Contact Us Back to the Newsroom Exousia Pro is Excited to Announce that it has Received Orphan Drug Designation from the FDA Tuesday, 28 October 2025 10:20 AM Topic:Â Company Update Exousia Pro is in a unique position, as only 11% of ODAs are awarded at the preclinical stage. ORLANDO, FLORIDA / ACCESS Newswire / October 28, 2025 / Exousia Pro, Inc. (formerly Marijuana, Inc.) (OTCPINK:MAJI), a clinical-stage biotech company using exosomes in the treatment of cancer and other maladies, is pleased to announce ...

PRINCETON, N.J., Oct. 23, 2025 (GLOBE NEWSWIRE) -- Grace Therapeutics, Inc. (Nasdaq: GRCE) (Grace Therapeutics or the Company), a late-stage, biopharma company advancing GTx-104, a clinical-stage, novel, injectable formulation of nimodipine being developed for I.V. infusion to address significant unmet medical needs in aneurysmal subarachnoid hemorrhage (aSAH) patients, today announced it has secured approximately $4.0 million in additional funding through exercises of common warrants that were previously i ...

Upon Approval of NDA Quoin Will Receive Seven Years Marketing Exclusivity for QRX003 in the US Orphan Drug Designation previously granted by the European Medicines Agency in May 2025 ASHBURN, Va., Oct. 21, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (“Quoin” or the “Company”), a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead ...

Accessibility StatementSkip Navigation NEW YORK, Oct. 21, 2025 /PRNewswire/ -- Hoth Therapeutics, Inc. (NASDAQ: HOTH)Â today highlighted FDA Orphan Drug Designation for HT-KIT and new preclinical data demonstrating >80% suppression of KIT expression and significant tumor-volume reduction by Day 8 in systemic mastocytosis and GIST models. HT-KIT, a precision antisense oligonucleotide (ASO) targeting KIT mRNA, also completed GLP-validated bioanalytical methods supporting IND-enabling studies; Japan Patent No. ...

Sanofi’s Tzield accepted for expedited review in the US for stage 3 type 1 diabetes through FDA Commissioner's National Priority Voucher pilot program If approved, Tzield would be the first disease-modifying therapy to delay the progression of stage 3 T1D in adults and pediatric patients eight years of age and older recently diagnosed with stage 3 T1DTzield is also being reviewed under the accelerated approval program Paris, October 20, 2025. The US Food and Drug Administration (FDA) has accepted for exped ...

Key Takeaways Esperion named ESP-2001 its new preclinical candidate for primary sclerosing cholangitis.ESP-2001 was discovered with Evotec and has shown liver injury reduction in preclinical studies.Esperion will hold exclusive global rights to develop and commercialize ESP-2001.Esperion Therapeutics (ESPR) announced that it has nominated ESP-2001, a highly specific allosteric ATP citrate lyase (“ACLY”) inhibitor, as its new preclinical development candidate for the treatment of primary sclerosing cholangit ...

公司核心事件 - Kiniksa Pharmaceuticals 宣布其候选药物 KPL-387 获得美国FDA授予的孤儿药认定，用于治疗心包炎（包括复发性心包炎）[1] - KPL-387 是一种独立开发的、完全人源化的免疫球蛋白IgG2单克隆抗体，可与人类白细胞介素-1受体1结合，抑制细胞因子IL-1α和IL-1β的信号传导活性[1][5] 药物开发进展与预期 - KPL-387 2/3期复发性心包炎试验的2期剂量探索部分数据预计将在2026年下半年公布[2] - 公司认为KPL-387有潜力通过每月一次皮下自我注射的液体剂型给药，为患者提供额外的治疗选择[2][5] 孤儿药认定的意义 - 美国FDA授予孤儿药认定的标准是所治疗的罕见病在美国患病人数少于20万[3] - 获得孤儿药认定可使公司享有财务激励，包括临床试验费用的资助机会、税收优惠和用户费用减免[3] - 孤儿药认定还豁免了该药物在该适应症中进行儿科研究的要求[3] 公司战略与业务重点 - Kiniksa是一家生物制药公司，致力于发现、获取、开发和商业化针对未满足医疗需求的疾病的新型疗法[4] - 公司的业务重点集中在心血管适应症领域[1][4] - 公司的产品组合基于强大的生物学原理或经过验证的作用机制，并具有差异化的潜力[4]

- Data from ongoing DELIVER trial demonstrated sustained functional improvement through 18 months; results from Registrational Expansion Cohort expected late 2025 - WALTHAM, Mass., Sept. 29, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company, today announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amen ...

Jaguar Health (NasdaqCM:JAGX) 2025 Conference September 25, 2025 02:55 PM ET Company ParticipantsLisa Conte - Founder, CEO, President & DirectorNoneAnd welcome back, everyone. We have an update from Jaguar Health trades on the Nasdaq under the symbol j a g x. It's a commercial stage pharmaceutical company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress. So happy to welcome back founder, ...

Akeso is actively advancing the international clinical development for ligufalimab, which is being evaluated in both hematologic malignancies and solid tumors. In addition to its application in AML, patient enrollment has been completed in a randomized, double-blind, multicenter Phase II study assessing ligufalimab combined with azacitidine in higher-risk myelodysplastic syndromes (HR-MDS). Ligufalimab is also the first CD47 monoclonal antibody to enter registrational Phase III trials in solid tumors. Two P ...