Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Kineta Inc. (Seattle)

Dalazatide

Synthetic analog of a peptide isolated from a Caribbean sea anemone

Active plaque psoriasis

Phase Ib active plaque psoriasis clinical trial showed validated blood biomarkers that confirm the drug's mechanism of action for psoriasis

6/10/16

Momenta Pharmaceuticals Inc. (Cambridge, Mass.)

M281

An anti-FcRn monoclonal antibody

Autoimmune disease

Dosed the first healthy volunteers in a phase I randomized, double-blind, placebo-controlled, ascending-dose cohort study

6/10/16

Samsung Bioepis Co. Ltd. (Incheon, Korea)

Benepali, Flixabi and SB5

Etanercept, infliximab and adalimumab; anti-TNF-alpha biosimilar portfolio

Autoimmune disorders

Long-term data showed Benepali and Flixabi sustained comparable efficacy and safety profiles to etanercept and infliximab reference products (Enbrel, Amgen Inc.; Remicade, Johnson & Johnson, respectively) up to week 100 and week 78; in a one-year phase III study, SB5 demonstrated comparable efficacy and safety to the reference product (Humira, Abbvie Inc.)

6/9/16

Sandoz Inc. (division of Novartis AG; Basel, Switzerland)

Biosimilar etanercept and rituximab

Biosimilars to Enbrel and Mabthera

Autoimmune diseases

Results from two key studies comparing its biosimilars to originator products Enbrel and Mabthera showed the primary endpoints of achieving PK bioequivalence were met

6/10/16

UCB SA (Brussels)

Bimekizumab

A selective monoclonal antibody that inhibits IL-17A and IL-17F

Rheumatology

Phase Ib data showed that bimekizumab demonstrated fast and sustained efficacy on disease activity measures in both skin and joints and was well-tolerated

6/13/16

CANCER

Actinium Pharmaceuticals Inc. (New York)

Actimab-A

Monoclonal antibody HuM195 and radioisotope acitnium-225

Acute myeloid leukemia with low peripheral blast burden

Phase I data showed it produced a 50% complete response at the selected phase II dose

6/2/16

Agenus Inc. (Lexington, Mass.) and Incyte Corp. (Wilmington, Del.)

ICAGN1876

Anti-GITR agonist antibody

Advanced or metastatic solid tumors

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

6/23/16

Apogenix GmbH (Heidelberg, Germany)

APG101

A fully human fusion protein

Low to intermediate-1 risk transfusion-dependent patients with myelodysplastic syndromes

Final top-line results from a phase I trial showed the drug was well-tolerated and efficiently stimulated erythropoiesis

6/16/16

Ariad Pharmaceuticals Inc. (Cambridge, Mass.)

AP32788

A tyrosine kinase inhibitor designed as a targeted therapy

Non-small-cell lung cancer

Started a phase I/II trial

6/1/16

Cascadian Therapeutics Inc. (Seattle)

ONT-380

Small-molecule HER2 inhibitor

HER2-positive locally advanced or metastatic breast cancer

Updated phase Ib data showed the overall objective response rate, as defined by RECIST 1.1, was 58%, with one complete response, 13 partial responses, six patients with stable disease and four patients with progressive disease

6/15/16

Cellectar Biosciences Inc. (Madison, Wis.)

CLR 125

A radiotherapeutic isotope conjugated to the company's phospholipid-drug conjugate technology

Triple-negative breast cancer

Phase I data demonstrated that a single dose of CLR 125 reduced the volume of human-derived primary triple-negative breast cancer xenografts by about 60%, compared to a control vehicle (p<0.001), as well as significantly extending survival; CLR 125 also significantly weakened the progression of micrometastases (p< 0.01) and reduced established metastases (p< 0.01) compared to the control vehicle

6/24/16

Cellectis SA (Paris)

UCART19

CAR T-cell candidate

Relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia

Treated the first patient in the open-label, noncomparative phase I study

6/22/16

Celyad SA (Mont-Saint-Guibert, Belgium)

NKR-2

T-cell therapy

Acute myeloid leukemia or multiple myeloma

Infused the first patient enrolled in the fourth dose level of its phase I/IIa trial

6/24/16

Cerulean Pharma Inc. (Waltham, Mass.)

CRLX301

A tumor-targeted NDC designed to release docetaxel as its payload

Advanced solid tumors

First patient was dosed in the phase IIa study of an ongoing phase I/IIa trial

6/24/16

Corcept Therapeutics Inc. (Menlo Park, Calif.)

Mifepristone

Cortisol receptor antagonist

Metastatic triple-negative breast cancer

Preliminary efficacy data from its phase I/II trial showed that as determined using RECIST criteria, three patients showed partial response, defined as 30% or greater reduction in tumor size, eight had stable disease and 11 had progressive disease; the combination of mifepristone and Halaven (eribulin, Eisai Inc.) was well-tolerated. Neutropenia was manageable with administration of growth factor

6/6/16

Corcept Therapeutics Inc. (Menlo Park, Calif.)

CORT125134

Selective cortisol modulator

Solid tumors

Began dosing patients in a phase I/II trial in combination with Abraxane (nab-paclitaxel, Celgene Corp.)

6/6/16

Curis Inc. (Lexington, Mass.)

CA-170

An oral, small molecule inhibitor of immune checkpoints PD-L1 and V-domain immunoglobulin suppressor of T-cell activation

Cancer

The first patient was dosed in a phase I trial

6/22/16

Glycomimetics Inc. (Rockville, Md.)

GMI-1271

E-selectin antagonist

Relapsed/refractory acute myeloid leukemia

Data from the phase I portion of the phase I/II trial of GMI-1271, combined with induction chemotherapy, showed an overall response rate of 47% among 19 patients, including those who were older than 60 years of age, with primary refractory or relapsed disease, poor cytogenetic risk factors, including FLT-3 ITDs, and/or extramedullary disease; the first patient with newly diagnosed acute myeloid leukemia was dosed in the phase II portion of its phase I/II study GMI-1271 in combination with chemotherapy

6/13/16

Karyopharm Therapeutics Inc. (Newton, Mass.)

KPT-330

Selinexor; an oral selective inhibitor of nuclear export

Relapsed/refractory multiple myeloma

Data from its phase Ib study showed that of the 16 evaluable patients treated in the selinexor plus dexamethasone plus Velcade (bortezomib, Takeda Oncology) arm, 11 responded (one complete response, three with very good partial responses [VGPRs] and seven with partial responses [PRs]) for an overall response rate (ORR) of 69%

6/13/16

Karyopharm Therapeutics Inc. (Newton, Mass.)

KPT-9274

An oral, dual-acting p21-activated kinase 4 and nicotinamide phosphoribosyltransferase inhibitor

Advanced solid malignancies

Dosed the first patient in a phase I trial

6/23/16

Mabvax Therapeutics Holdings Inc. (San Diego)

MVT-5873

Humab-5B1

Locally advanced or metastatic adenocarcinoma

Started patient enrollment in the phase I trial

6/8/16

Medivation Inc. (San Francisco)

Pidilizumab

Antibody with immune-mediated antitumor effects

Diffuse intrinsic pontine glioma

Results from a phase I/II study demonstrated potential benefit in pediatric patients; mean event-free and overall survival estimates were 12 months and 15.6 months, respectively; three patients with DIPG remained progression-free at 16.3 months, 22 months and 24 months, respectively

6/17/16

Mina Therapeutics Ltd. (London)

MTL-CEBPA

Small activating RNA (saRNA) product

Advanced liver cancer

The first patient has been treated in a phase I study

6/3/16

Mundipharma International Corp. Ltd. (Basel, Switzerland)

EDO-S101

A fusion molecule combining an alkylating agent with a pan-HDAC inhibitor

Hematologic malignancies

Started a phase I trial

6/1/16

Nordic Nanovector ASA (Oslo, Norway)

HH1

Anti-CD37 antibody

Non-Hodgkin lymphoma

Ongoing phase I/II data demonstrate that pre-dosing with HH1, prior to injection with Betalutin (177Lu-HH1), significantly increases the pharmacokinetic exposure of NHL tumor cells to Betalutin (p < 0.001) while protecting against hematological side effects

6/13/16

Novonco Therapeutics Inc. (Los Angeles)

COH29

Designed to target ribonucleotide reductase

Solid tumors

Started a phase I trial testing COH29 in patients with solid tumors, including breast, colon, ovarian, pancreatic, stomach and lung cancers

6/24/16

Prothena Corp. (Dublin)

PRX003

Monoclonal antibody

Melanoma

Phase I double-blind, placebo-controlled, single ascending dose study data showed it was safe and well-tolerated following a single infusion, up to and including the highest dose level tested of 30 mg/kg

6/10/16

Scancell Holdings plc

SCIB1

DNA-based cancer vaccine

Melanoma

Company had to bin supplies as routine testing showed the clinical material no longer conformed to its original specification, delaying a phase I/II study of eight melanoma patients

6/20/16

Seattle Genetics Inc. (Bothell, Wash.)

SGN-CD33A

Vadastuximab talirine

Acute myeloid leukemia

SGN-CD33A in combination with hypomethylating agents (HMAs; azacitidine, decitabine) in frontline patients who had declined intensive therapy suggest that the addition of 33A improves the rates of response and durable remissions in comparison to that seen historically from using the current standard of care alone.

6/14/16

Sunesis Pharmaceuticals Inc. (South San Francisco)

Vosaroxin

A topoisomerase II inhibitor causing site-selective DNA damage

Acute myeloid leukemia and high-risk myelodysplastic syndrome

Updated results from an ongoing phase Ib/II University of Texas MD Anderson Cancer Center-sponsored trial of vosaroxin in combination with decitabine in older patients with previously untreated acute myeloid leukemia and high-risk myelodysplastic syndrome demonstrates a CR/CRp/CRi rate of 76% and a median overall survival of 16.1 months

6/14/16

Symphogen A/S (Copenhagen) and Selexis SA (Geneva)

Sym015

A multitargeting monoclonal antibody mixture against the MET receptor

Solid tumors

Moved Sym015 into a phase I dose-escalation study

6/30/16

Targovax ASA (Oslo, Norway)

TG01

Therapeutic peptide vaccine

Resected pancreatic cancer

The open label phase I/II trial of TG01 in combination with gemcitabine is fully recruited

6/8/16

TG Therapeutics Inc. (New York)

TGR-1202

Orally available PI3K delta inhibitor

Relapsed/refractory chronic lymphocytic leukemia or mantle cell lymphoma

Data from a phase I/Ib study of TGR-1202 in combination with Imbruvica (ibrutinib, Abbvie Inc.) showed an overall response rate (ORR) of 82% (nine of 11 patients) in CLL, with one patient achieving a complete response (CR) confirmed by a negative bone marrow and several other patients approaching a CR radiographically; 60% (six of 10 patients) with MCL achieved ORR, with clinical benefit observed in two additional patients

6/13/16

Tyme Technologies Inc. (New York)

SM-88

Designed to target only active cancer cells

Prostate cancer

Has begun recruiting for a phase Ib/II trial, using its compound, SM-88, to treat prostate cancer

6/14/16

Zenith Epigenetics Corp. (Calgary, Alberta)

ZEN-3694

A bromodomain inhibitor

Metastatic castration resistant prostate cancer

Dosed the first patient in its phase I trial

6/17/16

Ziopharm Oncology Inc. (Boston)

Ad-RTS-hIL-12

Single intratumoral injection

Recurrent or progressive glioblastoma or grade III malignant glioma

Completed enrollment in the first and second dosing cohorts and initiated enrollment in a third cohort in the phase I study of Ad-RTS-hIL-12 plus orally administered veledimex

6/29/16

CARDIOVASCULAR

Achillion Pharmaceuticals Inc. (New Haven, Conn.)

ACH-4471

Small-molecule factor D inhibitor

Paroxysmal nocturnal hemoglobinuria and C3 glomerulopathy

Achillion reported interim phase I results showing it achieved peak plasma concentrations between one and 2.5 hours after oral dosing; up to 100% inhibition of complement activity was achieved in all dose groups, and duration of inhibition was dose dependent

6/13/16

Alexion Pharmaceuticals Inc. (New Haven, Conn.)

ALXN1210

Longer-acting anti-C5 antibody

Paroxysmal nocturnal hemoglobinuria

Interim data from a phase I/II study showed that ALXN1210 achieved rapid and sustained reductions in mean levels of lactate dehydrogenase, a marker of hemolysis, in 100% of treated patients, which were observed through up to five once-monthly dosing intervals

6/13/16

Global Blood Therapeutics Inc. (South San Francisco)

GBT440

Believed to work by blocking polymerization and the resultant sickling of red blood

Sickle cell disease

Results from its ongoing phase I/II GBT440-001 study showed a durable reduction in hemolysis from baseline, as evidence by a rapid and sustained reduction in bilirubin starting as early as day four

6/13/16

Ra Pharmaceuticals Inc. (Cambridge, Mass.)

RA101495

An inhibitor of C5-mediated hemolysis

Paroxysmal nocturnal hemoglobinuria

Phase I data showed its RA101495 demonstrated a favorable safety and tolerability profile in the first-in-human study, with pharmacokinetic and pharmacodynamic properties supporting development of the drug as a self-administered, subcutaneous option

6/13/16

Regenerx Biopharma-ceuticals Inc. (Rockville, Md.)

Thymosin beta 4

Protein therapy

Acute ST segment elevation myocardial infarction

Data from a 10-patient pilot trial demonstrated after six months, the left ventricular ejection fraction based on two different measurements improved by more than 50% (p<0.05), and the stroke volume, amount of blood ejected by the left ventricle, improved by about 50% (p<0.05) in the Tbeta4-pre-treated group

6/15/16

Spark Therapeutics Inc. (Philadelphia)

SPK-9001

Lead investigational candidate in its SPK-FIX program

Hemophilia B

Updated results of the first cohort from the ongoing phase I/II trial show that the low dose cohort of four subjects enrolled in the study experienced consistent and sustained factor IX activity levels following a single administration of SPK-9001 at the initial dose level (5 x 1011 vg/kg) studied in the trial

6/14/16

Tigenix NV (Leuven, Belgium)

AlloCSC-01

Cardiac stem cell treatment

Acute myocardial infarction

Preliminary six-month results from the CAREMI trial, an exploratory phase I/II study, confirm that intracoronary delivery is well tolerated during the acute and subacute phases of the infarct

6/20/16

Uniqure N.V. (Amsterdam, the Netherlands)

AMT-060

Gene therapy

Hemophilia B

Additional data from its phase I/II trial showed that five patients who received a single administration of AMT-060, at the initial low dose of 5x1012 gc/kg, saw improvements in their disease phenotype and achieved sustained increases in FIX activity, with a median of 5.4% (expressed as percent of normal) at six months post treatment

6/14/16

CENTRAL NERVOUS SYSTEM

Acorda Therapeutics Inc. (Ardsley, N.Y.)

CVT-427

Inhaled zolmitriptan candidate

Headaches

During a phase I intra-patient, single ascending dose trial, oral and nasal spray formulations had a median Tmax of 1.5 hours and 3 hours, respectively vs. a median Tmax of 0.17 hours for all four dose levels of CVT-427

6/10/16

Acura Pharmaceuticals Inc. (Palatine, Ill.)

LTX-04P

A hydromorphone hydrochloride immediate-release tablet

Pain

During a pharmacokinetic study in healthy adults, LTX-04P successfully retarded the release of the active opioid ingredient when four, six and eight intact tablets were ingested; the data came from the second cohort of study 400

6/10/16

Alnylam Pharmaceuticals Inc. (Cambridge, Mass.)

ALN-TTRsc02

Subcutaneously administered RNAi candidate

Transthyretin-mediated amyloidosis

Initiated a phase I trial

6/10/16

Arrien Pharmaceuticals LLC (Salt Lake City)

ARN-6039

Inverse agonist of RAR-related orphan receptor gamma

Relapsing, remitting and progressive multiple sclerosis

Initiated phase I trials

6/6/16

Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.)

Nuedexta

Dextromethorphan hydrobromide/quinidine sulfate

Pseudobulbar affect in Alzheimer's disease, dementias, stroke and traumatic brain injury

Complete results from the PRISM II study showed the Center for Neurologic Study–Lability Scale (CNS-LS) score improved from a mean of 20.5 at baseline to 12.8 (P<0.001) at the 90-day endpoint, consistent with results seen in the phase III trial of Nuedexta; PBA episodes were reduced by 72.6% (P<0.001) compared to baseline at the 90-day endpoint

6/10/16

Kempharm Inc. (Coralville, Iowa)

KP511

Prodrug of hydromorphone

Pain

Results from a phase I proof-of-concept trial showed comparable hydromorphone exposure between 4 mg Dilaudid (hydromorphone hydrochloride, Purdue Pharma LP) oral liquid and an equimolar 8 mg dose of KP511

6/30/16

Living Cell Technologies Ltd. (Sydney)

Ntcell

Cell therapy candidate

Parkinson's disease

81 weeks after treatment all four patients who took part in the phase I/IIa trial showed reversal of the progression of disease, as measured by the Unified Parkinson's Disease Rating Scale

6/8/16

Marinus Pharmaceuticals Inc. (Radnor, Pa.)

Ganaxolone IV

An intravenous formulation of its CNS-selective GABAA modulator

Status epilepticus

Dosed the first subject in a phase I trial

6/23/16

Neuromax Ltd. (Moscow) and Aquilus Pharmaceuticals Inc. (Winchester, Mass.)

AQU-005

A dual active inhibitor of matrix metalloprotease type 2 and 9

Chronic neuropathic pain

Started a phase I, open-label, safety, tolerability and pharmacokinetics study

6/29/16

Retrophin Inc. (San Diego)

RE-024

Replacement therapy for pantothenate kinase-associated neurodegeneration

Parkinson's disease

Data from physician-initiated treatment with RE-024 showed it was safe and well-tolerated in two adults with PKAN who experienced clinically meaningful improvements, followed by stabilization of disease progression over 47 weeks of treatment

6/24/16

Sage Therapeutics Inc. (Cambridge, Mass.)

SAGE-217

Orally active compound intended to enhance GABA receptor-mediated inhibition in the brain

GABAA dysfunction-related disorders (essential tremor and seizures associated with epilepsy)

Top-line results from its phase I program showed it was found to be generally well-tolerated, with no serious adverse events

6/9/16

Voyager Therapeutics Inc. (Cambridge, Mass.)

VY-AADC01

Gene therapy

Advanced Parkinson's disease

Interim surgical results from an ongoing phase Ib study showed it was well-tolerated with no treatment-related serious adverse events

6/23/16

Zynerba Pharmaceuticals Inc. (Devon, Pa.)

ZYN002

Cannabidiol gel

Epilepsy, osteoarthritis and fragile X syndrome

Top-line results from a phase I, multiple rising-dose trial demonstrated that ZYN002 CBD gel was safe and well-tolerated at all dose levels

6/29/16

DIABETES

Adocia SAS (Lyon, France) and Eli Lilly and Co. (Indianapolis)

Biochaperone Lispro

An ultra-rapid formulation of insulin lispro

Diabetes

Top-line results from a phase I trial that enrolled 15 healthy Japanese subjects showed an acceleration of Biochaperone Lispro pharmacokinetic and pharmacodynamic profiles relative to Humalog, as well as the linearity of exposure as a function of the dose administered in the pharmacokinetic profile

6/1/16

NGM Bio Inc. (South San Francisco)

NGM313

An agonistic antibody engineered to selectively activate the beta-klotha-FGFR1c receptor complex

Type 2 diabetes, obesity and nonalcoholic steatohepatitis

Started a phase I trial of NGM313

6/9/16

Poxel SA (Lyon, France)

PXL770

Direct adenosine monophosphate-activated protein kinase activator

Diabetes

Six single ascending oral doses exhibited a favorable safety and tolerability profile in 64 healthy men enrolled in a phase I trial, with no serious adverse events reported nor safety signals

6/21/16

GASTROINTESTINAL

Theravance Biopharma Inc. (Dublin)

TD-1473

Orally administered gastrointestinal-targeted JAK inhibitor

Gastrointestinal disorders

Phase I results showed that TD-1473 was generally safe and well-tolerated as a single dose (up to 1,000 mg) and as a daily dose (up to 300 mg) given for 14 days, with no serious adverse events

6/13/16

INFECTION

Arrowhead Pharmaceuticals Inc. (Pasadena, Calif.)

ARC-521

RNAi-based candidate

Chronic hepatitis B virus infection

Initiated a phase I/II safety, tolerability and pharmacokinetics study of ARC-521

6/10/16

Enanta Pharmaceuticals Inc. (Watertown, Mass.)

EDP-494

A pan-genotypic cyclophilin inhibitor

Genotype 1 or genotype 3 chronic hepatitis C virus

Started a phase I, proof-of-concept study

6/21/16

Genticel SA (Toulouse, France)

GTL001

A bivalent vaccine candidate

Human papillomavirus 16 or 18

Phase I data showed it was generally safe and well-tolerated and induced a specific immune response in women with normal cytology infective with HPV 16 or 18, and provided initial evidence it could promote clearance of the virus in that population

6/2/16

Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.) and Geneone Life Science Inc. (Seoul, South Korea)

GLS-5700

Synthetic Zika DNA vaccine

Zika virus

FDA approved the start of a phase I trial

6/21/16

Mologen AG (Berlin)

MGN1703

TLR9 agonist

HIV

The Danish Aarhus University Hospital, treated the first patients in the extension phase of the phase I/IIa TEACH trial

6/29/16

INFLAMMATORY

Taiwan Liposome Co. Ltd. (Taipei, Taiwan)

TLC599

A new formulation entrapping a corticosteroid using TLC's sustained-release platform, Bioseizer

Osteoarthritis

Recruited 40 patients for its phase I/II trial

6/23/16

MISCELLANEOUS

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

PAG-519

Experimental therapy for pyruvate kinase deficiency

Pyruvate kinase deficiency

During a phase I healthy volunteers study, dosing of PAG-519 resulted in a dose-dependent increase in pyruvate kinase-R activity as evidenced by a substantial increase in adenosine triphosphate and decrease in 2,3-DPG (2,3-diphosphoglycerate) levels, which are important biomarkers of PKR activation in healthy volunteers

6/10/16

Apellis Pharmaceuticals Inc. (Louisville, Ky.)

APL-2

Complement C3 inhibitor

Paroxysmal nocturnal hemoglobinuria

Results from two phase I trials showed that pharmacological doses were safe and well-tolerated, with a pharmacokinetic and pharmacodynamic profile that supports daily subcutaneous administration

6/24/16

Eyegate Pharmaceuticals Inc. (Waltham, Mass.)

EGP-437

Iontophoretic

Ocular inflammation and pain in cataract surgery

Interim data from its phase Ib/Iia trial showed that a majority had a positive response, with some patients in the 9 mA-min and 14 mA-min dose cohorts presenting with clinically relevant reductions in anterior chamber cell count - zero or trace ACC levels at day 14

6/2/16

Fibrocell Science Inc. (Exton, Pa.)

FCX-007

Genetically-modified drug candidate

Recessive dystrophic epidermolysis bullosa

Started recruiting adult patients for its phase I/II trial

6/14/16

Galderma SA (Lausanne, Switzerland)

Trifarotene

A new retinoic acid receptor agonist

Lamellar ichthyosis

Was demonstrated to be safe and well-tolerated in a phase I study

6/10/16

Gensight Biologics SA (Paris)

GS010

AAV2 containing the human wild-type ND4 gene

Leber's hereditary optic neuropathy

Results of a phase I/II study testing GS010 in 15 patients showed that at 48 weeks post-injection in patients with an onset of disease of less than two years, there was a gain of +30 letters (-0.59 LogMAR) observed in the treated eye and +13 letters (-0.25 LogMAR) in the untreated eye, a difference of 17 letters in favor of the treated eye

6/9/16

True North Therapeutics Inc. (South San Francisco)

TNT009

A first-in-class monoclonal antibody that selectively inhibits the Classical Complement pathway by targeting C1s

Cold agglutinin disease

New clinical data showed encouraging initial results for the first CAD patients dosed in the ongoing phase Ib study

6/14/16

RESPIRATORY

Acerus Pharmaceuticals Corp. (Toronto)

Natesto

A testosterone nasal gel

Seasonal allergies

The phase I pharmacokinetic study demonstrated that the absorption and efficacy of the nasal gel is maintained even when patients with seasonal allergies (rhinitis) are forcibly challenged with pollen from Dactylis glomerata

6/20/16

Concert Pharmaceuticals Inc. (Lexington, Mass.)

CTP-656

A next-generation CFTR potentiator

Cystic fibrosis

Data from a phase I multiple ascending-dose trial of CTP-656 showed the drug provided substantially superior key exposure parameters

6/13/16

Paradigm Biopharma-ceuticals Ltd. (Melbourne, Australia)

PPS

Pentosan polysulfate sodium

Hay fever

Will be delivered intranasally for the first time in human volunteers in a phase I trial

6/10/16

Polyphor Ltd. (Allschwil, Switzerland)

POL6014

Inhaled therapy; macrocycle drug candidate designed to selectively and reversibly inhibit human neutrophil elastase

Cystic fibrosis

Phase I data showed it was well-tolerated; no serious adverse events were reported, and the measured pharmacokinetic parameters increased proportionally with the dose administered using a customized Pari Eflow aerosol inhaler

6/7/16

Vaxart Inc. (South San Francisco)

Vaccine

F-protein-based RSV tablet vaccine

Respiratory syncytial virus

Started a phase I trial

6/29/16

Vectura Group plc (Chippenham, U.K.)

VR942

Inhaled biologic immunomodulatory candidate

Asthma

Completed a phase I study, which met the primary objective of safety and tolerability of once-daily single or repeat doses in healthy volunteers and mild asthmatics, respectively; no immunogenicity concerns were detected, and pharmacokinetic analysis demonstrated undetectable systemic exposure consistent with the mode of administration

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.