Anaptysbio Inc. (San Diego)


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


Apitope Ltd. (Hasselt, Belgium)


Peptide therapy

Graves' disease

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


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



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


Coherus Biosciences Inc. (Redwood City, Calif.)


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


Exicure Inc. (Skokie, Ill.)


A spherical nucleic acid

Mild to moderate psoriasis

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



Adaptimmune Therapeutics plc (Oxford, U.K.)


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


Adaptimmune Therapeutics plc (Oxford, U.K.)


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


Advaxis Inc. (Princeton, N.J.)


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


Amcure GmbH (Eggenstein-Leopoldshafen, Germany)


Targets CD44v6


Treated the first patient in a phase I/Ib study


Amgen Inc. (Thousand Oaks, Calif.)



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


Aptose Biosciences Inc. (San Diego)


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


Beigene Ltd. (Beijing)


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


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


Checkpoint Therapeutics Inc. (New York)


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

Advanced solid tumors

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


Daiichi Sankyo Co. Ltd. (Tokyo)


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


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


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


Five Prime Therapeutics Inc. (South San Francisco)


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


Started the phase Ib portion of the trial


Innate Pharma SA (Marseilles, France)


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)


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


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


Kite Pharma Inc. (Santa Monica, Calif.)


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)


Madison Vaccines Inc. (Madison, Wis.)


Targets the human androgen receptor; vaccine

Prostate cancer

Expanded its phase I trial to a second site


Morphosys AG (Martinsried, Germany)


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


Oncbiomune Pharmaceuticals Inc. (Baton Rouge)



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


Pharmaengine Inc. (Taipei, Taiwan)


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


Regen Biopharma Inc. (San Diego)


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


Targovax Inc. (Oslo, Norway)


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


TG Therapeutics Inc. (New York)


Oral PI3K delta inhibitor

Relapsed or refractory lymphoma

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


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


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


A novel OXPHOS inhibitor

Acute myeloid leukemia

Initiated a phase I study



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


Global Blood Therapeutics Inc. (South San Francisco)


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


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


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


Theravance Biopharma Inc. (Dublin)


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



Atyr Pharma Inc. (San Diego)


First-in-class intravenous protein therapeutic

Rare myopathies

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


C2N Diagnostics LLC (St. Louis)


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


Diamedica Inc. (Minneapolis)


a recombinant tissue kallikrein protein

Neurological and kidney disease

The first patient was dosed in the phase Ib trial


Kempharm Inc. (Coralville, Iowa)


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


Filed an IND with the FDA to begin human trials


Marinus Pharmaceuticals Inc. (Radnor, Pa.)


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


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


Midazolam nasal spray


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



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)


Midatech plc (Abingdon, U.K.)


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



Theravance Biopharma Inc. (Dublin)


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



Ampliphi Biosciences Corp. (San Diego)


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


Entasis Therapeutics Inc. (Waltham, Mass.)


A next-generation beta-lactamase inhibitor

Acinetobacter baumannii infection

Started a phase I trial


Eurocrine Vaccines AB (Solna, Sweden)

Immunose Flu

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


Started a phase I/II trial


Geovax Labs Inc. (Atlanta)

HIV vaccine

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


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


Helocyte Inc. (New York) and City of Hope



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


Immunovaccine Inc. (Halifax, Nova Scotia)


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


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



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


Rational Vaccines Inc. (Springfield, Ill.)


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


Transgene SA (Strasbourg, France)


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



Abeona Therapeutics Inc. (New York)



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


Abeona Therapeutics Inc. (New York)


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


Alphamab Co. Ltd. (Suzhou, China)


A long-acting human follicle-stimulating hormone


Is starting a phase I trial in China


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


Avexis Inc. (Chicago)


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


Bioblast Pharma Ltd. (Tel Aviv, Israel)


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


Catabasis Pharmaceuticals Inc. (Cambridge, Mass.)



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)


Chemocentryx Inc. (Mountain View, Calif.)


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


Panoptica Inc. (Bernardsville, N.J.)


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


Radiomedix Inc. (Houston)


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


Rockwell Medical Inc. (Wixom, Mich.)


Anemia therapy, intravenous, to replace iron and maintain hemoglobin


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



Celtaxys Inc. (Atlanta)


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


Concert Pharmaceuticals Inc. (Lexington, Mass.)


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


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


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



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