagimet's denifanstat shows promise in treating MASH, acne, and glioblastoma, while Y-mAbs' SADA platform technology offers a novel approach to cancer treatment.