Company Product Description Indication Status
Aldeyra Therapeutics Inc., of Lexington, Mass. ADX-629 Oral RASP inhibitor COVID-19 Received a study may proceed letter from the FDA for a phase II study in hospitalized adult patients
Apros Therapeutics Inc., of San Diego APR-003 Oral gastrointestinal- and liver-targeted TLR7 agonist Colorectal cancer FDA cleared IND for a phase I dose-escalation study in patients with advanced unresectable CRC with malignant liver lesions
Ascendis Pharma A/S, of Copenhagen, Denmark Transcon hGH (lonapegsomatropin) Long-acting, once-weekly prodrug of somatropin (human growth hormone) Growth hormone deficiency Submitted MAA to EMA seeking approval for treatment of pediatric patients diagnosed with GHD
Beigene Ltd., of Cambridge, Mass., and Beijing Brukinsa (zanubrutinib) BTK inhibitor  Waldenström's macroglobulinemia New drug submission accepted by Health Canada and granted priority review status
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Roctavian (valoctocogene roxaparvovec) Gene therapy Severe hemophilia A Company disclosed in 8-K filing that it received EMA’s joint assessment report related to ongoing review of MAA; EMA requests company to submit full 52-week results from 134 patients in ongoing phase III study with the 6e13 vg/kg dose; last patient expected to reach 52 weeks in November 2020, and Biomarin is working with EMA to enable potential submission of requested data by end of first quarter of 2021
Canbridge Pharmaceuticals Inc., of Beijing and Cambridge, Mass. Hunterase (idursulfase beta injection) Enzyme replacement therapy Mucopolysaccharidosis II (Hunter syndrome) Approved by China’s NMPA for long-term treatment
Moleculin Biotech Inc., of Houston Annamycin Anthracycline Soft tissue sarcomas  Completed pre-IND meeting with FDA regarding development plan, including study design and dosing strategy for initial phase Ib/II protocol for STS with lung metastases
Nanomab Technology Ltd., of London NM-01 Imaging agent; PD-L1 modulator Cancer Submitted request to U.K.’s MHRA to start phase II study assessing PD-L1 expression using 99mTc-NM-01 as a SPECT/CT radiotracer and its correlation to PD-L1 expression results based on routine immunohistochemical testing in biopsy diagnostic specimens
Peptilogics Inc., of Pittsburgh PLG-0206 Broad-spectrum antibiotic peptide Prosthetic joint infections FDA granted orphan status
Precision Biosciences Inc., of Durham, N.C. PBCAR-269A CAR T-cell therapy; APRIL receptor modulator Relapsed/refractory multiple myeloma FDA granted fast track designation
Reneo Pharmaceuticals Inc., of San Diego REN-001 Selective PPAR delta agonist LCHAD deficiency and MELAS syndrome EMA’s Committee for Orphan Medicinal Products granted orphan designation
Sellas Life Sciences Group Inc., of New York Galinpepimut-S Targets WT1 protein Acute myeloid leukemia France’s regulatory authority cleared the start of the pivotal phase III Regal study in patients with AML who have achieved complete remission after second-line anti-leukemic therapy


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