Workflow
Fennec Pharma(FENC) - 2022 Q4 - Annual Report
FENCFennec Pharma(FENC)2023-03-28 16:00

Product Approval and Market Exclusivity - PEDMARK® received FDA approval on September 20, 2022, making it the first and only treatment approved to reduce the risk of ototoxicity associated with cisplatin in pediatric patients[13]. - The company announced the commercial availability of PEDMARK® in the United States on October 17, 2022[13]. - The company received Orphan Drug Exclusivity for PEDMARK® in January 2023, providing seven years of market exclusivity until September 20, 2029[17]. - PEDMARK® is estimated to potentially benefit over 10,000 children annually in the U.S. and Europe receiving platinum-based chemotherapy[20]. - The company has received Orphan Drug Exclusivity, preventing FDA approval of any generic versions for at least 7 years from PEDMARK®'s approval date of September 20, 2022[46]. - The company is pursuing a Pediatric Use Marketing Authorization (PUMA) in the EU, which could provide an additional 10 years of market protection[26]. - The company can apply for Pediatric Marketing Use Authorization (PUMA) for drugs developed exclusively for children, which provides 8 plus 2 years of regulatory protection[92][94]. - The European Orphan Drug Regulation provides eight years of data exclusivity and two years of marketing exclusivity for drugs treating rare conditions affecting five or fewer per 10,000 people in the EU[128]. Intellectual Property and Patent Issues - The company has three patents listed for PEDMARK® in the FDA's Orange Book, with two patents expiring in 2039 and one in 2038[18]. - Hope Medical Enterprises, Inc. filed two petitions for inter partes review (IPR) to invalidate U.S. Patent No. 10,596,190 and U.S. Patent No. 10,792,363 related to PEDMARK®[40]. - The U.S. '190 Patent is set to expire in January 2038, while the U.S. '363 Patent is set to expire in July 2039[45]. - The company expects a decision on the IPR for both patents in May 2023, which can be appealed by the losing party[42]. - The company plans to vigorously defend its intellectual property rights related to PEDMARK®, but an invalidation could adversely affect its market exclusivity[44]. - The company is actively pursuing additional patent applications in the U.S. and internationally for PEDMARK®[18]. - The company must list all patents related to its drug products in the FDA's Orange Book upon NDA approval, which can affect generic competition[96]. Financial Performance and Risks - The company reported a net loss of approximately 23.71millionfortheyearendedDecember31,2022,andanaccumulateddeficitofapproximately23.71 million for the year ended December 31, 2022, and an accumulated deficit of approximately 203.2 million as of the same date[184]. - The company has cash and cash equivalents of 23.8millionavailableasofDecember31,2022,whichisexpectedtofundoperationsforatleastthenext12months[189].−Thecompanyhasahistoryofsignificantlossesandhasgeneratedlimitedrevenuefromproductsalessinceinception,highlightingfinancialrisks[174].−ThecompanymayrequireadditionalfinancingtoobtainregulatoryapprovalandcommercializePEDMARK®,whichcoulddelayorlimitproductdevelopment[174].−ThecompanymayrequireadditionalfinancingtoobtainmarketingapprovalofPEDMARK®andcommercializeitabroad[189].ManufacturingandSupplyChain−ThecompanyisreliantoncontractmanufacturersforPEDMARK®productionandhasnoplanstobuildin−housemanufacturingcapabilities[51].−Thecompanyhasenteredintoagreementswithsuppliersfortheactivepharmaceuticalingredient(API)andthird−partymanufacturersforPEDMARK®vials[52].−TheOHSUAgreementforPEDMARK®includesmilestonepaymentsandroyaltiesonnetsales,withtheagreementexpiringin2038or8yearsfromthelastpatentclaimexpiration[55][58].−CompliancewithcGMPregulationsismandatoryforthird−partymanufacturers,impactingqualitycontrolandassurance[208].−TheFDAissuedaCompleteResponseLetter(CRL)inAugust2020andNovember2021duetodeficienciesinthethird−partymanufacturingfacilityforPEDMARK®[208].RegulatoryEnvironmentandCompliance−ThedrugapprovalprocessintheU.S.typicallytakesseveralyearsandrequiressubstantialresources,withthereviewprocessformarketingapplicationsgenerallytaking12to18months[71][78].−CompaniesmustensurecompliancewithcurrentGoodManufacturingPractices(cGMP)andmayfacelegalorregulatoryactionsfornon−compliance,includingrecallsandcivilpenalties[82][83].−Thecompanyisrequiredtodiscloseclinicaltrialresultswithinapplicabletimeperiods,withpenaltiesforfailuretodoso[71].−TheapprovalprocessinCanadaandtheU.S.isconsideredamongthemostrigorousintheworld,requiringadherencetostrictsafety,efficacy,andqualitystandards[72].−Thecompanyissubjecttovariouslawsandregulations,includingthoserelatedtoGoodManufacturingPractices,whichmustbeadheredtoforpharmaceuticalproductmanufacturing[90].−Thecompanyfacesrisksrelatedtomaintainingregulatorycomplianceandpotentialenforcementactions[180].−Non−compliancewithFDAregulationscouldleadtoreputationalharmandsanctions,includingfundingissuesanddrugeffectivenessconcerns[209].−Potentialconsequencesofnon−complianceincludedelays,warningletters,fines,andproductrecalls[210].−Thereisariskoftotalorpartialsuspensionofproductionifregulatoryissuespersist[211].MarketCompetitionandChallenges−Thebiotechnologyandpharmaceuticalindustriesarehighlycompetitive,withpotentialcompetitorshavingmoreresourcesandexperience[60].−Thecompanyissubjecttosubstantialcompetitioninthebiopharmaceuticalmarket,whichcouldaffectitsmarketposition[175].−ThecompanyanticipatessubstantialregulatoryreviewpriortothecommercializationofPEDMARK®outsideoftheUnitedStates[188].−TheongoingCOVID−19pandemicmayimpactoperations,buthasnotmateriallyaffectedthecompanytodate[59].−TheongoingCOVID−19pandemicposesriskstothecompany′sbusinessoperationsandfinancialcondition[175].−Thecompanyfacesscrutinyregardingdrugpricing,particularlyfororphandrugs,whichcouldaffectitspricingstrategies[122].ResearchandDevelopment−Researchanddevelopmentexpensesdecreasedto23.8 million available as of December 31, 2022, which is expected to fund operations for at least the next 12 months[189]. - The company has a history of significant losses and has generated limited revenue from product sales since inception, highlighting financial risks[174]. - The company may require additional financing to obtain regulatory approval and commercialize PEDMARK®, which could delay or limit product development[174]. - The company may require additional financing to obtain marketing approval of PEDMARK® and commercialize it abroad[189]. Manufacturing and Supply Chain - The company is reliant on contract manufacturers for PEDMARK® production and has no plans to build in-house manufacturing capabilities[51]. - The company has entered into agreements with suppliers for the active pharmaceutical ingredient (API) and third-party manufacturers for PEDMARK® vials[52]. - The OHSU Agreement for PEDMARK® includes milestone payments and royalties on net sales, with the agreement expiring in 2038 or 8 years from the last patent claim expiration[55][58]. - Compliance with cGMP regulations is mandatory for third-party manufacturers, impacting quality control and assurance[208]. - The FDA issued a Complete Response Letter (CRL) in August 2020 and November 2021 due to deficiencies in the third-party manufacturing facility for PEDMARK®[208]. Regulatory Environment and Compliance - The drug approval process in the U.S. typically takes several years and requires substantial resources, with the review process for marketing applications generally taking 12 to 18 months[71][78]. - Companies must ensure compliance with current Good Manufacturing Practices (cGMP) and may face legal or regulatory actions for non-compliance, including recalls and civil penalties[82][83]. - The company is required to disclose clinical trial results within applicable time periods, with penalties for failure to do so[71]. - The approval process in Canada and the U.S. is considered among the most rigorous in the world, requiring adherence to strict safety, efficacy, and quality standards[72]. - The company is subject to various laws and regulations, including those related to Good Manufacturing Practices, which must be adhered to for pharmaceutical product manufacturing[90]. - The company faces risks related to maintaining regulatory compliance and potential enforcement actions[180]. - Non-compliance with FDA regulations could lead to reputational harm and sanctions, including funding issues and drug effectiveness concerns[209]. - Potential consequences of non-compliance include delays, warning letters, fines, and product recalls[210]. - There is a risk of total or partial suspension of production if regulatory issues persist[211]. Market Competition and Challenges - The biotechnology and pharmaceutical industries are highly competitive, with potential competitors having more resources and experience[60]. - The company is subject to substantial competition in the biopharmaceutical market, which could affect its market position[175]. - The company anticipates substantial regulatory review prior to the commercialization of PEDMARK® outside of the United States[188]. - The ongoing COVID-19 pandemic may impact operations, but has not materially affected the company to date[59]. - The ongoing COVID-19 pandemic poses risks to the company's business operations and financial condition[175]. - The company faces scrutiny regarding drug pricing, particularly for orphan drugs, which could affect its pricing strategies[122]. Research and Development - Research and development expenses decreased to 3.5 million in fiscal year 2022 from $5.0 million in 2021, reflecting a shift towards commercial readiness and launch activities for PEDMARK®[164]. - The company has established relationships with contract research organizations and universities to enhance its drug development capabilities[163]. - Phase 3 clinical trials usually take two to four years to complete and involve several hundred to several thousand patients, making them the most time-consuming and expensive part of the clinical trial program[78]. Sales and Reimbursement - The company emphasizes the importance of securing third-party reimbursement for PEDMARK®, which is critical for sales[27]. - Sales of PEDMARK® will depend on reimbursement by payers, which may be limited or unavailable in certain market segments[178]. - Federal law mandates pharmaceutical manufacturers to pay rebates to state Medicaid programs, with the basic rebate for innovator products set at 23.1% of the average manufacturer price[145]. - Effective January 1, 2024, the cap on rebate amounts for drugs will be eliminated, potentially allowing rebates to exceed the average price received by manufacturers[146]. Legislative and Regulatory Changes - The company anticipates ongoing legislative and regulatory changes in the U.S. healthcare system that could significantly impact its business[116]. - Proposed changes to the Orphan Drug Act may limit benefits for drugs treating more than 200,000 individuals, creating uncertainty for future product approvals[124]. - Pediatric exclusivity can extend marketing protection by an additional six months if pediatric data is submitted in response to FDA requests[125]. - The FDA's Breakthrough Therapy Designation aims to expedite the development of drugs showing substantial improvement over existing therapies for serious conditions[129]. - Fast Track Designation allows for expedited review of drugs addressing unmet medical needs, with a determination made within 60 days of the request[130]. - Priority Review can shorten the review time to six to eight months for drugs that address serious conditions or unmet medical needs[136].