A Long-term Non-interventional Study to Assess the Incidence of Skin Malignancies in Patients With Dystrophic and Junctional Epidermolysis Bullosa Receiving Treatment With Filsuvez

In patients with epidermolysis bullosa (EB), collagen does not form properly, so their skin is very fragile and blisters easily. Such patients are also at greatly increased risk of developing skin cancers. Filsuvez is a topical gel used to promote healing of skin lesions in patients with certain types of EB. In this observational study, patients with either dystrophic EB (DEB) or junctional EB (JEB) will receive standard of care treatment, whether Filsuvez or something else, and will be followed for up to 5 years. The main purpose is to see if the use of Filsuvez affects the likelihood of developing skin malignancies in these patient populations.

Start Date30 Nov 2024

Sponsor / Collaborator

Amryt Pharma Holdings Ltd.

[+1]

NCT06502990 / RecruitingPhase 3

Open-label, Phase 3b Study to Evaluate Effectiveness, Safety and Pharmacokinetic Parameters of Metreleptin in Patients Under 6 Years of Age With Generalised Lipodystrophy and Associated Diabetes Mellitus and/or Hypertriglyceridaemia

This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia

Start Date31 Jul 2024

Sponsor / Collaborator

Amryt Pharma Holdings Ltd.

NCT06484868 / RecruitingPhase 4

A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy

This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy

Start Date08 Feb 2024

Sponsor / Collaborator

Amryt Pharma Holdings Ltd.

100 Clinical Results associated with Amryt Pharma Plc

0 Patents (Medical) associated with Amryt Pharma Plc

Literatures (Medical) associated with Amryt Pharma Plc

17 Nov 2023·The Journal of Clinical Endocrinology & Metabolism

MPOWERED Trial Open-Label Extension: Long-term Efficacy and Safety Data for Oral Octreotide Capsules in Acromegaly

Article

Author: Obermayer-Pietsch, Barbara ; Pokramovich, Yulia ; Macut, Djuro ; Haviv, Asi ; Poteshkin, Yury ; Dreval, Alexander ; Gilgun-Sherki, Yossi ; Fleseriu, Maria ; Biermasz, Nienke ; Strasburger, Christian J ; Bondar, Irina ; Molitch, Mark

AbstractContextThe MPOWERED core trial (NCT02685709) and open-label extension (OLE) phase investigated long-term efficacy and safety of oral octreotide capsules (OOC) in patients with acromegaly. Core trial primary endpoint data demonstrated noninferiority to injectable somatostatin receptor ligands (iSRLs). Core trial completers were invited to participate in the OLE phase.ObjectiveTo assess long-term efficacy and safety of OOC in patients with acromegaly who previously responded to and tolerated both OOC and injectable octreotide/lanreotide and completed the core phase.MethodsThe unique study design of transitioning between OOC and iSRLs allowed within-patient evaluations. The proportion of biochemical responders (insulin-like growth factor I < 1.3 × upper limit of normal) at end of each extension year who entered that year as responders was the main outcome measure.ResultsAt year 1 extension end, 52/58 patients from both the monotherapy and the combination therapy groups were responders (89.7%; 95% CI 78.8-96.1), 36/41 (87.8%; 95% CI 73.8-95.9) in year 2, and 29/31 (93.5%; 95% CI 78.6-99.2) in year 3. No new or unexpected safety signals were detected; 1 patient withdrew owing to treatment failure. Patients who transitioned from iSRLs in the core trial to OOC in the OLE phase reported improved treatment convenience/satisfaction and symptom control.ConclusionPatient-reported outcome data support for the first time that transitioning patients randomized to iSRL (who previously responded to both OOC and iSRLs) back to OOC had a significant effect on patients’ symptoms score in a prospective cohort. The MPOWERED OLE showed long-term maintenance of response and sustained safety with OOC.

05 Oct 2023·Journal of the Endocrine Society

THU057 Predictors Of Response To Oral Octreotide Capsules In Patients With Acromegaly In A Large Prospective International Trial

Author: Strasburger, Christian Joseph ; Leonova, Nina Vasiljevna ; Pokramovich, Yulia ; Bondar, Irina ; Isaeva, Elena ; Fleseriu, Maria ; Dreval, Alexander ; Biermasz, Nienke ; Macut, Djuro P ; Molitch, Mark E ; Melmed, Shlomo ; Raverot, Gerald ; Haviv, Asi

AbstractDisclosure: M. Fleseriu: Consulting Fee; Self; Amryt (fka Chiasma), Ipsen, Ionis Pharmaceuticals Inc., Recordati, Pfizer, Inc. Research Investigator; Self; Amryt(fka Chiasma), Crinetics, Ionis Pharmaceuticals Inc., Recordati. Other; Self; Deputy Editor at EJE. A. Dreval: Research Investigator; Self; Research Investigator: Amryt(fka Chiasma). Y. Pokramovich: Research Investigator; Self; Research Investigator: Amryt(fka Chiasma). I. Bondar: Research Investigator; Self; Amryt(fka Chiasma). E. Isaeva: Research Investigator; Self; Chiasma. D.P. Macut: Research Investigator; Self; Principle Investigator: Amryt(fka Chiasma). Speaker; Self; Novartis Pharmaceuticals, Novo Nordisk, Sanofi, Pfizer, Inc., (spouse) Novo Nordisk. Other; Self; member of the Executive Committee of the European Society of Endocrinology (ESE). M.E. Molitch: Consulting Fee; Self; Corcept, Janssen Research & Development Company, Merck, Novo Nordisk, Pfizer, Inc., Novartis Pharmaceuticals. Grant Recipient; Self; Amryt(fka Chiasma), Crinetics, Ionis Pharmaceuticals Inc. Research Investigator; Self; Bayer, Inc. N.V. Leonova: Research Investigator; Self; Chiasma. G. Raverot: Consulting Fee; Self; Pfizer, Inc., Recordati Rare Diseases, Ipsen. Research Investigator; Self; Chiasma, Pfizer, Inc., Novartis Pharmaceuticals. Speaker; Self; Ipsen, Pfizer, Inc., Recordati Rare Diseases. A. Haviv: Employee; Self; Amryt(fka Chiasma). N. Biermasz: Advisory Board Member; Self; Recordati, Pfizer, Inc., Macro Registry. Grant Recipient; Self; Amryt(fka Chiasma). C.J. Strasburger: Advisory Board Member; Self; Sandoz, Ipsen, Recordati. Consulting Fee; Self; Ascendis, Amryt(fka Chiasma), Novo Nordisk, Merck, Sandoz, Recordati. Speaker; Self; Ipsen, Novo Nordisk, HRA Pharmaceuticals. S. Melmed: Advisory Board Member; Self; Ionis Pharmaceuticals Inc., Crinetics. Consulting Fee; Self; Ipsen. Grant Recipient; Self; Pfizer, Inc.Background: Oral octreotide capsules (OOC) are a treatment option for patients with acromegaly in several countries. Global MPOWERED trial (NCT02685709; Fleseriu et al, Lancet Diabetes Endocrinol. 2022; 10: 102-111)1 showed that OOC were noninferior to injectable somatostatin receptor ligands (iSRLs; octreotide or lanreotide) in maintenance of biochemical response in those previously responding to both OOC and iSRLs. Clinical characteristics predictive of response are useful to devise and implement personalized acromegaly treatment. Objective To identify patient and disease characteristics to predict the likelihood of OOC responsiveness. Methods Eligibility criteria included adults aged 18-75 years, acromegaly diagnosis at screening, and ≥6 months iSRL treatment (≥4 months responding to stable dose). All patients received OOC during the 26-week run-in phase. Responders at week 24 (IGF-I <1.3 × ULN and mean integrated GH <2.5 ng/mL) were randomized to receive OOC or iSRLs during the subsequent randomized controlled treatment (RCT) phase.1 Multivariate logistic regression analyses were performed to assess the relationship of baseline characteristics with OOC response during the run-in phase by comparing the group randomized to the RCT phase (those responding at end of run-in who opted to continue, n=92) to the group not randomized (n=54). Results One hundred forty-six patients enrolled in the run-in phase; 116 patients completed, 94 OOC responders, 92 randomized to RCT phase. IGF-I level prior to initiating OOC was the only predictor of response in the run-in phase for randomization into the RCT phase; randomized mean (SD) 0.8 × ULN (0.25), not randomized 1.0 × ULN (0.25), adjusted P<.0001. Other acromegaly characteristics, including duration of acromegaly, history of surgery and/or radiation, baseline pituitary adenoma size, residual postoperative pituitary adenoma size, GH level (randomized mean [SD] 0.78 ng/mL [0.644]; not randomized 1.06 ng/mL [1.010]), and signs/symptoms of GH excess (eg, joint pain, fatigue, sweating, swelling of extremities, snoring, sleep apnea, carpal tunnel syndrome, headache, and others) were not associated with subsequent OOC response (adjusted P=.11-P=1.0). Prior iSRL dose received was not a predictor of OOC response (adjusted P=1.0); randomized n (%): low dose 19 (20.7), middle 33 (35.9), high 40 (43.5); not randomized: low 11 (20.4), middle 22 (40.7), high 21 (38.9). Conclusions Baseline IGF-I level on iSRLs, but not other acromegaly baseline characteristics, were the sole predictor of subsequent OOC response. This IGF-I-value association may be expected, as response at end of run-in was determined by a strictly defined IGF-I cutoff. These results may guide clinicians considering transitioning patients with acromegaly from iSRLs to OOC.Presentation: Thursday, June 15, 2023

26 Jun 2023·British Journal of Dermatology

BG04 Oleogel-S10 (birch triterpenes) in the treatment of epidermolysis bullosa wounds: 24-month efficacy and safety data from the EASE study

Author: Sprecher, Eli ; Kiritsi, Dimitra ; Maher, Laura ; L. Bruckner, Anna ; Kern, Johannes S. ; Murrell, Dedee ; Cunningham, Tracy

AbstractEpidermolysis bullosa (EB) is a rare debilitating disorder characterized by skin fragility, blister formation and impaired wound healing. The abnormal wound healing seen in EB is a key driver for systemic disease and can lead to serious clinical complications, deformities and symptoms with a devastating impact on quality of life (QoL). The 90-day double-blind phase (DBP) of the phase III EASE study previously demonstrated accelerated wound healing for oleogel-S10 (birch triterpenes) vs. control gel in EB. Here, we report the safety and total wound burden results from the 24-month open-label phase (OLP), in which all patients received treatment with oleogel-S10. EB Disease Activity and Scarring Index (EBDASI) and body surface area percentage (BSAP) data are reported without visit windows to reflect a real-world situation more accurately, particularly considering the COVID-19 pandemic. The patient population was made up of those with dystrophic EB (n = 178; 86.8%) and junctional EB (n = 25; 12.2%). Further baseline characteristics are available in Table 1. In total, 141 patients (68.8%) completed the OLP. The mean (SD) treatment duration for all patients was 584.7 (246.1) days. Adverse events were reported in 77.1% of all patients in the OLP vs. 81.7% of those receiving oleogel-S10 in the DBP. Mean BSAP for patients treated with oleogel-S10 in the DBP reduced from 12.1% at study entry to 6.1% on completion of the EASE study. Similarly, the mean EBDASI skin activity score in the oleogel-S10 group improved from 19.6 to 15.1 at the end of the OLP. In addition, reductions in both BSAP and EBDASI from OLP baseline were observed in patients who transitioned from control gel to oleogel-S10 in the OLP. Oleogel-S10 has a reassuring long-term safety profile. Furthermore, the long-term impact of accelerated wound healing is evident through the sustained reduction in wound burden over time. This is encouraging given the regular reoccurrence of patients’ fragile wounds in the natural course of this chronic disease. Table 1Baseline characteristics

106

News (Medical) associated with Amryt Pharma Plc

18 Jun 2024

·www.biospace.com

Gamida Cell Announces New Leadership Under Ownership of Highbridge Capital Management

Stem-cell innovator poised to advance the commercialization of its cancer treatment, with $30 million of initial new capital and access to substantial additional funds to drive growth Experienced management team joins company, with more than 80 years of cumulative experience expanding access to innovative treatments for the patients who need them most BOSTON--(BUSINESS WIRE)-- Gamida Cell Ltd. (the “Company”), a cell therapy pioneer working to turn cells into powerful therapeutics, today announced new executive leadership following a successful restructuring transaction ("Transaction”) with certain funds managed by Highbridge Capital Management, LLC (collectively, “Highbridge”) on May 24, 2024. As part of the Transaction, Highbridge invested $30 million of new capital into Gamida Cell, and is expected to provide substantial additional capital to support ongoing operations, including the research and development, manufacturing, and commercialization of its cell therapeutics. Following the Transaction, Gamida Cell now operates as a wholly owned subsidiary of Ayrmid Limited, a UK entity. To advance the commercialization of Omisirge® (omidubicel-onlv), a NAM-modified cell therapy for allogenic hematopoietic stem cell transplant, Gamida Cell has named industry stalwart Dr. Joe Wiley as Chief Executive Officer, Rory Nealon as Chief Financial Officer, and Sheila Frame as Chief Commercial Officer. “Gamida Cell has made great strides in advancing potentially curative cell therapies for cancer and has demonstrated the tremendous potential of its life-saving therapies to improve prospects for patients who cannot find appropriate donors,” said Wiley. “With Highbridge's support, I look forward to leading Gamida Cell into its next phase as we work with hospitals across the U.S. to expand access to Omisirge for the patients who need it most." Wiley, Nealon, and Frame collectively bring more than 80 years of experience in building, growing, and commercializing innovative pharmaceutical treatments globally: Dr. Joe Wiley founded Amryt Pharma and served as the CEO until it was acquired by Chiesi Farmaceutici in April 2023. Prior to his role at Amryt, Wiley served as the Principal at Sofinnova Ventures and Medical Director at Astellas Pharma, and he held multiple investment management roles following his work as a neurologist. Rory Nealon was the co-founder, Chief Financial and Operating Officer at Amryt Pharma from 2015 through its acquisition in 2023. Before joining Amryt, he served executive roles at Trinity Biotech, first as the Chief Financial Officer then as the Chief Operations Officer. Sheila Frame most recently served as President Americas of Amryt Pharma until it’s sale to the Chiesi Farmaceutici in 2023. Frame has spent much of her career in executive positions leading the commercialization and growth of several important pharmaceutical products in oncology, immunology, and rare diseases in the US as well as globally. “Through our recent transaction and infusion of new capital, we are demonstrating our commitment to supporting this mission-driven company as it works toward broadening the availability of its critically important treatment,” said Jonathan Segal, Co-Chief Investment Officer at Highbridge. “We thank Gamida’s prior management team for their dedication in advancing Omisirge and we look forward to a productive collaboration with the newly appointed leadership team to drive Gamida Cell’s future success.” About Gamida Cell Gamida Cell is a cell therapy pioneer working to turn cells into powerful therapeutics. The company’s proprietary nicotinamide (NAM) technology leverages the properties of NAM to enhance and expand cells, creating allogeneic cell therapy products and candidates that are potentially curative for patients with hematologic malignancies. These include Omisirge® (omidubicel-onlv), an FDA-approved nicotinamide modified allogeneic hematopoietic progenitor cell therapy, and GDA-201, an intrinsic NK (natural killer) cell therapy candidate being investigated for the treatment of hematologic malignancies. Gamida Cell operates as a wholly owned subsidiary of Ayrmid Limited, a UK entity. For additional information, please visit or follow Gamida Cell on LinkedIn, X, Facebook or Instagram. About Highbridge Capital Management Founded in 1992, Highbridge Capital Management, LLC (“Highbridge”) is a global alternative investment firm offering differentiated credit and volatility focused solutions across a range of liquidity and investment pro including hedge funds, drawdown vehicles, and co-investments. The firm seeks to generate attractive risk-adjusted returns for sophisticated investors, which include financial institutions, public and corporate pension funds, sovereign wealth funds, endowments and family offices. Highbridge is headquartered in New York, with a research presence in London. In 2004 Highbridge established a strategic partnership with J.P. Morgan. Highbridge has over $4 billion in assets under management, as of April 1, 2024, and holds meaningful investments across the global healthcare and life sciences spectrum.

Cell TherapyAcquisitionExecutive Change

01 May 2024

·www.biospace.com

Poolbeg Pharma PLC Announces Significant POLB 001 Patent Granted in USA

Significant POLB 001 Patent Granted in United States Immunomodulator II patent grant further strengthens robust intellectual property portfolio for partnering LONDON, UK / ACCESSWIRE / May 1, 2024 / Poolbeg Pharma (AIM:POLB)(OTCQB:POLBF, 'Poolbeg' or the 'Company'), a biopharmaceutical company focussed on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, announces that, further to its announcement on20 March 2024,the Company has received the fully granted patent from the US Patent Office for its Immunomodulator II patent application. The claims cover a class of drugs (including POLB 001) for treating hypercytokinaemia (cytokine storm) and for preventing hypercytokinaemia in a patient after an immune response has been triggered. This encompasses cytokine storm that is induced in any disease indication. This further strengthens the Company's robust intellectual property portfolio for POLB 001, thereby enhancing the value and attractiveness of POLB 001 to potential Pharma partners. Poolbeg is actively building a comprehensive and worldwide IP portfolio, with patents in place covering p38 MAP kinase inhibitors for the treatment of severe influenza; and covering POLB 001 for the treatment of hypercytokinaemia. Further patent applications have been filed and have complementary coverage as the Company continues to expand its patent portfolio covering POLB 001 and the wider class of p38 MAP kinase inhibitors, particularly in respect of the treatment of cytokine release syndrome following cancer immunotherapy. Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg Pharma, commented:"The grant received from the US Patent Office highlights the strong progress we continue to make in developing and strengthening our patent portfolio for POLB 001, across multiple disease indications. This further enhances the value and attractiveness of POLB 001 for potential partners. "We believe POLB 001 holds immense promise in addressing unmet medical needs in cancer immunotherapy-induced cytokine release syndrome (CRS) and severe influenza, positively impacting patients and healthcare systems alike. Its impressive data package and strong patent portfolio, coupled with the promising >US$10 billion market opportunity in cancer immunotherapy-induced CRS alone, strengthens our confidence in its potential to positively impact global health and generate value for our shareholders." Enquiries Poolbeg Pharma Plc Jeremy Skillington, CEO Ian O'Connell, CFO +44 (0) 207 183 1499 Cavendish Capital Markets Ltd (Nominated Adviser & Joint Broker) Geoff Nash, Charlie Beeson, Nigel Birks, Harriet Ward (ECM) +44 (0) 207 220 0500 Shore Capital Stockbrokers Ltd (Joint Broker) David Coaten, Harry Davies-Ball (Corporate Advisory), Malachy McEntyre, Isobel Jones (Corporate Broking) +44 (0) 207 408 4090 J&E Davy (Joint Broker) Anthony Farrell, Niall Gilchrist +353 (0) 1 679 6363 Optimum Strategic Communications Nick Bastin, Vici Rabbetts, Elena Bates +44 (0) 208 078 4357 poolbeg@optimumcomms.com About Poolbeg Pharma Poolbeg Pharma plc is committed to the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, with a growing emphasis on rare and orphan diseases. Its model focusses upon developing its exciting clinical assets and commercialising approved and marketed drugs to support the growth of the Company and the development of its robust pipeline of innovative products, thereby driving significant value creation. Poolbeg is led by an experienced leadership team with a history of delivering significant shareholder value. The team has been strengthened by the appointment of three former members of the Amryt Pharma plc leadership team, with the intention of repeating Amryt's success and generating near term revenues. Poolbeg's clinical programmes target large addressable markets including cancer immunotherapy-induced CRS, infectious disease, and metabolic conditions such as obesity with the development of an oral GLP-1R agonist. It uses a cost-effective development philosophy to generate high quality human data to support partnering and further development. Its AI-led infectious disease programmes analyse unique data from human challenge trials to identify clinically relevant drug targets and treatments, leading to faster development and greater commercial appeal. For more information, please go to or follow us on Twitter and LinkedIn @PoolbegPharma. Forward-Looking Statements This announcement may contain forward-looking statements and the words "expect", "anticipate", "intends", "plan", "estimate", "aim", "forecast", "project" and similar expressions (or their negative) identify certain of these forward-looking statements. The forward-looking statements in this announcement are based on numerous assumptions and Poolbeg's present and future business strategies and the environment in which Poolbeg expects to operate in the future. Forward-looking statements involve inherent known and unknown risks, uncertainties and contingencies because they relate to events and depend on circumstances that may or may not occur in the future and may cause the actual results, performance or achievements to be materially different from those expressed or implied by such forward-looking statements. These statements are not guarantees of future performance or the ability to identify and consummate investments. Many of these risks and uncertainties relate to factors that are beyond Poolbeg's ability to control or estimate precisely, such as future market conditions, currency fluctuations, the behaviour of other market participants, the outcome of clinical trials, the actions of regulators and other factors such as Poolbeg's ability to obtain financing, changes in the political, social and regulatory framework in which Poolbeg operates or in economic, technological or consumer trends or conditions. Past performance should not be taken as an indication or guarantee of future results, and no representation or warranty, express or implied, is made regarding future performance. No person is under any obligation to update or keep current the information contained in this announcement or to provide the recipient of it with access to any additional relevant information. This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit . SOURCE: Poolbeg Pharma PLC View the original press release on accesswire.com

Executive ChangeImmunotherapy

30 Apr 2024

·www.biospace.com

Poolbeg Pharma PLC Announces Option Agreement to Acquire Orphan Drug Candidate

Exclusive Option Agreement to Acquire Orphan Drug Candidate for Behçet's Disease Novel therapeutic with Fast Track and Orphan Designation LONDON, UK / ACCESSWIRE / April 30, 2024 / Poolbeg Pharma (AIM:POLB)(OTCQB:POLBF), 'Poolbeg' or the 'Company'), a biopharmaceutical company focussed on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, announces that it has entered into an exclusive 12-month option agreement with Silk Road Therapeutics Inc, for a nominal fee, to acquire a novel topical muco-adherent formulation of Pentoxifylline (tPTX) for the treatment of oral ulcers in patient's suffering from Behçet's Disease. Key Highlights 12-month option agreement with Silk Road Therapeutics to acquire novel topical muco-adherent formulation of Pentoxifylline (tPTX), a treatment for oral ulcers in patient's suffering from Behçet's Disease which currently has no cure Phase 2 trial successfully completed, demonstrating superiority over standard of care Secured Orphan Drug Designation and Fast Track Designation from the FDA Positioned for a potential 505(b)(2) approval pathway in the U.S. Poolbeg will continue due diligence throughout the term of the exclusive option agreement which will include engagement with Silk Road Therapeutics to ascertain the clinical approval pathway There is a clear unmet medical need for an effective treatment for this rare disease, which has no cure. Behçet's Disease causes inflammation of blood vessels and tissues, resulting in debilitating symptoms, the most common being oral ulcers which impact essential functions like eating, drinking and speaking.[1] Patients also suffer from reduced psychosocial quality of life[2]. Current standard of care is inadequate with safety concerns and there is a clear need for alternative treatment options. The topical formulation of PTX, a small molecule with broad anti-inflammatory activity, has successfully demonstrated accelerated oral ulcer healing and decreased pain compared to standard of care in patients with Behçet's Disease in a Phase 2 clinical trial. The drug is positioned for a potential 505(b)(2) approval pathway in the U.S. which can result in a much less expensive and much faster route to approval and commercialisation. tPTX has successfully secured FDA Fast Track Designation, designed to expedite development and review of certain medicines. It has also received Orphan Drug Designation from the FDA, providing seven years of market exclusivity upon marketing authorisation. Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg Pharma, said: "The foundation of any pharma company is a strong pipeline of assets, and we believe that topical PTX could fit well into our pipeline alongside our other assets as we increase our focus on rare and orphan diseases. "We are delighted to enter this exclusive option agreement with Silk Road Therapeutics and Dr Timothy Coté, whose extensive experience, including as Director of the FDA Office of Orphan Products Development, adds invaluable insights into the regulatory pathway and ultimate approval and commercialisation of the drug. "tPTX has the potential to transform the lives of patients suffering from Behçet's Disease, who currently lack effective and safe long term treatment options. Under this exclusive option agreement, we will advance our due diligence process and engage with Silk Road Therapeutics to further understand the clinical pathway to approval, and we look forward to updating the market in due course." Timothy R. Coté, M.D., M.P.H., Chief Executive Officer of Silk Road Therapeutics, said: "Poolbeg Pharma shares our commitment to advancing healthcare innovation to support rare disease patients. With Fast Track Designation and Orphan Drug Designation secured, topical PTX has the potential to swiftly reach patients and offer a potentially transformative solution for those suffering from Behçet's Disease." Investor presentation Poolbeg Pharma CEO, Jeremy Skillington, PhD, and CBO David Allmond, along with Dr Timothy Coté, CEO of Silk Road Therapeutics,will provide a live presentation via the Investor Meet Company platform on Tuesday 30 April 2024 at 6pm BST. The presentation is open to analysts and investors, those who already follow Poolbeg on the Investor Meet Company platform will automatically be invited. Investors can sign up to Investor Meet for free and add Poolbeg Pharma plc to their company dashboard via: Enquiries Poolbeg Pharma Plc Jeremy Skillington, CEO Ian O'Connell, CFO +44 (0) 207 183 1499 Cavendish Capital Markets Ltd (Nominated Adviser & Joint Broker) Geoff Nash, Charlie Beeson, Nigel Birks, Harriet Ward (ECM) +44 (0) 207 220 0500 Singer Capital Markets (Joint Broker) Phil Davies, Sam Butcher +44 (0) 207 496 3000 J&E Davy (Joint Broker) Anthony Farrell, Niall Gilchrist +353 (0) 1 679 6363 Optimum Strategic Communications Nick Bastin, Vici Rabbetts, Elena Bates +44 (0) 208 078 4357 poolbeg@optimumcomms.com About Behçet's Disease Behçet's Disease is a rare condition that results in inflammation of the blood vessels and tissues. Inflammation across multiple parts of the body leads to diverse symptoms, the most prevalent being recurrent oral ulcers, in addition to genital ulcers and inflammation of the eye and skin lesions. It is not yet known what triggers the disease or causes it to persist. However, it is clear there is a significant immune component with some suggestions that exaggerated responses to pathogens and increased cytokine and chemokine production may play key roles in the disease.[3] About Poolbeg Pharma Poolbeg Pharma plc is committed to the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, with a growing emphasis on rare and orphan diseases. Its model focusses upon developing its exciting clinical assets and commercialising approved and marketed drugs to support the growth of the Company and the development of its robust pipeline of innovative products, thereby driving significant value creation. Poolbeg is led by an experienced leadership team with a history of delivering significant shareholder value. The team has been strengthened by the appointment of three former members of the Amryt Pharma plc leadership team, with the intention of repeating Amryt's success and generating near term revenues. Poolbeg's clinical programmes target large addressable markets including cancer immunotherapy-induced CRS, infectious disease, and metabolic conditions such as obesity with the development of an oral GLP-1R agonist. It uses a cost-effective development philosophy to generate high quality human data to support partnering and further development. Its AI-led infectious disease programmes analyse unique data from human challenge trials to identify clinically relevant drug targets and treatments, leading to faster development and greater commercial appeal. For more information, please go to or follow us on Twitter and LinkedIn @PoolbegPharma. Forward-Looking Statements This announcement may contain forward-looking statements and the words "expect", "anticipate", "intends", "plan", "estimate", "aim", "forecast", "project" and similar expressions (or their negative) identify certain of these forward-looking statements. The forward-looking statements in this announcement are based on numerous assumptions and Poolbeg's present and future business strategies and the environment in which Poolbeg expects to operate in the future. Forward-looking statements involve inherent known and unknown risks, uncertainties and contingencies because they relate to events and depend on circumstances that may or may not occur in the future and may cause the actual results, performance or achievements to be materially different from those expressed or implied by such forward-looking statements. These statements are not guarantees of future performance or the ability to identify and consummate investments. Many of these risks and uncertainties relate to factors that are beyond Poolbeg's ability to control or estimate precisely, such as future market conditions, currency fluctuations, the behaviour of other market participants, the outcome of clinical trials, the actions of regulators and other factors such as Poolbeg's ability to obtain financing, changes in the political, social and regulatory framework in which Poolbeg operates or in economic, technological or consumer trends or conditions. Past performance should not be taken as an indication or guarantee of future results, and no representation or warranty, express or implied, is made regarding future performance. No person is under any obligation to update or keep current the information contained in this announcement or to provide the recipient of it with access to any additional relevant information. [1] Hatemi G, Yurttas B, Kutlubay Z, Cote T, Derkunt S, Yazici Y, Yazici H. Pentoxifylline Gel for Oral Ulcers in Patients with Behçet's Syndrome [abstract]. Arthritis Rheumatol. 2019; 71 (suppl 10). . Accessed April 17, 2024. [2] Ali S, Nagieb CS, Fayed HL. Effect of Behcet's disease-associated oral ulcers on oral health related quality of life. Spec Care Dentist. 2023 Jul-Aug;43(4):435-442. doi: 10.1111/scd.12782. Epub 2022 Sep 20. PMID: 36127751. [3] behcetsuk.org This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit . SOURCE: Poolbeg Pharma PLC View the original press release on accesswire.com

Orphan DrugFast TrackPhase 2Clinical ResultDrug Approval

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Drug(Targets)	Indications	Global Highest Phase

Octreotide Acetate ( SSTR )	Acromegaly More	Approved
Metreleptin ( LEPR )	Lipodystrophy More	Approved
Birch triterpenes	Epidermolysis Bullosa, Junctional More	Approved
Lomitapide Mesylate ( MTTP )	Hyperlipidemia Type IIa More	Approved
AEG-41174 ( FLT3 x JAK2 )	Hematologic Neoplasms More	Discontinued

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