Company Product Description Indication Status
Phase I
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)
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
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
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
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 
Phase II
American Brivision Corp., of Fremont, Calif. ABV-1505 Norepinephrine transporter inhibitor Attention deficit hyperactivity disorder  Initiated new phase II part I trial for adults; 6 patients will receive low-dose treatment (380 mg) 3 times a day for 28 days followed by a high-dose treatment (760 mg) 3 times a day for another 28 days
Aptinyx Inc., of Evanston, Ill.   NYX-458 NMDA receptor modulator Cognitive impairment associated with Parkinson’s disease Initiated 135-patient study to evaluate daily oral dosing of 10 mg, 30 mg or 100 mg, compared to placebo, over 12-week period; effects of NYX-458 will be evaluated across multiple endpoints related to attention, memory, executive function, visuospatial deficits and patient quality of life; top-line data expected in second half of 2021
Axsome Therapeutics Inc., of New York AXS-12 (reboxetine) Norepinephrine reuptake inhibitor Cataplexy in narcolepsy Met prespecified primary endpoint by demonstrating a highly statistically significant reduction from baseline in the mean weekly number of cataplexy attacks, averaged for the 2-week treatment period (overall treatment effect), as compared to placebo (p<0.001)
Bioarctic AB, of Stockholm, and Eisai Co. Ltd., of Tokyo BAN-2401 Amyloid beta-targeting monoclonal antibody Alzheimer’s disease Data from ongoing phase IIb study showed amyloid reductions in the brain that occurred as a result of treatment persisted after treatment was stopped; differences in benefits on clinical outcomes were maintained after stopping treatment in patients who received BAN-2401 at 2 highest doses vs. placebo
Biolinerx Ltd., of Tel Aviv, Israel BL-8040 CXCR4 inhibitor Pancreatic cancer Preliminary data from triple-combination arm of the ongoing phase IIa Combat/Keynote-202 study showed combination of Keytruda and chemotherapy elicited high response and disease control rates in patients with metastatic disease; best response for the evaluable population showed 4 partial response and 8 stable disease patients; median progression-free survival and overall survival not yet reached; survival data remain on track for mid-2020
Cytokinetics Inc., of South San Francisco Reldesemtiv Fast skeletal muscle troponin activator Amyotrophic lateral sclerosis Subgroup analyses from failed FORTITUDE-ALS trial of all dose groups combined vs. placebo showed similar effect on slow vital capacity (SVC), ALS Functional Rating Scale-Revised (ALSFRS-R) and muscle strength by hand held dynamometry (HHD) at 12 weeks regardless of treatment with edaravone and/or riluzole; for use and non-use of edaravone, the treatment difference for reldesemtiv relative to placebo for ALSFRS-R was 1.25 points (p=0.06) and 0.77 points (p=0.06), respectively, and decline in SVC was 3.07 percentage points less on reldesemtiv vs. placebo in patients using edaravone (p=0.14), and 1.21 percentage points less vs. placebo in patients not using edaravone (p=0.32); HHD declined 6.94 percentage points less vs. placebo for patients taking edaravone (p=0.14), and 1.31 percentage points those not taking it (p=0.57); treatment difference with or without riluzole on ALSFRS-R was 0.86 (p=0.03) and 0.84 (p=0.28) points, respectively, while decline in SVC was 1.64 percentage points less (p=0.16) and 1.81 percentage points less (p=0.46), respectively, and decline in HHD was 2.22 (p=0.36) and 4.36 (p=0.27) less, respectively
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-3918 Pozelimab Paroxysmal nocturnal hemoglobinuria Top-line data validated weekly 800-mg subcutaneous dosing regimen, following an initial intravenous loading dose; results from initial 6-patient cohort show pozelimab reduced lactate dehydrogenase to normal levels at week 8; no adverse events were serious or led to discontinuation
Phase III
Acadia Pharmaceuticals Inc., of San Diego Pimavanserin Small molecule Dementia-related psychosis  Top-line results from Harmony study in 392 patients met the primary endpoint and was stopped at the preplanned interim analysis by significantly reducing risk of relapse of psychosis by 2.8-fold vs. placebo (1-sided p=0.0023); met key secondary endpoint by significantly reducing risk of discontinuation for any reason by 2.2-fold (1-sided p=0.0024)
Allena Pharmaceuticals Inc., of Newton, Mass.   Reloxaliase Oxalate decarboxylase Enteric hyperoxaluria Realigning resources to support the continued development for patients with enteric hyperoxaluria following positive top-line results from Urirox-1; plans to re-engage with FDA to discuss measures to potentially streamline ongoing Urirox program through modifications to the adaptive Urirox-2 trial design
Allergan plc, of Dublin Ubrogepant Oral, small-molecule CGRP receptor antagonist Acute migraine Data published in The New England Journal of Medicine from second pivotal trial showed ubrogepant vs. placebo led to significantly greater rates of pain freedom and freedom from most bothersome migraine-associated symptom at 2 hours with both 50-mg and 100-mg doses
Aurinia Pharmaceuticals Inc., of Victoria, British Columbia Voclosporin Calcineurin inhibitor Lupus nephritis Results from pivotal Aurora trial, in combination with mycophenolate and low-dose corticosteroids, met primary endpoint of renal response rates of 40.8% for voclosporin vs. 22.5% for control (OR 2.65; p < 0.001); all prespecified hierarchical secondary endpoints achieved statistical significance in favor of voclosporin, which included renal response at 24 weeks, partial renal response at 24 and 52 weeks, time to achieve urinary protein-to-creatinine ratio (UPCR) ≤ 0.5, and time to 50% reduction in UPCR
Revance Therapeutics Inc., of Newark, Calif. DaxibotulinumtoxinA Long-acting neuromodulator Moderate to severe glabellar lines Pooled results from Sakura 1 and 2 studies published in the Journal of the American Academy of Dermatology showed primary endpoint of 2-point composite response at week 4 was achieved in 73.8% of patients in DAXI group vs. 0.5% in placebo group (p<0.0001); proportion of responders was similar regardless of whether baseline glabellar line severity had been moderate (75.4%) or severe (71.2%)
Rhythm Pharmaceuticals Inc., of Boston Setmelanotide MC4R agonist Bardet-Biedl syndrome or Alström syndrome Completed enrollment in study testing drug for treatment of insatiable hunger and severe obesity in patients with BBS and Alström syndrome; top-line data anticipated in fourth quarter of 2020 or early in first quarter of 2021
Sage Therapeutics Inc., of Cambridge, Mass. SAGE-217 Oral neuroactive steroid GABAA receptor positive allosteric modulator Major depressive disorder Mountain study did not meet primary endpoint of statistically significant reduction from baseline vs. placebo in 17-item Hamilton Rating Scale for Depression (HAM-D) total score at day 15; treatment was associated with mean reduction of 12.6 in HAM-D total score vs. 11.2 for placebo (p=0.115); patients in 30-mg group achieved statistically significant reductions in the HAM-D total score at days 3, 8 and 12 (p<0.018 for each timepoint)


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