Welcome!

News Feed Item

FDA Grants Orphan-Drug Designation for Novel Terlipressin Formulation for the Treatment of Ascites

Ascites a Serious Complication of Liver Cirrhosis with Two-year Survival 50%

BOTHELL, Wash. and CHICAGO, Jan. 4, 2013 /PRNewswire/ -- PharmaIN Corporation and LAT Pharma LLC today announced that the US Food and Drug Administration (FDA) has granted their request for orphan-drug designation for terlipressin for the treatment of ascites due to all etiologies except for cancer. Ascites, or fluid accumulation in the abdomen, is a serious complication of liver cirrhosis. The companies' lead new drug candidate PHT101 (or PGC-C12E-Terlipressin) incorporates novel drug delivery technologies that may enable once-daily administration via subcutaneous injection dosing in chronic outpatient populations.

Chronic liver disease/cirrhosis is the 12th leading cause of death due to disease in the US, killing an estimated 27,000 people each year. Approximately 60% of cirrhosis patients eventually develop ascites. Ascites patients face a significantly increased risk of other life-threatening complications, such as spontaneous bacterial peritonitis (SBP) and hepatorenal syndrome (HRS), with a very poor prognosis of 50% survival two years after onset. Current treatments include salt-restricted diet, high doses of diuretics, and frequent paracentesis, the removal of large volumes of ascitic fluid with a needle through the abdominal wall. People with ascites are also often candidates for liver transplantation.

Elijah Bolotin, PhD, President of PharmaIN, said, "Orphan-drug designation by the FDA indicates the importance of this new drug candidate to a desperately ill group of patients. We are excited about the medical potential of PHT101, which synergistically incorporates two of our proprietary technologies designed to enable safer and more effective drug therapy: PGC-Hydrophobic Core nanocarrier-based delivery and unique fatylation that leads to a pro-drug."

"Orphan-drug designation represents a major milestone toward making PHT101 available to patients suffering from ascites due to liver cirrhosis," said John Thottathil, PhD, Chief Scientific Officer of LAT Pharma. "PHT101 would bring a novel, mechanistic approach to ascites therapy as opposed to the current standard of care involving symptom relief. Orphan status may accelerate our clinical development program. It also opens the door to special funding opportunities, such as the Orphan Product Grants Program, and provides important incentives to investors, including seven years of market exclusivity and certain tax credits."

Terlipressin reduces portal vein pressure and increases mean arterial pressure (MAP) in cirrhotic patients with splanchnic vasodilation. Increasing MAP in ascites patients could potentially down-regulate the excessive salt and water retention that leads to ascitic fluid buildup. For more than 20 years unmodified IV-bolus terlipressin has been used in Europe and Asia as rescue therapy for hepatorenal syndrome (HRS) and esophageal variceal bleeding (EVB). Scores of publications have demonstrated the safety and efficacy of IV terlipressin in hospitals, and many have suggested the medical potential of an outpatient version of the drug.

PHT101 is in preclinical testing and is protected by a US patent issued in 2011. International patent applications have been filed. The novel drug delivery technologies are covered by a separate patent estate.

About PharmaIN Corporation

Based in Bothell, WA, PharmaIN is focused on improving the efficacy, safety, and speed to market of injectable peptides/proteins (biologics) for the treatment of cirrhotic liver disease, congestive heart failure (CHF), cancer, inflammation, and other serious medical conditions. The Company has developed proprietary Protected Graft Copolymer (PGC) nanocarrier excipients (15 patent families) and other drug delivery and fatylation technologies. Unique benefits of these technologies include prolonging circulation time in the blood by protecting against enzymatic degradation while preserving drug activity; creating optimal pharmacokinetic/pharmacodynamic profiles to avoid peaks and troughs in drug concentrations; and enabling more effective targeting of cancer, inflammation, and infection sites because of the carrier's uniquely small size (10-20nm). PGC is a highly versatile nanocarrier platform as demonstrated by the improved performance of small peptides through very large proteins, both for subcutaneous and intravenous administration. For more about PharmaIN visit www.pharmain.com.

About LAT Pharma LLC

LAT Pharma was founded in 2006 with the express goal of inventing, developing, and commercializing a lifesaving new therapy for people afflicted with advanced liver disease and its deadly complications. Each member of the Chicago-based management team has decades of pharma/biotech industry experience primarily in the area of new drug development and commercialization. The company is supported by medical advisors who are worldwide leaders in chronic liver disease. LAT Pharma holds an exclusive global license to all unique terlipressin compounds arising from its collaboration with PharmaIN and is responsible for clinical development, marketing, and out-licensing of PHT101. For more about LAT Pharma visit www.lat-pharma.com.

SOURCE LAT Pharma LLC

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
“DevOps is really about the business. The business is under pressure today, competitively in the marketplace to respond to the expectations of the customer. The business is driving IT and the problem is that IT isn't responding fast enough," explained Mark Levy, Senior Product Marketing Manager at Serena Software, in this SYS-CON.tv interview at DevOps Summit, held Nov 4–6, 2014, at the Santa Clara Convention Center in Santa Clara, CA.
SYS-CON Events announced today that CollabNet, a global leader in enterprise software development, release automation and DevOps solutions, will be a Bronze Sponsor of SYS-CON's 20th International Cloud Expo®, taking place from June 6-8, 2017, at the Javits Center in New York City, NY. CollabNet offers a broad range of solutions with the mission of helping modern organizations deliver quality software at speed. The company’s latest innovation, the DevOps Lifecycle Manager (DLM), supports Value S...
All organizations that did not originate this moment have a pre-existing culture as well as legacy technology and processes that can be more or less amenable to DevOps implementation. That organizational culture is influenced by the personalities and management styles of Executive Management, the wider culture in which the organization is situated, and the personalities of key team members at all levels of the organization. This culture and entrenched interests usually throw a wrench in the work...
With billions of sensors deployed worldwide, the amount of machine-generated data will soon exceed what our networks can handle. But consumers and businesses will expect seamless experiences and real-time responsiveness. What does this mean for IoT devices and the infrastructure that supports them? More of the data will need to be handled at - or closer to - the devices themselves.
Building a cross-cloud operational model can be a daunting task. Per-cloud silos are not the answer, but neither is a fully generic abstraction plane that strips out capabilities unique to a particular provider. In his session at 20th Cloud Expo, Chris Wolf, VP & Chief Technology Officer, Global Field & Industry at VMware, will discuss how successful organizations approach cloud operations and management, with insights into where operations should be centralized and when it’s best to decentraliz...
Join IBM November 2 at 19th Cloud Expo at the Santa Clara Convention Center in Santa Clara, CA, and learn how to go beyond multi-speed it to bring agility to traditional enterprise applications. Technology innovation is the driving force behind modern business and enterprises must respond by increasing the speed and efficiency of software delivery. The challenge is that existing enterprise applications are expensive to develop and difficult to modernize. This often results in what Gartner calls ...
Translating agile methodology into real-world best practices within the modern software factory has driven widespread DevOps adoption, yet much work remains to expand workflows and tooling across the enterprise. As models evolve from pockets of experimentation into wholescale organizational reinvention, practitioners find themselves challenged to incorporate the culture and architecture necessary to support DevOps at scale. In his session at @DevOpsSummit at 20th Cloud Expo, Anand Akela, Senior...
Web Real-Time Communication APIs have quickly revolutionized what browsers are capable of. In addition to video and audio streams, we can now bi-directionally send arbitrary data over WebRTC's PeerConnection Data Channels. With the advent of Progressive Web Apps and new hardware APIs such as WebBluetooh and WebUSB, we can finally enable users to stitch together the Internet of Things directly from their browsers while communicating privately and securely in a decentralized way.
In his keynote at @ThingsExpo, Chris Matthieu, Director of IoT Engineering at Citrix and co-founder and CTO of Octoblu, focused on building an IoT platform and company. He provided a behind-the-scenes look at Octoblu’s platform, business, and pivots along the way (including the Citrix acquisition of Octoblu).
Multiple data types are pouring into IoT deployments. Data is coming in small packages as well as enormous files and data streams of many sizes. Widespread use of mobile devices adds to the total. In this power panel at @ThingsExpo, moderated by Conference Chair Roger Strukhoff, panelists will look at the tools and environments that are being put to use in IoT deployments, as well as the team skills a modern enterprise IT shop needs to keep things running, get a handle on all this data, and deli...
Grape Up is a software company, specialized in cloud native application development and professional services related to Cloud Foundry PaaS. With five expert teams that operate in various sectors of the market across the USA and Europe, we work with a variety of customers from emerging startups to Fortune 1000 companies.
The 20th International Cloud Expo has announced that its Call for Papers is open. Cloud Expo, to be held June 6-8, 2017, at the Javits Center in New York City, brings together Cloud Computing, Big Data, Internet of Things, DevOps, Containers, Microservices and WebRTC to one location. With cloud computing driving a higher percentage of enterprise IT budgets every year, it becomes increasingly important to plant your flag in this fast-expanding business opportunity. Submit your speaking proposal ...
Financial Technology has become a topic of intense interest throughout the cloud developer and enterprise IT communities. Accordingly, attendees at the upcoming 20th Cloud Expo at the Javits Center in New York, June 6-8, 2017, will find fresh new content in a new track called FinTech.
SYS-CON Events announced today that Interoute, owner-operator of one of Europe's largest networks and a global cloud services platform, has been named “Bronze Sponsor” of SYS-CON's 20th Cloud Expo, which will take place on June 6-8, 2017 at the Javits Center in New York, New York. Interoute is the owner-operator of one of Europe's largest networks and a global cloud services platform which encompasses 12 data centers, 14 virtual data centers and 31 colocation centers, with connections to 195 add...
The Internet of Things is clearly many things: data collection and analytics, wearables, Smart Grids and Smart Cities, the Industrial Internet, and more. Cool platforms like Arduino, Raspberry Pi, Intel's Galileo and Edison, and a diverse world of sensors are making the IoT a great toy box for developers in all these areas. In this Power Panel at @ThingsExpo, moderated by Conference Chair Roger Strukhoff, panelists discussed what things are the most important, which will have the most profound e...