Company (location) |
Product |
Description |
Indication |
Status |
Date |
Autoimmune | |||||
Aurinia Pharmaceuticals Inc. (Victoria, British Columbia) |
Voclosporin |
Calcineurin inhibitor |
Lupus nephritis |
Additional data from its phase IIb AURA-LV study showed that statistically improved efficacy over the control arm previously shown at 24 and 48 weeks was attained while maintaining stable serum magnesium, potassium and blood pressure levels; voclosporin-treated groups showed statistically significant improvement in speed and rates of complete and partial remission (CR); of the low-dose voclosporin patients who achieved CR at 24 weeks, 100% remained in CR at 48 weeks |
6/6/17 |
Aurinia Pharmaceuticals Inc. (Victoria, British Columbia) |
Voclosporin |
Calcineurin inhibitor |
Lupus nephritis |
Duration of remission data from its phase IIb AURA-LV study showed that patients on voclosporin stayed in remission approximately twice the amount of time as those in the control group; treatment with low-dose voclosporin showed statistically improved efficacy over the control arm at both 24 and 48 weeks, with a doubling of remission rates at 48 weeks vs. the control arm (49% vs. 24%); of the low-dose voclosporin patients that achieved complete remission (CR) at 24 weeks, 100% remained in CR at 48 weeks |
6/19/17 |
Biopharmx Corp. (Menlo Park, Calif.) |
BPX-01 |
1% and 2% minocycline |
Moderate to severe inflammatory, non-nodular acne vulgaris |
Phase IIb data showed it reduced the number of inflammatory lesions in acne patients by 59% vs. 44% in vehicle with no serious drug-related adverse side effects experienced, with data also suggesting it may lessen the severity of lesions |
6/2/17 |
Biopharmx Corp. (Menlo Park, Calif.) |
BPX-01 |
Topical hydrophilic gel formulation of the minocycline |
Acne vulgaris |
Data of a previously disclosed phase IIb trial showed it reduced acne lesions in subjects by 59%, compared to 44% for placebo, and BPX-01 produced at least a two-grade reduction in investigator global assessment with clear to almost clear in 25 percent of patients |
6/23/17 |
Celgene International Sàrl (Boudry Switzerland; subsidiary of Celgene Corp.) |
CC-220 |
Oral immunomodulatory compound that binds to and modulates cereblon |
Systemic lupus erythematosus |
Results from the phase IIa SLE-001 trial showed the Cutaneous Lupus Area and Severity Activity Index (CLASI) score was reduced by 4.3 to 7.8 points in the CC-220 treatment groups, compared to a 0.4-point increase in the CLASI score in patients treated with placebo; between 22.2% and 50% of patients treated with CC-220 had a four-point or more reduction in their SELENA-SLEDAI score, compared to just 12.5% of patients treated with placebo |
6/15/17 |
Chemocentryx Inc. (Mountain View, Calif.) |
Avacopan (CCX-168) |
Complement 5a receptor inhibitor |
IgA nephropathy |
Results from an open-label phase II pilot study showed that three of the seven patients had significant improvement to the protein-to-creatinine ratio < 1 g/g; at the end of the 12-week period following treatment, the protein ratio in two of those three patients returned to baseline levels, while the improvement was maintained in the third |
6/7/17 |
Devonian Health Group Inc. (Quebec City) |
Thykamine |
Developed using Devonian's Supra Molecular Complex Extraction and Stabilisation Technology (SUPREX) |
Mild to moderate atopic dermatitis |
Plans to begin a phase IIa study at the end of the month |
6/23/17 |
Pharmalink AB (Stockholm) |
Nefecon |
Targeted-release formulation of the corticosteroid budesonide |
Primary IgA nephropathy |
Phase IIb trial data showed it met its primary endpoint and concluded that the use of Nefecon, in addition to standardized rigorous blood pressure control (optimized RAS blockade), reduced proteinuria and stabilized estimated glomerular filtration rate (eGFR) in patients at risk of developing end-stage renal disease |
6/6/17 |
Selecta Biosciences Inc. (Watertown, Mass.) |
SEL-212 |
SVP-rapamycin in combination with uricase enzyme pegsiticase |
Chronic severe gout |
Data from its ongoing phase II trial showed a correlation between the prevention of antidrug antibodies and the maintenance of pegsiticase activity and serum uric acid (sUA) control; in the control cohorts receiving pegsiticase alone, within the first month of treatment, 50% of patients reported experiencing a gout flare, compared to 15% of patients receiving SEL-212 |
6/16/17 |
Cancer | |||||
Acceleron Pharma Inc. (Cambridge, Mass.) |
Dalantercept |
Protein therapeutic that prevents BMP9 from interacting with activin receptor-like kinase 1 |
Advanced renal cell carcinoma |
The DART phase II study of dalantercept plus axitinib did not achieve its primary endpoint, to demonstrate a statistically significant increase in progression-free survival (PFS); median PFS for dalantercept plus axitinib was 6.8 months vs. 5.6 months for placebo plus axitinib; in the key secondary endpoint of PFS for patients who received two or more prior systemic cancer therapies, median PFS for dalantercept plus axitinib was 8.1 months vs. seven months for placebo plus axitinib; the confirmed objective response rate (ORR) for dalantercept plus axitinib was 19% vs. 25% for the placebo arm; the company decided to halt development of dalantercept |
6/14/17 |
Aduro Biotech Inc. (Berkeley, Calif.) |
CRS-207 |
LADD-based immunotherapy |
Malignant pleural mesothelioma |
The first patient was dosed in its phase II trial testing CRS-207 in combination with PD-1 inhibitor Keytruda (pembrolizumab, Merck & Co. Inc.) |
6/30/17 |
Advaxis Inc. (Princeton, N.J.) |
Axalimogene filolisbac |
Targeted Listeria monocytogenes-based immunotherapy |
Persistent or recurrent metastatic carcinoma of the cervix |
Advaxis researchers identified pre-treatment baseline levels of serum proteins strongly associated with overall survival (OS) benefit in patients who were treated with axalimogene filolisbac in the phase II GOG/NRG-0265 study, which had a 12-month survival rate of 38% (19/50); analysis of pre-treatment baseline serum proteins of 45 patients from the study showed that levels of a group of four closely aligned proteins were strongly correlated with OS; one patient cluster (n=25) had relatively lower levels of the proteins and exhibited an OS of 56%, while the second cluster (n=20) exhibited an OS of 15%; the data were statistically significant (HR=0.23; 95% CI: 0.10-0.48; P<.001) and suggested that baseline levels of these analytes have prognostic value for OS |
6/27/17 |
Aslan Pharmaceuticals Pte. Ltd. (Singapore) |
Varlitinib |
Small-molecule pan-HER inhibitor |
Biliary tract cancer |
Enrolled the first patient in its phase II/III TreeTopp (Treatment Opportunity with varlitinib in biliary tract cancer) study |
6/30/17 |
Aveo Oncology Inc. (Cambridge, Mass.) |
Tivozanib |
VEGF tyrosine kinase inhibitor |
Advanced renal cell carcinoma |
Its phase I/II TiNivo trial testing tivozanib in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.) has moved into the phase II portion |
6/9/17 |
Bayer AG (Leverkusen, Germany) |
Copanlisib |
Pan-class I phosphatidylinositol 3-kinase (PI3K) inhibitor |
Relapsed or refractory diffuse large B-cell lymphoma (DLBCL) |
For patients in the per-protocol sets of the DLBCL study, the objective tumor response rate was 25%, with five complete responses and five partial responses; stable disease was seen in 12 patients |
6/9/17 |
Bayer AG (Leverkusen, Germany) |
Copanlisib |
Intravenously administered pan-class I phosphatidylinositol 3-kinase (PI3K) inhibitor |
Relapsed or refractory indolent B-cell lymphoma |
Data from the CHRONOS-1 trial showed the 142 treated patients had a median duration of treatment of 22 weeks (range 1-105) at the time of analysis; the overall response rate was 59.2%, including 12% complete response and 47.2% partial response, with 29.6% stable disease and 2.1% progressive disease; tumor shrinkage as best response was observed in 91% of evaluable patients |
6/16/17 |
Bristol-Myers Squibb Co. (New York) |
Opdivo |
Nivolumab |
Relapsed or progressed classical Hodgkin lymphoma |
Extended follow-up data from the phase II CheckMate -205 study in patients with cHL after autologous stem cell transplant (ASCT) showed that, in 63 patients who hadn't received Adcetris (brentuximab vedotin, Seattle Genetics Inc.) previously, Opdivo produced an objective response rate (ORR) of 65%, with a complete response (CR) in 29% of patients; in 80 patients who received Adcetris after ASCT, the ORR was 68%, with CR in 13% of patients; the median DOR was 16 months and the median PFS was 14.7 months in those patients; in a third cohort of 100 patients who received Adcetris before, after, or both before and after ASCT, the ORR was 73%, with CR in 12% of patients |
6/19/17 |
Enzychem Lifesciences Corp. (Daejeon, South Korea) |
EC-18 |
Mosedipimod; synthetic palmitoyl-2-linoleoyl-3-acetyl-rac-glycerol |
Chemotherapy-induced neutropenia |
Initiated the dose-escalation stage of a global phase II trial |
6/20/17 |
Epizyme Inc. (Cambridge, Mass.) |
Tazemetostat |
EZH2 inhibitor |
Relapsed or refractory follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) |
Interim data from the ongoing phase II trial showed that 92% of patients with relapsed or refractory FL and EZH2-activating mutations saw their tumors shrink; as of the June 1 data cut-off, 26% of people with FL and wild-type (WT) EZH2 enrolled in the trial had seen their tumors shrink; there were also four complete responses among the FL patients enrolled in the trial; among DLBCL patients with EZH2 mutations, 29% saw their tumors get smaller |
6/15/17 |
Exelixis Inc. (South San Francisco) |
Cabometyx |
Cabozantinib |
Previously untreated advanced renal cell carcinoma |
A review by a blinded independent radiology review committee confirmed the primary efficacy endpoint results of investigator-assessed progression-free survival from the 157-patient CABOSUN phase II trial comparing Cabometyx to Sutent (sunitinib, Pfizer Inc.) |
6/20/17 |
Galera Therapeutics Inc. (Malvern, Pa.) |
GC-4419 |
Dismutase mimetic |
To reduce the incidence and duration of oral mucositis in head and neck cancer patients receiving chemoradiation therapy |
Completed enrollment in its phase IIb study |
6/19/17 |
Helsinn Group (Lugano, Switzerland) and Mei Pharma Inc. (San Diego) |
Pracinostat |
Oral histone deacetylase inhibitor |
Higher-risk myelodysplastic syndromes |
The first patient was dosed in a phase II dose-optimization study of pracinostat in combination with azacitidine in patients previously untreated with hypomethylating agents |
6/15/17 |
Kura Oncology Inc. (La Jolla, Calif.) |
Tipifarnib |
Inhibitor of protein farnesylation |
Peripheral T-cell lymphoma |
Preliminary phase II data indicate that it has antitumor activity; the results identify the CXCL12/CXCR4 pathway as a potential target of tipifarnib; among the 18 patients in stages 1 and 2 of the trial, three achieved a partial response (PR), and two of the PRs occurred in the two patients on study with angioimmunoblastic T-cell lymphoma |
6/15/17 |
Lion Biotechnologies Inc. (San Carlos, Calif.) |
LN-145 |
Adoptive cell transfer therapy that uses an autologous TIL manufacturing process |
Recurrent and/or metastatic squamous cell carcinoma of the head and neck |
The first patient was dosed in a phase II trial |
6/5/17 |
Medicenna Therapeutics Corp. (Toronto) and Brainlab |
MDNA-55 |
Targeted immunotherapy agent |
Recurrent glioblastoma |
Patients in the phase IIb trial have been treated using drug delivery technology from Brainlab; investigators used convection enhanced delivery to inject MDNA-55, together with an imaging agent, directly into the tumor |
6/9/17 |
Merrimack Pharmaceuticals Inc. (Cambridge, Mass.) |
MM-141 |
Istiratumab; bispecific antibody targeting both the IGF-1 receptor and the HER3 receptor |
Metastatic pancreatic cancer |
Enrolled the last patient in its phase II CARRIE study testing MM-141 in combination with Abraxane (nab-paclitaxel, Celgene Corp.) /gemcitabine chemotherapy compared to placebo plus chemotherapy in previously untreated patients |
6/20/17 |
Modra Pharmaceuticals BV (Amsterdam) |
ModraDoc-006/r |
Oral formulation of docetaxel |
Metastatic castration-resistant prostate cancer |
The first patients in its phase II trial have been treated |
6/15/17 |
Molecular Partners AG (Zurich, Switzerland) |
MP-0250 |
Darpin candidate designed to hit both VEGF and HGF |
Multiple myeloma |
The first patient was dosed in the phase II study |
6/8/17 |
Newlink Genetics Corp. (Ames, Iowa) |
Indoximod |
Indoleamine-(2,3)-dioxygenase pathway inhibitor |
Metastatic breast cancer |
Top-line data from a phase II trial of indoximod in combination with taxane chemotherapy did not meet its pre-specified endpoints of a statistically significant difference between the two treatment groups as to progression-free survival, overall survival or objective response rate |
6/6/17 |
Newlink Genetics Corp. (Ames, Iowa) |
Indoximod |
IDO pathway inhibitor |
Metastatic breast cancer |
Reported a failed phase II experiment with indoximod, in combination with taxane chemotherapy |
6/9/17 |
Novartis AG (Basel, Switzerland) |
CTL-019 |
Tisagenlecleucel |
Relapsed or refractory diffuse large B-cell lymphoma |
An interim analysis of its multicenter phase II JULIET study showed a three-month overall response rate (ORR) of 45%, with 37% achieving a complete response (CR) and 8% achieving a partial response (PR); among 51 patients with three months or more of follow-up or earlier discontinuation, best ORR was 59% (95% CI, 44.2-72.4; p<0.0001), with 43% achieving CR and 16% achieving PR, and 57% of treated patients experienced any grade cytokine release syndrome (CRS) and 26% experienced grade 3/4 CRS (17% grade 3; 9% grade 4) using the Penn Grading Scale |
6/8/17 |
Novocure Ltd. (St. Helier, Jersey) |
Optune |
Tumor Treating Fields delivery system |
Bevacizumab-refractory recurrent glioblastoma |
The first patient was enrolled in the RTOG Foundation's phase II trial testing Optune with Avastin (bevacizumab, Roche Holding AG) |
6/9/17 |
Oncbiomune Pharmaceuticals Inc. (Baton Rouge, La.) |
Proscavax |
Therapeutic cancer vaccine |
Early stage prostate cancer |
The protocol was submitted to the FDA for its upcoming phase II trial |
6/2/17 |
Oxford Biomedica plc (Oxford, U.K.) |
CTL-019 |
Tisagenlecleucel-T); chimeric antigen receptor T cell therapy |
Relapsed and refractory diffuse large B-cell lymphoma |
Findings by Novartis AG, of Basel, Switzerland, from the phase II JULIET trial showed it met its primary objective at interim analysis; data showed a three-month overall response rate (ORR) of 45%, with 37% achieving a complete response (CR) and 8% achieving a partial response (PR), respectively; among 51 patients with three months or more of follow-up or earlier discontinuation, best ORR was 59% (p<0.0001) with 43% achieving CR and achieving 16% PR; CR and PR rates at three months were 37% and 8%, respectively |
6/15/17 |
Soligenix Inc. (Princeton, N.J.) |
SGX942 |
Dusquetide; innate defense regulator |
Oral mucositis in patients with head and neck cancer |
Final results from its phase II trial showed it reduced the median duration of severe oral mucositis by 50% compared to placebo in all patients and by 67% compared to placebo in patients receiving the most aggressive chemoradiation therapy for treatment of their head and neck cancer |
6/20/17 |
Sophiris Bio Inc. (San Diego) |
PRX-302 |
Topsalysin |
Clinically significant localized prostate cancer |
The first patient was dosed in its phase IIb trial |
6/9/17 |
Tapimmune Inc. (Jacksonville, Fla.) |
TPIV-200 |
T-cell vaccine candidate |
Triple-negative breast cancer |
Enrolled 50% (40/80) of the patients in its phase II study |
6/28/17 |
Tolero Pharmaceuticals Inc. (Salt Lake City) |
Alvocidib |
Inhibitor of cyclin-dependent kinase 9 |
MCL-1-dependent relapsed/refractory acute myeloid leukemia |
Is adding European centers to its ongoing phase II study |
6/22/17 |
Vascular Biogenics Ltd. (Tel Aviv, Israel; operating as VBL Therapeutics) |
VB-111 |
Ofranergene obadenovec |
Recurrent glioblastoma (rGBM), recurrent platinum-resistant ovarian cancer and radioiodine refractory differentiated thyroid cancer |
Updated data from three completed phase II trials showed that rGBM patients treated with VB-111 had a 12 month survival rate of 54% compared to 23% of patients who had limited exposure of a therapeutic dose of VB-111; in ovarian cancer, 53% of patients treated with VB-111 in combination with paclitaxel were alive at 15 months while no patients in the sub-therapeutic dose were alive at the 15-month time point; in patients with thyroid cancer, 53% of those who received multiple doses of VB-111 were alive at 24 months, compared to 33% of those who received a single, sub-therapeutic dose of VB-111 |
6/20/17 |
Vaxon Biotech SA (Paris) |
Vx-001 |
Therapeutic cancer vaccine based on optimized cryptic tumor peptides |
Non-small-cell lung cancer |
A phase IIb trial showed it was highly efficacious in a specific subgroup of patients who had never smoked or were light smokers with non-immunogenic tumors; in that subgroup, there was a statistically significant increase in overall survival: 20.2 months vs. 7.9 months in the placebo group (p=0.0001); the time-to-treatment failure rate also increased to 5.6 months, compared to 3.3 months in the placebo group (p=0.005); the 12-month survival increased to 80% against 8% in the placebo group (p=0.0003) |
6/2/17 |
Verastem Inc. (Boston) |
Duvelisib |
Dual inhibitor of PI3K-delta and PI3K-gamma |
Indolent non-Hodgkin lymphoma |
Long-term follow-up data from the Dynamo study showed it met its primary endpoint of overall response rate (p = 0.0001) at the final analysis; of the 129 patients enrolled, 61 responded (one complete response, 60 partial responses), for an overall response rate (ORR) of 47%; the ORR in each of the three disease subgroups included: 43% in follicular lymphoma (n=83); 68% in small lymphocytic lymphoma (n=28); and 33% in marginal zone lymphoma (n=18) |
6/9/17 |
Xenetic Biosciences Inc. (Lexington, Mass.) |
XBIO-101 |
Sodium cridanimod; small-molecule immunomodulator and interferon inducer |
Endometrial cancer |
Patient enrollment commenced in its phase II study of XBIO-101 in conjunction with progestin therapy in a population of patients who have either failed progestin monotherapy or who have been identified as having progesterone receptor negative |
6/27/17 |
Cardiovascular | |||||
Arca Biopharma Inc. (Westminster, Colo.) |
Gencaro |
Bucindolol hydrochloride |
Atrial fibrillation |
Said the database has been locked for its GENETIC-AF phase IIb trial testing Gencaro so the data safety monitoring board (DSMB) can perform an interim efficacy analysis |
6/21/17 |
Ergomed plc (London) |
Peprostat |
Based on a synthetic peptide designed to eliminate the risk of exposure to blood-derived materials |
Intraoperative surgical bleeding |
The phase IIb proof-of-concept study reached its recruitment midpoint ahead of schedule |
6/22/17 |
Gemphire Therapeutics Inc. (Livonia, Mich.) |
Gemcabene (CI-1027) |
Small molecule |
Homozygous familial hypercholesterolemia |
Top-line data from the open-label study of eight patients on stable maximally tolerated lipid-lowering therapies showed that gemcabene dosed at 600 mg reduced low-density lipoprotein cholesterol (LDL-C) by a mean of 30% (p=0.0047; range -51% to +2%) and showed an effect even in patients with no LDL receptor activity |
6/30/17 |
Central nervous system | |||||
Aegis Therapeutics Inc. (San Diego) |
DFN-02 |
Intranasal spray formulation composed of sumatriptan 10 mg and Aegis' Intravail permeation-enhancing excipient |
Migraine with or without aura |
Phase II results showed that a significantly higher proportion of subjects experienced two-hour pain freedom with DFN-02 compared with placebo: 43.8% (n=48) vs. 22.5% (n=40), p<0.05; DFN-02 was also significantly better than placebo at alleviating patient-identified most bothersome symptom (MBS), from among nausea, photophobia and phonophobia (70.7% vs. 39.5% MBS free at two hours post dose; p<0.01) |
6/29/17 |
Alkermes plc (Dublin) |
ALKS-5461 |
Targets the kappa opioid receptor |
Depression |
Is launching a phase IIIb trial intended to bolster its case for approval |
6/13/17 |
Araclon Biotech SL (Zaragoza, Spain) |
ABvac-40 |
Active immunotherapy that uses the C-terminal fragment of the amyloid beta 40 peptide as an immunogen |
Alzheimer's disease |
Received approval from the Spanish Agency of Medicinal Products and Medical Devices to begin phase II trials |
6/29/17 |
Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.; subsidiary of Otsuka America Inc.) |
AVP-786 |
Consists of deudextromethorphan and ultra-low-dose quinidine |
Neurobehavioral disinhibition, including aggression, agitation and irritability, in patients with traumatic brain injury |
Started enrolling in a randomized, double-blind, placebo-controlled phase II study |
6/8/17 |
Bial SA (S. Mamede do Coronado, Portugal) and Eisai Co. Ltd. (Tokyo) |
Zebinix |
Eslicarbazepine acetate; voltage-gated sodium channel blocker |
Focal-onset epilepsy |
Data from their study 208 phase II trial testing it in children ages 6-16 years old showed it produced no statistically significant difference on attention compared to placebo as measured by Power of Attention differences between Zebinix and placebo, with a Least Square mean difference of 33.2 milliseconds |
6/26/17 |
Braeburn Pharmaceuticals Inc. (Princeton, N.J.) |
Long-acting buprenorphine (CAM-2038) |
Subcutaneous buprenorphine depot formulation |
Opioid use disorder |
Phase II data showed it attained the primary endpoint for both arms of CAM-2038 dosing levels by producing an immediate and sustained blockade of hydromorphone effects (liking maximum effect, 24 mg, 0.813; p<0.001, and 32 mg 0.753; p<0.001) and suppression of withdrawal (Clinical Opiate Withdrawal Scale, 24 mg 0.617; p<0.001, and 32 mg 0.751: p<0.001); CAM-2038 produced a rapid rise of buprenorphine in plasma with maximum concentration around 24 hours, with an apparent half-life of four to five days, and about 50% accumulation of tough concentration from first to second dose |
6/23/17 |
Cingulate Therapeutics LLC (Kansas City) |
CTX-1301 |
Stimulant medications |
Attention deficit hyperactivity disorder |
Started dosing in its proof-of-concept trial |
6/1/17 |
Mallinckrodt plc (Staines-Upon-Thames, U.K.) |
H.P. Acthar Gel |
Repository corticotropin injection |
Amyotrophic lateral sclerosis |
The first patient has been enrolled in the PENNANT phase IIb trial |
6/16/17 |
Novartis AG (Basel, Switzerland) and Amgen Inc. (Thousand Oaks, Calif.) |
AMG-334 |
Erenumab |
Chronic migraine |
A new analysis from a pivotal phase II study showed that, among the patients experiencing 15 or more headache days a month in the erenumab study (n=667), 41% met strict criteria for medication overuse; compared to a 3.5-day reduction in placebo, both dosages of erenumab reduced mean monthly migraine days by 6.6 by the end of the study (both p<0.001 vs. placebo); days requiring acute pain-relief medications were also significantly reduced in both dosage arms (2.1-day reduction for placebo, compared to 5.4 days for erenumab 70 mg and 4.9 for erenumab 140 mg; both p<0.001 vs. placebo) |
6/12/17 |
Probiodrug AG (Halle, Germany) |
PQ912 |
Glutaminylcyclase inhibitor |
Early stage Alzheimer's disease |
Topline data from an exploratory phase IIa trial showed no statistically significant differences between the two study arms in terms of total number of adverse events (49/60 for the drug treatment arm vs. 45/60 for the control arm) or in the number of patients who experienced a serious adverse event (n=8 for the drug treatment arm vs. n=5 for the control arm); dropout rates were considerably higher in the drug treatment group than in the placebo group (n=6 and n=0, respectively, p=0.027) |
6/13/17 |
Promius Pharma LLC (Princeton, N.J.; subsidiary of Dr. Reddy's Laboratories) |
DFN-02 |
Sumatriptan 10 mg and a permeation-enhancing excipient 0.2% 1-O-n-Dodecyl-beta-D-Maltopyranoside |
Migraine with or without aura |
Its phase II study met its primary endpoint, the proportion of subjects with migraine pain freedom at two hours post-dose (2hPF); findings showed that 43.8% (n=48) of patients experienced 2hPF with DFN-02 compared with 22.5% (n=40) for placebo (p<0.05) |
6/8/17 |
Revance Therapeutics Inc. (Newark, Calif.) |
RT-002 |
Daxibotulinum-toxinA injectable |
Moderate to severe isolated cervical dystonia |
Additional results from its phase II study showed a duration of effect of at least 24 weeks for both dose groups – 100 units to 240 units; it also showed a clinically significant mean reduction of the Toronto Western Spasmodic Torticollis Rating Scale-Total score from baseline at week four – the primary efficacy endpoint – in both group A (37%) and group B (39%), with the majority of that benefit maintained through week 24 |
6/7/17 |
Sage Therapeutics Inc. (Cambridge, Mass.) |
Brexanolone (SAGE-547) |
Allosteric modulator of synaptic and extrasynaptic GABAA receptors |
Severe postpartum depression |
Results from a phase II study showed that treatment with brexanolone resulted in a clinically meaningful and statistically significant mean reduction in the 17-item Hamilton Rating Scale for Depression total score that began at 24 hours and was maintained at similar magnitude until the 30-day follow-up; the drug was well-tolerated, with no observations of deaths, serious adverse events or discontinuations |
6/14/17 |
Xenon Pharmaceuticals Inc. (Burnaby, British Columbia) |
TV-45070 |
Sodium channel Nav1.7 inhibitor |
Postherpetic neuralgia |
It failed to outperform placebo in a phase II trial |
6/28/17 |
Zosano Pharma Inc. (Fremont, Calif.) |
M207 |
Formulation of zolmitriptan (Zomig, Impax Laboratories Inc.) delivered utilizing Adhesive Dermally-Applied Microarray technology |
Migraine |
Received the final minutes from recent end of phase II meetings with the FDA; the purpose was to confirm three key elements to the continued development of M207; Zosano received confirmation that a single efficacy study, the recently completed Zotrip trial, is sufficient to support an NDA filing for M207; the FDA confirmed the previously disclosed design of the long-term safety study as sufficient to support an NDA filing for M207 |
6/27/17 |
Diabetes | |||||
Aerpio Pharmaceuticals Inc. (Cincinnati) |
AKB-9778 |
Inhibitor of the intracellular domain of VE-PTP |
Moderate to severe nonproliferative diabetic retinopathy |
The first patient was dosed in its 150-patient TIME-2b phase IIb trial |
6/30/17 |
Eiger Biopharma-ceuticals Inc. (Palo Alto, Calif.) |
Exendin 9-39 |
31-amino acid peptide designed to target and block GLP-1 receptors |
Hypoglycemia in post-bariatric surgical patients |
Completed enrollment of the phase II multiple ascending-dose study of subcutaneous exendin 9-39 |
6/7/17 |
Geneuro SA (Geneva) |
GNbAC1 |
Monoclonal antibody |
Type 1 diabetes |
Treated the first patient in its phase IIa study |
6/20/17 |
Genkyotex SA (Geneva) |
GKT-831 |
NOX 1 and 4 enzyme inhibitor |
Type 1 diabetes and kidney disease |
Said an investigator-initiated phase II study will be conducted at Monash University and the Baker Heart and Diabetes Institute in Melbourne, Australia |
6/29/17 |
Origin Inc. (Princeton, N.J.) |
Nitric oxide |
Plasma-generated |
Chronic diabetic foot ulcers |
The first 52 patients have been randomized in the phase IIb GENESIS trial |
6/2/17 |
Poxel SA (Lyon, France) |
Imeglimin |
Oral anti-diabetic agent |
Type 2 diabetes |
Additional results from the phase IIb study in Japan showed statistically significant (p<0.0001) decreases in the key secondary endpoints of fasting plasma glucose, glycated albumin and percentage of patients reaching a target HbA1c of less than 7%; a statistically significant dose-dependent (500 mg p=0.008,1,000 mg p=0.0008, and 1,500 mg p<0.0001) improvement of the homeostasis model assessment of beta-cell function was also observed; there was a significant decrease in two of the most relevant liver enzymes that are considered biomarkers in liver disease |
6/7/17 |
Zealand Pharma A/S (Copenhagen, Denmark) and Beta Bionics Inc. (Boston) |
Dasiglucagon |
Glucagon analogue |
Type 1 diabetes |
Safety data from a phase IIa trial of dasiglucagon in Beta Bionics' iLet dual-hormone artificial pancreas system in 10 adult patients showed that, under challenging conditions with basal rate of insulin up to twice the normal rate, the artificial pancreas system was able to keep glucose levels in the range of 70 mg/dL to180 mg/dL approximately 70% of the time when dasiglucagon was used in the system and 65% of the time when recombinant glucagon was used; no severe hypoglycemic episodes were observed, and the time below 60 mg/dL glucose was approximately 13% for dasiglucagon and approximately 18% for recombinant glucagon |
6/23/17 |
Gastrointestinal | |||||
Index Pharmaceuticals Holding AB (Stockholm) |
Cobitolimod |
Immunotherapeutic candidate |
Moderate to severe active ulcerative colitis |
The first patient was enrolled in CONDUCT, a phase IIb dose-optimization study |
6/22/17 |
Topivert Pharma Ltd. (London) |
TOP1288 |
Narrow-spectrum kinase inhibitor |
Inflammatory bowel disease and dry eye syndrome |
Disclosed successful completion of three clinical studies TV02, TV03 and TV04 |
6/27/17 |
Infection | |||||
Aclaris Therapeutics Inc. (Malvern, Pa.) |
A-101 45% |
High-concentration hydrogen peroxide topical solution |
Common warts (verruca vulgaris) |
Started two phase II trials to evaluate the safety, tolerability and dose frequency compared with its vehicle (placebo) |
6/30/17 |
Biotron Ltd. (Sydney) |
BIT-225 |
Antiviral drug |
HIV-1 |
A phase II study (BIT225-009) of BIT-225 is currently in progress in Thailand, and a second site has been initiated |
6/16/17 |
Galectin Therapeutics Inc. (Norcross, Ga.) |
GR-MD-02 |
Complex carbohydrate drug targeting galectin-3 |
Nonalcoholic steatohepatitis cirrhosis |
The independent data safety monitoring board concluded that its phase IIb NASH-CX trial should continue |
6/14/17 |
Janssen Research & Development LLC (unit of Johnson & Johnson; New Brunswick) |
JNJ-63623872 |
Pimodivir |
Seasonal influenza A |
Treatment with pimodivir during the phase II Topaz trial significantly decreased viral load over seven days vs. placebo; patients treated with pimodivir and Tamiflu (oseltamivir, Roche Holding AG) also demonstrated a significantly lower viral load compared with those who received pimodivir alone at the same dose; no significant safety concerns were noted with pimodivir treatment in the study |
6/15/17 |
Regulus Therapeutics Inc. (La Jolla, Calif.) |
RG-101 |
N-acetylgalacto-samine (GalNAc)-conjugated asset targeting miR-122 |
Chronic hepatitis C virus |
Is halting development rather than pursuing resolution of a clinical hold placed by the FDA following a second case of jaundice in the phase II program |
6/13/17 |
Inflammatory | |||||
Centrexion Therapeutics Inc. (Boston) |
CNTX-4975 |
Targets the capsaicin receptor (TRPV1) |
Chronic pain associated with knee osteoarthritis |
Six-month efficacy data from the phase IIb TRIUMPH trial show large and statistically significant pain relief continuing through 24 weeks from a single 1-mg injection of CNTX-4975 (p=0.0002), as well as statistically and clinically significant improved knee stiffness and physical function during the trial |
6/15/17 |
Medivir AB (Stockholm) |
MIV-711 |
Cathepsin K inhibitor |
Moderate knee osteoarthritis |
The open-label phase IIa extension study has completed enrollment and the independent data monitoring committee recommended continuation based on a review of the accumulated safety data |
6/12/17 |
Miscellaneous | |||||
Aequus Pharmaceuticals Inc. (Vancouver, British Columbia) |
AQS-1303 |
Long-acting transdermal nausea patch containing pyridoxine and doxylamine |
Nausea and vomiting of pregnancy |
Filed a clinical trial application with Health Canada in preparation for a proof-of-concept study |
6/16/17 |
Aldeyra Therapeutics Inc. (Lexington, Mass.) |
ADX-102 |
Aldehyde trap |
Dry eye disease |
Enrolled the first patient in a phase IIa trial |
6/7/17 |
Aldeyra Therapeutics Inc. (Lexington, Mass.) |
ADX-102 |
aldehyde trapper |
Allergic conjunctivitis |
Data from a phase IIb trial showed demonstrated statistically significant efficacy of 0.5% ADX-102 over vehicle in reducing ocular itching over one hour post-challenge (p < 0.03), and effect sizes were generally larger than those observed in the prior phase IIa clinical trial |
6/15/17 |
Amag Pharmaceuticals Inc. (Waltham, Mass.) |
Digoxin immune fab |
Polyclonal antibody |
Severe preeclampsia in pregnant women |
Said Velo Bio LLC, of Morrisville, N.C., enrolled the first patient in a phase IIb/IIIa study |
6/2/17 |
Bluebird Bio Inc. (Cambridge, Mass.) |
Lenti-D |
Gene therapy |
Cerebral adrenoleukodystrophy |
Data from its Starbeam Study (ALD-102) phase II/III trial in boys under 18 years old showed that, after two years of follow-up, 15 of the 17 (88%) patients remain free of major functional disabilities (MFDs), the primary endpoint of the trial, exceeding the prespecified MFD survival benchmark of 76% |
6/28/17 |
Castle Creek Pharmaceuticals Inc. (Parsippany, N.J.) |
CCP-020 |
Diacerein 1% ointment |
Epidermolysis bullosa simplex |
The first patient was enrolled in the phase II/III DELIVERS trial evaluating the safety and efficacy |
6/6/17 |
Clearside Biomedical Inc. (Alpharetta, Ga.) |
CLS-TA |
Suspension formulation of the corticosteroid triamcinolone acetonide |
Macular edema associated with retinal vein occlusion |
Preliminary results from a non-interventional and retrospective trial of patients who had participated in the completed phase II TANZANITE trial showed that 17 of the 23 patients in the combination arm of the TANZANITE trial, or 74%, did not receive any additional treatment over the nine-month period, compared to only four of 23 patients, or 17%, in the Eylea-only arm |
6/9/17 |
Genkyotex SA (Geneva) |
GKT-831 |
NOX1 and NOX4 enzyme inhibitor |
Primary biliary cholangitis |
Started enrolling patients in its phase II trial |
6/28/17 |
Hansa Medical AB (Lund, Sweden) |
IdeS |
Enzyme that degrades IgG antibodies |
Goodpasture's disease (severe anti-GBM antibody disease) |
The first patient has been treated with IdeS in an investigator-initiated phase II study |
6/21/17 |
Mithra Pharmaceuticals SA (Liège, Belgium) |
Estelle |
15 mg Estetrol (E4) and 3 mg drospirenone (DRSP) |
Contraceptive |
Data from the phase IIb FIESTA study showed individuals treated with Estelle reported satisfaction above those treated with E4/levonorgestrel (LNG), 20 mg E4/DRSP and the Qlaira reference group; well-being also was higher for the E4/DRSP doses compared to E4/LNG and was comparable to Qlaira; the proportion of women with weight loss of 2 kg or more after three and six cycles also was highest in the Estelle group |
6/27/17 |
Otonomy Inc. (San Diego) |
Otiprio |
Ciprofloxacin otic suspension |
Acute otitis media with tympanostomy tubes (AOMT) |
Completed an end-of-phase II review with the FDA for Otiprio as a treatment for pediatric patients; based on advice from the FDA during the meeting, Otonomy plans to conduct a single, sham-controlled, phase III trial of approximately 200 pediatric patients with AOMT to support approval of Otiprio for that indication |
6/23/17 |
Ra Pharmaceuticals Inc. (Cambridge, Mass.) |
RA-101495 |
Subcutaneously administered macrocyclic peptide inhibitor of complement component 5 |
Paroxysmal nocturnal hemoglobinuria |
In a dose-finding phase II trial it was tested in three patients who hadn't previously received Soliris (eculizumab, Alexion Pharmaceuticals Inc.), of which two have completed seven weeks of follow-up and are included in the initial dataset that showed a rapid decline in lactate dehydrogenase (LDH) with a mean LDH at week seven of 1.6 times the upper limit of normal |
6/28/17 |
Reata Pharmaceuticals Inc. (Irving, Texas) |
Omaveloxo-lone |
Nrf2 activator |
Friedreich's ataxia (FA) |
Part one of the phase II trial demonstrated it induced Nrf2, which is suppressed in FA patients, and this was associated with improvements in mitochondrial and neurological function; dose-dependent and time-dependent effects on the modified FA rating scale were observed at the pharmacodynamically active doses, and the maximum effect was observed at the 160 mg dose level |
6/5/17 |
Revance Therapeutics Inc. (Newark, Calif.) |
RT-002 |
Daxibotulinum-toxinA; botulinum toxin drug |
Glabellar (frown) lines |
Results of the BELMONT phase II trial showed the 40-unit dose of RT-002 showed statistical and clinical superiority over 20 units of Botox Cosmetic (onabotulinumtoxinA, Allergan plc), with 67% of patients receiving RT-002 having glabellar line severity rated by investigators as none or mild, compared to 32% of patients who received Botox after 16 weeks; at 24 weeks, the difference was also statistically significant, with 31% of RT-002 patients still responding compared to 12% of placebo patients |
6/16/17 |
Stealth Biotherapeutics Inc. (Boston) |
Elamipretide |
Targets the inner mitochondrial membrane |
Primary mitochondrial myopathy |
Data from the MMPOWER-2 continuation phase II trial, which treated 30 patients from the original MMPOWER trial for four weeks, patients taking elamipretide walked 20 additional meters vs. placebo during the six-minute walk test, although the difference wasn't statically significant |
6/30/17 |
Wilson Therapeutics AB (Stockholm) |
WTX-101 |
Bis-choline tetrathiomolybdate |
Wilson's disease |
Data from a phase II trial showed it met its primary endpoint of copper control (p < 0.001), and significant improvements from baseline to week 24 were observed for both patient-reported disability (p < 0.001) and rater-assessed neurological status ( p < 0.0001), measured using the Unified Wilson Disease Rating Scale Part 2 and 3, respectively |
6/9/17 |
Xencor Inc. (Monrovia, Calif.) |
Xmab-5871 |
Monoclonal antibody that targets CD19 |
Active IgG4-related disease |
Interim data from an ongoing phase II study showed that 93% of patients achieved a response to therapy, 12 of them within two weeks of their first dose |
6/19/17 |
Zynerba Pharmaceuticals Inc. (Devon, Pa.) |
ZYN-002 |
Cannabidiol (CBD) gel |
Fragile X syndrome |
Met its enrollment target of 16 patients in its phase II FAB-C study testing it in children |
6/9/17 |
Respiratory | |||||
Bavarian Nordic A/S (Copenhagen, Denmark) |
MVA-BN RSV |
Universal vaccine candidate designed to induce protective immune responses |
Respiratory syncytial virus (RSV) subtypes (A and B) |
Results from a 421-subject phase II study showed that, administered at two different doses, it produced a two- to fourfold boost in antibodies compared to placebo two weeks after a single vaccination; a five- to 10-fold increase in T-cell responses to the five RSV proteins were observed in the majority of subjects one week after a single vaccination; in a subset of patients who were given a second dose at day 28, the immune response was boosted compared to placebo at the three-month time point post-vaccination |
6/28/17 |
Novavax Inc. (Gaithersburg, Md.) |
RSV F Vaccine |
RSV F protein recombinant nanoparticle vaccine candidate |
Respiratory syncytial virus |
Data from the second of its two phase II trials in women of child-bearing age were published, detailing the antibody response elicited by the vaccine, including 11.6-fold to 12.7-fold increases in anti-F IgG responses in women receiving a single dose of 120 mcg RSV F vaccine with 0.2 mg or 0.4 mg of aluminium |
6/8/17 |
Novoteris LLC (Garden Grove, Calif.) |
Thiolanox |
Inhaled nitric oxide |
Cystic fibrosis |
The FDA and the Therapeutic Products Directorate of Health Canada have cleared its phase II trial application and it will now begin recruiting subjects into its study |
6/6/17 |
Protalix Biotherapeutics Inc. (Carmiel, Israel) |
Alidornase alfa (PRX-110) |
Plant cell-expressed, chemically modified recombinant DNase I enzyme resistant to inhibition by actin |
Cystic fibrosis |
Phase II results showed that treatment resulted in a mean absolute increase in percent predicted forced expiratory volume in 1 second, or ppFEV1, of 3.4 points from baseline, and an increase in ppFEV1 of 3.3 points, when compared to measurements taken from patients at enrollment before the switch from Pulmozyme (dornase alfa) to alidornase alfa |
6/8/17 |
Verona Pharma plc (London) |
RPL-554 |
Inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 |
Chronic obstructive pulmonary disease |
Gained regulatory approval in five European countries, the U.K., Germany, Romania, Bulgaria and the Czech Republic, for a phase IIb dose-ranging trial |
6/28/17 |
Windtree Therapeutics Inc. (Warrington, Pa.) |
Aerosolized KL4 surfactant |
Synthetic, peptide-containing surfactant |
Respiratory distress syndrome in premature infants |
Completed enrollment in its AEROSURF phase IIb trial |
6/5/17 |
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Notes The date indicated refers to the BioWorld issue in which the news item can be found. For more information about individual companies and/or products, see Cortellis. | |||||
