Pulmonary Disease, Chronic Obstructive

Taylor Tieden for BioSpace On the FDA’s docket this month are two expansion bids, one for GSK’s asthma drug Nucala into COPD and another for Merck’s oral cancer drug for a pair of rare tumors. Amid the growing macro-level headwinds in the vaccine space, the FDA this month is expected to decide on Moderna’s mRNA COVID-19 shot, alongside a handful of other big verdicts. Read below for more. GSK Asks FDA to Approve Asthma Injection Nucala for COPD GSK is proposing its subcutaneous biologic Nucala to treat adult patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype. The FDA’s decision is due on May 7. A pulmonary disease, COPD is characterized by inflammation in the lungs, manifesting as breathlessness, coughs and airflow obstruction. According to GSK, some 40% of patients suffer from disease exacerbations caused by type 2 inflammation, which in turn is linked to high eosinophil counts in the blood. Nucala , a monoclonal antibody, targets and blocks the IL-5 cytokine, which is crucial to maintain the activity of eosinophils. GSK is supporting Nucala’s COPD application with data from the Phase III MATINEE trial. The pharma released a topline readout from the study in September 2024, noting that Nucala elicited a “statistically significant and clinically meaningful reduction” in yearly rates of moderate or severe disease exacerbations as opposed to placebo. Nucala is currently approved for eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome, and as an add-on maintenance treatment for asthma and chronic rhinosinusitis with nasal polyps. Arcutis Seeks Zoryve Expansion into Body and Scalp Psoriasis By May 22, the FDA will release its verdict on Arcutis Biotherapeutics’ proposed expansion for Zoryve into body and scalp psoriasis. Arcutis is backing its expansion bid with data from the Phase III ARRECTOR trial, which found that 66.4% of Zoryve-treated patients achieved clear or almost-clear skin, as opposed to 27.8% in placebo patients. Results also showed that 65.3% of patients in the Zoryve arm saw significant improvements in itch, versus 30.3% in the placebo group. Arcutis also filed findings from a Phase IIb study as well as long-term efficacy and safety data for Zoryve in plaque psoriasis. Zoryve is a topical phosphodiesterase 4 inhibitor that works by helping to suppress inflammation in the skin. The drug won FDA approval in 2011 for the treatment of plaque psoriasis in patients aged 12 years and up. If approved for body and scalp psoriasis, Zoryve would be a “truly meaningful innovation” for patients who suffer from inadequately controlled symptoms, Arcutis CEO Frank Watanabe said in a September 2024 release. Sanofi Eyes Extension of Meningococcal Disease Shot to Infants, Toddlers Sanofi’s meningococcal disease vaccine MenQuadfi is currently approved for the primary immunization of children aged two years and up. As a booster, it can be used in those 13 years and older who continue to be at risk of meningococcal disease. Sanofi is seeking to expand MenQuadfi’s label to also include infants and toddlers from six weeks to 23 months of age. The FDA is currently reviewing this proposal and is due to release its decision on May 23. If approved, MenQuadfi could protect this younger population from the A, C, W and Y serogroups of Neisseria meningitidis . In an April presentation before the CDC’s Advisory Committee for Immunization Practices, Sanofi presented data from several trials, including the Phase III MET42 study. Results showed that MenQuadfi was safe to use in infants and toddlers, with most side effects being mild or moderate in severity. Efficacy-wise, MenQuadfi was able to match a commercially approved vaccine in immune response and seroprotection. Liquidia Awaits Final Yutrepia Approval in Interstitial Lung Disease On May 23, Liquidia is expecting to win final approval of its inhalation powder Yutrepia for the treatment of patients with pulmonary arterial hypertension associated with interstitial lung disease (PH-ILD). The FDA gave the drug tentative approval in August 2024, indicating that it had cleared all efficacy and safety hurdles and satisfied regulatory standards of product quality. However, Liquidia still needed to wait out regulatory exclusivity for a competing product. Yutrepia is Liquidia’s inhalable version of treprostinil, a vasodilator that has known activity against pulmonary hypertension. Yutrepia is backed by data from the Phase III INSPIRE study, which found that the drug was both safe and well-tolerated in patients who were naïve to treprostinil or who were switching to Yutrepia from nebulized treprostinil. The INSPIRE trial found that patients treated with Yutrepia saw symptoms stabilize or improve over one year of dosing. Merck Hopes to Tack Two Rare Cancers Onto Welireg’s Label Merck is hoping to expand its oral cancer drug Welireg into advanced pheochromocytoma and paraganglioma (PPGL). The FDA’s decision deadline is May 26. PPGL refers to two types of rare tumors of the adrenal gland. Diagnosed in some 2,000 patients per year, they are typically caused by specific genetic mutations. Symptoms include high blood pressure, palpitations and headaches. According to Merck, up to 25% of PPGL cases are already at the metastatic stage at diagnosis. Merck is supporting Welireg’s PPGL bid with data from Cohort A1 of the Phase II LITESPARK-015 study. The pharma did not provide specific data in a January news release , revealing only that the application is based on objective response rate and duration of response in PPGL patients after Welireg treatment. The FDA has granted the drug priority review in this indication. Welireg is an orally available inhibitor of the HIF-2α transcription factor, which is involved in oxygen sensing and in the body’s response to hypoxia. The drug is indicated for patients with renal cell carcinoma (RCC) and those with von Hippel-Lindau disease—a rare genetic disease involving the formation of tumors across various organs—who also require treatment for associated RCC. If approved, Welireg will become the only drug for advanced PPGL in the U.S., Merck said. After Delay, Eton Nears Approval for Oral Hydrocortisone By May 28, the FDA will release its verdict on Eton Pharmaceuticals’ ET-400, an oral formulation of hydrocortisone, for the treatment of adrenal insufficiency in infants. This new target action date comes after the FDA in February announced that it needed more time to review Eton’s data package for ET-400. Eton submitted supplemental information in December last year, in response to a request from the regulator. ET-400 is a proprietary oral formulation of hydrocortisone that, according to the biotech, is stable at room temperature. If approved, ET-400 will follow in the footsteps of Eton’s Alkindi Sprinkle, which also has hydrocortisone as an active ingredient, but is formulated as oral granules. Alkindi Sprinkle, approved in 2020 , is designed to rapidly deliver glucocorticoids to children with adrenocortical insufficiency. In a July 2024 announcement , Eton CEO Sean Brynjelsen said that the approval of ET-400 would allow the biotech to “capture a greater percentage of the oral hydrocortisone market” and, alongside Alkindi Sprinkle, would open up a peak opportunity of “more than $50 million annually.” Moderna Seeks Approval for Next-Generation COVID-19 Shot The FDA’s last big milestone for the month belongs to Moderna and its investigational next-generation COVID-19 vaccine mRNA-1283. The regulator is expected to release its decision on May 31. In June 2024, the company released Phase III data for the experimental shot, touting better efficacy than Spikevax, its currently approved COVID-19 vaccine. Moderna did not provide specific data, only claiming that mRNA-1283 elicited a “higher immune response” against SARS-CoV-2 than Spikevax. Of note, Moderna emphasized at the time that mRNA-1283 could induce robust responses against Omicron and original strains of the virus—a benefit the company claimed was most evident in seniors over 65 years of age. The CDC’s vaccine advisors were supposed to convene in late February to discuss various vaccines, including mRNA-1283, before the meeting was postponed . The panel finally convened last month.

Serve You Rx, a pharmacy benefit manager (PBM), is collaborating with Amazon Pharmacy to offer home delivery services for maintenance medication to its members. Serve You Rx members will have access to online transactions; transparent, upfront pricing; automatic refill options; 24/7 access to clinical pharmacists; customer care support; and free same-day home delivery of prescriptions in some regions. Active prescriptions with Serve You Rx Home Delivery Pharmacy will be automatically transferred to Amazon Pharmacy without interruption in care. At the same time, specialty patients will transition to specialty pharmacy Waltz Health's AI-powered platform on the same day. "Our collaboration with Serve You Rx represents a meaningful step forward in transforming the pharmacy experience," Tanvi Patel, vice president and general manager of Amazon Pharmacy, told MobiHealthNews. "By combining Amazon's customer-obsessed approach with Serve You Rx's deep expertise in pharmacy benefits management, we're able to offer members a convenient medication experience. This relationship allows us to deliver not just prescriptions, but peace of mind, with 24/7 pharmacist access, transparent pricing and the reliable delivery service customers expect from Amazon." Justin Jasniewski, CEO of Serve You Rx, told MobiHealthNews, "Our strategic partnerships with Amazon Pharmacy and Waltz Health represent a bold step forward in redefining the pharmacy benefit experience. "By combining Amazon’s world-class, customer-focused home delivery capabilities with Waltz Health's cutting-edge, AI-powered specialty drug platform, we're building a seamless, member-centric ecosystem that addresses the full spectrum of pharmacy needs, from routine maintenance medications to complex specialty therapies." THE LARGER TREND In 2024, Amazon Pharmacy expanded its same-day medication-delivery offerings to residents of New York City and the greater Los Angeles area, providing patients with rapid access to prescription medicines for diabetes, flu, high blood pressure and other common conditions. The company was able to provide same-day delivery services due to small facilities that hold thousands of medications near the major metropolitan areas. That same year, Amazon Pharmacy and hybrid primary care provider One Medical launched a pilot program to expand Amazon's hybrid primary care offerings by giving One Medical providers the ability to request medication consultations for patients with complex medication regimens. The focus of the pilot, which launched in the fourth quarter of 2023, is on elderly patients who manage multiple chronic conditions. Consultations include the review of medication interaction or de-prescribing medication. In 2023, Amazon Pharmacy launched free drone delivery for prescription medications in 60 minutes or less for eligible customers in College Station, Texas. More than 500 medications for common conditions were made available via drone delivery, including flu, asthma and pneumonia. Once an order is placed, a pharmacist loads the medication into a Prime Air drone, and it is delivered to a customer's front door within an hour. That same year, Amazon Pharmacy partnered with multiple pharma companies to offer a new feature that automatically applies manufacturer-sponsored coupons to a patient's medication order when eligible. Amazon's online pharmacy works with GSK, Kaléo, Novo Nordisk and Dexcom to integrate coupons for their brand-name drugs, including COPD or asthma inhaler Trelegy; AUVI-Q, an injection for life-threatening allergic reactions; weight loss drug Wegovy; and sensors and transmitters for Dexcom's G6 and G7 continuous glucose monitors.

Positive high-level results from the Phase III KALOS and LOGOS trials in patients with uncontrolled asthma showed that AstraZeneca’s fixed-dose triple-combination therapy Breztri Aerosphere (budesonide/glycopyrronium/formoterol fumarate or BGF (320/28.8/9.6μg)) met all primary endpoints, demonstrating a statistically significant and clinically meaningful improvement in lung function compared with dual-combination inhaled corticosteroid/long-acting beta2-agonist (ICS/LABA) medicines. KALOS and LOGOS were replicate, randomised, double-blind trials designed to investigate Breztri as a potential treatment for asthma.1,2 The trials evaluated the efficacy and safety of Breztri versus maintenance treatment with ICS/LABA in adults and adolescents with uncontrolled asthma.1,2 Asthma is a common, chronic respiratory disease characterised by inflammation and muscle tightening in the airway (bronchoconstriction), which can make it difficult to breathe.3 As many as 262 million people worldwide are affected by asthma,3 and it is estimated that nearly half of those treated with dual therapy remain uncontrolled, which can significantly limit lung function and decrease quality of life.4,5 Alberto Papi, Professor and Chair of Respiratory Medicine at the University of Ferrara, and Director of the Respiratory Unit, CardioRespiratory Department, S. Anna University Hospital, Ferrara, Italy, and primary investigator, said: “Despite advancements in asthma treatments, millions of patients remain uncontrolled, which can cause frequent breathlessness, coughing and wheezing, significantly impacting their ability to perform daily activities. The results from the KALOS and LOGOS trials are exciting and demonstrate the potential of budesonide/glycopyrronium/formoterol to evolve the standard of care to more effectively treat asthma in a single inhaled triple therapy for patients who remain uncontrolled with dual maintenance therapy.” Sharon Barr, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “We are excited by the positive results from the KALOS and LOGOS trials, which demonstrate that Breztri could help improve the lives of the millions of patients living with asthma. These asthma data build on the well-established profile of Breztri in COPD, and we look forward to sharing with regulatory authorities to bring this important medicine to a wider group of patients.” There were no new safety or tolerability signals identified for Breztri in KALOS or LOGOS. Full results from the two Phase III trials will be shared with regulatory authorities and presented at an upcoming medical meeting. Breztri is an inhaled triple-combination therapy approved for the treatment of chronic obstructive pulmonary disease (COPD) in adults in more than 80 countries worldwide including the US, EU, China and Japan. Notes Asthma Asthma is a prevalent, chronic respiratory disease affecting as many as 262 million people worldwide,3 including over 25 million in the US.6 When uncontrolled, inflammation and muscle tightening in the airway (bronchoconstriction) may cause wheezing, breathlessness, chest tightness, coughing, and even death.3,7 Many patients remain uncontrolled despite the availability of standard of care medicines and continue to experience significant limitations on lung function and reduced quality of life.4,5 KALOS and LOGOS Phase III trials KALOS and LOGOS are replicate confirmatory, randomised, double-blind, double-dummy, parallel group, multi-centre, 24-to-52-week variable length Phase III trials to assess the efficacy and safety of BGF (320/28.8/9.6μg and 320/14.4/9.6μg) compared with two fixed-dose, dual-combination therapies of budesonide, an ICS, and formoterol fumarate, a LABA: PT009 (in an Aerosphere inhaler) and Symbicort pressurised metered-dose inhaler (pMDI).1,2 KALOS and LOGOS included approximately 4,400 randomised patients. The trial design was optimised to evaluate the 320/28.8/9.6μg dose of BGF. The primary efficacy endpoints for the two individual trials were a change from baseline in forced expiratory volume in 1 second (FEV1) area under the curve 0 to 3 hours (AUC0-3) at Week 24 and trough FEV1 over 12-24 weeks and over 24 weeks.1,2 In addition to the two registrational trials (KALOS and LOGOS),two qualifying trials, LITHOS and VATHOS, also met their primary endpoints.8,9 LITHOS and VATHOS included approximately 1,000 randomised patients. Breztri/Trixeo Aerosphere Budesonide/glycopyrronium/formoterol fumarate (BGF), approved under the brand name Breztri Aerosphere in Japan, China and the US, and Trixeo Aerosphere in the EU, is a single-inhaler, fixed-dose triple-combination of formoterol fumarate, a LABA, glycopyrronium bromide, a long-acting muscarinic antagonist (LAMA), with budesonide, an ICS, and delivered via the Aerosphere pMDI. Breztri/Trixeo Aerosphere is approved to treat adults with COPD in more than 80 countries worldwide including the US, EU, China, Japan, and was prescribed to more than 5.5 million patients globally in 2024.10 AstraZeneca in Respiratory & Immunology Respiratory & Immunology, part of AstraZeneca BioPharmaceuticals, is a key disease area and growth driver to the Company. AstraZeneca is an established leader in respiratory care with a 50-year heritage and a growing portfolio of medicines in immune-mediated diseases. The Company is committed to addressing the vast unmet needs of these chronic, often debilitating, diseases with a pipeline and portfolio of inhaled medicines, biologics and new modalities aimed at previously unreachable biologic targets. Our ambition is to deliver life-changing medicines that help eliminate COPD as a leading cause of death, eliminate asthma attacks and achieve clinical remission in immune-mediated diseases. AstraZeneca AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca Contacts For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here. References Matthew Bowden Company Secretary AstraZeneca PLC You are now leaving AstraZeneca.com You have selected a link that will take you to a site maintained by a third party who is solely responsible for its contents. AstraZeneca provides this link as a service to website visitors. 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