New Interim Data in RRMM Patients With Extramedullary Disease from the Pivotal Phase 2 Horizon-study Presented at International Myeloma Workshop

On September 15, 2019 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported updated interim efficacy and safety data from the ongoing pivotal Phase 2 study, HORIZON, at the 17th International Myeloma Workshop (IMW) meeting in Boston, Massachusetts, USA (Press release, Oncopeptides, SEP 15, 2019, View Source [SID1234539502]). The data focused on patients with extramedullary disease (EMD) and was presented in an oral presentation by Professor Paul G. Richardson. Oncopeptides will host a conference call to review this data on Monday, September 16, 2019, at. 08.30 (CET).

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The results presented at IMW 2019 by Professor Paul G Richardson in the plenary session "Late Breaking Abstracts" under the title: Activity of Melflufen in RRMM Patients with Extramedullary Disease in the Phase 2 Study (OP-106 HORIZON) – Promising Results in a High-Risk Population, are based on a data cut in the HORIZON study dated July 30, 2019 when 136 patients were treated compared to previously reported data including 121 patients. Based on literature, HORIZON represents the largest clinical cohort to date of myeloma patients with extra medullary disease (EMD).

Relapsed-refractory multiple myeloma (RRMM) patients with extramedullary disease (EMD) is a very difficult to treat patient population. In recent single-agent studies (with or without steroids) including RRMM patients with EMD have shown overall response rates (ORR) between 0 – 17%. Only daratumumab has shown any relevant clinical activity with a response rate of 17% in daratumumab naïve myeloma patients with EMD.

In the HORIZON study, an overall response rate of 23% was achieved with treatment with melflfufen and dexamethasone in RRMM patients with EMD. These patients were mainly penta refractory (had undergone at least five prior treatments and were resistant to at least one proteasome inhibitor, one IMiD, and anti-CD38 treatment). The EMD patients who responded to the treatment had a median survival (OS) of 18.5 months compared to a median survival of 5.1 months for the EMD patients who did not respond to treatment. The difference observed suggests a clear clinical benefit for these patients.

Professor Paul G Richardson comments
"We are experiencing a significant increase in the number of relapsed and refractory myeloma patients with extramedullary disease and without effective treatment options. The observation in the HORIZON trial, that melflufen shows activity in these patients and may impact favorably on survival outcome, is very encouraging.

"In my view, given the relative lack of therapeutic alternatives for these patients, moving rapidly into melflufen-based combination strategies in patients with extramedullary disease, as well as exploring earlier lines of therapy, should be the next priority", said Professor Paul G Richardson, Professor of Medicine at Harvard Medical School and Clinical Program Leader, Director of Clinical Research at the Jerome Lipper Multiple Myeloma Center Dana-Farber Cancer Institute in Boston, Massachusetts, USA.

Overall Conclusions From the Presentation

As of July 30, 2019, 136 patients had been treated in HORIZON with melflufen and dexamethasone. 44 out of the 136 patients had confirmed EMD diagnosis at baseline
91% of EMD patients were triple-class refractory and 73% penta refractory
Similar ORR in non-EMD and EMD patients with an ORR of 27% and 23% respectively
Response rates in EMD patients was seemingly higher than historical studies
Median OS was 5.8 months for patients with EMD and 11.6 months for patients without EMD
Median OS in EMD responders and non-responders was 18.5 months and 5.1 months, respectively
Median OS in non-EMD responders and non-responders was 17.2 months and 8.5 months, respectively
The treatment was generally well tolerated, with manageable toxicity with a low overall incidence of nonhematologic Adverse Events (AE) including infections; no treatment-related deaths
The full presentation from IMW is available on the company’s website under:
www.oncopeptides.com / Investors&media / Presentations / IMW 2019

Conference call for investors, analysts and the media
CEO Jakob Lindberg will review of the results presented at IMW, on Monday September 16, 2019 at 08:30 (CET).
The conference call will also be streamed via a link on the company website: www.oncopeptides.com.

Phone numbers for participants from:
Sweden: +46 8 505 583 68
Europe: +44 3333 009 271
USA: +1 833 823 05 87

The information in the press release is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person above, on September 15, 2019 at 14.30 (CET).

About the OP-106 HORIZON study
Patient recruitment in the HORIZON study is ongoing. The interim data presented at IMW 2019 is based on a data cut-off dated July 30, 2019, with 136 patients treated. The goal is to include 150 patients in the study. The patients in the study are refractory to pomalidomide and/or daratumumab after failing on immunomodulatory drugs (IMiDs) and proteasome inhibitors (PIs).

Oncopeptides has initiated preparations for submitting a New Drug Application (NDA) for accelerated approval of melflufen in the US based on the available HORIZON data. The detailed plan for the filing process is still under development, but Oncopeptides currently targets to submit the application to FDA during the first quarter of 2020. This could then lead to the first melflufen market approval in the US in 2020.

More information can be found at: View Source;rank=2

About Melflufen
Melflufen is a lipophilic peptide-conjugated alkylator that rapidly delivers a highly cytotoxic payload into myeloma cells through peptidase activity. It belongs to the novel class Peptidase Enhanced Cytotoxics (PEnC), which is a family of lipophilic peptides that exhibit increased activity via peptidase cleavage and have the potential to treat many cancers. Peptidases play a key role in protein homeostasis and feature in cellular processes such as cell-cycle progression and programmed cell death. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and immediately cleaved by peptidases to deliver an entrapped hydrophilic alkylator payload. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the peptidase cleavage, and induces irreversible DNA damage and apoptosis. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies.

New DARZALEX® (daratumumab) Data from GRIFFIN Study Show High Response Rate and Depth of Response in Patients with Newly Diagnosed Multiple Myeloma Who are Transplant-Eligible

On September 15, 2019 The Janssen Pharmaceutical Companies of Johnson & Johnson reported results from the randomized Phase 2 GRIFFIN (MMY2004) study showing that the addition of DARZALEX (daratumumab) to bortezomib, lenalidomide and dexamethasone (VRd) induced higher response rates in newly diagnosed patients with multiple myeloma who were eligible for high-dose therapy and autologous stem cell transplantation (ASCT) compared with VRd alone (Press release, Johnson & Johnson, SEP 15, 2019, View Source [SID1234539501]). The data, selected as a late-breaking abstract, were presented at the 17th International Myeloma Workshop (IMW) meeting in Boston.

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"The GRIFFIN study is the second randomized study to investigate the benefit of daratumumab for patients with newly diagnosed multiple myeloma who are eligible for a transplant, and the first in combination with lenalidomide for this population," said Peter M. Voorhees, M.D., GRIFFIN principal investigator at Levine Cancer Institute. "This study adds to the growing body of evidence for the addition of daratumumab to proteasome inhibitor/immunomodulatory combination therapy in the transplant setting."

Results from the Phase 2 GRIFFIN study showed that by the end of six cycles of therapy and transplant, a greater percentage of patients receiving DARZALEX-VRd achieved the primary endpoint of stringent complete response (sCR) compared with VRd alone (42 percent vs. 32 percent, respectively; Odds Ratio [OR] = 1.57; 95 percent confidence interval [CI], 0.87-2.82; P=0.1359), meeting the prespecified 2-sided alpha of 0.2 for a positive study.1 The addition of DARZALEX to VRd resulted in higher rates of overall response (99 percent vs. 92 percent), very good partial response or better (91 percent vs. 73 percent) and complete response (CR) or better (52 percent vs. 42 percent) compared with VRd alone, respectively.1 Notably, the rate of minimal residual disease (MRD) negativity among patients achieving a CR or better more than doubled in the DARZALEX-VRd arm compared with VRd alone (59 percent vs. 24 percent).1 The most common (≥10 percent) Grade 3/4 treatment-emergent adverse events (TEAEs) for DARZALEX-VRd were neutropenia (32 percent), lymphopenia (23 percent), thrombocytopenia (16 percent) and leukopenia (15 percent).1 Grade 1/2 infections occurred more frequently in the DARZALEX-VRd arm, but there was no difference in the rate of Grade 3/4 infections between the DARZALEX-VRd and VRd arms.1 Infusion-related reactions (IRRs) occurred in 41 percent of patients who received DARZALEX-VRd, which were primarily Grade 1/2 and during the initial infusion.1

"This primary analysis of the GRIFFIN study builds on the safety and efficacy data in the initial group of 16 patients presented at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting," said Andree Amelsberg, M.D., MBA, Vice President, Oncology Medical Affairs, Janssen Biotech, Inc. "It provides further support for evaluation of DARZALEX in the transplant-eligible patient population, which is important as we continue our work to discover new therapeutic approaches to improve outcomes for patients."

In addition to GRIFFIN, data from the Phase 2 PLEIADES (MMY2040) study, presented during an oral session at the IMW meeting, showed that an investigational DARZALEX subcutaneous (SC) formulation delivered in combination with standard-of-care treatment regimens showed similar clinical activity and safety to DARZALEX intravenous (IV) regimens.2 The study is the first to evaluate SC DARZALEX in different combination regimens for patients with newly diagnosed multiple myeloma as well as those who were relapsed/refractory to current treatment options.2

"We’re excited about the opportunity to progress the innovation represented by the DARZALEX subcutaneous formulation, which can be administered over the course of minutes and has the potential to offer a reduction in infusion-related events as compared to the approved intravenous formulation," said Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Oncology, Janssen Research & Development, LLC. "Data from the PLEIADES study demonstrates that the DARZALEX subcutaneous formulation can also be safely administered in combination with standard backbone regimens used for treatment naïve and relapsed/refractory patients with multiple myeloma. It has been included in our recent submission of a Biologics License Application to the U.S. Food and Drug Administration seeking approval of a new DARZALEX subcutaneous formulation for patients with multiple myeloma."

Results from the PLEIADES study showed the median duration of administration was 5 minutes across all cohorts, compared with more than 3 hours with IV infusions.2 Rates of any grade IRRs and injection-site reactions were 7.5 percent across all cohorts, with one Grade 3 IRR in one cohort and no Grade 4 IRRs.2 Grade 3/4 TEAEs were reported by more than 50 percent of patients across cohorts, and TEAEs leading to treatment discontinuation were less than 8 percent in all cohorts.2 Safety profiles in all cohorts were consistent with the IV administration of DARZALEX in combination with these regimens.2

About the GRIFFIN Trial1
The open-label Phase 2 GRIFFIN (MMY2004) study has enrolled and treated adult patients, age 29-70 years, with newly diagnosed multiple myeloma who were eligible for high-dose therapy/ASCT, including 16 patients in a safety run-in phase and more than 200 patients in the subsequent randomized portion of the study. During induction (Cycles 1-4) and consolidation (Cycles 5-6) in the randomized part of the study, all patients received 25 mg of lenalidomide orally on Days 1-14, 1.3 mg/m2 of bortezomib subcutaneously on Days 1, 4, 8 and 11, and 20 mg of dexamethasone on Days 1, 2, 8, 9, 15 and 16 every 21 days. In the DARZALEX-VRd arm, DARZALEX 16 mg/kg IV was given on Days 1, 8 and 15 of Cycles 1-4 and on Day 1 of Cycles 5-6. During maintenance (Cycles 7-32), all patients received 10 mg daily of lenalidomide (15 mg beginning at Cycle 10 if tolerated) on Days 1-21 every 28 days. In the DARZALEX-VRd arm, DARZALEX 16 mg/kg IV was given every 56 days; this has been amended to give DARZALEX every 28 days based on emerging pharmacokinetic data. Maintenance therapy with lenalidomide may be continued beyond Cycle 32 per local standard of care.

About the PLEIADES Trial2
The non-randomized, open-label, parallel assignment Phase 2 PLEIADES (MMY2040) study included 199 adults with either newly diagnosed or relapsed/refractory multiple myeloma. Patients with newly diagnosed multiple myeloma were treated with 1,800 mg of the SC formulation in combination with either bortezomib, lenalidomide and dexamethasone (D-VRd) or bortezomib, melphalan and prednisone (D-VMP). Patients with relapsed or refractory disease were treated with 1,800 mg of the SC formulation plus lenalidomide and dexamethasone (D-Rd). The primary endpoint for the D-VMP and D-Rd cohorts was overall response rate. The primary endpoint for the D-VRd cohort was very good partial response or better rate. An additional cohort of patients with relapsed and refractory multiple myeloma treated with daratumumab plus carfilzomib and dexamethasone was subsequently added to the study.

About DARZALEX (daratumumab)
DARZALEX (daratumumab), the first CD38-directed antibody approved anywhere in the world, is the only CD38-directed antibody approved to treat multiple myeloma.3 CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease.4 DARZALEX binds to CD38 and inhibits tumor cell growth causing myeloma cell death.3 DARZALEX may also have an effect on normal cells.3 DARZALEX is being evaluated in a comprehensive clinical development program across a range of treatment settings in multiple myeloma, such as in frontline and relapsed settings.5,6,7,8,9,10,11,12 Additional studies are ongoing or planned to assess its potential in other malignant and pre-malignant hematologic diseases in which CD38 is expressed, such as smoldering myeloma.13,14

In the U.S., DARZALEX received initial FDA approval in November 2015 as a monotherapy for patients with multiple myeloma who have received at least three prior lines of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent, or who are double refractory to a PI and an immunomodulatory agent.15 DARZALEX received additional approvals in November 2016 in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy.16 In June 2017, DARZALEX received approval in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a PI.17 In May 2018, DARZALEX received approval in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for ASCT, making it the first monoclonal antibody approved for newly diagnosed patients with this disease.18 In March 2019, a supplemental Biologics License Application was submitted to the U.S. FDA seeking approval of DARZALEX in combination with bortezomib, thalidomide and dexamethasone for newly diagnosed patients with multiple myeloma who are eligible for ASCT based on the Phase 3 CASSIOPEIA study. Most recently, in June 2019, DARZALEX received approval in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are transplant ineligible.19

In August 2012, Janssen Biotech, Inc. entered into a global license and development agreement with Genmab A/S, which granted Janssen an exclusive license to develop, manufacture and commercialize DARZALEX.20 For the full U.S. Prescribing Information, please visit www.DARZALEX.com.

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.21,22 When damaged, these plasma cells rapidly spread and replace normal cells with tumors in the bone marrow.20,21 In 2019, it is estimated that 32,110 people will be diagnosed and 12,960 will die from the disease in the U.S.23 While some patients with multiple myeloma have no symptoms, most patients are diagnosed due to symptoms, which can include bone fracture or pain, low red blood counts, tiredness, high calcium levels, kidney problems or infections.24

IMPORTANT SAFETY INFORMATION3

CONTRAINDICATIONS

DARZALEX (daratumumab) is contraindicated in patients with a history of severe hypersensitivity (eg, anaphylactic reactions) to daratumumab or any of the components of the formulation.

WARNINGS AND PRECAUTIONS

Infusion Reactions – DARZALEX can cause severe and/or serious infusion reactions, including anaphylactic reactions. In clinical trials, approximately half of all patients experienced an infusion reaction. Most infusion reactions occurred during the first infusion and were Grade 1-2. Infusion reactions can also occur with subsequent infusions. Nearly all reactions occurred during infusion or within 4 hours of completing DARZALEX. Prior to the introduction of post-infusion medication in clinical trials, infusion reactions occurred up to 48 hours after infusion. Severe reactions have occurred, including bronchospasm, hypoxia, dyspnea, hypertension, laryngeal edema, and pulmonary edema. Signs and symptoms may include respiratory symptoms, such as nasal congestion, cough, throat irritation, as well as chills, vomiting, and nausea. Less common symptoms were wheezing, allergic rhinitis, pyrexia, chest discomfort, pruritus, and hypotension.

Pre-medicate patients with antihistamines, antipyretics, and corticosteroids. Frequently monitor patients during the entire infusion. Interrupt infusion for reactions of any severity and institute medical management as needed. Permanently discontinue therapy if an anaphylactic reaction or life-threatening (Grade 4) reaction occurs and institute appropriate emergency care. For patients with Grade 1, 2, or 3 reactions, reduce the infusion rate when re-starting the infusion.

To reduce the risk of delayed infusion reactions, administer oral corticosteroids to all patients following DARZALEX infusions. Patients with a history of chronic obstructive pulmonary disease may require additional post-infusion medications to manage respiratory complications. Consider prescribing short- and long-acting bronchodilators and inhaled corticosteroids for patients with chronic obstructive pulmonary disease.

Interference with Serological Testing – Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive Indirect Antiglobulin Test (Indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab infusion. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient’s serum. The determination of a patient’s ABO and Rh blood type are not impacted. Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX. Type and screen patients prior to starting DARZALEX.

Neutropenia and Thrombocytopenia – DARZALEX may increase neutropenia and/or thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to the manufacturer’s prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. DARZALEX dose delay may be required to allow recovery of neutrophils and/or platelets. No dose reduction of DARZALEX is recommended. Consider supportive care with growth factors for neutropenia or transfusions for thrombocytopenia.

Interference with Determination of Complete Response – Daratumumab is a human IgG kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some patients with IgG kappa myeloma protein.

Adverse Reactions – The most frequently reported adverse reactions (incidence ≥20%) were: infusion reactions, neutropenia, thrombocytopenia, fatigue, asthenia, nausea, diarrhea, constipation, decreased appetite, vomiting, muscle spasms, arthralgia, back pain, pyrexia, chills, dizziness, insomnia, cough, dyspnea, peripheral edema, peripheral sensory neuropathy, bronchitis, pneumonia and upper respiratory tract infection.

DARZALEX in combination with lenalidomide and dexamethasone (DRd): The most frequent (≥20%) adverse reactions for newly diagnosed or relapsed refractory patients were, respectively, infusion reactions (41%, 48%), diarrhea (57%, 43%), nausea (32%, 24%), fatigue (40%, 35%), pyrexia (23%, 20%), upper respiratory tract infection (52%, 65%), muscle spasms (29%, 26%), dyspnea (32%, 21%), and cough (30%, 30%). In newly diagnosed patients, constipation (41%), peripheral edema (41%), back pain (34%), asthenia (32%), bronchitis (29%), pneumonia (26%), decreased appetite (22%), and peripheral sensory neuropathy (24%) were also reported. In newly diagnosed patients, serious adverse reactions (≥2% compared to Rd) were dehydration (2%), bronchitis (4%), and pneumonia (15%), and treatment-emergent Grade 3-4 hematology laboratory abnormalities (≥20%) were leukopenia (35%), neutropenia (56%), and lymphopenia (52%). In relapsed/refractory patients, serious adverse reactions (≥2% compared to Rd) were pneumonia (12%), upper respiratory tract infection (7%), influenza (3%), and pyrexia (3%), and treatment-emergent Grade 3-4 hematology laboratory abnormalities (≥20%) were neutropenia (53%) and lymphopenia (52%).

DARZALEX in combination with bortezomib, melphalan, and prednisone (DVMP): The most frequently reported adverse reactions (≥20%) were upper respiratory tract infection (48%), infusion reactions (28%), and peripheral edema (21%). Serious adverse reactions (≥2% compared to the VMP arm) were pneumonia (11%), upper respiratory tract infection (5%), and pulmonary edema (2%). Treatment-emergent Grade 3-4 hematology laboratory abnormalities (≥20%) were lymphopenia (58%), neutropenia (44%), and thrombocytopenia (38%).

DARZALEX in combination with bortezomib and dexamethasone (DVd): The most frequently reported adverse reactions (≥20%) were peripheral sensory neuropathy (47%), infusion reactions (45%), upper respiratory tract infection (44%), diarrhea (32%), cough (27%), peripheral edema (22%), and dyspnea (21%). The overall incidence of serious adverse reactions was 42%. Serious adverse reactions (≥2% compared to Vd) were upper respiratory tract infection (5%), diarrhea (2%), and atrial fibrillation (2%). Treatment-emergent Grade 3-4 hematology laboratory abnormalities (≥20%) were lymphopenia (48%) and thrombocytopenia (47%).

DARZALEX in combination with pomalidomide and dexamethasone (DPd): The most frequent adverse reactions (>20%) were fatigue (50%), infusion reactions (50%), upper respiratory tract infection (50%), cough (43%), diarrhea (38%), constipation (33%), dyspnea (33%), nausea (30%), muscle spasms (26%), back pain (25%), pyrexia (25%), insomnia (23%), arthralgia (22%), dizziness (21%), and vomiting (21%). The overall incidence of serious adverse reactions was 49%. Serious adverse reactions reported in ≥5% of patients included pneumonia (7%). Treatment-emergent Grade 3-4 hematology laboratory abnormalities (≥20%) were anemia (30%), neutropenia (82%), and lymphopenia (71%).

DARZALEX as monotherapy: The most frequently reported adverse reactions (≥20%) were infusion reactions (48%), fatigue (39%), nausea (27%), back pain (23%), pyrexia (21%), cough (21%), and upper respiratory tract infection (20%). The overall incidence of serious adverse reactions was 33%. The most frequent serious adverse reactions were pneumonia (6%), general physical health deterioration (3%), and pyrexia (3%). Treatment-emergent Grade 3-4 hematology laboratory abnormalities (≥20%) were lymphopenia (40%) and neutropenia (20%).

Oncopeptides Announces Two Poster Presentations at the International Myeloma Workshop

On September 14, 2019 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that our two poster presentations have been made public at the 17th International Myeloma Workshop (IMW) meeting in Boston, Massachusetts, USA (Press release, Oncopeptides, SEP 14, 2019, View Source [SID1234539499]).

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The posters presented at IMW
The first poster presentation was held during Poster Session I, Multiple Myeloma Genomics – under the title: Aminopeptidase gene expression in myeloma.

The second poster presentation was held during Poster Session II, Treatment of Previously Treated Myeloma –under the title: Quality of life treatment for relapsed/refractory multiple myeloma, a systematic review.

The posters are available on the company webpage under:
www.oncopeptides.com / Investors&Media / Presentations / IMW 2019

For further information, please contact:
Jakob Lindberg, CEO of Oncopeptides
E-mail: [email protected]
Telephone: +46 8 615 20 40

Rein Piir, Head of Investor Relations at Oncopeptides
E-mail: [email protected]
Cell phone: +46 70 853 72 92

This information was submitted for publication at 13.00 CET September 14, 2019.

Karyopharm Announces XPOVIO™ (Selinexor) Presentations at the 17th International Myeloma Workshop

On September 13, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported that five abstracts highlighting clinical data for XPOVIO (selinexor) will be presented at the 17th International Myeloma Workshop (IMW) taking place September 12-15, 2019 in Boston (Press release, Karyopharm, SEP 13, 2019, View Source [SID1234539498]).

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A key abstract at this meeting is a poster presentation titled "Outcomes of Triple Class Refractory Penta-Exposed Multiple Myeloma (MM)," (Cornell, et al) which compares the overall survival (OS) rate from the retrospective MAMMOTH study (Leukemia, 2019) which evaluated outcomes from patients with relapsed or refractory multiple myeloma after their disease became refractory to CD38 monoclonal antibodies with a similarly-matched cohort of patients from Karyopharm’s Phase 2b STORM study. Patients in STORM, who received selinexor and dexamethasone as first line of therapy after their disease became triple class refractory (n=64) as compared with matched patients receiving currently available therapies from the MAMMOTH cohort (n=128) showed an unadjusted hazard ratio (HR) for death of 0.64 (p=0.043), while an adjusted analysis, which takes into consideration differences in baseline characteristics between the two groups, showed a HR of 0.55 (p=0.009) (see Figure 1 below). The median OS on selinexor-dexamethasone was 11.1 months and on MAMMOTH was 9.2 months. Patients in the MAMMOTH study received a single (n=6, 4.7%) or combination of two or more anti-multiple myeloma agents (n=122, 95.3%). A PDF copy of this poster will be available here once it has been presented at the meeting.

Additional abstracts to be presented include an oral presentation highlighting updated data from the Phase 1b/2 STOMP study (White, et al) evaluating selinexor and dexamethasone in combination with Revlimid (lenalidomide) for the treatment of patients with relapsed or refractory multiple myeloma, and three additional posters (Gavriatopoulou et al, Tariq et al and Vogl et al) which describe various analyses from the Phase 2b STORM study evaluating XPOVIO in patients with heavily pretreated multiple myeloma.

"The multiple data presentations at IMW continue to reinforce our belief in the potential of XPOVIO as a new therapeutic option for patients with relapsed or refractory multiple myeloma," said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. "There is a growing need for novel treatment approaches for patients with relapsed or refractory multiple myeloma. The unique mechanism of action of XPOVIO, the first and only oral nuclear export inhibitor approved in the U.S., provides physicians with a new therapeutic option for patients with heavily pretreated multiple myeloma and we continue to be excited about future development of this novel agent."

Details for the IMW 2019 presentations are as follows:

Oral Presentation

Title: Safety and Efficacy of the Combination of Selinexor, Lenalidomide and Dexamethasone (SRd) in Patients with Relapsed/Refractory Multiple Myeloma (RRMM)
Lead author: Darrell White, Dalhouise University
Abstract #: AB353
Session: Multiple Myeloma Novel Agents
Date and Time: Saturday, September 14, 2019; 4:45 – 5:00 PM ET
Location: Hynes Ballroom

Poster Presentations

Title: Outcomes of Triple Class Refractory Penta-Exposed Multiple Myeloma (MM)
Lead author: Robert Cornell, Vanderbilt University Medical Center
Abstract #: 866
Session: Multiple Myeloma Novel Agents – Poster Session I
Date and Time: Friday, September 13, 2019; 6:30 – 8:00 PM ET
Location: Hynes Auditorium

Title: Effect of Age on the Safety and Efficacy of Selinexor in Patients with Relapsed Refractory Multiple Myeloma: A Post-hoc Analysis of the STORM Study
Lead author: Maria Gavriatopoulou, National and Kapodistrian University of Athens School of Medicine
Abstract #: 582
Session: Multiple Myeloma Novel Agents – Poster Session I
Date and Time: Friday, September 13, 2019; 6:30 – 8:00 PM ET
Location: Hynes Auditorium

Title: Efficacy and Safety of Selinexor for Heavily Pretreated Multiple Myeloma Treatment – A Systematic Review
Lead author: Muhammad Junaid Tariq, John H. Stroger, Jr. Hospital of Cook County
Abstract #: 617
Session: Multiple Myeloma Novel Agents – Poster Session I
Date and Time: Friday, September 13, 2019; 6:30 – 8:00 PM ET
Location: Hynes Auditorium

Title: Improvements in Renal Function with Selinexor in Relapsed/Refractory Multiple Myeloma: Post-hoc Analyses from the STORM Study
Lead author: Dan Vogl, Abramson Cancer Center, University of Pennsylvania
Abstract #: 592
Session: Multiple Myeloma Novel Agents – Poster Session I
Date and Time: Friday, September 13, 2019; 6:30 – 8:00 PM ET
Location: Hynes Auditorium

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. In addition to receiving accelerated FDA approval of XPOVIO in July 2019 in combination with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody, Karyopharm has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for conditional approval of selinexor. Selinexor is also being studied in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). In 2018, Karyopharm reported positive top-line results from the Phase 2b SADAL study evaluating selinexor in patients with relapsed or refractory DLBCL after at least two prior multi-agent therapies and who are ineligible for transplantation, including high dose chemotherapy with stem cell rescue. Selinexor has received Fast Track designation from the FDA for the patient population evaluated in the SADAL study. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), as a potential backbone therapy in combination with approved therapies (STOMP), in liposarcoma (SEAL), in recurrent gliomas (KING) and in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

IMPORTANT SAFETY INFORMATION

Thrombocytopenia

XPOVIO can cause thrombocytopenia, leading to potentially fatal hemorrhage. Thrombocytopenia was reported as an adverse reaction in 74% of patients, and severe (Grade 3-4) thrombocytopenia occurred in 61% of patients treated with XPOVIO. The median time to onset of the first event was 22 days. Bleeding occurred in 23% of patients with thrombocytopenia, clinically significant bleeding occurred in 5% of patients with thrombocytopenia and fatal hemorrhage occurred in <1% of patients.

Monitor platelet counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment. Institute platelet transfusion and/or other treatments as clinically indicated. Monitor patients for signs and symptoms of bleeding and evaluate promptly. Interrupt and/or reduce dose, or permanently discontinue based on severity of adverse reaction.

Neutropenia

XPOVIO can cause neutropenia, potentially increasing the risk of infection. Neutropenia was reported as an adverse reaction in 34% of patients, and severe (Grade 3-4) neutropenia occurred in 21% of patients treated with XPOVIO. The median time to onset of the first event was 25 days. Febrile neutropenia was reported in 3% of patients.

Obtain neutrophil counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment. Monitor patients for signs and symptoms of concomitant infection and evaluate promptly. Consider supportive measures including antimicrobials for signs of infection and use of growth factors (e.g., G-CSF). Interrupt and/or reduce dose, or permanently discontinue based on severity of adverse reaction.

Gastrointestinal Toxicity

Gastrointestinal toxicities occurred in patients treated with XPOVIO.

Nausea/Vomiting

Nausea was reported as an adverse reaction in 72% of patients, and Grade 3 nausea occurred in 9% of patients treated with XPOVIO. The median time to onset of the first nausea event was 3 days.

Vomiting was reported in 41% of patients, and Grade 3 vomiting occurred in 4% of patients treated with XPOVIO. The median time to onset of the first vomiting event was 5 days.

Provide prophylactic 5-HT3 antagonists and/or other anti-nausea agents, prior to and during treatment with XPOVIO. Manage nausea/vomiting by dose interruption, reduction, and/or discontinuation. Administer intravenous fluids and replace electrolytes to prevent dehydration in patients at risk. Use additional anti-nausea medications as clinically indicated.

Diarrhea

Diarrhea was reported as an adverse reaction in 44% of patients, and Grade 3 diarrhea occurred in 6% of patients treated with XPOVIO. The median time to onset of diarrhea was 15 days.

Manage diarrhea by dose modifications and/or standard anti-diarrheal agents; administer intravenous fluids to prevent dehydration in patients at risk.

Anorexia/Weight Loss

Anorexia was reported as an adverse reaction in 53% of patients, and Grade 3 anorexia occurred in 5% of patients treated with XPOVIO. The median time to onset of anorexia was 8 days.

Weight loss was reported as an adverse reaction in 47% of patients, and Grade 3 weight loss occurred in 1% of patients treated with XPOVIO. The median time to onset of weight loss was 15 days.

Monitor patient weight at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment. Manage anorexia and weight loss with dose modifications, appetite stimulants, and nutritional support.

Hyponatremia

XPOVIO can cause hyponatremia; 39% of patients treated with XPOVIO experienced hyponatremia, 22% of patients experienced Grade 3 or 4 hyponatremia. The median time to onset of the first event was 8 days.

Monitor sodium level at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment. Correct sodium levels for concurrent hyperglycemia (serum glucose >150 mg/dL) and high serum paraprotein levels. Treat hyponatremia per clinical guidelines (intravenous saline and/or salt tablets), including dietary review. Interrupt and/or reduce dose, or permanently discontinue based on severity of adverse reaction.

Infections

In patients receiving XPOVIO, 52% of patients experienced any grade of infection. Upper respiratory tract infection of any grade occurred in 21%, pneumonia in 13%, and sepsis in 6% of patients. Grade ≥3 infections were reported in 25% of patients, and deaths resulting from an infection occurred in 4% of patients. The most commonly reported Grade ≥3 infections were pneumonia in 9% of patients, followed by sepsis in 6%. The median time to onset was 54 days for pneumonia and 42 days for sepsis. Most infections were not associated with neutropenia and were caused by non-opportunistic organisms.

Neurological Toxicity

Neurological toxicities occurred in patients treated with XPOVIO.

Neurological adverse reactions including dizziness, syncope, depressed level of consciousness, and mental status changes (including delirium and confusional state) occurred in 30% of patients, and severe events (Grade 3-4) occurred in 9% of patients treated with XPOVIO. Median time to the first event was 15 days.

Optimize hydration status, hemoglobin level, and concomitant medications to avoid exacerbating dizziness or mental status changes.

Embryo-Fetal Toxicity

Based on data from animal studies and its mechanism of action, XPOVIO can cause fetal harm when administered to a pregnant woman. Selinexor administration to pregnant animals during organogenesis resulted in structural abnormalities and alterations to growth at exposures below those occurring clinically at the recommended dose.

Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with a female partner of reproductive potential to use effective contraception during treatment with XPOVIO and for 1 week after the last dose.

ADVERSE REACTIONS

The most common adverse reactions (incidence ≥20%) are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea, and upper respiratory tract infection.

The treatment discontinuation rate due to adverse reactions was 27%; 53% of patients had a reduction in the XPOVIO dose, and 65.3% had the dose of XPOVIO interrupted. The most frequent adverse reactions requiring permanent discontinuation in 4% or greater of patients who received XPOVIO included fatigue, nausea, and thrombocytopenia. The rate of fatal adverse reactions was 8.9%.

Amgen To Present At The Bank of America Merrill Lynch Global Healthcare Conference

On September 13, 2019 Amgen (NASDAQ:AMGN) reported that it will present at the Bank of America Merrill Lynch Global Healthcare Conference at 9:45 a.m. British Summer Time on Wednesday, Sept. 18, 2019, in London (Press release, Amgen, SEP 13, 2019, View Source [SID1234539495]). David W. Meline, executive vice president and chief financial officer at Amgen, will present at the conference. Live audio of the presentation can be accessed from the Events Calendar on Amgen’s website, www.amgen.com, under Investors. A replay of the webcast will also be available on Amgen’s website for at least 90 days following the event.

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