Company Product Description Indication Status
Phase I
Altimmune Inc., of Gaithersburg, Md. Nasoshield Intranasal anthrax vaccine candidate Anthrax Phase Ib data in healthy subjects showed serum binding antibody responses to the protective antigen of Bacillus anthracis were significantly greater than in the placebo arm; antibody responses blocking anthrax toxin were blunted compared to protective antigen antibody responses; nasal mucosal IgA responses specific for protective antigen were observed in up to 80% of subjects post-vaccination
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 Orally dissolving thin film formulation of dexmedetomidine Opioid withdrawal symptoms Top-line data showed phase Ib/II Release study met its primary safety endpoint across multiple doses given twice-daily over 7 days, with dose-dependent exposures observed across all doses; patients in multiple dose cohorts treated with BXCL-501 had numerical improvements in retention rates, a key goal of opioid withdrawal treatment
Celon Pharma SA, of Kielpin, Poland CPL-280 GPR40 agonist Diabetic neuropathy Met primary endpoint in healthy volunteer study, with no adverse safety signals detected
Celsion Corp., of Lawrenceville, N.J. GEN-1 DNA-mediated IL-12 immunotherapy Advanced ovarian cancer Data from phase I/II Ovation 2 study showed 14 of 17 (82%) patients treated with GEN-1 had a R0 resection, which indicates a microscopically margin-negative complete resection in which no gross or microscopic tumor remains in the tumor bed; 7 of 12 (58%) in the control arm had an R0 resection; interim data represents 41% improvement in R0 resection rates for GEN-1 patients
Onconova Therapeutics Inc., of Newtown, Pa., and Hanx Biopharmaceuticals Inc., of Wuhan, China ON-123300 Multikinase inhibitor HR+ HER2- metastatic breast cancer and other advanced relapsed/refractory cancers Hanx enrolled 3 patients in the second dosing cohort in China
Passage Bio Inc., of Philadelphia PBGM-01 AAV gene therapy Infantile GM1 gangliosidosis Dosed the first patient in a phase I/II Imagine-1 trial
Springworks Therapeutics Inc., of Stamford, Conn. Nirogacestat Gamma-secretase inhibitor Relapsed or refractory multiple myeloma First patient dosed in phase Ib trial testing combination with teclistamab (Janssen Research & Development LLC), a bispecific antibody targeting BCMA and CD3
Synthetic Biologics Inc., of Rockville, Md. SYN-020 Recombinant bovine intestinal alkaline phosphatase Gastrointestinal diseases Started enrollment and dosed 6 healthy participants in phase Ia single ascending-dose trial
Phase II
Acceleron Pharma Inc., of Cambridge, Mass. Sotatercept TGF-beta Pulmonary arterial hypertension Data published in The New England Journal of Medicine from Pulsar trial showed patients on stable background PAH-specific therapies treated with sotatercept experienced a statistically significant reduction in pulmonary vascular resistance, the trial’s primary endpoint, at week 24 vs. placebo; study also achieved an all-dose mean improvement from baseline in the key secondary endpoint of 6-minute walk distance
Azurrx Biopharma Inc., of Delray Beach, Fla. MS-1819 Recombinant lipase enzyme Cystic fibrosis with exocrine pancreatic insufficiency Top-line data from phase IIb Option 2 trial testing drug against current porcine enzyme replacement therapy showed study did not meet primary endpoint, defined as coefficient of fat absorption
Leap Therapeutics Inc., of Cambridge, Mass. DKN-01 Monoclonal antibody targeting Dickkopf-1 Gastric or gastroesophageal junction cancer Completed enrollment in the phase IIa Distinguish study testing DKN-01 with tislelizumab (Beigene Ltd.); initial data expected in the second half of 2021
Newron Pharmaceuticals SpA, of Milan Evenamide Targets voltage-gated sodium channels Schizophrenia In study 010, evenamide didn’t produce any QTcF prolongation compared to placebo in healthy volunteers, while moxifloxacin produced a 17.3 ms median maximum increase; in study 008, no symptoms suggestive of severe CNS events, symptoms/signs of seizures, EEG diagnosis of seizure like activity or cardiac events were observed in 138 patients with schizophrenia taking a second-generation atypical antipsychotic and evenamide; there were no differences in laboratory, ECG or vital signs abnormalities between evenamide and placebo-treated patient
Shanghai Yingli Pharmaceutical Co. Ltd., of Shanghai Linperlisib PI3Kdelta inhibitor Relapsed/refractory follicular lymphoma Overall response rate was 80.9% for 89 evaluable patients; disease control rate was 96.6%
Synaptogenix Inc., of New York Bryostatin-1 Protein kinase C agonist  Moderately severe Alzheimer's disease Treated 43 patients of the target of 100 patients; 17 sites enrolling patients in the phase IIb study
Phase III
AB Science SA, of Paris Masitinib  Tyrosine kinase inhibitor Metastatic castrate-resistant prostate cancer  Results from study AB12003 are expected in April 2021
Abbvie Inc., of North Chicago Rinvoq (upadacitinib) JAK inhibitor Active psoriatic arthritis after 1 or more non-biologic disease modifying anti-rheumatic drugs Data published in The New England Journal of Medicine showed 54% and 58% of patients taking 15-mg and 30-mg doses, respectively, achieved Leeds Enthesitis Index of 0 at week 24, compared to 32% of patients taking placebo (p<0.001 for both doses) and 47% of patients taking Humira (adalimumab)
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Lumasiran RNAi targeting hydroxyacid oxidase 1 Primary hyperoxaluria type 1 Data from the Illuminate-A study published in The New England Journal of Medicine showed lumasiran reduced 24-hour urinary oxalate excretion from baseline to month 6 by 53.5% relative to placebo; 84% of patients taking lumasiran achieved normal or near-normal levels of urinary oxalate compared to 0% for placebo
Kedrion Biopharma SpA, of Fort Lee, N.J. 10% intravenous  immunoglobulin  Antibodies Primary immunodeficiency disease Enrolled first patient in the Kidscares10 study; primary efficacy objective is to show the drug produces a rate of acute serious bacterial infections of less than 1
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162b2 RNAi-based vaccine COVID-19 prophylaxis Updated results from the study showed the vaccine was 91.3% effective from 7 days to 6 months after the second dose; 100% effective against severe COVID-19 as defined by the CDC and 95.3% effective against severe COVID-19 as defined by the FDA; 100% effective against the South African variant
Relmada Therapeutics Inc., of New York REL-1017 NMDA receptor channel blocker Major depressive disorder Started the 364-patient Reliance II study; primary endpoint is the change from baseline on the Montgomery and Asberg Depression Rating Scale (MADRS) score at day 28; MADRS at day 7 is a secondary endpoint; data expected in the first half of 2022
Revance Therapeutics Inc., of Nashville, Tenn. DaxibotulinumtoxinA Acetylcholine receptor antagonist; botulinum toxin A stimulator  Cervical dystonia In the Aspen-1 study, mean improvement from baseline in Toronto Western Spasmodic Torticollis Rating Scale total score was 12.7 for 125U dose, 10.9 for 250U dose and 4.3 for placebo (p<0.0001 and p=0.0006 for 125U and 250U doses, respectively)


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