bluebird bio Presents New Data from Clinical Studies of LentiGlobinTM Gene Therapy in Transfusion-Dependent ?-Thalassemia at American Society of Hematology Annual Meeting

On December 10, 2017 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported data from two studies of its LentiGlobin gene therapy product candidate in patients with transfusion-dependent β-thalassemia (TDT) (Press release, bluebird bio, DEC 10, 2017, View Source [SID1234522484]). Data from the Northstar (HGB-204) and Northstar-2 (HGB-207) studies were presented today at the 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) by Janet Kwiatkowski, M.D., MSCE, of Children’s Hospital of Philadelphia, and Mark C. Walters, M.D., of UCSF Benioff Children’s Hospital, respectively.

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"Addressing the underlying genetic cause of TDT to restore production of functional hemoglobin can potentially eliminate or reduce the need for chronic blood transfusions in people with this disease, which we expect will reduce the risk of iron overload and associated long-term complications of TDT, and may allow cessation of chelation therapy," said Dave Davidson, chief medical officer, bluebird bio. "Northstar-2 is the first clinical trial to use our refined manufacturing process for LentiGlobin drug product. Early data from this study demonstrates consistently higher in vivo vector copy numbers and HbAT87Q hemoglobin levels, potentially enabling patients to consistently achieve near-normal or normal total hemoglobin levels. It is important to demonstrate the long-term benefit of gene therapy, and follow-up data of up to three years from the first Northstar study show that nearly all patients with non-β0/β0 genotypes were transfusion-free. We are engaged with the regulatory authorities in the context of the Breakthrough Designation from FDA, and PRIME and Adaptive Pathways from EMA, and look forward to submitting these data to seek marketing approval for LentiGlobin in TDT."

"People with transfusion-dependent thalassemia need regular blood transfusions to survive, but chronic transfusions lead to unavoidable iron overload that can result in multi-organ damage and shortened life span. Eliminating or reducing the need for transfusions can reduce the risk of these long-term complications," said Janet L. Kwiatkowski, MD, MSCE, Director of the Thalassemia Program at the Children’s Hospital of Philadelphia and Associate Professor of Pediatrics at the Perelman School of Medicine of the University of Pennsylvania, and a primary investigator of the Northstar and Northstar-2 studies. "The growing body of data from the Northstar studies indicate LentiGlobin gene therapy may enable transfusion independence for the majority of patients with non-β0/β0 genotypes – and that this effect has been durable during the 3 years of follow-up."

Clinical Outcomes up to 3 Years Following LentiGlobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar HGB-204 Study (Oral Abstract #360)
Presenter: Janet Kwiatkowski, M.D., MSCE, Children’s Hospital of Philadelphia, Philadelphia, PA
Date and Time: Sunday, December 10 at 10:45 a.m.
Location: Building B, Level 2, B213-B214

The Northstar study is an open-label, single-dose, international, multi-center Phase 1/2 study designed to evaluate the efficacy and safety of LentiGlobin for the treatment of patients with TDT. The study has completed its treatment phase and 18 patients with TDT (eight with β0/β0 and 10 with non-β0/β0 genotypes) received LentiGlobin drug product (DP). Results as of September 21, 2017 include:

All 18 patients have ≥18 months follow up, with 10 completing two-year analysis. Three patients have three years of follow up (median follow-up: 27.4 months; min-max: 17.5-36.5 months).
Nine of ten patients with non-β0/β0 genotypes were free from chronic transfusions for a median of 29 months (range: 14.7-33.1 months).
Patients with non-β0/β0 genotypes who were able to achieve freedom from chronic transfusions had HbAT87Q concentrations of 3.6-9.3.
The one patient with a non-β0/β0 genotype who still required periodic transfusions was treated with LentiGlobin with a VCN in the lower range (VCN: 0.3 copies/diploid genome).
Two of eight patients with β0/β0 genotypes have not received a transfusion in more than a year (16.7 months and 15.7 months). At the patients’ last study visits (Month 36 and Month 18, respectively), total hemoglobin levels were 10.2 and 10.3 g/dL and HbAT87Q levels were 9.7 and 7.0 g/dL, respectively.
Clinically meaningful reductions in transfusion volume and frequency were observed in five of the six patients with β0/β0 genotypes who have continued to receive transfusions.
For the 18 study participants, the median DP vector copy number (VCN) was 0.7 (range: 0.3-1.5) copies/diploid genome, the median cell dose was 8.1 (range: 5.2-18.1) x 106 CD34+ cells/kg, and the proportion of transduced CD34+ cells was 17-58 percent.
The safety profile of LentiGlobin DP continues to be consistent with myeloablative conditioning with single-agent busulfan. No Grade 3 or higher DP-related adverse events (AEs) have been observed, and there is no evidence of clonal dominance.
All study participants remain enrolled in the trial, and there have been no reports of graft versus host disease (GVHD).
Results from the HGB-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of LentiGlobin Gene Therapy for Transfusion-Dependent β-thalassemia (TDT) in Patients with non-β0/β0 Genotypes (Oral Abstract #526)
Presenter: Mark C. Walters, M.D., UCSF Benioff Children’s Hospital, Oakland, Calif.

Date and Time: Sunday, December 10 at 5:15 p.m.
Location: Building C, Level 1, C101 Auditorium

The Northstar-2 study is an ongoing, open-label, single-dose, international, multicenter Phase 3 study designed to evaluate the efficacy and safety of LentiGlobin for the treatment of patients with TDT and non-β0/β0 genotypes. As of December 1, 2017, drug product had been manufactured for 10 patients. The median LentiGlobin DP VCN these patients received was 3.3 (range: 2.4-5.4) copies/diploid genome) compared to a median DP VCN of 0.7 (range: 0.3-1.5) copies/diploid genome in the Phase 1/2 Northstar study. Results in treated patients, ages 15 to 24 years, include:

Seven patients had been infused with LentiGlobin as of October 13, 2017. The median follow-up was 3 months (range: 1-9 months).
All three patients who have ≥6 months follow-up are transfusion-free, and 2/3 have achieved or are approaching a normal total hemoglobin level (up to 12.5 g/dl total Hb; range in three patients: 8.4 – 12.5) without transfusions (up to 10.2 g/dL vector-derived HbAT87Q).
Five of six patients treated in the study with ≥3 months follow-up data available as of December 1, 2017 are making at least 6 g/dL of HbAT87Q.
The safety profile of LentiGlobin to date is similar to that observed in the Northstar study, and consistent with myeloablative conditioning with single-agent busulfan. No DP-related AEs have been observed.
All study participants remain enrolled in the trial, and there have been no reports of graft failure or graft versus host disease (GVHD).
Webcast Information
bluebird bio will host a webcast at 8:30 p.m. ET on Sunday, December 10, 2017. The webcast can be accessed under "Calendar of Events" in the Investors and Media section of the company’s website at www.bluebirdbio.com.

About TDT
Transfusion-dependent β-thalassemia (TDT) is a severe genetic disease characterized by reduced or absent hemoglobin levels that results in severe anemia and ineffective red blood cell production. Supportive care for people with TDT consists of a lifelong regimen of chronic blood transfusions to enable survival and suppress symptoms of the disease, and iron chelation therapy to manage iron overload that results from the transfusions. Despite the availability of supportive care, many people with TDT experience serious complications and organ damage due to underlying disease and iron overload.

Allogeneic hematopoietic stem cell transplant (HSCT) is currently the only available option to address the underlying genetic cause of TDT, though it carries significant risks. Complications of allogeneic HSCT include a risk of treatment-related mortality, graft failure, graft-versus-host disease (GvHD) and opportunistic infections, particularly in patients who undergo non-sibling matched allogenic HSCT.

Takeda and Seattle Genetics to Present Positive Data from Phase 3 ECHELON-1 Clinical Trial Evaluating ADCETRIS® (brentuximab vedotin) in Frontline Advanced Hodgkin Lymphoma

On December 10, 2017 Takeda Pharmaceutical Company Limited (TSE:4502) and Seattle Genetics, Inc. (NASDAQ: SGEN) reported that data from the Phase 3 ECHELON-1 clinical trial evaluating ADCETRIS (brentuximab vedotin) as part of a frontline combination chemotherapy regimen in untreated advanced classical Hodgkin lymphoma will be presented in the Plenary Scientific Session at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting on Sunday, December 10, 2017 (Press release, Seattle Genetics, DEC 10, 2017, View Source;p=RssLanding&cat=news&id=2321955 [SID1234522497]). The data were also simultaneously published online in the New England Journal of Medicine and will be published in the print edition on January 25, 2018. Topline data were reported in June 2017 demonstrating the ECHELON-1 trial met its primary endpoint of a statistically significant improvement in modified progression-free survival (modified PFS) per Independent Review Facility (IRF) versus the control arm. ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of classical Hodgkin lymphoma. ADCETRIS is currently not approved as a frontline therapy for Hodgkin lymphoma.

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"For patients with advanced stage Hodgkin lymphoma, approximately one in three do not achieve long-term remission after standard frontline therapy, which is why the results of ECHELON-1 could be important to this group of patients," said Jesús Gomez Navarro, M.D., Vice President, Head of Oncology Clinical Research and Development, Takeda. "The trial demonstrated that combination treatment with ADCETRIS resulted in a statistically significant improvement in modified progression-free survival versus the control arm. For patients treated with ADCETRIS+AVD, there was a 23 percent reduction in the occurrence of an event, defined as progression, death or need for subsequent anti-cancer therapy for patients not in a complete response, compared to those who were treated with ABVD. We are excited about these clinical trial results and the potential impact ADCETRIS may have in the treatment of patients with advanced stage Hodgkin lymphoma if approved by health authorities for frontline use."

"The standard of care in the treatment of Hodgkin lymphoma has not changed over the last several decades and there remains an unmet need for additional regimens in frontline treatment. Current regimens include bleomycin, which is known to be associated with unpredictable and potentially fatal pulmonary toxicity," said Joseph M. Connors, M.D., FRCPC, Clinical Director, Center for Lymphoid Cancer at BC Cancer in Vancouver, Canada. "Increasing the durable response rate with a frontline therapy that also removes bleomycin from the regimen, represents a major step forward for the Hodgkin lymphoma community. Reducing the risk of relapse, is an important concern for patients and their physicians. In the trial, 33 percent fewer patients treated in the ADCETRIS containing regimen required subsequent salvage chemotherapy or high dose chemotherapy and transplant compared to the patients treated with ABVD. Lastly, the safety profile of ADCETRIS+AVD in the trial was generally consistent with that known for the single-agent components of the regimen."

"The ECHELON-1 Phase 3 clinical trial results were selected by ASH (Free ASH Whitepaper) as one of only six abstracts to be featured in the Plenary Scientific Session, and the data were also published simultaneously today in the New England Journal of Medicine. This study represents a bold effort that began more than five years ago to improve upon the current standard of care regimen that has not significantly changed in more than four decades. We’d like to thank the many patients and physicians who participated in this landmark trial," said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. "These data demonstrate statistically superior activity of an ADCETRIS-containing regimen over ABVD, the current standard of care, including the primary endpoint of modified PFS per IRF, and secondary endpoints trended in favor of the ADCETRIS-containing regimen as well. Importantly, patients treated with the ADCETRIS-containing regimen required fewer subsequent therapies after frontline treatment. The results of the ECHELON-1 study supported FDA Breakthrough Therapy Designation for ADCETRIS in combination with chemotherapy for frontline advanced classical Hodgkin lymphoma, and we recently submitted a supplemental Biologics License Application to the FDA. Our goal is to make this regimen available to patients in the U.S. with advanced Hodgkin lymphoma in the first half of 2018."

Brentuximab Vedotin Plus Doxorubicin, Vinblastine, Dacarbazine (A+AVD) as Frontline Therapy Demonstrates Superior Modified Progression-Free Survival versus ABVD in Patients with Previously Untreated Stage III or IV Hodgkin Lymphoma (HL): The Phase 3 ECHELON-1 Study (Plenary Scientific Session on Sunday, December 10, 3:40 p.m. ET at the Georgia World Congress Center, Building C, Level 1, Hall C2 – C3)

Key findings, which will be presented by Dr. Joseph M. Connors and published in the New England Journal of Medicine, include:

The trial achieved its primary endpoint with the combination of ADCETRIS+AVD resulting in a statistically significant improvement in modified PFS versus the control arm of ABVD as assessed by an Independent Review Facility (IRF) (HR 0.77; p-value=0.035). This corresponds to a 23 percent reduction in the risk of progression, death or need for additional anticancer therapy.
Per IRF assessment, the two-year modified PFS rate for patients in the ADCETRIS+AVD arm was 82.1 percent compared to 77.2 percent in the control arm.
Per investigator assessment, the two-year modified PFS rate for patients in the ADCETRIS+AVD arm was 81.0 percent compared to 74.4 percent in the control arm (HR 0.73; p-value=0.007). This corresponds to a 27 percent reduction in the risk of progression, death or need for additional anticancer therapy.
All secondary endpoints trended in favor of the ADCETRIS+AVD arm, including interim analysis of overall survival (OS; HR 0.72; p-value=0.19). Other secondary endpoints include:
Complete response (CR) rate at the end of randomized regimen in the ADCETRIS+AVD arm was 73 percent compared to 70 percent in the control arm (p-value=0.22).
Objective response rate (ORR) at the end of randomized regimen in the ADCETRIS+AVD arm was 86 percent compared to 83 percent in the control arm (p-value=0.12).
Deauville score ≤2 after completion of frontline therapy was 85 percent in the ADCETRIS+AVD arm compared to 80 percent in the control arm (p-value=0.03).
Certain pre-specified subgroups of patients appeared to benefit more with ADCETRIS+AVD versus ABVD including: patients treated in North America; patients with involvement of >1 extranodal site; patients with International Prognostic Score (IPS) 4 – 7; males; patients with Stage IV disease; and patients aged <60 years.
In the ADCETRIS+AVD arm, 33 percent fewer patients received subsequent salvage chemotherapy or high-dose chemotherapy and transplant.
The safety profile of ADCETRIS+AVD in the ECHELON-1 trial was generally consistent with that known for the single-agent components of the regimen.
The most common clinically relevant adverse events of any grade that occurred in at least 15 percent of patients in the ADCETRIS+AVD and ABVD arms were: neutropenia (58 and 45 percent, respectively), constipation (42 and 37 percent, respectively), vomiting (33 and 28 percent, respectively), fatigue (both 32 percent), peripheral sensory neuropathy (29 and 17 percent, respectively), diarrhea (27 and 18 percent, respectively), pyrexia (27 and 22 percent, respectively), peripheral neuropathy (26 and 13 percent, respectively), abdominal pain (21 and 10 percent, respectively) and stomatitis (21 and 16 percent, respectively). In both the ADCETRIS+AVD and ABVD arms, the most common Grade 3 or 4 events were neutropenia, febrile neutropenia and neutrophil count decrease.
Febrile neutropenia was reduced through the use of prophylactic growth factors (G-CSF) in a subset of patients. In the ADCETRIS+AVD arm of the study, the rate of febrile neutropenia without the use of G-CSF was 21 percent and with the use of G-CSF was reduced to 11 percent. G-CSF primary prophylaxis with ADCETRIS+AVD resulted in overall comparable safety profile to ABVD, decreasing incidence of febrile neutropenia, neutropenia and serious adverse events. Primary prophylaxis with G-CSF was recommended for all patients.
On the ADCETRIS+AVD arm, peripheral neuropathy events were observed in 67 percent of patients compared to 43 percent on the control arm. In the ADCETRIS+AVD arm, the majority of peripheral neuropathy events were Grade 1 or 2. Grade ≥3 events were reported in 11 percent of patients and Grade 4 events were reported in less than one percent of patients. In the control arm, Grade ≥3 events were reported in two percent of patients and there were no Grade 4 events. Two-thirds of the patients with peripheral neuropathy in the ADCETRIS+AVD arm reported resolution or improvement at last follow-up.
Pulmonary toxicity was reported in two percent of patients in the ADCETRIS+AVD arm versus seven percent of patients in the ABVD arm; Grade ≥3 events were reported in less than one percent versus three percent, in the ADCETRIS and control arms respectively.
Nine on study deaths occurred in the ADCETRIS+AVD arm, of which seven were due to neutropenia or associated complications (all occurred in patients who had not received primary prophylaxis with G-CSF with the exception of one patient who entered the trial with pre-existing neutropenia). The remaining two deaths were due to myocardial infarction. In the control arm, there were 13 on study deaths, of which 11 were due to or associated with pulmonary-related toxicity, one was due to cardiopulmonary failure and one death had unknown cause.
ECHELON-1 Trial Design

ECHELON-1 is a randomized, open-label, two-arm, multi-center Phase 3 study designed to compare ADCETRIS and AVD (Adriamycin, vinblastine and dacarbazine) to ABVD (Adriamycin, bleomycin, vinblastine and dacarbazine) as frontline therapy in patients with previously untreated advanced classical Hodgkin lymphoma.
The primary endpoint is modified PFS per IRF. Modified PFS is defined as time to progression, death, or evidence of non-complete response after completion of frontline therapy per IRF followed by subsequent anticancer therapy.
The key secondary endpoint is OS. Other secondary objectives include assessment of CR rate, ORR, event-free survival (EFS), disease-free survival (DFS), duration of response (DOR), rate of Cycle 2 PET negativity, quality of life measures (EORTC QLQ C-30) and safety profile in the ADCETRIS+AVD versus ABVD arms.
The study enrolled 1,334 patients who had histologically-confirmed diagnosis of Stage III or IV Hodgkin lymphoma and had not been previously treated with systemic chemotherapy or radiotherapy. The median age of the patients enrolled in the study was 35 in the ADCETRIS+AVD arm and 37 in the ABVD arm.
Patients received ADCETRIS+AVD or ABVD on Days 1 and 15 of each 28-day cycle for up to six cycles.
The multi-center trial was conducted at 218 sites in 21 countries across North America, Europe, South America, Australia, Asia and Africa.
The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for ADCETRIS in combination with chemotherapy for the frontline treatment of patients with advanced classical Hodgkin lymphoma. Seattle Genetics submitted a supplemental Biologics License Application to the FDA on November 1, 2017. Takeda has begun to submit data from the ECHELON-1 trial to regulatory agencies in its territories, starting with the European Medicines Agency (EMA) on November 29, 2017.

About Classical Hodgkin Lymphoma

Lymphoma is a general term for a group of cancers that originate in the lymphatic system. There are two major categories of lymphoma: Hodgkin lymphoma and non-Hodgkin lymphoma. Classical Hodgkin lymphoma is distinguished from other types of lymphoma by the presence of one characteristic type of cell, known as the Reed-Sternberg cell. The Reed-Sternberg cell expresses CD30.

According to the American Cancer Society, approximately 8,260 cases of Hodgkin lymphoma will be diagnosed in the United States during 2017 and more than 1,000 will die from the disease. According to the Lymphoma Coalition, over 62,000 people worldwide are diagnosed with Hodgkin lymphoma each year and approximately 25,000 people die each year from this cancer.

About ADCETRIS

ADCETRIS is being evaluated broadly in more than 70 clinical trials, including three Phase 3 studies: the ECHELON-1 trial in frontline classical Hodgkin lymphoma that supported the recent FDA Breakthrough Therapy Designation and submission of the supplemental Biologics License Application (BLA) for use in this setting, the ongoing ECHELON-2 trial in frontline mature T-cell lymphomas, and the ongoing CHECKMATE 812 trial of ADCETRIS in combination with Opdivo (nivolumab) for relapsed/refractory Hodgkin lymphoma.

ADCETRIS is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics’ proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-positive tumor cells.

ADCETRIS injection for intravenous infusion has received FDA approval for four indications: (1) regular approval for adult patients with pcALCL or CD30-expressing MF who have received prior systemic therapy, (2) regular approval for the treatment of patients with classical Hodgkin lymphoma after failure of autologous hematopoietic stem cell transplantation (auto-HSCT) or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates, (3) regular approval for the treatment of classical Hodgkin lymphoma patients at high risk of relapse or progression as post-auto-HSCT consolidation, and (4) accelerated approval for the treatment of patients with systemic anaplastic large cell lymphoma (sALCL) after failure of at least one prior multi-agent chemotherapy regimen. The sALCL indication is approved under accelerated approval based on overall response rate. Continued approval for the sALCL indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Health Canada granted ADCETRIS approval with conditions for relapsed or refractory Hodgkin lymphoma and sALCL in 2013, and non-conditional approval for post-ASCT consolidation treatment of Hodgkin lymphoma patients at increased risk of relapse or progression.

ADCETRIS was granted conditional marketing authorization by the European Commission in October 2012 for two indications: (1) for the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma following autologous stem cell transplant (ASCT), or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, and (2) the treatment of adult patients with relapsed or refractory sALCL. The European Commission extended the current conditional marketing authorization of ADCETRIS and approved ADCETRIS for the treatment of adult patients with CD30-positive Hodgkin lymphoma at increased risk of relapse or progression following ASCT.

ADCETRIS has received marketing authorization by regulatory authorities in 69 countries for relapsed or refractory Hodgkin lymphoma and sALCL. See important safety information below.

Seattle Genetics and Takeda are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seattle Genetics has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seattle Genetics and Takeda are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs.

ADCETRIS (brentuximab vedotin) Important Safety Information (European Union)

CONTRAINDICATIONS

ADCETRIS is contraindicated for patients with hypersensitivity to brentuximab vedotin and its excipients. In addition, combined use of ADCETRIS with bleomycin is contraindicated as it causes pulmonary toxicity.

SPECIAL WARNINGS & PRECAUTIONS

Progressive multifocal leukoencephalopathy (PML): John Cunningham virus (JCV) reactivation resulting in PML and death can occur in patients treated with ADCETRIS. PML has been reported in patients who received ADCETRIS after receiving multiple prior chemotherapy regimens.

Patients should be closely monitored for new or worsening neurological, cognitive, or behavioral signs or symptoms, which may be suggestive of PML. Suggested evaluation of PML includes neurology consultation, gadolinium-enhanced magnetic resonance imaging of the brain, and cerebrospinal fluid analysis for JCV DNA by polymerase chain reaction or a brain biopsy with evidence of JCV. ADCETRIS dosing should be held for any suspected case of PML and should be permanently discontinued if a diagnosis of PML is confirmed.

Pancreatitis: Acute pancreatitis has been observed in patients treated with ADCETRIS. Fatal outcomes have been reported. Patients should be closely monitored for new or worsening abdominal pain, which may be suggestive of acute pancreatitis. Patient evaluation may include physical examination, laboratory evaluation for serum amylase and serum lipase, and abdominal imaging, such as ultrasound and other appropriate diagnostic measures. ADCETRIS should be held for any suspected case of acute pancreatitis. ADCETRIS should be discontinued if a diagnosis of acute pancreatitis is confirmed.

Pulmonary Toxicity: Cases of pulmonary toxicity, some with fatal outcomes, have been reported in patients receiving ADCETRIS. Although a causal association with ADCETRIS has not been established, the risk of pulmonary toxicity cannot be ruled out. New or worsening pulmonary symptoms should be promptly evaluated and treated appropriately.

Serious infections and opportunistic infections: Serious infections such as pneumonia, staphylococcal bacteremia, sepsis/septic shock (including fatal outcomes), and herpes zoster, and opportunistic infections such as Pneumocystis jiroveci pneumonia and oral candidiasis have been reported in patients treated with ADCETRIS. Patients should be carefully monitored during treatment for emergence of possible serious and opportunistic infections.

Infusion-related reactions (IRR): Immediate and delayed IRR, as well as anaphylaxis, have occurred with ADCETRIS. Patients should be carefully monitored during and after an infusion. If anaphylaxis occurs, administration of ADCETRIS should be immediately and permanently discontinued and appropriate medical therapy should be administered. If an IRR occurs, the infusion should be interrupted and appropriate medical management instituted. The infusion may be restarted at a slower rate after symptom resolution. Patients who have experienced a prior IRR should be premedicated for subsequent infusions. IRRs are more frequent and more severe in patients with antibodies to ADCETRIS.

Tumor lysis syndrome (TLS): TLS has been reported with ADCETRIS. Patients with rapidly proliferating tumor and high tumor burden are at risk of TLS. These patients should be monitored closely and managed according to best medical practice.

Peripheral neuropathy (PN): ADCETRIS treatment may cause PN, both sensory and motor. ADCETRIS-induced PN is typically cumulative and reversible in most cases. Patients should be monitored for symptoms of PN, such as hypoesthesia, hyperesthesia, paresthesia, discomfort, a burning sensation, neuropathic pain, or weakness. Patients experiencing new or worsening PN may require a delay and a dose reduction or discontinuation of ADCETRIS.

Hematological toxicities: Grade 3 or Grade 4 anemia, thrombocytopenia, and prolonged (equal to or greater than one week) Grade 3 or Grade 4 neutropenia can occur with ADCETRIS. Complete blood counts should be monitored prior to administration of each dose.

Febrile neutropenia: Febrile neutropenia has been reported. Patients should be monitored closely for fever and managed according to best medical practice if febrile neutropenia develops.

Stevens-Johnson syndrome (SJS): SJS and toxic epidermal necrolysis (TEN) have been reported with ADCETRIS. Fatal outcomes have been reported. If SJS or TEN occurs, treatment with ADCETRIS should be discontinued and appropriate medical therapy should be administered.

Gastrointestinal (GI) Complications: GI complications, some with fatal outcomes, including intestinal obstruction, ileus, enterocolitis, neutropenic colitis, erosion, ulcer, perforation and haemorragh, have been reported. New or worsening GI symptoms should be promptly evaluated and treated appropriately.

Hepatotoxicity: Elevations in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) have been reported. Serious cases of hepatotoxicity, including fatal outcomes, have also occurred. Liver function should be tested prior to treatment initiation and routinely monitored in patients receiving ADCETRIS. Patients experiencing hepatotoxicity may require a delay, dose modification, or discontinuation of ADCETRIS.

Hyperglycemia: Hyperglycemia has been reported during trials in patients with an elevated body mass index (BMI) with or without a history of diabetes mellitus. However, any patient who experiences an event of hyperglycemia should have their serum glucose closely monitored. Anti-diabetic treatment should be administered as appropriate.

Renal and Hepatic Impairment: There is limited experience in patients with renal and hepatic impairment. Available data indicate that MMAE clearance might be affected by severe renal impairment, hepatic impairment, and by low serum albumin concentrations. The recommended starting dose in patients with hepatic impairment or severe renal impairment is 1.2 mg/kg administered as an intravenous infusion over 30 minutes every 3 weeks. Patients with renal or hepatic impairment should be closely monitored for adverse events.

Sodium content in excipients: This medicinal product contains a maximum of 2.1 mmol (or 47 mg) of sodium per dose. To be taken into consideration for patients on a controlled sodium diet.

INTERACTIONS

Patients who are receiving a strong CYP3A4 and P-gp inhibitor, concomitantly with ADCETRIS may have an increased risk of neutropenia and should be closely monitored. Co-administration of ADCETRIS with a CYP3A4 inducer did not alter the plasma exposure of ADCETRIS but it appeared to reduce plasma concentrations of MMAE metabolites that could be assayed. ADCETRIS is not expected to alter the exposure to drugs that are metabolized by CYP3A4 enzymes.

PREGNANCY: Women of childbearing potential should be using two methods of effective contraception during treatment with ADCETRIS and until 6 months after treatment. There are no data from the use of ADCETRIS in pregnant women, although studies in animals have shown reproductive toxicity. ADCETRIS should not be used during pregnancy unless the benefit to the mother outweighs the potential risks to the fetus. If a pregnant woman needs to be treated, she should be clearly advised on the potential risk to the fetus.

LACTATION (breast-feeding): There are no data as to whether ADCETRIS or its metabolites are excreted in human milk, therefore a risk to the newborn/infant cannot be excluded. With the potential risk, a decision should be made whether to discontinue breast-feeding or discontinue/abstain from therapy with ADCETRIS.

FERTILITY: In nonclinical studies, ADCETRIS treatment has resulted in testicular toxicity, and may alter male fertility. Men being treated with this medicine are advised not to father a child during treatment and for up to 6 months following the last dose.

ADVERSE REACTIONS

Serious adverse drug reactions were: pneumonia, acute respiratory distress syndrome, headache, neutropenia, thrombocytopenia, constipation, diarrhea, vomiting, nausea, pyrexia, peripheral motor neuropathy, peripheral sensory neuropathy, hyperglycemia, demyelinating polyneuropathy, tumor lysis syndrome, and Stevens-Johnson syndrome.

In the clinical studies of ADCETRIS, adverse reactions defined as very common (≥1/10) were: infection, upper respiratory tract infection, neutropenia, PN (sensory and motor), cough, dyspneoa, diarrhea, nausea, vomiting, constipation, abdominal pain, alopecia, pruritus, myalgia, arthralgia, fatigue, chills, pyrexia, infusion-related reactions and weight decreased. Adverse reactions defined as common (≥1/100 to <1/10) were: Sepsis/septic shock, herpes zoster, pneumonia, herpes simplex, anemia, thrombocytopenia, hyperglycemia, dizziness, demyelinating polyneuropathy, ALT/AST increased, rash, and back pain.

ADCETRIS (brentuximab vedotin) U.S. Important Safety Information

BOXED WARNING: PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY (PML)

JC virus infection resulting in PML and death can occur in ADCETRIS-treated patients.

Contraindication

ADCETRIS concomitant with bleomycin due to pulmonary toxicity (e.g., interstitial infiltration and/or inflammation).

Warnings and Precautions

Peripheral neuropathy (PN): ADCETRIS causes PN that is predominantly sensory. Cases of motor PN have also been reported. ADCETRIS-induced PN is cumulative. Monitor for symptoms such as hypoesthesia, hyperesthesia, paresthesia, discomfort, a burning sensation, neuropathic pain, or weakness. Institute dose modifications accordingly.
Anaphylaxis and infusion reactions: Infusion-related reactions (IRR), including anaphylaxis have occurred with ADCETRIS. Monitor patients during infusion. If an IRR occurs, interrupt the infusion and institute appropriate medical management. If anaphylaxis occurs, immediately and permanently discontinue the infusion and administer appropriate medical therapy. Premedicate patients with a prior IRR before subsequent infusions. Premedication may include acetaminophen, an antihistamine, and a corticosteroid.
Hematologic toxicities: Prolonged (≥1 week) severe neutropenia and Grade 3 or 4 thrombocytopenia or anemia can occur with ADCETRIS. Febrile neutropenia has been reported with ADCETRIS. Monitor complete blood counts prior to each ADCETRIS dose. Consider more frequent monitoring for patients with Grade 3 or 4 neutropenia. Monitor patients for fever. If Grade 3 or 4 neutropenia develops, consider dose delays, reductions, discontinuation, or G-CSF prophylaxis with subsequent doses.
Serious infections and opportunistic infections: Infections such as pneumonia, bacteremia, and sepsis or septic shock (including fatal outcomes) have been reported in ADCETRIS-treated patients. Closely monitor patients during treatment for bacterial, fungal, or viral infections.
Tumor lysis syndrome: Closely monitor patients with rapidly proliferating tumor and high tumor burden.
Increased toxicity in the presence of severe renal impairment: The frequency of ≥Grade 3 adverse reactions and deaths was greater in patients with severe renal impairment compared to patients with normal renal function. Avoid use in patients with severe renal impairment.
Increased toxicity in the presence of moderate or severe hepatic impairment: The frequency of ≥Grade 3 adverse reactions and deaths was greater in patients with moderate or severe hepatic impairment compared to patients with normal hepatic function. Avoid use in patients with moderate or severe hepatic impairment.
Hepatotoxicity: Serious cases, including fatal outcomes, have occurred in ADCETRIS-treated patients. Cases were consistent with hepatocellular injury, including elevations of transaminases and/or bilirubin, and occurred after the first ADCETRIS dose or rechallenge. Preexisting liver disease, elevated baseline liver enzymes, and concomitant medications may increase the risk. Monitor liver enzymes and bilirubin. Patients with new, worsening, or recurrent hepatotoxicity may require a delay, change in dose, or discontinuation of ADCETRIS.
PML: JC virus infection resulting in PML and death has been reported in ADCETRIS-treated patients. First onset of symptoms occurred at various times from initiation of ADCETRIS therapy, with some cases occurring within 3 months of initial exposure. Other possible contributory factors other than ADCETRIS include prior therapies and underlying disease that may cause immunosuppression. Consider PML diagnosis in patients with new-onset signs and symptoms of central nervous system abnormalities. Hold ADCETRIS if PML is suspected and discontinue ADCETRIS if PML is confirmed.
Pulmonary toxicity: Noninfectious pulmonary toxicity events including pneumonitis, interstitial lung disease, and acute respiratory distress syndrome, some with fatal outcomes, have been reported. Monitor patients for signs and symptoms, including cough and dyspnea. In the event of new or worsening pulmonary symptoms, hold ADCETRIS dosing during evaluation and until symptomatic improvement.
Serious dermatologic reactions: Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN), including fatal outcomes, have been reported with ADCETRIS. If SJS or TEN occurs, discontinue ADCETRIS and administer appropriate medical therapy.
Gastrointestinal (GI) complications: Acute pancreatitis, including fatal outcomes, has been reported in ADCETRIS-treated patients. Other fatal and serious GI complications, including perforation, hemorrhage, erosion, ulcer, intestinal obstruction, enterocolitis, neutropenic colitis, and ileus have been reported in ADCETRIS-treated patients. Lymphoma with preexisting GI involvement may increase the risk of perforation. In the event of new or worsening GI symptoms, perform a prompt diagnostic evaluation and treat appropriately.
Embryo-fetal toxicity: Based on the mechanism of action and animal studies, ADCETRIS can cause fetal harm. Advise females of reproductive potential of the potential risk to the fetus, and to avoid pregnancy during ADCETRIS treatment and for at least 6 months after the final dose of ADCETRIS.
Most Common (≥20%) Adverse Reactions: peripheral sensory neuropathy, fatigue, nausea, diarrhea, neutropenia, upper respiratory tract infection, and pyrexia.

Drug Interactions

Concomitant use of strong CYP3A4 inhibitors or inducers, or P-gp inhibitors, has the potential to affect the exposure to monomethyl auristatin E (MMAE).

Use in Specific Populations

Moderate or severe hepatic impairment or severe renal impairment: MMAE exposure and adverse reactions are increased. Avoid use.

Advise males with female sexual partners of reproductive potential to use effective contraception during, and for at least 6 months after the final dose of ADCETRIS treatment.

Advise patients to report pregnancy immediately and avoid breastfeeding while receiving ADCETRIS.

For additional Important Safety Information, including BOXED WARNING, please see the full Prescribing Information for ADCETRIS at www.seattlegenetics.com or www.ADCETRIS.com.

ImmunoGen Presents New Clinical and Preclinical Data at ASH Annual Meeting

On December 9, 2017 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that new data from the Company’s ongoing Phase 1 study of IMGN779, a next-generation CD33-targeting ADC, in patients with relapsed or refractory adult acute myeloid leukemia (AML) were presented at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta (Press release, ImmunoGen, DEC 9, 2017, View Source [SID1234522464]). Poster presentations on preclinical data for IMGN779 in combination with cytarabine and CD123-targeting IMGN632 in acute lymphoblastic leukemia (ALL) are also being presented at the meeting.

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The Phase 1 data presented at ASH (Free ASH Whitepaper) demonstrate that IMGN779 was well-tolerated with no dose-limiting toxicities (DLTs) observed in patients with relapsed or refractory AML across nine dose levels administered once every two weeks (Q2W) and one dose level administered once a week (QW). In addition, anti-leukemia activity was seen at doses ≥0.39 mg/kg in both schedules in patients with poor prognostic features. The maximum tolerated dose has not been reached and dose escalation continues. Data across the first seven dose levels on the Q2W schedule were presented in June at the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).

"The data at ASH (Free ASH Whitepaper) build on the initial safety and anti-leukemia data presented earlier this year at EHA (Free EHA Whitepaper), and further support continued dose escalation of IMGN779, a novel, next-generation treatment for AML," said Anna Berkenblit, M.D., vice president and chief medical officer of ImmunoGen. "Given investigator enthusiasm and high unmet need, the dosing cohorts have been rapidly enrolling and we are very encouraged by the initial findings with IMGN779. We are continuing to dose escalate on the every two week schedule and, to evaluate the potential of continuous exposure, we have opened a weekly dosing schedule in parallel. We look forward to establishing the optimal dose and schedule, and quickly moving this compound into later stages of development."

Phase 1 Data on IMGN779 in AML

Key findings presented from the Phase 1 study of IMGN779 at ASH (Free ASH Whitepaper) (Abstract #1312) include the following:

IMGN779 displays a tolerable safety profile.
No DLTs were observed on either administration schedule at doses examined – up to 0.91 mg/kg Q2W and 0.39 mg/kg QW.
No increase in the nature, frequency, or severity of any treatment-emergent adverse event was observed with increasing dose.
This profile has enabled repeat dosing, with one patient showing a 93% reduction in bone marrow blasts with extended treatment and who remains on therapy through Cycle 14.
Pharmacokinetic (PK) exposures and pharmacodynamic (PD) CD33 saturation continue to increase with dose, and support further escalation and exploration of both the QW and Q2W schedules.
Anti-leukemia activity was seen at doses ≥0.39 mg/kg in both schedules with:
16 of 17 patients showing a decrease in peripheral blasts within 10 days after first dose with a median maximal decrease of 71%; and
Seven of 17 patients showing a 48%-96% reduction in bone marrow blasts. These seven patients had poor prognostic features (e.g., prior intense therapy, primary refractory disease, RAS/TP53/FLT3/IDH mutations).
This ongoing Phase 1 trial is designed to establish the maximum tolerated dose and determine the recommended Phase 2 dose for IMGN779 administered as monotherapy. The trial is also intended to evaluate safety and tolerability, and characterize PK, PD, and preliminary anti-leukemia activity in relapsed or refractory AML.

Preclinical Presentations on IMGN779 in Combination with Cytarabine and IMGN632 in ALL

Supporting data evaluating the mechanism, anti-leukemia efficacy, and tolerability of repeated dosing of IMGN779 in combination with cytarabine using in vitro and in vivo human AML preclinical models were also presented. Key findings from the poster presentation (Abstract #1357) include:

The combination of IMGN779 and cytarabine increased DNA damage response, cell cycle arrest, and apoptosis in vitro when compared to single agent treatment.
The combination of IMGN779 and cytarabine lead to increased survival and greater numbers of complete responses in in vivo preclinical AML models.
Use of cytarabine increased cell surface CD33 levels on AML cells, suggesting a novel mechanism for potentiating IMGN779 uptake.
Preclinical data (Abstract #2718) on IMGN632 reporting the prevalence of CD123 expression in acute lymphoblastic leukemia (ALL), and assessing the anti-leukemia activity of IMGN632 on ALL cells will also be presented. Among the findings:

CD123 expression is prevalent across ALL subtypes including 90% of B-cell ALL (B-ALL) and nearly half of T-cell ALL patient samples.
IMGN632 demonstrates promising activity against B-ALL cell lines and patient samples in vitro, including the elimination of more than 90% of B-ALL blasts in 6 out of 8 patient samples. Normal cells were not affected by IMGN632 at 100-fold higher concentrations.
More information can be found at www.hematology.org, including abstracts.

Poster Session Schedule and Details

Title (Abstract #1312): "IMGN779, a Next-Generation CD33-Targeting Antibody-Drug Conjugate (ADC) Demonstrates Initial Antileukemia Activity in Patients with Relapsed or Refractory Acute Myeloid Leukemia"
Poster session #613: Saturday, December 9, 5:30 – 7:30 PM ET.
Title (Abstract #1357): "IMGN779, a Next Generation CD33-Targeting ADC, Combines Effectively With Cytarabine in Acute Myeloid Leukemia (AML) Preclinical Models, Resulting in Increased DNA Damage Response, Cell Cycle Arrest and Apoptosis In Vitro, and Prolonged Survival In Vivo"
Poster session #616: Saturday, December 9, 5:30 – 7:30 PM ET.
Title (Abstract #2718): "CD123 Expression Patterns and Potential of IMGN632, a CD123-Targeted Antibody Drug Conjugate, in Acute Lymphoblastic Leukemia"
Poster session #618: Sunday, December 10, 6:00 – 8:00 PM ET.
About IMGN779
IMGN779 is a novel ADC that combines a high-affinity, humanized anti-CD33 antibody, a cleavable disulfide linker, and one of ImmunoGen’s novel indolino-benzodiazepine payloads, called IGNs, which alkylate DNA without crosslinking, resulting in potent preclinical anti-leukemia activity with relative sparing of normal hematopoietic progenitor cells.1,2 IMGN779 is in Phase 1 clinical testing for the treatment of AML.

About IMGN632
IMGN632 is a humanized anti-CD123 antibody-drug conjugate that is a potential treatment for AML, blastic plasmacytoid dendritic cell neoplasm (BPDCN), myelodysplastic syndrome, B-cell acute lymphocytic leukemia, and other CD123-positive malignancies. IMGN632 uses a novel IGN payload, linker and antibody technology and in AML xenograft models has demonstrated a large therapeutic index.3 ImmunoGen has filed an investigational new drug (IND) application for IMGN632 and expects to open a Phase I study before year end.

About IGNs
Indolino-benzodiazepine cancer-killing agents, or IGNs, are a new class of cancer-killing agent developed by ImmunoGen for use in ADCs. These ultra-potent, DNA-acting IGNs alkylate DNA without crosslinking, which preclinically has resulted in potent anti-leukemia activity with relative sparing of normal hematopoietic progenitor cells.4,5 IMGN779, a CD33-targeting ADC in Phase 1 testing for AML, was the first IGN ADC to enter clinical testing. IMGN632, a CD123-targeting ADC entering Phase 1 testing for AML and BPDCN, deploys a novel IGN payload.

About Acute Myeloid Leukemia (AML)
AML is a cancer of the bone marrow cells that produce white blood cells. It causes the marrow to increasingly generate abnormal, immature white blood cells (blasts) that do not mature into effective infection-fighting cells. The blasts quickly fill the bone marrow, impacting the production of normal platelets and red blood cells. The resulting deficiencies in normal blood cells leave the patient vulnerable to infections, bleeding problems and anemia.

It is estimated that, in the U.S. alone, 21,380 patients will be diagnosed with AML this year and 10,590 patients will die from the disease.6

Seattle Genetics Highlights Updated Analyses from Phase 3 ALCANZA Clinical Trial of ADCETRIS® (Brentuximab Vedotin) in CD30-Expressing Cutaneous T-Cell Lymphoma at ASH Annual Meeting

On December 9, 2017 Seattle Genetics, Inc. (Nasdaq: SGEN) reported updated results from the phase 3 ALCANZA clinical trial evaluating ADCETRIS (brentuximab vedotin) in CD30-expressing cutaneous T-cell lymphoma (CTCL) at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place in Atlanta, Georgia, December 9-12, 2017 (Press release, Seattle Genetics, DEC 9, 2017, View Source;p=RssLanding&cat=news&id=2321936 [SID1234522472]). The presentation highlighted longer-term durability data from the phase 3 ALCANZA clinical trial of single-agent ADCETRIS for the treatment of patients with primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF). Together, these comprise approximately 70 percent of CTCL diagnoses and the majority of patients who require systemic therapy.

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"The updated analyses from the phase 3 ALCANZA clinical trial presented at this year’s ASH (Free ASH Whitepaper) annual meeting are based on longer-term follow-up by investigators. Since the initial presentation at the ASH (Free ASH Whitepaper) Annual Meeting in 2016, the ALCANZA results continue to show superior clinical efficacy of ADCETRIS over standard-of-care therapies in patients with CD30-expressing CTCL," said Jonathan Drachman, M.D., Chief Medical Officer and Executive Vice President, Research and Development at Seattle Genetics. "The updated analyses demonstrate that the primary and secondary endpoints per investigator are better than previously reported by independent review facility assessment. CTCL is a debilitating and disfiguring disease. With the recent FDA approval of ADCETRIS for use in two common subtypes based on the ALCANZA results, we are now able to provide patients with a clinically meaningful therapeutic option in the approved settings. This is a significant milestone for the lymphoma community and for our goal to make ADCETRIS available to as many patients as possible with CD30-expressing lymphomas."

Updated Analyses of the International, Open-Label, Randomized, Phase 3 ALCANZA Study: Longer-term Evidence for Superiority of Brentuximab Vedotin Versus Methotrexate or Bexarotene for CD30-Positive Cutaneous T-Cell Lymphoma (Abstract #1509, poster presentation on Saturday, December 9, 2017)

ALCANZA was a randomized, open-label phase 3 study designed to evaluate single-agent ADCETRIS versus a control arm of investigator’s choice of standard of care therapies, methotrexate or bexarotene, in patients with CD30-expressing pcALCL or MF. Patients with pcALCL must have received at least one prior systemic or radiation therapy and patients with MF must have received at least one prior systemic therapy. A total of 131 patients were randomized with 128 patients in the intent-to-treat population. Sixty-four patients were assigned to the ADCETRIS arm and 64 patients were assigned to the control arm. Patients received ADCETRIS or investigator’s choice of methotrexate or bexarotene for up to approximately one year.

Data from longer-term patient follow-up per investigator assessment in the phase 3 ALCANZA trial after a median observation time of 33.9 months from the first dose of ADCETRIS versus physician’s choice include:

The trial achieved its primary endpoint of demonstrating a highly statistically significant improvement in the rate of objective response lasting at least four months (ORR4) in the ADCETRIS arm versus the control arm. The ORR4 per investigator assessment was 60.9 percent in the ADCETRIS arm compared to 7.8 percent in the control arm (p-value <0.001).
The key secondary endpoints per investigator, including complete response (CR) rate, progression-free survival (PFS) and reduction in the burden of symptoms during treatment (per Skindex-29 questionnaire), continued to be all highly statistically significant in favor of the ADCETRIS arm.
The median PFS per investigator in the ADCETRIS arm was 15.8 months compared to 3.6 months in the control arm (HR 0.373; 95% CI, 0.245-0.569; p-value <0.001).
The CR rate in the ADCETRIS arm was 18.8 percent compared to zero percent in the control arm (p-value <0.001).
Patient-reported quality of life assessed by the Skindex-29 questionnaire showed significantly greater symptom reduction for patients in the ADCETRIS arm versus the control arm (mean maximum change of -28.08 vs -8.62; p-value <0.001).
At time of the analyses, 47 patients (73 percent) in the ADCETRIS arm and 48 patients (75 percent) in the physician’s choice arm had received one or more subsequent skin-directed or systemic therapy. The median time to next treatment in the ADCETRIS arm was significantly longer at 14.2 months compared with the physician’s choice arm at 6.1 months (p-value <0.001). In the ADCETRIS versus physician’s choice arms, the probability of patients not requiring subsequent skin-directed or systemic therapy was greater at one year (65.5 percent vs. 15.3 percent) and two years (24.6 percent vs. 4.4 percent).
Peripheral neuropathy events were observed in 44 of 66 patients (67 percent) in the ADCETRIS arm and four of 62 patients (six percent) in the physician’s choice arm. In the ADCETRIS arm, 86 percent of patients reported resolution or improvement in peripheral neuropathy events, with 59 percent reporting resolution of all events after a median of 30 weeks and 27 percent reporting some improvement after a median of 13 weeks. Eighteen patients had ongoing peripheral neuropathy events, including 15 patients with Grade 1 and three patients with Grade 2.
In November 2017, the U.S. Food and Drug Administration (FDA) approved ADCETRIS for the treatment of adult patients with pcALCL or CD30-expressing MF who have received prior systemic therapy based on the results of the phase 3 ALCANZA clinical trial.

About CTCL

Lymphoma is a general term for a group of cancers that originate in the lymphatic system. There are two major categories of lymphoma: Hodgkin lymphoma and non-Hodgkin lymphoma. Cutaneous lymphomas are a category of non-Hodgkin lymphoma that primarily involve the skin. According to the Cutaneous Lymphoma Foundation, CTCL is the most common type of cutaneous lymphoma and typically presents with red, scaly patches or thickened plaques of skin that often mimic eczema or chronic dermatitis. The most common subtypes of CTCL include mycosis fungoides and primary cutaneous anaplastic large cell lymphoma. Progression from limited skin involvement may be accompanied by skin tumor formation, ulceration and exfoliation, complicated by itching and infections. Advanced stages are defined by involvement of lymph nodes, peripheral blood and internal organs.

According to the American Cancer Society and the Leukemia and Lymphoma Society, CTCL represents approximately four percent of non-Hodgkin lymphoma, which is about 2,800 patients. Not all newly diagnosed patients require systemic therapy. The standard treatment for CTCL patients includes skin-directed therapies, radiation and systemic therapies. Prior to the FDA approval of ADCETRIS, systemic therapies approved for treatment demonstrated 30 to 45 percent objective response rates, with low complete response rates and low durability as demonstrated by a median time to next systemic treatment of 3.9 months for chemotherapy and 4.5 months for histone deacetylase inhibitors.

About ADCETRIS

ADCETRIS is being evaluated broadly in more than 70 clinical trials, including three phase 3 studies: the completed ECHELON-1 trial in frontline classical Hodgkin lymphoma that supported the recent FDA Breakthrough Therapy Designation and submission of the supplemental Biologics License Application (BLA) for use in this setting, the ongoing ECHELON-2 trial in frontline mature T-cell lymphomas, and the ongoing CHECKMATE 812 trial of ADCETRIS in combination with Opdivo (nivolumab) for relapsed/refractory Hodgkin lymphoma.

ADCETRIS is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics’ proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells.

ADCETRIS injection for intravenous infusion has received FDA approval for four indications: (1) regular approval for adult patients with primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy, (2) regular approval for the treatment of patients with classical Hodgkin lymphoma after failure of autologous hematopoietic stem cell transplantation (auto-HSCT) or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates, (3) regular approval for the treatment of classical Hodgkin lymphoma patients at high risk of relapse or progression as post-auto-HSCT consolidation, and (4) accelerated approval for the treatment of patients with systemic anaplastic large cell lymphoma (sALCL) after failure of at least one prior multi-agent chemotherapy regimen. The sALCL indication is approved under accelerated approval based on overall response rate. Continued approval for the sALCL indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Health Canada granted ADCETRIS approval with conditions for relapsed or refractory Hodgkin lymphoma and sALCL in 2013, and non-conditional approval for post-ASCT consolidation treatment of Hodgkin lymphoma patients at increased risk of relapse or progression.

ADCETRIS was granted conditional marketing authorization by the European Commission in October 2012 for two indications: (1) for the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma following autologous stem cell transplant (ASCT), or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, and (2) the treatment of adult patients with relapsed or refractory sALCL. The European Commission extended the current conditional marketing authorization of ADCETRIS and approved ADCETRIS for the treatment of adult patients with CD30-positive Hodgkin lymphoma at increased risk of relapse or progression following ASCT.

ADCETRIS has received marketing authorization by regulatory authorities in 69 countries for relapsed or refractory Hodgkin lymphoma and sALCL. See important safety information below.

Seattle Genetics and Takeda are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seattle Genetics has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seattle Genetics and Takeda are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs.

BeiGene Presents Preliminary Phase 1b Data on BTK Inhibitor Zanubrutinib (BGB-3111) in Non-Hodgkin’s Lymphoma at the 59th American Society of Hematology Annual Meeting

On December 9, 2017 BeiGene, Ltd. (NASDAQ:BGNE), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly targeted and immuno-oncology drugs for the treatment of cancer, reported preliminary clinical data from an ongoing Phase 1b trial of its investigational Bruton’s Tyrosine Kinase (BTK) inhibitor zanubrutinib (BGB-3111) in patients with non-Hodgkin’s lymphoma (NHL) in an oral presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, GA (Press release, BeiGene, DEC 9, 2017, View Source;p=RssLanding&cat=news&id=2321942 [SID1234522476]). The preliminary data included patients with aggressive NHL subtypes such as diffuse large B-cell lymphoma (DLBCL) and mantle cell lymphoma (MCL) as well as indolent NHL subtypes such as follicular lymphoma (FL) and marginal zone lymphoma (MZL). The Phase 1b data suggest that zanubrutinib was generally well-tolerated and had anti-tumor activity across these NHL subtypes.

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"In this Phase 1b trial, zanubrutinib was well-tolerated across multiple NHL subtypes, with very low rates of toxicity-related treatment discontinuation in both indolent and aggressive disease settings. These preliminary data also indicate that zanubrutinib’s complete and sustained BTK occupancy translates into high response rates in NHL subtypes beyond Waldenström’s macroglobulinemia and Chronic lymphocytic leukemia, for which data have previously been reported," commented Constantine Tam, MD, Disease Group Lead for Low Grade Lymphoma and Chronic Lymphocytic Leukemia at Peter MacCallum Cancer Centre, Director of Haematology at St. Vincent’s Hospital, Australia, and lead author of the presentation.

"Building upon the promising Phase 1b data we have presented for zanubrutinib, we are pleased to report results from additional NHL subtypes enrolled in our Phase 1b trial. We continue to pursue broad development of zanubrutinib with ongoing pivotal trials in a range of NHL subtypes both globally and in China," commented Jane Huang, MD, Chief Medical Officer, Hematology at BeiGene.

Summary of Results from the Ongoing Phase 1b Trial

The multi-center, open-label Phase 1b trial of zanubrutinib in patients with B-cell malignancies is being conducted in Australia, New Zealand, South Korea, the United States, and Europe, and consists of a dose-escalation phase and a dose-expansion phase in disease-specific cohorts. The ongoing dose-expansion component is testing doses of 160 mg twice daily (BID) or 320 mg once a day (QD). As of September 15, 2017, the date of the most recent data cutoff, 99 patients with NHL subtypes other than chronic lymphocytic leukemia/small lymphocytic lymphoma and Waldenström’s macroglobulinemia were enrolled in the study, including 34 patients in the indolent lymphoma cohort, which consisted of 24 patients with FL and 10 patients with MZL, and 65 patients in the aggressive lymphoma cohort, which consisted of 27 patients with DLBCL and 38 patients with MCL. The median follow-up time was 5.6 months (0.3-22.3 months) and 5.1 months (0.1-31.9) for indolent and aggressive lymphoma, respectively.

At the time of data cutoff, the most frequent adverse events (AEs) (occurring in ≥15% of patients) of any attribution among 34 patients with indolent lymphoma were petechiae/purpura/contusion (24%), upper respiratory tract infection (URTI) (21%), nausea (18%) and pyrexia (15%). The most frequently reported grade 3 or greater AEs (occurring in ≥5% of patients) of any attribution were anemia (9%), neutropenia (9%), urinary tract infection (6%), and abdominal pain (6%). Serious AEs were reported in 11 patients (32%). Of those, four patients had serious AEs that were considered possibly related to zanubrutinib, including one case each of nausea, urinary tract infection, diarrhea, and creatinine increase.

The most frequent AEs (occurring in ≥15% of patients) of any attribution among 65 patients with aggressive lymphoma were petechiae/purpura/contusion (25%), diarrhea (23%), constipation (22%), fatigue (18%), URTI (18%), anemia (17%), cough (15%), pyrexia (15%), and thrombocytopenia (15%). The most frequently reported grade 3 or greater AEs (occurring in ≥5% of patients) of any attribution were anemia (11%), neutropenia (9%), thrombocytopenia (9%), and pneumonia (6%). Serious AEs were reported in 26 patients (40%). Of those, three patients had serious AEs that were considered possibly related to zanubrutinib, including one case each of peripheral edema and joint effusion (occurring in the same patient), pneumonia, and pneumonitis.

At the time of data cutoff, 26 patients with indolent lymphoma including 17 patients with FL and nine patients with MZL were evaluable for efficacy. In patients with FL, the overall response rate (ORR) was 41% with complete responses (CRs) in 18% and partial responses (PRs) in 24% of patients. Stable disease (SD) was observed in 41% of patients. Progressive disease (PD) was observed in one patient. In patients with MZL, the ORR was 78% with no CR, and PRs in 78% of patients. SD was observed in 22% of patients. No PD was observed.

Fifty-eight patients with aggressive lymphoma including 26 patients with DLBCL and 32 patients with MCL were evaluable for efficacy. In patients with DLBCL, the ORR was 31% with CRs in 15% and PRs in 15% of patients. In patients with MCL, the ORR was 88% with CRs in 25% and PRs in 63% of patients.

About Zanubrutinib

Zanubrutinib (BGB-3111) is an investigational small molecule inhibitor of BTK that has demonstrated higher selectivity against BTK than ibrutinib (a BTK inhibitor currently approved by the U.S. Food and Drug Administration and the European Medicines Agency) based on biochemical assays, higher exposure than ibrutinib based on their respective Phase 1 experience in separate studies, and sustained 24-hour BTK occupancy in both the peripheral blood and lymph node compartments.