Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Celgene Corp. (Summit, N.J.)

Ozanimod

The oral selective sphingosine 1-phosphate 1 and 5 receptor modulator

Relapsing multiple sclerosis

Met the primary endpoint, reduction in annualized relapse rate for ozanimod compared to weekly Avonex (interferon beta-1a, Biogen Inc.); top-line phase III SUNBEAM results showed statistically significant and clinically meaningful improvements in ARR for both ozanimod arms compared to the intramuscular Avonex; both doses of ozanimod outperformed Avonex on several secondary endpoints, including number of gadolinium-enhancing MRI lesions and number of new or enlarging T2 MRI lesions at month 12

2/21/17

Eli Lilly and Co. (Indianapolis)

Baricitinib

Oral selective JAK1 and JAK2 inhibitor

Moderate to severe rheumatoid arthritis

Pivotal phase III data from RA-BEAM show that starting as early as week eight, and sustained through week 52, a higher proportion of patients achieved ACR50 and ACR70 response compared to Humira (adalimumab, Abbvie Inc.); those improvements were statistically significant compared to adalimumab at weeks 12, 20, 28, 32 and 40

2/17/17

Janssen-Cilag International NV (Beerse, Belgium; unit of Johnson & Johnson)

Stelara

Ustekinumab; an antibody targeting IL-12 and IL-23

Moderate to severe Crohn's disease

Two-year data from the IM-UNITI long-term extension study evaluating the efficacy and safety of Stelara showed that treatment with ustekinumab maintained clinical response and remission for up to two years with no new safety signals observed; among randomized patients who entered the long-term study period and continued to receive ustekinumab through week 96, 79.2% of patients receiving ustekinumab every 12 weeks and 87.1% of patients receiving ustekinumab every eight weeks were in remission, while 90.9% of patients and 94.3% of patients showed clinical response at week 92, respectively

2/21/17

Merck KGaA (Darmstadt, Germany)

Cladribine

Synthetic antineoplastic agent

Relapsing remitting multiple sclerosis

A post hoc analysis of data generated in its phase III trial, CLARITY, showed statistically significant reduction in brain atrophy in patients on a short course of investigational cladribine tablets over two years vs. placebo; patients with lower rates of brain atrophy showed the highest probability of remaining free from disability progression at two years

2/14/17

Pfizer Inc. (New York)

Xeljanz

Tofacitinib citrate

Moderate to severe rheumatoid arthritis

Top-line results from ORAL Strategy, a phase IIIb/IV study showed Xeljanz plus methotrexate met its primary endpoint in demonstrating noninferiority to Humira (adalimumab, Abbvie Inc.), while Xeljanz as a monotherapy did not meet the endpoint when compared to Humira plus MTX

2/17/17

Pfizer Inc. (New York) and Celltrion Healthcare Inc. (South Korea)

Inflectra

Infliximab CT-P13

Crohn's disease

Phase III data showed that treatment with Inflectra has similar efficacy and safety to treatment with Remicade (infliximab, Johnson & Johnson); the randomized, 54-week trial in 214 patients met its primary endpoint, demonstrating that, at six weeks, Inflectra was similar to Remicade in the treatment of Crohn's disease, thereby meeting the criterion for noninferiority

2/21/17

Takeda Pharmaceutical Co. Ltd. (Osaka, Japan)

Vedolizumab

A humanized monoclonal antibody designed to specifically antagonize the alpha4beta7 integrin

Moderately to severely active ulcerative colitis and Crohn's disease

Findings from two five-year interim analyses from the ongoing open-label GEMINI long-term safety study indicated that long-term treatment of vedolizumab in responders was associated with long-term clinical response and remission, in addition to health-related quality of life improvements for patients over a five-year period

2/21/17

Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) and Tigenix NV (Leuven, Belgium)

Cx601

A suspension of allogeneic expanded adipose-derived stem cells

Crohn's disease

Data from the phase III ADMIRE-CD trial showed that Cx601 maintained long-term remission of treatment refractory complex perianal fistulas over 52 weeks; a significantly greater proportion of patients in the Cx601 group vs. the control group achieved clinical and radiological combined remission (56.3% and 38.6%, p=0.010), and clinical remission (59.2% and 41.6%, p=0.013) at week 52 in the modified intention-to-treat population (mITT)

2/21/17

CANCER

Advaxis Inc. (Princeton, N.J.)

Axalimogene filolisbac

Lm Technology immunotherapy

High-risk locally advanced cervical cancer

A phase III trial enrolled and dosed its first patient

2/7/17

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

Rocapuldencel

Roca, formerly AGS-003

Metastatic renal cell carcinoma

Is stopping the pivotal phase III ADAPT trial for futility on the advice of the independent data monitoring committee (IDMC); during a planned interim analysis, the IDMC concluded the therapy was unlikely to show a statistically significant improvement in overall survival in the intent-to-treat population

2/23/17

Astrazeneca plc (London)

Lynparza

PARP inhibitor; olaparib tablets

HER2-negative metastatic breast cancer harboring germline BRCA1 or BRCA2 mutations

Results from its phase III OlympiAD trial showed a statistically significant and clinically meaningful improvement in progression-free survival compared with those who received chemotherapy (capecitabine, vinorelbine or eribulin)

2/21/17

Aveo Oncology Inc. (Cambridge, Mass.)

Tivozanib

A once-daily, VEGF receptor tyrosine kinase inhibitor

Refractory advanced renal cell carcinoma

Its pivotal, phase III TIVO-3 study, testing tivozanib against Nexavar (sorafenib, Bayer AG), completed the first safety review by the safety monitoring committee, which concluded that no safety concern was observed for tivozanib and recommended that the study replace the small number of patients who dropped out prior to starting treatment

2/24/17

Bionomics Ltd. (Adelaide, Australia)

BNC105

A vascular disrupting agent

Advanced melanoma

BNC105 will be evaluated in a combination trial with blockbuster PD-1 inhibitor Keytruda (pembrolizumab, Merck & Co. Inc.); the study will enroll patients with advanced cases of melanoma, who were unresponsive to standard treatments

2/22/17

Boston Biomedical Inc. (Cambridge, Mass.)

Napabucasin

An orally administered agent aimed at inhibiting cancer stemness pathways by targeting STAT3

Metastatic pancreatic cancer

Started dosing of the first patient in a phase III study, Canstem 111P, designed to test napabucasin in combination with standard of care – Abraxane (nab-paclitaxel, Celgene Corp.) plus gemcitabine)

2/22/17

Cyclacel Pharmaceuticals Inc. (Berkeley Heights, N.J.)

Sapacitabine

Nucleoside analogue

Acute myeloid leukemia (AML)

Top-line results from the pivotal phase III SEAMLESS study testing it in elderly patients with newly diagnosed AML, who are not candidates for or have refused intensive induction chemotherapy, showed the study did not meet the primary endpoint of demonstrating statistically significant improvement in overall survival (OS) vs. active control; an improved rate of complete remission, a secondary endpoint, was observed in patients who had discontinued therapy at the time of analysis

2/24/17

Daiichi Sankyo Group (Tokyo)

Tivantinib

Oral c-Met inhibitor

Hepatocellular carcinoma

Missed its primary endpoint of improving overall survival vs. placebo in the phase III trial, called METIV-HCC

2/21/17

Ergomed plc (London)

Zoptrex

Zoptarelin doxorubicin

Advanced, recurrent or metastatic endometrial cancer

Said co-development partner Aeterna Zentaris Inc., of Charleston, S.C., completed the pivotal phase III trial

2/1/17

Immune Pharmaceuticals Inc. (New York)

Ceplene

Histamine dihydrochloride

Acute myeloid leukemia

Is launching REMAIN, an international overall survival study testing the combination of Ceplene and low-dose Proleukin (IL-2); patient recruitment is set to start later this year

2/21/17

Janssen-Cilag International NV (Beerse, Belgium; unit of Johnson & Johnson)

Zytiga

Abiraterone acetate

Asymptomatic and mildly symptomatic metastatic castration-resistant prostate cancer

Data showed radiographic progression-free survival (rPFS) of 16.5 months and treatment duration of 11.6 months in men treated with Zytiga plus prednisone, in the real-world and outside the clinical trial setting

2/21/17

Kiadis Pharma NV

ATIR101

Allodepleted T-cell Immunotherapeutics

Acute leukemia

Initiated its randomized, controlled, transatlantic phase III trial

2/7/17

Loxo Oncology Inc. (Stamford, Conn.)

LOXO-101

Larotrectinib; an oral and selective drug targeting tropomyosin receptor kinases

Unresectable or metastatic solid tumors with NTRK-fusion proteins

Completed enrollment for the primary efficacy analysis in the pivotal trial testing larotrectinib

2/22/17

Northwest Biotherapeutics Inc. (Bethesda, Md.) 

Dcvax-L

Dendritic cell-based vaccine 

Glioblastoma multiforme

Updated its phase III trial, reporting that the partial clinical hold has been lifted by the FDA and the study has accumulated a sufficient number of events toward the progression-free survival endpoint but not yet for the overall survival endpoint

2/7/17

Novocure Ltd. (St. Helier, Jersey)

Tumor treating fields (TTFs)

Low intensity, alternating electric fields that disrupt cell division through physical interactions

Non-small-cell lung cancer (NSCLC)

The first patient was enrolled in LUNAR, its phase III pivotal trial to treat advanced NSCLC following failure in platinum-based therapy

2/16/17

Tracon Pharmaceuticals Inc. (San Diego)

TRC105

An antibody to endoglin

Cutaneous angiosarcoma

Started patient dosing in its phase III TAPPAS trial, which is being conducted under a special protocol assessment (SPA) with the FDA, and will randomize patients to receive either TRC105 in combination with Votrient (pazopanib, Glaxosmithkline plc)

2/17/17

Xbiotech Inc. (Austin, Texas)

Xilonix

Antibody therapy

Colorectal cancer

An independent data monitoring committee performed a prospectively planned, unblinded analysis of the phase III XCITE study of antibody therapy Xilonix and reported that the FDA fast-tracked study had no safety concerns, with indications of efficacy sufficient to recommend proceeding without modification; the primary endpoint is overall survival, with secondary endpoints including objective response rate, progression-free survival, change in lean body mass and patient-reported quality-of-life measures

2/22/17

CARDIOVASCULAR

Amgen Inc. (Thousand Oaks, Calif.)

Repatha

Evolocumab

High cholesterol

Results from the phase III FOURIER trial suggested that combining the antibody with optimized statin treatment could reduce the risk of heart attack and other cardiovascular (CV) events in patients with arterial plaque buildups; it met its primary composite endpoint and the key secondary composite endpoint and no new safety issues were observed

2/6/17

Ardelyx Inc. (Fremont, Calif.)

Tenapanor

Oral, experimental medication that blocks the NHE3 sodium transporter

Hyperphos-phatemia in patients with end-stage renal disease who are on dialysis

Phase III data showed it met its primary endpoint and was generally well-tolerated, with the responder population (n=80 out of 164) turning up a mean reduction in serum phosphorus from baseline to the end of the eight-week treatment period of 2.56 mg/dL, with a reduction of up to 5.7 mg/dL; in the group, 33% of patients had a reduction in serum phosphorus of greater than 3 mg/dL

2/16/17

Armetheon Inc. (Menlo Park, Calif.)

Tecarfarin

Oral anticoagulant therapy

Patients who require anticoagulation with a vitamin K antagonist

Reached agreement with the FDA for a single, 1,000-patient final pivotal trial for tecarfarin prior to filing an NDA; the study will be included in an amendment of the existing special protocol assessment

2/10/17

Armetheon Inc. (Menlo Park, Calif.)

Tecarfarin

Experimental oral anticoagulant

Mechanical and prosthetic heart valves, as well as those with renal dysfunction

The EMA's Scientific Advice Working Group will allow the company to run a single, 1,000-patient pivotal study prior to filing a market authorization application

2/15/17

Bioverativ Inc. (Waltham, Mass.) and Swedish Orphan Biovitrum AB (Stockholm)

Alprolix

Coagulation factor IX [recombinant] Fc fusion protein

Severe hemophilia B

Results from the Kids B-LONG phase III trial in previously treated children was generally well-tolerated and resulted in low bleeding rates in participants; children (n=30) treated prophylactically with Alprolix had a median annual bleed rate of two overall and zero spontaneous joint bleeds; 10 of 30 (33%) experienced no bleeding episodes, and 19 of 30 (63%) reported no joint bleeding on study; overall, 92% of bleeding episodes were controlled by one or two infusions of Alprolix

2/3/17

Janssen Research & Development LLC (Titusville, N.J.)

Xarelto

Rivaroxaban

Major adverse cardiac events (MACE), including cardiovascular death, myocardial infarction and stroke in patients with coronary artery disease or with peripheral artery disease

Said the phase III COMPASS trial, evaluating the efficacy and safety of Xarelto, is stopping earlier than planned based on the recommendation of the study's independent data monitoring committee, as the primary MACE endpoint has reached its pre-specified criteria for superiority

2/10/17

La Jolla Pharmaceutical Co. (San Diego)

LJPC-501

Angiotensin II

Catecholamine-resistant hypotension

Phase III data in the ATHOS-3 study, conducted under a special protocol assessment (SPA), showed the percentage of patients achieving a prespecified target blood pressure response, proved highly statistically significant: 23% of the 158 placebo-treated patients had a blood pressure response compared to 70% of the 163 LJPC-501-treated patients (p<0.00001); a trend toward longer survival turned up as well, namely a 22% reduction in mortality risk through day 28

2/28/17

Nono Inc. (Toronto)

NA-1

PSD-95 inhibitor

Acute ischemic stroke

Started a phase III trial, termed ESCAPE-NA-1, to test the efficacy and safety of NA-1 for reducing functional disability in patients with acute ischemic stroke who are candidates for endovascular revascularization

2/28/17

Tenax Therapeutics Inc. (Morrisville, N.C.)

Levosimendan

Calcium sensitizer/K-ATP activator

Reduction of morbidity and mortality in cardiac surgery patients at risk for developing low cardiac output syndrome

It failed to meet the co-primary endpoints of a phase III study conducted under an FDA special protocol assessment; treatment with the medicine failed to achieve statistically significant reductions in the dual endpoint of death or use of a mechanical assist device at 30 days and in the quad endpoint of death, myocardial infarction, need for dialysis or use of a mechanical assist device at 30 days

2/1/17

CENTRAL NERVOUS SYSTEM

Amag Pharmaceuticals Inc. (Waltham, Mass.) and Palatin Technologies Inc. (Cranbury, N.J.)

Rekynda

Bremelanotide

Premenopausal women with hypoactive sexual desire disorder

Additional results from the phase III RECONNECT studies (301, 302) of Rekynda showed both studies met their co-primary endpoints of improvements in desire and decrease in associated distress; Rekynda was associated with statistically significant improvements in the total FSFI score, including arousal, lubrication, orgasm and satisfaction domain scores (all p≤0.01)

2/28/17

Collegium Pharmaceutical Inc. (Canton, Mass.)

Xtampza ER 

Oxycodone extended-release capsules

Drug abuse

Top-line data from a trial to evaluate the abuse potential and pharmacokinetics of oral administration compared to chewed Xtampza ER and crushed immediate-release oxycodone in solution in non-dependent, recreational drug abusers showed the study met its primary and secondary endpoints of statistically significant lower "drug liking" and "take drug again"; when taken chewed, Xtampza ER also was bioequivalent to taking it intact

2/2/17

H. Lundbeck A/S (Valby, Denmark)

Idalopirdine

Selective 5-HT6 receptor antagonist

Alzheimer's disease (AD)

Said a pair of phase III trials with idalopirdine in AD failed, and the company and partner Tokyo-based partner Otsuka Pharmaceutical Co. Ltd. will not try to gain approval

2/9/17

Recro Pharma Inc. (Malvern, Pa.)

N1539

Intravenous meloxicam

Hip and knee replacements, hernia repair and spinal, colorectal and other major surgeries

Completed enrollment in its double-blind phase III study

2/28/17

Sunovion Pharmaceuticals Inc. (Marlborough, Mass.)

Latuda

Lurasidone HCl

Depression associated with bipolar I disorder

Its phase III trial testing Latuda in children and adolescents, ages 10 to 17, with depression associated with bipolar I disorder met its primary endpoint, the change from baseline to week six on the Children Depression Rating Scale, Revised total score, and the key secondary endpoint was change from baseline to week six on the Clinical Global Impression-Bipolar Version, Severity of Illness score for depression

2/28/17

Trevena Inc.

Olinvo

Oliceridine

Moderate to severe acute pain following bunionectomy and abdominoplasty

Top-line results from its pivotal phase III APOLLO-1 and -2 efficacy studies showed that all dose regimens across both trials achieved the primary endpoint of statistically greater analgesic efficacy than placebo; it also showed dose-related trends of improvements vs. morphine on multiple measures of respiratory safety and gastrointestinal tolerability in acute pain management

2/22/17

Zogenix Inc. (Emeryville, Calif.)

ZX008

Fenfluramine hydrochloride

Dravet syndrome

Started the clinical efficacy portion of phase III study 1504 to test ZX008, clobazam and valproate when added to stiripentol, followed by a randomized, double-blind, placebo-controlled parallel group evaluation of the efficacy, safety and tolerability as adjunctive antiepileptic therapy to a drug regimen that includes stiripentol, clobazam and valproate

2/14/17

Zosano Pharma Corp. (Fremont, Calif.)

M207

Zolmitriptan-coated microneedle patch

Migraine

It met both co-primary endpoints the pivotal phase III Zotrip trial, delivering freedom from pain at two hours for 41.5% of treated patients; both results bested a placebo at two hours after treatment, 41.5% of patients treated with the 3.8-mg dose patch reported freedom from pain vs. just 14.3% for a placebo patch (p < 0.0001); at the same time point, 68.3% of treated patients reported being free of their most bothersome symptom vs. 42.9% in the placebo arm (p < 0.0009); the 3.8-mg version of the quarter-sized patch also helped about 27% of patients achieve freedom from pain just one hour after treatment vs. about doing the same for only about 10% of those given a placebo (p < 0.0084)

2/14/17

GASTROINTESTINAL

Redhill Biopharma Ltd. (Tel Aviv, Israel)

Bekinda 24 mg

Extended-release, once-daily oral pill formulation of ondansetron

Acute gastroenteritis and gastritis

Enrolled the last patient in a phase III study

2/14/17

Synergy Pharmaceuticals Inc. (New York)

Trulance

Plecanatide

Chronic idiopathic constipation

Results from a pivotal phase III trial demonstrated the efficacy and safety of Trulance, which is designed to work by replicating the function of uroguanylin

2/8/17

INFECTION

Cempra Inc. (Chapel Hill, N.C.)

Taksta

Oral antibiotic; fusidic acid

Acute bacterial skin and skin structure infections

Early clinical responses to Taksta proved noninferior to those for Pfizer Inc.'s oral Zyvox (linezolid) during a phase III trial, meeting the primary endpoint

2/27/17

Chimerix Inc. (Durham, N.C.)

Brincidofovir

Nucleotide analogue

Adenovirus (AdV) infection

Final data from the AdVise trial in allogeneic hematopoietic cell transplant (HCT) recipients revealed declines in AdV viral load of ≥2 log10 c/mL or below the limit of detection at week four were observed in 76% of pediatric patients and 45% of adult patients; in cohort A, 55% of patients with baseline low immunity (CD4 counts <50 cells/μL) achieved ≥2 log10 c/mL decline or undetectable AdV at week four, while in cohort B, 52% of patients with baseline low immunity achieved ≥2 log10 c/mL decline or undetectable AdV over the same period of time

2/23/17

Gilead Sciences Inc. (Seattle)

Genvoya

Elvitegravir 150 mg, cobicistat 150 mg, emtricitabine 200 mg and tenofovir alafenamide 10 mg

HIV-1 infection

Data from two phase III studies (Studies 104 and 111) evaluating the safety and efficacy in treatment-naïve adults showed that through week 144, it demonstrated significantly higher rates of virologic suppression compared to Gilead's Stribild, based on the percentage of patients with HIV-1 RNA levels less than 50 copies/mL; patients receiving Genvoya also demonstrated favorable renal and bone laboratory parameters compared to those treated with Stribild

2/15/17

Janssen Sciences Ireland (Cork, Ireland; unit of Johnson & Johnson)

Combo of Viiv Healthcare's DTG and Janssen's Edurant

Dolutegravir and rilpivirine

HIV

Phase III Sword 1 and 2 trial results showed the combo achieved non-inferior viral suppression (HIV-1 RNA <50 c/mL) at 48 weeks compared with a three- or four-drug regimen in both pooled and individual analyses

2/15/17

Merck & Co. Inc. (Kenilworth, N.J.)

MK-8228

Letermovir; a member of the class of non-nucleoside CMV inhibitors known as 3,4 dihydroquinazolines

Cytomegalo-virus

Results of a phase III study showed letermovir met its primary endpoint, demonstrating that significantly fewer patients with undetectable cytomegalovirus (CMV) DNA at the start of study treatment developed clinically significant CMV infection through week 24 post-hematopoietic stem cell transplant (HSCT)

2/28/17

Motif Bio plc (London)

Iclaprim

Antibiotic

Acute bacterial skin and skin structure infections

The last patient finished the treatment phase in REVIVE-1, the phase III trial

2/1/17

Theratechnologies Inc. (Montreal)

TMB-301

Ibalizumab

HIV-1 infection

Additional results from the 24-week ibalizumab phase III trial showed that patients with multidrug-resistant HIV-1 infection experienced a mean increase in CD4+ T cell of 48 cells/µL after 24 weeks of treatment with ibalizumab plus an optimized background regimen

2/16/17

VBI Vaccines Inc. (Cambridge, Mass.)

Sci-B-Vac

Vaccine

Hepatitis B

Received positive scientific advice from the EMA's Committee for Medicinal Products for Human Use (CHMP) regarding the development path in Europe; the vaccine demonstrated safety and efficacy in more than 300,000 patients in currently licensed markets, and the CHMP expressed its support of VBI's proposed plan to proceed to the phase III studies

2/8/17

Nabriva Therapeutics AG (Vienna)

Lefamulin

Semi-synthetic pleuromutilin antibiotic

Moderate to severe community-acquired bacterial pneumonia

Following a blinded interim analysis, an independent committee recommended no sample size adjustment for the lefamulin evaluation against pneumonia, or LEAP 1, phase III trial testing it compared to moxifloxacin

2/3/17

Summit Therapeutics plc (Oxford, U.K.)

Ridinilazole

Antibiotic

C. difficile infection (CDI) 

Outlined its phase III program following recent regulatory meetings with the FDA and European Medicines Agency; Summit intends to design the trial to evaluate superiority of ridinilazole over standard of care in the treatment of CDI

2/2/17

INFLAMMATORY

Flexion Therapeutics Inc. (Burlington, Mass.)

Zilretta

FX006

Osteoarthritis (OA) of the knee

Enrolled the first patient in a study evaluating the safety of repeat administration of Zilretta

2/24/17

MISCELLANEOUS

Acacia Pharma Group Ltd. (Cambridge, U.K.)

Baremsis

Amisulpride, formerly APD421

Postoperative nausea and vomiting (PONV)

Its fourth and final pivotal phase III study in PONV that occurred despite prophylaxis with standard anti-emetics met the primary endpoint of complete response, measured as successful resolution of PONV without recurrence or need for further antiemetic rescue in the 24-hour period following rescue treatment; of 2,285 patients, 705 (31%) went on to experience PONV and were randomized into the trial

2/14/17

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

Macrilen

Macimorelin

Adult growth hormone deficiency

Completed a full review of data from its confirmatory phase III trial for use as a growth hormone stimulation test and concluded the data were sufficient to make a second run at registration with the FDA

2/14/17

Amag Pharmaceuticals Inc. (Waltham)

Makena

Hydroxyprogesterone caproate injection

To reduce the risk of preterm birth

Pharmacokinetic results showed Makena administered subcutaneously demonstrated bioequivalence to the intramuscular (IM) injection on area under the curve (AUC0-to-inf 2,386 ng/mL compared to 2,086 ng/mL), with the 90% confidence interval for the ratio of AUC (105.17 to 124.39) falling within the 80% to 125% range; the mean maximum or peak plasma concentration (Cmax) for Makena subcutaneous was slightly higher than for the IM (7.3 ng/mL compared to 6.3 ng/mL), with the 90% confidence interval for the ratio of Cmax (96.6 to 138.7%) falling outside of the bioequivalence range of 80% to 125%

2/3/17

Clearside Biomedical Inc. (Alpharetta, Ga.)

Zuprata

Suspension formulation of corticosteroid triamcinolone acetonide

Macular edema associated with retinal vein occlusion

Enrolled the first patient in a phase III trial, dubbed SAPPHIRE, testing Zuprata in combination with Eylea (aflibercept, Regeneron Pharmaceuticals Inc.)

2/17/17

Cumberland Pharmaceuticals Inc. (Nashville, Tenn.)

Caldolor injection

Ibuprofen

Fever

A multicenter study demonstrated that it delivered significant fever reduction in hospitalized children and that a single 10-mg/kg dose of intravenous ibuprofen provided a significant reduction of temperature for febrile pediatric patients vs. 10 mg/kg acetaminophen at two hours and four hours post-treatment

2/8/17

Gensight Biologics SA (Paris)

GS010

Gene therapy

Leber's hereditary optic neuropathy

Completed enrollment in the phase III REVERSE study, the first of two parallel randomized, double-masked, sham-controlled trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene

2/22/17

Intercept Pharmaceuticals Inc. (New York)

OCA

Obeticholic acid

Nonalcoholic steatohepatitis (NASH)

Following formal consultation with FDA, Intercept now only needs to achieve one endpoint of the two co-primary endpoints in REGENERATE, its phase III trial, to be considered successful; the two endpoints are proportion of OCA-treated patients relative to placebo achieving at least one stage of liver fibrosis improvement with no worsening NASH and the proportion of OCA-treated patients relative to placebo achieving NASH resolution with no worsening of liver fibrosis

2/13/17

Mediwound Ltd. (Yavne, Israel)

Nexobrid

Contains a concentrate of proteolytic enzymes enriched in bromelain

Severe thermal burns

The EMA endorsed the extension of the CIDS (Children Innovative Debridement Study) population to include patients ages 1 to 18; Mediwound will initiate the second stage of the phase III study that allows inclusion of younger pediatric burn patients beginning at the minimum age of 1 instead of 4. 

2/7/17

Mithra Pharmaceuticals SA (Liège, Belgium)

Estelle

Combined oral contraceptive based on estetrol (E4) and drospirenone (DRSP)

Birth control

Completed recruitment into the phase III study in Europe and Russia

2/8/17

Ohr Pharmaceutical Inc. (New York)

Squalamine lactate

Ophthalmic solution

Neovascular age-related macular degeneration

Paused enrollment in the first phase III trial; the company said the decision was not related to any safety issue; the trial will continue with its current protocol for patients already enrolled

2/15/17

Radius Health Inc. (Waltham, Mass.)

Abaloparatide-SC

Peptide designed to engage the parathyroid hormone receptor

Postmenopausal osteoporosis

ACTIVExtend trial showed that patients previously treated with daily abaloparatide-SC for 18 months in the ACTIVE trial who were transitioned to alendronate for six months of follow-up had significantly greater reduction of 87% in the incidence of new vertebral fractures (p< 0.001), a reduction of 52% in nonvertebral fractures (p = 0.02) and a reduction of 58% in major osteoporotic fractures (p < 0.01) vs. placebo patients also transitioned to alendronate

2/2/17

Regenerx Biopharma-ceuticals Inc. (Rockville, Md.)

RGN-137

Thymosin beta 4-based dermal wound healing gel

Epidermolysis bullosa

Received a positive response from the FDA for its phase III trial design; the randomized, multicenter, double-blind, placebo-controlled study will evaluate the efficacy and safety of RGN-137 topically administered to about 200 EB patients

2/28/17

RESPIRATORY

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

Relvar Ellipta

Fluticasone furoate/vilanterol; inhaled corticosteroid/long-acting beta2agonist combination

Asthma

Results from a noninferiority lung function study demonstrated that patients with well-controlled asthma were able to switch to the once-daily Relvar Ellipta from the twice-daily Seretide Accuhaler, without compromising their lung function

2/24/17

Pharmaxis Ltd. (Sydney)

Bronchitol

Mannitol

Cystic fibrosis

The last of 423 patients has concluded treatment in its international phase III trial

2/24/17

Notes

Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.

The date indicated refers to the BioWorld Today issue in which the news item can be found.

For more information about individual companies and/or products, see Thomson Reuters Cortellis.