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
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
SOURCE Paratek Pharmaceuticals, Inc.