Inclusion body myositis

A Transformational Year: ImaginAb expands Pipeline to include Radiopharmaceutical Therapies, enters Multiple Partnerships, and Drives Forward Development of Investigational CD8 ImmunoPET Agent

Retrieved on: 
Tuesday, October 4, 2022
CD8, Society for Immunotherapy of Cancer, RPT, Prognosis, GMA, Society, GMP, AstraZeneca, Boehringer Ingelheim, Medication, Technology, BOT, Research, IND, Biologics license application, Memorial Sloan Kettering Cancer Center, Immunotherapy, Cave rescue, MSKCC, Atezolizumab, Patient, Papillary renal cell carcinoma, COVID-19, Phase II, Roche, CRO, PET, University of California, Davis, Partnership, BLA, Pfizer, Inclusion body myositis, SITC, United Kingdom, IPredict, Radiopharmaceutical, University, IBM, Melanoma, Data analysis, CMO, Fine chemical, Pharmaceutical industry, Medical imaging, Biotechnology

ImaginAb continues the clinical development of CD8 ImmunoPET with a target to file a Biologics License Application (BLA) submission in the next two years.

Key Points: 
  • ImaginAb continues the clinical development of CD8 ImmunoPET with a target to file a Biologics License Application (BLA) submission in the next two years.
  • In August 2021, ImaginAb completed its Phase I IBM study showing utility of CD8 ImmunoPET to detect disease pathology in Inclusion Body Myositis (IBM) patients.
  • ImaginAb formally launched its Phase IIb 'iPREDICT' study to measure CD8 ImmunoPET imaging predictivity and prognosis, building on the data from its promising Phase IIa 'BOT' study.
  • These GMA agreements include:
    PharmaLogic, the fastest growing radiopharmaceutical manufacturer in North America, to manufacture and distribute CD8 ImmunoPET for ImaginAb.

A Transformational Year: ImaginAb expands Pipeline to include Radiopharmaceutical Therapies, enters Multiple Partnerships, and Drives Forward Development of Investigational CD8 ImmunoPET Agent

Retrieved on: 
Tuesday, October 4, 2022
CD8, Society for Immunotherapy of Cancer, RPT, Prognosis, GMA, Society, GMP, AstraZeneca, Boehringer Ingelheim, Medication, Technology, BOT, Research, IND, Biologics license application, Memorial Sloan Kettering Cancer Center, Immunotherapy, Cave rescue, MSKCC, Atezolizumab, Patient, Papillary renal cell carcinoma, COVID-19, Phase II, Roche, CRO, PET, University of California, Davis, Partnership, BLA, Pfizer, Inclusion body myositis, SITC, United Kingdom, IPredict, Radiopharmaceutical, University, IBM, Melanoma, Data analysis, CMO, Fine chemical, Pharmaceutical industry, Medical imaging, Biotechnology

ImaginAb continues the clinical development of CD8 ImmunoPET with a target to file a Biologics License Application (BLA) submission in the next two years.

Key Points: 
  • ImaginAb continues the clinical development of CD8 ImmunoPET with a target to file a Biologics License Application (BLA) submission in the next two years.
  • In August 2021, ImaginAb completed its Phase I IBM study showing utility of CD8 ImmunoPET to detect disease pathology in Inclusion Body Myositis (IBM) patients.
  • ImaginAb formally launched its Phase IIb 'iPREDICT' study to measure CD8 ImmunoPET imaging predictivity and prognosis, building on the data from its promising Phase IIa 'BOT' study.
  • These GMA agreements include:
    PharmaLogic, the fastest growing radiopharmaceutical manufacturer in North America, to manufacture and distribute CD8 ImmunoPET for ImaginAb.

Local Non-Profit Partners with Rock Legend to Fight Rare Disease

Retrieved on: 
Tuesday, February 15, 2022
Grammy Awards, Inclusion body myositis, Medical college, Hospital, Patient, Autoimmune disease, Human, Degenerative disease, Science, Medical College of Wisconsin, Johns Hopkins, Education, Johns Hopkins School of Medicine, Microdeletion syndrome, Goldenhar syndrome, Precision medicine, Awareness, Children's Wisconsin, Music, Diary, IBM, Disease, Harmony, Fund, Research, Diagnosis, Horizon Therapeutics, Autobiography, Human Genome Sciences, Pharmaceutical industry, Entertainment

"We could not be more pleased to welcome Peter Frampton as a Rare Storyteller," said Kerry Hughes, Executive Director of Harmony 4 Hope.

Key Points: 
  • "We could not be more pleased to welcome Peter Frampton as a Rare Storyteller," said Kerry Hughes, Executive Director of Harmony 4 Hope.
  • "Rare Storytellers is a rapidly growing network of patients, musicians and medical professionals currently representing over 30 rare diseases.
  • Peter Frampton, one of the most celebrated artists in rock history, was diagnosed with Inclusion Body Myositis (IBM), a rare autoimmune disease, in 2015.
  • Through music centered fundraisers and corporate sponsored Rare Storytellers, H4H provides grants to external research entities annually.

SHAREHOLDER ALERT: Pomerantz Law Firm Reminds Shareholders with Losses on their Investment in Orphazyme A/S of Class Action Lawsuit and Upcoming Deadline – ORPH

Retrieved on: 
Wednesday, September 1, 2021
Company, Niemann–Pick disease, ALS, Securities Exchange Act of 1934, Lists of schools in England, Register, Eastern Division, Securities Act of 1933, U.S. Securities and Exchange Commission, Form, GLOBE, SEC, Complaint, Lists of action films, Court, Ext, Person, Food, Endpoint security, NDA, Alpha, ADS, Inclusion body myositis, Clinical trial, IPO, NASDAQ, Amyotrophic lateral sclerosis, Safety, Prospectus, NPC, News, Patient, Wind, District court, Class, IBM, Registration statement, Guggenheim, Defendant, Nasdaq, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Security (finance), Pharmaceutical industry, Animal

NEW YORK, Aug. 31, 2021 (GLOBE NEWSWIRE) -- Pomerantz LLP announces that a class action lawsuit has been filed against Orphazyme A/S (Orphazyme or the Company) (NASDAQ: ORPH) and certain of its officers and directors.

Key Points: 
  • NEW YORK, Aug. 31, 2021 (GLOBE NEWSWIRE) -- Pomerantz LLP announces that a class action lawsuit has been filed against Orphazyme A/S (Orphazyme or the Company) (NASDAQ: ORPH) and certain of its officers and directors.
  • To discuss this action, contact Robert S. Willoughby at [email protected] or 888.476.6529 (or 888.4-POMLAW), toll-free, Ext.
  • Those who inquire by e-mail are encouraged to include their mailing address, telephone number, and the number of shares purchased.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, the Pomerantz Firm pioneered the field of securities class actions.

Orphazyme reports business highlights and financial results in Interim Report First Half 2021

Retrieved on: 
Tuesday, August 31, 2021
Journal of Inherited Metabolic Disease, Emergency Use Authorization, Journal, ALS, EMA, CHMP, Interim, Marginal cost, Market Access Program, ATU, DKK, JIMD, FTE, Research, Endpoint security, Management, Inclusion body myositis, Review, Safety, EAP, HIV disease progression rates, Patient, IBM, Conference, Sale, FDA, Q&A, Animal, Pharmaceutical industry

Copenhagen,Denmark,August 31,2021 Orphazyme A/S (ORPHA.CO; ORPH) (the Company), a late-stage biopharmaceutical company, today announces its Interim Report First Half 2021 for the period January 1 June 30, 2021.

Key Points: 
  • Copenhagen,Denmark,August 31,2021 Orphazyme A/S (ORPHA.CO; ORPH) (the Company), a late-stage biopharmaceutical company, today announces its Interim Report First Half 2021 for the period January 1 June 30, 2021.
  • We anticipate reaching net revenues of between DKK 30 and DKK 40 million by year-end December 31, 2021.
  • Orphazyme will host an investor call during which Management will present the Interim Report First Half 2021.
  • The call will be held on Tuesday, August 31, 2021 at 2.00 PM CEST/8.00 AM EDT.

SHAREHOLDER ALERT: Pomerantz Law Firm Reminds Shareholders with Losses on their Investment in Orphazyme A/S of Class Action Lawsuit and Upcoming Deadline – ORPH

Retrieved on: 
Saturday, August 14, 2021
Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Guggenheim, Court, NASDAQ, U.S. Securities and Exchange Commission, Class, Form, NPC, Prospectus, Registration statement, Clinical trial, Ext, News, Wind, District court, Company, Person, Food, Securities Act of 1933, Lists of action films, Nasdaq, Complaint, GLOBE, SEC, Niemann–Pick disease, NDA, FDA, Amyotrophic lateral sclerosis, Register, Defendant, Patient, Inclusion body myositis, ALS, Lists of schools in England, Securities Exchange Act of 1934, IPO, ADS, Alpha, Safety, Eastern Division, Endpoint security, IBM, Security (finance), Pharmaceutical industry, Animal

NEW YORK, Aug. 13, 2021 (GLOBE NEWSWIRE) -- Pomerantz LLP announces that a class action lawsuit has been filed against Orphazyme A/S (Orphazyme or the Company) (NASDAQ: ORPH) and certain of its officers and directors.

Key Points: 
  • NEW YORK, Aug. 13, 2021 (GLOBE NEWSWIRE) -- Pomerantz LLP announces that a class action lawsuit has been filed against Orphazyme A/S (Orphazyme or the Company) (NASDAQ: ORPH) and certain of its officers and directors.
  • To discuss this action, contact Robert S. Willoughby at [email protected] or 888.476.6529 (or 888.4-POMLAW), toll-free, Ext.
  • Those who inquire by e-mail are encouraged to include their mailing address, telephone number, and the number of shares purchased.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, the Pomerantz Firm pioneered the field of securities class actions.

SHAREHOLDER ALERT: Pomerantz Law Firm Reminds Shareholders with Losses on their Investment in Orphazyme A/S of Class Action Lawsuit and Upcoming Deadline – ORPH

Retrieved on: 
Thursday, August 5, 2021
NDA, Company, Register, Form, Clinical trial, U.S. Securities and Exchange Commission, Food, Prospectus, Eastern Division, Safety, Registration statement, District court, Securities Act of 1933, Complaint, NASDAQ, IBM, Endpoint security, Patient, Amyotrophic lateral sclerosis, Lists of schools in England, ADS, IPO, Securities Exchange Act of 1934, Niemann–Pick disease, Wind, Person, Inclusion body myositis, FDA, Court, Lists of action films, Defendant, Class, SEC, News, GLOBE, Nasdaq, Ext, Guggenheim, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NPC, Alpha, ALS, Security (finance), Pharmaceutical industry, Animal

NEW YORK, Aug. 05, 2021 (GLOBE NEWSWIRE) -- Pomerantz LLP announces that a class action lawsuit has been filed against Orphazyme A/S (Orphazyme or the Company) (NASDAQ: ORPH) and certain of its officers and directors.

Key Points: 
  • NEW YORK, Aug. 05, 2021 (GLOBE NEWSWIRE) -- Pomerantz LLP announces that a class action lawsuit has been filed against Orphazyme A/S (Orphazyme or the Company) (NASDAQ: ORPH) and certain of its officers and directors.
  • To discuss this action, contact Robert S. Willoughby at [email protected] or 888.476.6529 (or 888.4-POMLAW), toll-free, Ext.
  • Those who inquire by e-mail are encouraged to include their mailing address, telephone number, and the number of shares purchased.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, the Pomerantz Firm pioneered the field of securities class actions.

CytRx Notes Orphazyme’s Announcement of Topline Results from Trial for Arimoclomol in the Treatment of Inclusion Body Myositis

Retrieved on: 
Thursday, April 1, 2021
Infectious diseases, FDA, Clinical trials, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Oncology, Branches of biology, Rare diseases, Neurological disorders, Medical specialties, Lipid storage disorders, Myositis, Inclusion bodies, Niemann–Pick disease, type C, Niemann–Pick disease, Niemann, Inclusion body myositis, Amyotrophic lateral sclerosis

According to Orphazyme, the primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale.

Key Points: 
  • According to Orphazyme, the primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale.
  • Participants were randomized (1:1 ratio) to receive either arimoclomol citrate (400 mg three times daily) or placebo for up to 20 months.
  • Orphazyme is developing arimoclomol in four indications including Amyotrophic Lateral Sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and Inclusion Body Myositis ("IBM").
  • Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.

Reporting of transactions in Orphazyme’s shares made by persons discharging managerial responsibilities

Retrieved on: 
Monday, February 1, 2021
Rare diseases, Neurological disorders, Health, Clinical medicine, Lipid storage disorders, Niemann–Pick disease, type C, Inclusion body myositis, Niemann–Pick disease, Amyotrophic lateral sclerosis, Myositis, Breakthrough therapy

Arimoclomol, the Orphazymes lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), sporadic Inclusion Body Myositis (sIBM) and Gaucher disease.

Key Points: 
  • Arimoclomol, the Orphazymes lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), sporadic Inclusion Body Myositis (sIBM) and Gaucher disease.
  • Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland.
  • Orphazymes shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA).
  • In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC.

CytRx Issues Statement Regarding U.S. Regulatory Review of Arimoclomol for Niemann-Pick Disease Type C

Retrieved on: 
Thursday, December 31, 2020
Oncology, FDA, Health, Clinical trials, Pharmaceutical, Biotechnology, Rare diseases, Neurological disorders, Medical specialties, Branches of biology, Lipid storage disorders, Niemann–Pick disease, type C, Inclusion body myositis, Niemann–Pick disease, Myositis, Inclusion bodies, Priority review, Amyotrophic lateral sclerosis, CytRx Corporation, Orphazyme

Notably, the FDA has confirmed that the extension does not impede eligibility for a Pediatric Rare Disease Priority Review Voucher.

Key Points: 
  • Notably, the FDA has confirmed that the extension does not impede eligibility for a Pediatric Rare Disease Priority Review Voucher.
  • Arimoclomol previously received the FDAs Fast-Track and Breakthrough Therapy Designations for NPC as well as Orphan Drug and Rare Pediatric Disease Designations.
  • Orphazyme is developing arimoclomol in four indications including Amyotrophic Lateral Sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).
  • Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.