Tachycardia, Sinus

Results of positron emission tomography (PET) trial and second multiple ascending dose (MAD-2) trial support RAP-219's transformative potential for epilepsy and other central nervous system (CNS) disorders Continued momentum in the Phase 2a trial of RAP-219 in patients with refractory focal epilepsy, with topline results expected in the third quarter of 2025Expect to initiate a Phase 2a trial in patients with bipolar mania in the third quarter of 2025, with topline results expected in the first half of 2027 Appointed recognized translational and clinical drug development leader, Jeffrey Sevigny, M.D., as chief medical officer Ended the year with $305.3 million in cash, cash equivalents, and short-term investments, excluding restricted cash, which is expected to fund operations through the end of 2026 BOSTON and SAN DIEGO, March 11, 2025 (GLOBE NEWSWIRE) -- Rapport Therapeutics, Inc. (Nasdaq: RAPP) (“Rapport” or the “Company”), a clinical-stage biotechnology company dedicated to the discovery and development of small molecule precision medicines for patients with neurological or psychiatric disorders, today reported financial results for the quarter and full year ended December 31, 2024, and provided a business update. “With continued execution in the fourth quarter, Rapport is well positioned to deliver on multiple meaningful development milestones. The RAP-219 Phase 2a trial in patients with refractory focal epilepsy continues to advance with strong momentum; patient enrollment and dosing are progressing as planned, and the trial remains on track to deliver topline results in the third quarter of 2025. Based on data from 100 healthy subjects dosed, we continue to have confidence in RAP-219’s potential as a transformational medicine. The recently released PET and MAD-2 trial results demonstrated neuroanatomical specificity and favorable tolerability, further reinforcing our belief in its differentiated precision profile,” said Abraham N. Ceesay, CEO of Rapport. "We are committed to the pipeline-within-a-product strategy for RAP-219 and anticipate initiating a Phase 2a trial in bipolar mania in the third quarter of this year while continuing planning for our Phase 2a trial in diabetic peripheral neuropathic pain." BUSINESS HIGHLIGHTS RAP-219 Lead Program Announced PET and MAD-2 Data, Further Supporting RAP-219's Transformative Potential for CNS Disorders: In January, the Company announced results from its healthy volunteer PET and MAD-2 trials for RAP-219. Data demonstrated that neuroanatomical specificity was achieved through RAP-219’s selective targeting of TARPγ8. In Cohort 1 of the human PET trial, which used the dosing regimen utilized in the Company’s ongoing Phase 2a trial in patients with refractory focal epilepsy, RAP-219 achieved target receptor occupancy associated with maximal seizure protection in preclinical models within five days and was generally well tolerated, which we believe further supports the use of such dosing regimen in the Phase 2a trial.Favorable Tolerability Across Four Phase 1 Trials. A total of four Phase 1 trials have been conducted to date -- a single ascending dose trial, two multiple ascending dose trials, and a PET trial (final study report in progress) -- with 100 healthy volunteers exposed to RAP-219. In all of these trials, RAP-219 was generally well tolerated with no serious adverse events (“SAEs”). There were three treatment discontinuations (3%) that were attributed to treatment emergent adverse events (“TEAEs”), with no TEAEs greater than Grade 2. No clinically significant laboratory, electrocardiogram (ECG), or vital sign abnormalities were reported in the SAD or two MAD trials. While the final study report is in progress, PET trial TEAEs are generally consistent with other Phase 1 trials. Among the 48 participants exposed to RAP-219 in the two MAD trials, the most common TEAEs were headache (n=5), sinus tachycardia (n=4), and brain fog, insomnia, bowel movement irregularity, dry mouth, and medical device site reaction (n=3 each). Among the 16 participants exposed to placebo, the most common TEAEs were abdominal pain, brain fog, constipation, cough, decreased appetite, dizziness, medical device site reaction, and second-degree atrioventricular block (n=1 each). Focal Epilepsy Presented Data at AES Demonstrating Consistent Association Between Decreases in RNS Measured Long Episodes and Meaningful Reductions in Clinical Seizures: In December, Rapport presented novel findings on the association between the seizure biomarker used in the Company’s proof-of-concept trial for RAP-219 and clinical seizures. Patients with refractory focal epilepsy who would meet the enrollment criteria for the ongoing RAP-219 Phase 2a trial were selected from NeuroPace’s long-term study database for this post-hoc analysis. The analysis demonstrated a linear relationship between changes in long episode (LE) frequency and clinical seizure frequency and identified the benchmark to predict clinically meaningful seizure reduction. Rapport presented the data at the American Epilepsy Society (AES) Annual Meeting in Los Angeles.Phase 2a Trial Topline Results Expected in Third Quarter 2025. The Company expects to announce topline results for its Phase 2a trial in patients with refractory focal epilepsy in the third quarter of 2025, narrowing the timeline from prior guidance. Bipolar Disorder Established Timeline for Initiation of Phase 2a Trial of RAP-219 in Bipolar Mania. The Company expects to initiate a Phase 2a trial in patients with bipolar mania in the third quarter of 2025, with topline results expected in the first half of 2027. Peripheral Neuropathic Pain Finalizing Plans to Conduct Phase 2a Trial in Diabetic Peripheral Neuropathic Pain (DPNP). In the fourth quarter of 2024, the Company was notified by the FDA that the IND submitted by the Company for the initiation of a Phase 2a proof-of-concept trial of RAP-219 for the treatment of DPNP was placed on clinical hold, and the FDA requested additional information and protocol design amendments. The Company is now finalizing its trial plans with a goal of advancing work in this area of high unmet need as quickly as possible. Corporate Updates Appointment of Dr. Jeffrey Sevigny as Chief Medical Officer. Dr. Sevigny is a physician-scientist with more than 15 years of leadership in translational and clinical drug development, including as chief medical officer at Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly, and senior vice president of neuroscience at Eli Lilly. Dr. Sevigny has spearheaded groundbreaking research across neuroscience and rare diseases. His experience spans the full spectrum of development, from discovery to late-stage clinical trials and regulatory approvals. With a strong record of portfolio development and building high-performing organizations, he brings deep strategic and operational experience to Rapport. FOURTH QUARTER AND YEAR-END 2024 FINANCIAL RESULTS Net Loss: Net Loss for the fourth quarter of 2024 was $20.0 million, as compared to $13.5 million for the prior year period. For the full year 2024, net loss was $78.3 million, which compared to a net loss of $34.8 million for the full year 2023.Research and Development (R&D) Expenses: R&D expense was $17.2 million for the fourth quarter of 2024, as compared to $11.8 million for the prior year period. The increase in R&D expense was primarily driven by operational costs related to clinical development and costs to support the progression of the Company’s overall pipeline. For the full year 2024, R&D expense was $60.9 million, which compared to $28.0 million for the full year 2023.General and Administrative (G&A) Expenses: G&A expense was $6.3 million for the fourth quarter of 2024, as compared to $3.0 million for the prior year period. The increase in general and administrative expense was primarily driven by costs associated with the growth of the business, in addition to costs incurred to satisfy the requirements of operating as a public company. For the full year 2024, G&A expense was $22.1 million, which compared to $8.2 million for the full year 2023.Cash Position: The Company ended the fourth quarter with $305.3 million in cash, cash equivalents and short-term investments, compared to $320.7 million as of September 30, 2024. The decrease was primarily due to cash outflows on operating activities in the fourth quarter of 2024.Cash Runway: The Company expects that cash, cash equivalents, and short-term investments as of December 31, 2024 will enable the Company to fund its operating expenses and capital expenditure requirements through the end of 2026. About RAP-219 RAP-219 is a clinical-stage AMPA receptor (AMPAR) negative allosteric modulator (NAM) designed to achieve neuroanatomical specificity through its selective targeting of a receptor associated protein (RAP) known as TARPγ8, which is associated with neuronal AMPARs. Whereas AMPARs are distributed widely in the central nervous system, TARPγ8 is expressed only in discrete regions, including the hippocampus and neocortex, where focal seizures often originate. By contrast, TARPγ8 has minimal expression in the hindbrain, where drug effects are often associated with intolerable adverse events. With this precision approach, the Company believes RAP-219 has the potential to provide a differentiated profile as compared to traditional neuroscience medications. Due to the role of AMPA biology in various neurological disorders and the selective targeting of TARPγ8, the Company believes RAP-219 has pipeline-in-a-product potential and is evaluating the compound as a transformational treatment for patients with focal epilepsy, bipolar disorder, and peripheral neuropathic pain. Availability of Other Information About Rapport TherapeuticsRapport Therapeutics uses and intends to continue to use its Investor Relations website and LinkedIn (Rapport Therapeutics) as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company’s Investor Relations website and LinkedIn, in addition to following the Company’s press releases, SEC filings, public conference calls, presentations, and webcasts. The contents of the Company’s website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. About Rapport TherapeuticsRapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing small molecule precision medicines for patients with neurological or psychiatric disorders. The Company’s founders have made pioneering discoveries related to the function of receptor associated proteins (RAPs) in the brain. Their findings form the basis of Rapport’s RAP technology platform, which enables a differentiated approach to generate precision small molecule product candidates with the potential to overcome many limitations of conventional neurology drug discovery. Rapport’s precision neuroscience pipeline includes the Company’s lead investigational drug, RAP-219, designed to achieve neuroanatomical specificity through its selective targeting of a RAP expressed in only discrete regions of the brain. The Company is currently pursuing RAP-219 as a potential treatment for refractory focal epilepsy, bipolar mania and diabetic peripheral neuropathic pain. Additional preclinical and late-stage discovery stage programs are also underway, including targeting chronic pain and hearing disorders. Forward-Looking StatementsThis press release contains​ “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, but are not limited to, express or implied statements regarding: the clinical development of RAP-219 for the treatment of drug-resistant focal epilepsy, peripheral neuropathic pain and bipolar disorder, including the initiation, timing, progress and results of our ongoing and planned clinical trials; the Company’s ability to resolve a clinical hold with the FDA; the potential activity and tolerability of RAP-219; the potential of Rapport’s RAP technology platform; and expectations for Rapport’s uses of capital, expenses and financial results, including its cash runway through the end of 2026. Forward looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect Rapport’s business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to the company’s research and development activities; Rapport’s ability to execute on its strategy including obtaining the requisite regulatory approvals on the expected timeline, if at all; uncertainties relating to preclinical and clinical development activities; the company’s dependence on third parties to conduct clinical trials, manufacture its product candidates and develop and commercialize its product candidates, if approved; Rapport’s ability to attract, integrate and retain key personnel; risks related to the company’s financial condition and need for substantial additional funds in order to complete development activities and commercialize a product candidate, if approved; risks related to regulatory developments and approval processes of the U.S. Food and Drug Administration and comparable foreign regulatory authorities; risks related to establishing and maintaining Rapport’s intellectual property protections; and risks related to the competitive landscape for Rapport’s product candidates; as well as other risks described in​ “Risk Factors,” in the company’s Registration Statement on Form S-1, and most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Rapport’s subsequent filings with the Securities and Exchange Commission. Rapport expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Rapport Therapeutics, Inc.Consolidated Balance Sheet Data(In thousands) (unaudited) December 31,2024 December 31,2023 Assets Current assets Cash and cash equivalents $56,805 $70,169 Short-term investments 248,475 77,309 Restricted cash 105 85 Prepaid expenses and other current assets 4,417 3,309 Total current assets 309,802 150,872 Property and equipment, net 3,529 1,916 Operating lease right of use asset, net 1,442 2,084 Other assets 160 551 Total assets $314,933 $155,423 Liabilities, Convertible Preferred Stock and Stockholders’ Equity (Deficit) Current liabilities Accounts payable $1,954 $2,502 Accrued expenses and other current liabilities 6,076 5,631 Operating lease liability 737 670 Total current liabilities 8,767 8,803 Series B preferred stock tranche right liability — 4,200 Operating lease liability, net of current portion 739 1,476 Total liabilities 9,506 14,479 Commitments and contingencies Series A convertible preferred stock — 89,487 Series B convertible preferred stock — 77,091 Stockholders’ equity (deficit) Undesignated preferred stock — — Common Stock 37 4 Additional paid-in capital 429,657 19,796 Accumulated other comprehensive income (522) 4 Accumulated deficit (123,745) (45,438)Total stockholders’ equity (deficit) 305,427 (25,634)Total liabilities, convertible preferred stock, and stockholders’ equity $314,933 $155,423 Rapport Therapeutics, Inc.Condensed Consolidated Statement of Operations(In thousands, except share and per share data) (unaudited) For the three months ended December 31, For the year ended December 31, 2024 2023 2024 2023 Operating expenses Research and development $17,199 $11,799 $60,935 $27,999 General and administrative 6,322 2,995 22,120 8,180 Total operating expenses 23,521 14,794 83,055 36,179 Loss from operations (23,521) (14,794) (83,055) (36,179)Other income (expense): Interest income 3,541 1,375 12,138 2,527 Change in fair value of preferred stock tranche right liability — (94) (7,390) (1,124)Total other income, net 3,541 1,281 4,748 1,403 Net loss before income taxes (19,980) (13,513) (78,307) (34,776)Provision for income taxes — 6 — 10 Net loss (19,980) $(13,519) $(78,307) $(34,786)Net loss per share attributable to common stockholders, basic and diluted $(0.57) $(8.01) $(3.78) $(23.10)Weighted-average common shares outstanding, basic and diluted 35,069,296 1,687,087 20,738,338 1,505,774 Rapport Therapeutics, Inc.Condensed Consolidated Statements of Cash Flows(In thousands)(unaudited) For the three months ended December 31, For the year ended December 31, 2024 2023 2024 2023 Net cash used in operating activities $(14,446) $(10,357) $(64,828) $(27,181)Net cash used in investing activities 31,930 (78,661) (170,141) (78,860)Net cash provided by financing activities 7 (140) 221,625 145,136 Net increase in cash, cash equivalents and restricted cash $17,491 $(89,158) $(13,344) $39,095 Contact Julie DiCarlo Head of Communications & IR Rapport Therapeutics jdicarlo@rapportrx.com

100 percent of evaluable patients for minimal residual disease (MRD) testing achieved MRD negativity in MajesTEC-5 as induction therapy and MajesTEC-4 as maintenance therapy SAN DIEGO, Dec. 8, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE:JNJ) today announced new frontline data featuring TECVAYLI® (teclistamab-cqyv) from two investigational studies in patients with newly diagnosed multiple myeloma (NDMM) in induction and maintenance settings. The MajesTEC-5 (Abstract #493) and MajesTEC-4 (Abstract #494) studies establish the potential of TECVAYLI® for use in newly diagnosed patients, with promising efficacy and a tolerable safety profile. These data were highlighted as oral presentations at the 2024 American Society of Hematology (ASH) Annual Meeting.1,2 Forty-nine patients with transplant-eligible NDMM were treated with TECVAYLI® in combination with DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj), lenalidomide and dexamethasone (Tec-DRd) or DARZALEX FASPRO®, bortezomib, lenalidomide and dexamethasone (Tec-DVRd) as induction therapy in the MajesTEC-5 study.1 All patients who were evaluated for MRD negativity after cycle 3 of induction therapy achieved MRD negativity (10-5) and maintained through cycle 6.1 "These data from the MajesTEC-5 study build on the growing body of evidence of TECVAYLI combinations that support the potential combinability of TECVAYLI with other effective therapies, demonstrating high rates of MRD-negative responses for evaluable patients with newly diagnosed multiple myeloma," said Rachel Kobos, M.D., Vice President, Oncology Research & Development, Johnson & Johnson Innovative Medicine. "At Johnson & Johnson, our deep expertise and understanding of multiple myeloma has shaped the regimens we're developing, including our bispecific antibodies in new combinations, and we're committed to exploring the full potential of our therapies to improve outcomes for patients." The safety profiles were manageable and consistent with individual safety profiles.1 No treatment-emergent adverse events (TEAEs) led to study treatment discontinuation or death; cytokine release syndrome (CRS; Grade 1 or 2) occurred in 65 percent of patients.1 No patients experienced immune effector cell-associated neurotoxicity syndrome (ICANS).1 Grade 3/4 TEAEs included lymphopenia (43 percent), neutropenia (57 percent) and infections (35 percent).1 "There remains opportunity to achieve even deeper and more sustained outcomes for a broader patient population in the frontline setting," said Marc S. Raab, M.D., Heidelberg University Hospital, Germany.* "These data reinforce the potential of TECVAYLI when used in earlier lines and show that TECVAYLI can be leveraged to optimize existing standard regimens in combination." Results from the safety run-in of the Phase 3 MajesTEC-4 study highlighted the potential of TECVAYLI® to be administered as a maintenance therapy following autologous stem cell transplant (ASCT).2 MajesTEC-4 is the first study to present data on a B-cell maturation antigen (BCMA) bispecific as monotherapy or combination therapy after ASCT.2 Low rates of non-hematologic Grade 3/4 TEAEs and discontinuation of treatment due to all TEAEs (5.3 percent) were observed. CRS events were all Grade 1/2, mostly occurring during step-up dosing, and ICANS was not observed. Neutropenia and infections were the most common Grade 3/4 TEAEs.2 Grade 3/4 neutropenia at 6 months showed a decreased trend in cohorts 2 and 3 with less frequent TECVAYLI® dosing (cohort 1: 94 percent, cohort 2: 63 percent, cohort 3: 47 percent).2 A similar trend was observed for all-grade infections (cohort 1: 94 percent; cohort 2: 78 percent; cohort 3: 77 percent).2 All evaluable patients in cohort 1 who underwent MRD assessment after 12 months of therapy were MRD negative, and 100 percent of evaluable patients assessed in cohorts 2 and 3 were also MRD negative at cycle 6.2 Further analysis of combination therapies will be evaluated in the Phase 3 MajesTEC-7 study, which is currently enrolling. About MajesTEC-5 Study MajesTEC-5 (NCT05695508) is an ongoing, Phase 2 study of teclistamab and talquetamab, evaluating the safety and efficacy of combination regimens in participants with newly diagnosed transplant eligible multiple myeloma.3 About MajesTEC-4 Study MajesTEC-4 (NCT05243797) is an ongoing, multicenter, randomized, open-label, Phase 3 study of teclistamab in combination with lenalidomide and teclistamab alone versus lenalidomide alone in participants with newly diagnosed multiple myeloma as maintenance therapy following autologous stem cell transplantation.4 About MajesTEC-7 Study MajesTEC-7 (NCT05552222) is a Phase 3 randomized study comparing teclistamab in combination with daratumumab SC and lenalidomide (Tec-DR) and talquetamab in combination with daratumumab SC and lenalidomide (Tal-DR) versus daratumumab SC, lenalidomide, and dexamethasone (DRd) in participants with newly diagnosed multiple myeloma who are either ineligible or not intended for autologous stem cell transplant as initial therapy.5 About TECVAYLI® TECVAYLI® (teclistamab-cqyv) received approval from the U.S. FDA in October 2022 as an off-the-shelf (or ready-to-use) antibody that is administered as a subcutaneous treatment for adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.6 The European Commission (EC) granted TECVAYLI® conditional marketing authorization (CMA) in August 2022 as monotherapy for the treatment of adult patients with RRMM who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody, and have demonstrated disease progression since the last therapy. In August 2023, the EC granted the approval of a Type II variation application for TECVAYLI®, providing the option for a reduced dosing frequency of 1.5 mg/kg every two weeks in patients who have achieved a complete response (CR) or better for a minimum of six months. TECVAYLI® is a first-in-class, bispecific T-cell engager antibody therapy that uses innovative science to activate the immune system by binding to the CD3 receptor expressed on the surface of T-cells and to the B-cell maturation antigen (BCMA) expressed on the surface of multiple myeloma cells and some healthy B-lineage cells. In February 2024, the U.S. FDA approved the supplemental Biologics License Application (sBLA) for TECVAYLI® for a reduced dosing frequency of 1.5 mg/kg every two weeks (Q2W) in patients with relapsed or refractory multiple myeloma who have achieved and maintained a CR or better for a minimum of six months. For more information, visit . About DARZALEX FASPRO® and DARZALEX® DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) received U.S. FDA approval in May 2020 and is approved for nine indications in multiple myeloma, four of which are for frontline treatment in newly diagnosed patients who are transplant eligible or ineligible. It is the only subcutaneous CD38-directed antibody approved to treat patients with MM. DARZALEX FASPRO® is co-formulated with recombinant human hyaluronidase PH20, Halozyme's ENHANZE® drug delivery technology. DARZALEX® is the first CD38-directed antibody approved to treat multiple myeloma. DARZALEX®-based regimens have been used in the treatment of more than 585,000 patients worldwide and more than 239,000 patients in the U.S. alone. In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize daratumumab. For more information, visit . About Multiple Myeloma Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.7 In multiple myeloma, these plasma cells proliferate and spread rapidly and replace normal cells in the bone marrow with tumors.8 Multiple myeloma is the third most common blood cancer worldwide and remains an incurable disease.9 In 2024, it was estimated that more than 35,000 people will be diagnosed with multiple myeloma in the U.S. and more than 12,000 people would die from the disease.10 People living with multiple myeloma have a 5-year survival rate of 59.8 percent.11 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels and kidney problems or infections.12,13 TECVAYLI® IMPORTANT SAFETY INFORMATION INDICATION AND USAGE TECVAYLI® (teclistamab-cqyv) is a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). WARNINGS AND PRECAUTIONS Cytokine Release Syndrome - TECVAYLI® can cause cytokine release syndrome (CRS), including life-threatening or fatal reactions. In the clinical trial, CRS occurred in 72% of patients who received TECVAYLI® at the recommended dose, with Grade 1 CRS occurring in 50% of patients, Grade 2 in 21%, and Grade 3 in 0.6%. Recurrent CRS occurred in 33% of patients. Most patients experienced CRS following step-up dose 1 (42%), step-up dose 2 (35%), or the initial treatment dose (24%). Less than 3% of patients developed first occurrence of CRS following subsequent doses of TECVAYLI®. The median time to onset of CRS was 2 (range: 1 to 6) days after the most recent dose with a median duration of 2 (range: 1 to 9) days. Clinical signs and symptoms of CRS included, but were not limited to, fever, hypoxia, chills, hypotension, sinus tachycardia, headache, and elevated liver enzymes (aspartate aminotransferase and alanine aminotransferase elevation). Initiate therapy according to TECVAYLI® step-up dosing schedule to reduce risk of CRS. Administer pretreatment medications to reduce risk of CRS and monitor patients following administration of TECVAYLI® accordingly. At the first sign of CRS, immediately evaluate patient for hospitalization. Administer supportive care based on severity and consider further management per current practice guidelines. Withhold or permanently discontinue TECVAYLI® based on severity. TECVAYLI® is available only through a restricted program under a REMS. Neurologic Toxicity including ICANS - TECVAYLI® can cause serious or life-threatening neurologic toxicity, including Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS). In the clinical trial, neurologic toxicity occurred in 57% of patients who received TECVAYLI® at the recommended dose, with Grade 3 or 4 neurologic toxicity occurring in 2.4% of patients. The most frequent neurologic toxicities were headache (25%), motor dysfunction (16%), sensory neuropathy (15%), and encephalopathy (13%). With longer follow-up, Grade 4 seizure and fatal Guillain-Barré syndrome (one patient each) occurred in patients who received TECVAYLI®. In the clinical trial, ICANS was reported in 6% of patients who received TECVAYLI® at the recommended dose. Recurrent ICANS occurred in 1.8% of patients. Most patients experienced ICANS following step-up dose 1 (1.2%), step-up dose 2 (0.6%), or the initial treatment dose (1.8%). Less than 3% of patients developed first occurrence of ICANS following subsequent doses of TECVAYLI®. The median time to onset of ICANS was 4 (range: 2 to 8) days after the most recent dose with a median duration of 3 (range: 1 to 20) days. The most frequent clinical manifestations of ICANS reported were confusional state and dysgraphia. The onset of ICANS can be concurrent with CRS, following resolution of CRS, or in the absence of CRS. Monitor patients for signs and symptoms of neurologic toxicity during treatment. At the first sign of neurologic toxicity, including ICANS, immediately evaluate patient and provide supportive therapy based on severity. Withhold or permanently discontinue TECVAYLI® based on severity per recommendations and consider further management per current practice guidelines. Due to the potential for neurologic toxicity, patients are at risk of depressed level of consciousness. Advise patients to refrain from driving or operating heavy or potentially dangerous machinery during and for 48 hours after completion of TECVAYLI® step-up dosing schedule and in the event of new onset of any neurologic toxicity symptoms until neurologic toxicity resolves. TECVAYLI® is available only through a restricted program under a REMS. TECVAYLI® and TALVEY® REMS - TECVAYLI® is available only through a restricted program under a REMS called the TECVAYLI® and TALVEY® REMS because of the risks of CRS and neurologic toxicity, including ICANS. Hepatotoxicity - TECVAYLI® can cause hepatotoxicity, including fatalities. In patients who received TECVAYLI® at the recommended dose in the clinical trial, there was one fatal case of hepatic failure. Elevated aspartate aminotransferase (AST) occurred in 34% of patients, with Grade 3 or 4 elevations in 1.2%. Elevated alanine aminotransferase (ALT) occurred in 28% of patients, with Grade 3 or 4 elevations in 1.8%. Elevated total bilirubin occurred in 6% of patients with Grade 3 or 4 elevations in 0.6%. Liver enzyme elevation can occur with or without concurrent CRS. Monitor liver enzymes and bilirubin at baseline and during treatment as clinically indicated. Withhold TECVAYLI® or consider permanent discontinuation of TECVAYLI® based on severity. Infections - TECVAYLI® can cause severe, life-threatening, or fatal infections. In patients who received TECVAYLI® at the recommended dose in the clinical trial, serious infections, including opportunistic infections, occurred in 30% of patients, with Grade 3 or 4 infections in 35%, and fatal infections in 4.2%. Monitor patients for signs and symptoms of infection prior to and during treatment with TECVAYLI® and treat appropriately. Administer prophylactic antimicrobials according to guidelines. Withhold TECVAYLI® or consider permanent discontinuation of TECVAYLI® based on severity. Monitor immunoglobulin levels during treatment with TECVAYLI® and treat according to guidelines, including infection precautions and antibiotic or antiviral prophylaxis. Neutropenia - TECVAYLI® can cause neutropenia and febrile neutropenia. In patients who received TECVAYLI® at the recommended dose in the clinical trial, decreased neutrophils occurred in 84% of patients, with Grade 3 or 4 decreased neutrophils in 56%. Febrile neutropenia occurred in 3% of patients. Monitor complete blood cell counts at baseline and periodically during treatment and provide supportive care per local institutional guidelines. Monitor patients with neutropenia for signs of infection. Withhold TECVAYLI® based on severity. Hypersensitivity and Other Administration Reactions - TECVAYLI® can cause both systemic administration-related and local injection-site reactions. Systemic Reactions - In patients who received TECVAYLI® at the recommended dose in the clinical trial, 1.2% of patients experienced systemic-administration reactions, which included Grade 1 recurrent pyrexia and Grade 1 swollen tongue. Local Reactions - In patients who received TECVAYLI® at the recommended dose in the clinical trial, injection-site reactions occurred in 35% of patients, with Grade 1 injection-site reactions in 30% and Grade 2 in 4.8%. Withhold TECVAYLI® or consider permanent discontinuation of TECVAYLI® based on severity. Embryo-Fetal Toxicity - Based on its mechanism of action, TECVAYLI® may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with TECVAYLI® and for 5 months after the last dose. ADVERSE REACTIONS The most common adverse reactions (≥20%) were pyrexia, CRS, musculoskeletal pain, injection site reaction, fatigue, upper respiratory tract infection, nausea, headache, pneumonia, and diarrhea. The most common Grade 3 to 4 laboratory abnormalities (≥20%) were decreased lymphocytes, decreased neutrophils, decreased white blood cells, decreased hemoglobin, and decreased platelets. Please read full Prescribing Information , including Boxed WARNING, for TECVAYLI®. DARZALEX FASPRO® INDICATIONS AND IMPORTANT SAFETY INFORMATION  INDICATIONS DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) is indicated for the treatment of adult patients with multiple myeloma: In combination with bortezomib, lenalidomide, and dexamethasone for induction and consolidation in newly diagnosed patients who are eligible for autologous stem cell transplant In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor (PI) In combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy As monotherapy in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double refractory to a PI and an immunomodulatory agent IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS  DARZALEX FASPRO® is contraindicated in patients with a history of severe hypersensitivity to daratumumab, hyaluronidase, or any of the components of the formulation. WARNINGS AND PRECAUTIONS   Hypersensitivity and Other Administration Reactions   Both systemic administration-related reactions, including severe or life-threatening reactions, and local injection-site reactions can occur with DARZALEX FASPRO®. Fatal reactions have been reported with daratumumab-containing products, including DARZALEX FASPRO®. Systemic Reactions   In a pooled safety population of 1249 patients with multiple myeloma (N=1056) or light chain (AL) amyloidosis (N=193) who received DARZALEX FASPRO® as monotherapy or in combination, 7% of patients experienced a systemic administration-related reaction (Grade 2: 3.2%, Grade 3: 0.7%, Grade 4: 0.1%). Systemic administration-related reactions occurred in 7% of patients with the first injection, 0.2% with the second injection, and cumulatively 1% with subsequent injections. The median time to onset was 2.9 hours (range: 5 minutes to 3.5 days). Of the 165 systemic administration-related reactions that occurred in 93 patients, 144 (87%) occurred on the day of DARZALEX FASPRO® administration. Delayed systemic administration-related reactions have occurred in 1% of the patients. Severe reactions included hypoxia, dyspnea, hypertension, tachycardia, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Other signs and symptoms of systemic administration-related reactions may include respiratory symptoms, such as bronchospasm, nasal congestion, cough, throat irritation, allergic rhinitis, and wheezing, as well as anaphylactic reaction, pyrexia, chest pain, pruritus, chills, vomiting, nausea, hypotension, and blurred vision.   Pre-medicate patients with histamine-1 receptor antagonist, acetaminophen, and corticosteroids. Monitor patients for systemic administration-related reactions, especially following the first and second injections. For anaphylactic reaction or life-threatening (Grade 4) administration-related reactions, immediately and permanently discontinue DARZALEX FASPRO®. Consider administering corticosteroids and other medications after the administration of DARZALEX FASPRO® depending on dosing regimen and medical history to minimize the risk of delayed (defined as occurring the day after administration) systemic administration-related reactions.   Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with daratumumab-containing products. If ocular symptoms occur, interrupt DARZALEX FASPRO® and seek immediate ophthalmologic evaluation prior to restarting DARZALEX FASPRO®.   Local Reactions   In this pooled safety population, injection-site reactions occurred in 7% of patients, including Grade 2 reactions in 0.8%. The most frequent (>1%) injection-site reaction was injection-site erythema. These local reactions occurred a median of 5 minutes (range: 0 minutes to 6.5 days) after starting administration of DARZALEX FASPRO®. Monitor for local reactions and consider symptomatic management.   Neutropenia   Daratumumab may increase neutropenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX FASPRO® until recovery of neutrophils. In lower body weight patients receiving DARZALEX FASPRO®, higher rates of Grade 3-4 neutropenia were observed.   Thrombocytopenia   Daratumumab may increase thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Consider withholding DARZALEX FASPRO® until recovery of platelets.   Embryo-Fetal Toxicity   Based on the mechanism of action, DARZALEX FASPRO® can cause fetal harm when administered to a pregnant woman. DARZALEX FASPRO® may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX FASPRO® and for 3 months after the last dose.   The combination of DARZALEX FASPRO® with lenalidomide, thalidomide, or pomalidomide is contraindicated in pregnant women because lenalidomide, thalidomide, and pomalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, thalidomide, or pomalidomide prescribing information on use during pregnancy.   Interference With Serological Testing   Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab administration. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient's serum. The determination of a patient's ABO and Rh blood type are not impacted.   Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX FASPRO®. Type and screen patients prior to starting DARZALEX FASPRO®.   Interference With Determination of Complete Response   Daratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some DARZALEX FASPRO®-treated patients with IgG kappa myeloma protein.   ADVERSE REACTIONS   In multiple myeloma, the most common adverse reaction (≥20%) with DARZALEX FASPRO® monotherapy is upper respiratory tract infection. The most common adverse reactions with combination therapy (≥20% for any combination) include fatigue, nausea, diarrhea, dyspnea, insomnia, headache, pyrexia, cough, muscle spasms, back pain, vomiting, hypertension, upper respiratory tract infection, peripheral sensory neuropathy, constipation, pneumonia, and peripheral edema.   The most common hematology laboratory abnormalities (≥40%) with DARZALEX FASPRO® are decreased leukocytes, decreased lymphocytes, decreased neutrophils, decreased platelets, and decreased hemoglobin.   Please click here to see the full Prescribing Information for DARZALEX FASPRO®. About Johnson & Johnson At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity. Learn more at or at . Follow us at @JanssenUS and @JNJInnovMed. Janssen Research & Development, LLC and Janssen Biotech, Inc. are both Johnson & Johnson companies. Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of TECVAYLI® (teclistamab-cqyv) and DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj). The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc., and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc. nor Johnson & Johnson undertake to update any forward-looking statement as a result of new information or future events or developments. * Marc S. Raab, M.D., has provided consulting, advisory, and speaking services to Johnson & Johnson; he has not been paid for any media work. 1Raab, Marc, S., et al, 493 Phase 2 Study of Teclistamab-Based Induction Regimens in Patients with Transplant-Eligible (TE) Newly Diagnosed Multiple Myeloma (NDMM): Results from the GMMG-HD10/DSMM-XX (MajesTEC-5) Trial. 2024 American Society of Hematology Annual Meeting. December 2024. 2 Zamagni, Elena, et al., 494 Phase 3 Study of Teclistamab (Tec) in Combination with Lenalidomide (Len) and Tec Alone Versus Len Alone in Newly Diagnosed Multiple Myeloma (NDMM) As Maintenance Therapy Following Autologous Stem Cell Transplantation (ASCT): Safety Run-in (SRI) Results from the MajesTEC-4/EMN30 Trial. 2024 American Society of Hematology Annual Meeting. December 2024. 3 GMMG-HD10 / DSMM-XX / 64007957MMY2003, MajesTEC-5 (HD10/DSMMXX). . Accessed November 2024. 4 Phase 3 Study of Teclistamab in Combination With Lenalidomide and Teclistamab Alone Versus Lenalidomide Alone in Participants With Newly Diagnosed Multiple Myeloma as Maintenance Therapy Following Autologous Stem Cell Transplantation (MajesTEC-4). . Accessed November 2024. 5 A Study of Teclistamab in Combination With Daratumumab and Lenalidomide (Tec-DR) and Talquetamab in Combination With Daratumumab and Lenalidomide (Tal-DR) in Participants With Newly Diagnosed Multiple Myeloma (MajesTEC-7). . Accessed November 2024. 6 U.S. FDA Approves TECVAYLI® (teclistamab-cqyv), the First Bispecific T-cell Engager Antibody for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma. . Accessed November 2024. 7 Rajkumar SV. Multiple myeloma: 2020 update on diagnosis, risk-stratification and management. Am J Hematol. 2020;95(5):548-5672020;95(5):548-567. 8 National Cancer Institute. Plasma Cell Neoplasms. . Accessed November 2024. 9 City of Hope. Multiple Myeloma: Causes, Symptoms & Treatments. . Accessed November 2024. 10American Cancer Society. Key Statistics About Multiple Myeloma. . Accessed November 2024. 11SEER Explorer: An interactive website for SEER cancer statistics [Internet]. Surveillance Research Program, National Cancer Institute. . Accessed November 2024. 12 American Cancer Society. What is Multiple Myeloma? . Accessed November 2024. 13 American Cancer Society. Multiple Myeloma Early Detection, Diagnosis, and Staging. . Accessed November 2024. SOURCE Johnson & Johnson WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Favorable Findings from the Longest Public Clinical Study in CIDP Support HYQVIA as an Effective Long-Term Treatment Option for Maintaining Stable Disease Course in CIDP ADVANCE Clinical Program Reflects Takeda’s Commitment to Continued Research on the Role of Immunoglobulin (IG) Therapy for Patients with Rare Neuroimmunological Disorders OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced data from the Phase 3 ADVANCE-CIDP 3 clinical trial, a long-term extension study evaluating the safety and efficacy of HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] in patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Results showed favorable long-term safety and tolerability of HYQVIA, and a low relapse rate, supporting its use as maintenance treatment for CIDP. These findings will be presented in a poster session on Sunday, June 23, 2024 at the Peripheral Nerve Society (PNS) Annual Meeting in Montreal, Canada. HYQVIA is the first and only facilitated subcutaneous immunoglobulin (fSCIG) for CIDP, approved earlier this year by the U.S. Food and Drug Association (FDA) as maintenance therapy in adults with CIDP and by the European Commission for patients of all ages with CIDP post-stabilization with intravenous immunoglobulin (IVIG). HYQVIA’s hyaluronidase component facilitates the dispersion and absorption of large immunoglobulin (IG) volumes in the subcutaneous space between the skin and the muscle. This allows high-volume IG administration (equivalent to volumes administered intravenously) into the subcutaneous tissue over a short time. As a result, HYQVIA can be infused up to once monthly (every two, three or four weeks). HYQVIA can be self-administered after appropriate patient or caregiver training or administered by a healthcare professional in a medical office, infusion center or at a patient’s home.1 “The long-term data from the ADVANCE-CIDP 3 clinical trial allow us to further characterize the safety, efficacy and tolerability pro HYQVIA and reinforces its role as a long-term, up-to once monthly maintenance treatment for this complex, chronic condition,” said Kristina Allikmets, senior vice president and head of Research & Development for Takeda’s Plasma-Derived Therapies Business Unit. “These results reflect our continued commitment to bringing the benefits of our differentiated immunoglobulin therapies to patients with neuroimmunological disorders, and providing a range of effective treatment options that address the individual needs of a broad range of patients.” The ADVANCE-CIDP 3 clinical trial is the longest extension study ever performed within context of a clinical trial in CIDP to date. The study, which enrolled 85 patients from the ADVANCE-CIDP 1 clinical trial, evaluated the safety, tolerability and immunogenicity of HYQVIA. The primary outcome measure was safety/tolerability and immunogenicity. The median duration of HYQVIA treatment was 33 months (0 to 77 months) with a cumulative overall follow-up time of 220 patient years. The findings were consistent with the known safety and tolerability pro HYQVIA and no new safety concerns were observed.2 Key findings showed: The median monthly dose of HYQVIA across all patients was 64 (28.0 to 200.0) g/4 weeks. The mean infusion duration per dose of HYQVIA was 135.5 minutes with 88.2% of doses administered every 4 weeks and 92.3% of doses administered across two infusion sites. HYQVIA was well tolerated among the 3487 infusions administered; 3 (0.1%) infusions had a reduced infusion rate, were interrupted or stopped due to intolerability. Overall, adverse events (AEs) were reported in 89.4% of patients. AEs related to HYQVIA were reported in 60% of patients. Most AEs were mild or moderate and self-limiting, and consistent with the established safety pro HYQVIA. The most common AEs per infusion (≥0.02 events per infusion) were headache, infusion site erythema, pyrexia, nausea, erythema, infusion site pruritis, fatigue and infusion site pain. Serious AEs possibly related to HYQVIA occurred in three patients (one event each): infection at the infusion site, exacerbation of migraine and fibromyalgia after infusion, and exacerbation of heart failure that resolved following treatment. HYQVIA maintained stable disease course in patients with CIDP. Thirteen percent of patients with data available experienced a relapse during the entire observation period with an annualized relapse rate of 4.5%. “Results of the ADVANCE-CIDP 3 study help provide additional confidence to those living with CIDP and their healthcare providers regarding the potential for extended maintenance of their condition with a facilitated subcutaneous immunoglobulin,” said Dr. Robert Hadden, MD, Consultant Neurologist, Neurology Department, King’s College Hospital, London, UK and Department of Basic & Clinical Neuroscience, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK and ADVANCE-CIDP 3 presenting author. “This treatment allows the convenience of potential self-treatment at home, typically only once every four weeks.” CIDP is an acquired, immune-mediated condition affecting the peripheral nervous system that is characterized by progressive, symmetric weakness in distal and proximal limbs and impaired sensory function in the extremities.3 The role of IG therapy for CIDP has been well-established4 and is considered a standard of care for this complex and heterogeneous condition in guidelines from the European Academy of Neurology and Peripheral Nerve Society due to its broad immunomodulatory and anti-inflammatory effects.5 Nearly a quarter of all IG therapy is used in the treatment of CIDP.6 About HYQVIA® HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] is a liquid medicine containing Recombinant Human Hyaluronidase and immunoglobulins (IG) and is approved by the European Medicines Agency (EMA) as a replacement therapy in adults, children and adolescents with primary immunodeficiency (PI) and with secondary immunodeficiency (SID) who suffer from severe or recurrent infections, ineffective antimicrobial treatment, and either proven specific antibody failure (PSAF) or serum IgG level of <4 g/L. In addition, it is approved by the EMA as maintenance therapy in adults, children and adolescents (0-18 years) with chronic inflammatory demyelinating polyneuropathy (CIDP) after stabilization with intravenous immunoglobulin therapy (IVIG). In the United States it is approved to treat adults and children two years of age and older with PI as a well as a maintenance therapy for adult patients with CIDP. HYQVIA is infused under the skin into the fatty subcutaneous tissue. HYQVIA contains IG collected from human plasma. IG are antibodies that maintain the body’s immune system. The hyaluronidase part of HYQVIA facilitates the dispersion and absorption of IG in the subcutaneous space between the skin and the muscle. HYQVIA is infused up to once a month (every two, three or four weeks for CIDP; every three or four weeks for PI). About the ADVANCE-CIDP 3 Clinical Trial ADVANCE-CIDP 3 was a long-term extension of ADVANCE-CIDP 1, a Phase 3, double-blind, randomized, placebo-controlled study.7 All patients entering ADVANCE-CIDP 3 received open-label HYQVIA and continued to receive the same dose and dosing regimen from ADVANCE-CIDP 1 (mean monthly dose equivalent of 1.1 g/kg).7 The primary objective was to analyze long-term safety, tolerability and immunogenicity. Efficacy was an exploratory outcome, including evaluation of CIDP relapse. Further information about the ADVANCE-CIDP 3 clinical trial is available at ClinicalTrials.gov under study identifier NCT02955355. HyQvia® (Human normal immunoglobulin) 100 mg/ml solution for infusion for subcutaneous use European PRESCRIBING INFORMATION Always refer to the Summary of Product Characteristics (SmPC) and the local prescribing information of your country before prescribing. Presentation: HyQvia is a dual vial unit consisting of one vial of 10% human normal immunoglobulin (Ig) and one vial of recombinant human hyaluronidase (see the SmPC for details). Indications: Replacement therapy in adults, children and adolescents (0-18 years) in: primary immunodeficiency syndromes (PID) with impaired antibody production; secondary immunodeficiencies (SID) in patients who suffer from severe or recurrent infections, ineffective antimicrobial treatment and either proven specific antibody failure (PSAF) or serum IgG level of <4 g/l. PSAF is a failure to mount at least a 2-fold rise in IgG antibody titre to pneumococcal polysaccharide and polypeptide antigen vaccines. Immunomodulatory therapy in adults, children and adolescents (0 to 18 years) in: chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy after stabilization with IVIg. Dosage and administration: For subcutaneous use only. Therapy should be initiated and monitored under the supervision of a physician experienced in the treatment of immunodeficiency/CIDP. The product should be brought to room temperature before use. Inspect both vials for discolouration and particulate matter before administration. Do not use heating devices including microwaves. Do not shake or mix the components of the two vials. Suggested infusion site(s) are the middle to upper abdomen and thighs. The two components of the medicinal product must be administered sequentially through the same needle beginning with the recombinant human hyaluronidase followed by Ig 10%. Please see the SmPC for infusion rates. The full contents of the recombinant human hyaluronidase vial should be administered regardless of whether the full contents of the Ig 10% vial is administered. Longer needles may be used under medical supervision to prevent infusion site leakage. Home treatment should be initiated and monitored by a physician experienced in the guidance of patients for home treatment. Posology: Dose and dosage regimen may need to be individualised for each patient dependent on the response. The dose and dose regimens are dependent on the indication. Dose based on body weight may require adjustment in underweight or overweight patients. Replacement therapy in PID: Patients naïve to Ig therapy: The dose required to achieve a trough level of 6 g/L is approximately 0.4-0.8 g/kg body weight/month. The dose interval to maintain steady state levels varies from 2-4 weeks. IgG trough levels should be measured and assessed in conjunction with the incidence of infection. To reduce the rate of infection, it may be necessary to increase the dose and aim for higher trough levels (>6 g/l). At the initiation of therapy, it is recommended that the treatment intervals for the first infusions be gradually prolonged from a 1-week dose to up to a 3- or 4-week dose. Patients previously treated with intravenous (IV) Ig: For patients switching directly from IV Ig, or who have had a previous IV dose that can be referenced, the medicinal product should be administered at the same dose and at the same frequency as their previous IV Ig treatment. Patients previously treated with Ig administered subcutaneously: the initial dose of HyQvia is the same as for subcutaneous treatment but may be adjusted to 3- or 4-week intervals. The first infusion should be given one week after the last treatment with the previous Ig. Replacement therapy in SID: the recommended dose is 0.2-0.4 g/kg every 3 to 4 weeks. IgG levels should be measured and assessed in conjunction with the incidence of infection. Dose should be adjusted as necessary to achieve optimal protection against infections, an increase may be necessary in patients with persisting infection; a dose decrease can be considered when the patient remains infection free. Immunomodulatory therapy in CIDP: Before initiating therapy, the weekly equivalent dose should be calculated by dividing the planned dose by the planned dose interval in weeks. The typical dosing interval range for HyQvia is 3- to 4-weeks. The recommended subcutaneous dose is 0.3 to 2.4 g/kg body weight per month, administered in 1-or 2-sessions over 1-or 2-days. The patient's clinical response should be the primary consideration in dose adjustment. The dose may need to be adapted to achieve the desired clinical response. In clinical deterioration, the dose may be increased to the recommended maximum of 2.4 g/kg monthly. If the patient is clinically stable, periodic dose reductions may be needed to observe whether the patient still needs IG therapy. A titration schedule that permits gradual dose increase over time (ramp-up) is recommended to ensure the patient’s tolerability until the full dose is reached. During the titration schedule, the calculated HyQvia dose and recommended dose intervals must be followed for the first and second infusions. Depending on the treating physician's discretion, in patients who tolerate the first 2 infusions well, subsequent infusions may be administered by gradually increasing doses and dose intervals, considering the volume and total infusion time. An accelerated titration schedule may be considered if the patient tolerates the SC infusion volumes and the first 2 infusions. Doses less than or equal to 0.4 g/kg may be administered without a titration schedule, provided acceptable patient tolerance. Patients must be on stable doses (Variations in the dosing interval of up to ±7 days or monthly equivalent dose amount of up to ±20% between the subject’s IgG infusions are considered a stable dose) of IVIg. Before initiating therapy with the medicinal product, the weekly equivalent dose should be calculated by dividing the last IVIg dose by the IVIg dose interval in weeks. The starting dose and dosing frequency are the same as the patient’s previous IVIg treatment. The typical dosing interval for HyQvia is 4-weeks. For patients with less frequent IVIg dosing (greater than 4-weeks), the dosing interval can be converted to 4-weeks while maintaining the same monthly equivalent IgG dose. The calculated one-week dose (1st infusion) should be administered 2 - weeks after the last IVIg infusion (see Table 1 of the SmPC). One week after the first dose, the next weekly equivalent dose (2nd infusion) should be administered. A titration schedule can take up to 9-weeks (see Table 1 of the SmPC), depending on the dosing interval and tolerability. On a given infusion day, the maximum infusion volume should not exceed 1200 mL for patients weighing ≥40 kg or 600 mL for <40 kg. Suppose the maximum daily dose limit is exceeded or the patient cannot tolerate the infusion volume. In that case, the dose may be administered over multiple days in divided doses with 48-to 72-hours between doses to allow absorption of infusion fluid at the infusion site(s). The dose can be administered up to 3 infusion sites with a maximum infusion volume of 600 mL per site (or as tolerated). If using three sites, the maximum is 400 mL per site. Paediatric population: Replacement therapy and Immunomodulatory therapy: follow adult dosage guidance. Contraindications: Hypersensitivity to any ingredient or human IG especially in patients with antibodies against IgA; systemic hypersensitivity to hyaluronidase or human recombinant hyaluronidase; HyQvia must not be given IV or intramuscularly. Warnings and precautions: If HyQvia is accidentally administered into a blood vessel, patients could develop shock. The recommended infusion rate given in the SmPC should be adhered to. Infuse slowly and monitor closely throughout the infusion period, particularly patients starting therapy. Patients may require monitoring for up to 1 hour after administration. Manage infusion related events by slowing the infusion rate or stopping the infusion. Treatment will depend on the nature and severity of the adverse event. Patients should be reminded to report chronic inflammation and nodules which occur at the infusion site or other locations. For home treatment, patients should have the support of another responsible person in case of adverse reactions. Record treatment with HyQvia and batch number in patients’ notes. Hypersensitivity: Hypersensitivity reactions are possible in patients with anti-IgA antibodies who should only be treated with HyQvia if alternative treatments are not possible and under close medical supervision. In case of hypersensitivity, shock or anaphylactic-like reactions, discontinue the infusion immediately and treat the patient for shock. Rarely, human normal IG can induce a fall in blood pressure with anaphylactic reaction. In high-risk patients HyQvia should only be administered where supportive care is available for life threatening reactions. Patients should be informed of the early signs of anaphylaxis/ hypersensitivity. Pre-medication may be used as a preventative measure. Hypersensitivity to recombinant human hyaluronidase: Any suspicion of allergic or anaphylactic like reactions following recombinant human hyaluronidase administration requires immediate discontinuation of the infusion and standard medical treatment should be administered, if necessary. Immunogenicity of recombinant human hyaluronidase: Development of non-neutralising antibodies and neutralizing antibodies to the recombinant human hyaluronidase component has been reported in patients receiving HyQvia in clinical studies. Thromboembolism: Thromboembolic events including myocardial infarction, stroke, deep venous thrombosis and pulmonary embolism have been observed with IG treatment and cannot be excluded with use of HyQvia. Ensure adequate hydration prior to treatment. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk. Patients should be informed about initial symptoms and advised to contact their physician immediately upon onset. Haemolytic anaemia: IG products contain antibodies to blood groups (e.g. A, B, D) which may act as haemolysins. Monitor for signs and symptoms of haemolysis. Aseptic meningitis syndrome: has been reported, symptoms usually begin within several hours to 2 days following treatment. Patients should be informed about initial symptoms. Discontinuation of IG treatment may result in remission within several days without sequelae. Interference with serological testing: After infusion of immunoglobulins, the transitory rise of the various passively transferred antibodies in the patient’s blood may result in misleading positive results in serological testing. Passive transmission of antibodies to erythrocyte´s surface antigens may interfere with some serological tests for red cell antibodies. Infusions of immunoglobulin products may lead to false positive readings in assays that depend on detection of β-D glucans for diagnosis of fungal infections. Transmissible agents: Infectious diseases due to the transmission of infective agents cannot be totally excluded. Sodium content: The recombinant human hyaluronidase component contains 4.03 mg sodium/mL. To be taken into consideration by patients on a controlled sodium diet. Traceability: The name and the batch number of the administered product should be clearly recorded. Interactions: Live attenuated virus vaccines – postpone vaccination for 3 months after treatment with HyQvia. For measles vaccine, impairment may persist for up to 1 year, so check antibody status. Please see the SmPC for details. Fertility, pregnancy and lactation: Safety during pregnancy has not been established and immunoglobulins are excreted into the milk, therefore use with caution in pregnant and breastfeeding mothers. Effects on ability to drive and use machines: The ability to drive and operate machines may be impaired by some adverse reactions e.g., dizziness associated with this medicinal product. Patients who experience adverse reactions during treatment should wait for these to resolve before driving or operating machines. Undesirable effects: Very common (≥1/10 patients): Headache, Blood pressure increased and Hypertension, Nauseam Diarrhoea, Vomiting, Arthralgia, Local reactions (Infusion site discomfort, Infusion site pain, Injection site pain, Puncture site pain and Tenderness; infusion site erythema and Injection site erythema; Infusion site oedema, Injection site oedema, infusion site swelling, Injection site swelling and Swelling (local), Feeling hot, Asthenia, Fatigue, Lethargy and Malaise. Common (≥1/100, <1/10 patients): Migraine, Tremor, Paraesthesia, Sinus tachycardia and Tachycardia, Hypotension, Dyspnoea, Abdominal distension, Erythema, Pruritus, Rash, Rash erythematous, Rash macular, Rash maculo-papular and Rash popular Urticaria, Myalgia, Limb discomfort and Pain in extremity, Back pain, Joint stiffness, Musculoskeletal chest pain, Groin pain, Hemosiderinuria, Infusion related reaction, Infusion site bruising, Injection site bruising, Infusion site haematoma, Injection site haematoma, Infusion site haemorrhage and Vessel puncture site bruise, Infusion site reaction, Injection site reaction and Puncture site reaction, Infusion site mass, Injection site mass and Infusion site nodule, Infusion site discoloration, Infusion site rash and Injection site rash, Infusion site induration and Injection site induration, Infusion site warmth, Infusion site paraesthesia and Injection site paraesthesia, Infusion site inflammation, Chills, Oedema, Oedema peripheral and Swelling (systemic), Localised oedema, Peripheral swelling and Skin oedema, Gravitational oedema, Oedema genital, Scrotal swelling and Vulvovaginal swelling, Hyperhidrosis, Coombs direct test positive and Coombs test positive. Uncommon (≥ 1/1 000 to < 1/100): Cerebrovascular accident and Ischaemic stroke, Burning sensations. Other undesirable effects (rare or unknown frequency): Meningitis aseptic, Hypersensitivity, Direct Coombs’ test positive, Infusion site leakage, Influenza-like illness. Refer to the SmPC for details on full side effect and interactions. Marketing Authorisation (MA) numbers: 2.5g EU/1/13/840/001, 5g EU/1/13/840/002, 10g EU/1/13/840/003, 20g EU/1/13/840/004, 30g EU/1/13/840/005. Name and address of MA holder: Baxalta Innovations GmbH, Industriestrasse 67, A-1221 Vienna, Austria. HyQvia is a registered trade name. PI approval code: PI-02941 Date of preparation: June 2024. Further information is available on request Adverse events should be reported to the authorities in your country as required by local law. Adverse events should also be reported to Takeda at: GPSE@takeda.com. For Full U.S. Prescribing Information, please visit: About Takeda Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit . 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HyQvia 100 mg/mL solution for infusion for subcutaneous use Summary of Product Characteristics. Available at . 2 Hadden R et al. Hyaluronidase-facilitated subcutaneous immunoglobulin 10% for chronic inflammatory demyelinating polyradiculoneuropathy: final results from a long-term safety and tolerability study. Poster presented at: Peripheral Nerve Society (PNS) Annual Meeting, 22–25 June 2024, Montréal, Canada. P89. 3 Dalakas MC; Medscape. Advances in the diagnosis, pathogenesis and treatment of CIDP. Nat Rev Neurol. 2011;7(9):507-517. 4 Eftimov F, et al. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2013;(12):CD001797. 5 Van den Bergh PYK, et al. European Academy of Neurology/Peripheral Nerve Society guideline on diagnosis and treatment of chronic inflammatory demyelinating polyradiculoneuropathy: Report of a joint Task Force-Second revision [published correction appears in J Perpher Nerv System. 2022 Mar;27(1):94]. 6 Adivo 2020 data (incl. US, Canada, France, Germany, UK, Italy, Spain, Sweden, Netherlands, Japan, Taiwan, Australia, Brazil, Turkey, Russia, Argentina, Egypt, Kazakhstan, Saudi Arabia, Colombia) represents ~84% of Global IG Consumption (excl. China and India) 7 ClinicalTrials.gov. Long-Term Tolerability and Safety of HYQVIA/​HyQvia in CIDP. July 24, 2023. Last accessed May 2024 from