Company Product Description Indication Status
Phase I
Boost Pharma, of Copenhagen, Denmark, and Karolinska Development AB, of Stockholm BT-101 Stem cell therapy Osteogenesis imperfecta Long-term data showed more than 50% of the treated patients did not experience any bone fractures in the second year after the last dose; overall reduction of nearly 78% compared to the pre-treatment period
Celldex Therapeutics Inc., of Hampton, N.J. CDX-622 Bispecific antibody targets neutralizing the alarmin thymic stromal lymphopoietin (TSLP) and depleting mast cells via stem cell factor (SCF) starvation Inflammation and fibrosis CDX-622 was well-tolerated; no dose limiting toxicities, serious adverse events, or infusion reactions and no emergent events related to systemic KIT inhibition; mAb-like pharmacokinetics (serum half-life of approximately 18 days at 9 mg/kg) without any evidence of antidrug antibodies; rapid and sustained decrease (~50%) in circulating tryptase consistent with systemic mast cell inhibition and depletion
Parabilis Medicines Inc., of Cambridge, Mass. FOG-001 Direct inhibitor of β-cateninTCF4 Microsatellite stable colorectal cancer and other Wnt-pathway activated solid tumors Data showed well-tolerated; no grade 4/5 treatment-related adverse events or discontinuations; favorable pharmacokinetic profile; objective response rate of 43% and a disease control rate of 83%, per RECIST 1.1 in non-CRC patients with Wnt pathway activating mutations; 50% DCR was achieved within the efficacious monotherapy dose range in MSS CRR
Vesper Bio ApS, of Copenhagen, Denmark VES-001 Competitive sortilin inhibitor that selectively prevent degradation of progranulin Asymptomatic carriers with frontotemporal degeneration (FTD) caused by mutations in the progranulin gene Oral therapy showed dose-dependent increases in progranulin protein levels in both plasma and cerebrospinal fluid; >95% mean increase in progranulin levels in CSF, compared to baseline; favorable safety and tolerability profile during the three-month daily dosing regimen; fewer adverse events
Phase III
Astrazeneca plc, of Cambridge, U.K. Gefurulimab Complement C5 inhibitor Anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalized myasthenia gravis Study achieved primary and secondary endpoints; improvement from baseline in MG-ADL total score at week 26 compared to placebo (p<0.0001); clinically meaningful improvement was observed as early as week one, and was sustained through week 26; clinically meaningful improvement in QMG total score (p<0.0001) at week 4 and sustained through week 26 (p<0.0001); well-tolerated; consistent safety profile; mild to moderate in severity (grade 1 or 2)
Hightide Therapeutics Inc., of Shenzhen, China HTD-1801 Ionic salt of berberine and ursodeoxycholic acid; gut-liver anti-inflammatory metabolic modulator Type 2 diabetes mellitus Monotherapy showed reduction from baseline in HbA1c with HTD-1801 (-1.3%) was superior to placebo at week 24, maintained (-1.2% at week 52); placebo patients who switched to HTD-1801 saw a reduction in HbA1c of -1.3% at week 52; reduction from baseline in HbA1c with HTD-1801 (-1.2%, as an add-on therapy to Metformin) was superior to placebo and maintained -1.1% at week 52; placebo patients who switched to HTD-1801 saw a reduction in HbA1c of -1.2% at week 52; significant reductions in low-density lipoprotein cholesterol (LDL-C) and non-high-density lipoprotein cholesterol (non-HDL-C); continued reductions in key inflammatory biomarkers, including gamma-glutamyl transferase (GGT) and high-sensitivity C-reactive protein (hs-CRP); estimated glomerular filtration rate (eGFR) remained stable in the overall population; favorable and consistent with the double-blind phase
Stallergenes Greer plc, of London Staloral Sublingual solution of allergen extracts; allergen immunotherapy Birch pollen-induced allergic rhino-conjunctivitis (ARC) with or without asthma Study achieved primary endpoint in a pediatric population (5-17); 41% improvement in the ARC total combined score during the second pollen season compared to placebo; statistically significant (p<0.0001) and clinically meaningful with a between-group difference of -2.62 points; statistically significant difference (-0.41, p<0.0001) when using the combined symptom and medication score; similar results were also observed, both statistically significant and clinically relevant in first pollen season; consistent safety profile

Notes

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