Company Product Description Indication Status
Phase I
Basilea Pharmaceutica Ltd., of Basel, Switzerland Lisavanbulin  Binds to the colchicine site of tubulin Recurrent glioblastoma or high-grade glioma Overall clinical benefit rate was 44% at 6 months at daily doses of 25-30 mg; plans to start phase 2 expansion study in patients who are EB1-positive shortly
Blueprint Medicines Corp., of Cambridge, Mass. Ayvakit (avapritinib) Kinase inhibitor Advanced systemic mastocytosis In the Explorer study, overall response rate was 76% in 53 patients, including 36% with complete remission with full or partial recovery of peripheral blood counts; median overall survival hadn't been reached yet
GT Biopharma Inc., of Beverly Hills, Calif. GTB-3550  Fusion protein conjugate composed of the variable regions of the heavy and light chains of anti-CD16 and anti-CD33 antibodies and a modified form of IL-15 CD33-positive leukemias  First patient enrolled at dose level 3; patient treated at 25-mcg/kg/day dose showed a decrease in acute myeloid leukemia blast levels from 18% to 12% in the bone marrow
Jasper Therapeutics Inc., of Redwood City, Calif. JSP-191 Monoclonal antibody targeting CD117 Myelodysplastic syndromes and acute myeloid leukemia undergoing hematopoietic cell transplantation First patient treated in the study testing the drug as a conditioning agent; study will test 3 dose levels plus standard conditioning regimen of low-dose radiation and fludarabine; primary outcome measure is the safety and tolerability of JSP-191
Miceros BV, of Hague, the Netherlands XZ-700 Targets endolysin in Staphylococcus aureus Mild to moderate atopic dermatitis Enrolled first of 48 patients in phase I/IIa study comparing 3 different concentrations of the drug to placebo; results expected toward the end of 2021
Nucana plc, of Edinburgh, U.K. NUV-3373 Thymidylate synthase inhibitor Metastatic colorectal cancer Drug had favorable pharmacokinetic and tolerability profile that was unaffected by leucovorin; some patients had stable disease for a longer period of time on NUC-3373 than on their prior line of therapy
Nucana plc, of Edinburgh, U.K. NUC-7738 Nucleoside analogue Advanced solid tumors Drug had favorable pharmacokinetic and tolerability profile; significant reductions in tumor volume in 2 patients
PDC*Line Pharma SA, of Liege, Belgium PDC*lung-01 Irradiated human plasmacytoïd dendritic cells loaded separately with 7 distinct synthetic human leukocyte antigen serotype-restricted peptide encoded by a tumor antigen  Non-small-cell lung cancer After completing the low-dose cohort in phase I/II trial, dosed first patient in the high-dose cohort; also opened a cohort testing the low dose plus pembrolizumab
Sorrento Therapeutics Inc., of San Diego Resiniferatoxin  Binds to TRPV1 receptors Intractable cancer pain Phase Ib data showed epidural injection resulted in dose-response relationship, with majority of responders at 15-ug and 25-ug dose levels; of 17 subjects, 11 achieved the clinical efficacy endpoint, defined as 30% decrease in pain, using NPRS scores
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (Ifenprodil)  N-methyl-D-aspartate receptor antagonist COVID-19 Enrolled 75 of the expected 150 patients in phase IIb/III trial
Blueprint Medicines Corp., of Cambridge, Mass. Ayvakit (avapritinib) Kinase inhibitor Advanced systemic mastocytosis The interim analysis of the Pathfinder study showed overall response rate was 75% in 32 patients, including 19% with complete remission with full or partial recovery of peripheral blood counts
Durect Corp., of Cupertino, Calif. DUR-928 Endogenous sulfated oxysterol Severe alcoholic hepatitis Plans to start phase IIb study in October comparing 2 dose levels to placebo; primary endpoint is 90-day survival; secondary endpoints include 28-day survival, the rate of adverse events, Lille and MELD scores and time in the intensive care unit
Frequency Therapeutics Inc., of Woburn, Mass. FX-322 Activates progenitor cells and regenerates auditory hair cells in the inner ear Sensorineural hearing loss Completed enrollment in the 95-patient phase IIb study; results expected in the second quarter of 2021
Novo Nordisk A/S, of Bagsværd, Denmark Insulin icodec Once-weekly insulin Type 2 diabetes In a switching study from other basal insulins, blood sugar time in range of 3.9–10 mmol/L during weeks 15 and 16 was 73% for insulin icodec and 65% for insulin glargine U100
Pulmatrix Inc., of Lexington, Mass., and Cipla Technologies LLC, of Mumbai, India Pulmazole Dry powder formulation of antifungal itraconazole  Asthma patients with allergic bronchopulmonary aspergillosis Stopping study that was put on hold due to the COVID-19 pandemic; plans to start a larger phase IIb study with a longer treatment duration
Santhera Pharmaceuticals Holding AG, of Pratteln, Switzerland Vamorolone  Anti-inflammatory delta 9,11 steroid Duchenne muscular dystrophy Data published in PLOS Medicine showed drug improved all motor outcomes from baseline to month 18: time to stand velocity (p=0.012), run/walk 10 meters velocity (p<0.001), climb 4 stairs velocity (p=0.001), 6-minute walk test, (p=0.001) and North Star Ambulatory Assessment (p<0.001)
Vaccinex Inc., of Rochester, N.Y. Pepinemab Anti-semaphorin 4D antibody Early manifest and prodromal Huntington’s disease In the 179-patient cohort B1 of the Signal study, drug didn't improve the Huntington’s Disease Cognitive Assessment Battery or the Clinical Global Impression of Change
Viralclear Pharmaceuticals, Inc., a unit of Biosig Technologies Inc., of Westport, Conn. Merimepodib  Broad-spectrum, oral antiviral  COVID-19 Expanded expected enrollment in the study testing merimepodib plus remdesivir from 40 to 80 patients with focus on hospitalized patients requiring non-invasive ventilation/high-flow oxygen devices
Phase III
Bayer AG, of Leverkusen, German  Nubeqa (darolutamide) Androgen receptor inhibitor  Non-metastatic castration-resistant prostate cancer Ad hoc analysis of the Aramis study showed 97.2% of patients taking Nubeqa plus androgen deprivation therapy (ADT) received the planned dose compared to 98.4% of patient who received ADT alone
Gensight Biologics SA, of Paris Lumevoq (endogene nolparvovec) Gene therapy using a mitochondrial targeting sequence Leber hereditary optic neuropathy Pooled data from phase III Rescue and Reverse trials and natural history studies found statistically significant and clinically meaningful difference between visual outcomes for treated and untreated patients; treated eyes showed progressive and sustained improvement from month 12 to month 52, in contrast to the absence of recovery over the same period for untreated eyes; at month 18, the difference became statistically significant (p=0.01); by month 48, difference between mean visual acuity in treated patients and untreated patients was both statistically significant (p<0.01) and clinically meaningful (-0.33 LogMAR, or +16.5 ETDRS letters equivalent, in favor of treated eyes
Sinovac Biotech Ltd., of Beijing Coronavac  Inactivated COVID-19 vaccine COVID-19 prophylaxis Started study, which will enroll 1,300 health care workers in the first stage and approximately 12,000 members of the general population in the second phase

Notes

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

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