March 2024 | Corporate Presentation
www.fennecpharma.com
FEN-1321-v9 | 1 |
Safe Harbor Statement
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.govand www.sedar.com.
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Fennec Pharmaceuticals is a Commercial Stage Biotechnology
Company Dedicated to Improving the Lives of Children with Cancer
- PEDMARK® is FDA-approved to reduce the risk of ototoxicity or hearing loss associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors.
- Unique formulation of sodium thiosulfate specifically developed in pediatrics
- U.S. commercial launch October 2022
- 7 years U.S. market exclusivity with Orphan Drug Designation
- Orange Book Patents provide protection until 2039
- In Europe:
- EU approval received June 2023; UK approval received October 2023
- 10 YEARS E.U. exclusivity with Pediatric-useMarketing Authorization (PUMA)
- Entered into exclusive licensing agreement with Norgine to commercialize PEDMARQSI in Europe, Australia and New Zealand
US packaging represented
Please see Important Safety Information at the end of this presentation
Full Prescribing Information is available at www.PEDMARK.com
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PEDMARK® Registrational Trials Met Their Endpoints
Registrational Trials Design | Manageable Safety Profile |
SIOPEL 6: randomized, controlled, open-label | COG ACCL0431: randomized, controlled, open- | |
label study of 125 patients aged 1 to 18 years w/ | ||
study of 114 patients aged 1 month to 18 years | ||
solid tumors receiving a chemotherapy regimen | ||
receiving cisplatin-based chemotherapy for | ||
for any stage disease that included a cumulative | ||
standard risk hepatoblastoma | ||
cisplatin dose of ≥200 mg/m2 | ||
-
The most common adverse reactions
(≥25% with difference between arms of >5% compared to cisplatin alone) in SIOPEL6 are:
Primary Endpoint: Hearing Loss | ‒ Vomiting |
PEDMARK reduced the relative risk of hearing loss | PEDMARK reduced the relative risk of hearing loss |
by 48% compared with cisplatin alone* | by 49% compared with cisplatin alone** |
‒ | Nausea |
‒ | Decreased hemoglobin |
p = 0.002 |
63.0 |
% |
29/46 |
32.7% |
18/55 |
Overall Hearing Data
p = 0.004
Overall Hearing Data
‒ Hypernatremia |
• The most common adverse reaction |
(≥25% with difference between arms |
of >5% compared to cisplatin alone) |
in COG ACCL0431 is: |
‒ Hypokalemia |
*A total of 109 children were randomly assigned to receive cisplatin plus sodium thiosulfate (n=57) or cisplatin alone (n=52) and could be evaluated. The absolute hearing threshold was assessed in 101 children. PEDMARK's
N = 101 evaluable patients | N = 104 evaluable patients | ||||||
Brock et al, N Engl J Med 2018;378:2376-85. | Freyer et al, www.thelancet.com/oncology Vol 18 January 2017 |
FDA-approved label included patients without data that were lost to follow up and assumed to have hearing loss.
**A total of 125 children were randomly assigned to receive either sodium thiosulfate (n=61) or observation (n=64). Of these, 104 participants were assessable for the primary endpoint. PEDMARK's FDA-approved label included patients without data that were lost to follow up and assumed to have hearing loss. The on-label reduction in relative risk of hearing loss with PEDMARK is 25% compared with cisplatin alone.
Please see Important Safety Information at the end of this presentation | |
Full Prescribing Information is available at www.PEDMARK.com | 4 |
Cisplatin is the Standard of Care for Pediatric Solid Tumors
Cisplatin | Penicillin of Chemotherapy
- Interferes with DNA replication killing fast proliferating cells
- Administered as intravenous infusion in normal saline
- For treatment of solid and hematological malignancies
- Relatively short half-life
- First licensed in 1979
- Introduced in pediatric patients in 1980s
- It is on the WHO's List of Essential Medicines
- High cure rates achieved in pediatric patients, in contrast to adults
Common Childhood Cancers Treated
with Cisplatin
• Brain and CNS cancers | • | Osteosarcoma | |
• | Neuroblastoma | • | Germ cell tumors |
• | Hepatoblastoma | • | Retinoblastoma |
Treatment plan depends on the individual cancer diagnosis, stage of disease and patient age
Platinum cancer drugs. Available at cisplatin.org Accessed September 7, 2022.
Robertson J, et al. Bull World Health Organ. 2016 Oct 1; 94(10): 735-742.
Ward et al. CA Cancer J Clin. 2014;64:83-103.
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Pediatric Oncology Incidence is Consistent in U.S. and EU
Annual incidence of pediatric solid tumor cases eligible for platinum-based therapy in both U.S. and EU markets*
U.S. Market | European Market |
5,016 | |
5,925 | |
~30% 1,462 Metastatic | ~30% 1,710Metastatic |
~70% 3,554 Localized, non-metastatic | ~70% 4,215 Localized, non-metastatic |
*Sources: http://accis.iarc.fr/results/2003/pdfs/summaryincidencetables.pdf Accessed Feb 2022; Ward, E CA CANCER J CLIN 2014;64:83-103 Localized vs metastatic breakdown based on Qualitative Market Research Study Completed February 2018
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One of the unfortunate
complications with cisplatin
therapy is ototoxicity
*Actor Portrayal
Corporate Deck 7 7
Cisplatin Results in High-Frequency Hearing Loss in Children
Common Clinical Presentation of Hearing Loss
- High frequency (≥4 kHz) sensorineural hearing loss1,2
- Bilateral (both ears)
- Progressive
- Irreversible
- Can progress to include lower frequencies (<4 kHz)3
- Can be accompanied by tinnitus3
- Prolonged retention of platinum may cause hearing loss progression after completion of therapy4
- Hearing aids may be necessary in up to 40%; and cochlear implants in an additional percentage of children affected3
Risk Factors for Ototoxicity1,2
- Younger age (<5 years of age)
- Cranial irradiation
- Total dose and duration of platinum agent
- Exposure to other ototoxic medications
- Pre-existingrenal insufficiency
- Pre-exposureto therapies that impair hearing ability
- Genetic factors
Hearing loss that is serious enough for hearing aid use has been independently associated with declines in cognition and educational performance5
1. Waissbluth S et al. Int J Pediatr Otorhinolaryngol. 2018;111:174-179. 2. Paken J et al. J Toxicol. 2016;2016:1809394.
3. Langer T et al. Trends in Pharmacological Sciences. 2013;34:458-469. 4. Sprauten M. J. Clin Oncol. 2012;30:300-307. 5. Schreiber et al., Neuro Oncol, 2014;16(8):1129-36.
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CIO Can Occur Early in Treatment - As Early As Cycles 1-21
-10 | |
0 | |
dB | 10 |
20 | |
in | |
30 | |
level | |
40 | |
threshold | |
50 | |
Hearing | 60 |
70 | |
80 | |
90 | |
100 |
0.125 | 0.250 | 0.500 | 1 | 1.5 | 2 | 3 | 4 | 6 | 8 | 10 | |||
*Audiogram of 1 patient | Frequency in kHz | ||||||||||||
Key: | Pre | Post 1st block | Post 2nd block | Post 3rd block | Post 4th block | 1st follow up | |||||||
Audiogram indicates how loud a sound must be to hear it at a given frequency.
- Ototoxicity is a cisplatin dose-limiting toxicity1 meaning that efficacy of chemotherapy could be compromised due to ototoxicity management
- Effects can be seen as soon as the second or third dose of cisplatin
- Survivors are at risk of hearing deterioration years after completion of therapy2
1. Langer T et al. Trends in Pharmacological Sciences. 2013;34:458-469. 2. Bertolini P et al, J Pediatric Hem Onc 2004;26:649-655.
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Clinical Manifestations
Effects on growth and development
• Certain consonants (f/th/p/k/h/t) are inaudible, compromising speech |
recognition and comprehension in young children1 |
• High frequency hearing loss affects recognition of plurals such as /s/ in |
'ducks' and /z/ in 'girls', resulting in delayed language development3 |
• Speech perception in background noise is hindered, resulting in poorer |
school performance (e.g., literacy)1,4,5 |
• Impaired perception of music and ambient noises, resulting in a poorer |
quality of life1 |
Career
Limitations
(Lost
Earnings)
Speech
Impairment
Social
Isolation
Life-Long
• Delayed neurocognitive and psychosocial development1 |
Hearing loss is associated with a lower IQ, phonetic decoding and reading comprehension6
Excess
Medical
Visits
ImpactLife-Long Impact
Impaired Learning
Development
Delay
(Behavior)
1. Langer T et al. Trends in Pharmacological Sciences. 2013;34:458-469. 2. Bertolini P et al, J Pediatric Hem Onc 2004;26:649-655.
3. Brock PR et al. J Clin Oncol 2010;30:2408-2417. 4. Gurney JG et al. Pediatrics. 2007;e1229-e1236. 5. Crandell CC. Ear & Hearing. 1993; 14:210-217.
6. Hennegan, K, Silber A, Dehipawala S, Chithran K, Lockhart D. Poster PIH67. ISPOR Annual Meeting 2020.
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Fennec Pharmaceuticals Inc. published this content on 31 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 20 April 2024 07:43:01 UTC.