Company (location)







Aivita Biomedical Inc., of Irvine, Calif.


Stem cells


Dosed the first 2 patients in trial, which will enroll about 55 patients to receive patient-specific cancer treatment, which is administered in a series of subcutaneous injections alongside standard care


Bavarian Nordic A/S, of Copenhagen


Prime-boost immunotherapy

Advanced chordoma

Dosed the first patient; 29-patient, 2-stage study will test combination of MVA-BN-Brachyury followed by booster of recombinant fowlpox virus FPV-Brachyury and radiation on objective response rate within 12 months of radiation therapy


Bayer AG, of Leverkusen, Germany

Stivarga (regorafenib)

Dual-acting VEGFR 1 to 3 , TIE, KIT, RET, BRAF, CRAF, PDGFR and FGFR inhibitor


Global Coalition for Adaptive Research and Bayer said regorafenib will be first drug to enter GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment), an adaptive platform trial that will evaluate multiple therapies for patients with newly diagnosed and recurrent GBM


Bergenbio ASA, of Bergen, Norway


Selective oral AXL inhibitor

Non-small-cell lung cancer

Drug is tested in combination with Keytruda (pembrolizumab, Merck & Co. Inc.); 24 patients have been enrolled during the first stage of the trial, and the median time that patients lived without progression of their disease (mPFS) was 5.9 months in AXL-positive patients (n=10), greater than the mPFS of 3.3 months in patients whose tumors did not show any AXL expression per the company's biomarker test (n=11)


Beyondspring Inc., of New York


GEF-H1 activator

Chemotherapy-induced neutropenia in non-small-cell lung cancer

Patients taking Neulasta (pegfilgrastim, Amgen Inc.) had neutrophil-to-lymphocyte ratios of greater than 5 and lymphocyte-to-monocyte ratios of less than 3.2, which wasn't seen in patients taking plinabulin; promyelocytes or myelocytes were observed in 77% of patients taking Neulasta, compared to 14% of patients given plinabulin (p<0.001); neutrophil bands were seen in greater than 25% of patients given Neulasta, compared to 0% of patients taking plinabulin (p<0.03)


Biomarck Pharmaceuticals Ltd., of Durham, N.C.


Peptide inhibiting MARCKS protein

Stage 4 non-small-cell lung cancer

Completed enrollment in study comparing standard of care (pemetrexed/carboplatin) to SOC plus BIO-11006 in 60 patients; primary endpoint is progression-free survival at 3 months; initial results expected in March 2019


Celgene Corp., of Summit, N.J., and Bluebird Bio Inc., of Cambridge, Mass.


Anti-BCMA CAR T-cell therapy

Relapsed and refractory multiple myeloma

Completed enrollment in the KarMMa pivotal study


Delmar Pharmaceuticals Inc., of Vancouver, British Columbia


Bifunctional DNA-targeting agent

MGMT-unmethylated, bevacizumab-naïve recurrent glioblastoma multiforme

Updated data show as of Oct. 31, 44 of 48 patients have enrolled; of 27 subjects completing at least 2 cycles of treatment, 9 (33.33%) exhibited stable disease; 7 patients are currently receiving treatment, while 22 are being followed for survival and 19 are deceased thus far


G1 Therapeutics Inc., of Research Triangle Park, N.C.


Myelopreservation therapy

Small-cell lung cancer

Top-line data from a study in combination with chemotherapy and checkpoint inhibitor Tecentriq (atezolizumab, Roche Holding AG) in first-line patients showed trilaciclib reduced clinically relevant consequences of myelosuppression vs. placebo across three lineages – neutrophils, red blood cells and platelets; statistically significant improvements in both primary endpoints of occurrence of grade 4 neutropenia and duration of grade 4 neutropenia in cycle 1; statistically significant reduction in grade 4 thrombocytopenia and clinically meaningful reduction in red blood cell transfusions


Gradalis Inc., of Dallas


Cellular immunotherapy

Ewing's sarcoma

Initial data from part 2 of the trial testing Vigil in combination with temozolomide/irinotecan showed 5 of 8 patients enrolled are still alive; 2 patients experienced RECIST partial response and retrospectively confirmed histological complete response; 4 had durable stable disease as best response at 6 months or longer


Iovance Biotherapeutics Inc., of San Carlos, Calif.


Autologous cell therapy

Metastatic melanoma

Drug yielded overall response rate of 38% in 47 consecutively dosed patients, including 1 complete response and 17 partial responses, 4 of which were unconfirmed as of Oct. 25, pending upcoming second assessments; a median duration of response of 6.4 months with a range of 1.3+ to 14+ months; all patients were unsuccessfully treated with prior anti-PD-1 treatment; mean prior systemic therapies for all patients was 3.3


Lidds AB, of Uppsala, Sweden

Liproca Depot

Controlled-release anti-androgen 2-hydroxyflutamide

Prostate cancer

Added 2 major university clinics in Lithuania to speed up enrollment in ongoing phase IIb study


Merrimack Pharmaceuticals Inc., of Cambridge, Mass.

MM-121 (seribantumab)

Anti-HER3 monoclonal antibody

Non-small-cell lung cancer and metastatic breast cancer

Company is discontinuing development of all ongoing MM-121 programs based on interim results from its phase II Sherloc study in NSCLC, including termination of the phase II Sherboc study in metastatic breast cancer


Mirati Therapeutics Inc., of San Diego


Oral, class I and IV selective HDAC inhibitor

Non-small-cell lung cancer

Data from ongoing trial in combination with PD-L1 drug Imfinzi (durvalumab, Astrazeneca plc) in patients with documented disease progression after prior checkpoint inhibitor therapy showed 12 of 29 evaluable patients had tumor reduction; 6 patients demonstrated tumor reduction of greater than 30%, 5 achieved confirmed partial response; 4, including 2 responding patients, remained on treatment at the time of data cut-off


Mirati Therapeutics Inc., of San Diego


Spectrum-selective kinase inhibitor of receptor tyrosine kinases, including TAM family receptors (TYRO3, Axl, Mer), split family receptors (VEGFR2, KIT) and RET

Non-small-cell lung cancer

Preliminary biomarker data from ongoing trial in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.) demonstrated a CD8+ T effector cell response in patients who achieved a clinical benefit, suggesting a therapy-driven restoration of the antitumor immune response in patients who had become refractory to prior checkpoint inhibitor treatment


Oncbiomune Pharmaceuticals Inc., of Baton Rouge, La.


Immunotherapy consisting of PSA with IL-2 and GM-CSF

Low-risk localized prostate cancer

Plans to start 120-patient study on or about Dec. 17, 2018, that will measure PSA and cancer progression for 2 years in patients treated with Proscavax or assigned active surveillance, enrolled at a 2-to-1 ratio


Oncology Venture A/S, of Hoersholm, Denmark


Liposomal formulation of cisplatin

Metastatic breast cancer

Data from ongoing study shows 50% response rate (5/10) in the upper 1/3 of patients selected using DRP technology to track and match protocol for treatment, and 24% response rate 6/25 in the upper 2/3 of DRP-selected patients


Oncolytics Biotech Inc., of Calgary, Alberta


Intravenously delivered immuno-oncolytic virus

Advanced pancreatic adenocarcinoma

Treated first patient in study in combination with Keytruda (pembrolizumab, Merck & Co. Inc.); primary objective is overall response rate by iRECIST criteria, while secondary objectives include safety, immune response, progression-free survival and 1-year, 2-year and median overall survival


Oncosec Medical Inc., of San Diego

Tavo (tavokinogene telseplasmid)

Plasmid IL-12

Metastatic melanoma

2 of 9 patients who had completed 12 weeks of treatment experienced a partial response and 1 patient had stable disease (22% best overall response rate and 33% disease control rate) by RECIST v1.1 criteria; tumor responses were noted in both treated and untreated lesions; of 2 responding patients, both had multiple prior rounds of anti-PD-1 therapy, with no response, and 1 had also progressed after 4 cycles of Opdivo (nivolumab, Bristol-Myers Squibb Co.) and Yervoy (ipilimumab, Bristol-Myers Squibb Co.); tumor responses were associated with treatment-related up-regulation of immune-based transcripts in the tumor microenvironment, as well as increased frequencies of intratumoral T cells within 3 weeks of therapy


Oncosec Medical Inc., of San Diego

Tavo (tavokinogene telseplasmid)

Intratumoral plasma encoded IL-12 plus electroporation

Triple-negative breast cancer

First patient treated in KEYNOTE-890, testing combination of TAVO and Keytruda (pembrolizumab, Merck & Co. Inc.) in patients with late-stage disease


Oncosec Medical Inc., of San Diego

Tavo (tavokinogene telseplasmid)

Intratumoral delivery of DNA-based interleukin-12

Metastatic melanoma

New abscopal response data showed monotherapy treatment resulted in 47% of patients experiencing tumor regression in at least 1 untreated lesion


Sellas Life Sciences Group Inc., of New York

Neuvax (nelipepimut-S)

HER2-directed cancer immunotherapy

HER2-low-expressing breast cancer

Secondary efficacy analysis across HLA allele subgroups in phase IIb trial of nelipepimut-S, with or without trastuzumab show in triple-negative breast cancer subgroup with HLA-A24+ allele type, the combination treatment produced "p" value of 0.003 and 90.6% relative reduction in risk of relapse or death at 24 months


Sellas Life Sciences Group Inc., of New York



High-risk, high-expression HER2-positive breast cancer

Completed enrollment in investigator-sponsored study in combination with Herceptin (trastuzumab, Roche Holding AG); data from 100-patient study expected in the 4Q2019


Tracon Pharmaceuticals Inc., of San Diego


Base excision repair pathway inhibitor


Combination with Temodar (temozolomide) was tolerable but did not meet the primary efficacy endpoint of demonstrating objective responses by Response Assessment in Neuro-Oncology criteria in the initial 19 enrolled patients; 2 patients met the secondary endpoint of progression-free survival beyond 6 months


X4 Pharmaceuticals Inc., of Cambridge, Mass.


CXCR4 allosteric antagonist

Solid tumors

Demonstrated that single-agent drug has the ability to help restore immunity within the tumor microenvironment (TME), based on infiltration and activation of cytotoxic CD8+ T cells and increased inflammatory status in the TME, following once-daily, oral administration 



Hemostemix Inc., of Calgary, Alberta


Angiogenic cell precursor therapy

Critical limb ischemia

Received approval for 3 medical centers to be added as trial sites; 14 treated in study so far, and 29 have been screened to assess whether they meet enrollment criteria


Mesoblast Ltd., of New York


Allogeneic mesenchymal precursor cell

End-stage heart failure

Failed to meet the primary endpoint in a phase IIb trial in patients implanted with a left ventricular assist device, but it delivered meaningful clinical results


Pharmazz Inc., of Naperville, Ill.

PMZ-2010 (centhaquin)

Resuscitative agent acting through alpha-2B-adrenergic stimulation

Hypovolemic shock

Results showed drug significantly improved all 3 surrogate clinical endpoints of blood pressure, blood lactate and base-deficit vs. standard of care; significant (mean diff. -29.39; 95% Cl of diff -37.85 to -20.94; p<0.0001) increase in systolic pressure was observed, along with significant (mean diff. -17.13; 95% Cl of diff -23.45 to -10.81; p<0.0001) increase observed in diastolic blood pressure; significant (p=0.0028) decrease in blood lactate levels occurred at day 3 compared to baseline at day 0


Phasebio Pharmaceuticals Inc., of Malvern, Pa.


Sustained-release analogue of human vasoactive intestinal peptide fused to ELP half-life extension technology

Pulmonary arterial hypertension

Dosed the first patient in a phase IIb trial to assess safety, tolerability and efficacy of once-weekly subcutaneous injections in about 60 PAH patients


Tenax Therapeutics Inc., Morrisville, N.C.


Calcium sensitizer

Pulmonary hypertension

Stanford University School of Medicine activated as the first site for trial in patients with pulmonary hypertension and heart failure with preserved ejection fraction



Allakos Inc., of San Carlos, Calif.


Antibody that selectively depletes mast cells and eosinophils

Chronic urticaria

Enrollment completed in the open-label, 6-month study; top-line data from chronic spontaneous, Xolair-naïve cohort expected early in the first quarter of 2019; top-line results from remaining cohorts expected in the middle of the first quarter of 2019


Concert Pharmaceuticals Inc., of Lexington, Mass.


JAK1 and JAK2 inhibitor

Moderate to severe alopecia areata

At the 8-mg twice-daily dose, 47% of patients achieved at least a 50% relative reduction in their overall severity of alopecia tool (SALT) score from baseline, which was a statistically significant improvement over placebo (p <0.001); at the 4-mg dose, 21% of patients achieved at least a 50% relative reduction in their overall SALT score from baseline, but it wasn't significantly different from placebo; results from the cohort treated with 12 mg twice-daily are expected in the third quarter of 2019


Encore Dermatology Inc., of Malvern, Pa.

Impoyz (clobetasol propionate) cream, 0.025%

Binds to cytoplasmic glucocorticoid receptors


Data published in SKIN: The Journal of Cutaneous Medicine showed subjects treated with Impoyz had significantly lower mean post-treatment clobetasol propionate plasma concentrations vs. subjects treated with Temovate cream 0.05% (56.3 vs. 152.5 pg/mL, p=0.014); both treatment groups evidenced similar improvements in global psoriasis severity as measured by improvements in the Investigator's Global Assessment from baseline to day 15


Factor Therapeutics Ltd., of Brisbane, Australia


Truncated vitronectin linked to IGF-1

Venous leg ulcers

Stopped all development after a phase II trial failed to meet all endpoints, which included reduction in wound size, proportion of patients with ulcers fully healed and time to achieve full wound healing


Inflarx NV, of Jena, Germany


Anti-human complement factor C5a antibody

Moderate or severe hidradenitis suppurativa

Completed enrollment of the 16-week study measuring the Hidradenitis Suppurativa Clinical Response scores of patients treated with 4 doses or placebo; study also includes a 28-week open-label extension; top-line results are expected in the first half of 2019


Kalvista Pharmaceuticals Inc., of Cambridge, Mass.


Plasma kallikrein inhibitor

Hereditary angioedema

Plans to start an enlarged study of approximately 50 patients before the end of 2018; study will include an in-patient safety, pharmacokinetic and pharmacodynamic portion and an out-patient crossover phase to measure efficacy of drug compared to placebo; data expected in late 2019


Morphosys AG, of Martinsried, Germany

Tremfya (guselkumab)

Anti-IL-23 monoclonal antibody

Hidradenitis suppurativa

Said licensee Janssen Research & Development LLC initiated a phase II study in patients with moderate to severe HS


Samumed LLC, of San Diego


Wnt pathway activator


Dosed first subject in its phase II/III trial



Novan Inc., of Morrisville, N.C.


Topical nitric oxide-releasing candidate

External genital warts caused by human papillomavirus

Data published in the Journal of Drugs in Dermatology showed percentage of patients who experienced complete clearance was higher for treatment groups vs. vehicle, with greatest difference seen between SB-206 12% once daily (33.3%, p-0.010) and vehicle once daily (4.3%)


Oramed Pharmaceuticals Inc., of New York


Oral insulin capsule

Type 2 diabetes

Randomized more than 50% of the expected 285 patients in the 90-day dose-ranging pivotal study; data expected in the coming year


Rhythm Pharmaceuticals Inc., of Boston


MC4R agonist

Alström Syndrome

One patient maintained a weight loss of 20.9% and a reduction in baseline hunger score of 45.5% following 56 weeks of observation, including 50 weeks of treatment at a therapeutic dose; 2 other patients had weight loss of 5.4% and 5.5% following 46 weeks and 20 weeks of observation, including 36 weeks and 13 weeks on therapeutic doses, respectively; 1 of those patients had a 66.7% reduction in hunger score


Soleno Therapeutics Inc., of Redwood City, Calif.


Diazoxide choline controlled release

Prader-Willi Syndrome

Patients who received 10 weeks of treatment had statistically significant loss of total body fat mass, without additional caloric restrictions, with larger doses resulting in greater loss of body fat


Valbiotis SA, of La Rochelle, France


Based on combination of 5 plant extracts selected for their multitarget effect on metabolism

Risk reduction of type 2 diabetes

Last subject included in phase IIa trial



Durect Corp., of Cupertino, Calif.


Anti-inflammatory agent

Alcoholic hepatitis

Amended its ongoing phase IIa trial of I.V. DUR-928 in patients with AH to accelerate the initiation of dosing of severe AH patents


Galmed Pharmaceuticals Ltd., of Tel-Aviv, Israel


SCD1 modulator

Nonalcoholic steatohepatitis

Phase IIb data identified aramchol 600 mg as potentially effective to resolve NASH and improve fibrosis; improved liver enzymes and glycemic control; results favor further testing in phase III trial


Genkyotex SA, of Archamps, France


NOX1/4 inhibitor

Fibrotic diseases including nonalcoholic steatohepatitis and idiopathic pulmonary fibrosis

Met primary and secondary interim endpoints with statistical significance after 6 weeks of treatment; GKT-831 achieved greater GGT reductions (29%, p<0.01 vs. placebo) in patients with higher baseline GGT (≥ 2.5 XULN, n=68), suggesting it may also benefit patients with more advanced disease, and progressive reductions from baseline to week 2 and to week 6 suggest further improvements can be achieved with continued treatment


Gilead Sciences Inc., of Foster City, Calif.


Selective, nonsteroidal farnesoid X receptor agonist

Advanced fibrosis due to nonalcoholic steatohepatitis

A decline of at least 30% in hepatic fat measured by magnetic resonance imaging-proton density fat fraction (MRI-PDFF) was observed in 38.9% of patients treated with 100 mg (p=0.011 vs. placebo), 14% treated with 30 mg (p=0.87) and 12.5% with placebo; improvements in liver biochemistry tests (serum GGT) and markers of reduced bile acid synthesis (serum C4 and bile acids) were observed in the 30-mg and 100-mg arms


Gilead Sciences Inc., of Foster City, Calif.


Selective, nonsteroidal farnesoid X receptor agonist

Primary sclerosing cholangitis

After 12 weeks of treatment, patients receiving 100 mg demonstrated significant improvements in liver biochemistry tests, with a median reduction in serum alkaline phosphatase of 20.5% vs. an increase of 3.4% with placebo (p=0.029), median reduction in gamma-glutamyl transferase of 30.3% vs. an increase of 1.1% with placebo (p<0.001), median reduction in alanine aminotransferase of 49.4% vs. 12.9% with placebo (p=0.009) and a median reduction in aspartate aminotransferase of 42.3% vs. 10.8% with placebo (p=0.019); in both treated groups, reduced serum levels of C4, an intermediate in the synthesis of bile acids, were observed compared with placebo (-23.2% for 100 mg, p=0.21; and -30.5% for 30 mg, p=0.024)


Lipocine Inc., of Salt Lake City


Prodrug of bioidentical testosterone

Assessing liver fat changes in hypogonadal men at risk of developing nonalcoholic steatohepatitis

Completed enrollment in proof-of-concept study using magnetic resonance imaging, proton density fat fraction technique; results expected in the first quarter of 2019


NGM Biopharma-ceuticals Inc., of South San Francisco


Engineered version of FGF19

Nonalcoholic steatohepatitis

Preliminary histology data show treatment with 1-mg doses resulted in rapid and robust impact on both fibrosis stage and disease activity, as measured by the nonalcoholic fatty liver disease activity score at 12 weeks


Qu Biologics Inc., of Vancouver, British Columbia


Site-specific immunomodulator

Crohn's disease

Enrolled 10 of 20 patients with moderate to severe disease for the first stage of the trial designed to demonstrate mucosal healing with SSI treatment


Theravance Biopharma Inc., of Dublin


Oral, gut-selective pan-JAK inhibitor

Crohn's disease

Dosed the first patient in a double-blind, placebo-controlled, parallel-group study to enroll about 160 patients with moderately to severely active disease; primary endpoint is improvement in Crohn's Disease Activity Index score at 12 weeks; includes an active treatment extension phase


Viking Therapeutics Inc., of San Diego


Liver-selective thyroid receptor beta agonist

Nonalcoholic fatty liver disease and elevated LDL-C

Data from 12-week trial, which met primary endpoint of reduction in LDL-C vs. placebo (p=0.0121 for 10-mg QOD group, p=0.0269 for 10-mg QD group and p=0.0061 for combined treatment groups); patients also demonstrated statistically significant improvements in other lipids, including atherogenic proteins apolipoprotein B and lipoprotein (a), and showed statistically significant reductions in liver fat content vs. placebo


Genitourinary/Sexual Health

Fervent Pharmaceuticals Inc., of Greenville, N.C.


Oral version of approved drug; nonhormonal/nonherbal/non-antidepressant treatment

Vasomotor symptoms associated with menopause

Completed 2-month phase IIa trial in 112 women


Urovant Sciences Ltd., of Basel, Switzerland


Oral beta-3 adrenergic agonist

Overactive bladder

Phase IIb data published in European Urology showed once-daily treatment was well-tolerated and improved OAB symptoms; in part 1, patients on vibegron 50 mg and 100 mg had clinically and statistically significant decreases in the daily number of micturitions, urgency urinary incontinence episodes, total incontinence episodes and urgency episodes at week 8 vs. placebo; in part 2, on the primary endpoint – reduction in micturitions – treatment was statistically significant and similar to results observed in part 1



Twi Biotechnology Inc., of Taipei, Taiwan


Control released formulation of AC-201

Hemophilic arthropathy

Completed enrollment in the 22-patient study; data expected in the second quarter of 2019


Uniqure NV, of Amsterdam


Gene therapy expressing a FIX-Padua variant

Hemophilia B

Mean FIX levels 6 weeks after administration in 3 patients was 31% of normal



Amplyx Pharmaceuticals Inc., of San Diego


Prodrug of APX-001A, a small-molecule inhibitor of Gwt1

Candida infections

First patient dosed


Ark Biosciences Inc., of Shanghai


RSV F-protein inhibitor

Respiratory syncytial virus

Results from part 1 of the VICTOR study in hospitalized RSV-infected infants, treatment showed dose-dependent efficacy, reducing signs and symptom scores


Entasis Therapeutics Holdings Inc., of Waltham, Mass.


Oral antibiotic inhibiting DNA synthesis

Uncomplicated gonorrhea

Data from the study published in The New England Journal of Medicine showed 98% of the 49 patients treated with the 2-gram zoliflodacin dose, 100% of the 47 patients taking the 3-gram dose and 100% of the 21 patients taking ceftriaxone were considered cured of their urogenital gonorrhea based on culture results after 6 days; plans to start a phase III study in 2019


Gilead Sciences Inc., of Foster City, Calif.

Epclusa (sofosbuvir 400 mg/velpatasvir 100 mg)

NS5B and NS5A inhibitor

HCV-infected patients with severe renal impairment undergoing dialysis

Treatment with once-daily single-tablet regimen for 12 weeks in genotypes 1, 2, 3, 4 or 6 HCV resulted in cure rates (SVR12, or undetectable viral load 12 weeks after completion of therapy) of 95% (n=56/59) with only 2 patients experiencing virologic failure


Gilead Sciences Inc., of Foster City, Calif.

Harvoni (ledipasvir/sofosbuvir)

NS5A inhibitor and NS5B inhibitor

Hepatitis C virus in pediatric patients

In patents, ages 3 to 5, with genotypes 1 or 4, 97% were cured and none experienced virologic failure


Micurx Pharmaceuticals Inc., of Foster City, Calif.

Contezolid acefosamil (MRX-4)

Prodrug of oral antibiotic Contezolid (MRX-I)

Acute bacterial skin and skin structure infections caused by gram-negative bacteria

Enrolled first patient in 200-patient study to evaluate safety and efficacy of oral and intravenous formulations for 10 to 14 days of therapy vs. linezolid; expected to complete by mid-2019


Novan Inc., of Morrisville, N.C.


Topical antiviral gel

Molluscum contagiosum

Higher rates of complete clearance of all molluscum lesions at week 12 for the 2 highest doses, SB-206 8% and 12% twice-daily, more than double the rate observed in the vehicle group; clear treatment effect observed as early as week 4 in the percent reduction of molluscum lesions; company plans to initiate phase III program in the first half of 2019 with top-line results possible by the end of 2019 or early 2020


Paratek Pharmaceuticals Inc., of Boston


Tetracycline antibiotic

Acute pyelonephritis

Dosed the first patient in study testing oral and intravenous administration; top-line data are expected in the second half of 2019


Spring Bank Pharmaceuticals Inc., of Hopkinton, Mass.

Inarigivir soproxil

Selectively activates RIG-1

Chronic hepatitis B virus

Data demonstrate a dose-dependent antiviral effect on HBV DNA, with reduction of up to 2.75log10 at 100 mg and similar reductions in HBV RNA; reduction in HBsAg in 13 predefined (HBsAg reduction > 0.5log10) responder patients (7 HBeAg negative, 6 HBeAg positive) showed mean reduction of 0.8log10 (range 0.5log10 – 1.4log10)


Themis Bioscience GmbH, of Vienna, Austria


Chikungunya fever vaccine

Chikungunya fever

Primary endpoint, defined as the presence of neutralizing antibodies against Chikungunya, 4 weeks after administration of 1 or 2 MV-CHIK injections, was met across all treatment groups; results published in The Lancet


Visterra Inc., of Waltham, Mass.


Monoclonal antibody targeting influenza A

Influenza A infection

There were positive trends in the resolution of influenza symptoms after treatment; TCID50 viral culture endpoints favored VIS-410 in the modified intent to treat population; a post-hoc subgroup analysis showed patients who were baseline culture positive and had HAI of ≤40 had robust improvements in TCID50 with VIS410 treatment



Cymabay Therapeutics Inc., of Newark, Calif.


Orally active peroxisome proliferator-activated receptor delta agonist

Primary biliary cholangitis

At 52 weeks, the mean decreases in alkaline phosphatase (AP) were -47% and -46% in the 5/10- and 10-mg groups, respectively; a key secondary outcome was the composite responder rate measured at week 52 where a responder was defined as a patient with AP <1.67 x ULN, ≥15% decrease in AP, and total bilirubin ≤ULN; at 52 weeks, 59% and 71% of patients met the composite endpoint in the 5/10- and 10-mg groups, respectively


Generon Biomed Holding Ltd., of Shanghai


Recombinant fusion protein containing 2 human interleukin-22 and human IgG2-Fc

Alcoholic hepatitis

In diseased patients treated with F-652, there was a significant decrease in the Mayo End-Stage Liver Disease (MELD) score, total bilirubin, ALT and AST at day 42 when compared to baseline; subjects with severe disease had larger improvement in MELD scores at day 42; Lille score was ≤0.45 (defined as responders) in 15/18 patients (83%); the half-life of the drug following the first dose was 85 ± 16 hours; no severe adverse events related to the study drug; esophageal varices count decreased significantly at day 42 in parallel to the decreased MELD scores


Principia Biopharma Inc., of South San Francisco


Oral, small-molecule, reversible covalent BTK inhibitor


Primary efficacy endpoint – Control of Disease Activity within 4 weeks – was achieved by more than 50% of patients and drug was generally well-tolerated



Cytokinetics Inc., of South San Francisco

Reldesemtiv (CK-2127107)

Next-generation fast skeletal muscle troponin activator

Amyotrophic lateral sclerosis

Completed patient enrollment in the FORTITUDE-ALS study designed to assess change from baseline in percent predicted slow vital capacity and other measures of skeletal function after 12 weeks



Actinogen Medical Ltd., of Sydney


Blocks activity of 11beta-HSD1

Mild Alzheimer's disease

Enrolled the final patient in the XanADu trial; 186-patient trial is comparing Xanamem to placebo in patients with mild dementia due to AD; results expected in the second quarter of 2019


Antibe Therapeutics Inc., of Toronto


Hydrogen sulfide-releasing derivative of naproxen

Pain and inflammation

Completed part 1 of phase IIb dose-ranging study in 24 healthy volunteers showing cyclo-oxygenase inhibition at 250-mg dose was consistent with earlier studies and marked inhibition was observed with the 2 lower doses; drug was safe and well-tolerated; part 2 of the study remains on track to start in January 2019, with top-line read-out in the second quarter of 2019


Aptinyx Inc., of Evanston, Ill.


Modulator of NMDA receptors

Diabetic peripheral neuropathy

Completed enrollment; top-line data expected in the first quarter of 2019


Axsome Therapeutics Inc., of New York


Dextromethorphan and bupropion

Major depressive disorder

Completed enrollment in randomized, double-blind, active-controlled ASCEND (Assessing Clinical Episodes in Depression) study; top-line results expected in early January 2019


Cognition Therapeutics Inc., of Pittsburgh

Elayta (CT-1812)

Displaces amyloid beta oligomers from their synaptic receptors

Mild to moderate Alzheimer's disease

Treated first patient in Shine safety study of up to 160 patients; the study will be conducted in 2 parts with 24 initial patients treated at 1 of 2 doses for 6 months before the remaining patients are enrolled


H. Lundbeck A/S, of Valby, Denmark


Partial agonist activity at serotonin 5-HT1A and dopamine D2 receptors, and antagonist activity at serotonin 5-HT2A receptors

Post-traumatic stress disorder

Positive results (in intention-to-treat population) as measured by the Clinician-Administered PTSD Scale for DSM-5 total score change from baseline vs. placebo, when brexpiprazole and sertraline given as combination treatment (p<0.01); the treatment effects of brexpiprazole alone did not demonstrate clinically meaningful differences vs. placebo on primary endpoint (p>0.35); the treatment effects of sertraline alone also did not demonstrate clinically meaningful differences vs. placebo on primary endpoint (p>0.60)


Intelgenx Corp., of Saint Laurent, Quebec

Montelukast Versafilm

Leukotriene receptor antagonist

Mild to moderate Alzheimer's disease

Started patient dosing in the phase IIa trial to test safety, feasibility, tolerability and efficacy following daily dosing for 26 weeks


Minerva Neurosciences Inc., of Waltham, Mass.


Dual 5-HT2A /Sigma 2 antagonist


All doses tested up to 256-mg phase III dose appear safe and well-tolerated with no significant changes in cardiac repolarization and QTc intervals


Opiant Pharmaceuticals Inc., of Santa Monica, Calif.


Naloxone nasal spray

Bulimia nervosa

Last patient visit occurred Nov. 2, and top-line data are expected in the first quarter of 2019


Recro Pharma Inc., of Malvern, Pa.

I.V. meloxicam

Blocks cyclooxygenase

Pain following open abdominal hysterectomy

Data published in Anesthesia & Analgesia showed significant analgesic effect, with all doses producing statistically significant improvements vs. placebo in summed pain intensity difference over the first 24 hours post-dose and time-weighted pain relief scores over the first 24 hours post-dose; associated with significantly reduced need for rescue medication (total rescue opioid consumption was 42%-71% lower for all evaluated doses vs. placebo); 3 doses (15 mg, 30 mg and 60 mg) were associated with significantly lower overall rescue opioid consumption vs. the morphine group


Sanbio Group, of Tokyo


Cell therapy

Traumatic brain injury

STEMTRA trial met its primary endpoint, showing patients with chronic motor deficits treated with SB-623 cells had statistically significant improvement in motor function vs. control patients, based on Fugl-Meyer Motor Scale; treated patients showed average of 8.7-point improvement from baseline vs. 2.4 points in the control group at 24 weeks


Saniona AB, of Copenhagen, and Cadent Therapeutics, of Cambridge, Mass.


SK positive allosteric modulator

Essential tremor

Started study


Vaccinex Inc., of Rochester, N.Y.

VX-15 (pepinemab)

Anti-semaphorin 4D antibody

Huntington's disease

In the 36-patient cohort A of the Signal study, there were no concerning safety signals and biomarker imaging results were encouraging; company is currently enrolling cohort B



Eyenovia Inc., of New York

Microdose delivery of latanoprost

Prostanoid selective FP receptor agonist

Intraocular pressuring lowering

Full results published in Clinical Ophthalmology showed subjects successfully self-administered high-precision microdose latanoprost 88% of the time after limited training, marking a significant improvement over standard-of-care eyedropper (<50%); single microdose, which uses 75% less drug and preservative than standard eyedrop, achieved 29% IOP lowering from baseline


Nightstar Therapeutics plc, of London


Comprises AAV8 vector containing codon-optimized cDNA designed to produce human RPGR inside the eye

X-linked retinitis pigmentosa

Plans to start phase II/III expansion study to test safety and efficacy in patients with a diagnosis of XLRP due to RPGR mutations, as confirmed by genetic testing; primary endpoint will evaluate changes in retinal sensitivity and secondary endpoints include both anatomical and functional efficacy and safety; study expected to start by end of 2018


Opthea Ltd., of Melbourne, Australia


Soluble form of VEGFR-3

Wet age-related macular degeneration

Completed recruitment in phase IIb study, with 351 treatment-naïve patients enrolled; primary data are expected in the fourth quarter of 2019


Opthea Ltd., of Melbourne, Australia


Soluble form of VEGFR-3

Wet age-related macular degeneration

Last patient enrolled in the phase IIb trial, with total of 366; top-line data expected in the fourth quarter of 2019


Wize Pharma Inc., of Hod Hasharon, Israel


Sodium hyaluronate

Dry eye syndrome in patients with moderate to severe conjunctivochalasis

Top-line results demonstrated statistical significance vs. placebo (p=0.0079) on primary endpoint, defined as the reduction in Lissamine green conjunctival staining (LGCS) score from baseline to 3 months; also demonstrated strong trend toward significance (P=0.0713) with average reduction in LGCS score between baseline and 3 months of -3.5 and -1.6 in the LO2A and placebo groups, respectively



Blaze Bioscience Inc., of Seattle

Tozuleristide (BLZ-100)

Targeting peptide and fluorescent dye

Fluorescence-guided surgery

Enrolled the first patient in the phase II/III trial for detecting pediatric primary central nervous system tumors in subjects receiving tozuleristide and imaged with the Canvas System


Mereo Biopharma Group plc, of London

MPH-966 (alvelestat)

Oral neutrophil elastase inhibitor

Alpha-1 antitrypsin deficiency

Dosed the first patient; top-line data expected in the second half of 2019


Oragenics Inc., of Tampa, Fla.


Human trefoil factor 1

Oral mucositis

Received clearance from the Paul Erlich Institute and the Medicines and Healthcare products Regulatory Agency to enroll patients in Germany and the U.K., respectively, into an ongoing 200-patient placebo-controlled study, measuring the duration, time to development, and overall incidence of oral mucositis



Attenua Inc., of San Mateo, Calif.


Agonist of alpha-7 neuronal nicotinic receptors


Treated first patient in randomized, double-blind, dose-escalation, crossover study testing efficacy and safety in up to 49 patients with refractory chronic cough; additional trials planned for 2019



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