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Aura Biosciences Announces Update of Clinical Safety and Efficacy Data on Lead Candidate AU-011 for Choroidal Melanoma

Aura Biosciences, a biotechnology company developing a new class of
therapies to target and selectively destroy cancer cells using viral
capsid conjugates, today announced new interim safety and efficacy data
from an open-label Phase 1b/2 study of its lead program, light-activated
AU-011 for the treatment of primary choroidal melanoma. The findings
were presented by Ivana Kim, M.D., Co-Director of the Ocular Melanoma
Center at Massachusetts Eye and Ear, at the 2018 World Ophthalmology
Congress in Barcelona.

AU-011 is an investigational, first-in-class targeted therapy in
development for the treatment of primary choroidal melanoma. FDA has
already granted Fast Track Designation and orphan drug designation,
recognizing that there are no FDA-approved therapies and that the
disease is serious and life-threatening.

AU-011 is being evaluated in a Phase 1b/2 open-label, multicenter trial
designed to evaluate the safety and efficacy of single and multiple
ascending doses in 30 adult subjects with clinically diagnosed small to
medium primary choroidal melanoma.

Interim data presented today show that AU-011 has been generally
well-tolerated with no related serious adverse events, no severe adverse
events and no dose-limiting toxicities observed. Adverse events were
manageable with standard of care treatments and had no further clinical
sequelae. Pre-treatment visual acuity was maintained in all subjects
that have been followed for 6 to 12 months.

Early efficacy results are very promising with two subjects in the first
multiple-ascending-dose cohort showing evidence of reduction in tumor
height at 3 months. Further evidence of preliminary efficacy has also
been demonstrated with subtherapeutic doses in the single-ascending-dose
cohorts providing stable disease with vision preservation up to 12
months.

“We are excited by these preliminary findings showing AU-011 provided
local tumor control without loss of visual acuity for a majority of
subjects dosed to date,” said Dr. Kim. “That is an encouraging sign of
progress toward developing a new treatment for this cancer that could
preserve much more vision than radiotherapy, which is the current
standard of care but not FDA approved for this indication.”

“These findings indicate that Aura’s novel, targeted, light-activated
treatment could hold real promise for patients with choroidal melanoma,”
said Cadmus Rich, M.D., Chief Medical Officer of Aura. “Our team is
looking forward to continuing our Phase 1b/2 study with Dr. Kim and our
other collaborators at leading ophthalmology centers across the country.”

About choroidal melanomaChoroidal melanoma is a rare and
aggressive type of eye cancer. Choroidal melanoma is the most common
primary ocular tumor and develops in the uveal tract of the eye. No
targeted therapies are available at present, and current radiotherapy
treatments can be associated with severe visual loss and other long-term
sequelae such as dry eye, glaucoma, cataracts and radiation retinopathy.
The most common current treatment is plaque radiotherapy, which involves
surgical placement of a radiation device against the exterior of the eye
over the tumor. This technique can control the melanoma but can also
lead to radiation-related cataract, retinopathy, optic nerve damage and
loss of vision. The alternative is enucleation, or removal of the eye.
Choroidal melanoma metastasizes to the liver in about 40 percent of
cases in the long term (source: OMF),
and only 15 percent of patients whose melanoma has metastasized survive
beyond five years after diagnosis (source: ACS).

About light-activated AU-011AU-011 is a first-in-class
targeted therapy in development for the primary treatment of choroidal
melanoma. The therapy consists of patented viral capsid conjugates (VCC)
with IR-700DX dye molecules that are activated with an ophthalmic laser.
The VCCs bind selectively to unique receptors on cancer cells in the eye
and are derived from technology originally pioneered by Dr. John
Schiller of the Center for Cancer Research at the National Cancer
Institute (NCI), recipient of the 2017 Lasker-DeBakey Award. The
IR-700DX dye molecules are produced by LI-COR Biosciences and are
licensed exclusively to Aura for treating ocular cancers. Upon
activation with an ophthalmic laser, the drug rapidly and specifically
disrupts the membranes of tumor cells while sparing key eye structures,
which may allow for the potential of preserving patients’ vision and
reducing other long-term complications of radiation treatment. AU-011
can be delivered using equipment commonly found in the ophthalmologist’s
office and does not require a surgical procedure, pointing to a
potentially less invasive, more convenient therapy for patients and
physicians. 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.

About Aura BiosciencesAura Biosciences is developing a new
class of therapies to selectively target and destroy cancer cells. Its
lead program, AU-011 in choroidal melanoma, is being developed under a
CRADA with the National Cancer Institute (NCI), part of the National
Institutes of Health. For more information, visit www.aurabiosciences.com.

About Massachusetts Eye and EarMassachusetts
Eye and Ear, founded in 1824, is an international center for
treatment and research and a teaching hospital of Harvard Medical
School. A member of Partners HealthCare, Mass. Eye and Ear specializes
in ophthalmology (eye care) and otolaryngology–head and neck surgery
(ear, nose and throat care). Mass. Eye and Ear clinicians provide care
ranging from the routine to the very complex. Also home to the world’s
largest community of hearing and vision researchers, Mass. Eye and Ear
has pioneered new treatments for blindness, deafness and diseases of the
head and neck. Our scientists are driven by a mission to discover the
basic biology underlying these conditions and to develop new treatments
and cures. For more information about life-changing care and research at
Mass. Eye and Ear, please visit our blog, Focus
or visit MassEyeAndEar.org.

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