PGIC

Disc Reports Topline Results from Phase 2 AURORA Study of Bitopertin in Patients with Erythropoietic Protoporphyria (EPP)

Retrieved on: 
Monday, April 1, 2024
PGIC, EPP, Patient, Light, Ruxolitinib, IRON, Sunlight, Bitopertin, Anemia, Pain, Medical imaging

On the key secondary endpoint of cumulative time in sunlight on days without pain, bitopertin patients had a positive response consistent with BEACON results, but the endpoint did not meet statistical significance due to strong placebo performance.

Key Points: 
  • On the key secondary endpoint of cumulative time in sunlight on days without pain, bitopertin patients had a positive response consistent with BEACON results, but the endpoint did not meet statistical significance due to strong placebo performance.
  • Subjects were randomized 1:1:1 to receive 20 mg of bitopertin, 60 mg of bitopertin, or placebo once daily for 17 weeks.
  • Two patients discontinued treatment due to treatment-emergent AEs, both in the 60 mg dose group: one due to dizziness and one due to a skin rash.
  • Please register for management’s webcast on the Events and Presentations page of Disc’s website ( https://ir.discmedicine.com/ ).

Neuralace Medical Announces FDA Clearance of Axon Therapy for Chronic Painful Diabetic Neuropathy

Retrieved on: 
Thursday, January 11, 2024
PGIC, Patient, Research, MD, Health, SAN, RCT, Pain management, Pain, CX, Quality of life, Hypoesthesia, PDN, FDA, Silviculture

SAN DIEGO, Jan. 11, 2024 /PRNewswire/ -- Neuralace Medical, Inc., an innovator in pain management technology, today proudly announces the FDA clearance of its groundbreaking product, Axon Therapy (mPNS), for the treatment of chronic Painful Diabetic Neuropathy (PDN). This landmark approval marks the first-ever FDA clearance of a non-invasive, magnetic peripheral nerve stimulation (mPNS) treatment for Painful Diabetic Neuropathy, offering new hope to millions suffering from this debilitating condition.

Key Points: 
  • SAN DIEGO, Jan. 11, 2024 /PRNewswire/ -- Neuralace Medical, Inc., an innovator in pain management technology, today proudly announces the FDA clearance of its groundbreaking product, Axon Therapy (mPNS), for the treatment of chronic Painful Diabetic Neuropathy (PDN).
  • This landmark approval marks the first-ever FDA clearance of a non-invasive, magnetic peripheral nerve stimulation (mPNS) treatment for Painful Diabetic Neuropathy, offering new hope to millions suffering from this debilitating condition.
  • Axon Therapy utilizes a pioneering approach of magnetic peripheral nerve stimulation (mPNS) to deliver a quick, painless, and non-invasive treatment.
  • "The FDA clearance of Axon Therapy is a monumental step forward in the treatment of PDN," said Keith Warner, CEO of Neuralace Medical.

Neuraptive Therapeutics, Inc. Announces Achievement of Proof of Concept in The NEUROFUSE Study of NTX-001 for the Adjunct Treatment of Transected Peripheral Nerves

Retrieved on: 
Monday, January 8, 2024
FDA, Health, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, Quality of life, Science, Axon, Multimedia, Physician, Regeneration, Pain, Hope, Participation, Nerve, MHQ, Therapy, SAE, Randomness, Wallerian degeneration, Control, Standard of care, Nerve injury, Clinical trial, PGIC, Patient, Safety, SOC, EVP

Neuraptive Therapeutics, Inc. is dedicated to revolutionizing patient care through innovative therapies, and NTX-001 has the potential to redefine the standard of care.

Key Points: 
  • Neuraptive Therapeutics, Inc. is dedicated to revolutionizing patient care through innovative therapies, and NTX-001 has the potential to redefine the standard of care.
  • Key highlights from the NEUROFUSE Study:
    The NEUROFUSE Study is a 48-week, Multicenter, Randomized, Controlled, Evaluator-Blinded Study comparing NTX-001 as an adjunct treatment to standard of care vs. standard of care alone.
  • The study was to enroll 60 patients, and patients were randomized in a 1:1 ratio between treatments.
  • For more information about Neuraptive Therapeutics, Inc., and our pioneering work in peripheral nerve injury treatments, please visit Neuraptive.com.

argenx Highlights 2024 Strategic Priorities

Retrieved on: 
Monday, January 8, 2024
Immunoglobulin therapy, GMG, FDA, PRV, Congenital myasthenic syndrome, ARDA, Research Councils UK, Euronext, Inflammation, Cohort, PGIC, NRDL, Autoimmunity, Efgartigimod alfa, Dermatomyositis, Conference, MuSK protein, IND, CIDP, Pharmacokinetics, MMN, Safety, Therapy, Patient, ALS, Immune system, IL-6, Medicine, International Myeloma Foundation, IIP, FSS, Neonatal Fc receptor, Multifocal motor neuropathy, Growth, Pharmaceutical industry

argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.

Key Points: 
  • argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.
  • In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.
  • As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*.
  • Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion.

Cogent Biosciences Announces Positive Initial Data from Phase 2 SUMMIT Trial Evaluating Bezuclastinib in Patients with Nonadvanced Systemic Mastocytosis (NonAdvSM)

Retrieved on: 
Saturday, December 9, 2023
Patient, Cogent, OLE, MAS, Deficit spending, ISM, GIST, Fourth grade, Poster, Bleeding, American Society of Hematology, APEX, Disease, KIT, MD Anderson Cancer Center, Society, Stromal cell, SSM, PD, ASH, Mutation, VAF, Hyperkinesia, Part, PGIC, Webcast, Safety, University, PEAK, Dermatology, Pharmaceutical industry

Twenty patients in Part 1a were treated with either bezuclastinib or placebo plus best supportive care for all arms.

Key Points: 
  • Twenty patients in Part 1a were treated with either bezuclastinib or placebo plus best supportive care for all arms.
  • Patients were enrolled with the following sub-types: 18 patients with indolent systemic mastocytosis (ISM) and two patients with smoldering systemic mastocytosis (SSM).
  • In patients with completed questionnaires:
    By week 12, bezuclastinib patients showed a median best improvement of 37% on MC-QoL vs. 24% for placebo patients.
  • Data from Part 1 of the Phase 2 APEX clinical trial evaluating bezuclastinib in patients with advanced systemic mastocytosis (AdvSM) will be presented in a poster session at ASH on Monday, December 11, 2023 at ASH.

Aurora Spine Corporation Announces Newly Published Paper on First 6-month Clinical Evaluation of its ZIP™ Fusion Implant

Retrieved on: 
Monday, December 4, 2023
ASG, Walking, Principal investigator, Neurosurgery, Physician, ODI, Buttocks, FDA, Pain, Decompression, Chronic pain, Spinal cord, Visual analogue scale, IRB, Low back pain, Trial of the century, Groin, PROMIS, PGIC, Institutional review board, Back pain, ZIP, Human, Patient, Safety, Journal, Leg, Spinal stenosis, Degenerative disc disease, Oswestry Disability Index, Birth control

This 6-month interim analysis at 42% enrollment of patients was conducted to determine prolonged safety and efficacy of the ZIP interspinous fusion device.

Key Points: 
  • This 6-month interim analysis at 42% enrollment of patients was conducted to determine prolonged safety and efficacy of the ZIP interspinous fusion device.
  • Dr. Sebastian Koga, a neurosurgeon from Covington, LA emphasized, “Our study demonstrates that the ZIP device provides a stable platform for posterior fusion.
  • I am happy to contribute objective clinical evidence to this area of spine surgery.
  • A previously published retrospective study has also demonstrated the ZIP device’s safety and effectiveness in the hands of interventional pain physicians.

Swing Therapeutics Announces Positive Results from Pivotal Phase 3 Trial of Stanza, a Digital Therapy for Fibromyalgia Symptoms

Retrieved on: 
Tuesday, November 14, 2023
Biotechnology, Technology, FDA, Health, General Health, Pharmaceutical, Health Technology, Medical Devices, Apps, Applications, Software, Clinical Trials, ACT, RCT, Fatigue, Fibromyalgia, Depression, De novo, Pivotal, Randomized controlled trial, Food, Commitment, Therapy, University of Michigan, CBT, Cognitive behavioral therapy, Patient, PROMIS, American College of Rheumatology, PGIC, American College, BDI, MD, Sleep, Phase 3, University, University of Cincinnati, FIQ, Doctor of Philosophy, Guideline, ACR, Chronic pain, PIPS, Pharmaceutical industry, Medical imaging, Medical device, Stanza, Swing

Swing Therapeutics, a leading developer of digital therapies for chronic pain conditions, today announced the successful completion of and positive results from its pivotal Phase 3 PROSPER-FM trial on Stanza, a self-guided smartphone-based behavioral therapy for the treatment of fibromyalgia symptoms in adult patients.

Key Points: 
  • Swing Therapeutics, a leading developer of digital therapies for chronic pain conditions, today announced the successful completion of and positive results from its pivotal Phase 3 PROSPER-FM trial on Stanza, a self-guided smartphone-based behavioral therapy for the treatment of fibromyalgia symptoms in adult patients.
  • Results from this multi-center, randomized controlled trial (RCT) were presented at the American College of Rheumatology (ACR) 2023 annual meeting this week.
  • Swing received De Novo marketing authorization from the U.S. Food and Drug Administration (FDA) for Stanza in May 2023, making it the first prescription digital therapeutic indicated for treating fibromyalgia symptoms.
  • It delivers acceptance and commitment therapy (ACT), a type of cognitive behavioral therapy (CBT) with proven efficacy in treating fibromyalgia and other chronic pain conditions.

Cogent Biosciences Reports Recent Business Highlights and Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 2, 2023
Patient, Research, MAS, IND, American Society of Clinical Oncology, APEX, FGFR2, PEAK, G&A, Novel, Safety, Conference, D, PGIC, Society, WT, Cogent, Pharmacokinetics, ASH, Clinical trial, Efficacy, Sunitinib, ORR, GIST, Part, Drug discovery, ASCO, American Society of Hematology, ENA, Acceleration, Pharmaceutical industry, Nursing, Video game, Q3

WALTHAM, Mass. and BOULDER, Colo., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today reported recent business highlights and financial results for the third quarter ended September 30, 2023.

Key Points: 
  • and BOULDER, Colo., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today reported recent business highlights and financial results for the third quarter ended September 30, 2023.
  • Cogent will host an investor webcast to review the results on Monday, December 11, 2023 at 8:00 a.m.
  • G&A Expenses: General and administrative expenses were $9.5 million for the third quarter of 2023 as compared to $6.9 million for the third quarter of 2022.
  • G&A expenses include non-cash stock compensation expense of $4.8 million for the third quarter of 2023 compared to $2.6 million for the third quarter of 2022.

Teva to Present New Data Supporting Safety, Efficacy and Real-World Effectiveness of AUSTEDO® (deutetrabenazine) Tablets at the 2023 HSG Annual Meeting

Retrieved on: 
Thursday, November 2, 2023
Health, Neurology, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, FDA, VMAT2, NNH, NYSE, Georgetown University School of Medicine, Medication, HD, MD, NNT, Psychiatry, START, Doctor of Philosophy, PGIC, TEVA, Yahoo Beijing Global R&D Center, Teva, Tetrabenazine, Interim, Annual general meeting, Teva Pharmaceuticals, TASE, TMC, Education, Safety, HSG, Patient, Pharmaceutical industry

Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), today announced new HD data will be presented at the Huntington Study Group® (HSG) Annual Meeting on November 2-4 in Phoenix, AZ.

Key Points: 
  • Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), today announced new HD data will be presented at the Huntington Study Group® (HSG) Annual Meeting on November 2-4 in Phoenix, AZ.
  • TMC scores mostly improved with AUSTEDO treatment, and the safety profile was consistent with AUSTEDO’s known safety profile.
  • NNTs for treatment success based on CGIC and PGIC ranged from 3-4 and 4-5, respectively, for the VMAT2 inhibitors.
  • Significant NNHs included:
    Together, the data provide additional insight into the real-world efficacy and safety of AUSTEDO for patients with HD chorea.

Fulcrum Announces Completion of Enrollment in the Phase 3 REACH Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD)

Retrieved on: 
Thursday, September 7, 2023
NCT, Losmapimod, MFI, FSHD, ClinicalTrials.gov, Quality, PGIC, RWS, Neuro, Patient, Quality of life, FDA, Therapy, Safety, Pharmaceutical industry, Ascending limb of loop of Henle, REACH

“We are very pleased to have enrolled 260 patients in REACH, our global Phase 3 pivotal clinical trial for patients with FSHD,” said Alex C. Sapir, Fulcrum’s president and chief executive officer.

Key Points: 
  • “We are very pleased to have enrolled 260 patients in REACH, our global Phase 3 pivotal clinical trial for patients with FSHD,” said Alex C. Sapir, Fulcrum’s president and chief executive officer.
  • The trial enrolled 260 patients who were randomized 1:1 to receive either losmapimod, administered orally as a 15 mg tablet twice a day, or placebo, and will be evaluated over a 48-week treatment period.
  • The primary endpoint of the study is the absolute change from baseline in Reachable Workspace (RWS).
  • Secondary endpoints include muscle fat infiltration (MFI), Patient Global Impression of Change (PGIC), and Quality of Life in Neurological Disorders of the Upper Extremity (Neuro QoL UE).