Company Product Description Indication Status
Phase I
Cstone Pharmaceuticals Co. Ltd., of Suzhou, China CS-3002 Inhibits cyclin-dependent kinases 4 and 6 Advanced solid tumors Study stopped for strategic and business reasons
Treadwell Therapeutics Inc., of New York CFI-40094 Polo-like kinase 4 inhibitor Leukemia  Started patient dosing in the TWT-202 study testing CFI-40094 as a monotherapy and in combination with hypomethylating agents
Phase II
AB Science SA, of Paris Masitinib   Tyrosine kinase inhibitor that targets mast cells and macrophages Metastatic castrate-resistant prostate cancer The phase IIb/III AB12003 study comparing masitinib plus docetaxel and prednisone to docetaxel and prednisone alone met its primary endpoint of progression-free survival
Cadent Therapeutics, part of Basel, Switzerland-based Novartis AG CAD-1883 Positive allosteric modulator of SK channels Spinocerebellar ataxia Novartis, which acquired Cadent in January 2021, decided not to proceed with Synchrony-1 study as planned
Eloxx Pharmaceuticals Inc., of Waltham ELX-02 CFTR gene modulator; ribosomal protein modulator  Cystic fibrosis with at least 1 G542X allele Adding a fifth treatment arm testing ELX-02 plus Kalydeco (ivacaftor, Vertex Pharmaceuticals Inc.)
Synairgen plc, of Southampton, U.K. SNG-001 Inhaled interferon beta COVID-19 A post-hoc analysis of patients who had significant breathlessness in the home cohort and the hospitalized cohort showed the 33 patients treated with SNG-001 were 3.41 times more likely to recover than the 36 patients who took placebo (p=0.004)
Tessa Therapeutics Ltd., of Bedminster, N.J. Autologous CD30 CAR T CAR T targeting CD30 Relapsed/refractory classical Hodgkin lymphoma Completed enrollment of the 12-patient pilot cohort; plans to start enrollment of the 82-patient pivotal cohort in the second half of 2021
Phase III
Mesoblast Ltd., of New York Remestemcel-L Culture-expanded mesenchymal stromal cells Ventilator-dependent COVID-19 with moderate/severe acute respiratory distress syndrome  Remestemcel-L reduced mortality through day 60 by 46% in the prespecified group below age 65, but not in patients 65 or older; remestemcel-L plus dexamethasone reduced mortality by 75% and increased days alive off mechanical ventilation in patients under age 65 compared to dexamethasone alone
Phathom Pharmaceuticals Inc., of Florham Park, N.J. Vonoprazan  Potassium-competitive acid blocker H. pylori infection Vonoprazan plus amoxicillin and clarithromycin therapy produced an H. pylori eradication rate of 84.7% compared to 78.5% for vonoprazan plus amoxicillin and 78.8% for lansoprazole plus amoxicillin and clarithromycin (p<0.0001 and p=0.0037 for the vonoprazan triple and double combinations, respectively, compared to lansoprazole triple combination for noninferiority)
Scynexis Inc., of Jersey City, N.J. Brexafemme (ibrexafungerp) Glucan synthase inhibitor Vulvovaginal candidiasis A subanalysis of the VANISH-303 study of patients with non-albicans Candida infection showed Brexafemme produced a 42.1% clinical cure rate at day 10 and 52.6% symptom resolution rate at the day 25; in severe patients, clinical cure rate at day 10 was 63.3% for Brexafemme compared to 44% for placebo (p=0.007)
Vifor Fresenius Medical Care Renal Pharma Ltd., of St. Gallen, Switzerland Velphoro (PA-21) Non-calcium, iron-based, chewable phosphate binder Chronic kidney disease on dialysis The PA-CL-CHINA-01 study met its primary endpoint demonstrating noninferiority vs. sevelamer carbonate in the change from baseline in serum phosphorus levels at week 12; Velphoro lowered serum phosphorus levels faster than sevelamer carbonate with fewer average daily tablets (3.23 for Velphoro vs. 6.31 for sevelamer carbonate)

Notes

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