Pedmark (sodium thiosulfate) / Fennec Pharma, Norgine - LARVOL DELTA

Testing the Addition of Pedmark to Cisplatin Chemotherapy for Reducing Drug-Induced Ear Damage in Men With Stage II-III Metastatic Testicular Germ Cell Tumors (clinicaltrials.gov) - P1 | N=44 | Not yet recruiting | Sponsor: City of Hope Medical Center

New P1 trial • Genito-urinary Cancer • Germ Cell Tumors • Oncology • Otorhinolaryngology • Solid Tumor • Testicular Cancer • Testicular Non-seminoma • Testicular Seminoma

Norgine strengthens rare disease portfolio with acquisition of Theravia (PRNewswire) - "Norgine and Theravia announced today that they have entered into a definitive agreement under which Norgine will acquire Theravia, an international pharmaceutical company specialising in cutting-edge treatments for patients with rare and debilitating conditions....With the acquisition of Theravia, Norgine will now have...core products in its rare disease portfolio (PEDMARQSI, eflornithine, mavorixafor, SIKLOS) thereby comprising a franchise of critical scale with the ability to be a key growth driver in the medium-to-long-term."

M&A • Dermatology • Genetic Disorders • Hematological Disorders • Otorhinolaryngology

Fennec Pharmaceuticals Reports Fourth Quarter and Full-Year 2024 Financial Results and Provides Business Update (GlobeNewswire) - "Achieved Full-Year PEDMARK Net Product Sales of $29.6 Million, Up 40% Year-Over-Year, and Generated PEDMARK Q4 2024 Net Product Sales of $7.9 Million....Ex-U.S. Opportunities for PEDMARK: In Japan, the investigator-initiated clinical trial (STS-J01) in Japan evaluating PEDMARK fully enrolled in Q4 2024 and the results of the trial are expected in the second half of 2025 with the potential evaluation for registration of PEDMARK in Japan thereafter."

P2 data • Sales • Oncology • Solid Tumor

Real-World Experience Using Sodium Thiosulfate Pentahydrate Off-Label for Cisplatin Otoprotection in Children, Adolescents, and Young Adults. (PubMed, Pediatr Blood Cancer) - "STS-P off-label for otoprotection following cisplatin was tolerable in a real-world setting across age groups and cancer types. Formal testing in larger studies of different STS formulations is needed to explore possible differences in toxicity and CIHL prevention."

Journal • Real-world evidence • Oncology • Otorhinolaryngology • Pediatrics • Solid Tumor

Fennec Pharmaceuticals Announces Commercial Launch of PEDMARQSI In Germany (GlobeNewswire) - "Fennec Pharmaceuticals Inc...announced that Norgine Pharmaceuticals Ltd., a leading European specialist pharmaceutical company, has commercially launched PEDMARQSI (sodium thiosulfate injection) in Germany....The data from two open-label, randomized Phase 3 trials, SIOPEL 6 (pivotal) and the Clinical Oncology Group (COG) Protocol ACCL0431, demonstrated an approximate 50% reduction in the occurrence of cisplatin-induced ototoxicity in patients treated with cisplatin and sodium thiosulfate vs. those treated with cisplatin alone."

Launch Europe • Otorhinolaryngology • Solid Tumor

Fennec Pharmaceuticals Announces Pedmarqsi Positive Recommendation by NICE for the Prevention of Cisplatin-Induced Hearing Loss in England and Wales (GlobeNewswire) - "Fennec Pharmaceuticals Inc...announced that Norgine Pharmaceuticals Ltd...has received positive final draft guidance from National Institute for Health and Care Excellence (NICE) recommending PEDMARQSI for the prevention of cisplatin-induced hearing loss in patients (aged 1 month to 17 years) with localized, non-metastatic, solid tumors."

NICE • Otorhinolaryngology

Fennec Pharmaceuticals Reports Third Quarter 2024 Financial Results and Provides Business Update (GlobeNewswire) - "Increasing Momentum and Successful Reimbursement in the Adolescent and Young Adult (AYA) Segment Following Strategic Investments to Drive Awareness of Ototoxicity & Adoption of PEDMARK; PEDMARQSI is expected to be available in select markets in Europe in the coming months, which will generate additional revenue for Fennec in 2025 and beyond; Investigator-initiated clinical trial (STS-J01) in Japan evaluating PEDMARK fully enrolled in October 2024...Results of the trial are expected in 2025 with the potential evaluation for registration of PEDMARK in Japan thereafter."

Launch Europe • Reimbursement • Trial status • Oncology • Solid Tumor

THE VALUE AND CHALLENGES OF LOCAL APPLICATION OF SODIUM-THIOSULFATE (STS) AS OTOPROTECTANT IN CISPLATIN-TREATED CANCER PATIENTS: PERSPECTIVES FOR CHILDREN BASED ON A LITERATURE REVIEW (SIOP 2024) - "The burden of intratympanic procedures in children (who require recurrent sedation), together with the low level of evidence of efficacy, leads to the conclusion that (for now) this strategy is unfeasible for otoprotection in cisplatin-exposed children with cancer. STS* = Pedmarqsi/Pedmark (Fennec compound)"

Clinical • Review • Anesthesia • Oncology • Pediatrics

Effect of sodium thiosulfate anhydrous on cisplatin-induced ototoxicity in pediatric cancers: Turkish compassionate use treatment protocol—Updated results. (ASCO 2024) - "Background: Cisplatin induced ototoxicity (CIO), with permanent hearing loss is observed in >60% pediatric patients in Turkey. PEDMARK was well tolerated, provided via a Turkish CUP to reduce the CIO in pediatric patients with nonmetastatic solid tumors. No hearing loss was observed/ detected post-treatment and the tumor outcomes were consistent with the Phase 3 studies. This is very encouraging."

Clinical • Brain Cancer • Gastrointestinal Cancer • Hepatoblastoma • Hepatology • Medulloblastoma • Nasopharyngeal Carcinoma • Oncology • Otorhinolaryngology • Pediatrics • Solid Tumor

Fennec Pharmaceuticals Reports First Quarter 2024 Financial Results and Provides Business Update (GlobeNewswire) - "Achieved PEDMARK net product revenue of approximately $7.4 million in the first quarter of 2024 and total net revenues of $25.4 million, which is inclusive of $18.0 million in revenue from the Norgine transaction. Amended permanent J-Code, which became effective on April 1, 2024, now clearly specifies PEDMARK from other formulations of sodium thiosulfate (STS)."

Reimbursement • Sales • Oncology • Solid Tumor

Fennec Pharmaceuticals Reports Full Year and Fourth Quarter 2023 Financial Results and Provides Business Update (GlobeNewswire) - "Entered into exclusive licensing agreement to commercialize PEDMARQSI in Europe, Australia and New Zealand. Fennec received approximately $43 million upfront and has the potential to receive up to approximately $230 million in additional commercial and regulatory milestones, and double-digit tiered royalties up to the mid-twenties....Net product sales of $21.3 million in fiscal 2023 compared to $1.5 million in 2022. The Company had gross profit of $20.0 million for fiscal year ended 2023. The increase in sales reflects strong growth in new patient starts and accounts."

Licensing / partnership • Sales • Oncology • Solid Tumor

Fennec Pharmaceuticals and Norgine Enter into Exclusive Licensing Agreement to Commercialize PEDMARQSI in Europe, Australia, and New Zealand (GlobeNewswire) - "Fennec Pharmaceuticals...and Norgine...announced an exclusive licensing agreement under which Norgine will commercialize PEDMARQSI in Europe, Australia and New Zealand. PEDMARQSI is the first and only approved therapy in the EU and U.K. for the prevention of ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients 1 month to < 18 years of age with localized, non-metastatic solid tumors. Under the terms of the licensing agreement, Fennec will receive €40 million in upfront consideration and up to €210 million in additional commercial and regulatory milestone payments and double-digit tiered royalties on net sales of PEDMARQSI in the licensed territories up to the mid-twenties. Norgine will be responsible for all commercialization activities in the licensed territories..."

Licensing / partnership • Solid Tumor

FDA Issues Reminder of Non-Substitution of PEDMARK (sodium thiosulfate injection) for Pediatric Patients Receiving Cisplatin (GlobeNewswire) - "Fennec Pharmaceuticals Inc...announced that the U.S. Food and Drug Administration (FDA) has issued a public reminder to healthcare providers that PEDMARK (sodium thiosulfate injection) is not substitutable with other sodium thiosulfate products as explicitly directed in its prescribing label. PEDMARK is the first and only FDA approved therapy indicated to reduce the risk of ototoxicity (e.g., permanent hearing loss) associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors...The FDA stated that such substitutions pose potential health risks, including: Potassium chloride exposure which, at high doses, can lead to increased risk of acute cardiac events and other serious adverse reactions. Potassium chloride is not present in PEDMARK....Sodium nitrite is co-packaged with sodium thiosulfate as a separate vial in some products; it is not present in PEDMARK."

FDA event • Oncology

Fennec Announces Incremental $5 Million Investment from Petrichor (GlobeNewswire) - "Fennec Pharmaceuticals Inc...announced the third closing of $5 million senior secured promissory notes under the existing agreement with Petrichor. The original investment agreement provided access to up to $20 million of additional financing through December 31, 2023. As part of this closing, Fennec and Petrichor have amended the agreement to provide access to up to $15 million of additional financing through December 31, 2024. 'We appreciate Petrichor’s continued support of Fennec and our efforts to grow the use of PEDMARK within the global pediatric cancer community'..."

Financing • Oncology

Fennec Pharmaceuticals Announces Third Quarter 2023 Financial Results and Provides Business Update (GlobeNewswire) - "'We continued to see strong commercial performance with PEDMARK^® in the third quarter demonstrated by net product revenue of $6.5 million representing 96% quarter over quarter growth'....Research and development expenses decreased by $0.8 million for the three months ended September 30, 2023, compared to the same period in 2022. The Company’s research and development activities for the quarter ended September 30, 2023 consisted of costs associated with investigator initiated clinical trials. During the same period in 2022 and prior to approval of PEDMARK^®, manufacturing costs pertaining to PEDMARK^® were allocated to R&D expense in the period incurred, and following approval are reflected in inventory."

Commercial • Sales • Oncology

Fennec Pharmaceuticals Announces Second Quarter 2023 Financial Results and Provides Business Update (GlobeNewswire) - "Fennec Pharmaceuticals Inc...today reported its financial results for the second quarter ended June 30, 2023 and provided a business update. 'During the second quarter, we continued to make strong progress with the U.S. commercial launch of PEDMARK®, delivering net revenue of $3.3 million, which is a 98% increase in net revenue over the first quarter of 2023.'"

Sales • Oncology • Otorhinolaryngology

Fennec Pharmaceuticals Announces European Commission Marketing Authorization for Pedmarqsi (sodium thiosulfate) to Reduce the Risk of Hearing Loss in Pediatric Oncology Patients (GlobeNewswire) - "Fennec Pharmaceuticals Inc...announced Pedmarqsi™– known as PEDMARK^® in the U.S. – was granted marketing authorization by the European Commission. Pedmarqsi is the first and only approved therapy in the EU for the prevention of ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localised, non-metastatic, solid tumors. Further, Pedmarqsi was granted the marketing authorization under the paediatric-use marketing authorization (PUMA) which includes 8 years plus 2 years of data and market protection....The CHMP’s decision was based on safety and efficacy data from two pivotal open-label, randomized Phase 3 trials (SIOPEL 6 and Clinical Oncology Group [COG] Protocol ACCL0431)...The marketing authorization is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein."

European regulatory • Oncology • Solid Tumor

"$FENC Q1 PEDMARK net sales $1.67M" (@BioStocks)

A New Approach for Reducing the Risk of Ototoxicity Associated with Cisplatin in Pediatric Patients 1 Month of Age and older, with Localized Non-Metastic Solid Tumors (ASPHO 2023) - "The goal of this program is to introduce the new FDA-approved medication PEDMARK, indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastic sold tumors. Content will include a discussion of related clinical trials, dosing and administration, and safety."

Adverse events • Clinical • Oncology • Pediatrics • Solid Tumor

Fennec Pharmaceuticals Receives Positive CHMP Opinion for Pedmarqsi (sodium thiosulfate) to Reduce the Risk of Hearing Loss in Pediatric Oncology Patients (GlobeNewswire) - "Fennec Pharmaceuticals Inc...announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion and recommended granting a Marketing Authorization for Pedmarqsi™ (sodium thiosulfate) – known as PEDMARK^® in the U.S. – for the prevention of ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localized, non-metastatic, solid tumors. The CHMP adopted its positive opinion on safety and efficacy data from two pivotal open-label, randomized Phase 3 trials (SIOPEL 6 and Clinical Oncology Group [COG] Protocol ACCL0431)...The CHMP's recommendation will now be reviewed by the European Commission, ratification of the CHMP recommendation is expected by early June 2023."

European regulatory • Oncology • Solid Tumor

Fennec Pharmaceuticals Reports Full Year and Fourth Quarter 2022 Financial Results (GlobeNewswire) - "The FDA granted Orphan Drug Exclusivity to PEDMARK^® (sodium thiosulfate injection) in January 2023....The seven-year market exclusivity for PEDMARK^® began on September 20, 2022, the date of its FDA approval, and continues until September 20, 2029. Additionally, in the approved prescribing label, the FDA has explicitly directed that PEDMARK^® is not substitutable with other sodium thiosulfate products."

Orphan drug • Oncology

Fennec Pharmaceuticals Announces Updated NCCN Clinical Practice Guidelines Recommend PEDMARK (sodium thiosulfate injection) to Reduce the Risk of Cisplatin-Induced Hearing Loss in Pediatric Patients (GlobeNewswire) - "Fennec Pharmaceuticals Inc...announced that the National Comprehensive Cancer Network^® (NCCN) has updated its clinical practice guidelines for Adolescent and Young Adult (AYA) Oncology to include PEDMARK^® (sodium thiosulfate injection) to help reduce the risk of ototoxicity (hearing loss) associated with cisplatin use in pediatric patients with localized, non-metastatic solid tumors....The update to the Clinical Guidelines for AYA Oncology follows the U.S. Food and Drug Administration (FDA) approval of PEDMARK in September 2022, which was based upon safety and efficacy data from two pivotal open-label, randomized Phase 3 trials (Clinical Oncology Group [COG] Protocol ACCL0431 and SIOPEL 6), which compared PEDMARK plus a cisplatin-based regimen to cisplatin-based regimens alone for the reduction of cisplatin-induced hearing loss in pediatric patients."

NCCN guideline • Oncology • Solid Tumor

Drug Approved to Prevent Cisplatin-Associated Ototoxicity in Children. (PubMed, Am J Nurs) - "Sodium thiosulfate injection (Pedmark) has been approved to decrease the risk of ototoxicity associated with cisplatin use in children who are at least one month old and have localized nonmetastatic solid tumors.Nurses should regularly weigh children taking this drug, as the dose is based on weight. Patients should be premedicated with antiemetics to prevent nausea and vomiting and monitored closely for evidence of electrolyte imbalances."

Journal • Oncology • Solid Tumor

Sodium Thiosulfate: Pediatric First Approval. (PubMed, Paediatr Drugs) - "Sodium thiosulfate (Pedmark) is a chemoprotectant/antioxidant developed by Fennec Pharmaceuticals (formerly Adherex Technologies) to reduce to risk of hearing loss associated with cisplatin. Sodium thiosulfate is under regulatory review in the EU for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to < 18 years of age with localised, non-metastatic, solid tumours. This article summarizes the milestones in the development of sodium thiosulfate leading to this pediatric first approval for reducing the risk of ototoxicity associated with cisplatin in pediatric patients."

Journal • Review • Oncology • Otorhinolaryngology • Pediatrics • Solid Tumor

FDA approves sodium thiosulfate to reduce the risk of ototoxicity associated with cisplatin in pediatric patients with localized, non-metastatic solid tumors (FDA) - "On September 20, 2022, the Food and Drug Administration approved sodium thiosulfate (Pedmark, Fennec Pharmaceuticals Inc.) to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month and older with localized, non-metastatic solid tumors....Efficacy was evaluated in two multicenter open-label, randomized controlled trials in pediatric patients undergoing treatment with cisplatin-based chemotherapy for cancer: SIOPEL 6 (NCT00652132) and COG ACCL0431 (NCT00716976)."

sNDA • Oncology • Solid Tumor