Clinical Catch-Up for September 31-October 4


It was another busy week for clinical trials. Although not exclusively, a lot of results came out of the European Society of Medical Oncology Congress (ESMO) held in Barcelona, Spain from September 27 through October 1. Here’s a look.

The University of Texas MD Anderson Cancer Center published results from its BEACON CRC Phase III clinical trial of 665 patients with BRAF V600E-mutated metastatic colorectal cancer who had disease progression after one or two previous treatments. Patients received either a triplet therapy of encorafenib, binimetinib, and cetuximab or a doublet therapy of encorafenib and cetuximab, or the investigators’ choice of either cetuximab and irinotecan or cetuximab and FOLFIRI (folinic acid, fluorouracil, and irinotecan), the control group. The median overall survival (OS) in the triplet group was 9 months, 5.4 months for the control group, and 8.4% in the doublet group.

SkylineDx announced validation of its skin cancer (melanoma) diagnostic test on the first, independent European dataset. The test uses genetic information from a patient’s tumor cells with patient-specific characteristics. The test accurately predicted the risk of metastases in the lymph nodes without a patient requiring surgery to remove part of the lymph nodes.

Stemedica reported positive results from the Phase IIa clinical trial of its IV infusion of allogeneic ischemia-tolerant mesenchymal stem cells (MSC) in chronic stroke. The primary outcome was safety based on behavior, CT scans and lab results. Secondary endpoints were related to a variety of behavioral changes, including neurological assessments, daily tasks, mental status and depression.

Ayala Pharmaceuticals presented encouraging preliminary results from its ongoing Phase II ACCURACY trial of AL101 in patients with recurrent/metastatic adenoid cystic carcinoma (ACC) with progressing disease and Notch activating mutations. AL101 is an investigational small molecule, gamma-secretase inhibitor that selectively inhibits Notch 1, 2, 3 and 4. There are currently no standard drug therapies for ACC.

Checkpoint Therapeutics announced positive interim results for its ongoing Phase I trial of cosibelimab in cutaneous squamous cell carcinoma (CSCC) and non-small cell lung cancer (NSCLC). Cosibelimab is a differentiated high affinity anti-PD-L1 antibody with functional Fc domain. So far, in the CSCC cohort there was a 50% objective response rate (ORR) and in the NSCLC cohort a 40% ORR.

Tyme presented supporting data of its Phase II TYME-88-Panc trial of SM-88 (racemetyrosine) as an oral monotherapy in patients with advanced pancreatic cancer. It showed encouraging overall survival (OS) results and a well-tolerated safety profile. The company also presented data from its Phase II trial of SM-88 in patients with non-metastatic, biochemical-recurrent prostate cancer that were positive.

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ORYZON Genomics released additional data from CLEPSIDRA, its ongoing Phase II trial of iadademstat in combination with standard-of-care in relapsed extensive disease (ED) small cell lung cancer (SCLC). The combination showed promising clinical efficacy with 6 of 8 patients. There were 4 partial remissions and 2 long-term disease stabilizations. Iadademstat is an inhibitor of the epigenetic enzyme LSD1. It also released data on its central nervous system epigenetic drug vafidemstat. This was from three different cohorts of psychiatric patients in the Phase II REIMAGINE trial: attention deficit hyperactivity disorder (ADHD), autistic spectrum disorder (ASD) and borderline personality disorder (BPD).

Cellectar Biosciences announced data from the diffuse large B-cell lymphoma (DLBCL) cohort in its Phase II CLOVER-1 trial of CLR 131 in relapsed or refractory select B-cell malignancies. Interim data showed durable responses, including a 33% ORR, a 16.6% complete response rate (CR) and a 50% clinical benefit rate (CBR).

Tarveda Therapeutics presented data from the Phase I part of its Phase I/IIa clinical trial of PEN-866 for a variety of solid tumors. Early evidence showed the drug was well tolerated and had anti-tumor activity. PEN-866 is a small molecule miniature drug conjugate linked to a potent topoisomerase 1 inhibitor (SN-38) payload that targets and binds to Heat Shock Protein 90 (HSP90).

Adaptimmune updated data from its ongoing Phase I trial with SPEAR T-cells targeting MAGE-A4 in synovial sarcoma. The data showed a clear benefit. “These results are truly meaningful in this rare and deadly disease because patients with advanced synovial sarcoma have very few treatment options,” said Elliot Norry, the company’s interim chief medical officer. “These data and the recent FDA orphan drug designation for ADP-A2M4 in sarcoma are important positive steps to expedite further development.”

AstraZeneca and Merck presented results from the Phase III PROfound trial in metastatic castration-resistant prostate cancer (mCRPC) who have a mutation in their homologous recombination repair (HRR) genes and whose disease has progressed on previous treatment with abiraterone or enzalutamide. The trial showed a statistically significant and clinically meaningful improvement with Lynparza in the primary endpoint of radiographic progression-free survival (rPFS) in BRCA1/2 or ATM-mutated tumors reducing the risk of disease progression or death by a median of 7.4 months versus 3.6 months.

Rakuten Medical released post-hoc analysis of safety, pharmacokinetic, immunogenicity and exploratory biomarker data from its RM-1929 photoimmunotherapy (PIT) Phase I and I/IIa studies. They evaluated the safety and anti-tumor activity of RM-1929 PIT in patients with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC). They reported clinically meaningful anti-tumor activity and a positive safety profile.

Zai Lab’s partner, GlaxoSmithKline, released results from PRIMA, the Phase III trial of niraparib as a monotherapy for the first-line maintenance of platinum responsive advanced ovarian cancer. Niraparib is a PARI inhibitor. The treatment resulted in a 38% reduction in the risk of disease progression or death in the overall population.

TLC, a special pharma company, initiated a Phase III trial of TLC599 in osteoarthritis of the knee. TLC599 is a BioSeizer sustained release formulation of dexamethasone sodium phosphate (DSP). The trial, EXCELLENCE, is a multi-center, randomized, double-blind, placebo- and active comparator-controlled pivotal trial that will look at about 500 patients with knee osteoarthritis at 40 to 50 sites in the U.S. and Australia.

Octernal Therapeutics opened a Phase Ib expansion cohort of its trial of cirmtuzumab in combination with ibrutinib in mantle cell lymphoma. Cirmtuzumab is a ROR1-targeted monoclonal antibody. Ibrutinib is marketed by Johnson & Johnson as Imbruvica.

Catalyst Biosciences updated enrollment on its Phase IIb trial of dalcinonacog alfa (DalcA). DalcA is a next generation subcutaneously (SQ) administered Factor IX therapy being developed for hemophilia B. Two subjects completed dosing and washout, and Factor IX levels exceeded the trial efficacy endpoint. Enrollment is ongoing. Catalyst expects final data in the first half of 2020.

Heron Therapeutics announced positive topline results from its Phase IIIb trial of HTX-011 in total knee arthroplasty (TKA). The trial showed the drug significantly reduced pain and opioid use compared to placebo through 72 hours and significantly decreased pain compared to bupivacaine solution, the standard-of-care local anesthetic for postoperative pain control. HTX-011 is a dual-acting, fixed-dose combination of bupivacaine with a low dose of the nonsteroidal anti-inflammatory meloxicam.

Novartis reported positive final results from its Phase III trial of Cosentyx for non-radiographic axial spondyloarthritis (nr-axSpA). The drug showed a significant and clinically meaningful decrease in disease activity compared to placebo. This included a sustained response and consistent safety profile for 52 weeks.

Eli Lilly presented data from its Phase IV IXORA-R trial, a head-to-head study of an IL-17A inhibitor and an IL-23/p19 inhibitor in plaque psoriasis. In the trial, Lilly’s Taltz (ixekizumab) showed superiority to Johnson & Johnson’s Tremfya (guselkumab) in patients with moderate to severe plaque psoriasis. The primary endpoint was superiority in the proportion of patients with moderate to severe plaque psoriasis achieving complete skin clearance as measured by the Psoriasis Area Severity Index (PASI) 100 at Week 12.

Sarepta Therapeutics announced the nine-month functional data from three Limb-girdle muscular dystrophy Type 2E (LGMD2E) patients who received SRP-9003. The therapy is a gene therapy that transduces skeletal and cardiac muscle with a gene that codes for the full-length, native beta-sarcoglycan protein, whose absence causes LGMD2E. All three patients showed improvements on functional measures and a significant decrease in creatine kinase that was maintained over nine months.

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