Kabuki syndrome

ORYZON Reports Financial Results and Corporate Update for Quarter Ended December 31, 2023

Retrieved on: 
Monday, February 26, 2024
Borderline personality disorder, HOPE, HDAC6, National Cancer Institute, EVOLUTION, CMT, Agitation, Data analysis, Convertible bond, ICIS, ALS, SCLC, Azacitidine, Duration, ICI, IIS, Sciatic nerve, Deficit spending, Paclitaxel, DSM-IV codes, Development, Genomics, ALS Association, FMS, LSD1, AML, Clinical Global Impression, MSKCC, Conference, Aggression, Safety, Biomarker, CNS, Venetoclax, CRADA, OHSU, MRD, Clinical trial, BPD, Measure (mathematics), IND, FCCC, Dor, Human, Innovation, EMA, Kabuki syndrome, NET, NCI, Aes, Glomus tumor, FDA, GST, Food, Platinum, Schizophrenia, Phase II, Charcot–Marie–Tooth disease, Medicine, Patient, BEST, Disease, Pharmaceutical industry

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the fourth quarter.

Key Points: 
  • Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the fourth quarter.
  • Research and development (R&D) expenses were $3.9 and $16.6 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $5.0 and $18.1 million for the quarter and twelve months ended December 31, 2022.
  • General and administrative expenses were $1.2 and $4.2 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $1.2 and $4.8 million for the quarter and twelve months ended December 31, 2022.
  • Net losses were $1.4 and $5.0 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $1.6 and $5.9 million for the quarter and twelve months ended December 31, 2022.

ORYZON Reports Financial Results and Corporate Updates for Quarter Ended September 30, 2023

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Friday, October 27, 2023
Research, ORY, Fruit, DMC, Food, Deficit spending, Risk, Medicine, ECNP, Schizophrenia, Platinum, ICMJE recommendations, SCLC, Charcot–Marie–Tooth disease, Sciatic nerve, Psychotherapy, Paclitaxel, NET, FCCC, MRD, Safety, EVOLUTION, BPD, Kabuki syndrome, Patient, Borderline personality disorder, ALS, Fox Chase Cancer Center, Genomics, Human, Duration, Glomus tumor, Measure (mathematics), Clinical trial, Dor, DSM-IV codes, Animal, European College of Neuropsychopharmacology, LSD1, FMS, CMT, FDA, IND, Innovation, ISIN, CNS, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, R, HOPE, AML, Failure, Medical device, Pharmaceutical industry, Nursing, Cryptocurrency, EU, Science

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the third quarter.

Key Points: 
  • Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the third quarter.
  • The company has started a structured dialogue with corporate pharma to identify a suitable partner to move forward the asset to Phase III, pending positive results.
  • General and administrative expenses were $0.7 and $2.9 million for the quarter and nine months ended September 30, 2023, respectively, compared to $0.7 and $3.3 million for the quarter and nine months ended September 30, 2022.
  • Net losses were $0.8 and $3.4 million for the quarter and nine months ended September 30, 2023, respectively, compared to $0.7 and $3.9 million for the quarter and nine months ended September 30, 2022.

ORYZON Reports Financial Results and Corporate Update for Quarter Ended March 31, 2023

Retrieved on: 
Wednesday, May 10, 2023
Medicine, HOPE, Genomics, Glomus tumor, DSM-IV codes, Hospital, Patient, Borderline personality disorder, R, ALS, Charcot–Marie–Tooth disease, Columbia University, LSD1, Human, IDMC, Platinum, Innovation, Research, NET, CMT, Kabuki syndrome, Phase, FDA, IND, Conference, Safety, Duration, Schizophrenia, Seaver Autism Center, Measure (mathematics), Mental health, Deficit spending, ORY, FCCC, Dor, FMS, Hospital Universitario La Paz, BPD, Fox Chase Cancer Center, Clinical trial, Paclitaxel, CNS, Icahn School of Medicine at Mount Sinai, Hospital das Clínicas, AML, Mount Sinai Hospital, Spectrum, ISIN, ICMJE recommendations, Treatment, Pharmaceutical industry, Nursing, Video game, Science, SCLC

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon has made strong clinical progress in the first quarter this year.

Key Points: 
  • Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon has made strong clinical progress in the first quarter this year.
  • Research and development (R&D) expenses were $4.4 million for the quarter ended March 31, 2023, compared to $4.2million for the quarter ended March 31, 2022.
  • General and administrative expenses were $1.2 million for the first quarter ended March 31, 2023, compared to $1.3 million for the first quarter ended March 31, 2022.
  • Net losses were $1.4 million for the first quarter ended March 31, 2023, compared to $1.7 million for the first quarter ended March 31, 2022.

ORYZON Announces Positive Results from Planned Interim Analysis of PORTICO, a Phase 2b Adaptive Trial in Borderline Personality Disorder

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Monday, April 3, 2023
Psychiatry, LSD1, BPD, Borderline personality disorder, EudraCT, Schizophrenia, Genomics, IDMC, Hope, Aggression, CNS, DSM-IV codes, ORY, Patient, Central nervous system disease, ISIN, Agitation, FBXW7 neurodevelopmental syndrome, Safety, Pharmaceutical industry, Kabuki syndrome

These results were determined by an Independent Data Monitoring Committee (IDMC) that met on Thursday, March 30, 2023.

Key Points: 
  • These results were determined by an Independent Data Monitoring Committee (IDMC) that met on Thursday, March 30, 2023.
  • Based planned interim analysis of the first 90 participants who completed treatment, the trial was determined to be non-futile.
  • Based in the efficacy and safety data, the IDMC recommended to continue the trial without any modifications.
  • The trial is currently actively recruiting patients in Europe and in the US and aims to include about 188 patients distributed between two arms.

ORYZON to Give Updates on Corporate Progress in November

Retrieved on: 
Friday, November 4, 2022
EVOLUTION, Pipeline, Medulloblastoma, ISIN, ESCAPE, CNS, Clinical trial, Government budget balance, Schizophrenia, Degenerative disease, Hematological Cancer Research Investment and Education Act, Prospectus, Borderline personality disorder, Moderate, GLOBE, Mouse, IND, FLT3, NCI, NET, EHA, COVID-19, Mission statement, Forward-looking statement, CRADA, Patient, Cancer, ORY, Kabuki syndrome, Azacitidine, MS, Sale, Spanish, Multiple sclerosis, ESMO, Cooperative research and development agreement, LSD1, Aggression, Genomics, Market, Mercado, Time, Wilms' tumor, SCLC, Squamous cell carcinoma, Ageing, Safety, Conference, Neuroinflammation, CNMV, ALICE, AD, Development, Syndrome, Public expenditure, AML, Person, Communication, Amnesia, Pharmaceutical industry, EU

Oryzon will participate at the Inv€$tival Showcase LSX Leaders 2022 conference, which will be held on November 14 in London (United Kingdom). The company will take part in a panel entitled “CNS and the Market -- What is New in the Pipeline and in Deal Activity?”on November 14 at 15:00 local time. Click on link for more info about the Inv€$tival Showcase LSX Leaders conference

Key Points: 
  • Oryzons medical team will participate at the Kabuki Syndrome Foundation Annual Conference, which will be held virtually on November 4.
  • In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases.
  • Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon.
  • This press release is not an offer of securities for sale in the United States or any other jurisdiction.

ORYZON to Present Preliminary Blinded Aggregate Safety Data From Vafidemstat’s Ongoing Phase IIb PORTICO Trial in Borderline Personality Disorder

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Friday, September 16, 2022
Adult, Multiple sclerosis, Safety, CNMV, EVOLUTION, EudraCT, Ageing, AD, Data monitoring committee, Aggression, Mercado, Amnesia, Patient, Clinical trial, Suicide, Government budget balance, Communication, Time, ISIN, ORY, Sale, Mouse, Person, Public expenditure, Lists of diseases, Prospectus, Neuroinflammation, Particle-size distribution, Borderline personality disorder, COVID-19, Degenerative disease, Forward-looking statement, ESCAPE, Genomics, BPD, GLOBE, Spanish, Agitation, Conference, Mission statement, LSD1, Self-harm, Schizophrenia, Program, MS, Mental health, Moderate, Kabuki syndrome, CNS, BDP, Pharmaceutical industry, Phase

Blinded aggregate safety data presented at ECMH correspond to the initial randomized 43 patients (data cut-off, 30 June 2022).

Key Points: 
  • Blinded aggregate safety data presented at ECMH correspond to the initial randomized 43 patients (data cut-off, 30 June 2022).
  • In July 2022, the independent Data Monitoring Committee for PORTICO reviewed the available safety data and determined that the trial should continue.
  • Oryzons oral communication at ECMH is entitled PORTICO, a double-blind, randomized placebo-controlled, adaptive Phase IIb trial with vafidemstat in Borderline Personality Disorder.
  • Currently, vafidemstat is in two Phase IIb trials in borderline personality disorder (PORTICO) and in schizophrenia patients (EVOLUTION).

ORYZON to Give Updates on Corporate Progress in June

Retrieved on: 
Monday, June 6, 2022
Public expenditure, EST, Mission statement, Congress, Time, Lists of diseases, Government budget balance, Spanish, FLT3, Jefferies, Hematological Cancer Research Investment and Education Act, CNMV, Amnesia, IND, Sale, Ageing, Cancer, LSX, Borderline personality disorder, Forward-looking statement, Neuroinflammation, Mercado, Degenerative disease, Person, Moderate, Acute myeloid leukemia, Mouse, SCLC, CNS, ALICE, Communication, Genomics, Aggression, COVID-19, ESCAPE, ESMO, AML, European Hematology Association, Patient, AD, Schizophrenia, Medulloblastoma, Multiple sclerosis, Azacitidine, Wilms' tumor, LSD1, NET, ASH, EVOLUTION, ISIN, GLOBE, MS, Prospectus, ORY, Clinical trial, Squamous cell carcinoma, Kabuki syndrome, Safety, Pharmaceutical industry, Fine chemical

Oryzon will participate at the Jefferies Healthcare 2022 conference, which will be held on June 8-10 in New York (USA).

Key Points: 
  • Oryzon will participate at the Jefferies Healthcare 2022 conference, which will be held on June 8-10 in New York (USA).
  • The company will provide a corporate update on June 10 at 12:45 pm EST and will also hold one-to-one meetings with pharmaceutical companies and global investors.
  • In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases.
  • Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon.

ORYZON Starts Preclinical Collaboration on Kabuki Syndrome with Kennedy Krieger Institute and Johns Hopkins University

Retrieved on: 
Tuesday, May 3, 2022
Communication, Johns Hopkins School of Medicine, Neuroinflammation, Public expenditure, Spanish, Kabuki syndrome, Person, ESCAPE, Aggression, Mercado, Gene, Forward-looking statement, Mission statement, AD, Epigenetics, COVID-19, MLL2, Schizophrenia, Patient, Clinical trial, Prospectus, Lewis Milestone, CNMV, Moderate, Amnesia, Degenerative disease, EVOLUTION, Genomics, Genetics, LSD1, ORY, Mouse, Lists of diseases, Kennedy Krieger Institute, Time, Neurology, ISIN, Borderline personality disorder, Sale, Ageing, Pediatrics, Neuro, GLOBE, Government budget balance, CNS, Johns Hopkins University, MS, Intellectual disability, Chromatin, Multiple sclerosis, Pharmaceutical industry, KMT2D

Kabuki syndrome (KS) is an autosomal dominant/X-linked disorder that affects multiple organ systems including neuro, immune, auditory and cardiac systems.

Key Points: 
  • Kabuki syndrome (KS) is an autosomal dominant/X-linked disorder that affects multiple organ systems including neuro, immune, auditory and cardiac systems.
  • Founded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company considered as the European leader in epigenetics.
  • In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases.
  • Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon.

Crinetics Pharmaceuticals Reports Positive Top-Line Results from CRN04777 Phase 1 Study Multiple-Ascending Dose Cohorts

Retrieved on: 
Wednesday, March 30, 2022
Method, Plasma, SAD, Adult, Research, SEC, MAD, Ageing, Congenital hyperinsulinism, Turner syndrome, Carcinoid syndrome, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Nasdaq, Intellectual property, Insulin, GLOBE, Wilms' tumor, Disease, IV, Seizure, Trial of the century, Beckwith–Wiedemann syndrome, Sotos syndrome, Conference, ACTH, Gene, Glucose, Hypoglycemia, Congenital adrenal hyperplasia, Therapy, ID, Pharmacokinetics, Clinical trial, Webcast, Medication, HI, Drug discovery, SAN, Acromegaly, FDA, Apnea, Conference call, Patient, Volunteering, Degenerative disease, Half-life, Fasting, Incidence, Food, Coma, Brain damage, Prevalence, Birth defect, Kabuki syndrome, Safety, Hyperinsulinism, COVID-19, Șaeș, Pharmaceutical industry, Medical device

SAN DIEGO, March 30, 2022 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, today announced positive top-line results from the multiple-ascending dose (MAD) cohorts of healthy volunteers in a first-in-human Phase 1 clinical study of CRN04777. These results showed a rapid and sustained reduction of insulin secretion, including in a pharmacologic model of congenital hyperinsulinism (HI). CRN04777 is the company's investigational nonpeptide somatostatin receptor type 5 (SST5) agonist in development as an oral treatment for congenital monogenic and syndromic hyperinsulinism.

Key Points: 
  • Results showed CRN04777 treatment led to rapid, sustained and dose-dependent decreases in fasting insulin, which in-turn led to dose-dependent increases in fasting plasma glucose.
  • Pharmacokinetic and exposure profiles were consistent with expectations from the SAD cohorts, with CRN04777 being orally bioavailable with a half-life of approximately 40 hours.
  • In 2021, Crinetics initiated a Phase 1 clinical study in healthy volunteers to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of CRN04777.
  • Results from the Phase 1 study demonstrated pharmacologic proof-of-concept for CRN04777, with strong dose-dependent suppression of insulin secretion in healthy volunteers.

ORYZON to Present New Clinical Data and Corporate Updates at International Conferences in September and October

Retrieved on: 
Wednesday, September 22, 2021
ISIN, Person, Conference, Precision medicine, Structure, Spanish, PMS, Communication, Genomics, Kabuki syndrome, HOPE, EST, Safety, ORY, Public expenditure, Research, Mercado, BPD, BIO, Hospital Universitario La Paz, Development of the nervous system, Prospectus, Phenotype, Borderline personality disorder, CNS, Clinical trial, RRMS, Patient, ECNP, Pediatrics, Sale, CNMV, Neuroscience, Hospital das Clínicas, Functional genomics, SPMS, Forward-looking statement, LSD1, Kennedy Krieger Institute, GLOBE, Department, Treatment, KOL, European Committee for Sports History, Assistant, Congress, Central nervous system disease, Mission statement, Sclerosis (medicine), Psychiatry, Degeneration theory, International Conference on Nuclear Disarmament, Oslo, 2008, CET, Schizophrenia, Pharmaceutical industry, Medical device

Oryzon will present preliminary data from the ongoing collaboration with INGEMM in Phelan McDermid Syndrome (PMS) at the International Conference on Autism, ICA-2021, which will be held on September 23-24.

Key Points: 
  • Oryzon will present preliminary data from the ongoing collaboration with INGEMM in Phelan McDermid Syndrome (PMS) at the International Conference on Autism, ICA-2021, which will be held on September 23-24.
  • The communication entitled "Phenotype and Psychometric Characterization of Phelan McDermid Syndrome Patients" will be presented by Dr. Carlos Buesa, Oryzons CEO.
  • Executive directors of the company will attend the BIO Spain 2021 event, which will take place September 27-October 1.
  • The company will moderate a KOL panel discussion on precision medicine therapeutic approaches for CNS disorders and the potential of LSD1 inhibitors like vafidemstat.