Company
(location)

Product

Description

Indication

Status

Date

CANCER

Amgen Inc. (Thousand Oaks, Calif.)

Blinatumomab

Bispecific antibody B-precursor

Newly diagnosed negative B-lineage acute lymphoblastic leukemia

Launched a randomized phase III trial comparing overall survival after treatment with blinatumomab in combination with chemotherapy vs. chemotherapy alone in adults ages 35 to 70 years

1/13/14

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

CPX-351

Cytarabine: daunorubicin; liposome injection

High-risk acute myeloid leukemia

Phase III data of CPX-351 vs. the conventional cytarabine and daunorubicin treatment regimen as a first-line therapy in older patients has reached 50% of the study’s planned enrollment of 300 patients

1/10/14

Cell Therapeutics Inc. (Seattle)

Opaxio

Paclitaxel poliglumex

Ovarian cancer

Completed patient enrollment in the GOG-0212 phase III trial testing Opaxio as a maintenance therapy; the primary endpoint is overall survival, while secondary endpoints include progression-free survival, safety and quality of life

1/29/14

Celsion Corp. (Lawrenceville, N.J.)

Thermodox

Heat-activated liposomal encapsulation of doxorubicin

Primary liver cancer

Phase III HEAT study data supported the continued clinical development in a prospective phase III pivotal study; although HEAT did not meet its primary endpoint of progression-free survival, in the patient subgroup treated in the Thermodox arm whose RFA procedures lasted longer than 45 minutes, clinical results indicated a 55% improvement in overall survival (p = 0.0495)

1/28/14

Immunomedics Inc. (Morris Plains, N.J.)

90Y

Yttrium-90-clivatuzumab tetraxetan

Pancreatic cancer

Launched a phase III registration study

1/13/14

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

Xtandi

Enzalutamide; oral androgen receptor inhibitor

Post-chemotherapy

Phase III data showed a statistically significant overall survival benefit compared with placebo, reducing the risk of death by 29% (HR = 0.71; p < 0.0001), compared with placebo, and the benefit held despite the use of subsequent therapies, at a rate of 40% in the Xtandi arm, 70% in placebo

1/30/14

Patrys Ltd. (Melbourne, Australia)

PAT-SC1

The target antigen, CD55, is an isoform of the membrane-bound CD55

Gastric cancer

Data from a 10 year follow-up study showed that survival is significantly higher for PAT-SC1 treated patients as compared to an historical control group of patients who underwent a gastric resection but were not treated with PAT-SC1, which was well tolerated in all treated patients with no significant side effects

1/23/14

Pharmacyclics Inc. (Sunnyvale, Calif.) and Janssen Biotech Inc. (unit of Johnson & Johnson; New Brunswick, N.J.)

Imbruvica

Ibrutinib

Relapsed or refractory chronic lymphocytic leukemia or small lymphocytic

An independent data monitoring committee unanimously recommended the phase III study of Imbruvica vs. Arzerra be stopped early because the primary endpoint and a key secondary endpoint have been met

1/8/14

CARDIOVASCULAR

Amgen Inc. (Thousand Oaks, Calif.)

Evolocumab

Anti-PCSK9 antibody

High cholesterol

Phase III data in patients with high cholesterol who cannot tolerate statins met its co-primary endpoints

1/24/14

Cardium Therapeutics Inc. (San Diego)

Generx

Alferminogene tadenovec, Ad5FGF-4

Myocardial ischemia due to arteriosclerosis

Phase III data suggest that a one-time non-surgical administration of Generx can boost cardiac blood flow (myocardial perfusion)

1/2/14

Emmaus Medical Inc. (Torrance, Calif.)

L-glutamine

Levoglutamide

Sickle cell disease

Patient visits were completed in the phase III trial

1/9/14

Oxygen Biotherapeutics Inc. (Morrisville, N.C.)

Levosimendan

Calcium sensitizer

Cardiac disease

Company is moving the drug into a phase III study under an SPA; it will enroll about 760 cardiac patients

1/7/2014

CENTRAL NERVOUS SYSTEM

Endo Pharmaceuticals Inc. (Malvern, Pa.; subsidiary of Endo Health Solutions Inc.) and Biodelivery Sciences International Inc. (Raleigh, N.C.)

BEMA

Buprenorphine

Chronic pain

Top-line results from the pivotal phase III study showed it met its primary efficacy endpoint, showing that BEMA buprenorphine resulted in improved chronic pain relief (p < 0.005) compared to placebo

1/27/14

Envivo Pharmaceuticals Inc. (Watertown, Mass.)

Encenicline-hydrochloride

Alpha-7 potentiator

Alzheimer's disease

Launched a second phase III program

1/23/14

GASTROINTESTINAL

NPS Pharmaceuticals Inc. (Bedminster, N.J.)

Gattex

Teduglutide (rDNA origin)

Short bowel syndrome

Started a global registration study in pediatric patients who are dependent on parenteral support

1/3/14

INFECTION

Melinta Therapeutics Inc. (New Haven, Conn.)

Delafloxacin

A fluoroquinolone

Uncomplicated gonorrhea

Began a phase III trial

1/10/14

Paxvax Inc. (Menlo Park, Calif.)

PXVX0200

Also known as CVD 103-HgR; oral vaccine

Cholera

Interim phase III data showed the vaccine appeared well tolerated and the rate of diarrhea was reduced in the vaccine group compared with placebo

1/10/14

INFLAMMATORY

Ampio Pharmaceuticals Inc. (Greenwood, Colo.)

Ampion

Aspartyl-alanyl diketopiperazine

Osteoarthritis of the knee

Received institutional review board approval and FDA investigational new drug application clearance, and patient enrollment has commenced for a phase III trial

1/14/14

Shield Therapeutics Ltd. (London)

ST10

An oral ferric iron replacement therapy

Anemia caused by inflammatory bowel disease

Pivotal phase III data showed the product could replace the need to administer intravenous iron; patients receiving the therapy had a mean increase in hemaglobin levels of 2.3 grams/deciliter at 12 weeks, meeting the primary endpoint

1/7/14

MISCELLANEOUS

Debiopharm Group (Lausanne, Switzerland)

Triptorelin pamoate

A 22.5 mg, six-month formulation

Central precocious puberty

Completed the recruitment of patients for a phase III study

1/14/14

Mesoblast Ltd. (Melbourne, Australia)

MPCs

Allogeneic mesenchymal precursor cells

Chronic moderate to severe discogenic low-back pain

Phase III data showed a single injection into degenerating intervertebral discs reduced low-back pain and improved function for at least 12 months

1/31/14

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

NX-1207

BPH candidate

Benign prostatic hyperplasia

Two pivotal studies – NX02-0017 and NX02-0018 – have completed enrollment

1/29/14

Prosensa Holding N.V. (Leiden, the Netherlands)

Drisapersen

An antisense oligonucleotide which induces exon skipping of exon 51

Duchenne muscular dystrophy

Phase III data showed a 49 m difference between those on continual treatment (n = 52) and those who had been on placebo for 48 weeks followed by active drug (n = 31); those previously participating in the DMD114117 study (DEMAND II) showed a 52 m difference at 96 weeks

1/17/14

Relypsa Inc. (Redwood City, Calif.)

Patiromer

An oral potassium binder

Hyperkalemia

Results from the pre-defined exploratory endpoints from its two-part pivotal phase III trial show that as a result of recurrent hyperkalemia significantly more placebo patients required dose modification of their RAASi therapies (62%) than patiromer patients (6%); with more patiromer patients (94%) still on RAASi medication at the end of the trial than placebo patients (48%)

1/14/14

Repros Therapeutics Inc. (The Woodlands, Texas)

Androxal

Enclomiphene

Testosterone replacement therapy

Initiated two clinical studies comparing Androxal to the leading U.S. testosterone replacement therapy; each study will enroll 120 men, 40 each into three parallel arms: Androxal, approved topical gel and placebo

1/9/14

ZS Pharma Inc. (Coppell, Texas)

ZS-9

A highly selective oral sorbent designed specifically to trap potassium ions

Hyperkalemia

Reported top-line results from ZS003, its pivotal phase III trial, showing the drug met the pre-specified endpoint for the 12-day extended treatment phase, demonstrating that both 5 g and 10 g of ZS-9 given once-daily maintained a statistically and clinically significant reduction in serum potassium (K+) relative to placebo; it also was well tolerated with a gastrointestinal (GI) adverse event rate and an overall adverse event rate similar to placebo across all doses tested

1/14/14

Notes

BLA = Biologics license application; CMA = Continuous marketing application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment.

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.