Company (Location)

Product

Description

Indication

Status (Date)#

CANCER

AB Science SA (Paris)

Masitinib

Orally administered tyrosine kinase inhibitor

Gleevec-resistant gastrointestinal stromal tumors

Phase II data of masitinib against Sutent showed median overall survival was not reached for masitinib vs. 15 months for Sutent (2/2)

Active Biotech AB (Lund, Sweden) and Ipsen SA (Paris)

TASQ

Tasquinimod

Castrate-resistant prostate cancer

Phase II data showed treatment side effects were mild to moderate, manageable and less frequent after two months of therapy, while adverse events included gastrointestinal disorders, fatigue and musculoskeletal pain (2/27)

Advaxis Inc. (Princeton, N.J.)

ADXS-HPV

An immunmotherapy

Cervical intraepithelial neoplasia 2/3 (precursor to cervical cancer)

Completed the first three dose cohorts in the Phase II trial, and first cohort results showed 52% of lesions regressed to CIN 1 or normal; no serious adverse events were reported (2/28)

Apeiron Biologics AG (Vienna, Austria)

APN201

Superoxide dismutase

To prevent radiation dermatitis in breast cancer

Started a double-blind, placebo-controlled trial (2/28)

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

AGS-003

Arcelis immunotherapy

Advanced renal cell carcinoma

Phase II data of AGS-003 in combination with Sutent showed a statistically significant correlation between antitumor memory T-cell responses and overall survival (2/1); Phase II data showed treatment prolonged survival in patients with unfavorable-risk metastatic renal cell carcinoma (2/7)

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

Sapacitabine

A nucleoside analogue

Myelo-dysplastic syndromes

Top-line response data from its Phase II trial showed that eight patients responded with two complete remissions (2/14)

Geron Corp. (Menlo Park, Calif.)

Imetelstat

Telomerase inhibitor

Metastatic breast cancer

Completed enrollment in a Phase II trial (2/6)

GTx Inc. (Memphis, Tenn.)

Capesaris

GTx-758

Advanced prostate cancer

FDA placed a clinical hold on its three ongoing Phase II and Phase IIb trials due to an increased risk of venous thromboembolic events, or blood clots (2/22)

Ligand Pharmaceuticals Inc. (San Diego)

PG-free Melphalan

Captisol-enabled propylene glycol-free melphalan

Cancer

Phase II data showed that the trial met all endpoints (2/3)

OncoGenex Pharmaceuticals Inc. (Bothell, Wash.)

OGX-427

Hsp7 inhibitor

Metastatic castrate-resistant prostate cancer

Phase II data showed 71% of chemotherapy-naive patients treated with OGX-427 plus prednisone had no disease progression at 12 weeks compared with 33% of those treated with prednisone alone (2/3)

OncoSec Medical Inc. (San Diego)

OMS

Electroimmuno-therapy

Merkel cell carcinoma

Treated several patients in a Phase II trial (2/15)

OncoSec Medical Inc. (San Diego)

OMS

ElectroImmuno-therapy

Metastatic melanoma

The first patient has been dosed in a Phase II trial (2/29)

Plexxikon Inc. (unit of Daiichi Sankyo Group) and Roche AG (Basel, Switzerland)

Zelboraf

Vemurafenib

Metastatic melanoma

Phase II data showed it nearly doubled median survival times (2/23)

Threshold Pharmaceuticals Inc. (South San Francisco)

TH-302

A second-generation hypoxia-targeted molecule

First-line advanced pancreatic cancer

Phase IIb data showed it hit its primary endpoint (2/22)

Titan Pharma Ltd. (Jerusalem)

TL-118

Designed to inhibit several nonoverlapping pathways involved in angiogenesis

Pancreatic cancer

Started a Phase II trial (2/14)

Ziopharm Oncology Inc. (New York)

Zymafos

Palifosfamide, ZIO-201

Soft-tissue sarcoma

Phase II data of Zymafos plus doxorubicin vs. doxorubicin alone showed a meaningfully positive trend favoring Zymafos; two years after treatment, about 40% of Zymafos patients were alive, compared with 30% of control patients (2/14)

CARDIOVASCULAR

Remedy Pharmaceuticals Inc. (New York)

RP-1127

An NCCa-ATP channel inhibitor

Severe stroke

Phase II data showed that major swelling occurred in 12.5% of the treated group compared to 88% in the historical group; no significant hemorrhages were found in treated patients, compared to 30% in the historical group (2/7)

DIABETES

Mesoblast Ltd. (Melbourne, Australia)

Mesenchymal Precursor Cells

Off-the-shelf, allogeneic MPCs

Type II diabetes

FDA cleared the firm to start a Phase II trial (2/1)

SK Biopharma-ceuticals (Seoul, South Korea)

SKL11197

New chemical entity

Diabetic peripheral neuropathy

Began a Phase II trial (2/23)

INFECTION

Affinium Pharmaceuticals Inc. (Austin, Texas)

AFN-1252

Oral antibiotic

Acute bacterial skin and skin structure infections

Dosed the first patient in a Phase II trial (2/2)

Agennix AG (Planegg, Germany)

Talactoferrin

Recombinant version of lactoferring

Sepsis

A data safety monitoring board found 28-day mortality was greater in the talactoferrin arm than in the placebo arm, curtailing the Phase II/III trial (2/3)

AiCuris GmbH & Co. KG (Wuppertal, Germany)

Letermovir

AIC246

HCMV-seropositive allogeneic human blood precursor cell recipients

Phase II data showed that it demonstrated efficacy and superior tolerability in a placebo-controlled, dose-ranging trial (2/15)

Anacor Pharmaceuticals Inc. (Palo Alto, Calif.) and GlaxoSmithKline plc (London)

GSK2251052

A systemic antibiotic

Gram-negative infections

GSK suspended enrollment in Phase I and II trials due to a microbiological finding in patients enrolled in the Phase IIb trial in complicated urinary tract infections; the finding was unrelated to safety but could have the potential to reduce efficacy (2/7)

BiondVax Pharmaceuticals Ltd. (Nes Ziona, Israel)

BVX-005

Universal influenza vaccine

Influenza

Phase II data showed it was safe and well tolerated (2/17)

Chimerix Inc. (Research Triangle Park, N.C.)

CMX001

A lipid-antiviral-conjugate designed to deliver cidofovir-diphosphate

To prevent cytomegalovirus viremia and/or CMV disease

Phase II data showed it produced a statistically significant benefit vs. placebo 13 weeks post-hematopoietic stem cell transplant (2/7)

Gilead Sciences Inc. (Foster City, Calif.)

GS-7977

Nucleotide analogue polymerase inhibitor

Hepatitis C virus

Phase II data showed that six out of eight patients with a prior null response to an interferon regimen relapsed within four weeks of completing a course of GS-7977 plus ribavirin (2/21)

Idenix Pharmaceuticals Inc. (Cambridge, Mass.)

IDX184

A pan-genotypic oral nucleotide polymerase inhibitor

Hepatitis C virus

A partial clinical hold has been removed by the FDA, after Phase IIb data and a recommendation from a data safety monitoring board showed the source of liver toxicity was IDX320 (2/6)

Moberg Derma AB (Stockholm, Sweden)

MOB-015

A topical drug

Nail fungus

Phase II data showed it is unlikely to produce results sufficient for out-licensing (2/10)

Vertex Pharmaceuticals Inc. (Cambridge, Mass.)

VX-222

Interferon-free non-nucleoside polymerase inhibitor

Hepatitis C virus

Phase II data of VX-222 in combination with Incivek and ribavirin in genotype Ia or 1b HCV showed viral loads below the lower limit of quantification for 80% of patients at week two, and 83% of patients at week 12 (2/24)

MISCELLANEOUS

Genzyme (Cambridge, Mass.; unit of Sanofi SA; Paris)

Eliglustat tartrate

An oral glucosylceramide analogue

Gaucher disease

Four-year follow-up data from a Phase II trial showed that patients with Type I disease who were treated with eliglustat tartrate demonstrated sustained or further improvement across all endpoints, including markers of bone disease (2/9)

PregLem SA (Geneva)

Esmya

Ulipristal

Uterine fibroids

Data from the PEARL I and PEARL II studies confirmed Esmya's activity in improving symptoms of uterine fibroids; data showed it controlled bleeding in more than 90% of patients (2/3)

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.