EMA orphan drug designation is awarded to drugs intended to treat rare diseases that are life-threatening or chronically debilitating Preeclampsia, a serious obstetric complication, is a leading cause of near-term morbidity and mortality for mothers and their babies, and can lead to long-term consequences for both An investigational drug candidate, CBP-4888 leverages the precise targeting ability of siRNAs to downregulate sFlt1, a key pathogenic mediator of preeclampsia, in the placenta CBP-4888

April 08, 2025 -- Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, is supporting key educational programs and initiatives throughout the month of April in recognition of Esophageal Cancer Awareness Month. The Company is proudly collaborating with the Esophageal Cancer Action Network (ECAN), the American Foregut Society (AFS) and The Gut Doctor PodcastLLC to promote esophageal cancer prevention, education and advocacy. “As eso

April 08, 2025 -- Lipella Pharmaceuticals Inc. (Nasdaq: LIPO) (“Lipella” or the “Company”), a clinical-stage biotechnology company focused on developing innovative therapies for unmet medical needs, today announced the completion of enrollment in its Phase 2a multicenter, dose-ranging trial evaluating LP-310, a liposomal tacrolimus oral rinse for the treatment of oral lichen planus (OLP). The Phase 2a trial has now fully enrolled participants across all three dose cohorts (0.25 mg, 0.50 mg and

–EFFICACY: 92% median reduction in disabling seizures during 7-12-month efficacy endpoint period, and 80% (4/5) of patients responding with >75% seizure reduction in Cohort 1. –DURABILITY: All responders (4/4) in Cohort 1 continue to have durable seizure control for 18-24 months after a single-dose of NRTX-1001. –COGNITION AND QUALITY OF LIFE: All patients (5/5) in Cohort 1 had significantly improved quality-of-life test scores, with no patient experiencing persistent decline in cognition. –

TNX-102 SL is a sublingual formulation of cyclobenzaprine designed for transmucosal delivery and durable activity in treating fibromyalgia TNX-102 SL demonstrated statistically significant improvement in the primary endpoint of reduction in fibromyalgia pain in two pivotal double-blind randomized Phase 3 studies FDA Prescription Drug User Fee Act (PDUFA) goal date of August 15, 2025, for TNX-102 SL for the management of fibromyalgia If approved by FDA, it would become the first member of a new c

April 07, 2025 -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million. “We have reached another key milestone with the closing on the sale of our PRV, which we received following the successful approval of MIPLYFFATM (arimoclomol) from the U.S. Food and

Reinforces potential of nebokitug to become the first approved drug with disease-modifying activity for PSC--a deadly disorder that currently has no effective treatments Treatment in PSC patients with moderate/advanced disease for 48 weeks resulted in continued improvements across key biomarkers of liver injury, inflammation and fibrosis, including ELF score, fibrosis-related ELF score components and PRO-C3 Patients treated with nebokitug for 48 weeks showed a significantly lower number of clini

2,000+ Average-Risk Patient Study Seeks to Confirm Mainz Biomed’s Industry-Leading Results in the Detection of Advanced Precancerous Lesions (APL) in Independent Samples Study is on Track to Report Top-Line Results in Fourth Quarter 2025 March 27, 2025 -- Mainz Biomed N.V. (NASDAQ:MYNZ) (“Mainz Biomed” or the “Company”), a molecular genetics diagnostic company specializing in the early detection of cancer, announced today the first patient has been enrolled in eAArly DETECT 2, a feasibility stu

Designations Follow Recent Investigational New Drug (IND) Application Clearance for the RESOLUTION Basket Study of ALLO-329 in Rheumatology Dual CD19/CD70 CAR T Specifically Designed to Enhance Therapeutic Benefit, Expanding Treatment Potential Across a Range of Autoimmune Indications Leverages Proprietary Dagger® Technology to Reduce or Eliminate Lymphodepletion, Potentially Expanding Access to a Broader Patient Population Phase 1 RESOLUTION Trial Initiation Planned for Mid-2025 with Initial Pr

-- AjD is a Mental Health Condition Triggered By a Stressful Life Event; Particularly Common in People with Serious Medical Illness -- -- AjD Represents an Area of Significant Unmet Need; No FDA-Approved Therapies Currently Available -- -- Expansion of Clinical Development Program into AjD in Cancer and Other Medical Illnesses Reflects Broad Potential of RE104 to Revolutionize Mental Health Treatment -- -- Initiation of REKINDLE Phase 2 Trial Expected Mid-2025 -- April 07, 2025 -- Reunion Neuro