On February 11, 2025 Veracyte, Inc. (Nasdaq: VCYT), a leading cancer diagnostics company, reported that 17 Decipher-focused abstracts will be presented at the 2025 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU) (Press release, Veracyte, FEB 11, 2025, View Source [SID1234650178]). The findings demonstrate the market leadership of its Decipher tests in better informing treatment decisions for patients with prostate and bladder cancers, compared to standard approaches, and in helping to drive innovation that will inform the future of urologic cancer care. The conference will take place February 13-15 in San Francisco.

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"The scale and scope of data to be presented at the ASCO (Free ASCO Whitepaper) GU meeting underscore the impact our whole-transcriptome-based Decipher tests are having in treating patients with urologic cancers, and how our GRID, or Genomic Resource for Intelligent Discovery, research tool is helping to advance scientific understanding of these diseases," said Elai Davicioni, Ph.D., Veracyte’s medical director, Urology. "Our commitment to generating robust clinical evidence with collaborators has led to our market-leading Decipher Prostate test achieving the highest status among gene expression tests in the most recent, and prior, NCCN guidelines.* We look forward to using a similar approach for our Decipher Bladder test in bladder cancer."

"Clinical rigor and robust evidence generation are important for a molecular test to gain widespread adoption and inclusion in guidelines for helping physicians and their patients with prostate cancer make better-informed treatment decisions," said Daniel Spratt, M.D., of the University Hospitals Seidman Cancer Center, Case Western Reserve University. "National guidelines have acknowledged the large body of work from randomized Phase 3 trials that have been profiled with Decipher and include the Decipher test as an advanced risk-stratification tool that can aid in shared-decision making for patients."

Key Decipher-focused findings to be presented at the ASCO (Free ASCO Whitepaper) GU Symposium are:

Title:

Decipher Score as a Predictor of Response to Treatment Intensification in the NRG Oncology-RTOG 0534 (SPPORT) Phase III Randomized Post-Prostatectomy Salvage Radiotherapy Trial

Presenter:

Alan Pollack, M.D., Ph.D., University of Miami Health System

Format:

Poster (#K29)

Abstract #:

399

Date/Time:

Thursday, February 13, 2025; 11:25 a.m.—12:45 p.m. PST

Location:

Level 1, West Hall; On Demand

Summary:

A post-hoc analysis of the NRG Oncology/RTOG 05-34 (SSPORT) Phase 3, randomized trial shows that the Decipher Prostate test may inform treatment intensification strategies in patients undergoing salvage radiotherapy by identifying those who will benefit from pelvic nodal radiation.

Title:

A non-coding RNA based classifier for favorable outcomes in clinically organ confined bladder cancer

Presenter:

Yair Lotan, M.D., UT Southwestern Medical Center

Format:

Poster (#G27)

Abstract #:

831

Date/Time:

Friday, February 14, 2025; 11:30 a.m.—12:45 p.m. PST

Location:

Level 1, West Hall; On Demand

Summary:

Study findings show that the Decipher Bladder test accurately identifies patients whose bladder cancer has a luminal molecular subtype, which is associated with favorable outcomes. Such findings could help clinicians determine which patients are less likely to benefit from intensified therapy.

Title:

Gene signature predictor of dose-response to prostate radiation: validation of PORTOS in phase III trials

Presenter:

Shuang Zhao, M.D., University of Wisconsin-Madison

Format:

Oral Presentation

Abstract #:

308

Date/Time:

Thursday, February 13, 2025; 8:00 a.m.—9:40 a.m. PST

Location:

Level 3, Ballroom; Live Stream

Summary:

Analyses of two prospective, randomized, Phase 3 trials (RTOG 01-26 and SAKK 09/10) show that the PORTOS gene expression signature – currently part of the Decipher GRID research tool – predicts which patients will benefit from dose-escalated radiation therapy in both primary and salvage treatment settings.

Additional notable Decipher-focused presentations include:

Title:

Decipher Risk Stratification of Radiorecurrent Prostate Cancer: Correlative Analysis of the F-SHARP Trial of Salvage Reirradiation

Presenter:

Abhishek Solanki, M.D., M.S., Loyola University Chicago

Format:

Poster (#L15)

Abstract #:

419

Date/Time:

Thursday, February 13, 2025; 11:25 a.m.—12:45 p.m. PST

Location:

Level 1, West Hall; On Demand

Summary:

Radiation therapy is increasingly used for patients whose prostate cancer has recurred following initial radiation treatment. This study suggests the Decipher Prostate test can identify patients who are likely to benefit from more radiation alone versus those who may also need further treatment intensification.

Title:

Androgen receptor activity in biopsy specimens at initial diagnosis of prostate cancer and correlation with outcomes and treatment response

Presenter:

Nicole Handa, M.D., Northwestern University, Feinberg School of Medicine

Format:

Poster (#L5)

Abstract #:

409

Date/Time:

Thursday, February 13, 2025; 11:25 a.m.—12:45 p.m. PST

Location:

Level 1, West Hall; On Demand

Summary:

Using the Decipher GRID research tool to assess transcriptome-wide expression data and clinical factors, investigators found that low androgen receptor activity (AR-A) was associated with poor outcomes for prostate cancer patients. They suggest such patients could potentially benefit from post-operative radiation therapy and PARP inhibitors.

Title:

Risk Stratification Using the Decipher 22-gene Genomic Classifier (GC) and Digital Pathology Artificial Intelligence (DPAI) in Nearly 10,000 Localized Prostate Cancer Patients

Presenter:

Daniel Spratt, M.D., University Hospitals Seidman Cancer Center, Case Western Reserve University

Format:

Poster (#L4)

Abstract #:

408

Date/Time:

Thursday, February 13, 2025; 11:25 a.m.—12:45 p.m. PST

Location:

Level 1, West Hall; On Demand

Summary:

This large study found that adding digital pathology artificial intelligence (DPAI) tools to the Decipher Prostate Genomic Classifier did not further improve the molecular test’s prognostic performance. Ongoing research is underway to explore whether there may be specific clinical states where the combination of DPAI and the Decipher test may add meaningful clinical utility.

"The extensive Decipher-focused data at the 2025 ASCO (Free ASCO Whitepaper) GU Symposium demonstrate the power of our novel Veracyte Diagnostics Platform, which begins with delivering high-performing tests using a comprehensive, whole-transcriptome approach. This enables additional research, which in turn supports further innovation to help more patients," said Phillip Febbo, M.D., Veracyte’s chief scientific officer and chief medical officer.

Veracyte’s Decipher team will be at Booth #37 at the 2025 ASCO (Free ASCO Whitepaper) GU Symposium. More information, including a full list of Decipher-focused abstracts being presented, can be found here.

About Decipher Prostate

The Decipher Prostate Genomic Classifier is a 22-gene test, developed using RNA whole-transcriptome analysis and machine learning, that helps inform treatment decisions for patients with prostate cancer. The test is performed on biopsy or surgically resected samples and provides an accurate risk of developing metastasis with standard treatment. Armed with this information, physicians can better personalize their patients’ care and may recommend less-intensive options for those at lower risk or earlier, more-intensive treatment for those at higher risk of metastasis. The Decipher Prostate test has been validated in many dozens of published studies involving more than 100,000 patients. It is the only gene expression test to achieve "Level 1B" evidence status and inclusion in the risk-stratification table in the most recent NCCN Guidelines* for prostate cancer. More information about the Decipher Prostate test can be found here.

About Decipher Bladder

The Decipher Bladder Genomic Classifier is a 219-gene test, developed using RNA whole-transcriptome analysis and machine learning, that is designed for use in patients following bladder cancer diagnosis who face questions regarding treatment intensity. The test classifies bladder tumors into five molecular subtypes, each having distinct tumor biology and potential clinical implications. This information can help physicians and their patients better understand the degree of benefit that would likely be gained from neoadjuvant chemotherapy and/or the likelihood of harboring non-organ-confined disease at time of surgery, respectively. More information about the Decipher Bladder test can be found here.

About Decipher GRID

The Decipher GRID database includes more than 200,000 whole-transcriptome profiles from patients with urologic cancers and is used by Veracyte and its partners to contribute to continued research and help advance understanding of prostate and other urologic cancers. GRID-derived information is available on a Research Use Only basis. More information about Decipher GRID can be found here.

On February 11, 2025 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported that the U.S. Food and Drug Administration (FDA) has approved GOMEKLI (mirdametinib), SpringWorks’ MEK inhibitor, for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection (Press release, SpringWorks Therapeutics, FEB 11, 2025, View Source [SID1234650177]). With the approval, SpringWorks was granted a rare pediatric disease priority review voucher (PRV) by the FDA.

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"The NF1-PN patient community has a great need for more treatment options. With today’s approval, we are honored to serve both adults and children with NF1-PN and provide them with a therapy that has the potential to shrink their tumors and offer meaningful symptomatic relief," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are grateful to each clinical trial participant, their families, the investigators, and the patient advocacy groups involved in the journey towards making GOMEKLI available in the U.S. I am proud that we are delivering on our commitment to patients with devastating diseases with our company’s second FDA approval in less than 18 months."

NF1 is a genetic disorder that currently affects approximately 100,000 children and adults in the United States.2,3 Patients with NF1 have approximately a 30-50% lifetime risk of developing plexiform neurofibromas, or PNs, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment.2,4 There are approximately 40,000 people in the United States living with NF1-PN, the majority of whom are adults that have not had an approved medicine until GOMEKLI.5 Plexiform neurofibromas can transform into malignant peripheral nerve sheath tumors, an aggressive and potentially fatal disease.6 Surgical removal can be challenging due to the infiltrative tumor growth pattern of plexiform neurofibromas along nerves, and up to approximately 85% of plexiform neurofibromas are considered not amenable to complete resection.7,8,9

"Patients with NF1-PN often face significant challenges with their health and have had limited treatment options to manage this devastating condition," said Christopher Moertel, M.D., Medical Director Pediatric Neuro-Oncology and Neurofibromatosis Programs and Kenneth and Betty Jayne Dahlberg Professor of Pediatrics, University of Minnesota, and lead investigator of the ReNeu trial. "It was very encouraging in the ReNeu trial to see that GOMEKLI provided deep and durable responses, with a manageable safety profile that enabled patients to stay on therapy. This approval represents an important advance, especially for adults who previously did not have an approved treatment."

GOMEKLI was approved under Priority Review and SpringWorks received a rare pediatric disease priority review voucher from the FDA. GOMEKLI was previously granted Orphan Drug and Fast Track designations for the treatment of NF1-PN.

The FDA approval of GOMEKLI is based on results from the Phase 2b ReNeu trial, which enrolled 114 patients with NF1-PN ≥2 years of age (58 adults and 56 pediatric patients).10 GOMEKLI met the primary endpoint of confirmed objective response rate (ORR), as assessed by blinded independent central review, demonstrating a 41% ORR (N= 24/ 58) in adults and 52% in children (N=29/56).10 Tumor volume reductions were deep and durable; the median best percentage change in target PN volume was -41% (range: -90 to 13%) in adults and -42% (range: -91 to 48%) in children.10 Eighty-eight percent of adults and 90% of children with a confirmed response had a response of at least 12 months duration, and 50% and 48%, respectively, had a response of at least 24 months duration.10 Patients in both cohorts also experienced early and sustained significant improvements from baseline in pain, and quality of life, as assessed across multiple patient-reported outcome tools.10

GOMEKLI demonstrated a manageable safety and tolerability profile.1 The most common adverse events (>25%) reported in adults receiving GOMEKLI were rash, diarrhea, nausea, musculoskeletal pain, vomiting and fatigue.1 The most common adverse events (>25%) occurring in children were rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.1 Please see additional Important Safety Information below, including Warnings & Precautions relating to ocular toxicity, left ventricular dysfunction, dermatologic adverse reactions, and embryo-fetal toxicity.

"We are excited to celebrate the extraordinary milestone of our partners and long-term friends at SpringWorks for the NF1-PN community. This FDA approval shows the power of collaboration to advance innovative science for drugs that may otherwise not have been taken forward," said Annette Bakker, Ph.D., Chief Executive Officer of the Children’s Tumor Foundation. "When industry, researchers, and organizations like ours driving treatment innovation join forces, scientific progress moves faster, and patients gain access to the therapies they need. Every treatment approval is hard-won, built on research, persistence, and partnership. Today, that work delivers a critical new option for NF patients of all ages."

"NF1-PN is a complex, devastating disease that affects not only individual patients, but entire families. Treatment advances are crucial to achieving better outcomes for patients and this FDA approval offers hope for NF patients and their families," said Kim Bischoff, Executive Director, NF Network.

SpringWorks is dedicated to helping patients with NF1-PN access GOMEKLI and to providing support throughout their treatment journey. The SpringWorks CareConnections program is a comprehensive patient support program that offers personalized support services and resources to eligible GOMEKLI patients, including insurance coverage information and access support, financial assistance and personalized educational and emotional support. Physicians and patients can contact 1-844-CARES-55 (1-844-227-3755) or visit www.springworkstxcares.com for more information.

GOMEKLI is available in 1 and 2 mg capsules and in a 1mg tablet for oral suspension, which dissolves easily in water. GOMEKLI is expected to be available through a specialty pharmacy and specialty distributor network in the United States within two weeks.

SpringWorks’ Marketing Authorization Application for mirdametinib for the treatment of children and adults with NF1-PN was validated by the European Medicines Agency (EMA) and is currently under review; a decision is expected from the European Commission in 2025.

About NF1-PN

Neurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene, which encodes for neurofibromin, a key suppressor of the MAPK pathway.11,12 NF1 is the most common form of neurofibromatosis, with an estimated global birth incidence of approximately 1 in 2,500 individuals, and there are approximately 100,000 patients living with NF1 in the United States.2,3 The clinical course of NF1 is heterogeneous and manifests in a variety of symptoms across numerous organ systems, including abnormal pigmentation, skeletal deformities, tumor growth and neurological complications, such as cognitive impairment.13 Patients with NF1 have an 8 to 15-year mean reduction in their life expectancy compared to the general population.14

NF1 patients have approximately a 30-50% lifetime risk of developing plexiform neurofibromas, or PN, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment; in rare cases, NF1-PN may be fatal.2,4,6 NF1-PNs are most often diagnosed in the first two decades of life.2 These tumors can be aggressive and are associated with clinically significant morbidities; typically, they grow more rapidly during childhood.15,16

Surgical removal of these tumors can be challenging due to the infiltrative tumor growth pattern along nerves and can lead to permanent nerve damage and disfigurement.6 Up to approximately 85% of plexiform neurofibromas are considered not amenable to complete resection.7,8,9

About GOMEKLI (mirdametinib)

GOMEKLI is an oral, small molecule MEK inhibitor approved in the United States for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Ocular Toxicity: GOMEKLI can cause ocular toxicity including retinal vein occlusion (RVO), retinal pigment epithelium detachment (RPED), and blurred vision. In the adult pooled safety population, ocular toxicity occurred in 28% of patients treated with GOMEKLI: 21% were Grade 1, 5% were Grade 2 and 1.3% were Grade 3. RVO occurred in 2.7%, RPED occurred in 1.3%, and blurred vision occurred in 9% of adult patients. In the pediatric pooled safety population, ocular toxicity occurred in 19% of patients: 17% were Grade 1 and 1.7% were Grade 2. Conduct comprehensive ophthalmic assessments prior to initiating GOMEKLI, at regular intervals during treatment, and to evaluate any new or worsening visual changes such as blurred vision. Continue, withhold, reduce the dose, or permanently discontinue GOMEKLI as clinically indicated.

Left Ventricular Dysfunction: GOMEKLI can cause left ventricular dysfunction. GOMEKLI has not been studied in patients with a history of clinically significant cardiac disease or LVEF <55% prior to initiation of treatment. In the ReNeu study, decreased LVEF of 10 to <20% occurred in 16% of adult patients treated with GOMEKLI. Five patients (9%) required dose interruption, one patient (1.7%) required a dose reduction, and one patient required permanent discontinuation of GOMEKLI. The median time to first onset of decreased LVEF in adult patients was 70 days. Decreased LVEF of 10 to <20% occurred in 25%, and decreased LVEF of ≥20% occurred in 1.8% of pediatric patients treated with GOMEKLI. One patient (1.8%) required dose interruption of GOMEKLI. The median time to first onset of decreased LVEF in pediatric patients was 132 days. All patients with decreased LVEF were identified during routine echocardiography, and decreased LVEF resolved in 75% of patients. Before initiating GOMEKLI, assess ejection fraction (EF) by echocardiogram. Monitor EF every 3 months during the first year and then as clinically indicated. Withhold, reduce the dose, or permanently discontinue GOMEKLI based on severity of adverse reaction.

Dermatologic Adverse Reactions: GOMEKLI can cause dermatologic adverse reactions including rash. The most frequent rashes included dermatitis acneiform, rash, eczema, maculo-papular rash and pustular rash. In the pooled adult safety population, rash occurred in 92% of patients treated with GOMEKLI and required permanent discontinuation in 11% of adult patients. In the pooled pediatric safety population, rash occurred in 72% of patients treated with GOMEKLI and resulted in permanent discontinuation of GOMEKLI in 3.4% of patients. Initiate supportive care at first signs of dermatologic adverse reactions. Withhold, reduce the dose, or permanently discontinue GOMEKLI based on severity of adverse reaction.

Embryo-Fetal Toxicity: GOMEKLI can cause fetal harm when administered to a pregnant woman. Verify the pregnancy status of females of reproductive potential prior to the initiation of GOMEKLI. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Also advise patients to use effective contraception during treatment with GOMEKLI and for 6 weeks after the last dose (females) or 3 months after the last dose (males).

ADVERSE REACTIONS

The most common adverse reactions (>25%) in adult patients were rash (90%), diarrhea (59%), nausea (52%), musculoskeletal pain (41%), vomiting (38%), and fatigue (29%). Serious adverse reactions occurred in 17% of adult patients who received GOMEKLI. The most common Grade 3 or 4 laboratory abnormality (>2%) was increased creatine phosphokinase.

The most common adverse reactions (>25%) in pediatric patients were rash (73%), diarrhea (55%), musculoskeletal pain (41%), abdominal pain (39%), vomiting (39%), headache (34%), paronychia (32%), left ventricular dysfunction (27%), and nausea (27%). Serious adverse reactions occurred in 14% of pediatric patients who received GOMEKLI. The most common Grade 3 or 4 laboratory abnormalities (>2%) were decreased neutrophil count and increased creatine phosphokinase.

USE IN SPECIFIC POPULATIONS

Pregnancy & Lactation. Verify the pregnancy status of patients of reproductive potential prior to initiating GOMEKLI. Due to the potential for adverse reactions in a breastfed child, advise patients not to breastfeed during treatment with GOMEKLI and for 1 week after the last dose.

You are encouraged to report negative side effects of prescription drugs to the FDA. To report suspected adverse reactions, contact SpringWorks Therapeutics at 1-888-400-SWTX (1-888-400-7989) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see accompanying full Prescribing Information for more information.

On February 11, 2025 Vivoryon Therapeutics N.V. (Euronext Amsterdam: VVY; NL00150002Q7) (Vivoryon), a clinical stage company focused on the discovery and development of small molecule medicines to modulate the activity and stability of pathologically altered proteins, reported to host a virtual R&D update with KOL (Key Opinion Leader) speakers on February 18, 2025 (Press release, Vivoryon Therapeutics, FEB 11, 2025, View Source [SID1234650176]).

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The webcast will include presentations from management and KOLs discussing further analyses and data on varoglutamstat’s observed beneficial effect on kidney function, an update on the Company’s anticipated clinical development plan for varoglutamstat in kidney disease, varoglutamstat’s potential market positioning, and an opportunity for investor and analyst questions.

Featured KOL speakers include:

Tobias B. Huber, MD – Chair of the Center of Internal Medicine and Director of the III. Department of Medicine – University Medical Center Hamburg-Eppendorf (UKE), Germany. Acting as Medical Advisor for clinical study design and certain R&D activities.
Kevin Carroll, PhD – CEO, KJC Statistics. Acting as statistical analysis expert, providing and calculating statistical read-outs and advising on clinical study statistical aspects.
Webcast details

Date: February 18, 2025

Time: 3:00 pm CET / 9:00 am EST

Further details of the event and participation information are available at: View Source

About Varoglutamstat
Varoglutamstat (PQ912) is a proprietary, potent and selective inhibitor of human glutaminyl cyclases QPCT and QPCTL with therapeutic potential in indications including inflammatory and fibrotic diseases, neurodegenerative diseases, cancer and others. Initially advanced development aiming to treat Alzheimer’s disease (AD), varoglutamstat has been investigated in a number of different clinical studies. Based on the known anti-inflammatory and anti-fibrotic activity of varoglutamstat, the protocol for the Phase 2b VIVIAD study in early AD included the investigation of kidney function (measured using eGFR) and measurement of biomarkers of kidney inflammation and fibrosis to explore the role of QPCT/L inhibition on kidney function. eGFR was also analyzed as a prospectively defined safety parameter in the VIVA-MIND Phase 2 study in the U.S.

On February 11, 2025 Royalty Pharma plc (Nasdaq: RPRX) reported financial results for the fourth quarter and full year 2024 and introduced full year 2025 guidance for Portfolio Receipts (Press release, Royalty Pharma , FEB 11, 2025, View Source [SID1234650175]).

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"We had an incredibly successful 2024, delivering double-digit growth in Royalty Receipts, which was significantly above our initial guidance, and deploying $2.8 billion of capital on value-enhancing royalties" said Pablo Legorreta, Royalty Pharma’s founder and Chief Executive Officer. "We are very excited for the opportunities ahead as the fundamentals of our business have never been stronger. Additionally, we have already taken two major steps at the start of 2025 to enhance shareholder value, announcing the acquisition of our external manager, which is expected to result in multiple financial and strategic benefits, and a new $3 billion share repurchase program, which highlights the confidence we have in our business and the attractive value we see in our shares. With a robust transaction pipeline and significant financial flexibility, I am confident that Royalty Pharma is well positioned to deliver attractive, compounding growth over the long term."

Strong Royalty Receipts growth; Portfolio Receipts growth impacted by a high base of comparison

•
Royalty Receipts grew 12% to $729 million in the fourth quarter and 13% to $2,771 million for full year 2024, driven by strong performance from Evrysdi, the CF franchise, Trelegy, Tremfya and new royalty acquisitions.

•
Portfolio Receipts increased 1% to $742 million in the fourth quarter of 2024; Portfolio Receipts decreased 8% from $3,049 million to $2,801 million for full year 2024, largely reflecting $525 million in Biohaven-related milestone payments received in 2023.

Capital Deployment of $2.8 billion in 2024 with royalties on eight new therapies added to the portfolio

•
Record year for synthetic royalty transactions for Royalty Pharma with $925 million announced in 2024.

•
Significantly expanded development-stage portfolio by acquiring royalties on four potential new therapies.

Exciting new product launches expected across the royalty portfolio in 2025

•
Royalty Pharma to benefit in 2025 from new product launches, including Servier’s Voranigo, Bristol Myers Squibb’s Cobenfy, Ascendis’ Yorvipath, Syndax and Incyte’s Niktimvo and Geron’s Rytelo.

Financial guidance for full year 2025 (excludes contribution from future transactions)

•
Royalty Pharma expects 2025 Portfolio Receipts to be between $2,900 million and $3,050 million, representing expected growth of 4% to 9%.

Financial & Liquidity Summary

Three Months Ended
December 31, Twelve Months Ended
December 31,
(unaudited)
($ and shares in millions) 2024 2023 Change 2024 2023 Change
Portfolio Receipts

742 736 1% 2,801 3,049 (8)%
Net cash provided by operating activities

743 773 (4)% 2,769 2,988 (7)%
Adjusted EBITDA (non-GAAP)*

669 682 (2)% 2,565 2,806 (9)%
Portfolio Cash Flow (non-GAAP)*

678 687 (1)% 2,452 2,708 (9)%
Weighted average Class A ordinary shares outstanding – diluted

589 598 (1)% 594 603 (1)%

*
See "Liquidity and Capital Resources" section. Adjusted EBITDA and Portfolio Cash Flow are non-GAAP liquidity measures calculated in accordance with the credit agreement.

Portfolio Receipts Highlights

Three Months Ended December 31,
(unaudited)
($ in millions) 2024 2023 Change
Products:

Marketers: Therapeutic Area:
Cystic fibrosis franchise

Vertex Rare disease 237 207 14%
Trelegy

GSK Respiratory 74 60 23%
Tysabri

Biogen Neuroscience 61 68 (11)%
Evrysdi

Roche Rare disease 56 20 182%
Xtandi

Pfizer, Astellas Cancer 46 38 20%
Imbruvica

AbbVie, J&J Cancer 46 50 (10)%
Promacta

Novartis Hematology 44 44 (1)%
Tremfya

Johnson & Johnson Immunology 39 35 11%
Cabometyx/Cometriq

Exelixis, Ipsen, Takeda Cancer 20 18 11%
Spinraza

Biogen Rare disease 15 17 (13)%
Orladeyo

BioCryst Rare disease 11 8 36%
Trodelvy

Gilead Cancer 11 10 10%
Erleada

Johnson & Johnson Cancer 11 9 25%
Nurtec ODT/Zavzpret

Pfizer Neuroscience 7 5 49%
Other products(5)

54 63 (14)%

Royalty Receipts

729 651 12%
Milestones and other contractual receipts

13 84 (85)%

Portfolio Receipts

742 736 1%
Results for full year 2024 and 2023 are shown in Table 5. Amounts shown in the table may not add due to rounding.

Royalty Receipts was $729 million in the fourth quarter of 2024, an increase of 12% as compared to $651 million in the fourth quarter of 2023. The increase was primarily driven by strong growth from Evrysdi, the cystic fibrosis franchise, Trelegy, Xtandi and Tremfya. Royalty receipts from Evrysdi included the benefit of the additional royalties acquired in October 2023 and June 2024.

Portfolio Receipts was $742 million in the fourth quarter of 2024, an increase of 1% as compared to $736 million in the fourth quarter of 2023. The increase was primarily driven by the same Royalty Receipts increases noted above, offset by a decrease in milestones and other contractual receipts, which reflected a $50 million payment related to the oral formulation of zavegepant in the prior period.

Liquidity and Capital Resources

Royalty Pharma’s liquidity and capital resources are summarized below:

As of December 31, 2024, Royalty Pharma had cash and cash equivalents of $929 million and total debt with principal value of $7.8 billion.

During the fourth quarter of 2024, Royalty Pharma repurchased approximately two million Class A ordinary shares for $50 million. For full year 2024, Royalty Pharma repurchased approximately eight million Class A ordinary shares for $230 million. The weighted-average number of diluted Class A ordinary shares outstanding for the fourth quarter of 2024 was 589 million as compared to 598 million for the fourth quarter of 2023. The weighted-average number of diluted Class A ordinary shares outstanding for full year 2024 was 594 million as compared to 603 million for full year 2023.

In January 2025, Royalty Pharma’s Board of Directors authorized a new share repurchase program under which Royalty Pharma may repurchase up to $3.0 billion of its Class A ordinary shares. Royalty Pharma intends to repurchase $2.0 billion of its shares in 2025, subject to market conditions. The total value of shares repurchased will depend on the discount to the intrinsic value at which its Class A ordinary shares are trading. This new share repurchase program replaces the unused $465 million of the company’s original $1.0 billion share repurchase program that was announced in March 2023.

Liquidity Summary

Three Months Ended
December 31, Twelve Months Ended
December 31,
(unaudited)
($ in millions) 2024 2023 2024 2023
Portfolio Receipts

742 736 2,801 3,049
Payments for operating and professional costs

(72) (54) (236) (243)
Adjusted EBITDA (non-GAAP)

669 682 2,565 2,806
Interest received/(paid), net

8 5 (113) (98)
Portfolio Cash Flow (non-GAAP)

678 687 2,452 2,708
Amounts may not add due to rounding.

•
Adjusted EBITDA (non-GAAP) was $669 million in the fourth quarter of 2024. Adjusted EBITDA is calculated as Portfolio Receipts minus payments for operating and professional costs.

•
Portfolio Cash Flow (non-GAAP) was $678 million in the fourth quarter of 2024. Portfolio Cash Flow is calculated as Adjusted EBITDA minus interest paid or received, net. This measure reflects the cash generated by Royalty Pharma’s business that can be redeployed into value-enhancing royalty acquisitions, used to repay debt, returned to shareholders through dividends or share purchases, or utilized for other discretionary investments.

Refer to Table 4 for Royalty Pharma’s reconciliation of each non-GAAP measure to the most directly comparable GAAP financial measure, net cash provided by operating activities.

Capital Deployment was $522 million in the fourth quarter of 2024, consisting primarily of the acquisitions of royalties on Niktimvo and Rytelo. Capital Deployment reflects cash payments during the period for new and previously announced transactions. Capital Deployment was $2.8 billion for full year 2024.

The table below details Capital Deployment by category:

Capital Deployment

Three Months Ended
December 31, Twelve Months Ended
December 31,
(unaudited)
($ in millions) 2024 2023 2024 2023
Acquisitions of financial royalty assets

(496) (1,002) (2,506) (2,116)
Development-stage funding payments – upfront and milestone

—  —  —  (50)
Development-stage funding payments – ongoing

(1) (1) (2) (2)
Purchases of available for sale debt securities

—  —  (150) — 
Milestone payments

(25) —  (75) (12)
Investments in equity method investees

—  (2) (11) (13)
Acquisitions of other financial assets

—  —  (18) — 
Contributions from legacy non-controlling interests – R&D

0 0 1 1

Capital Deployment

(522) (1,005) (2,761) (2,192)

Amounts may not add due to rounding.

In January 2025, Royalty Pharma announced the sale of the MorphoSys Development Funding Bonds for $511 million in upfront cash (press release). This payment, combined with payments previously received, results in total cash proceeds of $530 million on the $300 million investment that was made in September 2022. The proceeds strengthen Royalty Pharma’s balance sheet and provide added flexibility to pursue its disciplined capital allocation strategy.

Royalty Transactions

For full year 2024, Royalty Pharma announced new transactions of up to approximately $2.8 billion. The announced transactions amount reflects the entire amount of capital committed for new transactions during the year, including potential future milestones.

Recent transactions include:

•
In November 2024, Royalty Pharma acquired a synthetic royalty on Rytelo from Geron Corporation for an upfront payment of $125 million (press release). Rytelo is approved for the treatment of certain adult patients with low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia. Following the acquisition, Royalty Pharma is entitled to receive tiered royalties on U.S. net sales on Rytelo.

•
In November 2024, Royalty Pharma acquired a synthetic royalty on Niktimvo from Syndax Pharmaceuticals, Inc. for an upfront payment of $350 million (press release). Niktimvo is approved for the treatment of chronic graft-versus-host disease and will be co-commercialized by Incyte. Following the acquisition, Royalty Pharma is entitled to receive royalties on U.S. net sales on Niktimvo.

The information in this section should be read together with Royalty Pharma’s reports and documents filed with the SEC at www.sec.gov and the reader is also encouraged to review all other press releases and information available in the Investors section of Royalty Pharma’s website at www.royaltypharma.com.

Internalization Transaction

In January 2025, Royalty Pharma agreed to acquire its external manager, RP Management, LLC (the "Manager") (press release). This transaction to simplify Royalty Pharma’s corporate structure is expected to result in multiple benefits for shareholders. On a financial basis, the acquisition is expected to reduce costs and enhance economic returns on investments. Specifically, the acquisition will generate cash savings of greater than $100 million in 2026, rising to greater than $175 million in 2030 and driving cumulative savings of greater than $1.6 billion over ten years. The acquisition also increases shareholder alignment, enhances corporate governance, ensures management continuity and simplifies Royalty Pharma’s corporate structure.

The total transaction value of approximately $1.1 billion(7) consists of approximately 24.5 million shares of Royalty Pharma equity that will vest over five to nine years, approximately $100 million in cash(8), and the assumption of $380 million of the Manager’s existing debt.

The closing of the internalization transaction is subject to shareholders’ approval of the issuance of the share consideration and other customary closing conditions, including required regulatory approvals. The transaction is estimated to close during the second quarter of 2025.

Key Developments Relating to the Portfolio

The key developments related to Royalty Pharma’s royalty interests are discussed below based on disclosures from the marketers of the products.

TEV-‘749 In January 2025, Teva announced that TEV-‘749 (olanzapine LAI) achieved Phase 3 targeted injections without PDSS (post-injection delirium/sedation syndrome), and the full safety presentation is expected in the second quarter of 2025.
Cystic fibrosis franchise
In December 2024, Vertex announced the U.S. Food and Drug Administration (FDA) approval of the new triple-combination modulator Alyftrek (vanzacaftor triple) for the treatment of cystic fibrosis in people ages 6 and older with at least one responsive mutation.

In November 2024, Vertex announced that it had completed regulatory submissions for the vanzacaftor triple in the European Union, the United Kingdom, Canada, Australia, New Zealand and Switzerland, and reviews are underway.

Skytrofa In December 2024, Ascendis announced the U.S. FDA accepted for review its supplemental Biologics License Application (sBLA) in adult growth hormone deficiency for Skytrofa. The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025.
aficamten In December 2024, Cytokinetics announced that the FDA accepted its New Drug Application (NDA) for aficamten for the treatment of Obstructive Hypertrophic Cardiomyopathy. The FDA has assigned the NDA a Prescription Drug User Fee Act date of September 26, 2025. Additionally, the European Medicines Agency validated the Marketing Authorization Application for aficamten, and it will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP).
Trodelvy In November 2024, Gilead announced plans to voluntarily withdraw the U.S. accelerated approval of Trodelvy for use in pre-treated adult patients with locally advanced or metastatic urothelial cancer, following the results of the Phase 3 TROPiCS-04 trial.

Airsupra In October 2024, AstraZeneca announced that positive high-level results from the BATURA Phase 3b trial showed Airsupra met the primary endpoint, demonstrating a statistically significant and clinically meaningful reduction in the risk of a severe exacerbation when used as an as-needed rescue medication in response to symptoms compared to as-needed albuterol. These positive results triggered a milestone payment from AstraZeneca, of which Royalty Pharma received its pro rata portion of $27 million in January 2025.
MK-8189 In October 2024, Merck updated its public disclosures to remove MK-8189 from its pipeline chart and Royalty Pharma does not anticipate making a further investment in this program.
pelabresib In October 2024, Novartis announced that based on its review of 48-week data from the Phase 3 MANIFEST-2 study, longer follow-up time is needed to determine the regulatory path for pelabresib in myelofibrosis. Novartis will continue to follow patients in MANIFEST-2 and evaluate the potential for additional studies to support registration.
trontinemab In October 2024, Roche presented its latest Phase 1b/2a interim results for trontinemab at the Clinical Trials on Alzheimer’s Disease (CTAD) conference, which demonstrated rapid and robust amyloid plaque depletion after 12 to 28 weeks of treatment and an overall favorable safety profile with very limited amyloid related imaging abnormalities (ARIA-E) observed.
2025 Financial Outlook

Royalty Pharma has provided guidance for full-year 2025, excluding new transactions and borrowings announced after the date of this release, as follows:

Provided February 11, 2025

Portfolio Receipts
$2,900 million to $3,050 million

(Growth of ~+4% to 9% year/year)

Payments for operating and professional costs Approximately 10% of Portfolio Receipts(1)
Interest paid $260 million
The above Portfolio Receipts guidance represents expected growth of 4% to 9% in 2025. Royalty Pharma’s full-year 2025 guidance reflects a negligible estimated foreign exchange impact to Portfolio Receipts, assuming current foreign exchange rates prevail for the rest of 2025.

2025 guidance for payments for operating and professional costs and interest paid does not reflect the impact of the internalization transaction announced on January 10, 2025 and will be updated following the closing of the internalization transaction, which is expected to be in the second quarter of 2025.

Total interest paid is based on the semi-annual interest payment schedule of Royalty Pharma’s existing notes and is anticipated to be approximately $260 million in 2025. Interest paid is anticipated to be approximately $138 million in the first quarter of 2025, which includes the first interest payment on the $1.5 billion notes issued in June 2024. Interest paid in the third quarter of 2025 is anticipated to be $119 million. De minimis amounts are anticipated in the second and fourth quarter of 2025. These projections assume no additional debt financing in 2025, including no drawdown on the revolving credit facility. In 2024, Royalty Pharma collected interest of $46 million on its cash and cash equivalents.

Royalty Pharma today provides this guidance based on its most up-to-date view of its prospects. This guidance assumes no major unforeseen adverse events or changes in foreign exchange rates and excludes the contributions from transactions announced subsequent to the date of this press release.

Financial Results Call

Royalty Pharma will host a conference call and simultaneous webcast to discuss its fourth quarter and full year 2024 results today at 8:30 a.m., Eastern Time. Please visit the "Investors" page of the company’s website at View Source to obtain conference call information and to view the live webcast. A replay of the conference call and webcast will be archived on the company’s website for at least 30 days.

On February 11, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the Biologics License Application (BLA) for linvoseltamab for the treatment of adult patients with relapsed/refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy or those who received three prior lines of therapy and are refractory to the last line of therapy (Press release, Regeneron, FEB 11, 2025, View Source [SID1234650174]). The target action date for the FDA decision is July 10, 2025.

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Acceptance of the BLA resubmission follows the resolution of third-party fill/finish manufacturing issues, which was the sole approvability issue identified by the FDA in the previous submission. The BLA is supported by data from the pivotal LINKER-MM1 trial investigating linvoseltamab in R/R MM, and linvoseltamab is also under review by the European Medicines Agency (EMA) for the same patient population.

Linvoseltamab is investigational and has not been approved by any regulatory authority.

About Multiple Myeloma
As the second most common blood cancer, there are over 187,000 new cases of MM diagnosed globally every year, with more than 36,000 diagnosed and 12,000 deaths anticipated in the U.S. in 2025. In the U.S., there are approximately 8,000 people who have MM that has progressed after three lines of therapy, and 4,000 whose disease has progressed after four or more therapies. The disease is characterized by the proliferation of cancerous plasma cells (MM cells) that crowd out healthy blood cells in the bone marrow, infiltrate other tissues and cause potentially life-threatening organ injury. Despite treatment advances, MM is not curable and while current treatments are able to slow progression of the cancer, most patients will ultimately experience cancer progression and require additional therapies.

About the Linvoseltamab Clinical Development Program
Linvoseltamab is an investigational BCMAxCD3 bispecific antibody designed to bridge B-cell maturation antigen (BCMA) on MM cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.

The ongoing, open-label, multicenter Phase 1/2 dose-escalation and dose-expansion LINKER-MM1 trial is investigating linvoseltamab in 282 enrolled patients with relapsed/refractory MM. The Phase 1 dose-escalation portion of the trial – which is now complete – primarily assessed safety, tolerability and dose-limiting toxicities across nine dose levels of linvoseltamab and explored different administration regimens. The ongoing Phase 2 dose expansion portion is assessing the safety and anti-tumor activity of linvoseltamab, with the primary endpoint of objective response rate. Key secondary endpoints include duration of response, progression-free survival, rate of minimum residual disease negative status and overall survival.

Eligibility in the Phase 2 portion requires patients to have received at least three prior lines of therapy or have triple-class refractory MM. Linvoseltamab is administered with an initial step-up dosing regimen followed by the full 200 mg dose administered weekly. At week 16, all patients transition to every two-week dosing. A response-adapted regimen further enables patients to shift to every four-week dosing if they achieve a very good partial response or better and have completed at least 24 weeks of therapy. The regimen requires a total of two 24-hour hospitalizations for safety monitoring.

Linvoseltamab is being investigated in a broad clinical development program exploring its use as a monotherapy as well as in combination regimens across different lines of therapy in MM, including earlier lines of treatment, as well as plasma cell precursor disorders. They include evaluating linvoseltamab in a Phase 1b trial (LINKER-MM2) in combination with other cancer treatments in R/R MM as well as a Phase 3 confirmatory trial (LINKER-MM3) as a monotherapy in R/R MM. For more information on Regeneron’s clinical trials in blood cancer, visit the clinical trials website, or contact via clinicaltrials@regeneron.com or 844-734-6643.