PROMIS

ProMIS Neurosciences Publishes in the Journal of Biological Chemistry on the Interaction Between Pathogenic Proteins as a Treatment Target for ALS

Retrieved on: 
Tuesday, April 9, 2024

ProMIS is developing antibodies selectively targeting misfolded forms of TDP-43 and SOD1.

Key Points: 
  • ProMIS is developing antibodies selectively targeting misfolded forms of TDP-43 and SOD1.
  • ALS is a fatal neurodegenerative disease of motor neurons.
  • “Publication of these data underscores the connection of misfolded proteins and ALS and supports targeting our TDP-43-specific epitope with PMN267 as a potential therapeutic approach,” stated Neil Warma, Chief Executive Officer of ProMIS Neurosciences.
  • “PMN267 is advancing through preclinical development and is showing promise as a potential treatment for ALS.

Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency

Retrieved on: 
Monday, April 8, 2024

BOSTON, April 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Key Points: 
  • “We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • The patients were assigned to three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4).
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

ProMIS Neurosciences Announces Full Year 2023 Financial Results and Recent Highlights

Retrieved on: 
Monday, April 1, 2024

This is a very exciting opportunity for both ProMIS and the AD patients we aim to serve.

Key Points: 
  • This is a very exciting opportunity for both ProMIS and the AD patients we aim to serve.
  • ProMIS dosed the first participants in a first-in-human Phase 1a clinical trial of PMN310 as a potential treatment for AD in November 2023 (Study NCT06105528 ).
  • Using a proprietary computational platform, ProMIS identified potential conformational epitopes (misfolded portions) unique to toxic alpha-synuclein involved in synucleinopathies.
  • Cash and cash equivalents were $12.6 million as of December 31, 2023, compared to $5.9 million as of December 31, 2022.

ProMIS Neurosciences Strengthens Global Intellectual Property Portfolio

Retrieved on: 
Monday, March 11, 2024

The United States Patent and Trademark Office also recently granted US Patent 11,905,317 directed to immunogenic compositions of matter that are being developed for treating AD.

Key Points: 
  • The United States Patent and Trademark Office also recently granted US Patent 11,905,317 directed to immunogenic compositions of matter that are being developed for treating AD.
  • Protection for the immunogenic compositions of matter has also been obtained in Japan, Korea, India and Australia.
  • Further, patent applications were also recently allowed in the U.S. and Japan for two other Aβ targets.
  • “We are building a fortress of intellectual property protection around our novel approach to targeting toxic misfolded proteins to treat neurodegenerative diseases and these newly allowed patents validate that innovation in important geographies around the world,” stated Neil Warma, Chief Executive Officer of ProMIS Neurosciences.

WebPT and Limber Health Announce New MSK Measure Set to Equally Compare Clinician Outcomes

Retrieved on: 
Thursday, February 1, 2024

PHOENIX, Feb. 1, 2024 /PRNewswire/ -- Two-industry leaders WebPT, the rehab therapy platform for Practice Experience Management (PXM), and Limber Health, a digital health solution enabling the delivery of hybrid musculoskeletal (MSK) care, together announce the launch of a single unified outcome measure set.

Key Points: 
  • PHOENIX, Feb. 1, 2024 /PRNewswire/ -- Two-industry leaders WebPT , the rehab therapy platform for Practice Experience Management (PXM), and Limber Health , a digital health solution enabling the delivery of hybrid musculoskeletal (MSK) care, together announce the launch of a single unified outcome measure set.
  • WebPT and Limber Health, accepted the request from CMS and completed this incredible effort to harmonize quality measure sets in collaboration with other industry leaders, including Intermountain Health , Upstream Rehabilitation , and ATI Physical Therapy .
  • The new MSK Measure Set is co-stewarded by WebPT's MSK and Rehabilitative Care Outcomes QCDR and Limber Health's Advancing MSK Care and Rehabilitation QCDR QCDR.
  • Additionally, this new MSK Measure set also includes the PROMIS measures previously supported via Limber Health's former measure set, the LMBR Quality Measures.

ProMIS Neurosciences Achieves Milestone in Development of Therapeutic Alpha-Synuclein Vaccine

Retrieved on: 
Monday, January 22, 2024

“We are excited about the potential of our ground-breaking technology in neurodegenerative diseases,” said Neil Warma, Chief Executive Officer of ProMIS Neurosciences.

Key Points: 
  • “We are excited about the potential of our ground-breaking technology in neurodegenerative diseases,” said Neil Warma, Chief Executive Officer of ProMIS Neurosciences.
  • Using a proprietary computational platform, ProMIS identified potential conformational epitopes (misfolded portions) unique to toxic alpha-synuclein involved in synucleinopathies.
  • Formulations of several of these epitopes were tested in mouse vaccination studies leading to the selection of a lead vaccine candidate for testing in mouse models replicating cognitive and motor deficits of human disease.
  • Results from the vaccination studies are being submitted for presentation at the 2024 meeting of the American Academy of Neurology (AAN) taking place from April 13-18, 2024 in Denver, Colorado.

ProMIS Neurosciences Issues Letter to Shareholders

Retrieved on: 
Monday, January 8, 2024

TORONTO, Ontario and CAMBRIDGE, Massachusetts, Jan. 08, 2024 (GLOBE NEWSWIRE) -- ProMIS Neurosciences Issues Letter to ShareholderProMIS Neurosciences Inc. (Nasdaq: PMN), a biotechnology company focused on the generation and development of antibody therapeutics targeting toxic misfolded proteins in neurodegenerative diseases such as Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA), today announced that its Chief Executive Officer, Neil Warma, issued the following letter to the Company’s shareholders.

Key Points: 
  • TORONTO, Ontario and CAMBRIDGE, Massachusetts, Jan. 08, 2024 (GLOBE NEWSWIRE) -- ProMIS Neurosciences Issues Letter to ShareholderProMIS Neurosciences Inc. (Nasdaq: PMN), a biotechnology company focused on the generation and development of antibody therapeutics targeting toxic misfolded proteins in neurodegenerative diseases such as Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA), today announced that its Chief Executive Officer, Neil Warma, issued the following letter to the Company’s shareholders.
  • Our ultimate goal is to get these potential new drugs to patients who are in desperate need of safe and effective treatment options.
  • This specificity is expected to improve efficacy outcomes and prevent any off-target safety issues, which are common with other AD therapies currently marketed or in development.
  • As we navigate this transformative period, I am confident that ProMIS will emerge stronger, more resilient, and better positioned to make significant contributions to healthcare.

ProMIS Neurosciences, Inc. Announces Leadership Transition

Retrieved on: 
Wednesday, January 3, 2024

Mr. Warma’s appointment is expected to provide a seamless transition as the Company remains focused on advancing the ongoing clinical program for PMN310 in Alzheimer’s disease and forging strategic partnerships to accelerate ProMIS’ broad potential for groundbreaking drug development.

Key Points: 
  • Mr. Warma’s appointment is expected to provide a seamless transition as the Company remains focused on advancing the ongoing clinical program for PMN310 in Alzheimer’s disease and forging strategic partnerships to accelerate ProMIS’ broad potential for groundbreaking drug development.
  • His leadership and his neuroscience background, combined with a profound understanding of our mission in novel drug development, positions the Company well for growth during this transition period," said Gene Williams, Chairman of the Board of ProMIS.
  • “We thank Dr. Farfel for her contributions and leadership during her tenure at ProMIS and wish her continued success in her endeavors,” added Mr. Williams.
  • Mr. Warma has held senior leadership positions at several biotech and pharma companies, including Novartis, Opexa Therapeutics and I-Mab BioPharma, Inc.

Tonix Pharmaceuticals Announces Highly Statistically Significant and Clinically Meaningful Topline Results in Second Positive Phase 3 Clinical Trial of TNX-102 SL for the Management of Fibromyalgia

Retrieved on: 
Wednesday, December 20, 2023

CHATHAM, N.J., Dec. 20, 2023 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that the Phase 3 RESILIENT study evaluating TNX-102 SL (cyclobenzaprine HCl sublingual tablets) met its pre-specified primary endpoint in the second of two positive Phase 3 clinical trials, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia (Table 1). Statistically significant and clinically meaningful results were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function. Additionally, as it relates to improving daily pain, treatment with TNX-102 SL showed a robust and clinically meaningful analgesic effect size of 0.38, with rapid onset of action, separating from placebo for each week of the study. TNX-102 SL was well tolerated with an adverse event profile comparable to prior studies, and no new safety signals were observed. Tonix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024 for TNX-102 SL for the management of fibromyalgia. An estimated 6 million to 12 million U.S. adults are living with fibromyalgia, the majority of whom are women.

Key Points: 
  • TNX-102 SL is a non-opioid, centrally-acting analgesic, the active ingredient of which has a known, favorable safety profile from decades of use.
  • “The sublingual formulation of TNX-102 SL, which uses our proprietary Protectic® and Angstro® technologies, is integral to our treatment paradigm.
  • Consistent with the proposed mechanism that TNX-102 SL acts in fibromyalgia through improving sleep quality, TNX-102 SL showed statistically significant improvement of sleep by two main measures.
  • Adverse events resulted in premature study discontinuation in 6.1% of those who received TNX-102 SL compared with 3.5% of placebo recipients.

ProMIS Neurosciences Announces Publication on Novel Target for ALS

Retrieved on: 
Wednesday, December 20, 2023

TORONTO, Ontario and CAMBRIDGE, Massachusetts, Dec. 20, 2023 (GLOBE NEWSWIRE) -- ProMIS Neurosciences Inc. (Nasdaq: PMN), a biotechnology company focused on the generation and development of antibody therapeutics targeting toxic misfolded proteins in neurodegenerative diseases such as Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA), today announced a publication identifying Receptor for Activated C-Kinase 1 (RACK1) as a novel misfolded protein target for ALS and frontotemporal lobar degeneration (FTLD). The article published in the online edition of Acta Neuropathologica Communications is titled, "Targeting RACK1 to alleviate TDP-43 and FUS proteinopathy-mediated suppression of protein translation and neurodegeneration."

Key Points: 
  • The article published in the online edition of Acta Neuropathologica Communications is titled, "Targeting RACK1 to alleviate TDP-43 and FUS proteinopathy-mediated suppression of protein translation and neurodegeneration."
  • Under normal conditions, TDP-43 and FUS are predominantly localized in the nucleus of cells, where they participate in the regulation of protein expression.
  • In disease, however, they typically become mislocalized in the cytoplasm of neurons where they form aggregates.
  • The study authors reported that pathological FUS and TDP-43 both co-aggregate with RACK1 resulting in suppression of protein synthesis.