Phase 2


Redefining Targeted Therapies


Aura Biosciences, based in Boston, MA is developing a new class of therapies to target and destroy cancer cells selectively, while leaving surrounding tissue unharmed and stimulating the immune system. By safely eliminating cancer locally, the goal is to can transform the lives of people with a wide range of cancers that are poorly managed today. Their lead clinical programme in ocular melanoma (OM), also known as choroidal or uveal melanoma, is designed to remove cancer cells in the back of the eye as a first-line therapy, while allowing for the potential of preserving patients' vision and avoiding the comorbidities of radioactivity. The goal is to treat small ocular melanomas locally, potentially long before the disease progresses and metastasizes to the liver, where it almost always is fatal. Development of a first-in-class, non-radioactive treatment option to selectively destroy cancer cells stimulating the immune system would create the possibility to transform the treatment of this and other cancers where the disease can be detected early. By enabling physicians to treat cancer more selectively, effectively and safely than they can do today, Aura aim's to eliminate the need for invasive procedures that carry significant morbidity, including vision loss, and which often do little to improve a patient's overall survival.


The Aura team combines expertise in cancer cell biology, ophthalmology and targeted therapies together with experience in the development and commercialisation of orphan drugs for highly unmet medical needs

  • Elisabet de los Pinos, Chief Executive Officer
  • Julie Feder, Chief Financial Officer
  • Cadmus Rich, Chief Medical Officer
  • Stephen Monks, Senior Vice President of Development
  • Michele Keough, Senior Vice President of R&D operations

Unmet need

Choroidal melanoma is a rare and aggressive type of eye cancer. There are no drugs approved and current radiotherapy treatments are associated with severe visual loss and other long-term sequelae such as dry eye, glaucoma, cataracts and radiation retinopathy.

Market potential

  • 11,000 patients diagnosed annually
  • Estimates of ~$700m market worldwide

Novel technology – Tumour targeted viral-like particle bioconjugates

Aura's light-activated technology consists of virallike particles, modelled on the human papillomavirus (HPV), conjugated to infrared-activated small molecules. The particles are administered through an intravitreal injection into the eye. The viral-like particle bioconjugates (VPBs) then bind selectively to cancer cells in the eye.

Upon activation with an ophthalmic laser, the small molecules generate reactive oxygen species that selectively destroy the membrane integrity of cancer cells, leading to acute cellular necrosis and activating the immune system without damaging the overlaying retina, with the goal of enabling vision preservation for patients.


AU-011 is a first-in-class targeted therapy in development for the primary treatment of choroidal melanoma. The therapy consists of proprietary viral-like particle bioconjugates (VPB) that are activated with an ophthalmic laser. AU-011 for the treatment of choroidal melanoma has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration and is currently in clinical development.

Ocular oncologyPrimary choroidal melanoma
Choroidal metastases
Cancers of the ocular surface
Solid tumoursPrimary bladder carcinoma
Technology platformImmuno-oncology
Gene therapy

Intellectual property

  • 11 Patents issued; 4 Allowed Applications; 24 Pending Applications
  • Patent protection to end of 2034
  • Claims cover AU-011 composition of matter and method of use as well as technology platform
  • All patents owned/co-owned by Aura or exclusively licensed to Aura
  • Worldwide commercial rights

Milestones achieved

Summary of results of AU-011 Phase 1b/2 study

  • Single and multiple administrations of AU-011 are well tolerated
  • Inflammation is manageable, starts around the tumour and supports MOA
  • Steroids can be started after inflammation is observed – allows immune response
  • Preliminary results show re-treatment after 12 months is safe and well tolerated
Biological activity
  • Reduction in tumour thickness and loss of orange pigment in multiple patients
  • Biopsy eight months after treatment demonstrates no remaining melanoma cells and presence of inflammatory and RPE cells
Efficacy endpointsTumour control
  • 90% of subjects at therapeutic dose with single cycle up to six months follow-up
  • Durability of tumour response observed >12-18 months, even at subtherapeutic doses in SAD (first two subjects with 24-month follow-up in March 2019)

Vision Preservation
  • Vision is preserved at six months or longer (up to 18 months)
  • Vision preserved after treatment even in high-risk patients

Future milestones

  • Phase 3 trial expected to start in 1H 2020, interim results at ~18 months and primary at 24 months
  • Possibility for NDA filing in 2023