Company Product Description Indication Status
Phase I
Applied Genetic Technologies Corp., of Alachua, Fla. AGTC-501 Adeno-associated virus-based gene  X-linked retinitis pigmentosa  At 6 months, the treatment increased mean sensitivity relative to baseline as assessed by microperimetry, which was durable through month 12; 10 of 15 evaluable patients in groups 2, 4, 5 and 6 that meet inclusion criteria for the planned Vista phase II/III trial had robust and durable signs of improvements in visual sensitivity through month 6 for groups 5 and 6 and through month 12 for groups 2 and 4
Applied Genetic Technologies Corp., of Alachua, Fla. AGTC-401 Adeno-associated virus-based gene  Achromatopsia  Case study of 2 patients in the ACHMB3 trial and 1 patient in the ACHMA3 trial showed the treatment improved visual sensitivity and photosensitivity in the patients
Cabaletta Bio Inc., of Philadelphia DSG3-CAART Chimeric autoantibody receptor T cells Mucosal-dominant pemphigus vulgaris In the first cohort of patients there were no clinically relevant adverse events, including cytokine release syndrome or neurotoxicity, during the 8-day acute safety window; currently enrolling patients in the second cohort at a treatment dose of 100M DSG3-CAART cells
Denali Therapeutics Inc., of South San Francisco, and Biogen Inc., of Cambridge, Mass. BIIB-122/DNL-151 LRRK2 inhibitor Parkinson’s disease  In the phase I study in 184 healthy volunteers and the phase Ib study of 36 patients, the drug produced dose-dependent reduction of the lysosomal lipid BMP in urine; there was evidence of target and pathway engagement by the drug as measured by pS935 LRRK2 and pT73 Rab10, respectively
Lineage Cell Therapeutics Inc., of Carlsbad, Calif. Opregen Allogeneic retinal pigment epithelium cells Dry age-related macular degeneration In cohort 4 of the phase I/IIa study, 10 of 12 treated eyes were at or above baseline visual acuity at their last assessment 4.5 months to approximately 3 years post-transplant; best corrected visual acuity improved by up to 19 letters; 10/12 untreated eyes were below pre-treatment baseline values at the same time points
Regulus Therapeutics Inc., of La Jolla RGLS-4326 Oligonucleotide inhibitor of miR-17 Autosomal dominant polycystic kidney disease In the first cohort of 9 patients, mean PC1 biomarker increased by more than 50% from baseline to day 71; mean PC2 levels increased by more than 20%, which wasn’t statistically significant
Repare Therapeutics Inc., of Cambridge, Mass. RP-6306 Targets PKMYT1 CCNE1-amplified, FBXW7-altered and other PKMYT1 inhibitor-sensitive cancers Treated first of approximately 70 patients in the study designed to test the safety and tolerability of RP-6306 and to establish the recommended phase II dose and dosing schedule
Phase II
Jcyte Inc., of Newport Beach, Calif. Jcell Human retinal progenitor cells  Retinitis pigmentosa People in target population treated in phase IIb study with single 6-million-cell dose had early and sustained mean improvement in primary endpoint of BCVA of +16.27 letters vs. sham +1.85 letters from baseline to final 12 month study visit (p=0.003)
Sarepta Therapeutics Inc., of Cambridge, Mass. SRP-5051
DMD gene modulator
Duchenne muscular dystrophy  Part A of Momentum study showed monthly dose of 30 mg/kg delivered mean exon skipping of 10.79% and mean dystrophin expression of 6.55%, 18x the exon skipping and 8x the dystrophin production as eteplirsen, dosed weekly for 24 weeks
Sunovion Pharmaceuticals Inc., of Marlborough, Mass. SEP-363856 Trace amine-associated receptor agonist with serotonin 5-HT1A agonist activity Schizophrenia Data from open-label extension study showed short-term treatment was associated with improvement relative to placebo in negative symptoms of schizophrenia as assessed by the Brief Negative Symptom Scale (BNSS) total score and BNSS factor scores; long-term treatment was associated with additional mean improvement from baseline to week 26
Phase III
Adverum Biotechnologies Inc., of Redwood City, Calif. ADVM-022 (AAV.7m8 vector encoding aflibercept) VEGF gene inhibitor Wet age-related macular degeneration Long-term data from Optic trial showed 60% of participants (9/15) were injection-free beyond 1 year following 2 x 10^11 vg/eye dose and 87% (13/15) were injection free following 6 x 10^11 vg/eye dose
Aeglea Biotherapeutics Inc., of Austin, Texas Pegzilarginase Arginase-I stimulator Arginase 1 deficiency Randomization for pivotal Peace trial completed, with top-line data expected in fourth quarter of 2021
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Oxlumo (lumasiran) Hydroxyacid oxidase 1 modulator Primary hyperoxaluria type 1 12-month analysis of Illuminate-A study showed, among those with valid renal ultrasounds at baseline (n=24), 46% (11/24) improved in nephrocalcinosis grade relative to baseline, 17% (4/24) remained stable and 13% (3/24) worsened; of 14 with baseline nephrocalcinosis and available ultrasounds, 79% (11/14) improved relative to baseline and 73% (8/11) of these improved in both kidneys
Bristol Myers Squibb Co., of New York Mavacamten Myosin inhibitor Obstructive hypertrophic cardiomyopathy Interim results from ongoing 5-year Mava-LTE extension study of Explorer-HCM trial showed durable improvement in left ventricular outflow tract gradients, diastolic function N-terminal-pro hormone B-type natriuretic peptide and symptoms
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Peginterferon lambda Interferon receptor modulator COVID-19 Additional arm of up to 800 high-risk people will be added to ongoing Together platform study to assess agent in outpatients with infection; primary endpoint is reduction in ER visits and hospitalizations vs. placebo
Eli Lilly and Co., of Indianapolis Taltz (ixekizumab) Monoclonal antibody targeting interleukin 17A Plaque psoriasis In 1-year network meta-analysis based on AUC, Taltz showed numerically greater cumulative benefits on completely clear skin vs. 7 other biologics, measured by PASI 100, providing 159 cumulative days (95% credible interval, 147.4-170 days) or 23 weeks of PASI 100 
Novan Inc., of Durham, N.C. SB-206 (berdazimer sodium) Topical antiviral gel Molluscum contagiosum Final participant completed last week-12 visit in pivotal B-Simple4 study; top-line results targeted by end of second quarter of 2021
Novartis AG, of Basel, Switzerland Beovu (brolucizumab-dbll) VEGF-A ligand inhibitor Diabetic macular edema 1-year results from Kestrel and Kite studies showed Beovu, dosed at 6 mg, met primary endpoints of noninferiority in change in BCVA from baseline vs. aflibercept 2 mg; 3-mg arm, tested only in Kestrel, did not meet primary endpoint
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373 COVID-19 spike glycoprotein modulator COVID-19 Pediatric expansion initiated in ongoing pivotal Prevent-19 trial and set to enroll up to 3,000 people age 12 to 17
Sandoz, unit of Novartis AG, of Basel, Switzerland Aflibercept biosimilar Dual VEGF-A/VEGF-B ligand inhibitor Wet age-related macular degeneration First participant expected to enroll shortly in confirmatory Mylight trial vs. Eylea (aflibercept, Bayer AG/Regeneron Pharmaceuticals Inc.); primary endpoint is mean change in BCVA score from baseline to week 8
Seqirus USA Inc., of Summit, N.J. Flucelvax Quadrivalent  Cell-based quadrivalent seasonal influenza vaccine Seasonal influenza Vaccine was as safe and immunogenic as standard quadrivalent seasonal influenza vaccine in children 6 months through <4 years of age during U.S. 2019/20 influenza season
Sunovion Pharmaceuticals Inc., of Marlborough, Mass. Latuda (lurasidone) 5-HT/dopamine D2 antagonist  Bipolar depression Post-hoc analysis of pooled data from 2 placebo-controlled studies showed treatment improved psychic anxiety from baseline to week 6 both as monotherapy (p<0.001 for 20-60 mg/d; p<0.01 for 80-120 mg/d) and as adjunctive therapy with lithium/valproate (p<0.01); treatment also associated with improvement in somatic anxiety from baseline to week 6 as adjunctive therapy with lithium/valproate (p<0.01); numeric improvement in somatic anxiety observed for monotherapy treatment (p=0.09 for 20-60 mg/d; p=NS for 80-120 mg/d)
Xenothera SAS, of Nantes, France XAV-19 Anti-SARS-CoV-2 polyclonal antibody COVID-19 Started Eurovax study to recruit 722 patients with moderate COVID-19, hospitalized or monitored remotely; trial will take place in Greece, Bulgaria, Romania, Spain and Turkey
Zealand Pharma A/S, of Copenhagen, Denmark Dasiglucagon Glucagon analogue Severe hypoglycemia in diabetes Data published in Diabetes Care showed administration resulted in reversal of hypoglycemia (with a median recovery time of 10 minutes) with 99% of trial participants reaching recovery within 15 minutes
Phase IV
Mallinckrodt plc, of Dublin Acthar Gel (repository corticotropin injection) Melanocortin receptor agonist; adrenocorticotrophic hormone ligand Non-infectious keratitis Data from study in adults with treatment-resistant, severe disease showed after 12 weeks of treatment, 50% experienced improvements in symptom bother score by at least 12 points using the Impact of Dry Eye on Everyday Life patient-reported outcome assessment

Notes

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