Company Product Description Indication Status
Acasti Pharma Inc., of Laval, Quebec Capre Krill oil-derived omega-3 phospholipid Hypertriglyceridemia Trilogy 1 briefing package submitted to FDA to gain alignment on data interpretation, proposed revisions to prespecified statistical analysis plan for still-blinded Trilogy 2 and plan for pooling data from both studies to support NDA filing; formal response expected by June 30, 2020
Akarx Inc., unit of Dova Pharmaceuticals Inc., of Durham, N.C., a Swedish Orphan Biovitrum AB company Doptelet (avatrombopag)  Thrombopoietin receptor agonist Thrombocytopenia China NMPA approved drug to treat adults with chronic liver disease scheduled to undergo a procedure
Alpha Cognition Inc., of Vancouver, British Columbia ALPHA-1062 Acetylcholinesterase inhibitor; nicotinic acetylcholine receptor modulator Alzheimer's disease At pre-IND meeting, FDA suggested that single pivotal trial, scheduled to begin in mid-2021, may be sufficient to obtain marketing approval
Annovis Bio Inc., of Berwyn, Pa. ANVS-401 Acetylcholinesterase/beta-amyloid synthesis/huntingtin/synuclein alpha/tau protein inhibitor Alzheimer's disease; Parkinson's disease Phase II trial expected to enroll 68 individuals across both indications set to begin recruiting in May 2020 and treating participants in June 2020 after FDA accepted protocol without comments
Banner Life Sciences LLC, of High Point, N.C. Bafiertam (monomethyl fumarate)  Nuclear erythroid 2-related factor 2 stimulator Relapsing multiple sclerosis Delayed-release drug, bioequivalent of Tecfidera (dimethyl fumarate, Biogen Inc.), granted final approval by FDA through 505(b)(2) pathway
Biocardia Inc., of San Carlos, Calif. Cardiamp cell therapy Autologous bone marrow-derived stem cells Cardiac failure FDA-recommended modifications adopted for primary endpoint of Cardiamp-HF trial, revised to outcomes composite score based on 3-tiered Finkelstein-Schoenfeld hierarchical analysis, along with associated statistical analysis plan; tiers include all-cause death, non-fatal major adverse coronary and cerebrovascular events and change from baseline in 6-minute walk distance at 12 months
Galectin Therapeutics Inc., of Norcross, Ga. Belapectin (GR-MD-02) Galectin-3 inhibitor Nonalcoholic steatohepatitis cirrhosis Protocol submitted to FDA for seamless adaptive design phase IIb/III NASH-RX trial assessing prevention of esophageal varices in individuals with disease; trials expected to begin in second quarter of 2020
Genmab A/S, of Copenhagen Daratumumab CD38-targeting antibody Multiple myeloma EMA’s Committee for Medicinal Products for Human Use recommended use of subcutaneous formulation for treating adults in front-line and relapsed/refractory settings
Glaxosmithkline plc, of London Zejula (niraparib) PARP 1/2 inhibitor Advanced ovarian cancer FDA approved sNDA for first-line monotherapy maintenance of individuals with platinum-responsive disease regardless of biomarker status
Inmed Pharmaceuticals Inc., of Vancouver, British Columbia INM-755 (cannabinol) Keratin modulator Epidermolysis bullosa Dutch regulatory and ethics board approved CTA for second phase I trial in healthy volunteers, expected to begin once COVID-19 pandemic-related restrictions are lifted sometime after May 20, 2020
Junshi Biosciences Co. Ltd., of Shanghai Toripalimab PD-1-targeting monoclonal antibody Recurrent/metastatic nasopharyngeal carcinoma China’s NMPA accepted a supplemental NDA seeking approval for use in patients who failed at least 2 lines of systemic therapy
Marker Therapeutics Inc., of Houston MT-401 Multitumor-associated antigen-specific T-cell therapy Acute myeloid leukemia FDA granted orphan drug designation to treat individuals with disease following allogeneic stem cell transplant
Medivir AB, of Stockholm MIV-818 (liver-targeted troxacitabine prodrug) DNA polymerase inhibitor Hepatocellular carcinoma EU's Committee for Orphan Medicinal Products issued positive opinion on orphan medicinal product designation
Neon Therapeutics Inc., of Cambridge, Mass. NEO-PTC-01 Neoantigen-stimulated autologous T-cell therapy Metastatic melanoma Dutch Health Authority accepted CTA for phase I dose-finding trial in individuals with disease that did not respond to checkpoint inhibitors; study expected to begin in third quarter of 2020
Pharming Group NV, of Leiden, the Netherlands Ruconest (conestat alfa) Recombinant C1 esterase inhibitor Hereditary angioedema  European Commission approved drug to treat acute disease attacks in children 2 and older 
Seelos Therapeutics Inc., of New York SLS-005 Trehalose Sanfilippo syndrome FDA granted orphan designation
Zentalis Pharmaceuticals Inc., of New York ZN-d5 BCL-2 inhibitor Hematologic malignancies FDA cleared IND application

Notes

For more information about individual companies and/or products, see Cortellis.

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