Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Immunotherapy

About Pieter Droppert

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Posts by Pieter Droppert

Midostaurin in AML – Better Late than Never!

Acute Myeloid Leukemia (AML) is challenging disease to treat and quite distinctly different from its cousin, acute lymphoblastic leukemia (ALL).  The first is more common in adults, while the second is more prevalent in children.  Success rates with pediatric ALL have far outstripped what we have achieved with adults in AML to date, partly due to the elderly nature of the disease making for poorer outcomes with stem cell transplants (SCT), as well as increased clonal heterogeneity and cytogenetic complexity with age.

Quite a few FLT3 inhibitors have come and gone over the years – many keen observers will remember Cephalon’s (now Teva) TKI called CEP-701, which was tested in relapsed/refractory disease and Elderly AML, for example, and slid off largely unnoticed to dog drug heaven.

How much does clinical trial design impact a drug’s success or failure?

Sometimes quite a bit, as this story with midostaurin demonstrates; limited activity in advanced disease but much more dramatic results in the upfront setting.  Clearly, sometimes testing drugs in later disease does not predict their future performance elsewhere!

To put more colour on the data presented at ASH, we interviewed a thought leader in adult AML for his perspective on the FLT3 R&D developments.

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New Horizons in CLL Treatment

ASH Exhibit HallIn recent years, there’s been a lot of progress in the treatment of chronic lymphocytic leukemia (CLL). New targeted therapies such as ibrutinib (Imbruvica) and idelalisib (Zydelig) have been approved and have helped extend the lives of patients with this disease further. However, there still remains a need for new treatment options.

Several new drugs are on the horizon for CLL.  At ASH there were a number of presentations for venetoclax, formerly known as ABT-199/GDC-0199, it’s a BCL-2 inhibitor, which is being co-developed by AbbVie and Genentech.  We’ve written extensively about it on the blog.  One of the challenges with venetoclax is the potential for Tumor Lysis Syndrome (TLS) – we heard at ASH that starting a patient on the drug needs to be carefully managed and monitored, with high risk patients hospitalized.

Other new drugs on the longer term horizon for CLL include acalabrutinib (Acerta) and BGB-3111 (BeiGene), both next generation BTK inhibitors and potential competitive threats to ibrutinib. The CLL market is becoming interesting again!

At ASH 2015, I spoke with Ian W. Flinn, MD, PhD. Director, Blood Cancer Research Program at the Sarah Cannon Research Institute in Nashville, TN. At ASH, Dr Flinn presented data for a CLL trial of venetoclax combined with obinutuzumab, a CD20 targeted monoclonal antibody; data was obtained in both the upfront and relapsed/refractory setting.

In a wide ranging conversation, we talked about some of the data of note in Orlando, what the future direction is in CLL, and what to look forward to at ASH 2016.

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New Treatments for CLL with Deletions in 17p

ASH15 LBA Session

ASH 2015 LBA Session

The annual meeting of the American Society of Hematology (ASH) has a few quirks compared to other meetings. One of these is that all the “Late Breakers” are presented together on the last morning of the meeting.

It’s a rather unfortunate time given many have already headed back to their busy clinics or left for SABCS in San Antonio and ‘late breakers’ by definition, often offer new data that’s really noteworthy.

The result can also be a bit of a hodgepodge session that you have sit to listen through to get to those presentations you really want to hear.

At ASH this year there were two late breakers on new treatment options for CLL patients with a 17p deletion (Del17p). This is a pretty challenging group to treat.  Although ibrutinib is indicated for this patient group, many sadly relapse. There’s an unmet medical need for new treatment options. At ASH we heard data for idelalisib (PI3K-delta) and venetoclax (Bcl2).

After the session, I briefly spoke with Dr Kanti Rai (New York) for his reaction to the data. Dr Rai (pictured below) received the 2014 Wallace H. Coulter Award for Lifetime Achievement in Hematology.

Dr Kanti Rai receives 2014 ASH Lifetime Achievement Award

Dr Kanti Rai receives 2014 ASH Lifetime Achievement Award

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Understanding the Cellectis UCART19 Case Report – Part 2 of an Interview with Prof Waseem Qasim

Prof Qasim ASH 2015 posterPart 2 of the ASH 2015 interview Professor Waseem Qasim kindly gave BSB at the American Society of Hematology (ASH) discusses the poster he presented at the meeting (Abstract 2046:) “First Clinical Application of Talen Engineered Universal CAR19 T Cells in B-ALL.”

Prof Qasim is a Consultant in Paediatric Immunology and Bone Marrow Transplantation at Great Ormond Street Hospital (GOSH) and Professor of Cell & Gene Therapy at the Institute of Child Health which is part of University College London (UCL).

The “devil is in the detail” so it was a privilege to be taken through the case by Prof Qasim, and in the process, better understand the treatment rational, as well some of the challenges and unanswered questions that will need to be addressed moving forwards.

The first-in-man use of the UCART19 allogeneic CAR T cell therapy from Cellectis was done under the Compassionate Use guidelines of the UK Medicines and Health Products Regulatory Agency (MHRA). A phase 1 clinical trial is planned for early 2016.

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Potential of Gene Editing – Part 1 of Interview with Prof Waseem Qasim

Prof Qasim UCART19 ASH 2015 PosterMy highlight of the 2015 annual meeting of the American Society of Hematology (ASH) was interviewing Professor Waseem Qasim, who is a Consultant in Paediatric Immunology and Bone Marrow Transplantation at Great Ormond Street Hospital (GOSH) and Professor of Cell & Gene Therapy at the Institute of Child Health, which is part of University College London (UCL).

In a poster presented at ASH 2015, Prof Qasim together with colleagues from GOSH & UCL reported the “first in man” use of a gene edited, off-the-shelf, allogeneic CAR T cell from Cellectis, a company we have written extensively about on the blog. It was probably one of the leading posters at the meeting, at least in terms of the amount of interest it generated, and the crowds I saw reading it.

There was far too much content in the interview for one blog post, so we’ve split into two, with Part 1 focusing on gene editing and Part 2 discussing in detail the case reported in the poster.

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Manic Monday at ASH 2015

ASH 2015 Manic Monday

The #ASH15 wall of people marching to the poster hall just after 5pm

Orlando – it’s Monday at the annual meeting of the American Society of Hematology (ASH) annual meeting, a day I call “Manic Monday” because there are so many simultaneous sessions, you end up running around frazzled, in/out of sessions, in the hope of catching all the presentations of interest.

It’s particularly challenging if you are in a full session — you won’t be able to get back in if you leave — which results in having to make difficult choices on what to see and where to run to.  Some of the myeloma thought leaders were urging colleagues to tweet sessions they couldn’t be in, so “Manic Monday” may be a good time to contribute to the collective ASH Twittersphere.

We’re starting today’s rolling post with my notes from the lymphoma New Drugs session yesterday, then we’ll be updating the blog as the day goes by, as the opportunity permits.

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Highlights from Plenary Sunday at #ASH15

ASH 2015 Hematology Orlando

ASH 2015 — taken before 7am!

Orlando – a presentation in the plenary session at #ASH15, the 2015 annual meeting of the American Society of Hematology, is the pinnacle of any doctor or researcher working in the hematology field.

Yesterday, we had the privilege to interview Dr Richard Stone (Dana-Farber Cancer Institute) ahead of his plenary presentation at ASH:

Abstract 6: The Multi-Kinase Inhibitor Midostaurin (M) Prolongs Survival Compared with Placebo (P) in Combination with Daunorubicin (D)/Cytarabine (C) Induction (ind), High-Dose C Consolidation (consol), and As Maintenance (maint) Therapy in Newly Diagnosed Acute Myeloid Leukemia (AML) Patients (pts) Age 18-60 with FLT3 Mutations (muts): An International Prospective Randomized (rand) P-Controlled Double-Blind Trial (CALGB 10603/RATIFY [Alliance])

Anyone who has been to an ASH education session on AML knows how hard a nut it is to crack, so it’s wonderful to see some positive data, in what is commonly considered to be a “graveyard” disease.

The trial has taken a long time to come to fruition, so all credit to Dr Stone and colleagues. We’ll be writing up more about the data in our post meeting coverage.  For additional background, you can also check out our FLT3 preview in AML, which details some of the history and context for this study. The data from the phase 3 study is likely to form the backbone of a registration filing for Novartis with this compound in the near future based on successfully meeting the trial endpoints.

We also kick off today’s highlights with quick reflections on some of the hot topics that emerged yesterday including Bluebird Bio’s lentiglobin, Bellicum’s pipeline and .

During the day, as the opportunity presents, we’ll also be providing commentary on sessions we attend.

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Highlights from Saturday at ASH 2015 Annual Meeting #ASH15

ASH 2015 OrlandoOrlando – the 2015 annual meeting of the American Society of Hematology (Twitter #ASH15) kicks off in earnest today at the Orange County Convention Center.

There’s a comprehensive press program at #ASH15, which will no doubt drive a lot of the media coverage, but in addition to discussing some of the highlights, we’ll be going off the beaten path, and gathering noteworthy data for our post-meeting coverage.

There’s always a lot of great posters at the meeting, as well as useful educational and scientific sessions.

If you are interested in checkpoints in hematology, do check out the Scientific Session chaired by Dr Krishna Komanduri (@drkomanduri) “Checkpoint, Please?” It takes place from 9.30 am to 11 am today. It’s repeated again from 4 to 5pm on Sunday.

As the conference program notes, “this session will provide a broad overview of checkpoint inhibition and its therapeutic potential in the setting of solid tumors, alloreactivity and treatment of hematologic neoplasms.”

This is a rolling blog post, throughout the day we’ll be adding commentary as the opportunity presents.

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Exploring the Potential of Therapeutic Cancer Vaccines

Dr Chris Heery (@ChrisHeery) is a medical oncologist at the National Cancer Institute (NCI) who works in the Laboratory of Tumor Immunology and Biology (LTIB) with Jeffrey Schlom, James Gulley and other translational scientists.

The aim of the LTIB is to develop novel immunotherapies for cancer. One of the ways this is accomplished is through a Cooperative Research and Development Agreement (CRADA), in essence a joint venture with the private sector.

At the recent Society for Immunotherapy of Cancer (SITC) annual meeting, Dr Heery presented a poster on the results of a phase 1 clinical trial to evaluate the safety and tolerability of a therapeutic vaccine, MVA-BN Brachyury, targeting brachyury. See Bavarian Nordic Press Release Nov 3, 2015.

We previously heard at ASCO 2015 about the rational for targeting brachyury from Dr James Gulley (see post: Future of Prostate Cancer Immunotherapy).

It was a pleasure to talk with Dr Heery about his poster and what the potential of therapeutic cancer vaccines may be in the cancer immunotherapy arsenal.

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Novel Immunotherapies and Combinations

“Novel Immunotherapies and Combinations” was the title of the talk by Dr Mario Sznol (Yale) at the recent Immunotherapy Patient Forum co-hosted by Global Resource for Advancing Cancer Organization (GRACE) and the Melanoma Research Alliance at the 2015 SITC annual meeting.

At the forum, Dr Sznol also led a breakout session, where he reviewed what is melanoma, the treatment of primary melanoma and management of advanced disease, as well as answering questions from the patients and patient advocates.

Often at medical meetings you hear the results of a clinical trial that is but one piece of the jigsaw, so it was interesting to hear a more comprehensive overview of the disease.

Dr Sznol kindly spoke with BSB about his vision for the future of cancer immunotherapies. This post includes excerpts from the interview along with additional commentary.

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