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Phase I clinical data reported in December 2019

Company Product Description Indication Phase I status


Alligator Bioscience AB, of Lund, Sweden ATOR-1017 Activates 4-1BB receptors Metastasized cancer First of approximately 50 patients dosed in study designed to test the safety and tolerability of the drug and to determine the recommended phase II dose
Aprea Therapeutics Inc., of Boston APR-246 Small molecule designed to reactivate mutant and inactivated p53 protein TP53-mutated myelodysplastic syndromes and acute myeloid leukemia In the French trial, updated data for 24 evaluable patients enrolled before June 2019 showed an overall response rate 71%, including complete remission for 54%; including 3 additional patients enrolled after the cutoff changed ORR to 74% and CR rate to 59%; median overall survival hasn’t been reached for the 53 enrolled patients
Beigene Ltd., of Beijing Tislelizumab Monoclonal antibody targeting PD-1 Platinum-resistant ovarian cancer Treatment with tislelizumab plus sitravatinib produced an overall response rate of 23.5% in seven patients, all of which were partial responses; median progression-free survival was 18 weeks
BJ Bioscience Inc., of Hangzhou, China BJ-001 IL-15 fusion protein Cancer cells that overexpress integrins such as ?v?3, ?v?5, and ?v?6 First patient dosed
Cellectis SA, of Paris UCART-22 B-lymphocyte cell adhesion molecule modulator Relapsed/refractory B-cell acute lymphoblastic leukemia First participant dosed in dose-escalation Balli-01 study evaluating safety, expansion, persistence and clinical activity of allogeneic candidate
Cstone Pharmaceuticals Ltd., of Suzhou, China CS-3005 Adenosine A2a receptor antagonist Advanced solid tumors Received approval from the HREC in Australia and acknowledgement from Australia's TGA on the trial
Curis Inc., of Lexington, Mass. CA-4948 IRAK4 kinase inhibitor Relapsed or refractory non-Hodgkin's lymphoma, including patients with diffuse large B-cell lymphoma, Waldenström's macroglobulinemia and oncogenic MYD88 mutations 5 of the 6 patients evaluable for anticancer activity at the 2 highest dose levels of CA-4948 have experienced reduced tumor burden
Curis Inc., of Lexington, Mass. Fimepinostat Small-molecule dual inhibitor of PI3K/HDAC  Relapsed or refractory diffuse large B-cell lymphoma, including patients with double-hit/double-expressor lymphoma Study testing the drug with venetoclax (Venclexta, Abbvie Inc./Roche Holding AG) has enrolled 11 patients, 6 patients in the first cohort and 5 patients in the second cohort; favorable safety profile so far
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist Triple-negative breast cancer First participant with CCR5+ metastatic disease enrolled in phase Ib/II trial showed no detectable circulating or metastatic tumor cells in peripheral blood, and additional reductions in CCR5 expression were seen on cancer-associated cells at 8 weeks; additional patient with metastatic breast cancer enrolled under emergency use IND
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist Metastatic triple-negative breast cancer Further data from first patient in phase Ib/II trial continue to show no detectable circulating tumor cells (CTC) or putative metastatic tumor cells in peripheral blood and additional reductions in CCR5 expression on cancer-associated cells at 11 weeks of treatment; additional data in an emergency IND protocol involving 1 metastatic breast cancer patient demonstrated shrinkage of tumor (via MRI) after 3 weeks of treatment
Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J. Fulvestrant Estrogen receptor antagonist Hormone-related breast cancer Dosing begun in pilot study
Exicure Inc., of Skokie, Ill. AST-008 TLR-9 agonist Solid tumors Preliminary data from ongoing phase Ib/II trial showed study drug, alone and in combination with pembrolizumab (Keytruda, Merck & Co. Inc.), produced cytokine and chemokine expression and immune cell activation in patient blood; of 4 participants with Merkel cell carcinoma, 1 who previously progressed on anti-PD-1 therapy has confirmed stable disease with decreased target lesion diameters for > 12 weeks and 1 had target lesion complete response and confirmed overall partial response > 24 weeks; 9 participants had progressive disease, 2 had not been evaluated and 1 was not evaluable
Faron Pharmaceuticals Oy, of Turku, Finland Clevegen Cancer immunotherapy targeting Clever-1 positive tumor associated macrophages Metastatic or inoperable solid tumors Shown to down-regulate a range of major immuno-oncology checkpoints
Gritstone Oncology Inc., of Emeryville, Calif. GRANITE Personalized immunotherapy of 20 tumor-specific neoantigens Advanced cancer In the GO-004 study, CD8+ T cells against multiple neoantigens were observed via IFN-g ELISpot in 4 patients; the T cells were able to produce IL-2 and Granzyme B
Gritstone Oncology Inc., of Emeryville, Calif. SLATE Off-the-shelf immunotherapy of 20 tumor-specific neoantigens Advanced cancer In the GO-005 study, therapy was well tolerated with no dose-limiting toxicities observed in 3 patients across 2 dose levels
Heat Biologics Inc., of Durham, N.C. HS-130 Engineered to secrete OX40L-Fc fusion protein Advanced solid tumors First patient dosed
Helix Biopharma Corp., of Richmond Hill, Ontario L-DOS47 Urease Previously treated patients with advanced pancreatic cancer Started enrollment in the study testing 3 doses of the drug plus doxorubicin in 9 total patients followed by a phase II study of 11 additional patients
Hookipa Pharma Inc., of New York HB-201 Human papillomavirus E6/E7 protein inhibitor HPV 16-positive cancer First of 100 participants with treatment-refractory disease dosed in open-label phase I/II trial evaluating safety, tolerability and preliminary efficacy based on antitumor activity defined by RECIST 1.1 and immunogenicity, both as monotherapy and in combination with immune checkpoint inhibitor
Immix Biopharma Inc., of Los Angeles Imx-110 NF-kB/Stat3/pan-kinase inhibitor curcumin plus doxorubicin encased in a nano-sized delivery system Advanced solid tumors Synergy Hematology Oncology institutional review board approved dosing patients
Immunocore Ltd., of Oxfordshire, U.K. IMC-C103C T-cell redirecting bispecific biologic MAGE-A4-expressing cancers First patient dosed
Immutep Ltd., of Sydney Eftilagimod alpha (IMP-321) HLA class II antigen stimulator Advanced solid malignancies No dose limiting toxicities seen in the first 6 patients treated with 6 mg eftilagimod plus Bavencio (avelumab, Merck KGaA and Pfizer Inc.); started recruitment of second cohort of 6 patients to be treated with 30 mg eftilagimod plus Bavencio
Inmune Bio Inc., of La Jolla, Calif. INB-03 TNF inhibitor Advanced solid tumors Drug was well tolerated; IL-6 decreased by more than 50% in half of the patients; selected 1 mg/kg as dose for phase II testing; next study in trastuzumab resistant, HER2+ breast cancer
Innovent Biologics Inc., of Suzhou, China IBI-110 Anti-lymphocyte activation gene 3 recombinant fully human monoclonal antibody Advanced malignancies First patient dosed
Lixte Biotechnology Holdings Inc., of East Setauket, N.Y. LB-100 Protein phosphatase 2A inhibitor Glioblastoma NCI enrolled first 2 of 8 planned participants in study assessing ability of LB-100 to enter brain and penetrate recurrent tumors when surgical removal of cancers is indicated
Mateon Therapeutics Inc., of Agoura Hills, Calif. OXi-4503 Cis-combretastatin A1 dipotassium diphosphate Acute myeloid leukemia Study in 26 evaluable patients with previously treated relapsed AML met primary endpoint; adding Combretastatin A1 to standard chemotherapy cytarabine was generally well-tolerated and maximum tolerated dose was identified as recommended dose for further clinical work
Moleculin Biotech Inc., of Houston Annamycin Lipophilic anthracycline derivative Acute myeloid leukemia Of 10 participants treated and evaluated at or above 120 mg/m2, interim results showed 1 complete response with incomplete recovery of white blood cells and/or platelets and 2 partial responses, where bone marrow blasts were reduced 50% to below 25%; 1 additional participant was bridged to bone marrow transplant
Morphosys AG, of Planegg, Germany Tafasitamab (MOR-208) B-lymphocyte antigen CD19 modulator Diffuse large B-cell lymphoma First participant dosed in phase Ib study evaluating safety and preliminary efficacy of drug in addition to rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone (R-CHOP) and in combination with lenalidomide in addition to R-CHOP in adults with newly diagnosed disease
Nantkwest Inc., of Culver City, Calif.
PD-L1 t-haNK
Immunoglobulin gamma Fc receptor III modulator; PD-L1 modulator Solid tumors First 6 participants with locally advanced or metastatic disease enrolled in part 1 of Quilt-3.064 trial were infused in outpatient setting without reported cytokine toxicities or immune-related adverse events
Neuclone Pharmaceuticals Ltd., of Sydney Neuceptin Biosimilar candidate of Herceptin (trastuzumab, Roche Holding AG) Breast cancer Met all prespecified criteria demonstrating clinical pharmacokinetic similarity to Herceptin
Nordic Nanovector ASA, of Oslo, Norway Betalutin CD37-targeting radioimmunotherapy Relapsed/refractory diffuse large B-cell lymphoma No safety issues in the 3 completed cohorts; a reversible dose limiting toxicity experienced in 1 patient in the final dosing cohort; evidence of disease control in some patients; data to be presented in the first half of 2020
Nouscom GmbH, of Basel, Switzerland NOUS-209 Off-the-shelf therapeutic vaccine based on shared tumor neoantigens Microsatellite instable high gastric, colorectal and gastroesophageal junction cancers  First patient dosed
Noxopharm Ltd., of Sydney Veyonda (NOX-66) Suppository of ecto-NOX disulfide-thiol exchanger type 2 inhibitor idronoxil Late-stage prostate cancer In 15 evaluable patients, Veyonda plus low-dose palliative radiotherapy produced a tumor response rate of 66%, including 1 partial response and 9 patients with stable disease; 5/16 patients had a >50% reduction in PSA; 10/16 patients had a >30% reduction in pain response; company plans to run a phase II/III study
Noxxon Pharma NV, of Berlin NOX-A12 Targets C-X-C chemokine ligand 12 Brain cancer Data safety monitoring board reviewed 10-week data from first patient treated with NOX-A12 plus radiotherapy and recommended enrolling the rest of the cohort
Oncternal Therapeutics Inc., of San Diego TK-216 ETS transcription factor inhibitor Ewing sarcoma Opened phase Ib expansion cohort of ongoing trial to evaluate recommended phase II dose regimen (200 mg/m2/day for 14 days) in combination with vincristine in relapsed/refractory disease
Pioneer Project, of Marseille, France Monalizumab, MEDI-9447, AZD-6738 and AZD-9150 NKG2A inhibitor, CD73 inhibitor, ATR inhibitor and Stat3 inhibitor PD1(L1) inhibitor-resistant advanced non-small-cell lung cancer First patient treated in study testing each drug in combination with Imfinz (durvalumab, Astrazeneca plc); primary endpoint is the percentage of patients that achieve complete response, partial response or maintain a stable disease at 12 weeks
Qbiotics Group Ltd., of Brisbane, Australia Tigilanol tiglate Protein kinase C stimulator Head and neck squamous cell carcinoma First participant dosed in phase I/II trial evaluating optimal dosing and safety
Sunesis Pharmaceuticals Inc., of South San Francisco Vecabrutinib BTK inhibitor Relapsed/refractory chronic lymphocytic leukemia and other B-cell malignancies Clinical benefit observed in covalent BTK inhibitor-resistant patients with poor prognostic features and in both wild-type and C481-mutated BTK disease; in the 300-mg dose group (cohort 5), stable disease was observed in 3 of 5 patients, and 2 remain on treatment in cycle 5 and cycle 6, including a wild-type BTK patient with -40% change in tumor burden at first scan
Vedanta Biosciences Inc., of Cambridge, Mass. VE-800 Rationally defined bacterial consortium of 11 commensal strains that activate cytotoxic CD8+ T cells Selected types of advanced or metastatic cancer Study begun in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.)
X4 Pharmaceuticals Inc., of Cambridge, Mass. Mavorixafor (X4P-001) CXCR4 chemokine antagonist Waldenström’s macroglobulinemia Initiated phase Ib trial, in combination with ibrutinib (Imbruvica, Pharmacyclics LLC/Janssen Biotech Inc.), expected to enroll 12 to 18 participants with acquired “gain of function” mutation in CXCR4 in addition to MYD88 mutation; trial also designed to measure changes from baseline in biomarkers serum immunoglobulin M and hemoglobin
Y-Mabs Therapeutics Inc., of New York Naxitamab Anti-GD2 3F8 monoclonal antibody Neuroblastoma and other solid tumors Investigator-sponsored front-line study of 34 participants with high-risk stage 4 neuroblastoma in first complete remission showed 72% event-free survival at 24 months compared to 63% reported at 24 months for population of 89 individuals treated with competing antibody; 20% of those treated with study drug received bone marrow transplant compared with all treated with competing anti-GD2 antibody; overall survival for study drug was 86% at 24 months compared to 84% for competing antibody
Zenith Epigenetics Ltd., of Calgary, Alberta ZEN-3694 BET inhibitor Metastatic castration-resistant prostate cancer Completed the phase Ib/II, ZEN-3694 plus enzalutamide combination trial in patients who had progressed on an androgen receptor (AR) antagonist; data show it provided a significant and meaningful radiographic progression-free survival benefit of 44 weeks vs. 24 weeks for patients receiving only the AR antagonist
Zenith Epigenetics Ltd., of Calgary, Alberta ZEN-3694 BET inhibitor Triple-negative breast cancer Due to clinical activity in some patients dosed to date, Zenith is exploring the potential expansion to include additional tumor types that may respond to ZEN-3694 plus Pfizer Inc.'s talazoparib, a poly ADP ribose polymerase inhibitor
Zentalis Pharmaceuticals Inc., of New York ZN-c3 Small molecule DNA damage response candidate targeting protein tyrosine kinase WEE1 Solid tumor Dosed the first patient in a phase I/II trial evaluating ZN-c3 in patients with advanced solid tumors
Zhejiang Medicine Co. Ltd., of Shaoxing, China, and Ambrx Inc., of San Diego ARX-788 Antibody-drug conjugate targeting HER2 Pretreated metastatic HER2 positive breast cancer Overall response rates of 14%, 36%, 56% and 63% for doses of 0.88 mg/kg, 1.1 mg/kg, 1.3 mg/kg and 1.5 mg/kg, respectively
Company Product Description Indication Phase I status


Kancera AB, of Stockholm KAND-567 Fractalkine ligand inhibitor Acute myocardial infarction Immunological analysis of blood samples from healthy participants in ongoing phase Ib program showed study drug blocked immune cells known to cause acute and chronic inflammatory diseases; effect on clinically relevant biomarkers provides additional support for potential cardiovascular protective effect
Company Product Description Indication Phase I status


Aslan Pharmaceuticals Pte. Ltd., of Singapore ASLAN-004 Monoclonal antibody targeting IL-13 receptor alpha1 subunit Moderate to severe atopic dermatitis After at least 1 month of treatment at the lowest dose of 200 mg, 3 patients had reductions of 85%, 70% and 59% in their Eczema Area and Severity Index scores from baseline; data monitoring committee will review data in late December before opening second dosing cohort; study designed to test doses up to 600 mg followed by an expansion cohort of 12 patients on drug and 6 on placebo at the most efficacious dose
Biomx Inc., of Ness Ziona, Israel BX-001 Naturally occurring phage targeting Cutibacterium acnes Acne vulgaris Completed enrollment of 75 patients in study designed to measure the safety and tolerability of the drug; C. acnes levels will be measured as an exploratory endpoint; data expected in the first quarter of 2020
Topas Therapeutics GmbH, of Hamburg, Germany TPM-203 Small peptide-loaded nanoparticles Pemphigus vulgaris First of 24 patients enrolled in the study testing single-ascending doses, followed by multiple doses
Company Product Description Indication Phase I status


Arecor Ltd., of Cambridge, U.K. AT-247 Mealtime insulin formulation Post-prandial glucose control Provided a favorable pharmacokinetic/pharmacodynamic profile compared with Novorapid (insulin, Novo Nordisk A/S) and Fiasp (insulin, Novo Nordisk A/S)
Axovant Gene Therapies Ltd., of New York AXO-AAV-GM1 Gene therapy expressing GLB1 GM1 gangliosidosis First patient in the expanded access had clinically significant improvements based on neurological exam, the Vineland-3 scale, Clinical Global Impression assessments and nutritional status 6 months after treatment
Homology Medicines Inc., of Bedford, Mass. HMI-102 Gene therapy expressing PAH Phenylketonuria First patient in cohort 2 (mid-dose) had reductions in Phe of 35% and 48% and increases in Tyr levels of 72% and 85% from baseline to week 1 and week 4, respectively
Lannett Co. Inc., of Philadelphia Insulin glargine biosimilar Insulin ligand; insulin receptor agonist Type 1 and 2 diabetes In 27 healthy volunteers, single subcutaneous dose matched reference biologic Lantus (Sanofi SA), meeting primary endpoints of pharmacokinetics and pharmacodynamics safety
Neurovive Pharmaceutical AB, of Lund, Sweden KL-1333 Modulates cellular levels of NAD+ Healthy volunteers (eventually primary mitochondrial disease) Completed recruitment of 5 cohorts of healthy volunteers who received multiple ascending doses
Regenxbio Inc., of Rockville, Md. RGX-121 Gene therapy expressing iduronate-2-sulfatase Mucopolysaccharidosis Type II In 3 patients in cohort 1, heparan sulfate in the cerebral spinal fluid was reduced by a mean of 33.3% from baseline to week 8; first patient treated in cohort 2
Rezolute Inc., of Redwood City, Calif. AB-101 Ultra-long acting basal insulin Type 1 and type 2 diabetes AB101 was administered subcutaneously in 3 ascending dosing cohorts, which resulted in slow onset and sustained insulin levels or more than 7 days; at the higher doses, the drug volume was greater than anticipated, as was variability in onset time between patients
Rezolute Inc., of Redwood City, Calif. AB-101 Ultra-long basal insulin Type I and II diabetes Top-line phase I data show a slow onset and sustained insulin levels and activity for more than 7 days when administered subcutaneously in three ascending dosing cohorts; at higher doses, the necessary drug volume was greater than anticipated; company intends to conduct additional formulation development before advancing
Synlogic Inc., of Cambridge, Mass. SYNB-1618 Synthetic biotic Phenylketonuria Data from bridging study in healthy volunteers identified maximum tolerated dose of 2 x 1012 live cells (5.3 x 1011 colony forming units) and showed that dose ramp improved tolerability and that pH buffering was required for maximum Phe-consuming activity of the strain
Company Product Description Indication Phase I status


Hepion Pharmaceuticals Inc., of Edison, N.J. CRV-431 Hepatitis B structural protein inhibitor; peptidyl-prolyl cis-trans isomerase A inhibitor Nonalcoholic steatohepatitis Multiple ascending-dose trial escalated to next dosing level of 150 mg daily for 28 days
Innovation Pharmaceuticals Inc., of Beverly, Mass. Brilacidin Defensin peptidomimetic Ulcerative colitis Screening for trial of delayed-release tablets in healthy volunteers expected to begin in early January following approval from U.K.'s MHRA; study to assess safety, tolerability and pharmacokinetics of oral drug and evaluate delivery directly to colon
Kaleido Biosciences Inc., of Lexington, Mass. KB-174 Mixture of oligosaccharides Well-compensated cirrhosis In a 15N-labeled tracer test, treatment with drug for 28 days reduced urinary 15N excretion by a median of 26%, compared to a 3% median reduction for patients taking the negative control maltodextrin; plans to start a study in patients with hepatic encephalopathy in the second half of 2020
Meiji Seika Pharma Co. Ltd., of Tokyo DMB-3115 Biosimilar ustekinumab (Stelara, Johnson & Johnson), a recombinant immunoglobulin G1kappa monoclonal antibody targeting IL-12 and IL-23 Crohn's disease Trial begun
Poxel SA, of Lyon, France PXL-065 Deuterium-stabilized R-pioglitazone Healthy volunteers (eventually non-alcoholic steatohepatitis) Pharmacokinetic assessment showed plasma exposure (Cmax and area under the curve) increased in a dose-proportional manner up to the maximum dose tested; dossing fed vs. fasted didn't produce a clinically meaningful difference; plans to start a 120-patient phase II study in the second quarter of 2020
Ose Immunotherapeutics SA, of Nantes, France OSE-127 Monoclonal antibody targeting CD127 receptor Ulcerative colitis and Sjogren’s syndrome Results in healthy volunteers showed good safety and tolerability profile; pharmacokinetic and pharmacodynamic parameters consistent and demonstrate dose-proportionality across several dose levels up to 10 mg/kg
Company Product Description Indication Phase I status

Genitourinary/sexual function

Arch Biopartners Inc., of Toronto Metablock LSALT peptide Acute kidney injury in patients undergoing cardiac surgery Phase I completed dosing of all scheduled volunteers; met primary endpoints of safety and tolerability; no drug-related adverse events observed in 44 people
Company Product Description Indication Phase I status


Neuclone Pharmaceuticals Ltd., of Sydney Neulara Biosimilar of Stelara (ustekinumab) Healthy volunteers (eventually plaque psoriasis, psoriatic arthritis, Crohn disease and ulcerative colitis) Completed dosing of subjects in study comparing a single dose of Neulara to U.S. and EU sourced Stelara; data expected in the third quarter of 2020
Syndax Pharmaceuticals Inc., of Waltham, Mass. SNDX-6352 Anti-CSF-1R monoclonal antibody Chronic graft-vs.-host disease Both evaluable patients treated at the highest dose of 1 mg/kg had a partial response as did the 1 patient treated at 0.5 mg/kg; cohort testing 3 mg/kg is open; plans to advance into a phase II expansion cohort at 1 mg/kg in first quarter of 2020
Company Product Description Indication Phase I status


Cullinan Oncology LLC, of Cambridge, Mass. VK-2019 First-in-class EBNA1 inhibitor Epstein Barr Virus positive (EBV+) cancers Dosing initiated; in preclinical models of EBV-associated cancers, VK-2019 eliminated EBV, resulting in tumor growth inhibition
Cullinan Oncology LLC, of Cambridge, Mass. CLN-081 Epidermal growth factor tyrosine kinase inhibitor Non-small cell lung cancer Dosing initiated; it was engineered to inhibit EGFR variants with exon 20 insertion mutations, while sparing wild-type EGFR. CLN-081 is initially in development to target NSCLC driven by EGFR exon 20 insertion mutations
Ico Therapeutics Inc., of Vancouver, British Columbia Oral amphotericin B Antifungal Vulvovaginal candidiasis First 2 cohorts received the first dose
Ico Therapeutics Inc., of Vancouver, British Columbia Amphotericin B Lipophilic molecule Vulvovaginal candidiasis All subjects in first of 2 cohorts in phase Ib study have received 100-mg doses over 10 days, with no drug-related adverse events observed to date; 1 additional cohort is expected to be dosed in early Q1 2020, followed by initiation of a phase II study comparing oral Amphotericin B to fluconazole in a head-to-head study
Company Product Description Indication Phase I status


Atyr Pharma Inc., of San Diego ATYR-1923 Targets neuropilin-2 receptor Pulmonary sarcoidosis ATYR-1923 was safe and well tolerated in 15 patients who have received at least one dose of the drug
Company Product Description Indication Phase I status


Wave Life Sciences Ltd., of Cambridge, Mass. Suvodirsen Stereopure oligonucleotide Duchenne muscular dystrophy Interim analysis showed no change from baseline in dystrophin expression; development discontinued
Company Product Description Indication Phase I status


Alzecure Pharma AB, of Stockholm ACD-856 Stimulates neurotrophic signaling Healthy volunteers (eventually Alzheimer's disease, sleep disorders and traumatic brain injuries) Started study testing the half-life of the drug; data expected in the first half of 2020
Athira Pharma Inc., of Seattle NDX-1017 Small-molecule therapeutic designed to enhance activity of hepatocyte growth factor and its receptor, MET Alzheimer's disease NDX-1017 showed dose-dependent and consistent changes in brain activity across all treated cohorts
Blackthorn Therapeutics Inc., of San Francisco BTRX-335140 Kappa opioid receptor antagonist Depression and other central nervous system disorders Shown to engage receptors in the brain at levels that are projected to be effective in treating relevant symptoms
Cerevel Therapeutics Inc., of Boston CVL-865 Alpha 2/3/5-subtype GABAA positive allosteric modulator Epilepsy Observed to be rapidly absorbed and well-tolerated in healthy volunteers
Chondrial Therapeutics Inc., of Bala Cynwyd, Pa. CTI-1601 Frataxin fusion protein prodrug Friedreich’s ataxia Dosed first patient in the study testing the safety, tolerability and pharmacokinetics of single ascending doses; top-line data expected by the end of 2020
Chondrial Therapeutics, of Bala Cynwyd, Pa. CTI-1601 Recombinant fusion protein intended to deliver human frataxin Friedreich's ataxia First patients have been dosed in a phase I double-blind, placebo-controlled trial; FDA granted rare pediatric disease designation and fast-track designation
Cortexyme Inc., of South San Francisco COR-388 Small-molecule gingipain inhibitor Alzheimer's disease After 28 days, a statistically significant decrease in ApoE fragments (both ApoE4 and ApoE3) was observed in subjects treated with drug vs. those treated with placebo
Eicosis LLC, of Davis, Calif. EC-5026 Inhibits she Pain management Dosed first patient
Eleusis Ltd., of London Lysergic acid diethylamide Psychoactive drug Healthy older volunteers (eventually Alzheimer’s disease) Data published in Psychopharmacology showed low doses of the drug produced no deviations from baseline or abnormalities on safety or cognitive outcome measures
Inmune Bio Inc., of La Jolla, Calif. Xpro-1595 Targets soluble tumor necrosis factor Alzheimer's disease Treated first of up to 18 patients who express biomarkers of inflammation; 12-week study will measure safety and reduction of inflammation
Lexicon Pharmaceuticals Inc., The Woodlands, Texas LX-9211 Inhibitor of adaptor associated kinase 1 Neuropathic pain Demonstrated a favorable safety and pharmacokinetics (PK) profile supportive of once-daily dosing and exhibited dose proportional PK
Modag GmbH, of Wendelsheim, Germany Anle-138b Binds oligomeric structures of alpha-synuclein Healthy volunteers (eventually Parkinsonian disorders) Started study testing the safety, tolerability and pharmacokinetic profile of single and multiple ascending doses of the drug
Sage Therapeutics Inc., of Cambridge, Mass. SAGE-718 Oxysterol-based positive allosteric modulator of N-methyl-D-aspartate receptor Huntington’s disease Drug was well-tolerated in patients and healthy volunteers; in patients, drug improved performance compared to baseline on assessments of executive functioning; patient data to be presented in 2020; plans to move drug into phase II development
Samus Therapeutics Inc., of Boston PU-AD Epichaperome inhibitor Alzheimer's disease PU-AD was well-tolerated after single oral doses, with any adverse events being mild and self-limited, and demonstrated pharmacokinetic linearity
Solid Biosciences Inc., of Cambridge, Mass. SGT-001 Adeno-associated viral vector-mediated gene transfer Duchenne muscular dystrophy Data from 2 patients dosed in the second cohort of IGNITE DMD show SGT-001 microdystrophin expression and associated neuronal nitric oxide synthase function, providing evidence that SGT-001 has the potential to provide therapeutic benefit; also, a previously reported SAE experienced by a third patient in the 2E14 vg/kg dose group has fully resolved; Solid received a clinical hold letter from the FDA and will continue working to address the hold and determine a path forward
Tonix Pharmaceuticals Holding Corp., of New York TNX-601 CR Controlled release formulation of tianeptine, a modulator of the glutamatergic system Healthy volunteers (eventually major depressive disorder and posttraumatic stress disorder) Drug was well tolerated; plans to run an efficacy study outside the U.S. in 2020
Trevena Inc., of Chesterbrook, Pa. TRV-250 G protein-selective delta-receptor agonist Acute migraine Drug was well-tolerated with a pharmacokinetics profile appropriate for an acute migraine therapy
Vaccinex Inc., of Rochester, N.Y. Pepinemab Humanized monoclonal antibody that binds and blocks the signaling activity of semaphorin 4D  Neurodegenerative disease Presented evidence suggesting the drug prevents transformation of brain glial cells from their normal supportive activities to inflammation
Wave Life Sciences Ltd., of Cambridge, Mass. WVE-120102 HTT gene inhibitor Huntington’s disease Top-line data from ongoing phase Ib/IIa Precision-HD2 trial showed statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein in cerebrospinal fluid, with analysis to assess dose response across treatment groups (2, 4, 8 or 16 mg) suggesting statistically significant response in mHTT reduction at highest doses tested (p=0.03); however, no difference seen in total HTT compared to placebo
Xenon Pharmaceuticals Inc. of Burnaby, British Columbia, and Neurocrine Biosciences Inc., of San Diego XEN-901 Nav1.6 sodium channel inhibitor SCN8A-related epilepsy Study in healthy adults complete; developing a pediatric-specific, granule formulation with an IND expected in the middle of 2020
Company Product Description Indication Phase I status


Lineage Cell Therapeutics Inc. (formerly Biotime Inc.), of Carlsbad, Calif. Opregen Human embryonic stem cell-derived retinal pigmented epithelial cell therapy Dry age-related macular degeneration First cohort 4 participant treated with subretinal delivery system and thaw-and-inject formulation gained 25 readable letters (5 lines) 6 months following administration, as assessed by Early Treatment Diabetic Retinopathy Scale, representing improved visual acuity from baseline of 20/250 to 20/100 in treated eye
National Eye Institute, of Bethesda, Md. iPSC-derived RPE implant Retinal pigment epithelial cells from induced pluripotent stem cells Advanced-stage geographic atrophy Started 12-patient study testing the safety of the therapy
Proqr Therapeutics NV, of Leiden, the Netherlands QR-1123 RHO gene modulator Retinitis pigmentosa First of about 35 adults with autosomal dominant disease due to P23H mutation in rhodopsin gene dosed in phase I/II Aurora trial; improvement of visual function and retinal structure will be assessed through visual acuity, visual field and optical coherence tomography; initial data expected in 2021
Company Product Description Indication Phase I status


Spero Therapeutics Inc., of Cambridge, Mass. SPR-720 Oral antimicrobial Nontuberculous mycobacterial pulmonary disease Generally well-tolerated, with a pharmacokinetic profile that supports further development 
Company Product Description Indication Phase I status


Soligenix Inc., of Princeton, N.J. Rivax Ricin toxin A-chain subunit vaccine Healthy adults (ricin toxin exposure) Started trial
Trevena Inc., of Chesterbrook, Pa. TRV-734 Mu-opioid receptor selective agonist Opioid use disorder Initiated proof-of-concept study funded by National Institute on Drug Abuse; study will enroll about 50 opioid-dependent patients undergoing stable methadone maintenance therapy; primary objective is to assess ability of drug to reduce acute opioid craving symptoms, as measured by Subjective Opioid Withdrawal Scale

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