Coya Therapeutics is hoping to gain approval of its amyotrophic lateral sclerosis (ALS) disease-modifying therapy (DMT) based on its Phase II trial.
Following the positive outcome of a Phase I trial (NCT06307301) of COYA 302 in ALS, Coya Therapeutics spoke with the US Food and Drug Administration (FDA) to initiate a mid-stage study, which was given the go-ahead in August 2025.
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Coya Therapeutics’ CEO, Fred Grossman, told Clinical Trials Arena that the company hopes that if successful, the Phase II trial will set the drug up for approval in ALS.
“Other therapies available for ALS were approved on a single trial – primarily due to the unmet need and lack of adequate therapies. Depending on the data we gather when the trial reaches its six-month endpoint, we’ll move forward accordingly,” stated Grossman.
The company has now initiated the ALSTARS study (NCT07161999), from which it hopes to seek potential approval based on the intial six month period.
The three-arm trial, which aims to enrol 120 patients across 25 sites in the US and Canada, will assess the efficacy and safety of COYA 302 in a biweekly and once-monthly subcutaneous dosing regimen. The candidate will be tested against placebo for an initial six month period.
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By GlobalDataAfter the initial six-month period, Coya will conduct a blinded extension phase, which will allow patients originally given placebo to receive COYA 302 for a further six months.
“The placebo group will present some interesting data, as it will allow us to look at if patients can be stabilised on one of these doses after six months on placebo,” Grossman noted.
The primary endpoint for the trial will be ALS Functional Rating Scale-Revised (ALSFRS-R) scores.
Addressing unmet needs in ALS
While four medications have been approved for use in ALS, patients still face a plethora of unmet needs.
“ALS is a relentless disease that often leads to death within five years of diagnosis,” said Grossman.
Coya hopes to target this unmet need with COYA 302, which combines a low dose of interleukin-2 (IL-2) and cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) to both enhance Treg prevalence and reduce inflammation – ensuring that the Tregs do not revert to a pro-inflammatory state.
The technology for COYA 302 is based on T-regulatory cells (Tregs). In October 2025, Shimon Sakaguchi, scientific advisor to Coya Therapeutics, along with Mary Brunkow of the Seattle Institute for Systems Biology and Fred Ramsdell of Sonoma Biotherapeutics, was awarded the Nobel Prize in Physiology or Medicine for his work in uncovering the role of Tregs in managing inflammation.
Grossman said that the drug has already been shown to stabilise ALS-associated decline and durably increase the number and suppressive potential of Tregs in the Phase I trial. Following an extension period, Coya said it also observed stabilisation in patients for “close to a year”.
COYA 302’s role in inflammatory disease
Alongside its efforts to get COYA 302 to patients with ALS, Coya is also exploring the potential of the therapy in frontotemporal dementia (FTD), which “shares commonality from a genetic molecular perspective”, noted Grossman.
“We will initiate the trial following FDA approval of our investigational new drug (IND) application in FTD,” he added.
However, Grossman believes that COYA 302’s potential could extend further, as the drug’s mechanism is “agnostic” to a specific neurodegenerative disease.
“COYA 302 has potential applications in Parkinson’s, and we fully intend to move forward in other indications as well,” Grossman commented.
