RegulationBreakthrough Therapy (US), Orphan Drug (US), Orphan Drug (KR), Orphan Drug (AU), Priority Review (AU), Priority Review (CN), Orphan Drug (JP)

Gene Sequence

Sequence Code 5386H

Source: *****

Sequence Code 5425H

Source: *****

Sequence Code 5467L

Source: *****

Clinical Trials associated with Emicizumab

NCT04563520 / RecruitingPhase 3IIT

aPCC and Emicizumab Safety Study in Congenital Hemophilia A Patients With Inhibitors (SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis)

The purpose of the aPCC-emicizumab safety study is to prospectively investigate the safety and hemostatic efficacy of a personalized dose of aPCC in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis during acute bleeding events or prior to procedures.

Start Date01 Nov 2024

Sponsor / Collaborator

Emory University

[+1]

CTRI/2024/08/071826 / RecruitingPhase 4IIT

Outcome of Emicizumab Prophylaxis in Patients of Haemophilia A with or without Inhibitor An Early Experience from A Tertiary Care Hospital From Eastern India - NIL

Start Date15 Aug 2024

Sponsor / Collaborator-

NCT05248594 / RecruitingNot ApplicableIIT

Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

We have developed a questionnaire to elucidate the dosing, frequency and indication for the use of emicizumab in patients with Hemophilia A (mild, moderate or severe) ages 0-3 years. We are also collecting data on any pre-, peri and post surgical practices while on emicizumab. More importantly, we are asking if pediatricians are planning to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when they are planning to do so. We hope that this data will help inform understanding of current use of emicizumab in infants and young children as a form of primary prophylaxis, especially when venous access has historically been a limiting factor.

Start Date01 Aug 2024

Sponsor / Collaborator

Montefiore Medical Center

[+1]

100 Clinical Results associated with Emicizumab

100 Translational Medicine associated with Emicizumab

100 Patents (Medical) associated with Emicizumab

618

Literatures (Medical) associated with Emicizumab

31 Dec 2024·mAbs

A pivotal decade for bispecific antibodies?

Article

Author: Surowka, Marlena ; Klein, Christian

Bispecific antibodies (bsAbs) are a class of antibodies that can mediate novel mechanisms of action compared to monospecific monoclonal antibodies (mAbs). Since the discovery of mAbs and their adoption as therapeutic agents in the 1980s and 1990s, the development of bsAbs has held substantial appeal. Nevertheless, only three bsAbs (catumaxomab, blinatumomab, emicizumab) were approved through the end of 2020. However, since then, 11 bsAbs received regulatory agency approvals, of which nine (amivantamab, tebentafusp, mosunetuzumab, cadonilimab, teclistamab, glofitamab, epcoritamab, talquetamab, elranatamab) were approved for the treatment of cancer and two (faricimab, ozoralizumab) in non-oncology indications. Notably, of the 13 currently approved bsAbs, two, emicizumab and faricimab, have achieved blockbuster status, showing the promise of this novel class of therapeutics. In the 2020s, the approval of additional bsAbs can be expected in hematological malignancies, solid tumors and non-oncology indications, establishing bsAbs as essential part of the therapeutic armamentarium.

01 Dec 2024·PEDIATRIC BLOOD & CANCER

CHIEF: A retrospective self‐control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis

Article

Author: Malvar, Jemily ; Carneiro, Kendall ; Young, Guy ; Mashiach, Daniel ; Knight, Susan ; Mead, Patrice

Abstract:

Background:

Hemophilia A (HA) is an X‐linked bleeding disorder diagnosed by a deficiency in factor VIII (FVIII). For severe HA (SHA), prophylaxis clotting factor concentrates (CFC) has become the standard of care; however, it imparts a high treatment burden and typically results in an annualized bleeding rate (ABR) of 2–6. Emicizumab, a subcutaneously administered FVIII substitute, has become the de facto standard‐of‐care prophylaxis for children with SHA in many countries. Previous clinical trials of emicizumab have assessed ABR in patients greater than 12 years without inhibitors, and in children less than 12 years with inhibitors; however, there is little information published regarding the ABR of emicizumab compared to CFC in non‐inhibitor SHA children.

Methods:

Using a retrospective electronic medical record chart review, we conducted a self‐control analysis of 15 patients less than 12 years of age during equivalent periods of CFC versus emicizumab prophylaxis.

Results:

The mean ABR on CFC and emicizumab was 1.79 and 1.13 (p = .092), respectively, with a substantially decreased rate of joint bleeds (CFC 0.94; emicizumab 0.33; p = .001) and spontaneous bleeds (CFC 0.79; emicizumab 0.23; p = .008). No safety events were recorded for patients while administering emicizumab. The mean annual cost of CFC prophylaxis was $515,340 (SD $199,540), compared to $328,410 (SD $137,230) for emicizumab prophylaxis (p < .001).

Conclusion:

Emicizumab resulted in an improved ABR compared to CFC, especially for joint and spontaneous bleeds, had fewer administration complications, and was substantially less expensive compared to CFC prophylaxis; however, more research is necessary for a complete understanding of the effect of emicizumab on joint health and muscle bleeds.

01 Dec 2024·Blood Research

Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization

Article

Author: Cappuccilli, Maria ; Leogrande, Melania ; Perrone, Valentina ; Esposti, Luca Degli

Abstract:

Purpose:

This real-world analysis described the Hemophilia A (HA) population in Italy, evaluating drug utilization and consumption of factor VIII (FVIII) products of patients under prophylaxis and on-demand therapy.

Methods:

From Jan-2017 to Jun-2022, male patients with HA were identified through prescriptions of FVIII products [extended half-life FVIII, standard half-life recombinant FVIII, and plasma-derived FVIII (EHL FVIII, SHL rFVIII, and pdFVIII, respectively)], or emicizumab or FVIII plus von Willebrand factor or HA-related hospitalization using administrative flows of Italian healthcare entities. Patients on treatment with FVIII products during 2021–2022 were stratified by treatment regimen (prophylaxis/on-demand). The mean annual consumption expressed in International Units (IU) of EHL FVIII and SHL FVIII in patients treated during 2021–2022 having at least 12-month follow-up were assessed.

Results:

Among included HA patients, 145 (39.5%) received EHL FVIII and 222 (60.5%) SHL FVIII. Of 165 patients on prophylaxis, 105 (64%) received an EHL FVIII and 60 (36%) an SHL FVIII. The mean annual consumption of FVIII was 336,700 IU (median 319,000 IU) for EHL FVIII and 440,267 IU (median 360,500 IU) for SHL FVIII. Specifically, for patients on EHL FVIII, the most common drugs were efmoroctocog alfa (N = 51) and damoctocog alfa pegol (N = 50), followed by turoctocog alfa pegol (N = 25) and rurioctocog alfa pegol (N = 19). Of 702 HA patients initially treated with FVIII products, 74 (10.5%) switched to emicizumab during follow-up.

Conclusion:

These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection.

News (Medical) associated with Emicizumab

24 Oct 2024

·www.pharmaceutical-technology.com

Be Bio raises $82m to advance haemophilia B gene therapy to Phase I/II trial

Be Bio’s BE-101 works by inserting the human Factor IX (FIX) gene into primary human B cells. Image credit: Shutterstock/ ardhikauw. Be Biopharma has raised $82m in financing to advance a gene therapy targeting hemophilia B to a Phase I/II trial, whilst also adding an ex-Roche exec who was involved with the launch of a haemophilia A treatment to its roster. The financing had big pharma involvement in the form of Bristol Myers Squibb and Takeda Ventures, with venture capital firms ARCH Venture Partners, Atlas Venture, and RA Capital Management participating. Be Bio said the funds, which were also from Alta Partners and the Longwood Fund, will be allocated to the hemophilia B candidate BE-101 and a newly unveiled programme for hypophosphatasia called BE-102. Alongside the funds, the US biopharma added Suha Patel and Dr. Kiran Patki to its ranks, who will serve as senior vice president of commercial & franchise strategy and senior vice president of clinical development, respectively. Patel was involved in launching the haemophilia A treatment Hemlibra (emicizumab-kxwh) at Roche’s Genentech, whilst Patki previously worked at rare disease biotech Rally Bio. Be Bio’s CEO Dr. Joanne Smith-Farrell said that “some valued colleagues will be moving on” as the company shifts resources toward product development. Be Bio’s BE-101 is a genetically engineered B cell therapeutic that works by inserting the human Factor IX (FIX) gene into primary human B cells, which allows for continuous expression of active FIX levels with a single infusion. Haemophilia B, which affects around 40,000 globally, is an X-linked recessive bleeding disorder caused by a deficiency in clotting factor IX and has traditionally been treated with regular infusions of the protein. The advancement of gene therapies is changing the treatment paradigm for this disorder, with single infusions reducing the burden on patients taking repeated treatments. There are currently two US Food and Drug Administration (FDA)-approved gene therapies for haemophilia B – CSL Behring’s Hemgenix (etranacogene dezaparvovec) and Pfizer’s Beqvez (fidanacogene elaparvovec ). Be Bio’s candidate has an FDA orphan drug designation and fast track status, which confers benefits such as the possibility of seven years of market exclusivity. See Also: TreeFrog and University of Pittsburgh probe TLS role in immuno-oncology Lilly’s Kisunla does not demonstrate “good value” for UK’s NHS The FDA approved an investigational new drug application (IND) in March this year, greenlighting the company to initiate a trial evaluating the gene therapy. The multi-centre, first-in-human dose escalation study (NCT06611436), called BeCoMe-9, will evaluate the safety and preliminary efficacy of intravenously administered BE-101 in adults with moderately severe to severe haemophilia B. The trial is estimated to enrol 24 patients and has a primary endpoint of adverse events and serious adverse events one year post dose. Smith-Farrell said: “We are eager to clinically demonstrate BE-101’s potential to provide a highly durable FIX replacement therapy for Hemophilia B patients. Additionally, we are excited to advance BE-102 to address the needs of a large patient population with few to no therapeutic options.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Orphan DrugGene TherapyFast TrackClinical Study

23 Oct 2024

·www.globenewswire.com

[Ad hoc announcement pursuant to Art. 53 LR] Roche’s strong sales growth of 9% (CER) continues in the third quarter of 2024; Group sales increase 6% in the first nine months

Group sales grew by 6%1 at constant exchange rates (CER) (2% in CHF) in the first nine months, driven by the high demand for both our medicines and diagnostics; excluding COVID-19-related products, sales increased by 8%In the third quarter, Group sales rose by 9% (6% in CHF), as they did in the second quarter Pharmaceuticals Division sales rose by 7% in the first nine months; the strong growth of 9% in the base business2 was driven by continued high demand for our newer medicines to treat severe diseases; Vabysmo (serious eye diseases), Phesgo (breast cancer) and Ocrevus (multiple sclerosis) were major growth driversDiagnostics Division sales increased by 5% in the first nine months, while the base business2 grew by 8% due to higher demand for immunodiagnostic, pathology and molecular solutionsHighlights: US approval for Itovebi (inavolisib) for breast cancer, Ocrevus Zunovo subcutaneous injection for multiple sclerosis and Tecentriq Hybreza subcutaneous formulation for various types of cancerEU approval for Vabysmo for retinal vein occlusion (RVO), a serious eye disease, and PiaSky for paroxysmal nocturnal haemoglobinuria (PNH), a rare life-threatening blood conditionPositive phase III data for Gazyva/Gazyvaro (lupus nephritis, a kidney disease), Xofluza (influenza) and Tecentriq (lung cancer). New positive phase II data for fenebrutinib (multiple sclerosis), and new positive long-term data for Evrysdi (spinal muscular atrophy)Acquired AntlerA Therapeutics for a novel target in ophthalmology, and signed agreement for the acquisition of two next-generation CDK inhibitor drugs targeting breast cancer from Regor PharmaceuticalsClosing of acquisition of LumiraDx’s point-of-care technology to expand access to diagnostic testing in primary care and low- and middle-income countriesLaunch of the cobas Respiratory flex test, the first to use our new Temperature-Activated Generation of Signal (TAGS) technologyWHO endorsement for CINtec PLUS testing for cervical cancer prevention Outlook for 2024 confirmed Roche CEO Thomas Schinecker: “Our strong growth momentum continued in the third quarter, reflecting the high demand for our innovative medicines and diagnostic solutions and their positive impact on patients’ lives around the world. We made significant progress in our pharmaceuticals portfolio in the last quarter with five important regulatory approvals for our medicines, three positive phase III read-outs, and two acquisitions to strengthen our oncology and ophthalmology pipelines. Itovebi (inavolisib) recently received US approval based on clinical data demonstrating a reduction of more than 50% in the risk of death or worsening disease for people suffering from a form of advanced, hard-to-treat breast cancer. In addition, we had positive phase III results for Gazyva/Gazyvaro in lupus nephritis, a potentially life-threatening kidney disease for which limited treatment options are available today. We confirm our outlook for 2024.” Sales CHF millions As % of sales % change January‒September 2024 2023 2024 2023 At CER In CHF Group 44,984 44,053 100.0 100.0 6 2 Pharmaceuticals Division 34,257 33,372 76.2 75.8 7 3 United States 18,166 17,430 40.4 39.6 7 4 Europe 6,613 6,259 14.7 14.2 7 6 Japan 2,083 2,937 4.6 6.7 -21 -29 International* 7,395 6,746 16.5 15.3 19 10 Diagnostics Division 10,727 10,681 23.8 24.2 5 0 All figures shown in the table were restated to reflect the shift of the Foundation Medicine (FMI) business from the Pharmaceuticals Division to the Diagnostics Division. *Asia-Pacific, CEETRIS (Central Eastern Europe, Türkiye, Russia and Indian subcontinent), Latin America, Middle East, Africa, Canada, others Outlook for 2024 confirmed Roche expects an increase in Group sales in the mid single digit range (CER). Core earnings per share are targeted to grow in the high single digit range (CER), excluding the impact from the resolution of tax disputes in 2023. Roche expects to further increase its dividend in Swiss francs. Group salesIn the first nine months of 2024, Group sales increased by 6% at CER (2% in CHF) to CHF 45.0 billion as strong demand for our novel medicines as well as diagnostic products including immunodiagnostic, pathology and molecular solutions more than offset the anticipated decline in COVID-19-related sales and the impact of biosimilar/generic erosion. The appreciation of the Swiss franc against most currencies had an adverse impact on the sales reported in Swiss francs compared to constant exchange rates. The Pharmaceuticals Division sales increased by 7% to CHF 34.3 billion, while the base business (excluding COVID-19) grew by 9%, driven primarily by higher sales of Vabysmo (severe eye diseases), Phesgo (breast cancer), Ocrevus (multiple sclerosis), Hemlibra (haemophilia) and Polivy (blood cancer). These five medicines together generated total sales of CHF 13.2 billion, an increase of CHF 2.7 billion (CER) from the first nine months of 2023. The eye medicine Vabysmo, launched in early 2022, continues to be a major growth driver, generating sales of CHF 2.8 billion on growing demand in all regions. Sales of Avastin (various types of cancer), Herceptin (breast and gastric cancer) and MabThera/Rituxan (blood cancer, rheumatoid arthritis) decreased by a combined CHF 0.5 billion as the impact of biosimilar competition slowed further. Sales of the COVID-19 medicine Ronapreve were negligible compared with CHF 0.5 billion in the first nine months of 2023. In the United States, sales grew by 7% as strong sales of Vabysmo, Ocrevus, Polivy and Xolair (food allergies) were partially offset by the continued decline in sales of medicines for which patent protection has expired. Vabysmo achieved CHF 2.1 billion in sales, showing a high uptake in both new patients and patients switching from other medications. In Europe, sales rose by 7%, driven by demand for Vabysmo as well as by the continued uptake of Phesgo, Ocrevus, Evrysdi (spinal muscular atrophy) and Hemlibra. This was partially offset by lower sales of medicines for which patent protection has expired and of Perjeta (breast cancer) due to the ongoing conversion of patients to Phesgo. Sales in Japan were down 21%, mainly due to the base effect of the supply of Ronapreve (COVID-19) to the government in the first quarter of 2023. Excluding this effect, sales in Japan were 3% lower as strong demand for Phesgo and Vabysmo was more than offset by the impact of government price cuts and lower sales of medicines for which patent protection has expired. Sales in the International region surged by 19%, led by demand for Perjeta, Hemlibra, Tecentriq (cancer immunotherapy), Phesgo and Ocrevus as well as the launch of Elevydis (gene therapy, Duchenne muscular dystrophy). Sales in China increased by 8%, driven by Xofluza, Perjeta, Polivy and Avastin. The Diagnostics Division sales increased by 5% to CHF 10.7 billion, while the base business (excluding COVID-19) grew by 8%. Immunodiagnostic products, which include cardiac, oncology and thyroid tests, were the main growth drivers (10%). Additional growth came from pathology and molecular solutions. Sales of COVID-19 tests were CHF 0.1 billion in the first nine months of 2024 compared with CHF 0.4 billion in the corresponding period last year. Sales growth was reported across regions, with the Europe, Middle East and Africa (EMEA) region growing by 5%, North America by 6%, Asia-Pacific by 2% and Latin America by 18%. Pharmaceuticals: key developments Compound Milestone Regulatory Itovebi (inavolisib)Breast cancer FDA approves Itovebi, a targeted treatment for advanced hormone receptor (HR)-positive, HER2-negative breast cancer with a PIK3CA mutation Approval is based on phase III INAVO120 results, showing the regimen based on Itovebi (inavolisib) more than doubled progression-free survival compared with palbociclib and fulvestrant alone in the first-line settingThis approval helps address an urgent unmet need in breast cancer for people with a mutated PIK3CA gene, one of the most commonly mutated genes in HR-positive disease and which is associated with poor prognosisItovebi is Roche’s first targeted therapy approved for people with HR-positive disease, the most prevalent breast cancer subtype, marking an important step in our ambition to continue bringing innovative medicines to more people with breast cancer More information: Media Release, 11 October 2024 Ocrevus ZunovoMultiple sclerosis FDA approves Ocrevus Zunovo as the first and only twice-a-year 10-minute subcutaneous injection for people with relapsing and progressive multiple sclerosis Ocrevus Zunovo has the potential to expand treatment options to centres without intravenous (IV) infrastructure or with IV constraints, like at a doctor’s officeThis approval is backed by a decade of proven safety and efficacy data of Ocrevus IV, with over 350,000 people treated globallyOcrevus Zunovo offers people with multiple sclerosis more options to access treatment based on their individual needs More information: Media Release, 16 September 2024 Tecentriq Hybreza Various types of cancer FDA approves Tecentriq Hybreza, the first and only subcutaneous anti-PD-(L)1 cancer immunotherapy Tecentriq Hybreza provides patients and physicians with greater flexibility of treatment options while showing safety and efficacy consistent with intravenous (IV) TecentriqNew subcutaneous option reduces treatment time to approximately seven minutes, compared with 30‒60 minutes for IV infusion More information: Media Release, 13 September 2024 PiaSkyRare blood disease PiaSky approved in the EU as the first monthly subcutaneous treatment for people with paroxysmal nocturnal haemoglobinuria (PNH) With the option of self-administration, PiaSky (crovalimab) has the potential to reduce treatment burden for people with PNH and their caregivers in EuropeApproval is based on COMMODORE 2, where subcutaneous (SC) PiaSky once a month was equivalent to intravenous eculizumab every two weeksPiaSky advances C5 inhibition through innovative recycling technology, which enables its monthly SC administration More information: Media Release, 27 August 2024 Vabysmo Severe eye diseases European Commission approves Vabysmo for treatment of retinal vein occlusion (RVO) Approval is based on data from two phase III studies in branch and central retinal vein occlusion (RVO) showing early and sustained vision improvements non-inferior to aflibercept, and robust retinal drying with VabysmoAdditional submitted data shows that up to 60% of people receiving Vabysmo were able to extend treatment intervals to three or four monthsVabysmo is already approved in several countries, including the US and Japan, for RVO and in nearly 100 countries for people with neovascular or ‘wet’ age-related macular degeneration (nAMD) and diabetic macular edema (DME) More information: Media Release, 30 July 2024 Phase III, pivotal and other key readouts EvrysdiSpinal muscular atrophy Majority of children with spinal muscular atrophy (SMA) treated with Evrysdi are able to sit, stand and walk independently, two-year data demonstrate Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before six weeks of age, with most achieving motor milestones similar to children without SMAAll children were able to swallow and feed orally, with none requiring permanent ventilationEvrysdi is the only non-invasive SMA therapy and is approved in over 100 countries, with more than 16,000 people with SMA treated globally More information: Media Release, 14 October 2024 Gazyva/GazyvaroKidney disease Positive phase III results for Gazyva/Gazyvaro show superiority to standard therapy alone in people with lupus nephritis The REGENCY study met its primary endpoint, demonstrating statistically significant and clinically meaningful treatment benefits in people with active lupus nephritisGazyva/Gazyvaro is designed to target an underlying cause of lupus nephritis, aiming to prevent or delay progression to end-stage kidney diseaseLupus nephritis is a potentially life-threatening manifestation of an autoimmune disease affecting 1.7 million people worldwide, primarily women; up to one-third of people on current treatments will progress to end-stage kidney disease within 10 years More information: Media Release, 26 September 2024 Xofluza Influenza Positive phase III results show Xofluza significantly reduces the transmission of influenza viruses Data from the CENTERSTONE study shows single-dose Xofluza reduces transmission of influenza from an infected person to household membersThis is the first time that any antiviral used in the treatment of a respiratory viral illness has demonstrated a transmission reduction benefit in a global phase III studyReducing the spread of infection in the household could help limit transmission within communities and societies, easing the burden of both seasonal and pandemic influenza on healthcare systems More information: Media Release, 19 September 2024 Fenebrutinib Multiple sclerosis Fenebrutinib demonstrated near-complete suppression of disease activity and disability progression for up to 48 weeks in patients with relapsing multiple sclerosis New phase II data show vast majority of patients experiencing no relapses or disability progressionFenebrutinib suppressed acute and chronic MRI lesionsFenebrutinib’s safety profile was consistent with previous and ongoing clinical trials across multiple diseases including more than 2,700 people to date More information: Media Release, 4 September 2024 Susvimo Severe eye disease New data for Susvimo demonstrates sustained efficacy in two serious diabetic eye conditions Two-year phase III data presented at ASRS 2024 show Susvimo’s potential as an alternative to eye injections to treat diabetic macular edema (DME) and diabetic retinopathy (DR)Safety data were consistent with the known safety profile for Susvimo in people with DME and DRAdditionally, the US FDA has accepted the filing application for Susvimo in DME and DR based on one-year Pagoda and Pavilion study data More information: Media Release, 18 July 2024 Other Pharma Research and Early Development Center Roche opens Pharma Research and Early Development Center in Basel to accelerate scientific innovation Switzerland’s most innovative research and development centre underscores Roche’s long-term investment in scientific advancement to meet patient needsThe new centre will simplify and increase collaboration, thereby accelerating scientific innovation More information: Media Release, 10 September 2024 Pharmaceuticals sales Sales CHF millions As % of sales % change January–September 2024 2023 2024 2023 At CER In CHF Pharmaceuticals Division 34,257 33,372 100.0 100.0 7 3 United States 18,166 17,430 53.0 52.2 7 4 Europe 6,613 6,259 19.3 18.8 7 6 Japan 2,083 2,937 6.1 8.8 -21 -29 International* 7,395 6,746 21.6 20.2 19 10 All figures shown in the table were restated to reflect the shift of the Foundation Medicine (FMI) business from the Pharmaceuticals Division to the Diagnostics Division. * Asia-Pacific, CEETRIS (Central Eastern Europe, Türkiye, Russia and Indian subcontinent), Latin America, Middle East, Africa, Canada, others Top-selling medicines Total United States Europe Japan International CHF m % CHF m % CHF m % CHF m % CHF m % Ocrevus Multiple sclerosis 5,056 9 3,640 7 961 11 - - 455 26 Hemlibra Haemophilia A 3,280 10 1,906 5 690 10 259 4 425 42 Vabysmo Eye diseases (nAMD, DME, RVO) 2,816 79 2,146 67 454 148 86 37 130 256 Perjeta3 Breast cancer 2,809 -1 1,029 -4 502 -17 92 -36 1,186 17 Tecentriq Cancer immunotherapy 2,703 1 1,325 -8 649 5 277 -1 452 32 Actemra/RoActemra3 RA, COVID-19 1,948 5 949 11 508 -11 225 9 266 17 Xolair3 Asthma 1,737 11 1,737 11 - - - - - - Kadcyla3 Breast cancer 1,494 6 574 4 428 -2 71 4 421 22 Evrysdi Spinal muscular atrophy 1,246 21 429 15 435 18 66 10 316 35 Phesgo Breast cancer 1,244 58 404 29 543 44 88 - 209 104 Alecensa Lung cancer 1,151 7 372 12 217 1 144 3 418 8 Herceptin3 Breast and gastric cancer 1,063 -11 201 -20 227 -15 11 -48 624 -5 MabThera/Rituxan3 Blood cancer, RA 1,023 -16 615 -17 109 -21 12 -26 287 -10 Avastin 3 Various cancer types 943 -17 289 -20 63 -17 149 -33 442 -8 Activase/TNKase3 Cardiac diseases 895 2 850 1 - - - - 45 5 Polivy Blood cancer 817 41 410 83 142 6 143 -4 122 79 Gazyva/Gazyvaro3 Blood cancer 670 13 333 15 185 8 21 -16 131 21 Pulmozyme3 Cystic fibrosis 329 0 213 -5 55 -3 1 17 60 23 Mircera3 Anaemia related to kidney disease 304 -1 - - 31 -6 28 -23 245 4 CellCept3 Immunosuppressant 283 1 17 -21 81 -13 27 -9 158 17 DME: diabetic macular edema / nAMD: neovascular or ‘wet’ age-related macular degeneration / RVO: retinal vein occlusion / RA: rheumatoid arthritis Diagnostics: key developments Product Milestone VENTANA CLDN18 assay Gastric or gastro-oesophageal junction cancer Roche obtains CE certification for the first companion diagnostic to identify patients with gastric and gastro-oesophageal junction cancer eligible for targeted treatment with VYLOY The new VENTANA CLDN18 (43-14A) RxDx assay helps fulfil an unmet medical need by enabling clinicians to identify patients with gastric or gastro-oesophageal junction (GOJ) cancer who may benefit from a targeted treatment optionCLDN18.2 is an emerging biomarker in gastric and GOJ cancers and helps predict the likelihood of response to targeted therapyAs the leader in companion diagnostics, Roche continues to build on its commitment to improve personalised healthcare to enable better patient outcomes More information: Media Release, 10 October 2024 cobas Respiratory flex testRespiratory illnesses Roche launches the first test to use its breakthrough TAGS technology for high-throughput, simultaneous detection of 12 respiratory viruses The new Temperature-Activated Generation of Signal (TAGS) technology enables up to 15 targets to be detected simultaneously in a single patient sample on the high-throughput molecular diagnostic analysers cobas 5800, 6800 and 8800TAGS has the potential to revolutionise testing for other infectious diseases in the future, by bringing high-throughput customised syndromic panel testing to the routine clinical laboratoryThe first TAGS-based test to be made available, the cobas Respiratory flex, offers fast, efficient detection of up to 12 of the most common respiratory viruses with the flexibility for targeted testing, expediting accurate diagnosis, optimising antimicrobial use and saving time in the lab More information: Media Release, 24 September 2024 CINtec PLUS Cervical cancer WHO endorses dual-stain cytology (CINtec PLUS) testing in its cervical cancer prevention guidelines, advancing patient care and underlining Roche’s role in pioneering cervical cancer solutions CINtec PLUS Cytology is the only FDA-approved and CE-marked dual-stain test to triage human papillomavirus (HPV)-positive cervical cancer screening test resultsDual-stain biomarkers aid in detection of cervical precancer and may reduce the number of women who undergo unnecessary colposcopy procedures while allowing earlier intervention for those who are at higher risk of developing cervical cancerThis recognition follows the American Society for Colposcopy and Cervical Pathology (ASCCP)’s recent inclusion of dual-stain testing in cervical cancer screening guidelines, as well as other WHO prequalifications of Roche’s cobas HPV test More information: Media Release, 23 September 2024 cobas MPXV test, LightMix Mpox Roche responds to WHO’s declaration of a global health emergency due to the ongoing mpox outbreak Roche is committed to supporting all those working to overcome the mpox outbreak by providing access to high-quality polymerase chain reaction (PCR) testingRoche confirms that its cobas MPXV test and the LightMix research-use-only kits detect the latest mpox virus variantsWe are actively working to enhance laboratory testing capacity for mpox worldwide More information: Media Release, 20 August 2024 LumiraDx Roche closes acquisition of LumiraDx’s point-of-care technology to expand access to diagnostic testing in primary care The acquisition of LumiraDx’s point-of-care technology received all required antitrust and regulatory clearancesLumiraDx’s transformative point-of-care solution will complement Roche’s diagnostics portfolio across clinical chemistry, immunochemistry, coagulation and molecular, and across multiple disease areasBy integrating LumiraDx, Roche will further its ambition to deliver decentralised solutions that expand global access to testing in primary care settings worldwide More information: Media Release, 29 July 2024 Diagnostics sales Sales CHF millions As % of sales % change January–September 2024 2023 2024 2023 At CER In CHF Diagnostics Division 10,727 10,681 100.0 100.0 5 0 Customer Areas4 Core Lab 6,052 5,836 56.4 54.6 9 4 Molecular Lab5 1,903 1,897 17.7 17.8 4 0 Near Patient Care6 1,616 1,902 15.1 17.8 -10 -15 Pathology Lab 1,156 1,046 10.8 9.8 15 11 Regions Europe, Middle East and Africa 3,589 3,569 33.5 33.4 5 1 North America5 3,222 3,103 30.0 29.1 6 4 Asia-Pacific 3,146 3,263 29.3 30.5 2 -4 Latin America 770 746 7.2 7.0 18 3 More information on Roche sales in the first nine months of 2024: Q3 2024 presentationAppendix with tables About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in diagnostics and pharmaceuticals with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com. All trademarks used or mentioned in this release are protected by law. References [1] Unless otherwise stated, all growth rates and comparisons to the previous year in this document are at constant exchange rates (CER: average rates 2023) and all total figures quoted are reported in CHF. [2] Pharmaceuticals Division base business: excluding COVID-19 medicine Ronapreve. Diagnostics Division base business: excluding COVID-19-related products. [3] Products launched before 2015. [4] Core Lab: diagnostics solutions in the areas of immunoassays, clinical chemistry and CustomBiotech. Molecular Lab: diagnostics solutions for pathogen detection and monitoring, donor screening, sexual health and genomics, genomic tumour profiling. Near Patient Care: diagnostics solutions in emergency rooms, medical practices and directly with patients, including integrated personalised diabetes management. Pathology Lab: diagnostics solutions for tissue biopsies and companion diagnostics. [5] Sales in the Molecular Lab customer area include sales from the Foundation Medicine business, which moved under the responsibility of the Diagnostics Division from the Pharmaceuticals Division effective 1 January 2024. The comparative information for 2023 has been restated accordingly. [6] Sales in the new Near Patient Care customer area include sales from Diabetes Care and the Point of Care business, both previously shown as separate customer areas. The comparative information for 2023 has been restated accordingly. Cautionary statement regarding forward-looking statements This document contains certain forward-looking statements. These forward-looking statements may be identified by words such as ‘believes’, ‘expects’, ‘anticipates’, ‘projects’, ‘intends’, ‘should’, ‘seeks’, ‘estimates’, ‘future’ or similar expressions or by discussion of, among other things, strategy, goals, plans or intentions. Various factors may cause actual results to differ materially in the future from those reflected in forward-looking statements contained in this document, such as: (1) pricing and product initiatives of competitors; (2) legislative and regulatory developments and economic conditions; (3) delay or inability in obtaining regulatory approvals or bringing products to market; (4) fluctuations in currency exchange rates and general financial market conditions; (5) uncertainties in the discovery, development or marketing of new products or new uses of existing products, including without limitation negative results of clinical trials or research projects, unexpected side effects of pipeline or marketed products; (6) increased government pricing pressures; (7) interruptions in production; (8) loss of or inability to obtain adequate protection for intellectual property rights; (9) litigation; (10) loss of key executives or other employees; and (11) adverse publicity and news coverage. The statement regarding earnings per share growth is not a profit forecast and should not be interpreted to mean that Roche’s earnings or earnings per share for this or any subsequent period will necessarily match or exceed the historical published earnings or earnings per share of Roche. Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: media.relations@roche.com Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Nina Mählitz Phone: +41 79 327 54 74 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Dr Bruno Eschli Phone: +41 61 68-75284 e-mail: bruno.eschli@roche.com Dr Sabine Borngräber Phone: +41 61 68-88027 e-mail: sabine.borngraeber@roche.com Dr Birgit Masjost Phone: +41 61 68-84814 e-mail: birgit.masjost@roche.com Investor Relations North America Loren Kalm Phone: +1 650 225 3217 e-mail: kalm.loren@gene.com Attachments 23102024_Roche Q32023 appendix tables 23102024_Roche Media Release Q32024_EN

Phase 3Clinical ResultDrug ApprovalAcquisitionPhase 2

14 Oct 2024

·www.biopharmadive.com

Pfizer drug for hemophilia approved by FDA

Dive Brief:The Food and Drug Administration on Friday approved a new kind of medicine for hemophilia, clearing Pfizers Hympavzi for people with either the A or B forms of the inherited bleeding condition.Adults and children 12 years of age or older who havent developed proteins, or inhibitors, that prevent clotting drugs from working well are eligible to receive Hympavzi, the FDA said. The drug is taken once per week via an under-the-skin injection.Typically, medicines for hemophilia replace the blood clotting factor that people with the disease lack. By contrast, Hympavzi works by reducing activity of an anticoagulatory protein called tissue factor pathway inhibitor, or TFPI. This increases enzymes that help blood clump together,which in turn can prevent bleeding from occurring.Dive Insight:Approval of Hympavzi expands Pfizers portfolio of hemophilia medicines months after the company secured U.S. clearance of a gene therapy for the B form of the condition. Another of the companys gene therapies, for the A form, recently met the goals of a late-stage study, too.We look forward to launching this latest medical breakthrough and to now offer three distinct classes of hemophilia medicines an anti-TFPI, gene therapy, and recombinant factor treatments that can meet the unique treatment needs of a wide range of patients, said Aamir Malik, Pfizers U.S. commercial chief, in a statement.The drug is part of a new generation of treatments that are reaching market, substantially expanding the options patients with the condition have. Roches Hemlibra has become a new preventive standard for people with hemophilia A, while Sanofi recently secured approval of a long-acting factor replacement drug called Altuviiio.The new options have complicated the case for gene therapy, which promises a yearslong fix for the condition with a single infusion. Uptake of treatments from CSL Behring, BioMarin Pharmaceutical and, most recently, Pfizer, has been slow.Hympavzi offers more flexibility, both in its weekly dosing and in its utility for both hemophilia A and B. In testing, preventive treatment with Pfizers drug proved superior to on-demand use of factor replacement, substantially lowering annualized bleeding rates. Bleeding rates with Hympavzi were lower compared to preventive factor treatment as well.Treatment with Hympavzi was commonly associated with injection site reactions, headache and itching. The FDAs label for the drug also warns of circulating blood clots, hypersensitivity and embryofetal toxicity.Regulators in Europe recommended clearance for Hympavzi last month, sending it to the European Commission for marketing authorization.The regulatory advances are good news for Pfizer, which has struggled through a series of research setbacks and doubts about its growth that recently culminated in a public challenge from activist investor Starboard Value. '

Drug ApprovalGene Therapy

100 Deals associated with Emicizumab

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KEGG	Wiki	ATC	Drug Bank
D10821	Emicizumab	B02BX06	DB13923

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Coagulation Protein Disorders	JP	Chugai Pharmaceutical Co., Ltd.	23 Mar 2018
Hemophilia A	US	Genentech, Inc.	16 Nov 2017

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Factor 8 Deficiency, Acquired	Phase 2	AT	Hoffmann-La Roche, Inc.	23 Mar 2021
Factor 8 Deficiency, Acquired	Phase 2	DE	Hoffmann-La Roche, Inc.	23 Mar 2021
Von Willebrand Disease, Type 3	Phase 1	US	Genentech, Inc.	01 Nov 2022
Hemophilia	Phase 1	CN	Roche China Holding Ltd.	12 Mar 2018
Hemophilia	Phase 1	CN	F. Hoffmann-La Roche Ltd.	12 Mar 2018

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04431726 (HAVEN 7, CTgov)	Phase 3	Hemophilia A	55	Emicizumab	dcdfpsawfw(ovoflxdmvr) = xdyigboxru jwxbksegdx (cbpgghgyyn, usftktotee - ppxgiqeqyg) View more	-	14 Jun 2024
EHA2024	Not Applicable	-	25	octocog alfa	ekjfkphcoa(smydrvmlts) = kytqcutdxe dqoukmvuvy (cehiimrlmv, 38)	-	14 May 2024
				emicizumab	ekjfkphcoa(smydrvmlts) = yhjuunxohn dqoukmvuvy (cehiimrlmv, 9)
EHA2024	Not Applicable	Hemophilia A	16	Emicizumab	poqcynlgix(dschlqoilx) = rglhtfyvgg zzcowxoice (vubytkrhio )	Negative	14 May 2024
				FVIII replacement	poqcynlgix(dschlqoilx) = ljdbxanphp zzcowxoice (vubytkrhio )
NCT03191799 (STASEY, Pubmed) Manual	Phase 3	Hemophilia A	56	Emicizumab	muxshrjotw(hhmmsrfemx) = euqqwlzyet qjhoguaihf (nmiocwssek ) View more	Positive	12 Jan 2024
				Emicizumab (underwent major surgeries managed with additional hemostatic medication)	muxshrjotw(hhmmsrfemx) = zonypygagc qjhoguaihf (nmiocwssek )
ASH2023	Not Applicable	Hemophilia A	-	Emicizumab	isscdiybts(xdkylnegkf) = dqasbcypxh ndlnczyovq (uhngyomnmy )	-	11 Dec 2023
				Efanesoctocog alfa	isscdiybts(xdkylnegkf) = hvgzwdqccs ndlnczyovq (uhngyomnmy )
ASH2023	Not Applicable	Hemophilia A	127	Emicizumab prophylaxis	hahevljqcl(xgwelowklw) = noylktteaj efuynvoenk (plgppjsmkl ) View more	-	10 Dec 2023
ASH2023	Not Applicable	Factor 8 Deficiency, Acquired	-	Emicizumab	dyuuekiyrt(canbaevaik) = pgtwckejuj hfdqmtykre (qrrooifpfp ) View more	Positive	10 Dec 2023
				Immunosuppressive therapy (IST)	dyuuekiyrt(canbaevaik) = wllvvljsmc hfdqmtykre (qrrooifpfp ) View more
HAVEN 7 (BusinessWire) Manual	Phase 3	Hemophilia A	55	Hemlibra	laqhvewqaq(tacvzljxvd) = xbrhmmhaqg kkdsngzsfo (argqrpkeea ) View more	Positive	09 Dec 2023
NCT04431726 (HAVEN 7, ASH2023)	Phase 3	Hemophilia A	55	Emicizumab prophylaxis	tgkvdjyhbm(dgyqgvvbng) = pgyqujztfm brjmhwjcht (jnbufbrfth ) View more	-	09 Dec 2023
ISTH2023	Not Applicable	Hemophilia A	4	Factor VIIa	giiaxrxbjt(kwocddgpcx) = pexofgouez hdapdmruit (ninujncxkp )	Positive	24 Jun 2023

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