Von Willebrand Diseases

President Ronald Reagan recognized March as “National Hemophilia Month” in 1986. BioTek reMEDys has identified Bleeding Disorders Awareness Month (BDAM) as an opportunity to drive change, piggybacking on the national event to call for awareness of safe medication use in patients with the conditions. The specialty pharmacy organization serves patients with rare conditions including bleeding disorders. Because of that role, BioTek reMEDys is “well aware of the critical importance of education—for patients, loved ones, caregivers and providers, as well as pharmacists, to make sure that those living with bleeding disorders get the specific treatments they need,” CEO Chaitanya Gadde said in a statement.  BioTek reMEDys provides therapy-specific patient education materials to support patients, the company said, but has identified a broader need for awareness of safe medication use and treatments. Gadde discussed the role of BDAM in getting the message out.  “Bleeding Disorders Awareness Month is an opportunity and a reminder to advocate for education as a priority throughout the healthcare continuum as we work towards recognizing the challenging journey patients often have on the way to a correct diagnosis and treatment,” Gadde said. The awareness month has a long history. President Ronald Reagan recognized March as “National Hemophilia Month” in 1986. Thirty years later, the initiative evolved into Bleeding Disorders Awareness Month, expanding the focus beyond hemophilia to include von Willebrand disease, rare factor disorders and other conditions. Since then, a wide range of groups, from Genentech to the state of North Dakota, have acknowledged the month and tried to use it to raise awareness of bleeding disorders and the needs of people who live with them.

Strong CSL Behring portfolio growth especially Ig FINANCIAL HIGHLIGHTS 4 Revenue $8.05 billion, up 11% at CC3 NPAT $1.90 billion1, up 17% NPAT $1.94 billion1 at CC3, up 20% NPATA $2.02 billion1,2 , up 11% NPATA $2.06 billion1,2 at CC3, up 13% NPATA1,2 earnings per share $4.182, up 11% NPATA1,2 earnings per share $4.26 at CC3 up 13% Interim dividend 5 of US$1.19 per share Converted to Australian currency, the interim dividend is approximately A$1.81 per share, up 12% Guidance reaffirmed – FY24 NPATA2,4 anticipated to be in the range of approximately $2.9 billion to $3.0 billion2 at CC3 MELBOURNE, Australia, Feb. 13, 2024 /PRNewswire/ -- CSL Limited (ASX:CSL; USOTC:CSLLY) today announces a reported net profit after tax of $1.90 billion1 for the 6 months ended 31 December 2023, up 20% on a constant currency basis3. Underlying profit (NPATA) was $2.02 billion1,2, up 13% on a constant currency basis to $2.06 billion1,2,3. Dr. Paul McKenzie, CSL's Chief Executive Officer and Managing Director said, "Our strong first-half result for the 2024 financial year was driven by CSL Behring's exceptional performance across its portfolio, especially immunoglobulins. The plasma initiatives we have implemented are starting to drive gross margin recovery. "CSL Seqirus achieved solid growth in a challenging season. Its portfolio of differentiated products outperformed the market. "For CSL Vifor we are well prepared for the transitioning iron market." PERFORMANCE CSL Behring Total revenue was $5,238 million, up 14%3 when compared to the prior comparable period. Immunoglobulin (Ig) product sales of $2,757 million, increased 23%3 with strong growth recorded across all geographies driven by global plasma supply and patient demand. PRIVIGEN® / INTRAGRAM® (Immune Globulin Intravenous (Human), 10% Liquid) sales grew 27%3 as the momentum from the prior year continued in improving product availability and patient diagnosis rates. HIZENTRA® (Immune Globulin Subcutaneous (Human), 20% Liquid) sales were up 18%3 driven by patient diagnosis rates. HIZENTRA® continues to be the clear market leader for subcutaneous immunoglobulin. Underlying demand for Ig continues to be strong due to significant patient needs in core indications – namely Primary Immune Deficiency, Secondary Immune Deficiency and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Albumin sales of $613 million, were up 8%3. Sales were strong in emerging markets with solid growth in the US and Europe. Growth in China was modest, tempered by competitive pressure. Haemophilia product sales of $662 million increased 8%3. IDELVION®, CSL Behring's novel long-acting recombinant factor IX product achieved growth of 7%3 and continues to be the market leader in key markets. HEMGENIX®, the first and only gene therapy for haemophilia B was successfully launched in the US in FY23 and patient referrals have been accelerating. The haemophilia A market continued to be competitive resulting in a modest decline in sales for AFSTYLA®, a novel recombinant factor VIII product. Plasma-derived haemophilia products, however, achieved growth of 8% driven by HUMATE® / HAEMATE®, therapies for the treatment of patients with von Willebrand disease. Specialty products sales of $976 million, were up 6%3 led predominately by demand for KCENTRA® and HAEGARDA®. KCENTRA® (4 factor prothrombin complex concentrate) recorded sales growth of 12%3, as it continues to further penetrate the warfarin reversal market in the US. HAEGARDA®, our therapy for patients with Hereditary Angioedema, increased 9%, driven by the continued shift from on-demand to prophylaxis treatment and a strong performance in the UK and Europe. Garadacimab (Anti-FXIIa) for HAE, was filed for regulatory approval in the US and EU. Plasma Collections Plasma collections remain strong. The cost of collections, which includes donor compensation and labour, continued to trend down. A new roll out plan for the RIKA plasmapheresis devices was developed. Deployment across the US fleet is expected over the next 18 months. In addition, results from an individualised nomogram trial conducted by our supplier have been submitted for regulatory approval. CSL Seqirus Total revenue of $1,804 million, was up 2%3 driven by the adjuvanted influenza vaccine FLUAD®, which increased by 14%3. This growth was achieved against a backdrop of reduced rates of immunisation and highlights the strength of CSL Seqirus' differentiated product portfolio. During the period: Self-amplifying mRNA vaccine for COVID was approved by Japan's Ministry of Health, Labour and Welfare aQIVc, a next generation influenza vaccine combining adjuvant technology with cell-based manufacturing, enrolled its last patient in the Phase III clinical study in January 2024. CSL Vifor Total revenue was $1,011 million. The prior comparable period included only 5 months revenue following the acquisition of Vifor Pharma in August 2022. During the period: Preparations were made for the transitioning iron market There was strong performance from the long-acting erythropoiesis-stimulating agent, MIRCERA® TAVNEOS® was successfully launched in multiple European countries While the strategic potential of the business remains strong, we have dampened our near-term growth aspirations for CSL Vifor. Expense Performance Research and development (R&D) expenses were $669 million8 , up 11%3 when compared to the prior comparable period. The increase in expenses reflects higher costs associated with the progression of the R&D portfolio and investment in R&D infrastructure. Selling and marketing expenses (S&M) were $707 million8, up 2%3 in comparison to the prior comparable period. An additional month of CSL Vifor and an increase in labour costs accounts for the increase in S&M expenses while other S&M expenses were held in line with the prior comparable period. General and administrative (G&A) expenses were $323 million8, down 7%3 due to favourable foreign exchange differences and efficiencies generated from the centralisation of the group's Enabling Functions. Depreciation and amortisation (D&A) expense (excluding acquired intellectual property) was $297 million, up 1%3. Net finance costs were $234 million9, up 32%3. The increase in net finance costs was due to the debt associated with the acquisition of Vifor Pharma and higher interest rates. Financial position Cashflow from operations was $1,069 million, up 9%. The increase was driven by higher profitability and overall growth in sales. This was partly offset by higher payments for income tax and interest. Cash outflow from investing was $702 million, down significantly when compared to the prior comparable period as payment for the acquisition of Vifor Pharma was made in the prior period. CSL's balance sheet remains in a strong position with net assets of $19,162 million. Current assets increased by 10% to $10,146 million. The main driver was an increase in receivables due to the increase in sales and the seasonality of CSL Seqirus. Non-current assets increased by 1% to $27,158 million in comparison to the previous year. Current liabilities increased by 2% to $4,718 million. The increase in interest-bearing liabilities and borrowings (bank debt) was offset by the decrease in trade and other payables and current tax liabilities. Non-current liabilities decreased by 3% to $13,424 million. The decrease was due to the reclassification of certain bank borrowings as current, coupled with repayment across the Group's debt portfolio including the private placement senior notes. Outlook (at FY23 exchange rates) Commenting on CSL's outlook, Dr. McKenzie said, "For FY24, I am pleased to reaffirm our previous guidance. CSL's underlying profit, NPATA is expected be in the range of approximately $2.9 billion to $3.0 billion at constant currency3, representing growth over FY23 of approximately 13-17%[6]" "CSL is in a strong position to deliver annualised double-digit earnings growth over the medium term. "The strong growth in our immunoglobulins franchise is expected to continue as patient demand remains strong. "We have a number of initiatives underway in plasma collections that are improving efficiencies and processing times, supporting continued expansion in CSL Behring's gross margin. "Our transformational gene therapy product for haemophilia B patients, HEMGENIX®, is attracting significant interest from patients and health care professionals and patient referrals have accelerated. We expect more patients dosed in the second half of this financial year. "CSL Seqirus has performed well in a challenging season. However, due to the seasonality of this business we anticipate it to post a loss in the second half of the fiscal year. "For CSL Vifor, we are operating within an evolving iron market. While there are challenges for near-term growth, we are well positioned for iron competition in the EU and further geographic expansion. Our focus remains on unlocking value by leveraging capabilities across the CSL group", Dr. McKenzie concluded. In compiling the company's financial forecasts for FY24, a number of key variables that may have a significant impact on guidance have been identified and these have been included in the endnote[7]. FURTHER INFORMATION Additional details about CSL's results are included in the company's 4D statement, investor presentation slides and webcast, all of which can be found on CSL's website A glossary of medical terms can also be found on the website. 1 Attributable to CSL shareholders 2 Statutory net profit after tax (NPAT) before impairment and amortisation of acquired intellectual property, business acquisition and integration costs and the unwind of the inventory fair value uplift. 3 Constant currency (CC) removes the impact of exchange rate movements, facilitating the comparability of operational performance. For further detail refer to CSL's Financial Statements for the Half Year ended December 2023 (Directors Report). 4 All figures are expressed in US dollars unless otherwise stated. 5 For shareholders with an Australian registered address, the interim dividend of US$1.19 per share (approximately A$1.81) is expected to be paid on 3 April 2024. For shareholders with a New Zealand registered address the interim dividend of US$1.19 per share (approximately NZ$1.94) is expected to be paid on 3 April 2024. The exchange rates will be fixed at the record date of 12 March 2024. All other shareholders will be paid in US$. CSL also offers shareholders the opportunity to receive dividend payments in US$ by direct credit to a US bank account. 6 % growth rates excludes the one-off gain from the sale of property in FY23 (NPATA $44m). 7 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; ability to collect plasma; 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 CSL's ability to protect its patents and other intellectual property. 8 Underlying results are adjusted to exclude amortisation of acquired intellectual property ($132 million), business acquisition and integrations costs and the unwind of the inventory fair value uplift. 9 At reported currency For further information, please contact: Investors: Bernard Ronchi Director, Investor Relations CSL Limited P: +61 3 9389 3470 E: [email protected] Stephen McKeon Director, Investor Relations CSL Limited P: +61 402 231 696 E: [email protected] Media: Jimmy Baker Communications CSL Limited P: +61 450 909 211 E: [email protected] SOURCE CSL Limited

HMB-001 was well tolerated with no drug-related adverse or thromboembolic events; dose-dependent improvements in blood-clotting activity support the potential of HMB-001 as a first-in-class prophylactic treatment for Glanzmann Thrombasthenia and other blood clotting disorders Preclinical HMB-001 findings were published in the February 2024 issue of Nature Cardiovascular Research COPENHAGEN, Denmark and CAMBRIDGE, Mass., Feb. 9, 2024 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today presented initial results from Phase 1 of its ongoing evaluation of HMB-001. A novel bispecific antibody, HMB-001 is in development as a prophylactic treatment for the bleeding disorder Glanzmann Thrombasthenia (Glanzmann). The data were presented as one of 10 abstracts selected for the SLAM oral presentation session at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) held this week in Frankfurt, Germany. "The first clinical data for HMB-001 in Glanzmann suggest that the demonstrated mechanism is suitable as a new prophylactic treatment for people with neglected blood clotting disorders who face severe, potentially life-threatening bleeds every day," said Joe Vogel, Senior Director and Program Lead for HMB-001 at Hemab. "With Phase 2 already underway, we are committed to advancing clinical evaluation of HMB-001 toward bringing life-changing preventative treatments to improve patients' quality of life." The Phase 1/2 first-in-human open-label study in patients with Glanzmann is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of HMB-001. As part of the UK-based Phase 1 single-ascending dose, seven patients received subcutaneous HMB-001 at 0.2 mg/kg, (n=1), 0.5 mg/kg (n=3) or 1.25 mg/kg (n=3). Over a 56-day observation period, HMB-001 was well tolerated; the most common adverse events (AEs) were mild or moderate in severity. No AEs or serious adverse events were deemed related to HMB-001. There were no dose-limiting toxicities and no thromboembolic events reported. Treatment with HMB-001 resulted in a dose-dependent pharmacodynamic effect of endogenous Factor VIIa accumulation that was associated with decreases in prothrombin time and improvement in exploratory thrombin generation analyses. The pharmacokinetic profile and half-life of HMB-001 support dosing every two weeks or a less-frequent dosing regimen. "People living with bleeding disorders like Glanzmann face devastating bleed events and considerable psychosocial impacts—from severe pain and social stigmatization to depression and isolation—but have no approved preventative treatments to turn to," said Suthesh Sivapalaratnam, MD, PhD, Queen Mary University of London, Barts Health NHS Trust. "These Phase 1 data support further investigation into the promise of HMB-001 for patients and providers across the globe who are awaiting innovation to improve the standard of care." Hemab has begun dosing and is actively enrolling in Phase 2, the multiple-ascending dose of the study, which will be expanded to sites across Europe and the U.S. For more information, visit clinicaltrials.gov (NCT06211634). In addition, Hemab announced that preclinical research findings of HMB-001 are now available online as part of the February 2024 issue of Nature Cardiovascular Research. The study results demonstrate the ability of HMB-001 to accumulate and potentiate the activity of endogenous Factor VIIa selectively on activated platelets, providing a sustained and localized procoagulant activity that may support prophylactic treatment of Glanzmann or other bleeding disorders. Read the paper here. About Glanzmann Thrombasthenia Glanzmann Thrombasthenia (Glanzmann) is a rare and severe bleeding disorder associated with debilitating and sometimes life-threatening bleeding episodes. Initial findings from the international Glanzmann's 360 (GT360) natural history study—Hemab's research initiative in partnership with UK specialist research consultancy Haemnet—found that 87% of the 104 respondents reported experiencing at least one bleed in the previous week, with 37% of those bleeds requiring medical treatment. These bleeding episodes significantly impact the mental health and quality of life of people living with Glanzmann. Low mood, emotional problems, and social isolation were reported by participants (66%, 50%, and 44% respectively), and 80% reported that they missed school or work due to bruising or bleeding. To date, there are no effective prophylactic treatment options for Glanzmann. About HMB-001 HMB-001 is bispecific antibody that binds and stabilizes endogenous Factor VIIa with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for accumulation of endogenous Factor VIIa in the body, recruitment of Factor VIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 is designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia with the potential to treat other debilitating rare bleeding disorders. The U.S. Food and Drug Administration granted Fast Track Designation to HMB-001 for the treatment of Glanzmann. About Hemab Therapeutics Hemab is a clinical-stage biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA and Copenhagen, Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company's strategic guidance, Hemab 1-2-5™, targets the development of 5 assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, Von Willebrand Disease, and others. Learn more at hemab.com. Follow us on LinkedIn and X (formerly known as Twitter). SOURCE Hemab Therapeutics