Xencor Reports Fourth Quarter and Full Year 2018 Financial Results

On February 25, 2019 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of autoimmune disease, asthma and allergic diseases, and cancer, reported financial results for the fourth quarter and full year ended December 31, 2018 and provided a review of recent business and clinical highlights (Press release, Xencor, FEB 25, 2019, View Source [SID1234533628]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"In 2018, we rapidly expanded our bispecific antibody oncology pipeline to position ourselves at the forefront of this growing field, starting two Phase 1 programs and submitting INDs for an additional two for which we will soon begin dosing patients. We also reported encouraging initial data from the Phase 1 study of XmAb14045 in patients with acute myeloid leukemia, and we are now working with the FDA to resolve the partial clinical hold on the study. To end the year, our partner Alexion announced the early U.S. marketing approval of Ultomiris for adult patients with PNH, making it the first approved antibody with XmAb technology," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "Earlier this month we entered into a co-development partnership and profit share with Genentech for our first bispecific Fc engineered cytokine, XmAb24306, and our IL-15 program, and we are planning an extensive clinical program to explore combination agents. We will support Genentech’s efforts to submit an IND for XmAb24306 in the second half of 2019 and plan to continue the expansion of our oncology pipeline this year."

Dr. Dahiyat added, "Given our focus on the growing opportunities provided by our bispecific Fc technology, we do not plan to start late-stage clinical development for obexelimab, which has demonstrated disease modifying activity in lupus and IgG4-related disease, prior to securing a partner. This approach will allow us to maximize the drug candidate’s potential for the broadest set of patients."

Recent Business Highlights and Upcoming Clinical Plans

XmAb14045: XmAb14045 is a CD123 x CD3 bispecific antibody being evaluated through a Phase 1 study in patients with relapsed or refractory acute myeloid leukemia and other CD123-expressing hematologic malignancies.

Partial Clinical Hold: On February 20, 2019, Xencor announced that the U.S. Food and Drug Administration (FDA) had placed a partial clinical hold on the Phase 1 study pending review of additional details regarding two patient deaths, safety and efficacy information across the study, and satisfactory review of amendments to the study protocol and related documents. One patient experienced cytokine release syndrome (CRS) after their first dose, the treatment of which was complicated by the patient’s decision to withdraw care. Another patient developed acute pulmonary edema following several doses of XmAb14045. Xencor is coordinating a response to the partial hold by the FDA and plans to continue development of XmAb14045 pending resolution of the partial hold.
Multiple Complete Remissions Achieved: In December 2018, initial data from the Phase 1 study, presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, indicated multiple complete remissions had been achieved with weekly dosing of XmAb14045 in this heavily-pretreated patient population. Cytokine release syndrome (CRS) was the most common toxicity occurring in 55% of patients. 6% of patients experienced Grade 3 or 4 CRS. CRS was more severe on the initial dose and was generally manageable with premedication. Additional adverse events consistent with CRS but not reported as such, including chills, fever, tachycardia, hypotension and hypertension within 24 hours of infusion, were reported in an additional 29% of patients. 28% of evaluable patients (n=5/18) achieved either complete remission (CR) or CR with incomplete hematologic recovery (CRi) at the two highest initial dose levels studied (1.3 and 2.3 mcg/kg weekly).
Collaboration for XmAb24306 and Novel IL-15 Cytokines with Genentech: In February 2019, Xencor entered into a research and license agreement with Genentech, a member of the Roche Group, to develop and commercialize novel IL-15 cytokine therapeutics, including XmAb24306, an IL-15/IL-15Rα cytokine complex engineered with Xencor’s bispecific Fc domain and Xtend Fc technology and Xencor’s most advanced preclinical cytokine program. Xencor will pay 45% of development costs and receive 45% of profits and losses. Genentech will commercialize medicines worldwide, and Xencor has the option to co-promote in the United States. Additionally, the companies will engage in a two-year research program to discover new IL-15 drug candidates, including ones targeted to specific immune cell populations. Xencor will receive $120 million upfront and will be eligible to receive up to $160 million in development milestones for the XmAb24306 program and up to $180 million in development milestones for each new IL-15 drug candidate. The agreement is subject to customary closing conditions, including Hart-Scott-Rodino clearance, and closing is expected to occur in the first quarter of 2019.

Oncology Pipeline: Xencor’s bispecific Fc domains are being used to develop several classes of novel drug candidates in oncology, including: CD3 bispecific antibodies, tumor microenvironment (TME) activator bispecific antibodies and cytokines. Xencor’s XmAb Fc domains confer long circulating half-lives, stability and ease of manufacture.

CD3 Bispecific Antibodies: Xencor’s initial bispecific antibody programs are tumor-targeted antibodies that contain both a tumor antigen binding domain and a cytotoxic T-cell binding domain (CD3). These bispecific antibodies activate T cells for highly potent and targeted killing of malignant cells. In January 2019, Xencor announced that as part of a strategic pipeline reprioritization, its partner Novartis decided to return its rights to develop and commercialize XmAb13676 (CD20 x CD3) and that the Company intends to continue development of XmAb13676 as planned. In addition to working toward resolution of the partial clinical hold on the Phase 1 study of XmAb14045, initial data from the Phase 1 studies of XmAb13676 in patients with B-cell malignancies and XmAb18087 (SSTR2 x CD3) in patients with neuroendocrine tumors or gastrointestinal stromal tumors, are expected in the second half of 2019.
TME Activator Bispecific Antibodies: Xencor’s bispecific pipeline includes a suite of TME activators that engage multiple, different targets, such as T-cell checkpoint or agonist receptors. Initial data from DUET-2, a Phase 1 study of XmAb20717 (PD-1 x CTLA-4) in patients with advanced solid tumors, are expected in the second half of 2019. Initiation of a Phase 1 study of XmAb23104 (PD-1 x ICOS) in patients with select advanced solid tumors and initiation of a Phase 1 study of XmAb22841 (CTLA-4 x LAG-3) in patients with select advanced solid tumors as a monotherapy and in combination with pembrolizumab are expected in the first half of 2019.
Cytokines: Xencor uses its bispecific Fc domain and Xtend technology to engineer cytokines that have potency tuned to improve therapeutic index and have longer half-life. The Company’s first cytokine candidate is XmAb24306, which is being co-developed with Genentech. IL-15 cytokines, like XmAb24306, may be promising candidates for oncology combination therapies, and under the Genentech Agreement, Xencor retained the right to perform clinical studies of collaboration products in combination with other therapeutic agents, subject to certain requirements. XmAb24306 is currently in IND-enabling studies, and the Company will support Genentech’s efforts to submit an IND application for this candidate in the second half of 2019.
Obexelimab (XmAb5871): Obexelimab is a first-in-class monoclonal antibody that targets CD19 with its variable domain and uses Xencor’s XmAb immune inhibitor Fc domain to target FcγRIIb, a receptor that inhibits B-cell function. Obexelimab has the potential to address a key unmet need in autoimmune diseases due to its combination of potent reversible B-cell inhibition without B-cell depletion, enabling the immune system to resume natural function once treatment is no longer needed. Phase 2 clinical studies have demonstrated the potential disease modifying ability of obexelimab in autoimmune indications such as systemic lupus erythematosus (SLE) and IgG4-related disease (IgG4-RD). Data from these studies demonstrate the potential of obexelimab in these and other B-cell mediated autoimmune indications. The Company is seeking to partner obexelimab with a partner that has the infrastructure and resources to continue late-stage development of obexelimab and maximize the potential of this drug candidate for a broad set of patient populations.

Partnered XmAb Programs: Eight pharmaceutical companies and the National Institutes of Health are advancing novel drug candidates either discovered at Xencor or that rely on Xencor’s proprietary XmAb technology. Several such programs are currently undergoing clinical testing, including MOR208, which is in Phase 3 development as a combination agent for the treatment of relapsed or refractory diffuse large B-cell lymphoma, and AMG 424, a CD38 x CD3 bispecific antibody, which Amgen announced had entered into a Phase 1 study for the treatment of patients with multiple myeloma in the third quarter of 2018. In the fourth quarter of 2018, Amgen announced that AMG 509, a bispecific antibody that is being developed for prostate cancer, is currently in preclinical development.

In December 2018, Ultomiris, the first antibody that incorporates an XmAb technology was approved by the FDA for commercial marketing. Ultomiris is a complement inhibitor indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) that was developed by Alexion, and it incorporates Xtend Fc technology which allows for a longer duration of action and less frequent dosing regimens compared to Soliris. Marketing authorizations that Alexion submitted to regulatory authorities in the EU and Japan are under review.

Fourth Quarter and Full Year Ended December 31, 2018 Financial Results

Effective January 1, 2018, Xencor adopted the new revenue recognition standard, Accounting Standard Codification 606 (ASC 606). In addition to adopting the standard for 2018, revenue reported for the prior period ending December 31, 2017 has been revised to reflect the new standard.

Cash, cash equivalents and marketable securities totaled $530.5 million as of December 31, 2018, compared to $363.3 million at December 31, 2017. The increase reflects net proceeds of $245.5 million from Xencor’s sale of additional stock in March 2018, partially offset by cash used to fund operating activities in the twelve months ended December 31, 2018.

Total revenue for the three- and twelve-month periods ended December 31, 2018 was $11.6 and $40.6 million, respectively, compared to $30.2 and $46.2 million of revenue reported for the same periods in 2017. Revenues in the three months ended December 31, 2018 were primarily milestone payments received from the Company’s Alexion collaboration, and revenues for the twelve months ended December 31, 2018 included milestone payments received from the Alexion collaboration and revenue recognized under the Company’s Novartis collaboration.

Research and development expenditures for the fourth quarter ended December 31, 2018 were $27.1 million, compared to $20.4 million for the same period in 2017. Total research and development expenditures for the year ended December 31, 2018 were $97.5 million, compared to $71.8 million for the same period in 2017. The increased research and development spending for the three and twelve months ended December 31, 2018 reflects additional spending on Xencor’s bispecific Fc technologies and its expanding pipeline of bispecific oncology candidates.

General and administrative expenses for the fourth quarter ended December 31, 2018 were $5.5 million, compared to $4.4 million in the same period in 2017. Total general and administrative expenditures for the year ended December 31, 2018 were $22.5 million, compared to $17.5 million for the same period in 2017. The increased spending on general and administrative expenses for the three and twelve months ended December 31, 2018 reflects increased compensation costs including increased stock-based compensation charges.

Non-cash, stock-based compensation expense for the year ended December 31, 2018 was $20.5 million, compared to $13.7 million for same period in 2017.

Net loss for the fourth quarter ended December 31, 2018 was $18.2 million, or $(0.32) on a fully diluted per share basis, compared to net income of $7.4 million, or $0.15 on a fully diluted per share basis, for the same period in 2017. The net loss reported for three months ended December 31, 2018 over the income for the same period in 2017 is primarily due to revenue recognized from Xencor’sNovartis and Amgen collaborations in 2017 compared to revenue recognized from Xencor’s Alexion collaboration in 2018. For the year ended December 31, 2018, net loss was $70.4 million, or $(1.31) on a fully diluted per share basis, compared to a net loss of $38.5 million, or $(0.82) on a fully diluted per share basis, for the same period in 2017. The increased loss for the year ended December 31, 2018 over amounts for the same period in 2017 is primarily due to increased spending in research and development and general and administrative in 2018.

The total shares outstanding were 56,279,542 as of December 31, 2018, compared to 47,002,488 as of December 31, 2017. The additional shares outstanding at December 31, 2018 reflect the 8,395,000 shares sold in Xencor’sMarch 2018 financing.

Financial Guidance
Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations into 2023. Xencor expects to end 2019 with approximately $575 to $600 million in cash, cash equivalents and marketable securities.

Conference Call and Webcast
Xencor will host a conference call today at 4:30 p.m. ET (1:30 p.m. PT) to discuss these fourth quarter and full year 2018 financial results and provide a corporate update.

The live call may be accessed by dialing (877) 359-9508 for domestic callers or (224) 357-2393 for international callers and referencing conference ID number 6482077. A live webcast of the conference call will be available online from the Investors section of the Company’s website at www.xencor.com. The webcast will be archived on the company’s website for 90 days.

Sorrento Therapeutics to Present Corporate Update at Two Investor Conferences

On February 25, 2019 Sorrento Therapeutics, Inc. (NASDAQ: SRNE, "Sorrento") reported that senior management will provide a corporate overview at the upcoming SVB/Leerink Global Healthcare Conference, and the Oppenheimer & Co. 29th Annual Healthcare Conference, both taking place in New York City (Press release, Sorrento Therapeutics, FEB 25, 2019, http://investors.sorrentotherapeutics.com/news-releases/news-release-details/sorrento-therapeutics-present-corporate-update-two-investor [SID1234533627]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

A copy of the presentation materials will be made available on the company website (www.sorrentotherapeutics.com).

SVB/LEERINK Global Healthcare Conference

Date: Thursday, February 28
Time: 3:30pm Eastern Time
Location: Lotte New York Palace Hotel
Webcast: View Source
Oppenheimer & Co. 29th Annual Healthcare Conference

Date: Wednesday, March 20
Time: 2:45pm Eastern Time
Location: Westin Grand Central Hotel, Ambassador Room
Webcast: View Source

Oncopeptides to present at the Cowen and Company Annual Healthcare Conference on Monday 11th March at 4:10 pm Eastern Standard Time

On February 25, 2019 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that Jakob Lindberg, CEO at Oncopeptides, will present the company at Cowen and Company 39th Annual Healthcare Conference in Boston on March 11th at 4:10 pm Eastern Standard Time, the presentation will be webcasted (Press release, Oncopeptides, FEB 25, 2019, View Source [SID1234533626]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

To access the webcast please use the link below:

http://wsw.com/webcast/cowen52/onco.st/

For further information, please contact:

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

This information was submitted for publication at 14:00 CET February 25, 2018

Agios Reports Updated Data from Phase 1 Study of Ivosidenib in Combination with Azacitidine Demonstrating Deep and Durable Responses in Newly Diagnosed IDH1 Mutant Acute Myeloid Leukemia (AML) Patients

On February 25, 2019 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported updated data from a Phase 1 study evaluating ivosidenib (TIBSOVO; AG-120) in combination with azacitidine in newly diagnosed isocitrate dehydrogenase-1 (IDH1) mutant acute myeloid leukemia (AML) patients (Press release, Agios Pharmaceuticals, FEB 25, 2019, View Source [SID1234533625]). The data were featured at the 17th International Symposium on Acute Leukemias taking place in Munich.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"With longer follow up from the ongoing Phase 1 study, the ivosidenib and azacitidine combination data in newly diagnosed AML patients are striking, with a 65% CR+CRh rate, 57% CR rate and the majority of CR patients achieving IDH1 mutation clearance," said Courtney DiNardo, M.D., lead investigator and assistant professor, department of leukemia at the University of Texas MD Anderson Cancer Center. "The combination regimen showed a 12-month survival rate of 82%, which is impressive given the age and comorbidities associated with patients who are not eligible for intensive chemotherapy. From a safety perspective, results from the combination were consistent with the safety profiles of each drug used alone and cytopenias were in line with those seen for azacitidine alone and favorable compared with other emerging hypomethylating agent combinations."

"As the Phase 1 data have matured, we saw an increase in patients achieving deep and durable remissions, validating our belief that the combination of azacitidine and ivosidenib has the potential to be a compelling treatment option and the cornerstone of therapy for frontline AML patients with an IDH1 mutation who are ineligible for intensive chemotherapy," said Chris Bowden, M.D., chief medical officer at Agios. "We will further evaluate the clinical benefit of ivosidenib in this treatment combination as part of the ongoing Phase 3 AGILE trial."

About the Ongoing Phase 1/2 Study
The ongoing Phase 1/2 study is evaluating an investigational use of ivosidenib or enasidenib in combination with azacitidine in patients with newly diagnosed IDH mutant AML unable to receive intensive chemotherapy. Data presented are from the ivosidenib arm of the Phase 1b portion of the study, in which 23 patients received 500 mg of ivosidenib daily plus azacitidine. Enrollment in the ivosidenib arm is complete.

As of the August 1, 2018 data cutoff, 14 (61%) patients remained on study.
The median number of treatment cycles was 8 (range 1-22).
The median age was 76 years old, and 52% of patients were age 75 or older.
74% of patients had de novo AML and 26% had secondary AML.
Ivosidenib Safety

The most common all-grade adverse events (AEs) regardless of cause occurring in ≥50% of patients were nausea (61%), diarrhea (57%), anemia (52%) and thrombocytopenia (52%).
The most common Grade 3/4 AEs were thrombocytopenia (48%), anemia (44%) and febrile neutropenia (44%).
Investigator reported IDH differentiation syndrome (DS) was reported in four patients, of which three were serious AEs. All four cases resolved, including two who achieved a complete response (CR), one stable disease and one was not evaluable for response.
Mean neutrophil and platelet counts were maintained near or above thresholds for CR with partial hematologic recovery (CRh) while on study treatment with ivosidenib and azacitidine. CRh is defined as <5% of blasts in the bone marrow, no evidence of disease and partial recovery of peripheral blood counts (ANC >500/microliter and platelets >50,000/microliter).
Ivosidenib Efficacy

Overall, 78% (18/23) of patients had a response.
65% (15/23) of patients had a CR+CRh
57% (13/23) of patients had a CR.
The median duration of CR (95% CI 7.7, NE) as well as CR+CRh (95% CI 7.7, NE) had not been reached.
The median time to response was 1.8 months (range 0.7-3.8 months) and the median time to CR was 3.5 months (range 0.8-6 months).
The 12-month survival rate was 82%.
The median duration of follow-up was 9.5 months (range 1.3-24 months).
For patients who achieved a CR, IDH1 mutation clearance was observed in 9 of 13 patients with available bone marrow mononuclear cells (BMMCs) and 10 of 13 patients with available peripheral blood mononuclear cells (PBMCs) as quantified by a sensitive digital PCR assay with lower limit of sensitivity for mutant IDH1 of 0.02-0.04% (or 10-4).
Ivosidenib is not approved in any country for the treatment of patients with newly diagnosed AML or approved in combination with azacitidine.

About TIBSOVO (ivosidenib)

TIBSOVO (ivosidenib) is an isocitrate dehydrogenase-1 (IDH1) inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test. For more information, visit TIBSOVO.com.

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.
WARNINGS AND PRECAUTIONS

Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 19% (34/179) of patients with relapsed or refractory AML treated with TIBSOVO experienced differentiation syndrome. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal if not treated. Symptoms of differentiation syndrome in patients treated with TIBSOVO included noninfectious leukocytosis, peripheral edema, pyrexia, dyspnea, pleural effusion, hypotension, hypoxia, pulmonary edema, pneumonitis, pericardial effusion, rash, fluid overload, tumor lysis syndrome, and creatinine increased. Of the 34 patients who experienced differentiation syndrome, 27 (79%) recovered after treatment or after dose interruption of TIBSOVO. Differentiation syndrome occurred as early as 1 day and up to 3 months after TIBSOVO initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, initiate dexamethasone 10 mg IV every 12 hours (or an equivalent dose of an alternative oral or IV corticosteroid) and hemodynamic monitoring until improvement. If concomitant noninfectious leukocytosis is observed, initiate treatment with hydroxyurea or leukapheresis, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms and administer corticosteroids for a minimum of 3 days. Symptoms of differentiation syndrome may recur with premature discontinuation of corticosteroid and/or hydroxyurea treatment. If severe signs and/or symptoms persist for more than 48 hours after initiation of corticosteroids, interrupt TIBSOVO until signs and symptoms are no longer severe.

QTc Interval Prolongation: Patients treated with TIBSOVO can develop QT (QTc) prolongation and ventricular arrhythmias. One patient developed ventricular fibrillation attributed to TIBSOVO. Concomitant use of TIBSOVO with drugs known to prolong the QTc interval (e.g., anti-arrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) and CYP3A4 inhibitors may increase the risk of QTc interval prolongation. Conduct monitoring of electrocardiograms (ECGs) and electrolytes. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or in those who are taking medications known to prolong the QTc interval, more frequent monitoring may be necessary.

Interrupt TIBSOVO if QTc increases to greater than 480 msec and less than 500 msec. Interrupt and reduce TIBSOVO if QTc increases to greater than 500 msec. Permanently discontinue TIBSOVO in patients who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia.

Guillain-Barré Syndrome: Guillain-Barré syndrome occurred in <1% (2/258) of patients treated with TIBSOVO in the clinical study. Monitor patients taking TIBSOVO for onset of new signs or symptoms of motor and/or sensory neuropathy such as unilateral or bilateral weakness, sensory alterations, paresthesias, or difficulty breathing. Permanently discontinue TIBSOVO in patients who are diagnosed with Guillain-Barré syndrome.

ADVERSE REACTIONS

The most common adverse reactions (≥20%) of any grade were fatigue (39%), leukocytosis (38%), arthralgia (36%), diarrhea (34%), dyspnea (33%), edema (32%), nausea (31%), mucositis (28%), electrocardiogram QT prolonged (26%), rash (26%), pyrexia (23%), cough (22%), and constipation (20%).
The most frequently reported ≥Grade 3 adverse reactions (≥5%) were electrocardiogram QT prolonged (10%), dyspnea (9%), leukocytosis (8%), tumor lysis syndrome (6%), and differentiation syndrome (5%).
Serious adverse reactions (≥5%) were differentiation syndrome (10%), leukocytosis (10%), and electrocardiogram QT prolonged (7%). There was one case of progressive multifocal leukoencephalopathy (PML).
DRUG INTERACTIONS

Strong or Moderate CYP3A4 Inhibitors: Reduce TIBSOVO dose with strong CYP3A4 inhibitors. Monitor patients for increased risk of QTc interval prolongation.

Strong CYP3A4 Inducers: Avoid concomitant use with TIBSOVO.

Sensitive CYP3A4 Substrates: Avoid concomitant use with TIBSOVO.

QTc Prolonging Drugs: Avoid concomitant use with TIBSOVO. If co-administration is unavoidable, monitor patients for increased risk of QTc interval prolongation.

LACTATION

Many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed children, advise women not to breastfeed during treatment with TIBSOVO and for at least 1 month after the last dose.

Please see full Prescribing Information, including Boxed WARNING.

About Acute Myelogenous Leukemia (AML)
AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. Undifferentiated blast cells proliferate in the bone marrow rather than mature into normal blood cells. AML incidence significantly increases with age, and the median age of diagnosis is 68. The vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is approximately 27 percent. IDH1 mutations are present in about 6 to 10 percent of AML cases.

Actinium’s Late Breaking Oral Presentation at 2019 TCT Meetings Reports New Data on Donor Chimerism Indicating Deep Engraftment in All Iomab-B Treated Patients in the Pivotal Phase 3 SIERRA Trial

On February 25, 2019 Actinium Pharmaceuticals, Inc. (NYSE American: ATNM), reported that new data from the ongoing pivotal Phase 3 SIERRA trial for Iomab-B was reported in a late breaking oral presentation at the 2019 Transplantation & Cellular Therapy Meetings of ASBMT and CIBMTR (TCT Meetings) that was held on February 20th – 24th (Press release, Actinium Pharmaceuticals, FEB 25, 2019, View Source [SID1234533624]). Dr. Sergio Giralt, Chief, Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center presented the late breaking oral presentation. It was reported that all patients who received Iomab-B, received a BMT or Bone Marrow Transplant with 100% (28/28) of patients achieving engraftment and Donor Chimerism. The new data indicated that 92% (26/28) of these patients achieved Full Donor Chimerism prior to day 100, which is defined as at least 95% of donor cells being engrafted in the recipient. Full Donor Chimerism prior to day 100 is a clinically significant outcome that indicates acceptance of donor cells and transplant success.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are excited that data from the SIERRA trial continue to demonstrate strong engraftment, particularly in this patient population who have limited access to BMT, which is the only curative treatment option, with current chemotherapy based conditioning approaches", said Dr. Mark Berger, Actinium’s Chief Medical Officer. "Full Donor Chimerism is an important metric in this setting that indicates patients receiving Iomab-B are having successful transplants, which is significant for the SIERRA trial. I am delighted that we were able to report these new data on strong donor chimerism to the transplant community at the TCT Meetings after reporting at ASH (Free ASH Whitepaper) in December that all patients receiving Iomab-B received a BMT and achieved engraftment. We are motivated by the positive reception that the engraftment, safety and feasibility data have received from trial investigators and referring physicians. These data from the SIERRA trial will serve us well as we work to complete enrollment of the SIERRA trial and bring this important therapy to patients with significant unmet needs."

The SIERRA trial (Study of Iomab-B in Elderly Relapsed or Refractory AML) is a 150-patient pivotal Phase 3 multi-center randomized trial that will compare outcomes of patients who receive Iomab-B and a BMT to those patients receiving physician’s choice of salvage chemotherapy, defined as conventional care, as no standard of care exists for this patient population. The primary endpoint of the SIERRA trial is dCR or durable Complete Remission of 6 months. The SIERRA trial is currently enrolling patients at 18 sites in the U.S and Canada including many of the leading BMT sites based on volume. Patients with active, relapsed or refractory AML have dismal prognoses and are typically not offered potentially curative transplant as an option, largely because salvage treatments have a limited ability to produce a complete remission, which is necessary prior to conventional BMT if conventional BMT is to be successful. However, with Iomab-B targeted conditioning, a complete remission prior to starting the Iomab-B conditioning is not necessary for a successful transplant. Iomab-B is an ARC or Antibody Radiation-Conjugate that targets CD45, an antigen expressed on leukemia, lymphoma and immune cells, and delivers Iodine-131 that kills targeted cells via linear energy transfer. Safety and feasibility data from the first 38 patients (25% of planned enrollment) in the SIERRA trial including donor chimerism data that were presented in a late breaking oral session at TCT can accessed here. Additional safety and feasibility analyses will occur when 50% and 75% of patients have been enrolled. The SIERRA trial also permits ad hoc interim analyses that may be requested at Actinium’s discretion to assess safety and efficacy when 70 and 110 patients have reached the primary endpoint of 6-month dCR. However, these interim analyses will not expend meaningful alpha and repowering of the study is not required as trial size cannot be increased after an Ad-hoc interim analysis.

Key highlights from the SIERRA Trial presented at ASH (Free ASH Whitepaper) and the TCT Meetings include:

All patients receiving a therapeutic dose of Iomab-B engrafted despite active disease with high blast count (median 30%, or median 45% for crossover patients)
15 of 19 (79%) patients in the control arm failed to achieve a complete response
67% (10/15) of patients eligible for crossover successfully transplanted after Iomab-B treatment
Patients receiving Iomab-B received a BMT more quickly post-randomization (28 days) than patients receiving conventional care (67 days)
In the conventional care arm, there was no difference in time to BMT for patients that crossed over to Iomab-B (66 days) compared to those achieving complete remission with conventional care (67 days)
No Grade 3 or 4 Iomab-B infusion related reactions with all Iomab-B infusions completed
No 100-Day non-relapse mortality in patients randomized to Iomab-B arm
All patients receiving Iomab-B and a BMT (28/28) achieved Donor Chimerism prior to day 100
94% of patients initially randomized to receive Iomab-B and a BMT (17/18) achieved Full Donor Chimerism > 95% prior to day 100 with 1 patient achieving 65% donor chimerism
90% of patients who crossed-over to receive Iomab-B and a BMT (9/10), after salvage chemotherapy in the control arm failed to produce a CR or Complete Response, also achieved Full Donor Chimerism > 95% prior to day 100 with 1 patient achieving 86% donor chimerism
Sandesh Seth, Actinium’s Chairman and CEO said, "Iomab-B has been studied extensively across multiple clinical trials and disease indications where it has consistently demonstrated the ability to condition patients for BMT in a well-tolerated manner with high engraftment rates and improved clinical outcomes including a survival benefit. As the lead candidate in our pipeline, it is heartening to see interim safety and feasibility data consistent with prior clinical evidence as the trend of strong engraftment with Iomab-B continues in the pivotal multi-center SIERRA trial. In addition, our other posters at TCT supporting the value proposition of Iomab-B from a healthcare economics perspective are also illuminative of the potential opportunity available. We are also excited to note the strong data from the Iomab-ACT program for targeted lymphodepletion prior to CART and other adoptive cell therapies supportive of clinical advancement that was presented at this TCT Meeting. We look forward to providing additional updates as we continue to build and advance our industry leading, multi-asset, multi-indication targeted conditioning portfolio."