Clinigen Healthcare Ltd.

- The Expanded Access Program (EAP) allows physicians to request access to TransCon PTH, the company’s investigational parathyroid hormone replacement therapy, for eligible patients in the United States - TransCon PTH is under Priority Review by the FDA, with a target PDUFA date of April 30, 2023 COPENHAGEN, Denmark, Jan. 04, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the online portal is now open for physicians wanting to request access to TransCon PTH (palopegteriparatide), the Company’s investigational parathyroid hormone replacement therapy, through the U.S. Expanded Access Program (EAP). Requests for access must be made by the patient’s treating physician through the program administrator, Clinigen Healthcare LTD, at Palopegteriparatide - Clinigen (clinigengroup.com). or by searching “palopegteriparatide” on the Clinigen Direct homepage: https://www.clinigengroup.com/direct/en/. To qualify, patients must be adults diagnosed with hypoparathyroidism who live in the U.S., have prior PTH treatment experience, and meet other criteria outlined below and in the full treatment protocol. Inclusion Criteria: Diagnosis of hypoparathyroidism;Patients with previous PTH-treatment experience;Serum albumin-adjusted calcium level ≥7.8 mg/dL and 25(OH) vitamin D in the normal range within 2 weeks before first dose;Male or female at least 18 years of age;Body mass index (BMI) 17–40 kg/m2; andBe willing and able to give written informed consent by signing an Institutional Review Board (IRB)-approved Informed Consent Form (ICF). Exclusion Criteria: Diagnosis of pseudohypoparathyroidism;Currently enrolled in an investigational drug or device study or has used an investigational drug or device within 30 days or 5 half-lives (whichever is longer) of Day 1;Severe renal impairment (estimated glomerular filtration rate <30 mL/min/1.73m2);Increased risk for osteosarcoma, such as those with Paget's disease of bone or unexplained elevations of alkaline phosphatase, hereditary disorders predisposing to osteosarcoma, or with a prior history of substantial external beam or implant radiation therapy involving the skeleton;Active malignancy within past 2 years excluding successfully resected thyroid carcinoma and non-melanoma skin cancer;Severe or decompensated cardiac disease within 26 weeks, including but not limited to class IV or Stage D heart failure, unstable angina, myocardial infarction or uncontrolled arrhythmias;Pregnant or lactating females, or females intending to become pregnant; orKnown allergy and/or sensitivity to palopegteriparatide or its excipients or prior PTH therapy. “We are pleased to have worked with the FDA to make TransCon PTH available for eligible patients in the U.S.,” said Edward Trott, M.D., Vice President, Global Medical Affairs at Ascendis Pharma. “Recognizing the needs expressed by the hypopara community, we will continue to work with the FDA and others to make TransCon PTH, if approved, more broadly available as quickly as possible.” Clinigen representatives are also available to U.S. healthcare professionals by phone toll-free at 1 (877) 768-4303. Full details of Ascendis Pharma’s TransCon PTH US EAP program (NCT05654701) are listed on https://clinicaltrials.gov/ct2/show/NCT05654701 and navigator.reaganudall.org. TransCon PTH (palopegteriparatide) is an investigational prodrug in development for adult patients with hypoparathyroidism. The safety and efficacy of TransCon PTH have not been established and TransCon PTH is not currently approved by the FDA. TransCon PTH is currently under Priority Review by the FDA, with a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2023. About Expanded AccessExpanded access is per FDA regulation a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. Please visit the FDA’s Expanded Access website for more information. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon platform to build a leading, fully integrated, global biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Heidelberg and Berlin, Germany; Palo Alto and Redwood City, California; and Princeton, New Jersey. Please visit ascendispharma.com to learn more. Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ PDUFA date of April 30, 2023 with respect to the FDA’s Priority Review of TransCon PTH, (ii) Ascendis’ ability to make TransCon PTH more broadly available as quickly as possible, (iii) Ascendis’ ability to apply its platform technology to build a leading, fully integrated global biopharma company, and (iv) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis products and product candidates; unforeseen safety or efficacy results in its development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including inflation, and the effects on its business from the worldwide COVID-19 pandemic and the ongoing conflict in the region surrounding Ukraine and Russia. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on March 2, 2022 and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma Group. © January 2023 Ascendis Pharma A/S. Investor Contacts:Media Contact:Tim LeeMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 374-6343+1 (650) 709-8875tle@ascendispharma.commedia@ascendispharma.comir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com

SUMMERLIN, Nev., Sept. 22, 2021 /PRNewswire/ -- Secura Bio, Inc. (Secura Bio) - ( ), an integrated pharmaceutical company dedicated to the worldwide development and commercialization of impactful oncology therapies, today announced that it has entered into a distribution agreement for COPIKTRA (duvelisib) with Clinigen Group Plc (Clinigen) covering 39 countries in Europe including Austria, Belgium, Croatia, Czech Republic, Denmark, Finland, France, Greece, Hungary, Ireland, Italy, The Netherlands, Poland, Portugal, Spain, Sweden and Switzerland. Secura Bio will promote and distribute COPIKTRA directly in Germany and the United Kingdom. Clinigen is a global, specialist pharmaceutical services and products company who partner with pharmaceutical and biotech companies to provide ethical access to medicines for patients with unmet medical needs. COPIKTRA was granted marketing authorization by the European Medicines Agency in May 2021 as monotherapy for the treatment of relapsed or refractory Chronic Lymphocytic Leukemia (CLL) in patients, who have received at least two prior therapies and for the treatment of Follicular Lymphoma (FL) that is refractory to at least two prior systemic therapies. CLL and FL are slow-growing incurable blood cancers that can lead to life-threatening complications such as anemia, serious infections, and bone marrow failure requiring treatment. The goal of therapy for patients with these cancers is to improve overall survival and quality of life. COPIKTRA is a dual inhibitor of PI3K-delta and gamma pathways, which are involved in the proliferation of malignant cells and are thought to play a role in the formation and maintenance of the supportive tumor microenvironment. Secura Bio is currently working with the haematology community and relevant authorities to ensure COPIKTRA is widely reimbursed and commercially launched across Europe in the coming months. "Partnering with Clinigen will enable COPIKTRA, an oncology therapeutic with a novel mode of action, to be widely and rapidly available to patients across Europe." said Joseph M. Limber, President and CEO of Secura Bio. "Leveraging Clinigen's commercial resources, supply and distribution infrastructure and regulatory expertise will allow physicians and patients throughout Europe to have access to another valuable and beneficial option to combat the debilitating illnesses of CLL and FL. We are excited to be working with Clinigen to ensure successful market access, commercialization and supply of Copiktra across the European geographies". Sam Herbert, Chief Operating Officer and Head of Products Division at Clinigen said that "Clinigen is very pleased to be partnering with Secura Bio. This new and exclusive agreement will help address a significant unmet need for patients with B-cell blood cancers across Europe. This agreement underlines Clinigen's strength in partnering with pharmaceutical companies and leveraging our significant commercial knowledge and infrastructure to ensure the right medicine gets to the right patients at the right time." Below is the full list of countries covered by the agreement: Austria, Belgium, Bulgaria, Croatia, Cyprus, Czech Republic, Denmark, Estonia, Finland, France, Greece, Hungary, Ireland, Italy, Latvia, Lithuania, Luxembourg, Malta, The Netherlands, Poland, Portugal, Romania, Slovakia, Slovenia, Spain, Sweden, Albania, Andorra, Bosnia Herzegovina, Iceland, Kosovo, Liechtenstein, Monaco, Montenegro, Northern Macedonia, Norway, San Marino, Serbia, Switzerland About Secura Bio, Inc. Secura Bio is an integrated, commercial-stage pharmaceutical company dedicated to the worldwide development and commercialization of impactful oncology therapies for physicians and their patients. For more information on Secura Bio, please visit . About Clinigen Clinigen Group plc (AIM: CLIN) is a global, specialist pharmaceutical services and products company focused on providing ethical access to medicines. Its' mission is to deliver the right medicine to the right patient at the right time. The Group operates from sites in North America, Europe, Africa and the Asia Pacific. Clinigen has more than 1,000 employees across five continents in 14 countries, with supply and distribution hubs and operational centres of excellence in key long-term growth regions. The Group works with 34 of the top 50 pharmaceutical companies; interacting with over 20,000 healthcare professionals across more than 120 countries. For more information on Clinigen, please visit About COPIKTRA®(duvelisib) COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first United States FDA approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant cells. PI3K signaling may lead to the proliferation of malignant cells and is thought to play a role in the formation and maintenance of a supportive tumor microenvironment. COPIKTRA is indicated in the United States for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and has accelerated approval for refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status in the United States and is being investigated in combination with other agents through investigator-sponsored studies. For more information on COPIKTRA, please visit . Information about duvelisib clinical trials can be found on . IMPORTANT SAFETY INFORMATION ABOUT COPIKTRA WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS See full prescribing information for complete boxed warning Fatal and/or serious infections occurred in 31% (4% fatal) of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected. Fatal and/or serious diarrhea or colitis occurred in 18% (<1% fatal) of COPIKTRA-treated patients. Monitor for the development of severe diarrhea or colitis. Withhold COPIKTRA. Fatal and/or serious cutaneous reactions occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Withhold COPIKTRA. Fatal and/or serious pneumonitis occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Monitor for pulmonary symptoms and interstitial infiltrates. Withhold COPIKTRA. INDICATIONS AND USAGE COPIKTRA is a kinase inhibitor indicated for the treatment of adult patients with: Relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior therapies. Relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. Accelerated approval based on overall response rate; continued approval may be contingent upon confirmatory trials. WARNINGS AND PRECAUTIONS Hepatotoxicity: Monitor hepatic function. Neutropenia: Monitor blood counts. Embryo-Fetal toxicity: COPIKTRA can cause fetal harm. Advise patients of potential risk to a fetus and to use effective contraception. ADVERSE REACTIONS The most common adverse reactions (≥20%) are diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain, and anemia. DRUG INTERACTIONS CYP3A inducers: Avoid co-administration with strong CYP3A inducers. CYP3A inhibitors: Monitor for COPIKTRA toxicities when co-administered with strong or moderate CYP3A inhibitors. Reduce COPIKTRA dose to 15 mg twice daily when co-administered with strong CYP3A4 inhibitors. CYP3A substrates: Monitor for signs of toxicities when co-administering COPIKTRA with sensitive CYP3A substrates. USE IN SPECIFIC POPULATIONS Lactation: Advise women not to breastfeed. Please click here to see full Prescribing Information, including Boxed WARNING, for COPIKTRA (duvelisib). About Peripheral T-cell Lymphoma Peripheral T-cell lymphoma (PTCL) is a rare, aggressive type of non-Hodgkin lymphoma (NHL) that develops in mature white blood cells called "T cells" and "natural killer (NK) cells"1 which circulate in the bloodstream and the lymphatic system. PTCL accounts for between 10-15% of all non-Hodgkin lymphomas (NHLs) and generally affects people aged 60 years and older. Although there are many different subtypes of peripheral T-cell lymphoma, they often present in a similar way, with widespread, enlarged, painless lymph nodes in the neck, armpit or groin. There are currently no well-established standards of care for patients with relapsed or refractory disease. To report Adverse Reactions, contact FDA at 1-800-FDA-1088 (1-800-332-1088) or and Secura Bio at 1-800-9SECURA (1-844-973-2872). Related Links Company Codes: LSE:CLIN, OTC-PINK:CGNGY