Darvadstrocel ( TiGenix)

Thanks to the FDA approval, Takeda said it’s looking to reverse a previous impairment loss tied to Eohilia’s FDA rejection in 2021. Eohilia, an oral suspension formulation of the corticosteroid budesonide, has surprisingly won over the FDA even after the drug’s developer, Takeda, had at one point abandoned the program following an FDA rejection. Monday, Takeda announced the FDA’s approval for Eohilia for treating people 11 years and older with the inflammatory disease eosinophilic esophagitis (EoE). This makes Eohilia the first FDA-sanctioned oral med for the condition. In 2022, Sanofi and Regeneron’s injectable Dupixent claimed the title as the first FDA-approved therapy for EoE. “As the treatment needs and goals of patients with EoE can vary, I welcome the flexibility that Eohilia offers as an oral medication,” Ikuo Hirano, M.D., from Northwestern University, an investigator in Eohilia’s clinical trials, said in a statement Monday. Eosinophilic esophagitis is a chronic inflammatory disease that can cause painful damage to the esophagus, resulting in difficulty in swallowing. The condition affects about 1 in 2,000 people in the U.S., and Takeda has estimated Eohilia could reach $300 million to $500 million in peak sales. Eohilia’s approval comes after some twists and turns. After a regulatory delay caused by additional data submissions, the FDA in late 2021 handed Takeda a rejection, blighting Eohilia’s prospect of becoming the very first official EoE drug. After reviewing the FDA’s complete response letter, Takeda realized that the agency wanted data from an additional clinical trial, and that it could not take the drug any further. “After a deep and careful assessment of the evolving treatment landscape in EoE, as well as considerations including operational challenges required for an additional clinical study, we could not justify further Eohilia development,” Takeda’s R&D chief Andy Plump, M.D., Ph.D., said during an investor call at the time. Fast forward to October 2023, when Takeda suddenly said it had refiled Eohilia in the prior month. This time, Plump said “strong data and remaining unmet need in the U.S. spurred significant grassroots support” for Eohilia from the gastrointestinal and EoE communities. Takeda worked with these communities to include additional data around Eohilia’s benefits in the resubmission, he explained. According to Plump’s comment and Eohilia’s current label, Takeda apparently played down findings from a one-year maintenance study and instead focused on the drug’s 12-week data from a phase 3 and a phase 2 trial in its resubmission. Although that strategy got Eohilia the green light from the FDA, it also led to a restriction. In Eohilia’s label, the FDA notes that the drug hasn’t shown to be effective for longer than 12 weeks. The label later points out that in a phase 3 extension study, continued treatment with Eohilia beyond 12 weeks didn’t show a statistically significant improvement versus re-randomization to placebo on some efficacy endpoints. Still, the drug appears to be a good option for 12 weeks of treatment. Between the phase 3 and phase 2 trials, Eohilia, given at 2mg twice daily, respectively helped 53% and 38% patients achieve histological remission after 12 weeks of treatment, versus 1% and 2.4% for those who received placebo. By comparison, Sanofi and Regeneron won Dupixent’s EoE nod with a 24-week analysis showing the weekly antibody drug led to histological remissions in 60% and 59% of patients in two parts of a phase 3 trial, versus 5% and 6% for the placebo group. As for disease symptoms measured by the patient-reported Dysphagia Symptom Questionnaire (DSQ), Eohilia delivered 10.2- and 14.5-point improvements, compared with 6.5- and 5.9-point improvements for placebo. Results on the same endpoint for Dupixent were 21.9 and 23.8 points, versus 9.6 and 13.9 points for placebo. Thanks to the approval, Takeda said it’s looking to reverse a previously recorded impairment loss tied to Eohilia’s rejection, but that it doesn’t expect the impact to be material. Takeda’s business has come under pressure lately after the Japanese pharma’s blockbuster ADHD drug Vyvanse lost U.S. market exclusivity in August. Adding to the woes are a phase 3 flop for the company’s stem cell therapy Alofisel in complex perianal fistulas, the global market withdrawal of EGFR lung cancer med Exkivity, and the voluntary retraction of an application for the dengue vaccine Qdenga in the U.S. The company has recently won two other FDA nods, one for colorectal cancer drug Fruzaqla, which Takeda in-licensed from Hutchmed a year ago; and the other for rare disease therapy Adzynma in congenital thrombotic thrombocytopenic purpura.

Dr. Gordon will have oversight over the SRF's global clinical trial program CONQUEST and will contribute to the research program across the SRF portfolio SAN FRANCISCO, Jan. 25, 2024 /PRNewswire/ -- The Scleroderma Research Foundation (SRF)— the nation's largest non-profit funder of scleroderma research—announces the appointment of Gregory Gordon, MD, JD as its first Chief Medical Officer (CMO). As CMO, Dr. Gordon will provide both strategic management and operations oversight for the recently announced global clinical trial program CONQUEST, which will involve 130 medical centers in 22 countries. He will also liaise with CONQUEST's two pharmaceutical partners—Sanofi and Boehringer Ingelheim—as well as any other partners that opt to bring innovative therapies to the CONQUEST platform. Dr. Gordon's contributions are expected to add focus to the ongoing work of SRF—in particular regarding its efforts to bring new and innovative therapeutic alternatives to scleroderma patients and the scleroderma community worldwide. Continue Reading Dr. Gregory Gordon joins Scleroderma Research Foundation as Chief Medical Officer "Dr. Gordon, with his impressive track record spanning over fifteen years in pivotal leadership roles in the biotechnology industry, brings a unique and vital perspective to the Scleroderma Research Foundation. His diverse experience is an invaluable asset to the SRF and the entire scleroderma community. Among his contributions, he will enhance our already extensive community outreach and patient engagement initiatives," said Joanne Gold, Executive Director of SRF. "I'm excited to be part of an organization with a long history of funding and supporting innovative research for the scleroderma community," said Dr. Gordon, who officially started his role in January. "I am pleased to join the SRF at this pivotal time to help realize the SRF's vision of building a global community tuned to rapid and efficient clinical development with CONQUEST." "With CONQUEST, the Scleroderma Research Foundation is deepening its commitment and support for research that addresses the high unmet needs of scleroderma patients. We are very excited to welcome Dr. Gordon to the SRF team with his deep experience and proven capabilities and his ability to drive the CONQUEST program forward. CONQUEST represents the first platform approach to clinical trials in rare autoimmune disease more generally and in particular to scleroderma," said Luke Evnin, Ph.D., Chairman of the SRF. Dr. Gordon joined the SRF after leading a team of physicians overseeing the development of potential treatments for multiple neurologic and pediatric diseases at PTC Therapeutics, where he was Vice President of Clinical Development. Originally a primary care physician at Harvard Vanguard Medical Associates, he became a well-respected figure in pharmaceutical development, working with teams responsible for successfully bringing multiple treatments to patients, including linaclotide (Linzess™), darvadstrocel (Alofisel™), and setmelanotide (Imcivree™). He has previously headed clinical development and operations at Rhythm Pharmaceuticals, served as the Head of US Medical at TiGenix, and served as the Global Clinical Affairs Lead at Nestlé Health Science. Before attending medical school at the State University of New York at Stony Brook, Dr. Gordon earned a J.D. from Columbia Law School and worked as an attorney in New York City. Greg is a native of Larchmont, New York. He now lives in Brookline, just outside Boston, with his wife and two sons. About the Scleroderma Research Foundation The Scleroderma Research Foundation (SRF), a 501(c)(3) organization, was established in 1987 with a mission to fund and facilitate the most promising, highest-quality research aimed at new treatments and, ultimately, a cure for scleroderma. Led by a Scientific Advisory Board comprised of some of the most highly regarded scientists in the nation, the SRF's research program actively seeks out the leading scientific minds from disparate fields including autoimmunity, immunology, genetics, and fibrosis to join the scleroderma research community. In addition to its core research program, the SRF recently announced the initiation of CONQUEST, a global platform clinical trial. The SRF has also led the formation of two other large-scale projects aimed at accelerating scleroderma research: The CONQUER Registry, the first nationwide longitudinal registry for scleroderma patients, and The GRASP Project, which examines scleroderma in the African American community. The SRF is also dedicated to educating people living with scleroderma and their caregivers as they learn about how to best manage the challenges of the disease. About CONQUEST Using a model first created over a decade ago to accelerate oncology drug development, the SRF platform clinical trial is the first of its kind in autoimmune diseases. In its initial iteration, CONQUEST will focus on Interstitial Lung Disease secondary to Scleroderma (SSc-ILD) which manifests in patients as lung fibrosis. In the future, the CONQUEST platform will be expanded to address other manifestations of scleroderma. The ground-breaking effort, conceived and led by the SRF, will enroll patients across more than 130 centers in more than 22 countries. CONQUEST allows multiple therapies to be evaluated under a common trial infrastructure with a common control arm. CONQUEST will initially enroll approximately 400 patients but with the advantages of the platform design, only approximately 134 patients will be randomized to the placebo arm (to receive standard-of-care). Accordingly, the design is very patient-centric since patients generally enroll in a trial to get access to novel medicines. However, the trial retains high statistical power for analysis of efficacy endpoints since the data from the placebo patients from all concurrently enrolled sub-protocols (e.g. Sanofi and Boehringer Ingelheim) will be pooled for efficacy analysis. This efficiency is not available in traditional trials and is one of the features that underscore the uniqueness of CONQUEST. By assembling a global network of high-performing centers that are dedicated to treating scleroderma, the SRF and its pharmaceutical partners expect to make enduring contributions to the scleroderma community by creating an ecosystem designed to enable success in new drug development. Sanofi and Boehringer Ingelheim are the inaugural partners for CONQUEST, which was first announced on August 1, 2023. Media Contact: Julie Richter | [email protected] | 480-818-8022 SOURCE Scleroderma Research Foundation