Phase I

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)

Pompe disease

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


Pan-HER inhibitor

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

Alzheimer's disease

Trial completed

Equillium Inc., of San Diego

Itolizumab (EQ-001)

Anti-CD6 monoclonal antibody

Lupus nephritis

Trial begun

IGM Biosciences Inc., of Mountain View, Calif.


CD20 x CD3 bispecific IgM antibody

Relapsed/refractory B-cell non-Hodgkin lymphoma

Trial begun

Reneuron Group plc, of London

hRPC therapy

Human retinal progenitor cell therapy

Retinitis pigmentosa

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

Phase II

Arqule Inc., of Burlington, Mass.


Pan-AKT inhibitor

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.

Spinraza (nusinersen)

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

Risdiplam (RG-7916)

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

Imaging agent

Prostate cancer

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

Phase III

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

Cosentyx (secukinumab)

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.

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