Drug Developer Begins Trial of Autism Treatment
WASHINGTON — Yamo Pharmaceuticals recently enrolled its first patient in a phase 2 clinical trial that may result in medicines able to treat the core behaviors of autism spectrum disorder.
“There are 20 million or more people with autism around the world, and from everything we’ve seen they are desperate for something,” said Eugene Prahin, the chief financial officer at Yamo Pharmaceuticals, during a phone call with The Well News.
Autism is a group of complex neuro-developmental disorders which can impact a person’s social communication and interactions.
Prior research suggests that genetic and environmental factors are root causes of autism, which typically begins in early childhood and continues throughout a person’s life.
Currently, there are no approved treatments which target the core symptoms of autism. According to Prahin, there are only a few antipsychotic medicines that are approved to target autism-related irritability.
“[The inventor] thought that people with autism are showing a flight or fight response, and that a drug that regulates the central nervous system activity should help these kids,” said Prahin.
A drug known as L1-79, which inhibits production of an organic compound called catecholamine, was exactly that.
To develop L1-79, Yamo Pharmaceuticals took an existing drug and developed a less severe and less toxic version.
In 2016, Yamo Pharmaceuticals completed a 12-week open label trial with L1-79 involving eight patients of various ages, from toddler to adult, in clinics in New York and New Jersey who took the pill twice a day.
Based on the positive results of the open label study, Yamo Pharmaceuticals worked with the U.S. Food and Drug Administration to develop a safety and dose ranging study involving 42 participants, aged 13 to 21, from 2016-2018.
In May 2018, the FDA granted Yamo Pharmaceuticals a Fast Track Designation based on all of the available study results.
“At the end of the last study, a number of parents were so impacted by kids being on L1-79 that they requested a special message to FDA about the benefits of the drug,” said Prahin.
Prahin said that a lot of the parents said prior to the study their children did not have normal sleeping patterns, or were prone to self-harm, were aggressive with families and care takers, or found it hard to show empathy, but after the trial of L1-79 the parents noticed changes in these behaviors.
The behavior changes were also observed by third parties who were unaware of the trial, like a school bus driver who experienced a new type of interaction with the participant.
“It’s not just these kids who have difficult lives but their families. It’s the kind of stuff where a minor improvement really changes a family’s life,” said Prahin.
The phase 2 clinical trial, which started last week, will involve two cohorts of participants to receive either a high or low dose of L1-79 ranging from 400-600 mg a day.
The company will continue to recruit participants between the ages of 12-21 who have an IQ over 70 from six clinical sites that are leaders in the autism space, such as the Thompson Autism Center at the Children’s Hospital of Orange County.
They also plan to offer a compassionate use program, in conjunction with any future phase 3 studies, for parents who have seen positive results in the children who underwent a previous trial.
“Our objective is not to exclude anyone, but to identify the best patient population to show efficacy,” said Prahin.
The trial received funding from the Autism Impact Fund, whose mission is to revolutionize the status quo for diagnosing, treating and living with autism through a venture capital model, which has also funded six other companies outside of Yamo Pharmaceuticals that are focused on reforming care for those living with autism.
The phase 2 clinical trial is expected to end in the middle of next year, Prahin said, and future clinical studies will seek to incorporate a younger demographic.
“Assuming the study we just started is successful, after that, we will do two phase 3 pivotal studies, and if those show statistical significance, then it will likely be approved by FDA, as long as they are otherwise okay with our drug’s efficacy and safety profile,” said Prahin.
“If the drug works the way the parents say it works — we still need to prove that — but it would be a tremendous change and benefit to both kids and families,” continued Prahin.
Alexa can be reached at email@example.com
This article has been updated to note that Yamo Pharmaceuticals plans to offer a compassionate use program. in conjunction with any future phase 3 studies, for parents who have seen positive results in the children who underwent a previous trial.
In The News
GUILDFORD, England — Researchers at the University of Surrey have joined the growing chorus of scientists and entrepreneurs who believe... Read More
GUILDFORD, England — Researchers at the University of Surrey have joined the growing chorus of scientists and entrepreneurs who believe perovskite, often referred to as a kind of “miracle” material, just may be the key to speeding the mass production of low-cost solar panels. At the... Read More
WASHINGTON — NASA is making a new effort to figure out the origins of "Unidentified Aerial Phenomena," otherwise known as... Read More
WASHINGTON — NASA is making a new effort to figure out the origins of "Unidentified Aerial Phenomena," otherwise known as UFOs. The space agency announced Thursday it plans to apply scientific methods to information that is publicly available on the unexplained sightings. “Unidentified phenomena in the... Read More
WASHINGTON — Government investment in research and development has led to triumphs in war and economic prosperity, but as lawmakers... Read More
WASHINGTON — Government investment in research and development has led to triumphs in war and economic prosperity, but as lawmakers work on the Bipartisan Innovation Act, the question is: Will that continue? “If the United States wants to maintain its global leadership position — national security,... Read More
WASHINGTON — While manufacturing and industry processes are cited as responsible for emitting the most greenhouse gases, food systems also... Read More
WASHINGTON — While manufacturing and industry processes are cited as responsible for emitting the most greenhouse gases, food systems also account for a hefty portion — 31% — of global emissions, and the U.S. and China, as food superpowers, are the two largest contributors. Emissions from... Read More
MIAMI — It happens every year, yet it never ceases to grab our attention, disrupt our sleep and on occasion... Read More
MIAMI — It happens every year, yet it never ceases to grab our attention, disrupt our sleep and on occasion even terrify. We’re talking, of course, about the Atlantic hurricane season, the period extending from June 1 to Nov. 30 when hurricanes are most likely to... Read More
ALEDO, Texas — The message is clear. Roadside safety messages intended to prevent highway accidents were found instead to contribute... Read More
ALEDO, Texas — The message is clear. Roadside safety messages intended to prevent highway accidents were found instead to contribute to the number of crashes along road segments. Researchers from the University of Minnesota’s Carlson School of Management and the University of Toronto’s Munk School of... Read More