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Yahoo
03-06-2025
- Business
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PharmaMar presents at ASCO that the Zepzelca®(lurbinectedin) and atezolizumab (Tecentriq®) combination significantly improves survival as first-line maintenance therapy for extensive-stage small cell lung cancer
First-line maintenance combination therapy reduced the risk of disease progression or death by 46%, with a median overall survival of 13.2 months vs 10.6 months for atezolizumab alone from the point of randomization First Phase 3 study to demonstrate statistically significant and clinically meaningful improvements in both progression-free and overall survival in ES-SCLC first-line maintenance PharmaMar has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for lurbinectedin Results presented at the ASCO 2025 Annual Meeting and simultaneously published in The Lancet PharmaMar to host a webcast with Key Opinion Leaders on Thursday, June 12th, to review lurbinectedin data MADRID, June 3, 2025 /PRNewswire/ -- PharmaMar (MSE: PHM) has announced positive results from the Phase 3 IMforte study of Zepzelca® (lurbinectedin) in combination with atezolizumab (Tecentriq®) as a first-line maintenance treatment for people with extensive-stage small cell lung cancer (ES-SCLC), following induction therapy with carboplatin, etoposide and atezolizumab. The study met both primary endpoints, demonstrating statistically significant improvements in progression-free survival (PFS) and overall survival (OS) compared to atezolizumab alone. IMforte is the first global Phase 3 trial to demonstrate clinically meaningful PFS and OS benefits in the first-line maintenance setting for ES-SCLC and supports maintenance therapy with lurbinectedin plus atezolizumab as a new standard of care for patients. The data were presented today in an oral session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago and published simultaneously in The Lancet. Data from the trial served as the basis for the supplemental New Drug Application (sNDA) submission to the FDA by Jazz Pharmaceuticals, as well as for the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) by PharmaMar. Following induction therapy with carboplatin, etoposide and atezolizumab, patients who did not have disease progression were randomized to receive lurbinectedin plus atezolizumab or atezolizumab alone. From the point of randomization, the median PFS was 5.4 months for the lurbinectedin plus atezolizumab combination versus 2.1 months for atezolizumab alone (stratified HR = 0.54, 95% CI: 0.43–0.67; p < 0.0001), and median OS was 13.2 months versus 10.6 months (stratified hazard ratio [HR] = 0.73; 95% CI: 0.57–0.95; p = 0.0174). The combination reduced the risk of disease progression or death by 46% and the risk of death by 27% compared to atezolizumab alone. The lurbinectedin plus atezolizumab combination had no new or unexpected safety signals. "Small cell lung cancer is an aggressive and devastating disease; at the time of diagnosis, the large majority of patients have already progressed to extensive-stage disease and only one out of five survive longer than two years," said Luis Paz-Ares, M.D., Ph.D., head of medical oncology at the Hospital Universitario 12 de Octubre in Madrid, Spain, and the IMforte trial principal investigator. "The IMforte results are very encouraging showing a potentially practice-changing option that could improve survival for patients with a very high unmet need." "Upon approval, patients will have access to lurbinectedin earlier in the treatment paradigm, where there's potential to increase duration of response in a broader patient population, delaying disease progression and extending survival," said Javier Jiménez Jiménez, Chief Medical Officer of PharmaMar. Each year, approximately 63,000 to 72,000 new cases of small cell lung cancer (SCLC) are reported in Europe. Most of these patients are diagnosed with extensive stage disease, which is aggressive and often difficult to treat, with poor prognosis.[i],[ii],[iii] Legal warning This press release does not constitute an offer to sell or the solicitation of an offer to buy securities, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction. Phase 3 IMforte Trial Results These primary results are from the global Phase 3 IMforte trial, which evaluated lurbinectedin plus atezolizumab as a first-line maintenance therapy in patients with ES-SCLC. 483 patients were randomized after completion of 4 cycles of induction therapy with atezolizumab plus carboplatin and etoposide. From the point of randomization, the median OS for the lurbinectedin plus atezolizumab regimen was 13.2 months versus 10.6 months for atezolizumab alone (stratified hazard ratio [HR] = 0.73; 95% CI: 0.57–0.95; p = 0.0174). From the point of randomization, the median PFS by independent assessment was 5.4 months versus 2.1 months, respectively (stratified HR = 0.54, 95% CI: 0.43–0.67; p < 0.0001). Treatment duration for patients in the lurbinectedin plus atezolizumab arm was twice as long as the atezolizumab arm, with a median maintenance treatment duration of 4.2 months versus 2.1 months, respectively. The lurbinectedin plus atezolizumab combination as maintenance therapy was generally well tolerated with no new safety signals identified. In the lurbinectedin plus atezolizumab and atezolizumab arms, respectively, treatment-related adverse events (TRAEs) occurred in 83.5% versus 40.0% of patients, with Grade 3-4 TRAEs in 25.6% versus 5.8% and Grade 5 TRAEs in 0.8% (two patients with sepsis and febrile neutropenia) versus 0.4% (one patient with sepsis). AEs led to treatment discontinuation in 6.2% of patients in the lurbinectedin plus atezolizumab arm and 3.3% of patients in the atezolizumab arm. PharmaMar will host a Key Opinion Leader webcast on June 12nd to review lurbinectedin data. The webcast will include a discussion panel of Dr. Martin Wermke from TU Dresden and Dr. Nicolas Girard from Institut Curie. The webcast may be accessed from the Investors section at About the IMforte Phase 3 Trial IMforte (NCT05091567) is an ongoing Phase 3, randomized, multicenter maintenance trial evaluating the efficacy, safety and pharmacokinetic of lurbinectedin plus atezolizumab, compared with standard-of-care first-line maintenance with atezolizumab alone, in adults (≥18 years) with ES-SCLC, following induction therapy with carboplatin, etoposide and atezolizumab. The primary endpoints for this study are OS and IRF-assessed PFS in the maintenance phase. The trial consists of two phases: an induction phase and a maintenance phase. Participants were required to have an ongoing response or stable disease per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 after the induction phase of four cycles of carboplatin, etoposide, and atezolizumab to be considered for eligibility screening for the maintenance phase. Eligible participants were randomized in a 1:1 ratio to receive either lurbinectedin plus atezolizumab or atezolizumab in the maintenance phase. The trial is sponsored by Roche and co-funded by Jazz Pharmaceuticals. Additional information about the trial, including eligibility criteria and a list of clinical trial sites, can be found at: ( Identifier: NCT05091567). About PharmaMar PharmaMar is a biopharmaceutical company focused on the research and development of new oncology treatments, whose mission is to improve the healthcare outcomes of patients afflicted by serious diseases with our innovative medicines. The Company is inspired by the sea, driven by science, and motivated by patients with serious diseases to improve their lives by delivering novel medicines to them. PharmaMar intends to continue to be the world leader in marine medicinal discovery, development and innovation. PharmaMar has developed and now commercializes Yondelis® in Europe by itself, as well as Zepzelca® (lurbinectedin), in the US; and Aplidin® (plitidepsin), in Australia, with different partners. In addition, it has a pipeline of drug candidates and a robust R&D oncology program. PharmaMar has other clinical-stage programs under development for several types of solid cancers: lurbinectedin, ecubectedin, PM534 and PM54. Headquartered in Madrid (Spain), PharmaMar has subsidiaries in Germany, France, Italy, Belgium, Austria, Switzerland and The United States. PharmaMar also wholly owns Sylentis, a company dedicated to researching therapeutic applications of gene silencing (RNAi). To learn more about PharmaMar, please visit us at About Zepzelca® Zepzelca® (lurbinectedin), also known as PM1183, is an analog of the marine compound ET-736 isolated from the sea squirt Ecteinascidia turbinata in which a hydrogen atom has been replaced by a methoxy group. It is a selective inhibitor of the oncogenic transcription programs on which many tumors are particularly dependent. Together with its effect on cancer cells, lurbinectedin inhibits oncogenic transcription in tumor-associated macrophages, downregulating the production of cytokines that are essential for the growth of the tumor. Transcriptional addiction is an acknowledged target in those diseases, many of them lacking other actionable targets. [i] Cancer today. (s. f.). Alvarado-Lunda G, Morales-Espinosa D. Treatment for small cell lung cancer, where are we now? – A review. Transl Lung Cancer Res. 2016;5(1):26-38.[iii] SEER Explorer Lung and Bronchus Cancer, Recent Trends in SEER Incidence Rates, 2000-2016, by Age, Updated June 27, 2024. Accessed October 10, 2024. Photo - - View original content to download multimedia: Sign in to access your portfolio
Yahoo
02-06-2025
- Business
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MaaT Pharma Advances Toward Commercialization And Submits Marketing Authorization Application to the European Medicines Agency (EMA) for Xervyteg® (MaaT013) in Acute Graft-versus-Host Disease
MaaT Pharma submitted today a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its product candidate MaaT013, under the registered brand name of Xervyteg®. Xervyteg® has the potential if approved, to become the first microbiota therapeutic approved by the EMA and the first one in hemato-oncology globally. The MAA submitted to the EMA is based on data from the Pivotal ARES study, evaluating the safety and efficacy of Xervyteg® in adult patients with acute Graft-versus-Host Disease including gastro-intestinal involvement who received two prior lines of therapy and supported by data from the ongoing Early Access Program. MaaT Pharma prepares for a potential 2026 commercial launch through a strategic partnership to address this key unmet need in hemato-oncology. LYON, France, June 02, 2025--(BUSINESS WIRE)--Regulatory News: MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today announced the submission of the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its lead drug candidate MaaT013, under the registered brand name of Xervyteg®. If approved, the Marketing Authorization would establish Xervyteg® as the first microbiota therapeutic approved by the EMA, and the first one globally for a hematology indication. Xervyteg® would also be the first approved therapy for the treatment of acute Graft-versus-Host Disease including gastro-intestinal involvement (GI-aGvHD) following 2 prior lines of systemic therapy. "Submitting our MAA to the EMA marks a major regulatory milestone for MaaT Pharma and a meaningful advancement for patients with refractory aGvHD—a life-threatening complication of stem cell transplantation with no approved therapies," said Hervé Affagard, Co-founder and CEO of MaaT Pharma. "We are now closer to providing a much-needed treatment option and remain deeply committed to advancing immunomodulating microbiota technologies in hemato-oncology, where new solutions are urgently needed." While advancing toward the commercialization of Xervyteg® (if approved) in Europe, MaaT Pharma is also actively exploring strategic partnerships to ensure broad access in timely fashion. The Company has active discussions with experienced partners who share its mission of delivering meaningful advancements to patients. In parallel to the MAA submission, MaaT Pharma continues to provide access to Xervyteg® in Europe and the U.S. through its Early Access Program (EAP)1 for patients with aGvHD and other indications. In 2024, physician demand for Xervyteg® under the EAP (n=107) increased by 75% compared to 2023, driven by growing adoption across Europe and in the U.S. In France where the EAP first started, MaaT Pharma has captured 25% of the addressable market on a yearly basis in 2024. Overall, this position reflects the increasing recognition of Xervyteg® as a valuable treatment option for GI-aGvHD patients. aGvHD is the most severe complication of allogeneic stem cell transplantation, a standard-of-care treatment with curative intent offered to patients with blood cancers and some non-malignant hematological conditions. aGvHD refractoriness to current treatments is frequently encountered and severely impacts prognosis. In particular, patients with aGvHD failing both steroid and ruxolitinib typically exhibit a dismal prognosis, with a median survival of 28 days and 85% mortality at one year (Abedin et al 2021). Currently, no therapy is approved for third-line aGvHD, underscoring the urgent need for innovative therapies capable of improving survival and quality of life. The MAA is supported by positive results from the Pivotal ARES study, a single-arm, open-label, multicenter European study evaluating the efficacy and safety of Xervyteg® in GI-aGvHD as third-line therapy in 66 patients. Notably, the study met its primary endpoint, achieving a gastrointestinal overall response rate (GI-ORR) of 62% at Day 28, significantly exceeding the expected 38% response rate, and an overall response rate across all organs of 64% at Day 28. Among responding patients at Day 28, the majority exhibited full resolution of GvHD clinical manifestations (i.e., complete response), an important finding predictive of durable control of aGvHD clinical manifestations over time. The 12-month probability of survival was 54% (vs 15% Abedin et al, 2021). Importantly, patients who exhibited gastrointestinal response at Day 28 had a significantly better probability of survival than non-responders (67% vs 28% respectively, p <0.0001), indicating that Xervyteg®-mediated aGvHD control is associated with a remarkable survival benefit. Additional secondary endpoints, including overall survival, will become available in late H2 2025. The Company also integrated supporting safety and efficacy data from 186 aGvHD patients2 treated under its ongoing EAP, which aligns with the positive topline results of the ARES trial and further supports Xervyteg®'s strong efficacy and favorable safety profile in aGvHD. The safety and tolerability of Xervyteg® has been monitored by an independent Data Safety Monitoring Board (DSMB). In March 2025, the DSMB reviewed the overall safety of the trial (after all patients completed Day 28 visit or were discontinued earlier) and confirmed that "given the remarkable efficacy results, the study results show an acceptable safety profile and a favourable benefit /risk ratio". The DSMB members will continue to review safety on an ongoing basis until the 1-year follow-up. The EMA will review the application under the centralized marketing authorization procedure and potentially a marketing authorization could be granted in H2 2026. This centralized procedure means that a single marketing authorization application can be submitted to the EU, and if granted by the European Commission, the authorization is valid in all EU Member States as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway. About acute Graft-versus-Host Disease Acute Graft-versus-Host Disease occurs in patients within 100 days of undergoing a stem cell or bone marrow transplant, where the transplanted cells initiate an immune response and attack the transplant recipient's organs, causing inflammation of the skin, liver and/or gastro-intestinal tract and leading to significant morbidity and mortality. GI involvement is associated with severe complications such as profound diarrhea, abdominal pain, intestinal bleeding, and death. These complications are often life-threatening, with increased mortality risk, due to the challenges of managing severe GI inflammation and the associated risks of infection, malnutrition, and organ failure. The standard first line therapy for treating aGvHD is the use of systemic steroids. If patients do not respond to steroids, they are considered Steroid Resistant (SR) and other agents can be administered. Currently the only agent approved for treating SR aGvHD after failure of steroid treatment is ruxolitinib, which is currently approved for this indication in USA and has received approval from the European Medical Agency's Committee for Human Medicinal Products (CHMP) on March 25, 2022. About Xervyteg® (MaaT013) MaaT Pharma's Microbiome Ecosystem Therapies (MET) are designed to leverage a full microbiome ecosystem to restore balance and maximize clinical benefits for patients with severe, treatment-induced dysbiosis in acute diseases. Xervyteg® (MaaT013) is a full-ecosystem, off-the-shelf, standardized, pooled-donors, enema Microbiome Ecosystem TherapyTM for acute, hospital use. It is characterized by a consistently high diversity and richness of microbial species and the presence of ButycoreTM (a group of bacterial species known to produce anti-inflammatory metabolites). Xervyteg® (MaaT013) aims to restore the symbiotic relationship between the patient's functional gut microbiome and their immune system to correct the responsiveness and tolerance of immune functions and thus reduce steroid-resistant, gastrointestinal (GI)-aGvHD. Xervyteg® (MaaT013) has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). About MaaT Pharma MaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021. Forward-looking Statements All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company's control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as "target," "believe," "expect," "aim", "intend," "may," "anticipate," "estimate," "plan," "project," "will," "can have," "likely," "should," "would," "could" and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company's control that could cause the Company's actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements. 1 Updated data from the Early Access Program will be presented at the EHA Annual Congress in Milan, June 12–16, 20252 The cutoff date was October 3, 2024 View source version on Contacts MaaT Pharma – Investor RelationsGuilhaume DEBROAS, of Investor Relations+33 6 16 48 92 50invest@ Rx Communications Group –U.S. Investor RelationsMichael MillerManaging Director+1-917-633-6086mmiller@ MaaT Pharma – Media RelationsPauline RICHAUDSenior PR & Corporate Communications Manager+33 6 14 06 45 92media@ Catalytic Agency –U.S. Media RelationsHeather SheaMedia relations for MaaT Pharma+1 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
21-05-2025
- Business
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Savara Inc. (SVRA): Analysts See 212% Upside Potential
We recently published an article titled . Savara Inc. (NASDAQ:SVRA) was one of the stocks that was covered in that article. Wall Street analysts believe SVRA has a 212% upside potential over the next 12 months. Close-up of an inhaler, representing the inhalation of the molgramostim product candidate. Savara Inc. (NASDAQ:SVRA) is a clinical-stage biopharmaceutical company dedicated to addressing the unmet needs of patients suffering from rare respiratory diseases. Founded in 1995, Savara is a key player in this niche field through innovative therapeutic development and a commitment to delivering life-changing solutions to patients worldwide. The company focuses on diseases that have limited treatment options, ensuring its research has a significant impact on improving health outcomes. The cornerstone of Savara Inc. (NASDAQ:SVRA) current pipeline is MOLBREEVI, a promising therapeutic designed to treat autoimmune pulmonary alveolar proteinosis (aPAP), a rare lung disease characterized by the accumulation of surfactant in the alveoli, impeding gas exchange and leading to respiratory distress. MOLBREEVI is currently undergoing Phase 3 clinical trials and has already demonstrated notable improvements in gas exchange, as well as measurable clinical benefits for patients. Savara is actively pursuing regulatory approval through a rolling Biologics License Application (BLA) submission to the FDA, and it is preparing for a Marketing Authorization Application (MAA) to the European Medicines Agency. These efforts reflect Savara's commitment to bringing MOLBREEVI to market as efficiently as possible. Unlike many biopharmaceutical companies, Savara employs a unique business model that emphasizes outsourcing the majority of its clinical development and manufacturing activities to specialized vendors and consultants. This approach enables the company to operate in a capital-efficient manner, maximizing resources while maintaining the highest standards of quality and innovation. Additionally, Savara's leadership team has extensive experience in navigating the complexities of drug development and commercialization, further strengthening its strategic position in the industry. Financially, Savara Inc. (NASDAQ:SVRA) is in a strong position, with approximately $196 million in cash and short-term investments as of its latest financial report. This robust financial reserve ensures the company's operations remain funded through Q2 2027, providing a solid foundation for advancing its pipeline and achieving its goals. Analysts have expressed optimism about Savara's prospects, citing the company's growing momentum, innovative pipeline, and prudent investment strategy as key drivers of its twelve-month average price target of $9.44, which reflects a substantial upside potential of 212.84%. Overall, Savara Inc. (NASDAQ:SVRA) ranks 13th on our list of 13 Best Multibagger Stocks to Invest in Now. While we acknowledge the potential of SVRA to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than SVRA and that has 100x upside potential, check out our report about this cheapest AI stock. READ NEXT: 10 Best Low Volatility Stocks to Buy Now and Starter Stock Portfolio: 12 Safe Stocks to Buy Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
Yahoo
21-05-2025
- Business
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Savara Inc. (SVRA): Analysts See 212% Upside Potential
We recently published an article titled . Savara Inc. (NASDAQ:SVRA) was one of the stocks that was covered in that article. Wall Street analysts believe SVRA has a 212% upside potential over the next 12 months. Close-up of an inhaler, representing the inhalation of the molgramostim product candidate. Savara Inc. (NASDAQ:SVRA) is a clinical-stage biopharmaceutical company dedicated to addressing the unmet needs of patients suffering from rare respiratory diseases. Founded in 1995, Savara is a key player in this niche field through innovative therapeutic development and a commitment to delivering life-changing solutions to patients worldwide. The company focuses on diseases that have limited treatment options, ensuring its research has a significant impact on improving health outcomes. The cornerstone of Savara Inc. (NASDAQ:SVRA) current pipeline is MOLBREEVI, a promising therapeutic designed to treat autoimmune pulmonary alveolar proteinosis (aPAP), a rare lung disease characterized by the accumulation of surfactant in the alveoli, impeding gas exchange and leading to respiratory distress. MOLBREEVI is currently undergoing Phase 3 clinical trials and has already demonstrated notable improvements in gas exchange, as well as measurable clinical benefits for patients. Savara is actively pursuing regulatory approval through a rolling Biologics License Application (BLA) submission to the FDA, and it is preparing for a Marketing Authorization Application (MAA) to the European Medicines Agency. These efforts reflect Savara's commitment to bringing MOLBREEVI to market as efficiently as possible. Unlike many biopharmaceutical companies, Savara employs a unique business model that emphasizes outsourcing the majority of its clinical development and manufacturing activities to specialized vendors and consultants. This approach enables the company to operate in a capital-efficient manner, maximizing resources while maintaining the highest standards of quality and innovation. Additionally, Savara's leadership team has extensive experience in navigating the complexities of drug development and commercialization, further strengthening its strategic position in the industry. Financially, Savara Inc. (NASDAQ:SVRA) is in a strong position, with approximately $196 million in cash and short-term investments as of its latest financial report. This robust financial reserve ensures the company's operations remain funded through Q2 2027, providing a solid foundation for advancing its pipeline and achieving its goals. Analysts have expressed optimism about Savara's prospects, citing the company's growing momentum, innovative pipeline, and prudent investment strategy as key drivers of its twelve-month average price target of $9.44, which reflects a substantial upside potential of 212.84%. Overall, Savara Inc. (NASDAQ:SVRA) ranks 13th on our list of 13 Best Multibagger Stocks to Invest in Now. While we acknowledge the potential of SVRA to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than SVRA and that has 100x upside potential, check out our report about this cheapest AI stock. READ NEXT: 10 Best Low Volatility Stocks to Buy Now and Starter Stock Portfolio: 12 Safe Stocks to Buy Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
Yahoo
13-05-2025
- Business
- Yahoo
XOMA Royalty Reports First Quarter 2025 Financial Results and Highlights Business Achievements
: The Marketing Authorization Application (MAA) for Day One Biopharmaceuticals and Ipsen's tovorafenib was accepted for review by the European Marketing Authority (EMA) and Takeda initiated its Phase 3 trial exploring mezagitamab for the treatment of chronic primary immune thrombocytopenia : Acquired an economic interest in Castle Creek Biosciences' D-Fi (FCX-007) through participation in a syndicated royalty financing transaction and successfully sold all unpartnered Kinnate assets Received $18.0 million in the first quarter of 2025 including $13.4 million in royalty receipts EMERYVILLE, Calif. , May 13, 2025 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its first quarter 2025 financial results and highlighted recent actions that have the potential to deliver shareholder value. 'We are committed to generating value for shareholders through prudent cash deployment, strict expense control, and opportunistic share repurchases. Our first quarter highlights included the progression of key pipeline assets, solid cash receipts, and an increase in our share repurchase activity,' stated Owen Hughes, Chief Executive Officer of XOMA Royalty. 'With accelerating royalty receipts and a robust pipeline, we believe a path to sustained cashflow generation is tangible.' Royalty and Milestone Acquisitions Partner Asset and Transaction Detail Castle Creek XOMA Royalty added a royalty interest in D-Fi (FCX-007), a Phase 3 asset being developed by Castle Creek Biosciences, to the portfolio. D-Fi is being studied in dystrophic epidermolysis bullosa (DEB), a rare progressive and debilitating skin disorder. D-Fi has been granted Orphan Drug Designation for the treatment of DEB, as well as Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations by the Royalty contributed $5 million to Castle Creek Biosciences' $75 million syndicated royalty financing transaction. Partner Updates through May 9, 2025 Partner Event Rezolute In January, Rezolute received Breakthrough Therapy Designation from FDA for ersodetug (RZ358) for the treatment of hypoglycemia due to congenital hyperinsulinism (cHI) February, the company announced the Independent Data Monitoring Committee (DMC) reviewed the safety data from eight infants ages 3 months to 1 year enrolled in the open-label portion of the sunRIZE Phase 3 study of ersodetug for the treatment of hypoglycemia due to cHI. Their conclusion was the safety profile was such that infants may now be enrolled in the double-blind, placebo-controlled April, Rezolute announced the Independent DMC recommended the sunRIZE Phase 3 trial continue as planned with no need to increase sample size. Enrollment is on track and is expected to be completed in May 2025. Topline data is anticipated in December 2025.3In May, the company announced the FDA has granted Breakthrough Therapy Designation (BTD) to its investigational therapy, ersodetug, for the treatment of hypoglycemia caused by tumor HI.4 Affitech Research AS XOMA Royalty paid $6 million in milestones to Affitech related to VABYSMO® (faricimab-svoa) achieving specific sales thresholds. This was the final payment due to Affitech. Daré Bioscience Announced its intention to make its Sildenafil Cream, 3.6%, available by prescription under Section 503B of the Food and Drug Cosmetic Act while it pursues a parallel path to obtain FDA approval. Daré anticipates Sildenafil Cream will be available via one 503B-registered outsourcing facility partner in the fourth quarter of 2025. Day One Biopharmaceuticals Ipsen, Day One's partner outside of the U.S., filed a Marketing Authorization Application (MMA) with the European Medicines Agency for tovorafenib as a treatment for pediatric low-grade glioma (pLGG)5. XOMA Royalty earned a $4.0 million milestone related to this filing. Takeda The first patient was dosed in Takeda's Phase 3 clinical trial investigating mezagitamab as a treatment for adults with chronic primary immune thrombocytopenia (ITP). This achievement triggered a $3.0 million milestone payment, net, to XOMA Royalty in the second Event Kinnate In early 2025, XOMA Royalty sold the five unpartnered Kinnate assets to several parties. Per the terms of the acquisition, a portion of any upfront payments received by XOMA Royalty will be distributed to the Kinnate CVR holders. Anticipated 2025 Events of Note Partner Event Day One Biopharmaceuticals The European Medicines Agency (EMA) decision regarding Day One's Marketing Authorization Application (MAA) for tovorafenib, a treatment for the most common childhood brain tumor, pediatric low-grade glioma (pLGG). Rezolute Completion of enrollment in sunRIZE Phase 3 clinical trial, which is investigating ersodetug in infants and children with cHI. Topline data are expected in December 20253. First patient dosed in the Phase 3 registrational study for ersodetug for the treatment of hypoglycemia due to tumor hyperinsulinism6. Gossamer / Chiesi Presentation of topline results from the Phase 3 PROSERA study, a global registrational clinical trial in patients with WHO Function Class II and III pulmonary arterial hypertension (PAH).7 Initiation of a registrational Phase 3 study in pulmonary hypertension associated with interstitial lung disease (PH-ILD) in 2025.1 Daré Bioscience Successfully makes Sildenafil Cream available via prescription in the fourth quarter of 2025 as a compounded drug under Section 503B of the of one of two registrational Phase 3 clinical trials investigating Sildenafil Cream, 3.6%, for the treatment of female sexual arousal disorder8. First Quarter 2025 Financial Results Tom Burns, Chief Financial Officer of XOMA Royalty, commented, 'In the first quarter, we received $18 million in cash, $13.4 million from our partners' commercial sales and $4.6 million from milestones and fees. As our partners continue to execute well on their commercial product launch activities and we learn of new commercial opportunities within our portfolio, our line of sight on becoming cash flow positive on a consistent basis exclusively from the cash payments received from royalties grows clearer. With this outlook, we deployed $0.5 million to repurchase 25,828 shares of our common stock.' Income and Revenue: XOMA Royalty recorded total income and revenues of $15.9 million for the first quarter of 2025, compared to $1.5 million for the comparable period in 2024. The increase for the first quarter of 2025 was primarily driven by income recorded under the effective interest rate method related to VABYSMO®, a milestone of $4.0 million associated with Day One and Ipsen's MAA filing with the EMA, a $4.0 million payment related to our collaboration agreement with Takeda, and $1.5 million in estimated royalties related to OJEMDA™. Research and Development (R&D) Expenses: R&D expenses were $1.3 million in the first quarter of 2025, compared to $33,000 in the first quarter of 2024. The increase of approximately $1.3 million for the first quarter of 2025 was due to $1.0 million in pass-through licensing fees to an undisclosed licensor related to the Phase 3 milestone achieved by Takeda under our Takeda Collaboration Agreement, combined with clinical trial costs related to KIN-3248. General and Administrative (G&A) Expenses: G&A expenses were $8.1 million in the first quarter of 2025, compared with $8.5 million for the same period in 2024. The decrease of $0.4 million was primarily due to a decrease of $0.9 million in stock compensation costs, partially offset by an increase in consulting costs of $0.5 million related to our Kinnate acquisition. In the quarter ended March 31, 2025, G&A expenses included $2.0 million of non-cash stock-based compensation expenses, compared to $2.9 million in the first quarter of 2024. The $0.9 million difference between the two periods is primarily driven by the timing of expense recognition related to the performance stock unit grant awarded to Mr. Hughes in connection with his appointment as full-time CEO in January 2024. Interest Expense: Interest expense was $3.5 million and $3.6 million for the first quarters of 2025 and 2024 respectively. Interest expense relates to the Blue Owl Loan established in December 2023. Amortization of Intangible Assets: Amortization of intangible assets relates to the IP acquired in the Company's acquisition of Pulmokine in November 2024. Other Income/Expense, net: The Company reported other expense, net, of $0.1 million in the first quarter of 2025, compared to other income, net, of $2.0 million in the comparable period of 2024. The reduction during the first quarter of 2025 was primarily driven by a decrease in the fair value of our investments in two public companies' equity securities and a decrease in investment income due to decreased balances and decreased market interest rates on XOMA Royalty's investments. Net Income (Loss): Net income for the first quarter ended March 31, 2025, was $2.4 million, compared to a net loss of $8.6 million in the first quarter of 2024. Cash: On March 31, 2025, XOMA Royalty had cash and cash equivalents of $95.0 million (including $4.8 million in restricted cash), compared with cash and cash equivalents of $106.4 million (including $4.8 million in restricted cash) on December 31, 2024. In the first quarter of 2025, XOMA Royalty received $18.0 million in cash receipts including $13.4 million in royalties and commercial payments and $4.6 million in milestones and fees. In the first quarter of 2025, XOMA Royalty deployed $5.0 million to acquire a new Phase 3 milestone and royalty asset, used $0.5 million to repurchase 25,828 shares, and paid $1.4 million in dividends on the XOMA Royalty Perpetual Preferred stocks. About XOMA Royalty CorporationXOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit or follow XOMA Royalty Corporation on LinkedIn. Forward-Looking Statements/Explanatory NotesCertain statements contained in this press release are 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, including statements regarding the timing and amount of potential commercial payments to XOMA Royalty and other developments related to VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], DSUVIA® (sufentanil sublingual tablet), and Sildenafil Cream, 3.6%; the potential occurrences of the events listed under 'Anticipated 2025 Events of Note'; the anticipated timings of regulatory filings and approvals related to assets in XOMA Royalty's portfolio; and the potential of XOMA Royalty's portfolio of partnered programs and licensed technologies generating substantial milestone and royalty proceeds over time. In some cases, you can identify such forward-looking statements by terminology such as 'anticipate,' 'intend,' 'believe,' 'estimate,' 'plan,' 'seek,' 'project,' 'expect,' 'may,' 'will', 'would,' 'could' or 'should,' the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA Royalty's performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; and if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, our third-party licensees will not be able to market them. Other potential risks to XOMA Royalty meeting these expectations are described in more detail in XOMA Royalty's most recent filing on Form 10-Q and in other filings with the Securities and Exchange Commission. Consider such risks carefully when considering XOMA Royalty's prospects. Any forward-looking statement in this press release represents XOMA Royalty's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA Royalty disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to 'portfolio' in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to 'assets' in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty's milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. XOMA CORPORATION CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (unaudited) (in thousands, except share and per share amounts) Three Months Ended March 31, 2025 2024 Income and Revenues: Income from purchased receivables under the EIR method $ 6,070 $ - Income from purchased receivables under the cost recovery method 5,525 - Revenue from contracts with customers 4,000 1,000 Revenue recognized under units-of-revenue method 317 490 Total income and revenues 15,912 1,490 Operating expenses: Research and development 1,293 33 General and administrative 8,146 8,461 Amortization of intangible assets 544 - Total operating expenses 9,983 8,494 Income (Loss) from operations 5,929 (7,004 ) Other income (expense) Interest expense (3,467 ) (3,551 ) Other income (expense), net (95 ) 1,960 Net income (loss) $ 2,367 $ (8,595 ) Net income (loss) available to (attributable to) common stockholders, basic $ 705 $ (9,963 ) Basic net income (loss) per share available to (attributable to) common stockholders $ 0.06 $ (0.86 ) Weighted average shares used in computing basic net income (loss) per share available to (attributable) to common stockholders 11,969 11,580 Net income (loss) available to (attributable to) common stockholders, diluted $ 999 $ (9,963 ) Diluted net income (loss) per share available to (attributable to) common stockholders $ 0.06 $ (0.86 ) Weighted average shares used in computing diluted net income (loss) per share available to (attributable) to common stockholders 17,781 11,580 XOMA CORPORATION CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share and per share amounts) March 31, December 31, 2025 2024 ASSETS (unaudited) Current assets: Cash and cash equivalents $ 90,265 $ 101,654 Short-term restricted cash 1,410 1,330 Investment in equity securities 2,382 3,529 Trade and other receivables, net 5,544 1,839 Short-term royalty and commercial payment receivables under the EIR method 12,240 14,763 Short-term royalty and commercial payment receivables under the cost recovery method 413 413 Prepaid expenses and other current assets 971 2,076 Total current assets 113,225 125,604 Long-term restricted cash 3,352 3,432 Property and equipment, net 29 32 Operating lease right-of-use assets 304 319 Long-term royalty and commercial payment receivables under the EIR method 4,857 4,970 Long-term royalty and commercial payment receivables under the cost recovery method 59,916 55,936 Exarafenib milestone asset (Note 4) 3,307 3,214 Investment in warrants 605 - Intangible assets, net 25,365 25,909 Other assets - long term 1,790 1,861 Total assets $ 212,750 $ 221,277 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable $ 2,319 $ 1,053 Accrued and other liabilities 1,221 5,752 Contingent consideration under RPAs, AAAs, and CPPAs - 3,000 Operating lease liabilities 459 446 Unearned revenue recognized under units-of-revenue method 1,370 1,361 Preferred stock dividend accrual 1,368 1,368 Current portion of long-term debt 13,697 11,394 Total current liabilities 20,434 24,374 Unearned revenue recognized under units-of-revenue method – long-term 4,084 4,410 Exarafenib milestone contingent consideration (Note 4) 3,307 3,214 Long-term operating lease liabilities 362 483 Long-term debt 99,934 106,875 Total liabilities 128,121 139,356 Stockholders' equity: Preferred Stock, $0.05 par value, 1,000,000 shares authorized: 8.625% Series A cumulative, perpetual preferred stock, 984,000 shares issued and outstanding as of March 31, 2025 and December 31, 2024 49 49 8.375% Series B cumulative, perpetual preferred stock, 1,600 shares issued and outstanding as of March 31, 2025 and December 31, 2024 — — Convertible preferred stock, 5,003 shares issued and outstanding as of March 31, 2025 and December 31, 2024 — — Common stock, $0.0075 par value, 277,333,332 shares authorized, 11,952,889 and 11,952,377 shares issued and outstanding as of March 31, 2025 and December 31, 2024, respectively 90 90 Additional paid-in capital 1,319,607 1,318,766 Accumulated other comprehensive income 118 73 Accumulated deficit (1,235,235 ) (1,237,057 ) Total stockholders' equity 84,629 81,921 Total liabilities and stockholders' equity $ 212,750 $ 221,277 XOMA CORPORATION CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited) (in thousands) Three Months Ended March 31, 2025 2024 Cash flows from operating activities: Net income (loss) $ 2,367 $ (8,595 ) Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: Adjustment for income from EIR method purchased receivables 1,743 — Stock-based compensation expense 1,983 2,856 Common stock contribution to 401(k) 141 118 Amortization of intangible assets 544 — Depreciation 3 2 Accretion of long-term debt discount and debt issuance costs 427 306 Non-cash lease expense 17 14 Change in fair value of equity securities 1,147 (252 ) Change in fair value of available-for-sale debt securities classified as cash equivalents 45 — Changes in assets and liabilities: Trade and other receivables, net (3,705 ) 1,001 Prepaid expenses and other assets 1,176 213 Accounts payable and accrued liabilities (3,265 ) (105 ) Operating lease liabilities (108 ) (15 ) Unearned revenue recognized under units-of-revenue method (317 ) (490 ) Net cash provided by (used in) operating activities 2,198 (4,947 ) Cash flows from investing activities: Payments of consideration under RPAs, AAAs and CPPAs (8,000 ) (15,000 ) Receipts under RPAs, AAAs and CPPAs 1,307 7,771 Purchase of property and equipment — (17 ) Net cash used in investing activities (6,693 ) (7,246 ) Cash flows from financing activities: Principal payments – debt (5,066 ) (3,616 ) Debt issuance costs and loan fees paid in connection with long-term debt — (581 ) Payment of preferred stock dividends (1,368 ) (1,368 ) Repurchases of common stock (545 ) (13 ) Proceeds from exercise of options and other share-based compensation 325 1,956 Taxes paid related to net share settlement of equity awards (240 ) (1,334 ) Net cash used in financing activities (6,894 ) (4,956 ) Net decrease in cash, cash equivalents and restricted cash (11,389 ) (17,149 ) Cash, cash equivalents and restricted cash at the beginning of the period 106,416 159,550 Cash, cash equivalents and restricted cash at the end of the period $ 95,027 $ 142,401 Supplemental Cash Flow Information: Cash paid for interest $ 6,078 $ 3,780 Cash paid for taxes $ 277 $ — Non-cash investing and financing activities: Accrual of contingent consideration under the Affitech CPPA $ — $ 3,000 Preferred stock dividend accrual $ 1,368 $ 1,368 Investor contact:Juliane SnowdenXOMA Royalty Corporation+ Media contact:Kathy VincentKV Consulting & Management+1-310-403-8951kathy@ 1
