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

    LIBERTYVILLE, Ill., and BOSTON, April 3 /PRNewswire/ -- Families of
 Spinal Muscular Atrophy (FSMA) and Paratek Pharmaceuticals, Inc. today
 announced they have extended their joint R&D collaboration to develop a
 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 is focused 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.
     "We are excited by the progress in our program with Paratek, which in
 the past year has demonstrated positive results in early cell models of
 this disease," said Kenneth Hobby, Executive Director of FSMA. "The
 continuation of this collaboration also represents FSMA's broader
 commitment to expand our relationships with life sciences companies and
 build a pipeline of drug candidates. As reported recently in the Wall
 Street Journal, we believe these kinds of interactions and early stage
 support can provide a critical impetus for companies to innovate new
 therapies for serious diseases such as SMA that may have been otherwise
 overlooked."
     Dr. Ken Tanaka, Paratek Pharmaceuticals' Vice President of Research and
 Development, stated, "It is an honor to work with the committed and
 resourceful team at FSMA. Together with FSMA and with the team of
 scientists led by Dr. Adrian Krainer at Cold Spring Harbor Laboratory and
 Dr. Arthur Burghes at Ohio State University, we have come to a greater
 understanding of the underlying biology of SMA, especially the processes
 that regulate SMN splicing. Our lead candidate has been shown in cells in
 culture and recently in cells in animals to enhance the expression of the
 SMN2 gene. Our goal is to find a compound that can restore normal levels of
 SMN protein in nerve tissue and prepare it for human studies to test the
 ability of this drug to slow or even reverse the disease process."
     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, being developed in collaboration with Merck, has the
 potential to be 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.
 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
 oral non-antibacterial tetracycline derivatives for multiple sclerosis with
 Merck-Serono S.A. 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
 http://www.paratekpharm.com/.
     About Families of SMA
     FSMA is the leading 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. In addition, Families of SMA has funded
 more than $3 million in patient support efforts.
     Since its founding in 1984, FSMA-sponsored research has made
 significant contributions to better understanding SMA and advancing new
 therapies towards human clinical testing. These accomplishments include:
     * Identification of a mutation in the SMN1 gene as the cause of the
       disease. A second copy of the gene called SMN2 produces reduced amounts
       of SMN protein due to a defect in splicing. In treating the disease,
       groups hope to increase synthesis of the SMN2 proteins which can
       compensate for the loss of the SMN1 gene.
     * The establishment of Project CURESMA, a clinical trial network, which is
       currently testing two medications for their possible impact in
       increasing SMN protein production from the SMN2 gene. This network will
       also serve as the conduit for future human drug trials.
     For more information visit the website www.curesma.org or call
 1-800-886-1762.
 
 

SOURCE Paratek Pharmaceuticals, Inc.

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