Pharma Strategy Blog

Aggregating the EHA 2010 tweets

After the success of the American Society of Clinical Oncology (ASCO) Twitter aggregation, with over 250 readers over the course of the weekend, I thought it would be fun to repeat the experiment for the European Hematology Association (EHA) meeting in Barcelona this weekend.  The tweet volume is likely to be significantly less tough, with fewer people tweeting from the event.

Most of the topics I'm interested in will be on leukemias, lymphomas and myeloma, so if you are interested in these cancers, you can follow the feed below.  I've aggregated two hashtags, #EHA10 and #EHA2010. 

Additionally, Dr Anas Younes from MD Anderson Cancer Center will also be attending and following his tweet stream may also be instructive, especially if you are interested in his specialty, lymphomas.

Do feel free to join in remotely in the back channel and add links, comments or ask questions if you feel so inclined. The more the merrier!

I'm particularly looking forward to the Patient Advocacy symposium chaired by Jan Geissler, an awesome guy who just happens to have CML.  While the science and clinical data are important, it's also critical to hear and learn from the advocacy viewpoint.  

On the healthcare (#hcsmeu) front, I'm also hoping to meet fellow Twitter buddies, Miguel and Angel for an impromptu tweetup of #hcsmeuES.  If you're on Twitter and interested in healthcare, check them all out, all three are great gentlemen I would highly recommend!

Managing Multiple Myeloma: turning an acute cancer into a chronic disease

The other day while travelling home on a long train journey, I was browsing the NY Times app on my iPhone and came across an interesting story about multiple myeloma in the Health section:

"For many patients with cancers like chronic lymphoma, chronic myelocytic leukemia and now multiple myeloma, longevity lies in the ability of science to remain one step ahead of the malignancy by unraveling its genetic and molecular underpinnings and producing treatments tailored to counter them."

Now, I don't think there is actually something called chronic lymphoma (non-Hodgkins lymphoma (NHL), chronic lymphocytic leukemia (CLL) and chronic myeloid leukemia (CML), yes), but you get the gist of what the reporter is saying - acute deadly hematologic cancers are gradually being turned into chronic diseases.  

The main reasons behind this are several fold:

1. Greater understanding of the science and biology underlying the diseases.
2. Increased number of available therapies that can be sequenced, prolonging overall survival.
3. Move from chemotherapy to more targeted therapies, improving quality of life.
4. Active drug pipeline in myeloma across several companies, offering increased hope for further advancements in the disease as new combinations evolve.
5. Updating of treatment guidelines and new standards (e.g. see this Myeloma example)

In addition, the growth of Myeloma support groups has provided a wealth of online information, practical advice, and emotional support for people suffering with the disease. Some great resources include:

1. Multiple Myeloma Research Foundation (run by an incredible myeloma survivor, Kathy Giusti)
2. International Myeloma Foundation
3. Leukemia and Lymphoma Society
4. National Organisation of Rare Diseases
5. American Cancer Society

In addition, Pharma companies such as Millennium, Celgene and Onyx/Proteolix are also doing their bit to improve research and outcomes in multiple myeloma in R&D, and some provide people with balanced and fair disease information - not everything has to be promotional.  One such example is an unbranded site about Myeloma supported by Millennium-Takeda, the manufacturers of bortezomib (Velcade) called My Multiple Myeloma.  Velcade is a proteasome inhibitor that has become the cornerstone of front-line therapy either without or before a stem cell transplant.

Now, let's take a look at the My Multiple Myeloma website. I should disclose here that the agency who are responsible for building the site are a client, although I wasn't involved in the website project itself.  I've long had an interest in unbranded sites and disease information from my marketing days, so let's look at what's available at the moment.  

The opening site page offers some sensible choices with clear navigation encouraging people to take charge of their disease and some shots of real people who are experiencing multiple myeloma, you can click through and read more about their stories and experiences:

One of the things that many people forget is that cancer is a complex disease, not only in terms of treatment options, but all the other factors that need to be considered.  Googling for information can be overwhelming and none of the current advocacy sites offer a one-stop shop for everything.  The site looks to reduce this overload and provide a central starting repository for information.  Further into My Multiple Myeloma, you can find some other interesting and relevant information such as coping with the disease, working with the health care professionals involved, costs and insurance, understanding emotions, lifestyle advice, and where to find clinical trials for example:

It will be interesting to see how the site evolves over time and hopefully adds useful tools to allow people to engage and share information with each other.  Providing relevant information is a good start, albeit a static one, but allowing interactivity and sharing of ideas will help bond those affected even more.  

It would be cool to add other practical things such as a wiki for transplant centers, contacts, and other academic centers specialising in the treatment of myeloma, for example.  That way, people can find a hospital near them, or even a means of finding local support groups perhaps.

In the past, I have seen significant value in watching patients converse, help and support each other in disease forums because many-to-many is so much more powerful and effective than an isolated n of 1. People need to feel that they are not alone in their fight against cancer, having a sense of support and community really does help mobilise heart and desire to beat the disease. 

It always annoys me when people think Pharma companies are only interested in promotional or DTC programs that make money or add ROI; in oncology and hematology, I've yet to meet someone from the industry who didn't care about people, improving survival and outcomes or making a difference to people's lives. 

Maybe cancer is just a different world... after all, we have very few placebo controlled trials in this arena; new therapies and combinations typically go head to head against the standard of care in a survival of the fittest approach.  May the best ones win and make a difference, which is great news for people suffering from any cancer.  

Hope is one thing, but improved outcomes and information are even better.

Kumar, S., Mikhael, J., Buadi, F., Dingli, D., Dispenzieri, A., Fonseca, R., Gertz, M., Greipp, P., Hayman, S., Kyle, R., Lacy, M., Lust, J., Reeder, C., Roy, V., Russell, S., Short, K., Stewart, A., Witzig, T., Zeldenrust, S., Dalton, R., Rajkumar, S., & Bergsagel, P. (2009). Management of Newly Diagnosed Symptomatic Multiple Myeloma: updated Mayo Stratification of Myeloma and Risk-Adapted Therapy (mSMART) Consensus Guidelines Mayo Clinic Proceedings, 84 (12), 1095-1110 DOI: 10.4065/mcp.2009.0603

Update on Multiple Myeloma: are molecular remissions possible without a transplant?

By strange coincidence this morning I was listening in to the Celgene R&D Day presentations including an update on their plans for their iMiD therapies thalidomide (Thalomid) and lenalidomide (Revlimid), while reading a couple of interesting papers about Millennium-Takeda's bortezomib (Velcade) that were just published in the Journal of Clinical Oncology last month and another this week for the treatment of multiple myeloma. All three of these drugs have changed the treatment of multiple myeloma from a certain death sentence to a treatable disease.

I'll post more about the Celgene pipeline in a later post, but the papers are well worth sharing and discussing.  One report looked at the phase III data for the VISTA (Velcade as Initial Standard Therapy in Multiple Myeloma) trial.  This study was presented at the recent American Society of Hematology (ASH) meeting in December (see previous post), but the paper now includes updated survival data in a large cohort of 682 people:  

"With a median follow-up of 36.7 months, there was a 35% reduced risk of death with VMP versus MP (hazard ratio, 0.653; P < .001); median OS was not reached with VMP versus 43 months with MP; 3-year OS rates were 68.5% versus 54.0%.  Response rates to subsequent thalidomide (41% v 53%) and lenalidomide based therapies (59% v 52%) appeared similar after VMP or MP; response rates to subsequent bortezomib-based therapy were 47% versus 59%. 

Among patients treated with VMP (n = 178) and MP (n = 233), median survival from start of subsequent therapy was 30.2 and 21.9 months, respectively, and there was no difference in survival from salvage among patients who received subsequent bortezomib, thalidomide, or lenalidomide. Rates of adverse events were higher with VMP versus MP during cycles 1 to 4, but similar during cycles 5 to 9. With VMP, 79% of peripheral neuropathy events improved within a median of 1.9 months; 60% completely resolved within a median of 5.7 months."

The bold for emphasise is mine, but anytime you see a greater than 30% reduced risk of death from a treatment, one is bound to sit up and take notice, especially if you have a loved one suffering from the disease.  

What do these results mean?  Well, in practice they clearly demonstrate that VMP significantly prolongs OS versus MP after lengthy follow-up and extensive subsequent therapy for myeloma.  What was particularly encouraging is that first-line bortezomib use does not induce more resistant relapse, which is also important for people sufffering from the disease. 

The combination of VMP used upfront therefore appears more beneficial than first treating with conventional agents such as melphalan and prednisone and saving bortezomib and other novel therapies until relapse.   Historically, oncologists often leave the big guns in their back pocket, but the data from this trial may well change their thinking about treatment strategies in multiple myeloma, at least.

The second paper examined the effect on minimal residual disease, by qualitative and real-time quantitative polymerase chain reaction (RQ-PCR), of a consolidation regimen that included the combination of bortezomib (Velcade), thalidomide (Thalomid), and dexamethasone (VTD) in people with multiple myeloma who responded to autologous stem-cell transplantation (auto-SCT).  Although the results are more preliminary - this was a phase II trial with only a small number of patients (n=39) - they are very encouraging indeed.  

This study is particularly important because it may be the first to document the occurrence of persistent molecular remissions (almost undetectable disease) in a proportion of people with multiple myeloma treated without allogeneic transplantation.  To put this is context, until now, a transplant was the only known curative treatment that has been shown to induce molecular remission, so to achieve that state with therapy alone is something that will give many people goosebumps.

Why?

Because SCT are associated with 20% treatment related mortality alone, so if that number can be reduced by excluding the risk of death, then it offers hope not just for those who are ineligible for a transplant, but as another option for those who are.

The researchers also noted that:

"The major reduction in tumor load recorded by RQ-PCR after VTD suggests that unprecedented levels of tumor cell reduction can be achieved in MM thanks to the new nonchemotherapeutic drugs."

That's all sorts of awesome news, albeit in a small study, but it does augur well for the future if the results can be repeated in a phase III study.

Ladetto, M., Pagliano, G., Ferrero, S., Cavallo, F., Drandi, D., Santo, L., Crippa, C., De Rosa, L., Pregno, P., Grasso, M., Liberati, A., Caravita, T., Pisani, F., Guglielmelli, T., Callea, V., Musto, P., Cangialosi, C., Passera, R., Boccadoro, M., & Palumbo, A. (2010). Major Tumor Shrinking and Persistent Molecular Remissions After Consolidation With Bortezomib, Thalidomide, and Dexamethasone in Patients With Autografted Myeloma Journal of Clinical Oncology DOI: 10.1200/JCO.2009.23.7172 

Mateos, M., Richardson, P., Schlag, R., Khuageva, N., Dimopoulos, M., Shpilberg, O., Kropff, M., Spicka, I., Petrucci, M., Palumbo, A., Samoilova, O., Dmoszynska, A., Abdulkadyrov, K., Schots, R., Jiang, B., Esseltine, D., Liu, K., Cakana, A., van de Velde, H., & San Miguel, J. (2010). Bortezomib Plus Melphalan and Prednisone Compared With Melphalan and Prednisone in Previously Untreated Multiple Myeloma: Updated Follow-Up and Impact of Subsequent Therapy in the Phase III VISTA Trial Journal of Clinical Oncology DOI: 10.1200/JCO.2009.26.0638

Aeterna Zentaris and Keryx's Perifosine gets fast track designation for colorectal cancer

Last month at the ASCO GI Symposium, Keryx and Aeterna Zentaris reported statistically significant benefit in survival from updated results of a randomized, double-blind, placebo-controlled phase II study of KRX-0401 (perifosine) for the treatment of advanced metastatic colorectal cancer. 

The study was based on 35 evaluable, but heavily pre-treated patients, with relapsed or refractory metastatic colon cancer were randomized to receive capecitabine (Xeloda) from Roche at 825 mg/m2 BID on days 1 - 14 every 21 days plus either perifosine (P-CAP) or placebo at 50 mg daily (CAP).

The results showed that the P-CAP arm had 1 PR and 8 SD for an overall response rate of 64%, while the CAP arm had 0 PR and only 3 SD for a response rate of 27%.

In addition, looking at the overall survival, the data favoured the P-CAP arm a MOS of 15.3 weeks compared with 6.8 weeks for CAP alone.

Typical adverse events reported appeared to be relatively mild:

"The P-CAP combination was well-tolerated with Grade 3 and 4 adverse events of > 10% incidence for the P-CAP arm versus CAP arm as follows: anemia (15% vs. 0%), fatigue (0% vs. 11%), abdominal pain (5% vs. 11%), and hand-foot syndrome (30% vs. 0%)."

Perifosine is also being developed for the treatment of multiple myeloma, which is currently in phase III development.  The compound is an inhibitor of Akt and phosphoinositide 3-kinase (PI3K), as well as other pathways such as JNK and MAPK. These pathways are associated with programmed cell death (apoptosis), cell growth, cell differentiation and cell survival as shown in the diagram below:

Source: Aeterna Zentaris

Interestingly, this morning's news brought an announcement that the FDA have granted Fast Track Designation for the perifosine in advanced, refractory colorectal cancer.  Based on the phase II data, a randomised phase III trial is expected to begin this quarter based on a similar trial design.

Clearly, it will be a little while before the data is available, but definitely one to watch out for in the future given new therapy options are always needed in the refractory cancer setting.

#ASH09 Update: Multiple Myeloma

In the past, single agent and double agent regimens have been a mainstay in the treatment of multiple myeloma, yet the five year survival rate has remained steadfastly low around 3-4 years, on average. Typically, younger and fitter patients do better than elderly patients with a poorer performance status, yet the majority of new diagnoses are found in the elderly.  Finding new treatment approaches is therefore a key imperative.

2009 has seen the advent of new triple, and even quadruple, combinations in an effort to improve efficacy and survival, hopefully not at the expense of increased side effects. Two of the most commonly used drugs, bortezomib and lenalidomide, for example, differ in their side effect profile with an increased tendency to peripheral neuropathy and deep venous thrombosis, respectively.  New dosing regimens have begun to look at new strategies in the form of modified dosing regimens, which are potentially more patient friendly, without compromising efficacy.

Important trial results from two European groups at this American Society of Hematology (ASH) meeting, for example, demonstrated that a four drug combination improved durable responses and progression-free survival in elderly patients.  Both studies showed that a weekly schedule of bortezomib (Velcade) maintained efficacy with fewer toxic side effects (i.e. significantly decreased the incidence of grade 3-4 peripheral neuropathy) compared to the standard twice weekly schedule.

In newly diagnosed myeloma patients the combination of bortezomib with melphalan-prednisone (VMP) has been previously shown to be superior to MP, while in relapsed-refractory patients the four drug combination bortezomib-melphalan-prednisone-thalidomide (VMPT) induced a high proportion of complete responses (CR).

An Italian phase III study therefore looked at the four drug combination in the upfront setting and showed that induction therapy with a four drug combination of bortezomib, melphalan, prednisone, and thalidomide (VMPT) followed by maintenance therapy with bortezomib and thalidomide (VT) achieved superior response rates and PFS compared to VMP.

A Spanish study noted that previously in elderly patients with newly diagnosed myeloma, the VISTA trial demonstrated that the combination of bortezomib plus melphalan – prednisone (VMP) is significantly superior to MP alone.  However, it is unclear which agent is the optimal partner for bortezomib: an alkylating agent or an immunomodulatory drug, so they set out to answer this critical question by comparing VMP to VTP, where T is thalidomide. 

The results indicated that:

"1. Both modified induction schedules (VMP and VTP) are highly effective with similar ORR and CR rates, but a clear different toxicity profile (more neutropenia, but less cardiac toxicity and peripheral neuropathy with VMP)

2. Maintenance therapy with either VT and VP markedly improve the quality of responses with a good safety profile

3. The combination of these induction and maintenance schedules seems to overcome the poor prognosis of high-risk cytogenetic abnormalities in elderly MM patients"

It should be noted that while melphalan causes more neutropenia, the cardiac toxicity seen with thalidomide is more difficult to manage, so the VMP combination is likely to be more suitable going forward in newly diagnosed elderly patients with myeloma.

One triple combination that garnered a lot of interest earlier this year at ASCO, was a trial looking at combining lenalidomide (Revlimid), bortezomib (Velcade) and dexamethasone (RVD) in relapsed or refractory myeloma patients, with overall response rates (ORR) of 69%, including 26% complete/near complete responses (CR/nCR) and manageable toxicities Anderson et al. ASCO 2009). The phase I portion of the study (Richardson et al. IMW 2009) found the maximum tolerated dose (MTD) of this combination in newly diagnosed myeloma patients to be lenalidomide (25 mg/day), bortezomib (1.3 mg/m²), and dexamethasone (20 mg). In all phase I patients, the ORR was 100%, including 31% CR, 9% nCR, and 75% very good PR (VGPR). 

At this ASH meeting, we heard results reported for patients treated in the phase II portion of the study as conditioning prior to autologous stem cell transplantation (ASCT) by Dr Richardson, who concluded that:

"These phase II results suggest that RVD is a highly effective combination, with a pre-ASCT ORR of 100% and high rates of CR/nCR, and encouraging time-to-event analyses to date. RVD was well tolerated, with limited rates of grade 3 peripheral neuropathy and DVT/PE despite prolonged use of bortezomib and lenalidomide." 

The data are summarised in the table below, based on all patients (n=35):

Patients	ORR, %	CR/nCR, %	≥VGPR, %
Response at cycle 4 (n=31)	78	12	12
Response at cycle 8 (n=24)	100	33	67
Best response	100	54	69

Some of the most exciting new developments in myeloma could be found in both the oral and poster sessions. 

A new generation proteasome inhibitor, carfilzomib (PX-171), has been generating consistent results in the bortezomib refractory setting.  An ongoing phase II study presented at ASH looked at carfilzomib monotherapy in myeloma patients with relapsed or refractory disease following 1–3 prior therapies, ie a heavily pre-treated population.  Updated data for the bortezomib-treated cohort were reported at ASH and the authors concluded that:

"18% ORR (CBR 30%) is notable for this steroid- and anthracycline-sparing regimen. Single-agent carfilzomib is well tolerated, even in patients with renal insufficiency, and both myelosuppression and peripheral neuropathy are uncommon." 

Carfilzomib was originally developed by Proteolix, who have just been acquired by Onyx.  This should ensure more solid funding for the development of the phase III program, which looks promising in the relapsed, refractory setting.

Perifosine (Keryx) modulates Akt, and a number of other signal transduction pathways, including the JNK and MAPK, all of which are associated with programmed cell death, cell growth, cell differentiation and cell survival.  Updated data from a phase I/II study in 84 patients who were heavily pre-treated with a median of 5 prior lines of therapy (range 1-13), showed that the overall response rate (ORR) was 41%, for the bortezomib relapsed group, 65%, and for the bortezomib refractory group, 43%. 

Approximately 60% of patients demonstrated progression (or stable disease (SD) for 4 cycles) at some point in their treatment and received 20 mg dexamethasone, four times per week, in addition to perifosine plus bortezomib.  Responses occurred both with patients taking perifosine in combination with bortezomib and with patients receiving the combination plus dexamethasone.  The ORR for the perifosine plus bortezomib arm was 25%, with an increase to 38% when dexamethasone was added.  Survival data in the 73 evaluable patients was 6.4 months for PFS and 25 moths for overall survival (OS).

Another exciting new compound in development is MLN4924 from Millennium, the novel mechanism of which was recently described in detail (see article below) and was the also subject of a Nature article.  The compound works very differently from proteasome inhibitors, which target the proteasome substrate.  MLN4924 is involved with neddylation rather than direct ubiquination. 

Neddylation activating enzyme (NAE) is the E1-activating enzyme for the ubiquitin-like protein Nedd8.  NAE catalyzes the first step in the neddylation cascade leading to modification of cullin-based ubiquitin ligase activity.  This results in specific protein substrates, with important roles in cancer cell survival, being targeted for degradation as shown the diagram below:

Source: Soucy et al., 2009

The end result is that MLN4924 inhibits neddylation by disrupting the cullin-RING ligase-mediated protein turnover, leading to apoptotic death in human tumour cells by a new mechanism of action, the deregulation of S-phase DNA synthesis.

A phase I dose escalation study in myeloma and NHL patients with MLN4924 was reported at ASH.  The primary objectives were to determine the maximum tolerated dose (MTD) and safety profile of MLN4924, as well as describe the pharmacokinetics (PK) and pharmacodynamics (PD) in blood.  Among the 22 patients enrolled to date, the median age was 65 years, 14 had myeloma and 8 had NHL. 

The results showed that NEDD8-Cullin levels in peripheral blood myeloma cells (PBMCs) were inhibited and Nrf-2 target gene transcripts in whole blood were higher vs. baseline after MLN4924 administration, which is indicative of NAE inhibition.  Cdt-1 and Nrf-2 levels in skin increased above baseline following the second dose of MLN4924, which is indicative of NAE inhibition in peripheral tissue.  These results are promising and provide early proof of concept for the role of neddylation in myeloma.

Overall, this was a good meeting on the myeloma front with lots of promise for triple and quadruple combinations, as well as new agents being developed in both the newly diagnosed and refractory settings that augers well for the future, both in terms of improved survival and also more tolerable regimens.

Soucy, T., Smith, P., & Rolfe, M. (2009). Targeting NEDD8-Activated Cullin-RING Ligases for the Treatment of Cancer Clinical Cancer Research, 15 (12), 3912-3916 DOI: 10.1158/1078-0432.CCR-09-0343

Soucy, T., Smith, P., Milhollen, M., Berger, A., Gavin, J., Adhikari, S., Brownell, J., Burke, K., Cardin, D., Critchley, S., Cullis, C., Doucette, A., Garnsey, J., Gaulin, J., Gershman, R., Lublinsky, A., McDonald, A., Mizutani, H., Narayanan, U., Olhava, E., Peluso, S., Rezaei, M., Sintchak, M., Talreja, T., Thomas, M., Traore, T., Vyskocil, S., Weatherhead, G., Yu, J., Zhang, J., Dick, L., Claiborne, C., Rolfe, M., Bolen, J., & Langston, S. (2009). An inhibitor of NEDD8-activating enzyme as a new approach to treat cancer Nature, 458 (7239), 732-736 DOI: 10.1038/nature07884

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How the costs of cancer care negatively impact patients

Pharma Strategy Blog has reached an exciting double milestone today - 500 posts and 100,000 views!  Although the blog was started in 2006, most of the posts have been written over the last 18 months on a wide variety of topics from AIDS/HIV to cancer to rare stromal tumours, as well as notes from numerous conferences and observations on diseases, the Pharma industry, mergers & acquisition, industry leaders and even political reform of health care.  We hope you have all enjoyed the ride and will continue to do so in 2010.  Do keep your suggestions for interesting topics to cover coming in, we're only as good as the readers who share in the experience.

Right now, I'm in New Orleans covering the American Society of hematology meeting, which is one of my favourite annual meetings, and one I've regularly attended for the last 14 years.  Just before I headed south I interviewed Dr Mary Ann Burg about the recent survey undertaken by the Association of Social Work (ASOW).  The organisation released an important report late last week, describing the financial and emotional impact of the cost of cancer care on patients, caregivers and how social workers can help facilitate and ease the process as part of a multi-factorial team.

What particularly struck me forcefully in the brief discussion with Dr Burg is summarised in this chart below:

The excellent AOSW report also focused on multiple myeloma as an example of what happens to cancer patients going through treatment and how the costs of prescription drugs can have an impact.  For example:

This data reminded me very clearly how much more expensive Celgene's Revlimid is compared to Millennium's Velcade (around a third to twice the cost).  Myeloma is a big focus at this ASH, as you will see in other posts this week around the new exciting data coming out in lymphomas, leukemias and myeloma.

The report was also poignant given that the WSJ reported this morning that Allos Therapeutics were launching pralatrexate (Folotyn) in PTCL, a rare form of NHL, at a cost of $30,000 per month.  To me, that's unconscionable, especially as the drug has only be seen to shrink tumours and not impact overall survival.  We'll see how they fare, but I think I would rather die with my dignity intact than face the sort of extreme stress and financial worries described in the excellent ASOW report than even the co-pay would require.  It does beg the question of:

1. What price a life?
2. What were they thinking?

Part of being a sensible corporate citizen in the pharma industry is recognising that we all have a duty to be fair minded, responsible and caring; greed in it's extreme form at the expense of patients well being is just nauseous and reprehensible.

Here's a rare call for action. 

Please read the AOSW information here and digest the full impact of their findings for yourself.  This affects all of us, as industry professionals, friends, caregivers, maybe even as a patient.  Share  it with others, if you blog or Tweet, write something about the report yourself or share this post or the ASOW link.

More on the ASH meeting and more about the AOSW will continue in other posts later this week.

Onyx Pharma and Proteolix look to tie the knot

After the furore around Pfizer and figitumumab on Friday, this morning brought entirely different kind of news with Onyx Pharma seeking to purchase a small biotech based in South San Francisco called Proteolix.  Regular readers of this blog will be familiar with previous notes on Proteolix's proteasome inhibitor, PR-171 or carfilzomib, which will potentially be a major competitor to Millennium's Velcade (bortezomib) if it is approved by the regulatory authorities.

Onyx already have sorafenib (Nexavar), on the market for several VEGF-mediated cancers such as renal cell cancer and hepatocellular (liver) carcinoma.  They are under increasing competition from Pfizer's sunitinib (Sutent) in the same cancer types, as well as 4 other therapies recently approved in renal cancer, including Novartis's everolimus (Afinitor).  It therefore makes sense to expand the product pipeline and replenish it with relatively late stage and interesting compounds.

There may be new phase II data in multiple myeloma and possibly even in non-Hodgins lymphoma (NHL) presented at the forthcoming American Society of Hematology (ASH) meeting in December and early data from the phase IIb trial is expected next year.  Current therapies for myeloma are fairly effective, but are limited by neurotoxicity issues.  It will be interesting to see if carfilzomib has an improved safety or efficacy profile based on the new data.  The early data was very promising, but as with all R&D, larger scale clinical trials are always necessary to see what happens in a larger pool of patients and whether the responses are durable over time.

If the results hold up, then this could well be a good investment for Onyx, because it expands their pipeline with new agents and different tumour targets.  It's also good to see the biotech companies getting smart about combining resources in terms of R&D and marketing muscle.  Overall this deal looks a win-win for both parties.

Spreading the risk is no bad thing sometimes.

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