Aduro Biotech Inc., of Berkeley, Calif.

ADU-S100 (MIW-815)

STING pathway activator

Recurrent or metastatic head and neck squamous cell carcinoma

Dosed first patient in a trial testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.) as first-line treatment; planned population will consist of 33 adults with PD-L1-positive disease


Apogenix AG, of Heidelberg, Germany


CD95 antagonist

Solid tumors

Review in Cancer Management and Research suggested study drug may offer treatment option for individuals with malignancies beyond glioblastoma, where proof-of-concept study showed 5-year overall survival rate of 7% for those dosed with asunercept in combination with radiotherapy compared to 0% for those treated with radiotherapy alone


Aslan Pharmaceuticals Pte. Ltd., of Singapore


EGFR/ErbB-2 (HER2/neu) inhibitor

Biliary cancer

Patients treated with varlitinib plus capecitabine in second-line achieved a response rate of 11%, including 2 complete responses; median progression-free survival of 2.7 months and overall survival of 5.8 months


Aveo Oncology Inc., of Cambridge, Mass., and Eusa Pharma Inc., of Hemel Hempstead, U.K.

Fotivda (tivozanib)

VEGF receptor tyrosine kinase inhibitor

Advanced or metastatic renal cell carcinoma

Fotivda plus Opdivo (nivolumab, Bristol-Myers Squibb Co.) produced an overall median progression-free survival of 18.9 months in 25 patients treated at the study's full dose and schedule; median PFS for 12 previously untreated patients was 18.5 months, while median PFS for 13 previously treated patients had not yet been reached; objective response rate was 56%, including 1 complete response in a naïve patient; disease control rate was 96%


Beigene Ltd., of Beijing


Anti-PD-1 antibody

Advanced lung cancer

Tislelizumab plus a doublet chemotherapy produced overall response rate in 36 patients of 66.7%; ORR was 43.8% (7/16) in patients with nonsquamous non-small-cell lung cancer (NSCLC), 80% (12/15) in patients with squamous NSCLC (cohort A), 66.7% (4/6) in patients with squamous NSCLC (cohort B) and 76.5% (13/17) in patients with small-cell lung cancer (SCLC); median progression-free survival was 9 months in patients with nonsquamous NSCLC, 7 months in squamous NSCLC (cohort A), 6.9 months in SCLC, and in squamous NSCLC (cohort B) the median PFS had not yet been reached


Beigene Ltd., of Beijing


Anti-PD-1 antibody

Advanced esophageal, gastric or gastroesophageal junction carcinoma

Patients treated with tislelizumab plus cisplatin and fluorouracil had an overall response rate of 46.7%, including 7 of 15 patients with a partial response; median duration of response was 12.8 months; median progression-free survival was 10.4 months; median overall survival had not been reached


Bergenbio ASA, of Bergen, Norway


AXL tyrosine kinase receptor inhibitor

Non-small-cell lung cancer

Combination therapy in immuno-oncology-naive participants was well-tolerated in chemorefractory population, with most common adverse events including fatigue (48%), increased transaminases (43%) and diarrhea (33%)


Beyondspring Inc., of New York


Sequesters tubulin heterodimers

Breast cancer

Increasing Neulasta (pegfilgrastim, Amgen Inc.) doses caused a statistically significant dose-dependent increase in thrombocytopenia (p<0.0001 for all grade thrombocytopenia); at 6 mg of Neulasta, the frequency of grades 1, 2 and 3 thrombocytopenia were 56%, 19% and 19%, respectively, compared to 20%, 7% and 0%, respectively for patients treated with plinabulin (20mg/m2)


Can-Fite Biopharma Ltd., of Petach Tikva, Israel


Binds A3 adenosine receptor

Second-line advanced hepatocellular carcinoma

Median overall survival wasn't improved for whole study population; in the subpopulation of 56 patients with Child Pugh B7 cirrhosis, median OS was 6.8 months for patients treated with namodenoson compared to 4.3 months for placebo; partial response rate was 9% for namodenoson and 0% for placebo; 1-year survival was 44% for namodenoson and 18% for placebo (p=0.028)


Debiopharm International SA, of Lausanne, Switzerland


Apoptosis protein inhibitor

Head and neck cancer

Following completion of 2-year follow-up, randomized, double-blind study in high-risk, previously untreated patients with locally advanced squamous cell carcinoma of the head and neck met primary endpoint of locoregional control along with prolonged progression-free survival


Generex Biotechnology Corp., of Miramar, Fla.


Peptide vaccine

Metastatic triple-negative breast cancer

Enrolled first patient in trial to test safety and tolerability of AE-37 in combination with Keytruda (pembrolizumab, Merck & Co. Inc.), as well as objective response rate


Idera Pharmaceuticals Inc., of Exton, Pa.


Toll-like receptor 9 agonist

Solid tumors

Started open-label Illuminate-206 trial, combining drug with ipilimumab; primary endpoint is objective response rate


Imaginab Inc., of Los Angeles


T-cell surface glycoprotein CD8 tracer

Imaging agent

First participant enrolled in study designed to visualize immune system using whole-body in vivo PET imaging of CD8 T cells before and after immunotherapy-based treatment


Immunicum AB, of Göteborg, Sweden


Allogeneic dendritic cell-based vaccine

Metastatic renal cell carcinoma

Top-line data from Mereca trial (n = 88) showed that survival as of July 2019 was 57% (32 of 56) for ilixadencel plus sunitinib (Sutent, Pfizer Inc.) vs. 43% (13 of 30) for sunitinib alone; objective response rate was 44% (20 of 45) for ilixadencel combo vs. 48% (12 of 25) for sunitinib monotherapy; at 18-month follow-up, combo group showed longer median duration of response than sunitinib monotherapy (7.1 vs. 2.9 months) and higher percentage of ongoing response (60%, or 12 of 20, vs. 33%, or 4 of 12, respectively)


Immutep Ltd., of Sydney

Eftilagimod alpha (IMP-321)

HLA class II antigen stimulator

Head and neck squamous cell carcinoma; non-small-cell lung cancer

Interim analysis of cohort 1 in part A (first-line NSCLC) of TACTI-002 combo trial evaluating drug and Keytruda (pembrolizumab, Merck & Co. Inc.) showed 7 of 17 (41.2 %) participants achieved partial response according to RECIST 1.1 as of data cut-off of Sept. 6; 6 additional participants had disease stabilization as best overall response, for disease control rate of 76.5 %; data monitoring committee recommended opening cohort 2 (n = 19); recruitment of first cohorts in parts B and C continues


Infinity Pharmaceuticals Inc., of Cambridge, Mass.


Inhibits phosphoinositide-3-kinase-gamma

Front-line triple-negative breast cancer and front-line renal cell cancer

Started the Mario-3 study testing PI-549 plus Tecentriq (atezolizumab, Roche Holding AG) with and without Abraxane (nab-paclitaxel, Celgene Corp.)


Ipsen SA, of Paris

Onivyde (liposomal irinotecan)

Topoisomerase I inhibitor

Small-cell lung cancer

Best overall response (partial response plus stable disease) was 72% with an objective response rate of 44%; 44% (11/25) of patients achieved a partial response with 68% of patients (17/25) experiencing tumor shrinkage


Janssen Pharmaceutical Cos., part of New Brunswick, N.J.-based Johnson & Johnson

Darzalex (daratumumab)

CD38-directed antibody

Multiple myeloma

Results from Griffin study showed addition of Darzalex to bortezomib, lenalidomide and dexamethasone (VRd) induced higher response rates in newly diagnosed patients eligible for high-dose therapy and autologous stem cell transplantation vs. VRd alone; at end of 6 cycles, 42% in Darzalex/VRd arm hit primary endpoint of stringent complete response vs. 32% for VRd alone (p=0.1359, meeting prespecified 2-sided alpha of 0.2 for positive study); overall response in Darzalex/VRd group was 99% vs. 92% for VRd-only; minimal residual disease activity for patients achieving complete response or greater was 59% for Darzalex/VRd group vs. 24% for VRd group


Janssen Pharmaceutical Cos., part of New Brunswick, N.J.-based Johnson & Johnson

Darzalex (daratumumab)

CD38-directed antibody

Multiple myeloma

Data from Pleiades study in both newly diagnosed and relapsed/refractory patients showed a subcutaneous formulation delivered in combination with standard-of-care regimens showed similar clinical activity and safety to I.V. regimens


Kura Oncology Inc., of San Diego


Protein farnesyltransferase inhibitor

Urothelial carcinoma

Top-line data from ongoing investigator-sponsored single-arm trial designed to enroll at least 18 participants with relapsed/refractory HRAS mutant disease at single site in in Korea showed that 5 of 13 evaluable participants had confirmed objective responses, according to RECIST 1.1 criteria, for overall response rate of 38%; 4 experienced progression-free survival of > 6 months


Leap Therapeutics Inc., of Cambridge, Mass.


Anti-Dickkopf-1 antibody

Advanced gynecological malignancies

Findings from P204 study showed, as of data cut-off, 1 patient with Wnt signaling alterations had her monotherapy partial response deepen into complete response; across study, patients with Wnt activating mutations demonstrated longer progression-free survival (n=21, 175 days) vs. those without Wnt activating mutations (n=67, 63 days); patients with DKK1-high tumors had longer PFS


Lidds AB, of Uppsala, Sweden

Liproca Depot

Androgen receptor antagonist

Hormone-dependent prostate cancer

Preliminary results from phase IIb dose-finding study showed strong maximum prostate-specific antigen (PSA) decrease and sustained PSA reduction, with maximum reduction during months 2-4


Medicenna Therapeutics Corp., of Toronto


IL-4-guided toxin

Recurrent glioblastoma

Median overall survival was 11.8 months in 21 patients treated at low doses in phase IIb trial; first 12 of 25 patients receiving the high dose had a median OS of 16.7 months; median OS of 13.7 months for 12 patients with high IL-4 receptor expression treated with the low doses compared to 8.1 months for 8 patients expressing no/low IL-4R


Merck KGaA, of Darmstadt, Germany


MET tyrosine kinase inhibitor

Locally advanced or metastatic non-small-cell lung cancer

Study combining drug with tyrosine kinase inhibitor osimertinib is open for enrollment; recruiting patients with EGFR-mutated, MET-amplified, locally advanced or metastatic disease with acquired resistance to prior EGFR tyrosine kinase inhibitor therapy


Momotaro-Gene Inc., of Okayama, Japan


Gene therapy delivering the reduced expression in immortalized cells/dickkopf-3 gene

Relapsed malignant pleural mesothelioma

Treated first of up to 12 patients in the study testing MTG-201 with a PD-1 inhibitor; primary endpoint is objective response rate; disease stabilization, duration of response, progression-free survival and overall survival will be measured as secondary endpoints; safety and the exploratory biomarkers of activity and the immunogenicity of MTG-201 will also be measured


Oncopeptides AB, of Stockholm


Lipophilic peptide-conjugated alkylator

Multiple myeloma

Interim data from pivotal Horizon trial showed activity in patients with relapsed/refractory disease, many of whom also have extramedullary disease; overall response rate was 28% and clinical benefit rate was 40%; 86% of evaluated patients achieved disease stabilization or better


Oncopeptides AB, of Stockholm


Lipophilic peptide-conjugate alkylator

Relapsed/refractory multiple myeloma

Interim data from pivotal Horizon study in patients with extramedullary disease (EMD) showed overall response rate of 23% with melflufen plus dexamethasone; EMD patients who responded had median survival of 18.5 months vs. 5.1 months for EMD patients who did not respond


Oncopeptides AB, of Stockholm


Lipophilic peptide-conjugated alkylator

Relapsed/refractory multiple myeloma

Last patient was included in OP-106 Horizon pivotal study testing combination with dexamethasone


Oryzon Genomics SA, of Madrid, Spain


Small-molecule inhibitor of epigenetic enzyme LSD1

Relapsed small-cell lung cancer

Administration of combination of iadademstat with standard of care during the first 6 cycles produced tumor reduction of 78.7%, as defined by RECIST criteria


Solasia Pharma KK, of Tokyo


Mitochondrial targeted agent

Relapsed or refractory peripheral T-cell lymphoma

Patient registration reached target number of cases in Japan, South Korea, Taiwan and Hong Kong; pivotal study is testing monotherapy, with tumor response as primary outcome measure; results expected in 2020


TG Therapeutics Inc., of New York


Glycoengineered monoclonal antibody targeting an epitope on the CD20 antigen found on mature B-lymphocytes

Chronic lymphocytic leukemia intolerant to prior BTK or PI3K delta inhibitor therapy

Estimated median progression-free survival of 50 evaluable patients was 23.5 months; overall survival had not been reached at a median follow-up of 15.7 months; 58% of patients have been on umbralisib for a duration longer than their prior BTK or PI3k inhibitor


Theralase Technologies Inc., of Toronto


Light activated photodynamic compound

Bacillus Calmette-Guérin unresponsive or intolerant non-muscle invasive bladder cancer

First of about 100 patients treated in study measuring complete response rate and duration of response


Tiziana Life Sciences plc, of New York


Inhibitor of several cyclin dependent kinases

Advanced unresectable or metastatic hepatocellular carcinoma resistant or intolerant to sorafenib

Median time to progression and progression-free survival were both 5.9 months in phase IIa trial; 60.7% of the 28 patients had stable disease and 1 patient (3.6%) had a partial response


Trovagene Inc., of San Diego


Oral PLK1 inhibitor

Acute myeloid leukemia

Initiated enrollment of 32 patients who are either treatment-naïve and not candidates for induction therapy or who have relapsed following up to 1 prior regimen, to receive onvansertib in combination with decitabine; primary endpoint of objective response will be assessed in patients who complete at least 1 cycle


Tyme Technologies Inc., of New York

SM-88 (racemetyrosine)

Modified tyrosine derivative

Metastatic pancreatic cancer

Patients achieved at least an 80% reduction in circulating tumor cell burden, demonstrated a 60% decrease in risk of death


Urogen Pharma Ltd., of New York


Sustained-release mitomycin C gel

Bladder cancer

Trial completed enrollment; among interim cohort of 32 patients, 63% achieved a complete response


VBI Vaccines Inc., of Cambridge, Mass., and Glaxosmithkline plc, of London

VBI-1901 with AS-01B adjuvant system

Cancer vaccine immunotherapeutic

Recurrent glioblastoma

Plans to add a 10-patient arm to part B of the ongoing study to test the combination of VBI's VBI-1901 with GSK's AS-01B adjuvant system, which will begin enrolling in the second half of 2019



Phasebio Pharmaceuticals Inc., of Malvern, Pa.


Monoclonal antibody antigen-binding fragment


Drug achieved immediate and sustained reversal of ticagrelor in older (ages 50-64) and elderly (ages 65-80) patients; data to be presented at an upcoming medical congress


Recardio Inc., of San Francisco


Small-molecule DPP-4 inhibitor

ST-segment elevation myocardial infarction

Launched enrollment in trial testing combination with filgrastim in patients in early recovery post-myocardial infarction; outcomes expected in early 2020



Concert Pharmaceuticals Inc., of Lexington, Mass.


Dual JAK1/JAK2 kinase inhibitor

Alopecia areata

Randomized, double-blind, sequential dose trial of participants with moderate to severe disease treated with 8 mg or 12 mg twice daily met primary endpoint of percentage of patients achieving ≥ 50% relative change from baseline at 24 weeks using Severity of Alopecia Tool (SALT), with statistically significant differences of 47% and 58%, respectively, compared to placebo response of 9% (p<0.001); these dose cohorts also differed from placebo in number of participants achieving ≥ 75% and ≥ 90% relative change in SALT from baseline at 24 weeks and in rating significantly greater improvement on Patient Global Impression of Improvement Scale


Concert Pharmaceuticals Inc., of Lexington, Mass.


Oral JAK1/2 inhibitor

Alopecia areata

Completed patient enrollment of open-label trial testing once-daily 24-mg dosing vs. twice-daily 12-mg dosing; top-line data expected in first half of 2020


Foamix Pharmaceuticals Ltd., of Rehovot, Israel


Combination of minocycline and adapalene, as a topical foam

Acne vulgaris

First patient enrolled out of planned 400 participants, ages 12 and older, with moderate to severe acne vulgaris; testing vs. adapalene, minocycline and vehicle foam; primary endpoint is proportion of patients achieving "clear" or "minimal" IGA rating at 12 weeks


Menlo Therapeutics Inc., of Redwood City, Calif.


NK1 receptor antagonist

Chronic pruritus of unknown origin

Completed enrollment of 233 patients; results expected in the first quarter of 2020



Aeglea Biotherapeutics Inc., of Austin, Texas


Arginase-I stimulator

Arginase 1 deficiency

10 participants dosed subcutaneously in ongoing phase II open-label extension trial; subcutaneous administration shown to control plasma arginine similarly to intravenous administration and well-tolerated, with no patient discontinuations


Agios Pharmaceuticals Inc., of Cambridge, Mass.

Mitapivat (AG-348)

Oral, small-molecule allosteric activator of wild-type and mutated pyruvate kinase-R enzymes

Pyruvate kinase deficiency

Data from core and extension phases of Drive PK study, published in The New England Journal of Medicine, showed 26 of 52 patients (50%) achieved clinically significant maximum hemoglobin increase of >1 g/dL in the Core Period with improvement in other markers of hemolysis as of data cutoff; in patients with hemoglobin increases of >1 g/dL in Core Period, the mean maximum hemoglobin increase was 3.4 g/dL (range, 1.1–5.8 g/dL); median time to first hemoglobin increase of >1 g/dL was 10 days (range 7–187 days)


Bluebird Bio Inc., of Cambridge, Mass.


Gene therapy

Cerebral adrenoleukodystrophy

Updated data from phase II/III Starbeam study in boys 17 and under show, of those who reached 24 months of follow-up, 88% (15/17) continue to be alive and major functional disability (MFD)-free; of 14 patients with less than 24 months follow-up, no evidence of MFDs


Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Zealand Pharma A/S, of Copenhagen, Denmark


Dual-acting glucagon-like peptide 1 GLP-1/glucagon agonist

Type 2 diabetes, obesity

BI will initiate phase II development of agent in-licensed from Zealand, initially in dose-finding, placebo and active comparator proof-of-concept trial


Dance Biopharm Holdings Inc., of Durham, N.C.


Second-generation aerosol inhalant formulation of human insulin

Non-insulin-dependent diabetes

Had comparable pharmacodynamic properties and more rapid onset of action compared to insulin lispro, with median differences of 6.5 to 20 min (p<0.02)


Entera Bio Ltd., of Jerusalem

Oral hPTH(1-34)

Parathyroid hormone


Twice-daily, 3 times daily and 4 times daily produced a dose-dependent increase in 1,25-dihydroxyvitamin D; 4 times daily dose also increased serum albumin-corrected calcium and decreased serum phosphate and urinary calcium


Inventiva SA, of Daix, France


Galactosyl-transferase modulator

Mucopolysac-charidosis type VI

Initiated biomarker study examining leukocyte glycosaminoglycan levels in 3 children and skin glycosaminoglycan levels in 3 adults with MPS VI before and following enzyme replacement therapy and in 6 age-matched control subjects to inform ongoing phase IIa Improves study


Matinas Biopharma Holdings Inc., of Bedminster, N.J.


Omega-3 fatty acid

Elevated triglycerides

Started pre-screening patients for the Enhance-it study comparing MAT-9001 to Vascepa (icosapent ethyl, Amarin Cop. Plc); enrollment to begin in the first quarter of 2020 with top-line data expected in the second half of 2020


Oramed Pharmaceuticals Inc., of New York


Oral insulin capsule

Type 2 diabetes

Treated last patient in primary cohort of phase IIb HbA1c trial; top-line data expected in fourth quarter of 2019


Orchard Therapeutics Ltd., of Boston


Gene therapy expressing alpha-L-iduronidase

Mucopolysac-charidosis type I

Supranormal alpha-L-iduronidase enzyme expression seen in peripheral blood with first patient treated achieving levels 10 times normal at 12 months; first 2 patients, with 12 months and 6 months of follow-up, had rapid metabolic correction of glycosaminoglycans levels in the urine and cerebrospinal fluid; first patient had resumed growth, improved motor skills and a stable cognitive score; study expected to enroll 8 patients by the first half of 2020


Spruce Biosciences Inc., of San Francisco


Oral, once-daily corticotropin-releasing factor type-1 antagonist

Congenital adrenal hyperplasia

Phase IIa study in patients with classic CAH accompanied by elevated androgens at baseline showed clinically relevant reductions in all parameters; mean reductions from baseline of 74% for adrenocorticotropic hormone (ACTH), 82% for 17-hydroxyprogesterone and 55% for androstenedione (A4); 60% of patients with elevated, abnormal ACTH and 40% of patients with elevated A4 saw reductions to normalization by week 12


Valbiotis SA, of La Rochelle, France


5 plant extracts


In addition to previously announced reductions in hyperglycemia, body weight and waist size, Valedia reduced triglycerides by 32.2% (p< 0.01), fatty liver index by 18.7%(p<0.001) and LDL cholesterol by 11.7% (p<0.05), all compared to placebo; systolic blood pressure was reduced by 10.57 mmHg (p<0.01) compared to placebo and by 18.86 mmHg (p <0.001) relative to placebo in patients with systolic blood pressure levels higher than 130 mmHg at the start of the study


Vtv Therapeutics Inc., of High Point, N.C.


Glucokinase activator

Type 1 diabetes

In the Simplici-T1 study, 6 patients treated with drug had an 11% increase in time in range per day from baseline to the end of treatment and a 12% increase during waking hours, relative to the 9 patients on placebo; treatment reduced the total daily mealtime bolus insulin dose by 23% compared to 4% for placebo; patients on drug had fewer level 1 (≥54-70 mg/dl) and level 2 (<54 mg/dl) hypoglycemic events than patients in the placebo group


Xeris Pharmaceuticals Inc., of Chicago

Fixed-ratio co-formulation of pramlintide and insulin

Synthetic hormone resembling amylin plus insulin

Type 1 diabetes

Began dosing in open-label, crossover study in 18 adults; primary objective is to evaluate pharmacodynamic and pharmacokinetic properties of single injection vs. single doses of regular insulin and regular insulin plus pramlintide co-administered as separate injections; data expected in first half of 2020



Arena Pharmaceuticals Inc., of San Diego


S1P receptor modulator

Ulcerative colitis

Steady-state trough concentrations achieved and maintained from weeks 1 to 12 in individuals with moderately to severely active disease dosed with 1 mg or 2 mg once-daily; statistically significant exposure-response relationships favoring 2-mg dosing were shown, including reductions from baseline in circulating lymphocyte count and improvements in modified Mayo Clinic Score


Cindome Pharma Inc., of Cincinnati


Deuterated domperidone; dopamine D2 receptor antagonist


Enrolled first patient


Durect Corp., of Cupertino, Calif.


Epigenetic regulator

Alcoholic hepatitis

Completed phase IIa trial, showing that 19 patients treated with DUR-928 had statistically significantly greater reductions from baseline in bilirubin (day 7 and 28) and Model of End-Stage Liver Disease score (day 28), as well as statistically significantly lower Lille scores vs. historical control group


Enanta Pharmaceuticals Inc., of Watertown, Mass.


Farnesoid X receptor agonist

Nonalcoholic steatohepatitis

Top-line results showed phase IIa Argon-1 study achieved primary endpoint with statistically significant ALT reduction of 28 U/L in 2.5-mg arm vs. 15 U/L for placebo at week 12 (p=0.049); statistically significant reduction in liver fat content seen at 2.5-mg dose as measured by MRI-PDFF (p<0.001), and 45% of participants were MRI-PDFF responders (≥ 30% fat reduction)


Enanta Pharmaceuticals Inc., of Watertown, Mass.


Farnesoid X receptor agonist

Primary biliary cholangitis

Company halted enrollment in Intrepid study based on preliminary data from existing participants; study will continue as is based on data safety monitoring board recommendation


Inventiva SA, of Daix, France


PPAR agonist

Nonalcoholic steatohepatitis

Completed recruitment for the phase IIb Native trial, randomizing 247 patients; headline results are expected in the first half of 2020


Lipocine Inc., of Salt Lake City


Testosterone prodrug; androgen receptor agonist

Nonalcoholic steatohepatitis

First patient dosed in Lift study, a paired-biopsy study in confirmed pre-cirrhotic NASH men; top-line primary endpoint is liver fat reduction; data expected in mid-2020


Northsea Therapeutics BV, of Naarden, the Netherlands


Fatty acid

Nonalcoholic steatohepatitis

Treated first patient in the 264-patient Icona study comparing 2 doses of the drug to placebo over 52 weeks; primary endpoint is the resolution of NASH without worsening of fibrosis; secondary endpoints include changes in nonalcoholic fatty liver disease activity score, histological scores for steatosis, ballooning, inflammation and fibrosis, liver enzymes and bilirubin; interim analysis of secondary endpoints from first 90 patients for 16 weeks is planned for mid-2020


Oragenics Inc., of Tampa, Fla.


Trefoil factor 1

Severe oral mucositis

Enrolled 158 of about 200 patients with plans to complete enrollment by the end of 2019; following review of safety data, the data and safety monitoring board recommended the study continue with no adjustments or further review


Genitourinary/Sexual Health

Daré Bioscience Inc., of San Diego

Sildenafil cream, 3%

PDE5 inhibitor

Female sexual arousal disorder

Completed non-interventional content validity study required before initiating at-home, product dosing portion of phase IIb program, expected to begin before year-end following type C meeting with FDA


Vascular Therapies Inc., of Cresskill, N.J.


Sirolimus formulation

Improving vascular access outcomes in kidney disease

Preliminary results included outcomes of 2 cohorts of Access study, with 78% and 73% suitable for dialysis at 6 and 12 months, respectively; median time to first dialysis was 49 days; 12-month secondary patency was 73%; no product-related serious adverse events reported


Xortx Therapeutics Inc., of Calgary, Alberta and Teijin Pharma Ltd., of Tokyo


Non-purine xanthine oxidase inhibitor tablet

Diabetic nephropathy

TMX-049 met primary endpoint, significantly decreasing serum uric acid and urinary albumin proteinuria; no new safety concerns observed



Fibrogen Inc., of San Francisco


HIF prolyl hydroxylase inhibitor

Chemotherapy-induced anemia

First of about 100 anemic cancer patients with non-myeloid malignancy and hemoglobin level at or below 10 g/dL while undergoing myelosuppressive chemotherapy dosed in 16-week study; primary efficacy endpoint is maximum change in hemoglobin level from baseline without red blood cell transfusion; secondary endpoints include hemoglobin change from baseline, % of participants with hemoglobin response and % who require 1 or more red blood cell transfusions



Hansa Biopharma AB, of Lund, Sweden


Cleaves IgG

Sensitized kidney transplant patients

Pooled data of 46 patients showed drug reduced donor-specific antibody (DSA) levels and all crossmatches were converted to negative; post-transplant, 33% of patients had antibody-mediated rejection episodes, but there was no strong correlation between DSA levels and rejections



Adrenomed AG, of Hennigsdorf, Germany


Adrenomedullin ligand inhibitor

Septic shock

Enrollment completed in AdrenOSS-2 trial, which randomized 300 participants with early septic shock and elevated blood levels of adrenomedullin; primary endpoints are safety and tolerability over 90-day period, with Sepsis Support Index as key secondary endpoint


Amplyx Pharmaceuticals Inc., of San Diego

Fosmanogepix (APX-001)

Inhibits highly conserved fungal enzyme Gwt1


2 independent review committee completed safety and efficacy assessment of data from 50% of planned population, determining Fosmanogepix showed high level of treatment success at end of treatment from first 10 patients; recommended study continue according to protocol, for total enrollment of 20 patients


Aridis Pharmaceuticals Inc., of San Jose, Calif.


Fully human IgG1 monoclonal antibody

Pseudomonas aeruginosa-related ventilator-associated pneumonia

Study missed primary endpoint of superiority in clinical cure rates on day 21 compared to placebo and showed statistically significant imbalance in all-cause mortality and in serious adverse event rates that favored placebo, prompting company to halt development


Micurx Pharmaceuticals Inc., of Foster City, Calif.

Contezolid acefosamil


Acute bacterial skin and skin structure infection

MRX4-201 trial vs. linezolid met all primary and secondary endpoints, with potentially improved hematologic safety profile; 77.9% in contezolid acefosamil group vs. 78.5% in linezolid group in intent-to-treat (ITT) population had favorable responses at early assessment visit (48-72 hours) after start; outcomes were similar in ITT population at post-therapy evaluation (7-14 days after end of therapy), at 76.3% in contezolid acefosamil group and 73.8% in linezolid group; proportion of subjects with neutrophil and platelet values below lower limit of normal or substantially abnormal were lower in contezolid acefosamil group vs. linezolid group


Pfizer Inc., of New York


20-valent pneumococcal conjugate vaccine

Prevention of invasive disease and otitis media caused by Streptococcus pneumoniae serotypes

Initial 3 doses provided preliminary evidence that the vaccine candidate in healthy infants has an overall safety profile that is similar to Prevnar 13; candidate induced immune responses for all 20 serotypes in infants


Takeda Pharmaceutical Co. Ltd., of Osaka, Japan

TAK-620 (maribavir)

Serine threonine protein kinase UL97 inhibitor

Cytomegalo-virus infection

The New England Journal of Medicine published results from 12-week study in individuals with CMV infection following hematopoietic cell transplant or solid organ transplant; 62% treated with any dose of maribavir showed effect within 3 weeks of treatment vs. 56% with valganciclovir; at 6 weeks, response rates were 79% and 67%, respectively


Valneva SE, of Saint-Herblain, France


Outer surface protein A modulator

Lyme disease

Completed patient recruitment



Cellular Biomedicine Group Inc., of New York and Shanghai


Allogenic human adipose-derived mesenchymal progenitor cell therapy

Knee osteoarthritis

First phase II study plans to recruit 108 patients with Kellgren and Lawrence grade II-III KOA at 6 hospitals in China


Immunic Inc., of San Diego


Blocks the enzyme dihydroorotate dehydrogenase on activated T and B cells 

Ulcerative colitis

Unblinded and independent data review committee concluded the lowest dose (10 mg) was found not to be likely ineffective and the highest dose (45 mg) was not intolerable; company intends to continue the trial with all 3 dosing arms.


Viela Bio Inc., of Gaithersburg, Md.


CD19 binder

Neuromyelitis optica spectrum disorder

Demonstrated significant reduction in risk of NMOSD attack and reduced disability scores as measured by expanded disability status scale, hospitalizations and new central nervous system magnetic resonance imaging lesions; results published in The Lancet



Acceleron Pharma Inc., of Cambridge, Mass.


Targets TGF-beta protein superfamily

Facioscapulo-humeral muscular dystrophy

Treatment did not achieve functional secondary endpoints; though drug demonstrated statistically significant increase in mean total muscle volume, the primary endpoint, the increase failed to translate to statistically significant improvements in functional tests; Acceleron will not conduct further clinical trials of ACE-083 in FSHD


Antisense Therapeutics Ltd., of Melbourne, Australia


Inhibitor of CD49d expression

Duchenne muscular dystrophy

Preliminary data from first 6 patients treated for 24 weeks showed mean change from baseline in pinch and grip were 0.1 and 0.3, respectively


Neurana Pharmaceuticals Inc., of San Diego


Blocks voltage-gated sodium and calcium channels

Acute muscle spasms of the back

In the 415-patient Star study, the 600-mg dose improved subject-rated pain due to acute back spasm using a Numerical Rating Scale (p=0.004); plans to start a phase III study in 2020



Acadia Pharmaceuticals Inc., of San Diego


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

Parkinson's disease patients with depressive symptoms

In the open-label, 47-patient study, 17-item Hamilton Depression Rating Scale (HAMD-17) total score improved from baseline to week 8 (p<0.0001); improvement seen as early as week 2 (p<0.0001); 60% of patients had an improvement of at least 50% on the HAMD-17 total score at week 8; 44.4% of patients reached remission (HAMD-17 ≤7); improvements were also seen in the Clinical Global Impression-Severity scale (baseline to week 8, p<0.0001) and the SCOPA-Global Sleep Quality scale (baseline to week 8, p<0.0001)


Acadia Pharmaceuticals Inc., of San Diego


5-HT 2a receptor inverse agonist

Major depressive disorder

The Journal of Clinical Psychiatry published results from 10-week, Clarity study (n=207) in which pimavanserin met primary endpoint by reducing 17-item Hamilton Depression Rating Scale (HAMD-17) score vs. placebo (p=0.039); adding drug to first-line SSRI or SNRI therapy also reduced HAMD-17 scores vs. placebo (p=0.0003), and pimavanserin showed statistically significant reductions vs. placebo in key secondary endpoint of Sheehan Disability Scale score (p=0.004)


Alzprotect SAS, of Lille, France


Modulates the degradation of both APP and tau protein fragments

Progressive supranuclear palsy

Launched a 36-patient phase IIa study to test the tolerability and pharmacokinetics of 2 doses compared to placebo for 3 months, followed by a 3-month weaning observation period; 20-plus biomarkers will also be evaluated; data expected to be published in 2021


Anavex Life Sciences Corp., of New York

Anavex2-73 (blarcamesine)

Activates sigma-1 receptor protein

Rett syndrome

Preliminary data from Anavex2-73-RS-001 study on first 6-patient cohort receiving low dose of about 5 mg daily oral liquid dose for 7 weeks showed significant improvement vs. baseline on both efficacy endpoints, the Rett Syndrome Behavior Questionnaire (p=0.027) and Clinical Global Impression-Improvement (p=0.003) scores; independent data safety monitoring board recommended study continue without modifications


Arena Pharmaceuticals Inc., of San Diego


Cannabinoid CB2 receptor agonist


Patient-reported outcomes across all domains evaluated improved at 8 weeks compared to baseline in trial evaluating treatment of visceral pain associated with gastrointestinal disorders


Axon Neuroscience SE, of London


Vaccine to generate antibodies against pathological tau

Alzheimer's disease

Neurofilament light chain increased 12.6% from baseline over 2 years in patients treated with AADvac1 vs. 27.7% for patients on placebo (p=0.0039); 3 cerebrospinal fluid Alzheimer's-specific biomarkers, including 2 variants of pathological tau (phospho-Tau181 and phospho-Tau217), were reduced in patients treated with AADvac1; positive signals for cognitive endpoints were observed among younger patients in the study


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

Spinraza (nusinersen)

Antisense oligonucleotide targeting survival motor neuron

Spinal muscular atrophy

Starting the 126-patient phase II/III Devote study comparing 2 loading doses of 50 mg 15 days apart followed by a maintenance dose of 28 mg every 4 months to the current regimen of 4 loading doses with 12-mg maintenance doses every 4 months; trial will also be used to determine how to safely and efficiently transition patients from the currently approved regimen to the higher dose


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

Vazegepant (BHV-3500)

Calcitonin gene-related peptide receptor antagonist

Acute migraine

Completed enrollment in the study measuring pain freedom and freedom from the most bothersome migraine-associated symptom at 2 hours post-dose; study is testing 3 doses of the intranasal drug compared to placebo in more than 2,100 patients; top-line data expected in the fourth quarter of 2019


Cassava Sciences Inc., of Austin, Texas


Filamin A modulator

Alzheimer's disease

Top-line data from open-label phase IIa study in 13 patients showed treatment for 28 days reduced biomarkers of AD pathology, neuroinflammation and neurodegeneration, achieving 100% responder rate; total tau decreased 20% (p<.001); phosphorylated tau decreased 34% (p<.0001); neurofilament light chain, marker for neurodegeneration, decreased 22% (p<.0001); neurogranin, marker for cognitive decline, decreased 32% (p<.0001); neuroinflammatory marker YKL-40, indicator of microglial activation, decreased 9% (p<.0001); IL-6 decreased 14% (p<.0001); IL-1-beta decreased 11% (p<.0001); tumor necrosis factor alpha decreased 5% (p=.001)


Cassava Sciences Inc., of Austin, Texas


Targets scaffolding protein filamin A

Alzheimer's disease

Started a phase IIb study and dosed first 2 patients; trial will evaluate safety, tolerability and drug effects on validated biomarkers; primary endpoint is improvement in biomarkers of Alzheimer's from baseline to day 28


Cerevel Therapeutics LLC, of Boston


Selective partial agonist of the dopamine D1 and D5 receptors

Early stage Parkinson's disease

In 57 patients, mean change from baseline at week 15 in the MDS-UPDRS Part III score was -9 for tavapadon and -4.3 for placebo (p=0.0407); 50% of patients treated with tavapadon reported being "much improved" or "very much improved" on the PGI-C scale, compared with 25% in the placebo group; daytime sleepiness on the Epworth Sleepiness Scale at weeks 9 and 15 were similar for tavapadon and placebo


Cortexyme Inc., of South San Francisco


Virulence factor inhibitor targeting gingipains from P. gingivalis

Alzheimer's disease

Started patient screening in Europe for ongoing phase II/III Gain study in subjects with mild to moderate disease; top-line results expected in the fourth quarter of 2021


India Globalization Capital Inc., of Bethesda, Md.


Liquid formulation of tetrahydrocannabinol and an amyloid-beta 40 protein production inhibitor

Alzheimer's disease

Institutional review board in Puerto Rico approved its protocol for a double-blind, placebo-controlled, efficacy, safety and tolerability study; company will next apply for IND


Marinus Pharmaceuticals Inc., of Radnor, Pa.


GABA A receptor agonist

Refractory status epilepticus

Top-line data showed study evaluating intravenous (I.V.) ganaxolone met primary endpoint, with no participants (n=17) progressing to I.V. anesthetics within 24 hours of treatment initiation; median time to status cessation was 5 minutes (n = 15 evaluable); positive trends in health outcomes such as hospital length of stay seen at target dose of 713 mg/day


Mitsubishi Tanabe Pharma Corp., of Jersey City, N.J.


Continuous delivery of carbidopa/levodopa via subcutaneous infusion

Parkinson's disease

Secondary, patient-reported outcomes showed those in the continuous 24-hour treatment group reported statistically significant change in United Parkinson Disease Rating Scale ADL score vs. baseline (p=0.02); quality of life also improved in that group (p=0.02) and patients reported improvement in 6 of 8 Parkinson's disease Questionnaire quality of life domains


Neuralstem Inc., Germantown, Md.


Human neural stem cells

Chronic ischemic stroke

Enrollment completed with 22 participants randomized to treatment or sham surgery, who will be evaluated for efficacy measures for 12 months following surgery


Neurotrope Inc., of New York


Protein kinase C agonist

Moderate to severe Alzheimer's disease

Did not achieve statistical significance on the primary endpoint, which was change from baseline to week 13 in the Severe Impairment Battery total score


Orchard Therapeutics Ltd., of Boston


Gene therapy expressing arylsulfatase-A

Metachromatic leukodystrophy

Comparison of 16 late infantile patients to an untreated age-matched natural history cohort showed an improvement in gross motor function measurement of 65.6 percentage points (p<0.001) and 71.5 percentage points (p<0.001) at 2 and 3 years of follow-up, respectively; in 13 early juvenile patients, improvement was 42 percentage points (p=0.036) and 56.7 percentage points (p=0.001) at 2 and 3 years of follow-up, respectively; cognitive performance scores were maintained within normal range for most treated patients while untreated natural history cohort showed severe cognitive impairment


Oryzon Genomics SA, of Madrid, Spain


Inhibits LSD1 and MAOB

Autism spectrum disorder

Drug improved Clinical Global Impression of Severity and Improvement scales (p=0.0005 and p=0.0019, respectively), Neuropsychiatric Inventory total score (p=0.0019) and NPI 4-item Agitation/Aggression subscale (p=0.0098)


Ovid Therapeutics Inc., of New York


Inhibits cholesterol 24-hydroxylase

Rare developmental and epileptic encephalopathies

In the Endymion extension study, 6 patients had an 84% reduction in seizure frequency from baseline for weeks 25-36; 4 patients had a 90% reduction in seizure frequency from baseline for weeks 37-48


Sage Therapeutics Inc., of Cambridge, Mass.


Next-generation positive allosteric modulator

Major depressive disorder

Results from pivotal trial, published in The New England Journal of Medicine, showed clinically meaningful improvement in symptoms across multiple measures and time points; at day 15, 64% of patients achieved remission, defined as a score of 7 or less on the Hamilton Rating Scale for Depression (HAM-D), compared with 26% in the placebo group; at day 15, the HAM-D response rates were 78.6% vs. 40.5% in SAGE-217 and placebo groups, respectively


Tetra Therapeutics Inc., of Grand Rapids, Mich.


PDE4D inhibitor

Early Alzheimer's disease

Surpassed 50% enrollment in Picasso AD trial


Zynerba Pharmaceuticals Inc., of Devon, Pa.

Zygel (ZYN-002)

Transdermal cannabidiol

Developmental and epileptic encephalopathy

Of the 33 patients with focal impaired-awareness seizures, median seizure frequency was reduced by 44% at month 2 and by 51% at month 6; in 11 patients with Lennox-Gastaut and Dravet syndromes, median seizure frequency was reduced by 6% at month 2 and by 51% at month 6



Apellis Pharmaceuticals Inc., of Crestwood, Ky.

APL-2 (pegcetacoplan)

Inhibits C3 and C3b of the complement cascade

Geographic atrophy secondary to age-related macular degeneration

Data from Filly study published in Ophthalmology showed the treatment produced a 29% reduction in the growth of GA lesion area from baseline to month 12 in the monthly treatment group (p=0.008) and a 20% reduction in the every other month (EOM) treatment group (p=0.067) compared to the pooled sham group; post-hoc analysis found reductions of 45% (p=0.0004) and 33% (p=0.009) for APL-2 monthly and EOM, respectively, compared to sham, over the last 6 months of treatment


Graybug Vision Inc., of Redwood City, Calif.


Microparticle depot formulation of tyrosine kinase inhibitor sunitinib malate

Macular edema secondary to diabetic macular edema or retinal vein occlusion

Started phase IIa 20-patient study testing 2 doses of the drug; primary endpoint is safety; best corrected visual acuity, contrast sensitivity testing and time to rescue treatment will be measured as secondary endpoints


Nicox SA, of Sophia Antipolis, France


Ophthalmic suspension of fluticasone propionate nanocrystals


Completed enrollment in trial testing drug in acute exacerbations of blepharitis; top-line data expected in the fourth quarter of 2019


Ocuphire Pharma Inc., of Farmington Hills, Mich.


Ophthalmic formulation of alpha-1/2 inhibitor phentolamine mesylate

Open-angle glaucoma or ocular hypertension

Enrolled 39 glaucoma patients in Orion-1 study to evaluate efficacy in lowering intraocular pressure and safety


Opthea Ltd., of Melbourne, Australia


Soluble form of VEGF3

Wet age-related macular degeneration

New data from 366-patient phase IIb study in combination with Lucentis (ranibizumab, Roche Holding AG) vs. Lucentis alone showed in patients who received 2-mg OPT-302 combination therapy had mean wet AMD total lesion area at week 24 a decrease from baseline by 4.33 mm2, compared to 3.11 mm2 in control group, a relative benefit of 39% (p=0.0137); mean choroidal neovascularization area was also reduced from baseline to week 24 in combination group by 4.97 mm2, compared to 3.59 mm2 in control group, a relative benefit of 38% (p=0.0052)



Rhythm Pharmaceuticals Inc., of Boston


MC4R agonist


4 new indications added to the basket: SRC1 deficiency obesity; SH2B1 deficiency obesity; MC4R deficiency obesity; and Smith-Magenis syndrome


Saniona AB, of Copenhagen


Combination of tesofensine and metoprolol

Prader-Willi syndrome

3 adolescent patients in phase IIa trial lost an average of 2.6% over the 3-month period on 0.25-mg/day Tesomet compared to a weight gain of 2.3% during the 3-month period on 0.125-mg/day Tesomet and a weight gain of 2.2% in the 3-month period when 2 received placebo and 1 patient received 0.125-mg/day Tesomet; average hyperphagia score was reduced by 69% from baseline at the end of the period on 0.25 mg/day and 33% at the end of the period on 0.125-mg/day Tesomet



Fibrogen Inc., of San Francisco


Connective tissue growth factor ligand inhibitor

Idiopathic pulmonary fibrosis

Phase II Praise study published in The Lancet Respiratory Medicine showed mean change from baseline to week 48 in percentage of predicted FVC was -2.9% in pamrevlumab group vs. -7.2% in placebo group (between-group difference 4.3%; p=0.033)


Pulmatrix Inc., of Lexington, Mass.


Dry powder formulation of antifungal itraconazole

Allergic bronchopulmonary aspergillosis

Dosed first of about 64 patients in placebo-controlled study testing 3 dose levels of the drug for 28 days; primary endpoint is safety and tolerability; pharmacokinetics in plasma and sputum, effect on biomarkers of inflammation, pulmonary function, asthma symptoms and aspergillus burden in sputum will be measured as secondary endpoints



Achieve Life Sciences Inc., of Seattle


Plant-based alkaloid that targets nicotinic acetylcholine receptor

Nicotine dependence

The 3-mg 3-times-daily dose, which will be used in phase III development, produced a 54% abstinence rate at week 4 compared to 16% for placebo (p<0.0001); 4-week continuous abstinence rate for weeks 5 through 8 was 30% for cytisinicline compared to 8% for placebo (p= 0.005)


Achieve Life Sciences Inc., of Seattle


Nicotinic acetylcholine receptor modulator

Nicotine dependence

Completed study finding lack of dose-limiting toxicity even at the highest 30-mg single, oral dose



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


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