LIBERTYVILLE, Ill. and BOSTON, May 17 /PRNewswire/ -- Families of
Spinal Muscular Atrophy (FSMA) and Paratek Pharmaceuticals, Inc. today
announced a joint collaboration to develop a promising drug candidate for
the treatment of Spinal Muscular Atrophy (SMA), the leading genetically
inherited cause of death of children under the age of two years. The
collaboration will focus on optimizing and advancing into the clinic a
novel small molecule within Paratek's library derived from the tetracycline
class of compounds.
Spinal Muscular Atrophy is an often-fatal genetic disorder resulting
from the loss of both copies of the Survival Motor Neuron (SMN1) gene. This
causes a chronic deficiency in the production of the SMN protein, which is
essential to the proper functioning of the motor neurons in the spinal cord
and to the control of muscles in the limbs, neck and chest.
"This new drug development collaboration will focus on optimizing the
drug features of a newly identified lead compound that directly influences
SMN2 gene splicing," said Dr. Jill Jarecki, FSMA Research Director. "FSMA-
sponsored research has contributed to the identification of the SMN1 gene
as well as a second disease-modifying copy of the gene called SMN2.
Normally, the SMN2 gene produces reduced amounts of SMN protein due to a
defect in mRNA splicing. This project aims to develop a SMA drug that
safely and effectively restores the proper amount of SMN protein in the
body in order to slow or reverse the disease process by correcting the
splicing of the SMN2 gene."
"Paratek is excited about this collaboration for several reasons.
First, we already have a compound with validated activity in hand," said
Dr. Ken Tanaka, Vice President of Research and Development for Paratek. "In
addition, this project is of great scientific interest, as the compound has
been shown to directly regulate SMN splicing and should lead to a greater
biological understanding of the underlying disease process. With our
chemistry expertise and the splicing assay capabilities at Cold Spring
Harbor, we believe we have the right resources and team in place to lead us
to a novel drug for treating SMA."
The Krainer laboratory at Cold Spring Harbor will be assessing
additional Paratek compounds from Paratek's collection for effects on SMN2
splicing. In parallel, Paratek will be developing new proprietary compounds
based on this initial lead. In related work, the FSMA scientific advisory
board has recently awarded Dr. Michelle Hastings of Cold Spring Harbor
Laboratory a two- year grant to investigate the molecular mechanism of this
class of compounds.
"FSMA is excited to be funding this drug discovery effort," said Audrey
Lewis, Executive Director of FSMA. "We believe that this compound will work
differently from the other SMN-increasing compounds that FSMA is
developing, providing our community with an additional pathway to
potentially treat this disease."
About Paratek Pharmaceuticals, Inc.
Paratek Pharmaceuticals, Inc. is engaged in the discovery and
commercialization of new therapeutics that treat serious and
life-threatening diseases, with a particular focus on the growing worldwide
problem of antibiotic resistance. Paratek is advancing novel compounds that
can circumvent or block bacterial resistance. Paratek's lead compound, MK-
2764/PTK 0796, is a broad spectrum antibiotic with oral and IV formulations
for the treatment of the most common community and hospital bacterial
infections, including those caused by resistant strains such as MRSA. This
project is being advanced in collaboration with Merck & Co., Inc. In
addition to its tetracycline-derived antibacterials, Paratek is developing
small molecule drugs that can prevent infection by interfering with
Multiple Adaptational Response (MAR) mechanisms in bacteria.
Outside the antibacterial therapeutic area, Paratek has also
established an effort to exploit its novel tetracycline derivatives and
their unique mechanism of action in selected anti-inflammatory and
neurodegenerative conditions, including a collaboration to develop novel
non-antibacterial tetracycline derivatives for multiple sclerosis with
Serono SA. Paratek has an active chemical synthesis effort to produce novel
and diverse small molecules, with the goal of developing non-antibacterial
compounds with improved activity in serious inflammatory and
neurodegenerative diseases based upon a growing body of clinical and basic
research supporting this approach.
Paratek is privately held and headquartered in Boston, Massachusetts,
USA. For more information, visit Paratek's website at www.paratekpharm.com.
About Families of SMA
FSMA is the largest international organization dedicated solely to
eradicating SMA by promoting and supporting research in both the private
and public sector, helping families cope through informational programs and
support, and educating the public and the medical community about SMA. The
organization, originally founded in 1984 by a small group of parents, has
grown to more than 32 chapters and affiliates worldwide and more than 5,000
member families and is a founding member of the International Alliance for
Spinal Muscular Atrophy. FSMA receives the majority of its funding through
volunteer efforts, funding over $25 million to date, and continues to
increase its funding commitments each year with $15 million in new research
planned over the next three years. For more information visit the website
www.curesma.org or call 1-800-886-1762.
SOURCE Paratek Pharmaceuticals, Inc.