Clinical Catch-Up: September 16-20

Clinical data

Last week was an unusually busy week for clinical trial news, with numerous companies presenting results at conferences. Here’s a look at the top stories.

Celgene announced topline data from its Phase III QUAZAR AML-001 trial. The study evaluated CC-486, the company’s oral azacytidine, as maintenance treatment in newly diagnosed acute myeloid leukemia (AML) patients who achieved first complete response (CR) or complete response with incomplete blood count recovery (CRi) with induction chemotherapy.

The primary endpoint of the trial was overall survival (OS). The trial demonstrated the drug resulted in a highly statistically significant and clinically meaningful improvement in overall survival (OS) compared to placebo. A key secondary endpoint, relapse-free survival (RFS), also hit statistically significant improvement.

Cellectar Biosciences presented data from Cohort 6 of its Phase I dose escalation trial of CLR 131 in relapsed or refractory multiple myeloma. The data was of four patients in the cohort who received a fractionated dose of the drug and achieved a 50% overall response rate. Two of the patients had a partial response and two achieved minimal responses. CLR 131 is a small molecule, radiotherapeutic Phospholipid Drug Conjugate (PDC) that delivers cytotoxic radiation directly and selectively to cancer cells.

Janssen of Johnson & Johnson announced data from the Phase II GRIFFIN trial of Darzalex (daratumumab) and bortezomib, lenalidomide and dexamethasone (VRd) in newly diagnosed patients with multiple myeloma eligible for high-dose therapy and autologous stem cell transplantation (ASCT) compared to VRd alone. The combination induced higher response rates in the patient population. A greater percentage hit the primary endpoint of stringent complete response (sCR) compared to VRd alone, 42% to 32%, respectively.

Allena Pharmaceuticals completed enrollment in its Phase III URIROX-1 clinical trial. The trial is evaluating reloxaliase in patients with enteric hyperoxaluria. Topline data is expected in the fourth quarter of this year. Reloxaliase is an oral, recombinant oxalate-degrading enzyme. Hyperoxaluria is characterized by elevated levels of oxalate in the urine either from a genetic defect or from excess absorption of oxalate from the diet.

Kura Oncology dosed the first patient in its Phase I clinical trial of KO-539 in relapsed or refractory acute myeloid leukemia (AML). KO-539 is a first-in-class inhibitor of the minim-mixed lineage leukemia (menin-MLL) interaction. The Phase I trial is focused on finding the maximum tolerated dose of the drug in this patient population.

Cytokinetics announced data from the Phase I trial of CK-3773274 in obstructive hypertrophic cardiomyopathy (HCM). CK-274 is a novel selective cardiac myosin inhibitor. They reported the trial met its primary and secondary objectives of safety and tolerability of single and multiple oral doses of the drug and to describe the pharmacokinetics and pharmacodynamics as measured by echocardiography. HCM is an inherited cardiovascular disease where the heart muscle becomes abnormally thick, which can result in chest pain, dizziness, shortness of breath, fainting and high risk of developing ventricular arrhythmias, atrial fibrillation, stroke, heart failure and sudden cardiac arrest.

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Akcea Therapeutics presented data from the Phase I trial of AKCEA-TTR-LRX in patients with transthyretin (TTR) amyloidosis or ATTR. Patients receiving the drug showed mean reduction in TTR levels at 13 weeks and at week 4. No adverse events led to interruption in dosing. The drug is an antisense compound developed using Ionis Pharmaceutical’s proprietary Ligand Conjugated Antisense (LICA) technology platform. The companies plan to initiate the Phase III program for the drug later this year.

Novartis announced positive new data from its PREVENT Phase III clinical trial. The trial is evaluating the efficacy and safety of Cosentyx (secukinumab) in non-radiographic axial spondyloarthritis (nr-axSpA). The trial is still ongoing but met its primary endpoint of ASAS40. ASAS40 is the ASAS Response Criteria of 40%, meaning there was an improvement of at least 40% and an absolute improvement of at least 10 units on a 0-100 scale in at least three of the following components: patient global assessment, pain assessment, function, and inflammation.

AveXis, a Novartis company, presented new interim data from the Phase III SPR1NT trial of Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1. The data is from the SPR1NT trial as of May 31. At that time, 10 children with two copies of SMN2, 12 patients with three copies of SMN2 and one with four copies were treated. The mean age in the two-copy group was 6.6 months at the last follow-up and in the three-copy cohort, 4.6 months. In those two groups, patients had completed their six-month swallow evaluation and had normal swallow function. Of the 22 patients in the study, all were alive and off permanent ventilation.

Acceleron Pharma announced that its Phase II trial of ACE-083 in facioscapulohumeral muscular dystrophy (FSHD) did not hit its functional secondary endpoints. As a result, the company is halting trials of the drug, despite showing significant increases in mean total muscle volume that was, however, not statistically significant in functional tests.

Biogen halted its Phase II trial of BG00011 in idiopathic pulmonary fibrosis (IPF) over safety concerns. The drug is designed to selectively disrupt the TGF-beta pathway, which has a central role in fibrotic diseases. The company is conducting end-of-study and safety follow-up visits.

Biogen also announced that it had initiated a new clinical trial, DEVOTE, to evaluate if a higher dose of Spinraza (nusinersen) can offer more efficacy for spinal muscular atrophy (SMA) in a broad patient population.

AC Immune initiated a Phase II trial of PI-2620, its Tau positron-emission tomography (PET) tracer. PI-2620 is a next-generation tracer developed using the company’s proprietary Morphomer discovery platform in collaboration with Life Molecular Imaging. It binds to Tau deposits, which with beta-amyloid plaques, are hallmarks of Alzheimer’s disease. The Phase II trial is being conducted in the UK and will run about three years.

Rafael Pharmaceuticals is expanding its Phase III trial for metastatic pancreatic cancer into France. The AVENGER 500 trial is a global, multicenter trial evaluating CPI-613 (devimistat) in combination with modified FOLFIRINOX as a first-line treatment in patients with metastatic adenocarcinoma of the pancreas. The company recently expanded the trial into Israel. It will now open in 13 sites throughout France.

Sanofi presented research showing that reductions in blood glucose levels with Soliqua/Suliqua in adults with type 2 diabetes were sustained to 52 weeks in a single-arm extension of its LixiLan-G Phase III clinical trial. The primary endpoint is showing a statistically superior decrease in average blood sugar level after 26 weeks compared to continuing treatment with a daily or weekly GLP-1 RA treatment. The data was presented at the European Association for the Study of Diabetes Annual Meeting.

Sanofi also presented data from a head-to-head controlled trial comparing Toujeo to insulin degludec in adults starting insulin for the first time to treat type 2 diabetes. Patients with moderate-severe renal impairment, Toujeo showed glycemic advantages compared to degludec. This was follow-up, secondary analysis from the BRIGHT study. The primary results were published in 2018.

Centrexion completed patient enrollment in its third Phase III trial, VICTORY-3. The trial is evaluating CNTX-4975 in chronic moderate-to-severe knee osteoarthritis (OA) pain in one or both knees. Topline data are expected in the first quarter of 2020. CNTX-4975 is a synthetic, ultra-pure intra-articular injection of trans-capsaicin designed to be injected directly into the site of the pain.

Otonomy is initiating a Phase I/II clinical trial of OTO-413 in patients with hearing loss. OTO-413 is a sustained-exposure formulation of brain-derived neurotrophic factor (BDNF). The trial will evaluate the safety and exploratory efficacy of the drug in patients with speech-in-noise hearing difficulty.

Pacira BioSciences completed enrollment in its Phase III trial of Exparel (bupivacaine liposome injectable suspension) in pediatric patients age six to less than 17 undergoing spinal or cardiac surgeries. The trial is dubbed PLAY and enrolled 98 patients to evaluate the pharmacokinetics and safety of Exparel for two patients group, those aged 12 to less than 17 and pages six to less than 12 years. It includes bupivacaine HCl as an active comparator arm for the older patient population.

Aclaris Therapeutics Phase III THWART-2 trial met its primary endpoints. The trial is of A-101 45% Topical Solution for the treatment of common warts. It met the primary and all secondary efficacy endpoints, hitting clinically and statistically significant clearance of common warts.

SCYNEXIS completed the last-patient/last-visit in its Phase III VANISH 303 trial of inbrexafungerp to treat serious fungal infections. As a result, the company expects topline data to be released earlier than expected. Ibrexafungerp is the first example out of a novel family of antifungal compounds called triterpenoids. The drug is being treated for both serious outpatient fungal infections and hospital-based, life-threatening fungal infections.

Apellis Pharmaceuticals released details about its Phase II FILLY trial of intravitreal APL2 (pegcetacoplan) for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The trial was a Phase II, multicenter, randomized, single-masked, sham-controlled trial in 246 patients with that indication in the U.S., Australia, and New Zealand. At the one-year mark, patients treated with APL-2 showed a 29% decrease in the growth of GA lesion area in the every-month group and a 20% reduction in the every-other-month (EOM) treatment group compared to the pooled sham group.

vTv Therapeutics presented additional positive data from its Phase II Simplici-T1 trial in patient with type 1 diabetes. The trial assessed TTP399 as an adjunct to insulin therapy in adults with T1D. In the first presentation, TTP399 decreased HbA1c levels by 0.7% relative to placebo. The drug increased Time in Range by 11% and reduced the total daily mealtime bolus insulin dose by 23%.

Zynerba presented positive topline data from its Phase II BELIEVE 1 trial of Zygel as a transdermal gel for pediatric epileptic encephalopathy. These are a group of rare epilepsy syndromes, including Dravet syndrome and Lennox-Gastaut syndrome. Patients receiving the drug had 44% and 58% monthly median reductions in seizures compared to baseline from month two to month six.

AlzProtect received approval from the French Agency for the Safety of Health Products to launch a Phase IIa trial of AZP2006 in Progressive Supranuclear Palsy (PSP). The trial will be conducted at the Pitie, Salpetriere University Hospital in Paris and at the University Hospital of Lille, France.

Oramed Pharmaceuticals treated the last patient in its primary cohort of the Phase IIb HbA2c trial of ORMD-0801 for type 2 diabetes. The trial is a dose-ranging trial to determine the appropriate dosage for the drug, with topline data expected in the fourth quarter of this year. The company focuses on creating oral formulations for drugs that were previously only injectable, in this case, insulin.

Rafael Pharmaceuticals has expanded its Phase III trial of CPI-613 (devimistat) in older patients with relapsed or refractory acute myeloid leukemia (AML) into South Korea. The drug is being evaluated in combination with high dose cytarabine and mitoxantrone (CHAM). It is currently in multiple U.S. and European sites.

Dance Biopharm presented data from its Phase II trial of Dance 501, a novel gentle mist formulation of human insulin in type 2 diabetes. The data showed clinically meaningful benefit over rapid-acting insulin injections. The trial enrolled 24 patients with type 2 diabetes on daily insulin therapy.

Foamix Pharmaceuticals enrolled the first patient in its Phase II trial of its topical combination foam, minocycline 3% and adapalene 0.3% (FCD105) for moderate-to-severe acne vulgaris. The trial will enroll about 400 patients aged 12 years and older and is being conducted at multiple locations throughout the U.S.

ASLAN Pharmaceuticals presented late-breaking topline data from a Phase II trial conducted in China of varlitinib and capecitabine in second-line biliary tract cancer (BTC) patients. They presented the data at the 2019 Chinese Society of Clinical Oncology (CSCO) meeting in Xiamen, China. The trial enrolled 62 patients in China that had progressed on gemcitabine-based chemotherapy. ORR was 11% in evaluable patients, MFS was 2.7 months and OS was 5.8 months.

Scripps Whittier Diabetes Institute reported data from the CONCLUDE trial comparing Tresiba (insulin degludec) to insulin glargine U300 in adults with type 2 diabetes uncontrolled on basal insulin with or without oral anti-diabetic drugs. The primary endpoint, which was the rate of overall symptomatic low blood sugar in the maintenance period of 36 weeks, was numerically lower but not statistically significantly lower in favor of Tresiba during the entire 88-week treatment period. It did decrease the rate of severe hypoglycemia by 80%.

Regeneron Pharmaceuticals and Sanofi published positive data from two Phase III trials of Dupixent (Idupilumab) in adults with recurring severe chronic rhinosinusitis with nasal polyps. The data, published in The Lancet, showed that adding Dupixent to standard-of-care nasal spray decreased the size of the polyps and decreased the severity of nasal congestion, which were the co-primary endpoints.

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