This Phase II pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg (250mg for body weight <50 kg) daily administered orally compared to placebo over a treatment period of 24 weeks in males with BMD.
Funding Source - FDA OOPD

Start Date07 Jul 2022

Sponsor / Collaborator

ReveraGen BioPharma, Inc.

[+1]

NCT04348890 / WithdrawnPhase 1/2

A Phase I/II Open-Label, Proof-of-Concept Study of Vamorolone in Children and Adolescents With Mild-Moderately Active Ulcerative Colitis

This is a Phase I/II, multi-center, open-label proof-of-concept study of vamorolone. Twenty participants with a flare of mild or moderately active Ulcerative Colitis (defined as a Pediatric Ulcerative Colitis Activity Index [PUCAI] 10-60) will be enrolled and receive vamorolone 6 mg/kg/day orally once daily for 8 weeks.

Start Date01 Sep 2020

Sponsor / Collaborator

ReveraGen BioPharma, Inc.

NCT03439670 / CompletedPhase 2

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years with DMD.

Start Date29 Jun 2018

Sponsor / Collaborator

ReveraGen BioPharma, Inc.

[+2]

100 Clinical Results associated with ReveraGen BioPharma, Inc.

0 Patents (Medical) associated with ReveraGen BioPharma, Inc.

Literatures (Medical) associated with ReveraGen BioPharma, Inc.

01 Nov 2019·Inflammation research : official journal of the European Histamine Research Society ... [et al.]Q3 · MEDICINE

Vamorolone, a dissociative steroidal compound, reduces collagen antibody-induced joint damage and inflammation when administered after disease onset

Q3 · MEDICINE

Article

Author: Knoblach, Susan M ; Panchapakesan, Karuna ; Kanneboyina, Priya ; Lipson, Rachel ; Fiorillo, Alyson A ; Kanneboyina, Ila ; Partridge, Terence ; Cornish, Michaelyn R ; Nagaraju, Kanneboyina ; Phadke, Aditi ; Yu, Qing ; Damsker, Jesse M ; Morales, Melissa

OBJECTIVE AND DESIGN:

The objective of this study was to assess the effect of vamorolone, a first-in-class dissociative steroidal compound, to inhibit inflammation when administered after disease onset in the murine collagen antibody-induced arthritis model of arthritis.

ANIMALS:

84 DBA1/J mice were used in this study (n = 12 per treatment group).

TREATMENT:

Vamorolone or prednisolone was administered orally after disease onset for a duration of 7 days.

METHODS:

Disease score and bone erosion were assessed using previously described scoring systems. Cytokines were measured in joints via immunoassay, and joint cathepsin B activity (marker of inflammation) was assessed using optical imaging of joints on live mice.

RESULTS:

We found that vamorolone treatment led to a reduction of several disease parameters including disease score, joint inflammation, and the presence of pro-inflammatory mediators to a degree similar of that observed with prednisolone treatment. More importantly, histopathological analysis of affected joints showed that vamorolone treatment significantly reduced the degree of bone erosion while this bone-sparing property was not observed with prednisolone treatment at any of the tested doses.

CONCLUSIONS:

While many intervention regimens in other studies are administered prior to disease onset in animal models, the current study involves delivery of the potential therapeutic after disease onset. Based on the findings, vamorolone may offer an efficacious, yet safer alternative to conventional steroidal compounds in the treatment of rheumatoid arthritis and other inflammatory diseases.

01 Sep 2016·Inflammation research : official journal of the European Histamine Research Society ... [et al.]Q3 · MEDICINE

VBP15, a novel dissociative steroid compound, reduces NFκB-induced expression of inflammatory cytokines in vitro and symptoms of murine trinitrobenzene sulfonic acid-induced colitis

Q3 · MEDICINE

Article

Author: Heier, Christopher R ; Panchapakesan, Karuna ; Conklin, Laurie S ; Sandler, Anthony D ; McCall, John M ; Dillingham, Blythe C ; Sadri, Soheil ; Damsker, Jesse M

OBJECTIVE AND DESIGN:

The goal of this study was to assess the capacity of VBP15, a dissociative steroidal compound, to reduce pro-inflammatory cytokine expression in vitro, to reduce symptoms of colitis in the trinitrobenzene sulfonic acid-induced murine model, and to assess the effect of VBP15 on growth stunting in juvenile mice.

MATERIALS:

In vitro studies were performed in primary human intestinal epithelial cells. Colitis was induced in mice by administering trinitrobenzene sulfonic acid. Growth stunting studies were performed in wild type outbred mice.

TREATMENT:

Cells were treated with VBP15 or prednisolone (10 μM) for 24 h. Mice were subjected to 3 days of VBP15 (30 mg/kg) or prednisolone (30 mg/kg) in the colitis study. In the growth stunting study, mice were subjected to VBP15 (10, 30, 45 mg/kg) or prednisolone (10 mg/kg) for 5 weeks.

METHODS:

Cytokines were measured by PCR and via Luminex. Colitis symptoms were evaluated by assessing weight loss, intestinal blood, and stool consistency. Growth stunting was assessed using an electronic caliper.

RESULTS:

VBP15 significantly reduced the in vitro production of CCL5 (p < 0.001) IL-6 (p < 0.001), IL-8 (p < 0.05) and reduced colitis symptoms (p < 0.05). VBP15 caused less growth stunting than prednisolone (p < 0.001) in juvenile mice.

CONCLUSION:

VBP15 may reduce symptoms of IBD, while decreasing or avoiding detrimental side effects.

01 Dec 2015·Respiratory researchQ2 · MEDICINE

Pharmacologically-induced mitotic synchrony in airway epithelial cells as a mechanism of action of anti-inflammatory drugs

Q2 · MEDICINE

ArticleOA

Author: Tsegaye, Y ; Reeves, E K M ; Freishtat, R J ; Benton, A S ; Nino, G ; Damsker, J M ; Watson, A M ; Alcala, S E ; Haider, S K

BACKGROUND:

Mitotic synchrony is the synchronous progression of a population of cells through the cell cycle and is characteristic of non-diseased airway epithelial cells. However, we previously showed that asthmatic airway epithelial cells are characterized by mitotic asynchrony and are pro-inflammatory as a result. Glucocorticoids can induce mitotic synchrony that in turn suppresses the pro-inflammatory state of diseased cells, suggesting a novel anti-inflammatory mechanism of action. Herein, we benchmarked traditional glucocorticoids against the ability of a new clinical-stage dissociative steroidal drug, VBP15, for mitotic resynchronization and associated anti-inflammatory activity in asthmatic airway epithelial cells.

METHODS:

Primary airway epithelial cells differentiated at air-liquid interface were exposed to VBP15, dexamethasone or vehicle following in vitro mechanical injury. Basolateral cytokine secretions (TGF-β1, IL-6, IL-10, IL-13, and IL-1β) were analyzed at different time points using cytometric bead assays and mitosis was examined by flow cytometry.

RESULTS:

VBP15 improved mitotic synchrony of proliferating asthmatic cells in air-liquid interface cultures compared to vehicle-exposed cultures. VBP15 also significantly reduced the basolateral secretion of pro-inflammatory (i.e. IL-1β) and pro-fibrogenic cytokines (i.e. TGF-β1) in air-liquid interface-differentiated asthmatic epithelial cultures following mechanical injury.

CONCLUSION:

VBP15 improves mitotic asynchrony and injury-induced pro-inflammatory and fibrogenic responses in asthmatic airway epithelial cultures with efficacy comparable to traditional glucocorticoids. As it is predicted to show superior side effect profiles compared to traditional glucocorticoids, VBP15 holds potential for treatment of asthma and other respiratory conditions.

News (Medical) associated with ReveraGen BioPharma, Inc.

27 Nov 2024

·www.prnewswire.com

Myotonic Dystrophy Clinical Trial Pipeline Boom as Over 20 Companies Leading the Charge in Research and Development | DelveInsight

An increase in research and development (R&D) activities in myotonic dystrophy is expected to drive market growth by enhancing the understanding of the disease, accelerating drug discovery, and creating innovative treatments. With more R&D, new therapeutic options such as gene therapies, antisense oligonucleotides, and targeted small molecules can be developed, addressing the unmet needs in myotonic dystrophy care. Increased R&D also attracts funding and partnerships, fostering a competitive environment that can lead to improved patient outcomes and market expansion. LAS VEGAS, Nov. 27, 2024 /PRNewswire/ -- DelveInsight's ' Myotonic Dystrophy Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline myotonic dystrophy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the myotonic dystrophy pipeline domain. Key Takeaways from the Myotonic Dystrophy Pipeline Report DelveInsight's myotonic dystrophy pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for myotonic dystrophy treatment. Key myotonic dystrophy companies such as Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others are evaluating new myotonic dystrophy drugs to improve the treatment landscape. Promising myotonic dystrophy pipeline therapies such as AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others are under different phases of myotonic dystrophy clinical trials. On November 7, 2024, ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, announced that the US Food and Drug Administration has granted Rare Pediatric Designation (RPD) to ATX-01 for the treatment of myotonic dystrophy type 1. In November 2024, Dyne Therapeutics announced that the US Food and Drug Administration has cleared the Investigational New Drug application for DYNE-101, which is being evaluated in the ongoing, global Phase I/II ACHIEVE trial in adults with myotonic dystrophy type 1 (DM1). The ACHIEVE trial currently includes 56 participants and is fully enrolled through the 6.8 mg/kg Q8W cohort (approximate ASO dose). In August 2024, PepGen announced that both Health Canada and the United Kingdom Medicines and Healthcare products Regulatory Agency have cleared the Company's clinical trial application (CTA) submissions for the FREEDOM2 trial, and PepGen expects to initiate patient dosing in the second half of 2024. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-controlled, MAD clinical trial evaluating PGN-EDODM1 in approximately 24 adult patients with DM1 in Canada, the United Kingdom, and in the United States, subject to regulatory clearance for the treatment of myotonic dystrophy. In May 2024, Avidity Biosciences announced that the US Food and Drug Administration granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1. In April 2024, PepGen presented a poster on the PGN-EDODM1 program at the 14th International Myotonic Dystrophy Consortium (IDMC-14) Meeting titled "FREEDOM-DM1: Phase 1 Study Design to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1." In March 2024, Entrada achieved a milestone under its global collaboration with Vertex related to the clinical advancement of Vertex's Phase I/II clinical trial of VX-670, which triggered a USD 75 million payment. The Company expects to receive this payment in the second quarter of 2024. The Company is eligible to receive up to USD 485 million, inclusive of milestones achieved to date, for the successful achievement of certain research, development, regulatory and commercial milestones, and tiered royalties on future net sales for any products that may result from this collaboration agreement. In February 2024, PepGen Inc. announced that the US Food and Drug Administration had granted Fast Track designation to PGN-EDODM1, an investigational candidate for the treatment of myotonic dystrophy type 1. In January 2024, Juvena Therapeutics receives the FDA Orphan Drug Designation for JUV-161 for the treatment of Myotonic Dystrophy Type 1. Request a sample and discover the recent advances in myotonic dystrophy treatment drugs @ Myotonic Dystrophy Pipeline Report The myotonic dystrophy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage myotonic dystrophy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the myotonic dystrophy clinical trial landscape. Myotonic Dystrophy Overview Myotonic dystrophy is a genetic disorder characterized by progressive muscle wasting and weakness. It is caused by mutations in specific genes that lead to an abnormal expansion of repetitive DNA sequences. The two main types of myotonic dystrophy are Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2). DM1 is caused by a CTG repeat expansion in the DMPK gene, while DM2 is linked to a CCTG repeat expansion in the CNBP gene. Symptoms of myotonic dystrophy can vary but often include muscle stiffness (myotonia), weakness, and atrophy, primarily affecting the distal muscles and those of the face. Additional symptoms may involve cataracts, cardiac arrhythmias, endocrine issues, and cognitive difficulties. Diagnosis typically involves a combination of clinical evaluation, family history assessment, and genetic testing to identify the specific mutations. Electromyography can also be used to detect myotonia. Currently, there is no cure for myotonic dystrophy, and treatment focuses on managing symptoms. This may include physical therapy to maintain muscle function, medications to alleviate myotonia, and regular monitoring for cardiac issues. Genetic counseling is recommended for affected individuals and their families to understand the inheritance patterns and potential risks. Find out more about myotonic dystrophy treatment drugs @ Drugs for Myotonic Dystrophy Treatment A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report: Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials Myotonic Dystrophy Therapeutics Assessment The myotonic dystrophy pipeline report proffers an integral view of the myotonic dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Myotonic Dystrophy Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Myotonin protein kinase expression inhibitors, RNA interference, Sodium channel antagonists, Glycogen synthase kinase 3 beta inhibitors, Histamine H3 receptor antagonists, Histamine H3 receptor inverse agonists, MicroRNA inhibitor, RNA inhibitors, RNA splicing modulators Key Myotonic Dystrophy Companies: Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others. Key Myotonic Dystrophy Pipeline Therapies: AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others. Dive deep into rich insights for new drugs for myotonic dystrophy treatment, visit @ Myotonic Dystrophy Drugs Table of Contents For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs Related Reports Myotonic Dystrophy Market Myotonic Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key myotonic dystrophy companies, including Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, among others. Myotonic Dystrophy Epidemiology Forecast Myotonic Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted myotonic dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Duchenne muscular dystrophy companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics , among others. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Fast TrackPhase 2Orphan Drug

12 Nov 2024

·www.globenewswire.com

Santhera Enters into Exclusive Supply Agreement for AGAMREE® (Vamorolone) with Ali Al Suwaidi Trading Establishment (ASTE) in Qatar

Pratteln, Switzerland, November 12, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive supply agreement with Ali Al Suwaidi Trading Est. (ASTE) for the treatment of patients with Duchenne muscular dystrophy (DMD) with AGAMREE under a Named Patient Program. The agreement enables ASTE, a pioneering healthcare distributor with over four decades of experience in Qatar, to address the high unmet need of DMD patients in the region. As the sole distributor of AGAMREE to Qatari health institutions, ASTE will collaborate with Santhera to facilitate the management of patients under the Named Patient Program. “This distribution agreement with ASTE marks another significant milestone for Santhera as we expand the global availability of AGAMREE. By partnering with expert organizations that have a strong understanding of specialist markets, we ensure optimal patient access,” said Geert Jan van Daal, MD, PhD, Chief Commercial Officer of Santhera. “This collaboration represents our first step into another important geographical region, advancing our mission to make AGAMREE available to Duchenne patients across the globe.” “As a longstanding distributor in Qatar's healthcare sector, we are proud to partner with Santhera to facilitate the access of this innovative treatment to patients with Duchenne muscular dystrophy,” said Ali Mohamed Al-Suwaidi, Chairman of Ali Al Suwaidi Trading Est. “With our extensive experience in specialty pharmaceutical distribution and established relationships with healthcare institutions across Qatar, we are well-positioned to ensure AGAMREE reaches the patients who need it most. This collaboration aligns perfectly with our mission to deliver innovative healthcare solutions to the Qatari market.” About AGAMREE® (vamorolone) 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 [1-4]. 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 [1-4]. 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 [1, 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 no restriction of growth [5] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [6]. 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. References:[1] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.[2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [3] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293[4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508[5] Ward et al., WMS 2022, FP.27 - Poster 71. Link.[6] Hasham et al., MDA 2022 Poster presentation. Link. About Duchenne muscular dystrophy 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. About SantheraSanthera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary 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 vamorolone for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. For further information, please visit www.santhera.com. AGAMREE® is a trademark of Santhera Pharmaceuticals. About Ali Al Suwaidi Trading Est (ASTE)ASTE is one of the leading distributors in Qatar with a history of 42 years in the industry. It is a Qatari-owned company, serving the healthcare sector through pharmaceuticals, medical and laboratory equipment distribution. Through selective partnerships, ASTE strives to identify latent needs and offer the most innovative and efficient products and services to efficiently cater to the healthcare system. Its vision is to be the trusted partner in healthcare and life sciences solutions, driving positive outcomes and consistently improving people´s well-being. ASTE invariably seeks to contribute to the development of the healthcare sector in the region and to continue to empower lives through innovative healthcare solutions. For further information please contact: public-relations@santhera.com orAndrew Smith, Chief Financial Officerandrew.smith@santhera.com Disclaimer / Forward-looking This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements. # # # Attachment 2024 11 12_PR Partnering ASTE_e_final

License out/inDrug Approval

04 Nov 2024

·www.prnewswire.com

Exosome Therapies on the Rise as 70+ Major Companies Driving Development in the Field | DelveInsight

Exosomes are emerging as key tools in personalized and regenerative medicine, offering precise, targeted therapy with low immune risk. Their potential spans cancer, chronic disease, and regenerative applications, driven by biopharma's investment in biologics and gene therapies. Stem cell-derived exosomes further enhance tissue repair and immune modulation, positioning exosomes as transformative in next-gen therapeutics. LAS VEGAS, Nov. 4, 2024 /PRNewswire/ -- DelveInsight's ' Exosomes Pipeline Insight 2024 ' report provides comprehensive global coverage of available, marketed, and pipeline exosomes-based therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the exosomes-based therapies pipeline domain. Key Takeaways from the Exosomes-based Therapies Pipeline Report DelveInsight's exosomes pipeline report depicts a robust space with 70+ active players working to develop 80+ pipeline therapies for various indications. Key exosomes-based therapies companies such as Capricor Therapeutics, Regeneus, Aegle Therapeutics, Innovex Therapeutics, Coya Therapeutics, EV Therapeutics, StemXO Therapeutics, Brexogen, ILIAS Biologics, Organicell Regenerative Medicine, Codiak BioSciences, Direct Biologics, Exostemtech, Brexogen, OmniSpirant, ShiftBio Inc., and others are evaluating new exosomes-based therapies to improve the treatment landscape. Promising exosomes-based therapies in the pipeline in various stages of development include CAP-1002, Progenza, AGLE 102, ExoCoVac, COYA 201, EV-101, StemXO Endosome Therapy, BRE AD01, ILB 202, Zofin, exoSTING, ExoFlo, CARTISOME, BRE MI01, OS006, SBI-407, and others. In July 2024, Aruna Bio announced the issuance of a US Patent for the composition of matter for neural exosomes used to deliver therapeutics to the brain. The company is on a rack to initiate Phase Ib/IIa clinical trial of AB126 in acute ischemic stroke in the second half of 2024. In July 2024, ILIAS Biologics announced that it had received the final clinical study report (CSR) for ILB-202, a Phase I clinical trial completed in Australia in November last year. In April 2024, Biological Dynamics joined the growing list of customers adopting the ExoVerita Pro instrument, a cost-efficient, automated exosomal enrichment platform. In April 2024, EXO Biologics secures up to EUR 16 million in Series A funding for exosome therapeutic development and clinical supply. In April 2024, ExoXpert entered a strategic partnership with Neucore Bio to leverage ExoXpert's platform for the evaluation of advanced exosome loading. In April 2024, INOVIQ and Promega signed a global supply and distribution agreement for EXO-NET. Last year's company entered into a Co-Marketing agreement. In March 2024, VivaZome Therapeutics and La Trobe University collaborated to explore the therapeutic potential of extracellular vesicles in models of stroke in the laboratory of Professor Chris Sobey. In January 2024, Aegle Therapeutics announced positive 12-week data for the first patient enrolled in Phase I/IIa clinical trial of AGLE-102 for the treatment of severe second-degree burns. Request a sample and discover the recent advances in exosomes-based therapies @ Exosomes Pipeline Outlook The exosomes-based therapies pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage exosomes-based therapies, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the exosomes-based therapies pipeline landscape. Exosomes Overview Exosomes are small extracellular vesicles, typically ranging from 30 to 150 nanometers in diameter, that are secreted by various cell types. They play a crucial role in intercellular communication by facilitating the transfer of proteins, lipids, and RNA between cells. This process can influence various physiological and pathological conditions, including immune responses, cancer progression, and neurodegenerative diseases. By carrying bioactive molecules, exosomes can modulate the behavior of recipient cells, contributing to processes such as tissue repair, immune regulation, and the spread of cancer cells. Their ability to transport molecular cargo makes them a subject of extensive research, particularly in the context of disease mechanisms and therapeutic applications. In recent years, exosomes have garnered significant interest in the field of biomedicine as potential biomarkers for disease diagnosis and as vehicles for targeted drug delivery. Researchers are exploring their use in therapeutic strategies, such as the delivery of RNA-based therapies and proteins, due to their natural ability to evade the immune system and penetrate cellular membranes. Additionally, exosomes derived from stem cells or engineered for specific therapeutic purposes hold promise for regenerative medicine, offering a novel approach to enhance tissue regeneration and repair. The ongoing investigation into the biology of exosomes continues to reveal their multifaceted roles in health and disease, paving the way for innovative applications in diagnostics and therapeutics. Find out more about exosomes-based therapies @ Exosomes-based Therapies in Development A snapshot of the Exosomes-based Therapies Pipeline Drugs mentioned in the report: Learn more about the emerging Exosomes-based therapies @ Exosomes-based Therapies Clinical Trials Exosomes-based Therapies Therapeutics Assessment The exosome-based therapies pipeline report proffers an integral view of the emerging exosome-based therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Exosomes-based Therapies Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Cell replacements, Inflammation mediator modulators, Collagen type VII replacements, Immunosuppressants, NF-kappa B inhibitor Key Exosomes-based Therapies Companies: Capricor Therapeutics, Regeneus, Aegle Therapeutics, Innovex Therapeutics, Coya Therapeutics, EV Therapeutics, StemXO Therapeutics, Brexogen, ILIAS Biologics, Organicell Regenerative Medicine, Codiak BioSciences, Direct Biologics, Exostemtech, Brexogen, OmniSpirant, ShiftBio Inc., and others. Key Exosomes-based Therapies in Pipeline: CAP-1002, Progenza, AGLE 102, ExoCoVac, COYA 201, EV-101, StemXO Endosome Therapy, BRE AD01, ILB 202, Zofin, exoSTING, ExoFlo, CARTISOME, BRE MI01, OS006, SBI-407, and others. Dive deep into rich insights for exosomes-based therapies assessment, visit @ Exosomes-based Therapies Therapeutics Assessment Table of Contents For further information on the exosomes-based therapies pipeline therapeutics, reach out @ Exosomes-based Therapies Pipeline Landscape Related Reports Exosome Therapies Market Exosome Therapies Market Size, Target Population, Competitive Landscape & Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key exosome therapies companies, including Direct Biologics, Capricor Therapeutics, Aegle Therapeutics, Vitti Labs, Rion, Aruna Bio, EXO Biologics, EV Therapeutics, ReNeuron, Coya Therapeutics, Evox Therapeutics , among others. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DMD companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics , among others. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. Atopic Dermatitis Market Atopic Dermatitis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key atopic dermatitis companies, including Arcutis Biotherapeutics, Amgen, Kyowa Kirin, Dermavant Sciences, Cara Therapeutics, Pfizer, Arena Pharmaceuticals, BioMimetix, Eli Lilly and Company, Aldeyra Therapeutics, Inc., Hangzhou Yirui Pharmaceutical Technology Co., Ltd, LEO Pharma, Corvus Pharmaceuticals, Inc., Brexogen Inc., Sanofi, Shaperon, UCB Pharma, Q32 Bio Inc., Akeso, Apogee Therapeutics, Inc., Allakos Inc., Biosion, Inc. , among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1

100 Deals associated with ReveraGen BioPharma, Inc.

100 Translational Medicine associated with ReveraGen BioPharma, Inc.

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Pipeline

Pipeline Snapshot as of 26 Dec 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

NDA/BLA

Current Projects

Drug(Targets)	Indications	Global Highest Phase

Vamorolone ( GR )	Muscular Dystrophy, Duchenne More	NDA/BLA

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