X4 Pharmaceuticals, Inc.

FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months

3 months ago

• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration. • Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients. • Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.

Bain Capital Acquires Mitsubishi Tanabe Pharma in $3.3 Billion Deal

4 months ago

• Bain Capital has agreed to acquire Mitsubishi Tanabe Pharma, a 350-year-old pharmaceutical company, in a deal valued at $3.3 billion, aiming to capitalize on growth opportunities in Japan's life sciences sector. • The acquisition will transform Mitsubishi Tanabe into a standalone company, focusing on vaccine development and treatments for neurological, cardiometabolic, and immunological conditions. • The Osaka-based company, which employs over 5,000 people globally, will maintain its operations while benefiting from Japan's initiatives to accelerate drug development.

EMA Validates Marketing Authorization Application for Mavorixafor in WHIM Syndrome Treatment

4 months ago

X4 Pharmaceuticals has achieved a significant milestone as the European Medicines Agency (EMA) validates their Marketing Authorization Application for mavorixafor in treating WHIM syndrome. This development follows the drug's recent U.S. approval under the brand name XOLREMDI for patients 12 years and older with this rare immunodeficiency disorder.

Mavorixafor Advances in Europe for WHIM Syndrome Treatment Following Positive Phase 3 Results

4 months ago

• X4 Pharmaceuticals' Marketing Authorization Application (MAA) for mavorixafor to treat WHIM syndrome is under EMA review, potentially the first EU-approved drug for the condition. • Mavorixafor's MAA is supported by Phase 3 trial results, showing reduced infection rates and severity in WHIM syndrome patients with a good safety profile. • Norgine and X4 Pharmaceuticals have an exclusive licensing agreement to commercialize mavorixafor in Europe, Australia, and New Zealand upon regulatory approval. • Mavorixafor, already approved in the U.S. as XOLREMDI, is a CXCR4 antagonist, aiming to increase circulating neutrophils and lymphocytes in WHIM syndrome patients.

Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments

4 months ago

• Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions. • Several companies, including Intellia Therapeutics and IGM Biosciences, are cutting a significant portion of their workforce to focus on key programs and extend cash runways. • These workforce reductions are often accompanied by discontinuation of certain drug development programs and a shift in strategic focus towards core assets. • The layoffs reflect a broader trend in the industry as companies adapt to evolving market dynamics and prioritize efficiency and long-term sustainability.

FDA Approvals in 2024: Novel Therapies for MASH, WHIM Syndrome, Pediatric Glioma, MDS, and Bladder Cancer

5 months ago

• The FDA approved Madrigal's Rezdiffra, the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), addressing a significant unmet need in liver disease. • X4 Pharmaceuticals' Xolremdi gained approval as the first targeted therapy for WHIM syndrome, a rare immunodeficiency, marking a milestone for the company. • Day One Biopharmaceuticals' Ojemda secured approval for BRAF-altered pediatric low-grade glioma, offering a new treatment option for this common childhood brain tumor. • Geron Corporation's Rytelo, a telomerase inhibitor, received FDA approval for lower- to intermediate-risk myelodysplastic syndromes (MDS), after 34 years in business. • ImmunityBio's Anktiva, a novel IL-15 superagonist, was approved for non-muscle invasive bladder cancer, providing a new therapeutic approach for BCG-unresponsive patients.

X4 Pharmaceuticals' Mavorixafor Shows Promise in Chronic Neutropenia Phase 2 Trial

6 months ago

• X4 Pharmaceuticals announced positive Phase 2 results for mavorixafor in chronic neutropenia (CN), demonstrating increased neutrophil counts. • The Phase 3 4WARD trial of mavorixafor in CN is progressing, with full enrollment expected by mid-2025. • XOLREMDI® (mavorixafor) U.S. launch for WHIM syndrome is underway, with EMA submission anticipated in early 2025. • Market research indicates high awareness and increased screening for WHIM syndrome, supporting XOLREMDI adoption.

X4 Pharmaceuticals Announces Positive Phase 2 Results for Mavorixafor in Chronic Neutropenia

6 months ago

• X4 Pharmaceuticals reported positive Phase 2 clinical data for mavorixafor in chronic neutropenia (CN), showing increased neutrophil counts. • The Phase 2 study demonstrated that once-daily oral mavorixafor was well-tolerated and effectively elevated participants' neutrophil counts. • X4's pivotal Phase 3 trial of mavorixafor in CN is progressing as planned, with full enrollment expected by mid-2025. • The U.S. launch of XOLREMDI® (mavorixafor) for WHIM syndrome is underway, and EMA submission is anticipated by early 2025.

Acadia Pharmaceuticals Secures $150M in Priority Review Voucher Sale, Setting High Benchmark

7 months ago

• Acadia Pharmaceuticals has successfully sold its FDA priority review voucher for $150 million, marking one of the highest valuations for such vouchers in recent transactions. • The voucher, earned through Daybue's approval for Rett syndrome treatment, will net Acadia $100 million after sharing proceeds with development partner Neuren Pharmaceuticals. • The transaction reflects increasing voucher values amid FDA's planned program sunset, with proceeds earmarked for commercial operations, business development, and rare disease research.

FDA Approves XOLREMDI (mavorixafor) as First Targeted Therapy for WHIM Syndrome

a year ago

• The FDA has approved XOLREMDI (mavorixafor) for patients 12 years and older with WHIM syndrome, marking the first targeted therapy for this rare immunodeficiency. • XOLREMDI, a CXCR4 antagonist, demonstrated significant improvements in absolute neutrophil and lymphocyte counts in the pivotal 4WHIM Phase 3 clinical trial. • The approval was based on data showing increased time above threshold for neutrophil and lymphocyte counts, along with a reduction in infection rates compared to placebo. • X4 Pharmaceuticals has launched X4Connect to support patients in accessing XOLREMDI, which will be available through PANTHERx Rare specialty pharmacy.