Simulations Plus, Inc | June 24, 2022
Simulations Plus, Inc. a leading provider of modeling and simulation software and services for pharmaceutical safety and efficacy, today announced that its Cognigen division worked with a major pharmaceutical company to implement modeling and simulation activities to support the development of a candidate COVID-19 therapy. This modeling and simulation work provided significant contributions towards regulatory submissions for the compound.
Early in the COVID-19 pandemic, the pharmaceutical company initiated a collaboration to support the accelerated development of their compound. Together with scientists from the company, Sébastien Bihorel, Pharm.D., Ph.D., Senior Director of Pharmacometrics and Workflow Automation, led this cooperative team effort on behalf of Simulations Plus. Cognigen scientists provided real-time data processing and analysis, allowing for rapid knowledge updates about pharmacokinetics and efficacy in patients with COVID‑19 as the clinical trials proceeded.
“We are extremely proud of this collaboration, which further underscores the importance and critical role of modeling and simulation in informing development-related decisions and improving the efficiency of drug development,”
Jill Fiedler-Kelly, Cognigen division president
About Simulations Plus
Serving clients worldwide for 25 years, Simulations Plus is a leading provider in the biosimulation market providing software and consulting services supporting drug discovery, development, research, and regulatory submissions. We offer solutions that bridge machine learning, physiologically based pharmacokinetics, quantitative systems pharmacology/toxicology, and population PK/PD modeling approaches. Our technology is licensed and applied by major pharmaceutical, biotechnology, and regulatory agencies worldwide.
Safe Harbor Statement Under the Private Securities Litigation Reform Act of 1995 – With the exception of historical information, the matters discussed in this press release are forward-looking statements that involve a number of risks and uncertainties. Words like “believe,” “expect” and “anticipate” mean that these are our best estimates as of this writing, but that there can be no assurances that expected or anticipated results or events will actually take place, so our actual future results could differ significantly from those statements. Factors that could cause or contribute to such differences include, but are not limited to: our ability to maintain our competitive advantages, acceptance of new software and improved versions of our existing software by our customers, the general economics of the pharmaceutical industry, our ability to finance growth, our ability to continue to attract and retain highly qualified technical staff, our ability to identify and close acquisitions on terms favorable to the Company, and a sustainable market. Further information on our risk factors is contained in our quarterly and annual reports and filed with the U.S. Securities and Exchange Commission.
Tris Pharma, Inc. | July 28, 2022
Tris Pharma, Inc. a fully integrated pharmaceutical company with a robust portfolio of CNS products announced that it has successfully developed an oxybate formulation for the treatment of cataplexy or excessive daytime sleepiness n patients 7 years of age and older with narcolepsy that has the potential to be significantly advantageous to existing approved therapies, such as Jazz Pharmaceuticals' blockbuster XYWAV® and XYREM® franchise. The Tris oxybate formulation is believed to provide an advantage over approved therapies by utilizing Tris's proprietary LiquiXR technology platform and our newly developed RaftWorks design to provide a unique oxybate formulation offering both once-nightly dosing, and very low sodium content. The Tris oxybate formulation is investigational and not yet approved by any regulatory agency.
Both XYWAV® and XYREM® require twice a night dosing – once at bedtime and again 2.5 to 4 hours later for the second dose. Further, XYREM contains an express warning based on its high sodium content and associated risk to patients with heart failure, hypertension or impaired renal function. The Tris oxybate formulation seeks to improve on existing therapies by providing convenient once per night dosing, and significantly reduced sodium content such that it will contain less than 100 mg of sodium per 9.0 gm of maximum sodium oxybate equivalent dose. Based on the significant potential advantage the Tris oxybate formulation could provide over existing available therapies, Tris is pursuing an Orphan Drug Designation for its unique oxybate formulation.
In a human clinical study, Tris established about 100% bioavailability for one dose of its formulation against two doses of XYREM at 4.5 gm each, equivalent to the 9.0 gm maximum dose of sodium oxybate. If approved, the Tris product will provide significant advantages in that patients will not have to wake up in the middle of the night to take a second dose and will have all the benefits of a very low sodium product.
The Tris oxybate product achieved its clinical objectives by combining two distinct proprietary Tris technologies: the LiquiXR platform, and the newly developed RaftWorks design. Working together, these unique formulation platforms provide an interpenetrating network raft in-situ, in which sodium-free drug particles are entrapped in a raft that allows for extended release of the active ingredient. Many drugs, such as oxybate, are poorly absorbed because they have a narrow window of absorption within the human gastro-intestinal tract. The RaftWorks approach permits a slow and extended release of oxybate from a floating raft in-situ, thereby facilitating absorption of the active ingredient within the targeted area in the gastro-intestinal tract. Further, the raft disintegrates at a predetermined time due to the trigger mechanism that is built into the formulation.
"This unique formulation design by Tris scientists potentially achieves drug delivery capabilities that have never previously been achieved within our industry. We are looking to build on this approach and develop extended-release formulations for other drugs that are limited by their narrow window of absorption."
Ketan Mehta, Founder and Chief Executive Officer of Tris
Narcolepsy, a chronic and disabling neurological disorder that affects the stability of sleep and wakefulness, is characterized primarily by excessive daytime sleepiness, with or without episodic loss of muscle tone usually triggered by strong emotions during wakefulness (cataplexy) 1-4. Additional symptoms can vary by person and may include disrupted nighttime sleep, and REM-sleep phenomena (sleep paralysis, hallucinations). The exact number of individuals with narcolepsy in the United States is unknown. Several studies have estimated the prevalence of narcolepsy in the United States to be approximately 1 (range: 0.6 to 1.6) in 2,000 people 2,4-6. There is currently no cure for narcolepsy; treatment of symptoms includes medications and lifestyle modifications 1.
About Tris Pharma
Tris is a fully integrated, innovation-driven CNS company that provides a differentiated approach to target unmet medical needs, including the application of novel technologies designed to enhance patient benefits across therapeutic categories. Tris's CNS portfolio includes treatments for pain; addiction; spasticity in multiple sclerosis, cerebral palsy; narcolepsy; and ADHD.
Inozyme Pharma Inc. | June 17, 2022
Inozyme Pharma, Inc. a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced a partnership with Rady Children’s Institute for Genomic Medicine (RCIGM) to advance and evaluate a novel newborn screening technology to facilitate diagnosis of genetic diseases. The partnership includes several leading genomics, biotechnology companies and patient advocacy groups and focuses specifically on a diagnostic and precision medicine guidance tool called BeginNGS™, which incorporates rapid Whole Genome Sequencing (rWGS®) to currently screen newborns for approximately 400 genetic diseases.
“Newborn screening will be essential to identifying and initiating timely intervention in children with rare genetic disorders like GACI (generalized arterial calcification of infancy) as we advance INZ-701 through clinical testing. We look forward to working with Rady Children’s Institute for Genomic Medicine, and with the BeginNGS consortium, to advance the use of this promising screening technology.”
Catherine Nester, vice president, physician and patient strategies at Inozyme Pharma
RCIGM is in a pilot evaluation that aims to supplement existing newborn screening protocols at birthing hospitals throughout the United States. The pilot program’s goal is for BeginNGS to become the genetic disease screening standard, with testing expanding to approximately 1,000 [disorders] and sequencing of 3.7 million newborns annually. Founding members of the public-private BeginNGS consortium include Inozyme, Alexion, Travere Therapeutics, and several patient advocacy groups that are helping to advance this program.
“RCIGM helped pioneer the use of rWGS for diagnosis of genetic disease in intensive care settings,” said Stephen Kingsmore, MD, DSc, president and CEO of RCIGM. “With the proven clinical utility of diagnostic rWGS , we are using that experience to screen, diagnose, and help treat genetic conditions at or before onset of symptoms. Through a public-private consortium of leading organizations such as Inozyme, and advocacy groups in pediatrics, genetics, biopharma, biotech, and information technology, we aim to scale newborn sequencing to every life-threatening childhood genetic disease, RCIGM believes now is the time to end the diagnostic and therapeutic odyssey for all children with treatable genetic diseases.”
BeginNGS developed through a research collaboration with Alexion; AstraZeneca’s Rare Disease group; Illumina, Inc.; TileDB; Fabric Genomics; and Genomemon, which uses rWGS to diagnose and identify treatment options for genetic conditions before symptoms begin. This approach represents an advance over current pediatric uses of rWGS that focus mainly on children who are already critically ill. Once a diagnosis is made, BeginNGS uses Genome-to-Treatment (GTRx™), a tool that provides immediate treatment guidelines to help physicians understand genetic conditions and their available treatment options.
Addressing the Need for Enhanced Newborn Screening Tools
Traditional newborn screening is one of the most successful public health programs in the United States. Of nearly 4 million babies born annually, 98 percent are tested in the first days of life. The BeginNGS test identifies serious childhood diseases that have effective treatments. States currently screen for only 31 to 76 of the hundreds of severe, childhood genetic diseases that have available treatments. Adding a new condition to the screening protocol is slow (5 to 6 years per condition), laborious, and costly. In the last decade, WGS has increased in speed, diagnostic performance, and scalability. BeginNGS will not replace the current biochemical newborn screening paradigm; rather, it is designed to complement the newborn screening processes and infrastructure that are already in place.
“We are thrilled at the prospect of newborn screening to assist in early identification of infants affected by ENPP1 Deficiency and ABCC6 Deficiency via Inozyme’s collaboration with Rady Children’s Institute for Genomic Medicine. Early diagnosis is crucial to improving a baby’s chances of survival and long-term health if they have these rare and devastating diseases,” said Christine O’Brien and Liz Molloy, co-presidents of GACI Global.
About Rady Children’s Institute for Genomic Medicine
Rady Children’s Institute for Genomic Medicine is transforming pediatric critical care by advancing disease-specific healthcare for infants and children with rare disease. Discoveries at the Institute are enabling rapid diagnosis and targeted treatment of critically ill newborns and pediatric patients at Rady Children’s Hospital-San Diego and a growing network of more than 60 children’s hospitals nationwide. The vision is to expand delivery of this life-changing technology to enable the practice of Rapid Precision Medicine™ at children’s hospitals across the nation and the world. RCIGM is a non-profit, research institute embedded within Rady Children’s Hospital and Health Center.
About Inozyme Pharma
Inozyme Pharma, Inc. is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy, INZ-701, to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies. INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.