A Phase II Study of Type-1 Polarized Dendritic Cell (aDC1) -Based Treatment in Combination With Tumor-Selective Chemokine Modulation (CKM: Interferon Alpha 2b, Rintatolimod and Celecoxib) in Melanoma Patients With Primary PD-1/PD-L1 Resistance

This phase II trial studies how well polarized dendritic cell (aDC1) based therapy, interferon alpha-2, rintatolimod, and celecoxib work together in treating patients with HLA-A2 positive (+) melanoma that has not responded to previous treatment (refractory). The aDC1 cell-based treatment contains white blood cells (dendritic cells or DCs) that stimulates the immune system. Interferon alpha-2 can improve the body's natural response to infections and other diseases. It can also interfere with the division of cancer cells and slow tumor growth. Rintalolimid may stimulate the immune system. Celecoxib is a drug that reduces pain. This study is being done to find out if the combination of the study cell-based treatment (aDC1 dendritic cells) and interferon alpha-2, rintatolimod, and celecoxib can prevent the growth and/or progression of melanoma.

Start Date20 Nov 2024

Sponsor / Collaborator

Roswell Park Comprehensive Cancer Center

[+1]

NCT04379518 / SuspendedPhase 1/2IIT

Phase 1/2A Study of Rintatolimod and IFN Alpha Regimen in Cancer Patients With COVID-19

This phase I/IIa trial studies the best dose and side effects of rintatolimod and interferon (IFN) alpha-2b in treating cancer patients with COVID-19 infection. Interferon alpha is a protein important for defense against viruses. It activates immune responses that help to clear viral infection. Rintatolimod is double stranded ribonucleic acid (RNA) designed to mimic viral infection by stimulating immune pathways that are normally activated during viral infection. Giving rintatolimod and interferon alpha-2b may activate the immune system to limit the replication and spread of the virus.

Start Date17 Nov 2020

Sponsor / Collaborator

Roswell Park Comprehensive Cancer Center

[+1]

CTRI/2020/04/024498 / Not yet recruitingPhase 2

COMPARATIVE EVALUATION OF TOPICAL CYCLOSPORINE A AND TOPICAL INTERFERON ALPHA 2B AS AN ADJUNCT TO LIMBAL CONJUNCTIVAL AUTOGRAFT IN PTERYGIUM SURGERY

Start Date15 Apr 2020

Sponsor / Collaborator-

100 Clinical Results associated with INTERFERON ALFA-2B(Merck Sharp & Dohme )

100 Translational Medicine associated with INTERFERON ALFA-2B(Merck Sharp & Dohme )

100 Patents (Medical) associated with INTERFERON ALFA-2B(Merck Sharp & Dohme )

1,383

Literatures (Medical) associated with INTERFERON ALFA-2B(Merck Sharp & Dohme )

01 Sep 2024·EUROPEAN JOURNAL OF OPHTHALMOLOGY

Unusual presentation of ocular surface squamous neoplasia in a young healthy patient: A case report

Article

Author: Paganoni, Giorgio ; Knutsson, Karl Anders ; Rama, Paolo ; Bandello, Francesco ; Tombolini, Beatrice

Purpose:

To report an unusual case of ocular surface squamous neoplasia (OSSN) associated with human papilloma virus (HPV)-16 infection with an atypical morphology in a young otherwise healthy patient.

Case description:

A 17 year-old healthy male was referred to our department for evaluation of a corneal infiltrate with anterior stromal neovascularization in the right eye. One year before, the patient underwent an excision of a corneo-conjunctival lesion that was located inferiorly in the same eye. Histopathological analysis had shown moderate and severe dysplasia of the conjunctival epithelium and resulted positive for HPV-16. We performed a diagnostic incisional biopsy of the limbal conjunctiva and of the corneal epithelium for histological examination and molecular testing for HPV and Chlamydia by using polymerase chain reaction (PCR). Histopathologic evaluation demonstrated low-grade dysplasia of conjunctiva. PCR testing of the corneal epithelium was positive for HPV-16, similarly to the first biopsy performed by another centre. The patient was successfully treated with topical interferon alfa-2b (1,000,000 IU/ml) for a total of six months. After the treatment, the corneal infiltrate improved dramatically with regression of neovascularization and improvement of corneal transparency and vision.

Discussion:

The present report described an atypical presentation of HPV-related OSSN due to its unusual morphology, young age of onset and absence of associated comorbidity.

Conclusion:

Conservative treatment with topical interferon-alpha 2b could be used to treat successfully HPV-16 positive OSSN, with no corneal irregularity or potential loss of vision compared to surgical excision.

01 Jul 2024·Annals of hematology

ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options

Article

Author: Mayer, Jiri ; De Stefano, Valerio ; Alvarez-Larrán, Alberto ; Klade, Christoph ; Tomuleasa, Ciprian ; Sacha, Tomasz ; Cirici, Blanca Xicoy ; Bieniaszewska, Maria ; Buxhofer-Ausch, Veronika ; Illés, Arpád ; Al-Ali, Haifa Kathrin ; Nicolini, Franck-Emmanuel ; Crisan, Ana-Manuela ; Guglielmelli, Paola ; Krejcy, Kurt ; Koschmieder, Steffen ; Reiter, Andreas Johannes ; Krauth, Maria-Theresa ; Danaila, Catalin Doru ; Kiladjian, Jean-Jacques ; Unger, Martin ; James, Chloe ; Empson, Victoria ; Heidel, Florian H ; Terpos, Evangelos ; Nagy, Zsolt György ; Beggiato, Eloise ; Wölfler, Albert ; Lazaroiu, Mihaela-Cornelia ; Cerna, Olga ; Breccia, Massimo ; Schlager, Stefanie ; Griesshammer, Martin ; Pappa, Vassiliki ; García-Gutierrez, Valentin ; Gora-Tybor, Joanna ; Grosicki, Sebastian ; Palandri, Francesca ; Döhner, Konstanze ; Marin, Francisca Ferrer ; Schmidt, Stefan

Abstract:

Interferon-based therapies, such as ropeginterferon alfa-2b have emerged as promising disease-modifying agents for myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET). Current ET treatments aim to normalize hematological parameters and reduce the thrombotic risk, but they do not modify the natural history of the disease and hence, have no impact on disease progression. Ropeginterferon alfa-2b (trade name BESREMi®), a novel, monopegylated interferon alfa-2b with an extended administration interval, has demonstrated a robust and sustained efficacy in polycythemia vera (PV) patients. Given the similarities in disease pathophysiology and treatment goals, ropeginterferon alfa-2b holds promise as a treatment option for ET. The ROP-ET trial is a prospective, multicenter, single-arm phase III study that includes patients with ET who are intolerant or resistant to, and/or are ineligible for current therapies, such as hydroxyurea (HU), anagrelide (ANA), busulfan (BUS) and pipobroman, leaving these patients with limited treatment options. The primary endpoint is a composite response of hematologic parameters and disease-related symptoms, according to modified European LeukemiaNet (ELN) criteria. Secondary endpoints include improvements in symptoms and quality of life, molecular response and the safety profile of ropeginterferon alfa-2b. Over a 3-year period the trial assesses longer term outcomes, particularly the effects on allele burden and clinical outcomes, such as disease-related symptoms, vascular events and disease progression. No prospective clinical trial data exist for ropeginterferon alfa-2b in the planned ET study population and this study will provide new findings that may contribute to advancing the treatment landscape for ET patients with limited alternatives.

Trial registration:

EU Clinical Trials Register; EudraCT, 2023-505160-12-00; Registered on October 30, 2023.

01 Jan 2024·Clinics in dermatology

Periocular and ocular surface nonmelanoma skin cancer

Article

Author: Feng, Paula W ; Falcone, Madina ; Feng, Hao ; Shao, Kimberly ; Hooper, Jette

Periocular and ocular surface nonmelanoma malignancies, including basal cell carcinoma (BCC), squamous cell carcinomas (SCC), and ocular surface squamous neoplasia (OSSN), are rare, but their management requires special considerations. The most common periocular malignancy is BCC, which constitutes 80% to 96% of tumors, followed by SCC, which represents 5% to 10% of tumors. OSSN represents a spectrum of diseases that encompass dysplastic alteration to the squamous epithelium of the eye. OSSN ranges from squamous dysplasia to conjunctival intraepithelial neoplasia/carcinoma in situ to invasive SCC, which is the most common ocular malignancy. These tumors can be staged using the eighth edition of the American Joint Committee on Cancer categorization system. The standard of care for periocular malignancies is Mohs micrographic surgery, while medical management with 5-fluorouracil (5-FU), interferon alfa-2b (INF), and mitomycin C (MMC) or "no touch" surgical excision are options for OSSN. Systemic therapies, including sonic hedgehog inhibitors for BCC and epidermal growth factor inhibitors and immune-checkpoint inhibitors for SCC, can be utilized for advanced disease. Recurrence rates are higher for periorbital and ocular malignancies than their respective cutaneous counterparts. These carcinomas and their respective treatments have unique side effects and considerations in an effort to preserve visual function.

News (Medical) associated with INTERFERON ALFA-2B(Merck Sharp & Dohme )

03 Dec 2024

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Ferring Advances Three Studies in ADSTILADRIN® (nadofaragene firadenovec-vncg) Clinical Trial Program

PARSIPPANY, N.J.--( BUSINESS WIRE )--Ferring Pharmaceuticals today announced it has advanced three studies in the ADSTILADRIN ® (nadofaragene firadenovec-vncg) clinical trial program – two studies in patients with non-muscle invasive bladder cancer (NMIBC), and one study in patients with upper tract urothelial cancer (UTUC). The Company also announced researchers will present at the 25 th Annual Meeting of the Society of Urologic Oncology (SUO) the study protocol for patients with intermediate-risk NMIBC and additional data from a Phase 3, open-label study demonstrating clinically meaningful cystectomy-free survival (CFS) after five years among patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC who achieved an initial complete response at three months with ADSTILADRIN. ADSTILADRIN is the first and only intravesical non-replicating gene therapy approved by the U.S. Food and Drug Administration (FDA) in patients with high-risk BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). Study sites in the U.S. were activated for two studies in the ABLE ( A DSTILADRIN in BL adder Canc E r) clinical trial program in NMIBC: The three-arm, Phase 2 ABLE-22 ( NCT06545955 ) trial is assessing the efficacy and safety of ADSTILADRIN as a monotherapy and as part of combination with chemotherapy or an immune checkpoint inhibitor for high-risk BCG-unresponsive NMIBC. Investigators in all three arms of the ABLE-22 study will have the option to re-induce appropriate patients who do not achieve a complete response to the initial single dose or combination regimen, an option that was not included in the original Phase 3 study. The Phase 3B ABLE-32 ( NCT06510374 ) trial is assessing the efficacy and safety of ADSTILADRIN in intermediate-risk NMIBC (IR NMIBC), for which there are no U.S. FDA-approved treatment options. Ferring also initiated the LUNAR ( L ow-Grade U TUC Treated with N adofaragene firadenovec A dministered to R enal Pelvis) study ( NCT06668493 ), a Phase 1-2, multi-center, multi-national, open-label, single-arm, repeat dose clinical trial evaluating the safety, tolerability, and efficacy of ADSTILADRIN instilled to the renal pelvis in patients with low-grade UTUC (LG UTUC). “The availability of an innovative locally delivered gene therapy has the potential to be organ sparing for appropriate NMIBC and UTUC patients. The extension of the ADSTILADRIN clinical trial program – with a focus on IR NMIBC and LG UTUC where there is a desperate need for new treatments, and generation of mono and combination therapy data, including re-induction, for high-risk BCG-unresponsive NMIBC – is key to understanding how ADSTILADRIN may be able to help even more patients,” said Joern Jakobsen, M.D., Ph.D., Vice President and Head of Global Research and Medical for Uro-Oncology and Urology, Ferring Pharmaceuticals. “We are pleased to have these studies underway to build on the evidence reported in the original Phase 3 study. Formalizing the collection of re-induction data in patients who do not achieve a complete response at their first three-month assessment also will add to our pursuit of strategies to potentially offer more patients the opportunity to preserve their bladder and avoid radical surgery.” About ADSTILADRIN Poster Presentations at SUO ADSTILADRIN poster titles and presentation times at the SUO 2024 Annual Meeting, December 4-6, are: Incidence and Pathologic Outcomes of Cystectomy in Patients With Bacillus Calmette-Guérin-Unresponsive Non-Muscle Invasive Bladder Cancer With Carcinoma In Situ Following Treatment With Nadofaragene Firadenovec-vncg, Poster #215, Friday, December 6 from 10:00-11:00 a.m. CST ABLE-32: A Randomized, Controlled, Phase 3B Clinical Trial of Nadofaragene Firadenovec-vncg Versus Observation in Patients With Intermediate-Risk Non–Muscle-Invasive Bladder Cancer, Poster #232, Friday, December 6 from 1:45-2:45 p.m. CST About the ADSTILADRIN Clinical Trial Program Results from the final analysis of the 60-month follow-up data from the pivotal ADSTILADRIN Phase 3 clinical trial were announced earlier this year. Re-induction in patients who did not achieve a complete response at three months after a single initial dose was not included in the protocol. 1 “The original Phase 3 study design was purposefully conservative and only allowed patients who demonstrated a complete response at three months to continue quarterly treatment with ADSTILADRIN as investigators confirmed the overall safety with low risk of progression of gene therapy in this patient population,” said Colin P.N. Dinney, M.D., Chairman of the Department of Urology, Division of Surgery, University of Texas MD Anderson Cancer Center, Houston, and a lead investigator of the Phase 3 study. “The ADSTILADRIN Phase 3 clinical trial transformed our understanding of the treatment of patients with BCG-unresponsive NMIBC and paved the way to allow re-induction in subsequent studies.” Earlier this quarter, Ferring decided to prioritize collection of ADSTILADRIN re-induction data in patients with high-risk BCG-unresponsive NMIBC who did not have a complete response to the initial single dose of ADSTILADRIN through data sources within the clinical setting, including ABLE-22 and ABLE-41 ( NCT06026332 ), and discontinued the previously announced ABLE-42 clinical trial. The ABLE-41 study – currently enrolling patients – is exploring early utilization, experiences, and outcomes of ADSTILADRIN in the routine care setting in high-risk BCG-unresponsive NMIBC patients. “The approval of ADSTILADRIN and its successful launch has transformed the treatment landscape for BCG-unresponsive NMIBC patients, offering them new hope for durable bladder preservation. We hope to see efficacy together with the same low treatment burden and manageable safety profile for ADSTILADRIN in IR NMIBC and as part of combination therapy in BCG-unresponsive NMIBC,” said Bipin Dalmia, Global Head of Uro-Oncology & Urology Franchise, Ferring Pharmaceuticals. “Expanding our program within NMIBC and also to UTUC with the LUNAR study is another step forward in our leadership journey to establish ADSTILADRIN as the new standard of care and the backbone therapy for patients across the urothelial cancer disease spectrum.” About the ABLE and LUNAR Clinical Trials ABLE-22 is a Phase 2, randomized, multi-center, open-label three-arm trial evaluating the efficacy and safety of ADSTILADRIN as a monotherapy or in combination with chemotherapy (gemcitabine and docetaxel) or an immune checkpoint inhibitor (pembrolizumab) in adult patients with high-grade BCG-unresponsive NMIBC. High-grade tumors are more likely to grow and spread quickly. The study’s primary endpoint is a complete response (defined as absence of low- and high-grade disease) at month 3 (or month 6 for re-induced patients). Appropriate patients in all three treatment arms of ABLE-22 who do not have a complete response to the first three-month assessment will have the potential to receive re-induction with a second quarterly dose of ADSTILADRIN. ABLE-32 is a Phase 3B, randomized, controlled trial assessing the potential efficacy and safety of ADSTILADRIN in patients with IR NMIBC. More than 450 participants from 100 global sites are expected to be enrolled and will either receive quarterly doses of ADSTILADRIN or undergo continued observation following transurethral resection of bladder tumor (TURBT) within 60 days prior to randomization. The primary efficacy endpoint is recurrence-free survival (RFS), from baseline to first documented recurrence, progression, or death, whichever occurs first during the treatment period. ABLE-41 is an ongoing, multi-center, non-interventional, real-world evidence (RWE) study following patients with NMIBC aged 18 years or older who are being treated with ADSTILADRIN in a clinical setting and had not previously received this therapy in a clinical trial. The study is exploring early utilization, experiences, and outcomes of ADSTILADRIN in the routine care setting, including re-induction with a second quarterly dose in patients without a complete response to the initial single dose. The first patient was enrolled in September 2023. LUNAR is a Phase 1-2 single-arm, open label trial evaluating the safety, tolerability, and efficacy of ADSTILADRIN in patients with low-grade UTUC. A safety lead-in period will be conducted for the first six patients. Absence of UTUC in the renal pelvis at months 3 or 6 will be defined as a complete response. Duration of response up to month 30 will be defined as the time from first achieved complete response to disease recurrence, progression to high-grade disease or disease-specific death, whichever occurs first. About ADSTILADRIN ADSTILADRIN ® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer. ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849 ). 2 Ferring is leading the future in uro-oncology treatment with ADSTILADRIN at the center, while expanding access with the support of new, state-of-the-art manufacturing facilities. As announced in January 2024, ADSTILADRIN is fully available and accessible in the U.S. ADSTILADRIN has confirmed 99 percent coverage for commercial and government-insured patients. As of April 1, 2024, in accordance with the Centers for Medicare and Medicaid Services (CMS), ADSTILADRIN established an Average Sales Price (ASP). Since the establishment of ASP, all covered claims submitted for reimbursement have received payment within an average of 25 days. 3 Ferring is committed to investing in novel therapies, developing life-changing solutions that address unmet medical needs, and aiding the uro-oncology community in helping patients live better lives. More information is available in the U.S. at the dedicated Ferring Uro-Oncology channel on LinkedIn . About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall. 4 In the United States, bladder cancer is the sixth most common cancer, 5 fourth among men, 6 and it is estimated that there will be approximately 83,190 new cases of bladder cancer in the U.S. in 2024. 6 Historically, 75% of bladder cancer presents as NMIBC. 7 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease. 8 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder). 9 About Low-Grade Upper Tract Urothelial Cancer (UTUC) UTUC accounts for about 10 percent of all urothelial cancers - cancer of the lining within the kidneys, bladder, or ureters – with the majority occurring in the lower tract or bladder. 10 Clinicians use several standardized assessment and clinical staging methods to risk-stratify patients into “low” or “high” risk for progression to identify patients who are most likely to benefit from kidney-sparing treatment. 10 Tumors that block the ureter or kidney can cause hydronephrosis (swelling of the kidney), infections, and impairment of kidney function. 10 Low-grade tumors comprise all low-risk and some high-risk tumors in UTUC with management goals including treatment of visible tumors and preservation of the urinary tract. 10 There is an estimated total incidence of just over 7,000 new UTUC cases each year in the U.S., 10 and the prevalence appears to be increasing. 11 INDICATION ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product. WARNINGS AND PRECAUTIONS: Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy. Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals. DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration. USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose. ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination). You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING. Please click to see the full Prescribing Information . About Ferring Pharmaceuticals Ferring Pharmaceuticals is a privately-owned, research-driven, specialty biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive medicine and maternal health, and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. Our company was founded in 1950 and is headquartered in Saint-Prex, Switzerland. Ferring employs more than 7,000 people worldwide and markets its medicines in over 100 countries. Ferring USA is based in Parsippany, New Jersey, and employs more than 900 employees. For more information, please visit www.ferringusa.com , call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn , and X (Twitter) . References: Narayan VM, Boorjian SA, Alemozaffar M, et al. Efficacy of Intravesical Nadofaragene Firadenovec for Patients with Bacillus Calmette-Guerin-unresponsive Non-muscle-invasive Bladder Cancer: 5-year Follow-up from a Phase 3 Trial. J Urol. 2024;212(1):74-86. doi:10.1097/JU.0000000000004020. ADSTILADRIN in Patients With High Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. Available at: https://clinicaltrials.gov/ct2/show/NCT02773849 . Accessed November 5, 2024. Ferring Access Support benefits investigations for commercial and government-insured patients through March 15, 2024. Urology Care Foundation. Non-muscle Invasive Bladder Cancer: Symptoms, Diagnosis & Treatment. Available at: https://www.urologyhealth.org/urology-a-z/n/non-muscle-invasive-bladder-cancer . Accessed November 5, 2024. National Cancer Institute. Cancer Statistics. Available at: https://www.cancer.gov/about-cancer/understanding/statistics#:~:text=The%20most%20common%20cancers%20%28listed%20in%20descending%20order,pancreatic%20cancer%2C%20leukemia%2C%20thyroid%20cancer%2C%20and%20liver%20cancer . Accessed November 5, 2024. American Cancer Society. Key Statistics for Bladder Cancer. Available at: https://www.cancer.org/cancer/types/bladder-cancer/about/key-statistics.html . Accessed November 5, 2024. Babjuk M, Burger M, Capoun O, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer. Eur Urol. 2022 Jan;81(1):75-94. Lidagoster S, et al. BCG and Alternative Therapies to BCG Therapy for Non-Muscle-Invasive Bladder Cancer. Curr Oncol. 2024 Feb 16;31(2):1063-1078. doi: 10.3390/curroncol31020079. National Comprehensive Cancer Network. Bladder Cancer (Version 4.2024). Available at: https://www.nccn.org/professionals/physician_gls/pdf/bladder.pdf . Accessed November 5, 2024. Coleman JA, Clark PE, Bixler BR, et al. Diagnosis and Management of Non-metastatic Upper Tract Urothelial Carcinoma: AUA/SUO Guideline. J Urol. 2023;209(6):1071-1081. Raman, J and Shore, ND. Management of Low-grade Upper Tract Urothelial Carcinoma: An Unmet Need. Rev Urol. 2020;22(1):1–8.

Phase 3Phase 2Drug ApprovalGene TherapyClinical Result

25 Nov 2024

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PharmaEssentia Announces Nine Data Presentations at ASH Annual Meeting

BURLINGTON, Mass.--( BUSINESS WIRE )--PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced a slate of nine new abstracts exploring the use of ropeginterferon alfa-2b-njft, known in market as BESREMi ® , will be presented during the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, CA. Presentations at ASH include a quality-of-life evaluation for patients with polycythemia vera (PV) or essential thrombocythemia (ET) and treated with ropeginterferon alfa-2b compared to other best available therapies; identifying the efficacy and safety of ropeginterferon alfa-2b in low-risk PV patients and the potential of an AI-powered approach to discovering potential new indications for ropeginterferon alfa-2b. “With new clinical findings, AI-powered results and real-world analyses, we are excited to continue to advance our knowledge on the potential of ropeginterferon alfa-2b in treating chronic and life-threatening myeloproliferative neoplasms,” said Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia. “PharmaEssentia maintains its commitment to educating providers and patients to make informed decisions to improve care and outcomes for people living with myeloproliferative neoplasms such as PV and ET.” Ropeginterferon Alfa-2b presentations at ASH include: Ropeginterferon Alfa-2b Induces Apoptosis and Differentiation of Leukemia Stem Cells and Separates GVL Effects from GVHD after Allogeneic Hematopoietic Cell Transplantation Oral Presentation Abstract #902: Monday, December 9, 2024, 2:45-4:15 PM PT Quality of Life Evaluation for Patients on Ropeginterferon Alpha-2b Versus Other Best Available Therapies Poster Presentation Abstract #1791: Saturday, December 7, 2024, 5:30-7:30 PM PT Ropeginterferon in Low-risk Patients with Polycythemia Vera Poster Presentation Abstract #1799: Saturday, December 7, 2024, 5:30-7:30 PM PT HOPE-PMF: A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b in Patients with Early/Lower-Risk Primary Myelofibrosis Poster Presentation Abstract #3191.1: Sunday, December 8, 2024, 6:00-8:00 PM PT Predicting Molecular Response through Half Reduction of Neutrophil-to-Lymphocyte ratio (NLR) in Polycythemia Vera Treatment with Ropeginterferon Poster Presentation Abstract #3170: Sunday, December 8, 2024, 6:00-8:00 PM PT Remarkable molecular response in Chinese PV patients treated with Ropeginterferon alfa-2b Poster Presentation Abstract #4562: Monday, December 9, 2024, 6:00-8:00 PM PT Ropeginterferon Alfa-2b (ROPEG) and Peginterferon Alfa-2a (PEG) at Low Dose with Response-Based Titration (LDRT) Have Comparable Efficacy and Tolerability in Polycythemia Vera (PV) Poster Presentation Abstract #4568: Monday, December 9, 2024, 6:00-8:00 PM PT Accelerating Drug Discovery: AI-Powered Indication Exploration for Ropeginterferon Alfa-2b Poster Presentation Abstract #4982: Monday, December 9, 2024, 6:00-8:00 PM PT Treatment Patterns of Ropeginterferon Alfa-2b-njft in the real-world setting Poster Presentation Abstract #5184: Monday, December 9, 2024, 6:00-8:00 PM PT About PharmaEssentia PharmaEssentia (TWSE: 6446), headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website , LinkedIn or Twitter . About Polycythemia Vera (PV) Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2. 1 About Essential Thrombocythemia (ET) Essential thrombocythemia (ET) is a myeloproliferative neoplasm (MPN) characterized by an overproduction of platelets in the blood that results from a genetic mutation; data indicates a JAK2 gene mutation is present in approximately half of diagnosed patients. ET is estimated to affect up to 57 per 100,000 people in the U.S. The disease is most commonly diagnosed through routine blood work and is most common in people over the age of 50, with women 1.5 more times more likely to be diagnosed than men. As a chronic, progressive disease, ET requires regular monitoring and appropriate treatment. Over time, the disease may progress into more deadly conditions such as myelofibrosis or acute leukemia. 2,3 About BESREMi ® (ropeginterferon alfa-2b-njft) BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients. BESREMi has orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. BESREMi has been approved in more than 40 countries, with approval from the European Medicines Agency (EMA) in 2019, by the US Food and Drug Administration (FDA) in 2021, and by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023. It was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders. Please see full Prescribing Information , including Boxed Warning. Indication BESREMi is indicated for the treatment of adults with polycythemia vera. Important Safety Information WARNING: RISK OF SERIOUS DISORDERS Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. In many, but not all cases, these disorders resolve after stopping therapy. CONTRAINDICATIONS Existence of, or history of severe psychiatric disorders, particularly severe depression, suicidal ideation, or suicide attempt Hypersensitivity to interferons including interferon alfa-2b or any of the inactive ingredients of BESREMi. Moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic impairment History or presence of active serious or untreated autoimmune disease Immunosuppressed transplant recipients WARNINGS AND PRECAUTIONS Depression and Suicide: Life-threatening or fatal neuropsychiatric reactions have occurred in patients receiving interferon alfa-2b products, including BESREMi. These reactions may occur in patients with and without previous psychiatric illness. Other central nervous system effects, including suicidal ideation, attempted suicide, aggression, bipolar disorder, mania and confusion have been observed with other interferon alfa products. Closely monitor patients for any symptoms of psychiatric disorders and consider psychiatric consultation and treatment if such symptoms emerge. If psychiatric symptoms worsen, it is recommended to discontinue BESREMi therapy. Endocrine Toxicity: These toxicities may include worsening hypothyroidism and hyperthyroidism. Do not use BESREMi in patients with active serious or untreated endocrine disorders associated with autoimmune disease. Evaluate thyroid function in patients who develop symptoms suggestive of thyroid disease during BESREMi therapy. Discontinue BESREMi in patients who develop endocrine disorders that cannot be adequately managed during treatment with BESREMi. Cardiovascular Toxicity: Toxicities may include cardiomyopathy, myocardial infarction, atrial fibrillation and coronary artery ischemia. Patients with a history of cardiovascular disorders should be closely monitored for cardiovascular toxicity during BESREMi therapy. Avoid use of BESREMi in patients with severe or unstable cardiovascular disease, (e.g., uncontrolled hypertension, congestive heart failure (≥ NYHA class 2), serious cardiac arrhythmia, significant coronary artery stenosis, unstable angina) or recent stroke or myocardial infarction. Decreased Peripheral Blood Counts: These toxicities may include thrombocytopenia (increasing the risk of bleeding), anemia, and leukopenia (increasing the risk of infection). Monitor complete blood counts at baseline, during titration and every 3-6 months during the maintenance phase. Monitor patients for signs and symptoms of infection or bleeding. Hypersensitivity Reactions: Toxicities may include serious, acute hypersensitivity reactions (e.g., urticaria, angioedema, bronchoconstriction, anaphylaxis). If such reactions occur, discontinue BESREMi and institute appropriate medical therapy immediately. Transient rashes may not necessitate interruption of treatment. Pancreatitis: Pancreatitis has occurred in 2.2% of patients receiving BESREMi. Symptoms may include nausea, vomiting, upper abdominal pain, bloating, and fever. Patients may experience elevated lipase, amylase, white blood cell count, or altered renal/hepatic function. Interrupt BESREMi treatment in patients with possible pancreatitis and evaluate promptly. Consider discontinuation of BESREMi in patients with confirmed pancreatitis. Colitis: Fatal and serious ulcerative or hemorrhagic/ischemic colitis have occurred in patients receiving interferon alfa products, some cases starting as early as 12 weeks after start of treatment. Symptoms may include abdominal pain, bloody diarrhea, and fever. Discontinue BESREMi in patients who develop these signs or symptoms. Colitis may resolve within 1 to 3 weeks of stopping treatment. Pulmonary Toxicity: Pulmonary toxicity may manifest as dyspnea, pulmonary infiltrates, pneumonia, bronchiolitis obliterans, interstitial pneumonitis, pulmonary hypertension, and sarcoidosis. Some events have resulted in respiratory failure or death. Discontinue BESREMi in patients who develop pulmonary infiltrates or pulmonary function impairment. Ophthalmologic Toxicity: These toxicities may include severe eye disorders such as retinopathy, retinal hemorrhage, retinal exudates, retinal detachment and retinal artery or vein occlusion which may result in blindness. During BESREMi therapy, 23% of patients were identified with an eye disorder. Eyes disorders ≥5% included cataract (6%) and dry eye (5%). Advise patients to have eye examinations before and during BESREMi therapy, specifically in those patients with a retinopathy-associated disease such as diabetes mellitus or hypertension. Evaluate eye symptoms promptly. Discontinue BESREMi in patients who develop new or worsening eye disorders. Hyperlipidemia: Elevated triglycerides may result in pancreatitis. Monitor serum triglycerides before BESREMi treatment and intermittently during therapy and manage when elevated. Consider discontinuation of BESREMi in patients with persistently, markedly elevated triglycerides. Hepatotoxicity: These toxicities may include increases in serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT) and bilirubin. Liver enzyme elevations have also been reported in patients after long-term BESREMi therapy. Monitor liver enzymes and hepatic function at baseline and during BESREMi treatment. Discontinue BESREMi in patients who develop evidence of hepatic decompensation (characterized by jaundice, ascites, hepatic encephalopathy, hepatorenal syndrome or variceal hemorrhage) during treatment. Renal Toxicity: Monitor serum creatinine at baseline and during therapy. Avoid use of BESREMi in patients with eGFR <30 mL/min. Discontinue BESREMi if severe renal impairment develops during treatment. Dental and Periodontal Toxicity: These toxicities may include dental and periodontal disorders, which may lead to loss of teeth. In addition, dry mouth could have a damaging effect on teeth and mucous membranes of the mouth during long-term treatment with BESREMi. Patients should have good oral hygiene and regular dental examinations. Dermatologic Toxicity: These toxicities have included skin rash, pruritus, alopecia, erythema, psoriasis, xeroderma, dermatitis acneiform, hyperkeratosis, and hyperhidrosis. Consider discontinuation of BESREMi if clinically significant dermatologic toxicity occurs. Driving and Operating Machinery: BESREMi may impact the ability to drive and use machinery. Patients should not drive or use heavy machinery until they know how BESREMi affects their abilities. Patients who experience dizziness, somnolence or hallucination during BESREMi therapy should avoid driving or using machinery. Embryo-Fetal Toxicity: Based on the mechanism of action, BESREMi can cause fetal harm when administered to a pregnant woman. Pregnancy testing is recommended in females of reproductive potential prior to treatment with BESREMi. Advise females of reproductive potential to use an effective method of contraception during treatment with BESREMi and for at least 8 weeks after the final dose. ADVERSE REACTIONS The most common adverse reactions reported in > 40% of patients in the PEGINVERA study (n=51) were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. In the pooled safety population (n=178), the most common adverse reactions greater than 10%, were liver enzyme elevations (20%), leukopenia (20%), thrombocytopenia (19%), arthralgia (13%), fatigue (12%), myalgia (11%), and influenza-like illness (11%). DRUG INTERACTIONS Patients on BESREMi who are receiving concomitant drugs which are CYP450 substrates with a narrow therapeutic index should be monitored to inform the need for dosage modification for these concomitant drugs. Avoid use with myelosuppressive agents and monitor patients receiving the combination for effects of excessive myelosuppression. Avoid use with narcotics, hypnotics or sedatives and monitor patients receiving the combination for effects of excessive CNS toxicity. USE IN SPECIFIC POPULATIONS Pregnancy: Based on mechanism of action and the role of interferon alfa in pregnancy and fetal development, BESREMi may cause fetal harm and should be assumed to have abortifacient potential when administered to a pregnant woman. There are adverse effects on maternal and fetal outcomes associated with polycythemia vera in pregnancy. Advise pregnant women of the potential risk to a fetus. Lactation: There are no data on the presence of BESREMi in human or animal milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed children from BESREMi, advise women not to breastfeed during treatment and for 8 weeks after the final dose. Females of Reproductive Potential: BESREMi may cause embryo-fetal harm when administered to a pregnant woman. Pregnancy testing prior to BESREMi treatment is recommended for females of reproductive potential. Advise female patients of reproductive potential to use effective contraception during treatment with BESREMi and for at least 8 weeks after the final dose. Pediatric Use: Safety and effectiveness in pediatric patients have not been established. Geriatric Use: In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function and of concomitant disease or other therapy. Forward Looking Statement This press release may contain forward looking statements, including statements regarding the clinical benefits to be derived from ropeginterferon alfa-2b, the commercial opportunity and competitive positioning, new indications or labeling for ropeginterferon alfa-2b, and business prospects for ropeginterferon alfa-2b. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and similar legislation and regulations under Taiwanese law. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward looking statements as a result of various factors. These factors include whether BESREMi is successfully commercialized and adopted by physicians and patients, the extent to which reimbursement is available for BESREMi, and the ability to receive FDA and other regulatory approvals for additional indications for BESREMi. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. © 2024 PharmaEssentia Corporation. All rights reserved. PharmaEssentia, the PharmaEssentia logo, and BESREMi are trademarks or registered trademarks of PharmaEssentia Corporation. 1 Cerquozzi S, Tefferi A. Blast transformation and fibrotic progression in polycythemia vera and essential thrombocythemia: a literature review of incidence and risk factors. Blood Cancer J . 2015;5, e366; DOI:10.1038/bcj.2015.95 2 Mehta J, Wang H, Iqbal SU, Mesa R. Epidemiology of myeloproliferative neoplasms in the United States. Leuk Lymphoma. 2014 Mar;55(3):595-600 3 “What is Essential Thrombocythemia?” MPN Research Foundation. 2020. Available at: http://www.mpnresearchfoundation.org/Essential-Thrombocythemia

Drug ApprovalPhase 3ASHClinical ResultOrphan Drug

04 Sep 2024

·www.businesswire.com

PharmaEssentia Announces Licensing Agreement with FORUS Therapeutics to Commercialize BESREMi® (ropeginterferon alfa-2b-njft) in Canada

BURLINGTON, Mass.--( BUSINESS WIRE )--PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that it has entered into an exclusive licensing agreement with FORUS Therapeutics Inc (“FORUS”) for the registration and distribution of BESREMi ® (ropeginterferon alfa-2b-njft) for the treatment of polycythemia vera (PV), in Canada. FORUS focuses on the hematology and oncology market in Canada, with extensive experience in local drug registration and commercialization. Under the terms of the agreement, PharmaEssentia is licensing BESREMi for PV to FORUS in Canada, with potential expansion to other investigational myeloproliferative neoplasms (MPN) indications in the future. FORUS will oversee the drug registration and commercialization of BESREMi in Canada, with potential milestone payments of up to $107 million USD – including for securing approval of BESREMi in PV and meeting sales milestones. FORUS will make a portion of the milestone payments within 2024 and pay PharmaEssentia double-digit percentage royalties on sales. “FORUS and PharmaEssentia are aligned in our missions to bring new treatments to patients with hematologic and oncologic conditions that may meaningfully enhance their lives,” said Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia. “We continue to expand scientific data to demonstrate the clinical value of BESREMi for patients with MPNs such as PV, and we look forward to collaborating with FORUS to introduce BESREMi in Canada, to expand our footprint in North America and to benefit even more patients with PV.” “Strategic partnerships pave the way to unlocking innovation and advancing healthcare solutions,” said Kevin Leshuk, President and CEO of FORUS. “We are extremely pleased and honored to be partnering with PharmaEssentia so that we can deliver potentially transformative outcomes for those living with PV and hopefully other hematologic conditions in the future.” BESREMi has been approved for the treatment of PV in approximately 40 countries and regions, including the United States, Japan, China, and the European Union. About PharmaEssentia PharmaEssentia (TWSE: 6446), headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website , LinkedIn or Twitter . About FORUS Therapeutics FORUS Therapeutics is a Canadian biopharmaceutical company dedicated to advancing differentiated, novel medicines for hematologic malignancies and other forms of cancer. Our mission is to bring solutions to cancer patients, caregivers, physicians and our partners by accelerating unique and important treatments that meaningfully enhance life. www.forustherapeutics.com About Polycythemia Vera (PV) Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2. 1 About BESREMi ® (ropeginterferon alfa-2b-njft) BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients. BESREMi has orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. BESREMi has been approved in more than 40 countries, with approval from the European Medicines Agency (EMA) in 2019, by the US Food and Drug Administration (FDA) in 2021, and by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023. It was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders. Please see full Prescribing Information , including Boxed Warning. Indication BESREMi is indicated for the treatment of adults with polycythemia vera. Important Safety Information WARNING: RISK OF SERIOUS DISORDERS Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. In many, but not all cases, these disorders resolve after stopping therapy. CONTRAINDICATIONS Existence of, or history of severe psychiatric disorders, particularly severe depression, suicidal ideation, or suicide attempt Hypersensitivity to interferons including interferon alfa-2b or any of the inactive ingredients of BESREMi. Moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic impairment History or presence of active serious or untreated autoimmune disease Immunosuppressed transplant recipients WARNINGS AND PRECAUTIONS Depression and Suicide: Life-threatening or fatal neuropsychiatric reactions have occurred in patients receiving interferon alfa-2b products, including BESREMi. These reactions may occur in patients with and without previous psychiatric illness. Other central nervous system effects, including suicidal ideation, attempted suicide, aggression, bipolar disorder, mania and confusion have been observed with other interferon alfa products. Closely monitor patients for any symptoms of psychiatric disorders and consider psychiatric consultation and treatment if such symptoms emerge. If psychiatric symptoms worsen, it is recommended to discontinue BESREMi therapy. Endocrine Toxicity: These toxicities may include worsening hypothyroidism and hyperthyroidism. Do not use BESREMi in patients with active serious or untreated endocrine disorders associated with autoimmune disease. Evaluate thyroid function in patients who develop symptoms suggestive of thyroid disease during BESREMi therapy. Discontinue BESREMi in patients who develop endocrine disorders that cannot be adequately managed during treatment with BESREMi. Cardiovascular Toxicity: Toxicities may include cardiomyopathy, myocardial infarction, atrial fibrillation and coronary artery ischemia. Patients with a history of cardiovascular disorders should be closely monitored for cardiovascular toxicity during BESREMi therapy. Avoid use of BESREMi in patients with severe or unstable cardiovascular disease, (e.g., uncontrolled hypertension, congestive heart failure (≥ NYHA class 2), serious cardiac arrhythmia, significant coronary artery stenosis, unstable angina) or recent stroke or myocardial infarction. Decreased Peripheral Blood Counts: These toxicities may include thrombocytopenia (increasing the risk of bleeding), anemia, and leukopenia (increasing the risk of infection). Monitor complete blood counts at baseline, during titration and every 3-6 months during the maintenance phase. Monitor patients for signs and symptoms of infection or bleeding. Hypersensitivity Reactions: Toxicities may include serious, acute hypersensitivity reactions (e.g., urticaria, angioedema, bronchoconstriction, anaphylaxis). If such reactions occur, discontinue BESREMi and institute appropriate medical therapy immediately. Transient rashes may not necessitate interruption of treatment. Pancreatitis: Pancreatitis has occurred in 2.2% of patients receiving BESREMi. Symptoms may include nausea, vomiting, upper abdominal pain, bloating, and fever. Patients may experience elevated lipase, amylase, white blood cell count, or altered renal/hepatic function. Interrupt BESREMi treatment in patients with possible pancreatitis and evaluate promptly. Consider discontinuation of BESREMi in patients with confirmed pancreatitis. Colitis: Fatal and serious ulcerative or hemorrhagic/ischemic colitis have occurred in patients receiving interferon alfa products, some cases starting as early as 12 weeks after start of treatment. Symptoms may include abdominal pain, bloody diarrhea, and fever. Discontinue BESREMi in patients who develop these signs or symptoms. Colitis may resolve within 1 to 3 weeks of stopping treatment. Pulmonary Toxicity: Pulmonary toxicity may manifest as dyspnea, pulmonary infiltrates, pneumonia, bronchiolitis obliterans, interstitial pneumonitis, pulmonary hypertension, and sarcoidosis. Some events have resulted in respiratory failure or death. Discontinue BESREMi in patients who develop pulmonary infiltrates or pulmonary function impairment. Ophthalmologic Toxicity: These toxicities may include severe eye disorders such as retinopathy, retinal hemorrhage, retinal exudates, retinal detachment and retinal artery or vein occlusion which may result in blindness. During BESREMi therapy, 23% of patients were identified with an eye disorder. Eyes disorders ≥5% included cataract (6%) and dry eye (5%). Advise patients to have eye examinations before and during BESREMi therapy, specifically in those patients with a retinopathy-associated disease such as diabetes mellitus or hypertension. Evaluate eye symptoms promptly. Discontinue BESREMi in patients who develop new or worsening eye disorders. Hyperlipidemia: Elevated triglycerides may result in pancreatitis. Monitor serum triglycerides before BESREMi treatment and intermittently during therapy and manage when elevated. Consider discontinuation of BESREMi in patients with persistently, markedly elevated triglycerides. Hepatotoxicity: These toxicities may include increases in serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT) and bilirubin. Liver enzyme elevations have also been reported in patients after long-term BESREMi therapy. Monitor liver enzymes and hepatic function at baseline and during BESREMi treatment. Discontinue BESREMi in patients who develop evidence of hepatic decompensation (characterized by jaundice, ascites, hepatic encephalopathy, hepatorenal syndrome or variceal hemorrhage) during treatment. Renal Toxicity: Monitor serum creatinine at baseline and during therapy. Avoid use of BESREMi in patients with eGFR <30 mL/min. Discontinue BESREMi if severe renal impairment develops during treatment. Dental and Periodontal Toxicity: These toxicities may include dental and periodontal disorders, which may lead to loss of teeth. In addition, dry mouth could have a damaging effect on teeth and mucous membranes of the mouth during long-term treatment with BESREMi. Patients should have good oral hygiene and regular dental examinations. Dermatologic Toxicity: These toxicities have included skin rash, pruritus, alopecia, erythema, psoriasis, xeroderma, dermatitis acneiform, hyperkeratosis, and hyperhidrosis. Consider discontinuation of BESREMi if clinically significant dermatologic toxicity occurs. Driving and Operating Machinery: BESREMi may impact the ability to drive and use machinery. Patients should not drive or use heavy machinery until they know how BESREMi affects their abilities. Patients who experience dizziness, somnolence or hallucination during BESREMi therapy should avoid driving or using machinery. Embryo-Fetal Toxicity: Based on the mechanism of action, BESREMi can cause fetal harm when administered to a pregnant woman. Pregnancy testing is recommended in females of reproductive potential prior to treatment with BESREMi. Advise females of reproductive potential to use an effective method of contraception during treatment with BESREMi and for at least 8 weeks after the final dose. ADVERSE REACTIONS The most common adverse reactions reported in > 40% of patients in the PEGINVERA study (n=51) were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. In the pooled safety population (n=178), the most common adverse reactions greater than 10%, were liver enzyme elevations (20%), leukopenia (20%), thrombocytopenia (19%), arthralgia (13%), fatigue (12%), myalgia (11%), and influenza-like illness (11%). DRUG INTERACTIONS Patients on BESREMi who are receiving concomitant drugs which are CYP450 substrates with a narrow therapeutic index should be monitored to inform the need for dosage modification for these concomitant drugs. Avoid use with myelosuppressive agents and monitor patients receiving the combination for effects of excessive myelosuppression. Avoid use with narcotics, hypnotics or sedatives and monitor patients receiving the combination for effects of excessive CNS toxicity. USE IN SPECIFIC POPULATIONS Pregnancy: Based on mechanism of action and the role of interferon alfa in pregnancy and fetal development, BESREMi may cause fetal harm and should be assumed to have abortifacient potential when administered to a pregnant woman. There are adverse effects on maternal and fetal outcomes associated with polycythemia vera in pregnancy. Advise pregnant women of the potential risk to a fetus. Lactation: There are no data on the presence of BESREMi in human or animal milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed children from BESREMi, advise women not to breastfeed during treatment and for 8 weeks after the final dose. Females of Reproductive Potential: BESREMi may cause embryo-fetal harm when administered to a pregnant woman. Pregnancy testing prior to BESREMi treatment is recommended for females of reproductive potential. Advise female patients of reproductive potential to use effective contraception during treatment with BESREMi and for at least 8 weeks after the final dose. Pediatric Use: Safety and effectiveness in pediatric patients have not been established. Geriatric Use: In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function and of concomitant disease or other therapy. Forward Looking Statement This press release may contain forward looking statements, including statements regarding the clinical benefits to be derived from ropeginterferon alfa-2b, the commercial opportunity and competitive positioning, new indications or labeling for ropeginterferon alfa-2b, and business prospects for ropeginterferon alfa-2b. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and similar legislation and regulations under Taiwanese law. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward looking statements as a result of various factors. These factors include whether BESREMi is successfully commercialized and adopted by physicians and patients, the extent to which reimbursement is available for BESREMi, and the ability to receive FDA and other regulatory approvals for additional indications for BESREMi. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. © 2024 PharmaEssentia Corporation. All rights reserved. PharmaEssentia, the PharmaEssentia logo, and BESREMi are trademarks or registered trademarks of PharmaEssentia Corporation. 1 Cerquozzi S, Tefferi A. Blast transformation and fibrotic progression in polycythemia vera and essential thrombocythemia: a literature review of incidence and risk factors. Blood Cancer J . 2015;5, e366; DOI:10.1038/bcj.2015.95

License out/inDrug ApprovalOrphan Drug

100 Deals associated with INTERFERON ALFA-2B(Merck Sharp & Dohme )

External Link

KEGG	Wiki	ATC	Drug Bank
D02745	INTERFERON ALFA-2B(Merck Sharp & Dohme )	L03AB05	DB00105

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Carcinoid Tumor	EU	Merck Sharp & Dohme BV	09 Mar 2000
Carcinoid Tumor	IS	Merck Sharp & Dohme BV	09 Mar 2000
Carcinoid Tumor	LI	Merck Sharp & Dohme BV	09 Mar 2000
Carcinoid Tumor	NO	Merck Sharp & Dohme BV	09 Mar 2000
Multiple Myeloma	EU	Merck Sharp & Dohme BV	09 Mar 2000
Multiple Myeloma	IS	Merck Sharp & Dohme BV	09 Mar 2000
Multiple Myeloma	LI	Merck Sharp & Dohme BV	09 Mar 2000
Multiple Myeloma	NO	Merck Sharp & Dohme BV	09 Mar 2000
Philadelphia chromosome positive chronic myelogenous leukemia	EU	Merck Sharp & Dohme BV	09 Mar 2000
Philadelphia chromosome positive chronic myelogenous leukemia	IS	Merck Sharp & Dohme BV	09 Mar 2000
Philadelphia chromosome positive chronic myelogenous leukemia	LI	Merck Sharp & Dohme BV	09 Mar 2000
Philadelphia chromosome positive chronic myelogenous leukemia	NO	Merck Sharp & Dohme BV	09 Mar 2000
Hepatitis C	CN	Schering-Plough Corp.	27 Oct 1999
Kaposi Sarcoma	CN	Schering-Plough Corp.	27 Oct 1999
AIDS-related Kaposi Sarcoma	US	Merck Sharp & Dohme LLC	04 Jun 1986
Condylomata Acuminata	US	Merck Sharp & Dohme LLC	04 Jun 1986
Follicular Lymphoma	US	Merck Sharp & Dohme LLC	04 Jun 1986
Hairy Cell Leukemia	US	Merck Sharp & Dohme LLC	04 Jun 1986
Hepatitis B, Chronic	US	Merck Sharp & Dohme LLC	04 Jun 1986
Hepatitis C, Chronic	US	Merck Sharp & Dohme LLC	04 Jun 1986

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Melanoma, Cutaneous Malignant	Phase 3	-	Merck Sharp & Dohme LLC	05 Aug 1998
Acquired Immunodeficiency Syndrome	Phase 1	US	Schering-Plough Corp.	31 Aug 2001

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


APAO2024	Not Applicable	Malignant melanoma of conjunctiva Adjuvant	-	Interferon alpha-2b (IFN-α2b) eye drops	ojzabkaozp(aszigolwzd) = yykmblatxm gcmimgtlnx (uongmxeezx ) View more	Positive	22 Feb 2024
APAO2024	Not Applicable	Malignant melanoma of conjunctiva Adjuvant	-	Mitomycin C (MMC) eye drops	ojzabkaozp(aszigolwzd) = wlwrzcyrhb gcmimgtlnx (uongmxeezx ) View more	Positive	22 Feb 2024
APAO2023	Not Applicable	Neoplasms	-	Recombinant Interferon alpha 2b (IFNÎ±2b) 3 million international units (IU) in 0.5 mL	zbrnfnswjz(vzyauagaoe) = post- injection flu like illness for 2 days fdzxanlejk (wsowoyuogy )	-	23 Feb 2023
NCT03403634 (CTgov)	Phase 2	Liver metastases \| Colorectal cancer recurrent \| Colorectal Cancer	19	Laboratory Biomarker Analysis+Intron A+Heberon Alfa+Urifron+Rintatolimod+Celecoxib	wzzpzlufow(lwfhkdblku) = rnahlrujer urztujhmvc (ooenpsjcwc, ovwltkkfjf - nmqhqjffnc) View more	-	02 Mar 2022
Pubmed \| Melanoma Res	Not Applicable	Melanoma \| Retinal Diseases	56	interferon α-2b (Melanoma patients with HD-IFN-induced retinopathy)	libbtuzcir(btdrdxqedk) = sxlwgtjnja ayyvhpwtyg (uukvvfxbve ) View more	-	01 Dec 2021
NCT03552549 (MK-4031-002 \| C98-135, CTgov)	Phase 2/3	Melanoma, Cutaneous Malignant \| Melanoma	126	PEG-Intron (PEG-Intron)	rriykljqgh(bykcajhvco) = xeeadkzvin wiqroxjtzm (czcvlsbkpi, xkjiuzweep - mxcxfwvmzw) View more	-	24 Jul 2019
NCT03552549 (MK-4031-002 \| C98-135, CTgov)	Phase 2/3	Melanoma, Cutaneous Malignant \| Melanoma	126	Intron A (INTRON A)	rriykljqgh(bykcajhvco) = cwwnkhdgni wiqroxjtzm (czcvlsbkpi, rjsvqjpkbk - ypawqyzljr) View more	-	24 Jul 2019
NCT01460875 (CTgov)	Not Applicable	Melanoma, Cutaneous Malignant	34	laboratory biomarker analysis+Intron A+Heberon Alfa	dthmqyabwo(oxbeyfsyzy) = ckunejccxa hwjixtvfmv (xdxtahikcz, rqmvtvygat - irtfzqggmh) View more	-	02 Nov 2018
NCT01100528 (CTgov)	Phase 2	Uveal Melanoma	38	Recombinant Interferon Alfa-2b+Dacarbazine	agcmkcrnzc(ngdgrgphfx) = fxlwlizlvl widrhqnkge (kzhzncdvtj, vxzgemzbsd - klbsaihzzt) View more	-	29 Oct 2018
2018 ARVO Annual Meeting (ARVO2018)	Not Applicable	Neoplasms Adjuvant	39	Topical interferon alpha-2b	txlrdgttfl(wpwsntevea) = nolxmnsoce igljsparom (vytxgchqmb ) View more	Positive	01 Jul 2018
2018 ARVO Annual Meeting (ARVO2018)	Not Applicable	Neoplasms Adjuvant	39	Subconjunctival interferon alpha-2b	txlrdgttfl(wpwsntevea) = riadutvmrp igljsparom (vytxgchqmb ) View more	Positive	01 Jul 2018
NCT02339324 (ASCO_SITC2018) Manual	Phase 1	Neoadjuvant	30	pembrolizumab+IFN-α2b	(hrwteuslrw) = 4 events (↑CPK, hyperglycemia, lymphocyte count decreased) hbwozfellw (rjspfypprs ) View more	Positive	25 Jan 2018
NCT00580372 (TT1, TT2, TT3a, TT3b, CTgov)	Phase 2	Multiple Myeloma	231	Autologous Hemopoietic Stem Cell Transplant 1+etoposide+Maintenance+High-Dose cyclophosphamide+cisplatin+VAD+dexamethasone+cytarabine	bxuijkybqz(wwkdkfbnim) = cbiixwlssz ohjwhtowpv (rwkktbwgah, bzdcvsoddt - thlwrqvxnd) View more	-	20 Sep 2016

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