Astellas Pharma Inc | August 19, 2022
Astellas Pharma Inc. announced that the U.S. Food and Drug Administration has accepted the company's New Drug 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
The PDUFA target action date is February 22, 2023, following use of a priority review voucher (PRV). Astellas booked ¥13.1 billion of amortization of the intangible asset relating to PRV as R&D expense in the first quarter of fiscal year 2022.
"The FDA's acceptance of our NDA for fezolinetant brings us one step closer to advancing care for women in the U.S. who experience VMS. We look forward to the FDA's review of our application, and the potential to offer a first-in-class nonhormonal treatment option to reduce the frequency and severity of moderate to severe VMS associated with menopause."
Ahsan Arozullah, M.D., M.P.H., Senior Vice President and Head of Development Therapeutic Areas, Astellas
The NDA 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 the U.S., Canada and Europe. 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 NDA, Astellas proposes a 45 mg daily dose, which is subject to the FDA's review.
Fezolinetant is an investigational nonhormonal selective neurokinin 3 (NK3) receptor antagonist. The safety and efficacy of fezolinetant are under investigation and have not been established.
The impact of this acceptance on Astellas' financial results of the current fiscal year ending March 31, 2023, is expected to be minor.
About the BRIGHT SKY™ Phase 3 Program
The BRIGHT SKY pivotal trials, SKYLIGHT 1™ (NCT04003155) and SKYLIGHT 2™ (NCT04003142), 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 In the U.S., about 60% to 80% of women experience these symptoms during or after the menopausal transition and, worldwide, more than half of women 40 to 64 years of age experience VMS.3,4,5,6 VMS can have a disruptive impact on women's daily activities and overall quality of life.
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.
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.
Eyenovia | September 06, 2022
Eyenovia, Inc. an ophthalmic pharmaceutical technology company developing a pipeline of microdose array print therapeutics, brings human-centric design to ophthalmology with its novel Optejet® dispenser.
Traditional eye drops normally overdose the patient's eye with medication and preservatives. Eyenovia's easy-to-use, novel Optejet® dispenser administers precisely 8µL of medication to the eye, effectively treating the patient's condition while creating a more gentle dosing experience.
"The unique delivery mechanism of the Optejet® will fundamentally change how we think about eye medication. Right now, traditional eye drops can result in significant dose-related side effects for the treatment of chronic illnesses like glaucoma. The Optejet® dispenser is designed for patients to experience easier administration and fewer side effects with similar efficacy."
Michael Rowe, CEO of Eyenovia
The Optejet® dispenser administers ophthalmic solutions horizontally using Eyenovia's proprietary Microdose Array Print (MAP™) technology. The system functions similar to an inkjet printer, delivering accurate microdoses through a precision spray. Traditional eye drops deliver four to five times larger volume than the human eye can actually hold, which causes eye drops to roll down the face and overdose the eye with preservatives.
Poor delivery of eye drops is more than an inconvenience; it can prevent patients from using necessary medication because of the side effects. For example, with medication to help dilate the pupils for eye exams, up to 23% of patients experienced stinging and burning with the eye drops, causing patients to forgo eye exams. In Eyenovia studies, less than 1% of patients experienced stinging and burning.
"Medications only work if patients take them, and with the Optejet, we have designed a way for patients to benefit from an easy-to-use, appropriate, lower dose spray that results in similar efficacy. We're excited for physicians and providers to be able to offer this revolutionary technology in the near future to patients who deserve a more positive experience," said Rowe.
Eyenovia's first marketed invention is expected to be MydCombi™, a first-in-class drug-device combination microdose for in-office pupil dilation. This potentially will help save physician's chair time, eliminating the traditional three eyedrops used for a patient's eye exam. Other exciting innovations in the Eyenovia pipeline include MicroLine and MicroPine.
About Eyenovia, Inc.
Eyenovia, Inc. is an ophthalmic pharmaceutical technology company developing a pipeline of microdose array print therapeutics. Eyenovia is currently focused on the late-stage development of microdosed medications for mydriasis, presbyopia, and myopia progression.
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.