Biotech Strategy Blog

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

About Pieter Droppert

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Posts by Pieter Droppert

It’s been quite a roller coaster week so far for CAR T cell therapies, with Gilead announcing its intended acquisition of Kite Pharma on Monday.  If that wasn’t enough, Wednesday brought another surprise – the FDA approved a rare double header – for Novartis’s CTL019, now known as tisagenlecleucel (Kymriah) for pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), as well as a new indication for Genentech’s tocilizumab (Actemra) for the treatment of CAR T cell-induced severe or life-threatening cytokine release syndrome (CRS) in patients two years of age and older.

Inevitably there has been much hullabaloo and much anticipation over the expected price tag that might accompany the first cell therapy product approved in the US. Whatever your view on this, many of us were no doubt relieved it came in under $500K ($475K, with no charge for the product in the first month if there was a manufacturing failure or no response).  While undoubtedly pricey, frankly it could have been a lot worse given the relatively small patient population.

Of course, the approved therapy isn’t the only expected high ticket item – there’s also hospital costs (including highly trained physicians and nurses), ICU costs (for very sick patients), supportive care costs (including tocilizumab if CRS occurs), not to mention any lab, diagnostic or monitoring tests required. All of these will no doubt push the total bill nearer to $1M.  In children though, the lifetime value of curative intent and many additional years of life is a much easier to grasp concept for payers than adding a few extra months at a high cost in adults.

These issues do raise the stakes for Kite and what they plan to do strategically in aggressive lymphomas, where there is a larger pool of eligible people for therapy, which must be offset by lower response rates (vs. pALL) and likely lower durability based on the data we’ve seen to date.  If we are truly moving into a world of value based pricing in the US, then efficacy and tolerability will ultimately have an impact on the perceived cost and value of treatment.

As Warren Buffett, the famous value investor has been want to say, “Price is what you pay, value is what you get.”

Whoa that’s a lot to think about – who knows what Friday may bring at this rate – and we still have the Kite Axi-Cel approval in aggressive lymphomas to go yet…

Meanwhile, there’s also a high unmet medical need for new effective treatment options in Acute Myeloid Leukemia (AML), especially in the relapsed/refractory setting, which is why we’re firm supporters of the Beat AML trial that the Leukemia and Lymphoma Society are pioneering. (See: Interview with Dr Brian Druker).

There’s also interest from several companies in a variety of novel targets, including CD123.

Notre Dame, Paris

Cellectis recently announced the enrollment of the first AML patient into their trial of an allogeneic CAR T cell therapy (UCART123) targeting CD123.

Quite aside from the issue of addressing whether such a product can be administered safely and effectively, one major why there is notable interest in Cellectis is because an allogeneic CAR T cell therapy offers the potential of a much cheaper off-the-shelf product as well as enhanced performance from an abundance of fit immune cells from healthy donors rather than tired or exhausted ones from people who are sick, thereby reducing the risk of manufacturing failure.

While in Paris, I spoke with Cellectis Chief Medical Officer, Loan Hoang-Sayag, MD about the trial design and CD123 as a target in AML.

This is the third and final post in our summer mini-series on gene editing and allogeneic CAR T cell therapy.

The first in the series featured an interview with Professor Waseem Qasim (Link), and the second with Cellectis CSO, Dr Philippe Duchateau (Link).

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Paris – amazingly it’s now 3 years since we interviewed Cellectis (NASDAQ: $CLLS) CEO André Choulika and CSO Philippe Duchateau (See post: Can Cellectis revolutionise CAR T cell therapy):

Cellectis CSO CEO

Cellectis Senior Management – Drs Duchateau and Choulika

Since then, we’ve followed the company over time, including an interview with one of their leading scientists, Dr Julianne Smith at ASH 2014, followed by the initial results of their first allogeneic CAR T cell therapy UCART19 presented at #ASH15 by Professor Qasim.

It’s hard to believe 3 years have gone by so quickly! As regular readers know what we often do on BSB is follow stories longitudinally, so while in Paris for an Immuno-Oncology Summit we thought it a rather timely opportunity to revisit Cellectis and take stock of where they’re at and ask what the future may hold for them?

With the recent news that Gilead have acquired Kite Pharma, there’s going to be a lot of interest in what companies such as Cellectis are doing to bring allogeneic “off the shelf” CAR T cell therapy to market.

This is the penultimate post in our summer mini-series on gene editing and allogeneic CAR T cell therapy and features a candid interview with Dr Philippe Duchateau, Chief Scientific Officer, at Paris based Cellectis.

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When we think of pioneers in the CAR T cell therapy space, one person who comes to mind is Waseem Qasim, Professor of Cell and Gene Therapy at the Institute of Child Health at University College London, and a Consultant Immunologist and Pediatrician at Great Ormond Street Hospital (GOSH).

Institute of Child Health

As readers may recall back in 2015, he gave the first allogeneic CAR T cell therapy under compassionate use to an infant with ALL, and in the process undoubtedly saved her life.

The subsequent case report published in Blood was the talk of 2015 annual meeting of the American Society of Hematology (ASH) in Orlando.

The poster focused on the first child that Prof Qasim treated and attracted a phenomenal amount of attention:

Prof Qasim UCART19 #ASH15 Poster

Where are we now with allogeneic CAR T cell therapy?

It’s been 18 months since we spoke to Prof Qasim, so while in London over the summer BSB caught up with him in his office at the Institute of Child Health.

This interview is the first in our latest 3-part mini-series on allogeneic CAR T cell therapy, which runs throughout this week. Here’s a teaser clip:

Kite’s first autologous product, Axi-Cel (in aggressive lymphomas), heads for regulatory approval in the US (PDUFA date November 29th), offering Gilead a hematology launch product with a high unmet need and, presumably, a relatively high price tag to match. Inevitably, some critical attention will subsequently be focused on the pipeline and whether they will move towards allogeneic CAR-T cell therapy (reduces cost of goods and increases profit margin) as well as how the TCR platform in solid tumours will fare.

It’s certainly a timely point to consider allogeneic CAR T cell therapies again given that things are rapidly heating up in the cell therapy niche following the Gilead announcement yesterday that they are acquiring Kite Pharma for $11.9 Billion.

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Paris: If cancer immunotherapy is a revolution in how cancer is treated, then Dr Jérôme Galon is a revolutionary.

In addition to being a Research Director at INSERM in Paris, Dr Galon is one of the co-founders of Marseille based HalioDx, an immuno-oncology diagnostics company that is commercializing the research from his laboratory.

Last month, while in Paris, I had the great pleasure to talk with him in his office at the Centre de Recherche des Cordeliers (CRC) on the left bank of Paris.

At the time of the French revolution in 1789, it was the gathering place of the “Club des Cordeliers,” for famous revolutionaries such as Danton, Marat, and Camille Desmoulins. Dr Galon told me it was where the Declaration des droits de l’homme et du citoyen de 1789 (Declaration of the Rights of Man and Citizen) was signed.

As such, it’s a very appropriate place to find a cancer immunotherapy revolutionary…

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Berlin Wall Museum

Preserved section of the Berlin Wall

I have a personal interest in Alzheimers Disease, my mother Audrey died from it three years ago back in 2014.

Since then, I’ve watched with fascination and excitement the progress made in using the body’s own immune system against cancer. There’s still a long way to go, but a revolution in treating cancer is underway, as we’ve been documenting on this blog and the Novel Targets Podcast.

In recent years in the United States we’ve also seen grand initiatives targeting cancer such as Vice President Biden’s Moonshot, as well as large philanthropic support e.g. the creation of the Parker Institute for Cancer Immunotherapy.

Sadly, we’ve not seen the same level of interest in targeting dementia or funding research into new treatments for Alzheimers disease.

In the United States, the media doesn’t talk much about Alzheimers (compared to cancer), unlike for example, in the United Kingdom where any promising data is heralded with headlines that frequently deliver “hype over hope.”

Alzheimers is an insidious disease that removes the ability of the person to advocate and care for themselves, instead placing the burden on families and caregivers, often for extended periods of time. Ultimately many people end up in supported living or nursing homes.

As we debate healthcare insurance in the United States, who is going to pay for the cost of dementia care as the population grows older? Caring for dementia is arguably the greatest public health challenge that the western world faces.

Which is why I was excited to talk with a researcher who is thinking outside of the box and leading the way in how we could use our immune system against Alzheimers.

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Paris, France – Last week I had the pleasure of attending a two day Immuno-Oncology Summit, an industry sponsored CME event organized by the European Academy of Tumor Immunology (EATI) and Miltenyi Biotec.

The summit was held at the Centre de Recherche des Cordeliers (CRC) in the quartier Latin on the historic left bank of Paris, a short walk away from Notre Dame cathedral.

Paris Immuno-Oncology Summit 2017

Most of the attendees were French researchers so this opportunity afforded them a chance to hear from leading researchers at the forefront of cancer immunology, including several who travelled from the United States to speak at the event.

CRC Courtyard Statue

I have yet to attend a cancer immunotherapy meeting where I didn’t come away with new insights into what is a fast moving field, where it’s important to see “connections” beyond a tumor type or target.

This post offers top-line commentary highlights on five key presentations at the summit. There were two parallel tracks and a lot of interesting speakers at what was an enjoyable meeting, so think of this like a postcard from Paris.

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Florence, Italy: Today at the EACR-AACR-SIC Conference on “The Challenges of Optimizing Immuno and Targeted Therapies,Tom Powles MRCP MD, Clinical Professor of Geniturinary Oncology at Barts Cancer Institute in London, gave a special lecture on the IMvigor211 trial (NCT02302807).

EACR AACR SIC Conf Banner

This was a phase 3 study of the PD-L1 checkpoint inhibitor atezolizumab compared with chemotherapy in participants with locally advanced or metastatic urothelial bladder cancer.

Prof Tom Powles (Barts)

Prof Tom Powles (Barts Cancer Institute)

Readers may recall we interviewed Prof Powles back in August 2015 about the potential for the PD-L1 checkpoint inhibitor atezolizumab in urothelial bladder cancer? (See post: Atezolizumab PD-L1 Checkpoint Inhibitor will Change Bladder Cancer Treatment.)

We also featured the atezo data presented by Dr Jonathan Rosenberg (MSKCC) at ESMO 2015 on Episode 7 of the Novel Targets Podcast, where we also heard Prof Powles tell us about the long durable responses he had obtained in clinical practice in some of his patients.

Subsequently on May 18, 2016 the US Food and Drug administration (FDA) granted accelerated approval to atezolizumab (Tecentriq) for urothelial bladder cancer (link to news release).

Fast forward a year to May 9, 2017 and the surprise announcement that the confirmatory phase 3 trial (IMvigor211) failed to meet its primary endpoint (link to Genentech press release).

So what happened? Why did the atezo phase 3 trial end up being negative when we saw durable responses in the randomised phase 2 trial and other PD-1 checkpoint inhibitors have shown an overall survival benefit in the same indication?

Many in the media only want to write about positive data, but in science we often learn as much from our failures as we do from our successes, perhaps even more sometimes.

IMvigor211 was expected to be a positive trial especially after the recent Merck success gaining an overall survival benefit for pembrolizumab, so the negative result is noteworthy and one that anyone in the field of cancer immunotherapy drug development will want to understand.

Professor Powles kindly spoke to BSB and shared his perspective.

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One of my favourite pastimes at cancer conferences is discussions with up and coming young researchers about their current experiments and what they learn from them.

The poster hall rugby scrum at #ASCO17

In the spotlight today is one of the gems from the poster halls at ASCO this month…

Here we explore how liver mets, which is a common site of metastases, can influence the response of cancer immunotherapy.

The findings from this research highlight some intriguing biology as well as offer some hints about where to go next.

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At AACR17 one of the fascinating topics that came up in several presentations was exosomes, what they are, and how the information they contain can be used to best effect.

One of the evangelists of exosomes, and their potential in cancer research is Theresa Whiteside, PhD who is a Professor of Pathology, Immunology and Otolaryngology at the University of Pittsburgh.

At the recent 2017 American Association for Cancer Research (AACR) annual meeting, Dr Whiteside gave two fascinating talks in education symposia.  Afterwards, she kindly spoke to BSB about her research.

Love them or hate them, exosomes were a hot topic in Washington DC and something you should be aware of, if you aren’t already.

This post continues our volley of expert interviews from AACR17 and is the ninth in the series.

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Long time attendees at the annual meeting of the American Association for Cancer Research (AACR) know that there are usually interesting posters and sessions buried on the last day of the meeting.

This year was no exception, with a major symposium on CAR T Cell Cancer Immunotherapy chaired by Michael Jensen MD (pictured right).

BSB readers will recall we interviewed him at the 2016 BMT Tandem meeting in Honolulu (See post: Optimizing CD19 CAR T cell therapy).  Excerpts from this interview also featured in Episode 14 – Cell Therapy Pioneers of the Novel Targets Podcast.

The CAR T symposium on the last day of AACR was one of my highlights of the meeting. The three speakers were:

  • Michael Jensen, MD (Seattle Children’s) Engineering Next Generation CAR T cells using Synthetic Biology-Inspired Technologies
  • Terry J. Fry, MD (National Cancer Institute) Defining and overcoming limitations of CD19 CAR immunotherapy in pediatric ALL
  • Christine E. Brown, PhD (City of Hope) Progress and Challenge in CAR T Cell Therapy for Brain Tumors

Each of these presentations would merit a full blog post in their own right, but in this particular post we’re focusing on CAR T cell therapy targeting glioblastoma multiforme (GBM).

GBM is the most common primary malignant brain tumor, and one with a dismal prognosis – the 5-year survival rate is only around 5%, so there is also a high unmet medical need for new effective treatment options. This devastating disease has proven to be a miserable graveyard for Pharma over the last decade, with many agents unfortunately ending up in dog drug heaven.

After her AACR17 presentation, Dr Brown kindly spoke to BSB.

This post is part of our series of thouight leader interviews from AACR17. It also continues our ongoing posts on the adoptive cellular therapy landscape, and in particular, CAR modified T cells.

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