AU-011 Demonstrates Targeted Cytotoxicity In Vitro and In Vivo in Both Breast and Lung Tumors, Demonstrating the Potential to Treat Human Choroidal Metastases

Tumor Distribution of AU-011 in the Treatment of Choroidal Melanoma Also Presented Using Intravitreal and Suprachoroidal Administration

CAMBRIDGE, MA – May 3, 2021 – Aura Biosciences, a clinical-stage oncology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the presentation of supportive preclinical data in multiple tumor models of choroidal metastases and in choroidal melanoma using suprachoroidal administration. The data will be featured in two virtual poster presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, taking place May 1-7, 2021.

Choroidal metastasis is a second ocular oncology indication for Aura’s lead VDC candidate AU-011, which is also currently in Phase 2 development for the treatment of choroidal melanoma. New preclinical data presented at the meeting demonstrates the potential of AU-011 for the treatment of choroidal metastases from breast and lung cancer, which are the most common causes of metastases in the choroid.

 “Thanks to recent advances with treatment options for metastatic cancer, patients are living longer, and the incidence of choroidal metastases from breast and lung cancer has become an increasingly important issue that dramatically impacts patient’s vision and their overall quality of life,” said Amy Schefler, MD, FACS, Associate Professor of Clinical Ophthalmology, Weill Cornell Medical College and Ocular Oncology Specialist, Retina Consultants of Houston. “The data show that AU-011 has the potential to offer a new, vision preserving targeted therapy that may eliminate the need for the treatment with local radiotherapy for many patients with choroidal metastases. We believe these data support the further clinical development of AU-011 in this additional ocular oncology indication which has the potential to transform visual outcomes and the quality of life for patients with cancer and we look forward to sharing these results with the medical and scientific community at the meeting.”

Effect of AU-011 on Preclinical Models of Choroidal Metastases

The purpose of this study was to evaluate the potential of AU-011 to treat choroidal metastases by evaluating its effect on a panel of human breast and lung cancer cell lines in vitro and in vivo in a murine breast cancer model. In both cell line panels, AU-011 demonstrated potent cell binding and cell killing activity with EC50 in the picomolar range. In vivo, a single systemic administration of AU-011 in a syngeneic breast cancer mouse model demonstrated potent cytotoxic activity with significant reduction of tumor growth, further corroborating data previously obtained in other cancer models.  These results suggest that AU-011 can bind to and kill tumor cells derived from the most common cancer types known to metastasize to the choroid and supports further development of AU-011 as a vision preserving therapy for the treatment of choroidal metastases.

Tumor distribution of AU-011 with IVT and SC Administration in an Orthotopic Rabbit Model of Uveal Melanoma

Aura evaluated the distribution of AU-011 with both intravitreal (IVT)  and suprachoroidal (SC) administration in an orthotopic rabbit model of choroidal melanoma, which demonstrated both routes of administration achieved tumor distribution to the choroid and to the tumor. Following SC administration, negligible levels of AU-011 were observed in the vitreous, but high exposure levels were observed in the tumor and choroid/retina. The exposure of AU-011 in the tumor was approximately 5 times higher when AU-011 was administered by SC injection compared to IVT injection. The exposure remained high in the tumor up to 48 hours post injection for both routes of administration.

Cadmus Rich, MD, MBA, Chief Medical Officer and Head of Research and Development at Aura Biosciences commented, “The suprachoroidal delivery route has the potential to offer certain advantages over intravitreal injection, including superior distribution, higher bioavailability and less unintended exposure in the vitreous and other key ocular structures. We believe these characteristics may lead to less intraocular inflammation, an improved safety profile and a broader therapeutic index. A Phase 2 clinical trial evaluating SC administration of AU-011 in patients with choroidal melanoma is currently ongoing.”

Details for the ARVO 2021 presentations are as follows:

Title: Development of AU-011 for Choroidal Metastasis

Presenter: Cadmus Rich, Aura Biosciences

Abstract #: 3547059

Session:  Advances in intra and extra ocular pathologies

Date and time:  May 5, 2021 from 2:45 – 4:30 PM EDT

URL: https://arvo2021.arvo.org/meetings/virtual/rX366WKTbayR7Pehy

Title: Ocular Distribution and Exposure of AU-011 After Suprachoroidal or Intravitreal Administration in an Orthotopic Rabbit Model of Human Uveal Melanoma

Presenter: Anneli Savinainen, Aura Biosciences

Abstract #: 3543497

Session: Recent Advances in Uveal Melanoma

Date and time:  May 2, 2021 from 9:00 to 10:45 AM EDT

URL: https://arvo2021.arvo.org/meetings/virtual/Kicj9AczkcowRfxLs

The presentations can be accessed by ARVO members on the ARVOLearn website.  The presentations can also be accessed by visiting the “Presentations” section of “News and Publications” page of the Aura Biosciences website.

About Choroidal Melanoma and Choroidal Metastasis

Choroidal melanoma is a rare and life-threatening type of eye cancer. It is the most common primary intraocular cancer in adults 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 on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes in approximately 50 percent of patients with liver involvement in 80-90% of cases and, unfortunately, metastatic disease is universally fatal (source: OMF). Choroidal metastases is when cancer that started elsewhere in the body spreads to the eye.  The common primary cancers that metastasize to the choroid include, in descending order, breast, lung, gastrointestinal, kidney, skin and prostate. In 31% of cases a primary cancer diagnosis is not made at the time of the choroidal metastasis diagnosis. There is a very high unmet need for a new vision sparing targeted therapy that could enable early treatment intervention for this life-threatening rare disease given the lack of approved therapies, and the comorbidities of radioactive treatment options.

About Light-Activated AU-011

AU-011 is a first-in-class virus-like drug conjugate (VDC) therapy in clinical development for the first line treatment of choroidal melanoma. The virus-like component of the VDC selectively binds unique heparin sulphate proteoglycans (HSPGs) that are modified and overexpressed on the tumor cell surface of malignant cells in the choroid and delivers a potent cytotoxic drug that is activated with infrared light. Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant cells with a pro-immunogenic cell death that can activate the immune system generating long term anti-tumor immunity. The unique specificity of tumor binding by the VDC enables the preservation of 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 an 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 is currently in Phase 2 clinical development and the company plans to expand the clinical program into choroidal metastasis.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates (VDCs) to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity. The VDC technology platform is based on the discoveries of NIH Distinguished Investigator Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI). The company has the goal of developing this technology in multiple cancer indications with an initial focus in ocular oncology, a group of rare diseases for which there are no approved drugs. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of choroidal melanoma, a vision and life-threatening form of eye cancer where standard of care radioactive treatments leave patients with major vision loss and severe comorbidities. Aura has demonstrated the efficacy and safety of AU-011 in a Phase 1b/2 trial, including high rates of tumor control and vision preservation. Future pipeline applications for Aura’s technology include additional ocular oncology indications like choroidal metastases and solid tumor indications like non-muscle invasive bladder cancer.  Aura is headquartered in Cambridge, MA. For more information, visit www.aurabiosciences.com or follow us on Twitter.

Investor and Media Contact:

Joseph Rayne
Argot Partners
617.340.6075 | joseph@argotpartners.com

 Results Support VDCs induction of Pro Immunogenic Cell Death and the Generation of Long Lasting Adaptive Anti-Tumor Immunity –

Combination of VDCs with Checkpoint Inhibitors Achieves a High Complete Response Rate and Prevents Long Term Tumor Recurrence –

CAMBRIDGE, MA – April 14, 2021 – Aura Biosciences, a clinical-stage oncology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the online publication of data in the peer-reviewed medical journal Cancer Immunology Research, a journal of the American Association for Cancer Research, that supports the broad application of the Company’s proprietary VDC technology platform for treating cancer. The manuscript, titled, “Virus-like Particle-drug Conjugates Induce Protective, Long-lasting Adaptive Anti-Tumor Immunity in the Absence of Specifically Targeted Tumor Antigens,” describes promising long term anti-tumor activity of AU-011, the Company’s lead VDC candidate, as a monotherapy and in combination with checkpoint inhibitor antibodies in preclinical studies conducted in collaboration with the Center for Cancer Research at the National Cancer Institute of the National Institutes of Health.

“Collectively, these promising results confirm treatment of AU-011 resulted in targeted tumor cytotoxicity with hallmarks of immunogenic cell death that may promote a durable anti-tumor immune response,” said Cadmus C. Rich, MD, MBA, Chief Medical Officer and Head of R&D for Aura. “Additionally, the additive activity of AU-011 in combination with checkpoint inhibitors has shown a high level of durable complete responses and prevention of tumor recurrence, warranting continued research into its potential clinical utility to effectively treat multiple types of tumors like non-muscle invasive bladder cancer as a primary treatment and further prevent metastatic disease.”

Key findings from the manuscript include:

• In vitro and in vivo studies in immunocompetent murine tumor models demonstrated a dose-dependent cytotoxic response of AU-011 with an upregulation of the markers of immunogenic cell death like caspase-1 and calreticulin surface expression demonstrating that AU-011 mediated cell death was able to generate potent immune stimulatory conditions within the tumor microenvironment.

• A single in vivo dose administration of AU-011 caused rapid cell death leading to long term complete responses in 50% of all animals.  Combination with immune checkpoint inhibitor antibodies improved therapeutic efficacy resulting in 70-100% complete response rate that was durable 100 days post-treatment with 50-80% of those animals displaying protection from secondary tumor re-challenge.

• Depletion studies of CD4+ or CD8+ T-cells at the time of AU-011 treatment or tumor re-challenge confirmed the involvement of both cell populations in the mechanism of action of AU-011 and the promotion of long-lasting anti-tumor protection.

“These promising findings further reinforce the therapeutic advantages of VDCs in treating cancer compared to other available treatments, which include the broad tumor selectivity and multivalent binding of the virus-like particles compared to antibodies, the ability to deliver hundreds of cytotoxic molecules and the generation of long-lasting anti-tumor immunity,” said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. “While our initial clinical focus has been in ocular oncology, our VDC approach has wide application as a single agent and as a combination therapy in a variety of solid tumors, including non-muscle invasive bladder cancer, which is expected to enter the clinic in 2022 We remain focused on advancing our novel VDC approach to transform the treatment of tumors and improve outcomes for patients with cancer.”

About AU-011 (belzupacap sarotalocan)

AU-011 is a first-in-class virus-like drug conjugate (VDC) therapy in development for the first line treatment of choroidal melanoma. The virus-like component of the VDC selectively binds unique heparan sulphate proteoglycans (HSPGs) that are modified and overexpressed on the tumor cell surface of choroidal melanoma (and other tumor types) and delivers a potent cytotoxic drug that is activated with infrared light. Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant melanoma cells with a pro-immunogenic cell death that can activate the immune system generating long term anti-tumor immunity. The unique specificity of tumor binding by the VDC enables the preservation of key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. The possibility of early treatment intervention and the activation of the immune system could lead to a reduction in the metastases rate for patients with this life-threatening disease. AU-011 can be delivered using equipment commonly found in an 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 Phase 2 clinical development.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates (VDCs) to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity. The VDC technology platform is based on the discoveries of NIH Distinguished Investigator Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI). The company has the goal of developing this technology in multiple cancer indications with an initial focus in ocular oncology, a group of rare diseases for which there are no approved drugs. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of choroidal melanoma, a vision and life-threatening form of eye cancer where standard of care radioactive treatments leave patients with major vision loss and severe comorbidities. Aura has demonstrated the efficacy and safety of AU-011 in a Phase 1b/2 trial, including high rates of tumor control and vision preservation. Future pipeline applications for Aura’s technology include additional ocular oncology indications like choroidal metastases and solid tumor indications like non-muscle invasive bladder cancer.  Aura is headquartered in Cambridge, MA. For more information, visit www.aurabiosciences.com or follow us on Twitter.

Investor and Media Contact:

Joseph Rayne

Argot Partners

617.340.6075 | joseph@argotpartners.com

CAMBRIDGE, MA – March 24, 2021 – Aura Biosciences, a clinical-stage oncology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the appointment of Mark De Rosch, Ph.D. as Chief Operating Officer. In his role, he will be responsible for leading Aura’s global operations and regulatory strategy.

Dr. De Rosch brings to Aura more than 30 years of experience in leading global regulatory and development strategies across many therapeutic areas, including oncology and ophthalmology. He has expertise in transitioning companies from early-stage to late-stage and in building effective teams to achieve clinical and corporate objectives.

“Mark joins our team during an exciting time at Aura. His decades of experience with global regulatory strategy, as well as building successful teams and pipelines, will be invaluable as we work to realize the full potential of our VDC technology platform,” said Elisabet de los Pinos, Ph.D., founder and CEO of Aura. “His proven track record and strong scientific acumen aligns well with our goals as we advance AU-011 toward late-stage pivotal development.”

“I am delighted to join Aura at this important time in the Company’s history and I look forward to being part of its promising future, as we work to become a leader in ocular oncology,” said Dr. De Rosch. “Aura’s experienced team and the novel VDC technology make this a compelling opportunity. I look forward to contributing my global regulatory, operational and organizational expertise to grow the Company and advance this leading technology to transform the outcomes for cancer patients.”

 Dr. De Rosch joins Aura from Epizyme, where he served as Chief Regulatory Officer and led regulatory efforts for their first approved product, TAZVERIK® (tazemetostat). Prior to Epizyme, Dr. De Rosch served as Senior Vice President, Regulatory Affairs and Quality Assurance for Nightstar Therapeutics (acquired by Biogen in 2019), where he developed and implemented global regulatory roadmaps for their gene therapy programs in choroideremia and retinitis pigmentosa. Prior to Nightstar, he served as Senior Vice President, Regulatory Affairs, Quality Assurance and CMC at Akebia Therapeutics.  Before that, Dr. De Rosch served in roles of increasing responsibility at several life science and healthcare consulting firms. Dr. De Rosch holds a Ph.D. and an M.S. in inorganic chemistry from the University of California, San Diego and a B.S. in chemistry/biochemistry from the University of Wisconsin-Parkside.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates (VDCs) to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity. The VDC technology platform is based on the pioneering discoveries of NIH distinguished investigator Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI), recipient of the 2017 Lasker-DeBakey Award. The company has the goal of developing this technology in multiple cancer indications with an initial focus in ocular oncology, a group of rare diseases that have no drugs approved. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of choroidal melanoma, a vision and life-threatening form of eye cancer where standard of care radioactive treatments leave patients with major vision loss and severe comorbidities. In a Phase 1b/2 study, AU-011 demonstrated compelling efficacy, including high rates of tumor control and vision preservation including patients with tumors close to the fovea and optic disk, along with a favorable safety profile. Future pipeline applications for Aura’s technology include additional ocular oncology indications like choroidal metastases and solid tumor indications like non-muscle invasive bladder cancer.  Aura is headquartered in Cambridge, MA. For more information, visit www.aurabiosciences.com or follow us on Twitter.

 Investor and Media Contact:

Joseph Rayne

Argot Partners

617.340.6075 | joseph@argotpartners.com

Proceeds to Support Pivotal Program of AU-011 for the First Line Treatment of Choroidal Melanoma and Expansion of Virus-like Drug Conjugate (VDC) Platform in additional Ocular Oncology Indications and Solid Tumors

Lead Investors Matrix Capital Management and Surveyor Capital (a Citadel company) are Joined by New Investors Rock Springs Capital and Adage Capital Management LP, Along with All Existing Institutional Investors 

CAMBRIDGE, MA – March 22, 2021 – Aura Biosciences, a clinical-stage oncology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the closing of an oversubscribed $80 million financing. The financing was led by Matrix Capital Management and Surveyor Capital (a Citadel company) with participation from new investors, including Rock Springs Capital, Adage Capital Management LP and Velosity Capital. Existing investors Medicxi, Advent Life Sciences, Lundbeckfonden Ventures, Arix Bioscience, Chiesi Ventures, Ysios Capital and Columbus Venture Partners also participated in the round.

Aura intends to use the proceeds from this financing to advance the clinical development of its VDC technology platform, including the pivotal Phase 3 program for AU-011, the Company’s lead candidate in development for the first line treatment of choroidal melanoma, and ongoing research for additional programs in ocular oncology, as well as expanding the VDC technology into bladder cancer, the first non-ophthalmic solid tumor indication.

 “Aura is pioneering the development of a new class of targeted therapies for life-threatening cancers with our novel VDC technology platform. This funding from a syndicate of distinguished investors enables us to advance AU-011 into a pivotal Phase 3 program for the first line treatment of choroidal melanoma, a rare, life- and vision-threatening form of cancer with no drugs approved. It also allows us to continue to expand the reach of our VDC technology in additional ocular oncology indications and in the treatment of solid tumors like bladder cancer where there is a high unmet medical need for better targeted therapies to treat early and reduce the incidence of metastasis,” said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.

 In connection with this financing, Karan Takhar, Senior Managing Director of Matrix Capital Management, will join Aura’s Board of Directors.

 Mr. Takhar said, “Matrix believes in the long-term potential of Aura’s VDC technology to further strengthen the Company’s position as a leader in ocular oncology and beyond within other types of cancers in need of better treatment options. We look forward to supporting Aura’s leadership team through this next stage of pipeline growth and transition into late-stage development with the commencement of the AU-011 pivotal program.”

 About AU-011 (belzupacap sarotalocan)

AU-011 is a first-in-class virus-like drug conjugate (VDC) therapy in development for the first line treatment of choroidal melanoma. The virus-like component of the VDC selectively binds unique heparan sulphate proteoglycans (HSPGs) that are modified and overexpressed on the tumor cell surface of choroidal melanoma cells (and other tumors) and delivers a potent cytotoxic drug that is activated with infrared light. Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant melanoma cells with a pro-immunogenic cell death that is believed to activate the immune system generating long term anti-tumor immunity. The unique specificity of tumor binding by the VDC enables the preservation of key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. The possibility of early treatment intervention and the activation of the immune system could lead to a reduction in the metastatic rate for patients with this life-threatening disease. AU-011 can be delivered using equipment commonly found in an 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 Phase 2 clinical development.

 About Aura Biosciences

 Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates (VDCs) to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity. The VDC technology platform is based on the pioneering discoveries of NIH Distinguished Investigator Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI). The company has the goal of developing this technology in multiple cancer indications with an initial focus in ocular oncology, a group of rare diseases that have no drugs approved. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of choroidal melanoma, a vision and life-threatening form of eye cancer where standard of care radioactive treatments leave patients with major vision loss and severe comorbidities. Aura has demonstrated the efficacy and safety of AU-011 in a Phase 1b/2 trial, including high rates of tumor control and vision preservation. Future pipeline applications for Aura’s technology include additional ocular oncology indications like choroidal metastases and solid tumor indications like non-muscle invasive bladder cancer. Aura is headquartered in Cambridge, MA. For more information, visit www.aurabiosciences.com or follow us on Twitter.

Investor and Media Contact:

Joseph Rayne

Argot Partners

617.340.6075 | joseph@argotpartners.com

CAMBRIDGE, MA –January 5th, 2021– Aura Biosciences, a clinical-stage oncology company developing a novel class of drug conjugate therapies for multiple oncology indications, today announced the appointment of David Johnson to its Board of Directors. Mr. Johnson is a biopharmaceutical business leader with more than 25 years of experience in drug development and currently serves as Chief Executive Officer at VelosBio, a clinical-stage oncology company developing novel antibody-drug-conjugates and bispecific antibodies.

“With David’s impressive track record in oncology and as an accomplished CEO, his engagement and guidance will help Aura drive AU-011 for the treatment of choroidal melanoma to registration and commercialization,” said George Golumbeski, Ph.D., Chairman of the Board of Aura Biosciences. “In the last few years, David has led several biopharma transactions, including the acquisition of VelosBio by Merck for $2.75 billion. He has also successfully raised over $500 million in capital for his last two companies. We are really excited to have David’s strategic guidance as we work to accelerate the clinical development of our pipeline of innovative oncology therapies.”

 “I am excited to be joining Aura’s Board of Directors during such an important period of growth for the Company,” said Mr. Johnson. “I look forward to leveraging my extensive clinical, business development, operational and executive leadership experience in the biotechnology industry to support Aura’s Board of Directors and leadership team in achieving their goal of advancing a new class of oncology drugs for life-threatening cancers.”

 Prior to founding VelosBio, Mr. Johnson was the Chief Executive Officer at Acerta Pharma, an oncology-focused pharmaceutical company, where he led the Company through a critical phase of growth from approximately 40 to over 150 employees and from a signal-seeking, first-in-human trial to more than 20 active clinical studies. Under his leadership, Acerta designed and launched three registration-directed trials, including two global Phase 3 trials for acalabrutinib, an irreversible oral Bruton’s tyrosine kinase (BTK) inhibitor initially focused on hematological malignancies. Mr. Johnson and his leadership team ultimately led the acquisition of Acerta by AstraZeneca in a deal valued at up to $7 billion.

 Before Acerta, Mr. Johnson held roles with increasing responsibility within commercial, pipeline development, medical affairs, and clinical development organizations at various healthcare companies including Hoffman-La Roche, Immunex (acquired by Amgen), Millennium (acquired by Takeda), Favrille, Gloucester (acquired by Celgene), and Calistoga (acquired by Gilead). He has made significant contributions to drugs ultimately garnering regulatory approvals, including bortezomib (Velcade®), romidepsin (Istodax®), idelalisib (Zydelig®), and acalabrutinib (Calquence®). In addition to Aura’s Board, Mr. Johnson serves on the Board of Directors of Zentalis Pharmaceuticals, a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapeutics targeting fundamental biological pathways of cancers. Mr. Johnson received a Bachelor’s degree from Indiana University.

 About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biopharmaceutical company developing a new class of oncology therapies based on a novel drug conjugate technology for initial application in ocular and bladder cancers with the potential to treat other cancers. The Company’s proprietary technology platform utilizes virus-like drug conjugates (VDCs) that have a dual mechanism of action with targeted necrosis of cancer cells, followed by a T-cell mediated anti-tumor response. This novel technology platform uses Virus-Like Particles to bind to a novel tumor cell surface target and can deliver hundreds of cytotoxic molecules selectively to tumor cells, while sparing surrounding healthy tissue. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the first line treatment of choroidal melanoma, a vision and life-threatening form of eye cancer for which there are currently no approved therapies. In a Phase 1b/2 study, AU-011 demonstrated compelling efficacy, including high rates of tumor control and vision preservation, along with a favorable safety profile, in patients with choroidal melanoma. Future pipeline applications for Aura’s technology include choroidal metastases and non-muscle invasive bladder cancer.  Aura is headquartered in Cambridge, MA. For more information, visit www.aurabiosciences.com or follow us on Twitter.

 Investor and Media Contact:

Joseph Rayne

Argot Partners

617.340.6075 | joseph@argotpartners.com

Late Breaker to be Presented by Leading Ocular Oncologist, Carol L. Shields, M.D., Director, Ocular Oncology Service, Wills Eye Hospital

 A Total of Three Abstracts Highlighting AU-011 Selected for Presentation

CAMBRIDGE, MA – November 10, 2020 – Aura Biosciences, a clinical-stage biopharmaceutical company developing a novel class of tumor targeted therapies for initial application in primary tumors such as choroidal melanoma, today announced that an abstract has been selected as a late breaking presentation as part of the Retina Subspecialty Day at the upcoming American Academy of Ophthalmology (AAO) 2020 Virtual Annual Meeting, taking place November 14-17, 2020. The abstract details results from the ongoing intravitreal administration Phase 1b/2 clinical study and suprachoroidal administration Phase 2 clinical study evaluating AU-011 in patients with choroidal melanoma.

This late-breaking presentation will be given by Carol L. Shields, M.D., Director, Ocular Oncology Service at Wills Eye Hospital, and Professor of Ophthalmology at Thomas Jefferson University. Other key presentations will be given by Brian P. Marr, M.D., Director, Division of Ophthalmic Oncology, New York-Presbyterian/Columbia Medical Center and Ivana K. Kim, M.D., Co-Director, Ocular Melanoma Center, Massachusetts Eye and Ear, and Associate Professor of Ophthalmology, Harvard Medical School, and will also highlight updated clinical data from Aura’s ongoing Phase 1b/2 AU-011 clinical trial, and the overall development plan including the ongoing Phase 2 study evaluating suprachoroidal administration of AU-011, also in patients with primary choroidal melanoma.

Details for the AAO 2020 presentations are as follows:

Late breaker title: AU-011, a Targeted Therapy for Primary Treatment of Choroidal Melanoma (CM) via Intravitreal (IVT) and Suprachoroidal (SC) Administration

Presenter: Carol S. Shields, MD, Wills Eye Hospital

Session: RET10V

Date and time: Friday, November 13, 2020 from 3:15 – 3:20 PM PT

Location: Virtual Live Meeting Broadcast

Title: AU-011 for Choroidal Melanoma: Trial Update

Presenter: Brian P. Marr, MD, New York-Presbyterian/Columbia medical Center

Session: PTH05V

Date and time: Friday, November 13, 2020 from 4:02 – 4:07 PM PT

Location: Virtual Live Meeting Broadcast

 

Title: Update from an Ongoing Phase 1b/2 Open-label Trial with Intravitreal (IVT) AU-011 for Choroidal Melanoma (CM) and Further Development Plan

Presenter: Ivana K. Kim, MD, Massachusetts Eye and Ear

Session: PA034

Date and time: Available on demand Nov 14-15, 2020

Location: Virtual Meeting Live Broadcast

 

About Choroidal Melanoma

Choroidal melanoma is a rare and life-threatening type of eye cancer. It is the most common primary intraocular cancer in adults 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 on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes in approximately 50 percent of patients with liver involvement in 80-90% of cases and, unfortunately, metastatic disease is universally fatal (source: OMF). There is a very high unmet need for a new vision sparing targeted therapy that could enable early treatment intervention for this life-threatening rare disease given the lack of approved therapies, and the comorbidities of radioactive treatment options.

About AU-011 (belzupacap sarotalocan)

AU-011 is a first-in-class targeted therapy in development for the treatment of primary choroidal melanoma. The therapy consists of proprietary viral-like particle bioconjugates (VPB) that are activated with an ophthalmic laser. The VPBs 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. Upon activation with an ophthalmic laser, the drug rapidly and specifically disrupts the cell membrane of malignant 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 an 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 Suprachoroidal Administration

In addition to intravitreal administration, Aura is also investigating AU-011 using the suprachoroidal (SC) route of administration.  Aura believes that delivering AU-011 into the suprachoroidal space (SCS) ™ within the eye, has the potential to offer certain advantages over intravitreal injection, including higher bioavailability at the tumor site and reduced exposure of non-targeted tissues, which may lead to an improved therapeutic index for AU-011.  Collectively, these features could allow for the treatment of a wider range of tumor sizes, and, therefore, a larger number of patients.  The Company is partnered with Clearside Biomedical for use of Clearside’s SCS Microinjector™ for administration of AU-011 into the SCS.  In preclinical research presented as part of the ARVO 2020 virtual program, AU-011 showed excellent distribution in the SCS, complete necrosis of tumors following laser activation in an animal model of choroidal melanoma and no clinical signs of anterior segment or posterior segment inflammation.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biopharmaceutical company developing a new class of oncology therapies based on a novel tumor targeting technology for initial application in primary tumors such as ocular and bladder cancers with the potential to treat other cancers.  The Company’s proprietary technology platform utilizes viral-like particle bioconjugates (VPBs) that have a dual selectivity mechanism with targeted binding to cancer cells, followed by activation using a common laser.  These potent cytotoxic therapies result in acute tumor cell necrosis, while sparing surrounding healthy tissue and activating the immune system to generate an anti-tumor response.  Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the treatment of choroidal melanoma, a vision and life-threatening form of eye cancer for which there are currently no approved therapies.  In a Phase 1b/2 study, AU-011 demonstrated compelling efficacy, including high rates of tumor control and vision preservation, along with a favorable safety profile, in patients with choroidal melanoma.  The development of AU-011 is partially supported by funding awarded under a CRADA from the U.S. National Institutes of Health’s National Cancer Institute.  Future pipeline applications for Aura’s technology include choroidal metastases and primary bladder carcinoma.  Aura is headquartered in Cambridge, MA.  For more information, visit www.aurabiosciences.com or follow us on Twitter.

Investor and Media Contact:

Joseph Rayne

Argot Partners

617.340.6075 | joseph@argotpartners.com

CAMBRIDGE, MA – September 11, 2020 – Aura Biosciences, a clinical-stage biopharmaceutical company developing a novel class of tumor targeted therapies for initial application in primary tumors such as choroidal melanoma, today announced the dosing of the first patient in a Phase 2 study evaluating the safety and efficacy of suprachoroidal (SC) administration of AU-011, the Company’s lead product candidate, as a potential first-line treatment for patients with primary choroidal melanoma.

“I am honored to have enrolled the first subject in this exciting trial of AU-011 that, if approved, may offer patients the first targeted therapy that has the potential to not only control tumors but also to preserve vision in the majority of those treated — which could usher in a very beneficial paradigm shift in the management of this challenging disease,” commented Timothy S. Fuller, M.D. from Texas Retina Associates in Dallas TX. “In addition, the suprachoroidal injection is a straightforward procedure that ocular oncologists should easily be able to perform.”

Carol L. Shields, M.D., Director, Ocular Oncology Service at Wills Eye Hospital, Professor of Ophthalmology at Thomas Jefferson University, and Lead Investigator of the study, commented, “Choroidal melanoma is the most common intraocular cancer in adults and is of grave concern for patients based on its potential to develop into metastatic disease. We are very excited to partner with Aura Biosciences in the launch of this innovative clinical trial on behalf of the patients who need a new first line treatment option for early stage disease, where all current treatments are extremely invasive and result in severe vision loss in many patients. We believe this approach could provide an opportunity for the early treatment of a broader range of patients with this deadly disease.”

“Dosing of the first patient in this Phase 2 study is a key milestone for both Aura and the AU-011 development program,” said Cadmus C. Rich, M.D., M.B.A., Chief Medical Officer and Head of R&D for Aura Biosciences. “The suprachoroidal delivery route is compelling because it offers certain potential advantages over intravitreal injection, including higher bioavailability of drug to the posterior segment of the eye where choroidal melanomas are located, and reduced exposure of non-targeted tissues which may lead to an improved safety profile.”

The first cohort of this Phase 2 study (NCT04417530) demonstrated favorable safety data with no safety findings and no adverse events noted. The objectives of the study include the assessment of safety and preliminary efficacy of the treatment of choroidal melanoma utilizing suprachoroidal administration of AU-011. Other objectives include determining the highest tolerated dosing regimen, as well as assessing immunogenicity.

About Choroidal Melanoma

Choroidal melanoma is a rare and aggressive type of eye cancer. Choroidal melanoma is the most common primary intraocular tumor in adults 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 on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes in approximately 50 percent of cases with liver involvement in 80-90% of cases and, unfortunately, metastatic disease is universally fatal (source: OMF). There is a very high unmet need for a new vision sparing targeted therapy that could enable early treatment intervention for this life-threatening rare disease given the lack of approved therapies, and the comorbidities of radioactive treatment options.

About AU-011 (belzupacap sarotalocan)

AU-011 is a first-in-class targeted therapy in development for the treatment of primary choroidal melanoma. The therapy consists of proprietary viral-like particle bioconjugates (VPB) that are activated with an ophthalmic laser. The VPBs 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. Upon activation with an ophthalmic laser, the drug rapidly and specifically disrupts the cell membrane 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 an 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 Suprachoroidal Administration

In addition to intravitreal administration, Aura is also investigating AU-011 using the suprachoroidal (SC) route of administration. Aura believes that delivering AU-011 into the suprachoroidal space (SCS) ™ within the eye, has the potential to offer certain advantages over intravitreal injection, including higher bioavailability at the tumor site and reduced exposure of non-targeted tissues, which may lead to an improved therapeutic index for AU-011. Collectively, these features could allow for the treatment of a wider range of tumor sizes, and, therefore, a larger number of patients. The Company is partnered with Clearside Biomedical for use of Clearside’s SCS Microinjector™ for administration of AU-011 into the SCS. In preclinical research presented as part of the ARVO 2020 virtual program, AU-011 showed excellent distribution in the SCS, complete necrosis of tumors following laser activation in an animal model of choroidal melanoma and no clinical signs of anterior segment or posterior segment inflammation.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biopharmaceutical company developing a new class of oncology therapies based on a novel tumor targeting technology for initial application in primary tumors such as ocular and bladder cancers with the potential to treat other cancers. The Company’s proprietary technology platform utilizes viral-like particle bioconjugates (VPBs) that have a dual selectivity mechanism with targeted binding to cancer cells, followed by activation using a common laser. These potent cytotoxic therapies result in acute tumor cell necrosis, while sparing surrounding healthy tissue and activating the immune system to generate an anti-tumor response. Aura’s lead product candidate belzupacap sarotalocan (AU-011) is currently in Phase 2 development for the treatment of choroidal melanoma, a vision and life-threatening form of eye cancer for which there are currently no approved therapies. In a Phase 1b/2 study, AU-011 demonstrated compelling efficacy, including high rates of tumor control and vision preservation, along with a favorable safety profile, in patients with choroidal melanoma. The development of AU-011 is partially supported by funding awarded under a CRADA from the U.S. National Institutes of Health’s National Cancer Institute. Future pipeline applications for Aura’s technology include choroidal metastases and primary bladder carcinoma. Aura is headquartered in Cambridge, MA. For more information, visit www.aurabiosciences.com or follow us on Twitter.

Contacts

Investors and Media:
Joseph Rayne
Argot Partners
617.340.6075 | joseph@argotpartners.com