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. |