Company Product Description Indication Status
Phase I
Axcella Health Inc., of Cambridge, Mass. AXA-1665 Composition of naturally occurring amino acids Child-Pugh A and B cirrhosis Results from AXA1665-001 safety, tolerability and biomarker crossover study that showed potential to mitigate hyperammonemia, dysregulated amino acid metabolism and physical dysfunction published in Clinical and Translational Gastroenterology
Betagenon/Balticgruppen Bio AB, of Stockholm O-304 AMPK activator Non-insulin-dependent diabetes Pharmacokinetic and safety study, in combination with dapagliflozin (Forxiga, Astrazeneca plc), initiated in healthy volunteers 
Enterprise Therapeutics Ltd., of Brighton, U.K. ETD-002 TMEM16A potentiator Cystic fibrosis First healthy volunteers dosed
I-Mab Biopharma Co. Ltd., of Shanghai Plonmarlimab (TJM-2) GM-CSF ligand inhibitor Rheumatoid arthritis First of 63 participants dosed in phase Ib study in China
Merck & Co. Inc., of Kenilworth, N.J. (known as MSD outside the U.S.) MK-5475 Antiviral COVID-19 Study to test drug in patients with pneumonia and hypoxemia withdrawn for business reasons
Precigen Inc., of Germantown, Md. PRGN-2009 Adenovirus vector-based vaccine Solid tumors First participant dosed in phase I/II study in HPV-associated disease; phase I portion will use 3+3 dose escalation to evaluate safety as monotherapy and recommended phase II dose (R2PD) followed by safety assessment at R2PD with bifunctional fusion protein bintrafusp alfa (M-7824, Glaxosmithkline plc/Merck KGaA) in recurrent or metastatic cancer
Phase II
Amplyx Pharmaceuticals Inc., of San Diego MAU-868 Capsid protein VP1 inhibitor BK virus viremia First of 36 expected participants dosed; efficacy endpoint is reduction of BKV plasma viral load
Ansun Biopharma Inc., of San Diego DAS-181 Exo-alpha sialidase modulator COVID-19 Phase II/III study withdrawn in regulatory stage; no COVID-19 in EU countries
Ascletis Pharma Inc., of Hangzhou, China ASC-22 (envafolimab) PD-L1 inhibitor Hepatitis B virus infection First participant dosed in single-dose-escalation (0.3, 1 and 2.5 mg/kg) phase IIa study in chronic disease
Azurrx Biopharma Inc., of New York MS-1819 Lipase modulator Exocrine pancreatic insufficiency European sites initiated in phase IIb Option 2 trial in people with cystic fibrosis
Clovis Oncology Inc., of Boulder, Colo. Rubraca (rucaparib)  PARP1/2/3 inhibitor Prostate cancer Additional data from Triton2 study in metastatic castration-resistant disease harboring BRCA1/2 mutations, published online in Journal of Clinical Oncology, confirmed objective response rates of 43.5% and 50.8%, respectively, in 115 people with BRCA alteration with or without measurable disease; confirmed PSA response rate was 54.8% 
GNI Group Ltd., of Tokyo F-351 (hydronidone) Aminotransferase stimulator; TGF beta receptor antagonist Liver fibrosis Study in 168 people with HBV-related disease met primary endpoint of statistically significant improvement in liver fibrosis score over 52-week treatment vs. placebo (p=0.025); 270-mg/day group showed best Ishak score improvement
Neuroptika Inc., of Cambridge, Mass. NRO-1 GDNF receptor agonist Dry eye disease Enrollment completed; top-line data expected in fourth quarter of 2020
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373 COVID-19 Spike glycoprotein modulator COVID-19 infection Phase IIb trial initiated in South Africa; 1 cohort will evaluate candidate in approximately 2,665 healthy adults, with second cohort assessing safety and immunogenicity in about 240 HIV-positive adults
Syndax Pharmaceuticals Inc., of Waltham, Mass. Axatilimab (SNDX-6352) Anti-CSF-1R monoclonal antibody COVID-19 Study in hospitalized patients with respiratory involvement secondary to COVID-19 suspended, given enrollment challenges partly attributable to constantly changing COVID-19 treatment landscape
Unity Biotechnology Inc., of South San Francisco UBX-0101 p53/MDM2 interaction inhibitor Osteoarthritis Study in 183 people with moderate to severe pain missed primary endpoint, showing no statistically significant difference between any arm (0.5, 2 or 4 mg) of study drug vs. placebo at 12 weeks on change from baseline in WOMAC-A; company halting development in indication
Phase III
Alkermes plc, of Dublin ALKS-3831 Samidorphan plus olanzapine Stable schizophrenia Data from the 561-patient Enlighten-2 study published in American Journal of Psychiatry showed least squares mean percent weight change from baseline was 4.21% for ALKS-3831 and 6.59% for olanzapine; 17.8% and 29.8% of patients taking ALKS-3831 and olanzapine, respectively, gained 10% or more of body weight
Chiasma Inc., of Needham, Mass. Mycapssa Delayed-release somatostatin analogue Acromegaly  Data from the 56-patient Chiasma Optimal study published in the Journal of Clinical Endocrinology & Metabolism showed IGF-1 levels were within 97% of the upper limit of normal (ULN) for all patients receiving Mycapssa at the end of the treatment compared to 1.69 times the ULN for patients receiving placebo; growth hormone levels were < 2.5 ng/mL in 77.7% of patients taking drug compared to 30.4% of the placebo group (p=0.0007)

Notes

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

No Comments