MOUNTAIN VIEW, Calif., June 24, 2025 (GLOBE NEWSWIRE) -- Iridex Corporation (Nasdaq: IRIX), a worldwide leader providing innovative and versatile laser-based medical systems, delivery devices, and accessories for the treatment of glaucoma and retinal diseases, announced today that the first patient has been successfully enrolled in the independent, investigator-led DAME Trial to evaluate the clinical efficacy, safety, cost-effectiveness, and patient acceptability of adding subthreshold MicroPulse treatments ...

Core Insights - Immunic, Inc. reported long-term data from the phase 2 EMPhASIS trial of vidofludimus calcium, showing 92.3% of patients remained free of 12-week confirmed disability worsening (CDW) and 92.7% free of 24-week CDW at week 144 [1][3] - The drug demonstrated a favorable safety and tolerability profile, with no new safety signals emerging during treatment durations of up to 5.5 years [1][3] Group 1: Clinical Trial Data - The phase 2 EMPhASIS trial included 268 patients with relapsing-remitting multiple sclerosis (RRMS), achieving both primary and key secondary endpoints with high statistical significance [4] - A total of 29 CDW events were confirmed at 12 weeks, with 44.8% associated with relapse-associated worsening (RAW) and 13.8% with progression independent of relapse activity (PIRA) [3] - The open-label extension (OLE) period provided approximately 952 treatment years of data, reinforcing the drug's safety and tolerability [2][3] Group 2: Drug Mechanism and Potential - Vidofludimus calcium is a first-in-class nuclear receptor-related 1 (Nurr1) activator, combining neuroprotective, anti-inflammatory, and anti-viral effects [6][7] - The drug has shown therapeutic activity in both relapsing-remitting and progressive multiple sclerosis patients, significantly reducing brain lesions and confirmed disability worsening [6][7] - The ongoing phase 3 clinical trials aim to further evaluate the drug's efficacy and safety, with top-line data expected by the end of 2026 [7]

Core Insights - Liquidia Corporation has received an additional $50 million under its financing agreement with Healthcare Royalty following a legal victory against United Therapeutics Corporation, allowing for the commercial sale of YUTREPIA™ [1][2] - The funding will accelerate the launch of YUTREPIA, advance Liquidia's clinical pipeline, and support future manufacturing operations [2] - Liquidia has now received a total of $175 million of the $200 million available under the financing agreement, with an additional $25 million contingent on achieving net sales of YUTREPIA exceeding $100 million by June 30, 2026 [2] Company Overview - Liquidia Corporation is focused on developing innovative therapies for rare cardiopulmonary diseases, particularly pulmonary hypertension [8] - The company utilizes its proprietary PRINT® technology to create drug formulations, including YUTREPIA, which is approved for treating pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) [8] - Liquidia is also developing L606, a sustained-release formulation of treprostinil, and markets generic Treprostinil Injection for PAH treatment [8] Product Information - YUTREPIA is an inhaled dry-powder formulation of treprostinil designed for ease of use and enhanced lung deposition [5] - The product is currently undergoing clinical trials, including the ASCENT trial, to evaluate its safety and tolerability in patients with PH-ILD [5] - YUTREPIA was previously known as LIQ861 during its investigational studies [5] Market Context - Pulmonary arterial hypertension (PAH) affects an estimated 45,000 patients in the U.S., with no current cure, making symptom management and quality of life improvement critical [3] - Pulmonary hypertension associated with interstitial lung disease (PH-ILD) has a prevalence of over 60,000 patients in the U.S., with many cases underdiagnosed [4]

Core Insights - Novo Nordisk announced new data from the phase III FRONTIER clinical program for its investigational candidate, Mim8, as a prophylaxis treatment for hemophilia A, showing promising results in switching from Roche's Hemlibra treatment [1][2][4] Group 1: Study Results - The phase IIIb FRONTIER5 study indicated that switching from Hemlibra to Mim8 was well-tolerated with no safety issues reported in adults and adolescents, regardless of inhibitor status [2][4][7] - Patient-reported outcomes showed a strong preference for the Mim8 pen-injector, which was easier to use compared to the Hemlibra injection system, suggesting high potential for patient compliance [2][4] - Mim8 maintained clotting activity without adverse events or antibody detection, with no thromboembolic events or treatment-related adverse events leading to discontinuation [4][7] Group 2: Treatment Administration - Patients received their first Mim8 maintenance dose on the scheduled day of their next Hemlibra dose, with options for weekly, biweekly, or monthly dosing [6] - Steady-state levels of Mim8 were achieved by week 16, and Roche's Hemlibra was fully cleared by week 26, indicating a smooth transition [6] Group 3: Future Outlook - Novo Nordisk plans to submit Mim8 for regulatory review in 2025, with additional data from the phase III FRONTIER program expected in 2025 and 2026 [8] - Currently, Mim8 is not approved by regulatory authorities anywhere in the world [8] Group 4: Industry Context - Hemophilia A affects approximately 1.1 million people globally, with up to 30% of those with severe hemophilia A developing inhibitors that reduce the effectiveness of replacement therapies [9]

Core Insights - Cumberland Pharmaceuticals Inc. presented positive results from the Phase 2 FIGHT DMD trial for ifetroban, a novel oral therapy targeting Duchenne muscular dystrophy (DMD) heart disease, at the PPMD conference, indicating its potential to protect the heart and reduce cardiac damage in DMD patients [1][2][8] Group 1: Trial Results - The 12-month Phase 2 FIGHT DMD trial demonstrated a significant 5.4% improvement in left ventricular ejection fraction (LVEF) in patients treated with high-dose ifetroban compared to a control group [4] - High-dose ifetroban treatment was associated with reduced blood levels of cardiac damage markers (NT-proBNP and cardiac troponin I), while these markers increased in placebo-treated patients, suggesting ifetroban's potential to prevent ongoing cardiac injury [5] - All patients who completed the study opted to continue with the open-label extension, indicating confidence in the treatment [7] Group 2: Pharmacokinetics and Tolerability - The study revealed that DMD patients receiving higher doses of ifetroban achieved similar plasma levels to typical adults without evidence of drug accumulation, supporting the 300 mg daily dosing used in the high-dose group [6] - Ifetroban was well-tolerated with an acceptable pharmacokinetic profile in patients with DMD, despite the higher dosing requirements [6] Group 3: Market Potential and Regulatory Pathway - Ifetroban is positioned to address the leading cause of death in DMD patients, with no approved treatment specifically targeting DMD heart disease, highlighting a critical unmet medical need [9][10] - The company plans to analyze long-term treatment results and engage in discussions with the FDA regarding the regulatory pathway forward based on the trial's encouraging results [10]

ArriVent BioPharma (AVBP) Update / Briefing June 23, 2025 08:00 AM ET Speaker0 Good morning and welcome to the Forte Biosciences Investor Webcast. At this time, all attendees are in a listen only mode. A Q and A session will follow the formal presentations. As a reminder, this call is being recorded and a replay will be made available on the Forte Biosciences Biosciences website following the conclusion of the event. I'd now like to turn the call over to Doctor. Paul Wagner, Chief Executive Officer of Forte ...

FORTE BIOSCIENCES CLINICAL STAGE FB-102 3 FB102 CELIAC DISEASE PHASE 1B RESULTS JUNE 2025 1 CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS 2 Certain statements contained in this presentation regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities and Exchange Act of 1934, as amended, and the Private Securities Litigation Act of 1995, known as the PSLRA. These include statements regarding management's intention, plans, beliefs, ...

Core Insights - Johnson & Johnson announced new data from an indirect treatment comparison (ITC) showing that IMAAVY (nipocalimab-aahu) provides consistent and sustained disease control in adults with generalized myasthenia gravis (gMG) compared to other approved FcRn blockers [1][2][4] Company Overview - IMAAVY received U.S. FDA approval for a broad population of gMG patients, including those who are anti-AChR and anti-MuSK antibody positive, as well as pediatric patients aged 12 and older [1][4] - The company is committed to helping patients with chronic autoantibody conditions and continues to research the potential impact of IMAAVY [4][10] Treatment Efficacy - The ITC included data from the pivotal Phase 3 Vivacity-MG3 study, demonstrating that IMAAVY showed comparable onset of symptom relief at Week 1 and statistically significant improvements in MG-ADL scores at multiple timepoints up to 24 weeks [2][5][9] - IMAAVY exhibited significantly greater mean improvements in MG-ADL scores compared to other FcRn blockers, with notable differences observed at Weeks 8-24 [5][8] Regulatory and Market Position - IMAAVY is approved in the U.S. for adult and pediatric patients with gMG, and a Marketing Authorisation Application (MAA) has been submitted to the European Medicines Agency (EMA) for approval in Europe [4][10] - The ITC methodology used in the analysis adheres to global health technology assessment standards, providing valuable comparative data for regulatory agencies and medical guideline committees [4][7] Patient Population and Disease Context - Myasthenia gravis affects an estimated 700,000 people worldwide, with approximately 100,000 individuals in the U.S. living with gMG [6][9] - The disease predominantly affects women and can manifest in pediatric patients, with a significant portion of cases diagnosed in girls [6][9]

MELBOURNE, Australia and SAN FRANCISCO, June 23, 2025 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that David Stamler, M.D., Chief Executive Officer of Alterity, will participate in a Fireside Chat hosted by MST Access on Wednesday, 25 June 2025 in Australia / Tuesday, 24 June 2025 in the United States. Dr. Stamler will provide a co ...

Core Insights - Skye Bioscience, Inc. is focused on developing nimacimab, an anti-obesity drug that targets peripheral CB1 receptors, aiming to address unmet needs in obesity treatment [1][2][3] - The company has launched a video series titled "Anatomy of Progress" to provide updates on nimacimab's development and its advantages over traditional treatments [1][2] Group 1: Product Development - Nimacimab is a peripherally-restricted CB1 antibody that aims to provide weight loss benefits without the neuropsychiatric side effects associated with small molecule CB1 inhibitors [4][3] - The video series discusses the mechanistic advantages of nimacimab, emphasizing its potential to revolutionize obesity treatment by addressing biological resistance to traditional therapies [3][4] Group 2: Clinical Insights - Skye's management participated in a panel discussing the clinical and preclinical experiences with nimacimab, highlighting its peripheral blockade of the CB1 pathway [5] - Presentations at the American Diabetes Association's 85th Scientific Sessions included data on nimacimab's effectiveness in promoting weight loss and reducing obesity-induced inflammation [6][8] Group 3: Market Positioning - The company is positioning nimacimab as a differentiated therapeutic option in the obesity market, particularly as a non-GLP1 alternative, based on feedback from obesity physicians [7] - The market opportunity for nimacimab is framed by its unique target product profile, which is expected to complement existing incretin-based therapies [7][9] Group 4: Research Findings - Preclinical studies demonstrated that nimacimab promotes metabolic homeostasis and improves hormonal regulation in diet-induced obesity models [8] - New biomarker data presented indicates significant reductions in obesity-related inflammation and liver steatosis, supporting nimacimab's potential as a standalone and combination therapy [6][8]