AZ’s selumetinib for rare disease gets FDA breakthrough status

FDA | April 02, 2019

AstraZeneca and Merck & Co/MSD’s selumetinib has been given a breakthrough designation from the US FDA for a rare paediatric disease, after failing to make the grade in other cancers. The MEK 1/2 inhibitor has picked up the breakthrough status for neurofibromatosis type 1 (NF1), an incurable genetic condition that affects one in 3,000-4,000 newborns worldwide and for which it also received an orphan drug designation from the EMA last year. NF1 can cause lumps under the skin, pigmentation and in around 25% of cases benign tumours on nerve sheaths called plexiform neurofibromas that can cause pain, motor dysfunction and disfigurement. Some patients also experience other complications such as learning difficulties, visual impairment, twisting and curvature of the spine, high blood pressure, and epilepsy. There are no approved therapies for the disease. The new status marks something of a renaissance for selumetinib, which has previously failed as a monotherapy in various solid tumours including thyroid cancer, lung cancer, and uveal melanoma.

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A Nutanix enterprise cloud reduces the cost and complexity of IT infrastructure for healthcare while improving scaling, availability, and data protection. Conventional infrastructure is no longer able to address healthcare challenges, such as regulatory hurdles, resource constraints, highly mobile workers, and rapid application proliferation.

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PHARMA TECH

Apollomics Inc., a Late-Stage Clinical Biopharmaceutical Company to be Listed on Nasdaq Through Business Combination

Apollomics, Inc. | September 15, 2022

Apollomics Inc. a late-stage clinical biopharmaceutical company, and Maxpro Capital Acquisition Corp. announced a definitive agreement for a business combination that would result in Apollomics becoming a publicly traded company on the Nasdaq Global Market. The business combination is expected to close in the first quarter of 2023 and Apollomics is expected to be listed on Nasdaq under the ticker symbol “APLM.” Apollomics’ broad pipeline of drug candidates includes late-stage clinical assets for the treatment of patients with difficult-to-treat cancers. Apollomics’ mission is to develop assets in critically important areas of unmet need. The Company’s leading drug candidates address certain subpopulations within lung cancer and leukemia. Globally, both lung cancer and leukemia affect over 2 million people annually. The Company is dedicated to discovering and developing oncology therapies of different mechanisms of action to inhibit cancer. Its diverse portfolio of innovative drug candidates for treating difficult-to-treat cancers includes precision therapy targeting tumors with specific mutations, as well as assets addressing broader cancer conditions. The Company’s pipeline of nine clinical, preclinical and discovery drug candidates has the potential to improve treatment of a number of tumor types. Upon the closing of the transaction, Apollomics will continue to be led by current Chairman and CEO, Dr. Guo-Liang Yu, Ph.D., a serial entrepreneur, and his team. Dr. Yu is a pharmaceutical researcher with more than 300 patents. “Apollomics’ announcement represents the next major milestone on our journey to provide solutions for patients with difficult-to-treat cancers,” Dr. Yu said. “We anticipate that the funds available to us from this transaction will help us accelerate development of our oncology pipeline.” Apollomics expects results from its global Phase 2 multi-cohort clinical trial of vebreltinib in NSCLC and other solid tumors with cMET dysregulation in 2023, which the Company believes may support its first New Drug Application (“NDA”) with the U.S. Food and Drug Administration (“FDA”) while generating clinical data for different indications. In addition, the Company expects to complete patient recruitment of its uproleselan Phase 3 study in China in 2023. “Our goal was to find an exciting company with a growing pipeline of innovative product candidates that could positively affect the lives of millions of people. Our team is excited to combine with Apollomics as it has met and exceeded all our key selection criteria. Together with Apollomics, Maxpro will do everything we can to support the Company’s vision of treating patients with difficult-to-treat cancers.” Moses Chen, CEO of Maxpro About Apollomics Inc. Apollomics Inc. is an innovative clinical-stage biopharmaceutical company focused on the discovery and development of oncology therapies with the potential to be combined with other treatment options to harness the immune system and target specific molecular pathways to inhibit cancer. Apollomics currently has a pipeline of nine drug candidates across multiple programs, six of which are currently in the clinical stage of development. Apollomics’ lead programs include investigating its core product, vebreltinib (APL-101), a potent, selective c-Met inhibitor for the treatment of non-small cell lung cancer and other advanced tumors with c-Met alterations, which is currently in a Phase 2 multicohort clinical trial in the United States, and developing an anti-cancer enhancer drug candidate, uproleselan (APL-106), a specific E-Selectin antagonist that has the potential to be used adjunctively with standard chemotherapy to treat acute myeloid leukemia and other hematologic cancers, which is currently in Phase 1 and Phase 3 clinical trials in China. About Maxpro Capital Acquisition Corp. Maxpro is a blank check company formed for the purposes of effecting a merger, capital share exchange, asset acquisition, share purchase, reorganization, or similar business combination with one or more businesses in the healthcare and technology industries. In October 2021, Maxpro consummated a $103.5 million initial public offering of 10.35 million units (including the underwriters’ full exercise of their over-allotment option), each unit consists of one share of Class A common stock and one redeemable warrant, each warrant entitles the holder to purchase one share of Class A common stock at a price of $11.50 per share. EF Hutton, division of Benchmark Investments LLC, served as the sole book-running manager of Maxpro’s initial public offering.

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BUSINESS INSIGHTS

Glaukos Announces More Than 1 Million iStent® Technologies Implanted Worldwide

Glaukos | September 12, 2022

Glaukos Corporation an ophthalmic medical technology and pharmaceutical company focused on novel therapies for the treatment of glaucoma, corneal disorders and retinal diseases announced a significant milestone as its iStent® family of technologies have now been implanted in more than one million procedures worldwide. This market-leading milestone is a testament to the decades of investment from Glaukos as the corporate founder and pioneer of the now well-established micro-invasive glaucoma surgery (MIGS) marketplace. “One million iStents implanted is a tremendous accomplishment and I’m grateful to Glaukos for their continued dedication, investment and pioneering spirit to advance the development of innovative glaucoma technologies designed to improve the treatment paradigm for the benefit of patients worldwide,” said Ike Ahmed MD, Professor, Department of Ophthalmology and Visual Sciences at John A. Moran Eye Center in Utah, United States and Chief Innovation Officer at Prism Eye Institute in Toronto, Canada. “As one of the early adopters, I have experienced first-hand how iStent, as the first MIGS device, and subsequent generations have fundamentally revolutionized the way we think and treat glaucoma over the last decade. Today, the disease state is more manageable, and we are intervening earlier with micro-invasive surgical options. It’s been an incredible journey thus far and I’m excited to see what the future holds for our glaucoma patients.” “iStent technologies have allowed me to change the way I treat my patients for the better. Before the introduction of iStent and MIGS, we were limited to eye drops, which we know to be problematic for patients, or invasive surgery that carried more risk than needed for mild-to-moderate glaucoma. iStent filled that gap between eye drops and invasive surgery. In my practice today, iStent inject W is my most commonly used surgical option for glaucoma patients undergoing cataract surgery. We now have patient data out past 9 years in my practice showing the iStent is still effective and safe at lowering IOP and reducing medications. I’m eagerly anticipating the exciting future innovations from Glaukos.” John Berdahl, MD, Vance Thompson Vision, South Dakota “I am proud of this tremendous achievement for our company and the MIGS marketplace, reflecting decades of investment and successful advancement of our strategic vision to transform the treatment of chronic, debilitating eye diseases through development of novel, sustainable therapies,” said Thomas Burns, Glaukos chairman and chief executive officer. “I would like to recognize our customers and employees around the world who have poured countless hours into making this market-leading achievement a reality and am grateful for their continued support going forward as we strive to create transformative platform technologies designed to improve the standard-of-care and meet unmet patient needs.” Glaukos is proud to be the corporate pioneer and global market leader in MIGS, with its family of iStent technologies supported by more than 200 peer-reviewed publications, 20 plus years of clinical and commercial experience and more than one million iStent devices implanted worldwide since its inception. The company believes it offers the industry’s most comprehensive offering of minimally-invasive, tissue-sparing glaucoma solutions, supporting its goal to provide a full range of options to fit surgeons’ individual glaucoma treatment algorithms that offer the most favorable short- and long-term benefit-to-risk calculus at every stage of disease progression, from ocular hypertension through refractory disease, and in both combo-cataract and standalone procedures. Glaukos remains dedicated to innovation and bringing customers around the world best-in-class technologies to serve their patients, continuing to invest upwards of 30% of revenue back into R&D, including new product development. A significant number of clinical studies for both current and future products are ongoing, with additional studies commencing in the near future. About Glaukos Glaukos is an ophthalmic medical technology and pharmaceutical company focused on developing and commercializing novel therapies for the treatment of glaucoma, corneal disorders and retinal diseases. Glaukos first developed Micro-Invasive Glaucoma Surgery as an alternative to the traditional glaucoma treatment paradigm, launching its first MIGS device commercially in 2012, and continues to develop a portfolio of technologically distinct and leverageable platforms to support ongoing pharmaceutical and medical device innovations. Products or product candidates for each of these platforms are designed to advance the standard of care through better treatment options across the areas of glaucoma, corneal disorders and retinal diseases. About iStent inject W Trabecular Micro-Bypass System (U.S.) Indication for Use: The iStent inject W Trabecular Micro-Bypass System Model G2-W is indicated for use in conjunction with cataract surgery for the reduction of intraocular pressure (IOP) in adult patients with mild to moderate primary open-angle glaucoma.

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BUSINESS INSIGHTS

Atomwise Signs Strategic Multi-Target Research Collaboration with Sanofi for AI-Powered Drug Discovery

Atomwise | August 22, 2022

Atomwise, a leader in using artificial intelligence (AI) for small molecule drug discovery, announced that it has established a strategic and exclusive research collaboration with Sanofi that will leverage its AtomNet® platform for computational discovery and research of up to five drug targets. The Atomwise approach shifts the mode of drug discovery away from serendipitous discovery and toward search based on structure, making the drug discovery process more rational, effective, and efficient. The AtomNet platform incorporates deep learning for structure-based drug design, enabling the rapid, AI-powered search of Atomwise’s proprietary library of more than 3 trillion synthesizable compounds. “At Sanofi, we are committed to bringing higher quality medicines to patients faster, empowered by our advanced AI drug discovery engine. We are excited to partner with Atomwise, given their leadership in the field of virtual screening and AI-based molecular design. Together, we aim at making the drug discovery process more efficient and effective in particular when very limited information is available to support drug design.” Frank Nestle, Global Head of Research and Chief Scientific Officer at Sanofi As part of this agreement, Sanofi will pay Atomwise $20 million upfront to identify, synthesize, and advance lead compounds for up to five targets which will be exclusive to Sanofi. Subsequent payments pegged to key research, development, and sales milestones could total more than $1 billion. In addition, tiered royalties have been established for products developed through the collaboration. “At Atomwise, our mission is to use our unique technology to make better medicines, faster, by unlocking targets that have been inaccessible to traditional small molecule discovery approaches,” said Abraham Heifets, co-founder and CEO of Atomwise. “We are pleased to enter into this collaboration with Sanofi, which serves as continued validation of the important role that AI-powered platforms will play in accelerating the discovery of new therapies for diseases and conditions that may have gone untreated due to challenging or uncharacterized drug targets.” About Atomwise Atomwise is a technology-enabled pharmaceutical company leveraging the power of AI to revolutionize small molecule drug discovery. The Atomwise team invented the use of deep learning for structure-based drug design; this technology underpins Atomwise’s best-in-class AI discovery engine, which is differentiated by its ability to find and optimize novel chemical matter. Atomwise has extensively validated its discovery engine, having demonstrated the ability to find compounds with therapeutic potential for biological targets in 90% of internal programs and more than 70% of the company’s 270 academic collaborations, including across a wide variety of protein types and multiple “hard to drug” targets. Atomwise is building a wholly-owned pipeline of small-molecule drug candidates, with three programs in lead-optimization and over 30 programs in discovery. The company has raised over $194 million from leading venture capital firms and collaborations to advance its mission to make better medicines, faster.

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