Cancer | Cardiovascular | Dermatologic | Endocrine/metabolic | Gastrointestinal | Genitourinary/sexual function | Hematologic | Immune | Infection | Musculoskeletal | Neurology/psychiatric | Ocular | Other/miscellaneous | Respiratory | Toxicity/intoxication |

Phase III clinical data reported in March 2020

Company Product Description Indication Phase III status

Cancer

Astrazeneca plc, of Cambridge, U.K. Imfinzi (durvalumab) PD-L1 inhibitor Unresectable, stage IV bladder cancer Danube trial did not meet primary endpoints of improving overall survival vs. standard-of-care chemotherapy for Imfinzi monotherapy in patients whose tumor cells and/or tumor-infiltrating immune cells express high levels of PD-L1 or for Imfinzi plus tremelimumab in patients regardless of PD-L1 expression
Astrazeneca plc, of Cambridge, U.K. Imfinzi (durvalumab) PD-L1 inhibitor Small-cell lung cancer Top-line data from final analysis of Caspian combination trial with choice of standard-of-care (SoC) chemotherapies showed sustained overall survival (OS) for extensive-stage disease in first-line setting; second arm testing anti-CTLA4 monoclonal antibody tremelimumab with Imfinzi and SoC did not meet primary endpoint of statistically significant OS improvement
Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. Cediranib VEGF receptor tyrosine kinase inhibitor Platinum-sensitive relapsed ovarian cancer Results showed GY004 study testing combination with Lynparza (olaparib) vs. platinum-based chemotherapy did not meet primary endpoint in intent-to-treat population of statistically significant improvement in progression-free survival
Bristol Myers Squibb Co., of New York, and Abbvie Inc., of North Chicago Empliciti (elotuzumab) Monoclonal antibody targeting signaling lymphocyte activation molecule family member 7 First-line multiple myeloma In the Eloquent-1 study, Empliciti plus Revlimid (lenalidomide) and dexamethasone didn't improve progression-free survival compared to Revlimid plus dexamethasone; data to be presented at a future medical meeting
Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J., and Symbio Pharmaceuticals Ltd., of Tokyo Treakisym (bendamustine) PARP modulator Low-grade non-Hodgkin’s lymphoma, mantle cell lymphoma and chronic lymphocytic leukemia Completed enrollment in Japanese study testing a 10-minute rapid infusion of the drug
EMD Serono, the biopharmaceutical business of Merck KGaA, of Darmstadt, Germany, and Pfizer Inc., of New York Avelumab PD-L1 antibody Squamous cell carcinoma of the head and neck Accepted recommendation from independent data monitoring committee that Javelin trial testing combination with chemoradiotherapy (CRT) vs. standard-of-care CRT in untreated locally advanced disease is unlikely to show statistically significant improvement in primary endpoint of progression-free survival based on a preplanned interim analysis
Idera Pharmaceuticals Inc., of Exton, Pa. Tilsotolimod (IMO-2125) Synthetic Toll-like receptor 9 agonist Anti-PD-1-refractory advanced melanoma Completed enrollment in Illuminate-301 trial testing combination with Yervoy (ipilimumab, Bristol-Myers Squibb Co.); top-line overall response rate and other preliminary data expected in the first quarter of 2021
Karyopharm Therapeutics Inc., of Newton, Mass. Xpovio (selinexor) Exportin 1 inhibitor Multiple myeloma Boston study assessing study drug dosed once-weekly in combination with once-weekly Velcade (bortezomib, Takeda Pharmaceutical Co. Ltd.) and low-dose dexamethasone vs. standard twice-weekly Velcade plus low-dose dex met primary endpoint of statistically significant increase in progression-free survival, with median of 13.93 months for Xpovio arm vs. 9.46 months for control, or reduction of 47% (p=0.0066)
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) PD-1 inhibitor Classical Hodgkin lymphoma Pivotal, confirmatory Keynote-204 trial in adults with relapsed/refractory disease met 1 primary endpoint of statistically significant improvement in progression-free survival vs. brentuximab vedotin (Adcetris, Seattle Genetics Inc./Takeda Pharmaceutical Co. Ltd.) based on interim analysis conducted by an independent DMC; per prespecified analysis plan, other dual primary endpoint of overall survival was not formally tested at interim analysis so study is continuing
Onconova Therapeutics Inc., of Newtown, Pa. Rigosertib RAS mimetic Myelodysplastic syndrome Completed planned enrollment of 360 higher-risk patients in Inspire trial
Oncosec Medical Inc., of San Diego Tavo (tavokinogene telseplasmid) Plasmid-based interleukin-12 delivered via electroporation gene delivery system Metastatic melanoma Data published in the Annals of Oncology showed the objective overall response rate was 35.7% in the main study, with a complete response rate of 17.9%; the median progression-free survival in the main study was 3.7 months while the median overall survival was not reached at a median follow-up of 29.7 months
Polyphor AG, of Allschwil, Switzerland Balixafortide CXCR4 chemokine antagonist Breast cancer Following first prespecified interim safety analysis on first 193 randomized participants with HER2-negative, locally recurrent or metastatic disease, independent data safety monitoring board recommended pivotal study continue without modifications
QED Therapeutics Inc., of San Francisco, a subsidiary of Bridgebio LLC Infigratinib (BGJ-398) Oral FGFR1-3 selective tyrosine kinase inhibitor Invasive urothelial carcinoma Patients dosed in Proof 302 trial testing drug as adjuvant treatment in patients whose cancers harbor susceptible FGFR3 genetic alterations and who are at high risk of recurrence following surgical resection; primary outcome is disease-free survival
Rafael Pharmaceuticals Inc., of Cranbury, N.J. Devimistat (CPI-613) 2 oxoglutarate dehydrogenase inhibitor; dual pyruvate dehydrogenase inhibitor/kinase stimulator Metastatic pancreatic cancer > 75% of expected 500 participants enrolled ahead of scheduled in pivotal Avenger 500 combination trial with modified Folfirinox as first-line therapy; COVID-19 task force monitoring trials to make changes, as needed
Roche Holding AG, of Basel, Switzerland Venclexta/Venclyxto (venetoclax) BCL-2 protein inhibitor Acute myeloid leukemia Viale-A combination study with hypomethylating agent azacitidine showed statistically significant improvement in overall survival in those with previously untreated disease who were ineligible for intensive induction chemotherapy vs. azacitidine alone; details expected to report at undisclosed medical meeting
Soligenix Inc., of Princeton, N.J. SGX-301 (synthetic hypericin) Heat-shock protein inhibitor Cutaneous T-cell lymphoma Cycle 1 data from pivotal Flash trial that enrolled 169 participants randomized 2-to-1 to study drug or placebo showed statistically significant treatment response (p=0.04) in Composite Assessment of Index Lesion Score primary endpoint assessment at 8 weeks
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Ninlaro (ixazomib) Oral proteasome inhibitor Multiple myeloma Addition of ixazomib to lenalidomide and dexamethasone in Tourmaline-MM2 trial resulted in an improvement in median progression-free survival of 13.5 months (p=0.073); but did not meet the threshold for statistical significance
Vascular Biogenics Ltd. (VBL Therapeutics), of Tel Aviv, Israel VB-111 (ofranergene obadenovec) Dual CD95/TNF receptor modulator Colorectal cancer Data safety monitoring committee reviewed unblinded data and determined study has met the interim prespecified criterion, of an absolute percentage advantage of 10% or higher in CA-125 response in the VB-111 treated arm vs. control; recommended study proceed without modification
Company Product Description Indication Phase III status

Cardiovascular

Biocardia Inc., of San Carlos, Calif. Cardiamp Autologous bone marrow-derived stem cell therapy Heart failure Following prespecified safety review, independent data safety monitoring board recommended pivotal trial continue as planned
Milestone Pharmaceuticals Inc., of Montreal Etripamil Calcium channel inhibitor Paroxysmal supraventricular tachycardia Node-301 trial evaluating termination of SVT episodes in at-home setting missed primary endpoint of time to conversion of SVT to sinus rhythm vs. placebo over 5-hour period following study drug administration (median time to conversion of 25 minutes for etripamil vs. 50 minutes for placebo, p=0.12)
Novartis AG, of Basel, Switzerland Inclisiran PCSK9 gene inhibitor Atherosclerotic cardiovascular disease Findings from pivotal Orion-10 and -11 trials, published online in The New England Journal of Medicine, showed study drug, at 17 months, resulted in placebo-adjusted LDL-C reductions of 52% and 50%, respectively, and time-adjusted reductions from months 3 through 18 of 54% and 49%, respectively
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone methyl Activator of Nrf2 Connective tissue disease-associated pulmonary arterial hypertension Stopped Catalyst study due to risk of COVID-19; initial review of data by the data safety monitoring board suggest the study is unlikely to meet the primary endpoint of improvement in 6-minute walk distance compared to placebo at week 24
Resverlogix Corp., of Calgary, Alberta Apabetalone Bromodomain containing protein inhibitor Acute coronary syndrome BETonMACE study, reported in Journal of the American Medical Association, did not reach significance in primary outcome measure but showed trend toward reduction in MACE vs. placebo (10.3% vs. 12.4%, respectively) in those with type 2 diabetes with acute coronary syndrome and low HDL cholesterol; clinically significant reductions in primary endpoint observed in prespecified subgroups, including those with baseline estimated glomerular flow rate of <60 mL/min/1.73 m2
Company Product Description Indication Phase III status

Dermatologic

Abeona Therapeutics Inc., of New York EB-101 COL7A1 gene stimulator Recessive dystrophic epidermolysis bullosa First of up to 15 participants treated in pivotal Viital study assessing effectiveness in about 30 large, chronic wound sites; primary outcome measure is wound healing vs. untreated wound sites on same patients; secondary endpoints include assessments of pain and patient-reported outcomes
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. Topical roflumilast PDE4 inhibitor Plaque psoriasis Submitted protocol amendment to FDA to include children, ages 2-11, in ongoing Dermis-1 and Dermis-2 pivotal trials
Avita Medical Ltd., of Valencia, Calif. Recell System Autologous skin cell suspension Soft tissue reconstruction of skin defects Enrolled first of at least 65 patients in the study comparing the Recell System to conventional skin grafting; primary endpoints are superior donor skin sparing and noninferior incidence of healing by 8 weeks post-treatment
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. Berotralstat (BCX-7353) Oral plasma kallikrein antagonist Prophylaxis of hereditary angioedema attacks Data from Apex-2 trial showed 150-mg once-daily dose reduced patients’ monthly use of standard-of-care (SoC) on-demand medicine by 53.6% (p<0.001) vs. placebo, and reduced number of HAE attacks requiring acute SoC treatment by 49.2% (p<0.001) vs. placebo
Can-Fite Biopharma Ltd., of Petach Tikva, Israel Piclidenoson A3 adenosine receptor agonist Moderate to severe plaque psoriasis Implementing an interim analysis of the Comfort study; data expected in the fourth quarter of 2020
Otsuka Pharmaceutical Co. Ltd., of Tokyo, and Medimetriks Pharmaceuticals Inc., of Fairfield, N.J. Difamilast PDE4 inhibitor Atopic dermatitis In both the adult and pediatric trials, saw IGA success rates were higher in the active drug treatment groups than in the vehicle groups; no major adverse reactions
Pfizer Inc., of New York Abrocitinib Janus kinase 1 inhibitor Atopic dermatitis Jade Compare study met co-primary efficacy endpoints in treating moderate to severe disease; percentage who achieved each co-primary efficacy endpoint at week 12 was statistically superior with both 100 mg and 200 mg doses vs. placebo and superiority to placebo with both doses was maintained at week 16; active control dupilumab (Dupixent, Regeneron Pharmaceuticals Inc./Sanofi SA) was superior to placebo at weeks 12 and 16; key secondary endpoint, percentage with clinically significant reduction in itch by week 2 of treatment, was statistically superior for 200 mg abrocitinib vs. dupilumab and numerically higher but not statistically significantly higher for 100 mg vs. dupilumab
Company Product Description Indication Phase III status

Endocrine/metabolic

Dicerna Pharmaceuticals Inc., of Lexington, Mass. Nedosiran LDHA gene inhibitor Primary hyperoxaluria Initial observations on 14 participants from Phyox1 trial enrolled in Phyox3 suggested study drug was well-tolerated, with no injection-site reactions or drug-related severe adverse events; on at least 2 visits, normalization or near-normalization of urinary oxalate levels were seen in 4 patients who received at least 3 monthly doses
Novartis AG, of Basel, Switzerland Inclisiran PCSK9 gene inhibitor Heterozygous familial hypercholesterolemia Findings from pivotal Orion-9 trial, published online in The New England Journal of Medicine, showed study drug, at 17 months, reduced LDL-C by 50% with time-adjusted reduction of 45% from months 3 through 18 vs. placebo
Provention Bio Inc., of Oldwick, N.J. PRV-031 (teplizumab) CD3 antagonist Type 1 diabetes Paused Protect study due to COVID-19 pandemic
Company Product Description Indication Phase III status

Gastrointestinal

Allakos Inc., of Redwood City, Calif. Antolimab (AK-002) Sialic acid-binding Ig-like lectin 8 inhibitor Eosinophilic gastritis; eosinophilic duodenitis Initiated trial expected to enroll ~160 participants with active, biopsy-confirmed EG and/or EoD, randomized 1-to-1 to 1 mg/kg of study drug for first month followed by 5 doses of 3 mg/kg monthly or monthly placebo; co-primary endpoints are proportion achieving ? 4 eosinophils in 5 high-powered fields (hpfs) in stomach and/or ?15 eosinophils in 3 hpfs in duodenum and absolute change in Total Symptom Score
Genfit SA, of Lille, France Elafibranor Dual PPAR alpha/delta agonist Fibrosis; primary biliary cholangitis COVID-19 pandemic not expected to affect receipt of FDA feedback from Resolve-It trial in fibrosis, where final visits of first 1,000 participants occurred and database related to cohort was locked on schedule at end of February; extension phase of Resolve-It continues but screening of new participants temporarily halted; initiation of phase III study in PBC placed on hold
Ironwood Pharmaceuticals Inc., of Boston IW-3718 (colesevelam) Gastric-retentive formulation of bile acid sequestrant colesevelam Gastroesophageal reflux No longer guiding for top-line data in the second half of 2020 due to certain clinical sites suspending enrollment and lower patient participation; trial is 70% enrolled
Phathom Pharmaceuticals Inc., of Florham Park, N.J. Vonoprazan H+ K+ ATPase inhibitor Erosive esophagitis Randomization in pivotal Phalcon-EE trial, initiated in fourth quarter of 2019, paused temporarily to cooperate with efforts to halt spread of COVID-19; top-line data expected in 2021
Company Product Description Indication Phase III status

Genitourinary/sexual function

Astrazeneca plc, of Cambridge, U.K. Farxiga (dapagliflozin) SGLT2 inhibitor Chronic kidney disease DAPA-CKD study will be stopped early following a recommendation from an independent data monitoring committee due to overwhelming efficacy; data to be presented in a forthcoming medical meeting
Iterum Therapeutics plc, of Dublin Sulopenem Broad-spectrum penem beta-lactam antibiotic Complicated and uncomplicated urinary tract infection Top-line data from Sure 1 and 2 studies expected early in second quarter of 2020
Obseva SA, of Geneva Linzagolix GNRH receptor antagonist Uterine fibroids Results from fully enrolled Primrose 1 and 2 trials remain on track to report in second quarter of 2020
Obseva SA, of Geneva Linzagolix GNRH receptor antagonist Endometriosis Screening and randomization in ongoing Edelweiss 2 and 3 trials placed on voluntary hold due to COVID-19; study sites continue to manage enrolled participants
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone methyl Activator of Nrf2 Autosomal dominant polycystic kidney disease Pausing enrollment in study due to COVID-19
Retrophin Inc., of San Diego Sparsentan Blocks endothelin receptor type A and angiotensin receptor Focal segmental glomerulosclerosis Duplex study enrolled first 190 patients; interim data after completion of 36 weeks of treatment expected in the first quarter of 2021
Urovant Sciences, a subsidiary of Sumitovant Biopharma Ltd., of London Vibegron Beta 3 adrenoceptor agonist Overactive bladder Results from Empowur trial, reported in March 2019, were published online in Journal of Urology
Company Product Description Indication Phase III status

Hematologic

Bristol Myers Squibb Co., of Princeton, N.J., and and Acceleron Pharma Inc., of Cambridge, Mass. Reblozyl (luspatercept-aamt) TGF beta receptor antagonist; TGF beta ligand inhibitor; GDF modulator Anemia; beta-thalassemia Published results from pivotal Believe trial in The New England Journal of Medicine following the therapy's November 2019 approval
Novo Nordisk A/S, of Bagsvaerd, Denmark Concizumab Inhibits tissue factor pathway inhibitor Hemophilia A and B Studies explorer7 and 8 were paused as a result of occurrence of nonfatal thrombotic events in 3 patients enrolled; company and independent data monitoring committee are assessing the events; phase II program, explorer5, also paused
Uniqure NV, of Amsterdam, the Netherlands Etranacogene dezaparvovec (AMT-061) AAV5-based gene therapy Hemophilia B Achieved targeted dosing of patients in the Hope-B pivotal trial
Company Product Description Indication Phase III status

Immune

Aimmune Therapeutics Inc., of Brisbane, Calif. Palforzia (peanut allergen powder-dnfp) Oral immunotherapy Peanut allergy New data from ARC004 follow-on trial to 52-week Palisade study showed patients tolerated more peanut protein, experienced fewer adverse events and continued immunomodulation as evidenced by reductions in peanut-specific immunoglobulin blood levels after an additional 56 weeks of daily treatment
Medday Pharmaceuticals SA, of Paris MD-1003 (biotin) Dual acetyl CoA carboxylase-1/2 stimulator Progressive multiple sclerosis Second pivotal and confirmatory trial (SP12) missed primary endpoint of reversing functional disability; study also failed to meet secondary endpoints
Company Product Description Indication Phase III status

Infection

Biondvax Pharmaceuticals Ltd., of Jerusalem M-001 (Multimeric-001) Non-flagellin-based, single-protein vaccine Influenza virus infection Ongoing pivotal European trial of universal flu candidate so far unaffected by coronavirus pandemic; CRO and statistician expect study to reach targeted number of swab samples, with results expected by year-end
Cytodyn Inc., of Vancouver, Wash. Leronlimab CCR5 antagonist COVID-19 2 additional individuals treated for respiratory complications at undisclosed New York hospital under emergency IND from FDA, bringing total treated to 4
Genentech Inc., unit of Roche Holding AG, of Basel, Switzerland Actemra (tocilizumab) IL-6 antagonist/modulator COVID-19 Pending FDA authorization, trial expected to begin in early April in collaboration with BARDA to assess study drug plus standard of care in hospitalized adults with severe COVID-19 pneumonia vs. placebo plus standard of care; global study expected to enroll 330 participants, with primary and secondary endpoints including clinical status, mortality, mechanical ventilation and ICU variables
Gilead Sciences Inc., of Foster City, Calif. Descovy (emtricitabine 200 mg/tenofovir alafenamide 25 mg) tablets Antiretroviral therapy HIV pre-exposure prophylaxis Longer-term results from Discovery trial for PrEP use showed continued noninferior efficacy and continued favorable changes in key markers of renal and bone safety at week 96 vs. Truvada (emtricitabine 200 mg and tenofovir disoproxil fumarate 300 mg); those results were achieved in the overall study population of men and transgender women at risk for HIV infection, as well as in study subpopulations of participants 50 and older, those younger than 25, and those with moderate renal impairment
Humanigen Inc., of Burlingame, Calif. Lenzilumab GM-CSF ligand inhibitor COVID-19 Company seeking to advance development for prevention and treatment of cytokine storm that can lead to acute respiratory distress syndrome associated with SARS-CoV-2 infection; trials expected to begin upon approval by regulatory authorities; company also plans to make study drug available on compassionate use basis
Novan Inc., of Morrisville, N.C. SB-206 (berdazimer sodium) Nitric oxide-releasing macromolecule Molluscum contagiosum infection Safety evaluation of B-Simple program through week 24 showed treatment-emergent adverse events (TEAEs) reported in > 5% of those in treated groups were application site pain and/or erythema; most TEAEs were mild or moderate, and all study drug discontinuations in treatment arm were due to application site reactions; no treatment-related SAEs reported across both studies; type C teleconference with FDA remains scheduled for April 1, 2020
Novavax Inc., of Gaithersburg, Md. Nanoflu Recombinant quadrivalent seasonal influenza vaccine Influenza virus infection Pivotal trial in adults 65 and older achieved primary endpoints of day 28 ratio of geometric mean titers (24% to 66% higher) responses and difference in seroconversion rates (11.4 to 20.4 percentage points higher) for vaccine's 4 strains vs. Fluzone Quadrivalent (Sanofi SA); Nanoflu was well-tolerated and safety profile was comparable to comparator with modest increase in local adverse events; BLA filing expected under FDA accelerated approval pathway
Pfizer Inc., of New York 20-valent pneumococcal conjugate vaccine (20vPnC, PF-06482077) Multivalent Streptococcus pneumoniae vaccine Streptococcus pneumoniae infection Top-line results in participants with no history of pneumococcal vaccination showed noninferiority objectives were met in those 60 and older for serotypes in common with Prevnar 13 (pneumococcal 13-valent conjugate vaccine [diphtheria CRM197 protein], Pfizer) at 1 month after vaccination and for 6 of 7 additional serotypes vs. pneumococcal polysaccharide vaccine PPSV-23 (Pneumovax 23, Merck & Co. Inc.); 1 new serotype missed noninferiority criteria by small margin; secondary immunogenicity objectives in 18- to 59-year-old participants compared to 60- to 64-year-olds met noninferiority for all serotypes
Phathom Pharmaceuticals Inc., of Florham Park, N.J. Vonoprazan H+ K+ ATPase inhibitor Helicobacter pylori infection Randomization in pivotal Phalcon-HP combination trial with amoxicillin (dual therapy) and with amoxicillin and clarithromycin (triple therapy), initiated in fourth quarter of 2019, paused temporarily to cooperate with efforts to halt spread of COVID-19; top-line data expected in 2021
Seres Therapeutics Inc., of Cambridge, Mass. SER-109 Ecobiotic (encapsulated mixture of bacterial spores from fecal microbiota) Clostridium difficile infection Completed enrollment of Ecospor III trial at 181 individuals with recurrent disease; enrollment halted prior to expected 188 participants due to COVID-19 pandemic; top-line results expected in mid-2020
Viiv Healthcare Ltd., of London Cabotegravir Integrase inhibitor HIV 48-week data from Atlas-2M study in combination with rilpivirine met primary endpoint, showing administration every 8 weeks was noninferior when compared to administration every 4 weeks
Company Product Description Indication Phase III status

Musculoskeletal

Ipsen SA, of Paris Palovarotene Retinoic acid receptor gamma agonist Fibrodysplasia ossificans progressiva Paused dosing in the global Move trial as well as the ongoing phase II extension studies based on results of a futility analysis as part of the prespecified interim analysis; reinitiating dosing in patients 14 years and older
Company Product Description Indication Phase III status

Neurology/psychiatric

Addex Therapeutics Ltd., of Geneva Dipraglurant Metabotropic glutamate receptor 5 antagonist Parkinson’s disease Will delay start of registrational efficacy and safety study (Study 301) in levodopa-induced dyskinesia
Alkermes plc, of Dublin ALKS-3831 Once-daily, oral atypical antipsychotic drug Schizophrenia Data from 4-week Enlighten-1 trial testing ALKS-3831 vs. placebo in 403 patients experiencing acute exacerbation of schizophrenia were published in the Journal of Clinical Psychiatry; drug is under FDA review, with PDUFA date of Nov. 15, 2020
Avadel Pharmaceuticals plc, of Dublin FT-218 (sodium oxybate) GABA B receptor agonist Narcolepsy Last visit completed in Rest-On study that enrolled 212 participants to assess effectiveness of study drug in Micropump controlled-release formulation to treat excessive daytime sleepiness and cataplexy under FDA special protocol assessment; top-line data expected in second quarter of 2020
Avexis, unit of Novartis AG, of Basel, Switzerland Zolgensma (onasemnogene abeparvovec-xioi) SMN1 gene stimulator Spinal muscular atrophy Interim data from ongoing Spr1nt study continued to show participants achieved age-appropriate motor milestones when treated presymptomatically; 7 of 8 participants with 2 copies of SMN2 who achieved ability to sit independently did so within WHO window of normal development
Axsome Therapeutics Inc., of New York AXS-05 NMDA receptor antagonist Treatment-resistant depression In the Stride-1 study, AXS-05 produced a mean reduction of 11.6 points on the Montgomery-Åsberg Depression Rating Scale (MADRS) at week 6 (primary endpoint) compared to a 9.4-point reduction for patients taking bupropion (p=0.117); study met secondary endpoint MADRS score over the entire 6-week treatment period with mean reductions of 8.6 for AXS-05 vs. 6.7 for bupropion (p=0.031)
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Rimegepant CGRP receptor antagonist Migraine prevention Rimegepant produced a reduction of 4.5 days with migraines per month, compared to a 3.7-day reduction for placebo (p=0.0176); in patients not taking concomitant preventive treatment reduction was 4.9 days and 3.7 days for rimegepant and placebo, respectively (p=0.0020); 48% of patients taking rimegepant had at least a 50% reduction from baseline in the mean number of moderate to severe migraine days per month compared to 41% of patients taking placebo
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 (sublingual dexmedetomidine) Alpha 2A adrenoceptor agonist Agitation Enrollment on track in Serenity I and II trials assessing agitation associated with schizophrenia and bipolar disorder, respectively, with more than one-third of participants enrolled and treated; per previous guidance, trials expected to be completed by midyear 2020
Eisai Co. Ltd., of Tokyo, and Bioarctic AB, of Stockholm BAN-2401 Amyloid beta-targeting monoclonal antibody Alzheimer’s disease Study expands sites to 4 Swedish clinics; data expected in 2022
GW Pharmaceuticals plc, of Carlsbad, Calif. Epidiolex (cannabidiol) CBD oral solution Dravet syndrome Results from GWPCARE2 trial published in JAMA Neurology showed 2 doses, 10 and 20 mg/kg/day, significantly reduced convulsive seizure frequency vs. placebo in children, ages 2 to 18, with highly treatment-resistant disease; primary endpoint outcomes for the 10- and 20-mg/kg/day arms showed seizure reductions of 49% and 46% from baseline, respectively, vs 27% for placebo (10 mg/kg/day, p=0.0095 and 20 mg/kg/day, p=0.0299)
H. Lundbeck A/S, of Copenhagen, Denmark Vyepti (eptinezumab-jjmr) CGRP receptor antagonist Chronic migraine Data published in Neurology showed pivotal Promise 2 study met primary endpoint of statistically significant reductions in mean monthly migraine days (MMDs) after first dose over months 1 to 3; preventive effects of recently approved drug were shown as early as day 1 post-infusion and sustained throughout study; one-third of those who took drug experienced 75% or > reduction in number of mean MMDs at month 1
Company Product Description Indication Phase III status

Ocular

Eyegate Pharmaceuticals Inc., of Waltham, Mass. Ocular bandage gel (dexamethasone, iontophoresis delivery) Glucocorticoid receptor agonist Dry eye disease Study drug showed 25% improvement in central corneal region staining vs. 15% for control (Refresh Preservative-Free lubricant, Allergan plc), 8.2% improvement over 2-week period vs. short-term worsening and no benefit for control in high order ocular aberrations, and trend in improved best corrected visual acuity vs. no improvement for control
Eyepoint Pharmaceuticals Inc., of Watertown, Mass. Yutiq (fluocinolone acetonide intravitreal implant) Glucocorticoid receptor agonist Uveitis Top-line 36-month follow-up data from second phase III in chronic non-infectious disease showed long-term ability to reduce flares, with recurrence rate of 46.5%
Iveric Bio Inc. Zimura (avacincaptad pegol) Complement C5a receptor antagonist; complement cascade inhibitor Dry age-related macular degeneration Company decided to delay initiation of enrollment of patients in the second pivotal trial
Kala Pharmaceuticals Inc., of Watertown, Mass. Eysuvis (loteprednol etabonate, KPI-121) Steroid delivered via a mucus-penetrating particle Dry eye disease Drug improved ocular discomfort severity (ODS) at day 15 compared to placebo in the overall intent-to-treat (ITT) population (p=0.0002) and in the predefined subgroup of ITT patients with more severe ocular discomfort at baseline (p=0.0007); in the ITT population, compared to placebo, drug also improved conjunctival hyperemia at day 15 (p<0.0001), ODS at day 8 (p=0.0282) and total corneal staining at day 15 (p=0.0042)
Mimetogen Pharmaceuticals Inc., of Montreal Tavilermide Nerve growth factor mimetic Dry eye disease Completed enrollment of the 600-patient MIM-728 study; data expected in the fourth quarter of 2020
Oyster Point Pharma Inc., of Princeton, N.J. OC-01 Nicotinic acetylcholine receptor agonist Dry eye disease Completed enrollment of 758 patients in the Onset-2 study; data expected by the end of the second quarter of 2020; day 28 data impacted by COVID-19, which is predicted to reduce power for primary endpoint to 99%
Proqr Therapeutics NV, of Leiden, the Netherlands Sepofarsen CEP290 gene modulator Leber congenital amaurosis 10 Enrollment in pivotal trial paused due to COVID-19 pandemic
Company Product Description Indication Phase III status

Other/miscellaneous

Novo Nordisk A/S, of Bagsvaerd, Denmark Saxenda (liraglutide) GLP-1 analogue Obesity Results published in The New England Journal of Medicine showed the drug was superior in reducing body mass index standard deviation score at 56 weeks with a -0.22 estimated treatment difference; the study, conducted in adolescents, ages 12 to 18, was a postmarketing requirement of FDA and EMA
Company Product Description Indication Phase III status

Respiratory

Merck and Co. Inc., of Kenilworth, N.J. Gefapixant (MK-7264) Selective P2X3 receptor antagonist Refractory or unexplained chronic cough Top-line results from 2 trials showed primary efficacy endpoints were met for the gefapixant 45-mg twice-daily treatment arms, demonstrating statistically significant decrease in 24-hour coughs per hour vs. placebo at 12 and 24 weeks; 15-mg twice-daily treatment arms did not meet primary efficacy endpoint in either study
Company Product Description Indication Phase III status

Toxicity/intoxication

Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 (repurposed ondansetron) 5-HT 3 receptor antagonist Alcohol use disorder Pivotal trial in genetically targeted patients modified to protect participants during COVID-19 pandemic, including reducing number of in-person visits to clinical sites, replacing some visits with telemedicine behavioral treatments and assessments, removing non-essential elements from in-person assessments to reduce visit duration

No Comments