Company Product Description Indication Status
Phase I
Affibody AB, of Solna, Sweden ABY-039 FcRn inhibitor Myasthenia gravis Completed study in healthy volunteers; due to tolerability observations that would limit the target product profile of subcutaneous high-dose once-monthly maintenance injections, company terminated program
Applied Therapeutics Inc., of New York AT-007 CNS-penetrant aldose reductase inhibitor Galactosemia Additional biomarker and efficacy data from Action-Galactosemia phase I/II trial showed higher-dose 40-mg/kg cohort demonstrated plasma galactitol reduction of 55%, an incremental improvement in efficacy vs. the 20-mg/kg dose; reduction in galactitol was statistically significant vs. placebo (p<0.01); all patients on 40-mg/kg dose demonstrated significant reduction in galactitol from baseline, and reduction in galactitol was rapid and sustained over the 28-day dosing period
Applied Therapeutics Inc., of New York AT-007 CNS-penetrant aldose reductase inhibitor Galactosemia Initiated Action-Galactosemia Kids pediatric trial in children, ages 2-17
Cardiff Oncology Inc., of San Diego Onvansertib ATP competitive inhibitor of serine/threonine polo-like-kinase 1 enzyme Relapsed or refractory acute myeloid leukemia Phase Ib data showed anti-leukemic activity observed at wide range of doses (27-90 mg/m2); of 21 evaluable patients, 7 (33%) achieved objective response; 5 of 16 (31%) who achieved a complete response were treated at the 4 highest dose levels (27-90 mg/m2); 3 patients remain on treatment, with time since clinical response of 6, 12 and 15 months, respectively
Immunicum AB, of Stockholm Ilixadencel Cell-based off-the-shelf immune primer Solid tumors Sixth patient completed safety period in ongoing phase Ib/II Iliad combination trial with checkpoint inhibitor Keytruda (pembrolizumab, Merck & Co. Inc.); dose-escalation committee confirmed no dose-limiting toxicities and study can move into non-staggered inclusion phase
Sinovac Biotech Ltd., of Beijing Coronavac Vaccine  COVID-19 Preliminary phase I/II data in healthy volunteers showed no severe adverse events; vaccine induces neutralizing antibodies 14 days after vaccination, with a 0, 14-day schedule; neutralizing antibody seroconversion rate is above 90%, which concludes vaccine candidate can induce positive immune response
Phase II
Amylyx Inc., of Cambridge, Mass. AMX-0035 Targets mitochondrial and endoplasmic reticulum-dependent neuronal degeneration pathways Alzheimer’s disease Completed enrollment in Pegasus trial, dosing 96 of previously planned 100 participants; top-line data expected in early 2021
Atyr Pharma Inc., of San Diego ATYR-1923 Immunomodulator COVID-19 Dosed first patient with severe respiratory complications due to COVID-19
Cardiff Oncology Inc., of San Diego Onvansertib ATP competitive inhibitor of serine/threonine polo-like-kinase 1 enzyme Relapsed or refractory acute myeloid leukemia Data from ongoing phase II study showed, of 7 patients completing 1 cycle of treatment at data cutoff, 28% achieved objective response; biomarker data showed decreases in mutant ctDNA after 1 cycle of treatment were highly predictive of clinical response
Catalyst Biosciences Inc., of South San Francisco Dalca Next-generation subcutaneous factor IX therapy Hemophilia B Phase IIb data showed that 28 days of daily dosing achieved protective target FIX levels of >12% in all participants, with FIX levels of up to 27% and a half-life of 2.5 to 5.1 days with no bleeds, demonstrating effective prophylaxis and the potential for lower or less frequent dosing; no neutralizing anti-drug antibodies were detected and no serious adverse events were reported
Humanigen Inc., of Burlingame, Calif. Lenzilumab Humaneered anti-GM-CSF antibody COVID-19 Data from trial in patients with COVID-19 pneumonia showed rapid clinical improvement with median time to recovery of 5 days, median time to discharge of 5 days and 100% survival to data cutoff date; patients also demonstrated rapid improvement in oxygenation, temperature, inflammatory cytokines and key hematological parameters consistent with improved clinical outcomes
Immunic Inc., of New York IMU-838 Oral DHODH inhibitor COVID-19 Dosed first patients in Calvid-1 trial; top-line data expected later this year
Inventiva SA, of Daix, France Lanifibranor (IVA-337) Small-molecule PPAR activator Nonalcoholic steatohepatitis Top-line data showed 24-week phase IIb Native study met primary endpoint in intent-to-treat population at dose of 1,200 mg/day with a statistically significant (p=0.004) decrease of at least 2 points in SAF activity score (combining hepatocellular inflammation and ballooning), compared to baseline, with no worsening of fibrosis; 49% of patients in that dose group achieved primary endpoint vs. 27% in placebo arm 
Kyowa Kirin Co. Ltd., of Tokyo Tenapanor NHE3 inhibitor Chronic kidney disease Phase II data in Japanese study evaluating decrease in mean pill burden showed statistical significance, with 71.6% (p<0.001) of patients achieving at least a 30% reduction in mean pill burden; overall mean reduction in phosphate binder usage was 80% (reduction from 14.7 to 3 pills per day), while maintaining serum phosphorus control
Momenta Pharmaceuticals Inc., of Cambridge, Mass. Nipocalimab (M-281) IgG1 anti-FcRn monoclonal antibody Generalized myasthenia gravis Top-line data from interim analysis of Vivacity-MG study showed efficacy in myasthenia gravis activities of daily living (MG-ADL) score, the primary endpoint; 52% of patients had rapid, significant and durable reductions in MG-ADL scores (at least 2-point reduction from baseline for at least 4 consecutive weeks) across all 4 dosing arms, vs.15% for placebo (p=0.017); statistically significant relationship observed between IgG reduction and clinical benefit for patients taking nipocalimab (p<0.0001); patients across all 4 dosing arms showed rapid reductions in MG-ADL scores, with clinically meaningful changes from baseline within 2 weeks; study expected to complete in third quarter of 2020
Navidea Biopharmaceuticals Inc., of Dublin, Ohio Tc99m tilmanocept Imaging agent Rheumatoid arthritis Full enrollment in NAV3-31 phase IIb trial; study on track for last patient to be screened and evaluated by end of 2020
Solasia Pharma KK, of Tokyo SP-02 (darinaparsin) Mitochondrial-targeted agent Relapsed or refractory peripheral T-cell lymphoma Results from pivotal study in Asia showed primary endpoint of antitumor effect was achieved, with no new safety concerns identified
Tarsus Pharmaceuticals Inc., of Irvine, Calif. TP-03 Topical ophthalmic drug Demodex blepharitis  Results from phase IIa Mars study showed 4 weeks of treatment was well-tolerated, with statistically significant decreases in mean collarette score and mite density beginning at day 14 and continuing throughout the study period of 90 days 
Phase III
Ardelyx Inc., of Fremont, Calif. Tenapanor NHE3 inhibitor Chronic kidney disease Data from ongoing Normalize extension study testing monotherapy or combination with sevelamer to achieve serum phosphorus levels in normal range in CKD patients on dialysis showed mean serum phosphorous reduction of 2.33 mg/dL, from a mean baseline phosphorus of 7.27 mg/dL at the beginning of the Phreedom trial to a mean of 4.94 mg/dL at the time of analysis; of the 171 patients in interim analysis who completed up to 9 months of treatment in extension study, up to 47.4% achieved a normal serum phosphorus level, and of those, the majority were on tenapanor alone or tenapanor with low-dose sevelamer of ≤3 sevelamer tablets per day
Bayer AG, of Whippany, N.J. Finerenone (BAY 94-8862) Nonsteroidal, selective mineralocorticoid receptor antagonist Heart failure Started Finearts-AF study testing morbidity and mortality in patients with symptomatic heart failure (NYHA class II-IV) with left ventricular ejection fraction of ≥40%; primary objective is to demonstrate superiority over placebo in reducing rate of composite endpoint of cardiovascular death and total (first and recurrent) heart failure (HF) events (defined as hospitalizations for HF or urgent HF visits)
Beyondspring Inc., of New York Plinabulin Immune and stem cell modulator Chemotherapy-induced neutropenia Top-line results from Protective-2 study in combination with Neulasta showed significant enhancement over Neulasta alone in the rate of grade 4 neutropenia prevention (p<0.01), the primary endpoint; interim results also met key secondary endpoint, the duration of severe neutropenia in cycle 1 (p<0.05)
Eli Lilly and Co., of Indianapolis Baricitinib JAK1/2 inhibitor COVID-19 Enrolled first patient in study testing baricitinib in hospitalized adults with COVID-19; about 400 patients expected to be enrolled, with data in the next few months
Genentech, of South San Francisco, a member of the Roche Group, and PTC Therapeutics Inc., of South Plainfield, N.J. Risdiplam SMN-2 splicing modifier Spinal muscular atrophy 2-year data from part 1 of pivotal Sunfish trial showed drug significantly improved motor function after 24 months of treatment compared to natural history data in people, ages 2-25, with type 2 or 3 SMA; preliminary 12-month data from Jewelfish trial in people with all types of SMA, ages 6 months to 60 years, previously treated with other SMA therapies, showed rapid and sustained increases in survival motor neuron protein levels
Glycomimetics Inc., of Rockville, Md. Rivipansel Glycomimetic candidate that binds to E-, P- and L-selectin Acute vaso-occlusive crisis Post hoc analysis from Reset study showed patients treated within about 26 hours of onset of pain experienced statistically significant improvements in the primary efficacy endpoint of time to readiness for discharge compared to placebo (p=0.03, median improvement was 58 hours); statistically significant reduction in soluble E-selectin, a biomarker indicating the drug had intended biological effect
H. Lundbeck A/S, of Copenhagen Vyepti (eptinezumab-jimr) Monoclonal antibody targeting CGRP Migraine Post-hoc findings from Promise 1 study in episodic migraine and Promise 2 study in chronic migraine further support use as preventive treatment, with onset of action day 1 after administration; separate post-hoc subgroup analysis of Promise 2 showed that for patients diagnosed with both chronic migraine and medication-overuse headache (MOH), Vyepti consistently reduced use of acute headache medications in patients with chronic migraine to levels below MOH thresholds over 6 months 
Iveric Bio Inc., of New York Zimura (avacincaptad pegol) Complement C5 inhibitor Geographic atrophy secondary to age-related macular degeneration 18-month data support previous 12-month data, at which time Zimura met prespecified primary endpoint; reduction in mean rate of GA growth over 18 months was 28.11% for 2-mg group as compared to corresponding sham control group and 29.97% for 4-mg group as compared to corresponding sham control group
Oncoimmune Inc., of Rockville, Md. CD24Fc  Biologic that fortifies innate immune checkpoint against excessive inflammation caused by tissue injuries COVID-19 First 70 patients randomized in study of severe and critical COVID-19; institutional review board reviewed safety data and approved continuing enrollment while interim analysis occurs
Phathom Pharmaceuticals Inc., of Florham Park, N.J. Vonoprazan  Oral small-molecule potassium-competitive acid blocker Erosive esophagitis Randomized first new patients in Phalcon-EE study after temporarily pausing new patient randomization due to COVID-19
Phathom Pharmaceuticals Inc., of Florham Park, N.J. Vonoprazan  Oral small-molecule potassium-competitive acid blocker H. pylori infection Randomized first new patients in Phalcon-HP study after temporarily pausing new patient randomization due to COVID-19
Roche Holding AG, of Basel, Switzerland Venclexta (venetoclax) Targets Bcl-2 protein Acute myeloid leukemia Results from Viale-A study showed combination with azacitidine in previously untreated patients ineligible for intensive induction chemotherapy reduced the risk of death (overall survival) by 34% vs. azacitidine alone (median OS=14.7 months vs. 9.6 months; p<0.001); combination also led to higher rates of composite complete remission at 66.4% vs. 28.3% with azacitidine alone (p<0.001) 


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