Company

Product

Description

Indication

Status

Phase I

Asana Biosciences LLC, of Lawrenceville, N.J.

ASN-008

Sodium channel blocker

Pruritus associated with atopic dermatitis

Granted permission by the FDA to start clinical trials

Lexicon Pharmaceuticals Inc., of The Woodlands, Texas

LX-2761

SGLT1 inhibitor

Healthy volunteers and type 2 diabetes

In a single ascending-dose study, LX-2761 had minimal absorption, no systemic effect and didn't increase urine glucose excretion from baseline; in diabetic patients, drug reduced postprandial glucose and increased plasma levels of GLP-1

Lexicon Pharmaceuticals Inc., of The Woodlands, Texas

LX-2761

SGLT1 inibitor

Type 2 diabetes

In a multiple ascending-dose, placebo-controlled study, treatment with drug reduced postprandial glucose and increased plasma levels of GLP-1 with minimal effect on urinary glucose excretion

Medigene AG, of Martinsried, Germany

Dendritic cell vaccine

Patient-derived cells

Acute myeloid leukemia

Interim data from 20 patients showed 12-month overall survival of 80%; 12-month progression-free survival was 60%; 5 of 8 relapses occurred within first 80 days after start of vaccination

OSE Immunothera-peutics SA, of Nantes, France

OSE-127

MAb targeting the interleukin-7 receptor

Autoimmune diseases and chronic inflammation

63 healthy volunteers were dosed; trial aims to evaluate the safety and tolerability of single- and multiple-ascending intravenous and subcutaneous doses

PCI Biotech Holding ASA, of Oslo, Norway

Fimachem

Chemotherapy with photochemical internalization

Inoperable extrahepatic bile duct cancer

In an extension study, a second treatment administered 3 to 4 months after the initial treatment was deemed safe by the cohort review committee; pivotal study scheduled to start in the first half of 2019

Transgene SA , of Strasbourg, France

TG-4001 + avelumab

Human anti-programmed death ligand (PD-L1) antibody

HPV-16+[1] recurrent or metastatic malignancies

Primary endpoint (safety and tolerability) was met; 9 patients received escalating doses of TG-4001 combined with a fixed dose of avelumab; no dose-limiting toxicity was observed, confirming a satisfactory tolerability profile for the combined regimen, allowing the trial to progress to the phase II

Viriom Inc., of San Diego

VM-1500A-LAI

Non-nucleoside reverse transcriptase inhibitor

HIV-uninfected volunteers

Started study testing the safety, tolerability and pharmacokinetics of VM-1500A-LAI in single and multiple ascending doses

Phase II

Apexigen Inc., of San Carlos, Calif.

APX-005M

MAb targeting CD40

Advanced sarcomas

In a collaboration with Columbia University Irving Medical Center, company plans to test drug plus doxorubicin and olaratumab; primary endpoint is objective response rate; progression-free survival will be measured as a secondary endpoint

Da Volterra SA, of Paris

DAV-132

Captures residual antibiotics in the colon

High-risk of Clostridium difficile infection

Enrolled first of 260 patients in the Shield study in patients receiving oral or intravenous fluoroquinolone antibiotics (moxifloxacin, levofloxacin, ciprofloxacin); primary endpoint is safety, but reduction of infection and antibiotic-associated diarrhea will also be measured in patients receiving antibiotics plus DAV-132 compared to antibiotics alone; reduction of fecal antibiotics concentration, maintenance of bacterial diversity and reduction of the emergence of antibacterial resistance will also be measured

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

Trilaciclib

CDK4/6 inhibitor 

2nd-/3rd-line small-cell lung cancer

In the 91-patient study, 40.6% of patients treated with trilaciclib plus topotecan had grade 4 neutropenia, compared to 75.9% of patients treated with topotecan alone (adjusted 1-sided p=0.0160); duration of grade 4 neutropenia in cycle 1 was shorter: 2 days for trilaciclib vs. 8 days for placebo (adjusted 1-sided p < 0.0001); treatment with trilaciclib resulted in a 45.0% reduction in number of G-CSF administrations per cycle and a 58.7% reduction in the number of red blood cell transfusions (on/after week 5) per week compared to placebo

Hutchison China Meditech Ltd., of London, and Astrazeneca plc, of Cambridge, U.K.

Savolitinib

c-Met inhibitor

MET+ EGFR mutated non-small-cell lung cancer who have progressed after Tagrisso (osimertinib)

Started 170-patient Savannah study testing savolitinib plus Tagrisso; data expected in 2021

Kalytera Therapeutics Inc., of San Rafael, Calif.

CBD

Cannabidiol

Prevention of acute graft vs. host disease

Interim data from 12 patients showed no patients developed grade 3 or 4 graft vs. host disease (GVHD); there was 1 patients with grade 2 acute GVHD; plans to test 2 additional higher doses

Sirnaomics Inc., of Gaithersburg, Md.

STP-705

In situ squamous cell carcinoma nonmelanoma skin cancer

siRNA to inhibit expression of TGF-beta1 and COX-2

FDA agreed to proposed trial design; study scheduled to begin in first half of 2019

VBI Vaccines Inc., of Cambridge, Mass.

VBI-1501

Cytomegalovirus vaccine

Cytomegalovirus prophylaxis

Following discussions with Health Canada, company plans to run a 110-patient trial comparing safety and immunogenicity of 3 doses of the vaccine to placebo; toxicology studies need to be completed before study can begin around the end of 2019

Phase III

Acasti Pharma Inc., of Laval, Quebec

Capre (omega-3 phospholipid)

Lipid derived from krill oil

Severe hypertriglyceridemia

Completed enrollment of over 1,450 patients in the Trilogy studies; top-line data expected before the end of 2019

Aldeyra Therapeutics Inc., of Lexington, Mass.

Reproxalap

Aldehyde trap

Allergic conjunctivitis

Completed enrollment of over 300 patients in the Alleviate study comparing 2 doses of the drug to placebo; primary outcome is patient-reported ocular itching; data expected in early 2019

Amicus Therapeutics Inc., of Cranbury, N.J.

AT-GAA

GAA protein with optimized carbohydrate structures plus a pharmacological chaperone

Pompe disease

First patient dosed in the Propel study comparing drug to alglucosidase alfa; primary endpoint is change in six-minute walk distance from baseline to week 52; respiratory function and additional measures of muscle function and muscle strength will also be assessed

Apellis Pharmaceuticals Inc., of Crestwood, Ky.

APL-2

Cyclic peptide inhibitor of C3 and C3b

Geographic atrophy

Following the manufacture of a new lot of drug, company plans to restart enrollment in the Derby and Oaks studies in the second quarter of 2019 and complete enrollment in the first quarter of 2020

Aradigm Corp., of Hayward, Calif.

Apulmiq (formerly Linhaliq)

Liposomal formulation of ciprofloxacin

Non-cystic fibrosis bronchiectasis with Pseudomonas aeruginosa chronic lung infection

Third party evaluation of the Orbit-3 and Orbit-4 studies confirm previously reported conclusions; company plans to meet with FDA

Astellas Pharma Inc., of Tokyo, and New York-based Pfizer Inc.

Xtandi (enzalutamide)

Androgen receptor inhibitor

Metastatic hormone-sensitive prostate cancer

In the 1,150-patient Arches study, drug plus androgen deprivation therapy (ADT) improved radiographic progression-free survival compared to ADT alone; data to be presented at a future medical congress

Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J.

Lynparza (olaparib)

PARP inhibitor

Relapsed BRCA-mutated ovarian cancer after 2 or more lines of treatment

In the 266-patient Solo-3 study, Lynparza improved objective response rate and progression-free survival compared to physician's choice single-agent chemotherapy (paclitaxel, topotecan, pegylated liposomal doxorubicin or gemcitabine); data to be presented at a forthcoming medical meeting

Astrazeneca plc, of Cambridge, U.K., and Fibrogen Inc., of San Francisco

Roxadustat

Hypoxia-inducible factor prolyl hydroxylase inhibitor

Anemia in chronic kidney disease

In the 2,781-patient Olympus study, drug produced a statistically-significant improvement in the mean change from baseline in hemoglobin levels averaged over weeks 28 to 52 compared to placebo; in the 2,133-patient Rockies study, drug produced a statistically-significant improvement in the mean change from baseline in hemoglobin levels averaged over weeks 28 to 52 compared to epoetin alfa; data to be presented at a forthcoming medical meeting

Deciphera Pharmaceuticals Inc., of Waltham, Mass.

Ripretinib (DCC-2618)

KIT and PDGFR-alpha inhibitor

Second-line gastrointestinal stromal tumor

Started Intrigue study comparing drug to sunitinib; primary efficacy endpoint is median progression-free survival; objective response rate and overall survival will also be measured

Retrophin Inc., of San Diego

Fosmetpantotenate

Converted to phosphopantothenic acid

Pantothenate kinase-associated neurodegeneration

Completed enrollment of approximately 82 patients in the Fort study; primary endpoint is the change from baseline in the Pantothenate Kinase-Associated Neurodegeneration Activities of Daily Living scale through 24 weeks; top-line data expected in the third quarter of 2019

Supernus Pharmaceuticals Inc., of Rockville, Md.

SPN-812

Norepinephrine reuptake inhibitor

Attention deficit hyperactivity disorder

In the P302 study, at week 6, 200 mg and 400 mg produced a -16.0 point change (p=0.0232) and a -16.5 point change (p=0.0091) from baseline in the ADHD-RS-5 total score, respectively, vs. -11.4 points for placebo


Notes

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