Company |
Product |
Description |
Indication |
Status |
Phase I |
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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 |
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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 |
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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 For more information about individual companies and/or products, see Cortellis. |