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Phase III clinical data reported in December 2019

Company Product Description Indication Phase III status


Athenex Inc., of Buffalo, N.Y. Oral paclitaxel Chemotherapy Metastatic breast cancer In the 402-patient study, 35.8% of patients taking oral paclitaxel plus encequidar (Pac+E) had a confirmed tumor response compared to 23.4% of patients taking intravenous paclitaxel (IV Pac) (p=0.011); for population with evaluable post-baseline scan, confirmed response rates were 50.3% for Pac+E vs 29.6% for IV Pac (p=0.0005)
Aveo Oncology, a unit of Aveo Pharmaceuticals Inc., of Cambridge, Mass. Tivozanib (Fotivda) VEGF receptor tyrosine kinase inhibitor Renal cell carcinoma Data from TIVO-3 study, reported in September, were published in The Lancet Oncology
Beigene Co. Ltd., of Beijing Brukinsa (zanubrutinib) BTK inhibitor Waldenström’s macroglobulinemia In the Aspen study, relapsed or refractory patients had a very good partial response (VGPR) rate of 28.9% in the zanubrutinib arm and 19.8% in the ibrutinib arm (p=0.1160); overall population had VGPR rates of 28.4% for zanubrutinib and 19.2% for ibrutinib (p=0.0921)
Cstone Pharmaceuticals Ltd., of Suzhou, China, and Blueprint Medicines Corp., of Cambridge, Mass. Avapritinib Oral selective inhibitor of KIT and PDGFRA Advanced gastrointestinal stromal tumors Completed target patient enrollment in China and reached Voyager trial enrollment target globally; study testing drug as third- or fourth-line treatment vs. regorafenib
Diffusion Pharmaceuticals Inc., of Charlottesville, Va. TSC Trans sodium crocetinate Glioblastoma multiforme Data showed increased survival in inoperable patients enrolled in 19-patient lead-in portion of Intact study in combination with standard of care
Eli Lilly and Co., of Indianapolis Selpercatinib (LOXO-292) RET tyrosine kinase receptor inhibitor Non-small-cell lung cancer Opened Libretto-431 study, expected to enroll 400 individuals with advanced or metastatic treatment-naïve RET fusion-positive disease, randomized 1-to-1 to study drug or platinum-based and pemetrexed therapy with or without pembrolizumab (Keytruda, Merck & Co. Inc.); primary endpoint is progression-free survival and secondary endpoints include overall survival, overall response rate, duration of response and intracranial ORR
Eli Lilly and Co., of Indianapolis Selpercatinib (LOXO-292) RET tyrosine kinase inhibitor Medullary thyroid cancer Libretto-531 trial opened to assess drug vs. physician's choice of cabozantinib or vandetanib in 400 participants with advanced or metastatic treatment-naïve RET-mutant disease; efficacy endpoints are progression-free survival, treatment failure-free survival, overall survival, overall response rate and duration of response; crossover permitted at progression for those randomized to standard of care
Fergene LLC, subsidiary of Ferring Pharmaceuticals SA, of Saint-Prex, Switzerland Nadofaragene firadenovec
IFNA2 gene stimulator
Bladder cancer Study in 157 participants with high-grade non-muscle invasive disease met primary endpoint with 53% achieving complete response (CR) at 3 months and 24% continuing to show CR at 12 months; additional data showed 73% high-grade recurrence-free (HGRF) survival in those with papillary disease at 3 months and 44% HGRF survival at 12 months
Macrogenics Inc., of Rockville, Md. Margetuximab Monoclonal antibody targeting HER2 oncoprotein HER2-positive metastatic breast cancer Updated Sophia results showed overall survival data favored margetuximab in combination with chemotherapy vs. trastuzumab/chemotherapy in patients previously treated with HER2-targeted therapies in intention-to-treat population; however, those data did not reach statistical significance at second interim analysis as of September 2019 cut-off after 270 events (median OS=21.6 months vs. 19.8 months; p=0.326); final prespecified OS analysis planned after 385 events have accrued, which is projected in second half of 2020
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Monoclonal antibody targeting PD-1 First-line metastatic nonsquamous non-small-cell lung cancer with PD-L1 expression Exploratory analysis of the Keynote-042 study showed Keytruda reduced the risk of death by 58% in patients with any KRAS mutation and by 72% in patients with the KRAS G12C mutation compared to chemotherapy
Pellepharm Inc., unit of Bridgebio Pharma Inc., of San Francisco Patidegib topical gel Hedgehog protein inhibitor Gorlin syndrome Fully enrolled pivotal trial
Progenics Pharmaceuticals Inc., of New York PyL PSMA-targeted PET imaging agent Prostate cancer Condor study met its primary endpoint with a correct localization rate of 84.8% to 87% among the 3 blinded independent readers
Roche Holding AG, of Basel Switzerland Perjeta (pertuzumab) and Herceptin (trastuzumab) Monoclonal antibodies targeting HER2 HER2-positive early breast cancer Subcutaneous fixed-dose combination (FDC) of the drug was non-inferior for levels of Perjeta in the blood; 59.7% of patients receiving the FDC and 59.5% of patients treated with intravenous Perjeta and Herceptin achieved a total pathological complete response
Roche Holding AG, of Basel, Switzerland Perjeta (pertuzumab) HER2-targeting antibody HER2-positive early breast cancer Data from second interim overall survival analysis of Aphinity study in combination with Herceptin (trastuzumab) and chemotherapy, conducted after median follow-up of about 74 months, showed Perjeta-based regimen reduced risk of recurrence or death by 24% vs. Herceptin/chemotherapy/placebo; at 6 years, 90.6% of patients in Perjeta arm have not seen their cancer return vs. 87.8% in placebo arm, for an absolute benefit of 2.8%
Roche Holding AG, of Basel, Switzerland, and Exelixis Inc., of Alameda, Calif. Tecentriq (atezolizumab) Monoclonal antibody targeting PD-L1 Previously untreated BRAF V600 mutation-positive advanced melanoma In the Imspire150 study, Tecentriq (atezolizumab) plus Cotellic (cobimetinib) and Zelboraf (vemurafenib) improved progression-free survival compared to placebo plus Cotellic and Zelboraf; data to be presented at an upcoming medical meeting
Seattle Genetics Inc., of Bothell, Wash. Tucatinib Tyrosine kinase inhibitor selective for HER2 without significant EGFR inhibition HER2-positive metastatic breast cancer Data from pivotal HER2Climb study, published in The New England Journal of Medicine, showed addition of tucatinib to trastuzumab and capecitabine improved overall survival, reducing risk of death by 34% compared to trastuzumab and capecitabine alone; primary endpoint of progression-free survival showed addition of tucatinib was superior to trastuzumab and capecitabine alone, with a 46% reduction in risk of disease progression or death (p<0.00001); estimated PFS at 1 year was 33% in tucatinib arm vs. 12% in trastuzumab/capecitabine-only arm, and median PFS was 7.8 months and 5.6 months, respectively
Soligenix Inc., of Princeton, N.J. SGX-301 Synthetic hypericin Cutaneous T-cell lymphoma Completed enrollment in Flash study; top-line results expected in first quarter of 2020
Company Product Description Indication Phase III status


Akari Therapeutics plc, of New York Nomacopan C5 complement inhibitor Hematopoietic stem cell transplant-related thrombotic microangiopathy IND open; study to start in the first quarter of 2020; part A is a dose confirmation study; part B is a single arm responder-based efficacy study
Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis Jardiance (empagliflozin) Sodium glucose co-transporter-2 inhibitor Chronic heart failure with reduced and preserved ejection fraction In the Emperial-Reduced and Emperial-Preserved studies there were no significant changes in 6-minute walk test over 12 weeks between Jardiance and placebo; data to be presented in 2020
Mesoplast Ltd., of New York Revascor  Mesenchymal precursor cells Advanced chronic heart failure Study surpassed the number of primary endpoint events required for trial completion; last patient visits being scheduled for January 2020; data expected by mid-2020
Omeros Corp., of Seattle Narsoplimab Complement cascade inhibitor; MASP-2 inhibitor Hematopoietic stem cell transplant-associated thrombotic microangiopathy In pivotal trial, 56% who received at least 1 dose achieved complete responder status, meeting FDA-agreed response criteria; 68% who received protocol-specified dosing of at least 4 weeks achieved complete responder status; 100-day survival following diagnosis was 65% for those who received at least 1 dose, 81% for those who received at least 4 weeks of dosing and 93% in complete responder group; statistically significant (p < 0.01) improvements in platelet count, LDH and haptoglobin seen across all treated groups
Pluristem Therapeutics Inc., of Haifa, Israel PLX-PAD Allogeneic cells Critical limb ischemia Study has enrolled 75% of the planned 246 patients
Rexgenero Ltd., of London REX-001 (rexmyelocel-T) Autologous bone marrow-derived mature mononuclear cell therapy Chronic limb ischemia First U.K. trial site opened in 2 Salamander trials evaluating 60 participants with CLI and rest pain and 78 with CLI and non-healing ischemic ulcers
Company Product Description Indication Phase III status


Can-Fite Biopharma Ltd., of Petach Tikva, Israel Piclidenoson A3 adenosine receptor agonist Moderate to severe plaque psoriasis Comfort study enrolled more than 50% of the 407 planned patients
Leo Pharma A/S, of Ballerup, Denmark Tralokinumab Monoclonal antibody targeting IL-13 Moderate to severe atopic dermatitis Met all primary and secondary endpoints in 3 pivotal Ecztra studies in adults; marketing applications planned for 2020
MC2 Therapeutics A/S, of Copenhagen Wynzora Calcipotriene and betamethasone dipropionate Plaque psoriasis Treatment success, defined as a minimum 2-point decrease in the Physician Global Assessment score to clear or almost clear disease at week 8, was 52% for Wynzora (p<0.05 compared to Daivobet); Wynzora improved mPASI from baseline to week 8 greater than Daivobet (p<0.05); change in mPASI at week 4 was 68% for Wynzora and better than Daivobet (p<0.001)
Sol-Gel Technologies Ltd., of Ness Ziona, Israel Twyneo Combination of microencapsulated tretinoin 0.1% and microencapsulated benzoyl peroxide 3% cream Acne vulgaris Demonstrated statistically significant improvement on all co-primary endpoints, including proportion of patients who achieved at least a 2-grade reduction from baseline and clear (grade 0) or almost clear (grade 1) at week 12 on a 5-point Investigator Global Assessment scale; an absolute change from baseline in inflammatory lesion count at week 12; and an absolute change from baseline in non-inflammatory lesion count at week 12
UCB SA, of Brussels, Belgium Bimekizumab IL-17A and IL-17F inhibitor Moderate to severe plaque psoriasis Results from Be Sure study met co-primary endpoints at week 16, demonstrating superiority to adalimumab in achieving at least 90% improvement in Psoriasis Area and Severity Index (PASI 90) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1); met all ranked secondary endpoints with statistical significance, including superior total skin clearance at weeks 16 and 24, as measured by PASI 100, vs. adalimumab; bimekizumab was statistically superior to adalimumab in achieving rapid response, defined as PASI 75 at week 4
Company Product Description Indication Phase III status


Acasti Pharma Inc., of Laval, Quebec Capre Omega-3 phospholipid Severe hypertriglyceridemia Top-line results for Trilogy 1 pivotal trial expected in January due to unexpected delay in data processing and transfer from central testing laboratory to the statistical consultants for independent and external validation
Acer Therapeutics Inc., of Newton, Mass. ACER-001 Taste-masked, immediate-release formulation of sodium phenylbutyrate Urea cycle disorders Part B of the pivotal study comparing ACER-001 to Buphenyl is fully enrolled; study scheduled to be completed in the first quarter of 2020
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
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Lumasiran RNAi targeting glycolate oxidase Primary hyperoxaluria type 1 In the Illuminate-A study, drug improved 24-hour urinary oxalate excretion averaged across months 3 to 6 from baseline relative to placebo (p<0.0001); all 6 secondary endpoints were positive (p<0.001); plans to submit NDA and MAA in early 2020
Ardelyx Inc., of Fremont, Calif. Tenapanor Inhibits sodium hydrogen exchanger 3 Hyperphosphatemia in patients with chronic kidney disease on dialysis In the Phreedom study, treatment with tenapanor for 26 weeks produced a mean reduction in serum phosphorus of 2 mg/dL with 77% of the 408 patients experiencing a decline; in the 12-week placebo-controlled withdrawal period, drug produced a difference in least square mean serum phosphorus change of -1.4 mg/dL compared to placebo (p<0.0001)
Lexicon Pharmaceuticals Inc., of The Woodlands, Texas Sotagliflozin Inhibits sodium-glucose co-transporter types 1 and 2 Type 2 diabetes with inadequate glycemic control on a dipeptidyl peptidase 4 inhibitor with or without metformin Sotagliflozin reduced A1C at week 26 compared to placebo; drug was noninferior to empagliflozin on A1C reduction from baseline at week 26; data to be presented at a future medical meeting
Poxel SA, of Lyon, France, and Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan Imeglimin Tetrahydrotriazine Type 2 diabetes In the Times 2 study, imeglimin reduced HbA1c over 52 weeks as a monotherapy (0.46%) and when added on to various diabetes treatments (0.12%-0.92%)
Zealand Pharma A/S, of Copenhagen Dasiglucagon Glucagon analogue Congenital hyperinsulinism Trial opened to evaluate drug in up to 12 children with CHI ages 7 days to 1 year; endpoints include reduction of intravenous glucose infusion rate, number of hypoglycemic events and total amount of carbohydrate administered
Company Product Description Indication Phase III status


Intercept Pharmaceuticals Inc., of New York Obeticholic acid Farnesoid X receptor agonist Fibrosis Results from interim analysis of ongoing Regenerate study in participants with nonalcoholic steatohepatitis, reported in February, published in The Lancet; data served as basis for NDA, accepted by FDA in November
Phathom Pharmaceuticals Inc., of Buffalo Grove, Ill. Vonoprazan fumarate Potassium competitive acid blocker Erosive esophagitis Dosed first of expected 1,000 participants in pivotal Phalcon-EE trial assessing healing and maintenance of healing in EE and relief of heartburn
Company Product Description Indication Phase III status

Genitourinary/sexual function

Obseva SA, of Geneva Linzagolix GnRH receptor antagonist Heavy menstrual bleeding due to uterine fibroids. In the 535-patient Primrose 2 study, responder rate was 93.9% for the 200-mg dose with hormonal add-back therapy (ABT) 56.7% for the 100 mg without ABT, compared to 29.4% for the placebo group (p<0.001 for both); both doses also met secondary endpoints of amenorrhea (p<0.001), reduction in pain (p<0.001), improvement in quality of life (p<0.001) and improvement in hemoglobin levels (p<0.002)
Company Product Description Indication Phase III status


Akari Therapeutics plc, of London Nomacopan Complement C5 factor inhibitor; leukotriene BLT receptor antagonist Pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy Based on end-of-phase II meeting with FDA, single-arm responder-based pivotal study will be initiated, with treatment for up to 24 weeks; data from 7 participants in part A will be used to confirm dosing and endpoints for part B, which will begin recruiting following interim efficacy and safety readout from part A and meeting with FDA; primary endpoints are disease response defined by renal improvement and reduced transfusion dependence
Company Product Description Indication Phase III status


Adamas Pharmaceuticals Inc., of Emeryville, Calif. ADS-5102 Amantadine Multiple sclerosis with walking impairment In the 594-patient Inroads study, 21.1% of patients taking the 274 mg dose responded, defined as at least a 20% improvement in walking speed over 25 feet from baseline to 12 weeks post-treatment, compared to 11.3% of patients taking placebo (p=0.01); 17.6% of patients taking the 137 mg dose responded (0=0.08)
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
Medday Pharmaceuticals SAS, of Paris MD-1003 Formulation of high-dose pharmaceutical-grade biotin Primary and secondary progressive multiple sclerosis Last patient study visit occurred in the SPI2 study; top-line data expected by the end of the first quarter of 2020
Principia Biopharma Inc., of South San Francisco PRN-1008 Bruton tyrosine kinase inhibitor Pemphigus Final data expected in second half of 2021 rather than first half of 2022 due to accelerated enrollment time lines
Sichuan Clover Biopharmaceuticals Inc., of Chengdu, China SCB-808 (etanercept biosimilar) TNF antagonist/binding agent; type II TNF receptor modulator Ankylosing spondylitis First participant dosed in trial in China assessing efficacy, safety and pharmacokinetics of prefilled syringe formulation of drug via subcutaneous administration vs. originator drug
Xenikos BV, of Nijmegen, the Netherlands T-Guard Toxin-conjugated monoclonal antibodies that target CD3 and CD7 Steroid-refractory acute graft-versus-host disease First of 47 patients treated in the single-arm study; primary endpoint is complete response rate on day 28; secondary endpoints include duration of complete response, overall response rate and overall survival rate six months after treatment
Company Product Description Indication Phase III status


Contrafect Corp., of Yonkers, N.Y. Exebacase Lysin Staph aureus bacteremia, including right-sided endocarditis Started 350-patient Disrupt study comparing exebacase to placebo with both groups getting standard antibiotics; primary endpoint is clinical response at day 14 for patients with methicillin-resistant Staph aureus
Iterum Therapeutics plc, of Dublin Sulopenem Oral and I.V. penem antibiotic Complicated intra-abdominal infections Top-line results from Sure 3 trial showed the difference in clinical response at day 28 in micro-modified intent-to-treat population was 4.7% with a 95% confidence interval of -10.3% to 1%, just missing the noninferiority requirement set by the FDA that the lower limit of the difference in the outcome rates be >-10%
Iterum Therapeutics plc, of Dublin Sulopenem Broad-spectrum penem beta-lactam antibiotic Urinary tract infection Fully enrolled Sure 1 trial in uncomplicated UTIs; top-line data expected in first quarter of 2020
Moberg Pharma AB, of Stockholm MOB-015 (topical terbinafine) Squalene epoxidase inhibitor Mild to moderate toenail onychomycosis At 52 weeks, 4.5% of patients treated with drug achieved a complete cure compared to 0% of those receiving vehicle (p=0.019); 70% of patients treated with drug achieved a mycological cure, defined as both negative KOH and negative dermatophyte culture (p<0.0001); 15.4% of patients treated with drug achieved treatment success, defined as mycological cure and almost or completely clear great toenail (p= 0.0018)
Mycovia Pharmaceuticals Inc., of Durham, N.C. VT-1161 Antifungal Recurrent vulvovaginal candidiasis Completed enrollment of over 200 patients in the Ultraviolet study; top-line data expected in the second half of 2020
Phathom Pharmaceuticals Inc., of Buffalo Grove, Ill. Vonoprazan Potassium competitive acid blocker H. pylori infection Initiated pivotal Phalcon-HP trial to evaluate vonoprazan in combination with amoxicillin (vonoprazan dual therapy) and vonoprazan in combination with amoxicillin and clarithromycin (vonoprazan triple therapy); primary endpoint is the percentage of patients with successful eradication of H. pylori infection
Company Product Description Indication Phase III status


Bone Therapeutics SA, of Gosselies, Belgium JTA-004 Noncellular viscosupplement Knee osteoarthritis Submitted CTA for the 676-patient study comparing the reduction of pain for JTA-004, placebo and Hylan G-F 20; enrollment expected to begin in early 2020 with results in the second quarter of 2021
Optinose Inc., of Yardley, Pa. Xhance Corticosteroid Chronic sinusitis Data from the Exhance-3 study published in Rhinology showed Sinonasal Outcome Test-22 improved from baseline to 1 month by 20 points and 21 points in patients with and without nasal polyps, respectively; after 3 months, 48% of the patients who started the study with polyps had polyp elimination in at least one nostril
Selecta Biosciences Inc., of Waterton, Mass. SEL-212 ImmTOR + pegadricase; designed to control serum uric acid levels Chronic refractory gout Completed patient enrollment in the 6-month, head-to-head, phase II Compare clinical trial with 150 patients, comparing SEL-212 vs. a bi-weekly dose of pegloticase; primary endpoint is maintenance of serum uric acid levels of <6mg/dL at 3 and 6 months; top-line results are expected by mid-2020
Company Product Description Indication Phase III status


Biomarin Pharmaceutical Inc., of San Rafael, Calif. Vosoritide Targets NPR-B Achondroplasia Drug produced a placebo-adjusted change from baseline in growth velocity after one year of 1.6 cm/yr (p<0.0001)
Pluristem Therapeutics Inc., of Haifa, Israel PLX-PAD Allogeneic cells Muscle regeneration Completed enrollment of 50% of planned 240 patients; study treating muscle injury following arthroplasty for hip fracture, as compared to placebo
Stealth Biotherapeutics Inc., of Boston Elamipretide Mitochondrial-targeted compound Primary mitochondrial myopathy Mmpower-3 study did not meet its primary endpoints assessing changes in the six-minute walk test and Primary Mitochondrial Myopathy Symptom Assessment Total Fatigue Score
Tonix Pharmaceuticals Holding Corp., of New York TNX-102 SL (cyclobenzaprine HCl sublingual tablets) Analgesic Fibromyalgia Enrolled first of approximately 470 patients in the Relief study measuring daily diary pain severity score change from baseline to week 14
Company Product Description Indication Phase III status


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)
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
Avadel Pharmaceuticals plc, of Dublin FT-218 Once-nightly formulation of sodium oxybate Narcolepsy Completed enrollment of 212 patients in the Rest-On study; top-line data expected in the second quarter of 2020
Axsome Therapeutics Inc., of New York AXS-05 NMDA receptor antagonist Major depressive disorder In the 327-patient Gemini study, treatment with drug for 6 weeks reduced the Montgomery-Åsberg Depression Rating Scale total score by 16.6 points, compared to an 11.9-point reduction for placebo (p=0.002); rates of remission from depression were 39.5% for AXS-05 compared to 17.3% for placebo (p<0.001)
Axsome Therapeutics Inc., of New York AXS-07 (meloxicam + rizatriptan) Dual 5-HT 1b/1d receptor agonist; cyclooxygenase 2 inhibitor Migraine Momentum trial achieved statistical significance on regulatory co-primary endpoints of pain freedom (p<0.001) and freedom from most bothersome symptom (p=0.002) at 2 hours, compared to placebo and showed superiority to rizatriptan active comparator on key secondary endpoint of sustained pain freedom 2 to 24 hours after dosing (p=0.038); study drug also showed greater and more sustained migraine pain relief (p=0.006) and reduced use of rescue medication (p<0.001), compared to rizatriptan
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 Alpha 2A adrenoceptor agonist Agitation Serenity program initiated in individuals with schizophrenia and bipolar disorder; primary endpoint of 2 adaptive trials of up to 750 individuals is change in acute agitation measured by Positive and Negative Syndrome Scale, examining Excited Component change from baseline, compared to placebo; top-line data expected in mid-2020
Eisai Inc., of Woodcliff Lake, N.J. Fycompa (perampanel) AMPA glutamate receptor antagonist Partial-onset seizures In the 91-patient Freedom study, seizure freedom rates at 26 weeks were 64.6% and 77% for 4-mg dose and combined 4 mg plus 8 mg, respectively; in the 85-patient Fame study, after a 24-week maintenance period, 50% responder rate was 80%, 75% responder rate was 71.8% and seizure freedom rate was 47% by adjunctive anti-epileptic drug use
Greenwich Biosciences Inc., of Carlsbad, Calif, a subsidiary of GW Pharmaceuticals plc Epidiolex (cannabidiol) Duel cannabinoid CB1/CB2 receptor modulator Seizures associated with tuberous sclerosis complex Treatment with 25 mg/kg/day and 50 mg/kg/day reduced seizures by 49% and 48%, respectively, compared to a 27% reduction for placebo (p=0.0009 and p=0.0018, respectively); 36% and 40% of patients, respectively, had a 50% or greater reduction in seizures compared to 22% of patients taking placebo (p=0.0692 and p=0.0245, respectively)
Intra-Cellular Therapies Inc., of New York Lumateperone Serotonin 5-HT2A receptor antagonist Bipolar depression In Study 404, compared to placebo, lumateperone improved Montgomery-Åsberg Depression Rating Scale at week 6 by an effect size (ES) of 0.56 (p<0.0001); compared to placebo, drug also improved Clinical Global Impression Scale for Bipolar for Severity of Illness (CGI-BP-S) Total Score (p<0.001; ES = 0.46) and CGI-BP-S Depression Score (p<0.001; ES = 0.50)
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)
Supernus Pharmaceuticals Inc., of Rockville, Md. SPN-810 5-HT2B receptor antagonist Impulsive aggression in attention deficit hyperactivity disorder Additional analysis of P301 study showed excluding patients with a baseline score of 6 episodes or less per week resulted in a median percent reduction of 59.2% in the average weekly frequency of impulsive aggression episodes from baseline (p=0.017)
Zogenix Inc., of Emeryville, Calif. Fintepla (ZX-008, fenfluramine oral solution) Inhibits activity of glutamate N-methyl-D-aspartate receptors Dravet syndrome Results published in JAMA Neurology showed adding Fintepla treatment to patients experiencing high number of convulsive seizures despite antiepileptic regimens including stiripentol led to significant and clinically meaningful (> 50%) reduction in monthly convulsive seizure frequency
Company Product Description Indication Phase III status


Aldeyra Therapeutics Inc., of Lexington, Mass. Reproxalap Inhibitor of reactive aldehyde species Moderate to severe dry eye disease In part 1 of Renew study of 422 total patients, drug improved patient-reported visual analogue scale (VAS) ocular dryness from weeks 2 to 12 (p=0.0004); reproxalap was statistically superior to vehicle in VAS ocular endpoints for itching (p=0.03), foreign body sensation (p=0.004), discomfort (p=0.003), photophobia (p=0.004) and pain (p=0.03) and on the Ocular Discomfort & 4-Symptom Questionnaire ocular endpoints for dryness (p=0.01), discomfort (p=0.03), burning (p=0.03), grittiness (p=0.003) and stinging (p=0.02); fluorescein nasal region ocular staining wasn’t statistically significant for reproxalap compared to placebo
Aldeyra Therapeutics Inc., of Lexington, Mass. ADX-2191 Inhibits cell growth diminishing scar formation To prevent proliferative vitreoretinopathy Enrolled the first patient in the phase III Guard trial; ADX-2191 has fast-track designation and orphan drug designation in the U.S.
Eyegate Pharmaceuticals Inc., of Waltham, Mass. Ocular bandage gel (OBG; dexamethasone, iontophoresis delivery) Glucocorticoid receptor agonist Postoperative pain and inflammation Additional data from pivotal study in post-photorefractive keratectomy showed OBG prompted more rapid corneal wound closure than standard-of-care bandage contact lens (BCL); at day 2, average wound size for eyes treated with OBG was 3.61 mm2 vs. 6.66 mm2 for those treated with BCL
Ocugen Inc., of Malvern, Pa. OCU-300 Ophthalmic nanoemulsion of brimonidine tartrate Ocular graft-vs.-host disease Study reached 50% enrollment of the 60 planned patients; top-line results expected in the second half of 2020
Company Product Description Indication Phase III status


Kamada Ltd., of Rehovot, Israel AAT Alpha-1 antitrypsin Alpha-1 antitrypsin deficiency First of up to 250 patients enrolled in the InnovAATe study testing FEV1 over 2 years
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
Company Product Description Indication Phase III status


Novartis AG, of Basel, Switzerland QMF-148 Once-daily, fixed-dose combination of indacaterol acetate and mometasone furoate Asthma Data from 52-week Palladium trial showed superiority to mometasone furoate (MF) at medium and high doses in improving lung function, meeting primary endpoint; statistically significant superiority vs. MF alone also demonstrated in key secondary endpoint of improvement in asthma control; other secondary analyses showed improvements in lung function when comparing IND/MF to a LABA/ICS standard of care (salmeterol xinafoate/fluticasone propionate)
Novartis AG, of Basel, Switzerland Fevipiprant (QAW-039) DP2 receptor antagonist Moderate-to-severe asthma Pooled data from the Luster-1 and Luster-2 studies didn't meet the clinically relevant threshold for reduction in rate of moderate-to-severe exacerbations compared to placebo over the 52-week treatment period for either of the doses; data to be published at an upcoming medical meeting
Company Product Description Indication Phase III status


Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 Genetically targeted therapeutic agent Alcohol use disorder Completed validation of the genetic biomarker test to be used in the study; in process of obtaining regulatory approvals in Europe to start study

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