Phase I

Aileron Therapeutics Inc., of Watertown, Mass.


Dual MDM2/MDM4 protein inhibitor

Small-cell lung cancer

First participant completed initial treatment cycle in first trial evaluating drug as myelopreservation agent in those with p53-mutant disease treated with topotecan; study will enroll up to 40 participants and use standard gene tests in biomarker-based selection strategy to identify recommended phase II dose

AL-S Pharma AG, unit of Neurimmune Holding AG, of Schlieren, Switzerland


Superoxide dismutase 1 inhibitor

Amyotrophic lateral sclerosis

First participant enrolled in trial evaluating safety, tolerability and pharmacokinetics

Amphivena Therapeutics Inc., of South San Francisco


Dual CD3/CD33 modulator

Solid tumors

First participant dosed in all-comers, dose-finding trial, with expansion cohorts planned in 2020

Aura Biosciences Inc., of Cambridge, Mass.


Light-activated viral-like particle bioconjugates

Choroidal melanoma

Updated data from ongoing phase Ib/II trial showed multiple administrations of treatment were well-tolerated; in subset of patients with documented tumor growth prior to enrollment (n=17), treatment resulted in tumor control in 15 (88%; p=0.0117), informing design and conduct of planned phase III registration trial

Destiny Pharma plc, of Brighton, U.K.

XF-73 (exeporfinium chloride)

Porphyrin-based compound

Staphylococcus aureus

Data, reported in the Journal of Global Antimicrobial Resistance, showed favorable safety and local tolerability profile of nasal gel formulation, meeting primary objective; exposure to study drug produced rapid reduction in levels of nasal S. aureus in all participants

Exicure Inc., of Chicago


SNA targeted to mRNA encoding interleukin-17 receptor-alpha


Treatment resulted in decrease in levels of psoriasis and inflammation markers downstream of the IL-17RA target; statistically significant reduction in keratin 16 expression, a key marker of psoriasis (p=0.002); reductions in inflammatory markers beta defensin 4A, IL-19 and IL-36A vs. psoriatic skin at baseline

Hepagene Therapeutics Inc., of Shanghai


Farnesoid X receptor modulator

Nonalcoholic steatohepatitis

First healthy volunteer dosed in single- and multiple ascending-dose study assessing safety, tolerability, pharmacokinetics, pharmacodynamics and food effect

Hope Biosciences Inc., of Houston


Autologous adipose-derived mesenchymal stem cells

Traumatic brain injury; hypoxic-ischemic encephalopathy

Opened single-arm phase I/II study expected to enroll 24 participants, with safety and treatment effect of cell therapy administered via 3 intravenous infusions over 6-week period as primary objective; follow-up evaluations will occur 6 and 12 months post-infusion

Miragen Therapeutics Inc., of Boulder, Colo.


MicroRNA-92 inhibitor

Vascular disease

New data from 2 trials in healthy volunteers showed single and multiple doses increased angiogenesis, based on increased perfusion and histological markers of neoangiogenesis, and reduced alpha-smooth muscle actin expression, correlated with activation of myofibroblasts; in intradermal multiple ascending-dose study, mean granulation tissue area decreased in treated individuals compared to those who received placebo to treat wounds

Nektar Therapeutics, of San Francisco


IL-15 receptor agonist

Non-Hodgkin lymphoma; multiple myeloma

Initiated study of NKTR-255 as monotherapy in relapsed or refractory (r/r) disease; study also will evaluate combination with multiple targeted antibodies that function through antibody-dependent cell-mediated cytotoxicity mechanism, including daratumumab (Darzalex, Genmab A/S and Johnson & Johnson) in adults with r/r MM

Noxxon Pharma NV, of Berlin


CXCL12 inhibitor

Brain cancer

First participant with newly diagnosed disease enrolled and treated in phase I/II dose-escalation study in combination with external-beam radiotherapy assessing safety and initial efficacy to define recommended phase II dose

Sernova Corp., of London, Ontario

Cell Pouch

Cell therapy

Type 1 diabetes

Enduring levels of C-peptide, measured up to 30 days and ongoing, as biomarker of transplanted beta cell insulin production detected in bloodstream of first participant in ongoing phase I/II study

Phase II

Atossa Genetics Inc., of Seattle

Fulvestrant delivered via intraductal microcatheter technology

Anti-estrogen treatment

Early stage breast cancer or ductal carcinoma in situ

Institutional review board approved study testing delivery directly into breast milk ducts in patients scheduled for mastectomy or lumpectomy within 14 to 45 days; primary endpoint will compare safety, tolerability and distribution of fulvestrant in comparison to intramuscular injection

Axsome Therapeutics Inc., of New York

AXS-05 (dextromethorphan/bupropion modulated delivery tablet)

NMDA receptor antagonist with multimodal activity

Major depressive disorder

Completed enrollment in Gemini study; top-line results expected in fourth quarter of 2019

Boehringer Ingelheim GmbH, of Ingelheim, Germany


Inhaled epithelial sodium channel inhibitor

Cystic fibrosis

First patient enrolled in Balance-CF-1 trial to test different doses on the impact of lung function vs. placebo when added to standard of care in adults and adolescents

Imbrium Therapeutics LP, of Stamford, Conn.


Small molecule

Insomnia associated with alcohol cessation

First dose administered in study in adults with moderate or severe alcohol use disorder who are experiencing IAAC; primary endpoint is change from baseline of wakefulness after sleep onset, as measured by polysomnography

Polypid Ltd., of Petah Tikva, Israel


Prolonged and constant release of broad-spectrum antibiotic doxycycline

Prevention of surgical site infections in abdominal surgery

Top-line results from 201-patient study showed statistically significant decrease in surgical site infections (SSIs) of 57% vs. standard of care alone (p<0.0136) in intent-to-treat population; in per-protocol population, treatment achieved statistically significant decrease in SSIs of 68% vs. SoC alone (p<0.0024)

Phase III

Aurinia Pharmaceuticals Inc., of Victoria, British Columbia


Semisynthetic structural analogue of cyclosporine

Lupus nephritis

Last patient study visit has occurred in Aurora trial; data on track to be reported by end of the fourth quarter 2019

Eli Lilly and Co., of Indianapolis


Pegylated IL-10

Metastatic pancreatic cancer

Top-line results showing Sequoia trial, testing combination with FOLFOX vs. FOLFOX alone in patients whose disease had progressed during or following first-line gemcitabine-containing regimen, did not meet primary endpoint of overall survival


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


No Comments