Clinical Catch-Up: September 6-10

Clinical Trial_Compressed

It was a very busy week for clinical trial announcements, although there were only three that were COVID-19-related. Here’s a look.


AzurRx BioPharma announced that an independent data monitoring committee approved starting patient enrollment in Part 2 of the ongoing RESERVOIR Phase II trial of FW-1022 for COVID-19-related GI infections. FW-1022 is an oral formulation of micronized niclosamide developed to remove SARS-CoV-2 from the GI tract. The company also reported it had activated seven new clinical trial sites in India for the RESERVOIR trial.

CytoDyn treated the first patient in its pivotal Phase III COVID-19 trial in Brazil for patients with severe symptoms. They will be treated with leronlimab, a CCR5 antagonist.

Bharat Biotech indicated its intranasal COVID-19 vaccine candidate will likely enter the Phase II part of its Phase II/III trial within the next few weeks. Phase I data was promising, showing it was well-tolerated and blocks viral entry into the body.


BioMarin Pharmaceutical announced the U.S. Food and Drug Administration (FDA) had placed a clinical hold on its BMN 307 Phearless Phase I/II study. The trial is investigating BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy in adults with phenylketonuria (PKU). The study was paused based on interim safety data from a preclinical, non-GLP pharmacology study that found liver tumors in laboratory animals receiving the therapy.

Horizon Therapeutics enrolled the first patient in a Phase IV trial of Tepezza for treatment of chronic (inactive) thyroid eye disease (TED). Tepezza (teprotumumab-trbw) is the only FDA-approved therapy for TED.

Adaptimmune Therapeutics updated data from its Phase I ADP-A2AFP trial for patients with liver cancer. They reported a complete response in one patients and tumor reductions with stable diseases that lasted more than 16 weeks in two patients, as well as disease control in most patients at the target dose.

AzurRx BioPharma submitted an Investigational New Drug (IND) application to the FDA for a clinical trial of its formulations of niclosamide for Grade 1 and 2 colitis and diarrhea in oncology patients receiving treatment with immune checkpoint inhibitors. The planned Phase Ib/IIa PASSPORT trial will test niclosamide as an oral, immediate-release tablet and a topical rectal enema foam formulation. The drug is currently in a Phase II RESERVOIR trial for COVID-19-related GI infections with a micronized niclosamide formulation. 

Motus GI Holdings announced GastroZentrum Lippe, a private endoscopy clinic in Germany, has enrolled patients into the EU outpatient study of the Pure-Vu System to study the clinical outcomes in patients with a history of poor bowel preparation using a low volume preparation with limited diet restrictions and the PureVu System prior to colonoscopy. The EU study will enroll about 44 patients.

Soleno Therapeutics announced topline data from its ongoing open-label extension study, C602, evaluating its once-daily DCCR (Diazoxide Choline) Extended-Release tablets for patients with Prader-Willi Syndrome. There were mean improvements in hyperphagia (extreme hunger) and statistically significant improvements in all behavioral domains.

Erasca and Pfizer inked a clinical trial collaboration and supply agreement for the BRAF inhibitor encorafenib (Braftovi). The deal will support a clinical proof-of-concept study of ERAS-007, an oral ERK1/2 inhibitor in combination with encorafenib and the EGFR inhibitor cetuximab for patients with BRAF V600E-mutant metastatic colorectal cancer (mCRC). It will part of the Phase Ib/II HERKULES-3 trial which is expected to start in the second half of 2021. They also announced they had dosed the first patient in the HERKULES-2 Phase I/B trial.

Gain Therapeutics announced topline data from its study conducted at the University of Maryland School of Medicine (UMSOM). It evaluated two lead Structurally Targeted Allosteric Regulators (STARs) compounds, GT-02287 and GT-02329, for the treatment of Gaucher and GBA1 Parkinson’s disease. The study demonstrated positive effects on all tested phenotypes.

Galera Therapeutics announced final results from its Phase I/II pilot trial of GC4419 in unresectable or borderline resectable locally advanced pancreatic cancer (LAPC) patients undergoing stereotactic body radiation therapy (SBRT). GC4419 is a dismutase mimetic. Improvements were observed in overall survival, progression-free survival, local tumor control and time to distant metastases.

EyeGate Pharmaceuticals completed target enrollment of 21 patients in its Phase II proof-of-concept study of PP-001 in patients with ocular surface inflammation due to ocular surface diseases including dry eye. PP-001 is an immune-modulating molecule, a dihydroorotate dehydrogenenase inhibitor.

Hutchmed and AstraZeneca initiated the SANOVO Phase III trial in China of Orpathys (savolitinib) in combination with AstraZeneca’s third-generation, irreversible EGFR TKI, Tagrisso (Osimertinib) as first-line treatment in certain non-small cell lung cancer patients whose tumors harbor EGFR mutation and overexpress MET. The first patient was dosed on September 7, 2021.

Sanofi announced that its Phase III PEGASUS clinical trial of rilzabrutinib for pemphigus failed to hit the primary or key secondary endpoints. Rilzabrutinib is an oral BTK inhibitor. Pemphigus is a rare, debilitating autoimmune disorder that results in blisters on the skin and mucous membranes. The primary endpoint of the trial was complete remission from weeks 29 to 37 with minimal doses of corticosteroids. The data shows that there was no significant difference between rilzabrutinib and placebo.

WAVE Life Sciences initiated dosing in its Phase Ib/IIa SELECT-HD trial of WVE-003 for Huntington’s disease. WVE-003 is a stereopure antisense oligonucleotide that targets SNP3, a single nucleotide polymorphism on the mutant huntingtin allele.

Viewpoint Molecular Targeting received the greenlight from the FDA for its Phase I imaging study of VM-alpha-NET for imaging of neuroendocrine tumors (NETs). The study will be run at the University of Iowa Hospitals and Clinics.

Genmab and AbbVie published results of the dose escalation part of the Phase I/II EPCORE NHL-1 first-in-human dose escalation and cohort expansion study of epcoritamab (DuoBody-CD3XCD20) in r/r B-cell non-Hodgkin’s lymphoma. No dose-limiting toxicities were observed and 48mg was identified as the recommended Phase II dose (RP2D).

MeiraGTx announced new data from subjects treated in the Phase I/II dose escalation phase of Study MGT009 indicating AAV5-RPGR reverses course of disease progression. The gene therapy is being developed for the treatment of X-linked retinitis pigmentosa (XLRP).

Dermavant Sciences dosed the first patient in its Phase III trial of tapinarof for atopic dermatitis. Tapinarof is a novel, therapeutic aryl hydrocarbon receptor modulating agent in development for plaque psoriasis and atopic dermatitis.

Denali Therapeutics commenced dosing in its Phase Ib trial of DNL343 for amyotrophic lateral sclerosis (ALS). The drug is a brain-penetrant small molecule activator of EIF2B.

Veru updated data from its Phase Ib/II trial of sabizabulin in 80 men with metastatic castration resistant prostate cancer who have progressed on at least one novel androgen receptor targeting agent. The drug is a new oral chemical entity that targets bindings sites of microtubules to disrupt the cytoskeleton and androgen receptor transport.

PDS Biotechnology completed enrollment of the lead-in safety cohort of its VERSATILE-002 Phase II trial of PDS0101 with Merck’s checkpoint inhibitor Keytruda (pembrolizumab) for cancers caused by HPV infections. PDS0101 uses PDS’s Versamune platform to combine targeted antigens in HPV-expressing cancers.

CG Oncology announced a clinical trial collaboration with Bristol Myers Squibb to evaluate their CG0070 in combination with BMS’s checkpoint inhibitor Opdivo (nivolumab) for metastatic urothelial cancer in a Phase I/II trial. CG0070 is an oncolytic immunotherapy.

Icoxavax initiated a Phase I/Ib trial of IVX-121, a VLP vaccine candidate against respiratory syncytial virus. The vaccine displays an RSV stabilized pre-fusion F antigen.

AstraZeneca announced that the MANDALA and DENALI Phase III trials of PT027 (albuterol/budesonide) at both 180/170mcg and 180/80mcg doses, met all primary endpoints, showing statistically significant benefits in patients with asthma compared to individual components albuterol. PT027 is a first-in-class inhaled, fixed-dose combination of albuterol, a short-acting beta2-agonist (SABA), and budesonide, an inhaled corticosteroid, which is being developed by AstraZeneca and Avillion.

Applied Therapeutics reported initial biomarker data from the pediatric ACTION-Galactosemia Kids study in addition to baseline analysis of the 47 children enrolled in the trial. The data demonstrated a substantial decrease in plasma galactitol of about 35% after 30 days, which was statistically significant compared to placebo.

Clene announced the final patient visit had been completed in its Phase II RESCUE-ALS trial. The trial evaluation CNM-AU8 in patients with early ALS. ZZCNM-Au8 is a catalytically-active gold nanocrystal suspension.

Sio Gene Therapies dosed the first Type 1 (early infantile) patient in the low-dose cohort of its ongoing Phase I/II trial of AXO-AAV-GM1 for GM1 gangliosidosis. The therapy is an adeno-associated viral vector 9-based gene therapy.

Apellis Pharmaceuticals reported topline results from its Phase III DERBY and OAKS clinical trials for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The studies evaluated pegcetacoplan. OAKS hit the primary endpoint for both monthly and every-other-month treatment. DERBY missed the primary endpoint of GA lesion growth. GA is an advanced type of age-related macular degeneration. It is a leading cause of blindness. GA lesions occur in the central part of the retina, called the macula, which handles central vision. The lesions in GA are driven by excessive complement activation, a part of the immune system, and C3 is the only target for precisely controlling complement overactivation. Pegcetacoplan is a targeted C3 therapy.

scPharmaceuticals presented data from its recently completed FREEDOM-HF study of subcutaneous Furoscix in heart failure patients with mild to moderate volume overload. The data showed the therapy resulted in significantly reduced HF-related and overall healthcare costs via reduction of HF-related hospitalizations and rehospitalizations.

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