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Myriad Genetics Announces Results from a Large 2,000 Patient Clinical Utility Study of Its myRisk® Hereditary Cancer Test at the 53rd Annual Meeting of ASCO

On June 2, 2017 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported results from Study 005, a large 2,000 patient prospective study of the Myriad myRisk Hereditary Cancer test, which will be featured in three poster presentations at the 53rd annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (Press release, Myriad Genetics, JUN 2, 2017, View Source [SID1234519349]).

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The data will be presented by research collaborators from University of Southern California (USC) Norris Comprehensive Cancer Center and Stanford University Cancer Institute. The key findings are that more than 50 percent of the mutations identified were in patients who would not meet current testing guidelines and 34 percent of mutations were identified in unexpected genes, confirming the clinical utility of multi-gene panel testing to improve hereditary cancer-risk assessment.

"We are very excited to present new data on our myRisk Hereditary Cancer test which shows our ongoing commitment to collaborate with leading academic centers and advance the field of hereditary cancer testing," said Johnathan Lancaster, M.D., Ph.D., chief medical officer, Myriad Genetic Laboratories. "Importantly this study demonstrates that more than half of mutations would be missed with current testing guidelines and 34 percent of mutations identified were unexpected and not predicted by personal and/or family history. This study will provide vital data to facilitate review of medical guidelines in light of advances made in next generation sequencing."

The data are highlighted below and abstracts are available at: abstracts.asco.org.

myRisk Hereditary Cancer Poster Presentations
Title: Performance of Mutation Risk Prediction Models in a Racially Diverse Multi-Gene Panel Testing Cohort.
Presenter: Gregory Idos, M.D., USC Norris Comprehensive Cancer Center.
Date: Monday, June 5, 2017, 1:15 — 4:45 p.m.
Location: Poster 181; Abstract 1523

National Comprehensive Cancer Network (NCCN) guidelines recommend germline genetic testing for patients with a mutation carrier probability (CP) score of five percent or higher. An analysis of data from Study 005 evaluated the percentage of pathogenic mutations in a population of racially diverse patients with a CP score less than five percent. In total 2,000 patients were tested using the myRisk Hereditary Cancer test and 242 were found to have pathogenic mutations. The results showed that more than 50 percent of patients with BRCA1/2 or mismatch repair (MMR) mutations had a CP score less than five percent. Four percent of patients with BRCA1/2 mutations did not meet NCCN guidelines for hereditary breast and ovarian cancer syndrome, and 13 percent of patients with MMR mutations did not meet NCCN criteria for Lynch syndrome testing. Importantly, these findings support lowering the guideline-recommended CP threshold for genetic testing to help ensure that more patients can access genetic testing.

Title: Yield of multiplex panel testing exceeds expert opinion and validated prediction models.
Presenter: Gregory Idos, M.D., USC Norris Comprehensive Cancer Center.
Date: Monday, June 5, 2017, 1:15 — 4:45 p.m.
Location: Poster 183; Abstract 1525.

Data from Study 005 were evaluated to determine the diagnostic yield and clinical utility of panel testing using the myRisk Hereditary Cancer 28-gene test in 2,000 patients undergoing hereditary cancer-risk assessment. Approximately 81 percent of patients were women and 40 percent were Hispanic. Differential diagnoses were generated after standard clinical genetics assessment and before genetic testing. Differences between the differential diagnoses and genetic testing results were evaluated to determine the added diagnostic yield of multi-gene panel testing. The results show that 12.1 percent of patients tested positive for a pathogenic mutation. The most common mutations were in BRCA1 (17 percent) and BRCA2 (15 percent), APC (8 percent), CHEK2 (7 percent) and ATM (7 percent). Importantly, however, 34 percent of the mutations were not clinically suspected before genetic testing, which demonstrates the significant added value of the myRisk Hereditary Cancer test in hereditary cancer-risk assessment.

Title: Yield of multiplex panel testing exceeds expert opinion and validated prediction models.
Presenter: Allison Kurian, M.D., Stanford University Cancer Institute.
Date: Monday, June 5, 2017, 1:15 — 4:45 p.m.
Location: Poster 234; Abstract 1576.

Study 005 also evaluated the safety of gene panel testing among patients who were undergoing cancer-risk assessment with the myRisk Hereditary Cancer test. The analysis of 2,000 patients found that 12.1 percent of patients had pathogenic mutations. Overall, self-reported preventative surgery rates were low (mastectomy 9.2 percent, hysterectomy 1.6 percent, and oophorectomy 1.8 percent). There was no difference in preventative surgery rates between patients with a variant of uncertain significance (VUS) and mutation negative patients (p=0.21). Importantly, most patients never/rarely had thoughts of cancer affecting their daily activities, did not regret genetic testing and wanted to know all the results. Patients with a pathogenic mutation reported higher distress and uncertainty scores than VUS or negative patients, whose distress (p=0.06) and uncertainty (p=0.04) scores were similar. Relatives of mutation positive patients completed genetic testing more often than VUS or negative patients. This study demonstrated that multi-gene panel testing did not result in inappropriate medical management or increased distress/uncertainty among VUS and negative patients.

"The use of multi-gene panels for the clinical assessment of hereditary cancer risk is rapidly increasing in the era of personalized medicine and it’s important that we understand the benefits and risks of genetic testing on patients," said Lancaster. "Study 005 showed that multi-gene panel testing effectively improved hereditary cancer risk assessment and the results did not lead to unwarranted treatment or adverse effects."

About Myriad myRisk Hereditary Cancer
The Myriad myRisk Hereditary Cancer test uses an extensive number of sophisticated technologies and proprietary algorithms to evaluate 28 clinically significant genes associated with eight hereditary cancer sites including: breast, colon, ovarian, endometrial, pancreatic, prostate and gastric cancers and melanoma. The myRisk Hereditary Cancer test offers physicians several distinct advantages over other commercial tests, including:

Unsurpassed lab accuracy:
85,000 base pairs with ~100 percent accuracy.
856 steps using 23 major technology platforms.
100 proprietary software applications.

Industry leading variant classification:
More than 20 years of investment in research.
>2.5 million patients tested; 50,000 variants identified.
Five proprietary methods with 99.5 percent validity.

Exceptional customer service:
More than 40,000 ordering physicians annually.
450 field educators.
Extensive reimbursement support.
Lifetime commitment to patients.

Innate Pharma strengthens its proprietary pipeline with the acquisition of anti-C5aR, a first-in-class clinical-stage antibody,from novo Nordisk A/S

On June 2, 2017 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH) reported that it enters into an agreement with Novo Nordisk A/S granting Innate Pharma full worldwide exclusive rights to develop and commercialize a first-in-class clinical-stage anti-C5aR antibody (IPH5401) representing a novel therapeutic approach in immuno-oncology (Press release, Innate Pharma, JUN 2, 2017, View Source [SID1234519348]).

IPH5401 is a strategic fit to Innate Pharma’s current immuno-oncology pipeline. It adds a clinical-stage proprietary product that reinforces Innate Pharma’s position in the field of antibodies targeting the tumor microenvironment beyond the Company’s activities in the adenosine pathway. Innate Pharma plans to start trials with IPH5401 in oncology in 2018.

Through C5aR triggering, C5a induces accumulation and activation of myeloid-derived suppressor cells (MDSC) and neutrophils in the tumor microenvironment. These cells are associated with a poor prognosis across numerous tumor types. They secrete pro-tumor and pro-angiogenic factors and have emerged as a major immunosuppressive mechanism, associated with resistance to checkpoint blockers. By targeting and blocking this pathway, anti-C5aR has the potential to enhance anti-tumor immunity across a range of solid and hematologic tumors.

Novo Nordisk A/S has conducted two Phase I trials with anti-C5aR in patients with rheumatoid arthritis, where a good safety profile was demonstrated.

The terms of the transaction provide for a total upfront payment of €40m, of which €37.2m will be paid in new Innate Pharma shares and €2.8m in cash. Novo Nordisk A/S will be eligible for €370m in development, regulatory and sales milestone payments. Novo Nordisk A/S will also be eligible for double digit royalties on net sales.

After the issuance of the new Innate Pharma shares, the stake of Novo Nordisk A/S in Innate Pharma will increase from 10.3% currently to between 14.6% to 15.8% (See "About the transaction section").

The closing is expected to take place on July 12, 2017 at the latest.

Mondher Mahjoubi, Chief Executive Officer of Innate Pharma, commented: "With the acquisition of the first-in-class anti-C5aR antibody, we are broadening our proprietary clinical pipeline. We believe anti-C5aR has a high potential for cancer patients in multiple indications and look forward to beginning clinical development of this promising asset in 2018. Innate Pharma has a strong track record of value creation from in-licensed assets with lirilumab and monalizumab. This acquisition strengthens our asset base further and supports Innate Pharma’s transition towards becoming a fully-integrated biopharmaceutical company."

Mads Krogsgaard Thomsen, Chief Science Officer of Novo Nordisk A/S, added: "In light of Innate’s success within the immuno-oncology field, we believe that Innate is the ideal partner for the anti-C5aR program and we are looking forward to seeing the program advance further in development."

Novartis presents updated data that reinforce the efficacy and safety of Kisqali® (ribociclib) plus letrozole as a first-line option for HR+/HER2- advanced or metastatic breast cancer

On June 2, 2017 – Novartis reported updated findings from the Phase III MONALEESA-2 study that reinforce the efficacy and safety of Kisqali (ribociclib) plus letrozole in postmenopausal women with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced or metastatic breast cancer (Press release, Novartis, JUN 2, 2017, View Source [SID1234519346]). After an additional 11 months of follow-up, a median progression-free survival (PFS) of 25.3 months (95% CI: 23.0-30.3) for Kisqali plus letrozole and 16.0 months (95% CI: 13.4-18.2) for letrozole alone was observed (HR=0.568 (95% CI: 0.457-0.704; p<0.0001))[1]. These data will be presented on Sunday, June 4, 2017 at the 53rd Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago (Abstract #1038).

These updated results further support that Kisqali plus letrozole improves PFS as a first-line treatment across all patient subgroups[1]. After two years of treatment, the progression-free survival rate was 54.7% in the Kisqali plus letrozole arm compared to 35.9% in patients treated with letrozole alone[1]. In a cohort of 213 US patients treated as part of MONALEESA-2, the median PFS was 27.6 months with Kisqali plus letrozole and 15.0 months with letrozole alone (HR=0.527 (95% CI: 0.351-0.793))[1].

Treatment benefit remained consistent across all patient subgroups regardless of demographics or disease characteristics, including women with visceral disease and those diagnosed de novo[1]. In women with measurable disease at baseline, 55% saw their tumor size shrink by at least 30% (overall response rate (ORR)) compared to 39% with letrozole plus placebo[1]. Follow-up to measure overall survival is ongoing as data remain immature[1].

"This new look at the MONALEESA-2 data, after an additional year of follow-up, demonstrates the continued efficacy of ribociclib plus letrozole," said Gabriel N. Hortobagyi, MD, Professor of Medicine, Department of Breast Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center and MONALEESA-2 Principal Investigator. "With more than two years of follow-up, the PFS data confirm the inclusion of ribociclib plus an aromatase inhibitor as a strong option among first-line treatments for HR-positive, HER2-negative advanced breast cancer."

A separate analysis of patient-reported, health-related quality of life (HRQoL) outcomes from the MONALEESA-2 trial presented at ASCO (Free ASCO Whitepaper) (Abstract #1020) showed no significant difference in quality of life for women taking Kisqali plus letrozole compared to those taking letrozole alone[2]. This suggests that adverse events did not significantly impact HRQoL[2].

Updated safety data from the MONALEESA-2 trial show the safety profile of Kisqali plus letrozole remained consistent and the incidence of laboratory and electrocardiogram (ECG) irregularities is similar to that observed at the first interim analysis[1]. At the time of this updated analysis, the most common (>=10%) grade 3/4 laboratory abnormalities were as follows for Kisqali plus letrozole compared to letrozole alone: decreased neutrophils (62.6% vs 1.5%), decreased leukocytes (36.8% vs 1.5%), decreased lymphocytes (16.2% vs 3.9%) and elevated alanine aminotransferase (11.4% vs 1.2%)[1].

"Updated MONALEESA-2 results validate the sustained efficacy and established safety profile of Kisqali plus letrozole in patients with HR+/HER2- metastatic breast cancer and confirm the data that supported its recent FDA approval," said Vas Narasimhan, MD, Head, Global Drug Development and Chief Medical Officer, Novartis. "We are excited about the potential of Kisqali, and are continuing to evaluate its activity in several Phase III trials with multiple hormonal therapy combinations across a broad range of patient populations, including in the adjuvant setting. We look forward to sharing new results with the scientific community in the coming months and years."

The MONALEESA clinical trial program includes two additional Phase III trials in advanced breast cancer, MONALEESA-3 and MONALEESA-7, which are evaluating the efficacy and safety of Kisqali in combination with other endocrine partners. Novartis also is enrolling patients in a study to further evaluate Kisqali in men and pre- or postmenopausal women (CompLEEment-1) and initiating Phase III trials evaluating Kisqali in the adjuvant therapy setting (EarLEE-1 and EarLEE-2).

Kisqali was approved by the US Food and Drug Administration (FDA) on March 13, 2017, as a first-line treatment for HR+/HER2- metastatic breast cancer in combination with any aromatase inhibitor.

Full US prescribing information for Kisqali can be found at View Source

About Kisqali (ribociclib)
Kisqali (ribociclib) is a selective cyclin-dependent kinase inhibitor, a class of drugs that help slow the progression of cancer by inhibiting two proteins called cyclin-dependent kinase 4 and 6 (CDK4/6). These proteins, when over-activated, can enable cancer cells to grow and divide too quickly. Targeting CDK4/6 with enhanced precision may play a role in ensuring that cancer cells do not continue to replicate uncontrollably.

Kisqali was developed by the Novartis Institutes for BioMedical Research (NIBR) under a research collaboration with Astex Pharmaceuticals.

About the Kisqali Clinical Trial Program
MONALEESA-3 is evaluating Kisqali in combination with fulvestrant compared to fulvestrant alone in postmenopausal women with HR+/HER2- advanced breast cancer who have received no or a maximum of one prior endocrine therapy. MONALEESA-7 is investigating Kisqali in combination with endocrine therapy and goserelin compared to endocrine therapy and goserelin alone in premenopausal women with HR+/HER2- advanced breast cancer who have not previously received endocrine therapy. These trials are fully enrolled.

Novartis will initiate two multicenter, randomized, double-blind Phase III clinical trials, EarLEE-1 and EarLEE-2, to evaluate the safety and efficacy of Kisqali with endocrine therapy as adjuvant therapy in pre- and postmenopausal women who have not previously received treatment with CDK4/6. EarLEE-1 will assess Kisqali with adjuvant endocrine therapy compared to adjuvant endocrine therapy alone in women with HR+/HER2- high-risk early breast cancer. EarLEE-2 will investigate Kisqali with adjuvant endocrine therapy compared to adjuvant endocrine therapy alone in women with HR+/HER2- intermediate-risk early breast cancer.

The CompLEEment study is evaluating the safety and efficacy of Kisqali plus letrozole in men and pre- or postmenopausal women with HR+/HER2- advanced breast cancer with no prior hormonal therapy for advanced disease. This open-label, multicenter, Phase IIIb CompLEEment-1 trial is currently enrolling participants.

More information about these studies can be found at ClinicalTrials.gov.About Novartis in Advanced Breast Cancer
For more than 25 years, Novartis has been at the forefront of driving scientific advancements for breast cancer patients and improving clinical practice in collaboration with the global community. With one of the most diverse breast cancer pipelines and the largest number of breast cancer compounds in development, Novartis leads the industry in discovery of new therapies and combinations, especially in HR+ advanced breast cancer, the most common form of the disease.

Kisqali (ribociclib) Important US Safety Information
Kisqali (ribociclib) is a prescription medicine used in combination with an aromatase inhibitor as the first hormonal-based therapy to treat women who have gone through menopause with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer. It is not known if Kisqali is safe and effective in children. Kisqali can cause a heart problem known as QT prolongation. This condition can cause an abnormal heartbeat and may lead to death. Patients should tell their health care provider right away if they have a change in their heartbeat (a fast or irregular heartbeat), or if they feel dizzy or faint. Kisqali can cause serious liver problems. Patients should tell their health care provider right away if they get any of the following signs and symptoms of liver problems: yellowing of the skin or the whites of the eyes (jaundice), dark or brown (tea-colored) urine, feeling very tired, loss of appetite, pain on the upper right side of the stomach area (abdomen), and bleeding or bruising more easily than normal. Low white blood cell counts are very common when taking Kisqali and may result in infections that may be severe. Patients should tell their health care provider right away if they have signs and symptoms of low white blood cell counts or infections such as fever and chills. Before taking Kisqali, patients should tell their health care provider if they are pregnant, or plan to become pregnant as Kisqali can harm an unborn baby. Females who are able to become pregnant and who take Kisqali should use effective birth control during treatment and for at least 3 weeks after the last dose of Kisqali. Do not breastfeed during treatment with Kisqali and for at least 3 weeks after the last dose of Kisqali. Patients should tell their health care provider about all of the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements since they may interact with Kisqali. Patients should avoid pomegranate or pomegranate juice, and grapefruit or grapefruit juice while taking Kisqali. The most common side effects (incidence >=20%) of Kisqali when used with letrozole include white blood cell count decreases, nausea, tiredness, diarrhea, hair thinning or hair loss, vomiting, constipation, headache, and back pain. The most common grade 3/4 side effects in the Kisqali + letrozole arm (incidence >2%) were low neutrophils, low leukocytes, abnormal liver function tests, low lymphocytes, and vomiting. Abnormalities were observed in hematology and clinical chemistry laboratory tests.

Dynavax Presents Updated Data for SD-101 in Combination with KEYTRUDA(R) (pembrolizumab) Highlighting an ORR in 7 out of 7 Patients Naive to an Anti-PD-1 or Anti-PD-L1 Therapy

On June 2, 2017 Dynavax Technologies Corporation (NASDAQ: DVAX) reported the presentation of findings in patients with metastatic melanoma in the dose escalation phase of an ongoing Phase 1b/2 study investigating SD-101, Dynavax’s intratumoral TLR9 agonist, in combination with KEYTRUDA (pembrolizumab), an anti-PD-1 therapy developed by Merck, known as MSD outside the United States and Canada (Press release, Dynavax Technologies, JUN 2, 2017, View Source [SID1234519345]). Results evaluating 19 patients for efficacy and 22 patients for safety were presented in a poster at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago:

In 7 anti-PD-1/L1-naïve patients, SD-101 used in combination with KEYTRUDA resulted in an overall response rate (ORR) of 100%, with a complete response (CR) rate of 29%. This is a meaningful increase over use of KEYTRUDA alone, which has already shown a 33% ORR, with a 6% CR.(1)
In 12 patients with advanced (stage IIIc/IV) melanoma who had previously failed on anti-PD-1 treatment, introduction of SD-101 resulted in tumor shrinkage in 42% of patients, with 17% having a partial response (PR), indicating an anti-tumor immune response generated by SD-101.
The combination of SD-101 and KEYTRUDA in this study, mobilized both innate and adaptive immune response in study participants.
Tumor shrinkage was observed in non-injected visceral lesions.
"Having 7 out of 7 patients naïve to anti-PD-1/L1 treatment responding is very encouraging," said Antoni Ribas, M.D., Ph.D., of the Jonsson Comprehensive Cancer Center at the University of California, Los Angeles, and lead investigator. "These results are supported by tumor shrinkage in patients who had previously progressed on anti-PD-1 treatment and by confirmatory laboratory biomarker assessments in tumor biopsies. If these clinical results are sustained in the ongoing trial, this combination, which mobilizes both innate and adaptive immune responses in patients, could represent an important advancement in immuno-oncology."

SD-101 in combination with KEYTRUDA generally was well-tolerated. No dose-limiting toxicities of the combination were observed in any dose cohort, and a maximum tolerated dose (MTD) was not identified. The most common treatment-emergent adverse events were injection site reactions and transient grade 1 to 2 flu-like symptoms, including fever, chills and myalgia. The study also includes biomarker assessments, suggesting that treatment with SD-101 and KEYTRUDA resulted in increased tumor-infiltrating lymphocytes and decreased Th2 in tumor biopsies, consistent with induction of an antitumor immune response.

About MEL-01 (KEYNOTE-184)

The dose-escalation and expansion study of SD-101 in combination with KEYTRUDA includes patients with histologically or cytologically confirmed unresectable Stage IIIc/IV melanoma. The primary endpoints of the trial are MTD and evaluation of the safety of intratumoral SD-101 in combination with KEYTRUDA. In addition, the trial is investigating response as assessed by the investigator according to RECIST v1.1, biomarker assessments and duration of response. Patients previously treated with anti-PD-1 and other immunotherapies are included.

About SD-101

SD-101 is Dynavax’s proprietary, second-generation, Toll-like receptor 9 (TLR9) agonist CpG-C class oligodeoxynucleotide. SD-101 is being studied for its multiple anti-tumor activities in innate immune cells and activation of plasmacytoid dendritic cells to stimulate T cells specific for antigens released from dying tumor cells. TLR9 agonists such as SD-101 enhance T and B cell responses and provide potent Type 1 interferon induction and maturation of plasmacytoid dendritic cells to antigen-presenting cells. SD-101 is being evaluated in several Phase 1/2 oncology studies to assess its safety and activity.

For information about SD-101 trials that are currently recruiting patients, please visit www.clinicaltrials.gov.

Endocyte Announces Clinical Updates for EC1456 and EC1169

On June 2, 2017 (GLOBE NEWSWIRE) — Endocyte, Inc. (NASDAQ:ECYT), a leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy, reported updates on clinical development for EC1456, its folate receptor-targeted tubulysin cancer therapy, and EC1169, its prostate-specific membrane antigen targeted (PSMA-targeted) tubulysin prostate cancer therapy (Press release, Endocyte, JUN 2, 2017, View Source [SID1234519344]).

Endocyte will narrow the EC1169 development program to focus only on the cohort of taxane-exposed, metastatic castration-resistant prostate cancer (mCRPC) patients, for which a top-line efficacy assessment of the expansion phase of this phase 1 trial is expected before the end of 2017. An interim assessment confirmed clinical activity of the drug in the taxane-exposed cohort with a partial response in one patient, stable disease in other patients, and other markers of activity. Endocyte believes EC1169 may have benefit in taxane-exposed patients, with more advanced disease, where upregulation of PSMA increases with prior treatments. Endocyte will stop enrollment of taxane-naïve mCRPC patients in the EC1169 trial.

In addition, the company plans to stop enrollment in the EC1456 trial, where a careful assessment in folate receptor-positive (FR-positive) disease across multiple cohorts of patients and multiple dosing schedules did not yield the level of clinical activity necessary to support continued advancement of this agent. However, enrollment of a small number of patients in the EC1456 ovarian cancer surgical study will continue in order to inform other SMDC programs in development. Preliminary data to-date from the ovarian surgical study provides evidence that patients with FR-positive disease are being successfully identified with the use of the etarfolatide imaging agent, but the intratumoral levels of the EC1456 drug payload may be lower than predicted by pre-clinical models.

"Endocyte is a data-driven company, and we are committed to the disciplined management of clinical programs as the science guides us," said Mike Sherman, president and CEO at Endocyte. "Recently gained insight into the safety and efficacy of EC1169 and EC1456, coupled with our commitment to the productive investment of capital, has led us to refocus efforts on our most promising programs, which include our CAR T-cell SMDC adaptor platform, our dual-targeted DNA crosslinker drug EC2629, and the cohort of taxane-exposed patients receiving EC1169."

Endocyte is advancing its work with Seattle Children’s Research Institute aimed at bringing its CAR T-cell bi-specific adaptor molecule to the clinic in 2018. In addition, the company plans to file its IND for EC2629 in mid-2017, which includes a potent DNA-targeted warhead with clinically proven activity. To Endocyte’s knowledge, this is the first drug candidate designed to simultaneously target both tumor cells and tumor associated macrophages (TAMs), which contribute to disease progression by interfering with natural anti-tumor immune responses.

Endocyte also announced today plans to reduce its workforce by approximately 40 percent in order to better focus its resources on the company’s highest value opportunities, while maintaining key capabilities.

"As we refocus our clinical development efforts, we are also aligning our investments and resources to advance our most compelling pipeline programs to key inflection points," added Sherman. "We are very grateful for all the contributions over the years from our dedicated, talented team of employees, who have devoted so much of themselves towards helping advance our efforts to bring our innovative, targeted therapies to patients with cancer and other serious diseases. We’ll be working closely with those affected by the restructuring to support them through this difficult but necessary transition."

Endocyte anticipates one-time charges of approximately $2.4 million related to termination benefits and the accelerated closure of the EC1456 trial. As a result of this restructuring, the company is revising its guidance for 2017 and now expects its cash balance at the end of 2017 to be approximately $105 million, with a more substantial positive impact from the restructuring beginning in 2018.

About EC1169 and the Phase 1 Trial

EC1169 is an investigational therapeutic SMDC constructed of a high affinity PSMA-targeting ligand conjugated through a bioreleasable linker system to a potent microtubule inhibitor, tubulysin B hydrazide (TubBH). Patient PSMA-status is determined using the investigational companion imaging agent, EC0652. EC1169 and EC0652 are currently being evaluated in a phase 1b study in up to 50 taxane-exposed mCRPC patients at a maximum clinical EC1169 dose of 6.5 mg/m2. Endocyte is stopping enrollment of taxane-naïve mCRPC patients (ClinicalTrials.gov Identifier: NCT02202447).

The open-label, multicenter, non-randomized, study is divided into two parts. The first part of the study, now complete, was designed to determine the maximum clinical dose and recommended Phase 2 dose of EC1169 in patients with mCRPC.

Endocyte is currently enrolling the second part of the study which is designed to evaluate the safety and efficacy of EC1169 in taxane-exposed patients, with a primary study endpoint of radiographic progression free survival in patients selected as PSMA-positive. The trial is expected to complete enrollment in Q3 2017 with a more mature endpoint assessment by year-end.

About EC1456 and the Phase 1 Trial

EC1456 is an investigational therapeutic SMDC constructed of a high affinity FR-targeting ligand conjugated through a spacer and bioreleasable linker system to a potent cytotoxic microtubule inhibitor, TubBH. Patient FR-status is determined using the investigational companion imaging agent, etarfolatide.

Endocyte completed the dose escalation portion of the study designed to determine the maximum clinical dose for multiple dosing schedules and is stopping enrollment of the phase 1b study which was evaluating EC1456 at a dose of 6.0mg/m2 twice weekly (BIW) in up to 40 FR-positive non-small cell lung cancer (NSCLC) patients (as determined by an etarfolatide scan).

Endocyte is currently conducting an ovarian cancer surgical study to characterize etarfolatide imaging and intratumoral EC1456 through a multifaceted analysis of collected tissue samples after administration of the drug.