BOSTON, Oct. 15 /PRNewswire/ -- Paratek Pharmaceuticals (Boston, MA) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announced today that a jointly funded drug development program for Spinal Muscular Atrophy (SMA) has been awarded a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS).
The five-year cooperative agreement encompasses pre-clinical drug development up to the time of an Investigational New Drug (IND) Application to the FDA. The program is focused on developing a novel small molecule of the tetracycline family from within Paratek's propriety compound library. The potential for success of the drug candidate is evaluated at key stages of the development using quantitative go/no-go milestones that have been established in the agreement between Paratek and the NINDS. The Krainer Laboratory at Cold Spring Harbor Laboratory and the Hastings Laboratory at Rosalind Franklin University are also key collaborators in the program and will also receive funding under the NINDS cooperative agreement.
Spinal Muscular Atrophy is the leading genetic cause of death of infants. It 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 to the control of muscles in the limbs, neck and chest. SMA is typically marked by the deterioration of the muscles that control crawling, walking, swallowing or breathing. There are no approved therapies for the treatment of SMA. Approximately 1 in 6,000 babies born is affected. One in 40 people are genetic carriers, indicating approximately 7.5 million carriers in the United States.
The compounds under investigation at Paratek are intended to correct RNA splicing of a low functioning back-up gene to SMN1 called SMN2, which will in turn increase SMN protein levels. The leading drug candidate has been shown to accomplish this in a number of tissues in animal models of the disease. The focus of the project with this new grant funding will be to optimize the drug-like properties of this compound class through directed medicinal chemistry.
"At Families of SMA we are excited that our $2 million initial investment at an early stage of this project has provided the preliminary data to leverage larger funding amounts from NIH. We feel this grant award is wonderful validation of the Families of SMA research funding program and more specifically of Paratek's promising drug program for SMA," said Jill Jarecki Ph.D., Research Director at Families of SMA.
"The Families of SMA drug discovery strategy is to invest funds to enable companies to begin early-stage programs for this orphan disease, and as programs progress to later stages, we look for funding to transition from non-profit to commercial and government sources. Clearly Paratek's program is a successful demonstration of that approach," said Kenneth Hobby, President at Families of SMA.
Dr. Stuart B. Levy, M.D., Paratek's Vice Chairman, Chief Scientific Officer and Co-Founder, stated, "We are very grateful for the support of Families of SMA, which has helped get us to this exciting moment. We are encouraged that through the NINDS funding, we shall identify a drug candidate for this devastating disease."
John D. Porter, Ph.D., Program Director for the Paratek award at NINDS, stated, "This award was made through the NINDS Cooperative Program in Translational Research -- a novel, milestone-driven program that solicits ideas for therapies from disease communities, evaluates those ideas through peer review, and both optimizes and assesses candidate therapies at each stage of development. This cooperative project combines the corporate resources and expertise of Paratek, the research experience of the academic investigators, the focus, insights, and funding of the Families of SMA, and the funding and project management experience of NINDS staff. It represents a powerful strategy for developing new therapies for SMA and other diseases."
Families of SMA and SMA Europe (through SMA Trust), a consortium of European SMA advocacy groups, have also co-funded a recent grant award to Paratek Pharmaceuticals for their SMA drug program. This award will cover aspects of the program not funded by the NINDS allocation, including funding to the laboratory of Dr. Louise Simard at the University of Manitoba.
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 involving technology initially developed by Paratek co-founder Dr. Stuart Levy's laboratory at Tufts University School of Medicine, and licensed to Paratek. Paratek's lead compound, PTK 0796, is a broad-spectrum antibiotic derived from the tetracycline class with oral and IV formulations that are being developed for the treatment of the most common and serious hospital and community bacterial infections. Oral and IV formulations of PTK 0796 are being compared to Zyvox(R) in Phase 3 clinical studies to treat cSSSIs. Phase 3 studies in additional indications such as CABP are planned.
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 other orphan splicing disorders.
Paratek has active collaborations with Novartis, Merck Serono, Warner-Chilcott and FSMA to develop tetracycline derived small molecule drugs for life-threatening 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
Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by: Funding and advancing a comprehensive research program; Supporting SMA families through networking, information and services; Improving care for all SMA patients; Educating health professionals and the public about SMA; Enlisting government support for SMA; Embracing all touched by SMA in a caring community. FSMA's vision is a world where Spinal Muscular Atrophy is treatable and curable.
A small group of parents started Families of SMA in 1984. They wanted to raise funds for SMA research to cure the disease, and support all affected families. Back then, very little was known about Spinal Muscular Atrophy. Very little research was being conducted. No one knew the cause of the disease let alone how to find a treatment and a cure. There were no family support services and no clinical trials. Patients and families affected by SMA were on their own and had little hope.
Today, FSMA has a different story to tell. Families of SMA has created hope for the SMA community that did not exist in 1984. FSMA has raised and funded over $50 million for SMA research. Support comes from generous individual donations and numerous fundraising events held by volunteer families and our Chapters.
Families of SMA funds and directs the leading SMA research programs. The successful results and progress from basic research to drug discovery programs to clinical trials provides real hope for families and patients:
Families of SMA is a non-profit, 501(c)3 tax exempt organization with 26 Chapters throughout the United States and over 65,000 members and supporters.
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