Cancer

4SC AG, of Planegg, Martinsried, Germany

Domatinostat

Small-molecule class I selective HDAC inhibitor

Unresectable, advanced-stage cutaneous melanoma

Safety review committee recommended continuation of the second dose cohort in the ongoing phase Ib/II SENSITIZE trial enrolling patients who are refractory or nonresponding to prior treatment with anti-PD-1 antibodies

7/3/18

Adaptimmune Therapeutics plc, of Oxford, U.K.

SPEAR T cells

T cells targeting MAGE-A10

Non-small-cell lung cancer

In the second cohort of 3 patients who received 1B cells, there was no evidence of toxicity related to off-target binding or alloreactivity; the safety review committee recommended enrolling the third dose cohort, which will receive 1.2B-6B cells

7/18/18

Aravive Biologics Inc., of Houston

AVB-S6-500

GAS6 inhibitor

Healthy volunteers (eventually ovarian cancer)

In the 32-subject study, AVB-S6-500 met the safety and tolerability endpoints for the trial and suppressed serum GAS6 level

7/10/18

Arcus Biosciences Inc., of Hayward, Calif.

AB-154

Anti-TIGIT antibody

Tumors associated with high levels of TIGIT or CD155

Received regulatory approval and plans to start study shortly; trial will test safety, pharmacokinetics, pharmacodynamics and clinical activity of AB-154 as a monotherapy and in combination with anti-PD-1 antibody AB-122; preliminary dose-escalation data are expected in 2019

7/30/18

Arcus Biosciences, of Hayward, Calif.

AB-928

Adenosine receptor antagonist

Healthy volunteers (eventually non-small-cell lung cancer and other tumors)

In 85-subject study, drug was well-tolerated at all evaluated doses, including 200 mg; pharmacodynamic data showed dose between 75 mg and 150 mg once daily should be sufficient to achieve greater than 90% inhibition of the adenosine 2a receptor pathway; plans to run a phase I/Ib study testing 75 mg of AB-928 with an anti-PD-1 antibody, AB-122

7/17/18

Asterias Biotherapeutics Inc., of Fremont, Calif.

AST-VAC2

Allogeneic dendritic cells expressing a modified form of telomerase

Non-small-cell lung cancer

After reviewing data from the first patient, the safety review committee recommended the study continue and for parallel enrollment of second and third patients

7/11/18

Aura Biosciences, of Cambridge, Mass.

AU-011

Viral capsid conjugates with IR-700DX dye molecules

Primary choroidal melanoma

In the ongoing 30-patient trial, 100% of the patients met the endpoint of stable disease at 3 months with several having evidence of reduction in tumor height; plans to complete enrollment in October

7/23/18

Bristol-Myers Squibb Co., of New York, and Gritstone Oncology Inc., of Emeryville, Calif.

GRANITE-001, Opdivo (nivolumab) and Yervoy (ipilimumab)

Personalized neoantigen immunotherapy, anti-PD-1 antibody and anti-CTLA-4 antibody

Advanced solid tumors

Companies are collaborating to test GRANITE-001 with Opdivo and with Opdivo plus Yervoy in dose-escalation study that will start before the end of 2018

7/19/18

Cannabics Pharmaceuticals Inc., of Bethesda, Md.

Cannabics SR 5 mg

Cannabinoid-based therapy

Cancer anorexia cachexia syndrome

Trial concluded

7/3/18

CBT Pharmaceuticals Inc., of Pleasanton, Calif.

CBT-501 (genolimzumab)

IgG4 humanized monoclonal antibody targeting PD-1

Advanced solid tumors

Safety review committee recommended moving into phase Ib portion of study in approximately 20 patients

7/26/18

Celsion Corp., of Lawrenceville, N.J.

Thermadox

Heat-activated liposomal encapsulation of doxorubicin

Liver cancer

Data published in The Lancet Oncology show 7 out of 10 patients treated with a focused ultrasound application had doxorubicin concentrations within the liver tumor that were between 2 and 10 times higher; average increase of doxorubicin concentration was 3.7 times across all 10 patients

7/10/18

Dnatrix Inc., of Houston

DNX-2401 (tasadenoturev)

Oncolytic virus

Pediatric diffuse midline gliomas

Updated data showed the drug can be administered safely to the pons in the brainstem of pediatric patients prior to radiotherapy, with minimal side effects

7/2/18

Helix Biopharma Corp., of Richmond Hill, Ontario

L-DOS-47

Antibody conjugated urease

Metastatic nonsquamous non-small-cell lung cancer

After reviewing data from the fourth dosing cohort of the LDOS001 study testing L-DOS-47 plus pemetrexed and carboplatin, the safety review committee recommended starting enrollment of patients into the fifth dosing cohort; 5 out of 11 patients have had a partial response to the drug with tumor reductions of 33% to 91%; plans to enroll 7 more patients in next 3 cohorts

7/25/18

Hutchison China Meditech Ltd., of London

Sulfatinib 

VEGFR, FGFR and CSF-1R kinase inhibitor

Pancreatic neuroendocrine tumors and biliary tract cancer

Started open-label study in the U.S. measuring progression-free survival, objective response rate, disease control rate, duration of response, time to response, overall survival, safety and tolerability of the drug

7/23/18

Immunicum AB, of Gothenburg, Sweden

Ilixadencel

Activated allogeneic dendritic cells

Head and neck cancer, non-small-cell lung cancer and gastric and gastroesophageal junction adenocarcinoma

FDA approved the clinical trial protocol for study testing intratumorally administered ilixadencel in combination with checkpoint inhibitors; dose-escalation phase Ib portion includes 21 patients; phase II portion will include up to 150 patients comparing the combination to checkpoint inhibitors alone; plans to enroll the first patient in the second half of 2018

7/23/18

Minomic International Ltd., of Sydney

Miltuximab

Anti-glypican 1 antibody conjugated to the radioactive isotope 67Gallium

Metastatic prostate, bladder and pancreatic cancers

Completed dosing of all 12 patients in study designed to assess the safety and tolerability as well as the tumor targeting, pharmacokinetics and relative accumulation of the drug in different organs

7/25/18

Moleculin Biotech Inc., of Houston

WP-1066

Targeter of STAT3 pathway

Glioblastoma and brain metastases in adults

Opened enrollment for a physician-sponsored trial

7/31/18

Newlink Genetics Corp., of Ames, Iowa

Indoximod

Small molecule targeting the IDO pathway

Newly diagnosed diffuse intrinsic pontine glioma

Updated data in combination with front-line radiation and maintenance chemotherapy showed 10 of 10 patients demonstrated initial symptomatic improvement; 8 of 10 had completed radiation, with the remaining 2 continuing radiotherapy; 9 of 10 remain on study, with the longest time on study of 8.5 months

7/1/18

Oncolys Biopharma Inc., of Tokyo

Telomelysin (OBP-301)

Oncolytic virus

Esophageal cancer

In 13 patients treated with drug plus radiation, there were 8 complete responses and 3 partial responses

7/19/18

Onconano Medicine Inc., of Southlake, Texas

ONM-100 

Imaging agent targeting tumor acidosis

Tumors and metastatic lymph nodes in solid cancers during surgery

Started open-label study measuring safety, pharmacokinetics and imaging feasibility in dose-escalation and dose-expansion phases

7/11/18

Oncosec Medical Inc., of San Diego

Immunopulse tavokinogene telseplasmid

Plasmid encoded IL-12

Triple-negative breast cancer

A case study of 2 patients in the ongoing OMS-140 study showed systemic immune response in post-protocol treatment with immune checkpoint inhibitors

7/17/18

Provectus Pharmaceuticals Inc., of Knoxville, Tenn.

PV-10

Oncovirus

Multiple tumors

Its ongoing, multi-center, open-label, phase I trial of PV-10 for patients with hepatic lesions has expanded to include a single-site cohort of 10 uveal melanoma patients with hepatic metastases

7/31/18

Regeneus Ltd., of Sydney, Australia

RGSH4K

Cancer vaccine

Solid tumors

Reported positive results from a phase I ACTIVATE trial of its cancer vaccine, which met the primary endpoint of safety and tolerability; 12 patients, heavily pretreated with chemotherapy or radiotherapy, with various advanced solid tumors received RGSH4K in 3 dose cohorts; 3 vaccines were administered in the treatment phase, given at 3-week intervals, and patients had the option to continue dosing in an extension phase; all dose levels were safe and well tolerated, achieving the safety primary endpoint with no dose-limiting toxicities and no serious adverse events related to the vaccine

7/31/18

Rimo Therapeutics Inc., of Elk Grove Village, Ill.

RIMO-301

Nanoscale metal-organic framework

Advanced tumors

First patient enrolled in the open-label study designed to determine the maximum tolerated dose, pharmacokinetics and preliminary antitumor activity of the drug

7/16/18

Seattle Genetics Inc., of Bothell, Wash.

Tucatinib

HER2 inhibitor

Advanced HER2-positive breast cancer

Data published in The Lancet Oncology show tucatinib plus Herceptin and Xeloda produced a median progression-free survival of 7.8 months with an objective response rate of 61%; in a separate study testing tucatinib plus Kadcyla, which was published in JAMA Oncology, the combination produced a median PFS of 8.2 months and an ORR of 47%

7/11/18

Targovax ASA, of Oslo, Norway, and Sotio AS, of Prague

ONCOS-102 and DCVAC/PCa

Oncolytic virus and autologous dendritic cells

Advanced metastatic castration-resistant prostate cancer

Dosed first of up to 15 patients in the open-label SP015 study testing the safety and immune activation of the combination

7/12/18

Tolero Pharmaceuticals Inc., of Salt Lake City

TP-0184

ACVR1 inhibitor

Advanced solid tumors

Treated first patient in the open-label, dose-escalation study testing the safety, pharmacokinetics and pharmacodynamics of the drug, as well as measuring the initial antitumor activity

7/25/18

Xencor Inc., of Monrovia, Calif.

XmAb-20717

Bispecific antibody targeting PD-1 and CLTA-4

Advanced solid tumors

Dosed the first patient in a in the DUET-2, multiple-dose, dose-escalation study to evaluate safety and tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary antitumor activity

7/13/18

Cardiovascular

Foresee Pharmaceuticals Co. Ltd., of Taipei, Taiwan

FP-045

ALDH2 activator

Healthy volunteers (eventually peripheral arterial diseases and Fanconi anemia)

Drug was deemed safe and tolerable at all 3 doses tested in 24 subjects for 7 days  

7/26/18

Omeicos Therapeutics GmbH, of Berlin

OMT-28

18-EEQ analogue

Healthy volunteers (eventually atrial fibrillation)

Drug was safe and tolerable up to 60 mg, the maximum dose tested, without any clinically relevant effect on QT-prolongations, changes in the PR and QRS intervals or on cardiac conduction; plans to start a phase II study

7/17/18

Valbiotis SA, of Paris

VAL-070

Designed to reduce LDL cholesterol

Mild to moderate dyslipidemia

Results from a phase I/II pilot study showed the product was safe and well-tolerated; data on blood LDL-cholesterol support use of that parameter as the primary endpoint of the phase II development

7/10/18

Vivus Inc., of Campbell, Calif.

VI-0106

Soft capsule formulation of tacrolimus

Healthy volunteers (eventually pulmonary arterial hypertension)

Preliminary results from the 28-subject study showed drug has a favorable pharmacokinetic profile with an extended Tmax, a lowered Cmax and an increased area under the curve compared to immediate-release tacrolimus

7/10/18

Dermatologic

Blade Therapeutics Inc., of South San Francisco

BLD-2660

Dimeric calpain inhibitor

Healthy volunteers (eventually fibrosis)

Started study testing the safety and pharmacokinetics; data expected by the fourth quarter of 2018

7/17/18

Osiris Therapeutics Inc., of Columbia Md.

Grafixpl Prime

Membrane of cells processed via Prestige Lyotechnology

Chronic venous leg ulcer

Enrolled first of 200 patients in study measuring the proportion of patients with complete ulcer closure by week 12 after treatments with Grafixpl plus standard of care compared to standard of care alone; study design includes a crossover for the control group

7/11/18

Sienna Biopharmaceuticals Inc., of Westlake Village, Calif.

SNA-120 (pegcantratinib)

TrkA inhibitor

Pruritus associated with psoriasis

After topical application under maximal use conditions there was no accumulation of SNA-120 in the plasma of the 30 patients enrolled in the study; in a post hoc analysis, 57% of patients had at least a 4-grade improvement in the Itch Numeric Rating Scale

7/24/18

Endocrine/Metabolic

Armagen Inc., of Calabasas, Calif.

AGT-181

Enzyme replacement therapy

Mucopolysacc-haridosis type I

Data published in Orphanet Journal of Rare Diseases show AGT-181 stabilized neurocognitive development quotient and the cortical grey matter volume of patients' brains; the drug also stabilized or improved urinary glycosaminoglycan levels, hepatic and spleen volumes and shoulder range of motion

7/10/18

Codexis Inc., of Redwood City, Calif.

CDX-6114

Enzyme replacement

Healthy volunteers (eventually phenylketonuria)

Dosed first of up to 32 healthy volunteers in a dose-escalation study measuring safety, tolerability and pharmacology in up to 4 cohorts; results expected in the fourth quarter of 2018

7/9/18

Neurovia Inc., of San Francisco

NV-1205

Restores ability to process very long chain fatty acids

Childhood cerebral adrenoleukodystrophy

Started the open-label, dose-escalation study to assess the safety, tolerability and pharmacokinetics of the drug

7/25/18

Proclara Biosciences Inc., of Cambridge, Mass.

NPT-189

Targets LC and TTR deposits

Healthy volunteers (eventually systemic amyloidoses)

Dosed first subject in study testing the safety and tolerability of NPT-189

7/26/18

Sangamo Therapeutics Inc., of Richmond, Calif.

SB-318

Zinc finger nuclease delivering a corrective copy of IDUA

Mucopolysacc-haridosis type I

First patient treated in open-label Empowers study testing the safety, tolerability and preliminary efficacy of SB-318 in up to 9 adult patients

7/23/18

Ultragenyx Pharmaceutical Inc., of Novato, Calif.

DTX-401

Gene therapy expressing G6Pase-alpha

Glycogen storage disease type Ia

Dosed first patient in study measuring drug's safety, tolerability and therapeutic response, including time to hypoglycemia, impact on biomarkers such as lipids, uric acid and liver glycogen; data from first cohort of 3 patients expected in the second half of 2018

7/26/18

Gastrointestinal

Cohbar Inc., of Menlo Park, Calif.

CB4-211

Analogue of MOTS-c

Obese patients with nonalcoholic fatty liver diseases

Started study with part a testing safety, tolerability and pharmacokinetics of CB-4211 in healthy volunteers and part b testing patients' changes in liver fat, body weight and biomarkers for nonalcoholic steatohepatitis and obesity

7/12/18

Landos Biopharma Inc., of Blacksburg, Va.

BT-11

Activates LANCL2 pathway

Healthy volunteers (eventually inflammatory bowel disease)

Dosed first of 70 subjects in trial testing the safety and tolerability of drug

7/12/18

Poxel SA, of Lyon, France

PXL-770

AMPK activator

Healthy volunteers (eventually nonalcoholic steatohepatitis)

In the 48-subject multiple ascending-dose portion of the study, the drug was well-tolerated up to the highest dose tested of 500 mg, including no prolongation of the TQT interval; a drug-drug interaction study of 12 subjects taking 250 mg of PXL-770 and the standard dose of rosuvastatin showed a lack of pharmacokinetic interaction between the 2 drugs; company plans to run a phase IIa study in patients

7/18/18

Genitourinary/Sexual Function

Cytori Therapeutics Inc., of San Diego

Cytori Cell Therapy

Autologous adipose-derived regenerative cells

Erectile dysfunction

Data from trial published in Urology showed 21 patients with erectile dysfunction following a radical prostatectomy had an improvement in their International Index of Erectile Function-5 score from a median of 6 at baseline to 8 after 12 months (p=0.004); the subset of 15 patients with urinary continence at baseline improved scores from 6 to 13 (p=0.012); Erection Hardness Score didn't improve in overall population, but urinary continence group had a median score improvement from 1 at baseline to 2 at 12 months (p=0.03)

7/9/18

Forendo Pharma Oy, of Turku, Finland

FOR-6219

Inhibits the conversion of low potency estrone into highly potent estradiol in endometriotic tissues

Endometriosis

Gained clearance from the U.K.'s Medicines and Healthcare products Regulatory Agency to commence study 

7/4/18

Hematologic

Achillion Pharmaceuticals Inc., of New Haven, Conn.

ACH-5548

Complement factor D inhibitor

Healthy volunteers (considering paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy and immune complex-mediated membranoproliferative glomerulonephritis)

Dosed first of approximately 28 subjects, testing safety and tolerability as primary endpoints; pharmacokinetics, pharmacodynamics and alternative pathway inhibition will also be studied; data expected in the second half of 2018

7/10/18

Catalyst Biosciences Inc., of South San Francisco

CB-2679d

Factor IX

Hemophilia B

2 patients in cohort 6 developed neutralizing antibodies to the drug, which was transient in 1 patient and fell below the level of quantification at a follow-up visit; in the other patient, the neutralizing antibody level fell below 1 Bethesda Unit at a follow-up visit

7/18/18

Immune

Argenx NV, of Breda, the Netherlands

Efgartigimod (ARGX-113)

FcRn antagonist

Healthy volunteers (currently myasthenia gravis and pemphigus vulgaris)

Data published in Journal of Clinical Investigation show the drug had an acceptable tolerability profile and reduced total immunoglobulin G levels by approximately 75%

7/24/18

Aurigene Discovery Technologies Ltd., of Boston

AUR-101

Inverse agonist of the receptor RoRγt

IL17-driven immunological conditions

Began dosing in healthy volunteer trial to evaluate safety signals, pharmacodynamic modulation and recommended dosage for phase II; data expected in December 2018

7/11/18

Bio-Thera Solutions Ltd., of Guangzhou, China

BAT-1806

Biosimilar of Actemra (tocilizumab)

Healthy volunteers

Started dosing of a 130-subject study comparing the pharmacokinetics and safety of BAT-1806 to U.S.-sourced Actemra and EU-sourced Roactemra

7/26/18

Kymab Group Ltd., of Cambridge, U.K.

KY-1005

Monoclonal antibody targeting OX40L

Healthy volunteers (eventually atopic dermatitis and other immune-mediated diseases)

Drug had a favorable safety profile, was well-tolerated and blocked T-cell-driven inflammation in the skin of 64 subjects tested in the placebo-controlled study; plans to start a phase IIa study in atopic dermatitis in late 2018

7/30/18

Principia Biopharma Inc., of South San Francisco

PRN-2246

BTK inhibitor

Multiple sclerosis

Dosing completed; candidate achieved full peripheral BTK occupancy at clinically relevant dose levels; cerebral spinal fluid exposure was confirmed

7/27/18

Infection

Appili Therapeutics Inc., of Halifax, Nova Scotia

ATI-1501

Taste-masked formulation of antibiotic metronidazole

Healthy volunteers (eventually bacterial infections)

In the 44-subject study, a 500-mg dose produced equivalent systemic drug levels to a 500-mg Flagyl (metronidazole) tablet under fasted and fed conditions; a subset of 25 subjects who were asked their palatability and preference of the 2 drugs preferred ATI-1501 over metronidazole crushed in applesauce

7/30/18

Contravir Pharmaceuticals Inc., of Edison, N.J.

CRV-431

Analogue of cyclosporine A

Healthy volunteers and hepatitis B virus infection

Completed dosing of first cohort in part 1 of trial testing drug in healthy volunteers; pharmacokinetic profile was in line with anticipated exposures based on preclinical data

7/23/18

Destiny Pharma plc, of Brighton, U.K.

XF-73

Antimicrobial

Healthy volunteers (eventually postsurgical staphylococcal infections)

In a dermal irritation study, XF-73 had similar irritancy potential to water; drug wasn't observed in blood samples

7/26/18

Hemispherx Biopharma Inc., of Orlando, Fla.

AMP-600

Ampligen combined with Flumist

Influenza prophylaxis

Data show no evidence of increase in adverse effects due to increase in Ampligen dose or after repeat vaccinations; treatment generated antibodies against influenza subtypes not present in seasonal vaccine

7/9/18

Johnson & Johnson, of New Brunswick, N.J.

HIV vaccine

Investigational mosaic-based preventive vaccine regimen

HIV 1

Announced the first long-term immune response data for this mosaic-based preventive vaccine regimen; a robust HIV antibody response was maintained in all healthy volunteers who received the lead vaccine regimen at 96 weeks, one year after the last vaccination in the Approach study which includes 393 healthy HIV-uninfected adults; no unexpected vaccine-related adverse events were reported

7/24/18

Spero Therapeutics Inc., of Cambridge, Mass.

SPR-994

Carbapenem-class antibiotic

Healthy volunteers (eventually gram-negative infections)

Interim analysis of data shows drug was well-tolerated at doses from 100 mg to 900 mg daily with linear pharmacokinetics over the range; active drug metabolite stayed above the minimum inhibitory concentration to decrease growth for 90% of the pathogen for more than 50% of an 8-hour dosing interval; final data expected in the third quarter of 2018; plans to start phase III around year-end 2018

7/9/18

Vaxart Inc., of South San Francisco

Norovirus vaccine

Oral vaccine tablet

Norovirus

Data showed the high-dose group met the primary endpoint, an increase in serum blocking antibody titers, with 78% of subjects showing >/= 2-fold rise after single immunization (p=0.0003); more than 80% of recipients of the high-dose vaccine developed mucosally primed norovirus-specific circulating antibody secreting cells, IgA-positive memory B cells expressing the alpha3beta7 gut homing receptor, and fecal IgA

7/12/18

Musculoskeletal

Asterias Biotherapeutics Inc., of Fremont, Calif.

ASTOPC-1

Oligodendrocyte progenitor cell population derived from human embryonic stem cells

Severe cervical spinal cord injury

Cohorts 3 and 4 in the Scistar study reported no adverse events and 92% (11/12) of subjects with magnetic resonance imaging scans at 12 months consistent with the formation of a tissue matrix at the injury site; at 12 months, 100% (6/6) of cohort 3 subjects have recovered at least one motor level on at least one side, with one subject having recovered two motor levels on one side; at 12 months, 83% (5/6) of cohort 4 subjects have recovered at least one motor level on at least one side, with one subject having recovered two motor levels on one side

7/31/18

Sarepta Therapeutics Inc., of Cambridge, Mass.

Microdys-trophin gene therapy

Gene therapy

Duchenne muscular dystrophy

FDA placed on clinical hold a phase I/IIa trial at the Research Institute at Nationwide Children's Hospital citing the presence of a trace amount of DNA fragment in research-grade third-party-supplied plasmid; in vivo testing performed by the institute showed trace fragment does not result in protein expression and is quickly cleared; an action plan will be submitted to the FDA for review, with no material delay anticipated in dosing patients, originally planned by year-end 2018

7/26/18

Neurology/Psychiatric

Aeromics Inc., of Branford, Conn.

AER-271

AQP4 inhibitor

Healthy volunteers (eventually severe ischemic stroke)

Dosed first cohort of patients in study testing the pharmacokinetics, safety and tolerability of AER-271 in single ascending and multiple ascending doses; data expected in May 2019

7/9/18

Asterias Biotherapeutics Inc., of Fremont, Calif.

AST-OPC1

Oligodendrocyte progenitor cells from human embryonic stem cells

Severe cervical spinal cord injury

6-month follow-up shows 100% cell engraftment in the 4 patients in cohort 5 treated with 20 million cells; all 4 patients had at least 1 motor level improvement on at least 1 side with 2 patients having 1 motor level improvement bilaterally

7/17/18

Corium International Inc., of Menlo Park, Calif.

Corplex Donepezil

Cholinesterase inhibitor delivered via transdermal patch

Alzheimer's disease

Pharmacokinetic and pharmacodynamic results from the bioequivalence study showed equivalence to Aricept (donepezil) characterized by the relationship between plasma donepezil concentrations and red blood cell acetylcholinesterase inhibition

7/25/18

Proclara Biosciences Inc., of Cambridge, Mass.

NPT-088 

Active fragment of g3p and human-IgG1-Fc

Mild to moderate Alzheimer's disease

Completed enrollment in study testing the safety and tolerability of NPT-088 as well as disease pathology via amyloid beta and tau PET imaging; top-line data expected in first quarter of 2019

7/26/18

Purdue Pharma LP, of Stamford, Conn.

Potential first-in-class small molecule

Undisclosed

Insomnia associated with alcohol cessation

Proof of concept study planned

7/31/18

Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan

IPSC-derived dopaminergic progenitors

Dopamine progenitor cells generated from induced pluripotent stem cells

Parkinson's disease

Kyoto University Hospital is starting a physician-initiated study to test the safety and efficacy of transplanting the cells into the bilateral putamen of 7 patients

7/30/18

Titan Pharmaceuticals Inc., of San Francisco

Ropinirole implant

Dopamine agonist delivered using Proneura technology

Idiopathic Parkinson's disease

Independent data safety monitoring board for phase I/II study completed review of data from the first cohort and recommended the trial continue with enrollment of the second cohort; however, Titan will postpone further enrollment until resources allow

7/3/18

Zynerba Pharmaceuticals Inc., of Devon, Pa.

ZYN-001

Prodrug of tetrahydrocannabinol

Neuropsychiatric disorders including Tourette syndrome

Top-line results indicate that target blood levels of 5 to 15 ng/ml THC were not achieved

7/5/18

Ocular

Applied Genetic Technologies Corp., of Gainesville, Fla.

ACHM-CNGB3

AAV-based gene therapy expressing the CNGB3 protein

Achromatopsia

Enrolled the seventh patient; expects to complete dose escalation in the first quarter of 2019

7/23/18

Applied Genetic Technologies Corp., of Gainesville, Fla., and Biogen Inc., of Cambridge, Mass.

XLRP 

AAV-based gene therapy expressing the RS1 protein

X-linked retinitis pigmentosa

Enrolled first patient in second cohort; dose escalation expected to be completed in the first quarter of 2019

7/23/18

Horama SA, of Paris

HORA-PDE6B

Gene therapy expressing PDE6B

Retinitis pigmentosa

First of 12 patients enrolled in the open-label, dose-escalation study

7/11/18

Opthea Ltd., of Melbourne, Australia

OPT-302

VEGFR-3

Diabetic macular edema

OPT-302 administered in combination with Eylea (aflibercept) to 9 patients was well-tolerated at all 3 doses

7/26/18

Thrombogenics NV, of Leuven, Belgium

THR-317

Recombinant humanized monoclonal antibody directed against receptor-binding site of PIGF

Diabetic macular edema

Day 150 top-line results from the phase I/II trial met the primary endpoint of safety for both 4-mg and 8-mg doses; overall, patients receiving the 8-mg dose achieved better visual acuity outcomes, with initial data from day 90, 30 days after last injection, showing 30% achieved a 15-letter or better vision gain vs. baseline; at day 150, 30% of patients in the 8-mg group showed 10-letter or better vision gain and 10% showed a 15-letter or better vision gain

7/19/18

Other/Miscellaneous

Glenmark Pharmaceuticals Ltd., of Mahwah, N.J.

GBR-310

Biosimilar to Xolair (omalizumab)

Healthy volunteers (eventually allergic asthma and chronic idiopathic urticaria)

The 168-subject study showed drug and reference product had similar pharmacokinetic, pharmacodynamic, safety and immunogenicity  

7/25/18

Histogen Inc., of San Diego

Hair stimulating complex (HSC-660)

Soluble formulation of subset of naturally secreted growth factors

Female diffuse hair loss

Active recruitment and enrollment of trial participants is underway

7/2/18

Respiratory

Bellus Health Inc., of Laval, Quebec

BLU-5937

Antagonist of P2X3 receptor

Healthy volunteers (eventually chronic cough)

Dosed first of up to 90 healthy adults testing the safety, tolerability and pharmacokinetics of the drug compared to placebo; part 1 tests single ascending doses in up to 6 cohorts of 10 subjects while part 2 will test multiple ascending doses in up to 3 cohorts of 10 subjects; data expected in the fourth quarter of 2018

7/9/18

Pulmatrix Inc., of Lexington, Mass.

Pulmazole 

Isperse formulation of antifungal itraconazole

Allergic bronchopulmonary aspergillosis in patients with asthma

In part 3 of study, drug was safe and well-tolerated with approximately 50-fold higher lung exposure and approximately 85-fold lower systemic exposure compared to oral Sporanox (itraconazole) with 1/10th the dose; plans to start a phase II study in fourth quarter of 2018

7/17/18

Toxicity and Intoxication

Omeros Corp., of Seattle

OMS-527 

PDE7 inhibitor

Healthy volunteers (eventually nicotine addiction)

First cohort of patients dosed in the single ascending- and multiple ascending-dose trial that is expected to be completed in the first half of 2019

7/17/18

Notes

For more information about individual companies and/or products, see Cortellis.