Phase I

Aeglea Biotherapeutics Inc., of Austin, Texas

Pegzilarginase

Enhanced human arginase

Arginase 1 deficiency

Study completed enrollment with 15 patients; interim results show drug produced reductions in plasma arginine and related guanidino compounds; in 3 patients who have completed 8 weeks of treatment, there were improvements in 1 or more instruments of neuromotor function; pivotal trial planed for the first half of 2019 

Aicuris Anti-Infective Cures GmbH, of Wuppertal, Germany

AIC-649

Inactivated parapoxvirus particle

Chronic hepatitis B infection

Maximum tolerated dose was not reached in the 32-patient study that tested 4 ascending doses; drug increased plasma levels of IL-1beta, IL-6, IL-8 and IFN-gamma and decreased levels of IL-10; plans to run further clinical trials

Apellis Pharmaceuticals Inc., of Crestwood, Ky.

APL-2

Inhibitor of complement factor C3

Paroxysmal nocturnal hemoglobinuria

In the Pharoah study, the fourth of 4 patients discontinued treatment with eculizumab and is on APL-2 monotherapy; hemoglobin levels have gone from 8.9 g/dL on eculizumab monotherapy to 11.9 g/dL on eculizumab plus APL-2 to 11.4 g/dL on APL-2 monotherapy; reticulocytes were 2.7, 1.2 and 0.8 times the upper limit of normal on eculizumab monotherapy, eculizumab plus APL-2 and APL-2 monotherapy, respectively

Catalyst Biosciences Inc., of South San Francisco

Marzeptacog alfa

Recombinant factor VIIa variant

Hemophilia A or B with or without inhibitors

Data published in the Journal of Thrombosis and Haemostasis showed the drug had linear dose-response pharmacokinetics across the 4.5-30 μg/kg dose range; drug produced a dose-dependent shortening of activated partial thromboplastin time and prothrombin time, as well as an increase in peak thrombin

Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa., and Geneone Life Science Inc., of Seoul, Korea

GLS-6150

Vaccine

Hepatitis C infection prophylaxis

Dosed first of 24 patients comparing immune responses of 2 doses of the vaccine to placebo; data expected in 2019

Kodiak Sciences Inc., of Palo Alto, Calif.

KSI-301

Anti-VEGF biopolymer conjugate

Diabetic macular edema

Completed enrollment in the study designed to test the ocular and systemic safety of the drug while determining the maximum tolerated dose

Noxxon Pharma NV, of Berlin

NOX-A12 (olaptesed pegol)

CXCL12 inhibitor

Metastatic, microsatellite stable pancreatic and colorectal cancer

Completed patient recruitment in the study testing NOX-A12 alone and in combination with Keytruda (pembrolizumab, Merck & Co.); data from patients treated with NOX-A12 monotherapy will be presented at the Fourth CRI-CIMT-EATI-AACR International Cancer lmmunotherapy Conference; data from patients treated with the combination will be available at the end of 2018

Pieris Pharmaceuticals Inc., of Boston

PRS-343

Bispecific monoclonal antibody targeting HER2 and 4-1BB

Advanced or metastatic HER2-positive solid tumors

Dosed first patient in study testing safety, tolerability and antitumor effects of PRS-343 plus the anti-PD-L1 drug Tecentriq (atezolizumab, Roche Holding AG)

Redx Pharma plc, of Alderley Park, U.K.

RXC-004

Porcupine inhibitor

Solid tumors

U.K.'s MHRA agreed, in principle, to the company's plan to restart the trial with a lower dose with subsequent dose escalation; new study protocol will also include enhanced safety monitoring

Synlogic Inc., of Cambridge, Mass.

SYNB-1618 

Metabolizes phenylalanine

Healthy volunteers and phenylketonuria

In healthy volunteers portion of the study, SYNB-1618 resulted in significant dose-dependent production of biomarkers associated with SYNB-1618 activity; top-line data from patients with phenylketonuria expected in mid-2019

Telix Pharmaceuticals Ltd., of Melbourne, Australia

TLX-101

Targeted radiopharmaceutical

Recurrent glioblastoma

Received ethics approval to start a phase I/II study

Therapeutics Solutions International Inc., of Oceanside, Calif.

Stemvacs 

Cellular immunotherapy

Advanced tumors

Completed study showing the treatment is safe; plans to make the treatment available under the Right to Try law

Zenith Epigenetics Ltd., of Calgary, Alberta

ZEN-3694

Small-molecule BET inhibitor

Metastatic castrate-resistant prostate cancer

Data from a case study involving a CURATE.AI profile showed the drug played a role in mediating low PSA levels and preventing disease progression; using the dosing suggested by the artificial intelligence platform, the combination of ZEN-3694 and enzalutamide resulted in the patient achieving a durable response now ongoing for > 16 months

Phase II

Abivax SA, of Paris

ABX-464

Increases expression of anti-inflammatory microRNA miR124

Modest to severe ulcerative colitis

Top-line phase IIa results showed drug was safe and well-tolerated, with significant efficacy based on both clinical and endoscopic endpoints; final 8-week data showed clinical remission of 35% for ABX-464 vs. 11% for placebo; mucosal healing was 50% vs. 11% for placebo; clinical response was 70% vs. 30% for placebo

Altimmune Inc., of Gaithersburg, Md.

Nasovax

Intranasal influenza vaccine

Influenza

New phase IIa data showed durable, dose-dependent protective immune responses, significant mucosal immune response 1 month after vaccination compared to both placebo and Fluzone, and a clean safety profile; an influenza-specific mucosal antibody (IgA) response was demonstrated at all dose levels, with the highest responses in the highest dose groups, while Fluzone and placebo groups demonstrated no response

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

BCX-7353 

Plasma kallikrein inhibitor

Hereditary angioedema

In 33 patients experiencing 95 hereditary angioedema attacks, BCX-7353 produced a 3.9-point decrease in the visual analogue scale through 4 hours compared to a 3.1-point increase for placebo-treated patients (p=0.0024); 64.1% and 32.3% of attacks treated with BCX-7353 and placebo, respectively, had no or mild symptoms through 24 hours (p=0.0038); data from treatment with lower doses are expected in the first quarter of 2019

Biophytis SA, of Paris

Sarconeos

Based on activation of the MAS receptor

Sarcopenia

Enrolled more than 200 of the 334 patients in the phase IIb SARA-OBS study, with 60 patients having passed the 6-month observational phase and ready to enter the interventional phase

Can-Fite Biopharma Ltd., of Petach Tikva, Israel

Namodenoson (CF-102)

Binds to A3 adenosine receptor

Advanced hepatocellular carcinoma

Top-line results are expected by year-end; study enrolled patients whose disease has progressed on sorafenib therapy

Neurorx Inc., of Wilmington, Del.

NRX-100 followed by oral NRX-101

Ketamine/fixed-dose combination of D-cycloserine and lurasidone

Severe bipolar depression with acute suicidal ideation and behavior

Top-line data from the STABIL-B trial demonstrated the drug was well-tolerated with no serious adverse events or discontinuations for side effects; statistically significant differences were seen between the NRX-101 and lurasidone groups

Opiant Pharmaceuticals Inc., of Santa Monica, Calif.

OPTN-001

Naloxone nasal spray

Bulimia nervosa

Completed patient enrollment; top-line data expected in the first quarter of 2019

Opko Health Inc., of Miami

Rayaldee (extended-release prohormone of calcitriol)

Active form of vitamin D3

Vitamin D insufficiency and stage 5 chronic kidney disease requiring hemodialysis

Initiated study; first cohort of about 44 patients will be treated for 26 weeks in randomized, open-label design to identify dose for second cohort

Zogenix Inc., of Emeryville, Calif.

ZX-008

Low-dose fenfluramine hydrochloride

Lennox-Gastaut syndrome

Data published in Epilepsia showed drug provided sustained, clinically meaningful seizure reduction in the majority of patients and was generally well-tolerated; patients achieved a 53% median reduction in convulsive seizure frequency during the 20-week treatment period of the core study; reduction in convulsive seizure frequency of at least 50% was seen in 62% of patients, with a reduction of at least 75% being reported in 23%

Phase III

Biogen Inc., of Cambridge, Mass.

BIIB-093
(I.V. glibenclamide)

High-affinity inhibitor of SUR1-TRPM4 channels

Severe cerebral edema in large hemispheric infarction

Enrolled the first patient in the CHARM study; trial will include 680 patients and evaluate treatment within 10 hours following stroke onset; primary endpoint is modified Rankin Scale, assessed at 90 days

Eyegate Pharmaceuticals Inc., of Waltham, Mass.

EGP-437

Reformulated topically active corticosteroid dexamethasone phosphate delivered via Eyegate II system

Non-infectious anterior segment uveitis

Top-line results showed clinical efficacy, defined as a reduction in anterior chamber cell score throughout the study, but did not demonstrate noninferiority to prednisolone acetate ophthalmic solution control group

Horizon Pharma plc, of Dublin

Teprotumumab

Fully human monoclonal antibody inhibiting insulin-like growth factor 1 receptor

Moderate to severe active thyroid eye disease

Completed enrollment in the confirmatory phase III trial; top-line results expected in the second quarter of 2019

Sage Therapeutics Inc., of Cambridge, Mass.

Zulresso (brexanolone)

Allosteric modulator of both synaptic and extrasynaptic GABAA receptors

Postpartum depression

Analysis published in The Lancet demonstrated significant and clinically meaningful reductions in HAM-D total score following treatment with brexanolone 90 mcg/kg/h at the primary timepoint of 60 hours vs. placebo; statistically significant improvement in the HAM-D total score was first observed within 24 hours of initiating treatment and treatment response was durable through 30-day follow-up

Notes

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