On February 24, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing next-generation human enzyme therapeutics as solutions for diseases with high unmet medical need, today reported its fourth-quarter and full-year 2019 financial results, and provided recent corporate and program highlights (Press release, Aeglea BioTherapeutics, FEB 24, 2020, View Source [SID1234554631]).

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"Our accomplishments this past year illustrate how we are working to reimagine the potential of human enzymes as transformative solutions for challenging rare genetic disorders," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "In addition to delivering compelling Phase 1/2 data from our lead program for Arginase 1 Deficiency, we’ve made solid progress with our pivotal trial enrollment and started laying the groundwork for the commercial launch. We have also continued to make significant progress in our pipeline, further demonstrating the effectiveness of our platform in advancing programs in multiple therapeutic areas."

"We begin 2020 with a clear vision and heightened conviction of the impact for patients we can create with human enzyme therapeutics. We believe that we are well positioned to achieve a number of important milestones this year, armed with a strong leadership team with deep discovery, development and commercial expertise and driven by our collective goal to address the needs of the communities we serve," concluded Dr Quinn.

Recent Highlights

Pegzilarginase in Arginase 1 Deficiency

The Company expects to complete enrollment of its global, pivotal Phase 3 Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints (PEACE) trial in the third quarter of 2020 and to provide topline data in the first quarter of 2021.

Based on a recent genetic prevalence analysis of Arginase 1 Deficiency (ARG1-D), and with more than 200 patients already identified worldwide, the Company now estimates an addressable patient population of greater than 2,500, up from an estimate of 1,000 patients based solely on initial insights using newborn screening data.

The Company’s patient-identification strategy, informed by critical insights from disease analysis and trial experience, has already identified more than 100 patients in the United States, representing a 40% penetration into the genetic prevalent population.

ACN00177 in Homocystinuria

In January, Aeglea announced the filing of its Clinical Trial Application (CTA) with the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) for ACN00177, a novel engineered human enzyme therapy designed to treat homocystinuria, a serious metabolic disorder that results in elevated levels of plasma homocysteine.

The Company expects to initiate a Phase 1/2 trial for ACN00177 in the second quarter of 2020, with initial human proof of concept in the first quarter of 2021.

The Company estimates an addressable homocystinuria patient population of greater than 5,000.

Upcoming Events

Aeglea will be attending the following investor conferences in the coming quarter. Details of the presentations and webcasts will be announced prior to the events.

19th Annual Needham Healthcare Conference, April 14-15, New York City

H.C. Wainwright 2020 Global Life Sciences Conference, April 20-21, London, United Kingdom

Further, Aeglea leadership looks forward to participating in dialogue about our enzyme therapeutics platform during the following industry events, with additional details forthcoming.

Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), April 26-29, Austin, Texas

World Orphan Drug Congress USA 2020, April 29 to May 1, Oxon Hill, Maryland

Fourth Quarter and Full Year 2019 Financial Results

As of December 31, 2019, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $73.4 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.

Research and development expenses totaled $17.6 million for the fourth quarter of 2019 and $11.8 million for the fourth quarter of 2018. The increase was primarily associated with investing in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase; ramp-up in toxicology, investigational new drug (IND)-enabling studies, and manufacturing activities for ACN00177 in Homocystinuria; and personnel-related expenses.

Research and development expenses totaled $64.6 million for the year ended December 31, 2019, compared with $36.7 million for the year ended December 31, 2018. The increase was primarily due to investing in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase; a ramp-up in toxicology, IND-enabling studies, and manufacturing activities for ACN00177 in Homocystinuria; and expanded clinical development activity and personnel-related expenses.

General and administrative expenses totaled $4.3 million for the fourth quarter of 2019 and $3.5 million for the fourth quarter of 2018. This increase was primarily due to additional employee headcount, commercial readiness support, and facilities to support company growth.

General and administrative expenses totaled $15.7 million for the year ended December 31, 2019, compared with $12.6 million for the year ended December 31, 2018. This increase was primarily due to additional employee headcount, compensation, and facilities to support company growth.

Net loss totaled $21.5 million and $14.9 million for the fourth quarter of 2019 and 2018, respectively, with non-cash stock compensation expense of $1.2 million and $1.4 million for the fourth quarter of 2019 and 2018, respectively. Net loss totaled $78.3 million and $44.3 million for the years ended December 31, 2019 and 2018, respectively, with non-cash stock compensation expense of $4.9 million and $4.3 million for the years ended December 31, 2019 and 2018, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. Aeglea’s Phase 1/2 and Phase 2 open-label extension (OLE) data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company’s single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

About ACN00177 in Homocystinuria

Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities including severe osteoporosis, developmental delay, intellectual disability, lens dislocation and severe myopia. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of homocystinuria. The Company has submitted a Clinical Trial Application (CTA) with the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) and expects to initiate a Phase 1/2 trial in the second quarter of 2020.

On February 24, 2020 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported financial results for the fourth quarter and full year ended December 31, 2019 and provided an update on its recent corporate activities and outlook (Press release, Halozyme, FEB 24, 2020, View Source [SID1234554652]).

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"I am pleased to report 2020 is off to a strong start as we have substantially completed our actions to reposition the company to focus on ENHANZE," said Dr. Helen Torley, president and chief executive officer. "As a result of our decisive actions, we expect to become sustainably profitable in the second quarter. In addition, we anticipate multiple events that will grow the value of our ENHANZE technology in 2020. Both the subcutaneous formulation of Darzalex and the fixed-dose combination of Perjeta-Herceptin are under regulatory review in the U.S. and EU with potential approvals and launches expected this year. We anticipate three ENHANZE pipeline candidates to begin Phase 3 testing, one to begin Phase 2, and at least five new Phase 1 trial starts. With these key developments, a transition to profitability and plans for continued return of capital, Halozyme has never been in a stronger position to deliver value for our shareholders. With a target of 19 products in clinical development or already commercialized by the end of this year, we continue to see the potential for our royalty revenues to achieve $1 billion by 2027."

Fourth Quarter 2019 and Recent Highlights

In December, Janssen selected for development with ENHANZE the targets EGFR and cMET on an exclusive basis as part of the bispecific antibody (JNJ-61186372), which is being studied in solid tumors.
Halozyme recently completed $200 million worth of share repurchases under the Board of Directors authorized capital return program to repurchase up to $550 million of the Company’s outstanding common stock over the next three years. This repurchase plan was first authorized by the Board of Directors in November 2019 and has $350 million remaining under the authorization. The Company plans to repurchase up to an additional $150M worth of shares in 2020.
In November 2019, Halozyme completed the sale of $460.0 million aggregate principal amount of Convertible Senior Notes due 2024. A portion of the net proceeds from the offering were used to repurchase $200 million of the Company’s stock and $26.1 million to repay all outstanding amounts under loan agreements with Oxford Finance and Silicon Valley Bank.
In November 2019, Halozyme announced strategic actions to reposition the Company with a focus on its ENHANZE drug delivery technology and immediately implemented a restructuring following the announcement of results of the HALO-301 clinical study. Since then, the Company has completed the majority of the restructuring and remains on track to become a sustainably profitable company beginning in the second quarter of 2020.
In October 2019, collaboration partner Roche nominated one new undisclosed target to be studied utilizing the ENHANZE technology, triggering a $10 million milestone payment to Halozyme.
In October 2019, collaboration partner Bristol-Myers Squibb initiated a Phase 1 study of relatlimab in combination with nivolumab utilizing the ENHANZE technology.
Fourth Quarter and Full Year 2019 Financial Highlights

Revenue for the fourth quarter was $53.7 million compared to $60.2 million for the fourth quarter of 2018. The year-over-year decrease was primarily driven by a $25 million upfront payment from Roche in the year ago period 2018 related to the expansion of Roche’s collaboration with Halozyme, partially offset by higher API sales to our partners in the current period. Revenue for the quarter included $17.2 million in royalties, which compared to $19.3 million in the prior year period.

Total revenues for the full year were $196.0 million, compared with $151.9 million in 2018, representing growth of 29% year over year.
Research and development expenses for the fourth quarter were $45.1 million, compared to $36.7 million for the fourth quarter of 2018. The increase in expenses was due to $17.2 million in restructuring and one-time charges related to the shift in strategic focus to the Company’s ENHANZE drug delivery technology, partially offset by $9.4 million lower PEGPH20 clinical trial related costs.

Research and development expenses for the full year were $140.8 million, compared with $150.3 million in 2018.
Selling, general and administrative expenses for the fourth quarter were $23.9 million, compared to $18.0 million for the fourth quarter of 2018. The increase was due to restructuring and other one-time charges of $11.2 million, partially offset by lower personnel costs.

Selling, general and administrative expenses for the full year were $77.3 million, compared with $60.8 million in 2018.
Net loss for the fourth quarter was $34.4 million, or 0.24 per share, compared to a net loss in the fourth quarter of 2018 of $2.1 million, or $0.01 per share.

Net loss for the full year was $72.2 million or $0.50 per share, compared to $80.3 million or $0.56 per share in 2018.
Cash, cash equivalents and marketable securities were $421.3 million at December 31, 2019, compared to $354.5 million at December 31, 2018.
Financial Outlook for 2020

Halozyme is reiterating its 2020 financial guidance ranges as first announced on January 14, 2020. For 2020, Halozyme expects:

Revenues of $230 million to $245 million, representing growth of 17% to 25%;
Earnings per share on a GAAP basis of $0.60 to $0.75 with the first quarter of sustainable profitability beginning in Q2 2020.
The guidance for earnings per share does not reflect any potential impact from the Company’s plans to repurchase an additional number of shares, up to $150 million worth, during 2020. The amount and timing of shares repurchased during 2020 will be subject to a variety of factors including market conditions, other business considerations and applicable legal requirements.

Webcast and Conference Call

Halozyme will webcast its Quarterly Update Conference Call for the fourth quarter of 2019 today, Monday, February 24, 2020 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a replay will be available following the close of the call. To access the webcast and additional documents related to the call, please visit halozyme.com approximately fifteen minutes prior to the call to register, download and install any necessary audio software. The call may also be accessed by dialing (877) 824-0907 (domestic callers) or (647) 689-5655 (international callers). A telephone replay will be available after the call by dialing (800) 585-8367 (domestic callers) or (416) 621-4642 (international callers) using replay ID number 1266162.

On February 24, 2020 Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, reported its participation in the following upcoming investor conferences (Press release, Moderna Therapeutics, FEB 24, 2020, View Source [SID1234554668]):

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Cowen and Company 40th Annual Health Care Conference in Boston on Tuesday, March 3, 2020 at 11:20 a.m. ET.
Barclays Global Healthcare Conference in Miami on Wednesday, March 11, 2020 at 9:30 a.m. ET.
Oppenheimer 30th Annual Healthcare Conference in New York City on Tuesday, March 17, 2020 at 11:30 a.m. ET.
A live webcast of each presentation will be available under "Events and Presentations" in the Investors section of the Moderna website at View Source A replay of each webcast will be archived on Moderna’s website for 30 days following the presentation.

On February 24, 2020 Theravance Biopharma, Inc. ("Theravance Biopharma" or the "Company") (NASDAQ: TBPH) reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Theravance, FEB 24, 2020, View Source [SID1234554685]). Revenue for the fourth quarter and full year 2019 was $29.5 million and $73.4 million, respectively. Full-year operating loss was $251.9 million or $191.5 million excluding share-based compensation expense. Cash, cash equivalents and marketable securities totaled $285.8 million as of December 31, 2019.

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Rick E Winningham, Chairman and Chief Executive Officer, commented: "2019 was a year of achievement for Theravance Biopharma across our business. We successfully launched YUPELRI with Mylan and advanced our development- and research-stage pipeline, further building a diversified portfolio with promising, differentiated programs in every stage of development. Our roster of partnerships continued to strengthen, with ongoing successful collaborations with Mylan for YUPELRI and Janssen for TD-1473 and TD-5202. In addition, we entered into a new agreement with Pfizer to out-license our skin-selective pan-JAK inhibitor program. Our partnerships complement and expand our capabilities and execution and underscore our potential to transform the treatment of serious diseases."

"As we look ahead, 2020 will be an important year for our Company. We have established a strong capital position, augmented by our partnerships, as well as TRELEGY ELLIPTA royalties and YUPELRI commercialization. We are optimistic about future data readouts, especially our wholly owned programs — ampreloxetine in nOH and TD-8236 in asthma — which could both represent new treatment paradigms for patients with debilitating chronic diseases. The combined strengths of our research engine, pipeline, proven development expertise and commercial infrastructure have set the stage for a data- and catalyst-rich 2020 — a year that we believe can deliver meaningful value for stakeholders."

Corporate Highlights

Partnered with Mylan:
YUPELRI (revefenacin) inhalation solution (lung-selective nebulized long-acting muscarinic antagonist (LAMA)):

First and only once-daily, nebulized bronchodilator approved in the U.S. for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD)
One year post-launch — continued strong customer acceptance across key market metrics; combined Theravance Biopharma/Mylan sales infrastructures covering the hospital, hospital discharge, and home health settings
Data as of October 2019 show that YUPELRI achieved an 86% share of the nebulized LAMA market and a 10.7% share of the long-acting nebulized market (including Durable Medical Equipment)
Partnered with Janssen:
TD-5202 (gut-selective irreversible JAK3 inhibitor for inflammatory intestinal diseases):

TD-5202 was generally well tolerated as a single oral dose up to 2000 milligrams and as a twice-daily oral dose up to 2000 milligrams total per day given for 10 consecutive days in healthy subjects
TD-1473 (gut-selective oral pan-Janus kinase (JAK) inhibitor for inflammatory intestinal diseases):

Phase 2b/3 induction and maintenance study in ulcerative colitis (RHEA) and Phase 2 induction study in Crohn’s disease (DIONE) progressing
Data from the Phase 2b portion of the ulcerative colitis and Phase 2 Crohn’s disease studies planned for late-2020
Ampreloxetine (TD-9855, norepinephrine reuptake inhibitor (NRI) for symptomatic nOH):

Ongoing registrational program in symptomatic neurogenic orthostatic hypertension (nOH) comprised of two studies:
Phase 3 four-week treatment study (SEQUOIA) to demonstrate efficacy, with data expected in late 2020
Phase 3 four-month open label study followed by a six-week randomized withdrawal phase (REDWOOD) to demonstrate durability of response
TD-8236 (lung-selective inhaled pan-JAK inhibitor for inflammatory lung diseases):

Part C extension portion of the Phase 1 trial assessing additional biomarkers in more severe asthmatics underway with results expected in mid-2020
Phase 2 lung allergen challenge initiated in 4Q 2019; data expected in mid-2020
TRELEGY ELLIPTA (first once-daily single inhaler triple therapy for COPD)2:

4Q 2019 net sales of $221.5 million and full-year 2019 net sales of $661.3 million; Theravance Biopharma entitled to approximately 5.5% to 8.5% (tiered) of worldwide net sales of the product
Product now launched for COPD in 38 markets, including China
GSK filed sNDA 2Q 2019 for mortality benefit compared with ANORO in COPD and sNDA for use in patients with asthma in 3Q 2019
Notes:
1 Theravance Biopharma’s full-year operating loss, excluding share-based compensation expense, was below the 2019 guidance of $200 million to $210 million operating loss excluding share-based compensation due to the Pfizer upfront payment of $10 million being recognized as revenue in late December.
2 As reported by Glaxo Group Limited or one of its affiliates (GSK); reported sales converted to USD; economic interest related to TRELEGY ELLIPTA (the combination of fluticasone furoate, aclidinium, and vilanterol, (FF/UMEC/VI), jointly developed by GSK and Innoviva, Inc.) entitles Company to upward tiering payments equal to approximately 5.5% to 8.5% on worldwide net sales of the product (net of Theravance Respiratory Company, LLC ("TRC LLC") expenses paid and the amount of cash, if any, expected to be used in TRC over the next four fiscal quarters). 75% of the income from Company’s investment in TRC is pledged to service outstanding PhaRMASM notes, 25% of income from Company’s investment in TRC is retained by Company.

Fourth Quarter and Full Year Financial Results

Revenue: Revenue for the fourth quarter of 2019 was $29.5 million, comprised of collaboration revenue of $9.6 million primarily attributed to the upfront payment from Janssen for TD-1473, licensing revenue of $10.0 million related to the upfront payment from Pfizer for rights to our skin-selective pan-JAK inhibitor program, and revenue from the Mylan collaboration agreement of $9.9 million. Revenue for the fourth quarter represents a $13.8 million increase over the same period in 2018. The increase was primarily due to licensing revenue associated with the upfront payment from Pfizer and an increase in revenue from the Mylan collaboration agreement, partially offset by a decrease in product sales which resulted from the sale of VIBATIV to Cumberland Pharmaceuticals in late-2018. Full-year 2019 revenue was $73.4 million, comprised of collaboration revenue of $31.3 million primarily associated with our global collaboration with Janssen, licensing revenue of $28.5 million related to upfronts from Pfizer and Mylan and revenue from the Mylan collaboration of $13.7 million.

Research and Development Expenses: Research and Development (R&D) expenses for the fourth quarter of 2019 were $67.0 million, compared to $52.3 million in the same period in 2018. The increase was primarily due to an increase in employee-related costs and share-based compensation related to long-term retention and incentive awards, plus external-related costs associated with the progression of our key programs. Full-year 2019 R&D expenses were $219.2 million, or $190.3 million excluding non-cash share-based compensation.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses for the fourth quarter of 2019 were $33.0 million, compared to $25.5 million in the same period in 2018. The increase was primarily due to an increase in share-based compensation related to long-term retention and incentive awards. Full-year 2019 SG&A expenses were $106.1 million, or $74.6 million excluding non-cash share-based compensation.

Cash, Cash Equivalents and Marketable Securities Cash, cash equivalents and marketable securities totaled $285.8 million as of December 31, 2019.
2020 Financial Guidance

Operating Expenses: The Company expects full-year 2020 operating loss, excluding share-based compensation, of $205 million to $225 million. Operating loss guidance does not include:
Royalty income for TRELEGY ELLIPTA which the Company recognizes in its statement of operations as "income from investment in TRC, LLC;" or
Potential future business development collaborations
Note: timing and cost of clinical studies associated with key programs, among other factors, could impact financial guidance.

Additionally, as announced on February 11th, 2020, we closed our public offering of 5,500,000 ordinary shares at a price to the public of $27.00 per share. The gross proceeds to Theravance Biopharma from the offering are approximately $148.5 million, before deducting underwriting discounts and commissions and estimated offering expenses.

Conference Call and Live Webcast Today at 5:00 pm ET

Theravance Biopharma will hold a conference call and live webcast accompanied by slides today at 5:00 pm ET (2:00 pm PT / 10:00 pm GMT). To participate in the live call by telephone, please dial (855) 296-9648 from the US, or (920) 663-6266 for international callers, and use the confirmation code 5775588. Those interested in listening to the conference call live via the internet may do so by visiting Theravance Biopharma’s website at www.theravance.com, under the Investor Relations section, Presentations and Events.

A replay of the conference call will be available on Theravance Biopharma’s website for 30 days through March 25, 2020. An audio replay will also be available through 8:00 pm ET on March 2, 2020 by dialing (855) 859-2056 from the U.S., or (404) 537-3406 for international callers, and then entering confirmation code 5775588.

On February 24, 2020 Aura Biosciences, a clinical-stage biopharmaceutical company developing a new class of tumor targeted therapies for initial application in primary tumors such as ocular and bladder cancers, reported the presentation of updated clinical data from its ongoing Phase 1b/2 clinical trial evaluating the safety and efficacy of light-activated AU-011, the Company’s lead product candidate for the first line treatment of primary choroidal melanoma, at the 43rd Annual Macula Society Meeting, which took place February 19-22, 2020 in San Diego, CA (Press release, Aura Biosciences, FEB 24, 2020, View Source [SID1234554632]).

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"The Phase 1b/2 data being recognized at the Macular Society meeting this year show that AU-011 continues to be well tolerated, with a statistically significant reduction in tumor growth rate and vision preservation," said Amy C. Schefler, M.D., Weill Cornell Medical College and Retina Consultants of Houston.

"The high tumor control rates observed to date, together with the high rate of vision preservation, including in patients at high risk for vision loss with sub-macular and peripapillary tumors continue to support AU-011’s significant potential to change the treatment paradigm for patients battling choroidal melanoma," said Cadmus Rich, MD, Chief Medical Officer and Head of Research and Development of Aura Biosciences. "I am proud of the work the company has done to make a significant progress in this ultra-rare disease."

Updated Results from the Phase 1b/2 Study Evaluating AU-011

Dr. Schefler gave an oral presentation titled, "Study Update of an Ongoing Phase 1b/2 Open-label Clinical Trial of AU-011 for the Treatment of Small to Medium Choroidal Melanoma," which highlighted updated data from the ongoing open-label Phase 1b/2 clinical trial, and described the design of the planned Phase 3 trial that Aura expects to initiate during the second half of 2020.

The updated data demonstrate that multiple administrations of light-activated AU-011 were well-tolerated. Among the patients evaluated for safety (n=52), the most common treatment-related adverse events (AEs) were expected and included anterior chamber inflammation, posterior chamber inflammation and increase in intraocular pressure; all were manageable with standard-of-care treatments and the majority 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. There were two treatment-related serious AEs (vision loss; 3.6%). Tumor control and vision preservation data continue to be supportive of the planned Phase 3 registration trial.

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 Light-Activated AU-011

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.