Phoenix Nest Announces New NIH Grant for Sanfilippo Syndrome (MPSIIIC)

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New York – The Eunice Kennedy Shriver National Institute of Child Health and Human Development at the National Institutes of Health (NIH) recently awarded $224,873 in grant funding to Phoenix Nest, Inc. and University of California San Diego to perform structure-based optimization of a novel pharmacological chaperone therapy for MPSIIIC.

Sanfilippo syndrome (MPS III) is a progressive neurological disorder that is generally diagnosed in childhood and can lead to premature death. The new grant aims to optimize a small molecule as a potential chaperone for MPS III types C. This disorder is caused by deficiency of the enzyme heparan sulfate acetyl CoA: a-glucosaminide N-acetyltransferase (HGSNAT), which is essential for degradation of heparan sulfate. Currently there is no specific treatment for MPS III.

The disease is an excellent candidate for small molecule chaperone therapy as well as gene therapy. The team recently identified promising compounds and the goal of this grant is to resolve the tertiary structure of HGSNAT and use that information to direct the synthesis of potent inhibitors/chaperones of HGSNAT. Compounds identified in this study will provide leads for future optimization. This will leverage the vast X-ray crystallography experience of Professor Geoffrey Chang (Skaggs School of Pharmacy and Pharmaceutical Sciences and the Department of Pharmacology at University of California San Diego), extensive pharmaceutical medicinal chemistry experience of Dr. Joel Freundlich (Rutgers University) and extensive MPSIIIC biology expertise of Dr. Alexey Pshezhetsky (CHU Ste-Justine) and Phoenix Nest’s drug discovery knowledge.

“I am very excited about collaborating on this Sanfilippo syndrome IIIC project, as my laboratory develops innovative techniques for overcoming the challenges of producing and crystallizing integral membrane proteins suitable for biophysical analysis,” said Professor Geoffrey Chang.

“Our research to date has used traditional medicinal chemistry concepts to improve the activity of our compounds and we would benefit if we knew how and where they were binding to this enzyme,” said Dr. Sean Ekins, CEO of Phoenix Nest, Inc. “Having a structure would help us perform structure-based drug design.”

“Years of effort by our scientists and families have brought us to this point,” said Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest. “We are grateful to the NIH for their continued support for this ultra-rare disease, as it provides a ray of light for the many families and their children that struggle with this everyday. It is our goal to raise awareness and provide treatments and we will continue to be at the forefront of recruiting world-class scientists to help us in our quest.”

About Phoenix Nest, Inc. -Phoenix Nest, Inc. is a biotechnology company focused on Sanfilippo Syndrome founded by and solely owned by several Sanfilippo Syndrome families. They actively collaborate with academic researchers to fund and commercialize their technologies. Phoenix Nest, Inc. also licenses assets from companies and universities that may have utility for Sanfilippo Syndrome. For more information, please visit http://www.phoenixnestbiotech.com.

For further information, please contact Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest, 347-689-2186 jwood@phoenixnestbiotech.com

Phoenix Nest and LABioMed receive over $1.7M in grant funding

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LOS ANGELES - The National Institute of Neurological Disorders and Stroke recently awarded over $1.7 million in grant funding to Phoenix Nest, Inc. to continue its partnership with the Los Angeles Biomedical Institute (LA BioMed) to research the development of therapies for treating different forms of a devastating inherited genetic disorder, Sanfilippo disease, also known as MPS III.

MPS III disease is a progressive neurological disorder that is generally diagnosed in childhood and can lead to premature death. The new grants are for therapies to combat devastating brain disease due to MPS III types B and D.

Funding for work in MPS IIID is for recombinant enzyme therapy and is a Phase 2 Small Business Technology Transfer (STTR) award, following a successful $223,102 Phase 1 award. Phoenix Nest and LA BioMed were also awarded a Phase 1 STTR grant to develop a novel stem cell therapy for MPS IIIB syndrome. Both treatments have the potential to completely transform the care of children with MPS III disease, as no approved treatments currently exist for any type.

"We are pleased to continue our partnership with Phoenix Nest, Inc. in this important research to help children living with MPS III B and D. After promising results obtained in our first preclinical studies for an enzyme replacement for MPS IIID, we will next look at efficacy in a disease model with a goal of moving towards the clinic as quickly as we can," said Patricia I. Dickson, MD, LA BioMed lead researcher and director of the institute's MPS Research Laboratory.

Dr. Dickson has specific expertise in the development of intrathecal enzyme replacement therapies for treating MPS by replacing the missing enzyme through the patients' spinal fluid.

"We are very grateful to the National Institute of Neurological Disorders and Stroke for continuing to fund this project to find more effective therapies for MPS IIID, which is so debilitating to the patients and causes enormous stress for the families involved," said Sean Ekins, PhD, chief executive officer of Phoenix Nest, Inc. "The additional funding will be used to manufacture an enzyme for further testing. The research is seeking to develop a therapy that will limit or reverse the neurological damage caused by MPS IIID by delivering an enzyme, recombinant human alpha-N-acetylglucosamine-6-sulfatase (rhGNS), intrathecally to effectively treat the underlying causes of the neurologic symptoms of MPS IIID."

The National Institute of Neurological Disorders and Stroke also provided funding for development of a treatment for MPS IIIB. "This represents a novel approach to use stem cells to make the enzyme alpha-N-acetylglucosaminidase that is needed to achieve therapeutic benefit," said Dr. Dickson.

"Phoenix Nest, Inc. is solely focused on Sanfilippo syndrome, and we are honored to continue collaborating with Drs. Dickson, Tsui-Fen Chou, Michelina Iacovino and their teams so that we can develop multiple treatments for MPS IIIB and MPS IIID," said Dr. Ekins.

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About LA BioMed

Founded in 1952, LA BioMed is one of the country's leading nonprofit independent biomedical research institutes. It has approximately 100 principal researchers conducting studies into improved treatments and therapies for cancer, inherited diseases, infectious diseases, illnesses caused by environmental factors and more. It also educates young scientists and provides community services, including prenatal counseling and childhood nutrition programs. LA BioMed is academically affiliated with the David Geffen School of Medicine at UCLA and located on the campus of Harbor-UCLA Medical Center. For more information, please visit http://www.LABioMed.org

About Phoenix Nest, Inc.

Phoenix Nest, Inc. is a biotechnology company focused on Sanfilippo Syndrome founded by and solely owned by several Sanfilippo Syndrome families. They actively collaborate with academic researchers to fund and commercialize their technologies. Phoenix Nest, Inc. also licenses assets from companies and universities that may have utility for Sanfilippo Syndrome. For more information, please visit http://www.phoenixnestbiotech.com.

For further information, please contact Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest, 347-689-2186 jwood@phoenixnestbiotech.com

Phoenix Nest employee wins first place in Annual California State University Student Research Competition

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Don Clarke, a Master’s student conducting research in Dr. Iacovino’s lab, won first place in the Biology and Agricultural Science Division of the 30th Annual California State University Student Research Competition. He presented his research “Development of a Stem cell/gene therapy approach to treat Sanfilippo syndrome type B” at the annual event which highlights the exceptional research that is conducted by CSU undergraduate and graduate students. The event was held at California State University, Bakersfield on April 29-30.

Phoenix Nest to present posters at upcoming meetings

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Phoenix Nest and LABioMed scientists will present posters at the forthcoming WORLDsymposium and ASBMB meetings.

At World:

Moen DR, Zhang XY, Kan S-H, Wood J, Ekins S, Chou TF and Dickson PI "Uptake and lysosomal delivery of recombinant human alpha-N-acetylglucosamine-6-sulfatase to mucopolysaccharidosis IIID fibroblasts"

Wood J, Moen DR and Ekins S "Virtual collaborations for developing Sanfilippo syndrome treatments on a shoestring"

At ASBMB:

Moen DR, Wang DM, Zhang XY, Kan S-H, Wood J, Ekins S, Chou TF and Dickson PI "Uptake and lysosomal delivery of recombinant human alpha-N-acetylglucosamine-6-sulfatase to mucopolysaccharidosis IIID fibroblasts"

We look forward to seeing you there!