Medicares new CAR-T coverage rules could boost adoption of slow-growing meds

FiercePharma | August 08, 2019

The national Medicare coverage rule for CAR-T cell treatments is finally here. By changing some requirements in the previous draft version, the federal government lifts some restrictions and burdens on healthcare providers and could in turn boost adoption. In a national coverage decision, Medicare will pay for CAR-T therapies, the Centers for Medicare and Medicaid Services said Wednesday. For Gilead Sciences and Novartis, which market FDA-approved CAR-T treatments Yescarta and Kymriah, respectively, the policy could help improve the drugs lackluster sales now that doctors will have the peace of mind that they can get reimbursement for these costly therapies. “Today’s decision makes it very clear that ‘Yes, this is covered.’ We’re paying that not only for CAR-T, but all the related services. The administration of the drug. The collection of the cells. The manipulation of the cells and then putting it back into the patient. And then any of their outpatient or inpatient care as well,” CMS Administrator Seema Verma said during a call with reporters. The update could be “a key pivot point” for uptake in Medicare patients, Gilead’s new CEO Daniel O’Day said during a conference call last week ahead of the CMS announcement. “We know that the use of Yescarta in Medicare patients also has an influence in centers on how they treat their non-Medicare patients,” he added. “Lots to continue to innovate here, not just on the science, but also on the reimbursement and access programs.”

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Aptar Expands Pharmaceutical Services Announces Exclusive Collaboration Between Aptar Pharma and Fluidda

Aptar Pharma and Fluidda | September 23, 2022

Nanopharm, an Aptar Pharma company and leader in contract research and development services for orally inhaled and nasal drug products announced an exclusive collaboration with Fluidda, a leader in the field of Functional Respiratory Imaging. The companies will leverage their respective proprietary technology platforms to help accelerate U.S. Food & Drug Administration approvals for orally inhaled generic products via the alternative bioequivalence pathway. Nanopharm was acquired by Aptar in 2019, as part of the company’s strategy to expand its services offerings and partner with pharmaceutical companies earlier in the drug development process. Nanopharm has pioneered the development of the alternative bioequivalence regulatory pathway for U.S. FDA approval of generic OIDPs for Asthma and Chronic Obstructive Pulmonary Disease using its proprietary in vitro and in silico service platform, SmartTrack™. Fluidda’s proprietary in silico platform FRI delivers quantitative predictions of regional drug deposition in disease state lungs using Computational Fluid Dynamics. The FRI platform provides critical information to help understand the availability and activity of the drug at the site of action in the lungs, when complemented by Nanopharm’s local lung physiologically-based pharmacokinetic model platform and its in vitro data. This novel approach is intended to allow pharma companies to file Abbreviated New Drug Application dossiers without the need to perform time-consuming, costly and often unpredictable clinical end-point studies. Similarly, it can support 505(b)(2) filings, by derisking and abbreviating clinical studies. “We are pleased to solidify our relationship with Fluidda and its world-leading FRI technology. A company would have to spend several years to achieve the current combined expertise and experience of Nanopharm and Fluidda. The collaboration between Nanopharm and Fluidda has the potential to revolutionize this arduous regulatory pathway,” Dr. Jag Shur, Vice President, Science & Technology at Nanopharm Having already worked together closely for a number of years, Nanopharm and Fluidda have gained a unique insight into the complex and continually evolving regulatory requirements. This exclusive collaboration deepens the relationship between Fluidda and Nanopharm, benefiting both patients and customers with an uncompromised and holistic approach in developing the scientific rationale to demonstrate bioequivalence using only in vitro and in silico methodologies. The first potential approval of an OIDP using the alternative bioequivalence approach is pending, and, when approved, will further validate Nanopharm’s SmartTrack™ as the go-to solution for alternative bioequivalence studies and should accelerate demand for the companies’ collective services. With momentum building for the transition to new lower global warming potential (GWP) propellants for pMDIs, SmartTrack™ will also help companies to understand and modulate the impact of these new propellants on drug deposition and dissolution in the lungs, giving confidence in the performance of the reformulated product before embarking on any necessary clinical studies. Dr. Jan de Backer, CEO of Fluidda, stated, “Fluidda has already demonstrated the applicability of its FRI platform to provide more objective data for evaluating and administering inhaled drug products accurately. Nanopharm’s unique SmartTrack™ platform provides us with clinically-relevant input data without having to actually go into the clinic, which really adds another level of confidence to the models, and provides an integrated perspective.” Guillaume Brouet, Vice President, Analytical, Regulatory and Scientific Affairs at Aptar Pharma, commented, “Aptar Pharma is delighted to collaborate with Fluidda on this important development, which reinforces our mission to help customers derisk and accelerate their drug product development programs.” About Aptar Aptar Pharma is part of AptarGroup, Inc., a global leader in the design and manufacturing of a broad range of drug delivery, consumer product dispensing and active material science solutions and services. Aptar’s innovative solutions and services serve a variety of end markets including pharmaceutical, beauty, personal care, home care, food and beverage. Aptar Pharma’s analytical, laboratory and regulatory services add value at every stage of the drug development process, accelerating and de-risking the program along the way. Nanopharm, an Aptar Pharma company, is a leading provider of specialized analytical and product development services, with a focus on orally inhaled and nasal drug products. Aptar is headquartered in Crystal Lake, Illinois and has 13,000 dedicated employees in 20 countries. About Fluidda FLUIDDA, founded in 2005, is the world leader in the field of Functional Respiratory Imaging (FRI). This technique combines HRCT scans and Computational Fluid Dynamics technology (CFD), which offers vast improvements by making clinical trials shorter, faster and thus, more cost effective. FRI also helps patients and healthcare providers in offering a unique entry point in personalized medicine, by optimizing diagnosis, monitoring disease progression and the effects of therapy including accurate assessment of the deposition of inhalation medication. Fluidda’s mission is to optimize treatment pathways, reduce healthcare costs and to limit the go-to-market time of respiratory drugs, pulmonology medical devices and therapies. Fluidda has offices in Belgium, the United States and Portugal.

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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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BUSINESS INSIGHTS

Athira Pharma Advances Phase 2/3 LIFT-AD Clinical Study of Fosgonimeton in Mild-to-Moderate Alzheimer’s Patients

Athira Pharma, Inc. | October 18, 2022

Athira Pharma, Inc. a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced that following an unblinded interim efficacy and futility analysis, an independent data monitoring committee recommended continuation of the LIFT-AD study of fosgonimeton in patients with mild-to-moderate Alzheimer’s disease. The committee also determined that, with the additional enrollment of fewer than 150 patients for a total enrollment of less than 300 patients without background therapy the study will be well powered for the primary endpoint given the preliminary effect size observed. The primary endpoint of LIFT-AD is the Global Statistical Test, an unweighted composite score comprising measures of cognition and function. Results from the completed exploratory ACT-AD Phase 2 study showed a favorable safety profile and suggested positive effects on measures of cognition function and neurodegeneration in patients taking fosgonimeton without background therapy. Guided by these results, the Company proactively amended LIFT-AD to focus on patients not on background therapy. The unblinded interim analysis was then conducted in approximately 100 patients not on background therapy to corroborate observations from ACT-AD and ensure LIFT-AD is well powered to determine the effect of fosgonimeton on clinically meaningful and commercially relevant endpoints. “The results from the data monitoring committee’s unblinded analysis give us confidence in a potentially positive outcome for LIFT-AD, as stringent evaluation criteria were applied based on validated and clinically meaningful cognitive and functional outcomes. This analysis supports the potential clinical benefits of fosgonimeton treatment and underscores the rationale for continued development of this promising new therapy.” Hans Moebius, M.D., Ph.D., Chief Medical Officer of Athira “We are very excited by the results of this independent review as we believe they mitigate the risk of the fosgonimeton development plan, support the potential clinical benefit of fosgonimeton and inform the sample size needed to achieve success with LIFT-AD,” said Mark Litton, Ph.D., President and Chief Executive Officer of Athira. “We are now targeting to complete enrollment in mid-2023 and report topline data in early 2024. Importantly, we have a strong balance sheet to execute our plans through key data readouts and beyond. Moving forward, we remain keenly focused on advancing this novel investigational therapy with the hope of positively impacting the lives of millions of Alzheimer’s patients. “Our goal with fosgonimeton is to demonstrate its ability to improve cognition and function and to ultimately provide neuroprotection. The ACT-AD study suggested these benefits, and the results of the LIFT-AD interim analysis corroborate those findings,” added Dr. Litton. “We believe any drug that can demonstrate neuroprotection could become a treatment of choice for mild-to-moderate Alzheimer’s patients.” About the LIFT-AD Clinical Study LIFT-AD is a randomized, double-blind, placebo-controlled, parallel-group study of fosgonimeton for patients with mild-to-moderate Alzheimer’s disease. Patients are randomized across two dose groups and one placebo group on a 1:1:1 basis to receive a subcutaneous injection of fosgonimeton or placebo once daily over a treatment course of 26 weeks. The primary endpoint for LIFT-AD is the Global Statistical Test, an unweighted composite score comprising measures of cognition (Alzheimer's Disease Assessment Scale-Cognitive Subscale and function. Additional information on the LIFT-AD study can be found at: NCT04488419. About Fosgonimeton Fosgonimeton is a small molecule designed to enhance the activity of hepatocyte growth factor (HGF) and its receptor, MET, to impact neurodegeneration and regenerate brain tissue. The function of the HGF/MET receptor system may be impaired in the brain under conditions of neurodegeneration. In addition to Alzheimer’s disease, fosgonimeton has the potential to address the broader dementia population, including Parkinson’s disease dementia and Dementia with Lewy bodies, as the mode of action focuses on network recovery and synaptic signal transmission in the brain. The ACT-AD trial was supported by a grant from the National Institute on Aging of the National Institutes of Health under Award Number R01AG06268. The information presented in this press release is solely the responsibility of Athira and does not necessarily represent the official views of the National Institutes of Health. About Athira Pharma, Inc. Athira Pharma, Inc., headquartered in the Seattle, Washington area, is a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration. Athira aims to provide rapid cognitive improvement and alter the course of neurological diseases with its novel mechanism of action. Athira is currently advancing its pipeline of therapeutic candidates targeting the HGF/MET neurotrophic system for Alzheimer’s and Parkinson’s disease dementia, Dementia with Lewy bodies and neuropsychiatric indications.

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