A Phase 2, Randomized, Open-label (With Blinded Plasminogen Activator and Placebo Control Groups) Study to Evaluate the Effects of Different Intra-thrombus Infusion Regimens of Plasmin (Human) Compared to Plasminogen Activator and Placebo In Patients With Acute Lower Extremity Native Artery or Bypass Graft Occlusion

The primary purpose of this Phase 2 study is to optimize Plasmin delivery by comparing different delivery regimens in patients with peripheral arterial occlusion. The study includes a blinded plasminogen activator treatment group and a blinded plasminogen activator placebo group. The study will also assess safety and tolerability of Plasmin at 150 and 250 mg doses.

Start Date01 Dec 2010

Sponsor / Collaborator

Grifols Therapeutics, Inc.

NCT00418483 / CompletedPhase 1

A Sequential Phase I/II Dose Escalation and Dose Selection Safety Study of Regional Intra-thrombus Plasmin (Human) Infusion In Acute Lower Extremity Native Artery or Bypass Graft Occlusion

The purpose of this study is to evaluate the safety of increasing doses of intra-thrombus Plasmin (Human) in acute peripheral arterial occlusion (aPAO). The ability of these Plasmin doses to dissolve the clots will be estimated by arteriography.

Start Date01 Mar 2007

Sponsor / Collaborator

Grifols Therapeutics, Inc.

100 Clinical Results associated with Human plasma-derived fibrinolysin (Grifols)

100 Translational Medicine associated with Human plasma-derived fibrinolysin (Grifols)

100 Patents (Medical) associated with Human plasma-derived fibrinolysin (Grifols)

Literatures (Medical) associated with Human plasma-derived fibrinolysin (Grifols)

01 Nov 2024·JOURNAL OF PEDIATRIC SURGERY

A Phase 3, Randomized, Active-controlled, Single-blind Clinical Trial to Evaluate the Efficacy of Fibrin Sealant Grifols in Achieving Hemostasis in Pediatric Surgery

Article

Author: Slavkovic, Andjelka ; Balanescu, Laura ; Camprubí, Sandra ; Hanna, Kim ; James, Michael K. ; Jenovari, Zoltan ; Querolt, Montse ; Gajdobranski, Djordje ; Bozzo, Jordi ; Mikickovic, Maja ; Kamenov, Krasimir ; Vajda, Péter ; Camprubí, Sandra ; Querolt, Montse ; Irtan, Sabine ; Mondou, Elsa ; Mondou, Elsa ; Kalinova, Krasimira ; Ninov, Borislav ; Sharif, Khalid ; Simeonov, Simeon ; Sretenović, Aleksandar ; Wiener, Isodoro ; Hanna, Kim

BACKGROUND:

In this study, two fibrin sealant products, Fibrin Sealant Grifols (FS Grifols 80 mg/mL fibrinogen; 500 IU/mL thrombin) and Evicel (fibrinogen 55-85 mg/mL; thrombin 800-1200 IU/mL) were studied for efficacy in achieving hemostasis at a targeted bleeding site (TBS) on parenchymous or soft tissue in pediatric surgeries.

METHODS:

This phase 3, single-blind, active comparator, non-inferiority trial compared the number of patients achieving hemostasis at a TBS at four (T₄ - primary endpoint), seven (T₇) and 10 (T₁₀) minutes after application, Safety and tolerability were assessed by recording adverse events during and after procedures. Eligible patients were <18 years old undergoing elective, open, non-cardiac thoracic, abdominal or pelvic surgeries. Preterm (<37 weeks gestation) and newborn (0-27 days) infants were eligible.

RESULTS:

At T₄, 98.7% of FS Grifols group (n = 91) and 95.4% of the Evicel group (n = 87) achieved hemostasis. All patients with residual bleeding at T₄ were undergoing soft tissue surgery. All patients achieved hemostasis by T₇. At T₁₀, all patients achieved hemostasis except one (FS Grifols (no observation recorded)). There were no incidents of persistent bleeding. For FS Grifols, 26.5% of patients had treatment-emergent adverse events (TEAEs) and 18.4% for Evicel. One TEAE (moderate procedural pain - FS Grifols group) was considered possibly related to study treatment. Three patients died for reasons unrelated to the study medications.

CONCLUSIONS:

FS Grifols was safe and effective at achieving hemostasis in pediatric patients having parenchymous or soft tissue surgeries. The efficacy of FS Grifols was non-inferior to Evicel.

LEVEL OF EVIDENCE:

01 Jan 2024·CLINICAL AND APPLIED THROMBOSIS-HEMOSTASIS

Real-World Efficacy and Safety of Plasma-Derived Von Willebrand Factor-Containing Factor VIII Concentrates in Patients With Von Willebrand Disease in Italy

Article

Author: Tosetto, Alberto ; Santoro, Rita Carlotta ; Santi, Roberto Mario ; Borchiellini, Alessandra ; De Cristofaro, Raimondo ; Federici, Augusto B. ; Mir, Roser ; Mairal, Esther ; Santoro, Cristina ; Barillari, Giovanni ; Zanon, Ezio ; Pieri, Lisa

Plasma-derived von Willebrand factor-containing factor VIII concentrates (pd-VWF/FVIII-C) are the mainstay of treatment in von Willebrand disease (VWD). Real-world data on efficacy and safety of these pd-VWF/FVIII-C are required. To retrospectively evaluate the efficacy and safety of pd-VWF/FVIII-C (Fanhdi® and Alphanate®, Grifols) in clinical practice in Italy. A multicentric, observational, retrospective study at 10 Italian centers was conducted. Eligible patients diagnosed with inherited VWD (ISTH criteria) were treated with either Fanhdi® or Alphanate® for bleeding episodes, prevention of surgical bleeding and secondary long-term prophylaxis (SLTP) according to clinical practice with medical records collected from January 2007 to December 2019. Efficacy/safety of pd-VWF/FVIII-C was assessed according to FDA-agreed objective criteria following regulatory procedures. Fifty-seven patients (M/F: 21/36) were enrolled in the study with the following VWD types: VWD1 (n = 29, 52%), VWD2A (n = 10, 18%), VWD2B (n = 7, 12%), VWD2M (n = 2, 4%), VWD2N (n = 1, 2%), VWD2 unclassified (n = 1, 2%), and VWD3 (n = 7, 12%). These pd-VWF/FVIII-C were used to manage 58 bleeding episodes (n = 24 patients), 100 surgeries (n = 47 patients), and 7 SLTP (n = 6 patients). Global clinical efficacy with these pd-VWF/FVIII-C was reported to be excellent/good in 85% of bleeding episodes, 98% of surgeries, and 100% of SLTP. As far as safety, no adverse-drug-related episodes, immunogenic or thrombotic events were reported. This study confirmed that Fanhdi® and Alphanate® were effective and safe in the management of bleeding episodes, the prevention of bleeding during surgeries and for SLTP in Italian patients with inherited VWD.

01 Mar 2023·EClinicalMedicine

Subcutaneous anti-COVID-19 hyperimmune immunoglobulin for prevention of disease in asymptomatic individuals with SARS-CoV-2 infection: a double-blind, placebo-controlled, randomised clinical trial

Article

Background:

Anti-COVID-19 hyperimmune immunoglobulin (hIG) can provide standardized and controlled antibody content. Data from controlled clinical trials using hIG for the prevention or treatment of COVID-19 outpatients have not been reported. We assessed the safety and efficacy of subcutaneous anti-COVID-19 hyperimmune immunoglobulin 20% (C19-IG20%) compared to placebo in preventing development of symptomatic COVID-19 in asymptomatic individuals with SARS-CoV-2 infection.

Methods:

We did a multicentre, randomized, double-blind, placebo-controlled trial, in asymptomatic unvaccinated adults (≥18 years of age) with confirmed SARS-CoV-2 infection within 5 days between April 28 and December 27, 2021. Participants were randomly assigned (1:1:1) to receive a blinded subcutaneous infusion of 10 mL with 1 g or 2 g of C19-IG20%, or an equivalent volume of saline as placebo. The primary endpoint was the proportion of participants who remained asymptomatic through day 14 after infusion. Secondary endpoints included the proportion of individuals who required oxygen supplementation, any medically attended visit, hospitalisation, or ICU, and viral load reduction and viral clearance in nasopharyngeal swabs. Safety was assessed as the proportion of patients with adverse events. The trial was terminated early due to a lack of potential benefit in the target population in a planned interim analysis conducted in December 2021. ClinicalTrials.gov registry: NCT04847141.

Findings:

461 individuals (mean age 39.6 years [SD 12.8]) were randomized and received the intervention within a mean of 3.1 (SD 1.27) days from a positive SARS-CoV-2 test. In the prespecified modified intention-to-treat analysis that included only participants who received a subcutaneous infusion, the primary outcome occurred in 59.9% (91/152) of participants receiving 1 g C19-IG20%, 64.7% (99/153) receiving 2 g, and 63.5% (99/156) receiving placebo (difference in proportions 1 g C19-IG20% vs. placebo, -3.6%; 95% CI -14.6% to 7.3%, p = 0.53; 2 g C19-IG20% vs placebo, 1.1%; -9.6% to 11.9%, p = 0.85). None of the secondary clinical efficacy endpoints or virological endpoints were significantly different between study groups. Adverse event rate was similar between groups, and no severe or life-threatening adverse events related to investigational product infusion were reported.

Interpretation:

Our findings suggested that administration of subcutaneous human hyperimmune immunoglobulin C19-IG20% to asymptomatic individuals with SARS-CoV-2 infection was safe but did not prevent development of symptomatic COVID-19.

Funding:

Grifols.

News (Medical) associated with Human plasma-derived fibrinolysin (Grifols)

07 Nov 2024

·www.prnewswire.com

Rigel Reports Third Quarter 2024 Financial Results and Provides Business Update

Third quarter total revenue of $55.3 million, which includes TAVALISSE® net product sales of $26.3 million, REZLIDHIA® net product sales of $5.5 million and GAVRETO® net product sales of $7.1 million Entered into an agreement with Kissei to develop and commercialize REZLIDHIA in all potential indications in Japan, the Republic of Korea and Taiwan, recording an upfront cash payment of $10.0 million during the third quarter Initial data from the ongoing Phase 1b study evaluating R289, a dual IRAK1/4 inhibitor, in LR-MDS to be presented at the 66th ASH Annual Meeting Conference call and webcast scheduled today at 4:30 p.m. Eastern Time SOUTH SAN FRANCISCO, Calif., Nov. 7, 2024 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today reported financial results for the third quarter ended September 30, 2024, including sales of TAVALISSE® (fostamatinib disodium hexahydrate) for the treatment of chronic immune thrombocytopenia (ITP); REZLIDHIA® (olutasidenib) for the treatment of relapsed or refractory (R/R) mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML); and GAVRETO® (pralsetinib) for the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) and advanced or metastatic thyroid cancer, and recent business progress. "2024 has been a significant year for Rigel, marked by the acquisition of GAVRETO, our third commercial product, strong revenue growth across our commercial portfolio, and the advancement of our development pipeline," said Raul Rodriguez, Rigel's president and CEO. "This great progress is underpinned by our focus on financial discipline, resulting in positive third-quarter and year-to-date net income. As we close out the year, we will continue driving momentum in our commercial portfolio and hematology and oncology development pipeline." Third Quarter 2024 Business Update Commercial Update Commercial strength continues for all products with record bottles shipped to patients and clinics and total bottles sold. GAVRETO became commercially available from Rigel in June 2024. Third-quarter results reflect the successful transition of existing patients on therapy to Rigel's product. For the fourth quarter, the focus will be on continuing to transition patients. The following table summarizes total bottles shipped for the third quarter: In September, Rigel entered into an exclusive license and supply agreement with Kissei Pharmaceutical Co., Ltd. ("Kissei") to develop and commercialize REZLIDHIA in all potential indications in Japan, the Republic of Korea and Taiwan. Under the terms of the agreement, Rigel received an upfront cash payment of $10.0 million from Kissei, with the potential for up to an additional $152.5 million in development, regulatory and commercial milestone payments. In late October, Rigel issued a Dear Health Care Provider (DHCP) letter related to a new safety signal for GAVRETO after consultation with the U.S. Food and Drug Administration (FDA). The DHCP letter has been posted to the GAVRETO Healthcare Provider website at . Clinical and Development Update Rigel continues to advance its Phase 1b clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R2891, a novel and selective dual IRAK1/4 inhibitor, in patients with R/R lower-risk myelodysplastic syndrome (LR-MDS). Enrollment in the fifth dose level (500mg / 250mg split dose) is underway. In early November, Rigel announced six poster presentations highlighting data from the company's commercial and clinical-stage hematology and oncology portfolio at the upcoming 66th American Society of Hematology (ASH) Annual Meeting and Exposition. Initial data from the ongoing Phase 1b study evaluating R289 in patients with R/R LR-MDS indicate that R289 was generally well tolerated in a heavily pretreated LR-MDS patient population, the majority of whom were high transfusion burden at study entry. As of the data cutoff, 14 of 19 patients were evaluable for efficacy and per International Working Group (IWG) 2018, RBC-transfusion independence (RBC-TI)/hematologic improvement (HI-E) occurred in 36% of patients receiving R289 doses ≥500 mg QD, with a median duration of RBC-TI of 29 weeks. RBC-TI >24 weeks was achieved in 2 high transfusion burden patients following 3 and 5 prior therapies, including a hypomethylating agent. The company will also present additional data for olutasidenib in patients with R/R mIDH1 AML and MDS. In September, Rigel announced the first patient was enrolled in a Phase 1b/2 triplet therapy trial of decitabine and venetoclax in combination with REZLIDHIA in patients with mIDH1 AML, which is being sponsored and conducted by The University of Texas MD Anderson Cancer Center (MD Anderson). This is the first trial in Rigel's multi-year strategic development alliance with MD Anderson. A paper detailing the differences in molecular structure, binding characteristics and clinical outcomes between olutasidenib and ivosidenib, including response rates in patients previously treated with ivosidenib or venetoclax, was published by Dr. Justin M. Watts, Associate Professor of Medicine, Division of Hematology, Chief, Leukemia Section at the University of Miami Health System, in Current Treatment Options in Oncology in October 2024. Third Quarter 2024 and Year-To-Date Financial Update For the third quarter ended September 30, 2024, total revenues were $55.3 million, consisting of $26.3 million in TAVALISSE net product sales, $5.5 million in REZLIDHIA net product sales, $7.1 million in GAVRETO net product sales, and $16.4 million in contract revenue from collaborations. TAVALISSE net product sales grew 8% compared to $24.5 million in the same period of 2023. REZLIDHIA net product sales grew 107% compared to $2.7 million in the same period of 2023. GAVRETO became commercially available from Rigel in June 2024. Contract revenue from collaborations consisted of $13.0 million from Kissei Pharmaceutical Co., Ltd. (Kissei) related to an upfront fee from sublicensing olutasidenib and delivery of drug supplies, as well as $3.3 million from Grifols S.A. (Grifols) and $0.1 million from Medison Pharma Trading AG (Medison) related to delivery of drug supplies and earned royalties. Total costs and expenses were $41.3 million compared to $32.6 million for the same period of 2023. The increase in costs and expenses was mainly due to higher cost of product sales, driven primarily by increased products sales, a sublicensing revenue fee to Forma, increased royalties and amortization of intangible assets. In addition, there were increases in personnel-related costs and commercial-related expenses. Rigel reported net income of $12.4 million, or $0.71 basic and $0.70 diluted per share, compared to a net loss of $5.7 million, or $0.33 basic and diluted per share, for the same period of 2023. The basic and diluted share and per share amounts for the prior period have been restated to reflect the 1-for-10 reverse stock split effected on June 27, 2024 on a retroactive basis. For the nine months ended September 30, 2024, total revenues were $121.7 million, consisting of $73.8 million in TAVALISSE net product sales, $15.6 million in REZLIDHIA net product sales, $9.0 million in GAVRETO net product sales, and $23.3 million in contract revenue from collaborations. TAVALISSE net product sales grew 8% compared to $68.1 million in the same period of 2023. REZLIDHIA net product sales grew 133% compared to $6.7 million in the same period of 2023. As mentioned above, GAVRETO became commercially available from Rigel in June 2024. Contract revenue from collaborations consisted of $17.5 million from Kissei related to an upfront fee from sublicensing olutasidenib and delivery of drug supplies, as well as $5.5 million from Grifols and $0.2 million from Medison related to delivery of drug supplies and earned royalties. Total costs and expenses were $114.1 million compared to $103.5 million for the same period of 2023. The increase in costs and expenses was mainly due to higher cost of product sales driven primarily by increased products sales, a sublicensing revenue fee to Forma, increased royalties and amortization of intangible assets. In addition, there were increases in personnel-related costs, stock-based compensation expense and commercial-related expenses. These increases were partially offset by decreased research and development costs due to the timing of clinical trial activities related to R289, the company's dual IRAK 1/4 inhibitor program, as well as reduced trial activities related to the completed Phase 3 clinical trials of fostamatinib in patients with COVID-19 and in patients with warm antibody hemolytic anemia (wAIHA). Rigel reported net income of $3.1 million, or $0.18 basic and diluted per share, compared to a net loss of $25.8 million, or $1.49 basic and diluted per share, for the same period of 2023. As discussed above, the share and per share amounts for the prior period have been restated to reflect the 1-for-10 reverse stock split on a retroactive basis for the periods presented. Cash, cash equivalents and short-term investments as of September 30, 2024 was $61.1 million, compared to $49.1 million as of June 30, 2024, and $56.9 million as of December 31, 2023. Conference Call and Webcast with Slides Today at 4:30pm Eastern Time Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time). Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company's website at . The webcast will be archived and available for replay after the call via the Rigel website. About ITP In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body's own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP. About AML Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that there will be about 20,800 new cases in the United States, most in adults, in 2024.2 Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.3 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.4 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need. About NSCLC It is estimated that over 230,000 adults in the U.S. will be diagnosed with lung cancer in 2024. Lung cancer is the leading cause of cancer death in the U.S, with NSCLC being the most common type accounting for 80-85% of all lung cancer diagnoses.5 RET fusions are implicated in approximately 1-2% of patients with NSCLC.6 About TAVALISSE® TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Please click here for Important Safety Information and Full Prescribing Information for TAVALISSE. About REZLIDHIA® REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. Please click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING, for REZLIDHIA. About GAVRETO® GAVRETO is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).* *Thyroid indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Please click here for Important Safety Information and Full Prescribing Information for GAVRETO. To report side effects of prescription drugs to the FDA, visit or call 1-800-FDA-1088 (800-332-1088). TAVALISSE, REZLIDHIA and GAVRETO are registered trademarks of Rigel Pharmaceuticals, Inc. About Rigel Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in South San Francisco, California. For more information on Rigel, the Company's marketed products and pipeline of potential products, visit . R289 is an investigational compound not approved by the FDA. The American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Revised June 5, 2024. Accessed June 30, 2024: Leukaemia Care. Relapse in Acute Myeloid Leukaemia (AML). Version 3. Reviewed October 2021. Accessed June 30, 2024: Thol F, Schlenk RF, Heuser M, Ganser A. How I treat refractory and early relapsed acute myeloid leukemia. Blood (2015) 126 (3): 319-27. Accessed June 30, 2024. doi: The American Cancer Society. Key Statistics for Lung Cancer. Revised January 29, 2024. Accessed June 30, 2024: Kato, S. et al. RET Aberrations in Diverse Cancers: Next-Generation Sequencing of 4,871 Patients. Clin Cancer Res. 2017;23(8):1988-1997 doi: 10.1158/1078-0432.CCR-16-1679 Forward Looking Statements This press release contains forward-looking statements relating to, among other things, expected commercial and financial results, expectations for developing and commercializing REZLIDHIA in certain international markets, study results relating to safety and tolerability of R289 for the treatment of lower-risk myeloid dysplastic syndrome, expectations for development of Rigel's commercial portfolio and hematology and oncology pipeline, and expectations for Rigel's partnering and collaboration efforts. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements can be identified by words such as "plan", "potential", "may", "look to", "expects", "will", "initial", and similar expressions in reference to future periods. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Rigel's current beliefs, expectations, and assumptions and hence they inherently involve significant risks, uncertainties and changes in circumstances that are difficult to predict and many of which are outside of our control. Therefore, you should not rely on any of these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the commercialization and marketing of fostamatinib, olutasidenib and pralsetinib; risks that the FDA, European Medicines Agency, PMDA or other regulatory authorities may make adverse decisions regarding fostamatinib, pralsetinib or olutasidenib; risks that clinical trials may not be predictive of real-world results or of results in subsequent clinical trials; risks that fostamatinib, pralsetinib or olutasidenib may have unintended side effects, adverse reactions or incidents of misuses; the availability of resources to develop Rigel's product candidates; market competition; as well as other risks detailed from time to time in Rigel's reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended June 30, 2024 and subsequent filings. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. Rigel does not undertake any obligation to update forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise, and expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein, except as required by law. Contact for Investors & Media: Investors: Rigel Pharmaceuticals, Inc. 650.624.1232 [email protected] Media: David Rosen Argot Partners 646.461.6387 [email protected] SOURCE Rigel Pharmaceuticals, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

License out/inClinical ResultPhase 1Drug ApprovalASH

29 Oct 2024

·www.globenewswire.com

Grifols Receives FDA Approval to Treat Surgical Bleeding in Pediatric Patients With its Fibrin Sealant Solution

Younger patients in the U.S. can now benefit from Grifols Fibrin Sealant (FS) and its positive effect on surgical outcomes, including a fast time to hemostasisGrifols surgical bleeding management treatments form part of an increasingly robust portfolio of innovative therapeutics enhancing the health and well-being of patients BARCELONA, Spain, Oct. 29, 2024 (GLOBE NEWSWIRE) -- Grifols (MCE:GRF, MCE:GRF.P, NASDAQ:GRFS), a global healthcare company and leading manufacturer of plasma-derived medicines, today announced that its plasma-protein based fibrin sealant (FS) for controlling surgical bleeding has received approval from the United States Food and Drug Administration (FDA) for pediatric patients. The U.S. indication for children and adolescents extends the availability of FS, which is already approved for this patient segment in Europe, in addition to adults. During surgery Grifols FS promotes hemostasis and tissue sealing, resulting in reduced blood loss and potentially fewer complications. Grifols FS biosurgery treatment is commercialized as VISTASEAL™ in the U.S. and Canada, and VERASEAL™ in Europe and elsewhere. Both brands are marketed and distributed by Johnson & Johnson MedTech, as part of a strategic collaboration between the two companies announced in 2019. Grifols FS combines two plasma proteins, fibrinogen and thrombin, and is applied with Johnson & Johnson MedTech’s airless spray technology to rapidly form clots. The FS solution is now available in 18 countries. In early 2023, Grifols announced that it had met all primary and secondary endpoints of its phase 3b study evaluating the administration of Grifols FS to pediatric patients, defined as those not having reached 18 years of age. Researchers conducted a global prospective, randomized, active-controlled, single-blind, parallel group clinical trial designed to evaluate the safety and efficacy of the FS as an adjunct to hemostasis during surgery in pediatric patients (compared with an active control). The study included a total of 178 patients enrolled and treated across 18 recruitment centers. A greater than 95% efficacy rate was achieved in both treatment arms, with hemostasis within four minutes of application. In addition, Grifols FS demonstrated a good safety and tolerability profile, as the distribution of adverse events was comparable between arms. “Developing innovative biosurgery solutions reflects Grifols’ ability to provide patients with more medicines across many therapeutic areas to enhance their well-being,” said Joerg Schuettrumpf, Grifols Chief Scientific Innovation Officer. It’s estimated that between roughly one-third and two-thirds of open surgeries experience disruptive bleeding,1 while challenging and uncontrollable bleeding during surgery is associated with high mortality rates.1,2 About VISTASEAL VISTASEAL is a single-use product that uses a combination of human fibrinogen and human thrombin to assist with mild to moderate bleeding control when standard surgical techniques such as suture or cautery are ineffective. The sealant is applied in a thin layer over the bleeding tissue in order to generate a cross-linked fibrin clot to achieve hemostasis. VISTASEAL can be utilized in high-risk patients when there are concerns regarding coagulopathy, antiplatelets, anticoagulants, and friable tissue. VISTASEAL™ Fibrin Sealant (Human) IMPORTANT SAFETY INFORMATION INDICATIONVISTASEAL™, a fibrin sealant, is indicated as an adjunct to hemostasis for mild to moderate bleeding in patients undergoing surgery when control of bleeding by standard surgical techniques (such as suture, ligature, and cautery) is ineffective or impractical. VISTASEAL™ is effective in heparinized patients. CONTRAINDICATIONSDo not inject directly into the circulatory system. Do not use for the treatment of severe or brisk arterial bleeding.Do not use in patients with history of anaphylaxis or severe systemic reactions to human blood products.Do not use VISTASEAL™ for spraying unless the minimum recommended distance from the applicator tip to the bleeding site can be achieved. WARNINGS AND PRECAUTIONS Thromboembolic events may occur if VISTASEAL™ is administered intravascularly. Hypersensitivity reactions can occur. May carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. ADVERSE REACTIONSThe most common adverse reactions (reported in >1% of patients) were procedural pain, and nausea. For complete indications, contraindications, warnings, precautions, and adverse reactions, please reference full package insert.MEDIA CONTACTS: Grifols Press Office media@grifols.comTel. +34 93 571 00 02 SpainDuomo ComunicaciónTel.: +34 91 311 92 89 – +34 91 311 92 90Raquel Lumbreras (Tel. +34 659 572 185)Raquel_lumbreras@duomocomunicacion.comBorja Gómez (Tel. + 34 659 572 185)Borja_gomez@duomocomunicacion.com InvestorsInvestors Relations & Sustainabilityinversores@grifols.com - investors@grifols.comTel. +34 93 571 02 21 About Grifols Grifols is a global healthcare company founded in Barcelona in 1909 committed to improving the health and well-being of people around the world. A leader in essential plasma-derived medicines and transfusion medicine, the company develops, produces, and provides innovative healthcare services and solutions in more than 110 countries. Patient needs and Grifols’ ever-growing knowledge of many chronic, rare and prevalent conditions, at times life-threatening, drive the company’s innovation in both plasma and other biopharmaceuticals. Grifols is focused on treating conditions across a broad range of therapeutic areas: immunology, hepatology and intensive care, pulmonology, hematology, neurology, and infectious diseases. A pioneer in the plasma industry, Grifols continues to grow its network of donation centers, the world’s largest with over 390 across North America, Europe, Africa and the Middle East, and China. As a recognized leader in transfusion medicine, Grifols offers a comprehensive portfolio of solutions designed to enhance safety from donation to transfusion, in addition to clinical diagnostic technologies. It provides high-quality biological supplies for life-science research, clinical trials, and for manufacturing pharmaceutical and diagnostic products. The company also supplies tools, information and services that enable hospitals, pharmacies and healthcare professionals to efficiently deliver expert medical care. Grifols, with more than 23,000 employees in more than 30 countries and regions, is committed to a sustainable business model that sets the standard for continuous innovation, quality, safety, and ethical leadership. The company’s class A shares are listed on the Spanish Stock Exchange, where they are part of the Ibex-35 (MCE:GRF). Grifols non-voting class B shares are listed on the Mercado Continuo (MCE:GRF.P) and on the U.S. NASDAQ through ADRs (NASDAQ:GRFS). For more information about Grifols, please visit grifols.com LEGAL DISCLAIMERThe facts and figures contained in this report that do not refer to historical data are “future projections and assumptions.” Words and expressions such as “believe,” “hope,” “anticipate,” “predict,” “expect,” “intend,” “should,” “will seek to achieve,” “it is estimated,” “future” and similar expressions, in so far as they relate to the Grifols group, are used to identify future projections and assumptions. These expressions reflect the assumptions, hypotheses, expectations and predictions of the management team at the time of writing this report, and these are subject to a number of factors that mean that the actual results may be materially different. The future results of the Grifols group could be affected by events relating to its own activities, such as a shortage of supplies of raw materials for the manufacture of its products, the appearance of competitor products on the market, or changes to the regulatory framework of the markets in which it operates, among others. At the date of compiling this report, the Grifols group has adopted the necessary measures to mitigate the potential impact of these events. Grifols, S.A. does not accept any obligation to publicly report, revise or update future projections or assumptions to adapt them to events or circumstances subsequent to the date of writing this report, except where expressly required by the applicable legislation. This document does not constitute an offer or invitation to buy or subscribe shares in accordance with the provisions of the following Spanish legislation: Royal Legislative Decree 4/2015, of 23 October, approving recast text of Securities Market Law; Royal Decree Law 5/2005, of 11 March and/or Royal Decree 1310/2005, of 4 November, and any regulations developing this legislation. In addition, this document does not constitute an offer of purchase, sale or exchange, or a request for an offer of purchase, sale or exchange of securities, or a request for any vote or approval in any other jurisdiction. The information included in this document has not been verified nor reviewed by the external auditors of the Grifols group. 1 Corral M, Ferko N, Hollmann S, Broder MS, Chang E. Health and economic outcomes associated with uncontrolled surgical bleeding: a retrospective analysis of the Premier Perspectives Database. Clinicoecon Outcomes Res. 2015;7:409-421. doi:10.2147/CEOR.S863692 Marietta M, Facchini L, Pedrazzi P, Busani S, Torelli G. Pathophysiology of bleeding in surgery. Transplant Proc. 2006;38(3):812-814. doi:10.1016/j.transproceed.2006.01.047

Phase 3Clinical ResultDrug Approval

22 Oct 2024

·www.globenewswire.com

Grifols Partners with BARDA for Proof-of-Concept Testing of Ocular Immunoglobulin as Treatment for Sulfur Mustard-Induced Eye Injury

Initiative will test Grifols ocular surface immunoglobulin (OSIG) eye drops to evaluate their nonclinical efficacy in neutralizing symptoms from exposure to sulfur mustard, a chemical warfare agent A successful preclinical study of this potentially innovative therapeutic could lead to an FDA license for one of the first medical treatments for sulfur mustard ocular injury Grifols also plans to start a phase 2 clinical trial of an OSIG for dry eye disease in the first half of 2025, part of the company’s broadening innovation pipeline to enhance the lives of patients BARCELONA, Spain, Oct. 22, 2024 (GLOBE NEWSWIRE) -- Grifols (MCE:GRF, MCE:GRF.P, NASDAQ:GRFS), a global healthcare company and leading manufacturer of plasma-derived medicines, today announced it has established a partnership with the Biomedical Advanced Research and Development Authority (BARDA) to test investigational ocular surface immunoglobulin (OSIG) eye drops for their ability to treat ocular damage from sulfur mustard exposure. BARDA is part of the Administration for Strategic Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS). If the preclinical evaluation is successful, the United States Food and Drug Administration (FDA) could eventually license what would be one of the first medical treatments to counteract the long-term effects of sulfur mustard ocular injury. Sulfur mustard, sometimes referred to as mustard gas, is a chemical warfare agent that reacts rapidly with ocular tissue and can cause pain, photophobia and mustard gas keratopathy, a corneal injury that can lead to blindness. Grifols will develop a treatment for sulfur mustard ocular exposure by repurposing an investigational OSIG therapeutic currently in development for dry eye disease (DED). The nonclinical studies, conducted in partnership with BARDA, may provide evidence for the anti-inflammatory and immunomodulatory properties of OSIG and its ability to alleviate the long-term effects of sulfur mustard exposure. Specifically, research will investigate how OSIG’s anti-inflammatory properties can prevent the immune system from mistakenly attacking self-antigens, which in this case are proteins modified due to exposure to sulfur mustard. In cases of sulfur mustard exposure, the immune system can errantly target these self-antigens. By neutralizing the immune response caused by the self-antigens, OSIG could help protect ocular tissue and support recovery in people exposed to sulfur mustard. Grifols announced in March 2023 a collaboration with Chicago-based Selagine, which focuses on developing novel therapeutics for ocular diseases, to treat DED with immunoglobulin eye drops. The potential treatment, expected to enter clinical development in the first half of 2025, would become the first immunoglobulin medicine indicated for DED, which affects more than 100 million people globally. “Grifols is applying its leadership in immunoglobulins, a powerful class of medicines with a unique mechanism of action, to develop safe, effective and readily available IG-based ocular treatments to alleviate conditions that seriously impact people’s eyesight and quality of life,” said Joerg Schuettrumpf, Grifols Chief Scientific Innovation Officer. “We continue to build an innovation pipeline focused on providing more and better treatments for patients.” Grifols’ work with BARDA to create an OSIG to neutralize the effects of sulfur mustard ocular exposure comes shortly after the company’s GigaGen subsidiary announced a contract with BARDA, worth up to $135 million over six years, to develop recombinant polyclonal antibody therapies for biothreats including botulinum neurotoxins. This project has been supported in whole or in part with federal funds from the HHS, ASPR and BARDA under contract number 75A50124C00050. MEDIA CONTACTS: Grifols Press Office media@grifols.comTel. +34 93 571 00 02 SpainDuomo ComunicaciónTel.: +34 91 311 92 89 – +34 91 311 92 90Raquel Lumbreras (Tel. +34 659 572 185)Raquel_lumbreras@duomocomunicacion.com Borja Gómez (Tel. + 34 659 572 185)Borja_gomez@duomocomunicacion.com InvestorsInvestors Relations & Sustainabilityinversores@grifols.com - investors@grifols.comTel. +34 93 571 02 21 About Grifols Grifols is a global healthcare company founded in Barcelona in 1909 committed to improving the health and well-being of people around the world. A leader in essential plasma-derived medicines and transfusion medicine, the company develops, produces, and provides innovative healthcare services and solutions in more than 110 countries. Patient needs and Grifols’ ever-growing knowledge of many chronic, rare and prevalent conditions, at times life-threatening, drive the company’s innovation in both plasma and other biopharmaceuticals. Grifols is focused on treating conditions across a broad range of therapeutic areas: immunology, hepatology and intensive care, pulmonology, hematology, neurology, and infectious diseases. A pioneer in the plasma industry, Grifols continues to grow its network of donation centers, the world’s largest with over 390 across North America, Europe, Africa and the Middle East, and China. As a recognized leader in transfusion medicine, Grifols offers a comprehensive portfolio of solutions designed to enhance safety from donation to transfusion, in addition to clinical diagnostic technologies. It provides high-quality biological supplies for life-science research, clinical trials, and for manufacturing pharmaceutical and diagnostic products. The company also supplies tools, information and services that enable hospitals, pharmacies and healthcare professionals to efficiently deliver expert medical care. Grifols, with more than 23,000 employees in more than 30 countries and regions, is committed to a sustainable business model that sets the standard for continuous innovation, quality, safety, and ethical leadership. The company’s class A shares are listed on the Spanish Stock Exchange, where they are part of the Ibex-35 (MCE:GRF). Grifols non-voting class B shares are listed on the Mercado Continuo (MCE:GRF.P) and on the U.S. NASDAQ through ADRs (NASDAQ:GRFS). For more information about Grifols, please visit grifols.com LEGAL DISCLAIMER The facts and figures contained in this report that do not refer to historical data are “future projections and assumptions”. Words and expressions such as “believe”, “hope”, “anticipate”, “predict”, “expect”, “intend”, “should”, “will seek to achieve”, “it is estimated”, “future” and similar expressions, in so far as they relate to the Grifols group, are used to identify future projections and assumptions. These expressions reflect the assumptions, hypotheses, expectations and predictions of the management team at the time of writing this report, and these are subject to a number of factors that mean that the actual results may be materially different. The future results of the Grifols group could be affected by events relating to its own activities, such as a shortage of supplies of raw materials for the manufacture of its products, the appearance of competitor products on the market, or changes to the regulatory framework of the markets in which it operates, among others. At the date of compiling this report, the Grifols group has adopted the necessary measures to mitigate the potential impact of these events. Grifols, S.A. does not accept any obligation to publicly report, revise or update future projections or assumptions to adapt them to events or circumstances subsequent to the date of writing this report, except where expressly required by the applicable legislation. This document does not constitute an offer or invitation to buy or subscribe shares in accordance with the provisions of the following Spanish legislation: Royal Legislative Decree 4/2015, of 23 October, approving recast text of Securities Market Law; Royal Decree Law 5/2005, of 11 March and/or Royal Decree 1310/2005, of 4 November, and any regulations developing this legislation. In addition, this document does not constitute an offer of purchase, sale or exchange, or a request for an offer of purchase, sale or exchange of securities, or a request for any vote or approval in any other jurisdiction. The information included in this document has not been verified nor reviewed by the external auditors of the Grifols group.

Immunotherapy

100 Deals associated with Human plasma-derived fibrinolysin (Grifols)

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Indication	Highest Phase	Country/Location	Organization	Date
Occlusion of artery	Phase 2	US	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	BE	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	BG	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	CZ	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	DE	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	IN	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	PE	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	PL	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	RO	Grifols Therapeutics, Inc.	01 Dec 2010
Occlusion of artery	Phase 2	RS	Grifols Therapeutics, Inc.	01 Dec 2010

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01222117 (Pubmed)	Phase 2	Occlusion of artery	174	150 mg plasmin without BOC	oavajduxjl(jvrnljyzhg) = qannsysawe pqafwvkvrq (hmesejdhod ) View more	-	01 Jan 2019
				rtPA without BOC	dfeenmmkxa(gsjzhwtjbj) = upkllttlxu ahmatfmlji (vxtwkfiqzj ) View more
NCT01014975 (CTgov)	Phase 1/2	Infarction, Middle Cerebral Artery \| Acute Ischemic Stroke	40	Plasmin (Human) (20 mg Plasmin (Human))	pywuqbntes(iqdgzpsnbx) = pstkhsfrvd vmazeidlmw (ujymnjqbse, psuhovatrk - esdxzhclkj) View more	-	21 Oct 2015
				Plasmin (Human) (40 mg Plasmin (Human))	pywuqbntes(iqdgzpsnbx) = kcqsorcimc vmazeidlmw (ujymnjqbse, gjfsxzbdid - yazywjxqbo) View more
ARVO2009	Not Applicable	Retinal Vein Occlusion	-	Intravitreal Plasmin Injection	kkydjzjabz(wkmecxuqvn) = lsreawxxat ejteadarqi (qmimjbztzu ) View more	-	01 Apr 2009
ARVO2007	Not Applicable	-	-	Plasmin + Hyaluronidase	uxpauuwzxm(nrvvrurpxd) = mnkqivtrzg zinzerwins (ibrqvwupik ) View more	-	01 May 2007
				Plasmin	uxpauuwzxm(nrvvrurpxd) = gcqgwlwgze zinzerwins (ibrqvwupik ) View more
AAO2004	Not Applicable	Proliferative retinopathy with diabetes mellitus	6	Plasmin	mnrfqvqojo(eakzebhntf) = ntkiemhyaq nzxznjxwyb (wzkbtavtji )	Positive	24 Oct 2004
				Plasmin	mnrfqvqojo(eakzebhntf) = hocnavxhje nzxznjxwyb (wzkbtavtji )
ARVO2002	Not Applicable	-	16	Plasmin 1U combined with hyaluronidase 20U	bzdbddammr(cmvdmxkkkd) = pmugcjsswa hjmfvqdebm (ayoozpdako )	Positive	01 Dec 2002
				Plasmin 1U alone	bzdbddammr(cmvdmxkkkd) = vrensiywgr hjmfvqdebm (ayoozpdako )

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