Tag: DIS

BEERSE, BELGIUM, June 26, 2026 (GLOBE NEWSWIRE) -- Johnson & Johnson today announced new data across 12 abstracts at the European Academy of Neurology (EAN) 2026 Congress that offer additional insight into the use of IMAAVY^®▼(nipocalimab) throughout clinically relevant points in the generalised myasthenia gravis (gMG) treatment journey. The analyses include adults with anti-AChR^a or anti-MuSK^b antibody-positive gMG who were early in their disease course or had lower baseline symptom burden – providing insight into the potential importance of addressing pathogenic immunoglobulin G (IgG) early in disease progression where use of advanced therapies may be less common.^1,2 Additional research to be shared include outcomes shortly after common infections, which are a known cause of disease exacerbations in gMG, and plans to address evidence gaps in the use of nipocalimab during pregnancy.^3,4

SAN DIEGO, June 26, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference, taking place June 25-27, 2026, in Orlando, Florida, alongside previously reported results from the Company’s HOPE-3 Phase 3 trial.

MANCHESTER, United Kingdom and OSAKA, Japan, June 18, 2026 (GLOBE NEWSWIRE) -- F2G Ltd and Shionogi & Co., Ltd (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D) (“Shionogi”) today announced positive topline results from the global Phase 3 OASIS study (NCT05101187), comparing the investigational oral antifungal drug olorofim versus AmBisome® (liposomal amphotericin B for injection) followed by standard of care (SOC) in patients with invasive aspergillosis whose infection is either refractory to or unsuitable for azole therapy.

HAMPTON, N.J., June 14, 2026 (GLOBE NEWSWIRE) -- Celldex (NASDAQ:CLDX) announced today the presentation of long-term results from the Phase 2 study of barzolvolimab in a flash talk session at the European Academy of Allergy and Clinical Immunology (EAACI) Annual Meeting in Istanbul, Türkiye. The data presented demonstrated that barzolvolimab treatment results in rapid, significant, and durable improvements in angioedema in patients with chronic spontaneous urticaria (CSU) refractory to antihistamines. These results were sustained off-treatment, seven months after completion of barzolvolimab dosing (Week 76). The data continue to demonstrate barzolvolimab’s potential to shift the goal of CSU treatment from symptom control to disease modification and further support the ongoing Phase 3 trials of barzolvolimab in CSU.

Basel, June 12, 2026 – Novartis today presented data from the RemIND trial at the European Academy of Allergy and Clinical Immunology (EAACI) Congress showing that Rhapsido^®  (remibrutinib) met its primary endpoints across the three most common chronic inducible urticaria (CIndU) subtypes, becoming the first-ever treatment to demonstrate efficacy in a global Phase III CIndU clinical trial⁷. In the RemIND trial, higher rates of complete responses were observed at week 12, with responses seen as early as week 2 in two subtypes. These results demonstrate that Rhapsido may provide sustained relief for patients whose disease remains inadequately controlled after treatment with second-generation H1-antihistamines⁷.

“Chronic inducible urticaria (CIndU) is a form of chronic hives in which everyday triggers—such as pressure, heat, cold, or sunlight—can lead to itchy wheals, and there are currently no approved targeted treatment options,” said Prof. Dr. med. Martin Metz, Deputy Director, Institute of Allergology, Charité–Universitätsmedizin Berlin, Germany. “The RemIND results across the three most common CIndU subtypes highlight the potential of Rhapsido as an important new treatment option for patients with significant unmet need.”

The three most prevalent CIndU subtypes are symptomatic dermographism (SD), cold urticaria, and cholinergic urticaria, with SD being the most common⁸. Novartis has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking approval of Rhapsido for the treatment of SD subtype and will continue additional filings to health authorities globally throughout 2026. Rhapsido is approved for use in the U.S., European Union, China and several other countries for the treatment of chronic spontaneous urticaria (CSU), in adult patients with inadequate response to H1-antihistamines.

“Rhapsido significantly improves symptom control for patients living with the three most common subtypes of chronic inducible urticaria, and it has the potential to become the first approved targeted therapy. This is a major step forward for CIndU patients who have limited options,” said Angelika Jahreis, Global Head, Immunology Development, Novartis. “The CIndU data presented today are consistent with Rhapsido’s proven efficacy and favorable safety profile in chronic spontaneous urticaria and demonstrate Novartis’ commitment to developing truly meaningful innovation for patients with complex immune-mediated diseases.”

Primary endpoint results at Week 12 in the RemIND trial¹

MILAN, Italy, June 10, 2026 – Italfarmaco S.p.A. today announced that Health Canada has accepted for review the New Drug Submission (NDS) for givinostat (Duvyzat^®) for the treatment of Duchenne muscular dystrophy (DMD). Health Canada has granted givinostat Priority Review status, a designation reserved for drugs that may provide a significant improvement in the benefit-risk profile over existing therapies.

“Families affected by Duchenne muscular dystrophy in Canada currently have limited access to treatment options that can slow functional decline,” said Dr. Jean K. Mah, a Canadian investigator for the EPIDYS and OLE studies and Director of the Pediatric Neuromuscular Program at the Alberta Children’s Hospital in Calgary, Alberta. “As a clinician treating DMD patients, I am encouraged by this positive step in the Canadian regulatory progress and givinostat’s therapeutic potential demonstrated in the EPIDYS Phase 3 study."

An estimated 1000 boys in Canada are living with DMD, one of the most severe and common forms of childhood muscular dystrophy.¹ As part of its commitment to the Canadian DMD community, Italfarmaco will establish a local Canadian affiliate to lead ongoing discussions with health authorities throughout the regulatory review process and to support patients and their families as well as healthcare providers. Subject to Health Canada’s review, marketing authorisation could be granted before the end of 2026.

“Duchenne muscular dystrophy places an enormous burden on boys and their families, and we understand the urgency of bringing new treatment options to communities where significant unmet need remains,” said Francesco Di Marco, Chief Executive Officer of Italfarmaco Group. “Health Canada’s acceptance of the New Drug Submission for givinostat with Priority Review is an encouraging step forward. As we expand our presence in Canada, we are committed to working with clinicians, patient organisations, and other stakeholders to support the Duchenne community and help advance access for appropriate patients, if approved.”

The submission builds on many years of clinical research, including contributions from Canadian patients, families, investigators, and three clinical trial sites in Canada. It is supported by data from the EPIDYS Phase 3 multicentre, randomized, double-blind, placebo-controlled trial (NCT02851797) evaluating givinostat in ambulant boys with DMD aged six and older. The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment in patients treated with givinostat twice daily, in addition to corticosteroids, compared to placebo and corticosteroids. During the study, the most common treatment-related adverse events (frequency ≥1/10 boys) associated with givinostat were decreased platelet count/thrombocytopenia, increased blood triglyceride/hypertriglyceridemia, diarrhoea and abdominal pain; none of the severe or serious adverse events were treatment-related or resulted in study withdrawal. givinostat tolerability was managed with appropriate monitoring and dose adjustments. No other safety concerns were observed.² Patients from the EPIDYS study remain under observation in a long-term study assessing tolerability and efficacy, with follow-up extending up to eleven years (NCT03373968).

Givinostat has received regulatory approvals in multiple regions, including the US, the UK, the EU, and the UAE for the treatment of DMD. Additional regulatory submissions are ongoing in other geographies, reflecting Italfarmaco’s commitment to enabling access for as many individuals living with DMD as possible.


About Duchenne Muscular Dystrophy

BRUSSELS, Belgium and CAMBRIDGE, Mass., June 04, 2026 (GLOBE NEWSWIRE) -- UCB (Euronext Brussels: UCB) and Biogen Inc. (Nasdaq: BIIB) today announced data, comprising two posters and three abstracts, at the European Alliance of Associations for Rheumatology (EULAR) 2026 Congress, demonstrating the clinical profile of dapirolizumab pegol (DZP), an investigational biologic in patients with systemic lupus erythematosus (SLE).

– EZMEKLY is the first and only therapy to receive marketing authorization in the EU for both adults and children (≥2 years) with NF1-PN, a rare genetic disorder with debilitating symptoms –

– EZMEKLY is the first and only therapy to receive marketing authorization in the EU for both adults and children (≥2 years) with NF1-PN, a rare genetic disorder with debilitating symptoms –