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Phase I clinical data reported in March 2020

Company Product Description Indication Phase I status


4D pharma plc, of Leeds, U.K.
Single strain live biotherapeutic product Advanced solid tumors Additional interim data from first 6 participants in part A of ongoing phase I/II combination trial with anti-PD-1 Keytruda (pembrolizumab, Merck & Co. Inc.) showed 2 partial responses with evidence of tumor shrinkage who remain on study (1 for > 10 months) and 1 with stable disease who remains on study for > 8 months; evidence of increased tumor-infiltrating lymphocytes seen following therapy
Actinium Pharmaceuticals Inc., of New York Actimab-A Antibody radiation conjugate that targets CD33 receptor on blood cancer cells and delivers cytotoxic radiation via radioisotope Actinium-225 Relapsed or refractory acute myeloid leukemia First patient treated in third cohort
Aptevo Therapeutics Inc., of Seattle APVO-436 Anti-CD123, anti-CD3 targeted bispecific antibody Acute myeloid leukemia; myelodysplastic syndrome Trial advancing on schedule with dosing in cohort 6 set to begin shortly; dose levels now entering potential therapeutic range; no evidence of dose-limiting toxicities observed in cohort 5; no evidence of drug-induced anti-drug antibodies observed to date
Aravive Biologics Inc., of Houston AVB-500 GAS6/AXL inhibitor Advanced urothelial carcinoma First patient dosed in investigator-sponsored trial in combination with avelumab (Bavencio, EMD Serono Inc./Pfizer Inc.)
Bolt Biotherapeutics Inc., of Redwood City, Calif. BDC-1001 Immune-stimulating antibody conjugate HER2-expressed solid tumors Patient dosing begun in an open-label, dose-escalation and dose expansion study of BDC-1001 monotherapy
CNS Pharmaceuticals Inc., of Houston Berubicin Anthracycline Glioblastoma multiforme The 1 patient out of 25 who had a durable complete response in the 2006 study remains cancer free as of February 2020; company plans to start a phase II study in the second half of 2020
Compugen Ltd., of Holon, Israel COM-701 Antibody targeting PVRIG Advanced solid tumors Updated data from dose-escalation study in patients who have exhausted all available standard therapies show treatment tolerated in eighth cohort, given 20 mg/kg on Q4 weekly dosing schedule, with preliminary antitumor activity in 2 patients, including a partial response in a patient with microsatellite stable primary peritoneal cancer on monotherapy and a confirmed partial response in a patient with microsatellite stable colorectal cancer treated in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.)
Cytodyn Inc., of Vancouver, Wash. Leronlimab CCR5 antagonist Breast cancer Results in first 5 patients showed positive outcomes in metastatic triple-negative breast cancer and metastatic breast cancer
Debiopharm International SA, of Lausanne, Switzerland Debio-0123 WEE-1 inhibitor Advanced solid tumors Trial begun
Faron Pharmaceuticals Oy, of Turku, Finland Clevegen Precision cancer immunotherapy targeting Clever-1-positive tumor-associated macrophages Metastatic or inoperable solid tumors Data monitoring committee advised to continue study as planned
Five Prime Therapeutics Inc., of South San Francisco Bemarituzumab FGFR2b antibody FGFR2b-selected gastroesophageal adenocarcinoma Results published in the Journal of Clinical Oncology showed overall response rate in patients with high FGFR2b-overexpressing GEA was 17.9%, with 5 of 28 patients achieving a confirmed partial response
Forty Seven Inc., of Menlo Park, Calif. FSI-174 cKIT tyrosine kinase inhibitor Hematologic cancer Start of trial in healthy volunteers delayed due to COVID-19
Gemoab GmbH, of Dresden, Germany UniCAR-T-CD123 Switchable CAR T targeting CD123 Late-stage, relapsed/refractory acute leukemia expressing CD123 antigen First patient apheresed in study testing the feasibility, safety and potential efficacy of CAR T with continuous infusion of the CD123-specific targeting module TM123
GT Biopharma Inc., of Beverly Hills, Calif. GTB-3550 Protein immune engager that binds to NK cells and targets them specifically to leukemia cells Relapsed/refractory acute myeloid leukemia  First patient dosed
GT Biopharma Inc., of Beverly Hills, Calif. GTB-3550 Trispecific recombinant fusion protein conjugate composed of variable regions of heavy and light chains of anti-CD16 and anti-CD33 antibodies and modified form of IL-15 Acute myeloid leukemia Started retreatment of first patient to receive lowest dose in phase I/II trial; following initial treatment, patient achieved stable disease with respect to number of AML blasts observed in bone marrow before and after and showed no adverse events
Imcheck Therapeutics SAS, of Marseille, France ICT-01 Gamma delta T cell-activating monoclonal antibody Solid and hematologic tumors Dosed first patient in a phase I/IIa Eviction 2-part, open-label trial to assess the safety, tolerability and activity of ICT-01 as monotherapy and in combination with an immune checkpoint inhibitor in patients with advanced, relapsed/refractory disease
Immutep Ltd., of Sydney Eftilagimod alpha Soluble LAG-3-Ig fusion protein Metastatic breast cancer EOC Pharma completed patient recruitment in the study being conducted in China
Innate Pharma SA, of Marseille, France IPH-5201 Anti-CD39 monoclonal antibody Solid tumor First patient dosed in trial evaluating monotherapy and combination with durvalumab with or without oleclumab
Intensity Therapeutics Inc., of Westport, Conn. INT230-6 Cisplatin and vinblastine plus penetration enhancer molecule Triple-negative breast cancer, Merkel cell carcinoma, chordoma, desmoid tumor and soft tissue sarcoma Safety profile when given with Keytruda (pembrolizumab, Merck & Co. Inc.) appears similar to monotherapy
Inxmed (Shanghai) Co. Ltd., of Shanghai IN-10018 Focal adhesion kinase inhibitor Metastatic uveal, NRAS-mutant melanoma First participant dosed in phase Ib trial as monotherapy and in combination with MEK inhibitor cobimetinib (Roche Holding AG) that will include 6 sites in U.S. and 3 in Australia
Medivir AB, of Boston MIV-818 Prodrug designed to selectively treat liver cancer Liver cancer Pharmacokinetic analysis showed that patients were exposed only to low levels of MIV-818 and troxacitabine outside of the liver, providing experimental support for MIV-818's liver targeting
Medivir AB, of Stockholm MIV-818 Prodrug of troxacitabine Liver tumor First patient dosed in phase Ib study, seeking to establish safety and tolerability profile while exploring efficacy
Mersana Therapeutics Inc., of Cambridge, Mass. XMT-1536 Sodium phosphate cotransporter 2b (NaPi2b) inhibitor Ovarian and non-small-cell lung cancer Ongoing dose-escalation study showed confirmed responses and durable stable disease (SD) in heavily pretreated patients; at 43 mg/m2 dose, 2/7 achieved partial responses (PRs) and 4/7 achieved SD for disease control rate (DCR) of 6/7 (86%); in subset of evaluable patients treated at >30 mg/m2 with higher NaPi2b expression, 5/15 achieved PR and 6/15 achieved SD for DCR of 11/15 (73%); in subset with lower NaPi2b expression, 0/9 achieved PR and 5/9 achieved SD for DCR of 5/9 (55%)
Mina Therapeutics Ltd., of London MTL-CEBPA Up-regulates CCAAT/enhancer binding protein alpha Advanced solid tumors Treated the first patient in the Timepoint study testing the drug in combination with Keytruda (pembrolizumab, Merck & Co. Inc.); study includes dose-escalation phase followed by a dose-expansion phase
Oncolytics Biotech Inc., of San Diego Pelareorep Oncolytic retrovirus Breast cancer Safety committee provided favorable assessment from ongoing Aware-1 combination study with Tecentriq (atezolizumab, Roche Holding AG) in individuals with early stage disease; viral replication in majority of tumors seen in those enrolled in cohort 1, creating pro-inflammatory effect in tumor microenvironment
Oncopeptides AB, of Stockholm Melflufen Caspase-3 stimulator; peptidase modulator Multiple myeloma Findings from phase I/II O-12-M1 combination study with dexamethasone in individuals with relapsed/refractory disease, published in The Lancet Haematology, showed overall response rate of 31%, median duration of response of 8.4 months, median overall survival of 20.7 months and median progression-free survival of 5.7 months
Oncternal Therapeutics Inc., of San Diego Cirmtuzumab Receptor tyrosine kinase-like orphan receptor 1 inhibitor Mantle cell lymphoma Interim data from ongoing phase I/II Cirll combination trial with ibrutinib (Imbruvica, Johnson & Johnson/Pharmacyclics LLC) in relapsed/refractory disease showed 50% complete response (CR) rate in 6 of 12 evaluable patients, including 1 complete metabolic response by PET scan; CRs are ongoing, including 1 at > 21 months on study; data also showed 33% partial response (PR) rate (4 of 12), 17% stable disease rate (2 of 12) and 83% best objective response (CR or PR) rate
PDC*Line Pharma SA, of Liege, Belgium PDC*lung-01 Irradiated human plasmacytoïd dendritic cells loaded with 7 tumor antigens Non-small-cell lung cancer First patient treated in the study designed to to assess the safety, tolerability, immunogenicity and preliminary clinical activity of the drug
Psioxus Therapeutics Ltd., of Oxford, U.K. NG-641 4-transgene tumor-microenvironment modifying cancer gene therapy Solid tumors Started Star study to assess safety, tolerability and preliminary antitumor activity
Redhill Biopharma Ltd., of Tel Aviv, Israel Yeliva (opaganib) Sphingosine kinase 2 inhibitor Cholangiocarcinoma Following full enrollment of 39 people with advanced, unresectable intrahepatic, perihilar and extrahepatic disease evaluable for efficacy, preliminary phase I/IIa data showed signal of activity in multiple participants, leading to opening of second arm evaluating study drug in combination with hydroxychloroquine; third arm evaluating combination with Redhill's serine protease inhibitor, RHB-107 (upamostat), is subject to discussions with FDA
Revolution Medicines Inc., of Redwood City, Calif. RMC-4630 SHP2 inhibitor Solid tumors with RAS mutations focusing on pancreatic cancer Netherlands Cancer Institute, with financial support from the American Association for Cancer Research, plans to start the Sherpa study testing the drug plus an ERK inhibitor in the second half of 2020
Surface Oncology Inc., of Cambridge, Mass. SRF-617 CD39 antagonist Advanced solid tumors Phase I/Ib dose-escalation study initiated with combination arms of gemcitabine and Abraxane, anti-PD-1 or A2A/A2B small-molecule inhibitor AB-928 (Arcus Biosciences Inc.); planned cohorts will focus on pancreatic and gastric cancer and tumors showing resistance to anti-PD-1 therapy; biopsy expansion cohort designed to provide data on changes in tumor tissue CD39 enzymatic activity related to treatment; initial clinical update expected by year-end 2020
Targovax ASA, of Oslo, Norway ONCOS-102 Adenovirus-based cancer therapy Advanced anti-PD1 refractory melanoma Completed enrollment of 12 patients into part 2 of the study testing 12 injections of ONCOS-102 and treatment with Keytruda (pembrolizumab, Merck & Co. Inc.); data expected in the second half of 2020
Tcr2 Therapeutics Inc., of Cambridge, Mass. TC-110 TRuC-T cell candidate targeting CD19 Hematological malignancy  Trial started
Tolero Pharmaceuticals Inc., of Salt Lake City Dubermatinib (TP-0903) AXL receptor tyrosine kinase inhibitor Acute myeloid leukemia Joined The Leukemia & Lymphoma Society in Beat AML Master Clinical Trial for newly diagnosed patients 60 or older; dubermatinib selected for a new arm in the trial for patients with TP53 mutations and/or complex karyotype; primary objectives are to determine the safety and maximum tolerated dose of and evaluate the composite complete response rate
VBI Vaccines Inc., of Cambridge, Mass., VBI-1901 GM-CSF receptor agonist Recurrent glioblastoma Updated data from patients in part A of the study showed the 12-month overall survival rate was 83% for 6 patients who responded to the vaccine, compared to 33% for 9 vaccine nonresponders; median OS for responders was 14 months, compared to 7.75 months for nonresponders
Company Product Description Indication Phase I status


JSC Grindeks, of Riva, Latvia Methyl-?-butyrobetaine (metyl-GBB) Inhibitor of l-carnitine biosynthesis and transport Cardiovascular disease Completed study
Kancera AB, of Stockholm KAND-567 Fractalkine blocker Myocardial infarction Final part of phase Ib program showed desired tolerability profile for intravenous administration complements previous phase I studies that show the same desired characteristics after oral treatment for 7 days
Synlogic Inc., of Cambridge, Mass. SYNB-1618 Synthetic Biotic medicine designed to consume phenylalanine in the gastrointestinal tract Phenylketonuria Enrollment likely slower due to COVID-19 outbreak
Company Product Description Indication Phase I status


Asana Biosciences LLC, of Lawrenceville, N.J. ASN-008 Topical sodium ion channel blocker Pruritus Single application achieved a clinically meaningful reduction in pruritus of ? 4 points on the NRS in 2-3 hours
Biomx Inc., of Ness Ziona, Israel BX-001 Phage therapy Acne Top-line results showed study met primary endpoint of safety and tolerability as well as a statistically significant (p=0.036) reduction of Cutibacterium acnes levels for the high dose vs. placebo
Cabaletta Bio Inc., of Philadelphia DSG3-CAART Desmoglein 3 chimeric autoantibody receptor T cells Mucosal pemphigus vulgaris Anticipates a delay in reporting the acute safety data from the first cohort in DesCAARTes trial due to COVID-19 pandemic
Inmed Pharmaceuticals Inc., of Vancouver, British Columbia INM-755 Cannabinol cream Epidermolysis bullosa and potentially other dermatological diseases Completed enrollment in study of healthy subjects; results expected in second half of 2020
Company Product Description Indication Phase I status


Cyclo Therapeutics Inc., of Gainesville, Fla. Trappsol Cyclo Cholesterol binding agent Niemann Pick disease type C Safety review committee determined that study drug has acceptable safety profile; top-line data expected in May 2020
Orchard Therapeutics plc, of London OTL-201 Autologous lentiviral hematopoietic stem cell SGSH gene therapy Mucopolysaccharidosis type IIIA (Sanfilippo syndrome) Enrollment timelines in ongoing phase I/II program expected to shift by at least 3 months due to COVID-19 pandemic
Company Product Description Indication Phase I status


Allakos Inc., of Redwood City, Calif. Antolimab (AK-002) Sialic acid-binding Ig-like lectin 8 inhibitor Mast cell gastrointestinal disease 6-month treatment resulted in 64% mean reduction in Total Symptom Score (TSS)-8 compared to baseline; 5 of 7 participants had > 50% reduction in TSS-8
Allakos Inc., of Redwood City, Calif. Antolimab (AK-002) Sialic acid-binding Ig-like lectin 8 inhibitor Eosinophil/mast cell-related diseases Placebo-controlled trial initiated to evaluate subcutaneously administered formulation in healthy volunteers
Arrowhead Pharmaceuticals, Inc., of Pasadena, Calif. ARO-HSD RNAi targeting HSD17B13 Nonalcoholic steatohepatitis Treated first patient in the AROHSD1001 study testing safety, tolerability, pharmacokinetics and pharmacodynamic effects
Cohbar Inc., of Menlo Park, Calif. CB4-211 Mitochondria-based therapeutic Nonalcoholic steatohepatitis Anticipating delays in the completion of the CB4-211 phase Ib study as a result of a pause by some CRO partners in response to the COVID-19 pandemic
Pliant Therapeutics Inc., of South San Francisco PLN-1474 Integrin alpha-V/beta-1 antagonist Nonalcoholic steatohepatitis First cohort of healthy participants dosed in dose-escalation trial in program partnered with Novartis AG
Terns Pharmaceuticals Inc., of Foster City, Calif. TERN- 201 Semicarbazide-sensitive amine oxidase Healthy volunteers (eventually nonalcoholic steatohepatitis) Drug was safe, well-tolerated and reduced plasma semicarbazide-sensitive amine oxidase activity
Company Product Description Indication Phase I status

Genitourinary/sexual function

Applied Genetic Technologies Corp., of Gainesville, Fla. AAV-CNGB3 gene therapy Gene therapy Achromatopsia Completed enrollment of adults in phase I/II trials; pediatric enrollment continuing; interim data of all adult dose groups expected in second half of 2020
Dare Bioscience Inc., of San Diego Sildenafil cream, 3.6% Phosphodiesterase type 5 inhibitor Healthy women (eventually female sexual arousal disorder) In first 6 subjects, there were statistically meaningful differences in genital temperature changes after treatment with active cream compared to placebo
Company Product Description Indication Phase I status


Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. BCX-9930 Factor D inhibitor Paroxysmal nocturnal hemoglobinuria First patients, naïve to eculizumab and ravulizumab, dosed in a proof-of-concept trial; company expects to report data in the second quarter
Company Product Description Indication Phase I status


Inflazome Ltd., of Dublin Inzomelid Small-molecule inhibitor of NLRP3 inflammasome Autoinflammatory disease Completed study, with results supporting progression into phase II
Provention Bio Inc., of Oldwick, N.J. PRV-3279 Humanized diabody targeting B-cell surface proteins CD32B and CD79B Systemic lupus erythematosus Top-line data from phase Ib portion in healthy volunteers showed drug was well-tolerated, with no serious adverse events, and pharmacokinetic parameters were generally dose-proportional; high levels of B-cell engagement resulted in durable pharmacodynamic responses; anti-drug antibody production was observed at both dose levels tested, but immunogenicity was found not to affect exposure, safety or pharmacodynamic parameters
Puretech Health plc, of Boston LYT-100 Deuterated pirfenidone Healthy volunteers (eventually lymphedema) First of 100 volunteers dosed in the multiple ascending-dose and food effect study designed to test the safety, tolerability and pharmacokinetic profile of the drug; data expected later in 2020
Company Product Description Indication Phase I status


Altasciences Co. Inc., of Laval, Quebec, and Roivant Sciences Ltd., of New York Gimsilumab Fully human monoclonal antibody targeting GM-CSF SARS-CoV-2 patients at risk of developing acute respiratory distress syndrome Completed study; Roivant said it will prioritize trials in patients with COVID-19
Arbutus Biopharma Corp., of Warminster, Pa. AB-729 RNA interference therapeutic targeted to hepatocytes using covalently conjugated N-acetylgalactosamine delivery technology Hepatitis B Dosing at either 60 mg or 180 mg was generally safe and well-tolerated and resulted in meaningful reductions in HBsAg levels
Artugen Therapeutics Ltd., of Concord, Mass., and Dublin ART-24 Direct-acting live biotherapeutic Prevention of recurrence of Clostridium difficile infection First patient dosed testing safety, tolerability and pharmacokinetics in patients recently cured of CDI
Gilead Sciences Inc., of Foster City, Calif. Vesatolimod Toll-like receptor 7 agonist HIV infection Phase Ib data showed TLR7 stimulation by study drug was associated with modestly increased time to viral rebound vs. placebo with median time to viral rebound (>50 copies/mL) of 4.1 weeks for vesatolimod vs. 3.9 weeks for placebo (p=0.036); for rebound to >200 copies/mL, median time was 5 weeks vs. 4 weeks, respectively (p=0.024); 4 in vesatolimod group had no virologic rebound (>50 c/mL) for 6 or more weeks; enhanced immune function and decreased levels of intact HIV DNA also seen with study drug
Gilead Sciences Inc., of Foster City, Calif. GS-6207 Disrupts HIV capsid HIV-1 Phase Ib results testing subcutaneous formulation showed antiviral activity through last day of monotherapy, day 10, with significantly greater reductions in HIV-1 RNA vs. placebo across all treatment groups (20 to 750 mg; all p<0.0001)
Inflarx NV, of Jena, Germany IFX-1 Monoclonal anti-C5a antibody Severe COVID-19-induced pneumonia Enrolled first patient into randomized trial
Kiniksa Pharmaceuticals Inc., of Hamilton, Bermuda Mavrilimumab Fully human monoclonal antibody targeting GM-CSFR? COVID-19 Pilot study of 6 patients with severe COVID-19 pneumonia and hyperinflammation showed all had resolved fever, without progressing to mechanical ventilation; follow-on controlled study in Italy planned; company engaging with FDA regarding path for potential phase II/III development in COVID-19 pneumonia
Lattice Biologics Ltd., of Belgrade, Mont. Amnioboost Amniotic fluid-derived stem cell therapy Severe acute respiratory syndrome First of 10 participants with laboratory confirmed COVID-19 infection and evidence of lung involvement requiring supplemental oxygen or mechanical ventilation enrolled in placebo-controlled study in Seattle; therapy involves ~ 5M Amnioboost stem cells on day 1 of enrollment and another ~ 5 million on day 2; daily scores based on scale of clinical outcomes for treatment and placebo groups will be assessed on day 7 and study halted if no evidence of treatment difference
Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-2203 (oral amphotericin B) Fungicidal Cryptococcal meningitis Uganda National Drug Authority informed all affected sponsors and clinical investigators that recruitment of new clinical trial participants in the county would be immediately suspended as a result of COVID-19; as a result, enrollment of new patients in phase I/II Enact trial has been temporarily paused
Moderna Inc., of Cambridge, Mass. mRNA-1273 mRNA vaccine SARS-CoV-2 Dosed first participant in study being conducted by NIH
Partner Therapeutics Inc., of Lexington, Mass. Leukine (sargramostim) CSF-1 agonist; GM-CSF receptor agonist COVID-19 Sarpac trial initiated at University Hospital Ghent to assess efficacy of approved drug in nebulized form to treat respiratory illness associated with COVID-19, measured by lung function and patient outcomes
Pluristem Therapeutics Inc., of Haifa, Israel PLX cells Allogeneic mesenchymal-like cells COVID-19 Dosed 3 more patients in 2 different hospitals in Israel under a compassionate use program
Recce Pharmaceuticals Ltd., of Sydney RECCE-327 Bactericidal permeability/outer membrane protein inhibitor Bacterial infection Execution of agreement for independent intravascular safety assessment at Australian-based trial facility expected to be finalized in first quarter of 2020; discussions underway with Australian teaching hospital to initiate independent phase I/II topical efficacy study in bacterial wound, burn and skin infections
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole inhalation powder Antifungal Invasive pulmonary aspergillosis  Completed all 4 dose-escalation cohorts of the single ascending-dose phase of the trial
Company Product Description Indication Phase I status


Arch Biopartners Inc., of Toronto Metablok LSALT peptide Organ damage caused by inflammation Cohort of eight healthy volunteers added
Arch Biopartners Inc., of Toronto Metablok LSALT peptide Prevention of organ inflammation Completed dosing of final 8 healthy volunteers, which received highest daily dose tested for 3 consecutive days; met primary endpoints of safety and tolerability
Evelo Biosciences Inc., of Cambridge, Mass. EDP-1815 Anti-inflammatory agent Psoriasis Showed significant activity on multiple individual systemic markers of inflammation
Company Product Description Indication Phase I status


Discgenics Inc., of Salt Lake City IDCT Allogeneic, injectable discogenic cell therapy Degenerative disc disease Enrollment completed
Dynacure SAS, of Strasbourg, France DYN-101 Antisense medicine designed to modulate expression of dynamin 2 Myotubular and centronuclear myopathies  First patient dosed
Fulcrum Therapeutics Inc., of Cambridge, Mass. Losmapimod Selective p38?/? mitogen activated protein kinase inhibitor Facioscapulohumeral muscular dystrophy Complete results showed dose-dependent and sustained target engagement in blood; evidence of target engagement observed in skeletal muscle biopsies; data support selection of 15-mg dose twice daily in ongoing phase IIb trial and phase II open-label study
Novartis AG, of Basel, Switzerland AVXS-101 Gene therapy Spinal muscular atrophy type 2 Met the primary efficacy endpoint, achieving a mean increase from baseline of 6 points in the Hammersmith Functional Motor Scale-Expanded 
Company Product Description Indication Phase I status


Auris Medical Holding Ltd., of Hamilton, Bermuda AM-201 Intranasal betahistine Prevention of antipsychotic-induced weight gain and somnolence Last patient had last treatment visit
Eton Pharmaceuticals Inc., of Deer Park, Ill. ET-101 (topiramate oral solution) GABA receptor agonist; NMDA receptor antagonist; sodium channel inhibitor Partial-onset/primary generalized tonic-clonic seizures Bioequivalence study in healthy males under fasting conditions showed 90% confidence intervals for pharmacokinetic parameters were within 80% to 125% of criteria vs. FDA-approved oral solid comparator product; food effect study conducted under fed conditions showed bioavailability unaffected
FSD Pharma Inc., of Toronto Ultra-micronized-PEA Fatty acid amide palmitoylethanolamide Healthy volunteers (eventually pain) Started study testing the safety, tolerability and pharmacokinetics of single and multiple ascending doses of the drug
Genetx Biotherapeutics LLC, of Sarasota, Fla., and Ultragenyx Pharmaceutical Inc., of Novato, Calif. GTX-102 Antisense oligonucleotide Angelman syndrome Dosed the first patient in open-label phase I/II KIK-AS trial
Mind Medicine Inc., of New York 18-MC (18-methoxycoronaridine) Alpha-3-beta-4 nicotinic receptor antagonist Opioid use disorder Enrollment initiated in additional study in adult volunteers
Prevail Therapeutics Inc., of New York PR-001 Gene therapy expressing GBA1 Parkinson’s disease with GBA1 mutations Enrollment continues in the Propel study putting the company on track to report interim data in the second half of 2020
Seelos Therapeutics Inc., of New York Intranasal ketamine (SLS-002)
NMDA receptor antagonist
Suicidal ideation Additional interim data showed all vital signs-related adverse events (AEs) seen in combined studies were mild and transient in nature; no serious AEs related to vital signs were observed
Uniqure NV, of Amsterdam, the Netherlands AMT-130 AAV-based gene therapy Huntington’s disease First 2 patients in phase I/II trial enrolled, but due to the expanding impact of the COVID-19 pandemic, their procedures have been temporarily postponed
Company Product Description Indication Phase I status


Allergan plc, of Dublin, and Editas Medicine Inc., of Cambridge, Mass. AGN-151587 (EDIT-101) CRISPR medicine delivered by subretinal injection Leber congenital amaurosis 10  First patient dosed
Proqr Therapeutics NV, of Leiden, the Netherlands QR-504a TCF4 gene modulator Corneal dystrophy Trial start delayed due to COVID-19 pandemic
Company Product Description Indication Phase I status


Decibel Therapeutics Inc., of Boston DB-020 Sodium thiosulfate Cisplatin-induced hearing loss Phase Ia results demonstrating safety and tolerability in healthy volunteers published in Investigational New Drugs; administration resulted in only nominal systemic increases of sodium thiosulfate, suggesting that DB-020 should have no impact on the efficacy of cisplatin; phase Ib trial in patients initiated
Proqr Therapeutics NV, of Leiden, the Netherlands QR-421a USH2A gene modulator Usher syndrome Interim analysis of phase I/II Stellar trial that enrolled 14 adults (8 to single dose of study drug and 6 to sham) showed sham treatment produced no consistent pattern of response above noise level; 1 of 4 in low-dose (50 µg) group classified as responder with onset of action observed by 3 month visit and benefit maintained for 6 months or >; 1 of 4 in mid-dose (100 µg) group classified as responder with onset of action observed by 3 months; 100-µg cohort will be expanded to additional participants homozygous for exon 13 mutations, with dose escalation to 200 µg planned in parallel, but enrollment paused due to COVID-19 pandemic
Translate Bio Inc., of Lexington, Mass. MRT-5005 Inhaled mRNA therapeutic Cystic fibrosis Company anticipates related interruptions in enrollment, dosing and follow-up due to COVID-19
Company Product Description Indication Phase I status


Enlivex Therapeutics Ltd., of Ness Ziona, Israel Allocetra Immunotherapy Severe sepsis Acute Physiology and Chronic Health Evaluation score of Allocetra-treated group was 12.3, and corresponding probability of mortality of at least 1 patient in that group was predicted at 85% based on hospital’s ICU staff’s clinical assessment of each patient’s overall condition at admission; no Allocetra-treated patients died during the 28-day study period, as compared to 27% 28-day mortality in the matched controls group

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