Vanessa Research Gains Approval to Conduct Phase II Clinical Trial for Treatment of Rare Pediatric Disease
Study will evaluate safety and efficacy of Shylicine®, the first-ever investigational treatment developed for microvillus inclusion disease
Vanessa Research — a biomedical company founded on the desire to “give hope where none existed” by making an impact on healthcare markets that are often overlooked — has been granted regulatory approval by the Turkish Ministry of Health to conduct a Phase II Clinical Trial for Shylicine®, an investigational oral solution developed to treat the rare and lethal microvillus inclusion disease.
About the Disease
Microvillus inclusion disease (MVID) is an exceedingly rare genetic gastrointestinal disorder that afflicts infants with diarrhea so severe that malnutrition and dehydration are inevitable, and often lethal, if the condition is not appropriately treated.
The current standard of care for MVID is total parenteral nutrition, in which fluids and nutrients are delivered intravenously for up to 24 hours each day. This can often result in infection, damage to the veins, and liver failure. Patients may ultimately seek a small bowel and/or liver transplantation; a process which comes with many challenges and complications of its own.
Vice President of Research and Development, Dmitry Kravtsov, M.D. identified the role that intestinal cell immaturity plays in the severity of diarrhea while working as a research scientist at the Yale School of Medicine. His work suggested that the failure of the MVID intestine may not be permanent, and that the immature cells of the gut could be restored with appropriate intervention – allowing a patient’s intestine to absorb necessary hydration and nutrition. These findings have the potential for broader applications in the field of secretory diarrhea: currently, Vanessa Research is in the preclinical development stage for Hunazine®, a treatment designed to neutralize the cholera toxin in affected patients.
About the Trial
Shylicine® is intended to serve as an oral treatment that could potentially eliminate patients’ dependence on total parenteral nutrition. The twelve-week Shylicine® trial will assess the safety and efficacy of the investigational treatment. The primary endpoint is reduction in the volume of diarrhea and frequency of stool, as well as a decrease in the duration of feeding via total parenteral nutrition.
In addition to receipt of approval to begin the trial, Vanessa Research has been granted a drug import permit, which will allow the U.S.-based company to deliver Shylicine® to the trial site, the Ankara University School of Medicine.
“Authorization by the Turkish Ministry of Health and the support of the Ankara University School of Medicine are critical, given that a relatively high volume of reported MVID cases are located in the country,” explains Norman Gray, CEO of Vanessa Research. “This is a critical step towards addressing the unmet needs of this rare disease community.”
About Vanessa Research
Vanessa Research is a biomedical company founded on the desire to “give hope where none existed” by making an impact on healthcare markets that are often overlooked. VR’s continually-expanding pipeline of innovative medicine, medical devices, and consumer and public health products reflects the company’s inventive nature and commitment to delivering solutions that increase access to quality care. The company’s flagship products, Shylicine® and Hunazine® are the first-ever drug developed to treat the rare, lethal microvillus inclusion disease and a treatment designed to neutralize the cholera toxin, respectively.
VR is based in Hamden, Connecticut, with offices in Budapest, Hungary; Munich, Germany; and Navarre, Spain.
Director of Marketing and Communications
The information contained in this release is as of June 4, 2019. VR assumes no obligation to update forward‐looking statements contained in this release as the result of new information or future events or developments.
This release contains forward‐looking information about VR’s Shylicine®, treatment candidate for the indication of secretory diarrhea caused by microvillus inclusion disease, including its potential benefits, and VR’s plans to initiate Phase 2 trials in the coming months, that involves substantial risks and uncertainties which could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical trial completion dates and regulatory submission dates, as well as the possibility of unfavorable clinical trial results, including unfavorable new clinical data or additional analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations, and, even when we view data as sufficient to support the safety and/or effectiveness of a product candidate, regulatory authorities may not share our views and may require additional data or may deny approval altogether; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any biologics license applications may be filed in any jurisdictions for Shylicine® for any indications; whether and when any such applications may be approved by regulatory authorities, which will depend on the assessment by such regulatory authorities of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted and, if approved, whether Shylicine® will be commercially successful; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of Shylicine®; uncertainties regarding the ability to obtain recommendations from technical committees and other public health authorities as well as uncertainties regarding the commercial impact of any such recommendations; and competitive developments.