Company |
Product |
Description |
Indication |
Status |
Date |
AUTOIMMUNE | |||||
Adamas Pharmaceuticals Inc. (Emeryville, Calif.) |
ADS-5102 |
Amantadine HCl extended-release capsules |
Multiple sclerosis |
Phase II proof-of-concept data suggest that ADS-5102 is well tolerated in the MS patient population and has a significant positive impact on walking speed; an approximately 15% placebo-adjusted improvement in walking speed was seen in the T25FW (p<0.05) |
6/14/16 |
Anthera Pharmaceuticals Inc. (Hayward, Calif.) |
Sollpura |
Liprotamase; pancreatic enzyme replacement therapy |
Exocrine pancreatic insufficiency and systemic lupus erythematosus |
The phase III SOLUTION study completed screening, while the first patients in the phase III CHABLIS 7.5 study evaluating blisibimod to treat systemic lupus erythematosus (SLE) were screened |
6/30/16 |
Astrazeneca plc (London) |
Anifrolumab |
A monoclonal antibody designed to block all type 1 interferons |
Moderate to severe systemic lupus erythematosus |
Significantly more anifrolumab-treated patients achieved an SLE Responder Index response at day 169 with sustained reduction of oral corticosteroids, compared to patients receiving placebo; even greater effect sizes were observed in the 75% of patients who had a high type I interferon gene signature at baseline; a new analysis of the phase II trial revealed a greater reduction in disease activity with anifrolumab treatment vs. placebo in individual organ domains frequently involved in lupus |
6/9/16 |
Aurinia Pharmaceuticals Inc. (Victoria, British Columbia) |
Voclosporin |
Calcineurin inhibitor |
Lupus nephritis |
Completed an analysis of the first seven patients to complete 24 weeks in its open-label AURION (Aurinia early Urinary protein Reduction Predicts Response) study; at 24 weeks, 57% (four of seven) of patients continued to be in complete remission as measured by a urinary protein creatinine ratio of ≤ 0.5mg/mg, eGFR within 20% of baseline and concomitant steroid dose of less than 5mg/day |
6/30/16 |
Biogen Inc. (Cambridge, Mass.) |
Opicinumab |
Anti-LINGO-1; an investigational, fully human monoclonal antibody |
Multiple sclerosis |
Missed both the primary and secondary efficacy endpoints in the phase II study Synergy |
6/8/16 |
Biopharmx Corp. (Menlo Park, Calif.) |
BPX-01 |
A topical gel formulation of minocycline |
Acne |
Topline results from its phase IIa safety study showed the product reported no cutaneous toxicity, resulted in no adverse effects, and found no detectable levels of minocycline in the patients' bloodstreams |
6/30/16 |
Coherus Biosciences Inc. (Redwood City, Calif.) |
CHS-131 |
A once-daily, oral candidate |
Multiple sclerosis |
A phase IIb study demonstrated about a 50% decrease in the incidence of new contrast-enhancing lesions over six months when compared to placebo and was generally well-tolerated, without evidence of immune suppression or the side effects commonly seen in other oral multiple sclerosis (MS) therapies |
6/29/16 |
Corbus Pharmaceuticals Holdings Inc. (Norwood, Mass.) |
Resunab |
A synthetic oral endocannabinoid mimetic |
Diffuse cutaneous systemic sclerosis |
Completed patient enrollment in its phase II trial |
6/17/16 |
Cymabay Therapeutics Inc. (Newark, Calif.) |
MBX-8025 |
An orally administered selective peroxisome proliferator-activated receptor delta agonist |
Primary biliary cholangitis |
Discontinued its phase II study after determining the study met its objective of establishing proof of concept by showing marked improvements in biochemical markers of cholestasis; the study also identified a treatment-emergent signal of transaminase elevations |
6/1/16 |
Flex Pharma Inc. (Boston) |
FLX-787 |
Chemically synthesized transient receptor potential ion channel activator |
Cramps, spasms and/or spasticity as a consequence of multiple sclerosis |
Started a phase II study in Australia |
6/16/16 |
Geneuro SA (Geneva) |
GNbAC1 |
A monoclonal antibody designed to neutralize MSRV-Env |
Relapsing-remitting multiple sclerosis |
The first patients were treated with lead product GNbAC1 in a phase IIb trial that will enroll 260 patients and will examine the cumulative number of active brain lesions, as determined by MRI after six months, as the primary endpoint |
6/1/16 |
Santalis Pharmaceuticals Inc. (San Antonio) |
EISO |
10% East Indian sandalwood oil serum formulation |
Mild to moderate plaque psoriasis |
Positive results from its single-center, open-label, efficacy and tolerability proof-of-concept study showed the EISO serum formulation to be safe, well tolerated, and efficacious for patients 18-65 years of age with a maximum of 10% treatable body surface area involvement of mild-to-moderate plaque psoriasis |
6/28/16 |
Twi Biotechnology Inc. (Taipei, Taiwan) |
AC-201CR |
First-in-class, orally available small molecule |
Gout |
Completed patient enrollment in its phase II trial |
6/28/16 |
Ziarco Pharma Ltd. (Discovery Park, Sandwich) |
ZPL-389 |
A once daily oral histamine H4 receptor antagonist |
Moderate to severe atopic dermatitis |
Full results from its first phase IIa proof-of-concept study demonstrated evidence of efficacy and safety of ZPL-389 |
6/14/16 |
CANCER | |||||
Acetylon Pharmaceuticals Inc. (Boston) |
ACY-1215 |
Ricolinostat |
Relapsed or relapsed-and-refractory multiple myeloma |
The ACE-MM-102 phase II trial showed an overall response rate of 46%, with a clinical benefit rate of 58%, and a disease control rate of 82%, nine months duration of response, and seven months progression free survival |
6/14/16 |
Advaxis Inc. (Princeton, N.J.) |
AXAL |
Axalimogene filolisbac; immunotherapy |
Persistent or recurrent metastatic anal cancer |
The first patient was dosed in the phase II trial of the firm's FAWCETT study |
6/9/16 |
Ariad Pharmaceuticals Inc. (Cambridge, Mass.) |
Iclusig |
Ponatinib; BCR-ABL inhibitor |
Resistant or intolerant chronic myeloid leukemia or Philadelphia chromosome-positive acute lymphoblastic leukemia |
Long-term follow-up data from its pivotal phase II PACE trial showed that patients treated with Iclusig continued to demonstrate anti-leukemic activity with a median follow-up of 4 years for CP-CML; additionally, 96% of CP-CML patients who underwent ponatinib dose reductions while in response maintained their responses at the four year point |
6/14/16 |
Biolinerx Ltd. (Tel Aviv, Israel) |
BL-8040 |
CXCR4 antagonist |
Pancreatic cancer |
Filed regulatory submissions to start a phase IIa trial of BL-8040 in combination with PD-1 inhibitor Keytruda (pembrolizumab, Merck & Co. Inc.) |
6/29/16 |
Biospecifics Technologies Corp. (Lynbrook, N.Y.) |
CCH |
Collagenase clostridium histolyticum |
Human lipoma |
Positive, statistically significant top-line results from a phase II trial showed it met its primary endpoint of reduction in the visible surface area of the target lipomas relative to placebo, as determined by caliper, at six months post-injection (p<0.0001), and also met all secondary efficacy endpoints |
6/14/16 |
Boehringer Ingelheim GmbH (Ingelheim, Germany) |
Olmutinib (BI 1482694) |
Third-generation EGFR mutation-specific tyrosine kinase inhibitor |
EGFR mutation-positive non-small-cell lung cancer |
Started the ELUXA trial program |
6/3/16 |
Bristol-Myers Squibb Co. (New York) |
Opdivo |
Nivolumab |
Classical Hodgkin lymphoma |
Results from CheckMate-205, a multicohort, noncomparative, single-arm, phase II registrational trial evaluating Opdivo showed the primary endpoint of objective response rate per an independent radiologic review committee was 66.3%; median time to response was 2.1 months and estimated median duration of remission was 7.8 months |
6/13/16 |
Debiopharm International SA (Lausanne, Switzerland) |
Debio 1143 |
IAP inhibitor |
Epithelial ovarian cancer |
Started a phase II study |
6/8/16 |
Eli Lilly and Co. (Indianapolis) |
Olaratumab |
A human IgG1 monoclonal antibody that is designed to disrupt the PDGF Receptor-α pathway |
Advanced soft tissue sarcoma |
Patients with advanced soft tissue sarcoma not amenable to radiotherapy or surgery who were treated with a combination of olaratumab and doxorubicin achieved 6.6 months of median progression-free survival vs. 4.1 months for those given placebo and doxorubicin (p=0.0615); overall survival was statistically significant, with patients on the olaratumab arm of the phase Ib/II study having achieved a median OS of 26.5 months compared to 14.7 months with doxorubicin alone (p=0.0003) |
6/13/16 |
Epizyme Inc. (Cambridge, Mass.) |
Tazemeto-stat |
An EZH2 inhibitor |
Non-Hodgkin lymphoma |
Phase II data indicate that tazemetostat demonstrated a favorable safety profile and clinical activity consisting of objective responses in a heavily pre-treated patient population |
6/21/16 |
Hutchison China Meditech Ltd. (Hong Kong) |
Savolitinib |
C-Met inhibitor |
Epidermal growth factor receptor mutant non-small-cell lung cancer |
Initiated a phase II expansion of the ongoing TATTON trial |
6/21/16 |
Immunomedics Inc. (Morris Plains, N.J.) |
Sacituzumab govitecan (IMMU-132) |
Anti-Τrop-2-SN-38 antibody-drug conjugate |
Triple-negative breast cancer |
Phase II results showed it had an objective response rate (ORR) of 33% in pretreated patients; as of May 2016, the ORR was 33%, with a confirmed ORR of 28% and a median duration of response of nearly 11 months; the median progression-free survival was 5.6 months and the median overall survival was 14.3 months |
6/6/16 |
Infinity Pharmaceuticals Inc. |
Duvelisib |
Oral, dual inhibitor of phosphoinositide-3-kinase-delta and P13K-gamma |
Refractory indolent non-Hodgkin lymphoma |
Phase II data showed it met its primary endpoint by demonstrating an overall response rate of 46% |
6/15/16 |
Isofol Medical AB (Gothenburg, Sweden) |
Modufolin |
Folate-based compound |
Colorectal cancer |
Started the next dose level of Modufolin in combination with 5-FU and oxaliplatin or F-5U and irinotecan in the ongoing, incremental-dose cohort, phase II trial |
6/3/16 |
Kura Oncology Inc. (La Jolla, Calif.) |
Tipifarnib |
Farnesyltransferase inhibitor |
Lower risk myelodysplastic syndromes |
The first patient has been dosed in a phase II trial |
6/20/16 |
Mateon Therapeutics Inc. (South San Francisco) |
CA4P |
Vascular-disrupting agent |
Platinum-resistant ovarian cancer |
Enrolled the first patient in the phase II/III FOCUS study |
6/24/16 |
Morphosys AG (Martinsried, Germany) |
MOR208 |
Anti-CD19 antibody |
Non-Hodgkin's lymphoma |
A case-study report from an ongoing phase Iia study shows that third-line monotherapy resulted in an ongoing complete response of more than 26 months' duration |
6/20/16 |
Onconova Therapeutics Inc. (Newtown, Pa.) |
Rigosertib |
Oral therapy |
First- or second-line higher-risk myelodyspastic syndromes |
Interim data from the ongoing phase II trial of oral rigosertib in combination with azacitidine showed an overall response rate of 77%, or 23 of 30 patients |
6/14/16 |
Oncosec Medical Inc. (San Diego) |
Immuno-pulse IL-12 |
Designed to enhance the local delivery and uptake of DNA-based immune-targeting agents such as IL-12 |
Treatment-refractory, metastatic and unresectable squamous cell carcinoma of the head and neck |
Preliminary findings from OMS-I130, a phase II trial, indicated that the therapy might lead to an increase in CD8-positive T cells and adaptive immune resistance |
6/17/16 |
Pharmaengine Inc. (Taipei, Taiwan) |
PEP503 |
NBTXR3; a nanoparticle formulation of hafnium oxide crystals |
Soft tissue sarcoma |
The first patient has been dosed in a global pivotal phase II/III trial |
6/2/16 |
Portola Pharmaceuticals Inc. (South San Francisco) |
Cerdulatinib |
An oral, dual Syk/JAK kinase inhibitor |
Resistant or relapsed hematologic cancer |
Dosed the first patient in a phase IIa study |
6/2/16 |
Prima Biomed Ltd. (Sydney) |
IMP321 |
An antigen-presenting cell activator based on immune checkpoint LAG-3 |
Hormone receptor-positive metastatic breast cancer |
Reported initial safety data from the first cohort in its phase IIb study showing the 6 mg dose in combination with paclitaxel has proved safe and well-tolerated, with no drug-related serious adverse events; data also showed activation of blood monocytes/dendritic cells and CD8 T cells |
6/23/16 |
Sophiris Bio Inc. (La Jolla, Calif.) |
Topsalysin |
An inactivated pore-forming protein |
Prostate cancer |
Biopsy results from all 18 patients enrolled in the phase IIa proof-of-concept study showed that Topsalysin demonstrated an ability to ablate tumor cells in 50% of patients (9/18 patients) six months after treatment in a patient population with pre-identified, clinically significant prostate cancer |
6/10/16 |
Tapimmune Inc. (Jacksonville, Fla.) |
TPIV 200 |
A folate receptor alpha T-cell vaccine |
Triple-negative breast cancer |
Dosed the first patient in a phase II trial |
6/22/16 |
TG Therapeutics Inc. (New York) |
TG-1101 |
Glycoengineered anti-CD20 monoclonal antibody |
Diffuse large B-cell lymphoma |
The first patient has been enrolled in a registration-directed phase IIb study |
6/8/16 |
CARDIOVASCULAR | |||||
Catabasis Pharmaceuticals Inc. (Cambridge, Mass.) |
CAT-2054 |
An oral candidate designed to inhibit sterol regulatory element-binding protein |
Hypercholes-terolemia |
Top-line results from its phase IIa trial showed it did not meet the primary endpoint of reduction in LDL-cholesterol from baseline |
6/9/16 |
Cerenis Therapeutics SA (Toulouse, France) |
CER-001 |
ApoA-I-containing HDL mimetic drug |
High cholesterol |
Results of the LOCATION phase II study demonstrated CER-001's ability to penetrate the vessel walls to preferentially target atherosclerotic plaques and to increase cholesterol efflux capacity |
6/3/16 |
Chemocentryx Inc. (Mountain View, Calif.) |
CCX168 |
Orally administered small molecule that is a selective inhibitor of the complement C5a receptor |
Anti-neutrophil cytoplasmic auto-antibody-associated vasculitis |
Top-line results from its phase II CLASSIC (CLinical ANCA Vasculitis Safety Study of Inhibitor of C5aR) study with CCX168, showing the study met its objectives, in that the addition of CCX168 to current standard-of-care (SOC) therapy did not add safety concerns beyond those seen with SOC alone |
6/17/16 |
Inflarx GmbH (Jena, Germany) |
IFX-1 |
A monoclonal anti-complement C5a antibody |
Cardiac surgery |
The first patient was dosed preventively in the phase II trial of IFX-1 |
6/9/16 |
Reata Pharmaceuticals Inc. (Irving, Texas) |
Bardoxolone methyl |
An oral once-daily antioxidant inflammation modulator |
Pulmonary arterial hypertension |
Interim data from the extension phase II LARIAT trial showed that the increase in six-minute walk distance (6MWD) through 16 weeks of treatment, which was previously reported, was sustained through 32 weeks of treatment and was not significantly different from that at week 16 in the same set of patients |
6/24/16 |
Regeneron Pharmaceuticals Inc. (Tarrytown, N.Y.) |
REGN1500 |
Evinacumab; monoclonal antibody to angiopoietin-like protein 3 |
Homozygous familial hypercholesterolemia |
Interim results from the first four patients from an ongoing proof-of-concept study showed that evinacumab, added to current lipid-lowering therapy, reduced low-density lipoprotein cholesterol levels by an additional 55% (mean reduction; range 25 to 90%) at week four compared to baseline |
6/1/16 |
Stealth Biotherapeutics Inc. (Boston) |
Elamipretide |
Investigational drug with the potential to modify disease by preserving energetics and restoring normal energy production in mitochondria, while decreasing oxidative stress |
Heart failure |
Started two phase II studies Progress-HF in patients with heart failure with reduced ejection fraction and Restore-HF in those with preserved ejection fraction |
6/28/16 |
The Medicines Co. (Parsippany, N.J.) |
PCSK9si |
RNAi proprotein convertase subtilisin/kexin type 9 synthesis inhibitor |
High cardiovascular risk and elevated low-density lipoprotein-cholesterol |
Completed patient enrollment in the phase II ORION-1 study |
6/3/16 |
CENTRAL NERVOUS SYSTEM | |||||
Addex Therapeutics SA (Geneva) |
Dipraglurant |
mGluR5 negative allosteric modulator |
Levodopa-induced dyskinesia associated with Parkinson's disease |
Results from a phase IIa study suggested it improved dyskinesia and showed no major safety concerns; the four-week, 76-patient study showed dipraglurant significantly reduced peak dose dyskinesia (modified Abnormal Involuntary Movement Scale) on day one (50 mg, 20%; p = 0.04) and on day 14 (100 mg, 32%; p = 0.04) and across a three-hour post-dose period on day 14 (p = 0.04) |
6/3/16 |
Alder Biopharma-ceuticals Inc. (Bothell, Wash.) |
ALD403 |
Calcitonin gene-related peptide antibody |
Migraine |
Phase II data showed that a single intravenous (IV) dose of 100 mg or 300 mg met the primary efficacy endpoint, producing a 75% reduction in migraine days over 12 weeks in 33% and 31% of patients, respectively (p < 0.05); a single administration of ALD403 also resulted in an immediate and durable mean reduction in migraine days from baseline throughout the 12 weeks, meeting the secondary efficacy endpoint |
6/10/16 |
Amgen Inc. (Thousand Oaks, Calif.) |
AMG 334 |
Fully human monoclonal antibody |
Chronic migraine |
Top-line findings from its global phase II study showed it met its primary endpoint of change in monthly migraine days, with a statistically significant reduction in migraine days for both the 70 mg and 140 mg doses |
6/10/16 |
Axon Neuroscience SE (Bratislava, Slovakia) |
Adamant |
Disease-modifying tau vaccine |
Alzheimer's disease |
Started the Adamant phase II study |
6/23/16 |
Bionomics Ltd. (Adelaide, Australia) |
BNC210 |
An orally administered negative allosteric modulator of the alpha-7 nicotinic acetylcholine receptor |
Generalized anxiety disorder |
Completed enrollment in its U.K.-based phase II trial |
6/27/16 |
Bone Therapeutics SA (Gosselies, Belgium) |
Preob |
Derived from patients' bone marrow, but the mesenchymal stem cells are isolated and differentiated ex vivo to bone-forming osteoblast cells |
Osteonecrosis |
Hit the primary endpoint of a phase IIb trial, showing a 24-month response rate of 70% vs. 37% for those in the control group |
6/8/16 |
Collegium Pharmaceutical Inc. (Canton, Mass.) |
Hydrocodone Deterx |
Two prospective formulations |
Pain |
Top-line results from a pharmacokinetic proof-of-concept study evaluating two prospective formulations of Hydrocodone Deterx, which showed that both candidates were bioequivalent when comparing crushed and intact dosing, demonstrating resistance to physical manipulation |
6/23/16 |
Fibrocell Science Inc. (Exton, Pa.) |
Laviv |
Azficel-T |
Vocal cord scarring resulting in chronic or severe dysphonia |
Primary endpoints were not met in its phase II trial |
6/9/16 |
Frontier Biotechnologies Inc. (Nanjing, China) |
AB001 |
Super-thin patch (200 micrometers thick) containing a nonsteroidal anti-inflammatory drug |
Back pain |
A phase II trial testing AB001 met the primary endpoint at week two, demonstrating statistically significant (p=0.023) and clinically meaningful low back pain relief against placebo |
6/23/16 |
Marinus Pharmaceuticals Inc. (Radnor, Pa.) |
Ganaxolone |
A CNS-selective GABAA modulator |
Anxiety and attention in fragile X syndrome |
Top-line data from a phase IIa exploratory, investigator-sponsored study showed the primary endpoint of Clinical Global Impression of Improvement was not met, but treatment with ganaxolone improved anxiety and hyperactivity across multiple measures in FXS patients with high baseline anxiety |
6/29/16 |
Stealth Biotherapeutics Inc. (Boston) |
Elamipretide |
Elamipretide |
Primary mitochondrial myopathy |
Phase II MMPOWER data demonstrated statistically significant improvements in distance walked in six minutes, the primary endpoint |
6/20/16 |
Stemcells Inc. (Newark, Calif.) |
HuCNS-SC |
Stem cells |
Spinal cord injury |
Terminated its phase II Pathway study following review of data and obtaining concurrence of its interim analysis data monitoring committee, which showed an overall improvement in patients treated with the company's cells but not a magnitude of effect that warrants further study |
6/1/16 |
T3D Therapeutics Inc. (Research Triangle Park, N.C.) |
T3D-959 |
Designed to improve neurometabolic dysfunction |
Mild to moderate Alzheimer's disease |
Achieved a milestone in its phase IIa study completing enrollment in the open-label, parallel-designed study; received FDA approval for an open-label, 26-week extension study, extending its phase IIa feasibility study of T3D-959 |
6/9/16 |
The Medicines Co. (Parsippany, N.J.) |
ABP-700 |
A positive allosteric modulator of the GABAA receptor |
For procedural sedation |
Dosed the first patient in a phase II study |
6/15/16 |
Tonix Pharmaceuticals Holdings Corp. (New York) |
TNX-102 SL |
Cyclobenzaprine HCl sublingual tablets |
Military-related post traumatic stress disorder |
Results from its phase II, dose-finding study (AtEase), showed that the 5.6-mg dose was efficacious and well-tolerated and will be used in the upcoming phase III study in PTSD |
6/1/16 |
DIABETES | |||||
Aerpio Therapeutics Inc. (Cincinnati) |
AKB-9778 |
A small molecule, Tie-2 activating agent |
Diabetic macular edema |
Clinical data from the phase IIa study showed the combination of AKB-9778 (dosed at 15 mg BID subcutaneously) and Lucentis (ranibizumab injection dosed at 0.3 mg intravitreally) provided a clinically significant benefit in reduction of macular edema, as measured by central subfield thickness (CST), compared to Lucentis alone at month 2 (p=0.02) and at end of treatment at month 3 (p=0.008) |
6/14/16 |
Akcea Therapeutics Inc. (Cambridge, Mass.) |
Volanesorsen |
Antisense drug |
High plasma triglyceride levels and type 2 diabetes |
Positive data from a phase II study showed that, in addition to reductions in plasma TG and apolipoprotein C-III (ApoC-III), a key factor in TG regulation, volanesorsen improved insulin sensitivity and glucose control |
6/28/16 |
Resverlogix Corp. (Calgary, Alberta) |
RVS-208 |
BET inhibitor |
Prediabetes mellitus |
Findings gathered showed that treatment with RVS-208 for 29 to 33 days led to a reduction in glucose absorption and also suppression of endogenous glucose production |
6/3/16 |
GASTROINTESTINAL | |||||
Redhill Biopharma Ltd. (Tel-Aviv, Israel) |
Bekinda |
Ondansetron 12 mg |
Diarrhea-predominant irritable bowel syndrome |
The first patients in a phase II trial have been dosed |
6/21/16 |
INFECTION | |||||
Abivax SA (Paris) |
ABX464 |
Small molecule inhibiting HIV replication |
HIV/AIDs |
The first patient was enrolled in the ABX4640004 study, the second phase IIa trial testing small-molecule ABX464 in HIV/AIDs patients. |
6/6/16 |
Aicuris Anti-Infectives Cures GmbH (Wuppertal, Germany) |
Pritelivir |
A helicase-primase inhibitor |
Herpes simplex virus-2 |
Phase II data found that no changes in HSV-2 sequence occurred during treatment and no observations of resistance mediating mutations was observed; it demonstrated reduced viral shedding and genital lesions in phase II |
6/24/16 |
Ascletis Inc. (Hangzhou, China) |
ASC08 and ASC16 |
Direct-acting antiviral agents danoprevir, an NS3/4A protease inhibitor, and ravidasvir, an NS5A inhibitor |
Hepatitis C viral infections |
Interim data from the phase II EVEREST study showed that, after 12 weeks of interferon-free treatment, virologic response rate was 100% in Chinese hepatitis C virus genotype 1 noncirrhotic patients treated with the regimen |
6/22/16 |
Cidara Therapeutics Inc. (San Diego) |
CD101 |
Echinocandin-class antifungal |
Acute vulvovaginal candidiasis |
Dosed the first patient in Radiant, a phase II trial |
6/10/16 |
Contravir Pharmaceuticals Inc. (Edison, N.J.) |
CMX157 |
An analogue of tenofovir |
Chronic hepatitis B virus |
Began screening patients for a head-to-head phase IIa study comparing it to tenofovir DF (TDF, Gilead Sciences Inc.), following a positive recommendation from an independent data safety monitoring board |
6/2/16 |
Galmed Pharmaceuticals Ltd. (Tel Aviv, Israel) |
Aramchol |
SCD1 enzyme inhibitor |
Non-alcoholic steatohepatitis |
Randomized half of the patients, or 120, in the phase IIb ARREST study |
6/2/16 |
Galmed Pharmaceuticals Ltd. (Tel Aviv, Israel) |
Aramchol |
Synthetic fatty-acid/bile-acid conjugate |
Nonalcoholic fatty liver disease and nonalcoholic steatohepatitis |
Expanded the operations of its ongoing phase IIb ARREST study into China and has started a pharmacokinetic (PK) study in healthy Chinese subjects domiciled in the U.S.; the company expects to enroll up to 64 subjects |
6/8/16 |
Genocea Biosciences Inc. (Cambridge, Mass.) |
GEN-003 |
Genital herpes therapy |
Genital herpes |
Data from the phase II dose-optimization trial showed statistically significant reductions in viral shedding and sustained efficacy 12 months after completion of dosing |
6/10/16 |
Genticel SA (Paris) |
GTL001 |
HPV 16/18 immunotherapeutic candidate |
Human papillomavirus |
Interim results at 18 months from the phase II trial showed no new unexpected safety events but also no statistical difference in viral clearance in the treated total population or in the subgroups, compared to natural clearance in the placebo group |
6/24/16 |
Immuron Ltd. (Melbourne, Australia) |
IMM-124E |
Composed of anti-lipopolysaccharide antibodies and adjuvants |
Non-alcoholic steatohepatitis |
Phase II trial has reached its 50% recruitment milestone with 56 patients having been randomized and an additional four patients to be randomized last week |
6/27/16 |
Microbion Corp. (Bozeman, Mont.) |
MBN-101 |
Broad-spectrum anti-bacterial |
Orthopedic implant-related infections |
The first patient was enrolled in a phase IIa study |
6/17/16 |
Regulus Therapeutics Inc. (La Jolla, Calif.) |
RG-101 |
MicroRNA antagonist |
Hepatitis C viral infections |
Top-line results from the primary endpoint analysis of one of the company's ongoing phase II studies found that RG-101 plus Harvoni (sofosbuvir + ledipasvir, Gilead Sciences Inc.) continues to demonstrate 100% response rates in all ten patients that received it; FDA issued a verbal clinical hold on the phase II program following a second case of jaundice |
6/8/16 |
Spring Bank Pharmaceuticals Inc. (Hopkinton, Mass.) |
SB 9200 |
A small-molecule nucleic acid hybrid compound |
Immuno-active, treatment-naïve hepatitis B virus |
Started dosing the first cohort of patients in the ACHIEVE phase II program evaluating SB 9200 |
6/23/16 |
Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) |
TAK-214 |
Norovirus vaccine |
Moderate or severe acute gastroenteritis due to norovirus |
Started a phase IIb field efficacy trial |
6/21/16 |
Trek Therapeutics PBC (Cambridge, Mass.) |
TD-6450 |
A multivalent NS5A inhibitor |
Genotype 1b hepatitis C virus infection |
Started a phase IIa study testing TD-6450 plus faldaprevir, with and without ribavirin |
6/22/16 |
Valneva SE (Lyon, France) |
VLA43 |
Vaccine |
Pseudomonas aeruginosa |
Phase II/III results confirmed good immunogenicity and an acceptable safety profile but failed to confirm the strong reduction in all-cause mortality seen in an earlier phase II study; overall survival, a secondary endpoint, also did not differ between the VLA43 treatment group and the placebo group |
6/6/16 |
MISCELLANEOUS | |||||
Acceleron Pharma Inc. (Cambridge, Mass.) and Celgene Corp. (Summit, N.J.) |
Luspatercept |
Modified activin receptor type IIB fusion protein |
Lower-risk myelodysplastic syndromes |
51% of patients during an ongoing long-term phase II extension study achieved increased hemoglobin levels; 35% of patients treated with the candidate achieved transfusion independence in the three-month base study |
6/13/16 |
Agios Pharmaceuticals Inc. (Cambridge, Mass.) |
AG-348 |
A first-in-class, oral activator of both wild-type and mutated pyruvate kinase-R enzymes |
Pyruvate kinase deficiency |
Data at the European Hematology Association congress demonstrated that AG-348 achieved proof-of-concept in an ongoing phase II study (DRIVE-PK) |
6/14/16 |
Alexion Pharmaceuticals Inc. (New Haven, Conn.) |
Strensiq |
Asfotase alfa |
Hypophos-phatasia |
Data from an open-label phase II trial involving children, ages 6 to 12.5 at study entry, showed that those treated for up to five years with Strensiq sustained improvements seen at the six-month primary endpoint cutoff, including improvements in patient growth, strength, motor function, agility and quality of life, which for most patients meant achieving normal values for age- and sex-matched peers; the study met its primary endpoint, showing significant radiographic improvement at six months |
6/17/16 |
Anthera Pharmaceuticals Inc. (Hayward, Calif.) |
Blisibimod |
A peptibody that targets B-cell activating factor |
Immunoglobulin A nephropathy |
Interim data from the phase II BRIGHT-SC study suggested a positive trend in the reduction of proteinuria compared to patients who received placebo; however, at week 24 the numerical reduction in proteinuria in patients treated with blisibimod compared to placebo did not meet the predefined statistical primary endpoint of complete or partial response |
6/30/16 |
Ardelyx Inc. (Fremont, Calif.) |
Tenapanor |
Inhibitor of the sodium-proton exchanger NHE3 |
Hyperphos-phatemia in end-stage renal disease |
Company said it will increase patient enrollment from 150 to 200 to strengthen the phase II trial and maintain a power of 90% |
6/23/16 |
Bioblast Pharma Ltd. (New Haven, Conn.) |
Trehalose |
Intravenous solution |
Oculopharyn-geal muscular dystrophy |
Plans to initiate a phase IIb trial |
6/10/16 |
Biocon Ltd. (Bangalore, India) and Quark Pharmaceuticals Inc. (Fremont, Calif.) |
QPI-1007 |
A siRNA drug candidate |
Ocular neuro-protection |
The first patient was randomized in India in the global phase II/III trial |
6/24/16 |
Capricor Therapeutics Inc. (Los Angeles) |
CAP-1002 |
Allogeneic cardiosphere-derived cell therapy |
Duchenne muscular dystrophy-associated cardiomyopathy |
Patient enrollment has now exceeded 50% of the 24-patient target in the HOPE-Duchenne trial evaluating CAP-1002, its allogeneic cardiosphere-derived cell therapy, in patients with Duchenne muscular dystrophy-associated cardiomyopathy. Capricor expects to complete enrollment in the third quarter of 2016 and to report top-line data in the first quarter of 2017. |
6/24/16 |
Cara Therapeutics Inc. (Shelton, Conn.) |
CR845 |
Kappa opioid agonist |
Moderate to severe uremic pruritis |
Phase II patients receiving intravenous [I.V.] CR845 experienced a 54% greater reduction in worst itch scores than those receiving placebo (p-value = 0.016), with an average reduction of -48 percent from baseline as measured by the VAS. I.V.; CR845-treated patients exhibited statistically significant reductions in both daytime (-51%, p=0.03) and nighttime (-75%, p=0.007) worst itch scores compared to placebo treatment |
6/30/16 |
Hansa Medical AB (Malmo, Sweden) |
Ides |
Antibody-degrading enzyme |
Kidney transplant |
Additional data from its ongoing phase II study showed that Ides reduced to zero the level of donor-specific antibodies in all kidney transplant patients within six hours of treatment |
6/16/16 |
Millendo Therapeutics Inc. (Ann Arbor, Mich.) |
ATR-101 |
Adrenal-selective small-molecule inhibitor of ACAT1 |
Congenital adrenal hyperplasia |
Started a phase II trial |
6/9/16 |
Neurovive Pharmaceutical AB (Lund, Sweden) |
Ciclomulsion |
Ciclosporin |
Acute kidney injury |
A clinical research team completed its planned enrollment of 150 evaluable patients in the phase II study, CiPRICS (Ciclosporin to Protect Renal function In Cardiac Surgery) |
6/8/16 |
Noveome Biotherapeutics Inc. (Pittsburgh) |
ST266 |
Secretome |
Moderate to severe periodontitis |
Started a phase II trial |
6/16/16 |
Soligenix Inc. (Princeton, N.J.) |
SGX942 |
Lead clinical Innate Defense Regulator, containing the active ingredient dusquetide |
Oral mucositis |
Phase II showed the reduction in duration of severe oral mucositis in treated patients ranged from 50% in the overall population to 67% in the population with the most severe disease |
6/7/16 |
Stealth Biotherapeutics Inc. (Boston) |
Elamipretide |
Formerly Ocuvia |
Leber's hereditary optic neuropathy |
Started Resight, a phase II study |
6/2/16 |
Stealth Biotherapeutics Inc. (Boston) |
Elamipretide |
Systemically delivered |
Primary mitochondrial myopathy |
MMPOWER phase II trial yielded positive data |
6/7/16 |
Summit Therapeutics plc (Oxford, U.K.) |
Ezutromid |
Formerly SMT C1100 |
Duchenne muscular dystrophy |
Enrolled the first patient in Phaseout DMD, a phase II proof-of-concept trial |
6/20/16 |
Tonix Pharmaceuticals Holdings Corp. (New York) |
TNX-102 SL |
Cyclobenzaprine HCl sublingual tablets |
Fibromyalgia |
Results from a retrospective analysis from its phase IIb BESTFIT clinical study demonstrate improvements in the key domains of fibromyalgia and showed that TNX-102 SL has broad activity confirmed by different experimental responder analyses |
6/10/16 |
Wilson Therapeutics AB (Stockholm) |
Decuprate |
Bis-choline tetrathiomolybdate; WTX101 |
Wilson disease |
Preliminary data from the ongoing phase II trial of Decuprate showed that during the study, liver status measured using the Revised King's Score (Modified Nazer Score), has improved or remained stable in 20 out of 20 patients at the last observation compared to baseline |
6/1/16 |
RESPIRATORY | |||||
Aimmune Therapeutics Inc. (Vienna) |
AR101 |
A proprietary formulation of characterized peanut flour and pharmaceutical-grade ingredients contained in industry-standard capsules |
Peanut allergy |
New phase II extended maintenance therapy data showed that the safety and tolerability profile improved with continued treatment, particularly in a low-dose extended maintenance regimen |
6/14/16 |
Allergy Therapeutics plc (Worthing, U.K.) |
Grassma-tampl |
An ultra-short course subcutaneous allergen specific immunotherapy |
Grass pollen allergy |
Phase II dose-ranging study (G204) did not determine a recommended dose for the phase III trial; a further dose range-finding study will be implemented prior to proceeding into the planned pivotal phase III study |
6/28/16 |
Global Blood Therapeutics Inc. (South San Francisco) |
GBT440 |
A hemoglobin modifier |
Idiopathic pulmonary fibrosis and hypoxemia |
Started a phase IIa study |
6/21/16 |
Kadmon Corp. LLC (New York) |
KD025 |
Rho-associated coiled-coil kinase 2, or ROCK2, inhibitor |
Idiopathic pulmonary fibrosis |
The first patient was dosed in a phase II trial |
6/6/16 |
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. |