Secura Bio Acquires Global Rights To Farydak®

On March 12, 2019 Secura Bio, Inc. (www.securabio.com), an integrated, commercial-stage biopharmaceutical company dedicated to the worldwide commercialization of significant oncology therapies, reported that it has acquired the global rights to Farydak (panobinostat) from Novartis (Press release, Secura Bio, MAR 12, 2019, View Source [SID1234553835]).

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Farydak (panobinostat) is a prescription oral medication used in combination with proteasome inhibitors and corticosteroids to treat patients with relapsed or refractory multiple myeloma who had received prior treatments. Farydak is a member of the histone deacetylase (HDAC) inhibitor family with a very potent and unique mode of action slowing the growth of multiple myeloma cells.

Multiple myeloma is a cancer that forms malignant plasma cells in the bone marrow, and is the second most common blood cancer with over 160,000 cases diagnosed globally per year, with over 30,000 in the United States. Despite the increased availability of treatments, the disease is characterized by recurrent relapses and remains incurable for most patients leading to approximately 106,000 deaths per year worldwide, over 12,000 of which are in the United States.

On February 23, 2015 Farydak received FDA accelerated approval for use in patients with multiple myeloma, and on August 28, 2015 it was approved by the European Medicines Agency for the same use. Farydak has received marketing authorization in 53 countries to date for the treatment of patients with multiple myeloma.

Secura Bio is dedicated to working with health care professionals to ensure Farydak’s benefit to patients is well understood by cancer care teams and by ensuring that the medicine is available to those who need it. Secura Bio is also investigating the opportunity to develop new potential dosages and product combinations for Farydak.

"We are very pleased to acquire worldwide rights to Farydak as we execute our plan to aggressively build a meaningful, worldwide oncology portfolio," said Joseph M. Limber, President and CEO of Secura Bio. "Farydak will be a critical cornerstone of our strategy, as it offers a potentially valuable and high-growth treatment option for physicians in their therapeutic regimen for multiple myeloma patients when combined with a variety of other multiple myeloma therapies."

"Farydak represents an exciting agent with a unique mechanism of action that is part of a promising class of drugs in this setting," said study investigator Paul Richardson, MD, Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center at Dana–Farber Cancer Institute. "Importantly, Farydak has been shown to improve progression–free survival in relapsed multiple myeloma patients who have received at least two prior regimens, including bortezomib and an IMiD, which is an area of particular unmet medical need."

About Farydak (panobinostat)

Farydak (panobinostat) is a histone deacetylase (HDAC) inhibitor that inhibits the enzymatic activity of HDACs at nanomolar concentrations. HDACs catalyze the removal of acetyl groups from the lysine residues of histones and some non-histone proteins. Inhibition of HDAC activity results in increased acetylation of histone proteins, an epigenetic alteration that results in a relaxing of chromatin, leading to transcriptional activation. In vitro, panobinostat caused the accumulation of acetylated histones and other proteins, inducing cell cycle arrest and/or apoptosis of some transformed cells. Increased levels of acetylated histones were observed in xenografts from mice that were treated with panobinostat. Panobinostat shows more cytotoxicity towards tumor cells compared to normal cells.

About multiple myeloma

Epigenetics is the cell programming that governs gene expression and cell development. In multiple myeloma, the normal epigenetic process is disrupted (also called epigenetic dysregulation) resulting in the growth of cancerous plasma cells, potential resistance to current treatment, and ultimately disease progression.

Multiple myeloma impacts approximately 81,000 people in the United States. Multiple myeloma is a cancer of the plasma cells, a kind of white blood cell present in bone marrow—the soft, blood–producing tissue that fills the center of most bones. The cancer is caused by the production and growth of abnormal cells within the plasma, which multiply and build up in the bone marrow, pushing out healthy cells and preventing them from functioning normally. Multiple myeloma is an incurable disease with a high rate of relapse (when the cancer returns) and resistance (when the therapy stops working), despite currently available treatments. It typically occurs in individuals 60 years of age or older, with few cases in individuals younger than 40.

Farydak Important Safety Information

Farydak can cause serious side effects, including diarrhea and heart problems.

Diarrhea is common with Farydak and can be severe. Patients should tell their healthcare provider (HCP) right away if they have abdominal (stomach) cramps, loose stool, diarrhea, or feel like they are becoming dehydrated. HCPs may prescribe medicines to help prevent or treat these side effects. Taking or using stool softeners or laxative medicines may worsen diarrhea, patients should talk to their HCP before taking or using these medicines.

Farydak can cause severe heart problems which can lead to death. Risk of heart problems may be increased with a condition called "long QT syndrome" or other heart problems. Patients should call their HCP and get emergency medical help right away if they have any of the following symptoms of heart problems: chest pain, faster or slower heartbeat, palpitations (feel like heart is racing), feel lightheaded or faint, dizziness, blue colored lips, shortness of breath, or swelling in legs.

Farydak can cause severe bleeding which can lead to death. It may take patients longer than usual to stop bleeding while taking Farydak. Patients should tell their HCP right away if they get any of the following signs of bleeding: blood in stools or black stools (look like tar), pink or brown urine, unexpected bleeding or bleeding that is severe or that cannot be controlled, vomit blood or vomit looks like coffee grounds, cough up blood or blood clots, increased bruising, feeling dizzy or weak, confusion, change in speech, or headache that lasts a long time.

Farydak is a prescription medicine used, in combination with bortezomib and dexamethasone, to treat people with a type of cancer called multiple myeloma after at least two other types of treatment have been tried. It is not known if Farydak is safe and effective in children.

Patients should tell their HCP about all of the medicines they take, including prescription and over–the–counter medicines, vitamins and herbal supplements.

Patients should take Farydak exactly as the HCP tells them to take it. The HCP will tell patients how much Farydak to take and when to take it. The HCP may change the dose or stop treatment temporarily if patients experience side effects. Patients should not change the dose or stop taking Farydak without first talking with their HCP.

Patients should avoid eating star fruit, pomegranate or pomegranate juice, and grapefruit or grapefruit juice while taking Farydak. These foods may affect the amount of Farydak in the blood.

Low blood cell counts are common with Farydak and can be severe. Low platelet count (thrombocytopenia) can cause unusual bleeding or bruising under the skin. Low white blood cell count (neutropenia) can cause infections. Low red blood cell count (anemia) may make a patient feel weak, tired, or they may get tired easily, look pale, or feel short of breath.

There is an increased risk of infection while taking Farydak. Patients should contact their HCP right away if they have a fever or have any signs of an infection including sweats or chills, cough, flu–like symptoms, shortness of breath, blood in phlegm, sores on body, warm or painful areas on body, or feeling very tired.

Patients should call their HCP right away with any of the following symptoms of liver problems: feel tired or weak, loss of appetite, dark amber colored urine, upper abdominal pain, yellowing of skin or the white of eyes.

The most common side effects of Farydak include tiredness, nausea, swelling in arms or legs, decreased appetite, fever and vomiting. Patients should tell their HCP if they have any side effect that is bothersome or that does not go away.

Guardant Health Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Full Year 2019 Outlook

On March 12, 2019 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary blood tests, vast data sets and advanced analytics, reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Guardant Health, MAR 12, 2019, View Source [SID1234534251]).

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Recent Highlights

Revenue of $32.9 million for the fourth quarter and $90.6 million for the full year of 2018, representing 64% and 82% increases, respectively, over the corresponding periods of 2017

NILE study, a head-to-head comparison of the Guardant360 assay to standard-of-care tissue testing met its primary endpoint, detecting a similar number of targetable biomarkers in NSCLC patients, which supports utilization of Guardant360 testing ahead of tissue testing

Launched the LUNAR assay to academic and biopharmaceutical researchers to support investigation of clinical applications, including for residual disease/recurrence monitoring and early detection

Announced an agreement with AstraZeneca to support the development of the Guardant360 and GuardantOMNI assays for use as companion diagnostics for Tagrisso and Imfinzi, respectively
"During 2018, we made important headway on key initiatives and are continuing to see strong adoption of our liquid biopsy platform, which has driven increased revenue of more than 80 percent over the prior year," said Helmy Eltoukhy, PhD, Chief Executive Officer. "We believe the recent NILE readout is an important catalyst supporting a blood-first paradigm for first-line use of Guardant360 testing for treatment selection ahead of tissue testing, offering lung cancer patients improved care with a faster time to treatment."

"In addition, we are encouraged by the progress we are making with our LUNAR program and look forward to presenting pilot LUNAR data at the AACR (Free AACR Whitepaper) conference next month. As a result of these developments, we are ramping up investment to accelerate our research and development efforts toward earlier cancer detection," continued Dr. Eltoukhy.

Investor presentation provided by bluebird bio, Inc. on March 12, 2019.

On March 12, 2019 bluebird bio, Inc. presented an Investor presentation (Press release, bluebird bio, MAR 12, 2019, View Source [SID1234534273]).

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Bellicum Pharmaceuticals Provides Operational Update and Reports Financial Results for the Fourth Quarter and Year Ended December 31, 2018

On March, 12 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Bellicum Pharmaceuticals, MAR 12, 2019, View Source [SID1234534233]).

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"We believe that Bellicum is well-positioned for an exciting and eventful 2019 as a result of our investments to strengthen our clinical development and executive teams over the past 24 months," said Bellicum’s President & CEO Rick Fair. "Based on promising preclinical and initial clinical data on BPX-601, we have prioritized GoCAR-T candidates incorporating our iMC activation switch in our clinical development strategy. In addition to progressing our Phase 1/2 trial of BPX-601, we intend to submit IND applications for two novel, dual-switch GoCAR-T clinical programs in 2019. For rivo-cel, we anticipate topline Phase 2 results in pediatric patients in the second quarter of 2019, which will form the basis for European Marketing Authorisation Application (MAA) submissions planned for late 2019."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

Bellicum Advances and Expands Clinical Program for GoCAR-T Platform

BPX-601

Bellicum completed enrollment in the dose-escalation portion of its ongoing Phase 1/2 study of BPX-601 in patients with advanced pancreatic cancer expressing prostate stem cell antigen (PSCA). Bellicum presented positive initial clinical data from Part 1 of the study at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress in December 2018 and the Gastrointestinal Cancers Symposium in January 2019 that showed a promising safety profile. In several patients reported, enhanced cell expansion, prolonged cell persistence, and early evidence of clinical activity and disease control were observed. The trial protocol has been amended to add the complete lymphodepletion conditioning regimen cyclophosphamide/fludarabine (Cy/Flu) and to include patients with gastric and prostate cancers. Once this safety cohort has been completed, Bellicum plans to incorporate an additional cohort to evaluate repeat rimiducid dosing to re-activate iMC over time, which is intended to deepen and extend the treatment effect. Initial results from these cohorts are expected in 2019.
Controllable Dual-Switch Candidates

BPX-603 is Bellicum’s first controllable dual-switch GoCAR-T product candidate, which incorporates both the iMC activation switch and the CaspaCIDe safety switch. BPX-603 is designed to target solid tumors that express the human epidermal growth factor receptor 2 antigen (HER2). HER2 is a validated antigen for cancer therapies, and academic CAR-T cell clinical studies have shown evidence of antitumor activity. These CAR-T approaches targeting HER2 have been limited by modest clinical efficacy and off-tumor/on-target toxicity. Bellicum believes that its dual-switch GoCAR-T technology may be uniquely suited to improve upon these earlier efforts by driving greater efficacy through iMC activation while enabling clinicians to manage any treatment-emergent toxicities with CaspaCIDe. Bellicum expects to submit an IND application for BPX-603 and to initiate a clinical trial in 2019. BPX-802 is a dual-switch GoCAR-T product candidate targeting an antigen expressed in hematological malignancies. Bellicum expects to submit an IND application for BPX-802 in late 2019.
Company Prepares for Rivo-cel Registration in E.U. for Pediatric Patients and Initiates Global Trial for Adult and Adolescent Patients

Bellicum presented late interim analyses of the pediatric study BP-004 at ASH (Free ASH Whitepaper) in December 2018. The results demonstrated that 90.9% of rivo-cel treated patients experienced Event Free Survival (EFS) at 180 days compared to 87.7% of patients undergoing matched unrelated donor (MUD) HSCT. Non-inferiority on EFS versus MUD HSCT is the primary endpoint for European regulatory review. The results also demonstrated that rivo-cel treated patients had high rates of relapse-free (82.9%) and overall (94.4%) survival with a median follow up of 20 months. Lastly, the results demonstrated a best overall response rate of 70% in patients that developed advanced or steroid-refractory GvHD when treated with rimiducid, with the majority being complete responses. The Company expects topline results from the BP-004 study in the second quarter of 2019 and intends to submit MAAs for rivo-cel and rimiducid in late 2019. The Company recently initiated THRIVE, a pivotal randomized global Phase 2/3 clinical trial of rivo-cel in adult and adolescent patients 12 years and older with intermediate- and high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). In the Phase 3 portion of the trial, patients will be randomized to receive either a/b T cell and B-cell depleted haploidentical HSCT plus rivo-cel without GvHD prophylaxis, or a haploidentical HSCT followed by cyclophosphamide post-transplant, commonly referred to as the "Baltimore regimen," which is the current standard of care for haplo-HSCT in the U.S. and Europe.
CORPORATE UPDATES

Company Makes Key Additions to Management Team and Board of Directors; Forms Scientific and Clinical Advisory Board

Atabak Mokari was appointed Chief Financial Officer. He brings 20 years of financial leadership in life sciences, medtech and healthcare investment banking, and joined the Company from IRIDEX Corporation, a NASDAQ-listed medical technology company where he served as Chief Financial Officer and VP Corporate Development. Aaron Foster, Ph.D., was promoted to Senior Vice President, Head of Research, and will oversee the Company’s research organization. Dr. Foster was previously Vice President, Translational Research & New Product Development. Prior to joining Bellicum in 2012, he was Assistant Professor at Baylor College of Medicine at the Center for Cell & Gene Therapy, where he led a group researching adoptive T cell therapies, cancer vaccines, and nanotherapeutics. Concurrently, David Spencer, Ph.D., stepped down from his operating role as Chief Technology Officer, and has assumed an advisory position on the Company’s Scientific Advisory Board. Judith Klimovsky, M.D., joined the Board of Directors, replacing Frank McGuyer. Dr. Klimovsky currently serves as Executive Vice President & Chief Development Officer of Genmab, an international biotechnology company developing antibody therapeutics for cancer, where she leads the company’s product development efforts. Company formed a new Scientific and Clinical Advisory Board comprised of the following individuals:
— Malcom Brenner, M.D., Ph.D. (Baylor College of Medicine)
— Marco Davila, M.D., Ph.D. (Moffitt Cancer Center)
— Gianpietro Dotti, M.D. (University of North Carolina Lineberger Comprehensive Cancer Center and Professor for the Department of Microbiology and Immunology at UNC-Chapel Hill)
— Daniel Powell Jr., Ph.D. (University of Pennsylvania, Perelman School of Medicine)
— Naiyer Rizvi, M.D. (Columbia University Irving Medical Center)
— Charles Sentman, Ph.D. (Dartmouth, Geisel School of Medicine)
— David Spencer, Ph.D. (Co-Founder, Bellicum Pharmaceuticals)
Fourth Quarter and Full Year 2018 Financial Results

Cash Position and Guidance: Bellicum reported cash, restricted cash and investments totaling $98.0 million as of December 31, 2018, compared to $106.5 million at December 31, 2017. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through the end of 2019.

R&D Expenses: Research and development expenses were $19.8 million and $71.2 million for the fourth quarter and year ended December 31, 2018, respectively, compared to $14.3 million and $65.7 million during the comparable periods in 2017. The higher expenses in the fourth quarter and full year 2018 compared to respective periods in 2017 were primarily due to an increase in costs related to Bellicum’s GoCAR-T product platform and general research and development expenses, partially offset by a decrease in expenditures related to rivo-cel.

G&A Expenses: General and administrative expenses were $7.0 million and $25.0 million for the fourth quarter and year ended December 31, 2018, respectively, compared to $5.1 million and $21.0 million during the comparable periods in 2017. The higher expenses in the fourth quarter and full year 2018 compared to respective periods in 2017 were primarily due to increased personnel related costs due to hiring additional employees.

Net Loss: Bellicum reported a net loss of $27.2 million for the fourth quarter of 2018 and $98.0 million for the year ended December 31, 2018, compared to a net loss of $21.9 million and $91.8 million for the comparable periods in 2017. The results included non-cash, share-based compensation charges of $3.0 million and $13.8 million for the fourth quarter and year ended December 31, 2018, respectively, and $3.4 million and $13.6 million for the comparable periods in 2017.

Shares Outstanding:

At December 31, 2018, Bellicum had 43,564,596 shares of common stock outstanding.

Upcoming Near-term Potential Milestones

BPX-601 – Expect to report data from additional cohorts in ongoing Phase 1/2 clinical trial in 2019
— Cy/Flu regimen cohort: mid-year 2019
— Repeat rimiducid dosing cohort: late 2019 BPX-603 – Expect IND submission and clinical trial initiation in 2019 BPX-802 – Expect IND submission in late 2019 Rivo-cel:
— Expect topline results from the BP-004 study in the second quarter of 2019
— Plan to submit MAAs for rivo-cel and rimiducid in late 2019
Conference Call and Webcast

Bellicum management will host a webcast and conference call at 5:00 p.m. Eastern today to discuss the financial results. To access the call, participants should dial 877-407-3103 (U.S. domestic) and 201-493-6791 (international) at least 10 minutes prior to the start of the call. The event will be webcast live and can also be accessed in the Investors & Media section of bellicum.com. An archived version of the webcast will also be available for replay in the Investors & Media section of the Bellicum website following the call.

About BPX-601

BPX-601, the Company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence, and enable the CAR-T to override key immune inhibitory mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, gastric, and prostate cancers.

About Rivo-cel (BPX-501)

Rivo-cel (rivogenlecleucel) is an allogeneic polyclonal T-cell product designed to accelerate immune recovery after HSCT and to reduce relapse of leukemia following a stem cell transplant. The cell treatment contains a diverse repertoire of T cells which may contribute to a robust graft vs. leukemia effect. Rivo-cel’s anti-infective benefits may also reduce morbidity and mortality, as patients are highly susceptible to infection following a transplant. The product’s CaspaCIDe safety switch enables this approach by allowing physicians to reduce the number of alloreactive cells in the event of uncontrolled GvHD. Rivo-cel addresses a major unmet need in adult and pediatric leukemia, lymphoma and inherited blood disease patients following a haploidentical stem cell transplant.

Company Presentation, March 2019

On March 12, 2019 Y-mAbs Therapeutics presented Corporate presentation (Presentation, Y-mAbs Therapeutics, MAR 12, 2019, View Source [SID1234534247]).

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