CHARLOTTESVILLE, Va.,
July 22, 2021 /PRNewswire/
-- HemoShear Therapeutics, Inc., a clinical stage company
developing treatments for rare metabolic disorders, today announced
that the first two patients have been dosed in the HERO (HElp
Reduce Organic Acids) Phase 2 clinical trial of HST5040, an oral
small molecule drug being investigated for the treatment of
patients with methylmalonic acidemia (MMA) and propionic acidemia
(PA). This trial seeks to advance the first oral therapy designed
to directly address underlying biochemical causes of these rare and
life-threatening diseases.
HST5040 is being developed by HemoShear to lower toxic
metabolites that build up in MMA and PA as a result of genetic
deficiencies in key enzymes required to break down certain amino
acids from protein. These toxins can result in severe organ damage,
developmental deficits, and premature death. Despite the
current standard of care involving dietary control, carnitine
supplementation and in more severe cases, organ transplantation,
there remains a high unmet medical need in MMA and PA. As a
small molecule, HST5040 has the ability to distribute to all
affected tissues and thus has the potential to be active throughout
the body, including the liver, kidneys, brain, heart and muscles.
HST5040 is designed for convenient daily administration at home as
a liquid formulation taken either orally or through a gastric
feeding tube. The FDA has granted HemoShear's HST5040 Orphan Drug,
Fast Track and Rare Pediatric Disease designations to treat MMA and
PA.
"There are currently no targeted pharmacologic treatments for
MMA or PA that can improve quality of life or extend lifespan for
these devastating diseases," said Kimberly
Chapman, MD, PhD, genetic metabolist at Children's National
Hospital in Washington, DC.
"The patient community is excited for the possibility of a
convenient, daily oral treatment approach."
The HERO study is enrolling at least 12 patients with MMA or PA
aged 2 and older. The study will include three sequential treatment
periods: an open-label, within-patient, dose escalation
period; followed by a randomized, double-blind, placebo-controlled
crossover period; and then an open-label, long-term extension
period. If the data are encouraging, enrollment in the long-term
extension period of the HERO study may be expanded to include
patients who have had liver or kidney transplants and those with
additional types of MMA.
The first study participants to receive HST5040 are pediatric PA
patients under the care of Jennifer
Gannon, MD, the principal investigator at Children's Mercy
Kansas City. Additional study sites include Boston Children's
Hospital, UMPC Children's Hospital of Pittsburgh, Children's National Hospital, Rady
Children's Hospital of San Diego, University
of Florida, University of
Minnesota, University of Utah, Vanderbilt
University Medical Center and Yale Center for Clinical
Investigation. More information can be found at
www.MMA-PAHero.com or
ClinicalTrials.gov (NCT04732429).
"This is a tremendous milestone for patients and families who
desperately need a treatment that can potentially target all organs
affected by these diseases," said Jim
Powers, Chairman and CEO of HemoShear. "We named this the
HERO study because of the challenges brave families face every day
living with MMA and PA. We look forward to monitoring the impact of
HST5040 on participants in the study."
About MMA and PA
Methylmalonic acidemia (MMA) and propionic acidemia (PA) are
rare genetic disorders caused by the deficiency of key enzymes
required to metabolize certain amino acids from protein. MMA and PA
are diagnosed through newborn screening in the United States and in select countries in
Europe, the Middle East and the rest of the world. The
diseases result in the buildup of toxic metabolites that can lead
to frequent acute metabolic decompensations, severe organ damage,
seizures, developmental deficits, and premature death. In
the United States, about 1 in
70,000 newborns is diagnosed with MMA, and about 1 in 240,000 is
diagnosed with PA. Both diseases are more common in the
Middle East and North Africa. There are an estimated 4,000 MMA
and PA patients in the US and Europe combined.
About HemoShear Therapeutics
HemoShear Therapeutics, Inc. is a privately held clinical stage
company developing treatments for rare metabolic disorders with
significant unmet patient need. HemoShear's drug discovery
platform, REVEAL-Tx™, enables the Company's scientists to
create best-in-class, biologically relevant human disease models to
uncover the underlying mechanisms of disease, translate those
discoveries into drug targets, and select candidates that may treat
patients successfully. In addition to the Company's
proprietary rare disease programs, HemoShear has exclusive
partnerships to identify novel therapeutic approaches in
nonalcoholic steatohepatitis (NASH) and an undisclosed rare liver
disease with Takeda, and in gout with Horizon Therapeutics.
For more information visit www.HemoShear.com.
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SOURCE HemoShear Therapeutics