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Pipeline
| Company | Drug/Device | Medical Condition | Status |
|---|---|---|---|
| Cellenkos, Inc. | CK0801 | bone marrow failure syndromes, including aplastic anemia, hypoplastic myelodysplasia and primary myelofibrosis | Phase I trial initiated |
| Xencor, Inc. | XmAb22841 | advanced solid tumors | Phase I trial initiated |
| Alnylam Pharmaceuticals, Inc. | ALN-AGT | hypertension | Phase I trial initiated enrolling 168 subjects |
| UCB Neuropore Therapies, Inc. |
UCB0599 | Parkinson’s disease | Phase Ib trial initiated |
| Amphera B.V. | MesoPher | pancreatic cancer | Phase II trial initiated enrolling 10 adult subjects with surgically resected pancreatic cancer who have received adjuvant standard of care treatment |
| Immunovant | IMVT-1401 | moderate-to-severe active Graves' ophthalmopathy (GO) | Phase II trial initiated enrolling eight subjects |
| Rejenevie Therapeutics | AR-100 immune restoration therapy | immunology | Phase II trial initiated enrolling 20 healthy subjects 55 years of age or older in Freeport, Bahamas |
| Ironwood Pharmaceuticals, Inc. Allergan PLC |
MD-7246 | abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) | Phase II trial initiated enrolling 400 subjects |
| Verona Pharma PLC | metered-dose inhaler formulation of ensifentrine (RPL554) | moderate-to-severe chronic obstructive pulmonary disease (COPD) | Phase II trial initiated enrolling 36 subjects at two sites in the UK |
| Minoryx Therapeutics | leriglitazone (MIN-102) | Friedreich’s Ataxia | Phase II trial initiated enrolling 36 subjects 12 years of age or older at numerous sites across Europe |
| GENFIT | elafibranor | nonalcoholic fatty liver disease (NAFLD) | Phase II trial initiated enrolling 16 subjects with NAFL as identified with magnetic resonance spectroscopy (H-MRS) |
| Arcutis, Inc. | ARQ-151 | atopic dermatitis | Phase IIa trial initiated |
| Akero Therapeutics, Inc. | AKR-001 | non-alcoholic steatohepatitis (NASH) | Phase IIa trial initiated enrolling 80 subjects |
| Ansun Biopharma, Inc. | DAS181 | lower respiratory tract parainfluenza virus | Phase III trial initiated enrolling immunocompromised subjects at sites in the U.S., Europe and Asia |
| Eyenovia Inc. | MicroPine | pediatric progressive myopia | Phase III trial initiated enrolling 400 subjects ages three to 12 years of age |
| Incyte | pemigatinib (INCB54828) | cholangiocarcinoma | Phase III trial initiated enrolling 432 subjects |
| Allergan plc Gedeon Richter PLC |
VRAYLAR (cariprazine) | manic or mixed episodes associated with bipolar I disorder in adults | sNDA approval granted by the FDA |
| Cadent Therapeutics | CAD-1883 | spinocerebellar ataxia (SCA) | Orphan Drug designation granted by the FDA |
| Bayer | Aliqopa (copanlisib) | marginal zone lymphoma (MZL) | Breakthrough Therapy designation granted by the FDA |
| Imara, Inc. | IMR-687 | sickle cell disease | Fast Track designation granted by the FDA |
| NovoCure | NovoTTF-100L System in combination with pemetrexed plus platinum-based chemotherapy | malignant pleural mesothelioma (MPM) | Approval granted by the FDA |
| AveXis Inc. | Zolgensma (onasemnogene abeparvovec-xioi) | spinal muscular atrophy (SMA) in children less than two years of age | Approval granted by the FDA |
| Novartis | Piqray (alpelisib) tablets in combination with the endocrine therapy fulvestran | hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated advanced or metastatic breast cancer in postmenopausal women and men | Approval granted by the FDA |
| Novartis | Jakafi (ruxolitinib) | steroid-refractory acute GVHD in adult and pediatric subjects 12 years and older | Approval granted by the FDA |
| Celgene Corporation | REVLIMID (lenalidomide) in combination with a rituximab product (R²) | previously treated follicular lymphoma (FL) or marginal zone lymphoma (MZL) in adults | Approval granted by the FDA |
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