Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Alkermes plc (Dublin)

ALKS-8700

Oral monomethyl fumarate prodrug candidate

Relapsing-remitting multiple sclerosis

Started a new phase III study comparing it to Cambridge, Mass.-based Biogen Inc.'s Tecfidera (dimethyl fumarate)

3/17/17

Allergan plc (Dublin)

Oral sarecycline

Narrow-spectrum tetracycline-derived antibiotic

Moderate to severe acne

Positive data from dual phase III studies showed it proved statistically significantly superior to placebo on two efficacy endpoints: an Investigators Global Assessment (IGA) scale score and inflammatory lesion counts (p < 0.004)

3/28/17

Coherus Biosciences Inc. (Redwood City, Calif.)

CHS-1420

Biosimilar of Humira (adalimumab, Abbvie Inc.)

Psoriasis

It met the primary endpoint in a clinical pharmacokinetic (PK) bioequivalence study that compared it to Humira in healthy subjects; the study met the criteria for clinical PK similarity on all prospectively defined PK endpoints: maximum serum concentration, area under the time-concentration curve from first to last time point measured and area under the time-concentration curve from first time point extrapolated to infinity; the 90% confidence intervals of the geometric mean ratios for all PK endpoints fell well within the bioequivalence boundaries of 80% to 125%

3/3/17

Eli Lilly and Co. (Indianapolis)

Taltz

Ixekizumab; targets IL-17a

Moderate to severe plaque psoriasis

The IXORA-S study showed that, at 24 weeks, patients treated with Taltz (ixekizumab) achieved higher response rates compared to patients treated with Stelara (ustekinumab, Johnson & Johnson), including 83% of patients who achieved Psoriasis Area Severity Index (PASI) 90 – the study's primary endpoint – compared to 59% of patients who achieved PASI 90 after treatment with Stelara; the study also found that 91.2% of patients treated with Taltz achieved PASI 75 compared to 81.9% of patients treated with Stelara (p=0.015), and 49.3% of patients treated with Taltz achieved PASI 100 compared to 23.5% of patients treated with Stelara (p=0.001)

3/7/17

Foamix Pharmaceuticals Ltd. (Rehovot, Israel)

FMX-101

4% minocycline foam

Moderate to severe acne

Top-line data from two late-stage studies showed it demonstrated statistical significance compared to vehicle on both co-primary endpoints in Trial 05, but did not demonstrate statistical significance on one of the two co-primary endpoints in Trial 04, specifically IGA success

3/28/17

Johnson & Johnson (New Brunswick, N.J.)

Guselkumab

Anti-interleukin 23 candidate

Plaque psoriasis

In a second pivotal trial, it outperformed Abbvie Inc.'s Humira (adalimumab), meeting its co-primary endpoints at week 16, with 84.1% of patients receiving guselkumab and 67.7% of patients receiving Humira achieving an Investigator's Global Assessment score of cleared or minimal disease

3/6/17

Novartis AG (Basel, Switzerland)

Cosentyx

Secukinumab; fully human, targeted treatment inhibits the IL-17A cytokine

Moderate to severe psoriasis

A new analysis showed that patients treated with Cosentyx rapidly regained clear or almost clear skin following relapse during a treatment pause; by week 16 of retreatment, 94% of patients regained a PASI 75 score, 79% of prior PASI 90 responders regained a PASI 90 score and 67% of prior PASI 100 response regained a PASI 100 score

3/7/17

Novartis AG (Basel, Switzerland)

Cosentyx

Secukinumab

Moderate-to-severe psoriasis

New data suggests patients with continuous treatment maintained their high level of response; among the patients that discontinued treatment, 21% maintained skin clearance for up to one year without treatment and 10% maintained skin clearance after two years without treatment

3/22/17

Redhill Biopharma Ltd. (Tel-Aviv, Israel)

RHB-104

Antibiotic combination therapy

Crohn's disease

The first patient was dosed in the open-label extension of a phase III study

3/22/17

Sandoz (division of Novartis AG; Basel, Switzerland)

GP-2017

Biosimilar adalimumab

Moderate to severe, chronic plaque psoriasis

The confirmatory phase III study met its primary endpoint by demonstrating equivalent efficacy to the reference medicine, Humira (Abbvie Inc.); the primary endpoint of the study was the proportion of patients who achieved a 75% improvement at week 16, as measured by the Psoriasis Area and Severity Index (PASI)

3/7/17

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

Dupixent

Dupilumab

Atopic dermatitis (AD)

Results from the one-year phase III CHRONOS study showed that, at 52 weeks, the mean improvement in EASI from baseline was 80% for patients who received Dupixent weekly and 78% for patients who received Dupixent every two weeks compared to 46% for patients on placebo (p < 0.0001)

3/7/17

Tigenix NV (Leuven, Belgium)

Cx-601

Allogeneic stem cell product

Crohn's disease

Received feedback from the FDA on an improved protocol for its global phase III trial testing Cx601; the agency agreed a BLA could be filed based on the efficacy and safety follow-up of patients assessed at week 24, instead of week 52; furthermore, the FDA has agreed to accept fewer patients than originally planned in the study, and has endorsed a broader target population that will ultimately facilitate the recruitment process

3/8/17

CANCER

Actinium Pharmaceuticals Inc. (New York)

Iomab-B

CD45-targeting monoclonal antibody

Relapsed or refractory acute myeloid leukemia

The EMA commented that the trial design, primary endpoint and planned statistical analysis of the U.S. pivotal phase III SIERRA trial are acceptable and can serve as the basis for submission of a marketing authorization application

3/22/17

Amgen Inc. (Thousand Oaks, Calif.)

Kyprolis

Carfilzomib

Relapsed or refractory multiple myeloma

Results from a planned overall survival interim analysis of the phase III head-to-head ENDEAVOR trial showed it met the key secondary endpoint of OS, demonstrating that patients with relapsed or refractory multiple myeloma treated with Kyprolis and dexamethasone (Kd) lived 7.6 months longer than those treated with Velcade (bortezomib, Takeda Oncology Inc.) and dexamethasone (Vd) (median OS 47.6 months for Kd vs. 40 for Vd)

3/1/17

Amgen Inc. (Thousand Oaks, Calif.)

Blincyto

Blinatumomab; bispecific CD19-directed CD3 T-cell engager antibody

Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia

Results from the phase III TOWER study evaluating the efficacy of Blincyto vs. standard-of-care (SOC) chemotherapy in high-risk adult patients showed that median overall survival (OS) was 7.7 months for Blincyto vs. four months for SOC (p=0.012); data also showed the survival benefit for Blincyto was independent of allogeneic stem cell transplant (alloSCT), as the median OS, censored at the time of alloSCT, was 6.9 months for the drug vs. 3.9 months for SOC; evaluation of key secondary endpoints showed that remission rates were also higher for Blincyto vs. SOC (34% vs. 16%), and patients receiving Blincyto also had a higher rate of combined complete remission or complete remission with partial or incomplete hematologic recovery (44% vs. 25%)

3/3/17

Amgen Inc. (Thousand Oaks, Calif.)

Kyprolis

Carfilzomib

Relapsed or refractory multiple myeloma

Results from a planned overall survival (OS) interim analysis of the phase III head-to-head ENDEAVOR trial showed it met the key secondary endpoint of OS, demonstrating that patients treated with Kyprolis and dexamethasone (Kd) lived 7.6 months longer than those treated with Velcade (bortezomib) and dexamethasone (Vd) (median OS 47.6 months for Kd versus 40.0 for Vd, HR=0.79; 95% CI: 0.65 – 0.96; p=0.01)

3/7/17

Amgen Inc. (Thousand Oaks, Calif.)

Xgeva

Denosumab

Multiple myeloma

Phase III showed it met the primary endpoint, demonstrating non-inferiority to zoledronic acid in delaying the time to first on-study skeletal-related event (SRE) (HR=0.98, 95% CI: 0.85, 1.14; p=0.01);

3/7/17

Armo Biosciences Inc. (Redwood City, Calif.)

AM-0010

Pegylated interleukin-10

Advanced pancreatic cancer

The first patient was dosed in the pivotal phase III trial to evaluate it in combination with FOLFOX (folinic acid, 5-fluorouracil and oxaliplatin)

3/30/17

Arqule Inc. (Burlington, Mass.)

Tivantinib

Small-molecule c-Met receptor tyrosine kinase inhibitor

c-Met diagnostic-high inoperable hepatocellular carcinoma

Said partner Kyowa Hakko Kirin Co. Ltd., of Tokyo, reported top-line results for the JET-HCC phase III trial in Japan, which did not meet its primary endpoint of progression-free survival; the study enrolled about 190 Japanese patients with a history of prior Nexavar (sorafenib, Amgen Inc. and Bayer AG) therapy, to evaluate the efficacy and safety of tivantinib

3/28/17

Astrazeneca plc (Wilmington, Del.)

Lynparza

Olaparib

Ovarian cancer

Results from the phase III SOLO-2 trial demonstrated a significant improvement in progression-free survival (PFS) in germline BRCA-mutated, platinum-sensitive, relapsed ovarian cancer patients; the trial met its primary endpoint of investigator-assessed PFS (p<0.0001; median 19.1 months vs. 5.5 months); PFS as measured by blinded independent central review evaluation, a pre-specified sensitivity analysis supporting the primary endpoint, demonstrated a median PFS of 30.2 months vs. 5.5 months for placebo, representing an improvement of 24.7 months (p<0.0001)

3/15/17

Beigene Ltd. (Beijing)

BGB-3111

BTK inhibitor

Relapsed or refractory mantle cell lymphoma

Dosed the first patient in a pivotal trial in Chinese patients

3/3/17

Beigene Ltd. (Beijing)

BGB-3111

Bruton's tyrosine kinase inhibitor

Relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma

The first patient was dosed in a pivotal trial in Chinese patients

3/13/17

Cel-Sci Corp. (Vienna, Va.)

Multikine

Leukocyte interleukin, injection

Head and neck cancer

Received the official minutes from its Feb. 8 meeting with the FDA regarding the partial clinical hold placed on the phase III trial; the action items for the company to pursue include providing an updated investigator's brochure and current procedures for compliance with requirements under 21 CFR 312 Subpart D to address the partial hold and providing a list of major protocol deviations, along with a plan to identify major protocol deviations across all patients enrolled

3/7/17

Cornerstone Pharmaceuticals Inc. (Cranbury, N.J.)

CPI-613

Targets the mitochondrial tricarboxylic acid cycle

Acute myeloid leukemia (AML) and pancreatic cancer

Following a successful end-of-phase I type B meetings with the FDA, the company has a clear clinical and regulatory path forward to conduct pivotal trials of CPI-613

3/27/17

Eli Lilly and Co. (Indianapolis)

Abemaciclib

Selective ATP-competitive inhibitor of CDK4 and CDK6 kinases

Hormone-receptor-positive, HER2-negative breast cancer

Adding abemaciclib to the already-approved drug fulvestrant in the phase III Monarch 2 trial reduced the risk of disease worsening or death for women more than adding a placebo

3/21/17

Gamida Cell Ltd. (Jerusalem)

Nicord

A stand-alone graft derived from a single umbilical cord blood unit

Blood cancer

The first patient was transplanted in its phase III study of Nicord; about 120 patients, ages 16 to 60, will be enrolled, and the primary endpoint is the time to neutrophil engraftment

3/1/17

Genentech (unit of Roche Group; Basel, Switzerland)

Perjeta regimen

Pertuzumab; targets the HER2 receptor

HER2-positive early breast cancer

Said the Phase III APHINITY study met its primary endpoint and showed that adjuvant (after surgery) treatment with the combination of Perjeta, Herceptin (trastuzumab) and chemotherapy (the Perjeta-based regimen) achieved a statistically significant reduction in the risk of recurrence of invasive disease or death (invasive disease-free survival) compared to Herceptin and chemotherapy alone

3/2/17

Hutchison China Meditech Ltd. (Hong Kong)

Fruquintinib

VEGF and VEGF receptor inhibitor

Colorectal cancer

It met the primary endpoint in a phase III pivotal registration trial; it demonstrated a clinically meaningful and a statistically significant increase in overall survival (OS) in the intention-to-treat population of patients treated with fruquintinib plus best supportive care (BSC) as compared to patients treated with placebo plus BSC

3/6/17

Hutchison Medipharma Ltd. (Chi-Med; Shanghai)

Fruquintinib

Targeted VEGFR inhibitor

Colorectal cancer

The phase III FRESCO study in Chinese patients met primary and secondary endpoints of improving overall survival and progression-free survival

3/7/17

Immunocellular Therapeutics Ltd. (Los Angeles)

ICT-107

Dendritic cell-based vaccine

Newly diagnosed glioblastoma

Said its recent amendment to the phase III protocol for ICT-107 is being implemented in the U.S.; the amendment enables patients to be randomized 30 days after commencing screening procedures, accelerating the time to randomization by about two months

3/7/17

LSK Biopharma (Salt Lake City)

Apatinib

Small-molecule angiogenesis inhibitor

Advanced or metastatic gastric cancer

Enrolled the first patient in a global, multicenter phase III trial; the company expects to enroll approximately 459 patients across 95 sites in 12 countries in Europe, the U.S., South Korea, Japan and Taiwan

3/15/17

Myovant Sciences Inc. (Brisbane, Calif.)

Relugolix

Oral, once-daily, small-molecule gonadotropin-releasing hormone receptor antagonist

Advanced prostate cancer

Started a phase III trial, called HERO, to evaluate the safety and efficacy in men with androgen-sensitive advanced prostate cancer who require at least one year of continuous androgen deprivation therapy

3/3/17

Tesaro Inc. (Marlborough, Mass.)

Niraparib

Poly ADP-ribose polymerase inhibitor

Ovarian cancer

Results of several secondary endpoints from the company's phase III Engot-Ov16/Nova trial, including chemotherapy-free interval, time to second subsequent therapy, and progression-free survival, demonstrate the positive and durable treatment effect of niraparib in a broad population of patients, regardless of germline BRCA mutation status

3/14/17

TG Therapeutics Inc. (New York)

TG-1101

Ublituximab; glycoengineered anti-CD20 candidate

Chronic lymphocytic leukemia (CLL)

Its phase III study met its primary endpoint; previously treated high-risk CLL patients saw a significantly better overall response rate (ORR) when adding TG-1101 to Imbruvica (ibrutinib); the absolute difference in ORR attributed to the combination vs. Imbruvica alone was about 30% (p ≤ 0.001)

3/7/17

CARDIOVASCULAR

Acasti Pharma Inc. (Laval, Quebec)

Capre

Omega-3 phospholipid

Severe hypertriglyceridemia

Plans to proceed with its phase III development program, following the company's recent end-of-phase II meeting with the FDA

3/31/17

Amgen Inc. (Thousand Oaks, Calif.)

Repatha

Evolocumab

High cholesterol

Reported that patients receiving apheresis to reduce low-density lipoprotein cholesterol (LDL-C) during a phase III study of Repatha had a significantly reduced need for the procedure, meeting its primary endpoint; the trial also met its secondary endpoints of percent change from baseline to week four in LDL-C, non-high-density lipoprotein cholesterol and total cholesterol:HDL-C ratio

3/14/17

Amgen Inc. (Thousand Oaks, Calif.)

Repatha

Evolocumab

High cholesterol

Phase III FOURIER data showed when added to optimized statin therapy, Repatha led to an overall 15% risk reduction of a major adverse cardiovascular event (MACE) and reduced the rate of heart attacks by 27%, of stroke by 21%, and of stents and heart bypass procedures by 22%; it did not affect either the risk of cardiovascular death or hospitalization for chest pain

3/20/17

Amgen Inc. (Thousand Oaks, Calif.)

Repatha

Evolocumab; proprotein convertase subtilisin kexin type 9 inhibitor

High cholesterol

Reported detailed results from the cognitive function trial (EBBINGHAUS) evaluating 1,974 patients in the Repatha cardiovascular outcomes study, FOURIER; the study demonstrated that the effect of Repatha on the primary endpoint of executive function was non-inferior to placebo

3/21/17

Biocardia Inc. (San Carlos, Calif.)

Cell-based therapy

Designed to deliver a high dose of a patient's own bone marrow cells directly to the point of cardiac dysfunction

Ischemic heart failure

The first patient was treated in the pivotal phase III CardiAMP trial

3/1/17

Boehringer Ingelheim Pharmaceuticals Inc. (Ridgefield, Conn.)

Pradaxa

Dabigatran etexilate mesylate

Atrial fibrillation

Reported that, in patients undergoing catheter ablation, uninterrupted treatment with Pradaxa reduced the risk of major bleeding complications vs. uninterrupted warfarin during the company's Re-Circuit trial; the trial showed a 5.3% absolute risk reduction in its primary endpoint, with major bleeds occurring in five of 317 of patients receiving Pradaxa vs. 22 of 318 of patients receiving warfarin

3/21/17

Boehringer Ingelheim Pharmaceuticals Inc. (Ridgefield, Conn.; part of Boehringer Ingelheim GmbH) and Eli Lilly and Co. (Indianapolis)

Jardiance

Empagliflozin

Chronic heart failure

Initiated a dual phase III trial program, called Emperor HF; the trials will involve not only adults with type 2 diabetes who have heart failure, but also heart failure patients who do not have diabetes

3/20/17

CCM Duopharma Biotech Bhd (Kuala Lumpur, Malaysia)

PDA-10

Erythropoietin (EPO) biosimilar

Kidney failure

A phase III trial demonstrated equivalence to the reference produce in terms of pharmacokinetics, pharmacodynamics and toxicity

3/20/17

Daiichi Sankyo Co. Ltd. (Tokyo)

Edoxaban

Oral, once-daily direct factor Xa inhibitor

Atrial fibrillation (AF)

The first patient was enrolled in the ENTRUST-AF PCI phase IIIb trial designed to test a treatment regimen based on edoxaban against a vitamin K antagonist-based regimen in patients with AF following successful percutaneous coronary intervention with stent placement

3/7/17

Daiichi Sankyo Europe GmbH (Munich; unit of Daiichi Sankyo Co. Ltd.)

Edoxaban

Oral, once-daily direct factor Xa inhibitor

Atrial fibrillation in patients undergoing catheter ablation

The first patient was enrolled in the ELIMINATE-AF study, a randomized phase IIIb trial

3/31/17

Dalcor Pharmaceuticals Inc. (London and Montreal)

Dalcetrapib

Modulates plasma cholesteryl ester transfer protein activity, boosting high-density lipoproteins

Acute coronary syndrome (AA genotype in the ADCY9 gene)

Randomized more than 1,000 patients of the expected 5,000 for the phase III dal-Gene trial, a cardiovascular outcomes study in patients who have the AA genotype in the ADCY9 gene

3/8/17

Janssen Pharmaceuticals Inc. (part Johnson & Johnson; New Brunswick, N.J.)

Xarelto

Rivaroxaban

Venous thromboembolism (VTE)

Phase III data showed that patients taking Xarelto had significantly fewer recurrent blood clots and similar rates of major bleeding compared to those taking 100 mg of aspirin once daily; Xarelto 10 mg reduced the risk of recurrent VTE by 74%, while Xarelto 20 mg reduced it by 66%

3/21/17

Novartis AG (Basel, Switzerland)

Entresto

Sacubitril/valsartan

Chronic heart failure

Results of a post-hoc analysis in a subgroup of patients with reduced ejection fraction heart failure (HF) and diabetes suggests that it improved glycemic control, as assessed by hemoglobin A1c testing, compared to ACE-inhibitor enalapril

3/21/17

Novartis AG (Basel, Switzerland)

RLX-030

Serelaxin; recombinant form of human relaxin-2

Acute heart failure

Results from the global phase III RELAX-AHF-2 study showed it did not meet its primary endpoints of reduction in cardiovascular death through day 180 or reduced worsening heart failure through day five when added to standard therapy

3/23/17

Proteon Therapeutics Inc. (Waltham, Mass.)

Vonapanitase

Recombinant human elastase

Chronic kidney disease (CKD)

After disclosing top-line results from the first phase III trial, which evaluated the safety and efficacy of a single dose of vonapanitase in patients with CKD receiving or expecting to receive hemodialysis who underwent surgical creation of a radiocephalic arteriovenous fistula, the company disclosed changes to PATENCY-2, the second phase III study; the protocol amendment reordered the existing endpoints for the PATENCY-2 trial, establishing secondary patency and fistula use for hemodialysis as co-primary endpoints

3/17/17

Resverlogix Corp. (Calgary, Alberta)

Apabetalone

RVX000222; BET inhibitor

High-risk cardiovascular disease

The independent data and safety monitoring board for the phase III BETonMACE trial completed a third planned safety review and recommended that the study should continue as planned without any modifications

3/20/17

CENTRAL NERVOUS SYSTEM

AC Immune SA (Lausanne, Switzerland) and Genentech Inc. (unit of Roche Holding AG; Basel, Switzerland)

Crenezumab

An anti-amyloid beta antibody

Alzheimer's disease (AD)

Decided to start a second phase III trial, CREAD2, which will recruit 750 patients

3/1/17

Accera Inc.

AC-1204

Targets neurodegenerative conditions

Alzheimer's disease (AD)

Phase III NOURISH trial failed to deliver statistically significant improvements in memory and cognition vs. placebo, possibly influenced by a reformulation of the drug

3/1/17

Acorda Therapeutics Inc. (Ardsley, N.Y.)

CVT-301

Inhalable formulation of levodopa

Parkinson's disease

Results from two ongoing, long-term safety studies showed no differences in pulmonary function between the group receiving CVT-301 and an observational control group

3/30/17

Adamas Pharmaceuticals Inc. (Emeryville, Calif.)

ADS-5102

Amantadine hydrochloride extended-release capsules

Levodopa-induced dyskinesia in Parkinson's disease

Data from EASE LID 2, its phase III open-label, long-term safety and efficacy study demonstrated the safety and tolerability of ADS-5102 and a reduction in LID and off-time symptoms for up to 64 weeks

3/1/17

Elite Pharmaceuticals Inc. (Northvale, N.J.)

Reformulated Sequestox

Immediate-release oxycodone with sequestered naltrexone abuse-deterrent opioid product

Severe acute pain

Started pharmacokinetic testing in a three-way, crossover trial to evaluate the relative comparative bioavailability and bioequivalence of the modified formulation of Sequestox to the original formulation of Sequestox and to a comparator product

3/14/17

Jazz Pharmaceuticals plc (Dublin)

JZP-258

Follow-on to Xyrem (sodium oxybate)

Narcolepsy

Enrolled the first patient in a phase III trial

3/17/17

Jazz Pharmaceuticals plc (Dublin)

JZP-110

Selective dopamine and norepinephrine reuptake inhibitor

Excessive sleepiness associated with obstructive sleep apnea (OSA)

Efficacy results from two phase III Treatment of OSA and Narcolepsy Excessive Sleepiness (TONES) studies showed that in TONES 3, it demonstrated highly statistically significant improvement in the co-primary endpoints of Maintenance of Wakefulness test and Epworth Sleepiness scale at all doses; in the key secondary endpoint of Patient Global Impression of Change scale, the candidate showed highly statistically significant improvement

3/21/17

Nektar Therapeutics Inc. (San Francisco)

NKTR-181

Mu-opioid agonist

Moderate to severe chronic low back pain

The trial met the primary efficacy endpoint in the phase III SUMMIT-07 study, showing improved pain relief with NKTR-181 compared to placebo (p=0.0019); key secondary endpoints of the study also were met with high statistical significance

3/21/17

Neurocrine Biosciences Inc. (San Diego)

Ingrezza

Valbenazine; selective vesicular monoamine transporter 2 inhibitor

Tardive dyskinesia (TD)

Results from the Kinect 3 phase III study showed it demonstrated a significant and meaningful reduction in TD symptoms compared with placebo in participants with underlying schizophrenia, schizoaffective disorder or mood disorder

3/22/17

Obseva SA (Geneva)

Nolasiban

An oral oxytocin receptor antagonist

Infertility

Started a phase III program testing nolasiban to improve pregnancy and live birth rates in women undergoing assisted reproduction; the study, IMPLANT2, is expected to enroll about 760 women who are undergoing assisted reproduction by in vitro fertilization or intracytoplasmic sperm injection for low fertility

3/8/17

Ocular Therapeutix Inc. (Bedford, Mass.)

Dextenza 0.4 mg

Dexamethasone insert

Post-surgical ocular pain and inflammation

Results of a patient experience study showed all patients reported it was comfortable, and 96% felt the insert was extremely or very convenient vs. topical eye drops on a tapered schedule, with 88% saying they would request the insert if they were to undergo cataract surgery again

3/16/17

Revance Therapeutics Inc. (Newark, Calif.)

Daxibotulin-umtoxinA

For injection

Moderate to severe glabellar lines

Completed patient enrollment in two pivotal trials of its SAKURA phase III program

3/8/17

Tonix Pharmaceuticals Holding Corp. (New York)

TNX-102 SL 5.6 mg

Rapidly disintegrating sublingual tablet containing 2.8 mg of cyclobenzaprine HCl

Post-traumatic stress disorder

Enrolled the first participant in the phase III HONOR study

3/29/17

INFLAMMATORY

Atlantic Healthcare plc (Cambridge, U.K.)

ICAM-1

Alicaforsen; targets Toll-like receptor 9

Pouchitis

In addition to targeting ICAM-1, alicaforsen has been shown to target Toll-like receptor 9 (TLR-9); the drug is in a pivotal phase III trial for the treatment of pouchitis

3/1/17

Tigenix NV (Leuven, Belgium)

Cx-601

Adipose-derived stem cell candidate

Complex perianal fistulas in Crohn's disease patients

Data from the phase III ADMIRE-CD trial showed that out of the 212 patients randomized, 37 patients concluded the long-term extension from week 52 to week 104, with top-line data showing that the clinical remission rate and difference between groups, as was previously observed at weeks 24 and 52, was maintained at week 104; the tolerability of Cx601 also was maintained and the safety profiles for both treatment and placebo groups were similar for the duration of the trial

3/10/17

MISCELLANEOUS

Aeterna Zentaris Inc. (Charleston, S.C.)

Macimorelin diagnostic test

Ghrelin agonist

Adult growth hormone deficiency

A review of confirmatory phase III data showed it is of comparable accuracy to insulin tolerance tests

3/6/17

Akcea Therapeutics Inc. (Cambridge, Mass.)

Volanesorsen

Antisense drug designed to reduce the production of ApoC-III

Familial chylomicronemia syndrome

The pivotal phase III APPROACH study met its primary endpoint of reducing triglyceride levels; for the primary endpoint of the study, volanesorsen-treated patients (n=33) achieved a statistically significant (p<0.0001) mean reduction in triglycerides of 77% from baseline after three months of treatment, compared to a mean increase of 18% in placebo-treated patients (n=33)

3/7/17

Amryt plc (London)

AP-101

Gel that contains a dry extract from birch bark

Epidermolysis bullosa

Started EASE, its phase III trial

3/29/17

Dermira Inc. (Menlo Park, Calif.)

Glycopyrronium tosylate (formerly DRM-04)

Topical anticholinergic for primary axillary hyperhidrosis

Severe sweating

New time course data from phase III studies showed that it not only resulted in statistically significant improvements in sweating severity at four weeks, but also helped patients began to see reductions in sweating severity after the first week of treatment

3/6/17

Pharnext SA (Paris)

PXT-3003

Pleodrug

Mild to moderate Charcot-Marie-Tooth disease type 1A

The first two patients entered the international phase III extension study PLEO-CMT-FU

3/17/17

Rexgenero Ltd. (London)

REX-001

Rexmyelocel-T; autologous, bone-marrow-derived cell therapy

Critical limb ischemia (CLI)

Started a phase III trial in diabetic patients with CLI

3/23/17

Stealth Biotherapeutics Inc. (Boston)

Elamipretide

Targets the inner mitochondrial membrane, restoring electron transport to improve cellular energy and reduce oxidative stress

Mitochondrial myopathy (MM)

Started RePOWER, an observational study and pre-trial registry expected to help the firm design an interventional phase III trial

3/3/17

Stemline Therapeutics Inc. (New York)

SL-401

Targeted therapy directed to the interleukin-3 receptor

Blastic plasmacytoid dendritic cell neoplasm (BPDCN)

Enrollment of stage 3 in the pivotal trial has been completed

3/24/17

RESPIRATORY

Aimmune Therapeutics Inc. (Brisbane, Calif.)

AR-101

Biologic oral immunotherapy

Peanut allergy

Phase III screening data and phase II adherence data showed that, in all, 457 patients (78%) were reactors, who experienced dose-limiting symptoms in the double-blind, placebo-controlled food challenge at or before the 100 mg dose (median = 44 mg cumulative amount of peanut protein)

3/7/17

Allergy Therapeutics plc (Worthing, U.K.)

Pollinex Quattro Birch

Ultra-short course, aluminium-free immunotherapy

Birch pollen-induced rhinoconjunctivitis

Recruited the first patient in its phase III study, anticipated to involve more than 550 patients over 50 sites across Germany, Sweden, Austria and Poland

3/17/17

Anthera Pharmaceuticals Inc. (Hayward, Calif.)

Sollpura

Liprotamase

Cystic fibrosis (CF)

Findings from the extension period of the phase III SOLUTION noninferiority study showed that during weeks seven through 20, all patients treated with Sollpura maintained their weight at 56.4 kg-56.9 kg and all patients treated with Pancreaze maintained their weight at 54.6 kg-54.8 kg; both groups showed small increases in height, with Sollpura increasing from 163.2 cm to 163.7 cm, and Pancreaze increasing from 160.6 cm to 160.8 cm

3/31/17

Asit Biotech SA (Brussels, Belgium)

gp-Asit+

Immunotherapy candidate

Grass pollen rhinitis

Results from its 554-patient, phase III trial showed it consistently improved clinical symptoms and reduced medication use in patients by between 15% and 21% compared to placebo, depending on the type of analyses performed (peak vs. entire pollen season, intention-to-treat [ITT] vs. per protocol population)

3/1/17

Boehringer Ingelheim GmbH (Ingelheim, Germany)

Ofev

Nintedanib

Progressive fibrosing lung conditions

The first patient has been enrolled in a trial investigating its efficacy and safety in conditions other than idiopathic pulmonary fibrosis

3/22/17

DBV Technologies SA (Montrouge, France)

Viaskin Peanut 250 mcg

Immunotherapy that aims to safely desensitize peanut allergic subjects to peanut by increasing the level of peanut proteins consumed symptom-free

Peanut allergy

Completed enrollment in a phase III study designed to assess the safety and routine clinical use

3/13/17

Glaxosmithkline plc (London)

Nucala

Mepolizumab

Severe asthma

Phase IIIb MUSCA study results showed that patients treated with Nucala added-on to standard of care achieved clinically and statistically significant improvements in their health-related quality of life and lung function; the study met all its primary and secondary endpoints, and results showed the St. Georges respiratory questionnaire (SGRQ) score (primary endpoint), a measure of quality of life, improved by 7.7 units from baseline vs. placebo (p=0.001) after 24 weeks

3/7/17

Glenmark Pharmaceuticals Ltd. (Mumbai, India)

GSP-301 nasal spray

Fixed-dose combination of mometasone furoate (25 mcg) and olopatadine hydrochloride (665 mcg)

Seasonal allergic rhinitis

Phase III trial results showed that treatment with GSP 301 demonstrated statistically significant and clinically meaningful improvement from baseline in average morning and evening patient-reported 12-hour reflective Total Nasal Symptom Score, compared to placebo (p <0.001), olopatadine (p=0.028) and mometasone (p=0.019); all investigational treatments administered in the trial were well-tolerated, and showed no meaningful differences in reported adverse events across study arms

3/30/17

PTC Therapeutics Inc. (South Plainfield, N.J.)

Ataluren

Protein restoration therapy

Nonsense mutation cystic fibrosis (nmCF)

The phase III Ataluren Confirmatory Trial (ACT CF) missed both primary and secondary endpoints, and PTC said it was halting development in CF, closing ongoing extension studies and withdrawing its marketing authorization application in Europe to treat CF

3/3/17

Vertex Pharmaceuticals Inc. (Boston)

Tezacaftor (VX-661)

Oral CFTR corrector

Cystic fibrosis

Reported positive data from two phase III studies testing tezacaftor plus Kalydeco (ivacaftor); the study met its primary endpoint with a mean absolute improvement in ppFEV1 through 24 weeks of 4.0 percentage points from baseline compared to placebo (p < 0.0001)

3/30/17

Notes

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