Phase I

Akcea Therapeutics Inc., of Boston

ION-682884 (AKCEA-TTR-LRx)

Antisense drug that inhibits transthyretin

Familial amyloid neuropathy

Healthy volunteers who received injections saw significant reductions in TTR levels following treatment combined with a positive safety and tolerability profile

Arrowhead Pharmaceuticals Inc., of Pasadena, Calif.


APOC3 gene inhibitor


Single 100-mg dose in healthy volunteers reduced plasma triglycerides by 63% and APOC3 protein by 94% without serious or severe adverse events

Arrowhead Pharmaceuticals Inc., of Pasadena, Calif.


ANGPTL3 gene inhibitor


Single 200-mg dose in healthy volunteers reduced plasma triglycerides by 66% and ANGPTL3 protein by 79% without drug-related serious or severe adverse events

Cytokinetics Inc., of San Francisco


Myosin inhibitor

Hypertrophic cardiomyopathy

Study met primary and secondary objectives to assess safety and tolerability of single and multiple oral doses; will advance to phase II trial in patients with obstructive hypertrophic cardiomyopathy expected to begin in Q4 2019

Discgenics Inc., of Salt Lake City


Cell therapy based on allogeneic disc tissue-derived progenitor cells

Degenerative disc disease

Second planned safety review supported trial continuation in first 30-person cohort with no changes to protocol

Kodiak Sciences Inc., of Palo Alto, Calif.


VEGF receptor antagonist

Wet age-related macular degeneration

Early durability, efficacy and safety data support pivotal Dazzle study design evaluating every 3-, 4- and 5-month dosing

Kura Oncology Inc., of San Diego


Inhibitor of menin-mixed lineage leukemia interaction

Relapsed or refractory acute myeloid leukemia

Dosed first patient in open-label, dose-escalation trial to determine maximum tolerated dose

Omeros Corp., of Seattle


PDE7 inhibitor

Addictions and compulsive disorders

Healthy volunteer study met primary endpoints of safety and tolerability, with favorable and dose-proportional pharmacokinetic profile supporting once-daily dosing; no apparent food effect on plasma exposure

Phase II

Amplyx Pharmaceuticals Inc., of San Diego

Fosmanogepix (APX-001)

Inhibits highly conserved fungal enzyme Gwt1


2 independent review committees 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

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

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

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



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



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

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

Phase III

Allena Pharmaceuticals Inc., of Newton, Mass.


Oral, recombinant oxalate-degrading enzyme

Enteric hyperoxaluria

Completed enrollment in pivotal Urirox-1 trial; top-line data expected in fourth quarter of 2019

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.


RNAi therapeutic

Transthyretin amyloidosis with cardiomyopathy

Started Apollo-B trial; primary endpoint is change from baseline in 6-minute walk test at 12 months; secondary endpoints will test efficacy on quality of life using Kansas City Cardiomyopathy Questionnaire Overall Summary and composite endpoints of mortality and hospitalization

Biocardia Inc., of San Carlos, Calif.

Cardiamp cell therapy

Autologous bone marrow cells delivered via catheter-based procedure

Heart failure

Independent data safety monitoring board (DSMB) completed prespecified data review for pivotal trial and recommended study continue as planned; primary endpoint is 6-minute walk distance 12 months post-treatment; next DSMB review is anticipated in first quarter of 2020

Eyegate Pharmaceuticals Inc., of Waltham, Mass.

Ocular bandage gel eye drop

Modified form of natural polymer hyaluronic acid

Corneal wound repair following photorefractive keratectomy surgery

Completed randomization of more than 75% of patients in pivotal study

Resverlogix Corp., of Calgary, Alberta

Apabetalone (RVX-208)

Small-molecule selective BET inhibitor

Cardiovascular disease

Following database lock, Betonmace top-line data are expected on or about Sept. 30, 2019


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


No Comments