Company Product Description Indication Status
Phase I
Black Diamond Therapeutics Inc., of Cambridge, Mass. BDTX-189 Erbb2 tyrosine kinase receptor inhibitor; epidermal growth factor receptor antagonist Advanced solid tumors  Company disclosed in 8-K filing that, following a discussion with FDA to discuss registrational potential of phase II portion of phase I/II trial, agency issued partial clinical hold on phase II enrollment; trial may only enroll up to 50 patients before results of routine 3-month GLP toxicology studies have been submitted and accepted; partial hold is not based on any safety findings and has no impact on completion of phase I study, including planned safety expansion cohort
Corcept Therapeutics Inc., of Menlo Park, Calif. Relacorilant Nonsteroidal, selective modulator of the glucocorticoid receptor Adrenal cancer with cortisol excess  Enrolled first patient in phase Ib trial testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in unresectable or metastatic disease
Curasen Therapeutics Inc., of San Carlos, Calif. Salbutamol Beta 2 adrenoceptor agonist  Neurodegenerative disease Study in 12 healthy subjects showed statistically significant increase in cerebral blood flow in the thalamic region of the brain at the highest dose of 1.8 mg, from 40.0±6 to 50.6±9.4 ml/100g/min, representing an increase of 27.4% (p=0.0081); dose-dependent increases in heart rate observed with salbutamol were eliminated by pre-treatment with nadolol (10-40 mg), along with side effects such as hyperglycemia, hypokalemia and tremor
Equillium Inc., of La Jolla, Calif. Itolizumab Anti-CD6 monoclonal antibody Acute graft-vs.-host disease Data confirm that a majority of patients in Equate study achieved a complete response within 15 days, which was maintained through day 85; there was a clinically meaningful reduction in corticosteroid use in those patients
Immunitybio Inc., of Culver City, Calif. hAd5 T-cell COVID-19 vaccine candidate Human adenovirus-based T-cell COVID-19 vaccine COVID-19 Met safety requirements for first 12 participants in phase Ib trials testing sublingual and oral formulations; independent safety review committee recommended study continue with no modifications to trial design
Magenta Therapeutics Inc., of Cambridge, Mass. MGTA-145 CD117-targeted antibody conjugated to amanitin Stem cell mobilization Data from healthy volunteer study show combination with plerixafor is an effective, single-day mobilization and collection regimen for autologous and allogeneic hematopoietic stem cell (HCSs) transplant; combination rapidly mobilized large numbers of HSCs and showed durable engraftment, successful gene-modification and immunosuppressive properties by reducing GVHD in preclinical models
Moderna Inc., of Cambridge, Mass. mRNA-1283 Next-generation, refrigerator-stable mRNA vaccine COVID-19 Dosed first participants to assess 3 dose levels, administered to healthy adults as a 2-dose series, 28 days apart, and 1 dose level given to healthy adults in a single dose
Rubius Therapeutics Inc., of Cambridge, Mass. RTX-240 Dual 4-1BBL/IL-15TP expressing red blood cell therapy Advanced solid tumors Ongoing phase I/II trial with 15 participants evaluable for efficacy showed 1 confirmed partial response (PR) with 54% reduction in target lesions in metastatic anal cancer who progressed on anti-PD-L1 therapy, 1 unconfirmed PR with complete resolution of target hepatic lesion and resolution of 14/15 hepatic lesions in metastatic uveal melanoma who progressed on anti-PD-1, and 6 with stable disease
Sernova Corp., of London, Ontario Cell Pouch Cell therapy Type 1 diabetes Pre-screening of final 2 participants underway to complete phase I/II enrollment of 7 people
Tilt Biotherapeutics Ltd., of Helsinki, Finland TILT-123 Dual TNF-alpha/IL-2 cytokine armed oncolytic adenovirus Metastatic melanoma Tunintil trial achieved primary safety endpoint in first dose cohort and is proceeding to second of 5 dose levels, with interim data expected by year-end 2021
Phase II
Aclaris Therapeutics Inc., of Wayne, Pa. ATI-1777 Dual JAK1/JAK3 inhibitor Atopic dermatitis Phase IIa trial fully enrolled; data expected by mid-2021
Atara Biotherapeutics Inc., of South San Francisco Tabelecleucel (Tab-cel) Allogeneic Epstein-Barr virus T-cell therapy Post-transplant lymphoproliferative disease Combined long-term analysis from 3 studies showed people with EBV+ PTLD after HCT that was relapsed/refractory to rituximab and who achieved either complete (CR) or partial response (PR) after Tab-cel derived similar 1-year overall survival (OS) rates of 86.7% and 85.7%, respectively, and 2-year OS rates of 81.6% and 85.7%, respectively; objective response rate (PR+CR) was 62% (31/50), with best overall response of CR (n=24) or PR (n=7)
Atyr Pharma Inc., of San Diego ATYR-1923 Neuropilin 2 modulator COVID-19 infection In 32 people hospitalized with infection and severe respiratory complications who did not require mechanical ventilation, study drug showed trend of overall improvement in key biomarkers vs. placebo, including greater reduction in levels of interferon gamma, interleukin-6 and monocyte chemoattractant protein 1
Bioaegis Therapeutics Inc., of Morristown, N.J. Gelsolin Recombinant human plasma gelsolin Severe COVID-19 pneumonia Completed enrollment in trial involving hospitalized patients
Bioarctic AB, of Stockholm, and Eisai Co. Ltd., of Tokyo Lecanemab (BAN-2401) Beta-amyloid antagonist Alzheimer's disease Amyloid imaging substudies in primary phase IIb and ongoing open-label extension studies showed effects on reducing amyloid in the brain persisted for at least 2 years after dosing discontinued
Bluebird Bio Inc., of Cambridge, Mass. Elivaldogene autotemcel (Lenti-D) ABCD1 gene stimulator Adrenoleukodystrophy At 24 months of follow-up from phase II/III Starbeam study, 90% of participants (27/30) remained alive and free of major functional disabilities
Edesa Biotech Inc., of Toronto EB-05 TLR4 antagonist COVID-19 infection >50% of expected participants enrolled in phase II portion of ongoing phase II/III study of single-dose treatment for people hospitalized with infection with or at risk of developing acute respiratory distress syndrome 
Galecto Inc., of Boston GB-0139 Galectin-3 inhibitor Idiopathic pulmonary fibrosis Following data safety monitoring board interim review of phase IIb Galactic-1 study, enrollment discontinued in 10-mg monotherapy arm and 3-mg combination arm with nintedanib and pirfenidone; 3-mg monotherapy and placebo arms will continue to enroll
Eli Lilly and Co., of Indianapolis Donanemab Beta-amyloid antagonist Alzheimer's disease Trailblazer-Alz met primary endpoint, showing study drug slowed decline by statistically significant 32% vs. placebo at 76 weeks compared to baseline, as measured by integrated Alzheimer's Disease Rating Scale, in people with early symptomatic disease; data published simultaneously in The New England Journal of Medicine 
Ose Immunotherapeutics SA, of Nantes, France, and French cooperative group ARCAGY-GINECO Tedopi (OSE-2101) Neoepitope-based vaccine  Ovarian cancer Trial initiated in people with recurrent disease to evaluate therapy alone and in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) for maintenance treatment after chemotherapy
Quantum Leap Healthcare Collaborative, of San Francisco Otezla (apremilast) PDE4 inhibitor COVID-19 infection Drug from Amgen Inc. dropped from I-SPY COVID trial due to futility
Timber Pharmaceuticals Inc., of Woodcliff Lake, N.J. TMB-001 (topical isotretinoin) Vitamin A derivative Moderate to severe congenital ichthyosis Phase IIb Control study has randomized 50% of the expected participants
Zenith Epigenetics Corp., of Calgary, Alberta ZEN-3694 BET inhibitor Metastatic castration-resistant prostate cancer resistant to a first-line androgen receptor signaling inhibitor First patient treated in the investigator-led study testing ZEN-3694 plus Keytruda (pembrolizumab, Merck & Co. Inc.) and Xtandi (enzalutamide, Pfizer Inc.)
Phase III
Bluebird Bio Inc., of Cambridge, Mass. Elivaldogene autotemcel (Lenti-D) ABCD1 gene stimulator Adrenoleukodystrophy With 19 participants evaluable for safety in ALD-104 study, 17 achieved neutrophil engraftment and 15 had platelet engraftment, with no events of acute or chronic GVHD and no reports of graft failure or rejection, insertional oncogenesis or replication competent lentivirus
Gamida Cell Ltd., of Boston Omidubicel  Cell therapy High-risk hematologic malignancies undergoing a bone marrow transplant Median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for standard umbilical cord blood transplant (p < 0.001); all 3 secondary endpoints demonstrated a statistically significant improvement for omidubicel compared to umbilical cord blood transplant 
Molecular Partners AG, of Zurich-Schlieren, Switzerland, and Novartis AG, of Basel Ensovibep (MP-0420) ACE-2 inhibitor; COVID19 spike glycoprotein inhibitor COVID-19 infection Study drug to be included in NIH-sponsored Activ-3 trial, with ensovibep arm initially to enroll 300 adults hospitalized with mild to moderate infection; primary efficacy endpoint is time from randomization to sustained recovery for 14 days following discharge
Novartis AG, of Basel Zolgensma (onasemnogene abeparvovec) Gene therapy expressing SMN Spinal muscular atrophy In the SPR1NT study of presymptomatic patients with 2 or 3 copies of SMN2, 11 of 14 patients with 2 copies were able to sit without support for at least 30 seconds; 8 of 15 patients with 3 copies could stand alone for at least 3 seconds; in the long-term follow-up studies, 91% of the 23 patients in the I.V. cohort and 50% of the 8 patients in the IT cohort were not receiving concomitant disease-modifying SMA therapy; in the Restore registry study, 22 of 23 patients with 2 or more CHOP INTEND assessments improved or maintained their score
Ocuphire Pharma Inc., of Farmington Hills, Mich. Nyxol (phentolamine mesylate eye drops) Alpha 1/alpha 2 adrenoceptor antagonist   Mydriasis  In the Mira-2 study, 49% of study eyes treated with Nyxol returned to ≤ 0.2 mm of their baseline pupil diameter at 90 minutes compared to 7% of study eyes treated with placebo (p<0.0001); multiple secondary efficacy endpoints also met statistical significance; full results to be presented at the 2021 ASCRS Annual Meeting in July 2021
Opthea Ltd., of Melbourne, Australia OPT-302 VEGF-C/D trap inhibitor Treatment-naïve wet age-related macular degeneration First patient treated in the pivotal program comparing OPT-302 in combination with either Lucentis (ranibizumab, Roche Holding AG) or Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) to Lucentis or Eylea alone; primary endpoint for both studies is Best Corrected Visual Acuity from baseline to week 52
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris Libtayo (cemiplimab) Monoclonal antibody targeting PD-1 Recurrent or metastatic cervical cancer Study is being stopped early based on the recommendation of the independent data monitoring committee; compared to chemotherapy, Libtayo reduced the risk of death by 31% in the total population (p<0.001), by 27% in patients with squamous cell carcinoma (p=0.003) and by 44% in patients with adenocarcinoma (p<0.005)
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan TAK-620 (maribavir) Anti-cytomegalovirus compound Transplant recipients with refractory, with or without resistance, cytomegalovirus (CMV) infection/disease In the Solstice study, 55.7% of 117 transplant recipients with refractory, with or without resistance, CMV infection/disease treated with maribavir achieved confirmed CMV viremia clearance compared to 23.9% of 117 treated with 1 or a combination of antivirals ganciclovir, valganciclovir, foscarnet or cidofovir (p<0.001); 62.8% of the 121 transplant recipients with confirmed genotypic resistant CMV infection at baseline treated with maribavir achieved confirmed CMV viremia clearance at week 8 compared 20.3% of the 69 patients treated with antiviral(s)
Tonix Pharmaceuticals Holding Corp., of Chatham, N.J TNX-102 SL (cyclobenzaprine HCl sublingual tablets) Non-opioid, centrally acting analgesic Fibromyalgia The Rally study has randomized 50% of the 670 expected participants; interim results expected in the third quarter of 2021; top-line data expected in the fourth quarter of 2021


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