ILB, Potential ALS Therapy in Phase 2 Trial,Named Orphan Drug in Europe

ILB, Potential ALS Therapy in Phase 2 Trial,Named Orphan Drug in Europe
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The European Commission has granted orphan drug designation to ILB, Tikomed‘s investigational treatment for amyotrophic lateral sclerosis (ALS).

In the EU, this designation is based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP), a part of the European Medicines Agency (EMA). To quality for orphan drug status, an investigational medication must aim to treat a serious condition that affects fewer than five in 10,000 people in the EU, and have sufficient preliminary data to support possible efficacy.

The designation confers benefits including reduced fees and taxes, free scientific advice and help designing studies from the EMA, access to a centralized market authorization procedure, and — if the therapy is approved — 10 years of market exclusivity in the EU.

“This orphan drug designation for our lead drug candidate ILB for the treatment of ALS, is a significant milestone for us in our mission to develop a promising new therapeutic treatment for this chronically debilitating and life-threatening disease for which there is a great unmet need,” Anders Kristensson, CEO of Tikomed, said in a press release.

ILB is a pleiotropic molecule — that is, it is believed to have multiple effects in the body. Its active component, a patented form of dextran sulphate, targets multiple pathways that are involved in the loss of function and death of neurons, which is characteristic of ALS and other neurological diseases. It is administered via a subcutaneous (under-the-skin) injection.

ILB is currently being assessed in a Phase 2 clinical trial (NCT03705390) taking place at Birmingham University Hospital in the U.K. This safety and tolerability study is testing once weekly injections of ILB at 2 mg/kg for up to 48 weeks. It  may still be enrolling eligible patients; information is available here.

ILB was tested in an earlier Phase 2 trial (NCT03613571) at Sahlgrenska University Hospital in Sweden, but the study was stopped early because it only enrolled 13 of 15 intended ALS patients. It reported no safety concerns raised in those treated; it was stopped because the slow pace of enrollment was affecting established treatment development timelines.

Tikomed recently secured over $5 million in funding to support the clinical development of ILB and other investigational medications.

“This [orphan drug] designation from the Committee for Orphan Medicinal Products further acknowledges the compelling clinical data with ILB from our ongoing clinical trial program in ALS,” Kristensson said.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.
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Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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