Cancer | Cardiovascular | Dermatologic | Endocrine/metabolic | Gastrointestinal | Genitourinary/sexual function | Immune | Infection | Inflammatory | Neurology/psychiatric | Ocular | Respiratory | Toxicity/intoxication |

Phase III clinical data reported in February 2020

Company Product Description Indication Phase III status

Cancer

Abbvie Inc., of North Chicago Venclexta (venetoclax) BCL-2 protein inhibitor Acute myeloid leukemia Viale-C combination trial with low-dose cytarabine (LDAC) vs. LDAC plus placebo missed primary endpoint of statistically significant improvement in overall survival (OS), showing 25% reduction in risk of death vs. LDAC with placebo (p=0.11); median OS for venetoclax with LDAC arm was 7.2 months vs. 4.1 months for LDAC alone, extended to median OS of 8.4 months vs. 4.1 months, respectively, after 6 additional months of follow-up
Actinium Pharmaceuticals Inc., of New York Iomab-B (iodine-131 apamistamab) Antibody-radiation conjugate comprising anti-CD45 antibody apamistamab and radioisotope I-131 Acute myeloid leukemia Results from Sierra trial showed rates of febrile neutropenia, sepsis and mucositis being markedly lower in the Iomab-B arm; updated results from the 50% enrollment midpoint analysis showed that, on an intent-to-treat basis, 78% of patients in the Iomab-B arm are potentially evaluable for the primary endpoint compared to 13% in the control arm
Astellas Pharma Inc., of Tokyo, and Pfizer Inc., of New York Xtandi (enzalutamide) Androgen receptor inhibitor Prostate cancer Prosper combination trial with androgen deprivation therapy (ADT) vs. placebo plus ADT in non-metastatic castration-resistant disease showed statistically significant improvement in secondary endpoint of overall survival, with adverse events generally consistent with previously reported data
Bayer AG, of Leverkusen, Germany Vitrakvi (larotrectinib) TRK inhibitor Solid tumors Updated data published in The Lancet Oncology showed overall response rate (ORR) of 79%, including ORRs of 73% in adults, 92% in pediatric patients and 75% in those with brain metastases; median progression-free survival was 28.3 months
Bio-Thera Solutions Ltd., of Guangzhou, China BAT-1706 Biosimilar of Avastin (bevacizumab, Roche Holding AG) Nonsquamous non-small-cell lung cancer BAT-1706 and EU reference product had equivalent overall response rates
Bristol-Myers Squibb Co., of Princeton, N.J. Opdivo (nivolumab) PD-1-inhibiting antibody Previously treated advanced or metastatic renal cell carcinoma 5-year follow-up results from CheckMate -025 study show that treatment with Opdivo delivers superior overall survival (26% of patients alive compared to 18%) and objective response rates (23% for Opdivo vs. 4%) compared to those treated with everolimus
Exelixis Inc., of Alameda, Calif. Cabometyx (cabozantinib ) Kinase inhibitor Thyroid cancer Reported enrollment of the first 100 patients in Cosmic-311 pivotal trial in patients with radioactive iodine-refractory differentiated disease who have progressed after up to 2 VEGF receptor-targeted therapies; analysis of first 100 patients for the co-primary endpoint of objective response rate, and an interim analysis of progression-free survival expected in the second half of 2020
Isofol Medical AB, of Gothenburg, Sweden Arfolitixorin Folate-based therapy Metastatic colorectal cancer First patient in Japan initiated treatment in Agent study testing first-line treatment
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Anti-PD-1 therapy Metastatic triple-negative breast cancer Pivotal Keynote-355 trial in combination with chemotherapy nab-paclitaxel, paclitaxel or gemcitabine/carboplatin) met 1 of its dual primary endpoints of progression-free survival in patients whose tumors expressed PD-L1 (combined positive score [CPS] ?10); based on an interim analysis, first-line treatment demonstrated a statistically significant and clinically meaningful improvement in PFS compared to chemotherapy alone; data monitoring committee recommended trial continue without changes to evaluate the other dual primary endpoint of overall survival
Polyphor AG, of Allschwil, Switzerland Balixafortide CXCR4 chemokine antagonist Breast cancer Pivotal Fortress combination study with eribulin (Halaven, Eisai Ltd.) in HER2-negative, locally recurrent or metastatic disease randomized 192 (50%) of participants, with enrollment exceeding plan; full recruitment of 384 patients expected at end of third quarter 2020, with data cut for first co-primary endpoint of overall response rate confirmed for end of first quarter 2021; co-primary endpoint of progression-free survival expected in fourth quarter 2021
Soligenix Inc., of Princeton, N.J. SGX-301 Heat-shock protein inhibitor Cutaneous T-cell lymphoma Case study showed that participant with folliculotropic mycosis fungoides who completed Flash trial, including open-label cycles and 6-month follow-up, sustained complete clearance of disease for at least 4 years; top-line data from trial expected in first quarter 2020
Company Product Description Indication Phase III status

Cardiovascular

Akcea Therapeutics Inc., of Boston, and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. AKCEA-TTR-LRx Antisense drug Transthyretin-mediated amyloid cardiomyopathy Initiated Cardio-TTRansform cardiovascular outcomes study; primary composite endpoint is cardiovascular (CV) mortality and frequency of CV clinical events comparing 2 study arms at week 120.
Amgen Inc., of Thousand Oaks, Calif.; Cytokinetics Inc., of South San Francisco; and Servier SAS, of Suresnes, France Omecamtiv mecarbil Myosin stimulator Cardiac failure After second and final planned interim analysis, data monitoring committee recommended Galactic-HF trial that enrolled > 8,200 participants continue without changes; top-line results expected in fourth quarter of 2020
Cytokinetics Inc., of South San Francisco Omecamtiv mecarbil Myosin stimulator Heart failure with reduced ejection fraction Design of event-driven Galactic-HF cardiovascular outcomes trial published in Journal of American College of Cardiology: Heart Failure; study designed to enroll about 8,000 heart failure patients to evaluate whether study drug, dosed twice daily in addition to standard of care, reduces risk of heart failure events and cardiovascular death
Kowa Research Institute, of Research Triangle Park, N.C., part of Kowa Co. Ltd., of Nagoya, Japan Pemafibrate (K-877) Selective peroxisome proliferator activator receptor-alpha modulator Triglyceride reduction Achieved goal to randomize 10,000 subjects into Prominent cardiovascular outcomes trial to evaluate triglyceride reduction and increasing functional HDL in high-risk patients with diabetes with high triglyceride and low HDL-C levels who are already taking statins; study will test whether K-877 reduces occurrence of heart attacks, hospitalizations for unstable angina requiring unplanned revascularization, stroke or death from cardiovascular causes
United Therapeutics Corp., of Silver Spring, Md. Tyvaso (treprostinil) Prostacyclin analogue World Health Organization Group 3 pulmonary hypertension associated with interstitial lung disease In the Increase study, after 16 weeks of treatment, patients taking Tyvaso increased the distance they could walk in 6 minutes by 21 meters compared to placebo (p=0.0043); study also met all secondary endpoints; sNDA expected to be filed by mid-2020
Company Product Description Indication Phase III status

Dermatologic

Arcutis Pharmaceuticals Inc., of Westlake Village, Calif. ARQ-151 Phosphodiesterase type 4 inhibitor topical cream Plaque psoriasis Enrolled first patient in the open-label extension study, Dermis-OLE, testing safety of the drug for 24 weeks
Crescita Therapeutics Inc., of Laval, Quebec Topical dermatological agent Corticosteroid Plaque psoriasis 2 pivotal studies using candidate formulated with multiplexed molecular penetration enhancer technology met primary endpoint, with statistically significantly greater number of participants achieving investigator's global assessment treatment success (p< 0.001), based on intent-to-treat population
Foamix Pharmaceuticals Ltd., of Rehovot, Israel FMX-103 Tetracycline antibiotic Moderate to severe papulopustular rosacea Data published in the Journal of the American Academy of Dermatology from studies FX2016-11 and FX2016-12 showed the drug met both primary endpoints of absolute inflammatory lesion reduction and Investigator's Global Assessment treatment success at week 12; drug reduced inflammatory lesions compared to vehicle as early as week 4
Foamix Pharmaceuticals Ltd., of Rehovot, Israel FMX-103 (minocycline, 1.5% foam) Tetracycline antibiotic Rosacea Results from an integrated efficacy analysis of the 2 pivotal trials showed significantly greater reduction of inflammatory lesion counts from baseline to week 12 compared to vehicle (-18 vs. -14.9; p<0.001); significantly greater number of subjects receiving FMX-103 achieved IGA treatment success and at least a 2-grade improvement at week 12, (50.6% vs. 41%; p<0.001), respectively
Incyte Corp., of Wilmington, Del. Ruxolitinib cream Topical version of JAK1/2 inhibitor Atopic dermatitis Second pivotal study, True-AD1, met primary endpoint, showing more patients treated with ruxolitinib cream 0.75% (50% of patients) or 1.5% (53.8% of patients) achieved Investigator’s Global Assessment Treatment Success, with at least a 2-point improvement from baseline at week 8, than vehicle control (15.1% of patients; p < 0.0001 and p < 0.0001, respectively); 506% in 0.75% arm and 62.1% in 1.5% arm achieved at least a 75% improvement in EASI score vs. baseline, compared to 24.6% in vehicle control (p < 0.0001 and p < 0.0001, respectively)
Novan Inc., of Morrisville, N.C. SB-206 Nitric oxide-releasing topical gel Molluscum contagiosum Last patient completed final visit in the B-Simple program; 24-week safety data expected in March 2020
Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. Olumiant Baricitinib Moderate to severe atopic dermatitis Breeze-AD5 trial met the primary endpoint defined by the proportion of patients achieving at least a 75% or greater change from baseline in their Eczema Area and Severity Index at week 16
Vanda Pharmaceuticals Inc., of Washington Tradipitant Neurokinin-1 receptor antagonist Pruritus in atopic dermatitis Epione trial in adults did not meet its primary endpoint in reduction of pruritus across overall study population; antipruritic effect of tradipitant was robust in mild AD study population
Company Product Description Indication Phase III status

Endocrine/metabolic

Acasti Pharma Inc., of Laval, Quebec Capre Omega-3 phospholipid Severe hypertriglyceridemia Company identified some unexpected and inconsistent findings in Trilogy 1 study; plans to submit a request to meet with the FDA in the second quarter of 2020; unblinding of Trilogy 2 expected in the third quarter of 2020
Allena Pharmaceuticals Inc., of Newton, Mass. Reloxaliase Oxalate-degrading enzyme Hyperoxaluria Reached agreement in principle with the FDA on a streamlined design for Urirox-2, the second pivotal trial; study is designed to incorporate adaptive design elements that could allow for changes to sample size and duration of treatment based on accrued kidney stone (KS) disease progression rates and the conditional probability of achieving ultimate statistical success for KS disease progression in the long-term follow-up phase; Allena now plans to reduce the target enrollment from 400 subjects to 200 subjects to support a potential BLA filing for accelerated approval
Sanifit Therapeutics SA, of Palma, Spain SNF-472 (myo-inositol hexaphosphate) Calcification inhibitor Calciphylaxis First participant dosed in pivotal trial assessing effect of drug added to background care for individuals with end-stage kidney disease on treatment with hemodialysis; endpoints are improvement in wound healing, assessed by Bates-Jensen Wound Assessment Tool score, and pain, assessed by Visual Analog Scale
Company Product Description Indication Phase III status

Gastrointestinal

Genfit SA, of Lille, France Elafibranor Dual PPAR alpha/delta agonist Nonalcoholic steatohepatitis Unblinding of Resolve-IT data will be delayed to incorporate the latest FDA insights expected by the end of March
Janssen Pharmaceutical Cos., part of New Brunswick, N.J.-based Johnson & Johnson Stelara (ustekinumab) Human IL-12 and IL-23 antagonist Moderately to severely active Crohn’s disease Interim data from phase IIIb Stardust study showed at week 16, 79% of patients achieved clinical response and 67% were in clinical remission after receiving 1 ~6-mg/kg intravenous dose followed by 1 90-mg subcutaneous dose
Takeda Pharmaceuticals Ltd., of Osaka, Japan Entyvio (vedolizumab) Humanized monoclonal antibody designed to antagonize alpha4beta7 integrin Moderately to severely active Crohn’s disease Results from Visible 2 trial as maintenance therapy showed, at week 52, significantly more patients on vedolizumab SC vs. placebo were in clinical remission (48% vs. 34.3%, p=0.008),meeting primary endpoint; study evaluated patients achieving clinical response at week 6 following 2 doses of open-label intravenous induction therapy at weeks 0 and 2
Company Product Description Indication Phase III status

Genitourinary/sexual function

Myovant Sciences Inc., of Basel, Switzerland Relugolix Gonadotropin-releasing hormone receptor antagonist Uterine fibroids In the Liberty extension study testing relugolix plus estradiol and norethindrone acetate for 1 year, 87.7% of women responded, defined as a menstrual blood loss volume of less than 80 mL and a 50% or greater reduction from baseline in menstrual blood loss volume during the last 35 days of treatment measured using the alkaline hematin method; average reduction in menstrual blood loss from baseline at 1 year was 89.9%
Company Product Description Indication Phase III status

Immune

Abbvie Inc., of North Chicago Rinvoq (upadacitinib) JAK inhibitor Psoriatic arthritis Dosed at 15 mg and 30 mg once daily, drug met primary endpoint of ACR20 response at week 12 (71% and 79%, respectively) vs. placebo (36%) in adults with active disease (p<0.0001); 30-mg dose achieved superiority to adalimumab in ACR20 response at week 12 and both doses achieved noninferiority vs. adalimumab; both doses inhibited radiographic progression at week 24 compared to placebo (p<0.01)
Can-Fite Biopharma Ltd., of Petach Tikva, Israel Piclidenoson A3 adenosine receptor agonist Rheumatoid arthritis Completed enrollment of about 50% of the 525 patients planned for the Acrobat study; implementing an interim analysis
Company Product Description Indication Phase III status

Infection

Allecra Therapeutics GmbH, of Saint-Louis, France Exblifep (cefepime-enmetazobactam) Combination of extended-spectrum beta-lactamase inhibitor and fourth-generation cephalosporin Complicated urinary tract infections, including acute pyelonephritis Allium trial met FDA and EMA prespecified primary endpoints and demonstrated superiority over piperacillin-tazobactam; overall success was 79.1% for treatment vs. 58.9% for piperacillin-tazobactam
Citius Pharmaceuticals Inc., of Cranford, N.J. Mino-Lok Antibiotic lock solution Catheter-related bloodstream infections Trial randomized 72nd patient, passing halfway point for enrollment; next major milestone is 75% interim analysis, expected to be achieved in first half of 2020
Gilead Sciences Inc., of Foster City, Calif. Remdesivir Nucleotide analogue COVID-19 Started 2 trials to test safety and efficacy in adults diagnosed with the novel coronavirus; each trial to enroll about 1,000 patients at medical centers primarily across Asian countries
Polypid Ltd., of Petah Tikva, Israel D-Plex100 Prolonged and constant release of broad-spectrum antibiotic doxycycline Prevention of sternal wound infection post-cardiac surgery First patient enrolled in Shield trial; primary endpoint is the infection rate as measured by the proportion of subjects with a sternal wound infection event within 90 days post-sternotomy; trial will enroll a minimum of 1,284 subjects, with a maximum of about 1,600 subjects
Rebiotix SA, of Saint-Prex, Switzerland RBX-2660 Microbiota suspension Recurrent Clostridioides difficile Completed enrollment in pivotal trial
Scynexis Inc., of Jersey City, N.J. Ibrexafungerp 1,3 beta glucan synthase inhibitor Vulvovaginal candidiasis Enrollment in second pivotal study, Vanish-306, completed ahead of schedule; top-line data expected early in second quarter 2020, with NDA submission of oral ibrexafungerp on track for second half of 2020
Company Product Description Indication Phase III status

Inflammatory

Ampio Pharmaceuticals Inc., of Englewood, Colo. Ampion Low-molecular-weight fraction of 5% human serum albumin Osteoarthritis of the knee Data from the AP-013 trial showed that of the 1,034 patients to be enrolled, 875 patients had been dosed, with enrollment continuing to progress; exclusions for participation in the trial average about 35%
Company Product Description Indication Phase III status

Neurology/psychiatric

Adamas Pharmaceuticals Inc., of Emeryville, Calif. Gocovri Amantadine Parkinson’s disease Reported in the Journal of Parkinson’s Disease final results from 2-year open-label study that demonstrated a sustained improvement in levodopa-induced dyskinesia (LID) among patients using Gocovri (amantadine) extended-release capsules; results demonstrated the treatment effect on motor complications was maintained for at least 2 years; median treatment duration was 1.9 years
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Vutrisiran (ALN-TTRsc02) RNAi targeting TTR hATTR amyloidosis with polyneuropathy Completed enrollment in the HELIOS-A study
Axsome Therapeutics Inc., of New York AXS-07 (meloxicam + rizatriptan) Dual 5-HT 1b/1d receptor agonist; cyclooxygenase 2 inhibitor Migraine Enrollment of about 300 participants completed in Intercept study, with data expected in first quarter of 2020; co-primary endpoints are proportion of individuals free from headache pain and proportion who no longer suffer from their most bothersome migraine-associated symptom, both 2 hours after dosing
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Troriluzole Modulates glutamate Generalized anxiety disorder In the 402-patient study, drug produced a mean improvement change from baseline of -9.28 points on the Hamilton Anxiety Rating Scale total score, compared to -9.35 points for placebo (p=0.917)
Intra-Cellular Therapies Inc., of New York Caplyta (lumateperone) Serotonin 5-HT2A receptor antagonist Schizophrenia Results from the Caplyta trial published in JAMA Psychiatry showed Caplyta 42 mg met the primary endpoint and demonstrated antipsychotic efficacy with statistically significant superiority over placebo at week 4, met the key secondary endpoint of statistically significant improvement on the Clinical Global Impression Scale for Severity of Illness, and showed significant antipsychotic efficacy as early as week 1
Marinus Pharmaceuticals Inc., of Radnor, Pa. Ganaxolone Positive allosteric modulator of GABAA receptors CDKL5 deficiency disorder Reached 100-patient enrollment target for pivotal Marigold study in children and young adults; top-line results expected in third quarter of 2020
Minerva Neurosciences Inc., of Waltham, Mass. Roluperidone 5-HT 2a receptor antagonist; opioid receptor sigma antagonist 2 Schizophrenia Enrollment completed in pivotal trial assessing effect on negative symptoms in schizophrenia, with 515 participants randomized; top-line data expected in second quarter of 2020
Nono Inc., of Toronto Nerinetide PSD-95 antagonist Acute ischemic stroke Results from pivotal Escape-NA1 study in patients selected to undergo endovascular thrombectomy, showed nerinetide, without prior administration of alteplase, resulted in medically important improvements in functional independence, reduced mortality and reduced infarction volumes vs. placebo data; analysis of the primary endpoint of proportion of patients who achieve functional independence after 90 days across all trial participants (those not receiving alteplase and those who received alteplase before nerinetide) did not reach statistical significance; data published in The Lancet
PTC Therapeutics Inc., of South Plainfield, N.J., and Genentech, a unit of Roche Holding AG, of Basel Switzerland Risdiplam Survival motor neuron-2 splicing modifier Type 2 or 3 spinal muscular atrophy In part 2 of the Sunfish study, drug produced a 1.55-point mean difference in Motor Function Measure 32 (MFM-32) scale compared to placebo (p=0.0156); Revised Upper Limb Module improved by 1.59 points compared to placebo (p=0.0028); 78.1% of children ages 2-5 had a ?3 point increase in MFM-32 compared to 52.9% of children taking placebo; 57.1% of patients ages 18-25 had stabilization, defined as a ?0 point increase in MFM-32, compared to 37.5% of those on placebo
Teva Pharmaceutical Industries Ltd., of Jerusalem Austedo (deutetrabenazine) Vesicular monoamine transporter 2 inhibitor Tourette syndrome Artists 2 trial missed primary endpoint of reduction in motor and phonic tics vs. placebo as assessed by Total Tic Score of Yale Global Tic Severity Scale
Tonix Pharmaceuticals Holding Corp., of New York Tonmya (cyclobenzaprine, TNX-102 SL) Muscle relaxant Post-traumatic stress disorder Recovery study will stop enrollment after interim results found that Tonmya is unlikely to meet the primary endpoint of change in Clinician-Administered PTSD Scale for DSM-5 compared to placebo; unblinding expected in the second quarter after patients in the study continue until completion
Zogenix Inc., of Emeryville, Calif. Fintepla (ZX-008, fenfluramine oral solution) Inhibits activity of glutamate N-methyl-D-aspartate receptors Lennox-Gastaut syndrome Trial met its primary objective of demonstrating that Fintepla at a dose of 0.7 mg/kg/day was superior to placebo in reducing the frequency of drop seizures, based on the change between baseline and the titration and maintenance treatment period (p=0.0012); the same dose also demonstrated statistically significant improvements vs. placebo in key secondary efficacy measures, including the proportion of patients with a clinically meaningful reduction (?50%) in drop seizure frequency
Zynerba Pharmaceuticals Inc., of Devon, Pa. Zygel CBD Gel Cannabidiol Fragile X syndrome Completed enrollment in 14-week pivotal Connect-FX trial testing efficacy and safety for treating behavioral symptoms of fragile X in 210 children, ages 3-17, with full mutation FXS; top-line results expected in the second quarter of 2020; primary endpoint is change from baseline to the end of treatment period in the Aberrant Behavior Checklist-Community FXS Specific Social Avoidance subscale
Company Product Description Indication Phase III status

Ocular

Acucela Inc., of Seattle, a unit of Tokyo-based Kubota Pharmaceutical Holdings Co. Ltd. Emixustat hydrochloride Retinal pigment epithelium protein 65 inhibitor Macular atrophy secondary to Stargardt disease Study has enrolled 108 of a planned 162 patients, putting it on target to complete enrollment in 2020
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Eylea (aflibercept) Vascular endothelial growth factor inhibitor Moderately severe to severe nonproliferative diabetic retinopathy After 2 years of treatment in the Panorama study, Eylea reduced the likelihood of developing a vision-threatening complication or center-involved diabetic macular edema by 75% compared to the sham (nominal p<0.0001)
Regentree LLC, of Princeton, N.J., a joint venture of Gtreebnt Co. Ltd. and Regenerx Biopharmaceuticals Inc. RGN-259 Thymosin beta 4 ligand Dry eye syndrome Enrolled more than 350 patients of the over 700 expected patients in the ARISE-3 study
Company Product Description Indication Phase III status

Respiratory

Breath Therapeutics, a unit of Milan, Italy-based Zambon SpA Liposomal cyclosporine A for inhalation Immunosuppressive Bronchiolitis obliterans syndrome following lung transplantation Opened Boston-3 open-label extension study for patients who complete Boston-1 and -2 studies
Proteostasis Therapeutics Inc., of Boston Dirocaftor, posenacaftor and nesolicaftor CFTR potentiator, corrector and amplifier Cystic fibrosis Completed enrollment of 502 patients in the Choices study; ex vivo testing of patient-derived organoids is underway; clinical testing of patients whose organoids respond will begin in the second half of 2020
Company Product Description Indication Phase III status

Toxicity/intoxication

Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 Genetically targeted therapeutic agent Alcohol use disorder Started a phase III trial in Finland

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