Experimental oral therapy SPG302 may slow ALS progression
Treatment found safe, well tolerated in Phase 1/2a study in Australia
SPG302, an experimental oral therapy for amyotrophic lateral sclerosis (ALS), was well tolerated in an early clinical trial, and initial findings suggest it may help slow disease progression.
The new data come from a Phase 1/2a study (NCT05882695) conducted in Australia. The first parts of the study tested SPG302 in healthy volunteers, with results indicating an acceptable safety profile. In the Phase 2a portion, researchers tested SPG302 against a placebo in 23 people with ALS.
Topline results from this part were presented at the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) annual meeting, held Oct. 7-10, in Florida.
“We are very encouraged with the positive Phase 2a trial results for SPG302, in our initial proof of concept ALS study, demonstrating the potential of this new regenerative therapy,” Stella Sarraf, PhD, CEO and founder of SPG302’s developer Spinogenix, said in a company press release.
Understanding ALS and how SPG302 is designed to work
ALS is marked by the degeneration and death of motor neurons, the nerve cells responsible for controlling movement. Neurons communicate with each other via specialized junctions called synapses, but connections begin to deteriorate in very early stages of ALS.
SPG302 is an oral therapy designed to promote the growth of new synapses, potentially helping to restore lost functionality in the nervous system. This may ease ALS symptoms and slow disease progression.
In the Phase 2a portion of the Australian study, participants took 300 mg of SPG302 or a placebo daily for 28 days. After that, all participants received SPG302 for about five months.
The study’s main goal was to evaluate safety, and results were positive — with no treatment-related serious side effects reported over six months on SPG302.
Secondary outcomes showed 82% of participants given SPG302 had a stable or improved rate of decline at the end of treatment, as assessed with the ALS Functional Rating Scale-Revised (ALSFRS-R). When compared against historical controls, those treated with SPG302 had a 76% slower disease progression over six months.
Spinogenix also reported that measures of electrical activity in the brain indicated “improvements in ALS-associated patterns,” which the company said complements the data indicating a slowing of functional decline.
Company planning next steps and expanded access to therapy
Detailed results from the trial will be presented at the upcoming International Symposium on ALS/MND, scheduled for early December in San Diego.
Meanwhile, Spinogenix is planning the next stages of clinical testing of SPG302. Earlier this year, the company received clearance to launch an expanded access program, offering the therapy to 200 ALS patients in the U.S. who are not eligible to participate in clinical trials.
“As we plan our larger registrational directed ALS trial, we also remain committed to making this investigation therapy available to patients who do not qualify for clinical trials through our FDA-cleared Expanded Access Program,” Sarraf said.
SPG302 has received orphan drug designation in both the U.S. and Europe for the treatment of ALS. The designation encourages development of therapies for rare diseases that affect fewer than 200,000 people in the U.S. It also provides regulatory incentives such as tax breaks, fee waivers, and the potential for seven years of market exclusivity if the therapy is approved.
“There is a need to develop new therapies that target neuronal repair pathways for people with ALS. SPG302 represents an important new approach to target synapse loss in ALS,” said Merit Cudkowicz, MD, a member of Spinogenix’s scientific advisory board and director of Mass General Hospital’s Sean M. Healey & AMG Center for ALS.
“I look forward to the next phase of drug development to assess the potential of SPG302 and I am grateful to the leadership at Spinogenix for their additional commitment of a parallel expanded access program for people not eligible for their planned clinical efficacy trials,” Cudkowicz added.
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