Company Product Description Indication Status
Phase I

Atriva Therapeutics GmbH, of Tübingen, Germany


MEK inhibitor of viral replication

Healthy volunteers (eventually influenza)

ATR-002 was considered to be safe and well-tolerable in the single and multiple ascending-dose study; pharmacokinetic profile supports once-daily dosing

Celldex Therapeutics Inc., of Hampton, N.J.


Anti-CD40 monoclonal antibody

Advanced solid tumors

Interim data from 38 patients with pre- and post-scans showed 2 patients with radiographic evidence of tumor necrosis among 5 patients with recurrent/refractory head and neck squamous cell carcinoma; a patient with gastroesophageal carcinoma had 41% shrinkage of liver and lymph node target lesions; 6 patients had stable disease, including 4 on monotherapy and 2 on CDX-1140/CDX-301 combination

Corvus Pharmaceuticals Inc., of Burlingame, Calif.


Anti-CD73 antibody

Cancers that have failed standard therapies

Tumor regression seen in 4 of 9 evaluable patients receiving 6 mg/kg and higher

Formation Biologics Inc., of Austin, Texas


Inhibitor of TGF-beta 1 & 3

Advanced or metastatic solid tumor malignancies

Dose-escalation phase Ia study complete with 15 patients treated at 3 doses; several patients experienced stable disease, including 1 patient with prolonged stable disease

Forma Therapeutics Inc., of Watertown, Mass.


Fatty acid synthase inhibitor

Healthy volunteers (eventually nonalcoholic steatohepatitis)

FT-4101 at 6 mg and 9 mg reduced hepatic de novo lipogenesis by 42.6% (p=0.010) and 81.8% (p<0.0001), respectively, through 24 hours compared to placebo

Gilead Sciences Inc., of Foster City, Calif.


Capsid inhibitor

HIV infection

In healthy subjects, pharmacokinetics suggest drug has potential to be dosed up to every 6 months; in HIV patients at 20 mg to 450 mg, mean maximum reduction in HIV-1 RNA by day 10 was 1.4 to 2.2 log10copies/mL (p<0.0001 for all)

Ligand Pharmaceuticals Inc., of San Diego


Captisol-enabled contrast agent

Healthy volunteers (eventually X-ray imaging)

Pharmacokinetic parameters of area under the concentration-time curve and maximum concentration were similar between CE-Iohexol and Omnipaque (iohexol, GE Healthcare) with a geometric mean ratio of 1 for both; mean elimination constant was 0.3/hour for both treatments

Neon Therapeutics Inc., of Cambridge, Mass.


Neoantigen vaccine

Advanced or metastatic melanoma, smoking-associated non-small-cell lung cancer and bladder cancer

Of patients who started treatment in phase Ib trial, objective response rates were 59%, 39% and 27% in 27 melanoma patients, 18 NSCLC patients and 15 bladder cancer patients, respectively

Oncolytics Biotech Inc. of San Diego


Isolate of an unmodified reovirus

Early breast cancer

In the window-of-opportunity Aware-1 study, initial data showed replication of pelareorep occurs exclusively in tumor tissue and there was an increase in CelTIL score, a biomarker of T-cell clonality

Replimune Group Inc., of Woburn, Mass.


Engineered herpes simplex virus

Advanced cancers refractory to available therapy

Monotherapy produced tumor destruction, including delayed systemic post-study tumor reduction without further therapy; combination with Opdivo (nivolumab, Bristol-Meyers Squibb Co.) produced antitumor activity in multiple patients with a variety of tumor types, particularly in cutaneous squamous cell carcinoma and melanoma and also in microsatellite instability high colorectal cancer and esophageal cancer patients; first 3 of 4 patients with anti-CTLA4 and anti-PD-1-refractory cutaneous melanoma treated with RP-1 combined with Opdivo responded to therapy

Phase II

Anaptysbio Inc., of San Diego


Monoclonal antibody targeting IL-33

Moderate to severe atopic dermatitis

Each of the etokimab dosing arms failed to improve patients' Eczema Area and Severity Index scores relative to placebo at week 16; company plans update with additional data in the first quarter of 2020

Apellis Pharmaceuticals Inc., of Crestwood, Ky.

APL-2 (pegcetacoplan)

Complement C3 inhibitor

C3 glomerulopathy

6 participants in C3G cohort of Discovery study who received consistent administration from study days 1 to 84 showed downward trend in mean proteinuria, with mean reduction of 48.23% (11.22) in urine protein-to-creatinine ratio, from 2.03 (0.46) mg/mg to 1.05 (0.23) mg/mg (normal range <0.200 mg/mg), and normalization of mean serum albumin from baseline of 3.30 (0.32) g/dL to 3.98 (0.20) g/dL (normal range 3.50-5.50 g/dL)

Arrowhead Pharmaceuticals Inc., of Pasadena, Calif., and Janssen Pharmaceuticals Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson

JNJ-3989 (formerly ARO-HBV)

RNAi targeting hepatitis B virus

Chronic hepatitis B infection

In the AROHBV1001 study, JNJ-3989 plus a nucleos(t)ide analogue reduced HBeAg by 90% in 31 of 32 patients treated with 100 mg to 400 mg of JNJ-3989

Arrowhead Pharmaceuticals Inc., of Pasadena, Calif., and Janssen Pharmaceuticals Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson


Capsid assembly modulator

Chronic hepatitis B infection

JNJ-6379 plus JNJ-3989 and a nucleos(t)ide analogue produced a 90% reduction in HBsAg in all 12 patients

Bergenbio ASA, of Bergen, Norway


AXL inhibitor

Previously treated advanced non-small-cell lung cancer

Bemcentinib plus Keytruda (pembrolizumab, Merck & Co. Inc.) produced an overall response rate of 33% in AXL-positive patients; ORR was 7% in AXL-negative patients; median progression-free survival was 8.4 months in composite AXL-positive patients

Biothera Pharmaceuticals Inc., of Eagan, Minn.

Imprime PGG 

Dectin receptor agonist

Melanoma and chemorefractory metastatic triple-negative breast cancer

In 44 mTNBC patients, Imprime PGG plus Keytruda (pembrolizumab, Merck & Co. Inc.) produced a disease control rate of 25%, including 1 complete response and 6 partial responses; median overall survival was 16.4 months; 12-month OS rate was 57.6%; in 20 melanoma patients, combination produced a disease control rate of 50%, including 1 complete response; 12-month OS rate was 45%

Gilead Sciences Inc., of Foster City, Calif.


TLR-8 agonist

Hepatitis B virus infection

In study of 48 individuals with virally suppressed chronic disease, drug in combination with oral antivirals was well-tolerated and showed dose-dependent pharmacodynamic activity; 5% of those who received GS-9688 achieved ≥ 1 log10 IU/mL decline in HBV surface antigen levels or HBV e-antigen loss at 24 weeks

Gilead Sciences Inc., of Foster City, Calif.

Vemlidy (tenofovir alafenamide)

Nucleoside reverse transcriptase inhibitor

Hepatitis B virus infection

93 HBV patients with moderate to severe renal impairment or with ESRD on chronic hemodialysis who were virally suppressed on antiviral therapy were switched to Vemlidy; at week 24, all with ESRD and 97% with renal impairment met primary endpoint of maintaining viral load suppression; in those with renal impairment, switching to Vemlidy from tenofovir disoproxil fumarate (Viread) resulted in increased hip and spine bone mineral density and decreases in bone turnover markers

Immutep Ltd., of Sydney

Eftilagimod alpha (IMP-321)

HLA class II antigen stimulator

Non-small-cell lung cancer; head and neck squamous cell carcinoma

Interim data from TACTI-002 combination study with Keytruda (pembrolizumab, Merck & Co. Inc.) in up to 109 participants with first- or second-line NSCLC or second-line HNSCC showed overall response rate for those in part A stage 1 was 41%; 13 (76.5%) had reduction in target lesions; no responding participants (7/17) showed disease progression; 12 remain on treatment with median progression-free survival not reached

Iovance Biotherapeutics Inc., of San Carlos, Calif.


Tumor infiltrating lymphocyte-based autologous cell therapy

Metastatic melanoma

Cohort 2 of consecutively dosed post-PD-1 patients with stage IIIC/IV disease in ongoing study showed objective response rate of 35% assessed by independent review committee (IRC) and 36% assessed by investigator; median duration of response not reached as assessed by IRC (range 1.6+ to 21.2+ months) or investigator (range 2.2 to 21.2+ months) at 11.3 months median follow-up

Mirum Pharmaceuticals Inc., of Foster City, Calif.


Apical sodium co-dependent bile acid transporter inhibitor

Alagille syndrome

Consistent with results reported after 48 weeks of treatment, data on 23 participants from long-term extension of Iconic study showed statistically significant reductions in serum bile acids and pruritus and further improved in those who remained on treatment through 191 weeks compared to baseline (p<0.005 and p<0.0001, respectively); clinician scratch scale scores continued to improve (p<0.0001) and xanthomas continued to diminish (p<0.05)

Mirum Pharmaceuticals Inc., of Foster City, Calif.


Apical sodium co-dependent bile acid transporter inhibitor

Progressive familial intrahepatic cholestasis

Long-term extension of Indigo study in 25 children with bile salt export pump deficiency showed response was maintained in half of those with moderate mutations and was correlated to non-truncating mutations

Nantkwest Inc., of Culver City, Calif.

aNK (activated natural killer cells)

Combination chemoimmunotherapy vaccine

Metastatic Merkel cell carcinoma

Combination trial with IL-15 superagonist N-803 (Immunitybio Inc.) showed durable responses in 2 of 7 participants with advanced refractory disease, including 1 with radiologic complete response and 1 with durable partial response with more than 75% tumor regression and no subsequent therapy

Phase III

ALK-Abello A/S, of Copenhagen


Sublingual allergy immunotherapy

Seasonal allergic rhinitis

Study by Merck & Co. Inc. that randomized 1,025 children, ages 5 to 17, met primary endpoint of average total combined score (TCS) – sum of rhinoconjunctivitis daily symptom score (DSS) and rhinoconjunctivitis daily medication score (DMS) over peak ragweed season and key secondary endpoint of average TCS during entire season, with relative improvements in TCS vs. placebo of -38.3% (p<0.001) during peak season and -32.4% (p<0.001) during entire season

Exelixis Inc., of Alameda, Calif.


Mineralocorticoid receptor antagonist

Diabetic nephropathy

Partner Daiichi Sankyo Co. Ltd. reported that comparison study in 455 participants with incipient disease showed drug resulted in higher urinary albumin-to-creatinine ratio (UACR) remission rate vs. placebo (22.1% vs. 4%); treatment reduced UACR and was associated with reduction in progression from incipient to overt disease, both vs. placebo (-58.3% vs. +8.3% and 1.4% vs. 7.5%, respectively)

Fibrogen Inc., of San Francisco


HIF prolyl hydroxylase inhibitor

Anemia in chronic kidney disease

Himalayas trial that enrolled 1,043 anemia in incident dialysis participants with CKD achieved co-primary endpoints of noninferiority in mean hemoglobin (Hb) change from baseline, with mean Hb increase from 8.4 g/dL to 11 g/dL in treatment arm vs. 8.4 g/dL to 10.8 g/dL for epoetin alfa, and proportion of patients achieving Hb response during first 24 weeks, at 88.2% vs. 84.4%

Gilead Sciences Inc., of Foster City, Calif.

Vemlidy (tenofovir alafenamide)

Nucleoside reverse transcriptase inhibitor

Hepatitis B virus infection

Long-term analysis of 2 studies that enrolled 1,632 individuals showed hepatocellular carcinoma was observed in 21 patients, including 1% of Vemlidy cohort vs. 1.9% treated with tenofovir disoproxil fumarate (Viread) across 3 to 5 years of follow-up

Gilead Sciences Inc., of Foster City, Calif.

Vemlidy (tenofovir alafenamide)

Nucleoside reverse transcriptase inhibitor

Hepatitis B virus infection

In analysis from study of those with virally suppressed chronic disease, 243 previously treated with tenofovir disoproxil fumarate (TDF, Viread) for median of 4 years and switched to Vemlidy for 48 weeks showed improvement in bone and renal markers regardless of duration (<4 years vs. ≥ 4 years) of prior TDF use

Intercept Pharmaceuticals Inc., of New York

Obeticholic acid (OCA)

Farnesoid X receptor agonist

Liver fibrosis

OCA-treated patients in the primary ITT group in ongoing Regenerate study showed time- and dose-dependent improvements vs. placebo across noninvasive tests, including blood tests of fibrosis, as early as 3 months after treatment initiation; vibration-controlled transient elastography, an imaging assessment of liver stiffness and surrogate of fibrosis, decreased from baseline in both OCA groups but increased with placebo at 18 months

Takeda Pharmaceutical Co. Ltd.,

Takhzyro (lanadelumab-flyo)

Plasma kallikrein inhibitor"

Hereditary angioedema

Open-label extension of Help study in individuals 12 and older showed safety profile was consistent with original findings; drug, dosed 300 mg every 2 weeks, reduced mean attack rate by 87% overall compared with baseline (n=212); overall reduction of 92.6% observed in rate of attacks requiring acute treatment (n=212) and 83.6% in rate of moderate/severe attacks vs. baseline (n=212)


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