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Arix Bioscience PLC (LON:ARIX) said its portfolio firm, Aura Biosciences, has presented updated clinical data from an ongoing Phase 1b/2 clinical trial evaluating the safety and efficacy of light-activated AU-011, its lead candidate to treat primary choroidal melanoma, a rare and aggressive type of eye cancer.

The company said the data, presented as part of the Association for Research in Vision and Ophthalmology (ARVO) 2020 Annual Meeting, showed AU-011 was continuing to “demonstrate a favourable safety profile along with promising tumour control and vision preservation”.

“The clinical data presented this year at ARVO show that AU-011 has a favourable preliminary safety profile, controls tumour growth rate, and preserves vision in the vast majority of patients, including those at high risk for vision loss with tumours close to the fovea or optic nerve. We believe these data support further clinical investigation in pivotal studies”, said Prithvi Mruthyunjaya, Associate Professor of Ophthalmology and Director of Ocular Oncology Service at the Byers Eye Institute at Stanford University.

Positive data from Imara

In another release on Friday afternoon, the company said another portfolio firm, Imara Inc (NASDAQ:IMRA) has presented positive interim results from an ongoing Phase 2a clinical trial of IMR-687, a potential treatment for patients suffering from sickle cell disease.

The data, which was presented at the 25th European Hematology Association (EHA) Annual Congress, showed that IMR-687 was safe and well tolerated as a monotherapy and in combination with hydroxyurea (HU).

The data also showed that in the higher dose cohort, IMR-687 mono therapy showed a “statistically significant increase” in the number of F-cells, red blood cells containing fetal haemoglobin, high levels of which have been demonstrated to improve symptomology and substantially lower disease burden in both patients with sickle cell disease and patients with beta-thalassemia.

“These second interim data presented at EHA help reinforce the potential of IMR-687 as a novel, potentially disease modifying approach in the treatment of rare blood disorders, including sickle cell disease”, said Imara president and chief executive Rahul Ballal.

“We know increasing foetal haemoglobin correlates with improved clinical outcomes, and these promising Phase 2a data, which include early trends in reducing painful vaso-occlusive crises, continue to give us confidence that IMR-687 could have a meaningful impact in treating sickle cell disease”, Ballal added.

Imara added that it is initiating a Phase 2b clinical trial to test higher doses of IMR-687 for up to a one-year duration of therapy, anticipating that the increased exposure and duration “may result in further increases in [foetal haemoglobin] and, in turn, result in improved clinical outcomes for patients”.

Progress for Autolus CAR T cell therapy

In a separate announcement earlier on Friday morning, Arix highlighted new data announced by its portfolio firm, Autolus Therapeutics PLC (NASDAQ:AUTL) which shows progress on AUTO1 programme, its CAR T cell therapy.

Arix – which owns a 6.5% stake in Autolus – noted that the portfolio firm’s CAR T cell therapy is being investigated in the ongoing ALLCAR Phase 1/2 study of relapsed/refractory adult B-Acute Lymphocytic Leukemia (ALL), at the European Hematology Association EHA25 Virtual Congress beginning on June 11, 2020.

READ: Arix Bioscience portfolio companies Autolus and Harpoon present updates at ASCO

In its statement, Autolus pointed out that, as of the data cut-off date of May 13, 2020, 19 patients had received AUTO1.

It said AUTO1 was well tolerated, with no patients experiencing ≥ Grade 3 CRS, while three patients (16%) with high leukemia burden (>50% blasts) experienced Grade 3 neurotoxicity that resolved swiftly with the application of steroids.

Autolus noted that, of the 19 patients, 16 (84%) achieved MRD-negative CR, while two out of 16 patients received a transplant when in remission and CD19-negative relapse occurred in 3 (16%) patients.

The company concluded that the durability of remissions is encouraging, with Event Free Survival (EFS) and Overall Survival (OS) at 6 months being 62% and 72%, respectively, in all patients, and 76% and 92% respectively in the 13 patients treated with the closed (commercial) process.

It also noted that median EFS and OS has not been reached, at a median follow up of 12.2 months (range up to 24.4 months).

The full announcement can be accessed on Autolus’ investor website at

Investors can also listen to a webcast and view an accompanying slide presentation at

Shares in Arix were 2.7% lower at 90.5p in late-afternoon trading on Friday.

–Adds Aura and Imara announcements, updates share price–