PHARMA TECH

Ranok Therapeutics Announces Agreement with Pfizer on Targeted Protein Degradation

Ranok Therapeutics | November 30, 2021

Ranok Therapeutics Announces Agreement with Pfizer on Targeted Protein Degradation
Ranok Therapeutics Co. Ltd., an emerging biopharmaceutical company focused on developing breakthrough therapies for cancer and other serious diseases, announced an agreement with Pfizer Inc. to apply and evaluate Ranok’s CHAMP™ platform technology on an undisclosed cancer target. Terms of the agreement were not disclosed.

Ranok has developed a proprietary and innovative targeted protein degradation (TPD) platform technology, CHAMP™ (Chaperone-mediated Protein Degradation), which leverages Ranok’s founders’ expertise in protein homeostasis to take advantage of the cellular chaperone network, potentially improving drug safety and efficacy due to selective targeting of disease tissues.

We are very pleased to work with Pfizer to explore the application of CHAMP™ to an emerging cancer drug target, This represents our first pharmaceutical company research relationship and is an important step forward in establishing CHAMP™ as a preferred technology in the rapidly-growing field of targeted protein degradation. We look forward to uncovering new benefits of CHAMP™ through this research.”


Kevin P. Foley, Ph.D., Co-founder and Chief Scientific Officer of Ranok Therapeutics

This evaluation agreement builds upon Ranok’s recent momentum, including its $40 million Series B financing in August 2021.

About Ranok Therapeutics
Ranok is a privately held, emerging biopharmaceutical company that is pioneering its proprietary CHAMP™ platform technology, an innovative approach to targeted protein degradation (TPD) for the discovery and development of novel therapeutics. Ranok was founded by a leadership team composed of industry veterans from both Chinese and American biotech and pharmaceutical companies and is based in two of the world’s leading centers of innovation and medical research: Hangzhou China and Greater Boston, Massachusetts. By bringing together cutting-edge medical research from both China and the United States, Ranok’s goal is to create transformative medicines to benefit patients suffering from cancer and other serious diseases around the world. For more information, please visit the company website at www.ranoktherapeutics.com or follow us at LinkedIn and on Twitter @RanokTx.

About Ranok’s CHAMP™ platform
Chaperone-mediated Protein Degradation/Degrader  technology is based on our founders’ extensive backgrounds in protein homeostasis research. This technology takes advantage of the cellular chaperone network, distinguishing it from other targeted protein degradation (TPD) approaches. CHAMP™ has a number of unique advantages, including improved safety due to the selective targeting of tumors. Our proprietary R&D engine combines deep biological insight and chemistry expertise to iteratively discover and test new leads, rapidly identifying and advancing optimal development candidates for a variety of important disease targets.

Spotlight

The specialty pharmacy industry could reduce costs by $27.5 million annually (product and call center staff costs only) if visual temperature indicators were used as decision-making tools to determine if medicine should be used or returned when patients suspect heat damage. In addition, 97% of patients surveyed, who received medicine from specialty pharmacies with a visual temperature indicator included in the package, agree that they would want an indicator in all shipments and 95% agree the indicator increased their confidence in the medicines received.

Spotlight

The specialty pharmacy industry could reduce costs by $27.5 million annually (product and call center staff costs only) if visual temperature indicators were used as decision-making tools to determine if medicine should be used or returned when patients suspect heat damage. In addition, 97% of patients surveyed, who received medicine from specialty pharmacies with a visual temperature indicator included in the package, agree that they would want an indicator in all shipments and 95% agree the indicator increased their confidence in the medicines received.

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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

WuXi STA Opens New Large-scale Oligonucleotide and Peptide Manufacturing Facility

WuXi STA | July 07, 2022

WuXi STA, a subsidiary of WuXi AppTec, announced the opening of a new large-scale oligonucleotide and peptide manufacturing facility at its Changzhou campus. The new facility underscores WuXi STA's ongoing commitment to enhance its capacity and capability to meet the fast-growing customer needs for oligonucleotide and peptide therapeutics development and manufacturing worldwide, making more innovative therapies accessible to the patients. With the opening of the new 30,570 sq.ft. oligonucleotide plant, WuXi STA extends its leadership in oligonucleotide development and manufacturing worldwide, with four large-scale oligonucleotide production lines and more than 20 small- to mid-scale production lines that increase the overall manufacturing capacity of a single synthesis run from 1.9 mol to 6.0 mol. The new 22,260 sq.ft. peptide plant features three new production lines with reactors that can accommodate up to 1,000 L, increasing WuXi STA's overall Solid Phase Peptide Synthesis total reactor volume to 6,490 L. The new facility is an essential part of WuXi STA's comprehensive end-to-end Contract Research, Development, and Manufacturing Organization platform for new modalities. The platform supports oligonucleotides and peptides, including novel monomers and linkers, and complex conjugates, from early discovery to development and commercial production at any scale. With this enhanced manufacturing capacity and over 850 dedicated scientists, WuXi STA's new modality CRDMO platform can better enable global partners as their projects progress to late and commercial stages. In addition, WuXi STA offers injectable formulation development and manufacturing services, including Lipid NanoParticle technology as well as comprehensive analytical and Chemical, Manufacturing, and Controls dossier preparation services, which further accelerate the development of new oligonucleotide and peptide therapeutics for the market. "We are pleased to support our global partners in their efforts to develop new modality therapies for patients in need. WuXi STA is committed to enabling partners by enhancing our new modality CRDMO platform's capacity and capability to expedite the development and commercialization of more innovative therapies to benefit patients worldwide." Dr. Minzhang Chen, Co-CEO of WuXi AppTec and CEO of WuXi STA WuXi STA has a global network of multiple R&D and manufacturing sites across Asia, North America and Europe, including a new pharmaceutical manufacturing campus in Middletown, Delaware that is scheduled to open in 2024. About WuXi STA WuXi STA, a subsidiary of WuXi AppTec, is a leading pharmaceutical development and manufacturing capability and technology platform company serving the life sciences industry with operations across Asia, North America and Europe. As a premier Contract Research, Development, and Manufacturing Organization, WuXi STA offers our worldwide partners efficient, flexible, and high-quality solutions for integrated CMC solutions from preclinical to commercial uses.

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PHARMA TECH

European Medicines Agency Accepts Astellas' Marketing Authorization Application for Fezolinetant

Astellas Pharma Inc. | September 30, 2022

Astellas Pharma Inc. announced the European Medicines Agency has accepted for regulatory review the company's marketing authorization application for fezolinetant, an investigational oral, nonhormonal compound seeking approval for the treatment of moderate to severe vasomotor symptoms associated with menopause. VMS, characterized by hot flashes and/or night sweats, are common symptoms of menopause.1,2 "More than half of women 40 to 64 years of age experience hot flashes during menopause, with limited nonhormonal treatment options. The EMA's acceptance of our MAA for fezolinetant brings us one step closer to advancing a potential first-in-class nonhormonal treatment option for women in Europe who experience VMS associated with menopause, similar to the FDA acceptance of our NDA for women in the U.S." Ahsan Arozullah, M.D., M.P.H., Senior Vice President and Head of Development Therapeutic Areas, Astellas The MAA is supported by results from the BRIGHT SKY™ program, which included three Phase 3 clinical trials that collectively enrolled over 2,800 women with VMS across Europe, the U.S. and Canada. Results from the SKYLIGHT 1™ and SKYLIGHT 2™ pivotal trials characterize the efficacy and safety of fezolinetant for the treatment of moderate to severe VMS associated with menopause. Data from the SKYLIGHT 4™ safety study further characterizes the long-term safety profile of fezolinetant. Within the MAA, Astellas proposes a 45 mg daily dose, which is subject to the EMA's review. Fezolinetant is an investigational nonhormonal selective neurokinin 3 receptor antagonist. The safety and efficacy of fezolinetant are under investigation and have not been established. This acceptance will have no impact on Astellas' financial forecasts of the current fiscal year ending March 31, 2023. About the BRIGHT SKY™ Phase 3 Program The BRIGHT SKY pivotal trials, SKYLIGHT 1™ and SKYLIGHT 2™ enrolled over 1,000 women with moderate to severe VMS. The trials are double-blinded, placebo-controlled for the first 12 weeks followed by a 40-week treatment extension period. Women were enrolled at over 180 sites within the U.S., Canada and Europe. SKYLIGHT 4™ (NCT04003389) is a 52-week double-blinded, placebo-controlled study designed to investigate the long-term safety of fezolinetant. For SKYLIGHT 4, over 1,800 women with VMS were enrolled at over 180 sites within the U.S., Canada and Europe. About VMS Associated with Menopause VMS, characterized by hot flashes and/or night sweats, are common symptoms of menopause.1,2 Worldwide, more than half of women 40 to 64 years of age experience VMS with rates in Europe ranging from 56% to 97%.3,4,5 The prevalence of moderate to severe VMS in postmenopausal women in Europe has been reported at 40%.6 VMS can have a disruptive impact on women's daily activities and overall quality of life.1 About Fezolinetant Fezolinetant is an investigational oral, nonhormonal therapy in clinical development for the treatment of moderate to severe VMS associated with menopause. Fezolinetant works by blocking neurokinin B (NKB) binding on the kisspeptin/neurokinin/dynorphin (KNDy) neuron to moderate neuronal activity in the thermoregulatory center of the brain (the hypothalamus) to reduce the frequency and severity of moderate to severe VMS associated with menopause.7,8,9 The safety and efficacy of fezolinetant are under investigation and have not been established. There is no guarantee the agent will receive regulatory approval or become commercially available for the uses being investigated. About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients.

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