Company (location)







Aivita Biomedical Inc., of Irvine, Calif.


Autologous dendritic cells loaded with autologous tumor-associated antigens


65% of patients had decreases in 27 different biomarkers associated with tumor development


Bio-Path Holdings Inc., of Houston


RNAi nanoparticles targeting Grb2

Acute myeloid leukemia and myelodysplastic syndrome

Amended the study to include patients with high risk MDS and refractory/relapsed AML; one cohort will include the original untreated AML patients and now high risk MDS patients and a second cohort will include refractory/relapsed AML and high risk MDS patients


Delmar Pharmaceuticals Inc., of Vancouver, British Columbia


Bifunctional DNA-targeting agent


53% of assessed patients have achieved a complete response; 41% have achieved stable disease


Geron Corp., of Menlo Park, Calif.


Telomerase inhibitor

Lower-risk myelodysplastic syndromes

Opened patient screening for phase III portion of phase II/III Imerge trial in transfusion-dependent patients who are relapsed after or refractory to erythroid stimulating agents; about 170 patients to be enrolled; primary endpoint is 8-week transfusion independence rate


Immunicum AB, of Gothenburg, Sweden


Activated allogeneic dendritic cells

Newly diagnosed, intermediate and poor-prognosis metastatic renal cancer

In the Mereca study, ilixadencel plus Sutent (sunitinib, Pfizer Inc.) produced complete tumor responses in 5 out of 45 patients (11%) compared to 1 out of 25 patients (4%) for Sutent monotherapy; median overall survival hasn't been reached for either arm; 18-month survival was 63% for in the ilixadencel plus Sutent and 66% for Sutent alone


Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa.


DNA-based immunotherapy

HPV-related precancerous lesions of the anus

Completed enrollment in the 24-patient study; preliminary efficacy and safety data expected in the fourth quarter of 2019


Leap Therapeutics Inc., of Cambridge, Mass.


Anti-DKK1 antibody

Advanced gastroesophageal junction and gastric cancer

In combination with Keytruda (pembrolizumab, Merck & Co. Inc.), resulted in higher survival and objective response outcomes in patients whose tumors expressed high levels of DKK1; combination showed 22 weeks median progression-free survival and nearly 32 weeks median overall survival, with a 50% overall response rate and 80% disease control rate in patients who had not received prior anti-PD-1/PD-L1 therapy


Modra Pharmaceuticals BV, of Amsterdam


Oral therapeutic based on docetaxel

Metastatic, castration-resistant prostate cancer

Patient treatment begun


On Target Laboratories Inc., of West Lafayette, Ind.


Folate receptor-targeting ligand linked to near-infrared dye

Lung cancer

Completed study for detection of lung cancer nodules in patients during surgery; top-line data expected in first half of 2020


Rexahn Pharmaceuticals Inc., of Rockville, Md.


Oral, small-molecule nucleoside prodrug activated by UCK2

Metastatic pancreatic cancer

Updated data as of July 24, 2019, showed overall response rate of 23% observed in 40 newly diagnosed patients who have had at least 1 scan in phase IIa study in combination with Abraxane (nab-paclitaxel, Celgene Corp.); company previously reported ORR of 38% in first 24 patients with at least 1 scan; preliminary data indicate median progression-free survival is about 5.4 months


Sellas Life Sciences Group, of New York



Ductal carcinoma in situ of the breast

Completed enrollment in Vadis trial testing combination with GM-CSF in women who are HLA-A2+ or A3+, express HER2 at HIC 1+, 2+ or 3+ levels and are pre- or postmenopausal


Trovagene Inc., of San Diego


Serine/threonine polo-like-kinase 1 inhibitor

Metastatic castration-resistant prostate cancer

In 4 AR-V7 positive patients there was an immediate decrease in serum PSA levels, including a patient who had a 5-fold rise in PSA in the 2 months prior to enrollment; 2 patients achieved disease control; in arm B with a two-week dosing schedule and 50% greater drug exposure to onvansertib, the initial 3 patients had PSA stabilization or reduction



Altavant Sciences Inc., of Cary, N.C.

Rodatristat ethyl

Tryptophan hydroxylase inhibitor

Pulmonary arterial hypertension

Treated first of about 36 patients in the Elevate 1 study to test the safety and tolerability of the drug; effects on biomarkers of target engagement, exercise capacity, breathlessness and World Health Organization functional class will also be measured


Idorsia Ltd., of Allschwil, Switzerland


Dual endothelin receptor antagonist


In parallel-group study that randomized 490 individuals, where 430 completed double-blind treatment period, decreases in sitting systolic/diastolic automated office blood pressure from baseline to week 8 were 10.3/6.3, 15/9.9, 18.5/12 and 15.1/10 mmHg for aprocitentan 5, 10, 25 and 50 mg, respectively, vs. 7.7/4.9 mmHg for placebo and 12.8/8.4 mmHg for lisinopril; no changes in heart rate or body weight were seen for any dose of aprocitentan


Myokardia Inc., of South San Francisco


Myosin inhibitor

Hypertrophic cardiomyopathy

Data on 12 participants evaluable at 36 weeks from Pioneer-OLE study that enrolled 13 individuals with symptomatic, obstructive disease from phase II Pioneer-HCM study showed treatment resulted in reductions in resting and provoked left ventricular outflow tract gradient while maintaining left ventricular ejection fraction above 55% at all times of assessment; NT-proBNP, an established circulating blood marker of cardiac wall stress, decreased about 10-fold to mean value of 186 pg/mL (normal considered < 125 pg/mL); E/e', a non-invasive marker of left ventricular filling pressure, decreased from 12.8 to 8.5 (normal range is < 8)


Tenax Therapeutics Inc., of Morrisville, N.C.


Calcium sensitizer

Pulmonary hypertension and heart failure with preserved ejection fraction

The HELP study has enrolled 8 of 36 expected patients; 3 patients have completed the 6-week protocol and have rolled over to the 2-year extension study; study has 12 activated sites with 3 additional sites expected to be activated over the next few weeks; top-line data expected in the first quarter of 2020



Regenerx Biopharmaceuticals Inc., of Rockville, Md.


Thymosin-beta4-based dermal gel

Epidermolysis bullosa

First patient enrolled responded positively, with complete wound healing achieved in RGN-137-treated wound but not with placebo-treated wound; study ongoing



Akcea Therapeutics Inc., of Boston, Mass., and Ionis Pharmaceuticals Inc., of Carlsbad, Calif.

Waylivra (volanesorsen)

Antisense targeting ApoC-III

Familial partial lipodystrophy

In the 40-patient study, Waylivra reduced triglyceride levels at 3 months by a mean of 88%, compared to a 22% reduction in placebo-treated patients (p<0.001); significant triglyceride lowering maintained through 12 months; liver fat was reduced by 51.9% in patients treated with Waylivra compared to a 1.5% increase for placebo (p=0.004)


Daiichi Sankyo Europe GmbH, of Munich

Bempedoic acid

ATP citrate lyase inhibitor

Hypercholes-terolemia with type 2 diabetes

Bempedoic acid plus ezetimibe lowered low-density lipoprotein cholesterol (LDL-C) by 40% compared to ezetimibe alone; 39% of patients achieved LDL-C <70 mg/dl; 41% of patients had at least a 50% reduction in LDL-C; high-sensitivity C-reactive protein was reduced by 25% (p<0.001) compared to ezetimibe alone; no differences in hemoglobin A1c


Eloxx Pharmaceuticals Inc., of Waltham, Mass.


Eukaryotic ribosomal selective glycoside


Started Study 003, a single-arm, open-label study designed to assess safety, tolerability, pharmacokinetics and pharmacodynamics in patients with nephropathic cystinosis with at least 1 nonsense mutation in the cystinosin gene



Allakos Inc., of Redwood City, Calif.


Antibody targeting siglec-8

Eosinophilic gastritis and/or eosinophilic gastroenteritis

In 39 patients treated with the high or low dose of the drug, there was a 95% reduction in gastrointestinal tissue eosinophils compared to a 10% increase in the 20 patients treated with placebo (p<0.0001); 69% of patients treated were responders, defined as greater than a 75% reduction in biopsy eosinophil counts and a greater than 30% reduction in Total Symptom Score (TSS), compared to 5% for placebo (p=0.0008); TSS reduced by 53% compared to 24% for placebo (p=0.0012); 93% of the 14 patients with eosinophilic esophagitis had esophageal eosinophils reduced to <5 Eos/HPF and dysphagia was reduced by 53% on AK-002, compared to 17% on placebo


Immunic Inc., of San Diego


Blocks dihydroorotate dehydrogenase

Primary sclerosing cholangitis

Enrolled first of 30 patients in an investigator-sponsored study; primary endpoint is the change in serum alkaline phosphatase at 6 months compared to baseline


Immuron Ltd., of Melbourne, Australia


Bovine colostrum

Severe alcoholic hepatitis

Top-line results showed neither dose (2,400 mg or 4,800 mg) demonstrated that during 28-day treatment period there was no statistically significant reduction of serum endotoxin levels or markers of liver injury in treatment groups vs. placebo


Index Pharmaceuticals Holding AB, of Stockholm


Toll-like receptor 9 agonist

Moderate to severe ulcerative colitis

In the 213-patient Conduct study, 21.4% of patients taking 250 mg x 2 achieved clinical remission, defined by modified Mayo score, at week 6, compared to 6.8% for placebo (p=0.0495)


Landos Biopharma Inc., of Blacksburg, Va.


Targets the lanthionine synthetase C-like 2 pathway

Mild to moderate ulcerative colitis

Treated first of 195 patients in the study comparing 2 doses of the drug to placebo; primary endpoint is remission rate at week 12, as defined by total Mayo score of 2 or less with all subscores of 1 or less


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


Ammonia scavenger

Hepatic cirrhosis and hepatic encephalopathy

First of about 48 patients enrolled in a phase IIa study measuring changes in plasma ammonia concentration for 3 dosing regimens of MNK-6106 compared to rifaximin


Poxel SA, of Lyon, France


Adenosine monophosphate-activated protein kinase activator

Nonalcoholic steatohepatitis

Started a pharmacokinetic/pharmacodynamic (PK/PD) study as part of the ongoing efficacy and safety study; PK/PD data expected in fourth quarter of 2019; efficacy and safety data expected in second quarter of 2020



Beyondspring Inc., of New York


Sequesters tubulin heterodimers


In the phase II portion of the BPI-2358-105 study, the 20 mg/m2 dose over four treatment cycles improved patient reported quality of life measurements for global health status (p<0.0001), symptom scale (p<0.009), summary score (p<0.02), fatigue (p<0.03), pain (p<0.03) and insomnia (p<0.05)



Aimmune Therapeutics Inc., of Brisbane, Calif.


Characterized oral desensitization immunotherapy

Egg allergy

Randomized first of about 84 participants, ages 4 to 26, with hen egg allergy into U.S. study evaluating efficacy and safety using food challenge for assignment 2-to-1 to treatment or placebo; following initial low doses, dosing will be escalated gradually to fixed therapeutic dosing at 300 mg/day dried egg white protein; primary endpoint is proportion of those treated with AR-201 compared with placebo who tolerate single highest dose of at least 1,000 mg dried egg white protein with no more than mild allergy symptoms at exit food challenge


Emergo Therapeutics Inc., of Durham, N.C.


Metabolite of ketotifen, a second-generation H1-antihistamine and mast cell stabilizer

Seasonal allergies

Completed phase IIa study, showing statistical significance in alleviating allergy symptoms; phase IIb study in flu and flu-like illness expected to start in fourth quarter of 2019


Kadmon Holdings Inc., of New York


Selective oral inhibitor of Rho-associated coiled-coil kinase 2

Chronic graft-vs.-host disease

Open-label, registration study is fully enrolled; outcomes from planned interim analysis expected before year-end


Kalytera Therapeutics Inc., of San Francisco



Acute graft-vs.-host disease

1 of 12 patients in the low-dose cohort and none of the patients in the medium-dose cohort developed grades 2-4 acute GVHD; high-dose cohort won't be started; plans to run a phase III study


Mesoblast Ltd., of New York


Mesenchymal stromal cells from bone marrow of unrelated donor

Children with steroid-refractory chronic graft-vs.-host disease

Plans to provide product for an investigator-initiated study


Momenta Pharmaceuticals Inc., of Cambridge, Mass.


FcRn inhibitor

Warm autoimmune hemolytic anemia

Launched phase II/III adaptive design trial



Arrowhead Pharmaceuticals Inc., of Pasadena, Calif.

JNJ-3989 (formerly ARO-HBV); JNJ-6379

Liver-targeted investigational antiviral therapeutic for subcutaneous injection designed to treat work via the ribonucleic acid interference mechanism; orally administered capsid assembly modulator of the class that forms normal capsid structures

Hepatitis B

Partner Johnson & Johnson, of Whitehouse Station, N.J., began dosing with combination regimens, with a nucleos(t)ide analog, triggering $25 million milestone payment from Janssen Pharmaceuticals Inc. to Arrowhead.


Dare Bioscience Inc., of San Diego


Thermosetting bioadhesive vaginal gel containing clindamycin phosphate 2%

Bacterial vaginosis

Investigator-initiated proof-of-concept study showed clinical cure rate of 86% at test-of-cure endpoint


Evofem Biosciences Inc., of San Diego


Vaginal pH regulator

Prevention of acquisition of chlamydia and gonorrhea

Completed the 860-patient Amprevence study; top-line results expected in November 2019


Polypid Ltd., of Petah Tikva, Israel


Extended-release, broad-spectrum antibiotic

Prevention of post-abdominal surgery incisional site infections

Completed enrollment in study measuring incisional infection over 30 days; data expected in October 2019


Squarex Inc., of St. Paul, Minn.


Topical formulation of immunomodulator squaric acid dibutyl ester

Recurrent herpes labialis

Results from 139-subject trial showed single dose applied to upper arm significantly extended time to next outbreak and reduced both frequency and severity of outbreaks; greatest effects recorded during days 43 to 121 following application; phase III study planned after end-of-phase II FDA meeting later this year



Antibe Therapeutics Inc., of Toronto


Hydrogen sulfide-releasing derivative of the nonsteroidal anti-inflammatory drug naproxen

Osteoarthritis of the knee

The 360-patient study will complete enrollment in the 2Q19 rather than 3Q19 as previously guided



Alkahest Inc., of San Carlos, Calif.


Plasma fractions

Primary hip or knee arthroplasty

Treated first of about 45 patients in the AKST6021-211 study assessing post-surgery recovery and immunological profile at various timepoints


Fulcrum Therapeutics Inc., of Cambridge, Mass.


P38alpha/beta mitogen activated protein kinase inhibitor

Facioscapulo-humeral muscular dystrophy

Started phase IIb ReDUX4 study testing the ability of losmapimod to reduce DUX4-driven gene expression over 24 weeks; data expected in third quarter of 2020


Santhera Pharmaceuticals Holding AG, of Pratteln, Switzerland


Dissociative steroid

Duchenne muscular dystrophy

Results from the VBP15-003 extension study published in Neurology showed mean difference in change for time to stand from supine from baseline to week 24 was significant for the 2.0 and 6.0 mg/kg/day groups compared to the 0.25 mg/kg/day group (p=0.02 and p=0.04, respectively); mean change was also significant for 2.0 mg/kg/day group compared to an untreated comparator cohort (p=0.04); time to run/walk 10 meters, time to climb stairs, 6-minute walk test and North Star Ambulatory Test improved based on dose; treatment increased serum levels of osteocalcin; biomarkers for adrenal suppression and insulin resistance were better than published studies of corticosteroid therapy



Alkahest Inc., of San Carlos, Calif.


Plasma fraction

Mild to moderate Alzheimer's disease dementia

Patients treated for 5 days in week 1 and 5 days in week 13 had no decline in cognition, based on the 11-item AD Assessment Scale-cognitive subscale and the Mini-Mental State Examination, and negligible decline in function by the AD Cooperative Study Activities of Daily Living scale 23-item version


Emalex Biosciences Inc., of Chicago


Blocks dopamine action at the D1 receptor

Tourette syndrome

Enrolled first of about 150 patients in phase IIb study comparing ecopipam to placebo over 12 weeks; primary endpoint is Yale Global Tic Severity Scale score


Eton Pharmaceuticals Inc., of Deer Park, Ill.


Liquid formulation of an undisclosed FDA-approved drug used to treat a severe neurological condition

Healthy subjects

In a bioequivalence study, ET-104 demonstrated pharmacokinetic equivalence to an FDA-approved oral solid product with the same active ingredient; plans to file an NDA in fourth quarter of 2019


Harvest One Inc., of Vancouver, British Columbia

CBD Gelpell

Cannabidiol in a gelatine bead


Median reduction of monthly seizures was -82% in the 12-week treatment period in treatment-resistant children when added to current medications


Irlab Therapeutics AB, of Stockholm


Targets dopamine D3 receptor

Parkinson's disease dyskinesia

Analysis provided further evidence of the drug candidate's ability to significantly and meaningfully improve daytime movement quality


Neurotrope Inc., of New York


Activator of protein kinase C epsilon

Moderate to severe Alzheimer's disease

Top-line data from the study are expected in the third quarter


Retrotope Inc., of Los Altos, Calif.


Deuterated polyunsaturated fatty acid

Infantile neuroaxonal dystrophy

Fully enrolled all patients in open-label phase II/III study


Zynerba Pharmaceuticals Inc., of Devon, Pa.


Cannabidiol gel

Fragile X syndrome

Data from the FAB-C study showed the drug produced a significant 12-week improvement over baseline on several different scores



Opthea Ltd., of Melbourne, Australia


Soluble form of vascular endothelial growth factor receptor 3

Treatment-naive wet age-related macular degeneration

Patients treated with 2 mg of OPT-302 plus Lucentis (ranibizumab, Roche Holding AG) gained a mean of 14.2 letters of vision on the Early Treatment of Diabetic Retinopathy Study standardized eye chart at 24 weeks, compared to an improvement of 10.8 letters for Lucentis alone (p=0.0107) in phase IIb trial; 0.5-mg OPT-302 dose plus Lucentis produced a 9.4-letter improvement; 45% of patients treated with higher dose gained 15 or more letters, compared to 40.5% of patients treated with Lucentis alone; 70% and 57.8% of patients gained 10 or more letters for high-dose OPT-302 combination and Lucentis alone, respectively; mean central subfield thickness was reduced by 147 µm for high-dose combination, compared to a 134 µm reduction for Lucentis alone


Oxurion NV, of Leuven, Belgium


Antibody targeting placental growth factor

Diabetic macular edema

At month 3 in the 70-patient phase IIa study, treatment with THR-317 plus Lucentis (ranibizumab, Roche Holding AG/Novartis AG) improved Best Corrected Visual Acuity by a mean of 8.71 letters compared to 8.18 letters increase for patients treated with Lucentis alone; in patients with poor or no response to prior anti-VEGF therapy, the combination produced an 8.08 increase in letters, compared to 6.43 letters increase for Lucentis; in patients with a baseline of 65 letters or less, combination produced an increase of 11.14 letters compared to 8.88 letters for Lucentis


Topivert Pharma Ltd., of London


Anti-inflammatory kinase inhibitor

Dry eye disease

Phase IIb/III Theia-1 study didn't meet primary endpoints of ocular grittiness and total ocular surface staining at day 29; ocular discomfort, eye dryness and the Ocular Surface Disease Index were improved for drug compared to placebo



Arrowhead Pharmaceuticals Inc., of Pasadena, Calif.


Second-generation RNAi therapeutic

Alpha-1 antitrypsin deficiency

Dosed first patient in potentially pivotal Sequoia phase II/III trial; 120-patient, adaptive design study will identify single dose level in part A to be studied in part B, which will evaluate proportion of treated patients relative to placebo achieving a 2-point improvement in histologic grading scale of AATD-associated liver disease and no worsening of liver fibrosis on end-of-study biopsy



Bellerophon Therapeutics Inc., of Warren, N.J.


Pulsatile nitric oxide delivery system

Pulmonary hypertension associated with interstitial lung disease

Completed enrollment in cohort 2 of ongoing phase II/III trial


Eloxx Pharmaceuticals Inc., of Waltham, Mass.


Small molecule designed to restore production of full-length functional proteins

Cystic fibrosis

IND open; protocol endorsed by Cystic Fibrosis Foundation


Knopp Biosciences LLC, of Pittsburgh


Affects eosinophil maturation in bone marrow

Moderate to severe eosinophilic asthma

Started study testing 3 doses of the drug in about 100 patients; primary endpoint is change in blood absolute eosinophil count from baseline to week 12; secondary endpoints include changes in pre-bronchodilator FEV1 and asthma control outcomes (ACQ-7 questionnaire) from baseline to week 12; data expected in second half of 2020


Verona Pharma plc, of London


Dual PDE3/4 inhibitor in dry powder inhaler formulation

Maintenance treatment of chronic obstructive pulmonary disease

Primary endpoint met, showing statistically significant and clinically meaningful dose-dependent improvement in lung function; secondary lung function endpoints met, with data supportive of twice-daily dosing



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