Galena 2017 Outlook

Galena Biopharma to Provide Corporate and Clinical Update and 2017 Outlook During Presentation at Biotech Showcase 2017

“2017 looks to be promising for Galena Biopharma as we prepare to initiate our Phase 3 clinical trial in patients with essential thrombocythemia (ET) with GALE-401, our controlled release version of anagrelide,” said Mark W. Schwartz, Ph.D., President and Chief Executive Officer. “After a productive meeting with the U.S. Food and Drug Administration (FDA) last month, we were pleased to confirm that the GALE-401 development program is appropriate for a New Drug Application (NDA) filing using the 505(b)(2) regulatory pathway in patients who are intolerant to or failed to achieve an optimal response with hydroxyurea. In the U.S., ET has a prevalence of approximately 150,000 people and we estimate up to 25% of those patients who receive initial treatment with hydroxyurea may be candidates for the GALE-401 trial.  We expect to finalize the details of the Phase 3 clinical trial protocol including the trial size, endpoints, and dosing this quarter and initiate the trial in the second quarter of 2017.”

Dr. Schwartz continued, “We were also excited to announce last month that the Phase 2 clinical trial with NeuVax™ (nelipepimut-S) in Ductal Carcinoma in Situ (DCIS) resumed enrollment. In 2017, we expect enrollment to be completed in both of our NeuVax and trastuzumab combination trials. These investigator-sponsored trials (ISTs) are relatively low cost and allow us to leverage our resources to validate NeuVax as a potential treatment option whether as a stand-alone therapy or in combination with other agents. We believe there is substantial interest in the potential efficacy of NeuVax in a number of breast cancer indications and other malignancies such as gastric cancer, and ISTs such as these allow us to expand the utility of the agent.  Similarly, with our folate binding protein (FBP) programs, GALE-301 and GALE-302, our investigators provided a wealth of data in 2016 at various medical conferences including a November publication in Oncotarget on the GALE-301 interim analysis. These data on dosing and treatment schedules are extremely valuable as they highlight the potential utility of the vaccine in different cancer indications.”

Caregiving: What I’ve Learned

By Stephen W.

My wife was diagnosed with ET (Essential Thrombocythemia) in 2007. Since then, her health issues have varied. For several months, she will do very well with little to no problems, and then an episode that has included an ER visit on occasion, will throw us into action mode. Even our children know what is expected of them. We all becoming a caregiving team.

Caregiving has many layers. It requires endurance, listening skills, humility, open mindedness, flexibility, wisdom, empathy, managerial skills, problem solving skills, healthy choices, rest, outlets, support and in many ways, the needs similar to those we care for, except that we have to be willing to be the strength BEHIND the patient.

Endurance. There will be days when the one you are caring for requires everything you’ve got. It is easy to get burnt out. You may find yourself doing it all and feeling a bit resentful, at times. My suggestion is to get a backup caregiver. So many people offer help and we graciously decline with a thank you and assurance that we are okay. I now say, thank you and how can you help? Getting help doesn’t mean you are shrugging your responsibilities or care less about your loved one. It is a wise decision when times are tough. Sometimes just having someone grocery shop is a great gift.

Humility. Caregiving is not about you. I’ve learned to keep my place and learn from my wife and those that administer her medical care. I don’t have all the answers. My role is vital but there is no room for an ego.

Managerial skills. I never dreamed the managerial skills I learned in my career would come in handy for caregiving. Managing appointments, medical needs, insurance companies, children, household needs, and day to day care for the patient can be overwhelming without a system. When times are tough we run a tight ship with each of us knowing our role and responsibilities. Delegating is a way of life. We’ve learned to adjust quickly and as needed.

Keeping EVERYONE healthy. Parenting and caregiving for an adult are very similar. If we aren’t healthy, everyone suffers. Proper nutrition, exercise, rest, and finding outlets and support are essential. Recognize when you need a break and take it.

The Rewards. I’ve never felt closer to my wife. Our family is stronger and we know we can rely on one another. I’ve learned a lot about ET, but equally about life and love.

 

A Veteran’s Story: Gaining Knowledge and Advocating

I served in the Army during a more recent war than many of the veterans who have been diagnosed with an MPN.  I was diagnosed with Essential Thrombocythemia (ET) in early 2013. My son was only a year old and I had spent two weeks in a VA hospital while hematologists tried to rule out other illnesses. I was put on Hydroxyurea (Hydrea) almost immediately due to the aggressive rise of my platelet count. It was eventually controlled and I was sent off with a permanent Hydrea prescription.

My background in health care and my university education prompted me to research this illness and I learned a lot.  I read about alternative drugs, but the drug that stood out to me the most was peginterferon alfa-2a (Pegasys). It’s what was advised for young females of childbearing age, as it isn’t harmful to a fetus. I also learned that Pegasys is the only known drug that gives a glimmer of hope for remission. That’s when I decided I wanted to be on Pegasys. Hydrea was giving me terrible side effects.

I joined several MPN support groups on Facebook. This is probably the best thing I ever did for my condition. The wealth of information in those groups is astounding. I met many people who were very proactive in finding the correct treatments that alleviated some of their symptoms.

I was fortunate to have the wherewithal to find this out on my own.  I’m sure there are many veterans who do not or cannot advocate for themselves and are probably too tired, too sick or too confident in the VA to research this for themselves. Or maybe many of them had but had been denied, like I was.

At my visit with my VA oncologist, I mentioned Pegasys and my desire to be on it. I was told that Hydrea is the standard drug for ET and that all other patients are on it. I was adamant to get on Pegasys and was told that only Melanoma patients received it. Luckily, I had a receptive and understanding oncologist who said he’d research it first before giving it to me. After doing his own research, he agreed. I think I was the first ET-VA patient in Central Texas to be put on Pegasys. To this day, I believe I’m still the only one.

I was very lucky.  I’m sure this does not happen often with other VA physicians.  My other mission was to be seen by an MPN expert.  Making all these discoveries on my own really showed me how rare MPNs are and how unaware many VA hematologist/oncologists are about the best current available treatments.  This is my life…but not just mine, my son’s too.  I owe it to him to improve my health and extend my life.

My hematologist/oncologist agreed to submit a request to get seen at MD Anderson in Houston. I was placed into the Veteran’s Choice program and repeatedly given appointments with local civilian physician.  After letting the program know time and time again, that I wanted to go to MD Anderson, my oncologist told me it had been rejected because the VA doesn’t approve “second opinions.”

I was disappointed but grateful that, at least, I was on Pegasys. About a year later, I decided to prepare to have another baby. My VA gynecologist advised me to let my oncologist know of this. At my appointment, I told my oncologist my plans and he said he’d put in another request for the VA to cover a visit to see an MPN expert. I didn’t get my hopes up.

Sometime after, I got a call saying I was approved for two visits at MD Anderson. I saw an MPN expert who basically told me I was on the right medication (Pegasys) but that I was taking it too often because my white blood cell count was low and I was very fatigued (among other disturbing symptoms). He decreased the frequency and I felt much better with my white blood cell count being within normal range for the first time in three years. MD Anderson also tested me for mutations that the VA couldn’t perform. I had many questions answered and was finally at peace with how my condition was being handled. I was ready to move forward confidently, I felt closure.

None of this would have happened if I had been a man. The VA only approved my MD Anderson visit after learning about my intention to get pregnant. As a female, I’m a minority at the VA. There are many male vets who aren’t benefiting from a visit with an MPN expert.

If I hadn’t done my own research on ET, I would still be on Hydrea today. The side effects were very trying. Pegasys wasn’t even on their radar. Even if the VA approves prescriptions for Pegasys, Anagrelide, Jakafi or something else, the patients could be suffering from dosage/frequency problems, like I was. An MPN expert can help sort this out, and so much more.

I encourage MPN patients and their oncologists to do their own research. Veterans have the right to know about alternate treatments. Veterans also need to see knowledgeable experts and have access to blood tests that are relevant to their condition.

 

Stemline Therapeutics Announces Positive FDA Meeting and Agreement on Expedited Pathway to Full Approval of SL-401

NEW YORK, Jan. 05, 2017 (GLOBE NEWSWIRE) — Stemline Therapeutics, Inc. (Nasdaq:STML) announced today an agreement with the U.S. Food and Drug Administration (FDA) on the registration pathway for SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN). To support the filing of a Biologics License Application (BLA) for full approval in first-line BPDCN, Stemline is currently enrolling an additional small cohort, planned for 8-12 first-line BPDCN patients, into its ongoing Phase 2 trial. To date, approximately half of these new patients are enrolled into the study, with full enrollment expected this quarter. Stemline intends to file a BLA in 2H17, which is anticipated to undergo an expedited review given SL-401’s Breakthrough Therapy Designation. If successful, Stemline projects a commercial launch of SL-401 in 2018.

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Blog: Trouble Sleeping at Night?

By Marina Sampanes Peed

images-4Check Your Sleep Hygiene

We all know the importance of good hygiene techniques to avoid the flu and other illness: Wash your hands frequently. Clean keyboards, phones, remote controls, door handles…

My family jokes about my germ awareness and over-use of the phrase “proper hygiene.” So imagine my surprise when I learned that I have “poor sleep hygiene.” I should have known that the TV is not a sleep aid. Sleep hygiene refers to what you do before you try to fall asleep.

The Science of Sleep

Sleep health is matter of scientific study. According to the Stanford Center for Sleep Sciences and Medicine, there are over 100 different types of sleep disorders ranging from difficulty sleeping at night to problems with excessive daytime sleepiness. Most people lack understanding of how sleep impacts their health, and the dangers of poor sleep management.

Hazardous to your Health

Poor sleep health can contribute to: heightened inflammation, hypertension, heart disease, stroke, diabetes, obesity, erectile dysfunction, poor focus and attention, impaired memory and concentration, increased pain, workplace accidents, motor vehicle crashes, and early death. Small changes to your nightly routine can reduce your risks.

Control what you can – for free

The uncertainty of chronic illnesses like Myeloproliferative Neoplasia can be nerve-wracking. With so much uncertainty in life, let’s embrace the things we can control: sleep hygiene. Warning: it’s harder than it looks!

Sleep hygiene refers to your behaviors – your practices and habits leading up to bedtime. Do any of these common sleep habits sound familiar? Try the healthy habits and quit the unhealthy habits for one month – start one night at a time.

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I confess to be a work in progress. I use an apps on my phone to remind me to go to bed, guided sleep meditations, and one that tracks my sleep through my CPAP machine. I’m ordered the blue-blocking glasses and look forward to the strange looks from my family.

To learn more about healthy sleep, check out: www.sleeptember.org

Sleeptember® is a community of people with chronic health conditions, non-profit organizations, and other partners working together to raise awareness of how sleep impacts our health, safety, well-being, and productivity.

 

 

 

Siddhartha Mukherjee Offers Insight on the Future of MPN Treatment

Winning the 2011 Pulitzer Prize for General Non-Fiction and named one of the 100 most influential books written in English since 1923 by Time Magazine, The Emperor of All Maladies: A Biography of Cancer is certainly an impressive book. However, its author is equally as impressive: Siddhartha Mukherjee, M.D., Ph.D. is not only a Pulitzer Prize winner, but also an oncologist/hematologist and researcher He also served as the guest speaker at the award ceremony for the 2016 CURE Myeloproliferative Neoplasms (MPN) Heroes.

While there, CURE spent some time with Mukherjee to talk about MPN awareness, his newest book, The Gene: An Intimate History and the important role that “small data” in anti-cancer therapies.

There’s some difficulty with diagnosing MPNs, as well as a lack of information with physicians. Do you feel that awareness has gotten better?

MPNs have been a somewhat mysterious disorder for a very long time. And for a while, it was so poorly understood that physicians had a hard time understanding what the diagnosis was, how to diagnosis it appropriately, what the subtype was and how to appropriately characterize that subtype.

Now, as the awareness has grown, primarily driven by patients and patient advocates, I think there’s a growing awareness of what this family of disorders is, how it manifests and, perhaps most importantly, what the molecular lesions are, and what the genetics is for this family of diseases. I think all of this together – increased advocacy and better understanding of the molecular mechanism, and increasing physician awareness –  has driven a change in how people understand and diagnose these diseases.

What are some of the challenges patients face when they’re diagnosed with an MPN?

It’s a relatively rare illness and often there’s a lot of misinformation around this family of diseases. One of the challenges is to get the right information, to make sure that you connect with a physician who actually understands and knows how to treat this series of disorders, so you get the best and most optimal care.

There have recently been NCCN guidelines established for MPNs. Has that made a big difference in the community?

Absolutely. The presence of powerful, comprehensive guidelines really helps because rather than having ad-hoc ways of treating and understanding MPNs, there’s a more systematic way to understand, classify and treat MPNs. That’s helped a lot.

We need a lot more in these guidelines, but it’s an evolving field. The molecular characterization of MPNs is constantly evolving, and we’re still trying to understand what the various subtypes are and what the fundamental pathogenesis of these disorders is. That’s going to change.

But absolutely, yes, having guidelines helps a lot. It standardizes the procedures for treatment.

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CTI BioPharma Announces Removal Of Full Clinical Hold On Pacritinib

SEATTLE, Jan. 5, 2017 /PRNewswire/ — CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) today announced that the full clinical hold (February 2016) implemented by the U.S. Food and Drug Administration (FDA) on all clinical trials conducted under the Investigational New Drug (IND) application for pacritinib has now been removed. The Company’s complete response submission included, among other items, final Clinical Study Reports for both PERSIST-1 and 2 trials and a dose-exploration clinical trial protocol that the FDA requested.  The new trial, PAC203 plans to enroll up to approximately 105 patients with primary myelofibrosis who have failed prior ruxolitinib therapy to evaluate the safety and the dose response relationship for efficacy (spleen volume reduction at 24 weeks) of three dose regimens: 100 mg once-daily, 100 mg twice-daily (BID) and 200 mg BID. The 200 mg BID dose regimen was used in PERSIST-2. The Company expects to start the trial in the second quarter of 2017.

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Alox5 Blockade Eradicates JAK2V617F-Induced PV in Mice

Yaoyu Chen, Yi Shan, Min Lu, Ngoc DeSouza, Zhiru Guo, Ronald Hoffman, Aibin Liang and Shaoguang Li

Myeloproliferative neoplasms such as polycythemia vera (PV), which are associated with the JAK mutation V617F, remain incurable despite progress in the use of JAK2 inhibitors for treatment of some of these diseases. In this study, we employed mice that undergo JAK2V617F-induced PV as a tool to explore new candidate targets for therapy. Our investigations focused on the lipid metabolic enzyme arachidonate 5-lipoxygenase (Alox5), which we found to be strongly upregulated by JAK2V617F in hematopoietic cells in vitro and in vivo. Notably, genetic deletion of Alox5 or its inhibition in mice with a bioactive small-molecule inhibitor was sufficient to attenuate PV development. This therapeutic effect was associated with induction of a blockade in cell-cycle progression and also with apoptosis in PV cells. Genetic loss exerted an inhibitory effect on PV-initiating cells. Similarly, Alox5 inhibition was sufficient to suppress colony formation in human JAK2V617F-expressing CD34+ cells. Mechanistic investigations showed that Alox5 inhibition reduced AKT activation and decreased β-catenin expression in JAK2V617F-expressing cells. Together, our results define Alox5 as a key genetic effector of JAK2V617F in driving PV, and they identify this enzyme as a candidate therapeutic target to treat this refractory myeloproliferative neoplasm. Cancer Res; 77(1); 164–74. ©2016 AACR.

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