Company (location)

Product

Description

Indication

Status

Date

Autoimmune

Acea Biosciences Inc. (San Diego)

AC-0058

Irreversible Bruton's tyrosine kinase inhibitor

B-cell-related autoimmune diseases, including rheumatoid arthritis and systemic lupus erythematosus

Completed its phase I study; both trials met their primary endpoints, and the maximum tolerated dose was not reached in either case

6/15/17

UCB SA (Brussels, Belgium)

Cimzia

Certolizumab pegol; pegylated anti-TNF antibody

Rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis/ankylosing spondylitis and Crohn's disease

Results from its phase I CRIB trial, designed to measure the transfer of Cimzia across the placenta from pregnant women to their infants, found no measurable levels of Cimzia in 13 out of 14 infant blood samples at birth, and in all infant samples at weeks four and eight after birth

6/15/17

Cancer

ADC Therapeutics Sarl (Lausanne, Switzerland)

ADCT-402

CD19-directed antibody linked to a pyrrolobenzodiazepine (PBD)-dimer toxin

Relapsed or refractory non-Hodgkin's lymphoma (r/rNHL)

Reported that it attained an overall response rate of 61% in a phase I dose-escalation trial in patients who had reached a dose of at least 120 mcg/kg; for a subset of patients with diffuse large B-cell lymphoma, the response rate was 57% for those who had also crossed the same dose threshold

6/16/17

Aduro Biotech Inc. (Berkeley, Calif.)

ADU-S100 (also known as MIW-815)

STING pathway activator

Advanced/metastatic solid tumors or lymphomas

The FDA cleared the IND for it to be evaluated in combination with PDR-001, from Novartis AG, of Basel, Switzerland

6/2/17

Argenx NV (Breda, the Netherlands)

ARGX-110

Simple Antibody that blocks CD70 activity

Relapsed/refractory cutaneous T-cell lymphoma

Updated data from its phase Ib expansion study continued to show evidence of clinical and/or biological antitumor activity with the compound; a partial response and stable disease, respectively, was observed in three and seven out of 16 patients

6/15/17

Armo Biosciences Inc. (Redwood City, Calif.)

AM-0010

Pegilodecakin, pegylated interleukin-10

Advanced pancreatic ductal adenocarcinoma

Data from a phase I/Ib trial showed that, of the 21 patients who had been treated with a median of two prior therapies, 47% were alive one year after starting treatment with AM-0010 in combination with FOLFOX (folinic acid, 5-fluorouracil and oxaliplatin); as a monotherapy, AM-0010 produced a one-year survival of 22% in 22 patients who had been treated with a median of three prior therapies

6/30/17

Beigene Ltd. (Beijing)

BGB-3111

BTK inhibitor

Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL)

Updated data from an ongoing phase I study continue to demonstrate that the compound is well-tolerated and highly active in CLL/SLL, with an overall response rate of 94% and a treatment discontinuation rate of 3% at a median follow-up of 10.5 months for efficacy evaluation

6/15/17

Beigene Ltd. (Beijing)

BGB-3111

BTK inhibitor

Waldenström's macroglobulinemia

Updated phase I data showed it continues to be well-tolerated, with a partial response rate of 43% and with an overall response rate of 90% (38 of 42 efficacy-evaluable patients), with a median follow-up time of 12.3 months; major response rate in the study was 76% (32 of 42 patients) and partial responses were seen in 14 of 42 patients (33%)

6/16/17

Beigene Ltd. (Beijing)

BGB-3111

Bruton's tyrosine kinase inhibitor

Chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL) and follicular lymphoma (FL)

Data from a phase I trial testing BGB-3111 combined with Gazyva (obinutuzumab, Roche Holding AG) showed the combo was well-tolerated with an overall response rate (ORR) of 89%, including 22% complete responses (CRs), in 18 patients with CLL and SLL who were previously untreated, and an ORR of 92%, including 16% CRs, in 25 CLL/SLL patients who were relapsed/refractory (R/R) to other treatments; the combination produced an ORR of 73%, including 33% CRs, in 15 R/R FL patients

6/19/17

Beigene Ltd. (Beijing)

BGB-A317

Anti-PD-1 antibody

Solid tumors

Preliminary results from a cohort of patients in its phase Ia/Ib trial showed that, of the 27 patients with advanced hepatocellular carcinoma evaluable for response, 12 remained on treatment, including three with partial responses and nine with stable disease

6/30/17

Biolinerx Ltd. (Tel Aviv, Israel)

BL-8040

CXCR4 antagonist

Solid tumors

Said Genentech, a member of the Roche Group, of Basel, Switzerland, has filed three regulatory submissions to start phase Ib trials for BL-8040 in combination with Tecentriq (atezolizumab)

6/2/17

Blaze Bioscience Inc. (Seattle)

BLZ-100

Tozuleristide; tumor paint products

Brain cancer

Phase I data, from pediatric patients, showed it to have the potential to aid surgeons in achieving maximal safe surgical resection; BLZ-100 was well-tolerated in the dose levels studied with tumor fluorescence observed in a majority of evaluated tumors

6/16/17

Briacell Therapeutics Corp. (Berkeley, Calif.)

Briavax

Genetically engineered whole-cell vaccine derived from a human breast tumor cell line

Advanced breast cancer

Enrolled the second patients in a U.S.-based open-label phase I/IIa trial

6/2/17

Cellectis SA (New York)

UCART123

Gene-edited CAR T-cell product candidate targeting CD123

Acute myeloid leukemia

Treated the first patient in its phase I trial

6/29/17

Celyad SA (Mont-Saint-Guibert, Belgium)

CAR-T NKR-2

T-cell therapy

Solid tumors

Early results from the phase I THINK trial (THerapeutic Immunotherapy with CAR-T NKR-2) showed it achieving a confirmed stable disease according to the Response Evaluation Criteria In Solid Tumors criteria three months after initial treatment; median progression free survival in these patients under standard of care is between 1.9 and 3.2 months

6/20/17

Cytomx Therapeutics Inc. (South San Francisco)

CX-2009

Probody drug conjugate that targets CD-166

Advanced solid tumors

Treated the first patient in the dose-finding phase I/II PROCLAIM-CX-2009 (Probody Clinical Assessment In Man) trial

6/29/17

Genexine Inc. (Seongnam, South Korea)

GX-188E

DNA vaccine

Human papillomavirus-associated cancers

Genexine received approval from the South Korean Ministry of Food and Drug Safety for a phase Ib/II trial testing GX-188E in combination with Keytruda (pembrolizumab)

6/21/17

Genmab A/S (Copenhagen)

Tisotumab vedotin

Antibody-drug conjugate targeted to Tissue Factor

Solid tumors

Preliminary data from the ongoing phase I/II study showed 11 of 34 evaluable patients in the cervical cancer cohort in part two of the study achieved a response

6/19/17

Hutchison China Meditech Ltd. (Chi-Med; Hong Kong)

HMPL 453

Small-molecule inhibitor targeting fibroblast growth factor receptor

Locally advanced or metastatic solid tumors

The first patient was dosed in a phase I/II trial in patients for whom standard therapy either does not exist or has proved to be ineffective or intolerable, regardless of genetic status, to determine the maximum tolerated dose and recommended phase II dose

6/23/17

Mabvax Therapeutics Holdings Inc. (San Diego)

MVT-2163

Positron emission tomography imaging agent

Pancreatic cancer diagnosis

Reported that 12 patients have so far been treated in a phase I trial evaluating the safety and feasibility of MVT-2163 to image pancreatic tumors and other CA19-9-positive malignancies; it was administered alone and in combination with MVT-5873 and was well-tolerated in all cohorts; infusion reactions were resolved on the day of the injection, though some required supportive medication

6/15/17

Mabvax Therapeutics Holdings Inc. (San Diego)

MVT-1075

Fully human antibody radioimmunotherapy

CA19-9-positive malignancies, including pancreatic, colon and lung cancers

The first patient has been dosed in its phase I trial

6/28/17

Medical Prognosis Institute A/S (MPI; Hoersholm, Denmark)

APO-010

Targets the FAS-ligand

Multiple myeloma

The first patient entered the Oncology Venture APO-010 phase I/II trial

6/1/17

Mei Pharma Inc. (San Diego)

ME-401

Selective, oral PI3K delta inhibitor

Relapsed/refractory chronic lymphocytic leukemia and follicular lymphoma

An independent safety review committee completed its pre-specified review of the first cohort of six evaluable patients in a phase Ib, open-label, dose-escalation study and declared a minimum biologically effective dose for ME-401 at the starting dose of 60 mg and recommended escalation to a 120-mg dose cohort; to date, all six patients have been on study for a minimum of 10 weeks (ranging 10 to 28 weeks), with no reports of ALT/AST elevations, colitis or pneumonitis, events commonly reported with other drugs in that class

6/1/17

Minomic International Ltd. (Sydney)

Miltuximab (MIL-38)

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

Metastatic prostate, bladder and pancreatic cancers

Is proceeding to the second stage of the MILGa trial; in the first part of the study, two patients with pancreatic cancer and four with prostate cancer were dosed with miltuximab; based on the safety data from the first six patients, the trial's independent drug safety monitoring committee recommended enrolling the final six of 12 subjects to further measure safety and tolerability, as well as tumor targeting, pharmacokinetics and dosimetry to determine relative accumulation in different organs

6/30/17

Moleculin Biotech Inc. (Houston)

Annamycin

Anthracycline

Relapsed or refractory acute myeloid leukemia

Plans to expand its phase I/II trial to sites in Poland to increase the rate of patient accrual

6/16/17

Nordic Nanovector ASA (Oslo, Norway)

Betalutin

177Lu-satetraxetan-lilotomab; CD37-targeting antibody-radionuclide conjugate

Recurrent indolent non-Hodgkin's lymphoma

Updated results from its ongoing LYMRIT 37-01 phase I/II trial in patients who have failed multiple prior treatments showed that, as of the cut-off date of May 6, Betalutin produced a 64% overall response rate and a 28% complete response rate

6/15/17

Novartis AG (Basel, Switzerland)

CTL-119

Humanized CD19-directed CAR T-cell therapy

Relapsed/refractory chronic lymphocytic leukemia (CLL)

Findings from a pilot study of CTL-119 in combination with Imbruvica (ibrutinib, Abbvie Inc./Johnson & Johnson) in patients who had been taking ibrutinib for at least six months and who were not in complete remission, showed that eight of nine evaluable patients had no signs of CLL in their bone marrow at three months; one of those patients had a partial response

6/1/17

Onoclys Biopharma Inc. (Tokyo, Japan)

Telomelysin (OBP-301)

Oncolytic adenovirus modified to selectively replicate in cancer cells by introducing the human telomerase reverse transcriptase promotor

Advanced or metastatic solid tumors

A clinical trial notification was submitted to the Pharmaceuticals and Medical Devices Agency in Japan for a phase I trial testing Telomelysin in combination with Keytruda (pembrolizumab, Merck & Co. Inc.)

6/29/17

Prima Biomed Ltd. (Sydney)

IMP-321

Antigen-presenting cell activator

Unresectable or metastatic melanoma

Completed recruitment of the first two cohorts of six patients and commenced recruitment of the third cohort in its TACTI-mel study testing IMP-321 in combination with an approved anti-PD-1 antibody in patients who did not optimally respond to the checkpoint inhibitor alone

6/27/17

Seattle Genetics Inc. (Bothell, Wash.) and Bristol-Myers Squibb Co. (Princeton, N.J.)

Adcetris and Opdivo

Brentuximab vedotin and nivolumab

Relapsed or refractory classical Hodgkin lymphoma

Updated interim analysis data from the ongoing phase I/II study showed that 50 (85%) had an objective response, including 37 patients (63%) with a complete response and 13 patients (22%) with a partial response; five patients (8%) had stable disease and four patients (7%) had progressive disease

6/16/17

Spectrum Pharmaceuticals Inc. (Henderson, Nev.)

Folotyn

Pralatrexate

Relapsed or refractory peripheral T-cell lymphoma (PTCL)

Data from a phase I trial testing Folotyn plus Istodax (romidepsin, Celgene Corp.) showed that, of the 14 PTCL patients that could be evaluated for response, 10 achieved a response with four (29%) complete responses, six (43%) partial responses, and once patient with stable disease

6/20/17

Sun Biopharma Inc. (Minneapolis)

SBP-101

Polyamine compound

Locally advanced or metastatic pancreatic ductal adenocarcinoma

Completed the fifth patient cohort in the dose-escalation phase of its phase I trial; a preliminary review from patients in the first five cohorts showed that six of 20 (30%) had stable disease and 14 of 20 (70%) had progressive disease

6/8/17

TG Therapeutics Inc. (New York)

TGR-1202

Umbralisib; next-generation PI3K-delta inhibitor

Chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL)

Updated data from its ongoing phase I/Ib trial of TGR-1202 in combination with Imbruvica (ibrutinib, Abbvie Inc. and Johnson & Johnson) showed that 94% (16 of 17) of CLL patients achieved a complete response (CR), partial response or a partial response with lymphocytosis, with one patient achieving a CR and three additional patients with radiographic CR; one-year progression-free survival for CLL is 88% and overall survival at one year is 94%, with the longest patient on study for 29.5-plus months; in MCL, an objective response rate was 79% (11 of 13 patients), including one CR and one additional radiographic CR

6/15/17

TG Therapeutics Inc. (New York)

TG-1101 and TGR-1202

Ublituximab, a glycoengineered anti-CD20 monoclonal antibody, and umbralisib, a PI3K-delta inhibitor

Diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL)

Updated phase I/Ib data of TG-1101 in combination with TGR-1202 and bendamustine showed a 100% (four of four patients) objective response rate (ORR), including a 50% complete response (CR) rate, in patients with relapsed DLBCL; a 50% (six of 12 patients) ORR, including a 42% CR rate, was observed in patients with refractory DLBCL with durable CR and partial response rates observed; an 88% (seven of eight patients) ORR, including a 50% CR rate, was observed in patients with relapsed or refractory FL

6/16/17

Threshold Pharmaceuticals Inc. (Menlo Park, Calif.)

Evofosfamide (TH-302)

Hypoxia-activated prodrug of a bis-alkylating agent

Prostate cancer, pancreatic cancer, melanoma or human papillomavirus-negative squamous cell carcinoma of the head and neck

The University of Texas MD Anderson Cancer Center dosed the first patient in a phase I immunotherapy trial investigating Yervoy (ipilimumab, Bristol-Myers Squibb Co.) and evofosfamide for the treatment of patients for which standard therapy does not offer the potential for increased survival

6/14/17

Trillium Therapeutics Inc. (Toronto)

TTI-621

IgG1 SIRPaFc fusion protein targeting CD47

Hodgkin lymphoma

Expanded its intravenous dosing trial of TTI-621 to include an additional cohort of patients with Hodgkin lymphoma treated with a combination of TT-621 and Opdivo (nivolumab, Bristol-Myers Squibb Co.) as well as two additional cohorts of patients with T- and B-cell acute lymphoblastic leukemia and small-cell lung cancer treated with TTI-621 monotherapy

6/8/17

Trovagene Inc. (San Diego)

PCM-075

Polo-like kinase 1 inhibitor

Acute myeloid leukemia

Submitted an IND to the FDA to conduct a phase Ib/II trial

6/28/17

Unum Therapeutics Inc. (Cambridge, Mass.)

ACTR707

Uses Unum's Antibody-Coupled T-cell Receptor (ACTR) technology

Relapsed/refractory CD20-positive B cell non-Hodgkin lymphoma

Filed an investigational new drug application with the FDA for ACTR707 in combination with Rituxan (rituximab, Roche Holding AG/Biogen Inc.)

6/20/17

Ziopharm Oncology Inc. (Boston)

Ad-RTS-hIL-12 + veledimex

Gene therapy for controlled expression of IL-12

Recurrent glioblastoma multiforme

Started enrollment in the stereotactic arm of a phase I trial

6/29/17

Cardiovascular

Ablynx NV (Ghent, Belgium)

Caplacizumab

Bivalent anti-von Willebrand factor nanobody

Acquired thrombotic thrombocytopenic purpura

The first healthy volunteers in Japan were dosed in the phase I study

6/27/17

Arena Pharmaceuticals Inc. (San Diego)

Extended-release (XR) ralinepag

IP receptor agonist

Pulmonary arterial hypertension

Completed a phase I trial measuring the pharmacokinetic profile compared to the immediate-release (IR) formulation in healthy adults; the XR formulation dosed daily had lower maximum plasma concentrations compared to the IR formulation, but the XR formulation maintained similar total plasma concentrations

6/30/17

Athera Biotechnologies AB (Stockholm)

PC-mAb

Fully human antibody

End stage renal disease

Completed its multiple-dosing study in healthy volunteers

6/21/17

Catalyst Biosciences Inc. (South San Francisco) and Isu Abxis Co Ltd. (Seoul, South Korea)

CB-2679d/ISU-304

Coagulation factor IX variant

Severe hemophilia B

Completed dosing of the first of up to five patient cohorts in a phase I/II proof-of-concept trial

6/15/17

Incarda Therapeutics Inc. (Brisbane, Calif.)

Inrhythm

Inhaled flecainide

Symptomatic acute episodes of paroxysmal atrial fibrillation

Top-line data from a phase I study showed it met its endpoints of safety and tolerability; all doses of inhaled flecainide administered (estimated lung doses of 20 mg to 60 mg) were found to be safe and well-tolerated

6/8/17

Central nervous system

Affiris AG (Vienna)

Affitope PD03A

Synthetically produced alpha-Synuclein (aSyn)-mimicking peptide vaccine

Early Parkinson's disease

Top-line results of its phase I study, AFFiRiS011, showed both doses were well-tolerated locally and systemically; no serious adverse events or suspected unexpected serious adverse reactions related to the drug occurred; of adverse events, approximately 59% were local reactions, most of them mild and without dose dependency

6/8/17

Asterias Biotherapeutics Inc. (Fremont, Calif.)

AST-OPC1

Oligodendrocyte progenitor population derived from human embryonic stem cells

Cervical spinal cord injury

Nine-month follow-up data from the AIS-A 10 million cell cohort in the ongoing SCiStar phase I/IIa trial showed that three of six (50%) patients have now recovered two levels of motor function, and previously announced improvements in arm, hand and finger function at three months and six months following administration of AST-OPC1 have been confirmed and further increased at nine months

6/14/17

Cerveau Technologies Inc. (Boston)

MK-6240

Tau imaging agent

Neurodegen-erative diseases, including Alzheimer's disease

The FDA cleared its IND

6/19/17

Chromocell Corp. (North Brunswick, N.J.)

CC-8464

Inhibitor for the Nav1.7 ion channel

Pain

Initiated the multiple ascending-dose portion of its phase I trial

6/28/17

Ionis Pharmaceuticals Inc. (Carlsbad, Calif.)

IONIS-HTTRx

Antisense drug designed to reduce the production of the toxic mutant huntingtin gene

Huntington's disease

Completed enrollment in the phase I/IIa trial

6/23/17

Kempharm Inc. (Coralville, Iowa)

KP-511

Prodrug of hydromorphone

Severe pain

Pharmacokinetic findings showed marked reductions in the rate and extent of plasma hydromorphone exposure when compared with intranasal HM HCl; peak concentration (Cmax) was reduced by about 63% and mean overall HM exposure was about 58% and 48% lower as measured by AUClast and AUCinf, respectively, with KP-511 as compared to HM HCl; intranasal KP-511 demonstrated reductions in FDA-recommended human abuse-related endpoints compared with intranasal HM HCl

6/12/17

Living Cell Technologies Ltd. (Melbourne, Australia)

Ntcell

Cell therapy

Parkinson's disease

All four patients participating in the phase I/IIa study show no safety concerns, the primary endpoint of the study, which involved the implantation of 40 Ntcell capsules into the putamen on one side of the brain only; in all patients, Ntcell treatment continues to show improvement over baseline, with efficacy most evident in the measurement of motor function

6/7/17

Nobilis Therapeutics Inc. (Portland, Ore.)

NBTX-001

Inhalational gas mixture containing xenon

Panic disorder

Data from an 81-patient trial showed that after treatment, both groups – with and without other psychiatric co-morbidities – were found to have a dramatic reduction in the incidence of panic attacks, an effect that was sustained during the six months of study follow-up; patients with concomitant anxiety and depression were found to have a substantial decrease in symptom severity

6/15/17

Pharmaleads SAS (Paris)

PL-265

Dual enkephalinase inhibitor

Neuropathic pain

Results from a 32-patient phase I single ascending-dose trial of PL-265 in healthy volunteers showed it broke down into its active metabolite, which inhibited both of its target enzymes, aminopeptidase N and neprilysin; the maximum tolerated dose wasn't reached, but the 400-mg and 800-mg doses of PL-265 produced enough inhibition after 24 hours to justify further investigation

6/30/17

Prothena Corp. plc (Dublin)

PRX-002

Monoclonal antibody

Parkinson's disease

Baylor College of Medicine researchers investigating it in a phase Ib multiple ascending-dose study found that it may inhibit cell-to-cell transmission of alpha-synuclein, suggesting that it might modify disease progression; PRX-002 was well-tolerated and showed a rapid dose- and time-dependent reduction of alpha-synuclein levels of up to 97% after a single dose

6/9/17

Senmur Pharmaceuticals Inc. (Los Altos, Calif.)

SP-102

Non-opioid gel formulation

Radicular pain

Completed a phase I/II pharmacokinetic (PK) bridging trial, which achieved the primary PK endpoint and demonstrated that a single epidural injection can lead to a sustained analgesic effect lasting one month

6/27/17

Sorrento Therapeutics Inc. (San Diego)

Resinifera-toxin (RTX)

Non-opioid TRPV1 agonist designed to selectively target afferent nerve activation involved in chronic pain states

Intractable pain associated with cancer

The FDA authorized its IND

6/30/17

Voyager Therapeutics Inc. (Cambridge, Mass.)

VY-AADC01

Comprised of the adeno-associated virus-2 capsid and a cytomegalovirus promoter to drive AADC transgene expression

Advanced Parkinson's disease

Dosed the first patient in a phase I trial aimed at further optimizing the surgical delivery

6/27/17

Zynerba Pharmaceuticals Inc. (Devon, Pa.)

ZYN001

Pro-drug of tetrahydrocannabinol delivered via a transdermal patch

Fibromyalgia and neuropathic pain

Initiated its phase I program to assess single and multiple rising doses of several formulations to identify the optimal dose to take into phase II studies

6/27/17

Diabetes

Adocia SA (Lyon, France)

Biochaperone Glucagon

Aqueous formulation of human glucagon

Type I diabetes

Started a phase I study

6/2/17

Adocia SA (Lyon, France)

Biochaperone Combo

Combines long-acting insulin analogue glargine (Lantus, Sanofi SA) and fast-acting insulin analogue lispro (Humalog, Eli Lilly and Co.)

Type 2 diabetes

Top-line results from a phase Ib trial showed that use of the Biochaperone candidate resulted in a statistically significant glucose excursion reduction of 18% in the first two hours compared to Humalog; it was associated with a statistically lower number of hypoglycemic events per subject than Humalog (22 events in 14 subjects vs. 43 events in 20 subjects, respectively, p=0.003) and a higher time in target range during meal tests (blood glucose interval: 72-162 mg/dL, 202 min vs. Humalog 183 min; p=0.04)

6/8/17

Adocia SA (Lyon, France)

Biochaperone Combo 75/25

Combines long-acting insulin analogue glargine (Lantus, Sanofi SA) and fast-acting insulin analogue lispro (Humalog, Eli Lilly and Co.)

Type 2 diabetes

Initiated a phase Ib trial evaluating the dose-linearity of Biochaperone Combo 75/25 at three doses

6/8/17

Antriabio Inc. (Louisville, Colo.)

AB-101

Once-weekly basal insulin

Diabetes mellitus

Filed an IND application with the FDA and intends to initiate a phase I study

6/6/17

Gastrointestinal

Seres Therapeutics Inc. (Cambridge, Mass.)

SER-287

Preparation of highly purified bacterial spores

Mild to moderate ulcerative colitis

Completed enrollment for its ongoing phase Ib study of 58 patients who are failing current therapies

6/6/17

Infection

Achaogen Inc. (South San Francisco)

C-Scape (ACHN-383 and ACHN-789)

Combination of an approved beta-lactam and an approved beta-lactamase inhibitor

Multidrug-resistant gram-negative infections

Dosed the first patient in a phase I trial

6/2/17

Bird Rock Bio Inc. (La Jolla, Calif.)

Namacizumab (RYI-018 or JNJ-2463)

Therapeutic antibody to the cannabinoid 1 receptor

Fibrotic and metabolic disease, including nonalcoholic steatohepatitis

Completed a single ascending-dose, phase I trial

6/6/17

Enyo Pharma SA (Lyon, France)

EYP-001

Synthetic farnesoid X receptor agonist

Chronic hepatitis B virus infection

A phase Ia single and multiple ascending-dose trial testing it in healthy subjects has been completed, with results showing it to be safe and well-tolerated at all doses studied in 80 subjects

6/14/17

Genexine Inc. (Seongnam, South Korea)

GX-188E

HPV therapeutic DNA vaccine

HPV-induced advanced non-resectable cervical cancer

The Ministry of Food and Drug Safety in Korea granted approval to initiate a phase Ib/II trial of GX-188E in combination with Keytruda (pembrolizumab, Merck & Co. Inc.)

6/20/17

Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.)

GLS-5700

Vaccine

Zika virus

Completed enrollment of its phase I trial in Puerto Rico

6/16/17

Pfizer Inc. (New York)

PF-06760805

Vaccine

Group B streptococcus infection

Started a phase I trial

6/20/17

Vaccibody A/S (Oslo, Norway)

VB-10.16

Immunotherapy

High-grade cervical intraepithelial neoplasia (CIN 2/3) caused by human papillomavirus 16 (HPV16)

Results from the phase I part of the VB C-01 trial showed treatment was well-tolerated, with no serious adverse advents reported; immunological analyses of the peripheral blood demonstrated a strong induction of HPV16-specific T-cell immune responses in 12 of 14 patients evaluated

6/22/17

Inflammatory

Galapagos NV (Mechelen, Belgium)

GLPG-1972

Targets ADAMTS-5

Hip and/or knee osteoarthritis

Dosed the first patients in a phase Ib trial of GLPG-1972 to measure the safety and tolerability, pharmacokinetics and pharmacodynamics

6/21/17

Miscellaneous

Alnylam Pharmaceuticals Inc. (Cambridge, Mass.)

Givosiran (ALN-AS1)

RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) to treat

Acute hepatic porphyrias

Interim results from part C, cohorts 1 through 3 (N=12), of its ongoing phase I study showed it achieved potent silencing of the ALAS1 mRNA, resulting in lowering of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic heme intermediates that mediate acute attacks and chronic porphyria symptoms; in the first three unblinded treatment cohorts from Part C, givosiran-treated patients (N=9) experienced a mean 63% reduction in the annualized number of all porphyria attacks relative to the run-in period attack rate, with consistent effects observed across a range of baseline attack rates

6/27/17

Apellis Pharmaceuticals Inc. (Louisville, Ky.)

APL-2

Complement C3 inhibitor

Paroxysmal nocturnal hemoglobinuria

In the PADDOCK phase Ib trial, all three patients treated with APL-2 had lowered lactate dehydrogenase levels (LDH) from an average of 1,615 U/L to an average of 275 U/L, which is 1.1 times the upper limit of normal; in the PHAROAH phase Ib trial testing APL-2 in six patients who had suboptimal response to Soliris (eculizumab, Alexion Pharmaceuticals Inc.), after one month of treatment with both drugs, LDH decreased from an average of 280 U/L, which is 1.3 times the upper limit of normal, to an average of 163 U/L, which is just 0.8 times the upper limit of normal; hemoglobin levels in the patients also increased 36%

6/30/17

Applied Genetic Technologies Corp. (Gainesville, Fla.)

Recombinant adeno-associated virus vector expressing retinoschisin (rAAV2tYF-CB-hRS1)

Gene therapy

X-linked retinoschisis

Top-line safety data for the dose-escalation phase of the company's phase I/II trial; mild to moderate ocular inflammation was observed in the treated eye for the majority of patients and resolved or was controlled either without further intervention or after treatment with topical or oral corticosteroids; no treatment related serious adverse events were reported and the treatment was generally well tolerated

6/12/17

Arqule Inc. (Burlington, Mass.)

ARQ-092

Pan-AKT inhibitor

Overgrowth diseases driven by genetic alterations in the PI2K/AKT1 pathway

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

6/8/17

Biophytis SA (Paris)

Macuneos

Based on the activation of PPAR receptors to limit accumulation of A2E and slow down retinal degeneration

Age-related macular degeneration

Contracted with SGS Life Science Services to conduct the phase I/IIa MACA-PK study

6/19/17

Cytokinetics Inc. (South San Francisco)

CK-2127107

Fast skeletal muscle troponin activator

Spinal muscular atrophy

Started a phase Ib trial testing it in elderly adults with limited mobility

6/30/17

Fibrocell Science Inc. (Exton, Pa.)

FCX-007

Gene therapy

Recessive dystrophic epidermolysis bullosa

The remaining two patients in the NC1-positive cohort have been dosed in the phase I portion of the phase I/II trial, following a data safety monitoring board's recommendation to continue the study; no product-related adverse events were reported

6/9/17

Gensight Biologics SA (Paris)

GS-010

AAV2 containing the human wild-type ND4 gene

Leber's hereditary optic neuropathy

At week 96 post-injection, a mean gain of +29 ETDRS letters (-0.57 LogMAR) was observed in treated (TE) compared to baseline, with a mean gain of +15 ETDRS letters (-0.30 LogMAR) in untreated, resulting in a difference of +14 ETDRS letters in favor of TE

6/15/17

Micron Biomedical Inc. (Atlanta)

Microneedle patch

Drug delivery technology

Delivery method for flu vaccine

Phase I data using its microneedle patch technology showed that the seasonal flu vaccine delivered by microneedle patch was as safe and at least as immunogenic as vaccination with standard needle and syringe; the study demonstrated that patients could self-administer the patch and reported that more than 70% of subjects preferred the microneedle patch over a needle and syringe as their future vaccination method

6/29/17

Neurovive Pharmaceutical AB (Lund, Sweden) and Yungjin Pharm Corp. Ltd. (Seoul, South Korea)

KL-1333

Modulator of the cellular levels of NAD+

Genetic mitochondrial diseases such as MELAS and Kearns-Sayre syndrome

Said a 60-subject phase I study of KL-1333 enrolled its first healthy volunteer

6/28/17

Obseva SA (Geneva)

OBE-2109

Oral GnRH receptor antagonist

Bone mineral density loss and bleeding in estrogen suppression treatment

Completed a phase I trial designed to evaluate the pharmacokinetic/pharmacodynamic relationship of OBE-2109 combined with different doses of add-back therapy (ABT); the addition of ABT doses restored estradiol levels to the target range that Obseva believes would minimize bone mineral density loss; the bleeding pattern during the final four weeks of treatment was as expected, with the vast majority of patients achieving amenorrhea when treated with OBE-2109 alone, while rates of bleeding control were lower in treatment arms that included ABT

6/8/17

Ocera Therapeutics Inc. (Redwood City, Calif.)

Oral OCR-002

Ornithine phenylacetate; ammonia scavenger

Child-Pugh B cirrhosis

Dosed the first patients in part two of a phase I/IIa trial

6/2/17

Regenxbio Inc. (Rockville, Md.)

RGX-314

Gene therapy

Wet age-related macular degeneration

The first patient was dosed in a phase I trial

6/1/17

Rhythm Pharmaceuticals Inc. (Boston) and Camurus AB (Lund, Sweden)

Setmelano-tide (RM-493)

Melanocortin-4 receptor agonist formulated with Camurus' Fluidcrystal drug depot delivery technology

Rare genetic disorders of obesity

Initial results from an ongoing phase Ia trial showed it met pharmacokinetics and tolerability criteria for a once-weekly formulation

6/28/17

Rxi Pharmaceuticals Corp. (Marlborough, Mass.)

RXI-109

Self-delivering RNAi (sd-rxRNA) candidate

Advanced neovascular age-related macular degeneration

Completed enrollment in its phase I/II study RXI-109-1501

6/22/17

Sound Pharmaceuticals Inc. (Seattle)

SPI-1005

Mimics and induces glutathione peroxidase

Meniere's disease

Completed enrollment of its phase Ib trial

6/28/17

Summit Therapeutics plc (Oxford, U.K.)

Ezutromid

Utrophin modulator

Duchenne muscular dystrophy

Data from two phase I trials showed how formulation changes and dietary advice increased ezutromid exposure in patients

6/23/17

Synlogic Inc. (Cambridge, Mass.)

SYNB1020

Synthetic Biotic medicine

Hyperammon-emia in diseases such as urea cycle disorders and hepatic encephalopathy

The first subject was treated in its phase I trial

6/20/17

Tyrogenex Inc. (Rockville, Md.)

X-82

Vorolanib; dual inhibitor of VEGFR and PDGFR

Neovascular age-related macular degeneration

Results from its phase I dose-escalation study showed that, of the 25 patients who completed the 24 weeks of X-82 treatment, 60% required no anti-VEGF injections

6/9/17

Respiratory

Proteostasis Therapeutics Inc. (Cambridge, Mass.)

PTI-801

Transmembrane conductance regulator stimulant

Cystic fibrosis

Completed the healthy volunteer, single ascending-dose portion of its phase I study

6/8/17

Proteostasis Therapeutics Inc. (Cambridge, Mass.)

PTI-428

CF transmembrane conductance regulator amplifier

Cystic fibrosis (CF)

Preliminary data from the multiple ascending-dose cohort of its phase I trial showed that, based on safety and pharmacokinetics data from the first 12 patients, the safety review committee recommend starting the phase II portion of the trial to test a longer duration of treatment, in which the first patient has already been screened

6/30/17

Spyryx Biosciences Inc. (Durham, N.C.)

SPX-101

Inhaled peptide

Cystic fibrosis

Data from a phase I trial testing the safety and tolerability of SPX-101 for up to 14 days in healthy adults showed that in patients who received one of four doses of SPX-101 or placebo twice daily, there were no clinically meaningful changes in lung function as measured by forced expiratory volume in one second in any overall dose group

6/21/17

Verona Pharma plc (London)

RPL-554

Inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4

Chronic obstructive pulmonary disease and cystic fibrosis

The first subjects have been enrolled and dosed in a clinical pharmacokinetic trial in the U.S., following the acceptance of an IND by the FDA

6/6/17


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.