Funding to Boost Clinical Development of Investigational ALS Therapy ILB

Tikomed has secured $5.5 million in financing from investors to support the ongoing clinical development of ILB, its lead therapy candidate for degenerative neurological diseases, including amyotrophic lateral sclerosis (ALS).

The funding also will help develop Ibsolvmir, a therapy to increase the effectiveness of transplanting cells to treat patients with severe diabetes and chronic pancreatitis.

“We are delighted to have secured this funding, which shows the confidence our shareholders have in our unique drug candidates and expertise,” Anders Kristensson, Tikomed’s CEO, said in a press release.

ILB is a pleiotropic molecule, meaning it produces more than one benefit. It was designed to reduce the signals that lead to cell death, high levels of which are characteristic of several neurological diseases, including ALS.

Among other effects, ILB was reported to normalize the function of mitochondria (cells’ energy factories), block the damaging formation of plaques, and increase the formation of new blood vessels.

“With ILB we present a new class of drug which target the underlying processes responsible for the deterioration of nerve cells characteristically associated with ALS and other neurodegenerative disease,” Kristensson said.

In previous clinical studies ILB administered intravenously (into the blood) was found safe and well-tolerated with only minor and adverse side effects.

ILB is being tested in a Phase 2 clinical trial (NCT03705390) taking place at Birmingham University Hospital in the United Kingdom, under the supervision of Venkataramanan Srinivasan, MD. The trial is currently recruiting participants. More information about enrollment can be found here.

The trial will recruit 15 ALS patients who will be given 2 mg/kg ILB subcutaneously (injected under-the-skin) once weekly for 10 weeks.

The trial’s main goal is to assess the therapy’s safety and tolerability. Additional goals include the evaluation of functional assessments, such as communication, mobility, feeding, dressing, respiration and emotional well-being using the ALS Functional Rating Scale-revised total score (ALSFRS-R) and the ALS Assessment Questionnaire (ALSAQ-40).

Researchers also will measure the presence of neurofilament light chain, a biomarker of neurodegeneration, in patients’ blood.

Tikomed expects to announce trial results later this year.

ILB also was being tested in another Phase 2 trial (NCT03613571) at Sahlgrenska University Hospital in Sweden. However, the study was  terminated, partly because it failed to meet development timelines.