Company (location)

Product

Description

Indication

Status

Date

Cancer

Arvinas Inc., of New Haven, Conn.

ARV-471

Estrogen receptor (ER)-targeting Protac protein degrader

Locally advanced or metastatic ER positive, HER2 negative breast cancer

Treated first patient in the study testing the safety, tolerability, and pharmacokinetics of ARV-471; pharmacodynamics and anti-tumor activity will be measured as secondary endpoints; preliminary data expected in 2020

8/27/19

Basilea Pharmaceutica Ltd., of Basel, Switzerland

Derazantinib

Pan-fibroblast growth factor receptor kinase inhibitor 

Advanced urothelial cancer

Study begun in combination with Tecentriq (atezolizumab, Roche Holding AG)

8/13/19

Basilea Pharmaceutica Ltd., of Basel, Switzerland

BAL-101553

Prodrug of an active moiety that binds to the colchicine site of tubulin

Progressive or recurrent glioblastoma or high-grade glioma

In the 28-patient study, dose levels up to and including 25 mg per day were well tolerated; drug had anti-tumor activity, including 1 patient with strong expression of end-binding protein 1 who had a long-lasting response and is still on treatment; 5 patients had stable disease

8/27/19

Bioinvent International AB, of Lund, Sweden

BI-1206

Immunoglobulin gamma Fc receptor IIB antagonist

Solid tumors

Plans to conduct dose-finding, consecutive-cohort, open-label phase l/lla trial in combination with anti-PD1 Keytruda (pembrolizumab, Merck & Co. Inc.), following FDA acceptance of IND application

8/21/19

Cellect Biotechnology Ltd., of Tel Aviv, Israel

Apograft

Stem cells

Blood cancer disorders undergoing allogenic HLA-matched hematopoietic stem cell transplant

After reviewing data from the 9th patient in the phase I/II study, the data and safety monitoring board recommended dose escalation; patient recruitment expected to be complete by the end of 2019 with data in the first half of 2020

8/12/19

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China

BLU-667

Targets oncogenic RET alterations

RET-altered non-small-cell lung cancer, medullary thyroid cancer and other advanced solid tumors

First patient enrolled

8/13/19

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China, and Blueprint Medicines Corp., of Cambridge, Mass.

Avapritinib

Inhibitor of KIT and PDGFRA

Unresectable or metastatic gastrointestinal stromal tumors

Treated first patient in a registrational bridging study in China that includes a dose-escalation phase and a dose-expansion phase

8/26/19

Deciphera Pharmaceuticals Inc., of Waltham, Mass.

Ripretinib

Broad-spectrum KIT and PDGFRα inhibitor

Second-line through fourth-line-plus gastrointestinal stromal tumors 

Updated data from 178 GIST patients receiving ripretinib at doses of >100 mg daily; median progression-free survival with ripretinib was 6.3 months vs. 1 month for placebo

8/13/19

Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J.

Fulvestrant

Estrogen receptor antagonist

Healthy volunteers (eventually estrogen receptor-positive breast cancer)

Plans to start a pilot study shortly to test the pharmacokinetics and safety of the new formulation, followed by a pivotal study in patients that could be completed within approximately 12 months of starting enrollment of the study

8/5/19

Eureka Therapeutics Inc., of Emeryville, Calif.

ET-140202

T-cell therapy targeting alpha fetoprotein peptide/HLA-A2 complex

Advanced hepatocellular carcinoma

Started study to test the drug's safety and find the recommended phase II dose; overall response rate, progression-free survival and overall survival will be measured as secondary endpoints

8/5/19

Gritstone Oncology Inc., of Emeryville, Calif.

SLATE

Tumor-specific neoantigens delivered with an adenoviral vector and a self-amplifying RNA vector

Advanced solid tumors

Dosed first patient in study testing SLATE in combination with Bristol-Myers Squibb Inc.'s Opdivo (nivolumab) and Yervoy (ipilimumab); preliminary data expected at year-end

8/14/19

Hemispherx Biopharma Inc., of Ocala, Fla.

Ampligen

Acts as TLR3 agonist

Recurrent ovarian cancer

Final report supports safety in combination with Intron A and celecoxib; produced positive survival data with regard to stage IV patients

8/8/19

I-Mab Biopharma Co. Ltd., of Shanghai

TJ-004309

CD73 antibody

Advanced solid tumors

Partner Tracon Pharmaceuticals Inc., of San Diego, dosed first patient

8/1/19

Inmune Bio Inc., of La Jolla, Calif.

INB-03

Dominant-negative TNF inhibitor

Advanced solid tumors

Data from 6 patients enrolled in the first 2 cohorts showed drug was well-tolerated; the target INB-03 trough level was reached in all 3 of the patients in the 1-mg/kg group; IL-6 decreased by more than 50% in half of the patients

8/5/19

Jazz Pharmaceuticals plc, of Dublin

JZP-458

Recombinant crisantaspase

Healthy volunteers (eventually acute lymphoblastic leukemia or lymphoblastic lymphoma)

Study met its efficacy and safety objectives; company plans to start a phase II/III study in patients who are hypersensitive to E. coli-derived asparaginase products

8/6/19

Mersana Therapeutics Inc., of Cambridge, Mass.

XMT-1536

Antibody-drug conjugate targeting NaPi2b

Platinum-resistant ovarian cancer and non-small-cell lung cancer adenocarcinoma

Treated first patient in the expansion portion of the study; maximum tolerated dose hasn't been reached, so dose-escalation portion of the study will continue in parallel

8/20/19

Moleculin Biotech Inc., of Houston

WP-1066

Inhibits p-STAT3 and other oncogenic transcription factors and stimulates an immune response

Pediatric brain tumors

Emory University Clinical Trial Review Committee approved the start of the investigator-initiated study

8/20/19

Moleculin Biotech Inc., of Houston

WP-1220

P-STAT3 inhibitor

Cutaneous T-cell lymphoma

Enrollment completed

8/13/19

Nkmax America Inc., of Irvine, Calif.

SNK

Autologous natural killer cells

Cancer refractory to conventional therapy

Treated first of 9 patients in the study testing 3 dosing levels; primary endpoints are safety, and objective response will be measured as a secondary endpoint

8/13/19

Oncternal Therapeutics Inc., of San Diego

Cirmtuzumab

ROR1-targeted monoclonal antibody

Chronic lymphocytic leukemia

Opened phase II portion of phase I/II study testing drug in combination with ibrutinib following favorable outcomes in part 1 dose-finding and part 2 dose-confirming cohorts, including observed interim objective response rate of 100% for first 9 patients with evaluable data receiving recommended dosing regimen and completing 12 weeks of combined therapy

8/6/19

Pharmamar SA, of Madrid

Lurbinectedin

Inhibits oncogenic transcription

Advanced solid tumors

The 26-patient Japanese study determined that 3.2 mg/m2 is the recommended dose, which is the same as in Western patients

8/27/19

Precigen Inc., of Germantown, Md., a wholly owned subsidiary of Intrexon Corp.

PRGN-3005

CAR T containing a chimeric antigen receptor, membrane-bound interleukin‐15 and a kill switch

Advanced, recurrent platinum-resistant ovarian, fallopian tube or primary peritoneal cancer

Enrolled first of 41 patients to determine the maximum tolerated dose; antitumor activity will also be assessed

8/5/19

Redx Pharma plc, of Cheshire, U.K.

RXC-004

Porcupine inhibitor

Advanced malignancies

First patient treated; enrollment has begun for second cohort; data expected in 2020

8/19/19

Soricimed Biopharma Inc., of Toronto

SOR-C13

Selective inhibitor of TRPV6

Late-stage pancreatic cancer

Investigator-initiated phase Ib trial launched to refine dosing and further explore safety and efficacy

8/6/19

Vaccinex Inc., of Rochester, N.Y.

Pepinemab

Anti-CD100 humanized IgG4 monoclonal antibody

Non-small-cell lung cancer

Classical-Lung phase Ib/II trial evaluating pepinemab plus Bavencio (avelumab) achieved full enrollment

8/13/19

Ziopharm Oncology Inc., of Boston

Ad-RTS-hIL-12 plus veledimex

Controlled IL-12

Recurrent glioma

Data published in Science Translational Medicine from 31 patients showed the 20-mg veledimex dose had superior drug compliance and showed 12.7 months median overall survival at mean follow-up of 13.1 months; concurrent corticosteroids negatively impacted survival and patients had further median OS of 17.8 months when cumulative amount of dexamethasone was limited to less than 20 mg during active veledimex dosing

8/14/19

Cardiovascular

Myokardia Inc., of South San Francisco

MYK-224

Myosin inhibitor

Hypertrophic cardiomyopathy

Initiated single and multiple ascending-dose study in healthy volunteers, enrolled into cohorts of 8 and randomized 3-to-1 to MYK-224 or placebo; in addition to safety, pharmacodynamic effects on cardiac function and dimensions will be assessed using echocardiography; top-line results expected in mid-2020

8/21/19

Ra Pharmaceuticals Inc., of Cambridge, Mass.

Undisclosed

Macrocyclic peptide

Cardiovascular disease

Collaborator Merck & Co. Inc., of Kenilworth, N.J., dosed first participant in trial evaluating orally available agent against noncomplement cardiovascular target

8/21/19

Dermatologic

Evelo Biosciences Inc., of Cambridge, Mass.

EDP-1815

Monoclonal antibody

Mild to moderate psoriasis

Phase Ib findings from initial 12-patient cohort showed statistically significant (p>0.05) reduction in mean lesion severity score (LSS) at 28 days of 2 points, compared to mean increase of 0.25 points in patients on placebo; LSS reductions with EDP-1815 ranged from 0% to 67%; analysis of basal epithelium mitotic count, a secondary endpoint, showed mean reduction of 2.25 cells/mm2 vs. no change in placebo patients receiving placebo

8/6/19

Evelo Biosciences Inc., of Cambridge, Mass.

EDP-1066

Monoclonal microbial candidate

Mild to moderate psoriasis

Phase Ib analysis of change over dosing period of blood immune cell cytokine production following stimulation by lipopolysaccharide, an exploratory endpoint, showed patients who received a 3.3g (5x) dose had reduction in cytokine production consistent with a pharmacodynamic effect; no reduction was observed in patients receiving a 660mg (1x) dose of EDP-1066 or placebo

8/6/19

Exopharm Ltd., of Melbourne, Australia

Plexaris

Exosomes from platelets

Wound healing

Started the Plexoval study testing safety, wound closure and scarring over 42 days after a single injection of Plexaris in up to 20 patients

8/26/19

Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda

KPL-716

Fully human monoclonal antibody that targets oncostatin M receptor beta

Prurigo nodularis; diseases characterized by chronic pruritus

Showed a rapid and sustained reduction in Worst-Itch Numeric Rating Scale in subjects with moderate to severe atopic dermatitis

8/13/19

Mallinckrodt plc, of Staines-upon-Thames

Stratagraft

Regenerative bilayer tissue

Burns

Phase Ib data published in Burns showed 27 of 29 patients had wound closure by 3 months post-treatment; wound closure wasn't statically different than autograft treatment (p=0.491); no significant difference in Patient and Observer Scar Assessment Scale scores between Stratagraft and autograft; Stratagraft was less painful than autograft

8/13/19

Nkmax America Inc., of Irvine, Calif.

SNK-01

Autologous NK cell adoptive immunotherapy

Psoriasis

Dosed first of 9 participants with mild to severe disease in dose-escalation study in Mexico evaluating safety and tolerability of intravenous administration once a week for 4 weeks at 3 dosing levels

8/20/19

Endocrine/Metabolic

Applied Therapeutics Inc., of New York

AT-007

Aldose reductase inhibitor

Galactosemia

Completed single ascending-dose healthy volunteer portion of phase I/II study, showing drug is well-tolerated, with no drug-related adverse events or dose-limiting toxicities reported; demonstrated linear pharmacokinetic profile, favorable exposure and half-life consistent with once-daily dosing

8/8/19

Imcyse SA, of Liège, Belgium

IMCY-0098

Insulin-based modified peptide that drives the generation of cytolytic CD4 T cells

Recent-onset type 1 diabetes

In the 41-patient study with 3 dose groups, there weren't statistically significant differences between the dose cohorts, but higher dose cohorts had trends towards better outcomes; cytolytic CD4 T-cells were detected and effector T cells were decreased

8/27/19

Synlogic Inc., of Cambridge, Mass.

SYNB-1020

Engineered probiotic bacteria

Hyperam-monemia

In the 23-patient phase Ib/IIa study, plasma and urinary nitrate increased in patients treated with SYNB-1020, but blood ammonia wasn't lower relative to placebo; other exploratory endpoints were also not improved relative to placebo

8/20/19

Gastrointestinal

89Bio Ltd., of San Francisco

BIO89-100

Glycopegylated analogue of fibroblast growth factor 21

Nonalcoholic steatohepatitis or nonalcoholic fatty liver disease with high risk of NASH

First patients screened in phase Ib/IIa trial; 83 patients to be randomized to weekly or every other week subcutaneous dosing with BIO89-100 or placebo for up to 12 weeks; changes from baseline liver fat will be assessed, as measured by Magnetic Resonance Imaging – Proton Density Fat Fraction, as well as changes from baseline in lipid parameters, transaminases and other pharmacodynamic parameters and biomarkers

8/8/19

Chemomab Ltd., of Tel Aviv, Israel

CM-101

CCR3 chemokine modulator and eotaxin 2 ligand inhibitor

Liver fibrotic diseases

Single administration study evaluating subcutaneous (SC) formulation in 40 healthy volunteers showed that similar dose levels of SC and intravenous formulations yielded comparable exposure, half-life, safety, tolerability and target engagement, with both formulations supporting dosing once every 3 weeks; company will explore multiple administrations of SC formulation in nonalcoholic steatohepatitis

8/21/19

Terns Pharmaceuticals Inc., of Foster City, Calif.

TERN-201

Semicarbazide-sensitive amine oxidase inhibitor

Nonalcoholic steatohepatitis

Interim data show TERN-201 has been generally safe and well-tolerated and results in sustained inhibition of plasma SSAO activity

8/13/19

Hematologic

Bayer AG, of Leverkusen, Germany

Jivi

Antihemophilic factor pegylated-aucl

Severe hemophilia A

In a head-to-head study, showed improved pharmacokinetic properties compared with Eloctate (antihemophilic factor [recombinant], Fc fusion protein, Sanofi SA)

8/13/19

Hutchison China Meditech Ltd., of London

HMPL-523

Spleen tyrosine kinase inhibitor

Immune thrombocytopenia

First patient dosed in study testing the safety, tolerability, pharmacokinetics and preliminary efficacy of HMPL-523; the study, which will enroll 50-60 patients, includes a dose escalation stage and a dose expansion stage

8/23/19

Rockwell Medical Inc., of Wixom, Mich.

Triferic (ferric pyrophosphate citrate)

Iron replacement product

Anemia in end-stage renal disease and chronic kidney disease

Reported 2 studies in China; first study in healthy volunteers suggests pharmacokinetic parameters similar to U.S. subjects; second study suggests administration of dialysate Triferic in Chinese patients shows same iron transfer as U.S. hemodialysis patients

8/8/19

Immune

Bio-Thera Solutions Ltd., of Guangzhou, China

BAT-2506

Biosimilar to Simponi (golimumab)

Healthy volunteers

Dosed first of about 182 subjects in study comparing BAT-2506 to EU-sourced Simponi

8/14/19

Cyxone AB, of Stockholm

T-20-K

Anti-inflammatory

Multiple sclerosis

Achieved objective of determining safety and tolerability in healthy male volunteers

8/2/19

Denali Therapeutics Inc., of South San Francisco, and Sanofi SA, of Paris

DNL-758

Receptor-interacting serine/threonine-protein kinase 1 inhibitor

Healthy volunteers (eventually autoimmune diseases)

First patient dosed in study

8/5/19

Provention Bio Inc., of Oldwick, N.J.

PRV-3279

Humanized diabody targeting B-cell surface proteins CD32B and CD79B

Systemic lupus erythematosus

Dosed first patients in Prevail phase Ib/IIa study; part 1 will enroll healthy volunteers, while part 2 will involve SLE patients

8/8/19

Infection

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

ACX-362E

Pol IIIC inhibitor

Clostridioides difficile infection

Completed a 68-subject trial in healthy volunteers; multiple dose levels up to 450 mg bid for 10 days were shown to be well tolerated; any adverse events attributed to ACX-362E were mild, transitory, and did not interrupt treatment in any subject

8/28/19

Bioaegis Therapeutics Inc., of Morristown, N.J.

Recombinant human plasma gelsolin

Recombinant human plasma gelsolin

Community-acquired pneumonia

Phase Ib/IIa findings showed no serious or drug-related adverse events at the highest doses and analysis of pharmacokinetics demonstrated product half-life exceeded 12 to 16 hours, supporting once-daily dosing

8/7/19

Enlivex Therapeutics Ltd., of Ness Ziona, Israel

OTS Allocetra

Off-the-shelf immunotherapy

Severe sepsis

After reviewing the first 2 cohorts of 6 total patients in phase Ib trial, the safety review committee recommended enrolling the last cohort of 4 patients; drug was well-tolerated when used with standard-of-care treatment and all patients recovered from their severe sepsis

8/19/19

Hepion Pharmaceuticals Inc., of Edison, N.J.

CRV-431

Cyclophilin inhibitor

Hepatitis B virus infection

Dosed first patient in the 28-day multiple ascending-dose study testing 4 dose levels (75 mg to 375 mg) of CRV-431 plus Viread (tenofovir disoproxil fumarate, Gilead Sciences Inc.); a fifth cohort will test CRV-431 at 225 mg in HBV patients with Metavir scores of F2/F3

8/14/19

Musculoskeletal

Discgenics Inc., of Salt Lake City

ITCT

Allogeneic discogenic cells from intervertebral disc tissue

Mild to moderate degenerative disc disease

First patient was treated in a Japanese safety study comparing 2 dosage levels with a sham control

8/7/19

Neurology/Psychiatric

AC Immune SA, of Lausanne, Switzerland

ACI-35.030

Anti-phospho-tau vaccine

Alzheimer's disease and other tauopathies

Trial begun

8/1/19

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

AAV-CLN6

Gene therapy

CLN6 Batten disease

Interim results show evidence of disease stabilization in 7 of 8 children with data for up to 2 years post-treatment

8/1/19

Neuropore Therapies Inc., of San Diego

NPT520-34

Reduces astrocytic and microglial markers of neuroinflammation

Healthy volunteers (eventually amyotrophic lateral sclerosis)

Completed single-dose, safety and food effect studies; started study testing safety, tolerability and pharmacokinetics of repeated doses

8/14/19

Renetx Bio Inc., of New Haven, Conn.

AXER-204

Fusion protein that binds Nogo-A, MAG and Omgp

Chronic spinal cord injury

Treated first of about 24 patients in the Reset study testing the safety, tolerability and pharmacokinetics of the drug

8/14/19

Trevena Inc., of Chesterbrook, Pa.

Oliceridine

Opioid

Healthy volunteers

Completed enrollment in the QT study; top-line data expected in 4Q19

8/28/19

Ocular

Aerie Pharmaceuticals Inc., of Durham, N.C.

AR-13503 SR

Sustained-release implant of an inhibitor of Rho kinase and protein kinase C

Neovascular age-related macular degeneration or diabetic macular edema

Started dosing in the AR-13503-CS201 study that includes a dose-escalation phase in up to 12 patients followed by a 90-patient phase testing 2 doses of the implant either as a monotherapy or in combination with Eylea (aflibercept, Regeneron Pharmaceuticals Inc.), compared to Eylea alone

8/20/19

Regenxbio Inc., of Rockville, Md.

RGX-314

NAV AAV8 vector containing gene encoding for a monoclonal antibody fragment

Wet age-related macular degeneration

Interim update from phase I/IIa trial showed dose-dependent protein expression levels across all 5 dose cohorts; 5-% of subjects in cohort 3 remain free of anti-VEGF injections at 18 months

8/8/19

Other/Miscellaneous

Arch Biopartners Inc., of Toronto

Metablok

Peptide that blocks dipeptidase-1

Healthy volunteers (eventually inflammation, sepsis and cancer metastasis)

Started the single ascending dose arm of the 44-subject study to test the safety and pharmacokinetic profile of the drug; results expected later in 2019

8/22/19

Vivus Inc., of Campbell, Calif.

Qsymia (phentermine and topiramate extended-release)

Enhances satiety

Obesity

Data from 25 patients undergoing laparoscopic sleeve gastrectomy surgery published in Surgery for Obesity and Related Diseases showed patients treated with Qsymia lost 11.2% more of their initial body weight at 2 years post-surgery compared with controls (p=0.007); 61.5% of patients treated with Qsymia achieved a body mass index of less than 40 at 24 months post-surgery, compared to 47.5% of the control group

8/20/19


Notes

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

 

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