Company |
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. |