Apellis Trial Progress And Other News: The Good, Bad And Ugly Of Biopharma (NASDAQ:APLS)

Apellis Reports Completion of Enrollment for Two Phase 3 Studies

Apellis Pharmaceuticals Inc. (APLS) reported that it has completed enrollment for two of its Phase 3 studies which are DERBY and OAKS. Both the studies are designed to evaluate intravitreal pegcetacoplan for treating geographic atrophy (GA) secondary to age-related macular degeneration. There is currently no approved therapy for treating geographic atrophy.

Apellis has enrolled 1,259 patients across both the studies with 621 patients in DERBY study while OAKS has 638 patients in it. Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis, “People living with GA need a treatment and we believe targeting the complement system at C3 with pegcetacoplan has the potential to control the excessive complement activation that drives the irreversible growth of GA lesions that cause blindness.” The company expects the topline data to be available by the third quarter of 2021.

These trials are pivotal randomized Phase 3 studies and aim to evaluate the efficacy and safety of intravitreal pegcetacoplan with sham treatment in patients suffering from GA secondary to AMD. The primary endpoint of these trials is to analyze the decline in growth of GA lesion size. This metric is measured using fundus autofluorescence. The readings taken at month 12 are to be compared to baseline. Both the studies are multicenter, randomized and double-masked trials.

Apellis had carried out Phase 2 study FILLY with 246 patients. The trial was conducted across over 40 clinical sites in the United States, New Zealand and Australia. The study met its primary endpoint of decreasing the growth rate of the GA lesion compared to sham after 12 months of treatment. The drug candidate was administered monthly via intravitreal injection. It demonstrated a 29 percent decline in the rate of GA lesion growth. When the drug candidate was given every other month, the reduction was reported at 20 percent.

Intravitreal pegcetacoplan was found to be well tolerated in Phase 2 trial. During the course of 18 months period, 26 cases of exudative AMD were registered. 18 of these cases were from monthly treatment group while 7 were from every other month treatment group. The remaining case belonged to the sham group. However, no case of negative impact on visual acuity was shown. The Phase 3 DERBY and OAKS studies are commenced based on positive outcomes from the Phase 2 FILLY study.

Pegcetacoplan is an investigational targeted C3 therapy. The drug candidate works by controlling excessive complement activation, which may cause the start and progress of several serious illnesses. It is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer and binds exclusively to C3 and C3b. Apellis is assessing this drug candidate for several indications such as paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy, cold agglutinin disease and geographic atrophy.

Pegcetacoplan has been given Fast Track tag by the FDA for treating GA and PNH. Apart from Pegcetacoplan, Apellis is also working on other novel therapeutic compounds for the treatment of different debilitating autoimmune diseases using complement immunotherapy. The company is the first one to get chronic therapy with a C3 inhibitor to clinical trial level.

Novocure Advances Hepanova Trial For Advanced Liver Cancer

Novocure Ltd. (NVCR) reported its progress for Hepanova trial for testing Tumor Treating Fields in arrangement with sorafenib for treating patients with advanced liver cancer. This phase 2 pilot trial involves 25 patients with advanced hepatocellular carcinoma that are not qualified for surgical resection or local treatments.

The primary endpoint for the drug candidate is overall response rate. Secondary endpoints for the trial are in-field control rate, progression free survival rate at 12 months, overall survival rate at 1 year and distant metastases free survival rate at 1 year. Asaf Danziger, Novocure’s Chief Executive Officer said, “Tumor Treating Fields therapy has demonstrated efficacy in in vitro and in vivo models of hepatocellular carcinoma. We believe that Tumor Treating Fields’ mechanism of action is broadly applicable to solid tumor cancers.”

Tumor Treating Fields have shown effectiveness in in vitro and in vivo models of hepatocellular carcinoma. It can be delivered to the abdominal region. The data collated from preclinical studies showed that Tumor Treating Fields delivered at 150 kHz lessened HepG2 and Huh-7D12 cell counts as well as clonogenic potential. The use of Tumor Treating Fields in conjunction with sorafenib resulted in significant decline in the number of HepG2 and Huh-7D12 cells in comparison to each treatment given alone.

As per the data, Tumor Treating Fields delivered at 150 kHz plus sorafenib resulted in reduction of viability and clonogenicity. It also increased apoptosis and autophagy in vitro while causing a significant reduction in tumor volume in vivo. Currently the treatment using Tumor Treating Fields is not approved for liver cancer.

Tumor Treating Fields is a cancer therapy which works by turning electric fields into specific frequencies for disrupting cell division. Using this mechanism, the tumor growth may be inhibited, potentially leading to the decay of cancer cells. This type of treatment does not heat or stimulate tissue.

Outlook Therapeutics Provides Update on NORSE 2 Trial

Outlook Therapeutics Inc. (OTLK) provided updates about its NORSE 2 clinical trial seeking to evaluate ONS-5010 for user in retinal indications. The trial had started its patient enrollment in July 2019 and has registered 227 patients across 39 clinical trials in the United States. The stated enrollment goal for the trial was pegged at 220 patients.

Outlook Therapeutics is looking to report safety and efficacy data by the third quarter of 2021. ONS-5010 has the potential to be the first approve drug in its class ophthalmic formulation of bevacizumab for use in multiple retinal indications. Mark Humayun M.D., Medical Advisor for Outlook Therapeutics said, “Within the retina community, the use of anti-VEGF therapy continues to be the standard of care for many ophthalmic diseases. Given the high cost of the present FDA-approved anti-VEGF therapies, many physicians have turned to off-label repackaged bevacizumab supplied by compounding pharmacists.” ONS-5010 is a full-length, humanized anti-VEGF recombinant monoclonal antibody. In wet AMD, there is abnormally elevated level of secretion of VEFG in the eye.

The primary endpoint for the trial is the difference in proportion of patients who gain at least 15 letters in the best corrected visual acuity at 11 months. For this purpose, ONS-5010 dosed on a monthly basis will be compared to LUCENTIS given quarterly per the PIER regimen.

The company is looking to submit the BLA for the drug candidate under its PHSA regulatory pathway. It will be submitting for regulatory approvals in Japan, European Union and other countries. Upon approval, the drug candidate will be the first and only on-label ophthalmic formulation of bevacizumab for treating retinal diseases.

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