Advancing Novel treatments for the Hematological Malignancies

On January 8, 2024 Syros presented its corporate presentation (Presentation, Syros Pharmaceuticals, JAN 8, 2024, View Source [SID1234639113]).

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Company Overview

On January 8, 2024 Sutro Biopharma presented its corporate presentation (Presentation, Sutro Biopharma, JAN 8, 2024, View Source [SID1234639112]).

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SpringWorks Therapeutics Highlights 2023 Accomplishments and Anticipated Milestones for 2024 at the 42nd Annual J.P. Morgan Healthcare Conference

On January 8, 2024 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported that it will present at the 42nd Annual J.P. Morgan Healthcare Conference at 7:30 a.m. PT (10:30 a.m. ET), and a live webcast will be available at ir.springworkstx.com (Press release, SpringWorks Therapeutics, JAN 8, 2024, View Source [SID1234639111]). Ahead of the presentation, the Company highlighted its 2023 accomplishments and announced its anticipated key milestones for 2024.

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2023 Accomplishments

Nirogacestat

Received approval from the United States Food and Drug Administration (FDA) for OGSIVEO (nirogacestat), an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.
Published data from the Phase 3 DeFi trial of nirogacestat in adult patients with progressing desmoid tumors in the New England Journal of Medicine.
Presented additional data from the Phase 3 DeFi trial at several leading medical conferences. These presentations demonstrated rapid, sustained and consistent improvements in pain and functional status in patients receiving OGSIVEO using multiple assessment tools.
Completed enrollment in the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with recurrent ovarian granulosa cell tumors (OvGCT).
Continued to evaluate nirogacestat in patients with multiple myeloma as part of several B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in collaboration with industry leaders. Clinical data from two BCMA combination studies were presented at the European Hematology Association (EHA) (Free EHA Whitepaper) congress and provided further validation of the mechanistic approach supporting nirogacestat’s ability to enhance the activity of BCMA-directed therapies across modalities. In addition, a Regeneron-sponsored study was initiated to evaluate nirogacestat in combination with linvoseltamab, Regeneron’s bispecific antibody targeting BCMA and CD3.
Mirdametinib (NF1-PN)

Presented positive topline data from the pivotal Phase 2b ReNeu trial evaluating mirdametinib, an investigational oral MEK inhibitor, in pediatric and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). Data demonstrated a confirmed objective response rate of 52% in pediatric patients and 41% in adult patients, as assessed by Blinded Independent Central Review. Mirdametinib treatment resulted in deep and durable responses and led to significant improvements in key secondary patient-reported outcome measures. Mirdametinib was generally well tolerated in the ReNeu trial, with the majority of adverse events being Grade 1 or Grade 2.
Emerging Pipeline

Presented updated data from the Phase 1a/1b study of brimarafenib (BGB-3245), an investigational, selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, Ltd., in adult patients with advanced or refractory solid tumors harboring MAPK pathway aberrations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting. These data supported the advancement of brimarafenib into the Phase 1b cohort expansion portion of the study.
Shared updated clinical data from the Phase 1b trial evaluating mirdametinib in combination with BeiGene’s investigational RAF dimer inhibitor, lifirafenib, in patients with advanced or refractory solid tumors with RAS mutations, RAF mutations and other MAPK pathway aberrations at AACR (Free AACR Whitepaper). The combination showed antitumor activity in patients with various mutations across several solid tumor types and support the advancement of this combination into the dose-expansion portion of the study, which is evaluating the combination in patients with NRAS-mutated solid tumors.
Dosed the first patient in a Phase 1/2a combination study of brimarafenib and mirdametinib.
The FDA cleared the Investigational New Drug (IND) application submitted through MapKure for a combination study of brimarafenib with panitumumab, a monoclonal antibody targeting EGFR, in colorectal and pancreatic cancer patients with known MAPK pathway mutations. Amgen Inc. is supplying panitumumab pursuant to a clinical trial collaboration agreement with MapKure.
Submitted an IND application for SW-682, a novel, potent, and selective TEAD inhibitor development candidate targeting tumors driven by Hippo pathway mutations.
General Corporate

Continued to expand and strengthen the intellectual property portfolios for nirogacestat and mirdametinib, with Orange Book listable patents providing protection past 2040 for both nirogacestat and mirdametinib.
Strengthened balance sheet with upsized public offering; gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, were approximately $316.25 million. SpringWorks estimates that its cash, cash equivalents and marketable securities as of September 30, 2023 exceeded $700 million on a pro forma basis.
Anticipated 2024 Key Milestones

Nirogacestat

Continue to advance U.S. launch of OGSIVEO (nirogacestat) as the first and only approved therapy for adult patients with desmoid tumors and establish as standard of care.
Submit Marketing Authorisation Application (MAA) for nirogacestat for the treatment of adult patients with desmoid tumors to the European Medicines Agency (EMA) in the first half of 2024.
Report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with OvGCT in the second half of 2024.
Expand data set with additional clinical data of nirogacestat in combination with BCMA-directed therapies.
Mirdametinib (NF1-PN)

Submit a New Drug Application (NDA) to the FDA for mirdametinib for the treatment of children and adults with NF1-PN in the first half of 2024.
Present data from the pediatric and adult cohorts of the Phase 2b ReNeu trial of mirdametinib in NF1-PN at a medical congress in the first half of 2024 and submit for publication in a peer-reviewed journal in 2024.
Emerging Pipeline

Present additional data for brimarafenib as a monotherapy in MAPK-mutant solid tumors in the second half of 2024.
Support initiation of Phase 1b trial of brimarafenib in combination with panitumumab, a monoclonal antibody targeting EGFR, in colorectal and pancreatic cancer patients with known MAPK pathway mutations in the first quarter of 2024.
Initiate Phase 1 trial of SW-682, SpringWorks’ TEAD inhibitor, in Hippo mutant solid tumors in the first half of 2024.
"Our focus in 2024 is to deliver a successful U.S. launch of OGSIVEO as the first and only FDA-approved therapy for adults with desmoid tumors and to prepare for our second potential FDA approval by filing our NDA for mirdametinib as a treatment for patients with NF1-PN in the first half of the year," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are very pleased with our progress towards our goal of having two marketed products by 2025 given the highly positive topline data from our Phase 2b ReNeu trial and will simultaneously advance our broader pipeline of targeted oncology programs. Our achievements in 2023, our strong financial position and the durable IP protections for our lead assets position us for continued, long-term success as we execute on our mission to improve the lives of patients with devastating diseases."

Presentation at the 42nd Annual J.P. Morgan Healthcare Conference

SpringWorks will webcast its presentation from the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, 2024 at 7:30 a.m. PT (10:30 a.m. ET). To access the live webcast, please visit the Events & Presentations page within the Investors & Media section of the company’s website at View Source A replay of the webcast will be available on SpringWorks’ website for a limited time following the conference.

European Commission Approves Pfizer’s TALZENNA® in Combination with XTANDI® for Adult Patients with Metastatic Castration-Resistant Prostate Cancer

On January 8, 2024 Pfizer Inc. (NYSE: PFE) reported that the European Commission (EC) has approved TALZENNA (talazoparib), an oral poly ADP-ribose polymerase (PARP) inhibitor, in combination with XTANDI (enzalutamide), for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated (Press release, Seagen, JAN 8, 2024, View Source [SID1234639110]). With this approval, TALZENNA is now the first and only PARP inhibitor licensed in the European Union for use with XTANDI for patients with mCRPC, with or without gene mutations.

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"New treatment options are needed to increase the proportion of patients with metastatic castration-resistant prostate cancer who can benefit from current anticancer medicines that keep the disease under control for longer," said Robert Jones, MBChB, PhD, Professor of Clinical Cancer Research, University of Glasgow. "The European Commission’s approval of talazoparib in combination with enzalutamide offers a meaningful advancement for the treatment of patients with metastatic castration-resistant prostate cancer, the most advanced and aggressive stage of the disease."

"After years of fighting prostate cancer, it can be devastating for a patient to learn that their cancer has stopped responding to testosterone-lowering treatments. At this stage of the disease, the prognosis is generally poor," said Erik Briers, MS, PhD, Vice Chairman, Europa UOMO, a European advocacy movement for people with prostate cancer. "Patients urgently need new treatment options and TALZENNA in combination with XTANDI can bring new hope to these patients."

This approval by the European Commission of TALZENNA in combination with XTANDI for the mCRPC indication is valid in all 27 EU member states plus Iceland, Liechtenstein, and Norway.

The approval is based on data from the Phase 3 TALAPRO-2 trial, a multicenter, randomized, double-blind, placebo-controlled study, evaluating two mCRPC patient cohorts: Cohort 1 (all-comers [n=805]) and Cohort 2 (those with HRR gene mutations [HRRm; n=399]). The results from TALAPRO-2 Cohort 1, which were published in The Lancet, showed that treatment with TALZENNA plus XTANDI reduced the risk of disease progression or death by 37% versus placebo plus XTANDI (Hazard Ratio [HR]: 0.63; 95% Confidence Interval [CI], 0.51–0.78; P< 0.0001), meeting the study’s primary endpoint of improving radiographic progression-free survival (rPFS). At the time of the analysis, the median rPFS for those treated with TALZENNA plus XTANDI had not yet been reached versus 21.9 months for those treated with placebo plus XTANDI. Median rPFS is defined as the timepoint in which 50% of patients in each treatment arms have progressed. A trend in overall survival (OS), a key secondary endpoint, favoring TALZENNA plus XTANDI was also observed, though these data are immature. The safety of TALZENNA plus XTANDI in the TALAPRO-2 trial was generally consistent with the known safety profile of each medicine.

"Today’s approval of TALZENNA in combination with XTANDI represents an important advancement for men living with prostate cancer in Europe," said Chris Boshoff, M.D., Ph.D., Chief Oncology Officer, Executive Vice President, Pfizer. "The results from the pivotal TALAPRO-2 trial showed that this combination offers an effective treatment that addresses disease progression in patients with or without any specific gene mutation."

TALZENNA in combination with XTANDI was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with HRR gene-mutated mCRPC in June 2023. Pfizer has also shared the TALAPRO-2 data with other regulatory agencies to support regulatory filings.

About Metastatic Castration-Resistant Prostate Cancer

Metastatic castration-resistant prostate cancer (mCRPC) is a cancer that has spread beyond the prostate gland and has progressed despite medical or surgical treatment to lower testosterone. There were ~1.4 million new cases of prostate cancer reported worldwide in 2020, of which ~470,000 new cases were in Europe.1 Approximately 10%–20% of prostate cancer patients develop mCRPC within 5−7 years of diagnosis.2 Between 1.2%–2.1% of all prostate cancer cases globally are mCRPC.3

About TALAPRO-2

The Phase 3 TALAPRO-2 trial is a two-part, two-cohort, multicenter, randomized, double-blind, placebo-controlled study that enrolled 1,106 patients with mCRPC (with no systemic treatments initiated after documentation of mCRPC) at sites in the U.S., Canada, Europe, South America, and the Asia-Pacific region. The study included two patient cohorts: all-comers (n=805) and those with and without gene mutations (HRRm; n=399). Patients on androgen deprivation therapy (ADT) or who had bilateral orchiectomy in the trial were randomized to receive TALZENNA 0.5 mg/day plus XTANDI 160mg/day, or placebo plus XTANDI 160 mg/day.

The primary endpoint of the trial is radiographic progression-free survival (rPFS), defined as the time from the date of randomization to first objective evidence of radiographic progression by blinded independent review, or death, whichever occurs first, in both Cohort 1 (all-comers) and Cohort 2 (those with HRRm). Secondary endpoints include overall survival (OS), objective response rate, duration of response, and PSA response.

For more information on the TALAPRO-2 trial (NCT03395197), go to www.clinicaltrials.gov.

About TALZENNA (talazoparib)

TALZENNA (talazoparib) is an oral inhibitor of poly ADP-ribose polymerase (PARP), which plays a role in DNA damage repair. Preclinical studies have demonstrated that TALZENNA blocks PARP enzyme activity and traps PARP at the site of DNA damage, leading to decreased cancer cell growth and cancer cell death.

TALZENNA is approved in over 70 countries, including the U.S and the EU, as a once-daily monotherapy for the treatment of adult patients with deleterious or suspected deleterious germline breast cancer susceptibility gene (BRCA)-mutated (gBRCAm) human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer. In the U.S., TALZENNA is approved in combination with XTANDI (enzalutamide) for the treatment of adult patients with homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC). In the EU, TALZENNA is now approved in combination with enzalutamide for the treatment of adult patients with mCRPC in whom chemotherapy is not clinically indicated.

TALZENNA (talazoparib) Indication in the U.S.

TALZENNA is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated for:

Breast Cancer:

As a single agent, for the treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) HER2-negative locally advanced or metastatic breast cancer. Select patients for therapy based on an FDA-approved companion diagnostic for TALZENNA.
HRR Gene-mutated mCRPC:

In combination with enzalutamide for the treatment of adult patients with homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC).
TALZENNA (talazoparib) Important Safety Information

WARNINGS and PRECAUTIONS

Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML), including cases with a fatal outcome, has been reported in patients who received TALZENNA. Overall, MDS/AML has been reported in 0.4% (3 out of 788) of solid tumor patients treated with TALZENNA as a single agent in clinical studies. In TALAPRO-2, MDS/AML occurred in 2 out of 511 (0.4%) patients treated with TALZENNA and enzalutamide and in 0 out of 517 (0%) patients treated with placebo and enzalutamide. The durations of TALZENNA treatment in these five patients prior to developing MDS/AML were 0.3, 1, 2, 3, and 5 years, respectively. Most of these patients had received previous chemotherapy with platinum agents and/or other DNA damaging agents including radiotherapy.

Do not start TALZENNA until patients have adequately recovered from hematological toxicity caused by previous chemotherapy. Monitor blood counts monthly during treatment with TALZENNA. For prolonged hematological toxicities, interrupt TALZENNA and monitor blood counts weekly until recovery. If counts do not recover within 4 weeks, refer the patient to a hematologist for further investigations including bone marrow analysis and blood sample for cytogenetics. If MDS/AML is confirmed, discontinue TALZENNA.

Myelosuppression consisting of anemia, neutropenia, and/or thrombocytopenia have been reported in patients treated with TALZENNA. In TALAPRO-2, Grade ≥3 anemia, neutropenia, and thrombocytopenia were reported, respectively, in 45%, 18%, and 8% of patients receiving TALZENNA and enzalutamide. Overall, 39% of patients (199/511) required a red blood cell transfusion, including 22% (111/511) who required multiple transfusions. Discontinuation due to anemia, neutropenia, and thrombocytopenia occurred, respectively, in 7%, 3%, and 0.4% of patients.

Withhold TALZENNA until patients have adequately recovered from hematological toxicity caused by previous therapy. Monitor blood counts monthly during treatment with TALZENNA. If hematological toxicities do not resolve within 28 days, discontinue TALZENNA and refer the patient to a hematologist for further investigations including bone marrow analysis and blood sample for cytogenetics.

Embryo-Fetal Toxicity TALZENNA can cause fetal harm when administered to pregnant women. Advise male patients with female partners of reproductive potential or who are pregnant to use effective contraception during treatment with TALZENNA and for 4 months after receiving the last dose.

ADVERSE REACTIONS

Serious adverse reactions reported in >2% of patients included anemia (9%) and fracture (3%). Fatal adverse reactions occurred in 1.5% of patients, including pneumonia, COVID infection, and sepsis (1 patient each).

The most common adverse reactions (≥ 10%, all Grades), including laboratory abnormalities, for patients in the TALAPRO-2 study who received TALZENNA in combination with enzalutamide vs patients receiving placebo with enzalutamide were hemoglobin decreased (79% vs 34%), neutrophils decreased (60% vs 18%), lymphocytes decreased (58% vs 36%), fatigue (49% vs 40%), platelets decreased (45% vs 8%), calcium decreased (25% vs 11%), nausea (21% vs 17%), decreased appetite (20% vs 14%), sodium decreased (22% vs 20%), phosphate decreased (17% vs 13%), fractures (14% vs 10%), magnesium decreased (14% vs 12%), dizziness (13% vs 9%), bilirubin increased (11% vs 7%), potassium decreased (11% vs 7%), and dysgeusia (10% vs 4.5%).

Clinically relevant adverse reactions in <10% of patients who received TALZENNA with enzalutamide included abdominal pain (9%), vomiting (9%), alopecia (7%), dyspepsia (4%), venous thromboembolism (3%) and stomatitis (2%).

Based on animal studies, TALZENNA may impair fertility in males of reproductive potential.

DRUG INTERACTIONS

Coadministration with P-gp inhibitors The effect of coadministration of P-gp inhibitors on talazoparib exposure when TALZENNA is taken in combination with enzalutamide has not been studied. Monitor patients for increased adverse reactions and modify the dosage as recommended for adverse reactions when TALZENNA is coadministered with a P-gp inhibitor.

Coadministration with BCRP inhibitors Monitor patients for increased adverse reactions and modify the dosage as recommended for adverse reactions when TALZENNA is coadministered with a BCRP inhibitor. Coadministration of TALZENNA with BCRP inhibitors may increase talazoparib exposure, which may increase the risk of adverse reactions.

USE IN SPECIFIC POPULATIONS

Renal Impairment The recommended dosage of TALZENNA for patients with moderate renal impairment (CLcr 30 – 59 mL/min) is 0.35 mg taken orally once daily in combination with enzalutamide. The recommended dosage of TALZENNA for patients with severe renal impairment (CLcr 15 – 29 mL/min) is 0.25 mg taken orally once daily in combination with enzalutamide. No dose adjustment is required for patients with mild renal impairment. TALZENNA has not been studied in patients requiring hemodialysis.

Please see full U.S. Prescribing Information and Patient Information for TALZENNA (talazoparib) at www.TALZENNA.com.

About XTANDI (enzalutamide) and Important Safety Information

XTANDI (enzalutamide) is an androgen receptor signaling inhibitor. XTANDI is a standard of care and has received regulatory approvals in one or more countries around the world for use in men with metastatic castration-sensitive prostate cancer (mCSPC; also known as metastatic hormone-sensitive prostate cancer or mHSPC), metastatic castration-resistant prostate cancer (mCRPC), non-metastatic castration-resistant prostate cancer (nmCRPC) and nonmetastatic castration-sensitive prostate cancer (nmCSPC) with biochemical recurrence at high risk for metastasis (high-risk BCR). XTANDI is currently approved for one or more of these indications in more than 90 countries, including in the U.S., EU, and Japan. Over one million patients have been treated with XTANDI globally.4

Warnings and Precautions

Seizure occurred in 0.6% of patients receiving XTANDI in eight randomized clinical trials. In a study of patients with predisposing factors for seizure, 2.2% of XTANDI-treated patients experienced a seizure. It is unknown whether anti-epileptic medications will prevent seizures with XTANDI. Patients in the study had one or more of the following predisposing factors: use of medications that may lower the seizure threshold, history of traumatic brain or head injury, history of cerebrovascular accident or transient ischemic attack, and Alzheimer’s disease, meningioma, or leptomeningeal disease from prostate cancer, unexplained loss of consciousness within the last 12 months, history of seizure, presence of a space occupying lesion of the brain, history of arteriovenous malformation, or history of brain infection. Advise patients of the risk of developing a seizure while taking XTANDI and of engaging in any activity where sudden loss of consciousness could cause serious harm to themselves or others. Permanently discontinue XTANDI in patients who develop a seizure during treatment.

Posterior Reversible Encephalopathy Syndrome (PRES) There have been reports of PRES in patients receiving XTANDI. PRES is a neurological disorder that can present with rapidly evolving symptoms including seizure, headache, lethargy, confusion, blindness, and other visual and neurological disturbances, with or without associated hypertension. A diagnosis of PRES requires confirmation by brain imaging, preferably MRI. Discontinue XTANDI in patients who develop PRES.

Hypersensitivity reactions, including edema of the face (0.5%), tongue (0.1%), or lip (0.1%) have been observed with XTANDI in eight randomized clinical trials. Pharyngeal edema has been reported in post-marketing cases. Advise patients who experience any symptoms of hypersensitivity to temporarily discontinue XTANDI and promptly seek medical care. Permanently discontinue XTANDI for serious hypersensitivity reactions.

Ischemic Heart Disease In the combined data of five randomized, placebo-controlled clinical studies, ischemic heart disease occurred more commonly in patients on the XTANDI arm compared to patients on the placebo arm (3.5% vs 2%). Grade 3-4 ischemic events occurred in 1.8% of patients on XTANDI versus 1.1% on placebo. Ischemic events led to death in 0.4% of patients on XTANDI compared to 0.1% on placebo. Monitor for signs and symptoms of ischemic heart disease. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Discontinue XTANDI for Grade 3-4 ischemic heart disease.

Falls and Fractures occurred in patients receiving XTANDI. Evaluate patients for fracture and fall risk. Monitor and manage patients at risk for fractures according to established treatment guidelines and consider use of bone-targeted agents. In the combined data of five randomized, placebo-controlled clinical studies, falls occurred in 12% of patients treated with XTANDI compared to 6% of patients treated with placebo. Fractures occurred in 13% of patients treated with XTANDI and in 6% of patients treated with placebo.

Embryo-Fetal Toxicity The safety and efficacy of XTANDI have not been established in females. XTANDI can cause fetal harm and loss of pregnancy when administered to a pregnant female. Advise males with female partners of reproductive potential to use effective contraception during treatment with XTANDI and for 3 months after the last dose of XTANDI.

Adverse Reactions (ARs)

In the data from the five randomized placebo-controlled trials, the most common ARs (≥ 10%) that occurred more frequently (≥ 2% over placebo) in XTANDI-treated patients were musculoskeletal pain, fatigue, hot flush, constipation, decreased appetite, diarrhea, hypertension, hemorrhage, fall, fracture, and headache. In the bicalutamide-controlled study, the most common ARs (≥ 10%) reported in XTANDI-treated patients were asthenia/fatigue, back pain, musculoskeletal pain, hot flush, hypertension, nausea, constipation, diarrhea, upper respiratory tract infection, and weight loss.

In AFFIRM, the placebo-controlled study of metastatic CRPC (mCRPC) patients who previously received docetaxel, Grade 3 and higher ARs were reported among 47% of XTANDI-treated patients. Discontinuations due to ARs were reported for 16% of XTANDI-treated patients. In PREVAIL, the placebo-controlled study of chemotherapy-naive mCRPC patients, Grade 3-4 ARs were reported in 44% of XTANDI patients and 37% of placebo patients. Discontinuations due to ARs were reported for 6% of XTANDI-treated patients. In TERRAIN, the bicalutamide-controlled study of chemotherapy-naive mCRPC patients, Grade 3-4 ARs were reported in 39% of XTANDI patients and 38% of bicalutamide patients. Discontinuations with an AR as the primary reason were reported for 8% of XTANDI patients and 6% of bicalutamide patients.

In PROSPER, the placebo-controlled study of nonmetastatic CRPC (nmCRPC) patients, Grade 3 or higher ARs were reported in 31% of XTANDI patients and 23% of placebo patients. Discontinuations with an AR as the primary reason were reported for 9% of XTANDI patients and 6% of placebo patients.

In ARCHES, the placebo-controlled study of metastatic CSPC (mCSPC) patients, Grade 3 or higher ARs were reported in 24% of XTANDI-treated patients. Permanent discontinuation due to ARs as the primary reason was reported in 5% of XTANDI patients and 4% of placebo patients.

In EMBARK, the placebo-controlled study of nonmetastatic CSPC (nmCSPC) with high-risk biochemical recurrence (BCR) patients, Grade 3 or higher adverse reactions during the total duration of treatment were reported in 46% of patients treated with XTANDI plus leuprolide, 50% of patients receiving XTANDI as a single agent, and 43% of patients receiving placebo plus leuprolide. Permanent treatment discontinuation due to adverse reactions during the total duration of treatment as the primary reason was reported in 21% of patients treated with XTANDI plus leuprolide, 18% of patients receiving XTANDI as a single agent, and 10% of patients receiving placebo plus leuprolide.

Lab Abnormalities: Lab abnormalities that occurred in ≥ 5% of patients, and more frequently (> 2%) in the XTANDI arm compared to placebo in the pooled, randomized, placebo-controlled studies are hemoglobin decrease, neutrophil count decreased, white blood cell decreased, hyperglycemia, hypermagnesemia, hyponatremia, hyperphosphatemia, and hypercalcemia.

Hypertension: In the combined data from five randomized placebo-controlled clinical trials, hypertension was reported in 14.2% of XTANDI patients and 7.4% of placebo patients. Hypertension led to study discontinuation in < 1% of patients in each arm.

Drug Interactions

Effect of Other Drugs on XTANDI Avoid coadministration with strong CYP2C8 inhibitors. If coadministration cannot be avoided, reduce the dosage of XTANDI.

Avoid coadministration with strong CYP3A4 inducers. If coadministration cannot be avoided, increase the dosage of XTANDI.

Effect of XTANDI on Other Drugs Avoid coadministration with certain CYP3A4, CYP2C9, and CYP2C19 substrates for which minimal decrease in concentration may lead to therapeutic failure of the substrate. If coadministration cannot be avoided, increase the dosage of these substrates in accordance with their Prescribing Information. In cases where active metabolites are formed, there may be increased exposure to the active metabolites.

Please see Full Prescribing Information for additional safety information.

ROYALTY PHARMA HIGHLIGHTS ACCOMPLISHMENTS AND PROVIDES BUSINESS UPDATE AT 42nd ANNUAL J.P. MORGAN HEALTHCARE CONFERENCE

On January 8, 2024 Royalty Pharma plc (Nasdaq: RPRX) reported an update on its business performance, including recent key accomplishments, and the full year 2023 outlook for Portfolio Receipts (Press release, Royalty Pharma , JAN 8, 2024, View Source [SID1234639108]). Pablo Legorreta, Royalty Pharma’s founder and Chief Executive Officer, will discuss these updates tomorrow as part of a webcast presentation at the 42nd Annual J.P. Morgan Healthcare Conference to be held at 12:00 p.m. Eastern Time / 9:00 a.m. Pacific Time.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Royalty Pharma delivered record performance in 2023," said Pablo Legorreta. "We expect to achieve an all-time high for Portfolio Receipts of approximately $3.05 billion, representing our third consecutive year of double-digit underlying growth since our initial public offering in 2020. We deployed substantial capital to acquire royalties on eight therapies, including our strongest year ever for synthetic royalty transactions. We remain in an excellent position to deliver compounding growth in the years to come while executing on our mission and vision to accelerate innovation in life sciences and transform patient lives globally."

Record 2023 Financial Performance

Based on preliminary unaudited fourth quarter 2023 results, Royalty Pharma now expects to deliver Portfolio Receipts for full year 2023 of approximately $3,050 million, which includes a $50 million payment related to oral zavegepant and exceeds the upper end of its previous guidance range of $2,950 million to $3,000 million. This represents underlying growth of 11% year-over-year prior to Biohaven-related payments and reflects the strong performance of Royalty Pharma’s diversified royalty portfolio. Royalty Pharma also expects Net cash provided by operating activities to be approximately $2,980 million to $2,990 million for full year 2023.

Portfolio Receipts was previously referred to as Adjusted Cash Receipts. The calculation of Portfolio Receipts will result in the same total as under Royalty Pharma’s previous presentation of Adjusted Cash Receipts. This change of presentation will facilitate increased transparency into the economics of individual royalties, as Royalty Receipts by product and franchise, will be reported net of legacy non-controlling interests.

Royalty Pharma’s preliminary unaudited fourth quarter 2023 results provided in this press release are subject to change in connection with the completion of the company’s final adjustments and other developments that may arise during the preparation and audit of its financial statements. Royalty Pharma’s management will host a conference call to discuss Royalty Pharma’s fourth quarter and full year 2023 results in February 2024.

Strong Capital Deployment Added Innovative Therapies, Enhancing Long-term Growth

Since 2020, Royalty Pharma has announced transactions of approximately $13 billion, including $4.0 billion in 2023. Important additions to Royalty Pharma’s portfolio in the past year have included incremental royalties on Evrysdi, the fast-growing therapy for spinal muscular atrophy, as well as the potentially practice-changing, development-stage compounds pelacarsen for cardiovascular disease and KarXT for schizophrenia. Royalty Pharma also had its strongest year ever for synthetic royalties with announced transactions of $775 million, including Adstiladrin for bladder cancer, which is the first gene therapy added to its portfolio, Skytrofa, an approved therapy for growth hormone deficiency and TEV-‘749, an exciting development-stage compound for schizophrenia.

In total, 34 unique therapies have been added to the company’s portfolio since 2020 (of which 17 are either currently or projected to be blockbusters that generate annual sales of $1 billion or more based on consensus estimates). In aggregate, based on consensus sales forecasts, investments made since 2020 are estimated to add approximately $1.2 billion to Royalty Pharma’s Portfolio Receipts in 2025.

Biopharma Funding Environment Driving New Royalty Opportunities

The biopharmaceutical ecosystem is generating significant demand for capital to fund the ongoing wave of healthcare innovation. Reflecting this positive market backdrop, between 2019 and 2023, the number of in-depth reviews of new royalty opportunities conducted by Royalty Pharma increased by 133%, resulting in an 80% increase in announced annual transaction value (from $2.2 billion in 2019 to $4.0 billion in 2023).

Given Royalty Pharma’s unique role at the heart of funding life sciences innovation, Royalty Pharma believes that there will be significant opportunity to deploy capital and fund innovation, while creating value for its stakeholders. This is reflected in the company’s capital deployment target of $10 billion to $12 billion from 2022 to 2026 and in Royalty Pharma’s expectation that it has the potential over the longer term to double its average annual capital deployment to $4 billion to $5 billion.

Webcast of J.P. Morgan Healthcare Conference

Royalty Pharma will present at the 42nd Annual J.P. Morgan Healthcare Conference at 12:00 p.m. ET / 9:00 a.m. PT tomorrow. The webcast will be accessible from Royalty Pharma’s "Events" page at View Source The webcast will also be archived for a minimum of thirty days.