Investment

Opportunities in

Pediatric Health

September 2016

Leading Change for Better Pediatric Care

Leading Pediatric Academic Medical Center

Patient diversity

African-American 6%

Asian 6%

Latino 62%

Caucasian 25%

Native American 0.06%

Other 1%

Capacity

357 active beds

5,500 employees

802 medical staff

Annual Statistics

16,000 inpatients admissions

350,000 outpatient visits

>110,000 individual patients

78,000 Emergency Department visits

2,300 patients transported by

helicopter, learjet and ambulance

U.S. News & World report

ranked No.1 Children’s Hospital in California

Interdisciplinary Centers in Areas Critical to Pediatric Health

Center for Cancer and Blood Diseases One of the nation’s largest pediatric hematology-

oncology programs. Includes the only federally

funded basic and translational research programs

studying sickle cell disease on the west coast. Large,

integrated clinical trials program(167 active studies).

Home to 3 international phase I clinical trial

consortia. 5,200 unique patients.

Center for Fetal and Neonatal Medicine Treats the most critically ill preemies, newborns and

infants in Southern California.

Center for Endocrinology, Diabetes and

Metabolism Provides care to nearly 2,000 children with diabetes

and more than 5,000 children with other endocrine

disorders.

Children’s Orthopaedic Center One of the nation’s most comprehensive

programs seeing 23,500 patients and

performing 1,880 orthopaedic surgeries

annually.

Vision Center The largest pediatric ophthalmology

program in the U.S.

Cardiac Center One of the busiest cardiac centers in the

country, and serves as a global patient

reference center.

Neurosciences Center Provides a comprehensive continuum of

care bringing together services from

neurology, neurosurgery, psychiatry,

neuro-psychology, neuro-radiology, physical

therapy, occupational therapy, dietetics and

social work

Complete range of pediatric specialties in 5

departments and 32 divisions all devoted to children

The Saban Research Institute

The Saban Research Institute at Children’s

Hospital Los Angeles comprises basic,

translational and clinical research and is one

of the few freestanding pediatric hospitals in

the country where scientific inquiry is

combined with clinical care devoted

exclusively to children.

Among U.S. children’s hospitals, ranks

eighth for NIH funding

• $32.7 million in NIH funding (FY 2015)

• $74.8 million total research funding (FY

2015)

The Institute includes 198,000 square feet of

wet and dry lab space

• Core facilities fully equipped with state of

the art instrumentation

• Cutting edge biomedical informatics and

imaging technologies.

Home to leading international pediatric

cancer consortia: New Approaches to

Neuroblastoma Therapy (NANT),

Therapeutic Advances in Childhood

Leukemia/Lymphoma (TACL), Pediatric

Blood and Marrow Transplant Consortium

(PBMTC)

• 200 current clinical trials

• 1,625 square foot Clinical Trials Unit

The Center for Innovation raises standards in pediatric care through the

development, commercialization and implementation of novel products and

services in partnership with industry, venture capitalists, investors,

entrepreneurs and philanthropists.

The Center for Innovation operates the Consortium for Technology and

Innovation in Pediatrics (CTIP) a pediatric health accelerator serving

innovators and start-ups nationwide and funded in part by the US Food and

Drug Administration.

We evaluate over 80 technologies each year that seek to significantly

improve pediatric standards of care while offering broader market

opportunities.

The Center for Innovation

OUR PROCESS We have a deep understanding of

and access to pediatric markets

Pediatric clinical needs

Requirements for translating medical technologies to the bedside

Regulatory strategies needed to bring pediatric technologies to market

Reimbursement strategies

Drivers of clinical adoption and appropriate commercial strategies

Unmet need validation

Business model/case

Product development

Regulatory approval

Clinical acceptance

Reimbursement and

implementation

OUR ADVANTAGES We have access to unique resources to

support development & commercialization

A clinical advisory network in all areas of pediatric specialty with KOLs and domain area experts

Clinical and translational research collaborators

Hospital administrators and leaders

7 FDA-funded sites accelerating the commercialization of pediatric medical devices

150+ children’s hospitals participating in the International Society for Pediatric Innovation (iSPI)

Avexegen

A preclinical stage company developing Neuregulin-4 (NRG-4), a first-in-class oral therapy consisting of a naturally occurring peptide that functions to promote gut mucosal healing in patients suffering from Inflammatory Bowel Disease (IBD), as well as the orphan neonate condition necrotizing enterocolitis (NEC). Given its novel mechanism of action (MOA), NRG-4 is well positioned to make significant inroads into the IBD and NEC market segments, which combined are projected to exceed $10 billion in total market size by 2021.

Nanovalent

A preclinical stage oncology company developing a targeted drug delivery platform that uses polymerized liposomal nanoparticles that are highly robust, customizable, stable, cheap, and easy to manufacture. Nanovalent will pursue orphan designation for expedited commercialization of its first therapeutic candidate in Ewing Sarcoma, a rare pediatric cancer.

CerRx

A clinical stage oncology company seeking to improve the lives of cancer patients by bringing to the marketplace unique therapeutics that target the ceramide pathway. CerRx is developing a portfolio of lead compounds including an oral formulation of fenretinide to treat pediatric cancers, including neuroblastoma.

Therapeutics

TheraPEDS

A preclinical stage drug delivery company developing proprietary mucoadhesive oral drug formulations combined with taste masking chemistry to reformulate existing drugs for improved compliance and dosing in pediatric (and elderly) patients. The pediatric medicines market is projected to reach over $100B by 2019.

Hillhurst Biopharmaceuticals

A preclinical stage company developing HBI-002, an oral therapeutic targeting the cytoprotective Heme Oxygenase metabolic pathway for patients suffering from conditions associated with inflammation and apoptosis, such as sickle cell disease, transplantation, and cerebral injury. This therapeutic enables the use of a gasotransmitter for which seven completed Phase 1 and 2 clinical studies have demonstrated safety and tolerability, albeit with other delivery modalities that are associated with toxicity and other issues. These indications combined represent a greater than $6.8B market opportunity.

Therapeutics

Antibody-Dependent NK Exosome Therapeutic for Neuroblastoma, Leukemia and Other Cancers*

Antibody-Dependent Exosome Cytotoxicity (ADEC) represents a completely new approach to cancer immunotherapy with applicability to a wide array of malignancies in children and adults. It’s advantages include an allogeneic source, expanded from readily available PBMCs, a distinct mechanism of action from cytotoxic chemotherapies and targeted therapies, synergy with antibody therapy and their small size to penetrate sanctuary sites. *Available for licensing from CHLA

AM80, An oral therapy to reduce incidence of infections and related mortality in patients with chemotherapy-induced neutropenia when used in combination with GCSF*

For 20 years, G-CSF (Neupogen®, filgrastim) daily injection has been an effective treatment for increasing numbers of neutrophils and white blood count. However, despite this therapy, chemotherapy induced neutropenia continues to be a problem with infections, febrile neutropenia and associated deaths.

Am80 is an oral therapy that increases the bactericidal activity of neutrophils. Increased bactericidal activity reduces the risk of infection. Patients have a therapy that reduces their risk of infection and associated mortality while on chemotherapy. *Available for licensing from CHLA

Antibody Blockade of Integrin Alpha6 for the Treatment of Relapse Leukemia and Other Cancers*

Current therapies address tumor shrinkage, but not the tumor microenvironment in metastatic disease (bone marrow). Relapse of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) remain a significant problem with a high rate of bone marrow metastasis. Invasive breast cancer remains a significant problem (200,000 new cases diagnosed per year) in the US with a 20% mortality (relapse) rate. Bone marrow metastasis remains a problem for all 3.

P5G10 – A first in class blocking antibody to integrin a6 prevents tumor cell adhesion to bone marrow stroma. Reduced adhesion to bone marrow sensitizes tumor cells to chemotherapy. Patients have a therapy that reduces their risk of relapse disease and mortality associated with metastatic disease. *Available for licensing from CHLA

Therapeutics

Tourmaline Medical

A device company commercializing the STARFISH Luminal Expansion System (LXS), an innovative, patented device solution for short bowel syndrome (SBS). SBS is a devastating condition with no current cure that afflicts both pediatrics and adults. Currently, patient care and associated drugs to facilitate this care can impart costs of over $1M per year per patient. Unique regulatory pathways will allow Tourmaline to gain rapid and relatively low cost market access with a unique value proposition for the payor community. Patient care centers are highly concentrated allowing for an efficient commercial model that will drive rapid revenue growth and break-even. The STARFISH LXS System has been proven in small and large animals and published in over a dozen peer reviewed publications to date. The total addressable market for this device is $3B.

Epipace*

An injectable micropacemaker for fetuses, and for children and adults, to control abnormal heart rhythms. Epipace is developing an alternative approach to the new leadless pacemakers by implanting its micropacemaker on the exterior of the heart, or epicardially. This safer minimally-invasive approach enables for the first time, the pacing of fetuses, and offers significant benefits for children and adults. The addressable market for replacing epicardial systems and fetal pacing is $800M. The total pacemaker market is close to $10B.

*Available for licensing from CHLA

LifeFlow by 410 Medical, A Rapid Infusion System

Pediatric sepsis is responsible for over 75,000 hospitalizations, 10,000 deaths, and $4.8 billion in cost in the US each year. Multiple studies have shown that implementation of ACCM/PALS guidelines early in the course of sepsis, particularly antibiotics and aggressive fluid resuscitation, saves lives. Unfortunately, fluid delivery guidelines are rarely achieved in clinical practice. Numerous studies, and the experience of the Children’s Hospital Associated sepsis collaborative, have shown that barriers to fluid delivery remain one of the most common reasons for ineffective resuscitation in pediatric sepsis.The LifeFlow™ rapid infuser is a single-use, hand-operated, disposable device that allows fast and efficient fluid delivery to critically ill patients with shock and sepsis. Designed to help overcome common barriers to fluid resuscitation, the device is simple and intuitive, minimizes provider fatigue, and frees providers to address other patient care issues simultaneously. This solution is particularly useful in pre-hospital and emergency department settings where speed is important and personnel resources may be limited, and is also well suited for inpatient, critical care, and operating room environments.

Therapeutic Devices

NeoLight

Jaundice is one of the most common and deadliest afflictions facing babies. It occurs in 6 out of 10 newborn babies globally and affects over 2.4 million infants in the US alone. Jaundice phototherapy devices are a sub-set of the neonatal equipment market, which is projected to reach $4B by 2019. NeoLight has developed an innovative light-channeling technique which produces high-intensity light that will measurably reduce treatment time and eliminate complications associated with current phototherapy equipment on the market. This technology can potentially reduce treatment time by 40% thus saving the hospital $8000 per procedure.

Hypothermia Devices

Therapeutic hypothermia has been used as a treatment of choice for the management of inflammatory responses associated with soft tissue injuries and spinal cord injury, to ameliorate the effects of traumatic brain injuries and strokes, to improve neurologic outcomes after cardiac arrest and neonatal hypoxic-ischemic encephalopathy. However, existing cooling devices are not designed to address the requirements of stability, transportability and efficiency for treatment. Hypothermia Devices has developed flexible and ergonomic cooling/heating pads to be placed in contact with the skin and can be electronically controlled in order to produce rapid and efficient temperature regulation. The total addressable global market for this technology is $3.9B.

Polyvascular, A Stent Mounted Heart Valve

Congenital heart defects are the most common of all birth defects and remain amongst the leading causes of infant mortality in the developed world. Multiple types of congenital heart defects require valve replacement surgery. Pulmonary valves are usually replaced using cadaveric homograft valves, since mechanical valves have increased risk of thrombosis in the pulmonary position. Homograft pulmonary valves are not commonly available, particularly in sizes suitable for use in children. Moreover, these valves do not grow with the patient, becoming too small as the child grows. All these factors result in the need for repeated valve replacement surgeries. Therefore, there is an urgent need of solutions to limit the frequency of repeated valve replacement surgeries for these children. Polyvascular is developing an innovative stent mounted heart valve that does not contain any biologic tissue, limiting any immune sensitization and that can be delivered via cardiac catheterization, eliminating the need for open heart surgery. This heart valve is also expandable over time and will follow the child's growth, eliminating the need for multiple surgeries.

Therapeutic Devices

TESI, A point of care cell processing device for treating small bowel syndrome*

Necrotizing enterocolitis (NEC) is a gastrointestinal disease that affects premature infants. The current standard of care requires surgical removal of necrotized tissue, which requires life-long management of nutritional insufficiencies (transplant and/or arterial line feeding) associated with short bowel syndrome.

TESI is a device-based kit for processing salvaged small intestinal tissue during surgery to create bowel segments for implantation. A subset of cells is seeded on a biodegradable scaffold, which is implanted into the patient resulting in autologous tissue-engineered intestines. A proof of concept has been obtained in non-clinical models and pediatric scalability is being pursued in non-clinical studies. *Available for licensing from CHLA

Lully Sleep

According to the National Sleep Foundation, 36 Million children experience sleep disorders on a weekly basis. Night terrors are one of the leading causes of children not sleeping well at young ages. It is estimated that nearly 25% of children age 2-12 will experience at least one night terror. The Lully Sleep Guardian is designed to bring this clinically validated, effective technique to the hands of parents. Based on a child's specific sleep patterns the Lully system uses vibrations, prior to a transition into unhealthy deep sleep. The Sleep Guardian is based on a tried-and-true technique called scheduled awakenings. The Sleep Guardian's once-a-night vibrations partially wake your child, bringing him or her out of the night terror causing sleep patterns. Because children have high sleep pressure, they almost immediately return to deep sleep. This interrupts the unhealthy sleep cycle and prevents night terrors. Clinical studies have shown that this method has reduced 90% of night terrors.

Therapeutic Devices

Vaximmune

A device company focused on preventing the transmission of the most common infectious agents from mothers to their babies during labor, Group B Strep (GBS), Ureaplasma (UP) and E. Coli by developing a proprietary, disposable culture device that allows for the collection, stabilization and concentration of the relevant sample enabling the use of a rapid diagnostic to read out the test result. The kit contains a sample collection swab, a disposable culture device and a lateral flow assay. The sample can be collected by the patient or the physician and the test can be performed by the physician or the laboratory. This product supports a $700MM market opportunity in the US and a combined opportunity of $2.8BB in the developed world.

MIND Dx

Neuropsychiatric disorders are devastating illnesses where approximately one-fourth of adults have a mental illness and nearly half will develop one mental illness during their lifetime. The economic cost of care for mental illnesses is roughly $100 billion in year 2003 in the US and $193 billion are lost in the in earnings and wages. The gold standard for diagnoses is clinical interviews and history, resulting in misdiagnoses as high as 50% at initial presentation and time to proper diagnosis can take years. MindDx has developed machine-learning based diagnostic algorithms from MR images of the brain to diagnose neuropsychiatric disorders with high sensitivity and specificity. The market opportunity for ADHD is $800M and $40B for ADHD, bipolar, schizophrenia, depression and other psychiatric disorders combined.

Deton Corp

Deton is a company committed to eliminating the need for collection of sputum (the phlegm that resides deep in the airway) as a sample for the diagnosis of lower respiratory tract infections. Sputum sampling has a 50% failure rate resulting from the inability of the patient to produce a sample or sample contamination from the mouth. For these patients, results cannot be provided, leading to empirical treatment which, in turn, leads to the proliferation of antibiotic resistant bacteria. Failure rates are highest for pediatric and geriatric patients. A cough sample is a simple and effective replacement for sputum. There are approximately 10M sputum samples requested annually in the developed world, supporting a $1B market cap.

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Diagnostic Devices

CrestaBio

There are approximately 1,200,000 people living with or in remission from blood cancer in the US, with an estimated 150,000 new cases diagnosed annually. Minimal residual disease (MRD), defined as the number of cancerous cells found in the blood or bone marrow during or after treatment, is used to determine if a patient is free of cancer. MRD is used to monitor the effectiveness of therapy, determine when a patient is in remission and for early detection of relapse. When MRD is undetected and therefore untreated, it is the primary cause of relapse. Since the limit of detection of the diagnostic technology defines when a patient is free of cancer, improving the limit of detection directly results in an improvement in overall survival. Today flow cytometry has a 1 in 10,000 limit of detection and PCR can detect 1 in 100,000 for some subsets of leukemia. CrestaBio has developed a technology that combines the benefits of both methods into one test, while improving the sensitivity by three orders of magnitude. The technology allows for the rapid and cost-effective detection followed by capture and genetic characterization of 1 in 100,000,000 cells.

AZA Technology, A Portable Ammonemia Detector (PAD)

Hyperammonemia is a metabolic condition characterized by elevated levels of ammonia in the blood. Increased entry of ammonia to the brain is a primary cause of neurologic disorders, such as congenital deficiencies of urea cycle enzymes, hepatic encephalopathies, Reye syndrome, several other metabolic disorders, and some toxic encephalopathies. Early diagnosis and improved monitoring are important because hyperammonemia can be treated by effective medical interventions. In children, 400 infants are born annually in the U.S. with mutations in one of 44 genes that affect several metabolic pathways, and 400,000 infants who are born before 37 weeks of gestation are at risk for temporarily compromised urea cycles due to immature liver function. The current standard of practice requires at least 3 ml of blood that must be transported to a central laboratory for immediate processing. The child must endure intravenous puncture for blood near a central laboratory. Aza Technology, Inc has developed an innovative portable device that measures ammonia levels in blood obtained by finger or heel stick. This Portable Ammonemia Detector (PAD) uses a novel technology that adapts an inexpensive commercially available ammonia detector for use with blood. PAD measures blood ammonia within seconds from 0.01 ml (10 µL) of blood obtained by a simple finger or heel stick. PAD will allow screening for early diagnosis and thus save lives and protect cognitive function in many children. In addition, subsequent management of these children will improve when PAD is deployed to the bedside and outpatient clinics.

Diagnostic Devices

Hangar Hustle, A Game-Based Cognitive Assessment Tool

Attention deficits affect millions of people in the US; the percent of children 3-17 years of age with ADHD is 9.5% and the prevalence of ADHD in the U.S. adult population is approximately 4.4%. Beyond the personal costs to relationships, education, employment, is the economic cost to the nation in accidents, lost productivity, and additional healthcare services. Expeditious assessment means earlier access to treatment that will reduce the personal and economic impact of cognitive impairments. Blue Marble has developed an innovative game-based assessment tool designed to measure various forms of attention, working memory and executive functions in the course of video game play. This tool is intended to replace current paper-based or computerized assessment that are cumbersome, time consuming and non-engaging for the patient. Using this new technology will reduce the per-patient cost for assessment and will therefore stretch resources farther to serve more people.

Medulloblastoma Diagnostic Assay*

Medulloblastoma is the most common malignant pediatric brain tumor. Approximately 30% of patients remain incurable and current radiation therapy containing treatment protocols cause significant adverse long-term neurocognitive effects and endocrine dysfunction. In order to increase survival rates and reduce adverse sequelae, there is a need of early detection methods and refined patient stratification. This 31-gene diagnostic assay allows accurate identification of medulloblastoma subgroups and examines the expression of inflammation-related genes with respect to each subgroup. Treatment for medulloblastoma is currently conducted with a multi-modality approach (surgery, radiation therapy and chemotherapy) and the modality is heavily dependent on tumor staging and risk group categorization. The molecular classification of medulloblastoma using this platform will greatly improve current clinical risk stratification and therefore help with choosing the more efficient treatment for the patient. *Available for licensing from CHLA

Neuroblastoma Prognostic Assay*

Neuroblastoma, an embryonal tumor of the peripheral sympathetic nervous system, is one of the most common solid tumors in children, with approximately 45% of patients presenting with metastatic disease at diagnosis. Only 45% of those with high-risk metastatic neuroblastoma are cured with current treatments that include intensive chemotherapy and immunotherapy. Having a new means to identify individuals at extremely high-risk for disease progression and to assess response to therapy would be valuable for disease management.This 14-gene prognostic assay identifies high and ultra-high risk groups and a highly sensitive and specific 5-gene response assay to quantify circulating and bone marrow tumor cells in patients with neuroblastoma. These tests represent an integrated disease management platform that may be particularly useful in adapting and evaluating treatments, and in predicting outcome in children with neuroblastoma. A novel method for recovering high quality RNA from viable fresh and frozen specimens was established for use in these tests. This platform, with modifications, may also be useful in adults with cancer. *Available for licensing from CHLA

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Diagnostic Devices

Next Generation Cystic Fibrosis Diagnostic Tool*

Cystic fibrosis (CF) is a life-threatening genetic disease that primarily affects the lungs and digestive system. An estimated 30,000 children and adults in the United States (70,000 worldwide) have CF. CF is caused by mutations in a gene that produces a protein, called CFTR. The CFTR protein controls the flow of salt and water in and out of the cells of organs like the lungs and pancreas. CF is usually diagnosed by conducting a QPIT sweat test, which measures the amount of chloride in a person’s sweat. However, this test is only valid for patient with severe disease and does not take into account the spectrum of pathologies associated with variable degrees of CFTR dysfunction. This is particularly the case with adolescents, adults, and asymptomatic infants identified as being at risk for CF and presenting with milder symptoms of the disease. This new method is based on beta adrenergically induced sweat secretion. The rate of b-adrenergically stimulated sweating is used of diagnose CF disease and reflect the probability of underlying genotypes. B-adrenergic sweat secretion rates are assessed with an evaporimeter to provide an accurate, reliable and practical real-time assay over a range of CFTR functional activity in vivo. This new test will be a very valuable asset to diagnose CF disease and a possible biomarker for CF drug efficacy testing. *Available for licensing from CHLA

Diagnostic Devices

Prospiria, an optoaccountic guidance system for endotracheal tube placement

Over 25 million endotracheal tubes (ETs) are placed in patients each year. However, there is not an expedient solution to identify the 20 to 50 percent of ETs that are initially malpositioned, or that move during intubation. Incorrectly positioned ETs pose serious risk to patients, especially infants.

Prospiria has developed a patent-pending technology that uses optoacoustics (ultrasound signals generated by pulsed laser light) to rapidly, precisely, and noninvasively identify the position of endotracheal tubes that have been placed within the trachea. This technology will increase effectiveness and reduce complications by allowing clinicians to quickly confirm and monitor both initial and subsequent ET placement.

Inceptio Medical Technologies

An estimated, 2.5 million peripherally inserted central catheters (PICC) line placements are performed in the U.S. annually. Although a common procedure, PICC placements are particularly difficult in neonates because their arteries have such small diameters and low pressure for palpitation. Complications that can arise from misplacement include catheter induced perforation, thrombosis, and catheter induced arrhythmia. Inceptio has developed a novel technology for precisely locating the distal tip of a cardiac catheter. The system provides clinicians with real-time visualization during the procedure which greatly increasing the accuracy of placement and reduces complications. The U.S. PICC market is project to reach over $583M by 2017.

Lifebubble, an Umbilical Catheter Protective Device

Critically ill neonates in the neonatal intensive care unit (NICU) receive umbilical catheters (UCs) because the umbilical vein and artery in the umbilical cord are an easy and painless way to access the central circulation system. Many of these babies are premature and have immature immune systems, leaving them susceptible to bloodstream infections that are attributable to the presence of the umbilical catheter. The rate of UC central-line associated bloodstream infections (CLABSIs) in neonates is at least five times greater than CLABSIs in the adult population. Studies estimate that 5-15% of neonates with UCs develop CLABSIs, with rates highest for infants <1,250 grams. For adult central lines, an occlusive bandage can be applied to the site of catheter insertion that is relatively flat to the patient’s skin to keep it clean and dry. This standard cannot be applied to the UC that exits the vessel perpendicular to the skin through a piece of tissue (umbilical stump) that has a unique shape and that must dry out over the course of a few weeks. LIFEbubble is a innovative device that is tailored to the baby’s unique anatomy. It replaces the non-sterile tape configuration to reduce the migration of bacteria along the skin, protect the umbilical stump-catheter interface from accidental touch, and secure the UC to prevent entry of the non-clean catheter into the bloodstream. LIFEbubble provides a streamlined solution to improve the current standard of care and save the lives of vulnerable infants.

Positioning Devices

NonInvasix

NonInvasix is dedicated to the measurement and monitoring of venous oxygenation, delivering valuable information regarding the adequacy of oxygen perfusion to the brain. A platform technology with a wide clinical utility, the technology will first be applied to the monitoring of neonates in the NICU to predict hypoxic ischemic encephalopathy (HIE). The inability to predict poor perfusion to the brain leads to long term neurological deficit and can be avoided by monitoring brain venous oxygenation. HIE occurs at a rate of 1 per 1,000 live births, costing $900,000 per patient per lifetime and is responsible for $3.6B in annual economic burden.

GI Logic

Millions of adults, children and neonates struggle with digestive disorders, both acute and chronic. Diagnosing digestive problems requires a hospital visit, use of expensive imaging equipment or expensive invasive procedures. Current diagnostics monitor “one point in time episodes”, are expensive and not always immediately available. GI Logic is commercializing “AbStats”, a non-invasive monitoring technology that accurately measures intestinal activity “continuously” or a “snap shot”, providing an immediate measurement of digestive health. AbStats can be used in a hospital setting, a clinic setting or a residential environment. Disposable sensors prevent transfer of bacteria and web services interface provides for easy storing of information in electronic health records.

BestDose*

Dosing errors make up 37% of all preventable medication errors at a cost of $8B to the healthcare system. BestDose is developing an enterprise solution for individualized dosing, addressing a >$1B total market. Its peer-reviewed software platform that combines critical patient data and important drug characteristics to optimize dosing for any drug. Its solutions can be integrated into the EMR as part of the computerized physician ordering process (CPOE) and improve prescriber and pharmacist efficiency by accurately calculating dosages with a button click.

*Available for licensing from CHLA

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Monitoring/CDS Devices

Respivice*

Over 800,000 US children are intubated and receive mechanical ventilation each year. Current strategies to manage mechanical ventilators in children are inefficient, leading to prolonged intubation in 50% of patients and extubation failure in another 10% of patients. Respivce is a stand alone system that provides quantitative measures to (1) optimize ventilator weaning, (2) easily identify the most common reasons for extubation failure and (3) manage noninvasive ventilation after extubation. By avoiding these complications Respivice can reduce healthcare costs by $6.75B in an addressable market of $600M.

*Available for licensing from CHLA

E-Vent, A Digital Ventilator Management Protocol*

Ventilation management is the accumulation of multiple decisions. This type of iterative decision making is amenable to guidance using a medical treatment protocol. Medical treatment protocols facilitate consistent, evidence-based decisions for equivalent patient states and digital protocols improve adherence over paper protocols. E-Vent is an electronic decision support tool translated from an adult ventilator protocol that improves management of pediatric and adult patients with acute hypoxemic respiratory failure.

*Available for licensing from CHLA

4DX

A medical technology company established to commercialize four-dimensional lung imaging technology to provide the spatial and functional information needed to optimize treatment for each patient with conditions such as cystic fibrosis, asthma, emphysema and COPD. 4DX offers the resolution of CT with doses comparable to X-Ray and functional insights of a pulmonary function test in a single platform. The software has been successfully proven and patented and the company is now seeking to raise funds to scale their SAAS business and commercialize custom designed and dedicated scanning hardware for increased patients per hour of operation, reduced radiation dose and enhanced fidelity of scans. Enhanced scanning rates will make each dedicated scanner a valuable additional source of revenue for the analysis service.

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Monitoring/CDS Devices

Click Brain

The current standard of care using pediatric brain MRI for assessment of developmental disorders, neurological trauma and diseases is time-consuming, qualitative, and requires visual assessment of a multitude of 2D images. ClickBrain™ is a reimbursable, quantitative translational clinical decision support software that compares patients’ brain MRI morphometry to healthy normal subjects. ClickBrain™ is intended to significantly impact patient care by 1) increasing efficiency of interpretation and reporting, 2) promoting uniformity of interpretation regardless of radiologist experience level, thus reducing risk of misdiagnosis, and 3) enabling earlier diagnosis and quantitative longitudinal studies for treatment monitoring.

TimeStamp

Cerebrospinal fluid (CSF) flow can de obstructed due to a variety of medical conditions such as infections, meningitis, brain tumors, traumatic injuries etc. Because CSF is made continuously, medical conditions that block its normal flow will result in an over-accumulation of CSF. The resulting pressure of the fluid against brain tissue causes hydrocephalus. The incidence of pediatric hydrocephalous is about 1/1,000 births and early detection of CSF flow obstruction is a key component for proper treatment selection and diminution of downstream effects. Current techniques generate images that lose tissue/CSF contrast over the image capture process thereby complicating the radiologist's ability to identify CSF flow obstruction. TimeStamp is a new MRI-based image acquisition software that allows MRI images to maintain high image contrast for easier and faster interpretation by the radiologist.

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Monitoring/CDS Devices

Qidza

CDC reports that 1 out 6 children have developmental or behavioral problems and 1 in 9 US births are premature (Boyle et al.,2011), costing healthcare $30+ billion annually. Extensive research proves early detection can improve outcomes and reduce costly interventions (Glascoe et al, 1997; WHO, 2007). Yet 52% of pediatricians don’t have time or expertise to screen for developmental issues (American Academy of Pediatrics, 2014). To close the screening gap and offer parents peace of mind, Qidza is a mobile platform that enables parents work seamlessly with their physicians to track their children’s developmental milestones and improve their health and well‐being. Quizda has developed BabyNoggin, a preventative online tool to monitor and track your baby’s brain development from birth to age two. Within minutes you can screen your baby in the comfort of your home. The app offers an easy and fun way to interact with your baby and track his or her development. Data gathered from BabyNogin will improve efficiency of well-child visits to pediatrician and potentially improve clinical outcomes.

Neuromuscular Dynamics

It is well known that both sensory and motor systems contribute to the ability to maintain balance. Sensory inputs are necessary to detect unstable conditions and motor contributions are vital to initiate timely and appropriate responses to counteract these perturbations. The current clinical measures quantify the functional domains of strength and multi-join coordination but they do not quantify the functional domain of dexterity. This new strength-dexterity test device becomes unstable when compressed. Therefore, the maximal level of sustained compression is informative of the maximal ability of the neuromuscular system to stabilize unstable interactions. For the fingers, it is a metric of dexterous manipulation ability. For the legs, it is a metric of leg dexterity and the ability of the leg to regulate dynamical interactions with the ground.

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Monitoring/CDS Devices

LifeSpeed

Lifespeed is a health and wellness platform that allows families, medical professionals, and caretakers to safely store and share data. The tool provides deeper consumer engagement by offering a visually compelling experience to enable users (starting with chronically ill and mums) to manage their health profile efficiently. The consumer-centric platform/dashboard allows users to securely organize, archive and share medical data such as x-rays, data from wearables, lab data, forms and appointments (analogous to Pinterest + Dropbox). Glimpse allows users to invite doctors, caretakers or loved ones to view their data securely. For providers, Lifespeed offers easy-to-use tools for digital content distribution, communication and analysis of patient engagement. The integration of consumer populated data with existing medical records creates an ecosystem that offers comprehensive collaboration between consumers and providers. The platform facilitates a marketplace where stakeholders and third party developers bring apps to the dashboard. The Lifespeed dashboard also provides a medium where healthcare organizations can advertise to targeted communities through the consumer centered portal for health data management.

NICOlette

NICOlette’s first offering is a forthcoming parent-facing, HIPAA-compliant app called NicoBoard. It shares NICU patients’ electronic health record (EHR) data in visually intuitive ways designed to allow parents to quickly grasp the information. It extracts the EHR raw data and synthesizes it into a consumable format for parents so that they can feel competent and confident in participating in their child’s journey to wellness. In addition to parsing out easily digestible EHR data visualizations (such as color-coded graphs of a patient’s oxygen saturation, carbon dioxide levels and apnea episode history, for example), NicoBoard will also furnish parents with curated research relevant to newborn health. The idea is for parents to use the research to make more informed healthcare decisions on behalf of their babies.

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Patient Engagement

Thank You

Leading Change for Better Pediatric Care