FDA clears expanded access program for SPG302 in ALS patients
Treatment aims to promote growth of new synapses
Spinogenix has received clearance from the U.S. Food and Drug Administration (FDA) to launch an expanded access program (EAP) for SPG302, its oral treatment for amyotrophic lateral sclerosis (ALS).
EAPs, also known as compassionate use programs, allow people with serious or life-threatening conditions to access experimental therapies outside of clinical trials when no other treatment options are available.
A total of 200 eligible U.S. patients will be able to join the EAP, Spinogenix said. In addition to providing a treatment option to patients, the program will enable researchers to collect real-world safety and efficacy data on SPG302 that may support its development.
The program will enroll people who are not eligible for the company’s U.S.-based Phase 1/2 trial, which is expected to begin soon following FDA clearance last year.
“FDA clearance to launch this new EAP supports its awareness of SPG302’s safety and potential efficacy, and the need for ALS patients to have access to novel treatments,” Stella Sarraf, PhD, Spinogenix’s CEO and founder, said in a company press release. “This EAP reflects our dedication to giving hope to people living with ALS and their families, providing treatment to those who do not qualify for, or may be unable to participate in, our ongoing clinical trial.”
Expanded access program follows trial in Australia
“Innovation is urgently needed to tackle ALS, a disease with devastating impact, and we are committed to ensuring that no patients who may benefit are left behind,” Sarraf said.
ALS is a progressive disease that affects motor neurons, the nerve cells that control voluntary movement. Researchers don’t entirely understand the underlying causes of ALS, but some hypothesize that loss of synapses, the specialized junctions that allow neurons to communicate with each other, may play a role.
Current ALS treatments may help slow disease progression by easing certain disease mechanisms, but none can reverse existing damage. SPG302, on the other hand, is intended to promote the growth of new synapses, which has the potential to halt or even reverse declines in motor and cognitive function.
SPG302 has been investigated as a once-daily pill in a recently completed, three-part Phase 1/2a trial (NCT05882695) in Australia. In the first two parts, which involved healthy volunteers, the treatment was well tolerated and reached blood levels that were effective in animal models.
A third phase enrolled people with ALS who received either SPG302 or a placebo for 28 days. It was mainly designed to assess the therapy’s safety and pharmacological properties in ALS patients, but efficacy measures such as disease progression, lung function, and disease biomarkers were also assessed.
Results from this third part have not been shared yet, but patients have all been invited to join an active extension study (NCT06903286), also in Australia, to continue to receive SPG302 for up to one year.
Don Doctoroff, founder and chairman of Target ALS, was the first patient to receive SPG302 in the U.S. under a separate EAP protocol. He said his experience with the drug has “has shown that partial stabilization of the disease is a possibility.”
“The notion that ALS is an untreatable disease is outdated,” Doctoroff said.
SPG302 has received FDA orphan drug designation for ALS, a status that provides incentives for the development of therapies for rare diseases. Spinogenix is also developing SPG302 as a potential treatment for Alzheimer’s disease and schizophrenia.
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