By Raffaele Pereno
STOCKHOLM, Sweden – Reporting at BioEurope 2024, CheckOrphan had the opportunity to
meet a promising biotech company, whose lead product seeks to stop the progression of amyotrophic lateral sclerosis (ALS) in its tracks.
Tikomed AB is a privately owned pharmaceutical company based in Viken, Sweden. Adam
Bruce, co-founder and a member of the Board of Directors at Tikomed explained to CheckOrphan, “Over the last two decades, Tikomed has developed and commercialized innovative treatments for patients with severe and life-threatening conditions. Our drugs have the potential to improve outcomes in the treatment of neurological and ophthalmic diseases and for groundbreaking regenerative cell therapies. They give hope for patients lacking satisfactory alternatives.”
The first disease Tikomed has targeted is amyotrophic lateral sclerosis (ALS), and they are
doing it with ILB, Tikomed’s leading asset. ILB is a novel patented formulation of low molecular weight dextran.
ALS is characterized by the loss of motor neurons that in turn leads to muscle loss and,
ultimately, lung and heart failure. What makes ALS complex is that about 5% of people suffering from it have a distinct genetic mutation that is believed to leave those individuals much more susceptible to developing the disease.
However, over 90% of ALS cases – often referred to as sporadic ALS – do not have a clear
genetic mutation, yet, both sporadic and genetic ALS types share internal problems of oxidative stress, mitochondrial dysfunction, glutamate-induced excitotoxicity, apoptosis, neuroinflammation, axonal degeneration, and autoimmune reactions, indicating that ALS is a multifactorial syndrome rather than a single disease.
History of Dextran
Dextran was discovered in 1861 by French chemist Louis Pasteur during his studies on the
viscous fermentation of wine. Today, it is listed among the WHO Essential Medicines and is used medicinally as an antithrombotic agent.
By binding to erythrocytes, platelets, and vascular endothelium, it increases the dissolution of blood clots.
How does ILB work with ALS
ILB stimulates the release of heparin-binding factors, including Hepatocyte Growth Factors
(HGF), from the extracellular matrix of tissues, potentially delaying the onset of the disease.
An increase in HGF following brain damage is known to stimulate and bind to the MET receptor, which triggers neuroprotective and neurotrophic signals.
“ALS ends up disrupting many key pathways in the body, and where other treatments – besides gene therapy – have addressed one or two pathways, we have seen that ILB addresses almost all of the disrupted pathways, which is why we believe our clinical trial results have been quite favorable to date”, Mr. Bruce added.
Tikomed’s Clinical Studies in ALS
The first clinical study in patients with ALS, TM-105, administered ILB via prefilled syringes for subcutaneous injection once weekly for five weeks at a dosage of 1 mg/kg in 13 patients.
A second trial, in collaboration with the University of Birmingham, was recently published: “RG-17-250.”
This open-label study in 11 ALS patients doubled the drug exposure, administering ILB once a week over a 10-week period at a dosage of 2 mg/kg.
Results
Both studies showed a good safety and tolerability profile. Pharmacokinetics/Pharmacodynamics (PK/PD) was assessed as a secondary endpoint, showing a nice correlation between the concentration of ILB and the release of HGF.
Both studies also assessed quality of life using the Amyotrophic Lateral Sclerosis Functional
Rating Scale-Revised (ALSFRS-R), a multidomain assessment tool used by clinicians
to monitor disease progression in ALS patients. It serves as the gold standard for primary
efficacy outcomes in clinical trials, with scores ranging from 0 to 48, where higher scores
indicate better function. The ALSFRS-R did not show any clinical changes, suggesting that the disease progression may have been effectively slowed by the treatment.
Exploratory biomarker N-acetylaspartate (NAA) levels, indicative of mitochondrial dysfunction, showed improvements in both trials, although plasma neurofilament light chain, a non-specific marker of neuro-axonal injury, did not show any significant changes.
Currently, Tikomed is raising funds to run a phase III trial, which if successful, could lead to
ILB’s approval.
About TikoMed AB
TikoMed is committed to improve human life by exploring and harnessing the medical potential of the body’s ability to self-repair and regenerate. With an adaptive, multi-modal mechanism of action, TikoMed’s drug platform has the potential to rebalance the body’s inflammatory, immune and fibrotic responses to acute and chronic inflammation to enhance self-repair and regeneration.
TikoMed’s initial development programs include Amyotrophic Lateral Sclerosis (ALS), Traumatic Brain Injury (TBI) and islet cell transplantation.
TikoMed’s highly scalable proprietary technology aims to provide safe, qualitative and affordable medicine to as many patients as possible across the globe.
TikoMed is privately-owned and based in Viken, Sweden.
Contact
Adam Bruce – Board Member
Phone: +46-70-8238444
Email: [email protected]