Company

Product

Description

Indication

Status

Phase I

Briacell Therapeutics Corp., of Berkeley, Calif.

Bria-IMT

Immunotherapy

Advanced breast cancer

Updates from phase I/IIa trial in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) showed the top responder, who previously failed 12 prior regimens with 16 agents, experienced highly remarkable reduction in breast cancer tumors in the adrenal gland and outer lining of the brain; data from that patient added to company's 'matching hypothesis,' having matched Bria-IMT at 2 HLA loci

Compugen Ltd., of Holon, Israel

COM-701

Checkpoint inhibitor

Solid tumors

Completed enrollment in seventh dose level in the monotherapy (Arm A) and second dose level in the combination of COM7-01 with Opdivo (nivolumab, Bristol-Myers Squibb Co.) (Arm B), with no dose-limiting toxicities observed in those and prior dose level cohorts

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China

CS-1001

Anti-PD-L1 antibody

Esophageal squamous cell carcinoma

Results from phase Ia/Ib study from ESCC cohort receiving CS-1001 as first-line treatment combined with cisplatin and 5-fluorouracil showed objective response rate of 77.8%; 14 of 18 evaluable patients achieved partial response (PR) per RECIST 1.1; among those who achieved PR, 11 remained on treatment and 3 discontinued due to adverse events; all 14 have been progression-free; median duration of response not reached

Nanobiotix SA, of Paris

NBTXR-3

Hafnium oxide nanoparticles

Hepatocellular carcinoma

To date, 62.5% of evaluable patients have shown a complete response while the rest have shown partial response; results show that NBTXR-3 activated by stereotactic body radiation therapy was safe and well-tolerated at each of the 4 dose-escalation levels; compound remained in the tumor from injection through the end of radiation therapy without leakage to surrounding healthy tissue

PDS Biotechnology Corp., of Berkeley Heights, N.J.

PDS-0101

Combination of Versamune nanoparticles plus human papillomavirus-16 E6 and E7 antigens

Cervical intraepithelial neoplasia

Limited available clinical outcome data from previously reported phase I study found 60% of evaluable patients had a clinical response; drug was immunologically active at all 3 doses and resulted in increase (5- to 73-fold) in circulating HPV disease-attacking T cells in 10/12 subjects

Trovagene Inc., of San Diego

Onvansertib

Oral PLK1 inhibitor

Acute myeloid leukemia

Phase Ib dose-escalation trial confirmed onvansertib in combination with standard-of-care chemotherapy is safe and well-tolerated; primary efficacy endpoint of objective response was achieved in 5 of 21 patients; 30% of patients were biomarker positive, which was associated with an increase in response to treatment as measured by decreases in bone marrow blasts and the rate of complete response

Phase II

Aslan Pharmaceuticals Pte. Ltd., of Singapore

Varlitinib

EGFR/ErbB-2 (HER2/neu) inhibitor

Biliary cancer

Patients treated with varlitinib plus capecitabine in second-line achieved a response rate of 11%, including 2 complete responses; median progression-free survival of 2.7 months and overall survival of 5.8 months

Cindome Pharma Inc., of Cincinnati

CIN-102

Deuterated domperidone; dopamine D2 receptor antagonist

Gastroparesis

Enrolled first patient

Dance Biopharm Holdings Inc., of Durham, N.C.

Dance-501

Second-generation aerosol inhalant formulation of human insulin

Non-insulin-dependent diabetes

Had comparable pharmacodynamic properties and more rapid onset of action compared to insulin lispro, with median differences of 6.5 to 20 minutes (p<0.02)

Foamix Pharmaceuticals Ltd., of Rehovot, Israel

FCD-105

Combination of minocycline and adapalene, as a topical foam

Acne vulgaris

First patient enrolled out of planned 400 participants, ages 12 and older, with moderate to severe acne vulgaris; testing vs. adapalene, minocycline and vehicle foam; primary endpoint is proportion of patients achieving "clear" or "minimal" IGA rating at 12 weeks

India Globalization Capital Inc., of Bethesda, Md.

IGC-AD1

Liquid formulation of tetrahydrocannabinol and an amyloid-beta 40 protein production inhibitor

Alzheimer's disease

Institutional review board in Puerto Rico approved its protocol for a double-blind, placebo-controlled, efficacy, safety and tolerability study; company will next apply for IND

Spruce Biosciences Inc., of San Francisco

Tildacerfont

Oral, once-daily corticotropin-releasing factor type-1 antagonist

Congenital adrenal hyperplasia

Phase IIa study in patients with classic CAH accompanied by elevated androgens at baseline showed clinically relevant reductions in all parameters; mean reductions from baseline of 74% for adrenocorticotropic hormone (ACTH), 82% for 17-hydroxyprogesterone and 55% for androstenedione (A4); 60% of patients with elevated, abnormal ACTH and 40% of patients with elevated A4 saw reductions to normalization by week 12

Tetra Therapeutics Inc., of Grand Rapids, Mich.

BPN-14770

PDE4D inhibitor

Early Alzheimer's disease

Surpassed 50% enrollment in Picasso AD trial

Trovagene Inc., of San Diego

Onvansertib

Oral PLK1 inhibitor

Acute myeloid leukemia

Initiated enrollment of 32 patients who are either treatment-naïve and not candidates for induction therapy or who have relapsed following up to 1 prior regimen, to receive onvansertib in combination with decitabine; primary endpoint of objective response will be assessed in patients who complete at least 1 cycle

Xeris Pharmaceuticals Inc., of Chicago

Fixed-ratio co-formulation of pramlintide and insulin

Synthetic hormone resembling amylin plus insulin

Type 1 diabetes

Began dosing in open-label, crossover study in 18 adults; primary objective is to evaluate pharmacodynamic and pharmacokinetic properties of single injection vs. single doses of regular insulin and regular insulin plus pramlintide co-administered as separate injections; data expected in first half of 2020

Phase III

Avexis, unit of Novartis AG, of Basel, Switzerland

Zolgensma (onasemnogene abeparvovec-xioi)

Gene therapy

Spinal muscular atrophy type 1

Interim data from Spr1nt trial in presymptomatic patients demonstrated that, of 22 patients evaluated overall, all were alive and free of permanent ventilation; all patients with 2 copies of SNM2 achieved or maintained Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders score of greater than 50, with 7 achieving score of greater than or equal to 60 and 5 reaching maximum score of 64; of patients with 2 copies of SNM2, 6 (60%) were able to sit without support for at least 30 seconds at an average age of 7.6 months

Avexis, unit of Novartis AG, of Basel, Switzerland

Zolgensma (onasemnogene abeparvovec-xioi)

Gene therapy

Spinal muscular atrophy type 1

Interim results from long-term follow-up of Start study showed, as of May 31, all 10 were alive and continued to maintain developmental milestones; 2 patients, neither of whom received nusinersen treatment following Zolgensma infusion, gained the ability to stand with assistance; 2 patients previously were reported to be walking independently; all patients have maintained or demonstrated improvements in ventilatory status; 6 of 10 do not require daily respiratory support

Innovent Biologics Inc., of Suzhou, China

IBI-305

Biosimilar of anti-VEGF antibody bevacizumab

Advanced non-small-cell lung cancer

Results of study vs. bevacizumab, both in combination with paclitaxel/carboplatin, in first-line treatment showed, by cutoff date of March 31, overall response rates were 47.1% and 46.8% for IBI-305 and bevacizumab in full analysis set, respectively; ORR rate was 1.01, within predefined equivalence margin; investigator-evaluated median progression-free survival was 7.3 months and 7.5 months in the IBI-305 and bevacizumab groups, respectively, with no statistical difference (p=0.893)

Micurx Pharmaceuticals Inc., of Shanghai and Foster City, Calif.

Contezolid (MRX-1)

Oral oxazolidinone antibiotic

Complicated skin and soft tissue infection

Met primary endpoint of noninferiority vs. linezolid for clinical cure rate at test-of-cure visit (7-14 days after last dose), and was associated with fewer drug-related hematologic laboratory adverse events

Rafael Pharmaceuticals Inc., of Cranbury, N.J.

CPI-613 (devimistat)

Targets mitochondrial tricarboxylic acid cycle

Relapsed or refractory acute myeloid leukemia

Trial in older patients now active and enrolling in South Korea; study testing efficacy and safety in combination with high-dose cytarabine and mitoxantrone

Strongbridge Biopharma plc, of Dublin

Recorlev (levoketonconazole)

Cortisol synthesis inhibitor

Endogenous Cushing's syndrome

Data showed Sonics study, published in The Lancet, met primary endpoint, with 30% of patients achieving mean urinary free cortisol normalization at end of maintenance phase among intent-to-treat population, without a dose increase (p=0.015 vs. null hypothesis of ≤20%)

Phase IV

Mannkind Corp., of Westlake Village, Calif.

Afrezza

Inhaled insulin

Type 1 diabetes

Data from 1-year study comparing Afrezza to rapid-acting injected insulin analogue showed Afrezza provided significantly better glucose control in first 2 hours following a standardized meal; titration of Afrezza to about 1.5 to 2 times the unit dose of injected insulin aspart resulted in significantly lower post-meal glucose excursions and was associated with lower rates of overall and level 2 hypoglycemia

Novo Nordisk A/S, of Bagsvaerd, Denmark

Tresiba

Insulin degludec

Type 2 diabetes

Data from head-to-head Conclude study showed overall lower risk of hypoglycemia at significantly lower HbA1c vs. insulin glargine U300 in adults whose disease is uncontrolled on basal insulin with or without oral diabetic drugs

Notes

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