Amgen Inc., of Thousand Oaks, Calif.

Blincyto (blinatumomab)

Bispecific CD19-directed CD3 T-cell engager antibody

High-risk, B-cell acute lymphoblastic leukemia at first relapse

Study met primary endpoint of event-free survival compared to conventional consolidation chemotherapy; enrollment terminated early on recommendation from the independent data monitoring committee (DMC) based on positive Blincyto efficacy; a separate phase III study run by the Children's Oncology Group has closed accrual for high-risk and intermediate-risk patients based on a strong trend toward improved disease-free survival and overall survival, markedly lower toxicity and better minimal residual disease clearance for Blincyto compared to chemotherapy; DMC recommended continuing enrollment of low-risk patients


Amgen Inc., of Thousand Oaks, Calif., and Genmab A/S, of Copenhagen

Darzalex (daratumumab)

Monoclonal antibody targeting CD38

Relapsed or refractory multiple myeloma

In the Candor study, Darzalex (daratumumab) plus Kyprolis and dexamethasone (KdD) produced a 37% reduction in the risk of progression or death compared to Kyprolis and dexamethasone (Kd)(p=0.0014); median progression-free survival was 15.8 months for Kd and hasn't been reached for KdD


Astrazeneca plc, of Cambridge, U.K.

Imfinzi (durvalumab)

PD-L1 inhibitor

Small-cell lung cancer

Caspian trial found that, in combination with 4 cycles of standard-of-care (SOC) chemotherapy, risk of death was reduced by 27%, with median overall survival of 13 months for Imfinzi plus chemotherapy vs. 10.3 months for SOC; estimated 33.9% of patients alive at 18 months following treatment with Imfinzi plus chemo vs. 24.7% of patients following SOC


Astrazeneca plc, of Cambridge, U.K.

Imfinzi (durvalumab)

Monoclonal antibody targeting PD-L1

Previously untreated extensive-stage small-cell lung cancer

Risk of death was reduced by 27% with a median overall survival of 13 months for Imfinzi plus chemotherapy compared to 10.3 months for standard of care (SOC); 18-month OS was 33.9% vs. 24.7%, 12-month progression-free survival was 17.5% vs. 4.7%, objective response rate was 67.9% vs. 57.6% and 12-month duration of response was 22.7% vs. 6.3% for Imfinzi plus chemotherapy and SOC, respectively


Aveo Oncology Inc., of Cambridge, Mass.

Fotivda (tivozanib)

Vascular endothelial growth factor tyrosine kinase inhibitor

Refractory metastatic renal cell carcinoma

Updated data from the Tivo-3 study showed a hazard ratio of 0.99 for overall survival of Fotivda compared to Nexavar (sorafenib, Bayer AG)


Bristol-Myers Squibb Co., of Princeton, N.J.

Opdivo (nivolumab)

Monoclonal antibody inhibiting PD-1

Glioblastoma multiforme

Checkmate-548 trial in combination with standard of care (SOC) vs. SOC alone did not meet 1 of its primary endpoints, progression-free survival, in patients with newly diagnosed GBM that is O6-methylguanine-DNA methyltransferase-methylated; data monitoring committee recommended the trial continue as planned to allow data for other primary endpoint, overall survival, to mature


Bristol-Myers Squibb Co., of Princeton, N.J.

Opdivo (nivolumab)

PD-1-targeting antibody

Advanced non-small-cell lung cancer

Long-term pooled results from Checkmate-017 and Checkmate-057 studies in patients with previously treated disease showed that, at 5 years, those treated with Opdivo continue to experience long-term overall survival benefit vs. docetaxel (13.4% vs. 2.6%); OS benefit was observed across all subgroups; among patients with objective response to Opdivo, 32.2% continued to see response at 5 years vs. 0% with objective response to docetaxel; median duration of response was 19.9 months for Opdivo vs. 5.6 months for docetaxel


Celgene Corp., of Summit, N.J.


Cytidine nucleoside analogue

Newly diagnosed acute myeloid leukemia

In the 472-patient Quazar AML-001 study, maintenance treatment with CC-486 produced a highly statistically significant improvement in overall survival compared to placebo; improvement in relapse-free survival on CC-486 was also statistically significant compared to placebo; data to be presented at a future medical meeting; regulatory submissions planned for the first half of 2020


Genentech Inc., of South San Francisco, a unit of the Roche Group

Tecentriq (atezolizumab)

PD-L1-targeting antibody

Advanced nonsquamous and squamous non-small-cell lung cancer

Data showed Impower110 study testing Tecentriq as first-line monotherapy vs. cisplatin or carboplatin and pemetrexed or gemcitabine in patients without ALK or EGFR mutations met primary endpoint in interim analysis, demonstrating statistically significant overall survival benefit in patients with high PD-L1 expression vs. chemotherapy alone; study will continue to final analysis for people with lower levels of PD-L1 expression


Halozyme Therapeutics Inc., of San Diego, and Genentech Inc., of South San Francisco, a member of the Roche Group

Fixed-dose combination of Perjeta (pertuzumab) and Herceptin (trastuzumab)

HER2-targeting antibodies formulated for subcutaneous administration using Enhanze

HER2-positive early breast cancer

Federica study in combination with intravenous therapy showed noninferior levels of Perjeta in blood compared to standard intravenous infusion of Perjeta plus Herceptin and chemotherapy


Innate Pharma SA, of Paris, and Astrazeneca plc, of Cambridge, U.K.


Immune checkpoint inhibitor targeting NKG2A receptors on tumor-infiltrating cytotoxic CD8+ T cells and NK cells

Recurrent or metastatic squamous cell carcinoma of the head and neck

Astrazeneca will advance into randomized trial testing combination with cetuximab; study set to start in 2020


Innovent Biologics Inc., of Suzhou, China


Biosimilar of anti-VEGF antibody bevacizumab

Advanced non-small-cell lung cancer

Results of study vs. bevacizumab, both in combination with paclitaxel/carboplatin, in first-line treatment showed, by cut-off date of March 31, overall response rates were 47.1% and 46.8% for IBI-305 and bevacizumab in full analysis set, respectively; ORR rate was 1.01, within predefined equivalence margin; investigator-evaluated median progression-free survival was 7.3 months and 7.5 months in the IBI-305 and bevacizumab groups, respectively, with no statistical difference (p=0.893)


Innovent Biologics Inc., of Suzhou, China


Rituximab biosimilar

Diffuse large B-cell lymphoma

2 trials have shown the pharmacokinetic equivalence of IBI-301 with rituximab in CD20+ B-cell lymphoma patients who achieved complete response (CR)/CR unconfirmed after treatment, and the safety and tolerance similarity of both drugs


Merck & Co. Inc., of Kenilworth, N.J.

Keytruda (pembrolizumab)

Anti-PD-1 antibody

Advanced nonsquamous and squamous non-small-cell lung cancer

First-line treatment in combination with chemotherapy in pooled analysis from 3 trials of subgroup of 428 patients whose tumors do not express PD-L1 showed combo reduced risk of death by 44% vs. chemo alone; Keytruda-chemo combo reduced risk of disease progression or death by 33% vs. chemo alone; objective response rate was 46.9% for patients in combo arm vs. 28.6% for patients in chemo-only arm


Rafael Pharmaceuticals Inc., of Cranbury, N.J.

CPI-613 (devimistat)

Targets mitochondrial tricarboxylic acid cycle

Metastatic adenocarcinoma of the pancreas

Expanded Avenger 500 trial into France; pivotal study is evaluating CPI-613 in combination with modified FOLFIRINOX as first-line therapy


Rafael Pharmaceuticals Inc., of Cranbury, N.J.

CPI-613 (devimistat)

Targets mitochondrial tricarboxylic acid cycle

Relapsed or refractory acute myeloid leukemia

Trial in older patients now active and enrolling in South Korea; study testing efficacy and safety in combination with high-dose cytarabine and mitoxantrone


Tocagen Inc., of San Diego

Toca-511 and Toca-FC

Vocimagene amiretrorepvec and extended-release formulation of 5-fluorocytosine


Missed the primary endpoint of OS vs. standard of care treatment (11.1 months median vs. 12.2 months, HR=1.06, p=0.6154); Missed secondary endpoints too


Tyme Technologies Inc., of New York

SM-88 (racemetyrosine)

Cancer metabolism-based therapy

Metastatic pancreatic cancer

Started pivotal stage of Tyme-88-Panc trial


Urogen Pharma Ltd., of New York


Sustained-release mitomycin C gel

Ureter cancer

Pivotal Olympus trial showed UGN-101 demonstrated a 59% complete response rate in patients with low-grade upper tract urothelial cancer; estimated median time to recurrence was 13 months



Applied Therapeutics Inc., of New York


Aldose reductase inhibitor

Diabetic cardiomyopathy

Started the Arise-HF study comparing 2 doses of the drug to placebo in about 675 patients at high risk of progression to overt heart failure; primary endpoint is peak VO2 over 15 months; percent of patients progressing to overt heart failure, quality of life metrics (modified KCCQ), echocardiographic measurements and cardiac stress biomarkers, including NtproBNP, will also be measured


Astrazeneca plc, of Cambridge, U.K.

Brilinta (ticagrelor)

P2Y12 receptor antagonist

Coronary artery disease and type 2 diabetes with no prior heart attack or stroke

In the Themis study, Brilinta plus aspirin reduced the relative risk for the composite of cardiovascular death, heart attack or stroke by 10% compared with aspirin alone; in patients who had undergone a percutaneous coronary intervention, Brilinta plus aspirin reduced the risk by 15% compared to aspirin alone


Astrazeneca plc, of Cambridge, U.K.

Farxiga (dapagliflozin)

SGLT2 inhibitor

Heart failure and reduced ejection fraction

In the DAPA-HF study, Farxiga reduced the composite of cardiovascular (CV) death or worsening of heart failure by 26% (p<0.0001); the risk of experiencing a first episode of worsening heart failure was reduced by 30% (p<0.0001) and the risk of CV death was reduced by 18% (p=0.0294)


Biocardia Inc., of San Carlos, Calif.

Cardiamp cell therapy

Autologous bone marrow cells delivered via catheter-based procedure

Heart failure

Independent data safety monitoring board (DSMB) completed prespecified data review for pivotal trial and recommended study continue as planned; primary endpoint is 6-minute walk distance 12 months post-treatment; next DSMB review is anticipated in first quarter of 2020


Resverlogix Corp., of Calgary, Alberta

Apabetalone (RVX-208)

Small-molecule selective BET inhibitor

Cardiovascular disease

Following database lock, Betonmace top-line data are expected on or about Sept. 30, 2019


Resverlogix Corp., of Calgary, Alberta


Small-molecule BET inhibitor

Cardiovascular disease

Betonmace trial did not meet primary endpoint of reducing major adverse cardiovascular events, defined as cardiovascular death, non-fatal myocardial infarction and stroke, when added to standard of care in high-risk patients with type 2 diabetes, recent acute coronary syndrome and low HDL cholesterol


The Medicines Co., of Parsippany, N.J.


Cholesterol-lowering siRNA drug

Atherosclerotic cardiovascular disease

Orion-10 study met all primary and secondary endpoints, with durable and potent efficacy and safety at least as favorable as observed in Orion-11 trial; regulatory submissions expected in the U.S. in the fourth quarter of 2019 and in Europe in the first quarter of 2020


The Medicines Co., of Parsippany, N.J.


Cholesterol-lowering siRNA drug

Heterozygous familial hypercholesterolemia

Orion-9 study met all primary and secondary endpoints, demonstrating durable and potent efficacy; well-tolerated, with no treatment-related liver or renal laboratory anomalies; regulatory submissions expected in the U.S. in the fourth quarter of 2019 and in Europe in the first quarter of 2020



Aclaris Therapeutics Inc., of Wayne, Pa.

A-101 45% topical solution

High concentration of topical hydrogen peroxide solution

Common warts (verruca vulgaris)

Thwart-2 study met primary endpoint (p<0.0001), with higher proportion of subjects having all identified common warts reported as clear at day 60 vs. placebo; all secondary endpoints achieved statistical significance, including complete clearance at day 137 (p=0.0001), mean per subject percent of treated warts cleared at day 137 (p<0.0001) and time to complete clearance of all warts (p<0.0001); data from second trial, Thwart-1, expected in fourth quarter of 2019


Incyte Corp., of Wilmington, Del.

Ruxolitinib cream

JAK1/JAK2 inhibitor


Treated first patient in the True-V trial program that includes 2 studies of about 300 patients each; primary endpoint of both studies is proportion of patients achieving F-VASI75, defined as at least a 75% improvement from baseline in the F-VASI score at week 24


Lipidor AB, of Stockholm


Spray formulation of vitamin D derivative


Last patient enrolled in trial comparing drug with a marketed calcipotriol ointment; results expected to be presented in December


Mallinckrodt plc, of Staines-upon-Thames, U.K.


Regenerative tissue

Deep partial-thickness thermal burns

Top-line results showed pivotal trial met both primary endpoints; data showed significantly smaller area of burn wounds treated with Stratagraft required autografting by 3 months vs. those treated exclusively with autograft (p<0.0001); proportion of Stratagraft-treated wounds achieved durable wound closure at 3 months, exceeding predefined threshold for statistical significance; BLA expected to be submitted in second half of 2020


Pfizer Inc., of New York


Oral JAK1 inhibitor

Moderate to severe atopic dermatitis

Top-line data from Jade Mono-2, the second pivotal study, testing monotherapy in patients 12 and older showed that by week 12, percentage of patients achieving each co-primary endpoint and each key secondary endpoint with either dose was statistically significant vs. placebo; in addition, a statistically significant number of patients achieved reduction in pruritus by week 2, as measured by 4-point or larger reduction in itch severity using pruritus numerical rating scale



Acasti Pharma Inc., of Laval, Quebec


Omega-3 phospholipid

Severe hypertriglyceridemia

Both Trilogy studies have achieved 100% randomization, and nearly 80% of patients in both trials combined have completed 6-month plans; on track to report top-line Trilogy 1 results in December 2019 and Trilogy 2 results in January 2020


Allena Pharmaceuticals Inc., of Newton, Mass.


Oral, recombinant oxalate-degrading enzyme

Enteric hyperoxaluria

Completed enrollment in pivotal Urirox-1 trial; top-line data expected in fourth quarter of 2019


Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Onpattro (patisiran)

RNAi targeting transthyretin

Polyneuropathy of hereditary ATTR amyloidosis

Biomarker analysis of the Apollo study showed 66 proteins with changes after treatment with Onpattro; neuroaxonal damage marker neurofilament light chain (NfL) had the greatest statistical significance (p=3.95×10^-21) for change relative to placebo over the 18-month study period; there was a correlation between NfL reduction and improvement in mNIS+7 score


Alnylam Pharmaceuticals Inc., of Cambridge, Mass.


RNAi therapeutic targeting aminolevulinic acid synthase 1

Acute hepatic porphyria

In the Envision study, a post-hoc analysis using non-parametric stratified Wilcoxon test found patients on givosiran had significant reduction in daily worst pain (nominal p=0.0455); in exploratory analyses, reductions in pain were accompanied by fewer days of use of both opioid and non-opioid analgesics; givosiran did not impact daily worst fatigue or daily worst nausea at 6 months; in open-label extension (OLE), reduction in composite porphyria attack rate and reduction in levels of aminolevulinic acid (ALA) continued in patients receiving givosiran; placebo patients who crossed over had a rapid and sustained lowering of attack rate and ALA levels; in the phase I/II OLE, mean reductions in annualized attack rate and in annualized hemin use of greater than 90% were observed


Novartis AG, of Basel, Switzerland

Entresto (sacubitril/valsartan)

Neprilysin inhibitor and an angiotensin II receptor blocker

Heart failure patients with preserved ejection fraction

In the 4,822-patient Paragon-HF study, Entresto reduced the composite primary endpoint of total (first and recurrent) heart failure hospitalizations and cardiovascular death by 13% compared to valsartan (p=0.059); total heart failure hospitalizations were reduced by 15% (p=0.056)


Novo Nordisk A/S, of Bagsvaerd, Denmark


Fast-acting insulin aspart

Advanced type 2 diabetes

Data showed that, for people not optimally controlled on basal-bolus regimen, Fiasp reduced overall blood sugar levels vs. conventional insulin aspart; patients on Fiasp also reported superior reduction in 1-hour post-mean blood sugar increment vs. those on conventional insulin aspart; Fiasp showed statistically significantly lower rate of severe or blood sugar-confirmed hypoglycemia vs. conventional insulin aspart


Novo Nordisk A/S, of Bagsvaerd, Denmark

Ozempic (once-weekly semaglutide)

GLP-1 analogue

Type 2 diabetes

Results from 2 head-to-head trials showed Ozempic was superior to SGLT2 inhibitor canagliflozin in reducing HbA1c and body weight in people uncontrolled on metformin in Sustain 8 trial and superior to Victoza (liraglutide) in reducing HbA1C and body weight in patients in Sustain 10 study; data published in The Lancet Diabetes & Endocrinology


Novo Nordisk A/S, of Bagsvaerd, Denmark

Oral semaglutide

GLP-1 analogue

Type 2 diabetes

Exploratory analyses from phase IIIa Pioneer program showed 3-mg, 7-mg and 14-mg doses improved glycemic control across baseline HbA1c levels; greater reductions in HbA1c were shown with 7-mg and 14-mg doses vs. all comparators, including placebo, Jardiance (empagliflozin 25 mg), Januvia (sitagliptin 100 mg) or Victoza (liraglutide 1.8 mg)


Poxel SA, of Lyon, France


Targets mitochondrial bioenergetics

Type 2 diabetes

Data showed Times 1 trial in Japanese patients achieved statistical significance (p<0.0001) for primary endpoint, defined as change in HbA1c level vs. placebo at week 24, with HbA1c placebo-corrected mean change from baseline of 0.87%; achieved statistical significance on main secondary endpoint of decrease from baseline in fasting plasma glucose (FPG) vs. placebo at week 24 (p<0.0001), with FPG placebo-corrected mean change from baseline of -19 mg/dL


Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Farmaceutici SpA, of Parma, Italy

PRX-102 (pegunigalsidase alfa)

Alpha-galactosidase stimulator

Fabry disease

Completed enrollment of 24-month Balance trial evaluating drug compared to agalsidase beta (Fabrazyme, Sanofi SA/Genzyme Corp.) on renal function in participants with Fabry and progressing kidney disease previously treated with agalsidase beta; outcome measures include rate of renal function deterioration as well as cardiac assessment, Lyso-Gb3, pain, quality of life, immunogenicity and clinical events


Strongbridge Biopharma plc, of Dublin

Recorlev (levoketonconazole)

Cortisol synthesis inhibitor

Endogenous Cushing's syndrome

Data showed Sonics study, published in The Lancet, met primary endpoint, with 30% of patients achieving mean urinary free cortisol normalization at end of maintenance phase among intent-to-treat population, without a dose increase (p=0.015 vs. null hypothesis of ≤20%


The Medicines Co., of Parsippany, N.J.


siRNA targeting PCSK9

Atherosclerotic cardiovascular disease and familial hypercholesterolemia

In the Orion-11 study, inclisiran produced placebo-adjusted LDL-C reductions of 54% at day 510 and time-averaged placebo-adjusted LDL-C reductions of 50% from days 90 through 540 (p<0.0001 for both)


Zealand Pharma A/S, of Glostrup, Denmark


Glucagon receptor agonist; glucagon ligand


Median time to blood glucose recovery was 10 minutes for dasiglucagon vs. 30 minutes for placebo (p<0.001); median time to recovery for Glucagen was 10 minutes



Galmed Pharmaceuticals Ltd., of Tel Aviv, Israel


Stearoyl CoA desaturase-1 inhibitor

Nonalcoholic steatohepatitis; fibrosis

Initiated global phase III/IV Armor study expected to randomize about 2,000 participants 2-to-1 to drug at 300 mg BID or matching placebo; in first part, 1,200 participants will be treated for 52 weeks, with histology-based data expected to serve as basis to submit MAA under accelerated/conditional approval; in second part, all participants will continue with treatment assignment to study completion to confirm efficacy


Ritter Pharmaceuticals Inc., of Los Angeles


Stimulates growth of lactose-metabolizing bacteria in the colon

Lactose intolerance

Study failed to demonstrate statistical significance in prespecified primary endpoint, measuring lactose intolerance symptom reduction at day 61; treatment group reported 3.159 mean reduction vs. reported 3.42 mean reduction in placebo group (p=0.106); also missed first secondary endpoint of responders with meaningful treatment benefit (36.2% vs. 34.1%, p=0.284)


Genitourinary/Sexual Health

Akebia Therapeutics Inc., of Cambridge, Mass.


Hypoxia-inducible factor prolyl hydroxylase inhibitor

Anemia due to chronic kidney disease

Pro2tect studies are fully enrolled with 3,513 non-dialysis-dependent patients; data expected in mid-2020


Apellis Pharma-ceuticals Inc., of Crestwood, Ky.


Inhibits C3 and C3b complement proteins

Treatment-naïve paroxysmal nocturnal hemoglobinuria

Treated the first of 54 patients in the Prince (APL2-308) trial to measure hemoglobin stabilization from baseline in the absence of transfusion through week 26 and reduction in lactate dehydrogenase levels from baseline to week 26


Ardelyx Inc., of Fremont, Calif.


Inhibits sodium hydrogen exchanger 3

Chronic kidney disease on dialysis with hyperphosphatemia not controlled by phosphate binders

In the 235-patient Amplify study, tenapanor plus phosphate binders reduced serum phosphorus from baseline to the end of week 4 by 0.84 mg/dL, compared to a mean reduction of 0.19 mg/dL for phosphate binders alone (p=0.0004); up to 49.1% of patients treated with tenapanor plus a binder achieved a serum phosphorus of <5.5 mg/dL, compared to 23.5% of patients treated with a binder alone (p≤0.0097); FGF23 levels were reduced by 22% to 24% (p≤0.0027) compared to binder alone


Urovant Sciences Ltd., of Irvine, Calif.


Oral beta-3 adrenergic receptor agonist

Overactive bladder

Double-blind extension of Empowur study found vibegron further improved key OAB symptoms over 40-week extension period; 41% of patients on vibegron became "dry"; NDA filing planned by early 2020



Emmaus Life Sciences Inc., of Torrance, Calif.

Endari (levoglutamide)

Oral solution of L-glutamine

Sickle cell disease

Study showed 25% lower frequency of painful crises (p=0.005), 33% lower frequency of hospitalization (p=0.005) and > 60% decrease in frequency of acute chest syndrome among those treated with Endari (p=0.003)


Rigel Pharmaceuticals Inc., of South San Francisco, and Kissei Pharmaceuticals Co. Ltd., of Tokyo

Fostamatinib disodium hexahydrate

SYK inhibitor

Chronic immune thrombocytopenia

Kissei started trial in Japan


Uniqure NV, of Amsterdam

AMT-061 (etranacogene dezaparvovec)

Gene therapy expressing Padua variant of factor IX

Hemophilia B

Completed enrollment in the 56-patient Hope-B study; expects over enrollment of up to 6 patients before the end of September; data expected in 2020



Asit Biotech SA, of Brussels



Grass pollen rhinitis

All sites have reached end of grass pollen season, allowing for last patient visits; results expected by mid-December 2019


Biogen Inc., of Cambridge, Mass.

Tecfidera (dimethyl fumarate)

Activates the nuclear erythroid 2-related factor 2 transcriptional pathway

Relapsing multiple sclerosis

In the Endorse extension study, 51% of patients remained relapse-free over 10 years; 64% had no confirmed disability progression; 79% maintained the ability to walk without significant disability; meta-analysis of 18 databases of large real-world studies found Tecfidera was significantly more effective than interferon-beta, glatiramer acetate and teriflunomide, had comparable effectiveness to fingolimod and was less effective than natalizumab and alemtuzumab in reducing annualized relapse rate and delaying time to first relapse


Biogen Inc., of Cambridge, Mass., and Alkermes plc, of Dublin

Diroximel fumarate

Activates the nuclear erythroid 2-related factor 2 transcriptional pathway

Relapsing multiple sclerosis

Interim results from 888 patients in the Evolve-MS-1 study showed the drug was generally well-tolerated; less than 1% of patients taking the drug discontinued treatment due to gastrointestinal side effects; drug reduced annualized relapse rate by 79.4% and the mean number of gadolinium-enhancing lesions by 64.3% from baseline to 24 months


EMD Serono, of Rockland, Mass., a unit of Merck KGaA


BTK inhibitor

Relapsing multiple sclerosis

Started 2 global, pivotal trials, Evolution RMS 1 and 2) comparing evobrutinib with interferon-beta-1a; primary endpoint of both studies is annualized relapse rate at week 96


Genmab A/S, of Copenhagen, and Novartis AG, of Basel, Switzerland


Fully human CD20 monoclonal antibody

Relapsing multiple sclerosis

Head-to-head Asclepios studies vs. once-daily oral teriflunomide in adults met their primary endpoints of reduction in number of confirmed relapses, as measured by annualized relapse rate (ARR); reduction in ARR was 50.5% and 58.5% for ofatumumab vs. teriflunomide (both studies p<0.001), in Asclepios I and II, respectively; secondary endpoints showed highly significant suppression of gadolinium T1 lesions vs. teriflunomide and relative risk reduction of 34.4% in 3-month confirmed disability progression (p=0.002) vs. teriflunomide


Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson


S1P1 receptor modulator

Relapsing multiple sclerosis

In the Optimum study, annualized relapse rate in patients taking ponesimod was reduced by 30.5% compared to Aubagio (teriflunomide, Sanofi SA) up to week 108 (p=0.0003); mean difference in Fatigue Symptoms and Impacts Questionnaire - Relapsing Multiple Sclerosis, favoring ponesimod over Aubagio, was -3.57 (p=0.0019); cumulative number of combined unique active lesions was reduced by 56% for ponesimod compared to Aubagio (p<0.0001)


Roche Holding AG, of Basel Switzerland

Ocrevus (ocrelizumab)

Monoclonal antibody targeting CD20-positive B cells

Relapsing multiple sclerosis and primary progressive multiple sclerosis

In the Opera I study in RMS, Ocrevus reduced blood serum neurofilament light chain (NfL) by 43% from baseline to 96 weeks compared to a 31% reduction for interferon-beta-1a (p<0.001); in the Oratorio study in PPMS, Ocrevus reduced blood NfL levels by 16% from baseline to 96 weeks compared with a 0.2% reduction for placebo (p<0.001)


Roche Holding AG, of Basel, Switzerland

Ocrevus (ocrelizumab)

Monoclonal antibody targeting CD20

Multiple sclerosis

Drug reduced neurofilament light chain levels in the blood; 6-year data showed patients treated earlier had lower rates of disability progression compared with relapsing MS patients who switched from interferon beta-1-alpha or primary progressive MS patients who switched from placebo after the double-blind phase


Roche Holding AG, of Basel, Switzerland

Ocrevus (ocrelizumab)

Monoclonal antibody targeting CD20-positive B cells

Relapsing multiple sclerosis, primary progressive MS and relapsing-remitting MS

In the Opera OLE, 19% of RMS patients had 24-week confirmed disability progression (CDP) after 6 years of Ocrevus treatment, compared to 24% of patients treated with 2 years of interferon beta-1a plus 4 years of Ocrevus (p<0.05); in the Oratorio OLE, 52% of PPMS patients treated with Ocrevus for more than 6.5 years had 24-week CDP compared to 65% of patients who switched from placebo after the double-blind period (p=0.002); 9-hole peg test was 13% vs. 43% for continuous treatment vs. switchers, respectively (p=0.004); continuous treatment resulted in a 42% reduction in the risk of patients needing a wheelchair (EDSS≥7) over 6.5 years compared with switching (p=0.0112); interim analysis of Casting study showed 87% of RRMS patients who switched to Ocrevus had no evidence of disease activity after 48 weeks of treatment



Entasis Therapeutics Inc., of Waltham, Mass., and the Global Antibiotic Research and Development Partnership


Oral antibiotic

Uncomplicated gonorrhea

Started global, pivotal trial, fully funded and sponsored by GARDP; about 1,000 adults with urogenital gonorrhea to be randomized to either zoliflodacin or combination of ceftriaxone/azithromycin, with assessment 1 week later for persistence of infection; data expected in 2021


Genentech, a unit of Basel, Switzerland-based Roche Holding AG

Xofluza (baloxavir marboxil)

Inhibits cap-dependent endonuclease

Influenza treatment

In the Ministrone-2 study of children, drug was deemed safe; time to alleviation of influenza signs and symptoms was 138.1 hours for Xofluza compared to 150 hours for Tamiflu (oseltamivir, Gilead Sciences Inc.); median time to stop viral shedding was 24.2 hours for Xofluza compared to 75.8 hours for Tamiflu


Genentech, a unit of Basel, Switzerland-based Roche Holding AG

Xofluza (baloxavir marboxil)

Inhibits cap-dependent endonuclease

Influenza prevention

In the Bockstone study of people living with an infected household member; 1.9% of Xofluza-treated household contacts developed the flu compared with 13.6% of household contacts treated with placebo (p<0.0001); data were similar regardless of influenza A subtype and for household contacts who are at high risk of flu-associated complications and children under 12 years of age


Merck & Co. Inc., of Kenilworth, N.J.

Recarbrio (imipenem 500 mg, cilastatin 500 mg and relebactam 250 mg)

Beta-lactam antibiotic, inhibitor of renal dehydropeptidase and beta-lactamase inhibitor

Hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia

Restore-IMI 2 trial met primary and key secondary endpoints of noninferiority vs. piperacillin/tazobactam in day 28 all-cause mortality and clinical response at early follow-up


Micurx Pharmaceuticals Inc., of Shanghai and Foster City, Calif.

Contezolid (MRX-1)

Oral oxazolidinone antibiotic

Complicated skin and soft tissue infection

Met primary endpoint of noninferiority vs. linezolid for clinical cure rate at test-of-cure visit (7-14 days after last dose), and was associated with fewer drug-related hematologic laboratory adverse events


Nabriva Therapeutics plc, of Dublin


Semi-synthetic pleuromutilin antibiotic

Community-acquired bacterial pneumonia

Journal of the American Medical Association published results from Leap 2 trial, showing a short-course treatment (5 days) was noninferior to a 7-day regimen of moxifloxacin; lefamulin achieved high clinical response rates for both typical and atypical pathogens


Scynexis Inc., of Jersey City, N.J.

Ibrexafungerp (SCY-078)

Triterpenoid antifungal

Vulvovaginal candidiasis

Completed last patient visit in Vanish 303 study; top-line data expected by year-end



Novartis AG, of Basel, Switzerland

Cosentyx (secukinumab)

Inhibits interleukin-17A

Non-radiographic axial spondyloarthritis

Data showed ongoing Prevent trial met primary endpoint of ASAS40 at week 16, showing significant and clinically meaningful reduction in disease activity vs. placebo



Ipsen SA, of Paris


Injectable form botulinum neurotoxin type A

Upper and lower limb spasticity

Results from Engage study showed improvement in patients' voluntary movement as measured by composite active range of motion outcome; 72.1% were classified as responders



Acadia Pharmaceuticals Inc., of San Diego

Nuplazid (pimavanserin)

Selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors

Dementia-related psychosis

Planned interim analysis of pivotal study found primary endpoint of statistically significant longer time to relapse of psychosis with pimavanserin vs. placebo has been met; trial, called Harmony, is stopping early


Alnylam Pharmaceuticals Inc., of Cambridge, Mass.


RNAi therapeutic

Transthyretin amyloidosis with cardiomyopathy

Started Apollo-B trial; primary endpoint is change from baseline in 6-minute walk test at 12 months; secondary endpoints will test efficacy on quality of life using Kansas City Cardiomyopathy Questionnaire Overall Summary and composite endpoints of mortality and hospitalization


Avexis, unit of Novartis AG, of Basel, Switzerland

Zolgensma (onasemnogene abeparvovec-xioi)

Gene therapy

Spinal muscular atrophy type 1

Interim data from Spr1nt trial in presymptomatic patients demonstrated that, of 22 patients evaluated overall, all were alive and free of permanent ventilation; all patients with 2 copies of SNM2 achieved or maintained Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders score of greater than 50, with 7 achieving score of greater than or equal to 60 and 5 reaching maximum score of 64; of patients with 2 copies of SNM2, 6 (60%) were able to sit without support for at least 30 seconds at an average age of 7.6 months


Biogen Inc., of Cambridge, Mass., and Ionis Pharmaceuticals, of Carlsbad, Calif.

Spinraza (nusinersen)

Antisense oligonucleotide targeting survival motor neuron

Spinal muscular atrophy

In the Shine extension study, children with later-onset SMA (type 2 or type 3) experienced improvements or stabilization in 1 or more measures of motor function — Hammersmith Functional Motor Scale–Expanded, Upper Limb Module and 6-Minute Walk Test — for up to nearly 6 years


Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn.


CGRP receptor antagonist


Analyses from long-term study BHV3000-201 found statistically significant improvements in migraine-related disability at all time points (p<.0001)


Centrexion Therapeutics Corp., of Boston


Synthetic, ultra-pure intra-articular injection of trans-capsaicin

Chronic moderate to severe knee osteoarthritis pain

Completed patient enrollment in third trial, Victory-3, involving 857 patients with pain in 1 or both knees; top-line results expected in first quarter of 2020


Chugai Pharmaceutical Co. Ltd., of Tokyo, and Genentech Inc., of South San Francisco, part of the Roche Group


Anti-IL-6 receptor humanized recycling antibody

Neuromyelitis optica spectrum disorder

In Sakurastar study, monotherapy reduced risk of relapse by 55% (p=0.0184) in overall population, including AQP4-IgG seropositive and seronegative patients, achieving primary endpoint of time to first protocol-defined relapse in the double-blind period


Eisai Co. Ltd., of Tokyo, and Biogen Inc., of Cambridge, Mass.


Beta amyloid cleaving enzyme inhibitor

Early Alzheimer's disease

Discontinuing Mission AD1 and Mission AD2 studies on the recommendation of the data safety monitoring board due to unfavorable risk-benefit ratio


Gedeon Richter plc., of Budapest, Hungary


Dopamine D3/D2 receptor partial agonist


CGI-I improvement was similar for cariprazine and risperidone, but more patients taking cariprazine had "much" and "very much" improvement; patients with predominant negative symptoms had significantly better clinical improvement with cariprazine compared to risperidone; improvement in PANSS Factor Score for Negative Symptoms correlated with improvement in Personal and Social Performance Scale


Impel Neuropharma Inc., of Seattle


Nasal formulation of dihydroergotamine via Impel's Precision Olfactory Delivery

Acute migraine

Last patient enrolled in the 360-patient Stop-301 study; top-line data expected in early 2020


Intec Pharma Ltd., of Jerusalem


Carbidopa/levodopa in the biodegradable polymeric film Accordion Pill platform

Parkinson's disease

The baseline percentage of daily OFF time and daily OFF time in hours were similar for AP-CD/LD and immediate release (IR) CD/LD; 86.2% of patients taking AP-CD/LD optimized to ≥1,200 mg LD compared with 19.7% of patients taking IR-CD/LD; ad hoc analysis showed greater difference in mean daily OFF time between AP-CD/LD and IR-CD/LD in patients who were not dose limited during the AP titration process; in a pharmacokinetics study, AP-CD/LD 50/500 mg 3 times daily reduced plasma levodopa variability compared to IR-CD/LD dosed 5 times per day (p=0.0048)


Janssen Research & Development LLC, of Titusville, N.J., a unit of Johnson & Johnson

Spravato (esketamine)

NMDA receptor antagonist

Major depressive disorder

Both Aspire I and II studies met their primary efficacy endpoints, reducing depressive symptoms at 24 hours after first dose; Spravato 84 mg plus standard of care (SOC) showed superiority (p=0.006) over placebo plus SOC in rapidly reducing symptoms of MDD


Jazz Pharmaceuticals plc, of Dublin

JZP-258 (sodium oxybate + potassium oxybate + calcium oxybate + magnesium oxybate)

GABA B receptor agonist

Cataplexy; narcolepsy

Global study that enrolled 201 participants and randomized 134 achieved primary and key secondary endpoints, demonstrating statistically significant differences in weekly number of cataplexy attacks (p<0.0001) and Epworth Sleepiness Scale scores (p<0.0001) compared to placebo


Novartis AG, of Basel, Switzerland

Aimovig (erenumab)

CGRP receptor antagonist


4.5-year data show 77% of patients who continued treatment experienced at least a 50% reduction in monthly migraine days at the last month of assessment


Ovid Therapeutics Inc., of New York

OV-101 (gaboxadol)

Delta-selective GABAA receptor agonist

Angelman syndrome

First patient randomized in pivotal Neptune study; about 50 pediatric patients, ages 4 to 12, will be enrolled; primary endpoint is change in overall score on Clinical Global Impression-Improvement-Angelman syndrome scale; top-line data expected in mid-2020


Roche Holding AG, of Basel, Switzerland


Monoclonal antibody targeting IL-6

Neuromyelitis optica spectrum disorder

In the Sakurastar study, satralizumab significantly reduced the risk of relapse in patients who were seropositive for aquaporin-4 autoantibodies, as well as the overall intent-to-treat population


Sunovion Pharmaceuticals Inc., of Marlborough, Mass., and Psychogenics Inc., of Paramus, N.J.


Believed to activate TAAR1 and 5-HT1A receptors


Initiated Diamond studies; program includes 4 trials: a 6-week study in acutely psychotic adults and adolescents (13 to 17); a 6-week study in acutely psychotic adults with schizophrenia; a 52-week trial in adults and adolescents who completed either of 6-week studies; and a 52-week comparator-controlled, long-term safety study in adults with schizophrenia


Zosano Pharma Corp., of Fremont, Calif.


Formulation of zolmitriptan delivered using ADAM titanium microneedle technology

Acute migraine

Long-term safety study showed treatment result in pain freedom for 44% of attacks and freedom from patients' most bothersome symptoms for 62% of attacks at 2 hours; treatment was well-tolerated through 12 months of repeated use



Eyegate Pharmaceuticals Inc., of Waltham, Mass.

Ocular bandage gel eye drop

Modified form of natural polymer hyaluronic acid

Corneal wound repair following photorefractive keratectomy surgery

Completed randomization of more than 75% of patients in pivotal study


Gensight Biologics SA, of Paris


Gene therapy

Leber hereditary optic neuropathy

Results from week 96 of Rescue trial testing single intravitreal injections in 39 patients whose vision loss due to 11778-ND4 LHON began up to 6 months prior to treatment showed best-corrected visual acuity sustaining clinically meaningful improvement over nadir; treated eyes regained more than two-thirds of initial loss occurring in most acute phase of the disease; that improvement from nadir (-0.498 LogMAR mean improvement, or +24.9 ETDRS letters equivalent) corresponds to 5 lines of Snellen acuity, above the 3-line threshold commonly accepted as clinically meaningful level of visual improvement


Nevakar Inc., of Bridgewater, N.J.


Topical eye drop formulation of atropine


Completed enrollment in the 576-patient Champ study measuring myopia progression in children over 3 years


Ocular Therapeutix Inc., of Bedford, Mass.

Dextenza (dexamethasone)

Glucocorticoid receptor agonist

Allergic conjunctivitis

First of about 80 participants dosed in trial evaluating safety and efficacy of study drug vs. placebo vehicle punctum plug; primary efficacy endpoint is ocular itching following insertion of Dextenza at multiple time points during 30-day period



Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. and Sanofi SA, of Paris

Dupixent (dupilumab)

IL-13 receptor antagonist; IL-4 receptor antagonist

Nasal polyps

Highlighted publication in The Lancet of detailed positive results from 2 in adults with recurring severe chronic rhinosinusitis with nasal polyps despite previous treatment with surgery and/or systemic corticosteroids.


Strekin AG, of Basel, Switzerland


Intratympanic thermogel and oral formulation of PPAR agonist pioglitazone

Sensorineural hearing loss

Completed enrollment of the target of 165 patients in Restore study; top-line results anticipated in early 2020



Boehringer Ingelheim GmbH, of Ingelheim, Germany


Tyrosine kinase inhibitor

Fibrosing interstitial lung disease

Data from Inbuild trial, published in The New England Journal of Medicine, showed nintedanib slowed lung function decline by 57% across overall study population, as assessed by annual rate of decline in forced vital capacity over 52 weeks in patients with signs of progression


Novartis AG, of Basel, Switzerland

Inhaled QMF-149

Fixed dose of indacaterol acetate and mometasone furoate


Palladium trial met primary endpoint, showing QMF-149 improved trough forced expiratory volume in 1 second after 26 weeks vs. mometasone furoate, meeting primary endpoint; superior improvement in lung function achieved in patients who remain uncontrolled on treatment with inhaled corticosteroids at median or high dose or on long-acting beta agonist at low dose



For more information about individual companies and/or products, see Cortellis.


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