Periodic fever syndrome

Cerecor Doses First Patient in a Phase 1b Proof-of-Concept Clinical Trial of CERC-007 for the Treatment of Adult Onset Still’s Disease

Retrieved on: 
Wednesday, May 5, 2021
Medical specialties, Clinical medicine, Medicine, Rheumatology, Immunology, Blood tests, Ferritin, Iron metabolism, Monoclonal antibody, AOSD, Arthritis, Periodic fever syndrome

The Company anticipates initial data to be reported in the third quarter of 2021.\nCERC-007 is a high affinity, fully human anti-IL-18 monoclonal antibody (mAb).

Key Points: 
  • The Company anticipates initial data to be reported in the third quarter of 2021.\nCERC-007 is a high affinity, fully human anti-IL-18 monoclonal antibody (mAb).
  • IL-18 has been demonstrated to have a key role as a marker of disease activity and its concentration correlates with disease severity in AOSD patients.
  • AOSD is a rare auto-inflammatory disease associated with fever, rash, pharyngitis, arthritis, liver disease, and increased ferritin.
  • The primary objective of the study will be to determine the safety and tolerability of CERC-007 in AOSD patients.

AB2 Bio to Participate in Orphan Disease Panel Discussion at the LifeSci Partners 10th Annual Healthcare Corporate Access Event

Retrieved on: 
Tuesday, January 5, 2021
Medical specialties, Autoinflammatory syndromes, Clinical medicine, Periodic fever syndrome, Hemophagocytic lymphohistiocytosis, Medicine

The panel is entitled Rare Advantages and Unique Challenges Faced by Orphan Companies.

Key Points: 
  • The panel is entitled Rare Advantages and Unique Challenges Faced by Orphan Companies.
  • AB2 Bio is building a late-stage clinical pipeline with Tadekinig alfa, a novel IL-18 binding protein with established clinical proof-of-concept in three, life-threatening orphan autoinflammatory indications.
  • Tadekinig alfa is currently in late-stage development for the treatment of severe orphan autoinflammatory diseases, including primary and secondary HLH and Stills disease.
  • AB2 Bio is building a late-stage clinical pipeline with Tadekinig alfa, a novel IL-18 binding protein with established clinical proof-of-concept in three, life-threatening orphan autoinflammatory indications.

Improving the Diagnosis of Chronic Nonbacterial Osteomyelitis, an Underdiagnosed Chronic Autoinflammatory Syndrome

Retrieved on: 
Sunday, November 8, 2020
Bacterial diseases, Medical specialties, Skeletal disorders, Clinical medicine, Chronic recurrent multifocal osteomyelitis, Medicine, Autoinflammatory syndromes, Osteopathies, CRMO, Osteomyelitis, Periodic fever syndrome

NEW YORK, Nov. 8, 2020 /PRNewswire/ --Researchers have identified several factors that should help improve the diagnosis of chronic nonbacterial osteomyelitis (CNO), also known as chronic recurrent multifocal osteomyelitis (CRMO).

Key Points: 
  • NEW YORK, Nov. 8, 2020 /PRNewswire/ --Researchers have identified several factors that should help improve the diagnosis of chronic nonbacterial osteomyelitis (CNO), also known as chronic recurrent multifocal osteomyelitis (CRMO).
  • This chronic autoinflammatory syndrome, which can be debilitating, is underdiagnosed and is characterized by multiple foci of painful swelling of bones, mainly in the metaphyses of the long bones, in addition to the pelvis, the shoulder girdle and the spine.
  • Because CNO often mimics a bone infection or cancer, children with the condition often see various specialists before getting an accurate diagnosis.
  • The researchers say that clinicians can use the new information to help improve the diagnosis of CNO patients.

FDA Grants Orphan Drug Designation for Inflazome’s Inzomelid for the Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)

Retrieved on: 
Tuesday, July 14, 2020
Biotechnology, FDA, Health, Pharmaceutical, Clinical trials, Autoinflammatory syndromes, Medical specialties, Branches of biology, Health, Periodic fever syndrome, Cryopyrin-associated periodic syndrome, NALP3, Caps, Orphan drug, NLRP, Interleukin 1 beta, NLRC4, Inflazome, the NLPR3 Inflammasome, Cryopyrin-associated periodic syndromes, Orphan Drug Designation

Inflazome ( inflazome.com ), the pioneering inflammasome biotech company developing multiple drugs that stop harmful inflammation, today announces that it has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for Inzomelid in the treatment of Cryopyrin-Associated Periodic Syndrome (CAPS).

Key Points: 
  • Inflazome ( inflazome.com ), the pioneering inflammasome biotech company developing multiple drugs that stop harmful inflammation, today announces that it has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for Inzomelid in the treatment of Cryopyrin-Associated Periodic Syndrome (CAPS).
  • Orphan Drug Designation is an important regulatory milestone granted to drugs that are intended to treat rare diseases and conditions, such as CAPS.
  • Inzomelid is under development as an orally available, brain-penetrant drug and Somalix is under development as an orally available, peripherally-restricted drug.
  • Cryopyrin-associated periodic syndromes (CAPS), also called cryopyrin-associated autoinflammatory syndromes, are three diseases related to a defect in the NLRP3 gene.

Inzomelid completes Phase I studies and shows positive results in the treatment of Cryopyrin-Associated Periodic Syndrome (CAPS)

Retrieved on: 
Thursday, March 26, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Medicine, Autoinflammatory syndromes, Branches of biology, Cryopyrin-associated periodic syndrome, NALP3, Periodic fever syndrome, Muckle–Wells syndrome, Neonatal-onset multisystem inflammatory disease, FCAS, Caps, MWS, Inflazome, the NLPR3 Inflammasome, Cryopyrin-associated periodic syndromes

Inflazome ( inflazome.com ), the pioneering inflammasome biotech company developing multiple drugs that stop harmful inflammation, today announces the successful completion of a Phase I study of Inzomelid, alongside positive results from a Cryopyrin-Associated Periodic Syndrome (CAPS) patient dosed with Inzomelid.

Key Points: 
  • Inflazome ( inflazome.com ), the pioneering inflammasome biotech company developing multiple drugs that stop harmful inflammation, today announces the successful completion of a Phase I study of Inzomelid, alongside positive results from a Cryopyrin-Associated Periodic Syndrome (CAPS) patient dosed with Inzomelid.
  • The results support the progression of Inzomelid into Phase II clinical trials.
  • Drug exposures in the double blinded and randomised Phase I trial in healthy subjects increased linearly with dose, and correlated with markers of target engagement.
  • CAPS encompasses neonatal onset multisystem inflammatory disease (NOMID), Muckle-Wells syndrome (MWS) and familial cold autoinflammatory syndrome (FCAS).

Kiniksa Announces Breakthrough Therapy Designation for Rilonacept for the Treatment of Recurrent Pericarditis

Retrieved on: 
Wednesday, November 20, 2019
Medical specialties, Medicine, Immunology, Autoinflammatory syndromes, Pericarditis, RTT, Rilonacept, Periodic fever syndrome

We are pleased that the FDA has granted Breakthrough Therapy designation for rilonacept for the treatment of recurrent pericarditis, a painful and debilitating autoinflammatory cardiovascular disease, said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa.

Key Points: 
  • We are pleased that the FDA has granted Breakthrough Therapy designation for rilonacept for the treatment of recurrent pericarditis, a painful and debilitating autoinflammatory cardiovascular disease, said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa.
  • The final Phase 2 data presented at AHA showed rilonacept treatment improved clinically meaningful outcomes associated with unmet need in recurrent pericarditis, including rapid resolution of pericarditis episodes, tapering and discontinuation of corticosteroids without pericarditis recurrence, reduction in recurrences of pericarditis episodes while on treatment, and improvement in quality of life scores.
  • We continue to enroll RHAPSODY, our pivotal Phase 3 clinical trial of rilonacept in recurrent pericarditis, and expect top-line data in the second half of 2020.
  • Kiniksa is enrolling RHAPSODY, a global, randomized withdrawal (RW) design, pivotal Phase 3 clinical trial of rilonacept in patients with recurrent pericarditis in the U.S., Australia, Israel, and Italy.

Kiniksa Pharmaceuticals to Present at the 2nd Annual Evercore ISI HealthCONx Conference

Retrieved on: 
Wednesday, November 20, 2019
Finance, Investment, Evercore, Landforms, Periodic fever syndrome, Webcast, BERMUDA, NASDAQ, Lluís Companys

HAMILTON, Bermuda, Nov. 20, 2019 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) announced today that it will present at the 2nd Annual Evercore ISI HealthCONx Conference on Wednesday, December 4th, 2019 at 3:30 p.m. Eastern Time at the Four Seasons Hotel in Boston, MA.

Key Points: 
  • HAMILTON, Bermuda, Nov. 20, 2019 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) announced today that it will present at the 2nd Annual Evercore ISI HealthCONx Conference on Wednesday, December 4th, 2019 at 3:30 p.m. Eastern Time at the Four Seasons Hotel in Boston, MA.
  • A live webcast of Kiniksas presentation will be accessible through the Investors & Media section of the companys website at www.kiniksa.com .
  • A replay of the webcast will be available on Kiniksas website for 14 days following the conference.
  • Kiniksa has a pipeline of product candidates across various stages of development, focused on autoinflammatory and autoimmune conditions.

Kiniksa to Present Rilonacept Final Phase 2 Clinical Data at the American Heart Association Scientific Sessions 2019

Retrieved on: 
Monday, August 19, 2019
Medical specialties, Immunology, Medicine, Autoinflammatory syndromes, Immunosuppressants, Rilonacept, Interleukins, Periodic fever syndrome, Regeneron, Cytokines, Pericarditis

We look forward to the presentation of multiple sets of data at AHA to help broaden the understanding of disease burden and support rilonacept as a potential treatment solution for patients with recurrent pericarditis.

Key Points: 
  • We look forward to the presentation of multiple sets of data at AHA to help broaden the understanding of disease burden and support rilonacept as a potential treatment solution for patients with recurrent pericarditis.
  • Rilonacept is a weekly, subcutaneously-injected, recombinant fusion protein that blocks interleukin-1 (IL-1) and interleukin 1 (IL-1) signaling.
  • Kiniksa exclusively licensed rilonacept from Regeneron for recurrent pericarditis and certain other indications.
  • Kiniksa has a pipeline of product candidates across various stages of development, focused on autoinflammatory and autoimmune conditions.

Kiniksa Pharmaceuticals to Present at the 2019 Wedbush PacGrow Healthcare Conference

Retrieved on: 
Tuesday, August 6, 2019
Finance, Money, Landforms, Wedbush Securities, Webcast, Periodic fever syndrome, NASDAQ, BERMUDA

HAMILTON, Bermuda, Aug. 06, 2019 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) announced today that Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa, will present at the 2019 Wedbush PacGrow Healthcare Conference on Tuesday, August 13th, 2019 at 12:45 p.m. Eastern Time at The Parker New York in New York City.

Key Points: 
  • HAMILTON, Bermuda, Aug. 06, 2019 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) announced today that Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa, will present at the 2019 Wedbush PacGrow Healthcare Conference on Tuesday, August 13th, 2019 at 12:45 p.m. Eastern Time at The Parker New York in New York City.
  • A live webcast of Kiniksas presentation will be accessible through the Investors & Media section of the companys website at www.kiniksa.com .
  • A replay of the webcast will be available on Kiniksas website for 14 days following the conference.
  • Kiniksa has a pipeline of product candidates across various stages of development, focused on autoinflammatory and autoimmune conditions.

IFM Therapeutics, LLC Launches Subsidiary, IFM Tre, with $31 Million Series A Financing to Develop NLRP3 Antagonists for Inflammatory Diseases

Retrieved on: 
Thursday, July 19, 2018
Immunology, Medicine, Health, Biology, Inflammasome, NALP3, Autoimmunity, Inflammation, Immune system, Periodic fever syndrome

We have made substantial progress in our NLRP3 portfolio and look forward to bringing our candidates forward into the clinic."

Key Points: 
  • We have made substantial progress in our NLRP3 portfolio and look forward to bringing our candidates forward into the clinic."
  • However, abnormal activation of the NLRP3 inflammasome drives the onset and progression of numerous conditions, including metabolic, fibrotic, autoimmune, and neurodegenerative diseases.
  • IFM Tre's programs target the innate immune system by suppressing only the inflammation mediated by the NLRP3 pathway.
  • IFM Tre, a subsidiary of IFM Therapeutics, LLC, is a biopharmaceutical company developing a suite of small-molecule antagonists targeting inappropriate inflammatory responses of the innate immune system via the NLRP3 pathway, which is believed to underlie a variety of serious diseases.