An Open-Label, Phase 1b Safety and Efficacy Trial of Periganglionic Resiniferatoxin for the Treatment of Intractable Pain Due to Cancer-induced Bone Pain

Background:
Cancer-induced bone pain (CIBP) is common in people with cancer. Bone cancer can also lead to anxiety, depression, and reduced mobility and quality of life. Researchers believe a research drug called resiniferatoxin (RTX) may be able to help.
Objective:
To learn whether RTX is safe and can reduce cancer induced bone pain.
Eligibility:
People ages 18 and older with CIBP that is not relieved by standard treatments
Design:
Participants will have up to 6 outpatient visits over about 7 months. These will include:
Medical history
Physical exam
Blood and urine tests.
Thermal testing: a disk placed on the skin to test ability to sense temperature in and around the area of pain
Chest x-ray
EKG: stickers are placed on the chest to measure heart signals
ECG: measures electrical activity of the heart
Participants will have 1 inpatient visit lasting 2-4 days. This will include:
Catheter inserted into a vein in the arm. They are given anesthesia, sedation, and x-ray contrast.
A needle is passed through the skin of the back to inject the RTX.
Participants will keep a log of the pain medications they take after surgery.
Participants will be called 1 week and 2, 3, and 4 months after the injection.
Participants will be mailed surveys and questionnaires to complete 2, 3, and 4 months after the injection.

Start Date27 Aug 2026

Sponsor / Collaborator

National Institute of Neurological Disorders & Stroke

ChiCTR2600118758

/ SuspendedPhase 1IIT

A Phase I, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) Study and Food Affect (FE) study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Food Effect of RTX-117 in Healthy Volunteers

Start Date25 Feb 2026

Sponsor / Collaborator

Xili Technology (Shenzhen) Co., Ltd.

[+1]

ACTRN12625001215415

/ Not yet recruitingPhase 1

A Randomized, Placebo Controlled, Double-Blind, Single-Ascending Dose and Multiple-Ascending Dose First-in-Human Study to Investigate the Safety, Tolerability, and Pharmacokinetics, of Orally Administered RTX-001/101 in Healthy Adult Subjects

Start Date04 Nov 2025

Sponsor / Collaborator-

100 Clinical Results associated with Resiniferatoxin

100 Translational Medicine associated with Resiniferatoxin

100 Patents (Medical) associated with Resiniferatoxin

3,112

Literatures (Medical) associated with Resiniferatoxin

31 Dec 2026·RENAL FAILURE

Long-term real-world outcomes of rituximab in primary membranous nephropathy: a 154-patient cohort study from China

Article

Author: Fang, Ying-xin ; Shang, Wen-ya ; Zheng, Zhen-feng ; Wei, Li ; Cao, Xin ; Jia, Jun-ya ; Zhang, Xin-xin ; Yan, Tie-kun ; Xu, Peng-cheng ; Qi, Yan ; Gao, Xi-qian ; Gu, Qiu-hua ; Cheng, Xi

To investigate the long-term efficacy and safety of rituximab (RTX) in treating patients with primary membranous nephropathy (pMN), a total of 154 patients with pMN receiving RTX treatment were retrospectively enrolled in this study. The study included 92 patients who received RTX as an initial therapy, and 62 patients who received it as an alternative therapy. Over a median follow-up of 30.0 (24.0, 47.0) months after RTX treatment, 102/121 (84.3%) patients achieved immunological remission. And 128/154 (83.1%) patients achieved clinical remission (including 37.7% of CR and 45.5% of PR), accompanied by significant reduction in proteinuria and increase in serum albumin levels. Clinical response rates were comparable between the initial and alternative therapy groups (87.0 vs. 77.4%, p = 0.131), though patients in the alternative therapy group required a longer time to achieve remission. Among the 21 patients with eGFR < 60 mL·min^-1·(1.73 m²)^-1, 12 (57.1%) responded to RTX. Though two patients progressed to end-stage renal disease (ESRD) during follow-up, the remaining 19 patients showed an improvement in eGFR from 48.0 (38.0, 55.0) to 62.0 (36.0, 73.0) mL·min^-1·(1.73 m²)^-1. The clinical response in the RTX monotherapy group did not differ significantly from that in patients who received RTX in combination with glucocorticoids or other immunosuppressive agents. Twenty-eight patients experienced adverse events, most of which were mild and manageable. RTX, utilized as either an initial or alternative therapy, induced a significant clinical response in patients with pMN, including those with compromised renal function. These findings support RTX as an effective and well-tolerated option across various pMN patient profiles.

31 Dec 2026·RENAL FAILURE

The successful use of rituximab in children with IgA nephropathy and minimal change-like lesions: a case series

Article

Author: Hu, Manman ; Cheng, Yibo ; Wang, Hui ; Cui, Xueying ; Fu, Qian ; Jiang, Yeping ; Chang, Menglin

IgA nephropathy with minimal change disease (IgAN-MCD) is a rare glomerulopathy combining features of both entities, typically presenting as steroid-sensitive nephrotic syndrome (NS) with frequent relapses. While rituximab (RTX) is commonly used in pediatric glomerular diseases, its efficacy in IgAN-MCD remains unclear. Our retrospective study evaluated the efficacy and safety of RTX in eight children with IgAN-MCD who remained steroid-dependent or frequently relapsed despite treatment with glucocorticoids and second-line immunosuppressants before RTX. Over a follow-up period of 12-41 months, RTX significantly reduced the annual relapse rate by 1.1 episodes per year, prolonged remission by 3.5 months, and extended the steroid-free interval by 7.4 months. Overall, 87.5% of patients successfully discontinued glucocorticoids, and 50% stopped immunosuppressants. Three patients relapsed at 9, 16, and 19 months, likely due to insufficient CD19⁺ B-cell monitoring and lack of consolidation dosing, yet all achieved sustained remission by the final follow-up. No serious RTX-related adverse events were observed. These findings suggest that RTX effectively reduces relapse, prolongs remission, and decreases dependence on steroids and immunosuppressants in pediatric IgAN-MCD, with a favorable safety profile for refractory cases.

01 Aug 2026·LIFE SCIENCES

Sensory nerve desensitisation exerts differential effects on the severity of acute pancreatitis in rodent models

Article

Author: Kiss, Lóránd ; Kui, Balázs ; Pallagi, Petra ; Joó, Emese Réka ; Kormányos, Eszter Sára ; Rakonczay, Zoltán ; Venglovecz, Viktória ; Balla, Zsolt ; Fűr, Gabriella Mihalekné ; Hegyi, Péter ; Helyes, Zsuzsanna ; Maléth, József ; Pintér, Erika

Acute pancreatitis (AP) is a common gastroenterological disorder characterized by severe abdominal pain and inflammation. Despite its high mortality rate, the pathomechanism of AP remains incompletely understood, although neurogenic inflammation - where nerve-derived mediators trigger or amplify inflammatory responses - also appears to contribute to its pathogenesis. The aim of this study was to further investigate the role of neurogenic inflammation in AP and the impact of sensory nerve desensitisation on disease severity. Sensory afferent neurons were ablated using resiniferatoxin (RTX) prior to AP induction in four distinct rat and mouse models. Additionally, TRPV1 knock-out mice were employed to assess the contribution of this ion channel to AP development. Our findings reveal that RTX-induced sensory nerve desensitisation exacerbates the severity of necrotising AP induced by L-ornithine (L-Orn) and sodium taurocholate (NaTc) in rats, as evidenced by increased tissue damage, leukocyte infiltration or serum amylase activity. Conversely, in the cerulein (Cer)-induced oedematous AP rat model, RTX treatment significantly reduced leukocyte infiltration without affecting tissue oedema. In mice, RTX administration worsened the severity of Cer-induced AP, while TRPV1 gene deletion led to a modest reduction in disease severity. These results suggest that the impact of sensory nerve disruption on AP varies depending on the model and type of AP. The study underscores the important role of sensory neurons and TRPV1 ion channels in the pathogenesis and progression of AP, highlighting the potential for targeted therapies that modulate neurogenic inflammation.

News (Medical) associated with Resiniferatoxin

12 Feb 2026

·www.biospace.com

Aardvark Therapeutics Announces Establishment of New U.S. Subsidiary to Support Development of Its Dermatology Pipeline; Bryan Jones Named Chief Executive Officer

SAN DIEGO, Feb. 12, 2026 (GLOBE NEWSWIRE) -- Aardvark Therapeutics, Inc. (Aardvark) (Nasdaq: AARD), a clinical-stage biopharmaceutical company focused on developing novel, small-molecule therapeutics to activate innate homeostatic pathways for the treatment of metabolic diseases, today announced that it has established Ardia Therapeutics, Inc. (Ardia), a new wholly-owned U.S. subsidiary. Ardia will support development of a new dermatology pipeline focused on lead asset, DIA-615, a potential topical treatment for a variety of inflammatory skin diseases, including psoriasis. Bryan Jones, Ph.D., has been named Chief Executive Officer of Ardia and has transitioned out of his current role at Aardvark as Chief Operating Officer to lead Ardia. “We are proud to take this important step toward establishing Ardia as a new entity and having Bryan take the helm to advance our lead dermatologic asset, DIA-615, into the clinic,” said Tien Lee, M.D., Founder and Chief Executive Officer of Aardvark. “Bryan has made significant contributions to our success at Aardvark over the past five years, and we are excited to announce his appointment to Chief Executive Officer of Ardia. We believe he is the right leader to develop Ardia’s pipeline, which is focused on addressing serious unmet needs in severe skin diseases.” Dr. Jones brings over 30 years of experience with leading biotech and specialty pharmaceutical companies, including roles in product and business development. He has implemented multiple development programs during his successful career. “I am honored to take on this new role at Ardia, and I am excited by the potential of DIA-615 and the opportunity to grow Ardia’s dermatology portfolio which could have a lasting impact on patients,” said Dr. Jones. “I am very proud of what the Aardvark team has accomplished over the past several years and look forward to unlocking the value of DIA-615." Dr. Jones joined Aardvark in 2021 as Chief Business Officer and transitioned to Chief Operating Officer in August 2022. Prior to Aardvark, he was Chief Operating Officer and Co-Founder of Sollis Therapeutics, where he led the technology transfer from Medtronic, the manufacturing of a drug/device combination and the execution of the Phase 3 sciatica program. Before that, he was Vice President of Operations at Sorrento Therapeutics, where he led the resiniferatoxin program, including manufacturing, toxicology and clinical planning as well as being involved in both monoclonal antibody production, antibody drug conjugate research and cell therapy development. He has held roles of increasing responsibility, including Chief Operating Officer of two startup companies: Sherrington and Mt. Cook. Dr. Jones received his Ph.D. in genetics from the University of Washington and a bachelor’s degree in biology and biochemistry from Iowa State University. About Ardia Therapeutics, Inc. Ardia Therapeutics is a wholly-owned subsidiary of Aardvark Therapeutics, Inc. The company is focused on the development of DIA-615, a clinic-ready topical drug intended to treat a variety of inflammatory skin diseases, including psoriasis. The active ingredient induces specific members of a G protein-coupled receptor (GPCR) family, called TAS2Rs, to downregulate stressed endoplasmic reticulum in inflammatory cells. DIA-615 is designed to localize in the skin and target inflammatory cells that induce the lesions associated with skin diseases. About Aardvark Therapeutics, Inc. Aardvark is a clinical-stage biopharmaceutical company developing novel, small-molecule therapeutics designed to suppress hunger for the treatment of Prader-Willi Syndrome (PWS) and metabolic diseases. Hunger, which is the discomfort from not having eaten recently, is a distinct neural signaling pathway separate from appetite, the reward-seeking desire for food. Our programs explore therapeutic applications in hunger-associated indications and potential complementary uses with anti-appetite therapies. Our lead compound, oral ARD-101, is in Phase 3 clinical development for the treatment of hyperphagia associated with PWS, a rare disease characterized by insatiable hunger. Aardvark is also developing ARD-201, a planned fixed-dose combination of ARD-101 with a DPP-4 inhibitor, through two separate Phase 2 trials with a goal of addressing some of the limitations of currently marketed GLP-1 therapies for obesity and obesity-related conditions. For more information, visit . Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include, but are not limited to, statements concerning: Aardvark’s and Ardia’s business strategy, product candidates, ongoing clinical trials, planned clinical trials, expected timing for data readouts and reporting interim, preliminary or topline results, likelihood of success, as well as plans and objectives of management for future operations. The words, without limitation, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these or similar identifying words. Forward-looking statements in this press release include statements regarding DIA-615, including its potential as a topical treatment for a variety of inflammatory skin diseases, including psoriasis, the potential growth of Ardia’s dermatology portfolio and the potential value of DIA-615. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties related to potential delays in the commencement, enrollment and completion of clinical trials; the risk that Aardvark may use its capital resources sooner than expected and that they may be insufficient to allow Aardvark to achieve its anticipated milestones; the possibility that the past track records of Aardvark and its personnel may not be repeated or indicative of future success; risks related to its dependence on third parties for manufacturing, shipping and production of drug product for use in clinical trials and preclinical studies; the risk of unfavorable clinical trial results; the risk that results from earlier clinical trials and preclinical studies may not necessarily be predictive of future results; and other risks and uncertainties, including the factors described under the “Risk Factors” section of Aardvark’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2025 that Aardvark filed with the Securities and Exchange Commission on November 13, 2025. When evaluating Aardvark’s business and prospects, careful consideration should be given to these risks and uncertainties. Any forward-looking statements contained in this press release are based on the current expectations of Aardvark’s management team and speak only as of the date hereof, and Aardvark specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise, unless required by law. Investor Contact: Courtney Mogerley Argot Partners (212) 600-1902 Aardvark@Argotpartners.com Media Contact : Andrea Cohen Sam Brown LLC (917) 209-7163 Andreacohen@Sambrown.com

Executive ChangePhase 2Phase 3

19 Mar 2025

·www.ligandtracer.com

The Dynamic Power of Rituximab: Beyond Simple Affinity

Rituximab (RTX) binding to CD20 isn’t a straightforward equilibrium process. It is a dynamic, concentration-dependent interaction that goes beyond traditional affinity measurements.RTX is either mono- or bivalently bound to CD20 on the cell surface and it rapidly exchanges between these two states with the monovalent state prevailing at a high RTX dose while retention is enhanced at low RTX concentrations as more antibody molecules engage in bivalent interactions. Real-time interaction analysis on live cells revealed this concentration dependent mechanism that correlates with the ability to induce complement-dependent cytotoxicity (CDC). Describing RTX binding with a single affinity value overlooks the dynamic stabilization effect that plays a pivotal role in its mode of action. Binding kinetics on live cells provide a more complete picture of antibody performance that better correlates with functional properties.

22 Oct 2024

·pipelinereview.com

First participants enrolled in first-in-human Phase I clinical trial with nociceptin (NOP) receptor agonist

AACHEN, Germany I October 22, 2024 I Today, Grünenthal announced that the first participants have been enrolled in a first-in-human Phase I clinical trial for a nociceptin (NOP) receptor agonist. The trial will include 90 healthy volunteers and aims to demonstrate a favourable safety and tolerability profile and to confirm the pharmacokinetic characteristics of the compound after single and multiple ascending doses. The results of the trial are expected in Q3 2025. “Grünenthal pioneered the research into NOP receptor agonists to deliver a unique and transformative first-in-class therapy option to millions of patients suffering from chronic pain,” says Gillian Burgess, Head of Research, Grünenthal. “With a unique mechanism of action for treating chronic pain, these molecules have the potential to deliver robust pain relief combined with an improved safety profile compared to the available standard of care.” Pre-clinical data show that NOP receptor agonists have the potential to act as potent analgesics without abuse liability. 1 Leveraging the clinical data Grünenthal obtained during the development of its NOP receptor programme, Grünenthal now brings forward a candidate that shows best-in-class potency and selectivity for the NOP receptor. These properties are predicted to provide robust pain relief in a broad range of chronic pain indications without the serious central nervous system related side effects associated with available opioids. Grünenthal’s R&D pipeline includes multiple programmes across different stages, targets, modalities, and mechanisms of action to deliver innovative treatment options for patients suffering from pain and related diseases. Recently, a Phase I clinical trial with a Glucocorticoid Receptor Modulator (GRM) has been completed. The compound is developed to provide patients with a therapy option for chronic inflammatory diseases. In addition, Grünenthal is running a Phase III clinical trial with Qutenza ® (capsaicin) 8% topical system in post-surgical neuropathic pain, aiming to expand its label in the United States. A global Phase III programme investigating the efficacy, safety and tolerability of Resiniferatoxin in patients with painful osteoarthritis of the knee is currently ongoing. About the NOP Receptor The nociceptin (NOP) receptor is a G protein-coupled receptor whose natural ligand is the 17 amino acid neuropeptide known as nociceptin (N/OFQ). 2 NOP Receptor agonists have been shown to act as potent analgesics without abuse liability in pre-clinical models. 1 Although the NOP Receptor shares some sequence identity (~60%) with the opioid receptors μ-OP (MOP), κ-OP (KOP), and δ-OP (DOP), they possess little or no affinity for opioid peptides or morphine-like compounds. Likewise, opioid receptors possess little affinity towards NOP’s endogenous ligand nociceptin. 3 About Grünenthal Grünenthal is a global leader in pain management and related diseases. As a science-based, fully integrated pharmaceutical company, we have a long track record of bringing innovative treatments and state-of-the-art technologies to patients worldwide. Our purpose is to change lives for the better – and innovation is our passion. We are focusing all our activities and efforts on working towards our vision of a World Free of Pain. Grünenthal is headquartered in Aachen, Germany, and has affiliates in 27 countries across Europe, Latin America, and the U.S. Our products are available in approx. 100 countries. In 2023, Grünenthal employed around 4,400 people and achieved revenues of €1.8 billion. More information: https://www.grunenthal.com Follow us on: LinkedIn: Grunenthal Group Instagram: grunenthal 1 Lin AP, Ko MC. The therapeutic potential of nociceptin/orphanin FQ receptor agonists as analgesics without abuse liability. ACS Chem Neurosci. 2013 Feb 20;4(2):214-24. doi: 10.1021/cn300124f. Epub 2012 Nov 6. PMID: 23421672; PMCID: PMC3582300. 2 Henderson G, McKnight AT (August 1997). “The orphan opioid receptor and its endogenous ligand– nociceptin/orphanin FQ”. Trends in Pharmacological Sciences. 18 (8): 293–300. doi:10.1016/S0165- 6147(97)90645-3. PMID 9277133. 3 Butour JL, Moisand C, Mazarguil H, Mollereau C, Meunier JC (February 1997). “Recognition and activation of the opioid receptor-like ORL 1 receptor by nociceptin, nociceptin analogs and opioids”. European Journal of Pharmacology. 321 (1): 97–103. doi:10.1016/S0014-2999(96)00919-3. PMID 9083791. SOURCE: Grünenthal Group

Phase 1Phase 3Phase 2

100 Deals associated with Resiniferatoxin

External Link

KEGG	Wiki	ATC	Drug Bank
-	Resiniferatoxin	-	DB06515

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Pain	Phase 3	Japan	Grünenthal GmbH	26 Aug 2022
Pain	Phase 3	Canada	Grünenthal GmbH	26 Aug 2022
Pain	Phase 3	Czechia	Grünenthal GmbH	26 Aug 2022
Pain	Phase 3	France	Grünenthal GmbH	26 Aug 2022
Pain	Phase 3	Germany	Grünenthal GmbH	26 Aug 2022
Pain	Phase 3	Italy	Grünenthal GmbH	26 Aug 2022
Pain	Phase 3	Mexico	Grünenthal GmbH	26 Aug 2022
Pain	Phase 3	Netherlands	Grünenthal GmbH	26 Aug 2022
Pain	Phase 3	Poland	Grünenthal GmbH	26 Aug 2022
Arthritis Pain	Phase 3	United States	NovaQuest Capital Management LLC	30 Jan 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05449132 (CTgov)	Phase 3	Pain \| Osteoarthritis, Knee	466	RTX-GRT7039 (RTX-GRT7039)	wujppxxzat(wqtdxvulsq) = lsgooihroj yuhyydzvmc (qriyedkrou, 0.16) View more	-	18 Mar 2026
				Placebo (Placebo)	wujppxxzat(wqtdxvulsq) = liucuhtaqf yuhyydzvmc (qriyedkrou, 0.17) View more
NCT05377489 (CTgov)	Phase 3	Pain \| Osteoarthritis, Knee	714	RTX-GRT7039	crcusdderu = fbgotwpbgi hohzsprrbb (qirgcigwei, npnfiherqn - gkafqtnhgv) View more	-	28 Nov 2025
NCT05248386 (CTgov)	Phase 3	Pain \| Osteoarthritis, Knee	469	RTX-GRT7039 (RTX-GRT7039)	pgkazfgflb(wzsxpaumwx) = bbukfkiyrv gcuwyydzvp (lmaisfsmar, 0.15) View more	-	12 Aug 2025
				Placebo (Placebo)	pgkazfgflb(wzsxpaumwx) = uvfdlfzgrm gcuwyydzvp (lmaisfsmar, 0.15) View more
NCT04885972 (EULAR 12024 \| EULAR 22024) Manual	Phase 2	Osteoarthritis, Knee	67	RTX-GRT7039 0.5 mg	wvlpfvtobz(udhzuspxod) = qeommnmmzk xpphtuduqe (evvjharzaf, 19.79) View more	Positive	14 Jun 2024
				RTX-GRT7039 2 mg	wvlpfvtobz(udhzuspxod) = mkozidmjgo xpphtuduqe (evvjharzaf, 34.16) View more

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