Company (location) |
Product |
Description |
Indication |
Status |
Date |
Autoimmune | |||||
Abbvie Inc. (North Chicago) |
Upadacitinib (ABT-494) |
Oral JAK1 inhibitor |
Rheumatoid arthritis (RA) |
It met all primary and secondary endpoints in a phase III study; top-line results showed that, after 12 weeks of treatment with either a 15-mg or a 30-mg dose of upadacitinib, up to 66% of people with moderate to severe RA for whom ongoing treatment with conventional synthetic disease-modifying antirheumatic drugs such as methotrexate has fallen short achieved an ACR20 response; those who achieved it saw a 20% improvement in tender and swollen joint counts, self-assessed pain, global disease activity and physical function, as well as improvements in disease activity noted by their doctors, thus meeting the study's primary endpoint |
6/8/17 |
Biogen Inc. (Cambridge, Mass.) |
Benepali |
Etanercept |
Rheumatoid arthritis (RA), psoriatic arthritis (PsA) or axial spondyloarthritis (SpA) |
Two real-world studies evaluated the safety and efficacy of the drug in patients following a switch from reference etanercept; in 1,548 patients, disease activity was shown to be largely unaffected at three months post-switch and comparable to that observed in the three months prior to switch; in a separate cohort of 92 patients with RA, PsA or ankylosing spondylitis in the U.K., a low rate of treatment discontinuations due to inefficacy or adverse events was demonstrated at six months post-switch |
6/15/17 |
Boehringer Ingelheim GmbH (Ingelheim, Germany) |
BI-695501 |
Biosimilar adalimumab |
Moderately to severely active rheumatoid arthritis |
Results from the pivotal phase III VOLTAIRE-RA study confirmed that BI-695501 and Humira (adalimumab, Abbvie Inc.) have similar efficacy, safety and immunogenicity; the co-primary endpoints, which measured the proportion of patients achieving an ACR20 (American College of Rheumatology 20) improvement at weeks 12 and 24, were met |
6/15/17 |
Can-Fite Biopharma Ltd. (Petach Tikva, Israel) |
Piclidenoson |
A3 adenosine receptor agonist |
Rheumatoid arthritis |
Concluded an investigator meeting in Europe with about 100 rheumatologists for its global ACRobat study as a first-line treatment and replacement for the current standard of care, methotrexate |
6/9/17 |
Eli Lilly and Co. (Indianapolis) |
Taltz |
Ixekizumab |
Active psoriatic arthritis |
Data from the SPIRIT-P2 phase III study showed that 53% of patients treated every four week with Taltz and 48% of patients treated with Taltz every two weeks had ACR20 at 24 weeks, compared to 19% of those treated with placebo (p < 0.0001); 35% of Taltz patients treated every four weeks and 33% of Taltz patients treated every two weeks hit ACR50, compared to 19% of those on placebo (p < 0.0001); on ACR70, 22% and 12% of patients receiving Taltz every four weeks and every two weeks, respectively, hit the mark vs. 0% of those on placebo (p< 0.0001) |
6/16/17 |
Eli Lilly and Co. (Indianapolis) |
Taltz |
Ixekizumab |
Active psoriatic arthritis |
Results from the extension period of the phase III SPIRIT-P1 trial showed that patients treated with Taltz at either 80 mg every two weeks or every four weeks following a 160-mg starting dose experienced either no progression or minimal radiographic progression of structural joint damage as measured by the change from baseline in the van der Heijde modified Total Sharp Score for PsA at 52 weeks |
6/19/17 |
Galapagos NV (Mechelen, Belgium) |
Filgotinib |
JAK1 inhibitor |
Rheumatoid arthritis |
Data from the 559 patients treated with filgotinib in the DARWIN 3 extension trial for patients who completed DARWIN 1 or 2, showed that, based on an observed case analysis, 84%, 65%, 44% and 51% of patients reached ACR20, ACR50, ACR70 and DAS28-CRP remission at week 60, respectively |
6/21/17 |
Genentech Inc. (South San Francisco; member of the Roche Group) |
Ocrevus |
Ocrelizumab; humanized monoclonal antibody designed to target CD20-positive B cells |
Relapsing and primary progressive forms of multiple sclerosis (RMS and PPMS) |
New post-hoc analyses from phase III trials showed it reduced disease activity and disability progression, as measured by No Evidence of Progression or Active Disease (NEPAD); in RMS, Ocrevus increased the proportion of patients maintaining NEPAD by 82% compared with Rebif (interferon beta-1a, EMD Serono Inc.) at 96 weeks in a pooled exploratory analysis of the OPERA I and II studies; in the ORATORIO study, the proportion of PPMS patients who maintained NEPAD at 120 weeks was 29.9% for those who took Ocrevus compared to 9.4% for placebo |
6/26/17 |
Incyte Corp. (Wilmington, Del.) and Eli Lilly and Co. (Indianapolis) |
Olumiant |
Baricitinib |
Moderate to severe rheumatoid arthritis |
Analysis of data pooled from eight Olumiant clinical trials showed that patients with treated with Olumiant had 3.8 serious infections per 100 patient-years during the first 24 weeks of treatment, similar to the 4.2 serious infections per 100 patient-years seen in the placebo group |
6/19/17 |
Janssen Research & Development LLC (unit of Johnson & Johnson; New Brunswick) |
Simponi Aria |
Golimumab; anti-tumor necrosis factor-alpha therapy |
Active psoriatic arthritis |
Results from its pivotal phase III GO-VIBRANT trial showed it met its primary endpoint by a statistically significant margin with 75.1% of patients receiving 2 mg/kg of Simponi Aria achieving at least a 20% improvement in arthritis signs and symptoms as measured by the American College of Rheumatology (ACR20) at week 14, compared with 21.8% of patients receiving placebo |
6/15/17 |
Janssen-Cilag International NV (Beerse, Belgium; unit of Johnson & Johnson) and Glaxosmithkline plc (London) |
Sirukumab |
Anti-interleukin (IL)-6 antibody |
Rheumatoid arthritis (RA) |
Results from a pivotal study in patients whose needs aren't met by standard RA therapies showed that 54.3% of patients receiving a 50-mg dose of sirukumab every four weeks and about 59.3% receiving a 100-mg dose every two weeks achieved ACR20, or at least a 20% improvement in the signs and symptoms of disease, at week 52 |
6/16/17 |
Leo Pharma A/S (Ballerup, Denmark) |
Tralokinumab |
Interleukin-13-targeting antibody |
Atopic dermatitis |
The first patients have been dosed in the ECZema TRAlokinumab Trial no. 1 (ECZTRA 1) phase III trial |
6/16/17 |
Merck KGaA (Darmstadt, Germany) |
Mavenclad |
Cladribine tablets |
Relapsing multiple sclerosis |
Safety and efficacy data from the placebo-controlled CLARITY, CLARITY Extension and ORACLE-MS trials showed early and discontinuous reduction of peripheral blood B cells, whose numbers reached a nadir at 13 weeks post-treatment followed by rapid reconstitution toward baseline; moderate reduction in T-cell counts also was shown, although to a lesser degree than B cells; the reduction was more pronounced in CD4+ than CD8+ lymphocytes; lymphopenia was the most commonly reported adverse event in patients treated with cladribine tablets, with an incidence of infections of 48.3% compared to 42.5% with placebo |
6/27/17 |
Novartis AG (Basel, Switzerland) |
Cosentyx |
Secukinumab; interleukin-17A inhibitor |
Ankylosing spondylitis (AS) |
Results from the phase III MEASURE 1 extension study showed that, after three years of treatment, 80% of AS patients responded to the drug as measured by the Assessment of Spondyloarthritis International Society 20 response criteria |
6/16/17 |
Novartis AG (Basel, Switzerland) |
Cosentyx |
Secukinumab; interleukin-17A inhibitor |
Psoriatic arthritis |
A post-hoc analysis of the phase III FUTURE 2 study showed that 15% of patients treated with Cosentyx reported no pain or discomfort after four weeks of treatment compared to 5% for placebo |
6/16/17 |
Pfizer Inc. (New York) |
Xeljanz 5 mg |
Tofacitinib; JAK inhibitor |
Moderate to severe rheumatoid arthritis |
Results from the ORAL Strategy phase IIIb/IV study testing Xeljanz twice daily as a monotherapy or in combination with methotrexate (MTX) compared to Humira (adalimumab, Abbvie Inc.) plus MTX showed that on the primary endpoint, the percentage of patients achieving an ACR50 response at month six, 46% of patients in the Xeljanz plus MTX group, 38.3% in the Xeljanz monotherapy group and 43.8% in the Humira/MTX group achieved ACR50 |
6/19/17 |
Cancer | |||||
Aslan Pharmaceuticals Pte Ltd. (Singapore) |
Varlitinib |
Reversible, small-molecule pan-HER inhibitor |
Gastric cancer |
Received authorization from Singapore's Health Sciences Authority to initiate a pivotal study |
6/2/17 |
Aveo Oncology Inc. (Cambridge, Mass.) |
Tivozanib |
Long half-life inhibitor of all three vascular endothelial growth factor receptors |
Refractory advanced renal cell carcinoma |
The pivotal TIVO-3 trial, comparing tivozanib to Nexavar (sorafenib, Bayer AG), reached its enrollment target of 322 patients |
6/21/17 |
Boston Biomedical Inc. (Cambridge, Mass.) |
Napabucasin |
Cancer stem cell pathway inhibitor |
Advanced gastric and gastroesophageal junction cancer |
It unblinded a global phase III study after the trial's data and safety monitoring board (DSMB) determined that combining napabucasin with paclitaxel is unlikely to yield better overall survival at 36 months than paclitaxel alone; no safety concerns were uncovered in the prespecified interim analysis that triggered the DSMB recommendation, and the trial will continue in open label fashion |
6/27/17 |
Bristol-Myers Squibb Co. (New York) and Seattle Genetics Inc. (Bothell, Wash.) |
Opdivo and Adcetris combination |
Nivolumab and brentuximab vedotin |
Relapsed/refractory or transplant-ineligible advanced classical Hodgkin lymphoma |
Agreed to evaluate the combination of BMS' Opdivo and Seagen's Adcetris compared to Adcetris alone in a pivotal phase III trial in individuals |
6/5/17 |
Celgene Corp. (Summit, N.J.) |
Revlimid |
Lenalidomide |
Relapsed or refractory marginal zone lymphoma (MZL) |
Results from an interim analysis of the MAGNIFY phase IIIb trial of Revlimid plus Rituxan (rituximab, Roche Holding AG/Biogen Inc.) showed the combination produced an overall response rate (ORR) of 66% with a complete response rate, including unconfirmed responses (CR/CRu) of 44% in all 32 patients with MZL; in just the 14 patients with nodal MZL, the combo had an ORR of 57% with a CR/CRu rate of 57%; in the eight patients with splenic MZL, the ORR was 63% with a CR of 25%; the 10 patients with mucosa-associated lymphoid tissue lymphoma had an ORR of 80% with a CR/CRu rate of 40% |
6/20/17 |
Celgene Corp. (Summit, N.J.) and Acceleron Pharma Inc. (Cambridge, Mass.) |
Luspatercept |
Modified activin receptor type IIb fusion protein |
Myelodysplastic syndromes and beta-thalassemia |
Completed target enrollment in the MEDALIST and BELIEVE phase III trials |
6/2/17 |
Celltrion Inc. (Incheon, South Korea) |
CT-P10 |
CD20-targeting antibody |
Follicular lymphoma (FL) |
New data from the randomized, double-blind, controlled phase III study in 140 advanced FL patients showed that CT-P10 was noninferior compared to Rituxan (rituximab, Roche Holding AG/Biogen Inc.) when each were given in combination with standard chemotherapy of cyclophosphamide, vincristine and prednisone in patients with previously untreated advanced FL over eight cycles |
6/15/17 |
Clovis Oncology Inc. (Boulder, Colo.) |
Rubraca |
Rucaparib |
BRCA mutation-associated advanced ovarian cancer |
Topline data from the phase III ARIEL3 trial showed it successfully achieved the primary endpoint of improved progression-free survival (PFS) by investigator review in each of the three populations studied; PFS was also improved in the rucaparib group compared with placebo by blinded independent central review, a key secondary endpoint |
6/20/17 |
Delmar Pharmaceuticals Inc. (Vancouver, British Columbia) |
VAL-083 |
Small molecule chemotherapeutic |
Glioblastoma multiforme |
Received institutional review board approval to conduct its pivotal phase III trial |
6/23/17 |
Eleven Biotherapeutics Inc. (Cambridge, Mass.) |
Vicinium |
Single-protein, anti-epithelial cell adhesion molecule antibody fragment fused with Pseudomonas exotoxin A |
Non-muscle invasive bladder cancer |
Its phase III registration trial has exceeded 50% enrollment, and the independent data and safety monitoring board recommended that the trial continue as planned, without modification |
6/2/17 |
Helsinn Group (Lugano, Switzerland) |
NEPA |
Netupitant/palonosetron |
Nausea and vomiting associated with chemotherapy |
Data from a phase III trial in 828 chemotherapy-naive Asian patients showed that the primary efficacy endpoint of overall complete response – no emesis and no rescue medication – for on day one was noninferior to a three-day regimen of aprepitant and granisetron with a comparable safety profile |
6/21/17 |
Hutchison China Meditech Ltd. (Chi-Med; Hong Kong) and Astrazeneca plc (London) |
Savolitinib |
Inhibitor of c-MET receptor tyrosine kinase |
c-MET-driven papillary renal cell carcinoma |
Started a pivotal phase III registration study comparing savolitinib to Sutent (sunitinib, Pfizer Inc.) |
6/30/17 |
Janssen-Cilag International NV (Beerse, Belgium; unit of Johnson & Johnson) |
Imbruvica |
Ibrutinib; Bruton's tyrosine kinase inhibitor |
Relapsed or refractory mantle cell lymphoma |
Three-year follow-up data from the phase III RAY study showed a median progression-free survival of 25.4 months in patients who had one prior line of therapy, compared to 6.2 months for patients treated with Torisel (temsirolimus, Pfizer Inc.); median overall survival was also longer in patients treated with Imbruvica (42.1 months) compared to Torisel (27.0 months) |
6/20/17 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Keytruda |
Pembrolizumab; PD-1 inhibitor |
Multiple myeloma |
Accepted the recommendation of the data monitoring committee to pause new enrollment in the phase III KEYNOTE-183 and KEYNOTE-185 trials exploring Keytruda in combination with other therapies, following reports of deaths in the Keytruda groups; patients currently enrolled in those two studies will continue to receive treatment, and other studies of Keytruda continue unchanged |
6/14/17 |
Morphosys AG (Planegg, Germany) |
MOR-208 |
Fc-enhanced monoclonal antibody directed against CD19 |
Relapsed or refractory diffuse large B-cell lymphoma |
Opened enrollment in the pivotal phase III part of the B-MIND study of MOR-208, which is testing it in combination with bendamustine vs. Rituxan (rituximab, Roche Holding AG) plus bendamustine in patients who are not eligible for high-dose chemotherapy and autologous stem cell transplantation |
6/8/17 |
OSE Immuno-therapeutics SA (Nantes, France) |
Tedopi |
Combination of neo-10 epitopes selected and optimized from five tumorous antigens |
Non-small-cell lung cancer |
Following the recommendation by independent data monitory committee on the phase III study, Atalante 1, patient accrual is being temporarily halted while continuing treatment for patients already enrolled in order to further assess the study's current patient profile in relation to the potential benefit of Tedopi with more mature data |
6/26/17 |
Seattle Genetics Inc. (Bothell, Wash.) |
Vadastuximab talirine (SGN-CD33A) |
Antibody drug conjugate targeting CD33 |
Acute myeloid leukemia (AML) |
Is discontinuing the phase III CASCADE clinical trial testing it in combination with the hypomethylating agents (HMAs) azacitidine or decitabine compared to an HMA alone in frontline older AML patients after the Independent Data Monitoring Committee (IDMC) noted a higher rate of deaths, including fatal infections, in patients treated with vadastuximab talirine compared to the control arm of the trial; Seattle Genetics and the IDMC don't believe the safety concerns are related to hepatotoxicity; Seattle Genetics is suspending patient enrollment and treatment in all of its trials and will consult with the FDA to determine future plans |
6/20/17 |
Takeda Pharmaceutical Co. Ltd. (Cambridge, Mass.) and Seattle Genetics Inc. (Bothell, Wash.) |
Adcetris |
Brentuximab vedotin; CD30-directed antibody-drug conjugate |
Cutaneous T-cell lymphoma |
Phase III ALCANZA data showed it demonstrated statistically significant improvement in the rate of objective response lasting at least four months vs. the control arm (56.3% vs. 12.5%, p < 0.0001); key secondary endpoints, including complete response rate, progression-free survival and reduction in the burden of symptoms during treatment (Skindex-29), were all highly statistically significant in favor of the Adcetris arm |
6/8/17 |
Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) and Seattle Genetics Inc. (Bothell, Wash.) |
Adcetris |
Brentuximab vedotin; CD30-targeted antibody-drug conjugate |
Previously untreated advanced classical Hodgkin lymphoma |
The phase III ECHELON-1 trial met its primary endpoint of a statistically significant improvement in modified progression-free survival (PFS) vs. the control arm; results demonstrated that combination treatment with Adcetris resulted in a statistically significant improvement in modified PFS vs. the control arm as assessed by an Independent Review Facility (hazard ratio=0.770; p =0.035); the two-year modified PFS rate for patients in the Adcetris arm was 82.1% compared to 77.2% in the control arm |
6/27/17 |
TG Therapeutics Inc. (New York) |
TG-1101 |
Ublituximab; glycoengineered anti-CD20 monoclonal antibody |
Chronic lymphocytic leukemia |
Data from its phase III GENUINE trial showed that adding TG-1101 to Imbruvica (ibrutinib, Abbvie Inc. and Johnson & Johnson) improved overall response rate (ORR), complete response rate and minimal residual disease; the trial met its primary endpoint, demonstrating a statistically significant improvement in ORR, compared to ibrutinib alone in both the intent-to-treat population (p=0.001) and treated population (p<0.001) |
6/19/17 |
Xbiotech Inc. (Austin, Texas) |
Xilonix |
Antibody |
Colorectal cancer |
Performed its second prospectively planned, unblinded analysis of the phase III XCITE study, and the independent data monitoring committee recommended the early termination of the study since the findings were not sufficient to meet efficacy or the threshold for continuation |
6/12/17 |
Cardiovascular | |||||
Genentech Inc. and Chugai Pharmaceutical Co. Ltd. (both units of Roche Holding AG; Basel, Switzerland) |
Emicizumab |
Bispecific monoclonal antibody designed to bring together factors IXa and X to activate the natural coagulation cascade and restore the blood clotting process |
Hemophilia A with inhibitors to factor VIII |
Phase III data from HAVEN 1 showed a statistically significant and clinically meaningful reduction in the primary endpoint of bleed rate, at 87% (RR [risk rate]=0.13, p<0.0001) for emicizumab prophylaxis compared to on-demand treatment with bypassing agents (BPAs); after a median observation time of 31 weeks, 62.9% of patients receiving emicizumab experienced zero treated bleeds compared to 5.6% of those receiving on-demand BPAs |
6/27/17 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Anacetrapib |
Cholesteryl ester transfer protein inhibitor |
Cardiovascular events |
Top-line results from REVEAL (Randomized EValuation of the Effects of Anacetrapib through Lipid modification) showed it significantly reduced major coronary events, defined as the composite of coronary death, myocardial infarction and coronary revascularization, compared to placebo |
6/28/17 |
Novartis AG (Basel, Switzerland) |
Ilaris |
Canakinumab; interleukin-beta1 (IL-beta1) inhibitor |
Cardiovascular disease |
In the Cantos phase III trial it met the primary endpoint, a composite of heart attack, stroke and cardiovascular death |
6/23/17 |
Resverlogix Corp. (Calgary) |
Apabetalone (RVX-208) |
Small-molecule selective bromodomain and extra-terminal inhibitor |
Cardiovascular disease |
The independent Data and Safety Monitoring Board for its phase III BETonMACE trial completed a fourth planned safety review and recommended that the study should continue as planned without any modifications |
6/29/17 |
Central nervous system | |||||
Acorda Therapeutics Inc. (Ardsley, N.Y.) |
CVT-301 |
Inhaled levodopa |
Parkinson's disease |
A phase III trial met its primary endpoint, with an 84-mg dose helping enrolled patients achieve a statistically significant improvement in motor function vs. placebo, as measured by mean change in the Unified Parkinson's Disease Rating Scale – Part III at 30 minutes post-dose at week 12 (-9.83 vs. -5.91; p = 0.009); urinary tract infection was the only serious adverse event reported at a rate of higher than 1%, occurring at a rate of 14.8% in the treatment group and 10.2% in the observational control arm |
6/7/17 |
Adamas Pharmaceuticals Inc. (Emeryville, Calif.) |
ADS-5102 |
Amantadine extended-release capsules |
Parkinson's disease |
An updated analysis of efficacy and tolerability data from the phase III EASE LID 2 showed that, overall, results demonstrated it was well tolerated and the treatment effect on motor complications, as measured by the Movement Disorder Society-Unified Parkinson's Disease Rating Scale part IV score, was maintained for up to 88 weeks; analyses of data from two subgroups of patients both showed a rapid and sustained reduction in dyskinesia and off periods after introduction of ADS-5102 |
6/12/17 |
Adamas Pharmaceuticals Inc. (Emeryville, Calif.) |
ADS-5102 |
Amantadine |
Levodopa-induced dyskinesia (LID) in Parkinson's disease |
The phase III EASE LID trial met its prespecified primary endpoint, showing that patients who received ADS-5102 experienced greater decrease in LID at 12 weeks compared to placebo (p=0.0009); the improvement was maintained at 24 weeks, with ADS-5102-treated patients again showing a significantly greater decrease than placebo-treated patients (p=0.0008) |
6/14/17 |
Alder Biopharma-ceuticals Inc. (Bothell, Wash.) |
Eptinezumab |
Monoclonal antibody |
Migraine |
The phase III Promise 1 trial met the primary endpoint bell, demonstrating statistically significant reductions in monthly migraine days from baseline (average of 8.6 days) over weeks one through 12, with 4.3 monthly migraine days for 300 mg (p=0.0001) and 3.9 days for 100 mg (p=0.0179) compared to an average 3.2 days for placebo; secondary endpoints evaluating time points through the first quarterly dose include about a 75% reduction in monthly migraine days achieved over weeks one through four of 31.5% for 300 mg and (p=0.0066), and 30.8% for 100 mg (p=0.0112) compared to 20.3% for placebo |
6/28/17 |
Alkermes plc (Dublin) |
ALKS-3831 |
Oral atypical antipsychotic drug candidate |
Schizophrenia |
Initiated ENLIGHTEN-Early, a supportive study in the ENLIGHTEN development program, a phase III study comparing the weight gain profile of ALKS-3831 to olanzapine |
6/9/17 |
Alzheon Inc. (Framingham Mass.) |
Tramiprosate |
Amyloid-targeted drug |
Alzheimer's disease (AD) |
A subanalysis of data from two phase III trials testing tramiprosate in 2,025 patients showed it produced the largest efficacy signals in AD patients who were APOE4/4 homozygous with the mild form of the disease, as measured by ADAS-cog11, CDR-SB and the Disability assessment for dementia |
6/23/17 |
Cara Therapeutics Inc. (Stamford, Conn.) |
CR-845 |
Peripherally acting kappa opioid receptor agonist |
Postoperative pain |
Completed a pre-specified interim conditional power analysis of its adaptive phase III trial, and, based on the guidance of the independent data monitoring committee, the trial will continue to test it in up to 450 patients undergoing abdominal surgery; both doses of CR-845 were well-tolerated with no significant changes in the monitored safety parameters |
6/22/17 |
Eisai Co. Ltd. (Tokyo) |
Belviq |
Lorcaserin |
Obesity and weight loss |
The independent data monitoring committee recommended continuing its phase IIIb/IV Cardiovascular And Metabolic Effects of Lorcaserin In Overweight And Obese Patients (CAMELLIA) TIMI61 trial after the completion of a pre-specified interim safety analysis evaluating the incidence of Major Adverse Cardiovascular Events, defined as cardiovascular death, myocardial infarction or stroke |
6/26/17 |
Eli Lilly and Co. (Indianapolis) |
Galcanezumab |
Monoclonal antibody |
To prevent episodic and chronic migraine |
Additional findings from phase III studies showed patients treated with galcanezumab 120 mg and 240 mg doses experienced a statistically significantly greater decrease in the average number of monthly migraine headache days compared to patients treated with placebo; a statistically significantly greater percentage of patients treated with both doses of galcanezumab in both studies also achieved at least a 50%, 75% and 100% reduction in the number of migraine headache days compared to placebo over the six-month treatment period |
6/12/17 |
Indivior plc (Slough, U.K.) |
RBP-6000 |
Once-monthly injectable buprenorphine in the Atrigel delivery system |
Moderate-to-severe opioid use disorder |
The pivotal phase III clinical trial (RB-US-13-0001) met its primary and key secondary endpoints for both dosage regimens of RBP-6000, which demonstrated clinically and statistically significant differences in percentage abstinence and treatment success, defined as any subject with ≥80% of urine samples negative for opioids combined with self-reports negative for illicit opioid use from weeks five to 24, compared to placebo |
6/22/17 |
Jazz Pharmaceuticals plc (Dublin) |
JZP-110 |
Selective dopamine and norepinephrine reuptake inhibitor |
Excessive sleepiness associated with narcolepsy |
Results from its phase III TONES 2 study demonstrated that both the 150-mg and 300-mg doses met the co-primary endpoints of change in mean sleep latency on the Maintenance of Wakefulness Test (MWT) (p<0.0001) and change in Epworth Sleepiness Scale score (p<0.0001 for 150-mg and 300-mg doses and p<0.05 for 75-mg dose) from baseline to week 12; on the key secondary endpoint, defined as the percentage of patients who reported improvement on the Patient Global Impression of Change scale, JZP-110 significantly increased the percentage of patients who reported improvement in their overall condition at all doses relative to placebo at week 12 (p<0.0001 for 150-mg and 300-mg doses and nominal "p" value of p<0.05 at the 75-mg dose as the co-primary endpoint of MWT was not met at that dose) |
6/8/17 |
Merz Neurosciences (unit of Merz North America Inc.; Raleigh, N.C.) |
Xeomin |
Incobotulinum-toxinA |
Adult sialorrhea (unwanted drooling) due to Parkinson's disease and other neurologic disorders |
Top-line results from its pivotal phase III trial showed it met the co-primary endpoints, showing statistically significant improvement in change in unstimulated salivary flow rate and in patients' Global Impression of Change Scale at week four for those administered 100U incobotulinumtoxinA compared to baseline pre-injection vs. placebo (p=0.004 and p=0.002, respectively) |
6/8/17 |
Myovant Sciences Ltd. (Basel, Switzerland) |
Relugolix |
Small-molecule gonadotropin-releasing hormone receptor antagonist |
Endometriosis-associated pain |
Started a phase III program consisting of two trials, SPIRIT 1 and SPIRIT 2 |
6/30/17 |
Neuroderm Ltd. (Rehovot, Israel) |
ND-0612 |
Liquid formulation of levodopa/carbidopa |
Parkinson's disease |
Received a scientific advice letter from the EMA's Scientific Advice Working Party accepting the main design elements suggested by the company for the amended iNDiGO phase III study, including trial population and primary and secondary endpoints, as well as the suggested statistical analysis approach |
6/7/17 |
Scilex Pharmaceuticals Inc. (subsidiary of Sorrento Therapeutics Inc.; San Diego) |
Ztlido |
Lidocaine medicated plaster 1.8% |
Post-herpetic neuralgia |
Results from its comparative adhesion performance study comparing Ztlido to Versatis (medicated plaster 5%, Grünenthal GmbH) showed it demonstrated superior adhesion on the six-point adhesion scale with a mean adhesion score of 5.35 over the 12 hours of dosing in 44 healthy volunteers, compared to a score of 3.59 for Versatis; at the end of the 12-hour dosing period, Ztlido had a mean adhesion score of 5.006, compared to 2.268 for Versatis |
6/15/17 |
Sumitomo Dainippon Pharma Co. Ltd. (Osaka, Japan) |
Lurasidone hydrochloride |
Atypical antipsychotic |
Bipolar I depression |
The phase III trial met its primary endpoint in the 20-mg/day to 60-mg/day group; by pre-specified analysis in the intent-to-treat population (n=522), statistically significant improvement was demonstrated for the lurasidone 20 mg/day to 60 mg/day group compared to the placebo group at primary endpoint, namely, the change from baseline in MADRS* total score after six weeks of treatment (20 mg/day to 60 mg/day −13.6, placebo group -10.6; adjusted p=0.007) |
6/12/17 |
Teva Pharmaceutical Industries Ltd. (Jerusalem) |
Fremanezumab (TEV-48125) |
Anti-CGRP drug |
Prevention of migraine |
Phase III HALO study results showed patients treated with monthly and quarterly fremanezumab experienced clinically and statistically significant improvements in all endpoints and 12 pre-specified analyses; fremanezumab given monthly improved the average number of migraine days, relative to baseline, by 41.6% for the duration of the trial (-3.7 days vs. -2.2 days for placebo, p<0.0001); number of days with disability were decreased by 64.7% (p=0.0021) and medication consumption was decreased by 39% (p<0.0001). |
6/8/17 |
Teva Pharmaceutical Industries Ltd. (Jerusalem) |
Deutetrabenazine (SD-809) |
Small-molecule inhibitor of vesicular monoamine 2 transporter |
Tardive dyskinesia |
Results from the phase III AIM-TD (Addressing Involuntary Movements in Tardive Dyskinesia) trial showed it produced a statically significant improvement compared to placebo in the least-squares mean Abnormal Involuntary Movement Scale of 1.9 points and 1.8 points at the two higher doses of 36 mg/day and 24 mg/day, respectively |
6/30/17 |
Vtv Therapeutics Inc. (High Point, N.C.) |
Azeliragon |
Oral antagonist of the receptor for advanced glycation endproducts |
Mild Alzheimer's disease |
Completed enrollment for its phase III placebo-controlled STEADFAST trial |
6/2/17 |
Diabetes | |||||
Lexicon Pharmaceuticals Inc. (The Woodlands, Texas) |
Sotagliflozin |
Dual SGLT1 and SGLT2 inhibitor |
Type 1 diabetes |
Top-line results from its phase III inTandem3 trial showed it met its primary endpoint of superiority vs. placebo in the proportion of patients with A1C levels below 7% at week 24 and no episode of severe hypoglycemia and no episode of diabetic ketoacidosis after randomization; sotagliflozin demonstrated a generally well-tolerated safety profile |
6/12/17 |
Gastrointestinal | |||||
Redhill Biopharma Ltd. (Tel Aviv, Israel) |
Bekinda (RHB-102) |
Bimodal extended-release, once-daily oral pill formulation of ondansetron |
Acute gastroenteritis and gastritis |
Results from the phase III GUARD study showed that 65.6% of Bekinda-treated patients as compared to 54.3% of patients receiving placebo met the primary endpoint of patients that didn't vomit or require rescue medication or intravenous hydration from 30 minutes after the first dose until 24 hours post-dose |
6/15/17 |
Tigenix NV (Leuven, Belgium) |
Cx-601 |
Local administration of allogeneic expanded adipose-derived stem cells |
Complex perianal fistulas in patients with Crohn's disease |
Formally launched its global pivotal phase III trial |
6/14/17 |
Infection | |||||
Actelion Ltd. (Allschwil, Switzerland) |
Cadazolid |
Oxazolidine antibiotic |
Clostridium difficile-associated diarrhea |
The phase III IMPACT 1 study from the pivotal program met its primary endpoint of noninferiority to vancomycin, but the IMPACT 2 phase III missed the same endpoint; the global studies compared the efficacy and safety of cadazolid (250 mg administered orally twice daily for 10 days) vs. vancomycin (125 mg administered orally four times daily for 10 days) across 1,263 patients |
6/9/17 |
Agenus Inc. (Lexington, Mass.) and Glaxosmithkline plc (London) |
Shingrix |
Vaccine that contains Agenus' immune adjuvant, QS-21 Stimulon |
Shingles |
In a phase III trial it triggered a strong immune response in elderly patients previously treated with Zostavax |
6/22/17 |
Biotime Inc. (Alameda, Calif.) |
Renevia |
Medical device being developed as a replacement for whole adipose tissue in cell assisted lipotransfer procedures |
Facial lipoatrophy in HIV patients |
Based on the analysis of top-line data, the Renevia pivotal trial in Europe has met its primary endpoint of the change in hemifacial volume at six months in the treated patients compared to patients in the delayed treatment arm as measured by 3-D photographic volumetric assessment; on average, 5.1 cc of hemifacial volume was measured after six months, which represents an approximate 100% retention of transplanted volume; untreated patients had no incremental hemifacial volume after six months |
6/15/17 |
Genfit SA (Lille, France) |
Elafibranor |
Small molecule designed to act via dual peroxisome proliferator-activated alpha/delta pathways |
Non-alcoholic steatohepatitis |
The data safety monitoring board issued a positive recommendation for the continuation of the RESOLVE-IT phase III trial, without any modifications |
6/2/17 |
Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.) |
VGX-3100 |
DNA-based immunotherapy |
Cervical dysplasia caused by human papillomavirus |
Said it satisfied the FDA's request for additional information regarding the Celletra 5PSP delivery device and the agency removed the clinical hold; patient recruitment is set to start for its phase III program |
6/9/17 |
Kalobios Pharmaceuticals Inc. (Brisbane, Calif.) |
Benznidazole |
Oral antiparasitic |
Chagas disease |
Said the IND for benznidazole is active |
6/28/17 |
Redhill Biopharma Ltd. (Tel Aviv, Israel) |
Talicia |
RHB-105 |
H. pylori infections |
Started the phase III ERADICATE Hp 2 trial |
6/16/17 |
Inflammatory | |||||
Ampio Pharmaceuticals Inc. (Englewood, Colo.) |
Ampion |
Low molecular weight fraction of human serum albumin |
Kellgren-Lawrence grade IV osteoarthritis of the knee |
The first patient was dosed in the phase III AP-003-C study |
6/23/17 |
Miscellaneous | |||||
Biogen Inc. (Cambridge, Mass.) |
Spinraza |
Nusinersen |
Spinal muscular atrophy |
New analysis from phase III trials testing Spinraza showed that, in the ENDEAR trial, a greater portion of those treated with Spinraza demonstrated clinical benefits compared to untreated infants; data from the ENDEAR and CHERISH studies demonstrated that early treatment may lead to improved outcomes since infants in ENDEAR who were diagnosed earlier demonstrated a lower risk of death or permanent ventilation compared to untreated individuals; likewise, children with shorter disease durations in CHERISH demonstrated greater motor function improvement from baseline to 15 months compared to untreated individuals |
6/30/17 |
Bone Therapeutics SA (Gosselies, Belgium) |
Preob |
Autologous bone cell therapy |
Osteonecrosis of the hip |
Completed the recruitment of 44 treated patients required for the planned interim analysis of the phase III trial |
6/9/17 |
Durect Corp. (Cupertino, Calif.) |
Posimir |
Uses Durect's Sucrose Acetate isoButyrate Extended Release (SABER) technology to continuously deliver bupivacaine |
Cholecystectomy (gallbladder removal) surgery |
Completed enrollment in Persist, its pivotal phase III trial |
6/23/17 |
Histogenics Corp. (Waltham, Mass.) |
Neocart |
Cartilage-like, tissue engineered implant created from a patient's own cartilage cells |
Articular cartilage defects |
Completed enrollment in the 245-patient phase III trial |
6/29/17 |
Lipocine Inc. (Salt Lake City) |
LPCN-1021 |
Testosterone replacement therapy |
Male hypogonadism |
In the Dosing Validation (DV) study, 81% of patients achieved average testosterone levels within the normal range with a lower bound confidence interval of 72%; the Dosing Flexibility (DF) study restored 70% of the subjects' average testosterone levels within the normal range, confirming that twice-daily dosing without titration is the appropriate dosing regimen for LPCN-1021 |
6/21/17 |
Mallinckrodt plc (Staines-upon-Thames, U.K.) |
Stratagraft |
Regenerative skin tissue |
Promotion of autologous skin regeneration of complex skin defects due to thermal burns that contain intact dermal elements |
Enrolled the first patient in its phase III study |
6/8/17 |
Mithra Pharmaceuticals SA (Liege, Belgium) |
Estelle |
15 mg of Estetrol and 3 mg of drospirenone |
Contraceptive |
Completed recruitment into a safety study |
6/22/17 |
Neovii Pharmaceuticals AG (Rapperswil, Switzerland) |
Grafalon |
Anti-human T-lymphocyte immunoglobulin |
Graft-vs.-host disease (GvHD) |
Long-term outcomes of a trial conducted in Europe and Israel, looking at patients after standard GvHD prophylaxis with cyclosporine A and methotrexate with or without Grafalon in adult patients receiving myeloablative conditioning prior to hematopoietic stem cell transplantation from matched unrelated donors, showed that severe GvHD-free and relapse-free survival was 34% in the Grafalon group vs. 13% in the non-Grafalon group; the probability of being alive and free of immunosuppressive therapy was 47% in the Grafalon group vs. 11% in the non-Grafalon group |
6/27/17 |
Novartis AG (Basel, Switzerland) |
RTH-258 |
Brolucizumab; VEGF-A ligand inhibitor |
Neovascular age-related macular degeneration |
It met the primary and key secondary endpoints – noninferiority to aflibercept in mean change in best-corrected visual acuity (BCVA) from baseline to week 48 and average mean change over weeks 36 to 48 – in the phase III HAWK and HARRIER studies; brolucizumab showed longer-lasting efficacy compared to aflibercept, dosed every eight weeks; most patients – 57% of those treated in HAWK and 52% in HARRIER – were maintained on a regimen of brolucizumab, dosed every 12 weeks immediately following the loading phase through week 48 |
6/21/17 |
Oncbiomune Pharmaceuticals Inc. (Baton Rouge, La.) |
Andfrt |
Male fertility product |
Asthenozoo-spermia |
It demonstrated a clinically meaningful benefit |
6/7/17 |
Psivida Corp. (Watertown, Mass.) |
Durasert |
Fully bioerodible, long-term, sustained-release implant delivering latanoprost |
Posterior segment uveitis |
The second phase III study of Durasert three-year treatment achieved the primary endpoint by demonstrating a significant reduction in the recurrence of posterior segment uveitis through six months; 21.8% of Durasert-treated patients had a recurrence compared to 53.8% of patients in the sham group (p<0.001) |
6/14/17 |
Strongbridge Biopharma plc (Dublin) |
Recorlev |
Levoketoco-nazole |
Endogenous Cushing's syndrome |
Completed enrollment of its 90-patient phase III SONICS trial |
6/28/17 |
Sylentis SA (Madrid) |
SYL-1001 |
RNAi candidate |
Dry eye syndrome |
Started the first phase III study, HELIX |
6/2/17 |
Respiratory | |||||
Aimmune Therapeutics Inc. (Brisbane, Calif.) |
AR-101 |
Biologic oral immunotherapy |
Desensitization of patients with peanut allergy |
In the ongoing phase III PALISADE trial, more than 97% of patients currently have completed up-dosing |
6/14/17 |
Asit biotech SA (Brussels) |
gp-ASIT+ |
Mixture of natural allergen fragments obtained from a purified specific proteinic extract from Lolium perenne pollen |
Grass pollen rhinitis (hay fever) |
Results of its BTT009 phase III study showed it improved the combined clinical symptom and medication score (CSMS) in allergic rhinitis patients by 15.5% at peak pollen period compared to placebo; when measured over the entire pollen season the CSMS improved by 17.9% compared to placebo |
6/20/17 |
Pharmaxis Ltd. (Sydney) |
Bronchitol |
Mannitol |
Cystic fibrosis |
The international phase III trial met its primary endpoint, demonstrating the superiority of Bronchitol vs. the comparator on the primary endpoint of FEV1 change from baseline over a 26-week treatment period, with an effect of 54 mL (p = 0.020), corresponding to a 2.2% relative change (p = 0.025) |
6/14/17 |
Stallergenes Greer (London) |
Grass pollen sublingual immunotherapy (SLIT) treatment |
SLIT tablets |
Allergic rhinitis and allergic asthma |
Findings from a real-world evidence study on the long-term effectiveness, based on eight years of data, suggest that grass pollen SLIT tablets, including Oralair, improved control of allergic rhinitis and may offer a preventive effect on allergic asthma onset and worsening compared to symptomatic drug therapy; SLIT treatment was associated with a relative risk reduction of about 30% with respect to developing asthma throughout the treatment period and a relative risk reduction of about 40% during the follow-up period, compared to symptomatic treatment |
6/20/17 |
Vertex Pharmaceuticals Inc. (Boston) |
Orkambi |
Lumacaftor/ivacaftor |
Cystic fibrosis |
Results from a phase III study in children with CF, ages 6 through 11, who have two copies of the F508del mutation, showed the study met its primary endpoint of absolute change in lung clearance index (LCI2.5) through 24 weeks of treatment, demonstrating a statistically significant improvement in LCI2.5 among those treated with Orkambi compared to placebo |
6/12/17 |
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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. | |||||
