Company Product Description Indication Status
Phase I
Atara Biotherapeutics Inc., of South San Francisco ATA-188 Off-the-shelf, allogeneic EBV-specific T-cell immunotherapy Progressive forms of multiple sclerosis Enrolled first patient in phase Ib study; trial will measure change in disability measures compared to baseline and will include multiple measures of function and various biomarkers
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Valoctocogene roxaparvovec Gene therapy Severe hemophilia A Additional data from 4-year update of phase I/II study showed 93% reduction from baseline in annualized bleed rate (ABR) in 6e13-vg/kg cohort, with mean ABR of 1.3 and median of 0 in year 4 and 96% reduction in mean factor VIII usage to 5.4 infusions per year cumulatively; among 7 participants in 6e13-vg/kg cohort, 6 (86%) were bleed-free in fourth year; in 4e13-vg/kg cohort, cumulative mean ABR was reduced by 93% to 0.9 with continued absence of target joint bleeds in 5 of 6 subjects during 3 years observed, which represents a 93% reduction from baseline; 96% reduction in mean FVIII usage to 5.7 infusions per year cumulatively over 3 years from baseline 
Dynavax Technologies Corp., of Emeryville, Calif., and Merck & Co. Inc., of Kenilworth, N.J. SD-101 Targets Toll-like receptor 9 Metastatic melanoma or recurrent/metastatic head and neck squamous cell carcinoma Phase Ib/II study testing combination with Keytruda (pembrolizumab) terminated following strategic restructuring
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Mavrilimumab Fully human monoclonal antibody targeting GM-CSF receptor alpha COVID-19 pneumonia 28-day clinical outcomes data, published in The Lancet Rheumatology, showed treated patients had earlier and improved outcomes vs. controls; drug set to be evaluated in phase II/III trial
Nantpharma LLC, of Culver City, Calif. NANT-008 Nanoparticle-based formulation of polymeric micelles encapsulating paclitaxel Metastatic pancreatic adenocarcinoma Phase Ib/II study testing combination with 5-fluorouracil, bevacizumab, leucovorin and oxaliplatin suspended on institutional review board recommendation
Phase II
Affimed NV, of Heidelberg, Germany AFM-24 Tetravalent, bispecific EGFR/CD16A-binding innate cell engager Advanced solid EGFR-expressing malignancies Completed first dose cohort in phase I/IIa trial in patients whose disease has progressed after treatment with previous cancer therapies, with no dose-limiting toxicity observed; study cleared to proceed to cohort 2
Akero Therapeutics Inc., of South San Francisco AKR-001 Engineered to mimic biological activity profile of native FGF21 Nonalcoholic steatohepatitis  Dosed first subject in cohort C in patients with compensated cirrhosis, Child-Pugh class A, as part of the expansion of phase IIa Balanced trial; primary objective of cohort C is to assess safety and tolerability in NASH patients at greatest risk of progressing to end-stage liver disease
Ascentage Pharma Group International, of Suzhou, China APG-1387 IAP antagonist Chronic hepatitis B Dosed first patient in China; study testing combination with entecavir in both treatment-naive and -experienced patients; 104 patients to be enrolled globally
Ascletis Pharma Inc., of Hangzhou, China, and Sagimet Biosciences Inc., of Shaoxing, China TVB-2640 FASN inhibitor Nonalcoholic steatohepatitis Results from Fascinate-1 trial showed drug significantly reduced liver fat, the primary efficacy endpoint, with a 61% responder rate in 50-mg group; participants also showed improvement in markers of liver function and fibrosis; TVB-2640 also significantly decreased ALT by up to 20.4% and LDL-cholesterol by up to 7.6% at week 12; an additional 50-mg cohort of 25-30 patients in China has started screening
Astrazeneca plc, of Cambridge, U.K. Durvalumab and tremelimumab Anti-PD-L1 antibody and anti-CTLA4 antibody Bladder cancer Study testing neoadjuvant therapy in patients ineligible for cisplatin terminated due to low accrual 
Biosig Technologies Inc., of Westport, Conn. Merimepodib Broad-spectrum antiviral Advanced COVID-19 Started the clinical trial testing merimepodib plus remdesivir
Cara Therapeutics Inc., of Stamford, Conn. Korsuva (difelikefalin) Kappa opioid receptor agonist Moderate to severe pruritus in atopic dermatitis patients Enrollment target increased from 320 patients to 410 patients after a prespecified interim conditional power assessment; completion of enrollment expected in fourth quarter of 2020
Contrafect Corp., of Yonkers, N.Y. Exebacase  Recombinant lysin Staph aureus bloodstream infections Clinical responder rate at day 14 was 70.4% for exebacase plus antibiotics and 60% for antibiotics alone (p=0.314); in the methicillin-resistant Staphylococcus aureus subgroup responder rates were 42.8 percentage points higher (p=0.010); exebacase plus antibiotics produced a 21-percentage point reduction in the 30-day all-cause mortality (p=0.056)
Evgen Pharma plc, of Cheshire, U.K. SFX-01 Up-regulates Nrf2 pathway COVID-19 Drug selected for evaluation in phase II/III trial to be sponsored by University of Dundee to test whether it can reduce severity or prevent onset of acute respiratory distress syndrome associated with COVID-19
Inflarx NV, of Jena, Germany IFX-1  Monoclonal antibody targeting complement factor C5a Severe COVID-19 pneumonia In the phase II portion, 28-day all-cause mortality was 13% for 15 patients treated with IFX-1 and 27% for 15 patients given best supportive care; independent data safety monitoring board recommended continuation of the trial into the phase III part
Remegen Co. Ltd., of Yantai, China RC-48 (disitamab vedotin) HER2-targeting antibody-drug conjugate HER2-expressing advanced or metastatic gastric cancer Results from study in heavily treated patients showed confirmed objective response rate of 23.6%, with median progression-free survival of 4.1 months; median overall survival was 7.5 months
Phase III
Daré Bioscience Inc. of San Diego DARE-BV1  Thermosetting bioadhesive hydrogel containing clindamycin phosphate Bacterial vaginosis Started the Dare-BVfree study in 240 patients; primary endpoint is clinical cure of bacterial vaginosis, defined as resolution of specified clinical signs and symptoms 21 to 30 days after enrollment in the study
Eli Lilly and Co., of Indianapolis Emgality (galcanezumab-gnlm) Monoclonal antibody targeting calcitonin gene-related peptide Episodic and chronic migraine In a post-hoc analysis of the Evolve-1 and Evolve-2 studies, patients with episodic migraine treated with Emgality experienced 68.6 fewer severity-weighted hours of pain per month on average than at baseline compared to 36.2 fewer hours for placebo; patients with chronic migraine treated with Emgality experienced 102.6 fewer severity-weighted hours of pain per month compared to 44.4 fewer hours for placebo
Recordati SpA, of Milan, Italy Isturisa (osilodrostat)  Oral inhibitor of 11-beta-hydroxylase Cushing’s disease  In the Linc-4 study, 77% of patients taking Isturisa achieved normal mean urinary free cortisol (mUFC) at 12 weeks compared to 8% of patients taking placebo (p<0.0001); 81% of patients had sustained improvements in mUFC levels over 36 weeks

Notes

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