We are a late-stage clinical biopharmaceutical company focused on the discoveryand development of drugs for the treatment of cancer. Our core objective is toleverage our proprietary PDC delivery platform to develop PDCs that are designedto specifically target cancer cells and deliver improved efficacy and bettersafety as a result of fewer off-target effects. Our PDC platform possesses thepotential for the discovery and development of the next generation ofcancer-targeting treatments, and we plan to develop PDCs both independently andthrough research and development collaborations.
The COVID-19 pandemic, including variants thereof, has created uncertainties inthe expected timelines for clinical stage biopharmaceutical companies such asus, and because of such uncertainties, it is difficult for us to accuratelypredict expected outcomes. While we have commenced dosing in our CLOVER-WaMpivotal clinical study of iopofosine in WM, we have experienced material delaysin patient recruitment and enrollment as a result of continued resourcing issuesrelated to COVID-19 at study sites and potentially due to concerns amongpatients about participating in clinical studies during a public healthemergency. The COVID-19 pandemic is also affecting the operations of thirdparties upon whom we rely. We are unable to predict how the COVID-19 pandemicmay affect our ability to successfully progress our CLOVER-WaM pivotal clinicalstudy or any other clinical programs in the future. Moreover, there remainsuncertainty relating to the trajectory of the pandemic and whether it may causefurther delays in patient study recruitment. The impact of related responses anddisruptions caused by the COVID-19 pandemic may result in difficulties or delaysin initiating, enrolling, conducting or completing our planned and ongoingstudies and the incurrence of unforeseen costs as a result of disruptions inclinical supply of iopofosine or preclinical study or clinical study delays andour ability to obtain additional financing. The continued impact of COVID-19 onresults will largely depend on future developments, which are highly uncertainand cannot be predicted with confidence, such as the ultimate geographic spreadof the disease or variants thereof, the duration of the pandemic, vaccinationrates, travel restrictions and social distancing in the United States, Canadaand other countries, business closures or business disruptions, the ultimateimpact on financial markets and the global economy, and the effectiveness ofactions taken in the United States, Canada and other countries to contain andtreat the disease. In October 2021, we announced that we are collaborating withBBK Worldwide to provide new concierge services for patients participating inour clinical studies. These services are designed to improve patient's and theircaregivers access to high quality care and innovative treatments for theircancer.
Our lead PDC therapeutic, iopofosine is a small-molecule PDC designed to providetargeted delivery of iodine-131 directly to cancer cells, while limitingexposure to healthy cells. We believe this profile differentiates iopofosinefrom many traditional on-market treatments. Iopofosine is currently beingevaluated in the CLOVER-WaM Phase 2 pivotal study in patients withrelapsed/refractory (r/r) Waldenstrom's macroglobulinemia (WM), a Phase 2B studyin r/r multiple myeloma (MM) patients and the CLOVER-2 Phase 1 study for avariety of pediatric cancers.
The CLOVER-1 Phase 2 study met the primary efficacy endpoints from the Part Adose-finding portion, conducted in r/r B-cell malignancies. The CLOVER-WaM Studyis a pivotal registration study currently evaluating iopofosine in Brutontyrosine kinase inhibitor (BTKi) failed or suboptimal response in WM. TheCLOVER-1 Phase 2B study is ongoing where iopofosine remains under furtherevaluation in highly refractory MM patients.
The CLOVER-2 Phase 1 pediatric study is an open-label, sequential-group,dose-escalation study to evaluate the safety and tolerability of iopofosine inchildren and adolescents with relapsed or refractory cancers, includingmalignant brain tumors, neuroblastoma, sarcomas, and lymphomas (includingHodgkin's lymphoma). The study is being conducted internationally at sevenleading pediatric cancer centers.
The U.S. Food and Drug Administration ("FDA") granted iopofosine Fast TrackDesignation for WM patients having received two or more prior treatmentregimens, as well as r/r MM and r/r diffuse large B-cell lymphoma (DLBCL).Orphan Drug Designations (ODDs) have been granted for WM, MM, neuroblastoma,rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. Iopofosine was also grantedRare Pediatric Disease Designation (RPDD) for the treatment of neuroblastoma,rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. The European Commissiongranted an ODDs for r/r MM and WM.
Our product pipeline also includes one preclinical PDC chemotherapeutic program(CLR 1900) and several partnered PDC assets. The CLR 1900 Series is beingtargeted for solid tumors with a payload that inhibits mitosis (cell division) avalidated pathway for treating cancers.
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We have leveraged our PDC platform to establish four ongoing collaborationsfeaturing four unique payloads and mechanisms of action. Through research anddevelopment collaborations, our strategy is to generate near-term capital,supplement internal resources, gain access to novel molecules or payloads,accelerate product candidate development and broaden our proprietary andpartnered product pipelines.
Our PDC platform provides selective delivery of a diverse range of oncologicpayloads to cancerous cells, whether a hematologic cancer or solid tumor, aprimary tumor, or a metastatic tumor and cancer stem cells. The PDC platform'smechanism of entry does not rely upon specific cell surface epitopes or antigensas are required by other targeted delivery platforms. Our PDC platform takesadvantage of a metabolic pathway utilized by all tumor cell types in all stagesof the tumor cycle. Tumor cells modify specific regions on the cell surface as aresult of the utilization of this metabolic pathway. Our PDCs bind to theseregions and directly enter the intracellular compartment. This mechanism allowsthe PDC molecules to accumulate in tumor cells over time, which can enhance drugefficacy, and to avoid the specialized highly acidic cellular compartment knownas lysosomes, which allows a PDC to deliver molecules that previously could notbe delivered. Additionally, molecules targeting specific cell surface epitopesface challenges in completely eliminating a tumor because the targeted antigensare limited in the total number on the cell surface, have longer cycling timefrom internalization to being present on the cell surface again and availablefor binding and are not present on all of the tumor cells in any cancer. Thismeans a subpopulation of tumor cells always exist that cannot be targeted bytherapies targeting specific surface epitopes. In addition to the benefitsprovided by the mechanism of entry, PDCs offer the ability to conjugate payloadmolecules in numerous ways, thereby increasing the types of moleculesselectively delivered via the PDC.
The PDC platform features include the capacity to link with almost any molecule,provide a significant increase in targeted oncologic payload delivery and theability to target all types of tumor cells. As a result, we believe that we cangenerate PDCs to treat a broad range of cancers with the potential to improvethe therapeutic index of oncologic drug payloads, enhance or maintain efficacywhile also reducing adverse events by minimizing drug delivery to healthy cells,and increasing delivery to cancerous cells and cancer stem cells.
We employ a drug discovery and development approach that allows us toefficiently design, research and advance drug candidates. Our iterative processallows us to rapidly and systematically produce multiple generations ofincrementally improved targeted drug candidates.
In June 2020, the European Medicines Agency (EMA) granted us Small andMedium-Sized Enterprise (SME) status by the EMA's Micro, Small and Medium-sizedEnterprise office. SME status allows us to participate in significant financialincentives that include a 90% to 100% EMA fee reduction for scientific advice,clinical study protocol design, endpoints and statistical considerations,quality inspections of facilities and fee waivers for selective EMA pre andpost-authorization regulatory filings, including orphan drug and PRIMEdesignations. We are also eligible to obtain EMA certification of quality andmanufacturing data prior to review of clinical data. Other financial incentivesinclude EMA-provided translational services of all regulatory documents requiredfor market authorization, further reducing the financial burden of the marketauthorization process.
A description of our PDC product candidates follows:
Our lead PDC therapeutic, iopofosine, is a small-molecule, PDC designed toprovide targeted delivery of iodine-131 directly to cancer cells, while limitingexposure to healthy cells. We believe this profile differentiates iopofosinefrom many traditional on-market treatments and treatments in development.Iopofosine is currently being evaluated in the CLOVER-WaM Phase 2 pivotal studyin patients with r/r WM, a Phase 2B study in r/r MM patients and the CLOVER-2Phase 1 study for a variety of pediatric cancers.
Iopofosine is currently being evaluated in a pivotal study, CLOVER-WaM, inWaldenstrom's macroglobulinemia (WM) patients that have failed or had asuboptimal response to a BTKi therapy after receiving first line standard ofcare. The CLOVER-1 Phase 2 study met the primary efficacy endpoints from thePart A dose-finding portion, conducted in r/r B-cell malignancies, and is nowenrolling a MM expansion cohort (Phase 2B). The Phase 2B study will evaluatehighly refractory MM patients including triple, quad and penta class refractorypatients. The initial Investigational New Drug (IND) application was accepted bythe FDA in March 2014 with multiple INDs submitted since that time. The Phase 1study was designed to assess the compound's safety and tolerability in patientswith r/r MM (to determine maximum tolerated dose (MTD) and was initiated inApril 2015. The study completed enrollment and the final clinical study reportis expected in the second half of 2021. Initiated in March 2017, the primarygoal of the Phase 2A study was to assess the compound's efficacy in a broadrange of hematologic cancers.
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The CLOVER-2 Phase 1 pediatric study is being conducted internationally at sevenleading pediatric cancer centers. The study is an open-label, sequential-group,dose-escalation study to evaluate the safety and tolerability of iopofosine inchildren and adolescents with relapsed or refractory cancers, includingmalignant brain tumors, neuroblastoma, sarcomas, and lymphomas (includingHodgkin's lymphoma). The FDA previously accepted our IND application for a Phase1 open-label, dose escalating study to evaluate the safety and tolerability of asingle intravenous administration of iopofosine in up to 30 children andadolescents with cancers including neuroblastoma, sarcomas, lymphomas (includingHodgkin's lymphoma) and malignant brain tumors. This study was initiated duringthe first quarter of 2019. These cancer types were selected for clinical,regulatory and commercial rationales, including the radiosensitive nature andcontinued unmet medical need in the r/r setting, and the rare diseasedeterminations made by the FDA based upon the current definition within theOrphan Drug Act.
In December 2014, the FDA granted ODD for iopofosine for the treatment of MM. In2018, the FDA granted ODD and RPDD for iopofosine for the treatment ofneuroblastoma, rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. In May 2019,the FDA granted Fast Track designation for iopofosine for the treatment of MMand in July 2019 for the treatment of DLBCL, in September 2019 iopofosinereceived Orphan Drug Designation from the European Union for Multiple Myeloma,in January 2020, the FDA granted Orphan Drug Designation for iopofosineWaldenstrom's macroglobulinemia and the European Union granted Orphan DrugDesignation for iopofosine Waldenstrom's macroglobulinemia. The FDA granted FastTrack designation for iopofosine for the treatment of WM in May 2020.
As the result of iopofosine's RPDD designation, we may be eligible to receive apriority review voucher (PRV) if the product receives approval for any of thetreatment of neuroblastoma, rhabdomyosarcoma, Ewing's sarcoma or osteosarcoma.The FDA may award PRV to sponsors of a RPDD that meet its specified criteria.The key criteria to receiving a priority review voucher is that the diseasebeing treated is life-threatening and that it primarily effects individualsunder the age of 18. Under this program, a sponsor who receives an approval fora drug or biologic for a rare pediatric disease can receive a PRV that can beredeemed to receive a priority review of a subsequent marketing application fora different product. Additionally, the PRV's can be exchanged or sold to othercompanies so that the receiving company may use the voucher.
CLOVER-WaM: Phase 2 Study Pivotal Study in: Patients with r/r Waldenstrom'sMacroglobulinemia
In January 2021, we announced that a Type C guidance meeting with the FDA wasconducted in September of 2020. The results of that guidance meeting providedCellectar with an agreed upon path for conducting the CLOVER-WaM study; singlearm, pivotal study in Waldenstrom's macroglobulinemia patients that havereceived at least two prior lines of therapy and either failed or had asuboptimal response to BTKi therapy. The FDA agreed with the dose to be tested,our proposal for a safety and futility assessment to be conducted on the first10 patients, the endpoint to be assessed, the statistical analysis plan andstudy size of 50 patients. Based upon this agreement the pivotal study wasinitiated. WM is a rare, indolent and incurable form of non-Hodgkin's lymphoma(NHL) that is composed of a patient population in need of new and bettertreatment options.
Phase 2A Study: Patients with r/r Waldenstrom's Macroglobulinemia Cohort
Current data from our Phase 2A CLOVER-1 clinical study show that six WM patientsdemonstrated 100% overall response rate (ORR) and an 83.3% major response ratewith one patient achieving a complete response (CR), which continues at nearly27 months post- last treatment. While median treatment free survival ((TRS) alsoknown as treatment free remission (TFR)) and duration of response (DOR) has notbeen reached, the average treatment TFS/TFR is currently at 330 days. This mayrepresent an important improvement in the treatment of relapsed/refractory WM aswe believe no approved or late-stage development treatments for second- andthird-line patients have reported a CR to date.
Phase 2A Study: Patients with r/r Multiple Myeloma Cohort
In September 2020, we announced that a 40% ORR was observed in the subset ofrefractory multiple myeloma patients deemed triple class refractory who received60 mCi or greater total body dose (TBD). Triple class refractory is defined aspatients that are refractory to immunomodulatory, proteasome inhibitors andanti-CD38 antibody drug classes. The 40% ORR (6/15 patients) represents tripleclass refractory patients enrolled in Part A of Cellectar's CLOVER-1 study andadditional patients enrolled in Part B from March through May 2020 and received?60mCi TBD. All MM patients enrolled in the expansion cohort are required to betriple class refractory. The additional six patients enrolled in 2020 wereheavily pre-treated with an average of nine prior multi-drug regimens. Threepatients received a total body dose of ?60 mCi and three received less than 60mCi. Consistent with the data released in February 2020, patients receiving ?60mCi typically exhibit greater responses. Based on study results to date,patients continue to tolerate iopofosine well, with the most common and almostexclusive treatment emergent adverse events being cytopenias.
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Phase 2A: Patients with r/r non-Hodgkin's lymphoma Cohort
In February 2020, we announced positive data from our Phase 2a CLOVER-1 study inpatients with relapsed/refractory non-Hodgkin lymphoma (NHL) patients weretreated with three different doses (<50mCi, ~50mCi and ?60mCi TBD. Patients withr/r NHL who received <60mCi TBD and the ?60mCi TBD had a 42% and 43% ORR,respectively and a combined rate of 42%. These patients were also heavilypre-treated, having a median of three prior lines of treatment (range, 1 to 9)with the majority of patients being refractory to rituximab and/or ibrutinib.The patients had a median age of 70 with a range of 51 to 86. All patients hadbone marrow involvement with an average of 23%. In addition to these findings,subtype assessments were completed in the r/r B-cell NHL patients. Patients withDLBCL demonstrated a 30% ORR with one patient achieving a CR, which continues atnearly 24 months post-treatment. The ORR for chronic lymphocytic leukemia/smalllymphocytic lymphoma (CLL/SLL) and marginal zone lymphoma (MZL) patients was33%.
Based upon the dose response observed in the Phase 2A for patients receivingtotal body doses of 60mCi or greater, we determined that patient dosing ofiopofosine would be >60mCi TBD. Therefore, patients are now grouped as receiving<60mCi or >60mCi TBD.
The most frequently reported adverse events in all patients were cytopenias,which followed a predictable course and timeline. The frequency of adverseevents have not increased as doses were increased and the profile of cytopeniasremains consistent. Importantly, these cytopenias have had a predictable patternto initiation, nadir and recovery and are treatable. The most common grade ?3events at the highest dose (75mCi TBD) were hematologic toxicities includingthrombocytopenia (65%), neutropenia (41%), leukopenia (30%), anemia (24%) andlymphopenia (35%). No patients experienced cardiotoxicities, neurologicaltoxicities, infusion site reactions, peripheral neuropathy, allergic reactions,cytokine release syndrome, keratopathy, renal toxicities, or changes in liverenzymes. The safety and tolerability profile in patients with r/r NHL wassimilar to r/r MM patients except for fewer cytopenias of any grade. Based uponiopofosine being well tolerated across all dose groups and the observed responserate, especially in difficult to treat patients such as high risk and tripleclass refractory or penta-refractory, and corroborating data showing thepotential to further improve upon current ORRs and durability of thoseresponses, the study has been expanded to test a two-cycle dosing optimizationregimen with a target total body dose >60 mCi/m2 of iopofosine.
In July 2016, we were awarded a $2,000,000 National Cancer Institute (NCI)Fast-Track Small Business Innovation Research grant to further advance theclinical development of iopofosine. The funds supported the Phase 2 studyinitiated in March 2017 to define the clinical benefits of iopofosine in r/r MMand other niche hematologic malignancies with unmet clinical need. These nichehematologic malignancies include Chronic Lymphocytic Leukemia, Small LymphocyticLymphoma, Marginal Zone Lymphoma, Lymphoplasmacytic Lymphoma/WM and DLBCL. Thestudy is being conducted in approximately 10 U.S. cancer centers in patientswith orphan-designated relapse or refractory hematologic cancers. The study'sprimary endpoint is clinical benefit response (CBR), with secondary endpoints ofORR, progression free survival (PFS,) median Overall Survival (mOS) and othermarkers of efficacy following patients receiving one of three TBDs of iopofosine(<50mCi, ~50mCi and >60mCi), with the option for a second cycle approximately75-180 days later. Dosages were provided either as a single bolus orfractionated (the assigned dose level split into two doses) given day 1 and day15.
In May 2020, we announced that the FDA granted Fast Track Designation foriopofosine in WM in patients having received two prior treatment regimens ormore.
Phase 1 Study in Patients with r/r Multiple Myeloma
In February 2020, we announced the successful completion of our Phase 1 doseescalation study. Data from the study demonstrated that iopofosine was safe andtolerated up to a total body dose of approximately 95mCi in r/r MM. The Phase 1multicenter, open-label, dose-escalation study was designed to evaluate thesafety and tolerability of iopofosine administered in an up to 30-minute I.V.infusion, either as a single bolus dose or as fractionated doses. The r/rmultiple myeloma patients in this study received single cycle doses ranging fromapproximately 20mCi to 95mCi total body dose. An independent Data MonitoringCommittee determined that all doses used were safe and well-tolerated bypatients.
Iopofosine in combination with dexamethasone was under investigation in adultpatients with r/r MM. Patients had to be refractory to or relapsed from at leastone proteasome inhibitor and at least one immunomodulatory agent. The clinicalstudy was a standard three-plus-three dose escalation safety study to determinethe maximum tolerable dose. Multiple myeloma is an incurable cancer of theplasma cells and is the second most common form of hematologic cancers.Secondary objectives included the evaluation of therapeutic activity byassessing surrogate efficacy markers, which include M protein, free light chain(FLC), PFS and OS. All patients were heavily pretreated with an average of fiveprior lines of therapy. Iopofosine was deemed by an Independent Data MonitoringCommittee (IDMC) to be safe and tolerable up to its planned maximum single,bolus dose of 31.25 mCi/m2 or a total body dose of ~63 mCi. The four single dosecohorts examined were: 12.5 mCi/m2 (~25mCi TBD), 18.75 mCi/m2 (~37.5mCi TBD), 25mCi/m2(~50mCi TBD), and 31.25 mCi/m2(~62.5mCi TBD), all in combination with lowdose dexamethasone (40 mg weekly). Of the five patients in the first cohort,four achieved stable disease and one patient progressed at Day 15 afteradministration and was taken off the study. Of the five patients admitted to thesecond cohort, all five achieved stable disease however one patient progressedat Day 41 after administration and was taken off the study. Four patients wereenrolled to the third cohort and all achieved stable disease. In September 2017,we announced results for cohort 4, showing that a single infusion up to30-minutes of 31.25mCi/m2 of iopofosine was safe and tolerated by the threepatients in the cohort. Additionally, all three patients experienced CBR withone patient achieving a partial response (PR). We use the International MyelomaWorking Group (IMWG) definitions of response, which involve monitoring thesurrogate markers of efficacy, M protein and FLC. The IMWG defines a PR as agreater than or equal to 50% decrease in FLC levels (for patients in whom Mprotein is unmeasurable) or 50% or greater decrease in M protein. The patientexperiencing a PR had an 82% reduction in FLC. This patient did not produce Mprotein, had received seven prior lines of treatment including radiation, stemcell transplantation and multiple triple combination treatments including onewith daratumumab that was not tolerated. One patient experiencing stable diseaseattained a 44% reduction in M protein. In January 2019, we announced that thepooled mOS data from the first four cohorts was 22.0 months. In late 2018, wemodified this study to evaluate a fractionated dosing strategy to potentiallyincrease efficacy and decrease adverse events.
Cohort 5 and 6 were fractionated cohorts of 31.25 mCi/m2(~62.5mCi TBD) and 37.5mCi/m2(~75mCi TBD), each administered on day 1 and on day 8. Following thedetermination that all prior dosing cohorts were safe and tolerated, weinitiated a cohort 7 utilizing a 40mCi/m2 (~95mCi TBD) fractionated doseadministered 20mCi/m2 (~40mCi TBD) on days 1 and day 8. Cohort 7 was the highestpre-planned dose cohort and subjects have completed the evaluation period. Thestudy completed enrollment and the final clinical study report is expected inthe first half of 2021.
In May 2019, we announced that the FDA granted Fast Track Designation foriopofosine in fourth line or later r/r MM. Iopofosine is our small moleculeradiotherapeutic PDC designed to deliver cytotoxic radiation directly andselectively to cancer cells and cancer stem cells. It is currently beingevaluated in our ongoing CLOVER-1 Phase 2 clinical study in patients withrelapsed or refractory multiple myeloma and other select B-cell lymphomas.
Phase 1 Study in r/r Pediatric Patients with select Solid tumors, Lymphomas andMalignant Brain Tumors
In December 2017 the Division of Oncology at the FDA accepted our IND and studydesign for the Phase 1 study of iopofosine in children and adolescents withselect rare and orphan designated cancers. This study was initiated during thefirst quarter of 2019. In December 2017, we filed an IND application for r/rpediatric patients with select solid tumors, lymphomas and malignant braintumors. The Phase 1 clinical study of iopofosine is an open-label,sequential-group, dose-escalation study evaluating the safety and tolerabilityof intravenous administration of iopofosine in children and adolescents withcancers including neuroblastoma, sarcomas, lymphomas (including Hodgkin'slymphoma) and malignant brain tumors. Secondary objectives of the study are toidentify the recommended efficacious dose of iopofosine and to determinepreliminary antitumor activity (treatment response) of iopofosine in childrenand adolescents. In August 2020, it was announced that four dose levels 15mCi/m2up to 60mCi/m2 were deemed safe and tolerable by an independent Data MonitoringCommittee and evaluation of the next higher dose cohort, 75mCi/m2 was initiated.In November 2020, we announced that iopofosine had been measured in tumors,confirming that systemic administration of iopofosine crosses the blood brainbarrier and is delivered into tumors and that disease control has been exhibitedin heavily pretreated patients with ependymomas. In 2018, the FDA granted ODDand RPDD for iopofosine for the treatment of neuroblastoma, rhabdomyosarcoma,Ewing's sarcoma and osteosarcoma. Should iopofosine be approved for any of thesepediatric indications, the first approved RPDD would enable us to receive apriority review voucher. Priority review vouchers can be used by the sponsor toreceive priority review for a future New Drug Application ("NDA") or BiologicLicense Application ("BLA") submission, which would reduce the FDA review timefrom 12 months to six months. Currently, these vouchers can also be transferredor sold to another entity. In December 2020, the FDA extended the PriorityReview Voucher Program through September 2026 for rare pediatric diseases.
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Phase 1 Study in r/r Head and Neck Cancer
In August 2016, the University of Wisconsin Carbone Cancer Center ("UWCCC") wasawarded a five-year Specialized Programs of Research Excellence ("SPORE") grantof $12,000,000 from the National Cancer Institute and the National Institute ofDental and Craniofacial Research to improve treatments and outcomes for head andneck cancer, HNC, patients. HNC is the sixth most common cancer across the worldwith approximately 56,000 new patients diagnosed every year in the U.S. As a keycomponent of this grant, the UWCCC researchers completed testing of iopofosinein various animal HNC models and initiated the first human clinical studyenrolling up to 30 patients combining iopofosine and external beam radiation(EBRT) with recurrent HNC in Q4 2019. UWCCC has completed the part A portion ofa safety and tolerability study of iopofosine in combination with EBRT andpreliminary data suggest safety and tolerability in relapsed or refractory headand neck cancer. The reduction in the amount or fractions (doses) of EBRT hasthe potential to diminish the (number and severity of) adverse events associatedwith EBRT. Patients with head and neck cancer typically receive approximately60-70 Grays (Gy) of EBRT given as 2 - 3 Gy daily doses over a 6-week timeframe.Patients can experience long-term tumor control following re-irradiation in thissetting; however, this approach can cause severe injury to normal tissuestructures, significant adverse events and diminished quality of life. Part B ofthe study will further assess the safety and potential benefits of iopofosine incombination with EBRT in a cohort of up to 24 patients.
We believe our PDC platform has potential to provide targeted delivery of adiverse range of oncologic payloads, as exemplified by the product candidateslisted below, that may result in improvements upon current standard of care("SOC") for the treatment of a broad range of human cancers:
CLR 1900 Series is an internally developed proprietary PDC program leveraging a
novel small molecule cytotoxic compound as the payload. The payload inhibits
mitosis (cell division) and targets a key pathway required to inhibit rapidly
? dividing cells that results in apoptosis. We believe that this program could
produce a product candidate targeted to select solid tumors. Currently, the
program is in early preclinical development and if we elect to progress any
molecules further, we will select preferred candidates.
CLR 2000 Series is a collaborative PDC program with Avicenna Oncology, or
Avicenna, that we entered into in July 2017. Avicenna is a developer of
antibody drug conjugates ("ADCs"). The objective of the research collaboration
is to design and develop a series of PDCs utilizing Avicenna's proprietary
cytotoxic payload. Although Avicenna is a developer of ADCs, this collaboration
? was sought as a means to overcome many of the challenges associated with ADCs,
including those associated with the targeting of specific cell surface
epitopes. The CLR 2000 Series has demonstrated improved safety, efficacy and
tissue distribution with the cytotoxic payload in animal models. A candidate
molecule and a back-up have been selected for further advancement at a future
CLR 12120 Series is a collaborative PDC program with Orano Med for the
? development of novel PDCs utilizing Orano Med's unique alpha emitter, lead 212
conjugated to our phospholipid ether; the companies intend to evaluate the new
PDCs in up to three oncology indications.
Expanded ongoing collaboration with biotechnology company IntoCell Inc.,
? combining their novel linker chemistry with our validated targeting platform to
create novel next generation phospholipid drug conjugate therapeutics.
Co-development and commercialization collaboration with LegoChemBio, a clinical
? stage biotechnology company to utilize their proprietary drug conjugate
linker-toxin platform to further enhance our portfolio of next generation PDC
Research and development expense. Research and development expense consist ofcosts incurred in identifying, developing and testing, and manufacturing productcandidates, which primarily include salaries and related expenses for personnel,cost of manufacturing materials and contract manufacturing fees paid to contractmanufacturers and contract research organizations, fees paid to medicalinstitutions for clinical studies, and costs to secure intellectual property.The Company analyzes its research and development expenses based on fourcategories as follows: clinical project costs, preclinical project costs,manufacturing and related costs, and general research and development costs thatare not allocated to the functional project costs, including personnel costs,facility costs, related overhead costs and patent costs.
General and administrative expense. General and administrative expense consistsprimarily of salaries and other related costs for personnel in executive,finance and administrative functions. Other costs include insurance, costs forpublic company activities, investor relations, directors' fees and professionalfees for legal and accounting services.
Three Months Ended September 30, 2021 and 2020
Research and Development. Research and development expense for the three monthsended September 30, 2021 was approximately $3,937,000 compared to approximately$2,684,000 for the three months ended September 30, 2020.
General research and development costs 1,185,000 809,000 376,000
The overall increase in research and development expense of $1,253,000, or 47%,was primarily a result of an increase related to clinical project costs ofapproximately $1,058,000. General research and development costs increased dueto an increase in personnel slightly offset by a decrease in manufacturing andrelated costs and pre-clinical project costs.
General and administrative. General and administrative expense for the threemonths ended September 30, 2021 was approximately $1,882,000, compared toapproximately $1,226,000. The overall increase in general and administrativeexpense of $656,000, or 54% was primarily a result of an increase inprofessional fees and stock-based compensation expense.
Nine Months Ended September 30, 2021 and 2020
Research and Development. Research and development expense for the nine monthsended September 30, 2021 was approximately $13,198,000 compared to approximately$7,766,000 for the nine months ended September 30, 2020.
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General research and development costs 3,288,000 2,589,000 699,000
The overall increase in research and development expense of $5,432,000, or 70%,was primarily a result of an increase related to start-up costs for our WMpivotal study and clinical project costs of approximately $5,171,000 and generalresearch and development costs of approximately $699,000 offset by a decrease inmanufacturing and related costs.
General and administrative. General and administrative expense for the ninemonths ended September 30, 2021 was approximately $5,010,000, compared toapproximately $3,725,000. The overall increase in general and administrativeexpense of $1,285,000, or 34%, was primarily a result of an increase inprofessional fees and insurance, personnel costs and stock-based compensationexpense.
Liquidity and Capital Resources
As of September 30, 2021, we had cash and cash equivalents of approximately$40,345,000 compared to $57,165,000 as of December 31, 2020. This decrease wasdue primarily a result of research and development expense and general andadministrative expenses. Net cash used in operating activities during the ninemonths ended September 30, 2021 was approximately $18,058,000.
Our cash requirements have historically been for our research and developmentactivities, finance and administrative costs, capital expenditures and overallworking capital. We have experienced negative operating cash flows sinceinception and have funded our operations primarily from sales of common stockand other securities. As of September 30, 2021, we had an accumulated deficit ofapproximately $144,980,000.
We believe that the cash balance is adequate to fund our basic budgetedoperations for at least 12 months from the filing of these financial statements.However, our future results of operations involve significant risks anduncertainties. Our ability to execute our operating plan beyond that timedepends on our ability to obtain additional funding via the sale of equityand/or debt securities, a strategic transaction or otherwise. We plan toactively pursue all available financing alternatives; however, there can be noassurance that we will obtain the necessary funding. Other than theuncertainties regarding our ability to obtain additional funding, there arecurrently no known trends, demands, commitments, events or uncertainties thatare likely to materially affect our liquidity.
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