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.

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.

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.

Transgene Announces Upcoming Investor Meetings

On December 5, 2018 Transgene (Paris:TNG) reported that Management will participate in the upcoming investor events set out below (Press release, Transgene, DEC 5, 2018, View Source [SID1234531915]).

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Transgene will meet institutional investors at the LifeSci Advisors Corporate Access Event from January 7 to 9, 2019, in San Francisco, USA, concurrent with the J.P. Morgan Healthcare conference.

The Company will also attend:

Oddo Forum: January 10 & 11, 2019 – Lyon, France
Biomed Event: January 22, 2019 – Paris, France
Degroof Petercam Healthcare Seminar: January 31, 2019 – Brussels, Belgium
HC Wainwright Conference: April 8 & 9, 2019 – London, UK
Kempen Life Sciences Conference: April 16 & 17, 2019 – Amsterdam, Netherlands
Small Cap Event: April 16 & 17, 2019 – Paris, France

The phase 3 study of Darzalex ® ▼ (daratumumab) shows efficacy and safety data of the anti-CD38 monoclonal antibody in patients with recently diagnosed multiple myeloma

On December 5, 2018 Janssen pharmaceutical companies at Johnson & Johnson reported the results of the MAIA phase 3 study, which shows that the addition of daratumumab to lenalidomide and dexamethasone (Rd) significantly reduced the risk of progression of the disease or death in patients with recently diagnosed multiple myeloma who are not suitable for autologous stem cell transplantation (ASCT) ( abstract # LBA-2 ) (Press release, Johnson & Johnson, DEC 5, 2018, View Source [SID1234531914]). 1 These data were presented during the oral session of last-minute last-minute summaries at the 60th annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in San Diego, California.

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"The Phase 3 MAIA study reinforces the clinical profile of daratumumab in combination with a standard treatment regimen for newly diagnosed patients with multiple myeloma who are not eligible for transplantation," states Thierry Facon, MD, Service des Maladies du Sang, Hôpital Claude Huriez, Lille, France and principal investigator. "The positive data show the potential role of daratumumab in combination with lenalidomide and dexamethasone as a new important therapeutic approach for this patient population."

At a mean follow-up of 28 months, data from the phase 3 MAIA study showed that daratumumab in combination with Rd significantly reduced the risk of progression or death of the disease by 44 percent in newly diagnosed multiple myeloma patients who are not fit for transplantation, compared to treatment with Rd alone (hazard ratio [HR] = 0.56, 95% confidence interval [CI]: 0.43-0.73, p <0.0001). 1 The median progression free survival (PFS) for daratumumab-Rd has not yet been achieved, compared to 31.9 months for patients who received Rd alone. oneThe incorporation of daratumumab resulted in deeper responses with respect to Rd alone, including increased rates of complete response (CR) or higher (48 percent vs. 25 percent) and improved rates of very good partial response (VGPR) or better (79 percent, versus 53 percent). 1 Daratumumab-Rd induced a 3-fold higher rate of negativity to minimal residual disease (MRD) compared to those who received Rd alone (24 percent vs. 7 percent). one

"Estos datos subrayan el perfil clínico estable observado en los pacientes recién diagnosticados con mieloma múltiple que reciben terapia con daratumumab, incluso para aquellos que no son aptos para el trasplante", comenta la Dra. Catherine Taylor, directora del área de terapia hematológica para Europa, Oriente Medio y África (EMEA) de Janssen-Cilag Limited. "Es el tercer estudio en pacientes recientemente diagnosticados que ha alcanzado su principal criterio de evaluación y esperamos continuar proporcionando avances innovadores a pacientes con mieloma múltiple a través de nuestro robusto programa de investigación clínica, que cuenta con el potencial de revolucionar el tratamiento del cáncer atacando a la enfermedad en sus etapas más tempranas", añade Taylor.

The most common grade 3/4 adverse events arising from treatment (TEAE) for daratumumab-Rd (≥ 10 percent) included neutropenia (50 percent), lymphopenia (15 percent), pneumonia (14 percent), and anemia (12 percent). 1 Infusion-related reactions (RRI) occurred in 41 percent of patients, of whom only 3 percent were grade 3/4. 1 The incidence of invasive secondary neoplasms was 3 percent in the daratumumab-Rd group, compared to 4 percent with Rd alone. 1 TEAEs with a death result were 7 percent in the daratumumab group, compared to 6 percent in the Rd group.1 The safety profile of daratumumab was in line with that of previous studies. one

These data will support a future marketing authorization request for daratumumab in combination with Rd for this patient population.

#FINISH#

About the MAIA 1 trial

The randomized, open-label, multi-center phase 3 study included 737 newly diagnosed patients with multiple myeloma unsuitable for high-dose chemotherapy and ASCT between 45 and 90 years (mean age 73). Patients were randomly assigned to receive daratumumab-Rd or Rd only in cycles of 28 days. In the daratumumab-Rd treatment group, patients received IV daratumumab 16 milligrams per kilogram (mg / kg) weekly for cycles 1 – 2, every two weeks for cycles 3-6 and every 4 weeks for cycle 7 and later. Patients in the daratumumab-Rd and Rd treatment group received 25 mg of lenalidomide on days 1 – 21 of each 28-day cycle and dexamethasone at 40 mg once per week for each cycle.

About daratumumab

Daratumumab is a first-generation biologic drug against the CD38 antigen, a protein that is expressed at high levels on the surface of multiple myeloma tumor cells, regardless of the stage of the disease. 2 Daratumumab induces rapid death of cancer cells through multiple immunologically mediated mechanisms of action, including complementary cytotoxicity (CDC), cellular cytotoxicity by antibody dependence (ADCC), cellular phagocytosis by antibody dependence (ADCP) and apoptosis , in which a series of molecular steps in a cell leads to its death. 3 Daratumumab also reduced a subset of suppressor cells of myeloid origin (CD38 + MDSCs), CD38 + T regulatory cells (Tregs) and CD38 + B (Bregs). 3 Currently daratumumab is under study through a comprehensive clinical development program on different treatments for multiple myeloma, including parameters such as first-line treatment and relapse. 4,5,6,7,8,9,10,11 Likewise, there are additional studies in progress or planned in order to study their potential in malignant or premalignant hematological diseases in which the CD38 protein is expressed, such as latent myeloma. . 12-13 For more information, see www.clinicaltrials.gov.

In Europe, daratumumab is currently indicated for use in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are not eligible for an autologous stem cell transplant, whose previous treatment included an inhibitor of the proteasome and an immunomodulatory agent, and that they would have demonstrated a progression of the disease in the last treatment and in combination with lenalidomide and dexamethasone or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one previous therapy 3 . For more information on daratumumab, see the Summary of Product Characteristics inView Source .

In August 2012 , Janssen Biotech, Inc. and Genmab A / S signed a worldwide agreement that awarded Janssen an exclusive license to develop, manufacture and market daratumumab. 14

About multiple myeloma

Multiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells. 15 In 2016, more than 45,000 new cases were diagnosed in Europe and more than 29,000 patients died 16 . Up to half of the patients with a recent diagnosis do not reach the five-year survival rate 17 and almost 29% of the patients with MM will die within one year of diagnosis. 18

Although treatment may result in remission, unfortunately, most patients will most likely relapse as there is currently no cure. 19 Refractory multiple myeloma occurs when the disease progresses within 60 days of the last therapy. 20,21 Recurrent cancer occurs when the disease returns after a period of initial, partial or complete remission. 22 While some patients with MM have no symptoms at all, most patients are diagnosed due to symptoms that may include bone problems, low blood counts, elevated calcium, kidney problems, or infections. 2. 3 Patients who relapse after treatment with standard therapies, including PIs and immunomodulatory agents, have a poor prognosis and few treatment options are available. 24