Wellstat Therapeutics is a privately-owned biopharmaceutical company based in Gaithersburg, Maryland. The company is committed to discovering, developing, and commercializing innovative therapeutics that push the boundaries of medicine and improve the lives of patients with chronic and life-threatening diseases. Their focus areas include oncology, metabolic diseases, neurodegenerative diseases, and neurometabolic diseases. Wellstat Therapeutics has a diverse pipeline of novel small molecule therapeutic agents and owns a broad patent portfolio covering this technology. The company was founded in 1985 and has a highly experienced management team.

Tags

Skin and Musculoskeletal Diseases

Other Diseases

Infectious Diseases

Small molecule drug

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

No Data

Disease Domain	Count

Endocrinology and Metabolic Disease	2
Infectious Diseases	2
Nervous System Diseases	1
Neoplasms	1

Top 5 Drug Type	Count

Small molecule drug	6

Drugs associated with Wellstat Therapeutics Corp.

Uridine Triacetate

Target-

Mechanism-

Active Org.

BTG International, Inc.

Originator Org.

Wellstat Therapeutics Corp.

Active Indication

Drug intoxication [+1]

Inactive Indication

Bipolar Disorder [+1]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

First Approval Date04 Sep 2015

WT-501

Target-

Mechanism-

Active Org.

Wellstat Therapeutics Corp.

Originator Org.

Wellstat Therapeutics Corp.

Active Indication

Mitochondrial Myopathies

Inactive Indication-

Drug Highest PhasePhase 2

First Approval Ctry. / Loc.-

First Approval Date-

WT-35021

Target-

Mechanism-

Active Org.

Wellstat Therapeutics Corp.

Originator Org.

Wellstat Therapeutics Corp.

Active Indication

Solid tumor

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with Wellstat Therapeutics Corp.

NCT02110147 / CompletedPhase 3

Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria

This protocol has two parts - the Main Study which is 42 days in length and the Treatment Extension which allows the patients who complete the Main Study to continue receiving treatment with uridine triacetate. The purpose of this study is to replace oral administration of uridine with oral administration of uridine triacetate in patients with hereditary orotic aciduria who have received (or would reasonably be expected to receive) clinical benefit from treatment with exogenous uridine. It is also to document the continued clinical benefit of exogenous uridine when patients are switched from oral administration of uridine to oral administration of uridine triacetate.

Start Date30 Apr 2014

Sponsor / Collaborator

Wellstat Therapeutics Corp.

NCT00110864 / CompletedPhase 2

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of the Effects of PN2034 in Insulin-Dependent Patients With Type 2 Diabetes

This study is designed to evaluate the safety and efficacy of PN2034 in insulin-dependent type 2 diabetics as measured by the change in average daily insulin dose from baseline to week 12. The effects of PN2034 on HbA1c, fasting plasma glucose (FPG), and lipid levels will also be measured.

Start Date01 May 2005

Sponsor / Collaborator

Wellstat Therapeutics Corp.

NCT00110851 / CompletedPhase 2

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of the Effects of PN2034 in Type 2 Diabetes Patients on Concomitant Rosiglitazone and Metformin (or Avandamet) Therapy

This study is designed to evaluate the safety and efficacy of PN2034 administered to type 2 diabetics on concomitant rosiglitazone and metformin (or Avandamet) therapy as measured by change in glycosylated hemoglobin (HbA1c) levels from baseline to Week 12. Also the study will evaluate the effect of PN2034 on glucose control as measured by fasting plasma glucose (FPG) and on fasting lipid levels: triglycerides, total cholesterol, high-density lipoproteins (HDL), low-density lipoproteins (LDL), and very low-density lipoproteins (VLDL).

Start Date30 Apr 2005

Sponsor / Collaborator

Wellstat Therapeutics Corp.

100 Clinical Results associated with Wellstat Therapeutics Corp.

0 Patents (Medical) associated with Wellstat Therapeutics Corp.

Literatures (Medical) associated with Wellstat Therapeutics Corp.

01 Mar 2007·Current Cancer Drug TargetsQ4 · MEDICINE

Phase 1 clinical experience using intravenous administration of PV701, an oncolytic Newcastle disease virus

Q4 · MEDICINE

Review

Author: Lorence, Robert M. ; Roberts, M. Scot ; O'Neil, James D. ; Groene, William S. ; Miller, Jeffrey A. ; Mueller, Stephen N. ; Bamat, Michael K.

PV701 is a naturally-attenuated, non-recombinant, oncolytic strain of Newcastle disease virus that displays preclinical intravenous (IV) efficacy. PV701 is selective at killing human cancer cells versus normal human cells based on tumor specific defects in the interferon (IFN)-mediated antiviral response. This oncolytic virus displays a broad spectrum of antitumor activity in vitro and in vivo. Preclinical models successfully predicted key clinical parameters including the mechanism of toxicity, two complementary strategies (desensitization and slow infusion) to reduce toxicity, and the starting dose for phase 1 trials. In three phase 1 trials of 114 patients using IV administration of PV701, Wellstat Biologics Corporation has evaluated the effects of dose, schedule, and infusion rate for PV701. Three general classes of side effects were seen: flu-like symptoms; tumor-site-specific adverse events (AEs); and infusion reactions. The first PV701 dose desensitized the patient to the side effects of further doses, allowing a marked increase in the maximum tolerated dose for subsequent doses compared to the first dose. Tumor responses were first noted at the higher doses achieved using desensitization. In the most recent phase 1 trial of 19 patients at Hamilton, Ontario, that employed desensitization, high repeat doses, and a slower infusion rate (Hamilton Regimen), there were six responses (4 major; 2 minor) and a total of six patients with survival for at least 2 years. In addition, patient tolerability improved using the Hamilton Regimen compared to IV bolus dosing used previously. Phase 2 studies of this novel biologic agent are about to begin.

01 Dec 2006·Neurobiology of DiseaseQ2 · MEDICINE

Oral uridine pro-drug PN401 is neuroprotective in the R6/2 and N171-82Q mouse models of Huntington's disease

Q2 · MEDICINE

Article

Author: Saydoff, Joel A. ; Garcia, Rolando A. G. ; Browne, Susan E. ; Liu, Liansheng ; Sheng, Jin ; Brenneman, Denise ; Hu, Zhongyi ; Cardin, Sylvain ; Gonzalez, Alexis ; von Borstel, Reid W. ; Gregorio, Jason ; Burr, Holly ; Beal, M. Flint

Previously, uridine pro-drug 2',3',5'-tri-O-acetyluridine (PN401) was shown to be protective in the mitochondrial complex II inhibitor 3-nitropropionic acid model of Huntington's disease (HD). In this study, PN401 increased survival and improved motor function on the rotarod in both R6/2 and N171-82Q polyglutamine repeat mouse models of HD. PN401 significantly decreased neurodegeneration in both the piriform cortex and striatum although PN401 decreased huntingtin protein aggregates only in the striatum. Cortical and striatal brain-derived neurotrophic factor (BDNF) protein levels were reduced in the +/- compared to the -/- N171-82Q mice and PN401 treatment significantly increased cortical BDNF in both +/- and -/- mice, but PN401 did not affect striatal BDNF. These results suggest that PN401 may have beneficial effects in the treatment of neurodegenerative diseases such as HD.

20 Dec 2005·Brain ResearchQ4 · MEDICINE

Severe cytochrome c oxidase inhibition in vivo does not induce a pyrimidine deficiency; neuroprotective action of oral uridine prodrug PN401 requires supraphysiological levels of uridine

Q4 · MEDICINE

Article

Author: Garcia, Rolando A. G. ; Liu, Liansheng ; Hu, Zhongyi ; Gonzalez, Alexis ; von Borstel, Reid W. ; Saydoff, Joel A.

It has been hypothesized that mitochondrial respiratory chain dysfunction leads to a pyrimidine deficiency since the pyrimidine biosynthetic enzyme dihydroorotate dehydrogenase is coupled to the electron transport chain. The uridine prodrug triacetyluridine (PN401) is neuroprotective in several models of neurodegenerative disease involving respiratory chain toxins. Therefore, the therapeutic effects of PN401 might involve the correction of a pyrimidine deficiency secondary to respiratory chain impairment. We infused mice with the cytochrome c oxidase inhibitor azide, which inhibited brain complex IV activity. Chronic infusion of azide for 2 or 14 days induced significant toxicity and mortality but did not cause a pyrimidine deficit in the brain. In contrast, the pyrimidine synthesis inhibitor N-phosphonoacetyl-l-aspartate (PALA) produced a pyrimidine deficit with minimal mortality. Treatment with 6% PN401 decreased mortality and cerebrocortical apoptosis caused by azide. Previously, we found that optimal neuroprotection against mitochondrial complex II inhibition required 4-6% PN401. PN401 at 1, 3, 6 and 10% in chow induced nonlinear increases in plasma uridine with 6% PN401 elevating plasma uridine up to 80 muM, and these higher micromolar uridine levels were also required for neuroprotection in chemical hypoxia models in vitro. Our results indicate that severe complex IV inhibition in vivo does not lead to a pyrimidine deficiency, and therefore the protective effect of PN401 in the azide toxin model is not mediated through the correction of a pyrimidine deficiency. Furthermore, supraphysiological levels of uridine are required to produce optimal protective effects in disorders involving impairment of mitochondrial respiratory complex II or IV.

News (Medical) associated with Wellstat Therapeutics Corp.

13 Jul 2023

·www.biospace.com

SERB Pharmaceuticals acquires Vistogard® and Xuriden® from Wellstat Therapeutics

West Conshohocken, PA, July 13, 2023 (GLOBE NEWSWIRE) -- SERB Pharmaceuticals, a global specialty pharmaceutical company, announced today that it has acquired two life-saving medicines, Vistogard® (uridine triacetate) and Xuriden® (uridine triacetate), from Wellstat Therapeutics. Both medicines are commercially available in the US and currently distributed across Europe on a named patient or compassionate use basis. Vistogard® is an emergency treatment for adult and pediatric patients following a fluorouracil (5-FU) or capecitabine overdose regardless of the presence of symptoms, or who exhibit early-onset, severe or life- threatening toxicity affecting the cardiac or central nervous system, and/or early-onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of 5-FU or capecitabine administration.[i] 5-FU and capecitabine are chemotherapy agents commonly used to treat a wide variety of solid tumors. Xuriden® is a treatment for adult and pediatric patients with hereditary orotic aciduria (HOA)[ii], an ultra-rare, orphan genetic disorder associated megaloblastic anemia as well as failure to thrive, developmental delay, epilepsy, susceptibility to infection, and orotic acid crystals in the urine.[iii] Jeremie Urbain, President of SERB Pharmaceuticals, said: “We’re proud to add these two treatments to our expanding portfolio of medicines for complex, life-threatening conditions. Acquiring these medicines is in line with our strategy to focus on emergency care and rare diseases.” The products will be sold in the US by BTG Pharmaceuticals, SERB’s US entity. The products will transition seamlessly from Wellstat to BTG Pharmaceuticals and will continue to be available from the same distributors via the same ordering processes: To order Vistogard®, please call 1-800-914-0071. Vistogard® is distributed through Cardinal Health Specialty Pharmaceutical Distribution, available 24 hours/day, 7 days/week. Vistogard® and Xuriden® are available for dispensing through BioMatrix Specialty Pharmacy. To reach BioMatrix Specialty Pharmacy, please call: 844-374-0604. BTG Pharmaceuticals, a SERB company, will promote Vistogard® and continue promoting Voraxaze® through its oncology field force in the US. The companies did not disclose specific financial terms. Today’s news follows the announcement in May that SERB has acquired the exclusive US rights to bentracimab, which is in development as a ticagrelor reversal agent. About Fluorouracil (5-FU) and capecitabine toxicity 5-FU and capecitabine are both trusted chemotherapies received by thousands of patients, often in combination with other chemotherapy agents or radiation. They have a history of success in treating a range of cancers including breast, colorectal (bowel), gastric (stomach), and pancreatic cancer. According to the National Institute of Health (NIH), about 275,000 patients in the US receive 5-FU every year. Capecitabine, a prodrug that is enzymatically converted to 5-FU within the body, has about 60,000 treatment packages sold per year. Approximately 3% of these patients develop serious toxicities and more than 8,000 toxic reactions and 1,300 deaths every year are attributed to 5-FU and capecitabine..[iv] About HOA Hereditary orotic aciduria (HOA) is a very rare inborn error of pyrimidine metabolism caused by mutations reducing the activity of uridine monophosphate synthase (UMPS), which converts orotic acid to UMP. Uridine deficiency underlies many of the symptoms of HOA. When untreated, patients with HOA may develop a blood (hematologic) disorder called megaloblastic anemia as well as exhibiting failure to thrive, developmental delay, epilepsy, susceptibility to infection and orotic acid crystals in the urine (crystalluria).iii About SERB Pharmaceuticals SERB is a global specialty pharmaceutical company with a growing portfolio of medicines for emergency care and rare diseases. For over 30 years we have made treating these complex and life-threatening conditions possible, supporting clinicians, healthcare systems and governments while offering hope to patients and their families. SERB has the broadest antidote portfolio in the world, including medical countermeasures for chemical, biological, radiological and nuclear (CBRN) risks. As a fully integrated company, we have the experience and capabilities to acquire, develop, and manufacture our medicines to the highest standards, and make them available worldwide through our secure supply chain. SERB operates in the US as BTG Pharmaceuticals. Learn more at . About Vistogard® Indication VISTOGARD® is indicated for the emergency treatment of adult and pediatric patients: following a fluorouracil or capecitabine overdose regardless of the presence of symptoms, or who exhibit early-onset, severe or life- threatening toxicity affecting the cardiac or central nervous system, and/or early onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of fluorouracil or capecitabine administration. Limitations of use VISTOGARD® is not recommended for the non–emergent treatment of adverse reactions associated with fluorouracil or capecitabine because it may diminish the efficacy of these drugs. The safety and efficacy of VISTOGARD initiated more than 96 hours following the end of fluorouracil or capecitabine administration have not been established. Important Safety Information In clinical studies, adverse reactions occurring in >2% of patients receiving VISTOGARD® included vomiting (10%), nausea (5%), and diarrhea (3%). One person receiving uridine triacetate experienced grade 3 nausea and vomiting. VISTOGARD® was discontinued for adverse reactions in 2 (1.4%) patients. See full prescribing information. About Xuriden® Indication XURIDEN® is indicated in adult and pediatric patients for the treatment of hereditary orotic aciduria. Important Safety Information Contraindications – None. Warning and Precautions – None. Adverse reactions – No adverse reactions were reported with XURIDEN in patients with HOA. Pregnancy – There are no available data on XURIDEN use in pregnant women to inform drug-associated risk. Lactation– There are no data on the presence of XURIDEN in human milk, the effect on the breast-fed infant or the effect on milk production. The development and health benefits of breastfeeding should be considered along with the mother’s clinical need for XURIDEN and any potential adverse effects on the breastfed infant from XURIDEN or from the underlying maternal condition. Pediatric – The safety and effectiveness of XURIDEN have been established in pediatric patients. See full prescribing information. References [i] Wellstat Therapeutics Corporation (2017). Vistogard (uridine triacetate) US Package Insert. [ii] Wellstat Therapeutics Corporation (2019). Xuriden (uridine triacetate) US Package Insert. [iii] Al Absi HS, Sacharow S, Al Zein N, Al Shamsi A, Al Teneiji A. Hereditary orotic aciduria (HOA): A novel uridine-5-monophosphate synthase (UMPS) mutation. Mol Genet Metab Rep. 2021 Jan 9;26:100703. doi: 10.1016/j.ymgmr.2020.100703. PMID: 33489760; PMCID: PMC7807243. [iv] Santos C, Morgan BW, Geller RJ. The successful treatment of 5-fluorouracil (5-FU) overdose in a patient with malignancy and HIV/AIDS with uridine triacetate. Am J Emerg Med. 2017 May;35(5):802.e7-802.e8. doi: 10.1016/j.ajem.2016.11.038. Epub 2016 Nov 15. PMID: 27884585. Christopher W Sampson SERB Pharmaceuticals +44 7773 251 178 chris.sampson@serb.com

License out/inDrug Approval

01 Sep 2021

·www.globenewswire.com

DelveInsight Estimates a Promising Gout Pipeline Owing to

Los Angeles, USA, Aug. 31, 2021 (GLOBE NEWSWIRE) -- DelveInsight Estimates a Promising Gout Pipeline Owing to an Expected Entry of Novel Therapeutic Agents Around 40+ key companies are developing therapies for Gout. Selecta Biosciences appears to have the upper leg being the only company with its Gout drug candidate in the most advanced stage (Phase III). DelveInsight’s ‘Gout Pipeline Insights’ report offers comprehensive coverage of the emerging Gout therapeutics landscape in different stages of development from pre-clinical till a late-end stage, along with dormant, inactive, and abandoned drugs agents. The Gout Pipeline report provides a holistic picture of the ongoing clinical trials, partnerships taking place in the domain, recent happenings in space, and growth prospects across the Gout domain. Some of the key highlights from the Gout Pipeline report: Request for Sample to know more about the therapies that are set to grab maximum patient pool @ Gout Emerging Therapies and Forecast Gout is a chronic disease that is caused due to excess accumulation of uric acid in the body, a condition referred to as hyperuricemia. It is a painful and crystal-induced form of arthritis in which crystals of monosodium urate (MSU) precipitate and deposit in joints of lower limbs. Gout is treatable with medications and changes in diet and lifestyle. For more information on emerging drugs, visit Gout Pipeline Analysis Gout Pipeline Drugs Request for Sample to know more @ Gout Pipeline Analysis, Key Companies, and Futuristic Trends Gout Therapeutic Assessment The Gout Pipeline report lays down complete insights into active Gout pipeline therapies segmented by Stage, Product Type, Route of Administration, Molecule Type, Target, and Mechanism of Action. By Product Type By Stage By Molecule Type By Route of Administration By Mechanism of Action By Targets Get in touch with our Business executive for Informative Business Decisions, Licensing Services and Consulting Solutions Scope of the Report Coverage: GlobalKey Players: Protalix BioTherapeutics, Shanton Pharma, Horizon Therapeutics, JW Pharmaceutical, Jiangsu Hengrui Medicine Co., Nippon Chemiphar, Sinovent, LG Chem, Selecta Biosciences, TWi Biotechnology, Olatec Therapeutics, Hinova pharmaceuticals, Dyve Biosciences, InventisBio, Arrowhead Pharmaceuticals, Wellstat Therapeutics Corporation, Acquist Therapeutics, TaiwanJ Pharmaceuticals, Atom Bioscience Key Gout Pipeline Therapies: PRX-115, SAP-001, HZN-003, URC-102, SHR4640, NC-2700, XNW3009, LC350189, SEL-212, AC-201, Dapansutrile, HP501, DYV700, D-0120, ARO-XDH, WT-2107, ACQT1127, TJC0434, ABP-072, and many others. Reach out @ Gout Pipeline: Novel therapies and Emerging technologies Table of Contents Visit to know more of what’s covered @ Gout Emerging Therapies, Treatments and Ongoing Clinical Trials Related ReportsAcute Gout Flare MarketDelveInsight's "Acute gout Flare - Market Insights, Epidemiology, and Market Forecast-2030" report. Chronic Refractory Gout MarketDelveInsight's "Chronic Refractory Gout - Market Insights, Epidemiology, and Market Forecast-2030" report. Gout MarketDelveInsight's "Gout - Market Insights, Epidemiology, and Market Forecast-2030" report. Hyperkalemia Japan MarketDelveInsight’s ‘Hyperkalemia —Market Insights, Epidemiology, and Market Forecast–2030’ report. Acute Pancreatitis Market SizeDelveInsight's "Acute Pancreatitis (AP) Market Insights, Epidemiology and Market Forecast-2028" report. Browse through blog postsAngelman syndrome PipelineAutoimmune Diseases Market and Key Pharma Players Involved About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. For more insights, visit Pharma, Healthcare, and Biotech News

100 Deals associated with Wellstat Therapeutics Corp.

100 Translational Medicine associated with Wellstat Therapeutics Corp.

Corporation Tree

Boost your research with our corporation tree data.

view full example data

Pipeline

Pipeline Snapshot as of 03 Jul 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Preclinical

Phase 2 Clinical

Approved

Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

Uridine Triacetate	Drug intoxication More	Approved
WT-501	Mitochondrial Myopathies More	Phase 2
WT-39006	Mycoses More	Preclinical
WT-35021	Solid tumor More	Preclinical
WT-2107	Gout More	Preclinical

Deal

Boost your decision using our deal data.

view full example data

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Profit

Explore the financial positions of over 360K organizations with Synapse.

view full example data

Grant & Funding(NIH)

Access more than 2 million grant and funding information to elevate your research journey.

view full example data

Investment

Gain insights on the latest company investments from start-ups to established corporations.

view full example data

Financing

Unearth financing trends to validate and advance investment opportunities.

view full example data

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis