Phase I

Acurx Pharmaceuticals LLC, of White Plains, N.Y.


DNA polymerase inhibitor

Clostridium difficile infection

Microbiome analysis demonstrated that ACX-362E has a significantly more favorable effect than oral vancomycin due to less disruption of the microbiota in the gastrointestinal tract

Aptevo Therapeutics Inc., of Seattle


Bispecific antibody

Autoimmune diseases

Development halted; not meeting targeted product profile

Arbutus Biopharma Corp., of Warminster, Pa.


Oral capsid inhibitor

Hepatitis B virus

Development discontinued after 2 healthy volunteers developed hepatitis

Fulcrum Therapeutics Inc., of Cambridge, Mass.


Selective p38α/β mitogen activated protein kinase inhibitor

Facioscapulohumeral dystrophy

Patients treated with losmapimod also achieved dose-dependent concentrations in skeletal muscle, with a muscle to plasma exposure ratio of about 1-to-1; the 15-mg dose taken orally twice daily demonstrated sustained drug concentrations

Hutchison China Meditech Ltd., of London 


Spleen tyrosine kinase inhibitor

Relapsed/refractory lymphoma

Trial begun

Mei Pharma Inc., of San Diego


Oral inhibitor of PI3K delta

Follicular lymphoma; chronic lymphocytic leukemia; small lymphocytic lymphoma

Overall response rates from an ongoing phase Ib study were 78% in relapsed or refractory (r/r) follicular lymphoma (FL) and 89% in r/r chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL); median duration of response not yet reached in patients with FL or CLL/SLL on the IS regimen; median follow-up for FL and CLL/SLL patients is 9.2 months (range 3.4-20.7 months) and 7.4 months (range 2.6-14.7 months), respectively

Sarepta Therapeutics Inc., of Cambridge, Mass.


Gene therapy

Limb-girdle muscular dystrophy type 2E

9-month functional results from 3 participants who received SRP-9003 at dose of 5x1013vg/kg showed improvements in functional outcomes at day 270

Viacyte Inc., of San Diego


Pancreatic precursor cells

Type 1 diabetes

Preliminary data from the PEC-Direct trial show implanted cells, when effectively engrafted, can produce circulating C-peptide, a biomarker for insulin

Phase II

Biophytis SA, of Paris


Small molecule


22nd site opened in the U.S. for the SARA-INT phase IIb study

Cytokinetics Inc., of South San Francisco


Skeletal muscle troponin activator

Amyotrophic lateral sclerosis

Post-hoc analyses of Fortitude-ALS showed that, in the combined middle and faster progressing tertiles, the decline in ALSFRS-R total score from baseline to week 12 was smaller in patients who received any dose of reldesemtiv vs. placebo (LS mean treatment difference 1.15, p = 0.011), while no significant difference was observed in slower progressing patients who received reldesemtiv vs. placebo

Phase III

Amylyx Pharmaceuticals Inc., of Cambridge, Mass.


Combination sodium phenylbutyrate and tauroursodeoxycholic acid

Amyotrophic lateral sclerosis

Last participant completed planned 24 weeks of treatment in Centaur study

Gilead Sciences Inc., of Foster City, Calif.

Descovy (emtricitabine 200-mg and tenofovir alafenamide 25-mg tablets)

Fixed-dose combination of HIV nucleoside analogue reverse transcriptase inhibitors

HIV pre-exposure prophylaxis

Findings from Discover trial showed significant improvements in key measures of bone and renal safety parameters in subset of people who switched from Truvada (emtricitabine 200 mg and tenofovir disoproxil fumarate 300 mg tablets) to Descovy for PrEP

Octapharma USA, of Hoboken, N.J.


Von Willebrand factor replacement therapy

Von Willebrand disease

Introduced study to determine efficacy in prophylactic treatment of previously treated patients with type 3, type 2 (except 2N) or severe type 1 VWD; also introduced study to test Wilate use in VWD for childbirth


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


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