Clinical Catch-Up: October 18-22
25 Oct 2021
VaccineGene TherapyAntibodyFinancial StatementFirst in Class
It was yet another busy week for clinical trial news. Here’s a look.
COVID-19-Related
Valneva announced positive topline data from the Phase III trial Cov-Compare of its COVID-19 vaccine, VLA2001. The Cov-Compare trial recruited a total of 4,012 people 18 years and older across 26 sites in the UK. It hit the co-primary endpoints, demonstrating superiority against the AstraZeneca-Oxford vaccine (ChAdOx1-S) in geometric mean titer for neutralization antibodies and non-inferiority in terms of seroconversion rates two weeks after the second vaccination in adults 30 years and older.
A National Institute of Allergy and Infectious Diseases (NIAID) trial reported no faster recovery in hospitalized patients with COVID-19 pneumonia when treated with interferon beta-1a and Gilead Sciences’ Veklury (remdesivir) compared to Veklury alone. The ACTT-3 trial found no significant difference in mortality rates in either group, with a 5% mortality rate in the interferon group compared to 3% with Veklury alone.
Statera Biopharma announced the Loma Linda University Institutional Review Board had given approval to run a Phase I pilot study of STAT-205 to mitigate COVID-19 progression. STAT-205 is an immune-modulator that decreases elevated inflammatory responses and inhibitors viral replication in human lung cells.
Atea Pharmaceuticals’ Phase II MOONSONG trial did not hit the primary endpoint of reducing clinical symptoms of mild or moderate COVID-19. AT-527 is an oral direct-acting antiviral.
Pfizer and BioNTech announced topline results from a Phase III trial of a 30-microgram booster shot of their vaccine in more than 10,000 people 16 years of age and older. The booster dose, which was given to patients who had already received the primary two-dose regimen, restored vaccine protection against COVID-19 to the levels very similar to what was seen after the second dose.
A study by researchers from Israel’s Clalit health maintenance organization, Harvard University and Boston Children's Hospital found that the Pfizer-BioNTech COVID-19 vaccineCOVID-19 vaccine was 93% effective at halting symptomatic COVID-19 infections. And it was almost as good at stopping the Delta variant entirely.
The U.S. Centers for Disease Control and Prevention (CDC) released research from 19 U.S. pediatric hospitals from June through September, and found the Pfizer-BioNTech vaccine was 93% effective in preventing hospitalization from COVID-19 in 12- to 18-year-olds. This study followed 464 COVID-19 patients. About 72% of the kids had at least one underlying condition that made them susceptible to severe COVID-19, the study also found that 97% of the kids who ended up in the hospital weren’t vaccinated.
Non-COVID-19-Related
Biogen announced topline results from the Phase III VALOR trial of tofersen for amyotrophic lateral sclerosis (ALS). Specifically, the drug is being evaluated for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis. Toferson is an antisense drug. It binds to SOD1 messenger RNA (mRNA), which helps it degrade by RNase-H, with the goal of decreasing synthesis of SOD1 protein production. The study for ALS failed to hit the primary endpoint of change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R).
Vertex Pharmaceuticals announced positive early data from the first patient in its Phase I/II study of VX-880 in type 1 diabetes (T1D). The data was from Day 90. The therapy is a stem cell-derived, fully differentiated pancreatic islet cell replacement therapy. In the study, the patient received a single infusion of VX-880 at half the target dose along with immunosuppressive therapy. The patient showed successful engraftment and demonstrated fast and robust improvements in several measurements, including increases in fasting and stimulated C-peptide, improvements in glycemic control, including HbA1c. It also resulted in less need for medical insulin. The therapy appeared well tolerated.
Radius Health announced positive topline results from the ATOM Phase III trial of Tymlos (abaloparatide) subcutaneous injection in males with osteoporosis. It hit the primary endpoint, percentage change in lumbar spine bone mineral density compared to placebo.
Abivax reported new results from an extended set of patients in its ongoing open-label maintenance study, following the Phase IIb induction trial of once-daily oral 50mg ABX464 for ulcerative colitis. There was clinical remission in 58.4% of 101 patients after 48 weeks of receiving the drug with a favorable safety and tolerability profile.
Homology Medicines initiated a Phase I study of HMI-203 for adults with mucopolysaccharidosis type II (MPS II), or Hunter syndrome. HMI-203 is a one-time in vivo gene therapy candidate using a human hematopoietic stem cell-derived AAV vector to deliver functional copies of the IDS gene to multiple organs.
NervGen presented interim Phase I trial data from its Phase I program of NVG-291. The drug is a protein tyrosine phosphatase modulator being developed for treatment of spinal cord injury and multiple sclerosis.
Ultragenyx Pharmaceutical screened and enrolled multiple patients in its pivotal, seamless Phase I/II/III Cyprus2+ study of UX701 for Wilson disease. The therapy is an AAV9 gene therapy engineered to deliver stable expression of the ATP7B copper transporter after a single IV infusion.
Inari Medical announced planned enrollment of the PEERLESS trial to compare the clinical outcomes of patients with intermediate-high risk pulmonary embolism treated with the FlowTriever System compared to catheter-directed thrombolysis. It will include up to 700 patients and 60 sites in the U.S. and Europe.
ORYZON Genomics enrolled the first patient in the U.S. in its Phase IIb study of vafidemstat in Borderline Personality Disorder (BPD). The drug is a selective inhibitor of the epigenetic target LSD1.
BioXcel Therapeutics expanded its ongoing Phase Ib/II trial of BXCL701 for pre-treated metastatic castration-resistant prostate cancer patients with either de novo or treatment-emergent small-cell neuroendocrine carcinoma. The drug is an oral innate immune activator.
BiomX announced results from its Phase II cosmetic trial of BX001 to improve the appearance of skin in acne prone subjects. BX001 was shown to be safe and well-tolerated. It is a topical gel formulation of a cocktail of naturally occurring phage that target Cutibacterium acnes.
First Wave BioPharma received the go-ahead from the FDA for its Phase IIa PASSPORT trial of FW-ICI-AC for Grade 1 and 2 colitis and diarrhea in oncology patients receiving immune checkpoint inhibitors. FW-ICI-AC is a proprietary oral immediate-release tablet formulation of niclosamide, which has anti-inflammatory and anti-viral properties. The company also announced the initial members of the steering committee for its Phase IIa PASSPORT trial.
Entasis Therapeutics announced topline results from its Phase III ATTACK trial of SUL-DUR versus colistin in Acenetobacterbaumannii infections. The drug hit the primary endpoint of 28-day all-cause mortality in patients with carbapenem-resistant Acinetobacter infections.
VYNE Therapeutics enrolled the first patient in its Phase Ib/IIa trial of FMX114 for mild-to-moderate atopic dermatitis (AD). FMX114 is the company’s investigational combination gel formulation of tofacitinib and fingolimod.
Galera Therapeutics announced results from the Phase III ROMAN trial of avasopasem manganese for severe oral mucositis (SOM) in patients with locally advanced head and neck cancer undergoing standard-of-care radiotherapy. The trial failed to hit the primary endpoint of reduction in the incidence of SOM.
Opthea opened patient enrollment in the OPT-302 Phase III trial for wet age-related macular degeneration. OPT-302 is being developed for use in combination with anti-VEGF-A monotherapies to offer broader VEGF family inhibition.
Paratek Pharmaceuticals dosed the first patient in the Phase IIb trial of Nuzyra (omadacycline) for nontuberculous mycobacterial pulmonary disease caused by Mycobacterium abscessus complex. Nuzyra is a novel antibiotic, a modernized tetracycline.
Blade Therapeutics initiated an additional Phase I study of cudetaxestat for idiopathic pulmonary fibrosis. The drug is a non-competitive autotaxin inhibitor.
Enzychem Lifesciences announced positive Phase II trial data of EC-18 in chemoradiation-induced mucositis (CRIOM). EC-18 is a novel, first-in-class, small molecule oral immunomodulator. It decreased the duration and incidence of severe oral mucositis in patients with head and neck cancer receiving concurrent chemoradiation therapy.
Editas Medicine presented initial clinical data from the Phase I/II BRILLIANCE trial of EDIT-101 for Leber congenital amaurosis 10 (LCA10). There were encouraging efficacy signals suggestive of positive biological activity and potential early clinical benefits. EDIT-101 is administered via a subretinal injection to deliver its gene editing machinery directly to photoreceptor cells.
Menarini Group and Radius Health announced positive topline results from the Phase III EMERALD trial of elacestrant as a monotherapy for ER+/HER2- advanced or metastatic breast cancer (mBC). It hit both primary endpoints, progression-free survival (PFS) in the overall population and PFS in patients with tumors harboring the Estrogen Receptors 1 (ESR1) mutations.
Longeveron activated two new clinical sites for the currently enrolling ELPIS II Trial of Lomecel-B injection into the myocardium of infants with Hypoplastic Left Heart Syndrome (HLHS). Lomecel-B is an allogeneic, bone-marrow-derived medicinal signaling cell (MSC) product.
AmMax Bio announced positive interim data from its Phase II trial of AMB-05X for tenosynovial giant cell tumor (TGCT). The drug is a potent monoclonal antibody against CSF1R.
Beyond Air reported positive interim data from the ongoing LungFit GO pilot study in Australia. Patients self-administered high concentration inhaled NO at home to treat severe nontuberculous mycobacterial (NTM) lung disease. At the data cutoff, the data showed high concentration inhaled NO was well tolerated with no study discontinuations and no treatment-related serious adverse events.
VectiveBio received the greenlight from the FDA for its Phase II STARGAZE trial of apraglutide in steroid-refractory acute graft-versus-host disease (aGVHD). The drug is a next-generation, long-acting GLP-2 analog.
Telix Pharmaceuticals presented the first set of peer-reviewed data from the IPAX-1 Phase I/II trial of TLX101 in combination with external beam radiation therapy (XRT) in recurrent glioblastoma multiforme (GBM). The data confirmed the study hit its primary objective, demonstrating safety and tolerability, as well as overall survival of 15.97 months, to date, in second line GBM setting.
AstraZeneca announced a clinical hold on its trial of AZD5991 in patients with relapsed or refractory hematologic malignancies due to safety concerns. AZD5991 is a direct inhibitor of Mcl-1. There were reportedly indications of heart issues in one patient who received the combination treatment of AZD5991 with Roche’s venetoclax.
Frequency Therapeutics dosed the first patient in a new FX-322 Phase IIb study in a refined population with acquired sensorineural hearing loss (SNHL). FX-322 is a small molecule compound that has shown statistically significant and clinically meaningful hearing improvements in clinical trials.
BioCardia treated the first patient in its Phase III CardiAMP Cell Therapy Chronic Myocardial Ischemia Trial for patients with no option chronic myocardial ischemia with refractory angina. They are evaluating the company’s CardiAMP autologous cell therapy.
SCYNEXIS published results from its Phase III VANISH-303 and Phase II DOVE trials of Brexafemme (ibrexafungerp tablets) for treatment of vulvovaginal candidiasis (VVC). Brexafemme is an oral glucan synthase inhibitor.
Revolo Biotherapeutics activated its Phase II allergen sensitivity study in the UK of its first-in-class peptide, ‘1104, for the treatment of allergic disease. The drug is derived from mTB Chaperonin 60.1, a natural immune-regulatory protein.
PDS Biotechnology temporarily suspended recruitment of the NCI-led Phase II trial of PDS0101 (Versamune-HPV16) in combination with two investigational immune-modulating agents in advanced HPV cancers. There were no safety or efficacy issues. Rather, it is being called “a slight administrative delay.”
BeyondSpring treated the first patient in a Phase II study of plinabulin in combination with nivolumab and ipilimumab in patients with third-line recurrent small-cell lung cancer (SCLC) who failed checkpoint inhibitors and platinum-based chemotherapy. Plinabulin is a selective immunomodulating microtubule-binding agent (SIMBA), which is a potent antigen presenting cell inducer.
Sio Gene Therapies presented positive interim data from its ongoing Phase I/II trial of AXO-AAV-GM1 for GM1 gangliosidosis. The therapy is an AAV9-based gene therapy.
Metacrine reported interim results from a Phase IIa trial of MET642 for non-alcoholic steatohepatitis (NASH). MET642 is a farnesoid X receptor (FXR) agonist.
Tryp Therapeutic reported the FDA placed a clinical hold on its Phase IIa trial of TRP-8802 for eating disorders, including binge eating disorder, hypothalamic obesity, and Prader-Willi syndrome. TRP-8802 is an oral formulation of synthetic psilocybin.
Athira Pharma completed enrollment in the ACT-AD Phase II trial of ATH-1017 in mild-to-moderate Alzheimer’s disease. ATH-1017 is a small molecule designed to enhance the activity of Hepatocyte Growth Factor (HGF) at its receptor, MET.
Aadi Bioscience published primary analysis from six months the AMPECT trial as well as 1.5-year follow-up of ABI-009 in malignant perivascular epithelioid cell tumors. Nab-sirolimus in 31 patients had an assessed response rate of 39% with one complete and 11 partial responses; 52% had stable disease.
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