Developer gets OK to expand first-in-human trial of ALS therapy to Europe
PIONEER-ALS now testing safety of VTx-002 in adults at sites in US
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- PIONEER-ALS, a clinical trial evaluating VTx-002 as a treatment for ALS, has been given a green light to launch study sites in three European nations.
- The therapy targets toxic TDP-43 protein clumps, a hallmark of ALS.
- The trial, already underway in the U.S., aims to assess VTx-002's safety, tolerability, and early effectiveness in adults with ALS.
Vectory Therapeutics has been given a green light to expand its first-in-human clinical trial of VTx-002, its treatment candidate for amyotrophic lateral sclerosis (ALS), to sites in the U.K. and the European Union.
The Phase 1/2 PIONEER-ALS study (NCT07287397) is already underway in the U.S., and is still enrolling adults with the progressive disease at sites from coast to coast. Following clearance from the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and the European Medicines Agency (EMA), it will now add study sites in the U.K., Belgium, and the Netherlands, according to a company press release.
The trial, which recently dosed its first participant, is evaluating VTx-002’s safety, tolerability, and early effects on clinical endpoints that include lung function, disease progression, and muscle strength. Researchers are also examining changes in disease biomarkers and quality of life.
“Authorization from the MHRA and the EMA to initiate our Phase 1/2 clinical trial enables us to expand the clinical evaluation of our therapy globally and collaborate with leading investigators across numerous regions as we advance this potential new treatment for people living with ALS,” said Jim Scibetta, CEO of Vectory.
A hallmark of ALS, observed in about 97% of all cases, is the abnormal folding of the TDP-43 protein and its inclusion in toxic clumps, or aggregates.
The resulting loss of TDP-43 from the nucleus, where it exerts its normal functions, and the accumulation of these toxic protein clumps in nerve cells is believed to contribute to nerve cell damage and ALS progression.
VTx-002 targets toxic protein clumps in ALS
VTx-002 is designed to enable nerve cells to produce antibodies that target these toxic aggregates, tagging them for clearance by the immune system and restoring the essential function of TDP-43 in the nucleus.
The therapy uses a modified virus to deliver the genetic instructions coding for the antibody directly to nerve cells in the brain and spinal cord. It’s delivered as a one-time injection to the cisterna magna, a large fluid-filled space at the base of the brain. Treatment is expected to result in long-lasting antibody production by nerve cells, according to the developer.
The PIONEER-ALS study is enrolling about a dozen adults with ALS, who will receive one of two VTx-002 doses. All participants will be followed for about five years after the injection. The trial is designed so that each of the participants receive the experimental medication; there will be no control group.
The main goals of the study are to evaluate the treatment’s safety, tolerability, and preliminary signs of effectiveness. Researchers will also assess the drug’s pharmacokinetics, or how it moves into, through, and out of the body.
Among secondary and exploratory assessments are changes in blood levels of neurofilament light chain, a marker of nerve cell damage, and changes in new TDP-43-related biomarkers.
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