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.
Read the original press release here