Company Product Description Indication Status
Phase I
Altimmune Inc., of Gaithersburg, Md. Nasoshield Anthrax protective antigen modulator Bacillus anthracis infection First of 42 healthy participants dosed with intranasally administered study drug or placebo and will be followed for 6 months; readouts are serum titers of antibody to protective antigen and toxin-neutralizing antibody 28 and 56 days after dosing; stimulation of mucosal IgA immune response in nasal cavity will be assessed as potential additional benefit to serum antibody responses
Blueprint Medicines Corp., of Cambridge, Mass. Ayvakit (avapritinib)  KIT tyrosine kinase inhibitor; PDGF receptor alpha antagonist Gastrointestinal stromal tumor  Navigator trial data in participants with PDGFRA D842V-mutant disease, published in The Lancet Oncology, showed overall response rate of 88%; 9% achieved complete response; 12-month duration of response rate was 70%, with 12-month progression-free survival rate of 81% and 24-month overall survival rate of 81% 
Humanigen Inc. of Burlingame, Calif., and Kite, a unit of Gilead Sciences Inc., of Foster City, Calif. Lenzilumab  GM-CSF ligand inhibitor; immunoglobulin G1/kappa modulator Large B-cell lymphoma First participant infused in phase I/II study that includes variable regimens, including study drug, axicabtagene ciloleucel, cyclophosphamide and fludarabine; efficacy endpoints include complete response, duration of response, objective response rate and overall survival 
Impel Neuropharma Inc., of Seattle INP-105 Powder formulation of olanzapine for intranasal delivery Acute agitation Results from Snap 101 study in healthy volunteers published in The Journal of Clinical Psychiatry showed drug reached peak plasma levels about twice as fast as intramuscular olanzapine and 10 times faster than orally disintegrating tablets (ODT); maximum and total plasma levels were similar to intramuscular delivery of same dose and exceeded total plasma levels for ODT; pharmacodynamic measures of sedation, including Visual Analogue Scale, the Agitation and Calmness Evaluation Scale and Digit Symbol Substitution Test, all showed statistical significance compared to placebo
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. INO-4800 COVID-19 Spike glycoprotein modulator COVID-19 infection Analyses of 1-mg and 2-mg dose cohorts after 2 doses at week 6 showed 94% (34 of 36) demonstrated overall immunological response rates based on preliminary data assessing humoral and T-cell immune responses; 1 participant in 1-mg cohort and 2 in 2-mg cohort were excluded in analyses as they tested positive for COVID-19 immune responses at study entry, indicating prior infection
Meissa Vaccines Inc., of South San Francisco MV-012-968 Live attenuated vaccine  Respiratory syncytial virus infection Interim results of ongoing dose-ranging phase Ia trial showed vaccine candidate generated immune response in healthy adults and was safe and well-tolerated among healthy RSV sero-low adults assessed through day 56; 106 PFU dose resulted in no detectable shed vaccine virus nasally, yet induced RSV-specific mucosal IgA in most recipients, despite pre-existing immunity to RSV
Noxxon Pharma NV, of Berlin NOX-A12 Targets CXCL12 Newly diagnosed brain cancer Enrolled and treated first patient in middle-dose cohort of phase I/II trial; study testing 3 doses (200 mg, 400 mg and 600 mg/week), each combined with external-beam radiotherapy
Oncternal Therapeutics, Inc., of San Diego  Cirmtuzumab NTRKR1 tyrosine kinase receptor inhibitor Mantle cell lymphoma Based on previously presented interim phase I/II data from Cirll combination trial with ibrutinib (Imbruvica, Abbvie Inc.), study amended to increase number of participants with relapsed/refractory disease in phase II expansion cohort to at least 20 and to allow enrollment of those with broader range of prior BTK inhibitor treatments; company requested meeting with FDA to discuss interim data and seek guidance on potential accelerated approval pathway for combination in relapsed/refractory disease; Oncternal will continue treatment and follow-up of those with chronic lymphocytic leukemia (CLL) enrolled in Cirll study but will limit enrollment in phase II CLL cohort to approximately 35 to focus resources on MCL
Orexo AB, of Uppsala, Sweden OX-125 (nalmefene, intranasal) Opioid receptor antagonist Opioid dependence Pharmacokinetic crossover comparative bioavailability study in healthy volunteers that assessed absorption from 3 development formulations compared to nalmefene intramuscular injection showed extensive and rapid absorption and good tolerability
Rheacell GmbH & Co. KG, of Heidelberg, Germany Allo-APZ2-PAOD  Allogeneic ABCB5-positive mesenchymal stem cells  Peripheral arterial occlusive disease Phase I/II study terminated due to very slow enrollment amid COVID-19 pandemic
Rubius Therapeutics Inc., of Cambridge, Mass. RTX-240  CDw137 agonist; IL-15 receptor agonist Advanced solid tumors Dosing of first dose-escalation cohort in phase I/II trial completed with no observed adverse events 
Phase II
Aobiome Therapeutics Inc., of Cambridge, Mass.  B-244 Ammonia oxidizing bacteria-based therapeutic Pruritus Initiated phase IIb study expected to enroll 576 adults with history of mild to moderate disease; primary and key secondary efficacy endpoints include mean change in Worst Itch Numeric Rating Scale from baseline to week 4 and proportion of subjects with ≥4 point improvement in WI-NRS from baseline to week 4, respectively
Beyondspring Inc., of New York Plinabulin Guanine nucleotide exchange factor stimulator; tubulin receptor antagonist Breast cancer Combining study drug with low dose of pegfilgrastim produced statistically significant CD34+ cell counts (p<0.03) and increases to values comparable to, or numerically higher than, full dose of pegfilgrastim alone under severe myelosuppressive conditions
Can-Fite Biopharma Ltd., of Petach Tikva, Israel  Namodenoson Adenosine A3 receptor agonist; phosphatidylinositol 3 kinase subunit 3 inhibitor Nonalcoholic fatty liver disease with or without nonalcoholic steatohepatitis 25 mg confirmed as optimal dose based on MRI-PDFF analysis and liver enzymes, reduction of liver fibrosis and resolution of all cases of NASH
Galectin Therapeutics Inc., of Norcross, Ga. Belapectin (GR-MD-02) Galectin-3 inhibitor Nonalcoholic steatohepatitis Enrolled first patient in the NASH-RX study, which will include 315 patients treated for 78 weeks in the phase IIb part; phase III size will be determined by the phase IIb results
Marinus Pharmaceuticals Inc., of Radnor, Pa.  Ganaxolone  Positive allosteric modulator of GABAA receptors Tuberous sclerosis complex Enrolled first of about 30 patients in the open-label study; primary endpoint is percent change in 28‑day primary seizure frequency for the treatment period relative to baseline
Nordic Nanovector ASA, of Oslo, Norway Betalutin (177Lu-lilotomab satetraxetan) CD37-targeting antibody-radionuclide-conjugate 3rd-line relapsed/refractory follicular lymphoma  Plans to broaden the inclusion criteria for the Paradigme study to allow patients who have undergone a stem cell transplant to be enrolled
Novartis AG, of Basel, Switzerland LOU-064 BTK inhibitor Asthma Randomized, placebo-controlled study in patients with inadequately controlled asthma terminated due to company decision
Oncternal Therapeutics, Inc., of San Diego  Cirmtuzumab NTRKR1 tyrosine kinase receptor inhibitor Chronic lymphocytic leukemia Investigator-sponsored trial expected to assess combination of study drug and BCL-2 inhibitor venetoclax (Venclexta, Roche Holding AG/Abbvie Inc.) in relapsed/refractory disease 
Orthocell Ltd., of Perth, Australia, and Depuy Synthes Products, a unit of New Brunswick, N.J.-based Johnson & Johnson Ortho-ATI Tendon regeneration therapy Rotator cuff tendinopathy and tear Completed enrollment in the study
Reneuron Group plc., of London hRPC stem cell therapy Human retinal progenitor cell Retinitis pigmentosa Mean change from baseline in visual acuity in treated eye was +7.1 letters at 9 months for 8 patients, +11.9 letters at 12 months for 5 patients and +17 letters at 18 months for 1 patient
Revance Therapeutics Inc., of Newark, Calif DaxibotulinumtoxinA  Botulinum toxin Dynamic forehead lines and lateral canthal line In the forehead lines study, 100% of the 61 patients achieved a score of none or mild at week 4 in at least 1 treatment group; in the canthal line study, 88% of 63 patients achieved a score of none or mild at week 4 in at least 1 treatment group
Seattle Genetics Inc., of Bothell, Wash., and Genmab A/S, of Copenhagen, Denmark Tisotumab Antibody-drug conjugate targeting tissue factor Recurrent or metastatic cervical cancer after relapse or progression on or after prior treatment In the InnovaTV 204 study, confirmed objective response rate was 24%; median duration of response was 8.3 months
Phase III
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Givlaari (givosiran) RNAi targeting aminolevulinic acid synthase 1 Acute hepatic porphyria In the open-label extension, from 6 to 12 months, median annualized rate of composite porphyria attacks (AAR) was 0; 61.7% of patients were attack-free; patients who crossed over from placebo had a mean reduction in AAR of 76%
Amniox Medical Inc., of Miami, a Tissuetech Inc. company TTAX-01 Cryopreserved human umbilical cord Diabetic foot ulcers Started first of 2 planned studies, each to enroll 220 patients; Ambulate DFU and Ambulate DFU II will test TTAX-01 in achieving complete wound closure of complex nonhealing DFUs with high-risk factors of ulcer depth indicating exposed bone, tendon, muscle and/or joint capsule, and clinical suspicion of osteomyelitis
Exelixis Inc., of Alameda, Calif., and Roche Holding AG, of Basel, Switzerland Cabometyx (cabozantinib) Tyrosine kinase inhibitor Metastatic castration-resistant prostate cancer Started the 580-patient Contact-2 study testing Cabometyx plus Tecentriq (atezolizumab) compared to a second novel hormonal therapy (either abiraterone and prednisone or enzalutamide); primary endpoints are progression-free survival and overall survival
Iveric Bio Inc., of New York Zimura (avacincaptad pegol) Complement C5 inhibitor Geographic atrophy secondary to age-related macular degeneration First of about 400 patients dosed in the Gather2 study; primary endpoint is change of geographic atrophy growth at 12 months
Marinus Pharmaceuticals Inc., of Radnor, Pa.  Ganaxolone  Positive allosteric modulator of GABAA receptors Status epilepticus Finalized protocol for study of patients who have failed benzodiazepines and 2 or more intravenous anti-epileptic drugs; primary endpoints are proportion of patients with status epilepticus (SE) cessation within 30 minutes of treatment initiation without medications for the acute treatment of SE and proportion of patients with no progression to I.V. anesthesia for 36 hours following treatment initiation
Zynerba Pharmaceuticals Inc., of Devon, Pa. Zygel CBD transdermal gel Fragile X syndrome Study didn’t achieve statistical significance in primary or key secondary endpoints for the 212 patients in the study; a preplanned ad hoc analysis of patients having at least 90% methylation of the impacted FMR1 gene showed Zygel improved Social Avoidance subscale of the ABC-CFXS at 12 weeks compared to placebo (p=0.020)

Notes

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