Company Product Description Indication Status
Phase I
Abpro Corp., of Woburn, Mass. ABP-300 Human neutralizing antibody derived from patients who recovered from SARS-CoV-2 infection COVID-19 Results show favorable safety and pharmacokinetic profile in 42 healthy subjects, at doses from 4 mg/kg to 60 mg/kg; phase II/III program ongoing in patients with mild to moderate COVID-19
Biomx Inc., of Ness Ziona, Israel BX-002 Oral phage therapy targeting Klebsiella pneumoniae bacteria Inflammatory bowel disease and primary sclerosing cholangitis Phase Ia data showed drug safety and well-tolerated, with no serious adverse events; met objective of delivering high concentrations of viable phage to GI tract of about 1010 PFU (plaque forming units)
Bridgebio Pharma Inc., of Palo Alto, Calif., and affiliate Venthera BBP-681 (VT30 topical gel) Delivers PI3Kα inhibitor directly to affected tissue Venous, lymphatic and venolymphatic malformations Dosed first subject in phase I/II trial
Daiichi Sankyo Co. Ltd., of Tokyo, and Sarah Cannon Research Institute DS-6000 CDH6-directed antibody-drug conjugate Advanced renal cell carcinoma or ovarian cancer Dosed first patient with disease progression
Exonate Ltd., of Cambridge, U.K., and Janssen Pharmaceuticals Inc., part of New Brunswick, N.J.-based Johnson & Johnson EXN-407 Inhibits serine/arginine-protein kinase 1 Diabetic macular edema Dosed first patient in phase Ib/II trial; study aims to demonstrate safety and tolerability and an exploratory endpoint of efficacy through reduction in retinal thickness; top-line results expected in early 2022
Gedea Biotech AB, of Lund, Sweden pHyph vaginal tablet Restores pH to natural level Bacterial vaginosis Enrolled first patient in Nefertiti study
Kira Pharmaceuticals Inc., of Cambridge, Mass., and Suzhou, China P014 Bifunctional compound designed to block 2 separate rate-limiting steps in complement activation cascade  Complement-mediated diseases Dosed first healthy volunteer
Pinteon Therapeutics Inc., of Cambridge, Mass. PNT-001 Tau antibody Traumatic brain injury and other tauopathies Results showed drug was well-tolerated in healthy volunteers at doses that lead to potentially therapeutic drug concentration in cerebrospinal fluid
Selva Therapeutics Inc., of San Diego SLV-213 Small-molecule inhibitor of cathepsin L COVID-19 Met primary objective of demonstrating safety and tolerability; plans to advance into phase II study in non-hospitalized patients
Phase II
Arch Biopartners Inc., of Toronto Metablok LSALT peptide Acute lung injury and acute kidney injury caused by inflammation in severe COVID-19 Received approval in Turkey from ethics committee to dose additional 20 patients 
Calcimedica Inc., of La Jolla, Calif. Auxora (CM4620-IE) Small-molecule inhibitor of Orai1-containing CRAC channels Severe COVID-19 pneumonia New data showed levels of D-dimer, a key marker of clotting and inflammation, decreased in patients receiving Auxora plus standard of care (SOC) but increased in patients receiving SOC alone; 2 patients who received SOC alone developed femoral deep vein thrombosis, with 1 progressing to pulmonary embolism, while 0 patients who received Auxora developed thromboembolic disease
Cassava Sciences Inc., of Austin, Texas Simufilam Small molecule designed to restore normal shape and function of altered filamin A  Alzheimer’s disease Interim analysis showed 6-month treatment improved cognition scores by 1.6 points on ADAS-Cog11, a 10% mean improvement from baseline; also improved dementia-related behavior, such as anxiety, delusions and agitation, by 1.3 points on the Neuropsychiatric Inventory, a 29% mean improvement from baseline 
Chemomab Ltd., of Tel Aviv, Israel CM-101 CCL24 blocking monoclonal antibody Primary sclerosing cholangitis Enrolled first patient in phase IIa trial
Curis Inc., of Lexington, Mass. CA-4948 Small-molecule IRAK4 kinase inhibitor Anemia in patients with very low-, low- or intermediate-risk myelodysplastic syndromes Initiated investigator-sponsored Lucas trial; primary endpoint is to evaluate proportion of patients that develop an erythroid response according to IWG 2018 criteria
Gemini Therapeutics Inc., of Cambridge, Mass. GEM-103 Recombinant native complement modulator, full-length recombinant complement factor H protein Wet age-related macular degeneration Started phase IIa study testing drug as add-on therapy in patients who have or may be at risk for macular atrophy but require ongoing anti-VEGF treatment; top-line data expected in the second half of 2021
Immunovant Inc., of New York IMVT-1401 Fully human anti-FcRn monoclonal antibody Thyroid eye disease and warm autoimmune hemolytic anemia Paused dosing in ongoing trials after physiological signal consisting of elevated total cholesterol and LDL levels in treated patients emerged in Ascend Go-2 phase IIb study in TED
Secura Bio Inc., of Las Vegas Farydak (panobinostat) Oral pan-HDAC inhibitor Relapsed or relapsed/refractory multiple myeloma Data from Panorama-3 trial confirm efficacy of approved dose/schedule and demonstrate considerably improved safety profile for combination with subcutaneous bortezomib and oral dexamethasone; dosing demonstrated overall response rate of 62.2% vs. 60.7% for Panorama-1 study; median duration of response was 22 months vs. 13.1 months in Panorama-1
Verona Pharma plc, of London Pressurized metered-dose inhaler formulation of ensifentrine CFTR stimulator; PDE4 inhibitor; PDE3 inhibitor Moderate to severe chronic obstructive pulmonary disease Met all primary and secondary lung function endpoints in 7-day; magnitude of improvement in lung function was dose-ordered and highly statistically significant at peak and over 12-hour dosing interval vs. placebo, and supports twice-daily dosing 
Phase III
Arena Pharmaceuticals Inc., of San Diego Etrasimod Once-daily, oral S1P modulator Moderately to severely active ulcerative colitis Completed enrollment in Elevate UC 52 study, with 433 participants; top-line data expected in the first quarter of 2022
Bristol Myers Squibb Co., of New York Deucravacitinib Oral, selective TYK2 inhibitor Moderate to severe plaque psoriasis Results showed Poetyk PSO-2 trial met both co-primary endpoints vs. placebo, with significantly more patients achieving Psoriasis Area and Severity Index and a static Physician's Global Assessment score of clear or almost clear after 16 weeks of treatment; secondary endpoints also met, including superiority to Otezla (apremilast)
Diurnal Group plc, of Cardiff, U.K. Chronocort (hydrocortisone modified-release hard capsules) Corticosteroid agonist Congenital adrenal hyperplasia Results published in the Journal of Clinical Endocrinology and Metabolism found that although the standard-deviation-score-focused primary endpoint was missed, Chronocort improved morning and early afternoon biochemical control over standard glucocorticoid therapy; in the safety extension study, biochemical control was sustained for 18 months on median hydrocortisone doses in the range recommended for cortisol replacement therapy and lower than glucocorticoid doses normally used in treatment of CAH
Pharnext SA, of Paris PXT-3003 Fixed-dose combination of baclofen, naltrexone and sorbitol formulated as oral solution  Charcot-Marie-Tooth type 1A On track to begin first patient/first dose in pivotal Premier study before March 31, 2021
The Russian Direct Investment Fund, of Moscow Sputnik V Vaccine COVID-19 Data published in The Lancet showed efficacy of 91.6%; among cases analyzed, more than 98% of volunteers developed humoral immune response and 100% had cellular immune response; level of neutralizing antibodies was 1.3-1.5 times higher than in patients who recovered from COVID-19
Tiziana Life Sciences plc, of New York and London Foralumab Anti-CD3 monoclonal antibody COVID-19 Data from exploratory study in Brazil testing drug alone or in combination with dexamethasone showed 80% lung CT scan improvement in foralumab-only group and 75% in combination group vs. 43% for control; cytokine IL-6 reduction was 69%, 41% and 37%, respectively, while C-reactive protein reduction as 85%, 55% and 40%, respectively
Travere Therapeutics Inc., of San Diego Sparsentan Dual-acting antagonist of endothelin type A and angiotensin II type 1 receptors Focal segmental glomerulosclerosis Achieved prespecified interim FSGS partial remission of proteinuria endpoint after 36 weeks of treatment; drug showed statistically significant response on FPRE compared to the active control, irbesartan (p=0.009); based on data, company intends to pursue submissions for accelerated approval

Notes

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