A Phase 2b/3, Double-blind, Randomized, Placebo Controlled, Multicenter Study to Assess the Efficacy and Safety of VX-210 in Subjects With Acute Traumatic Cervical Spinal Cord Injury

The purpose of this study is to determine the efficacy and safety of VX-210 in subjects with Acute Traumatic Cervical Spinal Cord Injury. Secondary objectives include the specific evaluation of the effects of VX-210 on neurological recovery and daily function after spinal cord injury.

Start Date01 Jul 2016

Sponsor / Collaborator

Vertex Pharmaceuticals, Inc.

NCT02053883 / WithdrawnPhase 2/3

A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Efficacy and Safety of Cethrin in Subjects With Acute Cervical Spinal Cord Injury

This is a multicenter, randomized, double-blind, placebo-controlled Phase IIb/III study designed to evaluate the efficacy and safety of Cethrin as a treatment for acute cervical spinal cord injury. During the trial, high and low doses of Cethrin will be compared with placebo.

Start Date01 Jul 2015

Sponsor / Collaborator

BioAxone BioSciences, Inc.

NCT00500812 / CompletedPhase 1/2

A Phase I/IIa Dose-Ranging Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BA-210 and the Neurological Status of Patients Following Administration of a Single Extradural Application of Cethrin During Surgery for Acute Thoracic and Cervical Spinal Cord Injury

This trial is a multi-center, open-label, dose-escalation study designed to evaluate the safety, tolerability and pharmacokinetics of Cethrin in two types of spinal cord injury patients: those with a complete cervical injury or a complete thoracic injury. Dose levels from 0.3 mg - 9 mg of Cethrin will be administered.

Start Date01 Feb 2005

Sponsor / Collaborator

Vertex Pharmaceuticals, Inc.

100 Clinical Results associated with Praconase

100 Translational Medicine associated with Praconase

100 Patents (Medical) associated with Praconase

Literatures (Medical) associated with Praconase

01 Aug 2021·Journal of NeurotraumaQ2 · MEDICINE

A Randomized Controlled Trial of Local Delivery of a Rho Inhibitor (VX-210) in Patients with Acute Traumatic Cervical Spinal Cord Injury

Q2 · MEDICINE

Article

Author: Rizzo, Marco ; Kwon, Brian K. ; Shih, Ludy C. ; Dumont, Travis ; Lingam, Hari K. ; McKerracher, Lisa ; Harrop, James S. ; Aarabi, Bizhan ; Fourney, Daryl R. ; Chen, Yang ; Fehlings, Michael G. ; Vaccaro, Alexander ; Ahmad, Faiz ; Tsai, Eve C. ; Kim, Kee D. ; Anderson, Kim D.

Acute traumatic spinal cord injury (SCI) can result in severe, lifelong neurological deficits. After SCI, Rho activation contributes to collapse of axonal growth cones, failure of axonal regeneration, and neuronal loss. This randomized, double-blind, placebo-controlled phase 2b/3 study evaluated the efficacy and safety of Rho inhibitor VX-210 (9 mg) in patients after acute traumatic cervical SCI. The study enrolled patients 14-75 years of age with acute traumatic cervical SCIs, C4-C7 (motor level) on each side, and American Spinal Injury Association Impairment Scale (AIS) Grade A or B who had spinal decompression/stabilization surgery commencing within 72 h after injury. Patients were randomized 1:1 with stratification by age (<30 vs. ≥30 years) and AIS grade (A vs. B with sacral pinprick preservation vs. B without sacral pinprick preservation). A single dose of VX-210 or placebo in fibrin sealant was administered topically onto the dura over the site of injury during decompression/stabilization surgery. Patients were evaluated for medical, neurological, and functional changes, and serum was collected for pharmacokinetics and immunological analyses. Patients were followed up for up to 12 months after treatment. A planned interim efficacy-based futility analysis was conducted after ∼33% of patients were enrolled. The pre-defined futility stopping rule was met, and the study was therefore ended prematurely. In the final analysis, the primary efficacy end-point was not met, with no statistically significant difference in change from baseline in upper-extremity motor score at 6 months after treatment between the VX-210 (9-mg) and placebo groups. This work opens the door to further improvements in the design and conduct of clinical trials in acute SCI.

01 Jan 2021·Journal of Chemical NeuroanatomyQ4 · MEDICINE

Exploring the potential of RhoA inhibitors to improve exercise-recoverable spinal cord injury: A systematic review and meta-analysis

Q4 · MEDICINE

Review

Author: Li, Yu Qing ; Liu, RenShuai ; Luo, Min ; Yin, Mei ; Wu, Yue ; Zheng, Yu Rong ; Lu, Ya Feng

BACKGROUNDThe spinal cord is one of the central nervous system. Spinal cord injury (SCI) will cause loss of physical function and dysfunction below the injury site, causing them to lose sensation and mobility, thereby reducing the quality of life of patients. Although regular rehabilitation management can reduce its severity, the current effective treatment methods are limited to the treatment of secondary injuries to SCI. The purpose of treatment should not only include the restoration of the histology of the lesion, but also should focus on the restoration of sensory and mobility and. The key to effective treatment is to reduce secondary injuries. RhoA inhibitor can improve the pathophysiological changes related to secondary injury and promote the recovery of activity ability, so it may become a clinical drug for the treatment of SCI. This article systematically analyzed the effects of RhoA inhibitors on the promotion of axon regeneration and the recovery of mobility and compared the therapeutic effects of different inhibitors on SCI and their effects on physical function recovery.METHODSWe used a meta-analysis to systematically evaluate the effects of Rho inhibitors on SCI treatment and the recovery of body function.RESULTS21 articles (738 animals) were identified in the literatures search. Studies were selected if they reported the therapeutic effects of RhoA/ROCK inhibitors (BA-210, EGCG, β-elemene, C3-exoenzmye, LINGO-1-Fc, Ibuprofen, SiRhoA, iRhoA + FK506, Fasudil, p21Cip1/WAF1, HA-1007, Y-27,632 and C3bot154-182). We measure the functional recovery by BBB and BMS scores. The random effect model of weighted mean difference (WMD, 95 % confidence interval) was used to analyze the effects. The WMD of the forest graph was 2.277; 95 % CI: 1.705∼2.849, P < 0.001, suggesting that RhoA inhibitors can effectively treat SCI. In addition to EGCG, all the other agents also showed the effects on the activity recovery post-SCI (P < 0.05).CONCLUSIONβ-elemene, LINGO-1-Fc, Ibuprofen, SiRhoA, RhoA + FK506, Fasudil, p21Cip1/WAF1 and Y-27,632 have similar effects to BA-210, they can promote axon germination and nerve fiber regeneration after thoracic spinal cord injury and reduce the formation of syringomyelia and protect white matter, thereby improving locomotor recovery. RhoA inhibitors have great potential to restore motor function and provide a new trend for the treatment of SCI.

01 Jan 2019·Journal of Neurosurgery: SpineQ2 · MEDICINE

JNSPG 75th Anniversary Invited Review Article

Q2 · MEDICINE

Review

Author: Fehlings, Michael G. ; Badhiwala, Jetan H. ; Ahuja, Christopher S.

Acute traumatic spinal cord injury (SCI) is a devastating event with far-reaching physical, emotional, and economic consequences for patients, families, and society at large. Timely delivery of specialized care has reduced mortality; however, long-term neurological recovery continues to be limited. In recent years, a number of exciting neuroprotective and regenerative strategies have emerged and have come under active investigation in clinical trials, and several more are coming down the translational pipeline. Among ongoing trials are RISCIS (riluzole), INSPIRE (Neuro-Spinal Scaffold), MASC (minocycline), and SPRING (VX-210). Microstructural MRI techniques have improved our ability to image the injured spinal cord at high resolution. This innovation, combined with serum and cerebrospinal fluid (CSF) analysis, holds the promise of providing a quantitative biomarker readout of spinal cord neural tissue injury, which may improve prognostication and facilitate stratification of patients for enrollment into clinical trials. Given evidence of the effectiveness of early surgical decompression and growing recognition of the concept that “time is spine,” infrastructural changes at a systems level are being implemented in many regions around the world to provide a streamlined process for transfer of patients with acute SCI to a specialized unit. With the continued aging of the population, central cord syndrome is soon expected to become the most common form of acute traumatic SCI; characterization of the pathophysiology, natural history, and optimal treatment of these injuries is hence a key public health priority. Collaborative international efforts have led to the development of clinical practice guidelines for traumatic SCI based on robust evaluation of current evidence. The current article provides an in-depth review of progress in SCI, covering the above areas.

News (Medical) associated with Praconase

22 Jun 2022

·www.biospace.com

BioAxone BioSciences Announces Patent Issued for Pharmaceutical Compositions of BA-210, a Rho Inhibitor for the Treatment of Spinal Cord Injury

Seeking Global Licensing Partner for Phase 3 Clinical Development and Commercialization; FDA has granted Orphan Drug Status and Fast Track designation BOSTON--(BUSINESS WIRE)-- BioAxone BioSciences, Inc., a clinical stage biotechnology company committed to discovering and developing drugs for neurological conditions, today announced that a new patent has been issued for BA-210, a Phase 3-ready pro-regenerative drug candidate for the treatment of spinal cord injury. The new U.S. patent 11,324,802 covers pharmaceutical compositions of BA-210. “There is an urgent unmet need for new medicines for patients with acute spinal cord injuries,” said Lisa McKerracher, PhD, CEO, BioAxone BioSciences. “When central nervous system trauma occurs, neuronal Rho is ‘hyperactivated’ and that halts axon regeneration.” Dr. McKerracher continued, “Rho is the signaling molecule that acts as the ‘mastermind switch’ that regulates the growth and regeneration of injured axons. By inactivating Rho, BA-210 is designed to stimulate the regeneration of cut axons.” Rho as a target to treat spinal cord injury has been validated by multiple laboratories. After a spinal cord injury, the enzyme Rho regulates events that culminate in collapse of the neuronal growth cone, failure of axonal regeneration, and, ultimately, failure of motor and functional recovery. BA-210 is a first-in-class, topically applied, biologic Rho inhibitor. BioAxone BioSciences developed BA-210 to inactivate Rho and has brought the compound forward to clinical testing. BA-210 has both orphan drug status and Fast Track designation from the FDA. BioAxone Biosciences seeks a global licensing partner for clinical development and commercialization of the drug candidate which has a demonstrated safety pro reported clinical data from completed Phase 1b and Phase 2b clinical trials. The newly issued patent extends the commercial opportunity for BA-210. BioAxone has previously partnered a proprietary portfolio of ROCK2 selective Rho kinase inhibitors. The lead compound is an oral kinase inhibitor for treatment of cerebral cavernous malformation (CCM) and other neurovascular disorders. The NIH Small Business Innovation Research program supported proof of concept studies. About BioAxone BioSciences, Inc. Based in Boston, BioAxone BioSciences is a clinical stage, neuroscience biotechnology company which applies its deep understanding of axon regeneration and neuronal signaling pathways to discover and develop new medicines to transform the lives of patients afflicted with neurotrauma or neurovascular disorders. Leveraging 20 years of pioneering research in axon regeneration and diseases involving Rho/ROCK signaling, BioAxone’s lead drug candidate is BA-210, for the treatment of acute spinal cord injury. The company is seeking a global licensing partner for BA-210 to take on clinical development and commercialization. A second drug development program for treatment of spinal cord injury is focused on the intrinsic barriers to regeneration brought on by neuronal aging. This compound is in preclinical testing in collaboration with the Tanenbaum Open Science Institute of McGill University. For more information, visit .

CollaborateOrphan DrugFirst in ClassFast Track

08 Jun 2021

·www.biospace.com

BioAxone BioSciences Announces Notice of Allowance for Self-Delivering RNA Interference for the Treatment of Spinal Cord Injury

Issued claims relate to transient PTEN suppression for nerve regeneration BOSTON--(BUSINESS WIRE)-- BioAxone BioSciences, Inc., a biotechnology company applying an innovative understanding of axon regeneration and neuronal signaling to transform the lives of patients afflicted with neurotrauma or neurovascular disorders, today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO). The notice covers the critical patent application (US 16/321,229) for the proprietary method of treatment of spinal cord injury with BA-434, a novel sd-rxRNA compound that targets transient, non-permanent PTEN suppression to promote axon regeneration and reduce paralysis. Scientists have provided evidence that suppression of the protein PTEN is one of the most robust targets to promote regeneration of damaged axons. PTEN is also important in surveillance and defense against unwanted cell growth, including protection from cancer. However, until now the development of inhibitors to promote axon regeneration have focused on methods that permanently silence PTEN. “BA-434 is a therapeutically relevant drug candidate that overcomes the challenge of other PTEN inhibitors which deliver permanent suppression,“ said Lisa McKerracher, PhD, CEO, BioAxone BioSciences. “Our drug candidate delivers transient PTEN suppression that is robust in the model systems used in preclinical testing. We look forward to continued exploration of the opportunity to allow for axon regeneration to help serve this unmet need of patients with spinal cord injury.” BA-434 is BioAxone’s novel self-delivering RNA interference compound that reduces expression of PTEN, a protein that blocks axon regeneration in adult neurons. Research for development of BA-434 was supported by NIH Small Business Innovation Research grants. About BioAxone BioSciences, Inc. Based in Boston, BioAxone BioSciences is applying its deep understanding of axon regeneration and neuronal signaling pathways to transform the lives of patients afflicted with neurotrauma or neurovascular disorders. Leveraging 20 years of pioneering research in axon regeneration and diseases involving Rho/ROCK signaling, BioAxone has a clinical stage drug (BA-210) and a preclinical program (BA-434) targeting axon regeneration for treatment of spinal cord injury. BA-1049, intended for license, is the lead orally-available ROCK2 inhibitor to restore endothelial cell barrier function in cavernous angioma (CA) and cerebral aneurysms. Cavernous angioma, which has both spontaneous and genetic forms, and cerebral aneurysms, are considered unmet medical needs with no pharmacological treatment options currently available for patients. For more information, visit .

100 Deals associated with Praconase

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
acute spinal cord injury	Phase 3	US	Vertex Pharmaceuticals, Inc.	01 Jul 2016
acute spinal cord injury	Phase 3	CA	Vertex Pharmaceuticals, Inc.	01 Jul 2016
Cervical spinal cord injury	Phase 3	-	BioAxone BioSciences, Inc.	01 Jul 2015
Dystrophy, Macular	Phase 3	-	BioAxone Therapeutic, Inc.	-
Dystrophy, Macular	Phase 3	-	Vertex Pharmaceuticals, Inc.	-
Glaucoma	Phase 3	-	BioAxone Therapeutic, Inc.	-
Glaucoma	Phase 3	-	Vertex Pharmaceuticals, Inc.	-
Optic Neuritis	Phase 3	-	Vertex Pharmaceuticals, Inc.	-
Optic Neuritis	Phase 3	-	BioAxone Therapeutic, Inc.	-
Spinal Cord Injuries	Phase 3	-	BioAxone BioSciences, Inc.	-

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02669849 (CTgov)	Phase 2/3	Cervical spinal cord injury \| acute spinal cord injury	70	Placebo	fgwlymiumw(vdaqgrchfy) = roglddlefq qslxyylndb (nickunpoki, hhwquhmfej - trawgjmxff) View more	-	22 Jan 2020
NCT00500812 (Pubmed \| J Neurotrauma)	Phase 1	acute spinal cord injury	48	Cethrin-treated patients	ybyppsytoe(fdwnosdpyf) = there was a trend to improved sensory scores in treated patients with thoracic injury zsecokrksi (pmojsrwhmx )	-	01 Nov 2013
NCT00500812 (Pubmed \| J Neurotrauma)	Phase 1	acute spinal cord injury	48	BA-210 (Cethrin)	wntscsellq(cupkjvixza) = xkzaiwhfug ozydvrzomk (fbbgzxuefj )	-	01 May 2011

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