Ora, Inc. | September 29, 2022
Ora, Inc., the world's leading ophthalmology clinical research firm, has formally launched a revolutionary mobile research platform, Ora EyeCupTM, which promises to transform patient data capture through high-resolution imaging, artificial intelligence (AI) analysis, and real-time patient feedback. The Ora EyeCupTM, a finalist in the Most Valuable Clinical Technology Award category at the Reuters Events Pharma Awards USA 2022, will be presented by Dr. Gustavo De Moraes, Chief Medical Officer at Ora, at this year’s Eyecelerator Conference in Chicago, IL, September 29th.
The Ora EyeCupTM platform is a powerful combination of hardware and software — a smartphone attachment and software application which allows patients to take high-resolution images of their eyes and track disease signs and symptoms remotely. With over 138,000 images captured to date, 98% are considered gradable by researchers and AI. Post-capture processing utilizes AI-powered image analysis for precise clinical assessments of ocular redness, tear film stability, and corneal health. Along with this, the Ora EyeCupTM ensures protocol adherence through compliance assurance diaries for both symptom tracking and therapy dosing.
“At Ora, our technologists have spent the past two years developing this revolutionary system to capture patient data and provide a deeper understanding of therapeutic effect. The result is a trailblazing technology that captures high quality therapeutic data and improves the likelihood of study success.”
Dr. De Moraes
About Ora, Inc.
Ora is the world's leading full-service ophthalmic drug and device development firm with offices in the United States, United Kingdom, Australia, and Asia. For over 40 years, we have proudly helped our clients earn more than 55 product approvals and create vision beyond what they see. We support a wide array of organizations, from start-ups to global pharmaceutical and device companies, to efficiently bring their new products from concept to market. Ora's pre-clinical and clinical models, unique methodologies, and global regulatory strategies have been refined and proven across thousands of global projects. We bring together the world's most extensive and experienced team of ophthalmic experts, R&D professionals, and operations management to maximize the value of new product initiatives. Think ophthalmology, think Ora.
Alnylam Pharmaceuticals, Inc. | October 07, 2022
Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration approved a label expansion for OXLUMO® an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate and plasma oxalate levels in pediatric and adult patients. The approval is based on positive efficacy and safety results of the ILLUMINATE-C Phase 3 study of OXLUMO in patients with severe renal impairment, including those on hemodialysis.
PH1 is an ultra-rare genetic disease characterized by oxalate overproduction in the liver. The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones and nephrocalcinosis, which can progress to kidney failure. PH1 can also lead to oxalate deposition in multiple organs beyond the kidney, a condition known as systemic oxalosis.
The label expansion for OXLUMO is based on positive six-month results from ILLUMINATE-C, where treatment with lumasiran resulted in a substantial reduction in POx from baseline to Month Six in both dialysis-independent and -dependent patients. Patients in both cohorts had a reduction in POx as early as Month One. In Cohort A treatment with OXLUMO led to a 33% least squares (LS) mean reduction in POx from baseline to Month Sixand in Cohort B, treatment with OXLUMO led to a 42% LS mean reduction in POx from baseline to Month Six. OXLUMO demonstrated an encouraging safety and tolerability profile with no deaths or drug-related serious adverse events among enrolled patients. There were two treatment discontinuations due to adverse events in the extension period of the study, neither of which was drug related. The most common AE related to lumasiran was injection-site reaction reported in five of 21 patients which were all mild and transient.
“The significance of the label expansion of OXLUMO cannot be overstated, as this milestone provides crucial reassurance among a patient population with the highest unmet need, as well as their caregivers and loved ones, that OXLUMO is available in the US for patients living with PH1, including those with advanced disease. We are grateful to Alnylam for their continued commitment to the PH1 community and for ensuring that all patients — no matter how severe their disease — have the same opportunity to potentially benefit from OXLUMO treatment.”
Kim Hollander, Executive Director of the Oxalosis and Hyperoxaluria Foundation
In November of 2020, OXLUMO was approved by the FDA for the treatment of PH1 to lower UOx levels in pediatric and adult patients. It was also approved by the European Medicines Agency (EMA) for the treatment of PH1 in all age groups. The Committee for Medicinal Products for Human Use (CHMP) of the EMA delivered a positive opinion recommending variation to the marketing authorization of OXLUMO based on ILLUMINATE-C data from patients with advanced PH1 in September 2022. Lumasiran is also being evaluated in a global Phase 2 study as an investigational treatment in patients with recurrent kidney stone disease and elevated UOx levels.
OXLUMO® is an RNAi therapeutic targeting hydroxyacid oxidase 1. HAO1 encodes glycolate oxidase. Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. OXLUMO has received regulatory approvals from the U.S. Food and Drug Administration for the treatment of primary hyperoxaluria type 1 to lower urinary and plasma oxalate levels in pediatric and adult patients and from the European Medicines Agency for the treatment of PH1 in all age groups.
In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. In the ILLUMINATE-C study, OXLUMO resulted in substantial reductions in plasma oxalate in patients with advanced PH1. Across all three studies, injection site reactions were the most common drug-related adverse reaction.
OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly beginning one month after the last loading dose at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly.
About Alnylam Pharmaceuticals
Alnylam has led the translation of RNA interference into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® GIVLAARI® OXLUMO® AMVUTTRA® and Leqvio® being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.
Sumitovant Biopharma Ltd. | October 03, 2022
Sumitovant Biopharma Ltd. in conjunction with parent company Sumitomo Pharma Co., Ltd. announced it has submitted a non-binding proposal to the Audit Committee of the Board of Directors of Myovant Sciences Ltd. offering to acquire all outstanding shares of Myovant not already owned by Sumitovant for $22.75 per share in cash. The proposed transaction, if completed, would result in Myovant being delisted from the New York Stock Exchange.
The proposed acquisition consideration represents an equity value for Myovant of $2.4 billion and an enterprise value of $2.5 billion. The proposed per share consideration represents a premium of approximately 27% to Myovant's closing share price on September 30, 2022, and a premium of approximately 31% to the 60-day volume weighted average price of Myovant's shares through September 30, 2022.
The Proposal is a natural step in the well-established relationship between Sumitovant and Myovant, and represents an unprecedented opportunity to combine expertise, platforms, and resources to deliver innovative therapies addressing unmet patient needs in women's health and prostate cancer. Sumitovant and Sumitomo Pharma have been impressed with the culture and significant accomplishments of Myovant's employees. If a transaction is completed, we will continue to provide support and resources to Myovant to enable achievement of its mission to redefine care for the patients they so passionately serve.
Since the investment in Myovant in December 2019, both Sumitovant and Sumitomo Pharma have enjoyed a collaborative and successful relationship with Myovant that has facilitated the continued development and commercialization of Orgovyx® and Myfembree® in prostate cancer and women's health in and outside of the U.S. Further, Sumitovant and Sumitomo Pharma believe that the Proposal is financially compelling and will create significant and immediate value for Myovant's shareholders.
The Proposal provides that the proposed transaction will be subject to the approval of the shareholders of Myovant holding a majority of the shares not owned by Sumitovant.
In addition, Sumitovant and Sumitomo Pharma informed the Audit Committee in the Proposal that, in Sumitovant's capacity as a majority shareholder in Myovant, Sumitovant and Sumitomo Pharma are interested only in acquiring the shares of Myovant not already owned by Sumitovant and that in such capacity, Sumitovant has no interest in selling any of the Myovant shares it owns, nor would Sumitovant support any alternative sale, merger, or similar transaction involving Myovant.
The Proposal does not create any binding legal obligation between Sumitovant or Sumitomo Pharma and Myovant. Neither Sumitovant, Sumitomo Pharma nor Myovant will have any obligation or liability to the other with respect to a transaction unless and until definitive transaction documents are executed and delivered by the parties.
About Sumitovant Biopharma Ltd.
Sumitovant is a technology-driven biopharmaceutical company accelerating development and commercialization of new potential therapies for patients with rare conditions and other diseases. Through our proprietary computing and data platforms, scientific expertise and diverse company portfolio, Sumitovant has supported development of multiple FDA-approved products and a robust pipeline of early- through late-stage investigational assets addressing unmet patient needs in pediatrics, urology, oncology, women's health, specialty respiratory and infectious diseases. Sumitovant is a wholly owned subsidiary of Sumitomo Pharma.
About Sumitomo Pharma Co., Ltd.
Sumitomo Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China, and other Asian countries with about 7,000 employees worldwide. Sumitomo Pharma defines its corporate mission as "To broadly contribute to society through value creation based on innovative research and development activities for the betterment of healthcare and fuller lives of people worldwide."