Clinical Catch-Up: June 14-18
It was another busy week of clinical trial announcements. Here’s a look.
Aqualung Therapeutics completed a successful Type B meeting with the U.S. Food and Drug Administration (FDA) around IND-enabling and early human studies for ALT-100 (humanized mAb) to treat ARDS and ventilator-induced lung injury (VILI) in COVID-19 patients. They are now planning to move ahead with a Phase 0 study in ARDS patients. A Phase 0 trial is an exploratory IND study with the goal of quickly determining if a compound will work as desired in humans.
Seqirus co-authored the first study with Novavax of a Phase III trial of NVX-CoV2373, a COVID-19 vaccine co-administered with a seasonal influenza vaccine. NVX-CoV2373 is a recombinant protein COVID-19 vaccine candidate.
Celltrion Group presented top-line efficacy and safety data from the Phase III trial of regdanvimab in COVID-19. The drug is an anti-COVID-19 monoclonal antibody. It met all primary and key secondary endpoints in mild- to- moderate symptoms of COVID-19.
G1 Therapeutics initiated the Phase II PRESERVE 3 trial of Cosela (trilaciclib) with first-line platinum-based chemotherapy with checkpoint inhibitor avelumab maintenance therapy in untreated, locally advanced or metastatic urothelial carcinoma (mUC). Myeloprotection and anti-tumor efficacy are the endpoints.
FORMA Therapeutics announced new data from its ongoing Phase I trial of etavopivat in sickle cell disease The data showed the drug provided improved and sustained hematologic and hemolytic parameters. The drug is a novel selective red blood cell (RBC) pyruvate kinase-R (PKR) activator.
EyeGate Pharmaceuticals dosed the first patient in its Phase II proof-of-concept study of PP-001 for ocular surface inflammation. The drug is an immune-modulating molecule and an inhibitor of dihydroorotate dehydrogenase (DHODH).
GeneCast plans to launch a clinical trial for the early diagnosis of non-small cell lung cancer (NSCLC). It will evaluate the results of liquid biopsy-based EGFR testing with GENECAST’s ADPS technology in hopes it can identify the clinical effects of liquid biopsies on early cancer detection.
TILT Biotherapeutics submitted an IND for its Phase I trial of TILT-123 in ovarian cancer. TILT-123 is a double cytokine armed oncolytic adenovirus. It will be dosed with Merck’s checkpoint inhibitor Keytruda (pembrolizumab) in a dose escalation trial in platinum resistant or refractory ovarian cancer patients.
Eli Lilly and Company will be running a head-to-head trial comparing once-monthly injectable Emgality (galcanezumab-gnlm) with Nurtec ODT (Rimegepant) for prevention of migraine. Nurtec is an orally disintegrating tablet taken every other day. CGRP is a protein in the brain believed to play a major role in migraine. Emgality blocks CGRP receptors.
Atrallus presented clinical data on AT-01, a first-in-class pan-amyloid imaging agent. The data demonstrated that PET and CT imaging using AT-01 enabled detection of seven types of amyloids, ATTR2t, ATTRv, AL, ALys, AGel, AAPoA1 and ALECT2 and identified amyloid in anatomic sites not detected clinically, including pre-symptomatic cardiac amyloid.
ORYZON Genomics received the go-ahead from the FDA for its Phase IIb PORTICO trial of vafidemstat in patients with Borderline Personality Disorder (BPD). The drug is an oral, CNS optimized LDS1 inhibitor.
Otonomy initiated an expansion study of its Phase I/II trial of OTO-413 in patients with speech-in-noise hearing difficulty. It will enroll about 30 hearing loss patients, with about 20 being treated with a single intratympanic injection of OTO-413 and 10 receiving placebo. OTO-413 is a sustained-exposure formulation of brain-derived neurotrophic factor (BDNF), a naturally occurring protein involved in neuron growth and repair.
Salarius Pharmaceuticals initiated a Phase I/II trial of seclidemstat for hematologic cancers. The drug is a novel lysine specific demethylase 1 (LSDA) inhibitor. It will be conducted by The University of Texas MD Anderson Cancer Center.
VectivBio Holding dosed the first patient in its Phase II STARS Nutrition metabolic balance study of apraglutide in colon-in-continuity (CIC) patients with short bowel syndrome with intestinal failure (SBS-IF). Apraglutide is a next-generation, long-acting synthetic GLP-2 analog.
Clearside Biomedical announced positive safety data from Cohort 1 of OASIS, its ongoing Phase I/IIa trial of CLS-AX for neovascular age-related macular degeneration (wet AMD). CLS-AX, or axitinib injectable suspension, is administered by suprachoroidal injection via Clearside’s SCS Microinjector. The drug is a tyrosine kinase inhibitor (TKI) currently approved to treat renal cell cancer that achieves pan-VEGF blockade, directly inhibiting VEGF receptors-1, -2, and -3.
SpringWorks Therapeutics reported interim data from the Phase IIb ReNeu trial of mirdametinib for NF1-PN. Mirdametinib is a MEK inhibitor. NF1-PN is NF1-associated plexiform neurofibromas. The company also initiated a Phase I/II trial of mirdametinib for children, adolescents, and young adults with low-grade glioma. It is sponsored by St. Jude Children’s Research Hospital.
Spero Therapeutics initiated two Phase I trials of SPR206. One included a bronchoalveolar lavage trial assessing the penetration of SPR206 into the pulmonary compartment. The other is a renal impairment clinical trial. SPR206 is an IV-administered next-generation polymyxin product candidate.
Satsuma Pharmaceuticals announced positive pharmacokinetic, tolerability and safety data from its Phase I trial of STS101 for acute migraine. The data showed that all three dose strengths were well-tolerated and hit the target PK profile.
Motus GI Holdings enrolled the first patients in the EU study of the Pure-Vu System, studying the clinical outcomes in patients with a history of poor bowel preparation using a low volume preparation with limited diet restrictions and the Pure-Vu System. The Pure-Vu System integrated with standard and slim colonoscopes, as well as gastroscopes, to improve visualization during colonoscopy and upper GI procedures.
Freeline Therapeutics Holdings announced dosing of the second patient in the ongoing Phase I/II MARVEL-1 trial of FLT190, its liver-directed AAV gene therapy candidate for Fabry disease. FLT190 is an AAV gene therapy made up of their next generation potent AAVS3 engineered capsid, containing an expression cassette with a codon-optimized human GLA cDNA under the
Altimmune announced results from a prespecified six-week interim analysis of its ongoing 12-week Phase I trial of ALT-801 in healthy overweight and obese volunteers. ALT-8801 is an GLP-1/glucagon dual receptor agonist.
Apexigen initiated a collaborative Phase II trial of sotigalimab, its monoclonal antibody targeting CD40, in combination therapy for patients with recurrent BRCA wildtype ovarian cancer. They are collaborating with the Nordic Society of Gynaecological Oncology’s clinical trial unit. Sotigalimab is a monoclonal antibody targeting CD40.
Innovent Biologics dosed the first patient in a Phase II basket trial of taletrectinib for solid tumors containing NTRK fusion. Taletrectinib is a next-generation TKI designed to target ROS1 and NTRK fusion mutations.
Biogen dosed the first patient in its TOPAZ-1 Phase III trial of BIIB059 for active systemic lupus erythematosus (SLE). BIIB059 is a first-in-class, humanized IgG1 monoclonal antibodoy targeting blood dendritic cell antigen 2 (BDCA2).