Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Aurinia Pharmaceuticals Inc. (Victoria, British Columbia)

Voclosporin

Calcineurin inhibitor

Lupus nephritis

Top-line results from its phase IIb AURA-LV study showed that, at 48 weeks, the trial met the complete remission (CR) and partial remission (PR) endpoints, demonstrating statistically significantly greater CR and PR in patients in both low-dose and high-dose cohorts vs. the control group; no unexpected safety signals were observed and there were no additional deaths in the voclosporin-treated patients; however, there were three deaths and one malignancy reported in the control arm after completion of the study treatment period

3/3/17

Aurinia Pharmaceuticals Inc. (Victoria, British Columbia)

Voclosporin

Calcineurin inhibitor

Lupus nephritis (LN)

The AURION (Aurinia Early Urinary Protein Reduction Predicts Response Study) study achieved its primary objective by demonstrating that early biomarker response in active LN patients can be a significant predictor of renal response at 24 and 48 weeks; in the per-protocol analysis at 48 weeks, 71% of subjects (n=5/7) remain in complete remission as measured by a urinary protein creatinine ratio (UPCR) of ≤ 0.5mg/mg, eGFR within 20% of baseline and concomitant steroid dose of <5mg/day; a 25% reduction in UPCR at week eight was found to be highly predictive of achieving renal response at 24 and 48 weeks

3/28/17

Chugai Pharmaceutical Co. Ltd. (Tokyo)

Nemolizumab

Anti-interleukin-31 receptor A antibody

Atopic dermatitis

Data from its global phase II XCIMA study showed it resulted in significant improvement of the change in pruritus visual analogue scale at 12 weeks compared to placebo, hitting the primary endpoint; patients in the nemolizumab group had a score of -43.7% for the 0.1 mg/kg dose, -59.8% for the 0.5 mg/kg dose and -63.1% for the 2 mg/kg dose vs. 20.9% for placebo (p < 0.01 for all comparisons)

3/3/17

Galectin Therapeutics Inc. (Norcross, Ga.)

GR-MD-02

Galectin-3 inhibitor

Moderate to severe plaque psoriasis

Results from an exploratory phase IIa trial showed no serious adverse events, and that GR-MD-02 achieved an average PASI (Psoriasis Area and Severity Index) reduction of greater than 50% in all patients that participated in the trial; one patient exceeded the primary endpoint with an 80% reduction in PASI 30 days after the last infusion, while the other four patients reached PASI-50 by their 10th infusion

3/7/17

Leo Pharma A/S (Ballerup, Denmark)

Tralokinumab

Monoclonal antibody targeting cytokine IL-13

Moderate to severe atopic dermatitis

Results from a phase IIb dose-ranging efficacy and safety study in adults showed it demonstrated efficacy in the primary and key secondary endpoints; co-primary endpoints were change from baseline in Eczema Area Severity Index (EASI) and percentage of patients with clear or almost clear Investigator's Global Assessment (IGA 0/1) at week 12; after treatment with tralokinumab for 12 weeks, 150 mg and 300 mg tralokinumab significantly reduced total EASI from baseline (adjusted mean difference of -4.4, p=0.027 and -4.9, p=0.011, respectively) compared with placebo; the number of patients achieving EASI 50 at week 12 in the tralokinumab 300-mg group was significantly higher compared with placebo (73.4% vs. 51.9%, p=0.025)

3/6/17

Metriopharm AG (Zurich, Switzerland)

MP-1032

Immune modulator

Moderate to severe plaque psoriasis

Top-line results from its phase IIa trial showed that adverse events were mild and moderate, occurring in both the placebo and treatment groups, and consisted frequently of common cold, headache and itching; exploratory analyses regarding the potential effect of the drug on PASI scores suggest that patients with MP1032 exposure levels above 120 ng*hr/ml (n=16) have a median reduction from baseline in PASI score of 25% as compared to 12% in the placebo group at the end of treatment (week six)

3/15/17

Pharmalink AB (Stockholm)

Nefecon

Targeted-release formulation of the corticosteroid budesonide

Primary IgA nephropathy

Phase IIb results showed it met its primary endpoint and at nine months, mean urine protein creatinine ratio (UPCR) decreased by -26.4% with Nefecon [p=0.0066] (-29.3% with 16 mg/day [p=0.009], non-significant -23.7% with 8 mg/day [p=0.029]) vs. placebo; that effect was sustained throughout follow-up; mean UPCR decreased by -32% from baseline at 12 months for 16 mg/day vs. a 0.5% increase for placebo

3/30/17

TG Therapeutics Inc. (New York)

Ublituximab

Glycoengineered anti-CD20 monoclonal antibody

Relapsing multiple sclerosis

Preliminary results of a phase II study showed it demonstrated rapid and robust B-cell depletion

3/6/17

Xenon Pharmaceuticals Inc. (Burnaby, British Columbia)

XEN-801

Topical stearoyl Co-A desaturase-1 inhibitor

Moderate to severe facial acne

Is dropping development after it failed to separate from placebo in clearing lesions over the course of a 12-week phase II study; top-line efficacy results showed it reduced total inflammatory and non-inflammatory lesion counts by about 40%, the same as a placebo vehicle; key secondary endpoints, including lesion counts and change in investigator global assessment, were also the same between the treatment and placebo arms

3/27/17

CANCER

Advaxis Inc. (Princeton, N.J.)

Axalimogene filolisbac

Derived from Advaxis' Lm-based Antigen Delivery System

Persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix (PRmCC)

Results from the phase II GOG-0265 study demonstrated that 38% of patients (n = 19/50) with heavily pretreated PRmCC were alive 12 months following treatment with the drug; the expected 12-month survival rate of patients enrolled in the study was calculated to be 24.5%, which means drug-treated patients showed a 52% improvement over the expected survival rate and is the highest 12-month survival rate achieved to date in this setting

3/16/17

Amgen Inc. (Thousand Oaks, Calif.)

Blincyto

Blinatumomab; bispecific CD19-directed CD3 T-cell engager antibody construct

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

Results from the phase II open-label ALCANTARA study in patients who had failed at least one second-generation or later tyrosine kinase inhibitor showed that 16 patients (36%, 95% CI, 22-51%) achieved complete remission or complete remission with partial hematologic recovery within the first two cycles of treatment, with 14 of the 16 (31%, 96% CI, 18-47%) patients achieving complete remission with full hematologic recovery

3/31/17

Biolinerx Ltd. (Tel Aviv, Israel)

BL-8040

Short peptide

Acute myeloid leukemia, solid tumors and hematological indications

Partial results from an open-label phase II trial showed that all recipients transplanted so far have experienced a successful neutrophil engraftment, and BL-8040 treatment appeared to be safe and well-tolerated in donors

3/21/17

Biothera Pharmaceuticals Inc. (Eagan, Minn.)

Imprime PGG

Immunotherapy

Metastatic non-small-cell lung cancer

Results of its phase II study testing it in combination with Erbitux (cetuximab, Eli Lilly and Co.) and chemotherapy showed it was well-tolerated, with adverse events expected of the backbone therapy predominating; a retrospective analysis of the study suggests that the subset of patients with sufficient levels of anti-beta glucan antibody may achieve improved clinical outcomes

3/27/17

Cellceutix Corp. (Beverly, Mass.)

Kevetrin

Small-molecule inducer of p53

Solid tumors

Based on positive results from the phase I trial, the company is moving forward with a phase II trial of intravenously administered Kevetrin in patients with late-stage, platinum-resistant ovarian cancer

3/6/17

Cellceutix Corp. (Beverly, Mass.)

Brilacidin

Small-molecule antibiotic

Oral mucositis in patients with head and neck cancer

Results from its ongoing phase II study showed that, in a preliminary interim analysis, a marked reduction in incidence of Severe OM (WHO grade > 3) was observed in patients treated with Brilacidin who received at least 55 Gy cumulative units of radiation; incidence of severe OM was 22.2% in the Brilacidin arm vs. 70% in the control arm

3/28/17

Cellectar Biosciences Inc. (Madison, Wis.)

CLR-131

Phospholipid-drug conjugate

Multiple myeloma and other hematologic malignancies

Started a phase II trial

3/31/17

Clovis Oncology Inc. (Boulder, Colo.)

Rucaparib

Oral, potent, small-molecule poly (ADP-ribose) polymerase (PARP) inhibitor

Ovarian cancer

Reported that, among the 134 patients with a germline or somatic BRCA mutation enrolled in its ongoing phase II Ariel2 trial, the objective response rate (ORR), disease control rate (DCR) and median progression-free survival (PFS) in patients with a BRCA mutation were greatest in platinum-sensitive patients, followed in descending order by those who were platinum-resistant, and those who were platinum-refractory

3/14/17

Erytech Pharma SA (Paris)

Graspa

Eryaspase

Pancreatic cancer

Phase IIb results showed a 43% reduction in death risk; it met its co-primary endpoints, yielding significant improvement in progression-free survival (PFS) and overall survival (OS) in patients given the Graspa-plus-chemotherapy regimen vs. chemo alone

3/28/17

Genmab A/S (Copenhagen)

Daratumumab

Human IgG1k monoclonal antibody designed to bind with high affinity to the CD38 molecule

Three types of relapsed or refractory non-Hodgkin's lymphoma

Said partner Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson, decided not to initiate stage two of the phase II study; a review of data showed that two cohorts of the study did not reach the predefined futility thresholds of overall response rates (ORR) of 50% and 30%, respectively

3/31/17

Glycomimetics Inc. (Rockville, Md.)

GMI-1271

An E-selectin antagonist drug candidate

Acute myeloid leukemia

The first of two patient cohorts in its phase II trial completed enrollment; two arms combined will enroll a total of about 90 patients

3/8/17

Heat Biologics Inc. (Durham, N.C.)

HS-110

Viagenpumatucel-L

Non-small-cell lung cancer

Phase II data of HS-110 in combination with New York-based Bristol-Myers Squibb Co.'s Opdivo (nivolumab) showed a strong correlation between T-cell activation, tumor reductions and increased overall survival; immune responses to HS-110 were observed in all five patients that exhibited tumor reductions

3/22/17

Ignyta Inc. (San Diego)

Entrectinib

CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1 or ALK fusions

Brain tumors

Data showed that treatment in a patient with a primary brain tumor harboring an NTRK1 fusion resulted in a 60% regression in tumor size, and the resolution of clinical symptoms was maintained for 11 months on treatment

3/31/17

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

Sacituzumab govitecan (IMMU-132)

Antibody-drug conjugate

Metastatic triple-negative breast cancer

Phase II results showed the overall confirmed objective response rate was 30%, with two patients having complete remissions and 19 achieving partial responses (95% confidence interval [CI], 20 to 43%); median progression-free survival was six months (95% CI, 5 to 7.3 months) vs. the 3.5 month generally attributed to standard agents; the median overall survival during the trial was 16.6 months (95% CI, 11.1 to 20.6 months); grade 3 or higher adverse events included neutropenia (39%), leukopenia (16%), anemia (14%) and diarrhea (13%)

3/16/17

Juno Therapeutics Inc. (Seattle)

JCAR-015

CAR T-cell therapy

Acute lymphoblastic leukemia

Said it will not move forward with the phase II ROCKET trial following reports of a greater than expected incidence of severe neurotoxicity, along with five deaths from cerebral edema, in adult patients with relapsed/refractory ALL treated in the study, which was placed on hold for a second time in November

3/3/17

Karyopharm Therapeutics Inc. (Newton, Mass.)

Lowered-dose selinexor

First in class SINE XPO1 antagonist

Acute myeloid leukemia (AML)

The results of a planned interim analysis of the phase II SOPRA study evaluating single-agent selinexor in relapsed/refractory AML showed the test will not reach statistical significance for overall survival (OS), the study's primary endpoint; selinexor-treated patients who achieved a complete response did exhibit a substantial OS benefit as compared with the physician's choice (PC) arm, and the data safety monitoring board agreed that patients would be permitted to continue on the selinexor arm or the PC arm, as applicable, following discussion between the patient and his or her treating physician

3/3/17

Karyopharm Therapeutics Inc. (Newton, Mass.)

Selinexor (KPT-330)

Oral selective inhibitor of nuclear export compound

Cancer

Received written notice from the FDA that its clinical trials were placed on partial clinical hold, during which patients with stable disease or better may remain on therapy but no new patients may be enrolled. The FDA indicated the hold is due to incomplete information in the existing version of the investigator's brochure (IB); Karyopharm said it has amended the IB and submitted requested documents and said the hold is

not the result of any patient death or new information regarding the safety profile

3/14/17

Kite Pharma Inc. (Santa Monica, Calif.)

Axicabtagene ciloleucel

Chimeric antigen receptor T-cell candidate; previously KTE-C19, now called axi-cel

Chemorefractory aggressive B-cell non-Hodgkin lymphoma

Hit the primary endpoint in the pivotal trial, the phase II ZUMA-1 trial; ZUMA-1 succeeded on the main goal: objective response rate, or rates of tumor response (complete response plus partial response recorded after a single infusion of axi-cel, with a score of 82% (p < 0.0001)

3/1/17

Kura Oncology Inc. (La Jolla, Calif.)

Tipifarnib

Selective inhibitor of farnesyl transferase

HRAS mutant squamous cell carcinomas of the head and neck (SCCHN)

Updated results in the first stage of the second cohort of the phase II trial showed that, as of Feb. 28, two patients with HRAS mutant SCCHN with objective responses remain on study and are currently at treatment cycle 19 and cycle 12; among five patients with HRAS mutant salivary gland cancer, no objective responses were observed but three patients experienced disease stabilization and were on study for nine, 10 and 14 cycles

3/7/17

Nanobiotix SA (Paris)

NBTXR3

Aqueous suspension of nanoparticles

Soft tissue sarcoma

The independent data monitoring committee has completed the interim evaluation of the phase II/III trial and recommended the continuation of the trial

3/24/17

Progenics Pharmaceuticals Inc. (New York)

1404

PSMA-targeted small-molecule SPECT/CT imaging agent

To detect prostate cancer

Results of a phase II study demonstrated the sensitivity of 1404 to detect prostate cancer using both visual and semiquantitative tumor to background (TBR) scores; data showed the uptake within the prostate gland evaluated by both visual and TBR scores correlated significantly with Gleason Score

3/27/17

Progenics Pharmaceuticals Inc. (New York)

Azedra injection

Iobenguane I 131

Malignant and/or recurrent pheochromocytoma or paraganglioma, rare neuroendocrine tumors

The registrational phase IIb trial achieved its primary endpoint; data showed that 17 (25%) of the 68 evaluable patients experienced a 50% or greater reduction of all antihypertensive medication for at least six months; the lower limit of the 95% confidence interval was 16.15%, thus meeting the primary endpoint

3/31/17

Sellas Life Sciences Group (Hamilton, Bermuda)

Galinpepimut-S

WT1 immunotherapeutic

Multiple myeloma (MM)

Updated phase II data indicate a meaningful clinical benefit among high-risk MM patients, showing an 88% actuarial overall survival (OS) at the 18-month landmark in 18 evaluable patients; all patients had evidence of minimal residual disease after autologous stem cell transplant (ASCT) and 15 had high-risk cytogenetics at diagnosis, characteristics that typically result in low progression-free survival (PFS) rates that do not exceed 12 months following ASCT; current median PFS is 23.6 months, while median OS has not been reached

3/2/17

Spectrum Pharmaceuticals Inc. (Henderson, Nev.)

Poziotinib

Oral pan-HER inhibitor that irreversibly blocks signaling through the EGFR

Non-small-cell lung cancer

Said the University of Texas MD Anderson Cancer Center started a phase II trial in patients with EGFR exon 20 insertion mutations

3/31/17

Transgene SA (Strasbourg, France)

TG-4010

Immunotherapy

Metastatic non-small-cell lung cancer (NSCLC)

Dosed the first patient in a phase II trial evaluating its experimental immunotherapy TG4010 in combination with Opdivo (nivolumab) for the treatment of NSCLC after failure of one line of platinum-based chemotherapy

3/14/17

WntResearch AB (Stockholm)

Foxy-5

Designed to inhibit tumors from spreading

Advanced colon, prostate or breast cancer

In the phase Ib study, gene expression analyses show that the second highest of four investigated dose levels resulted in the best biological effect in patient tumor tissue, and the company has determined the dose level for its upcoming phase II trial; safety appeared to be favorable and all tested dose levels were well tolerated

3/22/17

CARDIOVASCULAR

Akcea Therapeutics Inc. (Cambridge, Mass.; subsidiary of Ionis Pharmaceuticals Inc.)

AKCEA-APO(a)-LRx

Antisense candidate

Hyperlipopro-teinemia(a) and established cardiovascular disease

Started a phase IIb dose-ranging study

3/31/17

Arca Biopharma Inc. (Westminster, Colo.)

Gencaro

Bucindolol hydrochloride; beta blocker and mild vasodilator

Atrial fibrillation

The 175th patient has been randomized into GENETIC-AF, a seamless design phase IIb/III trial to compare the safety and efficacy of Gencaro to Toprol-XL (metoprolol succinate, Astrazeneca plc)

3/7/17

CSL Behring (King of Prussia, Pa.)

CSL-830

Self-administered, subcutaneous c1-esterase inhibitor human replacement therapy

Hereditary angioedema (HAE) attacks

Results from the pivotal phase III COMPACT study showed it met its primary efficacy endpoint, significantly reducing the time-normalized number of HAE attacks, and met its secondary endpoints, including the responder rate (patients who had at least a 50% reduction in their attack rate) and the number of rescue medication uses

3/24/17

Cytokinetics Inc. (South San Francisco)

Omecamtiv mecarbil

Activator of cardiac myosin

Heart failure

Further results from its phase II Cosmic-HF study showed it improved myocardial deformation, a marker of myocardial function that has been related to outcomes

3/21/17

Ergomed plc (London)

Peprostat

Synthetic peptide hemostat

Intraoperative surgical bleeding

Started the phase IIb trial

3/21/17

Esperion Therapeutics Inc. (Ann Arbor, Mich.)

Triplet oral therapy

Bempedoic acid, ezetimibe and atorvastatin

Hypercholes-terolemia

Started a phase II study

3/8/17

Fibrogen Inc. (San Francisco)

Roxadustat

Oral hypoxia-inducible factor prolyl hydroxylase inhibitor

Anemia in patients with chronic kidney disease

Results from two phase II studies in China showed that, in dialysis-dependent and in non-dialysis-dependent CKD patients, roxadustat corrected and maintained hemoglobin levels regardless of the patients' baseline iron repletion status and levels of C-reactive protein; in the first study, hemoglobin levels were increased over baseline by more than 1 gm/dL in a significantly greater proportion of patients receiving roxadustat than patients receiving placebo (80% and 87.1% for the low- and high-dose roxadustat arms, respectively, vs. 23.3% in the placebo arm; p<0.0001 for both)

3/31/17

Gemphire Therapeutics Inc. (Livonia, Mich.)

Gemcabene (CI-1027)

Oral therapy targeting known lipid metabolic pathways

High cholesterol

Reported a non-statistically significant 13% mean increase in glucose disposal rate (GDR) compared vs. a 6.8% increase for placebo during a phase II study of insulin sensitization and LDL-C lowering in non-diabetic, obese patients; gemcabene 900 mg lowered LDL-C by 40% (p<0.0001) and total cholesterol by 27% (p<0.0001)

3/22/17

The Medicines Co. (Parsippany, N.J.) and Alnylam Pharmaceuticals Inc. (Cambridge, Mass.)

Inclisiran

PCSK9 synthesis inhibitor

Hypercholes-terolemia

Final results from the ORION-1 phase II study showed it delivered significant and sustained reductions of LDL-C and high standards of safety and tolerability; all patients who received the two-dose starting regimen displayed a significant response and the mean LDL-C reductions over time were practically constant

3/21/17

CENTRAL NERVOUS SYSTEM

AB Science SA (Paris)

Masitinib

Tyrosine kinase inhibitor

Amyotrophic lateral sclerosis

The phase II/III study AB10015 has met its pre-specified primary endpoint, which was based on the change from baseline to week 48 in the revised Amyotrophic Lateral Sclerosis Functional Rating Scale

3/21/17

Adynxx Inc. (San Francisco)

AYX1

Synthetic dsDNA inhibitor of EGR1

Acute post-surgical pain

The first patient was dosed in a phase II study

3/2/17

Aevi Genomic Medicine Inc. (previously Medgenics Inc.; Philadelphia)

AEVI-001

Oral non-stimulant

Attention-deficit hyperactivity disorder (ADHD)

Top-line results showed it failed to achieve the primary endpoint of reduction on the ADHD rating scale (ADHD-RS) in the SAGA trial in adolescents with mGluR-mutation positive (mGluR+) ADHD; the trial showed improvement on the inattention subscale (p=0.0515); 70% of patients treated with AEVI-001 met this response definition compared to 42% on placebo (p=0.0067); in a second pre-specified responder analysis of the Clinical Global Impression of Improvement scale, 57% treated with AEVI-001 achieved a score of much improved or very much improved compared to 33% on placebo (p=0.0155)

3/21/17

Genervon Biopharma-ceuticals LLC (Pasadena, Calif.)

GM-6

Small peptide delivered by injection

Amyotrophic lateral sclerosis (ALS)

Phase IIa data showed favorable shifts in ALS biomarkers and improved clinical and functional measures in ALSFRS-R and FVC

3/24/17

Mesoblast Ltd. (Melbourne, Australia)

Allogeneic mesenchymal precursor cell therapy

Mesenchymal precursor cells (MPCs)

Chronic low back pain due to intervertebral disc degeneration

Final phase II data showed a single intra-discal injection of 6 million mesenchymal precursor cells (MPC-06-ID) resulted in meaningful improvements in both pain and function that were durable for at least 36 months

3/22/17

Neuroderm Ltd. (Rehovot, Israel)

ND0612H

24-hour regimen of continuous delivery levodopa/carbidopa (LD/CD)

Advanced Parkinson's disease

A preliminary analysis of trial 006, a phase II study, demonstrated that it successfully met its primary, key secondary and additional secondary endpoints

3/2/17

Neurotrope Inc. (New York)

Bryostatin-1

A PKC epsilon activator

Moderate to severe Alzheimer's dementia

Concluded dosing and patient monitoring in its phase II double-blind, placebo-controlled study; patients underwent 12-week treatment with bryostatin-1, followed by a 30-day post-treatment evaluation, and the study is designed to assess the therapeutic efficacy of bryostatin-1

3/1/17

Newron Pharmaceuticals SpA (Milan, Italy)

Evenamide

NW-3509; antipsychotic

Schizophrenia

Data from a phase II study evaluating it as an add-on to a stable dose of risperidone or aripiprazole, in patients showing return of symptoms, showed the combo would combine reduction of aberrant electrical activity and normalization of glutamate release with blockade of 5HT2/D2 receptors, thus producing a therapeutic option for treatment

3/17/17

Newron Pharmaceuticals SpA (Milan, Italy)

Evenamide (NW-3509)

Sodium channel blocker

Schizophrenia

Results of a phase IIa study indicated that the mean change from baseline at day 28 for the Positive and Negative Syndrome Scale (PANSS) total score was greater for evenamide [-5.1 (9.67)] than for placebo [-3.7 (9.65)]; for the PANSS Positive Symptoms subscale, a statistically significant/near significant improvement from baseline (mean baseline score: 14.8 ± 2.8) to day 28 for evenamide, compared to placebo, was noted in the ANCOVA-LOCF [-1.28 (0.632), p=0.046], ANCOVA-OC [-1.48 (0.641), p=0.024] and MMRM [-1.19 (0.643), p=0.068] analyses

3/28/17

Zynerba Pharmaceuticals Inc. (Devon, Pa.)

ZYN-002

Cannabidiol gel

Epilepsy

Completed enrollment in the phase II STAR 1 (Synthetic Transdermal Cannabidiol for the Treatment of Epilepsy) trial evaluating it in adult patients with refractory focal seizures

3/14/17

DIABETES

Origin Inc. (Princeton, N.J.)

Plasma-generated nitric oxide

Technology to produce and deliver therapeutic quantities of plasma-generated nitric oxide

Chronic diabetic foot ulcers

Treatment has begun on the first patients in its U.S. dose-ranging GENESIS trial, a phase IIb-equivalent test

3/23/17

Poxel SA (Lyon, France)

Imeglimin

Oral anti-diabetic agent

Diabetes

Completed a QT/QTc cardiac safety study; doses of the drug showed no effect on the QT/QTc interval at a 2,250 mg or 6,000 mg

3/22/17

GASTROINTESTINAL

Axim Biotechnologies Inc. (New York)

Canchew

Cannabidiol gum

Irritable bowel syndrome

Enrolled 40 patients and started its phase II trial

3/8/17

Cellceutix Corp. (Beverly, Mass.)

Brilacidin

A small-molecule antibiotic

Mild-to-moderate ulcerative colitis

Interim results in the first two cohorts of its ongoing phase IIa trial; at day 42, the primary efficacy endpoint of clinical remission (measured by stool frequency, rectal bleeding and endoscopy sub-scores), was met in half of the 12 patients evaluated; patient quality of life, as assessed by the Short Inflammatory Bowel Disease Questionnaire, was improved in all 12 patients after six weeks of daily brilacidin treatment; three of six patients in the third and final cohort have been enrolled

3/9/17; 3/22/17

Galapagos NV (Mechelen, Belgium)

Filgotinib

Oral Janus kinase-1 inhibitor

Small bowel Crohn's disease as well as in fistulizing Crohn's disease

Said its partner, Gilead Sciences Inc., of Foster City, Calif., will lead two new phase II studies

3/13/17

Qu Biologics Inc. (Vancouver, British Columbia)

Qbecco

Site Specific Immunomodulators (SSIs)

Crohn's disease (CD) and ulcerative colitis

Reported positive genetic analyses of the recently completed phase II studies; the analyses identified common inflammatory bowel disease-related genotypes with a high likelihood of response to SSIs

3/6/17

Qu Biologics Inc. (Vancouver, British Columbia)

Site Specific Immunomod-ulators (SSI) therapy

Stimulates an innate immune response in targeted organs or tissues to reverse the chronic inflammation

Crohn's disease

Further analysis of the phase II study identified immune factors (cytokines and growth factors) in the blood of patients with Crohn's disease that may predict response to SSI therapy; these biomarkers may identify patients likely to respond to SSI treatment; trial results demonstrated that specific blood immune markers associated with immune activation and mucosal healing increased with SSI treatment and SSI response/remission

3/24/17

INFECTION

Cytuvax B.V. (Maastricht, the Netherlands)

HBAI-20

Vaccine

Hepatitis B

Started a phase II study assessing the efficacy and safety

3/9/17

Hepatera LLC (Moscow)

Myrcludex B

Inhibits the essential HBV receptor on the liver cell surface

Hepatitis B virus (HBV)

Completed patient recruitment for phase IIb trials

3/23/17

Infectex Ltd. (Moscow; portfolio company of Maxwell Biotech Venture Fund)

SQ-109

Small molecule

Multidrug-resistant pulmonary tuberculosis

Results of a phase IIb/III trial showed both the intent-to-treat and per-protocol (PP) patients treated with SQ109-containing regimens showed statistically significant improvement in clearance of lung bacteria; the sputum culture conversion rate (cessation of Mycobacteria excretion) of PP patients treated with SQ109 plus the standard regimen was 80%, compared to patients treated with the standard regimen plus placebo (61%) by the end of six months of treatment

3/28/17

Matinas Biopharma Holdings Inc. (Bedminster, N.J.)

MAT-2203

Oral, encochleated formulation of the broad spectrum fungicide, amphotericin B

Chronic or recurrent mucocutaneous candidiasis

The institutional review board of the National Institute of Allergy and Infectious Diseases at the NIH granted approval for a six-month open-label safety extension of the phase IIa study

3/7/17

Seres Therapeutics Inc. (Cambridge, Mass.)

SER-109

Composed of about 50 species of firmicutes spores

Multiply recurrent Clostridium difficile infection

Disclosed plans to start a new phase II study, ECOSPOR III

3/17/17

Takeda Pharmaceutical Co. Ltd. (Osaka, Japan)

TAK-003 (also referred to as TDV)

Live-attenuated tetravalent dengue vaccine

Dengue virus

Data from a six-month interim analysis showed it elicited a broad antibody response against all four dengue virus types, regardless of previous exposure; the increased presence of antibodies in the blood against the four serotypes ranged between 87% to 100% by month one and was sustained at month six in both the one-dose and two-dose groups; in participants who were not previously exposed to dengue infection before vaccination, seropositivity rates against dengue virus types 3 and 4 were improved after a second dose of vaccine

3/31/17

Vaccibody AS (Oslo, Norway)

VB10.16

Immunotherapy

High-grade cervical intraepithelial neoplasia caused by human papillomavirus 16

Said inclusion of patients in the expansion phase (phase IIa) of its trial BV C-01 can be initiated, after clearance from German regulatory authorities

3/3/17

Viamet Pharmaceuticals Inc. (Research Triangle Park, N.C.)

VT-1161

Selective orally available inhibitor of fungal CYP51

Onychomycosis of the toenail

Results from RENOVATE (Restoring Nail: An Oral VT-1161 Tablet Evaluation), its phase IIb trial, showed VT-1161-treated patients met the primary endpoint of complete cure of the target toenail at 48 weeks at a rate that was highly statistically significant compared with patients in the placebo group; in the intent-to-treat analysis, complete cure rates were 0% in the placebo arm compared to a range of 32% to 42% in the four VT-1161 arms of the study, with all arms achieving statistical significance vs. placebo

3/7/17

INFLAMMATORY

Immune Pharmaceuticals Inc. (New York)

Bertilimumab

Human monoclonal antibody that binds to eotaxin-1

Bullous pemphigoid

Broadened enrollment criteria based on data from three patients who have completed treatment to date in its 10-patient, open-label study, which showed the Bullous Pemphigoid Disease Activity Index was reduced by an average of 84% and oral prednisone was tapered down to 10 mg or less

3/1/17

Zynerba Pharmaceuticals Inc. (Devon, Pa.)

ZYN-002

Cannabidiol gel

Osteoarthritis

Enrollment has completed in the phase II STOP (Synthetic Transdermal Cannabidiol for the Treatment of Knee Pain due to Osteoarthritis) trial

3/14/17

MISCELLANEOUS

Cara Therapeutics Inc. (Stamford, Conn.)

Intravenous CR-845

Peripherally-acting kappa opioid receptor agonist

Uremic pruritus

Top-line results from Part A of its phase II/III trial showing it met both primary and secondary endpoints for efficacy (reduced itching and improved quality of life, respectively); patients receiving I.V. CR845 experienced a 68% greater reduction from baseline in worst itch scores than those receiving placebo (p-value < 0.0019), and experienced a 100% greater reduction from baseline in the average total Skindex-10 score at week eight than those receiving placebo (p-value < 0.0007)

3/29/17

Castle Creek Pharmaceuticals Inc. (Parsippany, N.J.)

Topical diacerein 1%

Interleukin 1 inhibitor

Epidermolysis bullosa simplex

Results from a placebo-controlled clinical trial showed a 60% reduction in blistering among patients treated with diacerein 1% versus a 15% reduction in the placebo group at four weeks; at three months, 67% of patients in the placebo group returned to baseline blistering levels, versus 12.5% of patients in the diacerein 1% treatment group

3/7/17

Clementia Pharmaceuticals Inc. (Montreal)

Palovarotene

Retinoic acid receptor gamma agonist

Fibrodysplasia ossificans progressiva

Preliminary results of its phase II part A open-label extension (OLE) trial support the results obtained in the double-blind phase II study; when the data are combined across those two studies, palovarotene resulted in approximately a 50% reduction in occurrence of new heterotopic ossification (HO) as compared to placebo; and the volume of new HO was decreased by 70% for palovarotene-treated subjects as compared to the placebo subjects

3/29/17

Cytokinetics Inc. (South San Francisco)

CK-2127107

Fast skeletal troponin activator

Spinal muscular atrophy

Started enrollment in the second cohort of a phase II trial

3/30/17

Ixaltis SA (Toulouse, France)

IXA-001

Litoxetine

Mixed urinary incontinence

Is launching its first phase II study

3/16/17

Menlo Therapeutics Inc. (Menlo Park, Calif.)

Serlopitant

Once-daily, orally administered 5 mg tablets

Pruritus associated with prurigo nodularis

Said a phase II trial (TCP-102) met its primary efficacy endpoint and key secondary endpoints, demonstrating a statistically significant reduction in pruritus (p<0.001 for the primary efficacy analysis at week eight) in subjects treated with serlopitant compared with placebo

3/9/17

Mereo Biopharma Group plc (London)

BGS-649

A once-weekly oral aromatase inhibitor

Hypogonado-tropic hypogonadism

The independent data monitoring committee recommended the continuation of all three different dose arms of the phase IIb BSG-649 study following a prospectively defined interim analysis of safety and efficacy

3/8/17

Neothetics Inc. (San Diego)

LIPO-202

Injectable formulation of salmeterol xinafoate, a long-acting ß2-adrenergic receptor agonist

Reduction of submental subcutaneous fat (double chin)

Completed enrollment for its phase II proof-of-concept trial, LIPO-202-CL-31

3/21/17

Neuren Pharmaceuticals Ltd. (Melbourne, Australia)

Trofinetide

Synthetic analogue of a naturally occurring neurotrophic peptide derived from IGF-1.

Rett syndrome

Top-line results for its phase II trial in girls showed that the highest dose of trofinetide achieved statistically significant clinical benefit compared with placebo for each of three syndrome-specific efficacy measures, the Rett Syndrome Behaviour Questionnaire (p=0.042), the Clinical Global Impression of Improvement (p=0.029) and the Rett Syndrome Domain Specific Concerns (p=0.025)

3/28/17

Ocera Therapeutics Inc. (Palo Alto, Calif.)

OCR-002

Intravenous ornithine phenylacetate

Hepatic encephalopathy

Further analysis from the STOP-HE trial confirmed that OCR-002 demonstrated a highly statistically significant reduction in ammonia levels over placebo, p=0.028; it was safe and well-tolerated, and higher doses had a lower percentage of deaths and life threatening adverse events compared to placebo

3/9/17

Omeros Corp. (Seattle)

OMS-721

MASP-2-targeting drug

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HCT-TMA)

Additional data from the ongoing phase II trial showed statistically significant and clinically meaningful improvements in TMA disease activity were observed over the course of treatment, specifically in mean platelet count, mean LDH and mean haptoglobin; mean creatinine also improved but did not reach statistical significance

3/2/17

Opiant Pharmaceuticals Inc. (Santa Monica, Calif.)

OPNT-001

Nasally delivered opioid antagonist candidate

Bulimia nervosa

Initiated a phase II trial

3/21/17

Opthea Ltd. (Melbourne, Australia)

OPT-302

VEGF-C/D trap therapy

Wet age-related macular degeneration

Completed a type C meeting with the FDA aimed at obtaining regulatory guidance on the clinical development program and proposed phase IIb trial

3/10/17

Regimmune Corp. (Tokyo)

RGI-2001

Liposomal formulation of a synthetic derivative of alpha-galactosylceramide

Graft-vs.-host disease

Phase IIa data showed it was safe and well-tolerated and expanded Foxp3+ regulatory T cells (Treg) mediated by the activation of invariant natural killer T cells in patients

3/10/17

Ritter Pharmaceuticals Inc. (Los Angeles)

RP-G28

Highly purified galactooligosaccharide

Lactose intolerance

Top-line findings from its phase IIb/III trial show a clinically meaningful benefit to subjects in the reduction of symptoms; the primary endpoint, defined as the proportion of subjects who were abdominal symptom responders, met statistical significance, in which 40% of the pooled dosing group and 26% of the placebo group responded (p=0.0159)

3/30/17

Samumed LLC (San Diego)

SM0-4554

Topical solution of the small-molecule compound

Androgenetic alopecia

Safety and efficacy results from a phase II scalp biopsy study showed that, in the intent-to-treat analysis, both treatment groups exhibited higher total follicle counts compared to vehicle; statistically significantly higher numbers of hair follicles, compared with the vehicle group, were observed for both treatment groups at one or more time points

3/7/17

Saniona AB (Copenhagen)

Tesomet

Fixed-dosed combination of tesofensine and metoprolol

Prader-Willi syndrome

Regulatory agencies in Czech Republic and Hungary have approved Saniona's clinical trial applications for a phase IIa study

3/21/17

Tyrogenex Inc. (Rockville, Md.)

X-82 (vorolanib)

Orally administered inhibitor of VEGFR and PDGFR

Wet age-related macular degeneration

Completed patient enrollment in the phase II APEX study

3/10/17

Ultragenyx Pharmaceutical Inc. (Novato, Calif.)

UX-007

Synthetic seven carbon fatty acid triglyceride

Glucose transporter type-1 deficiency syndrome (Glut1 DS)

Topline data from the phase II study in Glut1 DS patients with seizures showed the study did not meet the primary endpoint of reducing the frequency of total number of observable and absence seizures among patients; patients treated with UX007 (n=25) demonstrated a reduction of 13.4% in overall seizure frequency (p=0.41) relative to placebo (n=11)

3/24/17

Versartis Inc. (Menlo Park, Calif.)

Somavaratan

VRS-317; long-acting form of recombinant human growth hormone

Growth hormone deficiency

A comparison of pharmacokinetic and pharmacodynamic data from its U.S. and Japanese phase II studies in pediatric subjects showed similar responses achieved at the same range of somavaratan doses in both populations; variability between the two patient sets was minor, with no effect on treatment outcomes

3/3/17

Vtesse Inc. (Gaithersburg, Md.)

VTS-270

Mixture of 2-hydroxypropyl-B-cyclodextrins

Niemann-Pick Type C1 disease

Its ongoing phase IIb/III registrational study is fully enrolled

3/16/17

RESPIRATORY

Corbus Pharmaceuticals Holdings Inc. (Norwood, Mass.)

Anabasum (JBT-101; Resunab)

Synthetic oral endocannabinoid-mimetic drug that preferentially binds to the CB2 receptor expressed on activated immune cells and fibroblasts

Cystic fibrosis

Top-line data from its phase II study showed it achieved the primary objective by demonstrating an acceptable safety and tolerability profile at all doses, with no serious or severe adverse events; patients in the highest dose cohort of anabasum (20 mg orally, twice per day) had a 75% reduction in the annualized rate of pulmonary exacerbations requiring I.V. antibiotics compared to placebo cohort

3/31/17


Notes

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The date indicated refers to the BioWorld Today issue in which the news item can be found.

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