It was a busy week for clinical trial announcements. Here’s a look.
It was a busy week for clinical trial announcements. Here’s a look.
COVID-19-Related
All India Institute of Medical Sciences (AIIMS) indicated that Bharat Biotech’s COVID-19 vaccine, Covaxin, may offer immunity for nine to 12 months, based on mathematical models. The vaccine was already approved before the release of Phase III data and was used to inoculate healthcare workers during the Phase I trial.
Clene Nanomedicine initiated a Phase II trial of CNM-ZnAg liquid solution in acutely symptomatic, non-hospitalized COVID-19 patients. The primary endpoint will evaluate the rate of decreased hospitalizations at day 28. CNM-ZnAg is a proprietary zinc-silver ionic solution that has both antiviral and antibacterial properties as well as immune-boosting properties.
Oxford University announced it began testing its COVID-19 vaccine in children as young as six years of age. The study will include 300 children ages 6 to 17, with 240 receiving the vaccine co-developed with AstraZeneca. The remaining volunteers will receive a control meningitis vaccine that has been proven safe in children but is believed to mimic side effects of the COVID-19 vaccine.
Mesoblast Limited published results on the first two children treated with its mesenchymal stromal cell (MSC) product candidate, remestemcel-L for multisystem inflammatory syndrome (MIS-C) associated with COVID-19. MIS-C is a potentially life-threatening inflammatory conditions involving multiple critical organs and their vasculature. It is linked to previous, instead of active COVID-19 infection.
PTC Therapeutics initiated the second stage of the Phase II/III FITE19 clinical trial of PTC299 in COVID-19 patients. PTC299 is an oral drug with dual mechanisms of action that potentially inhibits viral replication and attenuates the uncontrolled inflammatory response post-infection. The drug targets dihydroorotate dehydrogenase (DHODH).
Altimmune received the go-ahead from the FDA for its Phase I trial of AdCOVID, its single-dose, intranasal COVID-19 vaccine. It will test the safety and immunogenicity of the vaccine in up to 180 healthy adult volunteers ages 18 to 55.
Pharma Mar SA received approval in the UK to initiate its Phase III NEPTUNO trial of Aplidin (plitidepsin) for hospitalized patients with moderate COVID-19. The trial is expected to run in 12 countries but was first authorized in the UK. It hopes to enroll more than 600 patients. Aplidin blocks the eEF1A protein, which SARS-CoV-2 uses to replicate and infect other cells.
Biophytis SA began patient recruitment in France and Belgium for Part 2 of its COVA Study assessing Sarconeos for acute respiratory failure associated with COVID-19. Sarconeos is an oral, small molecule being developed for sarcopenia and severe respiratory aspects of COVID-19, as well as in a pediatric formulation for Duchenne muscular dystrophy (DMD).
EmitBio announced statistically significant efficacy results from its Phase I/II trial of its medical device that directs precise wavelengths of safe, visible light targeted to the back of the throat and surrounding tissues. The device was designed for self-treatment of COVID-19 in an at-home setting.
Diffusion Pharmaceuticals completed and announced topline data from its Phase Ib trial of its diffusion-enhancing therapeutic, trans sodium crocetinate, in hospitalized COVID-19 patients with confirmed hypoxemia. The primary endpoint was safety and tolerability, with secondary endpoints being pharmacokinetic measurement of trans sodium crocetinate levels after dosing, relative improvements in blood oxygen levels, and other clinical measures associated with COVID-19.
Pfizer and BioNTech dosed the first patients in a clinical trial of 4,000 women in the late stages of pregnancy yesterday. The two companies will run the Phase II trial for 350 volunteers between 27 and 34 weeks’ gestation to confirm safety before moving into the Phase III portion for women between 24 and 34 weeks of pregnancy. AstraZeneca and Johnson & Johnson also plan to run trials in the coming months on pregnant women.
CMTx Biotech received a $315,000 Clinical Trial Planning Grant from the NIH’s National Center for Advancing Translational Sciences to finalize a Phase II trial of incyclinide. The drug is its candidate for treatment of hospitalized COVID-19 patients. The drug is a non-antimicrobial tetracycline formulation, which is the specialty of CMTx.
“Incyclinide has already been shown to be highly effective in a number of animal models of acute respiratory distress syndrome across several species,” noted Lorne Golub, co-founder of CMTx and SUNY Distinguished Professor of Oral Biology and Pathology.
Non-COVID-19-Related
Merck presented new data from the Phase III CLEAR trial (KEYNOTE-581/Study 307) of its checkpoint inhibitor Keytruda (pembrolizumab) and Eisai’s tyrosine kinase inhibitor Lenvima (lenvatinib) in renal cell carcinoma. The combination of Keytruda and Lenvima was compared to Lenvima plus everolimus versus Pfizer’s Sutent (sunitinib) for first-line treatment of patients with advanced renal cell carcinoma (RCC). The combination of Keytruda and Lenvima demonstrated statistically significant and clinically meaningful improvements in progression-free survival (PFS), overall survival (OS) and objective response rate (ORR) compared to sunitinib. The Lenvima-everolimus combination also demonstrated statistically significant improvements in PFS and ORR compared to sunitinib. In exploratory analysis, the data for PFS and OS were consistent across prespecified Memorial Sloan Kettering Cancer Center (MSKCC) risk groups, in other words, favorable, intermediate, and poor.
bluebird bio announced that it has placed its Phase I/II and Phase III trial of LentiGlobin gene therapy for sickle cell disease (SCD) on temporary suspension. The cause is a Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML). HGB-206 is the company’s ongoing Phase I/II trial of LentiGlobin for SCD. It includes three cohorts, A, B and C. In Group C, a refined manufacturing process designed to increase vector copy number was used. Group C also received LentiGlobin for SCD manufactured from hematopoietic (blood) stem cells (HSCs) collected from peripheral blood after mobilization with plerixafor, instead of by way of bone marrow harvest, which was the method used in Groups A and B. HGB-210 is their ongoing Phase III single-arm open-label trial. It is evaluating efficacy and safety of LentiGlobin for SCD in patients between two years and 50 years of age with sickle cell disease.
Histogen announced week 26 results from its Phase Ib/IIa trial of HST-001 in male patients with androgenic alopecia—hair loss. At the timepoint, patients receiving the drug demonstrated statistically significant change from baseline in total hairs in the target area. HST-001, or Hair Stimulating Complex (HSC), is designed to be a physician-administered treatment for hair loss.
Recce Pharmaceuticals formalized a deal with Fiona Stanley Hospital in Australia for a Phase I/II trial of RECCE 327, its new spray-on, broad-spectrum antibiotic for topical burn wound infections. It will enroll up to 30 patients.
Eli Lilly announced results from its SURPASS-3 and SURPASS-5 Phase III trials after 52 weeks and 40 weeks, respectively. SURPASS-3 compared tirzepatide to titrated insulin degludec and SURPASS-5 compared tirzepatide to placebo, both as an add-on to titrated insulin glargine. All primary and all key secondary endpoints were met. Tirzepatide is a once-weekly dual glucose-dependent insulinotropic polypeptide (GIP) and GLP-1 receptor agonist being evaluated for type 2 diabetes. The drug significantly reduced A1C and body weight in type 2 diabetes.
Selecta Biosciences and AskBio initiated first-in-human dose escalation Phase I trial of SEL-399. SEL-399 is an AAV serotype 8 empty vector capsid containing no DNA combined with ImmTOR. The trial is to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.
Salarius Pharmaceuticals completed the dose-escalation stage and established the recommended Phase II dose for its ongoing Phase I/II trial for seclidemstat in r/r Ewing sarcoma. Seclidemstat is a novel, oral reversible inhibitor of the lysine-specific histone demethylase 1 enzyme (LSD1), which plays a key role in the development and progression of certain cancers.
AstraZeneca and Merck announced the OlympiA Phase III trial for Lynparza (Olaparib) will move to early primary analysis and reporting after a recommendation from the Independent Data Monitoring Committee (IDMC). The IDMC decided the trial crossed the superiority boundary for its primary endpoint of invasive disease-free survival (iDFS) and demonstrated a sustainable, clinically relevant treatment effect for Lynparza compared to placebo for patients with germline BRCA-mutated high-risk HER2-negative early breast cancer.
Ocuphire Pharma initiated enrollment in its Phase II VEGA-1 trial of a combination kit of Nyxol and low-dose pilocarpine in presbyopia. Nyxol is believed to improve near vision based on its ability to reduce pupil diameter, which results in increased depth of field.
Dermavant Sciences announced positive safety and efficacy data from a planned interim analysis of PSOARING 3 of tapinarof in adults with plaque psoriasis. Tapinarof is a novel, once-daily aryl hydrocarbon receptor modulating agent (TAMA).
Flexion Therapeutics announced plans to advance FX201 into the high dose cohort of the Phase I trial. FX201 is an intra-articular, IL-1Ra gene therapy product for osteoarthritis.
ChemoCentryx published data from its Phase III ADVOCATE trial of avacopan in patients with anti-neutrophil cytoplasmic autoantibody-associated vasculitis (ANCA-associated vasculitis or ANCA vasculitis). Avacopan is an oral selective complement 51 receptor inhibitor. Treatment with avacopan in the absence of daily oral prednisone led to remission at week 26 and superiority in sustaining remission at 52 weeks compared to the prednisone group.
vTv Therapeutics initiated its Phase I trial of orally administered phosphodiesterase type 4 inhibitor, HPP737 in healthy adults. The drug is being developed for the treatment of psoriasis.
