Phase I

Arvinas Inc., of New Haven, Conn.

ARV-110

Androgen receptor-targeted protein degrader

Metastatic castration-resistant prostate cancer

Started treatment of patients in the open-label study that's expected to enroll 28-36 patients; prostate specific antigen levels, androgen receptor degradation, radiographic measurements of evaluable lesions and other exploratory markers of disease burden will be measured; preliminary data expected in second half of 2019

Glycotope GmbH, of Berlin

Gatipotuzumab

Anti-TA-MUC1 antibody

Solid tumors such as metastatic colorectal, lung, breast and head and neck cancers with measurable disease after failure of standard treatment options

Based on preliminary data from first 20 patients, Gatto study testing gatipotuzumab with either tomuzotuximab or a marketed anti-EGFR antibody was expanded

Proteostasis Therapeutics Inc., of Boston

PTI-801, PTI-808 and PTI-428

CFTR corrector, CFTR potentiator and CFTR amplifier

Cystic fibrosis

In a per protocol analysis of 27 patients in the 600-mg PTI-801 cohort, there was a mean absolute increase in ppFEV1 at day 14 of 5 percentage points versus baseline (p<0.001) and a decrease in sweat chloride concentration at day 14 of -19 mM compared to baseline (p<0.00001) and -24 mM compared to placebo (p<0.001); adjusting for patients predisposed to rapid pulmonary decline, ppFEV1 increased 6 percentage points versus baseline (p<0.005), and 8 percentage points versus baseline compared to placebo (p<0.05); 400 mg PTI-801 plus 300 mg PTI-808 produced a mean improvement in sweat chloride of -20 mM vs. baseline (p<0.0005); plans to test PTI-801 and PTI-808 doublet as well as triplet in phase II with enrollment expected to be completed in 2019

Sarepta Therapeutics Inc., of Cambridge, Mass.

AAVrh74.MHCK7.Micro-dystrophin

Gene therapy expressing micro-dystrophin

Duchenne muscular dystrophy

In open-label study in 4 patients, 81.2% of fibers on average were positive for dystrophin; North Star Ambulatory Assessment improved by an average of 6.5 points from baseline to day 270; time to rise improved by an average of 0.8 seconds; 4 stair up improved by an average of 1.2 seconds; 100 meter test improved by 7.95 seconds on average; creatine kinase significantly decreased from baseline

Sojournix Inc., of Waltham, Mass.

SJX-653

NK3 antagonist

Healthy adult men (eventually vasomotor symptoms)

Subjects treated with 15 mg had a 57% reduction in luteinizing hormone (LH) and a 55% reduction in total testosterone (T); maximum LH reduction of 70% at 30 mg and maximum T reduction of 68% at 60 mg; pharmacokinetic profile supports once-daily dosing

Phase II

Alkahest Inc., of San Carlos, Calif.

AKST-4290 (formerly ALK-4290)

CCR3 antagonist

Neovascular age-related macular degeneration

In the open-label ALK4290-201 study, 83% of patients' eyes had maintenance or improvement of Best Corrected Visual Acuity, with a mean of +7 letters gained; 21% of patients gained 15 or more letters

Menlo Therapeutics Inc., of Redwood City, Calif.

Serlopitant

NK1 receptor antagonist

Pruritus associated with prurigo nodularis

Data for the 128-patient study published in the Journal of the American Academy of Dermatology showed the least squares mean difference (serlopitant minus placebo) for average itch visual analog scale was -1.0 at week 4 (p=0.02) and -1.7 at week 8 (p<0.001)

Pharmamar SA, of Madrid

Lurbinectedin

RNA polymerase II inhibitor

Relapsed small-cell lung cancer

The 105-patient single-arm study met primary endpoint of overall response rate by investigator review and independent committee review; data to be presented at a future medical meeting

Spruce Biosciences Inc., of San Francisco

Tildacerfont

CRF1 receptor antagonist

Congenital adrenal hyperplasia

In patients treated for 2 weeks with 200, 600 and 1,000 mg once per day (6 weeks total), 10 of 10 patients had improvements in androstenedione (A4) and 8 of 10 patients had improvements in 17-hydroxyprogesterone (17-OHP) and adrenocorticotropic hormone (ACTH); in patients treated for 2 weeks with 200 mg twice per day, A4 improved in 6 of 8 patients, 17-OHP improved in 6 of 7 patients and ACTH improved in 5 of 7 patients

Theravance Biopharma Inc., of Dublin

Ampreloxetine (TD-9855)

Norepinephrine reuptake inhibitor

Neurogenic orthostatic hypotension

20-week data support previously announced clinical observations after 4 weeks of treatment

Vaccibody AS, of Oslo, Norway

VB-10.16

DNA vaccine against HPV16

High grade cervical intraepithelial neoplasia

Of 14 patients in study for a year, 12 had reductions in the lesion size and 8 had more than a 50% reduction in lesion size; 8 of 14 patients had negative HPV16 in 1 or both of the 2 tests at 12 months; 8 patients had histopathological regression to low grade neoplasia or no disease, while the 6 other patients without regression had upregulation of PD-L1 in the lesions; plans to start a phase II study in second half of 2019

Xeris Pharmaceuticals Inc., of Chicago

RTU glucagon

Room-temperature stable liquid glucagon

Congenital hyperinsulinemia

In a 4-patient study, drug produced reductions in glucose infusion rates (GIR) of 53%-65% compared to a 31% reduction to a 34% increase for those taking placebo; during open-label period, drug produced reductions of 44%-66%, relative to baseline GIR

Phase III

Aimmune Therapeutics Inc., of Brisbane, Calif.

AR-101

Oral immunotherapy

Peanut allergy

Proportion of patients treated with AR-101 for approximately 9 months who tolerated a 1,000-mg dose of peanut protein was significantly higher than in the placebo group (p<0.00001); median-tolerated dose of peanut protein for patients treated with AR-101 improved 100-fold, from 10 mg at baseline to 1,000 mg at end of treatment; data to be presented at the European Academy of Allergy and Clinical Immunology Congress; company plans to submit MAA in mid-2019

Alzheon Inc., of Framingham, Mass.

ALZ-801

Oral anti-amyloid

Alzheimer's disease

Plans to start phase III study later this year

Ascendis Pharma A/S, of Copenhagen, Denmark

Transcon Growth Hormone

Transiently conjugated growth hormone

Pediatric growth hormone deficiency

Over the 1-year study period, drug had comparable safety and tolerability compared to daily Genotropin and a significantly greater increase in annualized height velocity

Avanir Pharmaceuticals Inc., of Aliso Viejo, Calif.

AVP-786 (deudextromethorphan hydrobromide/quinidine sulfate)

CYP2D6 inhibitor

Moderate-to-severe agitation in patients with Alzheimer's dementia

One dose produced a significant improvement on the Cohen-Mansfield Agitation Inventory while the other dose showed a numerical improvement that wasn't significant; data to be published in a peer-reviewed journal

Cel-Sci Corp., of Vienna, Va.

Multikine (leukocyte interleukin)

Cancer immunotherapy

Advanced primary head and neck cancer

Study hasn't reached 298 events required to end the study despite published data predicting they would have occurred already

Novo Nordisk A/S, of Bagsvaerd, Denmark

Oral semaglutide

GLP-1 receptor agonist

Type 2 diabetes

In the Pioneer 3 study ,7 mg and 14 mg produced HbA1c reductions of 1.0% and 1.3% at 26 weeks, compared to a 0.8% reduction for patients treated with sitagliptin (p<0.001 for both); 3-mg dose produced a 0.6% reduction (p=0.09 for non-inferiority); 7 mg and 14 mg produced weight reductions of 2.2 kg and 3.1 kg, compared to a 0.6 kg reduction for sitagliptin (p<0.01 for both)

Onconova Therapeutics Inc., of Newtown, Pa.

Rigosertib

Targets RAS effector pathways

High-risk myelodysplastic syndromes

Surpassed 75% of expected enrollment of 360 patients, putting it on track to complete enrollment in second half of 2019

Radius Health Inc., of Waltham, Mass.

Abaloparatide

Parathyroid hormone-related protein analog

Osteoporosis in postmenopausal women

Post-hoc analysis of patients who also had osteoarthritis showed significant reduction in new vertebral fractures in patients treated with drug compared to placebo; bone mineral density increased from baseline in patients treated with abaloparatide versus placebo at the total hip (mean change 3.17% vs. -0.35%), femoral neck (2.81% vs. -0.36%) and lumbar spine (8.78% vs 0.86%) (p<0.0001)

Strongbridge Biopharma plc, of Dublin

Recorlev (levoketoconazole)

Cortisol synthesis inhibitor

Cushing's syndrome

In the Sonics study, patients with and without diabetes had similar mean urinary-free cortisol (mUFC) normalization; patients with diabetes had improvements in HbA1c and anti-diabetic medications were more often decreased than increased; in overall population, late night salivary cortisol was improved, but not as commonly normalized as mUFC

Notes

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