Company Product Description Indication Status
Phase I
Alligator Bioscience AB, of Lund, Sweden ATOR-1017 Activates 4-1BB receptors Metastasized cancer First of approximately 50 patients dosed in study designed to test the safety and tolerability of the drug and to determine the recommended phase II dose
Inmune Bio Inc., of La Jolla, Calif. INB-03 TNF inhibitor Advanced solid tumors Drug was well-tolerated; IL-6 decreased by more than 50% in half of the patients; selected 1 mg/kg as dose for phase II testing; next study in trastuzumab-resistant, HER2+ breast cancer
National Eye Institute, of Bethesda, Md. iPSC-derived RPE implant Retinal pigment epithelial cells from induced pluripotent stem cells Advanced-stage geographic atrophy Started 12-patient study testing the safety of the therapy
Tonix Pharmaceuticals Holding Corp., of New York TNX-601 CR Controlled-release formulation of tianeptine, a modulator of the glutamatergic system Healthy volunteers (eventually major depressive disorder and post-traumatic stress disorder) Drug was well-tolerated; plans to run an efficacy study outside the U.S. in 2020
Phase II
Alkahest Inc., of San Carlos, Calif. AKST-4290 CCR3 inhibitor Neovascular age-related macular degeneration Started 15-patient, open-label study measuring changes in choroidal blood flow
Antisense Therapeutics Ltd., of Melbourne, Australia ATL-1102 Antisense inhibitor of CD49d expression Duchenne muscular dystrophy Positive mean change of 0.9 in the PUL2.0 scores from baseline with 7 of the 9 patients having increases or no change; Myogrip and Myopinch improved; plans to run a phase IIb study following meetings with regulators
Basilea Pharmaceutica Ltd., of Basel, Switzerland Lisavanbulin (BAL-101553) Binds to the colchicine site of tubulin Recurrent glioblastoma and platinum-resistant ovarian cancer Across 2 studies, 2 glioblastoma patients responded; 4 of 9 ovarian cancer patients had tumor reduction that didn't meet response criteria; plans to run a biomarker-driven expansion study in mid-2020
Bellerophon Therapeutics Inc., of Warren, N.J. Inopulse Nitric oxide delivery system Pulmonary hypertension associated with interstitial lung disease Cohort 2 treated at 45 mcg/kg IBW/hr had an improvement in moderate to vigorous physical activity of 14 minutes per day (p=0.02); overall activity improved by 100 counts/min; St. George Respiratory Questionnaire (SGRQ) Total score improved by 3 points; SGRQ Activity score improved by 5 points; SGRQ Impacts score improved by 6 points; University of California, San Diego Shortness of Breath Questionnaire improved by 5 points
Biohaven Pharmaceutical Holding Company Ltd., of New Haven, Conn. Vazegepant CGRP receptor antagonist Acute treatment of migraine At 2 hours, 22.5% and 23.1% of patients treated with 10 mg and 20 mg of vazegepant, respectively, were pain free compared to 15.5% of patients treated with placebo (p=0.0113 and p=0.0055, respectively); 41.9% and 42.5% of patients treated with 10 mg and 20 mg of vazegepant, respectively, were free of their most bothersome symptom, compared to 33.7% of placebo-treated patients (p=0.0155 and p=0.0094, respectively)
Flexion Therapeutics Inc., of Burlington, Mass. Zilretta (triamcinolone acetonide) Extended release corticosteroid  Shoulder osteoarthritis or adhesive capsulitis First 3 patients enrolled in the Range study testing the overall change in daily shoulder pain with movement score from baseline to 8 weeks post injection; study expected to be complete in the first half of 2021
Glaxosmithkline plc, of London Belantamab mafodotin (GSK-2857916) Antibody-drug conjugate targeting B cell maturation antigen Heavily pre-treated multiple myeloma Data from the Dreamm-2 study published in The Lancet Oncology showed the drug produced an overall response rate of 31% in 97 patients
Pear Therapeutics Inc., of Boston Pear-006 Digital therapeutic Relapsing multiple sclerosis Treated first of approximately 30 patients in the 8-week study testing dosing, patient use, engagement and other clinical outcomes
Proteostasis Therapeutics Inc., of Boston Dirocaftor (PTI-808), posenacaftor (PTI-801) and nesolicaftor (PTI-428) CFTR modulators Cystic fibrosis Triple combination improved ppFEV1 by 8 percentage points compared to placebo and produced a mean improvement of -29 mmol/L in sweat chloride at day 28 in 28 F508del homozygous patients; in 40 F508del heterozygous patients, changes in sweat chloride concentration were statistically significant (p<0.01), but mean changes in ppFEV1 were not statistically significant
Phase III
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
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)
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
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
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
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
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
Pluristem Therapeutics Inc., of Haifa, Israel PLX-PAD  Allogeneic cells  Critical limb ischemia Study has enrolled 75% of the planned 246 patients
Phase IV
Mallinckrodt plc, Staines-upon-Thames, U.K. Acthar Gel Hormone  Severe keratitis Enrolled first of 30 patients in study testing improvement on the Impact of Dry Eye on Everyday Life (IDEEL) scale and IDEEL symptom bother scale at 12 weeks


For more information about individual companies and/or products, see Cortellis.

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