Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Bristol-Myers Squibb Co. (New York)

Orencia

Abatacept; T-cell co-stimulation blocker

Rheumatoid arthritis

Data from an observational study of anti-cyclic citrullinated peptide (anti-CCP, also known as ACPA) and rheumatoid factor (RF), both of which are biomarkers of poor prognosis, which may be associated with more severe disease progression and joint damage, showed that patients who tested positive via a common blood test for anti-CPP or RF were more likely to have greater response with Orencia than patients testing negative for the biomarkers

6/9/16

Can-Fite Biopharma Ltd. (Petach Tikva, Israel)

CF101

Piclidenoson; an A3 adenosine receptor agonist

Psoriasis

Submitted a protocol design to the EMA for a phase III trial

6/8/16

Coherus Biosciences Inc. (Redwood City, Calif.)

CHS-0214

Etanercept biosimilar (Enbrel, Amgen Inc.)

Autoimmune disorders

Data showed the equivalence of CHS-0214 to Enbrel with respect to efficacy as measured by the primary endpoint, ACR20 at 24 weeks

6/13/16

Eli Lilly and Co. (Indianapolis) and Incyte Corp. (Wilmington, Del.)

Baricitinib

An oral JAK1 and JAK2 inhibitor

Rheumatoid arthritis

Two phase III trials patients with rheumatoid arthritis treated with baricitinib reported significant improvements in quality of life symptoms and other patient-reported outcomes compared to methotrexate or Humira (adalimumab, Abbvie Inc.)

6/10/16

Janssen Research & Development LLC (Raritan, N.J.; a unit of Johnson & Johnson)

CNTO 136

An anti-interleukin-6 monoclonal antibody

Rheumatoid arthritis

Results from a 1,670-patient phase III study showed that those receiving sirukumab demonstrated significant inhibition of radiographic progression, or joint destruction, from baseline to week 52 and improvement in signs and symptoms of rheumatoid arthritis at week 16, the study's co-primary endpoints, when compared with patients receiving placebo

6/9/16

Mallinckrodt plc (Chesterfield, U.K.)

H.P. Acthar Gel

Repository corticotropin injection

Systemic lupus erythematosus

Study patients with persistently active SLE who continued on the gel throughout the 44-week extension period maintained the reductions achieved in certain measures of disease activity during the blinded phase

6/13/16

Oncobiologics Inc. (Cranbury, N.J.)

ONS-3010

A biosimilar of Humira (adalimumab, Abbvie Inc.)

Moderate to severe plaque psoriasis

Its phase III plan received the first of its European Union clinical trial authorizations, including in the U.K., Germany and Spain, for the biosimilarity study portion of the program

6/17/16

Pfizer Inc. (New York)

Xeljanz

Tofacitinib citrate

Active psoriatic arthritis

OPAL (Oral Psoriatic Arthritis triaL) Beyond, the second phase III study, met its primary efficacy endpoints; patients in the treatment arm of the study demonstrated a statistically significant (p<0.0001) improvement with tofacitinib 5 mg twice daily and 10 mg twice daily vs. placebo treatment as measured by American College of Rheumatology 20 response and Health Assessment Questionnaire Disability Index score at 3 months

6/8/16

CANCER

Actinium Pharmaceuticals Inc. (New York)

Iomab-B

Induction and conditioning agent

Relapsed or refractory acute myeloid leukemia

Initiated the pivotal phase III SIERRA (Study of Iomab-B in Elderly Relapsed or Refractoy AML) trial for Iomab-B, designed as an induction and conditioning agent to prepare patients with relapsed or refractory acute myeloid leukemia (AML) over age 55 for a hematopoietic stem cell transplant

6/30/16

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

Zoptrex

Zoptarelin doxorubicin

Advanced, recurrent endometrial cancer

Reconfirmed its expectation that the pivotal, phase III trial for Zoptrex is expected to be completed in the third quarter of 2016, with a planned new drug application for the candidate to follow in the first half of 2017

6/8/16

Amgen Inc. (Thousand Oaks, Calif.)

Blincyto

Blinatumomab

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

Data from a prespecified interim analysis of the phase III TOWER study showed that median overall survival was 7.7 months for Blincyto vs. four months for standard of care (p=0.012)

6/13/16

Argos Therapeutics Inc. (Durham, N.C.)

AGS-003

Immunotherapy

Metastatic renal cell carcinoma

Independent data monitoring committee for the pivotal phase III ADAPT trial recommended the continuation of the trial based on results of an interim data review

6/14/16

Array Biopharma Inc. (Boulder, Colo.), Pierre Fabre SA (Paris) and Merck KGaA (Darmstadt, Germany)

Binimetinib, encorafenib and Erbitux

MEK inhibitor, BRAF inhibitor and cetuximab, monoclonal antibody

BRAF-mutant colorectal cancer

Initiated the BEACON CRC trial, a global phase III study designed to assess the safety and efficacy of binimetinib (MEK inhibitor), encorafenib (BRAF inhibitor) and Erbitux (cetuximab, monoclonal antibody) in comparison to Erbitux and irinotecan-based therapy; the primary endpoint is overall survival and key secondary endpoints include progression-free survival and objective response rate

6/7/16

Bayer Healthcare Pharmaceuticals (Whippany, N.J.)

Stivarga

Regorafenib tablets

Unresectable hepatocellular carcinoma

Achieved a median overall survival improvement in patients with unresectable hepatocellular carcinoma who progressed after treatment with Nexavar tablets; patients taking Stivarga had a median overall survival of 10.6 months vs. 7.8 months for placebo plus best supportive care (p<0.001)

6/29/16

Beyondspring Pharmaceuticals Inc. (New York)

Plinabulin

Targets tumor vasculature and improves the immune system

Cancer

Global phase III study testing plinabulin was launched in China; the trial aims to enroll 550 patients in total, with 440 from China and 110 from the U.S. and Australia

6/29/16

Celator Pharmaceuticals Inc. (Ewing, N.J.)

Vyxeos

Cytarabine: daunorubicin liposome for injection

Acute myeloid leukemia

Phase III data showed it had statistically significant improvements, with an induction response rate of 68.2% vs. 25% (p = 0.007)

6/15/16

Daiichi Sankyo Co. Ltd. (Tokyo)

Patritumab

HER3 inhibitor

Locally advanced or metastatic non-small-cell lung cancer

The two-part phase III HER3-Lung study will not proceed into the second part, following the recommendation of an independent data monitoring committee that concluded the first part of the study did not meet the pre-defined efficacy criteria required to proceed; there were no safety concerns identified

6/1/16

Galena Biopharma Inc. (San Ramon, Calif.)

Neuvax

Neliipepimut-S

Breast cancer

Phase III PRESENT is being stopped for futility following a planned interim analysis by an independent data monitoring committee

6/30/16

Glide Technologies Ltd. (Oxford, U.K.)

Octreotide

100 mcg formulation delivered using needle-free Solid Dose Injector

Acromegaly and certain neuroendocrine tumors

Reported proof-of-concept results demonstrating that its solid formulation of a widely used dose of octreotide (100 mcg) achieved bioequivalence to the currently marketed immediate-release liquid injectable product (Sandostatin), approved for use in the treatment of acromegaly and certain neuroendocrine tumors; results showed Glide's formulation matched Sandostatin on both maximum peak concentration and area under the curve, and study subjects (n=20) confirmed the suitability of the SDI for self-administration

6/29/16

Immunocellular Therapeutics Ltd. (Los Angeles)

ICT-107

Patient-specific, dendritic cell-based immunotherapy

Glioblastoma

The first patient was treated in the phase III registrational trial

6/8/16

Incyte Corp. (Wilmington, Del.)

Epacadostat

Oral indoleamine 2,3-dioxygenase 1 inhibitor

Advanced or metastatic melanoma

Started Echo-301, a phase III trial testing epacadostat in combination with Keytruda (pembrolizumab; Merck & Co. Inc.)

6/24/16

Ipsen SA (Paris)

Somatuline Depot

Lanreotide

Carcinoid syndrome

Phase III trial results showed the adjusted mean percentage days with rescue octreotide use (the primary endpoint) was lower in the Somatuline group (33.7%) vs. the placebo group (48.5%) (p=0.017)

6/7/16

Janssen Research & Development LLC (Raritan, N.J; a unit of Johnson & Johnson)

Darzalex

Daratumumab

Multiple myeloma

Phase III MMY3003 (POLLUX) trial showed that Darzalex in combination with standard-of-care regimen Revlimid (lenalidomide, Celgene Corp.) and dexamethasone achieved a 63% reduction in the risk of disease progression or death, compared to Revlimid and dexamethasone alone in patients with multiple myeloma who had received at least one prior line of therapy (p<0.0001)

6/13/16

Mateon Therapeutics Inc. (South San Francisco)

CA4P

Fosbretabulin

Recurrent ovarian cancer

Updated data from the study called GOG-0186I show an improvement in overall survival of 3.2 months

6/21/16

Merck & Co. Inc. (Whitehouse Station, N.J.)

Keytruda

Pembrolizumab

Non-small-cell lung cancer

Phase III Keynote-24 trial met its primary endpoint by turning up superior data compared to chemo in progression-free survival as well as the secondary endpoint of overall survival

6/17/16

Nymox Pharmaceutical Corp. (Hasbrouck Heights, N.J.)

NX-1207

Fexapotide

Prostate cancer

Results from the company's seven-year prospective placebo controlled double-blind studies showed a major reduction in the incidence of prostate cancer, compared to placebo and compared to the known and expected normal incidence of the disease; the new data analysis from the Nymox fexapotide study has now shown the statistically significant and very low incidence of 1.3% for prostate cancer

6/23/16

Pharmamar SA (Madrid)

Plitidepsin

An antitumor agent originally isolated from the Mediterranean tunicate Aplidium albicans

Relapsed and refractory angioimmunoblastic T-cell lymphoma

Started a pivotal study

6/15/16

Provectus Biopharma-ceuticals Inc. (Knoxville, Tenn.)

PV-10

Injectable formulation of rose bengal disodium

In-transit melanoma

After a median follow-up of 11.7 months, disease control was achieved in 63% of patients; five patients (26%) achieved a complete response, with another five achieving partial responses and two patients (11%) showing stable disease at the time of last follow-up; 74% had a clinical response at time of first follow-up, and data showed younger patients and those with smaller lesions were more likely to respond to treatment

6/9/16

Sirtex Medical Ltd. (North Sydney, Australia)

SIR-Spheres Y-90

Resin microspheres

Unresectable primary liver cancer

A trial of SIR-Spheres Y-90 resin microspheres vs. Nexavar (sorafenib, Amgen Inc. and Bayer AG) has completed its target enrollment of at least 360 patients

6/7/16

Tesaro Inc.

Niraparib

With companion diagnostic Mychoice HRD by Myriad Genetics Inc.

Recurrent ovarian cancer

Findings from the phase III NOVA trial showed it achieved its primary endpoint of statistically significant progression-free survival (PFS) compared to control across germline BRCA mutation (gBRCAmut) carriers and non-gBRCAmut carriers, including those with homologous recombination deficient (HRD) tumors; the median PFS for gBRCAmut carriers treated with niraparib was 21 months compared to 5.5 months for control (p < 0.0001), while median PFS for HRD+ non-gBRCAmut carriers was 12.9 months compared to 3.8 months for control (p < 0.0001)

6/30/16

CARDIOVASCULAR

Bellerophon Therapeutics Inc. (Warren, N.J.)

Inopulse

Delivers nitric oxide therapy

Pulmonary arterial hypertension

The first subject was enrolled in the phase III INOvation-1 trial

6/17/16

Capricor Therapeutics Inc. (Los Angeles)

CAP-1002

An allogeneic cardiosphere-derived cell therapy

Advanced heart failure

Preliminary 12-month data from its DYNAMIC (Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells) trial showed that improvements from baseline in key cardiac function and dimensional indices were directionally maintained

6/17/16

Celyad SA (Mont-Saint-Guibert, Belgium)

C-Cure

Stem cell therapy

Congestive heart failure

Failed to reach the primary endpoint of a phase III trial; the primary endpoint of the Chart-1 placebo-controlled study, which recruited 271 patients, was a composite measure based on mortality, worsening of heart failure events, quality of life, six-minute walk test performance, end systolic volume and ejection fraction

6/29/16

Cytori Therapeutics Inc. (San Diego)

Cytori Cell Therapy

Using the Celution System

Chronic ischemic heart disease with left heart failure

Results from the ATHENA trials showed that improvements in the Minnesota Living with Heart Failure Questionnaire were observed in the treatment group relative to placebo – at three months (p = 0.54), at six months (p = 0.154) and at 12 months (p = 0.038)

6/29/16

Genentech Inc. (South San Francisco)

Actemra

Tocilizumab

Giant cell arteritis

Reported positive results from a phase III study known as GiACTA, which met its primary and key secondary endpoints, showing the compound, initially combined with a six-month steroid (glucocorticoid) regimen, more effectively sustained remission through one year compared to a six- or 12-month steroid-only regimen in newly diagnosed and relapsing people with GCA

6/7/16

Incyte Corp. (Wilmington, Del.)

Jakafi

Ruxolitinib; JAK inhibitor

Polycythemia vera resistant to or intolerant of hydroxyurea

New 28-week data from the phase III Response-2 study showed Jakafi was superior to best available therapy in maintaining hematocrit control (62.2% vs. 18.7%, respectively; p<0.0001) without the need for phlebotomy in patients with inadequately controlled polycythemia vera resistant to or intolerant of hydroxyurea who did not have an enlarged spleen

6/13/16

Prizer Inc. (New York)

Bococizumab

Anti-PCSK9 candidate

Low-density lipoprotein cholesterol

Two additional phase III studies, SPIRE-HR (high risk) and SPIRE-FH (familial hypercholesterolemia), met their primary endpoint, demonstrating a significant reduction in the percent change from baseline in low-density lipoprotein cholesterol at 12 weeks compared to placebo among adults at high and very high risk for cardiovascular events who were receiving a maximally tolerated dose of a highly effective statin

6/29/16

CENTRAL NERVOUS SYSTEM

Acelrx Pharmaceuticals Inc. (Redwood City, Calif.)

ARX-04

Sufentanil sublingual tablet, 30 mcg

Moderate to severe acute pain

Completed patient enrollment in its two remaining phase III studies

6/29/16

Adamas Pharmaceuticals Inc. (Emeryville, Calif.)

ADS-5102

Amantadine extended-release capsules

Levodopa-induced dyskinesia associated with Parkinson's disease

Data from its phase III clinical program showed improvement across all subgroups in the EASE LID study, and observed in EASE LID 2 was a reduction in motor complications associated with ADS-5102, maintained for up to 41 weeks based on the first interim analysis

6/23/16

Auris Medical Holding AG (Zug, Switzerland)

Keyzilen

AM-101

Acute and post-acute inner ear tinnitus

Completed patient enrollment in the phase III TACTT3 trial

6/29/16

Cara Therapeutics Inc. (Stamford, Conn.)

CR845

Intravenous

Postoperative pain

Resumed patient recruitment after the FDA removed a clinical hold on its CLIN3001 adaptive phase III trial

6/7/16

Colucid Pharmaceuticals Inc. (Cambridge, Mass.)

Lasmiditan

Targets 5-HT1F receptors in the trigeminal pathway

Migraine headaches

The last patient was randomized in its SAMURAI phase III trial

6/8/16

Contravir Pharmaceuticals Inc. (Edison, N.J.)

FV-100

Antiviral candidate

Postherpetic neuralgia

Amended the protocol for its ongoing phase III study

6/17/16

Endo International plc (Malvern, Pa.)

Belbuca

Buprenorphine buccal film

Chronic pain in patients requiring around-the-clock opioids

New data supported the safety and tolerability of Belbuca; adverse events occurred in 43.1% and 54% of patients during the titration (n=506) and long-term treatment (n=435) phases, respectively; for the efficacy endpoint, the average daily pain intensity score during the treatment phase was between 2.9 and 3.1 on a scale of 0 (no pain) to 10 (worst pain imaginable); the need for rescue medication to relieve breakthrough pain decreased from an average of three tablets in the titration to 1.1 tablets in the long-term treatment phase

6/6/16

GW Pharmaceuticals plc (London)

Epidiolex

Liquid formulation of plant-derived cannabidiol

Lennox-Gastaut syndrome

Hit the primary endpoint of a phase III trial

6/28/16

Highland Therapeutics Inc. (Toronto)

HLD-200 (Benjorna)

Delayed-release and extended-release methylphenidate capsules

Attention deficit hyperactivity disorder

Phase III pivotal data showed a 44% improvement in ADHD symptom scores, a highly statistically significant positive difference compared to the placebo group (p=0.002)

6/7/16

Intra-Cellular Therapies Inc. (New York)

ITI-007

Designed to combine serotonin 5-HT2A receptor antagonism, dopamine receptor phosphoprotein modulation, glutamatergic modulation and serotonin reuptake inhibition

Dementia including Alzheimer's disease

Started phase III development with about 360 patients

6/29/16

Jazz Pharmaceuticals plc (Dublin)

JZP-110

Wake-promoting agent designed to act as a selective dopamine and norepinephrine reuptake inhibitor

Excessive sleepiness in adult patients with narcolepsy or with obstructive sleep apnea

Phase III results demonstrated that each of the doses of JZP-110 that were studied, including the high therapeutic dose of 300 mg and the supratherapeutic doses of 600 mg and 1,200 mg, had consistently lower ratings on the primary endpoint of Peak Liking at the Moment and on the secondary endpoints of Overall Drug Liking and willingness to Take the Drug Again compared to the schedule IV stimulant phentermine at 90 mg

6/15/16

Marinus Pharmaceuticals Inc. (Radnor, Pa.)

Ganaxolone

CNS-selective GABAA modulator

Drug-resistant focal onset seizures

Phase III trial missed its endpoint, failing to mirror the reduction in seizure rate observed in earlier trials; the company is halting the program in adult seizures, but will continue development in pediatric orphan indications

6/14/16

Neurotrope Inc. (Newark, N.J.)

Bryostatin

Alzheimer's disease drug

Alzheimer's disease

Data published in the Journal of Biological Chemistry shows it increases the levels of the synaptic scaffolding protein PSD-95 and induces the movement of phosphorylated PSD-95 to the neuronal membranes

6/27/16

Shire plc (Dublin)

SHP465

A triple-bead mixed amphetamine salts

Attention deficit hyperactivity disorder

Top-line results showing that a four-week randomized, double-blind, parallel-group, placebo-controlled, forced-dose titration, efficacy and safety study met it primary endpoints for both doses of SHP465 (12.5 mg and 37.5 mg), administered once daily, in patients with ADHD; SHP465 showed superiority over placebo in ADHD symptom improvement (p < 0.001); a key secondary endpoint was also met for both doses, showing significant improvement over placebo in patients' global functioning (p<0.001)

6/30/16

Symbio Pharmaceuticals Ltd. (Tokyo)

SyB P-1501

Pain therapy

Acute postoperative pain during hospitalization

Initiated the phase III trial of SyB P-1501 in Japan

6/14/16

Trevena Inc. (King of Prussia, Pa.)

Oliceridine

TRV130; a mu receptor G protein pathway selective modulator

Moderate to severe acute pain following bunionectomy and abdominoplasty

Enrolled the first patients in the phase III APOLLO-1 and APOLLO-2 studies

6/9/16

Zogenix Inc. (Emeryville, Calif.)

ZX008

Low-dose fenfluramine

Seizures in Dravet syndrome

Initiated a second phase III trial

6/8/16

Zosano Pharma Corp. (Fremont, Calif.)

Zp-Triptan

Patch treatment

Acute migraine

Enrolled its first subject in a pivotal efficacy trial

6/20/16

GASTROINTESTINAL

Evoke Pharma Inc. (Solana Beach, Calif.)

EVK-001

A nasal delivery formulation of metoclopramide

Recurrent diabetic gastroparesis

The last subject completed treatment in its pivotal phase III study

6/2/16

DIABETES

Innocoll Holdings plc (Athlone, Ireland)

Cogenzia

INL-002; a collagen matrix with gentamicin

Diabetic foot ulcers

Completed enrollment of patients in its two pivotal phase III trials, COACT-1 and COACT-2

6/24/16

Novo Nordisk A/S (Bagsvaerd, Denmark)

Semaglutide

A glucagon-like peptide-1 analogue

Type 2 diabetes

Findings from a phase IIIa trial showed that from a mean baseline HbA1c of 8.2%, those receiving metformin, with or without sulfonylurea, achieved statistically significant and superior improvements in HbA1c reductions of 1.2% and 1.6% when treated with 0.5 mg and 1 mg semaglutide, respectively, vs. a 0.8 percent reduction with insulin glargine U100 (p<0.0001 for both)

6/1/16

Viromed Co. Ltd. (Seoul, South Korea)

VM202

DNA-based biopharmaceutical

Painful diabetic peripheral neuropathy

Dosed the first patient in the recently initiated phase III study

6/28/16

INFECTION

Achaogen Inc. (South San Francisco)

Plazomicin

Aminoglycoside antibiotic

Serious bacterial infections due to Enterobac-teriaceae, including carbapenem-resistant Enterobac-teriaceae

Achieved more than 50% patient enrollment in its ongoing phase III EPIC registration trial

6/3/16

Frontier Biotechnologies Inc. (Nanjing, China)

Albuvirtide

Fusion inhibitor

HIV

A phase III trial showed it met the primary objective based on an interim analysis; the results demonstrated that once-weekly albuvirtide plus ritonavir-boosted lopinavir was noninferior to WHO-recommended second-line three-drug regimen (control)

6/7/16

The Medicines Co. (Parsippany, N.J.)

Carbavance

Meropenem-vaborbactam

Complicated urinary tract infections

Met both FDA and EMA primary endpoints in a pivotal phase III trial; data showed statistical superiority to Pfizer Inc.'s Zosyn (piperacillin-tazobactam)

6/27/16

INFLAMMATORY

Kitov Pharmaceuticals Holdings Ltd. (Tel Aviv, Israel)

KIT-302

Comprises two approved drugs, celecoxib and amlodipine besylate

Osteoarthritis pain and hypertension

Data from its phase III study showed that celecoxib increased serum creatinine compared to placebo; in contrast, while amlodipine alone reduced serum creatinine (-2.55 umol/L), a greater reduction in plasma levels of creatinine was achieved in patients in the KIT-302 arm (-3.22 umol/L), suggesting better renal function

6/27/16

Novartis AG (Basel, Switzerland)

Cosentyx

Secukinumab

Ankylosing spondylitis and psoriatic arthritis

Data suggest it may lead to higher responses than Humira (adalimumab, Abbvie Inc.) in improving the signs and symptoms of people with ankylosing spondylitis (AS) and psoriatic arthritis (PsA) at 52 weeks

6/9/16

MISCELLANEOUS

Aerie Pharmaceuticals Inc. (Irvine, Calif.)

Rhopressa

Netarsudil ophthalmic solution 0.02%

To lower intraocular pressure in glaucoma or ocular hypertension

Completed patient enrollment in Rocket 4, its phase III trial

6/17/16

Alexion Pharmaceuticals Inc. (New Haven, Conn.)

Soliris

Eculizumab

Refractory generalized myasthenia gravis

Top-line results from the trial called Regain show it missed the primary endpoint, change from baseline in Myasthenia Gravis-Activities of Daily Living Profile at week 26 (p=0.0698), although it did well on a secondary endpoint, the change from baseline in Quantitative Myasthenia Gravis total score (p=0.0129), and on secondary endpoints of responder status in MG-ADL and QMB (p<0.05)

6/8/16

Antares Pharma Inc. (Ewing, N.J.)

Quickshot Testosterone

Administered subcutaneously

Hypogonadism

The last patient completed treatment in a dose-blinded, multiple-dose, concentration-controlled, 26-week phase III trial

6/2/16

Aytu Bioscience Inc. (Englewood, Colo.)

Natesto

Testosterone; a nasal gel

Hypogonadism

Clinical data for Natesto has shown that the pharmacokinetics, safety and efficacy of Natesto for restoring normal testosterone levels in men with low testosterone levels is not adversely affected by seasonal allergies

6/29/16

Bellus Health Inc.

Kiacta

Eprodisate

AA amyloidosis

Phase III data showed a confirmatory phase III study missed the primary endpoint of slowing renal function decline in patients with the orphan disease

6/21/16

Biorestorative Therapeutics Inc. (Melville, N.Y.)

MSCs

Bone marrow-derived mesenchymal stem cells

Lumbar degenerative disc disease

Completed a study, Re-consenting and Follow-Up of Adults from a Retrospective Study Using Autologous Transplantation of Marrow Derived Mesenchymal Stem Cells to Degenerated Intervertebral Disc, showing that no adverse events related to the intradiscal injection of autologous hypoxic-cultured, bone marrow-derived mesenchymal stem cells (MSCs) occurred at the time of injection or for up to six years post-injection

6/29/16

Catalyst Pharmaceuticals Inc. (Coral Gables, Fla.)

Firdapse

Amifampridine phosphate

Lambert-Eaton myasthenic syndrome

Reached agreement with the FDA on a confirmatory phase III study protocol

6/14/16

Cytori Therapeutics Inc. (San Diego)

Cytori Cell Therapy

Injected subcutaneously into fingers

Impaired hand function from scleroderma

Completed enrollment in a phase III trial, STAR, a randomized, placebo-controlled, double-blind study

6/8/16

Dermira Inc. (Menlo Park, Calif.)

DRM04

Glycopyrronium tosylate

Excessive underarm sweating (primary axillary hyperhidrosis)

Top-line results from its phase III ATMOS-1 and ATMOS-2 pivotal trials showed the drug delivered statistically significant declines in sweating severity for patients in both trials; in the ATMOS-2 trial, DRM04 demonstrated statistically significant improvements for both co-primary endpoints and both secondary endpoints compared to vehicle; in the ATMOS-1 trial, DRM04 demonstrated statistically significant improvements for one of the co-primary endpoints and both secondary endpoints

6/3/16

Fibrogen Inc. (San Francisco) and Astellas Pharma Inc. (Tokyo)

ASP1517 or FG-4592

Roxadustat; orally administered small molecule inhibitor of hypoxia-inducible factor prolyl hydroxylase

Anemia associated with chronic kidney disease

Initiated the first phase III clinical study in Japan

6/10/16

Ocular Therapeutix Inc. (Bedford, Mass.)

Dextenza

Sustained-release dexmethasone intracanalicular depot

Ocular itching associated with chronic allergic conjunctivitis

Top-line results from its second phase III trial showed it failed to achieve the primary endpoint, defined as the difference in the mean scores in ocular itching between the treatment and placebo groups at three time points seven days following insertion of the depots

6/7/16

Ophthotech Corp. (New York)

Fovista

Pegpleranib; anti-platelet-derived growth factor therapy

Wet age-related macular degeneration

Completed patient recruitment for its phase III trial

6/21/16

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

Dupilumab

Targets the interleukin-4 and interleukin-13 signaling pathways

Moderate to severe atopic dermatitis

A one-year phase III study, known as LIBERTY AD CHRONOS, met its primary and key secondary endpoints

6/7/16

Relypsa Inc. (Redwood City, Calif.)

Veltassa

Patiromer for oral suspension

Hyperkalemia and chronic kidney disease

Results of a pre-specified exploratory analysis of the phase III OPAL-HK trial found that patients taking Veltassa had significant decreases in systolic and diastolic blood pressure and reduced levels of aldosterone in the blood

6/29/16

Revance Therapeutics Inc. (Newark)

RT001

Daxibotu-linumtoxinA topical gel

Moderate to severe lateral canthal lines, or crow's feet

REALISE 1 phase III data show it did not achieve its co-primary endpoint, defined as composite measurements of 2-point or greater and 1-point or greater improvement in lateral canthal lines between baseline and 28 days after treatment, as graded by the Investigator's Global Assessment of Lateral Canthal Lines and the Patient Severity Assessment; other endpoints also were missed, although RT001 appeared to be well-tolerated; the company is not pursuing further clinical development

6/15/16

Santhera Pharmaceuticals AG (Liestal, Switzerland)

Raxone

Idebenone

Duchenne muscular dystrophy

Phase III data demonstrated its ability to slow the rate of decline of respiratory function over one year; peak expriatory flow for each patient in the drug treatment group declined by an average 2.57%, whereas it declined by an average 8.84% for those in the control group

6/2/16

Trevi Therapeutics Inc. (New Haven, Conn.)

Nalbuphine ER

Oral extended release opioid with an opioid receptor dual µ-antagonist/κ-agonist mechanism of action

Prurigo nodularis

Completed enrollment in a pivotal study

6/3/16

RESPIRATORY

ALK-Abellos A/S (Horsholm, Denmark)

Grazax

Immunotherapy

Asthma

Treatment reduced the proportion of patients with asthma symptoms or use of asthma medication, at two years post-treatment (p < 0.05), and reduced the proportion of patients experiencing asthma symptoms during the entire five-year trial period (p < 0.05)

6/15/16

Circassia Pharmaceutical plc

Cat-Spire

Immunotherapy

Cat allergy

Flopped in a phase III 1,200-subject trial, showing a 58.5 percent reduction in allergy symptoms in both the treatment and placebo arms

6/21/16

DBV Technologies SA (Montrouge, France)

Viaskin Peanut

Peanut allergy therapy

Peanut allergy

Reached its patient recruitment objective for PEPITES (PEanut EPIT Efficacy and Safety), its pivotal phase III trial of Viaskin Peanut for the treatment of peanut allergic children, with a total of 500 patients screened and the initial randomization target increased to 350 patients

6/29/16

Glaxosmithkline plc (London) and Innoviva Inc. (South San Francisco)

FF/UMEC/VI

Once-daily, triple-combination therapy (fluticasone furoate/umeclidinium/vilanterol) comprising an inhaled corticosteroid, a long-acting muscarinic antagonist and a long-acting beta agonist

Chronic obstructive pulmonary disease

Top-line results from the pivotal phase III FULFIL study met its two co-primary endpoints, demonstrating statistically significant improvements compared with twice-daily Symbicort Turbohaler (budesonide/formoterol 400 mcg/12 mcg, Astrazeneca plc) in both lung function as measured by trough FEV1 (p<0.001), and health-related quality of life as measured by the St. George's Respiratory Questionnaire (SGRQ, p<0.001), at the end of the 24-week study period

6/21/16

Raptor Pharmaceutical Corp. (Novato, Calif.)

Quinsair

Levofloxacin inhalation solution

Cystic fibrosis and chronic Pseudomonas aeruginosa infections

Subjects with three or more prior year pulmonary exacerbations who were randomized to receive Quinsair had a significantly lower incidence of pulmonary exacerbations when compared with their peers who were randomized to receive the active comparator, tobramycin inhalation solution, (p=0.026)

6/13/16

Theravance Biopharma Inc. (Dublin)

TD-4208

Revefenacin; a long-acting muscarinic antagonist

Chronic obstructive pulmonary disease

50% enrollment has been surpassed in each of the three ongoing Phase III trials

6/2/16

Vertex Pharmaceuticals Inc. (Cambridge, Mass.)

Kalydeco

Ivacaftor

Cystic fibrosis

Real-world data from an ongoing five-year observational study evaluating long-term outcomes showed that those who received Kalydeco for up to five years showed no new safety concerns, and outcomes across multiple measures of CF are indicative of disease modification

6/13/16

Vertex Pharmaceuticals Inc. (Cambridge, Mass.)

Orkambi

Lumacaftor/ivacaftor

Cystic fibrosis

Final safety data from a phase III study showed that the combination drug was well-tolerated, and the most common adverse events were cough, headache, infective pulmonary exacerbation, nasal congestion, abdominal pain, increased sputum and elevated liver enzymes

6/13/16

Notes

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