CML Advocates launch Leukaemia App

CML Advocates Network has launched its new mobile Chronic Myeloid Leukaemia (CML) adherence app called CML Today. It’s for CML patients to help them support adherence to tyrosine kinase inhibitors (TKIs) as well as to track their polymerase chain reaction (PCR) results.

The mobile app is available for FREE in English, French, Spanish, German, Portuguese, Arabic and Hebrew. More languages will follow soon.

The App will allow patients to:

• track the regular intake of their medicine
• remind them to take their prescribed medication
• track their PCR test results and other laboratory parameters
• facilitate patients connect with local support groups in their country.

I’ve downloaded the App for my iPhone and have found it easy to use and very clear; the reminder function is excellent. Whilst there are several Apps that support CML patients this is the only one, to my knowledge, developed by a patient advocacy network. I know the people who have developed it and I trust them, very important with medical data of this nature. This App gets my seal of approval and a big thumbs-up from me. Well done CML Advocates Network.

Find CML Today in the Apple App Store (iphone) and in the Google PlayChronic (Android).

https://itunes.apple.com/de/app/cml-today/id1038000286?l=en&mt=8
https://play.google.com/store/apps/details?id=com.appropo.cml_adherence_app&hl=de

Thanks, Kris

CML HORIZONS 2014 DAY 1

It was a long journey and an early morning. Sure, some people have travelled further and probably got up earlier but, sat writing this, I feel the pain. It was good to finally arrive in Serbia for CML Horizons 2014, no thanks to Air France who tried to derail me at every step. 

I was sad to have missed the opening video from Dr. Brian Drucker and the tribute to John Goldman, that pesky journey has much to answer for. I look forward to seeing the video and tribute on the CML Advocates website. So, things kicked off with a trip around the world with advocacy sessions: Latin America, Africa & Middle East, Asia-Pacific, North America and  Europe. This whistle-stop tour featured a key delegate from each region who presented an overview and the main challenges they faced. I was particularly struck, from Greg Stephens’ presentation, by the huge increase in CML deaths in North America. Whilst Greg is working on trying to understand what is causing this disastrous trend, I’d take a bet on it coming down to finance. The same old story. Very disappointing. It’s even more disappointing coming on the back of the excellent Leukaemia & Lymphoma Research, Impact Day, in London where we heard about the fantastic progress being made in the world of blood cancer.

Jan Geissler did his best to redress the balance with a more healthy European outlook but as we move West to East across Europe the challenges become greater. More disparity. All of the regions are working hard to bring a fair distribution of drugs that ultimately save lives. 

To the first medical session of the conference; Dr Neil Shah updated the delegates with the latest available treatments. Whilst we touched upon various forms of treatment including stem cell transplants and interferon-alpha the focus was on TKIs. The long term IRIS trial (7-8 year follow up), which is sadly coming to an end, shows a 93% survival rate considering only CML deaths for patients on Imatinib. Dr Shah felt that Imatinib has outperformed all expectations. We were presented with data that shows nilotinib and dasatinib as superior treatments to imatinib. For example in trails the complete cytogenetic response rate by 12 months on dasatinib is 83%, for imatinib 72%. For major molecular remission rate by 48 months, 76% on nilotinib and 58% on imatinib. This isn’t to say that imatinib isn’t the wonder drug it was first heralded as, without it this stunning progress could never have been made; and for those patients it suits, it works incredibly well. 

Next up, a fascinating update on therapy recommendations from Prof Giuseppe Saglio from the University of Turin, who really drilled the data down for us and showed a very logical progression of drugs and optimum responses. It is very clear that new drugs have given our clinicians excellent ammunition in the treatment of CML.

Finally, Dr Qian Jiang from the Peking University People’s Hospital who spoke about the role of transplant in CML. Whilst it is still a very important part of treating CML, the number of operations has steeply declined since the advent of TKIs. It is however, fascinating to see continuous development and improvement in this key area.

After a short question and answer session the day broke and we all raced back to our rooms to prepare for dinner. It was wonderful to catch up with old friends and see many new faces. Most conversations started, “has it really been a year?”. 

The CML community is lucky to have such a vibrant, resilient and informed group of people. With hard work and good bonds the group have take an orphan disease and put it firmly on the blood cancer map. Better than that, they have shown the way. The group is keen to talk about ‘advocacy’ and ‘blood cancer’ rather than isolate itself by being singular. This is the key, the group understands that the progress being made here and now will affect cancers of all types for generations to come.

Today has really set the pace for a fantastic conference. Stay tuned…same CML time….same CML channel.

NEW CML Resource & Knowledge Centre

Trust those great people at CML Advocates Network to drop a brilliant new resource in our lap. This is well worth checking out. K

Over the next three years, CML patients in more and more countries will be facing the use of generic TKIs and copy drugs to treat CML.

To provide patient organizations with background information on this important issue which has so far been quite difficult to find, we have launched a Resource & Knowledge Center on CML generics, copy drugs and substandard drugs.

Please see new section here: http://www.cmladvocates.net/generics

It provides:

• an unofficial directory listing all CML tyrosine kinase inhibitors (TKIs) that are – to our knowledge – available to date:  http://www.cmladvocates.net/generics/cml-drugs-register
• the results of our survey on generics, copy drugs and substandard drugs in CML which was conducted by CML Advocates Network in collaboration with iCMLf in March 2013. The survey summarizes 86 responses from 55 countries – the data is also available for download.
• the webstreams of our session on CML generics and substandard drugs at “CML Horizons 2013”, which featured a presentation of the WHO, of the CML Association of Serbia and the CML Advocates Network
• an index of scientific articles on the use of generic drugs in CML treatment: http://www.cmladvocates.net/generics/generics-publications
• our blog on CML generics. (This is only available after login for members of the CML Advocates Network to keep discussions private in the community) http://www.cmladvocates.net/generics/generics-blog
• a glossary with key terms and definitions in the area of drugs and generics: http://www.cmladvocates.net/generics/glossary

We will soon complement this by a “Best Practice Toolbox” for advocacy on generics in a few weeks, which is currently under construction. The “toolbox” will provide useful advice and tools on how to address this topic as an advocacy organization when generics or copy drugs are being introduced in your country.

Finally, we would like to stress that the Resource & Knowledge Center on CML generics, copy drugs and substandard drugs is a purely patient-driven, non-commercial initiative. It has no interest to promote, or assess, any of the drugs. The only intent is to increase transparency in a confusing environment.

We hope that you will find this Resource & Knowledge Center very useful!
We are looking forward to your active participation in the blog and the new knowledge center, and are happy to receive your feedback!

Nicole, Giora and Jan
CML Advocates Network –
http://www.cmladvocates.net

Tom Watson MP joins our cause!

I’m still trying to get my head around today! It may just be the most amazing thing to happen to the AccessCMLDrugs campaign to date. Tom Watson who is the Member of Parliament for West Bromwich East, Deputy Chair of the Labour Party and the Labour Party’s Campaign Co-ordinator has agreed to support our cause!

We met this afternoon in West Bromwich and spent some time going over the issues affecting CML patients, namely the unavailability of dasatinib. We discussed, in detail, the STOP trial data, the effectiveness of dasatinib, what the TKI (tyrosine kinase inhibitor) drugs mean to CML patients, survival rates and the cancer drug fund. I told him about our campaign so far and how hard it is get our voice heard, especially by NICE, the pharmaceuticals and his own party. He agreed that it is important to ensure that all CML drugs are available to patients, free at the point of delivery. He has told me that he will do all he can to support us, he looked me in the eye and he told me that he believed in what we are trying to do.

To say I was blown away is an understatement. Tom is one of the most powerful men in politics today and I’d gone prepared; I’d even taken my box of dasatinib to show him what this is all about. He was sharp, working out quickly how much the NHS could save if the STOP trials findings are conclusive and adopted in England. These being the ongoing trials where 40% of patients who achieve CMR (Complete Molecular Remission) over a period of time come off their drugs and don’t need them again. The other 60% take the drugs again and the leukaemia goes back into remission. Hopefully you never tire of me writing about this as a cure for cancer.

So that’s it in a nutshell. Tom wants me to put a report together and propose how I want him to help out. He was sincere, friendly and genuinely interested in leukaemia and blood cancer patients. I sat in my car for 20 minutes afterwards just taking everything in, it was emotional. The implication on what we are trying to achieve is huge and having someone like Tom behind us is simply fantastic (sadly that doesn’t feel like a big enough word).

I need to start gathering research and writing up that report but in the meantime I’m sure you will join me in thanking Tom for taking an interest in a handful of poorly people and their little fight to stop a big disease. Thank you.

Kris

Tom Watson, Member of Parliament (MP) for West Bromwich East, Deputy Chair of the Labour Party and the Labour Party’s Campaign Co-ordinator, with CML campaigner and patient Kris Griffin

CML Horizons Conference, Prague – Day 2

That was a late night! I have a habit of doing this, always the last to leave the party. I won’t tell you what time I crawled into bed, what happens in Prague stays in Prague. So after an omelette, espresso and eye cream the day begins. Upon entering the conference room I’m grabbed by a delegate I met last year who asks me how I am able to look younger than I did last year? Today is going to be a good day!

Jane Apperley, a leading light in haematology and transplant developments from the UK provides an interesting insight into the science of transplants and if they still have a role in treating CML. Of course the answer is yes. It’s clear that TKIs have helped and there is great work still happening, survival rates are good and improving, it’s becoming clear that an early transplant decision presents a better outlook. I look forward to sharing Jane’s slides with blog readers.

Tim Hughes took the stage again to present the latest data on STOP trials. It’s clear that his TWISTER trial is backing up the data on the French trial, 40% of patients stopping imatinib therapy under controlled conditions stay in remission. Where the CML came back treatment resumed and all patients returned to remission. Success criteria improved with combination therapies. Recent STOP 2G-TKI in France have shown that there is a success rate of around 60% with patients on dasatinib and nilotinib who stop under controlled circumstances. There are clearly many STOP trials happening, results are promising, interferon, and possibly immunotherapy, certainly help and it is well worth us keeping our eye on results. It’s important we talk about this and tell people, especially politicians, this is the cure we allude to but worry about pinning our hopes upon. Our hopes are well founded and just in the last year I’ve seen rapid developments.

“Killing the Last Leukeamic Cells” certainly got the attention of the audience and this was a genuine statement. Who’d have thought we would be having this conversation 10-15 years ago? The key, we found out, is detection and a far more sensitive method of tracking but, again, the talk was of cure and absolute belief that this can happen.

I thoroughly enjoyed giving my talk about being connected and using social media as an advocacy tool. It was a very humbling experience to be invited and I’m very grateful to the steering committee for the opportunity. Things went well, I felt like I could have gone on and on, always the same when you are enjoying yourself but I hope delegates were enthused and understood the power of being connected.

The afternoon session covered vital topics like quality of life, adherence and pregnancy. Fabio Efficace presented detailed data on quality of life and the devastating effects of fatigue on the CML patient. Understanding side-effects is key to developing new treatments and managing old ones. Quality of life is something that must go hand-in-hand with staying alive.

On the subject of staying alive we discovered that one fifth of patients surveyed in a recent poll were non-adherent (they didn’t take their tablets when they should). We know that high adherence is key to treatment success so this comes as a surprise. We need to support patients and inform that taking medication every day is important. It is easy to shirk responsibility and hope that someone else manages the message but the reality is that this is collective responsibility and we must all play a part in supporting the CML community and communicating how important adherence is.

Doctor Jane Apperley took to the stage again this time to present CML and pregnancy. There are clearly issues taking TKIs and the advice was for women to come off treatment during pregnancy. For men sperm banking is the safest method. It angers me when I read self confessed experts giving bad advice, particularly about pregnancy. This is not an issue we should be gambling with. Professor Apperley is a leading light on this subject and she is very passionate about enabling patients to become parents. Ultimately there is another life here at stake. I am thankful to my first consultant who ensured I sperm- banked on diagnosis. Without that advice I wouldn’t be a Dad now; thinking about my little boy Luca, this is a very sobering thought.

The day concluded with patient groups and advocates from around the world presenting the work their groups were doing. Bringing developments together in this way certainly gave the day a truly an international feel. It certainly bodes well for International CML Day later in the year.

The evening brings a chance to socialise and relax away from the hotel. What for breakfast tomorrow? What time will I get to bed? And how many more times will I try and crack a joke with a non-English speaker only for it to fall flat?

All these answers and more tomorrow. Same bat-time…same bat-channel.

Kris

The CML “club” in Prague

It’s late, I’m writing this in bed an hour after I had the BEST club sandwich I’ve ever eaten. I’ve spent most of the day travelling, finally arriving in Prague just a few hours ago. I’m speaking at the CML Horizons 2013 conference which is being held over the next 3 days. I was lucky enough to attend CML Horizons 2012 in Munich last year and it was only on my journey to the hotel with delegates I met last year that I remembered just how intense the conference is. After exchanging pleasantries and accounts of weather in our various countries the topic soon landed on generic drugs and pricing. All of a sudden, after 8 hours travelling, I had to up my game. The names of drugs escaped my brain, numbers suddenly became random strings and I found myself working hard to keep up. The 160+ delegates will be bombarded with info over the forthcoming days, ready for us to disseminate to our respective countries when we get home. It’ll be good to see friends and spend time with the Midlands crew: Tony Gavin (from Leukaemia Care) and Nigel Deekes (who runs the CML-UK Facebook group).

I’m looking forward to delivering my talk on using the Internet for advocacy, that’s on Saturday, and hearing from a variety of people from around the world speak about their particular CML specialism. The conference will inspire, invigorate and drive many of us on again to continue campaigning, publicising and pressing the issues that need to be dealt with.

I’m going to try and blog at the end of each day with details of what we have heard. Feel free to ask questions or leave a comment, in fact I’d love you too.

All the best,

Kris

Speaking and remission

I had some good news recently. My CML is still on the retreat and currently at 0.005% – on the verge of being undetectable – this has taken 5 years. As well as my brilliant support network I have to very grateful for dasatinib and all of the other TKIs that are giving people their lives back. It spurs me on…

I’m very humbled to have been asked to speak at various events this year and I’ll keep you up-to-date with where I am and when. If you find yourself in the same place please come and say hi – you’ll find me looking somewhat bewildered wondering why on earth people want to hear me talk 🙂

12 March 2013 – Medical Management Services – Melanoma workshop – London – DETAILS

20 March 2013 – Medical Management Services – Melanoma workshop – Warrington – DETAILS

11 April 2013 – Leukaemia Care – Leukaemia Support Group – Worcester

13 April 2013 – Leukaemia and Lymphoma Research – Impact Day – London

25 April 2013 – Leukaemia Care – East Midlands CML Support Group – Leicester

3-5 May 2013 – CML Horizons (11th International) – Expert Marketplace: Using the Internet in advocacy – Prague, Czech Republic – DETAILS

15 June 2013 – Leukaemia Care – Annual Conference – Worcester

All my best wishes,

Kris

Report: deaths from Chronic Myeloid Leukaemia (CML) have halved

The National Cancer Intelligence Network (NCIN) have this week released a report which has shown that deaths from Chronic Myeloid Leukaemia have halved since TKIs were introduced in 2001.
The report states that “What’s even more promising is that, in the last four years, second and third generations of these drugs have been developed. We believe more and more CML patients have been receiving TKI’s and we’d predict that the improvements in survival should be even greater in the future” – However, we know of the challenges patients will face on long term access to dasatinib, especially with no firm long term commitment to the future of the CDF. If we take into account the success of these new drugs over the last 4 years I predict that survival figure will sky-rocket. More reason to ensure access to all of the drugs for everyone.
Kris

IMPROVED TREATMENTS FOR CHRONIC MYELOID LEUKAEMIA HAVE DRAMATICALLY INCREASED SURVIVAL

Survival for people diagnosed with Chronic Myeloid Leukaemia (CML) has risen by nearly half, with around 58 per cent of people surviving their disease for at least five years compared with only around 40 per cent in the late 1990s, according to a new report from the National Cancer Intelligence Network (NCIN), published today (Wednesday)*.

The improvements are largely down to a family of drugs called Tyrosine Kinase Inhibitors (TKIs) which have now become the standard treatment for the disease. The first of these was imatinib (Glivec), which was licensed in 2001.

The Northern and Yorkshire Cancer Registry and Information Service (NYCRIS), on behalf of the NCIN Haematology Site Specific Clinical Reference Group (SSCRG), looked at the rates of people in England getting, dying from and surviving a range of different blood cancers between 1995 and 2008. And it is the first national study in England to look at survival for different types of leukaemia.

For patients diagnosed with CML, researchers found that the chance of surviving the disease for at least five years after diagnosis rose from 41 per cent to 57 per cent in men and from 38 per cent to 59 per cent in women between the late 1990s and the early 2000s**.

CML is a relatively rare form of leukaemia*** that mostly affects older people, with around 700 cases diagnosed in the UK every year****.

Dr Robin Ireland, chair of the SSCRG at the NCIN, said: “It’s really exciting to see the enormous difference new drugs can make in treating cancer. And, as this new data shows, TKI’s can be considered a revolutionary treatment for Chronic Myeloid Leukaemia.”

“Basic research has given us a greater biological understanding of cancer tumours, which has led to the development of successful targeted cancer drugs that are now the first line treatment for CML. TKIs target cancer cells by blocking the molecules they make, which stops them from multiplying. These drugs have completely changed the outlook for patients with this disease and it’s the first example of our improved understanding of cell molecular biology leading to the design of a specific inhibitor of the disease.

”Dr Steven Oliver, Haematological Cancer Epidemiology Lead at NYCRIS and lead author of the report, said: “This report shows that, although the number of people developing Chronic Myeloid Leukaemia hasn’t changed much since 2001, survival from the disease has greatly improved.”

“What’s even more promising is that, in the last four years, second and third generations of these drugs have been developed. We believe more and more CML patients have been receiving TKI’s and we’d predict that the improvements in survival should be even greater in the future.”

“Chris Carrigan, head of the National Cancer Intelligence Network (NCIN), said: “Being able to link data on the diagnosis, treatment and outcomes for cancer patients allows us to identify where improved cancer care is having an effect on peoples lives. The improvements in survival demonstrated here highlight the difference that effective treatments can make.”

Notes

*Haematological Malignancies in England: cancers diagnosed 2001 – 2008.Note that analyses of deaths from blood cancers for 2001-2008 were also reported, along with 5-year survival figures for patients diagnosed with different blood cancers in 1995-1999 compared with those diagnosed in 2000-2003.

**Absolute change in CML 5-year survival rates, in England, 1995-1999 compared with 2000-2003. Male rates 40.7% and 56.9%; female rates 38.4% and 58.7%, in 1995-1999 and 2000-2003 respectively.

***Leukaemias are a group of malignant diseases that affect the production of white blood cells – the body’s guards against foreign cells and infections. The different types of leukaemia affect different organs and vary in how quickly they spread. Different types of leukaemia affect different age groups and there are some wide differences in survival found across the age groups in certain forms of the disease.

**** There were 710 cases of CML registered in 2010 in the UK (Cancer Research UK Statistical Information Team)The NICE guidance recommends standard-dose imatinib as the first-line treatment for CML.

Source: http://www.ncin.org.uk/news_and_events/improved_treatments_for_chronic_myeloid_leukaemia_have_dramatically_increased_survival.aspx

FDA approves Synribo for chronic myelogenous leukaemia

More fantastic news regarding another drug to treat CML. More hope for those who fail on other TKIs. We must ensure our UK health system funds drugs like this as they become available. Kris

The U.S. Food and Drug Administration today approved Synribo (omacetaxine mepesuccinate) to treat adults with chronic myelogenous leukemia (CML), a blood and bone marrow disease.

An estimated 5,430 people will be diagnosed with CML in 2012, according to the National Institutes of Health. Synribo is intended to be used in patients whose cancer progressed after treatment with at least two drugs from a class called tyrosine kinase inhibitors (TKIs), also used to treat CML.

Synribo blocks certain proteins that promote the development of cancerous cells. It is injected just under the skin (subcutaneously) twice daily for 14 consecutive days over a 28-day cycle until white blood cell counts normalize (hematologic response). Synribo is then administered twice daily for seven consecutive days over a 28-day cycle as long as patients continue to clinically benefit from therapy.

“Today’s approval provides a new treatment option for patients who are resistant to or cannot tolerate other FDA-approved drugs for chronic or accelerated phases of CML,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “Synribo is the second drug approved to treat CML in the past two months.”

On Sept. 4, 2012, the FDA approved Bosulif (bosutinib) to treat patients with chronic, accelerated or blast phase Philadelphia chromosome positive CML who are resistant to or who cannot tolerate other therapies.

Synribo is approved under the FDA’s accelerated approval program, which allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts additional clinical studies to confirm the drug’s clinical benefit and safe use. Synribo also received orphan-product designation by the FDA because it is intended to treat a rare disease or condition.

The effectiveness of Synribo was evaluated using a combined cohort of patients whose cancer progressed after previous treatment with two or more TKIs. All participants were treated with Synribo.

The drug’s effectiveness in chronic phase CML was demonstrated by a reduction in the percentage of cells expressing the Philadelphia chromosome genetic mutation found in most CML patients. Fourteen out of 76 patients (18.4 percent) achieved a reduction in an average time of 3.5 months. The median length of the reduction was 12.5 months.

In accelerated phase CML, Synribo’s effectiveness was determined by the number of patients who experienced a normalization of white blood cell counts or had no evidence of leukemia (major hematologic response, or MaHR). Results showed five out of 35 patients (14.3 percent) achieved MaHR in an average time of 2.3 months. The median duration of MaHR in these patients was 4.7 months.

The most common side effects reported during clinical studies include a low level of platelets in the blood (thrombocytopenia), low red blood cell count (anemia), a decrease in infection-fighting white blood cells (neutropenia) which may lead to infection and fever (febrile neutropenia), diarrhea, nausea, weakness and fatigue, injection site reaction, and a decrease in the number of lymphocytes in the blood (lymphopenia).

Synribo is marketed by Frazer, Pa.-based Teva Pharmaceuticals. Bosulif is marketed by New York City-based Pfizer.

For more information:

• FDA: Office of Hematology and Oncology Products
• FDA: Approved Drugs: Questions and Answers
• FDA: Drug Innovation
• NIH: Chronic Myelogenous Leukemia

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Source: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm325895.htm

New CML drug shows promising results – ponatinib

I always read stories about new drugs with great joy. That there are scientists and companies dedicated to developing drugs that keep us alive is very inspiring. The naysayers will quote share values and profit margins but all that matters to me are those 2 little tablets (dasatinib) that I take each morning that keep me alive.
If this product comes to market let’s hope NICE don’t stop access to this drug too.
Kris

Ariad Pharmaceuticals is to expand into Europe following promising results for an investigational cancer drug.

The US biotech firm will site a European HQ in Switzerland to support the planned filing of ponatinib, a pan-BCR-ABL inhibitor which has performed well in chronic myloid leukemia (CML) patients in clinical trials.

Talking to Pharmafocus, Ariad’s president of R&D and chief scientific officer Tim Clackson said the Swiss office would be open “imminently”.

Ponatinib is the Cambridge, Massachussetts-based oncology specialist’s lead candidate and is expected to be filed with US and European regulators before September this year.

If approved it would be up against tyrosine kinase inhibitors (TKIs) such as Novartis’ Glivec (imatinib), Novartis’ Tasigna (nilotinib) and Bristol-Myers Squibb’s Sprycel (dasatinib).

But Ariad is confident ponatinib’s mode of action is different from these brands as it bypasses T315I, the so-called ‘gatekeeper’ mutation in CML and can treat patients with all mutations – or none at all, a group Clackson says are “the most difficult to treat”.

The product was developed from the ground up with exactly that function in mind, part of Ariad’s use of computational chemistry approach which Clackson says brings in “the building blocks to sculpt the drug exactly as we want”.

Many CML sufferers develop resistance to TKIs over time, but in the recent PACE trial patients at all stages of the disease had a positive clinical response to ponatinib.

It saw 54% of patients achieving a major cytogenetic response (MCyR), with 44% a complete cytogenetic response (CCyR). The median follow-up for chronic CML patients is 10.1 months.

Of those patients with the T315I mutation, 70% achieved a MCyR with 66% a CCyR.

When it came to the higher bar of measurement, 30% of chronic-phase patients achieved a major molecular response (MMR), something 50% of chronic patients with the T315I mutation also attained.

A phase III trial pitting ponatinib against Glivec is planned for the third quarter of the year.

CML is a cancer of the white blood cells which affects around 7,000 patients each year in Europe. “T315I was a death sentence,” says Clackson. “Now there’s another approach.”

Switzerland chosen as European base

Ariad is currently building a sales force and commercial infrastructure in the US and will be mirroring it in Europe, with “small” sales teams in the UK and other key European markets.

There are no plans for any R&D to be based on the continent. “We’ll continue to manufacture at various third parties in the US and Europe,” Clackson said.

The Swiss facility will be in the canton of Vaud, where Ariad “will be looking to establish scientific connections with universities, research institutes and hospitals”.

Tax incentives were one of the key reasons, along with pharmacological expertise and a skilled workforce, for choosing Switzerland over other European locations, Clackson says.

Ariad’s most high profile drug discovery before this was oral mTOR inhibitor ridaforolimus, for which two years ago Merck acquired the exclusive license for oncology indications.

Ponatinib has also attracted the attention of other pharma companies but Ariad has decided to develop and commercialise the drug itself.

“We had incredible interest in partnering,” says Clackson. “We had very, very substantial offers on the table. But it was an unequivocal decision to go it alone.”

Ariad’s other pipeline products include AP26113, an oral, dual inhibitor of anaplastic lymphoma kinase (ALK) and epidermal growth factor receptor (EGFR), which is to be developed in non-small cell lung cancers.

The company also has AP1903, which is being evaluated in patients with prostate cancer and is licensed to Bellicum Pharmaceuticals.

Adam Hill
http://www.inpharm.com/news/173231/ariad-touts-next-generation-cml-treatment
Published on 29/06/12