Retrotope Announces the Initiation of Expanded Access (Compassionate Use) Trial of RT001 in Amyotrophic Lateral Sclerosis (ALS)

Los Altos, California, UNITED STATES

First ALS patient dosed with RT001

LOS ALTOS, Calif., Sept. 17, 2018 (GLOBE NEWSWIRE) -- Retrotope today announced that it has supplied its drug RT001 for the initiation of an expanded access (EA) trial to treat patients with amyotrophic lateral sclerosis (ALS).  This EA program in ALS, requested by renowned investigators at major medical centers, is expected to enroll a few patients at each site and is intended to provide information to guide the design of future randomized placebo-controlled trials.  RT001 is the first-in-class of a new drug category called deuterated polyunsaturated fatty acids (D-PUFAs), which are a novel approach to protect against lipid peroxidation damage resulting in cell death that is a hallmark of numerous neurodegenerative diseases including Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, ALS and several inherited disorders including Friedreich’s Ataxia (FA) and infantile neuroaxonal dystrophy (INAD).  

ALS is a progressive neurodegenerative disease mainly involving dysfunction and death of motor neurons. Approximately 15,000 Americans are diagnosed with the disease annually. Current treatments do not arrest underlying disease, and patients usually die from respiratory failure three to five years following diagnosis. Although much remains unknown about ALS, there is increasing evidence that oxidative stress and mitochondrial dysfunction may play a role in its pathogenesis (

“Early results in clinical trials of RT001 and expanded access programs for a variety of neurodegenerative diseases, including Friedreich’s ataxia, infantile neuroaxonal dystrophy (INAD) and late-onset Tay Sachs, which involve oxidative mitochondrial dysfunction, have generated positive results along with a favorable safety profile,” said Peter G. Milner, M.D., Chief Medical Officer of Retrotope. “We look forward to providing updates on this and our other studies in the coming months.”

Robert J. Molinari, Ph.D., Chief Executive Officer and co-founder of Retrotope, added, “Patients are excited to try a potentially efficacious and disease-modifying treatment for largely untreated, devastating and fatal diseases. Therefore, while we prepare and initiate our pivotal studies of RT001 in INAD and Friedreich’s ataxia, our ability to supply drug to expanded access programs requested by patients and their physicians allows us to provide access to this important experimental therapy and generate valuable clinical information about our drug’s performance in different diseases.”

About Retrotope
Retrotope, a privately held, clinical-stage pharmaceutical company, is creating a new category of drugs to treat degenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being considered as disease-modifying drug therapies for many intractable diseases, such as Infantile Neuroaxonal Dystrophy (INAD), ALS, Late Onset Tay Sachs (LOTS), Progressive SupraNuclear Palsy (PSP), Huntington’s disease, mitochondrial myopathies, and retinopathies.  RT001, Retrotope’s first lead candidate, is being tested in placebo-controlled clinical trials for the treatment of Friedreich's ataxia, a fatal orphan disease, and in compassionate use studies for other neurodegenerative diseases such as INAD, LOTS, PSP, ALS and a genetic form of Alzheimer’s disease.  For more information about Retrotope, please visit

Retrotope Media Contact:
Nancie Steinberg, Burns McClellan
212-213-0006, ext. 318,

SOURCE: Retrotope, Inc. 4300 El Camino Real, Suite 201 Los Altos, CA 94022 650-575-7551