Company Product Description Indication Status
Phase I
Flexion Therapeutics Inc., of Burlington, Mass. FX-201 Helper-dependent adenovirus expressing an interleukin-1 receptor antagonist under the control of an inflammation-responsive promote Osteoarthritis  There was no evidence of systemic biodistribution of FX-201 in plasma or shedding in urine or at the injection site in any of the 5 treated patients; 40% of patients demonstrated substantial improvement in knee osteoarthritis pain at weeks 8, 12 and 24 following treatment
Lutris Pharma, of Tel Aviv, Israel LUT-014 B-Raf inhibitor EGFR inhibitor-induced acneiform lesions in metastatic colorectal cancer Data in Cancer Discovery showed all 6 patients who started with grade 2 rash in the low- and intermediate-dose cohorts improved over the 28-day treatment; improvement was maintained a month after treatment cessation
Organicell Regenerative Medicine Inc., of Miami Zofin Growth factors, cytokines, chemokines and extracellular vesicles/nanoparticles derived from perinatal tissue  Moderate to severe COVID-19 All of the first 10 patients in the study in India recovered from their symptoms and have since been discharged from the hospital; plans to expand study to an additional 65 patients; treatment expected to be completed by the end of June 2021
Redx Pharma plc, of Alderley Park, U.K. RXC-004 Porcupine inhibitor Advanced malignancies Treated first 3 patients in the study testing the safety and tolerability of RXC-004 plus Opdivo (nivolumab, Bristol Myers Squibb Co.); data expected in the second half of 2021
Trillium Therapeutics Inc., of Cambridge, Mass. TTI-621 SIRPa-IgG1 Fc fusion protein targeting CD47 Relapsed/refractory T- and B-cell lymphomas Objective response rate was 19% in 62 patients with cutaneous T-cell lymphoma, 18% in 22 patients with peripheral T-cell lymphoma and 29% in 7 patients with diffuse large B-cell lymphoma
Trillium Therapeutics Inc., of Cambridge, Mass. TTI-621 SIRPa-IgG1 Fc fusion protein targeting CD47 Second-line peripheral T-cell lymphoma, relapsed/refractory diffuse large B-cell lymphoma and first-line leiomyosarcoma Plans to start phase Ib/II program testing TTI-621 as a monotherapy for PTCL, in combination with an anti-PD-1 for DLBCL and in combination with doxorubicin for leiomyosarcoma
Trillium Therapeutics Inc., of Cambridge, Mass. TTI-622 SIRPalpha-IgG4 fusion protein targeting CD47 Relapsed/refractory lymphoma  In 27 patients treated with 0.8 mg/kg or higher, the objective response rate was 33%, including 7.4% complete responses
Trillium Therapeutics Inc., of Cambridge, Mass. TTI-622 SIRPalpha-IgG4 fusion protein targeting CD47 Relapsed/refractory multiple myeloma after at least 3 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug and an anti-CD38 antibody Treated first of approximately 30 patients with TTI-662 plus carfilzomib and dexamethasone in the phase Ib portion of the ongoing phase Ia/Ib study; primary endpoints are safety and overall response rate
Trillium Therapeutics Inc., of Cambridge, Mass. TTI-622 SIRPalpha-IgG4 fusion protein targeting CD47 First-line p53 mutant acute myeloid leukemia, first-line elderly or unfit p53 wild-type AML, relapsed/refractory diffuse large B-cell lymphoma, platinum-resistant ovarian cancer Plans to start phase Ib/II program testing TTI-622 in combination with azacitidine for p53 mutant AML, in combination with azacitidine and venetoclax for p53 wild type AML, in combination with an anti-PD-1 for DLBCL, in combination with chemotherapy for ovarian cancer and in a second solid tumor to be disclosed later in 2021
Phase II
Arrowhead Pharmaceuticals, Inc., of Pasadena, Calif., and Takeda Pharmaceutical Co. Ltd., of Tokyo ARO-AAT RNA interference targeting mutant AAT Alpha-1 antitrypsin deficiency After 48 weeks of treatment in the AROAAT2002 study, 4 of 5 patients achieved a 1 or greater stage improvement in Metavir fibrosis stage; 5th patient had no worsening of fibrosis; all 5 patients had reductions in histological globule assessment scores; total intra-hepatic Z-AAT decreased by 77%-97%
Cardiol Therapeutics Inc., of Oakville, Ontario Cardiolrx Cannabidiol  COVID-19 with pre-existing or significant risk factors for cardiovascular disease Randomized first patient in the phase II/III Lancer study; primary efficacy endpoint is percentage of patients who develop all-cause mortality, requirement for ICU admission and/or ventilatory support, as well as cardiovascular complications, including the development of heart failure, acute myocardial infarction, myocarditis, stroke, or new sustained or symptomatic arrhythmia, during the first 28 days
Innovation Pharmaceuticals Inc., of Wakefield, Mass. Brilacidin   Dual PDE3/4 inhibitor Hospitalized moderate to severe COVID-19 More than 60 of the expected 120 patients have been randomized in the study
Santhera Pharmaceuticals AG, of Pratteln, Switzerland, and Reveragen Biopharma Inc., of Rockville, Md. Vamorolone Dissociative steroid Duchenne muscular dystrophy Improvement in time to stand velocity of approximately 0.05 rises/second seen after 6 months was maintained at 2.5 years when compared to age-matched steroid-naive patients from the CINRG-DMD Natural History Study
Phase III
Beigene Co. Ltd., of Beijing Brukinsa (zanubrutinib) BTK inhibitor Relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma At the interim analysis of the Alpine study, Brukinsa produced superior objective response rate compared to Imbruvica (ibrutinib, Johnson & Johnson/Abbvie Inc.) by investigator assessment (p=0.0006); by independent review committee assessment, ORR was numerically higher but not statistically significant (p=0.0121); data to be presented at an upcoming major medical conference
G1 Therapeutics Inc., of Research Triangle Park, N.C. Cosela (trilaciclib) Myelopreservation agent Locally advanced unresectable or metastatic triple-negative breast cancer Started the Preserve 2 study in patients receiving first- or second-line gemcitabine and carboplatin chemotherapy, evaluated separately for overall survival; secondary endpoints include quality of life assessments
Novaliq GmbH, of Heidelberg, Germany Cyclasol Topical anti-inflammatory and immunomodulating ophthalmic solution, containing 0.1% cyclosporine A  Dry eye disease More than 50% of the 834 targeted patients have been randomized in the Essence-2 study; top-line results expected in the second half of 2021
Resverlogix Corp., of Calgary, Alberta Apabetalone Bromodomain and extra-terminal inhibitor  Chronic kidney disease, type-2 diabetes mellitus and recent acute coronary syndrome Data from the Betonmace study published in the American Society of Nephrology showed apabetalone produced a 52% hazard reduction for MACE events, such as cardiovascular death and heart failure, compared to placebo (p=0.03); in patients with CKD, apabetalone reduced alkaline phosphatase after 24 weeks of treatment (p=0.004)
Phase IV
EMD Serono, the biopharmaceutical business of Merck KGaA, of Darmstadt, Germany Mavenclad (cladribine) Adenosine deaminase inhibitor  Relapsing multiple sclerosis In the Magnify-MS sub-study, memory B cells were reduced as early as 1 month after starting treatment with lowest levels that were sustained for up to 12 months; naïve B cells began recovering immediately


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