On October 29, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported topline results from the Phase III MAIA study (MMY3008) of daratumumab in combination with lenalidomide and dexamethasone (DRd) versus Rd alone as treatment for newly diagnosed patients who are not candidates for high dose chemotherapy and autologous stem cell transplant (ASCT) (Press release, Genmab, OCT 29, 2018, View Source [SID1234530362]). The study met the primary endpoint of improving progression free survival (PFS) at a pre-planned interim analysis (Hazard Ratio (HR) = 0.55 (95% CI 0.43 – 0.72), p < 0.0001) resulting in a 45% reduction in the risk of progression or death in patients treated with DRd. The median PFS for patients treated with daratumumab in combination with Rd has not been reached, compared to an estimated median PFS of 31.9 months for patients who received Rd alone.

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Overall, the safety profile of daratumumab in combination with Rd is consistent with both the known safety profiles of the Rd regimen and daratumumab.

Based on the results at the pre-planned interim analysis conducted by an Independent Data Monitoring Committee (IDMC), it was recommended releasing the interim analysis results. Further analysis of the safety and efficacy data is ongoing and Janssen Biotech, Inc., which licensed daratumumab from Genmab in 2012, will discuss the potential for a regulatory submission for this indication with health authorities, and plans to submit the data to an upcoming medical conference and for publication in a peer-reviewed journal.

"We are highly encouraged by this data as this is the fifth randomized study showing a profound benefit when adding daratumumab to standard of care treatments in multiple myeloma, and the second showing efficacy for patients with newly diagnosed multiple myeloma who are not eligible for ASCT. As such this data increases our hope that daratumumab may one day help even more patients at the outset of treatment of this disease," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Today’s news does not impact Genmab’s 2018 financial guidance.

About the MAIA (MMY3008) study
The Phase III study (NCT02252172) is a randomized, open-label, multicenter study that includes 737 newly diagnosed patients with multiple myeloma who are not candidates for high dose chemotherapy and ASCT. Patients were randomized to receive either daratumumab in combination with lenalidomide (an immunomodulatory drug) and dexamethasone (a corticosteroid) or lenalidomide and dexamethasone alone. In the daratumumab treatment arm, patients received 16 milligrams per kilogram (mg/kg) weekly for first 8 weeks (Cycles 1 and 2), every other week for 16 weeks (Cycles 3 to 6) and then every 4 weeks (Cycle 7 and beyond) until progression of disease or unacceptable toxicity. Lenalidomide is administered at 25 mg orally on days 1 through 21 of each 28-day cycle, and dexamethasone was administered at 40 mg once a week for both treatment arms. Participants in both treatment arms will continue Rd until disease progression or unacceptable toxicity. The primary endpoint of the study is PFS.

About multiple myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lymphoma.2 Approximately 30,770 new patients are expected to be diagnosed with multiple myeloma and approximately 12,770 people are expected to die from the disease in the U.S. in 2018.3 Globally, it was estimated that 124,225 people would be diagnosed and 87,084 would die from the disease in 2015.4 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5

About DARZALEX (daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.6 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for treatment of adults with relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and frontline multiple myeloma settings and in amyloidosis. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases, such as NKT-cell lymphoma, myelodysplastic syndromes, B and T-ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.

On October 29, 2018 Kazia Therapeutics Limited (ASX: KZA;NASDAQ: KZIA), an Australian oncology-focused biotechnology company, is reported to shareholders regarding progress with GDC-0084, which is currently in human trials for glioblastoma and several other forms of brain cancer (Press release, Kazia Therapeutics, OCT 29, 2018, View Source [SID1234530440]).

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Key Points

Phase II clinical trial of GDC-0084 in glioblastoma is progressing well, with all sites open to recruitment and the first cohort of patients fully enrolled and undergoing treatment
Investigator-initiated clinical collaborations launched with St Jude Children’s Research Hospital in diffuse intrinsic pontine glioma (DIPG), and with Dana-Farber Cancer Institute in breast cancer brain metastases (BCBM)
Poster on the Kazia glioblastoma study to be presented at upcoming Society for Neuro-Oncology meeting in New Orleans, LA on 16 November 2018
Manufacture of an additional batch of GDC-0084 capsules for clinical trial use has commenced
Kazia CEO, Dr James Garner, commented, "We are delighted with progress across the GDC-0084 program. Our own study in glioblastoma is off to an excellent start, and we are very pleased to now also be working with two top-tier research hospitals to explore additional uses of the drug in other forms of brain cancer. The hard work that has been undertaken over the past twelve to eighteen months is now paying off, which sets the company up for several important and value-driving data read-outs from the GDC-0084 program during calendar 2019."

He added, "The PI3K inhibitor class has seen some dramatic developments in the past six months. We were excited to see FDA approve Copiktra (duvelisib) from Verastem in October 2018, bringing the number of FDA-approved PI3K inhibitors to three. The recent European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) meeting also saw promising data presented from Novartis for their PI3K inhibitor, alpelisib, in certain forms of breast cancer. It is clear that this class of drugs is well-established and well-proven. However, GDC-0084 remains the only PI3K inhibitor in mainstream development that is able to cross the blood-brain barrier, and this gives it a unique advantage in brain cancer."

Phase II Clinical Trial in Glioblastoma

All seven participating centres are fully open to recruitment. To date, the first cohort of three patients has been enrolled and are currently receiving treatment. If the first cohort is able to complete treatment without experiencing significant toxicity, a second cohort will be enrolled at a higher dose. A number of potential patients have already been identified for the second cohort and are undergoing pre-screening. Once the maximum tolerated dose (MTD) has been established in this first part of the trial, the remainder of the study will proceed at that dose.

Recruitment to date has exceeded expectations, and the study remains on track to report initial data early in calendar 2019. It is listed on clinicaltrials.gov as NCT03522298. The study is also listed in the clinical trial finder section of the US National Brain Tumor Society website.

Investigator-Initiated Collaborations

As previously announced, Kazia is supporting two leading US hospitals to explore the potential use of GDC-0084 in other forms of brain cancer.

St Jude Children’s Research Hospital in Memphis, TN has commenced a phase I human trial of GDC-0084 in children with diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas. This study is currently recruiting and is listed on clinicaltrials.gov as NCT03696355.

Dana-Farber Cancer Institute (DFCI) in Boston, MA is establishing a phase II human trial of GDC-0084 in women with breast cancer brain metastases (BCBM), which is breast cancer that has spread to the brain. This study is expected to commence recruitment in early calendar 2019, pending approval by the Institutional Review Board at DFCI, and is not yet listed on clinicaltrials.gov.

Kazia has been pleased to observe very strong interest from clinicians and scientists in a range of other potential exploratory studies of GDC-0084 and remains in discussion regarding several other potential clinical collaborations.

Publications

Kazia is gratified to have been accepted for a ‘trials in progress’ poster presentation at the upcoming 23rd Annual Scientific Meeting of the Society for Neuro-Oncology (SNO), which will be held in New Orleans, LA on 16-18 November 2018.

The Company will make the poster presentation available to all shareholders at approximately the same time it is presented at conference. The poster is expected to focus primarily on the design of the company-sponsored ongoing phase II study in glioblastoma, and the rationale for GDC-0084 in this group of patients, and it is not anticipated that meaningful clinical data will be available at this early stage.

Other presentations and publications of GDC-0084 data are anticipated in the next six to twelve months, and the Kazia team will be discussing appropriate opportunities with investigators at the upcoming SNO meeting.

Manufacturing

Work has begun to manufacture a second batch of capsules for use across the GDC-0084 clinical program, under international Good Manufacturing Practice (GMP) conditions. The company obtained approximately 48kg of drug substance as part of its transaction with Genentech in October 2016, and this remains highly stable. Kazia has planned to periodically formulate a portion of this material into capsules for clinical trial use, according to progress with the studies. Given the pace of recruitment to date, it has been considered appropriate to accelerate manufacture of a second batch of capsules to ensure continuity of supply. Production of a second batch will also strengthen the data available regarding GDC-0084 manufacture for regulatory purposes, and will ultimately help to inform commercial supply.

Regulatory Affairs

In accordance with FDA requirements, Kazia has been undertaking a 13-week toxicology study of GDC-0084 in two animal species. This is a routine requirement to support long-term use of the drug in human patients. The study is progressing well according to plans, and is on track to conclude before the end of calendar 2018.

Kazia has applied to the World Health Organisation (WHO) for an International Non-proprietary Name (INN) for GDC-0084. It is common for drugs to be referred to by a code number during early development, but companies typically seek allocation of an INN around the initiation of phase II development. INNs are a necessary step for eventual regulatory approval and are determined centrally by WHO. Kazia expects to receive the INN for GDC-0084 in late calendar 2019.

Intellectual Property

Since assuming responsibility for GDC-0084 from Genentech in October 2016, Kazia has continued to pursue robust protection for the intellectual property associated with the drug. Of note, patents have been granted in Australia (July 2017), the People’s Republic of China (March 2018), Hong Kong SAR (March 2017), and the United States (January 2017), as well as in a number of other jurisdictions. In general, the patents relating to GDC-0084 provide comprehensive protection until at least 2031

On October 29, 2018 Principia Biopharma Inc. (Nasdaq: PRNB), a clinical-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, reported the appointment of industry veteran Dolca Thomas, M.D. as its Chief Medical Officer (Press release, Principia Biopharma, OCT 29, 2018, View Source [SID1234530295]). Dr. Thomas joins Principia from Roche, where she was Vice President and Global Head of Translational Medicine for Immunology, Inflammation, and Infectious Disease. She brings approximately 15 years of industry and medical experience to Principia. Steve Gourlay, MBBS, FRACP, Ph.D. will remain with the company as a senior medical advisor through mid-year 2019.

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"We are very excited with the addition of Dolca to the Principia team, particularly as we pursue an expanding scope of clinical development activities including initiating our pivotal Phase 3 trial for PRN1008 in patients with pemphigus. Dolca is a seasoned pharma executive with broad and successful experience in executing late stage programs, specifically in immunology," said Martin Babler, Chief Executive Officer of Principia. "I also want to thank Steve for his leadership, dedication and contributions to Principia over the past five years. Steve led the clinical development of three different molecules, including taking PRN1008 from its first-in-human trial through design and preparation for Phase 3. We are pleased that he will continue to support the company as a senior medical advisor through the first half of next year."

"I am impressed with Principia’s approach to BTK inhibition in autoimmune disease and believe that application of the company’s proprietary Tailored Covalency platform may be applicable across a wide range of autoimmune and inflammatory conditions," said Dr. Thomas. "I have been involved in more than a dozen immunology product candidates, and I look forward to contributing to the potential success of Principia’s pipeline assets and moving the company towards late-stage clinical development."

Dr. Thomas brings 15 years of industry and medical experience with strategic and operational responsibility for clinical development, pharmacovigilance, and safety and medical affairs of approximately two dozen pharmaceutical products. Most recently, Dr. Thomas was Vice President and Global Head of Translational Medicine for Immunology, Inflammation, and Infectious Disease at Roche, where she was responsible for advancing multiple product candidates through clinical development. Prior to Roche, Dr. Thomas held roles of increasing responsibility at Pfizer, including Vice President of Clinical Development and Clinical Immunophenotyping, and Vice President and Chief Development Officer of the Biosimilars Research and Development Unit where she was responsible for all stages of development of multiple assets. Dr. Thomas began her industry career at Bristol-Myers Squibb as Director of Global Clinical Development in Immunology, where she was involved in the development and approval of belatacept.

Dr. Thomas has a B.A. in sociology from Cornell University, and received her M.D. degree from Cornell University. Dr. Thomas completed her residency in internal medicine, in addition to her post-doctoral training in nephrology and transplantation, at New York- Presbyterian Hospital, Weill Cornell Medical Center.

Dr. Thomas represented both Bristol-Myers Squibb and Pfizer on the Board of Directors of the Progressive Multifocal Leukoencephalopathy (PML) Consortium, a cross-industry consortium aimed at identifying more effective methods of predicting, preventing, and developing future treatments for PML. Dr. Thomas has many past collaborations with the Juvenile Diabetes Research Foundation (JDRF) where she has been the recipient of multiple JDRF research grants and awards. In addition, she served on Board of Directors for the NYC JDRF chapter.

On October 29, 2018 Novartis reported it was awarded the 2018 Prix Galien USA Award for Best Biotechnology Product for Kymriah (tisagenlecleucel) following its first-in-class approval by the US Food and Drug Administration (FDA) for the treatment of children and young adults with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL) (Press release, Novartis, OCT 29, 2018, View Source [SID1234530441]). Kymriah is a ground-breaking one-time treatment that uses a patient’s own T cells to fight cancer, and the only chimeric antigen receptor T cell (CAR-T) therapy approved for two distinct B-cell malignancies1. The award, which recognizes excellence in scientific innovation that improves the state of human health, was presented at a ceremony in New York City.

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"It is a great honor to receive this prestigious award for Kymriah," said Liz Barrett, CEO, Novartis Oncology. "The impact for patients with aggressive blood cancers who previously had limited treatment options is our greatest reward and drives us to continue pioneering efforts to reimagine the way cancer is treated."

The historic approval of Kymriah was a result of collaboration with researchers at University of Pennsylvania (Penn) who demonstrated the first successful use of gene transfer therapy to use the body’s immune cells to fight cancer. Novartis further researched this emerging class of drug, initiating the first global CAR-T trials and in August 2017, became the first company to gain a regulatory approval of a CAR-T cell therapy. This year, Kymriah has also been approved in the United States for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL); and for the treatment of pediatric and young adult patients with r/r ALL and adults with r/r DLBCL in the European Union, Canada and Switzerland. Of note, Kymriah is not approved for the treatment of patients with primary central nervous system lymphoma1.

Both B-cell ALL and DLBCL are aggressive malignancies, and for patients who relapse or don’t respond to therapy, there are limited treatment options2,3.

Novartis Prix Galien Awards
Considered "the pharmaceutical industry’s Nobel Prize," the Prix Galien recognizes excellence in scientific innovation that improves the state of human health, and acknowledges the technical, scientific and clinical research skills necessary to develop such innovative medicines. As a true testament to the company’s commitment to reimagine medicine, since 1970, Novartis has received more than 40 national Prix Galien awards in 15 countries for innovative therapies including Gleevec (imatinib mesylate), Kymriah (tisagenlecleucel), Parlodel (bromocriptine mesylate), Rimactane (rifampin), Sandimmune (cyclosporine), Sandostatin (octreotide acetate), Simulect (basiliximab) and Visudyne (verteporfin)4.

About Kymriah (tisagenlecleucel)
Kymriah is an innovative immunocellular therapy, manufactured individually for each patient by reprogramming the patient’s own immune system cells. Kymriah is the only approved CAR-T cell therapy manufactured using the 4-1BB costimulatory domain, which is critical for full activation of the therapy, enhancement of cellular expansion and durable persistence of the cancer-fighting cells. In 2012, Novartis and Penn entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.

Kymriah (tisagenlecleucel) US Important Safety information
Kymriah may cause side effects that are severe or life-threatening, such as Cytokine Release Syndrome (CRS) or Neurological Toxicities. Patients with CRS may experience symptoms including difficulty breathing, fever (100.4◦F/38◦C or higher), chills/shaking chills, severe nausea, vomiting and diarrhea, severe muscle or joint pain, very low blood pressure, or dizziness/lightheadedness. Patients may be admitted to the hospital for CRS and treated with other medications.

Patients with neurological toxicities may experience symptoms such as altered or decreased consciousness, headaches, delirium, confusion, agitation, anxiety, seizures, difficulty speaking and understanding, or loss of balance. Patients should be advised to call their healthcare provider or get emergency help right away if they experience any of these signs and symptoms of CRS or neurological toxicities.

Because of the risk of CRS and neurological toxicities, Kymriah is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called Kymriah REMS.

Serious allergic reactions, including anaphylaxis, may occur after Kymriah infusion.

Kymriah can increase the risk of life-threatening infections that may lead to death. Patients should be advised to tell their healthcare provider right away if they develop fever, chills, or any signs or symptoms of an infection.

Patients may experience prolonged low blood cell counts (cytopenia), where one or more types of blood cells (red blood cells, white blood cells, or platelets) are decreased. The patient’s healthcare provider will do blood tests to check all of their blood cell counts after treatment with Kymriah. Patients should be advised to tell their healthcare provider right away if they get a fever, are feeling tired, or have bruising or bleeding.

Patients may experience hypogammaglobulinemia, a condition in which the level of immunoglobulins (antibodies) in the blood is low and the risk of infection is increased. It is expected that patients may develop hypogammaglobulinemia with Kymriah, and may need to receive immunoglobulin replacement for an indefinite amount of time following treatment with Kymriah. Patients should tell their healthcare provider about their treatment with Kymriah before receiving a live virus vaccine.

After treatment with Kymriah, patients will be monitored lifelong by their healthcare provider, as they may develop secondary cancers or recurrence of their cancer.

Patients should not drive, operate heavy machinery, or do other dangerous activities for eight weeks after receiving Kymriah because the treatment can cause temporary memory and coordination problems, including sleepiness, confusion, weakness, dizziness, and seizures.

Some of the most common side effects of Kymriah are difficulty breathing, fever (100.4°F/38°C or higher), chills/shaking chills, confusion, severe nausea, vomiting and diarrhea, severe muscle or joint pain, very low blood pressure, dizziness/lightheadedness, and headache. However, these are not all of the possible side effects of Kymriah. Patients should talk to their healthcare provider for medical advice about side effects.

Prior to a female patient starting treatment with Kymriah, their healthcare provider may do a pregnancy test. There is no information available for Kymriah use in pregnant or breast-feeding women. Therefore, Kymriah is not recommended for women who are pregnant or breast feeding. Patients should talk to their healthcare provider about birth control and pregnancy.

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

After receiving Kymriah, patients should be advised that some commercial HIV tests may cause a false-positive test result. Patients should also be advised not to donate blood, organs, or tissues and cells for transplantation after receiving Kymriah.

On October 29, 2018 Aura Biosciences, a leader in the development of novel targeted therapies in ocular oncology, reported that updated clinical data from its Phase 1b/2 clinical trial evaluating the safety and efficacy of light-activated AU-011, the Company’s lead product candidate for the primary treatment of choroidal melanoma, was highlighted in an oral presentation at the American Academy of Ophthalmology (AAO) 2018 Annual Meeting being held October 27-30, 2018, at McCormick Place in Chicago (Press release, Aura Biosciences, OCT 29, 2018, View Source [SID1234530245]).

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Brian P. Marr, M.D. , Director, Division of Ophthalmic Oncology, New York-Presbyterian/Columbia University Medical Center, and principal investigator of the study, gave the oral presentation titled, "One-Year Results of a Phase 1b/2 Open-Label Clinical Trial of AU-011 for the Treatment of Primary Choroidal Melanoma." This open-label, multicenter trial is designed to investigate single and multiple ascending doses of light-activated AU-011 in approximately 36 adult subjects with clinically diagnosed primary choroidal melanoma.

The data presented at the meeting show that multiple administrations of light-activated AU-011 are well-tolerated with no related serious adverse events, severe adverse events or dose-limiting toxicities observed. Drug related adverse events were all expected and included anterior chamber inflammation, posterior chamber inflammation and increase in intraocular pressure, but all were manageable with standard-of-care treatments and resolved without clinical sequelae. Notably, the posterior inflammation appears to originate within and/or around the tumor which is consistent with AU-011’s mechanism of action of acute tumor necrosis.

Treatment with light-activated AU-011 achieved preservation of best corrected visual acuity (BCVA) with a mean change of -1.06 letters at 6 months and a mean change of -0.75 letters at 12 months. BCVA was preserved even in high risk patients with tumors close to the fovea or the optic disk, a factor that typically correlates with a higher risk of irreversible severe vision loss following radioactive treatments. Importantly, all patients (100%) achieved stable disease at the prespecified preliminary efficacy endpoint at 3 months. Biological activity has been confirmed with long term tumor control in those patients with documented growth before treatment, reduction in tumor thickness and localized inflammation around the tumor. An expansion cohort of the study is currently underway. The Company plans to initiate a pivotal Phase 3 clinical program following the Phase 1b/2 study.

"The currently available treatments for choroidal melanoma come with the risk of severe vision loss, especially for patients with melanomas that are located close to the fovea or optic disk," commented Dr. Marr. "These 12-month data demonstrate that light-activated AU-011 continues to be well-tolerated, including with multiple administrations, has evidence of tumor control and preservation of visual acuity. We look forward to executing on the expansion phase of the study and generating more clinical data for this innovative targeted therapy."

"To date, light-activated AU-011 has shown a compelling degree of tolerability and vision preservation, especially given the alternative radioactive treatment options for choroidal melanoma," said Cadmus Rich, M.D., Chief Medical Officer of Aura. "We believe that a minimally invasive, non-radiation-based treatment option that enables early intervention while preserving vision has the potential to transform the therapeutic landscape for this difficult to treat, often deadly form of melanoma. We are actively preparing the Phase 3 study designs and look forward to initiating this pivotal program."

About Choroidal Melanoma

Choroidal 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 on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes to the liver in about 40-50 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-011

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. 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.