PHOENIX NEST, LLC

6PP02

2020-08-18

http://phoenixnestbiotech.com/

2019-08-19

2012-03-24

2024-07-01

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Department of Health and Human Services

IDENTIFICATION OF POTENTIAL BIOMARKERS AND CLINICAL TOOLS FOR USE IN MUCOPOLYSACCHARIDOSIS IIIC PATIENTS. - PROJECT SUMMARY/ABSTRACT LYSOSOMAL STORAGE DISEASES (LSD) ARE RARE INHERITED METABOLIC DISORDERS CAUSED BY DEFECTS IN THE CELLULAR CATABOLIC SYSTEM. MUCOPOLYSACCHARIDOSIS TYPE IIIC (MPS IIIC OR SANFILIPPO DISEASE TYPE C) IS ONE SUCH LSD THAT IS CAUSED BY DEFICIENCY OF THE ENZYME HEPARAN SULFATE ACETYL COA: -GLUCOSAMINIDE N-ACETYLTRANSFERASE, (HGSNAT) ESSENTIAL FOR DEGRADATION OF HEPARAN SULFATE, A REPEATING CARBOHYDRATE GENERALLY FOUND ATTACHED TO PROTEOGLYCANS. THIS DISEASE CAUSES ACCUMULATION OF HEPARAN SULFATE RESULTING IN A PROGRESSIVE AND SEVERE NEUROLOGICAL DETERIORATION EARLY IN LIFE WITH LITTLE SOMATIC FEATURES. THE SYMPTOMS IN PATIENTS WITH MPS IIIC MAY PRESENT AT AN AVERAGE AGE OF 3.5 YEARS OF AGE WITH PSYCHOMOTOR DEVELOPMENTAL DELAYS AND BEHAVIORAL PROBLEMS. BEFORE THE AGE OF 15 YEARS VERBAL COMMUNICATION IS OFTEN LOST IN PATIENTS WITH MPS IIIC. MOST LOSE THE ABILITY TO WALK BETWEEN THE 20 AND 30 YEARS OF AGE. THE CONDITION IS FATAL BY AN AVERAGE AGE OF 34 YEARS (RANGE, 25-48). ENZYME REPLACEMENT THERAPIES ARE NOT AN OPTION SINCE THE PROTEIN IS LOCALIZED AND BOUND TO LYSOSOMAL MEMBRANE. THERE ARE CURRENTLY NO TREATMENTS AVAILABLE FOR TREATMENT OF MPS IIIC. INDIVIDUALS AFFECTED BY MPS IIIC ARE MANAGED WITH SUPPORTIVE CARE, CONSULTATION WITH MEDICAL PROFESSIONALS FROM MULTIPLE DISCIPLINES, PHYSICAL THERAPY, AND PHARMACOLOGICAL INTERVENTIONS TO ALLEVIATE SYMPTOMS. GENE THERAPY REPRESENTS A REASONABLE AND PROMISING APPROACH TO PROVIDE A MEANINGFUL AND LONG-TERM THERAPEUTIC BENEFIT FOR THIS POPULATION IN THE NEAR FUTURE. WE HAD A POSITIVE INTERACTION WITH THE FDA AND GOT GUIDANCE FOR MOVING OUR PROGRAM INTO THE CLINICS. IN PREPARATION FOR THE INTERVENTIONAL STUDY THE RECOMMENDATION WAS FOR A THOROUGH NATURAL HISTORY STUDY (NHS) IN THE AVAILABLE PATIENT POPULATION WITH A BROAD NET TO CAPTURE ENDPOINTS THAT ARE MOST LIKELY TO PREDICT THE CLINICAL BENEFITS IN INDIVIDUALS. SINCE THE NUMBER OF DIAGNOSED PATIENTS IS SMALL, INDIVIDUALS COULD POTENTIALLY SERVE AS THEIR OWN CONTROL AT THE TIME OF INTERVENTION, WHERE THE PATIENT WILL RECEIVE THE GENE THERAPY. MOST OF THE WORK WILL BE EXECUTED BY CONTRACTED SERVICE PROVIDERS. THE CLINICAL TRIAL ITSELF WILL BE CONDUCTED AT UT SOUTHWESTERN MEDICAL CENTER, DALLAS, TX. THE CLINICAL PROTOCOL WAS DESIGNED BY THE TEAM AT PHOENIX NEST WITH HELP OF KOLS AND CLINICAL EXPERTS IN THE MPS III FIELD. WE HAVE ENGAGED EXPERT THIRD PARTY CLINICAL SERVICE PROVIDERS TO HELP WITH THE EXECUTION, MONITORING AND DATA COLLECTION. CRUCIAL DATA COLLECTED FROM THE PATIENTS ON THIS STUDY WILL HELP US DEVELOP CLINICAL OUTCOMES THAT WILL BE TOOLS FOR MEASURING THE EFFICACY OF OUR AAV9 GENE THERAPY. THIS TRIAL WILL PUT A STEP CLOSER TO EXECUTING THE PIVOTAL TRIAL TO ASSESS THE EFFICACY OF OUR EXPERIMENTAL THERAPY AND ITS COMMERCIALIZATION.

881675.00

0.00

0.00

2022-09-19

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Department of Health and Human Services

DEVELOPMENT OF GENE REPLACEMENT THERAPY FOR SANFILIPPO SYNDROME TYPE C - PROJECT SUMMARY/ABSTRACT LYSOSOMAL STORAGE DISEASES (LSD) ARE RARE INHERITED METABOLIC DISORDERS CAUSED BY DEFECTS IN THE CELLULAR CATABOLIC SYSTEM. MUCOPOLYSACCHARIDOSIS TYPE IIIC (MPS IIIC OR SANFILIPPO DISEASE TYPE C) IS ONE SUCH LSD THAT IS CAUSED BY DEFICIENCY OF THE ENZYME HEPARAN SULFATE ACETYL COA: -GLUCOSAMINIDE N-ACETYLTRANSFERASE, (HGSNAT) ESSENTIAL FOR DEGRADATION OF HEPARAN SULFATE, A REPEATING CARBOHYDRATE GENERALLY FOUND ATTACHED TO PROTEOGLYCANS. THIS DISEASE CAUSES ACCUMULATION OF HEPARAN SULFATE RESULTING IN A PROGRESSIVE AND SEVERE NEUROLOGICAL DETERIORATION EARLY IN LIFE WITH LITTLE SOMATIC FEATURES. THE SYMPTOMS IN PATIENTS WITH MPS IIIC MAY PRESENT AT AN AVERAGE AGE OF 3.5 YEARS OF AGE WITH PSYCHOMOTOR DEVELOPMENTAL DELAYS AND BEHAVIORAL PROBLEMS. BEFORE THE AGE OF 15 YEARS VERBAL COMMUNICATION IS OFTEN LOST IN PATIENTS WITH MPS IIIC. MOST LOSE THE ABILITY TO WALK BETWEEN THE 20 AND 30 YEARS OF AGE. THE CONDITION IS FATAL BY AN AVERAGE AGE OF 34 YEARS (RANGE, 25-48). ENZYME REPLACEMENT THERAPIES ARE NOT AN OPTION SINCE THE PROTEIN IS LOCALIZED AND BOUND TO LYSOSOMAL MEMBRANE. THERE ARE CURRENTLY NO TREATMENTS AVAILABLE FOR TREATMENT OF MPS IIIC. INDIVIDUALS AFFECTED BY MPS IIIC ARE MANAGED WITH SUPPORTIVE CARE, CONSULTATION WITH MEDICAL PROFESSIONALS FROM MULTIPLE DISCIPLINES, PHYSICAL THERAPY, AND PHARMACOLOGICAL INTERVENTIONS TO ALLEVIATE SYMPTOMS. GENE THERAPY REPRESENTS A REASONABLE AND PROMISING APPROACH TO PROVIDE A MEANINGFUL AND LONG-TERM THERAPEUTIC BENEFIT FOR THIS POPULATION IN THE NEAR FUTURE. THE GOAL OF THIS SBIR PROJECT IS TO COMPLETE TISSUE LEVEL ASSESSMENTS IN THE DISEASE MODEL, SCALE UP MANUFACTURING AND ESTABLISH SAFETY ON A GLP REGULATED STUDY IN RATS. THESE ACTIVITIES WILL HELP BUILD A ROBUST BRIEFING PACKAGE FOR IND-FILING FOR OUR GENE THERAPY PRODUCT, JLK-247. THE PROGRESS WILL HELP US NAVIGATE THE “VALLEY OF DEATH” BY ESTABLISHING MILESTONES AND MAKING GO/NO-GO DECISIONS. THE SAFE THERAPEUTIC DOSE-RANGE ESTABLISHED WILL HELP US EXTRAPOLATE AND TRANSLATE THERAPEUTIC DOSES TO CLINICAL PHASE I TRIALS. THIS PROPOSAL LEVERAGES THE SCIENTIFIC EXPERTISE IN GENE THERAPY PRECLINICAL DEVELOPMENT AND REGULATORY EXPERIENCE OF SRIKANTH SINGAMSETTY, PHD (PHOENIX NEST, INC.) FOR THE PRODUCT DEVELOPMENT. JILL WOOD, BS (PHOENIX NEST, INC.), WILL MANAGE THE PROJECT FINANCES AND PERSONNEL RESPONSIBLE. PROFESSOR STEVEN GRAY, PHD (THE UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER, DALLAS, TX) IS OUR GENE THERAPY SUBJECT MATTER EXPERT. SUCCESSFUL COMPLETION OF THIS PROJECT WILL HELP WITH INITIATING CLINICAL TRIAL DOSING, A FIRST STEP TOWARDS A LONG-TERM THERAPY FOR MPSIIC, A DREADFUL AND FATAL PEDIATRIC DISEASE.

2990505.00

0.00

0.00

2021-09-21

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Department of Health and Human Services

EVALUATION OF CLINICAL OUTCOME ASSESSMENT (COA) AND POTENTIAL BIOMARKERS TO FACILITATE INTERVENTIONALTRIAL FOR MUCOPOLYSACCHARIDOSIS IIID PATIENTS - PROJECT SUMMARY SANFILIPPO DISEASE (MUCOPOLYSACCHARIDOSIS TYPE III; MPS III) IS A DEVASTATING NEURODEGENERATIVE LYSOSOMAL STORAGE DISORDER OF CHILDHOOD WHOSE PATHOLOGIC FEATURES ARE NEUROLOGIC: SLOWING OF DEVELOPMENT, SEVERE BEHAVIORAL PROBLEMS, PROGRESSIVE COGNITIVE DECLINE, DEMENTIA, AND DECLINE IN MOTOR SKILLS LEADING TO IMMOBILITY, UNRESPONSIVENESS, AND DEATH. WE HAVE FOCUSED ON MPS IIID CAUSED BY A DEFICIENCY OF ALPHA-N- ACETYLGLUCOSAMINE-6-SULFATASE (GNS). BECAUSE MPS IIID IS RARE (1 IN A MILLION) AND AFFECTS THE BRAIN (WHICH IS DIFFICULT TO TREAT) NO CURE OR TREATMENT IS AVAILABLE AND THERE ARE AT LEAST FIVE PATIENTS IN THE USA TO OUR KNOWLEDGE. SANFILIPPO PATIENT ORGANIZATIONS HAVE 19 ADDITIONAL CASES REGISTERED AROUND THE WORLD (SEE LETTER OF SUPPORT). DR. PATRICIA DICKSON AND DR. TSUI-FEN CHOU (LABIOMED) HAVE DEVELOPED AN ENZYME REPLACEMENT TREATMENT (ERT) FOR MPS IIID. OUR STRATEGY PROPOSES TO DELIVER RECOMBINANT HUMAN ALPHA-N-ACETYLGLUCOSAMINE-6-SULFATASE (RHGNS) VIA INTRACEREBROVENTRICULAR INFUSION TO EFFECTIVELY TREAT THE UNDERLYING CAUSES OF THE NEUROLOGIC SYMPTOMS THAT DOMINATE MPS III PATHOLOGY. ERTS CAN HAVE A DRAMATIC EFFECT ON THE QUALITY OF LIFE AND PATIENT DEVELOPMENT ESPECIALLY WHEN ADMINISTERED EARLY IN DEVELOPMENTS. THERE ARE SEVERAL EXAMPLES OF SUCCESSFULLY COMMERCIALIZED ERT’S (E.G. LARONIDASE (MPS I), IDURSULFASE (MPS II), ETC.) AND BIOMARIN RECENTLY RECEIVED APPROVAL FOR AN ERT FOR A FORM OF BATTEN DISEASE, CLN2. OTHER ERTS FOR MPS I, II, AND IIIB ARE IN PHASE I TRIALS. BOTH THE FDA AND INVESTORS ARE FAMILIAR WITH ERT AND ITS COMMERCIALIZATION PATH, WHICH WILL GREATLY INCREASE THE CHANCES OF REACHING A CLINICAL TRIAL. LABIOMED HAS FILED A US PATENT ON RHGNS AND PHOENIX NEST, INC. HAS LICENSED IT. OUR PIVOTAL NONCLINICAL AND MANUFACTURING PLANS ARE ON TRACK. WE HAD A POSITIVE INTERACTION WITH THE FDA AND GOT GUIDANCE FOR MOVING OUR PROGRAM INTO THE CLINICS. IN PREPARATION FOR THE INTERVENTIONAL STUDY THE RECOMMENDATION WAS FOR A THOROUGH NATURAL HISTORY STUDY (NHS) IN THE AVAILABLE PATIENT POPULATION WITH A BROAD NET TO CAPTURE ENDPOINTS THAT ARE MOST LIKELY TO PREDICT THE CLINICAL BENEFITS IN EACH INDIVIDUAL. SINCE THE NUMBER OF DIAGNOSED PATIENTS IS SMALL THE COLLECTED DATA FORM EACH INDIVIDUAL WOULD BE THEIR OWN CONTROL AT THE TIME OF INTERVENTION, WHERE THE PATIENT WILL RECEIVE THE RECOMBINANT ENZYME. MOST OF THE WORK WILL BE EXECUTED BY CONTRACTED SERVICE PROVIDERS. THE CLINICAL TRIAL ITSELF WILL BE CONDUCTED AT NYU UNDER THE GUIDANCE OF DR. LAU. SHE HAS EXPERIENCE WITH OVER 10 CLINICAL TRIALS INTERVENTIONAL AND OBSERVATIONAL. THE CLINICAL PROTOCOL WAS DESIGNED BY THE TEAM AT PHOENIX NEST WITH HELP OF KOLS AND CLINICAL EXPERTS IN THE MPS III FIELD. WE HAVE ENGAGED EXPERT THIRD PARTY CLINICAL SERVICE PROVIDERS TO HELP WITH THE EXECUTION, MONITORING AND DATA COLLECTION. CRUCIAL DATA COLLECTED FROM THE PATIENTS ON THIS STUDY WILL HELP US DEVELOP CLINICAL OUTCOMES THAT WILL BE TOOLS FOR MEASURING THE EFFICACY OF OUR RHGNS THERAPY AND WILL BE PRIMARY ENDPOINTS ON THE PIVOTAL STUDY. THE SUCCESS ON THIS TRIAL WILL PUT A STEP CLOSER TO EXECUTING THE PIVOTAL TRIAL TO ASSESS THE EFFICACY OF OUR EXPERIMENTAL THERAPY AND ITS COMMERCIALIZATION.

3519934.00

0.00

0.00

2020-05-02

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Small Business Administration

TO AID SMALL BUSINESSES IN MAINTAINING WORK FORCE DURING COVID-19 PANDEMIC.

0.00

63567.00

63567.00

2017-09-13

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Department of Health and Human Services

STRUCTURE-BASED OPTIMIZATION OF A NOVEL PHARMACOLOGICAL CHAPERONE THERAPY FOR MPSIIIC

224873.00

0.00

0.00