Yamo Pharmaceuticals Announces Positive Topline Results from Phase 2 Study of L1-79 for the Treatment of the Core Symptoms of Autism
July 30 2024 - 8:05AM
Yamo Pharmaceuticals, a clinical stage pharmaceutical company,
announced today positive topline results of its Phase 2 clinical
trial evaluating the effects of L1-79 in adolescents and young
adults with autism spectrum disorder (ASD). The data demonstrated a
statistically significant improvement with L1-79 compared to
placebo on the Vineland Adaptive Behavior Scale (Vineland-3)
Socialization standard score (difference compared to placebo: 7.09,
p-value = 0.02) during the first 12-weeks of the study.
The Vineland-3 Socialization Domain is a well-established
standardized assessment tool that looks at a person’s coping
skills, ability to engage in play, and leisure and interact with
others.
In addition, other endpoints, such as the Clinical Global
Impression of Severity of Illness (CGI-S) and the Caregiver Global
Impression of Change of 3 Most Bothersome Symptoms of ASD (CGI-3P),
demonstrated statistically significant improvements with L1-79
compared to placebo, corroborating the Vineland-3 findings. L1-79
was generally safe and well tolerated. There were no serious
adverse events or adverse events leading to withdrawal from the
trial with L1-79.
“It is very encouraging to see a more than seven-point
improvement in the Socialization Domain of the Vineland-3,” said J.
Thomas Megerian, MD, PhD, Yamo’s Chief Medical Officer. “We believe
that this is one of the largest statistically significant effects
observed to-date with a medication in ASD on the Vineland-3
Socialization standard score, representing a clinically meaningful
improvement in one of the core domains of function affected by ASD.
The Company is eager to meet with the FDA to discuss Phase 3 plans,
as well as to file for Breakthrough Therapy designation.”
“We are excited to receive these results in the hope to offer
patients and their families a treatment option that can impact the
core symptoms of ASD,” said Chuck Bramlage, Yamo’s Chief Executive
Officer.
About L1-79Evidence suggests that dysfunction
in the catecholaminergic system may play a role in the core
symptoms of ASD. L1-79 is a tyrosine hydroxylase inhibitor expected
to modulate the catecholaminergic pathways, and thereby improve the
core ASD symptoms. L1-79 was granted Fast Track designation by the
FDA in May of 2018.
About the StudyThis Phase 2, multi-center,
randomized, chronic-dosing (12-week) clinical trial was a
two-period placebo-controlled crossover trial that enrolled 58
adolescent and young adults with ASD. Patients were randomized 1:1
to one of two active treatment groups: L1-79 200 mg or 300 mg. On
day one of Period 1, participants in each dosing group were
randomized to receive either L1-79 or placebo BID for 12-weeks.
Following the conclusion of Period 1, participants underwent a
six-week washout period and then crossed over into Period 2, in
which each participant that received placebo in Period 1 received
L1-79 and vice versa BID for another 12-weeks.
Please refer to study identifier NCT05067582 at
www.clinicaltrials.gov for additional clinical trial
details.
About Autism Spectrum DisorderAutism spectrum
disorder or autism refers to a group of complex neurodevelopment
disorders that is defined in the Diagnostic and Statistics Manual
of Mental Disorders V (DSM-5) by “difficulties in social
communication and social interaction, and restricted and repetitive
behavior, interests or activities.” In the U.S., it is estimated
that 1 in 36 children have ASD, with boys being 3.8 times more
likely to be diagnosed than girlsi. Symptoms of repetitive and
characteristic patterns of behavior and difficulties with
social-communication and interaction start early in childhood and
continue throughout a person’s life.
While the causes of ASD are not known, research suggests that
both genes and environment play important roles. Since the
severity, specific types of symptoms, and causes of ASD vary
between individuals, it is referred to as a “spectrum’ disorder.”
Currently there are no approved pharmacological therapies which
address the core symptoms of ASD.
About Yamo PharmaceuticalsYamo Pharmaceuticals
is a clinical stage pharmaceutical company founded in 2015 to
develop L1-79, a novel therapy with the potential to improve the
core symptoms of ASD. Yamo Pharmaceuticals is a privately held
company. More information may be found at www.yamopharma.com.
Safe Harbor StatementThis press release
contains “forward-looking statements” concerning the development
and commercialization of Yamo Pharmaceuticals’ products, the
potential benefits and attributes of such products, and Yamo
Pharmaceuticals expectations regarding its prospects.
Forward-looking statements are subject to risks, assumptions and
uncertainties that could cause actual future events or results to
differ materially from such statements. These statements are made
as of the date of this press release. Actual results may vary. Yamo
Pharmaceuticals undertakes no obligation to update any
forward-looking statements for any reason.
Contact DataInvestor and Media ContactsChuck
Bramlage, CEOcbramlage@yamopharma.com
Eugene Prahin, CFOeprahin@yamopharma.com(212) 610-1570
i Maenner MJ, Warren Z, Williams AR, et al. Prevalence and
Characteristics of Autism Spectrum Disorder Among Children Aged 8
Years — Autism and Developmental Disabilities Monitoring Network,
11 Sites, United States, 2020. MMWR Surveill Summ 2023;72(No.
SS-2):1–14.