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."
SOURCE Paratek Pharmaceuticals