Biotech Strategy Blog

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

Posts tagged ‘myeloma’

Continuing our latest cell therapy mini-series, this time around we focus on a novel and creative approach to CAR-T cell therapy, which is quite different from what we have described before with other companies in this niche.

One emerging trend is the development of bi- and even tri- specific approaches designed to target multiple aberrations in the cancer cells, but what’s the best way to achieve this? Suppose we ditch the core dogma and try another way of doing things?

The entirely new concept making the splash is also coming from an emerging young biotech company few readers will likely have heard of, yet what they are doing reminds me we can borrow from the past and paraphrase a watch ad from the 1980’s for elegant and simple timepieces – some day all CAR-Ts will be made this way.

The secret sauce this time around isn’t quartz, however, but something completely different…

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Friday at #ASH19 – remains of the day or hope springs eternal with sunrise on Saturday morning?

Orlando: The annual meeting of the American Society of Hematology or ASH (Twitter #ASH19) is one of the four key Majors on cancer-related related research meeting circuit that BSB attends each year.

Just as golf has the Masters, the US Open, the Open, and PGA championships, so those on the cancer new product development circuit attend AACR, ASCO, ESMO, and ASH.

This year ASH is in sunny Orlando where you have to compete with the tourists for Uber rides if you want to venture to restaurants or events in the area.

Friday at ASH has traditionally been associated with the satellite symposia, colloquially known as “Super Fridays” that CME companies or organizations such as the Leukemia Lymphoma Society have traditionally run, but in recent years ASH has put on its own Friday events to compete with both the industry satellites and also academic events such as the BMT Winter Workshop we have attended in the past.  More choice is good on one hand, but bad on the other in that something has to give way.

Ron Levy (Stanford) and Stephen Ansell (Mayo) blazed the trail a few years ago with their Friday Scientific Workshop on Tumor Immune Interactions in Lymphoid malignancies. Regular BSB readers may recall the interview at ASH16 with Dr Levy where he reviewed some of the data in that year’s workshop (See post: Targeting the tumour environment in lymphomas.)

This year on Friday at ASH19 there were multiple scientific workshops you could attend. What were some of the presentations that caught our personal attention, what can we learn from them and why did they matter?

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Orlando

Yesterday we looked at ten innovative approaches centred around T cell-based developments emerging from the American Society of Hematology (ASH) meeting that is taking place in Orlando next month.

Let’s not forget, however, that there are also other immune cells, including NK cells and quite a few others, which can be manipulated into cancer therapeutics for the treatment of hematologic malignancies.

Some of these are intriguing early preclinical research that may form next generation technologies in the future, while others take the form of up and coming early clinical data that readers may be interested to learn more about.

Here we highlight nine emerging immunotherapy approaches to consider that don’t involve T cells…

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Continuing our bispecific mini-series, we now switch from small to large biotech with a look at what Amgen are doing in this niche. They have both regular bispecifics, as well as T cell bispecifics in their early pipeline.

Our latest company interview focuses on several early phase 1 new product developments.

Aside from the BiTEs, we also discuss the clinical program with one of their most promising small molecules, AMG 510, a KRAS selective inhibitor that has been drawing much attention since the chemical structure was unveiled at AACR earlier this year.

There was much ballyhoo and yet more garish headlines in the media at ASCO regarding ‘Amgen showed it had developed a medicine that shrank tumors in 50% of lung cancer patients’ – in 10 patients. Was it really 10 people or a much higher number if we consider intent to treat amongst evaluable patients? Then of course, taking a small sample size into consideration, the next 10 might produce quite different results. We might also see resistance set in down the road (e.g. at 9 to 12 months as we have with BRAFi), so these are really very early days, something we pointed out during the daily ASCO coverage.

To be clear, I can say that both companies included in yesterday’s (Neon Therapeutics) and today’s (Amgen) articles were sensible, thoughtful, and well measured in how they handled the data rollouts, but the media frenzy that occurred with each is quite something else.

Since we had quite a few BSB readers ask about both sets of data, having discussed Neon’s yesterday, today we offer an interview with an Amgen exec at the heart of their early stage programs…

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This is an extended update that I’ve been planning to write for some time, however, there was always some shiny new clinical data to highlight and discuss so it sadly stayed on the backburner!

Is the sun rising on CAR-T cell threats from the East?

Over the next few weeks, we will post some meaty reviews on various topics pertinent to cancer research R&D. They might involve a particular tumour type that is seeing extensive developments, an important or difficult target, or even a particular category approach, for example.

In the first of this new mini-series, we take a look at the CAR-T cell therapy niche and highlight some important new developments that are well worth watching out for.

Things are a-changin’ very rapidly here, including numerous R&D threats from the east (China) so it’s a good opportunity to take stock and look forward.

Here we go – hold on to your hats…

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After the snow flurry in Atlanta that induced much panic and minor frustration in equal measure after the trials and tribulations of a tiring travel day with delays and cancellations galore. Then you see attendees waking up to a veritable winter wonderland in the south:

Meanwhile, things soon settled down somewhat and life got back to normal at the American Society of Hematology (ASH), as this quick time lapse that 3NT recorded showed down the main thoroughfare :

It’s time for our annual meeting daily highlights and lowlights because after all, oncology R&D is not a rose tinted garden and thus not every session or compound you highlight pre-conference will turn out as expected and sometimes, there are pleasant surprises that few see coming so you could end up at sixes and sevens if you don’t watch out…

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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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As 2013 draws to a close, I though it would be a good time to add one last ASH post before finishing for the year. More to come in the form of the tumour summaries in January.

One of my favourite activities at conferences is finding interesting gems in the poster hall. In New Orleans this year there were not one, but two huge halls! That’s a lot of shoe leather involved in order to browse, chat with investigators or researchers and cover them all.

So what nuggets stood out to me this year?

Companies mentioned: KBIO, Gilead, Incyte, Seattle Genetics, Array, Amgen
Drugs covered: KB004, momelotinib, ruxolitinib, idelalisib, brentuximab (Adcetris), filanesib (ARRY-520), carfilzomib

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