Company Product Description Indication Status
Phase I

Assembly Biosciences Inc., of South San Francisco

ABI-H2158

Hepatitis B structural protein modulator

Hepatitis B virus infection

Interim data from phase Ib study, currently enrolling HBeAg-positive patients in sequential dose cohorts, showed antiviral activity from initial dose level of 100 mg, which produced mean declines of 2.3 log10 [range 1.7 – 3] and 2.1 log10 [range 1.5 - 2.7] from baseline to day 15 in HBV DNA and pgRNA, respectively

Celecor Therapeutics Inc., of San Diego

RUC-4

Platelet GPIIb/IIIa inhibitor

ST-elevation myocardial infarction

Study met primary endpoint; 0.075-mg/kg subcutaneous dose provided rapid (<15 minute following administration), intense (>80%) and sustained (<2 hours) inhibition of platelet aggregation

Celltrion Healthcare Co. Ltd., of Incheon, South Korea

CT-P13 SC (infliximab biosimilar)

TNF-alpha ligand inhibitor

Rheumatoid arthritis

In CT-P13 subcutaneous (SC) arm, mean serum concentration increased from week 6 and maintained generally consistent level from weeks 14 to 54 on biweekly injections; in those who switched from intravenous to SC at week 30, mean serum concentration gradually increased and also maintained consistent level to week 54, demonstrating noninferiority of SC formulation

Turning Point Therapeutics Inc., of San Diego

TPX-0046

RET tyrosine kinase receptor inhibitor

Advanced solid tumors

Initiated study expected to enroll about 50 TKI treatment-naïve and pretreated patients with RET-altered non-small-cell lung, thyroid and other advanced cancers in phase I dose-escalation portion and about 300 in multiple cohorts in phase II expansion

Phase II

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Lumasiran

Hydroxyacid oxidase 1 modulator

Hyperoxaluria

Open-label extension results at data cutoff of Sept. 12 showed 76% mean maximal reduction (range: 43%-91%) in urinary oxalate excretion relative to phase I/II baseline values in all cohorts (n=19) and relative to 82% mean maximal reduction (range: 62%-94%) after dosing across all cohorts (n=20)

Assembly Biosciences Inc., of South San Francisco

ABI-H0731

Hepatitis B structural protein inhibitor"

Hepatitis B virus infection

Final 24-week data from HBeAg-positive patients in 2 studies and interim data from ongoing open-label extension (OLE), where all participants receive study drug plus nucleos(t)ide analogues (Nrtl), showed 22/27 (81%) of 731+Nrtl treated patients among those with detectable DNA at baseline in study 201 achieved target not detected (TND) by week 24 vs. 0/12 (0%) who received Nrtl only (p<0.001); in study 202 (n=25), statistically significant reductions of pgRNA were observed by week 2 with 731+ETV combo (p<0.001); 21 of 25 patients from study 202 enrolled in OLE showed mean HBV DNA and pgRNA declines from baseline of 6.3 logs and 3 logs, respectively, at week 48

Cirius Therapeutics Inc., of San Diego

MSDC-0602K

Second-generation oral insulin sensitizer

Nonalcoholic steatohepatitis

12-month data from phase IIb Emminence study showed improvements in liver enzyme levels and markers of glycemic control, as well as dose-dependent trends for improvement in liver histology in patients with or without type 2 diabetes; data published in the Journal of Hepatology

Cocrystal Pharma Inc., of Bothell, Wash.

CC-31244

Hepatitis C virus NS5B polymerase inhibitor

Hepatitis C virus infection

U.S. phase IIa trial evaluating drug plus sofosbuvir/velpatasvir (Epclusa, Gilead Sciences Inc.) achieved primary endpoint; 8 of 12 (67%) participants with HCV achieved sustained virologic response 12 with 6 weeks of Epclusa combined with 2 weeks of CC-31244

Corbus Pharmaceuticals Holdings Inc., of Norwood, Mass.

Lenabasum

Cannabinoid CB2 receptor agonist

Dermatomyositis

With 18/20 (90%) of participants still enrolled in open-label extension at month 23, Cutaneous Dermatomyositis Activity and Severity Index activity score continued to improve, with mean change of -20.9 points from baseline; patient-reported outcomes, including disease activity, skin activity, itch, pain and effect on skin on functioning, symptoms and emotions, continued to improve or showed stable improvement from months 12 to 23

Corbus Pharmaceuticals Holdings Inc., of Norwood, Mass.

Lenabasum

Cannabinoid CB2 receptor agonist

Systemic sclerosis

With 29/36 (81%) of phase II participants enrolled in open-label extension at month 25, ACR CRISS score remained ≥ 0.95 from month 12 and mRSS improved > 9.2 points over same period; patient and physician global assessments of health, skin symptoms, itch, and patient-reported disability and function showed stabilization or continued improvement from months 12 through 25; mean forced vital capacity % predicted declined -2.0% from study start through latest data cut in September

Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium

Filgotinib

Oral JAK1 inhibitor

Moderately to severely active rheumatoid arthritis

Long-term data from phase IIb Darwin 3 study found, at week 156, 89.7%, 63% and 40% of patients in filgotinib monotherapy group achieved ACR20/50/70 responses, respectively; in filgotinib/methotrexate group, 87.2%, 72.4% and 45.5% achieved ACR20/50/70 responses, respectively

Mereo Biopharma Group plc, of London

Setrusumab (BPS-804)

Sclerostin inhibitor

Osteogenesis imperfecta

12-month top-line data showed dose-ranging phase IIb Asteroid study in adults with type I, III or IV disease missed primary endpoint of change in trabecular volumetric bone mineral density (vBMD) of the radius (wrist) vs. baseline, measured by high resolution peripheral quantitative computed tomography (HR-pQCT); secondary endpoint of increase in total vBMD at the wrist, measured by HR-pQCT of cortical and trabecular together, achieved statistical significance in medium and high-dose cohorts, with mean increases of 4.5% (p=0.028) and 4.11% (p=0.004), respectively

Myokardia Inc., of South San Francisco

Mavacamten

Myosin inhibitor

Hypertrophic cardiomyopathy

Maverick-HCM study confirmed safety and tolerability in those with non-obstructive disease but no statistically significant differences seen at 16 weeks in intent-to-treat population between active and placebo groups in exploratory endpoints, except for levels of biomarker NT-proBNP, which were reduced in those who received drug (p=0.004) vs. placebo

Promethera Biosciences SA, of Mont-Saint-Guibert, Belgium

Hepastem

Phenylalanine hydroxylase stimulator

Acute-on-chronic liver failure

Ongoing phase IIa study that recruited 24 participants met primary safety endpoint and showed improvement in Model for End Stage Liver Disease score, Child-Pugh score and bilirubin levels at 28 days and 3 months following treatment initiation

Roche Holding AG, of Basel, Switzerland

Gazyva (obinutuzumab)

CD20-targeting monoclonal antibody

Proliferative lupus nephritis

Nobility study met primary endpoint, with Gazyva plus standard of care demonstrating superiority vs. placebo plus standard of care; patients in the Gazyva arm showed increasing rates of complete renal response (CRR) from week 52 to week 76, with 40% achieving CRR, compared to 18% of patients in placebo group at week 76 (p=0.007); Gazyva met key secondary efficacy endpoints showing improved overall renal response and serologic markers of disease activity vs. placebo

Selecta Biosciences Inc., of Watertown, Mass.

SEL-212 (ImmTOR + pegadricase)

Uricase stimulator

Gout

When combination of pegadricase (0.2 mg/kg) and ImmTOR (0.1 or 0.15 mg/kg) was given in 3 cohorts across all 5 cycles, 66% of evaluable patients (21/32) maintained serum uric acid (sUA) levels below 6 mg/dL at week 20 after 5 monthly doses of SEL-212; 100% of those with sUA <6 mg/dL at 12 weeks maintained control through 20 weeks

Phase III

Akebia Therapeutics Inc., of Cambridge, Mass.

Vadadustat

HIF prolyl hydroxylase inhibitor

Anemia

As reported by partner Mitsubishi Tanabe Pharma Corp., of Osaka, Japan, 52-week data from correction and conversion pivotal study in 304 Japanese individuals with nondialysis-dependent chronic kidney disease (CKD) showed mean hemoglobin (Hb) level of 11.51 g/dL for drug vs. 11.58 g/dL for those who received darbepoetin alfa; 52-week data from conversion study in 323 Japanese hemodialysis participants with anemia due to CKD showed mean Hb level of 10.39 g/dL for vadadustat vs. 10.62 g/dL for darbepoetin alfa

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Lumasiran

Hydroxyacid oxidase 1 modulator

Hyperoxaluria

Initiated Illuminate-C study expected to enroll about 16 individuals with documented diagnosis of primary hyperoxaluria type 1; primary endpoint is percentage change in plasma oxalate from baseline to 6 months, with data expected late next year

Amarin Corp. plc, of Dublin

Vascepa (icosapent ethyl)

Apolipoprotein B antagonist; phospholipase A2 inhibitor

Cardiovascular disease

3,146 participants randomized in U.S. in global Reduce-IT cardiovascular outcomes trial showed 31% relative risk reduction and 6.5% absolute risk reduction in first occurrence of 5-point major adverse cardiovascular events; all prespecified hierarchical primary and secondary endpoints were reduced in U.S. subgroup, including myocardial infarction (8.8% to 6.7%, p=0.01), cardiovascular death (6.7% to 4.7%, p=0.007), stroke (4.1% to 2.6%, p=0.02) and all-cause mortality (9.8% to 7.2%, p=0.004)

Aslan Pharmaceuticals Pte. Ltd., of Singapore

Varlitinib

Pan-HER inhibitor

Biliary tract cancer

Pivotal TreeTopp study missed co-primary endpoints; median progression-free survival was 2.83 months for varlitinib in combination with capecitabine vs. 2.79 for control; overall response rate was 9.4% for varlitinib vs. 4.8% for control

Astrazeneca plc, of Cambridge, U.K., and Fibrogen Inc., of San Francisco

Roxadustat

HIF prolylhydroxylaseinhibitor

Anemia in chronickidney disease

Pooled analyses from phase III program showed, in nondialysis-dependent patients who received drug, risk of MACE, MACE+ and all-cause mortality was comparable to placebo; dialysis-dependent patients who received roxadustat had lower risk of MACE+ and no increased risk of MACE or all-cause mortality vs. epoetin alfa; in incident dialysis patients, those who received roxadustat had 30% lower risk of MACE and 34% lower risk of MACE+ vs. those who received epoetin alfa, with trend toward lower all-cause mortality for study drug

Fibrogen Inc., of San Francisco

Roxadustat

Oral inhibitor of HIF prolyl hydroxylase activity

Anemia in chronic kidney disease

Pooled analysis from 6 pivotal studies showed superiority to placebo in non-dialysis-dependent patients, showing improvement of 1.85 g/dL in Hb levels in baseline to average over 28-52 weeks vs. 0.14 g/dL for placebo, for overall difference of 1.72 g/dL (p<0.001); pooled analysis in dialysis patients showed superiority vs. epoetin alfa, with improvement of 1.22 g/dL in Hb levels from baseline to over 28-52 weeks vs. 0.99 g/dL, for overall treatment difference of 0.23 g/dL (p<0.0001); across both populations, effectively raised Hb levels regardless of iron depletion at baseline

Flexion Therapeutics Inc., of Burlington, Mass.

Zilretta (triamcinolone acetonide extended-release injectable suspension)

Activates glucocorticoid receptor

Moderate to severe osteoarthritis pain

Results from sensitivity analysis testing single administration showed significant improvement (p<0.05) in Average Daily Pain (ADP) scores vs. immediate-release triamcinolone acetonide in crystalline suspension (TAcs) (weeks 5-19) and placebo (weeks 1-20); proportion of patients reporting no knee pain (ADP score=0) at week 12 was higher with Zilretta (~28%) vs. TAcs (~8%)

Genentech, of South San Francisco, part of the Roche Group, and PTC Therapeutics Inc., of South Plainfield, N.J.

Risdiplam

Survival motor neuron-2 splicing modifier

Type 2 or 3 spinal muscular atrophy

Pivotal part 2 of Sunfish trial in patients, ages 2-25, met primary endpoint of change from baseline in Motor Function Measure 32 scale after 1 year of treatment vs. placebo

Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium

Filgotinib

Oral JAK1 inhibitor

Moderately to severely active rheumatoid arthritis

Subgroup analysis from Finch 2 trial in patients who previously had inadequate response to biologic DMARD therapy (DMARD-IR) showed both doses improved clinical outcomes vs. placebo, regardless of number and mechanism of action of prior bDMARDs; after 12 weeks of treatment, 70.3% treated with 200 mg and 58.2% treated with 100 mg achieved ACR20 score vs. 25.7% on placebo; 68% of bDMARD-IR patients in 200-mg group and 51.2% in 100-mg group who had been treated with more than 1 biologic MOA achieved ACR20 response vs. placebo (27.3%)

Horizon Therapeutics plc, of Dublin

Krystexxa (pegloticase injection

Pegylated uric acid specific enzyme

Chronic gout

Patient case series results showed that adding methotrexate to course of Krystexxa could help patients with disease refractory to conventional therapies; in 10 adults with uncontrolled gout who received Krystexxa during or with treatment of methotrexate, 8 were complete responders at 24 weeks

Janssen Pharmaceuticals Cos., of Beerse, Belgium, part of Johnson & Johnson

Invokana (canagliflozin)

SGLT2 inhibitor

Kidney disease

Analysis from Credence study found Invokana consistently reduced risk of renal and cardiovascular events in patients with various levels of kidney function, or estimated glomerular filtration rates (eGFR); greater renal absolute benefits were observed in those with most advanced renal insufficiency (eGFR <60 mL/min/1.73 m2)

Janssen Pharmaceuticals Cos., of Beerse, Belgium, part of Johnson & Johnson

Tremfya (guselkumab)

Monoclonal antibody against p19 subunit of IL-23

Active psoriatic arthritis

24-week data showed significant greater proportion of patients on Tremfya achieved at least 20% improvement in disease signs and symptoms (ACR20) vs. placebo; Discover-1 trial study showed 59% treated every 4 weeks (q4w) and 52% treated at weeks 0, 4 and every 8 weeks thereafter (q8w) achieved ACR20 vs. 22% on placebo (both p<0.001); Discover-2 showed 64% biologic-naïve patients receiving q4w or q8w achieved ACR vs. 33% on placebo (both p<0.001)

Notes

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