Company (location)







Advaxis Inc., of Princeton, N.J.

Axalimogene filolisbac

Listeria-based immunotherapy

High-risk, locally advanced cervical cancer

Evaluating the possibility of accelerating the interim analysis timeline and establishing a more stringent futility boundary; company anticipates that over the next couple of months it will finalize the redesign of the trial and review it with the FDA


Astrazeneca plc, of Cambridge, U.K.

Imfinzi (durvalumab)

Monoclonal antibody targeting PD-L1

Previously-untreated patients with stage IV non-small-cell lung cancer

In the Mystic study, Imfinzi monotherapy and Imfinzi plus tremelimumab didn't improve overall survival compared to standard of care chemotherapy by a statistically significant margin (HR=0.76 and 0.85, respectively)


Aveo Oncology Inc., of Cambridge, Mass.  



Renal cell carcinoma

TIVO-3 trial comparing Fotivda to sorafenib in 351 subjects with highly refractory advanced or metastatic disease met primary endpoint of demonstrating a statistically significant benefit in progression-free survival, with 44% improvement in median PFS and 26% reduction in risk of progression or death (p=0.02); median PFS was 5.6 months for tivozanib vs. 3.9 months for sorafenib; secondary endpoint of overall survival (OS) was not mature at time of final PFS analysis, with only 46% of potential OS events having been reported; final survival analysis per protocol is planned for August 2019


Beigene Ltd., of Beijing


BTK inhibitor

Relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma

Dosed the first patient; primary endpoint of 400-patient study is overall response rate


Bio-Thera Solutions Ltd., of Guangzhou, China


HER2-targeting antibody-drug conjugate

HER2-positive metastatic breast cancer

Started Chinese study in patients who previously received trastuzumab separately or in combination with taxanes; trial will compare BAT-8001 vs. lapatinib combined with capecitabine


Blue Earth Diagnostics Inc., of Burlington, Mass.

18F-fluciclovine positron emission tomography

Synthetic amino acid labeled with the radioisotope F18


Results demonstrated the overall ability of the readers to assess the diagnostic performance of 18F-fluciclovine with magnetic resonance imaging had a positive predictive value of more than 90%


Bone Therapeutics SA, of Gosselies, Belgium


Autologous osteoblastic cell therapy


Data and safety monitoring board said interim results suggest it is unlikely the primary objective will be achieved and recommended trial discontinuation; analysis was based on 44 patients with a 12-month follow-up


Bristol-Myers Squibb Co., of Princeton

Opdivo (nivolumab) and Yervoy (ipilimumab)

PD-1 inhibitor and CTLA-4 inhibitor

Small-cell lung cancer

CheckMate-451 study showed combo did not meet primary endpoint of overall survival vs. placebo as a maintenance therapy for patients with extensive-stage SCLC without disease progression after completion of first-line platinum-based chemotherapy


Deciphera Pharmaceuticals Inc., of Waltham, Mass.


KIT and PDGFRalpha inhibitor

Fourth-line and fourth-line-plus gastrointestinal stromal tumor

Completed enrollment in the placebo-controlled Invictus study measuring progression-free survival as the primary endpoint; top-line data expected in mid-2019


Foresee Pharmaceuticals Co. Ltd., of Taipei, Taiwan

FP-001 (LMIS 25 mg)

Leuprolide mesylate injectable suspension

Advanced prostate cancer

All patients completed participation in the single-arm study; 144 patients enrolled to receive 2 consecutive injections for aggregated treatment duration of 6 months; top-line data expected in the first quarter of 2019


Glycomimetics Inc., of Rockville, Md.

Uproleselan (GMI-1271)

Blocks E-selectin from binding with blood cancer cells 

Relapsed/refractory acute myeloid leukemia

First patient dosed


Halozyme Therapeutics Inc., of San Diego


Pegylated form of recombinant human hyaluronidase

Metastatic pancreas cancer

FDA agreed to company's request to change primary endpoint of the HALO-301 study to single endpoint of overall survival; planned interim analysis will not be conducted


Hutchison China Meditech Ltd., of London


Inhibitor of VEGFR 1, 2 and 3

Third-line advanced non-small-cell lung cancer

In the Faluca study, drug didn't improve overall survival compared to placebo; there was a statistically significant improvement in all secondary endpoints including progression-free survival, objective response rate, disease control rate and duration of response compared to placebo; data to be presented at an upcoming meeting


Isofol Medical AB, of Gothenburg, Sweden



Colorectal cancer

Started the ISO-CC-007 pivotal study to treat first-line (initial) metastatic colorectal cancer


Kura Oncology Inc., of San Diego


Inhibitor of farnesylation

HRAS-mutant head and neck squamous cell carcinoma

Initiated registration-directed trial to enroll 59 patients who received prior platinum-based therapy in the AIM-HN treatment cohort to evaluate objective response rate as the primary endpoint; SEQ-HN, a non-interventional screening and outcomes cohort, will serve as a case-control study, matching patients with recurrent or metastatic HNSCC with HRAS mutations against those who are HRAS wild-type


Merck & Co. Inc., of Darmstadt, Germany

Bavencio (avelumab)

PD-L1 blocker

Platinum-resistant/refractory ovarian cancer

Tested alone or in combination with pegylated liposomal doxorubicin (PLD) compared with PLD did not meet the prespecified primary endpoints of overall survival or progression-free survival 


Merck & Co. Inc., of Kenilworth, N.J.

Keytruda (pembrolizumab)

Anti-PD-1 antibody

Advanced or metastatic esophageal or esophagogastric junction carcinoma

KEYNOTE-181 trial met primary endpoint of overall survival in patients whose tumors expressed PD-L1 (Combined Positive Score [CPS] ≥10); treatment resulted in statistically significant improvement in OS vs. chemotherapy (paclitaxel, docetaxel or irinotecan) in patients with CPS ≥10, regardless of histology; in patients with squamous cell histology and in entire intention-to-treat (ITT) population, results were directionally favorable, though not statistically significant; key secondary endpoints of progression-free survival and objective response rate were not formally tested, as OS was not reached in the full ITT study population


Molecular Partners AG, of Zurich, Switzerland


Multi-DARPin therapeutic that binds to HGF and VEGF

Multiple myeloma

Data from ongoing study in combination with bortezomib and dexamethasone in patients who have failed standard therapies show durable responses in several patients with immediately previous proteasome inhibitor-based treatment


Pledpharma AB, of Stockholm



Colorectal cancer

Enrolled first patient in global program comprising 2 trials; POLAR-M will enroll 420 patients receiving chemo for metastatic colorectal cancer, while POLAR-A will comprise 280 patients undergoing adjuvant chemo for colorectal cancer


Rafael Pharmaceuticals Inc., of Newark, N.J.

CPI-613 (devimistat)

Targets altered regulation of metabolic processes specific to cancer cells

Relapsed or refractory acute myeloid leukemia

Started the pivotal ARMADA 2000 trial testing CPI-613 plus high-dose cytarabine and mitoxantrone in patients 60 and older with an ECOG performance status of 0 or 2; primary endpoint is complete remission, and secondary endpoints include overall survival and complete remission with partial hematologic recovery


Tracon Pharmaceuticals Inc., of San Diego

Carotuximab (TRC-105)

Antibody targeting endoglin


In the blinded study, 51 patients treated for 6 weeks, 35% of patients had a greater than 2-fold reduction in endoglin+ circulating tumor cells (CTCs), including 25% with a greater than 10-fold reduction; 37% had a 2-fold increase in endoglin+ CTCs, including 25% with a greater than 10-fold increase; 27% had no significant change in endoglin+ CTCs; interim analysis expected in the first quarter of 2019


Urogen Pharma Ltd., of Ra'anana, Israel


intravesical gel
formulation of
mitomycin C

Low-grade upper tract urothelial cancer

Completed enrollment of Olympus study; plans to begin rolling NDA submission to the FDA in 4Q2018 and complete the submission in the 2Q2019


VBL Therapeutics Ltd., of Tel Aviv, Israel

Ofranergene obadenovec (VB-111)

Vascular targeted drug

Recurrent glioblastoma

In the failed Globe study, VB-111 was administered in combination with Avastin (bevacizumab, Roche AG) rather than treating with VB-111 first and then Avastin after progression as occurred in the successful phase II study



Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y.

Praluent (alirocumab)

PCSK9-inhibiting antibody


Data published in The New England Journal of Medicine from 18,924-patient ODYSSEY OUTCOMES trial showed it met the primary endpoint, with Praluent significantly reducing risk of major cardiovascular events (MACE) in patients who had suffered acute coronary syndrome; MACE occurred in 903 patients (9.5%) in the Praluent group and in 1,052 patients (11.1%) in the placebo group (p<0.001)



Foamix Pharmaceuticals Ltd., of Rehovot, Israel


1.5% minocycline foam

Moderate to severe rosacea

Top-line data showed studies FX2016-11 and FX2016-12 met both co-primary endpoints of absolute change from baseline in inflammatory lesion count at week 12 and Investigator Global Assessment (IGA) treatment success at week 12, defined as an IGA score of 0 or 1, and at least a 2-grade improvement (decrease) from baseline


Shire plc, of Dublin

Takhzyro (lanadelumab)

Monoclonal antibody targeting plasma kallikrein

Hereditary angioedema

A post-hoc sensitivity analysis of the 26-week Help study found the reduction in attacks was greatest for patients during the 16-week steady state period (days 70-182)


Shire plc, of Dublin


Monoclonal antibody that inhibits plasma kallikrein

Hereditary angioedema

Data from HELP study published in the Journal of the American Medical Association showed trial met all primary and secondary endpoints, with all 3 regimens demonstrating statistically significant reductions in the mean monthly HAE attack rate vs. placebo; at 300 mg every 2 weeks, number of mean monthly HAE attacks reduced by 87% relative to placebo (adjusted p<0.001); those on 300 mg every 2 weeks had 83% fewer moderate to severe attacks vs. placebo, 87% fewer attacks that needed on-demand treatment vs. placebo and an 89% attack rate reduction vs. placebo from day 14 to 182



Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis

Jardiance (empagliflozin)

SGLT2 inhibitor

Type 2 diabetes

Initial results from real-world EMPRISE study showed treatment associated with a 44% relative risk reduction in hospitalization for heart failure vs. DPP-4 inhibitors in routine clinical practice in the U.S.


Corcept Therapeutics Inc., of Menlo Park, Calif.


Selective glucocorticoid receptor II antagonist

Cushing's syndrome

First patient dosed


Eli Lilly and Co., of Indianapolis

Trulicity (dulaglutide)

GLP-1 receptor agonist

Type 2 diabetes

Significantly reduced major adverse cardiovascular events, meeting the primary endpoint in the REWIND trial


Xeris Pharmaceuticals Inc., of Chicago

Liquid glucagon pen

Ready-to-use, room-temperature stable glucagon

Type 1 diabetes

In a crossover study comparing Xeris glucagon pen to the Glucagon Emergency Kit (GEK), the average time to symptom relief was comparable for autonomic symptoms (9.9 min vs. 9.8 min), neuroglycopenic symptoms (10.3 min vs. 9.9 min) and average total symptoms (13 min vs. 11.9 min); the drug more quickly resolved global hypoglycemia symptoms compared to GEK (11.6 min vs. 13.1 min); in a usability study, 14 of 16 participants (87.5%) successfully administered a complete injection using the glucagon pen vs. 5 of 16 (31.3%) using the GEK, and the administration was faster for the glucagon pen (47.9 seconds) vs. GEK (109.0 seconds); in a pediatric study of 31 patients with hypoglycemia, the pen produced mean increase in glucose of 23.3 mg/dL at 15 minutes and 42.2 mg/dL at 20 minutes


Genitourinary/Sexual Health

Antares Pharma Inc., of Ewing, N.J.

Xyosted (testosterone enanthate)

Androgen therapy

Testosterone replacement

26-week data identified increases in systolic blood pressure from baseline to week 26 of 125.6 mmHg to 129 mmHg (3.4 mmHg) and increases in diastolic blood pressure from 78.2 mmHg at baseline to 80 mmHg (1.8 mmHg); patients with hypertension at baseline appeared to experience less impact on blood pressure following treatment


Calliditas Therapeutics AB, of Stockholm


Oral formulation of budesonide

IgA nephropathy

Enrolled first patient in the pivotal study testing Nefecon vs. placebo on proteinuria; top-line data expected in the second half of 2020


Mallinckrodt plc, of Staines-upon-Thames, U.K.


V1 selective vasoconstrictor

Hepatorenal syndrome type 1

Post-hoc pooled analysis from two placebo-controlled studies showed treatment was associated with improved overall and transplant-free survival with lower baseline mean arterial pressure; that effect was seen independent of HRS reversal; key findings included overall survival at 90 days in the MAP <65 mm Hg group that was significantly higher among patients receiving terlipressin (17/25 [68%]) vs. placebo (6/25 [24%]): survival estimate was 0.680 vs. 0.209, respectively (p=0.005); no difference in OS at 90 days was observed between terlipressin (66/128 [51.6%]) and placebo (72/129 [55.8%]) in the MAP ≥65 mm Hg group: survival estimate was: 0.515 vs. 0.554, respectively (p=0.429)


Mithra Pharmaceuticals SA, of Liege, Belgium

Estelle (estetrol 15 mg/drospirenone 3 mg)

Combination oral contraceptive


Completed study, with top-line data on track to be reported in the first quarter of 2019


Obseva SA, of Geneva


Oral oxytocin receptor antagonist

Improving rates of pregnancy and live birth in patients undergoing assisted reproduction technology or in vitro fertilization

Started the IMPLANT 4 study; placebo-controlled trial will enroll about 800 patients undergoing IVF cycle with a day 5 single embryo transfer; primary endpoint is the proportion of patients successfully achieving ongoing pregnancy 10 weeks post embryo transfer, and live birth rate is a secondary endpoint


Urovant Sciences Ltd., of Basel, Switzerland


Oral, once-daily, small-molecule beta-3 agonist

Overactive bladder

Completed enrollment in EMPOWUR study testing treating in adults with symptoms of OAB; top-line data are expected by the end of March 2019



Aimmune Therapeutics Inc., of Brisbane, Calif.


Biologic oral immunotherapy 

Peanut allergy

PALISADE trial met its primary endpoint and key secondary endpoints; in primary analysis of peanut-allergic children and adolescents ages 4-17, AR-101 treatment resulted in a significant increase in the amount of peanut protein tolerated, compared to placebo; results published in The New England Journal of Medicine


DBV Technologies SA, of Montrouge, France

Viaskin Peanut

Epicutaneous immunotherapy

Peanut allergy

New data from pivotal PEPITES study show a majority of patients experience increase in peanut threshold reactivity after the first 12 months of treatment, with 62.6% increasing their peanut eliciting dose vs. 28% in the placebo group; an odds ratio (OR) analysis indicated that patients treated with active therapy were 4.3 times more likely to improve their peanut threshold reactivity level vs. placebo (OR = 4.3, p<0.001)


Medday Pharmaceuticals SAS, of Paris


Formulation of high-dose pharmaceutical-grade biotin

Progressive multiple sclerosis

Full enrollment completed in SPI2 study


Stallergenes Greer plc, of London


Sublingual allergy immunotherapy tablet

House dust mite-induced allergic rhinitis

Study met primary endpoint, indicating statistically significant reduction of the Total Combined Score, the sum of the Rhinitis Total Symptom Score and the Rescue Medication Score vs. placebo; also reached key secondary endpoints, including overall quality of life, and showed treatment was generally well-tolerated



Enanta Pharmaceuticals Inc., of Watertown, Mass., and Abbvie Inc., of North Chicago

Mavyret (glecaprevir/pibrentasvir)

NS5A inhibitor/NS3/4A protease inhibitor

Hepatitis C virus

Results from phase IIIb EXPEDITION-8 trial in treatment-naïve patients with compensated cirrhosis showed with 8 weeks of treatment, 100% of genotypes 1, 2, 4, 5 and 6 patients achieved sustained virologic response 12 weeks after treatment



Kolon Tissuegene Inc., of Rockville, Md.


Cell and gene therapy

Knee osteoarthritis

Dosed the first patient in its pivotal U.S. trials; studies will enroll up to 1,020 patients and will assess pain and function endpoints as well as MRI, x-ray and liquid biomarkers


Principia Biopharma Inc., of South San Francisco


Oral, small-molecule, reversible covalent BTK inhibitor


Pegasus trial begun



Alkermes plc, of Dublin

ALKS-3831 (olanzapine/samidorphan)

Oral, atypical antipsychotic


Top-line results from the ENLIGHTEN-2 pivotal trial show drug met pre-specified co-primary endpoints, demonstrating both a lower mean percent weight gain from baseline at 6 months vs. olanzapine group (p=0.003) and a lower proportion of patients who gained 10% or more of their baseline body weight at 6 months vs. olanzapine (p=0.003); NDA submission expected in mid-2019


Biohaven Pharmaceutical Holding Company Ltd., of New Haven, Conn,


CGRP receptor antagonist

Migraine prevention

Enrolled first patient in study measuring the change in the mean number of migraine days per month from baseline at week 12; secondary endpoints include the achievement of at least a 50% reduction from baseline in mean monthly migraine days and the mean number of rescue medication days per month; top-line data expected in 2019


GW Pharmaceuticals plc, of London



Seizures associated with Dravet syndrome

Top-line results showed second phase III trial achieved primary endpoint of reduction in convulsive seizures for both dose levels (49% for 10 mg/kg per day and 46% for 20 mg/kg per day vs. 27% reduction for placebo; p=0.0299 and p=0.0095, respectively); both doses also demonstrated statistically significant improvements on all key secondary endpoints


Tonix Pharmaceuticals Holding Corp., of New York 



Post-traumatic stress disorder 

Results and retrospective analyses from the P301 study (HONOR) revealed treatment effect in those who experienced trauma



Acucela Inc., of Seattle, a subsidiary of Tokyo-based Kubota Pharmaceutical Holdings Co. Ltd.

Emixustat hydrochloride

RPE65 inhibitor

Macular atrophy secondary to Stargardt disease

Enrolled first patient in a 160-patient placebo-controlled study measuring the rate of macular atrophy progression; changes in visual function parameters such as best-corrected visual acuity letter score and reading speed will also be measured


Clearside Biomedical Inc., of Alpharetta, Ga.

Xipere (suprachoroidal CLS-TA)

Suspension of corticosteroid triamcinolone acetonide formulated for administration to back of the eye

Retinal vein occlusion

Primary endpoint of SAPPHIRE trial not achieved in study testing combination of Xipere and Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) vs. Eylea monotherapy


Eyenovia Inc., of New York


Fixed-combination phenylephrine 2.5%/tropicamide 1% ophthalmic solution administered using Optejet


Enrolled the first patient in the first of 2 phase III trials (MIST-1 and MIST-2); top-line data expected in the first half of 2019



Endo International plc, of Dublin


Collagenase clostridium histolyticum


Results from 2 identical RELEASE studies showed subjects receiving CCH showed highly statistically significant levels of improvement in appearance of cellulite, as measured by the primary endpoint (RELEASE-1, p=0.006; RELEASE-2, p=0.002), which was at least a 2-level composite improvement in cellulite severity in the target buttock at day 71 vs. placebo.; RELEASE-1 passed 8 of 8 key secondary endpoints and RELEASE-2 passed 7 of 8


Gelesis Inc., of Boston


Non-systemic, superabsorbent hydrogel

Weight loss

Expanded data from GLOW study showed in intent-to-treat population, 59% of treated adults achieved weight loss of at least 5% vs. 42% in the placebo group; treated adults fell into 2 groups, the responders (6 of 10) who lost an average of 10% of their total body weight (about 22 pounds) and nearly 4 inches from their waists, and nonresponders who lost an average of 1% of total body weight (about 2 pounds; complete treatment group demonstrated superiority vs. placebo (-6.4% vs. -4.4%, p=0,0007)



Vectura Group plc, of Chippenham, U.K.


Drug-device combination consisting of budesonide delivered by Vectura's nebulizer inhalation system

Severe uncontrolled asthma

Study did not meet primary endpoint; top-line data indicated a trend in reduction of annualized rate of clinically significant exacerbations among those receiving VR-475 1 mg or 0.5 mg twice daily over 52-week treatment period but did not reach statistical significance vs. placebo; open-label arm with conventional nebulizer also failed to reach statistical significance vs. placebo


Vertex Pharmaceuticals Inc., of Boston

VX-659, tezacaftor and ivacaftor

Triple-combination CFTR correctors

Cystic fibrosis

Data showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in 2 studies; pre-specified interim analysis in people with 1 F508del mutation and 1 minimal function mutation showed mean absolute improvement in ppFEV1 of 14 percentage points from baseline at week 4 vs. placebo (p<0.0001); in study in people with 2 F508del mutations, the addition of VX-659 resulted in mean absolute improvement in ppFEV1 of 10 percentage points from baseline at week 4 vs. the control group (placebo added to tezacaftor and ivacaftor, p<0.000)



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