Amicus Therapeutics Inc., of Cranbury, N.J.
Recombinant human acid alpha-glucosidase (rhGAA) enzyme with an optimized carbohydrate structure (designated ATB200), administered with small-molecule pharmacological chaperone (designated AT2221)
Patients treated with AT-GAA for 24 months showed persistent and durable effects on 6-minute walk test distance and other measures of motor function and muscle strength, stability or increases in forced vital capacity, and reductions in biomarkers of muscle damage and disease substrate
Aslan Pharmaceuticals Ltd., of Singapore
Advanced solid tumors
Combined with FOLFIRI regimen, overall response rate was 13% and median progression-free survival was 4.2 months; durable responses were observed in a biliary tract cancer patient (1 prior line of chemotherapy) and a HER2-positive ovarian clear cell carcinoma patient (5 prior lines of chemotherapy), maintained at 7.5 and 6.1 months. respectively
Athira Pharma Inc., of Seattle
Enhances activity of hepatocyte growth factor and its receptor, MET
Equillium Inc., of San Diego
Anti-CD6 monoclonal antibody
IGM Biosciences Inc., of Mountain View, Calif.
CD20 x CD3 bispecific IgM antibody
Relapsed/refractory B-cell non-Hodgkin lymphoma
Reneuron Group plc, of London
Human retinal progenitor cell therapy
Improvements seen from baseline in visual acuity as measured by the number of letters read on the ETDRS chart; observed efficacy was rapid and profound in the first 3 patients in phase IIa segment of the study
Scholar Rock Inc., of Cambridge, Mass.
Selective inhibitor of the activation of myostatin
Type 2 and type 3 spinal muscular atrophy
Well-tolerated with no apparent safety signals and no dose-limiting toxicities identified up to the highest evaluated dose of 30 mg/kg; robust and sustained target engagement
Arqule Inc., of Burlington, Mass.
Proteus syndrome and PIK3CA-related overgrowth spectrum disorders
Treated first of 30-35 patients in the Mosaic study measuring the objective response to the drug
Biogen Inc., of Cambridge, Mass.
Antisense oligonucleotide targeting SMN
Spinal muscular atrophy
In the Nurture study, which treated infants in the pre-symptomatic stage before 6 weeks old that was published in Neuromuscular Disorders, patients were 25 months or older, alive and didn't need permanent ventilation; all patients could sit independently and 88% could walk independently; mean Hammersmith Infant Neurologic Examination, Section 2 score for both those with 2 or 3 SMN2 copies approached the maximum score of 26 points at the last assessment
Nicox SA, of Sophia Antipolis, France
Nitric oxide-donating bimatoprost analog
Open-angle glaucoma or ocular hypertension
NCX-470 lowered intraocular pressure from baseline by 7.6-9.8 mmHg as measured 3 times during day 28 compared to a reduction of 6.3-8.8 mmHg for latanoprost (p<0.025); plans to run a phase III study
PTC Therapeutics Inc., of South Plainfield, N.J., and Roche Holding AG, of Basel Switzerland
Modifies SMN2 mRNA splicing
Spinal muscular atrophy
In the Firefish study, after 16 months, 82% of the 17 high-dose patients had CHOP-INTEND score ≥40; 86% were event-free; in the Sunfish study, blood SMN protein levels increased by a median of 2-fold after 4 weeks of treatment; total MFM32 score, including a broad range of ages and functional status at baseline, improved compared with natural history; in the Jewelfish study, 45 patients had a greater than 2-fold increase in median SMN protein vs. baseline over 12 months
Restorbio Inc., of Boston
Inhibits the TORC1 pathway
Respiratory tract infection
Drug up-regulated interferon-stimulated antiviral gene expression in whole blood compared to placebo (p=0.00001) and decreased incidence of RTIs; a post-hoc analysis found time to alleviation of moderate to severe RTI symptoms was reduced by an average of 5 days (p=0.025) and the rate of all-cause hospitalization was reduced by 56% (p=0.047)
Theragnostics Ltd., of London
Gallium-68 (68Ga) THP-PSMA PET/CT
In the 50-patient Pronounced study, 1/3 of newly diagnosed prostate cancer patients and over 50% of patients with biochemically recurrent disease, including 75% in a post-radical radiotherapy setting, had their treatment plan altered to be more appropriate as a result of re-staging their prostate cancer
Xbiotech Inc., of Austin, Texas
Monoclonal antibody targeting interleukin-1 alpha
Enrolled first patient in study measuring disease severity using a combination of rheumatological, clinical and physiological measures over 12 weeks; study also includes an open-label extension from weeks 13-24
Contrafect Corp., of Yonkers, N.Y.
Staphylococcus aureus bacteremia, including right-sided endocarditis
At an end-of-phase-II meeting, the company obtained concurrence with the FDA for a single phase III study to support a BLA; study will enroll about 350 patients, measuring clinical response at day 14
Heron Therapeutics Inc.. of San Diego
Fixed-dose combination of local anesthetic bupivacaine and nonsteroidal anti-inflammatory drug meloxicam
Postoperative pain after total knee arthroplasty
Data in phase IIIb trial showed mean pain scores remained in the mild range through 72 hours post-surgery; median use of opioids was 4 to 5 pills of oxycodone through 72 hours; 75% of patients were discharged from the hospital without a prescription for opioids
Novartis AG, of Basel, Switzerland
Monoclonal antibody targeting interleukin-17A
Non-radiographic axial spondyloarthritis
The Prevent study met its primary endpoint of ASAS40 at week 52; data to be presented at a future scientific congress
Ra Pharmaceuticals Inc., of Cambridge, Mass.
Macrocyclic peptide targeting complement 5
Generalized myasthenia gravis
Dosed first of about 130 patients in the Raise study measuring the MG Activities of Daily Living score from baseline to week 12; top-line data expected in early 2021
For more information about individual companies and/or products, see Cortellis.