BOSTON and LIBERTYVILLE, Ill, Nov. 17 /PRNewswire/ -- Paratek Pharmaceuticals, Inc. and Families of Spinal Muscular Atrophy (FSMA), an organization founded to promote research leading to the effective treatment of Spinal Muscular Atrophy (SMA), a debilitating and often fatal disease, today announced an agreement to research Paratek's novel small molecules as a possible treatment for SMA. Using a specific subset of Paratek's novel compounds that were identified through a previous collaborative effort between researchers at the University of Massachusetts Medical School and Paratek Pharmaceuticals, investigators sponsored by Families of SMA will begin testing these compounds both in the laboratory and in animal models of SMA to evaluate their potential use in treating SMA.
Spinal Muscular Atrophy is a genetic disorder involving the deletion or mutation of one or both copies of the Survival Motor Neuron (SMN1) gene on chromosome 5. This mutation causes a chronic deficiency in the production of the SMN protein, 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. In the United States alone there are more than seven million carriers of the genetic risk factors for SMA, and the disease affects approximately one in every 6000 live births.
FSMA-sponsored research has contributed not only to the identification of the SMN1 gene, but also to the identification of another gene called SMN2. In most SMA patients the SMN2 gene contains a mutation that leads to a reduction in the amount of SMN protein produced by the gene and to functional defects in most of the protein that is produced. Previous FSMA-sponsored drug discovery work has identified a series of compounds that may increase the expression of the SMN2 gene and thus the amount of fully-functional protein produced. If a compound can be found that can do this safely and effectively, it may be possible to restore the proper amount of SMN protein in the body and slow or reverse the disease process.
"Paratek is excited to continue researching the application of its novel, oral, non-antibacterial small molecules in the area of SMA," said Dr. Ken Tanaka, Vice President of Research and Development for Paratek. "The collaboration represents an important initiative for patients with this serious and highly debilitating disease that affects thousands in the United States and many more around the world. We feel that this is an important application of our unique tetracycline chemistry technology."
"FSMA is committed to finding an effective treatment for Spinal Muscular Atrophy and we are excited to work with any organization that will help move us closer to our goal," said Audrey Lewis, Executive Director of FSMA. "The steps that are made today in research will lead us to finding an effective way of slowing the progression of SMA or halting it altogether."
As a preparation for clinical trials, FSMA is also funding Project CURE SMA. Project CURE SMA is a multi-centre study part of a worldwide effort to establish baseline measurements in SMA patients, in order to accurately assess the utility of a potential drug or treatment of interest. It is believed that this is the first time these types of clinical tests have been run prior to studying a possible treatment in clinical trials.
"Project CURE SMA represents an important step towards building a foundation from which to launch future therapeutic trials," said Lewis.
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's lead programs are advancing novel compounds related to the tetracycline family that can circumvent or block bacterial resistance, as well as drugs that can prevent infection by bacteria by interfering with Multiple Adaptational Response (MAR). Out of these efforts, Paratek has discovered a new class of antibiotics, the aminomethylcyclines, that target the need for new and potent antibacterials to overcome the problem of rapidly growing bacterial resistance. The Company's lead antibiotic clinical candidate, PTK0796 / BAY 73-6944, the first product from this class, is being developed in a collaborative partnership with Bayer AG. Paratek is privately held and headquartered in Boston, MA. For more information, visit Paratek's website at http://www.paratekpharm.com/.
Families of SMA is a volunteer-driven nonprofit organization based in Libertyville, IL. It was founded in 1984 by parents of children suffering from SMA. FSMA is the largest international organization dedicated solely to eradicating 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 has more than 22 chapters worldwide and more than 5,000 member families. FSMA has funded $12 million towards SMA research, and has already committed an additional $8 million over the next three years. For more information visit the website http://www.curesma.com/ or call 1-800-886-1762.
Families of Spinal Muscular Atrophy; Paratek Pharmaceuticals, Inc.