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Ruxoprubart Achieves Primary Endpoints in Phase 2 Trial for Paroxysmal Nocturnal Hemoglobinuria

  • NovelMed's ruxoprubart, a novel complement-targeting immunotherapy, met all primary efficacy endpoints in interim Phase 2 trial results for adults with paroxysmal nocturnal hemoglobinuria at 12 weeks.
  • The therapy demonstrated clinically meaningful benefits including transfusion avoidance, increased hemoglobin levels, reduced lactate dehydrogenase, and increased PNH clone size.
  • These positive results represent a significant advancement in PNH treatment, offering potential for improved patient outcomes in this rare hematologic disorder.
Rare Disease

Prothena Discontinues Birtamimab Development After Phase 3 Failure in AL Amyloidosis

  • Prothena's Phase 3 AFFIRM-AL trial of birtamimab for AL amyloidosis failed to meet its primary endpoint of time to all-cause mortality (HR=0.915, p-value=0.7680).
  • The company has announced complete discontinuation of birtamimab development, including stopping the open-label extension trial, following the disappointing clinical results.
  • Prothena plans substantial organizational restructuring with workforce reductions and will provide detailed plans to decrease operating expenses in June 2025.
Rare DiseaseMonoclonal Antibody

Chinese Base Editing Therapy Successfully Cures Thalassemia in Four Children

  • Four children with thalassemia have been successfully cured using CS-101, a novel DNA base editing therapy developed by CorrectSequence Therapeutics in China, marking a significant breakthrough in genetic treatment.
  • The therapy uses a transformer base editor (tBE) to precisely correct disease-causing DNA mutations, with patients showing complete recovery in as little as five weeks after a single injection.
  • Clinical trials led by Professor Zhai Xiaowen at Fudan University Children's Hospital have demonstrated promising results, with the treatment now advancing to larger-scale trials in China, the US, and UK.
Rare Disease

Ulefnersen Shows Unprecedented Recovery in Young Patients with Rare FUS-ALS

  • Columbia University researchers report that ulefnersen, an experimental drug, demonstrated remarkable efficacy in treating FUS-ALS, a rare genetic form of ALS affecting young people.
  • In a small case series of 12 patients, two showed exceptional responses, including one young woman who regained the ability to walk and breathe independently after treatment.
  • The therapy reduced neurofilament light, a biomarker of nerve damage, by up to 83% after six months, suggesting potential for not just slowing but reversing functional losses in early intervention.
Rare DiseasePrecision MedicineNeurology

Federal Circuit Upholds Pfizer's Victory in Gene Therapy Patent Dispute for Hemophilia Treatment

  • The Federal Circuit has affirmed the Patent Trial and Appeal Board's decision invalidating two patents covering experimental gene therapy for hemophilia treatment, handing Pfizer a significant legal victory.
  • The invalidated patents were related to novel gene therapy approaches for hemophilia, highlighting the competitive landscape in developing advanced treatments for this rare bleeding disorder.
  • This ruling represents an important precedent in the gene therapy intellectual property space, potentially impacting future development and commercialization of similar therapeutic approaches.
Rare Disease

Satellos Reports Promising Efficacy Signals for Novel DMD Treatment in Adult Patients

  • Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area.
  • The development comes amid setbacks in the DMD field, including Pfizer's withdrawal of its gene therapy fordadistrogene movaparvovec following a Phase III failure and a Phase II patient fatality.
  • The global DMD treatment market is projected to grow substantially from $2.3 billion in 2023 to $5.2 billion by 2033 across major markets, primarily driven by Elevidys and Santhera Pharmaceuticals' Agamree.
Rare Disease

Eli Lilly Enters $1.3 Billion RNA Therapy Partnership to Expand Genetic Hearing Loss Pipeline

  • Eli Lilly has formed a $1.3 billion partnership to develop RNA therapies targeting genetic hearing loss, significantly expanding their presence in this therapeutic area.
  • The collaboration aims to leverage RNA technology to address unmet needs in hearing disorders, potentially offering new treatment options for patients with genetic hearing impairments.
  • This strategic investment highlights the growing pharmaceutical interest in RNA-based approaches for previously untreatable conditions and reinforces Lilly's commitment to specialized therapeutic areas.
Rare Disease

SETU Secures €159,589 Grant for Novel Gene Therapy Research in Fatal Childhood Canavan Disease

  • SETU has received €159,589 from ELA International to develop an enhanced gene therapy for Canavan disease, marking the first Irish project funded by this organization since 2015.
  • The two-year research project, led by Dr. Lee Coffey, aims to introduce a high-performance version of the ASPA gene to address the fatal neurodegenerative disorder that primarily affects children.
  • The research was personally motivated by Dr. Coffey's twin brother's sons being diagnosed with Canavan disease, demonstrating how personal experience can drive scientific innovation and global collaboration.
Rare Disease

Trump's Tax Bill Passes House with Pharma Reforms: IRA Pill Penalty Remains Unchanged

  • The House of Representatives narrowly passed President Trump's "One Big Beautiful Bill Act" with a 215-214 vote, including modifications to drug price negotiations and restrictions on pharmacy benefit managers.
  • Despite Trump's previous executive order supporting changes to the "pill penalty," the legislation maintains the existing disparity in negotiation exclusion periods between small-molecule drugs and biologics.
  • The White House simultaneously released its MAHA report, which raises concerns about "overmedicalization" of children and calls for increased scrutiny of pharmaceutical industry influence on regulatory bodies.
Rare Disease

CureDuchenne Ventures Invests $1M in Entos Pharmaceuticals for Novel Duchenne Muscular Dystrophy Gene Therapy

  • CureDuchenne Ventures has invested $1 million in Entos Pharmaceuticals to develop a non-viral, redosable gene therapy for Duchenne muscular dystrophy using the Fusogenix PLV delivery platform.
  • The innovative technology aims to overcome limitations of current AAV-based approaches by delivering full-length dystrophin to muscle cells, allowing for redosing, and not excluding patients with pre-existing immunity.
  • Duchenne muscular dystrophy affects approximately 1 in 3,500-5,000 males, causing progressive muscle weakness that typically leads to wheelchair dependence in teens and shortened lifespan due to cardiopulmonary complications.
Rare Disease
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