Biotech Strategy Blog

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

Posts tagged ‘CD123 CART AML’

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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One of the most important challenges in cancer immunotherapy is overcoming immune resistance. For example, even with the high response rates seen in acute lymphoblastic leukemia (ALL) with CAR – T cell therapy, a significant number of patients relapse after an initial response.

Chinatown Honolulu

Chinatown, Honolulu 2016

Could immune resistance be reversed or prevented by the addition of appropriate checkpoint blockade? Which ones matter though, that is the critical question?  Rather than randomly picking ones to try, we need scientific evidence regarding these choices.

This post explores some of the latest data presented at the BMT Tandem meeting on the role of T cell immunoglobulin mucin–3 (TIM–3) and PD–1 upregulation in causing resistance.

If you’re not already a sub and want to read our coverage of ASH, BMT Tandem and the forthcoming AACR 2016 annual meeting, you can purchase individual access below. This week only – inspired by the story of Eddie Aikau in Hawaii – we have a special offer that we’ve never done before (and may never do again) of $75 off a quarterly subscription. The deal ends tomorrow Friday March 4th at 12 noon HST. Check it out!

Subscribers can login to read more about the latest data on how alternative checkpoint inhibitors may have a role to play in cancer treatment.  Welcome to the new folks who signed up this week, good to see y’all!

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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