Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Anaptysbio Inc. (San Diego)

ANB020

Anti-interleukin-33 therapeutic antibody

Atopic diseases

Top-line data from phase I trial that enrolled 96 healthy participants who received either a single subcutaneous or intravenous dose of ANB020 ranging between 10 mg and 750 mg, or four multiple doses ranging between 40 mg and 300 mg over a period of four consecutive weeks, showed the drug was well-tolerated and no dose-limiting toxicities were observed for any dosage

10/4/16

Apitope Ltd. (Hasselt, Belgium)

ATX-GD-59

Peptide therapy

Graves' disease

First patient was enrolled in a phase I trial that will recruit up to 30 patients

10/27/16

Celgene International Sarl (unit of Celgene Corp.; Summit, N.J.)

GED-0301

Mongersen

Active Crohn's disease

Data from a randomized, double-blind, multicenter, exploratory phase Ib study showed clinical improvement by week two, and clinical response (CDAI decrease ≥100) and remission (CDAI <150) rates were highest in the 12-week treatment group, at 67% and 48%, respectively

10/18/16

Coherus Biosciences Inc. (Redwood City, Calif.)

CHS-0214

Etanercept biosimilar

Autoimmune disorders

A phase I study achieved the primary pharmacokinetic (PK) BE endpoint, as the 90% confidence intervals for the geometric mean ratio for the two groups was within 80% to 125% for all PK parameters

10/10/16

Exicure Inc. (Skokie, Ill.)

AST-005

A spherical nucleic acid

Mild to moderate psoriasis

Results from its phase I trial demonstrated the drug met the safety and tolerability requirements

10/12/16

CANCER

Adaptimmune Therapeutics plc (Oxford, U.K.)

Fludarabine

NY-ESO SPEAR (Specific Peptide Enhanced Affinity Receptor) T-cell therapy

Ovarian cancer

The amended protocol for a phase I/II trial is now actively recruiting

10/13/16

Adaptimmune Therapeutics plc (Oxford, U.K.)

MAGE-A10 SPEAR

Specific peptide enhanced affinity receptor T-cell therapy

Inoperable or metastatic urothelial cancer, melanoma or squamous cell carcinoma of the head and neck

Started a phase I triple tumor study

10/19/16

Advaxis Inc. (Princeton, N.J.)

ADXS-PSA

Lm immunotherapy candidate

Metastatic castration-resistant prostate cancer (mCRPC)

Started part B of the KEYNOTE-046 trial testing the candidate in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in patients with previously treated mCRPC

10/21/16

Amcure GmbH (Eggenstein-Leopoldshafen, Germany)

AMC303

Targets CD44v6

Cancer

Treated the first patient in a phase I/Ib study

10/28/16

Amgen Inc. (Thousand Oaks, Calif.)

Blincyto

Blinatumomab

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

Results from the phase I/II '205 single-arm trial showed that 27 patients (39%) achieved complete remission within the first two cycles; among those patients, 52% had a complete minimal residual disease (MRD) response, a measure of eradication of residual disease at the molecular level

10/5/16

Aptose Biosciences Inc. (San Diego)

APTO-253

Anticancer small molecule

Hematologic cancers

Received a response from the FDA regarding the clinical hold for its phase Ib trial, with the agency requesting additional information, focusing exclusively on chemistry, manufacturing and control information on the final GMP drug product, and informing the firm that the hold would not be removed until that information was submitted and reviewed; Aptose said the request was due to the change to the drug substance from a salt to a free base, which modified the formulation

10/14/16

Beigene Ltd. (Beijing)

BGB-3111

BTK inhibitor

Waldenström's macroglobulinemia (WM)

Updated phase I data demonstrated it is well-tolerated and highly active in WM, with an overall response rate of 92%, including major responses in 83% and very good partial responses in 33% of patients, at a median follow-up time of eight months

10/10/16

Cellectar Biosciences Inc. (Madison, Wis.)

CLR 131

A single-dose infusion based on the phospholipid-drug conjugate technology designed to deliver cytoxic radioisotope iodine-131 directly to tumor cells

Relapsed or refractory multiple myeloma

Enrolled the first patient into the third cohort of the phase I study

10/7/16

Checkpoint Therapeutics Inc. (New York)

CK-101

Novel, oral, third-generation epidermal growth factor receptor inhibitor

Advanced solid tumors

The first patient has been dosed in a phase I/II study

10/4/16

Daiichi Sankyo Co. Ltd. (Tokyo)

DS-8201a

A novel HER2-targeting antibody-drug conjugate

Various cancers

Was well tolerated with no dose-limiting toxicities, and is moving into the second part of the phase I trial

10/13/16

Eisai Inc. (Woodcliff Lake, N.J.)

Lenvima

Lenvatinib; multiple receptor tyrosine kinase inhibitor

Solid tumors

Phase Ib data showed that Lenvima, in combination with anti-PD-1 drug Keytruda (pembrolizumab, Merck & Co. Inc.), resulted in two dose-limiting toxicities (DLTs) at the lenvatinib dose level in one group and no DLTs in another group testing lenvatinib 20 mg plus pembrolizumab 200 mg group, which was confirmed as the maximum tolerated dose

10/17/16

Five Prime Therapeutics Inc. (South San Francisco)

FPA008

Immunotherapy combination of cabiralizumab, a monoclonal antibody that inhibits colony stimulating factor-1 receptor, with PD-1 inhibitor Opdivo (nivolumab, Bristol-Myers Squibb Co.)

Tumors

Started the phase Ib portion of the trial

10/5/16

Innate Pharma SA (Marseilles, France)

IPH4102

An anti-KIR3DL2 humanized therapeutic antibody

Relapsed/refractory cutaneous T-cell lymphomas

Phase I data from the first seven dose levels (16 patients) showed that IPH4102 was well-tolerated, with no dose-limiting toxicity reported; as of Sept. 10, the best global response rate was 38% across all dosage levels; complete responses appeared with increasing doses and/or duration of exposure in skin and blood (respectively two and three, seen in four patients)

10/27/16

Karus Therapeutics Ltd. (Oxford, U.K.)

KA2237

A dual-selective inhibitor of P13K isoforms p110beta and p110delta

Relapsed, treatment-resistant B-cell lymphoma

First patients were dosed in a phase I study

10/6/16

Kite Pharma Inc. (Santa Monica, Calif.)

KTE-C19

Chimeric antigen receptor

Refractory diffuse large B-cell lymphoma

The first patient was enrolled in ZUMA-6, a phase Ib/II study of KTE-C19 in combination with the anti-PD-L1 cancer immunotherapy Tecentriq (atezolizumab, Genentech Inc./ Roche AG)

10/7/16

Madison Vaccines Inc. (Madison, Wis.)

MVI-118

Targets the human androgen receptor; vaccine

Prostate cancer

Expanded its phase I trial to a second site

10/28/16

Morphosys AG (Martinsried, Germany)

MOR202

Anti-CD38 candidate

Relapsed/refractory multiple myeloma

Seven out of nine patients treated with MOR202 in combination with lenalidomide plus dexamethasone during an ongoing phase I/IIa trial showed a complete or partial response to the treatment; in the patient group treated with MOR202 plus pomalidomide, three out of five patients showed an objective response, with two patients achieving a complete remission; median time to response was six weeks with most responses deepening over time

10/18/16

Oncbiomune Pharmaceuticals Inc. (Baton Rouge)

Proscavax

Vaccine

Prostate cancer

Reported that 16 patients are now enrolled in a phase Ia trial, 15 of whom have received at least one vaccine and 14 of whom have received all mandated vaccines; none of the patients have experienced a dose-limiting adverse event

10/10/16

Pharmaengine Inc. (Taipei, Taiwan)

PEP503

NBTXR3, a nanoparticle formulation of hafnium oxide crystals

Head and neck squamous cell carcinoma

The first patient was dosed in a phase Ib/II trial

10/20/16

Regen Biopharma Inc. (San Diego)

Hemaxellerate

Comprises cells extracted from the patient's own fat tissue

Chemotherapy-induced bone marrow suppression

Final clinical data on the first five patients treated with Hemaxellerate showed that none of the patients experienced any adverse events; at one month post-Hemaxellerate treatment, two patients (40%) had a dramatic increase in their circulating white blood cells to levels even above the normal range and all other patients had their white blood cells return to the normal range

10/25/16

Targovax Inc. (Oslo, Norway)

ONCOS-102

Human serotype 5 adenovirus optimized to induce systemic anti-tumor T cell responses 

Malignant mesothelioma

Clinical data from a recently completed phase I study showed how ONCOS-102 was able to immune activate patients with treatment-refractory malignant mesothelioma at a lesional level by performing both baseline and follow-up biopsies; the immune monitoring results showed an increase in tumor-infiltrating lymphocytes, pro-inflammatory cytokines and chemokines+G693

10/24/16

TG Therapeutics Inc. (New York)

TGR-1202

Oral PI3K delta inhibitor

Relapsed or refractory lymphoma

Launched a phase I/II study, in combination with proteasome inhibitor Kyprolis (carfilzomib, Amgen Inc.)

10/21/16

Tocagen Inc. (San Diego)

Toca 511 and Toca FC

Vocimagene amiretrorepvec and extended-release 5-fluorocytosine

Recurrent glioblastoma or anaplastic astrocytoma

Updated response and survival data came from a phase I, ascending-dose study of Toca 511 administered at the time of tumor removal followed by cycles of orally administered Toca FC; of the 24 evaluable patients, a median overall survival of 14.3 months was shown; the overall response rate was 20.8% with three complete responses and two partial responses

10/6/16

University of Texas MD Anderson Cancer Center's Institute for Applied Cancer Science

IACS-10759

A novel OXPHOS inhibitor

Acute myeloid leukemia

Initiated a phase I study

10/31/16

CARDIOVASCULAR

Biomarin Pharmaceutical Inc. (San Rafael, Calif.)

BMN 270

A gene therapy treatment

Severe hemophilia A

U.K.'s Medicines and Healthcare Products Regulatory Agency (MHRA) approved continued enrollment into the open-label phase I/II study of BMN 270; dosing had been suspended after enrolling the first nine patients in the study, due to observed increases in alanine aminotransferase levels that exceeded a prespecified threshold set by the company

10/17/16

Global Blood Therapeutics Inc. (South San Francisco)

GBT440

Oral, once-daily therapy

Sickle cell disease

Phase I/II data showed a rapid and durable reduction in hemolytic anemia and sickling over 90 days

10/10/16

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

PCSK9si

PCSK9 synthesis inhibitor

High cholesterol

Top-line results from the interim analysis with day 90 follow-up for all 501 patients enrolled in the ongoing ORION-1 study confirmed the significant and durable LDL-C reduction demonstrated up to day 90 in the preceding phase I study; PCSK9si was well-tolerated and no material safety issue was observed in the day 90 interim analysis of unblinded safety data, including no investigational drug-related elevation of liver enzymes, neuropathy or change in renal function

10/19/16

Theravance Biopharma Inc. (Dublin)

TD-0714

Neprilysin inhibitor

Major cardiovascular and renal diseases

Phase I data from a multiple ascending-dose trial showed the biological effect was demonstrated by dose-related increases in the levels of cyclic GMP (a well-precedented biomarker of NEP target engagement); the 50-mg, 100-mg and 200-mg doses (including the elderly cohort) produced maximal increases, while 10 mg was submaximal; the trial also confirmed the low levels of renal elimination for TD-0714 reported in the single ascending-dose study, which showed that less than 1% of the total TD-0714 dose was eliminated through the kidneys

10/26/16

CENTRAL NERVOUS SYSTEM

Atyr Pharma Inc. (San Diego)

Resolaris

First-in-class intravenous protein therapeutic

Rare myopathies

Data from the phase Ib/II trial reaffirm encouraging trends in safety, tolerability and pharmacokinetics

10/7/16

C2N Diagnostics LLC (St. Louis)

CZN-8E12

ABBV-8E12; an anti-tau antibody

Progressive supranuclear palsy

Completed the final follow-up visit of the last subject enrolled into its phase I, which enrolled 30 subjects

10/14/16

Diamedica Inc. (Minneapolis)

DM199

a recombinant tissue kallikrein protein

Neurological and kidney disease

The first patient was dosed in the phase Ib trial

10/6/16

Kempharm Inc. (Coralville, Iowa)

KP201/IR

Immediate-release abuse-deterrent hydrocodone product candidate with an acetaminophen-free formulation

Pain

Filed an IND with the FDA to begin human trials

10/26/16

Marinus Pharmaceuticals Inc. (Radnor, Pa.)

Ganaxolone

Intravenous CNS-selective GABAA modulator

Status epilepticus and other indications

A phase I dose-escalation study showed it achieved dose levels targeted for efficacy in patients with status epilepticus and other indications

10/20/16

Upsher-Smith Laboratories Inc. (Maple Grove, Minn.)

USL261

Midazolam nasal spray

Epilepsy

Data from a phase I study demonstrated that USL261, administered as single doses of 1.25 mg, 2.5 mg or 5 mg, was rapidly absorbed (median Tmax of approximately 15 minutes) and no dose-dependent differences in maximum plasma concentration were observed

10/31/16

DIABETES

Adocia SA (Lyon, France)

Hinsbet U100

A rapid-acting formulation of recombinant human insulin using Adocia's Biochaperone technology

Type I diabetes

Top-line results from a phase Ib trial showed it met its primary endpoint; Hinsbet treatment resulted in lower blood glucose one hour after the meal compared to Humulin treatment (BG1h=228 mg/dL with Hinsbet vs. 253 mg/dL with Humulin, LSM ratio, p=0.0002); secondary endpoints included the comparison of post-meal glycemic control over the first hour after the meal, with no significant difference found between Hinsbet and Humalog (p=0.5373); Hinsbet significantly reduced postprandial glucose excursion over the first hour compared to Humulin (p=0.0002)

10/28/16

Midatech plc (Abingdon, U.K.)

MTX102

Vaccine using its gold nanoparticle technology

Type 1 diabetes

Started a phase I trial that will involve eight subjects, ages 16 to 40, with type 1 diabetes and residual endogenous insulin production and will examine the general safety and tolerability of treatment

10/3/16

GASTROINTESTINAL

Theravance Biopharma Inc. (Dublin)

TD-1471

Orally administered and intestinally restricted pan-JAK inhibitor

Moderate to severe ulcerative colitis

Dosed the first patient in a phase Ib trial of TD-1473

10/5/16

INFECTIONS

Ampliphi Biosciences Corp. (San Diego)

AB-SA01

Phage cocktail

Staphylococcus aureus infections in patients with chronic rhinosinusitis

Enrollment in the trial has been completed in the phase I trial, and the safety monitoring committee determined that it was well-tolerated by all nine patients and that there were no drug-related serious adverse events

10/26/16

Entasis Therapeutics Inc. (Waltham, Mass.)

ETX2514

A next-generation beta-lactamase inhibitor

Acinetobacter baumannii infection

Started a phase I trial

10/27/16

Eurocrine Vaccines AB (Solna, Sweden)

Immunose Flu

A nose-drop formulation based on the Endocine technology and inactivated split antigens

Influenza

Started a phase I/II trial

10/27/16

Geovax Labs Inc. (Atlanta)

HIV vaccine

Adjuvanted DNA/MVA vaccine that co-expresses GM-CSF in the priming DNA vaccine

HIV

Phase I data showed it was well-tolerated and induced durable functional antibodies; data indicated the DNA/MVA prime-boost regimen induced functional humoral responses that included antibody-dependent cellular cytotoxicity, antibody avidity and Env IgG1- and IgG3-binding responses with specificities in the gp41 subunit of Env known to mediate virus capture and phagocytosis

10/21/16

Helocyte Inc. (New York) and City of Hope

Triplex

Vaccine

Cytomegalovirus in allogeneic hematopoietic stem cell transplant and solid organ transplant

Data from the phase I trial showed that Triplex was safe, well-tolerated and highly immunogenic when administered to healthy volunteers at multiple dose levels

10/31/16

Immunovaccine Inc. (Halifax, Nova Scotia)

DPX-RSV

Depovax-based, small B-cell epitope peptide vaccine candidate

Respiratory syncytial virus

Phase I data from six months or longer after vaccination confirmed earlier interim data on the ability of Depovax-formulated antigens to generate a relevant, durable immune response; the vaccine had a positive safety profile and was well-tolerated with no serious adverse events, and antigen-specific immune responses were detected at least six months after the last vaccination in 93% (15/16) of patients receiving DPX-RSV, in both low-dose and high-dose cohorts

10/14/16

Pharmajet Inc. (Golden, Colo.) and Vaccibody AS (Oslo, Norway)

VB10.16

Vaccine

Human papillomavirus

Phase I data showed the vaccine appears safe and well-tolerated, with a T-cell immune response indicating that it induces strong HPV16-specific tumor response

10/26/16

Rational Vaccines Inc. (Springfield, Ill.)

Theravax

HSV-2 vaccine, a live-attenuated vaccine

Herpes simplex virus 1 and 2

Results from a phase I trial showed it met its primary goal of establishing the safety and tolerability in patients who suffer with recurrent genital herpes caused by HSV-1 or HSV-2; all 17 participants who received the three-shot vaccination series indicated Theravax HSV-2 was more effective in reducing their genital herpes symptoms than antiviral drugs

10/18/16

Transgene SA (Strasbourg, France)

TG1050

Immunotherapy candidate

Chronic hepatitis B virus infection

The first patient was included in the multiple-dose cohort of the phase I/Ib trial; the+G682 continuation follows the positive recommendation of the safety review committee as no severe adverse events have been observed in patients receiving a single dose of TG1050

10/19/16

MISCELLANEOUS

Abeona Therapeutics Inc. (New York)

ABO-102

AAV-SGSH

Sanfilippo syndrome type A

Data safety monitoring board reviewed the initial safety data from the low-dose cohort (n=3) in the phase I/II trial of ABO-102 and authorized that the trial proceed with enrollment and dose escalation for the second cohort

10/6/16

Abeona Therapeutics Inc. (New York)

ABO-102

AAV gene therapy

MPS IIIA (Sanfilippo syndrome type A)

Updated results from an ongoing phase I/II trial showed the therapy was well-tolerated at the low dose; a significant average GAG (heparan sulfate) reduction in urine (57.6% +/- 8.2%) and cerebral spinal fluid (25.6% +/- 0.8%) and reduction in liver (17.1% +/- 1.9%) and spleen volume (17.6% +/- 7.1%) all were observed at 30 days post-intravenous injection

10/21/16

Alphamab Co. Ltd. (Suzhou, China)

KN015

A long-acting human follicle-stimulating hormone

Infertility

Is starting a phase I trial in China

10/25/16

Applied Genetic Technologies Corp. (Gainesville, Fla.)

Gene therapy

Adeno-associated virus (AAV)-based gene therapy

Achromatopsia caused by mutations in the CNGA3 gene

Filed an IND to conduct a phase I/II trial

10/21/16

Avexis Inc. (Chicago)

AVXS-101

An SMA gene therapy

Spinal muscular atrophy

Interim data from their ongoing phase I trial showed that two-thirds of patients in cohort 2 (the proposed therapeutic dose) had achieved the ability to sit unassisted, including one patient whose achievement of this milestone was confirmed after Sept. 15

10/11/16

Bioblast Pharma Ltd. (Tel Aviv, Israel)

Trehalose

Intravenous, 90 mg/mL; a low-molecular-weight disaccharide

Oculopharyngeal muscular dystrophy

Results from a double-blind, placebo-controlled pharmacokinetic study in healthy volunteers demonstrated a maximum tolerated dose of 54 g administered via I.V. over 60 minutes

10/25/16

Catabasis Pharmaceuticals Inc. (Cambridge, Mass.)

Edasalonexent

CAT-1004

Duchenne muscular dystrophy (DMD)

Data from part A of the MoveDMD trial of trial of edasalonexent showed that, after one week of dosing in boys, ages 4 to 7, a pre-selected NF-kB gene set was significantly inhibited in whole blood mRNA at each of the two higher doses (67 mg/kg and 100 mg/kg)

10/7/16

Chemocentryx Inc. (Mountain View, Calif.)

CCX168

Oral, complement 5a; newly designated avacopan

C3 glomerulopathy

After one month of initial treatment with avacopan, renal function (based on estimated glomerular filtration rate) stabilized; sequential kidney biopsies taken after the patient had been on avacopan for two and seven months showed continued improvement in kidney histology based on a decrease in glomerular endocapillary proliferation and a marked reduction in the number of glomerular inflammatory macrophages, as compared to the pre-treatment biopsy

10/28/16

Panoptica Inc. (Bernardsville, N.J.)

PAN-90806

A topical anti-vascular endothelial growth factor eye drop

Neovascular eye disease

Confirmed positive biological response to topical PAN-90806 in about 45% to 50% of treated patients, including outcomes such as vascular leakage, lesion morphology and vision; the company plans to initiate a phase I/II trial of a next-generation formulation in patients with neovascular age-related macular degeneration in 2017

10/18/16

Radiomedix Inc. (Houston)

Aminomedix

Proprietary formulation

Kidney radioprotection during peptide receptor radionuclide therapy (PRRT)

Completed its phase I/II trial; data confirmed the correct dose of the components of Aminomedix and also indicated both safety and effectiveness of the composition in reducing the kidney radiation absorbed dose while preventing clinically significant hyperkalemia, nausea and vomiting, during PRRT

10/21/16

Rockwell Medical Inc. (Wixom, Mich.)

Triferic

Anemia therapy, intravenous, to replace iron and maintain hemoglobin

Anemia

Data from its phase I healthy volunteer intravenous pharmacokinetic study demonstrated that Triferic iron is rapidly bound to transferrin and rapidly cleared from the circulation, with a half-life of between 1.2 and two hours

10/5/16

RESPIRATORY

Celtaxys Inc. (Atlanta)

Acebilustat

A once-daily oral anti-inflammatory drug

Cystic fibrosis

Phase I data support phase II development of oral acebilustat in once-daily doses of 50 mg and 100 mg, demonstrating a rapid and sustained effect on modulating leukotriene B4, the pharmacologic target of the drug, a leukotriene A4 hydrolase inhibitor

10/31/16

Concert Pharmaceuticals Inc. (Lexington, Mass.)

CTP-656

A next-generation CFTR potentiator

Cystic fibrosis

Phase I food effect and multiple ascending-dose results of CTP-656 showed that the exposure of CTP-656 was the same regardless of the fat content of a meal, and the safety, tolerability and pharmacokinetic profile of CTP-656 observed to date supports its development as a once-daily CFTR potentiator

10/28/16

Spyryx Biosciences Inc. (Durham, N.C.)

SPX-101

Inhaled SPLUNC1-derived peptide

Cystic fibrosis

Data showed it was found to be safe and well-tolerated in a completed phase I single-dose and ongoing multiple-dose study supported by a GLP toxicology package

10/26/16


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.

 

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