Cancer

Adaptimmune Therapeutics plc (Philadelphia)

SPEAR T-cells targeting MAGE-A10

T-cell therapy

Solid tumors

Safety data from its two ongoing pilot studies showed no evidence of toxicity related to off-target binding or alloreactivity; there were no reports of neurotoxicity safety events similar to CAR-T cell-related encephalopathy syndrome (CRES)1; in the NSCLC study, there was one serious adverse event of cytokine release syndrome (CRS), a Grade 4 event that resolved with treatment

1/9/18

Advaxis Inc. (Princeton, N.J.)

ADXS11-001

Axalimogene filolisbac

High-risk, locally advanced anal cancer

Phase I data showed that nine patients achieved a complete response, and eight patients (89%) remained disease-free at a median follow-up of 42 months; one patient progressed, approximately six months post completion of study treatment, and subsequently died from progressive disease, and one patient expired early in the study unrelated to study treatment

1/26/18

Agenus Inc. (Lexington, Mass.)

AGEN-1884 and AGEN-2034

Anti-CTLA4 and anti-PD1 antibodies

Advanced solid tumors

Launched a phase I/II combination trial and completed enrollment into the first cohort

1/23/18

Alteogen lnc. (Daejeon, South Korea)

ALT-P7

HER2-targeting antibody-drug conjugate

Advanced or recurrent HER2 positive metastatic breast cancer

Dosed the first patient in a phase I trial

1/31/18

Apexian Pharmaceuticals Inc. (Indianapolis)

APX-3330

Orally administered inhibitor of APE1/Ref 1

Solid tumors

Started a phase I trial

1/25/18

Athenex Inc. (Buffalo, N.Y.)

Oraxol

Oral formulation of paclitaxel combined with P-gp inhibitor HM-30181A

Breast cancer

Phase I/II data of 24 patients in Taiwan showed it produced a 50% partial response in the first 12 evaluable patients, with 50% showing stable disease; there was no progressive disease; follow-up time was 2.6 months; grade 4 neutropenia was observed in three patients, and all recovered completely with no patient death; there was no neuropathy

1/23/18

Athenex Inc. (Buffalo, N.Y.)

Oraxol

Oral formulation of paclitaxel combined with P-gp inhibitor HM-30181A

Gastric cancer

Phase Ib data of it plus Cyramza in patients who failed previous chemotherapies showed that of the six patients in the first cohort, two had partial responses and three had stable disease; one patient had progressive disease

1/23/18

Aveo Oncology Inc. (Cambridge, Mass.)

Fotivda

Tivozanib

Advanced, unresectable hepatocellular carcinoma

Phase Ib data showed at a median follow-up of 16.9 months, the study's primary endpoint of median progression-free survival (PFS) and PFS at week 24 were 5.5 months and 47%, respectively; a partial response was seen in four of 19 patients (21%) and stable disease (SD) in eight of 19 patients (42%), for a disease control rate of 63%; overall survival (OS) at six and 12 months was 58% and 25%, respectively, with a median OS of 7.5 months; four patients have maintained SD for more than two years

1/22/18

Basilea Pharmaceutica Ltd. (Basel, Switzerland)

BAL-101553

Oral small-molecule tumor checkpoint controller

Newly diagnosed glioblastoma

Started a phase I study and the first patient has been treated

1/4/18

Bavarian Nordic A/S (Copenhagen)

MVA-BN Brachyury vaccine

Prime-boost cancer immunotherapy

Metastatic or unresectable, locally advanced malignant solid tumors

Started a phase I trial

1/22/18

Cellestia Biotech AG (Basel, Switzerland)

CB-103

Oral pan-NOTCH inhibitor

Solid tumors, lymphomas and leukemias

Dosed the first patients in a phase I/IIa study

1/5/18

Chemocentryx Inc. (Mountain View, Calif.)

CCX-872

CCR2 inhibitor

Locally advanced/metastatic pancreatic cancer

Phase Ib showed that when it was used with the chemotherapy regimen FOLFIRINOX, the combination produced an overall survival (OS) of 29% at 18 months, compared to previously published OS rates of 18.6% using FOLFIRINOX alone

1/24/18

Constellation Pharmaceuticals Inc. (Cambridge, Mass.)

CPI-1205

Small-molecule inhibitor of enhancer of zeste homolog 2

Melanoma, other cancers

Started a phase Ib/II trial named Overcoming Resistance to Immune Oncology therapies by inhibiting EZH2 (ORIOn-E)

1/17/18

Daiichi Sankyo Co. Ltd. (Tokyo)

DS-8201

HER2-targeting antibody-drug conjugate

HER2-expressing gastric cancer

Phase I data in a subgroup of patients previously treated with Herceptin and chemotherapy showed it produced a confirmed overall response rate (ORR) of 45.5% in 44 evaluable patients, a disease control rate (DCR) of 81.8% with a median duration of response of seven months and a Kaplan-Meier estimate of median progression-free survival (PFS) of 5.8 months; a subgroup analysis of 23 patients previously treated with irinotecan showed it produced a confirmed ORR of 43.5%, a DCR of 82.6%, which lasted a median of 6.9 months, and a Kaplan-Meier estimate of median PFS of 4.1 months

1/19/18

Exelixis Inc. (South San Francisco)

Cabometyx

Cabozantinib

Locally advanced or metastatic solid tumors

Amended the protocol for its phase Ib trial to include four new expansion cohorts

1/5/18

Five Prime Therapeutics Inc. (South San Francisco)

FPA-144

Isoform-selective anti-FGF receptor 2b antibody

Previously untreated, advanced gastric or gastroesophageal cancer

Started dosing in the phase I portion of the FIGHT phase I/III study

1/4/18

Gamida Cell Ltd. (Cambridge, Mass.)

NAM-NK Cells

NAM-expanded natural killer cells

Relapsed or refractory CD20-positive non-Hodgkin lymphoma (NHL) or multiple myeloma

Started a phase I trial

1/17/18

Genentech Inc. (South San Francisco, unit of Roche Holding AG)

Cotellic

Cobimetinib

Metastatic colorectal cancer

Phase Ib showed that for the 84 evaluable patients, the median progression-free survival (PFS) was 1.9 months and the median overall survival (OS) was 9.8 months; the 42 patients with confirmed microsatellite-stable disease had a PFS of 2.5 months and a median OS of 13 months

1/23/18

Glenmark Pharmaceuticals Ltd. (Mumbai, India)

GBR-1302

Bispecific antibody

HER2-positive cancers

Preliminary biomarker findings from a phase I study demonstrated modulation of peripheral T-cell populations and cytokines; some subjects treated at the higher doses experienced cytokine release syndrome, which was mild and transient; dose escalation is ongoing

1/25/18

Immunogen Inc. (Waltham, Mass.)

IMGN-632

CD123-targeting antibody-drug conjugate

CD123-positive hematological malignancies

Treated the first patient in a phase I trial

1/5/18

Infinity Pharmaceuticals Inc. (Cambridge, Mass.)

IPI-549

Immuno-oncology drug

Advanced solid tumors

Its phase I/Ib trial is fully enrolled and the dose escalation of the combination component of the trial has been completed

1/9/18

Leap Therapeutics Inc. (Cambridge, Mass.)

TRX-518

Glucocorticoid-inducible TNF-superfamily receptor agonist

Advanced solid tumors

Treated the first patient in its phase I trial

1/17/18

Lycera Corp. (New York)

LYC-55716

Selective retinoic acid-related orphan receptor-gamma agonist

Metastatic non-small-cell lung cancer

Started a phase Ib trial

1/5/18

Macrogenics Inc. (Rockville, Md.)

Margetuximab

Fc-optimized anti-HER2 monoclonal antibody

Gastric cancer and gastroesophageal junction (GEJ) cancer

Phase Ib/II data showed that combining it with Keytruda resulted in an overall response rate (ORR) of 32% in patients with gastric cancer vs. 4% in patients with GEJ cancer; as of the Dec. 4, 2017, data cutoff, the total ORR across all evaluable patients was 18%; disease control rate, including partial responses and stable disease, was higher in patients with gastric vs. GEJ cancer (72% vs. 38%); median progression-free survival also was higher in gastric vs. GEJ patients (5.5 months vs. 1.4 months)

1/19/18

Nanobiotix SA (Paris)

NBTXR-3

Hafnium oxide nanoparticles

Liver cancers

Phase I/II data showed that of the seven evaluable patients, three achieved a complete response and three were partial responders

1/23/18

Nucana plc (Edinburgh, U.K.)

Acelarin

Nucleotide analogue

Front-line advanced biliary tract cancer

Analysis of the phase Ib ABC-08 study showed that when combined with cisplatin, it achieved high response rates and was well-tolerated in front-line advanced biliary tract cancer; eight patients were treated, a complete radiological response was achieved in one patient, a partial response was achieved in three patients and one patient achieved stable disease, for an objective response rate (ORR) of 50% and a disease control rate of 63% on an intent-to-treat basis

1/22/18

Pfizer Inc. (New York)

EOS-200271

Indoleamine 2,3-dioxygenase (IDO-1) inhibitor

Glioblastoma

Cut short a phase I study and handed back rights to Iteos Therapeutics SA (Gosselies, Belgium); interim data showed that it is able to penetrate the brain and confirmed the safety profile at doses up to 500 mg twice daily; however, at that dose the pharmacokinetics flattened out and the trial was ended before the maximum-tolerated dose was reached

1/5/18

Potenza Therapeutics Inc. (Cambridge, Mass.)

PTZ-201

TIGIT antagonist

Advanced solid tumors

Began a phase I, dose-escalation and expansion safety study

1/8/18

Radiomedix Inc. (Houston) and Areva Med LLC (Bethesda, Md.)

Alphamedix

Somatostatin analogue radiolabeled with 212Pb

Somatostatin receptor positive neuroendocrine tumors

Started a phase I trial

1/11/18

Rgenix Inc. (New York)

RGX-104

Oral small molecule targeting the liver X receptor

Advanced solid malignancies and lymphoma

It was able to deplete myeloid-derived suppressor cells in patients participating in the dose-escalation portion of a phase Ia/Ib trial, which resulted in robust activation of relevant T-cell populations

1/12/18

Sillajen Inc. (San Francisco)

Pexa-Vec

Pexastimogene devacirepvec

Metastatic colon cancer

Started a phase I/II trial

1/30/18

Sun Biopharma Inc. (Minneapolis)

SBP-101

Polyamine compound

Metastatic pancreatic cancer

Started a first-line dose-escalation study

1/30/18

Symbio Pharmaceuticals Ltd. (Tokyo)

Treakisym

Bendamustine

Progressive solid tumors

Started a phase I trial

1/23/18

Targovax AS (Oslo, Norway)

ONCOS-102

Virus-based oncolytic immunotherapy

Advanced melanoma

Immune activation data from the first four patients in its phase I trial showed that at week three, patients had increases of several pro-inflammatory cytokines, the relative level of cytotoxic CD8+ T cells and PD-1 expression on CD8+ T cells

1/5/18

Tracon Pharmaceuticals Inc. (San Diego)

TRC-105

Antibody targeting endoglin

Advanced hepatocellular carcinoma

Data from its ongoing phase Ib/II trial testing it plus Nexavar showed that of the eight evaluable patients, two had a partial response by RECIST 1.1 and three patients experienced a 50% or greater reduction in the concentration of the tumor marker alpha fetoprotein

1/22/18

VBI Vaccines Inc. (Cambridge, Mass.)

VBI-1901

Bivalent therapeutic vaccine

Recurrent glioblastoma multiforme

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

1/18/18

Dermatologic

Asana Biosciences Inc. (Lawrenceville, N.J.)

ASN-002

Oral, once-daily dual spleen tyrosine kinase and Janus kinase inhibito

Moderate to severe atopic dermatitis

It achieved clinical efficacy in a proof-of-concept study, met the safety and efficacy endpoints after four weeks of treatment and was well-tolerated

1/5/18

Kalvista Pharmaceuticals Inc. (Cambridge, Mass.)

KVD-900

Oral plasma kallikrein inhibitor

Oral hereditary angioedema

Started a phase I trial

1/8/18

Endocrine/Metabolic

Adocia SA (Lyon, France)

Biochaperone Combo 75/25

Basal insulin glargine and prandial insulin lispro

Type 2 diabetes

Both phase Ib primary endpoints – the assessments of dose-proportionality for total insulin exposure (AUCtotal_insulin 0-last) and maximal observed total plasma insulin concentration (Cmax) across three doses – were met (AUC0-last overall dose exposure slope 0.93; 95% confidence interval [0.58 ; 1.29] and Cmax overall dose exposure slope 0.80, 95% CI [0.43 ; 1.17]); a dose-proportionality relationship was demonstrated for all exposure pharmacokinetic parameters assessed in the early, intermediate and basal phases

1/26/18

Ascendis Pharma A/S (Copenhagen, Denmark)

Transcon PTH

Parathyroid hormone (PTH)

Hypopara-thyroidism

Phase I data showed it produced a half-life of approximately 60 hours; single ascending doses produced sustained dose-dependent elevations of serum calcium that lasted more than 72 hours and had low inter-subject variability as well as producing expected effects on renal calcium reabsorption and down regulation of endogenous PTH(1-84)

1/9/18

Diurnal Group plc (Cardiff, U.K.)

Alkindi

Hydrocortisone

Adrenal insufficiency

A food matrix compatibility study in healthy volunteers showed the pharmacokinetics of it when sprinkled onto soft food or yogurt were equivalent to it being administered directly

1/10/18

Saniona AB (Ballerup, Denmark)

Tesomet

Fixed-dose combination of tesofensine and metoprolol

Type 2 diabetes

Dosed the first 10 participants in a phase I trial

1/17/18

Ultragenyx Pharmaceutical Inc. (Novato, Calif.)

DTX301

Gene therapy

Ornithine transcarbamylase deficiency

Results from the first dose cohort of a phase I/II trial showed: the first patient increased their rate of ureagenesis from 200 umol/kg/hr at baseline to 335 umol/kg/hr at six weeks and 261 umol/kg/hr at 12 weeks; the second patient didn't show a clinically meaningful change in rate of ureagenesis during the 12 weeks; a third patient had a modest increase in ureagenesis at six weeks and hadn't reached 12 weeks as of the Dec. 22, 2017 cutoff

1/10/18

Viacyte Inc. (San Diego)

PEC-Direct

Islet cell replacement therapy

Type 1 diabetes

The first patients in cohort 2 were implanted with a potentially efficacious dose in the phase I/II study

1/8/18

Gastrointestinal

Seres Therapeutics Inc. (Cambridge, Mass.)

SER-287

Consortium of live bacterial spore

Mild to moderate ulcerative colitis

Phase Ib data showed treatment led to engraftment, as measured by the number of detectable SER-287-derived bacterial species, in a dose-dependent manner with daily dosing producing the most rapid and robust change in patients' microbiome; pre-treatment with vancomycin increased the engraftment compared to placebo pre-treatment

1/5/18

Topivert Pharma Ltd. (London)

TOP1288

Narrow spectrum protein kinase inhibitors

Ulcerative colitis (UC)

In a phase I TV03 trial, cells in the biopsies showed dose-dependent positive effects on markers of target engagement and biomarker responses; in a separate study, it and a placebo were delivered as a liquid enema in patients with UC, but a large response in the placebo arm made assessment of efficacy uninterpretable

1/10/18

Zealand Pharma A/S (Copenhagen)

Glepaglutide

GLP-2 analogue

Short bowel syndrome

Results from a phase I trial in 75 healthy subjects showed the pharmacokinetic profile of the drug supports once- or twice-weekly dosing

1/24/18

Genitourinary/Sexual Health

Ovascience Inc. (Waltham, Mass.)

Ovaprime

Fertility treatment

Primary ovarian insufficiency (POI) and poor ovarian response (POR)

Phase I data showed it was generally safe and well-tolerated

1/5/18

Hematologic

Protalex Inc. (Florham Park, N.J.)

PRTX-100

Purified form of Staphylococcal protein A

Persistent/chronic immune thrombocytopenia

Dosed the first patient in its fifth and highest dose cohort of the phase Ib PRTX-100-203 trial testing it at 24 mcg/kg in adults; one of the three patients treated at the fourth dose of 18 mcg/kg achieved a protocol-defined platelet response

1/19/18

Immune

Cellect Biotechnology Ltd. (Tel Aviv, Israel)

Apograft

Isolates stem cells through apoptosis

Graft vs. host disease

After one month follow-up in the phase I/II trial, all three patients have demonstrated complete acceptance of the stem cell transplant with no adverse events related to the treatment and no reported serious adverse events or suspected unexpected serious adverse reactions

1/5/18

Cynata Therapeutics Ltd. (Melbourne, Australia)

CYP-001

Mesenchymal stem cell product

Steroid-resistant acute graft-vs.-host disease (GvHD)

Phase I data showed all eight patients in cohort A, who were treated with 1 million cells/kg, up to a maximum of 100 million cells per infusion, had at least a partial response, defined as an improvement in the severity of GvHD by at least one grade compared to baseline; enrollment has opened in cohort B

1/23/18

Momenta Pharmaceuticals Inc. (Cambridge, Mass.)

M-281

Fully human anti-neonatal Fc receptor aglycosylated immunoglobulin G monoclonal antibody

Reduction of circulating IgG antibodies

Over the 98-day multiple ascending-dose phase I study, it exhibited no serious adverse events, was well-tolerated, and decreased circulating IgG levels up to 89% with a mean reduction of 84%

1/8/18

Infection

Achaogen Inc. (South San Francisco)

C-Scape

Oral combination of ceftibuten, cephalosporin and clavulanate

Complicated urinary tract infections

Phase I data showed it was well-tolerated across all doses studied, with no drug-drug interaction between the previously approved compounds when dosed in combination

1/3/18

Ampliphi Biosciences Corp. (San Diego)

AB-SA01 and AB-PA01

Bacteriophage therapeutics

Staphylococcus aureus  and pseudomonas aeruginosa infections

Seven patients with serious, life-threatening infections, not responding to antibiotics, were treated in the program, with six patients, 86%, achieving treatment success; treatment was well tolerated in all patients

1/5/18

Chimerix Inc. (Durham, N.C.)

Brincidofovir

Nucleotide analog with antiviral activity, IV formulation

Viral infections

Phase I data showed successful administration of two to four weeks without dose-limiting gastrointestinal events, supporting the progression to phase II studies of this version in virally infected transplant recipients; twice-weekly doses provided similar blood levels of the drug as the oral BCV 100 mg dose, but did not result in any reported diarrhea

1/9/18

Chimerix Inc. (Durham, N.C.)

CMX-521

Direct-acting antiviral

Norovirus

Initiated phase I clinical trials

1/9/18

Da Volterra SA (Paris)

DAV-132

Captures residual antibiotics in the colon

Clostridium difficile infection

It reduced exposure of intestinal microbiota to moxifloxacin by 99% in a study of 44 healthy volunteers; subjects who received moxifloxacin alone had a reduction in bacterial gene richness to 54.6% of baseline, affecting 39% of bacterial species identified, which failed to return to baseline after one month following treatment; in subjects treated with it plus moxifloxacin, the bacterial gene richness was 97.85 of baseline with 93% of bacterial species protected

1/17/18

SAB Therapeutics Inc. (Sioux Falls, S.D.)

SAB-301

Anti-MERS immunotherapy

Middle East respiratory syndrome (MERS)

It appeared safe in a phase I trial in healthy volunteers

1/12/18

Transgene SA (Strasbourg, France)

T-101

Vaccine

Chronic hepatitis B virus infection

The first patient was dosed in a phase I trial in China

1/18/18

Vaccitech Ltd. (Oxford, U.K.)

ChAdOx1

Vaccine

Middle East respiratory syndrome (MERS)

Began a phase I trial

1/16/18

Inflammatory

Galapagos NV (Mechelen, Belgium)

GLPG1972

Targets cartilage degrading enzyme ADAMTS-5

Osteoarthritis

Phase Ib data showed it was well tolerated with one treatment discontinuation with reversible abnormal liver function test on day 15 in the highest dose cohort; pharmacokinetics in a population of elderly individuals were similar to those in healthy volunteers, potentially supporting once-daily oral treatment

1/9/18

Musculoskeletal

Audentes Therapeutics Inc. (San Francisco)

AT-132

Gene therapy

X-linked myotubular myopathy

Data from the first dose cohort of the phase I/II Aspiro trial showed significant improvements in neuromuscular and respiratory function

1/5/18

Biohaven Pharmaceutical Holding Co. Ltd. (New Haven, Conn.)

BHV-0223

Sublingual formulation of riluzole

Amyotrophic lateral sclerosis

In 138 healthy volunteers, it achieved 90% of area-under-the-curve exposure and 113% of peak exposure compared to exposures generated by generic riluzole

1/10/18

Neurology/Psychiatric

Acura Pharmaceuticals Inc. (Palatine, Ill.)

LTX-03

Immediate-release hydrocodone bitartrate

Pain

Results from study AP-LTX-301 identified a formulation that the company contends optimizes the balance between providing therapeutic blood levels of drug for pain relief at a single tablet dose while retarding the bioavailability of drug when higher buffer levels are ingested

1/9/18

Biohaven Pharmaceutical Holding Co. Ltd. (New Haven, Conn.)

BHV-5000

Low trapping N-methyl-D-aspartate

Neurologic and neuropsychiatric indications

Started enrolling healthy volunteers in a study

1/19/18

Cortexyme Inc. (South San Francisco)

COR-388

Bacterial protease inhibitor

Alzheimer's disease

The first cohort has been dosed in the initial trial of its phase I program

1/5/18

Elite Pharmaceuticals Inc. (Northvale, N.J.)

Sequestox

Immediate-release oxycodone hydrochloride

Pain

Results from a pilot study showed it produced a Tmax range equivalent to the reference product, Roxicodone, when taken with a standard high fat meal

1/31/18

GT Biopharma Inc. (Los Angeles)

GTP-004

Pyridostigmine and ondansetron

Myasthenic syndromes

Completed phase I dosing in five healthy volunteers and showed the dose-limiting gastro-intestinal adverse events caused by pyridostigmine were alleviated when the patients received it, with subjects tolerating doses as high as 120 mg, the maximum allowed in the study

1/10/18

Ocular

Opthea Ltd. (Melbourne, Australia)

OPT-302

Soluble form of VEGFR-3

Center-involved diabetic macular edema

Started a phase Ib/IIa trial

1/4/18

Other/Miscellaneous

Elite Pharmaceuticals Inc. (Nortvale, N.J.) and Sung-en Pharma LLC (Princeton, N.J.)

Undisclosed

Undisclosed extended-release generic product

Undisclosed

A fasting study in healthy volunteers with product beads sprinkled on applesauce showed the drug was bioequivalent to the branded product

1/11/18

Tosk Inc. (Mountain View, Calif.)

TK-90

Targets side effects of certain cancer drugs

Mucositis

Positive preliminary safety and efficacy was seen in its phase I trial

1/5/18

Respiratory

Galapagos NV (Mechelen, Belgium)

GLPG-2851

C1 corrector

Cystic fibrosis

Started a phase I study enrolling healthy volunteers

1/4/18

Pulmatrix Inc. (Lexington, Mass.)

PUR-1900

Pulmazole

Allergic bronchopulmonary aspergillosis associated with asthma

Began screening subjects in a phase I trial

1/23/18

Notes

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

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