Ron Lowy, Chairman and CEO
Closely-held PharmaJet’s needle-free injection devices have the potential to rapidly improve the delivery of vaccines in emerging markets as well as pharmaceuticals currently under development.
“We are at the forefront of addressing the need for a safe, easy-to-use delivery system for fractional dose inactivated polio vaccine (fIPV), especially in developing countries where a needle and syringe in the field can be difficult to use,” Ron Lowy, chairman and CEO, says in an interview with BioTuesdays.
“Besides offering needle-free polio injections at a significantly lower total cost, our devices use 60% less vaccine for the same or better immune response at a time when there is a shortage of polio vaccine,” he contends. “The fight to eradicate polio has the potential to use 500 million doses of fIPV annually.”
PharmaJet also benefits from having access to more than 120 countries across six continents, excluding the U.S. and Europe, through its marketing partner, Mundipharma, and its commercial organization of more than 3,300 people.
Mr. Lowy explains that PharmaJet’s two needle-free devices – Tropis and Stratis – provide three forms of injections: intradermal, subcutaneous and intramuscular. They also eliminate needle stick injuries, needle reuse, cross contamination, and help reduce sharps waste disposal.
The Tropis device is designed for intradermal injections of vaccines, such as fIPV, and new DNA- and RNA-based pharmaceuticals into the skin layer. Tropis delivers medications and vaccines by means of a narrow, precise fluid stream, which penetrates the skin in about a tenth of a second.
PharmaJet’s Tropis Needleless Injection Device is Safe, Fast and Easy
PharmaJet initiated its Tropis program in 2011 and three years later, received funding from the Bill and Melinda Gates Foundation to optimize the device. In 2016, the company received CE Mark approval for the Tropis device and also inked a multi-year contract valued at up to $10-million-plus with the WHO to produce a stockpile of its devices and disposables.
“Tropis is the only WHO qualified, needle-free delivery system for flPV,” Mr. Lowy points out.
The WHO has endorsed the use of fIPV as an alternative to the oral polio vaccine, which is based on a live virus and has caused “wild” types of polio to spring up in developing countries. In addition, needle and syringe delivery of fIPV is not a viable option in many emerging markets due to the difficulty of administering an intradermal injection with this method.
According to Mr. Lowy, the Tropis device is simple and safe to use, and provides consistent dosing. “We can inoculate kids two-to-three times faster than competing needle and syringe products, use less vaccine, and provide better immunogenicity.”
As part of its fIPV country pipeline, Mundipharma and PharmaJet have identified Ghana, Nepal, Egypt, Nigeria, Myanmar, Tunisia, Tanzania, Chile, Nicaragua, Pakistan, Indonesia, Philippines and Cuba as its best short-term market opportunities.
The WHO estimates the global vaccine market is expected to hit $100-billion by 2024, compared with $37-billion in 2016. There were some 2.5 billion vaccine injections in 2016.
Mr. Lowy says Tropis devices are being used in nearly a dozen clinical studies in the U.S., some of which are assessing dose-sparing vaccines. The company has an FDA Master File in place for Tropis, which will revert to a 510(k) approval, subject to FDA approval of a study drug.
PharmaJet unlocks the benefits of ID, SC and IM deliveries without the hurdles of needle injections
PharmaJet’s second needleless injector is the Stratis, which is designed for deeper subcutaneous and intramuscular injections of analgesics and vaccines for influenza; and measles, mumps and rubella (MMR), for example. The device has 510(k) marketing clearance from the FDA and other jurisdictions around the world and is being considered for the U.S. government’s pandemic flu preparedness program.
Mr. Lowy explains that the Stratis injector has a more powerful spring, with accompanying IP, to penetrate into muscle and reach a consistent depth across individuals being inoculated.
PharmaJet has signed partnerships with India’s Serum Institute, the largest vaccine manufacturer in the world, and Sri Lankan company, A. Baur & Co., to supply Stratis devices for their MMR and other vaccines.
“Now that we’re in the market place, we’re in discussions with other Indian biotech companies to use our device with their approved vaccines,” he points out. “Doctors Without Borders also is considering a pilot program in Africa to target measles using our device.”
Mr. Lowy says the company and Mundipharma are proceeding systematically in China. “We’ve been working with two vaccine developers, Simnogen, and the Kunming Institute, to achieve cFDA approval of our devices in China.”
PharmaJet also is participating in more than 70 development programs to expand the indications that would use the Tropis and Stratis injectors, including HIV, cancer therapeutics, dengue fever, yellow fever, rabies, malaria, tuberculosis, chickenpox, hepatitis A and B, typhoid, Zika, and human papillomavirus, among others.
In its business and product development programs, Mr. Lowy says current vaccine opportunities are preparing the company for a faster entry into new pharmaceutical markets using PharmaJet devices.
“There are hundreds of potential pharmaceuticals that can be administered using our devices. Our devices are the only ones that can bridge [the gap] between current and future pharmaceutical [delivery] technologies,” he says.
The company also has developed a pre-filled next generation syringe that can reduce delivery time by an additional 50% to 80%, simplifying technicians’ workflows. “Some of our target markets for a pre-filled syringe are influenza and the global analgesics market, which presents a large opportunity to solve drug control issues.”
Mr. Lowy points to a few companies with needle-free injectors in the veterinary, diabetes and at-home markets that dispense expensive therapeutics to patients. “We don’t consider them to be competitors at this point, but we keep an eye on them.”
The company does not disclose financial results but revenue in 2018 was in the “mid-single-digit” millions of dollars and “could reach the lower double-digit millions of dollars in 2019,” Mr. Lowy says. “Our commercialization efforts are accelerating.”
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Lineagen champions clinical information solutions for autism spectrum disorders and other neurodevelopmental conditions
Michael Paul, President and CEO
In 2017, the company generated approximately $18-million in revenue, reflecting a more than 70%-plus compound annual growth rate in revenue from billable tests and other services between 2014 and 2017.
The company serves a market of some 7,700 developmental pediatricians, pediatric neurologists, geneticists, and certain non-specialists like general pediatricians in the U.S., who typically order four-to-five tests a month from Lineagen at about $2,000 a test, resulting in an estimated U.S. addressable market of $850-million annually. Primary sales are to pediatric specialists, who currently represent about 70% of orders and 82% of volume.
“Besides testing, we provide personalized, easy-to-understand test results, genetic counseling and clinical education, and end-to-end reimbursement support,” Dr. Paul says. Third-party insurers currently cover and reimburse FirstStep, fragile X and NextStep.
According to Dr. Paul, the prevalence of ASD has soared 600% during the past 20 years. Currently, the global prevalence of ASD is one-in-47 boys and one-in-68 for all children.
ASD/NDD Prevalence Continues to Increase and is Global
Closely-held Lineagen has developed and commercialized a comprehensive portfolio of behavioral screening and genetic tests for autism spectrum disorders (ASD) and other forms of childhood neurodevelopmental disorders (NDDs).
“We are the only commercial diagnostic provider dedicated solely to the genetics of childhood developmental disorders,” Michael Paul, president and CEO, says in an interview with BioTuesdays. “We work with leading academic institutions and advocacy groups, but childhood developmental disorders is all we do.”
In the past, physicians would often advise parents to “wait and see” because they simply didn’t have accurate and accessible tools to diagnose the underlying genetic cause for a child’s developmental delay. “Genetic testing today means treating correctly sooner,” Dr. Paul contends.
Lineagen’s portfolio of genetic testing includes FirstStepDx Plus, a proprietary chromosomal microarray to detect larger deletions and duplications in the genome, which Dr. Paul says is like finding an entire page that has been torn out of, or inserted into, a book.
“And when we can’t find something with FirstStep, we move to NextStepDx Plus,” he adds. “This is a next-generation sequencing test to look at a higher resolution of the genome and detect smaller genome changes not detected by a chromosomal microarray.” He likens this process to finding a single letter missing in a sentence.
Lineagen has also developed advanced testing for a range of other conditions. Some prominent examples include the genetic mutation responsible for fragile X syndrome, which causes a range of learning disabilities and cognitive impairment; pharmacogenetics testing of genes that can impact dosage and choice of drug types, including anti-seizure agents; and mitochondrial DNA testing for variants associated with neurodegenerative diseases.
“So from a single cheek swab collected by a physician or pediatric specialist, we can look at a child’s genome in multiple ways,” he points out. “This provides a clearer picture than ever before for individuals and families dealing with neurodevelopment disorders, while providing Lineagen with multiple revenue opportunities.”
Explaining the increase, he cites better testing methodologies, genetic mutations that occur during development and potential environmental causes, such as pollution. The age of the father at conception also has been linked as a risk factor for autism because sperm DNA is known to mutate with aging and these mutations can be passed to the child.
“Genetic testing is recommended as a first-line diagnostic for autism because an early diagnosis can change the course of clinical management,” Dr. Paul contends. “And estimates from academic institutions suggest that autism is 70% to 80% genetic in origin.”
And while there is an approximate 2% risk of a first child having autism, the risk jumps to 25% with a second child, if a first child in a family has been diagnosed with autism.
In many U.S. states, children from birth to 3 years of age can automatically qualify for early intervention services if they have a documented genetic cause of their ASD/NDD, he adds.
Dr. Paul says all of Lineagen’s patients receive a clinically meaningful result. Some 86% of tested patients receive either a pathogenic genetic finding for ASD/NDD; a genetic carrier status; or a variant of unknown significance, which Lineagen attempts to interpret with its higher genetic resolution PRISM interpretation program licensed from Toronto’s SickKids Hospital. In about 14% of patients, the company’s technology can rule out hundreds of genetic conditions so doctors and parents can focus on next steps.
“There is a behavioral treatment for autism spectrum disorders that has been clinically proven to be effective in children as young as 18 months of age, and that has been shown to sustain IQ gains of 20-to-30 points,” Dr. Paul says. “This can be the difference between independent and dependent living as an individual grows to adulthood.”
In January, Lineagen inked a partnership with PWNHealth, a telemedicine solutions provider, to make its clinical genetic tests more accessible to families that want to act on concerns about delays in a child’s development and not wait to see a pediatric specialist, which can typically take more than a year in the U.S.
Dr. Paul says an important focus for Lineagen in 2019 will be to advance its portfolio of digital channels to advance families’ access to genetic testing. These include M-CHAT.org (modified checklist for autism in toddlers), Understanding Childhood Development Disorders (UCDD) and MyDevelopingChild.com.
M-CHAT is a validated screener for identifying children at risk for ASDs and is recommended for use by the American Academy of Pediatrics and the CDC. UCDD is a networking and educational platform for families searching for information about genetic testing for ASD. And MyDevelopingChild is an educational platform to help parents understand that genetic testing is recommended for every child diagnosed with a development delay.
According to Dr. Paul, digital assets and telehealth open the door to expand current sales and diversify revenue, as well as Lineagen’s data asset portfolio, which currently exceeds 30,000 individuals tested and 50,000 tests performed.
“We’re making a big push to use Big Data to integrate phenotypic and genotypic data in order to apply artificial intelligence and machine learning to help personalize healthcare and improve treatment outcomes,” he says.
“This is critical because while genetic testing has been shown to be very effective in children as young as 18 months, these results need to be personalized because many children have genetic mutations that must be treated medically in addition to behavioral treatments.”
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Richard Carreon, Managing Director and CEO
ImpediMed’s (ASX:IPD) transition to a software-as-a-service (SaaS) subscription business model in 2018, in conjunction with the introduction of the SOZO Digital Health Platform, is already paying off on the top line.
“We moved from a consumable and capital equipment business model to a high-margin subscription model where doctors pay us a flat fee on a monthly basis as they test their patients,” Richard Carreon, managing director and CEO, says in an interview with BioTuesdays.
Subscription revenue contributed 30% of the company’s medical revenue for the fiscal second quarter ended Dec. 31, 2018, and our future subscription revenue pipeline was $7-million at the end of the quarter, up 49% from the previous quarter. “This means we are seeing a strong customer and SaaS revenue pipeline building,” he adds.
ImpediMed is the world leader in the design and manufacture of medical devices employing next generation bioimpedance spectroscopy (BIS) technologies for use in the non-invasive clinical assessment and monitoring of tissue composition and fluid status.
BIS technology is non-invasive, and provides fast, accurate and medically meaningful information
“BIS has been the subject of more than 470 peer-reviewed publications for tissue and fluid monitoring across many chronic diseases,” Mr. Carreon points out.
The company’s family of FDA cleared and CE Marked medical devices include SOZO and L-Dex. SOZO is a third-generation device for multiple indications, including lymphedema and chronic heart failure (CHF) monitoring in the clinic and at home. L-Dex measures extracellular fluid differences in the limbs for unilateral and bilateral lymphedema, which is commonly associated with removal of or damage to lymph nodes as part of cancer treatment.
Mr. Carreon says ImpediMed’s L-Dex technology is able to detect lymphedema at stage zero, up to 10 months before any physical swelling appears in a patient’s limbs. If detected early, the progression of lymphedema can be prevented and often reversed by wearing compression therapy.
“Multiple, independent, investigator-led clinical studies have reported significantly lower rates of persistent lymphedema by monitoring patients with L-Dex and [administering] compression therapy,” he adds.
Mr. Carreon explains that ImpediMed’s BIS devices apply a very low-level alternating electrical current to the body to measure impedance, or how well the body’s tissues resist current flow, at 256 electrical frequencies. Doctors testing for lymphedema, heart and renal failure, for example, are looking for buildup of a patient’s extracellular fluid.
“In less than 30 seconds, we get a detailed makeup of the fluid, fat and muscle in the body,” he adds. “And in the case of fluid, we can break that down even further into extracellular and intracellular fluid, with our devices detecting fluid shifts as little as 2 1/2 tablespoons.”
“Our technology is fast, accurate, sensitive, non-invasive, informative, actionable and medically meaningful,” Mr. Carreon contends. “We have a highly disruptive non-invasive digital health platform for the clinical monitoring of fluid and tissue.”
Mr. Carreon says SOZO has the potential to change health care by identifying early onset of disease states both in the clinic and at home, leading to improved patient outcomes.
SOZO® - Next Generation BIS Digital Health Platform
“The power of SOZO is long-term monitoring of patients so that clinicians can gain information on daily fluid changes in CHF patients and monthly or quarterly fluid changes in cancer patients.”
There are 15.5 million cancer survivors in the U.S., with 1.7 million new cases diagnosed annually. And one-in-three cancer survivors will develop lymphedema as a result of their cancer treatment, with 75% of those developing it in the first three years.
“SOZO assists with the early detection of lymphedema and could assist with other aspects of patient management, such as nutrition, bone density and muscle wasting,” Mr. Carreon contends. Cancer survivorship, following initial treatment and five-year follow up testing, represents an addressable annual market of more than $1.8-billion for ImpediMed.
In addition, cancer survivors have a 15-fold increased risk of developing CHF, which is a progressive disease where patients experience a permanent decline every time they have a major cardiac event. There are 6.5 million CHF patients in the U.S., of which 25% are classified as moderate-to-severe.
Mr. Carreon says SOZO can detect small changes in fluid levels that typically precede a major cardiac event, and that the event may be prevented by adjusting medication. Monitoring CHF failure patients represents an addressable market of more than $1-billion a year for ImpediMed.
SOZO’s worldwide customer base and adoption are accelerating, notably in large cancer centers in the U.S., because of SOZO’s ease of use and the company’s new subscription business model, he adds.
Citing the University of Kansas Cancer Center, one of four ImpediMed centers of excellence, Mr. Carreon says the institution subscribed for 14 SOZO devices last year. “The Kansas center is considering adding additional SOZO units and expanding testing to pelvic cancer patients in 2019. They expect more than 2,000 newly diagnosed cancer patients per year to be enrolled in its testing program.”
ImpediMed recently reported interim results from its PREVENT trial, a three-year follow up of 1,100 patients in the U.S. and Australia. The study is designed to determine if detection of extracellular fluid accumulation via BIS technology and subsequent early intervention can reduce the rate of lymphedema progression relative to rates where lymphedema was detected using standard tape measurements.
The primary endpoint is to achieve a relative 20% improvement over the standard of care. According to Mr. Carreon, top line results from the first year follow up of 500 patients demonstrated a 67% relative and a 10% absolute improvement in progression to persistent lymphedema in the L-Dex arm, compared to tape measurements. Second year follow up data will be reported in mid-2019.
“These interim data were medically significant,” he points out, adding that the PREVENT trial is designed to obtain additional data as a stepping-stone for reimbursement from private payers.
“We believe the PREVENT trial will establish SOZO as the standard of care for the early detection and monitoring of lymphedema in cancer survivors to either prevent the condition from developing or stop its progression.”
In CHF, Mr. Carreon says the company is initially targeting about 1.6 million class 3 patients, with the goal of providing daily monitoring either in a clinic or at home.
In a study with advanced heart failure patients that also had a CardioMEMS implant device, Mr. Carreon says SOZO BIS measurements had a correlation coefficient of 0.88 with changes in diastolic pulmonary artery pressure as measured by CardioMEMS.
“We were able to non-invasively correlate to CardioMEMS at a fraction of the $25,000 cost of an implant,” he adds.
In October 2018, ImpediMed began a study to follow some 200 CHF patients with daily measurements for 45 days at home after being discharged from hospital.
“The study will be a catalyst for initiating a broad market release of SOZO and obtaining favorable reimbursement to remotely manage patients at home,” Mr. Carreon says. “We only need to show an impact on reducing hospital readmissions and improving patient outcomes.”
ImpediMed plans to either partner SOZO in the CHF field or build its own sales and marketing team. “We have a sales staff of 12 people detailing SOZO for lymphedema, which we will continue to expand as we get closer to private payer reimbursement.”
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Interface Biologics to focus future growth in biotech with local drug delivery platform
Kelley Dealhoy, VP of Business Development
Closely-held Interface Biologics, best known for improving the safety and efficacy of medical devices through its Endexo surface modification technology, is pegging its future growth on biotech, with an initial focus on unmet needs in ophthalmology.
“For some time, we have been working to evolve from a company with a passive surface modification technology and OEM licensing model to a biotech company with a local drug delivery platform and our own product programs,” Kelley Dealhoy, VP of business development, says in an interview with BioTuesdays. “We are at an inflection point in the process of creating Interface 2.0.”
Interface was created in the early 2000s around its Endexo fluorinated surface modification technology, which enhances the biocompatibility of medical devices that come into contact with blood or other body fluids.
As an additive rather than a coating, Endexo is incorporated during the manufacturing process and migrates to the surface of a device. From a clinical perspective, the Endexo additive has been demonstrated to reduce the threat of infection and complications related to thrombus. Endexo’s partnerships include Fresenius Medical Care and Angiodynamics.
The company’s second technology platform is Kinesyx, with similar fluorinated oligomers that coat the surface of medical devices with various drugs with a tailored drug release profile, depending on therapeutic requirements.
Interface’s third technology, Epidel, is a “non-polymeric sustained drug delivery platform technology with significant advantages over current polymeric approaches. The technology is highly engineerable for the custom design of implants, drug release duration and kinetics,” Ms. Dealhoy explains.
Epidel’s lead candidate is a dexamethasone intravitreal implant, IBE-814, for the treatment of posterior inflammatory eye diseases, initially targeting diabetic macular edema (DME), retinal vein occlusion (RVO) and non-infectious uveitis (NIU). The company completed preclinical studies in 2018 and expects to start a Phase 2 program with IBE-814 in 2020.
“Our pre-IND meeting with the FDA in October 2018 was a pivotal point for the company, not only validating the technology but also confirming an efficient and cost effective regulatory pathway going forward in ophthalmology,” Ms. Dealhoy points out. “Within two years, we expect to have three programs in clinical development, including advancing the technology into pain and osteoarthritis,” she adds.
Ms. Dealhoy explains that the Epidel drug delivery technology has broad applications across a range of therapeutic areas and drug classes. While the company’s initial product is an injectable implant for inflammatory posterior eye disease, “we can formulate the technology into micro-and nano-particles and coatings for medical devices, including wafers for post-cataract inflammation and minimally invasive glaucoma surgical devices,” she adds.
Broad applications across a range of forms and applications
The company has focused on ophthalmology not only because of the size of the market but also because of the need for local drug delivery. The total addressable market for IBE-814 in posterior inflammatory diseases includes more than 1.1 million RVO patients in the U.S., 560,000 DME patients and as many as 120,000 NIU posterior patients.
In addition, there were 4.4 million cataract surgeries performed in the U.S. in 2017 and 2.7 million patients with open-angle glaucoma.
Ms. Dealhoy says IBE-814 has distinct advantages targeting DME, RVO and NIU, compared with the currently commercialized dexamethasone implant, Ozurdex. “Our 30-gauge implant is smaller, minimally invasive, more comfortable for patients and more convenient for ophthalmologists than Ozurdex’s 22-gauge implant.”
The Ozurdex implant also has an initial burst of drug release that tapers off and lasts for around three months, while IBE-814 is designed to deliver a constant dose for up to nine-to-12 months, and eliminate the side effects of polymer degradation, floaters and inflammation, she adds.
“Ophthalmologists tell us they want an implant that is more predictable and durable in terms of response.”
In the fourth quarter of 2018, Interface received FDA approval for a 505(b)(2) regulatory pathway for IBE-814, which would require the company to only match the efficacy and safety data of the Ozurdex implant.
“This would allow us to test our technology in an ocular indication that has a high probability of success, with a known active pharmaceutical ingredient (API) and delivery system,” Ms. Dealhoy suggests. “And it fits with our strategy of starting with an ocular steroid and then, based on a proven technology, filing NDAs for other APIs in other indications.”
Interface plans to spend most of 2019 confirming the chemistry, manufacturing and controls as well as the toxicology of IBE-814 in order to begin a Phase 2 proof-of-concept trial in Australia in 2020. The company also plans to initiate discussions with potential pharma partners to assess their interest in IBE-814.
“We also intend to initiate discussions with external venture capital firms to assess their interest in funding Interface 2.0 in conjunction with our existing investors and hope to complete a financing round by the end of 2019,” Ms. Dealhoy says.
If the Phase 2 study is successful, Interface intents to initially focus on an approval for DME in the U.S., which could be obtained within five years and require only one pivotal Phase 3 trial.
“With our initial focus on steroid delivery for ophthalmology, we have a strong program with a defined regulatory path and a short timeline to commercialization,” she says. “We see this as our first step in validating the Epidel technology platform and are excited about the additional opportunities in our pipeline.”
IBI Epidel Drug Delivery Platform Pipeline
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