Human plasma-derived fibrinolysin (Grifols)

DUBLIN--( BUSINESS WIRE )--The "The Corporate Reputation of Pharma in 2023/2024 - The Patient Perspective - USA Edition - The Views of 314 USA Patient Groups" report has been added to ResearchAndMarkets.com's offering. This review contains the results of a November 2023 to late-February 2024 survey, answered by 314 US-based patient groups. The 314 respondent US patient groups specialised in a wide range of therapy areas - the largest representation (113 respondent patient groups) being cancer patient groups. The 314 stated that, collectively, they had actively supported and served some 12 million US patients during 2023/2024. Continue reading, for details about: The headline US industry-wide results of the 2023 survey. The performance of the individual pharma companies included in the 2023 US analysis. Summary of Results Industry Wide Several issues in 2023 may have influenced the opinions of US patient groups responding to the 2023 'Corporate Reputation of Pharma' survey, including: the affordability of prescription drugs, and shortages of prescription drugs. On the latter subject, drug shortages reached an all-time high in the US during the first three months of 2024 (which included the final two months of the 2023 'Corporate Reputation' survey period). The perception of the pharma industry's reputation among respondent US patient groups declined in 2023, with 57% rating the industry's reputation as "Excellent" or "Good", compared with 65% saying the same in 2022. The year before that, the COVID-affected year of 2021, had seen the industry's positive rating peak at 67%. Between 2022 and 2023, sharp declines were reported by respondent US patient groups for: the quality of pharma's patient-group relations; its provision of patient information; and its ability to improve access to medicines. And, despite pharma's commitment to involving patients more in R&D, only 35% of 2023's respondent US patient groups rated the industry "Excellent" or "Good" at this - the first such decline in five years. Key Insights: The corporate reputation of pharma in the US, 2019-2023 Percentage of respondent US patient groups stating "Excellent" or "Good" How good or bad the pharma industry was at carrying out specific activities in the US, 2023 v. 2022 % of respondent US patient groups stating "Excellent" or "Good" What US patient groups say about pricing The US runs a complex pricing-and-reimbursement system - which also happens to be in a state of considerable flux. 2023's respondent US patient groups urge all companies to be as transparent as possible, and to work with other healthcare stakeholders in supporting patients who cannot afford new medicines (and who do not qualify for federal funding). With new medicines averaging $300,000 list price in the US in 2023 - an increase of more than 35% over 20223 - the issue of pricing is likely to remain a top patient concern in the US for the foreseeable future. "Somehow, the medications are available in other countries for much less, but we are billed/paying co-pays and deductibles that far outpace the costs of other health systems. So why? Let's talk about it." - National breast-cancer patient group, USA "Think about patients, when making pipeline decisions - even in light of the IRA [Inflation Reduction Act]. Patients before profits." - National lung-cancer patient group, USA "Companies should not treat patient engagement as if they are ticking off boxes. There should be meaningful engagement, co-led by patients and community organizations, that benefits everyone, and shows commitment to the patient community. Companies should reach out early in the R&D process." - National hepatitis patient group, USA "It is extremely difficult to maintain relationships, due to constant changes of [company] employees." - National rare-disease patient groups, USA "From our limited outreach to the pharma industry, I understand that they receive many requests, and have many layers of compliance. However, it is difficult, if not impossible, to understand the best way for patient-advocacy groups to work with pharma, and retain their independence." - National skin-conditions patient group, USA Companies included in the US element of the 2023 'Corporate Reputation' survey The 37 companies featured in the US arm of the 2023 'Corporate Reputation of Pharma' survey were selected on the criteria of size of revenue, or, by request from companies or patient groups. They include: AbbVie Amgen Astellas Pharma AstraZeneca Bayer Biogen Biomarin Boehringer Ingelheim Bristol Myers Squibb Chiesi Farmaceutici CSL Behring Daiichi Sankyo Eisai Eli Lilly Ferring Gilead Sciences Grifols GSK Ipsen Janssen Lundbeck Menarini Merck & Co Merck KGaA/EMD Serono Novartis Novo Nordisk Octapharma Otsuka Pfizer PTC Therapeutics Roche/Genentech Sanofi Servier Takeda UCB Vertex ViiV Healthcare Companies are assessed on issues of importance to patient groups Individual Company Findings for the USA, 2023 - and the Fastest Risers in the Rankings, 2023 v. 2022 The top-three rankings in the US in 2023 - out of all 37 companies As assessed by respondent US patient groups familiar with the company: Servier, 1st ViiV healthcare, 2nd Amgen, 3rd The top-three rankings in the US in 2023 (out of 32 companies) As assessed by respondent US patient groups working with the company: ViiV Healthcare, 1st Servier, 2nd Gilead Sciences, 3rd The top-three rankings in the US in 2023 - out of 15 'big-pharma' companies As assessed by respondent US patient groups familiar with the company: Amgen, 1st Gilead Sciences, 2nd Pfizer, 3rd As assessed by respondent US patient groups working with the company: Gilead Sciences, 1st Amgen, 2nd Roche/Genentech, 3rd Key Insights: The companies rising the most in the upper rankings in the US (out of all 37 companies), 2023 v. 2022, as assessed by respondent US patient groups familiar with the company. The companies rising the most in the upper rankings in the US (out of 32 companies), 2023 v. 2022, as assessed by respondent US patient groups working with the company. For more information about this report visit https://www.researchandmarkets.com/r/qouqv6 About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Second quarter total revenue of $36.8 million, which includes TAVALISSE® net product sales of $26.4 million, REZLIDHIA® net product sales of $5.2 million and GAVRETO® net product sales of $1.9 million Successfully completed NDA transfer of GAVRETO for the treatment of RET fusion-positive metastatic non-small cell lung cancer and advanced or metastatic thyroid cancer, with product available from Rigel beginning June 27, 2024 Conference call and webcast scheduled today at 4:30 p.m. Eastern Time SOUTH SAN FRANCISCO, Calif., Aug. 6, 2024 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today reported financial results for the second quarter ended June 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. "In the second quarter, we advanced key strategic initiatives including the successful transfer of GAVRETO to our commercial portfolio, enabling us to bring this important treatment option to current and newly prescribed patients without interruption," said Raul Rodriguez, Rigel's president and CEO. "The addition of GAVRETO, combined with record revenues from TAVALISSE and REZLIDHIA, has allowed us to approach net income break even. We look to maintain our financial discipline as we expand our commercial reach and advance our development programs." Second Quarter 2024 Business Update Commercial Update Commercial strength continues with record TAVALISSE and REZLIDHIA bottles shipped to patients and clinics and total bottles sold. In June 2024, Rigel announced the completion of the transfer of the New Drug Application (NDA) for the U.S. rights to GAVRETO. GAVRETO became commercially available from Rigel in the U.S. beginning June 27, 2024, ahead of the company's July 1 target for commercial availability. The following table summarizes total bottles shipped for the second quarter: Clinical and Development Update Rigel continues to advance its Phase 1b clinical trial evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R2891, a novel and selective IRAK1/4 inhibitor, in patients with relapsed/refractory lower-risk myelodysplastic syndrome (LR-MDS). Enrollment in the fourth dose level (250 mg twice daily) of the trial is underway. Preliminary data are expected by the end of 2024. In early August, The University of Texas MD Anderson Cancer Center, with Rigel's support, opened enrollment for a Phase 1b/2 trial of decitabine and venetoclax in combination with olutasidenib in patients with IDH1-mutated AML (NCT06445959). This is the first trial in Rigel's multi-year strategic development collaboration with MD Anderson. The Phase 1b part of the trial seeks to determine the safety and tolerability and recommended Phase 2 dose of decitabine and venetoclax in combination with olutasidenib. The primary objective of the Phase 2 part of the trial is to determine the complete remission rate in both newly diagnosed and relapsed/refractory patients. In late July, City of Hope National Medical Center opened enrollment for a pilot trial of olutasidenib as maintenance therapy following allogeneic hematopoietic cell transplantation (HCT). The primary objective of the trial is to evaluate the safety and tolerability of olutasidenib as post-HCT maintenance therapy in patients with mIDH1 AML, myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). Rigel presented the final long-term efficacy data from the registrational Phase 2 trial of REZLIDHIA in heavily pretreated patients with R/R mIDH1 AML, including those receiving prior venetoclax, in an oral presentation and 4 posters at the EHA2024 Hybrid Congress. In addition, the company presented 3 posters at the 2024 ASCO Annual Meeting, which included safety and efficacy of olutasidenib treatment in elderly patients with R/R mIDH1 AML and an overview of the Phase 1b trial of R289 in patients with LR-MDS. Dr. Jorge E. Cortes, Director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, and Phase 2 trial investigator, was published in the Expert Review of Hematology in May outlining the drug profile and summarizing key safety and efficacy data for olutasidenib, including in patients previously treated with venetoclax or ivosidenib. Second Quarter 2024 and Year-To-Date Financial Update For the second quarter ended June 30, 2024, total revenues were $36.8 million, consisting of $26.4 million in TAVALISSE net product sales, $5.2 million in REZLIDHIA net product sales, $1.9 million in GAVRETO net product sales, and $3.4 million in contract revenue from collaborations. TAVALISSE net product sales grew 24% compared to $21.3 million in the same period of 2023. REZLIDHIA net product sales grew 102% compared to $2.6 million in the same period of 2023. GAVRETO became commercially available from Rigel on June 27, 2024. Contract revenue from collaborations consisted of $2.2 million from Kissei Pharmaceutical Co., Ltd. (Kissei) related to delivery of drug supplies, $1.1 million from Grifols S.A. (Grifols) related to earned royalties, and $0.1 million from Medison Pharma Trading AG (Medison) related to delivery of drug supplies and earned royalties. Total costs and expenses were $36.4 million compared to $32.2 million for the same period of 2023. The increase in costs and expenses was partly due to higher cost of product sales, driven primarily by higher amortization of intangibles and royalties, increased personnel-related costs, and increased research and development costs due to the progress of clinical activities, including R289, the company's IRAK 1/4 inhibitor program. Rigel reported a net loss of $1.0 million, or $0.06 per basic and diluted share, compared to a net loss of $6.6 million, or $0.38 per basic and diluted share, for the same period of 2023. The basic and diluted share and per share amounts have been restated to reflect the 1-for-10 reverse stock split effected on June 27, 2024 on a retroactive basis for all periods presented. For the six months ended June 30, 2024, total revenues were $66.4 million, consisting of $47.5 million in TAVALISSE net product sales, $10.0 million in REZLIDHIA net product sales, $1.9 million in GAVRETO net product sales, and $6.9 million in contract revenue from collaborations. TAVALISSE net product sales grew 9% compared to $43.6 million in the same period of 2023. REZLIDHIA net product sales grew 150% compared to $4.0 million in the same period of 2023. As mentioned above, GAVRETO became commercially available from Rigel on June 27, 2024. Contract revenue from collaborations consisted of $4.5 million from Kissei related to delivery of drug supplies, $2.2 million from Grifols related to earned royalties, and $0.2 million from Medison related to delivery of drug supplies and earned royalties. Total costs and expenses were $72.9 million compared to $70.9 million for the same period of 2023. The increase in costs and expenses was partly due to higher cost of product sales, driven primarily by higher amortization of intangibles and royalties, increased personnel-related costs and higher stock-based compensation expenses mainly from performance awards. 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 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 warm antibody hemolytic anemia (wAIHA). Rigel reported a net loss of $9.3 million, or $0.53 per basic and diluted share, compared to a net loss of $20.1 million, or $1.16 per basic and diluted share, for the same period of 2023. As discussed above, the share and per share amounts have been restated to reflect the 1-for-10 reverse stock split on a retroactive basis for all periods presented. Cash, cash equivalents and short-term investments as of June 30, 2024 was $49.1 million, compared to $49.6 million as of March 31, 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 related to the potential and market opportunity of olutasidenib as a therapeutic for R/R AML and other conditions, the commercialization of fostamatinib for cITP, the commercialization of pralsetinib for the treatment of non-small cell lung cancer and advanced thyroid cancer, Rigel's ability to further develop its clinical stage product candidates and Rigel's partnering and collaboration efforts, as well as the progress of the Phase 1b clinical trial of R289 for the treatment of lower-risk myeloid dysplastic syndrome, olutasidenib's evaluation in acute myeloid leukemia (AML), including in patients receiving prior venetoclax, and in other hematologic cancers, and including olutasidenib as a maintenance therapy following allogeneic hematopoietic cell transplantation, and the use of decitabine and venetoclax in combination with olutasidenib for newly diagnosed or relapsed/refractory participants with IDH1-mutated myeloid malignancy. 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" 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 March 31, 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 212.600.1902 [email protected] SOURCE Rigel Pharmaceuticals, Inc.