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Clinical Catch-Up: July 26-30


With the Alzheimer’s Association International Conference (AAIC) conference last week, there were plenty of clinical trial news and updates. Here’s a look.


Claritas Pharmaceuticals reported that a study led by clinicians at Royal Brompton & Harefield NHS Foundation Trust validated the potential of its nitric oxide releasing compound, R-107, as a possible treatment for coronavirus and COVID-19 infection. In the study, inhaled nitric oxide significantly improved oxygen levels given to patients with severe COVID-19 pneumonia.

Moderna announced that as it continues its study of its COVID-19 vaccine in children ages 5 to 11, it is expanding the study to include evaluations of rare side effects, including heart inflammation (myocarditis and pericarditis). This will likely push back EUA for this population until the end of this year or early 2022.

eFFECTOR Therapeutics dosed the first patient in a Phase Ib trial of zotatifin in mild to moderate COVID-19. Zotatifin is a potent and sequence-selective inhibitor of eukaryotic translation initiation factor 4A mediated translation.


Abeona Therapeutics announced magnetic resonance imaging (MRI) data from its Phase I/II Transpher A trial in San Filippo Syndrome Type A (MPS IIIA). MPS IIIA is a rare, fatal disease of lysosomal storage. ABO-102 is a novel gene therapy. It is a one-time intravenous infusion. It uses a self-complementary AAV9 vector to transport a functional copy of the SGSH gene to CNS and peripheral organ tissues. The MRI data demonstrated that ABO-102 increased grey matter, corpus callosum and amygdala volumes in the brain. The results were in three pediatric patients with MPS IIIA. The results were observed at 24 months and compared to afflicted patients who did not receive the treatment.

INmune Bio announced the design of its upcoming Phase II trial of XPro in patients with mild Alzheimer’s disease. The announcement came with the release of additional biomarker data from the Phase Ib AD trial that demonstrated improvement in white matter (myelinated axons) that degenerate in AD patients. XPro or Xpro1595 is a next-generation inhibitor of tumor necrosis factor (TNF) that uses a dominant-negative TNF technology. Pegipanermin neutralizes soluble NTF without affecting trans-membrane TNF or TNF receptors.

Allergan, an AbbVie company, announced new data, including the full results of the Phase III GEMINI 1 trial of AGN-190583 (pilocapine 1.25%) ophthalmic solution for presbyopia. The drug met both its primary and key secondary efficacy endpoints, with patients hitting near and intermediate vision gains.

Biogen and Ionis Pharmaceuticals announced topline results from a Phase Ib clinical trial of BIIB080/MAPTRX. The drug is an investigational antisense therapy that targets microtubule-associated protein tau (MAPT) mRNA and prevents production of tau protein. The Phase Ib study of BIIB080/MAPTRX hit the primary objective of safety and tolerability in mild Alzheimer’s. It also showed robust time and dose dependent decreases of tau protein in cerebrospinal fluid (CSF) in these patients over the three-month treatment period as well as sustained reductions in the six-month post-treatment period.

Patients receiving the drug demonstrated dose-dependent decreases in total-tau concentration in CSF eight weeks after the last dose with a mean percentage reduction of 30%, 40% and 49% in the low, medium and high dose cohorts who were treated every four weeks, and 42% in the cohort treated every 12 weeks.

Merck announced positive data from the KEYNOTE-355 Phase III trial of Keytruda (pembrolizumab) with chemotherapy in metastatic triple-negative breast cancer. The combination demonstrated a statistically significant and clinically meaningful improvement in overall survival compared to chemotherapy alone.

BioVie presented data from a Phase III trial of NE3107 in Alzheimer’s. NE3107 is an oral small molecule, blood-brain permeable anti-inflammatory insulin sensitizer that binds ERK. The data highlighted its safety profile and broad mechanism of action against features of Alzheimer’s disease.

Genentech, a Roche company, published data from FIREFISH Part 2, a global study of Evrysdi (risdiplam) in babies ages 1-7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). It hit the primary endpoint with 29% sitting without support for at least five seconds by month 12.

Satsuma Pharmaceuticals randomizedthe first patient in its SUMMIT Phase III trial of STS101 for acute treatment of migraine. STS101 is proprietary nasal powder formulation of the anti-migraine drug dihydroergotamine mesylate (DHE).

ALX Oncology and Merck dosed the first patient in the Phase II ASPEN-04 trial of ALX148 and Merck’s Keytruda (pembrolizumab) and standard chemotherapy for advanced head and neck squamous cell carcinoma. ALX148 is a next-generation CD47 blockers. Keytruda is an anti-PD-1 checkpoint inhibitor.

Arcturus Therapeutics received approval from the UK Health Research Authority to start a Phase II trial of ARCT-810 for Ornithine Transcarbamylase (OTC) Deficiency. ARCT-810 is a novel mRNA-based therapeutic candidate.

Progenity announced successful completion of the validation study of its Preecludia rule-out test for preeclampsia. The PRO-104 study was a prospective, multi-center, observational study with more than 1,300 enrolled subjects. Test specimens were collected from pregnant patients aged 18 to 45 years and 28 0/7 to 36  6/7 weeks’ gestational age.

Eli Lilly and Company presented data analysis from the Phase II TRAILBLAZER-ALZ study that supports the argument that decreases in beta-amyloid in Alzheimer’s disease slows cognitive decline. Lilly’s data is two exploratory analyses from the TrailBLAZER-ALZ trial of their own monoclonal antibody, donanemab. It targets a modified form of beta-amyloid plaque known as N3pG. In one analysis, they found that larger changes to amyloid plaques after treatment with donanemab was “highly associated with less cognitive decline.” In addition, the patients with the most plaque clearance at 24 weeks also had less progression of yet another abnormal protein found in Alzheimer’s patients, tau. The second analysis demonstrated that donanemab resulted in a rapid decrease of a biomarker that is associated with Alzheimer’s pathology, plasma P-tau217, which was observed within 12 weeks.

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