Company Product Description Indication Status
Phase I
Aileron Therapeutics Inc., of Watertown, Mass. ALRN-6924 MDM2/MDMX dual inhibitor Non-small-cell lung cancer Enrollment target for upcoming phase Ib trial expanded from 40 to 60 participants with advanced p53-mutated disease receiving first-line carboplatin + pemetrexed; enrollment expected to begin in second quarter of 2021
Avadel Pharmaceuticals plc, of Dublin FT-218 (sodium oxybate Micropump controlled-release) GABA B receptor agonist Narcolepsy Results from 4 studies, published in Clinical Therapeutics, showed pharmacokinetic profile supported once-nightly dosing
Clearside Biomedical Inc., of Alpharetta, Ga. CLS-AX (axitinib injectable suspension) Tyrosine kinase inhibitor; pan-VEGF receptor antagonist Wet age-related macular degeneration Dosing completed in first cohort of ongoing phase I/IIa Oasis trial; initial safety data from cohort expected in mid-2021
Gemoab Monoclonals GmbH, of Dresden, Germany UniCAR-T-CD123 CDw123 modulator; CDw137 agonist Acute myeloid leukemia Blood published interim data from ongoing study in people with relapsed/refractory disease showing therapy was well-tolerated, with rapid recovery of hematopoiesis and early signs of efficacy
Hoth Therapeutics Inc., of New York Biolexa Antimicrobial topical formulation Atopic dermatitis Cohort 1 recruitment completed; top-line data expected by year-end 2021
Kymera Therapeutics Inc., of Watertown, Mass. KT-474 IRAK4 protein degrader  Atopic dermatitis; hidradenitis suppurativa Dosing initiated in healthy adults and  patients
NGM Biopharmaceuticals Inc., of South San Francisco NGM-120 GDF15/GFRAL inhibitor Pancreatic cancer First participant dosed in expansion of ongoing phase Ib study in people with metastatic disease; trial examining effect on cancer and cancer-related cachexia
Pipeline Therapeutics Inc., of San Diego PIPE-307 Muscarinic M1 receptor antagonist Multiple sclerosis Study initiated in Australia with goal of enrolling up to 72 healthy volunteers; top-line data expected in second half of 2021
Phase II
Biomx Inc., of Ness Ziona, Israel BX-001 Phage targeting Cutibacterium acnes Acne First of 140 participants with mild to moderate disease dosed; efficacy endpoints include measurements of lesions, IGA score and reduction of C. acnes bacterial levels on skin; 8-week data expected in third quarter of 2021, with full analysis of 12-week time point in fourth quarter of 2021
Cerecor Inc., of Rockville, Md. CERC-002 TNFSF14 inhibitor COVID-19-related acute respiratory distress syndrome Final analysis showed trial met primary efficacy endpoint of proportion of people alive and free of respiratory failure over 28-day study period vs. placebo in people hospitalized with infection and ARDS (n=62, p=0.044); efficacy was highest in prespecified subpopulation > age 60 (n=34, p=0.042); at both 28- and 60-day time points, about 50% trend in mortality reduction (22.5% vs. 10.8%) observed
Highlight Therapeutics SL, of Madrid BO-112  IFIH1 gene modulator; retinoic acid-inducible gene 1 modulator Melanoma Working with CRO Pivotal, of Madrid, first of 40 participants recruited for phase IIa study of RNA-based therapy in combination with anti-PD-1 to treat unresectable/metastatic disease that progressed on checkpoint inhibitors
Neurocrine Biosciences Inc., of San Diego Luvadaxistat (NBI-1065844/TAK-831) D-amino acid oxidase inhibitor Schizophrenia Interact study that randomized 256 adults with negative symptoms of schizophrenia missed primary endpoint, measured as change from baseline on PANSS NSFS at day 84, but met secondary endpoints of cognitive assessment
Neurosense Therapeutics Ltd., of Herzliya, Israel PrimeC (ciprofloxacin + celecoxib) Cyclooxygenase 2 inhibitor; endoribonuclease DICER stimulator Amyotrophic lateral sclerosis Phase IIa study in 15 participants showed drug was safe and tolerable and suggested disease decline was slower than expected compared with historical controls
Seres Therapeutics Inc., of Cambridge, Mass. SER-287 Microbiome therapeutic (encapsulated purified suspension of eubacterial spores) Ulcerative colitis Phase IIb Eco-Reset study achieved target enrollment of 201 people with mild to moderate disease; top-line data expected in mid-2021
Phase III
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. VGX-3100 Dual HPV E6/HPV E7 protein modulator Cervical dysplasia Pivotal Reveal 1 trial met primary endpoint of histopathological regression of high-grade squamous intraepithelial lesions + virologic clearance of HPV-16 and/or HPV-18 at week 36 in mITT population (n=193); 23.7% (31/131) responded in treatment group vs. 11.3% (7/62) for placebo (p=0.022)
Orbus Therapeutics Inc., of Palo Alto, Calif. Eflornithine (repurposed) Ornithine decarboxylase inhibitor Anaplastic astrocytoma Independent data monitoring committee recommended Stellar study continue without modification following preplanned interim futility analysis; full enrollment expected in second half of 2021, with preplanned interim superiority analysis expected in 2022
Scynexis Inc., of Jersey City, N.J. Ibrexafungerp 1,3 beta glucan synthase inhibitor Fungal infection Interim analyses of ongoing Furi study showed benefit against difficult-to-treat, severe, mucocutaneous and invasive infections in 30 of 33 participants and no disease progression; first interim analysis of Cares study showed activity in people hospitalized with Candida auris, including complete response in 8/10 with invasive candidiasis and candidemia


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