On January 4, 2019 Quanterix Corporation (NASDAQ:QTRX), a company digitizing biomarker analysis with the goal of advancing the science of precision health, is marking a little over one year since becoming a public company with significant milestones on its path toward revolutionizing healthcare (Press release, Quanterix, JAN 4, 2019, View Source [SID1234532470]). Quanterix’ Chief Executive Officer, President and Chairman will highlight these achievements and future plans during a presentation at the upcoming 37th Annual J.P. Morgan Healthcare Conference in San Francisco, CA, on Wednesday, Jan. 9, 2019 at 2 p.m., PST.

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Since going public and listing on the Nasdaq on Dec. 7, 2017 – in one of the most successful IPOs of its kind in recent years – Quanterix has continued to achieve accelerated double-digit, year-over-year revenue growth in each full quarter since the IPO. This has been driven by major investments in the company’s flagship Simoa technology, product expansion, acquisition, publications, global commercialization and accelerating market adoption of digital biomarkers for research.

"The past year has been rewarding for us as a company and we are showing no signs of slowing down," said Kevin Hrusovsky, Chief Executive Officer, President and Chairman of Quanterix. "We are committed to delivering on our promise of advancing the science of precision health and driving today’s healthcare revolution forward. Today we are empowering new advances in disease diagnosis and monitoring, and helping drug makers develop better drugs, and tomorrow we aspire to detect and prevent diseases before they even take hold. We’re already making a tremendous impact in neurology research and are now seeing incredible potential in immunoncology and immunotherapy monitoring as well, where the ability to determine the efficacy and toxicity of drugs earlier could save significant healthcare costs and save lives. We’re excited to return to J.P. Morgan to offer greater insight around our vision, 50% year-to-date growth through Q3’18, Q4’18 expectations and progress toward our greater mission of helping battle cancer and neurodegenerative diseases."

Significant 2018 Achievements:

Launched the SR-X Ultra-Sensitive Biomarker Detection System in January 2018, less than one month after public offering, offering researchers lower-cost and smaller footprint access to Simoa technology.
Acquired Aushon BioSystems and successfully integrated the two companies. The acquisition included a CLIA certified laboratory, expanding services and accelerating entry into pharmaceutical drug trial services, as well as access to novel immunoassay technology to which Simoa sensitivity algorithms are being applied to deliver next-generation Simoa capabilities.
Initiated a collaboration with DestiNA Genomics in an effort to transform microRNA biomarker detection, bringing together high-specificity and ultra-sensitivity for the first time.
Gained unrestricted rights back for its Simoa technology in IVD markets with the termination of a license agreement with bioMérieux.
Increased new publications dramatically by 145, bringing the total to 360 (nearly 50% growth).
Rapid adoption of proprietary Neurofilament light chain (NfL) assay, a biomarker previously mainly measured only in cerebral spinal fluid, but by using Simoa, researchers have shown it can be measured in blood and correlated to central spinal fluid (CSF) results. This advance has enabled many novel applications critical for advancing the early detection, treatment and prevention of neurological diseases, including multiple sclerosis (MS); Parkinson’s disease; Alzheimer’s disease; brain cancer; and traumatic brain injuries (TBIs).
Invited by the FDA to present recent progress and potential future strategy for NfL to senior agency officials.
Announced partnerships with Abbott Labs for blood screening and Oncogenesis for a cervical cancer screening test.
Launched a test bed/early access program for a new next-generation 10-plex Simoa planar array for oncology research and drug development with a benchtop imager called the SP-X Imaging and Analysis System.
Increased headcount from 149 to 181 (>20%) and expanded the leadership team with the hiring of Jackson Streeter, M.D., to lead corporate development and strategy, formerly Chief Medical Officer and Chief Executive Officer of Banyan Biomarkers and Dawn Mattoon, Ph.D., to lead strategic market and assay technology, formerly Head of Product Development at a leading antibody provider.
Ranked number 299 on Deloitte’s Technology Fast 500 and received several noteworthy recognitions for Quanterix’ Chief Executive Officer’s visionary leadership for healthcare disruption.
Last month was added to the Nasdaq Biotechnology Index (NBI) as part of the annual re-ranking and attracted additional analyst coverage.
Was lead sponsor for Powering Precision Health (PPH) annual summit held for the first time in Amsterdam, now an independent, nonprofit entity.
"We are proud of these achievements as they help demonstrate the power of our technology and the disruptive innovation Quanterix is leading in the field of precision health," continued Hrusovsky. "We feel there is still enormous opportunity in front of us as we facilitate industry collaboration, drive research advancement and fuel growth as we pursue minimally invasive digital biomarker disruption in oncology, neurodegeneration, immunology, infectious disease and inflammation.

Webcast Information
To access the live webcast of Quanterix’ presentation at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, Jan. 9, 2019 at 2 p.m., PST, please visit the News & Events page within the Investors section of the Quanterix website at www.quanterix.com. Replays of the webcast will be available for a limited period following the conference.

On January 4, 2019 Synaffix B.V. reported it has entered into a license agreement with Mersana Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody drug conjugates (ADCs) targeting cancers in areas of high unmet need (Press release, Synaffix, JAN 4, 2019, View Source [SID1234532772]). This agreement provides Mersana access to Synaffix’s industry-leading site-specific GlycoConnect ADC technology that has consistently demonstrated an ability to improve both the safety and the efficacy profiles of ADC product candidates. Synaffix is eligible to receive upfront and milestone payments on a per-target basis with a projected total deal value of $295 million, plus royalties­­.

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Under the terms of the agreement, Mersana has been granted a non-exclusive license to incorporate GlycoConnect into one of its ADC development candidates, as well as an option to expand to additional programs. This agreement follows a research collaboration between the two companies that was centered around multiple product candidates.

"After evaluating several site-specific conjugation platforms, we have chosen Synaffix’s GlycoConnect technology for use in future ADC candidates," said Anna Protopapas, President and Chief Executive Officer of Mersana. "We are excited about the potential of this technology as it is designed not to require additional antibody engineering or cell-line modifications, which would offer us the potential to create site-specific antibodies for use in our ADCs when required."

"We are particularly excited to enter into this agreement with a leading company in the field of ADCs such as Mersana," said Peter van de Sande, CEO of Synaffix, who added "This collaboration is another testimony of the additional value that GlycoConnect is able to provide to already cutting-edge ADC technologies, thereby enabling novel medicines that are uniquely positioned to address areas of unmet medical need."

Per the agreement, Mersana is responsible for the research, development, manufacturing and commercialization of any resulting ADC product while Synaffix will supply components that are specifically related to its proprietary GlycoConnect technology.

About GlycoConnect

The proprietary GlycoConnect technology of Synaffix is a platform ADC technology that utilizes proprietary enzymes and metal-free click conjugation to stably attach ADC payloads specifically to the native glycan of any antibody, a privileged site for ADC applications. This approach can be applied directly to an existing antibody without any DNA and or protein engineering and is compatible with all ADC payload classes. The growing experience of Synaffix and its collaboration partners continues to confirm the ability of GlycoConnect to consistently generate ADCs that are more effective and better tolerated when compared to the three major clinical-stage ADC conjugation technologies.

On January 4, 2019 Recursion, a clinical-stage biotechnology company combining artificial intelligence (AI), experimental biology, and automation to discover and develop drugs at scale, reported it has entered into a licensing agreement with the Ohio State Innovation Foundation (OSIF), the technology transfer function of The Ohio State University, gaining rights to OSU-HDAC42, a clinical-stage compound that will be developed by Recursion as REC-2282 (Press release, Recursion Pharmaceuticals, JAN 4, 2019, View Source [SID1234556193]). Recursion plans to develop the compound in neurofibromatosis type 2 (NF2), a devastating rare tumor syndrome.

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"We leveraged our unique discovery platform to identify potential therapies for NF2 from among known compounds with the potential for accelerated development," said Chris Gibson, Ph.D., co-founder and CEO of Recursion. "We found a strong signal in our data for REC-2282 as a potential treatment for NF2, and upon further diligence, discovered the drug was already being pursued in the treatment of this disease. The universe of experimental treatments is vast, but the data arbitrage generated empirically on our platform gave us the confidence to move forward quickly."

Under the terms of this agreement, Recursion obtains exclusive worldwide rights to develop and commercialize REC-2282. Full financial terms have not been disclosed.

"Adding REC-2282 to our clinical pipeline and driving to a rapid determination of its efficacy and safety for NF2 patients marks another important step in Recursion’s growth and more importantly may offer a better treatment option for this terribly underserved patient population," said Tim Considine, Senior Vice President, Strategic Development at Recursion. "We are excited to build on existing clinical data to advance this program to human proof of concept in NF2 as rapidly as possible."

Last year, Recursion announced that its Investigational New Drug (IND) application for another disease of unmet need, cerebral cavernous malformation (CCM), was cleared by the Food and Drug Administration (FDA), and that program is currently enrolling subjects in Phase 1.

About Neurofibromatosis Type 2

Neurofibromatosis type 2 is a genetic condition most commonly associated with bilateral vestibular schwannomas, also known as acoustic neuromas. These are benign (noncancerous) tumors that occur on the nerves responsible for balance and hearing in the inner ear. Patients can also have meningiomas, a slow-growing tumor that usually develops on the surface of the brain. Although these tumors are benign, they can cause loss of hearing and balance problems, and in severe cases can be life threatening. It is estimated that about one in 40,000 people has NF2. Approximately 50% of people with NF2 do not have a family history of the condition. Current treatment involves surgical removal of the tumors, which provides temporary relief of symptoms but bears a significant risk of hearing loss and other complications.

About REC-2282 (OSU-HDAC42)

REC-2282 is a pan-histone deacetylase (HDAC) inhibitor. There are multiple lines of evidence that REC-2282 exhibits both histone-independent and acetylation-independent mechanisms, at both epigenetic and cellular levels. The compound was previously in clinical development by Arno Therapeutics (as AR-42) for various solid and liquid tumors. Exploratory investigator-initiated studies have been conducted in patients with vestibular schwannomas and meningiomas. Rights to the compound were returned to OSIF in November 2017.

On January 4, 2019 Aura Biosciences, a leader in the development of novel targeted therapies in ocular oncology, reported that it has received written confirmation from the U.S. Food and Drug Administration (FDA) regarding agreement on the design of its Phase 3 registration trials designed to evaluate light-activated AU-011 for the treatment of patients with choroidal melanoma (Press release, Aura Biosciences, JAN 4, 2019, View Source [SID1234532454]). This written confirmation is the result of successful outcome of an "End of Phase 2" meeting with the FDA.

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The STARBRIGHT registration program will consist of two small identical clinical trials designed to assess the safety and efficacy of AU-011 versus sham control for the treatment of patients with small choroidal melanoma and high risk indeterminate lesions. Both trials, which will be titled STARBRIGHT1 and STARBRIGHT3, will be global, multicenter, randomized and masked, and will be conducted in parallel. The primary endpoint will be comprised of a combination of tumor control and vision preservation.

In addition to the design of the Phase 3 trials, the FDA agreed with Aura’s proposed safety database. The FDA also agreed that no further non-clinical studies are needed.

"We are pleased to have received such clear guidance from the FDA with respect to the Phase 3 STARBRIGHT program to be able to meet the scientific and regulatory requirements for marketing approval in the U.S." said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.

The currently available treatments for choroidal melanoma come with the risk of severe vision loss, especially for patients with melanomas located close to the fovea or optic disk. The ongoing Phase 1b/2 study with light-activated AU-011 has shown that the drug was well-tolerated, with clear evidence of tumor control and preservation of visual acuity at long term follow up.

"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," said Cadmus Rich, M.D., Chief Medical Officer of Aura. "Overall, the meeting removed any remaining uncertainty on the regulatory path to approval and highlighted FDA’s commitment to guide Aura toward a potential first drug approved for patients with this highly unmet medical need."

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.

On January 4, 2019 Sangamo Therapeutics, Inc. (NASDAQ: SGMO) reported that Sandy Macrae, CEO of Sangamo, will present a corporate overview at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9th at 11:30 a.m. PT in San Francisco (Press release, Sangamo Therapeutics, JAN 4, 2019, View Source [SID1234532471]). Dr. Macrae’s presentation will be followed by a separate Q&A session starting at 12:00 p.m. PT.

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The presentation and Q&A session will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will be archived on the Sangamo website for two weeks after the event.