Company Product Description Indication Status
Phase I
4D Pharma plc, of Leeds, U.K. MRx-0518 Live biotherapeutic Solid tumors with acquired resistance to prior immune checkpoint therapy Treated first patient in part B of study testing drug plus Keytruda (pembrolizumab, Merck & Co. Inc.) in up to 30 patients per tumor type cohort
Adverum Biotechnologies Inc., of Redwood City, Calif. ADVM-022 Gene therapy expressing aflibercept Wet age-related macular degeneration Completed dosing of cohort 4 in the Optic study; data from all 4 cohorts expected by the end of 2020
Avrobio Inc., of Cambridge, Mass. AVR-RD-02 Gene therapy expressing glucocerebrosidase Gaucher disease type 1 Treated first of 8-16 patients in phase I/II study
Avrobio Inc., of Cambridge, Mass. AVR-RD-04 Gene therapy expressing cystinosin Cystinosis Treated second of up to 6 patients in phase I/II study
Anavex Life Sciences Corp., of New York ANAVEX3-71 (AF710B) Targets sigma-1 and M1 muscarinic receptors Healthy volunteers (eventually neurodegenerative diseases) Enrolled first of at least 36 subjects in the study testing single escalating doses of the drug to measure safety, tolerability and pharmacokinetics
Gilead Sciences Inc., of Foster City, Calif. Lenacapavir (GS-6207) HIV-1 capsid inhibitor HIV infection Therapeutic plasma concentrations were sustained for at least 6 months following a single 900-mg dose, supporting 6-month dosing interval
Phase II
Bellus Health Inc., of Laval, Quebec BLU-5937 P2X3 antagonist Refractory chronic cough Highest dose, 200 mg twice daily, reduced placebo-adjusted awake cough frequency by 17% (p=0.09); in patients with at least 32.4 cough/hr at baseline, the reduction was 32% (p=0.0006)
Celixir plc, of Stratford-upon-Avon, U.K. CLXR-001 Allogeneic iMP cells Cardiomyopathy Suspending the Heartcel study and plans to replace the current CTA with a revised protocol that's scheduled to start enrollment in the third quarter of 2020
CSL Behring, a unit of Melbourne, Australia-based CSL Ltd. CSL-312 (garadacimab) Monoclonal antibody targeting factor XIIa Severe respiratory distress in COVID-19 patients with pneumonia Enrolled first of about 124 patients in the study testing the incidence of tracheal intubation or death
Inventiva SA, of Daix, France Lanifibranor Activates the 3 peroxisome proliferator‑activated receptor isoforms Type 2 diabetes with nonalcoholic fatty liver disease Reducing the number of participants from 64 to 34, based on the results of the phase IIb Native study
Merck & Co. Inc., of Kenilworth, N.J. Islatravir Nucleoside reverse transcriptase translocation inhibitor HIV infection Treatment with 0.25, 0.75 and 2.25 mg islatravir plus Pifeltro (doravirine) for 48 weeks resulted in 89.7%, 90% and 77.4%, respectively, of patients achieving HIV-1 RNA <50 copies/mL, compared to 83.9% of patients taking Delstrigo (doravirine/lamivudine/tenofovir disoproxil fumarate)
Oryzon Genomics SA, of Madrid Vafidemstat LSD1 inhibitor Borderline personality disorder, attention deficit hyperactivity disorder and autism spectrum syndrome Vafidemstat produced reductions in the CGI-S, CGI-I, NPI A/A and Total NPI scales and improved scores in the disease-specific scales: BPDCL for BPD and ADHD-RS for ADHD; reduction in C-SSRS scale was seen in BPD patients; correlation between most of the clinical outcomes and vafidemstat exposure was observed
Sunovion Pharmaceuticals Inc., of Marlborough, Mass. SEP-4199 Non-racemic ratio of amisulpride enantiomers with increased potency for serotonin 5-HT7 receptors relative to dopamine D2 receptors Major depressive episodes associated with bipolar I disorder Drug reduced Montgomery-Åsberg Depression Rating Scale total score by 19.5 and 19.3 points for the 200-mg and 400-mg doses, respectively, compared to a 16.2-point reduction for placebo (p=0.054 for both)
Phase III
Deciphera Pharmaceuticals Inc., of Waltham, Mass. Qinlock (ripretinib) Switch-control tyrosine kinase inhibitor that inhibits KIT and PDGFR-alpha Fourth-line gastrointestinal stromal tumor In the Invictus study, patients on the placebo arm who crossed over to Qinlock had a median progression-free survival of 4.6 months from start of Qinlock; 2 of 29 patients had a partial response; overall survival was 11.6 months from trial initiation
Gensight Biologics SA, of Paris Lumevoq (lenadogene nolparvovec) Gene therapy using a mitochondrial targeting sequence Leber hereditary optic neuropathy In the CLIN06 extension study, 3 years after treatment, 61 patients had a mean improvement against nadir of +20.5 letters equivalent and a mean improvement of +19.4 letters equivalent in the sham-treated eyes
Idorsia Ltd., of Allschwil, Switzerland Daridorexant Orexin receptor antagonist Insomnia Daridorexant 25 mg significantly improved sleep maintenance in a sleep lab by polysomnography and subjective total sleep time at home; results were statistically significant at month 1 and month 3; sleep onset and daytime functioning did not reach statistical significance
Immunomedics Inc., of Morris Plains, N.J. Trodelvy (sacituzumab govitecan-hziy) Antibody-drug conjugate targeting Trop-2 Metastatic triple-negative breast cancer without brain metastasis after at least 2 prior therapies for metastatic disease Trodelvy produced a progression-free survival of 5.6 months compared to 1.7 months for chemotherapy (p<0.0001); overall survival and objective response rate were also improved; data to be presented at an upcoming conference
Myovant Sciences Ltd., of Basel, Switzerland Relugolix Gonadotropin-releasing hormone receptor antagonist Heavy menstrual bleeding associated with uterine fibroids In the Liberty studies, relugolix plus estradiol and norethindrone acetate produced a 48.4-point reduction on the Bleeding and Pelvic Discomfort scale, compared to a 17.4-point reduction for placebo (p<0.0001); relugolix combination improved the physical and social activities on the RA scale by 45.1 points compared to a 15.8-point improvement for placebo (p<0.0001); 55.7% of patients had a >2-g/dL increase in hemoglobin levels from baseline to week 24 compared to 11.7% of patients on placebo (p<0.0001)
Obseva SA, of Geneva, and Boston Yselty (linzagolix) Gonadotropin-releasing hormone receptor antagonist Heavy menstrual bleeding due to uterine fibroids In the Primrose 1 study, at week 24, 75.5% of patients taking 200 mg linzagolix with add-back therapy (ABT) responded, defined as a menstrual blood loss of ≤80 mL and a ≥50% reduction from baseline, (p<0.001); responder rate for patients receiving 100 mg without ABT was 56.4% (p=0.003); pooled week 24 data from Primrose 1 and 2 showed a responder rate of 84.7% for linzagolix 200 mg with ABT and 56.6% for linzagolix 100 mg without ABT; in primrose 2, at 52 weeks, responder rates were 91.6% and 53.2% for 200 mg with ABT and 100 mg without ABT, respectively
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-CoV2 Double antibody cocktail targeting SARS-CoV-2 COVID-19 prophylaxis and treatment Started study in 2,000 uninfected people who have had close exposure to a COVID-19 patient; moved into phase III for the 2 adaptive phase I/II/III trials testing the cocktail in 1,850 hospitalized patients and 1,050 non-hospitalized patients with COVID-19
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Kevzara (sarilumab) Monoclonal antibody targeting IL-6 COVID-19 patients requiring mechanical ventilation Study didn't meet its primary composite endpoint; U.S.-based trial has been stopped, including a second cohort at a higher dose
Phase IV
Gilead Sciences Inc., of Foster City, Calif. Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide) HIV-1 integrase inhibitor; nucleoside reverse transcriptase inhibitor HIV infection In the study of 140 adults ages 65 and older, after 48 weeks, 92% of patients switched to Biktarvy maintained virologic suppression, defined as achieving HIV RNA<50 copies/mL


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