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Philadelphia – at the 2015 annual meeting of the American Association for Cancer Research (AACR), new data was presented that showed checkpoint inhibitors have a greater effect when they work in combination, they may also offer a new effective treatment option in Triple Negative Breast Cancer (TNBC).

Are two checkpoints are better than one?

At AACR 2015, F. Stephen Hodi MD (Dana-Farber Cancer Institute) presented results, published simultaneously in the New England Journal of Medicine, that showed in advanced melanoma, combining two checkpoint inhibitors (nivolumab and ipilimumab) showed better results than with one alone (ipilumumab). The authors in their NEJM paper conclude:

The objective-response rate and the progression-free survival among patients with advanced melanoma who had not previously received treatment were significantly greater with nivolumab combined with ipilimumab than with ipilimumab monotherapy. Combination therapy had an acceptable safety profile.

What is the potential for checkpoint inhibition in TNBC?

Yesterday at AACR, Leisha S. Emens MD, PhD (Johns Hopkins) presented the results in TNBC from a phase 1 trial of MPDL3280A (Roche/Genentech), a checkpoint inhibitor that targets the PD-L1/PD-1 signaling pathway.

Dr Emens (right) is shown in the picture below presenting at an AACR media briefing moderated by Louis M. Weiner MD, Dr Hodi is pictured left.

AACR 2015 Press Briefing

The only currently available treatment for TNBC is chemotherapy, but sadly patients often do not live long, and rapidly progress. Progression-free survival (PFS) is estimated to be around 4 months in TNBC. This means there is a real unmet medical need for effective new treatments. The fact that cancer immunotherapy, and in particularly checkpoint inhibitors targeting the PD-L1/PD-1 signaling pathway may have potential in this disease is huge.

Cancer immunotherapy and in particular checkpoint inhibitors are a hot topic at AACR. In this post we look in more detail at the data presented.

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Philadelphia – the 2015 annual meeting of the American Association for Cancer Research (AACR) is in full swing, with over 18,000 attendees, it’s probably the world’s largest meeting dedicated to cancer research. The theme is “Bringing Cancer Discoveries to Patients.”

AACR 2015 Annual Meeting Philadelphia

I challenge anyone not to attend, and come away inspired with new ideas on how the field of cancer research will evolve in coming years.

At this year’s annual meeting, not surprisingly, cancer immunotherapy is one of the hot topics. Yesterday there was the simultaneous publication of two papers in the New England Journal of Medicine (NEJM) to coincide with data presented at the meeting.

Pembrolizumab (Keytruda)  for the Treatment of Non–Small-Cell Lung Cancer

The conclusion of the paper by Edward B. Garon, MD (UCLA) et al was that:

Pembrolizumab had an acceptable side-effect profile and showed antitumor activity in patients with advanced non–small-cell lung cancer. PD-L1 expression in at least 50% of tumor cells correlated with improved efficacy of pembrolizumab.

The other paper published in the NEJM was for:

Pembrolizumab (Keytruda) versus Ipilimumab (Opdivo) in advanced Melanoma.

The conclusion of the paper by Caroline Robert, MD PhD, presented at AACR by Antoni Ribas, M.D., Ph.D.(UCLA) was:

The anti–PD-1 antibody pembrolizumab prolonged progression-free survival and overall survival and had less high-grade toxicity than did ipilimumab in patients with advanced melanoma.

AACR 2015 Press Briefing with Dr Topalian, Dr Ribas, Dr Garon

Dr Ribas (left) and Dr Garon (right) are pictured at an AACR media briefing chaired by Dr Suzanne Topalian (Johns Hopkins).

This year’s AACR annual meeting is to paraphrase Bertrand Tombal, “a Grand Cru year”. Not only in cancer immunotherapy, but in metabolism, epigenetics and advances in drug discovery.

We’re excited about the prospect of another three days at the meeting, but in the meantime in this post there’s some top-line thoughts for subscribers on some of the data that caught our attention over the weekend.

In recognition of AACR, there’s $50 off a quarterly subscription if you wish to purchase access (offer valid only during the week of the meeting – ends this Friday!)  If you’ve been sitting on the fence for a while wanting to try out BSB, now is a good opportunity to dip your toes in the water.

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European Lung Cancer Conference 2015Geneva – at the 2015 European Lung Cancer Conference today, Pasi A. Jänne, MD, PhD presented updated progression free survival and duration of response data for the phase 1 AURA trial of AZD9291 (AstraZeneca) in patients with EFGR-TKI-resistant advanced non-small cell cancer (Abstract LBA3).

Dr Jänne (pictured below at ASCO 2014) is Director, Lowe Center for Thoracic Oncology at the Dana-Farber Cancer Institute and a Professor of Medicine at Harvard Medical School.

Dr Pasi Jänne ASCO 2014

It’s hard to believe that it is only about two years since the first patient was enrolled in the phase 1 AURA trial of AZD9291, a third generation EGFR inhibitor. If the FDA regulatory submission takes place, as expected, in the second quarter of this year, then the drug could be approved for sale in the United States before the year end.

It has been fascinating to watch the race to market between rociletinib (Clovis Oncology) and AZD9291. It’s likely both could be approved in the United States before the year end.

That would be great news for lung cancer patients, given the absence of any approved therapy for patients who develop a T790M mutation and become resistant to EGFR inhibitors, such as Tarceva and Iressa.

Readers will know that we have been following the phase 1 AURA trial of AZD9291 since ECCO 2013 in Amsterdam, when the first clinical data was presented.

AstraZeneca are to be congratulated on what is a case study of rational scientific drug development; their path to market strategy highlights the benefit of well-designed early clinical trials. AZD9291 may end up receiving regulatory approval less than three years from the start of the first in man trial – that’s tremendous!

I had the privilege to interview Dr Jänne at ASCO last year, and again earlier this week, before he left Boston for Geneva and chatted with him about his AZD9291 presentation at European Lung.

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The 2015 Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2015) was held in Istanbul from March 22-25, where it offered a European perspective on some of the latest developments in cancer immunotherapy. 

EBMT 2015 banner

We’ve heard a lot in the United States about the early CAR T cell therapy clinical trial results from institutions such as UPenn, CHOP, MSKCC, Fred Hutchinson, Seattle Children’s, the NCI, and MD Anderson to name but a few, so it was good to see a leading a European center join the club: University College London (UCL).

While completing a Masters degree in Human and Applied Physiology at King’s College London, I spent several weeks training at UCL and particularly enjoyed the intercollegiality of the University of London.

At EBMT15, Dr Sara Ghorashian Clinical Training Fellow at the Insitute of Child Health at UCL, presented data on a phase 1 trial of Epstein Barr virus (EBV) specific T cells transduced with a first generation CD19 Chimeric Antigen Receptor (CAR). The trial data was first reported by Dr Ghorashian (pictured below) in an oral presentation at #ASH14 (Abstract 383).

Dr Sara Ghorashian ASH 2014

Dr Ghorashian stated at EBMT that UCL have several CAR T cell therapy trials planned.

autolus-logoReaders will be aware that earlier this year that UCL spun-off a series A funded company, Autolus, to commercialize their CAR T cell therapy research.

Although £30m from Syncona (a subsidiary of the Wellcome Trust) is not a lot of money by US investment standards, UCL is nonetheless a European center to watch if you have an interest in the CAR-T competitive landscape.

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EAU 2015 LogoMadrid, Spain – the results of the Medivation/Astellas TERRAIN clinical trial of enzalutamide (Xtandi) versus bicalutamide (Casodex) in men with metastatic castration resistant prostate cancer (mCRPC) were presented today at the European Association of Urology Congress in Madrid (Twitter #EAU15).

Professor Dr. med. Axel Heidenreich. Credit: Universitätsklinikum Aachen

Credit: Universitätsklinikum Aachen

The clinical trial data were presented in a plenary session at EAU15 by Axel Heidenreich (pictured left) who is Professor of Urology & Uro-oncology at the RWTH University and Head of Department & Director of the Urology Program at the University Hospital in Aachen, Germany.

How good are the results, and what impact will they have on the prostate cancer treatment landscape in Europe? Prof Heidenreich kindly spoke with Biotech Strategy Blog (BSB) and shared his thoughts.

Subscribers can login to read the full interview or you can purchase access by clicking on the blue Tinypass icon at the bottom of the page.

Update May 17, 2015: This post has been updated with the additional TERRAIN trial data presented by Professor Arnauld Villers (Lille) at the 2015 annual meeting of the American Urological Association (AUA) in New Orleans.

Prof Arnauld Villers presents TERRAIN trial data at AUA 2015

Prof Arnauld Villers presents TERRAIN trial data at AUA 2015

It remains exciting times in cancer immunotherapy with breakthrough new cell therapies and checkpoint inhibitors that enhance the effectiveness of T cells.

Cellectis LogoLast Friday, Paris based Cellectis filed their IPO registration statement with the Securities and Exchange Commission (Link to F-1).

They plan to raise $115M through an offering of American Depository Shares. You can read more about their allogeneic Chimeric Antigen Receptor (CAR) T cell approach in the two interviews we did with senior management last year.

Here’s an excerpt of the interview Cellectis CEO André Choulika, PhD gave Biotech Strategy Blog last year – it was the No1 post in 2014: Can Cellectis Revolutionize CAR-T cell Immunotherapy?

As multiple companies seek to move CAR-T cell therapies forward in clinical trials, what will be interesting to see is how this novel treatment fits in with existing therapies such as bone marrow transplants. Will it replace them, or be a bridge to a transplant that enables relapsed or refractory patients to have a second chance?

In addition, where are the potential opportunities beyond B-cell malignancies such as acute lymphoid leukemia (ALL) where there’s been dramatic success, particularly in children?

Dr Krishna KomanduriLast week Biotech Strategy Blog had the privilege to interview Dr Krishna Komanduri who is Director of the Adult Stem Cell Transplant Program at the University of Miami Sylvester Cancer Center and holds the Kalish Family Chair in Stem Cell Transplantation.

A physician scientist, he exudes a sense of calm professionalism – I am sure this must reassure many of his patients. Having a bone marrow transplant has been likened to jumping off a cliff in terms of what it does to one’s immune system.

In the last 2-3 years, he has dramatically increased the number of transplants at the University of Miami Sylvester Cancer Center.

Dr Komanduri (@DrKomanduri) was co-chair of the 2015 BMT Tandem meeting that took place earlier this month in San Diego. It’s the combined annual meeting of the American Society of Blood and Marrow Transplantation (ASMBT) and the Center for International Blood and Marrow Transplant Research (CIBMTR).

In a half hour interview he shared his thoughts on what was exciting at Tandem, where the field is going and some of the best abstracts at the meeting which included data on CAR-T cell therapy, GVHD and gene therapy.

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PLA General HospitalThe announcement earlier this week that Cellular Biomedicine Group (NASDAQ: CBMG) has acquired rights to the Chimeric Antigen Receptor T cell (CAR-T) therapy of the PLA General Hospital in Beijing (pictured right) should come as no surprise to industry watchers. (Link to Press Release).

The share price in $CBMG has risen from $16.31 on February 4 to $23.60 as of close of business on Feb 10, 2015 in what looks like a poorly kept secret!  It looks like most of the rise in share price took place immediately prior to the company’s formal Feb 9, 2015 announcement of the Chinese deal.

CBMG Share Price


Those following the cancer immunotherapy space have known for some time that several Chinese groups are working on CAR-T cell therapies that could be a threat if licensed or acquired.

Given the significant investor interest in this space, which is almost bordering on “tulip mania,” it’s entirely foreseeable that companies looking to share in this opportunity would go looking towards China.

One investor on Twitter in response to this news asked should Chinese data be trusted?

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Yumanity logoWe recently wrote about Syros Pharmaceuticals, one of whose founders, Dr Rick Young is based at the Whitehead Institute of MIT in Cambridge MA.

Another biopharma start-up company being spun out from research done at the Whitehead Institute for Biomedical Research is Yumanity Therapeutics.

The company recently launched with Tony Coles as CEO and Ken Rhodes as Chief Scientific Officer. Their focus is on transforming drug discovery for neurodegenerative diseases caused by protein misfolding.

The scientific founder is Dr Susan Lindquist, who spoke with Biotech Strategy Blog about her research and the Yumanity approach to drug development.

The company is committed to “improving human conditions. That’s why we call it Yumanity. The Y is for yeast, but it really is focused on humanity,” said Lindquist.

Dr Linquist started her interview by noting that as we live longer, we are more likely to get neurodegenerative diseases, starkly noting the reality of the lack of progress in drug development in this area:

“There is really, right now, nothing that we can do about them. We just do not understand how to move the needle on these and it’s really becoming an absolute crisis and it is taking a very substantial section of our healthcare budget as it is. As we continue to make better inroads against cancer and HIV and all of the other ills of mankind, it’s just going to get worse, I think. Everybody is beginning to appreciate that there is going to be an economic disaster and that we are going to ruining the next generation in a way that, at this point, is going to be tragic.”

So what is the approach Yumanity is taking, in the hope of succeeding where others have failed?

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Bellicum logoSeveral subscribers have written to ask what we think of Houston based Bellicum Pharmaceuticals?

Bellicum is a company that along with Novartis, Kite, Juno and Cellectis has a Chimeric Antigen Receptor (CAR) T cell therapy in development, amongst other things.

Readers already know the company had a successful IPO in December (NASDAQ: BLCM) and were reported to have raised $140M to fund future development.

This morning, the company announced enrollment of the first cohort of pediatric patients in a phase 1/2 dose escalation trial of BPX-501 (link to press release). This T cell therapy aims to mitigate the risk of graft versus host disease (GvHD) after an allogeneic haploid hematopoietic stem cell transplant.

BSB spoke with Bellicum CEO Tom Farrell and COO Dr Annemarie Moseley to answer some of the questions we think subscribers would like to know more about such as:

  • Market opportunity for BPX-501
  • Mechanism of action of BPX-501
  • Strategic direction the company is taking
  • Vision with regards to its CAR-T development
  • Milestones expected in 2015

We’ve provided some additional commentary on the challenges and opportunities Bellicum may face in the GvHD market and how we think the company stacks up against the competition in the CAR-T space. Be warned this piece is a long read: 6,000+ words!

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Readers don’t need Biotech Strategy Blog to tell them that Chimeric Antigen Receptor (CAR) T cell therapy (CAR-T), along with Checkpoint blockade, is one of the hottest areas of cancer drug development.

The last two days have seen pre #JPM15 deal activity with Kite Pharmaceuticals ($KITE) announcing a commercial collaboration with Amgen ($AMGN), which is not surprising given several of the Kite senior management team previously worked at the company.

Meanwhile, both Seattle based Juno Therapeutics ($JUNO) and Houston based Bellicum Pharmaceuticals ($BLCM) had successful IPO’s at the end of 2014. Interestingly, Bellicum are initially focusing most of their IPO funds, not on bringing their CAR-T to market, but on a novel cell therapy post stem cell transplant that aims to lower graft versus host disease (GvHD). GvHD is something we’ve been writing about regularly here!

Just this morning we’ve seen yet more CAR-T activity, with European Cardio3Biosciences (Euronext Brussels and Paris: CARD) acquiring the CAR-T technology of Oncyte (the oncology division of privately-held U.S. biotechnology company Celdara Medical).

There’s certainly a lot of activity in the CAR-T space and I expect we will hear more at next week’s JP Morgan Healthcare conference in San Francisco (#JPM15). One player in the CAR-T space who has not been gaining as much attention, and one that I think should not be dismissed, is Paris based Cellectis (Alternext: ALCLS.PA), who struck deals with both Servier and Pfizer last year. In June, BSB went to Paris and interviewed Chairman and CEO André Choulika, PhD and CSO Philippe Duchateau, PhD. At the recent American Society of Hematology (ASH) annual meeting in San Francisco, Julianne Smith, PhD (pictured below), Vice President CART Development at Cellectis, gave an in-depth interview to BSB. Dr Julianne Smith Cellectis ASH 2014 Interview Some key questions to address here are what are some of the important milestones for Cellectis in 2015 and and what makes the Cellectis CAR-T approach different from other companies in this space? Update Nov 7: This post now has two updates relating to the important news that came out after this post was published concerning the issuance by the USPTO of a gene editing patent that covers Cellectis’ intellectual property.  Subscribers can login to read more or you can purchase access by clicking on the blue icon below.

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