Phase 3, Multicenter, Randomized, Subject and Evaluator Blinded, Controlled Study Evaluating the Safety and Efficacy of NTX-001 Compared to Standard of Care (Neurorrhaphy) in the Treatment of Upper Extremity Transected Nerves Requiring Surgical Repair.

NTX-001 is a single use surgical product intended for use in conjunction with standard suture neurorrhaphy of severed nerves in patients between 18 and 80.

Start Date01 Jan 2025

Sponsor / Collaborator

Neuraptive Therapeutics, Inc.Startup

NCT04789044 / RecruitingPhase 2IIT

A Phase 2a Multicenter Randomized Controlled Trial Evaluating the Safety and Efficacy of Polyethylene Glycol (PEG) Mediated Fusion (PEG Fusion) Compared to Standard of Care in the Repair of Mixed Motor-sensory Acute Peripheral Nerve Injuries (PNI) for Rapid and Immediate Improvement in Outcome

The overall objective of this study is to determine the safety of PEG fusion when used with primary repair or reconstruction in patients with an acute upper extremity peripheral nerve injury. PEG is safe and effective for extending the half-life of circulating pharmaceutical products, when used in conjunction with a topical hemostatic agent in surgical wounds, and when used as a colon cleanser for endoscopic surgical procedures. However, PEG fusion has not been rigorously tested as a safe reagent to promote nerve regeneration in humans. Therefore, the goal of this Phase 2a clinical trial is to establish safety data and to examine the effect of PEG fusion on clinical outcomes including recovery of sensory and motor function. Results will be externally validated using data collected in the DoD funded prospective NERVE study and will provide preliminary evidence to power a larger phase II efficacy trial.

Start Date01 Jun 2023

Sponsor / Collaborator

Defense Language Institute Foreign Language Center

NCT05293522 / TerminatedPhase 2

A Phase 2a, Multicenter, Randomized, Subject and Evaluator Blinded, Controlled Study Evaluating the Safety and Efficacy of NTX-001 Compared to Standard of Care in the Treatment and Prevention of Facial Paralysis Requiring Surgical Repair.

This study involves the use of an Investigational Product called NTX-001. It is a product used in the repair of nerve injuries. It is used in the operating room. The main purposes of this study are to 1) see how safe NTX-001 is when used in nerve repair and, 2) see if your nerve becomes functional in a shorter period of time when compared to what is normally done to treat nerve injuries.

Start Date12 Jul 2022

Sponsor / Collaborator

Neuraptive Therapeutics, Inc.Startup

100 Clinical Results associated with NTX-001

100 Translational Medicine associated with NTX-001

100 Patents (Medical) associated with NTX-001

Literatures (Medical) associated with NTX-001

01 Nov 2020·Epilepsia

Progress report on new antiepileptic drugs: A summary of the Fifteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XV). I. Drugs in preclinical and early clinical development

Author: Koepp, Matthias J. ; Tomson, Torbjörn ; Perucca, Emilio ; Levy, René H. ; Perucca, Piero ; Johannessen, Svein I. ; White, H. Steve ; Bialer, Meir

AbstractSince 1992, the Eilat Conferences have provided a forum for all stakeholders in the epilepsy community to appraise the latest data on new antiepileptic drugs and emergency seizure treatments, including, in recent years, updates on progress with the development of novel monitoring and therapeutic devices. Because of the COVID‐19 pandemic, the Fifteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XV) was held as a fully virtual conference on July 27‐30, 2020 for the sessions on drugs and on August 3, 2020 for the sessions on devices, and was attended during the 5 days by >500 participants from 63 countries. This progress report summarizes key preclinical and initial (phase 1) clinical data on eight investigational treatments that are currently in early development, including 2‐deoxy‐D‐glucose, GAO‐3‐02, JNJ‐40411813, NBI‐921352, NTX‐001, sec‐butylpropylacetamide, XEN1101, and XEN496. This report provides an overview of current scenarios in the area of treatment discovery and development. The information presented illustrates a variety of innovative strategies, including exploration of compounds with novel mechanisms of action, transplantation of interneurons into epileptogenic brain regions, and the targeting of rare, previously neglected syndromes.

01 Dec 2019·BMC OphthalmologyQ4 · MEDICINE

Efficacy and safety of a novel naltrexone treatment for dry eye in type 1 diabetes

Q4 · MEDICINE

ArticleOA

Author: Zagon, Ian S ; Titunick, Michelle B ; Sassani, Joseph W ; McLaughlin, Patricia J

BACKGROUNDDry eye disease (DED) is a prevalent complication of diabetes and presents as reduced tear production and/or increased corneal surface sensitivity often with secondary ocular surface changes. This study examined the safety and efficacy of a proprietary new eye drop formulation for topical treatment of DED.METHODSType 1 diabetes (T1D) was established in male Sprague-Dawley rats to study the efficacy and safety of the investigational compound that contained 20 μg/ml of naltrexone (NTX). Tear production was measured by the Schirmer's 1 test, and ocular surface sensitivity was measured using an aesthesiometer. Diabetic rats received twice daily applications of a single drop (~ 0.02 ml) of the proprietary formulation (NTX-001) or vehicle onto one eye. For comparison, some diabetic rats received eye drops containing NTX in sterile Vigamox®. Safety was monitored by assessment of ocular histopathology in naïve male rats and naïve male rabbits receiving twice daily treatment of two drops for 30 days.RESULTSDry eye in T1D rats was reversed within hours of a single treatment of NTX-001, and over a period of 10 days NTX-001 restored corneal sensitivity and reversed dry eye relative to values measured in diabetic rats receiving vehicle. In comparison to NTX dissolved in Vigamox®, the proprietary NTX-001 was more effective at reversing dry eye. Safety studies in naïve rats and rabbits revealed no visible ocular pathology after 30 days of treatment.CONCLUSIONSAn investigational new eye drop containing 20 μg/ml NTX effectively reversed tear film deficits and restored corneal surface sensitivity in diabetic animals without causing toxic side effects.

News (Medical) associated with NTX-001

19 Sep 2024

·www.biospace.com

Neuraptive Therapeutics Announces Presentation of Phase 2 Topline Interim Results for NTX-001 at the 2024 American Society for Surgery of the Hand (ASSH) Annual Meeting

- Interim 24-week data from the NEUROFUSE study highlights the safety and efficacy of NTX-001 for peripheral nerve injuries. CHESTERBROOK, Pa.--(BUSINESS WIRE)--Neuraptive Therapeutics Inc., a leading biopharmaceutical company focused on novel treatments for peripheral nerve injuries, today announced that interim topline results from its ongoing Phase 2 NEUROFUSE Study will be presented at the 2024 American Society for Surgery of the Hand (ASSH) Annual Meeting, held September 19-21 in Minneapolis, MN. The presentation, titled Safety and Efficacy of NTX-001 in the Treatment of Acute Single Transected Peripheral Nerve Injuries: A Phase 2 Randomized Controlled Trial , was awarded a Top 10 Best Paper designation and will be presented by Dr. David Brogan of Washington University during the scientific paper session on Thursday, September 19. The presentation will highlight interim findings from the 24-week analysis of the NEUROFUSE study, which evaluates the safety and efficacy of NTX-001 in patients suffering from acute, single-transected upper extremity peripheral nerve injuries. NTX-001 is a drug-device combination designed to improve the speed and durability of functional recovery when used alongside standard surgical repair. These results represent an important milestone in the continued development of NTX-001 as a novel treatment option for peripheral nerve injuries. Key interim findings at 24 weeks include: NTX-001 demonstrated a significant improvement in MHQ total scores at 8- and 24 weeks post-operation compared to standard of care alone There were no safety findings associated with the use of NTX-001 At all-time points, patients treated with NTX-001 demonstrated significantly reduced post-operative pain compared with SOC alone. These results underscore the potential of NTX-001 to enhance functional recovery in peripheral nerve injury patients and support further development. According to Dr. Brogan, lead author on the paper and primary investigator at Washington University in St. Louis, “traumatic peripheral nerve transections are an unsolved problem in hand and upper extremity surgery, and the potential use of PEG fusion to meet this challenge has gained considerable interest among clinicians who deal with these devastating injuries. This data demonstrating improvement in pain and patient-reported outcomes is an exciting first step in addressing the needs of these patients.” “We are pleased to share these promising interim results from our Phase 2 trial of NTX-001 at such an important Scientific conference, and look forward to sharing full study results at future scientific conferences,” said Evan Tzanis, Chief Operating Officer and Head of R&D at Neuraptive Therapeutics, Inc. “We are immensely grateful to all those involved in this study, including the investigators, supporting staff at the clinical sites, our team, and most importantly, the patients who participated in the study,” said Tzanis. About ASSH The American Society for Surgery of the Hand is the oldest medical society dedicated to the hand and upper extremity. Its mission is to advance the science and practice of hand and upper extremity surgery through education, research, and advocacy on behalf of patients and practitioners. For more information, please visit . About Neuraptive Therapeutics, Inc. Neuraptive Therapeutics, Inc. is dedicated to the innovation and development of medical products and therapeutics to repair and regenerate peripheral nerves. The company is focused on addressing the unmet medical needs of patients and physicians dealing with the complex challenges of nerve injuries. Notice Regarding Forward-Looking Statements This press release contains forward-looking statements subject to various risks and uncertainties. Actual results could materially differ from those anticipated in the forward-looking statements due to various factors. Neuraptive Therapeutics, Inc. disclaims any obligation to update the information contained in these forward-looking statements. Contacts For more information about the NEUROFUSE Study or to inquire about Neuraptive Therapeutics, Inc., please contact: IR@neuraptive.com +1-484-787-3203

Phase 2Clinical Result

13 Sep 2024

·www.pharmaceutical-technology.com

Sanofi signs radioligand therapy licensing deal for rare cancers

Sanofi will advance the development of AlphaMedix along with RadioMedix and Orano Med. Credit: ArDanMe/Shutterstock. Sanofi has signed an exclusive licensing agreement with RadioMedix and Orano Med to advance the radioligand therapy AlphaMedix for rare cancers. US-based biotechnology company RadioMedix develops radiopharmaceuticals for positron emission topography imaging and targeted alpha therapy (TAT), targeting unmet medical needs in the cancer field. French company Orano Med specialises in lead-212 ( 212 Pb) radioligand therapies (RLTs) targeting cancer. The partnership is centred on AlphaMedix ( 212 Pb-DOTAMTATE), a late-stage project currently under evaluation for treating adult patients with unresectable or metastatic, progressive somatostatin-receptor expressing neuroendocrine tumours. AlphaMedix is a TAT comprising a somatostatin receptor-targeting peptide complex radiolabeled with 212 Pb which acts as an in vivo generator of alpha particles. See Also: FDA grants breakthrough therapy designation to Neuraptive’s NTX-001 FDA grants breakthrough therapy status to Arrowhead’s plozasiran The US Food and Drug Administration previously awarded breakthrough therapy designation for the asset to treat gastroenteropancreatic neuroendocrine tumours. This designation follows Phase I and II trials that demonstrated the therapy’s tolerability and significant tumour burden reduction, achieving a 62.5% durable response rate. AlphaMedix is in the final stage of Phase II clinical development. Sanofi will lead the worldwide commercialisation of the asset contingent on the receipt of approval. Orano Med will manage AlphaMedix’s manufacturing through its developing global industrial platform. RadioMedix and Orano Med will receive €100m upfront payment from Sanofi. Sanofi will pay up to a further €220m based on sales-based milestones apart from royalty payments. Sanofi development global head and chief medical officer Dietmar Berger stated: “We are excited to develop a leading-edge project in the rapidly evolving field of radioligand therapies in rare cancers. “Early results for 212 Pb have demonstrated its differentiated biophysical and clinical profile, reinforcing its potential to be a transformative radioligand therapeutic for patients across multiple difficult-to-treat rare cancers. This agreement underscores our efforts to explore innovative collaborations that leverage novel technologies to address the needs of people living with cancer.” The recent development comes after Sanofi announced the inauguration of its new modular vaccine and biomedicine production unit , Modulus, in Neuville-sur-Saône, France.

Phase 2License out/inClinical ResultVaccineBreakthrough Therapy

12 Sep 2024

·www.pharmaceutical-technology.com

India’s CDSCO suspends Entod’s licence for PresVu eye drops

The CDSCO has emphasised that the eye drops should not be sold without a valid prescription. Credit: Ahmet Misirligul/Shutterstock. Due to safety concerns and unauthorised claims, the Central Drugs Standard Control Organisation (CDSCO) of India has suspended the manufacturing and marketing license of Entod Pharmaceuticals’ PresVu eye drops, which contain 1.25% Pilocarpine w/v. The CDSCO’s suspension order noted that PresVu was not approved to make claims about reducing the need for reading glasses. The product was only permitted for treating presbyopia in adults, and not for any other benefits such as enhancing near vision. According to the CDSCO, Entod engaged in promotional activities that included “unauthorised claims” in the media and on social platforms. These activities showcased that PresVu could be an advanced alternative for near vision improvement – a claim not sanctioned by the regulator. See Also: FDA grants breakthrough therapy designation to Neuraptive’s NTX-001 FDA grants breakthrough therapy status to Arrowhead’s plozasiran The promotion of PresVu also led to concerns about its use as an over-the-counter drug, despite its approval only as a prescription medication. The CDSCO emphasised that eye drops should not be sold without a valid prescription, either in physical stores or online. The CDSCO had previously requested explanations from Entod Pharmaceuticals concerning these claims. The suspension order was a consequence of the company’s inadequate response to the regulator’s concerns. Entod Pharmaceuticals CEO Nikkhil Masurkar stated: “We will challenge this suspension in the court of law to get justice.” In a media release, Masurkar went on: “Our approval by DCGI was based on a valid controlled clinical trial in 234 patients which was successful in showing [the] efficacy and safety of these eye drops in patients [with] presbyopia, who used these drops without eyeglasses and could read additional lines on Snellen’s chart which is a yardstick of near vision improvement.” “Such eye drops on the same active ingredient and same concentration have been approved by the US FDA and marketed in the USA for the last three years without any serious complications. The FDA did not take any action on the companies marketing the same in the USA.”

Priority ReviewBreakthrough Therapy

100 Deals associated with NTX-001

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Arm Injuries	Phase 3	-	Neuraptive Therapeutics, Inc.Startup	01 Jan 2025
Peripheral Nerve Injuries	Phase 3	-	Neuraptive Therapeutics, Inc.Startup	01 Jan 2025
Facial Paralysis	Phase 2	US	Neuraptive Therapeutics, Inc.Startup	12 Jul 2022

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04572906 (NEUROFUSE, Biospace) Manual	Phase 2	Peripheral Nerve Injuries	-	NTX-001	(hpsjowdkrw) = clinically and statistically meaningful changes observed at eight and 24-week intervals. lsxfyhyynq (ixtonkvqjd ) View more	Positive	22 Feb 2024
				Standard of Care
NCT04572906 (NEUROFUSE, BusinessWire) Manual	Phase 2	Peripheral Nerve Injuries	60	NTX-001	(zcgcfkwhtg) = the MHQ change at baseline in Total Score at 12 weeks demonstrated that NTX-001 treatment was numerically improved over SOC but did not reach statistical significance for this global function assessment. arsijmpdyt (cgbeectjua ) View more	Positive	08 Jan 2024
				SOC

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