Actimed Therapeutics Announces US FDA Grant of Orphan Drug Designation to S-oxprenolol (ACM-002) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
September 04 2024 - 3:00AM
London, UK – 4th September 2024. Actimed
Therapeutics Ltd (“Actimed”), a UK based clinical stage specialty
pharmaceutical company focused on bringing innovation to the
treatment of cancer cachexia and other muscle wasting disorders,
announces that the US Food and Drug Administration (FDA) has
granted Orphan Drug Designation to S-oxprenolol (ACM-002) for the
treatment of amyotrophic lateral sclerosis (ALS).
ALS (also known as Lou Gehrig’s disease) is a progressive, fatal
neuromuscular disease that affects nerve cells in the brain and
spinal cord, eventually leading to a loss of muscle control and
paralysis. ALS prevents motor neurons sending impulses from the
brain and spinal cord to the muscles, causing muscle atrophy and
weakness and has a mean survival of 3.5 years. Voluntary muscle
action is progressively lost and in late-stage ALS patients may
have difficulty communicating, swallowing, moving, and breathing.
There is currently no cure for ALS.
Cachexia, a complex wasting disease, may occur in the early
stages of ALS and has a significant impact on quality of life,
proving terminal in some patients. In pre-clinical ALS models,
S-oxprenolol has demonstrated significant benefits including
slowing disease progression, improvement in survival, attenuation
of muscle loss, motor neuron protection, reduction in loss of body
mass and improvements in lean body mass1.
Robin Bhattacherjee, Chief Executive Officer of Actimed
Therapeutics commented “We are pleased to receive Orphan
Drug Designation for S-oxprenolol in ALS, where loss of body mass
and muscle wasting can have a significant impact on survival. ALS
is a devastating orphan disease of high, unmet medical need and the
early non-clinical data we have generated for S-oxprenolol in this
indication suggest that it could have a potential role in meeting
some of the important needs of this under-served patient
population”.
FDA Orphan Drug Designation is granted to investigational
therapies which are intended for the safe and effective treatment,
diagnosis or prevention of rare diseases or conditions that affect
fewer than 200,000 people in the United States. Orphan Drug
Designation provides several benefits to drug developers, including
certain development cost benefits in the US, increased FDA
interaction and eligibility for seven-year market exclusivity
following approval.
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About Actimed Therapeutics Actimed
Therapeutics is a clinical stage specialty pharmaceutical company
focused on bringing innovation to the treatment of muscle wasting
disorders to transform the care of an underserved and vulnerable
patient population.
The lead area of focus for Actimed is specifically in cachexia.
Cachexia is a wasting disease that is associated with cancer and
other serious chronic illnesses and with significant morbidity and
mortality. A significant number of cancer patients suffer from
cachexia2 and it is estimated that cachexia is responsible for up
to 20% of all cancer deaths3. A recent meta-analysis demonstrated
that cachexia was associated with an 82% higher relative risk of
mortality in patients with NSCLC versus no cachexia4.
Despite its prevalence and devastating clinical effects, there
is no globally approved drug for the treatment or prevention of
cancer-related cachexia.
The lead product of Actimed, S-pindolol benzoate (ACM-001.1)
targets multiple pathways that drive cachexia and has generated
promising proof of concept Phase 2a clinical data in cachexia
patients. Actimed is currently preparing for further clinical
studies in cancer cachexia having received an Investigational New
Drug (IND) approval from FDA for this programme in August 2023.
Actimed also owns the global rights to its second asset,
S-oxprenolol, which is being developed by the Company for the
muscle wasting seen in amyotrophic lateral sclerosis (ALS) where
loss of body mass and muscle wasting may impact survival5. Actimed
has licensed the global rights to develop and commercialise
S-oxprenolol for cancer cachexia and any other indications outside
of ALS to US company Faraday Pharmaceuticals.
FOR MORE INFORMATIONActimed
Therapeuticswww.actimedtherapeutics.com
MEDiSTRAVA ConsultingFrazer Hall, Erica
HollingsworthTel: +44 (0)203 928 6900Email:
actimed@medistrava.com
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1Suzuki et al, JCSM 2018; 9, 183-212. (Poster 5-17, page
200)2 Anker M et al., J. Cachexia, Sarcopenia and Muscle; 2019: 10:
22 – 243 Argilés JM et al, Nat Rev Cancer 2014; 14:754-624 Bonomi
P. et al. The mortality burden of cachexia in patients with
non-small-cell lung cancer: A meta-analysis; International
Conference of Sarcopenia, Cachexia and Wasting Disorders, June 17 –
18 2023, Stockholm, abstract 2-18, page 1395Wolf J et al., PMID
28184974 DOI: 10.1007/s00115-117-0293