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
Gilead Sciences announced it planned to soon start enrolling an open-label, single-arm Phase II/III clinical trial of remdesivir in about 50 pediatric patients with moderate-to-severe COVID-19, including newborns through adolescents. The trial will be performed at more than 30 locations in the U.S. and Europe.
“From the onset of the pandemic, Gilead has advanced the development of our investigational antiviral remdesivir for the treatment of COVID-19, in parallel with emerging knowledge about the disease,” said Merdad Parsey, Gilead’s chief medical officer. “While the novel coronavirus appears to disproportionally affect adults—especially the elderly and those with underlying health conditions—concerning reports have been documented of children and young adults being hospitalized with COVID-19 and related autoimmune symptoms.”
aTyr Pharma dosed the first patient in its Phase II trial of ATYR1923 in COVID-19 patients with severe respiratory complications. TYR1923 is a potential first-in-class immunomodulator.
Sinovac Biotech announced positive early data from a Phase I/II clinical trial of its COVID-19 vaccine, CoronaVac. The vaccine showed both favorable immunogenicity and safety. Sinovac’s Phase I/II trials looked at a total of 743 healthy volunteers ranging in age from 18 to 59 years. Of those patients, 143 are in Phase I and 600 are in Phase II. No severe adverse events have been reported in either phase. The Phase II trial demonstrated that the vaccine creates neutralizing antibodies 14 days after dosing. The antibody seroconversion rate is greater than 90%, which suggests the vaccine can induce a positive immune response.
Eli Lilly and Company reported it had enrolled the first patient in its Phase III trial of baricitinib in hospitalized adults with COVID-19. The drug is marketed as Olumiant for adults with moderately to severely active rheumatoid arthritis (RA). The company plans to enroll 400 patients and will be run in the U.S., Europe and Latin America. It will include hospitalized patients who have at least one elevated marker of inflammation but who are not in mechanical ventilation.
The Co-Immunity Project announced results from its Phase I of its testing to track and curb the spread of SARS-CoV-2. The project is a collaboration between the University of Louisville (Kentucky) and Louisville Healthcare CEO Council. The Phase I tested health care workers in Louisville. The bottom line was that precautionary measures worked in preventing the disease’s transmission among health care workers.
Veru enrolled the first patient in its Phase II trial of VERU-111, a novel microtubule depolymerization drug to fight COVID-19. The trial will study 40 hospitalized patients, 20 with VERU-111 and 20 with placebo who tested positive for SARS-CoV-2 and who are at high risk for ARDS.
Non-COVID-19-Related
BeyondSpring announced positive topline results at the pre-specified interim analysis of its PROTECTIVE-2 Phase III trial of plinabulin in combination with Amgen’s Neulasta compared to Neulasta alone in chemotherapy-induced neutropenia. The interim results demonstrated significant enhanced of the two-drug combination in the rate of Grade 4 neutropenia prevention. Plinabulin is a differentiated immune and stem cell modulator.
Catalyst Biosciences presented final efficacy and safety data from its Phase IIb trial of DalcA in hemophilia B. The drug is a next-generation subcutaneously administered Factor IX (FIX) therapy. The data showed that 28 days of daily DalcA achieved protective target FIX levels of more than 12% in all patients, with FIX levels of up to 27% and a half-life of 2.5 to 5.1 days with no bleeds.
Applied Therapeutics provided additional supportive biomarker efficacy and safety data for AT-007 in Galactosemia at 40 mg/kg. It also initiated the AT-007 pediatric trial, ACTION-Galactosemia Kids, in children age 2 to 17. The drug is a CNS penetrant Aldose Reductase inhibitor.
Brickell Biotech and Kaken Pharmaceuticals released positive Phase III results for its Phase III trial of sofprionium Bromide Gel, 5% in Japanese patients with primary axillary hyperhidrosis. The trial studied 281 Japanese patients randomized 1:1 to apply the drug or a placebo vehicle gel to the axillae for 42 days. All patients had Hyperhidrosis Disease Severity Scale (HDSS) scores of equal to or greater than 3 and greater than or equal to 50 mg/5 min gravimetric sweat production (GSP) in each axilla at baseline. All primary and secondary efficacy endpoints showed statistically significant differences.
vTv Therapeutics presented two poster sessions at the American Diabetes Association’s 80th Scientific Sessions, presenting clinical data from the positive Phase II Simplicit-T1 Study confirming the potential for TTP399 to provide benefit beyond insulin alone in type 1 diabetes. TTP399 is a once-daily pill that reduces HbA1c and improves time in range without increasing hypoglycemia or any signal for adverse events including diabetic ketoacidosis.
AbbVie reported positive results from the VIALE-A clinical trial of Venclexta plus azacytidine in previously untreated acute myeloid leukemia (AML) patients who were ineligible for intensive chemotherapy. The trial showed a 34% decrease in the risk of death compared to azacytidine in combination with placebo alone. Patients receiving the venetoclax combination showed improved median overall survival of 14.7 months compared to 9.6 months in the placebo arm and 66.4% of patients receiving the combination had a composite complete remission compared to 28.3% with the azacytidine-placebo arm.
AbbVie’s Rinvoq (upadacitinib) monotherapy demonstrated improvement in skin clearance and itch in its Phase III trial in atopic dermatitis. Rinfoq is an oral, once-daily, selective and reversible JAK inhibitor being studied in several immune-mediated inflammatory diseases. It is approved for moderately to severely active rheumatoid arthritis.
Momenta Pharmaceuticals announced positive topline data from an interim analysis of its Phase II Vivacity-MG study of nipocalimab (M281) in generalized myasthenia gravis (gMG). All four treatment arms showed efficacy in MG activities of daily living (MG-ADL) score, the primary endpoint. Nipocalimab is a high affinity, fully human, aglycosylated, effectorless IgG1 anti-FcRn monoclonal antibody.
Genentech presented two-year data from Part 1 of its pivotal SUNFISH trial in patients aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The drug showed significantly improved motor function after 24 months compared to natural history data.
Sarepta Therapeutics announced safety and tolerability data at one year from four Duchenne muscular dystrophy (DMD) patients who received SRP-9001 micro-dystrophin. SRP-9001 is a gene transfer therapy designed to delivery its micro-dystrophin-encoding gene to muscle tissue for the targeted production of micro-dystrophin protein.
Janssen Pharmaceutical, a Johnson & Johnson company, announced results from its Phase III trial of subcutaneous daratumumab (Darzalex) in newly diagnosed light chain amyloidosis. The data showed the drug in combination with cyclophosphamide, bortezomib, and dexamethasone (CyBorD) had a higher hematologic complete response rate (CR) compared to CyBorD alone.
Phathom Pharmaceuticals announced it had randomized the first new patients in each of its two Phase III clinical trials since pausing them during the pandemic. They are the PHALCON-EE and PHALCON-HP Phase III trials of vonoprazan, an oral small molecule potassium-competitive acid blocker (P-CAB). PHALCON-EE is in erosive esophagitis; PHALCON-HP is in patients with H. pylori infection.
Ardelyx announced positive data from its ongoing NORMALIZE trial of tenapanor as monotherapy or in combination with sevelamer in chronic kidney disease patients on dialysis. Tenapanor is a first-in-class, phosphate absorption inhibitor. The data is described as “unprecedented in terms of the proportion of patients able to achieve serum phosphorus levels less than 4.6mg/dl.”
PTC Therapeutics announced two-year data from Part 1 of the SUNFISH trial in type 2 or 3 SMA and new preliminary 12-month data from JEWELFISH. The data showed the drug improved motor function compared to natural history data.
Navidea Biopharmaceuticals announced full enrollment in its ongoing NAV3-31 Phase IIb trial of tilmanocept in rheumatoid arthritis. The company expects the last patient to be screened and evaluated by the end of 2020.
Amarin Corporation presented data from the REDUCE-IT trial showing that 4 g/day of Vascepa (icosapent ethyl) had a significant 23% reductions in both first and total primary composite major adverse cardiovascular events in diabetes patients. Decreases of 30% and 29% were seen in both first and total hard (3-point) MACE, the key secondary composite endpoint, respectively.
Biohaven Pharmaceuticals presented data from 25 abstracts at the 2020 American Headache Society (AHS) Virtual Annual Scientific Meeting. The materials demonstrated the efficacy, safety, tolerability and pharmacoeconomic value of Nurtec (rimegepant) for the prevention of migraine. The drug is the first and only calcitonin gene-related peptide (CGRP) receptor antagonist in an orally disintegrating tablet approved by the FDA for the acute treatment of migraine in adults.
Boehringer Ingelheim and Eli Lilly presented new interim analyses from EMPRISE, a real-world study of Jardiance (empagliflozin) in more than 200,000 adults with type 2 diabetes in routine clinical care compared with DPP-4 inhibitors or GLP-1 receptor agonists. The new data from the two analyses evaluated the risks for heart failure hospitalizations, all-cause mortality and MACE, while the second compared risk for cardiovascular outcomes in 23,000 adults aged 66 and older who were treated with Jardiance versus DPP-4 inhibitors and GLP-1 receptor agonists.
Eli Lilly announced Verzenio (abemaciclib) in combination with standard adjuvant endocrine therapy (ET) met the primary endpoint of invasive disease-free survival (IDFS), significantly reducing the risk of breast cancer recurrence or death compared to standard adjuvant ET alone. These were from a pre-planned interim analysis of the Phase III monarchE trial. Verzenio is the only CDK4 and 6 inhibitor to demonstrate a statistically significant reduction in the risk of cancer for people with high risk HR+, HER2- early breast cancer.
Dermavant Sciences published outcomes from its Phase IIb trial of tapinarof cream for atopic dermatitis. The most recent analyses of the trial showed a greater than or equal to 75% and greater than or equal to 90% improvement in the Eczema Area and Severity Index from baseline (EASI75, EASI90) in addition to mean percentage change in EASI scores, the mean change in percentage of body surface area affected, and mean change in total severity score.
MEDIAN Technologies announced the result of a preliminary retrospective study of the risk of recurrence in primary liver cancer based on a non-invasive biomarker. The iBiopsy imaging biomarker discovery platform produced the data on one of the three indications.
BGG World completed and published the data from a new clinical trial on its ApplePhenon on skin health in 59 healthy women over 12 weeks. The ApplePhenon reduced sunburn after UV irradiation and significantly lowered delta increase in melanin formulation after UV irradiation. ApplePhenon is produced from apples wide-harvested in central Asia and has a phytochemical profile marked by an oligomeric and a polymeric procyanidin fraction.
ZetrOZ Systems published data from a double-blind RCT study showing that low-intensity continuous ultrasound treatment significantly reduced upper trapezius myofascial pain of the neck and shoulder. The results indicated that applying daily continuous ultrasound treatment of 18,720 joules using a wearable sustained medicine (sam) devices resulted in a clinically meaningful improvement in the global rate of change (GROC) score for patients.
Pfizer and Sangamo Therapeutics provided follow-up data from the Phase I/II Alta study of giroctocogene fitelparvovec in hemophilia A. All five patients with severe hemophilia A dosed with the therapy sustained factor VIII activity levels, with a median of 64.2% by way of chromogenic assay. No patients demonstrated bleeding events or required FVIII infusions.
Genentech’s Tecentriq and chemotherapy met the primary endpoint of improved, pathological complete response regardless of PD-L1 status in early triple-negative breast cancer. Tecentriq (atezolizumab) is Genentech’s PD-L1 checkpoint inhibitor.
Natera began enrollments in the CIRCULATE-Japan and BESPOKE CRC clinical trials measuring outcomes of Signatera molecular residual disease (MRD) testing in resectable Stages II-IV colorectal cancer (CRC). Signatera is a custom-built circulating tumor DNA (ctDNA) test for treatment monitoring and molecular residual disease assessment in patients who were previously diagnosed with cancer.
Eloxx Pharmaceuticals resumed enrollment in Israel and Europe after pausing during the pandemic for its Phase II trial of ELX-02 in cystic fibrosis (CF). ELX-02 is a small molecule drug candidate designed to restore production of full-length functional proteins.
Genentech, a Roche company, reported that the Phase III IPATential150 trial of ipatasertib in combination with abiraterone and prednisone/prednisolone hit one of the co-primary endpoints and missed the other in metastatic castration-resistant prostate cancer (mCRPC) and whose tumors had PTEN loss.
The trial met radiographic progression-free survival (rPFS). The drug in combination with abiraterone and prednisone/prednisolone showed a statistically significant decrease in the risk of the disease getting worse or death compared to standard of care plus placebo. However, the second co-primary endpoint of rPFS in the overall study population was not met.
Finch Therapeutics reported positive topline results from PRISM3, its Phase II trial of CP101, an oral microbiome drug for the prevention of recurrent C. difficile infection (CDI). Clostridioides difficile infection is a dangerous antibiotic-resistant bacterial infection. CP101 is an investigational, oral microbiome drug designed to deliver complete microbiome communities in an oral, enteric release capsule. In addition to being tested for recurrent C. diff infection, it is being studied for the treatment of chronic hepatitis B.
The PRISM3 trial met the primary efficacy endpoint, with 74.5% of recurrent CDI patients who received it achieving a sustained clinical cure through week eight. This was compared to 61.5% of patients in the control group who received standard-of-care antibiotic therapy alone, which is statistically significant. The therapy was well-tolerated at eight weeks after treatment, with no treatment-related serious adverse events.
CymaBay Therapeutics presented new data from a previously completed open-label Phase II trial of seladelpar in primary biliary cholangitis. Seladelpar is a potent, selective, orally active PPARδ agonist.
