Company Product Description Indication Phase I status

Cancer

4D pharma plc, of Leeds, U.K. MRx-0518 Live biotherapeutic product Advanced malignancies after response to and progression on PD-1/PD-L1 inhibitors MRx0518 plus Keytruda (pembrolizumab, Merck & Co. Inc.) produced a partial response in 2 of 6 patients; 1 patient had stable disease; 2 patients withdrew due to progression; 1 patient withdrew due to disease-related serious adverse event prior to the restaging scan
Adaptimmune Therapeutics plc, of Oxfordshire, U.K. ADP-A2M4 Autologous T cells Soft tissue sarcoma Clinical responses in 7 of 14 patients with synovial sarcoma by RECIST 1.1; clinical benefit in 13 out of 14 patients
ADC Therapeutics SA, of Lausanne, Switzerland ADCT-402 (loncastuximab tesirine) Antibody-drug conjugate targeting CD19 CD19-positive relapsed/refractory B-cell non-Hodgkin lymphoma Data from 61 evaluable patients published in Clinical Cancer Research showed a 55% response rate at doses of ?120 µg/kg
Alx Oncology (previously Alexo Therapeutics Inc.), of Burlingame, Calif. ALX-148 CD47 antagonist Solid tumors As of Sept. 23, in 82 individuals with advanced disease who received drug in combination with standard regimens of trastuzumab (Herceptin, Roche Holding AG, n=30) or pembrolizumab (Keytruda, Merck & Co. Inc., n=52), those with HER2-positive gastric/gastroesophageal junction cancer (n=19) whose tumors progressed on systemic (including HER2-targeted) therapy showed objective response rate (ORR) of 21% and disease control rate (DCR) of 26%; those with squamous cell carcinoma of the head and neck (n=20) who progressed on platinum therapy showed ORR of 20% and DCR of 30%; ORR of 40% and DCR of 40% seen in checkpoint inhibitor-naïve participants (n=10)
Aravive Inc., of Houston AVB-500 Growth arrest specific protein 6 ligand inhibitor Ovarian cancer Data from first 31 participants with platinum-resistant recurrent disease treated at 10 mg/kg in ongoing phase Ib/II trial showed high serum drug levels were predictive of antitumor activity with statistically significant correlation to primary endpoint of progression-free survival (PFS; p=0.0066); those who met or exceeded minimal efficacious drug concentration showed greater than 4-fold increase in median PFS over those with low exposure (8.1 vs. 1.8 months; p=0.0016) and approximately 2-fold improvement in overall response rate (29% vs. 14%), including 1 complete response
Aravive Inc., of Houston, and Astrazeneca plc, of Cambridge, U.K. AVB-500 GAS6/AXL inhibitor Recurrent epithelial ovarian cancer Started recruitment in a phase I/II trial, testing combination with durvalumab, in patients with platinum-resistant disease
Bicycle Therapeutics plc, of Cambridge, U.K. BT-5528 Bicycle toxin conjugate targeting tumor antigen EphA2 Advanced solid tumors associated with EphA2 expression Dosed first patient in phase I/II study
Celldex Therapeutics Inc., of Hampton, N.J. CDX-1140 Anti-CD40 monoclonal antibody Advanced solid tumors Interim data from 38 patients with pre- and post-scans, showed 2 patients with radiographic evidence of tumor necrosis among 5 patients with recurrent/refractory head and neck squamous cell carcinoma; a patient with gastroesophageal carcinoma had 41% shrinkage of liver and lymph node target lesions; 6 patients had stable disease, including 4 on monotherapy and 2 on CDX-1140/CDX-301 combination
Clover Biopharmaceuticals Inc., of Chengdu, China SCB-313 TRAIL-Trimer fusion protein Malignant pleural effusions in cancer First patient dosed
Compugen Ltd., of Holon, Israel COM-701 Anti-PVRIG antibody Advanced solid tumors Data from ongoing study show initial signals of antitumor activity; well-tolerated with no dose-limiting toxicities observed
Corvus Pharmaceuticals Inc., of Burlingame, Calif. CPI-006 Anti-CD73 antibody Cancers that have failed standard therapies Tumor regression seen in 4 of 9 evaluable patients receiving 6 mg/kg and higher
Cstone Pharmaceuticals Co. Ltd., of Suzhou, China Tibsovo (ivosidenib) IDH1 gene inhibitor Acute myeloid leukemia First participant dosed in bridging registrational study in China designed to validate efficacy, safety and pharmacokinetics in patients with IDH1-mutant relapsed or refractory disease
Deciphera Pharmaceuticals Inc., of Waltham, Mass. DCC-3014 CSF-1 antagonist Tenosynovial giant cell tumor Preliminary data from ongoing study in 3 individuals with diffuse-type TGCT enrolled in dose-escalation portion showed tumor reductions from baseline of 48%, 25% and 24%, respectively, with 1 confirmed partial response sustained for 9 months and ongoing who showed 84% tumor reduction from baseline to cycle 10 day 1; symptomatic improvements in mobility and reduced pain also reported
Delta-Fly Pharma Inc., of Tokushima, Japan DFP-14927 Pegylated-DFP-10917 (nucleoside analogue) Advanced solid tumors Study initiated at MD Anderson Cancer Center
Dragonfly Therapeutics Inc., of Waltham, Mass. DF-1001 TriNKET candidate Advanced solid tumors Dosed first patient in phase I/II study; recruiting patients whose tumors express HER2
Effector Therapeutics Inc, of San Diego Zotatifin Selective small-molecule inhibitor of eukaryotic initiation factor 4A Advanced solid tumor malignancies Phase I/II study of zotatifin as monotherapy is initiated
Epicentrx Inc., of La Jolla, Calif. RRx-001 Small-molecule immunotherapy that targets the CD47-SIRP? axis Newly diagnosed glioblastoma RRx-001 combination therapy (radiotherapy + temozolomide) led to overall survival of 21.9 months, compared with historical OS data of 15-20 months; the RRx-001 combo also led to progression-free survival of 13 months, compared with historical PFS data of 6-7 months
Excellthera Inc., of Montreal ECT-001 UM-171-expanded allogeneic umbilical cord-derived CD34+ hematopoietic stem cell therapy Hematological malignancies Full data from first trial, published in The Lancet Haematology, indicated ECT-001 therapy is feasible, with low transplant-related mortality, low incidence of severe acute graft-vs.-host disease (GVHD) and absence of moderate to severe chronic GVHD; small cords may be used without compromising engraftment
Forma Therapeutics Inc., of Watertown, Mass. Olutasidenib Inhibitor of mutated isocitrate dehydrogenase 1 IDH1-mutated gliomas Demonstrated blood-brain barrier penetration as measured by cerebrospinal fluid exposure and disease control; confirmed partial response in 1 patient and stable disease in 10 patients with enhancing glioma with a median duration of treatment of 3.7 months
Formation Biologics Inc., of Austin, Texas AVID-200 Inhibitor of TGF-beta 1 & 3 Advanced or metastatic solid tumor malignancies Dose-escalation phase Ia study complete with 15 patients treated at 3 doses; several patients experienced stable disease, including 1 patient with prolonged stable disease
Harbour Biomed Therapeutics Ltd., of Cambridge, Mass. HBM-4003 Heavy-chain only antibody targeting CTLA4 Advanced solid tumors Started study
Ideaya Biosciences Inc., of South San Francisco IDE-196 Protein kinase C inhibitor Metastatic uveal melanoma 27 participants enrolled in dose-escalation portion, including 12 in MUM dose-limiting toxicity (DLT) cohorts, 14 in MUM overflow cohort and 1 with GNA11 cutaneous melanoma in non-MUM GNAQ/11 cohort; all in DLT cohorts remained on therapy with duration of treatment ranging from 1.3 to 4 months as of Oct. 28 data cut-off with no reported DLTs or adverse events > grade 3
Immodulon Therapeutics Ltd., of Uxbridge, U.K. IMM-101 Contains heat-killed whole cell Mycobacterium obuense Advanced melanoma Long-term survival data in patients with stage III/IV disease show 9 surviving patients at 5 years, and 8 surviving at 8 years
IMV Inc., of Dartmouth, Nova Scotia DPX-Survivac Survivin-based peptides Ovarian cancer In connection with ongoing phase Ib/II DeCidE1 study, immune-profiling of peripheral blood mononuclear cell and tumor samples suggests treatment regimen induced robust and sustained survivin-specific T-cell responses from nearly all evaluable subjects and T-cell infiltration into tumors without loss of functionality; comparison of T-cell receptor ?-chain repertoire analyses between pre- and on-treatment tumor biopsies shows new clonotypes can represent up to 90% of the intratumoral T-cell population
Innovent Biologics Inc., of Suzhou, China, and Hanmi Pharmaceutical Co. Ltd., of Beijing IBI-315 Recombinant bispecific antibody targeting PD-1 and HER2 HER2-expressing advanced solid malignancies First patient dosed; trial to determine safety, tolerability, initial efficacy and recommended phase II dose, either as monotherapy or in combination with chemotherapy
Intensity Therapeutics Inc., of Westport, Conn. INT230-6 Cisplatin and vinblastine plus a penetration enhancer molecule Advanced or metastatic solid tumors Disease control rate of 39% in 45 patients; 7 of 8 patients who had over 50% of total tumor burden injected had disease control
Molecular Templates Inc., of Austin, Texas, and Translational Research in Oncology, OF Edmonton, Alberta MT-5111 EGFR modulator; Erbb2 tyrosine kinase receptor modulator HER2-positive solid tumors Enrolled first participant in study of MT-5111 as monotherapy in individuals with advanced disease to evaluate safety and tolerability and determine recommended phase II dose
Moleculin Biotech Inc., of Houston WP-1220  Inhibitor of p-STAT3 Cutaneous T-cell lymphoma Data published in Blood showed 3 patients had a median reduction of 70.8% in their CAILS scores on day 84; fourth ongoing patient had a 26.7% reduction after 56 days
Neon Therapeutics Inc., of Cambridge, Mass. NEO-PV-01 Neoantigen vaccine Advanced or metastatic melanoma, smoking-associated non-small-cell lung cancer and bladder cancer Of patients who started treatment in phase Ib trial, objective response rates were 59%, 39% and 27% in 27 melanoma patients, 18 NSCLC patients and 15 bladder cancer patients, respectively
Oncolytics Biotech Inc. of San Diego Pelareorep Isolate of an unmodified reovirus Early breast cancer In the window-of-opportunity Aware-1 study, initial data showed replication of pelareorep occurs exclusively in tumor tissue and there was an increase in CelTIL score, a biomarker of T-cell clonality
Oncolytics Biotech Inc., of Calgary, Alberta Pelareorep Immuno-oncolytic virus Pancreatic adenocarcinoma Findings from phase Ib REO 024 study of combination with pembrolizumab (Keytruda, Merck & Co. Inc.), published in Clinical Cancer Research, showed objective responses indicating antitumor activity, long-term disease stabilization and promotion of inflamed phenotype in tumors
Oncternal Therapeutics Inc., of San Diego TK-216 Small-molecule inhibitor of ETS transcription factor oncoproteins Relapsed or refractory Ewing sarcoma Dose-finding arm nearing completion, after which company intends to begin enrolling in an expansion cohort to evaluate clinical response of treatment in combination with vincristine
Oncternal Therapeutics Inc., of San Diego TK-216 Small-molecule inhibitor of E26 transformation-specific family of oncoproteins Relapsed or refractory Ewing sarcoma Interim data show 1 of 2 patients treated in the current, highest exposure dose cohort of Study TK216-01 is without evidence of Ewing sarcoma after 8 months on study, and has tolerated treatments with TK-216 alone or combined with vincristine
Phoenix Molecular Designs Ltd., of Vancouver, British Columbia PMD-026 Ribosomal protein S6 kinase-2 inhibitor Breast cancer First participant dosed in phase I/Ib study enrolling individuals with metastatic breast cancer in dose-escalation phase as well as those with triple-negative breast cancer in phase Ib; study includes certified companion diagnostic designed to detect RSK2 activation in breast tumors and correlate response
Replimune Group Inc., of Woburn, Mass. RP-1 Engineered herpes simplex virus Advanced cancers refractory to available therapy Monotherapy produced tumor destruction, including delayed systemic post-study tumor reduction without further therapy; combination with Opdivo (nivolumab, Bristol-Meyers Squibb Co.) produced antitumor activity in multiple patients with a variety of tumor types, particularly in cutaneous squamous cell carcinoma and melanoma and also in microsatellite instability high colorectal cancer and esophageal cancer patients; first 3 of 4 patients with anti-CTLA4 and anti-PD-1-refractory cutaneous melanoma treated with RP1 combined with Opdivo responded to therapy
Sensei Biotherapeutics Inc., of Gaithersburg, Md. SNS-301 Nanoparticle-based immunotherapeutic cancer vaccine Head and neck tumor Dosed first patient in phase I/II trial in patients with ASPH?positive disease who previously received immune checkpoint inhibitors
Spring Bank Pharmaceuticals Inc., of Hopkinton, Mass. SB-11285 Stimulator of interferon gene agonist Advanced solid tumors First participants dosed in dose-escalation study with SB-11285 alone followed by combination with checkpoint inhibitor to determine recommended phase II dose; part 2 of trial will explore antitumor activity of intravenous drug in combination with checkpoint inhibitor
Syndax Pharmaceuticals Inc., of Waltham, Mass. SNDX-5613 Selective, oral Menin inhibitor Relapsed/refractory acute leukemia First patient dosed in Augment-101 phase I/II trial; initial data expected in 2020
Turning Point Therapeutics Inc., of San Diego TPX-0046 RET tyrosine kinase receptor inhibitor Advanced solid tumors Initiated study expected to enroll about 50 TKI treatment-naïve and pretreated patients with RET-altered non-small-cell lung, thyroid and other advanced cancers in phase I dose-escalation portion and about 300 in multiple cohorts in phase II expansion
Turning Point Therapeutics Inc., of San Diego Repotrectinib Tyrosine kinase inhibitor targeting ROS1 ALK-, NTRK- and ROS1-positive solid tumors Started phase I/II trial in pediatric patients; about 12 patients will be enrolled in dose-escalation portion followed by enrollment of 63 patients across 3 cohorts in dose-expansion cohort
Vaccibody AS, of Oslo VB10.NEO Cancer vaccine Various tumors Preliminary data from ongoing VB N-01 phase I/IIa trial showed clinical responses observed after treatment start in 50% of all analyzed patients across tumor types; all 4 head and neck cancer patients, the melanoma patient, the non-small-cell lung cancer patient and 1 of 8 renal cancer patients showed clinical response
VBI Vaccines Inc., of Cambridge, Mass. VBI-1901 GM-CSF receptor agonist Glioblastoma 33% reduction in tumor size observed in 1 patient in part B of ongoing phase I/IIa study; analysis of vaccine responders in part A showed 6-month overall survival for 6/6 participants vs. just 5/8 vaccine nonresponders
Y-mabs Therapeutics Inc., of New York Omburtamab B7-H3-targeting antibody Desmoplastic small round cell tumor Updated data from 33 patients who underwent gross total resection (GTR) showed those treated with omburtamab plus whole abdominopelvic intensity-modulated radiotherapy (WA-IMRT) had median overall survival of 59 months vs. 41 months for those receiving WA-IMRT only; adding omburtamab to GTR improved 5-year Kaplan Meier estimated OS from historical rate of about 20% to about 40%
Ziopharm Oncology Inc., of Phoenix Ad-RTS-hIL-12 plus veledimex Targets IL-12 Recurrent or progressive glioblastoma multiforme Interim analysis from ongoing substudy in combination with low-dose dexamethasone showed decrease in tumor from baseline, resulting in patient’s lesion being too small to measure, assessed as partial response, with follow-up ongoing; data from ongoing substudy in combination with anti-PD-1 drug Opdivo (nivolumab, Bristol-Myers Squibb Co.) showed decrease by 64% in patient’s tumor from baseline, resulting in partial response, with follow-up ongoing
Zymeworks Inc., of Vancouver ZW-25 Erbb2 tyrosine kinase receptor inhibitor Solid tumors Updated data in heavily pretreated patients show overall disease control rate was 70%, with 44% of participants seeing partial responses and 26% with stable disease, and 32% with disease control for greater than 6 months
Company Product Description Indication Phase I status

Cardiovascular

Celecor Therapeutics Inc., of San Diego RUC-4 Platelet GPIIb/IIIa inhibitor ST-elevation myocardial infarction Study met primary endpoint; 0.075-mg/kg subcutaneous dose provided rapid (<15 minute following administration), intense (>80%) and sustained (<2 hours) inhibition of platelet aggregation
Inflazome Ltd., of Dublin Somalix Inhibitor of the NLRP3 inflammasome Healthy volunteers (eventually cardiovascular disease, arthritis and other diseases) Study expected to be complete in March 2020
Triple-Gene LLC, of Germantown, Md., a subsidiary of Intrexon Corp. INXN-4001 Nonviral delivery of constitutively expressed multigenic plasmid designed to express human S100A1, SDF-1? and VEGF165 gene products Heart failure Completed enrollment in study investigating delivery via retrograde coronary sinus infusion in patients with implanted left ventricular assist device for mechanical support of end-stage heart failure, either as a bridge to transplant or destination therapy
Company Product Description Indication Phase I status

Dermatologic

Celldex Therapeutics Inc., of Hampton, N.J. CDX-0159 Kit tyrosine kinase inhibitor Urticaria Phase Ia study in health volunteers started
Company Product Description Indication Phase I status

Endocrine/metabolic

Acasti Pharma Inc., of Laval, Quebec Capre Omega-3 phospholipid Severe hypertriglyceridemia Data published in Clinical Therapeutics showed drug was well-tolerated in healthy subjects when given as multiple oral doses of 1 g, 2 g and 4 g per day; pharmacokinetic parameters appeared to be dose proportional over the dose range; bioavailability did not appear to be meaningfully affected by fat content of meal consumed before administration
Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. ARO-APOC3 Double-stranded oligomer RNAi targeting APOC3 Hypertriglyceridemia Dose-dependent reductions in serum APOC3 observed; no serious or severe adverse events
Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. ARO-ANG3 Double-stranded oligomer RNAi targeting ARO-ANG3 Homozygous familial hypercholesterolemia Dose-dependent reductions in serum ANGPTL3 observed; no drug-related severe or serious adverse events
Cohbar Inc., of Menlo Park, Calif. CB-4211  Analogue of MOTS-c, a mitochondrial-derived peptide Nonalcoholic steatohepatitis Phase Ia completed; initiated phase Ib, both for obese patients with nonalcoholic fatty liver diseases
Diurnal Group plc, of Cardiff, U.K. Ditest (native oral testosterone) Androgen receptor agonist Hypogonadism Primary endpoint, which compared rate and absorption of testosterone from single 120-mg dose with single dose of testosterone undecanoate 80 mg in fed state, was met in 24-patient study; Ditest achieved testosterone levels within healthy young male adult normal range after oral administration with less variability than testosterone undecanoate
Company Product Description Indication Phase I status

Gastrointestinal

Forma Therapeutics Inc., of Watertown, Mass. FT-4101 Fatty acid synthase inhibitor Healthy volunteers (eventually nonalcoholic steatohepatitis) FT-4101 at 6 mg and 9 mg reduced hepatic de novo lipogenesis by 42.6% (p=0.010) and 81.8% (p<0.0001), respectively, through 24 hours compared to placebo
Poxel SA, of Lyon, France PXL-065 (deuterated pioglitazone R-enantiomer) Mitochondrial pyruvate carrier inhibitor Nonalcoholic steatohepatitis Dosed at 7.5, 22.5 and 30 mg compared to 45 mg Actos (pioglitazone, Takeda Pharmaceutical Co. Ltd.) in 24 healthy participants, study drug showed favorable safety and tolerability with no serious adverse events; pharmacokinetic assessment showed plasma exposure increased in dose-proportional manner following oral administration; dose-independent stabilization of R-pioglitazone with deuterium seen at all tested doses; phase II trial expected to start in second quarter of 2020
Company Product Description Indication Phase I status

Genitourinary/sexual function

Arch Biopartners Inc., of Toronto Metablok Peptide blocking leukocyte recruitment Healthy volunteers (eventually acute kidney injury in cardiac surgery patients) First 3 single ascending-dose cohorts met the primary endpoints of safety and tolerability; started 3-day, multiple ascending-dose cohorts of 8 patients per cohort, which should complete by the end of November
Company Product Description Indication Phase I status

Hematologic

Crispr Therapeutics AG, of Zug, Switzerland, and Vertex Pharmaceuticals Inc., of Cambridge, Mass. CTX-001 CRISPR/Cas9 gene-editing therapy Transfusion-dependent beta thalassemia and severe sickle cell disease TDT patient was transfusion independent 9 months after treatment with total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin and 99.8% F-cells (erythrocytes expressing fetal hemoglobin); SCD patient was free of vaso-occlusive crises at 4 months with total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin and 94.7% F-cells
X4 Pharmaceuticals Inc., of Cambridge, Mass. Mavorixafor (X4P-001) Inhibitor of CXCR4 Neutropenia Initiated 14-day phase Ib trial designed to assess the safety and tolerability; trial also will evaluate neutrophil response when testing drug as an independent agent or in combination with G-CSF; trial will enroll up to 45 patients
Company Product Description Indication Phase I status

Immune

Celltrion Healthcare Co. Ltd., of Incheon, South Korea CT-P13 SC (infliximab biosimilar) TNF-alpha ligand inhibitor Rheumatoid arthritis In CT-P13 subcutaneous (SC) arm, mean serum concentration increased from week 6 and maintained generally consistent level from weeks 14 to 54 on biweekly injections; in those who switched from intravenous to SC at week 30, mean serum concentration gradually increased and also maintained consistent level to week 54, demonstrating noninferiority of SC formulation
Evelo Biosciences Inc., of Cambridge, Mass. EDP-1815 Monoclonal microbial Mild to moderate psoriasis Interim phase Ib data showed at the end of 28-day dosing period, high-dose cohort showed mean reduction in Lesion Severity Score; after dosing period, the high dose cohort showed continued reductions from baseline in the mean Lesion Severity Score and Psoriasis Area and Severity Index
Kezar Life Sciences Inc., of South San Francisco KZR-616 Selective immunoproteasome inhibitor Systemic lupus erythematosus Updated data from phase Ib portion of Mission study show differentiated safety profile from nonspecific proteasome inhibitors; dose of 60 mg is well-tolerated when administered via step-up dosing; KZR-616 appears to exhibit broad immunomodulatory activity
Company Product Description Indication Phase I status

Infection

Assembly Biosciences Inc., of South San Francisco ABI-H2158 Hepatitis B structural protein modulator Hepatitis B virus infection Interim data from phase Ib study, currently enrolling HBeAg-positive patients in sequential dose cohorts, showed antiviral activity from initial dose level of 100 mg, which produced mean declines of 2.3 log10 [range 1.7 – 3] and 2.1 log10 [range 1.5 - 2.7] from baseline to day 15 in HBV DNA and pgRNA, respectively
Atriva Therapeutics GmbH, of Tübingen, Germany ATR-002 MEK inhibitor of viral replication Healthy volunteers (eventually influenza) ATR-002 was considered to be safe and well-tolerable in the single and multiple ascending-dose study; pharmacokinetic profile supports once-daily dosing
Enlivex Therapeutics Ltd., of Ness Ziona, Israel OTS Allocetra Rebalances hyperimmune responses Severe sepsis None of the 6 patients treated with OTS Allocetra died within 28 days, compared to 29% of 37 matched controls; 100% of patients treated with OTS Allocetra recovered from sepsis within 28 days compared to 48% of matched control group; average maximal organ failure score was 4.5 for patients treated with OTS Allocetra with no patients experiencing increasing scores after admission, compared to 8.11 for the control group with 78% experiencing an increase in post-admission score
Enlivex Therapeutics Ltd., of Ness Ziona, Israel Allocetra Autologous mononuclear-enriched leukocytes plus the patients' own cells Sepsis Completed recruitment of 10 patients with severe sepsis for phase Ib trial
Gilead Sciences Inc., of Foster City, Calif. GS-6207 Capsid inhibitor HIV infection In healthy subjects, pharmacokinetics suggest drug has potential to be dosed up to every 6 months; in HIV patients at 20 mg to 450 mg, mean maximum reduction in HIV-1 RNA by day 10 was 1.4 to 2.2 log10copies/mL (p<0.0001 for all)
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole inhalation powder Triazole antifungal Pulmonary aspergillosis Trial begun
Valneva SE, of Saint Herblain, France VLA-1553 Live-attenuated vaccine Chikungunya virus infection With sustained immunogenicity profile in all dose groups, plans to accelerate program to pivotal phase III trial in 2020 (subject to FDA agreement); 100% seroconversion achieved at ay 14 after a single vaccination
VBI Vaccines Inc., of Cambridge, Mass., and Brii Biosciences Inc., of Durham, N.C. BRII-179 (VBI-2601) Recombinant, protein-based virus-like particle vaccine Hepatitis B virus infection Dosed first of expected 65 participants in phase Ib/IIa study assessing safety, tolerability and antiviral activity; initial data expected in second half of 2020
Company Product Description Indication Phase I status

Musculoskeletal

Anelixis Therapeutics Inc., of Cambridge, Mass. AT-1501 Anti-CD40L antibody Amyotrophic lateral sclerosis Completed study in healthy volunteers and participants with ALS; drug was well-tolerated at all doses
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. BCX-9250 Oral activin receptor-like kinase-2 inhibitor Fibrodysplasia ossificans progressiva Started trial evaluating single and multiple ascending doses in healthy volunteers; results expected in second half of 2020
Company Product Description Indication Phase I status

Neurology/psychiatric

Imbrium Therapeutics LP, subsidiary of Purdue Pharma LP, of Stamford, Conn., and Spinethera Inc., of Plymouth, Minn. SX-600 (reformulated dexamethasone acetate, epidural/sustained release) Glucocorticoid receptor agonist Pain First dose administered in phase I/II Salient study evaluating efficacy and safety of 12.5-mg and 25-mg doses to treat sciatica
Inflazome Ltd., of Dublin Inzomelid  Inhibitor of the NLRP3 inflammasome Healthy volunteers (eventually neuroinflammatory and neurodegenerative diseases) Study expected to be complete in January 2020
Kadimastem Ltd., of Ness Ziona, Israel Astrorx Astrocytes derived from human embryonic stem cells Amyotrophic lateral sclerosis Plans to submit amendment seeking permission to test repeated low dose instead of medium dose, as originally planned
Lineage Cell Therapeutics Inc., of Carlsbad, Calif. OPC1 Oligodendrocyte progenitor cell therapy Acute spinal cord injury Updated data from ongoing Scistar study showed continued overall safety profile, with robust motor recovery in upper extremities maintained through year 2 follow-ups available to date; for cohort 1, patients continue to be stable 2-4 years post treatment; 5 of 6 cohort 2 patients achieved at least 2 motor levels improvement over baseline on at least 1 side; 1 cohort 2 patient achieved 3 motor levels improvement on 1 side over baseline, with improvement maintained through 36-month follow-up visit; improvements in Upper Extremity Motor Score (UEMS) in cohort 2 observed at 24 months, with 3 having recovered back to UEMS in mid-to-high 40s
Seelos Therapeutics Inc., of New York SLS-002 (intranasal racemic ketamine) NMDA receptor antagonist Suicidal ideation First of expected 48 healthy volunteers dosed in drug-drug interaction (DDI) study in combination with venlafaxine ER or sertraline to evaluate pharmacokinetic profile, DDI and safety measures
Seelos Therapeutics Inc., of New York SLS-002 Intranasal racemic ketamine Major depressive disorder Dosed first subjects in pharmacokinetic and pharmacodynamic study expected to enroll 62 healthy volunteers; preliminary data expected in first quarter of 2020
Trevena Inc., of Chesterbrook, Pa. Oliceridine Opioid receptor mu agonist Pain Pharmacokinetic studies of intravenous drug in individuals with end-stage renal disease and in those with hepatic impairment, published in Clinical Pharmacology in Drug Development, showed no dose adjustments needed in those populations
Vivus Inc., of Campbell, Calif. Qsymia (phentermine and topiramate ER) Adrenergic receptor agonist Binge-eating disorder; bulimia nervosa Findings from study that enrolled 22 participants, published in International Journal of Eating Disorders, showed mean objective binge-eating days declined over 4 weeks from 16.2 at baseline to 4.2 and 13.2 days for study drug and placebo, respectively (p<0.0001); abstinence rates were 63.6% with Qsymia and 9.1% with placebo (p<0.0001); Qsymia was associated with mean weight decrease of 5.8 kg compared with mean weight gain of 0.4 kg on placebo
Company Product Description Indication Phase I status

Ocular

Oyster Point Pharma Inc., of Princeton, N.J. Varenicline Nicotinic acetylcholine receptor agonist Dry eye disease Top-line results indicate the relative bioavailability (systemic exposure as defined by adjusted geometric mean AUC0-inf) was 13 times lower for single dose of highest strength of OC-01 nasal spray as compared to single dose of highest strength Chantix (varenicline, Pfizer Inc.) tablet (7.46 vs. 99.67 h*ng/ml)
Company Product Description Indication Phase I status

Other/miscellaneous

Ligand Pharmaceuticals Inc., of San Diego CE-Iohexol Captisol-enabled contrast agent Healthy volunteers (eventually X-ray imaging) Pharmacokinetic parameters of area under the concentration-time curve and maximum concentration were similar between CE-Iohexol and Omnipaque (iohexol, GE Healthcare) with a geometric mean ratio of 1 for both; mean elimination constant was 0.3/hour for both treatments

Toxicity/intoxication

Adamis Pharmaceuticals Corp., of San Diego Zimhi (naloxone) Opioid antagonist Healthy volunteers Data published in the Journal of Addiction Research and Adolescent Behavior showed intramuscular Zimhi produced higher levels of naloxone with more rapid absorption compared to intranasal Narcan (naloxone, Emergent Biosolutions Inc.)

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