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77.6% of patients treated with palopegteriparatide in the PaTHway China Trial achieved the primary multi-component endpoint compared to 0.0% for placebo (p <0.0001) Palopegteriparatide was generally safe and well-tolerated, with no discontinuations related to study drug. SHANGHAI, Aug. 12, 2024 /PRNewswire/ -- VISEN Pharmaceuticals, an innovative biopharmaceutical company focused on endocrine diseases, today announced topline data from the 26-week randomized, double-blind, placebo-controlled portion of the Phase 3 PaTHway China Trial of Palopegteriparatide (TransCon PTH) in adults with chronic hypoparathyroidism. A statistically significantly higher proportion of patients treated with palopegteriparatide achieved the primary multi-component endpoint compared to placebo. The primary multi-component endpoint of PaTHway China – defined as serum calcium levels in the normal range (8.3–10.6 mg/dL or 2.07-2.64 mmol/L) and independence from conventional therapy (requiring no active vitamin D and ≤600 mg/day of oral calcium), with no increase in prescribed study drug within the four weeks prior to the Week 26 visit – was achieved by 77.6% of palopegteriparatide-treated patients (45 of 58), compared to 0.0% of patients (0 of 22) in the placebo group (p-value <0.0001). Results were consistent with those announced by Ascendis Pharma for its palopegteriparatide Phase 3 trial. The clinical trial's principal investigator, Professor XIA Weibo, Director of the Department of Endocrinology of Peking Union Medical College Hospital, said: "Hypoparathyroidism is the last endocrine deficiency state, where hormone replacement has not been established as the standard of care. The results of this trial suggest that parathyroid hormone (PTH) replacement therapy can effectively improve symptoms caused by blood calcium fluctuations, and facilitate attainment of normal serum calcium levels without concern for exacerbating hypercalciuria. In fact, treatment with TransCon PTH lowered urine calcium into the normal range. The dual effects of providing physiologic PTH while eliminating reliance on oral calcium supplements and active vitamin D for most treated patients may reduce the risks of long-term complications associated with conventional therapy, including soft-tissue calcifications and decreased kidney function. I believe the PTH replacement therapy could significantly improve the health and quality of life of patients living with hypoparathyroidism." Highlights of the Phase 3 PaTHway China Trial In the trial, 81 patients with hypoparathyroidism were randomized 3:1 to palopegteriparatide or placebo. All patients were on conventional therapy at the start of the trial. Both treatment arms underwent titration of conventional therapy and study drug (palopegteriparatide or placebo) according to an algorithm guided by serum calcium and intended to facilitate independence from conventional therapy. Primary Multi-component Endpoint: 77.6% of palopegteriparatide-treated patients (45 of 58) achieved the primary multi-component endpoint compared to 0.0% of patients (0 of 22) in the placebo group (p-value <0.0001). Other Selected Data: Palopegteriparatide treatment resulted in statistically significant improvement in patient-reported disease-specific physical symptoms compared to placebo, as shown on Hypoparathyroidism Patient Experience Scales (HPES) Symptom-Physical domain scores (p-value = 0.022). At Week 26, 89.7% of palopegteriparatide-treated patients (52 of 58) were able to achieve independence from conventional therapy (requiring no active vitamin D and ≤600 mg/day of oral calcium). Safety Summary: In the trial, palopegteriparatide was generally safe and well tolerated, with no discontinuations related to study drug. No serious TEAEs related to study drug were reported in the palopegteriparatide arm or placebo group. Palopegteriparatide significantly decreased 24-hour urinary calcium compared with placebo group. While palopegteriparatide-treated patients decreased their mean 24-hour urine calcium from 313 mg/day at baseline to 165 mg/day (normal ≤250 mg/day) at Week 26, mean 24-hour urine for placebo-treated patients was 300 mg/day at baseline and 253 mg/day at Week 26. Following an initial blinded 26-week study period of PaTHway China, 76 patients completing the blinded period opted to receive treatment with palopegteriparatide in the ongoing open-label extension portion of the study for up to three years. "When PTH is missing, the body is not able to function normally, and patients may experience a range of severe and potentially life-threatening short-term symptoms and long-term complications." Pony LU, CEO and Executive Director of VISEN Pharmaceuticals, said: "VISEN looks forward to bringing new treatment options for Chinese patients as quickly as possible to help them achieve normal quality of life. These positive results bring hope for a new potential treatment option for adult patient with hypoparathyroidism in China. We plan to file New Drug Application (NDA) to the NMPA as soon as all preparation work is completed." About Palopegteriparatide Palopegteriparatide (TransCon PTH) is an investigational prodrug of PTH (1-34), administered once daily, with sustained release of active PTH designed to provide PTH levels in the physiological range for 24 hours/day. VISEN Pharmaceuticals has been granted exclusive rights to develop, manufacture, and commercialize palopegteriparatide (TransCon PTH) in Greater China by Ascendis Pharma. Palopegteriparatide has been granted marketing authorization as a PTH replacement therapy indicated for the treatment of adults with chronic HP by the European Commission and Medicines and Healthcare products Regulatory Agency. Its NDA for palopegteriparatide for adults with hypoparathyroidism is under review by the U.S. FDA. About VISEN Pharmaceuticals VISEN is an innovative biopharmaceutical company focused on endocrine diseases. We are dedicated to providing innovative therapies and compassionate, patient-centric care, because we believe that achieving better treatment processes and outcomes results in living better lives. Putting patients' need first, VISEN is committed to providing first-in-class or best-in-class products and treatments for endocrine diseases. Our therapeutic areas cover endocrine diseases in adults and children, and rare endocrine diseases. VISEN comprises seasoned professionals with multinational pharmaceutical experiences and leverages cutting-edge technologies and leading resources across the world. We are focused on the Chinese market, and have established offices in Shanghai, Beijing, Hong Kong and Taipei. Our goal is to enable Chinese endocrine patients to benefit from the world's most advanced and reliable treatment solutions earlier. For more information, please visit our website: http://www.visenpharma.com/ Contact us:info@visenpharma.com
HONG KONG, Aug. 5, 2024 /PRNewswire/ -- META Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH). Primary hyperoxaluria is a rare genetic disorder that can lead to kidney stone formation, renal failure, and can be life-threatening in severe cases. The RPDD is intended to facilitate the development of drugs and biologics for serious and life-threatening rare pediatric diseases that affect fewer than 200,000 people in the U.S. and predominantly occur in patients aged 18 years and younger. This designation is pursuant to section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 360ff(a)(3)). About Primary Hyperoxaluria (PH) Primary Hyperoxaluria (PH) is an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body due to defects in enzymes responsible for oxalate metabolism in the liver and other organs. Patients typically present with kidney stones, nephrocalcinosis, renal failure, and oxalate deposition in other organs. Severe cases can lead to end-stage renal disease (ESRD) requiring dialysis, kidney transplantation, or combined liver-kidney transplantation. Symptoms of the disease usually appear at the age of 0 to 3. Without intervention, most patients will develop end-stage renal disease during adolescence, which severely threatens their lives. The incidence of PH is estimated to be 1/58,000, affecting more than 10,000 people in the United States and the European Union, and more than 20,000 people in China. Currently, there is no cure for primary hyperoxaluria. Existing treatments are primarily supportive care, including increased fluid intake to dilute oxalate in the urine, and medications such as pyridoxine (vitamin B6) to reduce oxalate production. However, patients' symptoms and disease progression cannot be effectively controlled. About META-001-PH META-001-PH is a groundbreaking small molecule drug developed by META for the treatment of primary hyperoxaluria. This innovative molecule is the product of a collaborative effort between META's chemistry team and XtalPi's (2228.HK) AI drug discovery team, who worked together from the initial scaffold screening to the preclinical compound nomination. XtalPi's automated robotic chemical synthesis lab was responsible for the chemical synthesis of the related molecule series, ensuring a precise and efficient production process. Preclinical experiments in animal disease models have shown that META-001-PH can significantly reduce urinary oxalate excretion by up to 80%. While existing therapeutic agents are unable to effectively control urinary oxalate levels in the long term, META-001-PH, administered orally daily, can maintain oxalate at normal levels, thus demonstrating the potential for better long-term control of kidney stone formation in patients with PH. META-001-PH has also demonstrated good tolerability and safety in preclinical animal models and is undergoing IND-enabling toxicology studies with a clinical Phase I safety assessment on healthy subjects planned for 2025H1 in Australia. This collaboration between META and XtalPi marks a significant advancement in the development of effective treatments for primary hyperoxaluria. About Rare Pediatric Disease Designation (RPDD) The Rare Pediatric Disease Designation (RPDD) is an eligibility determination for rare pediatric diseases that affect fewer than 200,000 patients and pose a serious life-threatening risk to children under the age of 18. The RPDD and Priority Review Voucher (PRV) program aims to recognize the significant need for therapies for rare pediatric diseases and to encourage the development of new treatments for these serious or life-threatening conditions. Under the program, sponsors will be eligible to receive a PRV upon approval of a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease. PRVs can be used for any subsequent product approval application, reducing the review time by 4-6 months, or can be traded with an average transfer price of more than $100 million in recent years. About META Pharmaceuticals Inc. META Pharmaceuticals Inc. (META) is an innovative biopharmaceutical company dedicated to discovering and developing urgently needed breakthrough treatments for a broad range of autoimmune disorders, metabolic diseases, and cancer. As the only company in the Asia Pacific Region leveraging the groundbreaking theory of immuno-metabolism, META leads the way in creating safer and more effective therapies that modulate cellular metabolism to regulate immune system function and other pathways. META has developed two distinct chemical series targeting META-001 for the treatment of autoimmune diseases and primary hyperoxaluria, respectively, which hold the potential to become the next-generation oral treatments for these conditions, addressing significant unmet clinical needs. META Pharmaceuticals Inc. is jointly incubated by XtalPi (2228.HK), the leader in the AI-pharmaceutical industry, Forcefield Ventures, and IMO Ventures, with investments from Tiantu Capital (1973.HK), Yael Capital, Fangyuan Capital, Lead Rich International, Decent Capital, and Bopu Capital. For more information, please visit http://en.metabiopharma.com/ and follow us on LinkedIn. Contact: contact@metabiopharma.com
TAIPEI, Aug. 5, 2024 /PRNewswire/ -- Taiwan-based Formosa Pharmaceuticals ("Formosa", 6838.TWO) announced today that the company has entered into an exclusive licensing agreement with Apotex Inc. ("Apotex"), for exclusive rights in Canada to the commercialization of clobetasol propionate ophthalmic suspension, 0.05% (APP13007), a patented innovative medicine for the treatment of inflammation and pain following ocular surgery. Clobetasol propionate ophthalmic suspension, 0.05% (APP13007) was approved by the U.S. Food and Drug Administration (FDA) on March 4, 2024. The licensing deal will include certain payments, including an upfront payment and milestone payments upon achievement of certain regulatory and sales milestones. APP13007's active ingredient is the superpotent corticosteroid, clobetasol propionate, and is derived from Formosa Pharma's proprietary APNT® nanoparticle formulation platform. The novel formulation enables a convenient and straightforward dosing regimen (twice daily for 14 days) while providing rapid and sustained relief of inflammation and pain, which in Phase 3 trials proved statistically and clinically superior to its matching placebo (p<0.001). APP13007 will enter a ~$C50M market for topical ophthalmic steroids and steroid combinations, growing at around 5% annually, driven by an estimated ~500,000 ocular surgeries annually in Canada. "Formosa Pharma is honored to partner with Apotex, with its storied history and reputation for success," said Erick Co, President and CEO of Formosa Pharmaceuticals. "Their commitment to branded ophthalmology products gives us full confidence that APP13007 will flourish in the Canadian market. We look forward to working with the Apotex team in providing this differentiated therapy to Canadian physicians and patients." "We are pleased to provide Canadian patients with APP13007, a new treatment option for post-operative inflammation and pain following ocular surgery," said Allan Oberman, President & CEO, Apotex. "Expanding our portfolio of innovative medicines, including our ophthalmic portfolio, along with our commercial infrastructure, enables us to support more patients along their journey to health." About Formosa Pharmaceuticals, Inc. Formosa Pharmaceuticals, Inc. (6838.TWO) is a clinical stage biotechnology company with primary focus in the areas of ophthalmology and oncology. The company's proprietary nanoparticle formulation technology (APNT®), through which APP13007 was developed, improves the dissolution and bioavailability of APIs for topical, oral, and inhaler administration. Resulting formulations have high uniformity, purity, and stability, thereby allowing the utilization of poorly soluble or extremely potent drug agents which otherwise may face insurmountable challenges in delivery and penetration to target tissues. For more details about Formosa Pharma and APNT®, visit www.formosapharma.com. About Apotex Inc. Apotex is a Canadian-based global health company. We improve everyday access to affordable, innovative medicines and health products for consumers worldwide, with a broad portfolio of generic, biosimilar, and innovative branded pharmaceutical products. Headquartered in Toronto, with regional offices globally, including in the United States, Mexico and India, we are the largest Canadian-based pharmaceutical company and a health partner of choice for the Americas for pharmaceutical licensing and product acquisitions. Learn more about us at www.apotex.com.
TEL-AVIV, Israel, July 30, 2024 /PRNewswire/ -- Neurim Pharmaceuticals ("Neurim") announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending an extension to the existing indication to include the treatment of Insomnia in children with Neurogenetic disorders (NGDs). Slenyto® is a child appropriate prolonged release melatonin formulation that was approved in the EU in 2018 for the treatment of insomnia in children with autism spectrum disorder (ASD) and/ or Smith-Magenis Syndrome. Slenyto® is the only authorised medication for insomnia in these disorders. The adopted recommended wording for the extended indication is: "Slenyto® is indicated for the treatment of insomnia in children and adolescents aged 2-18 with autism spectrum disorder (ASD) and/or neurogenetic disorders with aberrant diurnal melatonin secretion and /or nocturnal awakenings, where sleep hygiene measures have been insufficient". "The positive CHMP opinion on Slenyto® represents a significant milestone in addressing an unmet need for children with NGDs, who suffer severely from impaired sleep. Upon approval, Slenyto® would be the only medicinal product approved for sleep disorders in this unique paediatric population, allowing treatment initiation at an early age," said Prof. Nava Zisapel, CEO of Neurim Pharmaceuticals. Paul Gringras, Professor of paediatric Sleep Medicine, London, England commented: "Melatonin deficiency or misalignment is a common pathophysiological mechanism for insomnia in neurogenetic disorders. Slenyto® is a prolonged-release formulation of melatonin that delivers melatonin over the course of the night, mimicking the endogenous release profile in healthy subjects. Therefore, Slenyto® has the potential to treat the pathophysiology of insomnia in this population improving sleep latency, sleep continuity and total nighttime sleep duration. Slenyto® has been specifically formulated for use in children and has a favorable safety profile. The treatment will address a significant need in the medical management of many children and adolescents with NGDs". ABOUT INSOMNIA IN CHILDREN WITH NEUROGENETIC DISORDERS (NGDs) Sleep impairment is a common comorbidity in neurogenetic disorders, with up to 86% of children reported to experience disrupted sleep, starting at an early age. During early childhood, sleep plays an essential role in healthy cognitive and psychosocial brain development, influencing learning memory, emotional regulation, and brain structure. Children with neurogenetic disorders tend to develop insomnia and other sleep disorders at an early age. Insomnia has a significant impact on these children, aggravating disease symptoms and reducing quality of life in both patients and caregivers. Current practices recommend parent-directed behavioral sleep interventions as first-line treatment for pediatric insomnia in NGDs, with a 25% response rate. Slenyto® will be the first approved pharmacological treatment for insomnia in children and adolescents with NGDs. ABOUT SLENYTO® Slenyto® is an age-appropriate prolonged-release formulation of melatonin, a hormone involved in the regulation of circadian clock and sleep. The mini-tablet innovative formulation was specifically designed to facilitate ease of swallowing without resistance in pediatric ASD and NGD population, in response to the unmet medical need in the field of paediatric insomnia. Slenyto® delivers melatonin over the night to mimic the endogenous melatonin release profile in healthy subjects. In a Phase III study in ASD and SMS children and adolescents, Slenyto® improved sleep latency, sleep continuity and total nighttime sleep duration. The positive effect on sleep maintenance and duration was associated with an improvement in externalizing behavior, which correlated with an improvement in parents' well-being. The mechanism of action of Slenyto® on sleep latency, sleep maintenance and total sleep time is independent of the background disorder. Therefore, any patient with a neurogenetic disorder with sleep disturbances associated with aberrant diurnal melatonin secretion patterns and/or insufficient nighttime melatonin secretion will benefit from Slenyto®. ABOUT NEURIM PHARMACEUTICALS Neurim Pharmaceuticals Ltd. (www.neurim.com) is a neuroscience drug discovery and development company. Its first approved drug, CIRCADIN®, is approved for patients over the age of 55 who are suffering from insomnia and is commercially available in 45 countries around the world, including Europe, Asia-Pacific, Latin America, Africa and the Middle East. Neurim has a strong and innovative product pipeline targeting central nervous system (CNS) disorders. Contact: Guy ManorVP Commercial Operationsguym@neurim.com
Obese and overweight participants receiving bi-weekly doses of 12 mg, 18 mg, 24 mg, 48 mg, and once-weekly dose of 24 mg GZR18 for 30 weeks achieved mean percent changes in body weight from baseline of -11.15%, -13.22%, -14.25%, -17.29%, and -17.78%, respectively, with the placebo group at -0.99%; and at 30 weeks, participants' body weight continued to decrease. The clinical efficacy and safety of bi-weekly 48 mg and once-weekly 24 mg GZR18 injections were comparable, with no significant difference in mean percent change in body weight from baseline between the two groups (one-sided test, P > 0.025). GZR18 injections were safe and well tolerated, with the most commonly reported adverse events being gastrointestinal reactions, comparable to similar drugs. BEIJING, July 22, 2024 /PRNewswire/ -- Recently, Gan & Lee Pharmaceuticals(Gan & Lee, Shanghai Stock Exchange: 603087) announced that its independently developed long-acting GLP-1 receptor agonist (GLP-1 RA), GZR18 injection, has achieved positive results in a clinical trial for adults with obesity/overweight in China. Statement: 1. GZR18 injection is an investigational drug and has not yet been approved in China. 2. Gan & Lee Pharmaceuticals does not recommend the use of any unapproved drugs/indications. The phase IIb clinical trial (CTR20231695) is a multi-center, randomized, double-blind, placebo-controlled study conducted at 25 clinical trial centers in China, recruiting a total of 340 participants. The participants were overweight (BMI≥24 kg/m2) with at least one weight-related comorbidity or obese (BMI≥28 kg/m2) adults with poorly controlled diet and exercise. They were randomized to receive bi-weekly (Q2W) doses of 12 mg, 18 mg, 24 mg, 48 mg, and once-weekly (QW) dose of 24 mg GZR18 injection or placebo for 30 weeks (including a dose escalation period). The primary efficacy endpoint of the study was the percent change in body weight from baseline after 30 weeks of treatment. The study assessed changes from baseline in average weight, waist circumference, waist-to-hip ratio, body mass index (BMI), glycemic parameters, and the safety and tolerability of the drug. After 30 weeks of treatment, compared to the placebo group, participants receiving different doses and frequencies of GZR18 injection (12 mg, 18 mg, 24 mg, and 48 mg Q2W; 24 mg QW) had a significant reduction in the percent change in body weight from baseline. The mean percent changes in body weight from baseline was -11.15% (12 mg group, Q2W), -13.22% (18 mg group, Q2W), -14.25% (24 mg group, Q2W), -17.29% (48 mg group, Q2W), and -17.78% (24 mg group, QW), all more effective than the placebo group (reduced by 0.99%). There was no significant difference in mean percent change in body weight between the 48 mg Q2W group and the 24 mg QW group (one-sided test, P > 0.025). In this study, the bi-weekly GZR18 injections were generally safe and well tolerated, consistent with the known safety signals of GLP-1 receptor agonists. The most common adverse events were gastrointestinal reactions, most of which were mild to moderate in severity, comparable to other GLP-1 RAs. The comprehensive results of this phase IIb study are planned to be announced later this year and will be published in a peer-reviewed journal. About Gan & Lee Gan & Lee Pharmaceuticals developed the first Chinese domestic insulin analog. Currently, Gan & Lee has six core insulin products, including five insulin analog varieties: long-acting glargine injection (Basalin®), fast-acting lispro injection (Prandilin™), fast-acting aspart injection (Rapilin®), mixed protamine zinc lispro injection (25R) (Prandilin™25), aspart 30 injection (Rapilin®30), and one human insulin injection - mixed protamine human insulin injection (30R) (Similin®30). The company has two approved medical devices in China, namely reusable insulin injection pen (GanleePen), and disposable pen needle (GanleeFine®). In China's 2024 National Insulin-Specific Centralized Procurement, Gan & Lee Pharmaceuticals ranked second overall and first among domestic companies in terms of procurement demand for insulin analogs. The company is also making strides in international markets, with the disposable pen needle (GanleeFine®) approved by the US Food and Drug Administration (FDA) in 2020 and received GMP inspection approval from the European Medicines Agency (EMA) in 2024. These achievements significantly boost Gan & Lee's competitiveness in both international and domestic markets. In the future, Gan & Lee will strive for comprehensive coverage in diabetes treatment. Moving forward with its mission to become a world-class pharmaceutical company, Gan & Lee will also actively develop new chemical entities and biological drugs, focusing on treatments for metabolic diseases, cardiovascular diseases, and other therapeutic areas. Further Information: BPRD@ganlee.com (Media) BD@ganlee.com (Business Development)
MELBOURNE, Australia, July 18, 2024 /PRNewswire/ -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today provides an update on its revenue and operational performance for the quarter ended 30 June 2024 (Q2 2024). Q2 2024 Financial Performance and Guidance Upgrade The Company reports unaudited total revenue of approximately US$124M[1] (AU$189M) primarily generated from sales of Telix's prostate cancer imaging product Illuccix®. This represents an increase of 55% on the prior corresponding quarter (Q2 2023: US$80M or AU$120M) and an increase of 8% on the previous quarter (Q1 2024: US$115M or AU$175M). Revenue generated from sales of Illuccix® in the United States (U.S.) was approximately US$121M (AU$184M, Q2 2023: US$78M or AU$116M). On the basis of these results, the Company has upgraded revenue guidance for FY2024 which is now expected to be in the range of US$490M to US$510M (AU$745M to AU$776M at current exchange rates). This represents an approximate increase of 48% to 54% on 2023 revenue. Prior guidance was ranged at US$445M to US$465M[2] (AU$675M to AU$705M). Dr Christian Behrenbruch, Managing Director and Group Chief Executive Officer said, "We have continued to deliver excellent quarterly growth in both revenue and dose volume sales of Illuccix. We have leveraged our unrivalled scheduling flexibility and clinical differentiation, to increase our market share and minimise the impact of new entrants." Revenue guidance is based on approved products in jurisdictions with a marketing authorisation[3]. Telix reaffirms guidance for R&D expenditure, which remains at an expected 40-50% increase compared with 2023, funded by earnings. The above guidance is based on expected global and domestic economic conditions and is subject to known and unknown risks, uncertainties and other factors that may cause our actual results to differ materially. As such, investors are cautioned not to place undue reliance on this guidance and in particular Telix cannot guarantee a particular result. In compiling financial forecasts, a number of key variables that may have a significant impact on guidance have been identified and are included below as a footnote[4]. The Company will release its interim report and Appendix 4D for the six months ended 30 June 2024 on 22 August 2024. Q2 2024 Operational Highlights Telix continued to progress its extensive theranostic pipeline during the quarter. Highlights include positive data from prostate cancer therapy programs: TLX591 (177Lu rosopatamab tetraxetan): Telix reported a positive efficacy signal from the ProstACT SELECT trial of TLX591, its lead investigational radio antibody-drug conjugate (rADC) in prostate cancer. The median radiographic progression-free survival (rPFS) of 8.8 months compares favourably to small molecule radioligand therapy (RLT) agents at a similar stage of development and builds on earlier results from ProstACT SELECT confirming the favourable safety profile and clinical utility of the short, two-dose treatment regimen. TLX591 is being further evaluated in the Phase III ProstACT GLOBAL trial. The study received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) during the quarter. Multiple sites across the U.S. are now being activated and preparing to dose first patients. Patient recruitment continues at sites in Asia Pacific. TLX592 (225Ac-RADmAb®): Successful proof-of-concept study completed for targeted alpha therapy in prostate cancer. The initial results demonstrated Telix's proprietary RADmAb engineered antibody is rapidly eliminated from the blood circulation and cleared via the liver, desirable characteristics for use with alpha emitting radioisotopes. Based on the outcomes of this trial, the program will progress to a Phase I/II therapeutic trial with Actinium 225. Telix is progressing multiple marketing authorisation applications across its precision medicine (diagnostic) portfolio, including: New Drug Application (NDA) submission for new prostate cancer imaging agent TLX007-CDx (PSMA-PET imaging[5]): Telix submitted a NDA to the FDA for a new PSMA targeting prostate cancer imaging agent designed to expand the availability, distribution and scheduling flexibility for PSMA-PET imaging[6]. The FDA is expected to confirm by the end of July 2024 whether the submission is accepted for review. Confirmation of the PDUFA[7] goal date (review timeframe) is expected in early August 2024, in line with FDA procedure. Kidney cancer imaging agent TLX250-CDx (Zircaix®[8], 89Zr-DFO-girentuximab) regulatory filing completed: The Biologics License Application (BLA) submission to the FDA for kidney cancer imaging has now been completed[9]. The Company is expecting confirmation of the PDUFA goal date from the FDA by end of July 2024. Concurrently the expanded access and compassionate use programs for TLX250-CDx continue to dose patients across the U.S., Australia and Europe in a significant area of unmet patient need. Separately, a manuscript reporting the final results from the pivotal Phase III ZIRCON study is in the late stages of peer-review for publication in a leading medical journal. Brain cancer imaging agent TLX101-CDx (Pixclara™8, 18F-floretyrosine or 18F-FET) regulatory package: Telix participated in a pre-NDA meeting with the FDA on 12 June 2024, with the purpose of consulting on the final package and likely scope of label indications, including alignment on the final clinical package. On the basis of this positive discussion the Company is now finalising its NDA for submission during Q3 2024. European Union (EU), United Kingdom (UK) and Brazil regulatory filings for Illuccix®: The marketing authorisation applications for the EU and the UK are continuing to progress. All regulator questions raised in the EU marketing authorisation application within the standard clock stop period have been addressed and no substantive issues have been identified. The Company will communicate the final decision date once set by the competent authority, the German Federal Institute for Drugs and Medical Devices (Bundesinstitut für Arzneimittel und Medizinprodukte, BfArM). Telix is expecting to receive a regulator's assessment report by the end of July 2024 from the UK Medicines & Healthcare Products Regulatory Agency (MHRA), which is expected to include final questions and a timeline for the remainder of the review. The marketing authorisation application in Brazil with Telix's partner Grupo GSH is in the final stages of review with an approval decision anticipated from the Brazilian Health Regulatory Agency (Agencia Nacional de Vigilancia Sanitaria, ANVISA) during Q3 2024 based on current information. Subsequent to the end of Q2 2024, the U.S. Centers for Medicare & Medicaid Services (CMS) announced proposed changes for the Hospital Outpatient Prospective Payment System (OPPS) rule, which would improve payments to Medicare patients for diagnostic radiopharmaceuticals in the U.S., including Illuccix® and future Telix diagnostic products, if approved[10]. Telix welcomes the proposal to keep the payment for the diagnostic radiopharmaceuticals separate from the nuclear medicine test (scan) after transitional pass-through payment status expires, which will facilitate continued patient access[11]. The proposal is now in a 60-day comment period, with a final rule to be issued in early November 2024 and take effect 1 January 2025. About Telix Pharmaceuticals Limited Telix is a biopharmaceutical company focused on the development and commercialisation of therapeutic and diagnostic radiopharmaceuticals and associated medical devices. Telix is headquartered in Melbourne, Australia, with international operations in the United States, Europe (Belgium and Switzerland), and Japan. Telix is developing a portfolio of clinical and commercial stage products that aims to address significant unmet medical needs in oncology and rare diseases. Telix is listed on the Australian Securities Exchange (ASX: TLX). Telix's lead imaging product, gallium-68 (68Ga) gozetotide injection (also known as 68Ga PSMA-11 and marketed under the brand name Illuccix®), has been approved by the U.S. Food and Drug Administration (FDA)[12], by the Australian Therapeutic Goods Administration (TGA) [13], and by Health Canada[14]. No other Telix product has received a marketing authorisation in any jurisdiction. Visit www.telixpharma.com for further information about Telix, including details of the latest share price, announcements made to the ASX, investor and analyst presentations, news releases, event details and other publications that may be of interest. You can also follow Telix on X and LinkedIn.y ariables that could cause actual results to differ materially include: the success and timing of research and devel Telix Investor Relations Ms. Kyahn WilliamsonTelix Pharmaceuticals LimitedSVP Investor Relations and Corporate CommunicationsEmail: kyahn.williamson@telixpharma.com This announcement has been authorised for release by the Board of Telix Pharmaceuticals Limited. Legal Notices The information contained in this announcement is not intended to be an offer for subscription, invitation or recommendation with respect to shares of Telix Pharmaceuticals Limited (Telix) in any jurisdiction, including the United States. No representation or warranty, express or implied, is made in relation to the accuracy or completeness of the information contained or opinions expressed in the course of this announcement. The information contained in this announcement is subject to change without notification. This announcement may contain forward-looking statements that relate to anticipated future events, financial performance, plans, strategies or business developments. Forward-looking statements can generally be identified by the use of words such as "may", "expect", "intend", "plan", "estimate", "anticipate", "outlook", "forecast" and "guidance", or other similar words. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. Forward-looking statements are based on the Company's good-faith assumptions as to the financial, market, regulatory and other risks and considerations that exist and affect the Company's business and operations in the future and there can be no assurance that any of the assumptions will prove to be correct. In the context of Telix's business, forward-looking statements may include, but are not limited to, statements about: the initiation, timing, progress and results of Telix's preclinical and clinical studies, and Telix's research and development programs; Telix's ability to advance product candidates into, enrol and successfully complete, clinical studies, including multi-national clinical trials; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities; the commercialisation of Telix's product candidates, if or when they have been approved; estimates of Telix's expenses, future revenues and capital requirements; Telix's financial performance; developments relating to Telix's competitors and industry; and the pricing and reimbursement of Telix's product candidates, if and after they have been approved. Telix's actual results, performance or achievements may be materially different from those which may be expressed or implied by such statements, and the differences may be adverse. Accordingly, you should not place undue reliance on these forward-looking statements. You should read this announcement together with our risk factors, as disclosed in our most recently filed reports with the ASX or on our website. To the maximum extent permitted by law, Telix disclaims any obligation or undertaking to publicly update or revise any forward-looking statements contained in this announcement, whether as a result of new information, future developments or a change in expectations or assumptions. ©2024 Telix Pharmaceuticals Limited. The Telix Pharmaceuticals®, Illuccix®, Pixclara™8 and Zircaix®8 names and logos are trademarks of Telix Pharmaceuticals Limited and its affiliates – all rights reserved. [1] Conversion to AUD$ is at an average exchange rate realised during Q2 2024 of AUD$1 = US$0.658 [2] Telix ASX disclosures 22 February and 17 April 2024. [3] Illuccix® has received a marketing authorisation in Australia, Canada and the U.S. [4] Key variables that could cause actual results to differ materially include: the success and timing of research and development activities; decisions by regulatory authorities regarding approval of our products as well as their decisions regarding label claims; competitive developments affecting our products; the ability to successfully market new and existing products; difficulties or delays in manufacturing; trade buying patterns and fluctuations in interest and currency exchange rates; legislation or regulations that affect product production, distribution, pricing, reimbursement, access or tax; acquisitions and divestitures; research collaborations; litigation or government investigations; and Telix's ability to protect its patents and other intellectual property. [5] Imaging of prostate-specific membrane antigen with positron emission tomography. [6] Telix ASX disclosure 27 May 2024. [7] Prescription Drug User Fee Act. [8] Brand name subject to final regulatory approval. [9] Telix ASX disclosure 3 June 2024. [10] CMS Press Release 10 July 2024. [11] Telix ASX disclosure 11 July 2024. [12] Telix ASX disclosure 20 December 2021. [13] Telix ASX disclosure 2 November 2021. [14] Telix ASX disclosure 14 October 2022.
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