Company (location)

Product

Description

Indication

Status

Date

Cancer

ADC Therapeutics SA, of Lausanne, Switzerland

ADCT-602

Antibody-drug conjugate incorporating PBD drug linker and targeting CD22

Relapsed or refractory B-cell acute lymphoblastic leukemia

First patients dosed in a phase I/II trial testing safety, tolerability, pharmacokinetics and antitumor activity

11/27/18

Aduro Biotech Inc., of Berkeley, Calif.

ADUS-100

STING pathway activator

Advanced solid tumors or lymphomas

Well-tolerated with no dose-limiting toxicities reported; preliminary signs of clinical and biomarker activity observed in patients with advanced cancers, including those pretreated with checkpoint inhibitor therapy

11/9/18

Agios Pharmaceuticals Inc., of Cambridge, Mass.

AG-881

Inhibitor of IDH1 and IDH2

Advanced glioma

In 52 treated patients, 1 patient with enhancing disease and a 1p19q co-deletion had a confirmed, sustained partial response; another patient with enhancing disease and a 1p19q co-deletion had a confirmed, sustained minor response; 69% of patients had stable disease, including 82% of the 18 patients with non-enhancing disease who had an average volumetric six-month tumor growth of 6.8%

11/16/18

Alx Oncology Inc., of Burlingame, Calif.

ALX-148

Fusion protein comprising engineered high affinity CD47 binding domain of SIRPα linked to inactive Fc region of human immunoglobulin

Solid tumors

Generally well-tolerated in combination and no maximum tolerated dose was reached

11/7/18

Aptose Biosciences Inc., of San Diego

APTO-253

Inhibits expression of the MYC oncogene

Relapsed or refractory hematologic malignancies

Dosed first patient in re-initiation of study

11/26/18

Aravive Inc., of Houston

AVB-S6-500

Soluble Fc-fusion protein

Platinum-resistant recurrent ovarian cancer

No serious adverse events and AVB-S6-500 was well-tolerated across all doses; serum GAS6 (sGAS6) levels were suppressed for 22 and 29 days following single doses of 5 mg/kg and 10 mg/kg, respectively; weekly administration of 5 mg/kg resulted in suppression of sGAS6 in 4 out of 6 subjects for at least 3 weeks after the fourth dose

11/13/18

Arcus Biosciences Inc., of Hayward, Calif.

AB-122

Fully human IgG4 antibody that blocks interaction of PD-1 with its ligands, PD-L1 and PD-L2

Various tumor types

Drug was well-tolerated at all doses; data from the 3 patients in 80-mg once every 2 weeks and 6 patients in the 240-mg once every 2 weeks cohorts showed AB-122 achieved full and sustained receptor occupancy on peripheral blood T cells across all time points in the majority of patients

11/9/18

Arqule Inc., of Burlington, Mass.

ARQ-751-101

Inhibitor of the AKT serine/threonine kinase

Solid tumors

Presented data from dose-escalation study in adult patients with advanced solid tumors with AKT1, 2, 3 genetic alterations, activating PI3K mutations, PTEN-null, or other known actionable PTEN mutations; product demonstrated manageable toxicity at doses from 5 mg QD to 75 mg QD, and the recommended phase II dose was determined to be 75 mg QD

11/19/18

Asterias Biotherapeutics Inc., of Fremont, Calif.

VAC2

Immunotherapy containing mature dendritic cells derived from pluripotent stem cells

Non-small-cell lung cancer

Enrolled and dosed the fourth subject in trial sponsored by Cancer Research UK

11/27/18

Athenex Inc., of Buffalo, N.Y.

Oraxol

Formulation of paclitaxel combined with HM-30181A, a gastrointestinal tract-specific P-glycoprotein pump inhibitor

Advanced solid malignancies

Study begins using drug in combination with Keytruda (pembrolizumab, Merck & Co. Inc.)

11/26/18

Bavarian Nordic A/S, of Copenhagen, Denmark

CV-301

Targeted immunotherapy

Metastatic colorectal or pancreatic cancer

First patient dosed in combination with durvalumab, Astrazeneca plc's PD-L1 inhibitor, in combination with maintenance chemotherapy

11/2/18

Beigene Ltd., of Beijing, China

Pamiparib

PARP inhibitor

Glioblastoma multiforme

Among 15 evaluable newly diagnosed patients with unmethylated MGMT promoter status treated with pamiparib and radiation therapy, the disease control rate was 53.3%, including 2 partial response and 6 patients with stable disease; among the 10 recurrent/refractory patients treated with pamiparib and temozolomide, there were 2 partial responses and 3 patients with stable disease

11/16/18

Blueprint Medicines Corp., of Cambridge, Mass.

Avapritinib

KIT and PDGFRA inhibitor

Advanced gastrointestinal tumors

In PDGFRalpha D842V gastrointestinal tumors (GIST), the drug produced an 84% objective response rate; in fourth-line or later GIST, ORR was 20%; in regorafenib-naïve third- and fourth-line GIST, ORR was 25%; in second-line GIST, ORR was 25%

11/15/18

Celldex Therapeutics Inc., of Hampton, N.J.

CDX-1140

Fully human agonist anti-CD40 antibody

Solid tumors

17 patients enrolled; dose-dependent biological effects consistent with CD40-mediated immune activation observed and no maximum tolerated dose (MTD) identified to date; continued enrollment is ongoing to define the MTD and select a dose for disease-specific expansion cohorts that will be monitored for clinical activity

11/9/18

Celyad SA, of Mont-Saint-Guibert, Belgium

CYAD-101

Non-gene edited allogeneic CAR-T therapy

Unresectable metastatic colorectal cancer

First patient treated; drug administered concurrently with FOLFOX chemotherapy

11/30/18

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China

CS-1003

Monoclonal antibody

Advanced cancers

First patient enrolled and dosed in trial assessing the safety, tolerability and preliminary antitumor activity in Chinese patients

11/19/18

Cytomx Therapeutics Inc., of South San Francisco

CX-072

Probody therapeutic targeting PD-L1

Metastatic or locally advanced unresectable solid tumors or lymphomas

Protease activity was detected in the majority of patient biopsy samples (15 of 18, or 83%); CX-072 was cleaved and activated within tumors, with the total amount of activated CX-072 increasing with dose

11/9/18

Dnatrix Inc., of Houston

DNX-2440

Oncolytic virus expressing OX40 ligand

Recurrent glioblastoma

Treated first patient; study is evaluating safety and efficacy of DNX-2440, when administered at the time of biopsy, to patients for whom surgery is not possible or planned

11/26/18

Five Prime Therapeutics Inc., of South San Francisco

FPT-155

CD80 fusion protein

Advanced solid tumors

First patient dosed in the study that will measure safety as well as the pharmacokinetic and pharmacodynamic profile of the drug to determine best dose to test in phase Ib portion, which will further characterize the drug's profile as well as assess efficacy

11/14/18

Flx Bio Inc., of South San Francisco

FLX-475

CCR4 antagonist

Various tumors

Established clinical trial collaboration with Merck & Co. Inc. to conduct a phase I/II study evaluating safety and efficacy of Keytruda (pembrolizumab) and FLX-475; study will evaluate changes in the tumor microenvironment and antitumor activity of both monotherapy and combination therapy

11/5/18

Flx Bio Inc., of South San Francisco

FLX-475

Antagonist of CCR4

Healthy volunteers (eventually cancer)

Drug was well-tolerated at all doses, including the level projected to achieve or exceed maximal inhibition of human T-reg; drug's half-life and oral absorption support once-daily dosing; enrolling a phase I/II study in multiple types of cancer, testing drug as a monotherapy and in combination with Keytruda (pembrolizumab, Merck & Co. Inc.)

11/8/18

Immutep Ltd., of Sydney

Eftilagimod alpha (IMP-321)

Soluble LAG-3Ig fusion protein and APC activator

Unresectable or metastatic melanoma

Data from ongoing TACTI-mel trial in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in 24 patients show after 3 months, 3 of 6 patients experienced a partial response, a 50% overall response rate according to immune-related response criteria; current disease control rate for that group is 66%; no dose-limiting toxicity or new safety signal observed when combination is administered beginning cycle 1, day 1 of pembrolizumab treatment

11/27/18

IMV Inc., of Dartmouth, Nova Scotia (formerly Immunovaccine Inc.)

DPX-Survivac

T cell-activating immunotherapy

Recurrent ovarian cancer

Amended phase Ib/II trial after review of phase Ib portion showed efficacy signals in subpopulation of patients who received DPX-Survivac in combination with 100-mg dose epacadostat (n=5), including 100% tumor regressions and 100% disease control rate, with 60% of those patients (3/5) reaching best response of a partial response; based on 300-mg epacadostat cohort results, agreed to stop dosing patients with epacadostat; IMV will continue trial as a monotherapy study of DPX-Survivac in recurrent ovarian cancer subpopulation

11/20/18

Innovent Biologics Inc., of Suzhou, China

Sintilimab

Fully human PD-1 monoclonal antibody

First-line nonsquamous non-small-cell lung cancer

In combination with pemetrexed and cisplatin, treatment demonstrated objective response rate of 68.4%, based on data from 19 patients with at least 1 radiological assessment among the 21 patients in that cohort

11/9/18

Intensity Therapeutics Inc., of Westport, Conn.

INT-230-6

2 cancer agents and a penetration enhancer molecule

Advanced solid tumors

Intratumoral injections of drug were well-tolerated with no dose-limiting toxicity; a patient with cutaneous squamous cell carcinoma had increased tumor necrosis following treatment; a patient with a chordoma had tumor reduction, including reductions of some non-injected lesions

11/8/18

Ipsen SA, of Paris, and 3B Pharmaceuticals GmbH, of Berlin

Lu-IPN01087 (formerly 3BP-227)

Radionuclide targeting cancer cells expressing NTSR1

Advanced solid tumors

First patient dosed in dose-escalation trial to evaluate safety and activity, as well as identify optimum systemically administered dose of radiation to treat patients with solid tumors – pancreatic ductal adenocarcinoma, colorectal cancer, gastric cancer, gastrointestinal stromal tumors, Ewing sarcoma and squamous cell carcinoma of the head and neck – expressing NTSR1

11/20/18

Lytix Biopharma AS, of Oslo, Norway

LTX-315

Oncolytic peptide

Solid tumors

Study demonstrated that drug is safe to administer with manageable toxicities and with no added safety concerns when given in combination, either with ipilimumab or pembrolizumab

11/21/18

Merck & Co. Inc.

MK-4280

Anti-LAG-3 therapy

Advanced solid tumors

Preliminary findings demonstrated acceptable safety profiles with no dose-limiting toxicities and early signals of antitumor activity 

11/7/18

Merck & Co. Inc., of Kenilworth, N.J.

Keytruda (pembrolizumab)

PD-1-targeting antibody

Metastatic non-small-cell lung cancer, metastatic melanoma, metastatic urothelial carcinoma

In combination with Lenvima (lenvatinib, Eisai Co. Ltd.), interim analyses support further evaluation

11/9/18

Mirati Therapeutics Inc., of San Diego

Sitravatinib

Spectrum-selective kinase inhibitor that inhibits receptor tyrosine kinases, including TAM family receptors (TYRO3, Axl, Mer), split family receptors (VEGFR2, KIT) and RET

Advanced solid tumors

Used in combination with anti-PD1 antibody tislelizumab (Beigene Ltd.); first patient dosed

11/9/18

Moleculin Biotech Inc., of Houston

WP-1066

STAT3 inhibitor

Cancer

Preliminary results demonstrate drug bioavailability in ongoing trial

11/1/18

Nantkwest Inc., of Culver City, Calif.

Cancer Memory Vaccine

CD16-targeted, off-the-shelf natural killer cells

Refractory advanced metastatic cancer

In patients with advanced metastatic triple-negative breast cancer at fourth-line or greater, disease control rate was 80%; third-line or greater advanced metastatic pancreatic cancer patients had a 90% disease control rate with a median overall survival of 9.5 months, compared to 8.7 months for historical controls treated with first-line therapy; in fourth-line or greater advanced metastatic head and neck cancer, disease control rate was 67%, including 1 patient with a complete response; 86% of patients with refractory bladder cancer with carcinoma in-situ had a complete response, while 100% of patients with papillary carcinoma remain disease free

11/7/18

Neon Therapeutics Inc., of Cambridge, Mass.

NEO-PV-01

Neoantigen vaccine

Metastatic cancer

Vignettes of patients treated with NEO-PV-01 and Opdivo (nivolumab, Bristol-Myers Squibb Co.) demonstrate immunological changes in the blood and tumor that correlate with clinical responses

11/9/18

Newlink Genetics Corp., of Ames, Iowa

NLG-802

Prodrug of indoximod

Recurrent advanced solid malignancies

Well-tolerated; neither maximum tolerated dose nor maximum biologically achievable dose reached; produced 4-fold increase in CMAX and AUC after a single dose, and 4-5.5-fold increases in CMAX and AUC after continuous twice-daily dosing compared with the molar equivalent of indoximod dosing

11/9/18

Novo Nanovector ASA, of Oslo, Norway

Betalutin (177Lu-sate-traxetan-lilotomab)

Antibody-radionuclide conjugate

Second-line follicular lymphoma

First patient dosed in combination with rituximab (Rituxan, Roche Holding AG and Biogen Inc.)

11/2/18

Noxopharm Ltd., of Sydney

Veyonda (idronoxil)

Kinase inhibitor

Solid tumors

During phase Ib, Veyonda was well-tolerated as a monotherapy, with just 1 case of anemia; combination with low-dose carboplatin suspended tumor growth, or better, for at least 6 months in solid tumors in nearly half of patients considered unlikely to respond to further chemo; 83% of patients experienced 1 or more AEs, mostly on combo

11/29/18

Noxopharm Ltd., of Sydney

NOX-66

Formulation of idronoxil

Castrate-resistant prostate cancer

Data from the first 3 cohorts in the ongoing phase Ib DARRT-1 study in combination with palliative external beam radiotherapy show 2 of 3 patients in cohort 1 had stable disease at 12 weeks, 1 patient assessed in cohort 2 had a partial response at 12 week that continued at 24 weeks, and 1 patient in cohort 3 continues to have a partial response at 12 weeks with strong decreasing PSA response

11/13/18

Noxxon Pharma NV, of Berlin

NOX-A12

Olaptesed pegol

Brain tumors

Company is planning a dose-escalation trial of NOX-A12 plus radiotherapy in primary brain cancer patients who would not benefit medically from standard-of-care chemotherapy; aim is to establish safety and tolerability and define recommended phase II dose in combination with radiotherapy as well as assess signals of efficacy including changes in tumor vascularization, progression-free and overall survival

11/21/18

Oncomed Pharmaceuticals Inc., of Redwood City, Calif.

Etigilimab

Anti-TIGIT antibody

Late-stage metastatic cancers

No dose-limiting toxicities; stable disease was observed in 7 (38.9%) patients with prolonged disease control seen in some patients; longest durations of stable disease being 205 and 225 days

11/9/18

Onxeo SA, of Paris

AsiDNA

DNA damage response inhibitor

Solid tumors

Interim results from first 3 dose levels evaluated out of 6 planned in the DRIIV-1 study showed 10 patients received 112 infusions of AsiDNA ranging from 200 mg (DL1) to 600 mg (DL3); administration of DL4 (900 mg) is ongoing and the full dataset is expected in the first half of next year

11/5/18

Purdue Pharma LP, of Stamford, Conn.

Tinosta-mustine

Alkylating deacetylase inhibitor 

Newly diagnosed unmethylated O6-Methyl-guanine-DNA-methyl-transferase glioblastoma multiforme

First patient enrolled

11/6/18

Redhill Biopharma Ltd., of Tel-Aviv, Israel

Yeliva (opaganib)

Sphingosine kinase 2 inhibitor

Multiple myeloma

Results from open-label, dose-escalation phase Ib study in refractory or relapsed MM did not show any dose-limiting toxicities; out of 10 evaluable subjects, 2 had stable disease for more than 4 months and 1 patient achieved a very good partial response

11/14/18

Seattle Genetics Inc., of Bothell, Wash.

SEA-BMCA

Antibody targeting B-cell maturation antigen

Multiple myeloma (MM)

Dosed first patient in open-label, multicenter, dose-escalation and expansion trial evaluating safety and tolerability in patients with relapsed or refractory MM

11/14/18

Sensei Biotherapeutics Inc., of Gaithersburg, Md.

SNS-301

Immunotherapy targeting human aspartate β-hydroxylase 

Cancer

Data showed rapid and significant antigen-specific B-cell and T-cell responses 

11/6/18

Sorrento Therapeutics Inc., of San Diego

CD38 CAR T therapy

Cancer immunotherapy

Multiple myeloma

First patients were dosed

11/1/18

Sorrento Therapeutics Inc., of San Diego

Anti-CEA CAR T

Autologous anti-CEA CAR T cells

Liver metastases

With pressure-enabled drug delivery, 2 of 4 patients showed complete resolution of liver metastases on PET scan, and median overall survival (OS) was 8.3 months with mean OS of 9.8 months compared to standard of care's 3 to 6 months

11/6/18

Spirita Oncology LLC, of Boston

E-6201

MEK1 inhibitor

BRAF- or MEK-mutated metastatic melanoma

Started trial in patients with central nervous system metastases

11/26/18

Sun Biopharma Inc., of Minneapolis

SBP-101

Polyamine

Pancreatic ductal adenocarcinoma

Phase Ia/Ib combination of SBP-101 with gemcitabine and nab-paclitaxel in patients previously untreated for metastatic pancreatic ductal adenocarcinoma (PDA), continued to enroll patients. The phase Ia portion will treat up to 18 PDA patients in three cohorts to determine a recommended dose

11/5/18

TG Therapeutics Inc., of New York

TG-1701

BTK inhibitor

Relapsed or refractory B-cell malignancies

First cohort evaluating 100 mg once daily has been fully enrolled, and the first patient enrolled, a patient with relapsed/refractory mantle cell lymphoma, achieved a partial response at the first efficacy assessment; the remaining 2 patients are too early to evaluate 

11/13/18

Trisalus Life Sciences Inc., of Westminster, Colo.

Pressure-Enabled Drug Delivery

Improves drug delivery to tumor by creating a favorable pressure gradient that penetrates tumor microenvironment and increases drug concentration in tumor without increasing systemic toxicity

Solid tumors

Median overall survival (OS) of 8.3 months and mean OS of 9.8 months vs. 3 to 6 months standard of care; 2 of 4 stage IV patients who failed systemic chemotherapy showed metabolic complete response with no visible liver metastases on positron emission tomography

11/7/18

Ziopharm Oncology Inc., of Boston

Ad-RTS-hIL-12 plus veledimex

Gene therapy to control expression of interleukin 12

Recurrent glioblastoma

The 6 patients who received 20 mg or less of dexamethasone cumulatively over 15 days along with the gene therapy had median overall survival of 17.8 months compared to 6.4 months for 9 patients who received more than 20 mg of dexamethasone along with the gene therapy

11/16/18

Zymeworks Inc., of Vancouver, British, Columbia

ZW-25

Bispecific antibody

Cancer

Induced antitumor activity and was well-tolerated in heavily pretreated patients with a variety of HER2-expressing cancers; overall disease control rate, which includes patients with partial responses and stable disease was 82%

11/14/18

Cardiovascular

Acesion Pharma, of Copenhagen

AP-30663

SK channel inhibitor

Atrial fibrillation

I.V. AP-30663 was well-tolerated with ascending doses, with no significant adverse events in safety and tolerability study in 47 healthy subjects

11/5/18

Dermatologic

Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C.

BCX-7353 

Plasma kallikrein inhibitor

Hereditary angioedema

After a single 750 mg oral dose, mean drug concentrations were approximately 16x EC50 within 30 minutes, and remained at or above that level through at least 24 hours post-dose

11/16/18

Evelo Biosciences Inc., of Cambridge, Mass.

EDP-1815

Anti-inflammatory monoclonal microbial therapy 

Psoriasis and atopic dermatitis

First patient dosed

11/26/18

Endocrine/Metabolic

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Givosiran

RNAi therapeutic targeting aminolevulinic acid synthase 1

Acute hepatic porphyria

As of data cut-off at June 7, a robust treatment effect was maintained in treated patients with continued dosing in the open-label extension (OLE) study (n=16), with a mean time on treatment of 13.6 months and total time on treatment across the phase I and OLE studies of up to 25 months

11/9/18

Codexis Inc., of Redwood City, Calif.

CDX-6114

Orally administered enzyme

Healthy volunteers (eventually phenylketonuria)

Drug met all safety and tolerability endpoints at all 4 doses tested; dose-dependent pharmacodynamic response was observed, and there was no evidence of systemic exposure

11/8/18

Imcyse SA, of Liège, Belgium

Imotopes

Immunotherapy

Type 1 diabetes

Enrolled 41 patients with recent-onset type 1 diabetes into phase 1b study, which will test safety, immunological responses and clinical impact of Imotopes technology

11/5/18

Poxel SA, of Lyon, France

PXL-065

Deuterium-stabilized R-stereoisomer of pioglitazone

Nonalcoholic steatohepatitis 

Part 2 of study begun

11/26/18

Regenxbio Inc., of Rockville, Md.

RGX-121 

Gene therapy expressing IDS

Mucopoly-saccharidosis type II

Dosed first patient in study

11/7/18

Gastrointestinal

Bridge Biotherapeutics Inc., of Seongnam, South Korea

BBT-401

Pellino-1 inhibitor

Healthy subjects (eventually ulcerative colitis)

Drug was safe and well-tolerated; no or minimal systemic exposure was observed; plans to start a phase II study within this year

11/12/18

Cohbar Inc., of Menlo Park, Calif.

CB-4211

Mitochondria-based therapeutic

Nonalcoholic steatohepatitis and obesity

Study has been temporarily suspended in order to address mild injection site reactions that have been unexpectedly persistent

11/5/18

Palatin Technologies Inc., of Cranbury, N.J.

PL-8177

Melanocortin receptor 1 agonist

Ulcerative colitis

Subcutaneous doses demonstrated favorable pharmacokinetics and pharmacodynamic properties and no safety or tolerability concerns; data from a study investigating oral formulation expected by year-end

11/8/18

Protagonist Therapeutics Inc., of Newark, Calif.

PTG-200

Oral peptide interleukin-23 receptor antagonist

Crohn's disease

Results of the randomized, double-blind, placebo-controlled, single- and multiple-dose escalation study demonstrated that administration of PTG-200 was well-tolerated

11/6/18

Vedanta Biosciences Inc., of Cambridge, Mass.

VE-202

Oral, live biotherapeutic candidate

Inflammatory bowel disease

Started study in healthy volunteers; trial conducted by Janssen Research & Development LLC

11/27/18

Genitourinary/Sexual Health

Dare Bioscience Inc., of San Diego

Sildenafil cream 3.6%

PDE4 inhibitor

Female sexual arousal disorder

Enrolling patients in study designed to evaluate feasibility of using thermography technology to assess pharmacodynamics in normal healthy women; genital temperature, a surrogate for genital blood flow, will be captured and recorded utilizing an infrared camera capable of detecting heat patterns from blood flow in body tissues

11/27/18

Visterra Inc., of Waltham, Mass.

VIS-649

Monoclonal antibody targeting APRIL

Healthy volunteers (eventually immunoglobulin A nephropathy)

Started study in up to 45 subjects, measuring safety, pharmacokinetics and pharmacodynamics at up to 5 doses

11/8/18

Immune

Atara Biotherapeutics Inc., of South San Francisco

ATA-190

Autologous EBV-specific T-cell immunotherapy

Multiple sclerosis

Results published online in JCI Insight describe findings observed in 10 patients, 5 with primary and 5 with secondary progressive MS; safety and efficacy were monitored for up to 27 weeks and compound was well-tolerated and no severe adverse events were observed; 6 participants who received ATA-190 with strong EBV reactivity experienced clinical improvement

11/19/18

Napajen Pharma Co. Ltd., of Tokyo

NJA-730

Anti-CD-40 siRNA

Healthy volunteers (eventually graft-vs.-host disease)

Dosed first of 80 subjects in the single ascending dose and multiple ascending dose study measuring the safety, tolerability and pharmacokinetics of the drug, as well as CD-40 gene suppression

11/15/18

Sanford Burnham Prebys Medical Discovery Institute, of La Jolla, Calif.

LY3361237

Biologic

Immunological disorders

First healthy subject has been dosed in evaluating LY3361237, a biologic that inhibits inflammation by activating an immune checkpoint receptor

11/15/18

Tiziana Life Sciences plc, of London

Foralumab

Intranasal formulation of anti-CD3 monoclonal antibody

Autoimmune and anti-inflammatory diseases

Launched first-in-human study in healthy volunteers

11/28/18

Infection

Aridis Pharmaceuticals Inc., of San Jose, Calif.

AR-301

Fully human monoclonal IgG1 antibody

Severe pneumonia caused by Staphylococcus aureus

Results in 48 patients show the drug was well-tolerated as adjunctive therapy; data published in The Lancet Respiratory Medicine

11/7/18

Arrowhead Pharmaceuticals Inc., of Pasadena, Calif.

ARO-HBV

Subcutaneously administered RNAi candidate

Hepatitis B virus

Results from phase I/II AROHBV1001 study in normal healthy volunteers show drug was well-tolerated, with HBsAg reductions similar in HBeAG-positive and negative patients and in NUC-naïve and NUC-experienced patients; mean HBsAg reduction on day 57 for cohort 8 (n=4) was -1.7 log10 and for cohort 9 (n=4) was -1.9 log10; responses observed were consistent with ARO-HBV's ability to silence HBV mRNA from cccDNA and host-integrated viral DNA

11/9/18

Atea Pharmaceuticals Inc., of Boston

AT-527

Salt form of a purine nucleotide prodrug NS5B polymerase inhibitor

Chronic hepatitis C

Showed profound and equivalent pan-genotypic antiviral activity in cirrhotic and noncirrhotic infected patients

11/9/18

Blueberry Therapeutics Ltd., of Alderley Edge, U.K.

BB-2603

Squalene epoxidase inhibitor

Onychomycosis and concomitant tinea pedis

Study met all its primary and secondary endpoints to assess pharmacokinetic, safety and local tolerability; topical nano-formulation was shown to have an excellent safety profile with good local tolerability and no systemic exposure

11/26/18

Emergent Biosolutions Inc., of Gaithersburg, Md., and Valneva SE, of Saint-Herblain, France

VLA-1601

Vaccine

Zika virus

Study met primary endpoint, showing favorable safety profile at all doses and schedules tested; VLA-1601 was immunogenic in all treatment groups and induced both dose- and schedule-dependent neutralizing antibodies against Zika virus with kinetics expected for an inactivated, alum-adjuvanted whole-virus vaccine; seroconversion rates reached up to 85.7% on day 35

11/19/18

Synlogic Inc., of Cambridge, Mass.

Synthetic Biotics

SYNB-1020; SYNB-1618: genetically engineered beneficial microbes to perform or deliver critical functions missing or damaged due to disease

Cirrhosis

Study involved the level and inter-individual variability of fasting blood ammonia and the impact of a standard protein meal on blood ammonia levels; found that sample handling and processing have a major impact on ammonia levels and are critical for data quality; venous ammonia is elevated in a subset of patients with cirrhosis without a history of overt HE and increases significantly after a meal containing 20 g of protein for at least 2 hours

11/13/18

Vir Biotechnology Inc., of San Francisco

VIR-2218

RNA interference therapeutic

Chronic hepatitis B

Study begun with partner Alnylam Therapeutics Inc., of Cambridge, Mass.

11/26/18

Inflammatory

Novus Therapeutics Inc., of Irvine, Calif.

OP-0201

Drug-device consisting of surfactant dipalmitoylphosphatidylcholine and spreading agent cholesteryl palmitate suspended in propellant

Otitis media

Dosed the first adult subjects in the low-dose cohort

11/27/18

Musculoskeletal

Anelixis Therapeutics LLC, of Cambridge, Mass.

AT-1501

Anti CD40L antibody

Amyotrophic lateral sclerosis

Dosed the first subject; trial will enroll both healthy volunteers and a total of 8 patients with ALS

11/28/18

Catabasis Pharmaceuticals Inc., of Cambridge, Mass.

Edasalonexent (CAT-1004)

Oral NF-kB inhibitor

Duchenne muscular dystrophy

Data from MoveDMD trial published in the Journal of Neuromuscular Diseases showed drug was well-tolerated in pediatric patients and inhibited NF-kB, a link between loss of dystrophin and disease progression; multipart trial includes phase II and ongoing label-extension

11/27/18

Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J.

Oral edaravone

Free radical scavenge

Amyotrophic lateral sclerosis

Oral edaravone had similar pharmacokinetic parameters to intravenous infusion Eadicava (edaravone)

11/15/18

Myonexus Therapeutics Inc., of New Albany, Ohio, and Sarepta Therapeutics Inc., of Cambridge, Mass.

MYO-101

Gene therapy expressing beta-sarcoglycan

Beta-sarcoglycanopathy

Dosed first patient in cohort 1; all patients in first cohort expected to be dosed this year with data expected in 2019; cohort 2 will be placebo-controlled at same dose as cohort 1 or higher if results justify dose escalation

11/8/18

Spinalcyte LLC, of Houston

Cybrocell

Modified human dermal fibroblasts

Degenerative disc disease

In the 17-patient study, 12-months after injection, 54% of patients treated with Cybrocell met all three endpoints – Oswestry Disability Index, Visual Analogue Scale and disc height via MRI scans – compared to 17% of patients injected with placebo (p=0.0003); 83% of spinal discs injected with Cybrocell had an increase or no change in disc height compared to 25% of saline injected discs

11/14/18

Neurology/Psychiatric

Alector LLC, of South San Francisco

AL-002

TREM2 modulator

Alzheimer's disease

Started placebo-controlled Invoke study, testing the safety, tolerability, pharmacokinetics and pharmacodynamics of escalating single and multiple doses of AL-002 in healthy volunteers and patients with Alzheimer's disease

11/15/18

Aptinyx Inc., of Evanston, Ill.

NYX-2925

NMDA receptor modulator

Healthy subjects (eventually pain relief)

In a 32-subject trial, at all dose levels tested, NYX-2925 produced statically significant enhancements in synaptic plasticity 2 hours after administration as measured by electroencephalography; effects on enhanced stimulus processing persisted for at least 7 days; in a separate 27-patient, sleep-disruption study, NYX-2925 significantly enhanced various measures, including overall sleep duration and non-REM sleep duration without adversely affecting REM sleep

11/12/18

Asceneuron SA, of Lausanne, Switzerland

ASN-120290

Selective inhibitor of O-GlcNAcase enzyme

Progressive supranuclear palsy

Started trial aimed at quantifying target engagement of compound in the human brain using positron emission tomography to help guide dose selection for a planned efficacy study in PSP

11/20/18

Denali Therapeutics Inc., of South San Francisco

DNL-747

Brain-penetrant small-molecule inhibitor of RIPK1

Neurological and inflammatory diseases

Study in healthy volunteers achieved safety, pharmacokinetic and pharmacodynamic objectives; no serious adverse events at doses that achieved high levels of brain exposure and robust target engagement as measured by a blood-based biomarker of RIPK1 activity

11/19/18

International Stem Cell Corp., of Carlsbad, Calif.

ISC-hpNSC

Neural stem cells

Parkinson's disease

Reported 12-month results of the first cohort and 6-month interim results of second cohort, with the % off-time, and PD symptoms return, decreasing an average of 24% for the first cohort and 49% for the second cohort at 6 months post-transplantation; the % on-time increased an average of 19% for the first cohort and 33% for the second cohort; quality of life as measured by the Parkinson's Disease Quality of Life Score-39 Summary Index, improved 15% for the first cohort and 47% for the second cohort at 6 months post-transplantation

11/5/18

Momenta Pharmaceuticals Inc., of Cambridge, Mass.

M-281

Fully human anti-neonatal Fc receptor aglycosylated IgG1 monoclonal antibody

Myasthenia gravis and hemolytic disease of the fetus and newborn

Study found rapid onset and duration of target receptor occupancy, maximal reduction of circulating IgGs, and dose-dependent effect; results published in Clinical Pharmacology & Therapeutics

11/7/18

Tetra Bio-Pharma Inc., of Ottawa

PPP-001

3 strains of dried cannabis

Pain

Received no objection letter from Health Canada to conduct a phase I pharmacokinetics and safety study using a vaporized version of PPP-001

11/1/18

Tikomed AV, of Viken, Sweden

ILB

Pleiotropic molecule 

Amyotrophic lateral sclerosis

First patient treated in trial at Sahlgrenska University Hospital in Gothenburg, Sweden

11/2/18

Voyager Therapeutics Inc., of Cambridge, Mass.

VY-AADC

Gene therapy expressing AADC

Parkinson's disease

The 10 patients in the cohorts treated with the higher 2 doses had a combined mean improvement in good 'on' time of 2.4 hours and 2.6 hours per day from baseline after 12 months and 18 months, respectively; 'off' time plus 'on' time with troublesome dyskinesia decreased by 2.4 hours and 2.6 hours per day from baseline after 12 months and 18 months, respectively

11/7/18

Ocular

Adverum Biotechnologies Inc., of Menlo Park, Calif.

ADVM-022

Gene therapy

Wet age-related macular degeneration

First patient was dosed in the OPTIC trial, designed to assess the safety and tolerability at 24 weeks of a single intravitreal administration in patients who are responsive to anti-VEGF treatment; the study is expected to enroll 18 patients and will evaluate 3 doses of ADVM-022

11/19/18

Ascendis Pharma A/S, of Copenhagen

Transcon CNP

Long-acting prodrug of C-type natriuretic peptide

Achondroplasia

Preliminary results in healthy adults showed treatment provided continuous exposure to CNP with a pharmacokinetic profile designed to maximize efficacy with once-weekly dosing; phase II trial in pediatric achondroplasia patients set to start in mid-2019

11/28/18

Oncobiologics Inc., of Cranbury, N.J.

ONS-5010

Ophthalmic bevacizumab candidate

Wet age-related macular degeneration

Dosed the first patients

11/6/18

Other/Miscellaneous

Adverum Biotechnologies Inc., Menlo Park, Calif.

ADVM-043

AAVrh.10-based gene therapy 

A1AT deficiency

Based on the review of the Advance phase I/II study, the data did not demonstrate the potential to reach M-protein threshold levels of 11 µM; discontinuing development

11/2/18

Respiratory

Bellus Health Inc., of Laval, Quebec

BLU-5937

Selective, oral small-molecule antagonist of P2X3 receptor

Chronic cough

Top-line results showed good safety and tolerability, plus a pharmacokinetic profile supporting twice-a-day dosing; at anticipated therapeutic doses of 50 mg to 100 mg, BLU-5937 did not cause any loss of taste perception, with only 1 of 24 patients reporting transient taste alteration; phase II study in chronic cough expected to start in mid-2019

11/19/18

Immune Regulation Ltd., of Stevenage, U.K.

PIN-201104

Low-molecular-weight peptide derived from Mycobacterium tuberculosis

Asthma

Study confirmed the clean safety profile seen in preclinical studies, with the drug having a side effect profile comparable to placebo; majority of events were classed as mild and no serious or severe adverse events were reported; no dose-limiting safety or tolerability reported in the study and no injection site reactions were seen in the small cohort of healthy subjects receiving subcutaneous dosing

11/26/18

Pulmatrix Inc., of Lexington, Mass.

Pulmazole

Inhaled formulation of itraconazole

Allergic bronchopulmonary aspergillosis

Results of first-in-human study showed drug appeared to be safe and well-tolerated in normal healthy subjects at doses up to 35 mg, the maximal dose tested, over 14 days of administration; single doses of 20 mg and oral Sporanox 200 mg appeared to be safe and well-tolerated in asthmatic subjects; sustained low-level systemic exposure after single and multiple doses over 24 hours post-dose is indicative of high and sustained lung exposure and supports once-daily dosing; phase II trial in asthmatic patients with ABPA set to start in the fourth quarter of 2018

11/21/18

Theravance Biopharma Inc., of Dublin

TD-8236

Inhaled, lung-selective pan-JAK inhibitor

Respiratory disease

Dosed the first subject in 2-part trial; part A will test single ascending doses in healthy subjects, while part B will test multiple ascending doses in patients with mild, stable asthma

11/27/18

Notes

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