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Associated Press
27-05-2025
- Business
- Associated Press
Savara Receives Refusal to File (RTF) Letter From the U.S. Food and Drug Administration (FDA) for the Biologics License Application (BLA) for MOLBREEVI* to Treat Patients With Autoimmune Pulmonary Alveolar Proteinosis (autoimmune PAP)
LANGHORNE, Pa.--(BUSINESS WIRE)--May 27, 2025-- Savara Inc. (Nasdaq: SVRA) (the Company), a clinical-stage biopharmaceutical company focused on rare respiratory diseases, today announced that the Company received an RTF letter from the FDA for the BLA of MOLBREEVI as a therapy to treat patients with autoimmune PAP. Upon preliminary review, the FDA determined that the BLA submitted in March 2025 was not sufficiently complete to permit substantive review and requested additional data related to Chemistry, Manufacturing, and Controls (CMC). The RTF was not the result of safety concerns, and the FDA did not request or recommend additional efficacy studies. Within the next 30 days, the Company intends to request a Type A meeting with the Agency. Typically, Type A meetings are granted by the FDA within 30 days of the request. 'The requested CMC data outlined in the RTF letter are currently being generated, and we look forward to meeting with the FDA to align on next steps,' said Matt Pauls, Chair and Chief Executive Officer, Savara. 'Based on our understanding of the letter, we are confident we can thoroughly address the Agency's request and expect to resubmit our BLA in the fourth quarter of 2025. We remain highly confident in our program for autoimmune PAP and believe that our clinical data demonstrate that MOLBREEVI improves pulmonary gas transfer and respiratory health-related quality of life in this rare disease.' Pauls continued, 'As outlined in our Annual Report, we are working to establish a redundant supply chain. Pursuant to that strategy, we remain on track to complete the technology transfer with our second-source drug substance contract manufacturer in the fall. We have completed three upstream process performance qualification (PPQ) batches, are in the process of completing our downstream PPQ campaign and have begun our analytical comparability analysis.' The RTF does not impact previous designations granted by regulators for MOLBREEVI in autoimmune PAP. MOLBREEVI in autoimmune PAP has been granted Fast Track and Breakthrough Therapy Designations by the FDA, Orphan Drug Designation by the FDA and the European Medicines Agency (EMA), as well as Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations by the UK's Medicines and Healthcare Products Regulatory Agency (MHRA). About Autoimmune Pulmonary Alveolar Proteinosis (autoimmune PAP) Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in autoimmune PAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas exchange, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary lung infection develops. In the long term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant. About Savara Savara is a clinical-stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, MOLBREEVI*, is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (autoimmune PAP). MOLBREEVI is delivered via an investigational eFlow ® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of a large molecule. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at and LinkedIn. *MOLBREEVI is the FDA and EMA conditionally accepted trade name for molgramostim inhalation solution. It is not approved in any indication. MOLBREEVI is a trademark of Savara Inc. Forward-Looking Statements Savara cautions you that statements in this press release that are not a description of historical fact are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as 'expect,' 'intend,' 'plan,' 'anticipate,' 'believe,' and 'will,' among others. Such statements include, but are not limited to, statements related to the Company's intention to request a Type A meeting with the FDA, our confidence in our ability to thoroughly address the Agency's request, our expectations regarding the resubmission of the BLA and the timing of the resubmission, our belief that our clinical data demonstrate that MOLBREEVI improves pulmonary gas transfer and respiratory health-related quality of life in autoimmune PAP, and that we remain on track to complete the technology transfer with our second-source contract manufacturer in the fall. Savara may not actually achieve any of the matters referred to in such forward-looking statements, and you should not place undue reliance on these forward-looking statements. These forward-looking statements are based upon Savara's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risks associated with our ability to successfully develop, obtain regulatory approval for, and commercialize MOLBREEVI for autoimmune PAP; the occurrence and outcome of the planned Type A meeting with the FDA; our ability to address the FDA's request and successfully meet the requirements for resubmission; our ability to project future cash utilization and reserves needed for contingent future liabilities and business operations; the availability of sufficient resources for Savara's operations and to conduct or continue planned clinical development programs; and the timing and ability of Savara to raise additional capital as needed to fund continued operations. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. For a detailed description of our risks and uncertainties, you are encouraged to review our documents filed with the SEC including our recent filings on Form 8-K, Form 10-K, and Form 10-Q. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Savara undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law. View source version on CONTACT: Media and Investor Relations Contact Savara Inc. Temre Johnson, Executive Director, Corporate Affairs [email protected] KEYWORD: PENNSYLVANIA UNITED STATES NORTH AMERICA INDUSTRY KEYWORD: BIOTECHNOLOGY FDA HEALTH PHARMACEUTICAL CLINICAL TRIALS SOURCE: Savara Inc. Copyright Business Wire 2025. PUB: 05/27/2025 08:05 AM/DISC: 05/27/2025 08:05 AM
Yahoo
18-05-2025
- Health
- Yahoo
Savara Presented New Data From Pivotal Phase 3 IMPALA-2 Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at American Thoracic Society Conference (ATS) 2025
LANGHORNE, Pa., May 18, 2025--(BUSINESS WIRE)--Savara Inc. (Nasdaq: SVRA) (the Company), a clinical-stage biopharmaceutical company focused on rare respiratory diseases, today announced new data in two poster presentations at the ATS International Conference 2025. Data presented were from the Phase 3 IMPALA-2 clinical trial of molgramostim in aPAP and demonstrated that molgramostim reduces surfactant burden and improves health-related quality of life outcomes in patients with aPAP. ATS 2025 Posters Poster Title: Molgramostim Reduces Surfactant Burden and Number of Whole Lung Lavage Procedures in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP): Results From the IMPALA-2 Phase 3 Clinical TrialPresenter: Tisha S. Wang, M.D., Professor of Clinical Medicine, Senior Executive Clinical Vice Chair, University of California Los Angeles Department of MedicinePoster Number: 918Poster Location: PD05, Room 3009/3011 West Building, Level 3 Summary: Molgramostim reduced surfactant burden as measured by ground-glass opacification (GGO) scores, a radiological measure of surfactant burden. The mean reduction in GGO score from baseline to Week 24 was greater in the molgramostim group (n=78) than in the placebo group (n=79) (-2.1 vs. -1.1; P=0.0004) Fewer patients in the molgramostim group required rescue whole lung lavages (WLL) compared with placebo. During the 48-week double-blind treatment period, 6 patients (7.4%) in the molgramostim group underwent a total of 15 WLLs and 11 patients (13.3%) in the placebo group underwent a total of 24 WLLs Molgramostim reduces pulmonary surfactant burden, which drives the clinical manifestations of aPAP, and provides support for the potential beneficial treatment effect of molgramostim Poster Title: The Effects of Molgramostim on Respiratory Health-related Quality of Life and Patient-reported Outcomes in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP)Presenter: Ali Ataya, M.D., Associate Professor of Medicine, University of Florida, Division of Pulmonary and Critical Care MedicinePoster number: P31Poster Location: TP22, Area A, Hall F (North Building, Exhibition Level) Summary: Molgramostim showed benefit on measures of health-related quality of life (HRQoL) and patients' assessment of breathing problems and physical activity, including changes from baseline in St. George's Respiratory Questionnaire (SGRQ) Impact and Symptom scores, the EuroQol 5 Dimensions, 5 Levels (EQ-5D-5L), Patient Global Impression of Severity (PGIS), and Patient Global Impression of Change (PGIC) at Weeks 24 and 48, which were included as exploratory endpoints in the trial Molgramostim improved respiratory HRQoL as measured by changes from baseline to Week 24 in SGRQ Impact (P=0.0084) and Symptom scores compared with placebo, and the EQ-5D-5L, a generic HRQoL instrument comprised of a short descriptive system questionnaire that allows patients to rate their health across 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Odds ratios of responses on the EQ-5D-5L numerically favored the molgramostim group on 3 of the 5 dimensions (mobility, self-care, and usual activities) at Weeks 24 and 48 Molgramostim reduced the severity of breathing problems, as assessed by PGIS, at both Weeks 24 (P=0.0305) and 48 (P=0.0049). Additionally, more molgramostim patients reported improvements in overall change in daily physical activity level, as measured by more patients assessing themselves as "Much better" or "A little better" compared with placebo at Week 24 (P=0.0368) and Week 48 (P=0.0193) The abstracts were published in a supplement of the American Journal of Respiratory and Critical Care Medicine. For more details about the ATS International Conference please visit their website. The posters are available on the Congresses & Publications page of the Company's corporate website. About the IMPALA-2 Trial IMPALA-2 is a global, pivotal, Phase 3, 48-week, randomized, double-blind, placebo-controlled clinical trial designed to compare the efficacy and safety of molgramostim 300 mcg self-administered once daily by inhalation with matching placebo in patients with aPAP. The trial is being conducted at 43 clinical trial sites across 16 countries, including the U.S., Canada, Japan, South Korea, Australia, and countries in Europe, including Turkey. The primary efficacy assessment was diffusing capacity of the lungs for carbon monoxide (DLCO), a gas exchange measure, and the primary endpoint was change from baseline to Week 24 in percent predicted DLCO, with a secondary endpoint of change from baseline to Week 48 in percent predicted DLCO. Three additional secondary efficacy variables evaluated clinical measures of direct patient benefit: St. George's Respiratory Questionnaire (SGRQ) Total score, SGRQ Activity score, and exercise capacity using a treadmill test, with each endpoint measured at Weeks 24 and 48. The primary time point for efficacy assessments was at Week 24; however, efficacy was assessed through Week 48 to evaluate durability of effect. Safety was assessed through Week 48. All patients who completed the 48-week double-blind treatment period continued into a 96-week open-label period during which they are receiving molgramostim 300 mcg administered once daily. About Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in aPAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas exchange, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary lung infection develops. In the long term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant. About Savara Savara is a clinical-stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim inhalation solution (molgramostim), is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim is delivered via an investigational eFlow® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of a large molecule. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at and LinkedIn. View source version on Contacts Media and Investor Relations Contact Savara Johnson, Executive Director, Corporate Affairsir@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Forbes
28-03-2025
- Entertainment
- Forbes
Chef Jeremiah Langhorne On Being A Champion Of The Chesapeake
Chef Jeremiah Langhorne at the restaurant's garden in the Shaw neighborhood of Washington, D.C. This is where the creek empties, where the speck spook in the eelgrass. I'm no fisherman and everybody knows it. Heavy line, light lure — how much of my life does that sum up? Shuffle and shoal in the purblind glare. We haul in rocks and reds — or they do, mostly. The air's thick with salt and funk. Hot, I say. The captain's grave eyes . . . It's five years now. I'm still out there, a seagull, hovering angel watching them reeling me in. (From 'Angelus: Chesapeake Bay' by Ron Smith, former Poet Laureate of Virginia) Anyone who has dined at The Dabney, Chef Jeremiah Langhorne's award-winning restaurant in Washington, D.C., knows that attention to detail in food, service, and ambiance is an understatement when experiencing a meal there. From the 19th century brick row home that houses the restaurant, to the rustic decor, crackling fire in the middle of the open kitchen, and the menu which includes an illustrated postcard of D.C. in the 1800s, Langhorne and his team are hyper-intentional about the whole experience they offer guests. It is as if the past, present, and future are one. That guest experience, incorporating regional history from the inside out and on every plate, has taken years to cultivate and perfect, and as Langhorne will tell you himself, 'This is a lifelong pursuit and goal.' No matter how much training he has had in fine dining from all over the world, Langhorne continues to listen to the voice in his ear about shining a light on his own backyard. The Mid-Atlantic is his playing field, his inspiration for creativity, and motherland for his culinary activism. In his very mission to feed people, he has become a champion of the Chesapeake, its traumatic past and its hopeful future. From adding heat and warmth to depth of flavor and a link to history, open fire is at the core of ... More cooking at The Dabney. Opened in 2015, awarded Best New Restaurant (Bon Appetit) in 2016, a Michelin star in 2017, Best Chef (James Beard) in 2018, and local Mid-Atlantic publications along the way, The Dabney celebrates ten years this year, and can add another notch as it is currently a James Beard semi-finalist again, this time for an Outstanding Restaurant award (final selections are held in June). Like many chefs, however, Langhorne isn't kept awake at night by awards--albeit he appreciates the attention and doesn't discount its purpose--what keeps him up instead, is his mission at hand: to make delicious food, underscored by excellence, in a locale he reveres and hopes remains healthy and prosperous. With him on a boat in the middle of the Chesapeake last summer, Langhorne shared his philosophy about the area, what drives him and his team, and how he encourages other chefs to find ways to support the bay and its surrounding resources. And yet, figuring out how to do all of that without abusing it is a regular concern. Along with longtime colleague Phil Valliant, who now runs Valliant Oysters, and Captain Zack Hoisington, they set out to source dinner for the next day. Did You Know? Along the Chesapeake last summer, Chef Langhorne shared his insight and passion for the region. "The ... More Chesapeake Bay is an amazing body of water that, if protected, can provide us with incredible benefits. I think it's something special and worth protecting." When asked about that rumble inside, the desire to return home, he said, 'I knew that there was a lot of history here. I knew that there was a lot of depth, but there was no one on the surface talking about it or championing it as a chef. And so for me, it became really important to start to focus on what the Mid-Atlantic region was, as a cuisine, as a region; what were the ingredients and who were the people that made it so special.' Like many cultures, the regional cuisine and recipes and the ingredients found therein, become 'known and accepted as a wonderful expression, sometimes the best expression of a certain ingredient or a certain item,' Langhorne noted. 'In the same way that Brittany is known for incredible shellfish and lobsters in France, or you can go to certain prefectures in Japan for the best rice or the the best mushrooms.' With this in mind, Langhorne set out to study the region and local ingredients, and all the variables--like climate and soil-- that may affect them. 'I wanted to know what really was the best that the Mid-Atlantic had to offer, and what was the best the Chesapeake Bay Area had to offer,' he said. 'Obviously, I'm not going to accomplish that in five, ten, or even 25 years, but it's something that I felt was important to provide an identity for us.' Langhorne explains some of what he learned. That before the Industrial Revolution, the U.S. was on its way to creating an identity for regional food rivaling that of other rich, global cuisines. However, with advancements in machinery and shipping, for example, the world expanded. "We no longer needed to focus on what was growing outside our door," he said. Always foraging, Langhorne and Valliant found some edible greens on a break from the boat near Cape ... More Charles. Because we could soon preserve things, ship things, microwave things, and freeze things, that became the beginning of the end, of sorts, to what was a burgeoning identity for regional cooking in the U.S. Amidst his cooking career, Langhorne had a stint with Noma in Copenhagen--where utilizing what grows outside your door remains the mission despite its recent shift from restaurant dining to educational forum--an experience that inspired Langhorne to return to the U.S. with a narrowed focus. 'Then, when I spent time in Charleston, South Carolina, working with Sean Brock on reviving low country cuisine, I thought more and more about reviving the cuisine of my homeland and where I came from. And that's what we've been doing, and that's what will continue to do and work on forever.' Amidst Langhorne's deep dive into the area's history, he landed on The Oyster Wars [by David Faulkner] that describes the beginning of the bay as we know it. It documents the harmony that once was amongst settled Europeans and Native Americans and the abundance that was unlike anything seen before. Piles of oysters apparently reached 20, 30 ft. and for some time, several hundred years actually, was managed as a food source while keeping the bay clean and safe. Over time, however, millions of oysters bushes began leaving the Chesapeake, being shipped by train to Kansas City, to Chicago, or New York. 'And so as the East Coast and the Mid-Atlantic of the United States start to develop--that depletion started to really unbalance the health of the water in the bay. Oysters help filter the water, they help to keep it clean, and make sure that nature's balance stays in check." But, industrial usages, from the types of boats that were dredging oysters, or mono crop and farming, unfortunately put endless amounts of pesticides into the bay.' Made especially for the restaurant, every menu depicts a painted scene from the region's history. By the 1970s, the bay had become so abused, unsafe, untenable, fisheries were dying off. 'We used to have incredible things that we could get from the bay. We used to be able to get scallops, for example,' Langhorne noted. 'And then we humans, disrespected it and abused it. But thankfully, soon realized that we needed to change that.' Over the last 50 years, projects to re-energize and clean the bay have taken center stage and have been turning things around for the region. There's more health and abundance now and the trajectory is going in the right direction. Institutions like the Virginia Institute of Marine Science, for example, are constantly working to help promote different types of aquaculture. Today, the health of the bay is better, and yet not without its challenges. 'Another issue that has occurred and has been difficult to overcome, outside the shellfish realm, is blue catfish,' Langhorne said. 'It's an invasive species that was introduced from Asia and originally introduced for sport fishing. They are ferocious predators that will reproduce and grow and take up the majority of the biomass in the bay, not to mention eat up the native species.' But guess what? On the plus side, blue catfish are delicious. 'We serve them in the restaurant all the time, and we will continue to serve them,' Langhorne said. Langhorne believes that this issue is both a blessing and a curse. In terms of food security, the abundance of the blue catfish could assist for people in need, and on the flipside, rid the bay of a ferocious predator. 'There's a way to connect those dots, to provide delicious fish to more people and help the bay at the same time.' Dinner service at The Dabney. As The Dabney celebrates ten years in D.C. (officially in October) Langhorne reflects on the milestone. 'I'm really excited to look back at the work we've done and establish ourselves as real champions of the Mid-Atlantic region and the impact that we've had on it. Nevertheless, we're thinking about what's next and how we can double down on it and have an even broader impact for the future."
Yahoo
25-02-2025
- Yahoo
‘I just want to hug a dog': Wrongly convicted Maryland man welcomed home by rescue dog
MARYLAND () — A man who spent nearly 30 years in prison for a crime he did not commit was welcomed home last week by his family – and a rescue dog. James Langhorne was sentenced to life in prison for a murder he did not commit back in 1996. He spent nearly three decades in prison before the Office of the State's Attorney for Baltimore City vacated his wrongful conviction this year. At around 2:45 a.m. on Nov. 20, 1993, Lawrence Jones was walking home when he was approached by someone who pulled out a gun and shot him. The shooter fled and Jones succumbed to his injuries at the hospital later that night, . Fairfax County launches Conviction Integrity Unit for wrongful convictions The investigation went cold until July 1996, when information from a jailhouse informant and two now-recanted identifications led to Langhorne's arrest on Nov. 15 of that year. He was convicted of Jones's murder and sentenced to life in prison, plus 20 years. In February 2019, Langhorne requested the and the Office of the State's Attorney review his case. After an extensive, five-year investigation, officials concluded he was convicted of a crime he did not commit, according to the Feb. 13 about the vacatur. 'My sister, father, children, and grandchildren were there. MAIP was there. They made me feel welcome, told me to take my time, and process it. To cry to do what I needed. If I needed help they would be there. They even brought a dog out to see me,' Langhorne said at a press conference. Langhorne had grown up with dogs, rabbits and guinea pigs, instilling a love for animals from a young age. Several weeks before his exoneration, he told the (MAIP) that one of his first goals was to have his own dog. 'I grew up with animals, dogs – specifically German Shepherds,' he reportedly said. 'I just love all animals but I really want to have my own dog once I am out. I just want to hug a dog.' Tidal Basin seawall project closes new paths for cherry blossom visitors He was released from a Hagerstown prison on Feb. 17, 2025, and was welcomed back home by a rescue dog from the Humane Rescue Alliance (HRA). 'Jimmy's desire to hug a dog after his release underscores the transformative power of the human-animal bond,' said Lisa LaFontaine, President and Chief Executive Officer of HRA. Langhorne was introduced to Hope, a young Belgian Malinois who came to the shelter in November 2024. She approached him right away and he was able to experience 'the joy he had longed for, a feeling of love and connection that only animals can provide,' HRA and MAIP said. 'This is a new beginning for Jimmy, and we're so happy to witness these moments of joy,' said Shawn Armbrust, Executive Director of MAIP. 'Our legal efforts helped secure his release, but the emotional journey of healing ahead is just as important. The encounter with Hope is a reminder that sometimes, the simplest gestures of love and compassion can help rebuild a life.' Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
