Apexian Pharmaceuticals Presents Data on Their Lead Drug for Anti-Chemotherapy-Induced Peripheral Neuropathy (CIPN), APX3330, at ASCO Palliative Care and Symptom Management Meeting

On December 5, 2018 Apexian Pharmaceuticals reported that Chemotherapy causes chemotherapy-induced peripheral neuropathy (CIPN) in a significant number of patients, yet the pharmaceutical landscape is completely devoid of treatments to prevent CIPN (Press release, Apexian Pharmaceuticals, DEC 5, 2018, View Source [SID1234531916]). The tingling, burning, pain or numbness in the extremities can limit or stop cancer treatment. And, in half the patients affected, CIPN’s symptoms persist five years or more after treatment ends. Apexian Pharmaceuticals aims to change that with their lead compound, APX3330.

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Data presented at the meeting showed continued preclinical support for APX3330 as a potential anti-CIPN treatment, particularly for patients treated with cisplatin or oxaliplatin. Preclinical results presented show APX3330 can block tumor growth while protecting nerve cells.

APX3330, an oral treatment, is currently in a Phase I oncology trial for safety. A Phase II trial is planned in 2019 for anti-tumor and anti-CIPN.

Apexian’s founder and Chief Science Officer, Mark Kelley, PhD, presented the trial’s results in ASCO (Free ASCO Whitepaper)’s Symptom Management Meeting in San Diego, November 16-18, 2018.

Currently ASCO (Free ASCO Whitepaper) does not recommend any drug for preventing or treating CIPN.

"CIPN is a disease with high unmet need and it is exciting to see that APX3330 may have a role to play in addressing the need," says Steve Carchedi, President & CEO of Apexian. "We are committed to developing a portfolio of novel APE1/Ref-1 compounds that will enhance the lives of patients."

The success of APX3330 builds upon three decades of research by Kelley and his colleagues in modulating a key DNA repair protein, APE1/Ref-1. APX3330 tweaks the protein’s activity to prevent or repair neuronal damage without stimulating cancerous tumors.

APX3330 is Apexian’s lead compound in its growing drug development pipeline.

Apexian Pharmaceuticals presents data on their lead drug for anti-chemotherapy-induced peripheral neuropathy (CIPN), APX3330, at ASCO Palliative Care and Symptom Management meeting

On December 5, 2018 Apexian Pharmaceuticals reported that Chemotherapy causes chemotherapy-induced peripheral neuropathy (CIPN) in a significant number of patients, yet the pharmaceutical landscape is completely devoid of treatments to prevent CIPN (Press release, Apexian Pharmaceuticals, DEC 5, 2018, View Source [SID1234532133]). The tingling, burning, pain or numbness in the extremities can limit or stop cancer treatment. And, in half the patients affected, CIPN’s symptoms persist five years or more after treatment ends. Apexian Pharmaceuticals aims to change that with their lead compound, APX3330.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Data presented at the meeting showed continued preclinical support for APX3330 as a potential anti-CIPN treatment, particularly for patients treated with cisplatin or oxaliplatin. Preclinical results presented show APX3330 can block tumor growth while protecting nerve cells.

APX3330, an oral treatment, is currently in a Phase I oncology trial for safety. A Phase II trial is planned in 2019 for anti-tumor and anti-CIPN.

Apexian’s founder and Chief Science Officer, Mark Kelley, PhD, presented the trial’s results in ASCO (Free ASCO Whitepaper)’s Symptom Management Meeting in San Diego, November 16-18, 2018.

Currently ASCO (Free ASCO Whitepaper) does not recommend any drug for preventing or treating CIPN.

"CIPN is a disease with high unmet need and it is exciting to see that APX3330 may have a role to play in addressing the need", says Steve Carchedi, President & CEO of Apexian. "We are committed to developing a portfolio of novel APE1/Ref-1 compound’s that will enhance the lives of patients."

The success of APX3330 builds upon three decades of research by Kelley and his colleagues in modulating a key DNA repair protein, APE1/Ref-1. APX3330 tweaks the protein’s activity to prevent or repair neuronal damage without stimulating cancerous tumors.

APX3330 is Apexian’s lead compound in its growing drug development pipeline.

Phase 3 Trial of Rova-T as Second-line Therapy for Advanced Small-Cell Lung Cancer (TAHOE Study) Halted

On December 5, 2018 AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, reported the decision to stop enrollment for the TAHOE trial, a Phase 3 study evaluating Rovalpituzumab Tesirine (Rova-T) as a second-line therapy for advanced small-cell lung cancer (SCLC) (Press release, AbbVie, DEC 5, 2018, View Source [SID1234531917]). An Independent Data Monitoring Committee (IDMC) recommended stopping enrollment in TAHOE due to shorter overall survival in the Rova-T arm compared with the topotecan control arm. For patients currently on treatment with Rova-T in TAHOE, the IDMC recommended that investigators and patients make individual decisions as to whether or not to continue treatment based on patient level response. The recommendation from the IDMC to halt enrollment applies only to the TAHOE study and does not impact other Rova-T clinical studies.

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"Patients are our first priority and we are deeply grateful to the patients and physicians who participated in this trial," said Michael Severino, M.D., executive vice president, research and development and chief scientific officer, AbbVie. "We remain committed to discovering and developing transformative therapies for people living with cancer."

About the TAHOE Trial
The TAHOE trial is a randomized, open-label, two-arm, Phase 3 trial assessing the efficacy, safety and tolerability of Rova-T versus topotecan in participants with advanced or metastatic small-cell lung cancer (SCLC) with high levels of delta-like protein 3 (DLL3) and who have first disease progression during or following front-line platinum-based chemotherapy.

About Rovalpituzumab Tesirine (Rova-T)
Rova-T is an investigational antibody-drug conjugate targeting the cancer-stem cell-associated delta-like protein 3 (DLL3)1, which is expressed in more than 80 percent of small-cell lung cancer (SCLC) patient tumors, where it is prevalent on tumor cells, including cancer stem cells, but not present in healthy tissue.2 Rova-T combines a targeted antibody that delivers a cytotoxic agent directly to the DLL3-expressing cancer cells while minimizing toxicity to healthy cells. Rova-T is under investigation as a third-line treatment in SCLC.2 The expression of DLL3 suggests Rova-T may be useful across multiple tumor types, including metastatic melanoma, glioblastoma multiforme and some prostate, pancreatic and colorectal cancers.2

Rova-T is an investigational compound and its efficacy and safety have not been established by the FDA or any other health authority.

Daiichi Sankyo Presents Updated Phase 1 Data for U3-1402 in Patients with HER3-Expressing Metastatic Breast Cancer at 2018 San Antonio Breast Cancer Symposium (SABCS)

On December 5, 2018 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that updated efficacy and safety results from the ongoing phase 1/2 study of U3-1402, an investigational and potential first-in-class HER3-targeting antibody drug conjugate (ADC), in 42 heavily pretreated patients with HER3-positive metastatic breast cancer were presented during a Spotlight Session at the 2018 San Antonio Breast Cancer Symposium (SABCS) (Press release, Daiichi Sankyo, DEC 5, 2018, View Source [SID1234531918]).

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Updated efficacy data for 42 evaluable patients who received U3-1402 in dose levels between 1.6 mg/kg to 8.0 mg/kg in the dose escalation and dose finding parts of the study showed a confirmed overall response rate of 42.9 percent (18/42 patients) and a disease control rate of 90.5 percent (38/42 patients) at a median follow-up time of 10.5 months. A median duration of response was not reached (range: 2.8, 13.8+). The median progression-free survival was 8.3 months (range: 1.2, 16.8+). Efficacy was observed in all molecular subtypes. A total of 21 patients remain on treatment at the time of data cut-off on November 6, 2018.

"These results offer preliminary evidence of U3-1402 activity in HER3-positive metastatic breast cancer and further study is warranted," said Norikazu Masuda, MD, PhD, National Hospital Organization, Osaka National Hospital, Osaka, Japan, and an investigator for the trial. "The initial efficacy and safety data suggest that a HER3-targeting agent such as U3-1402 could provide a new treatment approach for patients with HER3-expressing metastatic breast cancer, who are in need of additional options to manage their disease."

Updated safety results were also reported. With a median exposure to treatment of 7.6 months, U3-1402 showed a manageable safety profile. The most common adverse events (≥30 percent, any grade) included nausea (85.7 percent), thrombocytopenia (71.4 percent), decreased appetite (66.7 percent), neutropenia (64.3 percent), leukopenia (59.5 percent), vomiting (54.8 percent), AST increased (47.6 percent), ALT increased (45.2 percent), anemia (38.1 percent), stomatitis (35.7 percent) and diarrhea (31.0 percent). The most common adverse events Grade ≥3 (>10 percent of patients) were thrombocytopenia (35.7 percent), neutropenia (28.6 percent), leukopenia (21.4 percent), anemia (16.7 percent) and ALT increased (11.9 percent). Fourteen (14) patients (33.3 percent) experienced serious adverse events regardless of causality, and seven (7) patients (16.7 percent) experienced serious adverse events that were treatment-related. One (1) patient experienced an adverse event leading to treatment discontinuation (2.4 percent), and there were no adverse events leading to death.

"We are encouraged by these preliminary findings with U3-1402, particularly because there are no therapies specifically approved for HER3-expressing breast cancer," said Kouichi Akahane, PhD, MBA, Executive Officer, Head of Oncology Function, R&D Division, Daiichi Sankyo. "Additionally, these findings continue to build evidence that supports the portability of Daiichi Sankyo’s proprietary DXd and linker ADC technology to other targets such as HER3."

About the Phase 1 Study

In this three-part open-label global phase 1/2 study, U3-1402 is given as an intravenous infusion every three weeks. The first part of the study (dose escalation) assessed the safety, tolerability and maximum tolerated dose of U3-1402 in HER3-positive (defined as IHC [immunohistochemistry] 2+/3+) metastatic breast cancer patients who are refractory or intolerant to standard treatment or for whom no standard treatment is available. A maximum tolerated dose has not been reached. Based on preliminary results, dose levels of 4.8 mg/kg and 6.4 mg/kg are being further evaluated. The second part of the study (dose finding) is assessing the safety and efficacy of U3-1402 and determining the recommended phase 2 dose in HER3-positive metastatic breast cancer patients who have received six or fewer prior chemotherapy regimens. The third part of the study (phase 2) is assessing the safety and efficacy of the recommended dose of U3-1402 in HER3-positive metastatic breast cancer patients who have received six or fewer prior chemotherapy regimens. The study is currently enrolling patients in Japan and the U.S. For more information about this study, please visit ClinicalTrials.gov.

About HER3-Positive Metastatic Breast Cancer

HER3 is a member of the human epidermal growth factor receptor family of tyrosine kinase receptors, which are associated with aberrant cell growth.¹ HER3 is overexpressed in several types of solid tumors including breast cancer.² HER3 overexpression has been independently associated with a poorer prognosis and reduced survival for patients with invasive breast cancer.¹ A wide range of incidence of HER3 overexpression has been reported in breast cancer, depending on the screening method and published series.¹ There are currently no targeted therapies approved specifically for HER3-expressing breast cancer or any HER3-expressing cancer.

About U3-1402

Part of the investigational ADC Franchise of the Daiichi Sankyo Cancer Enterprise, U3-1402 is an investigational and potential first-in-class HER3-targeting ADC. ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy ("payload") to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells. Designed using Daiichi Sankyo’s proprietary ADC technology, U3-1402 is comprised of a human anti-HER3 antibody attached to a novel topoisomerase I inhibitor payload by a tetrapeptide-based linker. It is designed to target and deliver chemotherapy inside cancer cells and reduce systemic exposure to the cytotoxic payload (or chemotherapy) compared to the way chemotherapy is commonly delivered.

U3-1402 is currently being evaluated in two clinical studies, including the phase 1/2 study for HER3-expressing metastatic or unresectable breast cancer in Japan and the U.S., and a phase 1 study for metastatic or unresectable EGFR-mutated non-small cell lung cancer (NSCLC) in the U.S.

U3-1402 is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

FDA grants priority review to Roche’s Tecentriq in combination with chemotherapy for the initial treatment of extensive-stage small cell lung cancer

On December 5, 2018 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the US Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for Tecentriq (atezolizumab), in combination with carboplatin and etoposide (chemotherapy), for the initial (first-line) treatment of people with extensive-stage small cell lung cancer (ES – SCLC) (Press release, Hoffmann-La Roche, DEC 5, 2018, View Source [SID1234531901]). The FDA is expected to make a decision on approval by 18 March 2019. A Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease.

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"It’s been more than 20 years since there has been a new initial treatment option for extensive-stage small cell lung cancer that delivers a clinically meaningful survival benefit," said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development. "We are working closely with the FDA to bring this Tecentriq-based regimen to people with this difficult-to-treat type of lung cancer as soon as possible."

This sBLA is based on results from the Phase III IMpower133 study, which met its co-primary endpoints of overall survival (OS) and progression-free survival (PFS) in the initial treatment of people with ES-SCLC. The safety profile of the combination was consistent with the safety profiles of the individual medicines, and no new safety signals were identified.

Tecentriq is currently approved by the FDA to treat people with metastatic non-small cell lung cancer (NSCLC) who have disease progression during or following platinum-containing chemotherapy, and have progressed on an appropriate FDA-approved targeted therapy if their tumour has ALK or EGFR gene abnormalities.

About the IMpower133 study
IMpower133 is a Phase III, multicentre, double-blinded, randomised placebo-controlled study evaluating the efficacy and safety of Tecentriq in combination with carboplatin and etoposide versus chemotherapy (carboplatin plus etoposide) alone in chemotherapy-naïve people with ES-SCLC. The study enrolled 403 people who were randomised equally (1:1) to receive:

Tecentriq in combination with carboplatin and etoposide (Arm A), or
Placebo in combination with carboplatin and etoposide (Arm B, control arm)
During the treatment-induction phase, people received treatment on 21-day cycles for four cycles, followed by maintenance with Tecentriq or placebo until progressive disease (PD) as assessed by the investigator using Response Evaluation Criteria in Solid Tumours Version 1.1 (RECIST v1.1). Treatment could be continued until persistent radiographic PD or symptomatic deterioration was observed.

The co-primary endpoints were:

PFS as determined by the investigator using RECIST v1.1 in the intention-to-treat (ITT) population
OS in the ITT population
IMpower133 met its OS and PFS co-primary endpoints as per the study protocol. The analysis showed that Tecentriq and chemotherapy helped people live significantly longer, compared with chemotherapy alone (OS=12.3 versus 10.3 months; hazard ratio [HR]=0.70; 95% CI: 0.54–0.91; p=0.0069) in the ITT population. [1] The 1-year OS rate for people who received the Tecentriq-based combination was 51.7%, compared with 38.2% for people who received chemotherapy alone. The Tecentriq-based combination also significantly reduced the risk of disease worsening or death (PFS) compared with chemotherapy alone (PFS=5.2 versus 4.3 months; HR=0.77; 95% CI: 0.62–0.96; p=0.017).[1] The 1-year PFS rate for people who received the Tecentriq-based combination was 12.6%, compared with 5.4% for people who received chemotherapy alone. Safety for the Tecentriq and chemotherapy combination appeared to be consistent with the known safety profile of the individual medicines, and no new safety signals were identified with the combination.

Grade 3–4 treatment-related adverse events (AEs) were reported in 56.6% of people receiving Tecentriq plus chemotherapy, compared with 56.1% of people receiving chemotherapy alone. [1]

About SCLC
Lung cancer is the leading cause of cancer death globally.[2] Each year 1.76 million people die as a result of the disease; this translates into more than 4,800 deaths worldwide every day. [2] Lung cancer can be broadly divided into two major types: non-small cell lung cancer (NSCLC) and SCLC, with SCLC accounting for approximately 15% of all lung cancer cases. [3] Survival rates for people with SCLC vary depending on the stage (extent) of the cancer at the time of diagnosis. [4] The five-year relative survival rate for people with stage I SCLC is approximately 31%; however, at stage IV, the five-year relative survival rate declines to approximately 2%.[5]

About Tecentriq
Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1 expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the activation of T cells. Tecentriq has the potential to be used as a foundational combination partner with cancer immunotherapies, targeted medicines and various chemotherapies across a broad range of cancers.

Currently, Roche has nine Phase III lung cancer studies underway, evaluating Tecentriq alone or in combination with other medicines.

Tecentriq is already approved in the European Union, United States and more than 80 countries for people with previously treated metastatic NSCLC and for certain types of untreated or previously treated metastatic urothelial carcinoma (mUC).