Families of Spinal Muscular Atrophy and Paratek Pharmaceuticals Announce Drug Discovery Collaboration for Spinal Muscular Atrophy

    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.