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

    LIBERTYVILLE, Ill., and BOSTON, June 17 /PRNewswire/-- Families of
 Spinal Muscular Atrophy (FSMA) and Paratek Pharmaceuticals, Inc. today
 announced they have extended and significantly expanded their joint
 research and development 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.
 
     The partners have agreed to extend their collaboration for a third year
 and to approximately triple the resources dedicated to the program, with
 both partners increasing their investment in the effort. The Krainer
 Laboratory at Cold Spring Harbor Laboratory and the Hastings Laboratory at
 Rosalind Franklin University of Medicine and Science are also key
 collaborators in the program.
 
     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 that the early, positive results of this collaborative
 program justify this new level of commitment," said Kenneth Hobby,
 Executive Director at FSMA. "Our drug discovery strategy is to invest funds
 to enable companies to begin early-stage programs for this orphan disease.
 As this and other programs progress to later stages, we are looking for
 funding to transition from non-profit to commercial sources."
 
     This exciting announcement also coincides with the SMA community
 bringing its annual conference to Boston, where Paratek is headquartered.
 This is the largest conference in the world for families affected by SMA
 and also for the researchers and companies working to discover treatments
 and a cure for this devastating disease. Mayor Thomas M. Menino has
 proclaimed Saturday, June 21st as "SMA Awareness Day" for the City of
 Boston. Mr. Hobby stated, "FSMA is excited to be in Boston for our
 conference this year -- an important hub of the biotech industry in the
 U.S. During our conference, we hope to increase awareness about SMA to the
 Boston biotech community."
 
     Jill Jarecki, Ph.D., FSMA's Research Director added, "The goal of this
 effort is to develop an SMA drug that safely and effectively restores the
 proper amount of SMN protein in the body by correcting the splicing of the
 SMN2 gene. Compounds generated by Paratek have been shown to accomplish
 this in a number of tissues in animal models of the disease."
 
     Dr. Stuart B. Levy, M.D., Paratek's Vice Chairman, Chief Scientific
 Officer and co-founder, stated, "We are extremely pleased with the
 continuation and expansion of our collaboration with FSMA and equally
 excited about the progress of our SMA program. Developing a clinical
 candidate for SMA, an orphan disease, plays an important role in
 demonstrating new applications of our technology platform based on
 tetracycline derivatives and in the extension of Paratek's R&D pipeline
 into genetic disorders."
 
     Paratek reported early preclinical research from the collaboration at
 the recent annual meeting of the American Academy of Neurology. In vitro
 data showed that tetracycline derivatives could modify defective splicing
 of mRNA resulting from the gene defect seen in SMA. More research,
 including in vivo preclinical studies, is planned and will be presented in
 the suitable peer-reviewed forum in the future.
 
     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, PTK
 0796, is a broad spectrum antibiotic derived from the tetracycline class
 with oral and IV formulations that is being developed for the treatment of
 the most common and serious hospital bacterial infections, including those
 caused by resistant strains such as MRSA (methicillin-resistant
 Staphylococcus aureus) and MDRSP (multi-drug resistant Streptococcus
 pneumoniae). Oral and IV formulations of PTK 0796 were compared to Zyvox(R)
 in a recently completed Phase 2 clinical study in complicated skin and skin
 structure infections (cSSSI). In addition to PTK 0796, Paratek is also
 developing other broad- and narrow-spectrum tetracycline antibiotics to
 treat hospital and community infections based on its novel tetracycline
 chemistry expertise.
 
     Outside of its tetracycline antibacterial program, Paratek has also
 identified small molecules that inhibit bacteria-specific transcription
 factors for Multiple Adaptational Response (MAR) genes which control
 bacterial virulence and resistance development.
 
     Based upon a growing body of clinical research and as part of its
 effort to exploit its novel tetracycline derivatives and their unique
 mechanism of action in selected inflammatory and neurodegenerative
 conditions, 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. In addition, Paratek is encouraged by early
 evidence of the ability of tetracycline derivatives to affect mRNA
 splicing, as in SMA, which may also have activity in related orphan genetic
 disorders, such as cystic fibrosis (CF), and Duchene Muscular Dystrophy.
 
     Paratek has active collaborations with Merck & Co., MerckSerono,
 Warner-Chilcott and FSMA to develop tetracycline derived small molecule
 drugs for bacterial infections, multiple sclerosis (MS), acne & rosacea,
 and spinal muscular atrophy (SMA), respectively. Paratek is privately held
 and headquartered in Boston, Massachusetts, USA. For more information about
 Paratek and its research and development initiatives, visit Paratek's
 website at http://www.paratekpharm.com.
 
     About Families of SMA
 
     FSMA is dedicated to developing a treatment and cure for SMA by
 promoting and supporting research, 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 50,000 members and supporters. FSMA
 receives the majority of its funding through volunteer efforts, investing
 almost $40 million to date. FSMA-sponsored research has made significant
 contributions to advancing new therapies towards human clinical testing.
 
     For more information, visit the website www.curesma.org or call
 1-800-886-1762.
 
 
 

SOURCE Paratek Pharmaceuticals, Inc.

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