Company Product Description Indication Status
Phase I
Actinium Pharmaceuticals Inc., New York Actimab-A Antibody radionuclide conjugate targeting CD33 Relapsed or refractory acute myeloid leukemia Completed enrollment of the second dose cohort in the phase I portion of the phase I/II study testing Actimab-A plus Venclexta (venetoclax, Roche Holding AG and Abbvie Inc.); data expected in the second half of 2021
Albireo Pharma Inc., of Boston A-3907 Apical sodium-dependent bile acid transporter inhibitor Primary sclerosing cholangitis and primary biliary cholangitis Treated first healthy participant in a study testing the safety, tolerability, pharmacokinetics and pharmacodynamics of an A-3907; top-line data expected in the second half of 2021
Aquestive Therapeutics Inc., of Warren, N.J. AQST-108 (dipivefrin) Sublingual formulation of dipivefrin, a prodrug of epinephrine Anaphylaxis  In healthy adults, Tmax for AQST-108 was 10-40 minutes and the Cmax was 1,276 pg/mL; AQST-108 was rapidly converted to epinephrine with a Tmax range of 11-106 minutes and a Cmax of 205 pg/mL compared to Tmax range of 4-75 minutes and 8-68 minutes and Cmax of 475 pg/mL and 388 pg/mL for the intramuscular and subcutaneous injections of epinephrine, respectively
Cyclo Therapeutics Inc., of Gainesville, Fla. Trappsol Cyclo (hydroxypropyl beta cyclodextrin) Lipid metabolism modulator; cholesterol binding agent  Niemann-Pick disease type C1 Of the 9 patients who completed the study, 8 had an improvement of at least 1 point in 2 domains of the 17-domain NPC Severity Scale after 48 weeks of treatment; clinicians rated 7 of patients as improving and 2 as stable; 3 patients were withdrawn from the study
Diffusion Pharmaceuticals Inc., Charlottesville, Va. Trans sodium crocetinate Oxygen enhancing therapeutic Hypoxia Completed dosing of 30 healthy volunteers in the study using a transcutaneous oxygen monitoring device to evaluate the pharmacodynamic effects of trans sodium crocetinate on peripheral tissue oxygenation; data expected in the second quarter of 2021
Enochian Biosciences Inc., of Los Angeles Donated natural killer cells and dendritic cells loaded with a component of cytomegalovirus Cell therapy Recurrent glioblastoma Data published in the American Journal of Case Reports showed the treatment resulted in complete remission for 15 months
Immodulon Therapeutics Ltd., of Uxbridge, U.K. IMM-101 Heat-killed mycobacterium obuense Limited metastatic pancreatic cancer following FOLFIRINOX treatment Enrolled first of 100 patients in the investigator-sponsored Mepanc-1 study testing IMM-101 in combination with stereotactic radiotherapy; primary efficacy endpoint is 1-year progression-free survival
Kineta Inc., of Seattle LHF-535 Antiviral Lassa fever and other viral hemorrhagic fevers of arenavirus origin LHF-535 was well-tolerated in 24 healthy participants; no concerning adverse events or safety issues; pharmacokinetics consistent with preclinical models
KM Biologics Co. Ltd., of Kumamoto, Japan KD-382 Live attenuated tetravalent vaccine Dengue virus infection prophylaxis KD-382 produced 100% seroconversion at an early stage and elicited long-lasting and neutralizing antibody response for all 4 serotypes in most subjects over a 1-year follow-up, including patients who received a single dose
Medivir AB, of Stockholm MIV-818 (liver-targeted troxacitabine prodrug) DNA polymerase inhibitor Advanced liver cancer Last patient included in the first part of the study testing MIV-818 as a monotherapy; preparing to start part 2 of the study testing MIV-818 with other therapies during the second half of 2021
Synlogic Inc., of Cambridge, Mass. SYNB-8802  Synthetic biotic engineered to consume oxalate Enteric hyperoxaluria In healthy volunteers on a high oxalate and low calcium diet, SYNB-8802 lowered baseline urinary oxalate levels by 28.6% compared to placebo at the 3e11 dose
Vasomune Therapeutics Inc., of Toronto AV-001 Tie2 receptor agonist COVID-19 In 48 healthy volunteers, AV-001 was safe and well-tolerated and there were no discontinuations owing to study medication, serious adverse events, severe adverse events, adverse events of special interest, clinically significant abnormal laboratory values or abnormal ECGs
Phase II
Angion Biomedica Corp., of Uniondale, N.Y. ANG-3777 HGF receptor agonist COVID-19-related pneumonia ALI-201 study in Brazil fully enrolled to assess effectiveness in reducing severity of infection and preventing progression to acute lung injury, with primary endpoint of survival free from mechanical ventilation or dialysis at 28 days; top-line data expected by mid-2021
Bristol Myers Squibb Co., of New York Relatlimab Anti-LAG-3 antibody Melanoma Phase II/III Relativity-047 trial evaluating fixed-dose combination of relatlimab + Opdivo (nivolumab) vs. Opdivo alone in previously untreated metastatic/unresectable disease met primary endpoint of progression-free survival; follow-up ongoing for secondary endpoint of overall survival
Devonian Health Group Inc., of Quebec City Thykamine (PUR-0110) IL-1 beta ligand modulator; TNF alpha ligand modulator Atopic dermatitis IGA success rates at week 4 were 6.7% for placebo (vehicle cream) compared to 19% for Thykamine cream 0.05% (p=0.053 vs. placebo), 30.8% for 0.1% dose (p=0.014 vs. placebo) and 12.1% for 0.25% dose (p=0.461 vs. placebo); success rate over placebo for 0.1% dose, selected as phase III dose, also reached at week 3 (p=0.04)
Otonomy Inc., of San Diego OTO-313 NMDA receptor antagonist Tinnitus Initiated trial that's expected to enroll about 140 people with persistent, early onset, moderate or greater disease; primary efficacy endpoint is responder analysis based on proportion who report clinically meaningful improvement in Tinnitus Functional Index from baseline to both month 1 and 2 following treatment; top-line results expected in mid-2022
Visus Therapeutics Inc., of Seattle Brimochol (carbachol + brimonidine tartrate eye drops) Cholinergic agent + alpha-2 agonist Presbyopia Initiated study that's expected to enroll about 42 people; primary endpoint is percentage who gain 3 or more lines in near visual acuity without losing distance vision; top-line data expected in mid-2021
Phase III
Albireo Pharma Inc., of Boston Odevixibat  Ileal bile acid transport inhibitor Alagille syndrome Enrolled first patient in the Assert study; primary endpoint is change in Albireo Observer-Reported Outcome scratching score from baseline to month 6; top-line data expected in 2022
Astrazeneca plc, of Cambridge, U.K. AZD-1222 COVID-19 spike glycoprotein modulator COVID-19 infection Primary analysis showed 76% efficacy against symptomatic infection, 100% efficacy against severe or critical disease and hospitalization, and 85% efficacy against symptomatic infection in people 65 and older
Horizon Therapeutics plc, of Dublin Krystexxa (pegloticase injection)  Uricase stimulator Gout First of up to 30 people with uncontrolled gout enrolled in Forward trial assessing monthly dosing of 16 mg of study drug + methotrexate; primary outcome for 48-week trial is proportion of responders during month 6 of treatment, measured as serum uric acid (sUA) <6 mg/dl at least 80% of time, and time to and duration of sUA normalization
Marinomed Biotech AG, of Korneuburg, Austria Inhaleen (Carragelose-based inhalation solution) Antiviral;  sulfated polymer from red seaweed COVID-19 infection First of 204 participants hospitalized with infection dosed; primary endpoint is improvement in clinical status on day 8 vs. placebo


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