ProThera Biologics Awarded $2-Million Fast Track NIH Grant

Company is Developing an Important Biomarker to Assess Outcomes in Neonates with Sepsis or Necrotizing Enterocolitis (NEC) in Support of Its Therapeutic  Development Programs

Providence, R.I., June 19, 2019 – ProThera Biologics, Inc., today announced that it has been awarded a $2.03-million Small Business Innovation Research (SBIR) Fast Track grant by the National Institutes of Health (NIH) to study the potential of a new test to assess prognosis of infants who have, or might develop, sepsis and necrotizing enterocolitis (NEC) and therefore determine which infants may need specific treatments.  

ProThera is developing Inter-alpha Inhibitor Proteins (IAIP) for the treatment of acute inflammatory diseases, including severe community-acquired pneumonia, neuro-inflammatory diseases, stroke and neonatal hypoxic brain injury, and infant diseases necrotizing enterocolitis (NEC) and neonatal sepsis. In these conditions, inflammation plays a key role in the pathology, and there are no effective treatments available. IAIP are naturally occurring proteins that circulate in the blood. Research at ProThera has shown that these proteins play an essential role in human health by controlling inflammation. They exert their anti-inflammatory effects through multiple mechanisms and have the potential to shift the paradigm of drug discovery, which has traditionally focused on “one drug to inhibit one pathway.” To date, no drugs have been able to control severe inflammation. ProThera’s IAIP holds great potential to modulate severe inflammation because it attenuates multiple pathways.  The levels of these anti-inflammatory proteins rapidly decline during acute illnesses, and a replenishment strategy for patients with Inter-alpha Inhibitors is under development.

ProThera is now developing a rapid bedside test, based on levels of Inter-alpha inhibitor proteins (IAIP) in blood. The test assesses the risk of infant patients developing neonatal sepsis (NS) and/or necrotizing enterocolitis (NEC) and will support the Company’s development of IAIP as a protein replacement therapy.  The test will indicate which infants may benefit from treatment with Inter-alpha Inhibitor Proteins and potentially other treatments such as antibiotics.  The test is expected to be simple, user-friendly, portable and suitable for use in the neonatal intensive care unit. NS and NEC are associated with high mortality and morbidity, including adverse neuro-developmental outcomes. Furthermore, both conditions are associated with systemic inflammatory responses, and their initial clinical presentations can be similar, non-specific and subtle, leading to a greater likelihood for misdiagnosis. It is important for clinicians to discern which patients are at risk for progressing to NS or NEC as clinical deterioration in both diseases can progress in a fulminant manner, resulting in shock and death within hours of clinical presentation.  Currently, there are no sensitive and specific tests for early detection of NS and NEC.

ProThera successfully completed a pilot-phase feasibility study for its test in January, 2019 with $166,522 in funding from the NIH. This work was also supported by a matching grant of $45,000 from the Innovate Rhode Island Fund administered by the Rhode Island Science and Technology Advisory Council (STAC).  Based on the success achieved during Phase I, ProThera was issued the second round of SBIR grant funding, bringing the total NIH funding to $2.03M. ProThera will work with key neonatologists, James Padbury, M.D., the Pediatrician-In-Chief and Chief of Neonatal/Perinatal Medicine at Women and Infants Hospital and the Oh–Zopfi Professor of Pediatrics and Perinatal Research at the Warren Alpert School of Medicine at Brown University, and Hala Chaaban, M.D. and Birju Shah, M.D., Assistant Professors in Pediatrics and neonatologists at the University of Oklahoma College of Medicine.

Necrotizing enterocolitis (NEC) is a devastating gastrointestinal disease, occurring predominantly in premature infants. It is characterized by inflammation and necrosis of the bowel wall that can rapidly progress to sepsis, multi-organ failure and death. Despite advances in clinical care, prevalence and mortality from NEC have not changed. Approximately 7- 10% of infants born weighing ≤1500g develop NEC; of those 20-50% do not survive. Sepsis is a devastating complication of prematurity that can develop from NEC or from other bacterial infections. In the early stages, infants can show symptoms which may indicate progression to severe systemic inflammation but there are no biomarker tests to confirm the prognosis.

“Detecting life-threatening, acute diseases at an early stage is a critical need for neonatal patients,” said Dr. Padbury. “So far, extremely promising results have been obtained with IAIP as a biomarker, and we look forward to further collaborating with ProThera.”

“We are very excited to be working with Drs. Padbury, Chaaban and Shah on this critical program,” said Dr. Yow-Pin Lim, co-founder and CEO of ProThera Biologics, who serves as Principal Investigator in this NIH-supported project. “Their extensive knowledge of neonatal diseases is very important for the success of the program.”

Denice Spero, Ph.D., president and chief business officer of ProThera Biologics, noted that “Our first goal in working with our collaborators is to demonstrate the value of the IAIP rapid test in predicting outcomes of infants with sepsis or NEC and then to test the device in clinical studies. IAIP have the advantage of being both a biomarker and a therapeutic. The biomarker aspects support the clinical development of the therapeutic.”  

About ProThera Biologics

ProThera Biologics is a Rhode Island-based biotherapeutics company developing Inter-alpha Inhibitor Proteins (IAIP) as a therapeutic for treating, life-threatening inflammatory conditions. For many diseases the body mounts an excessive, dysregulated inflammatory response causing high morbidity and mortality; current medications are unable to modulate this complicated process. ProThera data demonstrates a solution to this unmet medical need by delivering human plasma proteins (IAIP) which have evolved to control patient’s dysregulated immune responses.

ProThera is developing IAIP as a replacement therapy to treat severe inflammatory diseases where IAIP blood levels decrease as a result of the disease process.  The rationale for protein replacement therapy is common to other proteins derived from blood; replenishing the deficiency significantly improves patient outcomes. The isolation and sale of plasma proteins is a $19B industry. ProThera is positioning itself to partner therapeutic IAIP and the biomarker technology with blood products and pharmaceutical companies.   ProThera has demonstrated that IAIP are a novel biomarker for disease severity and is developing a rapid test diagnostic to measure levels of this protein. The diagnostic will be used in multiple clinical settings and will signal if a patient requires immediate treatment.

In addition to its private and venture funding, ProThera has invested an aggregate of $12.7 million in NIH grant funds toward the development of IAIP both as a therapeutic and as a biomarker to determine disease severity based on the inflammatory state of the patient.  ProThera has developed a purification process for IAIP, elucidated the potential uses of the proteins with its collaborators and has determined how these proteins dampen inflammation. The development of IAIP as a biomarker further supports the body of work developed by the Company.

About Women & Infants Hospital

Women & Infants Hospital of Rhode Island, a Care New England hospital, is one of the nation’s leading specialty hospitals for women and newborns. A major teaching affiliate of The Warren Alpert Medical School of Brown University for obstetrics, gynecology and newborn pediatrics, as well as a number of specialized programs in women’s medicine, Women & Infants is the 9th largest stand-alone obstetrical service in the country and the largest in New England with approximately 8,500 deliveries per year. A Designated Baby-Friendly® USA hospital, U.S. News & World Report 2014-15 Best Children’s Hospital in Neonatology and a 2014 Leapfrog Top Hospital, in 2009 Women & Infants opened what was at the time the country’s largest, single-family room neonatal intensive care unit.