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October 1, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the positive outcome of the LIONHEART study, confirming vamorolone’s distinctive action also as a mineralocorticoid receptor antagonist, setting it apart from other corticosteroids. The LIONHEART study, an open-label randomized, placebo- and eplerenone-controlled study involving 30 healthy adult male subjects, met its primary endpoint. It demonstrated a statistically significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo (p<0.0001) following a fludrocortisone challenge. This increased ratio in urine provides clinical evidence for vamorolone’s unique mode of action also as a mineralocorticoid receptor antagonist (MRA) in humans. Combined with its known properties as a dissociative glucocorticoid receptor agonist, these findings further differentiate vamorolone’s pharmacological profile, distinguishing it from other corticosteroids. Cardiac complications such as cardiomyopathy are a leading cause of morbidity and mortality in boys with Duchenne muscular dystrophy (DMD). While treatment with corticosteroids (and ACE inhibitors) has demonstrated a delay in the onset of cardiomyopathy, the addition of MRAs including eplerenone to standard of care has also shown an improvement in left ventricular systolic dysfunction, the benefit of which increases with earlier initiation [1-3]. “Mineralocorticoid receptor antagonists are strongly recommended but late when cardiac function is already reduced and tend to be used in the presence of myocardial fibrosis as detected in magnetic resonance imaging,” explained Prof Karim Wahbi, PhD, MD, Cardiologist at the APHP Hospital Cochin, Paris, France. “What is intriguing about this mechanistic study is whether there is a synergistic benefit of the anti-inflammatory and MRA effects of vamorolone on the evolution of cardiac disease in children who started treatment early or if vamorolone could be of benefit to those who are already experiencing cardiac symptoms and wish to remain on a corticosteroid” “The LIONHEART study is an important milestone to establish the proof of concept for a cardioprotective potential of vamorolone,” stated Shabir Hasham, MD, Chief Medical Officer of Santhera. “We continue to collect data from patients who have been on vamorolone for up to seven years allowing us to better characterize long-term outcomes including any beneficial impact on cardiac complications in DMD.” AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [4-7]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [4-7]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has been shown to be a dual mineralocorticoid antagonist and a dissociative glucocorticoid agonist based on animal experiments [7]. It has no restriction of growth [8] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [9]. AGAMREE (vamorolone), an orphan medicinal product, is approved for use in the United States (Prescribing Information), the European Union (Summary of Product Characteristics) and the United Kingdom for the treatment of DMD. The LIONHEART study (SNT-I-VAM-026) is an open label randomized, 3-arm, parallel-group, placebo and eplerenone controlled study to evaluate the mineralocorticoid receptor antagonism (MRA) effect of vamorolone in 30 healthy adult male subjects after challenge with fludrocortisone, a known mineralocorticoid receptor agonist. The primary endpoint was defined as the ratio of sodium to potassium (Na/K) and the corresponding logarithm of the ratio (log10(10*Na/K) in urine at different time-points. Further analysis of the data is ongoing and will be presented at medical conferences. References: [1] Birnkrant et al. (2018) Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. [2] Raman et a. (2015) Lancet Neurol 14: 153–161 doi:10.1016/s1474-4422(14)70318-7 [3] Raman et al. (2017) Orphanet J Rare Dis 12: 39. doi:10.1186/s13023-017-0590-8 [4] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [5] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [6] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293 [7] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [8] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [9] Hasham et al., MDA 2022 Poster presentation. Link. Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. For further information, please visit www.santhera.com. The content above comes from the network. if any infringement, please contact us to modify.

MENLO PARK, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrine, oncology, metabolism, and neurology disorders by modulating the effects of the hormone cortisol, today announced it has initiated MONARCH, a Phase 2b trial of its proprietary selective cortisol modulator miricorilant in patients with non-alcoholic steatohepatitis (NASH). MONARCH is a 150-patient, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of miricorilant in adult patients with biopsy-confirmed NASH. Patients will receive either 100 mg of miricorilant or placebo orally twice weekly for 48 weeks. The primary endpoint is reduction in liver fat, with NASH resolution and fibrosis improvement as key secondary endpoints. “We are excited to initiate the MONARCH study and to build on the promising results of our Phase 1b study, which demonstrated that miricorilant effectively reduces liver fat, improves liver health and key metabolic and lipid measures and is well-tolerated. Miricorilant has the potential to greatly benefit the millions of patients with NASH. We look forward to sharing more details about MONARCH at the upcoming AASLD conference,” says Bill Guyer, PharmD, Corcept’s Chief Development Officer. About NASH Non-alcoholic steatohepatitis (NASH) is an advanced form of non-alcoholic fatty liver disease (NAFLD) that afflicts millions of patients and is a leading cause of liver-related mortality. Cortisol modulation may serve as a treatment for NASH since cortisol activity at the glucocorticoid receptor and mineralocorticoid receptor has been implicated in the development and progression of NAFLD. There are no current medications approved to treat NASH. About Corcept Therapeutics For over 25 years, Corcept’s focus on cortisol modulation and its potential to treat patients across a wide variety of serious disorders has led to the discovery of more than 1,000 proprietary selective cortisol modulators. Corcept’s advanced clinical trials are being conducted in patients with hypercortisolism, solid tumors, amyotrophic lateral sclerosis (ALS) and liver disease (NASH). In February 2012, the company introduced Korlym®, the first medication approved by the U.S. Food and Drug Administration for the treatment of patients with Cushing’s syndrome. Corcept is headquartered in Menlo Park, California. For more information, visit Corcept.com. Forward-Looking Statements Statements in this press release, other than statements of historical fact, are forward-looking statements based on our current plans and expectations that are subject to risks and uncertainties that might cause our actual results to differ materially from those such statements express or imply. These risks and uncertainties include, but are not limited to, our ability to operate our business; risks related to the development of relacorilant, miricorilant, dazucorilant and our other product candidates, including their clinical attributes, regulatory approvals, mandates, oversight and other requirements; and the scope and protective power of our intellectual property. These and other risks are set forth in our SEC filings, which are available at our website and the SEC’s website. In this press release, forward-looking statements include those concerning the timing and substance of our Phase 2b trial in patients with NASH and the development of miricorilant as a treatment for NASH, including its clinical attributes, regulatory approvals, mandates, oversight, and other requirements. We disclaim any intention or duty to update forward-looking statements made in this press release. CONTACTInvestor inquiries:ir@corcept.comMedia inquiries:CorceptCommunications@corcept.com www.corcept.com

New York, Oct. 26, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of AGAMREE® (vamorolone), a structurally unique steroidal anti-inflammatory drug to treat children and adolescents living with Duchenne muscular dystrophy (DMD). This multi-functional drug shows potent inhibition of pro-inflammatory NFkB pathways via high-affinity binding to the glucocorticoid receptor, high-affinity antagonism for the mineralocorticoid receptor, and membrane stabilization properties. This novel therapy, supported in part by MDA Venture Philanthropy, demonstrates similar efficacy to traditional corticosteroids with reduced negative downstream impacts or side effects. Vamorolone will be made available in the United States by Catalyst Pharmaceuticals. "Corticosteroids have been a first line treatment for DMD for many years but their utility has always been limited by the side effect profile, which includes weight gain, short stature, and decreased bone density among others. The approval of AGAMREE (vamorolone) provides people living with Duchenne, and their families, a powerful tool to treat the disease, while limiting some negative side effects associated with corticosteroids,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. “MDA is extremely proud to have been involved with vamorolone since its inception. It was one of the earliest investments to be made by our venture philanthropy arm and we are gratified to see that paying off in the form of a viable new therapy for Duchenne patients and the community.” A co-founder of ReveraGen BioPharma, the company that originally developed the treatment, is Eric Hoffman, PhD, who started his career as an MDA-support post-doc working with Louis Kunkel, Ph.D. on the initial discovery of the degenerative DMD gene in the 1980s. Hoffman cites the success of achieving FDA approval to the robust design of the clinical trials showing key differentiators in the significance of AGAMREE (vamorolone). “Muscular Dystrophy Association has been invested in this treatment since the beginning and they truly made the program possible,” said Eric Hoffman, Ph.D., Associate Dean, Research and Research Development; Professor, Pharmaceutical Services at Binghamton University. “The main concern was getting an effective treatment to children as quickly as possible. This was truly a community effort and getting a multitude perspectives and feedback from MDA and others helped make this a reality for so many families.” For Neekeesha and Jay Griffin of Harrisburg, North Carolina, the approval marks the culmination of over six years of experience with vamorolone. Their now 14-year-old son Franklin was the first boy enrolled in the US clinical trial for the treatment back in 2016, and he has remained on the therapy after the trial through an expanded access program. Vamorolone has already had significant impacts on families, including yielding positive results for the Griffin family that receive multidisciplinary care at the MDA Care Center at Duke University. “My son was diagnosed with Duchenne muscular dystrophy, and he was the first person dosed with vamorolone right before his 7th birthday. Time wasn’t our best friend, and we were willing to try this treatment because of the care of Dr. Smith [Edward Smith, M.D.] and the support MDA showed us,” said Jay Griffin, MDA family member. “This approval means everything to all the kids that wouldn’t otherwise have alternative options. Our son hasn’t seen any of the typical side effects that come with other steroids so we’re sticking with it.” Edward Smith, M.D., was most recently MDA Co-Director of the MDA Care Center at Duke University Medical Center where he started the pediatric neuromuscular clinic in 2007 through 2023. He is now Adjunct Professor of Pediatrics at Duke University Medical Center and Medical Director and Principal Investigator at Rare Disease Research-NC, LLC. “Having a novel steroid available that provides the benefits of current steroid therapy but with fewer side effects is a big step forward in treatment options. You can really see the difference with AGAMREE (vamorolone). Boys are taller and improved bone health may help them avoid painful fractures that can lead to even earlier loss of ambulation,” said Dr. Smith. “A treatment like AGAMREE is critical and a welcome steroid alternative to improving the lives and conditions of so many of our patients living with Duchenne muscular dystrophy,” said Barry Byrne, M.D., Ph.D., Chief Medical Advisor, MDA and the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. “The success of the clinical trials gives us another welcome alternative for treatment, and we look forward to getting this to the community.” Muscular Dystrophy Association’s funding in Duchenne muscular dystrophy Since its inception, MDA has invested almost $227 million in DMD/BMD research. For more information, please visit MDA research. About Duchenne muscular dystrophy (DMD) Duchenne muscular dystrophy (DMD) is a rare and progressive genetic muscle disease caused by the lack of a critical membrane-stabilizing protein called dystrophin, which results in muscle degeneration and weakness. DMD symptom onset is in early childhood, usually between ages 2 and 3. The disease primarily affects boys, but in rare cases it can affect girls. In Europe and North America, the prevalence of DMD is approximately 6 per 100,000 individuals. Doctors often diagnose the disease between ages 3 and 6 when children show early signs of significant muscle weakness, such as delayed ability to sit, stand, or walk and difficulties learning to speak. Over time, DMD becomes fatal as muscle degeneration disrupts lung and heart function. There is no cure for DMD, however, lifespans have been extended and quality of life has been improved for many through physical therapy and medications to address certain symptoms. With the latest approval, AGAMREE becomes a new therapeutic option for people living with this rare form of muscular dystrophy. Clinical trials support approval of AGAMREE (vamorolone) FDA’s approval of AGAMREE was based on the data from the pivotal Phase 2b VISION-DMD study as supplemented with safety information collected from three open-label studies, including extension studies. In these trials, AGAMREE was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months. Compared with current standard-of-care corticosteroids, this novel corticosteroid treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory, and behavior. MDA Resource Center The neuromuscular community may contact the MDA Resource Center for resources and support by phone at 1-833-ASK-MDA1 (1-833-275-6321) or by email at ResourceCenter@mdausa.org. About Muscular Dystrophy Association Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube. Attachment New FDA Drug Approval for Duchenne Muscular Dystrophy Mary Fiance, Vice President, Strategic Communications Muscular Dystrophy Association press@mdausa.org