Fennec Pharmaceuticals Announces Pedmarqsi® Positive Recommendation by NICE for the Prevention of Cisplatin-Induced Hearing Loss in England and Wales

Rhea-AI Summary

Fennec Pharmaceuticals (NASDAQ:FENC) announced that PEDMARQSI® has received a positive recommendation from NICE for use in England and Wales. The drug is the first and only treatment available within NHS for preventing cisplatin-induced hearing loss in children and young people aged 1 month to 17 years.

Two Phase 3 trials demonstrated approximately 50% reduction in cisplatin-induced ototoxicity compared to cisplatin alone. This addresses a significant unmet need as there were no previous preventative treatments available.

The announcement follows Fennec's March 2024 licensing agreement with Norgine Pharmaceuticals for commercialization in Europe, Australia, and New Zealand. The deal included a $43 million upfront payment and potential additional payments up to $230 million, plus double-digit tiered royalties starting in mid-teens.

Positive

• First and only approved therapy for preventing cisplatin-induced hearing loss in pediatric patients
• Clinical trials showed 50% reduction in hearing loss occurrence
• Secured $43 million upfront payment from Norgine licensing deal
• Potential for additional $230 million in milestone payments
• Double-digit tiered royalties starting in mid-teens

Negative

• None.

Insights

Financial Analyst

The NICE recommendation marks a pivotal revenue opportunity for Fennec Pharmaceuticals in the UK market. The licensing agreement with Norgine, worth up to $273 million including $43 million upfront and $230 million in milestone payments, plus double-digit royalties starting in mid-teens, significantly strengthens Fennec's financial position. With a market cap of $152 million, this deal represents a transformative value proposition. The UK market access through NHS adds substantial recurring revenue potential, considering PEDMARQSI's unique position as the only approved therapy for cisplatin-induced hearing loss prevention. The tiered royalty structure, reaching mid-twenties percentages, indicates strong profit margin potential as sales volumes increase. Pediatric oncology treatments typically command premium pricing, especially for first-in-class therapies addressing unmet medical needs.

Medical Research Analyst

PEDMARQSI's 50% reduction in cisplatin-induced ototoxicity, demonstrated through two Phase 3 trials (SIOPEL 6 and COG ACCL0431), represents a breakthrough in supportive cancer care. The NICE recommendation validates the clinical significance of this first-in-class therapy. Cisplatin remains a cornerstone chemotherapy agent in pediatric solid tumors and its ototoxicity has been a long-standing challenge without pharmacological solutions. The age range approval (1 month to 17 years) covers the critical developmental period where hearing loss can severely impact language development, academic performance and social integration. This protective therapy maintains cisplatin's anticancer efficacy while significantly reducing its most debilitating long-term side effect, potentially improving treatment adherence and outcomes.

Market Research Analyst

The UK market entry through NICE recommendation establishes a strong precedent for other European markets, enhancing PEDMARQSI's commercial prospects across Norgine's licensed territories. The NHS coverage decision typically influences other healthcare systems, particularly in Europe and Commonwealth nations. This approval addresses a clear market gap in supportive cancer care, where prevention of long-term complications is increasingly prioritized. The exclusive licensing structure with Norgine leverages their established European specialty pharmaceutical network, optimizing market penetration. Considering the robust Phase 3 data and first-mover advantage, PEDMARQSI is well-positioned to become the standard of care in pediatric cisplatin chemotherapy protocols.

~ PEDMARQSI^® (anhydrous sodium thiosulfate) is the first and only treatment available within NHS England and Wales for the prevention of cisplatin-induced ototoxicity (hearing loss) in children and young people (1 month-17 years of age) ~

~ 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 ~

~ There is a clear unmet need for the prevention of hearing loss caused by cisplatin and until now, there have been no preventative pharmacological interventions available, despite the significant lifelong impact hearing loss has on cancer patients ~

RESEARCH TRIANGLE PARK, N.C., Dec. 20, 2024 (GLOBE NEWSWIRE) -- Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, today announced that Norgine Pharmaceuticals Ltd., a leading European specialist pharmaceutical company, 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.

PEDMARQSI is the first and only approved therapy in the EU and U.K. for the prevention of ototoxicity, or hearing loss, induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localized, non-metastatic solid tumors. In March 2024, Fennec entered into an exclusive licensing agreement under which Norgine will commercialize PEDMARQSI^® in Europe, Australia, and New Zealand.

“Securing access to PEDMARQSI is a critical milestone for the cancer community in England and Wales to help reduce the risk of ototoxicity, or permanent hearing loss, associated with cisplatin treatment,” said Jeff Hackman, chief executive officer and director of Fennec Pharmaceuticals. “We congratulate Norgine on their collaboration with NICE to reach this important agreement that recognizes the value of ototoxicity intervention as part of the cancer treatment journey.”

Under the terms of the previously announced exclusive licensing agreement with Norgine, Fennec received approximately $43 million in an upfront payment and will receive up to approximately $230 million in additional commercial and regulatory milestone payments along with double-digit tiered royalties on net sales of PEDMARQSI starting in the mid-teens and growing to the mid-twenties.

About Cisplatin-Induced Ototoxicity

Cisplatin and other platinum compounds are essential chemotherapeutic agents for the treatment of many pediatric malignancies. Unfortunately, platinum-based therapies can cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of pediatric cancer.ⁱ

The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids or cochlear implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over time.ⁱⁱ Infants and young children that are affected by ototoxicity at critical stages of development lack speech and language development and literacy, and older children and adolescents often lack social-emotional development and educational achievement.ⁱⁱⁱ

PEDMARK^® (sodium thiosulfate injection)
PEDMARK^® is the first and only U.S. Food and Drug Administration (FDA) approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment in pediatric patients with localized, non-metastatic, solid tumors. PEDMARK is also recommended for the Adolescent and Young Adult (AYA) population by the National Comprehensive Cancer Network^® as a preventative treatment option to reduce hearing loss associated with platinum-based chemotherapy in patients with localized, non-metastatic tumors. PEDMARK is a unique formulation of sodium thiosulfate in single-dose, ready-to-use vials for intravenous use in pediatric patients. PEDMARK is also the first and only therapeutic agent with proven efficacy and safety data with an established dosing regimen, across two open-label, randomized Phase 3 clinical studies, the Children’s Oncology Group (COG) Protocol ACCL0431 and SIOPEL 6.

In the U.S. and Europe, it is estimated that, annually, more than 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult, and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.

Indications and Usage
PEDMARK^® (sodium thiosulfate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors.

Limitations of Use
The safety and efficacy of PEDMARK have not been established when administered following cisplatin infusions longer than 6 hours. PEDMARK may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.

Important Safety Information
PEDMARK is contraindicated in patients with history of a severe hypersensitivity to sodium thiosulfate or any of its components.

Hypersensitivity reactions occurred in 8% to 13% of patients in clinical trials. Monitor patients for hypersensitivity reactions. Immediately discontinue PEDMARK and institute appropriate care if a hypersensitivity reaction occurs. Administer antihistamines or glucocorticoids (if appropriate) before each subsequent administration of PEDMARK. PEDMARK may contain sodium sulfite; patients with sulfite sensitivity may have hypersensitivity reactions, including anaphylactic symptoms and life-threatening or severe asthma episodes. Sulfite sensitivity is seen more frequently in people with asthma.

PEDMARK is not indicated for use in pediatric patients less than 1 month of age due to the increased risk of hypernatremia or in pediatric patients with metastatic cancers.

Hypernatremia occurred in 12% to 26% of patients in clinical trials, including a single Grade 3 case. Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or 4 occurring in 9% to 27% of patients. Monitor serum sodium and potassium levels at baseline and as clinically indicated. Withhold PEDMARK in patients with baseline serum sodium greater than 145 mmol/L.

Monitor for signs and symptoms of hypernatremia and hypokalemia more closely if the glomerular filtration rate (GFR) falls below 60 mL/min/1.73m².

Administer antiemetics prior to each PEDMARK administration. Provide additional antiemetics and supportive care as appropriate.

The most common adverse reactions (≥25% with difference between arms of >5% compared to cisplatin alone) in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia. The most common adverse reaction (≥25% with difference between arms of >5% compared to cisplatin alone) in COG ACCL0431 was hypokalemia.

Please see full Prescribing Information for PEDMARK^® at: www.PEDMARK.com.

About Fennec Pharmaceuticals
Fennec Pharmaceuticals Inc. is a specialty pharmaceutical company focused on the development and commercialization of PEDMARK^® to reduce the risk of platinum-induced ototoxicity in pediatric patients. Further, PEDMARK received FDA approval in September 2022 and European Commission approval in June 2023 and U.K. approval in October 2023 under the brand name PEDMARQSI. PEDMARK has received Orphan Drug Exclusivity in the U.S. and PEDMARQSI has received Pediatric Use Marketing Authorization in Europe which includes eight years plus two years of data and market protection. For more information, please visit www.fennecpharma.com.

Forward Looking Statements
Except for historical information described in this press release, all other statements are forward-looking. Words such as “believe,” “anticipate,” “plan,” “expect,” “estimate,” “intend,” “may,” “will,” or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include statements about our business strategy, timeline and other goals, plans and prospects, including our commercialization plans respecting PEDMARK^®, the market opportunity for and market impact of PEDMARK^®, its potential impact on patients and anticipated benefits associated with its use, and potential access to further funding after the date of this release. Forward-looking statements are subject to certain risks and uncertainties inherent in the Company’s business that could cause actual results to vary, including the risks and uncertainties that regulatory and guideline developments may change, scientific data and/or manufacturing capabilities may not be sufficient to meet regulatory standards or receipt of required regulatory clearances or approvals, clinical results may not be replicated in actual patient settings, unforeseen global instability, including political instability, or instability from an outbreak of pandemic or contagious disease, such as the novel coronavirus (COVID-19), or surrounding the duration and severity of an outbreak, protection offered by the Company’s patents and patent applications may be challenged, invalidated or circumvented by its competitors, the available market for the Company’s products will not be as large as expected, the Company’s products will not be able to penetrate one or more targeted markets, revenues will not be sufficient to fund further development and clinical studies, our ability to obtain necessary capital when needed on acceptable terms or at all, the Company may not meet its future capital requirements in different countries and municipalities, and other risks detailed from time to time in the Company’s filings with the Securities and Exchange Commission including its Annual Report on Form 10-K for the year ended December 31, 2023. Fennec disclaims any obligation to update these forward-looking statements except as required by law.

For a more detailed discussion of related risk factors, please refer to our public filings available at www.sec.gov and www.sedar.com.

PEDMARK^® and Fennec^® are registered trademarks of Fennec Pharmaceuticals Inc.

©2024 Fennec Pharmaceuticals Inc. All rights reserved. FEN-1604-v1

For further information, please contact:

Investors:
Robert Andrade
Chief Financial Officer
Fennec Pharmaceuticals Inc.
+1 919-246-5299

Corporate and Media:
Lindsay Rocco
Elixir Health Public Relations
+1 862-596-1304
lrocco@elixirhealthpr.com

_{ⁱ Rybak L. Mechanisms of Cisplatin Ototoxicity and Progress in Otoprotection. Current Opinion in Otolaryngology & Head and Neck Surgery. 2007, Vol. 15: 364-369.
ⁱⁱ Landier W. Ototoxicity and Cancer Therapy. Cancer. June 2016 Vol. 122, No.11: 1647-1658.
ⁱⁱⁱ Bass JK, Knight KR, Yock TI, et al. Evaluation and Management of Hearing Loss in Survivors of Childhood and Adolescent Cancers: A Report from the Children's Oncology Group. Pediatric Blood & Cancer. 2016 Jul;63(7):1152-1162.}

FAQ

What is the effectiveness rate of PEDMARQSI (FENC) in preventing cisplatin-induced hearing loss?

Clinical trials demonstrated approximately 50% reduction in cisplatin-induced ototoxicity in patients treated with PEDMARQSI compared to those treated with cisplatin alone.

What age group is PEDMARQSI (FENC) approved for in England and Wales?

PEDMARQSI is approved for patients aged 1 month to 17 years with localized, non-metastatic solid tumors.

What are the financial terms of Fennec's (FENC) licensing agreement with Norgine?

The agreement includes a $43 million upfront payment, up to $230 million in additional milestone payments, and double-digit tiered royalties starting in the mid-teens.

Which regions are covered under Fennec's (FENC) licensing agreement with Norgine?

The exclusive licensing agreement covers Europe, Australia, and New Zealand.

When did Fennec Pharmaceuticals (FENC) enter into the licensing agreement with Norgine?

Fennec entered into the exclusive licensing agreement with Norgine in March 2024.